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## Protocol Section
### Identification Module
**NCT ID:** NCT02536079
**Acronym:** NeST
**Brief Title:** NeuroAiD Safe Treatment Registry
**Official Title:** NeuroAiD Safe Treatment Registry
#### Organization Study ID Info
**ID:** NeST2014
#### Organization
**Class:** OTHER
**Full Name:** CHIMES Society
### Status Module
#### Completion Date
**Date:** 2025-12
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2023-02-17
**Type:** ACTUAL
**Last Update Submit Date:** 2023-02-15
**Overall Status:** RECRUITING
#### Primary Completion Date
**Date:** 2024-12
**Type:** ESTIMATED
#### Start Date
**Date:** 2014-06
**Status Verified Date:** 2023-02
#### Study First Post Date
**Date:** 2015-08-31
**Type:** ESTIMATED
**Study First Submit Date:** 2015-08-26
**Study First Submit QC Date:** 2015-08-26
### Sponsor Collaborators Module
#### Collaborators
**Class:** INDUSTRY
**Name:** Moleac Pte Ltd.
#### Lead Sponsor
**Class:** OTHER
**Name:** CHIMES Society
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** The NeST registry is a pro-active industry-academic collaboration to assess the use and safety of NeuroAiD in the real world setting. An online entry system was set up to allow easy data entry and retrieval of clinical information.
**Detailed Description:** The NeST Registry is designed as a product registry that would provide information on the use and safety of NeuroAiD in clinical practice. An online NeST Registry was set up to allow easy entry and retrieval of essential information including demographics, medical conditions, clinical assessments of neurological, functional, and cognitive state, compliance, concomitant medications and side effects if any, among patients on NeuroAiD. Participation is voluntary. Data collected are similar to information obtained during standard care and are prospectively entered by the participating physicians at baseline (before initialization of NeuroAiD) and during subsequent visits. The follow up visits are timed with clinical appointments. Anonymized data will be extracted and collectively analyzed. Initial target sample size for the registry is 2000.
### Conditions Module
**Conditions:**
- Brain Injury
- NeuroAiD Use
### Design Module
#### Design Info
**Observational Model:** COHORT
**Time Perspective:** PROSPECTIVE
#### Enrollment Info
**Count:** 2000
**Type:** ESTIMATED
**Patient Registry:** True
**Study Type:** OBSERVATIONAL
**Target Duration:** 3 Months
### Arms Interventions Module
### Interventions
#### Intervention 1
**Name:** NeuroAiD
**Other Names:**
- MLC601
- MLC901
**Type:** DRUG
### Outcomes Module
#### Other Outcomes
**Description:** Compliance assessed as "since last visit, taking NeuroAiD..." never, 'rarely,' 'sometimes,' 'often,' 'always.'
**Measure:** Number of patients complying to prescribed dosage of NeuroAiD
**Time Frame:** 1, 2, 3 months
**Description:** For Traumatic Brain injury cohort:This clinical scale is the most widely used outcome measure after TBI. The extended version is rated from 0 (dead) to 8 (full recovery)
**Measure:** Glasgow Outcome Scale Extended (GOS-E)
**Time Frame:** 1, 2, 3 months, additional visit
**Description:** For Traumatic Brain Injury Cohort. This questionnaire is a simple, 10-item measure of outcome mainly used with patients with mild to moderate head injuries. This questionnaire explores the occurrence of 16 post-concussive symptoms such as headaches, fatigue, and restlessness
**Measure:** Rivermead Post-Concussion Symptom Questionnaire (RPQ)
**Time Frame:** 1, 2, 3 months, additional visit
**Description:** For Traumatic Brain Injury cohort. This scale assesses functional disability by quantifying patient performance in 10 activities of daily life (ADLs). This scale measures performance and patient independence with respect to self-care, sphincter management, transfers and locomotion. The score is calculated by summing the response value to each item.
**Measure:** Modified Barthel Index
**Time Frame:** 1, 2, 3 months, additional visit
#### Primary Outcomes
**Description:** Adverse events categorized according to individual events, organ system, severity (mild, moderate, severe), and relatedness (based on WHO-World Health Organization - Uppsala Monitoring System as 'possibly' 'probably' or 'definitely' related)
**Measure:** Number of patients experiencing adverse events
**Time Frame:** 3 months
#### Secondary Outcomes
**Description:** 0 No symptoms at all
1. No significant disability despite symptoms; able to carry out all usual duties and activities
2. Slight disability; unable to carry out all previous activities, but able to look after own affairs without assistance
3. Moderate disability; requiring some help, but able to walk without assistance
4. Moderately severe disability; unable to walk without assistance and unable to attend to own bodily needs without assistance
5. Severe disability; bedridden, incontinent and requiring constant nursing care and attention
6. Dead
**Measure:** Functional status based on modified Rankin Scale
**Time Frame:** 1, 2, 3 months
**Description:** to assess level of consciousness: eye opening, Eye opening 4, Eye Opening Response
* Spontaneous--open with blinking at baseline 4 points
* To verbal stimuli, command, speech 3 points
* To pain only (not applied to face) 2 points
* No response 1 point;Verbal Response
* Oriented 5 points
* Confused conversation, but able to answer questions 4 points
* Inappropriate words 3 points
* Incomprehensible speech 2 points
* No response 1 point;Verbal Response
* Oriented 5 points
* Confused conversation, but able to answer questions 4 points
* Inappropriate words 3 points
* Incomprehensible speech 2 points
* No response 1 point;Motor Response
* Obeys commands for movement 6 points
* Purposeful movement to painful stimulus 5 points
* Withdraws in response to pain 4 points
* Flexion in response to pain (decorticate posturing) 3 points
* Extension response in response to pain (decerebrate rigidity )
**Measure:** Neurological status based on Glasgow Coma Scale
**Time Frame:** 1, 2, 3 months
**Description:** Quantify impairment caused by stroke 0- no stroke, 1-4 Minor stroke, 5-15 Moderate,16-20 Moderate to Severe stroke, 21-42 severe stroke
**Measure:** Neurological status based on National Institute of Health Stroke Scale
**Time Frame:** 1, 2, 3 months
**Description:** test involves 6 items test -1.what year is it now?2.What month is it now?3.Repeat phrase, about what time is it ? (within one hour),Count backwards 20 to 1,Say months in reversw order,repeat phrase just given,
**Measure:** Cognitive status based on Short Orientation-Memory-Concentration Test
**Time Frame:** 1, 2, 3 months
**Description:** Orientation, Registration,Attention and Calculation,Recall, Language, Copying,
**Measure:** Cognitive status based on Mini-Mental State Examination (MMSE)
**Time Frame:** 3, 6,9, 12 months
**Description:** World recall, Naming of objects and fingers,Commands,Constructional Praxis,Ideational Praxis,Orientation, World Recognition,Language,Comprehension of spoken language,Word finding difficulty, Remembering test instructions
**Measure:** Cognitive status based on Alzheimer's Disease Assessment Scale- cognitive subscale (ADAS-cog)
**Time Frame:** 3, 6, 9, 12 months
**Description:** Visio spatial /executive,naming, memory ,attention , language,abstraction, delayed recall
**Measure:** Cognitive status based on Montreal Cognitive Assessment (MoCA)
**Time Frame:** 3, 6, 9, 12 months
**Description:** Preparing Food 3 Eating 3 Preparing Drink 3 Drinking 3 Dressing 3 Hygiene 3 Teeth 3 Bath/Shower 3 Toilet/Commode 3 Transfers 3 Mobility 3 Orientation - Time 3 Orientation - Space 3 Communication 3 Telephone 3 Housework/Gardening 3 Shopping 3 Finances 3 Games/Hobbies 3 Transport 3
**Measure:** Functional outcome based on Bristol Activities of Daily Living (BADL)
**Time Frame:** 3, 6, 9, 12
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Male or female
* Any age
* Taking, or has been prescribed NeuroAiD for any duration as judged by the physician and/or the participant
* Agrees to be included in the registry and allows retrieval and analysis of data in accordance with local requirements
Exclusion Criteria:
* Unwillingness to participate
* Contraindication to NeuroAiD
**Sampling Method:** NON_PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- CHILD
- ADULT
- OLDER_ADULT
**Study Population:** The NeST Registry is a product-specific and safety outcome registry that includes patients who are taking NeuroAiD. Participation is entirely voluntary and an agreement will be obtained before inclusion. The decision on the use of NeuroAiD should be made following a discussion between participant and physician and only then will the option of participation in the registry be considered.
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Emily Lim
**Phone:** +6562113710
**Phone Ext:** 3184
**Role:** CONTACT
**Contact 2:**
**Email:** [email protected]
**Name:** Sylvain Durrleman, PhD
**Role:** CONTACT
#### Locations
**Location 1:**
**City:** Jakarta
**Contacts:**
***Contact 1:***
- **Name:** Lyna Soertidewi
- **Role:** CONTACT
**Country:** Indonesia
**Facility:** National Brain Center Hospital
**Status:** RECRUITING
**Location 2:**
**City:** Kuala Lumpur
**Contacts:**
***Contact 1:***
- **Name:** Ramesh Kumar
- **Role:** CONTACT
**Country:** Malaysia
**Facility:** University Kebangsaan Malaysia Medical Centre
**Status:** RECRUITING
#### Overall Officials
**Official 1:**
**Affiliation:** Raffles Neuroscience Centre, Raffles Hospital, Singapore
**Name:** Narayanaswamy Venketasubramanian, FRCP
**Role:** STUDY_CHAIR
**Official 2:**
**Affiliation:** Department of Neurosurgery, Faculty of Medicine, University Kebangsaan Malaysia Medical Centre, Kuala Lumpur, Malaysia
**Name:** Ramesh Kumar, FRCS
**Role:** PRINCIPAL_INVESTIGATOR
**Official 3:**
**Affiliation:** Department of Neurology, National Brain Center Hospital, Jakarta, Indonesia
**Name:** Lyna Soertidewi, MD
**Role:** PRINCIPAL_INVESTIGATOR
### References Module
#### References
**Citation:** Venketasubramanian N, Kumar R, Soertidewi L, Abu Bakar A, Laik C, Gan R. The NeuroAiD Safe Treatment (NeST) Registry: a protocol. BMJ Open. 2015 Nov 13;5(11):e009866. doi: 10.1136/bmjopen-2015-009866.
**PMID:** 26567259
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000001927
- Term: Brain Diseases
- ID: D000002493
- Term: Central Nervous System Diseases
- ID: D000009422
- Term: Nervous System Diseases
- ID: D000006259
- Term: Craniocerebral Trauma
- ID: D000020196
- Term: Trauma, Nervous System
- ID: D000014947
- Term: Wounds and Injuries
### Condition Browse Module - Browse Branches
- Abbrev: BC10
- Name: Nervous System Diseases
- Abbrev: BC26
- Name: Wounds and Injuries
- Abbrev: All
- Name: All Conditions
### Condition Browse Module - Browse Leaves
- ID: M5207
- Name: Brain Injuries
- Relevance: HIGH
- As Found: Brain Injury
- ID: M5204
- Name: Brain Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5742
- Name: Central Nervous System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M17685
- Name: Wounds and Injuries
- Relevance: LOW
- As Found: Unknown
- ID: M9349
- Name: Craniocerebral Trauma
- Relevance: LOW
- As Found: Unknown
- ID: M22023
- Name: Trauma, Nervous System
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000001930
- Term: Brain Injuries
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT04684979
**Brief Title:** Transplantation of Hematopoietic Stem Cells From HLA-compatible Donors in Patients With B-Cell Lymphoid Malignancies
**Official Title:** A Non-Myeloablative Conditioning Regimen With Peri-Transplant Rituximab and the Transplantation of Hematopoietic Stem Cells From HLA-compatible Related or Unrelated Donors in Patients With B-Cell Lymphoid Malignancies
#### Organization Study ID Info
**ID:** 2019-KOE-002
#### Organization
**Class:** OTHER
**Full Name:** Baptist Health South Florida
### Status Module
#### Completion Date
**Date:** 2027-03
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2023-11-01
**Type:** ACTUAL
**Last Update Submit Date:** 2023-10-31
**Overall Status:** WITHDRAWN
#### Primary Completion Date
**Date:** 2026-03
**Type:** ESTIMATED
#### Start Date
**Date:** 2022-03-28
**Type:** ACTUAL
**Status Verified Date:** 2023-10
#### Study First Post Date
**Date:** 2020-12-28
**Type:** ACTUAL
**Study First Submit Date:** 2020-12-22
**Study First Submit QC Date:** 2020-12-22
**Why Stopped:** The landscape of treatment for patients targeted for enrollment has significantly changed since the protocol was written and approved, and the research treatment is no longer the best option for this population.
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Baptist Health South Florida
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** True
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** This research study is being conducted to treat patients with B-cell lymphoid malignancies. These types of cancers include diffuse large cell (DLBCL) non-Hodgkin's lymphoma (NHL), mantle cell NHL, any indolent B cell NHL (such as follicular, small cell or marginal zone NHL), or chronic lymphocytic leukemia (CLL). Patients with these types of lymphomas have been shown to benefit from peripheral blood stem cell transplantation (PBSCT). PBSCT uses healthy blood stem cells from a donor to replace your diseased or damaged bone marrow. Before undergoing PBSCT, you'll receive chemotherapy and/or radiation to destroy your diseased cells and prepare your body for the donor cells. This is called a "conditioning regimen." Non-myeloablative (NMA) conditioning causes minimal cell death. This research study will look at a course of treatment using NMA conditioning regimen including low dose chemotherapy and low dose radiation as well as rituximab and PBSCT from a compatible donor. The primary aim is to obtain a preliminary estimate of the overall and event-free survival 1 year post-transplant after NMA.
**Detailed Description:** This is a phase 2 study of a treatment regimen consisting of a non-myeloablative (NMA) conditioning regimen incorporating low dose chemotherapy and low dose radiation as well as peri-transplant Rituximab and the transplantation of peripheral blood stem cells (PBSC) from an HLA compatible related or unrelated donor in patients with B cell lymphoid malignancies including diffuse large cell (DLBCLC) and mantle cell non-Hodgkin's lymphoma (MCL), indolent B cell NHL, or chronic lymphocytic leukemia (CLL). The study design will be based on a total of 90 patients, 30 recipients of related matched and 60 recipients of mismatched related or unrelated PBSCT.
It is anticipated that the accrual will last 5-6 years. At the conclusion of the study, the safety and a preliminary assessment of efficacy of NMA PBSCT will be determined. The treatment will be declared efficacious if the disease-free survival at 1 year in this patient population is at least 50%.
### Conditions Module
**Conditions:**
- B-Cell Lymphoid Malignancies
- Hematologic Malignancy
- Non-Hodgkin Lymphoma
**Keywords:**
- Hematologic Malignancies, NHL
### Design Module
#### Design Info
**Allocation:** NON_RANDOMIZED
**Intervention Model:** PARALLEL
**Intervention Model Description:** The study design will be based on a total of 90 patients, 30 recipients of related matched and 60 recipients of mismatched related or unrelated PBSCT.
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Type:** ACTUAL
**Phases:**
- PHASE2
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** This is a phase 2 study to evaluate NMA PBSCT incorporating peri-transplant rituximab and utilizing PBSC to augment graft cell dose in patients with selected B lymphoid malignancies. Salvage chemotherapy will be required as part of transplant eligibility, both to achieve debulking of disease to allow sufficient time for the development of a post-transplant GVL effect, and to contribute to recipient immune suppression and thus facilitate donor engraftment.
**Intervention Names:**
- Biological: Hematopoietic Stem Cells from HLA-compatible Related
**Label:** HLA-compatible Related Donor
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** This is a phase 2 study to evaluate NMA PBSCT incorporating peri-transplant rituximab and utilizing PBSC to augment graft cell dose in patients with selected B lymphoid malignancies. Salvage chemotherapy will be required as part of transplant eligibility, both to achieve debulking of disease to allow sufficient time for the development of a post-transplant GVL effect, and to contribute to recipient immune suppression and thus facilitate donor engraftment.
**Intervention Names:**
- Biological: Hematopoietic Stem Cells from HLA Unrelated
**Label:** Unrelated Donor
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- HLA-compatible Related Donor
**Description:** NMA PBSCT (Non-Myeloablative peripheral blood stem cell transplantation) incorporating rituximab and utilizing PBSC (Peripheral blood stem cells) to increase graft cell dose in patients with selected B lymphoid malignancies.
**Name:** Hematopoietic Stem Cells from HLA-compatible Related
**Type:** BIOLOGICAL
#### Intervention 2
**Arm Group Labels:**
- Unrelated Donor
**Description:** NMA PBSCT (Non-Myeloablative peripheral blood stem cell transplantation) incorporating rituximab and utilizing PBSC (Peripheral blood stem cells) to increase graft cell dose in patients with selected B lymphoid malignancies.
**Name:** Hematopoietic Stem Cells from HLA Unrelated
**Type:** BIOLOGICAL
### Outcomes Module
#### Primary Outcomes
**Description:** The primary aim of this study is to obtain a preliminary estimate of the overall and event-free survival at 1 year after NMA PBSCT with peri-transplant rituximab using an HLA matched or single HLA allele disparate related or unrelated donors
**Measure:** Estimate the overall and event-free survival
**Time Frame:** 1 year
#### Secondary Outcomes
**Description:** the speed of neutrophil and platelet recovery post allograft
**Measure:** Speed of Recovery Post Allograft
**Time Frame:** 100 days
**Description:** the incidence and speed of donor-derived engraftment
**Measure:** Response to Engraftment
**Time Frame:** 100 days
**Description:** The incidence and severity of acute GVHD(Graft Versus Host Disease) at 100 days
**Measure:** Status of Graft Versus Host Disease
**Time Frame:** 100 days
**Description:** The incidence and severity of chronic GVHD (Graft Versus Host Disease) at 1 year
**Measure:** Number of Participants with Graft Versus Host Disease
**Time Frame:** 1 year
**Description:** the incidence of serious infectious complications with their correlation with laboratory measurements of immune recovery
**Measure:** Number of Participants with Complications
**Time Frame:** 100 days
**Description:** the response to vaccination after PBSCT (Peripheral Blood Stem Cells Transplantation)
**Measure:** Response Rate to Vaccination
**Time Frame:** 100 days
**Description:** the incidence of Transplant Related Mortality at 100 and 180 days
**Measure:** Number of Transplant Related Mortality Incidences
**Time Frame:** 100 and 180 days
**Description:** the incidence of malignant relapse or disease progression at 1 and 2 years
**Measure:** Number of Relapse or Disease Progression Instances
**Time Frame:** 1 and 2 years
**Description:** the probabilities of overall and event-free survival at 2 years after Peripheral Blood Stem Cells Transplantation
**Measure:** Number of Overall and Event Free Survival
**Time Frame:** 2 years
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* :
* Patients aged 18-74 years at initial referral with a suitably matched related or unrelated donor who have provided their informed consent to participate in the clinical trial.
* If post-pubertal, females agree to take hormonal therapy to suppress menses unless a specific contra-indication to estrogen exists
Diagnosis:
* Patients with CD20+ aggressive B cell NHL (DLBCL, large cell transformation of indolent NHL/CLL, or mantle cell) OR CD20+ indolent NHL/CLL. Relapsed disease must be biopsy proven and CD20 positivity must be demonstrated within the 12 months prior to protocol enrollment.
Eligible patients with DLBCL NHL will:
* have relapsed disease following initial therapy but failed to mobilize or had bone marrow involvement and therefore are not suitable for an autologous transplant OR
* have high-intermediate or high-risk second-line age-adjusted International Prognostic Index score and be in 2nd CR/PR following an autologous transplant OR
* have failed an autologous transplant and be in PR or better after salvage chemotherapy.
Eligible patients with transformed indolent NHL/CLL will:
• have CR/PR of the large cell component of their disease after either salvage chemotherapy or an autologous transplant.
Eligible patients with mantle cell NHL will:
* be high-risk such as p53 positivity and be in 1st CR/PR after initial therapy OR
* have relapsed disease following initial therapy and be in 2nd or 3rd CR/PR after salvage chemotherapy.
Eligible patients with indolent B cell NHL (such as, but not limited to, follicular, small cell or marginal zone NHL) or CLL will:
• have 1st or subsequent progression or primary refractory disease (pre-allograft cytoreduction necessary but CR/PR not required).
Pre-allograft Salvage Chemotherapy:
* This can include a single autologous transplant using high dose chemotherapy conditioning if appropriate OR ≥ 2 cycles of intensive combination chemotherapy (e.g. RICE) as appropriate according to diagnosis and prior therapy.
* CLL patients who have received CAMPATH do not have to receive pre-allograft salvage chemotherapy.
Timing of PBSCT:
• Admission for PBSCT must be within 120 days of autologous transplantation OR 80 days of the last cycle of chemotherapy.
Organ Function and Performance Status Criteria:
* Karnofsky score ≥ 70 %
* calculated creatinine clearance ≥ 50 mL/min OR if creatinine ≥ 1.2, a history of renal dysfunction, age \> 50 years, prior transplant, and/or a single kidney, the patient must have a measured creatinine clearance (using 24 hour urine collection) ≥ 50 mL/min
* bilirubin \< 2.5, AST/ALT ≤ 3 x upper limit of normal (unless benign congenital hyperbilirubinemia)
* pulmonary function (spirometry and corrected DLCO) ≥ 50% normal
* left ventricular ejection fraction ≥ 40%
* albumin ≥ 2.5. Donor HLA-compatible related donors
* Patients who have an HLA-matched or one allele mismatched related donor are eligible for entry on this protocol. This will include a healthy related donor who is genotypically or phenotypically matched at least 9/10 of the A, B, C, DRB1, and DQB1 loci, as tested by high resolution.
HLA-compatible Unrelated donors • Patients who do not have a related HLA-matched donor but have an unrelated donor who is matched at
≥ 9/10 (allele mismatch only) of the A, B, C, DRB1, and DQB1 loci, as tested by high resolution.
Exclusion Criteria:
* Diagnosis: known negativity for CD20 pre-allograft; mantle cell or DLBCL NHL with progressive disease at allograft work-up
* Prior Therapy: prior allogeneic transplant (prior autologous transplant is acceptable)
* Cytoreduction and timing of NMA PBSCT: patients unable to complete planned cytoreduction due to therapy complications, or who undergo cytoreduction but are unable to proceed to allografting within the defined time period, are ineligible for allograft on protocol
* Active and uncontrolled infection at time of transplantation including active infection with Aspergillus or other mold, or HIV infection
* Patients positive for Hepatitis B or C at risk for viral reactivation.
* Inadequate performance status/organ function
* Pregnant or breast feeding
* Patient or guardian unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, follow-up and research tests.
**Maximum Age:** 74 Years
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Miami
**Country:** United States
**Facility:** Miami Cancer Institute at Baptist Health of South Florida
**State:** Florida
**Zip:** 33176
#### Overall Officials
**Official 1:**
**Affiliation:** Miami Cancer Institute at Baptist Health of South Florida
**Name:** Guenther Koehne, MD, PhD
**Role:** PRINCIPAL_INVESTIGATOR
### IPD Sharing Statement Module
**IPD Sharing:** NO
### References Module
#### See Also Links
**Label:** Miami Cancer Institute Website
**URL:** http://baptisthealth.net/cancer-care/home
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000009371
- Term: Neoplasms by Site
- ID: D000006402
- Term: Hematologic Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: BC15
- Name: Blood and Lymph Conditions
- Abbrev: BC20
- Name: Immune System Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M11220
- Name: Lymphoma
- Relevance: LOW
- As Found: Unknown
- ID: M21314
- Name: Hematologic Neoplasms
- Relevance: HIGH
- As Found: Hematologic Malignancies
- ID: M9490
- Name: Hematologic Diseases
- Relevance: LOW
- As Found: Unknown
- ID: T3543
- Name: Lymphosarcoma
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000009369
- Term: Neoplasms
- ID: D000019337
- Term: Hematologic Neoplasms
### Intervention Browse Module - Browse Branches
- Abbrev: ANeo
- Name: Antineoplastic Agents
- Abbrev: ARhu
- Name: Antirheumatic Agents
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M373
- Name: Rituximab
- Relevance: LOW
- As Found: Unknown
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT02044679
**Brief Title:** Evaluation of Spot Urine as a Biomarker of Fluid Intake in Real Life Conditions
#### Organization Study ID Info
**ID:** NU365
#### Organization
**Class:** INDUSTRY
**Full Name:** Danone Research
### Status Module
#### Completion Date
**Date:** 2014-03
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2016-09-19
**Type:** ESTIMATED
**Last Update Submit Date:** 2016-09-15
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2014-03
**Type:** ACTUAL
#### Start Date
**Date:** 2013-10
**Status Verified Date:** 2016-09
#### Study First Post Date
**Date:** 2014-01-24
**Type:** ESTIMATED
**Study First Submit Date:** 2014-01-22
**Study First Submit QC Date:** 2014-01-22
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Danone Research
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** Evaluate urine osmolality as a marker of fluid intake, in healthy subjects displaying a wide range of fluid intake behaviors
### Conditions Module
**Conditions:**
- Healthy Adults
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** NONE
#### Enrollment Info
**Count:** 82
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** + 1,5 to 2,0 L/day of water
**Intervention Names:**
- Other: Water
**Label:** A = Increase water intake 1
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** + 1,0 to 1,5 L/day of water
**Intervention Names:**
- Other: Water
**Label:** B = Increase water intake 2
**Type:** EXPERIMENTAL
#### Arm Group 3
**Description:** No change
**Intervention Names:**
- Other: No intervention
**Label:** C = no change
**Type:** OTHER
### Interventions
#### Intervention 1
**Arm Group Labels:**
- A = Increase water intake 1
- B = Increase water intake 2
**Name:** Water
**Type:** OTHER
#### Intervention 2
**Arm Group Labels:**
- C = no change
**Name:** No intervention
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Measure:** Osmolality
**Time Frame:** baseline
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Healthy female and male subjects aged 20- 30 years old (both ages included).
* BMI within the range 20-25 kg/m2 (both inclusive).
* Height within the range 1.60-1.75 m (both inclusive) for female subjects and 1.70-1.85 m (both inclusive) for male subjects.
* Monophasic contraceptive method (female subjects only)
* Smoking less than 10 cigarettes/day
* Fluid intake habits falling within one of three designated arms
Exclusion Criteria:
* Pregnant woman (as determined by a pregnancy test) or woman planning to become pregnant during the study; breast-feeding woman.
* Any clinically relevant acute or chronic diseases which could interfere with the subjects' safety during the trial, or expose them to undue risk, or which could interfere with the study objectives.
* Having participated in a clinical study for the renal diseases or having received any treatment related to the kidneys, cardiovascular disease or to hypertension in the last 12 months.
**Healthy Volunteers:** True
**Maximum Age:** 30 Years
**Minimum Age:** 20 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Rennes
**Country:** France
**Facility:** BIOTRIAL
**Zip:** 35042
### References Module
#### References
**Citation:** Lemetais G, Melander O, Vecchio M, Bottin JH, Enhorning S, Perrier ET. Effect of increased water intake on plasma copeptin in healthy adults. Eur J Nutr. 2018 Aug;57(5):1883-1890. doi: 10.1007/s00394-017-1471-6. Epub 2017 Jun 3.
**PMID:** 28578535
## Derived Section
### Condition Browse Module - Browse Branches
- Abbrev: BC17
- Name: Skin and Connective Tissue Diseases
- Abbrev: All
- Name: All Conditions
### Condition Browse Module - Browse Leaves
- ID: M8219
- Name: Exanthema
- Relevance: LOW
- As Found: Unknown
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT02118779
**Acronym:** OPTIMISTIC
**Brief Title:** Observational Prolonged Trial in Myotonic Dystrophy Type 1
**Official Title:** Observational Prolonged Trial in Myotonic Dystrophy Type 1 to Improve Quality of Life Standards, a Target Identification Collaboration
#### Organization Study ID Info
**ID:** 13/NE/0342
#### Organization
**Class:** OTHER
**Full Name:** Radboud University Medical Center
#### Secondary ID Infos
**Domain:** EU Seventh Framework Programme
**ID:** 305697
**Type:** OTHER_GRANT
**Domain:** Sponsor ref
**ID:** 6836
**Type:** OTHER
### Status Module
#### Completion Date
**Date:** 2016-10-17
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2017-07-18
**Type:** ACTUAL
**Last Update Submit Date:** 2017-07-13
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2016-03-29
**Type:** ACTUAL
#### Start Date
**Date:** 2014-04-02
**Type:** ACTUAL
**Status Verified Date:** 2017-07
#### Study First Post Date
**Date:** 2014-04-21
**Type:** ESTIMATED
**Study First Submit Date:** 2014-04-11
**Study First Submit QC Date:** 2014-04-18
### Sponsor Collaborators Module
#### Collaborators
**Class:** OTHER
**Name:** University of Newcastle Upon-Tyne
**Class:** OTHER
**Name:** Ludwig-Maximilians - University of Munich
**Class:** OTHER
**Name:** Assistance Publique - Hôpitaux de Paris
#### Lead Sponsor
**Class:** OTHER
**Name:** Radboud University Medical Center
#### Responsible Party
**Investigator Affiliation:** Radboud University Medical Center
**Investigator Full Name:** Annet Geerlings
**Investigator Title:** Coordinator Trialoffice Neurology
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** Myotonic dystrophy type1 (DM1) is a rare, inherited, chronic progressive disease as well as an autosomal dominant multisystemic disorder. It is the most common adult form of muscular dystrophy, with a prevalence of approximately 10 per 100,000 people affected. With 733 million people in Europe, we estimate that 75,000 people are DM1 patients in Europe.
The aim of OPTIMISTIC is to improve clinical practice in the management of patients with this rare disease for which no dedicated treatment is currently available. OPTIMISTIC is a multi-centre, randomised controlled trial designed to compare a two component tailored behavioural change intervention to increase physical activity against standard patient management regimes, with particular attention given to the definition of appropriate outcome measures and new clinical guidelines for DM1 management. The two components of the intervention are 1) cognitive behavioural therapy (CBT) and 2) graded physical activity and we will evaluate the intervention's effectiveness and safety against standard patient management.
Participants will be recruited from myotonic dystrophy clinics and neuromuscular centres in France, Germany, the Netherlands and the UK. A total of 286 male and female patients aged 18 years and older with genetically proven classical or adult DM1 suffering from severe fatigue (only DM1 patients with a CIS subscale fatigue score \> 35 are likely to benefit from the intervention), able to walk independently and able to complete the trial interventions will be included.
A key objective of OPTIMISTIC is to provide outcome measures that are relevant for the patients and have a rate of change that is appropriate for a clinical trial timeframe. In addition, OPTIMISTIC will identify genetic factors that predict outcome and potential biomarkers as surrogate outcome measures that best explain the observed clinical variation.
**Detailed Description:** Background DM1 is a rare, inherited, progressive disease as well as an autosomal dominant multisystemic disorder. It is the most common adult form of muscular dystrophy, with a prevalence of approximately 10 per 100,000 people affected. With 733 million people in Europe, we estimate that 75,000 people are DM1 patients in Europe. Typical symptoms of the disease include progressive muscle weakness and wasting from distal to proximal, ptosis, weakness of facial, jaw and anterior neck muscles, myotonia, daytime sleepiness, fatigue and cataract. Other symptoms of adult DM1 include cardiac conduction defects, as well as endocrine, gastrointestinal and cognitive dysfunction. DM1 is one of the most variable human diseases, has complex, multisystemic and progressively worsening clinical manifestations and leads to severe physical impairment, restricted social participation and premature death.
There is no pharmaceutical treatment for causal or symptomatic relief of DM1 core symptoms (with the exception of Modafinil for excessive daytime sleepiness). Thus the aim of treatment is to relieve impairments, reduce limitations and optimise participation. Physical activity has been acknowledged as an important factor for health in general. For patients with a slowly progressive neuromuscular disease, such as DM1, there is accumulating evidence for prescribing low-to-moderate-intensity strength and aerobic exercise training, and an active lifestyle. Nevertheless, recent reviews conclude that existing studies are limited in number and quality, and that there is a need for disease-specific, randomised, controlled trials investigating the effect on quality of life.
RATIONALE FOR THE STUDY It was demonstrated recently by an OPTIMISTIC research partner that severe fatigue, defined as a score equal to or higher than 35 on the subscale fatigue of the Checklist Individual Strength (CIS-fatigue), was reported by around 70% of patients with DM1. These severely fatigued patients had more problems with physical and social functioning as well as with their mental and general health than similar patients without severe fatigue. They also had more problems with concentration and planning. As such, experienced fatigue should be clearly distinguished from muscle weakness, which is probably the most common and characteristic symptom of DM1 and also of a lack of initiative (apathy) that is known to occur often in DM1.
In a longitudinal study, we built a model of perpetuating factors for fatigue in patients with DM1. It appeared that lack of physical activity, sleep disturbances and pain all contributed to experienced fatigue. In addition, loss of muscle strength and pain contributed to fatigue through a lower level of physical activity. Ultimately, experienced fatigue and physical activity both contributed to the level of societal participation. A lack of initiative further increased fatigue but also had a direct negative effect on societal participation. Thus, theoretically, in order to improve societal participation one should compensate for a reduced initiative, optimise physical activity and alleviate experienced fatigue. To alleviate fatigue one should address the fatigue maintaining factors identified by the model, e.g. experience of pain or sleep disorders.
The main rationale for the combination of CBT and physical activity is based on our DM1-specific model. The DM1-specific model shows that physical activity, experienced fatigue and lack of initiative are the main determinants of DM1 health status. OPTIMISTIC is the first model-based clinical trial in DM1. It evaluates the effect, and the maintenance of effects, of CBT combined with exercise training on the reduction of chronic fatigue in patients with DM1.
Importantly, the intervention will also involve caregivers where they are willing to take part. The disabilities associated with DM1 put considerable strain on caregivers and can also lead to a negative interaction with the patient. The intervention will aim to support caregivers by installing realistic expectations about what can be expected from the patient, teach caregivers how to help patients to stay as self-reliant as possible and also reduce caregivers strain by taking time for themselves. If a DM1 patient has no caregiver or significant other, or no caregiver or significant other willing to take part in the study, the patient will not be excluded from the study. All patients will be asked if the study team could approach them to inform them for further research. This contact does not constitute consent.
\*\* CMRI-substudy in People with DM1 People with DM1 are at high risk of developing a cardiac complication. However, it is not known whether the high prevalence of cardiac complications can be affected by a sedentary lifestyle. Given the high risk of cardiac complications and the possibility that becoming more physically active may help these complications, the University of Newcastle, will conduct a sub-study to perform Cardiac Magnetic Resonance Imaging (CMRI) in 40 eligible participants at baseline and at end of the intervention period to acquire the evidence upon which clinical judgement about the use of exercise as a clinical therapy that is safe, can be based.
Cardiac Magnetic resonance imaging (CMRI) uses a combination of harmless radiofrequency (RF) waves and powerful magnets to cause hydrogen nuclei within the cardiac cell molecules to vibrate and emit RF energy. The MRI scanner detects the energy emissions and converts them to viewable images. When diseases begin, there are changes in the heart's tissue. Because even minor changes in tissue affect the rates at which energy is emitted, many medical conditions can be detected at their very early stages. MRI is done to evaluate the structure and function of the heart and blood vessels. MRI may provide information that cannot be obtained by other tests such as chest X-ray, ECG, echocardiogram, or nuclear tests.
The CMRI examinations will be performed with the contract enhancement gadolinium, in 40 eligible participants, 20 in each group. Participants will undergo:
1. cardiac cine imaging, to evaluate cardiac morphology
2. systolic and diastolic function and
3. cardiac tagging to evaluate wall motion and torsion.
MRI safety will be established prior to baseline and end of intervention scan. This includes; assessing for in vivo ferrous material, claustrophobia, abnormal renal function and pregnancy. The PI will assess renal function from the participant's medical notes at screening, if required a blood tests for U\&Es will be requested. In addition, child-bearing potential female participants will consent to have urine pregnancy test performed.
For participants that have had a baseline CMRI and withdraw from the study prior to the end of the intervention period, will be invited to have an end of study CMRI if the period from their initial CMRI is greater than 3 months.
No further statistical review is required for the CMRI sub-study as only frequencies and associations will be assessed.
### Conditions Module
**Conditions:**
- Myotonic Dystrophy Type 1
**Keywords:**
- Myotonic Dystrophy Type 1
- DM1
- Physical Exercise
- Cognitive Behavioural Therapy
- Biomarkers
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** SINGLE
**Who Masked:**
- OUTCOMES_ASSESSOR
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 255
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Cognitive behavioural therapy (CBT) combined with exercise
**Intervention Names:**
- Behavioral: Behavioural change intervention
**Label:** Behavioural change intervention
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Standard care like usual (i.e. annual checks with neurologist, checks with cardiologist, if needed physical therapy)
**Label:** Standard Patient Management
**Type:** NO_INTERVENTION
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Behavioural change intervention
**Description:** The intervention is cognitive behaviour therapy (CBT). The CBT consists of six different modules. All patients will start with individual goal setting and psycho-education about the role of cognitive-behavioural variables in the disabilities patients' experience. The patient formulates his or her treatment goals in concrete terms and later on in the therapy the goals are realised step by step by the patient. The treatment is tailored to the patient's problems: which of the six modules a patient will receive is dependent on the scores on measures that have been collected at baseline assessment. Based on our previous experience with modular interventions we expect that most patients will receive less than four modules.
**Name:** Behavioural change intervention
**Type:** BEHAVIORAL
### Outcomes Module
#### Other Outcomes
**Description:** Biomarkers (urine and blood)
**Measure:** Explanatory and/or Predictive outcomes
**Time Frame:** Baseline, 10 and 16 months
#### Primary Outcomes
**Description:** The primary outcome measure will be the change in DM1-Activ score. DM1-Activ is a specific outcome measure of activity and participation for patients with DM1.
**Measure:** DM1-Activ
**Time Frame:** Baseline and 10 months
#### Secondary Outcomes
**Description:** Six minute walk test with BORG scale assessment
**Measure:** Six Minute Walk Test
**Time Frame:** Baseline and 10 months
**Measure:** Myotonic Dystrophy Health Index (MDHI)
**Time Frame:** Baseline and 10 months
**Measure:** Physical activity measured with actometer
**Time Frame:** Baseline and 10 months
**Measure:** Fatigue and Daytime Sleepiness Scale (FDSS)
**Time Frame:** Baseline and 10 months
**Measure:** Checklist Individual Strength (CIS)
**Time Frame:** Baseline and 10 months
**Measure:** Individualised Neuromuscular Quality of Life Questionnaire (InQoL)
**Time Frame:** Baseline and 10 months
**Measure:** Beck depression Inventory for Primary Care
**Time Frame:** Baseline and 10 months
**Measure:** Apathy Evaluation Scale (AES)
**Time Frame:** Baseline and 10 months
**Measure:** Stroop Test
**Time Frame:** Baseline and 10 months
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Able to provide informed consent
* Genetically proven DM1
* Suffering from severe fatigue (CIS fatigue \>35
* Able to walk independently
Exclusion Criteria:
* Neurological or orthopaedic co-morbidity interfering with the interventions or possibly influencing outcomes.
* Use of psychotropic drugs (except Modafinil, Ritalin and antidepressants where the dosing regimen has been stable for at least 12 months prior to screening). If the doses of Modafinil or Ritalin increase during the 10 months of the intervention then the participant will be excluded.
* Severe depression as screening (judged as meeting DSM-IV criteria for a depressive episode).
* Participation in another clinical trial of an investigational medicinal product (CTIMP) or other interventional study considered to influence outcomes being evaluated in OPTIMISTIC.
* Unable to complete study questionnaires.
* Subject participating in another clinical trial (other than observational trials and registries) concurrently or within 30 days prior to screening for entry into this study.
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Paris
**Country:** France
**Facility:** Assistance Publique-Hospitaux de Paris
**Location 2:**
**City:** Munich
**Country:** Germany
**Facility:** Friedrich Naur Institute
**Location 3:**
**City:** Nijmegen
**Country:** Netherlands
**Facility:** Radboud University Nijmegen Medical Centre
**Location 4:**
**City:** Newcastle
**Country:** United Kingdom
**Facility:** Newcastle University
**Zip:** NE1 3BZ
#### Overall Officials
**Official 1:**
**Affiliation:** Newcastle University
**Name:** Grainne Gorman, Dr
**Role:** PRINCIPAL_INVESTIGATOR
**Official 2:**
**Affiliation:** Radboud University Nijmegen Medical Centre, The Netherlands
**Name:** Baziel van Engelen, Prof
**Role:** PRINCIPAL_INVESTIGATOR
**Official 3:**
**Affiliation:** Munich University, Germany
**Name:** Benedikt Schoser, Prof
**Role:** PRINCIPAL_INVESTIGATOR
**Official 4:**
**Affiliation:** Assistance Publique-Hospitaux de Paris, France
**Name:** Guillaume Bassez, Prof
**Role:** PRINCIPAL_INVESTIGATOR
### References Module
#### References
**Citation:** van Engelen B; OPTIMISTIC Consortium. Cognitive behaviour therapy plus aerobic exercise training to increase activity in patients with myotonic dystrophy type 1 (DM1) compared to usual care (OPTIMISTIC): study protocol for randomised controlled trial. Trials. 2015 May 23;16:224. doi: 10.1186/s13063-015-0737-7.
**PMID:** 26002596
**Citation:** Wenninger S, Cumming SA, Gutschmidt K, Okkersen K, Jimenez-Moreno AC, Daidj F, Lochmuller H, Hogarth F, Knoop H, Bassez G, Monckton DG, van Engelen BGM, Schoser B. Associations Between Variant Repeat Interruptions and Clinical Outcomes in Myotonic Dystrophy Type 1. Neurol Genet. 2021 Mar 9;7(2):e572. doi: 10.1212/NXG.0000000000000572. eCollection 2021 Apr.
**PMID:** 33884298
**Citation:** Heskamp L, van Nimwegen M, Ploegmakers MJ, Bassez G, Deux JF, Cumming SA, Monckton DG, van Engelen BGM, Heerschap A. Lower extremity muscle pathology in myotonic dystrophy type 1 assessed by quantitative MRI. Neurology. 2019 Jun 11;92(24):e2803-e2814. doi: 10.1212/WNL.0000000000007648. Epub 2019 May 22.
**PMID:** 31118244
**Citation:** Okkersen K, Jimenez-Moreno C, Wenninger S, Daidj F, Glennon J, Cumming S, Littleford R, Monckton DG, Lochmuller H, Catt M, Faber CG, Hapca A, Donnan PT, Gorman G, Bassez G, Schoser B, Knoop H, Treweek S, van Engelen BGM; OPTIMISTIC consortium. Cognitive behavioural therapy with optional graded exercise therapy in patients with severe fatigue with myotonic dystrophy type 1: a multicentre, single-blind, randomised trial. Lancet Neurol. 2018 Aug;17(8):671-680. doi: 10.1016/S1474-4422(18)30203-5. Epub 2018 Jun 19.
**PMID:** 29934199
#### See Also Links
**Label:** Website providing Trial Information for patients and professionals
**URL:** http://optimistic-dm.eu/
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000009136
- Term: Muscular Dystrophies
- ID: D000020966
- Term: Muscular Disorders, Atrophic
- ID: D000009135
- Term: Muscular Diseases
- ID: D000009140
- Term: Musculoskeletal Diseases
- ID: D000020967
- Term: Myotonic Disorders
- ID: D000020271
- Term: Heredodegenerative Disorders, Nervous System
- ID: D000019636
- Term: Neurodegenerative Diseases
- ID: D000009422
- Term: Nervous System Diseases
- ID: D000009468
- Term: Neuromuscular Diseases
- ID: D000030342
- Term: Genetic Diseases, Inborn
### Condition Browse Module - Browse Branches
- Abbrev: BC05
- Name: Musculoskeletal Diseases
- Abbrev: BC10
- Name: Nervous System Diseases
- Abbrev: BC16
- Name: Diseases and Abnormalities at or Before Birth
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: BXM
- Name: Behaviors and Mental Disorders
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M12175
- Name: Myotonic Dystrophy
- Relevance: HIGH
- As Found: Myotonic Dystrophy
- ID: M12093
- Name: Muscular Dystrophies
- Relevance: LOW
- As Found: Unknown
- ID: M4589
- Name: Atrophy
- Relevance: LOW
- As Found: Unknown
- ID: M12092
- Name: Muscular Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M22697
- Name: Muscular Disorders, Atrophic
- Relevance: LOW
- As Found: Unknown
- ID: M12097
- Name: Musculoskeletal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M22698
- Name: Myotonic Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M22092
- Name: Heredodegenerative Disorders, Nervous System
- Relevance: LOW
- As Found: Unknown
- ID: M21558
- Name: Neurodegenerative Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M12411
- Name: Neuromuscular Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M23686
- Name: Genetic Diseases, Inborn
- Relevance: LOW
- As Found: Unknown
- ID: T6034
- Name: Quality of Life
- Relevance: LOW
- As Found: Unknown
- ID: T4024
- Name: Myotonic Dystrophy
- Relevance: HIGH
- As Found: Myotonic Dystrophy
- ID: T4025
- Name: Myotonic Dystrophy Type 1
- Relevance: HIGH
- As Found: Myotonic Dystrophy Type 1
- ID: T3963
- Name: Muscular Dystrophy
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000009223
- Term: Myotonic Dystrophy
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT04292379
**Brief Title:** An Innovative Model of Pediatric Acute Mental Health and Addictions Care
**Official Title:** An Innovative Model of Pediatric Acute Mental Health and Addictions Care to Increase Value to Children,Youth, and the Healthcare System
#### Organization Study ID Info
**ID:** Pro00092862
#### Organization
**Class:** OTHER
**Full Name:** University of Alberta
#### Secondary ID Infos
**Domain:** University of Calgary
**ID:** REB19-0357
**Type:** OTHER
### Status Module
#### Completion Date
**Date:** 2023-01-24
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2023-01-31
**Type:** ACTUAL
**Last Update Submit Date:** 2023-01-30
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2022-06-20
**Type:** ACTUAL
#### Start Date
**Date:** 2020-01-29
**Type:** ACTUAL
**Status Verified Date:** 2022-09
#### Study First Post Date
**Date:** 2020-03-03
**Type:** ACTUAL
**Study First Submit Date:** 2020-02-27
**Study First Submit QC Date:** 2020-02-27
### Sponsor Collaborators Module
#### Collaborators
**Class:** OTHER
**Name:** University of Calgary
#### Lead Sponsor
**Class:** OTHER
**Name:** University of Alberta
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** The investigators will implement and evaluate an integrated, evidence-based bundle of family-centred, pediatric emergency mental health and addictions care.
**Detailed Description:** The investigators will implement and evaluate an integrated, evidence-based bundle of family-centred, emergency mental health and addictions care. This care bundle will provide high value to families, matching resources and services to need while eliminating healthcare inefficiencies and closing care gaps. The bundle will introduce two well-established tools for healthcare providers to use in EDs, walk-in clinics, and urgent mental health care appointments. These tools are the Ask Suicide-Screening Questions (ASQ) and HEADS-ED, an assessment mnemonic (Home; Education \& Employment; Activities \& Peers; Drugs \& Alcohol; Suicidality; Emotions \& Behaviours; Discharge or Current Resources).
The bundle will also introduce new processes to improve healthcare system efficiencies, navigation and transitions between healthcare sectors. For families who receive care in the ED, the investigators will remove the physician gatekeeper role so that children/youth who are screened as low-risk will be offered a follow-up appointment in an urgent mental health care clinic within 24-48 hours. Those who screen as high-risk will see a mental health specialist and undergo a HEADS-ED assessment. Families who follow up in the clinic will receive care that applies a Choice And Partnership Approach (CAPA). CAPA is a collaborative approach to healthcare, where healthcare providers work in partnership with children/youth and their parents to identify choices for care that best match individual needs and preferences.
Study Goal: The investigators' goal is the right care, with the right people, at the right place and time.
Study Design: To measure the bundle's impact, the investigators will use an interrupted time series (ITS) design.
### Conditions Module
**Conditions:**
- Mental Health
- Mental Disorders
- Pediatrics
- Emergency Psychiatric
**Keywords:**
- Emergency Psychiatric Services
- Healthcare Delivery
### Design Module
#### Design Info
**Observational Model:** COHORT
**Time Perspective:** PROSPECTIVE
#### Enrollment Info
**Count:** 1992
**Type:** ACTUAL
**Study Type:** OBSERVATIONAL
### Outcomes Module
#### Other Outcomes
**Description:** Measured using coroner's data
**Measure:** Death by suicide within 30 days of the index ED visit
**Time Frame:** 30 days after the index ED visit
#### Primary Outcomes
**Description:** Measured in survey completed by the participant using the Warwick-Edinburgh Mental Wellbeing Scale (WEMWBS) for patients ≥14 years of age, and the Stirling Children's Wellbeing Scale (SCWBS) for patients \<14 years of age.
For the WEMWBS, each item is scored on a range from 1 to 5. The resulting total score will be between 14 and 70, with lower scores indicating poor wellbeing, and higher scores indicating greater wellbeing. Very low wellbeing is defined as a score of ≤ 45. A substantial decrease in wellbeing is defined as a decrease by 5 or more points.
For the SCWBS, each item is scored on a range from 1 to 5. The resulting total score will be between 12 and 60, with lower scores indicating poor wellbeing, and higher scores indicating greater wellbeing. Very low wellbeing is defined as a score of ≤ 30. A substantial decrease in wellbeing is defined as a decrease by 5 or more points.
**Measure:** Patient wellbeing at 30 days
**Time Frame:** 30 days after the index emergency department (ED) visit
#### Secondary Outcomes
**Description:** Measured in survey completed by the participant using the Warwick-Edinburgh Mental Wellbeing Scale (WEMWBS) for patients ≥14 years of age, and the Stirling Children's Wellbeing Scale (SCWBS) for patients \<14 years of age.
For the WEMWBS, each item is scored on a range from 1 to 5. The resulting total score will be between 14 and 70, with lower scores indicating poor wellbeing, and higher scores indicating greater wellbeing. Very low wellbeing is defined as a score of ≤ 45. A substantial decrease in wellbeing is defined as a decrease by 5 or more points.
For the SCWBS, each item is scored on a range from 1 to 5. The resulting total score will be between 12 and 60, with lower scores indicating poor wellbeing, and higher scores indicating greater wellbeing. Very low wellbeing is defined as a score of ≤ 30. A substantial decrease in wellbeing is defined as a decrease by 5 or more points.
**Measure:** Patient wellbeing at 90 and 180 days
**Time Frame:** 90 days, and 180 days after the index ED visit
**Description:** Measured in survey completed by the participant using the Family Quality of Life Scale (FQOL). The 25-item scale uses a 5-point rating with a maximum score of 125 indicating highest quality of life.
**Measure:** Family functioning
**Time Frame:** 30 days after the index ED visit
**Description:** Measured in survey completed by the participant using the Service Satisfaction Scale 10 (SSS-10). The scale consists of 12 items (parent version) or 10 items (youth version). Items are scored on a 5-point response scale with a total possible score of 60 (parent) or 50 (youth). Higher scores indicate higher satisfaction.
**Measure:** Satisfaction with acute mental health and addictions ED care
**Time Frame:** 72 hours after the index ED visit
**Description:** Measured using data collected in the patient electronic medical record
**Measure:** Proportion of children/youth admitted to child mental health service (child and adolescent psychiatry, mental health team, etc.)
**Time Frame:** Index ED visit (Day 0)
**Description:** Measured using data collected in the patient electronic medical record. ED Length of stay is defined as the time between patient triage and discharge from the emergency department
**Measure:** Length of ED stay for discharged patients with any mental health presenting complaint
**Time Frame:** Hours spent in the ED, measured at the index ED visit (Day 0)
**Description:** Measured using data collected in the patient electronic medical record
**Measure:** Proportion of ED revisits within 72 hours and 30 days for mental health and substance use disorders
**Time Frame:** 72 hours and 30 days after the index ED visit (Day 0)
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Patient is under 18 years of age
* Patient came to the emergency department with a mental health and/or addiction concern
* Patient presented with one of the following CEDIS complaints:
Anxiety, bizarre behaviour, concern for patient's welfare, deliberate self-harm, depression/suicidal, homicidal behaviour, insomnia, pediatric disruptive behaviour, situational crisis, violent behaviour
Exclusion Criteria:
* Brought to the ED by police, peace officer or EMS?
* Held under Form 10
* Features of schizophrenia, schizotypal and delusional disorders (e.g., hallucinations, delusions, active psychosis)
* Behavioural syndromes or other medical concerns requiring medical clearance (e.g., eating disorders)
* Significant self-harm requiring medical clearance (e.g., deep laceration, ingestion, hanging)
* Barriers to communication at triage (e.g., language)
* Previous participation in the study
**Maximum Age:** 17 Years
**Minimum Age:** 0 Years
**Sampling Method:** NON_PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- CHILD
**Study Population:** Patients less than 18 years of age that present to the emergency department with a mental health complaint, and do not require medical clearance by an emergency department physician.
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Calgary
**Country:** Canada
**Facility:** Alberta Children's Hospital
**State:** Alberta
**Zip:** T3B 6A8
**Location 2:**
**City:** Edmonton
**Country:** Canada
**Facility:** Stollery Children's Hospital
**State:** Alberta
**Zip:** T6G 2B7
#### Overall Officials
**Official 1:**
**Affiliation:** University of Alberta
**Name:** Amanda Newton, PhD
**Role:** PRINCIPAL_INVESTIGATOR
### IPD Sharing Statement Module
**Description:** IPD will not be shared with other researchers.
**IPD Sharing:** NO
### References Module
#### References
**Citation:** Freedman S, Thull-Freedman J, Lightbody T, Prisnie K, Wright B, Coulombe A, Anderson LM, Stang AS, Mikrogianakis A, VanRiper L, Stubbs M, Newton A; Pediatric Emergency Research Canada (PERC). Introducing an innovative model of acute paediatric mental health and addictions care to paediatric emergency departments: a protocol for a multicentre prospective cohort study. BMJ Open Qual. 2020 Dec;9(4):e001106. doi: 10.1136/bmjoq-2020-001106.
**PMID:** 33318032
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000020969
- Term: Disease Attributes
- ID: D000010335
- Term: Pathologic Processes
### Condition Browse Module - Browse Branches
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: All
- Name: All Conditions
- Abbrev: BXM
- Name: Behaviors and Mental Disorders
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M7796
- Name: Emergencies
- Relevance: HIGH
- As Found: Emergency
- ID: M4815
- Name: Mental Disorders
- Relevance: HIGH
- As Found: Mental Disorders
- ID: M14473
- Name: Psychotic Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M19100
- Name: Behavior, Addictive
- Relevance: LOW
- As Found: Unknown
- ID: M22700
- Name: Disease Attributes
- Relevance: LOW
- As Found: Unknown
- ID: T170
- Name: Acute Graft Versus Host Disease
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000004630
- Term: Emergencies
- ID: D000001523
- Term: Mental Disorders
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT04824079
**Brief Title:** Keynatinib in Treated Patients With NSCLC and Brain Metastases
**Official Title:** Study of Keynatinib in Patients With Advanced Non-small Cell Lung Cancer With Brain Metastasis or Progression of Brain Metastasis After Treatment With EGFR Inhibitor(s)
#### Organization Study ID Info
**ID:** MEDO-007-20002
#### Organization
**Class:** INDUSTRY
**Full Name:** Medolution Ltd.
### Status Module
#### Completion Date
**Date:** 2025-07-22
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2023-11-07
**Type:** ACTUAL
**Last Update Submit Date:** 2023-11-04
**Overall Status:** RECRUITING
#### Primary Completion Date
**Date:** 2025-04-22
**Type:** ESTIMATED
#### Start Date
**Date:** 2020-10-21
**Type:** ACTUAL
**Status Verified Date:** 2023-11
#### Study First Post Date
**Date:** 2021-04-01
**Type:** ACTUAL
**Study First Submit Date:** 2021-03-23
**Study First Submit QC Date:** 2021-03-26
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Medolution Ltd.
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** The purpose of this study is to evaluate the safety and efficacy of Keynatinib capsules in patients with advanced non-small cell lung cancer (NSCLC) with brain metastasis or progression of brain metastasis after treatment with EGFR inhibitors. As well as, to evaluate the penetration rate of Keynatinib in the Blood-Brain Barrier (BBB) and its PK characteristics, and the relationship between exposure levels with efficacy and safety.
**Detailed Description:** This is a multicenter, non-randomized, open, single-arm, phase IIa trial investigating the security and effectiveness of Keynatinib in patients with advanced non-small cell lung cancer (NSCLC) with brain metastasis or progression of brain metastasis after treatment with EGFR inhibitors.
### Conditions Module
**Conditions:**
- Non-Small Cell Lung Cancer With EGFR Mutation
- Brain Metastases
### Design Module
#### Design Info
**Allocation:** NA
**Intervention Model:** SINGLE_GROUP
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 30
**Type:** ESTIMATED
**Phases:**
- PHASE2
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** All subjects shall be treated with Keynatinib twice a day (once every 12±3 hours), 20 mg each time, fasting within 2 hours before and 1 hour after taking the drug, and taking warm water when taking the drug. Every 21 days is a treatment cycle .
**Intervention Names:**
- Drug: Keynatinib
**Label:** Keynatinib treatment group
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Keynatinib treatment group
**Description:** The recommended dose is 20 mg Keynatinib twice a day until disease progression， unacceptable toxicity death occurs or withdraw from the study, whichever occurs first.
**Name:** Keynatinib
**Other Names:**
- TL007
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Description:** The proportion of subjects whose optimal efficacy observed from the beginning of administration is CR or PR.
**Measure:** Objective Response Rate
**Time Frame:** From start of treatment to time of progression (in CNS or non-CNS disease) , intolerable toxicity, withdrawal of informed consent or death, whichever occurs first，assessed up to 2 years.
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
1. Unlimited gender, age ≥ 18 years；
2. Histologically or cytologically documented locally advanced, metastatic NSCLC (including L858R and/or Exon19 del mutation positive). The EGFR mutation assessed by local laboratory/or central laboratory via tissue/cytology or in plasma;
3. Subjects should have the following conditions: a. (Cohort 1) Progression on any of the first or second generation EGFR inhibitors (gefitinib, erlotinib, afatinib, etc.).B. (Cohort 2) Progression on any third generation EGFR inhibitor (oxitinib, etc.), prior or non-prior treatment with any first or second generation EGFR inhibitor;
4. The peripheral lesions did not progress after EGFR inhibitor treatment, but the occurrence of brain metastasis or progression of brain metastasis was confirmed by magnetic resonance imaging (MRI);
5. As determined by the investigator, no final surgical resection or radiotherapy is expected for all lesions;
6. Stable condition for at least 2 weeks prior to study medication without any corticosteroid or anticonvulsant therapy;
7. The status score of Eastern Cooperative Oncology Group (ECOG) is 0 to 1 points, and there was no deterioration during the first 2 weeks of enrollment;
8. Estimated survival time \> 3 months；
9. Sufficient bone marrow, liver, kidney and blood coagulation function；
10. Subjects must be willing to use barrier contraception；
11. Ability to provide informed consent, complete all study assessments and have complete medical record.
Exclusion Criteria:
1. Have previously received chemotherapy, immunotherapy or any other systemic antitumor therapies within 4 weeks before the first administration; Have previously received EGFR-TKI within 5× half-life before the first administration; Have received oral fluorouracil and other small-molecule targeted drugs (whichever is longer) within 2 weeks or 5× half-life before enrollment; Within 2 weeks before enrollment, subjects had received palliative radiotherapy for non-target lesions for symptom relief, and traditional Chinese medicine (including Chinese patent medicine) for tumor indication;
2. Have received whole brain radiation therapy;
3. Only leptomeningeal metastasis are present;
4. Historical intracranial hemorrhage not related to the tumor;
5. Major organ surgery (excluding needle biopsy) or significant trauma have been performed within 4 weeks prior to enrollment;
6. The drug or food with strong inhibition or induction of cytochrome P450 (CYP) isoenzyme CYP3A4 could not be stopped. The drug or food with strong inhibition or induction of cytochrome P450 (CYP) isoenzyme CYP3A4 had been used within 7 days before enrollment;
7. Any unresolved toxicities from prior therapy greater than CTCAE 5.0 grade 1 at the time of enrollment with the exception of alopecia;
8. Refractory nausea and vomiting, chronic gastrointestinal diseases, inability to swallow the tablet or previous significant bowel resection that would preclude adequate absorption of Keynatinib;
9. Past medical history of ILD, drug-induced ILD, radiation pneumonitis which required steroid treatment, or CT scan at baseline revealed the presence of idiopathic pulmonary fibrosis; Uncontrolled large pleural effusion or pericardial effusion; Active tuberculosis;
10. Any evidence of severe or uncontrolled systemic diseases, including uncontrolled hypertension and active bleeding diatheses or active infection including hepatitis B, hepatitis C and HIV;
11. Have a history of severe cardiovascular disease (NYHA cardiac function grade III or IV);
12. Other primary malignancies (except cured basal cell carcinoma of the skin and carcinoma in situ of the cervix) within 5 years;
13. Allergic to the active ingredient or excipient of Keynatinib;
14. Pregnant or lactating women.
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Beijing
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Yuankai Shi, PhD
- **Phone:** +86(010)-87788293
- **Role:** CONTACT
***Contact 2:***
- **Name:** Yuankai Shi, phD
- **Role:** PRINCIPAL_INVESTIGATOR
***Contact 3:***
- **Name:** Shengyu Zhou, phD
- **Role:** SUB_INVESTIGATOR
**Country:** China
**Facility:** Cancer Hospital, Chinese Academy of Medical Sciences
**State:** Beijing
**Status:** NOT_YET_RECRUITING
**Zip:** 100021
**Location 2:**
**City:** Fuzhou
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Wu Zhuang, phD
- **Phone:** +8613809500871
- **Role:** CONTACT
***Contact 2:***
- **Name:** Wu Zhuang, phD
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** China
**Facility:** Fujian Cancer Hospital
**State:** Fujian
**Status:** NOT_YET_RECRUITING
**Zip:** 350014
**Location 3:**
**City:** Zhengzhou
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Huijuan Wu, MD
- **Phone:** +8613838528387
- **Role:** CONTACT
***Contact 2:***
- **Name:** Huijuan Wu, MD
- **Role:** PRINCIPAL_INVESTIGATOR
***Contact 3:***
- **Name:** Yanqiu Zhao, MD
- **Role:** SUB_INVESTIGATOR
**Country:** China
**Facility:** Henan Cancer Hospital
**State:** Henan
**Status:** RECRUITING
**Zip:** 450003
**Location 4:**
**City:** Changsha
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Jianhua Chen, MD
- **Phone:** +8613807488036
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jianhua Chen, MD
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** China
**Facility:** Hunan Cancer Hospital
**State:** Hunan
**Status:** RECRUITING
**Zip:** 410013
**Location 5:**
**City:** Jinan
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Qisen Guo, phD
- **Phone:** +8613869199681
- **Role:** CONTACT
***Contact 2:***
- **Name:** Qisen Guo, phD
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** China
**Facility:** Shandong Cancer Hospital
**State:** Shandong
**Status:** RECRUITING
**Zip:** 250000
### IPD Sharing Statement Module
**IPD Sharing:** NO
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000012142
- Term: Respiratory Tract Neoplasms
- ID: D000013899
- Term: Thoracic Neoplasms
- ID: D000009371
- Term: Neoplasms by Site
- ID: D000009369
- Term: Neoplasms
- ID: D000008171
- Term: Lung Diseases
- ID: D000012140
- Term: Respiratory Tract Diseases
- ID: D000002283
- Term: Carcinoma, Bronchogenic
- ID: D000001984
- Term: Bronchial Neoplasms
- ID: D000009385
- Term: Neoplastic Processes
- ID: D000010335
- Term: Pathologic Processes
- ID: D000016543
- Term: Central Nervous System Neoplasms
- ID: D000009423
- Term: Nervous System Neoplasms
- ID: D000001927
- Term: Brain Diseases
- ID: D000002493
- Term: Central Nervous System Diseases
- ID: D000009422
- Term: Nervous System Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: BC08
- Name: Respiratory Tract (Lung and Bronchial) Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: BC10
- Name: Nervous System Diseases
### Condition Browse Module - Browse Leaves
- ID: M11172
- Name: Lung Neoplasms
- Relevance: HIGH
- As Found: Lung Cancer
- ID: M5546
- Name: Carcinoma, Non-Small-Cell Lung
- Relevance: HIGH
- As Found: Non-Small Cell Lung Cancer
- ID: M12307
- Name: Neoplasm Metastasis
- Relevance: HIGH
- As Found: Metastases
- ID: M20559
- Name: Disease Progression
- Relevance: LOW
- As Found: Unknown
- ID: M5534
- Name: Carcinoma
- Relevance: LOW
- As Found: Unknown
- ID: M5209
- Name: Brain Neoplasms
- Relevance: HIGH
- As Found: Brain Metastases
- ID: M14979
- Name: Respiratory Tract Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M16658
- Name: Thoracic Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M11168
- Name: Lung Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M14977
- Name: Respiratory Tract Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5540
- Name: Carcinoma, Bronchogenic
- Relevance: LOW
- As Found: Unknown
- ID: M5260
- Name: Bronchial Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M12330
- Name: Neoplastic Processes
- Relevance: LOW
- As Found: Unknown
- ID: M18937
- Name: Central Nervous System Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M12367
- Name: Nervous System Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M5204
- Name: Brain Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5742
- Name: Central Nervous System Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000008175
- Term: Lung Neoplasms
- ID: D000002289
- Term: Carcinoma, Non-Small-Cell Lung
- ID: D000009362
- Term: Neoplasm Metastasis
- ID: D000001932
- Term: Brain Neoplasms
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT00950079
**Brief Title:** Prevention of Contrast-Induced Nephropathy in Diabetic Patients With Undergoing Coronary Angiography
**Official Title:** Preventive Strategies of REnal Insufficiency in Patients With Diabetes Undergoing InterVENTion or Arteriography(PREVENT Trial)
#### Organization Study ID Info
**ID:** 2007-0180
#### Organization
**Class:** OTHER
**Full Name:** CardioVascular Research Foundation, Korea
### Status Module
#### Completion Date
**Date:** 2010-01
**Type:** ESTIMATED
#### Expanded Access Info
**Last Known Status:** RECRUITING
#### Last Update Post Date
**Date:** 2009-07-31
**Type:** ESTIMATED
**Last Update Submit Date:** 2009-07-30
**Overall Status:** UNKNOWN
#### Primary Completion Date
**Date:** 2009-08
**Type:** ESTIMATED
#### Start Date
**Date:** 2008-02
**Status Verified Date:** 2009-07
#### Study First Post Date
**Date:** 2009-07-31
**Type:** ESTIMATED
**Study First Submit Date:** 2009-07-06
**Study First Submit QC Date:** 2009-07-30
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** CardioVascular Research Foundation, Korea
#### Responsible Party
**Old Name Title:** Seong-Wook Park, MD, PhD
**Old Organization:** Asan Medical Center
### Oversight Module
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** The aim of this study is to determine whether sodium bicarbonate is superior to sodium chloride in preventing contrast induced nephropathy in diabetic patients undergoing coronary angiography or intervention.
**Detailed Description:** Contrast induced nephropathy (CIN) is a common cause of renal failure associated with prolonged hospitalization, significant morbidity or mortality, and cost. CIN has been reported to account for 10% of hospital acquired renal failure. Several strategies or medications were tried to prevent CIN. Of these, mucomyst and normal saline infusion are effective to prevent CIN. Sodium bicarbonate has shown mixed results from recent randomized trials. Moreover,limited data have been available for preventing CIN in diabetic renal dysfunction undergoing coronary angiography or intervention. Our hypothesis is that addition of sodium bicarbonate will be more efficacious than normal saline in preventing CIN at above situation.
### Conditions Module
**Conditions:**
- Contrast Induced Nephropathy
**Keywords:**
- sodium bicarbonate
- diabetes mellitus
- coronary angiography or intervention
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** SINGLE
**Who Masked:**
- PARTICIPANT
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 368
**Type:** ESTIMATED
**Phases:**
- PHASE4
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** sodium bicarbonate
**Intervention Names:**
- Drug: Sodium bicarbonate
**Label:** Sodium bicarbonate
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** saline infusion
**Intervention Names:**
- Drug: saline
**Label:** Saline
**Type:** ACTIVE_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Sodium bicarbonate
**Description:** 3 mL/kg for 1 hour immediately before contrast injection , 1mg/kg/hr during procedure and 6 hours after procedure
**Name:** Sodium bicarbonate
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- Saline
**Description:** 0.9% normal saline with 1 mL/kg/hr infusion 12 hours before procedure and 12 hours after procedure
**Name:** saline
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Measure:** Incidence of contrast-induced nephropathy
**Time Frame:** 24 and 48 hours after angiography or intervention
#### Secondary Outcomes
**Measure:** all-cause mortality
**Time Frame:** 30 days after index angiography
**Measure:** stroke
**Time Frame:** 30 days after index angiography
**Measure:** Dialysis
**Time Frame:** 30 days after index angiography
**Measure:** Myocardial infarction
**Time Frame:** 30 days after index angiography
**Measure:** all-cause mortality
**Time Frame:** 30 days to 6 months after index angiography
**Measure:** stroke
**Time Frame:** 30 days to 6 months after index angiography
**Measure:** dialysis
**Time Frame:** 30 days to 6 months after index angiography
**Measure:** myocardial infarction
**Time Frame:** 30 days to 6 months after index angiography
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* age\>18 years, no upper limits
* diabetes treated with insulin or oral hypoglycemic agents
* serum creatinine \>=1.1 and
* resting estimated glomerular filtration rate \<60ml/min/1.73 square meter
Exclusion Criteria:
* serum creatinine \>=8mg/dL
* resting estimated GFR \<15ml/min/1.73 square meter
* end stage renal disease on hemodialysis
* multiple myeloma
* uncontrolled hypertension (systolic BP \>160mmHg or diastolic BP\>100mmHg)
* acute myocardial infarction
* emergent coronary angioplasty or angiography
* recent use of contrast within 2 days
* allergic reaction to contrast
* pregnancy
* allergic to following medication : theophylline, dopamine, mannitol, fenoldopam, N-acetylcysteine
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Seong-Wook Park, MD, PhD
**Phone:** 2-3010-3153
**Phone Ext:** 82
**Role:** CONTACT
**Contact 2:**
**Email:** [email protected]
**Name:** Seung-Whan Lee, MD, PhD
**Phone:** 2-3010-3170
**Phone Ext:** 82
**Role:** CONTACT
#### Locations
**Location 1:**
**City:** Bucheon
**Contacts:**
***Contact 1:***
- **Name:** Nae-Hee Lee, MD, PhD
- **Role:** CONTACT
***Contact 2:***
- **Name:** Nae-Hee Lee, MD, PhD
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** Korea, Republic of
**Facility:** Soonchunhyang University Bucheon Hospital
**Status:** RECRUITING
**Location 2:**
**City:** Busan
**Contacts:**
***Contact 1:***
- **Name:** Jae-Sik Jang, MD
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jae-Sik Jang, MD
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** Korea, Republic of
**Facility:** Busan Saint Mary Hospital
**Status:** RECRUITING
**Location 3:**
**City:** Cheonan
**Contacts:**
***Contact 1:***
- **Name:** Won-Yong Shin, MD, PhD
- **Role:** CONTACT
***Contact 2:***
- **Name:** Dong-Gyu Jin, MD, PhD
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** Korea, Republic of
**Facility:** Soonchunhyang University Hospital, Cheonan
**Status:** RECRUITING
**Location 4:**
**City:** Chuncheon
**Contacts:**
***Contact 1:***
- **Name:** Bong-Ki Lee, MD, PhD
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bong-Ki Lee
- **Role:** CONTACT
***Contact 3:***
- **Name:** Bong-Ki Lee, MD, PhD
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** Korea, Republic of
**Facility:** Kangwon National University Hospital
**Status:** RECRUITING
**Location 5:**
**City:** Daejeon
**Contacts:**
***Contact 1:***
- **Name:** In-Whan Seong, MD, PhD
- **Role:** CONTACT
***Contact 2:***
- **Name:** In-Whan Seong, MD, PhD
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** Korea, Republic of
**Facility:** Chungnam National University Hospital
**Status:** RECRUITING
**Location 6:**
**City:** Gangneung
**Contacts:**
***Contact 1:***
- **Name:** Sang-Sik Cheong
- **Role:** CONTACT
***Contact 2:***
- **Name:** Sang-Sik Cheong, MD, PhD
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** Korea, Republic of
**Facility:** Gangneung Asan Hospital
**Status:** RECRUITING
**Location 7:**
**City:** Seoul
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Seong-Wook Park, MD, PhD
- **Phone:** 2-3010-3153
- **Phone Ext:** 82
- **Role:** CONTACT
***Contact 2:***
- **Email:** [email protected]
- **Name:** Seung-Whan Lee, MD, PhD
- **Phone:** 2-3010-3170
- **Phone Ext:** 82
- **Role:** CONTACT
***Contact 3:***
- **Name:** Seong-Wook Park, MD, PhD
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** Korea, Republic of
**Facility:** Asan Medical Center
**Status:** RECRUITING
**Zip:** 138-736
**Location 8:**
**City:** Seoul
**Contacts:**
***Contact 1:***
- **Name:** Min-Su Hyun, MD, PhD
- **Role:** CONTACT
***Contact 2:***
- **Name:** Min-Su Hyun, MD, PhD
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** Korea, Republic of
**Facility:** Soonchunhyang University Seoul Hospital
**Status:** RECRUITING
**Location 9:**
**City:** Ulsan
**Contacts:**
***Contact 1:***
- **Name:** Sang-Gon Lee, MD, PhD
- **Role:** CONTACT
***Contact 2:***
- **Name:** Sang-Gon Lee, MD, PhD
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** Korea, Republic of
**Facility:** Ulsan University Hospital
**Status:** RECRUITING
#### Overall Officials
**Official 1:**
**Affiliation:** Department of Medicine, Asan Medical Center, University of Ulsan College of Medicine
**Name:** Seong-Wook Park, MD, PhD
**Role:** PRINCIPAL_INVESTIGATOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000014570
- Term: Urologic Diseases
- ID: D000052776
- Term: Female Urogenital Diseases
- ID: D000005261
- Term: Female Urogenital Diseases and Pregnancy Complications
- ID: D000091642
- Term: Urogenital Diseases
- ID: D000052801
- Term: Male Urogenital Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC18
- Name: Nutritional and Metabolic Diseases
- Abbrev: BC19
- Name: Gland and Hormone Related Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BXS
- Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions
### Condition Browse Module - Browse Leaves
- ID: M7115
- Name: Diabetes Mellitus
- Relevance: LOW
- As Found: Unknown
- ID: M10698
- Name: Kidney Diseases
- Relevance: HIGH
- As Found: Nephropathy
- ID: M26718
- Name: Renal Insufficiency
- Relevance: LOW
- As Found: Unknown
- ID: M17319
- Name: Urologic Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M2875
- Name: Urogenital Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M27093
- Name: Female Urogenital Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M14127
- Name: Pregnancy Complications
- Relevance: LOW
- As Found: Unknown
- ID: M8399
- Name: Female Urogenital Diseases and Pregnancy Complications
- Relevance: LOW
- As Found: Unknown
- ID: M27095
- Name: Male Urogenital Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000007674
- Term: Kidney Diseases
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT00804479
**Acronym:** CALM-PD Cohort
**Brief Title:** The Long Term Impact of Initiating Pramipexole Versus Levodopa in Early Parkinson's Disease (CALM-PD Cohort Study)
**Official Title:** Unblinded, Multicenter, Prospective Follow-up of Long-term Consequences of Initiating Patients With Parkinson's Disease on Either Pramipexole or Levodopa.
#### Organization Study ID Info
**ID:** PPXAPD-0072-138
#### Organization
**Class:** OTHER
**Full Name:** University of Rochester
#### Secondary ID Infos
**ID:** RSRB #09283
### Status Module
#### Completion Date
**Date:** 2004-03
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2016-01-21
**Type:** ESTIMATED
**Last Update Submit Date:** 2016-01-20
**Overall Status:** TERMINATED
#### Primary Completion Date
**Date:** 2004-03
**Type:** ACTUAL
#### Start Date
**Date:** 2002-01
**Status Verified Date:** 2016-01
#### Study First Post Date
**Date:** 2008-12-08
**Type:** ESTIMATED
**Study First Submit Date:** 2008-12-04
**Study First Submit QC Date:** 2008-12-05
**Why Stopped:** Funding for the CALM-PD Cohort Study was terminated by sponsor.
### Sponsor Collaborators Module
#### Collaborators
**Class:** UNKNOWN
**Name:** Pharmacia Corp. (Peapack, NJ)
**Class:** INDUSTRY
**Name:** Boehringer Ingelheim
#### Lead Sponsor
**Class:** OTHER
**Name:** University of Rochester
#### Responsible Party
**Investigator Affiliation:** University of Rochester
**Investigator Full Name:** Robert Holloway
**Investigator Title:** Professor
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** To determine the long-term consequences (8 years) of initiating patients with Parkinson's disease on either pramipexole or levodopa. We hypothesize that patients initiating therapy with pramipexole compared with levodopa will demonstrate less self-reported disability as measured by the Modified Schwab and England (S/E) scale 8 years after randomization.
### Conditions Module
**Conditions:**
- Parkinson's Disease
### Design Module
#### Design Info
**Observational Model:** COHORT
**Time Perspective:** PROSPECTIVE
#### Enrollment Info
**Count:** 222
**Type:** ACTUAL
**Study Type:** OBSERVATIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Available 301 subjects enrolled in CALM-PD Available 82 subjects enrolled in CALM-PD imaging substudy
**Intervention Names:**
- Other: No intervention.
**Label:** no treatment
### Interventions
#### Intervention 1
**Arm Group Labels:**
- no treatment
**Name:** No intervention.
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Measure:** We hypothesize that patients initiating therapy with pramipexole compared with levodopa will demonstrate less self-reported disability as measured by the Modified Schwab and England.
**Time Frame:** 8 years from date randomized in CALM study
#### Secondary Outcomes
**Measure:** Our secondary specific aim is to develop and estimate a structural model that will allow us to uncover the causal pathways through which treatments effect outcomes.
**Time Frame:** 8 years from randomization of CALM-PD study
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Available 301 subjects enrolled in the CALM-PD study.
Exclusion Criteria:
* Those not enrolled in the CALM-PD study.
**Sampling Method:** NON_PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- CHILD
- ADULT
- OLDER_ADULT
**Study Population:** Available 301 subjects enrolled in CALM-PD
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Rochester
**Country:** United States
**Facility:** University of Rochester
**State:** New York
**Zip:** 14620
### References Module
#### References
**Citation:** Parkinson Study Group CALM Cohort Investigators. Long-term effect of initiating pramipexole vs levodopa in early Parkinson disease. Arch Neurol. 2009 May;66(5):563-70. doi: 10.1001/archneur.66.1.nct90001.
**PMID:** 19433655
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000020734
- Term: Parkinsonian Disorders
- ID: D000001480
- Term: Basal Ganglia Diseases
- ID: D000001927
- Term: Brain Diseases
- ID: D000002493
- Term: Central Nervous System Diseases
- ID: D000009422
- Term: Nervous System Diseases
- ID: D000009069
- Term: Movement Disorders
- ID: D000080874
- Term: Synucleinopathies
- ID: D000019636
- Term: Neurodegenerative Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC10
- Name: Nervous System Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: BC17
- Name: Skin and Connective Tissue Diseases
- Abbrev: BC18
- Name: Nutritional and Metabolic Diseases
### Condition Browse Module - Browse Leaves
- ID: M13213
- Name: Parkinson Disease
- Relevance: HIGH
- As Found: Parkinson's Disease
- ID: M22494
- Name: Parkinsonian Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M25603
- Name: Ganglion Cysts
- Relevance: LOW
- As Found: Unknown
- ID: M16358
- Name: Synovial Cyst
- Relevance: LOW
- As Found: Unknown
- ID: M4774
- Name: Basal Ganglia Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5204
- Name: Brain Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5742
- Name: Central Nervous System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M12029
- Name: Movement Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M2217
- Name: Synucleinopathies
- Relevance: LOW
- As Found: Unknown
- ID: M21558
- Name: Neurodegenerative Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000010300
- Term: Parkinson Disease
### Intervention Browse Module - Browse Branches
- Abbrev: AnDyAg
- Name: Anti-Dyskinesia Agents
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M1813
- Name: Pramipexole
- Relevance: LOW
- As Found: Unknown
- ID: M3373
- Name: Dexpramipexole
- Relevance: LOW
- As Found: Unknown
- ID: M10982
- Name: Levodopa
- Relevance: LOW
- As Found: Unknown
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT01810679
**Brief Title:** Perceval S Aortic Heart Valve Study- North America
**Official Title:** Clinical Investigation of the Perceval S Sutureless Heart Valve
#### Organization Study ID Info
**ID:** G120053
#### Organization
**Class:** INDUSTRY
**Full Name:** Sorin Group USA, Inc.
### Status Module
#### Completion Date
**Date:** 2018-04
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2016-11-16
**Type:** ESTIMATED
**Last Update Submit Date:** 2016-11-15
**Overall Status:** TERMINATED
#### Primary Completion Date
**Date:** 2016-02
**Type:** ACTUAL
#### Start Date
**Date:** 2013-04
**Status Verified Date:** 2016-11
#### Study First Post Date
**Date:** 2013-03-13
**Type:** ESTIMATED
**Study First Submit Date:** 2013-03-07
**Study First Submit QC Date:** 2013-03-11
**Why Stopped:** FDA approval
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Sorin Group USA, Inc.
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** To demonstrate the safety and effectiveness of the Perceval S heart valve when used to replace a diseased or dysfunctional aortic valve or aortic valve prosthesis.
**Detailed Description:** This investigation is a prospective, non-randomized, multi-center trial of the PERCEVAL valve implanted in patients requiring aortic valve replacement. The study will be conducted in a maximum of 25 centers in the United States. Patients will be evaluated at each of the following time intervals:preoperative, at implant, in the early postoperative period, in the late postoperative period (between 3 and 6 months postoperatively), at 1 year (between 11 and 13 months postoperatively), and annually until study completion. The duration of the study is anticipated to be 5 years.
### Conditions Module
**Conditions:**
- Aortic Valve Stenosis
- Aortic Valve Stenosis With Insufficiency
- Regurgitation, Aortic Valve
- Aortic Valve Incompetence
**Keywords:**
- aortic valve replacement
- aortic stenosis
- aortic steno-insufficiency
- Perceval
- Sutureless aortic heart valve
### Design Module
#### Design Info
**Allocation:** NA
**Intervention Model:** SINGLE_GROUP
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 355
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Treatment with the Perceval S Aortic Heart Valve
**Intervention Names:**
- Device: Perceval S Aortic Heart Valve
**Label:** Perceval S Aortic Heart Valve
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Perceval S Aortic Heart Valve
**Description:** Perceval S Sutureless Aortic Heart Valve
**Name:** Perceval S Aortic Heart Valve
**Type:** DEVICE
### Outcomes Module
#### Other Outcomes
**Description:** Rates of cross-clamp time, cardiopulmonary bypass time, length of stay in ICU, length of hospital, and quality of life
**Measure:** Supplementary Analyses
**Time Frame:** Discharge
#### Primary Outcomes
**Description:** To demonstrate that the complication and survival rates for the PERCEVAL S valve are comparable to appropriate historical controls manifested as objective performance criteria (OPCs) and to that reported for other stented bioprostheses in the literature
**Measure:** Primary Safety Endpoint
**Time Frame:** One-year
**Description:** To demonstrate that the rate of valve success for the PERCEVAL S valve is comparable to that of appropriate historical controls
**Measure:** Primary Efficacy Endpoint
**Time Frame:** One-year
#### Secondary Outcomes
**Description:** To demonstrate that the hemodynamic performance of the PERCEVAL S valve is comparable to that reported in the literature for other stented aortic bioprostheses
**Measure:** Secondary Efficacy Outcomes
**Time Frame:** One-year
**Description:** To demonstrate clinically significant improvements in overall patient condition by comparison of preoperative and postoperative NYHA functional classifications
**Measure:** Secondary Efficacy Outcomes
**Time Frame:** One-year
**Description:** To demonstrate that the rate of device technical success for the PERCEVAL S valve is comparable to that of appropriate historical controls
**Measure:** Secondary Efficacy Outcomes
**Time Frame:** One-year
### Eligibility Module
**Eligibility Criteria:** Inclusion criteria:
1. Subjects of age \>= 18 years.
2. Subjects with aortic valve stenosis or steno-insufficiency.
3. The subject is willing to sign the informed consent.
4. The subject in which preoperative evaluation indicated the need for native or prosthetic aortic valve replacement.
5. The subject is located in a geographic location that will enable the subject to return to the study site for all follow-up examinations (i.e. geographically stable).
6. Subject will be available to the investigator(s) for postoperative follow-up beyond one year.
Exclusion criteria:
1. The subject has preexisting valve prosthesis or annuloplasty ring in the mitral, pulmonic or tricuspid position.
2. The subject requires a double or multiple valve replacement or repair of the mitral, tricuspid, or pulmonic valve.
3. The subject has a previously implanted PERCEVAL valve that requires replacement.
4. Subjects requiring simultaneous cardiac procedures, apart from septal myectomy and/or coronary by-pass.
5. The subject has active endocarditis.
6. Subjects with active myocarditis
7. The subject is or will be participating in a concomitant research study of an investigational product.
8. Subjects with aneurysmal dilation or dissection of the ascending aortic wall.
9. The subject is a minor, drug abuser, alcohol abuser, prison inmate, institutionalized, or is unable to give informed consent.
10. The subject has a major or progressive non-cardiac disease that, in the investigator's experience, results in a life expectancy of less than 1 year, or the implant of the device produces an unacceptable increased risk to the patient.
11. Subjects with known hypersensitivity to nickel alloys.
12. The subject is undergoing renal dialysis for chronic renal failure or has hyperparathyroidism.
13. The subject has had an acute preoperative neurological deficit, myocardial infarction, or cardiac event that has not returned to baseline or stabilized ≥30 days prior to the planned valve implant surgery.
14. Subject is known to be noncompliant or is unlikely to complete the study.
15. Subjects with an aortic root enlargement, where the ratio between the diameter of the sino-tubular junction and the annulus diameter, assessed by TTE, is \> 1.3.
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Opelika
**Country:** United States
**Facility:** East Alabama Medical Center
**State:** Alabama
**Zip:** 36801
**Location 2:**
**City:** Tucson
**Country:** United States
**Facility:** University of Arizona
**State:** Arizona
**Zip:** 85724
**Location 3:**
**City:** Aurora
**Country:** United States
**Facility:** University of Colorado Denver
**State:** Colorado
**Zip:** 80045
**Location 4:**
**City:** Jacksonville
**Country:** United States
**Facility:** St. Vincent's Medical Center
**State:** Florida
**Zip:** 32204
**Location 5:**
**City:** Miami
**Country:** United States
**Facility:** Baptist Cardiac and Vascular Institute
**State:** Florida
**Zip:** 33176
**Location 6:**
**City:** Atlanta
**Country:** United States
**Facility:** Emory University
**State:** Georgia
**Zip:** 30308
**Location 7:**
**City:** Indianapolis
**Country:** United States
**Facility:** St. Vincent Heart Center of Indiana
**State:** Indiana
**Zip:** 46290
**Location 8:**
**City:** Portland
**Country:** United States
**Facility:** Maine Medical Center
**State:** Maine
**Zip:** 04102
**Location 9:**
**City:** Baltimore
**Country:** United States
**Facility:** University of Maryland
**State:** Maryland
**Zip:** 21201
**Location 10:**
**City:** Dearborn
**Country:** United States
**Facility:** Oakwood Hospital
**State:** Michigan
**Zip:** 48124
**Location 11:**
**City:** Rochester
**Country:** United States
**Facility:** Mayo Clinic
**State:** Minnesota
**Zip:** 55905
**Location 12:**
**City:** New York
**Country:** United States
**Facility:** New York Presbyterian - Weill Cornell Medical Center
**State:** New York
**Zip:** 10065
**Location 13:**
**City:** New York
**Country:** United States
**Facility:** Montefiore Medical Center/Albert Einstein College of Medicine
**State:** New York
**Zip:** 10467
**Location 14:**
**City:** New York
**Country:** United States
**Facility:** Lenox Hill/NS-LIJ
**State:** New York
**Zip:** 11075
**Location 15:**
**City:** Cleveland
**Country:** United States
**Facility:** Cleveland Clinic
**State:** Ohio
**Zip:** 44195
**Location 16:**
**City:** Toledo
**Country:** United States
**Facility:** ProMedica Toledo Hospital
**State:** Ohio
**Zip:** 43606
**Location 17:**
**City:** Philadelphia
**Country:** United States
**Facility:** University of Pennsylvania
**State:** Pennsylvania
**Zip:** 19104
**Location 18:**
**City:** Austin
**Country:** United States
**Facility:** Cardiothoracic and Vascluar Surgeons
**State:** Texas
**Zip:** 78756
**Location 19:**
**City:** Charlottesville
**Country:** United States
**Facility:** University of Virginia
**State:** Virginia
**Zip:** 22908
**Location 20:**
**City:** Falls Church
**Country:** United States
**Facility:** Inova Heart and Vascular Institute
**State:** Virginia
**Zip:** 22042
**Location 21:**
**City:** Seattle
**Country:** United States
**Facility:** University of Washington
**State:** Washington
**Zip:** 98195
#### Overall Officials
**Official 1:**
**Affiliation:** The Cleveland Clinic
**Name:** Rakesh Suri, MD, DPhil
**Role:** PRINCIPAL_INVESTIGATOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000020763
- Term: Pathological Conditions, Anatomical
- ID: D000082862
- Term: Aortic Valve Disease
- ID: D000006349
- Term: Heart Valve Diseases
- ID: D000006331
- Term: Heart Diseases
- ID: D000002318
- Term: Cardiovascular Diseases
- ID: D000014694
- Term: Ventricular Outflow Obstruction
### Condition Browse Module - Browse Branches
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: All
- Name: All Conditions
- Abbrev: BC14
- Name: Heart and Blood Diseases
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M6475
- Name: Constriction, Pathologic
- Relevance: HIGH
- As Found: Stenosis
- ID: M4338
- Name: Aortic Valve Insufficiency
- Relevance: HIGH
- As Found: Regurgitation, Aortic Valve
- ID: M4340
- Name: Aortic Valve Stenosis
- Relevance: HIGH
- As Found: Aortic Valve Stenosis
- ID: M22519
- Name: Pathological Conditions, Anatomical
- Relevance: LOW
- As Found: Unknown
- ID: M2379
- Name: Aortic Valve Disease
- Relevance: LOW
- As Found: Unknown
- ID: M9437
- Name: Heart Valve Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M9419
- Name: Heart Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M17440
- Name: Ventricular Outflow Obstruction
- Relevance: LOW
- As Found: Unknown
- ID: T449
- Name: Aortic Valve Stenosis
- Relevance: HIGH
- As Found: Aortic Valve Stenosis
### Condition Browse Module - Meshes
- ID: D000001024
- Term: Aortic Valve Stenosis
- ID: D000001022
- Term: Aortic Valve Insufficiency
- ID: D000003251
- Term: Constriction, Pathologic
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT00664079
**Acronym:** Thyroid
**Brief Title:** Thyroid Hormones in Critically Ill Children
**Official Title:** Thyroid Hormone Deficiency in Critically Ill Children
#### Organization Study ID Info
**ID:** 2005-10-4547
#### Organization
**Class:** OTHER
**Full Name:** Children's Hospital of Philadelphia
### Status Module
#### Completion Date
**Date:** 2008-12
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2015-03-12
**Type:** ESTIMATED
**Last Update Submit Date:** 2015-03-11
**Overall Status:** TERMINATED
#### Primary Completion Date
**Date:** 2008-06
**Type:** ACTUAL
#### Start Date
**Date:** 2005-10
**Status Verified Date:** 2008-07
#### Study First Post Date
**Date:** 2008-04-22
**Type:** ESTIMATED
**Study First Submit Date:** 2008-04-18
**Study First Submit QC Date:** 2008-04-18
**Why Stopped:** Sluggish enrollment.
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Children's Hospital of Philadelphia
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** Thyroid hormones are substances naturally made by the body and are important to many of your body's basic functions such as breathing and brain function. We are investigating whether or not these hormones are at lower levels in critically ill children which could lead to further health problems. We hope to get a better understanding of hormone levels and their effects on critically ill children to better help other children in the future.
**Detailed Description:** We hypothesize that critically ill children that require vasoactive infusions and/or invasive mechanical ventilation have thyroid hormone alterations. We will measure TSH, tT3, fT3, rT3, tT4, fT4, adn tyrosine concentrations in critically ill children with hypotension and/or respiratory failure and correlate thyroid hormone alterations to severity of illness, intensity of therapeutic interventions, and associated morbidity and mortality by using clinical outcomes parameters.
### Conditions Module
**Conditions:**
- Hypotension
- Respiratory Failure
**Keywords:**
- Thyroid Deficiency
- Thyroid Stimulating Hormone
### Design Module
#### Bio Spec
**Description:** Whole blood and serum
**Retention:** SAMPLES_WITHOUT_DNA
#### Design Info
**Observational Model:** COHORT
**Time Perspective:** PROSPECTIVE
#### Enrollment Info
**Count:** 22
**Type:** ACTUAL
**Study Type:** OBSERVATIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Patients who are receiving vasoactive medications and/or are mechanically ventilated.
**Intervention Names:**
- Other: Blood draws
**Label:** 1
### Interventions
#### Intervention 1
**Arm Group Labels:**
- 1
**Description:** 17 mls of blood will be drawn over a 5 day period from either a central venous catheter/arterial line or with scheduled phlebotomy. The following labs will be run tT3, fT3, rT#, tT4, fT4, TSH, and tyrosine.
**Name:** Blood draws
**Other Names:**
- Critically Ill Children
- Vasoactive Infusions in Children
- Mechanical Venilation in Children
- Thyroid Deficiency
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Measure:** Certain critically ill children requiring vasoactive infusions and/or mechanical ventilation have low concentrations of tT3, fT3, tT4, fT4, elevated rT3, adn inappropriate low/normal TSH.
**Time Frame:** When patient has completed the study.
#### Secondary Outcomes
**Measure:** Critically ill children with more severe thyroid hormone deficiencies will have greater severity of illness, intensity of therapeutic intervention, organ dysfunction, and increased morbidity and mortality.
**Time Frame:** When study is completed.
**Measure:** Critically ill children requiring vasoactive infusions and/or mechanical ventilation are a population in the ICU that has thyroid hormone pertubation and significant morbidity and mortality.
**Time Frame:** At completion of study
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Informed Consent
* Age of less than 12 months and less than or equal to 18
* Patient must weigh greater than 10 kgs.
* Patients must require vasoactive infusions and/or mechanical ventilation.
* Patients must be enrolled within 24 hours of meeting eligibility.
Exclusion Criteria:
* Patient with known or presumed pre-existing thyroid disease will be excluded
* Patients who receive thyroid supplementation will be excluded
* Patients with known or presumed hypothalamic and/or pituitary dysfunction that have thyroid hormone concentration abnormalities not related to an acute illness.
* Patients who are intubated for airway protection only.
* Patients intubated for neuromuscular disease
* Pregnant patients.
* Patients receiving amiodarone supplementation
* Patients who received blood product transfusions equaling more than 1/2 of their blood volume.
**Maximum Age:** 18 Years
**Minimum Age:** 12 Months
**Sampling Method:** NON_PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- CHILD
- ADULT
**Study Population:** Patients who are admitted to the Pediatric Intensive Care Unit at the Children's Hospital of Philadelphia will be approached if they meet inclusion criteria.
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Philadelphia,
**Country:** United States
**Facility:** The Children's Hospital of Philadelphia
**State:** Pennsylvania
**Zip:** 19104
#### Overall Officials
**Official 1:**
**Affiliation:** Children's Hospital of Philadelphia
**Name:** Athena Zuppa, MD, MSCE
**Role:** PRINCIPAL_INVESTIGATOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000014652
- Term: Vascular Diseases
- ID: D000002318
- Term: Cardiovascular Diseases
- ID: D000012120
- Term: Respiration Disorders
- ID: D000012140
- Term: Respiratory Tract Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC08
- Name: Respiratory Tract (Lung and Bronchial) Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC14
- Name: Heart and Blood Diseases
- Abbrev: BC19
- Name: Gland and Hormone Related Diseases
- Abbrev: BC23
- Name: Symptoms and General Pathology
### Condition Browse Module - Browse Leaves
- ID: M14968
- Name: Respiratory Insufficiency
- Relevance: HIGH
- As Found: Respiratory Failure
- ID: M10072
- Name: Hypotension
- Relevance: HIGH
- As Found: Hypotension
- ID: M16718
- Name: Thyroid Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M19010
- Name: Critical Illness
- Relevance: LOW
- As Found: Unknown
- ID: M17400
- Name: Vascular Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M14957
- Name: Respiration Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M14977
- Name: Respiratory Tract Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000012131
- Term: Respiratory Insufficiency
- ID: D000007022
- Term: Hypotension
### Intervention Browse Module - Browse Branches
- Abbrev: All
- Name: All Drugs and Chemicals
- Abbrev: AA
- Name: Amino Acids
### Intervention Browse Module - Browse Leaves
- ID: M9789
- Name: Hormones
- Relevance: LOW
- As Found: Unknown
- ID: T22
- Name: Tyrosine
- Relevance: LOW
- As Found: Unknown
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT03949179
**Brief Title:** Optimizing Management of Low Back Pain Through the Pain and Disability Drivers Management Model
**Official Title:** Optimizing Management of Low Back Pain Through the Pain and Disability Drivers Management Model: Study Protocol for a Feasibility Trial
#### Organization Study ID Info
**ID:** MP-31-2019-3131
#### Organization
**Class:** OTHER
**Full Name:** Université de Sherbrooke
### Status Module
#### Completion Date
**Date:** 2019-12-15
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2020-03-24
**Type:** ACTUAL
**Last Update Submit Date:** 2020-03-23
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2019-12-15
**Type:** ACTUAL
#### Start Date
**Date:** 2019-05-03
**Type:** ACTUAL
**Status Verified Date:** 2020-03
#### Study First Post Date
**Date:** 2019-05-14
**Type:** ACTUAL
**Study First Submit Date:** 2019-04-29
**Study First Submit QC Date:** 2019-05-10
### Sponsor Collaborators Module
#### Collaborators
**Class:** OTHER
**Name:** Quebec Pain Research Network
#### Lead Sponsor
**Class:** OTHER
**Name:** Université de Sherbrooke
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** This study aims to determine the feasibility of conducting a future randomized controlled trial to collect preliminary data on the effectiveness of a previously validated approach that takes into account all the pain and disability vectors associated with low back pain - the Pain and Disability Drivers Management Model (PDDM).
The overall objective is to provide data to assess the feasibility of implementing a future randomized clinical trial to evaluate the impact of the PDDM on the management of non-specific LBP in a clinical setting and to explore the short-term effect of using the model on patient's clinical outcomes.
**Detailed Description:** Background: Non-specific mechanical low back pain (LBP) is highly prevalent, recurrent and is a leading cause of disability worldwide. Despite increased efforts in improving care, the self-reported levels of disability in individuals with LBP have not improved in the last decade. In order to more effectively manage LBP through non-pharmacological approaches, evidence endorses the use of classification systems to support diagnosis and guide treatments. However, this approach to care is not without limitations and a more comprehensive and broader perspective is needed. Hence, we recently proposed and validated the Low Back Pain and Disability Drivers Management (PDDM) model, which aims to identify the domains influencing pain and disability to create a profile or phenotype to guide clinical decisions. The objective of this study is to assess the feasibility of conducting a trial and to gather preliminary effect outcomes of the intervention in clinical setting.
Methods: The design is a prospective single arm experimental design. Physiotherapists (PTs) working with a population suffering from musculoskeletal disorders and patients presenting with non-specific mechanical LBP from two different clinical settings will be recruited. The intervention consists of a one-day training workshop for PTs on using the PDDM to guide management decisions. PTs will then perform a 6-weeks follow-up with their participating patients. This feasibility study will assess recruitment and retention rates as well as PTs' acceptability and patients' satisfaction outcomes related to the intervention. Preliminary effect outcomes will also be gathered.
Discussion: This study will inform the feasibility and acceptability of the intervention and gather preliminary outcomes to conduct a future randomized controlled trial to measure the effectiveness of the intervention in managing non-specific mechanical LBP.
### Conditions Module
**Conditions:**
- Mechanical Low Back Pain
**Keywords:**
- Low back pain
- Pain management
- Rehabilitation
- Feasibility outcomes
### Design Module
#### Design Info
**Observational Model:** ECOLOGIC_OR_COMMUNITY
**Time Perspective:** PROSPECTIVE
#### Enrollment Info
**Count:** 86
**Type:** ACTUAL
**Study Type:** OBSERVATIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Participating clinicians will use the PDDM model to guide assessment and treatment of their patients for a 6-weeks period.
**Intervention Names:**
- Device: The Low Back Pain and Disability Drivers Management (PDDM) model
**Label:** Pain and Disability Drivers Management model
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Pain and Disability Drivers Management model
**Description:** The Low Back Pain and Disability Drivers Management (PDDM) model aims to identify the domains influencing pain and disability to guide clinical decisions. The model is composed of five domains upon which the clinician can base his assessment and orientate treatment allocation and includes: 1) nociceptive pain drivers (i.e., somatic, inflammatory or mixed pain), 2) nervous system dysfunction (NSD) drivers (i.e., sensitization of the peripheral and/or central nervous system), 3) comorbidity drivers (i.e., physical and/or mental health comorbidities), 4) cognitive-emotional drivers (i.e., maladaptive cognitions and/or behaviors) and 5) contextual drivers (i.e., occupational-related and social environmental contextual drivers). This profiling will inform and lead the clinician's treatment approach based on the combined contribution of each domain driving the experience of pain and disability.
**Name:** The Low Back Pain and Disability Drivers Management (PDDM) model
**Type:** DEVICE
### Outcomes Module
#### Primary Outcomes
**Description:** Recruitment rate: % of eligible clinicians who enrolled in the study (T1)
**Measure:** Feasibility of implementation
**Time Frame:** T1: clinician's baseline.
**Description:** Retention rate: % of contacted clinicians who accepted to participate and report data (T3-T1)
**Measure:** Feasibility of implementation
**Time Frame:** T1: clinician's baseline; T3 (6 weeks)
**Description:** Assessed via semi-structured phone interviews. It includes the clinician's appreciation of the training.
**Measure:** Clinician's acceptability of the workshop
**Time Frame:** T1: clinician's baseline (after the workshop)
**Description:** Assessed via semi-structured phone interviews and include clinician's perception of suitability of the assessment procedures to refine the diagnosis and to target adequate treatment.
**Measure:** Clinician's acceptability of the intervention
**Time Frame:** T3 (6 weeks)
#### Secondary Outcomes
**Description:** Change in BPI scores measured at T2 and T3 (T3-T2)
**Measure:** Nociceptive pain drivers : Change in Brief Pain Inventory (BPI) scores at 6 weeks
**Time Frame:** At T2: patient initial visit; T3: +6 weeks after initial visit
**Description:** Change in Pain Detect Questionnaire scores measured at T2 and T3 (T3-T2)
**Measure:** Nervous system dysfunction drivers: Change in Pain Detect Questionnaire scores at 6 weeks
**Time Frame:** At T2: patient initial visit; T3: +6 weeks after initial visit
**Description:** Change in CSI scores measured at T2 and T3 (T3-T2)
**Measure:** Nervous system dysfunction drivers: Change in Central Sensitization Index (CSI) scores at 6 weeks
**Time Frame:** At T2: patient initial visit; T3: +6 weeks after initial visit
**Description:** Change in SBT scores measured at T2 and T3 (T3-T2)
**Measure:** Cognitive-emotional drivers: Change in StartBackTool (SBT) scores at 6 weeks
**Time Frame:** At T2: patient initial visit; T3: +6 weeks after initial visit
**Description:** Change in OMPSQ scores measured at T2 and T3 (T3-T2)
**Measure:** Contextual drivers: Change in Orebro Musculoskeletal Pain Screening Questionnaire (OMPSQ) scores at 6 weeks
**Time Frame:** At T2: patient initial visit; T3: +6 weeks after initial visit
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
Clinicians:
* be working with a population suffering from musculoskeletal disorders such as LBP and have a valid license to practice physiotherapy in the province of Quebec
* agree to participate to the one-day training workshop (intervention)
* assess and initiate treatment of their patients presenting with non-specific LBP guided by our newly developed model
**Minimum Age:** 18 Years
**Sampling Method:** NON_PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
**Study Population:** Patients:
* Patients presenting with non-specific LBP according to the assessment conducted by the PT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Montréal
**Country:** Canada
**Facility:** Réseau de clinique PhysioExtra
**State:** Quebec
**Zip:** H2B 1J9
**Location 2:**
**City:** Sherbrooke
**Country:** Canada
**Facility:** CIUSSS de l'Estrie - CHUS
**State:** Quebec
**Zip:** J1H 5H3
#### Overall Officials
**Official 1:**
**Affiliation:** School of Rehabilitation, University of Sherbrooke
**Name:** Yannick Tousignant-Laflamme, PhD
**Role:** PRINCIPAL_INVESTIGATOR
### IPD Sharing Statement Module
**IPD Sharing:** NO
### References Module
#### References
**Citation:** Longtin C, Decary S, Cook CE, Martel MO, Lafrenaye S, Carlesso LC, Naye F, Tousignant-Laflamme Y. Optimizing management of low back pain through the pain and disability drivers management model: A feasibility trial. PLoS One. 2021 Jan 20;16(1):e0245689. doi: 10.1371/journal.pone.0245689. eCollection 2021.
**PMID:** 33471827
#### See Also Links
**Label:** Website (online resource) linked to the study that will be available to the participating clinicians in order to facilitate their use of the PDDM model
**URL:** https://pddmmodel.wordpress.com/
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000010146
- Term: Pain
- ID: D000009461
- Term: Neurologic Manifestations
### Condition Browse Module - Browse Branches
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: All
- Name: All Conditions
- Abbrev: BC10
- Name: Nervous System Diseases
### Condition Browse Module - Browse Leaves
- ID: M4714
- Name: Back Pain
- Relevance: HIGH
- As Found: Back Pain
- ID: M19433
- Name: Low Back Pain
- Relevance: HIGH
- As Found: Low Back Pain
- ID: M13066
- Name: Pain
- Relevance: LOW
- As Found: Unknown
- ID: M12404
- Name: Neurologic Manifestations
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000001416
- Term: Back Pain
- ID: D000017116
- Term: Low Back Pain
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT03465579
**Acronym:** BIOPSTAGE
**Brief Title:** Mp-3TMRI and 68Ga-PSMA PET/CT Guided Prostate Biopsy and Tumor Node Metastasis (TNM) Staging.
**Official Title:** Multi-cohort Investigational Study to Evaluate the Impact of Pelvic Mp-3TMRI and Whole-body 68Ga-PSMA PET/CT for Diagnosis of Clinically-significant Prostate Cancer and Pre-surgical Staging.
#### Organization Study ID Info
**ID:** IRST185.05
#### Organization
**Class:** OTHER
**Full Name:** Istituto Scientifico Romagnolo per lo Studio e la cura dei Tumori
### Status Module
#### Completion Date
**Date:** 2024-01
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2023-06-01
**Type:** ACTUAL
**Last Update Submit Date:** 2023-05-31
**Overall Status:** RECRUITING
#### Primary Completion Date
**Date:** 2024-01
**Type:** ESTIMATED
#### Start Date
**Date:** 2018-05-23
**Type:** ACTUAL
**Status Verified Date:** 2023-05
#### Study First Post Date
**Date:** 2018-03-14
**Type:** ACTUAL
**Study First Submit Date:** 2018-03-08
**Study First Submit QC Date:** 2018-03-08
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Istituto Scientifico Romagnolo per lo Studio e la cura dei Tumori
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** BIOPSTAGE is a prospective, non-randomized, diagnostic, multi-cohort investigational study to evaluate the impact of pelvic Multi-parametric 3-Tesla magnetic resonance imaging (mp-3TMRI) and whole-body 68Ga-PSMA PET/CT for diagnosis of clinically-significant prostate cancer and pre-surgical staging.
**Detailed Description:** The aim of this study is to characterize the diagnostic accuracy of both multi-parametric pelvic Magnetic Resonance Imaging (MRI) (T2-weighted, Diffusion Weighted Imaging (DWI), Dynamic Contrast Enhancement (DCE) and 68Ga-chelated Prostate Specific Membrane Antigen ligand (68Ga-PSMA) Positron Emission Tomography/Computed Tomography (PET /CT) in three cohorts of patients:
* COHORT 1 Guiding prostate biopsies in men with clinical suspicion and/or unconfirmed clinically-significant prostate cancer (CS-PCa) on initial prostate biopsy;
* COHORT 2 Targeting repeat prostate biopsy in patients on Active Surveillance (PRIAS Study), scheduled for PRIAS repeat biopsy;
* COHORT 3
Providing pelvic / whole-body pre-surgical staging in:
* 3a: men with high-risk PCa (HR-PCa);
* 3b: men candidates for nerve sparing surgery (NSS);
### Conditions Module
**Conditions:**
- 68Ga-PSMA PET/CT Guided Prostate Biopsy
- Mp-3TMRI Guided Prostate Biopsy
- Prostate Cancer
- TNM Staging
- Diagnosis
**Keywords:**
- Diagnosis Prostate Cancer
- TNM staging
- 68Ga-PSMA PET/CT guided prostate biopsy
- mp-3TMRI guided prostate biopsy
- pre-surgical staging
- clinically-suspected prostate cancer
### Design Module
#### Design Info
**Allocation:** NON_RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** NONE
**Primary Purpose:** DIAGNOSTIC
#### Enrollment Info
**Count:** 306
**Type:** ESTIMATED
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Men with suspected clinically-significant PCa (CS-PCa) who are candidates for prostate biopsy or after first-round negative transrectal ultrasonography (TRUS) biopsy
**Intervention Names:**
- Diagnostic Test: pelvic MRI
- Diagnostic Test: 68Ga-PSMA PET/CT
**Label:** Cohort 1
**Type:** OTHER
#### Arm Group 2
**Description:** Men framed in Active Surveillance (PRIAS study), scheduled for PRIAS repeat biopsy.
**Intervention Names:**
- Diagnostic Test: pelvic MRI
- Diagnostic Test: 68Ga-PSMA PET/CT
**Label:** Cohort 2
**Type:** OTHER
#### Arm Group 3
**Description:** Men with high-risk PCa (HR-PCa) prior to radical surgery.
**Intervention Names:**
- Diagnostic Test: pelvic MRI
- Diagnostic Test: 68Ga-PSMA PET/CT
**Label:** Cohort 3a
**Type:** OTHER
#### Arm Group 4
**Description:** Men diagnosed with CS-PCa prior to nerve-sparing prostate surgery (NSS).
**Intervention Names:**
- Diagnostic Test: pelvic MRI
- Diagnostic Test: 68Ga-PSMA PET/CT
**Label:** Cohort 3b
**Type:** OTHER
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Cohort 1
- Cohort 2
- Cohort 3a
- Cohort 3b
**Description:** multi-parametric pelvic MRI
**Name:** pelvic MRI
**Type:** DIAGNOSTIC_TEST
#### Intervention 2
**Arm Group Labels:**
- Cohort 1
- Cohort 2
- Cohort 3a
- Cohort 3b
**Description:** 68Ga-PSMA PET/CT
**Name:** 68Ga-PSMA PET/CT
**Type:** DIAGNOSTIC_TEST
### Outcomes Module
#### Primary Outcomes
**Description:** sensitivity of both 68Ga-PSMA PET/CT and mp-3TMRI imaging will be calculated as the ratio between the number of positive cases at 68Ga-PSMA PET/CT or mp-3TMRI and number of patients who underwent to these exams
**Measure:** sensitivity of both 68Ga-PSMA PET/CT and mp-3TMRI imaging
**Time Frame:** up to 36 months
**Description:** specificity will be calculated considering negative cases at 68Ga-PSMA PET/CT or mp-3TMRI and number of patients who underwent to these exams
**Measure:** specificity of both 68Ga-PSMA PET/CT and mp-3TMRI imaging
**Time Frame:** up to 36 months
**Description:** Positive predictive value will be calculated considering the ratio between positive patient at 68Ga-PSMA PET/CT/mp-3TMRI confirmed as positive throughout biopsy and overall positive patient at 68Ga-PSMA PET/CT/mp-3TMRI
**Measure:** Positive predictive value of both 68Ga-PSMA PET/CT and mp-3TMRI imaging
**Time Frame:** up to 36 months
**Description:** negative predictive value will be calculated considering the ratio between negative patient at 68Ga-PSMA PET/CT/mp-3TMRI confirmed as negative throughout biopsy and overall negative patient at 68Ga-PSMA PET/CT/mp-3TMRI
**Measure:** negative predictive value of both 68Ga-PSMA PET/CT and mp-3TMRI imaging
**Time Frame:** up to 36 months
#### Secondary Outcomes
**Description:** The number and percentage of treated patients undergoing grade 1 to 4 adverse events will be tabulated for each cohort and diagnostic procedure, using the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE) Version 4.03 should be used to assess and grade AE severity.
**Measure:** Incidence of adverse events
**Time Frame:** up to 30 days following study procedures
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
Cohort 1 - Biopsy guidance in clinically-suspected PCa
1. Men aged 45 to 75 years old with clinically-suspected PCa candidated for either initial or repeat TRUS-guided prostate biopsy, meeting any of the following criteria:
a) Abnormal PSA metrics, defined as follows:
A rising and/or persistently elevated serum PSA (i.e PSA \> 2.5ng/ml for men in the age group comprising 60 to 75 years old; PSA \> 3.0ng/ml for men in the age group comprising 45 to 60 years old) and at least one of the following PSA-based metrics:
i. Percent free PSA (%fPSA) \< 25% with PSA range 4-10ng/ml (NCCN); ii. PSA velocity (PSAvel) \>0.35 ng/mL/y; iii. PSA density (PSAden) \> 0.25ng/mL/cc iv. PSA \> 10ng/ml, 50% risk of PCa (EAU) b) Suspicious digital rectal examination (DRE), 5-30% risk of PCa; c) Prostate Cancer Gene 3 (PCA3) \> 35; d) Suspicious findings on first-round biopsy: i. A few Atypical Glands immediately adjacent to HG-PIN, 50% risk of PCa; ii. Atypical Small Acinar Proliferation, 40% of PCa; iii. Multifocal High-Grade Prostatic intraepithelial neoplasia (HG-PIN), 30% ; iv. Intraductal carcinoma as solitary finding, 90% of PCa;
Cohort 2 - Biopsy guidance on Active Surveillance
Men consenting to enter the PRIAS MRI side-study as per currently inclusion criteria in Version Number 1.0 dated August 20, 2013. These are:
1. Histologically-proven adenocarcinoma of the prostate;
2. Age ≥ 18
3. Men should be fit for curative treatment;
4. Clinical stage T1c or T2;
5. Gleason score 3+3=6;
6. One or two biopsy cores invaded with prostate cancer:
1. If an MRI, including targeted biopsies on positive lesions, is done at inclusion, there is no limit in the number of positive cores (that is, more than two, and no limit in the % of cancer present in the cores);
2. If saturation biopsies (either trans-perineal or trans-rectal) are done 15% of the cores can be positive with a maximum of 4 (i.e. 26 cores 4 cores can be positive) (all other inclusion criteria still apply);
7. PSA density (PSA D) less than 0.2;
8. PSA-level at diagnosis ≤ 10 ng/mL;
Cohort 3a - Pre-surgical TNM staging in high-risk prostate cancer
1. Male, aged 18 years or older;
2. Cyto / histological confirmation of PCa (i.e. TRUS-guided biopsies; TURP);
3. Any of the PCa high risk features for Organ-Confined Disease (OCD):
* Clinical T stage ≥ T2c;
* Gleason Score ≥ 8;
* Serum PSA \> 20 ng/mL;
4. Any of the PCa high-risk features for Locally-Advanced Disease (LAD):
* Clinical T stage ≥ T3b-T4 OR any T and clinical N1 disease;
* Gleason Score ≥ 8;
* Serum PSA \> 20 ng/mL;
5. Routine clinical staging (CTscan ± Bone scan) performed within 12 weeks enrolment returning negative or equivocal results for distant metastatic disease;
Cohort 3b - pelvic TNM staging of prostate cancer prior to nerve-sparing radical prostatectomy
1. Male, aged 18 years or older;
2. Cyto / histological confirmation of PCa (i.e. TRUS-guided biopsies; TURP);
3. All of the following PCa-related features must be met for nerve-sparing (either mono- or bi-lateral):
* Clinical T stage ≤ T2b;
* Gleason Score ≤ 7 (3+4) and maximum one biopsy with Gleason \> 6 at the ipsilateral side;
* Serum PSA \< 10 ng/mL;
Exclusion Criteria:
The participant may not enter the study if ANY of the following apply:
1. Hormone androgen deprivation therapy of any type within 6 months prior to enrollment.
2. Prior pelvic radiotherapy;
3. Sickle cell disease;
4. Insufficient renal function (eGFR \< 30 mL/min/1.73 m2);
5. Hip prosthesis, vascular grafting or other conditions affecting imaging;
6. Contraindication to MRI, including but not restricted to: pacemaker or other electronic im-plants, known metal in the orbit, MR incompatible surgical or cerebral aneurysm clips, shrapnel, tattoos, non-removable body piercings (relative contraindications);
7. History of allergic reactions attributed to compounds of similar chemical or biologic com-position to 68Ga-PSMA or Gadolinium-based contrast agents used in the study.
**Minimum Age:** 18 Years
**Sex:** MALE
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Oriana Nanni
**Phone:** +390543739266
**Role:** CONTACT
**Contact 2:**
**Email:** [email protected]
**Name:** Federica Matteucci, MD
**Phone:** +390543739100
**Role:** CONTACT
#### Locations
**Location 1:**
**City:** Forlì
**Contacts:**
***Contact 1:***
- **Name:** Roberta Gunelli
- **Role:** CONTACT
**Country:** Italy
**Facility:** AUSL della Romagna
**State:** FC
**Status:** RECRUITING
**Zip:** 47121
**Location 2:**
**City:** Meldola
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Federica Matteucci, MD
- **Phone:** +390543739100
- **Role:** CONTACT
**Country:** Italy
**Facility:** Istituto Scientifico Romagnolo per lo Studio e la Cura dei Tumori (IRST)
**State:** FC
**Status:** RECRUITING
**Zip:** 47014
#### Overall Officials
**Official 1:**
**Affiliation:** IRST IRCCS
**Name:** Federica Matteucci
**Role:** STUDY_CHAIR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000005834
- Term: Genital Neoplasms, Male
- ID: D000014565
- Term: Urogenital Neoplasms
- ID: D000009371
- Term: Neoplasms by Site
- ID: D000009369
- Term: Neoplasms
- ID: D000005832
- Term: Genital Diseases, Male
- ID: D000091662
- Term: Genital Diseases
- ID: D000091642
- Term: Urogenital Diseases
- ID: D000011469
- Term: Prostatic Diseases
- ID: D000052801
- Term: Male Urogenital Diseases
- ID: D000010335
- Term: Pathologic Processes
### Condition Browse Module - Browse Branches
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: BXS
- Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
### Condition Browse Module - Browse Leaves
- ID: M14335
- Name: Prostatic Neoplasms
- Relevance: HIGH
- As Found: Prostate Cancer
- ID: M12307
- Name: Neoplasm Metastasis
- Relevance: LOW
- As Found: Unknown
- ID: M8946
- Name: Genital Neoplasms, Male
- Relevance: LOW
- As Found: Unknown
- ID: M17315
- Name: Urogenital Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M2876
- Name: Genital Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M8944
- Name: Genital Diseases, Male
- Relevance: LOW
- As Found: Unknown
- ID: M2875
- Name: Urogenital Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M14333
- Name: Prostatic Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M27095
- Name: Male Urogenital Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000011471
- Term: Prostatic Neoplasms
- ID: D000004194
- Term: Disease
### Intervention Browse Module - Ancestors
- ID: D000019275
- Term: Radiopharmaceuticals
- ID: D000045504
- Term: Molecular Mechanisms of Pharmacological Action
### Intervention Browse Module - Browse Branches
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M352637
- Name: Gallium 68 PSMA-11
- Relevance: HIGH
- As Found: Independent
- ID: M21258
- Name: Radiopharmaceuticals
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: C000718244
- Term: Gallium 68 PSMA-11
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT04210479
**Brief Title:** Comparison of Bladder Filling vs. Non-Filling in Cesarean Hysterectomy for Placenta Percreta
**Official Title:** Bladder Filling in Cesarean Hysterectomy for Placenta Percreta: A Randomized Trial
#### Organization Study ID Info
**ID:** MS.19.12.939
#### Organization
**Class:** OTHER
**Full Name:** Mansoura University
### Status Module
#### Completion Date
**Date:** 2021-06-30
**Type:** ESTIMATED
#### Expanded Access Info
**Last Known Status:** NOT_YET_RECRUITING
#### Last Update Post Date
**Date:** 2019-12-24
**Type:** ACTUAL
**Last Update Submit Date:** 2019-12-23
**Overall Status:** UNKNOWN
#### Primary Completion Date
**Date:** 2021-03-31
**Type:** ESTIMATED
#### Start Date
**Date:** 2020-01-15
**Type:** ESTIMATED
**Status Verified Date:** 2019-12
#### Study First Post Date
**Date:** 2019-12-24
**Type:** ACTUAL
**Study First Submit Date:** 2019-12-21
**Study First Submit QC Date:** 2019-12-23
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Hatem AbuHashim
#### Responsible Party
**Investigator Affiliation:** Mansoura University
**Investigator Full Name:** Hatem AbuHashim
**Investigator Title:** Professor
**Type:** SPONSOR_INVESTIGATOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
### Description Module
**Brief Summary:** The placenta accreta spectrum (PAS) which includes accreta, increta, and percreta represents a significant obstetric challenge. PAS complicates as many as 1 per 500 pregnancies and this risk is increased with prior cesarean deliveries. Antenatal diagnosis of PAS allows for multidisciplinary planning and delivery before the onset of labor and/or vaginal bleeding. This approach has reduced maternal morbidity rates. including less blood loss, fewer transfusion requirements and, intraoperative urinary tract injury as well as improve fetal outcome.
Ultrasound evaluation is the recommended first-line modality for diagnosing PAS. Ultrasound features suggestive of PAS include loss of the normal retroplacental clear zone, attenuation of the uterine-bladder interface, reduced retroplacental myometrial thickness, presence of intraplacental lacunar spaces, and bridging vessels between the placenta and bladder. A systematic review reported that the antenatal diagnosis of PAS significantly lowered the rate of urinary tract injury (from 63% to 39%) during cesarean hysterectomies in these cases.
Unlike other elective cesarean hysterectomies, cesarean hysterectomy with a placenta previa increta/percreta, is more difficult. There is a greater need to both keep a margin from the vascular cervical-placental mass and simultaneously protect the urinary bladder. Case series reported that bladder filling helps the surgeon to more clearly identify the planes of dissection and secure the engorged aberrant vessels, thereby reduces bladder injury. Accordingly, a prospective randomized study in pregnant patients with placenta previa increta/percreta undergoing elective cesarean hysterectomy will be conducted to address this important issue.
### Conditions Module
**Conditions:**
- Placenta Accreta
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** SINGLE
**Who Masked:**
- OUTCOMES_ASSESSOR
**Primary Purpose:** PREVENTION
#### Enrollment Info
**Count:** 64
**Type:** ESTIMATED
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Bladder filling with 300ml diluted methylene blue.
**Intervention Names:**
- Procedure: Filled-bladder
**Label:** Filled-bladder
**Type:** EXPERIMENTAL
#### Arm Group 2
**Intervention Names:**
- Procedure: non filled-bladder
**Label:** non filled-bladder
**Type:** ACTIVE_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Filled-bladder
**Description:** Urinary bladder filling with 300 ml diluted methylene blue
**Name:** Filled-bladder
**Type:** PROCEDURE
#### Intervention 2
**Arm Group Labels:**
- non filled-bladder
**Description:** Pull up the empty (non-filled) urinary bladder using Allis forceps
**Name:** non filled-bladder
**Type:** PROCEDURE
### Outcomes Module
#### Primary Outcomes
**Description:** Unintentional bladder injury during elective cesarean hysterectomy
**Measure:** Rate of urinary bladder injury
**Time Frame:** Intra-operative (i.e. during surgery).
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Pregnant women in their third trimester (35-37 W).
* Placenta previa accreta spectrum identified by the Ultrasound (low lying anterior or major degree anterior).
* With at least one prior cesarean section.
* Elective cesarean hysterectomy.
* Evidence of gross placental invasion at the time of surgery (FIGO grade 3a.
Exclusion Criteria:
* Patients undergoing conservative treatment.
* Emergency cesarean hysterectomy.
* No evidence of gross placental invasion at the time of surgery.
* Posterior placenta.
**Maximum Age:** 44 Years
**Minimum Age:** 18 Years
**Sex:** FEMALE
**Standard Ages:**
- ADULT
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Hatem Abu Hashim, MD.FRCOG.PhD
**Phone:** +20502300002
**Role:** CONTACT
#### Overall Officials
**Official 1:**
**Affiliation:** Faculty of Medicine, Mansoura University
**Name:** Hatem Abu Hashim, MD. FRCOG. PhD
**Role:** STUDY_CHAIR
**Official 2:**
**Affiliation:** Samnoud General Hospital
**Name:** Mostafa Aboelenin, MBBCh
**Role:** PRINCIPAL_INVESTIGATOR
### References Module
#### References
**Citation:** Matsubara S. Caesarean hysterectomy for placenta praevia accreta: filling the bladder technique to identify an appropriate bladder separation site. J Obstet Gynaecol. 2013 Feb;33(2):163-4. doi: 10.3109/01443615.2012.740525. No abstract available.
**PMID:** 23445140
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000007744
- Term: Obstetric Labor Complications
- ID: D000011248
- Term: Pregnancy Complications
- ID: D000005261
- Term: Female Urogenital Diseases and Pregnancy Complications
- ID: D000091642
- Term: Urogenital Diseases
- ID: D000010922
- Term: Placenta Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BXS
- Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions
- Abbrev: All
- Name: All Conditions
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M13811
- Name: Placenta Accreta
- Relevance: HIGH
- As Found: Placenta Accreta
- ID: M10764
- Name: Obstetric Labor Complications
- Relevance: LOW
- As Found: Unknown
- ID: M14127
- Name: Pregnancy Complications
- Relevance: LOW
- As Found: Unknown
- ID: M2875
- Name: Urogenital Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M27093
- Name: Female Urogenital Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M8399
- Name: Female Urogenital Diseases and Pregnancy Complications
- Relevance: LOW
- As Found: Unknown
- ID: M13812
- Name: Placenta Diseases
- Relevance: LOW
- As Found: Unknown
- ID: T4583
- Name: Placenta Disorder
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000010921
- Term: Placenta Accreta
### Intervention Browse Module - Browse Branches
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M11726
- Name: Methylene Blue
- Relevance: LOW
- As Found: Unknown
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT00661479
**Brief Title:** An Exploratory Study to Evaluate the Safety of Brimonidine Intravitreal Implant in Patients With Retinitis Pigmentosa
#### Organization Study ID Info
**ID:** 190342-028D
#### Organization
**Class:** INDUSTRY
**Full Name:** Allergan
### Status Module
#### Completion Date
**Date:** 2010-05
**Type:** ACTUAL
#### Disp First Post Date
**Date:** 2011-02-09
**Type:** ESTIMATED
**Disp First Submit Date:** 2011-02-02
**Disp First Submit QC Date:** 2011-02-02
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2013-04-24
**Type:** ESTIMATED
**Last Update Submit Date:** 2013-03-13
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2009-12
**Type:** ACTUAL
#### Results First Post Date
**Date:** 2013-04-24
**Type:** ESTIMATED
**Results First Submit Date:** 2013-03-13
**Results First Submit QC Date:** 2013-03-13
#### Start Date
**Date:** 2008-07
**Status Verified Date:** 2013-03
#### Study First Post Date
**Date:** 2008-04-18
**Type:** ESTIMATED
**Study First Submit Date:** 2007-10-29
**Study First Submit QC Date:** 2008-04-16
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Allergan
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** This exploratory, 12-month, ascending-dose study will evaluate the safety and effects on visual function of a single injection of Brimonidine intravitreal implant in one eye of patients with Retinitis Pigmentosa.
### Conditions Module
**Conditions:**
- Retinitis Pigmentosa
### Design Module
#### Design Info
**Allocation:** NON_RANDOMIZED
**Intervention Model:** SINGLE_GROUP
##### Masking Info
**Masking:** SINGLE
**Who Masked:**
- PARTICIPANT
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 21
**Type:** ACTUAL
**Phases:**
- PHASE1
- PHASE2
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** 400 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
**Intervention Names:**
- Drug: 400 µg Brimonidine Tartrate Implant
- Other: Sham (no implant)
**Label:** 400 µg Brimonidine Tartrate Implant Group B
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** 200 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
**Intervention Names:**
- Drug: 200 µg Brimonidine Tartrate Implant
- Other: Sham (no implant)
**Label:** 200 µg Brimonidine Tartrate Implant Group B
**Type:** EXPERIMENTAL
#### Arm Group 3
**Description:** 100 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
**Intervention Names:**
- Drug: 100 µg Brimonidine Tartrate Implant
- Other: Sham (no implant)
**Label:** 100 µg Brimonidine Tartrate Implant Group B
**Type:** EXPERIMENTAL
#### Arm Group 4
**Description:** 100 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
**Intervention Names:**
- Drug: 100 µg Brimonidine Tartrate Implant
- Other: Sham (no implant)
**Label:** 100 µg Brimonidine Tartrate Implant Group A
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- 400 µg Brimonidine Tartrate Implant Group B
**Description:** 400 µg brimonidine tartrate implant in the study eye on Day 1.
**Name:** 400 µg Brimonidine Tartrate Implant
**Other Names:**
- Brimonidine Tartrate PS DDS®
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- 200 µg Brimonidine Tartrate Implant Group B
**Description:** 200 µg brimonidine tartrate implant in the study eye on Day 1.
**Name:** 200 µg Brimonidine Tartrate Implant
**Other Names:**
- Brimonidine Tartrate PS DDS®
**Type:** DRUG
#### Intervention 3
**Arm Group Labels:**
- 100 µg Brimonidine Tartrate Implant Group A
- 100 µg Brimonidine Tartrate Implant Group B
**Description:** 100 µg brimonidine tartrate implant in the study eye on Day 1.
**Name:** 100 µg Brimonidine Tartrate Implant
**Other Names:**
- Brimonidine Tartrate PS DDS®
**Type:** DRUG
#### Intervention 4
**Arm Group Labels:**
- 100 µg Brimonidine Tartrate Implant Group A
- 100 µg Brimonidine Tartrate Implant Group B
- 200 µg Brimonidine Tartrate Implant Group B
- 400 µg Brimonidine Tartrate Implant Group B
**Description:** Sham in the fellow eye on Day 1.
**Name:** Sham (no implant)
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Description:** BCVA is measured using an eye chart and is reported as the number of letters read correctly (ranging from 0 to 100 letters) in the study eye. The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity). A positive change from baseline indicates an improvement and a negative change from baseline indicates a worsening.
**Measure:** Change From Baseline in Best Corrected Visual Acuity (BCVA) in the Study Eye
**Time Frame:** Baseline, Month 6
#### Secondary Outcomes
**Description:** Change from baseline in contrast sensitivity in the study eye is measured using a Pelli-Robson contrast sensitivity chart at 1 meter. The contrast sensitivity chart contains letters that are darkest at the top and then get progressively lighter. Scores range from 0 to 48 and are based on the number of letters read correctly. A negative change from baseline indicates a worsening in contrast sensitivity and a positive change from baseline indicates an improvement.
**Measure:** Change From Baseline in Contrast Sensitivity in the Study Eye
**Time Frame:** Baseline, Month 6
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Retinitis Pigmentosa in both eyes
* Visual acuity between 20/40 to count fingers
Exclusion Criteria:
* Growth of new blood vessels in the eye
* Any intraocular surgery or laser in either eye in the last 6 months prior to Screening visit or between the Screening visit and Day 1
* Any ocular disease that can interfere with diagnosis and or assessment of disease progression
* Significant near-sightedness
* HIV
* Female patients who are pregnant, nursing, or planning pregnancy
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Arlington
**Country:** United States
**State:** Texas
**Location 2:**
**City:** Paris
**Country:** France
**Location 3:**
**City:** Tubingen
**Country:** Germany
**Location 4:**
**City:** Coimbra
**Country:** Portugal
#### Overall Officials
**Official 1:**
**Affiliation:** Allergan
**Name:** Medical Director
**Role:** STUDY_DIRECTOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000012164
- Term: Retinal Diseases
- ID: D000005128
- Term: Eye Diseases
- ID: D000015785
- Term: Eye Diseases, Hereditary
- ID: D000058499
- Term: Retinal Dystrophies
- ID: D000012162
- Term: Retinal Degeneration
- ID: D000030342
- Term: Genetic Diseases, Inborn
### Condition Browse Module - Browse Branches
- Abbrev: BC11
- Name: Eye Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC16
- Name: Diseases and Abnormalities at or Before Birth
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M15008
- Name: Retinitis
- Relevance: HIGH
- As Found: Retinitis
- ID: M15009
- Name: Retinitis Pigmentosa
- Relevance: HIGH
- As Found: Retinitis Pigmentosa
- ID: M14999
- Name: Retinal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M8271
- Name: Eye Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M18339
- Name: Eye Diseases, Hereditary
- Relevance: LOW
- As Found: Unknown
- ID: M29107
- Name: Retinal Dystrophies
- Relevance: LOW
- As Found: Unknown
- ID: M14997
- Name: Retinal Degeneration
- Relevance: LOW
- As Found: Unknown
- ID: M23686
- Name: Genetic Diseases, Inborn
- Relevance: LOW
- As Found: Unknown
- ID: T4945
- Name: Retinitis Pigmentosa
- Relevance: HIGH
- As Found: Retinitis Pigmentosa
### Condition Browse Module - Meshes
- ID: D000012173
- Term: Retinitis
- ID: D000012174
- Term: Retinitis Pigmentosa
### Intervention Browse Module - Ancestors
- ID: D000000959
- Term: Antihypertensive Agents
- ID: D000058647
- Term: Adrenergic alpha-2 Receptor Agonists
- ID: D000000316
- Term: Adrenergic alpha-Agonists
- ID: D000000322
- Term: Adrenergic Agonists
- ID: D000018663
- Term: Adrenergic Agents
- ID: D000018377
- Term: Neurotransmitter Agents
- ID: D000045504
- Term: Molecular Mechanisms of Pharmacological Action
- ID: D000045505
- Term: Physiological Effects of Drugs
### Intervention Browse Module - Browse Branches
- Abbrev: AnAg
- Name: Antihypertensive Agents
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M265
- Name: Brimonidine Tartrate
- Relevance: HIGH
- As Found: 1.4
- ID: M4277
- Name: Antihypertensive Agents
- Relevance: LOW
- As Found: Unknown
- ID: M20746
- Name: Adrenergic Agents
- Relevance: LOW
- As Found: Unknown
- ID: M3668
- Name: Adrenergic alpha-Agonists
- Relevance: LOW
- As Found: Unknown
- ID: M3673
- Name: Adrenergic Agonists
- Relevance: LOW
- As Found: Unknown
- ID: M20504
- Name: Neurotransmitter Agents
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: D000068438
- Term: Brimonidine Tartrate
### Misc Info Module
- Version Holder: 2024-05-24
## Results Section
### Adverse Events Module
**Description:** For Ocular AEs, only those occurring in the study eye are reported in the "Other Adverse Events" section. For SAEs, all ocular events were reported, regardless of eye.
#### Event Groups
**Group ID:** EG000
**Title:** 400 µg Brimonidine Tartrate Implant Group B
**Description:** 400 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
**ID:** EG000
**Other Num Affected:** 11
**Other Num at Risk:** 12
**Serious Number Affected:** 1
**Serious Number At Risk:** 12
**Title:** 400 µg Brimonidine Tartrate Implant Group B
**Group ID:** EG001
**Title:** 200 µg Brimonidine Tartrate Implant Group B
**Description:** 200 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
**ID:** EG001
**Other Num Affected:** 3
**Other Num at Risk:** 3
**Serious Number At Risk:** 3
**Title:** 200 µg Brimonidine Tartrate Implant Group B
**Group ID:** EG002
**Title:** 100 µg Brimonidine Tartrate Implant Groups A and B
**Description:** 100 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
**ID:** EG002
**Other Num Affected:** 6
**Other Num at Risk:** 6
**Serious Number Affected:** 1
**Serious Number At Risk:** 6
**Title:** 100 µg Brimonidine Tartrate Implant Groups A and B
**Frequency Threshold:** 5
#### Other Events
**Term:** Conjunctival Haemorrhage
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Conjunctival Hyperaemia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Foreign Body Sensation in Eyes
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Ocular Discomfort
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Nasopharyngitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA version 10.0
**Term:** Fatigue
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Glare
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Nausea
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Eye Pain
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Conjunctival Oedema
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Abdominal pain
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Abdominal Pain Upper
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Anxiety
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Psychiatric disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Bite
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Injury, poisoning and procedural complications
**Source Vocabulary:** MedDRA version 10.0
**Term:** Colour Blindness
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Congenital, familial and genetic disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Conjunctivitis Allergic
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Dehydration
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Dermatitis Allergic
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Dizziness
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Dry Eye
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Epididymitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Reproductive system and breast disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Eye Irritation
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Foot Fracture
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Injury, poisoning and procedural complications
**Source Vocabulary:** MedDRA version 10.0
**Term:** Headache
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Insomnia
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Psychiatric disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Lacrimation Increased
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Ligament Injury
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Injury, poisoning and procedural complications
**Source Vocabulary:** MedDRA version 10.0
**Term:** Myalgia
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Neuropathy Peripheral
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Orthostatic Hypotension
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Vascular disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Pharyngolaryngeal Pain
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Rhinitis
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA version 10.0
**Term:** Syncope
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Tinnitus
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Ear and labyrinth disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Vision Blurred
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Wound
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Injury, poisoning and procedural complications
**Source Vocabulary:** MedDRA version 10.0
**Term:** Visual Disturbance
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Abnormal Sensation in Eye
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Bacteriuria
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA version 10.0
**Term:** Bronchitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA version 10.0
**Term:** Ear Pain
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Ear and labyrinth disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Gastritis
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Influenza Like Illness
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Keratitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Muscle Contracture
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Myelitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Osteoarthritis
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Prostate Infection
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA version 10.0
**Term:** Renal Injury
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Injury, poisoning and procedural complications
**Source Vocabulary:** MedDRA version 10.0
**Term:** Restless Legs Syndrome
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Rhinitis Allergic
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Tendonitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Urinary Tract Infection
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA version 10.0
**Term:** Visual Acuity Reduced
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Vitreous Floaters
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Conjunctivitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Dental Caries
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA version 10.0
**Term:** Pharyngitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA version 10.0
#### Serious Events
**Term:** Syncope
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA version 10.0
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 12
**Group ID:** EG001
**Num At Risk:** 3
**Group ID:** EG002
**Num At Risk:** 6
**Term:** Myelitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA version 10.0
##### Stats
**Group ID:** EG000
**Num At Risk:** 12
**Group ID:** EG001
**Num At Risk:** 3
**Group ID:** EG002
**Num Affected:** 1
**Num At Risk:** 6
## Results Section - Baseline Characteristics Module
### Denomination Counts
**Group ID:** BG000
**Value:** 12
**Group ID:** BG001
**Value:** 3
**Group ID:** BG002
**Value:** 3
**Group ID:** BG003
**Value:** 3
**Group ID:** BG004
**Value:** 21
**Units:** Participants
### Group
**ID:** BG000
**Title:** 400 µg Brimonidine Tartrate Implant Group B
**Description:** 400 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
### Group
**ID:** BG001
**Title:** 200 µg Brimonidine Tartrate Implant Group B
**Description:** 200 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
### Group
**ID:** BG002
**Title:** 100 µg Brimonidine Tartrate Implant Group B
**Description:** 100 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
### Group
**ID:** BG003
**Title:** 100 µg Brimonidine Tartrate Implant Group A
**Description:** 100 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
### Group
**ID:** BG004
**Title:** Total
**Description:** Total of all reporting groups
### Measure
#### Measurement
**Group ID:** BG000
**Spread:** 18.70
**Value:** 46.3
#### Measurement
**Group ID:** BG001
**Spread:** 6.03
**Value:** 56.3
#### Measurement
**Group ID:** BG002
**Spread:** 4.04
**Value:** 48.7
#### Measurement
**Group ID:** BG003
**Spread:** 5.29
**Value:** 69.0
#### Measurement
**Group ID:** BG004
**Spread:** 16.36
**Value:** 51.3
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 5
#### Measurement
**Group ID:** BG001
**Value:** 1
#### Measurement
**Group ID:** BG002
**Value:** 3
#### Measurement
**Group ID:** BG003
**Value:** 0
#### Measurement
**Group ID:** BG004
**Value:** 9
**Category Title:** Female
#### Measurement
**Group ID:** BG000
**Value:** 7
#### Measurement
**Group ID:** BG001
**Value:** 2
#### Measurement
**Group ID:** BG002
**Value:** 0
#### Measurement
**Group ID:** BG003
**Value:** 3
#### Measurement
**Group ID:** BG004
**Value:** 12
**Category Title:** Male
**Class Title:**
**Denomination Units Selected:**
## Results Section - Baseline Characteristics Module
### Measure 1
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Age Continuous
**Unit of Measure:** Years
### Measure 2
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Sex: Female, Male
**Unit of Measure:** Participants
## Results Section - More Information Module
### Certain Agreement
**Other Details:** A disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 90 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo.
**Restriction Type:** OTHER
**Restrictive Agreement:** True
### Point of Contact
**Email:** [email protected]
**Organization:** Allergan, Inc
**Phone:** 714-246-4500
**Title:** Therapeutic Area Head,
## Results Section - Outcome Measures Module
### Outcome Measure 1
### Outcome Measure 2
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 14.61
- **Upper Limit:**
- **Value:** 48.5
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 14.18
- **Upper Limit:**
- **Value:** 51.0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:** 3.79
- **Upper Limit:**
- **Value:** 52.7
- **Comment:**
- **Group ID:** OG003
- **Lower Limit:**
- **Spread:** 3.46
- **Upper Limit:**
- **Value:** 4.0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 4.41
- **Upper Limit:**
- **Value:** 3.2
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 4.04
- **Upper Limit:**
- **Value:** -1.3
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:** 8.50
- **Upper Limit:**
- **Value:** 3.3
- **Comment:**
- **Group ID:** OG003
- **Lower Limit:**
- **Spread:** 3.79
- **Upper Limit:**
- **Value:** 0.3
**Title:**
#### Outcome Measure 2
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 8.48
- **Upper Limit:**
- **Value:** 15.5
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 7.21
- **Upper Limit:**
- **Value:** 18.0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:** 1.53
- **Upper Limit:**
- **Value:** 22.3
- **Comment:**
- **Group ID:** OG003
- **Lower Limit:**
- **Spread:** 3.46
- **Upper Limit:**
- **Value:** 3.0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 4.86
- **Upper Limit:**
- **Value:** 0.8
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 5.86
- **Upper Limit:**
- **Value:** -0.3
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:** 1.15
- **Upper Limit:**
- **Value:** 4.3
- **Comment:**
- **Group ID:** OG003
- **Lower Limit:**
- **Spread:** 1.73
- **Upper Limit:**
- **Value:** -2.0
**Title:**
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Description:** BCVA is measured using an eye chart and is reported as the number of letters read correctly (ranging from 0 to 100 letters) in the study eye. The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity). A positive change from baseline indicates an improvement and a negative change from baseline indicates a worsening.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** Safety Population: all patients treated on Day 1
**Reporting Status:** POSTED
**Time Frame:** Baseline, Month 6
**Title:** Change From Baseline in Best Corrected Visual Acuity (BCVA) in the Study Eye
**Type:** PRIMARY
**Unit of Measure:** Number of Letters Read Correctly
##### Group
**Description:** 400 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
**ID:** OG000
**Title:** 400 µg Brimonidine Tartrate Implant Group B
##### Group
**Description:** 200 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
**ID:** OG001
**Title:** 200 µg Brimonidine Tartrate Implant Group B
##### Group
**Description:** 100 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
**ID:** OG002
**Title:** 100 µg Brimonidine Tartrate Implant Group B
##### Group
**Description:** 100 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
**ID:** OG003
**Title:** 100 µg Brimonidine Tartrate Implant Group A
#### Outcome Measure 2
**Description:** Change from baseline in contrast sensitivity in the study eye is measured using a Pelli-Robson contrast sensitivity chart at 1 meter. The contrast sensitivity chart contains letters that are darkest at the top and then get progressively lighter. Scores range from 0 to 48 and are based on the number of letters read correctly. A negative change from baseline indicates a worsening in contrast sensitivity and a positive change from baseline indicates an improvement.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** Safety Population: all patients treated on Day 1
**Reporting Status:** POSTED
**Time Frame:** Baseline, Month 6
**Title:** Change From Baseline in Contrast Sensitivity in the Study Eye
**Type:** SECONDARY
**Unit of Measure:** Number of Letters Read Correctly
##### Group
**Description:** 400 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
**ID:** OG000
**Title:** 400 µg Brimonidine Tartrate Implant Group B
##### Group
**Description:** 200 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
**ID:** OG001
**Title:** 200 µg Brimonidine Tartrate Implant Group B
##### Group
**Description:** 100 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
**ID:** OG002
**Title:** 100 µg Brimonidine Tartrate Implant Group B
##### Group
**Description:** 100 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
**ID:** OG003
**Title:** 100 µg Brimonidine Tartrate Implant Group A
### Participant Flow Module
#### Group
**Description:** 400 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
**ID:** FG000
**Title:** 400 µg Brimonidine Tartrate Implant Group B
#### Group
**Description:** 200 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
**ID:** FG001
**Title:** 200 µg Brimonidine Tartrate Implant Group B
#### Group
**Description:** 100 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
**ID:** FG002
**Title:** 100 µg Brimonidine Tartrate Implant Group B
#### Group
**Description:** 100 µg brimonidine tartrate implant in the study eye and sham in the fellow eye on Day 1.
**ID:** FG003
**Title:** 100 µg Brimonidine Tartrate Implant Group A
#### Period
**Title:** Overall Study
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 12
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 3
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 3
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 3
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 12
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 3
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 3
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 3
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 0
**Pre-Assignment Details:** Patients were stratified by Best Corrected Visual Acuity (BCVA). Patients assigned to Group A had a BCVA of 20/320. Patients assigned to Group B had a BCVA worse than 20/40 and better than 20/320. Patients in Group A were randomized and treated prior to initiating enrollment in Group B. No patients from Group A participated in Group B.
**Has Results:** True |
## Protocol Section
### Identification Module
**NCT ID:** NCT05832879
**Brief Title:** Telehealth Treatment for Opioid Use Disorders
**Official Title:** A Pilot Study of Telehealth Treatment for Opioid Use Disorders
#### Organization Study ID Info
**ID:** 2000034414
#### Organization
**Class:** OTHER
**Full Name:** Yale University
#### Secondary ID Infos
**ID:** 1R61DA057675-01
**Link:** https://reporter.nih.gov/quickSearch/1R61DA057675-01
**Type:** NIH
### Status Module
#### Completion Date
**Date:** 2024-09
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-04-25
**Type:** ACTUAL
**Last Update Submit Date:** 2024-04-23
**Overall Status:** RECRUITING
#### Primary Completion Date
**Date:** 2024-08
**Type:** ESTIMATED
#### Start Date
**Date:** 2024-05
**Type:** ESTIMATED
**Status Verified Date:** 2024-04
#### Study First Post Date
**Date:** 2023-04-27
**Type:** ACTUAL
**Study First Submit Date:** 2023-04-14
**Study First Submit QC Date:** 2023-04-14
### Sponsor Collaborators Module
#### Collaborators
**Class:** NIH
**Name:** National Institute on Drug Abuse (NIDA)
#### Lead Sponsor
**Class:** OTHER
**Name:** Yale University
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** This study aims to use an Opioid Use Disorder (OUD) Telehealth Platform to reduce overdose events. This telehealth platform will be pilot tested to evaluate its preliminary efficacy in terms of motivating engagement in medications for OUD (MOUD), as well as its feasibility, acceptability and satisfaction to both first responders/providers and participants.
**Detailed Description:** The primary objective of the study is to assess effects of enrollment in a comprehensive telehealth platform, in adults with moderate or severe opioid use disorder with a history of at least one opioid overdose. Thirty adults with moderate or severe opioid use disorder with a history of at least one opioid overdose will be enrolled and the primary endpoint will be attendance at first appointment for medication for opioid use disorder at 30 days.
Secondary aims include assessing engagement in the first MOUD appointment at 90 days and self-report of the number of subsequent overdose events at 30 and 90 days. Exploratory aims include feasibility of intervention, readiness and intention to engage in treatment, acceptability and satisfaction of intervention, and comparison of MOUD engagement with data from previous in-person studies.
### Conditions Module
**Conditions:**
- Opioid Use Disorder
**Keywords:**
- Harm Reduction
- Medication for Opioid Use Disorder
### Design Module
#### Design Info
**Allocation:** NA
**Intervention Model:** SINGLE_GROUP
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 30
**Type:** ESTIMATED
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Participants will be assigned to receive the OUD Telehealth Platform, which will be delivered remotely by research staff.
**Intervention Names:**
- Behavioral: OUD Telehealth Platform
**Label:** OUD Telehealth Platform
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- OUD Telehealth Platform
**Description:** The platform is intended to encourage engagement in treatment with medications for opioid use disorder (MOUD) through a chat dialogue with users
**Name:** OUD Telehealth Platform
**Type:** BEHAVIORAL
### Outcomes Module
#### Primary Outcomes
**Description:** Overall attendance will be measured by attendance (yes or no) at the first buprenorphine appointment within 30 days of referral.
**Measure:** Attendance for MOUD
**Time Frame:** Up to 30 Days
#### Secondary Outcomes
**Description:** Engagement will be measured by demonstrated maintained attendance with MOUD treatment within 90 days of referral.
**Measure:** Engagement with MOUD
**Time Frame:** Up to 90 days
**Description:** A self-report of the number of subsequent overdose events at 30 days.
**Measure:** Overdose Events at 30 Days
**Time Frame:** Up to 30 days
**Description:** A self-report of the number of subsequent overdose events at 90 days.
**Measure:** Overdose Events at 90 Days
**Time Frame:** Up to 90 days
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Can speak, read and write in English
* Provision of signed and dated informed consent form
* Have a history of at least one opioid overdose
* Screened positive for OUD, moderate or severe, based on the Diagnostic and Statistical Manual-5th Edition
* Have a working cell phone number and working device that can access a web browser and receive texts (i.e., smartphone, tablet or computer) OR be willing to use a device provided by the study team
* Can provide a working email address OR be willing to create one
Exclusion Criteria:
* Current use of buprenorphine, methadone, or naltrexone for a substance use disorder
* Pregnancy or lactation
* Known current suicide risk based on participant self-report
* On parole or incarcerated at time of enrollment based on participant self-report
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Rebekah Heckmann, MD, MPH, MPA
**Phone:** (203) 737-8335
**Role:** CONTACT
**Contact 2:**
**Email:** [email protected]
**Name:** Michael Pantalon, PhD
**Phone:** (203) 785-4363
**Role:** CONTACT
#### Locations
**Location 1:**
**City:** New Haven
**Country:** United States
**Facility:** Yale School of Medicine
**State:** Connecticut
**Status:** RECRUITING
**Zip:** 06520
#### Overall Officials
**Official 1:**
**Affiliation:** Yale School of Medicine
**Name:** Rebekah Heckmann, MD, MPH, MPA
**Role:** PRINCIPAL_INVESTIGATOR
### IPD Sharing Statement Module
**Description:** Data will be shared as part of the HEAL Initiative Consortium.
**IPD Sharing:** YES
**Time Frame:** Following the close of the study and final reporting to funder.
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000079524
- Term: Narcotic-Related Disorders
- ID: D000064419
- Term: Chemically-Induced Disorders
- ID: D000001523
- Term: Mental Disorders
### Condition Browse Module - Browse Branches
- Abbrev: BC25
- Name: Substance Related Disorders
- Abbrev: BXM
- Name: Behaviors and Mental Disorders
- Abbrev: All
- Name: All Conditions
### Condition Browse Module - Browse Leaves
- ID: M12244
- Name: Opioid-Related Disorders
- Relevance: HIGH
- As Found: Opioid Use Disorder
- ID: M21837
- Name: Substance-Related Disorders
- Relevance: HIGH
- As Found: Opioid Use Disorder
- ID: M2057
- Name: Narcotic-Related Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M30302
- Name: Chemically-Induced Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M4815
- Name: Mental Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M14473
- Name: Psychotic Disorders
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000009293
- Term: Opioid-Related Disorders
- ID: D000019966
- Term: Substance-Related Disorders
### Intervention Browse Module - Browse Branches
- Abbrev: Analg
- Name: Analgesics
- Abbrev: CNSDep
- Name: Central Nervous System Depressants
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M4033
- Name: Analgesics, Opioid
- Relevance: LOW
- As Found: Unknown
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT03438279
**Brief Title:** Survival Study for Participants Treated With Ipilimumab-Nivolumab Combination Therapy
**Official Title:** Projecting Survival for Patients Treated With First-Line Ipilimumab-Nivolumab Combination Therapy Using a Prognostic Model and Cost Per Responder Model of Ipilimumab-Nivolumab Combination Therapy
#### Organization Study ID Info
**ID:** CA209-437
#### Organization
**Class:** INDUSTRY
**Full Name:** Bristol-Myers Squibb
### Status Module
#### Completion Date
**Date:** 2017-12-28
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2018-02-19
**Type:** ACTUAL
**Last Update Submit Date:** 2018-02-16
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2017-12-28
**Type:** ACTUAL
#### Start Date
**Date:** 2015-06-23
**Type:** ACTUAL
**Status Verified Date:** 2018-02
#### Study First Post Date
**Date:** 2018-02-19
**Type:** ACTUAL
**Study First Submit Date:** 2018-01-31
**Study First Submit QC Date:** 2018-02-16
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Bristol-Myers Squibb
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
### Description Module
**Brief Summary:** This study will review published trial literature and documents for Overall Survival (OS) to evaluate the association between the hazard of death and each baseline variable.
### Conditions Module
**Conditions:**
- Melanoma
### Design Module
#### Design Info
**Observational Model:** COHORT
**Time Perspective:** RETROSPECTIVE
#### Enrollment Info
**Count:** 600
**Type:** ACTUAL
**Study Type:** OBSERVATIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** patients who received ipilimumab as their first-line treatment
**Intervention Names:**
- Other: Non-Interventional
**Label:** First-Line Ipilimumab
### Interventions
#### Intervention 1
**Arm Group Labels:**
- First-Line Ipilimumab
**Description:** Non-Interventional
**Name:** Non-Interventional
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Description:** The OS prognostic model uses Individual patient data from a pooled analysis of 12 ipilimumab studies.
**Measure:** Prognostic Model of Overall Survival (OS)
**Time Frame:** 10 years
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Diagnosis of melanoma
Exclusion Criteria:
* Age less than 18 years old
**Minimum Age:** 18 Years
**Sampling Method:** NON_PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
**Study Population:** Individual patient data (IPD) from the ipilimumab pooled analysis of clinical trials and observational studies will be used to construct the prognostic model for OS in advanced melanoma patients.
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Boston
**Country:** United States
**Facility:** Local Institution
**State:** Massachusetts
**Zip:** 02199
#### Overall Officials
**Official 1:**
**Affiliation:** Bristol-Myers Squibb
**Name:** Bristol-Myers Squibb
**Role:** STUDY_DIRECTOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000018358
- Term: Neuroendocrine Tumors
- ID: D000017599
- Term: Neuroectodermal Tumors
- ID: D000009373
- Term: Neoplasms, Germ Cell and Embryonal
- ID: D000009370
- Term: Neoplasms by Histologic Type
- ID: D000009369
- Term: Neoplasms
- ID: D000009380
- Term: Neoplasms, Nerve Tissue
- ID: D000018326
- Term: Nevi and Melanomas
- ID: D000012878
- Term: Skin Neoplasms
- ID: D000009371
- Term: Neoplasms by Site
- ID: D000012871
- Term: Skin Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: BC17
- Name: Skin and Connective Tissue Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M11528
- Name: Melanoma
- Relevance: HIGH
- As Found: Melanoma
- ID: M20495
- Name: Neuroendocrine Tumors
- Relevance: LOW
- As Found: Unknown
- ID: M19845
- Name: Neuroectodermal Tumors
- Relevance: LOW
- As Found: Unknown
- ID: M20388
- Name: Neuroectodermal Tumors, Primitive
- Relevance: LOW
- As Found: Unknown
- ID: M12318
- Name: Neoplasms, Germ Cell and Embryonal
- Relevance: LOW
- As Found: Unknown
- ID: M12315
- Name: Neoplasms by Histologic Type
- Relevance: LOW
- As Found: Unknown
- ID: M12325
- Name: Neoplasms, Nerve Tissue
- Relevance: LOW
- As Found: Unknown
- ID: M12448
- Name: Nevus, Pigmented
- Relevance: LOW
- As Found: Unknown
- ID: M12446
- Name: Nevus
- Relevance: LOW
- As Found: Unknown
- ID: M20470
- Name: Nevi and Melanomas
- Relevance: LOW
- As Found: Unknown
- ID: M15681
- Name: Skin Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M15674
- Name: Skin Diseases
- Relevance: LOW
- As Found: Unknown
- ID: T4091
- Name: Neuroendocrine Tumor
- Relevance: LOW
- As Found: Unknown
- ID: T4092
- Name: Neuroepithelioma
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000008545
- Term: Melanoma
### Intervention Browse Module - Browse Branches
- Abbrev: ANeo
- Name: Antineoplastic Agents
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M1854
- Name: Nivolumab
- Relevance: LOW
- As Found: Unknown
- ID: M1348
- Name: Ipilimumab
- Relevance: LOW
- As Found: Unknown
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT04255979
**Brief Title:** A Study of HY209 in Healthy Male Volunteers for Sepsis
**Official Title:** A Randomized, Double-blind, Placebo-controlled Single Dosing, Dose Escalation Phase I Clinical Trial to Investigate the Safety/Tolerability and Pharmacokinetics of HY209 After Intravenous Administration in Healthy Male Volunteers
#### Organization Study ID Info
**ID:** HY209-IV
#### Organization
**Class:** INDUSTRY
**Full Name:** Shaperon
### Status Module
#### Completion Date
**Date:** 2020-06-12
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2020-11-30
**Type:** ACTUAL
**Last Update Submit Date:** 2020-11-27
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2020-06-12
**Type:** ACTUAL
#### Start Date
**Date:** 2019-12-05
**Type:** ACTUAL
**Status Verified Date:** 2020-11
#### Study First Post Date
**Date:** 2020-02-05
**Type:** ACTUAL
**Study First Submit Date:** 2020-02-03
**Study First Submit QC Date:** 2020-02-03
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Shaperon
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** A randomized, double-blind, placebo-controlled single dosing, dose escalation phase I clinical trial to investigate the safety/tolerability and pharmacokinetics of HY209 after intravenous administration in healthy male volunteers
**Detailed Description:** HY209, which is being developed for the treatment of sepsis, inhibits inflammation by promoting the differentiation and division of Myeloid-derived suppressor cells (MDSCs).
### Conditions Module
**Conditions:**
- Sepsis
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
**Intervention Model Description:** Randomized, double-blind, placebo-controlled single dosing, dose escalation
##### Masking Info
**Masking:** DOUBLE
**Who Masked:**
- PARTICIPANT
- INVESTIGATOR
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 40
**Type:** ACTUAL
**Phases:**
- PHASE1
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Single dose of HY209 0.1 mg/kg or placebo.
**Intervention Names:**
- Drug: HY209
**Label:** Cohort 1
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Single dose of HY209 0.2 mg/kg or placebo.
**Intervention Names:**
- Drug: HY209
**Label:** Cohort 2
**Type:** EXPERIMENTAL
#### Arm Group 3
**Description:** Single dose of HY209 0.4 mg/kg or placebo.
**Intervention Names:**
- Drug: HY209
**Label:** Cohort 3
**Type:** EXPERIMENTAL
#### Arm Group 4
**Description:** Single dose of HY209 0.8 mg/kg or placebo.
**Intervention Names:**
- Drug: HY209
**Label:** Cohort 4
**Type:** EXPERIMENTAL
#### Arm Group 5
**Description:** Single dose of HY209 1.6 mg/kg or placebo.
**Intervention Names:**
- Drug: HY209
**Label:** Cohort 5
**Type:** EXPERIMENTAL
#### Arm Group 6
**Description:** Single dose of HY209 3.2 mg/kg or placebo.
**Intervention Names:**
- Drug: HY209
**Label:** Cohort 6
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Cohort 1
- Cohort 2
- Cohort 3
- Cohort 4
- Cohort 5
- Cohort 6
**Description:** 6 Subjects will be assigned to drug (HY209) and 2 subjects will be assigned to placebo.
**Name:** HY209
**Other Names:**
- HY209-IV
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Description:** Number of subjects with abnormal laboratory values and/or adverse events that are related to treatment
**Measure:** Incidence of treatment emergent adverse events (TEAEs)
**Time Frame:** Up to Day 6
#### Secondary Outcomes
**Description:** Maximum concentration of HY209 in plasma
**Measure:** Maximum concentration (Cmax) of HY209
**Time Frame:** Up to Day 2
**Description:** Area under the plasma HY209 concentration-time curve over the time interval from 0 extrapolated to infinity
**Measure:** Ratio of area under curve infinity (AUCinf) of HY209
**Time Frame:** Up to Day 2
**Description:** Area under the plasma HY209 concentration-time curve over the time interval from 0 to the last quantifiable plasma concentration
**Measure:** Ratio of area under curve last (AUClast) of HY209
**Time Frame:** Up to Day 2
**Description:** Time of maximum concentration of HY209 in plasma
**Measure:** Time of maximum concentration (Tmax) of HY209
**Time Frame:** Up to Day 2
**Description:** Terminal half-life of HY209 in plasma
**Measure:** Terminal halif-life (t1/2) of HY209
**Time Frame:** Up to Day 2
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Healthy male aged from 19 to 45 at screening test
* BMI 18 kg/m2 \~ 27 kg/m2 at screening test
* Subjects found to be clinically healthy by medical history, physical examination, vital signs, electrocardiogram (ECG), and appropriate laboratory tests
* Those who must be capable of giving informed consent and willing to comply with all clinic visits and study-related procedures for the duration of study until study completion
Exclusion Criteria:
* Those who have a history of hypersensitivity or clinically significant hypersensitivity reactions to drugs (containing Taurodeoxycholate component, aspirin, antibiotics, etc.)
* Those who showed clinical symptoms suspected of acute infectious disease within 2 weeks before the first does, or whose body temperature (ear canal) measured at screening showed 38 ℃ or higher
* Those who have a clinically significant disease of liver, kidney, digestive, respiratory, endocrine, neurologic, blood/tumor, cardiovascular system history of those diseases
* Those who have a history of gastrointestinal diseases or surgery that may affect the absorption of the investigational drug
* Those who have a history of substance abuse or have tested positive for drugs of concern for misuse by urine drug screening
* Patients with the following blood pressure measured at the seat after resting for more than 5 minutes at the screening visit \[Systolic blood pressure (SBP): \< 90 mmHg or \> 150 mmHg, Diastolic blood pressure (DBP): \< 50 mmHg or \> 90 mmHg\]
* Those who participated in other clinical trials or bioequivalence within 6 months prior to the first dosing and received the drug
* Those who have donated whole blood within 2 months before the first dose or ingredient donation within 1 month or received blood transfusion within 1 month
* Those who have taken metabolic enzyme-induced and inhibitory drugs within 1 month before screening
* Those who consumed grapefruit / caffeine-containing foods within 3 days of the first dose and who cannot refrain from eating grapefruit-containing foods from 3 days before admission to discharge date
* Those who have taken specialty or herbal medicines within 2 weeks of the first dose or who have taken over-the-counter (OTC) within 1 week
* Caffeine overdose, alcohol overdose or oversmoker
* Those who have unusual eating habits or who are unable to eat the meals provided in this clinical trial
* Other investigator judged to be unsuitable as clinical subject
**Healthy Volunteers:** True
**Maximum Age:** 45 Years
**Minimum Age:** 19 Years
**Sex:** MALE
**Standard Ages:**
- ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Seoul
**Country:** Korea, Republic of
**Facility:** Seoul National University Hospital
**State:** Jongno-gu
**Zip:** 03080
#### Overall Officials
**Official 1:**
**Affiliation:** Seoul National University Hospital
**Name:** In-jin Jang
**Role:** PRINCIPAL_INVESTIGATOR
### IPD Sharing Statement Module
**IPD Sharing:** NO
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000007239
- Term: Infections
- ID: D000018746
- Term: Systemic Inflammatory Response Syndrome
- ID: D000007249
- Term: Inflammation
- ID: D000010335
- Term: Pathologic Processes
### Condition Browse Module - Browse Branches
- Abbrev: BC01
- Name: Infections
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: All
- Name: All Conditions
### Condition Browse Module - Browse Leaves
- ID: M20864
- Name: Sepsis
- Relevance: HIGH
- As Found: Sepsis
- ID: M16869
- Name: Toxemia
- Relevance: LOW
- As Found: Unknown
- ID: M10283
- Name: Infections
- Relevance: LOW
- As Found: Unknown
- ID: M6368
- Name: Communicable Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M16355
- Name: Syndrome
- Relevance: LOW
- As Found: Unknown
- ID: M20818
- Name: Systemic Inflammatory Response Syndrome
- Relevance: LOW
- As Found: Unknown
- ID: M10293
- Name: Inflammation
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000018805
- Term: Sepsis
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT00262379
**Brief Title:** Use or Non-use of Epoetin Beta in Patients Infected by Chronic Hepatitis C
**Official Title:** Multicenter Study, Randomized and Pragmatic, Comparing Two Therapeutic Strategies : Use or Non-use of Epoetin Beta in Patients Infected by Chronic Hepatitis C and Treated by Combination Therapy Peginterferon Alfa-2a Plus Ribavirin
#### Organization Study ID Info
**ID:** CP 2005-01
#### Organization
**Class:** OTHER_GOV
**Full Name:** University Hospital, Angers
### Status Module
#### Completion Date
**Date:** 2009-06
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2014-12-12
**Type:** ESTIMATED
**Last Update Submit Date:** 2014-12-11
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2009-06
**Type:** ACTUAL
#### Start Date
**Date:** 2005-12
**Status Verified Date:** 2014-12
#### Study First Post Date
**Date:** 2005-12-06
**Type:** ESTIMATED
**Study First Submit Date:** 2005-12-05
**Study First Submit QC Date:** 2005-12-05
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER_GOV
**Name:** University Hospital, Angers
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** The purpose is to demonstrate a correction of anemia in hepatitis C virus treatment with peginterferon plus ribavirin.
**Detailed Description:** Show that the correction of anemia by epoetin beta (NeoRecormon®) able to maintain a optimal dose of ribavirin (Copegus®). The study compares two therapeutic strategies : use or non-use of epoetin beta (NeoRecormon®) in patients infected by chronic hepatitis C and treated by combination therapy Peginterferon alfa-2a (Pegasys®) plus ribavirin (Copegus®). The main judgement criteria are :
* Sustained Viral Response (Week 72)
* Viral Response at the End of Treatment (Week 48)
* Quality of life
* Cumulative dose of ribavirin from D0-W24 and from W24-W48 periods
* Clinical and biological tolerance
### Conditions Module
**Conditions:**
- Chronic Hepatitis C
**Keywords:**
- Chronic hepatitis C
- peginterferon
- ribavirin
- epoetin beta
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 229
**Type:** ACTUAL
**Phases:**
- PHASE3
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** HCV treatment with peginterferon plus ribavirin during 48 weeks
**Label:** Group A
**Type:** NO_INTERVENTION
#### Arm Group 2
**Description:** HCV treatment with peginterferon plus ribavirin during 48 weeks plus epoetin beta under anemia conditions
**Intervention Names:**
- Drug: epoetin beta (NeoRecormon®)
**Label:** Groupb
**Type:** ACTIVE_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Groupb
**Description:** • Prescription of the epoetin beta : when blood concentration of hemoglobin is lower or equal to 12 g/dL in male or lower or equal to 11 g/dL in female
**Name:** epoetin beta (NeoRecormon®)
**Other Names:**
- NeoRecormon®
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Description:** Actual Plasma Hepatitis C Virus (HCV) Ribonucleic Acid (RNA) Values up to Week 24 in the Post-treatment Follow-up Period
**Measure:** Sustained Viral Response (Week 72)
**Time Frame:** Week 72
#### Secondary Outcomes
**Description:** Actual Plasma Hepatitis C Virus (HCV) Ribonucleic Acid (RNA) Values up to Week 48
**Measure:** • Viral Response at the End of Treatment (Week 48)
**Time Frame:** Week 48
**Description:** * Questionnaire HQLQ
* Fatigue Severity Scale HQLQ questionnaire and Fatigue Severity Scale
**Measure:** • Quality of life
**Time Frame:** D0, W4, W12, W24, W48, W72
**Description:** • Cumulating dose of ribavirin during following periods D0-W24 and W24-W48
**Measure:** • Cumulative dose of ribavirin from D0-W24 and from W24-W48 periods
**Time Frame:** D0, W4, W8, W12, W16, W20, W24, W28, W32, W36, W40, W44, W48
**Description:** Up to 72 weeks, includes all serious and other adverse events that newly occurred or worsened after treatment with PegIFNα-2a, ribavirin or epoetin beta
**Measure:** • Clinical and biological tolerance
**Time Frame:** D0, W4, W8, W12, W16, W20, W24, W28, W32, W36, W40, W44, W48, W52, W60, W72
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Male or female patients 18 years old or above
* Patient with French social security or other equivalent health assurance
* Patient with informed consent
* Serologic evidence of chronic hepatitis C by detectable anti-HCV antibodies
* Patient infected by HCV genotype 1, 4, 5 or 6
* Compensated liver disease (Child-Pugh ≤ 6)
* Negative urine or blood pregnancy test (for women of childbearing potential) documented within the 24-hour period prior the enrollment in the study
* All fertile males and females receiving ribavirin must have effective contraception during treatment and during the 6 following months
* Patient naive of treatment with installation of treatment by investigator following criteria for use Peginterferon alfa-2a (Pegasys) and ribavirin (Copegus)
Exclusion Criteria:
* Women with ongoing pregnancy or breast feeding
* Male partner of pregnancy woman
* Minor
* Major protected by French law for biomedical study
* Co-infection by HBV or HIV
* History or other evidence of decompensated liver disease or Child-Pugh score \> 6
* Clinical or radiological evidence (abdominal ultrasound, CT scan or MRI) of hepatocellular carcinoma
* IFN or ribavirin at any previous time
* Patient who received an erythropoetin within 2 months before inclusion
* History of epilepsy (during the last 6 months)
* Chronic cardiac insufficiency (stage III or IV in classification from the New York Heart Association \[NYHA\])
* Not controlled portal hypertension
* Antecedents or risk of venous thrombosis
* Surgery within 3 months before inclusion
* Serum creatinine level \>15 mg/mL (130µmol/L)
* Deficiency in vitamin B9 and/or B12 (suspected with macrocytosis \> 105 µm3)
* Thrombocytosis (platelets \> 500 000/mm3)
* Chronic inflammatory syndrome (CRP \> 10 mg/L)
* Deficiency not corrected in iron :
* Ferritin blood level \< 10 µg/L Or - Transferrin saturation coefficient \< 20 %
* History of neoplasia (except basocellular epithelioma and cervical cancer)
* Contraindications to use epoetin beta or an excipient from molecule to study (urea, polysorbate 20, glycine, leucine, isoleucine, threonine, glutamic acid, phenylalanine, benzoic acid, benzyl alcohol)
* Absence of written informed consent
* Exclusion time for another biomedical study
* Patient with blood concentration of hemoglobin lower or equal to 12 g/dL for male or lower or equal to 11 g/dL for female
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Aix en Provence
**Country:** France
**Facility:** H Aix en Provence
**Zip:** 13616
**Location 2:**
**City:** Angers
**Country:** France
**Facility:** UH Angers
**Zip:** 49933
**Location 3:**
**City:** Avignon
**Country:** France
**Facility:** H Avignon
**Zip:** 84 902
**Location 4:**
**City:** Bourgoin-Jallieu
**Country:** France
**Facility:** H Bourgoin-Jallieu
**Zip:** 38 317
**Location 5:**
**City:** Brest
**Country:** France
**Facility:** UH Brest
**Zip:** 29 609
**Location 6:**
**City:** Caen
**Country:** France
**Facility:** UH Caen
**Zip:** 14 033
**Location 7:**
**City:** Châteauroux
**Country:** France
**Facility:** H Châteauroux
**Zip:** 36 000
**Location 8:**
**City:** Clermont Ferrand
**Country:** France
**Facility:** UH Clermont Ferrand
**Zip:** 63009
**Location 9:**
**City:** Corbeil-Essonnes
**Country:** France
**Facility:** H Corbeil-Essonnes
**Zip:** 91 106
**Location 10:**
**City:** Creil
**Country:** France
**Facility:** H Creil
**Zip:** 60 100
**Location 11:**
**City:** Créteil
**Country:** France
**Facility:** H Créteil
**Zip:** 94010
**Location 12:**
**City:** Dijon
**Country:** France
**Facility:** UH Dijon
**Zip:** 21 079
**Location 13:**
**City:** Dreux
**Country:** France
**Facility:** H Dreux
**Zip:** 28100
**Location 14:**
**City:** Freyming-Merlebach
**Country:** France
**Facility:** H Freyming-Merlebach
**Zip:** 57 804
**Location 15:**
**City:** Grasse
**Country:** France
**Facility:** H Grasse
**Zip:** 06 130
**Location 16:**
**City:** Grenoble
**Country:** France
**Facility:** UH Grenoble
**Zip:** 38 043
**Location 17:**
**City:** La Roche sur Yon
**Country:** France
**Facility:** H La Roche sur Yon
**Zip:** 85925
**Location 18:**
**City:** Le Mans
**Country:** France
**Facility:** H Le Mans
**Zip:** 72000
**Location 19:**
**City:** Limoges
**Country:** France
**Facility:** UH Limoges
**Zip:** 87042
**Location 20:**
**City:** Lyon
**Country:** France
**Facility:** UH Lyon
**Zip:** 69 288
**Location 21:**
**City:** Marseille
**Country:** France
**Facility:** H Saint-Joseph
**Zip:** 13 285
**Location 22:**
**City:** Montauban
**Country:** France
**Facility:** H Montauban
**Zip:** 82 013
**Location 23:**
**City:** Montpellier
**Country:** France
**Facility:** UH Montpellier
**Zip:** 34 295
**Location 24:**
**City:** Montélimar
**Country:** France
**Facility:** H montélimar
**Zip:** 26 200
**Location 25:**
**City:** Nantes
**Country:** France
**Facility:** UH Nantes
**Zip:** 44 800
**Location 26:**
**City:** Orléans
**Country:** France
**Facility:** H Orléans
**Zip:** 45100
**Location 27:**
**City:** Paris
**Country:** France
**Facility:** H Tenon
**Zip:** 75 020
**Location 28:**
**City:** Paris
**Country:** France
**Facility:** H saint-Antoine
**Zip:** 75 571
**Location 29:**
**City:** Pau
**Country:** France
**Facility:** H Pau
**Zip:** 64 011
**Location 30:**
**City:** Poitiers
**Country:** France
**Facility:** UH Poitiers
**Zip:** 86 020
**Location 31:**
**City:** Rennes
**Country:** France
**Facility:** UH Rennes
**Zip:** 35 043
**Location 32:**
**City:** Rouen
**Country:** France
**Facility:** UH Rouen
**Zip:** 76 031
**Location 33:**
**City:** Saint Laurent du Var
**Country:** France
**Facility:** Arnault Tzanck Institute
**Zip:** 06721
**Location 34:**
**City:** Saint-Dizier
**Country:** France
**Facility:** H Saint-Dizier
**Zip:** 52 115
**Location 35:**
**City:** Toulouse
**Country:** France
**Facility:** UH Toulouse
**Zip:** 31 059
**Location 36:**
**City:** Tourcoing
**Country:** France
**Facility:** H Tourcoing
**Zip:** 59 208
**Location 37:**
**City:** Tours
**Country:** France
**Facility:** UH Tours
**Zip:** 37 170
#### Overall Officials
**Official 1:**
**Affiliation:** UH Angers
**Name:** Françoise Lunel-Fabiani, MD, PhD
**Role:** PRINCIPAL_INVESTIGATOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000008107
- Term: Liver Diseases
- ID: D000004066
- Term: Digestive System Diseases
- ID: D000006525
- Term: Hepatitis, Viral, Human
- ID: D000014777
- Term: Virus Diseases
- ID: D000007239
- Term: Infections
- ID: D000004769
- Term: Enterovirus Infections
- ID: D000010850
- Term: Picornaviridae Infections
- ID: D000012327
- Term: RNA Virus Infections
- ID: D000086982
- Term: Blood-Borne Infections
- ID: D000003141
- Term: Communicable Diseases
- ID: D000018178
- Term: Flaviviridae Infections
- ID: D000002908
- Term: Chronic Disease
- ID: D000020969
- Term: Disease Attributes
- ID: D000010335
- Term: Pathologic Processes
### Condition Browse Module - Browse Branches
- Abbrev: BC01
- Name: Infections
- Abbrev: BC06
- Name: Digestive System Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M9592
- Name: Hepatitis A
- Relevance: HIGH
- As Found: Hepatitis
- ID: M9591
- Name: Hepatitis
- Relevance: HIGH
- As Found: Hepatitis
- ID: M9607
- Name: Hepatitis, Chronic
- Relevance: HIGH
- As Found: Chronic Hepatitis
- ID: M9611
- Name: Hepatitis C
- Relevance: HIGH
- As Found: Hepatitis C
- ID: M21613
- Name: Hepatitis C, Chronic
- Relevance: HIGH
- As Found: Chronic Hepatitis C
- ID: M11107
- Name: Liver Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M7255
- Name: Digestive System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M8883
- Name: Gastrointestinal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M9610
- Name: Hepatitis, Viral, Human
- Relevance: LOW
- As Found: Unknown
- ID: M17522
- Name: Virus Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M10283
- Name: Infections
- Relevance: LOW
- As Found: Unknown
- ID: M6368
- Name: Communicable Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M7930
- Name: Enterovirus Infections
- Relevance: LOW
- As Found: Unknown
- ID: M13745
- Name: Picornaviridae Infections
- Relevance: LOW
- As Found: Unknown
- ID: M15149
- Name: RNA Virus Infections
- Relevance: LOW
- As Found: Unknown
- ID: M2593
- Name: Blood-Borne Infections
- Relevance: LOW
- As Found: Unknown
- ID: M20324
- Name: Flaviviridae Infections
- Relevance: LOW
- As Found: Unknown
- ID: M6147
- Name: Chronic Disease
- Relevance: LOW
- As Found: Unknown
- ID: M22700
- Name: Disease Attributes
- Relevance: LOW
- As Found: Unknown
- ID: T1303
- Name: Chronic Graft Versus Host Disease
- Relevance: HIGH
- As Found: Chronic
### Condition Browse Module - Meshes
- ID: D000006506
- Term: Hepatitis A
- ID: D000006526
- Term: Hepatitis C
- ID: D000019698
- Term: Hepatitis C, Chronic
- ID: D000006505
- Term: Hepatitis
- ID: D000006521
- Term: Hepatitis, Chronic
### Intervention Browse Module - Ancestors
- ID: D000006397
- Term: Hematinics
### Intervention Browse Module - Browse Branches
- Abbrev: Infl
- Name: Anti-Inflammatory Agents
- Abbrev: ARhu
- Name: Antirheumatic Agents
- Abbrev: Analg
- Name: Analgesics
- Abbrev: All
- Name: All Drugs and Chemicals
- Abbrev: Hemat
- Name: Hematinics
- Abbrev: Infe
- Name: Anti-Infective Agents
### Intervention Browse Module - Browse Leaves
- ID: M207501
- Name: Chrysarobin
- Relevance: LOW
- As Found: Unknown
- ID: M314
- Name: Epoetin Alfa
- Relevance: HIGH
- As Found: Distal
- ID: M15083
- Name: Ribavirin
- Relevance: LOW
- As Found: Unknown
- ID: M247369
- Name: Peginterferon alfa-2a
- Relevance: LOW
- As Found: Unknown
- ID: M9485
- Name: Hematinics
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: D000068817
- Term: Epoetin Alfa
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT00609479
**Brief Title:** Comparison of Two Methods for Treatment of Colles´s Fracture
**Official Title:** Comparison of External and Internal Fixationmethods for Distal Radius Fracture - a Randomized Study.
#### Organization Study ID Info
**ID:** JOS-1
#### Organization
**Class:** OTHER
**Full Name:** University of Aarhus
#### Secondary ID Infos
**ID:** JOS-1
### Status Module
#### Completion Date
**Date:** 2010-02
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2010-04-12
**Type:** ESTIMATED
**Last Update Submit Date:** 2010-04-09
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2010-02
**Type:** ACTUAL
#### Start Date
**Date:** 2008-01
**Status Verified Date:** 2010-04
#### Study First Post Date
**Date:** 2008-02-07
**Type:** ESTIMATED
**Study First Submit Date:** 2008-01-24
**Study First Submit QC Date:** 2008-02-06
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** University of Aarhus
#### Responsible Party
**Old Name Title:** MD/PhD-stud. Jesper Ougaard Schønnemann
**Old Organization:** Orthopedic Research Unit, Regionhospital Holstebro
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** The purpose of this study is to compare two different fixationmethods for fractures of the wrist(distal radius fractures). We are comparing an external fixation (Hoffmann-II-non-bridging) vs. an internal fixation(Micronail). Primary endpoint is patient satisfaction as scored by the DASH-questionnaire.Secondary followup is X-rays, strength, PRWE-questionnaire.
### Conditions Module
**Conditions:**
- Distal Radius Fracture
- Colle´s Fracture
**Keywords:**
- Distal radius fracture
- Colle´s fracture
- Internal fixation
- External fixation
- DASH
- PRWE
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 60
**Type:** ESTIMATED
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Internal fixation with Micronail. 41 patients.
**Intervention Names:**
- Procedure: Osteosynthesis
**Label:** 1
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** External fixation with Hoffmann-II-non-bridging. 41 patients.
**Intervention Names:**
- Procedure: Osteosynthesis
**Label:** 2
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- 1
- 2
**Description:** 1. Micronail osteosynthesis. A cast for the first 14 days, thereafter starting physiotherapy. Control at weeks 1,2,5,12.
2. Hoffmann-II-non-bridging osteosynthesis. Removal of device after 5 weeks. physiotherapy starts at 14 days. Control at weeks 1,2,5,12.
**Name:** Osteosynthesis
**Type:** PROCEDURE
### Outcomes Module
#### Primary Outcomes
**Measure:** DASH-questionnaire
**Time Frame:** 3 months
#### Secondary Outcomes
**Measure:** PRWE-questionnaire
**Time Frame:** 3 months
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Fracture Older type 2.
* Fracture Older type 3.
Exclusion Criteria:
* Fractures older than 3 weeks.
* Pregnancy.
* Seq. after previous fracture.
* Distal fragments volar cortex \< 10 mm.
* Open fracture larger than Gustillo 1.
* Disability due to other illness.
* Congenital abnormity or injury/disease in the affected extremity.
* Not capable of mentally/physically to cooperate.
* Not able to go to followup on the operating hospital.
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Herning
**Country:** Denmark
**Facility:** Regionhospital Herning
**Zip:** 7400
**Location 2:**
**City:** Holstebro
**Country:** Denmark
**Facility:** Regionhospital Holstebro
**Zip:** 7500
**Location 3:**
**City:** Viborg
**Country:** Denmark
**Facility:** Regionhospital Viborg
**Zip:** 8800
#### Overall Officials
**Official 1:**
**Affiliation:** Department of Orthopedics, University of Aarhus, Denmark
**Name:** Kjeld Søballe, MD; PhD; Prof.
**Role:** STUDY_CHAIR
### References Module
#### See Also Links
**Label:** Related Info
**URL:** http://www.orthoresearch.dk
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000014947
- Term: Wounds and Injuries
- ID: D000005543
- Term: Forearm Injuries
- ID: D000001134
- Term: Arm Injuries
- ID: D000014954
- Term: Wrist Injuries
- ID: D000072039
- Term: Fracture Dislocation
- ID: D000004204
- Term: Joint Dislocations
- ID: D000007592
- Term: Joint Diseases
- ID: D000009140
- Term: Musculoskeletal Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC26
- Name: Wounds and Injuries
- Abbrev: All
- Name: All Conditions
- Abbrev: BC05
- Name: Musculoskeletal Diseases
### Condition Browse Module - Browse Leaves
- ID: M26370
- Name: Fractures, Bone
- Relevance: HIGH
- As Found: Fracture
- ID: M14731
- Name: Radius Fractures
- Relevance: HIGH
- As Found: Radius Fracture
- ID: M6328
- Name: Colles' Fracture
- Relevance: HIGH
- As Found: Colles' Fracture
- ID: M2933
- Name: Wrist Fractures
- Relevance: HIGH
- As Found: Distal Radius Fracture
- ID: M17685
- Name: Wounds and Injuries
- Relevance: LOW
- As Found: Unknown
- ID: M8667
- Name: Forearm Injuries
- Relevance: LOW
- As Found: Unknown
- ID: M4444
- Name: Arm Injuries
- Relevance: LOW
- As Found: Unknown
- ID: M17692
- Name: Wrist Injuries
- Relevance: LOW
- As Found: Unknown
- ID: M909
- Name: Fracture Dislocation
- Relevance: LOW
- As Found: Unknown
- ID: M7385
- Name: Joint Dislocations
- Relevance: LOW
- As Found: Unknown
- ID: M10621
- Name: Joint Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M12097
- Name: Musculoskeletal Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000003100
- Term: Colles' Fracture
- ID: D000050723
- Term: Fractures, Bone
- ID: D000011885
- Term: Radius Fractures
- ID: D000092503
- Term: Wrist Fractures
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT01753479
**Brief Title:** Spectroscopy From Duodenum
**Official Title:** Duodenal Spectroscopy Study for Cancer Diagnosis
#### Organization Study ID Info
**ID:** OMSC-Mag-1
#### Organization
**Class:** INDUSTRY
**Full Name:** Olympus Corporation
### Status Module
#### Completion Date
**Date:** 2016-06-30
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2017-03-07
**Type:** ACTUAL
**Last Update Submit Date:** 2017-03-03
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2016-06-30
**Type:** ACTUAL
#### Start Date
**Date:** 2013-01
**Type:** ACTUAL
**Status Verified Date:** 2017-03
#### Study First Post Date
**Date:** 2012-12-20
**Type:** ESTIMATED
**Study First Submit Date:** 2012-12-17
**Study First Submit QC Date:** 2012-12-17
### Sponsor Collaborators Module
#### Collaborators
**Class:** OTHER
**Name:** M.D. Anderson Cancer Center
**Class:** OTHER
**Name:** Catholic University of the Sacred Heart
**Class:** OTHER
**Name:** Erasme University Hospital
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Olympus Corporation
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** Purpose of this study is to understand the clinical feasibility of duodenal spectroscopy to adenocarcinoma patients.
**Detailed Description:** Pancreatic cancer (PC) is the most lethal of all major cancers with a five year survival rate of 5 %. While stage I and II tumors leads to an improvement in survival, almost all PCs are currently diagnosed at more advanced non-resectable stages since minimally invasive technique which is capable of screening early-stage PC does not exist. Serum CA19-9 is not recommended as a screening technique because of its low sensitivity and specificity. Imaging modalities such as MRI, CT, EUS and ERCP are more accurate but are not appropriate screening tools due to their high cost, discomfort and complications. Therefore, there is a strong demand for a screening tool with high sensitivity and specificity which is highly acceptable for the patient. The investigators would like to look at the spectroscopy technique for pancreatic cancer diagnosis via an upper endoscopy. A definite diagnosis of the patient is made with histology, cytology or imaging diagnosis. Therefore this study can be positioned as a feasibility study.
### Conditions Module
**Conditions:**
- Pancreatic Adenocarcinoma
**Keywords:**
- Pancreatic adenocarcinoma
### Design Module
#### Design Info
**Allocation:** NA
**Intervention Model:** SINGLE_GROUP
##### Masking Info
**Masking:** NONE
**Primary Purpose:** DIAGNOSTIC
#### Enrollment Info
**Count:** 445
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Intervention Names:**
- Other: Spectroscopy device
**Label:** Test subject
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Test subject
**Description:** Spectrum data are collected using spectroscopy device via an instrumental channel of endoscope. Then spectrum data is analyzed. Numerical features (spectral slope and width of spectrum) are used for statistical analysis.
**Name:** Spectroscopy device
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Description:** To clarify that there is the statistically-significant difference between two cohorts.
**Measure:** The spectral data of the normal cohort and UICC stage II pancreatic ductal adenocarcinoma cohort
**Time Frame:** 1 year
#### Secondary Outcomes
**Description:** A receiver operating characteristic (ROC) is evaluated. A cut-off is then chosen from this ROC curve to maximize both sensitivity and specificity.
**Measure:** The sensitivity and specificity to detect UICC stage II pancreatic ductal adenocarcinoma among all participants.
**Time Frame:** 1 year
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Common inclusion criterion
* Age is 18 years or older.
* Informed consent was obtained.
* Inclusion criterion for normal cohort
* An upper GI endoscopy is scheduled to check upper abdominal symptoms.
* No findings of pancreatic disorder as documented by CT or MRI or EUS
* Inclusion criterion for PC suspicious cohort \* A EUS or ERCP is scheduled to suspected pancreatic disorder.
Exclusion Criteria:
* Common exclusion criterion
* Severe cardiac disease
* Severe respiratory disease
* Bleeding disorders
* Pregnancy
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Jacksonville
**Country:** United States
**Facility:** Mayo Clinic Florida
**State:** Florida
**Zip:** 32224
**Location 2:**
**City:** Houston
**Country:** United States
**Facility:** The University of Texas M. D. Anderson Cancer Center
**State:** Texas
**Zip:** 77030
**Location 3:**
**City:** Bruxelles
**Country:** Belgium
**Facility:** Hôpital Erasme
**State:** Brussels-Capital Region
**Zip:** 1070
**Location 4:**
**City:** Rome
**Country:** Italy
**Facility:** Università Cattolica del Sacro Cuore
**State:** Lazio
**Zip:** 00168
#### Overall Officials
**Official 1:**
**Affiliation:** Mayo Clinic
**Name:** Michael B Wallace, M.D.
**Role:** PRINCIPAL_INVESTIGATOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000002277
- Term: Carcinoma
- ID: D000009375
- Term: Neoplasms, Glandular and Epithelial
- ID: D000009370
- Term: Neoplasms by Histologic Type
- ID: D000009369
- Term: Neoplasms
### Condition Browse Module - Browse Branches
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: All
- Name: All Conditions
### Condition Browse Module - Browse Leaves
- ID: M3585
- Name: Adenocarcinoma
- Relevance: HIGH
- As Found: Adenocarcinoma
- ID: M5534
- Name: Carcinoma
- Relevance: LOW
- As Found: Unknown
- ID: M12320
- Name: Neoplasms, Glandular and Epithelial
- Relevance: LOW
- As Found: Unknown
- ID: M12315
- Name: Neoplasms by Histologic Type
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000000230
- Term: Adenocarcinoma
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT01909479
**Brief Title:** A Phase 3, Multicenter Study To Evaluate The Efficacy And Safety Of MOD-4023 In Adults With Growth Hormone Deficiency
**Official Title:** A Phase 3, Multicenter Study Designed To Evaluate The Efficacy And Safety Of A Long Acting hGH Product (MOD-4023) In Adult Subjects With Growth Hormone Deficiency
#### Organization Study ID Info
**ID:** CP-4-005
#### Organization
**Class:** INDUSTRY
**Full Name:** OPKO Health, Inc.
#### Secondary ID Infos
**ID:** 2013-000830-37
**Type:** EUDRACT_NUMBER
### Status Module
#### Completion Date
**Date:** 2018-08
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2022-08-12
**Type:** ACTUAL
**Last Update Submit Date:** 2022-08-11
**Overall Status:** TERMINATED
#### Primary Completion Date
**Date:** 2016-08
**Type:** ACTUAL
#### Results First Post Date
**Date:** 2022-08-12
**Type:** ACTUAL
**Results First Submit Date:** 2021-12-15
**Results First Submit QC Date:** 2022-08-11
#### Start Date
**Date:** 2013-06
**Status Verified Date:** 2022-08
#### Study First Post Date
**Date:** 2013-07-26
**Type:** ESTIMATED
**Study First Submit Date:** 2013-07-24
**Study First Submit QC Date:** 2013-07-25
**Why Stopped:** Main Study was completed. LT-OLE was discontinued as sufficient safety data has been generated.
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** OPKO Health, Inc.
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** This will be a randomized, double-blind, placebo-controlled, parallel-group, multicenter study in adult subjects with GHD to assess the safety and efficacy of a long-acting, once weekly injection of modified hGH (MOD-4023).
### Conditions Module
**Conditions:**
- Adult Growth Hormone Deficiency
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** QUADRUPLE
**Who Masked:**
- PARTICIPANT
- CARE_PROVIDER
- INVESTIGATOR
- OUTCOMES_ASSESSOR
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 202
**Type:** ACTUAL
**Phases:**
- PHASE3
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Intervention Names:**
- Drug: MOD-4023
**Label:** MOD-4023
**Type:** EXPERIMENTAL
#### Arm Group 2
**Intervention Names:**
- Other: Placebo
**Label:** Placebo
**Type:** PLACEBO_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- MOD-4023
**Description:** Individualized once weekly dose of MOD-4023
**Name:** MOD-4023
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- Placebo
**Description:** Once weekly administration of placebo
**Name:** Placebo
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Measure:** Change in Trunk Fat Mass, Expressed in Kilograms Measured With Dual-energy X-ray Absorptiometry, From Baseline to Week 26
**Time Frame:** Baseline to 26 weeks
#### Secondary Outcomes
**Measure:** Change in Total Fat Mass, Expressed in Kilograms, Measured With Dual-energy X-ray Absorptiometry, From Baseline to Week 26
**Time Frame:** Baseline to 26 weeks
**Measure:** Change in Lean Body Mass, Expressed in Kilograms Measured With Dual-energy X-ray Absorptiometry, From Baseline to Week 26
**Time Frame:** Baseline to 26 weeks
**Measure:** Change in Trunk Fat Mass, Expressed in Kilograms Measured With Dual-energy X-ray Absorptiometry, From Baseline to 52 Weeks
**Time Frame:** Baseline to 52 weeks
**Measure:** Change in Trunk Fat Mass, Expressed as % Change From Baseline, Measured With Dual-energy X-ray Absorptiometry, From Baseline to 26 and 52 Weeks
**Time Frame:** 26 weeks to 52 weeks
**Measure:** Trunk Fat Mass as Percentage of Total Fat Mass From Baseline to Week 26
**Time Frame:** Baseline to 26 weeks
**Measure:** Change in Biochemical Marker IGF-1
**Time Frame:** Baseline to 26 weeks
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Men and women between the age of 23 to 70 years old at screening, inclusive
* GHD subjects as defined in the Consensus guidelines for the diagnosis and treatment of adults with GH deficiency II (2007).
* No r-hGH replacement therapy or use of GH secretagogues for at least 9 months with any registered or investigational r-hGH or GH secretagogue product.
* The IGF-I level at screening ≤-1 SDS of the age and sex normal ranges according to the central laboratory measurements
* Subjects who are on a stable diet and exercise regime and do not have plans to modify their diet or exercise for at least 12 months
* Subject had a DXA screening and the results are interpretable according to the study plan.
Exclusion Criteria:
* Women who are pregnant or breast-feeding (at least 6 months delay from childbirth or lactation)
* Evidence of growth benign intracranial tumor within the last 12 months (determined by comparing a previous MRI to a new one obtained no more than 6 months prior to study entry to clarify dynamics of growth).
* History of any cancer. Exceptions to this exclusion criterion include resected in situ carcinoma of the cervix and squamous cell or basal cell carcinoma of the skin with complete local excision. Patients with GHD attributed to treatment of intracranial malignant lesions in childhood or adulthood (or, tumors) or leukemia may also be enrolled into the study provided that a recurrence-free survival period of at least 5 years is well documented in the study record.
* Signs of intracranial hypertension at screening
* Heart insufficiency, NYHA class \> 2 (Appendix B)
* History of overt diabetes mellitus (including currently treated, well-controlled DM) defined according to the American Diabetes Association (ADA) Criteriaa. A history of gestational diabetes, resolved after childbirth, is not exclusionary.
* History of Acromegaly
**Maximum Age:** 70 Years
**Minimum Age:** 23 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Kiryat Gat
**Country:** Israel
**Facility:** Opko Biologics
**Zip:** 8211804
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000004392
- Term: Dwarfism
- ID: D000001848
- Term: Bone Diseases, Developmental
- ID: D000001847
- Term: Bone Diseases
- ID: D000009140
- Term: Musculoskeletal Diseases
- ID: D000001849
- Term: Bone Diseases, Endocrine
- ID: D000007018
- Term: Hypopituitarism
- ID: D000010900
- Term: Pituitary Diseases
- ID: D000007027
- Term: Hypothalamic Diseases
- ID: D000001927
- Term: Brain Diseases
- ID: D000002493
- Term: Central Nervous System Diseases
- ID: D000009422
- Term: Nervous System Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC05
- Name: Musculoskeletal Diseases
- Abbrev: BC16
- Name: Diseases and Abnormalities at or Before Birth
- Abbrev: BC19
- Name: Gland and Hormone Related Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC10
- Name: Nervous System Diseases
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M7566
- Name: Dwarfism
- Relevance: LOW
- As Found: Unknown
- ID: M13791
- Name: Pituitary Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M7567
- Name: Dwarfism, Pituitary
- Relevance: HIGH
- As Found: Growth Hormone Deficiency
- ID: M7862
- Name: Endocrine System Diseases
- Relevance: HIGH
- As Found: Hormone Deficiency
- ID: M5126
- Name: Bone Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5127
- Name: Bone Diseases, Developmental
- Relevance: LOW
- As Found: Unknown
- ID: M12097
- Name: Musculoskeletal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5128
- Name: Bone Diseases, Endocrine
- Relevance: LOW
- As Found: Unknown
- ID: M10068
- Name: Hypopituitarism
- Relevance: LOW
- As Found: Unknown
- ID: M10077
- Name: Hypothalamic Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5204
- Name: Brain Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5742
- Name: Central Nervous System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: T2633
- Name: Growth Hormone Deficiency
- Relevance: HIGH
- As Found: Growth Hormone Deficiency
- ID: T3125
- Name: Isolated Growth Hormone Deficiency
- Relevance: HIGH
- As Found: Growth Hormone Deficiency
- ID: T2955
- Name: Hypopituitarism
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000004393
- Term: Dwarfism, Pituitary
- ID: D000004700
- Term: Endocrine System Diseases
### Intervention Browse Module - Browse Branches
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M9789
- Name: Hormones
- Relevance: LOW
- As Found: Unknown
### Misc Info Module
#### Removed Countries
- Country: United States
- Version Holder: 2024-05-24
## Results Section
### Adverse Events Module
**Description:** AEs collected and reported for each period of the study. Period 1, Baseline to 26 weeks Period 2, 26 - 52 weeks Period 3, \>52 weeks to end of study
#### Event Groups
**Group ID:** EG000
**Title:** MOD-4023 Period 1
**Deaths Num At Risk:** 133
**Description:** MOD-4023: Individualized once weekly dose of MOD-4023 (Period 1)
**ID:** EG000
**Other Num Affected:** 36
**Other Num at Risk:** 133
**Serious Number Affected:** 4
**Serious Number At Risk:** 133
**Title:** MOD-4023 Period 1
**Group ID:** EG001
**Title:** Placebo Period 1
**Deaths Num At Risk:** 65
**Description:** Placebo: Once weekly administration of placebo (Period 1. Only till week 26). Open label once weekly dose of MOD-4023 thereafter
**ID:** EG001
**Other Num Affected:** 27
**Other Num at Risk:** 65
**Serious Number Affected:** 5
**Serious Number At Risk:** 65
**Title:** Placebo Period 1
**Group ID:** EG002
**Title:** MOD-4023 Period 2 (Treatment Assignments at Period 1)
**Deaths Num At Risk:** 133
**Description:** MOD-4023: Individualized once weekly dose of MOD-4023 (Period 2)
**ID:** EG002
**Other Num Affected:** 28
**Other Num at Risk:** 133
**Serious Number Affected:** 5
**Serious Number At Risk:** 133
**Title:** MOD-4023 Period 2 (Treatment Assignments at Period 1)
**Group ID:** EG003
**Title:** Placebo Period 2 (Treatment Assignments at Period 1)
**Deaths Num At Risk:** 65
**Description:** Placebo assigned and treated in Period 1. Open-label MOD-4023 treatment in Periods 2 and 3
**ID:** EG003
**Other Num Affected:** 16
**Other Num at Risk:** 65
**Serious Number Affected:** 3
**Serious Number At Risk:** 65
**Title:** Placebo Period 2 (Treatment Assignments at Period 1)
**Group ID:** EG004
**Title:** MOD-4023 Period 3 (Treatment Assignments at Period 1)
**Deaths Num At Risk:** 111
**Description:** MOD-4023: Individualized once weekly dose of MOD-4023 (Period 3).
Period 3 was a long-term open label extension
**ID:** EG004
**Other Num Affected:** 46
**Other Num at Risk:** 111
**Serious Number Affected:** 10
**Serious Number At Risk:** 111
**Title:** MOD-4023 Period 3 (Treatment Assignments at Period 1)
**Group ID:** EG005
**Title:** Placebo Period 3 (Treatment Assignments at Period 1)
**Deaths Num Affected:** 1
**Deaths Num At Risk:** 50
**Description:** Placebo assigned and treated in Period 1. Open-label MOD-4023 treatment in Periods 2 and 3
**ID:** EG005
**Other Num Affected:** 30
**Other Num at Risk:** 50
**Serious Number Affected:** 8
**Serious Number At Risk:** 50
**Title:** Placebo Period 3 (Treatment Assignments at Period 1)
**Frequency Threshold:** 5
#### Other Events
**Term:** Injection site pain
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:**
**Term:** Nasopharyngitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:**
**Term:** Upper respiratory tract infection
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:**
**Term:** Pain in extremity
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:**
**Term:** Headache
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:**
**Term:** Arthralgia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:**
**Term:** Sinusitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:**
**Term:** Bronchitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:**
**Term:** Pyrexia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:**
**Term:** Paraesthesia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:**
**Term:** Diarrhoea
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:**
#### Serious Events
**Term:** Acute Myocardial Infarction
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Cardiac disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Atrial Fibrillation
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Cardiac disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Abdominal Pain
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Duodenal Ulcer Haemorrhage
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Gastric Disorder
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Diverticulitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Urinary Tract Infection
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Pain In Extremity
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Synovitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Dizziness
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Inguinal hernia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num Affected:** 1
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Asthenia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num Affected:** 1
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Oedema peripheral
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num Affected:** 1
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Cholecystitis acute
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Hepatobiliary disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num Affected:** 1
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Bronchitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num Affected:** 1
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Gastroenteritis viral
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num Affected:** 1
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Respiratory tract infection viral
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num Affected:** 1
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Upper limb fracture
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Injury, poisoning and procedural complications
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num Affected:** 1
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Dehydration
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num Affected:** 1
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Appendicitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num Affected:** 1
**Num At Risk:** 50
**Term:** Colonic abscess
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num Affected:** 1
**Num At Risk:** 50
**Term:** Gastroenteritis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num Affected:** 1
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Pneumonia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num Affected:** 1
**Num At Risk:** 50
**Term:** Pyelonephritis acute
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num Affected:** 1
**Num At Risk:** 50
**Term:** Angina pectoris
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Cardiac disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num Affected:** 1
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Atrial fibrillation
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Cardiac disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num Affected:** 1
**Num At Risk:** 50
**Term:** Myocardial infarction
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Cardiac disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num Affected:** 1
**Num At Risk:** 50
**Term:** Adrenal insufficiency
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Endocrine disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num Affected:** 1
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Pituitary-dependent Cushing's syndrom
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Endocrine disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num Affected:** 1
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Joint injury
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Injury, poisoning and procedural complications
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num Affected:** 1
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Pneumothorax traumatic
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Injury, poisoning and procedural complications
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num Affected:** 1
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Rib fracture
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Injury, poisoning and procedural complications
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num Affected:** 1
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Arthralgia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num Affected:** 1
**Num At Risk:** 50
**Term:** Lumbar spinal stenosis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num Affected:** 1
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Haemangioma
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps)
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num Affected:** 1
**Num At Risk:** 50
**Term:** Squamous cell carcinoma of lung
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps)
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num Affected:** 1
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Hemianopia heteronymous
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num Affected:** 1
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Spinal claudication
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num Affected:** 1
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Enterocolitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num Affected:** 1
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Pyrexia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num Affected:** 1
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Hyponatraemia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num At Risk:** 111
**Group ID:** EG005
**Num Affected:** 1
**Num At Risk:** 50
**Term:** Device loosening
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Product Issues
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num Affected:** 1
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Term:** Pneumonia aspiration
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 133
**Group ID:** EG001
**Num At Risk:** 65
**Group ID:** EG002
**Num At Risk:** 133
**Group ID:** EG003
**Num At Risk:** 65
**Group ID:** EG004
**Num Affected:** 1
**Num At Risk:** 111
**Group ID:** EG005
**Num At Risk:** 50
**Time Frame:** These events were collected throughout the duration of the study and cumulative to weeks Period 1, Baseline to 26 weeks Period 2, 26 - 52 weeks Period 3, >52 weeks to end of study **up to 2 years**
## Results Section - Baseline Characteristics Module
### Denomination Counts
**Group ID:** BG000
**Value:** 133
**Group ID:** BG001
**Value:** 65
**Group ID:** BG002
**Value:** 198
**Units:** Participants
### Group
**ID:** BG000
**Title:** MOD-4023
**Description:** MOD-4023: Individualized once weekly dose of MOD-4023
### Group
**ID:** BG001
**Title:** Placebo
**Description:** Placebo: Once weekly administration of placebo
### Group
**ID:** BG002
**Title:** Total
**Description:** Total of all reporting groups
### Measure
#### Measurement
**Group ID:** BG000
**Spread:** 12.6
**Value:** 43.9
#### Measurement
**Group ID:** BG001
**Spread:** 12.9
**Value:** 43.1
#### Measurement
**Group ID:** BG002
**Spread:** 12.6
**Value:** 43.9
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 57
#### Measurement
**Group ID:** BG001
**Value:** 25
#### Measurement
**Group ID:** BG002
**Value:** 82
**Category Title:** Female
#### Measurement
**Group ID:** BG000
**Value:** 76
#### Measurement
**Group ID:** BG001
**Value:** 40
#### Measurement
**Group ID:** BG002
**Value:** 116
**Category Title:** Male
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 110
#### Measurement
**Group ID:** BG001
**Value:** 53
#### Measurement
**Group ID:** BG002
**Value:** 163
**Class Title:** White
#### Measurement
**Group ID:** BG000
**Value:** 1
#### Measurement
**Group ID:** BG001
**Value:** 2
#### Measurement
**Group ID:** BG002
**Value:** 3
**Class Title:** Black or African American
#### Measurement
**Group ID:** BG000
**Value:** 15
#### Measurement
**Group ID:** BG001
**Value:** 8
#### Measurement
**Group ID:** BG002
**Value:** 23
**Class Title:** Asian
#### Measurement
**Group ID:** BG000
**Value:** 2
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 2
**Class Title:** Other
#### Measurement
**Group ID:** BG000
**Value:** 5
#### Measurement
**Group ID:** BG001
**Value:** 2
#### Measurement
**Group ID:** BG002
**Value:** 7
**Class Title:** Not Reported
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 31
#### Measurement
**Group ID:** BG001
**Value:** 15
#### Measurement
**Group ID:** BG002
**Value:** 46
**Class Title:** United States
#### Measurement
**Group ID:** BG000
**Value:** 88
#### Measurement
**Group ID:** BG001
**Value:** 43
#### Measurement
**Group ID:** BG002
**Value:** 131
**Class Title:** Europe
#### Measurement
**Group ID:** BG000
**Value:** 14
#### Measurement
**Group ID:** BG001
**Value:** 7
#### Measurement
**Group ID:** BG002
**Value:** 21
**Class Title:** East Asia
### Measure
#### Measurement
**Group ID:** BG000
**Spread:** 15.0
**Value:** 75.2
#### Measurement
**Group ID:** BG001
**Spread:** 17.9
**Value:** 77.8
#### Measurement
**Group ID:** BG002
**Spread:** 15.0
**Value:** 75.2
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Spread:** 3.2
**Value:** 27.0
#### Measurement
**Group ID:** BG001
**Spread:** 3.5
**Value:** 27.7
#### Measurement
**Group ID:** BG002
**Spread:** 3.2
**Value:** 27.0
**Class Title:**
**Denomination Units Selected:**
## Results Section - Baseline Characteristics Module
### Measure 1
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Age, Continuous
**Unit of Measure:** Year
### Measure 2
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Sex: Female, Male
**Unit of Measure:** Participants
### Measure 3
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Race/Ethnicity, Customized
**Unit of Measure:** Participants
### Measure 4
**Parameter Type:** NUMBER
**Title:** Region of Enrollment
**Unit of Measure:** participants
### Measure 5
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Weight
**Unit of Measure:** Kilogram
### Measure 6
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** BMI
**Unit of Measure:** Kilogram/meter^2
**Population Description:** 202 subjects met the study's eligibility criteria and were randomized to either MOD-4023 or placebo. Only 198 subjects actually treated and included in the efficacy analysis.
## Results Section - More Information Module
### Certain Agreement
**Restriction Type:** LTE60
**Restrictive Agreement:** True
### Point of Contact
**Email:** [email protected]
**Organization:** OPKO Health Inc
**Phone:** 3055754100
**Title:** OPKO Health Inc
## Results Section - Outcome Measures Module
### Outcome Measure 1
### Outcome Measure 2
### Outcome Measure 3
### Outcome Measure 4
### Outcome Measure 5
### Outcome Measure 6
### Outcome Measure 7
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 1.2
- **Upper Limit:**
- **Value:** -0.3
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 1.6
- **Upper Limit:**
- **Value:** 0.1
**Title:**
#### Outcome Measure 2
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 2.21
- **Upper Limit:**
- **Value:** -0.18
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 2.64
- **Upper Limit:**
- **Value:** 0.00
**Title:**
#### Outcome Measure 3
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 2.4
- **Upper Limit:**
- **Value:** 1.3
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 1.6
- **Upper Limit:**
- **Value:** 0.0
**Title:**
#### Outcome Measure 4
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 1.6
- **Upper Limit:**
- **Value:** -0.2
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 2.1
- **Upper Limit:**
- **Value:** -0.3
**Title:**
#### Outcome Measure 5
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 8.5
- **Upper Limit:**
- **Value:** -2.4
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 10.0
- **Upper Limit:**
- **Value:** 0.8
**Title:**
**Denomination:**
- **Group ID:** OG000
- **Value:** 125
- **Group ID:** OG001
- **Value:** 56
**Units:** Participants
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 3.1
- **Upper Limit:**
- **Value:** -1.6
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 2.8
- **Upper Limit:**
- **Value:** -1.2
**Title:**
**Denomination:**
- **Group ID:** OG000
- **Value:** 125
- **Group ID:** OG001
- **Value:** 55
**Units:** Participants
#### Outcome Measure 6
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 3.0
- **Upper Limit:**
- **Value:** -1.8
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 2.7
- **Upper Limit:**
- **Value:** 0.6
**Title:**
#### Outcome Measure 7
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 42.4
- **Upper Limit:**
- **Value:** 134.1
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 29.2
- **Upper Limit:**
- **Value:** 54.5
**Title:**
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** 198 of the 202 randomized subjects received treatment
**Reporting Status:** POSTED
**Time Frame:** Baseline to 26 weeks
**Title:** Change in Trunk Fat Mass, Expressed in Kilograms Measured With Dual-energy X-ray Absorptiometry, From Baseline to Week 26
**Type:** PRIMARY
**Unit of Measure:** kg
##### Group
**Description:** MOD-4023: Individualized once weekly dose of MOD-4023
**ID:** OG000
**Title:** MOD-4023
##### Group
**Description:** Placebo: Once weekly administration of placebo
**ID:** OG001
**Title:** Placebo
#### Outcome Measure 2
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** 3 subjects discontinued MOD-4023 due to consent withdrawn or lost to follow-up. 2 subjects discontinued placebo due to consent withdrawn or adverse event.
**Reporting Status:** POSTED
**Time Frame:** Baseline to 26 weeks
**Title:** Change in Total Fat Mass, Expressed in Kilograms, Measured With Dual-energy X-ray Absorptiometry, From Baseline to Week 26
**Type:** SECONDARY
**Unit of Measure:** kg
##### Group
**Description:** MOD-4023: Individualized once weekly dose of MOD-4023
**ID:** OG000
**Title:** MOD-4023
##### Group
**Description:** Placebo: Once weekly administration of placebo
**ID:** OG001
**Title:** Placebo
#### Outcome Measure 3
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** 3 subjects discontinued MOD-4023 due to consent withdrawn or lost to follow-up. 2 subjects discontinued placebo due to consent withdrawn or adverse event.
**Reporting Status:** POSTED
**Time Frame:** Baseline to 26 weeks
**Title:** Change in Lean Body Mass, Expressed in Kilograms Measured With Dual-energy X-ray Absorptiometry, From Baseline to Week 26
**Type:** SECONDARY
**Unit of Measure:** kg
##### Group
**Description:** MOD-4023: Individualized once weekly dose of MOD-4023
**ID:** OG000
**Title:** MOD-4023
##### Group
**Description:** Placebo: Once weekly administration of placebo
**ID:** OG001
**Title:** Placebo
#### Outcome Measure 4
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Reporting Status:** POSTED
**Time Frame:** Baseline to 52 weeks
**Title:** Change in Trunk Fat Mass, Expressed in Kilograms Measured With Dual-energy X-ray Absorptiometry, From Baseline to 52 Weeks
**Type:** SECONDARY
**Unit of Measure:** kg
##### Group
**Description:** MOD-4023: Individualized once weekly dose of MOD-4023
**ID:** OG000
**Title:** MOD-4023
##### Group
**Description:** Placebo: Once weekly administration of placebo
**ID:** OG001
**Title:** Placebo
#### Outcome Measure 5
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** 3 subjects discontinued MOD-4023 due to consent withdrawn or lost to follow-up. 2 subjects discontinued placebo due to consent withdrawn or adverse event.
**Reporting Status:** POSTED
**Time Frame:** 26 weeks to 52 weeks
**Title:** Change in Trunk Fat Mass, Expressed as % Change From Baseline, Measured With Dual-energy X-ray Absorptiometry, From Baseline to 26 and 52 Weeks
**Type:** SECONDARY
**Unit of Measure:** percentage of change from baseline
##### Group
**Description:** MOD-4023: Individualized once weekly dose of MOD-4023
**ID:** OG000
**Title:** MOD-4023
##### Group
**Description:** Placebo: Once weekly administration of placebo
**ID:** OG001
**Title:** Placebo
#### Outcome Measure 6
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Reporting Status:** POSTED
**Time Frame:** Baseline to 26 weeks
**Title:** Trunk Fat Mass as Percentage of Total Fat Mass From Baseline to Week 26
**Type:** SECONDARY
**Unit of Measure:** percentage of change from baseline
##### Group
**Description:** MOD-4023: Individualized once weekly dose of MOD-4023
**ID:** OG000
**Title:** MOD-4023
##### Group
**Description:** Placebo: Once weekly administration of placebo
**ID:** OG001
**Title:** Placebo
#### Outcome Measure 7
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Reporting Status:** POSTED
**Time Frame:** Baseline to 26 weeks
**Title:** Change in Biochemical Marker IGF-1
**Type:** SECONDARY
**Unit of Measure:** ug/L
##### Group
**Description:** MOD-4023: Individualized once weekly dose of MOD-4023
**ID:** OG000
**Title:** MOD-4023
##### Group
**Description:** Placebo: Once weekly administration of placebo
**ID:** OG001
**Title:** Placebo
### Participant Flow Module
#### Group
**Description:** MOD-4023: Individualized once weekly dose of MOD-4023
**ID:** FG000
**Title:** MOD-4023
#### Group
**Description:** Placebo: Once weekly administration of placebo
**ID:** FG001
**Title:** Placebo
#### Period
**Title:** From Baseline to Week 26
##### Withdraw
**Type:** Adverse Event
###### Reason
**Group ID:** FG000
**Number of Subjects:** 3
###### Reason
**Group ID:** FG001
**Number of Subjects:** 2
##### Withdraw
**Type:** Withdrawal by Subject
###### Reason
**Group ID:** FG000
**Number of Subjects:** 3
###### Reason
**Group ID:** FG001
**Number of Subjects:** 6
##### Withdraw
**Type:** Site and study closed by PI
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 1
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 135
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 67
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 129
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 58
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 6
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 9
#### Period
**Title:** 26 - 52 Weeks
##### Withdraw
**Type:** Adverse Event
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 1
##### Withdraw
**Type:** Withdrawal by Subject
###### Reason
**Group ID:** FG000
**Number of Subjects:** 2
###### Reason
**Group ID:** FG001
**Number of Subjects:** 1
##### Withdraw
**Type:** Lost to Follow-up
###### Reason
**Group ID:** FG000
**Number of Subjects:** 1
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
##### Milestone
**Type:** STARTED
**Comment:** One patient withdrew consent after completing Period 1
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 128
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 58
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 125
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 56
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 3
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 2
#### Period
**Title:** 52 Weeks to End of Study
##### Withdraw
**Type:** Withdrawal by Subject
###### Reason
**Group ID:** FG000
**Number of Subjects:** 18
###### Reason
**Group ID:** FG001
**Number of Subjects:** 8
##### Withdraw
**Type:** Adverse Event
###### Reason
**Group ID:** FG000
**Number of Subjects:** 2
###### Reason
**Group ID:** FG001
**Number of Subjects:** 1
##### Withdraw
**Type:** Death
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 1
##### Withdraw
**Type:** Lost to Follow-up
###### Reason
**Group ID:** FG000
**Number of Subjects:** 1
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
##### Withdraw
**Type:** Protocol Violation
###### Reason
**Group ID:** FG000
**Number of Subjects:** 1
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
##### Withdraw
**Type:** Progressive disease
###### Reason
**Group ID:** FG000
**Number of Subjects:** 1
###### Reason
**Group ID:** FG001
**Number of Subjects:** 1
##### Withdraw
**Type:** Personal reasons
###### Reason
**Group ID:** FG000
**Number of Subjects:** 2
###### Reason
**Group ID:** FG001
**Number of Subjects:** 1
##### Withdraw
**Type:** Sponsor's decision
###### Reason
**Group ID:** FG000
**Number of Subjects:** 1
###### Reason
**Group ID:** FG001
**Number of Subjects:** 1
##### Withdraw
**Type:** Study termination by sponsor
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 1
##### Withdraw
**Type:** Lack of contact
###### Reason
**Group ID:** FG000
**Number of Subjects:** 1
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
##### Withdraw
**Type:** Occurrence of malignancy
###### Reason
**Group ID:** FG000
**Number of Subjects:** 1
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
##### Withdraw
**Type:** Diabetes not controlled
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 1
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 111
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 50
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 83
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 35
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 28
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 15
**Has Results:** True |
## Protocol Section
### Identification Module
**NCT ID:** NCT06174779
**Brief Title:** A Study to Evaluate Efficacy and Safety of HM11260C in Adult Obesity Patients Without Diabetes Mellitus
**Official Title:** A Multicenter, Randomized, Double-blind, Phase 3 Study to Evaluate Efficacy and Safety of HM11260C in Adult Obesity Patients Without Diabetes Mellitus
#### Organization Study ID Info
**ID:** HM-EXC-301
#### Organization
**Class:** INDUSTRY
**Full Name:** Hanmi Pharmaceutical Company Limited
### Status Module
#### Completion Date
**Date:** 2026-07-31
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2023-12-18
**Type:** ACTUAL
**Last Update Submit Date:** 2023-12-08
**Overall Status:** NOT_YET_RECRUITING
#### Primary Completion Date
**Date:** 2025-12-31
**Type:** ESTIMATED
#### Start Date
**Date:** 2023-12-19
**Type:** ESTIMATED
**Status Verified Date:** 2023-12
#### Study First Post Date
**Date:** 2023-12-18
**Type:** ACTUAL
**Study First Submit Date:** 2023-12-08
**Study First Submit QC Date:** 2023-12-08
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Hanmi Pharmaceutical Company Limited
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
### Description Module
**Brief Summary:** A multicenter, randomized, double-blind, phase 3 study to evaluate efficacy and safety of HM11260C in adult obesity patients without diabetes mellitus
### Conditions Module
**Conditions:**
- Obesity
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** DOUBLE
**Who Masked:**
- PARTICIPANT
- INVESTIGATOR
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 420
**Type:** ESTIMATED
**Phases:**
- PHASE3
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Weekly administration by subcutaneous injection
**Intervention Names:**
- Drug: HM11260C
**Label:** HM11260C
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Weekly administration by subcutaneous injection
**Intervention Names:**
- Drug: Placebo
**Label:** Placebo
**Type:** PLACEBO_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- HM11260C
**Description:** Test drug
**Name:** HM11260C
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- Placebo
**Description:** Placebo drug
**Name:** Placebo
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Measure:** The percent change of body weight from baseline
**Time Frame:** baseline, 40 weeks
**Measure:** The proportion of subjects achieving body weight loss ≥ 5% from baseline
**Time Frame:** baseline, 40 weeks
#### Secondary Outcomes
**Measure:** The percent change of body weight from baseline
**Time Frame:** baseline, 24 weeks, 64 weeks
**Measure:** The proportion of subjects achieving body weight loss ≥ 5% from baseline
**Time Frame:** baseline, 24 weeks, 64 weeks
**Measure:** The proportion of subjects achieving body weight loss ≥ 10% from baseline
**Time Frame:** baseline, 24 weeks, 40 weeks, 64 weeks
**Measure:** Change of body weight from baseline
**Time Frame:** baseline, 24 weeks, 40 weeks, 64 weeks
**Measure:** Change of BMI from baseline
**Time Frame:** baseline, 24 weeks, 40 weeks, 64 weeks
**Measure:** Change of waist circumference from baseline
**Time Frame:** baseline, 24 weeks, 40 weeks, 64 weeks
**Measure:** Change of glucose metabolism parameters from baseline
**Time Frame:** baseline, 24 weeks, 40 weeks, 64 weeks
**Measure:** The percentage change of lipid profile parameters from baseline
**Time Frame:** baseline, 24 weeks, 40 weeks, 64 weeks
**Measure:** Change of blood pressure from baseline
**Time Frame:** baseline, 24 weeks, 40 weeks, 64 weeks
**Measure:** Change of IWQoL-Lite-CT(Physical function domain) from baseline
**Time Frame:** baseline, 24 weeks, 40 weeks, 64 weeks
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
1. Patients with following criteria :
* BMI ≥ 30 kg/m2 or
* 27 kg/m2 ≤ BMI \< 30 kg/m2 with more than one risk factors or comorbidities(hypertension, dyslipidemia, sleep apnea, cardiocerebrovascular disease)
2. A person who have failed at least once to weight control using diet and exercise therapy before screening visit.
Exclusion Criteria:
1. A person whose weight change exceeds 5kg within 3 months prior to screening visit.
2. Diabetes mellitus (Type 1, type 2, etc.) or HbA1c ≥ 6.5 %.
3. Administration of hypoglycemic agents.
4. Administration of medicines inducing weight gain.
5. Prader-Willi Syndrome or MC4R deficiency.
6. Cushing's Syndrome.
7. Administration of medicines for weight management.
8. A person who has a history of surgery(including device-based therapy) related to obesity or who has a plan during the clinical trial period.
9. Administration of Steroids for the systemic use.
10. Clinically significant gastric emptying abnormalities.
11. History(including family history) of medullary thyroid carcinoma or multiple endocrine adenomatosis.
12. History of acute or chronic pancreatitis.
13. A person who has the following clinical laboratory test results :
* TSH \< 0.4 mIU/L or TSH \> 6 mIU/L
* Calcitonin \> 100 ng/L
* Amylase or Lipase \> 3 x UNL
14. Severe liver disease (AST or AST \> 3 x UNL or Total bilirubin \> 1.5 x UNL).
15. Severe renal disease ( eGFR \< 30mL/min/1.73m 2 ).
16. QTc \> 450 ms.
17. Answered 'yes' to question 4 or 5 of the suicidal ideation domain in C-SSRS or history of suicide attempt.
18. PHQ-9 ≥ 15 or history of depression, anxiety, and other severe Mental illness.
19. History of alcohol addiction or drug abuse.
20. History of malignant tumors.
21. History of severe heart disease or severe neurovascular disease.
22. Hypersensitivity to investigational products or multi-drug allergy.
23. Positive to pregnancy test, nursing mother, intention on pregnancy.
24. Considered by investigator as not appropriate to participate in the clinical study with other reason.
**Minimum Age:** 19 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Na Young Kim
**Phone:** 82-2-410-9165
**Role:** CONTACT
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000050177
- Term: Overweight
- ID: D000044343
- Term: Overnutrition
- ID: D000009748
- Term: Nutrition Disorders
- ID: D000001835
- Term: Body Weight
- ID: D000044882
- Term: Glucose Metabolism Disorders
- ID: D000008659
- Term: Metabolic Diseases
- ID: D000004700
- Term: Endocrine System Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC18
- Name: Nutritional and Metabolic Diseases
- Abbrev: BC19
- Name: Gland and Hormone Related Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
### Condition Browse Module - Browse Leaves
- ID: M7115
- Name: Diabetes Mellitus
- Relevance: HIGH
- As Found: Diabetes Mellitus
- ID: M12701
- Name: Obesity
- Relevance: HIGH
- As Found: Obesity
- ID: M26186
- Name: Overweight
- Relevance: LOW
- As Found: Unknown
- ID: M25307
- Name: Overnutrition
- Relevance: LOW
- As Found: Unknown
- ID: M12684
- Name: Nutrition Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M5114
- Name: Body Weight
- Relevance: LOW
- As Found: Unknown
- ID: M11639
- Name: Metabolic Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M25403
- Name: Glucose Metabolism Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M7862
- Name: Endocrine System Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000009765
- Term: Obesity
- ID: D000003920
- Term: Diabetes Mellitus
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT05760079
**Acronym:** TSA
**Brief Title:** Lactoferrin for COVID-19-Induced Taste or Smell Abnormality
**Official Title:** A Pilot Study of Lactoferrin Among Patients Reporting Taste or Smell Abnormalities After COVID-19
#### Organization Study ID Info
**ID:** IRB00074029
#### Organization
**Class:** OTHER
**Full Name:** Wake Forest University Health Sciences
### Status Module
#### Completion Date
**Date:** 2022-09
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2023-03-08
**Type:** ACTUAL
**Last Update Submit Date:** 2023-03-07
**Overall Status:** WITHDRAWN
#### Primary Completion Date
**Date:** 2022-09
**Type:** ESTIMATED
#### Start Date
**Date:** 2022-06
**Type:** ESTIMATED
**Status Verified Date:** 2022-06
#### Study First Post Date
**Date:** 2023-03-08
**Type:** ACTUAL
**Study First Submit Date:** 2023-03-03
**Study First Submit QC Date:** 2023-03-07
**Why Stopped:** difficult to coordinate between all of the different support systems needed across various departments
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Wake Forest University Health Sciences
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** Common side effects of corona virus disease 2019 (COVID-19) include disruptions in taste and smell function, which may persist for prolonged periods of time following recovery and resolution of COVID-19 infection. These disruptions not only reduce the hedonic pleasure derived from eating, but may also be detrimental to quality of life and could pose additional health risks (malnutrition) among patients with chronic illness or those enduring long-term complications from their previous COVID-19 infection.
Previous studies conducted among patients with cancer experiencing taste and smell abnormalities have indicated improvement in taste and smell function following daily lactoferrin supplementation. Lactoferrin is a natural transferrin protein that scavenges and chelates iron byproducts produced as a function of lipid oxidation in the oral cavity following inflammation, infection, or toxicity of chemosensory tissues.
The purpose of this pilot investigation is to assess the feasibility and preliminary effectiveness of lactoferrin supplementation (750mg per day for 30 days) for the treatment of taste and smell disturbances following COVID-19 infection.
Approximately 40 patients who experienced disruptions in taste and smell following infection with COVID-19 will be recruited. Participants will complete baseline assessments (questionnaires, blood draw) and will be given 90 lactoferrin tablets (provided by Jarrow Formulas) in order to take 3 tablets per day for 30 days.
**Detailed Description:** Common side effects of corona virus disease 2019 (COVID-19) include disruptions in taste and smell function, which may persist for prolonged periods of time following recovery and resolution of COVID-19 infection. These disruptions not only reduce the hedonic pleasure derived from eating, but may also be detrimental to quality of life and could pose additional health risks (malnutrition) among patients with chronic illness or those enduring long-term complications from their previous COVID-19 infection.
Previous studies conducted among patients with cancer experiencing taste and smell abnormalities have indicated improvement in taste and smell function following daily lactoferrin supplementation. Lactoferrin is a natural transferrin protein that scavenges and chelates iron byproducts produced as a function of lipid oxidation in the oral cavity following inflammation, infection, or toxicity of chemosensory tissues.
The purpose of this pilot investigation is to assess the feasibility and preliminary effectiveness of lactoferrin supplementation (750mg per day for 30 days) for the treatment of taste and smell disturbances following COVID-19 infection. Primary outcomes will be subjective change in taste and smell abnormalities obtained via the Taste and Smell Questionnaire (TSQ).
Approximately 40 patients who experienced disruptions in taste and smell following infection with COVID-19 will be recruited. Participants will complete baseline assessments. Participants will be given 90 lactoferrin tablets (provided by Jarrow Formulas) in order to take 3 tablets per day for 30 days. Primary outcomes will be assessed immediately following the 30 day supplementation period. After 30 additional days without lactoferrin supplements (a washout period), participants will complete follow-up assessments.
### Conditions Module
**Conditions:**
- Covid19
- Taste Disorder, Secondary
- Taste Disorders
- Dysgeusia
- Smell Disorder
- Ageusia
- Anosmia
**Keywords:**
- Lactoferrin
- Taste disorder
- Smell disorder
- COVID-19
### Design Module
#### Design Info
**Allocation:** NA
**Intervention Model:** SINGLE_GROUP
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** 750mg lactoferrin (three 250mg tablets) per day for 30 days
**Intervention Names:**
- Dietary Supplement: Lactoferrin
**Label:** Lactoferrin Supplementation
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Lactoferrin Supplementation
**Description:** 750mg of Jarrow formulas taken three times daily for 30 days
**Name:** Lactoferrin
**Other Names:**
- Jarrow Formulas Apolactoferrin
**Type:** DIETARY_SUPPLEMENT
### Outcomes Module
#### Other Outcomes
**Description:** Participants will track their taste and smell function each day as well as their use of the lactoferrin supplement.
**Measure:** Daily self-monitoring
**Time Frame:** daily for 60 days
#### Primary Outcomes
**Description:** Ageusia or dysgeusia determined by subjective Smell and Taste Questionnaire (TSQ). As part of the questionnaire, participants will be asked to rate their individual taste and smell abnormalities as "insignificant," "mild," "moderate," "severe," or "incapacitating." The tool yields a taste complaint score (0-10) on the basis of subject responses to nine questions addressing changes to the sense of taste. One point is added for each reported taste complaint and two points for a rating of "severe" or "incapacitating" on the severity of the taste abnormality question.
**Measure:** Change in Taste disruption Scores
**Time Frame:** Baseline to day 60
**Description:** Anosmia or Dysnosmia determined by subjective Smell and Taste Questionnaire (TSQ). As part of the questionnaire, participants will be asked to rate their individual taste and smell abnormalities as "insignificant," "mild," "moderate," "severe," or "incapacitating." The tool yields a taste complaint score (0-6) on the basis of subject responses to five questions addressing changes to the sense smell. A smell complaint score (0-6) will be generated by adding one point for a positive response to each of five questions addressing self-perceived changes to the sense of smell. Two points will be assigned to a severity rating of "severe" or "incapacitating" for the severity of the smell abnormality question.
**Measure:** Change in Smell disruption Scores
**Time Frame:** Baseline to day 60
**Description:** The B-SIT is a shorter 12-item version of the full 40-item University of Pennsylvania Smell Identification Test (UPSIT). It is a validated 5 minute screening test for detecting smell loss. The test employs 12 well known cross cultural odors in a scratch and sniff format. Percentile norms are based upon nearly 4000 subjects via the B-SIT administration manual and both the test and the manual with a scoring key are available from Sensonics, Inc. (sensonics.com/smell-products/brief-smell-identification-test.html).
**Measure:** Change in Brief Smell Identification Test (B-SIT) Scores
**Time Frame:** Baseline to day 60
#### Secondary Outcomes
**Description:** Patient reported outcome of quality of life related to change in chemosensory function A validated, 35-item self-report measure that provides a comprehensive, convenient, and easy-to-use measure of an adult's appetite across 8 appetitive traits: Hunger (H), Food Responsiveness (FR), Emotional Over-Eating (EOE), Enjoyment of Food (EF), Satiety Responsiveness (SR), Emotional Under-eating (EUE), Food Fussiness (FF) and Slowness in Eating (SE). The AEBQ appears to be a reliable measure of appetitive traits in adults which translates well from the validated child measure (Child Eating Behaviour Questionnaire), and is unique in that it's scales assess food approach as well as food avoidance.95 Food Approach appetitive traits relevant to "Enjoyment of Food" will be assessed with 3 questions: "I enjoy eating", "I love food", and "I look forward to mealtimes". Internal reliability of the "Enjoyment of Food" subscale is 0.859 (Chronbach's alpha).
**Measure:** Adult Eating Behavior Questionnaire (AEBQ)
**Time Frame:** Baseline, day 30, day 60
**Description:** Patient reported outcome of factors related to anorexia and cachexia. Functional Assessment of Anorexia/Cachexia Treatment tool contains a validated 12 question additional concerns module, in addition to the Functional Assessment of Cancer Therapy - General (FACT-G), which measures nutritional quality of life.
**Measure:** Functional Assessment of Anorexia/Cachexia Tool (FAACT)
**Time Frame:** Baseline, day 30, day 60
**Description:** The EuroQOL is a self-reported generic health-related QoL (HRQoL) instrument that specifically addresses health status and has been tested among individuals with COVID-19 with taste and smell abnormality.4 It consists of five questions on mobility, self-care, usual activities, pain and discomfort and anxiety and depression with 3-point response categories (1=no problems, 2=some problems and 3=serious or extreme problems). The EQ-visual analogue scale (VAS) component of EQ-5D asks respondents to rate their overall health status from 0 (worst imaginable health) to 100 (best imaginable health).
**Measure:** Euro Quality of Life (EuroQOL)
**Time Frame:** Baseline, day 30, day 60
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* adult aged 18 or older; diagnosis of COVID-19 in the past 6 months; self-reported disruption in taste and smell beginning after infection with COVID-19
Exclusion Criteria:
* remains infectious from Covid-19; pregnant or lactating; taste or smell disruption existed prior to covid-19 infection; has cancer or is receiving cancer therapy; milk and/or iron allergy
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Overall Officials
**Official 1:**
**Affiliation:** Wake Forest University Health Sciences
**Name:** Glenn J Lesser, MD
**Role:** PRINCIPAL_INVESTIGATOR
### IPD Sharing Statement Module
**IPD Sharing:** NO
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000011024
- Term: Pneumonia, Viral
- ID: D000011014
- Term: Pneumonia
- ID: D000012141
- Term: Respiratory Tract Infections
- ID: D000007239
- Term: Infections
- ID: D000014777
- Term: Virus Diseases
- ID: D000018352
- Term: Coronavirus Infections
- ID: D000003333
- Term: Coronaviridae Infections
- ID: D000030341
- Term: Nidovirales Infections
- ID: D000012327
- Term: RNA Virus Infections
- ID: D000008171
- Term: Lung Diseases
- ID: D000012140
- Term: Respiratory Tract Diseases
- ID: D000012678
- Term: Sensation Disorders
- ID: D000009461
- Term: Neurologic Manifestations
- ID: D000009422
- Term: Nervous System Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC01
- Name: Infections
- Abbrev: BC08
- Name: Respiratory Tract (Lung and Bronchial) Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC16
- Name: Diseases and Abnormalities at or Before Birth
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: BC10
- Name: Nervous System Diseases
### Condition Browse Module - Browse Leaves
- ID: M2561
- Name: COVID-19
- Relevance: HIGH
- As Found: COVID-19
- ID: M12
- Name: Congenital Abnormalities
- Relevance: LOW
- As Found: Unknown
- ID: M12307
- Name: Neoplasm Metastasis
- Relevance: LOW
- As Found: Unknown
- ID: M2570
- Name: Anosmia
- Relevance: HIGH
- As Found: Anosmia
- ID: M3720
- Name: Ageusia
- Relevance: HIGH
- As Found: Ageusia
- ID: M7582
- Name: Dysgeusia
- Relevance: HIGH
- As Found: Dysgeusia
- ID: M4183
- Name: Olfaction Disorders
- Relevance: HIGH
- As Found: Smell Disorder
- ID: M16423
- Name: Taste Disorders
- Relevance: HIGH
- As Found: Taste Disorders
- ID: M13904
- Name: Pneumonia
- Relevance: LOW
- As Found: Unknown
- ID: M13914
- Name: Pneumonia, Viral
- Relevance: LOW
- As Found: Unknown
- ID: M10283
- Name: Infections
- Relevance: LOW
- As Found: Unknown
- ID: M6368
- Name: Communicable Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M14978
- Name: Respiratory Tract Infections
- Relevance: LOW
- As Found: Unknown
- ID: M17522
- Name: Virus Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M20490
- Name: Coronavirus Infections
- Relevance: LOW
- As Found: Unknown
- ID: M6555
- Name: Coronaviridae Infections
- Relevance: LOW
- As Found: Unknown
- ID: M23685
- Name: Nidovirales Infections
- Relevance: LOW
- As Found: Unknown
- ID: M15149
- Name: RNA Virus Infections
- Relevance: LOW
- As Found: Unknown
- ID: M11168
- Name: Lung Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M14977
- Name: Respiratory Tract Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M15490
- Name: Sensation Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M12404
- Name: Neurologic Manifestations
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000086382
- Term: COVID-19
- ID: D000086582
- Term: Anosmia
- ID: D000013651
- Term: Taste Disorders
- ID: D000004408
- Term: Dysgeusia
- ID: D000000370
- Term: Ageusia
- ID: D000000857
- Term: Olfaction Disorders
### Intervention Browse Module - Ancestors
- ID: D000000890
- Term: Anti-Infective Agents
### Intervention Browse Module - Browse Branches
- Abbrev: Infe
- Name: Anti-Infective Agents
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M10799
- Name: Lactoferrin
- Relevance: HIGH
- As Found: 175
- ID: M4214
- Name: Anti-Infective Agents
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: D000007781
- Term: Lactoferrin
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT03277079
**Acronym:** TACO
**Brief Title:** Low Dose Statins, Ezetimibe and Nutraceuticals
**Official Title:** Low Dose Statins, Ezetimibe and Nutraceuticals in Coronary Artery Disease Patients Intolerant to High Intensity Statin Treatment (TArget ChOlesterol)
#### Organization Study ID Info
**ID:** 123/D/2017
#### Organization
**Class:** OTHER
**Full Name:** University of Roma La Sapienza
### Status Module
#### Completion Date
**Date:** 2018-03-31
**Type:** ESTIMATED
#### Expanded Access Info
**Last Known Status:** NOT_YET_RECRUITING
#### Last Update Post Date
**Date:** 2017-09-08
**Type:** ACTUAL
**Last Update Submit Date:** 2017-09-06
**Overall Status:** UNKNOWN
#### Primary Completion Date
**Date:** 2017-12-31
**Type:** ESTIMATED
#### Start Date
**Date:** 2017-10-01
**Type:** ESTIMATED
**Status Verified Date:** 2017-09
#### Study First Post Date
**Date:** 2017-09-08
**Type:** ACTUAL
**Study First Submit Date:** 2017-09-06
**Study First Submit QC Date:** 2017-09-06
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** University of Roma La Sapienza
#### Responsible Party
**Investigator Affiliation:** University of Roma La Sapienza
**Investigator Full Name:** Francesco Pelliccia
**Investigator Title:** Assistant Professor
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** It remains unknown if the association between moderate to low intensity statin therapy and ezetimibe and nutraceuticals might have a therapeutic role in high-intensity statin intolerant patients
**Detailed Description:** The aim of this study will be to evaluate whether the association between statin and exetimibe vs statin + ezetimibe + nutraceuticals can lead to higher percentage of LDL target achievement
### Conditions Module
**Conditions:**
- Coronary Artery Disease
**Keywords:**
- coronary artery disease
- LDL cholesterol
- therapeutic target
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** SINGLE
**Who Masked:**
- PARTICIPANT
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 100
**Type:** ESTIMATED
**Phases:**
- PHASE4
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Low dose statin associated with ezetimibe
**Intervention Names:**
- Drug: statin + ezetimibe
- Drug: statin + ezetimibe + Nutraceuticals
**Label:** statin + ezetimibe
**Type:** ACTIVE_COMPARATOR
#### Arm Group 2
**Description:** Low dose statin associated with ezetimibe and Nutraceuticals
**Intervention Names:**
- Drug: statin + ezetimibe
- Drug: statin + ezetimibe + Nutraceuticals
**Label:** statin + ezetimibe + Nutraceuticals
**Type:** ACTIVE_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- statin + ezetimibe
- statin + ezetimibe + Nutraceuticals
**Description:** Combination of two drugs
**Name:** statin + ezetimibe
**Other Names:**
- Anticholesterol drug
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- statin + ezetimibe
- statin + ezetimibe + Nutraceuticals
**Description:** Combination of two grus plus nutraceuticals
**Name:** statin + ezetimibe + Nutraceuticals
**Other Names:**
- Anticholesterol drug
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Description:** LDL cholesterol \< 70 mg/dl
**Measure:** Therapeutic target
**Time Frame:** Up to 3 months
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
Patients with angiographically assessed diagnosis of coronary artery disease
Exclusion Criteria:
None
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Francesco Pelliccia, MD
**Phone:** 39064997
**Phone Ext:** 123
**Role:** CONTACT
**Contact 2:**
**Email:** [email protected]
**Name:** Giuseppe Marazzi, MD
**Phone:** 39064997
**Phone Ext:** 456
**Role:** CONTACT
#### Locations
**Location 1:**
**City:** Rome
**Country:** Italy
**Facility:** Sapienza University
**Zip:** 00161
#### Overall Officials
**Official 1:**
**Affiliation:** Sapienza University
**Name:** Carlo Gaudio, MD
**Role:** STUDY_CHAIR
### IPD Sharing Statement Module
**IPD Sharing:** NO
### References Module
#### References
**Citation:** Marazzi G, Campolongo G, Pelliccia F, Calabro Md P, Cacciotti L, Vitale C, Massaro R, Volterrani M, Rosano G. Usefulness of Low-Dose Statin Plus Ezetimibe and/or Nutraceuticals in Patients With Coronary Artery Disease Intolerant to High-Dose Statin Treatment. Am J Cardiol. 2019 Jan 15;123(2):233-238. doi: 10.1016/j.amjcard.2018.09.041. Epub 2018 Oct 19.
**PMID:** 30420184
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000006331
- Term: Heart Diseases
- ID: D000002318
- Term: Cardiovascular Diseases
- ID: D000001161
- Term: Arteriosclerosis
- ID: D000001157
- Term: Arterial Occlusive Diseases
- ID: D000014652
- Term: Vascular Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC14
- Name: Heart and Blood Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
### Condition Browse Module - Browse Leaves
- ID: M6549
- Name: Coronary Disease
- Relevance: HIGH
- As Found: Coronary Artery Disease
- ID: M6546
- Name: Coronary Artery Disease
- Relevance: HIGH
- As Found: Coronary Artery Disease
- ID: M19506
- Name: Myocardial Ischemia
- Relevance: HIGH
- As Found: Coronary Artery Disease
- ID: M10543
- Name: Ischemia
- Relevance: LOW
- As Found: Unknown
- ID: M9419
- Name: Heart Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M4469
- Name: Arteriosclerosis
- Relevance: LOW
- As Found: Unknown
- ID: M4465
- Name: Arterial Occlusive Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M17400
- Name: Vascular Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000003324
- Term: Coronary Artery Disease
- ID: D000017202
- Term: Myocardial Ischemia
- ID: D000003327
- Term: Coronary Disease
### Intervention Browse Module - Ancestors
- ID: D000000924
- Term: Anticholesteremic Agents
- ID: D000000960
- Term: Hypolipidemic Agents
- ID: D000000963
- Term: Antimetabolites
- ID: D000045504
- Term: Molecular Mechanisms of Pharmacological Action
- ID: D000057847
- Term: Lipid Regulating Agents
- ID: D000004791
- Term: Enzyme Inhibitors
### Intervention Browse Module - Browse Branches
- Abbrev: Lipd
- Name: Lipid Regulating Agents
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M409
- Name: Ezetimibe
- Relevance: HIGH
- As Found: Amount
- ID: M21155
- Name: Hydroxymethylglutaryl-CoA Reductase Inhibitors
- Relevance: HIGH
- As Found: Genotype
- ID: M4243
- Name: Anticholesteremic Agents
- Relevance: LOW
- As Found: Unknown
- ID: M4278
- Name: Hypolipidemic Agents
- Relevance: LOW
- As Found: Unknown
- ID: M4281
- Name: Antimetabolites
- Relevance: LOW
- As Found: Unknown
- ID: M28883
- Name: Lipid Regulating Agents
- Relevance: LOW
- As Found: Unknown
- ID: M7951
- Name: Enzyme Inhibitors
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: D000069438
- Term: Ezetimibe
- ID: D000019161
- Term: Hydroxymethylglutaryl-CoA Reductase Inhibitors
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT06400979
**Brief Title:** Aromatherapy for Management of Pain, Anxiety, and Nausea in the Acute Care Setting
**Official Title:** Aromatherapy for Management of Pain, Anxiety, and Nausea in the Acute Care Setting
#### Organization Study ID Info
**ID:** UCaliforniaDavis
#### Organization
**Class:** OTHER
**Full Name:** University of California, Davis
### Status Module
#### Completion Date
**Date:** 2022-05-01
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-05-06
**Type:** ACTUAL
**Last Update Submit Date:** 2024-05-01
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2022-05-01
**Type:** ACTUAL
#### Start Date
**Date:** 2020-12-01
**Type:** ACTUAL
**Status Verified Date:** 2024-05
#### Study First Post Date
**Date:** 2024-05-06
**Type:** ACTUAL
**Study First Submit Date:** 2024-01-06
**Study First Submit QC Date:** 2024-05-01
### Sponsor Collaborators Module
#### Collaborators
**Class:** INDUSTRY
**Name:** Beekley Medical
#### Lead Sponsor
**Class:** OTHER
**Name:** University of California, Davis
#### Responsible Party
**Investigator Affiliation:** University of California, Davis
**Investigator Full Name:** Carolyn Mendoza Mofidi
**Investigator Title:** Assistant Nurse Manager
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Is FDA Regulated Device:** True
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** The purpose of this interventional study is to investigate the effects of aromatherapy on an acute care unit and whether it is effective in decreasing physical or emotional stressors that occur as a hospitalized patient. This study aimed to expand the limited literature on aromatherapy use in hospitalized adults and its effectiveness in decreasing pain, anxiety, and nausea. The hypothesis was that use of aromatherapy would decrease pain, anxiety and nausea in hospitalized adults and increase patient satisfaction. While there is anecdotal evidence of its efficacy, few studies exist evaluating its effectiveness within peer-reviewed journals, specifically on acute care medical surgical units.
**Detailed Description:** The goal for the study was to collect a sample of one hundred adult patients admitted to an acute care medical surgical unit. A quasi-experimental study with a single-arm pre-/post-test design evaluated one-time use of inhaled aromatherapy on hospitalized adults on an acute care unit. Pre-test tools included a numeric pain rating scale, facial anxiety scale and Halpin's 0-5 nausea scale. Aromatherapy (Elequil aromatabs) was administered for 8 hours. Sleep, satisfaction, well-being, and concurrent medication use were assessed post-aromatherapy. Descriptive and inferential statistics were performed. These subjects had to be conscious, oriented, able to consent, and able to understand the purpose of the study. Exclusion criteria were otolaryngology free flap patients (as this was a study being conducted on an otolaryngology acute care unit and another study was occurring with this population), patients with known allergies to essential oils, those taking sleep medications, or who have an aroma/essential oil contraindication. The use of a pre and post survey was used to measure the effectiveness of aromatherapy on pain, anxiety, and nausea. A baseline assessment was made of these ailments via Qualtrix. Subjects chose the aromatherapy tab fragrance that best fit their chief complaint, which was placed on their gown. After eight hours, a post-application survey to assess pain, anxiety, nausea, sleep/relaxation, overall satisfaction, and overall wellbeing was administered via Qualtrix. A numeric pain scale from 0-10, a 0-5 Likert Anxiety scale and a 0-5 nausea scale were used to measure efficacy. Sleep, satisfaction, and wellbeing was assessed with yes/no. A chart review to determine any measurable benefits in the reduction of related medications while using aromatherapy was to be conducted once one hundred subjects were obtained. The period for this study was approximately fifteen months.
### Conditions Module
**Conditions:**
- Effectiveness of Aromatherapy
**Keywords:**
- aromatherapy
- acute care unit
- medical surgical unit
- inhaled aromatherapy
### Design Module
#### Design Info
**Allocation:** NA
**Intervention Model:** SINGLE_GROUP
**Intervention Model Description:** To collect a sample of one hundred adult patients admitted to an acute care medical surgical unit. A quasi-experimental study with a single-arm pre-/post-test design evaluated one-time use of inhaled aromatherapy on hospitalized adults on an acute care unit. Pre-test tools included a numeric pain rating scale, facial anxiety scale and 0-5 nausea scale by Halpin et al. Aromatherapy (Elequil aromatabs) was administered for 8 hours. Sleep, satisfaction, well-being, and concurrent medication use were assessed post-aromatherapy.
##### Masking Info
**Masking:** NONE
**Primary Purpose:** SUPPORTIVE_CARE
#### Enrollment Info
**Count:** 100
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Interventional aromatherapy tab used to deliver inhaled aromatherapy to participants
**Intervention Names:**
- Device: Elequil aromatab
**Label:** Aromatherapy aromatab
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Aromatherapy aromatab
**Description:** Aromatherapy impregnated tab utilized to dispense inhaled scent for the study
**Name:** Elequil aromatab
**Type:** DEVICE
### Outcomes Module
#### Primary Outcomes
**Description:** The use of a pre and post test to evaluate baseline and post data using 0-10 Numeric Scale where 0 is the best and 10 is the worst.
**Measure:** Aromatherapy is hypothesized to be effective in decreasing pain in the hospitalized patient.
**Time Frame:** 8 hours
**Description:** The use of a pre and post test to evaluate baseline and post data using 0-5 Likert scales where 0 is the best and 5 is the worst.
**Measure:** Aromatherapy is hypothesized to be effective in decreasing nausea in the hospitalized patient.
**Time Frame:** 8 hours
**Description:** The use of a pre and post test to evaluate baseline and post data using 0-5 Likert scale where 0 is the best and 5 is the worst.
**Measure:** Aromatherapy is hypothesized to be effective in decreasing anxiety in the hospitalized patient.
**Time Frame:** 8 hours
#### Secondary Outcomes
**Description:** The use of a posttest to evaluate effectiveness using a yes or no response.
**Measure:** Aromatherapy is hypothesized to increase satisfaction in the hospitalized patient
**Time Frame:** 8 hours
**Description:** The use of a posttest to evaluate effectiveness using a yes or no response.
**Measure:** Aromatherapy is hypothesized to increase well-being in the hospitalized patient
**Time Frame:** 8 hours
**Description:** The use of a posttest to evaluate effectiveness using a yes or no response.
**Measure:** Aromatherapy is hypothesized to increase sleep in the hospitalized patient
**Time Frame:** 8 hours
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Age 18 years or older,
* Admission to Tower 4 for an anticipated inpatient stay \>24 hours,
* Alert and oriented,
* Capable of using a visual scale to self-report symptoms,
* Naïve to the use of aromatherapy for the use of decreasing pain, anxiety and/or nausea to participate,
* Not on a pediatric service.
Exclusion Criteria:
* Cognitively impaired,
* Post-op from an otolaryngologic surgery (another study was being conducted concurrently with this patient population and we did not want to interfere with those results),
* Known impaired olfactory function (limited or no sense of smell),
* On any psychiatric holds (e.g., 5150's),
* Known allergies to essential oils,
* Sensitive or allergic to plants (specifically to lavender plants, orange blossoms, sandalwood trees, or peppermint leaves) as essential oils are natural aromas derived from plants,
* Active participant of another Research Protocol,
* Admitted as a "short stay" or on "observation" status,
* Have a known history of Atrial Fibrillation. The use of peppermint has known effects on stimulating atrial fibrillation,
* Is a prisoner,
* Known pregnancy, or
* Expected to be transferred out of Tower 4 and/or discharged from the hospital within twenty-four hours.
**Healthy Volunteers:** True
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Sacramento
**Country:** United States
**Facility:** UC Davis Medical Center
**State:** California
**Zip:** 95817
### IPD Sharing Statement Module
**Description:** We will not be sharing subject data after the conclusion of the study
**IPD Sharing:** NO
### References Module
#### References
**Citation:** Frosch PJ, Johansen JD, Menne T, Pirker C, Rastogi SC, Andersen KE, Bruze M, Goossens A, Lepoittevin JP, White IR. Further important sensitizers in patients sensitive to fragrances. Contact Dermatitis. 2002 Nov;47(5):279-87. doi: 10.1034/j.1600-0536.2002.470204.x.
**PMID:** 12534532
**Citation:** Bingham LJ, Tam MM, Palmer AM, Cahill JL, Nixon RL. Contact allergy and allergic contact dermatitis caused by lavender: A retrospective study from an Australian clinic. Contact Dermatitis. 2019 Jul;81(1):37-42. doi: 10.1111/cod.13247. Epub 2019 Apr 16.
**PMID:** 30779160
**Citation:** Boehm K, Bussing A, Ostermann T. Aromatherapy as an adjuvant treatment in cancer care--a descriptive systematic review. Afr J Tradit Complement Altern Med. 2012 Jul 1;9(4):503-18. doi: 10.4314/ajtcam.v9i4.7. eCollection 2012.
**PMID:** 23983386
**Citation:** Buckle J. The role of aromatherapy in nursing care. Nurs Clin North Am. 2001 Mar;36(1):57-72.
**PMID:** 11342402
**Citation:** Johnson JR, Rivard RL, Griffin KH, Kolste AK, Joswiak D, Kinney ME, Dusek JA. The effectiveness of nurse-delivered aromatherapy in an acute care setting. Complement Ther Med. 2016 Apr;25:164-9. doi: 10.1016/j.ctim.2016.03.006. Epub 2016 Mar 7.
**PMID:** 27062964
**Citation:** Cho MY, Min ES, Hur MH, Lee MS. Effects of aromatherapy on the anxiety, vital signs, and sleep quality of percutaneous coronary intervention patients in intensive care units. Evid Based Complement Alternat Med. 2013;2013:381381. doi: 10.1155/2013/381381. Epub 2013 Feb 17.
**PMID:** 23476690
**Citation:** Cooke B, Ernst E. Aromatherapy: a systematic review. Br J Gen Pract. 2000 Jun;50(455):493-6.
**PMID:** 10962794
**Citation:** Halpin A, Huckabay LM, Kozuki JL, Forsythe D. Weigh the benefits of using a 0-to-5 nausea scale. Nursing. 2010 Nov;40(11):18-20. doi: 10.1097/01.NURSE.0000389030.33760.7a. No abstract available.
**PMID:** 20975423
**Citation:** Lakhan SE, Sheafer H, Tepper D. The Effectiveness of Aromatherapy in Reducing Pain: A Systematic Review and Meta-Analysis. Pain Res Treat. 2016;2016:8158693. doi: 10.1155/2016/8158693. Epub 2016 Dec 14.
**PMID:** 28070420
**Citation:** Lis-Balchin M. Essential oils and 'aromatherapy': their modern role in healing. J R Soc Health. 1997 Oct;117(5):324-9. doi: 10.1177/146642409711700511.
**PMID:** 9519666
**Citation:** Long L, Huntley A, Ernst E. Which complementary and alternative therapies benefit which conditions? A survey of the opinions of 223 professional organizations. Complement Ther Med. 2001 Sep;9(3):178-85. doi: 10.1054/ctim.2001.0453.
**PMID:** 11926432
**Citation:** Maddocks-Jennings W, Wilkinson JM. Aromatherapy practice in nursing: literature review. J Adv Nurs. 2004 Oct;48(1):93-103. doi: 10.1111/j.1365-2648.2004.03172.x.
**PMID:** 15347415
**Citation:** Moeini M, Khadibi M, Bekhradi R, Mahmoudian SA, Nazari F. Effect of aromatherapy on the quality of sleep in ischemic heart disease patients hospitalized in intensive care units of heart hospitals of the Isfahan University of Medical Sciences. Iran J Nurs Midwifery Res. 2010 Fall;15(4):234-9.
**PMID:** 22049287
**Citation:** Quinlan-Colwell AD. Understanding the paradox of patient pain and patient satisfaction. J Holist Nurs. 2009 Sep;27(3):177-82; quiz 183-5. doi: 10.1177/0898010109332758. Epub 2009 Jul 8.
**PMID:** 19587387
**Citation:** Shin BC, Lee MS. Effects of aromatherapy acupressure on hemiplegic shoulder pain and motor power in stroke patients: a pilot study. J Altern Complement Med. 2007 Mar;13(2):247-51. doi: 10.1089/acm.2006.6189.
**PMID:** 17388768
**Citation:** Vickers A. Yes, but how do we know it's true? Knowledge claims in massage and aromatherapy. Complement Ther Nurs Midwifery. 1997 Jun;3(3):63-5. doi: 10.1016/s1353-6117(97)80035-2.
**PMID:** 9439251
#### See Also Links
**Label:** Instructional video on how to use aromatab
**URL:** https://vimeo.com/276457486
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000012817
- Term: Signs and Symptoms, Digestive
### Condition Browse Module - Browse Branches
- Abbrev: BXM
- Name: Behaviors and Mental Disorders
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: BC08
- Name: Respiratory Tract (Lung and Bronchial) Diseases
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M4324
- Name: Anxiety Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M12273
- Name: Nausea
- Relevance: HIGH
- As Found: Nausea
- ID: M27137
- Name: Respiratory Aspiration
- Relevance: LOW
- As Found: Unknown
- ID: M13066
- Name: Pain
- Relevance: LOW
- As Found: Unknown
- ID: M15622
- Name: Signs and Symptoms, Digestive
- Relevance: LOW
- As Found: Unknown
- ID: T170
- Name: Acute Graft Versus Host Disease
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000009325
- Term: Nausea
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT00005879
**Brief Title:** LY353381 in Preventing Breast Cancer in Women With Hyperplasia
**Official Title:** A Phase II Clinical Trial of a Selective Estrogen Receptor Modulator (LY353381*HCl) in High Risk Women With Fine Needle Aspiration Cytologic Evidence of Hyperplasia
#### Organization Study ID Info
**ID:** KUMC-HSC-7264-97
#### Organization
**Class:** OTHER
**Full Name:** University of Kansas Medical Center
#### Secondary ID Infos
**Domain:** PDQ (Physician Data Query)
**ID:** CDR0000067918
**Type:** REGISTRY
**Domain:** NCI
**ID:** NCI-P00-0146
**Type:** OTHER
**ID:** U01CA077165
**Link:** https://reporter.nih.gov/quickSearch/U01CA077165
**Type:** NIH
### Status Module
#### Completion Date
**Date:** 2008-07
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2017-05-30
**Type:** ACTUAL
**Last Update Submit Date:** 2017-04-19
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2008-07
**Type:** ACTUAL
#### Results First Post Date
**Date:** 2017-05-30
**Type:** ACTUAL
**Results First Submit Date:** 2017-01-23
**Results First Submit QC Date:** 2017-04-19
#### Start Date
**Date:** 2000-08
**Status Verified Date:** 2017-04
#### Study First Post Date
**Date:** 2003-01-27
**Type:** ESTIMATED
**Study First Submit Date:** 2000-06-02
**Study First Submit QC Date:** 2003-01-26
### Sponsor Collaborators Module
#### Collaborators
**Class:** NIH
**Name:** National Cancer Institute (NCI)
#### Lead Sponsor
**Class:** OTHER
**Name:** University of Kansas Medical Center
#### Responsible Party
**Investigator Affiliation:** University of Kansas Medical Center
**Investigator Full Name:** Carol Fabian, MD
**Investigator Title:** Director, Breast Cancer Prevention Unit
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** RATIONALE: Chemoprevention therapy is the use of certain drugs to try to prevent the development or recurrence of cancer. The use of LY353381 may be an effective way to prevent the development of breast cancer in women who have hyperplasia.
PURPOSE: Randomized phase II trial to study the effectiveness of LY353381 in preventing breast cancer in women who have hyperplasia.
**Detailed Description:** OBJECTIVES:
* Determine if LY353381 hydrochloride improves baseline cytology in women at high risk for breast cancer.
* Determine if this drug modulates other potential surrogate endpoint biomarkers or drug effect biomarkers.
* Determine if cytologic improvement is associated with initial presentation of the various stratification factors.
* Determine whether cytology is correlated with other potential surrogate endpoint biomarkers or drug effect biomarkers and whether change in cytology is correlated with change in the other biomarkers.
* Monitor the effects of this drug in terms of quality of life and women's health.
OUTLINE: This is a randomized, double-blind, multicenter study followed by an open-label study for both arms. Patients are stratified according to cytologic status (hyperplasia with atypia vs hyperplasia without atypia), mutation status (known carrier for BRCA1 or BRCA2 genes vs known not to be a carrier of mutant genes), menopausal status (premenopausal vs postmenopausal), estrogen-receptor status, and participating center. Patients are randomized to one of two treatment arms.
* Arm I: Patients receive oral LY353381 hydrochloride once daily for 6 months.
* Arm II: Patients receive oral placebo once daily for 6 months. Patients in both arms then receive oral LY353381 hydrochloride for an additional 6 months.
Quality of life is assessed at baseline and then at 6 and 12 months.
Patients are followed at 2 weeks and then annually for 5 years.
PROJECTED ACCRUAL: A total of 210-220 patients will be accrued for this study within 2.5-3 years.
### Conditions Module
**Conditions:**
- Breast Cancer
**Keywords:**
- breast cancer
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** QUADRUPLE
**Who Masked:**
- PARTICIPANT
- CARE_PROVIDER
- INVESTIGATOR
- OUTCOMES_ASSESSOR
**Primary Purpose:** PREVENTION
#### Enrollment Info
**Count:** 199
**Type:** ACTUAL
**Phases:**
- PHASE2
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Placebo
**Intervention Names:**
- Drug: Placebo
**Label:** Placebo
**Type:** PLACEBO_COMPARATOR
#### Arm Group 2
**Description:** LY353381, 20 mg daily
**Intervention Names:**
- Drug: arzoxifene
**Label:** Arzoxifene
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Arzoxifene
**Description:** one tablet daily
**Name:** arzoxifene
**Other Names:**
- LY353381
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- Placebo
**Description:** matched tablet dialy
**Name:** Placebo
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Description:** Change in the semi-quantitative score assigned by the designated cytopathologist.
Range 6-24. Score represents increasing abnormality (i.e., worse appearance) Sum composite of 6 cytomorphological features, each scored as 1-4.
**Measure:** Change in Masood Score
**Time Frame:** Baseline to 6 months
**Description:** Change (improvement) in categorical descriptor of cytologic abnormality as assigned by the primary cytopathologist.
Categories include: normal (non-proliferative), epithelial hyperplasia, epithelial hyperplasia with atypia.
**Measure:** Number of Participants With Improvement From Baseline in Cytomorphologic Abnormality at 6 Months
**Time Frame:** Baseline to 6 months
### Eligibility Module
**Eligibility Criteria:** DISEASE CHARACTERISTICS:
* Current random fine needle breast aspiration (FNA) evidence of 1 of the following:
* Hyperplasia with atypia
* Hyperplasia without atypia but with a 10-year modified Gail risk of at least 4%
* Hyperplasia without atypia but with a BRCAPRO risk of at least 25%
* Hyperplasia without atypia but with a known mutation in BRCA1 or BRCA2
* Hyperplasia without atypia but with a history of contralateral ductal carcinoma in situ or invasive breast cancer
* FNA must have been taken during days 1-14 of the menstrual cycle for premenopausal women
* Classified as ACR class I-III on mammogram with stepwedge within past 6 months If intact uterus and/or ovaries, must have color doppler transvaginal pelvic sonogram within past 6 months showing endometrial thickening no greater than 13 mm premenopausal or no greater than 8 mm postmenopausal
* No ovarian cysts felt to be possibly or probably non-physiologic that have not resolved to gynecologist's satisfaction on repeat sonogram
* Must agree to have or have had genetic counseling and genetic testing performed for BRCA1 and BRCA2
* No active cancer (e.g., detectable disease)
* Hormone receptor status:
* Not specified
PATIENT CHARACTERISTICS:
Age:
* 18 and over
Sex:
* Female
Menopausal status:
* Any
Performance status:
* Not specified
Life expectancy:
* At least 12 months
Hematopoietic:
* Hemoglobin greater than 10 g/dL
* Granulocyte count greater than 1,000/mm\^3
* No deficiencies in protein C, protein S, or antithrombin III
* No activated protein C resistance
Hepatic:
* Albumin greater than 3.0 g/dL
* Bilirubin less than 1.5 mg/dL
* AST less than 100 U/L
* Alkaline phosphatase less than 200 U/L
Renal:
* Creatinine less than 1.5 mg/dL
Cardiovascular:
* No history of deep venous thrombosis not related to trauma or pregnancy
* No severe coronary artery disease
* No history of prior stroke
Other:
* Not pregnant or nursing
* Negative pregnancy test
* Fertile patients must use effective contraception during and for 3 months after study
* No other active cancer
* No retinal vein thrombosis
* No concurrent severe poorly controlled migraine
* No factor V Leiden mutation carrier
PRIOR CONCURRENT THERAPY:
Biologic therapy:
* At least 12 months since prior immunotherapy
Chemotherapy:
* At least 3 months between completion of prior KUMC phase II difluoromethylornithine (DFMO) study and baseline aspiration
* At least 12 months since prior chemotherapy
Endocrine therapy:
* Must not have started or stopped hormone replacement therapy or oral contraceptives within 6 months of baseline aspiration
* Must continue all hormone replacement therapy and/or oral contraceptives that were being taken at time of baseline aspiration
* At least 12 months since prior tamoxifen, raloxifene, or other antihormonal therapy
Radiotherapy:
* At least 3 months since prior radiotherapy
Surgery:
* At least 6 months between prior oophorectomy and baseline aspiration
Other:
* At least 2 weeks since the start of other new medication that would be ingested for 1 or more months
**Healthy Volunteers:** True
**Maximum Age:** 65 Years
**Minimum Age:** 18 Years
**Sex:** FEMALE
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Kansas City
**Country:** United States
**Facility:** University of Kansas Medical Center
**State:** Kansas
**Zip:** 66160-7820
**Location 2:**
**City:** Dallas
**Country:** United States
**Facility:** U.S. Oncology Research, Inc.
**State:** Texas
**Zip:** 75246
#### Overall Officials
**Official 1:**
**Affiliation:** University of Kansas
**Name:** Carol J. Fabian, MD
**Role:** STUDY_CHAIR
### IPD Sharing Statement Module
**Description:** Global results will be published.
**IPD Sharing:** NO
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000009371
- Term: Neoplasms by Site
- ID: D000009369
- Term: Neoplasms
- ID: D000001941
- Term: Breast Diseases
- ID: D000012871
- Term: Skin Diseases
- ID: D000010335
- Term: Pathologic Processes
### Condition Browse Module - Browse Branches
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: BC17
- Name: Skin and Connective Tissue Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
### Condition Browse Module - Browse Leaves
- ID: M5220
- Name: Breast Neoplasms
- Relevance: HIGH
- As Found: Breast Cancer
- ID: M10016
- Name: Hyperplasia
- Relevance: HIGH
- As Found: Hyperplasia
- ID: M5218
- Name: Breast Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M15674
- Name: Skin Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000001943
- Term: Breast Neoplasms
- ID: D000006965
- Term: Hyperplasia
### Intervention Browse Module - Browse Branches
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M8116
- Name: Estrogens
- Relevance: LOW
- As Found: Unknown
- ID: M22599
- Name: Estrogen Receptor Modulators
- Relevance: LOW
- As Found: Unknown
- ID: M22597
- Name: Selective Estrogen Receptor Modulators
- Relevance: LOW
- As Found: Unknown
### Misc Info Module
- Version Holder: 2024-05-24
## Results Section
### Adverse Events Module
#### Event Groups
**Group ID:** EG000
**Title:** Placebo
**Description:** Placebo
Placebo: matched tablet dialy
**ID:** EG000
**Other Num Affected:** 100
**Other Num at Risk:** 101
**Serious Number Affected:** 5
**Serious Number At Risk:** 101
**Title:** Placebo
**Group ID:** EG001
**Title:** Arzoxifene
**Description:** LY353381, 20 mg daily
arzoxifene: one tablet daily
**ID:** EG001
**Other Num Affected:** 97
**Other Num at Risk:** 98
**Serious Number Affected:** 9
**Serious Number At Risk:** 98
**Title:** Arzoxifene
**Frequency Threshold:** 5
#### Other Events
**Term:** Asthenia
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Notes:** Fatigue
**Organ System:** General disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Vasodilation
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Notes:** Hot flashes
**Organ System:** General disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Leg Cramps
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Sweating
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Metrorrhagia
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Reproductive system and breast disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Urinary Frequency
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Renal and urinary disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Vaginitis
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Reproductive system and breast disorders
**Source Vocabulary:** CTCAE (3.0)
#### Serious Events
**Term:** APPENDICITIS
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** CTCAE (3.0)
##### Stats
**Group ID:** EG000
**Num Affected:** 2
**Num At Risk:** 101
**Num Events:** 2
**Group ID:** EG001
**Num At Risk:** 98
**Term:** SLIPPED DISK
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** CTCAE (3.0)
##### Stats
**Group ID:** EG000
**Num At Risk:** 101
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 98
**Num Events:** 1
**Term:** OVARIAN CYST
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Reproductive system and breast disorders
**Source Vocabulary:** CTCAE (3.0)
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 101
**Num Events:** 1
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 98
**Num Events:** 1
**Term:** ISCHEMIC COLITIS
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** CTCAE (3.0)
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 101
**Num Events:** 1
**Group ID:** EG001
**Num At Risk:** 98
**Term:** UTERINE DISORDER / MULTIPLE UTERINE FIBROIDS
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Reproductive system and breast disorders
**Source Vocabulary:** CTCAE (3.0)
##### Stats
**Group ID:** EG000
**Num At Risk:** 101
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 98
**Num Events:** 1
**Term:** ANGIOEDEMA
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Vascular disorders
**Source Vocabulary:** CTCAE (3.0)
##### Stats
**Group ID:** EG000
**Num At Risk:** 101
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 98
**Num Events:** 1
**Term:** DEEP VEIN THROMBOSIS RIGHT CALF
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Vascular disorders
**Source Vocabulary:** CTCAE (3.0)
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 101
**Num Events:** 1
**Group ID:** EG001
**Num At Risk:** 98
**Term:** RIGHT BIMALLEOLAR ANKLE FRACTURE
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** CTCAE (3.0)
##### Stats
**Group ID:** EG000
**Num At Risk:** 101
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 98
**Num Events:** 1
**Term:** BILATERAL CATARACTS
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** CTCAE (3.0)
##### Stats
**Group ID:** EG000
**Num At Risk:** 101
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 98
**Num Events:** 1
**Term:** ACUTE MYELOID LEUKEMIA
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps)
**Source Vocabulary:** CTCAE (3.0)
##### Stats
**Group ID:** EG000
**Num At Risk:** 101
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 98
**Num Events:** 1
**Term:** BREAST CANCER
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps)
**Source Vocabulary:** CTCAE (3.0)
##### Stats
**Group ID:** EG000
**Num At Risk:** 101
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 98
**Num Events:** 1
**Term:** PRIMARY DCIS IN BREAST
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps)
**Source Vocabulary:** CTCAE (3.0)
##### Stats
**Group ID:** EG000
**Num At Risk:** 101
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 98
**Num Events:** 1
**Time Frame:** Duration on study agent, nominally a maximum of 12 months.
## Results Section - Baseline Characteristics Module
### Denomination Counts
**Group ID:** BG000
**Value:** 101
**Group ID:** BG001
**Value:** 98
**Group ID:** BG002
**Value:** 199
**Units:** Participants
### Group
**ID:** BG000
**Title:** Placebo
**Description:** Placebo
Placebo: matched tablet dialy
### Group
**ID:** BG001
**Title:** Arzoxifene
**Description:** LY353381, 20 mg daily
arzoxifene: one tablet daily
### Group
**ID:** BG002
**Title:** Total
**Description:** Total of all reporting groups
### Measure
#### Measurement
**Group ID:** BG000
**Spread:** 7.1
**Value:** 46.5
#### Measurement
**Group ID:** BG001
**Spread:** 6.6
**Value:** 46.7
#### Measurement
**Group ID:** BG002
**Spread:** 6.9
**Value:** 46.6
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 101
#### Measurement
**Group ID:** BG001
**Value:** 98
#### Measurement
**Group ID:** BG002
**Value:** 199
**Category Title:** Female
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 0
**Category Title:** Male
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 101
#### Measurement
**Group ID:** BG001
**Value:** 98
#### Measurement
**Group ID:** BG002
**Value:** 199
**Class Title:** United States
### Measure
#### Measurement
**Group ID:** BG000
**Spread:** 2.6
**Value:** 64.8
#### Measurement
**Group ID:** BG001
**Spread:** 2.4
**Value:** 65.1
#### Measurement
**Group ID:** BG002
**Spread:** 2.5
**Value:** 65.0
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Spread:** 32
**Value:** 156
#### Measurement
**Group ID:** BG001
**Spread:** 40
**Value:** 157
#### Measurement
**Group ID:** BG002
**Spread:** 36
**Value:** 156
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Spread:** 5.5
**Value:** 26.3
#### Measurement
**Group ID:** BG001
**Spread:** 5.8
**Value:** 26.0
#### Measurement
**Group ID:** BG002
**Spread:** 5.6
**Value:** 26.1
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 52
#### Measurement
**Group ID:** BG001
**Value:** 51
#### Measurement
**Group ID:** BG002
**Value:** 103
**Category Title:** Pre
#### Measurement
**Group ID:** BG000
**Value:** 49
#### Measurement
**Group ID:** BG001
**Value:** 47
#### Measurement
**Group ID:** BG002
**Value:** 96
**Category Title:** Post
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 66
#### Measurement
**Group ID:** BG001
**Value:** 69
#### Measurement
**Group ID:** BG002
**Value:** 135
**Category Title:** None
#### Measurement
**Group ID:** BG000
**Value:** 35
#### Measurement
**Group ID:** BG001
**Value:** 29
#### Measurement
**Group ID:** BG002
**Value:** 64
**Category Title:** OC or HRT Use
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Spread:** 1.42
**Value:** 12.78
#### Measurement
**Group ID:** BG001
**Spread:** 1.45
**Value:** 12.82
#### Measurement
**Group ID:** BG002
**Spread:** 1.43
**Value:** 12.79
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Spread:** 10.4
**Value:** 20.6
#### Measurement
**Group ID:** BG001
**Spread:** 12.1
**Value:** 20.2
#### Measurement
**Group ID:** BG002
**Spread:** 11.2
**Value:** 20.4
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 17
#### Measurement
**Group ID:** BG001
**Value:** 17
#### Measurement
**Group ID:** BG002
**Value:** 34
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 23
#### Measurement
**Group ID:** BG001
**Value:** 27
#### Measurement
**Group ID:** BG002
**Value:** 50
**Class Title:** 0 Relatives
#### Measurement
**Group ID:** BG000
**Value:** 53
#### Measurement
**Group ID:** BG001
**Value:** 52
#### Measurement
**Group ID:** BG002
**Value:** 105
**Class Title:** 1 Relative
#### Measurement
**Group ID:** BG000
**Value:** 23
#### Measurement
**Group ID:** BG001
**Value:** 17
#### Measurement
**Group ID:** BG002
**Value:** 40
**Class Title:** 2 Relatives
#### Measurement
**Group ID:** BG000
**Value:** 2
#### Measurement
**Group ID:** BG001
**Value:** 2
#### Measurement
**Group ID:** BG002
**Value:** 4
**Class Title:** 3 Relatives
**Denomination Units Selected:**
## Results Section - Baseline Characteristics Module
### Measure 1
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Age, Continuous
**Unit of Measure:** years
### Measure 2
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Sex: Female, Male
**Unit of Measure:** Participants
### Measure 3
**Parameter Type:** NUMBER
**Title:** Region of Enrollment
**Unit of Measure:** participants
### Measure 4
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Height
**Unit of Measure:** inches
### Measure 5
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Weight
**Unit of Measure:** lb
### Measure 6
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** BMI
**Unit of Measure:** kg/m^2
### Measure 7
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Menopause Status
**Unit of Measure:** Participants
### Measure 8
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Hormone Use
**Unit of Measure:** Participants
### Measure 9
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Age at Menarche
**Unit of Measure:** years
### Measure 10
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Age at First Live Birth
**Unit of Measure:** years
### Measure 11
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Prior Biopsy
**Unit of Measure:** Participants
### Measure 12
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Number relatives with Breast Cancer
**Unit of Measure:** Participants
## Results Section - More Information Module
### Certain Agreement
**PI Sponsor Employee:** True
### Point of Contact
**Email:** [email protected]
**Organization:** University of Kansas Medical Center
**Phone:** 913-588-4523
**Title:** Bruce F. Kimler, Ph.D.
## Results Section - Outcome Measures Module
### Outcome Measure 1
### Outcome Measure 2
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 1.9
- **Upper Limit:**
- **Value:** -1.1
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 2.1
- **Upper Limit:**
- **Value:** -0.8
**Title:**
#### Outcome Measure 2
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 53
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 52
**Title:** No Improvement
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 31
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 30
**Title:** Improvement
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Description:** Change in the semi-quantitative score assigned by the designated cytopathologist.
Range 6-24. Score represents increasing abnormality (i.e., worse appearance) Sum composite of 6 cytomorphological features, each scored as 1-4.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** Analysis is restricted to subjects that complete the initial 6-month portion of the trial, have a repeat random periareolar fine needle aspiration, and are thus evaluable for change in Masood score.
**Reporting Status:** POSTED
**Time Frame:** Baseline to 6 months
**Title:** Change in Masood Score
**Type:** PRIMARY
**Unit of Measure:** units on a scale
##### Group
**Description:** Placebo
Placebo: matched tablet dialy
**ID:** OG000
**Title:** Placebo
##### Group
**Description:** LY353381, 20 mg daily
arzoxifene: one tablet daily
**ID:** OG001
**Title:** Arzoxifene
#### Outcome Measure 2
**Description:** Change (improvement) in categorical descriptor of cytologic abnormality as assigned by the primary cytopathologist.
Categories include: normal (non-proliferative), epithelial hyperplasia, epithelial hyperplasia with atypia.
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** Analysis is restricted to subjects that complete the initial 6-month portion of the trial, have a repeat random periareolar fine needle aspiration, and are thus evaluable for change in cytomorphology category.
**Reporting Status:** POSTED
**Time Frame:** Baseline to 6 months
**Title:** Number of Participants With Improvement From Baseline in Cytomorphologic Abnormality at 6 Months
**Type:** PRIMARY
**Unit of Measure:** Participants
##### Group
**Description:** Placebo
Placebo: matched tablet dialy
**ID:** OG000
**Title:** Placebo
##### Group
**Description:** LY353381, 20 mg daily
arzoxifene: one tablet daily
**ID:** OG001
**Title:** Arzoxifene
### Participant Flow Module
#### Group
**Description:** Placebo
Placebo: matched tablet dialy
**ID:** FG000
**Title:** Placebo
#### Group
**Description:** LY353381, 20 mg daily
arzoxifene: one tablet daily
**ID:** FG001
**Title:** Arzoxifene
#### Period
**Title:** Overall Study
##### Withdraw
**Type:** Withdrawal by Subject
###### Reason
**Group ID:** FG000
**Number of Subjects:** 8
###### Reason
**Group ID:** FG001
**Number of Subjects:** 10
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 101
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 98
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 93
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 88
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 8
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 10
**Recruitment Details:** Screen by random periareolar fine needle aspiration for presence of hyperplasia or hyperplasia with atypia.
**Has Results:** True |
## Protocol Section
### Identification Module
**NCT ID:** NCT02209779
**Brief Title:** Efficacy and Safety of Different Doses of BIRB 796 BS in Patients With Active Rheumatoid Arthritis
**Official Title:** A Randomised, Parallel, Double-blind, Placebo-controlled Study to Investigate Efficacy and Safety of Different Doses (5, 10, 20 and 30 mg) of BIRB 796 BS Administered Twice a Day Orally Over 4 Weeks in Patients With Active Rheumatoid Arthritis Who Have Failed at Least One DMARD
#### Organization Study ID Info
**ID:** 1175.11
#### Organization
**Class:** INDUSTRY
**Full Name:** Boehringer Ingelheim
### Status Module
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2014-08-06
**Type:** ESTIMATED
**Last Update Submit Date:** 2014-08-05
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2002-06
**Type:** ACTUAL
#### Start Date
**Date:** 2001-05
**Status Verified Date:** 2014-08
#### Study First Post Date
**Date:** 2014-08-06
**Type:** ESTIMATED
**Study First Submit Date:** 2014-08-05
**Study First Submit QC Date:** 2014-08-05
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Boehringer Ingelheim
#### Responsible Party
**Type:** SPONSOR
### Description Module
**Brief Summary:** The objective was to determine the effects of BIRB 796 BS on CRP and clinical parameters in Rheumatoid Arthritis as measures of efficacy, and on population pharmacokinetics and safety parameters
### Conditions Module
**Conditions:**
- Arthritis, Rheumatoid
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** DOUBLE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 167
**Type:** ACTUAL
**Phases:**
- PHASE2
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** twice daily doses of 5 mg for 4 weeks
**Intervention Names:**
- Drug: BIBR 796 BS
- Drug: Placebo
**Label:** BIBR 796 BS, low dose
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** twice daily doses of 10 mg for 4 weeks
**Intervention Names:**
- Drug: BIBR 796 BS
- Drug: Placebo
**Label:** BIBR 796 BS, medium dose 1
**Type:** EXPERIMENTAL
#### Arm Group 3
**Description:** twice daily doses of 20 mg for 4 weeks
**Intervention Names:**
- Drug: BIBR 796 BS
- Drug: Placebo
**Label:** BIBR 796 BS, medium dose 2
**Type:** EXPERIMENTAL
#### Arm Group 4
**Description:** twice daily doses of 30 mg for 4 weeks
**Intervention Names:**
- Drug: BIBR 796 BS
**Label:** BIBR 796 BS, high dose
**Type:** EXPERIMENTAL
#### Arm Group 5
**Intervention Names:**
- Drug: Placebo
**Label:** Placebo
**Type:** ACTIVE_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- BIBR 796 BS, high dose
- BIBR 796 BS, low dose
- BIBR 796 BS, medium dose 1
- BIBR 796 BS, medium dose 2
**Name:** BIBR 796 BS
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- BIBR 796 BS, low dose
- BIBR 796 BS, medium dose 1
- BIBR 796 BS, medium dose 2
- Placebo
**Name:** Placebo
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Measure:** Absolute difference to baseline in concentrations of C-reactive Protein (CRP)
**Time Frame:** before and after 4 weeks of treatment
#### Secondary Outcomes
**Measure:** Absolute difference to baseline in tender joint count (TJC, 68 joint count)
**Time Frame:** before and after 4 weeks of treatment
**Measure:** Absolute difference to baseline in swollen joint count (SJC, 66 joint count)
**Time Frame:** before and after 4 weeks of treatment
**Measure:** Patients assessment of pain on a visual analogue scale (VAS)
**Time Frame:** up to 57 days
**Measure:** Patients global assessment of disease activity (PADA) on a VAS
**Time Frame:** up to 57 days
**Measure:** Physicians global assessment of disease activity on a VAS
**Time Frame:** up to 57 days
**Measure:** Assessment of physical function by a standardised health assessment questionnaire (HAQ)
**Time Frame:** up to 57 days
**Measure:** Absolute difference to baseline in Erythrocyte sedimentation rate (ESR)
**Time Frame:** up to 57 days
**Measure:** Absolute difference to baseline in Cytokines Tumor Necrosis Factor (TNF)-α, soluble TNF-Receptor (sTNF-R), Interleukin (IL)-1ra, IL-6
**Time Frame:** Day 1, 8 and 29
**Measure:** Absolute difference to baseline in Matrix metalloprotease-3 (MMP-3)
**Time Frame:** Day 1, 8 and 29
**Measure:** Absolute difference to baseline in Vascular endothelial growth factor (VEGF)
**Time Frame:** Day 1, 8 and 29
**Measure:** Number of responders to American College of Rheumatology (ACR) preliminary response criteria for 20% improvement (ACR 20), ACR 50, ACR 70
**Time Frame:** after 4 weeks of treatment
**Measure:** Number of responders to European League against Rheumatism (EULAR) response criteria
**Time Frame:** after 4 weeks of treatment
**Measure:** Number of drop-outs due to lack of efficacy, according to final assessment of investigator
**Time Frame:** after 4 weeks of treatment
**Measure:** Assessment of maximum concentration (Cmax)
**Time Frame:** Day 15, 22, 29
**Measure:** Assessment of area under the curve (AUC) at steady state
**Time Frame:** Day 15, 22, 29
**Measure:** Number of patients with Adverse events
**Time Frame:** up to day 73
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Male or female from 18 to 75 years of age
* Diagnosis of Rheumatoid Arthritis (RA) established according to ACR criteria and date of diagnosis \>1 year to ≤ 15 years. The exclusion of patients with a disease duration \> 15 years was deleted in Amendment 2, effective January 22, 2002
* Patient belonging to functional class I, II, or III
* Failure of at least one Disease Modifying Antirheumatic Drug (DMARD) due to inefficacy
* Active disease, documented at visit 3, defined by ≥10 swollen joints in a 66 joint count and ≥ 12 tender joints in a 68 joint count
* CRP ≥ 2.0 mg/dl at visit 1 or visit 2
* Written informed consent in accordance with Good Clinical Practice and local legislation given prior to any study procedures, including washout of prohibited medications
Exclusion Criteria:
* Pregnancy (to be excluded by serum and urine β Human Chorion-Gonadotropin-test in women of childbearing potential) or breast feeding
* Female of childbearing potential (not 6 months post-menopausal or surgically sterilized) not using an approved form of birth control (hormonal contraceptives, oral or injectable/implantable, intra-uterine device (IUD))
* Inflammatory rheumatic disease other than RA
* Active vasculitis or any history of vasculitis (characterised by e.g. nail bed hemorrhages or infarcts, vasculitic purpura, ulcers or gangrenes, multisensory neuropathy, vasculitic retinopathy or scleritis of eyes). Isolated rheumatoid nodules of the skin are not a criterion for exclusion
* Treatment failure to a TNF-blocking agent. Treatment failure is defined as not achieving at least an ACR 20 response (e.g. in a clinical trial) or - in clinical practice - having the TNF-blocking agent discontinued due to ineffectiveness
* DMARD treatment within 4 weeks before visit 3
* Last dose given within the specified time period before visit 3 for one of the following compounds or drugs:
* Infliximab (Remicade®): 3 months
* D2E7 (a human TNF-α antibody): 3 months
* Leflunomide (Arava®): 1 year, with exception of patients having undergone elimination therapy (colestyramin 8 grams t.i.d. po for eleven consecutive days), this exclusion criterion was deleted in Amendment 1, effective September 3, 2001
* Drug classified as proton pump inhibitor: 7 days
* Drug classified as H2-receptor-blocker or antacid: 2 days
* Investigational agent: 5-fold of the respective plasma half life or 4 weeks, whichever is longer
* Treatment with systemic corticosteroids in a dose higher than 10 mg/day prednisone equivalent within 4 weeks prior to visit 3
* Change in treatment with nonsteroidal antiinflammatory drugs (NSAIDs) or systemic corticosteroids within 4 weeks prior to visit 3
* Synovectomy, joint surgery, radio-/chemo synoviorthesis or steroid injections (intraarticular, intravenous or intramuscular) within 4 weeks before visit 3
* Active infection or serious infectious diseases resulting in hospitalisation or requiring systemic anti-infective therapy within 4 weeks before visit 3
* Serologic evidence of active hepatitis B and/or C
* Known HIV-infection
* History of prior tuberculosis infection or suspicion of active infection at screening based on chest x-ray done within 6 month before visit 1
* History of cardiovascular, renal, neurologic, psychiatric, liver, gastrointestinal, immunologic or endocrine dysfunction if they are clinically significant. A clinically significant disease is defined as one which in the opinion of the investigator may either put the patient at risk because of participation in the study or a disease which may influence the results of the study or the patient's ability to participate in the study
* Recent history of heart failure, defined according to New York Heart Association criteria as being stage III or IV (i.e. three years or less) or myocardial infarction (i.e. one year or less) or patients with any cardiac arrhythmia requiring drug therapy. This exclusion criterion was slightly modified in Amendment 2, effective January 22, 2002.
* ECG results outside of the reference range of clinical relevance including, but not limited to QTcB \> 480 msec, PR interval \> 240 msec, QRS interval \> 110 msec
* History of malignant disease in the last 5 years or suspicion of active malignant disease except successfully treated squamous or basal cell carcinoma of the skin
* Clinically significant abnormal baseline hematology, blood chemistry or urinalysis if the abnormality defines a disease listed as an exclusion criterion
* Any of the following specific laboratory abnormalities:
* Alanine aminotransferase, aspartate aminotransferase, or total bilirubin greater than upper limit of normal (ULN) at visit 1 or measured within the last six months before visit 3. This exclusion criterion was modified in Amendment 1, effective September 3, 2001, allowing for a retest at Visit 2.
* Gamma-Glutamyltransferase, alkaline phosphatase or Lactate Dehydrogenase greater than 1.5 x ULN at visit 1
* creatinine or white blood cell count greater than 1.5 x ULN at visit 1
* History of drug or alcohol abuse within the past two years or active drug or alcohol abuse, present alcohol intake more than three drinks per day
* Inability to comply with the protocol
* Participation in another clinical trial within 30 days before visit 3
* Previous enrolment in this trial
* Hypersensitivity to trial drug
**Maximum Age:** 75 Years
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000007592
- Term: Joint Diseases
- ID: D000009140
- Term: Musculoskeletal Diseases
- ID: D000012216
- Term: Rheumatic Diseases
- ID: D000003240
- Term: Connective Tissue Diseases
- ID: D000001327
- Term: Autoimmune Diseases
- ID: D000007154
- Term: Immune System Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC05
- Name: Musculoskeletal Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC17
- Name: Skin and Connective Tissue Diseases
- Abbrev: BC20
- Name: Immune System Diseases
### Condition Browse Module - Browse Leaves
- ID: M4476
- Name: Arthritis
- Relevance: HIGH
- As Found: Arthritis
- ID: M4480
- Name: Arthritis, Rheumatoid
- Relevance: HIGH
- As Found: Arthritis, Rheumatoid
- ID: M10621
- Name: Joint Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M12097
- Name: Musculoskeletal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M15045
- Name: Rheumatic Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M6323
- Name: Collagen Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M6464
- Name: Connective Tissue Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M4629
- Name: Autoimmune Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M10200
- Name: Immune System Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000001168
- Term: Arthritis
- ID: D000001172
- Term: Arthritis, Rheumatoid
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT03699579
**Brief Title:** Pharmaco-Economic Study of Treatment Options in Patients With Advanced RCC
**Official Title:** Patients Characteristics, Treatment Utilization, Costs and Outcomes in Patients With Advanced or Metastatic Renal Cell Carcinoma (RCC) Who Received at Least One Prior Vascular Endothelial Growth Factor (VEGF)-Targeted Therapy in Taiwan
#### Organization Study ID Info
**ID:** A-TW-60000-022
#### Organization
**Class:** INDUSTRY
**Full Name:** Ipsen
### Status Module
#### Completion Date
**Date:** 2019-06-23
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2019-08-28
**Type:** ACTUAL
**Last Update Submit Date:** 2019-08-23
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2019-06-23
**Type:** ACTUAL
#### Start Date
**Date:** 2019-02-22
**Type:** ACTUAL
**Status Verified Date:** 2019-08
#### Study First Post Date
**Date:** 2018-10-09
**Type:** ACTUAL
**Study First Submit Date:** 2018-10-05
**Study First Submit QC Date:** 2018-10-05
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Ipsen
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Is US Export:** False
### Description Module
**Brief Summary:** This is a retrospective cohort study aiming to collect data on patients' characteristics, resource utilization, adverse events management and calculate costs attributed to current treatments of advance RCC patients who have received at least one prior VEGF-targeted therapy in Taiwan from National Health Insurance (NHI) perspective.
### Conditions Module
**Conditions:**
- Renal Cell Carcinoma Metastatic
- Renal Cell Carcinoma
**Keywords:**
- advanced or metastatic renal cell carcinoma (RCC)
### Design Module
#### Design Info
**Observational Model:** COHORT
**Time Perspective:** RETROSPECTIVE
#### Enrollment Info
**Count:** 28
**Type:** ACTUAL
**Study Type:** OBSERVATIONAL
### Outcomes Module
#### Primary Outcomes
**Measure:** Medication usage
**Time Frame:** From start point for 2.5 months
**Measure:** Treatment's duration
**Time Frame:** From start point for 2.5 months
#### Secondary Outcomes
**Measure:** Cost of medication used
**Time Frame:** From start point for 2.5 months
**Description:** (total calculated costs attributed to Adverse Effects (AEs) management according to the attached list of adverse events
**Measure:** Costs of adverse event management
**Time Frame:** From start point for 2.5 months
**Description:** Outpatient department visits, CT scan, blood test number and types
**Measure:** Frequency of health resource usage
**Time Frame:** From start point for 2.5 months
**Description:** Outpatient department visits, CT scan, blood test number and types
**Measure:** Cost of health resource usage
**Time Frame:** From start point for 2.5 months
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Patients with confirmed diagnosis of clear-cell renal cell carcinoma
* Patients with evidence of metastatic disease
* Patients who have received at least one previous VEGFR-targeted therapy, i.e sunitinib, pazopanib or sorafenib
* Patients who received care at the selected medical centers, utilizing the National Health Insurance (NHI) reimbursed system at the time of the disease
Exclusion Criteria:
* Patients enrolled in any clinical trial involving anti-cancer therapy
* Pregnant woman
**Minimum Age:** 18 Years
**Sampling Method:** NON_PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
**Study Population:** Hospital chart review study
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Taipei
**Country:** Taiwan
**Facility:** National Taiwan University Hospital
**Zip:** 100
**Location 2:**
**City:** Taipei
**Country:** Taiwan
**Facility:** Taipei Veterans General Hospital
**Zip:** 112
#### Overall Officials
**Official 1:**
**Affiliation:** Ipsen
**Name:** Ipsen Medical Director
**Role:** STUDY_DIRECTOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000009375
- Term: Neoplasms, Glandular and Epithelial
- ID: D000009370
- Term: Neoplasms by Histologic Type
- ID: D000009369
- Term: Neoplasms
- ID: D000000230
- Term: Adenocarcinoma
- ID: D000007680
- Term: Kidney Neoplasms
- ID: D000014571
- Term: Urologic Neoplasms
- ID: D000014565
- Term: Urogenital Neoplasms
- ID: D000009371
- Term: Neoplasms by Site
- ID: D000052776
- Term: Female Urogenital Diseases
- ID: D000005261
- Term: Female Urogenital Diseases and Pregnancy Complications
- ID: D000091642
- Term: Urogenital Diseases
- ID: D000007674
- Term: Kidney Diseases
- ID: D000014570
- Term: Urologic Diseases
- ID: D000052801
- Term: Male Urogenital Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: All
- Name: All Conditions
- Abbrev: BXS
- Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M5534
- Name: Carcinoma
- Relevance: HIGH
- As Found: Carcinoma
- ID: M5548
- Name: Carcinoma, Renal Cell
- Relevance: HIGH
- As Found: Renal Cell Carcinoma
- ID: M12320
- Name: Neoplasms, Glandular and Epithelial
- Relevance: LOW
- As Found: Unknown
- ID: M12315
- Name: Neoplasms by Histologic Type
- Relevance: LOW
- As Found: Unknown
- ID: M3585
- Name: Adenocarcinoma
- Relevance: LOW
- As Found: Unknown
- ID: M10703
- Name: Kidney Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M17320
- Name: Urologic Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M17315
- Name: Urogenital Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M2875
- Name: Urogenital Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M27093
- Name: Female Urogenital Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M14127
- Name: Pregnancy Complications
- Relevance: LOW
- As Found: Unknown
- ID: M8399
- Name: Female Urogenital Diseases and Pregnancy Complications
- Relevance: LOW
- As Found: Unknown
- ID: M10698
- Name: Kidney Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M17319
- Name: Urologic Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M27095
- Name: Male Urogenital Diseases
- Relevance: LOW
- As Found: Unknown
- ID: T4906
- Name: Renal Cell Carcinoma
- Relevance: HIGH
- As Found: Renal Cell Carcinoma
- ID: T1341
- Name: Clear Cell Renal Cell Carcinoma
- Relevance: HIGH
- As Found: Renal Cell Carcinoma
### Condition Browse Module - Meshes
- ID: D000002277
- Term: Carcinoma
- ID: D000002292
- Term: Carcinoma, Renal Cell
### Intervention Browse Module - Browse Branches
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M18681
- Name: Endothelial Growth Factors
- Relevance: LOW
- As Found: Unknown
- ID: M11900
- Name: Mitogens
- Relevance: LOW
- As Found: Unknown
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT03339479
**Brief Title:** Dielectric Property Test Compared With Frozen Pathological Section in the Rapid Diagnosis of Lung Nodules/Mass
**Official Title:** Dielectric Property Test Compared With Frozen Pathological Section in the Rapid Diagnosis of Lung Nodules/Mass: a Diagnostic Clinical Trial
#### Organization Study ID Info
**ID:** NFEC-2017-070
#### Organization
**Class:** OTHER
**Full Name:** Nanfang Hospital, Southern Medical University
### Status Module
#### Completion Date
**Date:** 2020-05-24
**Type:** ESTIMATED
#### Expanded Access Info
**Last Known Status:** RECRUITING
#### Last Update Post Date
**Date:** 2018-10-30
**Type:** ACTUAL
**Last Update Submit Date:** 2018-10-27
**Overall Status:** UNKNOWN
#### Primary Completion Date
**Date:** 2020-05-24
**Type:** ESTIMATED
#### Start Date
**Date:** 2017-05-25
**Type:** ACTUAL
**Status Verified Date:** 2018-05
#### Study First Post Date
**Date:** 2017-11-13
**Type:** ACTUAL
**Study First Submit Date:** 2017-08-21
**Study First Submit QC Date:** 2017-11-08
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Nanfang Hospital, Southern Medical University
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** Patients with lung nodules/mass found by CT (computed tomography) or PET (positron emission tomography) who agree to receive lung resection are arranged to test the dielectric property before receiving the frozen pathological examination. And the final pathological results are recognized as the standard diagnosis. Then the sensitivity and specificity of the dielectric property test will be evaluated comparing with the frozen pathological examination.
### Conditions Module
**Conditions:**
- Lung Nodules
- Lung Mass
**Keywords:**
- dielectric property
- lung nodules
- differential diagnosis
- frozen pathological examination
### Design Module
#### Design Info
**Allocation:** NON_RANDOMIZED
**Intervention Model:** PARALLEL
**Intervention Model Description:** self-control method
##### Masking Info
**Masking:** NONE
**Primary Purpose:** DIAGNOSTIC
#### Enrollment Info
**Count:** 502
**Type:** ESTIMATED
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** The patient with lung nodules/mass is firstly arranged to be tested for dielectric property after the nodules/mass resection and cutting open.
**Intervention Names:**
- Diagnostic Test: dielectric property test
**Label:** dielectric property test
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** The resected lung nodules/mass will be sent for frozen pathological examination after dielectric property test.
**Intervention Names:**
- Diagnostic Test: frozen pathological examination
**Label:** frozen pathological examination
**Type:** PLACEBO_COMPARATOR
#### Arm Group 3
**Description:** The resected lung nodules/mass will undergo the final pathological examination for final diagnosis after dielectric property test and frozen pathological examination.
**Intervention Names:**
- Diagnostic Test: final pathological examination
**Label:** final pathological examination
**Type:** OTHER
### Interventions
#### Intervention 1
**Arm Group Labels:**
- dielectric property test
**Description:** Test the dielectric property of resected lung nodules/mass through a touching probe after the lung tissue with nodules/mass has been resected and cut open, which usually takes only 1-5 minutes.
**Name:** dielectric property test
**Type:** DIAGNOSTIC_TEST
#### Intervention 2
**Arm Group Labels:**
- frozen pathological examination
**Description:** And after the dielectric property test, the lung tissue will be sent for frozen pathological examination.
**Name:** frozen pathological examination
**Type:** DIAGNOSTIC_TEST
#### Intervention 3
**Arm Group Labels:**
- final pathological examination
**Description:** All of the lung nodules/mass will be diagnosed by final pathological examination and recognized as the final diagnosis after surgery.
**Name:** final pathological examination
**Type:** DIAGNOSTIC_TEST
### Outcomes Module
#### Primary Outcomes
**Description:** true positive rate in diagnosing lung nodules/masses
**Measure:** sensitivity of dielectric property test in diagnosing lung nodules/masses
**Time Frame:** 3-5 years
**Description:** true negative rate in diagnosing lung nodules/masses
**Measure:** specificity of dielectric property test in diagnosing lung nodules/masses
**Time Frame:** 3-5 years
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Patients with lung nodules/mass found by CT or PET who agree to receive lung resection;
* Diameter of the lung nodule/mass is longer than 8 millimeters;
* More than 50% of the lung nodule/mass is solid component by image examination;
* Diameter of the solid component is longer than 5 millimeters by intraoperative exploration.
Exclusion Criteria:
* Cutting open the resected nodule/mass will interfered with the pathological examination;
* Diameter of the solid component is shorter than 5 millimeters by intraoperative cutting open;
* Cutting open the lung nodule/mass will cause great damage to the patient, such as inducing allergic shock.
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Di Lu, MD,PhD
**Phone:** 86+13268379321
**Role:** CONTACT
**Contact 2:**
**Email:** [email protected]
**Name:** Siyang Feng, MD
**Phone:** 86+13570930671
**Role:** CONTACT
#### Locations
**Location 1:**
**City:** Guandong
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Di Lu, MD, PhD
- **Phone:** 86+13268379321
- **Role:** CONTACT
***Contact 2:***
- **Email:** [email protected]
- **Name:** Siyang Feng, MD
- **Phone:** 86+13570930671
- **Role:** CONTACT
**Country:** China
**Facility:** Nanfang Hospital, Southern Medical University
**State:** Guangdong
**Status:** RECRUITING
**Zip:** 510515
#### Overall Officials
**Official 1:**
**Affiliation:** Nanfang Hospital, Southern Medical University
**Name:** Di Lu, MD,PhD
**Role:** PRINCIPAL_INVESTIGATOR
### IPD Sharing Statement Module
**Description:** The results of dielectric property test, frozen pathological test and final pathological examination of each individual participant.
**IPD Sharing:** UNDECIDED
### References Module
#### References
**Citation:** Gabriel C, Gabriel S, Corthout E. The dielectric properties of biological tissues: I. Literature survey. Phys Med Biol. 1996 Nov;41(11):2231-49. doi: 10.1088/0031-9155/41/11/001.
**PMID:** 8938024
**Citation:** Gabriel S, Lau RW, Gabriel C. The dielectric properties of biological tissues: II. Measurements in the frequency range 10 Hz to 20 GHz. Phys Med Biol. 1996 Nov;41(11):2251-69. doi: 10.1088/0031-9155/41/11/002.
**PMID:** 8938025
**Citation:** Gabriel S, Lau RW, Gabriel C. The dielectric properties of biological tissues: III. Parametric models for the dielectric spectrum of tissues. Phys Med Biol. 1996 Nov;41(11):2271-93. doi: 10.1088/0031-9155/41/11/003.
**PMID:** 8938026
**Citation:** Joines WT, Zhang Y, Li C, Jirtle RL. The measured electrical properties of normal and malignant human tissues from 50 to 900 MHz. Med Phys. 1994 Apr;21(4):547-50. doi: 10.1118/1.597312.
**PMID:** 8058021
**Citation:** Lu Y, Li B, Xu J, Yu J. Dielectric properties of human glioma and surrounding tissue. Int J Hyperthermia. 1992 Nov-Dec;8(6):755-60. doi: 10.3109/02656739209005023.
**PMID:** 1479201
**Citation:** Surowiec AJ, Stuchly SS, Barr JB, Swarup A. Dielectric properties of breast carcinoma and the surrounding tissues. IEEE Trans Biomed Eng. 1988 Apr;35(4):257-63. doi: 10.1109/10.1374. No abstract available.
**PMID:** 2834285
**Citation:** Fu F, Xin SX, Chen W. Temperature- and frequency-dependent dielectric properties of biological tissues within the temperature and frequency ranges typically used for magnetic resonance imaging-guided focused ultrasound surgery. Int J Hyperthermia. 2014 Feb;30(1):56-65. doi: 10.3109/02656736.2013.868534. Epub 2014 Jan 13.
**PMID:** 24417349
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000010335
- Term: Pathologic Processes
### Condition Browse Module - Meshes
- ID: D000004194
- Term: Disease
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT01655979
**Acronym:** MEP
**Brief Title:** Medium-term Bedrest Whey Protein (MEP)
#### Organization Study ID Info
**ID:** ESA-AO-06-BR
#### Organization
**Class:** OTHER
**Full Name:** DLR German Aerospace Center
### Status Module
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2012-08-02
**Type:** ESTIMATED
**Last Update Submit Date:** 2012-07-31
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2012-04
**Type:** ACTUAL
#### Start Date
**Date:** 2011-08
**Status Verified Date:** 2012-07
#### Study First Post Date
**Date:** 2012-08-02
**Type:** ESTIMATED
**Study First Submit Date:** 2012-07-23
**Study First Submit QC Date:** 2012-07-31
### Sponsor Collaborators Module
#### Collaborators
**Class:** OTHER
**Name:** European Space Agency
**Class:** OTHER
**Name:** University Hospital, Clermont-Ferrand
**Class:** UNKNOWN
**Name:** Institut Pluridisciplinaire Hubert Curien, Strasbourg, France
**Class:** OTHER
**Name:** Charite University, Berlin, Germany
**Class:** OTHER
**Name:** University of Milan
**Class:** OTHER
**Name:** Université de Nice Sophia Antipolis
**Class:** OTHER
**Name:** University of Ottawa
**Class:** OTHER
**Name:** Manchester Metropolitan University
**Class:** OTHER
**Name:** University of Toronto
**Class:** OTHER
**Name:** Medical University of Graz
**Class:** OTHER
**Name:** University of Cologne
**Class:** OTHER
**Name:** Radboud University Medical Center
**Class:** OTHER
**Name:** University Hospital, Lille
**Class:** OTHER
**Name:** Leiden University Medical Center
#### Lead Sponsor
**Class:** OTHER
**Name:** DLR German Aerospace Center
#### Responsible Party
**Type:** SPONSOR
### Description Module
**Brief Summary:** The human being has shown that he can live and work in the space environment, but due to the lack of essential mechanical load on muscle and bone, the fluid-shift as well as alterations in the acid-base balance (mainly on account of nutritional factors), the exposure to microgravity results in a gradual degradation of muscle, bone and cartilage, deconditioning of the cardiovascular system and metabolic changes. Countermeasures to prevent all the deconditioning of the physiological systems are not yet fully effective and require further investigation.
A commonly utilized model of simulating the physiological effects of microgravity on the human organism on ground is the 6° head-down-tilt bed rest. In the present study the model has been used to study potential countermeasures to spaceflight-associated deconditioning.
One of the most constrictive changes appearing during space flight as well as during bed rest, are disuse-induced muscle losses. These are associated with a decrease in muscle protein synthesis, rather then an increase in muscle protein breakdown. Besides an effective training countermeasure, nutritional countermeasures gain respect in this context: supplementing conventional diets with whey protein or essential amino acids has been shown to increase muscle protein synthesis. Due to these anabolic properties whey protein seems promising to counteract disuse-induced muscle wasting.
Drawbacks of a high protein intake are calciuric effects, ascribed to the proton-release when metabolizing sulfur-containing amino acids. The so called 'low grade metabolic acidosis' has also shown to activate osteoclastic bone resorption and muscle protein degradation. Therefore, to maximize the anabolic potential of a whey protein supplementation, the acidogenic properties need to be compensated. As previous works suggest, a shift of acid base balance into the acid direction and the resulting changes in bone and protein turnover may be hindered by supplementing alkaline mineral salts.
In this regard, a mid-term bed rest study was performed in order to investigate the effect of a combined whey protein (0.6 g/kg body weight/day) and potassium bicarbonate (90 mmol/day) supplementation as a potential countermeasure to multiple physiological and metabolic alterations on the human body resulting from real and simulated microgravity.
### Conditions Module
**Conditions:**
- Countermeasure Evaluation
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** CROSSOVER
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 10
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Intervention Names:**
- Dietary Supplement: Whey Protein + Potassium bicarbonate
- Other: Control
**Label:** MEP-1
**Type:** EXPERIMENTAL
#### Arm Group 2
**Intervention Names:**
- Dietary Supplement: Whey Protein + Potassium bicarbonate
- Other: Control
**Label:** MEP-2
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- MEP-1
- MEP-2
**Description:** 0.6 mmol WP/kg body weight + 90 mmol KHCO3 during bed rest
**Name:** Whey Protein + Potassium bicarbonate
**Type:** DIETARY_SUPPLEMENT
#### Intervention 2
**Arm Group Labels:**
- MEP-1
- MEP-2
**Description:** Bed rest without dietary supplement
**Name:** Control
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Measure:** Change in body composition
**Time Frame:** Baseline, after 21 days of bed rest
#### Secondary Outcomes
**Measure:** Plasma Volume
**Time Frame:** Baseline, after 21 days of bed rest
**Measure:** Maximum volume of oxygen uptake
**Time Frame:** Baseline, after 21 days of bed rest
**Description:** During a an Isometric Maximum Voluntary Contraction Test on the knee extensors \& flexors, the plantarflexors and dorsiflexors, the elbow extensors \& flexors the Isometric Torque will be measured in Nm.
**Measure:** Isometric torque
**Time Frame:** Baseline, after 21 days of bed rest
**Measure:** Muscle fatigue
**Time Frame:** Baseline, after 21 days of bed rest
**Measure:** Bone metabolism
**Time Frame:** Baseline, after 2,5,14,21 days of bed rest, 1, 5, 14, 28 days after finishing bed rest
**Measure:** Bone mineral density + content
**Time Frame:** Baseline, after 21 days of bed rest
**Measure:** Standing balance
**Time Frame:** Baseline, after 21 days of bed rest
**Description:** Locomotion will be assessed by Dynamic Gait Index, specific parameters are: total Score and Subscore
**Measure:** Locomotion
**Time Frame:** Baseline, after 21 days of bed rest
**Measure:** Body mass
**Time Frame:** Daily for a duration of 35 days
**Measure:** Intracranial pressure
**Time Frame:** Baseline, after 1,4, 7,10,13,14,15,16,17,18,19,20,21 days of bed rest,1,2,4 days after finishing bed rest
**Measure:** Monitoring of Vitamin K status
**Time Frame:** Baseline, after 2,5,14,21 days of bed rest, 1, 5 days after finishing bed rest
**Measure:** Fat metabolism
**Time Frame:** Baseline, after 21 days of bed rest
**Measure:** Glucose metabolism
**Time Frame:** Baseline, after 21 days of bed rest, 4 days after finishing bed rest
**Measure:** Nitrogen balance
**Time Frame:** Daily for a duration of 33 days
**Measure:** Energy metabolism
**Time Frame:** Baseline, after 21 days of bed rest
**Measure:** Glucocorticoid activity
**Time Frame:** Baseline, after 2,3,7,8,12,13,16,17 days of bed rest, 2,3 days after finishing bed rest
**Measure:** Muscle metabolism
**Time Frame:** Baseline, after 21 days of bed rest
**Measure:** Acid base balance
**Time Frame:** Baseline, after 2, 14, 21 days of bed rest, 5 days after finishing bed rest
**Description:** Muscle sympathetic nerve activity is measured by MSNA recording by microneurography technique.
**Measure:** Sympathetic activity during orthostatic stress
**Time Frame:** Baseline, after 21 days of bed rest
**Description:** Visual Orientation is assessed by 'Oriented Character Recognition Test' and Luminous Line Test. The main parameter is Score.
**Measure:** Visual Orientation
**Time Frame:** Baseline, after 6,12,20 days of bed rest, 2,4 days after finishing bed rest
**Measure:** Plasma galanin and adrenomedullin responses during head up tilt test (orthostatic stress)
**Time Frame:** Baseline, after 21 days of bed rest
**Measure:** Cartilage metabolism and -thickness
**Time Frame:** Baseline, after 2,3,5,7,14,21 days of bed rest, 5 days after finishing bed rest
**Description:** Blood cell count, reticulocytes, Haptoglobin, Bilirubin, Ferritin, EPO, Thrombopoietin, Urinary Urobilinogen and Fecal Urobilinogen (markers of blood cell degradation)
**Measure:** Hematopoetic system
**Time Frame:** Baseline, after 10, 21 days of bed rest, 1, 28 days after finishing bed rest
**Measure:** Fat accumulation in bone marrow
**Time Frame:** Baseline, after 10, 21 days of bed rest, 3, 28 days after finishing bed rest
**Measure:** Achilles tendon structure
**Time Frame:** Baseline, after 21 days of bed rest, 2, 28 days after finishing bed rest
**Measure:** Headache - frequency and quality
**Time Frame:** Baseline, daily during 21 days of bed rest
**Measure:** Muscle volume
**Time Frame:** Baseline, after 20, 21 days of bed rest, 3 days after finishing bed rest
**Measure:** Free water and fat content in muscle
**Time Frame:** Baseline, after 20, 21 days of bed rest, 3 days after finishing bed rest
**Description:** Orthostatic tolerance will be assessed by Head up tilt test. The following parameters are assessed to measure orthostatic tolerance: beat-to-beat heart rate \[bpm\], beat-to-beat blood pressure \[bpm\] time to presyncope \[min, s\]
**Measure:** Orthostatic tolerance
**Time Frame:** Baseline, after 21 days of bed rest
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Healthy males, 20 -45 years
* BMI: 20 - 25 kg/m2
* Height: 158 - 190 cm
* Weight: 65 - 85 kg
* maximum relative oxygen uptake: 30 - 60 ml/min/kg
* non-smokers
* successful medical and psychological screening
* Willingness to participate in the entire study
* signed informed consent
* social insurance
* Clear criminal background check
Exclusion Criteria:
* Abuse of drugs, medicine or alcohol
* Vegetarians, Vegans
* Migraines
* History of mental illness
* Claustrophobia
* History of: thyroid dysfunction, renal stones, diabetes, allergies, hypertension, hypocalcaemia, uric acidaemia, lipidaemia, hyperhomocysteinaemia
* Rheumatism
* Muscle-, Cartilage- or Joint Injuries
* Gastro-esophageal reflux disease, renal function disorder, Hiatus hernia
* Chronic back pain
* Bone diseases
* Herniated discs
* Achilles tendon injuries
* Cruciate ligament rupture or any other severe knee injury
* BMD more than 1.5 SD \< t-score
* History of orthostatic intolerance or vestibular disorders
* Anaemia
* Vitamin D Deficiency
* Positive response in thrombosis screening
* Use of metallic implants, osteosynthesis material
* Porphyria, Blood dyscrasia
* HIV, Hepatitis
* Increased Inner Eye pressure
* Intolerance to local anesthetics
* Participation in another study up to three month before study onset
**Healthy Volunteers:** True
**Maximum Age:** 45 Years
**Minimum Age:** 20 Years
**Sex:** MALE
**Standard Ages:**
- ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Cologne
**Country:** Germany
**Facility:** DLR German Aerospace Center
**Zip:** 51147
### References Module
#### References
**Citation:** Rudwill F, O'Gorman D, Lefai E, Chery I, Zahariev A, Normand S, Pagano AF, Chopard A, Damiot A, Laurens C, Hodson L, Canet-Soulas E, Heer M, Meuthen PF, Buehlmeier J, Baecker N, Meiller L, Gauquelin-Koch G, Blanc S, Simon C, Bergouignan A. Metabolic Inflexibility Is an Early Marker of Bed-Rest-Induced Glucose Intolerance Even When Fat Mass Is Stable. J Clin Endocrinol Metab. 2018 May 1;103(5):1910-1920. doi: 10.1210/jc.2017-02267.
**PMID:** 29546280
## Derived Section
### Intervention Browse Module - Browse Branches
- Abbrev: Ot
- Name: Other Dietary Supplements
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: T435
- Name: Whey Protein
- Relevance: HIGH
- As Found: Ingredients
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT00002779
**Brief Title:** Fludarabine Plus Octreotide in Treating Patients With Relapsed Low-Grade Non-Hodgkin's Lymphoma
**Official Title:** PHASE II TRIAL OF FLUDARABINE AND SANDOSTATIN FOR RELAPSED LOW-GRADE NON-HODGKIN'S LYMPHOMA
#### Organization Study ID Info
**ID:** NCCTG-947851
#### Organization
**Class:** OTHER
**Full Name:** Alliance for Clinical Trials in Oncology
#### Secondary ID Infos
**ID:** NCCTG-947851
**Domain:** NCI Physician Data Query
**ID:** CDR0000064787
**Type:** REGISTRY
### Status Module
#### Completion Date
**Date:** 2015-06
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2016-12-15
**Type:** ESTIMATED
**Last Update Submit Date:** 2016-12-14
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2000-07
**Type:** ACTUAL
#### Start Date
**Date:** 1998-02
**Status Verified Date:** 2016-12
#### Study First Post Date
**Date:** 2004-07-29
**Type:** ESTIMATED
**Study First Submit Date:** 1999-11-01
**Study First Submit QC Date:** 2004-07-28
### Sponsor Collaborators Module
#### Collaborators
**Class:** NIH
**Name:** National Cancer Institute (NCI)
#### Lead Sponsor
**Class:** OTHER
**Name:** Alliance for Clinical Trials in Oncology
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** RATIONALE: Drugs used in chemotherapy and hormone therapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining more than one drug may kill more cancer cells.
PURPOSE: Phase II trial to study the effectiveness of fludarabine plus octreotide in treating patients who have relapsed low-grade non-Hodgkin's lymphoma.
**Detailed Description:** OBJECTIVES: I. Determine the response rate and duration of response to fludarabine combined with octreotide and to octreotide alone in patients with relapsed indolent non-Hodgkin's lymphoma. II. Determine serum insulin-like growth factor-1 (IGF-1) and IGF-1 binding protein levels before and after treatment in this patient population. III. Determine somatostatin receptor subtypes in lymphoma biopsy samples from selected patients.
OUTLINE: Patients receive fludarabine IV over 10-30 minutes on days 1-5. Patients not currently receiving octreotide, receive a test dose of octreotide subcutaneously on day 1 during course 1 only and then receive octreotide intramuscularly monthly on day 1. Treatment repeats every 28 days for 4-6 courses. Patients then receive octreotide alone for 6-8 courses. Some patients may then receive another 12 courses of octreotide alone, for a total of 2 years of treatment. Patients are followed every 3 months for 5 years or until disease progression.
### Conditions Module
**Conditions:**
- Lymphoma
**Keywords:**
- Waldenström macroglobulinemia
- recurrent small lymphocytic lymphoma
- recurrent grade 1 follicular lymphoma
- recurrent grade 2 follicular lymphoma
- recurrent mantle cell lymphoma
- recurrent marginal zone lymphoma
- extranodal marginal zone B-cell lymphoma of mucosa-associated lymphoid tissue
- nodal marginal zone B-cell lymphoma
### Design Module
#### Design Info
**Allocation:** NA
**Intervention Model:** SINGLE_GROUP
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 34
**Type:** ACTUAL
**Phases:**
- PHASE2
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Patients receive fludarabine IV over 10-30 minutes on days 1-5. Patients not currently receiving octreotide, receive a test dose of octreotide subcutaneously on day 1 during course 1 only and then receive octreotide intramuscularly monthly on day 1. Treatment repeats every 28 days for 4-6 courses. Patients then receive octreotide alone for 6-8 courses. Some patients may then receive another 12 courses of octreotide alone, for a total of 2 years of treatment. Patients are followed every 3 months for 5 years or until disease progression.
**Intervention Names:**
- Drug: fludarabine phosphate
- Drug: octreotide acetate
**Label:** fludarabine + octreotide
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- fludarabine + octreotide
**Name:** fludarabine phosphate
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- fludarabine + octreotide
**Name:** octreotide acetate
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Measure:** response rate
**Time Frame:** Up to 5 years
### Eligibility Module
**Eligibility Criteria:** DISEASE CHARACTERISTICS: Histologically diagnosed indolent non-Hodgkin's lymphoma (NHL) of 1 of the following types: Diffuse small lymphocytic cell Follicular small cleaved cell Follicular mixed small and large cleaved cell Mantle cell lymphoma/leukemia (intermediate differentiated lymphoma) Preferentially treated on protocol NCCTG-958053 when available Monocytoid B-cell Mucosa-associated lymphoid tissue (MALT) Lymphoplasmacytic lymphoma (Waldenstrom's macroglobulinemia) Histology documented by lymph node (or other mass) or bone marrow biopsy within 6 months prior to entry Relapsed after cytotoxic chemotherapy regimens At least 1 measurable lesion by palpation, chest x-ray, CT, or MRI, e.g.: Lymph node at least 1.5 x 1.5 cm by palpation Spleen at least 3 cm below left costal margin The following exclude: CNS involvement by positive CSF cytology or CT/MRI B- or T-cell chronic lymphocytic leukemia Hairy cell leukemia Mycosis fungoides Aggressive lymphoma
PATIENT CHARACTERISTICS: Age: 18 and over Performance status: ECOG 0-2 Hematopoietic: Absolute neutrophil count at least 1,500/mm3 Platelet count at least 100,000/mm3 Hepatic: Total bilirubin no greater than 2 times normal OR Direct bilirubin no greater than 1.0 mg/dL above normal Renal: Creatinine no greater than 2.0 times normal Cardiovascular: No uncontrolled congestive heart failure No uncontrolled hypertension No uncontrolled angina pectoris Other: No uncontrolled or active infection No AIDS or HIV antibody No second malignancy within 5 years except: Carcinoma in situ of the cervix Resected nonmelanomatous skin cancer Prostate cancer in remission following radical retropubic prostatectomy or radiotherapy Not pregnant or nursing Negative pregnancy test Fertile patients must use effective contraception
PRIOR CONCURRENT THERAPY: Recovered from toxic effects of prior therapy Biologic therapy: See Disease Characteristics No concurrent interferon Chemotherapy: See Disease Characteristics No prior purine nucleoside analogues (e.g., fludarabine, pentostatin, or 2- chlorodeoxyadenosine) At least 3 weeks since prior chemotherapy (6 weeks since nitrosoureas) No other concurrent cytotoxic chemotherapy Endocrine therapy: No prior octreotide for lymphoma No concurrent corticosteriods except for Addison's disease Radiotherapy: Not specified Surgery: Not specified Other: No other concurrent investigational drugs
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Scottsdale
**Country:** United States
**Facility:** CCOP - Scottsdale Oncology Program
**State:** Arizona
**Zip:** 85259-5404
**Location 2:**
**City:** Peoria
**Country:** United States
**Facility:** CCOP - Illinois Oncology Research Association
**State:** Illinois
**Zip:** 61602
**Location 3:**
**City:** Urbana
**Country:** United States
**Facility:** CCOP - Carle Cancer Center
**State:** Illinois
**Zip:** 61801
**Location 4:**
**City:** Cedar Rapids
**Country:** United States
**Facility:** CCOP - Cedar Rapids Oncology Project
**State:** Iowa
**Zip:** 52403-1206
**Location 5:**
**City:** Des Moines
**Country:** United States
**Facility:** CCOP - Iowa Oncology Research Association
**State:** Iowa
**Zip:** 10309-1016
**Location 6:**
**City:** Sioux City
**Country:** United States
**Facility:** Siouxland Hematology-Oncology
**State:** Iowa
**Zip:** 51101-1733
**Location 7:**
**City:** Wichita
**Country:** United States
**Facility:** CCOP - Wichita
**State:** Kansas
**Zip:** 67214-3882
**Location 8:**
**City:** New Orleans
**Country:** United States
**Facility:** CCOP - Ochsner
**State:** Louisiana
**Zip:** 70121
**Location 9:**
**City:** Duluth
**Country:** United States
**Facility:** CCOP - Duluth
**State:** Minnesota
**Zip:** 55805
**Location 10:**
**City:** Rochester
**Country:** United States
**Facility:** Mayo Clinic Cancer Center
**State:** Minnesota
**Zip:** 55905
**Location 11:**
**City:** Saint Louis Park
**Country:** United States
**Facility:** CCOP - Metro-Minnesota
**State:** Minnesota
**Zip:** 55416
**Location 12:**
**City:** Omaha
**Country:** United States
**Facility:** CCOP - Missouri Valley Cancer Consortium
**State:** Nebraska
**Zip:** 68131
**Location 13:**
**City:** Bismarck
**Country:** United States
**Facility:** Quain & Ramstad Clinic, P.C.
**State:** North Dakota
**Zip:** 58501
**Location 14:**
**City:** Fargo
**Country:** United States
**Facility:** CCOP - Merit Care Hospital
**State:** North Dakota
**Zip:** 58122
**Location 15:**
**City:** Grand Forks
**Country:** United States
**Facility:** Altru Health Systems
**State:** North Dakota
**Zip:** 58201
**Location 16:**
**City:** Toledo
**Country:** United States
**Facility:** CCOP - Toledo Community Hospital Oncology Program
**State:** Ohio
**Zip:** 43623-3456
**Location 17:**
**City:** Danville
**Country:** United States
**Facility:** CCOP - Geisinger Clinical and Medical Center
**State:** Pennsylvania
**Zip:** 17822-2001
**Location 18:**
**City:** Rapid City
**Country:** United States
**Facility:** Rapid City Regional Hospital
**State:** South Dakota
**Zip:** 57709
**Location 19:**
**City:** Sioux Falls
**Country:** United States
**Facility:** CCOP - Sioux Community Cancer Consortium
**State:** South Dakota
**Zip:** 57105-1080
**Location 20:**
**City:** Regina
**Country:** Canada
**Facility:** Saskatchewan Cancer Agency
**State:** Saskatchewan
**Zip:** S4S 6X3
#### Overall Officials
**Official 1:**
**Affiliation:** Mayo Clinic
**Name:** Thomas E. Witzig, MD
**Role:** STUDY_CHAIR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000009370
- Term: Neoplasms by Histologic Type
- ID: D000009369
- Term: Neoplasms
- ID: D000008232
- Term: Lymphoproliferative Disorders
- ID: D000008206
- Term: Lymphatic Diseases
- ID: D000007160
- Term: Immunoproliferative Disorders
- ID: D000007154
- Term: Immune System Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: BC15
- Name: Blood and Lymph Conditions
- Abbrev: BC20
- Name: Immune System Diseases
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: All
- Name: All Conditions
- Abbrev: BC14
- Name: Heart and Blood Diseases
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M18116
- Name: Leukemia, Lymphocytic, Chronic, B-Cell
- Relevance: LOW
- As Found: Unknown
- ID: M11220
- Name: Lymphoma
- Relevance: HIGH
- As Found: Lymphoma
- ID: M22307
- Name: Lymphoma, Mantle-Cell
- Relevance: LOW
- As Found: Unknown
- ID: M11221
- Name: Lymphoma, Follicular
- Relevance: LOW
- As Found: Unknown
- ID: M20554
- Name: Lymphoma, B-Cell, Marginal Zone
- Relevance: LOW
- As Found: Unknown
- ID: M14850
- Name: Recurrence
- Relevance: LOW
- As Found: Unknown
- ID: M18828
- Name: Lymphoma, B-Cell
- Relevance: LOW
- As Found: Unknown
- ID: M11251
- Name: Waldenstrom Macroglobulinemia
- Relevance: LOW
- As Found: Unknown
- ID: M11222
- Name: Lymphoma, Non-Hodgkin
- Relevance: HIGH
- As Found: Non-Hodgkin Lymphoma
- ID: M12315
- Name: Neoplasms by Histologic Type
- Relevance: LOW
- As Found: Unknown
- ID: M11225
- Name: Lymphoproliferative Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M11203
- Name: Lymphatic Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M10206
- Name: Immunoproliferative Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M10200
- Name: Immune System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: T1308
- Name: Chronic Lymphocytic Leukemia
- Relevance: LOW
- As Found: Unknown
- ID: T3543
- Name: Lymphosarcoma
- Relevance: HIGH
- As Found: Lymphoma
- ID: T3601
- Name: Mantle Cell Lymphoma
- Relevance: LOW
- As Found: Unknown
- ID: T2361
- Name: Follicular Lymphoma
- Relevance: LOW
- As Found: Unknown
- ID: T640
- Name: B-cell Lymphoma
- Relevance: LOW
- As Found: Unknown
- ID: T3612
- Name: Marginal Zone Lymphoma
- Relevance: LOW
- As Found: Unknown
- ID: T5887
- Name: Waldenstrom Macroglobulinemia
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000008223
- Term: Lymphoma
- ID: D000008228
- Term: Lymphoma, Non-Hodgkin
### Intervention Browse Module - Ancestors
- ID: D000000970
- Term: Antineoplastic Agents
- ID: D000000964
- Term: Antimetabolites, Antineoplastic
- ID: D000000963
- Term: Antimetabolites
- ID: D000045504
- Term: Molecular Mechanisms of Pharmacological Action
- ID: D000007166
- Term: Immunosuppressive Agents
- ID: D000007155
- Term: Immunologic Factors
- ID: D000045505
- Term: Physiological Effects of Drugs
- ID: D000005765
- Term: Gastrointestinal Agents
- ID: D000018931
- Term: Antineoplastic Agents, Hormonal
### Intervention Browse Module - Browse Branches
- Abbrev: ANeo
- Name: Antineoplastic Agents
- Abbrev: All
- Name: All Drugs and Chemicals
- Abbrev: Gast
- Name: Gastrointestinal Agents
### Intervention Browse Module - Browse Leaves
- ID: M283230
- Name: Fludarabine
- Relevance: HIGH
- As Found: Comparison
- ID: M225513
- Name: Fludarabine phosphate
- Relevance: HIGH
- As Found: Intubation
- ID: M17982
- Name: Octreotide
- Relevance: HIGH
- As Found: Poorly
- ID: M4281
- Name: Antimetabolites
- Relevance: LOW
- As Found: Unknown
- ID: M10212
- Name: Immunosuppressive Agents
- Relevance: LOW
- As Found: Unknown
- ID: M10201
- Name: Immunologic Factors
- Relevance: LOW
- As Found: Unknown
- ID: M8881
- Name: Gastrointestinal Agents
- Relevance: LOW
- As Found: Unknown
- ID: M20966
- Name: Antineoplastic Agents, Hormonal
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: C000024352
- Term: Fludarabine
- ID: C000042382
- Term: Fludarabine phosphate
- ID: D000015282
- Term: Octreotide
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT00124579
**Brief Title:** S0417 Bortezomib, Thalidomide, and Dexamethasone in Treating Patients With Relapsed or Refractory Multiple Myeloma
**Official Title:** S0417 A Phase II Study of Bortezomib (Velcade™, PS-341), Thalidomide, and Dexamethasone in Patients With Refractory Multiple Myeloma
#### Organization Study ID Info
**ID:** S0417
#### Organization
**Class:** NETWORK
**Full Name:** SWOG Cancer Research Network
#### Secondary ID Infos
**Domain:** SWOG
**ID:** S0417
**Type:** OTHER
**ID:** U10CA032102
**Link:** https://reporter.nih.gov/quickSearch/U10CA032102
**Type:** NIH
### Status Module
#### Completion Date
**Date:** 2010-06
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2018-08-09
**Type:** ACTUAL
**Last Update Submit Date:** 2018-07-13
**Overall Status:** TERMINATED
#### Primary Completion Date
**Date:** 2008-06
**Type:** ACTUAL
#### Results First Post Date
**Date:** 2013-04-19
**Type:** ESTIMATED
**Results First Submit Date:** 2013-01-02
**Results First Submit QC Date:** 2013-03-08
#### Start Date
**Date:** 2005-08
**Status Verified Date:** 2018-07
#### Study First Post Date
**Date:** 2005-07-28
**Type:** ESTIMATED
**Study First Submit Date:** 2005-07-26
**Study First Submit QC Date:** 2005-07-26
**Why Stopped:** poor accrual
### Sponsor Collaborators Module
#### Collaborators
**Class:** NIH
**Name:** National Cancer Institute (NCI)
#### Lead Sponsor
**Class:** NETWORK
**Name:** SWOG Cancer Research Network
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** RATIONALE: Bortezomib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Biological therapies, such as thalidomide, may stimulate the immune system in different ways and stop cancer cells from growing. It may also stop the growth of cancer by blocking blood flow to the cancer. Drugs used in chemotherapy, such as dexamethasone, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. Giving bortezomib together with thalidomide and dexamethasone may kill more cancer cells.
PURPOSE: This phase II trial is studying how well giving bortezomib together with thalidomide and dexamethasone works in treating patients with relapsed or refractory multiple myeloma.
**Detailed Description:** OBJECTIVES:
* Determine the confirmed overall response rate (complete remission, remission, and partial remission) in patients with relapsed or refractory multiple myeloma treated with bortezomib, thalidomide, and dexamethasone.
* Determine overall and progression-free survival of patients treated with this regimen.
* Determine the qualitative and quantitative toxic effects of this regimen in these patients.
* Correlate, preliminarily, treatment with bortezomib with the activation of osteoblasts in these patients.
OUTLINE: This is a multicenter study.
* Induction therapy: Patients receive bortezomib IV on days 1, 4, 8, and 11, oral thalidomide once daily on days 1-21, and oral dexamethasone once daily on days 1, 2, 4, 5, 8, 9, 11, and 12. Treatment repeats every 21 days until achievement of confirmed complete remission (CR), remission (R), or partial remission (PR) OR for up to 8 courses in the absence of disease progression or unacceptable toxicity.
Patients achieving confirmed CR, R, or PR who reach a plateau prior to receiving the maximum 8 courses of induction therapy OR who achieve confirmed CR, R, or PR after receiving the maximum 8 courses of induction therapy proceed to maintenance therapy. Patients achieving stable disease after receiving the maximum 8 courses of induction therapy either proceed to maintenance therapy or receive further treatment with bortezomib, thalidomide, and dexamethasone off-study.
* Maintenance therapy: Patients receive oral dexamethasone on days 1-4. Courses repeat every 28 days for up to 3 years in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed within 30 days and then every 6 months for up to 5 years.
PROJECTED ACCRUAL: A total of 90 patients will be accrued for this study within 18 months.
### Conditions Module
**Conditions:**
- Multiple Myeloma
**Keywords:**
- stage I multiple myeloma
- stage II multiple myeloma
- stage III multiple myeloma
- refractory multiple myeloma
### Design Module
#### Design Info
**Allocation:** NA
**Intervention Model:** SINGLE_GROUP
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 7
**Type:** ACTUAL
**Phases:**
- PHASE2
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** bortezomib with thalidomide and dexamethasone
**Intervention Names:**
- Drug: bortezomib
- Drug: dexamethasone
- Drug: thalidomide
**Label:** bortezomib with thalidomide and dexamethasone
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- bortezomib with thalidomide and dexamethasone
**Description:** induction: 1 mg/m2 IV push days 1, 4, 8, 11 every 21 days
**Name:** bortezomib
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- bortezomib with thalidomide and dexamethasone
**Description:** induction: 20 mg/d PO days 1, 2, 4, 5, 8, 9, 11, 12 every 21 days maintenance: 40 mg days 1-4 every 28 days until progression
**Name:** dexamethasone
**Type:** DRUG
#### Intervention 3
**Arm Group Labels:**
- bortezomib with thalidomide and dexamethasone
**Description:** 100 mg/d PO days 1-21 every 21 days
**Name:** thalidomide
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Description:** Responses are defined as follows:
Complete Remission: Absence of bone marrow or blood findings of multiple myeloma. This includes disappearance of all evidence of serum and urine M-proteins on immunofixation electrophoresis studies. Normalization of serum concentrations of normal immunoglobulins is not required for CR. There must also be no evidence of increasing anemia. Bone marrow cellularity must be ≥ 20% with plasma cells ≤ 5%.
Remission: A ≥ 75% reduction in the serum M-protein, and if a urine M-protein (Bence-Jones protein) is present, either a ≥ 90% reduction in this protein, or a urine M-protein \< 0.2gm/day. Bone marrow plasma cells must be ≤ 5%.
Partial Remission: A ≥ 50% reduction in the serum M-protein, and if present, a ≥ 50% reduction in the urine M-protein (Bence-Jones protein). Bone marrow plasma cells must not be increased from baseline level.
**Measure:** Overall Response Rate Complete Remission (CR), Remission (R), and Partial Remission (PR).
**Time Frame:** 1 year
#### Secondary Outcomes
**Description:** To evaluate the qualitative and quantitative toxicities associated with this regimen.
**Measure:** Toxicity Evaluation
**Time Frame:** From date of protocol therapy start to date of protocol therapy end, i.e., up to about 3.5 years
**Description:** From date of initial registration to date of progression/relapse of disease (\> 25% increase from baseline in myeloma protein production or other signs of disease progression such as hypercalcemia, etc.) or death from any cause, whichever came first, up to 5 years
**Measure:** Progression-Free Survival
**Time Frame:** about 12-18 months
### Eligibility Module
**Eligibility Criteria:** DISEASE CHARACTERISTICS:
* Diagnosis of multiple myeloma (MM)
* Active disease
* Relapsed or refractory disease after ≥ 1 prior therapy for MM, that may have included autologous or allogeneic stem cell transplantation
* Relapse is defined as the occurrence of any of the following during or after prior treatment:
* Myeloma protein level increase by \> 100% from the lowest previously recorded level
* Myeloma protein level increase above the defined response criteria for partial remission
* Reappearance of any myeloma peak that had disappeared during the prior treatment
* Increase in the size and number of lytic bone lesions and/or focal lesions by x-ray, MRI, positron emission tomography, and/or CT scan
* Refractory disease is defined as no response (i.e., not achieving complete remission, remission, or partial remission) to prior therapy
* Measurable disease
* No evidence of POEMS (polyneuropathy, organomegaly, endocrinopathy, presence of M-protein, and skin changes) syndrome
* Must be registered on protocol SWOG-S0334
PATIENT CHARACTERISTICS:
Age
* 18 and over
Performance status
* Zubrod 0-2 (unless due to bone pain)
Life expectancy
* Not specified
Hematopoietic
* Absolute neutrophil count \> 1,000/mm\^3
* Platelet count \> 50,000/mm\^3
Hepatic
* AST or ALT ≤ 3 times upper limit of normal (ULN)
* Bilirubin ≤ 3 times ULN
Renal
* Creatinine clearance \> 30 mL/min
Cardiovascular
* No New York Heart Association class III or IV congestive heart failure
* No myocardial infarction within the past 6 months
* No poorly controlled hypertension
Other
* Not pregnant or nursing
* Negative pregnancy test
* Fertile female patients must use effective double method contraception for ≥ 4 weeks before, during, and for ≥ 4 weeks after completion of study treatment (during and for 4 weeks after completion of study treatment for male patients)
* No blood, ova, or sperm donation during study treatment
* No active infection requiring antibiotics
* No neurotoxicity ≥ grade 2
* No diabetes mellitus
* No other serious medical or psychiatric illness that would preclude study treatment
* No other malignancy within the past 3 years except adequately treated basal cell or squamous cell skin cancer or carcinoma in situ of the cervix
PRIOR CONCURRENT THERAPY:
Biologic therapy
* See Disease Characteristics
Chemotherapy
* At least 14 days since prior chemotherapy (28 days for nitrosoureas) and recovered
Endocrine therapy
* Not specified
Radiotherapy
* At least 14 days since prior radiotherapy and recovered
Surgery
* Not specified
Other
* No prior bortezomib alone or combined with thalidomide
* Concurrent participation on protocol SWOG-S0309 allowed
**Maximum Age:** 120 Years
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Little Rock
**Country:** United States
**Facility:** Arkansas Cancer Research Center at University of Arkansas for Medical Sciences
**State:** Arkansas
**Zip:** 72205
**Location 2:**
**City:** Alton
**Country:** United States
**Facility:** Saint Anthony's Hospital at Saint Anthony's Health Center
**State:** Illinois
**Zip:** 62002
**Location 3:**
**City:** Mount Vernon
**Country:** United States
**Facility:** Good Samaritan Regional Health Center
**State:** Illinois
**Zip:** 62864
**Location 4:**
**City:** Naperville
**Country:** United States
**Facility:** Hematology Oncology Consultants - Naperville
**State:** Illinois
**Zip:** 60540
**Location 5:**
**City:** Springfield
**Country:** United States
**Facility:** Regional Cancer Center at Memorial Medical Center
**State:** Illinois
**Zip:** 62781-0001
**Location 6:**
**City:** Beech Grove
**Country:** United States
**Facility:** St. Francis Hospital and Health Centers - Beech Grove Campus
**State:** Indiana
**Zip:** 46107
**Location 7:**
**City:** Richmond
**Country:** United States
**Facility:** Reid Hospital & Health Care Services, Incorporated
**State:** Indiana
**Zip:** 47374
**Location 8:**
**City:** Davenport
**Country:** United States
**Facility:** Genesis Regional Cancer Center at Genesis Medical Center
**State:** Iowa
**Zip:** 52803
**Location 9:**
**City:** Davenport
**Country:** United States
**Facility:** Genesis Medical Center - West Campus
**State:** Iowa
**Zip:** 52804
**Location 10:**
**City:** Salina
**Country:** United States
**Facility:** Tammy Walker Cancer Center at Salina Regional Health Center
**State:** Kansas
**Zip:** 67401
**Location 11:**
**City:** Wichita
**Country:** United States
**Facility:** Wesley Medical Center
**State:** Kansas
**Zip:** 67214
**Location 12:**
**City:** Ann Arbor
**Country:** United States
**Facility:** Saint Joseph Mercy Cancer Center
**State:** Michigan
**Zip:** 48106-0995
**Location 13:**
**City:** Ann Arbor
**Country:** United States
**Facility:** CCOP - Michigan Cancer Research Consortium
**State:** Michigan
**Zip:** 48106
**Location 14:**
**City:** Battle Creek
**Country:** United States
**Facility:** Battle Creek Health System Cancer Care Center
**State:** Michigan
**Zip:** 49017
**Location 15:**
**City:** Big Rapids
**Country:** United States
**Facility:** Mecosta County Medical Center
**State:** Michigan
**Zip:** 49307
**Location 16:**
**City:** Dearborn
**Country:** United States
**Facility:** Oakwood Cancer Center at Oakwood Hospital and Medical Center
**State:** Michigan
**Zip:** 48123-2500
**Location 17:**
**City:** Flint
**Country:** United States
**Facility:** Genesys Hurley Cancer Institute
**State:** Michigan
**Zip:** 48503
**Location 18:**
**City:** Flint
**Country:** United States
**Facility:** Hurley Medical Center
**State:** Michigan
**Zip:** 48503
**Location 19:**
**City:** Grand Rapids
**Country:** United States
**Facility:** CCOP - Grand Rapids
**State:** Michigan
**Zip:** 49503
**Location 20:**
**City:** Grand Rapids
**Country:** United States
**Facility:** Lacks Cancer Center at Saint Mary's Health Care
**State:** Michigan
**Zip:** 49503
**Location 21:**
**City:** Grand Rapids
**Country:** United States
**Facility:** Spectrum Health Hospital - Butterworth Campus
**State:** Michigan
**Zip:** 49503
**Location 22:**
**City:** Grand Rapids
**Country:** United States
**Facility:** Metro Health Hospital
**State:** Michigan
**Zip:** 49506
**Location 23:**
**City:** Grosse Pointe Woods
**Country:** United States
**Facility:** Van Elslander Cancer Center at St. John Hospital and Medical Center
**State:** Michigan
**Zip:** 48236
**Location 24:**
**City:** Holland
**Country:** United States
**Facility:** Holland Community Hospital
**State:** Michigan
**Zip:** 49423
**Location 25:**
**City:** Jackson
**Country:** United States
**Facility:** Foote Hospital
**State:** Michigan
**Zip:** 49201
**Location 26:**
**City:** Lansing
**Country:** United States
**Facility:** Sparrow Regional Cancer Center
**State:** Michigan
**Zip:** 48909
**Location 27:**
**City:** Muskegon
**Country:** United States
**Facility:** Hackley Hospital
**State:** Michigan
**Zip:** 49442
**Location 28:**
**City:** Port Huron
**Country:** United States
**Facility:** Mercy Regional Cancer Center at Mercy Hospital
**State:** Michigan
**Zip:** 48060
**Location 29:**
**City:** Saginaw
**Country:** United States
**Facility:** Seton Cancer Institute - Saginaw
**State:** Michigan
**Zip:** 48601
**Location 30:**
**City:** Southfield
**Country:** United States
**Facility:** Providence Cancer Institute at Providence Hospital - Southfield Campus
**State:** Michigan
**Zip:** 48075
**Location 31:**
**City:** Traverse City
**Country:** United States
**Facility:** Munson Medical Center
**State:** Michigan
**Zip:** 49684
**Location 32:**
**City:** Warren
**Country:** United States
**Facility:** St. John Macomb Hospital
**State:** Michigan
**Zip:** 48093
**Location 33:**
**City:** Cape Girardeau
**Country:** United States
**Facility:** Southeast Missouri Regional Cancer Center at Southeast Missouri Hospital
**State:** Missouri
**Zip:** 63701
**Location 34:**
**City:** Cape Girardeau
**Country:** United States
**Facility:** Saint Francis Medical Center
**State:** Missouri
**Zip:** 63703
**Location 35:**
**City:** Saint Louis
**Country:** United States
**Facility:** CCOP - St. Louis-Cape Girardeau
**State:** Missouri
**Zip:** 63141
**Location 36:**
**City:** Saint Louis
**Country:** United States
**Facility:** David C. Pratt Cancer Center at St. John's Mercy
**State:** Missouri
**Zip:** 63141
**Location 37:**
**City:** Springfield
**Country:** United States
**Facility:** CCOP - Cancer Research for the Ozarks
**State:** Missouri
**Zip:** 65802
**Location 38:**
**City:** Springfield
**Country:** United States
**Facility:** St. John's Regional Health Center
**State:** Missouri
**Zip:** 65804
**Location 39:**
**City:** Springfield
**Country:** United States
**Facility:** Hulston Cancer Center at Cox Medical Center South
**State:** Missouri
**Zip:** 65807
**Location 40:**
**City:** Billings
**Country:** United States
**Facility:** CCOP - Montana Cancer Consortium
**State:** Montana
**Zip:** 59101
**Location 41:**
**City:** Billings
**Country:** United States
**Facility:** Hematology-Oncology Centers of the Northern Rockies - Billings
**State:** Montana
**Zip:** 59101
**Location 42:**
**City:** Billings
**Country:** United States
**Facility:** Northern Rockies Radiation Oncology Center
**State:** Montana
**Zip:** 59101
**Location 43:**
**City:** Billings
**Country:** United States
**Facility:** St. Vincent Healthcare Cancer Care Services
**State:** Montana
**Zip:** 59101
**Location 44:**
**City:** Billings
**Country:** United States
**Facility:** Billings Clinic Cancer Center
**State:** Montana
**Zip:** 59107-5100
**Location 45:**
**City:** Billings
**Country:** United States
**Facility:** Billings Clinic - Downtown
**State:** Montana
**Zip:** 59107-7000
**Location 46:**
**City:** Bozeman
**Country:** United States
**Facility:** Bozeman Deaconess Cancer Center
**State:** Montana
**Zip:** 59715
**Location 47:**
**City:** Butte
**Country:** United States
**Facility:** St. James Healthcare Cancer Care
**State:** Montana
**Zip:** 59701
**Location 48:**
**City:** Great Falls
**Country:** United States
**Facility:** Big Sky Oncology
**State:** Montana
**Zip:** 59405
**Location 49:**
**City:** Great Falls
**Country:** United States
**Facility:** Great Falls Clinic - Main Facility
**State:** Montana
**Zip:** 59405
**Location 50:**
**City:** Great Falls
**Country:** United States
**Facility:** Sletten Regional Cancer Institute at Benefis Healthcare
**State:** Montana
**Zip:** 59405
**Location 51:**
**City:** Great Falls
**Country:** United States
**State:** Montana
**Zip:** 59405
**Location 52:**
**City:** Helena
**Country:** United States
**Facility:** St. Peter's Hospital
**State:** Montana
**Zip:** 59601
**Location 53:**
**City:** Kalispell
**Country:** United States
**Facility:** Glacier Oncology, PLLC
**State:** Montana
**Zip:** 59901
**Location 54:**
**City:** Kalispell
**Country:** United States
**Facility:** Kalispell Medical Oncology at KRMC
**State:** Montana
**Zip:** 59901
**Location 55:**
**City:** Kalispell
**Country:** United States
**Facility:** Kalispell Regional Medical Center
**State:** Montana
**Zip:** 59901
**Location 56:**
**City:** Missoula
**Country:** United States
**Facility:** Community Medical Center
**State:** Montana
**Zip:** 59801
**Location 57:**
**City:** Missoula
**Country:** United States
**Facility:** Guardian Oncology and Center for Wellness
**State:** Montana
**Zip:** 59804
**Location 58:**
**City:** Missoula
**Country:** United States
**Facility:** Montana Cancer Specialists at Montana Cancer Center
**State:** Montana
**Zip:** 59807-7877
**Location 59:**
**City:** Missoula
**Country:** United States
**Facility:** Montana Cancer Center at St. Patrick Hospital and Health Sciences Center
**State:** Montana
**Zip:** 59807
**Location 60:**
**City:** Kearney
**Country:** United States
**Facility:** Good Samaritan Cancer Center at Good Samaritan Hospital
**State:** Nebraska
**Zip:** 68848-1990
**Location 61:**
**City:** Middletown
**Country:** United States
**Facility:** Tucker Center for Cancer Care at Orange Regional Medical Center
**State:** New York
**Zip:** 10940-4199
**Location 62:**
**City:** Charlotte
**Country:** United States
**Facility:** Presbyterian Cancer Center at Presbyterian Hospital
**State:** North Carolina
**Zip:** 28233-3549
**Location 63:**
**City:** Goldsboro
**Country:** United States
**Facility:** Wayne Memorial Hospital, Incorporated
**State:** North Carolina
**Zip:** 27534
**Location 64:**
**City:** Hendersonville
**Country:** United States
**Facility:** Pardee Memorial Hospital
**State:** North Carolina
**Zip:** 28791
**Location 65:**
**City:** Rutherfordton
**Country:** United States
**Facility:** Rutherford Hospital
**State:** North Carolina
**Zip:** 28139
**Location 66:**
**City:** Cleveland
**Country:** United States
**Facility:** Cleveland Clinic Taussig Cancer Center
**State:** Ohio
**Zip:** 44195
**Location 67:**
**City:** Dayton
**Country:** United States
**Facility:** Grandview Hospital
**State:** Ohio
**Zip:** 45405
**Location 68:**
**City:** Dayton
**Country:** United States
**Facility:** Good Samaritan Hospital
**State:** Ohio
**Zip:** 45406
**Location 69:**
**City:** Dayton
**Country:** United States
**Facility:** David L. Rike Cancer Center at Miami Valley Hospital
**State:** Ohio
**Zip:** 45409
**Location 70:**
**City:** Dayton
**Country:** United States
**Facility:** Veterans Affairs Medical Center - Dayton
**State:** Ohio
**Zip:** 45428
**Location 71:**
**City:** Dayton
**Country:** United States
**Facility:** CCOP - Dayton
**State:** Ohio
**Zip:** 45429
**Location 72:**
**City:** Findlay
**Country:** United States
**Facility:** Blanchard Valley Medical Associates
**State:** Ohio
**Zip:** 45840
**Location 73:**
**City:** Independence
**Country:** United States
**Facility:** Community Oncology Group at Cleveland Clinic Cancer Center
**State:** Ohio
**Zip:** 44131
**Location 74:**
**City:** Kettering
**Country:** United States
**Facility:** Charles F. Kettering Memorial Hospital
**State:** Ohio
**Zip:** 45429
**Location 75:**
**City:** Middletown
**Country:** United States
**Facility:** Middletown Regional Hospital
**State:** Ohio
**Zip:** 45044
**Location 76:**
**City:** Troy
**Country:** United States
**Facility:** UVMC Cancer Care Center at Upper Valley Medical Center
**State:** Ohio
**Zip:** 45373-1300
**Location 77:**
**City:** Wooster
**Country:** United States
**Facility:** Cleveland Clinic - Wooster
**State:** Ohio
**Zip:** 44691
**Location 78:**
**City:** Wright-Patterson Air Force Base
**Country:** United States
**Facility:** United States Air Force Medical Center - Wright-Patterson
**State:** Ohio
**Zip:** 45433-5529
**Location 79:**
**City:** Xenia
**Country:** United States
**Facility:** Ruth G. McMillan Cancer Center at Greene Memorial Hospital
**State:** Ohio
**Zip:** 45385
**Location 80:**
**City:** Youngstown
**Country:** United States
**Facility:** Tod Children's Hospital
**State:** Ohio
**Zip:** 44501
**Location 81:**
**City:** Gresham
**Country:** United States
**Facility:** Legacy Mount Hood Medical Center
**State:** Oregon
**Zip:** 97030
**Location 82:**
**City:** Milwaukie
**Country:** United States
**Facility:** Providence Milwaukie Hospital
**State:** Oregon
**Zip:** 97222
**Location 83:**
**City:** Portland
**Country:** United States
**Facility:** Legacy Good Samaritan Hospital & Medical Center Comprehensive Cancer Center
**State:** Oregon
**Zip:** 97210
**Location 84:**
**City:** Portland
**Country:** United States
**Facility:** Providence Cancer Center at Providence Portland Medical Center
**State:** Oregon
**Zip:** 97213-2967
**Location 85:**
**City:** Portland
**Country:** United States
**Facility:** CCOP - Columbia River Oncology Program
**State:** Oregon
**Zip:** 97225
**Location 86:**
**City:** Portland
**Country:** United States
**Facility:** Providence St. Vincent Medical Center
**State:** Oregon
**Zip:** 97225
**Location 87:**
**City:** Portland
**Country:** United States
**Facility:** Legacy Emanuel Hospital and Health Center & Children's Hospital
**State:** Oregon
**Zip:** 97227
**Location 88:**
**City:** Tualatin
**Country:** United States
**Facility:** Legacy Meridian Park Hospital
**State:** Oregon
**Zip:** 97062
**Location 89:**
**City:** Anderson
**Country:** United States
**Facility:** AnMed Cancer Center
**State:** South Carolina
**Zip:** 29621
**Location 90:**
**City:** Spartanburg
**Country:** United States
**Facility:** CCOP - Upstate Carolina
**State:** South Carolina
**Zip:** 29303
**Location 91:**
**City:** Spartanburg
**Country:** United States
**Facility:** Gibbs Regional Cancer Center at Spartanburg Regional Medical Center
**State:** South Carolina
**Zip:** 29303
**Location 92:**
**City:** El Paso
**Country:** United States
**Facility:** Center for Cancer Medicine and Blood Disorders, PA
**State:** Texas
**Zip:** 79902
**Location 93:**
**City:** Fort Sam Houston
**Country:** United States
**Facility:** Brooke Army Medical Center
**State:** Texas
**Zip:** 78234
**Location 94:**
**City:** Houston
**Country:** United States
**Facility:** Baylor University Medical Center - Houston
**State:** Texas
**Zip:** 77030
**Location 95:**
**City:** Houston
**Country:** United States
**Facility:** Ben Taub General Hospital
**State:** Texas
**Zip:** 77030
**Location 96:**
**City:** Houston
**Country:** United States
**Facility:** Methodist Hospital
**State:** Texas
**Zip:** 77030
**Location 97:**
**City:** Houston
**Country:** United States
**Facility:** St. Luke's Texas Cancer Institute at St. Luke's Episcopal Hospital
**State:** Texas
**Zip:** 77030
**Location 98:**
**City:** Houston
**Country:** United States
**Facility:** Veterans Affairs Medical Center - Houston
**State:** Texas
**Zip:** 77030
**Location 99:**
**City:** Lackland Air Force Base
**Country:** United States
**Facility:** Wilford Hall Medical Center
**State:** Texas
**Zip:** 78236
**Location 100:**
**City:** Temple
**Country:** United States
**Facility:** CCOP - Scott and White Hospital
**State:** Texas
**Zip:** 76508
**Location 101:**
**City:** American Fork
**Country:** United States
**Facility:** American Fork Hospital
**State:** Utah
**Zip:** 84003
**Location 102:**
**City:** Cedar City
**Country:** United States
**Facility:** Sandra L. Maxwell Cancer Center
**State:** Utah
**Zip:** 84720
**Location 103:**
**City:** Logan
**Country:** United States
**Facility:** Logan Regional Hospital
**State:** Utah
**Zip:** 84321
**Location 104:**
**City:** Murray
**Country:** United States
**Facility:** Cottonwood Hospital Medical Center
**State:** Utah
**Zip:** 84107
**Location 105:**
**City:** Murray
**Country:** United States
**Facility:** Jon and Karen Huntsman Cancer Center at Intermountain Medical Center
**State:** Utah
**Zip:** 84157
**Location 106:**
**City:** Ogden
**Country:** United States
**Facility:** McKay-Dee Hospital Center
**State:** Utah
**Zip:** 84403
**Location 107:**
**City:** Provo
**Country:** United States
**Facility:** Utah Valley Regional Medical Center - Provo
**State:** Utah
**Zip:** 84604
**Location 108:**
**City:** Saint George
**Country:** United States
**Facility:** Dixie Regional Medical Center - East Campus
**State:** Utah
**Zip:** 84770
**Location 109:**
**City:** Salt Lake City
**Country:** United States
**Facility:** Latter Day Saints Hospital
**State:** Utah
**Zip:** 84103
**Location 110:**
**City:** Salt Lake City
**Country:** United States
**Facility:** Utah Cancer Specialists at UCS Cancer Center
**State:** Utah
**Zip:** 84106
**Location 111:**
**City:** Danville
**Country:** United States
**Facility:** Danville Regional Medical Center
**State:** Virginia
**Zip:** 24541
**Location 112:**
**City:** Martinsville
**Country:** United States
**Facility:** Ravenel Oncology Center at Memorial Hospital of Martinsville and Henry County
**State:** Virginia
**Zip:** 24115
**Location 113:**
**City:** Bellingham
**Country:** United States
**Facility:** St. Joseph Cancer Center
**State:** Washington
**Zip:** 98225
**Location 114:**
**City:** Bremerton
**Country:** United States
**Facility:** Olympic Hematology and Oncology
**State:** Washington
**Zip:** 98310
**Location 115:**
**City:** Mount Vernon
**Country:** United States
**Facility:** Skagit Valley Hospital Cancer Care Center
**State:** Washington
**Zip:** 98273
**Location 116:**
**City:** Seattle
**Country:** United States
**Facility:** Fred Hutchinson Cancer Research Center
**State:** Washington
**Zip:** 98104
**Location 117:**
**City:** Seattle
**Country:** United States
**Facility:** Harborview Medical Center
**State:** Washington
**Zip:** 98104
**Location 118:**
**City:** Seattle
**Country:** United States
**Facility:** Minor and James Medical, PLLC
**State:** Washington
**Zip:** 98104
**Location 119:**
**City:** Seattle
**Country:** United States
**Facility:** Group Health Central Hospital
**State:** Washington
**Zip:** 98112
**Location 120:**
**City:** Seattle
**Country:** United States
**Facility:** Swedish Cancer Institute at Swedish Medical Center - First Hill Campus
**State:** Washington
**Zip:** 98122-4307
**Location 121:**
**City:** Seattle
**Country:** United States
**Facility:** Polyclinic First Hill
**State:** Washington
**Zip:** 98122
**Location 122:**
**City:** Seattle
**Country:** United States
**Facility:** University Cancer Center at University of Washington Medical Center
**State:** Washington
**Zip:** 98195-6043
**Location 123:**
**City:** Sedro-Woolley
**Country:** United States
**Facility:** North Puget Oncology at United General Hospital
**State:** Washington
**Zip:** 98284
**Location 124:**
**City:** Spokane
**Country:** United States
**Facility:** Cancer Care Northwest - Spokane South
**State:** Washington
**Zip:** 99202
**Location 125:**
**City:** Vancouver
**Country:** United States
**Facility:** Southwest Washington Medical Center Cancer Center
**State:** Washington
**Zip:** 98668
**Location 126:**
**City:** Wenatchee
**Country:** United States
**Facility:** Wenatchee Valley Medical Center
**State:** Washington
**Zip:** 98801-2028
**Location 127:**
**City:** Sheridan
**Country:** United States
**Facility:** Welch Cancer Center at Sheridan Memorial Hospital
**State:** Wyoming
**Zip:** 82801
#### Overall Officials
**Official 1:**
**Affiliation:** Sparrow Regional Cancer Center
**Name:** Gordan Srkalovic, MD, PhD
**Role:** PRINCIPAL_INVESTIGATOR
**Official 2:**
**Affiliation:** The Cleveland Clinic
**Name:** Mohamad A. Hussein, MD
**Role:** PRINCIPAL_INVESTIGATOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000009370
- Term: Neoplasms by Histologic Type
- ID: D000009369
- Term: Neoplasms
- ID: D000020141
- Term: Hemostatic Disorders
- ID: D000014652
- Term: Vascular Diseases
- ID: D000002318
- Term: Cardiovascular Diseases
- ID: D000010265
- Term: Paraproteinemias
- ID: D000001796
- Term: Blood Protein Disorders
- ID: D000006402
- Term: Hematologic Diseases
- ID: D000006474
- Term: Hemorrhagic Disorders
- ID: D000008232
- Term: Lymphoproliferative Disorders
- ID: D000007160
- Term: Immunoproliferative Disorders
- ID: D000007154
- Term: Immune System Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: BC14
- Name: Heart and Blood Diseases
- Abbrev: BC15
- Name: Blood and Lymph Conditions
- Abbrev: BC20
- Name: Immune System Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M12058
- Name: Multiple Myeloma
- Relevance: HIGH
- As Found: Multiple Myeloma
- ID: M27588
- Name: Neoplasms, Plasma Cell
- Relevance: HIGH
- As Found: Multiple Myeloma
- ID: M12315
- Name: Neoplasms by Histologic Type
- Relevance: LOW
- As Found: Unknown
- ID: M21977
- Name: Hemostatic Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M5059
- Name: Blood Coagulation Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M17400
- Name: Vascular Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M13178
- Name: Paraproteinemias
- Relevance: LOW
- As Found: Unknown
- ID: M5077
- Name: Blood Protein Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M9490
- Name: Hematologic Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M9560
- Name: Hemorrhagic Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M11225
- Name: Lymphoproliferative Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M10206
- Name: Immunoproliferative Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M10200
- Name: Immune System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: T3947
- Name: Multiple Myeloma
- Relevance: HIGH
- As Found: Multiple Myeloma
### Condition Browse Module - Meshes
- ID: D000009101
- Term: Multiple Myeloma
- ID: D000054219
- Term: Neoplasms, Plasma Cell
### Intervention Browse Module - Ancestors
- ID: D000000893
- Term: Anti-Inflammatory Agents
- ID: D000000932
- Term: Antiemetics
- ID: D000001337
- Term: Autonomic Agents
- ID: D000018373
- Term: Peripheral Nervous System Agents
- ID: D000045505
- Term: Physiological Effects of Drugs
- ID: D000005765
- Term: Gastrointestinal Agents
- ID: D000005938
- Term: Glucocorticoids
- ID: D000006728
- Term: Hormones
- ID: D000006730
- Term: Hormones, Hormone Substitutes, and Hormone Antagonists
- ID: D000018931
- Term: Antineoplastic Agents, Hormonal
- ID: D000000970
- Term: Antineoplastic Agents
- ID: D000007166
- Term: Immunosuppressive Agents
- ID: D000007155
- Term: Immunologic Factors
- ID: D000007917
- Term: Leprostatic Agents
- ID: D000000900
- Term: Anti-Bacterial Agents
- ID: D000000890
- Term: Anti-Infective Agents
- ID: D000020533
- Term: Angiogenesis Inhibitors
- ID: D000043924
- Term: Angiogenesis Modulating Agents
- ID: D000006133
- Term: Growth Substances
- ID: D000006131
- Term: Growth Inhibitors
### Intervention Browse Module - Browse Branches
- Abbrev: Infl
- Name: Anti-Inflammatory Agents
- Abbrev: ANeo
- Name: Antineoplastic Agents
- Abbrev: AnEm
- Name: Antiemetics
- Abbrev: Gast
- Name: Gastrointestinal Agents
- Abbrev: All
- Name: All Drugs and Chemicals
- Abbrev: Infe
- Name: Anti-Infective Agents
### Intervention Browse Module - Browse Leaves
- ID: M7102
- Name: Dexamethasone
- Relevance: HIGH
- As Found: Children
- ID: M376
- Name: Bortezomib
- Relevance: HIGH
- As Found: 14 days
- ID: M235549
- Name: Dexamethasone acetate
- Relevance: LOW
- As Found: Unknown
- ID: M16559
- Name: Thalidomide
- Relevance: HIGH
- As Found: List
- ID: M4217
- Name: Anti-Inflammatory Agents
- Relevance: LOW
- As Found: Unknown
- ID: M4251
- Name: Antiemetics
- Relevance: LOW
- As Found: Unknown
- ID: M8881
- Name: Gastrointestinal Agents
- Relevance: LOW
- As Found: Unknown
- ID: M9047
- Name: Glucocorticoids
- Relevance: LOW
- As Found: Unknown
- ID: M9789
- Name: Hormones
- Relevance: LOW
- As Found: Unknown
- ID: M9788
- Name: Hormone Antagonists
- Relevance: LOW
- As Found: Unknown
- ID: M20966
- Name: Antineoplastic Agents, Hormonal
- Relevance: LOW
- As Found: Unknown
- ID: M10212
- Name: Immunosuppressive Agents
- Relevance: LOW
- As Found: Unknown
- ID: M10201
- Name: Immunologic Factors
- Relevance: LOW
- As Found: Unknown
- ID: M4222
- Name: Anti-Bacterial Agents
- Relevance: LOW
- As Found: Unknown
- ID: M4214
- Name: Anti-Infective Agents
- Relevance: LOW
- As Found: Unknown
- ID: M22318
- Name: Angiogenesis Inhibitors
- Relevance: LOW
- As Found: Unknown
- ID: M9231
- Name: Growth Inhibitors
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: D000013792
- Term: Thalidomide
- ID: D000003907
- Term: Dexamethasone
- ID: D000069286
- Term: Bortezomib
### Misc Info Module
- Version Holder: 2024-05-24
## Results Section
### Adverse Events Module
#### Event Groups
**Group ID:** EG000
**Title:** Bortezomib + Thal/Dex
**Description:** Induction (per 21-day cycle): Bortezomib - 1.0 mg/m2 (Days 1, 4, 8, and 11). Dexamethasone - 20 mg/d PO (Days 1, 2, 4, 5, 8, 9, 11, and 12). Thalidomide 100 mg (Days 1-21).
Maintenance (per 28-day cycle): thalidomide 100mg (Days 1-28). Dexamethasone 40 mg Days 1-4.
**ID:** EG000
**Other Num Affected:** 7
**Other Num at Risk:** 7
**Serious Number Affected:** 1
**Serious Number At Risk:** 7
**Title:** Bortezomib + Thal/Dex
**Frequency Threshold:** 5
#### Other Events
**Term:** Hemoglobin
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Blood and lymphatic system disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** SVT and nodal arrhythmia - Atrial fibrillation
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Cardiac disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** SVT and nodal arrhythmia - Sinus bradycardia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Cardiac disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Cushingoid appearance
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Endocrine disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Night blindness (nyctalopia)
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Vision-blurred vision
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Watery eye (epiphora, tearing)
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Constipation
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Diarrhea
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Distention/bloating, abdominal
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Flatulence
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Heartburn/dyspepsia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Nausea
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Pain - Abdomen NOS
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Vomiting
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Edema: head and neck
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Edema: limb
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Extremity-lower (gait/walking)
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Fatigue (asthenia, lethargy, malaise)
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Fever in absence of neutropenia, ANC lt1.0x10e9/L
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Pain-Other
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Rigors/chills
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Allergy/Immunology-Other
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Immune system disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Inf (clin/microbio) w/Gr 3-4 neuts - Bladder
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** CTCAE (3.0)
**Term:** Inf (clin/microbio) w/Gr 3-4 neuts - Lung
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** CTCAE (3.0)
**Term:** Inf w/normal ANC or Gr 1-2 neutrophils - Skin
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** CTCAE (3.0)
**Term:** Inf w/normal ANC or Gr 1-2 neutrophils - Soft tiss
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** CTCAE (3.0)
**Term:** Inf w/normal ANC or Gr 1-2 neutrophils - Up airway
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** CTCAE (3.0)
**Term:** Infection with unknown ANC - Upper airway NOS
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** CTCAE (3.0)
**Term:** Infection-Other
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** CTCAE (3.0)
**Term:** ALT, SGPT (serum glutamic pyruvic transaminase)
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** CTCAE (3.0)
**Term:** AST, SGOT
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** CTCAE (3.0)
**Term:** Alkaline phosphatase
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** CTCAE (3.0)
**Term:** Bilirubin (hyperbilirubinemia)
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** CTCAE (3.0)
**Term:** Creatinine
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** CTCAE (3.0)
**Term:** Leukocytes (total WBC)
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** CTCAE (3.0)
**Term:** Lymphopenia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** CTCAE (3.0)
**Term:** Neutrophils/granulocytes (ANC/AGC)
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** CTCAE (3.0)
**Term:** Platelets
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** CTCAE (3.0)
**Term:** Weight gain
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** CTCAE (3.0)
**Term:** Weight loss
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** CTCAE (3.0)
**Term:** Albumin, serum-low (hypoalbuminemia)
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Anorexia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Calcium, serum-high (hypercalcemia)
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Calcium, serum-low (hypocalcemia)
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Dehydration
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Glucose, serum-high (hyperglycemia)
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Glucose, serum-low (hypoglycemia)
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Magnesium, serum-low (hypomagnesemia)
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Potassium, serum-low (hypokalemia)
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Sodium, serum-low (hyponatremia)
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Muscle weakness, not d/t neuropathy - Extrem-lower
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Muscle weakness, not d/t neuropathy - body/general
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Pain - Back
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Pain - Bone
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Pain - Extremity-limb
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Pain - Joint
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Pain - Neck
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Pain - Tumor pain
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps)
**Source Vocabulary:** CTCAE (3.0)
**Term:** Dizziness
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Neurology-Other
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Neuropathy: motor
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Neuropathy: sensory
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Pain - Head/headache
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Tremor
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Confusion
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Psychiatric disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Insomnia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Psychiatric disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Mood alteration - depression
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Psychiatric disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Glomerular filtration rate
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Renal and urinary disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Proteinuria
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Renal and urinary disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Sexual/Reproductive Function-Other (Specify)
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Reproductive system and breast disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Allergic rhinitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Bronchospasm, wheezing
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Cough
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Dyspnea (shortness of breath)
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Hemorrhage, Respiratory tract NOS
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Hemorrhage, pulmonary/upper respiratory - Nose
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Pulmonary/Upper Respiratory-Other
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Dermatology/Skin-Other
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Dry skin
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Rash/desquamation
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Rash: erythema multiforme
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Skin breakdown/decubitus ulcer
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** CTCAE (3.0)
**Term:** Hypotension
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Vascular disorders
**Source Vocabulary:** CTCAE (3.0)
#### Serious Events
**Term:** Edema: limb
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** CTCAE (3.0)
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 7
**Term:** Bilirubin (hyperbilirubinemia)
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** CTCAE (3.0)
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 7
**Term:** Platelets
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** CTCAE (3.0)
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 7
**Term:** Death - Disease progression NOS
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps)
**Source Vocabulary:** CTCAE (3.0)
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 7
**Term:** Dyspnea (shortness of breath)
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** CTCAE (3.0)
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 7
## Results Section - Baseline Characteristics Module
### Denomination Counts
**Group ID:** BG000
**Value:** 7
**Units:** Participants
### Group
**ID:** BG000
**Title:** Bortezomib With Thalidomide and Dexamethasone
**Description:** Induction (per 21-day cycle): Bortezomib - 1.0 mg/m2 (Days 1, 4, 8, and 11). Dexamethasone - 20 mg/d PO (Days 1, 2, 4, 5, 8, 9, 11, and 12). Thalidomide 100 mg (Days 1-21).
Maintenance (per 28-day cycle): thalidomide 100mg (Days 1-28). Dexamethasone 40 mg Days 1-4.
### Measure
#### Measurement
**Group ID:** BG000
**Lower Limit:** 37.6
**Upper Limit:** 81.4
**Value:** 63.7
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 4
**Category Title:** Female
#### Measurement
**Group ID:** BG000
**Value:** 3
**Category Title:** Male
**Class Title:**
**Denomination Units Selected:**
## Results Section - Baseline Characteristics Module
### Measure 1
**Dispersion Type:** FULL_RANGE
**Parameter Type:** MEDIAN
**Title:** Age, Continuous
**Unit of Measure:** years
### Measure 2
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Sex: Female, Male
**Unit of Measure:** Participants
## Results Section - More Information Module
### Certain Agreement
### Point of Contact
**Email:** [email protected]
**Organization:** SWOG
**Phone:** (713) 792-2860
**Title:** Robert Z. Orlowski, MD, PhD
## Results Section - Outcome Measures Module
### Outcome Measure 1
### Outcome Measure 2
### Outcome Measure 3
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 2
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
#### Outcome Measure 2
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 14
**Title:**
#### Outcome Measure 3
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:** 4
- **Spread:**
- **Upper Limit:** 12
- **Value:** 8
**Title:**
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Description:** To evaluate the qualitative and quantitative toxicities associated with this regimen.
**Parameter Type:** NUMBER
**Population Description:** All participants receiving at least one dose of induction therapy
**Reporting Status:** POSTED
**Time Frame:** From date of protocol therapy start to date of protocol therapy end, i.e., up to about 3.5 years
**Title:** Toxicity Evaluation
**Type:** SECONDARY
**Unit of Measure:** Participants
##### Group
**Description:** Induction (per 21-day cycle): Bortezomib - 1.0 mg/m2 (Days 1, 4, 8, and 11). Dexamethasone - 20 mg/d PO (Days 1, 2, 4, 5, 8, 9, 11, and 12). Thalidomide 100 mg (Days 1-21).
Maintenance (per 28-day cycle): thalidomide 100mg (Days 1-28). Dexamethasone 40 mg Days 1-4.
**ID:** OG000
**Title:** Bortezomib + Thal/Dex
#### Outcome Measure 2
**Description:** Responses are defined as follows:
Complete Remission: Absence of bone marrow or blood findings of multiple myeloma. This includes disappearance of all evidence of serum and urine M-proteins on immunofixation electrophoresis studies. Normalization of serum concentrations of normal immunoglobulins is not required for CR. There must also be no evidence of increasing anemia. Bone marrow cellularity must be ≥ 20% with plasma cells ≤ 5%.
Remission: A ≥ 75% reduction in the serum M-protein, and if a urine M-protein (Bence-Jones protein) is present, either a ≥ 90% reduction in this protein, or a urine M-protein \< 0.2gm/day. Bone marrow plasma cells must be ≤ 5%.
Partial Remission: A ≥ 50% reduction in the serum M-protein, and if present, a ≥ 50% reduction in the urine M-protein (Bence-Jones protein). Bone marrow plasma cells must not be increased from baseline level.
**Parameter Type:** NUMBER
**Population Description:** Ninety patients is sufficient to distinguish between the null hypothesis that the response rate is 45% versus the alternative of a response rate of 60% with 89% power, using a one-sided test based on the binomial distribution with a significance level of 5%.
**Reporting Status:** POSTED
**Time Frame:** 1 year
**Title:** Overall Response Rate Complete Remission (CR), Remission (R), and Partial Remission (PR).
**Type:** PRIMARY
**Unit of Measure:** percentage of participants
##### Group
**Description:** Induction (per 21-day cycle): Bortezomib - 1.0 mg/m2 (Days 1, 4, 8, and 11). Dexamethasone - 20 mg/d PO (Days 1, 2, 4, 5, 8, 9, 11, and 12). Thalidomide 100 mg (Days 1-21).
Maintenance (per 28-day cycle): thalidomide 100mg (Days 1-28). Dexamethasone 40 mg Days 1-4.
**ID:** OG000
**Title:** Bortezomib With Thalidomide and Dexamethasone
#### Outcome Measure 3
**Description:** From date of initial registration to date of progression/relapse of disease (\> 25% increase from baseline in myeloma protein production or other signs of disease progression such as hypercalcemia, etc.) or death from any cause, whichever came first, up to 5 years
**Dispersion Type:** 95% Confidence Interval
**Parameter Type:** MEDIAN
**Population Description:** With ninety patients, we will have 82% power to rule out a null hypothesis of a 12-month median survival versus an alternative hypothesis of an 18-month median survival at a significance level of 5%.
**Reporting Status:** POSTED
**Time Frame:** about 12-18 months
**Title:** Progression-Free Survival
**Type:** SECONDARY
**Unit of Measure:** Months
##### Group
**Description:** Induction (per 21-day cycle): Bortezomib - 1.0 mg/m2 (Days 1, 4, 8, and 11). Dexamethasone - 20 mg/d PO (Days 1, 2, 4, 5, 8, 9, 11, and 12). Thalidomide 100 mg (Days 1-21).
Maintenance (per 28-day cycle): thalidomide 100mg (Days 1-28). Dexamethasone 40 mg Days 1-4.
**ID:** OG000
**Title:** Bortezomib With Thalidomide and Dexamethasone
### Participant Flow Module
#### Group
**Description:** Induction (per 21-day cycle): Bortezomib - 1.0 mg/m2 (Days 1, 4, 8, and 11). Dexamethasone - 20 mg/d PO (Days 1, 2, 4, 5, 8, 9, 11, and 12). Thalidomide 100 mg (Days 1-21).
Maintenance (per 28-day cycle): thalidomide 100mg (Days 1-28). Dexamethasone 40 mg Days 1-4.
**ID:** FG000
**Title:** Bortezomib With Thalidomide and Dexamethasone Induction
#### Period
**Title:** Induction
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 7
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 2
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 5
#### Period
**Title:** Maintenance
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 2
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 0
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 2
**Has Results:** True |
## Protocol Section
### Identification Module
**NCT ID:** NCT06226779
**Brief Title:** Baduanjin Versus Brisk Walking for Cognition in Schizophrenia
**Official Title:** Mindful Exercise Baduanjin Versus Brisk Walking for Cognitive Function in Patients With Schizophrenia
#### Organization Study ID Info
**ID:** 202400006B0
#### Organization
**Class:** OTHER
**Full Name:** Chang Gung Memorial Hospital
### Status Module
#### Completion Date
**Date:** 2025-02-15
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-01-26
**Type:** ACTUAL
**Last Update Submit Date:** 2024-01-18
**Overall Status:** RECRUITING
#### Primary Completion Date
**Date:** 2024-05-15
**Type:** ESTIMATED
#### Start Date
**Date:** 2024-01-16
**Type:** ACTUAL
**Status Verified Date:** 2024-01
#### Study First Post Date
**Date:** 2024-01-26
**Type:** ACTUAL
**Study First Submit Date:** 2024-01-18
**Study First Submit QC Date:** 2024-01-18
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Chang Gung Memorial Hospital
#### Responsible Party
**Investigator Affiliation:** Chang Gung Memorial Hospital
**Investigator Full Name:** Chen Chyi-Rong
**Investigator Title:** Occupational therapist
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** The current study intends to recruit participants with schizophrenia for the practice of Baduanjin, brisk walking, and health education after enrollment. The study also including a maintenance program. Cognitive and physical function assessments will be conducted before, after, and during follow-up tests. The research hypothesis posits that both Baduanjin and brisk walking will confer beneficial effects on various aspects of cognitive and physical functions.
**Detailed Description:** Cognitive function deficits are core features in patients with schizophrenia. These deficits need active intervention to prevent functional decline. Baduanjin and brisk walking show promise as interventions in patients with schizophrenia. This study investigated the effects of Baduanjin exercise versus brisk walking in patients with schizophrenia.
This study will be a single-blind, 3-arm, parallel, randomized controlled trial.
A total of 120 patients with schizophrenia will be enrolled and randomly assigned to the Baduanjin group, brisk walking group or control group. The intervention group will practice Badunjin exercise. While participants in brisk walking group will receive brisk walking at the same duration. The controls will watch videos that are not aimed at improving physical exertion. Maintenance programs will also be conducted for participants to encourage practicing in exercise after intervention. All group will undergo evaluation at three time points: baseline, immediately after intervention and 4-week follow up after intervention. The primary outcome will be cognitive function and physical function. Secondary outcomes will include mood, sleep quality, and quality of live.
### Conditions Module
**Conditions:**
- Schizophrenia
- Exercise
- Condition
**Keywords:**
- schizophrenia, mindful exercise, exercise, cognition
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** SINGLE
**Who Masked:**
- INVESTIGATOR
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 120
**Type:** ESTIMATED
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Parcipants will practice baduanjin exercise.
**Intervention Names:**
- Behavioral: Baduanjin exercsie
**Label:** Baduanjin
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Parcipants will perform brisk walking activities.
**Intervention Names:**
- Behavioral: Brisk walking
**Label:** Brisk walking
**Type:** ACTIVE_COMPARATOR
#### Arm Group 3
**Description:** Parcipants will receive health education classes and watch sport related videos.
**Intervention Names:**
- Behavioral: Health education
**Label:** health education
**Type:** OTHER
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Baduanjin
**Description:** The Baduanjin exercise consists of 8 postures, with emphasis on different body parts. The exercise is commonly practiced as follows: (1) 2 hands holding up the heavens, (2) drawing the bow to shoot the eagle, (3) separating the heaven and earth, (4) wise owl gazing backwards, (5) swaying the head and shaking the tail, (6) 2 hands holding the feet to strengthen the waist, (7) clenching the fists and glaring fiercely, and (8) bouncing on the toes.
**Name:** Baduanjin exercsie
**Other Names:**
- Mindful baduanjin
**Type:** BEHAVIORAL
#### Intervention 2
**Arm Group Labels:**
- Brisk walking
**Description:** The brisk walking will be gradually increased physical exertion from light to moderate of intensity. During the training period, the heart rate of participants will be monitored.
**Name:** Brisk walking
**Other Names:**
- aerobic walking
**Type:** BEHAVIORAL
#### Intervention 3
**Arm Group Labels:**
- health education
**Description:** Health education will include lecture classes. Video watching will watch videos which were not aim at improving physical exertion, such as sports games or sports related variety shows
**Name:** Health education
**Other Names:**
- video watching
**Type:** BEHAVIORAL
### Outcomes Module
#### Primary Outcomes
**Description:** BACS is a battery of cognition tests that measure verbal memory, working memory, motor speed, verbal fluency, attention and processing speed, and executive function.The BACS is comprised of seven subtests: (1) the List Learning Test, (2) Digit Sequencing Test, (3) Token Motor Test, (4) Category Instance Test, (5) Controlled Oral Word Association Test, (6) Symbol Coding, and (7) Tower of London Test.
**Measure:** Change score of the Brief Assessment of Cognition in Schizophrenia (BACS)
**Time Frame:** Baseline, immediate after treatment, 4-week follow up
**Description:** This test evaluates cardiovascular fitness and walking speed.
**Measure:** Change scores of 6-minute walk test
**Time Frame:** Baseline, immediate after treatment, 4-week follow up
**Description:** This test evaluates muscular endurance of lower-extremities.
**Measure:** Change scores of 30-second chair stand test
**Time Frame:** Baseline, immediate after treatment, 4-week follow up
**Description:** This test evaluates functional mobility, agility and balance
**Measure:** Change scores of Timed up-and-go test
**Time Frame:** Baseline, immediate after treatment, 4-week follow up
**Description:** This test requires carry a cup of water 3 cm from the top of the cup while performing the Timed up-and-go test. It evaluates dual task performance.
**Measure:** Change scores of dual task Timed up-and-go test (manual)
**Time Frame:** Baseline, immediate after treatment, 4-week follow up
**Description:** This test combine serial three counting and Timed up-and-go test simultaneously. It evaluates dual task performance.
**Measure:** Change scores of dual task Timed up-and-go test (cognitive)
**Time Frame:** Baseline, immediate after treatment, 4-week follow up
#### Secondary Outcomes
**Description:** DASS-21 is a self-report questionnaire that measure symptoms of depression, anxiety, and stress.
**Measure:** Change score of Depression, Anxiety Stress Scales-21 (DASS-21)
**Time Frame:** Baseline, immediate after treatment, 4-week follow up
**Description:** PSQI is a self-report assessment which contains 19 items to measure sleep quality
**Measure:** Change score of Pittsburg Sleep Quality Index
**Time Frame:** Baseline, immediate after treatment, 4-week follow up
**Description:** EQ-5D is a self-reporting questionnaire that measures five dimensions of health state: (1) mobility, (2) self-care, (3)usual activities, (4) pain/discomfort, and (5) anxiety/depression.
**Measure:** Change score of Euro-Qol 5-Dimmensions (EQ-5D).
**Time Frame:** Baseline, immediate after treatment, 4-week follow up
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
1. Having a diagnosis of schizophrenia according to the DSM-5.
2. Who will be above 20-65 years of age .
3. Having a stable mental status without shift in medication and keep in same dose for at least one month.
4. Being able to walk independently for 50 meters.
Exclusion Criteria:
1. Serious physical conditions that will impede participation, such as cardiovascular disease, musculoskeletal disease or pulmonary system disease.
2. Visual or auditory impairment that precludes completion of assessment.
3. Acute psychosis requiring hospitalization.
4. Presence of severe withdrawal or profound cognitive disability that cause difficulties in following instructions.
**Gender Based:** True
**Gender Description:** Based on self-representation of gender identity.
**Maximum Age:** 65 Years
**Minimum Age:** 20 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Chyi-Rong Chen, Master
**Phone:** 886-7-7317123
**Phone Ext:** 8755
**Role:** CONTACT
**Contact 2:**
**Email:** [email protected]
**Name:** Keh-chung Lin, Doctor
**Phone:** 886-2-33668180
**Role:** CONTACT
#### Locations
**Location 1:**
**City:** Kaohsiung
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Chyi-Rong Chen, Master
- **Phone:** +886-7-7317123
- **Phone Ext:** 8755
- **Role:** CONTACT
***Contact 2:***
- **Email:** [email protected]
- **Name:** Tzu-Ting Chen, Master
- **Phone:** +886-7-7317123
- **Phone Ext:** 8755
- **Role:** CONTACT
**Country:** Taiwan
**Facility:** Chang Gung Memorial Hospital, Kaohsiung Medical Center
**Status:** RECRUITING
**Zip:** 833
#### Overall Officials
**Official 1:**
**Affiliation:** Chang gung memorial hospital, Kaohsiung branch
**Name:** Chen Chyi-Rong, Master
**Role:** PRINCIPAL_INVESTIGATOR
### IPD Sharing Statement Module
**IPD Sharing:** NO
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000019967
- Term: Schizophrenia Spectrum and Other Psychotic Disorders
- ID: D000001523
- Term: Mental Disorders
### Condition Browse Module - Browse Branches
- Abbrev: BXM
- Name: Behaviors and Mental Disorders
- Abbrev: All
- Name: All Conditions
### Condition Browse Module - Browse Leaves
- ID: M15376
- Name: Schizophrenia
- Relevance: HIGH
- As Found: Schizophrenia
- ID: M4815
- Name: Mental Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M14473
- Name: Psychotic Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M21838
- Name: Schizophrenia Spectrum and Other Psychotic Disorders
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000012559
- Term: Schizophrenia
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT03027479
**Acronym:** METERMUS-IMC
**Brief Title:** Skeletal Muscle Energy Metabolism in Women With Weight Loss and Ovarian and/or Endometrial Cancer With Weight Loss
**Official Title:** Study of Differences in Skeletal Muscle Energy Metabolism Alterations in Women With Weight Loss and Ovarian and/or Endometrial Cancer Based on the Body Mass Index
#### Organization Study ID Info
**ID:** PHAO16-LO/METERMUS-IMC
#### Organization
**Class:** OTHER
**Full Name:** University Hospital, Tours
#### Secondary ID Infos
**Domain:** IdRCB
**ID:** 2016-A01323-48
**Type:** REGISTRY
**Domain:** CPP
**ID:** 2016-R27
**Type:** OTHER
### Status Module
#### Completion Date
**Date:** 2019-07-16
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2021-05-06
**Type:** ACTUAL
**Last Update Submit Date:** 2021-05-04
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2019-07-16
**Type:** ACTUAL
#### Start Date
**Date:** 2017-03-01
**Type:** ACTUAL
**Status Verified Date:** 2021-05
#### Study First Post Date
**Date:** 2017-01-23
**Type:** ESTIMATED
**Study First Submit Date:** 2017-01-19
**Study First Submit QC Date:** 2017-01-20
### Sponsor Collaborators Module
#### Collaborators
**Class:** UNKNOWN
**Name:** Inserm U1069 - Team Nutrition, Growth and Cancer
#### Lead Sponsor
**Class:** OTHER
**Name:** University Hospital, Tours
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** the aim is to study skeletal muscle metabolism alterations métaboliques associated with weight loss in women with ovarian and/or endometrial cancer according to BMI.
### Conditions Module
**Conditions:**
- Weight Loss
- Ovarian Carcinoma
- Endometrial Carcinoma
**Keywords:**
- weight loss
- ovarian cancer
- endometrial cancer
- energy metabolism
- Body mass index
- obesity
- muscle energy
### Design Module
#### Design Info
**Allocation:** NON_RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** NONE
**Primary Purpose:** HEALTH_SERVICES_RESEARCH
#### Enrollment Info
**Count:** 70
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** women with ovarian and/or endometrial cancer and weight loss will have muscle, adipose tissue, ovarian tumor and blood samples
**Intervention Names:**
- Other: Samples
**Label:** Case group
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** women scheduled for an intervention for benign ovarian/ endometrial disease and no weight loss will have muscle, adipose tissue and blood samples
**Intervention Names:**
- Other: Samples
**Label:** Control group
**Type:** OTHER
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Case group
- Control group
**Description:** Blood samples, muscle biopsy, adipose tissue samples and ovarian cancer tumor sample
**Name:** Samples
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Description:** Measurement of muscle mitochondrial bioenergetics by high resolution oxygraphy
**Measure:** Measurement of muscle mitochondrial bioenergetics according to BMI
**Time Frame:** muscle biopsy realized during surgery
#### Secondary Outcomes
**Description:** * Quantification of lipids on electron microscope
* Type of muscular fibers
* Analyze of gene expression and proteins involved in 1/ Mitochondrial metabolism 2/ lipid metabolism and 3/ muscular proteolysis by RTqPCR with SyBR Green and Western Blot
**Measure:** on muscle biopsy
**Time Frame:** muscle biopsy realized during surgery
**Description:** adipose tissue composition (fatty acids quantification, sterols, steroids)
* RT-qPCR quantification of molecules involved in lipid metabolism (synthesis, transport) and in inflammation (immune profile and cytokines)
* RTqPCR analysis, western blot and immunohistochemical expression of markers of transformation into brown adipose tissue
* in specific milieu, in vitro experimentation on skeletal muscular cells
**Measure:** on white adipose tissue
**Time Frame:** adipose tissue samples taken during surgery
**Description:** * lipid composition (fatty acids quantification, sterols, steroids)
* RT-qPCR quantification of molecules involved in lipid metabolism (synthesis, transport) and in inflammation (immune profil and cytokines)
* culture of tumour cells to evaluate resistance to chemotherapy (platinum)
**Measure:** on ovarian cancer tumor samples
**Time Frame:** ovarian tumor biopsies realized during surgery
**Description:** assessment of body composition (fat and muscle compartments)
**Measure:** on computerized tomography
**Time Frame:** 1 day of abdominal CT scan imaging procedure performed at diagnosis
### Eligibility Module
**Eligibility Criteria:** Case group selection criteria:
Inclusion Criteria:
* Women aged 18 years old and more
* Ovarian and/or endometrial cancer
* weight loss related to cancer (weight loss \> 5% weight in one month or 10% in 6 months)
* Indication to therapeutic surgery
* Women affiliated to social security scheme
* Women who can understand French and able to sign Informed consent
Exclusion Criteria:
* Unbalanced Diabetes
* Chronic neuromuscular disorder
* Any severe uncontrolled medical condition
Control group selection criteria:
Inclusion Criteria:
* Women aged 18 years and more
* Surgery Indication for a benign endometrial and/ or ovarian disease
* Patient affiliated to social security scheme
* Women who can understand French and able to sign Informed consent
Exclusion Criteria:
* Unbalanced Diabetes
* Chronic neuromuscular disorder
* Any severe uncontrolled medical condition
**Minimum Age:** 18 Years
**Sex:** FEMALE
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Tours
**Country:** France
**Facility:** Gynecology Department, University Teaching Hospital, Tours
**Zip:** 37044
#### Overall Officials
**Official 1:**
**Affiliation:** University Teaching Hospital of Tours
**Name:** Lobna OULDAMER, MD, PhD
**Role:** PRINCIPAL_INVESTIGATOR
### IPD Sharing Statement Module
**IPD Sharing:** NO
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000009375
- Term: Neoplasms, Glandular and Epithelial
- ID: D000009370
- Term: Neoplasms by Histologic Type
- ID: D000009369
- Term: Neoplasms
- ID: D000001836
- Term: Body Weight Changes
- ID: D000014594
- Term: Uterine Neoplasms
- ID: D000005833
- Term: Genital Neoplasms, Female
- ID: D000014565
- Term: Urogenital Neoplasms
- ID: D000009371
- Term: Neoplasms by Site
- ID: D000014591
- Term: Uterine Diseases
- ID: D000005831
- Term: Genital Diseases, Female
- ID: D000052776
- Term: Female Urogenital Diseases
- ID: D000005261
- Term: Female Urogenital Diseases and Pregnancy Complications
- ID: D000091642
- Term: Urogenital Diseases
- ID: D000091662
- Term: Genital Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: BC18
- Name: Nutritional and Metabolic Diseases
- Abbrev: BXS
- Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions
- Abbrev: BC19
- Name: Gland and Hormone Related Diseases
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M5534
- Name: Carcinoma
- Relevance: HIGH
- As Found: Carcinoma
- ID: M5114
- Name: Body Weight
- Relevance: HIGH
- As Found: Weight
- ID: M18102
- Name: Weight Loss
- Relevance: HIGH
- As Found: Weight Loss
- ID: M12701
- Name: Obesity
- Relevance: LOW
- As Found: Unknown
- ID: M12974
- Name: Ovarian Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M1704
- Name: Carcinoma, Ovarian Epithelial
- Relevance: LOW
- As Found: Unknown
- ID: M19235
- Name: Endometrial Neoplasms
- Relevance: HIGH
- As Found: Endometrial Cancer
- ID: M12320
- Name: Neoplasms, Glandular and Epithelial
- Relevance: LOW
- As Found: Unknown
- ID: M12315
- Name: Neoplasms by Histologic Type
- Relevance: LOW
- As Found: Unknown
- ID: M5115
- Name: Body Weight Changes
- Relevance: LOW
- As Found: Unknown
- ID: M17342
- Name: Uterine Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M8945
- Name: Genital Neoplasms, Female
- Relevance: LOW
- As Found: Unknown
- ID: M17315
- Name: Urogenital Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M17339
- Name: Uterine Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M8943
- Name: Genital Diseases, Female
- Relevance: LOW
- As Found: Unknown
- ID: M2876
- Name: Genital Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M2875
- Name: Urogenital Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M27093
- Name: Female Urogenital Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M14127
- Name: Pregnancy Complications
- Relevance: LOW
- As Found: Unknown
- ID: M8399
- Name: Female Urogenital Diseases and Pregnancy Complications
- Relevance: LOW
- As Found: Unknown
- ID: T4352
- Name: Ovarian Cancer
- Relevance: LOW
- As Found: Unknown
- ID: T4354
- Name: Ovarian Epithelial Cancer
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000002277
- Term: Carcinoma
- ID: D000016889
- Term: Endometrial Neoplasms
- ID: D000001835
- Term: Body Weight
- ID: D000015431
- Term: Weight Loss
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT06047379
**Brief Title:** Safety and Efficacy of NEO212 in Patients With Astrocytoma IDH-mutant, Glioblastoma IDH-wildtype or Brain Metastasis
**Official Title:** An Open-label Phase 1/2 Dose Finding, Safety and Efficacy Study of Oral NEO212 in Patients With Astrocytoma IDH-mutant, Glioblastoma IDH-wildtype or Uncontrolled Brain Metastasis in Patients With Select Solid Tumors.
#### Organization Study ID Info
**ID:** NEO212-01
#### Organization
**Class:** INDUSTRY
**Full Name:** Neonc Technologies, Inc.
### Status Module
#### Completion Date
**Date:** 2026-08-31
**Type:** ESTIMATED
#### Last Update Post Date
**Date:** 2024-02-14
**Type:** ACTUAL
**Last Update Submit Date:** 2024-02-13
**Overall Status:** RECRUITING
#### Primary Completion Date
**Date:** 2026-02-28
**Type:** ESTIMATED
#### Start Date
**Date:** 2023-11-01
**Type:** ACTUAL
**Status Verified Date:** 2024-02
#### Study First Post Date
**Date:** 2023-09-21
**Type:** ACTUAL
**Study First Submit Date:** 2023-08-17
**Study First Submit QC Date:** 2023-09-18
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Neonc Technologies, Inc.
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** True
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** This multi-site, Phase 1/2 clinical trial is an open-label study to identify the safety, pharmacokinetics, and efficacy of a repeated dose regimen of NEO212 for the treatment of patients with radiographically-confirmed progression of Astrocytoma IDH-mutant, Glioblastoma IDH-wildtype, and the safety, pharmacokinetics and efficacy of a repeated dose regimen of NEO212 when given with select SOC for the treatment of solid tumor patients with radiographically confirmed uncontrolled brain metastasis. The study will have three phases, Phase 1, Phase 2a and Phase 2b.
**Detailed Description:** This multi-site, Phase 1/2 clinical trial is an open-label study to identify the safety, pharmacokinetics, and efficacy of a repeated dose regimen of NEO212 for the treatment of patients with radiographically-confirmed progression of Astrocytoma IDH-mutant, Glioblastoma IDH-wildtype, and the safety, pharmacokinetics and efficacy of a repeated dose regimen of NEO212 when given with select SOC for the treatment of solid tumor patients with radiographically confirmed uncontrolled brain metastasis. The study will have three phases, Phase 1, Phase 2a and Phase 2b.
Phase 1 is a standard cohort dose escalation 3+3 design with a modified Fibonacci dose escalation used to determine the maximum tolerated dose to select a recommended Phase 2 dose (RP2D) for Phase 2a and Phase 2b. The initial dose of NEO212 will be 170mg and the dose will increase in successive cohorts (220, 400, 610, 810, and 1,000mg) until a MTD is reached and a RP2D is selected. There will be up to 42 patients enrolled in Phase 1. In the event two DLTs are experienced in any cohort, a dose de-escalation cohort will be followed (with half of the dose increase from the previous cohort) to determine the MTD/RP2D.
Phase 2a is a safety run-in study with a standard 3+3 design used to confirm the safety of the MTD/RP2D of NEO212 when given in combination with select SOC regimens for patients with uncontrolled brain metastasis. There will be up to 12 patients enrolled into each combination regimen to confirm safety. One dose below the NEO212 MTD/RP2D Cohort Dose will be administered as a starting dose to establish safety (3+3), before moving to Phase 2b with the MTD/RP2D (3+3). In the event that two DLTs are experienced for patient receiving the MTD/RP2D in combination with SOC, the dose de-escalation cohort will be expanded to determine the MTD for a newly established Phase 2b Treatment Group.
Phase 2b is a dose expansion study to assess efficacy of NEO212, at the MTD/RP2D in patients with Astrocytoma IDH-mutant, Glioblastoma IDH-wildtype as a single Treatment Group. A second Treatment Group to study the MTD/RP2D of NEO212 in combination with select SOC regimens in patients with solid tumors and uncontrolled brain metastasis established in Phase 2a will be evaluated. Phase 2b will be initiated for patients with Astrocytoma IDH-mutant or Glioblastoma IDH-wildtype alongside Phase 2a. There will be up to 28 patients enrolled to have 27 evaluable patients enrolled in each Phase 2b Treatment Group.
For all phases of the study, NEO212 will be self-administered daily for days 1-5 of a 28-day treatment cycle.
### Conditions Module
**Conditions:**
- Diffuse Astrocytoma, IDH-Mutant
- Glioblastoma, IDH-wildtype
- Brain Metastases, Adult
- Cervical Cancer
- Colorectal Cancer
- Esophageal Cancer
- Esophageal Squamous Cell Carcinoma
- Gastric Cancer
- Gastroesophageal Junction Adenocarcinoma
- Head and Neck Squamous Cell Carcinoma
- Melanoma
- Merkel Cell Carcinoma
- Microsatellite Instability-High Solid Malignant Tumor
- Mismatch Repair Deficient Solid Malignant Tumor
- Microsatellite Instability-High Colorectal Cancer
- Mismatch Repair Deficient Colorectal Cancer
- Non-small Cell Lung Cancer
- Renal Cell Carcinoma
- Small Cell Lung Cancer
- Squamous Cell Carcinoma
- Urothelial Carcinoma
**Keywords:**
- Astrocytoma
- IDH-mutant
- Glioblastoma
- IDH-wildtype
- Brain Metastases
- CNS Tumor
- GBM
- NeOnc
- Anova
- NEO212
- NEO100
- TMZ
### Design Module
#### Design Info
**Allocation:** NON_RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 134
**Type:** ESTIMATED
**Phases:**
- PHASE1
- PHASE2
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** - Unresectable or metastatic melanoma with uncontrolled metastases to the brain.
NEO212 - Starting one dose level under RP2D administered orally on days 1 - 5 of a 28-day treatment cycle, escalated to RP2D per Protocol dose escalation rules.
Ipilimumab - 3 mg/kg administered IV over 90 minutes every 3 weeks for a maximum of 3 doses per package insert.
**Intervention Names:**
- Drug: NEO212 Oral Capsule
- Drug: Ipilimumab
**Label:** Phase 2a Safety Run-In - NEO212 and Ipilimumab
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** The following primary cancers with uncontrolled metrastases to the brain:
* Unresectable or metastatic melanoma.
* NSCLC expressing PD-L1, with no EGFR or ALK genomic tumor aberrations.
* Metastatic NSCLC whose tumors express PD-L1.
* EGFR or ALK genomic tumor aberrations must have disease progression.
* SCLC.
* Unresectable, recurrent HNSCC whose tumors express PD-L1.
* HNSCC on or after platinum-containing chemotherapy.
* Urothelial carcinoma whose tumors express PD-L1.
* Urothelial carcinoma.
* Microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR).
* Microsatellite Instability-high or Mismatch Repair Deficient Colorectal Cancer (CRC).
* Gastric or gastroesophageal junction adenocarcinoma.
* Esophageal or gastroesophageal juncUon (GEJ).
* Cervical cancer.
* Merkel cell carcinoma.
NEO212 - Same as Arm 1.
Pembrolizumab - 200 mg administered every 3 weeks per package insert.
**Intervention Names:**
- Drug: NEO212 Oral Capsule
- Drug: Pembrolizumab
**Label:** Phase 2a Safety Run-In - NEO212 and Pembrolizumab
**Type:** EXPERIMENTAL
#### Arm Group 3
**Description:** The following primary cancers with uncontrolled metrastases to the brain:
* Unresectable or metastatic melanoma.
* Metastatic non-small cell lung cancer.
* Advanced renal cell carcinoma.
* Squamous cell carcinoma of the head and neck.
* Urothelial carcinoma.
* Microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) colorectal cancer.
* Unresectable esophageal squamous cell carcinoma (ESCC).
NEO212 - Starting one dose level under RP2D administered orally on days 1 - 5 of a 28-day treatment cycle, escalated to RP2D per Protocol dose escalation rules.
Nivolumab - 240 mg administered every 2 weeks per package insert
**Intervention Names:**
- Drug: NEO212 Oral Capsule
- Drug: Nivolumab
**Label:** Phase 2a Safety Run-In - NEO212 and Nivolumab
**Type:** EXPERIMENTAL
#### Arm Group 4
**Description:** - Colorectal cancer (CRC) with uncontrolled metastases to the brain who have been previously treated with fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy, an anti-VEGF therapy, and, if KRAS wild type, an anU-EGFR therapy.
NEO212 - Starting one dose level under RP2D administered orally on days 1 - 5 of a 28-day treatment cycle, escalated to RP2D per Protocol dose escalation rules.
Stivarga - 160 mg orally, once daily for the first 21 days of each 28-day cycle per package insert
**Intervention Names:**
- Drug: NEO212 Oral Capsule
- Drug: Regorafenib
**Label:** Phase 2a Safety Run-In - NEO212 and Stivarga (Regorafenib)
**Type:** EXPERIMENTAL
#### Arm Group 5
**Description:** - Colorectal cancer (CRC) with uncontrolled metastases to the brain.
NEO212 - Starting one dose level under RP2D administered orally on days 1 - 5 of a 28-day treatment cycle, escalated to RP2D per Protocol dose escalation rules.
Carboplatin - 300 mg/m2 IV on day 1 every 4 weeks for 6 cycles per package insert.
Paclitaxel - 135mg/m2 IV administered over 24 hours, every 3 weeks per package insert.
**Intervention Names:**
- Drug: NEO212 Oral Capsule
- Drug: Carboplatin
- Drug: Paclitaxel
**Label:** Phase 2a Safety Run-In - NEO212 and CarbolaUn (ParaplaUn) + Paclitaxel (Taxol)
**Type:** EXPERIMENTAL
#### Arm Group 6
**Description:** - Metastatic colorectal cancer (mCRC) with uncontrolled metastases to the brain, that is resistant to or has progressed following an oxaliplatin-containing regimen
NEO212 - Starting one dose level under RP2D administered orally on days 1 - 5 of a 28-day treatment cycle, escalated to RP2D per Protocol dose escalation rules.
FOLFIRI - 4 mg/kg aIV over 1 hour every 2 weeks.
Bevacizumab - 10 mg/kg IV every 2 weeks.
**Intervention Names:**
- Drug: NEO212 Oral Capsule
- Drug: FOLFIRI Protocol
- Drug: Bevacizumab
**Label:** Phase 2a Safety Run-In - NEO212 and FOLFIRI (Zaltrap) + Bevacizumab (Avastin)
**Type:** EXPERIMENTAL
#### Arm Group 7
**Description:** Patients receiving NEO212 alone for treatment of Astrocytoma IDH-mutant and Glioblastoma IDH-wildtype.
NEO212 - Starting one dose level under RP2D administered orally on days 1 - 5 of a 28-day treatment cycle, escalated to RP2D per Protocol dose escalation rules.
**Intervention Names:**
- Drug: NEO212 Oral Capsule
**Label:** Phase 2b efficacy - NEO212 for Astrocytoma IDH-mutant and Glioblastoma IDH-wildtype
**Type:** EXPERIMENTAL
#### Arm Group 8
**Description:** Patients receiving NEO212 in combination with select standard of care treatments for treatment of uncontrolled metastases to the brain.
NEO212 - Starting one dose level under RP2D administered orally on days 1 - 5 of a 28-day treatment cycle, escalated to RP2D per Protocol dose escalation rules.
SOC treatments established to be safe in Phase 2a of the study will be used in this arm of Phase 2b.
**Intervention Names:**
- Drug: NEO212 Oral Capsule
- Drug: Ipilimumab
- Drug: Pembrolizumab
- Drug: Nivolumab
- Drug: Regorafenib
- Drug: Carboplatin
- Drug: Paclitaxel
- Drug: FOLFIRI Protocol
- Drug: Bevacizumab
**Label:** Phase 2b efficacy - NEO212 & SOC for Uncontrolled Metastases to the Brain
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Phase 2a Safety Run-In - NEO212 and CarbolaUn (ParaplaUn) + Paclitaxel (Taxol)
- Phase 2a Safety Run-In - NEO212 and FOLFIRI (Zaltrap) + Bevacizumab (Avastin)
- Phase 2a Safety Run-In - NEO212 and Ipilimumab
- Phase 2a Safety Run-In - NEO212 and Nivolumab
- Phase 2a Safety Run-In - NEO212 and Pembrolizumab
- Phase 2a Safety Run-In - NEO212 and Stivarga (Regorafenib)
- Phase 2b efficacy - NEO212 & SOC for Uncontrolled Metastases to the Brain
- Phase 2b efficacy - NEO212 for Astrocytoma IDH-mutant and Glioblastoma IDH-wildtype
**Description:** NEO212 is a novel chemical entity that was generated by covalent conjugation of temozolomide (TMZ) with perillyl alcohol (POH).
**Name:** NEO212 Oral Capsule
**Other Names:**
- POH-TMZ
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- Phase 2a Safety Run-In - NEO212 and Ipilimumab
- Phase 2b efficacy - NEO212 & SOC for Uncontrolled Metastases to the Brain
**Description:** Ipilimumab, sold under the brand name Yervoy, is a monoclonal antibody medication that works to activate the immune system by targeting CTLA-4, a protein receptor that downregulates the immune system. Cytotoxic T lymphocytes can recognize and destroy cancer cells.
**Name:** Ipilimumab
**Other Names:**
- Yervoy
**Type:** DRUG
#### Intervention 3
**Arm Group Labels:**
- Phase 2a Safety Run-In - NEO212 and Pembrolizumab
- Phase 2b efficacy - NEO212 & SOC for Uncontrolled Metastases to the Brain
**Description:** Pembrolizumab, sold under the brand name Keytruda, is a humanized antibody used in cancer immunotherapy that treats melanoma, lung cancer, head and neck cancer, Hodgkin lymphoma, stomach cancer, cervical cancer, and certain types of breast cancer. It is given by slow injection into a vein.
**Name:** Pembrolizumab
**Other Names:**
- Keytruda
**Type:** DRUG
#### Intervention 4
**Arm Group Labels:**
- Phase 2a Safety Run-In - NEO212 and Nivolumab
- Phase 2b efficacy - NEO212 & SOC for Uncontrolled Metastases to the Brain
**Description:** Nivolumab, sold under the brand name Opdivo, is a medication used to treat a number of types of cancer.
**Name:** Nivolumab
**Other Names:**
- Opdivo
**Type:** DRUG
#### Intervention 5
**Arm Group Labels:**
- Phase 2a Safety Run-In - NEO212 and Stivarga (Regorafenib)
- Phase 2b efficacy - NEO212 & SOC for Uncontrolled Metastases to the Brain
**Description:** Regorafenib, sold under the brand name Stivarga among others, is an oral multi-kinase inhibitor developed by Bayer which targets angiogenic, stromal and oncogenic receptor tyrosine kinase. Regorafenib shows anti-angiogenic activity due to its dual targeted VEGFR2-TIE2 tyrosine kinase inhibition
**Name:** Regorafenib
**Other Names:**
- Stivagra
**Type:** DRUG
#### Intervention 6
**Arm Group Labels:**
- Phase 2a Safety Run-In - NEO212 and CarbolaUn (ParaplaUn) + Paclitaxel (Taxol)
- Phase 2b efficacy - NEO212 & SOC for Uncontrolled Metastases to the Brain
**Description:** Carboplatin, sold under the trade name Paraplatin among others, is a chemotherapy medication used to treat a number of forms of cancer. This includes ovarian cancer, lung cancer, head and neck cancer, brain cancer, and neuroblastoma. It is used by injection into a vein.
**Name:** Carboplatin
**Other Names:**
- Paraplatin
**Type:** DRUG
#### Intervention 7
**Arm Group Labels:**
- Phase 2a Safety Run-In - NEO212 and CarbolaUn (ParaplaUn) + Paclitaxel (Taxol)
- Phase 2b efficacy - NEO212 & SOC for Uncontrolled Metastases to the Brain
**Description:** Paclitaxel, sold under the brand name Taxol among others, is a chemotherapy medication used to treat ovarian cancer, esophageal cancer, breast cancer, lung cancer, Kaposi's sarcoma, cervical cancer, and pancreatic cancer. It is administered by intravenous injection
**Name:** Paclitaxel
**Other Names:**
- Taxol
**Type:** DRUG
#### Intervention 8
**Arm Group Labels:**
- Phase 2a Safety Run-In - NEO212 and FOLFIRI (Zaltrap) + Bevacizumab (Avastin)
- Phase 2b efficacy - NEO212 & SOC for Uncontrolled Metastases to the Brain
**Description:** FOLFIRI is a chemotherapy regimen for treatment of colorectal cancer. It is made up of the following drugs:
FOL - folinic acid (leucovorin), a vitamin B derivative with multiple applications, which in this context increases the cytotoxicity of 5-fluorouracil; F - fluorouracil (5-FU), a pyrimidine analog and antimetabolite which incorporates into the DNA molecule and stops synthesis; and IRI - irinotecan (Camptosar), a topoisomerase inhibitor, which prevents DNA from uncoiling and duplicating.
**Name:** FOLFIRI Protocol
**Other Names:**
- Zaltrap
**Type:** DRUG
#### Intervention 9
**Arm Group Labels:**
- Phase 2a Safety Run-In - NEO212 and FOLFIRI (Zaltrap) + Bevacizumab (Avastin)
- Phase 2b efficacy - NEO212 & SOC for Uncontrolled Metastases to the Brain
**Description:** Bevacizumab, sold under the brand name Avastin among others, is a medication used to treat a number of types of cancers and a specific eye disease. For cancer, it is given by slow injection into a vein and used for colon cancer, lung cancer, glioblastoma, and renal-cell carcinoma
**Name:** Bevacizumab
**Other Names:**
- Avastin
- Mvasi
- Zirabev
- Alymsys
- Vegzelma
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Description:** As determined by incidence and severity of adverse events according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE v5.0
**Measure:** Phase 1: safety and tolerability of increasing dose levels of orally administered NEO212 alone in patients with Astrocytoma IDH-mutant, Glioblastoma IDH-wildtype or patients with select solid tumors with uncontrolled metastases to the brain
**Time Frame:** 6 months
**Description:** Maximum Tolerated Dose of NEO212 as determined by the dose escalation rules.
**Measure:** Phase 1: Identify the maximum tolerated dose (MTD) of NEO212
**Time Frame:** 6 months
**Description:** Determine the recommended Phase 2 dose (RP2D) of NEO212
**Measure:** Phase 1: Determine the recommended Phase 2 dose (RP2D) of NEO212
**Time Frame:** 6 months
**Description:** Determined by incidence and severity of adverse events determined according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE v5.0).
**Measure:** Phase 2a: Assess the safety and tolerability of orally administered NEO212 in combination with select SOC regimens following a standard 3+3 design in patients with select solid tumors with uncontrolled metastases to the brain
**Time Frame:** 6 months
**Description:** Determine the intracranial progression-free survival rate at six months (PFS6) of orally administered NEO212 alone in patients with Astrocytoma IDH-mutant, Glioblastoma IDH-wildtype.
**Measure:** Phase 2b: Determine the intracranial progression-free survival rate at six months (PFS6) of orally administered NEO212 alone in patients with Astrocytoma IDH-mutant, Glioblastoma IDH-wildtype.
**Time Frame:** 6 months
**Description:** Determine the intracranial progression-free survival rate at six months (PFS6) of orally administered NEO212 in combination with select SOC regimens in patients with select solid tumors (see Appendix 2) with uncontrolled metastases to the brain.
**Measure:** Phase 2b: Determine the intracranial progression-free survival rate at six months (PFS6) of orally administered NEO212 in combination with select SOC regimens in patients with select solid tumors with uncontrolled metastases to the brain.
**Time Frame:** 6 months
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria
* Patient must be ≥ 18yrs of age.
* Patient must have the ability to understand, and the willingness to sign, a written informed consent form.
* Patient has been on a stable or decreasing dose of steroids for at least five days prior to the date of informed consent.
* Any toxicity from prior therapy must be resolved or at maximum Grade 1 prior to initiation of NEO212.
* If progression of disease occurs within 90 days or conformal radiation, the progression/recurrence must be outside of the radiation field or proven by biopsy/resection.
* Patient with Astrocytoma IDH-mutant, Glioblastoma IDH-wildtype must have a Karnofsky Performance Status (KPS) of ≥ 60.
* Patient with select solid tumors must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
* Patient must have an expected survival or at least three months.
* Patient must have a baseline MRI of the brain with gadolinium within 14 days of administration of NEO212.
* Patient must have a baseline CT scan with IV contrast and oral contrast of neck, chest, abdomen and pelvis within 14 days of administration of NEO212.
* Patients must be able to comply with all study assessments.
* If patient suffers from seizures (s)he must be controlled on a stable dose of anti-epileptics for 14-days prior to the date of informed consent.
* Patient must have adequate organ and marrow function as follows:
* Absolute neutrophil count ≥ 1,500/microliter
* Platelets ≥ 100,000/microliter
* Total bilirubin within normal institutional limits
* AST (SGOT) / ALT (SPGT) ≤ 2.5 x institutional upper limit of normal
* Creatinine clearance (CrCl) of \>60 mL/min (using the Cockcroft-Gault formula or 24-hour urine collection).
* Female patients of child-bearing potential and male patients must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) for 30 days prior to the first dose of NEO212, for the duration of study participation, and for 90 days following completion of therapy.
* 1. A female of child-bearing potential is any women (regardless of sexual orientation, not having undergone a tubal ligation, or remaining celibate by choice) who meets the following criteria:
* Has not undergone a hysterectomy or bilateral oophorectomy; or
* Has not been naturally postmenopausal for at least 12 consecutive months (i.e., has not had menses at any time in the preceding 12 consecutive months).
* A negative serum pregnancy test will be required of all female patients of child-bearing potential within seven days prior to the receipt of NEO212.
* A serum pregnancy test will be repeated immediately if pregnancy is suspected.
Phase 1: (dose escalation)
1. Patient must:
* have radiographically confirmed Astrocytoma IDH-mutant, Glioblastoma IDH-wildtype following previous radiation therapy or treatment with temozolomide and radiation, or
* have select solid tumors with uncontrolled metastases to the brain (confirmed by cranial CT or MRI) that is not controlled by surgery or radiation therapy and receiving one of the protocol approved SOC regimens.
2. Patients receiving prior systemic therapy must have a minimum wash-out period (defined as the period prior to receipt of the first dose of NEO212) of:
* 28 days or 5 half-lives (whichever is shorter) elapsed from the administration from any experimental agent;
* 2 weeks from administration of immunotherapies;
* 28 days from administration of cytotoxic agents; and
* 7 days from administration of non-cytotoxic agents (interferon, tamoxifen, thalidomide, cis-retinoic acid, and herbal medicine).
NOTE: No washout is necessary for alternating electrical fields.
Phase 2a: (safety run-in)
1. Patient must have select solid tumors with uncontrolled metastases to the brain (confirmed by cranial CT or MRI) that is not controlled by surgery or radiation therapy and receiving one of the protocol approved SOC regimens.
2. Patients receiving prior systemic therapy must be receiving one of the protocol approved Standard of Care (SOC) regimens.
3. Patient must have measurable/evaluable CNS disease per RANO or RANO-BM criteria.
4. Patient must have measurable/evaluable systemic disease per RECIST v1.1 criteria.
Phase 2b: (efficacy)
1. Patient must:
* have radiographically confirmed Astrocytoma IDH-mutant, Glioblastoma IDH-wildtype following previous radiation therapy or treatment with temozolomide and radiation, or
* have select solid tumors with uncontrolled metastases to the brain (confirmed by cranial CT or MRI) that is not controlled by surgery or radiation therapy and receiving one of the protocol approved SOC regimens.
2. Patients receiving prior systemic therapy must be receiving one of the protocol approved Standard of Care (SOC) regimens.
3. Patient must have measurable/evaluable CNS disease per RANO or RANO-BM criteria.
4. Patient must have measurable/evaluable systemic disease per RECIST v1.1 criteria.
5. Creatinine clearance (CrCl) of \>60 mL/min (using the Cockcroft-Gault formula or 24-hour urine collection).Female patients of child-bearing potential and male patients must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) for 30 days prior to the first dose of NEO212, for the duration of study participation, and for 90 days following completion of therapy
Exclusion Criteria: (all Phases)
1. Patient in Phase 1 concurrently receiving any other antitumor therapy.
2. Patient in Phase 2a or 2b who is concurrently receiving any SOC therapy not listed in Appendix 1.
3. Patients with metastases to the spinal cord parenchyma.
4. Patients with metastases to the meninges.
5. Patient has had more than one recurrence or progression of his/her CNS tumor(s).
6. Patient has received stereotactic or highly conformal radiotherapy to CNS lesions within 2 weeks before receipt of NEO212.
7. Patient with history of known leptomeningeal involvement.
8. Patient has prior history or new diagnosis of secondary cancer within five years prior to the date of informed consent, except for basal cell carcinoma or squamous cell carcinoma of the skin.
9. Patient has a corrected QT interval (using Fridericia's correction formula) (QTcF) of \> 470 msec, , a history of additional risk factors for TdP (e.g. heart failure, hypokalemia), and/or the use of concomitant medications that prolong QT/QTc interval.
10. Patient had surgery within 7 days prior to the date of informed consent.
11. Patient has not recovered to Grade 1 from treatment related adverse events due to chemotherapy, immunotherapy, or radiation therapy.
12. Patient had prior treatment with perillyl alcohol.
13. Patient has a history of allergic reactions attributed to perillyl alcohol.
14. Patients with an autoimmune disease that required systemic therapy or a medical condition that requires immunosuppression.
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Christopher Beardmore
**Phone:** 224 218 2408
**Role:** CONTACT
**Contact 2:**
**Email:** [email protected]
**Name:** Chloe Richmond
**Phone:** 224 218 2408
**Role:** CONTACT
#### Locations
**Location 1:**
**City:** Beverly Hills
**Contacts:**
***Contact 1:***
- **Name:** Mini Gil, MD
- **Phone:** 424-777-0708
- **Role:** CONTACT
***Contact 2:***
- **Name:** Kamlesh Sankhala, MD
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Precision NextGen Oncology
**State:** California
**Status:** RECRUITING
**Zip:** 90212
**Location 2:**
**City:** Tacoma
**Contacts:**
***Contact 1:***
- **Name:** Sue Quinsey
- **Phone:** 253-380-8173
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jorge Chaves, MD
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Northwest Medical Specialties
**State:** Washington
**Status:** RECRUITING
**Zip:** 98405
#### Overall Officials
**Official 1:**
**Affiliation:** NeOnc Technologies
**Name:** Tom Chen, MD, PhD
**Role:** STUDY_CHAIR
**Official 2:**
**Affiliation:** NeOnc Technologies
**Name:** Vincent Simmons, PhD
**Role:** STUDY_DIRECTOR
**Official 3:**
**Affiliation:** NeOnc Technologies
**Name:** Patrick Walters
**Role:** STUDY_DIRECTOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000009375
- Term: Neoplasms, Glandular and Epithelial
- ID: D000009370
- Term: Neoplasms by Histologic Type
- ID: D000012142
- Term: Respiratory Tract Neoplasms
- ID: D000013899
- Term: Thoracic Neoplasms
- ID: D000009371
- Term: Neoplasms by Site
- ID: D000008171
- Term: Lung Diseases
- ID: D000012140
- Term: Respiratory Tract Diseases
- ID: D000002283
- Term: Carcinoma, Bronchogenic
- ID: D000001984
- Term: Bronchial Neoplasms
- ID: D000007414
- Term: Intestinal Neoplasms
- ID: D000005770
- Term: Gastrointestinal Neoplasms
- ID: D000004067
- Term: Digestive System Neoplasms
- ID: D000004066
- Term: Digestive System Diseases
- ID: D000005767
- Term: Gastrointestinal Diseases
- ID: D000003108
- Term: Colonic Diseases
- ID: D000007410
- Term: Intestinal Diseases
- ID: D000012002
- Term: Rectal Diseases
- ID: D000018358
- Term: Neuroendocrine Tumors
- ID: D000017599
- Term: Neuroectodermal Tumors
- ID: D000009373
- Term: Neoplasms, Germ Cell and Embryonal
- ID: D000009380
- Term: Neoplasms, Nerve Tissue
- ID: D000018307
- Term: Neoplasms, Squamous Cell
- ID: D000000230
- Term: Adenocarcinoma
- ID: D000009385
- Term: Neoplastic Processes
- ID: D000010335
- Term: Pathologic Processes
- ID: D000005910
- Term: Glioma
- ID: D000018302
- Term: Neoplasms, Neuroepithelial
- ID: D000004938
- Term: Esophageal Neoplasms
- ID: D000006258
- Term: Head and Neck Neoplasms
- ID: D000004935
- Term: Esophageal Diseases
- ID: D000016543
- Term: Central Nervous System Neoplasms
- ID: D000009423
- Term: Nervous System Neoplasms
- ID: D000001927
- Term: Brain Diseases
- ID: D000002493
- Term: Central Nervous System Diseases
- ID: D000009422
- Term: Nervous System Diseases
- ID: D000027601
- Term: Polyomavirus Infections
- ID: D000004266
- Term: DNA Virus Infections
- ID: D000014777
- Term: Virus Diseases
- ID: D000007239
- Term: Infections
- ID: D000014412
- Term: Tumor Virus Infections
- ID: D000018278
- Term: Carcinoma, Neuroendocrine
- ID: D000042822
- Term: Genomic Instability
### Condition Browse Module - Browse Branches
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: All
- Name: All Conditions
- Abbrev: BXS
- Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: BC08
- Name: Respiratory Tract (Lung and Bronchial) Diseases
- Abbrev: BC06
- Name: Digestive System Diseases
- Abbrev: BC17
- Name: Skin and Connective Tissue Diseases
- Abbrev: BC10
- Name: Nervous System Diseases
- Abbrev: BC01
- Name: Infections
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M5534
- Name: Carcinoma
- Relevance: HIGH
- As Found: Carcinoma
- ID: M5548
- Name: Carcinoma, Renal Cell
- Relevance: LOW
- As Found: Unknown
- ID: M27510
- Name: Microsatellite Instability
- Relevance: HIGH
- As Found: Microsatellite Instability
- ID: M11172
- Name: Lung Neoplasms
- Relevance: HIGH
- As Found: Lung Cancer
- ID: M5546
- Name: Carcinoma, Non-Small-Cell Lung
- Relevance: LOW
- As Found: Unknown
- ID: M5551
- Name: Carcinoma, Transitional Cell
- Relevance: LOW
- As Found: Unknown
- ID: M16064
- Name: Stomach Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M28323
- Name: Small Cell Lung Carcinoma
- Relevance: HIGH
- As Found: Small Cell Lung Cancer
- ID: M5550
- Name: Carcinoma, Squamous Cell
- Relevance: HIGH
- As Found: Squamous Cell Carcinoma
- ID: M1733
- Name: Esophageal Squamous Cell Carcinoma
- Relevance: HIGH
- As Found: Esophageal Squamous Cell Carcinoma
- ID: M5830
- Name: Uterine Cervical Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M11528
- Name: Melanoma
- Relevance: LOW
- As Found: Unknown
- ID: M8088
- Name: Esophageal Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M12307
- Name: Neoplasm Metastasis
- Relevance: HIGH
- As Found: Metastases
- ID: M3585
- Name: Adenocarcinoma
- Relevance: LOW
- As Found: Unknown
- ID: M9019
- Name: Glioblastoma
- Relevance: HIGH
- As Found: Glioblastoma
- ID: M18937
- Name: Central Nervous System Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M4561
- Name: Astrocytoma
- Relevance: HIGH
- As Found: Astrocytoma
- ID: M17967
- Name: Carcinoma, Merkel Cell
- Relevance: HIGH
- As Found: Merkel Cell Carcinoma
- ID: M17890
- Name: Colorectal Neoplasms
- Relevance: HIGH
- As Found: Colorectal Cancer
- ID: M5209
- Name: Brain Neoplasms
- Relevance: HIGH
- As Found: Brain Metastases
- ID: M1689
- Name: Squamous Cell Carcinoma of Head and Neck
- Relevance: HIGH
- As Found: Head and Neck Squamous Cell Carcinoma
- ID: M12320
- Name: Neoplasms, Glandular and Epithelial
- Relevance: LOW
- As Found: Unknown
- ID: M12315
- Name: Neoplasms by Histologic Type
- Relevance: LOW
- As Found: Unknown
- ID: M14979
- Name: Respiratory Tract Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M16658
- Name: Thoracic Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M11168
- Name: Lung Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M14977
- Name: Respiratory Tract Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5540
- Name: Carcinoma, Bronchogenic
- Relevance: LOW
- As Found: Unknown
- ID: M5260
- Name: Bronchial Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M10448
- Name: Intestinal Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M8886
- Name: Gastrointestinal Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M7256
- Name: Digestive System Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M7255
- Name: Digestive System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M8883
- Name: Gastrointestinal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M6336
- Name: Colonic Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M10444
- Name: Intestinal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M14844
- Name: Rectal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M20495
- Name: Neuroendocrine Tumors
- Relevance: LOW
- As Found: Unknown
- ID: M19845
- Name: Neuroectodermal Tumors
- Relevance: LOW
- As Found: Unknown
- ID: M20388
- Name: Neuroectodermal Tumors, Primitive
- Relevance: LOW
- As Found: Unknown
- ID: M12318
- Name: Neoplasms, Germ Cell and Embryonal
- Relevance: LOW
- As Found: Unknown
- ID: M12325
- Name: Neoplasms, Nerve Tissue
- Relevance: LOW
- As Found: Unknown
- ID: M20451
- Name: Neoplasms, Squamous Cell
- Relevance: LOW
- As Found: Unknown
- ID: M12330
- Name: Neoplastic Processes
- Relevance: LOW
- As Found: Unknown
- ID: M9020
- Name: Glioma
- Relevance: LOW
- As Found: Unknown
- ID: M20446
- Name: Neoplasms, Neuroepithelial
- Relevance: LOW
- As Found: Unknown
- ID: M9348
- Name: Head and Neck Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M8085
- Name: Esophageal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M12367
- Name: Nervous System Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M5204
- Name: Brain Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5742
- Name: Central Nervous System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M10283
- Name: Infections
- Relevance: LOW
- As Found: Unknown
- ID: M6368
- Name: Communicable Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M23236
- Name: Polyomavirus Infections
- Relevance: LOW
- As Found: Unknown
- ID: M17522
- Name: Virus Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M7442
- Name: DNA Virus Infections
- Relevance: LOW
- As Found: Unknown
- ID: M17162
- Name: Tumor Virus Infections
- Relevance: LOW
- As Found: Unknown
- ID: M20423
- Name: Carcinoma, Neuroendocrine
- Relevance: LOW
- As Found: Unknown
- ID: M25088
- Name: Genomic Instability
- Relevance: LOW
- As Found: Unknown
- ID: T4906
- Name: Renal Cell Carcinoma
- Relevance: HIGH
- As Found: Renal Cell Carcinoma
- ID: T1341
- Name: Clear Cell Renal Cell Carcinoma
- Relevance: LOW
- As Found: Unknown
- ID: T5693
- Name: Transitional Cell Carcinoma
- Relevance: LOW
- As Found: Unknown
- ID: T5271
- Name: Small Cell Lung Cancer
- Relevance: HIGH
- As Found: Small Cell Lung Cancer
- ID: T5486
- Name: Stomach Cancer
- Relevance: LOW
- As Found: Unknown
- ID: T2141
- Name: Esophageal Cancer
- Relevance: HIGH
- As Found: Esophageal Cancer
- ID: T2518
- Name: Glioblastoma
- Relevance: HIGH
- As Found: Glioblastoma
- ID: T3716
- Name: Merkel Cell Carcinoma
- Relevance: HIGH
- As Found: Merkel Cell Carcinoma
- ID: T1858
- Name: Diffuse Astrocytoma
- Relevance: HIGH
- As Found: Diffuse astrocytoma
- ID: T4091
- Name: Neuroendocrine Tumor
- Relevance: LOW
- As Found: Unknown
- ID: T4092
- Name: Neuroepithelioma
- Relevance: LOW
- As Found: Unknown
- ID: T2519
- Name: Glioma
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000015266
- Term: Carcinoma, Merkel Cell
- ID: D000002277
- Term: Carcinoma
- ID: D000009369
- Term: Neoplasms
- ID: D000008175
- Term: Lung Neoplasms
- ID: D000015179
- Term: Colorectal Neoplasms
- ID: D000002294
- Term: Carcinoma, Squamous Cell
- ID: D000009362
- Term: Neoplasm Metastasis
- ID: D000005909
- Term: Glioblastoma
- ID: D000055752
- Term: Small Cell Lung Carcinoma
- ID: D000001932
- Term: Brain Neoplasms
- ID: D000001254
- Term: Astrocytoma
- ID: D000077195
- Term: Squamous Cell Carcinoma of Head and Neck
- ID: D000077277
- Term: Esophageal Squamous Cell Carcinoma
- ID: D000053842
- Term: Microsatellite Instability
### Intervention Browse Module - Ancestors
- ID: D000000972
- Term: Antineoplastic Agents, Phytogenic
- ID: D000000970
- Term: Antineoplastic Agents
- ID: D000050257
- Term: Tubulin Modulators
- ID: D000050256
- Term: Antimitotic Agents
- ID: D000050258
- Term: Mitosis Modulators
- ID: D000045504
- Term: Molecular Mechanisms of Pharmacological Action
- ID: D000074322
- Term: Antineoplastic Agents, Immunological
- ID: D000020533
- Term: Angiogenesis Inhibitors
- ID: D000043924
- Term: Angiogenesis Modulating Agents
- ID: D000006133
- Term: Growth Substances
- ID: D000045505
- Term: Physiological Effects of Drugs
- ID: D000006131
- Term: Growth Inhibitors
- ID: D000082082
- Term: Immune Checkpoint Inhibitors
### Intervention Browse Module - Browse Branches
- Abbrev: Micro
- Name: Micronutrients
- Abbrev: All
- Name: All Drugs and Chemicals
- Abbrev: ANeo
- Name: Antineoplastic Agents
- Abbrev: Hemat
- Name: Hematinics
- Abbrev: Infe
- Name: Anti-Infective Agents
- Abbrev: CNSDep
- Name: Central Nervous System Depressants
- Abbrev: Vi
- Name: Vitamins
- Abbrev: AA
- Name: Amino Acids
- Abbrev: HB
- Name: Herbal and Botanical
- Abbrev: Ot
- Name: Other Dietary Supplements
### Intervention Browse Module - Browse Leaves
- ID: M6191
- Name: Leucovorin
- Relevance: LOW
- As Found: Unknown
- ID: M8600
- Name: Fluorouracil
- Relevance: LOW
- As Found: Unknown
- ID: M29233
- Name: Levoleucovorin
- Relevance: LOW
- As Found: Unknown
- ID: M1692
- Name: Temozolomide
- Relevance: LOW
- As Found: Unknown
- ID: M19537
- Name: Paclitaxel
- Relevance: HIGH
- As Found: Surgery
- ID: M231
- Name: Albumin-Bound Paclitaxel
- Relevance: LOW
- As Found: Unknown
- ID: M4225
- Name: Antibodies
- Relevance: LOW
- As Found: Unknown
- ID: M4230
- Name: Antibodies, Monoclonal
- Relevance: LOW
- As Found: Unknown
- ID: M22318
- Name: Angiogenesis Inhibitors
- Relevance: LOW
- As Found: Unknown
- ID: M349416
- Name: Pembrolizumab
- Relevance: HIGH
- As Found: Blind
- ID: M257727
- Name: Aflibercept
- Relevance: LOW
- As Found: Unknown
- ID: M2853
- Name: Immunomodulating Agents
- Relevance: LOW
- As Found: Unknown
- ID: M1854
- Name: Nivolumab
- Relevance: HIGH
- As Found: Prospective
- ID: M246
- Name: Bevacizumab
- Relevance: HIGH
- As Found: Non-
- ID: M18650
- Name: Carboplatin
- Relevance: HIGH
- As Found: System
- ID: M1674
- Name: Oxaliplatin
- Relevance: LOW
- As Found: Unknown
- ID: M1671
- Name: Irinotecan
- Relevance: LOW
- As Found: Unknown
- ID: M1348
- Name: Ipilimumab
- Relevance: HIGH
- As Found: While
- ID: M8618
- Name: Folic Acid
- Relevance: LOW
- As Found: Unknown
- ID: M10184
- Name: Immunoglobulins
- Relevance: LOW
- As Found: Unknown
- ID: M17558
- Name: Vitamins
- Relevance: LOW
- As Found: Unknown
- ID: M17546
- Name: Vitamin B Complex
- Relevance: LOW
- As Found: Unknown
- ID: M206775
- Name: Perillyl alcohol
- Relevance: LOW
- As Found: Unknown
- ID: M3777
- Name: Ethanol
- Relevance: LOW
- As Found: Unknown
- ID: M4281
- Name: Antimetabolites
- Relevance: LOW
- As Found: Unknown
- ID: M17544
- Name: Vitamin A
- Relevance: LOW
- As Found: Unknown
- ID: M302395
- Name: Retinol palmitate
- Relevance: LOW
- As Found: Unknown
- ID: M26197
- Name: Tubulin Modulators
- Relevance: LOW
- As Found: Unknown
- ID: M26196
- Name: Antimitotic Agents
- Relevance: LOW
- As Found: Unknown
- ID: M1346
- Name: Antineoplastic Agents, Immunological
- Relevance: LOW
- As Found: Unknown
- ID: M9231
- Name: Growth Inhibitors
- Relevance: LOW
- As Found: Unknown
- ID: M2342
- Name: Immune Checkpoint Inhibitors
- Relevance: LOW
- As Found: Unknown
- ID: T447
- Name: Folinic Acid
- Relevance: LOW
- As Found: Unknown
- ID: T446
- Name: Folic Acid
- Relevance: LOW
- As Found: Unknown
- ID: T448
- Name: Folate
- Relevance: LOW
- As Found: Unknown
- ID: T475
- Name: Vitamin B9
- Relevance: LOW
- As Found: Unknown
- ID: T22
- Name: Tyrosine
- Relevance: LOW
- As Found: Unknown
- ID: T120
- Name: Cola
- Relevance: LOW
- As Found: Unknown
- ID: T468
- Name: Vitamin A
- Relevance: LOW
- As Found: Unknown
- ID: T462
- Name: Retinol
- Relevance: LOW
- As Found: Unknown
- ID: T418
- Name: Perillyl Alcohol
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: D000017239
- Term: Paclitaxel
- ID: D000068258
- Term: Bevacizumab
- ID: D000016190
- Term: Carboplatin
- ID: C000582435
- Term: Pembrolizumab
- ID: D000077594
- Term: Nivolumab
- ID: D000074324
- Term: Ipilimumab
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT00583479
**Brief Title:** Prospective Study of Celiac Block Injection: 1 vs. 2
**Official Title:** A Prospective Study of Celiac Block Technique: One Injection or Two?
#### Organization Study ID Info
**ID:** 0203-32
#### Organization
**Class:** OTHER
**Full Name:** Indiana University
### Status Module
#### Completion Date
**Date:** 2008-07
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2012-09-12
**Type:** ESTIMATED
**Last Update Submit Date:** 2012-09-11
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2008-07
**Type:** ACTUAL
#### Start Date
**Date:** 2002-06
**Status Verified Date:** 2012-09
#### Study First Post Date
**Date:** 2007-12-31
**Type:** ESTIMATED
**Study First Submit Date:** 2007-12-20
**Study First Submit QC Date:** 2007-12-20
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Indiana University
#### Responsible Party
**Old Name Title:** Dr. Julia LeBlanc, MD, MPH
**Old Organization:** Indiana University
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** The purpose of this prospective randomized study is to compare the clinical effectiveness of EUS-guided CB performed with a single injection versus two injections of medication into the celiac ganglion region.
### Conditions Module
**Conditions:**
- Chronic Pancreatitis
- Pancreatic Cancer
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** SINGLE
**Who Masked:**
- PARTICIPANT
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 100
**Type:** ESTIMATED
**Phases:**
- PHASE4
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** subjects who get one medication injection into the celiac ganglion during the EUS
**Intervention Names:**
- Other: one injection into the celiac ganglion
**Label:** A
**Type:** OTHER
#### Arm Group 2
**Description:** subjects who get divided dose of the medication injected into two locations within the celiac ganglion during the EUS
**Intervention Names:**
- Other: two injections into the celiac ganglion
**Label:** B
**Type:** OTHER
### Interventions
#### Intervention 1
**Arm Group Labels:**
- A
**Description:** one injection into the celiac ganglion of the standard medication for CB for with chronic pancreatitis or pancreatic cancer
**Name:** one injection into the celiac ganglion
**Type:** OTHER
#### Intervention 2
**Arm Group Labels:**
- B
**Description:** two injection into the celiac ganglion of the standard medication for CB for with chronic pancreatitis or pancreatic cancer
**Name:** two injections into the celiac ganglion
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Measure:** Compare the clinical effectiveness of EUS-guided CB performed with a single injection versus two injections of medication into the celiac ganglion region
**Time Frame:** 24-hour after the procedure; then every week thereafter until the subject is no longer in the study
#### Secondary Outcomes
**Measure:** change in medication use
**Time Frame:** at 24-hours after procedure and every week thereafter until the subject is no longer in the study
**Measure:** change is pain score
**Time Frame:** at 24-hours after procedure and every week thereafter until the subject is no longer in the study
**Measure:** complications related to CB
**Time Frame:** at 24-hours after procedure and every week thereafter until the subject is no longer in the study
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Patients with chronic abdominal pain from documented chronic pancreatitis or pancreatic cancer that are referred for EUS-guided celiac block will be eligible to participate in this study. Chronic pancreatitis and pancreatic cancer must be measured and documented by prior CT, ERCP, or EUS.
* Patients should not have significant co-morbidities such as uncontrolled heart failure, or severe chronic obstructive pulmonary disease (COPD) that would limit survivability of the sedation given or the procedure.
* Age \> or = to 18 years
* No evidence of significant active infection (ie. pneumonia, peritonitis, wound sepsis, etc)
* No evidence of serious ongoing illness such as uncontrolled metabolic disease (diabetes mellitus, hypothyroidism, etc)
* No evidence of dementia or altered mental status that would prohibit the giving and understanding of informed consent, and no evidence of psychiatric risk that would preclude adequate compliance with this protocol.
* Patient must provide signed written informed consent.
* Patients that have had a previous celiac plexus block are eligible for this study
Exclusion Criteria:
* The patient's celiac trunk should be imaged clearly under endoscopic ultrasound. The patient's celiac plexus should be accessed easily with a needle for the medication injection. If this cannot be done safely due to the patient's anatomy (intervening blood vessel or tumor), the patient will not be eligible for the procedure or study.
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Indianapolis
**Country:** United States
**Facility:** Clarian Health: Indiana University Hospital
**State:** Indiana
**Zip:** 46202
#### Overall Officials
**Official 1:**
**Affiliation:** Indiana University
**Name:** Julia LeBlanc, MD, MPH
**Role:** PRINCIPAL_INVESTIGATOR
### References Module
#### References
**Citation:** LeBlanc JK, Al-Haddad M, McHenry L, Sherman S, Juan M, McGreevy K, Johnson C, Howard TJ, Lillemoe KD, DeWitt J. A prospective, randomized study of EUS-guided celiac plexus neurolysis for pancreatic cancer: one injection or two? Gastrointest Endosc. 2011 Dec;74(6):1300-7. doi: 10.1016/j.gie.2011.07.073. Epub 2011 Oct 13.
**PMID:** 22000795
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000004066
- Term: Digestive System Diseases
- ID: D000010182
- Term: Pancreatic Diseases
- ID: D000002908
- Term: Chronic Disease
- ID: D000020969
- Term: Disease Attributes
- ID: D000010335
- Term: Pathologic Processes
### Condition Browse Module - Browse Branches
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: BC06
- Name: Digestive System Diseases
- Abbrev: BC19
- Name: Gland and Hormone Related Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: BC18
- Name: Nutritional and Metabolic Diseases
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M13110
- Name: Pancreatic Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M13115
- Name: Pancreatitis
- Relevance: HIGH
- As Found: Pancreatitis
- ID: M26260
- Name: Pancreatitis, Chronic
- Relevance: HIGH
- As Found: Chronic Pancreatitis
- ID: M5696
- Name: Celiac Disease
- Relevance: LOW
- As Found: Unknown
- ID: M7255
- Name: Digestive System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M8883
- Name: Gastrointestinal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M13102
- Name: Pancreatic Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M6147
- Name: Chronic Disease
- Relevance: LOW
- As Found: Unknown
- ID: M22700
- Name: Disease Attributes
- Relevance: LOW
- As Found: Unknown
- ID: T1303
- Name: Chronic Graft Versus Host Disease
- Relevance: LOW
- As Found: Unknown
- ID: T4387
- Name: Pancreatic Cancer
- Relevance: HIGH
- As Found: Pancreatic Cancer
### Condition Browse Module - Meshes
- ID: D000010195
- Term: Pancreatitis
- ID: D000050500
- Term: Pancreatitis, Chronic
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT01191879
**Brief Title:** A Comparison of p53-induced Genes Activation in Patients With and Without Acute Myocardial Infarction
**Official Title:** A Comparison of p53-induced Genes Activation as Possible Markers Differentiating Between Patients Presenting With Acute Myocardial Infarction and Controls
#### Organization Study ID Info
**ID:** MRNA-MI-1
#### Organization
**Class:** OTHER
**Full Name:** Meir Medical Center
### Status Module
#### Completion Date
**Date:** 2011-10
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2012-10-31
**Type:** ESTIMATED
**Last Update Submit Date:** 2012-10-30
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2011-10
**Type:** ACTUAL
#### Start Date
**Date:** 2010-11
**Status Verified Date:** 2012-10
#### Study First Post Date
**Date:** 2010-08-31
**Type:** ESTIMATED
**Study First Submit Date:** 2010-08-08
**Study First Submit QC Date:** 2010-08-30
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Meir Medical Center
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** The purpose of this study is to compare p53-induced genes activation as possible markers differentiating between patients presenting with acute myocardial infarction and controls.
**Detailed Description:** The diagnosis of acute myocardial infarction is based on the rise of bio-markers for cardiac necrosis such as troponin. While troponin measurement is highly sensitive for myocardial necrosis it has several limitations that influence its clinical use. First, since the troponin test is reliable only after 4-6 hours from symptoms onset, it has only limited value in the assessment of patients presenting earlier. Second, several clinical situations, most commonly renal dysfunction, are associated with increased troponin level and therefore may decrease the specificity of the test. Third, since troponin rise indicates myocardial infarction it is not useful in the common situations where there is myocardial ischemia without necrosis.
The P53 is a tumor suppressing gene activated in different stressful situations including hypoxia. This activation is associated with accelerated transcription (up to 30-50 folds from baseline) of different genes that are involved in apoptosis, DNA repair and in stopping the cell cycle. A study on pregnant women demonstrated high levels of fetal mRNA of these genes in maternal circulation. This gene expression correlated with other signs of fetal stress associated with hypoxia. Myocardial ischemia is another stressful event associated with tissue hypoxia. Nevertheless, the association of this gene expression with myocardial ischemia has not been investigated yet.
### Conditions Module
**Conditions:**
- Acute Myocardial Infarction
### Design Module
#### Design Info
**Observational Model:** CASE_CONTROL
**Time Perspective:** PROSPECTIVE
#### Enrollment Info
**Count:** 79
**Type:** ACTUAL
**Study Type:** OBSERVATIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Patients presenting with acute ST elevation myocardial infarction, admitted to the intensive cardiac care unit and that are planned for emergency primary PCI
**Intervention Names:**
- Other: Blood test
**Label:** acute MI group
#### Arm Group 2
**Description:** Patients undergoing a non-invasive evaluation of possible myocardial ischemia.
**Intervention Names:**
- Other: Blood test
**Label:** Controls
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Controls
- acute MI group
**Description:** Blood test
**Name:** Blood test
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Measure:** Expression of P53 induced genes in patients with and without acute myocardial infarction
**Time Frame:** Up to 4 hours following recruitment
#### Secondary Outcomes
**Measure:** Correlation between P53 associated gene expression with troponin level
**Time Frame:** After 5 days from recruitment
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
Acute MI group:
Patient presenting with chest pain lasting for at leasY 1 hour and no more than 6 hours accompanied by 1 of the following ECG criteria:
* ST segment elevation of anterior or inferior wall (at least 2 consecutive leads)
* New LBBB
Controls:
* Patients undergoing non-invasive evaluation of possible myocardial ischemia
Exclusion Criteria:
* Chronic lung disease requiring chronic treatment
* Any malignancy in the 5 year prior to enrollment
**Minimum Age:** 18 Years
**Sampling Method:** NON_PROBABILITY_SAMPLE
**Sex:** MALE
**Standard Ages:**
- ADULT
- OLDER_ADULT
**Study Population:** Aacute MI group- Patients with acute ST elevation MI planned for emergency primary PCI
Controls: Patients undergoing non-invasive evaluation of possible myocardial ischemia
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Kfar Saba
**Country:** Israel
**Facility:** Meir Medical Center
### References Module
#### References
**Citation:** Ashur-Fabian O, Avivi A, Trakhtenbrot L, Adamsky K, Cohen M, Kajakaro G, Joel A, Amariglio N, Nevo E, Rechavi G. Evolution of p53 in hypoxia-stressed Spalax mimics human tumor mutation. Proc Natl Acad Sci U S A. 2004 Aug 17;101(33):12236-41. doi: 10.1073/pnas.0404998101. Epub 2004 Aug 9.
**PMID:** 15302922
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000007511
- Term: Ischemia
- ID: D000010335
- Term: Pathologic Processes
- ID: D000009336
- Term: Necrosis
- ID: D000017202
- Term: Myocardial Ischemia
- ID: D000006331
- Term: Heart Diseases
- ID: D000002318
- Term: Cardiovascular Diseases
- ID: D000014652
- Term: Vascular Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC14
- Name: Heart and Blood Diseases
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: All
- Name: All Conditions
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M12155
- Name: Myocardial Infarction
- Relevance: HIGH
- As Found: Myocardial Infarction
- ID: M10282
- Name: Infarction
- Relevance: HIGH
- As Found: Infarction
- ID: M10543
- Name: Ischemia
- Relevance: LOW
- As Found: Unknown
- ID: M12284
- Name: Necrosis
- Relevance: LOW
- As Found: Unknown
- ID: M6546
- Name: Coronary Artery Disease
- Relevance: LOW
- As Found: Unknown
- ID: M19506
- Name: Myocardial Ischemia
- Relevance: LOW
- As Found: Unknown
- ID: M9419
- Name: Heart Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M17400
- Name: Vascular Diseases
- Relevance: LOW
- As Found: Unknown
- ID: T170
- Name: Acute Graft Versus Host Disease
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000009203
- Term: Myocardial Infarction
- ID: D000007238
- Term: Infarction
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT02325479
**Brief Title:** Local Uterine Application of Low Molecular Weight Heparin (LMWH) in Intra-cytoplasmic Sperm Injection (ICSI)
**Official Title:** Local Uterine Application of Low Molecular Weight Heparin (LMWH) in Intra-cytoplasmic Sperm Injection (ICSI) a Randomised Controlled Pilot Study
#### Organization Study ID Info
**ID:** 152014
#### Organization
**Class:** OTHER
**Full Name:** The Egyptian IVF-ET Center
### Status Module
#### Completion Date
**Date:** 2015-12
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2016-02-18
**Type:** ESTIMATED
**Last Update Submit Date:** 2016-02-17
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2015-05
**Type:** ACTUAL
#### Start Date
**Date:** 2015-01
**Status Verified Date:** 2016-02
#### Study First Post Date
**Date:** 2014-12-25
**Type:** ESTIMATED
**Study First Submit Date:** 2014-12-17
**Study First Submit QC Date:** 2014-12-21
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** The Egyptian IVF-ET Center
#### Responsible Party
**Investigator Affiliation:** The Egyptian IVF-ET Center
**Investigator Full Name:** Mona Aboulghar
**Investigator Title:** Professor Obstetrics and Gynecology Fetal Medicine unit Cairo University, IVF Consultant Egyptian IVF center -Maadi Cairo
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** Heparin also has the ability to bind with and modulate a wide variety of proteins, which can influence a number of physiological processes involved in implantation \& trophoblastic development. These processes include adhesion of the blastocyst to the endometrial surface \& trophoblastic differentiation \& invasion.
A recent Cochrane review hinted towards research to study the possible effects of the local (uterine), \& NOT SYSTEMIC application of heparin during Assisted reproductive technology (ART). Based on the above evidence the investigators hypothesize that low molecular weight heparin given intrauterine at mock embryo transfer after oocyte pick up will help improve pregnancy rates in patients undergoing ART.
**Detailed Description:** Following ethical approval of the committee of Egyptian IVF-ET center, a pilot prospective randomized controlled study will include 40 patients, in which the study arm (group A ) will be injected with LMWH intrauterine during mock embryo transfer, just after ovum pickup, while the control arm (group B) will be injected intrauterine with similar volume tissue culture media (G.2 plus ref. 10132, Vitrolife) also after ovum pickup. Admission into either group will be through randomization after inclusion \& exclusion criteria have been met and before start of controlled ovarian stimulation protocol.
The standard long gonadotrophin-releasing hormone (GnRH) agonist protocol will be used for patients with predicted normal response based on clinical \& hormonal profile ; 1 mg of leuprolide acetate daily s.c injection (Lucrin ®; Abbott, Hoofddorp, The Netherlands) is applied from the mid luteal phase onward till the day of human chorionic gonadotropin (HCG) injection.
Gonadotropins in the form of human menopausal gonadotropin (HMG) (Merional ®, IBSA, Institut Biochimique SA, Lugano, Switzerland) will be given by intramuscular injection (IM) from the 2nd day of menstruation, The starting dose range from 150 to 450 IU depending on the basal follicle stimulating hormone (FSH) level, Antral follicle count (AFC) , Patient's age and body mass index (BMI).
In all protocols, stimulation is monitored by trans vaginal ultrasonography and serial estradiol (E2) measurements starting from day 7 of the cycle and the gonadotropin dose is adjusted individually according to follicular response.
After the development of at least three leading follicles≥18 mm, 10,000 unit of HCG (Choriomon, IBSA, Institut Biochimique SA) is given IM, and a trans-vaginal ultrasound-guided oocyte retrieval is performed 36 hours later.
After scheduled oocyte pick up, a mock embryo transfer will be done using labotec catheter (Labotec, Gottingen Germany), and an injection of Enaoxaprin sodium (LMWH) (Clexane® Sanofi S.A Paris, France) will be given intrauterine in group A patients.
LMWH is safe in pregnancy, category B drug , and given empirically sometimes in luteal phase and in early 1st trimester without reported problems, it has a half life of 4 Hours, and therefore it should not have any negative effects on developing embryos as it will not be present at the time of transfer while already performing it s desired effect on the endometrium.
The dose of LMWH given was calculated comparatively according to the work done by our group Mansour et.al, 2011, where intrauterine administration 500 IU of HCG improved the implantation and pregnancy rates.
Given the fact that this has not been attempted before, the best way to compare two different drugs is through biological activity. So if the investigators want to use 500 IU of LMWH, and knowing that In the in vitro purified system, enoxaparin sodium has a high anti Xa activity (approximately 100 IU/mg), so to get 500 IU of LMWH we need 5mg. Clexane 20mg is packed in a 0.2ml syringe, And therefore the investigators need to inject 0.05ml intrauterine using labotec catheter. (1/4 OF THE ORIGINAL SYRINGES CONTENT..i.e 5mg).
The control arm (group B) will be injected intrauterine with similar volume of tissue culture media (G.2 plus ref. 10132, Vitrolife) Oocytes are then fertilized in vitro using ICSI and after three to five days embryo transfer will be done using labotec catheter (Labotec, Gottingen Germany) with ultrasound guidance.
Progesterone pessaries 400 mg twice daily (Cyclogest 400mg ®Actavis plc. Dublin, Ireland) is given as a luteal support starting from the day of embryo transfer and continued for 16 days after.
Pregnancy is defined as the occurrence of a positive β-HCG \>10 IU on day 12 after embryo transfer and a second higher value 2 days later, followed by ultrasonography confirmation of cardiac activity at 6 weeks gestation
A third party not involved in the actual study will moniter progress of the results and record them. Study will be stopped if more than 10 consecutive patients fail to get pregnant in the LMWH group. If the pregnancy rate \>25% in the intervention group by the end of 20 cases, then recruitment willl continue till 60 patients are enrolled on each study arm.
### Conditions Module
**Conditions:**
- Infertility
**Keywords:**
- Low Molecular Weight (LMWH) Heparin ICSI Enaoxaprin sodium
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** TRIPLE
**Who Masked:**
- PARTICIPANT
- CARE_PROVIDER
- INVESTIGATOR
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 120
**Type:** ACTUAL
**Phases:**
- PHASE1
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** After scheduled oocyte pick up, a mock embryo transfer will be done using labotec catheter (Labotec, Gottingen Germany), and an injection of Enaoxaprin sodium (LMWH) (Clexane® Sanofi S.A Paris, France) will be given intrauterine in group A patients, 500 IU of LMWH which is 5mg, in 0.05 ml.
**Intervention Names:**
- Drug: Enaoxaprin sodium
**Label:** Group A
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** The control arm (group B) will be injected intrauterine with similar volume of tissue culture media (G.2 plus ref. 10132, Vitrolife)
**Intervention Names:**
- Drug: tissue culture media
**Label:** Group B
**Type:** PLACEBO_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Group A
**Description:** intrauterine administration after oocyte pickup as the main experimental intervention
**Name:** Enaoxaprin sodium
**Other Names:**
- Clexane
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- Group B
**Description:** intrauterine administration after oocyte pickup as placebo
**Name:** tissue culture media
**Other Names:**
- (G.2 plus ref. 10132, Vitrolife)
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Description:** safety
**Measure:** pregnancy rate
**Time Frame:** 14 days
#### Secondary Outcomes
**Measure:** Early miscarriage rate
**Time Frame:** 12 weeks
**Measure:** Implanation rate
**Time Frame:** 3 weeks after ET
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
1. Normal serum prolactin level \& thyroid stimulating hormone (TSH)
2. Unexplained infertility
3. Tubal factor infertility
4. BMI \<30 kg/m2
Exclusion Criteria:
1. Reduced ovarian reserve by AFC, Anti-mullerian hormone (AMH)
2. Presence of non disconnected hydrosalpinges
3. Frozen embryo transfer cycles
4. Uterine Anomalies
5. Submucous fibroids and polyps
6. Uterine synechia
7. Contraindication of pregnancy e.g.: Somatic and mental diseases, which are contraindications for carrying of a pregnancy and childbirth, inborn malformations or acquired deformations of uterus cavity which make embryo implantation or carrying of a pregnancy impossible ,ovarian tumors
**Healthy Volunteers:** True
**Maximum Age:** 37 Years
**Minimum Age:** 20 Years
**Sex:** FEMALE
**Standard Ages:**
- ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Cairo
**Country:** Egypt
**Facility:** The Egyptian IVF-ET Center
**State:** Maadi
**Zip:** 11431
#### Overall Officials
**Official 1:**
**Affiliation:** IVF consultant
**Name:** Mona M Aboulghar, M.D
**Role:** PRINCIPAL_INVESTIGATOR
### References Module
#### References
**Citation:** Akhtar MA, Sur S, Raine-Fenning N, Jayaprakasan K, Thornton J, Quenby S, Marjoribanks J. Heparin for assisted reproduction: summary of a Cochrane review. Fertil Steril. 2015 Jan;103(1):33-4. doi: 10.1016/j.fertnstert.2014.09.005. Epub 2014 Oct 1.
**PMID:** 25282470
**Citation:** Mansour R, Tawab N, Kamal O, El-Faissal Y, Serour A, Aboulghar M, Serour G. Intrauterine injection of human chorionic gonadotropin before embryo transfer significantly improves the implantation and pregnancy rates in in vitro fertilization/intracytoplasmic sperm injection: a prospective randomized study. Fertil Steril. 2011 Dec;96(6):1370-1374.e1. doi: 10.1016/j.fertnstert.2011.09.044. Epub 2011 Nov 1.
**PMID:** 22047664
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000091662
- Term: Genital Diseases
- ID: D000091642
- Term: Urogenital Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: All
- Name: All Conditions
- Abbrev: BXS
- Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions
### Condition Browse Module - Browse Leaves
- ID: M5114
- Name: Body Weight
- Relevance: LOW
- As Found: Unknown
- ID: M10290
- Name: Infertility
- Relevance: HIGH
- As Found: Infertility
- ID: M2876
- Name: Genital Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M2875
- Name: Urogenital Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000007246
- Term: Infertility
### Intervention Browse Module - Ancestors
- ID: D000000925
- Term: Anticoagulants
- ID: D000005343
- Term: Fibrinolytic Agents
- ID: D000050299
- Term: Fibrin Modulating Agents
- ID: D000045504
- Term: Molecular Mechanisms of Pharmacological Action
### Intervention Browse Module - Browse Branches
- Abbrev: FiAg
- Name: Fibrinolytic Agents
- Abbrev: AnCoag
- Name: Anticoagulants
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M9579
- Name: Heparin
- Relevance: LOW
- As Found: Unknown
- ID: M20152
- Name: Enoxaparin
- Relevance: HIGH
- As Found: World Health Organization
- ID: M46053
- Name: Calcium heparin
- Relevance: LOW
- As Found: Unknown
- ID: M9581
- Name: Heparin, Low-Molecular-Weight
- Relevance: LOW
- As Found: Unknown
- ID: M1943
- Name: Tinzaparin
- Relevance: LOW
- As Found: Unknown
- ID: M20153
- Name: Dalteparin
- Relevance: LOW
- As Found: Unknown
- ID: M137075
- Name: Enoxaparin sodium
- Relevance: LOW
- As Found: Unknown
- ID: M4244
- Name: Anticoagulants
- Relevance: LOW
- As Found: Unknown
- ID: M8473
- Name: Fibrinolytic Agents
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: D000017984
- Term: Enoxaparin
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT04647279
**Acronym:** NMSSJ
**Brief Title:** New MRI Sequences in Spine and Joint
**Official Title:** Clinical Study of New MRI Sequences in Spine and Joint
#### Organization Study ID Info
**ID:** ZDWY.FSK.004
#### Organization
**Class:** OTHER
**Full Name:** Fifth Affiliated Hospital, Sun Yat-Sen University
### Status Module
#### Completion Date
**Date:** 2022-12-31
**Type:** ESTIMATED
#### Expanded Access Info
**Last Known Status:** RECRUITING
#### Last Update Post Date
**Date:** 2021-04-06
**Type:** ACTUAL
**Last Update Submit Date:** 2021-04-05
**Overall Status:** UNKNOWN
#### Primary Completion Date
**Date:** 2022-07-31
**Type:** ESTIMATED
#### Start Date
**Date:** 2020-06-01
**Type:** ACTUAL
**Status Verified Date:** 2021-04
#### Study First Post Date
**Date:** 2020-11-30
**Type:** ACTUAL
**Study First Submit Date:** 2020-11-27
**Study First Submit QC Date:** 2020-11-27
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Fifth Affiliated Hospital, Sun Yat-Sen University
#### Responsible Party
**Investigator Affiliation:** Fifth Affiliated Hospital, Sun Yat-Sen University
**Investigator Full Name:** ShaoLin Li
**Investigator Title:** Director of Radiology Department
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
### Description Module
**Brief Summary:** Low back pain and osteoarticular degeneration or injury is the leading worldwide cause of years lost to disability, accounting for 17 % of all patients with disabilities and its burden is growing alongside the increasing and aging population. The anatomical regions of the intervertebral disc include the central nucleus pulposus, the peripheral fibrocartilaginous annulus fibrosus, and the superior and inferior cartilaginous endplates (CEP). The CEP is a thin layer of hyaline cartilage located between the avascular intervertebral disc and the bony vertebral endplate. The endplate cartilage consists of chondrocytes interspersed throughout an extracellular matrix of proteoglycans, collagen (types I and II), and water. It plays an important role in the function and homeostasis. The CEP has been considered the pathway between the largely avascular disk tissues and the blood supply of the vertebral body and thus provides nutrition for disk cells. Many musculoskeletal tissues, including the CEP, cartilage-bone interface of articular joints, entheses, tendons, and ligaments, have components with very short T2 values (much less than 1 msec), which are orders of magnitude shorter than that of the nucleus of the disk (\~100 msec). In a conventional pulse sequence, such as proton density-weighted spin-echo (SE) or fast SE, with standard clinical section profile, the minimum echo time (TE) is typically 10 msec, which is much longer than that needed to capture the short-lived signal from these tissues. Recently, ultrashort echo sequence UTE technology has been introduced, and the TE time can be as low as 0.008ms. This range of TE is sufficient to capture signals from the cartilage endplate before it decays. With using new MRI technology, such as IR-UTE, UTE-MT, UTE-T2\*mapping, Maigc, DTI, and IVIM, which can quantitative tissues that have previously been "invisible" at conventional MR imaging, and provide imaging basis for early diagnosis of injury at molecular level. The purpose of this study was to investigate the clinical application value of different MRI sequences in spine and joint.
### Conditions Module
**Conditions:**
- Degeneration of Spine and Osteoarticular
**Keywords:**
- Magnetic resonance imaging
- Low back pain
- Disk degeneration
- Ultrashort echo time
- Osteoarticular degeneration
### Design Module
#### Design Info
**Observational Model:** OTHER
**Time Perspective:** PROSPECTIVE
#### Enrollment Info
**Count:** 300
**Type:** ESTIMATED
**Patient Registry:** True
**Study Type:** OBSERVATIONAL
**Target Duration:** 2 Years
### Arms Interventions Module
#### Arm Group 1
**Description:** Diagnostic Test: Quantitative MRI imaging IR-UTE, UTE-MT, Maigc, Ideal IQ, DTI, and IVIM
**Intervention Names:**
- Diagnostic Test: Dual-emission X-ray Absorptiometry
**Label:** Normal control group
#### Arm Group 2
**Description:** Diagnostic Test: Quantitative MRI imaging IR-UTE, UTE-MT, Maigc, Ideal IQ, DTI, and IVIM
**Intervention Names:**
- Diagnostic Test: Dual-emission X-ray Absorptiometry
**Label:** Osteopenia
#### Arm Group 3
**Description:** Diagnostic Test: Quantitative MRI imaging IR-UTE, UTE-MT, Maigc, Ideal IQ, DTI, and IVIM
**Intervention Names:**
- Diagnostic Test: Dual-emission X-ray Absorptiometry
**Label:** Osteoporosis
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Normal control group
- Osteopenia
- Osteoporosis
**Description:** The recruiters were divided into osteoporosis, osteopenia and normal by dual emission X-ray absorptiometry.
**Name:** Dual-emission X-ray Absorptiometry
**Type:** DIAGNOSTIC_TEST
### Outcomes Module
#### Primary Outcomes
**Description:** Quantitative MRI imaging(such as IR-UTE, UTE-MT, UTE-T2\*mapping, Maigc, Ideal IQ, DTI, and IVIM) used to quantitative diagnosis of disk degeneration, osteoporosis, osteoarticular degeneration etc.
**Measure:** New MRI sequences in the diagnosis of spine and joint
**Time Frame:** 2 years
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* 1.The patient had history and clinical symptoms of low back pain and joint sports injury 2.The patient had a history of osteoporosis; 3.There was no contraindication of MRI; 4.The patient who has clear lumbar intervertebral disc degeneration or herniation or osteoporosis or joint sports injury found by routine CT or MRI examination, and the clinical symptoms were consistent with the image.
Exclusion Criteria:
* 1. Patients with previous history of lumbar or joint surgery; 2. Patients with congenital bone deformity; 3. Patients with history of lumbar tumor and infection; 4. Cognitive function is limited or mental illness can not cooperate with imaging.
**Healthy Volunteers:** True
**Maximum Age:** 80 Years
**Minimum Age:** 18 Years
**Sampling Method:** NON_PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
**Study Population:** All suspected patients with osteoporosis and low back pain meet the inclusion criteria will include in this study. And patients who meet the criteria of the normal control group will include in this study.
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Jin Liu, Master
**Phone:** 0086 756 2528321
**Role:** CONTACT
**Contact 2:**
**Email:** [email protected]
**Name:** Shaolin Li, Director
**Phone:** 0086 756 2528321
**Role:** CONTACT
#### Locations
**Location 1:**
**City:** Zhuhai
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Jin Liu, Master
- **Phone:** 0086 756 2528321
- **Role:** CONTACT
***Contact 2:***
- **Email:** [email protected]
- **Name:** Shaolin Li, Doctor
- **Phone:** 0086 756 2528321
- **Role:** CONTACT
**Country:** China
**Facility:** The Fifth Affiliated Hospital of Sun Yat-sen University
**State:** Guangdong
**Status:** RECRUITING
**Zip:** 519000
### IPD Sharing Statement Module
**IPD Sharing:** NO
### References Module
#### References
**Citation:** Kawakami M, Tamaki T, Hayashi N, Hashizume H, Nishi H. Possible mechanism of painful radiculopathy in lumbar disc herniation. Clin Orthop Relat Res. 1998 Jun;(351):241-51.
**PMID:** 9646768
**Citation:** Bae WC, Statum S, Zhang Z, Yamaguchi T, Wolfson T, Gamst AC, Du J, Bydder GM, Masuda K, Chung CB. Morphology of the cartilaginous endplates in human intervertebral disks with ultrashort echo time MR imaging. Radiology. 2013 Feb;266(2):564-74. doi: 10.1148/radiol.12121181. Epub 2012 Nov 28.
**PMID:** 23192776
**Citation:** Freburger JK, Holmes GM, Agans RP, Jackman AM, Darter JD, Wallace AS, Castel LD, Kalsbeek WD, Carey TS. The rising prevalence of chronic low back pain. Arch Intern Med. 2009 Feb 9;169(3):251-8. doi: 10.1001/archinternmed.2008.543.
**PMID:** 19204216
**Citation:** Lotz JC, Fields AJ, Liebenberg EC. The role of the vertebral end plate in low back pain. Global Spine J. 2013 Jun;3(3):153-64. doi: 10.1055/s-0033-1347298. Epub 2013 May 23.
**PMID:** 24436866
**Citation:** Gatehouse PD, Bydder GM. Magnetic resonance imaging of short T2 components in tissue. Clin Radiol. 2003 Jan;58(1):1-19. doi: 10.1053/crad.2003.1157.
**PMID:** 12565203
**Citation:** Bae WC, Dwek JR, Znamirowski R, Statum SM, Hermida JC, D'Lima DD, Sah RL, Du J, Chung CB. Ultrashort echo time MR imaging of osteochondral junction of the knee at 3 T: identification of anatomic structures contributing to signal intensity. Radiology. 2010 Mar;254(3):837-45. doi: 10.1148/radiol.09081743.
**PMID:** 20177096
**Citation:** Bae WC, Du J, Bydder GM, Chung CB. Conventional and ultrashort time-to-echo magnetic resonance imaging of articular cartilage, meniscus, and intervertebral disk. Top Magn Reson Imaging. 2010 Oct;21(5):275-89. doi: 10.1097/RMR.0b013e31823ccebc.
**PMID:** 22129641
**Citation:** Pfirrmann CW, Metzdorf A, Zanetti M, Hodler J, Boos N. Magnetic resonance classification of lumbar intervertebral disc degeneration. Spine (Phila Pa 1976). 2001 Sep 1;26(17):1873-8. doi: 10.1097/00007632-200109010-00011.
**PMID:** 11568697
**Citation:** GBD 2015 Disease and Injury Incidence and Prevalence Collaborators. Global, regional, and national incidence, prevalence, and years lived with disability for 310 diseases and injuries, 1990-2015: a systematic analysis for the Global Burden of Disease Study 2015. Lancet. 2016 Oct 8;388(10053):1545-1602. doi: 10.1016/S0140-6736(16)31678-6. Erratum In: Lancet. 2017 Jan 7;389(10064):e1.
**PMID:** 27733282
**Citation:** Hoy D, Bain C, Williams G, March L, Brooks P, Blyth F, Woolf A, Vos T, Buchbinder R. A systematic review of the global prevalence of low back pain. Arthritis Rheum. 2012 Jun;64(6):2028-37. doi: 10.1002/art.34347. Epub 2012 Jan 9.
**PMID:** 22231424
## Derived Section
### Condition Browse Module - Browse Branches
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: All
- Name: All Conditions
- Abbrev: BC05
- Name: Musculoskeletal Diseases
### Condition Browse Module - Browse Leaves
- ID: M4714
- Name: Back Pain
- Relevance: LOW
- As Found: Unknown
- ID: M19433
- Name: Low Back Pain
- Relevance: LOW
- As Found: Unknown
- ID: M13066
- Name: Pain
- Relevance: LOW
- As Found: Unknown
- ID: M28405
- Name: Intervertebral Disc Degeneration
- Relevance: LOW
- As Found: Unknown
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT04827979
**Acronym:** ATTAIN
**Brief Title:** Daratumumab and Belatacept for Desensitization
**Official Title:** A Mechanistically Driven Therapy to Desensitize >98.0% cPRA Patients: Depletion of Plasma Cells With Anti-CD38 and Prevention of B Cell Activation With Costimulation Blockade (ITN090ST)
#### Organization Study ID Info
**ID:** DAIT ITN090ST
#### Organization
**Class:** NIH
**Full Name:** National Institute of Allergy and Infectious Diseases (NIAID)
#### Secondary ID Infos
**ID:** UM1AI109565
**Link:** https://reporter.nih.gov/quickSearch/UM1AI109565
**Type:** NIH
**Domain:** DAIT NIAID
**ID:** NIAID CRMS ID#: 38686
**Type:** OTHER
### Status Module
#### Completion Date
**Date:** 2028-04-30
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-01-26
**Type:** ACTUAL
**Last Update Submit Date:** 2024-01-24
**Overall Status:** RECRUITING
#### Primary Completion Date
**Date:** 2027-03-31
**Type:** ESTIMATED
#### Start Date
**Date:** 2021-11-01
**Type:** ACTUAL
**Status Verified Date:** 2024-01
#### Study First Post Date
**Date:** 2021-04-01
**Type:** ACTUAL
**Study First Submit Date:** 2021-03-30
**Study First Submit QC Date:** 2021-03-30
### Sponsor Collaborators Module
#### Collaborators
**Class:** NETWORK
**Name:** Immune Tolerance Network (ITN)
**Class:** INDUSTRY
**Name:** Bristol-Myers Squibb
**Class:** INDUSTRY
**Name:** PPD
**Class:** INDUSTRY
**Name:** Rho Federal Systems Division, Inc.
#### Lead Sponsor
**Class:** NIH
**Name:** National Institute of Allergy and Infectious Diseases (NIAID)
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** True
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** Some kidney transplant candidates have a very low chance of getting a kidney transplant because their immune systems are "highly sensitized" to most kidney donors. Being "highly sensitized" means that they will likely have to wait a long time (more than 5 years) before an acceptable donor is found for them or, they never receive a compatible donor, and die on waitlist. The purpose of this study is to find out whether two drugs, daratumumab (Darzalex®), and belatacept (Nulojix®), can make these kidney transplant candidates less sensitized, and make it easier and quicker to find a kidney donor for them.
**Detailed Description:** This study will enroll 15 eligible adult participants with end stage renal failure on dialysis who are on the waiting list for a deceased donor transplant with calculated panel reactive antibodies (cPRA) ≥99.9% or \>98% (with \>5 years of waiting time) or, those with cPRA \>98% and an human leukocyte antigen (HLA)-incompatible approved living donor who have not received a transplant after 1 year in a paired kidney exchange program. The study will evaluate whether the study treatment is safe and can lower the participant's immune system's sensitization to kidney donors, making it easier to find a well-matched kidney for them.
The study treatment is comprised of two drugs, Darzalex® (daratumumab) and Nulojix® (belatacept). Daratumumab is licensed for treatment of multiple myeloma and belatacept is licensed for prevention of rejection after kidney transplant. Eligible participants will receive infusions of daratumumab and belatacept over a 10-week period in Cohort 1. Eligible participants will receive infusions of daratumumab and belatacept over a 14-week period in Cohort 2. An interim safety and efficacy analysis will occur after the first 5 participants have received study treatment. All subjects will undergo HLA antibody assessments and bone marrow aspiration prior to and after completion of treatment and receive 42 weeks of follow up after completing treatment. Participants who prematurely discontinue study therapy will receive follow up through 56 weeks after their baseline visit. Subjects who receive a kidney transplant while in the study will receive standard of care immunosuppression and undergo 52 weeks of follow up. Living donors will participate for one study visit to provide blood collection.
### Conditions Module
**Conditions:**
- Highly Sensitized Prospective Kidney Transplant Recipients
**Keywords:**
- calculated panel reactive antibodies (cPRA)
- desensitization therapy
- human leukocyte antigen (HLA) desensitization
### Design Module
#### Design Info
**Allocation:** NON_RANDOMIZED
**Intervention Model:** SEQUENTIAL
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 15
**Type:** ESTIMATED
**Phases:**
- PHASE1
- PHASE2
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Multiple intravenous infusions of daratumumab and belatacept over 10 weeks:
* Daratumumab will be administered intravenously at a dose of 8 mg/kg weekly for 4 weeks, then every other week for 4 weeks (week 9 and week 11). The dose administered will be calculated based on the actual body weight of the subject at each visit.
* Belatacept will be administered intravenously at a dose of 10 mg/kg every 2 weeks starting at week 8 (dosed at weeks 8, 10, 12, and 14). The total infusion dose of belatacept will be based on the actual body weight of the subject at the baseline visit, and will not be modified during the course of therapy, unless there is a change in body weight of greater than 10%.
**Intervention Names:**
- Biological: daratumumab
- Biological: belatacept
- Procedure: Bone marrow aspiration
**Label:** Cohort 1 (N=5 Subjects)
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** The enrollment of ten additional subjects is dependent on the results in Cohort 1.
Multiple intravenous infusions of daratumumab and belatacept over 14 weeks:°
* Daratumumab will be administered intravenously at a dose of 8 mg/kg for the first dose, then 16 mg/kg for subsequent given weekly for 3 weeks, then every 2 weeks for 2 doses (week 9 and week 11). The dose administered will be calculated based on the actual body weight of the subject at each visit.
* Belatacept will be administered intravenously at a dose of 10 mg/kg every 2 weeks starting at week 8 (dosed at weeks 8, 10, 12, 14, 16 and 18). The total infusion dose of belatacept will be based on the actual body weight of the subject at the baseline visit and will not be modified during the course of therapy, unless there is a change in body weight of greater than 10%.
* Was modified based on the safety and efficacy analysis of Cohort 1.
**Intervention Names:**
- Biological: daratumumab
- Biological: belatacept
- Procedure: Bone marrow aspiration
**Label:** Cohort 2 (N=10 Subjects)
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Cohort 1 (N=5 Subjects)
- Cohort 2 (N=10 Subjects)
**Description:** Daratumumab is a CD38 (Cluster of Differentiation 38)-directed cytolytic monoclonal antibody indicated for the treatment of multiple myeloma. In this study, daratumumab will be used in highly sensitized subjects without myeloma who are awaiting a kidney transplant.
Definition of highly sensitized: Potential kidney transplant recipients with either:
* calculated panel reactive antibodies (cPRA) ≥99.9% awaiting deceased donor transplant, or
* cPRA \>98% (with \>5 years of waiting time) awaiting living donor transplant
**Name:** daratumumab
**Other Names:**
- Darzalex®
- immunoglobulin G1 kappa human monoclonal antibody
- IgG1k human mAb
**Type:** BIOLOGICAL
#### Intervention 2
**Arm Group Labels:**
- Cohort 1 (N=5 Subjects)
- Cohort 2 (N=10 Subjects)
**Description:** Belatacept, a monoclonal antibody, is indicated for the prophylaxis of organ rejection in adult patients receiving a kidney transplant. In this study, belatacept will be used in subjects who have not received a kidney transplant.
**Name:** belatacept
**Other Names:**
- Nulojix®
**Type:** BIOLOGICAL
#### Intervention 3
**Arm Group Labels:**
- Cohort 1 (N=5 Subjects)
- Cohort 2 (N=10 Subjects)
**Description:** Subjects will undergo a bone marrow aspiration prior to starting the study regimen and at 12 weeks after starting the study regimen. In subjects who undergo a kidney transplant during the study, another bone marrow aspiration will be done if it has been \>4 weeks since the previous bone marrow aspiration.
**Name:** Bone marrow aspiration
**Type:** PROCEDURE
### Outcomes Module
#### Primary Outcomes
**Description:** Proportion of subjects who have not met a subject stopping rule, and remain free of all of the following through 26 weeks after starting treatment or until receiving a transplant, whichever occurs earlier:
1. Grade 3 or higher infusion reaction
2. Grade 3 or higher infections
3. Any malignancy
The study site will grade the severity of adverse events experienced by the study subjects according to the criteria set forth in the National Cancer Institute's Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0 (Published November 27, 2017).
**Measure:** Proportion of subjects who have not met a subject stopping rule and remain free of all of the safety events listed in the outcome description, through 26 weeks after starting treatment or until receiving a transplant, whichever occurs earlier
**Time Frame:** Baseline up to 26 weeks post treatment initiation
**Description:** Proportion of subjects who meet any one of the following compared to Baseline (Visit 0):
1. Elimination of one human leukocyte antigen (HLA) antibody at Visit 12 (16 weeks ±7 days after starting treatment);
2. 50% or greater reduction in the mean fluorescence intensity (MFI) of at least three HLA antibodies at Visit 12 (16 weeks ±7 days after starting treatment); and/or
3. Kidney transplant with a previously incompatible donor within 26 weeks after starting treatment without graft loss due to acute antibody mediated rejection occurring within the first four weeks post-transplant and caused by an anamnestic response.
**Measure:** Proportion of subjects who meet any one of the pre-specified events detailed in the outcome description: from Baseline up to Week 26 - Cohort 1
**Time Frame:** Baseline (Visit 0) up to 26 weeks post treatment initiation
**Description:** Proportion of subjects who meet any one of the following compared to Baseline (Visit 0):
1. Elimination of one human leukocyte antigen (HLA) antibody at Visit 14 (26 weeks ±7 days after starting treatment);
2. 50% or greater reduction in the mean fluorescence intensity (MFI) of at least three HLA antibodies at Visit 14 (26 weeks ±7 days after starting treatment); and/or
3. Kidney transplant with a previously incompatible donor within 52 weeks after starting treatment without graft loss due to acute antibody mediated rejection occurring within the first four weeks post-transplant and caused by an anamnestic response.
**Measure:** Proportion of subjects who meet any one of the pre-specified events detailed in the outcome description: from Baseline up to Week 26 - Cohort 2
**Time Frame:** Baseline (Visit 0) up to 52 weeks post treatment initiation
#### Secondary Outcomes
**Description:** Proportion of subjects transplanted with a donor, to whom Donor Specific Antibodies (DSA) was previously positive and had reduced by \>/= 50% reduction at the time of transplant, within 52 weeks after starting treatment
Subjects may receive a kidney transplant while in the study, either from a living or deceased donor to whom they were previously compatible, or from a previously incompatible donor in case there is a significant reduction in HLA antibody.
**Measure:** Proportion of subjects transplanted with a previously incompatible donor within 52 weeks after starting treatment - Cohort 1
**Time Frame:** Within 52 weeks post treatment initiation
**Description:** Antibody mediated rejection (AMR) is an important cause of graft loss after organ transplantation and is caused by anti-donor-specific antibodies, especially anti- human leukocyte antigen (HLA) antibodies.
**Measure:** Proportion of subjects with biopsy-proven acute or chronic antibody mediated rejection (AMR) within 52 weeks post-transplant in subjects who undergo a kidney transplant
**Time Frame:** Within 52 weeks post-transplant
**Description:** Antibody mediated rejection (AMR) is an important cause of graft loss after organ transplantation and is caused by anti-donor-specific antibodies, especially anti- human leukocyte antigen (HLA) antibodies.
**Measure:** Number of biopsy-proven acute or chronic AMR events within 52 weeks post-transplant in subjects who undergo a kidney transplant
**Time Frame:** Within 52 weeks post-transplant
**Description:** A measure of infection-related morbidity.
**Measure:** Incidence of invasive fungal infections, mycobacterial infections or Pneumocystis jirovecii infection within 16 weeks after starting treatment
**Time Frame:** Within 16 weeks post treatment initiation
**Description:** A measure of infection-related morbidity.
**Measure:** Incidence of invasive fungal infections, mycobacterial infections or Pneumocystis jirovecii infection within 26 weeks after starting treatment
**Time Frame:** Within 26 weeks post treatment initiation
**Description:** A measure of infection-related morbidity.
**Measure:** Incidence of invasive fungal infections, mycobacterial infections or Pneumocystis jirovecii infection within 52 weeks after starting treatment
**Time Frame:** Within 52 weeks post treatment initiation
**Description:** CMV disease defined by the presence of detectable CMV in the blood and the presence of other clinical manifestations attributable to the CMV virus.
**Measure:** Incidence of cytomegalovirus (CMV) disease within 16 weeks after starting treatment
**Time Frame:** Within 16 weeks post treatment initiation
**Description:** CMV disease defined by the presence of detectable CMV in the blood and the presence of other clinical manifestations attributable to the CMV virus.
**Measure:** Incidence of cytomegalovirus (CMV) disease within 26 weeks after starting treatment
**Time Frame:** Within 26 weeks post treatment initiation
**Description:** CMV disease defined by the presence of detectable CMV in the blood and the presence of other clinical manifestations attributable to the CMV virus.
**Measure:** Incidence of cytomegalovirus (CMV) disease within 52 weeks after starting treatment
**Time Frame:** Within 52 weeks post treatment initiation
**Description:** CMV infection confirmed by the presence of detectable CMV in blood by polymerase chain reaction \[PCR\] diagnostic testing, regardless of whether signs or symptoms are present.
**Measure:** Incidence of cytomegalovirus (CMV) infection within 52 weeks after starting treatment
**Time Frame:** Within 52 weeks post treatment initiation
**Description:** As per diagnosis by local pathologist and treating physician.
**Measure:** Incidence of post-transplant lymphoproliferative disorder (PTLD)
**Time Frame:** Within 52 weeks post-transplant
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
Individuals who meet all of the following criteria are eligible for enrollment as study subjects-
1. Subject must be able to understand and provide informed consent
2. End stage renal disease (ESRD) on dialysis
3. United Network for Organ Sharing (UNOS) listed listed with current calculated panel reactive antibodies (cPRA) ≥99.9% or \>98% (with \>5 years of waiting time) awaiting deceased donor transplant
--Note: Those with cPRA \>98% with human leukocyte antigen (HLA)-incompatible approved living donor who have not received a transplant after 1 year in a paired kidney exchange program are also eligible
4. Evidence of established immunity to Epstein-Barr Virus (EBV) as demonstrated by serologic testing
5. Negative result of most recent tuberculosis (TB) testing or appropriately completed latent TB infection (LTBI) therapy.
* Testing should be conducted using either a PPD or interferon-gamma release assay (i.e., QuantiFERON-TB, T-SPOT.TB)
* Results from tests performed within 12 months prior to study entry are acceptable in the absence of any intervening exposure to TB
* Subjects with a positive test for LTBI must complete appropriate therapy for LTBI ---LTBI treatment regimens should be among those endorsed by the Centers for
Disease Control and Prevention (CDC), Division of TB Elimination, url:
https://www.cdc.gov/tb/topic/treatment/ltbi.htm
6. Negative Food and Drug Administration (FDA)-approved test for human immunodeficiency virus (HIV) diagnosis (at screening or as documented in medical record, up to 12 months prior to screening)
7. Negative Hepatitis C antibody test at screening or as documented in medical record, up to 12 months prior to screening
--If there is a history of treated hepatitis C then documentation of two consecutive negative HCV quantitative ribonucleic acid (RNA) Polymerase chain reaction (PCR) tests separated by at least 6 months is required. Untreated subjects with HCV RNA are eligible.
8. Negative result for SARS-CoV-2 by an FDA-authorized molecular diagnostic test. Examples include, but are not limited to RT-PCR, LAMP, TMA, and qSTAR.
9. Female subjects of reproductive potential must have a negative pregnancy test upon study entry
10. All subjects of reproductive potential must agree to use of contraception for the duration of the study
11. Subjects must have current vaccinations or documented immunity to varicella, measles, hepatitis B, pneumococcus, influenza, and zoster (if ≥50 years old)
* If subjects require administration of vaccines to meet eligibility requirements, they must wait at least 2 weeks between vaccination and the baseline (Visit 0) visit
Exclusion Criteria:
Individuals who meet any of these criteria are not eligible for enrollment as study subjects-
1. Inability or unwillingness of a subject to give written informed consent or comply with study protocol
2. Known active current or history of invasive fungal infection or non-tuberculous mycobacterial infection
3. Hepatitis B surface antigen or core antibody positive
4. Serious uncontrolled concomitant major organ disease excluding kidney failure
5. Previous non-kidney solid organ or bone marrow transplant
6. Any infection requiring hospitalization and intravenous (IV) antibiotics within 4 weeks of screening or by mouth (PO) antibiotics within 2 weeks
7. Primary or secondary immunodeficiency
8. History of active tuberculosis (TB), even if treated
9. History of positive result for 2019-novel Coronavirus (2019-nCoV) by real-time reverse transcriptase (RT-PCR)
10. Malignancy within the last 5 years except treated basal and squamous cell cancer of the skin or treated in situ cervical cancer
11. History of plasma cell dyscrasia
12. Alcohol, drug, or chemical abuse within 1 year
13. Difficult peripheral venous access
14. Need for uninterrupted anticoagulation
15. Neutropenia (absolute neutrophil count \<1000/uL) or thrombocytopenia (platelet count \<100,000/uL) within 4 weeks prior to study enrollment
16. Women who are currently pregnant or nursing
17. Treatment with any investigational agent within 4 weeks (or 5 half-lives of investigational drug, whichever is longer) of screening
18. Current treatment with other biological drug
19. Immunization with live vaccine within 2 weeks of study baseline (Visit 0) visit
20. Past or current medical problems or findings from physical examination or laboratory testing not listed above, which, in the opinion of the investigator, may:
* pose additional risks from participation in the study,
* interfere with the subject's ability to comply with study requirements, or
* impact the quality or interpretation of the data obtained from the study
**Maximum Age:** 65 Years
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** San Francisco
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Shreya Mall
- **Phone:** 415-353-8380
- **Role:** CONTACT
**Country:** United States
**Facility:** University of California at San Francisco Medical Center
**State:** California
**Status:** RECRUITING
**Zip:** 94143
#### Overall Officials
**Official 1:**
**Affiliation:** UCSF Kidney Transplant Research
**Name:** Flavio G. Vincenti
**Role:** STUDY_CHAIR
### IPD Sharing Statement Module
**Access Criteria:** Open access.
**Description:** The plan is to share data upon completion of the study in: Immunology Database and Analysis Portal (ImmPort), a long-term archive of clinical and mechanistic data from DAIT-funded grants and contracts.
**IPD Sharing:** YES
**Time Frame:** On average, within 24 months after database lock for the trial.
**URL:** https://www.immport.org/home
### References Module
#### See Also Links
**Label:** Immune Tolerance Network (ITN)
**URL:** https://www.immunetolerance.org/
## Derived Section
### Intervention Browse Module - Ancestors
- ID: D000007155
- Term: Immunologic Factors
- ID: D000045505
- Term: Physiological Effects of Drugs
- ID: D000000970
- Term: Antineoplastic Agents
- ID: D000082082
- Term: Immune Checkpoint Inhibitors
- ID: D000045504
- Term: Molecular Mechanisms of Pharmacological Action
- ID: D000074322
- Term: Antineoplastic Agents, Immunological
- ID: D000007166
- Term: Immunosuppressive Agents
- ID: D000018501
- Term: Antirheumatic Agents
### Intervention Browse Module - Browse Branches
- Abbrev: All
- Name: All Drugs and Chemicals
- Abbrev: ANeo
- Name: Antineoplastic Agents
- Abbrev: ARhu
- Name: Antirheumatic Agents
### Intervention Browse Module - Browse Leaves
- ID: M4225
- Name: Antibodies
- Relevance: LOW
- As Found: Unknown
- ID: M4230
- Name: Antibodies, Monoclonal
- Relevance: LOW
- As Found: Unknown
- ID: M272211
- Name: Daratumumab
- Relevance: HIGH
- As Found: National
- ID: M10184
- Name: Immunoglobulins
- Relevance: HIGH
- As Found: Colorectal Cancer
- ID: M10122
- Name: Immunoglobulin G
- Relevance: HIGH
- As Found: Optional
- ID: M19117
- Name: Immunoglobulins, Intravenous
- Relevance: LOW
- As Found: Unknown
- ID: M483
- Name: Abatacept
- Relevance: HIGH
- As Found: Potato
- ID: M10201
- Name: Immunologic Factors
- Relevance: LOW
- As Found: Unknown
- ID: M2342
- Name: Immune Checkpoint Inhibitors
- Relevance: LOW
- As Found: Unknown
- ID: M1346
- Name: Antineoplastic Agents, Immunological
- Relevance: LOW
- As Found: Unknown
- ID: M10212
- Name: Immunosuppressive Agents
- Relevance: LOW
- As Found: Unknown
- ID: M20604
- Name: Antirheumatic Agents
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: C000556306
- Term: Daratumumab
- ID: D000069594
- Term: Abatacept
- ID: D000007136
- Term: Immunoglobulins
- ID: D000007074
- Term: Immunoglobulin G
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT04751279
**Acronym:** JAK-SARC
**Brief Title:** Expression and Role of the JAK/STAT Pathway in Sarcoidosis Granuloma Cells
**Official Title:** Expression and Role of the JAK/STAT Pathway in Sarcoidosis Granuloma Cells
#### Organization Study ID Info
**ID:** APHP201205
#### Organization
**Class:** OTHER
**Full Name:** Assistance Publique - Hôpitaux de Paris
#### Secondary ID Infos
**Domain:** IDRCB
**ID:** 2020-A02330-39
**Type:** OTHER
### Status Module
#### Completion Date
**Date:** 2024-01-02
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2023-11-18
**Type:** ACTUAL
**Last Update Submit Date:** 2023-11-15
**Overall Status:** RECRUITING
#### Primary Completion Date
**Date:** 2024-01-01
**Type:** ESTIMATED
#### Start Date
**Date:** 2021-03-09
**Type:** ACTUAL
**Status Verified Date:** 2023-11
#### Study First Post Date
**Date:** 2021-02-12
**Type:** ACTUAL
**Study First Submit Date:** 2021-01-29
**Study First Submit QC Date:** 2021-02-10
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Assistance Publique - Hôpitaux de Paris
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** Recent studies suggest that the JAK/STAT signaling pathway constitutes a new step in the clinical and therapeutic progress of sarcoidosis. Further investigations are necessary to identify the most suitable patients to receive treatment targeting this pathway, in particular in cases of severe sarcoidosis refractory to the various therapeutic lines.
**Detailed Description:** The investigators hypothesize that the JAK/STAT signaling pathway constitutes a new step in the clinical and therapeutic progress of sarcoidosis, in particular in cases of severe sarcoidosis refractory to the various therapeutic lines. The main objective of this proposal is to evaluate the presence and stage of activation of the JAK/STAT pathway in PBMCs from inactive and active sarcoidosis patients. The second objective will be to determine the role of the JAK/STAT pathway in the formation and maintenance of granulomas and their association with the severity of sarcoidosis and fibrogenesis.
### Conditions Module
**Conditions:**
- Sarcoidosis
- JAK-STAT Pathway Deregulation
**Keywords:**
- Sarcoidosis
- JAK/STAT
### Design Module
#### Bio Spec
**Description:** Peripheral blood mononuclear cells
**Retention:** SAMPLES_WITHOUT_DNA
#### Design Info
**Observational Model:** CASE_ONLY
**Time Perspective:** PROSPECTIVE
#### Enrollment Info
**Count:** 80
**Type:** ESTIMATED
**Study Type:** OBSERVATIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Patients with pulmonary sarcoidosis without signs of chest activity (Benamore score \<2) and recent diagnosis (\<5 years)
**Intervention Names:**
- Other: Blood sample
**Label:** Patients with pulmonary sarcoidosis without signs of chest activity and recent diagnosis (<5 years)
#### Arm Group 2
**Description:** Patients with pulmonary sarcoidosis with signs of chest activity (Benamore score ≥2) and recent diagnosis (\<5 years)
**Intervention Names:**
- Other: Blood sample
**Label:** Patients with pulmonary sarcoidosis with signs of chest activity and recent diagnosis (<5 years
#### Arm Group 3
**Description:** Patients with pulmonary sarcoidosis without signs of activity (Benamore score \<2), persistent form (\>5 years)
**Intervention Names:**
- Other: Blood sample
**Label:** Patients with pulmonary sarcoidosis without signs of activity , persistent form (>5 years)
#### Arm Group 4
**Description:** Patients with pulmonary sarcoidosis with signs of chest activity (Benamore score ≥2), persistent form (\>5 years)
**Intervention Names:**
- Other: Blood sample
**Label:** Patients with pulmonary sarcoidosis with signs of chest activity , persistent form (>5 years)
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Patients with pulmonary sarcoidosis with signs of chest activity , persistent form (>5 years)
- Patients with pulmonary sarcoidosis with signs of chest activity and recent diagnosis (<5 years
- Patients with pulmonary sarcoidosis without signs of activity , persistent form (>5 years)
- Patients with pulmonary sarcoidosis without signs of chest activity and recent diagnosis (<5 years)
**Description:** Blood sample collection of 5 blood sample at the inclusion in the study the samle consite of 5 tubesEthylene Diamine Tetra Acetate (ADTA) of 7 ml
**Name:** Blood sample
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Description:** Evaluation will be carried out by:
- studying the expression of members of the JAK/STAT pathway, the expression of cytokines and chemokines phagocytosis and macrophage differentiation the expression of cytokines and chemokines
**Measure:** JAK/STAT familly numbers protein expression in PBMCs
**Time Frame:** 1 year after inclusion
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Patients with a diagnosis of mediastinopulmonary sarcoidosis made according to ATS / ERS / WASOG criteria
* Or patient suspected of having mediastino-pulmonary sarcoidosis, without any other probable causal factor identified on the usual standard examination at the time of the sample with the need for diagnostic confirmation at the end of the study according to the criteria of the ATS / ERS / WASOG
* Sarcoidosis with stage 1 to 4 pulmonary involvement
* Patients who had a chest CT scan in the 6 months preceding the sample. Examination carried out as part of routine care
Exclusion Criteria:
* Pregnancy.
* Opposition expressed to participation in the study.
* Patients on State Medical Aid.
**Maximum Age:** 80 Years
**Minimum Age:** 18 Years
**Sampling Method:** NON_PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
**Study Population:** Patients with mediastino-pulmonary sarcoidosis receiving or not receiving treatment for their sarcoidosis. Subjects treated for their sarcoidosis (corticosteroids and/or immunosuppressants) are not excluded because of two recent cases reported of multi-treated patients in which an involvement of the JAK/STAT pathway was demonstrated.
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Florence JENY, MD
**Phone:** 0148955129
**Role:** CONTACT
**Contact 2:**
**Email:** [email protected]
**Name:** Valerie BESNARD, PhD
**Role:** CONTACT
#### Locations
**Location 1:**
**City:** Bobigny
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Florence JENY, DR
- **Phone:** 01 48 95 74 35
- **Role:** CONTACT
***Contact 2:***
- **Email:** [email protected]
- **Name:** Zahia BEN ABDESSELAM, Dr
- **Phone:** 01 48 95 74 35
- **Role:** CONTACT
***Contact 3:***
- **Name:** Florence JENY, Dr
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** France
**Facility:** Service de pneumologie Hôpital Avicenne
**Status:** RECRUITING
**Zip:** 93000
#### Overall Officials
**Official 1:**
**Affiliation:** Assistance Publique - Hôpitaux Paris
**Name:** Florence JENY, MD
**Role:** PRINCIPAL_INVESTIGATOR
### References Module
#### References
**Citation:** Rotenberg C, Besnard V, Brillet PY, Giraudier S, Nunes H, Valeyre D. Dramatic response of refractory sarcoidosis under ruxolitinib in a patient with associated JAK2-mutated polycythemia. Eur Respir J. 2018 Dec 20;52(6):1801482. doi: 10.1183/13993003.01482-2018. Print 2018 Dec. No abstract available.
**PMID:** 30361243
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000008232
- Term: Lymphoproliferative Disorders
- ID: D000008206
- Term: Lymphatic Diseases
- ID: D000006968
- Term: Hypersensitivity, Delayed
- ID: D000006967
- Term: Hypersensitivity
- ID: D000007154
- Term: Immune System Diseases
- ID: D000010335
- Term: Pathologic Processes
### Condition Browse Module - Browse Branches
- Abbrev: BC15
- Name: Blood and Lymph Conditions
- Abbrev: BC20
- Name: Immune System Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
### Condition Browse Module - Browse Leaves
- ID: M15325
- Name: Sarcoidosis
- Relevance: HIGH
- As Found: Sarcoidosis
- ID: M9202
- Name: Granuloma
- Relevance: HIGH
- As Found: Granuloma
- ID: M11225
- Name: Lymphoproliferative Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M11203
- Name: Lymphatic Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M10018
- Name: Hypersensitivity
- Relevance: LOW
- As Found: Unknown
- ID: M10019
- Name: Hypersensitivity, Delayed
- Relevance: LOW
- As Found: Unknown
- ID: M10200
- Name: Immune System Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000012507
- Term: Sarcoidosis
- ID: D000006099
- Term: Granuloma
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT01055379
**Acronym:** ACCORDO
**Brief Title:** Rasagiline in Cognitive-impairment Related Depression: AzileCt in COgnitive-impairment Related DepressiOn
**Official Title:** A Randomised, Double-blind, Placebo-controlled Study to Evaluate if Rasagiline Can Improve Depressive Symptoms and Cognitive Function in Non-demented, Idiopathic Parkinson's Disease Patients: ACCORDO Study
#### Organization Study ID Info
**ID:** 12962A
#### Organization
**Class:** INDUSTRY
**Full Name:** Lundbeck Italia S.p.A.
#### Secondary ID Infos
**ID:** 2009-011144-19
**Type:** EUDRACT_NUMBER
### Status Module
#### Completion Date
**Date:** 2012-07
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2021-08-31
**Type:** ACTUAL
**Last Update Submit Date:** 2021-08-30
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2012-07
**Type:** ACTUAL
#### Start Date
**Date:** 2010-03
**Status Verified Date:** 2021-08
#### Study First Post Date
**Date:** 2010-01-25
**Type:** ESTIMATED
**Study First Submit Date:** 2010-01-22
**Study First Submit QC Date:** 2010-01-22
### Sponsor Collaborators Module
#### Collaborators
**Class:** INDUSTRY
**Name:** Teva Branded Pharmaceutical Products R&D, Inc.
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Lundbeck Italia S.p.A.
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** The primary endpoint for this study is the clinical response after 12 weeks of treatment, defined as a change in total score from baseline depressive symptoms as measured by the Beck Depression Inventory-Amended (BDI-IA) total score.
**Detailed Description:** ACCORDO is a multicentre, randomised, double-blind, and placebo-controlled study conducted in 12 Italian centres. Subjects are screened by means of the BDI-IA (cut-off 15) and randomised to treatment with rasagiline or placebo for 12 weeks.
Subjects have to be on stable treatment with dopaminergic agents at least 4 weeks before baseline, and maintained so during the course of the study.
The primary objective is to evaluate whether rasagiline compared to placebo improves depressive symptoms as evaluated by the BDI-IA total score.
### Conditions Module
**Conditions:**
- Depressive Symptoms
- Parkinson's Disease
**Keywords:**
- Rasagiline
- Depressive Symptoms
- Cognition
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** DOUBLE
**Who Masked:**
- PARTICIPANT
- INVESTIGATOR
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 121
**Type:** ACTUAL
**Phases:**
- PHASE4
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Intervention Names:**
- Drug: Rasagiline
**Label:** Rasagiline
**Type:** EXPERIMENTAL
#### Arm Group 2
**Intervention Names:**
- Drug: Placebo
**Label:** Placebo
**Type:** PLACEBO_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Rasagiline
**Description:** 1 mg/day for 12 weeks; orally
**Name:** Rasagiline
**Other Names:**
- Azilect
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- Placebo
**Description:** Once daily for 12 weeks; orally
**Name:** Placebo
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Measure:** Change from baseline in BDI-IA total score
**Time Frame:** 12 weeks
#### Secondary Outcomes
**Measure:** Evaluate if rasagiline compared to placebo improves cognitive function, over a treatment period of 12 weeks in idiopathic Parkinson´s Disease, using a formal neuropsychiatric cognitive test battery; quality of life (PDQ 39); apathy; ADL, motor function
**Time Frame:** 12 weeks
**Measure:** Change in quality of life using the PDQ-39 scale
**Time Frame:** 12 weeks
**Measure:** Change in apathy using the Apathy Scale
**Time Frame:** 12 weeks
**Measure:** Change in ADL and motor function using UPDRS scales part II and III, respectively
**Time Frame:** 12 weeks
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Outpatient, male or female aged \>=40 and \<80 years. The subject has a diagnosis of idiopathic Parkinson's Disease (PD) according to the United Kingdom Parkinson's Disease Society brain bank diagnostic criteria for PD for the clinical diagnosis of PD.
* Depressive symptoms with a minimum severity of \>=15 using the BDI-IA.
* Hoehn and Yahr stage I-III.
* Under stable (4 weeks prior to baseline) dopaminergic treatment without significant motor complication such as "on-off" phenomena and/or dyskinesia.
* The subject and/or legal representative and/or impartial witness is/are able to read and understand the Subject Information Sheet.
* The subject and/or legal representative has/have signed the Informed Consent Form (ICF) and if relevant the impartial witness has co-signed the ICF.
* If female, must: agree not to try to become pregnant during the study (female patients of childbearing potential will take pregnancy test, using a urine stick), AND use adequate contraception (adequate contraception is defined as oral/systemic contraception, intrauterine device, diaphragm in combination with spermicidal, or condom for male partner in combination with spermicidal), OR have been menopausal for at least 24 months prior to baseline, (OR) have been surgically sterilised prior to baseline, OR have had a hysterectomy prior to baseline.
Exclusion Criteria:
A subject, who meets one or more of the following criteria at the Baseline Visit, is not eligible for inclusion in this study:
* Motor complications such as wearing off and on-off phenomena.
* Mini-Mental State Examination (MMSE) \<26, corrected score.
* Diagnosis of current or history of major depressive episode according to DSM-IV-TR® criteria within 1 year before recruitment into the study.
* Presence of any other neurodegenerative disorder other than PD, based on judgement of investigator.
* Psychotic symptoms, e.g. hallucination and delirium (determined by clinical evaluation).
* Presence of any unstable or untreated systemic disorder such as diabetes, cardiac failure, or renal failure.
* Use of any prohibited concomitant medication according to the timelines provided in Appendix II.
* Patient who have undergone Deep Brain Stimulation surgery.
* Current treatment with antidepressants or history of treatment with antidepressants less than 1 month prior to randomisation.
* Current treatment or history of treatment less than 1 month prior to randomisation, with antipsychotics, cholinesterase inhibitors, memantine, amantadine, or anticholinergics.
* Current treatment with selegiline or history of treatment with selegiline less than 90 days prior to randomisation.
**Maximum Age:** 80 Years
**Minimum Age:** 40 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Cagliari
**Country:** Italy
**Facility:** IT010
**Zip:** 9134
**Location 2:**
**City:** Chieti
**Country:** Italy
**Facility:** IT007
**Zip:** 66013
**Location 3:**
**City:** Genova
**Country:** Italy
**Facility:** IT004
**Zip:** 16132
**Location 4:**
**City:** Lido di Camaiore
**Country:** Italy
**Facility:** IT005
**Zip:** 55043
**Location 5:**
**City:** Messina
**Country:** Italy
**Facility:** IT003
**Zip:** 98122
**Location 6:**
**City:** Milano
**Country:** Italy
**Facility:** IT012
**Zip:** 20135
**Location 7:**
**City:** Naples
**Country:** Italy
**Facility:** IT001
**Zip:** 80131
**Location 8:**
**City:** Roma
**Country:** Italy
**Facility:** IT011
**Zip:** 161
**Location 9:**
**City:** Rome
**Country:** Italy
**Facility:** IT008
**Zip:** 133
**Location 10:**
**City:** Torino
**Country:** Italy
**Facility:** IT015
**Zip:** 10126
**Location 11:**
**City:** Venezia
**Country:** Italy
**Facility:** IT013
**Zip:** 30126
**Location 12:**
**City:** Verona
**Country:** Italy
**Facility:** IT009
**Zip:** 37134
#### Overall Officials
**Official 1:**
**Affiliation:** [email protected]
**Name:** Email contact via H. Lundbeck A/S
**Role:** STUDY_DIRECTOR
### References Module
#### Available IPDs
**ID:** 2009-011144-19
**Type:** EMA EudraCT Results
**URL:** https://www.clinicaltrialsregister.eu/ctr-search/trial/2009-011144-19/results
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000020734
- Term: Parkinsonian Disorders
- ID: D000001480
- Term: Basal Ganglia Diseases
- ID: D000001927
- Term: Brain Diseases
- ID: D000002493
- Term: Central Nervous System Diseases
- ID: D000009422
- Term: Nervous System Diseases
- ID: D000009069
- Term: Movement Disorders
- ID: D000080874
- Term: Synucleinopathies
- ID: D000019636
- Term: Neurodegenerative Diseases
- ID: D000001526
- Term: Behavioral Symptoms
### Condition Browse Module - Browse Branches
- Abbrev: BXM
- Name: Behaviors and Mental Disorders
- Abbrev: All
- Name: All Conditions
- Abbrev: BC10
- Name: Nervous System Diseases
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: BC17
- Name: Skin and Connective Tissue Diseases
- Abbrev: BC18
- Name: Nutritional and Metabolic Diseases
### Condition Browse Module - Browse Leaves
- ID: M29705
- Name: Cognitive Dysfunction
- Relevance: LOW
- As Found: Unknown
- ID: M7058
- Name: Depression
- Relevance: HIGH
- As Found: Depressive Symptoms
- ID: M7061
- Name: Depressive Disorder
- Relevance: LOW
- As Found: Unknown
- ID: M13213
- Name: Parkinson Disease
- Relevance: HIGH
- As Found: Parkinson's Disease
- ID: M22494
- Name: Parkinsonian Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M25603
- Name: Ganglion Cysts
- Relevance: LOW
- As Found: Unknown
- ID: M16358
- Name: Synovial Cyst
- Relevance: LOW
- As Found: Unknown
- ID: M4774
- Name: Basal Ganglia Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5204
- Name: Brain Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5742
- Name: Central Nervous System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M12029
- Name: Movement Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M2217
- Name: Synucleinopathies
- Relevance: LOW
- As Found: Unknown
- ID: M21558
- Name: Neurodegenerative Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M4818
- Name: Behavioral Symptoms
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000010300
- Term: Parkinson Disease
- ID: D000003863
- Term: Depression
### Intervention Browse Module - Ancestors
- ID: D000008996
- Term: Monoamine Oxidase Inhibitors
- ID: D000004791
- Term: Enzyme Inhibitors
- ID: D000045504
- Term: Molecular Mechanisms of Pharmacological Action
- ID: D000018696
- Term: Neuroprotective Agents
- ID: D000020011
- Term: Protective Agents
- ID: D000045505
- Term: Physiological Effects of Drugs
### Intervention Browse Module - Browse Branches
- Abbrev: NeuroAg
- Name: Neuroprotective Agents
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M213846
- Name: Rasagiline
- Relevance: HIGH
- As Found: Sudden
- ID: M11960
- Name: Monoamine Oxidase Inhibitors
- Relevance: LOW
- As Found: Unknown
- ID: M7951
- Name: Enzyme Inhibitors
- Relevance: LOW
- As Found: Unknown
- ID: M20773
- Name: Neuroprotective Agents
- Relevance: LOW
- As Found: Unknown
- ID: M21869
- Name: Protective Agents
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: C000031967
- Term: Rasagiline
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT06029179
**Brief Title:** SZC Versus SPS for Treatment of Hyperkalemia in Hemodialysis Patients
**Official Title:** Sodium Zirconium Cyclosilicate Versus Sodium Polystyrene Sulfonate for Treatment of Hyperkalemia in Hemodialysis Patients: A Randomized Clinical Trial
#### Organization Study ID Info
**ID:** Hyperkalemia treatment in HD
#### Organization
**Class:** OTHER
**Full Name:** Alexandria University
### Status Module
#### Completion Date
**Date:** 2024-06-15
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-03-18
**Type:** ACTUAL
**Last Update Submit Date:** 2024-03-15
**Overall Status:** RECRUITING
#### Primary Completion Date
**Date:** 2024-05-15
**Type:** ESTIMATED
#### Start Date
**Date:** 2024-01-15
**Type:** ACTUAL
**Status Verified Date:** 2024-03
#### Study First Post Date
**Date:** 2023-09-08
**Type:** ACTUAL
**Study First Submit Date:** 2023-09-01
**Study First Submit QC Date:** 2023-09-01
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Alexandria University
#### Responsible Party
**Investigator Affiliation:** Alexandria University
**Investigator Full Name:** Mohamed Mamdouh Mahmoud Mohamed Elsayed , MD
**Investigator Title:** Lecturer
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
### Description Module
**Brief Summary:** This study aims to compare the effects of Sodium Zirconium Cyclosilicate versus Sodium Polystyrene Sulfonate for treatment of hyperkalemia in patients undergoing regular hemodialysis.
**Detailed Description:** Patients maintained on regular hemodialysis (HD) have a high risk of hyperkalemia (\>5.0 mmol/l). Hyperkalemia is a critical medical condition that can result in arrhythmias and sudden cardiac death. Treatment of hyperkalemia in HD patients is challenging.
Therapeutic options for the treatment of hyperkalemia in HD population include potassium binding resins, such as sodium polystyrene sulfonate (SPS), patiromer, and sodium zirconium cyclosilicate. There is limited data about the use of these agents in HD.
### Conditions Module
**Conditions:**
- Hyperkalemia
**Keywords:**
- Hyperkalemia
- Hemodialysis
- Sodium Zirconium Cyclosilicate
- Sodium Polystyrene Sulfonate
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
**Intervention Model Description:** This research is a prospective randomized multicentric clinical trial in which 60 hemodialysis patients will be randomly assigned to one of the study groups using block randomization with a ratio of 1:1.
* Group A: 30 patients will receive sodium zirconium cyclosilicate (SZC) 5 g once daily on non-dialysis days (15 gm/week) for 4 weeks.
* Group B: 30 patients will receive sodium polystyrene sulfonate 15 g once daily on non-dialysis days (45 gm/week) for 4 weeks.
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 60
**Type:** ESTIMATED
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** 30 patients will receive sodium zirconium cyclosilicate (SZC)
**Intervention Names:**
- Drug: sodium zirconium cyclosilicate (SZC)
**Label:** Group A
**Type:** ACTIVE_COMPARATOR
#### Arm Group 2
**Description:** 30 patients will receive sodium polystyrene sulfonate
**Intervention Names:**
- Drug: sodium polystyrene sulfonate
**Label:** Group B
**Type:** ACTIVE_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Group A
**Description:** 30 patients will receive sodium zirconium cyclosilicate (SZC) 5 g once daily on non-dialysis days (15 gm/week) for 4 weeks.
**Name:** sodium zirconium cyclosilicate (SZC)
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- Group B
**Description:** 30 patients will receive sodium polystyrene sulfonate 15 g once daily on non-dialysis days (45 gm/week) for 4 weeks.
**Name:** sodium polystyrene sulfonate
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Description:** By assessing serum K at baseline, 24 hrs after first dose, then weekly after the long interdialytic interval
**Measure:** Change in serum potassium
**Time Frame:** 4 weeks
#### Secondary Outcomes
**Description:** By assessing change in interdialytic weight
**Measure:** Change in interdialytic weight
**Time Frame:** 4 weeks
**Description:** By reporting any GIT SE
**Measure:** Gastrointestinal side effects
**Time Frame:** 4 weeks
**Description:** systolic and diastolic Blood pressure change
**Measure:** Change in Blood pressure
**Time Frame:** 4 weeks
**Description:** By reporting any serious adverse events.
**Measure:** Serious adverse events
**Time Frame:** 4 weeks
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
1. ≥3 month of maintenance hemodialysis, 3 times per week for 4 hours.
2. Adult patients with age above 18 years.
3. baseline serum potassium level \>5 mEq/L.
Exclusion Criteria:
1. Gastrointestinal diseases (constipation, bleeding, Hx of endoscopy, chronic diarrhea or diarrhea in the past month, malabsorption, GIT surgery, ischemic colitis, necrosis, perforation, ....).
2. Breast feeding or pregnancy.
3. Patients who receive medications to treat hyperkalemia 2 weeks before study.
4. myocardial infarction, acute coronary syndrome, stroke, seizure, or thromboembolic event within 8 weeks before study.
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Mohamed Mamdouh Elsayed, MD
**Phone:** 00201068055103
**Role:** CONTACT
#### Locations
**Location 1:**
**City:** Alexandria
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Mohamed Mamdouh Elsayed, MD
- **Phone:** 00201068055103
- **Role:** CONTACT
**Country:** Egypt
**Facility:** Faculty of Medicine, Aexandria University
**Status:** RECRUITING
**Zip:** 21526
#### Overall Officials
**Official 1:**
**Affiliation:** lecturer
**Name:** Mohamed Mamdouh Elsayed, MD
**Role:** PRINCIPAL_INVESTIGATOR
**Official 2:**
**Affiliation:** consultant
**Name:** Marwa Ahmed Abdelrahman, MD
**Role:** STUDY_CHAIR
**Official 3:**
**Affiliation:** Lecturer
**Name:** Mohamed Aly Abdelhalim, MD
**Role:** STUDY_CHAIR
### IPD Sharing Statement Module
**IPD Sharing:** UNDECIDED
### References Module
#### References
**Citation:** Yusuf AA, Hu Y, Singh B, Menoyo JA, Wetmore JB. Serum Potassium Levels and Mortality in Hemodialysis Patients: A Retrospective Cohort Study. Am J Nephrol. 2016;44(3):179-86. doi: 10.1159/000448341. Epub 2016 Sep 3.
**PMID:** 27592170
**Citation:** Hoppe LK, Muhlack DC, Koenig W, Carr PR, Brenner H, Schottker B. Association of Abnormal Serum Potassium Levels with Arrhythmias and Cardiovascular Mortality: a Systematic Review and Meta-Analysis of Observational Studies. Cardiovasc Drugs Ther. 2018 Apr;32(2):197-212. doi: 10.1007/s10557-018-6783-0.
**PMID:** 29679302
**Citation:** Beccari MV, Meaney CJ. Clinical utility of patiromer, sodium zirconium cyclosilicate, and sodium polystyrene sulfonate for the treatment of hyperkalemia: an evidence-based review. Core Evid. 2017 Mar 23;12:11-24. doi: 10.2147/CE.S129555. eCollection 2017. Erratum In: Core Evid. 2019 Feb 27;14:1.
**PMID:** 28356904
**Citation:** Fried L, Kovesdy CP, Palmer BF. New options for the management of chronic hyperkalemia. Kidney Int Suppl (2011). 2017 Dec;7(3):164-170. doi: 10.1016/j.kisu.2017.09.001. Epub 2017 Nov 17.
**PMID:** 30675431
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000014883
- Term: Water-Electrolyte Imbalance
- ID: D000008659
- Term: Metabolic Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC18
- Name: Nutritional and Metabolic Diseases
- Abbrev: All
- Name: All Conditions
### Condition Browse Module - Browse Leaves
- ID: M9998
- Name: Hyperkalemia
- Relevance: HIGH
- As Found: Hyperkalemia
- ID: M17624
- Name: Water-Electrolyte Imbalance
- Relevance: LOW
- As Found: Unknown
- ID: M11639
- Name: Metabolic Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000006947
- Term: Hyperkalemia
### Intervention Browse Module - Ancestors
- ID: D000002614
- Term: Chelating Agents
- ID: D000064449
- Term: Sequestering Agents
- ID: D000045504
- Term: Molecular Mechanisms of Pharmacological Action
### Intervention Browse Module - Browse Branches
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M240546
- Name: Polystyrene sulfonic acid
- Relevance: HIGH
- As Found: Visual stimulation
- ID: M5860
- Name: Chelating Agents
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: C000003321
- Term: Polystyrene sulfonic acid
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT01778179
**Brief Title:** A Fixed Triple Combination Cream for Solar Lentigines Associated to Cryotherapy
**Official Title:** Safety and Efficacy of a Triple Combination Cream as Adjuvant Treatment of Solar Lentigines With Cryotherapy.
#### Organization Study ID Info
**ID:** 2012-03
#### Organization
**Class:** OTHER
**Full Name:** Brazilan Center for Studies in Dermatology
### Status Module
#### Completion Date
**Date:** 2012-12
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2020-10-14
**Type:** ACTUAL
**Last Update Submit Date:** 2020-09-23
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2012-12
**Type:** ACTUAL
#### Results First Post Date
**Date:** 2020-09-17
**Type:** ACTUAL
**Results First Submit Date:** 2020-08-11
**Results First Submit QC Date:** 2020-08-27
#### Start Date
**Date:** 2011-04
**Status Verified Date:** 2020-09
#### Study First Post Date
**Date:** 2013-01-29
**Type:** ESTIMATED
**Study First Submit Date:** 2013-01-04
**Study First Submit QC Date:** 2013-01-25
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Brazilan Center for Studies in Dermatology
#### Responsible Party
**Investigator Affiliation:** Brazilan Center for Studies in Dermatology
**Investigator Full Name:** Doris Hexsel
**Investigator Title:** MD
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** To evaluate safety and efficacy of Tri-Luma® cream as an adjunctive treatment to cryotherapy when used in the pre- and post-procedure phases for the treatment of solar lentigines on the back of the hands and in the prevention of post-inflammatory hyperpigmentation after cryotherapy.
**Detailed Description:** To evaluate safety and efficacy of Tri-Luma® cream as an adjunctive treatment to cryotherapy when used in the pre- and post-procedure phases for the treatment of solar lentigines on the back of the hands and in the prevention of post-inflammatory hyperpigmentation after cryotherapy.
The study has 13 weeks for each subject. Five visits will take place: at Baseline, week 2, 5, 9 and 13 after the cryotherapy.
### Conditions Module
**Conditions:**
- Solar Lentigines
**Keywords:**
- Lentigines
- Cryotherapy
- hyperpigmentation
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** NONE
**Primary Purpose:** PREVENTION
#### Enrollment Info
**Count:** 50
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Subjects (group 1) will be treated daily for their solar lentigines with the investigational drug (Tri-Luma® cream) plus sunscreen for 2 weeks. At week 2, all the subjects will have the solar lentigines treated by cryotherapy (CRY-AC3® device).
Post-procedure phase (From Week 2 up to Week 13)
* Topical antibiotic treatment phase (Week 2 up to Week 5 - visit 2 to visit 3): At week 2, all the subjects will start to apply a topical antibiotic (Neomycin/Nebacetin® ointment) for the next 3 weeks.
* Treatment phase 2 (from week 5 up to week 13 - visit 3 up to visit 5):
The investigational drug (Tri-Luma® cream) plus sunscreen will be applied again (group 1) for a minimum of 4 weeks and up to 8 weeks, according to the Global Improvement of solar lentigines and absence of PIH.
**Intervention Names:**
- Drug: Tri-luma
- Procedure: Cryotherapy (CRY-AC3® device)
**Label:** Group 1
**Type:** ACTIVE_COMPARATOR
#### Arm Group 2
**Description:** Subjects (group 2) will be treated daily for their solar lentigines with sunscreen alone for 2 weeks. At week 2, all the subjects will have the solar lentigines will treated by cryotherapy (CRY-AC3® device).
Post-procedure phase (Week 2 up to Week 13)
* Topical antibiotic treatment phase (Week 2 up to Week 5 - visit 2 to visit 3): At week 2, all the subjects will start to apply a topical antibiotic (Neomycin/Nebacetin® ointment) for the next 3 weeks.
* Treatment phase 2 (from week 5 up to week 13 - visit 3 up to visit 5):
Sunscreen alone will be applied again (group 1) for a minimum of 4 weeks and up to 8 weeks, according to the Global Improvement of solar lentigines and absence of PIH.
**Intervention Names:**
- Procedure: Cryotherapy (CRY-AC3® device)
**Label:** Group 2
**Type:** PLACEBO_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Group 1
**Description:** Pre-procedure phase (From Baseline up to Week 2) treated daily for their solar lentigines with the investigational drug (Tri-Luma® cream) for 2 weeks.
Post-procedure phase (From week 2 up to Week 13 - Visit )
- Tri-Luma® cream treatment phase (from week 5 up to week 13 - visit 3 up to visit 5):The investigational drug (Tri-Luma® cream) will be applied again (group 1) for a minimum of 4 weeks and up to 8 weeks, according to the Global Improvement of solar lentigines and absence of PIH.
**Name:** Tri-luma
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- Group 1
- Group 2
**Description:** Procedure performed at week 2.
**Name:** Cryotherapy (CRY-AC3® device)
**Type:** PROCEDURE
### Outcomes Module
#### Primary Outcomes
**Description:** Solar lentigines count up to 13 weeks
**Measure:** Solar Lentigines Count
**Time Frame:** up to 13 weeks
#### Secondary Outcomes
**Description:** This assessment was made using a narrowband reflectance spectrophotometer (Mexameter MX18®, Courage-Khazaka, Germany). The measurement is based on absorption/reflection. The probe of the Mexameter® MX 18 emits three specific light wavelengths. A receiver measures the light reflected by the skin. As the quantity of emitted light is defined, the quantity of light absorbed by the skin can be calculated. The melanin is measured by two specific wavelengths (red: 660 nm and near infrared: 880 nm) chosen to correspond to different absorption rates by the pigments.
The quantification of melanin is presented as a value that varies from 0-999. The higher the value, the larger is the amount of melanin on the skin.
**Measure:** Change From Baseline in Melanin Levels
**Time Frame:** Baseline and up to 13 weeks
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Subject agreement to provide written informed consent and the willingness and ability to comply with all aspects of the protocol;
* Female and male subjects;
* Phototype II to IV;
* Subjects aged between 40 and 65 years;
* Subjects presenting at least 5 lesions of solar lentigines at the back hands with al least 3mm of diameter
* History of post-inflammatory hyperpigmentation on body or face
* Medical history and physical examination which, based on the investigator's opinion, do not prevent the subject from taking part in the study or from making use of the products under investigation;
* Subjects of childbearing age should present a negative urine pregnancy test at baseline and should be using a highly effective contraceptive method during all study;
* Availability of the subject throughout the study;
* Subject agreeing not to undergo other cosmetic or dermatological procedures during the participation in the study;
* Subjects with sufficient schooling and knowledge to enable them to cooperate to the degree required by the protocol.
Exclusion Criteria:
* Pregnant women or women intending to become pregnant in the following 5 months after screening;
* Lactation period;
* Subjects participating in other clinical trials;
* Any prior cosmetic procedures, including fillers, or scars that may interfere with the study results;
* Subjects with neoplastic, muscular or neurological diseases;
* Subjects with inflammation or active infection in the area to be studied;
* Subjects with a history of adverse effects, such as sensitivity to the components of any of the study drug formula,
* Subjects with a history of non-adherence to medical treatment or showing unwillingness to adhere to the study protocol;
* Any condition that, in the opinion of the investigator, can compromise the evaluation of the study.
**Healthy Volunteers:** True
**Maximum Age:** 65 Years
**Minimum Age:** 40 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Overall Officials
**Official 1:**
**Affiliation:** Brazilian Center for Studies in Dermatology
**Name:** Doris Hexsel, MD
**Role:** PRINCIPAL_INVESTIGATOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000008548
- Term: Melanosis
- ID: D000017495
- Term: Hyperpigmentation
- ID: D000010859
- Term: Pigmentation Disorders
- ID: D000012871
- Term: Skin Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC17
- Name: Skin and Connective Tissue Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
### Condition Browse Module - Browse Leaves
- ID: M10923
- Name: Lentigo
- Relevance: HIGH
- As Found: Lentigo
- ID: M19760
- Name: Hyperpigmentation
- Relevance: LOW
- As Found: Unknown
- ID: M11531
- Name: Melanosis
- Relevance: LOW
- As Found: Unknown
- ID: M13754
- Name: Pigmentation Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M15674
- Name: Skin Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000007911
- Term: Lentigo
### Intervention Browse Module - Browse Branches
- Abbrev: Infe
- Name: Anti-Infective Agents
- Abbrev: All
- Name: All Drugs and Chemicals
- Abbrev: Derm
- Name: Dermatologic Agents
### Intervention Browse Module - Browse Leaves
- ID: M4222
- Name: Anti-Bacterial Agents
- Relevance: LOW
- As Found: Unknown
- ID: M4224
- Name: Antibiotics, Antitubercular
- Relevance: LOW
- As Found: Unknown
- ID: M16255
- Name: Sunscreening Agents
- Relevance: LOW
- As Found: Unknown
- ID: M12300
- Name: Neomycin
- Relevance: LOW
- As Found: Unknown
### Misc Info Module
- Version Holder: 2024-05-24
## Results Section
### Adverse Events Module
#### Event Groups
**Group ID:** EG000
**Title:** Group 1
**Deaths Num At Risk:** 25
**Description:** Subjects treated daily for their solar lentigines with the triple combination (hydroquinone 4% tretinoin 0.05% fluocinolone acetonide 0.01%; Tri-Luma® cream) plus sunscreen for 2 weeks. At week 2, all the subjects had the solar lentigines treated by cryotherapy (CRY-AC3® device).
Post-procedure phase (Week 2 up to Week 13)
* Topical antibiotic treatment (Week 2 to Week 5): All the subjects applied a topical antibiotic (Neomycin/Nebacetin® ointment) for the following 3 weeks.
* Second treatment phase (Week 5 to Week 13):
Subjects of group 1 applied Tri-Luma® cream plus sunscreen again for a minimum of 4 weeks and up to 8 weeks, depending on the Global Improvement of solar lentigines score and absence of PIH.
**ID:** EG000
**Other Num Affected:** 3
**Other Num at Risk:** 25
**Serious Number At Risk:** 25
**Title:** Group 1
**Group ID:** EG001
**Title:** Group 2
**Deaths Num At Risk:** 25
**Description:** Subjects treated daily for their solar lentigines with sunscreen only for 2 weeks. At week 2, all the subjects had the solar lentigines treated by cryotherapy (CRY-AC3® device).
Post-procedure phase (Week 2 up to Week 13)
* Topical antibiotic treatment phase (Week 2 to Week 5): All the subjects applied a topical antibiotic (Neomycin/Nebacetin® ointment) for the next 3 weeks.
* Second treatment phase (Week 5 to Week 13):
Subjects of group 2 applied sunscreen only again for a minimum of 4 weeks and up to 8 weeks, depending on the Global Improvement of solar lentigines score and absence of PIH.
**ID:** EG001
**Other Num Affected:** 2
**Other Num at Risk:** 25
**Serious Number At Risk:** 25
**Title:** Group 2
**Frequency Threshold:** 0
#### Other Events
**Term:** Erythema
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:**
**Term:** Residual blisters from cryotherapy
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:**
**Time Frame:** Up to 13 weeks
## Results Section - Baseline Characteristics Module
### Denomination Counts
**Group ID:** BG000
**Value:** 25
**Group ID:** BG001
**Value:** 25
**Group ID:** BG002
**Value:** 50
**Units:** Participants
### Group
**ID:** BG000
**Title:** Group 1
**Description:** Subjects treated daily for their solar lentigines with the triple combination (hydroquinone 4% tretinoin 0.05% fluocinolone acetonide 0.01%; Tri-Luma® cream) plus sunscreen for 2 weeks. At week 2, all the subjects had the solar lentigines treated by cryotherapy (CRY-AC3® device).
Post-procedure phase (Week 2 up to Week 13)
* Topical antibiotic treatment (Week 2 to Week 5): All the subjects applied a topical antibiotic (Neomycin/Nebacetin® ointment) for the following 3 weeks.
* Second treatment phase (Week 5 to Week 13):
Subjects of group 1 applied Tri-Luma® cream plus sunscreen again for a minimum of 4 weeks and up to 8 weeks, depending on the Global Improvement of solar lentigines score and absence of PIH.
### Group
**ID:** BG001
**Title:** Group 2
**Description:** Subjects treated daily for their solar lentigines with sunscreen only for 2 weeks. At week 2, all the subjects had the solar lentigines treated by cryotherapy (CRY-AC3® device).
Post-procedure phase (Week 2 up to Week 13)
* Topical antibiotic treatment phase (Week 2 to Week 5): All the subjects applied a topical antibiotic (Neomycin/Nebacetin® ointment) for the next 3 weeks.
* Second treatment phase (Week 5 to Week 13):
Subjects of group 2 applied sunscreen only again for a minimum of 4 weeks and up to 8 weeks, depending on the Global Improvement of solar lentigines score and absence of PIH.
### Group
**ID:** BG002
**Title:** Total
**Description:** Total of all reporting groups
### Measure
#### Measurement
**Group ID:** BG000
**Spread:** 4
**Value:** 59
#### Measurement
**Group ID:** BG001
**Spread:** 6
**Value:** 56
#### Measurement
**Group ID:** BG002
**Spread:** 5
**Value:** 58
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 23
#### Measurement
**Group ID:** BG001
**Value:** 24
#### Measurement
**Group ID:** BG002
**Value:** 47
**Category Title:** Female
#### Measurement
**Group ID:** BG000
**Value:** 2
#### Measurement
**Group ID:** BG001
**Value:** 1
#### Measurement
**Group ID:** BG002
**Value:** 3
**Category Title:** Male
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 0
**Category Title:** American Indian or Alaska Native
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 0
**Category Title:** Asian
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 0
**Category Title:** Native Hawaiian or Other Pacific Islander
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 0
**Category Title:** Black or African American
#### Measurement
**Group ID:** BG000
**Value:** 25
#### Measurement
**Group ID:** BG001
**Value:** 24
#### Measurement
**Group ID:** BG002
**Value:** 49
**Category Title:** White
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 1
#### Measurement
**Group ID:** BG002
**Value:** 1
**Category Title:** More than one race
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 0
**Category Title:** Unknown or Not Reported
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 25
#### Measurement
**Group ID:** BG001
**Value:** 25
#### Measurement
**Group ID:** BG002
**Value:** 50
**Class Title:** Brazil
**Denomination Units Selected:**
## Results Section - Baseline Characteristics Module
### Measure 1
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Age, Continuous
**Unit of Measure:** years
### Measure 2
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Sex: Female, Male
**Unit of Measure:** Participants
### Measure 3
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Race (NIH/OMB)
**Unit of Measure:** Participants
### Measure 4
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Region of Enrollment
**Unit of Measure:** Participants
## Results Section - More Information Module
### Certain Agreement
### Point of Contact
**Email:** [email protected]
**Organization:** Brazilian Center for Studies in Dermatology
**Phone:** +55 51 30262633
**Title:** Dr. Doris Hexsel
## Results Section - Outcome Measures Module
### Outcome Measure 1
### Outcome Measure 2
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 7.0
- **Upper Limit:**
- **Value:** 25.7
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 11.0
- **Upper Limit:**
- **Value:** 26.0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 6.0
- **Upper Limit:**
- **Value:** 8.0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 6.0
- **Upper Limit:**
- **Value:** 11.0
**Title:**
#### Outcome Measure 2
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 69
- **Upper Limit:**
- **Value:** 297
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 68
- **Upper Limit:**
- **Value:** 279
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 59
- **Upper Limit:**
- **Value:** -95
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 65
- **Upper Limit:**
- **Value:** -41
**Title:**
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Description:** Solar lentigines count up to 13 weeks
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Reporting Status:** POSTED
**Time Frame:** up to 13 weeks
**Title:** Solar Lentigines Count
**Type:** PRIMARY
**Unit of Measure:** Number of solar lentigines
##### Group
**Description:** Subjects treated daily for their solar lentigines with the triple combination (hydroquinone 4% tretinoin 0.05% fluocinolone acetonide 0.01%; Tri-Luma® cream) plus sunscreen for 2 weeks. At week 2, all the subjects had the solar lentigines treated by cryotherapy (CRY-AC3® device).
Post-procedure phase (Week 2 up to Week 13)
* Topical antibiotic treatment (Week 2 to Week 5): All the subjects applied a topical antibiotic (Neomycin/Nebacetin® ointment) for the following 3 weeks.
* Second treatment phase (Week 5 to Week 13):
Subjects of group 1 applied Tri-Luma® cream plus sunscreen again for a minimum of 4 weeks and up to 8 weeks, depending on the Global Improvement of solar lentigines score and absence of PIH.
**ID:** OG000
**Title:** Group 1
##### Group
**Description:** Subjects treated daily for their solar lentigines with sunscreen only for 2 weeks. At week 2, all the subjects had the solar lentigines treated by cryotherapy (CRY-AC3® device).
Post-procedure phase (Week 2 up to Week 13)
* Topical antibiotic treatment phase (Week 2 to Week 5): All the subjects applied a topical antibiotic (Neomycin/Nebacetin® ointment) for the next 3 weeks.
* Second treatment phase (Week 5 to Week 13):
Subjects of group 2 applied sunscreen only again for a minimum of 4 weeks and up to 8 weeks, depending on the Global Improvement of solar lentigines score and absence of PIH.
**ID:** OG001
**Title:** Group 2
#### Outcome Measure 2
**Description:** This assessment was made using a narrowband reflectance spectrophotometer (Mexameter MX18®, Courage-Khazaka, Germany). The measurement is based on absorption/reflection. The probe of the Mexameter® MX 18 emits three specific light wavelengths. A receiver measures the light reflected by the skin. As the quantity of emitted light is defined, the quantity of light absorbed by the skin can be calculated. The melanin is measured by two specific wavelengths (red: 660 nm and near infrared: 880 nm) chosen to correspond to different absorption rates by the pigments.
The quantification of melanin is presented as a value that varies from 0-999. The higher the value, the larger is the amount of melanin on the skin.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Reporting Status:** POSTED
**Time Frame:** Baseline and up to 13 weeks
**Title:** Change From Baseline in Melanin Levels
**Type:** SECONDARY
**Unit of Measure:** Arbitrary units
##### Group
**Description:** Subjects treated daily for their solar lentigines with the triple combination (hydroquinone 4% tretinoin 0.05% fluocinolone acetonide 0.01%; Tri-Luma® cream) plus sunscreen for 2 weeks. At week 2, all the subjects had the solar lentigines treated by cryotherapy (CRY-AC3® device).
Post-procedure phase (Week 2 up to Week 13)
* Topical antibiotic treatment (Week 2 to Week 5): All the subjects applied a topical antibiotic (Neomycin/Nebacetin® ointment) for the following 3 weeks.
* Second treatment phase (Week 5 to Week 13):
Subjects of group 1 applied Tri-Luma® cream plus sunscreen again for a minimum of 4 weeks and up to 8 weeks, depending on the Global Improvement of solar lentigines score and absence of PIH.
**ID:** OG000
**Title:** Group 1
##### Group
**Description:** Subjects treated daily for their solar lentigines with sunscreen only for 2 weeks. At week 2, all the subjects had the solar lentigines treated by cryotherapy (CRY-AC3® device).
Post-procedure phase (Week 2 up to Week 13)
* Topical antibiotic treatment phase (Week 2 to Week 5): All the subjects applied a topical antibiotic (Neomycin/Nebacetin® ointment) for the next 3 weeks.
* Second treatment phase (Week 5 to Week 13):
Subjects of group 2 applied sunscreen only again for a minimum of 4 weeks and up to 8 weeks, depending on the Global Improvement of solar lentigines score and absence of PIH.
**ID:** OG001
**Title:** Group 2
### Participant Flow Module
#### Group
**Description:** Subjects treated daily for their solar lentigines with the triple combination (hydroquinone 4% tretinoin 0.05% fluocinolone acetonide 0.01%; Tri-Luma® cream) plus sunscreen for 2 weeks. At week 2, all the subjects had the solar lentigines treated by cryotherapy (CRY-AC3® device).
Post-procedure phase (Week 2 up to Week 13)
* Topical antibiotic treatment (Week 2 to Week 5): All the subjects applied a topical antibiotic (Neomycin/Nebacetin® ointment) for the following 3 weeks.
* Second treatment phase (Week 5 to Week 13):
Subjects of group 1 applied Tri-Luma® cream plus sunscreen again for a minimum of 4 weeks and up to 8 weeks, depending on the Global Improvement of solar lentigines score and absence of post inflammatory hyperpigmentation.
**ID:** FG000
**Title:** Group 1
#### Group
**Description:** Subjects treated daily for their solar lentigines with sunscreen only for 2 weeks. At week 2, all the subjects had the solar lentigines treated by cryotherapy (CRY-AC3® device).
Post-procedure phase (Week 2 up to Week 13)
* Topical antibiotic treatment phase (Week 2 to Week 5): All the subjects applied a topical antibiotic (Neomycin/Nebacetin® ointment) for the next 3 weeks.
* Second treatment phase (Week 5 to Week 13):
Subjects of group 2 applied sunscreen only again for a minimum of 4 weeks and up to 8 weeks, depending on the Global Improvement of solar lentigines score and absence of post inflammatory hyperpigmentation.
**ID:** FG001
**Title:** Group 2
#### Period
**Title:** Overall Study
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 25
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 25
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 23
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 22
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 2
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 3
**Has Results:** True |
## Protocol Section
### Identification Module
**NCT ID:** NCT03456479
**Brief Title:** Diagnosis of Cow's Milk Protein Allergy Among Infants and Children in Assuit University Children Hospital
**Official Title:** Diagnosis of Cow's Milk Protein Allergy Among Infants and Children in Assuit University
#### Organization Study ID Info
**ID:** cow's milk protein allergy
#### Organization
**Class:** OTHER
**Full Name:** Assiut University
### Status Module
#### Completion Date
**Date:** 2020-09-01
**Type:** ESTIMATED
#### Expanded Access Info
**Last Known Status:** NOT_YET_RECRUITING
#### Last Update Post Date
**Date:** 2018-03-07
**Type:** ACTUAL
**Last Update Submit Date:** 2018-03-03
**Overall Status:** UNKNOWN
#### Primary Completion Date
**Date:** 2020-03-01
**Type:** ESTIMATED
#### Start Date
**Date:** 2019-03-01
**Type:** ESTIMATED
**Status Verified Date:** 2018-03
#### Study First Post Date
**Date:** 2018-03-07
**Type:** ACTUAL
**Study First Submit Date:** 2018-03-03
**Study First Submit QC Date:** 2018-03-03
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Assiut University
#### Responsible Party
**Investigator Affiliation:** Assiut University
**Investigator Full Name:** Gamal Nady Rady Ibrahim
**Investigator Title:** Assiut - Egypt
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
### Description Module
**Brief Summary:** The study will assess the diagnosis of Cow's milk protein allergy (CMPA) among infant and children in Assiut University Children Hospital using skin prick test and specific serum IgE
**Detailed Description:** Food allergy is a reproducible specific immune response to the exposure to a given food. Cow's milk protein allergy (CMPA) is a clinically abnormal reaction to cow's milk protein (CMP) via immune mechanisms triggered by milk protein (Boyce etal., 2010) .
According to the European Academy for Allergy and Clinical Immunology (EAACI) and the World Allergy Organization (WAO), a hypersensitivity reaction to cow's milk involving the immune system can be called as CMPA.There is difference between CMPA and non-allergic cow's milk intolerance (e.glactose intolerance) which occurs as a result of lactase deficiency without involvement of the immune system (Venter etal ., 2013) .
CMPA is categorized into immunoglobulin IgE mediated, non-IgE mediated, or mixed CMPA(Sichereretal ., 2001) .
1.9% to 4.9% prevalence of CMPA among children with a peak in prevalence (2-3%) in the first year of life and prevalence of \<1% in children aged ≥6 years(Koletzkoetal ., 2012) .
### Conditions Module
**Conditions:**
- Cow Milk Allergy
### Design Module
#### Design Info
**Allocation:** NA
**Intervention Model:** SINGLE_GROUP
##### Masking Info
**Masking:** NONE
**Primary Purpose:** DIAGNOSTIC
#### Enrollment Info
**Count:** 30
**Type:** ESTIMATED
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
### Interventions
#### Intervention 1
**Description:** The study will assess the diagnosis of Cow's milk protein allergy (CMPA) among infant and children in Assiut University Children Hospital using skin prick test and specificserumIgE
**Name:** skin prick test - specific serum IgE
**Type:** DIAGNOSTIC_TEST
### Outcomes Module
#### Primary Outcomes
**Description:** The study will assess the diagnosis of Cow's milk protein allergy (CMPA) among infant and children in Assiut University Children Hospital using skin prick test and specificserumIgE
**Measure:** Diagnosis of cow's milk protein allergy
**Time Frame:** baseline
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
1. age : from first day of life - 2 years
2. exclusively breast feeding,breast-fed infants with supplementary foods and non-breast-fed infants with supplementary foods who complain from either frequentregurgitation,vomiting,diarrhea,constipation)with/without perianal rash),bloodin stool or pallor.
Exclusion Criteria:
* Infant and child with gastrointestinal symptoms due to specific aetiology .
**Maximum Age:** 2 Years
**Minimum Age:** 1 Day
**Sex:** ALL
**Standard Ages:**
- CHILD
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Gamal N Rady, Bachelor
**Phone:** 01220108053
**Role:** CONTACT
**Contact 2:**
**Name:** Shereen M Galal, Doctorate
**Phone:** 01003162310
**Role:** CONTACT
### References Module
#### References
**Citation:** Celik-Bilgili S, Mehl A, Verstege A, Staden U, Nocon M, Beyer K, Niggemann B. The predictive value of specific immunoglobulin E levels in serum for the outcome of oral food challenges. Clin Exp Allergy. 2005 Mar;35(3):268-73. doi: 10.1111/j.1365-2222.2005.02150.x.
**PMID:** 15784102
**Citation:** Brill H. Approach to milk protein allergy in infants. Can Fam Physician. 2008 Sep;54(9):1258-64.
**PMID:** 18791102
**Citation:** Vandenplas Y, De Greef E, Devreker T. Treatment of Cow's Milk Protein Allergy. Pediatr Gastroenterol Hepatol Nutr. 2014 Mar;17(1):1-5. doi: 10.5223/pghn.2014.17.1.1. Epub 2014 Mar 31.
**PMID:** 24749081
**Citation:** Sicherer SH, Noone SA, Koerner CB, Christie L, Burks AW, Sampson HA. Hypoallergenicity and efficacy of an amino acid-based formula in children with cow's milk and multiple food hypersensitivities. J Pediatr. 2001 May;138(5):688-93. doi: 10.1067/mpd.2001.113007.
**PMID:** 11343044
**Citation:** Koletzko S, Niggemann B, Arato A, Dias JA, Heuschkel R, Husby S, Mearin ML, Papadopoulou A, Ruemmele FM, Staiano A, Schappi MG, Vandenplas Y; European Society of Pediatric Gastroenterology, Hepatology, and Nutrition. Diagnostic approach and management of cow's-milk protein allergy in infants and children: ESPGHAN GI Committee practical guidelines. J Pediatr Gastroenterol Nutr. 2012 Aug;55(2):221-9. doi: 10.1097/MPG.0b013e31825c9482.
**PMID:** 22569527
**Citation:** Venter C, Brown T, Shah N, Walsh J, Fox AT. Diagnosis and management of non-IgE-mediated cow's milk allergy in infancy - a UK primary care practical guide. Clin Transl Allergy. 2013 Jul 8;3(1):23. doi: 10.1186/2045-7022-3-23.
**PMID:** 23835522
**Citation:** NIAID-Sponsored Expert Panel; Boyce JA, Assa'ad A, Burks AW, Jones SM, Sampson HA, Wood RA, Plaut M, Cooper SF, Fenton MJ, Arshad SH, Bahna SL, Beck LA, Byrd-Bredbenner C, Camargo CA Jr, Eichenfield L, Furuta GT, Hanifin JM, Jones C, Kraft M, Levy BD, Lieberman P, Luccioli S, McCall KM, Schneider LC, Simon RA, Simons FE, Teach SJ, Yawn BP, Schwaninger JM. Guidelines for the diagnosis and management of food allergy in the United States: report of the NIAID-sponsored expert panel. J Allergy Clin Immunol. 2010 Dec;126(6 Suppl):S1-58. doi: 10.1016/j.jaci.2010.10.007.
**PMID:** 21134576
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000007154
- Term: Immune System Diseases
- ID: D000005512
- Term: Food Hypersensitivity
- ID: D000006969
- Term: Hypersensitivity, Immediate
### Condition Browse Module - Browse Branches
- Abbrev: BC20
- Name: Immune System Diseases
- Abbrev: All
- Name: All Conditions
### Condition Browse Module - Browse Leaves
- ID: M10018
- Name: Hypersensitivity
- Relevance: HIGH
- As Found: Allergy
- ID: M18719
- Name: Milk Hypersensitivity
- Relevance: HIGH
- As Found: Cow's Milk Protein Allergy
- ID: M10200
- Name: Immune System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M8636
- Name: Food Hypersensitivity
- Relevance: LOW
- As Found: Unknown
- ID: M10020
- Name: Hypersensitivity, Immediate
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000006967
- Term: Hypersensitivity
- ID: D000016269
- Term: Milk Hypersensitivity
### Intervention Browse Module - Browse Branches
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M5616
- Name: Caseins
- Relevance: LOW
- As Found: Unknown
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT04015479
**Brief Title:** Peanut Protein Supplementation to Augment Muscle Growth and Improve Markers of Muscle Quality and Health in Older Adults
**Official Title:** Open-label Randomized Controlled Trial Investigating the Effects of Peanut Protein Powder Supplement on Muscle Growth, Muscle Quality and Other Health Biomarkers in Older Adults Engaging in a Ten-week, Whole-body Resistance Training Program
#### Organization Study ID Info
**ID:** 19-249
#### Organization
**Class:** OTHER
**Full Name:** Auburn University
### Status Module
#### Completion Date
**Date:** 2020-05-01
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2021-01-12
**Type:** ACTUAL
**Last Update Submit Date:** 2021-01-10
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2020-05-01
**Type:** ACTUAL
#### Start Date
**Date:** 2019-09-26
**Type:** ACTUAL
**Status Verified Date:** 2021-01
#### Study First Post Date
**Date:** 2019-07-11
**Type:** ACTUAL
**Study First Submit Date:** 2019-07-09
**Study First Submit QC Date:** 2019-07-09
### Sponsor Collaborators Module
#### Collaborators
**Class:** OTHER
**Name:** Edward Via Virginia College of Osteopathic Medicine
**Class:** OTHER
**Name:** The Peanut Institute
#### Lead Sponsor
**Class:** OTHER
**Name:** Auburn University
#### Responsible Party
**Investigator Affiliation:** Auburn University
**Investigator Full Name:** Andrew Fruge
**Investigator Title:** Assistant Professor
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** This study will evaluate the adaptations in skeletal muscle that occur in response to 10 weeks of weight training with or without peanut protein supplementation in older adult men and women.
**Detailed Description:** Aging is associated with declines in muscle mass, physical strength and physical function. Adequate quality protein intake in aging adults is critical to preventing functional decline. Peanuts provide a unique blend of amino acids that can provide several health benefits to aging adults. While supplementing with peanut protein (PP) powder as part of a resistance training program may increase myofibrillar protein synthesis (i.e., the gold standard molecular assessment in deciphering a muscle-building response), and improve skeletal muscle quality and body composition, no study to date has made this determination.
This is a two-phase study using both novel and conventional methods to assess how PP supplementation affects muscle tissue in older individuals who engage in resistance training. These two phases will be conducted as part of a 10-week randomized controlled trial in which men and women aged 50 years and older (n=60), will be stratified by gender and randomized to a resistance training intervention (whole body, two days per week) with PP powder (72g daily; n=15 males, n=15 females) provided during the intervention (immediate group, IG) or after the intervention (wait-list control, WLC, n=15 males, n=15 females). The aims of this study are to determine the acute (deuterium oxide tracer) and chronic (peripheral quantitative computed tomography) effects of PP during resistance training on skeletal muscle myofibrillar protein synthesis rates, changes in skeletal muscle size and quality, changes in whole and appendicular body composition (dual energy x-ray absorptiometry), changes in inflammatory markers and the fecal microbiome.
### Conditions Module
**Conditions:**
- Aging
- Sarcopenia
**Keywords:**
- muscle protein synthesis
- resistance training
- protein supplementation
- skeletal muscle physiology
- inflammatory cytokines
- fecal microbiome
- plant protein
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
**Intervention Model Description:** Intervention group will receive the supplement during the study period and the wait-listed control group will receive the supplement after the study period. Both groups will participate in resistance training during the 10 week period
##### Masking Info
**Masking:** DOUBLE
**Masking Description:** PI and Co-Is will be blind to participant randomization. One study staff member will be responsible for administering the supplements to participants per randomization
**Who Masked:**
- INVESTIGATOR
- OUTCOMES_ASSESSOR
**Primary Purpose:** PREVENTION
#### Enrollment Info
**Count:** 41
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Participants will complete 10 weeks of twice-weekly whole body resistance training. Peanut protein powder (72g/day) will be provided for daily consumption during the study period
**Intervention Names:**
- Dietary Supplement: Peanut Protein Powder
- Behavioral: Full body resistance training
**Label:** Immediate Intervention Group
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Participants will complete 10 weeks of twice-weekly whole body resistance training. Peanut protein powder will be provided after the study period
**Intervention Names:**
- Behavioral: Full body resistance training
**Label:** Wait-list Control Group
**Type:** ACTIVE_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Immediate Intervention Group
**Description:** Peanut protein powder will be provided to participants who will be instructed to consume 72g daily, mixed with water
**Name:** Peanut Protein Powder
**Type:** DIETARY_SUPPLEMENT
#### Intervention 2
**Arm Group Labels:**
- Immediate Intervention Group
- Wait-list Control Group
**Description:** Participants will undergo supervised resistance training two times per week (5 exercises, 3 sets of 8-12 repetitions per set)
**Name:** Full body resistance training
**Type:** BEHAVIORAL
### Outcomes Module
#### Primary Outcomes
**Description:** Change in right leg vastus lateralis myofibrillar protein synthesis rates using the integrated deuterium oxide technique from biopsies immediately before and 24 hours after resistance exercise
**Measure:** Change in acute myofibrillar protein synthesis rates
**Time Frame:** 24 hours
**Description:** peripheral quantitative computed tomography (pQCT) cross-sectional image of mid-right thigh assessed for total muscle cross-sectional area, subcutaneous adipose tissue area, total intra- and inter-muscular adipose tissue area and overall muscle density
**Measure:** Change in mid-thigh skeletal muscle area and quality
**Time Frame:** 0-10 weeks
#### Secondary Outcomes
**Description:** dual energy x-ray absorptiometry (DXA)
**Measure:** Change in whole-body and appendicular body composition
**Time Frame:** 0-10 weeks
**Description:** Muscle biopsy immunofluorescent staining for determination of type I and type II muscle fiber cross sectional area (fCSA) as a cellular determinant of skeletal muscle hypertrophy
**Measure:** Change in Type I and II Muscle Fiber Cross-Sectional Area
**Time Frame:** 0-10 weeks
**Description:** maximal isokinetic right leg extensions on an isokinetic dynamometer (BioDex)
**Measure:** Change in leg extensor isokinetic dynamometry
**Time Frame:** 0-10 weeks
**Description:** serum C-reactive protein, interleukin-6, tumor necrosis factor-alpha, plasma 8-hydroxy-2'deoxyguanosine
**Measure:** Change in inflammatory biomarkers
**Time Frame:** 0-10 weeks
**Description:** alpha- and beta-diversity of 16S bacterial rDNA
**Measure:** Change in fecal microbiome composition
**Time Frame:** 0-10 weeks
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* body mass index (body mass/height squared) less than 35 kg/m2
* resting blood pressure averaging less than 140/90 mmHg (with or without medication)
Exclusion Criteria:
* known peanut allergy
* actively participating in resistance training for more than 2 days/week
* any known overt cardiovascular or metabolic disease
* metal implants that will interfere with x-ray procedures
* medically necessary radiation exposure in the last six months (except dental x-ray)
* any medical condition that would contradict participating in an exercise program, giving blood or donating a skeletal muscle biopsy (i.e. blood clotting disorder or taking blood thinners)
**Healthy Volunteers:** True
**Maximum Age:** 80 Years
**Minimum Age:** 50 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Auburn
**Country:** United States
**Facility:** Auburn University
**State:** Alabama
**Zip:** 36849
#### Overall Officials
**Official 1:**
**Affiliation:** Auburn University
**Name:** Michael Roberts, PhD
**Role:** PRINCIPAL_INVESTIGATOR
### References Module
#### References
**Citation:** Lamb DA, Moore JH, Smith MA, Vann CG, Osburn SC, Ruple BA, Fox CD, Smith KS, Altonji OM, Power ZM, Cerovsky AE, Ross CO, Cao AT, Goodlett MD, Huggins KW, Fruge AD, Young KC, Roberts MD. The effects of resistance training with or without peanut protein supplementation on skeletal muscle and strength adaptations in older individuals. J Int Soc Sports Nutr. 2020 Dec 14;17(1):66. doi: 10.1186/s12970-020-00397-y.
**PMID:** 33317565
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000009133
- Term: Muscular Atrophy
- ID: D000020879
- Term: Neuromuscular Manifestations
- ID: D000009461
- Term: Neurologic Manifestations
- ID: D000009422
- Term: Nervous System Diseases
- ID: D000001284
- Term: Atrophy
- ID: D000020763
- Term: Pathological Conditions, Anatomical
### Condition Browse Module - Browse Branches
- Abbrev: BC10
- Name: Nervous System Diseases
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: All
- Name: All Conditions
### Condition Browse Module - Browse Leaves
- ID: M28396
- Name: Sarcopenia
- Relevance: HIGH
- As Found: Sarcopenia
- ID: M12090
- Name: Muscular Atrophy
- Relevance: LOW
- As Found: Unknown
- ID: M4589
- Name: Atrophy
- Relevance: LOW
- As Found: Unknown
- ID: M22619
- Name: Neuromuscular Manifestations
- Relevance: LOW
- As Found: Unknown
- ID: M12404
- Name: Neurologic Manifestations
- Relevance: LOW
- As Found: Unknown
- ID: M22519
- Name: Pathological Conditions, Anatomical
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000055948
- Term: Sarcopenia
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT01670279
**Brief Title:** Phase 1 Study to Assess the Safety/Tolerability of Brexpiprazole as Adjunctive Therapy in Elderly Subjects With Major Depressive Disorder
**Official Title:** A Phase 1, Multicenter, Randomized, Double-blind, Sequential Cohort, Placebo-controlled Trial to Assess the Safety and Tolerability of Ascending Multiple Oral Doses of Brexpiprazole as Adjunctive Therapy in the Treatment of Elderly Subjects With Major Depressive Disorder
#### Organization Study ID Info
**ID:** 331-12-291
#### Organization
**Class:** INDUSTRY
**Full Name:** Otsuka Pharmaceutical Development & Commercialization, Inc.
### Status Module
#### Completion Date
**Date:** 2013-05
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2016-02-04
**Type:** ESTIMATED
**Last Update Submit Date:** 2016-01-05
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2013-05
**Type:** ACTUAL
#### Results First Post Date
**Date:** 2016-02-04
**Type:** ESTIMATED
**Results First Submit Date:** 2015-08-04
**Results First Submit QC Date:** 2016-01-05
#### Start Date
**Date:** 2012-07
**Status Verified Date:** 2016-01
#### Study First Post Date
**Date:** 2012-08-22
**Type:** ESTIMATED
**Study First Submit Date:** 2012-08-07
**Study First Submit QC Date:** 2012-08-21
### Sponsor Collaborators Module
#### Collaborators
**Class:** INDUSTRY
**Name:** H. Lundbeck A/S
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Otsuka Pharmaceutical Development & Commercialization, Inc.
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** The purpose of this study is to assess the safety and tolerability of ascending multiple oral doses of brexpiprazole as adjunctive therapy in the treatment of elderly subjects with MDD.
**Detailed Description:** This is a phase 1, multicenter, randomized, double-blind, placebo-controlled, multiple ascending dose trial in 3 sequential cohorts of elderly subjects (age 70 to 85 years old) with MDD. Brexpiprazole will be administered as an adjunct treatment to the current antidepressant therapy that the subject is receiving. Total individual subject duration is expected to be no more than 119 days (a 30-day screening period, a 14-day washout period, up to 45-day in-clinic treatment period, and a 30-day follow-up after the last dose of trial medication).
### Conditions Module
**Conditions:**
- Major Depressive Disorder
**Keywords:**
- Major Depressive Disorder (MDD)
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** DOUBLE
**Who Masked:**
- PARTICIPANT
- INVESTIGATOR
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 18
**Type:** ACTUAL
**Phases:**
- PHASE1
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** 14 day titration phase and two fixed dose phases. The first fixed dose phase is 14 days with a daily dose of 2mg brexpiprazole/placebo. The second fixed dose phase is 14 days with a daily dose of 3 mg brexpiprazole/placebo.
**Intervention Names:**
- Drug: Brexpiprazole
**Label:** Cohort 1
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** 14 day titration phase and a 14 day fixed dose phase a daily dose of 3mg brexpiprazole/placebo.
**Intervention Names:**
- Drug: Brexpiprazole
**Label:** Cohort 2
**Type:** EXPERIMENTAL
#### Arm Group 3
**Description:** 21 day titration phase and a 14 day fixed dose phase a daily dose of 3mg brexpiprazole/placebo.
**Intervention Names:**
- Drug: Brexpiprazole
**Label:** Cohort 3
**Type:** EXPERIMENTAL
#### Arm Group 4
**Description:** Placebo
**Intervention Names:**
- Drug: Placebo
**Label:** Placebo
**Type:** PLACEBO_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Cohort 1
- Cohort 2
- Cohort 3
**Description:** up to 3mg oral dose once daily
**Name:** Brexpiprazole
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- Placebo
**Name:** Placebo
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Description:** Safety and tolerability of brexpiprazole was noted to be primary outcome measure. Brexpiprazole was judged to be tolerated if at least 6 out of 8 (75%) of the participants in a test cohort tolerated the dose after 14 days of QD dosing at the end of the fixed dose phase based on the blinded data. Dose toleration was defined as follows: during the course of the trial, the participants did not experience any moderate or severe adverse events (AEs) or potentially clinically relevant changes from Baseline in laboratory values, vital signs, electrocardiogram (ECG) tracings, Columbia-Suicide Severity Rating Scale (C-SSRS), or extrapyramidal symptom (EPS) ratings, which were assessed as possibly related to the study drug, and would have warranted a dose decrease or discontinuation of the study drug. The safety and tolerability of brexpiprazole was defined by parameters: AEs, laboratory values, vital signs, ECG, C-SSRS, or EPS ratings, the results of each of the parameters reported separately.
**Measure:** Number of Participants Who Tolerated Brexpiprazole
**Time Frame:** 45 Days
**Description:** The AEs were one of the primary parameters to measure the safety and tolerability of individual participants. The AEs were captured for all participants from the time the ICF was signed until the end of the trial. AEs were measured throughout the 14-day titration and 28-day fixed dose phase until follow-up (30 \[±2\] days after last dose of study medication).
**Measure:** Number of AEs Reported.
**Time Frame:** Throughout the study, up to 119 days
**Description:** The laboratory values were one of the primary parameters to measure the safety and tolerability of individual participants. Incidence of TEAEs of potential clinical relevance include abnormal values in serum chemistry, hematology, urinalyses and prolactin tests that were identified based on pre-defined criteria.
**Measure:** Incidence of Laboratory Values of Potential Clinical Significance
**Time Frame:** Titration Day 7, Fixed dose Day 14 and 28 and Last Visit
**Description:** The vital signs were one of the primary parameters to measure the safety and tolerability of individual participants. Incidence of TEAEs of potential clinical relevance included abnormal values in heart rate, systolic and diastolic blood pressure, respiratory rate and weight that were identified based on pre-defined criteria.
**Measure:** Incidence of Vital Signs of Potential Clinical Significance
**Time Frame:** Baseline, Titration Day 1, 2, 7, 8, Fixed Days 1, 2, 14, 15, 28, 29, Early Termination and Last Visit.
**Description:** The measurement of ECG was one of the primary parameters to measure the safety and tolerability of individual participants. Incidence of TEAEs of potential clinical relevance include abnormal changes in heart rate and ECG intervals of PR, QRS, QT, QTcB, and QTcF that were identified based on pre-defined criteria.
**Measure:** Incidence of ECG Evaluations of Potential Clinical Significance
**Time Frame:** Titratrion Day 1 and 7, Fixed dose Day 1, 14, 28, Early Termination
**Description:** The physical examination evaluation was one of the primary parameters to measure the safety and tolerability of individual participants. Incidence of TEAEs of potential clinical relevance include abnormal changes in the following body systems: head, ears, eyes, nose, and throat; thorax; abdomen; urogenital; extremities; neurological; and skin and mucosae.
**Measure:** Incidence of Physical Examination Evaluation of Potential Clinical Significance
**Time Frame:** Physical examination was performed at Screening, check-in, and discharge
**Description:** EPS was one of the primary parameters to measure the safety and tolerability of individual participants. The SAS is a rating scale used to measure EPS. The SAS scale consists of a list of 10 symptoms of parkinsonism (gait, arm dropping, shoulder shaking, elbow rigidity, wrist rigidity, head rotation, glabella tap, tremor, salivation, and akathisia), with each item rated from 0 to 4, with 0 being normal and 4 being the worst. The SAS Total score is sum of ratings for all 10 items, with possible Total scores from 0 to 40.
**Measure:** Mean Change From Baseline to Study Completion in Simpson-Angus Scale (SAS) Total Score
**Time Frame:** End of Titration, Day 15, Day 29, Early Termination and Last visit
**Description:** EPS was one of the primary parameters to measure the safety and tolerability of individual participants. The Barnes Akathisia Rating Scale was an EPS rating scale. The Barnes Akathisia Rating Scale was used to assess the presence and severity of akathisia. This scale consists of 4 items. Only the 4th item, the Global Clinical Assessment of Akathisia, was evaluated in this trial. This item is rated on a 6 point scale, with 0 being best (absent) and 5 being worst (severe akathisia).
**Measure:** Mean Change From Baseline to Study Completion in Barnes Akathisia Global Score
**Time Frame:** End of Titration, Day 15, Day 29, Early Termination and Last visit
**Description:** EPS was one of the primary parameters to measure the safety and tolerability of individual participants. The AIMS Scale was an EPS rating scale. The AIMS is a 12 item scale. The first 10 items are rated from 0 to 4 (0=best, 4=worst). Items 11 and 12, related to dental status, have dichotomous responses, 0=no and 1=yes. The AIMS Total Score is the sum of the ratings for the first seven items. The possible total scores are from 0 to 28.
**Measure:** Mean Change From Baseline to Study Completion in Abnormal Involuntary Movement Scale (AIMS) Rating Score.
**Time Frame:** End of Titration, Day 15, Day 29, Early Termination and Last visit
**Description:** The C-SSRS was one of the primary parameters to measure the safety and tolerability of individual participants. Suicidality was monitored during the trial using the C-SSRS. This scale consists of a baseline evaluation that assesses the lifetime experience of the participant with suicide events and suicidal ideation and a post-baseline evaluation that focuses on suicidality since the last trial visit.
**Measure:** Change From Baseline to Study Completion in C-SSRS Score.
**Time Frame:** Baseline, End of Titration, Fixed dose Day 14 and 28, Day 15 and 29, Early Termination, Last Visit
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Subjects who are able to provide written informed consent
* Ability to understand the nature of the trial and follow protocol requirements
* Male and female patients 70 to 85 years of age
* Subjects with normal or clinically stable findings on physical examination, medical history, clinical laboratory determinations, ECGs in relation to age
* BMI of 18 to 35 kg/m2.
* Stable subjects with a principal psychiatric diagnosis of MDD
* Subjects willing to discontinue all prohibited psychotropic and other prohibited medication
Exclusion Criteria:
* Sexually active males who are not practicing 2 different methods of birth control during the trial and for 30 days after the last dose of trial medication or who will not remain abstinent during the trial and for 30 days after the last dose
* Subjects who have had a vagus nerve stimulation device implanted or who have received ECT within 6 months of Screening
* Subjects with a current Axis I (DSM-IV-TR) diagnosis of:
* Delirium, dementia, amnestic, or other cognitive disorder
* Eating disorder (including anorexia nervosa or bulimia)
* Obsessive-compulsive disorder
* Panic disorder
* Posttraumatic stress disorder or current or prior Axis I (DSM-IV-TR) diagnosis of Schizophrenia, schizoaffective disorder, or other psychotic disorder, Bipolar I or II disorder or bipolar disorder not otherwise specified
* Subjects with a clinically significant current Axis II (DSM-IV-TR) diagnosis of borderline, antisocial, paranoid, schizoid, schizotypal, or histrionic personality disorder
* Subjects experiencing hallucinations, delusions, or any psychotic symptomatology
* Subjects who have Active Suicidal Ideation with Some Intent to Act and whose most recent episode occurred within the last 6 months
* Subjects who have met DSM-IV-TR criteria for substance abuse or dependence within the past 180 days
* Subjects with hypothyroidism or hyperthyroidism and/or an abnormal result for free T4 at Screening
* Subjects who currently have clinically significant neurological, hepatic, renal, metabolic, hematological, immunological, cardiovascular, pulmonary, or gastrointestinal disorders
* Subjects with IDDM
* Subjects with uncontrolled hypertension (DBP \> 95 mmHg) or symptomatic hypotension
* Subjects with epilepsy, a history of epilepsy, or a history of seizure
* Subjects with a positive drug screen for cocaine or other drugs of abuse
* The following laboratory test and ECG results are exclusionary:
1. Platelets ≤ 75,000/mm3
2. Hemoglobin ≤ 9 g/dL
3. Neutrophils, absolute ≤ 1000/mm3
4. AST \> 3 × upper limit of normal
5. ALT \> 3 × upper limit of normal
6. Creatinine ≥ 2 mg/dL
7. HbA1c ≥ 7%
8. QTcF ≥ 450 msec
* Treatment with a MAOI within the 2 weeks prior to the first dose of trial medication
* Use of benzodiazepines and/or hypnotics within 1 week prior the first dose of trial medication
* Use of oral neuroleptics within 30 days prior to or long-acting approved neuroleptics ≤ 1 full cycle plus 14 days prior to the first dose of trial medication on Day 1
* Prohibited concomitant medications used prior to randomization or anticipated need for such medications during the trial
* Subjects who would be likely to require prohibited concomitant therapy during the trial
* Subjects who received brexpiprazole in any prior clinical trial
* Subjects with a history of neuroleptic malignant syndrome
* Subjects with a history of true allergic response to more than 1 class of medications
* Prisoners or subjects who are compulsorily detained for treatment of either a psychiatric or physical illness
* Subjects who participated in a clinical trial within the last 180 days or who participated in more than 2 clinical trials within the past year.
**Maximum Age:** 85 Years
**Minimum Age:** 70 Years
**Sex:** ALL
**Standard Ages:**
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Kissimmee
**Country:** United States
**Facility:** Accurate Clinical Trials
**State:** Florida
**Location 2:**
**City:** Miami
**Country:** United States
**Facility:** Miami Jewish Health System
**State:** Florida
**Location 3:**
**City:** St. Louis
**Country:** United States
**Facility:** St. Louis Clinical Trials
**State:** Missouri
**Location 4:**
**City:** Philadelphia
**Country:** United States
**Facility:** CRI Lifetree- Philadelphia Research Center
**State:** Pennsylvania
#### Overall Officials
**Official 1:**
**Affiliation:** Otsuka Pharmaceutical Development & Commercialization, Inc.
**Name:** James M. Youakim, MD
**Role:** STUDY_DIRECTOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000019964
- Term: Mood Disorders
- ID: D000001523
- Term: Mental Disorders
- ID: D000001526
- Term: Behavioral Symptoms
### Condition Browse Module - Browse Branches
- Abbrev: BXM
- Name: Behaviors and Mental Disorders
- Abbrev: All
- Name: All Conditions
### Condition Browse Module - Browse Leaves
- ID: M7058
- Name: Depression
- Relevance: HIGH
- As Found: Depressive Disorder
- ID: M7061
- Name: Depressive Disorder
- Relevance: HIGH
- As Found: Depressive Disorder
- ID: M7060
- Name: Depressive Disorder, Major
- Relevance: HIGH
- As Found: Major Depressive Disorder
- ID: M21835
- Name: Mood Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M4815
- Name: Mental Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M14473
- Name: Psychotic Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M4818
- Name: Behavioral Symptoms
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000003866
- Term: Depressive Disorder
- ID: D000003863
- Term: Depression
- ID: D000003865
- Term: Depressive Disorder, Major
### Intervention Browse Module - Ancestors
- ID: D000018490
- Term: Serotonin Agents
- ID: D000018377
- Term: Neurotransmitter Agents
- ID: D000045504
- Term: Molecular Mechanisms of Pharmacological Action
- ID: D000045505
- Term: Physiological Effects of Drugs
- ID: D000018491
- Term: Dopamine Agonists
- ID: D000015259
- Term: Dopamine Agents
### Intervention Browse Module - Browse Branches
- Abbrev: All
- Name: All Drugs and Chemicals
- Abbrev: CaAg
- Name: Cardiotonic Agents
### Intervention Browse Module - Browse Leaves
- ID: M293045
- Name: Brexpiprazole
- Relevance: HIGH
- As Found: Urban
- ID: M15512
- Name: Serotonin
- Relevance: LOW
- As Found: Unknown
- ID: M20504
- Name: Neurotransmitter Agents
- Relevance: LOW
- As Found: Unknown
- ID: M7473
- Name: Dopamine
- Relevance: LOW
- As Found: Unknown
- ID: M20595
- Name: Dopamine Agonists
- Relevance: LOW
- As Found: Unknown
- ID: M17962
- Name: Dopamine Agents
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: C000591922
- Term: Brexpiprazole
### Misc Info Module
- Version Holder: 2024-05-24
## Results Section
### Adverse Events Module
**Description:** Participants who had received at least one dose of study medication were included in safety analysis.
#### Event Groups
**Group ID:** EG000
**Title:** Brexpiprazole-Cohort 1
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 2 mg brexpiprazole QD for 14 days, followed by 3 mg brexpiprazole QD for 14 days.
**ID:** EG000
**Other Num Affected:** 6
**Other Num at Risk:** 6
**Serious Number At Risk:** 6
**Title:** Brexpiprazole-Cohort 1
**Group ID:** EG001
**Title:** Brexpiprazole-Cohort 2
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 3 mg brexpiprazole QD for 14 days.
**ID:** EG001
**Other Num Affected:** 6
**Other Num at Risk:** 6
**Serious Number At Risk:** 6
**Title:** Brexpiprazole-Cohort 2
**Group ID:** EG002
**Title:** Placebo
**Description:** In the titration phase, participants received 0.5 mg placebo QD for 7 days, followed by 1 mg placebo QD for 7 days. In the fixed dose phase, Cohort 1: participants received 2 mg placebo QD for 14 days, followed by 3 mg placebo QD for 14 days. Cohort 2: participants received 3 mg placebo QD for 14 days.
**ID:** EG002
**Other Num Affected:** 1
**Other Num at Risk:** 5
**Serious Number At Risk:** 5
**Title:** Placebo
**Frequency Threshold:** 0
#### Other Events
**Term:** Atrioventricular block first degree
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Cardiac disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Hyperprolactinaemia
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Endocrine disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Diarrhoea
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Dyspepsia
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Nausea
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Cardiac murmur
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** MedDRA 15.0
**Term:** Electrocardiogram QT prolonged
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** MedDRA 15.0
**Term:** Weight increased
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** MedDRA 15.0
**Term:** Dizziness
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Headache
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Tremor
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Insomnia
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Psychiatric disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Pollakiuria
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Renal and urinary disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Oropharyngeal pain
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Arthritis
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Back pain
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Pain in extremity
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Upper respiratory tract infection
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 15.0
**Term:** Oedema peripheral
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Constipation
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Dental caries
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Gastrooesophageal reflux disease
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Vomiting
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Eye pruritis
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Dry eye
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA 15.0
**Term:** Blood pressure
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** MedDRA 15.0
**Time Frame:** AEs were captured for all participants from the signing of the informed consent and were measured throughout the 14-day titration period, 28-day fixed dose period until follow-up (30 [±2] days after last dose of study medication).
## Results Section - Baseline Characteristics Module
### Denomination Counts
**Group ID:** BG000
**Value:** 6
**Group ID:** BG001
**Value:** 7
**Group ID:** BG002
**Value:** 5
**Group ID:** BG003
**Value:** 18
**Units:** Participants
### Group
**ID:** BG000
**Title:** Brexpiprazole-Cohort 1
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 2 mg brexpiprazole QD for 14 days, followed by 3 mg brexpiprazole QD for 14 days.
### Group
**ID:** BG001
**Title:** Brexpiprazole-Cohort 2
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 3 mg brexpiprazole QD for 14 days.
### Group
**ID:** BG002
**Title:** Placebo
**Description:** In the titration phase, participants received 0.5 mg placebo QD for 7 days, followed by 1 mg placebo QD for 7 days. In the fixed dose phase, Cohort 1: participants received 2 mg placebo QD for 14 days, followed by 3 mg placebo QD for 14 days. Cohort 2: participants received 3 mg placebo QD for 14 days.
### Group
**ID:** BG003
**Title:** Total
**Description:** Total of all reporting groups
### Measure
#### Measurement
**Group ID:** BG000
**Spread:** 4.0
**Value:** 73.2
#### Measurement
**Group ID:** BG001
**Spread:** 5.3
**Value:** 76.0
#### Measurement
**Group ID:** BG002
**Spread:** 1.5
**Value:** 72.6
#### Measurement
**Group ID:** BG003
**Spread:** 4.2
**Value:** 74.1
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 4
#### Measurement
**Group ID:** BG001
**Value:** 5
#### Measurement
**Group ID:** BG002
**Value:** 4
#### Measurement
**Group ID:** BG003
**Value:** 13
**Category Title:** Female
#### Measurement
**Group ID:** BG000
**Value:** 2
#### Measurement
**Group ID:** BG001
**Value:** 2
#### Measurement
**Group ID:** BG002
**Value:** 1
#### Measurement
**Group ID:** BG003
**Value:** 5
**Category Title:** Male
**Class Title:**
**Denomination Units Selected:**
## Results Section - Baseline Characteristics Module
### Measure 1
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Age, Continuous
**Unit of Measure:** Years
### Measure 2
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Sex: Female, Male
**Unit of Measure:** Participants
## Results Section - More Information Module
### Certain Agreement
### Limitations and Caveats
**Description:** Cohort 3 was not initiated based on the safety/tolerability results from Cohorts 1 and 2.
### Point of Contact
**Organization:** Otsuka Pharmaceutical Development and Commercialization, Inc.
**Phone:** 800 562-3974
**Title:** Global Medical Affairs
## Results Section - Outcome Measures Module
### Outcome Measure 1
### Outcome Measure 2
### Outcome Measure 3
### Outcome Measure 4
### Outcome Measure 5
### Outcome Measure 6
### Outcome Measure 7
### Outcome Measure 8
### Outcome Measure 9
### Outcome Measure 10
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 6
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 7
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 5
**Title:**
#### Outcome Measure 2
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 12
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 33
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 5
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 6
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 6
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 3
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
#### Outcome Measure 3
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 2
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 3
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 2
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 2
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
#### Outcome Measure 4
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 2
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
#### Outcome Measure 5
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 2
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 3
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 3
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 2
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 2
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 2
**Title:**
#### Outcome Measure 6
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
#### Outcome Measure 7
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 0.5
- **Upper Limit:**
- **Value:** -0.3
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 0.8
- **Upper Limit:**
- **Value:** -0.5
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:** 0.8
- **Upper Limit:**
- **Value:** -1.0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:** N=0, data not collected.
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** NA
- **Upper Limit:**
- **Value:** NA
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 0.4
- **Upper Limit:**
- **Value:** 0.2
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:** 0.7
- **Upper Limit:**
- **Value:** -1.5
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 0.4
- **Upper Limit:**
- **Value:** -0.3
- **Comment:** N=0, data not collected.
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** NA
- **Upper Limit:**
- **Value:** NA
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:** 0.0
- **Upper Limit:**
- **Value:** 0.0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:** N=0, data not collected.
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** NA
- **Upper Limit:**
- **Value:** NA
- **Comment:** N=1, standard deviation is not applicable.
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** NA
- **Upper Limit:**
- **Value:** -1.0
- **Comment:** N=1, standard deviation is not applicable.
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:** NA
- **Upper Limit:**
- **Value:** 0.0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 0.4
- **Upper Limit:**
- **Value:** -0.2
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 0.6
- **Upper Limit:**
- **Value:** 0.0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:** 0.9
- **Upper Limit:**
- **Value:** -0.6
**Title:**
#### Outcome Measure 8
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 0.4
- **Upper Limit:**
- **Value:** -0.2
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 0.0
- **Upper Limit:**
- **Value:** 0.0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:** 0.6
- **Upper Limit:**
- **Value:** -0.5
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:** N=0, data not collected.
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** NA
- **Upper Limit:**
- **Value:** NA
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 0.0
- **Upper Limit:**
- **Value:** 0.0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:** 0.7
- **Upper Limit:**
- **Value:** -0.5
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 0.0
- **Upper Limit:**
- **Value:** 0.0
- **Comment:** N=0, data not collected.
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** NA
- **Upper Limit:**
- **Value:** NA
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:** 0.7
- **Upper Limit:**
- **Value:** -0.5
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:** N=0, data not collected.
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** NA
- **Upper Limit:**
- **Value:** NA
- **Comment:** N=1, standard deviation is not applicable.
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** NA
- **Upper Limit:**
- **Value:** 0.0
- **Comment:** N=1, standard deviation is not applicable.
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:** NA
- **Upper Limit:**
- **Value:** 0.0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 0.0
- **Upper Limit:**
- **Value:** 0.0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 0.0
- **Upper Limit:**
- **Value:** 0.0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:** 0.5
- **Upper Limit:**
- **Value:** -0.4
**Title:**
#### Outcome Measure 9
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 0.0
- **Upper Limit:**
- **Value:** 0.0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 0.4
- **Upper Limit:**
- **Value:** 0.2
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:** 0.0
- **Upper Limit:**
- **Value:** 0.0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:** N=0, data not collected.
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** NA
- **Upper Limit:**
- **Value:** NA
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 0.0
- **Upper Limit:**
- **Value:** 0.0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:** 0.0
- **Upper Limit:**
- **Value:** 0.0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 0.0
- **Upper Limit:**
- **Value:** 0.0
- **Comment:** N=0, data not collected.
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** NA
- **Upper Limit:**
- **Value:** NA
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:** 0.0
- **Upper Limit:**
- **Value:** 0.0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:** N=0, data not collected.
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** NA
- **Upper Limit:**
- **Value:** NA
- **Comment:** N=1, standard deviation is not applicable.
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** NA
- **Upper Limit:**
- **Value:** 0.0
- **Comment:** N=1, standard deviation is not applicable.
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:** NA
- **Upper Limit:**
- **Value:** 0.0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 0.0
- **Upper Limit:**
- **Value:** 0.0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 0.0
- **Upper Limit:**
- **Value:** 0.0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:** 0.0
- **Upper Limit:**
- **Value:** 0.0
**Title:**
#### Outcome Measure 10
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Description:** Safety and tolerability of brexpiprazole was noted to be primary outcome measure. Brexpiprazole was judged to be tolerated if at least 6 out of 8 (75%) of the participants in a test cohort tolerated the dose after 14 days of QD dosing at the end of the fixed dose phase based on the blinded data. Dose toleration was defined as follows: during the course of the trial, the participants did not experience any moderate or severe adverse events (AEs) or potentially clinically relevant changes from Baseline in laboratory values, vital signs, electrocardiogram (ECG) tracings, Columbia-Suicide Severity Rating Scale (C-SSRS), or extrapyramidal symptom (EPS) ratings, which were assessed as possibly related to the study drug, and would have warranted a dose decrease or discontinuation of the study drug. The safety and tolerability of brexpiprazole was defined by parameters: AEs, laboratory values, vital signs, ECG, C-SSRS, or EPS ratings, the results of each of the parameters reported separately.
**Parameter Type:** NUMBER
**Population Description:** Tolerability assessed in phase 1 trial in healthy participants (18-45 years) with MDD as adjunct therapy to ADTs; the efficacy assessed in phase 3 trials in participants (18-65 years) with MDD as adjunct therapy to ADTs. Thus, safety/tolerability of brexpiprazole in participants (\>65 years) with MDD as adjunct therapy to ADTs was not characterized.
**Reporting Status:** POSTED
**Time Frame:** 45 Days
**Title:** Number of Participants Who Tolerated Brexpiprazole
**Type:** PRIMARY
**Unit of Measure:** participants
##### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 2 mg brexpiprazole QD for 14 days, followed by 3 mg brexpiprazole QD for 14 days.
**ID:** OG000
**Title:** Brexpiprazole-Cohort 1
##### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 3 mg brexpiprazole QD for 14 days.
**ID:** OG001
**Title:** Brexpiprazole-Cohort 2
##### Group
**Description:** In the titration phase, participants received 0.5 mg placebo QD for 7 days, followed by 1 mg placebo QD for 7 days. In the fixed dose phase, Cohort 1: participants received 2 mg placebo QD for 14 days, followed by 3 mg placebo QD for 14 days. Cohort 2: participants received 3 mg placebo QD for 14 days.
**ID:** OG002
**Title:** Placebo
#### Outcome Measure 2
**Description:** The AEs were one of the primary parameters to measure the safety and tolerability of individual participants. The AEs were captured for all participants from the time the ICF was signed until the end of the trial. AEs were measured throughout the 14-day titration and 28-day fixed dose phase until follow-up (30 \[±2\] days after last dose of study medication).
**Parameter Type:** NUMBER
**Population Description:** The safety dataset included all randomized participants who received at least one dose of study medication.
**Reporting Status:** POSTED
**Time Frame:** Throughout the study, up to 119 days
**Title:** Number of AEs Reported.
**Type:** PRIMARY
**Unit of Measure:** Events
##### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 2 mg brexpiprazole QD for 14 days, followed by 3 mg brexpiprazole QD for 14 days.
**ID:** OG000
**Title:** Brexpiprazole-Cohort 1
##### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 3 mg brexpiprazole QD for 14 days.
**ID:** OG001
**Title:** Brexpiprazole-Cohort 2
##### Group
**Description:** In the titration phase, participants received 0.5 mg placebo QD for 7 days, followed by 1 mg placebo QD for 7 days. In the fixed dose phase, Cohort 1: participants received 2 mg placebo QD for 14 days, followed by 3 mg placebo QD for 14 days. Cohort 2: participants received 3 mg placebo QD for 14 days.
**ID:** OG002
**Title:** Placebo
#### Outcome Measure 3
**Description:** The laboratory values were one of the primary parameters to measure the safety and tolerability of individual participants. Incidence of TEAEs of potential clinical relevance include abnormal values in serum chemistry, hematology, urinalyses and prolactin tests that were identified based on pre-defined criteria.
**Parameter Type:** NUMBER
**Population Description:** The safety dataset included all randomized participants who received at least one dose of study medication.
**Reporting Status:** POSTED
**Time Frame:** Titration Day 7, Fixed dose Day 14 and 28 and Last Visit
**Title:** Incidence of Laboratory Values of Potential Clinical Significance
**Type:** PRIMARY
**Unit of Measure:** Participants
##### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 2 mg brexpiprazole QD for 14 days, followed by 3 mg brexpiprazole QD for 14 days.
**ID:** OG000
**Title:** Brexpiprazole-Cohort 1
##### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 3 mg brexpiprazole QD for 14 days.
**ID:** OG001
**Title:** Brexpiprazole-Cohort 2
##### Group
**Description:** In the titration phase, participants received 0.5 mg placebo QD for 7 days, followed by 1 mg placebo QD for 7 days. In the fixed dose phase, Cohort 1: participants received 2 mg placebo QD for 14 days, followed by 3 mg placebo QD for 14 days. Cohort 2: participants received 3 mg placebo QD for 14 days.
**ID:** OG002
**Title:** Placebo
#### Outcome Measure 4
**Description:** The vital signs were one of the primary parameters to measure the safety and tolerability of individual participants. Incidence of TEAEs of potential clinical relevance included abnormal values in heart rate, systolic and diastolic blood pressure, respiratory rate and weight that were identified based on pre-defined criteria.
**Parameter Type:** NUMBER
**Population Description:** The safety dataset included all randomized participants who received at least one dose of study medication.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Titration Day 1, 2, 7, 8, Fixed Days 1, 2, 14, 15, 28, 29, Early Termination and Last Visit.
**Title:** Incidence of Vital Signs of Potential Clinical Significance
**Type:** PRIMARY
**Unit of Measure:** Participants
##### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 2 mg brexpiprazole QD for 14 days, followed by 3 mg brexpiprazole QD for 14 days.
**ID:** OG000
**Title:** Brexpiprazole-Cohort 1
##### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 3 mg brexpiprazole QD for 14 days.
**ID:** OG001
**Title:** Brexpiprazole-Cohort 2
##### Group
**Description:** In the titration phase, participants received 0.5 mg placebo QD for 7 days, followed by 1 mg placebo QD for 7 days. In the fixed dose phase, Cohort 1: participants received 2 mg placebo QD for 14 days, followed by 3 mg placebo QD for 14 days. Cohort 2: participants received 3 mg placebo QD for 14 days.
**ID:** OG002
**Title:** Placebo
#### Outcome Measure 5
**Description:** The measurement of ECG was one of the primary parameters to measure the safety and tolerability of individual participants. Incidence of TEAEs of potential clinical relevance include abnormal changes in heart rate and ECG intervals of PR, QRS, QT, QTcB, and QTcF that were identified based on pre-defined criteria.
**Parameter Type:** NUMBER
**Population Description:** The safety dataset included all randomized participants who received at least one dose of study medication.
**Reporting Status:** POSTED
**Time Frame:** Titratrion Day 1 and 7, Fixed dose Day 1, 14, 28, Early Termination
**Title:** Incidence of ECG Evaluations of Potential Clinical Significance
**Type:** PRIMARY
**Unit of Measure:** Participants
##### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 2 mg brexpiprazole QD for 14 days, followed by 3 mg brexpiprazole QD for 14 days.
**ID:** OG000
**Title:** Brexpiprazole-Cohort 1
##### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 3 mg brexpiprazole QD for 14 days.
**ID:** OG001
**Title:** Brexpiprazole-Cohort 2
##### Group
**Description:** In the titration phase, participants received 0.5 mg placebo QD for 7 days, followed by 1 mg placebo QD for 7 days. In the fixed dose phase, Cohort 1: participants received 2 mg placebo QD for 14 days, followed by 3 mg placebo QD for 14 days. Cohort 2: participants received 3 mg placebo QD for 14 days.
**ID:** OG002
**Title:** Placebo
#### Outcome Measure 6
**Description:** The physical examination evaluation was one of the primary parameters to measure the safety and tolerability of individual participants. Incidence of TEAEs of potential clinical relevance include abnormal changes in the following body systems: head, ears, eyes, nose, and throat; thorax; abdomen; urogenital; extremities; neurological; and skin and mucosae.
**Parameter Type:** NUMBER
**Population Description:** Safety Sample was analyzed. Any clinically significant condition present at the post-treatment physical examination that was not present at the baseline examination was documented as an adverse event and followed to a satisfactory conclusion. There were no clinically significant physical examination findings reported in this study.
**Reporting Status:** POSTED
**Time Frame:** Physical examination was performed at Screening, check-in, and discharge
**Title:** Incidence of Physical Examination Evaluation of Potential Clinical Significance
**Type:** PRIMARY
**Unit of Measure:** Participants
##### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 2 mg brexpiprazole QD for 14 days, followed by 3 mg brexpiprazole QD for 14 days.
**ID:** OG000
**Title:** Brexpiprazole-Cohort 1
##### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 3 mg brexpiprazole QD for 14 days.
**ID:** OG001
**Title:** Brexpiprazole-Cohort 2
##### Group
**Description:** In the titration phase, participants received 0.5 mg placebo QD for 7 days, followed by 1 mg placebo QD for 7 days. In the fixed dose phase, Cohort 1: participants received 2 mg placebo QD for 14 days, followed by 3 mg placebo QD for 14 days. Cohort 2: participants received 3 mg placebo QD for 14 days.
**ID:** OG002
**Title:** Placebo
#### Outcome Measure 7
**Description:** EPS was one of the primary parameters to measure the safety and tolerability of individual participants. The SAS is a rating scale used to measure EPS. The SAS scale consists of a list of 10 symptoms of parkinsonism (gait, arm dropping, shoulder shaking, elbow rigidity, wrist rigidity, head rotation, glabella tap, tremor, salivation, and akathisia), with each item rated from 0 to 4, with 0 being normal and 4 being the worst. The SAS Total score is sum of ratings for all 10 items, with possible Total scores from 0 to 40.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** Safety Sample includes all randomized participants who receive at least one dose of study medication.
**Reporting Status:** POSTED
**Time Frame:** End of Titration, Day 15, Day 29, Early Termination and Last visit
**Title:** Mean Change From Baseline to Study Completion in Simpson-Angus Scale (SAS) Total Score
**Type:** PRIMARY
**Unit of Measure:** Units on a scale
##### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 2 mg brexpiprazole QD for 14 days, followed by 3 mg brexpiprazole QD for 14 days.
**ID:** OG000
**Title:** Brexpiprazole-Cohort 1
##### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 3 mg brexpiprazole QD for 14 days.
**ID:** OG001
**Title:** Brexpiprazole-Cohort 2
##### Group
**Description:** In the titration phase, participants received 0.5 mg placebo QD for 7 days, followed by 1 mg placebo QD for 7 days. In the fixed dose phase, Cohort 1: participants received 2 mg placebo QD for 14 days, followed by 3 mg placebo QD for 14 days. Cohort 2: participants received 3 mg placebo QD for 14 days.
**ID:** OG002
**Title:** Placebo
#### Outcome Measure 8
**Description:** EPS was one of the primary parameters to measure the safety and tolerability of individual participants. The Barnes Akathisia Rating Scale was an EPS rating scale. The Barnes Akathisia Rating Scale was used to assess the presence and severity of akathisia. This scale consists of 4 items. Only the 4th item, the Global Clinical Assessment of Akathisia, was evaluated in this trial. This item is rated on a 6 point scale, with 0 being best (absent) and 5 being worst (severe akathisia).
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** Safety Sample includes all randomized participants who receive at least one dose of study medication.
**Reporting Status:** POSTED
**Time Frame:** End of Titration, Day 15, Day 29, Early Termination and Last visit
**Title:** Mean Change From Baseline to Study Completion in Barnes Akathisia Global Score
**Type:** PRIMARY
**Unit of Measure:** Units on a scale
##### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 2 mg brexpiprazole QD for 14 days, followed by 3 mg brexpiprazole QD for 14 days.
**ID:** OG000
**Title:** Brexpiprazole-Cohort 1
##### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 3 mg brexpiprazole QD for 14 days.
**ID:** OG001
**Title:** Brexpiprazole-Cohort 2
##### Group
**Description:** In the titration phase, participants received 0.5 mg placebo QD for 7 days, followed by 1 mg placebo QD for 7 days. In the fixed dose phase, Cohort 1: participants received 2 mg placebo QD for 14 days, followed by 3 mg placebo QD for 14 days. Cohort 2: participants received 3 mg placebo QD for 14 days.
**ID:** OG002
**Title:** Placebo
#### Outcome Measure 9
**Description:** EPS was one of the primary parameters to measure the safety and tolerability of individual participants. The AIMS Scale was an EPS rating scale. The AIMS is a 12 item scale. The first 10 items are rated from 0 to 4 (0=best, 4=worst). Items 11 and 12, related to dental status, have dichotomous responses, 0=no and 1=yes. The AIMS Total Score is the sum of the ratings for the first seven items. The possible total scores are from 0 to 28.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** Safety Sample includes all randomized participants who receive at least one dose of study medication.
**Reporting Status:** POSTED
**Time Frame:** End of Titration, Day 15, Day 29, Early Termination and Last visit
**Title:** Mean Change From Baseline to Study Completion in Abnormal Involuntary Movement Scale (AIMS) Rating Score.
**Type:** PRIMARY
**Unit of Measure:** Units on a scale
##### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 2 mg brexpiprazole QD for 14 days, followed by 3 mg brexpiprazole QD for 14 days.
**ID:** OG000
**Title:** Brexpiprazole-Cohort 1
##### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 3 mg brexpiprazole QD for 14 days.
**ID:** OG001
**Title:** Brexpiprazole-Cohort 2
##### Group
**Description:** In the titration phase, participants received 0.5 mg placebo QD for 7 days, followed by 1 mg placebo QD for 7 days. In the fixed dose phase, Cohort 1: participants received 2 mg placebo QD for 14 days, followed by 3 mg placebo QD for 14 days. Cohort 2: participants received 3 mg placebo QD for 14 days.
**ID:** OG002
**Title:** Placebo
#### Outcome Measure 10
**Description:** The C-SSRS was one of the primary parameters to measure the safety and tolerability of individual participants. Suicidality was monitored during the trial using the C-SSRS. This scale consists of a baseline evaluation that assesses the lifetime experience of the participant with suicide events and suicidal ideation and a post-baseline evaluation that focuses on suicidality since the last trial visit.
**Parameter Type:** NUMBER
**Population Description:** Safety Sample includes all randomized participants who receive at least one dose of study medication.
**Reporting Status:** POSTED
**Time Frame:** Baseline, End of Titration, Fixed dose Day 14 and 28, Day 15 and 29, Early Termination, Last Visit
**Title:** Change From Baseline to Study Completion in C-SSRS Score.
**Type:** PRIMARY
**Unit of Measure:** Participants
##### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 2 mg brexpiprazole QD for 14 days, followed by 3 mg brexpiprazole QD for 14 days.
**ID:** OG000
**Title:** Brexpiprazole-Cohort 1
##### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 3 mg brexpiprazole QD for 14 days.
**ID:** OG001
**Title:** Brexpiprazole-Cohort 2
##### Group
**Description:** In the titration phase, participants received 0.5 mg placebo QD for 7 days, followed by 1 mg placebo QD for 7 days. In the fixed dose phase, Cohort 1: participants received 2 mg placebo QD for 14 days, followed by 3 mg placebo QD for 14 days. Cohort 2: participants received 3 mg placebo QD for 14 days.
**ID:** OG002
**Title:** Placebo
### Participant Flow Module
#### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 2 mg brexpiprazole QD for 14 days, followed by 3 mg brexpiprazole QD for 14 days.
**ID:** FG000
**Title:** Brexpiprazole-Cohort 1
#### Group
**Description:** In the titration phase, participants received 0.5 mg brexpiprazole QD for 7 days, followed by 1 mg brexpiprazole QD for 7 days. In the fixed dose phase, participants received 3 mg brexpiprazole QD for 14 days.
**ID:** FG001
**Title:** Brexpiprazole-Cohort 2
#### Group
**Description:** In the titration phase, participants were to receive 0.5 mg brexpiprazole or placebo QD for 7 days, followed by 1 mg brexpiprazole or placebo QD for 7 days, and then 2 mg brexpiprazole or placebo QD for 7 days. In the fixed dose phase, participants were to receive 3 mg brexpiprazole or placebo QD for 14 days. However, Cohort 3 was not conducted due to safety and tolerability results from Cohorts 1 and 2.
**ID:** FG002
**Title:** Brexpiprazole-Cohort 3
#### Group
**Description:** In the titration phase, participants received 0.5 mg placebo QD for 7 days, followed by 1 mg placebo QD for 7 days. In the fixed dose phase, Cohort 1: participants received 2 mg placebo QD for 14 days, followed by 3 mg placebo QD for 14 days. Cohort 2: participants received 3 mg placebo QD for 14 days.
**ID:** FG003
**Title:** Placebo
#### Period
**Title:** Overall Study
##### Withdraw
**Type:** Adverse Event
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 1
###### Reason
**Group ID:** FG002
**Number of Subjects:** 0
###### Reason
**Group ID:** FG003
**Number of Subjects:** 0
##### Withdraw
**Type:** Physician Decision
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 1
###### Reason
**Group ID:** FG002
**Number of Subjects:** 0
###### Reason
**Group ID:** FG003
**Number of Subjects:** 0
##### Withdraw
**Type:** Participant met withdrawal criteria
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
###### Reason
**Group ID:** FG002
**Number of Subjects:** 0
###### Reason
**Group ID:** FG003
**Number of Subjects:** 1
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 6
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 7
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 5
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 6
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 5
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 4
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 2
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 1
**Pre-Assignment Details:** The study included a 30-day screening period, a 14-day washout period, up to 45-day inpatient treatment period (titration: received brexpiprazole or placebo once daily \[QD\] for 14 or 21 days and fixed dose phase: received assigned fixed dose for 14 or 28 days), and a 30-day follow-up after the last dose of study drug.
**Recruitment Details:** This was a phase 1, multicenter, randomized, double-blind, placebo-controlled, multiple ascending dose trial planned in 3 sequential cohorts of elderly participants (70 to 85 years old) with major depressive disorder (MDD). Brexpiprazole was administered as an adjunct treatment to the current antidepressant therapy that the participant received.
**Has Results:** True |
## Protocol Section
### Identification Module
**NCT ID:** NCT04558879
**Brief Title:** Exercise and Sleep in Parkinson's Disease
**Official Title:** The Effect of Different Exercise Modalities on Sleep Quality and Architecture in People With Parkinson's Disease
#### Organization Study ID Info
**ID:** 35450
#### Organization
**Class:** OTHER
**Full Name:** McGill University
### Status Module
#### Completion Date
**Date:** 2025-12-01
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-03-15
**Type:** ACTUAL
**Last Update Submit Date:** 2024-03-13
**Overall Status:** RECRUITING
#### Primary Completion Date
**Date:** 2024-12-01
**Type:** ESTIMATED
#### Start Date
**Date:** 2021-09-15
**Type:** ACTUAL
**Status Verified Date:** 2024-03
#### Study First Post Date
**Date:** 2020-09-22
**Type:** ACTUAL
**Study First Submit Date:** 2020-09-09
**Study First Submit QC Date:** 2020-09-16
### Sponsor Collaborators Module
#### Collaborators
**Class:** OTHER
**Name:** Jewish Rehabilitation Hospital
#### Lead Sponsor
**Class:** OTHER
**Name:** McGill University
#### Responsible Party
**Investigator Affiliation:** McGill University
**Investigator Full Name:** Marc Roig
**Investigator Title:** Assistant Professor
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** This study will investigate the impact of two common exercise modalities, cardiovascular and resistance training, on sleep quality and architecture in persons with Parkinson's disease (PD), and whether these potential positive changes in sleep are associated with improvements in brain plasticity and different quality of life (QoL)-related aspects. Participants will perform either cardiovascular training (CT) or resistance training (RT) for 12 weeks, at least two times/week. The assessments will be performed at baseline and after training by an assessor blinded to the participants' group allocation.
**Detailed Description:** Background: Over 100,000 Canadians are currently living with PD. Every year, 6.600 new cases are diagnosed and this number is expected to double by 2031. Most (98%) of those persons experience sleep problems, which can appear even before the onset of the cardinal motor symptoms of the disease, affecting multiple aspects of their QoL. Persons with PD also show alterations in sleep architecture, which have been associated with faster disease progression. Since medications used to reduce sleep problems in PD have potential adverse side effects, exercise has been proposed as a potential non-pharmacological alternative to improve sleep quality and architecture in people with PD. However, the most beneficial type of intervention to improve sleep in this clinical population is still to be determined.
Objective: 1) To conduct a 12-week RCT comparing the effects of CT and RT on both objective and subjective measures of sleep quality and architecture in patients with mild-to-moderate PD; 2) To assess whether, regardless of the type of exercise, positive changes in sleep quality and architecture mediate exercise-induced improvements in cognitive and motor function as well as in different aspects that directly impact on QoL; 3) To explore whether, regardless of the type of exercise used, positive changes in sleep architecture will be associated with improvements in brain plasticity and motor learning.
Design: a single-blinded RCT in which assessments will be performed at baseline (pre) and after (post) training by an assessor blinded to the participants' group allocation.
Outcomes: 1) objective and subjective sleep quality as well as sleep architecture; 2) cognitive and motor function as well as fatigue, psychological functioning, and QoL; 3) motor learning and brain plasticity.
Methods: Changes in objective (i.e. sleep efficiency) and subjective measures of sleep quality will be assessed with actigraphy and the PD sleep scale version 2, respectively. Sleep architecture will be measured with polysomnography. Motor and cognitive function will be assessed with the Unified PD Rating Scale and the Scale for Outcomes in PD-Cognition, respectively. Fatigue, psychosocial functioning and QoL will be assessed with the PD Fatigue Scale, the Scale for Outcomes in PD-Psychosocial and the PD QoL Scale, respectively. Motor learning will be assessed using a visuomotor tracking task; whereas brain plasticity will be measured with transcranial magnetic stimulation applied over the primary motor cortex.
Expected results: 1) CT will be more effective than RT in improving objective and subjective sleep quality. RT and CT will be equally effective in improving sleep architecture; 2) Improvements in sleep quality and architecture will be associated with enhancements in cognition, motor function and different QoL-related aspects; 3) Positive changes in sleep architecture will mediated increases in brain plasticity and motor learning.
Impact: This will be the first study comparing the effect of CT and RT on sleep quality and architecture and exploring associations with cognitive and motor function as well as aspects that directly impact QoL. The results of the study will provide important information to design more personalized exercise-based treatments, which are patient-oriented and aimed to mitigate sleep complains in this clinical population.
### Conditions Module
**Conditions:**
- Parkinson Disease
**Keywords:**
- Parkinson's disease
- Rehabilitation
- Neurosciences
- Sleep
- Exercise
- Cardiovascular training
- Resistance training
- Cognition
- Motor function
- Quality of life
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 60
**Type:** ESTIMATED
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Cardiovascular training (CT) will be performed on a recumbent stepper. CT will start at low intensity, and through a linear progression will reach vigorous intensity; then, this intensity will be maintained until the end of the intervention. Each session will include five minutes of warm-up and cool-down performed at the beginning and the end of the training, respectively. Furthermore, five minutes of stretching will be performed after the cool down. CT's sessions will approximately last 45 minutes and will be interspersed with at least 48 hours of recovery.
**Intervention Names:**
- Behavioral: CT
**Label:** Cardiovascular training
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Resistance training (RT) intensity will be estimated using the percentage of one-maximal repetition (1-RM) defined as the maximal weight liftable for ten maximal repetitions with proper form. The program will include five exercises (leg press, lat machine, leg extension, leg curl, bench press) and will start at high-volume low-intensity. RT will follow a periodization to reach high-intensity low-volume at the end of the intervention (week 12). The training sessions will start with five-minute of warm-up performed on a recumbent stepper and will end with five-minute of stretching (cool-down). RT's sessions will approximately last 45 minutes and will be interspersed with at least 48 hours of recovery.
**Intervention Names:**
- Behavioral: RT
**Label:** Resistance training
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Cardiovascular training
**Description:** 12 weeks of exercise Cardiovascular Training
**Name:** CT
**Type:** BEHAVIORAL
#### Intervention 2
**Arm Group Labels:**
- Resistance training
**Description:** 12 weeks of exercise Resistance Training
**Name:** RT
**Type:** BEHAVIORAL
### Outcomes Module
#### Other Outcomes
**Description:** Paired-pulse of transcranial magnetic stimulation (TMS) protocol to estimate variation in cortical excitability measured as motor evoked potential (MEP).
**Measure:** Intra-cortical facilitation
**Time Frame:** 12 weeks
**Description:** Paired-pulse of transcranial magnetic stimulation (TMS) protocol to estimate variation in cortical excitability measured as motor evoked potential (MEP).
**Measure:** Short intra-cortical inhibition
**Time Frame:** 12 weeks
**Description:** Paired-pulse of transcranial magnetic stimulation (TMS) protocol to estimate variation in cortical excitability measured as length of the pause in electromyographic activity.
**Measure:** Silent period
**Time Frame:** 12 weeks
**Description:** Maximum rate of oxygen consumption measured during maximum physical effort.
**Measure:** Cardiorespiratory fitness
**Time Frame:** 12 weeks
#### Primary Outcomes
**Description:** Actigraphy; SE = total sleep time/time spent in bed.
**Measure:** Sleep efficiency (SE)
**Time Frame:** 12 weeks
**Description:** Parkinson's Disease Sleep Scale version 2 (PDSS-2); Score range from 0-60; higher scores represent worse sleep quality.
**Measure:** Subjective sleep quality
**Time Frame:** 12 weeks
**Description:** Polysomnography combined with electroencephalogram
**Measure:** Objective sleep measurements, including duration and percentage of sleep stages, total sleep time (TLT), wake after sleep onset (WASO), sleep latency (SL).
**Time Frame:** 12 weeks
#### Secondary Outcomes
**Description:** Unified Parkinson's Disease Rating Scale part III; Scores range from 0-56; higher scores represent a worse motor function.
**Measure:** Motor function
**Time Frame:** 12 weeks
**Description:** Scale for Outcomes in Parkinson's Disease-Cognition; Scores range from 0-43; higher scores reflect better performance.
**Measure:** Cognition
**Time Frame:** 12 weeks
**Description:** Parkinson's Disease Fatigue Scale; Scores range from 16-80; higher scores reflect a higher presence of fatigue.
**Measure:** Fatigue
**Time Frame:** 12 weeks
**Description:** Scale for Outcomes in Parkinson's Disease-Psychosocial; Scores range from 0-33; higher scores reflect a worse psychosocial functioning.
**Measure:** Psychosocial functioning
**Time Frame:** 12 weeks
**Description:** Parkinson's Disease Quality of Life Scale; Scores range from 0-128; higher scores indicate lower quality of life.
**Measure:** Quality of life-related aspects in Parkinson's disease
**Time Frame:** 12 weeks
**Description:** Visuomotor tracking task; accuracy in performing a novel motor task with the dominant hand.
**Measure:** Motor learning
**Time Frame:** 12 weeks
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Persons with mild-moderate idiopathic Parkinson's Disease (Modified Hoehn \& Yahr Scale stages 1-3);
* On a stable dosage of medication during the previous month;
* Having poor sleep quality defined as a score \> 18 in the PDSS-2(scores above this cut-off value define clinically relevant sleep disorders);
Exclusion Criteria:
* Having atypical parkinsonism, dementia or any other neurological, psychiatric or cardiovascular comorbidity affecting the ability to perform exercise;
* Presenting severe untreated obstructive sleep apnea (OSA);
* Having a Montreal Cognitive Assessment (MoCA) score \<21
* Having a Beck Depression Inventory (BDI version 2) score \>4
* Having absolute contraindications to exercise and to undergo transcranial magnetic stimulation (TMS);
* Currently are or will be enrolled in a drug or exercise trial during the duration of the study;
* Having participated in a structured exercise program \> 2 times per week in the two months prior to the enrollment in the study
**Healthy Volunteers:** True
**Maximum Age:** 80 Years
**Minimum Age:** 45 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Marc Roig, PhD
**Phone:** 514-398-4400
**Phone Ext:** 00841
**Role:** CONTACT
#### Locations
**Location 1:**
**City:** Laval
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Marc Roig, PhD
- **Phone:** 450-688-9550
- **Phone Ext:** 4677
- **Role:** CONTACT
***Contact 2:***
- **Name:** Josephine Salib, MSc
- **Phone:** 450-688-9550
- **Phone Ext:** 4217
- **Role:** CONTACT
**Country:** Canada
**Facility:** Jewish Rehabiliation Hospital
**State:** Quebec
**Status:** RECRUITING
**Zip:** H7V 1R2
### IPD Sharing Statement Module
**Description:** Results will be published in peer-review journals.
**IPD Sharing:** NO
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000020734
- Term: Parkinsonian Disorders
- ID: D000001480
- Term: Basal Ganglia Diseases
- ID: D000001927
- Term: Brain Diseases
- ID: D000002493
- Term: Central Nervous System Diseases
- ID: D000009422
- Term: Nervous System Diseases
- ID: D000009069
- Term: Movement Disorders
- ID: D000080874
- Term: Synucleinopathies
- ID: D000019636
- Term: Neurodegenerative Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC10
- Name: Nervous System Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: BC17
- Name: Skin and Connective Tissue Diseases
- Abbrev: BC18
- Name: Nutritional and Metabolic Diseases
- Abbrev: BXM
- Name: Behaviors and Mental Disorders
### Condition Browse Module - Browse Leaves
- ID: M13213
- Name: Parkinson Disease
- Relevance: HIGH
- As Found: Parkinson's Disease
- ID: M22494
- Name: Parkinsonian Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M25603
- Name: Ganglion Cysts
- Relevance: LOW
- As Found: Unknown
- ID: M16358
- Name: Synovial Cyst
- Relevance: LOW
- As Found: Unknown
- ID: M4774
- Name: Basal Ganglia Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5204
- Name: Brain Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5742
- Name: Central Nervous System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M12029
- Name: Movement Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M2217
- Name: Synucleinopathies
- Relevance: LOW
- As Found: Unknown
- ID: M21558
- Name: Neurodegenerative Diseases
- Relevance: LOW
- As Found: Unknown
- ID: T6034
- Name: Quality of Life
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000010300
- Term: Parkinson Disease
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT03166579
**Brief Title:** Effectiveness Evaluation of 'The Endeavour Programme'
**Official Title:** Evaluating the Effectiveness of 'The Endeavour Programme': DBT for Irish Adults With Borderline Personality Disorder
#### Organization Study ID Info
**ID:** NOSP
#### Organization
**Class:** OTHER
**Full Name:** Health Service Executive, Ireland
### Status Module
#### Completion Date
**Date:** 2016-08
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2017-05-25
**Type:** ACTUAL
**Last Update Submit Date:** 2017-05-23
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2016-08
**Type:** ACTUAL
#### Start Date
**Date:** 2010-07
**Type:** ACTUAL
**Status Verified Date:** 2017-05
#### Study First Post Date
**Date:** 2017-05-25
**Type:** ACTUAL
**Study First Submit Date:** 2017-05-22
**Study First Submit QC Date:** 2017-05-23
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Health Service Executive, Ireland
#### Responsible Party
**Investigator Affiliation:** Health Service Executive, Ireland
**Investigator Full Name:** Mary Joyce
**Investigator Title:** Project Co-ordinator
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** This study aims to evaluate the effectiveness of Dialectical Behaviour Therapy for adults with Borderline Personality Disorder attending Community Mental Health Services in Cork, Ireland.
The main objective of the current study is to determine if completion of a 12 month DBT programme is associated with improved outcomes in terms of borderline symptoms, anxiety, hopelessness, suicidal behaviour, depression and quality of life.
A secondary objective includes assessing client progress across multiple time-points throughout the treatment.
**Detailed Description:** Dialectical Behaviour Therapy (DBT) is noted to be an intervention with a growing evidence base which demonstrates its effectiveness in treating individuals with Borderline Personality Disorder (BPD). Nine randomised controlled trials have been completed at six independent sites. These trials have found a reduction in suicidal behaviours, intentional self-injury, depression, hopelessness and other difficulties associated with this mental health diagnosis.
While the efficacy of DBT has been demonstrated through multiple RCTs, few studies have examined the effectiveness of DBT in community mental health settings. In particular, no study to our knowledge has evaluated the standard 12-month DBT programme for adults with BPD in an Irish community setting. "Standard" DBT is delivered by a team of multidisciplinary mental health professionals, and comprises of individual therapy sessions for each patient, group skills training sessions, phone coaching and consultation meetings for the clinicians on the DBT team.
The current study thus aims to evaluate the effectiveness of the standard 12-month DBT programme (The Endeavour Programme) in an Irish community setting.
### Conditions Module
**Conditions:**
- Personality Disorder, Borderline
### Design Module
#### Design Info
**Allocation:** NA
**Intervention Model:** SINGLE_GROUP
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 71
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Participants receive the "standard" 12 month DBT programme where all four modes of treatment are delivered including: individual therapy, group skills training, telephone coaching and DBT team consultation.
**Intervention Names:**
- Behavioral: Dialectical Behaviour Therapy
**Label:** Dialectical Behaviour Therapy
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Dialectical Behaviour Therapy
**Description:** Dialectical Behaviour Therapy (DBT) is a psychological intervention which was originally developed for women with Borderline Personality Disorder. DBT is delivered by a team of multidisciplinary mental health professionals, and comprises of individual therapy sessions for each patient, group skills training sessions, phone coaching and consultation meetings for the clinicians on the DBT team. Group skills are delivered in blocks of three modules which teach mindfulness, distress tolerance, emotion regulation and interpersonal effectiveness. The three modules are delivered over a 24-week period and are then repeated.
**Name:** Dialectical Behaviour Therapy
**Type:** BEHAVIORAL
### Outcomes Module
#### Primary Outcomes
**Description:** Borderline Symptom List (BSL-23). The BSL-23 is a self-report questionnaire which comprises 23 items measuring borderline-typical symptomatology.
**Measure:** Borderline Symptoms
**Time Frame:** 12 months from pre-intervention to post-intervention
**Description:** Beck Scale for Suicide Ideation (BSS). The BSS is a 21-item self-report assessment developed to identify individuals at risk of suicide.
**Measure:** Suicidal ideation
**Time Frame:** 12 months from pre-intervention to post-intervention
**Description:** Beck Hopelessness Scale (BHS). The BHS is a 20 item self-report measure which assesses key aspects of hopelessness.
**Measure:** Hopelessness
**Time Frame:** 12 months from pre-intervention to post-intervention
**Description:** Beck Depression Inventory - Second Edition (BDI-II). The BDI-II is a 21 item self-report measure of symptoms and attitudes related to depression.
**Measure:** Depression
**Time Frame:** 12 months from pre-intervention to post-intervention
**Description:** Beck Anxiety Inventory (BAI). The BAI is a 21 is self-report multiple choice survey which evaluates both physiological and cognitive symptoms of anxiety
**Measure:** Anxiety
**Time Frame:** 12 months from pre-intervention to post-intervention
**Description:** World Health Organisation Quality of Life Questionnaire (WHOQOL-BREF). The WHOQOL-BREF was developed collaboratively in a number of centres to act as an international cross-culturally comparable quality of life assessment. It comprises 26 items across four domains: physical health (domain 1), psychological health (domain 2), social relationships (domain 3), and environment (domain 4).
**Measure:** Quality of Life
**Time Frame:** 12 months from pre-intervention to post-intervention
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Meet criteria for diagnosis of either Borderline Personality Disorder (DSM IV/V) or Emotionally Unstable Personality Disorder (ICD-10)
* Current emotion and behavioural dysregulation
* A history of self-harm behaviour
* The client will have discussed the diagnosis with a member of the DBT team and will have expressed an interest in, and commitment to the 12 month programme.
Exclusion Criteria:
* An active psychosis
* If the client has severe developmental delays, cognitive impairment or learning disabilities (exceeding mild range)
* If a clients' substance/drug dependence, eating disorder or any other mental health issue/behaviour is at such a level that it would impede their engaging with any of the modalities of DBT
**Healthy Volunteers:** True
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Overall Officials
**Official 1:**
**Affiliation:** Psychology Manager
**Name:** Daniel Flynn, PGDip
**Role:** PRINCIPAL_INVESTIGATOR
### IPD Sharing Statement Module
**IPD Sharing:** NO
### References Module
#### References
**Citation:** Flynn D, Kells M, Joyce M, Corcoran P, Gillespie C, Suarez C, Weihrauch M, Cotter P. Standard 12 month dialectical behaviour therapy for adults with borderline personality disorder in a public community mental health setting. Borderline Personal Disord Emot Dysregul. 2017 Sep 23;4:19. doi: 10.1186/s40479-017-0070-8. eCollection 2017.
**PMID:** 28989706
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000001523
- Term: Mental Disorders
### Condition Browse Module - Browse Branches
- Abbrev: BXM
- Name: Behaviors and Mental Disorders
- Abbrev: All
- Name: All Conditions
### Condition Browse Module - Browse Leaves
- ID: M13462
- Name: Personality Disorders
- Relevance: HIGH
- As Found: Personality Disorder
- ID: M5161
- Name: Borderline Personality Disorder
- Relevance: HIGH
- As Found: Personality Disorder, Borderline
- ID: M4815
- Name: Mental Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M14473
- Name: Psychotic Disorders
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000010554
- Term: Personality Disorders
- ID: D000001883
- Term: Borderline Personality Disorder
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT00600379
**Brief Title:** Virtual Reality Training Program for Ambulatory Patients With Chronic Gait Deficits After Stroke
**Official Title:** A Randomized Controlled Trial of a Virtual Reality Training Program for Ambulatory Patients With Mild-to-moderate Chronic Gait Deficits After Stroke
#### Organization Study ID Info
**ID:** SHEBA-07-4780-DT-CTIL
#### Organization
**Class:** OTHER_GOV
**Full Name:** Sheba Medical Center
### Status Module
#### Completion Date
**Date:** 2010-10
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2011-06-16
**Type:** ESTIMATED
**Last Update Submit Date:** 2011-06-15
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2010-07
**Type:** ACTUAL
#### Start Date
**Date:** 2008-01
**Status Verified Date:** 2011-06
#### Study First Post Date
**Date:** 2008-01-25
**Type:** ESTIMATED
**Study First Submit Date:** 2008-01-14
**Study First Submit QC Date:** 2008-01-24
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER_GOV
**Name:** Sheba Medical Center
#### Responsible Party
**Old Name Title:** Prof. David Tanne/Director, Stroke Center
**Old Organization:** Sheba Medical Center
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** The purpose of this study is to examine the feasibility and efficacy of a virtual reality program for ambulatory patients with mild-to-moderate chronic gait deficits after stroke.
**Detailed Description:** Virtual reality (VR) systems enable the learning of simple and complex skills in a controlled virtual environment; i.e., one in which the different components (constraints) of the environment can be displayed, graded, changed and monitored in a quantitative manner.
Small preliminary studies suggest that VR may be used to augment chronic stroke rehabilitation and may enhance cortical reorganization. Our aim is to examine the feasibility and efficacy of a virtual reality program for ambulatory patients with mild-to-moderate chronic gait deficits after stroke. Study design-a single center randomized controlled trial of an experimental group and a usual care group.
### Conditions Module
**Conditions:**
- Stroke
**Keywords:**
- stroke chronic gait deficits virtual reality training
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 48
**Type:** ESTIMATED
**Phases:**
- PHASE2
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Virtual Reality training for an overall of 18 sessions 2/week + usual care.
**Intervention Names:**
- Device: Virtual reality system (CAREN™ Integrated Reality System; MOTEK BV, Netherlands).
**Label:** A,
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Usual care
**Label:** B,
**Type:** NO_INTERVENTION
### Interventions
#### Intervention 1
**Arm Group Labels:**
- A,
**Description:** Training 2/w for 9 weeks (total 18 sessions).
**Name:** Virtual reality system (CAREN™ Integrated Reality System; MOTEK BV, Netherlands).
**Type:** DEVICE
### Outcomes Module
#### Primary Outcomes
**Measure:** Community ambulation using Step Activity Monitor (SAM)
**Time Frame:** Baseline X2, post training (week 9-10), retention (week 20-24)
**Measure:** Gait analysis (GaitRite system) including dual task
**Time Frame:** Baseline X2, post training (week 9-10), retention (week 20-24)
**Measure:** Body sway- displacement of center of pressure (CoP) as indicated by the reactive forces from platform.
**Time Frame:** Baseline X2, post training (week 9-10), retention (week 20-24)
**Measure:** Timed Up and Go
**Time Frame:** Baseline X2, post training (week 9-10), retention (week 20-24)
#### Secondary Outcomes
**Measure:** Functional Reach
**Time Frame:** Baseline X2, post training (week 9-10), retention (week 20-24)
**Measure:** Four Stick Stepping Test (FSST)
**Time Frame:** Baseline X2, post training (week 9-10), retention (week 20-24)
**Measure:** 3DGait Analysis system
**Time Frame:** Baseline X2, post training (week 9-10), retention (week 20-24)
**Measure:** 6 minute walk
**Time Frame:** Baseline X2, post training (week 9-10), retention (week 20-24)
**Measure:** Self-induced perturbations and reaction to perturbations on platform
**Time Frame:** Baseline X2, post training (week 9-10), retention (week 20-24)
### Eligibility Module
**Eligibility Criteria:** Main Inclusion Criteria:
* Stroke within 3-72 months.
* Mild-to-moderate residual gait deficits after the index stroke with preserved capacity for ambulation without or with an assistive device (e.g., walker, cane) or orthotics (e.g. AFO).
Main exclusion Criteria:
* Unstable cardiac or other medical condition or aphasia, dementia or other significant neurological disease limiting ability to train.
**Maximum Age:** 65 Years
**Minimum Age:** 16 Years
**Sex:** ALL
**Standard Ages:**
- CHILD
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Tel Hashomer
**Country:** Israel
**Facility:** Sheba Medical Center, Strok Center
**State:** Ramat Gan
**Zip:** 52621
#### Overall Officials
**Official 1:**
**Affiliation:** Sheba Medical Center
**Name:** David Tanne, MD
**Role:** PRINCIPAL_INVESTIGATOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000002561
- Term: Cerebrovascular Disorders
- ID: D000001927
- Term: Brain Diseases
- ID: D000002493
- Term: Central Nervous System Diseases
- ID: D000009422
- Term: Nervous System Diseases
- ID: D000014652
- Term: Vascular Diseases
- ID: D000002318
- Term: Cardiovascular Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC10
- Name: Nervous System Diseases
- Abbrev: BC14
- Name: Heart and Blood Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M22306
- Name: Stroke
- Relevance: HIGH
- As Found: Stroke
- ID: M5810
- Name: Cerebrovascular Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M5204
- Name: Brain Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5742
- Name: Central Nervous System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M17400
- Name: Vascular Diseases
- Relevance: LOW
- As Found: Unknown
- ID: T1303
- Name: Chronic Graft Versus Host Disease
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000020521
- Term: Stroke
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT00722579
**Acronym:** PRESILLION
**Brief Title:** A Study of the Presillion Stent in de Novo Coronary Lesions
**Official Title:** A Non-Randomized, Multi-Center, Single-Arm Safety Study of the Presillion Stent in de Novo Native Coronary Artery Lesions
#### Organization Study ID Info
**ID:** EC08-01
#### Organization
**Class:** INDUSTRY
**Full Name:** Cordis Corporation
### Status Module
#### Completion Date
**Date:** 2010-03
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2010-06-14
**Type:** ESTIMATED
**Last Update Submit Date:** 2010-06-11
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2008-12
**Type:** ACTUAL
#### Start Date
**Date:** 2008-07
**Status Verified Date:** 2010-06
#### Study First Post Date
**Date:** 2008-07-25
**Type:** ESTIMATED
**Study First Submit Date:** 2008-07-24
**Study First Submit QC Date:** 2008-07-24
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Cordis Corporation
#### Responsible Party
**Old Name Title:** Hans-Peter Stoll, MD
**Old Organization:** Cordis
### Oversight Module
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** The PRESILLION Study is a non-randomized, multi-center, single-arm study evaluating the safety of an approved Cobalt Chromium bare metal stent system for the treatment of ischemic heart disease attributable to a stenotic de novo lesion in a native coronary artery.
The study population will include 100 patients with up to two de novo native coronary artery lesions with a maximum lesion length of 30mm in a maximum of two major coronary arteries with reference vessel diameter \>= 2.5mm and \<= 4.0mm by visual estimation. Patients will be followed for 1 month and 6 month post-procedure for assessment of MACE and all other adverse events.
**Detailed Description:** The PRESILLION Stent System is intended for use in patients with symptomatic ischemic heart disease attributable to stenotic de novo lesions of native coronary arteries with reference vessel diameter from 2.5 mm to 4.0 mm with a lesion length up to 30 mm that are amenable to percutaneous treatment with coronary stenting. The stent is intended as a permanent implanted device.
The primary objective of this study is to evaluate the safety of the PRESILLION Stent System in the treatment of de novo stenotic lesions in native coronary arteries. The primary safety measure is the composite of MACE up to one (1) month follow up. The MACE rate shall meet the performance goal for bare metal stents in order to show the safety of the device.
The protocol has been amended and data will be collected for a time point as close as possible to (but after) the 6 months post index procedure in a non-interventional and retrospective manner. The data point will contain exactly the same follow-up information as was collected during the 1 month follow-up.
### Conditions Module
**Conditions:**
- Coronary Artery Disease
**Keywords:**
- Coronary Artery Disease
### Design Module
#### Design Info
**Allocation:** NON_RANDOMIZED
**Intervention Model:** SINGLE_GROUP
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 101
**Type:** ACTUAL
**Phases:**
- PHASE3
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** The PRESILLION TM Coronary Stent is an L-605 cobalt chromium (CoCr) stent.
**Intervention Names:**
- Device: PRESILLION cobalt chromium stent
**Label:** A
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- A
**Description:** PTCA with bare-metal stent
**Name:** PRESILLION cobalt chromium stent
**Other Names:**
- bare-metal stent
**Type:** DEVICE
### Outcomes Module
#### Primary Outcomes
**Measure:** Composite of MACE which includes cardiac death, myocardial infarction (Q-wave and non Q-wave) and clinically driven target lesion revascularization (TLR).
**Time Frame:** 1-month and 6-months post-procedure
#### Secondary Outcomes
**Measure:** Clinically driven Target Lesion Revascularization (TLR) defined as repeat PCI or CABG to the target lesion.
**Time Frame:** 1-month and 6-months post-procedure
**Measure:** Clinically driven Target Vessel Revascularization (TVR) defined as repeat PCI or CABG to the target vessel.
**Time Frame:** 1-month and 6-months post-procedure
**Measure:** Target Vessel Failure (TVF) defined as target vessel revascularization, recurrent myocardial infarction, or cardiac death that could not be clearly attributed to a vessel other than the target vessel.
**Time Frame:** 1-month and 6-months post-procedure
**Measure:** Myocardial Infarction (MI).
**Time Frame:** 1-month and 6-months post-procedure
**Measure:** Major bleeding.
**Time Frame:** 1-month and 6-months post-procedure
**Measure:** Device success.
**Time Frame:** Post-procedure
**Measure:** Lesion success.
**Time Frame:** Post-procedure
**Measure:** Procedure success.
**Time Frame:** Post-procedure
**Measure:** Incidence of acute and sub-acute stent thrombosis according the ARC definition.
**Time Frame:** 1-month and 6-months post-procedure
**Measure:** Stroke.
**Time Frame:** Post-procedure
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* The patient must be \>= 18 years of age.
* Patient is eligible for percutaneous coronary intervention (PCI).
* Acceptable candidate for coronary artery bypass surgery (CABG).
* Female patients of childbearing potential must have a negative pregnancy test within 7 days prior to enrolment and utilize reliable birth control for trial duration.
* Diagnosis of angina pectoris as defined by Canadian Cardiovascular Society Classification (CCS I, II, III, IV) or unstable angina pectoris (Braunwald Classification B\&C, I-II-III) or patients with documented silent ischemia.
* Treatment of up to two de novo native coronary artery lesions in a maximum of two major coronary arteries.
* Target reference vessel diameter of both lesions must be \>= 2.5mm and \<= 4.0mm in diameter (visual estimate).
* Target lesion length must be \<= 30mm and be covered by one study stent.
* Target lesion stenosis for both lesions is \> 50% and \< 100% (visual estimate).
* At least TIMI I coronary flow.
* Patient is willing to comply with the specified follow-up evaluation.
* Patient must provide written informed consent prior to the procedure using a form that is approved by the local Ethics Committee.
Exclusion Criteria:
* Recent myocardial infarction (either STEMI or non STEMI \< 48 hours prior to planned index procedure).
* The patient has unstable angina classified as Braunwald A I-II-III.
* The patient has unprotected left main coronary artery disease (stenosis \>50%).
* A significant (\> 50%) stenosis proximal or distal to the target lesion.
* Angiographic evidence of thrombus within the target lesion.
* Heavily calcified lesion and/or calcified lesion, which cannot be successfully predilated and/or an excessively tortuous vessel which makes it unsuitable for stent delivery and deployment.
* Left ventricular ejection fraction \<= 25%.
* Totally occluded lesion (TIMI 0 level).
* The patient has impaired renal function (creatinine 3.0mg/dL) at the time of treatment.
* The patient had a Cerebrovascular Accident (CVA) within the past 6 months.
* Prior stent within 10mm of target lesion.
* The target lesion is ostial in location (within 3.0mm of vessel origin).
* The target lesion involves a bifurcation with a diseased (\>50% stenotic) branch vessel \>= 2.0mm in diameter (or side branch requiring intervention of protection).
* The target lesion is located in a bypass graft. Note: stenting of lesions in bypassed native coronary arteries is allowed.
* Known allergies to the following: aspirin, clopidogrel bisulfate (Plavix ®) and ticlopidine (Ticlid ®), heparin, cobalt chromium, contrast agent (that cannot be managed medically).
* The patient has any significant medical condition which in the investigator's opinion may interfere with the patient's optimal participation in the study.
* The patient is currently participating in an investigational drug or device study that has not completed the primary endpoint or that clinically interferes with the study endpoints.
* Intervention of another lesion within 30 days prior to, or is planned or highly probably to be performed 30 days after the index procedure.
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Liège
**Country:** Belgium
**Facility:** CHU de Liège
**Zip:** B-4000
#### Overall Officials
**Official 1:**
**Affiliation:** CHU de Liège
**Name:** V. Legrand, MD, Phd
**Role:** PRINCIPAL_INVESTIGATOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000006331
- Term: Heart Diseases
- ID: D000002318
- Term: Cardiovascular Diseases
- ID: D000001161
- Term: Arteriosclerosis
- ID: D000001157
- Term: Arterial Occlusive Diseases
- ID: D000014652
- Term: Vascular Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC14
- Name: Heart and Blood Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
### Condition Browse Module - Browse Leaves
- ID: M6549
- Name: Coronary Disease
- Relevance: HIGH
- As Found: Coronary Artery Disease
- ID: M6546
- Name: Coronary Artery Disease
- Relevance: HIGH
- As Found: Coronary Artery Disease
- ID: M19506
- Name: Myocardial Ischemia
- Relevance: HIGH
- As Found: Coronary Artery Disease
- ID: M10543
- Name: Ischemia
- Relevance: LOW
- As Found: Unknown
- ID: M9419
- Name: Heart Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M4469
- Name: Arteriosclerosis
- Relevance: LOW
- As Found: Unknown
- ID: M4465
- Name: Arterial Occlusive Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M17400
- Name: Vascular Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000003324
- Term: Coronary Artery Disease
- ID: D000017202
- Term: Myocardial Ischemia
- ID: D000003327
- Term: Coronary Disease
### Intervention Browse Module - Ancestors
- ID: D000014131
- Term: Trace Elements
- ID: D000018977
- Term: Micronutrients
- ID: D000045505
- Term: Physiological Effects of Drugs
### Intervention Browse Module - Browse Branches
- Abbrev: Micro
- Name: Micronutrients
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M6097
- Name: Chromium
- Relevance: HIGH
- As Found: Transporter
- ID: M6265
- Name: Cobalt
- Relevance: HIGH
- As Found: Guardian
- ID: M21009
- Name: Micronutrients
- Relevance: LOW
- As Found: Unknown
- ID: M16885
- Name: Trace Elements
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: D000003035
- Term: Cobalt
- ID: D000002857
- Term: Chromium
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT01471379
**Brief Title:** Milnacipran (Savella) in Irritable Bowel Syndrome (IBS)
**Official Title:** A Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy of Milnacipran in the Treatment of Irritable Bowel Syndrome
#### Organization Study ID Info
**ID:** 11-1105a
#### Organization
**Class:** OTHER
**Full Name:** University of North Carolina, Chapel Hill
### Status Module
#### Completion Date
**Date:** 2013-02
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2017-04-13
**Type:** ACTUAL
**Last Update Submit Date:** 2017-03-15
**Overall Status:** TERMINATED
#### Primary Completion Date
**Date:** 2013-02
**Type:** ACTUAL
#### Results First Post Date
**Date:** 2013-12-24
**Type:** ESTIMATED
**Results First Submit Date:** 2013-07-03
**Results First Submit QC Date:** 2013-11-05
#### Start Date
**Date:** 2012-04
**Status Verified Date:** 2017-03
#### Study First Post Date
**Date:** 2011-11-16
**Type:** ESTIMATED
**Study First Submit Date:** 2011-11-10
**Study First Submit QC Date:** 2011-11-14
**Why Stopped:** Due to recruitment difficulties the study is terminated.
### Sponsor Collaborators Module
#### Collaborators
**Class:** INDUSTRY
**Name:** Forest Laboratories
#### Lead Sponsor
**Class:** OTHER
**Name:** Spencer Dorn, MD, MPH
#### Responsible Party
**Investigator Affiliation:** University of North Carolina, Chapel Hill
**Investigator Full Name:** Spencer Dorn, MD, MPH
**Investigator Title:** Assistant Professor of Medicine
**Type:** SPONSOR_INVESTIGATOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** Purpose: The investigators are proposing to examine the use of Savella® (Milnacipran) for treating irritable bowel syndrome (IBS) in women.
Participants: Eligible participants will meet the Rome III diagnostic criteria for IBS.
Procedures: This study will observe patients treated with Savella® as well as patients treated with a placebo (pill with no active drug). The investigators will monitor and compare several patient and symptom related outcomes, as well as evaluate health related quality of life, psychological distress and related psychosocial measures to determine if the addition of Savella® improves clinical pain response as well as secondary outcomes including quality of life.
**Detailed Description:** Irritable bowel syndrome (IBS) is a common functional gastrointestinal disorder characterized primarily by abdominal pain associated with bowel dysfunction. Like many other painful functional somatic syndromes (e.g. fibromyalgia) the pathophysiology of IBS includes abnormal responses to pain and dysregulation of brain-body pain pathways. IBS affects up to 10% of the population, is a leading reason for visits to gastroenterologists and primary care doctors, and, in the United States, annually accrues health care costs over $20 billion.
In their practice the investigators use centrally acting agents to treat IBS. Historically, the investigators have used tricyclic antidepressants based on results of clinical trials, including our NIH funded trial on desipramine. Nonetheless, these agents can produce side effects that limit their full application. More recently the investigators have begun to use SNRIs because they have been shown to benefit for various pain syndromes like diabetic neuropathy, fibromyalgia. The initial impression is that Milnacipran helps improve IBS symptoms and global well being. There is now a need to systematically determine Milnacipran's value for IBS.
### Conditions Module
**Conditions:**
- Irritable Bowel Syndrome
**Keywords:**
- Irritable Bowel Syndrome
- IBS
- Savella
- Milnacipran
- Abdominal Pain
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** DOUBLE
**Who Masked:**
- PARTICIPANT
- INVESTIGATOR
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 2
**Type:** ACTUAL
**Phases:**
- PHASE2
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Group A will begin treatment with Milnacipran 50mg BID (n=20) during Phase I and will be increased to 100mg BID during Phase II
**Intervention Names:**
- Drug: Milnacipran
**Label:** Group A (50mg - 100mg)
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Subjects in this arm will be maintained at Milnacipran 50mg BID for the entirety of the 12 weeks of the study.
**Intervention Names:**
- Drug: Milnacipran
**Label:** Group B (50mg x12)
**Type:** ACTIVE_COMPARATOR
#### Arm Group 3
**Description:** Group C will begin treatment with Placebo BID (n=20) during Phase I and will be given 50mg BID during Phase II
**Intervention Names:**
- Drug: Milnacipran
- Drug: Placebo
**Label:** Group C (Placebo - 50mg)
**Type:** PLACEBO_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Group A (50mg - 100mg)
- Group C (Placebo - 50mg)
**Description:** 50mg Milnacipran PO, BID, for 6 weeks.
**Name:** Milnacipran
**Other Names:**
- Savella
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- Group A (50mg - 100mg)
**Description:** Milnacipran, 100mg PO, BID, for six weeks
**Name:** Milnacipran
**Other Names:**
- Savella
**Type:** DRUG
#### Intervention 3
**Arm Group Labels:**
- Group B (50mg x12)
**Description:** Milnacipran, 50mg PO BID for 12 weeks
**Name:** Milnacipran
**Other Names:**
- Savella
**Type:** DRUG
#### Intervention 4
**Arm Group Labels:**
- Group C (Placebo - 50mg)
**Description:** Inactive pill, identical in shape, size, and appearance to active drug, PO, BID.
**Name:** Placebo
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Description:** Visual Analog Scale (VAS) scores (range 0-100 mm; 0 = none, 100 = worst pain) were recorded for pain before the beginning of the study, at 6 weeks of treatment and at the end visit i.e. 10 weeks. Ideally, VAS would have been administered at the 12th week; however, subject was terminated at the 10th week visit. A positive pain response (ie pain relief) was defined as \>30% decrease in the VAS score between baseline and the final study visit.
**Measure:** Number of Participants With Pain Response
**Time Frame:** Twelve Weeks
#### Secondary Outcomes
**Description:** After six weeks of treatment with Milnacipran, treatment groups were compared with placebo for clinically significant improvement in IBS-QOL. 11 point reduction in IBS-QOL compared to baseline was considered as clinically significant improvement.
**Measure:** Quality of Life ( IBS-QOL)
**Time Frame:** Six Weeks
**Description:** The study sought to determine if the Milnacipran arms had a greater proportion of adequate relief over the placebo group. Subjects were asked to answer 'yes' or 'no' as to whether or not they had adequate relief of pain due to irritable bowel syndrome.
**Measure:** Subject Self Reported Adequate Relief of Pain
**Time Frame:** Twelve Weeks
**Description:** Treatment Efficacy Questionnaire is a measure of treatment effectiveness. The score ranges from 1 to 48, 1 is minimum score and 48 is the maximum score. The investigators was looking to see if the Milnacipran treatment groups have a higher proportion of subjects with significant improvement in efficacy, judged as a TEQ score of \>28, compared to placebo group.
**Measure:** Treatment Efficacy Questionnaire (TEQ)
**Time Frame:** Twelve Weeks
**Description:** The investigator was looking to see if, for group A, when increased from 50 mg BID to 100 mg BID there is significant improvement of pain scores i.e. 30% pain reduction, and for group C, if there was significant improvement of pain scores when switched from placebo to 50 mg BID of Milnacipran
**Measure:** Dose Related Incremental Benefit in Pain Reduction Based on VAS
**Time Frame:** 12 Weeks
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Meet Rome III criteria for IBS and have no red flags.
* Must have had a colonoscopy within the previous 5 years to exclude inflammatory or other bowel disease
* Be fluent and literate in English
* Must either be of non-childbearing potential or agree to utilize approved birth control for the duration of the study
Exclusion Criteria:
* Diagnosis or treatment of any clinically symptomatic biochemical or structural abnormality of the GI tract within 6 months prior to screening, or active disease within 6 months prior to screening.
* Any other diagnosis to explain the abdominal pain,
* Clinical evidence of significant cardiovascular, respiratory, renal, hepatic, gastrointestinal, hematologic, neurologic, psychiatric or any disease that may interfere with the subject successfully completing the trial
* Hepatic dysfunction (ALT \[SGPT\] or AST \[SGOT\] \>3 times the upper limit of normal) or renal impairment (serum creatinine \> 2mg/dL)
* Has disease affecting electrolytes balance, such as SIADH with serum Sodium less than 130mmol/L
* Any evidence of or treatment of malignancy (other than localized basal cell, squamous cell skin cancer or cancer in situ that has been resected) within the previous year
* Any surgery on the stomach, small intestine or colon, excluding appendectomy
* A major psychiatric disorder (DSM-III-R or DSM-IV) including major depression or other psychoses that has required hospitalization in the last 1 year.
* History of attempted suicide or uncontrolled bipolar disorder.
* Currently using antidepressants for psychiatric conditions like major depression. Use of TCA or SSRI class antidepressant acceptable if being used specifically for treatment of bowel symptoms and patient is willing to taper off the medication
* Previous use of Milnacipran or other SNRI antidepressant (duloxetine, venlafaxine, desvenlafaxine)
* A diagnosis of seizure disorder
* A diagnosis of glaucoma
* Currently taking heparin or warfarin
**Maximum Age:** 79 Years
**Minimum Age:** 18 Years
**Sex:** FEMALE
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Chapel Hill
**Country:** United States
**Facility:** UNC Center for Functional GI and Motility Disorders
**State:** North Carolina
**Zip:** 27599
#### Overall Officials
**Official 1:**
**Affiliation:** University of North Carolina, Chapel Hill
**Name:** Spencer D Dorn, MD, MPH
**Role:** PRINCIPAL_INVESTIGATOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000004194
- Term: Disease
- ID: D000010335
- Term: Pathologic Processes
- ID: D000003109
- Term: Colonic Diseases, Functional
- ID: D000003108
- Term: Colonic Diseases
- ID: D000007410
- Term: Intestinal Diseases
- ID: D000005767
- Term: Gastrointestinal Diseases
- ID: D000004066
- Term: Digestive System Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: All
- Name: All Conditions
- Abbrev: BC06
- Name: Digestive System Diseases
### Condition Browse Module - Browse Leaves
- ID: M16355
- Name: Syndrome
- Relevance: HIGH
- As Found: Syndrome
- ID: M25118
- Name: Irritable Bowel Syndrome
- Relevance: HIGH
- As Found: Irritable Bowel Syndrome
- ID: M18311
- Name: Abdominal Pain
- Relevance: LOW
- As Found: Unknown
- ID: M13066
- Name: Pain
- Relevance: LOW
- As Found: Unknown
- ID: M6336
- Name: Colonic Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M6337
- Name: Colonic Diseases, Functional
- Relevance: LOW
- As Found: Unknown
- ID: M10444
- Name: Intestinal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M7255
- Name: Digestive System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M8883
- Name: Gastrointestinal Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000043183
- Term: Irritable Bowel Syndrome
- ID: D000013577
- Term: Syndrome
### Intervention Browse Module - Ancestors
- ID: D000068760
- Term: Serotonin and Noradrenaline Reuptake Inhibitors
- ID: D000014179
- Term: Neurotransmitter Uptake Inhibitors
- ID: D000049990
- Term: Membrane Transport Modulators
- ID: D000045504
- Term: Molecular Mechanisms of Pharmacological Action
- ID: D000018377
- Term: Neurotransmitter Agents
- ID: D000045505
- Term: Physiological Effects of Drugs
- ID: D000018712
- Term: Analgesics, Non-Narcotic
- ID: D000000700
- Term: Analgesics
- ID: D000018689
- Term: Sensory System Agents
- ID: D000018373
- Term: Peripheral Nervous System Agents
- ID: D000000928
- Term: Antidepressive Agents
- ID: D000011619
- Term: Psychotropic Drugs
### Intervention Browse Module - Browse Branches
- Abbrev: Analg
- Name: Analgesics
- Abbrev: All
- Name: All Drugs and Chemicals
- Abbrev: PsychDr
- Name: Psychotropic Drugs
- Abbrev: VaCoAg
- Name: Vasoconstrictor Agents
### Intervention Browse Module - Browse Leaves
- ID: M2003
- Name: Milnacipran
- Relevance: HIGH
- As Found: 131I-
- ID: M2015
- Name: Levomilnacipran
- Relevance: HIGH
- As Found: 131I-
- ID: M12575
- Name: Norepinephrine
- Relevance: LOW
- As Found: Unknown
- ID: M15512
- Name: Serotonin
- Relevance: LOW
- As Found: Unknown
- ID: M305
- Name: Serotonin and Noradrenaline Reuptake Inhibitors
- Relevance: LOW
- As Found: Unknown
- ID: M20504
- Name: Neurotransmitter Agents
- Relevance: LOW
- As Found: Unknown
- ID: M4032
- Name: Analgesics
- Relevance: LOW
- As Found: Unknown
- ID: M20786
- Name: Analgesics, Non-Narcotic
- Relevance: LOW
- As Found: Unknown
- ID: M4247
- Name: Antidepressive Agents
- Relevance: LOW
- As Found: Unknown
- ID: M14474
- Name: Psychotropic Drugs
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: D000078764
- Term: Milnacipran
- ID: D000078862
- Term: Levomilnacipran
### Misc Info Module
- Version Holder: 2024-05-24
## Results Section
### Adverse Events Module
#### Event Groups
**Group ID:** EG000
**Title:** Group A (50mg - 100mg)
**Description:** Group A will begin treatment with Milnacipran 50mg BID (n=20) during Phase I and will be increased to 100mg BID during Phase II
Milnacipran : 50mg Milnacipran PO, BID, for 6 weeks.
Milnacipran : Milnacipran, 100mg PO, BID, for six weeks
**ID:** EG000
**Other Num Affected:** 1
**Other Num at Risk:** 1
**Serious Number At Risk:** 1
**Title:** Group A (50mg - 100mg)
**Group ID:** EG001
**Title:** Group B (50mg x12)
**Description:** Subjects in this arm will be maintained at Milnacipran 50mg BID for the entirety of the 12 weeks of the study.
Milnacipran : Milnacipran, 50mg PO BID for 12 weeks
**ID:** EG001
**Serious Number At Risk:** 1
**Title:** Group B (50mg x12)
**Group ID:** EG002
**Title:** Group C (Placebo - 50mg)
**Description:** Group C will begin treatment with Placebo BID (n=20) during Phase I and will be given 50mg BID during Phase II
Milnacipran : 50mg Milnacipran PO, BID, for 6 weeks.
Placebo : Inactive pill, identical in shape, size, and appearance to active drug, PO, BID.
**ID:** EG002
**Title:** Group C (Placebo - 50mg)
**Frequency Threshold:** 1
#### Other Events
**Term:** hypertension
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Cardiac disorders
**Source Vocabulary:** MedDRA 10.0
**Time Frame:** 3 months
## Results Section - Baseline Characteristics Module
### Denomination Counts
**Group ID:** BG000
**Value:** 1
**Group ID:** BG001
**Value:** 1
**Group ID:** BG002
**Value:** 0
**Group ID:** BG003
**Value:** 2
**Units:** Participants
### Group
**ID:** BG000
**Title:** Group A (50mg - 100mg)
**Description:** Group A will begin treatment with Milnacipran 50mg BID (n=20) during Phase I and will be increased to 100mg BID during Phase II
Milnacipran : 50mg Milnacipran PO, BID, for 6 weeks.
Milnacipran : Milnacipran, 100mg PO, BID, for six weeks
### Group
**ID:** BG001
**Title:** Group B (50mg x12)
**Description:** Subjects in this arm will be maintained at Milnacipran 50mg BID for the entirety of the 12 weeks of the study.
Milnacipran : Milnacipran, 50mg PO BID for 12 weeks
### Group
**ID:** BG002
**Title:** Group C (Placebo - 50mg)
**Description:** Group C will begin treatment with Placebo BID (n=20) during Phase I and will be given 50mg BID during Phase II
Milnacipran : 50mg Milnacipran PO, BID, for 6 weeks.
Placebo : Inactive pill, identical in shape, size, and appearance to active drug, PO, BID.
### Group
**ID:** BG003
**Title:** Total
**Description:** Total of all reporting groups
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG003
**Value:** 0
**Category Title:** <=18 years
#### Measurement
**Group ID:** BG000
**Value:** 1
#### Measurement
**Group ID:** BG001
**Value:** 1
#### Measurement
**Group ID:** BG003
**Value:** 2
**Category Title:** Between 18 and 65 years
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG003
**Value:** 0
**Category Title:** >=65 years
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Spread:** 0
**Value:** 47
#### Measurement
**Group ID:** BG001
**Spread:** 0
**Value:** 66
#### Measurement
**Group ID:** BG003
**Spread:** 9.5
**Value:** 56.5
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 1
#### Measurement
**Group ID:** BG001
**Value:** 1
#### Measurement
**Group ID:** BG003
**Value:** 2
**Category Title:** Female
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG003
**Value:** 0
**Category Title:** Male
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 1
#### Measurement
**Group ID:** BG001
**Value:** 1
#### Measurement
**Group ID:** BG003
**Value:** 2
**Class Title:** United States
**Denomination Units Selected:**
## Results Section - Baseline Characteristics Module
### Measure 1
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Age, Categorical
**Unit of Measure:** Participants
### Measure 2
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Age, Continuous
**Unit of Measure:** years
### Measure 3
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Sex: Female, Male
**Unit of Measure:** Participants
### Measure 4
**Parameter Type:** NUMBER
**Title:** Region of Enrollment
**Unit of Measure:** participants
**Population Description:** The study was terminated early resulting in a small number of subjects enrolled leaving the ability to analyze only certain categories.
## Results Section - More Information Module
### Certain Agreement
### Point of Contact
**Email:** [email protected]
**Organization:** UNC Chapel Hill
**Phone:** 919-966-0141
**Title:** Dr. Spencer Dorn Associate Professor
## Results Section - Outcome Measures Module
### Outcome Measure 1
### Outcome Measure 2
### Outcome Measure 3
### Outcome Measure 4
### Outcome Measure 5
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
#### Outcome Measure 2
#### Outcome Measure 3
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
#### Outcome Measure 4
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
#### Outcome Measure 5
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Description:** Visual Analog Scale (VAS) scores (range 0-100 mm; 0 = none, 100 = worst pain) were recorded for pain before the beginning of the study, at 6 weeks of treatment and at the end visit i.e. 10 weeks. Ideally, VAS would have been administered at the 12th week; however, subject was terminated at the 10th week visit. A positive pain response (ie pain relief) was defined as \>30% decrease in the VAS score between baseline and the final study visit.
**Parameter Type:** NUMBER
**Reporting Status:** POSTED
**Time Frame:** Twelve Weeks
**Title:** Number of Participants With Pain Response
**Type:** PRIMARY
**Unit of Measure:** participants
##### Group
**Description:** Group A begins treatment with Milnacipran 50mg BID (n=20) during Phase I and will be increased to 100mg BID during Phase II
Milnacipran : 50mg Milnacipran PO, BID, for 6 weeks.
Milnacipran : Milnacipran, 100mg PO, BID, for six weeks
**ID:** OG000
**Title:** Group A (50mg - 100mg)
##### Group
**Description:** Subjects in this arm will be maintained at Milnacipran 50mg BID for the entirety of the 12 weeks of the study.
Milnacipran : Milnacipran, 50mg PO BID for 12 weeks
**ID:** OG001
**Title:** Group B (50mg x12)
##### Group
**Description:** Group C will begin treatment with Placebo BID (n=20) during Phase I and will be given 50mg BID during Phase II
Milnacipran : 50mg Milnacipran PO, BID, for 6 weeks.
Placebo : Inactive pill, identical in shape, size, and appearance to active drug, PO, BID.
**ID:** OG002
**Title:** Group C (Placebo - 50mg)
#### Outcome Measure 2
**Description:** After six weeks of treatment with Milnacipran, treatment groups were compared with placebo for clinically significant improvement in IBS-QOL. 11 point reduction in IBS-QOL compared to baseline was considered as clinically significant improvement.
**Reporting Status:** POSTED
**Time Frame:** Six Weeks
**Title:** Quality of Life ( IBS-QOL)
**Type:** SECONDARY
##### Group
**Description:** Group A will begin treatment with Milnacipran 50mg BID (n=20) during Phase I and will be increased to 100mg BID during Phase II
Milnacipran : 50mg Milnacipran PO, BID, for 6 weeks.
Milnacipran : Milnacipran, 100mg PO, BID, for six weeks
**ID:** OG000
**Title:** Group A (50mg - 100mg)
##### Group
**Description:** Subjects in this arm will be maintained at Milnacipran 50mg BID for the entirety of the 12 weeks of the study.
Milnacipran : Milnacipran, 50mg PO BID for 12 weeks
**ID:** OG001
**Title:** Group B (50mg x12)
##### Group
**Description:** Group C will begin treatment with Placebo BID (n=20) during Phase I and will be given 50mg BID during Phase II
Milnacipran : 50mg Milnacipran PO, BID, for 6 weeks.
Placebo : Inactive pill, identical in shape, size, and appearance to active drug, PO, BID.
**ID:** OG002
**Title:** Group C (Placebo - 50mg)
#### Outcome Measure 3
**Description:** The study sought to determine if the Milnacipran arms had a greater proportion of adequate relief over the placebo group. Subjects were asked to answer 'yes' or 'no' as to whether or not they had adequate relief of pain due to irritable bowel syndrome.
**Parameter Type:** NUMBER
**Reporting Status:** POSTED
**Time Frame:** Twelve Weeks
**Title:** Subject Self Reported Adequate Relief of Pain
**Type:** SECONDARY
**Unit of Measure:** percentage of participants
##### Group
**Description:** Group A will begin treatment with Milnacipran 50mg BID (n=20) during Phase I and will be increased to 100mg BID during Phase II
Milnacipran : 50mg Milnacipran PO, BID, for 6 weeks.
Milnacipran : Milnacipran, 100mg PO, BID, for six weeks
**ID:** OG000
**Title:** Group A (50mg - 100mg)
##### Group
**Description:** Subjects in this arm will be maintained at Milnacipran 50mg BID for the entirety of the 12 weeks of the study.
Milnacipran : Milnacipran, 50mg PO BID for 12 weeks
**ID:** OG001
**Title:** Group B (50mg x12)
##### Group
**Description:** Group C will begin treatment with Placebo BID (n=20) during Phase I and will be given 50mg BID during Phase II
Milnacipran : 50mg Milnacipran PO, BID, for 6 weeks.
Placebo : Inactive pill, identical in shape, size, and appearance to active drug, PO, BID.
**ID:** OG002
**Title:** Group C (Placebo - 50mg)
#### Outcome Measure 4
**Description:** Treatment Efficacy Questionnaire is a measure of treatment effectiveness. The score ranges from 1 to 48, 1 is minimum score and 48 is the maximum score. The investigators was looking to see if the Milnacipran treatment groups have a higher proportion of subjects with significant improvement in efficacy, judged as a TEQ score of \>28, compared to placebo group.
**Parameter Type:** NUMBER
**Population Description:** Only one subject was enrolled and was analyzed even though subject did not complete the study.
**Reporting Status:** POSTED
**Time Frame:** Twelve Weeks
**Title:** Treatment Efficacy Questionnaire (TEQ)
**Type:** SECONDARY
**Unit of Measure:** percentage of subject with score >28
##### Group
**Description:** Group A will begin treatment with Milnacipran 50mg BID (n=20) during Phase I and will be increased to 100mg BID during Phase II
Milnacipran : 50mg Milnacipran PO, BID, for 6 weeks.
Milnacipran : Milnacipran, 100mg PO, BID, for six weeks
**ID:** OG000
**Title:** Group A (50mg - 100mg)
##### Group
**Description:** Subjects in this arm will be maintained at Milnacipran 50mg BID for the entirety of the 12 weeks of the study.
Milnacipran : Milnacipran, 50mg PO BID for 12 weeks
**ID:** OG001
**Title:** Group B (50mg x12)
##### Group
**Description:** Group C will begin treatment with Placebo BID (n=20) during Phase I and will be given 50mg BID during Phase II
Milnacipran : 50mg Milnacipran PO, BID, for 6 weeks.
Placebo : Inactive pill, identical in shape, size, and appearance to active drug, PO, BID.
**ID:** OG002
**Title:** Group C (Placebo - 50mg)
#### Outcome Measure 5
**Description:** The investigator was looking to see if, for group A, when increased from 50 mg BID to 100 mg BID there is significant improvement of pain scores i.e. 30% pain reduction, and for group C, if there was significant improvement of pain scores when switched from placebo to 50 mg BID of Milnacipran
**Parameter Type:** NUMBER
**Reporting Status:** POSTED
**Time Frame:** 12 Weeks
**Title:** Dose Related Incremental Benefit in Pain Reduction Based on VAS
**Type:** SECONDARY
**Unit of Measure:** percentage of participants
##### Group
**Description:** Group A will begin treatment with Milnacipran 50mg BID (n=20) during Phase I and will be increased to 100mg BID during Phase II
Milnacipran : 50mg Milnacipran PO, BID, for 6 weeks.
Milnacipran : Milnacipran, 100mg PO, BID, for six weeks
**ID:** OG000
**Title:** Group A (50mg - 100mg)
##### Group
**Description:** Subjects in this arm will be maintained at Milnacipran 50mg BID for the entirety of the 12 weeks of the study.
Milnacipran : Milnacipran, 50mg PO BID for 12 weeks
**ID:** OG001
**Title:** Group B (50mg x12)
##### Group
**Description:** Group C will begin treatment with Placebo BID (n=20) during Phase I and will be given 50mg BID during Phase II
Milnacipran : 50mg Milnacipran PO, BID, for 6 weeks.
Placebo : Inactive pill, identical in shape, size, and appearance to active drug, PO, BID.
**ID:** OG002
**Title:** Group C (Placebo - 50mg)
### Participant Flow Module
#### Group
**Description:** Group A will begin treatment with Milnacipran 50mg twice a day (BID) (n=20) during Phase I and will be increased to 100mg BID during Phase II
Milnacipran : 50mg Milnacipran per orally (PO), BID, for 6 weeks.
Milnacipran : Milnacipran, 100mg PO, BID, for six weeks
**ID:** FG000
**Title:** Group A (50mg - 100mg)
#### Group
**Description:** Subjects in this arm will be maintained at Milnacipran 50mg BID for the entirety of the 12 weeks of the study.
Milnacipran : Milnacipran, 50mg PO BID for 12 weeks
**ID:** FG001
**Title:** Group B (50mg x12)
#### Group
**Description:** Group C will begin treatment with Placebo BID (n=20) during Phase I and will be given 50mg BID during Phase II
Milnacipran : 50mg Milnacipran PO, BID, for 6 weeks.
Placebo : Inactive pill, identical in shape, size, and appearance to active drug, PO, BID.
**ID:** FG002
**Title:** Group C (Placebo - 50mg)
#### Period
**Title:** Overall Study
##### Withdraw
**Type:** Adverse Event
###### Reason
**Group ID:** FG000
**Number of Subjects:** 1
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
###### Reason
**Group ID:** FG002
**Number of Subjects:** 0
##### Withdraw
**Type:** Study terminated
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 1
###### Reason
**Group ID:** FG002
**Number of Subjects:** 0
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 1
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 1
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 0
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 0
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 1
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 1
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 0
**Pre-Assignment Details:** Subjects undergo screening labs and questionnaires to make sure subjects are healthy and don't have any underlying conditions. Subjects who had clinically significant labs or Hospital Anxiety and depression scale(HADS)score more than 17 were excluded.
**Recruitment Details:** The subjects were recruited from community using University of North Carolina (UNC) mass email system, newspaper advertisement and UNC gastrointestinal (GI) clinic referral.
**Has Results:** True |
## Protocol Section
### Identification Module
**NCT ID:** NCT04653779
**Brief Title:** A Clinical Trial to Evaluate the Preference Regarding Convenience of Medication and Efficacy/Safety of SUGAMET®XR Tablet 5/1000mg
**Official Title:** A Multi-center, Open-label, Single-arm, Phase IV Clinical Trial to Evaluate the Preference Regarding Convenience of Medication, Efficacy and Safety After Switching to SUGAMET®XR Tablet 5/1000mg in Patients With Type 2 Diabetes and Renal Diseases
#### Organization Study ID Info
**ID:** DA1229_01_DM_IV
#### Organization
**Class:** INDUSTRY
**Full Name:** Dong-A ST Co., Ltd.
### Status Module
#### Completion Date
**Date:** 2021-07
**Type:** ESTIMATED
#### Expanded Access Info
**Last Known Status:** NOT_YET_RECRUITING
#### Last Update Post Date
**Date:** 2020-12-11
**Type:** ACTUAL
**Last Update Submit Date:** 2020-12-10
**Overall Status:** UNKNOWN
#### Primary Completion Date
**Date:** 2021-05
**Type:** ESTIMATED
#### Start Date
**Date:** 2020-12
**Type:** ESTIMATED
**Status Verified Date:** 2020-12
#### Study First Post Date
**Date:** 2020-12-04
**Type:** ACTUAL
**Study First Submit Date:** 2020-11-19
**Study First Submit QC Date:** 2020-11-27
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Dong-A ST Co., Ltd.
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
### Description Module
**Brief Summary:** Multi-center, open-label, single arm study to Evaluate the preference regarding convenience of medication, efficacy and safety of Sugamet XR tab. 5/1000mg in patients with Type 2 diabetes and renal diseases
### Conditions Module
**Conditions:**
- Diabetes Mellitus, Type 2
- Renal Disease
- Diabetes Mellitus
- Metabolic Disease
- Endocrine System Diseases
### Design Module
#### Design Info
**Allocation:** NA
**Intervention Model:** SINGLE_GROUP
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 54
**Type:** ESTIMATED
**Phases:**
- PHASE4
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Patients receive Evogliptin 5mg/Metformin 1000mg once a day
**Intervention Names:**
- Drug: Evogliptin 5mg/Metformin 1000mg
**Label:** Group A
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Group A
**Description:** Size reduction of a tablet formulation
**Name:** Evogliptin 5mg/Metformin 1000mg
**Other Names:**
- SUGAMET®XR tablet 5/1000mg
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Description:** Looking back on the experience of participating in the study, which would you prefer, any of the existing DPP-4 inhibitor/metformin 1000mg sustained-release combination drug or the SugarMet® sustained-release tablet 5/1000mg?
**Measure:** The preference regarding convenience of medication
**Time Frame:** 12 weeks
#### Secondary Outcomes
**Measure:** Change from baseline in HbA1c (%) After 12 weeks
**Time Frame:** 12 weeks
**Measure:** Change from baseline in HbA1C response rate(%) After 12 weeks
**Time Frame:** 12 weeks
**Measure:** Change from baseline in Glycated albumin After 12 weeks
**Time Frame:** 12 weeks
**Measure:** Change from baseline in e-GFR After 12 weeks
**Time Frame:** 12 weeks
**Description:** Urine Albumin-to-Creatinin Ratio
**Measure:** Change from baseline in UACR After 12 weeks
**Time Frame:** 12 weeks
**Description:** N-Acetyl-Glucosaminidase
**Measure:** Change from baseline in HbA1c (%) After 12 weeks NAG
**Time Frame:** 12 weeks
**Measure:** Change from baseline in Nephrin After 12 weeks
**Time Frame:** 12 weeks
**Measure:** Change from baseline in Cystatin-C After 12 weeks
**Time Frame:** 12 weeks
**Description:** Treatment Satisfaction Questionnaire for Medication-9 scores, higher scores mean a better outcome.
**Measure:** TSQM-9 scores
**Time Frame:** 12 weeks
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Aged ≥60 years
* Subjects with type 2 diabetes mellitus
* Subjects treated with DPP-4 inhibitor and 1,000mg/day dose of metformin 1T/qd for at least 8 weeks prior to screening
* Subjects with HbA1c≤7.5% at screening
* Subjects with 45mL/min/1.73m2≤eGFR≤90mL/min/1.73m2 at screening
* Subjects with fasting glucose≤200 at screening
* Subjects with 18.5kg/m2≤BMI≤40kg/m2 at screening
Exclusion Criteria:
* Patients with type 1 diabetes mellitus, secondary diabetes mellitus or gestational diabetes mellitus
* ESRD or Patients who have kidney dialysis
* Subjects with ALT and AST 3 times or higher than upper normal range
**Minimum Age:** 60 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Sung
**Phone:** 82-2-920-8369
**Role:** CONTACT
#### Locations
**Location 1:**
**City:** Bucheon
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Suncheonhyang Bucheon Hospital
- **Phone:** 82-32-621-6363
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bo Yeon Kim, MD
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** Korea, Republic of
**Facility:** Suncheonhyang Bucheon Hospital
**State:** Gyeonggi
**Zip:** 14584
**Location 2:**
**City:** Bucheon
**Contacts:**
***Contact 1:***
- **Name:** Se-jong hospital
- **Phone:** 82-32-340-1236
- **Role:** CONTACT
***Contact 2:***
- **Name:** Chong Hwa Kim, MD
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** Korea, Republic of
**Facility:** Se-jong hospital
**State:** Gyeonggi
**Zip:** 14754
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000044882
- Term: Glucose Metabolism Disorders
- ID: D000014570
- Term: Urologic Diseases
- ID: D000052776
- Term: Female Urogenital Diseases
- ID: D000005261
- Term: Female Urogenital Diseases and Pregnancy Complications
- ID: D000091642
- Term: Urogenital Diseases
- ID: D000052801
- Term: Male Urogenital Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC18
- Name: Nutritional and Metabolic Diseases
- Abbrev: BC19
- Name: Gland and Hormone Related Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BXS
- Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions
### Condition Browse Module - Browse Leaves
- ID: M7115
- Name: Diabetes Mellitus
- Relevance: HIGH
- As Found: Diabetes Mellitus
- ID: M7119
- Name: Diabetes Mellitus, Type 2
- Relevance: HIGH
- As Found: Diabetes Mellitus, Type 2
- ID: M10698
- Name: Kidney Diseases
- Relevance: HIGH
- As Found: Renal Disease
- ID: M11639
- Name: Metabolic Diseases
- Relevance: HIGH
- As Found: Metabolic Diseases
- ID: M7862
- Name: Endocrine System Diseases
- Relevance: HIGH
- As Found: Endocrine System Diseases
- ID: M25403
- Name: Glucose Metabolism Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M17319
- Name: Urologic Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M2875
- Name: Urogenital Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M27093
- Name: Female Urogenital Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M14127
- Name: Pregnancy Complications
- Relevance: LOW
- As Found: Unknown
- ID: M8399
- Name: Female Urogenital Diseases and Pregnancy Complications
- Relevance: LOW
- As Found: Unknown
- ID: M27095
- Name: Male Urogenital Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000007674
- Term: Kidney Diseases
- ID: D000003920
- Term: Diabetes Mellitus
- ID: D000003924
- Term: Diabetes Mellitus, Type 2
- ID: D000008659
- Term: Metabolic Diseases
- ID: D000004700
- Term: Endocrine System Diseases
### Intervention Browse Module - Ancestors
- ID: D000007004
- Term: Hypoglycemic Agents
- ID: D000045505
- Term: Physiological Effects of Drugs
### Intervention Browse Module - Browse Branches
- Abbrev: Hypo
- Name: Hypoglycemic Agents
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M11667
- Name: Metformin
- Relevance: HIGH
- As Found: Assessment
- ID: M10054
- Name: Hypoglycemic Agents
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: D000008687
- Term: Metformin
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT02633579
**Brief Title:** Effect of the Motilin Receptor Agonist, Erythromycin, on Hunger and Food Intake; Study of Role of Cholinergic Pathways
**Official Title:** The Role of Induced Phase 3 Contractions in the Control of Hunger and Food Intake
#### Organization Study ID Info
**ID:** ErythromycinHungerFoodIntake
#### Organization
**Class:** OTHER
**Full Name:** Universitaire Ziekenhuizen KU Leuven
### Status Module
#### Completion Date
**Date:** 2014-08
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2015-12-17
**Type:** ESTIMATED
**Last Update Submit Date:** 2015-12-14
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2014-08
**Type:** ACTUAL
#### Start Date
**Date:** 2012-10
**Status Verified Date:** 2015-10
#### Study First Post Date
**Date:** 2015-12-17
**Type:** ESTIMATED
**Study First Submit Date:** 2015-11-02
**Study First Submit QC Date:** 2015-12-14
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Universitaire Ziekenhuizen KU Leuven
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** In this study, the investigators will evaluate if the food intake associated with the infusion of erythromycin is caused by the phase 3 contractions or by another yet unknown effect of erythromycin. To obtain this the investigators will use atropine, a muscarinic receptor antagonist, to inhibit the formation of contractions induced by a low dose of erythromycin
**Detailed Description:** The role of induced phase 3 contractions in the control of hunger and food intake
1. Background In between meals the motor activity of the upper gastrointestinal tract is characterized by a cyclical pattern of contractions that consists of 3 phases: maximal contractile activity originating in the stomach and migrating down the small intestine (phase 3), followed by a phase of motor quiescence (phase 1) and a phase of progressively increasing motor activity (phase 2). In 1975 Itoh et. al. linked the phase 3 contractions of the migrating motor complex (MMC) to hunger sensations, by increasing the hunger sensations through exogenously administered motilin. The investigators previously demonstrated that phase 3 is associated with a 'hunger pang'. The trigger for the phase 3 initiation is unclear, but the gut hormones ghrelin and motilin are presumed to play a role. If either of these substances are administered intravenously, they induce a premature phase 3 in humans.
In previous studies, 'hunger pangs' could objectively be detected as peaks in subjective hunger ratings on a visual analog scale (VAS) using a custom made algorithm. A close association between phase 3 contractions of gastric origin and hunger peaks was observed and spontaneous food intake was also found to be associated with hunger peaks. By inducing phase 3 contractions through the administration of IV erythromycin, the time of food intake could be manipulated.
In a new study the investigators will evaluate if the food intake associated with the infusion of erythromycin is caused by the phase 3 contractions or by another yet unknown effect of erythromycin. To obtain this the investigators will use atropine, a muscarinic receptor antagonist, to inhibit the formation of contractions induced by a low dose of erythromycin. Since food intake is no longer a pure physiological act to still hunger sensations, it has obtained a social and emotional status in modern times where palatable food is abundant, the relationship of the subject towards food using multiple questionnaires will also be evaluated.
2. Aim To investigate whether contractions in the antrum are necessary to induce food intake with erythromycin or if erythromycin has a secondary effect to stimulate food intake.
3. Methodology 3.1. Subject selection Healthy volunteers will be recruited. See eligibility criteria for details
3.2. Questionnaires
Dutch eating behaviour questionnaire (DEBQ):
The DEBQ will be administered as a measure of dietary restraint and disinhibition. The DEBQ investigates three fields of behavioral eating: restrained eating, emotional eating and external eating.
Council of nutrition appetite questionnaire (CNAQ):
This questionnaire is an appetite-monitoring instrument developed by the Council for Nutritional Strategies in Long-Term Care.
Hospital Anxiety \& Depression Scale (HAD):
This questionnaire will be used to exclude subjects with mood \& anxiety disorders.
Power of food scale (PFS):
The PFS evaluates the psychological impact of living in an environment where palatable food is abundant.
3.3. Protocol Study design Gastric motility will be registered for 7h after an overnight fast (see below). During this period the volunteers are allowed to ingest a standardized liquid meal twice at time points of their choice. The volunteers are exposed to the liquid meal before the start of the experiment and the palatability of the meal is scored; this is done in a standardized way (same cup, temperature, duration, only smelling not tasting). The liquid meal is a low-caloric soup with a similar composition as the one used by Hjelland et. al. The volunteers are not aware that the investigators want to study the association between gastric motility, hunger and food intake; they receive the information that the investigators want to examine the effect of fasting on the motility of the stomach and hunger. During the experiment they will watch standardized movies with a neutral emotional content at standardized time points. They will rate hunger every 5 minutes and they will be offered the opportunity to drink the meal at a time point of their choice. At the start of the experiment two intravenous catheters will be placed in separate arms of the subject. During the entire study saline will be administered intravenously at a low rate to blind the subject towards the time of drug administration. The infusion bag will be positioned behind a curtain, in this way the time of drug administration will be blind for the subject. The administration of the drug, 40 mg of the motilin receptor agonist erythromycin lactobionate (Erythrocine; Abbott, Ottignies-Louvain-la-Neuve, Belgium), will be given in a randomized fashion at time point 90, 180, 270 or 360 min after the start of the study. An infusion of this macrolide antibiotic will be given at two of the above time points, one of these erythromycin infusions will be preceded by a 15 µg/kg IV bolus of atropine (Stellatropine; Pharmacobel, Brussels, Belgium) plus a 30 min infusion of 15 µg/kg/hr of atropine. The administration of atropine will be given 10 minutes before the infusion of erythromycin in the opposite arm of the erythromycin infusion. The arterial pulse frequency will be monitored continuously during the administration of atropine. If the start point of an infusion coincides with a spontaneous phase 3, then the infusion will be postponed for 15 min until the phase 3 is passed.
Manometry Recording of antroduodenal intraluminal pressures will be performed using a Manoscan® high resolution manometry catheter (outer diameter 4.2 mm, 36 channels spaced 1 cm apart). The catheter will be introduced via the nose and the position of the catheter, see figure 1, will be briefly checked by fluoroscopy (typically 5 seconds, never more than 25 seconds). This part will comply with the relevant guidelines of radioprotection and participants will be protected by a leaden shield that covers the lower abdomen, Personnel will carry a leaden jacket, will not be exposed to the primary beam and will wear dosimeters at all time. The catheter will be placed in such a way, that there are measuring points in the antrum and just distal to the pylorus to accurately record the migrating motor complex. The output of the manometry channels is recorded and monitored on-line via the ManoScan 360™ (Sierra Scientific Instruments, Los Angeles, CA). After an overnight fast, the manometry assembly will be introduced as described above and secured to the subject's nose with adhesive tape. The subjects will then be positioned in a comfortable sitting position with the knees bent (80°) and the trunk upright in a specifically designed bed.
Behavioural ratings At 5-minute intervals, volunteers will indicate scores for hunger and expected amount to eat on a 10cm VAS. Emotions will be scored at a 15-minute interval on an electronic grid mood scale.
### Conditions Module
**Conditions:**
- Healthy
**Keywords:**
- Healthy volunteers
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** CROSSOVER
##### Masking Info
**Masking:** SINGLE
**Who Masked:**
- PARTICIPANT
#### Enrollment Info
**Count:** 28
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** 40 mg erythromycin lactobionate was administered over a 20 min period in a volume of 100 ml sodium chloride 0.9 %
**Intervention Names:**
- Drug: erythromycin lactobionate
**Label:** Erythromycin lactobionate
**Type:** ACTIVE_COMPARATOR
#### Arm Group 2
**Description:** 40 mg erythromycin lactobionate was administered over a 20 min period in a volume of 100 ml sodium chloride 0.9 %; Atropine sulfate was given as an i.v. bolus (15 µg/kg) followed by a continuous infusion of 15 µg/kg/h over 30 min
**Intervention Names:**
- Drug: Erythromycin with atropine
**Label:** Erythromycin lactobionate with atropine
**Type:** ACTIVE_COMPARATOR
#### Arm Group 3
**Description:** Atropine sulfate was given as an i.v. bolus (15 µg/kg) followed by a continuous infusion of 15 µg/kg/h over 30 min.
Infusion of saline as a placebo for erythromycin was administered over a 20 min period in a volume of 100 ml sodium chloride 0.9 %
**Intervention Names:**
- Drug: Atropine
**Label:** Atropine
**Type:** PLACEBO_COMPARATOR
#### Arm Group 4
**Description:** Infusion of saline was administered over a 20 min period in a volume of 100 ml sodium chloride 0.9 %; also placebo for atropine was given as an i.v. bolus of saline followed by a continuous infusion over 30 min
**Intervention Names:**
- Drug: Saline
**Label:** Placebo
**Type:** PLACEBO_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Erythromycin lactobionate
**Description:** intravenous administration of erythromycin
**Name:** erythromycin lactobionate
**Other Names:**
- Erythromycin
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- Atropine
**Description:** intravenous administration of atropine
**Name:** Atropine
**Type:** DRUG
#### Intervention 3
**Arm Group Labels:**
- Erythromycin lactobionate with atropine
**Description:** intravenous administration of erythromycin with saline
**Name:** Erythromycin with atropine
**Type:** DRUG
#### Intervention 4
**Arm Group Labels:**
- Placebo
**Description:** intravenous administration of saline
**Name:** Saline
**Other Names:**
- Control
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Description:** visual analog scale
**Measure:** Change in hunger ratings from time of administration over 6 hours
**Time Frame:** 6 hours after intervention, assessment every 5 minutes
#### Secondary Outcomes
**Description:** decision to take a soup meal
**Measure:** Timing of food intake
**Time Frame:** 6 hours after intervention; up to 2 moments of food intake allowed
### Eligibility Module
**Eligibility Criteria:** Inclusion criteria:
* Female or male subjects aged 18 to 60
* Subject is capable and willing to give informed consent
* Female volunteers of child bearing potential must use oral, injected or implanted hormonal methods of contraception
Exclusion criteria:
* Female volunteer is pregnant or breastfeeding
* Gastrointestinal diseases, major abdominal surgery
* Major psychiatric illnesses
* Volunteers that use drugs affecting the gastrointestinal tract or the central nervous system
* Allergy for macrolide antibiotics
* Allergy for atropine
* Severe heartburn
* Enlarged prostate
* Blockage of urinary tract
* Acute closed-angle glaucoma
* Myasthenia gravies
* Severe heart disease
* Thyrotoxicosis
* Fever
* Liver problems
* High blood pressure or fast heart rate
* Lung disease
**Healthy Volunteers:** True
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### References Module
#### References
**Citation:** Szurszewski JH. A migrating electric complex of canine small intestine. Am J Physiol. 1969 Dec;217(6):1757-63. doi: 10.1152/ajplegacy.1969.217.6.1757. No abstract available.
**PMID:** 5353053
**Citation:** Itoh Z. AI, Takeuchi S., Couch E. F., editor. Hunger contractions and motilin. 5th International Symposium on Gastrointestinal Motility; 1975; Herentals, Belgium.
**Citation:** Tack J, Deloose E, Ang D, Scarpellini E, Vanuytsel T, Van Oudenhove L, Depoortere I. Motilin-induced gastric contractions signal hunger in man. Gut. 2016 Feb;65(2):214-24. doi: 10.1136/gutjnl-2014-308472. Epub 2014 Dec 24.
**PMID:** 25539673
**Citation:** Scarpellini E. HN, D. Ang, P. Vanden Berghe, I. Depoortere, J. Tack. Role of upper gastrointestinal motility stimulation in the occurence of hunger peaks. Gastroenterology. 2009;136:A330
**Citation:** Janssens J, Vantrappen G, Peeters TL. The activity front of the migrating motor complex of the human stomach but not of the small intestine is motilin-dependent. Regul Pept. 1983 Aug;6(4):363-9. doi: 10.1016/0167-0115(83)90265-3.
**PMID:** 6635258
**Citation:** Tack J, Depoortere I, Bisschops R, Delporte C, Coulie B, Meulemans A, Janssens J, Peeters T. Influence of ghrelin on interdigestive gastrointestinal motility in humans. Gut. 2006 Mar;55(3):327-33. doi: 10.1136/gut.2004.060426. Epub 2005 Oct 10.
**PMID:** 16216827
**Citation:** Coulie B, Tack J, Peeters T, Janssens J. Involvement of two different pathways in the motor effects of erythromycin on the gastric antrum in humans. Gut. 1998 Sep;43(3):395-400. doi: 10.1136/gut.43.3.395.
**PMID:** 9863486
**Citation:** Bayer LM, Bauers CM, Kapp SR. Psychosocial aspects of nutritional support. Nurs Clin North Am. 1983 Mar;18(1):119-28.
**PMID:** 6403929
**Citation:** Patel KA, Schlundt DG. Impact of moods and social context on eating behavior. Appetite. 2001 Apr;36(2):111-8. doi: 10.1006/appe.2000.0385.
**PMID:** 11237346
**Citation:** van Strien T, Oosterveld P. The children's DEBQ for assessment of restrained, emotional, and external eating in 7- to 12-year-old children. Int J Eat Disord. 2008 Jan;41(1):72-81. doi: 10.1002/eat.20424.
**PMID:** 17634965
**Citation:** Soetens B, Braet C, Van Vlierberghe L, Roets A. Resisting temptation: effects of exposure to a forbidden food on eating behaviour. Appetite. 2008 Jul;51(1):202-5. doi: 10.1016/j.appet.2008.01.007. Epub 2008 Feb 7.
**PMID:** 18342989
**Citation:** Wilson MM, Thomas DR, Rubenstein LZ, Chibnall JT, Anderson S, Baxi A, Diebold MR, Morley JE. Appetite assessment: simple appetite questionnaire predicts weight loss in community-dwelling adults and nursing home residents. Am J Clin Nutr. 2005 Nov;82(5):1074-81. doi: 10.1093/ajcn/82.5.1074.
**PMID:** 16280441
**Citation:** Lowe MR, Butryn ML, Didie ER, Annunziato RA, Thomas JG, Crerand CE, Ochner CN, Coletta MC, Bellace D, Wallaert M, Halford J. The Power of Food Scale. A new measure of the psychological influence of the food environment. Appetite. 2009 Aug;53(1):114-8. doi: 10.1016/j.appet.2009.05.016. Epub 2009 Jun 12.
**PMID:** 19500623
**Citation:** Hjelland IE, Ofstad AP, Narvestad JK, Berstad A, Hausken T. Drink tests in functional dyspepsia: which drink is best? Scand J Gastroenterol. 2004 Oct;39(10):933-7. doi: 10.1080/00365520410003344.
**PMID:** 15513330
**Citation:** Deloose E, Vos R, Janssen P, Van den Bergh O, Van Oudenhove L, Depoortere I, Tack J. The motilin receptor agonist erythromycin stimulates hunger and food intake through a cholinergic pathway. Am J Clin Nutr. 2016 Mar;103(3):730-7. doi: 10.3945/ajcn.115.113456. Epub 2016 Jan 27.
**PMID:** 26817505
## Derived Section
### Intervention Browse Module - Ancestors
- ID: D000000759
- Term: Adjuvants, Anesthesia
- ID: D000000889
- Term: Anti-Arrhythmia Agents
- ID: D000001993
- Term: Bronchodilator Agents
- ID: D000001337
- Term: Autonomic Agents
- ID: D000018373
- Term: Peripheral Nervous System Agents
- ID: D000045505
- Term: Physiological Effects of Drugs
- ID: D000018927
- Term: Anti-Asthmatic Agents
- ID: D000019141
- Term: Respiratory System Agents
- ID: D000009184
- Term: Mydriatics
- ID: D000010276
- Term: Parasympatholytics
- ID: D000018727
- Term: Muscarinic Antagonists
- ID: D000018680
- Term: Cholinergic Antagonists
- ID: D000018678
- Term: Cholinergic Agents
- ID: D000018377
- Term: Neurotransmitter Agents
- ID: D000045504
- Term: Molecular Mechanisms of Pharmacological Action
- ID: D000000900
- Term: Anti-Bacterial Agents
- ID: D000000890
- Term: Anti-Infective Agents
- ID: D000005765
- Term: Gastrointestinal Agents
- ID: D000011500
- Term: Protein Synthesis Inhibitors
- ID: D000004791
- Term: Enzyme Inhibitors
### Intervention Browse Module - Browse Branches
- Abbrev: Infe
- Name: Anti-Infective Agents
- Abbrev: Gast
- Name: Gastrointestinal Agents
- Abbrev: All
- Name: All Drugs and Chemicals
- Abbrev: AnArAg
- Name: Anti-Arrhythmia Agents
- Abbrev: AdjAn
- Name: Adjuvants, Anesthesia
- Abbrev: Resp
- Name: Respiratory System Agents
- Abbrev: CNSDep
- Name: Central Nervous System Depressants
### Intervention Browse Module - Browse Leaves
- ID: M8068
- Name: Erythromycin
- Relevance: HIGH
- As Found: 1 or 2
- ID: M8069
- Name: Erythromycin Estolate
- Relevance: HIGH
- As Found: 1 or 2
- ID: M18236
- Name: Erythromycin Ethylsuccinate
- Relevance: HIGH
- As Found: 1 or 2
- ID: M195803
- Name: Erythromycin stearate
- Relevance: HIGH
- As Found: 1 or 2
- ID: M4590
- Name: Atropine
- Relevance: HIGH
- As Found: Autism
- ID: M20758
- Name: Cholinergic Agents
- Relevance: LOW
- As Found: Unknown
- ID: M198321
- Name: Erythromycin lactobionate
- Relevance: HIGH
- As Found: Head part
- ID: M4107
- Name: Anesthetics
- Relevance: LOW
- As Found: Unknown
- ID: M4089
- Name: Adjuvants, Anesthesia
- Relevance: LOW
- As Found: Unknown
- ID: M4213
- Name: Anti-Arrhythmia Agents
- Relevance: LOW
- As Found: Unknown
- ID: M5269
- Name: Bronchodilator Agents
- Relevance: LOW
- As Found: Unknown
- ID: M20963
- Name: Anti-Asthmatic Agents
- Relevance: LOW
- As Found: Unknown
- ID: M21137
- Name: Respiratory System Agents
- Relevance: LOW
- As Found: Unknown
- ID: M12139
- Name: Mydriatics
- Relevance: LOW
- As Found: Unknown
- ID: M13189
- Name: Parasympatholytics
- Relevance: LOW
- As Found: Unknown
- ID: M20801
- Name: Muscarinic Antagonists
- Relevance: LOW
- As Found: Unknown
- ID: M20760
- Name: Cholinergic Antagonists
- Relevance: LOW
- As Found: Unknown
- ID: M20504
- Name: Neurotransmitter Agents
- Relevance: LOW
- As Found: Unknown
- ID: M4222
- Name: Anti-Bacterial Agents
- Relevance: LOW
- As Found: Unknown
- ID: M4214
- Name: Anti-Infective Agents
- Relevance: LOW
- As Found: Unknown
- ID: M8881
- Name: Gastrointestinal Agents
- Relevance: LOW
- As Found: Unknown
- ID: M7951
- Name: Enzyme Inhibitors
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: D000004917
- Term: Erythromycin
- ID: D000004918
- Term: Erythromycin Estolate
- ID: D000015643
- Term: Erythromycin Ethylsuccinate
- ID: C000011462
- Term: Erythromycin stearate
- ID: C000010948
- Term: Erythromycin lactobionate
- ID: D000001285
- Term: Atropine
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT05502679
**Brief Title:** Immediate Versus Late Weight Bearing After Tibial Plateau Fractures Internal Fixation
**Official Title:** Immediate Versus Late Weight Bearing After Tibial Plateau Fractures Internal Fixation: A Randomized Clinical Trial
#### Organization Study ID Info
**ID:** 17200756
#### Organization
**Class:** OTHER
**Full Name:** Assiut University
### Status Module
#### Completion Date
**Date:** 2024-05-01
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-03-28
**Type:** ACTUAL
**Last Update Submit Date:** 2024-03-27
**Overall Status:** RECRUITING
#### Primary Completion Date
**Date:** 2024-05-01
**Type:** ESTIMATED
#### Start Date
**Date:** 2022-09-01
**Type:** ACTUAL
**Status Verified Date:** 2024-03
#### Study First Post Date
**Date:** 2022-08-16
**Type:** ACTUAL
**Study First Submit Date:** 2022-08-11
**Study First Submit QC Date:** 2022-08-12
### Sponsor Collaborators Module
#### Collaborators
**Class:** OTHER
**Name:** Texas Tech University Health Sciences Center
#### Lead Sponsor
**Class:** OTHER
**Name:** Assiut University
#### Responsible Party
**Investigator Affiliation:** Assiut University
**Investigator Full Name:** Mariam ibrahim
**Investigator Title:** Principle investigator
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** Postoperative rehabilitation for tibial plateau fracture generally involves prolonged non-weight bearing time while other protocols use partial weight-bearing and bracing before full weight-bearing is recommended at 9 to 12 weeks following surgical fixation. No study to date has investigated the effect of standardized pragmatic exercise protocol added to immediate weight bearing after tibial plateau fractures surgical fixation on patient's functional outcomes, knee ROM, pain, radiographic boney alignment, gait, and return to work.
**Detailed Description:** Tibial plateau fractures can permanently affect patients' quality of life, including significant socio-economic impact due to time off work, compromised knee functional integrity, secondary knee osteoarthritis, knee flexion contractures, job loss due to functional limitations, and limited ability to return to pre-injury level of sports participation. Additionally, patients with tibial plateau fracture are at greater risk of death compared to an age- and the gender-matched reference population.
In orthopedics, weight-bearing refers to how much weight a person bears through an injured body part. During a single-leg stance, a person with no physical limitations will carry 100% of their body weight through each leg. Thus, grades of weight bearing are generally expressed as a percent of the body weight. Weight-bearing grades include (1) Non-weight bearing (NWB), which means the patient is not to put any weight through the affected limb(s); (2) Toe touch weight bearing (TTWB), which is poorly defined in the literature. In clinical practice, it is commonly described as having the ability to touch the toes to the floor without supporting weight from the affected limb. The pressure should be light enough to avoid crushing a potato crisp underfoot. Partial weight bearing (PWB) can range from anything greater than non-weight bearing to anything less than full weight bearing. The status is usually accompanied by a percentage figure to describe the extent of recommended weight bearing further. Most of the definitions in the literature define partial weight bearing as being 30% to 50% of a patient's body weight. Full weight bearing (FWB) means no restriction to weight bearing. In other words, 100% of a person's body weight can be transmitted through the designated limb. This term is somewhat interchangeable with the term 'weight bear as tolerated (WBAT), which allows them to self-limit their weight bearing up to full body weight. Restriction in weight bearing of the operated leg during standing and walking is needed to avoid complications during the postoperative recovery such as mal-union, fracture reduction loss, or hardware failure.
Postoperative rehabilitation for tibial plateau fracture generally involves prolonged non-weight bearing time, while other protocols use partial weight-bearing and bracing before full weight-bearing is recommended at 9 to 12 weeks following fixation. Early weight-bearing and early range of motion (ROM) for cartilage nourishment and preservation after selected lower limb surgical procedures are associated with positive postoperative outcomes, including decreased mortality and morbidity rate, functional improvements, reduced inpatient length of stay, and improved healing process. Early weight-bearing prescription, however, has to be carefully assessed, as it may result in fracture reduction loss, hardware failure, infection, malunion, or nonunion. The effectiveness of immediate partial post-operative weight-bearing in the management of lateral tibial plateau fractures resulted in favorable outcomes after immediate partial weight-bearing of 15 kg in cases of bicondylar tibial plateau fractures fixed with medial and lateral plating, and after immediate partial weight-bearing, up to 25 kg in all types of tibial plateau fractures fixed using a range of approaches.
By using locking plates for tibial plateau fracture surgical management, surgeons can safely allow immediate postoperative weight-bearing. Immediate weight bearing did not produce additional tibial plateau depression greater than 2 mm with Schatzker Type I, II, III, or Type V fractures. This could potentially reduce the rate of postoperative complications due to immobilization, such as deep venous thrombosis and joint stiffness.
Knee ROM limitations and altered gait characteristics are common complications after tibial plateau fractures. Most gait improvements occurred within the first postoperative six months. The total ROM at each lower limb joint showed positive correlations with the patients' capability to conduct normal activities of daily living.
To the authors' knowledge, no randomized control study to date has investigated in patients following tibial plateau fracture surgical fixation the effect of (1) adding immediate weight bearing to tolerance in addition to a specific, tailored exercise program adapted to the type and mechanism of tibial plateau fractures; and (2) adding phones follow-ups to improve compliance and decrease the cost of care.
### Conditions Module
**Conditions:**
- Fracture of Tibia Proximal Plateau
**Keywords:**
- rehabilitation
- radiography
- physical therapy
- intra-articular knee fracture
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
**Intervention Model Description:** Randomized Clinical Trial
##### Masking Info
**Masking:** DOUBLE
**Masking Description:** The first patient from each type of Schatzker classification 1-4 tibial plateau fractures will be randomly assigned to either WBG or TG. Then each patient will be alternatively assigned to WBG or TG as a stratification method to ensure that each group has equal distribution from each type of Schatzker classification 1-4 tibial plateau fractures. The randomization file will be generated by an investigator (TH) not involved in the data collection process, with the results stored in a spreadsheet accessible only to the investigator responsible for the subjects' group assignment (MI). This investigator will not participate in any data collection or subject treatment. Due to the nature of the study, participants will not be blinded to the group assignment and treatment they will receive. However, the investigators measuring the dependent variables will be blinded to the group assignment.
**Who Masked:**
- CARE_PROVIDER
- OUTCOMES_ASSESSOR
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 52
**Type:** ESTIMATED
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** 6-week non-weight bearing of the affected lower limb rehabilitation protocol (TG)
**Intervention Names:**
- Other: Pragmatic Exercise protocol
**Label:** Traditional Group
**Type:** ACTIVE_COMPARATOR
#### Arm Group 2
**Description:** Immediate lower limb weight bearing to tolerance rehabilitation protocol (WBG)
**Intervention Names:**
- Other: Weight bearing as tolerated
- Other: Pragmatic Exercise protocol
**Label:** Weight-bearing Group
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Weight-bearing Group
**Description:** Bearing weight on lower limb extremity
**Name:** Weight bearing as tolerated
**Type:** OTHER
#### Intervention 2
**Arm Group Labels:**
- Traditional Group
- Weight-bearing Group
**Description:** Designed exercise prescriptions according to the patients' needs
**Name:** Pragmatic Exercise protocol
**Type:** OTHER
### Outcomes Module
#### Other Outcomes
**Description:** yes or no question
**Measure:** Satisfaction with weight bearing protocol
**Time Frame:** 3 month after the surgery
**Description:** Scores range from 0-10 points, with higher scores indicating greater pain intensity.
**Measure:** The change in average pain intensity of the lower leg using the numeric Pain Rating Scale
**Time Frame:** Baseline, 2 and 6 weeks, 3 and 6 months after surgery
**Description:** Redflags regarding Articular congruency,the anatomical relationship of articular surfaces with or without hardware failure (Yes / NO) and visual intra-articular collapse will be measured to report bone alignment quality on x-ray
**Measure:** Anatomical relationship of articular surfaces on X-ray
**Time Frame:** 6 weeks after surgery
#### Primary Outcomes
**Description:** Arabic version of Oxford knee score.Functional knee questionnaire.The questionnaire consists of 12 questions that cover the function and pain of the knee. Each question is scored from 0 to 4 (0 being the worst outcome and 4 being the best). The overall score is the sum of all items and can range from 0 to 48, with higher scores corresponding to better outcomes.
**Measure:** The change in Oxford knee score (OKS) from 6 weeks to 3 months and 6 months after surgery
**Time Frame:** 6 weeks, 3 and 6 months after the surgery
**Description:** Measuring Active knee flexion and extension and at 3 month tibial rotation ROM will be measured
**Measure:** The change in active Knee range of motion
**Time Frame:** Baseline, 2 and 6 weeks, 3months after the surgery
**Description:** proximal medial tibial angle to detect varus / valgus angulation .
**Measure:** The change of radiograph measurements on X-ray
**Time Frame:** Baseline and 3 months after the surgery
**Description:** measurement of fracture gap, joint step off, tibial plateau width, tibial slope and depression will be measured to report quality of reduction and bony alignment.
**Measure:** The change on clinical impression of reduction quality on Computed tomography
**Time Frame:** Baseline and 3 months after the surgery
#### Secondary Outcomes
**Description:** work and Productivity Assessment outcomes are expressed as impairment percentages, with higher numbers indicating Greater impairment and less productivity, i.e., worse outcomes. minimum score is 0 and maximum is 100
**Measure:** The change of Return to work and Productivity Assessment (Arabic version).
**Time Frame:** 3- and 6-month post-surgery
**Description:** measuring the isometric muscle strength
**Measure:** The change in Hip Stability Isometric Test (HipSIT) and knee extensor strength using (handheld dynamometer)
**Time Frame:** 6 weeks and 3 months after the surgery
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
1. Women and men (18 to 65 years of age) admitted to Assiut University Hospital - Trauma unit with the diagnosis of traumatic tibial plateau closed fracture.
2. Open or arthroscopic internal fixation for tibial plateau fracture.
3. Reduction of tibia plateau depression is less than or equal to 2 mm (Beisemann et al. 2021)
4. Schatzker classification 1-4 tibial plateau fractures.
5. An Orthopedic surgeon with at least 5 years of surgery experience.
6. Precontoured and standard locking compression plates for the tibia plateau fracture internal fixation.
7. An excellent or good grade on Modified Rasmussen criteria.
Exclusion Criteria:
1-. Contralateral limb condition that prevents weight bearing 3. Ipsilateral injuries such as tibial or femoral fractures, hip fractures, or pelvic ring injuries.
4. Patients are required to wear a locking knee brace following the surgical fixation for a concomitant ligamentous knee injury.
5. Patient treated conservatively or with external fixation. 6. Surgical fixation is delayed for more than 10 days after the injury. 7. Requirement of involved leg fixed immobilization (e.g., cast) following the surgical fixation 8. Non-ambulatory pre-tibial plateau fracture 9. Pre-injury limitation to ROM of ipsilateral knee 10. Documented psychiatric disorder (aggressive, bipolar) requiring admission in the perioperative period.
11. Cognitive or mental condition that prevents the patient from following directions.
**Maximum Age:** 65 Years
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Mariam A Ibrahim, Master
**Phone:** +201001539399
**Role:** CONTACT
**Contact 2:**
**Email:** [email protected]
**Name:** Jean-Michel Brismee, Professor
**Phone:** 8067433243
**Role:** CONTACT
#### Locations
**Location 1:**
**City:** Assiut
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Mariam Ibrahim, Msc
- **Phone:** +201001539399
- **Role:** CONTACT
**Country:** Egypt
**Facility:** Assiut University Hospitals
**Status:** RECRUITING
**Zip:** 71515
#### Overall Officials
**Official 1:**
**Affiliation:** Texas Tech Health Sciences Center
**Name:** Jean-Michel Brismee, Professor
**Role:** STUDY_CHAIR
### IPD Sharing Statement Module
**Access Criteria:** Via email
**Description:** Deidentified data will be available for the other researchers and reviewers upon request.
**Info Types:**
- STUDY_PROTOCOL
- SAP
- ICF
**IPD Sharing:** YES
**Time Frame:** 30 days
### References Module
#### References
**Citation:** Ahmed KM, Said HG, Ramadan EKA, Abd El-Radi M, El-Assal MA. Arabic translation and validation of three knee scores, Lysholm Knee Score (LKS), Oxford Knee Score (OKS), and International Knee Documentation Committee Subjective Knee Form (IKDC). SICOT J. 2019;5:6. doi: 10.1051/sicotj/2018054. Epub 2019 Mar 8. Erratum In: SICOT J. 2019;5:27.
**PMID:** 30848244
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000014947
- Term: Wounds and Injuries
- ID: D000092443
- Term: Knee Fractures
- ID: D000007718
- Term: Knee Injuries
- ID: D000007869
- Term: Leg Injuries
### Condition Browse Module - Browse Branches
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: All
- Name: All Conditions
- Abbrev: BC26
- Name: Wounds and Injuries
### Condition Browse Module - Browse Leaves
- ID: M5114
- Name: Body Weight
- Relevance: LOW
- As Found: Unknown
- ID: M26370
- Name: Fractures, Bone
- Relevance: HIGH
- As Found: Fracture
- ID: M16737
- Name: Tibial Fractures
- Relevance: HIGH
- As Found: Fracture of Tibia
- ID: M2925
- Name: Tibial Plateau Fractures
- Relevance: HIGH
- As Found: Tibial Plateau Fractures
- ID: M2923
- Name: Knee Fractures
- Relevance: LOW
- As Found: Unknown
- ID: M17685
- Name: Wounds and Injuries
- Relevance: LOW
- As Found: Unknown
- ID: M10738
- Name: Knee Injuries
- Relevance: LOW
- As Found: Unknown
- ID: M10881
- Name: Leg Injuries
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000050723
- Term: Fractures, Bone
- ID: D000092463
- Term: Tibial Plateau Fractures
- ID: D000013978
- Term: Tibial Fractures
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT06347679
**Brief Title:** Effects of Aromatherapy on Anxiety and Pain During Dental Treatments in Adults: a Randomized Controlled Clinical Trial
**Official Title:** Effects of Aromatherapy on Anxiety and Pain During Dental Treatment in Adults: a Randomized Controlled Clinical Trial
#### Organization Study ID Info
**ID:** WCHSIRB-CT-2024-160
#### Organization
**Class:** OTHER
**Full Name:** Sichuan University
### Status Module
#### Completion Date
**Date:** 2026-04-01
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-04-04
**Type:** ACTUAL
**Last Update Submit Date:** 2024-03-28
**Overall Status:** NOT_YET_RECRUITING
#### Primary Completion Date
**Date:** 2025-04-01
**Type:** ESTIMATED
#### Start Date
**Date:** 2024-04-01
**Type:** ESTIMATED
**Status Verified Date:** 2024-03
#### Study First Post Date
**Date:** 2024-04-04
**Type:** ACTUAL
**Study First Submit Date:** 2024-03-28
**Study First Submit QC Date:** 2024-03-28
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Sichuan University
#### Responsible Party
**Investigator Affiliation:** Sichuan University
**Investigator Full Name:** He Cai
**Investigator Title:** Postdoc
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
### Description Module
**Brief Summary:** Dental anxiety is a common problem in dental care. The aim of this protocol is to evaluate the effect of orange and tea essential oil for the control of anxiety and pain in adults during dental treatments.
### Conditions Module
**Conditions:**
- Dental Anxiety
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** TRIPLE
**Who Masked:**
- CARE_PROVIDER
- INVESTIGATOR
- OUTCOMES_ASSESSOR
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 120
**Type:** ESTIMATED
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Intervention Names:**
- Other: Aroma diffuser with single-note sweet orange essential oil
**Label:** Group 1: single-note sweet orange essential oil
**Type:** EXPERIMENTAL
#### Arm Group 2
**Intervention Names:**
- Other: Aroma diffuser with single-note tea tree essential oil
**Label:** Group 2: single-note tea tree essential oil
**Type:** EXPERIMENTAL
#### Arm Group 3
**Intervention Names:**
- Other: Aroma diffuser with water
**Label:** Group 3: water
**Type:** SHAM_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Group 1: single-note sweet orange essential oil
**Description:** In a space of 3\*4\*2.6 cubic meters, positioned 10 cm away from the patient, add 4-6 drops (each drop being 0.05 ml) of 100% concentration single-note sweet orange essential oil into the aroma diffuser reservoir containing 120 ml of water.
**Name:** Aroma diffuser with single-note sweet orange essential oil
**Type:** OTHER
#### Intervention 2
**Arm Group Labels:**
- Group 2: single-note tea tree essential oil
**Description:** In a space of 3\*4\*2.6 cubic meters, positioned 10 cm away from the patient, add 4-6 drops (each drop being 0.05 ml) of 100% concentration single-note tea tree essential oil into the aroma diffuser reservoir containing 120 ml of water.
**Name:** Aroma diffuser with single-note tea tree essential oil
**Type:** OTHER
#### Intervention 3
**Arm Group Labels:**
- Group 3: water
**Description:** In a space of 3\*4\*2.6 cubic meters, positioned 10 cm away from the patient, add 4-6 drops (each drop being 0.05 ml) of water into the aroma diffuser reservoir containing 120 ml of water.
**Name:** Aroma diffuser with water
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Description:** The Modified Dental Anxiety Scale (MDAS) consists of 4 questions each with a 5 category rating scale, ranging from 'not anxious' to 'extremely anxious', hence, the total score varies from 4 to 20, with higher obtained scores describing more elevated levels of dental anxiety. MDAS is recorded before, during and after operation.
**Measure:** Anxiety scale 1 - Modified Dental Anxiety Scale (MDAS)
**Time Frame:** 5 minutes before operation/during operation (immediately after the most painful procedure)/immediately after operation
**Description:** The visual analog scale (VAS) is a validated, subjective measure for acute and chronic pain. Scores are recorded by making a handwritten mark on a 10-cm line that represents a continuum between "no pain" and "worst pain." VAS is recorded before, during and after operation.
**Measure:** Pain scale - Visual Analog Scale (VAS)
**Time Frame:** 5 minutes before operation/during operation (immediately after the most painful procedure)/immediately after operation
#### Secondary Outcomes
**Description:** The STAI-S evaluates the subjective feelings of tension, worry, nervousness, apprehension, and autonomic nervous system activation. This self-completed inventory consisted of 20 items on a four-point Likert scale running from one (i.e., not at all) to four (i.e., very much). Hence, the total score varies from 20 to 80, with higher obtained scores describing more elevated levels of anxiety. STAI-S is recorded before and after operation,
**Measure:** Anxiety scale 2 - State-Trait Anxiety Inventory for State Anxiety (STAI-S)
**Time Frame:** 5 minutes before operation/ immediately after operation
**Description:** systolic blood pressure (SBP) is measured using a manual sphygmomanometer, before, during and after operation.
**Measure:** Vital sign 1 - systolic blood pressure (SBP)
**Time Frame:** 5 minutes before operation/during operation (immediately after the most painful procedure, and every 10min after)/immediately after operation
**Description:** diastolic blood pressure (DBP) is measured using a manual sphygmomanometer, before, during and after operation.
**Measure:** Vital sign 2 - diastolic blood pressure (DBP)
**Time Frame:** 5 minutes before operation/during operation (immediately after the most painful procedure, and every 10min after)/immediately after operation
**Description:** Heart rate (HR）is measured as pulse rate using an oximeter, before, during and after operation.
**Measure:** Vital sign 3 - heart rate (HR）
**Time Frame:** 5 minutes before operation/during operation (immediately after the most painful procedure, and every 10min after)/immediately after operation
**Description:** oxygen saturation (SpO2）is measured using an oximeter, before, during and after operation.
**Measure:** Vital sign 4 - oxygen saturation (SpO2）
**Time Frame:** 5 minutes before operation/during operation (immediately after the most painful procedure, and every 10min after)/immediately after operation
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Patients who need dental treatment, such as resin filling, root canal treatment, periodontal initial treatment, tooth extraction, etc.
* Be able to understand the purpose of the study and volunteer to take part in the study.
* Be able to complete all assessments and follow-up during the study.
Exclusion Criteria:
* Patients who are allergic to the ingredients of the essential oils used in the experiment or to the materials used in the dental treatment.
* Patients who underwent only an oral examination.
* Patients with severe heart disease, hypertension, respiratory disease, or other systemic conditions that were not eligible for trial participation.
* Pregnant or lactating women.
* People with depressive symptoms, anxiety disorders or other mental illnesses that affect their mood state.
* People who are using sedative or analgesic drugs.
* Recent or current users of essential oil.
* Any circumstances considered by the investigator to be inappropriate for participation in this study.
**Healthy Volunteers:** True
**Maximum Age:** 40 Years
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** He Cai, MD PhD
**Phone:** +86-13281167556
**Role:** CONTACT
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000001523
- Term: Mental Disorders
### Condition Browse Module - Browse Branches
- Abbrev: BXM
- Name: Behaviors and Mental Disorders
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
### Condition Browse Module - Browse Leaves
- ID: M4324
- Name: Anxiety Disorders
- Relevance: HIGH
- As Found: Anxiety
- ID: M13066
- Name: Pain
- Relevance: LOW
- As Found: Unknown
- ID: M4815
- Name: Mental Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M14473
- Name: Psychotic Disorders
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000001008
- Term: Anxiety Disorders
### Intervention Browse Module - Browse Branches
- Abbrev: HB
- Name: Herbal and Botanical
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: T312
- Name: Tea
- Relevance: LOW
- As Found: Unknown
- ID: T244
- Name: Orange
- Relevance: HIGH
- As Found: Blood cells
- ID: T313
- Name: Tea Tree
- Relevance: HIGH
- As Found: Intrapulmonary
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT02844179
**Brief Title:** (+)-Alpha-Dihydrotetrabenazine Phase I
**Official Title:** (+)-Alpha-Dihydrotetrabenazine Phase I
#### Organization Study ID Info
**ID:** HUM00105810 -1
#### Organization
**Class:** OTHER
**Full Name:** University of Michigan
### Status Module
#### Completion Date
**Date:** 2017-06-27
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2017-08-22
**Type:** ACTUAL
**Last Update Submit Date:** 2017-08-21
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2017-06-27
**Type:** ACTUAL
#### Start Date
**Date:** 2016-07
**Status Verified Date:** 2017-08
#### Study First Post Date
**Date:** 2016-07-26
**Type:** ESTIMATED
**Study First Submit Date:** 2016-07-20
**Study First Submit QC Date:** 2016-07-25
### Sponsor Collaborators Module
#### Collaborators
**Class:** UNKNOWN
**Name:** Adeptio Pharmaceuticals
#### Lead Sponsor
**Class:** OTHER
**Name:** Kirk A. Frey
#### Responsible Party
**Investigator Affiliation:** University of Michigan
**Investigator Full Name:** Kirk A. Frey
**Investigator Title:** Professor of Radiology
**Type:** SPONSOR_INVESTIGATOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** This research study is intended to determine the initial safety and tolerability of single oral doses of the drug (+)-alpha-dihydrotetrabenaxine (HTBZ) in normal volunteers. HTBZ is believed to be the active ingredient in the FDA-approved drug tetrabenazine (TBZ, brand name Xenazine), prescribed for treatment of involuntary movements in patients with Huntington's chorea. TBZ is a mixture of closely-related compounds (isomers) and is readily metabolized (converted) in the human body to HTBZ and related isomers. Investigators believe that HTBZ, the drug to be studied in this research, is the active ingredient in TBZ. The present study will confirm safety and tolerability of HTBZ and will investigate its expected effects on brain sites that are the target of TBZ therapy.
### Conditions Module
**Conditions:**
- Healthy Subjects
### Design Module
#### Design Info
**Allocation:** NA
**Intervention Model:** SINGLE_GROUP
##### Masking Info
**Masking:** NONE
**Primary Purpose:** BASIC_SCIENCE
#### Enrollment Info
**Count:** 6
**Type:** ACTUAL
**Phases:**
- PHASE1
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Single dose administration of (+)-alpha-Dihydrotetrabenazine (HTBZ), escalating dosage amounts 7.5 - 30 mg orally
**Intervention Names:**
- Drug: HTBZ
**Label:** dose escalation
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- dose escalation
**Name:** HTBZ
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Measure:** VMAT2 occupancy by HTBZ determined by positron emission tomography (PET) scanning
**Time Frame:** 60 minutes post-administration of HTBZ
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* able to provide informed consent
Exclusion Criteria:
* pregnant or lactating female subjects
* Subjects taking medications that interfere with VMAT2 (ex amphetamine)
* History of significant neurologic or psychiatric conditions
* Significant active medical conditions
* Alcohol or illicit substance use or dependence
**Healthy Volunteers:** True
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Ann Arbor
**Country:** United States
**Facility:** University of Michigan hospitals
**State:** Michigan
**Zip:** 48109
#### Overall Officials
**Official 1:**
**Affiliation:** University of Michigan
**Name:** Kirk A Frey, MD, PhD
**Role:** PRINCIPAL_INVESTIGATOR
## Derived Section
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT04322279
**Brief Title:** Factors Associated With a Positive SARS-CoV-2 Serology in Contact Subjects at High/Moderate Risk of Coronavirus SARS-CoV-2 Infection. COVID19.
**Official Title:** Factors Associated With a Positive SARS-CoV-2 Serology in Contact Subjects at High/Moderate Risk of Coronavirus SARS-CoV-2 Infection. (CoV-CONTACT-SERO)
#### Organization Study ID Info
**ID:** C20-16
#### Organization
**Class:** OTHER_GOV
**Full Name:** Institut National de la Santé Et de la Recherche Médicale, France
#### Secondary ID Infos
**Domain:** RCB-ID
**ID:** 2020-A00609-30
**Type:** REGISTRY
### Status Module
#### Completion Date
**Date:** 2022-12-31
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2022-04-08
**Type:** ACTUAL
**Last Update Submit Date:** 2022-03-30
**Overall Status:** SUSPENDED
#### Primary Completion Date
**Date:** 2021-12-31
**Type:** ACTUAL
#### Start Date
**Date:** 2020-03-09
**Type:** ACTUAL
**Status Verified Date:** 2021-07
#### Study First Post Date
**Date:** 2020-03-26
**Type:** ACTUAL
**Study First Submit Date:** 2020-03-24
**Study First Submit QC Date:** 2020-03-24
**Why Stopped:** no topics to include
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER_GOV
**Name:** Institut National de la Santé Et de la Recherche Médicale, France
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
### Description Module
**Brief Summary:** In December 2019, a pneumonia due to a novel coronavirus (SARS-CoV-2) emerged in the city of Wuhan, in China. In a few weeks, the number of confirmed cases of SARS-CoV-2 infection has dramatically increased, with almost 150'000 cases and more than 6'000 reported deaths on March, 16th 2020.
Little is known on the rate of human-to-human transmission of this new coronavirus SARS-CoV-2 in the community and within the hospital.
Depending on the country, contact subjects considered to be at high or moderate risk of SARS-CoV-2 are, either isolated at home for a period of time defined by the health authorities or, on the contrary, continue their professional activity on the condition that they adopt measures to prevent transmission to those around them. In most European countries, healthcare workers adopt this second option. In all cases, it is most often recommended that contact persons monitor their state of health and communicate it to the persons dedicated to this action.
Whether such subjects become spreaders of the virus is not known, nor is the proportion of viral spreader who will develop a symptomatic infection.
In this study, we aim to evaluate the virological and clinical outcomes of subjects following a contact at high/moderate risk of SARS-CoV-2 acquisition, in community-subjects and/or healthcare workers.
The study population is represented by all subjects who had a contact with laboratory-confirmed SARS-CoV-2 cases and whose contact was considered to be at high/moderate risk of SARS-CoV-2 acquisition.
This include both children and adult subjects, subject without social security, and healthcare workers.
**Detailed Description:** Procedures added by the research:
Blood sampling for determination of the presence of SARS-CoV-2 type M immunoglobulins or type G immunoglobulins.
Blood sampling for whole exome sequencing
### Conditions Module
**Conditions:**
- Coronavirus
**Keywords:**
- contact subject
- high/moderate risk of transmission
### Design Module
#### Bio Spec
**Description:** Blood samples
**Retention:** SAMPLES_WITH_DNA
#### Design Info
**Observational Model:** COHORT
**Time Perspective:** PROSPECTIVE
#### Enrollment Info
**Count:** 300
**Type:** ESTIMATED
**Study Type:** OBSERVATIONAL
### Arms Interventions Module
### Interventions
#### Intervention 1
**Description:** SARS-CoV-2 serology
**Name:** Serology
**Type:** DIAGNOSTIC_TEST
#### Intervention 2
**Description:** Whole exome sequencing
**Name:** Sequencing
**Type:** GENETIC
### Outcomes Module
#### Primary Outcomes
**Description:** Positive serology defined as the presence of SARS-CoV-2 IgM or IgG and assessed by ELISA, microneutralisation assay
**Measure:** Proportion of subjects with SARS-CoV-2 positive serology at day 30 following the last high/moderate risk contact with a laboratory-confirmed SARS-CoV-2 case.
**Time Frame:** 30 days (+/-7)
#### Secondary Outcomes
**Description:** Positive serology defined as the presence of SARS-CoV-2 IgM or IgG and assessed by ELISA, microneutralisation assay
**Measure:** Factors associated with a SARS-CoV-2 positive serology at day 30 (+/-7);
**Time Frame:** 30 days (+/-7)
**Description:** ELISA, microneutralisation assay
**Measure:** Time (days) between the first positive SARS-CoV-2 serology and the first negative SARS-CoV-2 serology.
**Time Frame:** 365 days (+/-30)
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* High/moderate risk contact with a laboratory-confirmed SARS-CoV-2 case;
* Within the 14 days following the last contact with a laboratory-confirmed SARS-CoV-2 case;
* Obtaining informed consent.
Exclusion Criteria:
* Subject included in the CoV-CONTACT study
* Subject deprived of freedom
* Subject under a legal protective measure
**Sampling Method:** NON_PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- CHILD
- ADULT
- OLDER_ADULT
**Study Population:** The study population is represented by all subjects who had a contact with laboratory-confirmed SARS-CoV-2 cases and whose contact was considered to be at high/moderate risk of SARS-CoV-2 acquisition.
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Besançon
**Country:** France
**Facility:** Service de maladies infectieuses et tropicales Hôpital Jean Minjoz CHRU Besançon
**Zip:** 25030
**Location 2:**
**City:** Bordeaux
**Country:** France
**Facility:** Service des Maladies infectieuses et tropicales, Pôle Spécialités médicales, CHU Pellegrin
**Zip:** 33076
**Location 3:**
**City:** Clermont Ferrand
**Country:** France
**Facility:** Service des maladies infectieuses Hôpital Gabriel Montpied CHU de Clermont Ferrand
**Zip:** 63000
**Location 4:**
**City:** Dijon
**Country:** France
**Facility:** Centre d'investigation clinique 1432 Hôpital François Mitterrand CHU Bourgogne
**Zip:** 21 079
**Location 5:**
**City:** Grenoble
**Country:** France
**Facility:** Centre d'investigation clinique 1406 CHU Grenoble
**Zip:** 38043
**Location 6:**
**City:** Lille
**Country:** France
**Facility:** Centre d'Investigation Clinique 1403 -CHU Lille
**Zip:** 59037
**Location 7:**
**City:** Paris
**Country:** France
**Facility:** Centre d'Investigation Clinique Hôpital Saint Louis
**Zip:** 75010
**Location 8:**
**City:** Paris
**Country:** France
**Facility:** Centre d'investigation Clinique 1425, Hôpital Bichat Claude Bernard
**Zip:** 75018
**Location 9:**
**City:** Paris
**Country:** France
**Facility:** Hôpital Cochin CIC 1417 Bâtiment Lavoisier
**Zip:** 75679
**Location 10:**
**City:** Rennes
**Country:** France
**Facility:** Centre d'investigation clinique 1414 Service de Pharmacologie clinique CHU Rennes Hôpital Pontchaillou
**Zip:** 35033
**Location 11:**
**City:** Saint Denis
**Country:** France
**Facility:** Centre Hospitalier Félix Guyon Ile de la Réunion CHU nord
**Zip:** 97400
**Location 12:**
**City:** Saint Etienne
**Country:** France
**Facility:** Département maladie infectieux CHU Saint Etienne
**Zip:** 42055
**Location 13:**
**City:** Saint-Pierre
**Country:** France
**Facility:** Centre d'Investigation Clinique Ile de la Réunion CHU sud
**Zip:** 97448
**Location 14:**
**City:** Tours
**Country:** France
**Facility:** Centre Investigation Clinique 1415 CHRU Tours - Hôpital Bretonneau
**Zip:** 37000
**Location 15:**
**City:** Vandœuvre-lès-Nancy
**Country:** France
**Facility:** Centre Investigation Clinique 1433 CHRU de NANCY
**Zip:** 54511
**Location 16:**
**City:** Cayenne
**Country:** French Guiana
**Facility:** Service de Maladies infectieuses et tropicales Centre hospitalier
**Zip:** 97306
#### Overall Officials
**Official 1:**
**Affiliation:** Institut National de la Santé Et de la Recherche Médicale, France
**Name:** Xavier DUVAL, MD
**Role:** PRINCIPAL_INVESTIGATOR
### IPD Sharing Statement Module
**IPD Sharing:** UNDECIDED
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000007239
- Term: Infections
- ID: D000003333
- Term: Coronaviridae Infections
- ID: D000030341
- Term: Nidovirales Infections
- ID: D000012327
- Term: RNA Virus Infections
- ID: D000014777
- Term: Virus Diseases
- ID: D000011024
- Term: Pneumonia, Viral
- ID: D000011014
- Term: Pneumonia
- ID: D000012141
- Term: Respiratory Tract Infections
- ID: D000008171
- Term: Lung Diseases
- ID: D000012140
- Term: Respiratory Tract Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC01
- Name: Infections
- Abbrev: BC08
- Name: Respiratory Tract (Lung and Bronchial) Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
### Condition Browse Module - Browse Leaves
- ID: M2561
- Name: COVID-19
- Relevance: HIGH
- As Found: SARS-CoV-2 Infection
- ID: M10283
- Name: Infections
- Relevance: LOW
- As Found: Unknown
- ID: M6368
- Name: Communicable Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M20490
- Name: Coronavirus Infections
- Relevance: HIGH
- As Found: Coronavirus
- ID: M6555
- Name: Coronaviridae Infections
- Relevance: LOW
- As Found: Unknown
- ID: M23685
- Name: Nidovirales Infections
- Relevance: LOW
- As Found: Unknown
- ID: M17522
- Name: Virus Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M15149
- Name: RNA Virus Infections
- Relevance: LOW
- As Found: Unknown
- ID: M13904
- Name: Pneumonia
- Relevance: LOW
- As Found: Unknown
- ID: M13914
- Name: Pneumonia, Viral
- Relevance: LOW
- As Found: Unknown
- ID: M14978
- Name: Respiratory Tract Infections
- Relevance: LOW
- As Found: Unknown
- ID: M11168
- Name: Lung Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M14977
- Name: Respiratory Tract Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000018352
- Term: Coronavirus Infections
- ID: D000086382
- Term: COVID-19
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT04677179
**Acronym:** INSTRUCT-UC
**Brief Title:** A Study of LY3471851 in Adult Participants With Moderately to Severely Active Ulcerative Colitis (UC)
**Official Title:** An Adaptive Phase 2, Randomized, Double Blind, Placebo Controlled Study of LY3471851 (NKTR 358) in Patients With Moderately to Severely Active Ulcerative Colitis
#### Organization Study ID Info
**ID:** 17287
#### Organization
**Class:** INDUSTRY
**Full Name:** Nektar Therapeutics
#### Secondary ID Infos
**Domain:** Eli Lilly and Company
**ID:** J1P-MC-KFAH
**Type:** OTHER
**ID:** 2020-003017-35
**Type:** EUDRACT_NUMBER
### Status Module
#### Completion Date
**Date:** 2022-08-09
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2023-09-05
**Type:** ACTUAL
**Last Update Submit Date:** 2023-08-08
**Overall Status:** TERMINATED
#### Primary Completion Date
**Date:** 2022-08-09
**Type:** ACTUAL
#### Results First Post Date
**Date:** 2023-09-05
**Type:** ACTUAL
**Results First Submit Date:** 2023-08-08
**Results First Submit QC Date:** 2023-08-08
#### Start Date
**Date:** 2021-03-22
**Type:** ACTUAL
**Status Verified Date:** 2023-08
#### Study First Post Date
**Date:** 2020-12-21
**Type:** ACTUAL
**Study First Submit Date:** 2020-12-08
**Study First Submit QC Date:** 2020-12-17
**Why Stopped:** Study terminated due to enrollment futility.
### Sponsor Collaborators Module
#### Collaborators
**Class:** INDUSTRY
**Name:** Eli Lilly and Company
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Nektar Therapeutics
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** True
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** The reason for this study is to determine if the study drug LY3471851 is safe and effective in adult participants with active ulcerative colitis (UC). The study treatment will last about 52 weeks.
**Detailed Description:** In stage 1, two doses (high and low) of LY3471851 will be compared to placebo. In stage 2, up to two additional doses (to be confirmed) of LY3471851 will be compared to placebo.
LY3471851 (NKTR-358) is a potential first-in-class therapeutic that may address an underlying immune system imbalance in people with many autoimmune conditions. It targets the interleukin (IL-2) receptor complex in the body in order to stimulate proliferation of inhibitory immune cells known as regulatory T cells. By activating these cells, LY3471851 may act to bring the immune system back into balance.
### Conditions Module
**Conditions:**
- Colitis, Ulcerative
**Keywords:**
- T regulatory cells (Tregs)
- Interleukin 2
- Interleukin-2
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** DOUBLE
**Who Masked:**
- PARTICIPANT
- INVESTIGATOR
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 81
**Type:** ACTUAL
**Phases:**
- PHASE2
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Participants received a subcutaneous injection of high dose LY3471851 every 2 weeks from weeks 0 to 12. Week 12 responders entered the maintenance period and continued with the same treatment. Week 12 non-responders entered the extension period where they received subcutaneous injection of high dose LY3471851 every 2 weeks up to week 50. At week 26, extension period non-responders were discontinued from treatment. Post-treatment, participants entered follow-up period and were observed for 6 weeks for safety.
**Intervention Names:**
- Drug: LY3471851
**Label:** High dose LY3471851
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Participants received a subcutaneous injection of low dose LY3471851 every 2 weeks from weeks 0 to 12. Week 12 responders entered the maintenance period and continued with the same treatment. Week 12 non-responders entered the extension period where they received subcutaneous injection of high dose LY3471851 every 2 weeks up to week 50. At week 26, extension period non-responders were discontinued from treatment. Post-treatment, participants entered follow-up period and were observed for 6 weeks for safety.
**Intervention Names:**
- Drug: LY3471851
**Label:** Low dose LY3471851
**Type:** EXPERIMENTAL
#### Arm Group 3
**Description:** Participants received a subcutaneous injection of placebo every 2 weeks from weeks 0 to 12. Week 12 responders entered the maintenance period and continued with the same treatment. Week 12 non-responders entered the extension period where they received subcutaneous injection of high dose LY3471851 every 2 weeks up to week 50. At week 26, extension period non-responders were discontinued from treatment. Post-treatment, participants entered follow-up period and were observed for 6 weeks for safety.
**Intervention Names:**
- Drug: LY3471851
- Drug: Placebo
**Label:** Placebo
**Type:** PLACEBO_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- High dose LY3471851
- Low dose LY3471851
- Placebo
**Description:** administered SC
**Name:** LY3471851
**Other Names:**
- NKTR-358
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- Placebo
**Description:** administered SC
**Name:** Placebo
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Description:** Clinical remission is defined as achieving a Modified Mayo Score (MMS) sub-score for rectal bleeding=0, stool frequency=0, or stool frequency=1 with ≥ 1 point decrease from baseline, and endoscopy=0 or 1 (excluding friability). The MMS is a scoring system for assessment of UC and is composed of sub-scores of stool frequency (range: 0 to 3, where 0=normal number of stools, 3=5 or more stools more than normal), endoscopy (range: 0 to 3, where 0=normal or inactive disease, 3=severe disease \[spontaneous bleeding, ulceration\]), rectal bleeding (range: 0 to 3, where 0=no blood seen, 3=blood alone passed). Total MMS score is sum of all sub-scores and ranges from 0 to 9, with higher scores indicating higher disease activity.
**Measure:** Percentage of Participants Who Achieved Clinical Remission at Week 12
**Time Frame:** Week 12
#### Secondary Outcomes
**Description:** Clinical response is defined as a decrease in the MMS of ≥2 points and ≥30% decrease from baseline, and a decrease of ≥1 point in the rectal bleeding sub-score from baseline or a rectal bleeding score of 0 or 1. The MMS is a scoring system for assessment of UC and is composed of sub-scores of stool frequency (range: 0 to 3, where 0=normal number of stools, 3=5 or more stools more than normal), endoscopy (range: 0 to 3, where 0=normal or inactive disease, 3=severe disease \[spontaneous bleeding, ulceration\]), rectal bleeding (range: 0 to 3, where 0=no blood seen, 3=blood alone passed). Total MMS score is sum of all sub-scores and ranges from 0 to 9, with higher scores indicating higher disease activity.
**Measure:** Percentage of Participants Who Achieved Clinical Response at Week 12
**Time Frame:** Week 12
**Description:** Endoscopic remission is defined as achieving a MMS sub-score for endoscopy=0 or 1 (excluding friability). The MMS is a scoring system for assessment of UC and is composed of sub-scores of stool frequency (range: 0 to 3, where 0=normal number of stools, 3=5 or more stools more than normal), endoscopy (range: 0 to 3, where 0=normal or inactive disease, 3=severe disease \[spontaneous bleeding, ulceration\]), rectal bleeding (range: 0 to 3, where 0=no blood seen, 3=blood alone passed). Total MMS score is sum of all sub-scores and ranges from 0 to 9, with higher scores indicating higher disease activity.
**Measure:** Percentage of Participants Who Achieved Endoscopic Remission at Week 12
**Time Frame:** Week 12
**Description:** Endoscopic response is defined as a decrease of ≥1 point in the MMS endoscopy sub-score from baseline. The MMS is a scoring system for assessment of UC and is composed of sub-scores of stool frequency (range: 0 to 3, where 0=normal number of stools, 3=5 or more stools more than normal), endoscopy (range: 0 to 3, where 0=normal or inactive disease, 3=severe disease \[spontaneous bleeding, ulceration\]), rectal bleeding (range: 0 to 3, where 0=no blood seen, 3=blood alone passed). Total MMS score is sum of all sub-scores and ranges from 0 to 9, with higher scores indicating higher disease activity.
**Measure:** Percentage of Participants Who Achieved Endoscopic Response at Week 12
**Time Frame:** Week 12
**Description:** Symptomatic remission is defined as achieving a MMS sub-score for stool frequency=0, or stool frequency=1 with a decrease of ≥1 point from baseline, and rectal bleeding =0. The MMS is a scoring system for assessment of UC and is composed of sub-scores of stool frequency (range: 0 to 3, where 0=normal number of stools, 3=5 or more stools more than normal), endoscopy (range: 0 to 3, where 0=normal or inactive disease, 3=severe disease \[spontaneous bleeding, ulceration\]), rectal bleeding (range: 0 to 3, where 0=no blood seen, 3=blood alone passed). Total MMS score is sum of all sub-scores and ranges from 0 to 9, with higher scores indicating higher disease activity.
**Measure:** Percentage of Participants Who Achieved Symptomatic Remission at Week 12
**Time Frame:** Week 12
**Description:** Symptomatic response is defined as a ≥30% decrease from baseline in the composite clinical endpoint of the sum of MMS sub-scores of stool frequency and rectal bleeding. The MMS is a scoring system for assessment of UC and is composed of sub-scores of stool frequency (range: 0 to 3, where 0=normal number of stools, 3=5 or more stools more than normal), endoscopy (range: 0 to 3, where 0=normal or inactive disease, 3=severe disease \[spontaneous bleeding, ulceration\]), rectal bleeding (range: 0 to 3, where 0=no blood seen, 3=blood alone passed). Total MMS score is sum of all sub-scores and ranges from 0 to 9, with higher scores indicating higher disease activity.
**Measure:** Percentage of Participants Who Achieved Symptomatic Response at Week 12
**Time Frame:** Week 12
**Description:** Histologic Remission is defined as Geboes score \<2 or subscores = 0 for Grade 2a, 2b, 3, 4, and 5. The Geboes score is a 7-item instrument used to identify histologic changes in UC. The 7 items are Grade 0: Architectural changes (0=No abnormality to 3=Severe diffuse or multifocal abnormalities); Grade 1: Chronic inflammatory infiltrate (0=No increase to 3=Marked increase); Grade 2A: lamina propria eosinophils (0=No increase to 3=Marked increase); Grade 2B: lamina propria neutrophils (0= No increase to 3=Marked increase); Grade 3: Neutrophils in epithelium (0=None to 3=\>50% crypts involved); Grade 4: Crypt destruction(0=none to 3=Unequivocal crypt destruction),and Grade 5: Erosion or ulceration:(0=No erosion, ulceration or granulation to 4=Ulcer or granulation tissue). The grade with severe histological observation is considered the Geboes score and ranges from 0 to 4, with higher scores indicating severe disease.
**Measure:** Percentage of Participants Who Achieved Histologic Remission at Week 12
**Time Frame:** Week 12
**Description:** HEMH is defined as Geboes score \<2 AND endoscopic remission. Geboes score is a 7-item instrument used to identify histologic changes in UC. The 7-items are Grade 0: Architectural changes (0=No abnormality to 3=Severe diffuse or multifocal abnormalities); Grade 1: Chronic inflammatory infiltrate (0=No increase to 3=Marked increase); Grade 2A: lamina propria eosinophils (0=No increase to 3=Marked increase); Grade 2B: lamina propria neutrophils (0= No increase to 3=Marked increase); Grade 3: Neutrophils in epithelium (0=None to 3=\>50% crypts involved); Grade 4: Crypt destruction(0=none to 3=Unequivocal crypt destruction),and Grade 5: Erosion or ulceration:(0=No erosion, ulceration or granulation to 4=Ulcer or granulation tissue). The grade with severe histological observation is considered the Geboes score and ranges from 0 to 4, with higher scores indicating severe disease. Endoscopic remission is defined as achieving a MMS sub-score for endoscopy=0 or 1 (excluding friability).
**Measure:** Percentage of Participants Who Achieved Histologic-Endoscopic Mucosal Healing (HEMH)
**Time Frame:** Week 12
**Description:** IBDQ is a 32-item questionnaire that measures four aspects of participants' lives: symptoms directly related to the primary bowel disturbance (10 items), systemic symptoms (5 items), emotional function (12 items), and social function (5 items). Responses are graded on a 7-point Likert scale, where 7 denotes "not a problem at all" and 1 denotes "a very severe problem." The responses are summed to produce a total score ranging from 32 to 224, with higher score indicating a better quality of life. LS Mean was calculated using ANCOVA (analysis of covariance) model with treatment, baseline value, previous advanced therapy failure status (yes/no), baseline corticosteroid use (yes/no), baseline disease activity (MMS: \[4 to 6\] or \[7 to 9\]) and region (North America/Europe/Other) as fixed factors.
**Measure:** Change From Baseline in Inflammatory Bowel Disease Questionnaire (IBDQ) - Total Score
**Time Frame:** Baseline, Week 12
**Description:** C-trough is the concentration of drug in the blood immediately before the next dose was administered.
**Measure:** Pharmacokinetics (PK): Trough Concentration of LY3471851 (Ctrough) at Week 12
**Time Frame:** Predose at week 12
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Have moderately to severely active ulcerative colitis (UC) as defined by a modified Mayo score (MMS) of 4 to 9 with an endoscopic subscore (ES) ≥2, with endoscopy performed within 14 days before baseline.
* Have evidence of UC extending proximal to the rectum (with ≥15 centimeters (cm) of involved colon).
* Have up-to-date colorectal cancer surveillance performed according to local standard.
* Participants are either one of the following:
* Have failed conventional treatments including inability to tolerate oral or intravenous corticosteroids or immunomodulators (6-mercaptopurine or azathioprine or methotrexate), or history of corticosteroid dependence (an inability to successfully taper corticosteroids without return of UC) and neither failed or demonstrated intolerance to advanced therapy (eg, tumor necrosis factor (TNF) antagonists, anti-integrin therapies, anti-IL12/23p40 therapies, Janus kinase (JAK) inhibitor) OR,
* Have failed advanced therapies such as treatment with 1 or more advance therapies (eg, tumor necrosis factor \[TNF\] antagonists, anti-integrin therapies, anti-IL12/23p40 therapies, Janus kinase \[JAK\] inhibitor) at doses approved for the treatment of UC with documented history of failure to respond to or tolerate such treatment.
* Have had an established diagnosis of UC of ≥3 months in duration before baseline which includes endoscopic evidence of UC and a histopathology report that supports a diagnosis of UC. Supportive endoscopy and histopathology reports must be available in the source documents.
* Women of child-bearing potential (WOCBP) must test negative for pregnancy as indicated by a negative serum pregnancy test at the screening visit followed by a negative urine pregnancy test within 24 hours prior to first exposure to study drug.
Exclusion Criteria:
* Have been diagnosed with indeterminant colitis, proctitis (colitis limited to the rectum only; less than 15 centimeter (cm) from the anal verge or Crohn's disease.
* Have received any of the following for treatment of UC: cyclosporine, tacrolimus, mycophenolate mofetil or thalidomide within 2 weeks of screening, rectally administered corticosteroids or 5-aminosalicylic acid treatments within 2 weeks of screening.
* Have had or will need abdominal surgery for UC (for example, subtotal colectomy).
* Have failed 3 or more classes of advanced therapies approved for treatment of UC (eg, tumor necrosis factor \[TNF\] antagonists, anti-integrin therapies, anti-IL12/23p40 therapies, Janus kinase \[JAK\] inhibitor).
* Have evidence of toxic megacolon, intra-abdominal abscess, or stricture/stenosis within the small bowel or colon.
* Have any history or evidence of cancer of the gastrointestinal tract
* Have myocardial infarction, unstable ischemic heart disease, stroke or heart failure within 12 months prior to screening.
**Maximum Age:** 75 Years
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Litchfield Park
**Country:** United States
**Facility:** Dedicated Clinical Research
**State:** Arizona
**Zip:** 85340
**Location 2:**
**City:** Kissimmee
**Country:** United States
**Facility:** I.H.S. Health, LLC
**State:** Florida
**Zip:** 34741
**Location 3:**
**City:** Pensacola
**Country:** United States
**Facility:** Gastroenterology Associates of Pensacola, PA
**State:** Florida
**Zip:** 32503
**Location 4:**
**City:** Morristown
**Country:** United States
**Facility:** Atlantic Digestive Health Institute
**State:** New Jersey
**Zip:** 07960
**Location 5:**
**City:** Houston
**Country:** United States
**Facility:** Biopharma Informatic, LLC
**State:** Texas
**Zip:** 77084
**Location 6:**
**City:** San Antonio
**Country:** United States
**Facility:** Southern Star Research Institute, LLC
**State:** Texas
**Zip:** 78229
**Location 7:**
**City:** Ogden
**Country:** United States
**Facility:** Care Access Research - Ogden
**State:** Utah
**Zip:** 84403
**Location 8:**
**City:** Caba
**Country:** Argentina
**Facility:** DOM- Centro de Reumatologia
**State:** Buenos Aires
**Zip:** 1111
**Location 9:**
**City:** Caba
**Country:** Argentina
**Facility:** Centro de Educación Médica e Investigaciones Clínicas "Norberto Quirno" CEMIC
**State:** Buenos Aires
**Zip:** C1431FWO
**Location 10:**
**City:** Ciudad Autonoma De Buenos Air
**Country:** Argentina
**Facility:** Mautalen Salud e Investigacion-Centro de Osteopatías Médicas
**State:** Buenos Aires
**Zip:** C1128AAF
**Location 11:**
**City:** Tucumán
**Country:** Argentina
**Facility:** Centro Médico Privado de Reumatología
**Zip:** T4000AXL
**Location 12:**
**City:** Concord
**Country:** Australia
**Facility:** Concord Repatriation General Hospital
**State:** New South Wales
**Zip:** 2139
**Location 13:**
**City:** Albion
**Country:** Australia
**Facility:** Paratus Clinical Research Brisbane
**State:** Queensland
**Zip:** 4010
**Location 14:**
**City:** South Brisbane
**Country:** Australia
**Facility:** Mater Adult Hospital Brisbane
**State:** Queensland
**Zip:** 4700
**Location 15:**
**City:** Fitzroy
**Country:** Australia
**Facility:** St. Vincent's Hospital
**State:** Victoria
**Zip:** 3065
**Location 16:**
**City:** Brussels
**Country:** Belgium
**Facility:** Université Libre de Bruxelles - Hôpital Erasme
**State:** Bruxelles-Capitale, Région De
**Zip:** 1070
**Location 17:**
**City:** Tournai
**Country:** Belgium
**Facility:** Centre Hospitalier de Wallonie Picarde - Site Notre Dame
**State:** Wallonne, Région
**Zip:** 7500
**Location 18:**
**City:** Gent
**Country:** Belgium
**Facility:** AZ Maria Middelares
**Zip:** 9100
**Location 19:**
**City:** Brasília
**Country:** Brazil
**Facility:** Chronos Pesquisa Clínica
**State:** Distrito Federal
**Zip:** 72145-450
**Location 20:**
**City:** Porto Alegre
**Country:** Brazil
**Facility:** Nucleo de Pesquisa Clínica do Rio Grande do Sul-NPCRS
**State:** Rio Grande Do Sul
**Zip:** 90430-001
**Location 21:**
**City:** Campinas
**Country:** Brazil
**Facility:** HMCP - Hospital e Maternidade Celso Pierro - PUC-Campinas
**State:** Sao Paulo
**Zip:** 13060-904
**Location 22:**
**City:** Santo Andre
**Country:** Brazil
**Facility:** Pesquisare
**State:** Sao Paulo
**Zip:** 09080-110
**Location 23:**
**City:** Sao Paulo
**Country:** Brazil
**Facility:** Instituto de Assistencia Medica ao Servidor Publico Estudo Estadual
**State:** SP
**Zip:** 04039-004
**Location 24:**
**City:** Botucatu
**Country:** Brazil
**Facility:** Upeclin - Unidade de Pesquisa Clínica da Faculdade de Medicina de Botucatu - UNESP
**State:** São Paulo
**Zip:** 18618-687
**Location 25:**
**City:** São Bernardo do Campo
**Country:** Brazil
**Facility:** CEMEC - Centro Multidisciplinar de Estudos Clinicos EPP Ltda
**State:** São Paulo
**Zip:** 09715-090
**Location 26:**
**City:** Sao Paulo
**Country:** Brazil
**Facility:** Hepatogastro
**Zip:** 04543-001
**Location 27:**
**City:** Edmonton
**Country:** Canada
**Facility:** Gastroenterology and internal medicine research institute
**State:** Alberta
**Zip:** T5R 1W2
**Location 28:**
**City:** Halifax
**Country:** Canada
**Facility:** Gastroenterology Research, Nova Scotia Health Authority
**State:** Nova Scotia
**Zip:** B3H2Y9
**Location 29:**
**City:** Greenfield Park
**Country:** Canada
**Facility:** CISSS de la Montérégie - Centre Hôpital Charles-Le Moyne
**State:** Quebec
**Zip:** J4V2H1
**Location 30:**
**City:** Montreal
**Country:** Canada
**Facility:** McGill University
**State:** Quebec
**Zip:** H3G 1A4
**Location 31:**
**City:** HefeiCity
**Country:** China
**Facility:** The First Affiliated Hospital of Anhui Medical University
**State:** Anhui
**Zip:** 230022
**Location 32:**
**City:** Guangzhou
**Country:** China
**Facility:** First affiliated Hospital of Sun Yat-Sen University
**State:** Guangdong
**Zip:** 510080
**Location 33:**
**City:** Guangzhou
**Country:** China
**Facility:** The Sixth Affiliated Hospital, Sun Yat-Sen University
**State:** Guangdong
**Zip:** 510655
**Location 34:**
**City:** Wu Han
**Country:** China
**Facility:** Tongji Hosp Tongji Med Col Huazhong Univ of Sci & Tech
**State:** Hubei
**Zip:** 430030
**Location 35:**
**City:** Wuhan
**Country:** China
**Facility:** Union Hospital Tongji Medical College Huazhong University of Science and Technology
**State:** Hubei
**Zip:** 430022
**Location 36:**
**City:** Nanchang
**Country:** China
**Facility:** The First Affiliated Hospital of Nanchang University
**State:** Jiangxi
**Zip:** 330006
**Location 37:**
**City:** Taian
**Country:** China
**Facility:** Taian City Central Hospital
**State:** Shandong
**Zip:** 271000
**Location 38:**
**City:** Shanghai
**Country:** China
**Facility:** Shanghai Jiaotong University School of Medicine Ruijin Hospital
**State:** Shanghai
**Zip:** 200025
**Location 39:**
**City:** Hangzhou
**Country:** China
**Facility:** Sir Run Run Shaw Hospital
**State:** Zhejiang
**Zip:** 310018
**Location 40:**
**City:** Pilsen
**Country:** Czechia
**Facility:** A-Shine
**State:** Plzeň-město
**Zip:** 312 00
**Location 41:**
**City:** Olomouc
**Country:** Czechia
**Facility:** MUDr. Gregar, s.r.o.
**Zip:** 779 00
**Location 42:**
**City:** Olomouc
**Country:** Czechia
**Facility:** PreventaMed, s.r.o.
**Zip:** 779 00
**Location 43:**
**City:** Plzen-Lochotin
**Country:** Czechia
**Facility:** I. Interni klinika FN Plzen
**Zip:** 304 60
**Location 44:**
**City:** Slany
**Country:** Czechia
**Facility:** Nemocnice Slaný
**Zip:** 274 01
**Location 45:**
**City:** Grenoble Cedex 09
**Country:** France
**Facility:** CHU De Grenoble Hopital Albert Michallon
**Zip:** 38043
**Location 46:**
**City:** Mont-de-Marsan Cedex
**Country:** France
**Facility:** Centre Hospitalier de Mont de Marsan
**Zip:** 40024
**Location 47:**
**City:** Tbilisi
**Country:** Georgia
**Facility:** Acad. F. Todua Medical Center - Research Institute of Clinical Medicine
**Zip:** 0112
**Location 48:**
**City:** Tbilisi
**Country:** Georgia
**Facility:** Medical Center: Medinvestment
**Zip:** 0186
**Location 49:**
**City:** Budapest
**Country:** Hungary
**Facility:** Clinexpert SMO
**Zip:** 1033
**Location 50:**
**City:** Budapest
**Country:** Hungary
**Facility:** Óbudai Egészségügyi Centrum
**Zip:** 1036
**Location 51:**
**City:** Gyöngyös
**Country:** Hungary
**Facility:** Bugát Pál Kórház
**Zip:** 3200
**Location 52:**
**City:** Szekszard
**Country:** Hungary
**Facility:** CLINFAN Szolgáltató Kft
**Zip:** 7100
**Location 53:**
**City:** Rajkot
**Country:** India
**Facility:** Shree Giriraj Multispeciality Hospital
**State:** Gujarat
**Zip:** 360004
**Location 54:**
**City:** Surat
**Country:** India
**Facility:** Gujarat Hospital - Gastro and Vascular Centre
**State:** Gujarat
**Zip:** 395009
**Location 55:**
**City:** Nagpur
**Country:** India
**Facility:** Kingsway Hospital
**State:** Maharashtra
**Zip:** 440001
**Location 56:**
**City:** Nagpur
**Country:** India
**Facility:** Midas Multispeciality Hospital Pvt.Ltd.
**State:** Maharashtra
**Zip:** 440010
**Location 57:**
**City:** Jaipur
**Country:** India
**Facility:** SR Kalla Memorial Gastro & General Hospital
**State:** Rajasthan
**Zip:** 302006
**Location 58:**
**City:** Chennai
**Country:** India
**Facility:** Apollo Speciality Hospital - Teynampet
**State:** Tamil Nadu
**Zip:** 600035
**Location 59:**
**City:** Chandigarh
**Country:** India
**Facility:** Postgraduate Institute of Medical Education & Research
**Zip:** 160012
**Location 60:**
**City:** Telangana
**Country:** India
**Facility:** Gandhi Hospital
**Zip:** 500003
**Location 61:**
**City:** Beer Sheva
**Country:** Israel
**Facility:** Soroka Medical Center
**Zip:** 8410101
**Location 62:**
**City:** Nahariya
**Country:** Israel
**Facility:** Galilee Medical Center - Internal A
**Zip:** 22100
**Location 63:**
**City:** Rehovot
**Country:** Israel
**Facility:** Kaplan Medical Center
**Zip:** 7610001
**Location 64:**
**City:** Chikushino
**Country:** Japan
**Facility:** Fukuoka University Chikushi Hospital
**State:** Fukuoka
**Location 65:**
**City:** Sapporo-shi
**Country:** Japan
**Facility:** Tokushukai Sapporo Tokushukai Hospital
**State:** Hokkaido
**Zip:** 004 0041
**Location 66:**
**City:** Sapporo
**Country:** Japan
**Facility:** Sapporo Medical University Hospital
**State:** Hokkaido
**Location 67:**
**City:** Osaka-shi
**Country:** Japan
**Facility:** Infusion Clinic
**State:** Osaka-Fu
**Zip:** 530-0011
**Location 68:**
**City:** Fujiidera
**Country:** Japan
**Facility:** Sai Gastroenterologist Proctology
**State:** Osaka
**Zip:** 583-0027
**Location 69:**
**City:** Hamamatsu-shi
**Country:** Japan
**Facility:** Matsuda Hospital
**State:** Shizuoka-Ken
**Zip:** 4328061
**Location 70:**
**City:** Shinjuku-ku
**Country:** Japan
**Facility:** Center Hospital of the National Center for Global Health and Medicine
**State:** Tokyo-To
**Zip:** 162 8655
**Location 71:**
**City:** Koto-ku
**Country:** Japan
**Facility:** Showa University Koto Toyosu Hospital
**State:** Tokyo
**Zip:** 135 8577
**Location 72:**
**City:** Mitaka
**Country:** Japan
**Facility:** Kyorin University Hospital
**State:** Tokyo
**Zip:** 181-8611
**Location 73:**
**City:** Fukuoka
**Country:** Japan
**Facility:** Fukuoka University Hospital
**Zip:** 814-0180
**Location 74:**
**City:** Kagoshima
**Country:** Japan
**Facility:** Sameshima Hospital
**Zip:** 892-0846
**Location 75:**
**City:** Toyama
**Country:** Japan
**Facility:** Toyama Prefectural Central Hospital
**Zip:** 930-8550
**Location 76:**
**City:** Yamagata
**Country:** Japan
**Facility:** Yamagata University Hospital
**Zip:** 990-9585
**Location 77:**
**City:** Suwon-si
**Country:** Korea, Republic of
**Facility:** Ajou University Hospital
**State:** Gyeonggi-do
**Zip:** 443380
**Location 78:**
**City:** Seoul
**Country:** Korea, Republic of
**Facility:** Samsung Medical Center
**State:** Korea
**Zip:** 06351
**Location 79:**
**City:** Seoul
**Country:** Korea, Republic of
**Facility:** Seoul St. Mary's Hospital
**State:** Korea
**Zip:** 06591
**Location 80:**
**City:** Busan
**Country:** Korea, Republic of
**Facility:** Inje University Haeundae Paik Hospital
**Zip:** 48108
**Location 81:**
**City:** Gangwon-do
**Country:** Korea, Republic of
**Facility:** Yonsei University Wonju Severance Christian Hospital
**Zip:** 26426
**Location 82:**
**City:** Riga
**Country:** Latvia
**Facility:** Pauls Stradins Clinical Univeristy Hospital
**State:** Rīga
**Zip:** LV-1002
**Location 83:**
**City:** Warsaw
**Country:** Poland
**Facility:** NZOZ Vivamed
**State:** Mazowieckie
**Zip:** 03-580
**Location 84:**
**City:** Elblag
**Country:** Poland
**Facility:** Szpital Miejski Sw. Jana Pawla II
**Zip:** 82-300
**Location 85:**
**City:** Warszawa
**Country:** Poland
**Facility:** WIP Warsaw IBD Point Profesor Kierkus
**Zip:** 00-728
**Location 86:**
**City:** Zamosc
**Country:** Poland
**Facility:** ETG Zamość
**Zip:** 22-400
**Location 87:**
**City:** Oradea
**Country:** Romania
**Facility:** SC Pelican SRL
**State:** Bihor
**Zip:** 410469
**Location 88:**
**City:** Bucuresti
**Country:** Romania
**Facility:** SC Med Life SA
**Zip:** 010719
**Location 89:**
**City:** Bucuresti
**Country:** Romania
**Facility:** SC Centrul Medical Sana SRL
**Zip:** 011025
**Location 90:**
**City:** Bucuresti
**Country:** Romania
**Facility:** Spital Clinic Colentina
**Location 91:**
**City:** Cluj-Napoca
**Country:** Romania
**Facility:** Spitalul Clinic Judetean de Urgenta Cluj
**Zip:** 400006
**Location 92:**
**City:** Timisoara
**Country:** Romania
**Facility:** S.C. Materna Care S.R.L.
**Zip:** 300645
**Location 93:**
**City:** Moscow
**Country:** Russian Federation
**Facility:** Olla-Med
**State:** Moskva
**Zip:** 105554
**Location 94:**
**City:** Novosibirsk
**Country:** Russian Federation
**Facility:** Novosibirski Gastrocenter
**State:** Novosibirskaya Oblast'
**Zip:** 630007
**Location 95:**
**City:** Rostov-on-Don
**Country:** Russian Federation
**Facility:** Rostov State Medical University
**State:** Rostovskaya Oblast'
**Zip:** 344091
**Location 96:**
**City:** Moscow
**Country:** Russian Federation
**Facility:** Open Joint Stock Company Clinical and Diagnostic Center Euromedservice
**Location 97:**
**City:** Omsk
**Country:** Russian Federation
**Facility:** The University Clinic of OSMU
**Zip:** 644050
**Location 98:**
**City:** Saint-Petersburg
**Country:** Russian Federation
**Facility:** SPb SBIH "City Mariinskaya Hospital"
**Zip:** 194104
**Location 99:**
**City:** St. Petersburg
**Country:** Russian Federation
**Facility:** GOU VPO St-Petersburg SMA n/a Mechnikov Fed. Agen of Health
**Zip:** 195067
**Location 100:**
**City:** Banska Bystrica
**Country:** Slovakia
**Facility:** FNsP FDRoosevelta Banska Bystrica
**Zip:** 97517
**Location 101:**
**City:** Kosice
**Country:** Slovakia
**Facility:** ENDOMED s.r.o.
**Zip:** 04013
**Location 102:**
**City:** Kiev
**Country:** Ukraine
**Facility:** Medical Center of Limited Liability Company "Medical Center "Consilium Medical"
**State:** Kyiv
**Zip:** 4050
**Location 103:**
**City:** Lviv
**Country:** Ukraine
**Facility:** Lviv Railway Clinical Hospital
**State:** Lvivska Oblast
**Zip:** 79000
**Location 104:**
**City:** Lviv
**Country:** Ukraine
**Facility:** Lviv Regional Endocrinology Dispensary
**State:** Lvivska Oblast
**Zip:** 79000
**Location 105:**
**City:** Dnipro
**Country:** Ukraine
**Facility:** Medical Center of LLC Medical Center Clinic of Family Medicine
**Location 106:**
**City:** Kyiv
**Country:** Ukraine
**Facility:** International Institute of Clinical Trials LLC
**Zip:** 02091
**Location 107:**
**City:** Odesa
**Country:** Ukraine
**Facility:** Communal Enterprise "Odesa Regional Clinical Hospital"
**Zip:** 65000
**Location 108:**
**City:** Uzhgorod
**Country:** Ukraine
**Facility:** A. Novak Transcarpathian Regional Clinical Hospital
**Zip:** 88018
**Location 109:**
**City:** Vinnytsia
**Country:** Ukraine
**Facility:** CCH #1 Vinnytsia M.I. Pyrogov NMU Ch of Propaedeutics of IM
**Zip:** 21029
**Location 110:**
**City:** Vinnytsia
**Country:** Ukraine
**Facility:** Vinnytsia War Veterans Regional Clinical Hospital
**Location 111:**
**City:** Zaporizhzhia
**Country:** Ukraine
**Facility:** Diacenter LLC
**Zip:** 69076
**Location 112:**
**City:** Derby
**Country:** United Kingdom
**Facility:** Royal Derby Hospital
**State:** Derbyshire
**Zip:** DE22 3NE
**Location 113:**
**City:** Leytonstone
**Country:** United Kingdom
**Facility:** Whipps Cross University Hospital
**State:** London
**Zip:** E11 1NR
**Location 114:**
**City:** London
**Country:** United Kingdom
**Facility:** Guys/St. Thomas Hospital
**State:** Surrey
**Zip:** SE1 9RT
**Location 115:**
**City:** London
**Country:** United Kingdom
**Facility:** St. George's Hospital
**Zip:** SW17 0QT
**Location 116:**
**City:** York
**Country:** United Kingdom
**Facility:** York Hospital
**Zip:** YO31 8HE
#### Overall Officials
**Official 1:**
**Affiliation:** Nektar Therapeutics
**Name:** Study Director
**Role:** STUDY_DIRECTOR
### IPD Sharing Statement Module
**IPD Sharing:** NO
### References Module
#### See Also Links
**Label:** A Study of LY3471851 in Adult Participants With Moderately to Severely Active Ulcerative Colitis (UC) ( INSTRUCT-UC )
**URL:** https://trials.lillytrialguide.com/en-US/trial/V3FQqG8aJfjj6JtJ60xFt
## Document Section
### Large Document Module
#### Large Docs
- Date: 2021-06-03
- Filename: Prot_000.pdf
- Has ICF: False
- Has Protocol: True
- Has SAP: False
- Label: Study Protocol
- Size: 10139127
- Type Abbrev: Prot
- Upload Date: 2023-06-02T07:14
- Date: 2022-03-18
- Filename: SAP_001.pdf
- Has ICF: False
- Has Protocol: False
- Has SAP: True
- Label: Statistical Analysis Plan
- Size: 1285219
- Type Abbrev: SAP
- Upload Date: 2023-06-02T07:26
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000005759
- Term: Gastroenteritis
- ID: D000005767
- Term: Gastrointestinal Diseases
- ID: D000004066
- Term: Digestive System Diseases
- ID: D000003108
- Term: Colonic Diseases
- ID: D000007410
- Term: Intestinal Diseases
- ID: D000010335
- Term: Pathologic Processes
- ID: D000015212
- Term: Inflammatory Bowel Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC06
- Name: Digestive System Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
### Condition Browse Module - Browse Leaves
- ID: M6320
- Name: Colitis
- Relevance: HIGH
- As Found: Colitis
- ID: M6321
- Name: Colitis, Ulcerative
- Relevance: HIGH
- As Found: Colitis, Ulcerative
- ID: M17206
- Name: Ulcer
- Relevance: HIGH
- As Found: Ulcerative
- ID: M8875
- Name: Gastroenteritis
- Relevance: LOW
- As Found: Unknown
- ID: M7255
- Name: Digestive System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M8883
- Name: Gastrointestinal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M6336
- Name: Colonic Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M10444
- Name: Intestinal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M17917
- Name: Inflammatory Bowel Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000003092
- Term: Colitis
- ID: D000003093
- Term: Colitis, Ulcerative
- ID: D000014456
- Term: Ulcer
### Intervention Browse Module - Browse Branches
- Abbrev: ANeo
- Name: Antineoplastic Agents
- Abbrev: Analg
- Name: Analgesics
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M10411
- Name: Interleukin-2
- Relevance: LOW
- As Found: Unknown
### Misc Info Module
#### Removed Countries
- Country: Germany
- Version Holder: 2024-05-24
## Results Section
### Adverse Events Module
**Description:** All randomized participants who received at least one dose of study drug.
#### Event Groups
**Group ID:** EG000
**Title:** High Dose LY3471851 (Induction Treatment Period)
**Deaths Num At Risk:** 32
**Description:** Participants received a subcutaneous injection of high dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** EG000
**Other Num Affected:** 20
**Other Num at Risk:** 32
**Serious Number Affected:** 2
**Serious Number At Risk:** 32
**Title:** High Dose LY3471851 (Induction Treatment Period)
**Group ID:** EG001
**Title:** Low Dose LY3471851 (Induction Treatment Period)
**Deaths Num At Risk:** 35
**Description:** Participants received a subcutaneous injection of low dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** EG001
**Other Num Affected:** 15
**Other Num at Risk:** 35
**Serious Number At Risk:** 35
**Title:** Low Dose LY3471851 (Induction Treatment Period)
**Group ID:** EG002
**Title:** Placebo (Induction Treatment Period)
**Deaths Num At Risk:** 14
**Description:** Participants received a subcutaneous injection of placebo every 2 weeks from weeks 0 to 12.
**ID:** EG002
**Other Num Affected:** 5
**Other Num at Risk:** 14
**Serious Number At Risk:** 14
**Title:** Placebo (Induction Treatment Period)
**Group ID:** EG003
**Title:** High Dose LY3471851 (Maintenance Treatment Period)
**Deaths Num At Risk:** 8
**Description:** Week 12 responders from the high dose LY3471851 induction treatment period arm entered the maintenance period and continued with the same treatment.
**ID:** EG003
**Other Num Affected:** 4
**Other Num at Risk:** 8
**Serious Number At Risk:** 8
**Title:** High Dose LY3471851 (Maintenance Treatment Period)
**Group ID:** EG004
**Title:** Low Dose LY3471851 (Maintenance Treatment Period)
**Deaths Num At Risk:** 10
**Description:** Week 12 responders from the low dose LY3471851 induction treatment period arm entered the maintenance period and continued with the same treatment.
**ID:** EG004
**Other Num Affected:** 6
**Other Num at Risk:** 10
**Serious Number Affected:** 1
**Serious Number At Risk:** 10
**Title:** Low Dose LY3471851 (Maintenance Treatment Period)
**Group ID:** EG005
**Title:** Placebo (Maintenance Treatment Period)
**Deaths Num At Risk:** 5
**Description:** Week 12 responders from the placebo induction treatment period arm entered the maintenance period and continued with the same treatment.
**ID:** EG005
**Other Num Affected:** 1
**Other Num at Risk:** 5
**Serious Number At Risk:** 5
**Title:** Placebo (Maintenance Treatment Period)
**Group ID:** EG006
**Title:** High Dose LY3471851 (Extension Treatment Period)
**Deaths Num At Risk:** 26
**Description:** Week 12 non-responders entered the extension period where they received subcutaneous injection of high dose LY3471851 every 2 weeks up to week 50. At week 26, extension period non-responders were discontinued from treatment.
**ID:** EG006
**Other Num Affected:** 5
**Other Num at Risk:** 26
**Serious Number At Risk:** 26
**Title:** High Dose LY3471851 (Extension Treatment Period)
**Group ID:** EG007
**Title:** High Dose LY3471851 (Post-Treatment Follow-up Period)
**Deaths Num At Risk:** 25
**Description:** Participants randomised to high dose LY3471851 arm entered follow-up period from induction or maintenance, or extension periods and were observed for 6 weeks for safety. No treatments were administered.
**ID:** EG007
**Other Num at Risk:** 25
**Serious Number At Risk:** 25
**Title:** High Dose LY3471851 (Post-Treatment Follow-up Period)
**Group ID:** EG008
**Title:** Low Dose LY3471851 (Post-Treatment Follow-up Period)
**Deaths Num At Risk:** 30
**Description:** Participants randomised to low dose LY3471851 arm entered follow-up period from induction or maintenance, or extension periods and were observed for 6 weeks for safety. No treatments were administered.
**ID:** EG008
**Other Num Affected:** 1
**Other Num at Risk:** 30
**Serious Number Affected:** 1
**Serious Number At Risk:** 30
**Title:** Low Dose LY3471851 (Post-Treatment Follow-up Period)
**Group ID:** EG009
**Title:** Placebo (Post-Treatment Follow-up Period)
**Deaths Num At Risk:** 11
**Description:** Participants randomised to placebo arm entered follow-up period from induction or maintenance, or extension periods and were observed for 6 weeks for safety. No treatments were administered.
**ID:** EG009
**Other Num Affected:** 1
**Other Num at Risk:** 11
**Serious Number At Risk:** 11
**Title:** Placebo (Post-Treatment Follow-up Period)
**Frequency Threshold:** 5
#### Other Events
**Term:** Anaemia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Blood and lymphatic system disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Neutropenia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Blood and lymphatic system disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Extrasystoles
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Cardiac disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Abdominal pain
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Colitis ulcerative
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Nausea
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Application site reaction
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Hyperthermia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Influenza like illness
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Injection site reaction
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Malaise
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Pyrexia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Covid-19
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 25.1
**Term:** Erysipelas
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 25.1
**Term:** Influenza
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 25.1
**Term:** Nasopharyngitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 25.1
**Term:** Tonsillitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 25.1
**Term:** Injection related reaction
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Injury, poisoning and procedural complications
**Source Vocabulary:** MedDRA 25.1
**Term:** Body temperature increased
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** MedDRA 25.1
**Term:** Decreased appetite
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Arthritis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Back pain
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Musculoskeletal stiffness
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Myalgia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Headache
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Syncope
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Rhinorrhoea
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Acne
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Erythema
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** MedDRA 25.1
**Term:** Rosacea
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** MedDRA 25.1
#### Serious Events
**Term:** Proctitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 25.1
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 32
**Num Events:** 1
**Group ID:** EG001
**Num At Risk:** 35
**Group ID:** EG002
**Num At Risk:** 14
**Group ID:** EG003
**Num At Risk:** 8
**Group ID:** EG004
**Num At Risk:** 10
**Group ID:** EG005
**Num At Risk:** 5
**Group ID:** EG006
**Num At Risk:** 26
**Group ID:** EG007
**Num At Risk:** 25
**Group ID:** EG008
**Num At Risk:** 30
**Group ID:** EG009
**Num At Risk:** 11
**Term:** Vulval abscess
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 25.1
##### Stats
**Group ID:** EG000
**Num At Risk:** 32
**Group ID:** EG001
**Num At Risk:** 35
**Group ID:** EG002
**Num At Risk:** 14
**Group ID:** EG003
**Num At Risk:** 8
**Group ID:** EG004
**Num Affected:** 1
**Num At Risk:** 10
**Num Events:** 1
**Group ID:** EG005
**Num At Risk:** 5
**Group ID:** EG006
**Num At Risk:** 26
**Group ID:** EG007
**Num At Risk:** 25
**Group ID:** EG008
**Num At Risk:** 30
**Group ID:** EG009
**Num At Risk:** 11
**Term:** Lower limb fracture
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Injury, poisoning and procedural complications
**Source Vocabulary:** MedDRA 25.1
##### Stats
**Group ID:** EG000
**Num At Risk:** 32
**Group ID:** EG001
**Num At Risk:** 35
**Group ID:** EG002
**Num At Risk:** 14
**Group ID:** EG003
**Num At Risk:** 8
**Group ID:** EG004
**Num At Risk:** 10
**Group ID:** EG005
**Num At Risk:** 5
**Group ID:** EG006
**Num At Risk:** 26
**Group ID:** EG007
**Num At Risk:** 25
**Group ID:** EG008
**Num Affected:** 1
**Num At Risk:** 30
**Num Events:** 1
**Group ID:** EG009
**Num At Risk:** 11
**Term:** Syncope
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA 25.1
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 32
**Num Events:** 1
**Group ID:** EG001
**Num At Risk:** 35
**Group ID:** EG002
**Num At Risk:** 14
**Group ID:** EG003
**Num At Risk:** 8
**Group ID:** EG004
**Num At Risk:** 10
**Group ID:** EG005
**Num At Risk:** 5
**Group ID:** EG006
**Num At Risk:** 26
**Group ID:** EG007
**Num At Risk:** 25
**Group ID:** EG008
**Num At Risk:** 30
**Group ID:** EG009
**Num At Risk:** 11
**Time Frame:** Baseline to Follow-up (Up To Week 58)
## Results Section - Baseline Characteristics Module
### Denomination Counts
**Group ID:** BG000
**Value:** 32
**Group ID:** BG001
**Value:** 35
**Group ID:** BG002
**Value:** 14
**Group ID:** BG003
**Value:** 81
**Units:** Participants
### Group
**ID:** BG000
**Title:** High Dose LY3471851 (Induction Treatment Period)
**Description:** Participants received a subcutaneous injection of high dose LY3471851 every 2 weeks from weeks 0 to 12.
### Group
**ID:** BG001
**Title:** Low Dose LY3471851 (Induction Treatment Period)
**Description:** Participants received a subcutaneous injection of low dose LY3471851 every 2 weeks from weeks 0 to 12.
### Group
**ID:** BG002
**Title:** Placebo (Induction Treatment Period)
**Description:** Participants received a subcutaneous injection of placebo every 2 weeks from weeks 0 to 12.
### Group
**ID:** BG003
**Title:** Total
**Description:** Total of all reporting groups
### Measure
#### Measurement
**Group ID:** BG000
**Spread:** 12.5
**Value:** 39.2
#### Measurement
**Group ID:** BG001
**Spread:** 13.8
**Value:** 44.5
#### Measurement
**Group ID:** BG002
**Spread:** 15.2
**Value:** 45.7
#### Measurement
**Group ID:** BG003
**Spread:** 13.7
**Value:** 42.6
#### Denomination
**Units:** Participants
##### Denomination Counts
**Group ID:** BG000
**Value:** 32
**Group ID:** BG001
**Value:** 35
**Group ID:** BG002
**Value:** 14
**Group ID:** BG003
**Value:** 81
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 10
#### Measurement
**Group ID:** BG001
**Value:** 9
#### Measurement
**Group ID:** BG002
**Value:** 6
#### Measurement
**Group ID:** BG003
**Value:** 25
**Category Title:** Female
#### Measurement
**Group ID:** BG000
**Value:** 22
#### Measurement
**Group ID:** BG001
**Value:** 26
#### Measurement
**Group ID:** BG002
**Value:** 8
#### Measurement
**Group ID:** BG003
**Value:** 56
**Category Title:** Male
#### Denomination
**Units:** Participants
##### Denomination Counts
**Group ID:** BG000
**Value:** 32
**Group ID:** BG001
**Value:** 35
**Group ID:** BG002
**Value:** 14
**Group ID:** BG003
**Value:** 81
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 0
#### Measurement
**Group ID:** BG003
**Value:** 0
**Category Title:** American Indian or Alaska Native
#### Measurement
**Group ID:** BG000
**Value:** 5
#### Measurement
**Group ID:** BG001
**Value:** 4
#### Measurement
**Group ID:** BG002
**Value:** 2
#### Measurement
**Group ID:** BG003
**Value:** 11
**Category Title:** Asian
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 0
#### Measurement
**Group ID:** BG003
**Value:** 0
**Category Title:** Native Hawaiian or Other Pacific Islander
#### Measurement
**Group ID:** BG000
**Value:** 2
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 0
#### Measurement
**Group ID:** BG003
**Value:** 2
**Category Title:** Black or African American
#### Measurement
**Group ID:** BG000
**Value:** 25
#### Measurement
**Group ID:** BG001
**Value:** 31
#### Measurement
**Group ID:** BG002
**Value:** 12
#### Measurement
**Group ID:** BG003
**Value:** 68
**Category Title:** White
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 0
#### Measurement
**Group ID:** BG003
**Value:** 0
**Category Title:** More than one race
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 0
#### Measurement
**Group ID:** BG003
**Value:** 0
**Category Title:** Unknown or Not Reported
#### Denomination
**Units:** Participants
##### Denomination Counts
**Group ID:** BG000
**Value:** 32
**Group ID:** BG001
**Value:** 35
**Group ID:** BG002
**Value:** 14
**Group ID:** BG003
**Value:** 81
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 2
#### Measurement
**Group ID:** BG001
**Value:** 1
#### Measurement
**Group ID:** BG002
**Value:** 1
#### Measurement
**Group ID:** BG003
**Value:** 4
#### Denomination
**Units:** Participants
##### Denomination Counts
**Group ID:** BG000
**Value:** 31
**Group ID:** BG001
**Value:** 35
**Group ID:** BG002
**Value:** 14
**Group ID:** BG003
**Value:** 80
**Class Title:** Argentina
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 1
#### Measurement
**Group ID:** BG002
**Value:** 0
#### Measurement
**Group ID:** BG003
**Value:** 1
#### Denomination
**Units:** Participants
##### Denomination Counts
**Group ID:** BG000
**Value:** 31
**Group ID:** BG001
**Value:** 35
**Group ID:** BG002
**Value:** 14
**Group ID:** BG003
**Value:** 80
**Class Title:** Australia
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 1
#### Measurement
**Group ID:** BG003
**Value:** 1
#### Denomination
**Units:** Participants
##### Denomination Counts
**Group ID:** BG000
**Value:** 31
**Group ID:** BG001
**Value:** 35
**Group ID:** BG002
**Value:** 14
**Group ID:** BG003
**Value:** 80
**Class Title:** Belgium
#### Measurement
**Group ID:** BG000
**Value:** 2
#### Measurement
**Group ID:** BG001
**Value:** 4
#### Measurement
**Group ID:** BG002
**Value:** 0
#### Measurement
**Group ID:** BG003
**Value:** 6
#### Denomination
**Units:** Participants
##### Denomination Counts
**Group ID:** BG000
**Value:** 31
**Group ID:** BG001
**Value:** 35
**Group ID:** BG002
**Value:** 14
**Group ID:** BG003
**Value:** 80
**Class Title:** Czechia
#### Measurement
**Group ID:** BG000
**Value:** 3
#### Measurement
**Group ID:** BG001
**Value:** 3
#### Measurement
**Group ID:** BG002
**Value:** 0
#### Measurement
**Group ID:** BG003
**Value:** 6
#### Denomination
**Units:** Participants
##### Denomination Counts
**Group ID:** BG000
**Value:** 31
**Group ID:** BG001
**Value:** 35
**Group ID:** BG002
**Value:** 14
**Group ID:** BG003
**Value:** 80
**Class Title:** Hungary
#### Measurement
**Group ID:** BG000
**Value:** 3
#### Measurement
**Group ID:** BG001
**Value:** 1
#### Measurement
**Group ID:** BG002
**Value:** 1
#### Measurement
**Group ID:** BG003
**Value:** 5
#### Denomination
**Units:** Participants
##### Denomination Counts
**Group ID:** BG000
**Value:** 31
**Group ID:** BG001
**Value:** 35
**Group ID:** BG002
**Value:** 14
**Group ID:** BG003
**Value:** 80
**Class Title:** Japan
#### Measurement
**Group ID:** BG000
**Value:** 1
#### Measurement
**Group ID:** BG001
**Value:** 3
#### Measurement
**Group ID:** BG002
**Value:** 1
#### Measurement
**Group ID:** BG003
**Value:** 5
#### Denomination
**Units:** Participants
##### Denomination Counts
**Group ID:** BG000
**Value:** 31
**Group ID:** BG001
**Value:** 35
**Group ID:** BG002
**Value:** 14
**Group ID:** BG003
**Value:** 80
**Class Title:** South Korea
#### Measurement
**Group ID:** BG000
**Value:** 2
#### Measurement
**Group ID:** BG001
**Value:** 3
#### Measurement
**Group ID:** BG002
**Value:** 0
#### Measurement
**Group ID:** BG003
**Value:** 5
#### Denomination
**Units:** Participants
##### Denomination Counts
**Group ID:** BG000
**Value:** 31
**Group ID:** BG001
**Value:** 35
**Group ID:** BG002
**Value:** 14
**Group ID:** BG003
**Value:** 80
**Class Title:** Latvia
#### Measurement
**Group ID:** BG000
**Value:** 2
#### Measurement
**Group ID:** BG001
**Value:** 2
#### Measurement
**Group ID:** BG002
**Value:** 1
#### Measurement
**Group ID:** BG003
**Value:** 5
#### Denomination
**Units:** Participants
##### Denomination Counts
**Group ID:** BG000
**Value:** 31
**Group ID:** BG001
**Value:** 35
**Group ID:** BG002
**Value:** 14
**Group ID:** BG003
**Value:** 80
**Class Title:** Poland
#### Measurement
**Group ID:** BG000
**Value:** 5
#### Measurement
**Group ID:** BG001
**Value:** 5
#### Measurement
**Group ID:** BG002
**Value:** 3
#### Measurement
**Group ID:** BG003
**Value:** 13
#### Denomination
**Units:** Participants
##### Denomination Counts
**Group ID:** BG000
**Value:** 31
**Group ID:** BG001
**Value:** 35
**Group ID:** BG002
**Value:** 14
**Group ID:** BG003
**Value:** 80
**Class Title:** Russia
#### Measurement
**Group ID:** BG000
**Value:** 1
#### Measurement
**Group ID:** BG001
**Value:** 1
#### Measurement
**Group ID:** BG002
**Value:** 1
#### Measurement
**Group ID:** BG003
**Value:** 3
#### Denomination
**Units:** Participants
##### Denomination Counts
**Group ID:** BG000
**Value:** 31
**Group ID:** BG001
**Value:** 35
**Group ID:** BG002
**Value:** 14
**Group ID:** BG003
**Value:** 80
**Class Title:** Slovakia
#### Measurement
**Group ID:** BG000
**Value:** 7
#### Measurement
**Group ID:** BG001
**Value:** 7
#### Measurement
**Group ID:** BG002
**Value:** 4
#### Measurement
**Group ID:** BG003
**Value:** 18
#### Denomination
**Units:** Participants
##### Denomination Counts
**Group ID:** BG000
**Value:** 31
**Group ID:** BG001
**Value:** 35
**Group ID:** BG002
**Value:** 14
**Group ID:** BG003
**Value:** 80
**Class Title:** Ukraine
#### Measurement
**Group ID:** BG000
**Value:** 3
#### Measurement
**Group ID:** BG001
**Value:** 4
#### Measurement
**Group ID:** BG002
**Value:** 1
#### Measurement
**Group ID:** BG003
**Value:** 8
#### Denomination
**Units:** Participants
##### Denomination Counts
**Group ID:** BG000
**Value:** 31
**Group ID:** BG001
**Value:** 35
**Group ID:** BG002
**Value:** 14
**Group ID:** BG003
**Value:** 80
**Class Title:** United States
**Denomination Units Selected:**
## Results Section - Baseline Characteristics Module
### Measure 1
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Age, Continuous
**Unit of Measure:** years
### Measure 2
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Sex: Female, Male
**Unit of Measure:** Participants
### Measure 3
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Race (NIH/OMB)
**Unit of Measure:** Participants
### Measure 4
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** All randomized participants with non-missing region of enrollment data.
**Title:** Region of Enrollment
**Unit of Measure:** Participants
**Population Description:** All randomized participants.
## Results Section - More Information Module
### Certain Agreement
**Restriction Type:** GT60
**Restrictive Agreement:** True
### Point of Contact
**Email:** [email protected]
**Organization:** Nektar Therapeutics
**Phone:** 855-482-8676
**Title:** Study Director
## Results Section - Outcome Measures Module
### Outcome Measure 1
#### Analysis
**CI Lower Limit:** 0.03
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:** 95
**CI Upper Limit:** 11.32
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY
**Other Analysis Description:**
**P-Value:** 0.750
**P-Value Comment:**
**Parameter Type:** Odds Ratio (OR)
**Parameter Value:** 0.57
**Statistical Comment:**
**Statistical Method:** Cochran-Mantel-Haenszel
**Tested Non-Inferiority:**
#### Analysis
**CI Lower Limit:** 0.10
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:** 95
**CI Upper Limit:** 6.21
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY
**Other Analysis Description:**
**P-Value:** 0.838
**P-Value Comment:**
**Parameter Type:** Odds Ratio (OR)
**Parameter Value:** 0.80
**Statistical Comment:**
**Statistical Method:** Cochran-Mantel-Haenszel
**Tested Non-Inferiority:**
### Outcome Measure 2
#### Analysis
**CI Lower Limit:** 0.10
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:** 95
**CI Upper Limit:** 3.30
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY
**Other Analysis Description:**
**P-Value:** 0.563
**P-Value Comment:**
**Parameter Type:** Odds Ratio (OR)
**Parameter Value:** 0.57
**Statistical Comment:**
**Statistical Method:** Cochran-Mantel-Haenszel
**Tested Non-Inferiority:**
#### Analysis
**CI Lower Limit:** 0.17
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:** 95
**CI Upper Limit:** 3.64
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY
**Other Analysis Description:**
**P-Value:** 0.759
**P-Value Comment:**
**Parameter Type:** Odds Ratio (OR)
**Parameter Value:** 0.78
**Statistical Comment:**
**Statistical Method:** Cochran-Mantel-Haenszel
**Tested Non-Inferiority:**
### Outcome Measure 3
#### Analysis
**CI Lower Limit:** 0.02
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:** 95
**CI Upper Limit:** 1.93
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY
**Other Analysis Description:**
**P-Value:** 0.163
**P-Value Comment:**
**Parameter Type:** Odds Ratio (OR)
**Parameter Value:** 0.18
**Statistical Comment:**
**Statistical Method:** Cochran-Mantel-Haenszel
**Tested Non-Inferiority:**
#### Analysis
**CI Lower Limit:** 0.11
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:** 95
**CI Upper Limit:** 2.77
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY
**Other Analysis Description:**
**P-Value:** 0.439
**P-Value Comment:**
**Parameter Type:** Odds Ratio (OR)
**Parameter Value:** 0.54
**Statistical Comment:**
**Statistical Method:** Cochran-Mantel-Haenszel
**Tested Non-Inferiority:**
### Outcome Measure 4
#### Analysis
**CI Lower Limit:** 0.17
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:** 95
**CI Upper Limit:** 9.88
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY
**Other Analysis Description:**
**P-Value:** 0.821
**P-Value Comment:**
**Parameter Type:** Odds Ratio (OR)
**Parameter Value:** 1.29
**Statistical Comment:**
**Statistical Method:** Cochran-Mantel-Haenszel
**Tested Non-Inferiority:**
#### Analysis
**CI Lower Limit:** 0.37
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:** 95
**CI Upper Limit:** 5.49
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY
**Other Analysis Description:**
**P-Value:** 0.572
**P-Value Comment:**
**Parameter Type:** Odds Ratio (OR)
**Parameter Value:** 1.43
**Statistical Comment:**
**Statistical Method:** Cochran-Mantel-Haenszel
**Tested Non-Inferiority:**
### Outcome Measure 5
#### Analysis
**CI Lower Limit:** 0.14
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:** 95
**CI Upper Limit:** 10.16
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY
**Other Analysis Description:**
**P-Value:** 0.886
**P-Value Comment:**
**Parameter Type:** Odds Ratio (OR)
**Parameter Value:** 1.18
**Statistical Comment:**
**Statistical Method:** Cochran-Mantel-Haenszel
**Tested Non-Inferiority:**
#### Analysis
**CI Lower Limit:** 0.14
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:** 95
**CI Upper Limit:** 4.18
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY
**Other Analysis Description:**
**P-Value:** 0.784
**P-Value Comment:**
**Parameter Type:** Odds Ratio (OR)
**Parameter Value:** 0.78
**Statistical Comment:**
**Statistical Method:** Cochran-Mantel-Haenszel
**Tested Non-Inferiority:**
### Outcome Measure 6
#### Analysis
**CI Lower Limit:** 0.06
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:** 95
**CI Upper Limit:** 2.43
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY
**Other Analysis Description:**
**P-Value:** 0.331
**P-Value Comment:**
**Parameter Type:** Odds Ratio (OR)
**Parameter Value:** 0.37
**Statistical Comment:**
**Statistical Method:** Cochran-Mantel-Haenszel
**Tested Non-Inferiority:**
#### Analysis
**CI Lower Limit:** 0.10
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:** 95
**CI Upper Limit:** 2.52
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY
**Other Analysis Description:**
**P-Value:** 0.407
**P-Value Comment:**
**Parameter Type:** Odds Ratio (OR)
**Parameter Value:** 0.51
**Statistical Comment:**
**Statistical Method:** Cochran-Mantel-Haenszel
**Tested Non-Inferiority:**
### Outcome Measure 7
#### Analysis
**CI Lower Limit:** 0.38
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:** 95
**CI Upper Limit:** 6.00
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY
**Other Analysis Description:**
**P-Value:** 0.564
**P-Value Comment:**
**Parameter Type:** Risk Ratio (RR)
**Parameter Value:** 1.50
**Statistical Comment:**
**Statistical Method:** Cochran-Mantel-Haenszel
**Tested Non-Inferiority:**
#### Analysis
**CI Lower Limit:** 0.21
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:** 95
**CI Upper Limit:** 10.97
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY
**Other Analysis Description:**
**P-Value:** 0.681
**P-Value Comment:**
**Parameter Type:** Risk Ratio (RR)
**Parameter Value:** 1.51
**Statistical Comment:**
**Statistical Method:** Cochran-Mantel-Haenszel
**Tested Non-Inferiority:**
### Outcome Measure 8
#### Analysis
**CI Lower Limit:** -3.3
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:** 95
**CI Upper Limit:** 10.4
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY
**Other Analysis Description:**
**P-Value:** 0.317
**P-Value Comment:**
**Parameter Type:** Risk Difference (RD)
**Parameter Value:** 3.6
**Statistical Comment:**
**Statistical Method:** Cochran-Mantel-Haenszel
**Tested Non-Inferiority:**
#### Analysis
**CI Lower Limit:** -2.3
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:** 95
**CI Upper Limit:** 16.1
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY
**Other Analysis Description:**
**P-Value:** 0.238
**P-Value Comment:**
**Parameter Type:** Risk Difference (RD)
**Parameter Value:** 6.9
**Statistical Comment:**
**Statistical Method:** Cochran-Mantel-Haenszel
**Tested Non-Inferiority:**
### Outcome Measure 9
#### Analysis
**CI Lower Limit:** -12.17
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:** 95
**CI Upper Limit:** 31.60
**CI Upper Limit Comment:**
**Dispersion Type:** STANDARD_ERROR_OF_MEAN
**Dispersion Value:** 10.935
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY
**Other Analysis Description:**
**P-Value:** 0.378
**P-Value Comment:**
**Parameter Type:** LS Mean Difference
**Parameter Value:** 9.71
**Statistical Comment:**
**Statistical Method:** ANCOVA
**Tested Non-Inferiority:**
#### Analysis
**CI Lower Limit:** -21.78
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:** 95
**CI Upper Limit:** 19.88
**CI Upper Limit Comment:**
**Dispersion Type:** STANDARD_ERROR_OF_MEAN
**Dispersion Value:** 10.406
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY
**Other Analysis Description:**
**P-Value:** 0.928
**P-Value Comment:**
**Parameter Type:** LS Mean Difference
**Parameter Value:** -0.95
**Statistical Comment:**
**Statistical Method:** ANCOVA
**Tested Non-Inferiority:**
### Outcome Measure 10
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:** 0
- **Spread:**
- **Upper Limit:** 32.6
- **Value:** 14.3
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:** 0
- **Spread:**
- **Upper Limit:** 16.7
- **Value:** 7.1
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:** 3.5
- **Spread:**
- **Upper Limit:** 31
- **Value:** 17.2
**Title:**
#### Outcome Measure 2
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:** 10.6
- **Spread:**
- **Upper Limit:** 60.8
- **Value:** 35.7
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:** 21.2
- **Spread:**
- **Upper Limit:** 57.4
- **Value:** 39.3
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:** 23.5
- **Spread:**
- **Upper Limit:** 59.3
- **Value:** 41.4
**Title:**
#### Outcome Measure 3
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:** 4.9
- **Spread:**
- **Upper Limit:** 52.2
- **Value:** 28.6
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:** 1.3
- **Spread:**
- **Upper Limit:** 27.2
- **Value:** 14.3
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:** 8.6
- **Spread:**
- **Upper Limit:** 39.7
- **Value:** 24.1
**Title:**
#### Outcome Measure 4
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:** 0
- **Spread:**
- **Upper Limit:** 42.9
- **Value:** 21.4
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:** 14.8
- **Spread:**
- **Upper Limit:** 49.4
- **Value:** 32.1
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:** 20.3
- **Spread:**
- **Upper Limit:** 55.6
- **Value:** 37.9
**Title:**
#### Outcome Measure 5
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:** 0
- **Spread:**
- **Upper Limit:** 42.9
- **Value:** 21.4
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:** 14.8
- **Spread:**
- **Upper Limit:** 49.4
- **Value:** 32.1
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:** 11.3
- **Spread:**
- **Upper Limit:** 43.9
- **Value:** 27.6
**Title:**
#### Outcome Measure 6
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:** 16.9
- **Spread:**
- **Upper Limit:** 68.8
- **Value:** 42.9
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:** 24.5
- **Spread:**
- **Upper Limit:** 61.2
- **Value:** 42.9
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:** 26.7
- **Spread:**
- **Upper Limit:** 62.9
- **Value:** 44.8
**Title:**
#### Outcome Measure 7
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:** 0.0
- **Spread:**
- **Upper Limit:** 20.6
- **Value:** 7.1
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:** 0.0
- **Spread:**
- **Upper Limit:** 16.7
- **Value:** 7.1
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:** 0.0
- **Spread:**
- **Upper Limit:** 21.4
- **Value:** 10.3
**Title:**
#### Outcome Measure 8
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:** 0
- **Spread:**
- **Upper Limit:** 0
- **Value:** 0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:** 0
- **Spread:**
- **Upper Limit:** 10.4
- **Value:** 3.6
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:** 0
- **Spread:**
- **Upper Limit:** 16.1
- **Value:** 6.9
**Title:**
#### Outcome Measure 9
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 9.346
- **Upper Limit:**
- **Value:** 26.71
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 7.640
- **Upper Limit:**
- **Value:** 36.42
- **Comment:**
- **Group ID:** OG002
- **Lower Limit:**
- **Spread:** 7.143
- **Upper Limit:**
- **Value:** 25.76
**Title:**
#### Outcome Measure 10
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 49
- **Upper Limit:**
- **Value:** 91.3
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 105
- **Upper Limit:**
- **Value:** 139
**Title:**
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Description:** Clinical remission is defined as achieving a Modified Mayo Score (MMS) sub-score for rectal bleeding=0, stool frequency=0, or stool frequency=1 with ≥ 1 point decrease from baseline, and endoscopy=0 or 1 (excluding friability). The MMS is a scoring system for assessment of UC and is composed of sub-scores of stool frequency (range: 0 to 3, where 0=normal number of stools, 3=5 or more stools more than normal), endoscopy (range: 0 to 3, where 0=normal or inactive disease, 3=severe disease \[spontaneous bleeding, ulceration\]), rectal bleeding (range: 0 to 3, where 0=no blood seen, 3=blood alone passed). Total MMS score is sum of all sub-scores and ranges from 0 to 9, with higher scores indicating higher disease activity.
**Dispersion Type:** 95% Confidence Interval
**Parameter Type:** NUMBER
**Population Description:** All randomized participants who received at least one dose of study drug and had MMS data at week 12.
**Reporting Status:** POSTED
**Time Frame:** Week 12
**Title:** Percentage of Participants Who Achieved Clinical Remission at Week 12
**Type:** PRIMARY
**Unit of Measure:** percentage of participants
##### Group
**Description:** Participants received a subcutaneous injection of placebo every 2 weeks from weeks 0 to 12.
**ID:** OG000
**Title:** Placebo (Induction Treatment Period)
##### Group
**Description:** Participants received a subcutaneous injection of low dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** OG001
**Title:** Low Dose LY3471851 (Induction Treatment Period)
##### Group
**Description:** Participants received a subcutaneous injection of high dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** OG002
**Title:** High Dose LY3471851 (Induction Treatment Period)
#### Outcome Measure 2
**Description:** Clinical response is defined as a decrease in the MMS of ≥2 points and ≥30% decrease from baseline, and a decrease of ≥1 point in the rectal bleeding sub-score from baseline or a rectal bleeding score of 0 or 1. The MMS is a scoring system for assessment of UC and is composed of sub-scores of stool frequency (range: 0 to 3, where 0=normal number of stools, 3=5 or more stools more than normal), endoscopy (range: 0 to 3, where 0=normal or inactive disease, 3=severe disease \[spontaneous bleeding, ulceration\]), rectal bleeding (range: 0 to 3, where 0=no blood seen, 3=blood alone passed). Total MMS score is sum of all sub-scores and ranges from 0 to 9, with higher scores indicating higher disease activity.
**Dispersion Type:** 95% Confidence Interval
**Parameter Type:** NUMBER
**Population Description:** All randomized participants who received at least one dose of study drug and had MMS data at week 12.
**Reporting Status:** POSTED
**Time Frame:** Week 12
**Title:** Percentage of Participants Who Achieved Clinical Response at Week 12
**Type:** SECONDARY
**Unit of Measure:** percentage of participants
##### Group
**Description:** Participants received a subcutaneous injection of placebo every 2 weeks from weeks 0 to 12.
**ID:** OG000
**Title:** Placebo (Induction Treatment Period)
##### Group
**Description:** Participants received a subcutaneous injection of low dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** OG001
**Title:** Low Dose LY3471851 (Induction Treatment Period)
##### Group
**Description:** Participants received a subcutaneous injection of high dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** OG002
**Title:** High Dose LY3471851 (Induction Treatment Period)
#### Outcome Measure 3
**Description:** Endoscopic remission is defined as achieving a MMS sub-score for endoscopy=0 or 1 (excluding friability). The MMS is a scoring system for assessment of UC and is composed of sub-scores of stool frequency (range: 0 to 3, where 0=normal number of stools, 3=5 or more stools more than normal), endoscopy (range: 0 to 3, where 0=normal or inactive disease, 3=severe disease \[spontaneous bleeding, ulceration\]), rectal bleeding (range: 0 to 3, where 0=no blood seen, 3=blood alone passed). Total MMS score is sum of all sub-scores and ranges from 0 to 9, with higher scores indicating higher disease activity.
**Dispersion Type:** 95% Confidence Interval
**Parameter Type:** NUMBER
**Population Description:** All randomized participants who received at least one dose of study drug and had MMS data at week 12.
**Reporting Status:** POSTED
**Time Frame:** Week 12
**Title:** Percentage of Participants Who Achieved Endoscopic Remission at Week 12
**Type:** SECONDARY
**Unit of Measure:** percentage of participants
##### Group
**Description:** Participants received a subcutaneous injection of placebo every 2 weeks from weeks 0 to 12.
**ID:** OG000
**Title:** Placebo (Induction Treatment Period)
##### Group
**Description:** Participants received a subcutaneous injection of low dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** OG001
**Title:** Low Dose LY3471851 (Induction Treatment Period)
##### Group
**Description:** Participants received a subcutaneous injection of high dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** OG002
**Title:** High Dose LY3471851 (Induction Treatment Period)
#### Outcome Measure 4
**Description:** Endoscopic response is defined as a decrease of ≥1 point in the MMS endoscopy sub-score from baseline. The MMS is a scoring system for assessment of UC and is composed of sub-scores of stool frequency (range: 0 to 3, where 0=normal number of stools, 3=5 or more stools more than normal), endoscopy (range: 0 to 3, where 0=normal or inactive disease, 3=severe disease \[spontaneous bleeding, ulceration\]), rectal bleeding (range: 0 to 3, where 0=no blood seen, 3=blood alone passed). Total MMS score is sum of all sub-scores and ranges from 0 to 9, with higher scores indicating higher disease activity.
**Dispersion Type:** 95% Confidence Interval
**Parameter Type:** NUMBER
**Population Description:** All randomized participants who received at least one dose of study drug and had MMS data at week 12.
**Reporting Status:** POSTED
**Time Frame:** Week 12
**Title:** Percentage of Participants Who Achieved Endoscopic Response at Week 12
**Type:** SECONDARY
**Unit of Measure:** percentage of participants
##### Group
**Description:** Participants received a subcutaneous injection of placebo every 2 weeks from weeks 0 to 12.
**ID:** OG000
**Title:** Placebo (Induction Treatment Period)
##### Group
**Description:** Participants received a subcutaneous injection of low dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** OG001
**Title:** Low Dose LY3471851 (Induction Treatment Period)
##### Group
**Description:** Participants received a subcutaneous injection of high dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** OG002
**Title:** High Dose LY3471851 (Induction Treatment Period)
#### Outcome Measure 5
**Description:** Symptomatic remission is defined as achieving a MMS sub-score for stool frequency=0, or stool frequency=1 with a decrease of ≥1 point from baseline, and rectal bleeding =0. The MMS is a scoring system for assessment of UC and is composed of sub-scores of stool frequency (range: 0 to 3, where 0=normal number of stools, 3=5 or more stools more than normal), endoscopy (range: 0 to 3, where 0=normal or inactive disease, 3=severe disease \[spontaneous bleeding, ulceration\]), rectal bleeding (range: 0 to 3, where 0=no blood seen, 3=blood alone passed). Total MMS score is sum of all sub-scores and ranges from 0 to 9, with higher scores indicating higher disease activity.
**Dispersion Type:** 95% Confidence Interval
**Parameter Type:** NUMBER
**Population Description:** All randomized participants who received at least one dose of study drug and had MMS data at week 12.
**Reporting Status:** POSTED
**Time Frame:** Week 12
**Title:** Percentage of Participants Who Achieved Symptomatic Remission at Week 12
**Type:** SECONDARY
**Unit of Measure:** percentage of participants
##### Group
**Description:** Participants received a subcutaneous injection of placebo every 2 weeks from weeks 0 to 12.
**ID:** OG000
**Title:** Placebo (Induction Treatment Period)
##### Group
**Description:** Participants received a subcutaneous injection of low dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** OG001
**Title:** Low Dose LY3471851 (Induction Treatment Period)
##### Group
**Description:** Participants received a subcutaneous injection of high dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** OG002
**Title:** High Dose LY3471851 (Induction Treatment Period)
#### Outcome Measure 6
**Description:** Symptomatic response is defined as a ≥30% decrease from baseline in the composite clinical endpoint of the sum of MMS sub-scores of stool frequency and rectal bleeding. The MMS is a scoring system for assessment of UC and is composed of sub-scores of stool frequency (range: 0 to 3, where 0=normal number of stools, 3=5 or more stools more than normal), endoscopy (range: 0 to 3, where 0=normal or inactive disease, 3=severe disease \[spontaneous bleeding, ulceration\]), rectal bleeding (range: 0 to 3, where 0=no blood seen, 3=blood alone passed). Total MMS score is sum of all sub-scores and ranges from 0 to 9, with higher scores indicating higher disease activity.
**Dispersion Type:** 95% Confidence Interval
**Parameter Type:** NUMBER
**Population Description:** All randomized participants who received at least one dose of study drug and had MMS data at week 12.
**Reporting Status:** POSTED
**Time Frame:** Week 12
**Title:** Percentage of Participants Who Achieved Symptomatic Response at Week 12
**Type:** SECONDARY
**Unit of Measure:** percentage of participants
##### Group
**Description:** Participants received a subcutaneous injection of placebo every 2 weeks from weeks 0 to 12.
**ID:** OG000
**Title:** Placebo (Induction Treatment Period)
##### Group
**Description:** Participants received a subcutaneous injection of low dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** OG001
**Title:** Low Dose LY3471851 (Induction Treatment Period)
##### Group
**Description:** Participants received a subcutaneous injection of high dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** OG002
**Title:** High Dose LY3471851 (Induction Treatment Period)
#### Outcome Measure 7
**Description:** Histologic Remission is defined as Geboes score \<2 or subscores = 0 for Grade 2a, 2b, 3, 4, and 5. The Geboes score is a 7-item instrument used to identify histologic changes in UC. The 7 items are Grade 0: Architectural changes (0=No abnormality to 3=Severe diffuse or multifocal abnormalities); Grade 1: Chronic inflammatory infiltrate (0=No increase to 3=Marked increase); Grade 2A: lamina propria eosinophils (0=No increase to 3=Marked increase); Grade 2B: lamina propria neutrophils (0= No increase to 3=Marked increase); Grade 3: Neutrophils in epithelium (0=None to 3=\>50% crypts involved); Grade 4: Crypt destruction(0=none to 3=Unequivocal crypt destruction),and Grade 5: Erosion or ulceration:(0=No erosion, ulceration or granulation to 4=Ulcer or granulation tissue). The grade with severe histological observation is considered the Geboes score and ranges from 0 to 4, with higher scores indicating severe disease.
**Dispersion Type:** 95% Confidence Interval
**Parameter Type:** NUMBER
**Population Description:** All randomized participants who received at least one dose of study drug and had Geboes data at week 12.
**Reporting Status:** POSTED
**Time Frame:** Week 12
**Title:** Percentage of Participants Who Achieved Histologic Remission at Week 12
**Type:** SECONDARY
**Unit of Measure:** percentage of participants
##### Group
**Description:** Participants received a subcutaneous injection of placebo every 2 weeks from weeks 0 to 12.
**ID:** OG000
**Title:** Placebo (Induction Treatment Period)
##### Group
**Description:** Participants received a subcutaneous injection of low dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** OG001
**Title:** Low Dose LY3471851 (Induction Treatment Period)
##### Group
**Description:** Participants received a subcutaneous injection of high dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** OG002
**Title:** High Dose LY3471851 (Induction Treatment Period)
#### Outcome Measure 8
**Description:** HEMH is defined as Geboes score \<2 AND endoscopic remission. Geboes score is a 7-item instrument used to identify histologic changes in UC. The 7-items are Grade 0: Architectural changes (0=No abnormality to 3=Severe diffuse or multifocal abnormalities); Grade 1: Chronic inflammatory infiltrate (0=No increase to 3=Marked increase); Grade 2A: lamina propria eosinophils (0=No increase to 3=Marked increase); Grade 2B: lamina propria neutrophils (0= No increase to 3=Marked increase); Grade 3: Neutrophils in epithelium (0=None to 3=\>50% crypts involved); Grade 4: Crypt destruction(0=none to 3=Unequivocal crypt destruction),and Grade 5: Erosion or ulceration:(0=No erosion, ulceration or granulation to 4=Ulcer or granulation tissue). The grade with severe histological observation is considered the Geboes score and ranges from 0 to 4, with higher scores indicating severe disease. Endoscopic remission is defined as achieving a MMS sub-score for endoscopy=0 or 1 (excluding friability).
**Dispersion Type:** 95% Confidence Interval
**Parameter Type:** NUMBER
**Population Description:** All randomized participants who received at least one dose of study drug and had Geboes, MMS data at week 12.
**Reporting Status:** POSTED
**Time Frame:** Week 12
**Title:** Percentage of Participants Who Achieved Histologic-Endoscopic Mucosal Healing (HEMH)
**Type:** SECONDARY
**Unit of Measure:** percentage of participants
##### Group
**Description:** Participants received a subcutaneous injection of placebo every 2 weeks from weeks 0 to 12.
**ID:** OG000
**Title:** Placebo (Induction Treatment Period)
##### Group
**Description:** Participants received a subcutaneous injection of low dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** OG001
**Title:** Low Dose LY3471851 (Induction Treatment Period)
##### Group
**Description:** Participants received a subcutaneous injection of high dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** OG002
**Title:** High Dose LY3471851 (Induction Treatment Period)
#### Outcome Measure 9
**Description:** IBDQ is a 32-item questionnaire that measures four aspects of participants' lives: symptoms directly related to the primary bowel disturbance (10 items), systemic symptoms (5 items), emotional function (12 items), and social function (5 items). Responses are graded on a 7-point Likert scale, where 7 denotes "not a problem at all" and 1 denotes "a very severe problem." The responses are summed to produce a total score ranging from 32 to 224, with higher score indicating a better quality of life. LS Mean was calculated using ANCOVA (analysis of covariance) model with treatment, baseline value, previous advanced therapy failure status (yes/no), baseline corticosteroid use (yes/no), baseline disease activity (MMS: \[4 to 6\] or \[7 to 9\]) and region (North America/Europe/Other) as fixed factors.
**Dispersion Type:** Standard Error
**Parameter Type:** LEAST_SQUARES_MEAN
**Population Description:** All randomized participants who received at least one dose of study drug and had IBDQ data at baseline, week 12.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Week 12
**Title:** Change From Baseline in Inflammatory Bowel Disease Questionnaire (IBDQ) - Total Score
**Type:** SECONDARY
**Unit of Measure:** score on a scale
##### Group
**Description:** Participants received a subcutaneous injection of placebo every 2 weeks from weeks 0 to 12.
**ID:** OG000
**Title:** Placebo (Induction Treatment Period)
##### Group
**Description:** Participants received a subcutaneous injection of low dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** OG001
**Title:** Low Dose LY3471851 (Induction Treatment Period)
##### Group
**Description:** Participants received a subcutaneous injection of high dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** OG002
**Title:** High Dose LY3471851 (Induction Treatment Period)
#### Outcome Measure 10
**Description:** C-trough is the concentration of drug in the blood immediately before the next dose was administered.
**Dispersion Type:** Geometric Coefficient of Variation
**Parameter Type:** GEOMETRIC_MEAN
**Population Description:** All participants who received at least 1 dose of LY3471851 and had evaluable PK data.
**Reporting Status:** POSTED
**Time Frame:** Predose at week 12
**Title:** Pharmacokinetics (PK): Trough Concentration of LY3471851 (Ctrough) at Week 12
**Type:** SECONDARY
**Unit of Measure:** microgram per milliliter (µg/mL)
##### Group
**Description:** Participants received a subcutaneous injection of low dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** OG000
**Title:** Low Dose LY3471851 (Induction Treatment Period)
##### Group
**Description:** Participants received a subcutaneous injection of high dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** OG001
**Title:** High Dose LY3471851 (Induction Treatment Period)
### Participant Flow Module
#### Group
**Description:** Participants received a subcutaneous injection of high dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** FG000
**Title:** High Dose LY3471851 (Induction Treatment Period)
#### Group
**Description:** Participants received a subcutaneous injection of low dose LY3471851 every 2 weeks from weeks 0 to 12.
**ID:** FG001
**Title:** Low Dose LY3471851 (Induction Treatment Period)
#### Group
**Description:** Participants received a subcutaneous injection of placebo every 2 weeks from weeks 0 to 12.
**ID:** FG002
**Title:** Placebo (Induction Treatment Period)
#### Group
**Description:** Week 12 responders from the high dose LY3471851 induction treatment period arm entered the maintenance period and continued with the same treatment.
**ID:** FG003
**Title:** High Dose LY3471851 (Maintenance Treatment Period)
#### Group
**Description:** Week 12 responders from the low dose LY3471851 induction treatment period arm entered the maintenance period and continued with the same treatment.
**ID:** FG004
**Title:** Low Dose LY3471851 (Maintenance Treatment Period)
#### Group
**Description:** Week 12 responders from the placebo induction treatment period arm entered the maintenance period and continued with the same treatment.
**ID:** FG005
**Title:** Placebo (Maintenance Treatment Period)
#### Group
**Description:** Week 12 non-responders entered the extension period where they received subcutaneous injection of high dose LY3471851 every 2 weeks up to week 50. At week 26, extension period non-responders were discontinued from treatment.
**ID:** FG006
**Title:** High Dose LY3471851 (Extension Treatment Period)
#### Group
**Description:** Participants randomised to high dose LY3471851 arm entered follow-up period from induction or maintenance, or extension periods and were observed for 6 weeks for safety. No treatments were administered.
**ID:** FG007
**Title:** High Dose LY3471851 (Post-Treatment Follow-up Period)
#### Group
**Description:** Participants randomised to low dose LY3471851 arm entered follow-up period from induction or maintenance, or extension periods and were observed for 6 weeks for safety. No treatments were administered.
**ID:** FG008
**Title:** Low Dose LY3471851 (Post-Treatment Follow-up Period)
#### Group
**Description:** Participants randomised to placebo arm entered follow-up period from induction or maintenance, or extension periods and were observed for 6 weeks for safety. No treatments were administered.
**ID:** FG009
**Title:** Placebo (Post-Treatment Follow-up Period)
#### Period
**Title:** Induction Treatment Period
##### Withdraw
**Type:** Adverse Event
###### Reason
**Group ID:** FG000
**Number of Subjects:** 1
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
###### Reason
**Group ID:** FG002
**Number of Subjects:** 0
###### Reason
**Group ID:** FG003
**Number of Subjects:** 0
###### Reason
**Group ID:** FG004
**Number of Subjects:** 0
###### Reason
**Group ID:** FG005
**Number of Subjects:** 0
###### Reason
**Group ID:** FG006
**Number of Subjects:** 0
###### Reason
**Group ID:** FG007
**Number of Subjects:** 0
###### Reason
**Group ID:** FG008
**Number of Subjects:** 0
###### Reason
**Group ID:** FG009
**Number of Subjects:** 0
##### Withdraw
**Type:** Physician Decision
###### Reason
**Group ID:** FG000
**Number of Subjects:** 1
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
###### Reason
**Group ID:** FG002
**Number of Subjects:** 1
###### Reason
**Group ID:** FG003
**Number of Subjects:** 0
###### Reason
**Group ID:** FG004
**Number of Subjects:** 0
###### Reason
**Group ID:** FG005
**Number of Subjects:** 0
###### Reason
**Group ID:** FG006
**Number of Subjects:** 0
###### Reason
**Group ID:** FG007
**Number of Subjects:** 0
###### Reason
**Group ID:** FG008
**Number of Subjects:** 0
###### Reason
**Group ID:** FG009
**Number of Subjects:** 0
##### Withdraw
**Type:** Protocol Violation
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 1
###### Reason
**Group ID:** FG002
**Number of Subjects:** 0
###### Reason
**Group ID:** FG003
**Number of Subjects:** 0
###### Reason
**Group ID:** FG004
**Number of Subjects:** 0
###### Reason
**Group ID:** FG005
**Number of Subjects:** 0
###### Reason
**Group ID:** FG006
**Number of Subjects:** 0
###### Reason
**Group ID:** FG007
**Number of Subjects:** 0
###### Reason
**Group ID:** FG008
**Number of Subjects:** 0
###### Reason
**Group ID:** FG009
**Number of Subjects:** 0
##### Withdraw
**Type:** Withdrawal by Subject
###### Reason
**Group ID:** FG000
**Number of Subjects:** 4
###### Reason
**Group ID:** FG001
**Number of Subjects:** 1
###### Reason
**Group ID:** FG002
**Number of Subjects:** 1
###### Reason
**Group ID:** FG003
**Number of Subjects:** 0
###### Reason
**Group ID:** FG004
**Number of Subjects:** 0
###### Reason
**Group ID:** FG005
**Number of Subjects:** 0
###### Reason
**Group ID:** FG006
**Number of Subjects:** 0
###### Reason
**Group ID:** FG007
**Number of Subjects:** 0
###### Reason
**Group ID:** FG008
**Number of Subjects:** 0
###### Reason
**Group ID:** FG009
**Number of Subjects:** 0
##### Withdraw
**Type:** Study terminated by sponsor
###### Reason
**Group ID:** FG000
**Number of Subjects:** 7
###### Reason
**Group ID:** FG001
**Number of Subjects:** 11
###### Reason
**Group ID:** FG002
**Number of Subjects:** 0
###### Reason
**Group ID:** FG003
**Number of Subjects:** 0
###### Reason
**Group ID:** FG004
**Number of Subjects:** 0
###### Reason
**Group ID:** FG005
**Number of Subjects:** 0
###### Reason
**Group ID:** FG006
**Number of Subjects:** 0
###### Reason
**Group ID:** FG007
**Number of Subjects:** 0
###### Reason
**Group ID:** FG008
**Number of Subjects:** 0
###### Reason
**Group ID:** FG009
**Number of Subjects:** 0
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 32
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 35
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 14
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG004
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG005
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG006
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG007
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG008
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG009
**Number of Subjects:** 0
##### Milestone
**Type:** Received at Least One Dose of Study Drug
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 32
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 35
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 14
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG004
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG005
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG006
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG007
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG008
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG009
**Number of Subjects:** 0
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 19
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 22
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 12
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG004
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG005
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG006
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG007
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG008
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG009
**Number of Subjects:** 0
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 13
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 13
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 2
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG004
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG005
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG006
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG007
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG008
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG009
**Number of Subjects:** 0
#### Period
**Title:** Maintenance Treatment Period
##### Withdraw
**Type:** Lack of Efficacy
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
###### Reason
**Group ID:** FG002
**Number of Subjects:** 0
###### Reason
**Group ID:** FG003
**Number of Subjects:** 0
###### Reason
**Group ID:** FG004
**Number of Subjects:** 1
###### Reason
**Group ID:** FG005
**Number of Subjects:** 0
###### Reason
**Group ID:** FG006
**Number of Subjects:** 0
###### Reason
**Group ID:** FG007
**Number of Subjects:** 0
###### Reason
**Group ID:** FG008
**Number of Subjects:** 0
###### Reason
**Group ID:** FG009
**Number of Subjects:** 0
##### Withdraw
**Type:** Withdrawal by Subject
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
###### Reason
**Group ID:** FG002
**Number of Subjects:** 0
###### Reason
**Group ID:** FG003
**Number of Subjects:** 1
###### Reason
**Group ID:** FG004
**Number of Subjects:** 0
###### Reason
**Group ID:** FG005
**Number of Subjects:** 0
###### Reason
**Group ID:** FG006
**Number of Subjects:** 0
###### Reason
**Group ID:** FG007
**Number of Subjects:** 0
###### Reason
**Group ID:** FG008
**Number of Subjects:** 0
###### Reason
**Group ID:** FG009
**Number of Subjects:** 0
##### Withdraw
**Type:** Study terminated by sponsor
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
###### Reason
**Group ID:** FG002
**Number of Subjects:** 0
###### Reason
**Group ID:** FG003
**Number of Subjects:** 7
###### Reason
**Group ID:** FG004
**Number of Subjects:** 9
###### Reason
**Group ID:** FG005
**Number of Subjects:** 5
###### Reason
**Group ID:** FG006
**Number of Subjects:** 0
###### Reason
**Group ID:** FG007
**Number of Subjects:** 0
###### Reason
**Group ID:** FG008
**Number of Subjects:** 0
###### Reason
**Group ID:** FG009
**Number of Subjects:** 0
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 8
###### Achievement
**Group ID:** FG004
**Number of Subjects:** 10
###### Achievement
**Group ID:** FG005
**Number of Subjects:** 5
###### Achievement
**Group ID:** FG006
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG007
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG008
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG009
**Number of Subjects:** 0
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG004
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG005
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG006
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG007
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG008
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG009
**Number of Subjects:** 0
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 8
###### Achievement
**Group ID:** FG004
**Number of Subjects:** 10
###### Achievement
**Group ID:** FG005
**Number of Subjects:** 5
###### Achievement
**Group ID:** FG006
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG007
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG008
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG009
**Number of Subjects:** 0
#### Period
**Title:** Extension Treatment Period
##### Withdraw
**Type:** Lack of Efficacy
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
###### Reason
**Group ID:** FG002
**Number of Subjects:** 0
###### Reason
**Group ID:** FG003
**Number of Subjects:** 0
###### Reason
**Group ID:** FG004
**Number of Subjects:** 0
###### Reason
**Group ID:** FG005
**Number of Subjects:** 0
###### Reason
**Group ID:** FG006
**Number of Subjects:** 6
###### Reason
**Group ID:** FG007
**Number of Subjects:** 0
###### Reason
**Group ID:** FG008
**Number of Subjects:** 0
###### Reason
**Group ID:** FG009
**Number of Subjects:** 0
##### Withdraw
**Type:** Withdrawal by Subject
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
###### Reason
**Group ID:** FG002
**Number of Subjects:** 0
###### Reason
**Group ID:** FG003
**Number of Subjects:** 0
###### Reason
**Group ID:** FG004
**Number of Subjects:** 0
###### Reason
**Group ID:** FG005
**Number of Subjects:** 0
###### Reason
**Group ID:** FG006
**Number of Subjects:** 2
###### Reason
**Group ID:** FG007
**Number of Subjects:** 0
###### Reason
**Group ID:** FG008
**Number of Subjects:** 0
###### Reason
**Group ID:** FG009
**Number of Subjects:** 0
##### Withdraw
**Type:** Study terminated by sponsor
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
###### Reason
**Group ID:** FG002
**Number of Subjects:** 0
###### Reason
**Group ID:** FG003
**Number of Subjects:** 0
###### Reason
**Group ID:** FG004
**Number of Subjects:** 0
###### Reason
**Group ID:** FG005
**Number of Subjects:** 0
###### Reason
**Group ID:** FG006
**Number of Subjects:** 18
###### Reason
**Group ID:** FG007
**Number of Subjects:** 0
###### Reason
**Group ID:** FG008
**Number of Subjects:** 0
###### Reason
**Group ID:** FG009
**Number of Subjects:** 0
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG004
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG005
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG006
**Number of Subjects:** 26
###### Achievement
**Group ID:** FG007
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG008
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG009
**Number of Subjects:** 0
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG004
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG005
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG006
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG007
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG008
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG009
**Number of Subjects:** 0
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG004
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG005
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG006
**Number of Subjects:** 26
###### Achievement
**Group ID:** FG007
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG008
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG009
**Number of Subjects:** 0
#### Period
**Title:** Post-Treatment Follow-up Period
##### Withdraw
**Type:** Adverse Event
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
###### Reason
**Group ID:** FG002
**Number of Subjects:** 0
###### Reason
**Group ID:** FG003
**Number of Subjects:** 0
###### Reason
**Group ID:** FG004
**Number of Subjects:** 0
###### Reason
**Group ID:** FG005
**Number of Subjects:** 0
###### Reason
**Group ID:** FG006
**Number of Subjects:** 0
###### Reason
**Group ID:** FG007
**Number of Subjects:** 1
###### Reason
**Group ID:** FG008
**Number of Subjects:** 0
###### Reason
**Group ID:** FG009
**Number of Subjects:** 0
##### Withdraw
**Type:** Lack of Efficacy
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
###### Reason
**Group ID:** FG002
**Number of Subjects:** 0
###### Reason
**Group ID:** FG003
**Number of Subjects:** 0
###### Reason
**Group ID:** FG004
**Number of Subjects:** 0
###### Reason
**Group ID:** FG005
**Number of Subjects:** 0
###### Reason
**Group ID:** FG006
**Number of Subjects:** 0
###### Reason
**Group ID:** FG007
**Number of Subjects:** 2
###### Reason
**Group ID:** FG008
**Number of Subjects:** 3
###### Reason
**Group ID:** FG009
**Number of Subjects:** 0
##### Withdraw
**Type:** Lost to Follow-up
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
###### Reason
**Group ID:** FG002
**Number of Subjects:** 0
###### Reason
**Group ID:** FG003
**Number of Subjects:** 0
###### Reason
**Group ID:** FG004
**Number of Subjects:** 0
###### Reason
**Group ID:** FG005
**Number of Subjects:** 0
###### Reason
**Group ID:** FG006
**Number of Subjects:** 0
###### Reason
**Group ID:** FG007
**Number of Subjects:** 1
###### Reason
**Group ID:** FG008
**Number of Subjects:** 0
###### Reason
**Group ID:** FG009
**Number of Subjects:** 0
##### Withdraw
**Type:** Withdrawal by Subject
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
###### Reason
**Group ID:** FG002
**Number of Subjects:** 0
###### Reason
**Group ID:** FG003
**Number of Subjects:** 0
###### Reason
**Group ID:** FG004
**Number of Subjects:** 0
###### Reason
**Group ID:** FG005
**Number of Subjects:** 0
###### Reason
**Group ID:** FG006
**Number of Subjects:** 0
###### Reason
**Group ID:** FG007
**Number of Subjects:** 3
###### Reason
**Group ID:** FG008
**Number of Subjects:** 1
###### Reason
**Group ID:** FG009
**Number of Subjects:** 0
##### Withdraw
**Type:** Study terminated by sponsor
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
###### Reason
**Group ID:** FG002
**Number of Subjects:** 0
###### Reason
**Group ID:** FG003
**Number of Subjects:** 0
###### Reason
**Group ID:** FG004
**Number of Subjects:** 0
###### Reason
**Group ID:** FG005
**Number of Subjects:** 0
###### Reason
**Group ID:** FG006
**Number of Subjects:** 0
###### Reason
**Group ID:** FG007
**Number of Subjects:** 18
###### Reason
**Group ID:** FG008
**Number of Subjects:** 26
###### Reason
**Group ID:** FG009
**Number of Subjects:** 11
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG004
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG005
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG006
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG007
**Number of Subjects:** 25
###### Achievement
**Group ID:** FG008
**Number of Subjects:** 30
###### Achievement
**Group ID:** FG009
**Number of Subjects:** 11
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG004
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG005
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG006
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG007
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG008
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG009
**Number of Subjects:** 0
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG002
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG003
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG004
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG005
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG006
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG007
**Number of Subjects:** 25
###### Achievement
**Group ID:** FG008
**Number of Subjects:** 30
###### Achievement
**Group ID:** FG009
**Number of Subjects:** 11
**Pre-Assignment Details:** * (ii) Week 12 non-responders enter the extension period where they received high dose LY3471851. At week 26, those who responded to treatment continued with the same treatment, while non-responders discontinued and entered follow-up.
* a 6-week post-treatment follow-up period: Following treatment completion or discontinuation, participants entered the follow-up period and were observed for safety. No treatments were administered.
**Recruitment Details:** The study consisted of:
* a 12-week induction treatment period: participants randomly received either high dose LY3471851 or low dose LY3471851 or placebo.
* a 40-week maintenance/extension treatment period (final dose at week 50 and study assessments at week 52)
* (i) Week 12 responders enter the maintenance period where they continued to receive the same treatment to which they were randomly assigned.
(Continued..)
**Has Results:** True |
## Protocol Section
### Identification Module
**NCT ID:** NCT04050579
**Brief Title:** OPIE in the Thin Interventricular Septum
**Official Title:** A Single Center Trial of On-pump Intracardiac Echocardiography (OPIE) in the Thin Interventricular Septum
#### Organization Study ID Info
**ID:** s19-01180
#### Organization
**Class:** OTHER
**Full Name:** NYU Langone Health
### Status Module
#### Completion Date
**Date:** 2025-05-30
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2023-08-01
**Type:** ACTUAL
**Last Update Submit Date:** 2023-07-27
**Overall Status:** ENROLLING_BY_INVITATION
#### Primary Completion Date
**Date:** 2024-12-31
**Type:** ESTIMATED
#### Start Date
**Date:** 2020-09-15
**Type:** ACTUAL
**Status Verified Date:** 2023-07
#### Study First Post Date
**Date:** 2019-08-08
**Type:** ACTUAL
**Study First Submit Date:** 2019-08-06
**Study First Submit QC Date:** 2019-08-07
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** NYU Langone Health
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** True
**Is FDA Regulated Drug:** False
**Is US Export:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** This is a follow up investigation to our previous study entitled "On-pump intraoperative echocardiography (OPIE)" (clinicaltrials.gov NCT03094325) whereby we determined that left ventricular septal thickness as measured by the OPIE technique correlates highly with traditional methods of transthoracic and transesophageal echocardiography during septal myectomy for hypertrophic cardiomyopathy. OPIE may be especially useful in patients with a thin ventricular septal thickness as adequate treatment may rely on mere millimeters of myocardial resection. We therefore propose a study in which OPIE is compared to transthoracic and transesophageal echocardiography in patients with a thin interventricular septum. Subjects will receive the same perioperative care regardless of their involvement in the study. Patients who enroll in the study will undergo an additional intraoperative echocardiographic measurement that adds less than five minutes to total operative time.
### Conditions Module
**Conditions:**
- Hypertrophic Cardiomyopathy
### Design Module
#### Design Info
**Allocation:** NA
**Intervention Model:** SINGLE_GROUP
##### Masking Info
**Masking:** NONE
**Primary Purpose:** DEVICE_FEASIBILITY
#### Enrollment Info
**Count:** 20
**Type:** ESTIMATED
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** The Principal Investigator use the OPIE probe to measure the anterior basilar septal thickness. Myectomy will be performed and OPIE will be repeated.
**Intervention Names:**
- Device: on-pump intracardiac echocardiography
**Label:** OPIE in thin inverventricular septum
**Type:** OTHER
### Interventions
#### Intervention 1
**Arm Group Labels:**
- OPIE in thin inverventricular septum
**Description:** Subjects will be patients with hypertrophic cardiomyopathy with interventricular septae less than 2.0cm who are undergoing septal myectomy. The patients will have already had a number of preoperative transthoracic echocardiographs as part of their normal hypertrophic cardiomyopathy care. Before cardiopulmonary bypass, the basal anterior septal thickness will be measured by transesophageal echocardiograph as is performed in all septal myectomy procedures. The Principal Investigator will then use the OPIE probe to measure the anterior basilar septal thickness. Myectomy will be performed and OPIE will be repeated. The post-myectomy anterior basilar septal thickness will again be measured by transesophageal echocardiography.
**Name:** on-pump intracardiac echocardiography
**Type:** DEVICE
### Outcomes Module
#### Primary Outcomes
**Description:** correlation between OPIE and traditional imaging methods
**Measure:** correlations
**Time Frame:** at the conclusion of all data collection; approximately 6 to 12 months
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* All hypertrophic cardiomyopathy patients, inclusive of male and female and all racial/ethnic origins, age 18 or older who are scheduled to receive a septal myectomy with a preoperative transthoracic echocardiograph suggesting an interventricular septal width of less than 2.0 cm.
Exclusion Criteria:
* Those hypertrophic cardiomyopathy patients who are scheduled to receive a septal myectomy who are less than 18 years old or whose preoperative transthoracic echocardiograph suggests an interventricular septal width of greater than 2.0 cm.
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** New York
**Country:** United States
**Facility:** NYU Langone Health
**State:** New York
**Zip:** 10016
#### Overall Officials
**Official 1:**
**Affiliation:** NYU Langone Health
**Name:** Daniel Swistel, MD
**Role:** PRINCIPAL_INVESTIGATOR
### IPD Sharing Statement Module
**Description:** The de-identified participant data from the final research dataset used in the published manuscript will be shared upon reasonable request beginning 9 months and ending 36 months following article publication or as required by a condition of awards and agreements supporting the research provided the investigator who proposes to use the data executes a data use agreement with New York University Langone Health. Requests may be directed to: [email protected]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research.
**Info Types:**
- STUDY_PROTOCOL
- SAP
**IPD Sharing:** YES
**Time Frame:** Beginning 9 months and ending 36 months following article publication or as required by a condition of awards and agreements supporting the research.
### References Module
#### References
**Citation:** Williams DM, Nampi RG, Saric M, Grossi EA, Sherrid MV, Swistel DG. A novel imaging modality to guide septal myectomy for hypertrophic cardiomyopathy. Podium presentation at the Western Thoracic Surgical Society Association Annual Meeting. Squaw Valley, CA. June 28, 2019.
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000006331
- Term: Heart Diseases
- ID: D000002318
- Term: Cardiovascular Diseases
- ID: D000001020
- Term: Aortic Stenosis, Subvalvular
- ID: D000001024
- Term: Aortic Valve Stenosis
- ID: D000082862
- Term: Aortic Valve Disease
- ID: D000006349
- Term: Heart Valve Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC14
- Name: Heart and Blood Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M12154
- Name: Cardiomyopathies
- Relevance: HIGH
- As Found: Cardiomyopathy
- ID: M10035
- Name: Hypertrophy
- Relevance: LOW
- As Found: Unknown
- ID: M5568
- Name: Cardiomyopathy, Hypertrophic
- Relevance: HIGH
- As Found: Hypertrophic Cardiomyopathy
- ID: M9419
- Name: Heart Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M6475
- Name: Constriction, Pathologic
- Relevance: LOW
- As Found: Unknown
- ID: M4340
- Name: Aortic Valve Stenosis
- Relevance: LOW
- As Found: Unknown
- ID: M2379
- Name: Aortic Valve Disease
- Relevance: LOW
- As Found: Unknown
- ID: M9437
- Name: Heart Valve Diseases
- Relevance: LOW
- As Found: Unknown
- ID: T449
- Name: Aortic Valve Stenosis
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000009202
- Term: Cardiomyopathies
- ID: D000002312
- Term: Cardiomyopathy, Hypertrophic
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT04619979
**Brief Title:** Preoperative Anxiety on Postoperative Outcome and Sleep Quality in Patients Undergoing Laparoscopic Hysterectomy
**Official Title:** Effects of Preoperative Anxiety on Postoperative Outcome and Sleep Quality in Patients Undergoing Laparoscopic Hysterectomy
#### Organization Study ID Info
**ID:** Preoperative anxiety and sleep
#### Organization
**Class:** OTHER
**Full Name:** Shengjing Hospital
### Status Module
#### Completion Date
**Date:** 2022-08-10
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2022-08-17
**Type:** ACTUAL
**Last Update Submit Date:** 2022-08-13
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2022-03-01
**Type:** ACTUAL
#### Start Date
**Date:** 2021-10-01
**Type:** ACTUAL
**Status Verified Date:** 2022-08
#### Study First Post Date
**Date:** 2020-11-06
**Type:** ACTUAL
**Study First Submit Date:** 2020-11-02
**Study First Submit QC Date:** 2020-11-02
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Shengjing Hospital
#### Responsible Party
**Investigator Affiliation:** Shengjing Hospital
**Investigator Full Name:** Yanchao Yang
**Investigator Title:** principal investigator
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
### Description Module
**Brief Summary:** Sleep is a naturally occurring state of decreased arousal that is crucial for normal immune and cognitive function. Although surgery and anesthesia techniques have improved in recent years, sleep function and sleep cycles may still be altered perioperatively by surgery and other interventions under general anesthesia.Postoperative sleep fragmentation and poor sleep quality not only lead to hyperalgesia and delayed postoperative recovery, but can increase the risk of potential adverse effects, such as cognitive impairment, chronic pain and emotional disturbances, metabolic disorders, and pro-inflammatory changes. General anesthesia is a medically induced state of hyporesponsiveness that resembles natural sleep. Studies have shown that general anesthesia can lead to circadian rhythm time structure dyssynchrony, resulting in postoperative sleep disturbance, characterized by decreases in rapid eye movement (REM) and slow wave sleep (SWS). Previous studies have also reported that age, preoperative comorbidities, and severity of surgical trauma are independent factors associated with postoperative sleep disturbance. In addition, anxiety is an unpleasant sensation that compromises patients' comfort and well-being. A study by Ruis et al. estimated that 25-80% of patients admitted for surgery experienced preoperative anxiety, including fear of surgery and anesthesia-related fears. Furthermore, preoperative anxiety was recognized as a potential and preventable risk factor for severe postoperative pain and postoperative complications such as increased postoperative morbidity and mortality. Given that several prior studies have reported that preoperative anxiety has an effect on postoperative sleep quality in patients undergoing gynecological surgery, this study aimed to investigate the effect of preoperative anxiety on postoperative outcomes and sleep quality in patients undergoing gynecological surgery. Studying these results could enable us to better manage patients during the perioperative period to promote their postoperative recovery.
### Conditions Module
**Conditions:**
- General Anesthesia
- Postoperative Pain
- Postoperative Sleep Quality
- Preoperative Anxiety
### Design Module
#### Design Info
**Observational Model:** CASE_CONTROL
**Time Perspective:** PROSPECTIVE
#### Enrollment Info
**Count:** 356
**Type:** ACTUAL
**Study Type:** OBSERVATIONAL
### Arms Interventions Module
#### Arm Group 1
**Intervention Names:**
- Procedure: patients undergoing gynecological surgery under general anesthesia
**Label:** preoperative anxiety group
#### Arm Group 2
**Intervention Names:**
- Procedure: patients undergoing gynecological surgery under general anesthesia
**Label:** Non-preoperative anxiety group
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Non-preoperative anxiety group
- preoperative anxiety group
**Description:** patients undergoing gynecological surgery under general anesthesia
**Name:** patients undergoing gynecological surgery under general anesthesia
**Type:** PROCEDURE
### Outcomes Module
#### Primary Outcomes
**Description:** evaluate Numerical Rating Scale score (0: no pain to 10: severe pain)
**Measure:** postoperative pain
**Time Frame:** 24 hours after surgery
**Description:** evaluate postoperative sleep by using Athens insomnia scale(\<4: no insomnia; 4-6: suspicious insomnia; \>6: insomnia)
**Measure:** evaluate postoperative sleep quality by using Athens insomnia scale
**Time Frame:** first night before surgery
**Description:** evaluate postoperative sleep by using Athens insomnia scale (\<4: no insomnia; 4-6: suspicious insomnia; \>6: insomnia)
**Measure:** evaluate postoperative sleep quality by using Athens insomnia scale
**Time Frame:** first night after surgery
**Description:** evaluate postoperative sleep by using Athens insomnia scale (\<4: no insomnia; 4-6: suspicious insomnia; \>6: insomnia)
**Measure:** evaluate postoperative sleep quality by using Athens insomnia scale
**Time Frame:** third night after surgery
**Description:** evaluate the preoperative anxiety score before the surgery, APAIS contains 6 items rated on a five-point Likert scale, which represents two scales: anxiety (items 1, 2, 4, and 5) and need for information (items 3 and 6) Accordingly, the maximal score of the entire APAIS (APAIS-T) is 30 and the one expressing the patient's need for information (APAIS-I) is 10. The maximal score of the two items concerning anxiety about anesthesia (APAIS-A-An) and surgery (APAIS-A-Su) is also 10 each, resulting in a maximal score of 20 for total preoperative anxiety (APAIS-A-T). And APAIS-A-T \> 10 was used as a cut-off to define patients with high anxiety, the higher the score, the more serious the pre-operative anxiety is
**Measure:** preoperative anxiety score assessed by the Amsterdam preoperative anxiety and information scale (APAIS)".
**Time Frame:** baseline (before the surgery)
### Eligibility Module
**Eligibility Criteria:** The inclusion criteria were:
1. age between 18 and 75 years,
2. American Society of Anesthesiologists (ASA) medical status I or II,
3. laparoscopic hysterectomy, elective operation and surgery lasting 1-3 h.
The exclusion criteria included
1. cardiovascular disease,
2. chronic use of analgesics,
3. chronic use of antidepressants,
4. use of sleep-promoting drugs,
5. sleep disorders,
6. sleep apnea syndrome,
7. history of abnormal surgery or recovery from anesthesia,
8. psychosis,
9. patients with impaired verbal communication,
10. unwillingness to provide informed consent.
**Maximum Age:** 75 Years
**Minimum Age:** 18 Years
**Sampling Method:** PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
**Study Population:** This study enrolled patients undergoing gynecological surgery under general anesthesia at Shengjing Hospital of China Medical University.
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Shenyang
**Country:** China
**Facility:** Shengjing Hospital
**State:** Liaoning
**Zip:** 110004
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000001523
- Term: Mental Disorders
### Condition Browse Module - Browse Branches
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: All
- Name: All Conditions
- Abbrev: BXM
- Name: Behaviors and Mental Disorders
### Condition Browse Module - Browse Leaves
- ID: M13069
- Name: Pain, Postoperative
- Relevance: LOW
- As Found: Unknown
- ID: M4324
- Name: Anxiety Disorders
- Relevance: HIGH
- As Found: Anxiety
- ID: M13066
- Name: Pain
- Relevance: LOW
- As Found: Unknown
- ID: M4815
- Name: Mental Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M14473
- Name: Psychotic Disorders
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000001008
- Term: Anxiety Disorders
### Intervention Browse Module - Ancestors
- ID: D000002492
- Term: Central Nervous System Depressants
- ID: D000045505
- Term: Physiological Effects of Drugs
### Intervention Browse Module - Browse Branches
- Abbrev: CNSDep
- Name: Central Nervous System Depressants
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M4107
- Name: Anesthetics
- Relevance: HIGH
- As Found: Imaging
### Intervention Browse Module - Meshes
- ID: D000000777
- Term: Anesthetics
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT05643079
**Brief Title:** Medial Displacement Calcaneal Osteotomy and FDL- Transfer - With a Human, Allogeneic Cortical Bone Screw
**Official Title:** Medial Displacement Calcaneal Osteotomy and FDL- Transfer - a Prospective Comparative Study Between Metal/Bio-Tenodesis Screw (Arthrex) and the Human, Allogeneic Cortical Bone Screw (Shark Screw®️, Surgebright-GmbH)
#### Organization Study ID Info
**ID:** FDLTransferSharkScrew
#### Organization
**Class:** OTHER
**Full Name:** Orthopedic Hospital Vienna Speising
### Status Module
#### Completion Date
**Date:** 2028-02
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2023-03-27
**Type:** ACTUAL
**Last Update Submit Date:** 2023-03-23
**Overall Status:** RECRUITING
#### Primary Completion Date
**Date:** 2027-02
**Type:** ESTIMATED
#### Start Date
**Date:** 2022-02-10
**Type:** ACTUAL
**Status Verified Date:** 2023-03
#### Study First Post Date
**Date:** 2022-12-08
**Type:** ACTUAL
**Study First Submit Date:** 2022-10-21
**Study First Submit QC Date:** 2022-12-01
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Orthopedic Hospital Vienna Speising
#### Responsible Party
**Investigator Affiliation:** Orthopedic Hospital Vienna Speising
**Investigator Full Name:** Forian Wenzel-Schwarz
**Investigator Title:** Senior Physician Dr. med.
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
### Description Module
**Brief Summary:** The goal of this observational study is to compare the use of a screw made of human bone (Shark-Screw®, Surgebright-GmbH) with the metal/Bio-Tenodesis screw (Arthrex) in the treatment of the symptomatic flatfoot using the medializing calcaneus osteotomy with flexor digitorum longus transfer (FDL) in adult patients. The advantage of the human bone screw is that after surgery no hardware removal is necessary. The screw is transformed from the body to normal bone.
The main questions it aims to answer are:
* Can the human bone screw achieve union rates like the metal/Bio-Tenodesis screw?
* Is the time to union similar between the different screws?
* Is the complication rate similar between the different screws?
* Are the activity scores American Orthopaedic Foot and Ankle Society (AOFAS), Foot and Ankle Outcome Score (FAOS) and Foot Function Index (FFI) after surgery similar in the compared patient groups?
Participants will have
* the surgery
* follow-ups at 6 weeks, 6 months, 1 and 2 years.
* X-rays are performed at each follow up.
* CT-scans are performed after 6 months.
* activity scores are collected at the follow up after 6 months, 1 year and 2 years.
**Detailed Description:** Symptomatic flatfoot deformity in adults often occurs as a result of dysfunction of the Tibialis posterior tendon (TPTD), with a prevalence of 3.3%. Structured non-surgical treatment programs with orthotics and physiotherapy can achieve a high level of long-term subjective and functional satisfaction, with surgery being avoided in 70-89% of cases. Surgical intervention is indicated for progressive or uncontrolled symptoms. Flexible flatfoot deformity, stage II classified by Johnson and Strom, can be treated with a joint-preserving strategy, which usually includes a medializing calcaneal osteotomy, debridement of the tibialis posterior tendon, and transfer of the flexor digitorum longus (FDL) tendon. 87% satisfaction after ten years was observed in both pain relief and foot function and alignment.
Metal screws have been used for decades to treat bone fractures. The removal is the major disadvantage of conventional osteosyntheses and requires a second operation, with all the resulting risks of complications for each patient. The use of the bone screw from allogeneic cortical bone would reduce costs substantially, with a significant reduction in the average complication rate to 0.3%.
The idea of stabilizing fractures using compact bone instead of metal is not new. Obwegeser published the clinical use of 796 screw implants of allogeneic bone and reported that the only complication was the fracture of 6 screws (\<1%) and three screw loosening (\<0,5%).
Since 2016, the human, allogeneic cortical bone screw(Shark Screw®) has been used by two tissue banks, the Austrian Austrian tissue bank Surgebright-GmbH and the German Institute for Cell and Tissue Replacement (DIZG). The Shark Screw® graft was approved by the competent Austrian authority (AGES) in 2016. The bone graft immediately creates a solid, purely bony connection. This leads to bone remodeling, bone incorporation and optimal reparation in the surgical area. Depending on the loads and requirements, the bone connection adapts for the future. The bone grafts undergo a certified sterilization procedure at the DIZG, which was developed in 1985 at the Berlin Charité.
Due to the lack of further systematic investigations to objectively confirm the theoretically given and subjectively experienced product advantages, this observational study is now being conducted.
The aim of the present study is to evaluate the application of the human allogeneic cortical bone screw (Shark Screw®) and the metal/Bio-Tenodesis screw in the surgical treatment (medializing calcaneus osteotomy with FDL transfer) of symptomatic flatfoot and to systematically document corresponding clinical and radiological parameters before and after surgery.
### Conditions Module
**Conditions:**
- Flat Feet
- Flexor Tendon Rupture
- Tibialis Posterior Dysfunction
- Deformity, Foot
**Keywords:**
- human allogeneic cortical bone screw
- Shark Screw®
- flat foot deformity
- FDL Transfer
- Tibialis posterior dysfunction
- metal screw
- Bio-Tenodesis screw
- flat foot
### Design Module
#### Design Info
**Observational Model:** CASE_CONTROL
**Time Perspective:** PROSPECTIVE
#### Enrollment Info
**Count:** 40
**Type:** ESTIMATED
**Study Type:** OBSERVATIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** medializing calcaneal osteotomy, debridement of the tibialis posterior tendon, and/or transfer of the flexor digitorum longus (FDL) tendon with the following screws:
Metal-/Bio-Tenodesis screw (Arthrex, Naples, Florida, USA)
Metal-Screw:
ø 6,7 mm length: 40-60 mm
Bio-Tenodesis screw:
ø 4,00 mm, length: 10 mm ø 4,75 mm, length: 15 mm ø 5,50 mm, length: 15 mm
**Intervention Names:**
- Procedure: medializing calcaneal osteotomy and/or transfer of the flexor digitorum longus tendon (FDL) with metal/Bio-Tenodesis screws
**Label:** metal/Bio-Tenodesis group
#### Arm Group 2
**Description:** medializing calcaneal osteotomy, debridement of the tibialis posterior tendon, and/or transfer of the flexor digitorum longus (FDL) tendon with the following screws:
Shark Screw® (Surgebright-GmbH, 4040 Lichtenberg, Austria)
Versions used:
Shark Screw® diver ø: 5,0 mm, length: 35 mm Shark Screw® diver ø: 5,0 mm, length: 45 mm Shark Screw® tendon ø: 5,0 mm, length: 15 mm
**Intervention Names:**
- Procedure: medializing calcaneal osteotomy and/or transfer of the flexor digitorum longus tendon (FDL) with Shark Screws®
**Label:** human, allogeneic cortical bone screw (Shark Screw®)
### Interventions
#### Intervention 1
**Arm Group Labels:**
- metal/Bio-Tenodesis group
**Description:** The calcaneus osteotomy is performed dorsal proximal to plantar distal with caution of the peroneal tendons and sural nerve. After mobilization the dorsal fragment is displaced medially by\~10mm. Thereafter, a guide wire is placed from plantar-lateral into the ventral portion of the calcaneus under fluoroscopic control. After stab incision and length measurement, the osteotomy is fixed with a Metal/Bio-Tenodesis screw(MBS). The lateral projection of the edge is straightened. Opening of the tendon sheath of the FDL muscle and dissection distally to Henry's node. Settling of the tendon and arming with a "shuttle suture". Tendon diameter measurement. Place a guide wire at os naviculare directed from plantar to dorsal in 20°proximal guidance. Pull through the FDL tendon from plantar to dorsal and fixation with an MBS in 20°of pointed foot position and inversion with appropriate desired tension. The tendon is then sutured to the stump of the tibialis posterior tendon, retinaculum sutures.
**Name:** medializing calcaneal osteotomy and/or transfer of the flexor digitorum longus tendon (FDL) with metal/Bio-Tenodesis screws
**Other Names:**
- Metal/Bio-Tenodesis screw intervention
**Type:** PROCEDURE
#### Intervention 2
**Arm Group Labels:**
- human, allogeneic cortical bone screw (Shark Screw®)
**Description:** The calcaneus osteotomy is performed dorsal proximal to plantar distal with caution of the peroneal tendons and sural nerve. After mobilization the dorsal fragment is displaced medially by approx.10mm. Thereafter, a guide wire is placed from plantar-lateral into the ventral portion of the calcaneus under fluoroscopic control. After stab incision and length measurement, the osteotomy is fixed with a Shark Screw®. The lateral projection of the edge is straightened. Opening of the tendon sheath of the FDL muscle and dissection distally to Henry's node. Settling of the tendon and arming with a "shuttle suture". Tendon diameter measurement. Place a guide wire at os naviculare directed from plantar to dorsal in 20°proximal guidance. Pull through the FDL tendon from plantar to dorsal and fixation with the Shark Screw® in 20°of pointed foot position and inversion with appropriate desired tension. The tendon is then sutured to the stump of the tibialis posterior tendon, retinaculum sutures.
**Name:** medializing calcaneal osteotomy and/or transfer of the flexor digitorum longus tendon (FDL) with Shark Screws®
**Other Names:**
- Shark Screw® intervention
**Type:** PROCEDURE
### Outcomes Module
#### Primary Outcomes
**Description:** bony union will be evaluated
**Measure:** x-ray qualitative evaluation of changes in bony union of calcaneus osteotomy
**Time Frame:** 6 weeks, 6 months, 1 year, 2 years
**Description:** bony union will be evaluated
**Measure:** CT-scan qualitative evaluation of changes in bony union of calcaneus osteotomy
**Time Frame:** 6 months, 1 year
#### Secondary Outcomes
**Description:** The American Orthopedic Foot and Ankle Score (AOFAS) units in percent (%), the higher the better
**Measure:** changes in AOFAS score in comparison to presurgery
**Time Frame:** 6 months, 1 year and 2 years
**Description:** Foot and Ankle Outcome Score units in percent (%), the higher the better
**Measure:** changes in Foot and Ankle Outcome Score (FOAS) score in comparison to presurgery
**Time Frame:** 6 months, 1 year and 2 years
**Description:** Foot Function Index units in percent (%), the higher the better
**Measure:** changes in FFI score in comparison to presurgery
**Time Frame:** 6 months, 1 year and 2 years
**Description:** Visual Analog Scale Pain Score values between 0 and 10, 0= no pain, 10 sever pain
**Measure:** changes in Vas-Pain score in comparison to presurgery
**Time Frame:** 6 weeks, 6 months, 1 year and 2 years
**Description:** Complications will be recorded during surgery and the kind of complication described, number of patients with complications are given
**Measure:** complications
**Time Frame:** during surgery, 6 weeks, 6 months, 1 year and 2 years
**Description:** Reoperations will be recorded when needed and the kind of re-operation and number of patients with reoperations are given
**Measure:** reoperations
**Time Frame:** during surgery, 6 weeks, 6 months, 1 year and 2 years
**Description:** Pseudarthrosis will be recorded at the follow up at 2 years and number of patients with Pseudarthrosis are given
**Measure:** pseudoarthrosis
**Time Frame:** during surgery, 6 months, 1 year and 2 years
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria
* Indication for the use of a metal/Bio-Tenodesis screw or human bone screw in medializing calcaneus osteotomy with FDL transfer.
* BMI\< 40 kg/m²
Exclusion Criteria:
* Insufficient knowledge of German
* Alcohol and drug abuse
* Pregnant woman or nursing mother
* Foreseeable compliance problems
* Neoplastic diseases, malignant bone tumors, rheumatoid arthritis
* Active osteomyelitis
* History of foot surgery
* Advanced osteoarthritis of the lower ankle joint
* Ulcerations in the skin of the surgical area
* Immunosuppressive medications that cannot be discontinued
* BMI \>40
**Minimum Age:** 18 Years
**Sampling Method:** NON_PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
**Study Population:** adult patients with an indication for the use of a metal/Bio-Tenodesis screw or human bone screw in medializing calcaneus osteotomy with/without FDL transfer.
The patient has the choice of the treatment method after explanation advantages and disadvantages for each surgery method
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Florian Wenzel-Schwarz, MD
**Phone:** +43180182
**Phone Ext:** 3081
**Role:** CONTACT
#### Locations
**Location 1:**
**City:** Vienna
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Florian Wenzel-Schwarz, MD
- **Phone:** +43180182
- **Phone Ext:** 3081
- **Role:** CONTACT
***Contact 2:***
- **Name:** Florian Wenzel-Schwarz, MD
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** Austria
**Facility:** Abteilung für Kinderorthopädie und Fußchirurgie Orthopädisches Spital Speising
**Status:** RECRUITING
**Zip:** 1130
#### Overall Officials
**Official 1:**
**Affiliation:** Abteilung für Kinderorthopädie und Fußchirurgie Orthopädisches Spital Speising, Vienna, Austria
**Name:** Florian Wenzel-Schwarz, MD
**Role:** PRINCIPAL_INVESTIGATOR
### IPD Sharing Statement Module
**Description:** due to the sensibility of the row data individual data will only be available on request
**IPD Sharing:** NO
### References Module
#### References
**Citation:** Pastl K, Pastl E, Flory D, Borchert GH, Chraim M. Arthrodesis and Defect Bridging of the Upper Ankle Joint with Allograft Bone Chips and Allograft Cortical Bone Screws (Shark Screw(R)) after Removal of the Salto-Prosthesis in a Multimorbidity Patient: A Case Report. Life (Basel). 2022 Jul 11;12(7):1028. doi: 10.3390/life12071028.
**PMID:** 35888116
**Citation:** Pastl K, Schimetta W. The application of an allogeneic bone screw for osteosynthesis in hand and foot surgery: a case series. Arch Orthop Trauma Surg. 2022 Oct;142(10):2567-2575. doi: 10.1007/s00402-021-03880-6. Epub 2021 Apr 8.
**PMID:** 33834287
**Citation:** Brcic I, Pastl K, Plank H, Igrec J, Schanda JE, Pastl E, Werner M. Incorporation of an Allogenic Cortical Bone Graft Following Arthrodesis of the First Metatarsophalangeal Joint in a Patient with Hallux Rigidus. Life (Basel). 2021 May 24;11(6):473. doi: 10.3390/life11060473.
**PMID:** 34073841
**Citation:** Johnson KA, Strom DE. Tibialis posterior tendon dysfunction. Clin Orthop Relat Res. 1989 Feb;(239):196-206.
**PMID:** 2912622
**Citation:** Obwegeser JA. [Absorbable and bioconvertible osteosynthesis materials in maxillofacial surgery]. Mund Kiefer Gesichtschir. 1998 Nov;2(6):288-308. doi: 10.1007/s100060050077. German.
**PMID:** 9880999
**Citation:** Hanslik-Schnabel B, Flory D, Borchert GH, Schanda JE. Clinical and Radiologic Outcome of First Metatarsophalangeal Joint Arthrodesis Using a Human Allogeneic Cortical Bone Screw. Foot Ankle Orthop. 2022 Jul 29;7(3):24730114221112944. doi: 10.1177/24730114221112944. eCollection 2022 Jul.
**PMID:** 35924004
**Citation:** Amann P, Pastl K, Neunteufel E, Bock P. Clinical and Radiologic Results of a Human Bone Graft Screw in Tarsometatarsal II/+III Arthrodesis. Foot Ankle Int. 2022 Jul;43(7):913-922. doi: 10.1177/10711007221081533. Epub 2022 Apr 2.
**PMID:** 35373594
**Citation:** Sailer S, Lechner S, Flossmann A, Wanzel M, Habeler K, Krasny C, Borchert GH. Treatment of scaphoid fractures and pseudarthroses with the human allogeneic cortical bone screw. A multicentric retrospective study. J Orthop Traumatol. 2023 Feb 10;24(1):6. doi: 10.1186/s10195-023-00686-7.
**PMID:** 36765020
**Citation:** Krasny C, Radda C, Polke R, Schallmayer D, Borchert GH, Albrecht C. A human, allogeneic cortical bone screw for distal interphalangeal joint (DIP) arthrodesis: a retrospective cohort study with at least 10 months follow-up. Arch Orthop Trauma Surg. 2023 Jul;143(7):4557-4564. doi: 10.1007/s00402-023-04785-2. Epub 2023 Feb 9.
**PMID:** 36757467
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000014947
- Term: Wounds and Injuries
- ID: D000070558
- Term: Talipes
- ID: D000005531
- Term: Foot Deformities, Acquired
- ID: D000009140
- Term: Musculoskeletal Diseases
- ID: D000005532
- Term: Foot Deformities, Congenital
- ID: D000038061
- Term: Lower Extremity Deformities, Congenital
- ID: D000017880
- Term: Limb Deformities, Congenital
- ID: D000009139
- Term: Musculoskeletal Abnormalities
- ID: D000005534
- Term: Foot Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC16
- Name: Diseases and Abnormalities at or Before Birth
- Abbrev: All
- Name: All Conditions
- Abbrev: BC26
- Name: Wounds and Injuries
- Abbrev: BC05
- Name: Musculoskeletal Diseases
- Abbrev: BC17
- Name: Skin and Connective Tissue Diseases
### Condition Browse Module - Browse Leaves
- ID: M12
- Name: Congenital Abnormalities
- Relevance: HIGH
- As Found: Deformity
- ID: M15241
- Name: Rupture
- Relevance: HIGH
- As Found: Rupture
- ID: M8543
- Name: Flatfoot
- Relevance: HIGH
- As Found: Flat Foot
- ID: M8654
- Name: Foot Deformities
- Relevance: HIGH
- As Found: Deformity, Foot
- ID: M24664
- Name: Posterior Tibial Tendon Dysfunction
- Relevance: HIGH
- As Found: Tibialis Posterior Dysfunction
- ID: M8656
- Name: Foot Deformities, Congenital
- Relevance: LOW
- As Found: Unknown
- ID: M17685
- Name: Wounds and Injuries
- Relevance: LOW
- As Found: Unknown
- ID: M6255
- Name: Clubfoot
- Relevance: LOW
- As Found: Unknown
- ID: M586
- Name: Talipes
- Relevance: LOW
- As Found: Unknown
- ID: M8017
- Name: Equinus Deformity
- Relevance: LOW
- As Found: Unknown
- ID: M8655
- Name: Foot Deformities, Acquired
- Relevance: LOW
- As Found: Unknown
- ID: M12097
- Name: Musculoskeletal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M24718
- Name: Lower Extremity Deformities, Congenital
- Relevance: LOW
- As Found: Unknown
- ID: M20062
- Name: Limb Deformities, Congenital
- Relevance: LOW
- As Found: Unknown
- ID: M12096
- Name: Musculoskeletal Abnormalities
- Relevance: LOW
- As Found: Unknown
- ID: M8658
- Name: Foot Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000005413
- Term: Flatfoot
- ID: D000005530
- Term: Foot Deformities
- ID: D000037081
- Term: Posterior Tibial Tendon Dysfunction
- ID: D000000013
- Term: Congenital Abnormalities
- ID: D000012421
- Term: Rupture
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT04695379
**Acronym:** CO-MY-COVID
**Brief Title:** Follow-up of a Cohort of Patients With Myasthenic Syndrome and COVID-19 Infection
**Official Title:** Follow-up of a Cohort of Patients With Myasthenic Syndrome and COVID-19 Infection: Consequences on the Severity of Myasthenic Syndrome and Reciprocal Impact of the Two Pathologies on Their Respective Treatments
#### Organization Study ID Info
**ID:** CHUBX 2020/17
#### Organization
**Class:** OTHER
**Full Name:** University Hospital, Bordeaux
### Status Module
#### Completion Date
**Date:** 2020-06-18
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2023-10-06
**Type:** ACTUAL
**Last Update Submit Date:** 2023-10-05
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2020-06-18
**Type:** ACTUAL
#### Start Date
**Date:** 2020-04-27
**Type:** ACTUAL
**Status Verified Date:** 2023-10
#### Study First Post Date
**Date:** 2021-01-05
**Type:** ACTUAL
**Study First Submit Date:** 2020-12-31
**Study First Submit QC Date:** 2020-12-31
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** University Hospital, Bordeaux
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** Coronavirus disease 2019 (COVID-19), declared by the World Health Organization (WHO) as a "public health emergency of international concern" (January 31, 2020), has posed a significant threat to global health. This infectious disease, caused by the 'severe acute respiratory syndrome coronavirus-2'(SARS-CoV-2), was first reported in China at the end of 2019. As other coronaviruses, SARS-CoV-2 primarily targets the human respiratory system. The most common symptoms are fever, fatigue, and dry cough. During the second week of the disease, part of patients may progress to shortness of breath, then hypoxemia and severe pneumonia. Acute respiratory distress syndrome (ARDS), linked to some risk factors such as advanced age and underlying comorbidities (hypertension, diabetes, cardiovascular disease, and cerebrovascular disease), may be fatal and needs early supportive therapy and monitoring.
Some patients with COVID-19 experienced neurological complications including headache, dizziness, hypogeusia and/or anosmia, altered level of consciousness, strokes, seizures, and ataxia, less frequently neuromuscular disorders (NMD) such as acute inflammatory polyradiculoneuropathy. Among NMD, myasthenia gravis (MG) patients, particularly susceptible to infections causing crises, could be of special risk of COVID-19 ARDS. Some general recommendations were established for the management of NMD during the COVID-19 pandemic,with also specific recommendations for MG. However, only data on a small number of patients who were managed in hospital are currently available;in addition, only two cases of myasthenic crisis following COVID-19 were reported. For this reason, the French neuromuscular rare disease network (FILNEMUS: 'FILière NEuroMUSculaire') has created the 'CO-MY-COVID register' to describe the clinical course and prognosis of patients with COVID-19 and pre-existing myasthenic syndrome.
**Detailed Description:** Coronavirus disease 2019 (COVID-19), declared by the World Health Organization (WHO) as a "public health emergency of international concern" (January 31, 2020), has posed a significant threat to global health. This infectious disease, caused by the 'severe acute respiratory syndrome coronavirus-2'(SARS-CoV-2), was first reported in China at the end of 2019. Nowadays, with the exception of Antarctica, COVID-19 is a worldwide pandemic that continues to spread around the world (8,065,966 known cases and 437,604 deaths in June 16, 2020; https://gisanddata.maps.arcgis.com/). As other coronaviruses, SARS-CoV-2 primarily targets the human respiratory system. Its most convincing mode of transmission is inhalation of infectious aerosols or direct contact of infected people's droplets. The most common symptoms are fever, fatigue, and dry cough. During the second week of the disease, part of patients may progress to shortness of breath, then hypoxemia and severe pneumonia. Acute respiratory distress syndrome (ARDS), linked to some risk factors such as advanced age and underlying comorbidities (hypertension, diabetes, cardiovascular disease, and cerebrovascular disease), may be fatal and needs early supportive therapy and monitoring.
Some patients with COVID-19 experienced neurological complications including headache, dizziness, hypogeusia and/or anosmia, altered level of consciousness, strokes, seizures, and ataxia, less frequently neuromuscular disorders (NMD) such as acute inflammatory polyradiculoneuropathy. Among NMD, myasthenia gravis (MG) patients, particularly susceptible to infections causing crises, could be of special risk of COVID-19 ARDS. Some general recommendations were established for the management of NMD during the COVID-19 pandemic,with also specific recommendations for MG. However, only data on a small number of patients who were managed in hospital are currently available;in addition, only two cases of myasthenic crisis following COVID-19 were reported. For this reason, the French neuromuscular rare disease network (FILNEMUS: 'FILière NEuroMUSculaire') has created the 'CO-MY-COVID register' to describe the clinical course and prognosis of patients with COVID-19 and pre-existing myasthenic syndrome.
### Conditions Module
**Conditions:**
- Myasthenia Gravis
**Keywords:**
- COVID-19
- Neuromuscular Diseases
### Design Module
#### Design Info
**Observational Model:** COHORT
**Time Perspective:** PROSPECTIVE
#### Enrollment Info
**Count:** 34
**Type:** ACTUAL
**Study Type:** OBSERVATIONAL
### Outcomes Module
#### Primary Outcomes
**Description:** The severity of MG is measured by using the MGFA (Myasthenia Gravis Foundation of America) classification, giving the status of 'MG-improvement' (when the scores decreased or remained stable) or 'MG-worsening' (when the scores increased) (Jaretzki A, 3rd, Barohn RJ, Ernstoff RM, et al. Myasthenia gravis: recommendations for clinical research standards. Task Force of the Medical Scientific Advisory Board of the Myasthenia Gravis Foundation of America. Neurology 2000;55:16-23.)
**Measure:** Severity of Myasthenia Gravis evaluated by the Myasthenia Gravis of America (MGFA) score
**Time Frame:** 1 month after the inclusion visit
#### Secondary Outcomes
**Description:** The severity of MG is measured by using the MGFA (Myasthenia Gravis Foundation of America) classification, giving the status of 'MG-improvement' (when the scores decreased or remained stable) or 'MG-worsening' (when the scores increased) (Jaretzki A, 3rd, Barohn RJ, Ernstoff RM, et al. Myasthenia gravis: recommendations for clinical research standards. Task Force of the Medical Scientific Advisory Board of the Myasthenia Gravis Foundation of America. Neurology 2000;55:16-23.)
**Measure:** Severity of Myasthenia Gravis evaluated by the Myasthenia Gravis of America (MGFA) score
**Time Frame:** at inclusion (at the time of the COVID-19 diagnosis)
**Description:** The severity of MG is measured by using the MGFA (Myasthenia Gravis Foundation of America) classification, giving the status of 'MG-improvement' (when the scores decreased or remained stable) or 'MG-worsening' (when the scores increased) (Jaretzki A, 3rd, Barohn RJ, Ernstoff RM, et al. Myasthenia gravis: recommendations for clinical research standards. Task Force of the Medical Scientific Advisory Board of the Myasthenia Gravis Foundation of America. Neurology 2000;55:16-23.)
**Measure:** Severity of Myasthenia Gravis evaluated by the variation of the Myasthenia Gravis of America (MGFA) score
**Time Frame:** 3 months after the inclusion visit
**Description:** The severity of MG is measured by using the MGFA (Myasthenia Gravis Foundation of America) classification, giving the status of 'MG-improvement' (when the scores decreased or remained stable) or 'MG-worsening' (when the scores increased) (Jaretzki A, 3rd, Barohn RJ, Ernstoff RM, et al. Myasthenia gravis: recommendations for clinical research standards. Task Force of the Medical Scientific Advisory Board of the Myasthenia Gravis Foundation of America. Neurology 2000;55:16-23.)
**Measure:** Severity of Myasthenia Gravis evaluated by the variation of the Myasthenia Gravis of America (MGFA) score
**Time Frame:** 6 months after the inclusion visit
**Description:** The Myasthenia Gravis-specific Activities of Daily Living scale consists of the assessment of 8 parameters: speaking, chewing, swallowing, breathing, self-care activities (brushing the teeth or combing the hair), simple physical activities (getting up from a chair), double vision and eye lid dropping. Each parameter is subjected to assessment depending on the degree of symptoms intensification, awarding points from 0 to 3 points. The maximum number a patient may receive is 24 points. The higher the score of points, the bigger limitations of the patient in everyday life activities caused by intensification of myasthenia gravis (Wolfe GI, Herbelin L, Nations SP, Foster B, Bryan WW, Barohn RJ. Myasthenia gravis activities of daily living profile. Neurology 1999;52:1487-1489.)
**Measure:** The autonomy of the patients evaluated with the MG-ADL (Myasthenia Gravis-Activities of Daily Living) scale
**Time Frame:** at inclusion (at the time of the COVID-19 diagnosis)
**Description:** The Myasthenia Gravis-specific Activities of Daily Living scale consists of the assessment of 8 parameters: speaking, chewing, swallowing, breathing, self-care activities (brushing the teeth or combing the hair), simple physical activities (getting up from a chair), double vision and eye lid dropping. Each parameter is subjected to assessment depending on the degree of symptoms intensification, awarding points from 0 to 3 points. The maximum number a patient may receive is 24 points. The higher the score of points, the bigger limitations of the patient in everyday life activities caused by intensification of myasthenia gravis (Wolfe GI, Herbelin L, Nations SP, Foster B, Bryan WW, Barohn RJ. Myasthenia gravis activities of daily living profile. Neurology 1999;52:1487-1489.)
**Measure:** The autonomy of the patients evaluated with the MG-ADL (Myasthenia Gravis-Activities of Daily Living) scale
**Time Frame:** 1 month after the inclusion visit
**Description:** The Myasthenia Gravis-specific Activities of Daily Living scale consists of the assessment of 8 parameters: speaking, chewing, swallowing, breathing, self-care activities (brushing the teeth or combing the hair), simple physical activities (getting up from a chair), double vision and eye lid dropping. Each parameter is subjected to assessment depending on the degree of symptoms intensification, awarding points from 0 to 3 points. The maximum number a patient may receive is 24 points. The higher the score of points, the bigger limitations of the patient in everyday life activities caused by intensification of myasthenia gravis (Wolfe GI, Herbelin L, Nations SP, Foster B, Bryan WW, Barohn RJ. Myasthenia gravis activities of daily living profile. Neurology 1999;52:1487-1489.)
**Measure:** The autonomy of the patients evaluated with the MG-ADL (Myasthenia Gravis-Activities of Daily Living) scale
**Time Frame:** 3 months after the inclusion visit
**Description:** The Myasthenia Gravis-specific Activities of Daily Living scale consists of the assessment of 8 parameters: speaking, chewing, swallowing, breathing, self-care activities (brushing the teeth or combing the hair), simple physical activities (getting up from a chair), double vision and eye lid dropping. Each parameter is subjected to assessment depending on the degree of symptoms intensification, awarding points from 0 to 3 points. The maximum number a patient may receive is 24 points. The higher the score of points, the bigger limitations of the patient in everyday life activities caused by intensification of myasthenia gravis (Wolfe GI, Herbelin L, Nations SP, Foster B, Bryan WW, Barohn RJ. Myasthenia gravis activities of daily living profile. Neurology 1999;52:1487-1489.)
**Measure:** The autonomy of the patients evaluated with the MG-ADL (Myasthenia Gravis-Activities of Daily Living) scale
**Time Frame:** 6 months after the inclusion visit
**Description:** Risk factors for severe forms of COVID-19 are the following: age\>65, 'obesity' (body mass index (, BMI), \>30), 'chronic obstructive pulmonary disease' (COPD), 'obstructive sleep apnea syndrome' (OSAS), 'noninvasive ventilation' (NIV), 'arterial hypertension' , 'diabetes' and 'others'
**Measure:** Risk factors for severe forms of COVID-19
**Time Frame:** at inclusion (at the time of the COVID-19 diagnosis)
**Description:** Treatments for MG at the time of the diagnosis of COVID-19 are grouped into six categories: 'acetylcholinesterase inhibitors' (Ach-inh), 'corticosteroids', 'immunosuppressants', 'intravenous immunoglobulins' (IVIg) or 'subcutaneous immunoglobulins' (SCIg), 'plasmapheresis' (PLEX) and 'others
**Measure:** Treatments for MG at the time of the diagnosis of COVID-19
**Time Frame:** at inclusion (at the time of the COVID-19 diagnosis)
**Description:** The diagnosis of COVID-19 is considered as 'definite' if confirmed by a positive SARS-CoV-2 PCR (polymerase chain reaction) test and/or SARS-CoV-2 serology. The diagnosis of COVID-19 is considered 'probable' if: (i) the patient presented a viral syndrome and (ii) had contact with a confirmed patient considered to have a definite diagnosis of COVID-19 or had specific signs (anosmia, agueusia, skin signs) or had suggestive abnormalities on thoracic CT-scan.
**Measure:** Diagnosis of COVID-19
**Time Frame:** at inclusion (at the time of the COVID-19 diagnosis)
**Description:** The global severity of COVID-19 was based on the location of management of the patient during COIVD-19: 'home', 'medical unit' ('MU'), 'intensive care unit' ('ICU').
**Measure:** Severity of COVID-19
**Time Frame:** at inclusion (at the time of the COVID-19 diagnosis)
**Description:** Treatments for MG at the time of the diagnosis of COVID-19 are grouped into six categories: 'acetylcholinesterase inhibitors' (Ach-inh), 'corticosteroids', 'immunosuppressants', 'intravenous immunoglobulins' (IVIg) or 'subcutaneous immunoglobulins' (SCIg), 'plasmapheresis' (PLEX) and 'others
**Measure:** Treatments for MG during and after COVID-19
**Time Frame:** 1 month after the inclusion visit
**Description:** Treatments for MG at the time of the diagnosis of COVID-19 are grouped into six categories: 'acetylcholinesterase inhibitors' (Ach-inh), 'corticosteroids', 'immunosuppressants', 'intravenous immunoglobulins' (IVIg) or 'subcutaneous immunoglobulins' (SCIg), 'plasmapheresis' (PLEX) and 'others
**Measure:** Treatments for MG during and after COVID-19
**Time Frame:** 3 months after the inclusion visit
**Description:** Treatments for MG at the time of the diagnosis of COVID-19 are grouped into six categories: 'acetylcholinesterase inhibitors' (Ach-inh), 'corticosteroids', 'immunosuppressants', 'intravenous immunoglobulins' (IVIg) or 'subcutaneous immunoglobulins' (SCIg), 'plasmapheresis' (PLEX) and 'others
**Measure:** Treatments for MG during and after COVID-19
**Time Frame:** 6 months after the inclusion visit
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* 1. Child or adult patients, living or deceased, presenting or having presented a myasthenic syndrome and a COVID-19 infection 2. Myasthenic syndrome is established by:
* Either the presence of a specific antibody
* Either the presence of specific electrophysiological abnormalities
* Either an evocative symptomatology improved by a therapeutic test with an acetylcholinesterase inhibitor
* Either one or two pathogenic mutation (s) in a gene involved in congenital myasthenic syndromes (dominant or recessive disease).
3. COVID-19 infection is established by
* Either a positive PCR test
* Or a specific chest scanner
* Either a positive serology
* Either a clinical syndrome of COVID-19, validated by a committee of experts. 4. Patients affiliated or beneficiaries of a social security scheme 5. For living patients: patients who have been informed of the study and have not exercised their right of opposition or parents or holders of parental authority who have been informed of the study and have not exercised their right opposition.
For deceased patients: beneficiaries or parents / holders of parental authority having been informed of the study and not having exercised their right of objection.
Exclusion Criteria:
1. Persons placed under judicial protection
**Maximum Age:** 99 Years
**Minimum Age:** 1 Year
**Sampling Method:** NON_PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- CHILD
- ADULT
- OLDER_ADULT
**Study Population:** Child or adult patients, living or deceased, presenting or having presented a myasthenic syndrome and a COVID-19 infection
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Angers
**Country:** France
**Facility:** CHU d'Angers
**Zip:** 49933
**Location 2:**
**City:** Bordeaux
**Country:** France
**Facility:** CHU de Bordeaux
**Zip:** 33076
**Location 3:**
**City:** Bron
**Country:** France
**Facility:** Hospices Civils de Lyon
**Zip:** 69677
**Location 4:**
**City:** Garches
**Country:** France
**Facility:** APHP - Hopital Raymond Poincarré
**Zip:** 92380
**Location 5:**
**City:** Lille
**Country:** France
**Facility:** CHRU de Lille
**Zip:** 59037
**Location 6:**
**City:** Marseille
**Country:** France
**Facility:** Assistance Publique Hôpitaux de Marseille
**Zip:** 13385
**Location 7:**
**City:** Nantes
**Country:** France
**Facility:** CHU de Nantes
**Zip:** 44093
**Location 8:**
**City:** Paris
**Country:** France
**Facility:** APHP GH Pitié Salpétrière
**Zip:** 75013
**Location 9:**
**City:** Strasbourg
**Country:** France
**Facility:** CHU de Strasbourg
**Zip:** 67098
**Location 10:**
**City:** Toulouse
**Country:** France
**Facility:** CHU de Toulouse
**Zip:** 31059
#### Overall Officials
**Official 1:**
**Affiliation:** Université Hospital, Bordeaux
**Name:** Guilhem SOLE, MD
**Role:** PRINCIPAL_INVESTIGATOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000007239
- Term: Infections
- ID: D000011024
- Term: Pneumonia, Viral
- ID: D000011014
- Term: Pneumonia
- ID: D000012141
- Term: Respiratory Tract Infections
- ID: D000014777
- Term: Virus Diseases
- ID: D000018352
- Term: Coronavirus Infections
- ID: D000003333
- Term: Coronaviridae Infections
- ID: D000030341
- Term: Nidovirales Infections
- ID: D000012327
- Term: RNA Virus Infections
- ID: D000008171
- Term: Lung Diseases
- ID: D000012140
- Term: Respiratory Tract Diseases
- ID: D000020361
- Term: Paraneoplastic Syndromes, Nervous System
- ID: D000009423
- Term: Nervous System Neoplasms
- ID: D000009371
- Term: Neoplasms by Site
- ID: D000009369
- Term: Neoplasms
- ID: D000010257
- Term: Paraneoplastic Syndromes
- ID: D000020274
- Term: Autoimmune Diseases of the Nervous System
- ID: D000009422
- Term: Nervous System Diseases
- ID: D000019636
- Term: Neurodegenerative Diseases
- ID: D000020511
- Term: Neuromuscular Junction Diseases
- ID: D000009468
- Term: Neuromuscular Diseases
- ID: D000001327
- Term: Autoimmune Diseases
- ID: D000007154
- Term: Immune System Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC01
- Name: Infections
- Abbrev: BC08
- Name: Respiratory Tract (Lung and Bronchial) Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC10
- Name: Nervous System Diseases
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: BC20
- Name: Immune System Diseases
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M2561
- Name: COVID-19
- Relevance: HIGH
- As Found: COVID-19
- ID: M12411
- Name: Neuromuscular Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M10283
- Name: Infections
- Relevance: LOW
- As Found: Unknown
- ID: M6368
- Name: Communicable Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M16355
- Name: Syndrome
- Relevance: LOW
- As Found: Unknown
- ID: M12112
- Name: Myasthenia Gravis
- Relevance: HIGH
- As Found: Myasthenia Gravis
- ID: M18224
- Name: Lambert-Eaton Myasthenic Syndrome
- Relevance: HIGH
- As Found: Myasthenic Syndrome
- ID: M13904
- Name: Pneumonia
- Relevance: LOW
- As Found: Unknown
- ID: M13914
- Name: Pneumonia, Viral
- Relevance: LOW
- As Found: Unknown
- ID: M14978
- Name: Respiratory Tract Infections
- Relevance: LOW
- As Found: Unknown
- ID: M17522
- Name: Virus Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M20490
- Name: Coronavirus Infections
- Relevance: LOW
- As Found: Unknown
- ID: M6555
- Name: Coronaviridae Infections
- Relevance: LOW
- As Found: Unknown
- ID: M23685
- Name: Nidovirales Infections
- Relevance: LOW
- As Found: Unknown
- ID: M15149
- Name: RNA Virus Infections
- Relevance: LOW
- As Found: Unknown
- ID: M11168
- Name: Lung Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M14977
- Name: Respiratory Tract Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M13170
- Name: Paraneoplastic Syndromes
- Relevance: LOW
- As Found: Unknown
- ID: M22160
- Name: Paraneoplastic Syndromes, Nervous System
- Relevance: LOW
- As Found: Unknown
- ID: M12367
- Name: Nervous System Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M4629
- Name: Autoimmune Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M22094
- Name: Autoimmune Diseases of the Nervous System
- Relevance: LOW
- As Found: Unknown
- ID: M21558
- Name: Neurodegenerative Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M22297
- Name: Neuromuscular Junction Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M10200
- Name: Immune System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: T3973
- Name: Myasthenia Gravis
- Relevance: HIGH
- As Found: Myasthenia Gravis
- ID: T3299
- Name: Lambert Eaton Myasthenic Syndrome
- Relevance: HIGH
- As Found: Myasthenic Syndrome
### Condition Browse Module - Meshes
- ID: D000086382
- Term: COVID-19
- ID: D000009157
- Term: Myasthenia Gravis
- ID: D000015624
- Term: Lambert-Eaton Myasthenic Syndrome
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT03516279
**Brief Title:** Pembrolizumab and Dasatinib, Imatinib Mesylate, or Nilotinib in Treating Patients With Chronic Myeloid Leukemia and Persistently Detectable Minimal Residual Disease
**Official Title:** Phase II Study of Adding the Anti-PD-1 Pembrolizumab to Tyrosine Kinase Inhibitors in Patients With Chronic Myeloid Leukemia and Persistently Detectable Minimal Residual Disease
#### Organization Study ID Info
**ID:** EA9171
#### Organization
**Class:** NETWORK
**Full Name:** Eastern Cooperative Oncology Group
#### Secondary ID Infos
**Domain:** CTRP (Clinical Trial Reporting Program)
**ID:** NCI-2017-02161
**Type:** REGISTRY
**Domain:** ECOG-ACRIN Cancer Research Group
**ID:** EA9171
**Type:** OTHER
**Domain:** CTEP
**ID:** EA9171
**Type:** OTHER
**ID:** U10CA180820
**Link:** https://reporter.nih.gov/quickSearch/U10CA180820
**Type:** NIH
### Status Module
#### Completion Date
**Date:** 2031-08-31
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2023-11-28
**Type:** ACTUAL
**Last Update Submit Date:** 2023-11-27
**Overall Status:** RECRUITING
#### Primary Completion Date
**Date:** 2028-08-31
**Type:** ESTIMATED
#### Start Date
**Date:** 2019-06-26
**Type:** ACTUAL
**Status Verified Date:** 2023-11
#### Study First Post Date
**Date:** 2018-05-04
**Type:** ACTUAL
**Study First Submit Date:** 2018-04-24
**Study First Submit QC Date:** 2018-04-24
### Sponsor Collaborators Module
#### Collaborators
**Class:** NIH
**Name:** National Cancer Institute (NCI)
#### Lead Sponsor
**Class:** NETWORK
**Name:** ECOG-ACRIN Cancer Research Group
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** True
**Is US Export:** True
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** This phase II trial studies how well pembrolizumab and dasatinib, imatinib mesylate, or nilotinib work in treating patients with chronic myeloid leukemia and persistent detection of minimal residual disease, defined as the levels of a gene product called bcr-abl in the blood. Monoclonal antibodies, such as pembrolizumab, may interfere with the ability of cancer cells to grow and spread. Dasatinib, imatinib mesylate, and nilotinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Giving pembrolizumab and dasatinib, imatinib mesylate, or nilotinib may work better in treating patients with chronic myeloid leukemia.
**Detailed Description:** PRIMARY OBJECTIVES:
I. Assess the proportion of chronic myelogenous leukemia (CML) patients on stable-dose tyrosine kinase inhibitor (TKI) who convert to undetectable minimal residual disease (UMRD) (molecular response \[MR\]\^4.5) during or within 2 years of initiating pembrolizumab therapy.
SECONDARY OBJECTIVES:
I. Among patients who have converted to UMRD (MR\^4.5), assess the proportion of CML patients who maintain UMRD for 6 months and 12 months.
II. Among patients who have converted to UMRD (MR\^4.5), assess the proportion of CML patients who discontinue their TKI.
III. Among patients who have converted to UMRD (MR\^4.5), assess the proportion of CML patients who are UMRD and TKI-free at 2 years from first determined UMRD.
IV. Assess the proportion of CML patients who develop grade 3 or 4 immune related adverse events related to pembrolizumab treatment during the first 2 years after registration (not including grade 3 events that respond to corticosteroids and improve to grade 1 or less within 4 weeks).
OUTLINE:
Patients receive pembrolizumab intravenously (IV) over 30 minutes on day 1 and dasatinib, imatinib mesylate, or nilotinib orally (PO) as clinically indicated per the treating physician. Treatment repeats every 21 days for up to 18 courses in the absence of disease progression or unacceptable toxicity. Patients with detectable MRD after course 18 continue pembrolizumab and dasatinib, imatinib mesylate, or nilotinib every 21 days for up to an additional 18 courses in the absence of disease progression or unacceptable toxicity. Patients with UMRD at any time before course 18 discontinue pembrolizumab after course 18 and continue dasatinib, imatinib mesylate, or nilotinib every 21 days for up to an additional 18 courses in the absence of disease progression or unacceptable toxicity.
After completion of study treatment, patients are followed up every 6 months for 6 years from the date of registration.
### Conditions Module
**Conditions:**
- Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive
- Minimal Residual Disease
### Design Module
#### Design Info
**Allocation:** NA
**Intervention Model:** SINGLE_GROUP
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 40
**Type:** ESTIMATED
**Phases:**
- PHASE2
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Patients receive pembrolizumab IV over 30 minutes on day 1 and dasatinib, imatinib mesylate, or nilotinib PO as clinically indicated per the treating physician. Treatment repeats every 21 days for up to 18 courses in the absence of disease progression or unacceptable toxicity. Patients with detectable MRD after course 18 continue pembrolizumab and dasatinib, imatinib mesylate, or nilotinib every 21 days for up to an additional 18 courses in the absence of disease progression or unacceptable toxicity. Patients with UMRD at any time before course 18 discontinue pembrolizumab after course 18 and continue dasatinib, imatinib mesylate, or nilotinib every 21 days for up to an additional 18 courses in the absence of disease progression or unacceptable toxicity.
**Intervention Names:**
- Drug: Dasatinib
- Drug: Imatinib Mesylate
- Other: Laboratory Biomarker Analysis
- Drug: Nilotinib
- Biological: Pembrolizumab
**Label:** Treatment (pembrolizumab, dasatinib, imatinib, nilotinib)
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Treatment (pembrolizumab, dasatinib, imatinib, nilotinib)
**Description:** Given PO
**Name:** Dasatinib
**Other Names:**
- BMS-354825
- Sprycel
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- Treatment (pembrolizumab, dasatinib, imatinib, nilotinib)
**Description:** Given PO
**Name:** Imatinib Mesylate
**Other Names:**
- CGP 57148
- CGP57148B
- Gleevec
- Glivec
- STI 571
- STI-571
- STI571
**Type:** DRUG
#### Intervention 3
**Arm Group Labels:**
- Treatment (pembrolizumab, dasatinib, imatinib, nilotinib)
**Description:** Correlative studies
**Name:** Laboratory Biomarker Analysis
**Type:** OTHER
#### Intervention 4
**Arm Group Labels:**
- Treatment (pembrolizumab, dasatinib, imatinib, nilotinib)
**Description:** Given PO
**Name:** Nilotinib
**Other Names:**
- AMN 107
- Tasigna
**Type:** DRUG
#### Intervention 5
**Arm Group Labels:**
- Treatment (pembrolizumab, dasatinib, imatinib, nilotinib)
**Description:** Given IV
**Name:** Pembrolizumab
**Other Names:**
- Keytruda
- Lambrolizumab
- MK-3475
- SCH 900475
**Type:** BIOLOGICAL
### Outcomes Module
#### Primary Outcomes
**Description:** Will be reported with exact confidence intervals. The binomial test will be used to test the null hypothesis that this proportion is 0.2 with the alternative hypothesis being that this proportion is greater than 0.2.
**Measure:** Proportion of patients on tyrosine kinase inhibitor (TKI) who convert to undetectable minimal residual disease (UMRD)
**Time Frame:** Up to 2 years of initiating pembrolizumab
#### Secondary Outcomes
**Description:** Will be assessed among patients who achieve UMRD within two years of initiating pembrolizumab. The proportions will be reported with exact confidence intervals.
**Measure:** Proportion of patients who maintain UMRD for 6 months
**Time Frame:** Up to 6 years
**Description:** Will be assessed among patients who achieve UMRD within two years of initiating pembrolizumab. The proportions will be reported with exact confidence intervals.
**Measure:** Proportion of patients who maintain UMRD for 6 and 12 months
**Time Frame:** Up to 6 years
**Description:** Will be assessed among patients who achieve and maintain UMRD within two years of initiating pembrolizumab. The proportions will be reported with exact confidence intervals.
**Measure:** Proportion of patients who meet the criteria for discontinuing TKI
**Time Frame:** Up to 6 years
**Description:** Will be assessed among patients who have converted to UMRD (molecular response \[MR\]\^4.5) and discontinued TKI. The proportions will be reported with exact confidence intervals.
**Measure:** Proportion of patients who maintain UMRD for 2 years after first achieving UMRD
**Time Frame:** Up to 6 years
**Description:** Will be tabulated based on grade Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
**Measure:** Proportion of patients who develop grade 3 or 4 immune related adverse events (not including grade 3 events that respond to corticosteroids and improve to grade 1 or less within 4 weeks)
**Time Frame:** Up to 6 years
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* PREREGISTRATION (STEP 0): Patient has pathologically-confirmed chronic phase-CML on a first line TKI and must meet the following criteria:
* The patient has to be on first-line TKI therapy (the same TKI) for at least 2 years prior to pre-registration
* Has been in MMR (i.e. MR\^3) but still have detectable BCR-ABL transcript by a standard real-time quantitative polymerase chain reaction (RQ-PCR) assay with a limit of detection (sensitivity) of 4.5 for at least 12 months from the first documentation of the MMR
* Patient has not achieved MR\^4.5 (complete molecular remission \[CMR\]) within the time of initiation of TKI therapy and pre-registration
* PREREGISTRATION (STEP 0): Patient must be scheduled to undergo a standard of care bone marrow biopsy within 7 days of step 0 registration
* PREREGISTRATION (STEP 0): Peripheral blood must be collected for submission to Fred Hutchinson Cancer Research Center for central assessment of the establishment of BCR/ABL status to confirm patient?s eligibility for registration to Step 1; Fred Hutchinson will forward results within 1-2 business days of receipt of the peripheral blood to the submitting institution
* REGISTRATION TO TREATMENT (STEP 1): Institution has received central BCR-ABL test results confirming MRD positive status
* REGISTRATION TO TREATMENT (STEP 1): Patients have an Eastern Cooperative Oncology Group (ECOG) performance status of 0-1
* REGISTRATION TO TREATMENT (STEP 1): No active hemolytic anemia requiring immunosuppressive therapy or other pharmacologic treatment; patients who have a positive Coombs test but no evidence of hemolysis are NOT excluded from participation
* REGISTRATION TO TREATMENT (STEP 1): No current use of corticosteroids; EXCEPTION: Low doses of steroids (\< 10 mg of prednisone or equivalent dose of other steroid) used for treatment of non-hematologic medical condition (e.g. chronic adrenal insufficiency) is permitted
* REGISTRATION TO TREATMENT (STEP 1): No other active primary malignancy (other than non-melanomatous skin cancer or carcinoma in situ of the cervix) requiring treatment or limiting expected survival to =\< 2 years
* NOTE: If there is a history of prior malignancy, they must not be receiving other specific treatment (other than hormonal therapy for their cancer)
* REGISTRATION TO TREATMENT (STEP 1): Women must not be pregnant or breastfeeding; patients must also not expect to conceive or father children from the time of registration, while on study treatment, and continue for 120 days after the last dose of study treatment; all females of childbearing potential must have a negative urine or serum pregnancy within 72 hours prior to receiving the first dose of pembrolizumab; if the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required; a female of childbearing potential is any woman, regardless of sexual orientation or whether they have undergone tubal ligation, who meets the following criteria: 1) has not undergone a hysterectomy or bilateral oophorectomy; or 2) has not been naturally postmenopausal for at least 24 consecutive months (i.e., has had menses at any time in the preceding 24 consecutive months)
* REGISTRATION TO TREATMENT (STEP 1): Women of childbearing potential and sexually active males must use an accepted and effective method of contraception or to abstain from sexual from time of registration, while on study treatment, and continue for 120 days after the last dose of study treatment
* REGISTRATION TO TREATMENT (STEP 1): Patients must have been on a stable dose of the TKI for the last 3 months prior to pre-registration
* REGISTRATION TO TREATMENT (STEP 1): Patient may not be currently participating and receiving study therapy or have participated in a study of an investigational agent and received study therapy or used an investigational device within 4 weeks of the first dose of treatment
* REGISTRATION TO TREATMENT (STEP 1): Patient must not have a diagnosis of immunodeficiency or be receiving systemic steroid therapy or any other form of immunosuppressive therapy within 7 days prior to the first dose of treatment
* REGISTRATION TO TREATMENT (STEP 1): Patient must not have a known history of active TB (Bacillus Tuberculosis)
* REGISTRATION TO TREATMENT (STEP 1): Patient must not have a history of hypersensitivity to pembrolizumab or any of its excipients
* REGISTRATION TO TREATMENT (STEP 1): Patient must not have received a prior anti-cancer monoclonal antibody (mAb) within 4 weeks prior to study registration or have not recovered (i.e., =\< grade 1 or at baseline) from adverse events due to agents administered more than 4 weeks earlier
* REGISTRATION TO TREATMENT (STEP 1): Patient must not have had prior chemotherapy, targeted small molecule therapy (aside from imatinib, dasatinib, or nilotinib), or radiation therapy within 2 weeks prior to study registration; patients also must have recovered from all adverse events due to a previously administered agent
* Note: Patients with =\< grade 2 neuropathy are an exception to this criterion and may qualify for the study
* REGISTRATION TO TREATMENT (STEP 1): Patients who have received major surgery must have recovered adequately from the toxicity and/or complications from the intervention prior to starting therapy
* REGISTRATION TO TREATMENT (STEP 1): Patient must not have a known additional malignancy that is progressing or requires active treatment; exceptions include basal cell carcinoma of the skin or squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer
* REGISTRATION TO TREATMENT (STEP 1): Patient must not have known active central nervous system (CNS) metastases and/or carcinomatous meningitis; subjects with previously treated brain metastases may participate provided they are stable (without evidence of progression by imaging for at least four weeks prior to the first dose of protocol treatment and any neurologic symptoms have returned to baseline), have no evidence of new or enlarging brain metastases, and are not using steroids for at least 7 days prior to protocol treatment; this exception does not include carcinomatous meningitis which is excluded regardless of clinical stability
* REGISTRATION TO TREATMENT (STEP 1): Patient must not have active autoimmune disease that has required systemic treatment in the past 2 years (i.e. with use of disease modifying agents, corticosteroids or immunosuppressive drugs); replacement therapy (e.g., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment
* REGISTRATION TO TREATMENT (STEP 1): Patient must not have known history of, or any evidence of active, non-infectious pneumonitis
* REGISTRATION TO TREATMENT (STEP 1): Patient must not have an active infection requiring systemic therapy
* REGISTRATION TO TREATMENT (STEP 1): Patient must not have a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial, interfere with the subject's participation for the full duration of the trial, or is not in the best interest of the subject to participate, in the opinion of the treating investigator
* REGISTRATION TO TREATMENT (STEP 1): Patient must not have known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial
* REGISTRATION TO TREATMENT (STEP 1): Patient must not have received prior therapy with an anti-PD-1, anti-PD-L1, or anti-PD-L2 agent
* REGISTRATION TO TREATMENT (STEP 1): Patients who are Human Immunodeficiency Virus (HIV) positive are eligible if they have undetectable HIV viral load and CD4+ T-cell count ≥ 250/mm3.
* REGISTRATION TO TREATMENT (STEP 1): Patients with a known positive test for Hepatitis C virus ribonucleic acid (HCV antibody) indicating acute or chronic infection might be enrolled if the viral load by PCR is undetectable with/without active treatment.
* REGISTRATION TO TREATMENT (STEP 1): Patients must not have known history of hepatitis B (defined as Hepatitis B surface antigen \[HBsAg\] reactive).
* REGISTRATION TO TREATMENT (STEP 1): Patient must not have received a live vaccine within 30 days of planned start of study therapy
* NOTE: Seasonal influenza vaccines for injection are generally inactivated flu vaccines and are allowed; however intranasal influenza vaccines (e.g., Flu-Mist) are live attenuated vaccines, and are not allowed
* REGISTRATION TO TREATMENT (STEP 1): Absolute neutrophil count (ANC) \>= 1,500 /mcL, within 14 days prior to first dose of pembrolizumab
* REGISTRATION TO TREATMENT (STEP 1): Platelet count \>= 100,000 /mcL, within 14 days prior to first dose of pembrolizumab
* REGISTRATION TO TREATMENT (STEP 1): Hemoglobin (Hgb) \>= 9 g/dL OR \>= 5.6 mmol/L without transfusion of erythropoietin (EPO) dependency, within 14 days prior to first dose of pembrolizumab
* REGISTRATION TO TREATMENT (STEP 1): Serum creatinine =\< 1.5 X upper limit of normal (ULN) OR creatinine clearance (per institutional standards) \>= 60 mL/min for patient with creatinine levels \> 1.5 X ULN, within 14 days prior to first dose of pembrolizumab
* REGISTRATION TO TREATMENT (STEP 1): Serum total bilirubin =\< 1.5 X ULN OR direct bilirubin
=\< ULN for subjects with total bilirubin levels \> 1.5 X ULN, within 14 days prior to first dose of pembrolizumab
* REGISTRATION TO TREATMENT (STEP 1): Aspartate aminotransferase (AST) (serum glutamic oxaloacetic transaminase \[SGOT\]) and alanine aminotransferase (ALT) (serum glutamic pyruvic transaminase \[SGPT\]) =\< 2.5 X ULN OR =\< 5 X ULN for subjects with liver metastases, within 14 days prior to first dose of pembrolizumab
* REGISTRATION TO TREATMENT (STEP 1): Patients should not be receiving concomitant strong CYP3A4 inducers or inhibitors ≤ 7 days prior to registration due to their potential to effect the activity or pharmacokinetics of study agents and/or QT interval prolongation toxicity. Should treatment with any of these agents be required, consult with study chair.
* REGISTRATION TO TREATMENT (STEP 1): Patients should not have received prior allogeneic transplant.
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Goodyear
**Country:** United States
**Facility:** CTCA at Western Regional Medical Center
**State:** Arizona
**Status:** ACTIVE_NOT_RECRUITING
**Zip:** 85338
**Location 2:**
**City:** Phoenix
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 602-406-8222
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Cancer Center at Saint Joseph's
**State:** Arizona
**Status:** RECRUITING
**Zip:** 85004
**Location 3:**
**City:** Fort Smith
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 800-378-9373
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jay W. Carlson
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Mercy Hospital Fort Smith
**State:** Arkansas
**Status:** RECRUITING
**Zip:** 72903
**Location 4:**
**City:** Hot Springs
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** CHI Saint Vincent Cancer Center Hot Springs
**State:** Arkansas
**Status:** RECRUITING
**Zip:** 71913
**Location 5:**
**City:** Arroyo Grande
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 916-851-2283
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Mission Hope Medical Oncology - Arroyo Grande
**State:** California
**Status:** RECRUITING
**Zip:** 93420
**Location 6:**
**City:** San Luis Obispo
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Pacific Central Coast Health Center-San Luis Obispo
**State:** California
**Status:** RECRUITING
**Zip:** 93401
**Location 7:**
**City:** Santa Maria
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 916-851-2283
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Mission Hope Medical Oncology - Santa Maria
**State:** California
**Status:** RECRUITING
**Zip:** 93444
**Location 8:**
**City:** Colorado Springs
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Penrose-Saint Francis Healthcare
**State:** Colorado
**Status:** RECRUITING
**Zip:** 80907
**Location 9:**
**City:** Colorado Springs
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 303-777-2663
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Rocky Mountain Cancer Centers-Penrose
**State:** Colorado
**Status:** RECRUITING
**Zip:** 80907
**Location 10:**
**City:** Denver
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Porter Adventist Hospital
**State:** Colorado
**Status:** RECRUITING
**Zip:** 80210
**Location 11:**
**City:** Durango
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Mercy Medical Center
**State:** Colorado
**Status:** RECRUITING
**Zip:** 81301
**Location 12:**
**City:** Durango
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Southwest Oncology PC
**State:** Colorado
**Status:** RECRUITING
**Zip:** 81301
**Location 13:**
**City:** Golden
**Country:** United States
**Facility:** Mountain Blue Cancer Care Center
**State:** Colorado
**Status:** SUSPENDED
**Zip:** 80401
**Location 14:**
**City:** Lakewood
**Country:** United States
**Facility:** Rocky Mountain Cancer Centers-Lakewood
**State:** Colorado
**Status:** SUSPENDED
**Zip:** 80228
**Location 15:**
**City:** Lakewood
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Saint Anthony Hospital
**State:** Colorado
**Status:** RECRUITING
**Zip:** 80228
**Location 16:**
**City:** Littleton
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Littleton Adventist Hospital
**State:** Colorado
**Status:** RECRUITING
**Zip:** 80122
**Location 17:**
**City:** Longmont
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Longmont United Hospital
**State:** Colorado
**Status:** RECRUITING
**Zip:** 80501
**Location 18:**
**City:** Longmont
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 303-777-2663
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Rocky Mountain Cancer Centers-Longmont
**State:** Colorado
**Status:** RECRUITING
**Zip:** 80501
**Location 19:**
**City:** Parker
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Parker Adventist Hospital
**State:** Colorado
**Status:** RECRUITING
**Zip:** 80138
**Location 20:**
**City:** Parker
**Country:** United States
**Facility:** Rocky Mountain Cancer Centers-Parker
**State:** Colorado
**Status:** SUSPENDED
**Zip:** 80138
**Location 21:**
**City:** Pueblo
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Saint Mary Corwin Medical Center
**State:** Colorado
**Status:** RECRUITING
**Zip:** 81004
**Location 22:**
**City:** Pueblo
**Country:** United States
**Facility:** Rocky Mountain Cancer Centers - Pueblo
**State:** Colorado
**Status:** SUSPENDED
**Zip:** 81008
**Location 23:**
**City:** Thornton
**Country:** United States
**Facility:** Rocky Mountain Cancer Centers-Thornton
**State:** Colorado
**Status:** SUSPENDED
**Zip:** 80260
**Location 24:**
**City:** Derby
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 203-785-5702
- **Role:** CONTACT
***Contact 2:***
- **Name:** Amer M. Zeidan
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Smilow Cancer Hospital-Derby Care Center
**State:** Connecticut
**Status:** RECRUITING
**Zip:** 06418
**Location 25:**
**City:** Fairfield
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 203-785-5702
- **Role:** CONTACT
***Contact 2:***
- **Name:** Amer M. Zeidan
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Smilow Cancer Hospital Care Center-Fairfield
**State:** Connecticut
**Status:** RECRUITING
**Zip:** 06824
**Location 26:**
**City:** Guilford
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 203-785-5702
- **Role:** CONTACT
***Contact 2:***
- **Name:** Amer M. Zeidan
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Smilow Cancer Hospital Care Center - Guiford
**State:** Connecticut
**Status:** RECRUITING
**Zip:** 06437
**Location 27:**
**City:** Hartford
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 203-785-5702
- **Role:** CONTACT
***Contact 2:***
- **Name:** Amer M. Zeidan
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Smilow Cancer Hospital Care Center at Saint Francis
**State:** Connecticut
**Status:** RECRUITING
**Zip:** 06105
**Location 28:**
**City:** New Haven
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 203-785-5702
- **Role:** CONTACT
***Contact 2:***
- **Name:** Amer M. Zeidan
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Smilow Cancer Center/Yale-New Haven Hospital
**State:** Connecticut
**Status:** RECRUITING
**Zip:** 06510
**Location 29:**
**City:** North Haven
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 203-785-5702
- **Role:** CONTACT
***Contact 2:***
- **Name:** Amer M. Zeidan
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Yale-New Haven Hospital North Haven Medical Center
**State:** Connecticut
**Status:** RECRUITING
**Zip:** 06473
**Location 30:**
**City:** Torrington
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 203-785-5702
- **Role:** CONTACT
***Contact 2:***
- **Name:** Amer M. Zeidan
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Smilow Cancer Hospital-Torrington Care Center
**State:** Connecticut
**Status:** RECRUITING
**Zip:** 06790
**Location 31:**
**City:** Trumbull
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 203-785-5702
- **Role:** CONTACT
***Contact 2:***
- **Name:** Amer M. Zeidan
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Smilow Cancer Hospital Care Center-Trumbull
**State:** Connecticut
**Status:** RECRUITING
**Zip:** 06611
**Location 32:**
**City:** Waterbury
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 203-785-5702
- **Role:** CONTACT
***Contact 2:***
- **Name:** Amer M. Zeidan
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Smilow Cancer Hospital-Waterbury Care Center
**State:** Connecticut
**Status:** RECRUITING
**Zip:** 06708
**Location 33:**
**City:** Waterford
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 203-785-5702
- **Role:** CONTACT
***Contact 2:***
- **Name:** Amer M. Zeidan
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Smilow Cancer Hospital Care Center - Waterford
**State:** Connecticut
**Status:** RECRUITING
**Zip:** 06385
**Location 34:**
**City:** Frankford
**Country:** United States
**Facility:** Beebe South Coastal Health Campus
**State:** Delaware
**Status:** ACTIVE_NOT_RECRUITING
**Zip:** 19945
**Location 35:**
**City:** Lewes
**Country:** United States
**Facility:** Beebe Medical Center
**State:** Delaware
**Status:** ACTIVE_NOT_RECRUITING
**Zip:** 19958
**Location 36:**
**City:** Newark
**Country:** United States
**Facility:** Delaware Clinical and Laboratory Physicians PA
**State:** Delaware
**Status:** ACTIVE_NOT_RECRUITING
**Zip:** 19713
**Location 37:**
**City:** Newark
**Country:** United States
**Facility:** Helen F Graham Cancer Center
**State:** Delaware
**Status:** ACTIVE_NOT_RECRUITING
**Zip:** 19713
**Location 38:**
**City:** Newark
**Country:** United States
**Facility:** Medical Oncology Hematology Consultants PA
**State:** Delaware
**Status:** ACTIVE_NOT_RECRUITING
**Zip:** 19713
**Location 39:**
**City:** Newark
**Country:** United States
**Facility:** Christiana Care Health System-Christiana Hospital
**State:** Delaware
**Status:** ACTIVE_NOT_RECRUITING
**Zip:** 19718
**Location 40:**
**City:** Rehoboth Beach
**Country:** United States
**Facility:** Beebe Health Campus
**State:** Delaware
**Status:** ACTIVE_NOT_RECRUITING
**Zip:** 19971
**Location 41:**
**City:** Seaford
**Country:** United States
**Facility:** TidalHealth Nanticoke / Allen Cancer Center
**State:** Delaware
**Status:** ACTIVE_NOT_RECRUITING
**Zip:** 19973
**Location 42:**
**City:** Wilmington
**Country:** United States
**Facility:** Christiana Care Health System-Wilmington Hospital
**State:** Delaware
**Status:** ACTIVE_NOT_RECRUITING
**Zip:** 19801
**Location 43:**
**City:** Boise
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 734-712-3671
- **Role:** CONTACT
***Contact 2:***
- **Name:** John M. Schallenkamp
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Saint Alphonsus Cancer Care Center-Boise
**State:** Idaho
**Status:** RECRUITING
**Zip:** 83706
**Location 44:**
**City:** Caldwell
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 734-712-3671
- **Role:** CONTACT
***Contact 2:***
- **Name:** John M. Schallenkamp
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Saint Alphonsus Cancer Care Center-Caldwell
**State:** Idaho
**Status:** RECRUITING
**Zip:** 83605
**Location 45:**
**City:** Coeur d'Alene
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 406-969-6060
- **Role:** CONTACT
***Contact 2:***
- **Name:** John M. Schallenkamp
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Kootenai Medical Center
**State:** Idaho
**Status:** RECRUITING
**Zip:** 83814
**Location 46:**
**City:** Emmett
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 734-712-3671
- **Role:** CONTACT
***Contact 2:***
- **Name:** John M. Schallenkamp
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Walter Knox Memorial Hospital
**State:** Idaho
**Status:** RECRUITING
**Zip:** 83617
**Location 47:**
**City:** Meridian
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 734-712-3671
- **Role:** CONTACT
***Contact 2:***
- **Name:** John M. Schallenkamp
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Idaho Urologic Institute-Meridian
**State:** Idaho
**Status:** RECRUITING
**Zip:** 83642
**Location 48:**
**City:** Nampa
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 734-712-3671
- **Role:** CONTACT
***Contact 2:***
- **Name:** John M. Schallenkamp
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Saint Alphonsus Medical Center-Nampa
**State:** Idaho
**Status:** RECRUITING
**Zip:** 83686
**Location 49:**
**City:** Post Falls
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 406-969-6060
- **Role:** CONTACT
***Contact 2:***
- **Name:** John M. Schallenkamp
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Kootenai Cancer Center
**State:** Idaho
**Status:** RECRUITING
**Zip:** 83854
**Location 50:**
**City:** Sandpoint
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 406-969-6060
- **Role:** CONTACT
***Contact 2:***
- **Name:** John M. Schallenkamp
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Kootenai Cancer Clinic
**State:** Idaho
**Status:** RECRUITING
**Zip:** 83864
**Location 51:**
**City:** Aurora
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 630-978-6212
- **Role:** CONTACT
***Contact 2:***
- **Name:** Priyank P. Patel
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Rush - Copley Medical Center
**State:** Illinois
**Status:** RECRUITING
**Zip:** 60504
**Location 52:**
**City:** Bloomington
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 309-243-3605
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Illinois CancerCare-Bloomington
**State:** Illinois
**Status:** RECRUITING
**Zip:** 61704
**Location 53:**
**City:** Canton
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 309-243-3605
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Illinois CancerCare-Canton
**State:** Illinois
**Status:** RECRUITING
**Zip:** 61520
**Location 54:**
**City:** Carbondale
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 618-457-5200
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Memorial Hospital of Carbondale
**State:** Illinois
**Status:** RECRUITING
**Zip:** 62902
**Location 55:**
**City:** Carterville
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 618-985-3333
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** SIH Cancer Institute
**State:** Illinois
**Status:** RECRUITING
**Zip:** 62918
**Location 56:**
**City:** Carthage
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 309-243-3605
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Illinois CancerCare-Carthage
**State:** Illinois
**Status:** RECRUITING
**Zip:** 62321
**Location 57:**
**City:** Centralia
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 217-876-4740
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Centralia Oncology Clinic
**State:** Illinois
**Status:** RECRUITING
**Zip:** 62801
**Location 58:**
**City:** Chicago
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 312-695-1301
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jessica K. Altman
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Northwestern University
**State:** Illinois
**Status:** RECRUITING
**Zip:** 60611
**Location 59:**
**City:** Chicago
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 312-355-3046
- **Role:** CONTACT
***Contact 2:***
- **Name:** Irum Khan
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** University of Illinois
**State:** Illinois
**Status:** RECRUITING
**Zip:** 60612
**Location 60:**
**City:** Danville
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 800-446-5532
- **Role:** CONTACT
***Contact 2:***
- **Name:** Priyank P. Patel
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Carle on Vermilion
**State:** Illinois
**Status:** RECRUITING
**Zip:** 61832
**Location 61:**
**City:** Decatur
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 217-876-4740
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Cancer Care Specialists of Illinois - Decatur
**State:** Illinois
**Status:** RECRUITING
**Zip:** 62526
**Location 62:**
**City:** Decatur
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 217-876-4740
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Decatur Memorial Hospital
**State:** Illinois
**Status:** RECRUITING
**Zip:** 62526
**Location 63:**
**City:** Dixon
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 815-285-7800
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Illinois CancerCare-Dixon
**State:** Illinois
**Status:** RECRUITING
**Zip:** 61021
**Location 64:**
**City:** Effingham
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 800-446-5532
- **Role:** CONTACT
***Contact 2:***
- **Name:** Priyank P. Patel
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Carle Physician Group-Effingham
**State:** Illinois
**Status:** RECRUITING
**Zip:** 62401
**Location 65:**
**City:** Effingham
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 217-876-4740
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Crossroads Cancer Center
**State:** Illinois
**Status:** RECRUITING
**Zip:** 62401
**Location 66:**
**City:** Eureka
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 309-243-3605
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Illinois CancerCare-Eureka
**State:** Illinois
**Status:** RECRUITING
**Zip:** 61530
**Location 67:**
**City:** Galesburg
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 309-243-3605
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Illinois CancerCare-Galesburg
**State:** Illinois
**Status:** RECRUITING
**Zip:** 61401
**Location 68:**
**City:** Galesburg
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 309-344-2831
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Western Illinois Cancer Treatment Center
**State:** Illinois
**Status:** RECRUITING
**Zip:** 61401
**Location 69:**
**City:** Kewanee
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 309-243-3605
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Illinois CancerCare-Kewanee Clinic
**State:** Illinois
**Status:** RECRUITING
**Zip:** 61443
**Location 70:**
**City:** Lake Forest
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jessica K. Altman
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Northwestern Medicine Lake Forest Hospital
**State:** Illinois
**Status:** RECRUITING
**Zip:** 60045
**Location 71:**
**City:** Macomb
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 309-243-3605
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Illinois CancerCare-Macomb
**State:** Illinois
**Status:** RECRUITING
**Zip:** 61455
**Location 72:**
**City:** Mattoon
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 800-446-5532
- **Role:** CONTACT
***Contact 2:***
- **Name:** Priyank P. Patel
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Carle Physician Group-Mattoon/Charleston
**State:** Illinois
**Status:** RECRUITING
**Zip:** 61938
**Location 73:**
**City:** Mount Vernon
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 618-242-4600
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jay W. Carlson
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Good Samaritan Regional Health Center
**State:** Illinois
**Status:** RECRUITING
**Zip:** 62864
**Location 74:**
**City:** O'Fallon
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 217-876-4762
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Cancer Care Center of O'Fallon
**State:** Illinois
**Status:** RECRUITING
**Zip:** 62269
**Location 75:**
**City:** Ottawa
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 309-243-3605
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Illinois CancerCare-Ottawa Clinic
**State:** Illinois
**Status:** RECRUITING
**Zip:** 61350
**Location 76:**
**City:** Pekin
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 309-243-3605
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Illinois CancerCare-Pekin
**State:** Illinois
**Status:** RECRUITING
**Zip:** 61554
**Location 77:**
**City:** Peoria
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 309-243-3605
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Illinois CancerCare-Peoria
**State:** Illinois
**Status:** RECRUITING
**Zip:** 61615
**Location 78:**
**City:** Peoria
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 309-243-3605
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Methodist Medical Center of Illinois
**State:** Illinois
**Status:** RECRUITING
**Zip:** 61636
**Location 79:**
**City:** Peru
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 309-243-3605
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Illinois CancerCare-Peru
**State:** Illinois
**Status:** RECRUITING
**Zip:** 61354
**Location 80:**
**City:** Peru
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 815-664-4141
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Valley Radiation Oncology
**State:** Illinois
**Status:** RECRUITING
**Zip:** 61354
**Location 81:**
**City:** Princeton
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 309-243-3605
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Illinois CancerCare-Princeton
**State:** Illinois
**Status:** RECRUITING
**Zip:** 61356
**Location 82:**
**City:** Springfield
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 217-545-7929
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Southern Illinois University School of Medicine
**State:** Illinois
**Status:** RECRUITING
**Zip:** 62702
**Location 83:**
**City:** Springfield
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 800-444-7541
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Springfield Clinic
**State:** Illinois
**Status:** RECRUITING
**Zip:** 62702
**Location 84:**
**City:** Springfield
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 217-788-3528
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Memorial Medical Center
**State:** Illinois
**Status:** RECRUITING
**Zip:** 62781
**Location 85:**
**City:** Swansea
**Country:** United States
**Facility:** Southwest Illinois Health Services LLP
**State:** Illinois
**Status:** SUSPENDED
**Zip:** 62226
**Location 86:**
**City:** Urbana
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 800-446-5532
- **Role:** CONTACT
***Contact 2:***
- **Name:** Priyank P. Patel
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Carle Cancer Center
**State:** Illinois
**Status:** RECRUITING
**Zip:** 61801
**Location 87:**
**City:** Urbana
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 800-446-5532
- **Role:** CONTACT
***Contact 2:***
- **Name:** Priyank P. Patel
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** The Carle Foundation Hospital
**State:** Illinois
**Status:** RECRUITING
**Zip:** 61801
**Location 88:**
**City:** Yorkville
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 630-978-6212
- **Role:** CONTACT
***Contact 2:***
- **Name:** Priyank P. Patel
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Rush-Copley Healthcare Center
**State:** Illinois
**Status:** RECRUITING
**Zip:** 60560
**Location 89:**
**City:** Ames
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 515-956-4132
- **Role:** CONTACT
***Contact 2:***
- **Name:** Debra M. Prow
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Mary Greeley Medical Center
**State:** Iowa
**Status:** RECRUITING
**Zip:** 50010
**Location 90:**
**City:** Ames
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 515-239-4734
- **Role:** CONTACT
***Contact 2:***
- **Name:** Debra M. Prow
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** McFarland Clinic PC - Ames
**State:** Iowa
**Status:** RECRUITING
**Zip:** 50010
**Location 91:**
**City:** Boone
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 515-956-4132
- **Role:** CONTACT
***Contact 2:***
- **Name:** Debra M. Prow
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** McFarland Clinic PC-Boone
**State:** Iowa
**Status:** RECRUITING
**Zip:** 50036
**Location 92:**
**City:** Clive
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Medical Oncology and Hematology Associates-West Des Moines
**State:** Iowa
**Status:** RECRUITING
**Zip:** 50325
**Location 93:**
**City:** Clive
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Mercy Cancer Center-West Lakes
**State:** Iowa
**Status:** RECRUITING
**Zip:** 50325
**Location 94:**
**City:** Council Bluffs
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Alegent Health Mercy Hospital
**State:** Iowa
**Status:** RECRUITING
**Zip:** 51503
**Location 95:**
**City:** Creston
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Greater Regional Medical Center
**State:** Iowa
**Status:** RECRUITING
**Zip:** 50801
**Location 96:**
**City:** Des Moines
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 515-241-6727
- **Role:** CONTACT
***Contact 2:***
- **Name:** Robert J. Behrens
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Iowa Methodist Medical Center
**State:** Iowa
**Status:** RECRUITING
**Zip:** 50309
**Location 97:**
**City:** Des Moines
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 515-282-2921
- **Role:** CONTACT
***Contact 2:***
- **Name:** Robert J. Behrens
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Medical Oncology and Hematology Associates-Des Moines
**State:** Iowa
**Status:** RECRUITING
**Zip:** 50309
**Location 98:**
**City:** Des Moines
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 515-282-2200
- **Role:** CONTACT
***Contact 2:***
- **Name:** Robert J. Behrens
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Broadlawns Medical Center
**State:** Iowa
**Status:** RECRUITING
**Zip:** 50314
**Location 99:**
**City:** Des Moines
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Medical Oncology and Hematology Associates-Laurel
**State:** Iowa
**Status:** RECRUITING
**Zip:** 50314
**Location 100:**
**City:** Des Moines
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Mercy Medical Center - Des Moines
**State:** Iowa
**Status:** RECRUITING
**Zip:** 50314
**Location 101:**
**City:** Des Moines
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 515-241-8704
- **Role:** CONTACT
***Contact 2:***
- **Name:** Robert J. Behrens
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Iowa Lutheran Hospital
**State:** Iowa
**Status:** RECRUITING
**Zip:** 50316
**Location 102:**
**City:** Fort Dodge
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 515-956-4132
- **Role:** CONTACT
***Contact 2:***
- **Name:** Debra M. Prow
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** McFarland Clinic PC-Trinity Cancer Center
**State:** Iowa
**Status:** RECRUITING
**Zip:** 50501
**Location 103:**
**City:** Fort Dodge
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 515-574-8302
- **Role:** CONTACT
***Contact 2:***
- **Name:** Robert J. Behrens
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Trinity Regional Medical Center
**State:** Iowa
**Status:** RECRUITING
**Zip:** 50501
**Location 104:**
**City:** Jefferson
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 515-956-4132
- **Role:** CONTACT
***Contact 2:***
- **Name:** Debra M. Prow
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** McFarland Clinic PC-Jefferson
**State:** Iowa
**Status:** RECRUITING
**Zip:** 50129
**Location 105:**
**City:** Marshalltown
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 515-956-4132
- **Role:** CONTACT
***Contact 2:***
- **Name:** Debra M. Prow
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** McFarland Clinic PC-Marshalltown
**State:** Iowa
**Status:** RECRUITING
**Zip:** 50158
**Location 106:**
**City:** West Des Moines
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 515-343-1000
- **Role:** CONTACT
***Contact 2:***
- **Name:** Robert J. Behrens
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Methodist West Hospital
**State:** Iowa
**Status:** RECRUITING
**Zip:** 50266-7700
**Location 107:**
**City:** West Des Moines
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Mercy Medical Center-West Lakes
**State:** Iowa
**Status:** RECRUITING
**Zip:** 50266
**Location 108:**
**City:** Lawrence
**Country:** United States
**Facility:** Lawrence Memorial Hospital
**State:** Kansas
**Status:** SUSPENDED
**Zip:** 66044
**Location 109:**
**City:** Wichita
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 316-268-5374
- **Role:** CONTACT
***Contact 2:***
- **Name:** Shaker R. Dakhil
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Cancer Center of Kansas-Wichita Medical Arts Tower
**State:** Kansas
**Status:** RECRUITING
**Zip:** 67208
**Location 110:**
**City:** Wichita
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 800-362-0070
- **Role:** CONTACT
***Contact 2:***
- **Name:** Shaker R. Dakhil
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Ascension Via Christi Hospitals Wichita
**State:** Kansas
**Status:** RECRUITING
**Zip:** 67214
**Location 111:**
**City:** Wichita
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 316-268-5374
- **Role:** CONTACT
***Contact 2:***
- **Name:** Shaker R. Dakhil
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Cancer Center of Kansas - Wichita
**State:** Kansas
**Status:** RECRUITING
**Zip:** 67214
**Location 112:**
**City:** Wichita
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 316-268-5374
- **Role:** CONTACT
***Contact 2:***
- **Name:** Shaker R. Dakhil
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Wesley Medical Center
**State:** Kansas
**Status:** RECRUITING
**Zip:** 67214
**Location 113:**
**City:** Bardstown
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Flaget Memorial Hospital
**State:** Kentucky
**Status:** RECRUITING
**Zip:** 40004
**Location 114:**
**City:** Corbin
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Commonwealth Cancer Center-Corbin
**State:** Kentucky
**Status:** RECRUITING
**Zip:** 40701
**Location 115:**
**City:** Lexington
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Saint Joseph Radiation Oncology Resource Center
**State:** Kentucky
**Status:** RECRUITING
**Zip:** 40504
**Location 116:**
**City:** Lexington
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Saint Joseph Hospital East
**State:** Kentucky
**Status:** RECRUITING
**Zip:** 40509
**Location 117:**
**City:** London
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Saint Joseph London
**State:** Kentucky
**Status:** RECRUITING
**Zip:** 40741
**Location 118:**
**City:** Louisville
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Jewish Hospital
**State:** Kentucky
**Status:** RECRUITING
**Zip:** 40202
**Location 119:**
**City:** Louisville
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Saints Mary and Elizabeth Hospital
**State:** Kentucky
**Status:** RECRUITING
**Zip:** 40215
**Location 120:**
**City:** Louisville
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Jewish Hospital Medical Center Northeast
**State:** Kentucky
**Status:** RECRUITING
**Zip:** 40245
**Location 121:**
**City:** Shepherdsville
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Jewish Hospital Medical Center South
**State:** Kentucky
**Status:** RECRUITING
**Zip:** 40165
**Location 122:**
**City:** Baltimore
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 410-368-2910
- **Role:** CONTACT
***Contact 2:***
- **Name:** Carole B. Miller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Saint Agnes Hospital
**State:** Maryland
**Status:** RECRUITING
**Zip:** 21229
**Location 123:**
**City:** Adrian
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 517-265-0116
- **Role:** CONTACT
***Contact 2:***
- **Name:** Rex B. Mowat
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Hickman Cancer Center
**State:** Michigan
**Status:** RECRUITING
**Zip:** 49221
**Location 124:**
**City:** Monroe
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 800-444-3561
- **Role:** CONTACT
***Contact 2:***
- **Name:** Rex B. Mowat
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Toledo Clinic Cancer Centers-Monroe
**State:** Michigan
**Status:** RECRUITING
**Zip:** 48162
**Location 125:**
**City:** Rochester
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 855-776-0015
- **Role:** CONTACT
***Contact 2:***
- **Name:** Mark R. Litzow
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Mayo Clinic in Rochester
**State:** Minnesota
**Status:** RECRUITING
**Zip:** 55905
**Location 126:**
**City:** Saint Cloud
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 877-229-4907
- **Role:** CONTACT
***Contact 2:***
- **Name:** Donald J. Jurgens
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Coborn Cancer Center at Saint Cloud Hospital
**State:** Minnesota
**Status:** RECRUITING
**Zip:** 56303
**Location 127:**
**City:** Ballwin
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 314-251-7058
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jay W. Carlson
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Saint Louis Cancer and Breast Institute-Ballwin
**State:** Missouri
**Status:** RECRUITING
**Zip:** 63011
**Location 128:**
**City:** Bonne Terre
**Country:** United States
**Facility:** Parkland Health Center-Bonne Terre
**State:** Missouri
**Status:** SUSPENDED
**Zip:** 63628
**Location 129:**
**City:** Branson
**Country:** United States
**Facility:** Cox Cancer Center Branson
**State:** Missouri
**Status:** SUSPENDED
**Zip:** 65616
**Location 130:**
**City:** Cape Girardeau
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 573-334-2230
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Saint Francis Medical Center
**State:** Missouri
**Status:** RECRUITING
**Zip:** 63703
**Location 131:**
**City:** Cape Girardeau
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 573-651-5550
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Southeast Cancer Center
**State:** Missouri
**Status:** RECRUITING
**Zip:** 63703
**Location 132:**
**City:** Farmington
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 314-996-5569
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Parkland Health Center - Farmington
**State:** Missouri
**Status:** RECRUITING
**Zip:** 63640
**Location 133:**
**City:** Jefferson City
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 573-632-4814
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Capital Region Southwest Campus
**State:** Missouri
**Status:** RECRUITING
**Zip:** 65109
**Location 134:**
**City:** Joplin
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 417-347-4030
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jay W. Carlson
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Freeman Health System
**State:** Missouri
**Status:** RECRUITING
**Zip:** 64804
**Location 135:**
**City:** Joplin
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 417-556-3074
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jay W. Carlson
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Mercy Hospital Joplin
**State:** Missouri
**Status:** RECRUITING
**Zip:** 64804
**Location 136:**
**City:** Rolla
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 573-458-8776
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jay W. Carlson
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Delbert Day Cancer Institute at PCRMC
**State:** Missouri
**Status:** RECRUITING
**Zip:** 65401
**Location 137:**
**City:** Rolla
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 573-458-6379
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jay W. Carlson
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Mercy Clinic-Rolla-Cancer and Hematology
**State:** Missouri
**Status:** RECRUITING
**Zip:** 65401
**Location 138:**
**City:** Saint Joseph
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 816-271-7937
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jay W. Carlson
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Heartland Regional Medical Center
**State:** Missouri
**Status:** RECRUITING
**Zip:** 64506
**Location 139:**
**City:** Saint Louis
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 314-353-1870
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jay W. Carlson
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Saint Louis Cancer and Breast Institute-South City
**State:** Missouri
**Status:** RECRUITING
**Zip:** 63109
**Location 140:**
**City:** Saint Louis
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jay W. Carlson
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Mercy Hospital South
**State:** Missouri
**Status:** RECRUITING
**Zip:** 63128
**Location 141:**
**City:** Saint Louis
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 314-996-5569
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Missouri Baptist Medical Center
**State:** Missouri
**Status:** RECRUITING
**Zip:** 63131
**Location 142:**
**City:** Saint Louis
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 314-251-7066
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jay W. Carlson
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Mercy Hospital Saint Louis
**State:** Missouri
**Status:** RECRUITING
**Zip:** 63141
**Location 143:**
**City:** Sainte Genevieve
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 314-996-5569
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Sainte Genevieve County Memorial Hospital
**State:** Missouri
**Status:** RECRUITING
**Zip:** 63670
**Location 144:**
**City:** Springfield
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 417-269-4520
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jay W. Carlson
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Mercy Hospital Springfield
**State:** Missouri
**Status:** RECRUITING
**Zip:** 65804
**Location 145:**
**City:** Springfield
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 417-269-4520
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jay W. Carlson
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** CoxHealth South Hospital
**State:** Missouri
**Status:** RECRUITING
**Zip:** 65807
**Location 146:**
**City:** Sullivan
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 314-996-5569
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Missouri Baptist Sullivan Hospital
**State:** Missouri
**Status:** RECRUITING
**Zip:** 63080
**Location 147:**
**City:** Sunset Hills
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 314-996-5569
- **Role:** CONTACT
***Contact 2:***
- **Name:** Bryan A. Faller
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Missouri Baptist Outpatient Center-Sunset Hills
**State:** Missouri
**Status:** RECRUITING
**Zip:** 63127
**Location 148:**
**City:** Washington
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 636-390-1600
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jay W. Carlson
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Mercy Hospital Washington
**State:** Missouri
**Status:** RECRUITING
**Zip:** 63090
**Location 149:**
**City:** Anaconda
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 406-969-6060
- **Role:** CONTACT
***Contact 2:***
- **Name:** John M. Schallenkamp
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Community Hospital of Anaconda
**State:** Montana
**Status:** RECRUITING
**Zip:** 59711
**Location 150:**
**City:** Billings
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 800-996-2663
- **Role:** CONTACT
***Contact 2:***
- **Name:** John M. Schallenkamp
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Billings Clinic Cancer Center
**State:** Montana
**Status:** RECRUITING
**Zip:** 59101
**Location 151:**
**City:** Bozeman
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 406-969-6060
- **Role:** CONTACT
***Contact 2:***
- **Name:** John M. Schallenkamp
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Bozeman Deaconess Hospital
**State:** Montana
**Status:** RECRUITING
**Zip:** 59715
**Location 152:**
**City:** Great Falls
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 406-969-6060
- **Role:** CONTACT
***Contact 2:***
- **Name:** John M. Schallenkamp
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Benefis Healthcare- Sletten Cancer Institute
**State:** Montana
**Status:** RECRUITING
**Zip:** 59405
**Location 153:**
**City:** Great Falls
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 406-969-6060
- **Role:** CONTACT
***Contact 2:***
- **Name:** John M. Schallenkamp
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Great Falls Clinic
**State:** Montana
**Status:** RECRUITING
**Zip:** 59405
**Location 154:**
**City:** Helena
**Country:** United States
**Facility:** Saint Peter's Community Hospital
**State:** Montana
**Status:** SUSPENDED
**Zip:** 59601
**Location 155:**
**City:** Kalispell
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 406-969-6060
- **Role:** CONTACT
***Contact 2:***
- **Name:** John M. Schallenkamp
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Kalispell Regional Medical Center
**State:** Montana
**Status:** RECRUITING
**Zip:** 59901
**Location 156:**
**City:** Missoula
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 406-969-6060
- **Role:** CONTACT
***Contact 2:***
- **Name:** John M. Schallenkamp
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Community Medical Hospital
**State:** Montana
**Status:** RECRUITING
**Zip:** 59804
**Location 157:**
**City:** Bellevue
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 402-559-6941
- **Role:** CONTACT
***Contact 2:***
- **Name:** Vijaya R. Bhatt
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Nebraska Medicine-Bellevue
**State:** Nebraska
**Status:** RECRUITING
**Zip:** 68123
**Location 158:**
**City:** Grand Island
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** CHI Health Saint Francis
**State:** Nebraska
**Status:** RECRUITING
**Zip:** 68803
**Location 159:**
**City:** Kearney
**Country:** United States
**Facility:** Heartland Hematology and Oncology
**State:** Nebraska
**Status:** SUSPENDED
**Zip:** 68845
**Location 160:**
**City:** Kearney
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** CHI Health Good Samaritan
**State:** Nebraska
**Status:** RECRUITING
**Zip:** 68847
**Location 161:**
**City:** Lincoln
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Saint Elizabeth Regional Medical Center
**State:** Nebraska
**Status:** RECRUITING
**Zip:** 68510
**Location 162:**
**City:** Omaha
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 402-559-5600
- **Role:** CONTACT
***Contact 2:***
- **Name:** Vijaya R. Bhatt
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Nebraska Medicine-Village Pointe
**State:** Nebraska
**Status:** RECRUITING
**Zip:** 68118
**Location 163:**
**City:** Omaha
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Alegent Health Immanuel Medical Center
**State:** Nebraska
**Status:** RECRUITING
**Zip:** 68122
**Location 164:**
**City:** Omaha
**Country:** United States
**Facility:** Hematology and Oncology Consultants PC
**State:** Nebraska
**Status:** SUSPENDED
**Zip:** 68122
**Location 165:**
**City:** Omaha
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Alegent Health Bergan Mercy Medical Center
**State:** Nebraska
**Status:** RECRUITING
**Zip:** 68124
**Location 166:**
**City:** Omaha
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Alegent Health Lakeside Hospital
**State:** Nebraska
**Status:** RECRUITING
**Zip:** 68130
**Location 167:**
**City:** Omaha
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Creighton University Medical Center
**State:** Nebraska
**Status:** RECRUITING
**Zip:** 68131
**Location 168:**
**City:** Omaha
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 402-559-6941
- **Role:** CONTACT
***Contact 2:***
- **Name:** Vijaya R. Bhatt
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** University of Nebraska Medical Center
**State:** Nebraska
**Status:** RECRUITING
**Zip:** 68198
**Location 169:**
**City:** Papillion
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Midlands Community Hospital
**State:** Nebraska
**Status:** RECRUITING
**Zip:** 68046
**Location 170:**
**City:** Morristown
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 973-971-5900
- **Role:** CONTACT
***Contact 2:***
- **Name:** Charles M. Farber
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Morristown Medical Center
**State:** New Jersey
**Status:** RECRUITING
**Zip:** 07960
**Location 171:**
**City:** Middletown
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 845-333-1133
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jeffrey M. Stewart
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Garnet Health Medical Center
**State:** New York
**Status:** RECRUITING
**Zip:** 10940
**Location 172:**
**City:** Clinton
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 919-587-9084
- **Role:** CONTACT
***Contact 2:***
- **Name:** Samer S. Kasbari
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Southeastern Medical Oncology Center-Clinton
**State:** North Carolina
**Status:** RECRUITING
**Zip:** 28328
**Location 173:**
**City:** Goldsboro
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 919-587-9084
- **Role:** CONTACT
***Contact 2:***
- **Name:** Samer S. Kasbari
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Southeastern Medical Oncology Center-Goldsboro
**State:** North Carolina
**Status:** RECRUITING
**Zip:** 27534
**Location 174:**
**City:** Goldsboro
**Country:** United States
**Facility:** Wayne Memorial Hospital
**State:** North Carolina
**Status:** ACTIVE_NOT_RECRUITING
**Zip:** 27534
**Location 175:**
**City:** Jacksonville
**Country:** United States
**Facility:** Onslow Memorial Hospital
**State:** North Carolina
**Status:** ACTIVE_NOT_RECRUITING
**Zip:** 28546
**Location 176:**
**City:** Jacksonville
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 910-587-9084
- **Role:** CONTACT
***Contact 2:***
- **Name:** Samer S. Kasbari
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Southeastern Medical Oncology Center-Jacksonville
**State:** North Carolina
**Status:** RECRUITING
**Zip:** 28546
**Location 177:**
**City:** Cincinnati
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Good Samaritan Hospital - Cincinnati
**State:** Ohio
**Status:** RECRUITING
**Zip:** 45220
**Location 178:**
**City:** Cincinnati
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Bethesda North Hospital
**State:** Ohio
**Status:** RECRUITING
**Zip:** 45242
**Location 179:**
**City:** Cincinnati
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** TriHealth Cancer Institute-Westside
**State:** Ohio
**Status:** RECRUITING
**Zip:** 45247
**Location 180:**
**City:** Cincinnati
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** TriHealth Cancer Institute-Anderson
**State:** Ohio
**Status:** RECRUITING
**Zip:** 45255
**Location 181:**
**City:** Cleveland
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 216-778-8526
- **Role:** CONTACT
***Contact 2:***
- **Name:** William Tse
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** MetroHealth Medical Center
**State:** Ohio
**Status:** RECRUITING
**Zip:** 44109
**Location 182:**
**City:** Perrysburg
**Country:** United States
**Facility:** Mercy Health Perrysburg Cancer Center
**State:** Ohio
**Status:** SUSPENDED
**Zip:** 43551
**Location 183:**
**City:** Toledo
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 614-488-2118
- **Role:** CONTACT
***Contact 2:***
- **Name:** Rex B. Mowat
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Mercy Health - Saint Anne Hospital
**State:** Ohio
**Status:** RECRUITING
**Zip:** 43623
**Location 184:**
**City:** Toledo
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 800-444-3561
- **Role:** CONTACT
***Contact 2:***
- **Name:** Rex B. Mowat
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Toledo Clinic Cancer Centers-Toledo
**State:** Ohio
**Status:** RECRUITING
**Zip:** 43623
**Location 185:**
**City:** Lawton
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 877-231-4440
- **Role:** CONTACT
***Contact 2:***
- **Name:** Mohamad Khawandanah
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Cancer Centers of Southwest Oklahoma Research
**State:** Oklahoma
**Status:** RECRUITING
**Zip:** 73505
**Location 186:**
**City:** Oklahoma City
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 405-271-8777
- **Role:** CONTACT
***Contact 2:***
- **Name:** Mohamad Khawandanah
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** University of Oklahoma Health Sciences Center
**State:** Oklahoma
**Status:** RECRUITING
**Zip:** 73104
**Location 187:**
**City:** Oklahoma City
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 405-752-3402
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jay W. Carlson
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Mercy Hospital Oklahoma City
**State:** Oklahoma
**Status:** RECRUITING
**Zip:** 73120
**Location 188:**
**City:** Tulsa
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 918-505-3200
- **Role:** CONTACT
***Contact 2:***
- **Name:** Mohamad Khawandanah
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Oklahoma Cancer Specialists and Research Institute-Tulsa
**State:** Oklahoma
**Status:** RECRUITING
**Zip:** 74146
**Location 189:**
**City:** Baker City
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 734-712-3671
- **Role:** CONTACT
***Contact 2:***
- **Name:** John M. Schallenkamp
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Saint Alphonsus Medical Center-Baker City
**State:** Oregon
**Status:** RECRUITING
**Zip:** 97814
**Location 190:**
**City:** Ontario
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 734-712-3671
- **Role:** CONTACT
***Contact 2:***
- **Name:** John M. Schallenkamp
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Saint Alphonsus Medical Center-Ontario
**State:** Oregon
**Status:** RECRUITING
**Zip:** 97914
**Location 191:**
**City:** Chadds Ford
**Country:** United States
**Facility:** Christiana Care Health System-Concord Health Center
**State:** Pennsylvania
**Status:** ACTIVE_NOT_RECRUITING
**Zip:** 19317
**Location 192:**
**City:** Danville
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 570-271-5251
- **Role:** CONTACT
***Contact 2:***
- **Name:** Joseph J. Vadakara
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Geisinger Medical Center
**State:** Pennsylvania
**Status:** RECRUITING
**Zip:** 17822
**Location 193:**
**City:** Hazleton
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 570-459-2901
- **Role:** CONTACT
***Contact 2:***
- **Name:** Joseph J. Vadakara
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Geisinger Medical Center-Cancer Center Hazleton
**State:** Pennsylvania
**Status:** RECRUITING
**Zip:** 18201
**Location 194:**
**City:** Lewisburg
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 570-374-8555
- **Role:** CONTACT
***Contact 2:***
- **Name:** Joseph J. Vadakara
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Geisinger Medical Oncology-Lewisburg
**State:** Pennsylvania
**Status:** RECRUITING
**Zip:** 17837
**Location 195:**
**City:** Lewistown
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 717-242-7703
- **Role:** CONTACT
***Contact 2:***
- **Name:** Joseph J. Vadakara
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Lewistown Hospital
**State:** Pennsylvania
**Status:** RECRUITING
**Zip:** 17044
**Location 196:**
**City:** Pottsville
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 800-275-6401
- **Role:** CONTACT
***Contact 2:***
- **Name:** Joseph J. Vadakara
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Geisinger Cancer Services-Pottsville
**State:** Pennsylvania
**Status:** RECRUITING
**Zip:** 17901
**Location 197:**
**City:** Scranton
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 570-703-4768
- **Role:** CONTACT
***Contact 2:***
- **Name:** Joseph J. Vadakara
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Community Medical Center
**State:** Pennsylvania
**Status:** RECRUITING
**Zip:** 18510
**Location 198:**
**City:** Selinsgrove
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 570-374-8555
- **Role:** CONTACT
***Contact 2:***
- **Name:** Joseph J. Vadakara
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Geisinger Medical Oncology-Selinsgrove
**State:** Pennsylvania
**Status:** RECRUITING
**Zip:** 17870
**Location 199:**
**City:** State College
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 570-271-5251
- **Role:** CONTACT
***Contact 2:***
- **Name:** Joseph J. Vadakara
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Geisinger Medical Group
**State:** Pennsylvania
**Status:** RECRUITING
**Zip:** 16801
**Location 200:**
**City:** Wilkes-Barre
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 570-271-5251
- **Role:** CONTACT
***Contact 2:***
- **Name:** Joseph J. Vadakara
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Geisinger Wyoming Valley/Henry Cancer Center
**State:** Pennsylvania
**Status:** RECRUITING
**Zip:** 18711
**Location 201:**
**City:** Boiling Springs
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 864-522-2066
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jeffrey K. Giguere
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Prisma Health Cancer Institute - Spartanburg
**State:** South Carolina
**Status:** RECRUITING
**Zip:** 29316
**Location 202:**
**City:** Clinton
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 864-522-2066
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jeffrey K. Giguere
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Prisma Health Cancer Institute - Laurens
**State:** South Carolina
**Status:** RECRUITING
**Zip:** 29325
**Location 203:**
**City:** Easley
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 864-522-2066
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jeffrey K. Giguere
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Prisma Health Cancer Institute - Easley
**State:** South Carolina
**Status:** RECRUITING
**Zip:** 29640
**Location 204:**
**City:** Greenville
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 864-522-2066
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jeffrey K. Giguere
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** BI-LO Charities Children's Cancer Center
**State:** South Carolina
**Status:** RECRUITING
**Zip:** 29605
**Location 205:**
**City:** Greenville
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 864-522-2066
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jeffrey K. Giguere
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Prisma Health Cancer Institute - Butternut
**State:** South Carolina
**Status:** RECRUITING
**Zip:** 29605
**Location 206:**
**City:** Greenville
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 864-522-2066
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jeffrey K. Giguere
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Prisma Health Cancer Institute - Faris
**State:** South Carolina
**Status:** RECRUITING
**Zip:** 29605
**Location 207:**
**City:** Greenville
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 864-522-2066
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jeffrey K. Giguere
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Prisma Health Greenville Memorial Hospital
**State:** South Carolina
**Status:** RECRUITING
**Zip:** 29605
**Location 208:**
**City:** Greenville
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 864-522-2066
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jeffrey K. Giguere
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Prisma Health Cancer Institute - Eastside
**State:** South Carolina
**Status:** RECRUITING
**Zip:** 29615
**Location 209:**
**City:** Greer
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 864-522-2066
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jeffrey K. Giguere
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Prisma Health Cancer Institute - Greer
**State:** South Carolina
**Status:** RECRUITING
**Zip:** 29650
**Location 210:**
**City:** Seneca
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 864-522-2066
- **Role:** CONTACT
***Contact 2:***
- **Name:** Jeffrey K. Giguere
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Prisma Health Cancer Institute - Seneca
**State:** South Carolina
**Status:** RECRUITING
**Zip:** 29672
**Location 211:**
**City:** Chattanooga
**Country:** United States
**Facility:** Memorial Hospital
**State:** Tennessee
**Status:** SUSPENDED
**Zip:** 37404
**Location 212:**
**City:** Hixson
**Country:** United States
**Facility:** Pulmonary Medicine Center of Chattanooga-Hixson
**State:** Tennessee
**Status:** SUSPENDED
**Zip:** 37343
**Location 213:**
**City:** Ooltewah
**Country:** United States
**Facility:** Memorial GYN Plus
**State:** Tennessee
**Status:** SUSPENDED
**Zip:** 37363
**Location 214:**
**City:** Bryan
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Saint Joseph Regional Cancer Center
**State:** Texas
**Status:** RECRUITING
**Zip:** 77802
**Location 215:**
**City:** Martinsville
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 276-666-7489
- **Role:** CONTACT
***Contact 2:***
- **Name:** Mohammed Saad
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Sovah Health Martinsville
**State:** Virginia
**Status:** RECRUITING
**Zip:** 24115
**Location 216:**
**City:** Bremerton
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Harrison HealthPartners Hematology and Oncology-Bremerton
**State:** Washington
**Status:** RECRUITING
**Zip:** 98310
**Location 217:**
**City:** Bremerton
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Harrison Medical Center
**State:** Washington
**Status:** RECRUITING
**Zip:** 98310
**Location 218:**
**City:** Burien
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Highline Medical Center-Main Campus
**State:** Washington
**Status:** RECRUITING
**Zip:** 98166
**Location 219:**
**City:** Enumclaw
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Saint Elizabeth Hospital
**State:** Washington
**Status:** RECRUITING
**Zip:** 98022
**Location 220:**
**City:** Federal Way
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Saint Francis Hospital
**State:** Washington
**Status:** RECRUITING
**Zip:** 98003
**Location 221:**
**City:** Lakewood
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Saint Clare Hospital
**State:** Washington
**Status:** RECRUITING
**Zip:** 98499
**Location 222:**
**City:** Poulsbo
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Harrison HealthPartners Hematology and Oncology-Poulsbo
**State:** Washington
**Status:** RECRUITING
**Zip:** 98370
**Location 223:**
**City:** Tacoma
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 308-398-6518
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Franciscan Research Center-Northwest Medical Plaza
**State:** Washington
**Status:** RECRUITING
**Zip:** 98405
**Location 224:**
**City:** Tacoma
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 253-306-0532
- **Role:** CONTACT
***Contact 2:***
- **Name:** Richard L. Deming
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Northwest Medical Specialties PLLC
**State:** Washington
**Status:** RECRUITING
**Zip:** 98405
**Location 225:**
**City:** Morgantown
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 304-293-7374
- **Role:** CONTACT
***Contact 2:***
- **Name:** Kelly G. Ross
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** West Virginia University Healthcare
**State:** West Virginia
**Status:** RECRUITING
**Zip:** 26506
**Location 226:**
**City:** Eau Claire
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 800-782-8581
- **Role:** CONTACT
***Contact 2:***
- **Name:** Ali W. Bseiso
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Marshfield Medical Center-EC Cancer Center
**State:** Wisconsin
**Status:** RECRUITING
**Zip:** 54701
**Location 227:**
**City:** Eau Claire
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 855-776-0015
- **Role:** CONTACT
***Contact 2:***
- **Name:** Mark R. Litzow
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Mayo Clinic Health System-Eau Claire Clinic
**State:** Wisconsin
**Status:** RECRUITING
**Zip:** 54701
**Location 228:**
**City:** Eau Claire
**Contacts:**
***Contact 1:***
- **Name:** Site Public Contact
- **Phone:** 855-776-0015
- **Role:** CONTACT
***Contact 2:***
- **Name:** Mark R. Litzow
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Mayo Clinic Health System Eau Claire Hospital-Luther Campus
**State:** Wisconsin
**Status:** RECRUITING
**Zip:** 54703
**Location 229:**
**City:** Ladysmith
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 800-782-8581
- **Role:** CONTACT
***Contact 2:***
- **Name:** Ali W. Bseiso
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Marshfield Clinic - Ladysmith Center
**State:** Wisconsin
**Status:** RECRUITING
**Zip:** 54848
**Location 230:**
**City:** Marshfield
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 800-782-8581
- **Role:** CONTACT
***Contact 2:***
- **Name:** Ali W. Bseiso
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Marshfield Medical Center-Marshfield
**State:** Wisconsin
**Status:** RECRUITING
**Zip:** 54449
**Location 231:**
**City:** Minocqua
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 800-782-8581
- **Role:** CONTACT
***Contact 2:***
- **Name:** Ali W. Bseiso
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Marshfield Clinic-Minocqua Center
**State:** Wisconsin
**Status:** RECRUITING
**Zip:** 54548
**Location 232:**
**City:** Rice Lake
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 800-782-8581
- **Role:** CONTACT
***Contact 2:***
- **Name:** Ali W. Bseiso
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Marshfield Medical Center-Rice Lake
**State:** Wisconsin
**Status:** RECRUITING
**Zip:** 54868
**Location 233:**
**City:** Stevens Point
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 800-782-8581
- **Role:** CONTACT
***Contact 2:***
- **Name:** Ali W. Bseiso
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Marshfield Clinic Stevens Point Center
**State:** Wisconsin
**Status:** RECRUITING
**Zip:** 54482
**Location 234:**
**City:** Wausau
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 800-782-8581
- **Role:** CONTACT
***Contact 2:***
- **Name:** Ali W. Bseiso
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Marshfield Clinic-Wausau Center
**State:** Wisconsin
**Status:** RECRUITING
**Zip:** 54401
**Location 235:**
**City:** Weston
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 800-782-8581
- **Role:** CONTACT
***Contact 2:***
- **Name:** Ali W. Bseiso
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Marshfield Medical Center - Weston
**State:** Wisconsin
**Status:** RECRUITING
**Zip:** 54476
**Location 236:**
**City:** Wisconsin Rapids
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 800-782-8581
- **Role:** CONTACT
***Contact 2:***
- **Name:** Ali W. Bseiso
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Marshfield Clinic - Wisconsin Rapids Center
**State:** Wisconsin
**Status:** RECRUITING
**Zip:** 54494
**Location 237:**
**City:** Cody
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 800-996-2663
- **Role:** CONTACT
***Contact 2:***
- **Name:** John M. Schallenkamp
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Billings Clinic-Cody
**State:** Wyoming
**Status:** RECRUITING
**Zip:** 82414
**Location 238:**
**City:** Sheridan
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Site Public Contact
- **Phone:** 406-969-6060
- **Role:** CONTACT
***Contact 2:***
- **Name:** John M. Schallenkamp
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** United States
**Facility:** Welch Cancer Center
**State:** Wyoming
**Status:** RECRUITING
**Zip:** 82801
#### Overall Officials
**Official 1:**
**Affiliation:** ECOG-ACRIN Cancer Research Group
**Name:** Amer Zeidan
**Role:** PRINCIPAL_INVESTIGATOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000009370
- Term: Neoplasms by Histologic Type
- ID: D000009369
- Term: Neoplasms
- ID: D000006402
- Term: Hematologic Diseases
- ID: D000009196
- Term: Myeloproliferative Disorders
- ID: D000001855
- Term: Bone Marrow Diseases
- ID: D000002908
- Term: Chronic Disease
- ID: D000020969
- Term: Disease Attributes
- ID: D000010335
- Term: Pathologic Processes
- ID: D000009385
- Term: Neoplastic Processes
### Condition Browse Module - Browse Branches
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: BC15
- Name: Blood and Lymph Conditions
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M10945
- Name: Leukemia
- Relevance: HIGH
- As Found: Leukemia
- ID: M10955
- Name: Leukemia, Myeloid
- Relevance: HIGH
- As Found: Myelogenous Leukemia
- ID: M18123
- Name: Leukemia, Myelogenous, Chronic, BCR-ABL Positive
- Relevance: HIGH
- As Found: Chronic Myelogenous Leukemia, BCR-ABL1 Positive
- ID: M18125
- Name: Leukemia, Myeloid, Chronic-Phase
- Relevance: HIGH
- As Found: Chronic Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive
- ID: M20497
- Name: Neoplasm, Residual
- Relevance: HIGH
- As Found: Minimal Residual Disease
- ID: M12315
- Name: Neoplasms by Histologic Type
- Relevance: LOW
- As Found: Unknown
- ID: M9490
- Name: Hematologic Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M12149
- Name: Myeloproliferative Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M5134
- Name: Bone Marrow Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M6147
- Name: Chronic Disease
- Relevance: LOW
- As Found: Unknown
- ID: M22700
- Name: Disease Attributes
- Relevance: LOW
- As Found: Unknown
- ID: M12330
- Name: Neoplastic Processes
- Relevance: LOW
- As Found: Unknown
- ID: T3995
- Name: Myeloid Leukemia
- Relevance: HIGH
- As Found: Myeloid Leukemia
- ID: T1303
- Name: Chronic Graft Versus Host Disease
- Relevance: HIGH
- As Found: Chronic
- ID: T1309
- Name: Chronic Myeloid Leukemia
- Relevance: HIGH
- As Found: Chronic Myeloid Leukemia
- ID: T1311
- Name: Chronic Myeloproliferative Disorders
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000007938
- Term: Leukemia
- ID: D000007951
- Term: Leukemia, Myeloid
- ID: D000015464
- Term: Leukemia, Myelogenous, Chronic, BCR-ABL Positive
- ID: D000018365
- Term: Neoplasm, Residual
- ID: D000015466
- Term: Leukemia, Myeloid, Chronic-Phase
### Intervention Browse Module - Ancestors
- ID: D000074322
- Term: Antineoplastic Agents, Immunological
- ID: D000000970
- Term: Antineoplastic Agents
- ID: D000082082
- Term: Immune Checkpoint Inhibitors
- ID: D000045504
- Term: Molecular Mechanisms of Pharmacological Action
- ID: D000092004
- Term: Tyrosine Kinase Inhibitors
- ID: D000047428
- Term: Protein Kinase Inhibitors
- ID: D000004791
- Term: Enzyme Inhibitors
### Intervention Browse Module - Browse Branches
- Abbrev: ANeo
- Name: Antineoplastic Agents
- Abbrev: All
- Name: All Drugs and Chemicals
- Abbrev: AA
- Name: Amino Acids
### Intervention Browse Module - Browse Leaves
- ID: M349416
- Name: Pembrolizumab
- Relevance: HIGH
- As Found: Blind
- ID: M145649
- Name: Nilotinib
- Relevance: HIGH
- As Found: Cross-sectional
- ID: M410
- Name: Dasatinib
- Relevance: HIGH
- As Found: Web-
- ID: M2889
- Name: Tyrosine Kinase Inhibitors
- Relevance: LOW
- As Found: Unknown
- ID: M324
- Name: Imatinib Mesylate
- Relevance: HIGH
- As Found: Voiding
- ID: M1346
- Name: Antineoplastic Agents, Immunological
- Relevance: LOW
- As Found: Unknown
- ID: M2342
- Name: Immune Checkpoint Inhibitors
- Relevance: LOW
- As Found: Unknown
- ID: M25820
- Name: Protein Kinase Inhibitors
- Relevance: LOW
- As Found: Unknown
- ID: M7951
- Name: Enzyme Inhibitors
- Relevance: LOW
- As Found: Unknown
- ID: T22
- Name: Tyrosine
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: C000582435
- Term: Pembrolizumab
- ID: D000068877
- Term: Imatinib Mesylate
- ID: D000069439
- Term: Dasatinib
- ID: C000498826
- Term: Nilotinib
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT02569879
**Brief Title:** Impact of Boostrix™ Maternal Vaccination on Morbidity and Mortality of Pertussis Disease in Infants ≤6 Weeks of Age, in Bogota, Colombia.
**Official Title:** Impact of Boostrix™ Maternal Vaccination on Morbidity and Mortality of Pertussis Disease in Infants ≤6 Weeks of Age, in Bogota, Colombia.
#### Organization Study ID Info
**ID:** 201521
#### Organization
**Class:** INDUSTRY
**Full Name:** GlaxoSmithKline
### Status Module
#### Completion Date
**Date:** 2018-04-14
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-02-22
**Type:** ACTUAL
**Last Update Submit Date:** 2024-02-20
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2018-04-14
**Type:** ACTUAL
#### Start Date
**Date:** 2015-10-01
**Type:** ACTUAL
**Status Verified Date:** 2024-02
#### Study First Post Date
**Date:** 2015-10-07
**Type:** ESTIMATED
**Study First Submit Date:** 2015-10-01
**Study First Submit QC Date:** 2015-10-05
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** GlaxoSmithKline
#### Responsible Party
**Type:** SPONSOR
### Description Module
**Brief Summary:** This study is being conducted to assess impact of maternal immunisation against pertussis in infants ≤12 months of age before and after introduction of pertussis maternal immunisation in Bogota, Colombia from January 2005-December 2014.
**Detailed Description:** An observational, retrospective, ecological database study which involves systematic screening of the national databases of Bogota, to study pertussis related morbidity and mortality in infants ≤ 12 months of age before and after introduction of pertussis maternal immunisation in Bogota, Colombia from January 2005-December 2014.
### Conditions Module
**Conditions:**
- Diphtheria
- Acellular Pertussis
- Tetanus
- Aspergillosis, Allergic Bronchopulmonary
- Diphtheria-Tetanus-acellular Pertussis Vaccines
**Keywords:**
- Boostrix™
- Bagota
- maternal vaccination
- Pertussis
- Mortality
- Morbidity
### Design Module
#### Design Info
**Observational Model:** ECOLOGIC_OR_COMMUNITY
**Time Perspective:** RETROSPECTIVE
#### Enrollment Info
**Count:** 1
**Type:** ACTUAL
**Study Type:** OBSERVATIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** * All infants ≤12 months of age in Bogota, reported with pertussis disease in the national databases of Bogota, between January 2005 and December 2014.
* All infants ≤12 months of age in Bogota, deceased between January 2005 and December 2014 due to pertussis disease (primary diagnosis), based on death certificate information.
* All infants ≤12 months of age in Bogota, with ALRTI, between January 2005 and December 2014.
* All infants ≤12 months who have received primary pertussis vaccination in Bogota, between January 2005 and December 2014.
**Intervention Names:**
- Other: Pertussis maternal immunization
**Label:** Infants Group
#### Arm Group 2
**Description:** • Pregnant women will be included in the study to assess the vaccination coverage of Boostrix from March 2013 to December 2014
**Intervention Names:**
- Other: Pertussis maternal immunization
**Label:** Pregnant women Group
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Infants Group
- Pregnant women Group
**Description:** Retrospective time trend analysis before and after pertussis maternal immunization in Bogota, Colombia.
**Name:** Pertussis maternal immunization
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Measure:** Occurrence of reported cases of hospitalisations and deaths due to pertussis in infants ≤6 weeks of age, in post-vaccination period compared to pre-vaccination period of Boostrix vaccination
**Time Frame:** During the 9-year period (January 2005- December 2014)
#### Secondary Outcomes
**Measure:** Occurrence of reported cases of hospitalisations and deaths due to pertussis in infants 7 weeks to ≤12 months of age, in post-vaccination period compared to pre-vaccination period of Boostrix vaccination
**Time Frame:** During the 9-year period (January 2005- December 2014)
**Measure:** Occurrence of ALRTI in infants ≤6 weeks of age and 7 weeks to ≤12 months of age, following the introduction of pertussis maternal immunisation in Bogota, Colombia
**Time Frame:** During the 9-year period (January 2005- December 2014)
**Measure:** Description of the total number of doses of Boostrix administered in pregnant women, after its introduction into the UMV program in Bogota
**Time Frame:** During the 9-year period (January 2005- December 2014)
**Measure:** Description of the total number of doses of primary pertussis vaccine administered to infants ≤12 months of age in Bogota
**Time Frame:** During the 9-year period (January 2005- December 2014)
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
• Not applicable as since this retrospective database study will include all the available aggregated data of pertussis cases, pertussis related hospitalisations and deaths, ALRTI cases, primary pertussis vaccination coverage and Boostrix vaccination coverage.
Exclusion Criteria:
• Not applicable as since this retrospective database study will include all the available aggregated data of pertussis cases, pertussis related hospitalisations and deaths, ALRTI cases, primary pertussis vaccination coverage and Boostrix vaccination coverage.
**Maximum Age:** 12 Months
**Minimum Age:** 0 Days
**Sampling Method:** PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- CHILD
**Study Population:** * All infants ≤12 months of age in Bogota, reported with pertussis disease in the national databases of Bogota, between January 2005 and December 2014.
* All infants ≤12 months of age in Bogota, deceased between January 2005 and December 2014 due to pertussis disease (primary diagnosis), based on death certificate information.
* All infants ≤12 months of age in Bogota, with ALRTI, between January 2005 and December 2014.
* All infants ≤12 months who have received primary pertussis vaccination in Bogota, between January 2005 and December 2014.
* All pregnant women who have received Boostrix as a part of the UMV program in Bogota, between March 2013 and December 2014.
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Bogota
**Country:** Colombia
**Facility:** GSK Investigational Site
**Zip:** 110111
#### Overall Officials
**Official 1:**
**Affiliation:** GlaxoSmithKline
**Name:** GSK Clinical Trials
**Role:** STUDY_DIRECTOR
### IPD Sharing Statement Module
**IPD Sharing:** NO
### References Module
#### References
**Citation:** Carrasquilla G, Porras A, Martinez S, DeAntonio R, Devadiga R, Caceres DC, Juliao P. Incidence and mortality of pertussis disease in infants <12 months of age following introduction of pertussis maternal universal mass vaccination in Bogota, Colombia. Vaccine. 2020 Oct 27;38(46):7384-7392. doi: 10.1016/j.vaccine.2020.07.046. Epub 2020 Oct 2.
**PMID:** 33012607
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000001885
- Term: Bordetella Infections
- ID: D000016905
- Term: Gram-Negative Bacterial Infections
- ID: D000001424
- Term: Bacterial Infections
- ID: D000001423
- Term: Bacterial Infections and Mycoses
- ID: D000007239
- Term: Infections
- ID: D000012141
- Term: Respiratory Tract Infections
- ID: D000012140
- Term: Respiratory Tract Diseases
- ID: D000003015
- Term: Clostridium Infections
- ID: D000016908
- Term: Gram-Positive Bacterial Infections
- ID: D000020879
- Term: Neuromuscular Manifestations
- ID: D000009461
- Term: Neurologic Manifestations
- ID: D000009422
- Term: Nervous System Diseases
- ID: D000006996
- Term: Hypocalcemia
- ID: D000002128
- Term: Calcium Metabolism Disorders
- ID: D000008659
- Term: Metabolic Diseases
- ID: D000003354
- Term: Corynebacterium Infections
- ID: D000000193
- Term: Actinomycetales Infections
- ID: D000009181
- Term: Mycoses
- ID: D000055732
- Term: Pulmonary Aspergillosis
- ID: D000008172
- Term: Lung Diseases, Fungal
- ID: D000008171
- Term: Lung Diseases
- ID: D000012130
- Term: Respiratory Hypersensitivity
- ID: D000006969
- Term: Hypersensitivity, Immediate
- ID: D000006967
- Term: Hypersensitivity
- ID: D000007154
- Term: Immune System Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC01
- Name: Infections
- Abbrev: All
- Name: All Conditions
- Abbrev: BC08
- Name: Respiratory Tract (Lung and Bronchial) Diseases
- Abbrev: BC10
- Name: Nervous System Diseases
- Abbrev: BC18
- Name: Nutritional and Metabolic Diseases
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: BC20
- Name: Immune System Diseases
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M7347
- Name: Diphtheria
- Relevance: HIGH
- As Found: Diphtheria
- ID: M17656
- Name: Whooping Cough
- Relevance: HIGH
- As Found: Pertussis
- ID: M4535
- Name: Aspergillosis
- Relevance: HIGH
- As Found: Aspergillosis
- ID: M16511
- Name: Tetanus
- Relevance: HIGH
- As Found: Tetanus
- ID: M16515
- Name: Tetany
- Relevance: HIGH
- As Found: Tetanus
- ID: M4536
- Name: Aspergillosis, Allergic Bronchopulmonary
- Relevance: HIGH
- As Found: Aspergillosis, Allergic Bronchopulmonary
- ID: M6590
- Name: Cough
- Relevance: LOW
- As Found: Unknown
- ID: M10283
- Name: Infections
- Relevance: LOW
- As Found: Unknown
- ID: M6368
- Name: Communicable Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5163
- Name: Bordetella Infections
- Relevance: LOW
- As Found: Unknown
- ID: M4722
- Name: Bacterial Infections
- Relevance: LOW
- As Found: Unknown
- ID: M19249
- Name: Gram-Negative Bacterial Infections
- Relevance: LOW
- As Found: Unknown
- ID: M12136
- Name: Mycoses
- Relevance: LOW
- As Found: Unknown
- ID: M4721
- Name: Bacterial Infections and Mycoses
- Relevance: LOW
- As Found: Unknown
- ID: M14978
- Name: Respiratory Tract Infections
- Relevance: LOW
- As Found: Unknown
- ID: M14977
- Name: Respiratory Tract Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M6247
- Name: Clostridium Infections
- Relevance: LOW
- As Found: Unknown
- ID: M19252
- Name: Gram-Positive Bacterial Infections
- Relevance: LOW
- As Found: Unknown
- ID: M22619
- Name: Neuromuscular Manifestations
- Relevance: LOW
- As Found: Unknown
- ID: M12404
- Name: Neurologic Manifestations
- Relevance: LOW
- As Found: Unknown
- ID: M10046
- Name: Hypocalcemia
- Relevance: LOW
- As Found: Unknown
- ID: M11639
- Name: Metabolic Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5391
- Name: Calcium Metabolism Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M6574
- Name: Corynebacterium Infections
- Relevance: LOW
- As Found: Unknown
- ID: M28314
- Name: Pulmonary Aspergillosis
- Relevance: LOW
- As Found: Unknown
- ID: M11168
- Name: Lung Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M11169
- Name: Lung Diseases, Fungal
- Relevance: LOW
- As Found: Unknown
- ID: M10018
- Name: Hypersensitivity
- Relevance: LOW
- As Found: Unknown
- ID: M14967
- Name: Respiratory Hypersensitivity
- Relevance: LOW
- As Found: Unknown
- ID: M10020
- Name: Hypersensitivity, Immediate
- Relevance: LOW
- As Found: Unknown
- ID: M10200
- Name: Immune System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: T1885
- Name: Diphtheria
- Relevance: HIGH
- As Found: Diphtheria
- ID: T5926
- Name: Whooping Cough
- Relevance: HIGH
- As Found: Pertussis
- ID: T511
- Name: Aspergillosis
- Relevance: HIGH
- As Found: Aspergillosis
- ID: T5607
- Name: Tetanus
- Relevance: HIGH
- As Found: Tetanus
- ID: T286
- Name: Allergic Bronchopulmonary Aspergillosis
- Relevance: HIGH
- As Found: Aspergillosis, Allergic Bronchopulmonary
### Condition Browse Module - Meshes
- ID: D000014917
- Term: Whooping Cough
- ID: D000013742
- Term: Tetanus
- ID: D000004165
- Term: Diphtheria
- ID: D000001228
- Term: Aspergillosis
- ID: D000001229
- Term: Aspergillosis, Allergic Bronchopulmonary
- ID: D000013746
- Term: Tetany
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT04112979
**Acronym:** AUDIOPAW
**Brief Title:** Auditory Intraoperative Stimulation Effects on Pain and Agitation at Awakening (AUDIOPAW)
**Official Title:** Effects of Intraoperative Auditory Stimulation on Pain and Agitation at Awakening After Pediatric Adenotonsillectomy - 4 Arms, Double-blinded, Randomized, Controlled Clinical Trial
#### Organization Study ID Info
**ID:** RC 36/18
#### Organization
**Class:** OTHER
**Full Name:** IRCCS Burlo Garofolo
### Status Module
#### Completion Date
**Date:** 2019-08-24
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2019-10-02
**Type:** ACTUAL
**Last Update Submit Date:** 2019-10-01
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2019-08-23
**Type:** ACTUAL
#### Start Date
**Date:** 2018-03-01
**Type:** ACTUAL
**Status Verified Date:** 2019-10
#### Study First Post Date
**Date:** 2019-10-02
**Type:** ACTUAL
**Study First Submit Date:** 2019-08-06
**Study First Submit QC Date:** 2019-10-01
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** IRCCS Burlo Garofolo
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** Adenotonsillectomy (AT) is one of the most common pediatric surgical procedures. The management of postoperative pain and agitation after AT is still a controversial issue. Safety considerations limit the use of opioids and non-steroidal anti-inflammatory drugs, leading to inadequate control of the high levels of postoperative pain experienced by AT patients. Along with pain killers, non-pharmacological strategies have remarkable impact on pain management in children. A systematic review and meta-analysis published in The Lancet in 2015 on adult patients suggest that auditory stimulation with music in the perioperative setting, and even when patients are under general anaesthesia, can reduce postoperative pain, anxiety, and analgesia needs. The authors concluded that there is sufficient evidence to implement auditory stimulation in the treatment of all adult surgical patients, regardless of the mode of administration. Up to now, no research has investigated the effects of intraoperative auditory stimulation on pain and agitation upon awakening after AT in the pediatric population. Objective of this study is to determine the effects of intraoperative auditory stimulation on pain and agitation upon awakening after pediatric AT.
### Conditions Module
**Conditions:**
- Procedural Anxiety
- Procedural Pain
- Adenotonsillitis
**Keywords:**
- Auditory stimulation
- adenotonsillectomy
- pain
- agitation
- awakening
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** NONE
**Primary Purpose:** SUPPORTIVE_CARE
#### Enrollment Info
**Count:** 115
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** 45 deciBel Sensation Level (dB SL), normalized, high-frequency sampled W.A. Mozart Symphony n°4 in D K19 and n°5 in B-flat K22 (from Mozart Symphonies Vol. I - Adam Fisher, Dacapo Records, Frederiksberg C., Denmark, 2013)
**Intervention Names:**
- Behavioral: Music
**Label:** Auditory stimulation 1 (Music)
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** 45 dB SL, normalized and filtered, high-frequency sampled heartbeat sound, 75 bpm tempo, looped
**Intervention Names:**
- Behavioral: Mother's lap
**Label:** Auditory stimulation 2 (Mother's lap)
**Type:** EXPERIMENTAL
#### Arm Group 3
**Description:** Disposable foam earplugs with a noise attenuation of at least 30 deciBel (dB)
**Intervention Names:**
- Behavioral: Soundproof earplugs
**Label:** Soundproof earplugs
**Type:** EXPERIMENTAL
#### Arm Group 4
**Description:** Current standard of care
**Label:** No stimulation
**Type:** NO_INTERVENTION
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Auditory stimulation 1 (Music)
**Description:** 45 dB SL, normalized, high-frequency sampled W.A. Mozart Symphony n°4 in D K19 and n°5 in B-flat K22 (from Mozart Symphonies Vol. I - Adam Fisher, Dacapo Records, Frederiksberg C., Denmark, 2013)
**Name:** Music
**Type:** BEHAVIORAL
#### Intervention 2
**Arm Group Labels:**
- Auditory stimulation 2 (Mother's lap)
**Description:** 45 dB SL, normalized and filtered, high-frequency sampled heartbeat sound, 75 bpm tempo, looped
**Name:** Mother's lap
**Type:** BEHAVIORAL
#### Intervention 3
**Arm Group Labels:**
- Soundproof earplugs
**Description:** Disposable foam earplugs with a noise attenuation of at least 30 dB
**Name:** Soundproof earplugs
**Type:** BEHAVIORAL
### Outcomes Module
#### Primary Outcomes
**Description:** Pain score for collaborative children between 3 and 8 years will be evaluated with the self-administered Wong-Baker Faces Pain Rating scale, ranged 0-10.
Higher scores correspond to higher levels of pain.
**Measure:** Pain score (Wong-Baker Faces Pain Rating Scale), self-administered (collaborative children between 3 and 8 years)
**Time Frame:** Within 10 minutes after awakening
**Description:** Pain score for \>=8 years old children will be evaluated with Visual Analogue Scale (self administered if collaborative; administered by parents if non-collaborative children), ranged 0-10.
Higher scores correspond to higher levels of pain.
In accordance with previous studies (Ferreira-Valente et al. Validity of four pain intensity rating scales. Pain 2011;152:2399-2404), the scores of Wong-Baker Faces Pain Rating scale and Visual Analogue Scale will be considered comparable and combined in one outcome variable.
**Measure:** Pain score (Visual Analogue Scale), self-administered (collaborative children >=8 years) or administered by parents (non-collaborative children >=8 years)
**Time Frame:** Within 10 minutes after awakening
**Description:** For children \<=3 years, pain score will be evaluated by parents using the Face, Legs, Activity, Cry, Consolability (FLACC) scale, ranged 0-10.
Higher scores correspond to higher levels of pain.
**Measure:** Pain score (Face, Legs, Activity, Cry, Consolability scale), evaluated by parents (for children <=3 years)
**Time Frame:** Within 10 minutes after awakening
**Description:** For non-collaborative children between 3 and 7 years, pain score will be evaluated by parents using the Children's Hospital of Eastern Ontario Pain (CHEOPS) scale, ranged 0-10.
Higher scores correspond to higher levels of pain.
In accordance with previous studies, the scores of the Face, Legs, Activity, Cry, Consolability (FLACC) scale and of the Children's Hospital of Eastern Ontario Pain (CHEOPS) scale will be considered comparable and combined in one outcome variable.
**Measure:** Pain score (Children's Hospital of Eastern Ontario Pain scale), evaluated by parents (for non-collaborative children between 3 and 7 years)
**Time Frame:** Within 10 minutes after awakening
#### Secondary Outcomes
**Description:** Evaluated using the Paediatric Anesthesia Emergence Delirium (PAED) scale, ranging form 0 to 20. Higher scores correspond to higher levels of agitation.
**Measure:** Agitation
**Time Frame:** Within 10 minutes after awakening
**Description:** Number of postoperative pain killers drugs administered
**Measure:** Frequency of use of pain killer drugs
**Time Frame:** Within 6 hours after the awakening
### Eligibility Module
**Eligibility Criteria:** Inclusion criteria:
* age 2 to 17 years old
* scheduled for adenotonsillectomy
* recent audiometry and tympanometry evaluation (\<1 month)
Exclusion Criteria:
* no informed consent
* other combined surgical procedures
* comorbidities (e.g. congenital anomalies of the head-and-neck, psychomotor delay, autism spectrum disorders)
* American Society of Anesthesiologists (ASA) Classification score \>2
* ongoing chronic pharmacological therapy
* first language not Italian
* environmental noise exceeding limits in the operating room
**Maximum Age:** 17 Years
**Minimum Age:** 2 Years
**Sex:** ALL
**Standard Ages:**
- CHILD
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Trieste
**Country:** Italy
**Facility:** Institute for Maternal and Child Health - IRCCS Burlo Garofolo-
**Zip:** 34137
#### Overall Officials
**Official 1:**
**Affiliation:** IRCCS Burlo Garofolo
**Name:** Enrico Muzzi, MD
**Role:** STUDY_CHAIR
### References Module
#### References
**Citation:** Muzzi E, Ronfani L, Bossini B, Lezcano C, Orzan E, Barbi E. Effects of Intraoperative Auditory Stimulation on Pain and Agitation on Awakening After Pediatric Adenotonsillectomy: A Randomized Clinical Trial. JAMA Otolaryngol Head Neck Surg. 2021 Jul 1;147(7):638-645. doi: 10.1001/jamaoto.2021.0870.
**PMID:** 34014258
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000020820
- Term: Dyskinesias
- ID: D000009461
- Term: Neurologic Manifestations
- ID: D000009422
- Term: Nervous System Diseases
- ID: D000011596
- Term: Psychomotor Disorders
- ID: D000019954
- Term: Neurobehavioral Manifestations
- ID: D000096762
- Term: Aberrant Motor Behavior in Dementia
- ID: D000001526
- Term: Behavioral Symptoms
- ID: D000010146
- Term: Pain
### Condition Browse Module - Browse Branches
- Abbrev: BXM
- Name: Behaviors and Mental Disorders
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: BC10
- Name: Nervous System Diseases
### Condition Browse Module - Browse Leaves
- ID: M4324
- Name: Anxiety Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M13066
- Name: Pain
- Relevance: LOW
- As Found: Unknown
- ID: M1219
- Name: Pain, Procedural
- Relevance: HIGH
- As Found: Procedural Pain
- ID: M14452
- Name: Psychomotor Agitation
- Relevance: HIGH
- As Found: Agitation
- ID: M22574
- Name: Dyskinesias
- Relevance: LOW
- As Found: Unknown
- ID: M12404
- Name: Neurologic Manifestations
- Relevance: LOW
- As Found: Unknown
- ID: M14453
- Name: Psychomotor Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M21826
- Name: Neurobehavioral Manifestations
- Relevance: LOW
- As Found: Unknown
- ID: M6904
- Name: Dementia
- Relevance: LOW
- As Found: Unknown
- ID: M3259
- Name: Aberrant Motor Behavior in Dementia
- Relevance: LOW
- As Found: Unknown
- ID: M4818
- Name: Behavioral Symptoms
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000011595
- Term: Psychomotor Agitation
- ID: D000073818
- Term: Pain, Procedural
### Intervention Browse Module - Browse Branches
- Abbrev: All
- Name: All Drugs and Chemicals
- Abbrev: Gast
- Name: Gastrointestinal Agents
### Intervention Browse Module - Browse Leaves
- ID: M251057
- Name: TEMPO
- Relevance: LOW
- As Found: Unknown
- ID: M186190
- Name: Aluminum hydroxide, magnesium hydroxide, simethicone drug combination
- Relevance: LOW
- As Found: Unknown
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT02673879
**Acronym:** IMPI-2
**Brief Title:** IMPI 2 - A Trial of Intrapericardial Alteplase in Large Pericardial Effusion
**Official Title:** A Trial of Complete Percutaneous Pericardial Drainage Facilitated by Intrapericardial Alteplase Compared to Conventional Pericardiocentesis When Indicated in Adults With Large Pericardial Effusion Due to Tuberculous and Non-tuberculous Pericarditis
#### Organization Study ID Info
**ID:** HREC REF No. 370/2015
#### Organization
**Class:** OTHER
**Full Name:** University of Cape Town
### Status Module
#### Completion Date
**Date:** 2020-01
**Type:** ESTIMATED
#### Expanded Access Info
**Last Known Status:** RECRUITING
#### Last Update Post Date
**Date:** 2018-05-02
**Type:** ACTUAL
**Last Update Submit Date:** 2018-04-30
**Overall Status:** UNKNOWN
#### Primary Completion Date
**Date:** 2019-07
**Type:** ESTIMATED
#### Start Date
**Date:** 2016-02
**Status Verified Date:** 2016-02
#### Study First Post Date
**Date:** 2016-02-04
**Type:** ESTIMATED
**Study First Submit Date:** 2016-02-01
**Study First Submit QC Date:** 2016-02-01
### Sponsor Collaborators Module
#### Collaborators
**Class:** OTHER
**Name:** Walter Sisulu University
**Class:** OTHER
**Name:** Population Health Research Institute
#### Lead Sponsor
**Class:** OTHER
**Name:** University of Cape Town
#### Responsible Party
**Investigator Affiliation:** University of Cape Town
**Investigator Full Name:** Bongani M Mayosi
**Investigator Title:** Professor of Medicine
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** The Second Investigation of the Management of Pericarditis (IMPI-2) Trial will compare the effectiveness and safety of complete percutaneous pericardial drainage facilitated by intrapericardial alteplase (recombinant human tissue-type plasminogen activator) to conventional pericardiocentesis when indicated in 2176 patients with large pericardial effusion due to tuberculous and non-tuberculous pericarditis. An internal pilot study of 218 patients will initially confirm the feasibility of conducting a large-scale multi-centre clinical trial of intrapericardial fibrinolysis in patients with large pericardial effusion, and also provide preliminary safety data, following a dose finding study of intrapericardial alteplase.
**Detailed Description:** Intrapericardial fibrinolytic agents are used in the drainage of tuberculous, purulent, neoplastic and other inflammatory pericardial effusions to prevent recurrent effusions and constrictive pericarditis. This use is based on evidence from case reports and a small trial that did not have the statistical power to reliably evaluate the effect of pericardial drainage facilitated by intrapericardial fibrinolysis on safety and important clinical outcomes.
The Second Investigation of the Management of Pericarditis (IMPI-2) Trial will compare the effectiveness and safety of complete percutaneous pericardial drainage facilitated by intrapericardial alteplase (recombinant human tissue-type plasminogen activator) to conventional pericardiocentesis when indicated in 2176 patients with large pericardial effusion due to tuberculous and non-tuberculous pericarditis. An internal pilot study of 218 patients will initially confirm the feasibility of conducting a large-scale multi-centre clinical trial of intrapericardial fibrinolysis in patients with large pericardial effusion, and also provide preliminary safety data, following a dose finding study of intrapericardial alteplase.
Hypothesis: We hypothesise that patients with large pericardial effusion randomized to intrapericardial alteplase to ensure complete pericardial drainage will have at least a 35% reduction in cardiac tamponade requiring pericardiocentesis or constrictive pericarditis compared to conventional pericardiocentesis when indicated.
Objectives: The primary objectives of the IMPI-2 Trial are:
1. To demonstrate the feasibility of conducting a multicentre clinical trial of intrapericardial fibrinolysis in patients with large pericardial effusion, and to assess the safety of intrapericardial alteplase in an internal pilot study, and
2. To determine the effectiveness of intrapericardial alteplase in reducing the composite outcome of cardiac tamponade requiring pericardiocentesis or constrictive pericarditis in patients with large pericardial effusion in the full trial.
Should the internal pilot study demonstrate feasibility and safety; all 218 patients will be rolled-over into the full scale IMPI-2 trial of 2176 participants. The primary outcome is the first occurrence of cardiac tamponade requiring pericardiocentesis or constrictive pericarditis. The secondary safety endpoint is safety of intrapericardial fibrinolysis measured by effect on major bleeding, and serious and non-serious adverse events. The secondary efficacy outcomes are constrictive pericarditis, and cardiac tamponade requiring pericardiocentesis, analysed separately, and persistent or recurrent pericardial effusion without cardiac tamponade, hospitalisation, and death. The secondary diagnostic outcomes are proportion with bacteriologically confirmed tuberculosis from any organ or tissue; time to diagnosis of bacteriologically confirmed tuberculosis in days; accuracy of novel tests for the diagnosis of tuberculosis; proportion with specific diagnosis of any pericardial disease; time to diagnosis of a specific pericardial disease in days.
Study Design: IMPI-2 is a prospective randomized open blinded end-point trial that will enroll 2176 patients with large pericardial effusion over 36 months from up to 30 centres in South Africa and Africa. Eligible patients will be randomly assigned to receive complete pericardial drainage facilitated by intrapericardial fibrinolysis or conventional pericardiocentesis when indicated on enrollment to the study. Patients will be followed at 2 weeks, 6 weeks, 12 weeks, and in months 6, and 12 after enrollment. The IMPI Project Office, University of Cape Town, South Africa will manage and coordinate the study in association with the Pericarditis Research Unit, Walter Sisulu University, South Africa and the Population Health Research Institute, McMaster University, Canada.
Importance: IMPI-2 addresses very serious complications of large pericardial effusion (i.e., cardiac tamponade and constrictive pericarditis), which are associated with high mortality despite pericardiocentesis or pericardiectomy. This study will utilise the research network that was established by the IMPI trial which was completed in 2014.
### Conditions Module
**Conditions:**
- Pericardial Effusion
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** SINGLE
**Who Masked:**
- OUTCOMES_ASSESSOR
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 2176
**Type:** ESTIMATED
**Phases:**
- PHASE3
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Complete percutaneous pericardial drainage facilitated by intrapericardial alteplase.
**Intervention Names:**
- Other: Pericardiocentesis with Alteplase
**Label:** Pericardiocentesis with Alteplase
**Type:** ACTIVE_COMPARATOR
#### Arm Group 2
**Description:** Conventional pericardiocentesis when indicated.
**Intervention Names:**
- Other: Conventional Pericardiocentesis
**Label:** Conventional Pericardiocentesis
**Type:** OTHER
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Pericardiocentesis with Alteplase
**Description:** Complete percutaneous pericardial drainage facilitated by intrapericardial alteplase
**Name:** Pericardiocentesis with Alteplase
**Type:** OTHER
#### Intervention 2
**Arm Group Labels:**
- Conventional Pericardiocentesis
**Description:** Conventional pericardiocentesis when indicated
**Name:** Conventional Pericardiocentesis
**Type:** OTHER
### Outcomes Module
#### Other Outcomes
**Description:** The proportion with bacteriologically confirmed tuberculosis from any organ or tissue in each group will be based on findings on microscopy, Xpert MRB/RIF, culture, and / histology .
**Measure:** Proportion with proven tuberculosis
**Time Frame:** 12 months
**Description:** The time to diagnosis of bacteriologically confirmed tuberculosis in days in each group will be based on the date of sample collection and the date of first bacteriological confirmation from any organ or tissue.
**Measure:** Time to diagnosis of proven tuberculosis
**Time Frame:** 12 months
**Description:** The proportion of culture-positive tuberculosis cases who are not placed on treatment in either arm, or the proportion of these which would have been detected by the novel rapid index tests.
**Measure:** Proportion with proven tuberculosis on novel tests who are not put on treatment
**Time Frame:** 12 months
**Description:** The diagnostic accuracy of novel tests (e.g., Xpert MTB/RIF, Xpert ULTRA, InterGam) for the detection of culture-positive pericardial tuberculosis (a secondary analysis will be performed using clinically-diagnosed tuberculosis as reference standard)
**Measure:** Diagnostic accuracy of novel tests of tuberculosis
**Time Frame:** 12 months
**Description:** The proportion of drug-resistant tuberculosis cases detected.
**Measure:** Drug resistant tuberculosis
**Time Frame:** 12 months
**Description:** The proportion with a specific diagnosis of pericardial disease in each group will be based on findings on results of investigations for tuberculosis, cancer, purulent pericarditis and other disease.
**Measure:** Specific diagnosis of tuberculous pericarditis
**Time Frame:** 12 months
**Description:** The time to diagnosis of a specific pericardial disease in days in each group will be based on the date of sample collection and the date of first definitive result.
**Measure:** Time to diagnosis of specific pericardial disease
**Time Frame:** 12 months
#### Primary Outcomes
**Description:** Cardiac tamponade requiring pericardiocentesis shall refer to a combination of physical and echocardiographic findings, i.e., patients with clinical signs of tachycardia (\> 90 bpm), hypotension (systolic blood pressure \< 100 mmHg), elevated jugular venous pressure and/or pulsus paradoxus \> 10 mmHg plus evidence of a large pericardial effusion with echocardiographic signs of tamponade in the absence of other cardiac disease, as defined in the IMPI trial.
Constrictive pericarditis shall refer to a combination of physical and echocardiographic findings (i.e., patients with a prior history of pericardial effusion who have pulsus paradoxus, a raised jugular venous pressure with or without evidence of pericardial thickening on imaging) in the absence of either large pericardial effusion or other cardiac disease, as described in the IMPI trial.
**Measure:** Composite outcome of cardiac tamponade requiring pericardiocentesis or constrictive pericarditis.
**Time Frame:** 12 months
#### Secondary Outcomes
**Description:** Defined as clinically overt bleeding accompanied by one or more of the following: a decrease in the haemoglobin level of 2 g per decilitre or more over a 24-hour period, transfusion of 2 or more units of packed red cells, bleeding at a critical site (intracranial, intraspinal, intraocular, pericardial, intraarticular, intramuscular with compartment syndrome, or retroperitoneal), or fatal bleeding
**Measure:** Major bleeding
**Time Frame:** 12 months
**Description:** Defined as clinically overt bleeding that does not satisfy the criteria for major bleeding and that leads to hospital admission, physician-guided medical or surgical treatment.
**Measure:** Clinically relevant non-major bleeding
**Time Frame:** 12 months
**Description:** Any other form of bleeding that is not covered by safety outcomes 1-3
**Measure:** Any bleeding
**Time Frame:** 12 months
**Description:** Any other adverse events
**Measure:** Other adverse events
**Time Frame:** 12 months
**Description:** Refers to the echocardiographic presence of a pericardial effusion without criteria for cardiac tamponade requiring pericarditis during follow-up visits. The pericardial effusion is the same size or larger than that measured at the time of enrollment (where no pericardiocentesis was done) or post-pericardiocentesis.
**Measure:** Persistent pericardial effusion without cardiac tamponade
**Time Frame:** 12 months
**Description:** Refers to the echocardiographic presence of a pericardial effusion without criteria for cardiac tamponade requiring pericarditis during follow-up visits. Recurrence is present in the context of re-appearance of a pericardial effusion in the context where complete drainage was performed.
**Measure:** Recurrent pericardial effusion without cardiac tamponade
**Time Frame:** 12 months
**Description:** Refers to admission to hospital for at least 24 hours for any reason.
**Measure:** Hospitalisation for any cause; and death from any cause
**Time Frame:** 12 months
**Description:** Cardiac tamponade requiring pericardiocentesis shall refer to a combination of physical and echocardiographic findings, i.e., patients with clinical signs of tachycardia (\> 90 bpm), hypotension (systolic blood pressure \< 100 mmHg), elevated jugular venous pressure and/or pulsus paradoxus \> 10 mmHg plus evidence of a large pericardial effusion with echocardiographic signs of tamponade in the absence of other cardiac disease, as defined in the IMPI trial.
**Measure:** Cardiac tamponade requiring pericardiocentesis
**Time Frame:** 12 months
**Description:** Constrictive pericarditis shall refer to a combination of physical and echocardiographic findings (i.e., patients with a prior history of pericardial effusion who have pulsus paradoxus, a raised jugular venous pressure with or without evidence of pericardial thickening on imaging) in the absence of either large pericardial effusion or other cardiac disease, as described in the IMPI trial.
**Measure:** Constrictive pericarditis
**Time Frame:** 12 months
**Description:** Death from any cause
**Measure:** Death
**Time Frame:** 12 months
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
1. Age ≥ 18 years of age;
2. Confirmed large pericardial effusion on echocardiography (i.e., echo free space ≥1 cm anterior to the right ventricle of the heart in diastole);
3. Willingness to participate for the full duration of the trial (i.e., 12 months); and
4. Provision of written informed consent.
Exclusion Criteria:
1. Age \< 18 years;
2. Uraemic pericarditis (i.e., urea \> 21.4 mmol/l);
3. Thrombocytopenia (i.e., \< 100,000 platelets per µl);
4. Presence of a contra-indication to the administration of a fibrinolytic agent (i.e., major haemorrhage or major trauma; coincidental stroke; major surgery in the previous 5 days; blood pressure \>200/100 mmHg).
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Veronica Francis
**Phone:** +27832449895
**Role:** CONTACT
**Contact 2:**
**Email:** [email protected]
**Name:** Abolade A Awotedu, MBBS
**Phone:** +27822007694
**Role:** CONTACT
#### Locations
**Location 1:**
**City:** Cape Town
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Shaheen Pandie, MMed (Med)
- **Phone:** +27823199030
- **Role:** CONTACT
***Contact 2:***
- **Email:** [email protected]
- **Name:** Veronica Francis
- **Phone:** +27832449895
- **Role:** CONTACT
**Country:** South Africa
**Facility:** Groote Schuur Hospital
**State:** Western Cape
**Status:** RECRUITING
**Zip:** 7925
#### Overall Officials
**Official 1:**
**Affiliation:** University of Cape Town
**Name:** Bongani M Mayosi, DPhil
**Role:** PRINCIPAL_INVESTIGATOR
### IPD Sharing Statement Module
**Description:** Individual patient data will be made available on request at the end of the study, subject to approval by the Steering Committee of the IMPI-2 Trial.
**IPD Sharing:** YES
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000006331
- Term: Heart Diseases
- ID: D000002318
- Term: Cardiovascular Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC14
- Name: Heart and Blood Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC01
- Name: Infections
### Condition Browse Module - Browse Leaves
- ID: M13403
- Name: Pericarditis
- Relevance: LOW
- As Found: Unknown
- ID: M17127
- Name: Tuberculosis
- Relevance: LOW
- As Found: Unknown
- ID: M13400
- Name: Pericardial Effusion
- Relevance: HIGH
- As Found: Pericardial Effusion
- ID: M9419
- Name: Heart Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000010490
- Term: Pericardial Effusion
### Intervention Browse Module - Ancestors
- ID: D000005343
- Term: Fibrinolytic Agents
- ID: D000050299
- Term: Fibrin Modulating Agents
- ID: D000045504
- Term: Molecular Mechanisms of Pharmacological Action
### Intervention Browse Module - Browse Branches
- Abbrev: FiAg
- Name: Fibrinolytic Agents
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M13849
- Name: Tissue Plasminogen Activator
- Relevance: HIGH
- As Found: Bearing
- ID: M13848
- Name: Plasminogen
- Relevance: LOW
- As Found: Unknown
- ID: M8473
- Name: Fibrinolytic Agents
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: D000010959
- Term: Tissue Plasminogen Activator
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT01879579
**Acronym:** MITI
**Brief Title:** Mobile Insulin Titration Intervention
**Official Title:** The Mobile Insulin Titration Intervention (MITI) Study: Innovative Chronic Disease Management of Diabetes
#### Organization Study ID Info
**ID:** S12-03713
#### Organization
**Class:** OTHER
**Full Name:** NYU Langone Health
#### Secondary ID Infos
**ID:** UL1TR000038
**Link:** https://reporter.nih.gov/quickSearch/UL1TR000038
**Type:** NIH
### Status Module
#### Completion Date
**Date:** 2015-06
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2015-10-12
**Type:** ESTIMATED
**Last Update Submit Date:** 2015-09-15
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2015-03
**Type:** ACTUAL
#### Results First Post Date
**Date:** 2015-10-12
**Type:** ESTIMATED
**Results First Submit Date:** 2015-08-11
**Results First Submit QC Date:** 2015-09-15
#### Start Date
**Date:** 2013-06
**Status Verified Date:** 2015-09
#### Study First Post Date
**Date:** 2013-06-18
**Type:** ESTIMATED
**Study First Submit Date:** 2013-06-13
**Study First Submit QC Date:** 2013-06-13
### Sponsor Collaborators Module
#### Collaborators
**Class:** OTHER
**Name:** New York City Health and Hospitals Corporation
#### Lead Sponsor
**Class:** OTHER
**Name:** NYU Langone Health
#### Responsible Party
**Investigator Affiliation:** NYU Langone Health
**Investigator Full Name:** Natalie Levy
**Investigator Title:** Assistant Professor of Medicine
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** The purpose of this pilot study is to determine whether text message (and phone) communication can be effectively utilized to adjust long-acting insulin, compared to standard practice.
**Detailed Description:** The current practice of insulin titration for diabetics requires multiple in-person clinic visits, during which a patient's long-acting insulin dose is adjusted until the optimal dose to control glycemia is reached. Finding this optimal dose can take weeks in an ideal setting, but often takes much longer in a busy urban clinic such as Bellevue Hospital Center. Relaying titration instructions to patients via phone and text message has the potential to decrease the titration timeline, thus reducing the number of clinic visits and the time it takes patients to reach their target blood glucose levels.
For this pilot project, study staff will recruit patients who are initiating long-acting insulin treatment or initiating the titration of their existing long acting insulin treatment at Bellevue Hospital Center's Adult Primary Care Center. Patients who volunteer to enroll and provide informed consent will be randomized to one of two arms (MITI or current best practice arm) at the time of enrollment and stratified by whether the patient is initiating insulin treatment or initiating the titration of his/her existing insulin dose. The study staff will provide a cell phone (for temporary use) to any patients who are randomized to the MITI arm and don't own a personal cell phone (or whose personal cell phone is not able to receive the Sense Health text messages). Patients will use the cell phone free of cost to participate in the intervention (receive Sense Health text messages, send their fasting blood glucose levels, and speak with the diabetes nurse and study staff.)
Patients in the MITI arm will receive automated text messages 5 weekdays per week, for up to 12 weeks, from Sense Health. These text messages will request the patient's fasting blood glucose level. The patient will reply with his/her fasting blood glucose value, which will be logged in password-protected accounts on www.sensehealth.com. The clinic's diabetes nurses will check each patient's fasting blood glucose level on www.sensehealth.com each weekday and call any patient with fasting blood glucose values \< 80 mg/dL and \> 400 mg/dL. Each Thursday, patients will receive a phone call from a nurse, who will adjust the patient's insulin dose according to the study titration protocol. Each patient will continue to receive daily text messages and weekly phone calls until the first of three events occur: the patient reaches his/her optimal insulin dose for achieving glycemic control, 12 weeks elapse, or the patient withdraws from the study.
Patients in the current best practice arm (CBP) will attend scheduled clinic appointments during which a provider will review the patient's fasting blood glucose log and titrate the insulin dose according to current best practice.
Patients in both arms will continue receiving routine care, including HbA1c values every 3 months and other routine labs and measures as per standard of care.
### Conditions Module
**Conditions:**
- Diabetes Mellitus
**Keywords:**
- Insulin
- Telemedicine
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 61
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Mobile Insulin Titration Intervention (MITI) arm patients will relay their fasting blood glucose levels to the study staff via text message. The patient will receive insulin titration instructions through a weekly phone call with a diabetes nurse.
**Intervention Names:**
- Other: Mobile Insulin Titration Intervention
**Label:** Mobile Insulin Titration Intervention
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Current Best Practice (CBP) arm patients will be treated according to the current best practice of insulin titration. They will attend scheduled clinic visits during which the provider will review their blood glucose logs and provide insulin titration instructions.
**Label:** Current Best Practice
**Type:** NO_INTERVENTION
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Mobile Insulin Titration Intervention
**Description:** Patients send their fasting blood glucose levels to the clinic diabetes nurses via text message each weekday. The diabetes nurses call each patient once a week to give insulin titration instructions to replace in-person clinic visits for insulin titration.
**Name:** Mobile Insulin Titration Intervention
**Type:** OTHER
### Outcomes Module
#### Other Outcomes
**Description:** The number of text message replies from participants compared to the total number of text messages sent to participants (asking for blood glucose values). This outcome is given as a percent.
**Measure:** Percentage of Text Message Responses
**Time Frame:** 12 weeks
**Description:** The number of successful insulin titration phone calls compared to the total number of insulin titration phone calls assigned to the nurse. Successful phone calls are defined as when the nurse was able to reach the participant with one call attempt, two call attempts, or by voicemail. This outcome is given as a percent.
**Measure:** Percentage of Successful Phone Calls
**Time Frame:** 12 weeks
**Description:** The number of medication refill, emergency department, and walk-in clinic visits at Bellevue Hospital (non-insulin titration visits).
**Measure:** Patient Healthcare Utilization
**Time Frame:** 12 weeks
**Description:** Provider time spent on insulin titration visits by phone compared to insulin titration visits in the clinic.
**Measure:** Costs - Provider Time Spent on Insulin Titration Visits
**Time Frame:** 12 weeks
**Description:** The number of insulin titration visits (whether by phone or in the clinic).
**Measure:** Costs - Titration Visit Information
**Time Frame:** 12 weeks
**Description:** The time it took patients to travel to Bellevue Hospital, reported by patients in both study arms at baseline and at any subsequent clinic visits.
**Measure:** Costs - Patient Travel Time
**Time Frame:** 12 weeks
**Description:** At baseline, participants in both study arms (MITI and CBP) reported whether they had to pay co-pays for clinic visits at Bellevue Hospital.
**Measure:** Costs - Co-pays
**Time Frame:** baseline
**Description:** The study staff will interview MITI arm patients, using free-response questions, to assess their satisfaction with the intervention. The interviews will take place in person or over the phone at the patient's convenience, after the patient has reached his/her optimal insulin dose. If the patient does not reach optimal insulin dose, the interview will take place at approximately 12 weeks.
**Measure:** Qualitative Patient Satisfaction Interview
**Time Frame:** After patient reaches optimal insulin dose or at 12 weeks
#### Primary Outcomes
**Measure:** Percentage of Subjects Who Reach Optimal Long-acting Insulin Dose
**Time Frame:** 12 weeks
#### Secondary Outcomes
**Description:** The time it takes a patient to reach his/her optimal long-acting insulin dose will be measured for both study arms.
**Measure:** Time to Reach Optimal Long-acting Insulin Dose
**Time Frame:** 12 weeks
**Description:** Change in hemoglobin A1c
**Measure:** Hemoglobin A1c
**Time Frame:** baseline, 12 weeks (approximately 3 months)
**Description:** The Diabetes Treatment Satisfaction Questionnaire standard (DTSQs) will be used to measure the patient's satisfaction with diabetes treatment received prior to study participation. Scores on questionnaire range from 0 to 6: 0 = very dissatisfied, 6 = very satisfied.
**Measure:** Baseline Treatment Satisfaction
**Time Frame:** baseline
**Description:** The Diabetes Treatment Satisfaction Questionnaire standard (DTSQs) will be used to measure the patient's satisfaction with diabetes treatment received since initiation of long-acting insulin titration. Scores on questionnaire range from 0 to 6: 0 = very dissatisfied, 6 = very satisfied.
**Measure:** Treatment Satisfaction After Initiation of Insulin Titration
**Time Frame:** 12 weeks (approximately 3 months)
**Description:** The Diabetes Treatment Satisfaction Questionnaire change (DTSQc) will be used to measure the change in the patient's satisfaction with his/her diabetes treatment since initiation of long-acting insulin titration. Scores on questionnaire range from -3 to +3: -3 = much less satisfied now, +3 = much more satisfied now.
**Measure:** Change in Treatment Satisfaction
**Time Frame:** 12 weeks (approximately 3 months)
**Description:** The number of instances of hypoglycemia as indicated by fasting blood glucose levels or symptoms reported by patients in both study arms.
**Measure:** Incidence of Hypoglycemia
**Time Frame:** 12 weeks
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Initiating long-acting insulin treatment or initiating the titration of long-acting insulin treatment
* Speaks English or Spanish
* Hemoglobin A1c \> or = 8%
* Able and willing to inject insulin
* Able and willing to provide informed consent
Exclusion Criteria:
* Short-acting insulin treatment
* Systemic glucocorticoids
* Sustained elevated serum creatinine \> or = 1.5 mg/dL for men and \> or = 1.4 mg/dL for women
* Hypoglycemia unawareness
* Type 1 diabetes
**Maximum Age:** 70 Years
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** New York
**Country:** United States
**Facility:** Adult Primary Care Clinic, Bellevue Hospital Center
**State:** New York
**Zip:** 10016
#### Overall Officials
**Official 1:**
**Affiliation:** NYU Langone Health
**Name:** Natalie Levy, MD
**Role:** PRINCIPAL_INVESTIGATOR
### References Module
#### References
**Citation:** Riddle MC, Rosenstock J, Gerich J; Insulin Glargine 4002 Study Investigators. The treat-to-target trial: randomized addition of glargine or human NPH insulin to oral therapy of type 2 diabetic patients. Diabetes Care. 2003 Nov;26(11):3080-6. doi: 10.2337/diacare.26.11.3080.
**PMID:** 14578243
**Citation:** Davies M, Storms F, Shutler S, Bianchi-Biscay M, Gomis R; ATLANTUS Study Group. Improvement of glycemic control in subjects with poorly controlled type 2 diabetes: comparison of two treatment algorithms using insulin glargine. Diabetes Care. 2005 Jun;28(6):1282-8. doi: 10.2337/diacare.28.6.1282.
**PMID:** 15920040
**Citation:** Davies M, Lavalle-Gonzalez F, Storms F, Gomis R; AT.LANTUS Study Group. Initiation of insulin glargine therapy in type 2 diabetes subjects suboptimally controlled on oral antidiabetic agents: results from the AT.LANTUS trial. Diabetes Obes Metab. 2008 May;10(5):387-99. doi: 10.1111/j.1463-1326.2008.00873.x. Epub 2008 Mar 18.
**PMID:** 18355327
**Citation:** Blonde L, Merilainen M, Karwe V, Raskin P; TITRATE Study Group. Patient-directed titration for achieving glycaemic goals using a once-daily basal insulin analogue: an assessment of two different fasting plasma glucose targets - the TITRATE study. Diabetes Obes Metab. 2009 Jun;11(6):623-31. doi: 10.1111/j.1463-1326.2009.01060.x.
**PMID:** 19515182
**Citation:** Arora S, Peters AL, Agy C, Menchine M. A mobile health intervention for inner city patients with poorly controlled diabetes: proof-of-concept of the TExT-MED program. Diabetes Technol Ther. 2012 Jun;14(6):492-6. doi: 10.1089/dia.2011.0252. Epub 2012 Apr 23.
**PMID:** 22524591
**Citation:** Walker EA, Shmukler C, Ullman R, Blanco E, Scollan-Koliopoulus M, Cohen HW. Results of a successful telephonic intervention to improve diabetes control in urban adults: a randomized trial. Diabetes Care. 2011 Jan;34(1):2-7. doi: 10.2337/dc10-1005.
**PMID:** 21193619
**Citation:** Levy N, Moynihan V, Nilo A, Singer K, Bernik LS, Etiebet MA, Fang Y, Cho J, Natarajan S. The Mobile Insulin Titration Intervention (MITI) for Insulin Adjustment in an Urban, Low-Income Population: Randomized Controlled Trial. J Med Internet Res. 2015 Jul 17;17(7):e180. doi: 10.2196/jmir.4716.
**PMID:** 26187303
**Citation:** Levy N, Moynihan V, Nilo A, Singer K, Bernik LS, Etiebet MA, Fang Y, Cho J, Natarajan S. The Mobile Insulin Titration Intervention (MITI) for Insulin Glargine Titration in an Urban, Low-Income Population: Randomized Controlled Trial Protocol. JMIR Res Protoc. 2015 Mar 13;4(1):e31. doi: 10.2196/resprot.4206. Erratum In: JMIR Res Protoc. 2015 Dec 21;4(4):e138.
**PMID:** 25794243
## Derived Section
### Condition Browse Module - Browse Branches
- Abbrev: BC18
- Name: Nutritional and Metabolic Diseases
- Abbrev: BC19
- Name: Gland and Hormone Related Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M7115
- Name: Diabetes Mellitus
- Relevance: LOW
- As Found: Unknown
- ID: M6147
- Name: Chronic Disease
- Relevance: LOW
- As Found: Unknown
- ID: T1303
- Name: Chronic Graft Versus Host Disease
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Ancestors
- ID: D000007004
- Term: Hypoglycemic Agents
- ID: D000045505
- Term: Physiological Effects of Drugs
### Intervention Browse Module - Browse Branches
- Abbrev: Hypo
- Name: Hypoglycemic Agents
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M10365
- Name: Insulin
- Relevance: HIGH
- As Found: Day 1
- ID: M173166
- Name: Insulin, Globin Zinc
- Relevance: LOW
- As Found: Unknown
- ID: M10054
- Name: Hypoglycemic Agents
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: D000007328
- Term: Insulin
### Misc Info Module
- Version Holder: 2024-05-24
## Results Section
### Adverse Events Module
**Description:** We were able to assess serious adverse events for participants who completed the allocated intervention (and the participant who discontinued insulin early due to a mild possible allergy). No serious adverse events were found.
#### Event Groups
**Group ID:** EG000
**Title:** Mobile Insulin Titration Intervention
**Description:** Mobile Insulin Titration Intervention (MITI) arm patients will relay their fasting blood glucose levels to the study staff via text message. The patient will receive insulin titration instructions through a weekly phone call with a diabetes nurse.
Mobile Insulin Titration Intervention: Patients send their fasting blood glucose levels to the clinic diabetes nurses via text message each weekday. The diabetes nurses call each patient once a week to give insulin titration instructions to replace in-person clinic visits for insulin titration.
**ID:** EG000
**Other Num Affected:** 3
**Other Num at Risk:** 28
**Serious Number At Risk:** 28
**Title:** Mobile Insulin Titration Intervention
**Group ID:** EG001
**Title:** Current Best Practice
**Description:** Current Best Practice (CBP) arm patients will be treated according to the current best practice of insulin titration. They will attend scheduled clinic visits during which the provider will review their blood glucose logs and provide insulin titration instructions.
**ID:** EG001
**Other Num Affected:** 3
**Other Num at Risk:** 27
**Serious Number At Risk:** 27
**Title:** Current Best Practice
**Frequency Threshold:** 0
#### Other Events
**Term:** mild hypoglycemia
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:**
**Term:** mild possible allergy to insulin
**Assessment Type:** NON_SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:**
## Results Section - Baseline Characteristics Module
### Denomination Counts
**Group ID:** BG000
**Value:** 33
**Group ID:** BG001
**Value:** 28
**Group ID:** BG002
**Value:** 61
**Units:** Participants
### Group
**ID:** BG000
**Title:** Mobile Insulin Titration Intervention
**Description:** Mobile Insulin Titration Intervention (MITI) arm patients will relay their fasting blood glucose levels to the study staff via text message. The patient will receive insulin titration instructions through a weekly phone call with a diabetes nurse.
Mobile Insulin Titration Intervention: Patients send their fasting blood glucose levels to the clinic diabetes nurses via text message each weekday. The diabetes nurses call each patient once a week to give insulin titration instructions to replace in-person clinic visits for insulin titration.
### Group
**ID:** BG001
**Title:** Current Best Practice
**Description:** Current Best Practice (CBP) arm patients will be treated according to the current best practice of insulin titration. They will attend scheduled clinic visits during which the provider will review their blood glucose logs and provide insulin titration instructions.
### Group
**ID:** BG002
**Title:** Total
**Description:** Total of all reporting groups
### Measure
#### Measurement
**Group ID:** BG000
**Spread:** 11.22
**Value:** 48.48
#### Measurement
**Group ID:** BG001
**Spread:** 9.97
**Value:** 44.61
#### Measurement
**Group ID:** BG002
**Spread:** 10.75
**Value:** 46.70
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 15
#### Measurement
**Group ID:** BG001
**Value:** 16
#### Measurement
**Group ID:** BG002
**Value:** 31
**Category Title:** Female
#### Measurement
**Group ID:** BG000
**Value:** 18
#### Measurement
**Group ID:** BG001
**Value:** 12
#### Measurement
**Group ID:** BG002
**Value:** 30
**Category Title:** Male
**Class Title:**
**Denomination Units Selected:**
## Results Section - Baseline Characteristics Module
### Measure 1
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Age, Continuous
**Unit of Measure:** years
### Measure 2
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Sex: Female, Male
**Unit of Measure:** Participants
## Results Section - More Information Module
### Certain Agreement
**PI Sponsor Employee:** True
### Point of Contact
**Email:** [email protected]
**Organization:** New York University School of Medicine
**Phone:** 2122638924
**Title:** Dr. Natalie Levy
## Results Section - Outcome Measures Module
### Outcome Measure 1
#### Analysis
**CI Lower Limit:**
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:**
**CI Upper Limit:**
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY_OR_OTHER
**Other Analysis Description:**
**P-Value:** <0.0001
**P-Value Comment:**
**Parameter Type:**
**Parameter Value:**
**Statistical Comment:**
**Statistical Method:** Chi-squared
**Tested Non-Inferiority:** False
### Outcome Measure 2
#### Analysis
**CI Lower Limit:**
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:**
**CI Upper Limit:**
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY_OR_OTHER
**Other Analysis Description:**
**P-Value:** 0.007
**P-Value Comment:**
**Parameter Type:**
**Parameter Value:**
**Statistical Comment:**
**Statistical Method:** Interval-censoring survival analysis
**Tested Non-Inferiority:** False
### Outcome Measure 3
#### Analysis
**CI Lower Limit:**
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:**
**CI Upper Limit:**
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY_OR_OTHER
**Other Analysis Description:**
**P-Value:** 0.99
**P-Value Comment:**
**Parameter Type:**
**Parameter Value:**
**Statistical Comment:**
**Statistical Method:** Wilcoxon rank-sum test
**Tested Non-Inferiority:** False
### Outcome Measure 4
#### Analysis
**CI Lower Limit:**
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:**
**CI Upper Limit:**
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY_OR_OTHER
**Other Analysis Description:**
**P-Value:** 0.78
**P-Value Comment:**
**Parameter Type:**
**Parameter Value:**
**Statistical Comment:**
**Statistical Method:** Wilcoxon rank-sum test
**Tested Non-Inferiority:** False
### Outcome Measure 5
#### Analysis
**CI Lower Limit:**
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:**
**CI Upper Limit:**
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY_OR_OTHER
**Other Analysis Description:**
**P-Value:** 0.04
**P-Value Comment:**
**Parameter Type:**
**Parameter Value:**
**Statistical Comment:**
**Statistical Method:** Wilcoxon rank-sum test
**Tested Non-Inferiority:** False
### Outcome Measure 6
#### Analysis
**CI Lower Limit:**
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:**
**CI Upper Limit:**
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY_OR_OTHER
**Other Analysis Description:**
**P-Value:** 0.13
**P-Value Comment:**
**Parameter Type:**
**Parameter Value:**
**Statistical Comment:**
**Statistical Method:** Wilcoxon rank-sum test
**Tested Non-Inferiority:** False
### Outcome Measure 7
### Outcome Measure 8
### Outcome Measure 9
### Outcome Measure 10
### Outcome Measure 11
#### Analysis
**CI Lower Limit:**
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:**
**CI Upper Limit:**
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY_OR_OTHER
**Other Analysis Description:**
**P-Value:** 0.008
**P-Value Comment:**
**Parameter Type:**
**Parameter Value:**
**Statistical Comment:**
**Statistical Method:** Generalized estimating equation modeling
**Tested Non-Inferiority:** False
### Outcome Measure 12
#### Analysis
**CI Lower Limit:**
**CI Number of Sides:** TWO_SIDED
**CI Percentage Value:**
**CI Upper Limit:**
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY_OR_OTHER
**Other Analysis Description:**
**P-Value:** 0.003
**P-Value Comment:**
**Parameter Type:**
**Parameter Value:**
**Statistical Comment:**
**Statistical Method:** Wilcoxon rank-sum test
**Tested Non-Inferiority:** False
### Outcome Measure 13
### Outcome Measure 14
### Outcome Measure 15
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 88
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 37
**Title:**
#### Outcome Measure 2
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:** 1.29
- **Spread:**
- **Upper Limit:** 4.86
- **Value:** 3.00
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:** 2.96
- **Spread:**
- **Upper Limit:** 9.61
- **Value:** 7.07
**Title:**
#### Outcome Measure 3
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 2.64
- **Upper Limit:**
- **Value:** -1.90
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 2.63
- **Upper Limit:**
- **Value:** -1.81
**Title:**
#### Outcome Measure 4
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 1.14
- **Upper Limit:**
- **Value:** 4.99
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 0.61
- **Upper Limit:**
- **Value:** 5.20
**Title:**
#### Outcome Measure 5
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 0.54
- **Upper Limit:**
- **Value:** 5.74
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 0.52
- **Upper Limit:**
- **Value:** 5.53
**Title:**
#### Outcome Measure 6
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 0.71
- **Upper Limit:**
- **Value:** 2.71
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 0.95
- **Upper Limit:**
- **Value:** 2.42
**Title:**
#### Outcome Measure 7
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 5
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 3
**Title:**
#### Outcome Measure 8
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 84
**Title:**
#### Outcome Measure 9
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 91
**Title:**
#### Outcome Measure 10
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 6
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 11
**Title:**
#### Outcome Measure 11
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:** 3
- **Spread:**
- **Upper Limit:** 10
- **Value:** 6
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:** 20
- **Spread:**
- **Upper Limit:** 45
- **Value:** 30
**Title:**
#### Outcome Measure 12
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:** 2
- **Spread:**
- **Upper Limit:** 5
- **Value:** 3.5
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:** 1
- **Spread:**
- **Upper Limit:** 3
- **Value:** 2
**Title:**
#### Outcome Measure 13
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:** 30
- **Spread:**
- **Upper Limit:** 60
- **Value:** 45
**Title:**
#### Outcome Measure 14
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 37
**Title:**
#### Outcome Measure 15
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Parameter Type:** NUMBER
**Population Description:** No primary outcome data available for one participant in Current Best Practice arm who discontinued insulin early due to a mild possible allergy.
**Reporting Status:** POSTED
**Time Frame:** 12 weeks
**Title:** Percentage of Subjects Who Reach Optimal Long-acting Insulin Dose
**Type:** PRIMARY
**Unit of Measure:** percentage of participants
##### Group
**Description:** Mobile Insulin Titration Intervention (MITI) arm patients will relay their fasting blood glucose levels to the study staff via text message. The patient will receive insulin titration instructions through a weekly phone call with a diabetes nurse.
Mobile Insulin Titration Intervention: Patients send their fasting blood glucose levels to the clinic diabetes nurses via text message each weekday. The diabetes nurses call each patient once a week to give insulin titration instructions to replace in-person clinic visits for insulin titration.
**ID:** OG000
**Title:** Mobile Insulin Titration Intervention
##### Group
**Description:** Current Best Practice (CBP) arm patients will be treated according to the current best practice of insulin titration. They will attend scheduled clinic visits during which the provider will review their blood glucose logs and provide insulin titration instructions.
**ID:** OG001
**Title:** Current Best Practice
#### Outcome Measure 2
**Description:** The time it takes a patient to reach his/her optimal long-acting insulin dose will be measured for both study arms.
**Dispersion Type:** Inter-Quartile Range
**Parameter Type:** MEDIAN
**Population Description:** Participants who reached optimal long-acting insulin dose.
**Reporting Status:** POSTED
**Time Frame:** 12 weeks
**Title:** Time to Reach Optimal Long-acting Insulin Dose
**Type:** SECONDARY
**Unit of Measure:** weeks
##### Group
**Description:** Mobile Insulin Titration Intervention (MITI) arm patients will relay their fasting blood glucose levels to the study staff via text message. The patient will receive insulin titration instructions through a weekly phone call with a diabetes nurse.
Mobile Insulin Titration Intervention: Patients send their fasting blood glucose levels to the clinic diabetes nurses via text message each weekday. The diabetes nurses call each patient once a week to give insulin titration instructions to replace in-person clinic visits for insulin titration.
**ID:** OG000
**Title:** Mobile Insulin Titration Intervention
##### Group
**Description:** Current Best Practice (CBP) arm patients will be treated according to the current best practice of insulin titration. They will attend scheduled clinic visits during which the provider will review their blood glucose logs and provide insulin titration instructions.
**ID:** OG001
**Title:** Current Best Practice
#### Outcome Measure 3
**Description:** Change in hemoglobin A1c
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** All participants with hemoglobin A1c measurements recorded at baseline and 12 weeks.
**Reporting Status:** POSTED
**Time Frame:** baseline, 12 weeks (approximately 3 months)
**Title:** Hemoglobin A1c
**Type:** SECONDARY
**Unit of Measure:** mg/dL
##### Group
**Description:** Mobile Insulin Titration Intervention (MITI) arm patients will relay their fasting blood glucose levels to the study staff via text message. The patient will receive insulin titration instructions through a weekly phone call with a diabetes nurse.
Mobile Insulin Titration Intervention: Patients send their fasting blood glucose levels to the clinic diabetes nurses via text message each weekday. The diabetes nurses call each patient once a week to give insulin titration instructions to replace in-person clinic visits for insulin titration.
**ID:** OG000
**Title:** Mobile Insulin Titration Intervention
##### Group
**Description:** Current Best Practice (CBP) arm patients will be treated according to the current best practice of insulin titration. They will attend scheduled clinic visits during which the provider will review their blood glucose logs and provide insulin titration instructions.
**ID:** OG001
**Title:** Current Best Practice
#### Outcome Measure 4
**Description:** The Diabetes Treatment Satisfaction Questionnaire standard (DTSQs) will be used to measure the patient's satisfaction with diabetes treatment received prior to study participation. Scores on questionnaire range from 0 to 6: 0 = very dissatisfied, 6 = very satisfied.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** All participants who completed the treatment satisfaction questionnaire at baseline.
**Reporting Status:** POSTED
**Time Frame:** baseline
**Title:** Baseline Treatment Satisfaction
**Type:** SECONDARY
**Unit of Measure:** score on satisfaction scale
##### Group
**Description:** Mobile Insulin Titration Intervention (MITI) arm patients will relay their fasting blood glucose levels to the study staff via text message. The patient will receive insulin titration instructions through a weekly phone call with a diabetes nurse.
Mobile Insulin Titration Intervention: Patients send their fasting blood glucose levels to the clinic diabetes nurses via text message each weekday. The diabetes nurses call each patient once a week to give insulin titration instructions to replace in-person clinic visits for insulin titration.
**ID:** OG000
**Title:** Mobile Insulin Titration Intervention
##### Group
**Description:** Current Best Practice (CBP) arm patients will be treated according to the current best practice of insulin titration. They will attend scheduled clinic visits during which the provider will review their blood glucose logs and provide insulin titration instructions.
**ID:** OG001
**Title:** Current Best Practice
#### Outcome Measure 5
**Description:** The Diabetes Treatment Satisfaction Questionnaire standard (DTSQs) will be used to measure the patient's satisfaction with diabetes treatment received since initiation of long-acting insulin titration. Scores on questionnaire range from 0 to 6: 0 = very dissatisfied, 6 = very satisfied.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** All participants who completed the treatment satisfaction questionnaire at 12 weeks.
**Reporting Status:** POSTED
**Time Frame:** 12 weeks (approximately 3 months)
**Title:** Treatment Satisfaction After Initiation of Insulin Titration
**Type:** SECONDARY
**Unit of Measure:** score on satisfaction scale
##### Group
**Description:** Mobile Insulin Titration Intervention (MITI) arm patients will relay their fasting blood glucose levels to the study staff via text message. The patient will receive insulin titration instructions through a weekly phone call with a diabetes nurse.
Mobile Insulin Titration Intervention: Patients send their fasting blood glucose levels to the clinic diabetes nurses via text message each weekday. The diabetes nurses call each patient once a week to give insulin titration instructions to replace in-person clinic visits for insulin titration.
**ID:** OG000
**Title:** Mobile Insulin Titration Intervention
##### Group
**Description:** Current Best Practice (CBP) arm patients will be treated according to the current best practice of insulin titration. They will attend scheduled clinic visits during which the provider will review their blood glucose logs and provide insulin titration instructions.
**ID:** OG001
**Title:** Current Best Practice
#### Outcome Measure 6
**Description:** The Diabetes Treatment Satisfaction Questionnaire change (DTSQc) will be used to measure the change in the patient's satisfaction with his/her diabetes treatment since initiation of long-acting insulin titration. Scores on questionnaire range from -3 to +3: -3 = much less satisfied now, +3 = much more satisfied now.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** All participants who completed the treatment satisfaction questionnaire at 12 weeks.
**Reporting Status:** POSTED
**Time Frame:** 12 weeks (approximately 3 months)
**Title:** Change in Treatment Satisfaction
**Type:** SECONDARY
**Unit of Measure:** score on satisfaction scale
##### Group
**Description:** Mobile Insulin Titration Intervention (MITI) arm patients will relay their fasting blood glucose levels to the study staff via text message. The patient will receive insulin titration instructions through a weekly phone call with a diabetes nurse.
Mobile Insulin Titration Intervention: Patients send their fasting blood glucose levels to the clinic diabetes nurses via text message each weekday. The diabetes nurses call each patient once a week to give insulin titration instructions to replace in-person clinic visits for insulin titration.
**ID:** OG000
**Title:** Mobile Insulin Titration Intervention
##### Group
**Description:** Current Best Practice (CBP) arm patients will be treated according to the current best practice of insulin titration. They will attend scheduled clinic visits during which the provider will review their blood glucose logs and provide insulin titration instructions.
**ID:** OG001
**Title:** Current Best Practice
#### Outcome Measure 7
**Description:** The number of instances of hypoglycemia as indicated by fasting blood glucose levels or symptoms reported by patients in both study arms.
**Parameter Type:** NUMBER
**Population Description:** This outcome was analyzed for participants who completed the allocated intervention (and the participant who discontinued insulin early due to a mild possible allergy). Five participants reported hypoglycemia: 3 in the MITI arm and 2 in the CBP arm. All cases were mild.
**Reporting Status:** POSTED
**Time Frame:** 12 weeks
**Title:** Incidence of Hypoglycemia
**Type:** SECONDARY
**Unit of Measure:** instances of hypoglycemia
##### Group
**Description:** Mobile Insulin Titration Intervention (MITI) arm patients will relay their fasting blood glucose levels to the study staff via text message. The patient will receive insulin titration instructions through a weekly phone call with a diabetes nurse.
Mobile Insulin Titration Intervention: Patients send their fasting blood glucose levels to the clinic diabetes nurses via text message each weekday. The diabetes nurses call each patient once a week to give insulin titration instructions to replace in-person clinic visits for insulin titration.
**ID:** OG000
**Title:** Mobile Insulin Titration Intervention
##### Group
**Description:** Current Best Practice (CBP) arm patients will be treated according to the current best practice of insulin titration. They will attend scheduled clinic visits during which the provider will review their blood glucose logs and provide insulin titration instructions.
**ID:** OG001
**Title:** Current Best Practice
#### Outcome Measure 8
**Description:** The number of text message replies from participants compared to the total number of text messages sent to participants (asking for blood glucose values). This outcome is given as a percent.
**Parameter Type:** NUMBER
**Population Description:** Participants who completed the allocated intervention.
**Reporting Status:** POSTED
**Time Frame:** 12 weeks
**Title:** Percentage of Text Message Responses
**Type:** OTHER_PRE_SPECIFIED
**Type Units Analyzed:** text messages sent to participants
**Unit of Measure:** percentage of text messages
##### Group
**Description:** Mobile Insulin Titration Intervention (MITI) arm patients will relay their fasting blood glucose levels to the study staff via text message. The patient will receive insulin titration instructions through a weekly phone call with a diabetes nurse.
Mobile Insulin Titration Intervention: Patients send their fasting blood glucose levels to the clinic diabetes nurses via text message each weekday. The diabetes nurses call each patient once a week to give insulin titration instructions to replace in-person clinic visits for insulin titration.
**ID:** OG000
**Title:** Mobile Insulin Titration Intervention
#### Outcome Measure 9
**Description:** The number of successful insulin titration phone calls compared to the total number of insulin titration phone calls assigned to the nurse. Successful phone calls are defined as when the nurse was able to reach the participant with one call attempt, two call attempts, or by voicemail. This outcome is given as a percent.
**Parameter Type:** NUMBER
**Population Description:** Participants who completed the allocated intervention.
**Reporting Status:** POSTED
**Time Frame:** 12 weeks
**Title:** Percentage of Successful Phone Calls
**Type:** OTHER_PRE_SPECIFIED
**Type Units Analyzed:** titration phone calls assigned to nurse
**Unit of Measure:** percentage of phone calls
##### Group
**Description:** Mobile Insulin Titration Intervention (MITI) arm patients will relay their fasting blood glucose levels to the study staff via text message. The patient will receive insulin titration instructions through a weekly phone call with a diabetes nurse.
Mobile Insulin Titration Intervention: Patients send their fasting blood glucose levels to the clinic diabetes nurses via text message each weekday. The diabetes nurses call each patient once a week to give insulin titration instructions to replace in-person clinic visits for insulin titration.
**ID:** OG000
**Title:** Mobile Insulin Titration Intervention
#### Outcome Measure 10
**Description:** The number of medication refill, emergency department, and walk-in clinic visits at Bellevue Hospital (non-insulin titration visits).
**Parameter Type:** NUMBER
**Population Description:** All participants.
**Reporting Status:** POSTED
**Time Frame:** 12 weeks
**Title:** Patient Healthcare Utilization
**Type:** OTHER_PRE_SPECIFIED
**Unit of Measure:** hospital visits
##### Group
**Description:** Mobile Insulin Titration Intervention (MITI) arm patients will relay their fasting blood glucose levels to the study staff via text message. The patient will receive insulin titration instructions through a weekly phone call with a diabetes nurse.
Mobile Insulin Titration Intervention: Patients send their fasting blood glucose levels to the clinic diabetes nurses via text message each weekday. The diabetes nurses call each patient once a week to give insulin titration instructions to replace in-person clinic visits for insulin titration.
**ID:** OG000
**Title:** Mobile Insulin Titration Intervention
##### Group
**Description:** Current Best Practice (CBP) arm patients will be treated according to the current best practice of insulin titration. They will attend scheduled clinic visits during which the provider will review their blood glucose logs and provide insulin titration instructions.
**ID:** OG001
**Title:** Current Best Practice
#### Outcome Measure 11
**Description:** Provider time spent on insulin titration visits by phone compared to insulin titration visits in the clinic.
**Dispersion Type:** Inter-Quartile Range
**Parameter Type:** MEDIAN
**Population Description:** Insulin titration visits with a duration recorded.
**Reporting Status:** POSTED
**Time Frame:** 12 weeks
**Title:** Costs - Provider Time Spent on Insulin Titration Visits
**Type:** OTHER_PRE_SPECIFIED
**Type Units Analyzed:** type of insulin titration visit
**Unit of Measure:** minutes
##### Group
**Description:** Insulin titration visits that occurred over the phone in both study arms (MITI and CBP arms).
**ID:** OG000
**Title:** Insulin Titration Visits by Phone
##### Group
**Description:** Insulin titration visits that occurred in the clinic in both study arms (MITI and CBP arms).
**ID:** OG001
**Title:** Insulin Titration Visits in the Clinic
#### Outcome Measure 12
**Description:** The number of insulin titration visits (whether by phone or in the clinic).
**Dispersion Type:** Inter-Quartile Range
**Parameter Type:** MEDIAN
**Population Description:** Participants who completed the allocated intervention (and the participant who discontinued insulin early).
**Reporting Status:** POSTED
**Time Frame:** 12 weeks
**Title:** Costs - Titration Visit Information
**Type:** OTHER_PRE_SPECIFIED
**Unit of Measure:** insulin titration visits
##### Group
**Description:** Mobile Insulin Titration Intervention (MITI) arm patients will relay their fasting blood glucose levels to the study staff via text message. The patient will receive insulin titration instructions through a weekly phone call with a diabetes nurse.
Mobile Insulin Titration Intervention: Patients send their fasting blood glucose levels to the clinic diabetes nurses via text message each weekday. The diabetes nurses call each patient once a week to give insulin titration instructions to replace in-person clinic visits for insulin titration.
**ID:** OG000
**Title:** Mobile Insulin Titration Intervention
##### Group
**Description:** Current Best Practice (CBP) arm patients will be treated according to the current best practice of insulin titration. They will attend scheduled clinic visits during which the provider will review their blood glucose logs and provide insulin titration instructions.
**ID:** OG001
**Title:** Current Best Practice
#### Outcome Measure 13
**Description:** The time it took patients to travel to Bellevue Hospital, reported by patients in both study arms at baseline and at any subsequent clinic visits.
**Dispersion Type:** Inter-Quartile Range
**Parameter Type:** MEDIAN
**Reporting Status:** POSTED
**Time Frame:** 12 weeks
**Title:** Costs - Patient Travel Time
**Type:** OTHER_PRE_SPECIFIED
**Unit of Measure:** minutes
##### Group
**Description:** Participants in both study arms (MITI and CBP).
**ID:** OG000
**Title:** All Participants
#### Outcome Measure 14
**Description:** At baseline, participants in both study arms (MITI and CBP) reported whether they had to pay co-pays for clinic visits at Bellevue Hospital.
**Parameter Type:** NUMBER
**Reporting Status:** POSTED
**Time Frame:** baseline
**Title:** Costs - Co-pays
**Type:** OTHER_PRE_SPECIFIED
**Unit of Measure:** participants
##### Group
**Description:** Participants in both study arms (MITI and CBP).
**ID:** OG000
**Title:** All Participants
#### Outcome Measure 15
**Description:** The study staff will interview MITI arm patients, using free-response questions, to assess their satisfaction with the intervention. The interviews will take place in person or over the phone at the patient's convenience, after the patient has reached his/her optimal insulin dose. If the patient does not reach optimal insulin dose, the interview will take place at approximately 12 weeks.
**Reporting Status:** NOT_POSTED
**Time Frame:** After patient reaches optimal insulin dose or at 12 weeks
**Title:** Qualitative Patient Satisfaction Interview
**Type:** OTHER_PRE_SPECIFIED
### Participant Flow Module
#### Group
**Description:** Mobile Insulin Titration Intervention (MITI) arm patients will relay their fasting blood glucose levels to the study staff via text message. The patient will receive insulin titration instructions through a weekly phone call with a diabetes nurse.
Mobile Insulin Titration Intervention: Patients send their fasting blood glucose levels to the clinic diabetes nurses via text message each weekday. The diabetes nurses call each patient once a week to give insulin titration instructions to replace in-person clinic visits for insulin titration.
**ID:** FG000
**Title:** Mobile Insulin Titration Intervention
#### Group
**Description:** Current Best Practice (CBP) arm patients will be treated according to the current best practice of insulin titration. They will attend scheduled clinic visits during which the provider will review their blood glucose logs and provide insulin titration instructions.
**ID:** FG001
**Title:** Current Best Practice
#### Period
**Title:** Overall Study
##### Withdraw
**Type:** Phone incompatible with texting platform
###### Reason
**Group ID:** FG000
**Number of Subjects:** 3
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
##### Withdraw
**Type:** Did not enroll in texting platform
###### Reason
**Group ID:** FG000
**Number of Subjects:** 1
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
##### Withdraw
**Type:** Did not initiate insulin treatment
###### Reason
**Group ID:** FG000
**Number of Subjects:** 1
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
##### Withdraw
**Type:** Phenotypic type 1 diabetes
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 1
##### Withdraw
**Type:** Discontinued insulin - possible allergy
###### Reason
**Group ID:** FG000
**Number of Subjects:** 0
###### Reason
**Group ID:** FG001
**Number of Subjects:** 1
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 33
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 28
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 28
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 26
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 5
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 2
**Has Results:** True |
## Protocol Section
### Identification Module
**NCT ID:** NCT00483379
**Brief Title:** High Dose or High Dose Frequency Study of Alglucosidase Alfa
**Official Title:** An Exploratory, Open-Label Study of the Safety and Efficacy of High Dose or High Dosing Frequency Alglucosidase Alfa Treatment in Patients With Pompe Disease Who Do Not Have an Optimal Response to the Standard Dose Regimen
#### Organization Study ID Info
**ID:** AGLU03306
#### Organization
**Class:** INDUSTRY
**Full Name:** Sanofi
### Status Module
#### Completion Date
**Date:** 2010-07
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2014-03-07
**Type:** ESTIMATED
**Last Update Submit Date:** 2014-02-04
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2009-12
**Type:** ACTUAL
#### Results First Post Date
**Date:** 2011-04-22
**Type:** ESTIMATED
**Results First Submit Date:** 2011-02-07
**Results First Submit QC Date:** 2011-03-23
#### Start Date
**Date:** 2007-05
**Status Verified Date:** 2014-02
#### Study First Post Date
**Date:** 2007-06-07
**Type:** ESTIMATED
**Study First Submit Date:** 2007-06-06
**Study First Submit QC Date:** 2007-06-06
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Genzyme, a Sanofi Company
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** Pompe disease (also known as glycogen storage disease Type II) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with Pompe disease, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. The objective of this exploratory study is to evaluate the safety and efficacy of alternative dosing regimens of alglucosidase alfa in patients with Pompe disease who have not demonstrated an optimal response to the standard dosing regimen of 20 mg/kg every other week after a minimum of 6 months treatment immediately prior to study entry.
### Conditions Module
**Conditions:**
- Pompe Disease
- Glycogen Storage Disease Type II (GSD-II)
- Glycogenesis 2 Acid Maltase Deficiency
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 13
**Type:** ACTUAL
**Phases:**
- PHASE4
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**Intervention Names:**
- Biological: alglucosidase alfa
**Label:** alglucosidase alfa 20 mg/kg every week
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**Intervention Names:**
- Biological: alglucosidase alfa
**Label:** alglucosidase alfa 40 mg/kg every other week
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- alglucosidase alfa 20 mg/kg every week
- alglucosidase alfa 40 mg/kg every other week
**Description:** intravenous infusion
**Name:** alglucosidase alfa
**Other Names:**
- Recombinant human acid glucosidase
- Myozyme
**Type:** BIOLOGICAL
### Outcomes Module
#### Primary Outcomes
**Description:** Participants were enrolled based on clinical decline or sub-optimal clinical response in cardiac, respiratory and/or motor function parameters pre-study while on standard treatment. Each participant was evaluated at Week 52 for change from baseline in the criteria that declined; respiratory decline as measured by change in ventilator use is summarized in this outcome. Ventilator use might have improved (less use of ventilator support), had no change, or worsened (more use of ventilator support). Each participant served as his or her own control.
**Measure:** Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Respiratory Decline on Standard Treatment
**Time Frame:** Baseline, Week 52
**Description:** Participants were enrolled based on clinical decline or sub-optimal clinical response in cardiac, respiratory and/or motor function parameters pre-study while on standard treatment. Each participant was evaluated at Week 52 for change from baseline in the criteria that declined; motor function decline primarily based on Gross Motor Function Measure 66 and Pompe Pediatric Evaluation of Disability Inventory results is summarized. Participants could gain motor function (improve), had no change (declined stopped), or continued loss (worsened). Each participant served as his or her own control.
**Measure:** Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Motor Function Decline on Standard Treatment
**Time Frame:** Baseline, Week 52
**Description:** Overall safety summary of participants experiencing Adverse Events (AEs), Serious Adverse Events (SAEs), treatment-related AEs, and Infusion Associated Reactions (IARs). Summary is based on Treatment-emergent AEs (TEAEs), defined as AEs that occurred following the initiation of study treatment.
**Measure:** Summary of Participants Reporting Treatment-Emergent Adverse Events During the Treatment Period
**Time Frame:** Day 1 up to Week 52
#### Secondary Outcomes
**Description:** Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. Negative values indicate a smaller than mean LVM and values higher than 0 indicate a larger LVM than the mean. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy. The Z-scores for all parameters are calculated with reference to the normative data from the Children's Hospital, Boston, MA (Colan, 1992, J Am Coll Cardiol) based on the reference population with matched body surface area (BSA). Z-scores for LVM were provided by the central cardiologist.
**Measure:** Baseline Values for Left Ventricular Mass (LVM) Z-Scores
**Time Frame:** Day 0
**Description:** Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates a decrease and positive change from baseline an increase in LVM Z-score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy. The Z-scores for all parameters are calculated with reference to the normative data from the Children's Hospital, Boston, MA (Colan, 1992, J Am Coll Cardiol) based on the reference population with matched body surface area (BSA). Z-scores for LVM were provided by the central cardiologist.
**Measure:** Change From Baseline in Left Ventricular Mass (LVM) Z-Score at Week 52
**Time Frame:** Baseline, Week 52
**Description:** Cardiac pathophysiology was assessed by a central cardiologist using left ventricular mass index (LVMI) measured by echocardiogram at Baseline. Left Ventricular Mass is adjusted to the participant's body surface area in the calculation of LVMI.
**Measure:** Baseline Values for Left Ventricular Mass Index (LVMI)
**Time Frame:** Day 0
**Description:** Cardiac pathophysiology was assessed by a central cardiologist using left ventricular mass index (LVMI) measured by echocardiogram at Baseline and after 12 months of treatment (Week 52). Left Ventricular Mass is adjusted to the participant's body surface area in the calculation of LVMI.
**Measure:** Change From Baseline in Left Ventricular Mass Index (LVMI) at Week 52
**Time Frame:** Baseline, Week 52
**Description:** The change from baseline in ventilator use at the last assessment is summarized as improved (less use of ventilator support), no change, worsened (increased use of ventilator support), and did not use ventilator support.
**Measure:** Change From Baseline in Ventilator Use at Last Assessment (Approximately Week 52)
**Time Frame:** Baseline, approximately Week 52
**Description:** Body strength is measured by the MMT score on a scale of 0-10 with higher scores representing greater body strength.
**Measure:** Change From Baseline in Body Strength Measured by the Manual Muscle Testing (MMT) Total Score at Week 52
**Time Frame:** Baseline, Week 52
**Description:** The Gross Motor Function Measure 66 contains sixty-six questions with a total raw score range of 0 - 198. Raw scores are derived from the following dimensions: Lying and rolling = 12; Sitting = 45; Crawling and kneeling = 30; Standing = 39; Walking, running and jumping = 72. Higher scores indicate better gross motor functions.
**Measure:** Baseline Values of Raw Scores for Gross Motor Function Measure 66 (GMFM-66) Results
**Time Frame:** Day 0
**Description:** The Gross Motor Function Measure 66 contains sixty-six questions with a total raw score range of 0 - 198. Raw scores are derived from the following dimensions: Lying and rolling = 12; Sitting = 45; Crawling and kneeling = 30; Standing = 39; Walking, running and jumping = 72. Higher scores indicate better gross motor functions.
**Measure:** Change From Baseline in Raw Scores for Gross Motor Function Measure 66 (GMFM-66) Results at Week 52
**Time Frame:** Baseline, Week 52
**Description:** The Pompe PEDI is a disease specific version of the PEDI that was developed to assess functional capabilities and performance in children with Pompe disease from 2 months through adolescence. Baseline results for the mobility domain are reported. Scaled scores are used as an evaluative measure of change in performance over time with acquisition of new skills or new levels of independence. The range of scores is from 0-100 with scores near "0" reflecting low capability and scores near "100" reflecting high capability.
**Measure:** Baseline Values in Mobility as Measured by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI)
**Time Frame:** Day 0
**Description:** The Pompe PEDI is a disease specific version of the PEDI that was developed to assess functional capabilities and performance in children with Pompe disease from 2 months through adolescence. Change from baseline results for the mobility domain are reported. Scaled scores are used as an evaluative measure of change in performance over time with acquisition of new skills or new levels of independence. The range of scores is from 0-100 with scores near "0" reflecting low capability and scores near "100" reflecting high capability.
**Measure:** Change From Baseline in Mobility as Measured by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) at Week 52
**Time Frame:** Baseline, Week 52
**Description:** Health related quality of life is measured using the Physical Component Summary (PCS) score of the Medical Outcomes Study (MOS) Short Form Health Survey (SF-36) for participants ≥14 years of age. SF-36 normative-based scoring has a mean of 50 and a standard deviation of 10. Higher scores represent better quality of life.
**Measure:** Baseline Values for Normative Physical Component Summary of Medical Outcomes Study Short Form Health Survey (SF-36)
**Time Frame:** Day 0
**Description:** Health related quality of life is measured using the Physical Component Summary (PCS) score of the Medical Outcomes Study (MOS) Short Form Health Survey (SF-36) for participants ≥14 years of age. SF-36 normative-based scoring has a mean of 50 and a standard deviation of 10. Higher scores represent better quality of life.
**Measure:** Change From Baseline in Normative Physical Component Summary of Medical Outcomes Study Short Form Health Survey (SF-36) at Week 52
**Time Frame:** Baseline, Week 52
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* The patient or patient's legal guardian must provide signed, informed consent prior to performing any study-related procedures;
* The patient must have a clinical diagnosis of Pompe disease as defined by documented GAA deficiency in skin fibroblasts or blood;
* The patient must have been compliant with the standard dosing regimen of alglucosidase alfa (20 mg/kg every other week) for a minimum of 6 months immediately prior to study entry
* The patient must have clinical decline or sub-optimal improvement in at least one of the following parameters as compared to their condition prior to the beginning alglucosidase alfa treatment:
1. Cardiac: Left Ventricular Mass (LVM) Z-score ≥6 or LVM index ≥150 g/m2 after a minimum of 6 months of regular treatment with alglucosidase alfa; OR
2. Respiratory: New development of respiratory failure requiring the use of ventilatory assistance (invasive or non-invasive) after a minimum of 6 months of regular treatment with alglucosidase alfa. Ventilatory assistance must have been required for at least 4 weeks prior to study enrollment; OR
3. Motor Skills:
* For patients ≤ 2 years of age at study entry, failure to acquire at least 2 new gross motor milestones after a minimum of 6 months of regular treatment with alglucosidase alfa; OR
* For patients \> 2 years of age at study entry, worsening of proximal upper extremity muscle weakness as determined by the Investigator through loss of functional use of the upper extremities after a minimum of 6 months of regular treatment with alglucosidase alfa, OR
* For patients \> 8 years of age at study entry, worsening of proximal upper extremity muscle weakness as determined by the Investigator through longitudinal assessments of manual muscle testing after a minimum of 6 months of regular treatment with alglucosidase alfa, OR
* For patients previously ambulatory, progression to use of an assistive device for ambulation due to worsening of proximal lower extremity muscle weakness after a minimum of 6 months of regular treatment with alglucosidase alfa.
Exclusion Criteria:
* For patients \< 18 years of age, negative Cross-Reactive Immunologic Material (CRIM) assay result (added in protocol amendment #2);
* Any medical condition which, in the opinion of the Investigator, could interfere with treatment or evaluation of safety and/or efficacy of alglucosidase alfa;
* The patient is not currently receiving alglucosidase alfa;
* The patient has major congenital abnormality;
* The patient has used any investigational product (other than alglucosidase alfa in those regions where the product is not commercially available) within 30 days prior to study enrollment;
* The patient is pregnant or lactating.
**Minimum Age:** 6 Months
**Sex:** ALL
**Standard Ages:**
- CHILD
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Birmingham
**Country:** United States
**State:** Alabama
**Location 2:**
**City:** Stanford
**Country:** United States
**State:** California
**Location 3:**
**City:** Washington D.C.
**Country:** United States
**State:** District of Columbia
**Location 4:**
**City:** Chicago
**Country:** United States
**State:** Illinois
**Location 5:**
**City:** Kansas City
**Country:** United States
**State:** Kansas
**Location 6:**
**City:** Boston
**Country:** United States
**State:** Massachusetts
**Location 7:**
**City:** Grand Rapids
**Country:** United States
**State:** Michigan
**Location 8:**
**City:** Glenn Falls
**Country:** United States
**State:** New York
**Location 9:**
**City:** Durham
**Country:** United States
**State:** North Carolina
**Location 10:**
**City:** Parkville Victoria
**Country:** Australia
**Location 11:**
**City:** Calgary
**Country:** Canada
**State:** Alberta
#### Overall Officials
**Official 1:**
**Affiliation:** Genzyme, a Sanofi Company
**Name:** Medical Monitor
**Role:** STUDY_DIRECTOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000020140
- Term: Lysosomal Storage Diseases, Nervous System
- ID: D000020739
- Term: Brain Diseases, Metabolic, Inborn
- ID: D000001928
- Term: Brain Diseases, Metabolic
- ID: D000001927
- Term: Brain Diseases
- ID: D000002493
- Term: Central Nervous System Diseases
- ID: D000009422
- Term: Nervous System Diseases
- ID: D000008661
- Term: Metabolism, Inborn Errors
- ID: D000030342
- Term: Genetic Diseases, Inborn
- ID: D000002239
- Term: Carbohydrate Metabolism, Inborn Errors
- ID: D000016464
- Term: Lysosomal Storage Diseases
- ID: D000008659
- Term: Metabolic Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC16
- Name: Diseases and Abnormalities at or Before Birth
- Abbrev: BC18
- Name: Nutritional and Metabolic Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC10
- Name: Nervous System Diseases
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M9114
- Name: Glycogen Storage Disease
- Relevance: HIGH
- As Found: Glycogen Storage Disease
- ID: M9115
- Name: Glycogen Storage Disease Type II
- Relevance: HIGH
- As Found: Glycogen Storage Disease Type II
- ID: M18871
- Name: Lysosomal Storage Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5204
- Name: Brain Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5205
- Name: Brain Diseases, Metabolic
- Relevance: LOW
- As Found: Unknown
- ID: M22498
- Name: Brain Diseases, Metabolic, Inborn
- Relevance: LOW
- As Found: Unknown
- ID: M5742
- Name: Central Nervous System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M11641
- Name: Metabolism, Inborn Errors
- Relevance: LOW
- As Found: Unknown
- ID: M23686
- Name: Genetic Diseases, Inborn
- Relevance: LOW
- As Found: Unknown
- ID: M5498
- Name: Carbohydrate Metabolism, Inborn Errors
- Relevance: LOW
- As Found: Unknown
- ID: M11639
- Name: Metabolic Diseases
- Relevance: LOW
- As Found: Unknown
- ID: T2562
- Name: Glycogen Storage Disease Type 2
- Relevance: HIGH
- As Found: Pompe Disease
### Condition Browse Module - Meshes
- ID: D000006009
- Term: Glycogen Storage Disease Type II
- ID: D000006008
- Term: Glycogen Storage Disease
### Misc Info Module
- Version Holder: 2024-05-24
## Results Section
### Adverse Events Module
**Description:** In the event a single participant has experienced both a serious and a non-serious form of the same adverse event term, the individual has been included in the numerator ("number of affected participants") of both adverse event tables.
Events are listed independent of relationship to treatment reported.
#### Event Groups
**Group ID:** EG000
**Title:** Treatment: Alglucosidase Alfa 20 mg/kg Every Week
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** EG000
**Other Num Affected:** 6
**Other Num at Risk:** 6
**Serious Number Affected:** 2
**Serious Number At Risk:** 6
**Title:** Treatment: Alglucosidase Alfa 20 mg/kg Every Week
**Group ID:** EG001
**Title:** Treatment: Alglucosidase Alfa 40 mg/kg Every Other Week
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** EG001
**Other Num Affected:** 7
**Other Num at Risk:** 7
**Serious Number Affected:** 1
**Serious Number At Risk:** 7
**Title:** Treatment: Alglucosidase Alfa 40 mg/kg Every Other Week
**Group ID:** EG002
**Title:** Extension: Alglucosidase Alfa 20 mg/kg Every Week
**Description:** The extension period of the study allowed late-onset participants access to the same treatment they took in the treatment period until the product was commercially available.
**ID:** EG002
**Other Num Affected:** 1
**Other Num at Risk:** 1
**Serious Number Affected:** 1
**Serious Number At Risk:** 1
**Title:** Extension: Alglucosidase Alfa 20 mg/kg Every Week
**Group ID:** EG003
**Title:** Extension: Alglucosidase Alfa 40 mg/kg Every Other Week
**Description:** The extension period of the study allowed late-onset participants access to the same treatment they took in the treatment period until the product was commercially available.
**ID:** EG003
**Other Num Affected:** 1
**Other Num at Risk:** 2
**Serious Number At Risk:** 2
**Title:** Extension: Alglucosidase Alfa 40 mg/kg Every Other Week
**Frequency Threshold:** 0
#### Other Events
**Term:** Lymphadenitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Blood and lymphatic system disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Lymphadenopathy
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Blood and lymphatic system disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Right ventricular hypertrophy
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Cardiac disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Tachycardia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Cardiac disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Hypoacusis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Ear and labyrinth disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Dry eye
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Eyelid ptosis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Eye disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Abdominal discomfort
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Abdominal pain upper
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Amalgam tattoo
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Anal fissure
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Aphthous stomatitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Constipation
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Diarrhoea
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Haematochezia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Nausea
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Vomiting
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Adverse drug reaction
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Asthenia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Fatigue
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Generalised oedema
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Pyrexia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Abscess limb
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Acute sinusitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Candidiasis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Cellulitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Device related infection
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Ear infection
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Fungal skin infection
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Gastroenteritis viral
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Gastrointestinal viral infection
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Hordeolum
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Influenza
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Nasopharyngitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Otitis media
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Otitis media acute
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Pneumococcal infection
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Pneumonia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Rash pustular
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Respiratory tract infection bacterial
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Respiratory tract infection viral
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Sinusitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Streptococcal infection
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Subcutaneous abscess
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Tonsillitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Tooth abscess
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Upper respiratory tract infection
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Urinary tract infection
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Varicella
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Viral infection
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
**Term:** Arthropod bite
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Injury, poisoning and procedural complications
**Source Vocabulary:** MedDRA 13.0
**Term:** Contusion
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Injury, poisoning and procedural complications
**Source Vocabulary:** MedDRA 13.0
**Term:** Epicondylitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Injury, poisoning and procedural complications
**Source Vocabulary:** MedDRA 13.0
**Term:** Excoriation
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Injury, poisoning and procedural complications
**Source Vocabulary:** MedDRA 13.0
**Term:** Fall
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Injury, poisoning and procedural complications
**Source Vocabulary:** MedDRA 13.0
**Term:** Muscle strain
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Injury, poisoning and procedural complications
**Source Vocabulary:** MedDRA 13.0
**Term:** Vaccination complication
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Injury, poisoning and procedural complications
**Source Vocabulary:** MedDRA 13.0
**Term:** Aspiration tracheal abnormal
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** MedDRA 13.0
**Term:** Blood calcium increased
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** MedDRA 13.0
**Term:** Breath sounds abnormal
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** MedDRA 13.0
**Term:** Heart rate irregular
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** MedDRA 13.0
**Term:** Prostatic specific antigen increased
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** MedDRA 13.0
**Term:** Protein urine present
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** MedDRA 13.0
**Term:** White blood cells urine positive
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** MedDRA 13.0
**Term:** Dehydration
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Gout
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Hypocalcaemia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Hypoglycaemia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Hypokalaemia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Hypomagnesaemia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Back pain
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Muscle spasms
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Myalgia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Pain in extremity
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Dizziness
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Headache
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Hypotonia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Presyncope
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Restless legs syndrome
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Somnolence
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Tremor
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Pollakiuria
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Renal and urinary disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Asthma
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Atelectasis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Cough
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Dyspnoea
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Increased bronchial secretion
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Oropharyngeal pain
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Vasomotor rhinitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Dermatitis diaper
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Eczema
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Erythema
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Rash
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Rash erythematous
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Rash macular
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Urticaria
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Blood pressure fluctuation
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Vascular disorders
**Source Vocabulary:** MedDRA 13.0
**Term:** Hypotension
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Vascular disorders
**Source Vocabulary:** MedDRA 13.0
#### Serious Events
**Term:** Supraventricular tachycardia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Cardiac disorders
**Source Vocabulary:** MedDRA 13.0
##### Stats
**Group ID:** EG000
**Num At Risk:** 6
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 7
**Group ID:** EG002
**Num At Risk:** 1
**Group ID:** EG003
**Num At Risk:** 2
**Term:** Dysphagia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 13.0
##### Stats
**Group ID:** EG000
**Num At Risk:** 6
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 7
**Group ID:** EG002
**Num At Risk:** 1
**Group ID:** EG003
**Num At Risk:** 2
**Term:** Device related infection
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
##### Stats
**Group ID:** EG000
**Num At Risk:** 6
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 7
**Group ID:** EG002
**Num At Risk:** 1
**Group ID:** EG003
**Num At Risk:** 2
**Term:** Pneumonia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 13.0
##### Stats
**Group ID:** EG000
**Num Affected:** 2
**Num At Risk:** 6
**Group ID:** EG001
**Num At Risk:** 7
**Group ID:** EG002
**Num At Risk:** 1
**Group ID:** EG003
**Num At Risk:** 2
**Term:** Fibula fracture
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Injury, poisoning and procedural complications
**Source Vocabulary:** MedDRA 13.0
##### Stats
**Group ID:** EG000
**Num At Risk:** 6
**Group ID:** EG001
**Num At Risk:** 7
**Group ID:** EG002
**Num Affected:** 1
**Num At Risk:** 1
**Group ID:** EG003
**Num At Risk:** 2
**Term:** Weight decreased
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Investigations
**Source Vocabulary:** MedDRA 13.0
##### Stats
**Group ID:** EG000
**Num At Risk:** 6
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 7
**Group ID:** EG002
**Num At Risk:** 1
**Group ID:** EG003
**Num At Risk:** 2
**Term:** Respiratory failure
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** MedDRA 13.0
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 6
**Group ID:** EG001
**Num At Risk:** 7
**Group ID:** EG002
**Num At Risk:** 1
**Group ID:** EG003
**Num At Risk:** 2
**Time Frame:** Treatment period AEs were collected up to week 52. Extension period AEs were collected following completion of the treatment period until the product was commercially available (up to week 118).
## Results Section - Baseline Characteristics Module
### Denomination Counts
**Group ID:** BG000
**Value:** 6
**Group ID:** BG001
**Value:** 7
**Group ID:** BG002
**Value:** 13
**Units:** Participants
### Group
**ID:** BG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
### Group
**ID:** BG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
### Group
**ID:** BG002
**Title:** Total
**Description:** Total of all reporting groups
### Measure
#### Measurement
**Group ID:** BG000
**Spread:** 27.75
**Value:** 23.3
#### Measurement
**Group ID:** BG001
**Spread:** 15.56
**Value:** 16.8
#### Measurement
**Group ID:** BG002
**Spread:** 21.29
**Value:** 19.8
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 4
#### Measurement
**Group ID:** BG001
**Value:** 5
#### Measurement
**Group ID:** BG002
**Value:** 9
**Class Title:** <18 years
#### Measurement
**Group ID:** BG000
**Value:** 2
#### Measurement
**Group ID:** BG001
**Value:** 2
#### Measurement
**Group ID:** BG002
**Value:** 4
**Class Title:** >= 18 and <=65 years
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 0
**Class Title:** >65 years
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 2
#### Measurement
**Group ID:** BG001
**Value:** 3
#### Measurement
**Group ID:** BG002
**Value:** 5
**Category Title:** Female
#### Measurement
**Group ID:** BG000
**Value:** 4
#### Measurement
**Group ID:** BG001
**Value:** 4
#### Measurement
**Group ID:** BG002
**Value:** 8
**Category Title:** Male
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 0
**Category Title:** American Indian or Alaska Native
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 1
#### Measurement
**Group ID:** BG002
**Value:** 1
**Category Title:** Asian
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 0
**Category Title:** Native Hawaiian or Other Pacific Islander
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 0
**Category Title:** Black or African American
#### Measurement
**Group ID:** BG000
**Value:** 6
#### Measurement
**Group ID:** BG001
**Value:** 6
#### Measurement
**Group ID:** BG002
**Value:** 12
**Category Title:** White
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 0
**Category Title:** More than one race
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 0
**Category Title:** Unknown or Not Reported
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 4
#### Measurement
**Group ID:** BG001
**Value:** 5
#### Measurement
**Group ID:** BG002
**Value:** 9
**Class Title:** Infantile-onset Pompe Disease
#### Measurement
**Group ID:** BG000
**Value:** 2
#### Measurement
**Group ID:** BG001
**Value:** 2
#### Measurement
**Group ID:** BG002
**Value:** 4
**Class Title:** Late-onset Pompe Disease
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 0
**Class Title:** Cardiac
#### Measurement
**Group ID:** BG000
**Value:** 1
#### Measurement
**Group ID:** BG001
**Value:** 1
#### Measurement
**Group ID:** BG002
**Value:** 2
**Class Title:** Respiratory
#### Measurement
**Group ID:** BG000
**Value:** 5
#### Measurement
**Group ID:** BG001
**Value:** 6
#### Measurement
**Group ID:** BG002
**Value:** 11
**Class Title:** Motor Skills
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 3
#### Measurement
**Group ID:** BG002
**Value:** 3
**Class Title:** Positive
#### Measurement
**Group ID:** BG000
**Value:** 1
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 1
**Class Title:** Negative
#### Measurement
**Group ID:** BG000
**Value:** 5
#### Measurement
**Group ID:** BG001
**Value:** 4
#### Measurement
**Group ID:** BG002
**Value:** 9
**Class Title:** Unknown
**Denomination Units Selected:**
## Results Section - Baseline Characteristics Module
### Measure 1
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Title:** Age, Continuous
**Unit of Measure:** years
### Measure 2
**Parameter Type:** NUMBER
**Title:** Age, Customized
**Unit of Measure:** participants
### Measure 3
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Sex: Female, Male
**Unit of Measure:** Participants
### Measure 4
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Race (NIH/OMB)
**Unit of Measure:** Participants
### Measure 5
**Parameter Type:** NUMBER
**Title:** Life-stage of Disease Onset
**Unit of Measure:** participants
### Measure 6
**Description:** Participant counts of the parameter in clinical decline (cardiac, respiratory or motor skills as compared to their condition prior to the beginning alglucosidase alfa treatment) for which participants were included in the study.
**Parameter Type:** NUMBER
**Title:** Parameter in Clinical Decline
**Unit of Measure:** participants
### Measure 7
**Parameter Type:** NUMBER
**Title:** Cross-Reactive Immunologic Material (CRIM) Assay Result
**Unit of Measure:** participants
## Results Section - More Information Module
### Certain Agreement
**Other Details:** In multi-site studies, PI will delay submission of a publication until the earlier of; (i) publication of the multi-center data by Genzyme (ii)12-24 months after study completion or termination of the Study at all sites, or (iii) Genzyme notification that a publication will not occur. PI will send Genzyme a draft 60 days before publication. Genzyme can defer publication 90-120 days upon notifying PI that it will file a patent application on inventions contained in the draft.
**Restriction Type:** OTHER
**Restrictive Agreement:** True
### Limitations and Caveats
**Description:** This small exploratory study lacked a parallel control arm at the standard dose for a longer period; decline in respiratory or motor function prior to study was not collected systematically, thus change from baseline observations are inconclusive.
### Point of Contact
**Organization:** Genzyme Corporation
**Phone:** 800-745-4447
**Title:** Genzyme Medical Information
## Results Section - Outcome Measures Module
### Outcome Measure 1
### Outcome Measure 2
### Outcome Measure 3
### Outcome Measure 4
### Outcome Measure 5
### Outcome Measure 6
### Outcome Measure 7
### Outcome Measure 8
### Outcome Measure 9
### Outcome Measure 10
### Outcome Measure 11
### Outcome Measure 12
### Outcome Measure 13
### Outcome Measure 14
### Outcome Measure 15
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
#### Outcome Measure 2
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:** -1.2
- **Spread:**
- **Upper Limit:** 6.3
- **Value:** 0.3
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:** -1.2
- **Spread:**
- **Upper Limit:** 2.2
- **Value:** -0.3
**Title:**
#### Outcome Measure 3
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:** -0.8
- **Spread:** 1.04
- **Upper Limit:** 1.2
- **Value:** 0.3
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:** -0.1
- **Spread:** 0.33
- **Upper Limit:** 0.5
- **Value:** 0.4
**Title:**
#### Outcome Measure 4
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:** 49.0
- **Spread:**
- **Upper Limit:** 187.3
- **Value:** 62.1
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:** 45.4
- **Spread:**
- **Upper Limit:** 73.1
- **Value:** 56.5
**Title:**
#### Outcome Measure 5
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:** -10.9
- **Spread:** 14.44
- **Upper Limit:** 15.5
- **Value:** 12.5
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:** -5.3
- **Spread:** 5.95
- **Upper Limit:** 5.8
- **Value:** 4.0
**Title:**
#### Outcome Measure 6
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 2
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 3
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 3
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 4
**Title:**
#### Outcome Measure 7
#### Outcome Measure 8
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 60.52
- **Upper Limit:**
- **Value:** 65.0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 84.00
- **Upper Limit:**
- **Value:** 82.8
**Title:**
#### Outcome Measure 9
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 8.49
- **Upper Limit:**
- **Value:** 6.0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 6.12
- **Upper Limit:**
- **Value:** 6.7
**Title:**
#### Outcome Measure 10
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 20.94
- **Upper Limit:**
- **Value:** 38.3
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 21.26
- **Upper Limit:**
- **Value:** 46.8
**Title:**
#### Outcome Measure 11
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 4.28
- **Upper Limit:**
- **Value:** 0.6
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 3.84
- **Upper Limit:**
- **Value:** 3.5
**Title:**
#### Outcome Measure 12
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:** 2.84
- **Upper Limit:**
- **Value:** 31.3
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 9.33
- **Upper Limit:**
- **Value:** 36.0
**Title:**
#### Outcome Measure 13
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 2.5
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:** 11.24
- **Upper Limit:**
- **Value:** 4.4
**Title:**
#### Outcome Measure 14
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 2
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 4
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 2
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
#### Outcome Measure 15
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 6
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 7
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 2
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 6
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 7
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 6
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 6
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 3
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 2
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 2
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 2
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 2
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 1
**Title:**
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Description:** Participants were enrolled based on clinical decline or sub-optimal clinical response in cardiac, respiratory and/or motor function parameters pre-study while on standard treatment. Each participant was evaluated at Week 52 for change from baseline in the criteria that declined; respiratory decline as measured by change in ventilator use is summarized in this outcome. Ventilator use might have improved (less use of ventilator support), had no change, or worsened (more use of ventilator support). Each participant served as his or her own control.
**Parameter Type:** NUMBER
**Population Description:** All participants who enrolled due to decline in respiratory function while on standard treatment.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Week 52
**Title:** Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Respiratory Decline on Standard Treatment
**Type:** PRIMARY
**Unit of Measure:** participants
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 2
**Description:** Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. Negative values indicate a smaller than mean LVM and values higher than 0 indicate a larger LVM than the mean. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy. The Z-scores for all parameters are calculated with reference to the normative data from the Children's Hospital, Boston, MA (Colan, 1992, J Am Coll Cardiol) based on the reference population with matched body surface area (BSA). Z-scores for LVM were provided by the central cardiologist.
**Dispersion Type:** Full Range
**Parameter Type:** MEDIAN
**Population Description:** Full analysis population of participants with LVM data
**Reporting Status:** POSTED
**Time Frame:** Day 0
**Title:** Baseline Values for Left Ventricular Mass (LVM) Z-Scores
**Type:** SECONDARY
**Unit of Measure:** Z-score
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 3
**Description:** Z-Scores indicate the number of standard deviations (SD) from the mean in a normal distribution. A negative change from baseline indicates a decrease and positive change from baseline an increase in LVM Z-score. The normal range is -2 to 2 and greater than 2 may indicate left ventricular hypertrophy. The Z-scores for all parameters are calculated with reference to the normative data from the Children's Hospital, Boston, MA (Colan, 1992, J Am Coll Cardiol) based on the reference population with matched body surface area (BSA). Z-scores for LVM were provided by the central cardiologist.
**Dispersion Type:** Full Range
**Parameter Type:** MEDIAN
**Population Description:** Full analysis population of participants with LVM data at both timepoints
**Reporting Status:** POSTED
**Time Frame:** Baseline, Week 52
**Title:** Change From Baseline in Left Ventricular Mass (LVM) Z-Score at Week 52
**Type:** SECONDARY
**Unit of Measure:** Z-score
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 4
**Description:** Cardiac pathophysiology was assessed by a central cardiologist using left ventricular mass index (LVMI) measured by echocardiogram at Baseline. Left Ventricular Mass is adjusted to the participant's body surface area in the calculation of LVMI.
**Dispersion Type:** Full Range
**Parameter Type:** MEDIAN
**Population Description:** Full analysis population of participants with LVMI data
**Reporting Status:** POSTED
**Time Frame:** Day 0
**Title:** Baseline Values for Left Ventricular Mass Index (LVMI)
**Type:** SECONDARY
**Unit of Measure:** g/m^2
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 5
**Description:** Cardiac pathophysiology was assessed by a central cardiologist using left ventricular mass index (LVMI) measured by echocardiogram at Baseline and after 12 months of treatment (Week 52). Left Ventricular Mass is adjusted to the participant's body surface area in the calculation of LVMI.
**Dispersion Type:** Full Range
**Parameter Type:** MEDIAN
**Population Description:** Full analysis population of participants with LVMI data at both timepoints
**Reporting Status:** POSTED
**Time Frame:** Baseline, Week 52
**Title:** Change From Baseline in Left Ventricular Mass Index (LVMI) at Week 52
**Type:** SECONDARY
**Unit of Measure:** g/m^2
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 6
**Description:** The change from baseline in ventilator use at the last assessment is summarized as improved (less use of ventilator support), no change, worsened (increased use of ventilator support), and did not use ventilator support.
**Parameter Type:** NUMBER
**Population Description:** Full analysis population. The participant in the worsened category died after week 52.
**Reporting Status:** POSTED
**Time Frame:** Baseline, approximately Week 52
**Title:** Change From Baseline in Ventilator Use at Last Assessment (Approximately Week 52)
**Type:** SECONDARY
**Unit of Measure:** participants
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 7
**Description:** Body strength is measured by the MMT score on a scale of 0-10 with higher scores representing greater body strength.
**Population Description:** Full analysis population of participants \>= 8 years old. Due to the age restriction and small study population, the number of participants analyzed is too small for results to be meaningful.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Week 52
**Title:** Change From Baseline in Body Strength Measured by the Manual Muscle Testing (MMT) Total Score at Week 52
**Type:** SECONDARY
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 8
**Description:** The Gross Motor Function Measure 66 contains sixty-six questions with a total raw score range of 0 - 198. Raw scores are derived from the following dimensions: Lying and rolling = 12; Sitting = 45; Crawling and kneeling = 30; Standing = 39; Walking, running and jumping = 72. Higher scores indicate better gross motor functions.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** Full analysis population
**Reporting Status:** POSTED
**Time Frame:** Day 0
**Title:** Baseline Values of Raw Scores for Gross Motor Function Measure 66 (GMFM-66) Results
**Type:** SECONDARY
**Unit of Measure:** units on a scale
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 9
**Description:** The Gross Motor Function Measure 66 contains sixty-six questions with a total raw score range of 0 - 198. Raw scores are derived from the following dimensions: Lying and rolling = 12; Sitting = 45; Crawling and kneeling = 30; Standing = 39; Walking, running and jumping = 72. Higher scores indicate better gross motor functions.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** Full analysis population
**Reporting Status:** POSTED
**Time Frame:** Baseline, Week 52
**Title:** Change From Baseline in Raw Scores for Gross Motor Function Measure 66 (GMFM-66) Results at Week 52
**Type:** SECONDARY
**Unit of Measure:** units on a scale
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 10
**Description:** The Pompe PEDI is a disease specific version of the PEDI that was developed to assess functional capabilities and performance in children with Pompe disease from 2 months through adolescence. Baseline results for the mobility domain are reported. Scaled scores are used as an evaluative measure of change in performance over time with acquisition of new skills or new levels of independence. The range of scores is from 0-100 with scores near "0" reflecting low capability and scores near "100" reflecting high capability.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** Full analysis population
**Reporting Status:** POSTED
**Time Frame:** Day 0
**Title:** Baseline Values in Mobility as Measured by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI)
**Type:** SECONDARY
**Unit of Measure:** units on a scale
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 11
**Description:** The Pompe PEDI is a disease specific version of the PEDI that was developed to assess functional capabilities and performance in children with Pompe disease from 2 months through adolescence. Change from baseline results for the mobility domain are reported. Scaled scores are used as an evaluative measure of change in performance over time with acquisition of new skills or new levels of independence. The range of scores is from 0-100 with scores near "0" reflecting low capability and scores near "100" reflecting high capability.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** Full analysis population
**Reporting Status:** POSTED
**Time Frame:** Baseline, Week 52
**Title:** Change From Baseline in Mobility as Measured by the Pompe Pediatric Evaluation of Disability Inventory (Pompe PEDI) at Week 52
**Type:** SECONDARY
**Unit of Measure:** units on a scale
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 12
**Description:** Health related quality of life is measured using the Physical Component Summary (PCS) score of the Medical Outcomes Study (MOS) Short Form Health Survey (SF-36) for participants ≥14 years of age. SF-36 normative-based scoring has a mean of 50 and a standard deviation of 10. Higher scores represent better quality of life.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** Full analysis population of participants \>= 14 years old.
**Reporting Status:** POSTED
**Time Frame:** Day 0
**Title:** Baseline Values for Normative Physical Component Summary of Medical Outcomes Study Short Form Health Survey (SF-36)
**Type:** SECONDARY
**Unit of Measure:** units on a scale
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 13
**Description:** Health related quality of life is measured using the Physical Component Summary (PCS) score of the Medical Outcomes Study (MOS) Short Form Health Survey (SF-36) for participants ≥14 years of age. SF-36 normative-based scoring has a mean of 50 and a standard deviation of 10. Higher scores represent better quality of life.
**Dispersion Type:** Standard Deviation
**Parameter Type:** MEAN
**Population Description:** Full analysis population of participants \>= 14 years old.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Week 52
**Title:** Change From Baseline in Normative Physical Component Summary of Medical Outcomes Study Short Form Health Survey (SF-36) at Week 52
**Type:** SECONDARY
**Unit of Measure:** units on a scale
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 14
**Description:** Participants were enrolled based on clinical decline or sub-optimal clinical response in cardiac, respiratory and/or motor function parameters pre-study while on standard treatment. Each participant was evaluated at Week 52 for change from baseline in the criteria that declined; motor function decline primarily based on Gross Motor Function Measure 66 and Pompe Pediatric Evaluation of Disability Inventory results is summarized. Participants could gain motor function (improve), had no change (declined stopped), or continued loss (worsened). Each participant served as his or her own control.
**Parameter Type:** NUMBER
**Population Description:** All participants who enrolled due to decline in motor function while on standard treatment.
**Reporting Status:** POSTED
**Time Frame:** Baseline, Week 52
**Title:** Participants' Efficacy Response During the Treatment Period as Compared to Baseline for Participants With Motor Function Decline on Standard Treatment
**Type:** PRIMARY
**Unit of Measure:** participants
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Outcome Measure 15
**Description:** Overall safety summary of participants experiencing Adverse Events (AEs), Serious Adverse Events (SAEs), treatment-related AEs, and Infusion Associated Reactions (IARs). Summary is based on Treatment-emergent AEs (TEAEs), defined as AEs that occurred following the initiation of study treatment.
**Parameter Type:** NUMBER
**Population Description:** Safety population comprised of all participants who received intervention.
**Reporting Status:** POSTED
**Time Frame:** Day 1 up to Week 52
**Title:** Summary of Participants Reporting Treatment-Emergent Adverse Events During the Treatment Period
**Type:** PRIMARY
**Unit of Measure:** participants
##### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** OG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
##### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** OG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
### Participant Flow Module
#### Group
**Description:** Participants were treated with alglucosidase alfa 20 mg/kg every week for 52 weeks. This was the 'frequent dose' arm.
**ID:** FG000
**Title:** Alglucosidase Alfa 20 mg/kg Every Week
#### Group
**Description:** Participants were treated with alglucosidase alfa 40 mg/kg every other week for 52 weeks. This was the 'high dose' arm.
**ID:** FG001
**Title:** Alglucosidase Alfa 40 mg/kg Every Other Week
#### Period
**Title:** Treatment Period
##### Withdraw
**Type:** Adverse Event
###### Reason
**Group ID:** FG000
**Number of Subjects:** 1
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
##### Withdraw
**Type:** Withdrawal by Subject
###### Reason
**Group ID:** FG000
**Number of Subjects:** 1
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 6
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 7
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 4
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 7
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 2
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 0
#### Period
**Title:** Extension Period
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 1
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 2
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 1
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 2
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 0
**Pre-Assignment Details:** Fourteen participants were screened and enrolled; however, one withdrew before receiving any study infusions due to the burden of weekly trips to the medical center.
**Has Results:** True |
## Protocol Section
### Identification Module
**NCT ID:** NCT04125979
**Acronym:** CEVNPIMISELC
**Brief Title:** Clinical Evaluation of Vagal Nerve Preservation in Minimally Invasive Surgery for Early Lung Cancer
**Official Title:** Clinical Application of Vagus Nerve Preservation in Minimally Invasive Surgery for Early Lung Cancer
#### Organization Study ID Info
**ID:** 2019-LCYJ-006
#### Organization
**Class:** OTHER
**Full Name:** Shanghai Tongji Hospital, Tongji University School of Medicine
### Status Module
#### Completion Date
**Date:** 2022-01-31
**Type:** ESTIMATED
#### Expanded Access Info
**Last Known Status:** RECRUITING
#### Last Update Post Date
**Date:** 2019-10-14
**Type:** ACTUAL
**Last Update Submit Date:** 2019-10-11
**Overall Status:** UNKNOWN
#### Primary Completion Date
**Date:** 2021-01-30
**Type:** ESTIMATED
#### Start Date
**Date:** 2019-01-01
**Type:** ACTUAL
**Status Verified Date:** 2019-10
#### Study First Post Date
**Date:** 2019-10-14
**Type:** ACTUAL
**Study First Submit Date:** 2019-10-05
**Study First Submit QC Date:** 2019-10-11
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Shanghai Tongji Hospital, Tongji University School of Medicine
#### Responsible Party
**Investigator Affiliation:** Shanghai Tongji Hospital, Tongji University School of Medicine
**Investigator Full Name:** Yongxin Zhou
**Investigator Title:** Director
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** Through prospective, randomized and controlled clinical study, patients with early lung cancer who do not need lymph node dissection according to routine diagnosis and treatment were selected. The feasibility and safety of preserving vagal pulmonary branch intact during minimally invasive surgery were compared with traditional minimally invasive surgery, and the feasibility and safety of preserving vagal pulmonary branch intact during minimally invasive surgery were clarified. The effect of preserving pulmonary branches of vagus nerve in minimally invasive surgery of early lung cancer on preventing or reducing pulmonary complications after operation was evaluated by main observation indexes (incidence of pulmonary complications) and secondary evaluation indexes. It will provide a safer, simpler and more effective new technology for patients with early lung cancer undergoing minimally invasive surgery, and provide a basis for the popularization of this new technology.
**Detailed Description:** According to the suggestion of statistical experts and the minimum sample size, 120 IA1-2 patients who are going to undergo thoracoscopic lung surgery were selected according to the criteria of admission and exclusion. The risk and benefit were informed and the informed consent of the subjects was signed. The patients were numbered and randomly divided into two groups: group A with vagus nerve preservation during minimally invasive surgery and group B with traditional minimally invasive surgery for early lung cancer. The incidence of pulmonary complications within 5 weeks after operation (see the evaluation criteria for details), operation time, intraoperative bleeding volume, postoperative drainage volume, postoperative mortality, incidence of cardiovascular complications, rate of re-tracheal intubation, rate of re-admission to ICU, duration of stay in ICU, hospitalization costs were observed. Statistical analysis and evaluation of the safety of preserving pulmonary branches of vagus nerve in minimally invasive surgery and the effectiveness of preventing or reducing pulmonary complications after minimally invasive surgery.
### Conditions Module
**Conditions:**
- Lung Cancer Stage I
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
**Intervention Model Description:** 120 patients were divided into two groups. One group retained the pulmonary vagus nerve and the other group severed the vagus nerve.
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 120
**Type:** ESTIMATED
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Preservation of pulmonary branches of vagus nerve in minimally invasive surgery for lung cancer
**Intervention Names:**
- Other: In minimally invasive surgery，Vagus nerve preservation
**Label:** Preservation of pulmonary vagus nerve
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** In minimally invasive surgery for lung cancer, the pulmonary branches of vagus nerve were severed
**Intervention Names:**
- Other: In minimally invasive surgery，Vagus nerve is not preserved
**Label:** No pulmonary vagus nerve preservation
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Preservation of pulmonary vagus nerve
**Description:** In minimally invasive surgery for lung cancer, the experimental group retained the pulmonary branches of vagus nerve
**Name:** In minimally invasive surgery，Vagus nerve preservation
**Type:** OTHER
#### Intervention 2
**Arm Group Labels:**
- No pulmonary vagus nerve preservation
**Description:** In minimally invasive surgery for lung cancer, the control group did not retain the vagus nerve.
**Name:** In minimally invasive surgery，Vagus nerve is not preserved
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Description:** (1) dry cough lasting no less than 2 weeks after pneumonectomy; (2) no obvious abnormalities in chest X-ray; (3) excluding drug factors such as postnasal drip syndrome, bronchial asthma and ACEI
**Measure:** cough after pulmonary resection
**Time Frame:** From the date of randomized grouping to 5 weeks after operation, the evaluation time was as long as 5 weeks.
**Description:** Postoperative pneumonia should be considered if there are three or more of the following indicators:
1. Fever occurred 72 hours after operation, and the body temperature increased again within T \> 38 C or 72 hours.
2. Leukocyte count increased (\>12-15\*109/L) or increased again after the leucocyte count returned to normal value, exceeding 10\*109/L;
3. Chest imaging showed consolidation of lung tissue or increasing patchy shadow;
4. cough up purulent sputum or sputum culture positive;
If it contains 4, only one of the other items can be considered as a respiratory consultation to determine pulmonary infection, and need to replace antibiotics or prolong the use of antibiotics.
**Measure:** pulmonary infection
**Time Frame:** From the date of randomized grouping to 5 weeks after operation, the evaluation time was as long as 5 weeks.
**Description:** (1) Imaging findings suggest atelectasis or consolidation of the lungs; (2) signs of dyspnea; (3) decreased oxygen saturation to below 90%.
**Measure:** Atelectasis
**Time Frame:** From the date of randomized grouping to 5 weeks after operation, the evaluation time was as long as 5 weeks.
**Description:** Re-catheterization was needed; dyspnea symptoms; and drainage time was longer than 15 days.
**Measure:** hydrothorax
**Time Frame:** From the date of randomized grouping to 5 weeks after operation, the evaluation time was as long as 5 weeks.
**Description:** Tracheal intubation; Ventilator; ICU
**Measure:** Postoperative respiratory failure or ARDS or requiring tracheal intubation
**Time Frame:** From the date of randomized grouping to 5 weeks after operation, the evaluation time was as long as 5 weeks.
#### Secondary Outcomes
**Description:** Operation time;
**Measure:** Operation time;
**Time Frame:** From the date of randomized grouping to 5 weeks after operation, the evaluation time was as long as 5 weeks.
**Description:** Intraoperative bleeding volume;
**Measure:** Intraoperative bleeding volume;
**Time Frame:** From the date of randomized grouping to 5 weeks after operation, the evaluation time was as long as 5 weeks.
**Description:** Postoperative drainage volume;
**Measure:** Postoperative drainage volume;
**Time Frame:** From the date of randomized grouping to 5 weeks after operation, the evaluation time was as long as 5 weeks.
**Description:** Postoperative mortality
**Measure:** Postoperative mortality
**Time Frame:** From the date of randomized grouping to 5 weeks after operation, the evaluation time was as long as 5 weeks.
**Description:** Postoperative cardiovascular complications
**Measure:** Postoperative cardiovascular complications
**Time Frame:** From the date of randomized grouping to 5 weeks after operation, the evaluation time was as long as 5 weeks.
**Description:** Re-admission ICU rate;
**Measure:** Re-admission ICU rate;
**Time Frame:** From the date of randomized grouping to 5 weeks after operation, the evaluation time was as long as 5 weeks.
**Description:** Time of stay in ICU;
**Measure:** Time of stay in ICU;
**Time Frame:** From the date of randomized grouping to 5 weeks after operation, the evaluation time was as long as 5 weeks.
**Description:** Hospitalization days
**Measure:** Hospitalization days
**Time Frame:** From the date of randomized grouping to 5 weeks after operation, the evaluation time was as long as 5 weeks.
**Description:** Hospitalization expenses
**Measure:** Hospitalization expenses
**Time Frame:** From the date of randomized grouping to 5 weeks after operation, the evaluation time was as long as 5 weeks.
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* (1) 18-70 years of age, regardless of gender;
* (2) From May 2019 to December 2021, patients with lung cancer who underwent thoracoscopic pneumonectomy (wedge-shaped, segment and lobe) were admitted to our hospital. Postoperative pathological diagnosis was non-small cell lung cancer. Preoperative pathological staging was T1a-bN0M0 and IA1-2 (pathological staging refers to UICC 8th Edition TNM staging standard for lung cancer).
* (3) In addition to routine examinations, all patients underwent enhanced chest CT, cranial CT/MRI, abdominal B-mode ultrasound, whole body bone isotope scan or PET-CT to exclude distant metastasis.
* (4) Subjects clearly understand the purpose of the study, are willing and able to comply with the requirements to complete the study, and sign the informed consent.
Exclusion Criteria:
* (1) Patients with other infectious diseases (inflammation, tuberculosis, etc.) in the thoracic cavity;
* (2) Patients are unwilling to accept the new technique of preserving pulmonary branch of vagus nerve during operation;
* (3) Clinical unstable patients with severe cardiovascular, renal and respiratory system;
* (4) Participated in other clinical trials within 30 days;
* (5) Other reasons why researchers think it is inappropriate to participate in the experiment.
**Maximum Age:** 80 Years
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Wenli Wang, Master's degree
**Phone:** 13761295864
**Phone Ext:** 86021661110
**Role:** CONTACT
**Contact 2:**
**Email:** [email protected]
**Name:** Yongxin zhou, Doctor
**Phone:** 13681666828
**Role:** CONTACT
#### Locations
**Location 1:**
**City:** Shanghai
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Wenli Wang, Master
- **Phone:** 13761295864
- **Role:** CONTACT
***Contact 2:***
- **Email:** [email protected]
- **Name:** Shaorui Gu, scholor
- **Phone:** 18351977377
- **Role:** CONTACT
**Country:** China
**Facility:** Yongxin Zhou
**State:** Shanghai
**Status:** RECRUITING
**Zip:** 200000
#### Overall Officials
**Official 1:**
**Affiliation:** Tongji Hospital affiliated to Tongji University
**Name:** Yongxin zhou, Doctor
**Role:** STUDY_DIRECTOR
### IPD Sharing Statement Module
**IPD Sharing:** NO
### References Module
#### References
**Citation:** Chen W, Zheng R, Zeng H, Zhang S. Epidemiology of lung cancer in China. Thorac Cancer. 2015 Mar;6(2):209-15. doi: 10.1111/1759-7714.12169. Epub 2015 Mar 2.
**PMID:** 26273360
**Citation:** Sawabata N, Maeda H, Takeda S, Inoue M, Koma M, Tokunaga T, Matsuda H. Persistent cough following pulmonary resection: observational and empiric study of possible causes. Ann Thorac Surg. 2005 Jan;79(1):289-93. doi: 10.1016/j.athoracsur.2004.06.045.
**PMID:** 15620960
**Citation:** Sarna L, Evangelista L, Tashkin D, Padilla G, Holmes C, Brecht ML, Grannis F. Impact of respiratory symptoms and pulmonary function on quality of life of long-term survivors of non-small cell lung cancer. Chest. 2004 Feb;125(2):439-45. doi: 10.1378/chest.125.2.439.
**PMID:** 14769722
**Citation:** Weijs TJ, Goense L, van Rossum PSN, Meijer GJ, van Lier AL, Wessels FJ, Braat MN, Lips IM, Ruurda JP, Cuesta MA, van Hillegersberg R, Bleys RL. The peri-esophageal connective tissue layers and related compartments: visualization by histology and magnetic resonance imaging. J Anat. 2017 Feb;230(2):262-271. doi: 10.1111/joa.12552. Epub 2016 Sep 23.
**PMID:** 27659172
#### See Also Links
**Label:** pubmed
**URL:** http://www.ncbi.nlm.nih.gov/pubmed/?term=2.%09Chen+W%2C+Zheng+R%2C+Zeng+H%2C+et+al.+Epidemiology+of+lung+cancer+in+China.+Thorac+Cancer%2C+2015%2C+6(2)%3A+209-215.
**Label:** pubmed
**URL:** https://www.ncbi.nlm.nih.gov/pubmed/?term=Persistent+cough+following+pulmonary+resection%3A+observational+and+empiric+study+of+possible+causes
**Label:** pubmed
**URL:** http://www.ncbi.nlm.nih.gov/pubmed/14769722
**Label:** pubmed
**URL:** http://www.ncbi.nlm.nih.gov/pubmed/?term=Theperi-esophageal+connective+tissue+layers+and+related+compartments%3A+visualization+by+histology+and+magnetic+resonance+imaging.
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000012142
- Term: Respiratory Tract Neoplasms
- ID: D000013899
- Term: Thoracic Neoplasms
- ID: D000009371
- Term: Neoplasms by Site
- ID: D000009369
- Term: Neoplasms
- ID: D000008171
- Term: Lung Diseases
- ID: D000012140
- Term: Respiratory Tract Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: BC08
- Name: Respiratory Tract (Lung and Bronchial) Diseases
- Abbrev: All
- Name: All Conditions
### Condition Browse Module - Browse Leaves
- ID: M11172
- Name: Lung Neoplasms
- Relevance: HIGH
- As Found: Lung Cancer
- ID: M14979
- Name: Respiratory Tract Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M16658
- Name: Thoracic Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M11168
- Name: Lung Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M14977
- Name: Respiratory Tract Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000008175
- Term: Lung Neoplasms
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT06365879
**Brief Title:** To Compare Efficacy and Safety of CMAB007 and Xolair® in Patients With Chronic Spontaneous Urticaria
**Official Title:** A Multicenter, Randomized, Double-Blind, Positive Parallel Controlled Phase III Clinical Trial to Compare Omalizumab α(CMAB007) and Xolair® in Patients With Chronic Spontaneous Urticaria
#### Organization Study ID Info
**ID:** CMAB007-003
#### Organization
**Class:** INDUSTRY
**Full Name:** Taizhou Mabtech Pharmaceutical Co.,Ltd
### Status Module
#### Completion Date
**Date:** 2026-05-01
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-04-15
**Type:** ACTUAL
**Last Update Submit Date:** 2024-04-12
**Overall Status:** NOT_YET_RECRUITING
#### Primary Completion Date
**Date:** 2025-11-01
**Type:** ESTIMATED
#### Start Date
**Date:** 2024-05-01
**Type:** ESTIMATED
**Status Verified Date:** 2024-03
#### Study First Post Date
**Date:** 2024-04-15
**Type:** ACTUAL
**Study First Submit Date:** 2024-04-07
**Study First Submit QC Date:** 2024-04-12
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Taizhou Mabtech Pharmaceutical Co.,Ltd
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** This study is a multicenter, randomized, double-blind, positive parallel controlled phase III clinical trial to compare efficacy, immunogenicity, pharmacokinetics, pharmacodynamics and safety of omalizumab α(CMAB007) and Xolair® in patients with refractory chronic spontaneous urticaria
**Detailed Description:** The study will consist of three periods: a screening period (up to 2 weeks), a 12-week treatment period and a 8-week follow-up period. The total duration of the study is up to 22 weeks. After signing informed consent, subjects who meet the inclusion criteria and do not meet the exclusion criteria will be assigned to the CMAB007 or Xolair® in a 1:1 ratio. The subjects will treated with three doses of CMAB007 or Xolair®, subcutaneous injection every four weeks in the treatment period. After the end of the treatment period, an 8-week safety follow-up was conducted. The type and dosage of H1 antihistamines remained stable in the study.
### Conditions Module
**Conditions:**
- Chronic Spontaneous Urticaria
**Keywords:**
- Chronic spontaneous urticaria
- omalizumab
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** QUADRUPLE
**Who Masked:**
- PARTICIPANT
- CARE_PROVIDER
- INVESTIGATOR
- OUTCOMES_ASSESSOR
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 392
**Type:** ESTIMATED
**Phases:**
- PHASE3
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** patients received a dose of Omalizumab alpha (CMAB007) 300mg which consisted of two injections of omalizumab 150 mg vials every 4 weeks(Day 1, Week 4 and Week 8) in the treatment period
**Intervention Names:**
- Drug: CMAB007
**Label:** CMAB007
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** patients received a dose of Xolair® 300mg which consisted of two injections of omalizumab 150 mg vials every 4 weeks(Day 1, Week 4 and Week 8) in the treatment period
**Intervention Names:**
- Drug: Xolair
**Label:** Xolair®
**Type:** ACTIVE_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- CMAB007
**Description:** 300mg, SC, Q4W
**Name:** CMAB007
**Other Names:**
- Omalizumab alpha
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- Xolair®
**Description:** 300mg, SC, Q4W
**Name:** Xolair
**Other Names:**
- Omalizumab
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Description:** The severity of the itch was recorded by the patient twice daily in their Diary, on a scale of 0 (none) to 3 (intense/severe). Baseline ISS7 was calculated 7 days prior to the first treatment date. A weekly score (ISS7) was derived by adding up the average daily scores of the seven days preceding the visit. The possible range of the weekly score was therefore 0 to 21, where 0 is the best score and 21 is the worst score. The complete itch response was defined as ISS7 = 0.
Itch (Pruritus) Severity Score Scale:
* 0 = None
* 1= Mild (minimal awareness, easily tolerated)
* 2= Moderate (definite awareness, bothersome but tolerable)
* 3= Severe (difficult to tolerate)
**Measure:** Change From Baseline of the Itch Severity Score (ISS7) Score At Week 12
**Time Frame:** Week 12
#### Secondary Outcomes
**Description:** UAS7 is the sum of the HSS7 and the ISS7 scores. The possible range of the weekly UAS7 score is 0 to 42. A higher urticaria activity score indicates more severe symptoms. A negative change score from baseline indicates improvement.
**Measure:** Change From Baseline of Urticaria Activity Score (UAS7) At Week 12
**Time Frame:** Week 12
**Description:** Hives Severity Score (HSS), defined by number of hives, were recorded by the patient once daily in their Diary, on a scale of 0 (none) to 3 (intense/severe). A weekly number of hives score (NHS7) was derived by adding up the average daily scores of the seven days preceding the visit. The possible range of the weekly score was therefore 0 to 21. The complete hives response was defined as NHS7=0.
Hives Severity Score scale:
* 0= None
* 1=Mild (\< 20 hives/24 hours)
* 2=Moderate (20-50 hives/24 hours)
* 3=Severe (\>50 hives/24 hours)
**Measure:** Change From Baseline of Number of Hives Score (NHS7) At Week 12
**Time Frame:** Week 12
**Description:** The ISS7 MID response was defined as a reduction from Baseline in ISS7 of ≥ 5 points. Time to ISS7 MID response was the time (in weeks) from the date of the first dose to the date where ISS7 MID response was first achieved during Week 1 to Week 12.
**Measure:** Time to ISS7 MID Response by Week 12
**Time Frame:** Week 12
**Description:** UAS7 is the sum of the HSS7 and the ISS7 scores. The possible range of the weekly UAS7 score is 0 to 42. A higher urticaria activity score indicates more severe symptoms. A negative change score from baseline indicates improvement. Week 12 responders were defined as patients who achieved an absolute UAS7 ≤ 6 at Week 12.
**Measure:** Percentage of Patients With UAS7≤6 at Week 12
**Time Frame:** Week 12
**Description:** The ISS7 MID response was defined as a reduction from Baseline in ISS7 of ≥ 5 points.
**Measure:** Percentage of Patients With ISS7 Minimally Important Difference (MID) at Week 12
**Time Frame:** Week 12
**Description:** The urticaria control score (UCT) was a retrospective assessment of disease control over the past 4 weeks. The assessment contents four-items: physical symptoms of the urticaria (itch, hives\[welts\], and or swelling), quality of life, treatment control and overall disease control. The score range for each item is 0 to 4, and the total score is the sum of the four questions.The possible score range of the UCT is 0 to 16. A score of 16 indicates complete disease control. A score of \<12 on identifies patients with poorly controlled chronic urticaria (CU), and a score of ≥12 identifies those with well-controlled symptoms.
**Measure:** Change From Baseline of Urticaria Control Test (UCT) at Week 12
**Time Frame:** Week 12
**Description:** The possible range of the UCT is 0 to 16. A higher UCT indicates better control. The total score ≥12 indicates that urticaria is well controlled.
**Measure:** Percentage of Patients With UCT≥12 at Week 12
**Time Frame:** Week 12
**Description:** The possible range of the UCT is 0 to 16. A higher UCT indicates better control. The total score=16 indicates that urticaria is complete controlled.
**Measure:** Percentage of Complete Controls (UCT=16) at Week 12
**Time Frame:** Week 12
**Description:** Dermatology life quality index (DLQI) is a 10-item dermatology- specific health-related quality of life measure. Patients rated their dermatology symptoms as well as the impact of their skin condition on various aspects of their lives. An overall score was calculated as well as separate scores for the following domains: symptoms and feelings, daily activities, leisure, work and school, personal relationships, treatment. Each domain had 4 response categories ranging from 0 (not at all) to 3 (very much). "Not relevant" is a valid score also and is scored as 0. The DLQI total score is a sum of all 10 responses. Scores range from 0 to 30 with higher scores indicating greater health-related quality of life impairment.
**Measure:** Change From Baseline of Dermatology Life Quality Index (DLQI) Score at Week 12
**Time Frame:** Week 12
**Description:** UAS7 is the sum of the HSS7 and the ISS7 scores. The possible range of the weekly UAS7 score is 0 to 42. A higher urticaria activity score indicates more severe symptoms. A negative change score from baseline indicates improvement. Complete responders are defined as participants who achieved UAS7 = 0.
**Measure:** Percentage of Complete Responders (UAS7=0) at Week 12
**Time Frame:** Week 12
**Description:** The percentage of angioedema-free days from Weeks 4 to 12 was defined as the number of days for which a patient responded "No" to the angioedema question in the daily diary divided by the total number of days with a non-missing diary entry, starting at the Week 4 visit and ending the day prior to the Week 12 visit.
**Measure:** Percentage of Angioedema-free Days From Week 4 to Week 12
**Time Frame:** Week 4 to Week 12
**Description:** Number of patients reporting at least one adverse event in the study. An adverse event is defined as any untoward medical occurrence in a clinical trial participant graded according to the common terminology criteria for adverse events (CTCAE) v.5.0 criteria,including clinically-significant changes in physical examinations, laboratory safety tests, ECG and vital signs
**Measure:** Incidence of adverse events
**Time Frame:** Week 20
**Description:** Percentage of anti-drug antibody and neutralizing antibody in the study. Subjects with a positive antibody response to Omalizumab were determined to test neutralizing antibody.
**Measure:** Immunogenicity
**Time Frame:** Week 20
**Description:** AUC0-t, the area under the concentration-time curve from time zero to the time of the last quantifiable concentration, will be analyzed using a non-atrioventricular model.
**Measure:** AUC0-t
**Time Frame:** Week 20
**Description:** AUC0-inf, the area under the concentration-time curve from time zero to infinity, will be analyzed using a non-atrioventricular model.
**Measure:** AUC0-inf
**Time Frame:** Week 20
**Description:** Half time will be analyzed using a non-atrioventricular model.
**Measure:** Half time
**Time Frame:** Week 20
**Description:** Cmax is the maximum concentration.
**Measure:** Cmax
**Time Frame:** Week 20
**Description:** Tmax is the time of the maximum concentration.
**Measure:** Tmax
**Time Frame:** Week 20
**Description:** Clearance rate will be analyzed using a non-atrioventricular model.
**Measure:** Clearance Rate
**Time Frame:** Week 20
**Description:** Apparent Volume of Distribution will be analyzed using a non-atrioventricular model.
**Measure:** Apparent Volume of Distribution
**Time Frame:** Week 20
**Description:** Pharmacodynamics
**Measure:** Level of Total IgE and Free IgE
**Time Frame:** Week 20
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
1. Male or female patients 15 to 75 years old (both inclusive).
2. Diagnosis of CSU refractory to H1AH, as defined by all of the following:
* Diagnosis of CSU at the time of screening, urticaria history ≥ 6 months at the time of randomization
* The presence of itch and hives for ≥ 6 consecutive weeks within half year prior to randomization despite use of H1AH treatment during this time period;
* UAS7 score (range 0-42) ≥ 16 and itch component of UAS7 (range 0-21) ≥ 8 during 7 days prior to randomization (Day 1);
* In-clinic UAS ≥ 4 on at least one of the screening visit days;
* Patients must have been on an approved dose of an H1AH for CSU for at least the 3 consecutive days immediately prior to the screening visit and must have documented current use on the day of the initial screening visit.
3. Voluntarily sign the informed consent form. Willing and able to complete a daily symptom diary for the duration of the study, and comply with the protocol requirements.
4. Patients must not have had any missing diary entries in the 7 days prior to randomization.
5. Women of childbearing age have negative pregnancy tests and are not in the lactation period at the time of screening. Both male and female patients must agree to practice contraception from the signing of informed consent to 6 months after the last dose of study drugs.
Exclusion Criteria:
1. Chronic inducible urticaria. This includes but is not limited to: dermatographism (factitious urticaria), cold, heat, solar, delayed pressure, aquagenic, cholinergic or contact urticarias. Any of the following diseases, which may have symptoms of urticaria and/or angioedema: urticarial vasculitis, erythema multiforme, mastocytosis, hereditary or acquired angioedema, etc.
2. Suffer from other chronic pruritic dermatosis that may confound the results: atopic dermatitis, bullous pemphigoid, dermatitis herpetiformis, senile pruritus, psoriasis, etc.
3. CSU patients who had difficulty breathing episodes due to angioedema in the past six months.
4. Previous treatment with omalizumab within one year prior to signing the informed consent.
5. Hypersensitivity to omalizumab, study drug excipients or other biosimilars, or have a history of severe drug allergy or anaphylactic shock.
6. Use systemic or local corticosteroids, hydroxychloroquine, methotrexate, cyclosporin or cyclophosphamide, and tripterygium within 30 days prior to screening; Use compound glycyrrhizin, total glucosides of paeony and other traditional Chinese medicine within 14 days before screening; Use H2 antihistamines and leukotriene modulators within 7 days before screening; Use H1 antihistamines exceeding protocol requirements within 3 days prior to screening; Use other CSU drugs (including but not limited to biologics, small molecule drugs) within 3 months or 5 drug half-lives (whichever is longer) prior to screening.
7. Patients with a stool examination positive for ova or parasites at screening.
8. Active infections requiring treatment at screening, include but not limited to pulmonary infection, tuberculosis and acute bronchial asthma.
9. Have received live attenuated vaccine or intravenous immunoglobulin within 30 days before screening; Live attenuated vaccines are planned or received at any time during the study period.
10. History of malignancy of any organ or system within 5 years prior to screening (except for basal cell carcinoma, Cervical carcinoma in situ)
11. Evidence of cardiovascular disease (e.g., myocardial infarction, unstable angina, acute coronary syndrome, NYHA Grade III/VI left ventricular failure, arrhythmias and uncontrolled hypertension within 6 months prior to screening), neurological, psychiatric, pulmonary, renal, liver, endocrine, metabolic, hematological, gastrointestinal, or immune deficiencies that the investigators believe may compromise subjects' safety or interfere study results.
12. Presence of clinically significant examination, include but not limited:
* Abnormal liver function \[AST or ALT ≥ 2 x ULN, or total bilirubin ≥ 2 x ULN\];
* Abnormal renal function \[elevated serum creatinine \> 1.5 x ULN\] or estimated glomerular filtration rate (eGFR) \< 45 mL/min (using Cockcroft-Gault equation);
* Abnormal ECG, e.g.,corrected QTcF interval (using Fridericia's correction formula) ≥470ms (female) or 450ms (male), II-III degree atrioventricular block, tachyarrhythmia requiring treatment.
* Hematological abnormalities: hemoglobin\<100g/L, platelets\<100\*10\^9/L, white blood cells\<3.0\*10\^9/L, neutrophils\<1.5\*10\^9/L.
13. Patients with serological results positive for human immunodeficiency virus, treponema pallidum, hepatitis B or hepatitis C. (1) Hepatitis B surface antigen positive patients will be excluded; (2) Hepatitis B core antibody positive: 1) Hepatitis B surface antibody positive patients can be included in this study; 2) Patients with negative hepatitis B surface antibodies need to be tested for HBV-DNA (if HBV DNA is negative, patients can be included in this study; If the HBV DNA is positive, the patient will be excluded).
14. Participated in clinical trials of other drugs within 3 months or 5 drug half-lives (whichever is longer) prior to screening.
15. History of alcohol or drug abuse, or failure to take medication as prescribed.
16. Pregnant or nursing (lactating) women.
17. Currently taking or plan to take medications prohibited by the protocol at screening.
18. Other conditions deemed by investigator as unsuitable for this trial.
**Maximum Age:** 75 Years
**Minimum Age:** 15 Years
**Sex:** ALL
**Standard Ages:**
- CHILD
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Jinhua Xu, PhD
**Phone:** 021-52887781
**Role:** CONTACT
**Contact 2:**
**Email:** [email protected]
**Name:** Hui Tang, PhD
**Phone:** +86 13774395291
**Role:** CONTACT
#### Overall Officials
**Official 1:**
**Affiliation:** Huashan Hospital
**Name:** Jinhua Xu, PhD
**Role:** PRINCIPAL_INVESTIGATOR
**Official 2:**
**Affiliation:** Huashan Hospital
**Name:** Hui Tang, PhD
**Role:** PRINCIPAL_INVESTIGATOR
### IPD Sharing Statement Module
**IPD Sharing:** NO
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000017445
- Term: Skin Diseases, Vascular
- ID: D000012871
- Term: Skin Diseases
- ID: D000006969
- Term: Hypersensitivity, Immediate
- ID: D000006967
- Term: Hypersensitivity
- ID: D000007154
- Term: Immune System Diseases
- ID: D000002908
- Term: Chronic Disease
- ID: D000020969
- Term: Disease Attributes
- ID: D000010335
- Term: Pathologic Processes
### Condition Browse Module - Browse Branches
- Abbrev: BC17
- Name: Skin and Connective Tissue Diseases
- Abbrev: BC20
- Name: Immune System Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M17330
- Name: Urticaria
- Relevance: HIGH
- As Found: Urticaria
- ID: M2129
- Name: Chronic Urticaria
- Relevance: HIGH
- As Found: Chronic Spontaneous Urticaria
- ID: M15674
- Name: Skin Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M19714
- Name: Skin Diseases, Vascular
- Relevance: LOW
- As Found: Unknown
- ID: M10018
- Name: Hypersensitivity
- Relevance: LOW
- As Found: Unknown
- ID: M10020
- Name: Hypersensitivity, Immediate
- Relevance: LOW
- As Found: Unknown
- ID: M10200
- Name: Immune System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M6147
- Name: Chronic Disease
- Relevance: LOW
- As Found: Unknown
- ID: M22700
- Name: Disease Attributes
- Relevance: LOW
- As Found: Unknown
- ID: T1303
- Name: Chronic Graft Versus Host Disease
- Relevance: HIGH
- As Found: Chronic
### Condition Browse Module - Meshes
- ID: D000014581
- Term: Urticaria
- ID: D000080223
- Term: Chronic Urticaria
### Intervention Browse Module - Ancestors
- ID: D000018926
- Term: Anti-Allergic Agents
- ID: D000018927
- Term: Anti-Asthmatic Agents
- ID: D000019141
- Term: Respiratory System Agents
- ID: D000007155
- Term: Immunologic Factors
- ID: D000045505
- Term: Physiological Effects of Drugs
### Intervention Browse Module - Browse Branches
- Abbrev: AAll
- Name: Anti-Allergic Agents
- Abbrev: Resp
- Name: Respiratory System Agents
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M413
- Name: Omalizumab
- Relevance: HIGH
- As Found: Neoadjuvant Chemotherapy
- ID: M4225
- Name: Antibodies
- Relevance: LOW
- As Found: Unknown
- ID: M4230
- Name: Antibodies, Monoclonal
- Relevance: HIGH
- As Found: Neoadjuvant Chemotherapy
- ID: M10184
- Name: Immunoglobulins
- Relevance: LOW
- As Found: Unknown
- ID: M20962
- Name: Anti-Allergic Agents
- Relevance: LOW
- As Found: Unknown
- ID: M20963
- Name: Anti-Asthmatic Agents
- Relevance: LOW
- As Found: Unknown
- ID: M21137
- Name: Respiratory System Agents
- Relevance: LOW
- As Found: Unknown
- ID: M10201
- Name: Immunologic Factors
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: D000069444
- Term: Omalizumab
- ID: D000000911
- Term: Antibodies, Monoclonal
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT01403779
**Acronym:** KOSIMA
**Brief Title:** Comparison of the Cosmetic Outcome of Hypofractionated Versus Normofractionated IMRT in Treatment of Breast Cancer
**Official Title:** Comparison of the Cosmetic Outcome of Hypofractionated Versus Normofractionated Intensity Modulated Radiotherapy (IMRT) in Treatment of Breast Cancer: The KOSIMA Trial
#### Organization Study ID Info
**ID:** MA-KOSIMA-01
#### Organization
**Class:** OTHER
**Full Name:** Universitätsmedizin Mannheim
#### Secondary ID Infos
**Domain:** Arbeitsgemeinschaft Radiologische Onkologie der Deutschen Krebsgesellschaft
**ID:** ARO 2010-3
**Type:** OTHER
### Status Module
#### Completion Date
**Date:** 2017-02-02
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2022-11-07
**Type:** ACTUAL
**Last Update Submit Date:** 2022-11-02
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2017-02-02
**Type:** ACTUAL
#### Start Date
**Date:** 2010-07
**Status Verified Date:** 2022-11
#### Study First Post Date
**Date:** 2011-07-27
**Type:** ESTIMATED
**Study First Submit Date:** 2010-10-29
**Study First Submit QC Date:** 2011-07-26
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Universitätsmedizin Mannheim
#### Responsible Party
**Investigator Affiliation:** Universitätsmedizin Mannheim
**Investigator Full Name:** Frederik Wenz
**Investigator Title:** Prof. Dr.
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** Several multicenter studies have shown the equivalence of hypofractionated radiotherapy and normofractionated radiotherapy after breast-conserving surgery. However, the treatment in these studies was carried out with conventional techniques and not with the modern IMRT. Also the evaluation of quality of life and cosmetic outcome were not standardized.
This study is a two-arm prospective study comparing normofractionated and hypofractionated radiotherapy in patients with breast cancer using tangential IMRT techniques.
The primary endpoints are acute and chronic cosmetic breast changes. The secondary endpoint is the patients' quality of life.
Patients to be included are breast cancer 60 years old patients or older with tumour stages pTis-pT3, pN0-pN1a, M0 after breast-conserving surgery. Patients with right sided breast cancer are stratified to receive a hypofractionated treatment course (40.05 / 2.67Gy in 15 fractions) and the left sided breast cancer a normofractionated irradiation (50/2Gy in 25 fractions). In both arms, patients between 60-69 years are to receive a boost (16 Gy / 2Gy).
In both groups, a tangential intensity-modulated radiation technique aiming to achieve optimal dose homogeneity is applied.
Since higher single radiation dose to the heart can lead to higher morbidity and/or mortality, patient stratification according to the diseased side was adopted where the left-sided breast cancer patients would receive normofractionated 2Gy single dose. Therefore there is no randomization.
For classification and grading of adverse cosmetic events, the "Common Toxicity Criteria (CTC-AE V3.0) and the recognized LENT-SOMA scores are to be regularly documented. Quality of life is to be documented with two standardized, validated questionnaires "QLQ C30 and BR23" of the EORTC (European Organization for Research and Treatment of Cancer). The questionnaires are to be filled by the patients themselves at different time points during the study period.
A sum of grade III fibrosis, grade III telangiectasia and grade II hyperpigmentation of around 20% is expected after 2 years.
Therefore, calculation of the required number of cases based on an alpha of 0.05 and a power of 80% with a maximal tolerable toxicity difference of 15% within 2 years results in the need for recruiting 226 patients (113 in each arm) (non-inferiority of hypofractionated therapy).
### Conditions Module
**Conditions:**
- Tumors
- Breast Cancer
**Keywords:**
- Intensity-Modulated Radiation Therapy (IMRT)
- Breast cancer
- Hypofractionation
### Design Module
#### Design Info
**Allocation:** NON_RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 226
**Type:** ACTUAL
**Phases:**
- PHASE3
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** hypofractionated IMRT for right sided breast cancer
**Intervention Names:**
- Radiation: intensity modulated radiotherapy (IMRT) for breast cancer
**Label:** 1-Hypofractionated IMRT
**Type:** ACTIVE_COMPARATOR
#### Arm Group 2
**Description:** normofractionated IMRT for left sided breast cancer
**Intervention Names:**
- Radiation: intensity modulated radiotherapy (IMRT) for breast cancer
**Label:** 2-Normofractioated IMRT
**Type:** ACTIVE_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- 1-Hypofractionated IMRT
- 2-Normofractioated IMRT
**Description:** right sided breast cancer patients are stratified to receive a hypofractionated treatment course (40.05 / 2.67Gy in 15 fractions) and the left sided breast cancer patients a normofractionated irradiation (50/2Gy in 25 fractions). In both arms, patients between 60-69 years are to receive a boost (16 Gy / 2Gy).
**Name:** intensity modulated radiotherapy (IMRT) for breast cancer
**Other Names:**
- Arm 1, Arm 2
**Type:** RADIATION
### Outcomes Module
#### Primary Outcomes
**Measure:** acute and chronic cosmetic outcome
**Time Frame:** 2 years
#### Secondary Outcomes
**Measure:** acute and chronic cosmetic outcome, Quality of life
**Time Frame:** 2 years
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Histologically confirmed invasive or in situ breast cancer, tumor stage pTis-pT3, pN0-1a, M0
* Age ≥ 60 years
* signed informed consent from the patient
Exclusion Criteria:
* Stage pN1b-PN3, pT4 and / or M1
* incomplete surgical resection
* after mastectomy of the ipsilateral or contralateral breast
* breast reconstruction with implant or expander insert
* bilateral breast cancer
* Lack of compliance or consent
* Indications for irradiation of the axillary, supraclavicular or parasternal lymph nodes
**Minimum Age:** 60 Years
**Sex:** FEMALE
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Mannheim
**Country:** Germany
**Facility:** Department of Radiotherapy University Hospital Mannheim
**Zip:** 68167
#### Overall Officials
**Official 1:**
**Affiliation:** UMM
**Name:** Frederik Wenz, MD
**Role:** STUDY_CHAIR
### References Module
#### References
**Citation:** Sarria GR, Welzel G, Polednik M, Wenz F, Abo-Madyan Y. Prospective Comparison of Hypofractionated Versus Normofractionated Intensity-Modulated Radiotherapy in Breast Cancer: Late Toxicity Results of the Non-Inferiority KOSIMA Trial (ARO2010-3). Front Oncol. 2022 May 5;12:824891. doi: 10.3389/fonc.2022.824891. eCollection 2022.
**PMID:** 35600361
#### See Also Links
**Label:** German Abstract
**URL:** https://doi.org/10.1055/s-0033-1347536
**Label:** English Abstract
**URL:** http://dx.doi.org/10.1016/j.ijrobp.2013.06.552
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000009371
- Term: Neoplasms by Site
- ID: D000009369
- Term: Neoplasms
- ID: D000001941
- Term: Breast Diseases
- ID: D000012871
- Term: Skin Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: BC17
- Name: Skin and Connective Tissue Diseases
- Abbrev: All
- Name: All Conditions
### Condition Browse Module - Browse Leaves
- ID: M5220
- Name: Breast Neoplasms
- Relevance: HIGH
- As Found: Breast Cancer
- ID: M5218
- Name: Breast Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M15674
- Name: Skin Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000001943
- Term: Breast Neoplasms
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT01832779
**Acronym:** POEM
**Brief Title:** Peroral Endoscopic Myotomy (POEM) for the Treatment of Achalasia
**Official Title:** Prospective Evaluation of the Clinical Utility of Peroral Endoscopic Myotomy (POEM)
#### Organization Study ID Info
**ID:** IRB201300104
#### Organization
**Class:** OTHER
**Full Name:** University of Florida
### Status Module
#### Completion Date
**Date:** 2025-12
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-05-14
**Type:** ACTUAL
**Last Update Submit Date:** 2024-05-10
**Overall Status:** ACTIVE_NOT_RECRUITING
#### Primary Completion Date
**Date:** 2025-12
**Type:** ESTIMATED
#### Start Date
**Date:** 2013-03
**Status Verified Date:** 2024-05
#### Study First Post Date
**Date:** 2013-04-16
**Type:** ESTIMATED
**Study First Submit Date:** 2013-04-02
**Study First Submit QC Date:** 2013-04-11
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** University of Florida
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** Achalasia is a chronic disease of altered esophageal motility with resulting functional obstruction to the passage of food leading to poor quality of life and significant morbidity. The two main treatments available in the US are endoscopic balloon dilation and surgical myotomy. Each therapy has advantages and drawbacks and at present both are considered a first choice approach depending on patient preferences and local expertise. Surgical myotomy provides long lasting improvement in dysphagia but even when done laparoscopically is invasive and complex. Extensive acid reflux resulting in significant morbidity is routinely seen after surgical myotomy and additional anti-reflux operation is typically done at the time of the myotomy. Endoscopic balloon dilation is a simple minimally invasive outpatient procedure but improvement of symptoms tends to be shorter in duration and repeat dilations are commonly needed. Both therapies improve on dysphagia but tend to provide suboptimal control of chest pain which is one of the cardinal symptoms of achalasia.
The peroral endoscopic myotomy (POEM) was first introduced in Japan to address the suboptimal results with endoscopic balloon dilation and surgical myotomy. POEM is incisionless minimally invasive but in addition may have some further advantages over surgical myotomy including unlimited length of the myotomy with expected better control of chest pain and preservation of the anatomical anti-reflux barrier (angle of His and the cruse of the diaphragm) with expected lower incidence of acid reflux.
In Japan POEM has become the preferred modality for therapy of achalasia due to the excellent results and exceptional safety record. In the US, dedicated POEM devices were approved by the FDA just recently. As a result the bulk of the published data comes from Japan and very little is known regarding outcomes in US population. Therefore the investigators want to prospectively record our experience with POEM as done as part of routine medical care in US population. This will be a data recording study. All patients will receive standard medical care and no experimental interventions will be performed.
**Detailed Description:** Information about the subject's medical history, leading up to the need for an Achalasia treatment, the procedure itself and how the subject does after the procedure, including after the subject gets home, will be collected. This will be done by gathering relevant information from the subject's medical chart and/or by talking with the subject prior to and after the subject's medical procedures. There are no specific study procedures or tests. All information collected is part of the subject's medical care and will be collected even if the subject is not in the study.
### Conditions Module
**Conditions:**
- Achalasia
**Keywords:**
- Achalasia
### Design Module
#### Design Info
**Observational Model:** OTHER
**Time Perspective:** PROSPECTIVE
#### Enrollment Info
**Count:** 600
**Type:** ESTIMATED
**Study Type:** OBSERVATIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Information about teh subject's medical history, leading up to the need for an Achalasia treatment, the procedure itself and how the subject does after the procedure, including after the subject gets home, will be collected. This will be done by gathering relevant information from the subject's medical chart and/or by talking with the subject prior to and after the subject's medical procedures. There are no specific study procedures or tests. All information collected is part of the subject's medical care and will be collected even if the subject is not in the study.
**Intervention Names:**
- Other: Achalasia subjects
**Label:** Achalasia subjects
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Achalasia subjects
**Description:** Information about the subject's medical history, leading up to the need for an Achalasia treatment, the procedure itself and how the subject does after the procedure, including after the subject's gets home, will be collected. This will be done by gathering relevant information from the subject's medical chart and/or by talking with the subject prior to and after your medical procedures. There are no specific study procedures or tests. All information collected is part of the subject's medical care and will be collected even if the subject is not in the study.
**Name:** Achalasia subjects
**Other Names:**
- Peroral Endoscopic Myotomy
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Description:** Information about the subject's medical history, leading up to the need for an Achalasia treatment, the procedure itself and how the subject does after the procedure, including after the subject gets home, will be collected.
This information will be collected by gathering relevant information from the subject's medical chart and/or by talking with the subject prior to and after the subject's medical procedures.
There are no specific study procedures or tests. All information collected is part of the subject's medical care and will be collected even if the subject is not in the study.
**Measure:** Response to POEM treatment
**Time Frame:** approximately one week
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Age 18 years and older
* Scheduled to undergo POEM treatment
Exclusion Criteria:
* Any contraindication to performing endoscopy
* Participation in another research protocol that could interfere or influence the outcomes measures of the present study.
* The subject is unable/unwilling to give informed consent.
**Minimum Age:** 18 Years
**Sampling Method:** NON_PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
**Study Population:** Those subjects with Achalasia who have been referred for the POEM treatment for their Achalasia.
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Gainesville
**Country:** United States
**Facility:** University of Florida Health
**State:** Florida
**Zip:** 32610
#### Overall Officials
**Official 1:**
**Affiliation:** University of Florida
**Name:** Peter V Draganov, MD
**Role:** PRINCIPAL_INVESTIGATOR
### References Module
#### References
**Citation:** Perbtani YB, Mramba LK, Yang D, Suarez J, Draganov PV. Life after per-oral endoscopic myotomy: long-term outcomes of quality of life and their association with Eckardt scores. Gastrointest Endosc. 2018 Jun;87(6):1415-1420.e1. doi: 10.1016/j.gie.2018.01.019. Epub 2018 Feb 2.
**PMID:** 29410304
**Citation:** Pannu D, Yang D, Abbitt PL, Draganov PV. Prospective evaluation of CT esophagram findings after peroral endoscopic myotomy. Gastrointest Endosc. 2016 Sep;84(3):408-15. doi: 10.1016/j.gie.2016.02.022. Epub 2016 Feb 22.
**PMID:** 26907745
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000015154
- Term: Esophageal Motility Disorders
- ID: D000003680
- Term: Deglutition Disorders
- ID: D000004935
- Term: Esophageal Diseases
- ID: D000005767
- Term: Gastrointestinal Diseases
- ID: D000004066
- Term: Digestive System Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC06
- Name: Digestive System Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC09
- Name: Ear, Nose, and Throat Diseases
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M8081
- Name: Esophageal Achalasia
- Relevance: HIGH
- As Found: Achalasia
- ID: M17874
- Name: Esophageal Motility Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M17875
- Name: Esophageal Spasm, Diffuse
- Relevance: LOW
- As Found: Unknown
- ID: M6882
- Name: Deglutition Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M8085
- Name: Esophageal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M7255
- Name: Digestive System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M8883
- Name: Gastrointestinal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: T2993
- Name: Idiopathic Achalasia
- Relevance: HIGH
- As Found: Achalasia
- ID: T970
- Name: Cardiospasm
- Relevance: HIGH
- As Found: Achalasia
### Condition Browse Module - Meshes
- ID: D000004931
- Term: Esophageal Achalasia
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT03179579
**Brief Title:** Efficacy of HIPEC Combined With Systemic Chemotherapy and CRS on Peritoneal Metastases From Gastric Cancer
**Official Title:** A Phase III Study of Hyperthermic Intraperitoneal Chemotherapy (HIPEC) Combined With Systemic Chemotherapy And Cytoreductive Surgery (CRS) in the Treatment of Peritoneal Carcinomatosis From Gastric Cancer
#### Organization Study ID Info
**ID:** HIPEC-02
#### Organization
**Class:** OTHER
**Full Name:** Affiliated Cancer Hospital & Institute of Guangzhou Medical University
### Status Module
#### Completion Date
**Date:** 2022-08-01
**Type:** ESTIMATED
#### Expanded Access Info
**Last Known Status:** NOT_YET_RECRUITING
#### Last Update Post Date
**Date:** 2017-06-07
**Type:** ACTUAL
**Last Update Submit Date:** 2017-06-06
**Overall Status:** UNKNOWN
#### Primary Completion Date
**Date:** 2021-08-01
**Type:** ESTIMATED
#### Start Date
**Date:** 2017-08-01
**Type:** ESTIMATED
**Status Verified Date:** 2017-06
#### Study First Post Date
**Date:** 2017-06-07
**Type:** ACTUAL
**Study First Submit Date:** 2017-06-06
**Study First Submit QC Date:** 2017-06-06
### Sponsor Collaborators Module
#### Collaborators
**Class:** OTHER
**Name:** Tianjin Medical University Cancer Institute and Hospital
**Class:** OTHER
**Name:** Chinese PLA General Hospital
**Class:** OTHER
**Name:** Harbin Medical University
**Class:** OTHER
**Name:** Southern Medical University, China
**Class:** OTHER
**Name:** Guangdong Provincial People's Hospital
**Class:** OTHER
**Name:** Guangdong Provincial Hospital of Traditional Chinese Medicine
#### Lead Sponsor
**Class:** OTHER
**Name:** Shu-Zhong Cui
#### Responsible Party
**Investigator Affiliation:** Affiliated Cancer Hospital & Institute of Guangzhou Medical University
**Investigator Full Name:** Shu-Zhong Cui
**Investigator Title:** President of Affiliated Tumor Hospital of Guangzhou Medical
**Type:** SPONSOR_INVESTIGATOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** This project is a multi-center, prospective, randomized controlled clinical observation the safety and efficacy that stage IV limited peritoneal metastasis of gastric cancer patients accept hyperthermic intraperitoneal chemotherapy plus neoadjuvant chemotherapy with CRS and systemic chemotherapy. With advanced-stage gastric patients of confined to the peritoneal as the research object, with median progression-free survival and overall survival, adverse events as the end points.
**Detailed Description:** Peritoneal metastases in gastric cancer are considered as terminal disease with poor prognosis, the median survival time of this kind of patients is less than 1 year and even worse in China. Recently, cytoreductive surgery (CRS) combined with hyperthermic intraperitoneal chemotherapy (HIPEC) has been suggested to improve survival in patients with peritoneal metastases from gastric cancer. Even though the REGATTA trial demonstrated that the removal of the primary tumor is not necessarily beneficial, the role of operation aiming at R0 resection combined with new regimens like HIPEC and new chemotherapeutic agents is still confusing. Neoadjuvant chemotherapy prolonged OS of patients received macroscopically radical (CC0) surgery. Compared to systemic chemotherapy, HIPEC promotes chemotherapy to penetrate deeper into the cancer tissue, which needed multicenter randomized clinical trials (RCTs) to comfirm. Therefore, we has conducting prospective phase III trial of HIPEC combined with systemic chemotherapy and CRS for gastric cancer with peritoneal metastasis.
### Conditions Module
**Conditions:**
- Gastric Cancer
- Peritoneal Carcinomatosis
**Keywords:**
- Gastric Cancer
- Peritoneal Carcinomatosis
- Hyperthermic Intraperitoneal Chemotherapy
- Systemic Chemotherapy
- Cytoreductive Surgery
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** SINGLE
**Who Masked:**
- PARTICIPANT
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 88
**Type:** ESTIMATED
**Phases:**
- PHASE3
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** 1. HIPEC with paclitaxel 135 mg/m\^2+HIPEC with cisplatin 75 mg/m\^2±HIPEC with Raltitrexed 3 mg/m\^2 intraperitoneally in succession
2. 2 cycles of neoadjuvant chemotherapy: S-1 40-60 mg/m\^2, d1-d14, Bid p.o.+ oxaliplatin 130 mg/m\^2, d1, IV, every 3 weeks
3. Cytoreductive surgery
4. HIPEC with paclitaxel 135 mg/m\^2+HIPEC with cisplatin 75 mg/m\^2±HIPEC with Raltitrexed 3 mg/m\^2 intraperitoneally in succession
5. 4-6 cycles of adjuvant chemotherapy: S-1 40-60 mg/m\^2, d1-d14, Bid p.o.+ oxaliplatin 130 mg/m\^2, d1, IV, every 3 weeks
**Intervention Names:**
- Procedure: HIPEC with neoadjuvant chemotherapy
**Label:** Experimental group
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** 1. 3 cycles of neoadjuvant chemotherapy: S-1 40-60 mg/m\^2, d1-d14, Bid p.o.+ oxaliplatin 130 mg/m\^2, d1, IV, every 3 weeks
2. Cytoreductive surgery
3. 4-6 cycles of adjuvant chemotherapy: S-1 40-60 mg/m\^2, d1-d14, Bid p.o.+ oxaliplatin 130 mg/m\^2, d1, IV, every 3 weeks
**Intervention Names:**
- Procedure: Systemic chemotherapy
**Label:** Control group
**Type:** ACTIVE_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Experimental group
**Description:** HIPEC 3 times +NACT 2 cycles +CRS +HIPEC 3 times+ ACT 4-6 cycles
**Name:** HIPEC with neoadjuvant chemotherapy
**Other Names:**
- CRS with HIPEC and adjuvant cheomtherapy
**Type:** PROCEDURE
#### Intervention 2
**Arm Group Labels:**
- Control group
**Description:** NACT 3 cycles +CRS +ACT 4-6 cycles
**Name:** Systemic chemotherapy
**Other Names:**
- CRS with systemic chemotherpay
**Type:** PROCEDURE
### Outcomes Module
#### Primary Outcomes
**Description:** assess median overall survival rate during 3 years in both study arms
**Measure:** Median overall survival
**Time Frame:** 3 years
#### Secondary Outcomes
**Description:** determine percent of patients wtih Grade I-IV adverse events according to NCI criteria, Common Terminology Criteria for AE (CTCAE 4.0).
**Measure:** Risk factors for morbidity and mortality
**Time Frame:** Through study completion, an average of 1 year.
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
1. Gastic adenocarcinoma is diagnosed by histological and cytological examination.
2. Peritoneal carcinomastosis of gastric cancer is diagnosed by laparotomy or laparoscopic exploration.
3. According to Sugarbaker's peritoneal cancer index (PCI), PCI score of participant is no more than 20.
4. 18 \< Age \< 70 year old
5. Expected survival \> 3 months
6. Performance status: ECOG 0-1
7. Adequate bone marrow function Hb ≥9 g/dl (After correction in case of iron deficient anemia) WBC ≥ 3,500/mm3, Platelet ≥ 100,000/mm3
8. Adequate renal function Creatinine ≤ 1.5 mg/dl, and adequate hepatic function Bilirubin ≤ 1.5 mg/dl and AST and ALT ≤ 2.5×ULN
10. Voluntary participation after getting written informed consent.
Exclusion Criteria:
1. Except for peritoneal carcinomatosis, gastric cancer concurrently metastasized to liver, lung, para-aortic lymph node and other distant organs.
2. Extensive adhesion in peritoneal cavity
3. Previous History of other malignancies
4. Poorly controlled disease e.g. atrial fibrillation, stenocardia, cardiac insufficiency, persistent hypertension despite medicinal treatment, ejection fraction\<50%
5. Receiving other chemotherapy, radiotherapy or immunotherapy
6. Patients who are unsuitable candidates by doctor's decision
7. Without given written informed consent
**Maximum Age:** 70 Years
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Xian-Zi Yang, M.D
**Phone:** 8602066673666
**Role:** CONTACT
**Contact 2:**
**Email:** [email protected]
**Name:** Zhen Tang, M.M
**Phone:** 8602066673666
**Role:** CONTACT
#### Locations
**Location 1:**
**City:** Guangzhou
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Xianzi Yang, M.D
- **Phone:** 8602066673666
- **Role:** CONTACT
***Contact 2:***
- **Name:** shuzhong cui, M.D
- **Role:** PRINCIPAL_INVESTIGATOR
**Country:** China
**Facility:** Department of Abdominal Surgery (Section 2), Affiliated Tumor Hospital of Guangzhou Medical University
**State:** Guangdong
**Zip:** 510095
#### Overall Officials
**Official 1:**
**Affiliation:** Affiliated Cancer Hospital & Institute of Guangzhou Medical University
**Name:** Shu-Zhong Cui, M.D
**Role:** STUDY_CHAIR
### IPD Sharing Statement Module
**IPD Sharing:** NO
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000005770
- Term: Gastrointestinal Neoplasms
- ID: D000004067
- Term: Digestive System Neoplasms
- ID: D000009371
- Term: Neoplasms by Site
- ID: D000009369
- Term: Neoplasms
- ID: D000004066
- Term: Digestive System Diseases
- ID: D000005767
- Term: Gastrointestinal Diseases
- ID: D000013272
- Term: Stomach Diseases
- ID: D000009375
- Term: Neoplasms, Glandular and Epithelial
- ID: D000009370
- Term: Neoplasms by Histologic Type
- ID: D000000008
- Term: Abdominal Neoplasms
- ID: D000010532
- Term: Peritoneal Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: BC06
- Name: Digestive System Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: BC26
- Name: Wounds and Injuries
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M16064
- Name: Stomach Neoplasms
- Relevance: HIGH
- As Found: Gastric Cancer
- ID: M12307
- Name: Neoplasm Metastasis
- Relevance: LOW
- As Found: Unknown
- ID: M2454
- Name: Hyperthermia
- Relevance: LOW
- As Found: Unknown
- ID: M8464
- Name: Fever
- Relevance: LOW
- As Found: Unknown
- ID: M5534
- Name: Carcinoma
- Relevance: HIGH
- As Found: Carcinomatosis
- ID: M13443
- Name: Peritoneal Neoplasms
- Relevance: HIGH
- As Found: Peritoneal Carcinomatosis
- ID: M8886
- Name: Gastrointestinal Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M7256
- Name: Digestive System Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M7255
- Name: Digestive System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M8883
- Name: Gastrointestinal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M16062
- Name: Stomach Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M12320
- Name: Neoplasms, Glandular and Epithelial
- Relevance: LOW
- As Found: Unknown
- ID: M12315
- Name: Neoplasms by Histologic Type
- Relevance: LOW
- As Found: Unknown
- ID: M7
- Name: Abdominal Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M13441
- Name: Peritoneal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: T5486
- Name: Stomach Cancer
- Relevance: HIGH
- As Found: Gastric Cancer
### Condition Browse Module - Meshes
- ID: D000013274
- Term: Stomach Neoplasms
- ID: D000002277
- Term: Carcinoma
- ID: D000010534
- Term: Peritoneal Neoplasms
### Intervention Browse Module - Browse Branches
- Abbrev: ANeo
- Name: Antineoplastic Agents
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M252094
- Name: Raltitrexed
- Relevance: LOW
- As Found: Unknown
- ID: M19537
- Name: Paclitaxel
- Relevance: LOW
- As Found: Unknown
- ID: M231
- Name: Albumin-Bound Paclitaxel
- Relevance: LOW
- As Found: Unknown
- ID: M6182
- Name: Cisplatin
- Relevance: LOW
- As Found: Unknown
- ID: M1674
- Name: Oxaliplatin
- Relevance: LOW
- As Found: Unknown
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT00319579
**Brief Title:** Pilot Prospective Study: Long-term Health of Living Kidney Donors
**Official Title:** The Long Term Medical and Psychological Implications of Becoming a Living Kidney Donor: A Prospective Pilot Study
#### Organization Study ID Info
**ID:** R-04-204
#### Organization
**Class:** OTHER
**Full Name:** Lawson Health Research Institute
#### Secondary ID Infos
**ID:** LKD Prospective Study
### Status Module
#### Completion Date
**Date:** 2009-08
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-02-26
**Type:** ACTUAL
**Last Update Submit Date:** 2024-02-23
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2009-08
**Type:** ACTUAL
#### Start Date
**Date:** 2005-01
**Type:** ACTUAL
**Status Verified Date:** 2024-02
#### Study First Post Date
**Date:** 2006-04-27
**Type:** ESTIMATED
**Study First Submit Date:** 2006-04-27
**Study First Submit QC Date:** 2006-04-27
### Sponsor Collaborators Module
#### Collaborators
**Class:** OTHER_GOV
**Name:** Canadian Institutes of Health Research (CIHR)
#### Lead Sponsor
**Class:** OTHER
**Name:** Amit Garg
#### Responsible Party
**Investigator Affiliation:** Lawson Health Research Institute
**Investigator Full Name:** Amit Garg
**Investigator Title:** MD, MA, FRCPC, FACP, PhD
**Type:** SPONSOR_INVESTIGATOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** Kidney transplantation, a 'miracle' of modern medicine, is the preferred treatment option for End Stage Renal Disease compared to dialysis, patients who receive kidneys have a 70% reduction in risk of death, a dramatically improved quality of life and cost the health care system considerably less. As a result there are over 3000 Canadians, and 57,000 Americans on the waiting list for a kidney. To meet the shortage in cadaveric kidneys, rates of living kidney donation have nearly doubled over the last 10 years, and will continue to rise with growing demand.
Yet despite its advantages for the recipient, living kidney donation remains a complex ethical, moral and medical issue. The premise for accepting living donors is that the "minimal" risk of short and long-term medical harm realized by the donor is outweighed by the definite advantages to the recipient and potential psychosocial benefits of the altruistic gift to the donor. The only benefit for the living donor is psychological - donors experience increased self-esteem, feelings of well-being and improved health related quality of life with their altruistic act of assuming medical risk to help another. The short-term consequences of living donation are well established. On the other hand the long-term implications of living kidney donation are far less certain. This study will look at the long term implications of living kidney donation.
### Conditions Module
**Conditions:**
- Living Kidney Donors
**Keywords:**
- living kidney donors
- prospective
- medical
- psychosocial
### Design Module
#### Bio Spec
**Description:** Serum and urine samples
**Retention:** SAMPLES_WITHOUT_DNA
#### Design Info
**Observational Model:** COHORT
**Time Perspective:** PROSPECTIVE
#### Enrollment Info
**Count:** 330
**Type:** ACTUAL
**Study Type:** OBSERVATIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Living Kidney Donors with controls who have not donated a kidney and meet certain criteria at the time of the donor's donation (i.e. no hypertension, no kidney disease, etc.).
**Label:** Observation
### Outcomes Module
#### Primary Outcomes
**Measure:** Hypertension in living kidney donors
**Time Frame:** Annually for 5 years post donation
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
1. Age greater than 18 years old
2. A predicted Cockcroft-Gault creatinine clearance \> 80 mL/min
3. Average sBP \< 140 and dBP \< 90 based on 3 readings
4. A spot urine protein to creatinine ratio \< 15 mg/mmol
Exclusion Criteria:
1. A medical condition (such as cardiovascular disease, pulmonary disease, active cancer) or psychological condition (such as major affective disorder, personality disorder, a history of chemical dependence) which the local transplant centre deems unfit for donation. Blood group and immunological incompatibility (such as positive cross-match, poor HLA matches) are not reasons for exclusion
2. Evidence of a financial incentive for donation
3. A contraindication to general anaesthesia or surgery
4. A past medical history of hypertension
5. Use of antihypertensive class medications for any reason.(ACE-Is, ARBs, diuretics, beta-blockers, calcium channel blockers)
6. A past history of permanent proteinuria
7. The eligible non-donor is planning to donate their kidney within the next year
8. Unable to give informed consent
9. Unwilling to participate in the follow-up assessments at one, two, three, four and five years, or unwilling or unable to conduct home blood pressure or laboratory measurements
10. The living donor or eligible non-donor is currently pregnant
11. Despite being planned the donor nephrectomy does not take place
12. Enrolled in another clinical study which interferes with the conduct or outcomes of this study
**Healthy Volunteers:** True
**Minimum Age:** 18 Years
**Sampling Method:** NON_PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
**Study Population:** Living Kidney Donors
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Denver
**Country:** United States
**Facility:** University of Colorado Health Sciences Center
**State:** Colorado
**Zip:** 80262
**Location 2:**
**City:** West Haven
**Country:** United States
**Facility:** Yale University and VAMC
**State:** Connecticut
**Zip:** 06516
**Location 3:**
**City:** Detroit
**Country:** United States
**Facility:** Wayne State University
**State:** Michigan
**Zip:** 48201
**Location 4:**
**City:** Cleveland
**Country:** United States
**Facility:** Cleveland Clinic
**State:** Ohio
**Zip:** 44195
**Location 5:**
**City:** Richmond
**Country:** United States
**Facility:** Hume-Lee Transplant Center Renal/Pancreas Transplant Program
**State:** Virginia
**Zip:** 23298-0274
**Location 6:**
**City:** Clayton
**Country:** Australia
**Facility:** MonashMedical Centre
**State:** Victoria
**Zip:** 3168
**Location 7:**
**City:** Nedlands
**Country:** Australia
**Facility:** Sir Charles Gairdner Hospital
**State:** Western Australia
**Location 8:**
**City:** Edmonton
**Country:** Canada
**Facility:** University of Alberta
**State:** Alberta
**Zip:** T6G 2GS
**Location 9:**
**City:** Vancouver
**Country:** Canada
**Facility:** St. Paul's Hospital
**State:** British Columbia
**Zip:** V6Z 1Y6
**Location 10:**
**City:** Winnipeg
**Country:** Canada
**Facility:** University of Manitoba
**State:** Manitoba
**Zip:** R3A 1R9
**Location 11:**
**City:** Halifax
**Country:** Canada
**Facility:** Dalhousie University
**State:** Nova Scotia
**Zip:** B3H 1V8
**Location 12:**
**City:** Hamilton
**Country:** Canada
**Facility:** St. Joseph Hospital
**State:** Ontario
**Zip:** L8N 4A6
**Location 13:**
**City:** London
**Country:** Canada
**Facility:** London Health Sciences Centre
**State:** Ontario
**Zip:** N6C 6B5
**Location 14:**
**City:** Ottawa
**Country:** Canada
**Facility:** The Ottawa Hospital
**State:** Ontario
**Zip:** K1H 7W9
**Location 15:**
**City:** Toronto
**Country:** Canada
**Facility:** St. Michael's Hospital
**State:** Ontario
**Zip:** M5C 2T2
#### Overall Officials
**Official 1:**
**Affiliation:** Lawson Health Research Institute
**Name:** Amit X Garg, MD
**Role:** PRINCIPAL_INVESTIGATOR
### References Module
#### References
**Citation:** Garcia-Ochoa C, Feldman LS, Nguan C, Monroy-Caudros M, Arnold JB, Barnieh L, Boudville N, Cuerden MS, Dipchand C, Gill JS, Karpinski M, Klarenbach S, Knoll G, Lok CE, Miller M, Prasad GVR, Sontrop JM, Storsley L, Garg AX. Impact of Perioperative Complications on Living Kidney Donor Health-Related Quality of Life and Mental Health: Results From a Prospective Cohort Study. Can J Kidney Health Dis. 2021 Aug 11;8:20543581211037429. doi: 10.1177/20543581211037429. eCollection 2021.
**PMID:** 34394947
**Citation:** Garcia-Ochoa C, Feldman LS, Nguan C, Monroy-Cuadros M, Arnold J, Boudville N, Cuerden M, Dipchand C, Eng M, Gill J, Gourlay W, Karpinski M, Klarenbach S, Knoll G, Lentine KL, Lok CE, Luke P, Prasad GVR, Sener A, Sontrop JM, Storsley L, Treleaven D, Garg AX; Donor Nephrectomy Outcomes Research (DONOR) Network. Perioperative Complications During Living Donor Nephrectomy: Results From a Multicenter Cohort Study. Can J Kidney Health Dis. 2019 Jul 18;6:2054358119857718. doi: 10.1177/2054358119857718. eCollection 2019.
**PMID:** 31367455
## Derived Section
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT05653479
**Brief Title:** Safety and Blood Levels After a Single Injection of UPB-101 in Healthy Japanese and Non-Japanese Non-East Asian Adults
**Official Title:** A Randomized, Open-label, Parallel-group, Ethno-bridging Study Comparing the Pharmacokinetics and Safety of a Single Dose of UPB-101 in Healthy Japanese and Non-Japanese Non-East Asian Adults
#### Organization Study ID Info
**ID:** UPB-CP-02
#### Organization
**Class:** INDUSTRY
**Full Name:** Upstream Bio Inc.
### Status Module
#### Completion Date
**Date:** 2023-05-13
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2023-06-15
**Type:** ACTUAL
**Last Update Submit Date:** 2023-06-12
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2023-04-07
**Type:** ACTUAL
#### Start Date
**Date:** 2022-12-05
**Type:** ACTUAL
**Status Verified Date:** 2023-02
#### Study First Post Date
**Date:** 2022-12-16
**Type:** ACTUAL
**Study First Submit Date:** 2022-12-07
**Study First Submit QC Date:** 2022-12-07
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Upstream Bio Inc.
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** The goals of this clinical study are to characterize and compare the safety, tolerability, blood levels of UPB-101 when given to healthy Japanese and non-Japanese non-East Asian (NJNEA) adults. Eligible participant will be assigned to one of the 4 planned dosing treatment groups. Treatment groups 1, 2 and 3 will consist of Japanese adults who will be administered a single subcutaneous (SC) dose of UPB-101 (at 3 different strengths). Treatment group 4 will consist of NJNEA participants who will receive a single SC dose of UPB-101. All treatment groups will enroll and run in parallel.
**Detailed Description:** This is a randomized, open-label, parallel group, ethno-bridging study comparing the pharmacokinetics and safety of a single dose of UPB-101 in healthy Japanese and NJNEA adults. The study will include 4 planned dosing treatment groups. Treatment groups 1, 2 and 3 will consist of Japanese adults who will be administered a single subcutaneous (SC) dose of UPB-101 (at 3 different strengths). Treatment group 4 will consist of NJNEA participants who will receive a single SC dose of UPB-101. All treatment groups will enroll and run in parallel. Japanese participants will be enrolled and randomized on Day 1 into treatment groups 1, 2 or 3 and NJNEA participants will be assigned to treatment group 4.
Eight participants will be enrolled per treatment group, all will receive a single dose of UPB-101. Thus, a total of 32 male and female participants will be enrolled in the study (24 Japanese participants in treatment groups 1-3 and 8 NJNEA participants in treatment group 4).
### Conditions Module
**Conditions:**
- Healthy
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
**Intervention Model Description:** All treatment groups will enroll and run in parallel. Japanese participants will be enrolled and randomly assigned, on Day 1, to treatment groups 1, 2 or 3. NJNEA participants will be assigned to treatment group 4.
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 32
**Type:** ACTUAL
**Phases:**
- PHASE1
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Single subcutaneous injection of a dose of UPB-101 (formerly ASP7266) in 8 Japanese participants
**Intervention Names:**
- Drug: UPB-101
**Label:** Treatment Group 1
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Single subcutaneous injection of a dose of UPB-101 (formerly ASP7266) in 8 Japanese participants
**Intervention Names:**
- Drug: UPB-101
**Label:** Treatment Group 2
**Type:** EXPERIMENTAL
#### Arm Group 3
**Description:** Single subcutaneous injection of a dose of UPB-101 (formerly ASP7266) in 8 Japanese participants
**Intervention Names:**
- Drug: UPB-101
**Label:** Treatment Group 3
**Type:** EXPERIMENTAL
#### Arm Group 4
**Description:** Single subcutaneous injection of a dose of UPB-101 (formerly ASP7266) in 8 Non-Japanese Non-East Asian participants
**Intervention Names:**
- Drug: UPB-101
**Label:** Treatment Group 4
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Treatment Group 1
- Treatment Group 2
- Treatment Group 3
- Treatment Group 4
**Description:** UPB-101 Subcutaneous injection
**Name:** UPB-101
**Other Names:**
- Formerly ASP7266
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Measure:** Maximum observed concentration of UPB-101
**Time Frame:** Baseline through Day 85
**Measure:** Time to maximum observed concentration of UPB-101
**Time Frame:** Baseline through Day 85
**Measure:** Area under the concentration-time curve from time zero up to the last quantifiable concentration of UPB-101
**Time Frame:** Baseline through Day 85
#### Secondary Outcomes
**Measure:** Number of participants with treatment-emergent adverse events and serious adverse events
**Time Frame:** Baseline through Day 85
### Eligibility Module
**Eligibility Criteria:** Main Inclusion Criteria:
1. Male or female, aged 18 to 40
2. Body mass index (BMI) between 18 and 25 kg/m2
3. For Japanese (treatment groups 1, 2 and 3), participants must be: a) born in Japan, holding a Japanese passport, b) not living outside Japan for more than 5 years at the date of signing informed consent, c) have all 4 grandparents Japanese. For NJNEA treatment group 4, participants must be: a) Non-Japanese, b) Non-East Asian (Chinese, Korean, Mongolian or Taiwanese).
4. Healthy, as defined by: a) the absence of clinically significant illness and surgery within four weeks prior to dosing. b) the absence of clinically significant history of neurological, endocrine, cardiovascular, respiratory, haematological, immunological, psychiatric, gastrointestinal, renal, hepatic, and metabolic disease.
5. Agrees to follow the required contraceptive techniques
6. Agrees not to donate sperm or ova from the time of the administration of study medication until three months later (120 days)
Main Exclusion Criteria:
1. Current or recurrent disease (or condition), which may put the participant at risk, influence the results of the study, or otherwise affect their ability to participate in the study
2. Vital signs consistently outside the normal range at Screening or Day -1 and any other abnormal findings or vital signs, ECG, telemetry, physical examination or laboratory evaluation of blood and urine samples that the Investigator judges as likely to interfere with the study or pose an additional risk in participating.
3. Previous exposure or current infection with hepatitis B, C or tuberculosis (TB), other active recent infection, or history of any untreated or unresolved infection, including parasitic infection
4. Pregnant or breastfeeding female
5. Previous exposure to the study drug or known allergy/sensitivity to any of its ingredients
6. Positive test results for alcohol or drugs of abuse (including cotinine) at Screening or Day -1, or history or clinical evidence of substance and/or alcohol abuse within 2 years before screening
7. Use of tobacco or other nicotine-containing products in any form within 3 months prior to Day 1
8. Any recent vaccination, prescription or over-the-counter medication, including herbal remedies or dietary supplements
9. Recent donation of blood or blood products
**Healthy Volunteers:** True
**Maximum Age:** 40 Years
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** London
**Country:** United Kingdom
**Facility:** Richmond Pharmacology
#### Overall Officials
**Official 1:**
**Affiliation:** Upstream Bio
**Name:** Chaim Brickman
**Role:** STUDY_DIRECTOR
### IPD Sharing Statement Module
**IPD Sharing:** NO
## Derived Section
### Misc Info Module
#### Submission Tracking
- Estimated Results First Submit Date: 2024-04-05
##### Submission Infos
- MCP Release N: Unknown
- Release Date: 2024-04-05
- Reset Date: Unknown
- Unrelease Date: Unknown
- Unrelease Date Unknown: Unknown
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT00868179
**Brief Title:** A Cohort Study With 100 Subjects Having a Primary Total Knee Replacement, Taking Pradax Post Discharge for Ten Days
**Official Title:** A Cohort (Follow Up) Study With 100 Subjects Having a Primary Total Knee Replacement, Taking Pradax (Tablet) Post Discharge for Ten Days, Without the Need for Routine Coagulation Monitoring and Dose Adjustment
#### Organization Study ID Info
**ID:** Pradax
#### Organization
**Class:** OTHER
**Full Name:** Nova Scotia Health Authority
### Status Module
#### Completion Date
**Date:** 2012-04
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2022-11-14
**Type:** ACTUAL
**Last Update Submit Date:** 2022-11-08
**Overall Status:** WITHDRAWN
#### Primary Completion Date
**Date:** 2012-04
**Type:** ESTIMATED
#### Start Date
**Date:** 2009-04
**Status Verified Date:** 2022-11
#### Study First Post Date
**Date:** 2009-03-24
**Type:** ESTIMATED
**Study First Submit Date:** 2009-03-23
**Study First Submit QC Date:** 2009-03-23
**Why Stopped:** study never started
### Sponsor Collaborators Module
#### Collaborators
**Class:** OTHER
**Name:** Nova Scotia Health Authority
#### Lead Sponsor
**Class:** OTHER
**Name:** Ross Leighton
#### Responsible Party
**Investigator Affiliation:** Nova Scotia Health Authority
**Investigator Full Name:** Ross Leighton
**Investigator Title:** Orthopedic Surgeon
**Type:** SPONSOR_INVESTIGATOR
### Oversight Module
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** Currently standard of care for preventing blood clots in total knee replacement patients is the drug Fragmin which is a daily injection for 10 days after surgery. Patients are in hospital for 3 to 5 days after total knee replacement surgery and patients are taught in hospital to do their injections.
The investigators would like to introduce the drug Pradax. Pradax is a Health Canada approved once a day, oral drug that may prevent blood clots in the patient's leg. In this study the patient will receive the standard of care injection drug Fragmin while in hospital but on discharge home the patient will take the oral drug Pradax daily (2 tablets 110mg) for 10 days.
**Detailed Description:** We will enroll one hundred patients who are scheduled to have a total knee replacement. If you have signed the consent form for this study you will receive the standard of care treatment for blood clots, which is a daily injection drug called Fragmin. On the day of discharge from hospital study patients will receive a ten day supply of oral Pradax. to be taken once a day. The Orthopaedic research coordinator will call the study patients at post discharge day three and five to check on compliance and or concerns. All total knee replacement patients whether in study or not are seen in the Orthopaedic clinic at post op day 10 for staple removal and again at the 6 week point, 3 month, and 6 month and 1 year point. Standard of care x-rays are done also at these visits.
### Conditions Module
**Conditions:**
- Thromboembolism
**Keywords:**
- Thrombosis
- total knee replacement
### Design Module
#### Design Info
**Allocation:** NA
**Intervention Model:** SINGLE_GROUP
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Type:** ACTUAL
**Phases:**
- PHASE4
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** This is the only arm in the study and all will follow the same protocol for the study which is taking the pradax after total knee replacement
**Intervention Names:**
- Drug: Pradax
**Label:** Pradax
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Pradax
**Description:** Pradax (dabigatran etexilate) is a prodrug that when converted to the active from in the liver and plasma it becomes a reversible, competitive direct thrombin inhibitor. Thrombin catalyses the conversion of soluble fibrinogen into soluble fibrin, the final step in the coagulation cascade. Pradax competitively blocks the active site of both free and clot-bound thrombin, preventing the development of a thrombus. they will take 110 mg 1-4 hours post op. and 220mg daily until 10 days post op.
**Name:** Pradax
**Other Names:**
- Pradax dabigatran etexilate
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Measure:** The primary efficacy outcome will be the clinical evidence of any deep vein thrombosis, non-fatal pulmonary embolism or death from any causes within 28 days after surgery. The primary safety outcome is major bleeding
**Time Frame:** 28 day post op and 6 months radiograophically
#### Secondary Outcomes
**Measure:** Secondary efficacy end points will be the radiographic assessment of healing, the clinical assessment of weight-¬bearing status at 6 months post treatment, and the incidence of additional surgical/medical interventions to promote healing.
**Time Frame:** 6 Months
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Patients are eligible for the study if they are adult age, being greater than 18 years of age and less than 95 years of age. They must have the indications for a total knee replacement and have sufficiently passed the medically necessary tests by their surgeon to warrant total knee replacement.
Exclusion Criteria:
* Patients with active bleeding, or high risk of bleeding that contra-indicates the use of Pradax
* Renal or liver contra-indication necessitating adjustments of its dose.
* Clinically significant liver disease,
* Concomitant use of Proteus Ace inhibitors
* The use of the human immunitive deficiency virus
* The use of fibrinolynic agents
* Planned intermittent pneumatic compression or requirement of ongoing anticoagulation therapy
* Pregnancy
* Breast-feeding.
**Maximum Age:** 95 Years
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Halifax
**Country:** Canada
**Facility:** Halifax Infirmary
**State:** Nova Scotia
**Zip:** B3h 1V7
#### Overall Officials
**Official 1:**
**Affiliation:** Nova Scotia Health Authority
**Name:** Ross K Leighton, MD FRCSC
**Role:** PRINCIPAL_INVESTIGATOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000016769
- Term: Embolism and Thrombosis
- ID: D000014652
- Term: Vascular Diseases
- ID: D000002318
- Term: Cardiovascular Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC14
- Name: Heart and Blood Diseases
- Abbrev: All
- Name: All Conditions
### Condition Browse Module - Browse Leaves
- ID: M16682
- Name: Thromboembolism
- Relevance: HIGH
- As Found: Thromboembolism
- ID: M16686
- Name: Thrombosis
- Relevance: LOW
- As Found: Unknown
- ID: M7784
- Name: Embolism
- Relevance: LOW
- As Found: Unknown
- ID: M19128
- Name: Embolism and Thrombosis
- Relevance: LOW
- As Found: Unknown
- ID: M17400
- Name: Vascular Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000013923
- Term: Thromboembolism
### Intervention Browse Module - Ancestors
- ID: D000000991
- Term: Antithrombins
- ID: D000015842
- Term: Serine Proteinase Inhibitors
- ID: D000011480
- Term: Protease Inhibitors
- ID: D000004791
- Term: Enzyme Inhibitors
- ID: D000045504
- Term: Molecular Mechanisms of Pharmacological Action
- ID: D000000925
- Term: Anticoagulants
### Intervention Browse Module - Browse Branches
- Abbrev: Hemat
- Name: Hematinics
- Abbrev: All
- Name: All Drugs and Chemicals
- Abbrev: AnCoag
- Name: Anticoagulants
- Abbrev: Coag
- Name: Coagulants
- Abbrev: Infe
- Name: Anti-Infective Agents
- Abbrev: AA
- Name: Amino Acids
### Intervention Browse Module - Browse Leaves
- ID: M11110
- Name: Liver Extracts
- Relevance: LOW
- As Found: Unknown
- ID: M490
- Name: Dabigatran
- Relevance: HIGH
- As Found: Must
- ID: M16676
- Name: Thrombin
- Relevance: LOW
- As Found: Unknown
- ID: M4307
- Name: Antithrombins
- Relevance: LOW
- As Found: Unknown
- ID: M4306
- Name: Antithrombin III
- Relevance: LOW
- As Found: Unknown
- ID: M14343
- Name: Protease Inhibitors
- Relevance: LOW
- As Found: Unknown
- ID: M18391
- Name: Serine Proteinase Inhibitors
- Relevance: LOW
- As Found: Unknown
- ID: M19609
- Name: HIV Protease Inhibitors
- Relevance: LOW
- As Found: Unknown
- ID: M7951
- Name: Enzyme Inhibitors
- Relevance: LOW
- As Found: Unknown
- ID: M4244
- Name: Anticoagulants
- Relevance: LOW
- As Found: Unknown
- ID: T18
- Name: Serine
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: D000069604
- Term: Dabigatran
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT02751879
**Brief Title:** Real World Data on Gi(l)Otrif® Dose Adjustment
**Official Title:** Real-world Data on Gi(l)Otrif® Dose Adjustment in First-line Treatment, TKI-naïve, Advanced Non-small Cell Lung Cancer Patients With EGFR Activating Mutations
#### Organization Study ID Info
**ID:** 1200.270
#### Organization
**Class:** INDUSTRY
**Full Name:** Boehringer Ingelheim
### Status Module
#### Completion Date
**Date:** 2017-09-30
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2019-08-08
**Type:** ACTUAL
**Last Update Submit Date:** 2019-06-24
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2017-09-30
**Type:** ACTUAL
#### Results First Post Date
**Date:** 2019-08-08
**Type:** ACTUAL
**Results First Submit Date:** 2018-09-12
**Results First Submit QC Date:** 2019-06-24
#### Start Date
**Date:** 2016-11-24
**Type:** ACTUAL
**Status Verified Date:** 2019-06
#### Study First Post Date
**Date:** 2016-04-26
**Type:** ESTIMATED
**Study First Submit Date:** 2016-04-22
**Study First Submit QC Date:** 2016-04-22
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Boehringer Ingelheim
#### Responsible Party
**Type:** SPONSOR
### Description Module
**Brief Summary:** This is a non-interventional, multi-country, multi-site study based on existing data from medical records of patients treated with Gi(l)otrif® as part of the routine treatment according to the approved label. Data from real-world will help to understand if dose modifications are done similar as in LUX-Lung 3 trial and if the outcome on safety and effectiveness are as in trial settings. Furthermore, data on modified starting doses, the underlying reasons and effects on safety and outcome are needed.
### Conditions Module
**Conditions:**
- Carcinoma, Non-Small-Cell Lung
### Design Module
#### Design Info
**Observational Model:** COHORT
**Time Perspective:** RETROSPECTIVE
#### Enrollment Info
**Count:** 228
**Type:** ACTUAL
**Study Type:** OBSERVATIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** patients with Epidermal growth factor receptor (EGFR) mutation (common mutations), TKI-naïve advanced non small cell lung cancer (NSCLC), treated with Gi(l)otrif® as the first-line treatment for NSCLC within the approved label
**Label:** Non small cell lung cancer (NSCLC)
### Outcomes Module
#### Primary Outcomes
**Description:** An adverse drug reaction (ADR) is defined as a response to a medicinal product which is noxious and unintended. Grade 1, Grade 2, Grade 3 and Grade 4 ADR severity classes were considered for assessment of this outcome.
**Measure:** Percentage of Patients With Adverse Drug Reactions (ADR) by Severity Class.
**Time Frame:** From signing the informed consent onwards until the end of the study, up to 104 weeks.
**Description:** Time on treatment with Gi(l)otrif® in real-world setting has been calculated in this assessment. Time on treatment refers to time to treatment failure with Gi(l)otrif®
**Measure:** Time on Treatment With Gi(l)Otrif®
**Time Frame:** From first dose of Gi(l)otrif® treatment to last dose of Gi(l)otrif® treatment, up to 104 weeks.
**Description:** Time to progression was calculated from the date of first dose of Gi(l)otrif® treatment to the earliest date of documented progression (clinical, radiographic or both clinical/radiographic progression) or tumour-related death, whatever occurred first.
**Measure:** Time to Progression With Gi(l)Otrif®
**Time Frame:** From first dose of Gi(l)otrif® treatment to last dose of Gi(l)otrif® treatment, up to 104 weeks.
#### Secondary Outcomes
**Description:** Percentage of patients with a modified starting dose that is dose other than the recommended 40 mg daily of Gi(l)otrif® has been calculated to assess this outcome measure.
**Measure:** Percentage of Patients With a Modified Starting Dose of Gi(l)Otrif®
**Time Frame:** From first dose of Gi(l)otrif® treatment to last dose of Gi(l)otrif® treatment, up to 104 weeks.
**Description:** Different reasons for starting dose with modified dose that is dose other than recommended 40 mg once daily.
**Measure:** Percentage of Patients With Reasons for Modified Starting Dose of Gi(l)Otrif®
**Time Frame:** From first dose of Gi(l)otrif® treatment to last dose of Gi(l)otrif® treatment, up to 104 weeks.
### Eligibility Module
**Eligibility Criteria:** Inclusion criteria:
1. Age = 18 years
2. Patients with Epidermal growth factor receptor (EGFR) mutation (common mutations), tyrosine kinase inhibitors (TKI)-naïve advanced non small cell lung cancer (NSCLC), treated with Gi(l)otrif® as the first-line treatment for NSCLC within the approved label
3. Signed and dated written informed consent per regulations. (Exemption of a written informed consent for retrospective observational studies in some countries per local regulations and legal requirements.)
Exclusion criteria:
1. Any contraindication to Gi(l)otrif® as specified in label.
2. Patients with uncommon mutations are excluded as uncommon mutations are not within label in all participating countries (e.g. USA).
3. Patients still on treatment with Gi(l)otrif® will be excluded unless treatment period is \> or = 6 months.
4. Patients treated with Gi(l)otrif® within an interventional trial.
**Minimum Age:** 18 Years
**Sampling Method:** NON_PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
**Study Population:** NSCLC patients
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Bronx
**Country:** United States
**Facility:** Montefiore Medical Center
**State:** New York
**Zip:** 10461
**Location 2:**
**City:** Charlotte
**Country:** United States
**Facility:** Levine Cancer Institute
**State:** North Carolina
**Zip:** 28201
**Location 3:**
**City:** Wien
**Country:** Austria
**Facility:** SMZ Baumgartner Hoehe Otto Wagner Spital
**Zip:** 1140
**Location 4:**
**City:** Vancouver
**Country:** Canada
**Facility:** BC Cancer Agency - Vancouver
**State:** British Columbia
**Zip:** V5Z 4E6
**Location 5:**
**City:** Besancon
**Country:** France
**Facility:** HOP Jean Minjoz
**Zip:** 25030
**Location 6:**
**City:** Dijon Cedex
**Country:** France
**Facility:** HOP Dijon, Cardio-Pneumo, Dijon
**Zip:** 21079
**Location 7:**
**City:** Paris
**Country:** France
**Facility:** HOP Européen G. Pompidou
**Zip:** 75908
**Location 8:**
**City:** Paris
**Country:** France
**Facility:** HOP Tenon
**Zip:** 75970
**Location 9:**
**City:** Essen
**Country:** Germany
**Facility:** Ruhrlandklinik, Westdeutsches Lungenzentrum am Universitätsklinikum Essen gGmbH
**Zip:** 45147
**Location 10:**
**City:** Nürnberg
**Country:** Germany
**Facility:** Klinikum Nürnberg
**Zip:** 90419
**Location 11:**
**City:** Oldenburg
**Country:** Germany
**Facility:** Pius-Hospital, Oldenburg
**Zip:** 26121
**Location 12:**
**City:** Alessandria
**Country:** Italy
**Facility:** Azienda Ospedaliera Santi Antonio e Biagio e Cesare Arrigo
**Zip:** 15121
**Location 13:**
**City:** Lecce
**Country:** Italy
**Facility:** Azienda Ospedaliera Vito Fazzi
**Zip:** 73100
**Location 14:**
**City:** Aichi, Nagoya
**Country:** Japan
**Facility:** Aichi Cancer Center Hospital
**Zip:** 464-8681
**Location 15:**
**City:** Okayama, Kurashiki
**Country:** Japan
**Facility:** Kurashiki Central Hospital
**Zip:** 710-8602
**Location 16:**
**City:** Daegu
**Country:** Korea, Republic of
**Facility:** Yeungnam University Medical Center
**Zip:** 705-703
**Location 17:**
**City:** Jeonju
**Country:** Korea, Republic of
**Facility:** Chonbuk National University Hospital
**Zip:** 54907
**Location 18:**
**City:** Pusan
**Country:** Korea, Republic of
**Facility:** Pusan National Univ. Hosp
**Zip:** 49241
**Location 19:**
**City:** Ciudad De México
**Country:** Mexico
**Facility:** Centro Medico ABC
**Zip:** 01120
**Location 20:**
**City:** Monterrey
**Country:** Mexico
**Facility:** Organización para Cuidado Integral en Oncología S.A de C.V
**Zip:** 64060
**Location 21:**
**City:** Zapopan
**Country:** Mexico
**Facility:** Unidad de Cancerologia
**Zip:** 45050
**Location 22:**
**City:** Bydgoszcz
**Country:** Poland
**Facility:** Medical Practice,Bogdan Zurawski,Private Practice,Bydgoszcz
**Zip:** 85796
**Location 23:**
**City:** Cracow
**Country:** Poland
**Facility:** Grzegorz Czyzewicz Specialised Medical Practice, Cracow
**Zip:** 31331
**Location 24:**
**City:** Poznan
**Country:** Poland
**Facility:** Greater PL Cent.Pulmo.&Thor.Surg.Eugenia&Janusz Zeyland
**Zip:** 60-569
**Location 25:**
**City:** Singapore
**Country:** Singapore
**Facility:** National University Hospital
**Zip:** 119228
**Location 26:**
**City:** Singapore
**Country:** Singapore
**Facility:** National Cancer Centre
**Zip:** 169610
**Location 27:**
**City:** Badalona (Barcelona)
**Country:** Spain
**Facility:** Hospital Germans Trias i Pujol
**Zip:** 08916
**Location 28:**
**City:** Madrid
**Country:** Spain
**Facility:** Hospital La Princesa
**Zip:** 28006
**Location 29:**
**City:** Taichung
**Country:** Taiwan
**Facility:** China Medical University Hospital
**Zip:** 40447
#### Overall Officials
**Official 1:**
**Affiliation:** Boehringer Ingelheim
**Name:** Boehringer Ingelheim
**Role:** STUDY_CHAIR
### References Module
#### References
**Citation:** Halmos B, Tan EH, Soo RA, Cadranel J, Lee MK, Foucher P, Hsia TC, Hochmair M, Griesinger F, Hida T, Kim E, Melosky B, Marten A, Carcereny E. Impact of afatinib dose modification on safety and effectiveness in patients with EGFR mutation-positive advanced NSCLC: Results from a global real-world study (RealGiDo). Lung Cancer. 2019 Jan;127:103-111. doi: 10.1016/j.lungcan.2018.10.028. Epub 2018 Nov 2.
**PMID:** 30642537
## Document Section
### Large Document Module
#### Large Docs
- Date: 2016-03-01
- Filename: Prot_000.pdf
- Has ICF: False
- Has Protocol: True
- Has SAP: False
- Label: Study Protocol
- Size: 1535334
- Type Abbrev: Prot
- Upload Date: 2018-09-11T09:50
- Date: 2018-01-17
- Filename: SAP_001.pdf
- Has ICF: False
- Has Protocol: False
- Has SAP: True
- Label: Statistical Analysis Plan
- Size: 118143
- Type Abbrev: SAP
- Upload Date: 2018-09-11T09:50
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000002283
- Term: Carcinoma, Bronchogenic
- ID: D000001984
- Term: Bronchial Neoplasms
- ID: D000008175
- Term: Lung Neoplasms
- ID: D000012142
- Term: Respiratory Tract Neoplasms
- ID: D000013899
- Term: Thoracic Neoplasms
- ID: D000009371
- Term: Neoplasms by Site
- ID: D000009369
- Term: Neoplasms
- ID: D000008171
- Term: Lung Diseases
- ID: D000012140
- Term: Respiratory Tract Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: All
- Name: All Conditions
- Abbrev: BC08
- Name: Respiratory Tract (Lung and Bronchial) Diseases
### Condition Browse Module - Browse Leaves
- ID: M5534
- Name: Carcinoma
- Relevance: LOW
- As Found: Unknown
- ID: M11172
- Name: Lung Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M5546
- Name: Carcinoma, Non-Small-Cell Lung
- Relevance: HIGH
- As Found: Carcinoma, Non-Small-Cell Lung
- ID: M5540
- Name: Carcinoma, Bronchogenic
- Relevance: LOW
- As Found: Unknown
- ID: M5260
- Name: Bronchial Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M14979
- Name: Respiratory Tract Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M16658
- Name: Thoracic Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M11168
- Name: Lung Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M14977
- Name: Respiratory Tract Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000002289
- Term: Carcinoma, Non-Small-Cell Lung
### Intervention Browse Module - Browse Branches
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M11900
- Name: Mitogens
- Relevance: LOW
- As Found: Unknown
### Misc Info Module
- Version Holder: 2024-05-24
## Results Section
### Adverse Events Module
**Description:** An adverse event (AE) is defined as any untoward medical occurrence in a patient or clinical investigation subject administered a medicinal product and which does not necessarily have a causal relationship with this treatment. Full Analysis set (FAS): All registered patients with informed consent (as applicable with local regulations) and at least one administration of Gi(l) otrif® was used for this assessment. This was a single-arm retrospective analysis.
#### Event Groups
**Group ID:** EG000
**Title:** Gi(l)Otrif ≥ 40 mg
**Deaths Num Affected:** 8
**Deaths Num At Risk:** 157
**Description:** Patients were orally treated with starting dose of greater than or equal to 40 mg Gi(l)otrif tablet once daily.
**ID:** EG000
**Other Num Affected:** 147
**Other Num at Risk:** 157
**Serious Number Affected:** 11
**Serious Number At Risk:** 157
**Title:** Gi(l)Otrif ≥ 40 mg
**Group ID:** EG001
**Title:** Gi(l)Otrif ≤ 30 mg
**Deaths Num At Risk:** 71
**Description:** Patients were orally treated with starting dose of less than or equal to 30 mg Gi(l)otrif tablet once daily.
**ID:** EG001
**Other Num Affected:** 68
**Other Num at Risk:** 71
**Serious Number Affected:** 7
**Serious Number At Risk:** 71
**Title:** Gi(l)Otrif ≤ 30 mg
**Frequency Threshold:** 5
#### Other Events
**Term:** Diarrhoea
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 20.1
**Term:** Stomatitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 20.1
**Term:** Paronychia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 20.1
**Term:** Rash pustular
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 20.1
**Term:** Alopecia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** MedDRA 20.1
**Term:** Dermatitis acneiform
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** MedDRA 20.1
**Term:** Dry skin
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** MedDRA 20.1
**Term:** Pruritus
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** MedDRA 20.1
**Term:** Rash maculo-papular
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** MedDRA 20.1
**Term:** Nausea
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 20.1
**Term:** Dysgeusia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Nervous system disorders
**Source Vocabulary:** MedDRA 20.1
**Term:** Nail ridging
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Skin and subcutaneous tissue disorders
**Source Vocabulary:** MedDRA 20.1
#### Serious Events
**Term:** Leukocytosis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Blood and lymphatic system disorders
**Source Vocabulary:** MedDRA 20.1
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 157
**Group ID:** EG001
**Num At Risk:** 71
**Term:** Constipation
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 20.1
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 157
**Group ID:** EG001
**Num At Risk:** 71
**Term:** Diarrhoea
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 20.1
##### Stats
**Group ID:** EG000
**Num Affected:** 6
**Num At Risk:** 157
**Group ID:** EG001
**Num Affected:** 4
**Num At Risk:** 71
**Term:** Dysphagia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 20.1
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 157
**Group ID:** EG001
**Num At Risk:** 71
**Term:** Gastrooesophageal reflux disease
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 20.1
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 157
**Group ID:** EG001
**Num At Risk:** 71
**Term:** Nausea
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 20.1
##### Stats
**Group ID:** EG000
**Num At Risk:** 157
**Group ID:** EG001
**Num Affected:** 2
**Num At Risk:** 71
**Term:** Pancreatitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 20.1
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 157
**Group ID:** EG001
**Num At Risk:** 71
**Term:** Stomatitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Gastrointestinal disorders
**Source Vocabulary:** MedDRA 20.1
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 157
**Group ID:** EG001
**Num At Risk:** 71
**Term:** Pain
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** MedDRA 20.1
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 157
**Group ID:** EG001
**Num At Risk:** 71
**Term:** Sudden death
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** General disorders
**Source Vocabulary:** MedDRA 20.1
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 157
**Group ID:** EG001
**Num At Risk:** 71
**Term:** Abscess
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
**Source Vocabulary:** MedDRA 20.1
##### Stats
**Group ID:** EG000
**Num At Risk:** 157
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 71
**Term:** Hyponatraemia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Metabolism and nutrition disorders
**Source Vocabulary:** MedDRA 20.1
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 157
**Group ID:** EG001
**Num At Risk:** 71
**Term:** Hypoxia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** MedDRA 20.1
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 157
**Group ID:** EG001
**Num At Risk:** 71
**Term:** Pneumonitis
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** MedDRA 20.1
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 157
**Group ID:** EG001
**Num At Risk:** 71
**Term:** Respiratory failure
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
**Source Vocabulary:** MedDRA 20.1
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 157
**Group ID:** EG001
**Num At Risk:** 71
**Term:** Embolism
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Vascular disorders
**Source Vocabulary:** MedDRA 20.1
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 157
**Group ID:** EG001
**Num At Risk:** 71
**Time Frame:** From signing the informed consent onwards until the end of the study, up to 104 weeks.
## Results Section - Baseline Characteristics Module
### Denomination Counts
**Group ID:** BG000
**Value:** 157
**Group ID:** BG001
**Value:** 71
**Group ID:** BG002
**Value:** 228
**Units:** Participants
### Group
**ID:** BG000
**Title:** Gi(l)Otrif ≥ 40 mg
**Description:** Patients were orally treated with starting dose of greater than or equal to 40 mg Gi(l)otrif tablet once daily.
### Group
**ID:** BG001
**Title:** Gi(l)Otrif ≤ 30 mg
**Description:** Patients were orally treated with starting dose of less than or equal to 30 mg Gi(l)otrif tablet once daily.
### Group
**ID:** BG002
**Title:** Total
**Description:** Total of all reporting groups
### Measure
#### Measurement
**Group ID:** BG000
**Spread:** 12.00
**Value:** 64.50
#### Measurement
**Group ID:** BG001
**Spread:** 10.84
**Value:** 68.10
#### Measurement
**Group ID:** BG002
**Spread:** 11.74
**Value:** 65.62
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 90
#### Measurement
**Group ID:** BG001
**Value:** 48
#### Measurement
**Group ID:** BG002
**Value:** 138
**Category Title:** Female
#### Measurement
**Group ID:** BG000
**Value:** 67
#### Measurement
**Group ID:** BG001
**Value:** 23
#### Measurement
**Group ID:** BG002
**Value:** 90
**Category Title:** Male
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 16
#### Measurement
**Group ID:** BG001
**Value:** 9
#### Measurement
**Group ID:** BG002
**Value:** 25
**Category Title:** Hispanic or Latino
#### Measurement
**Group ID:** BG000
**Value:** 120
#### Measurement
**Group ID:** BG001
**Value:** 56
#### Measurement
**Group ID:** BG002
**Value:** 176
**Category Title:** Not Hispanic or Latino
#### Measurement
**Group ID:** BG000
**Value:** 21
#### Measurement
**Group ID:** BG001
**Value:** 6
#### Measurement
**Group ID:** BG002
**Value:** 27
**Category Title:** Unknown or Not Reported
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 1
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 1
**Category Title:** American Indian or Alaska Native
#### Measurement
**Group ID:** BG000
**Value:** 74
#### Measurement
**Group ID:** BG001
**Value:** 26
#### Measurement
**Group ID:** BG002
**Value:** 100
**Category Title:** Asian
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 1
#### Measurement
**Group ID:** BG002
**Value:** 1
**Category Title:** Native Hawaiian or Other Pacific Islander
#### Measurement
**Group ID:** BG000
**Value:** 1
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 1
**Category Title:** Black or African American
#### Measurement
**Group ID:** BG000
**Value:** 59
#### Measurement
**Group ID:** BG001
**Value:** 37
#### Measurement
**Group ID:** BG002
**Value:** 96
**Category Title:** White
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 0
**Category Title:** More than one race
#### Measurement
**Group ID:** BG000
**Value:** 22
#### Measurement
**Group ID:** BG001
**Value:** 7
#### Measurement
**Group ID:** BG002
**Value:** 29
**Category Title:** Unknown or Not Reported
**Class Title:**
**Denomination Units Selected:**
## Results Section - Baseline Characteristics Module
### Measure 1
**Description:** Age of all patients included in the trial
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEAN
**Population Description:** FAS
**Title:** Age, Continuous
**Unit of Measure:** Years
### Measure 2
**Description:** Gender distribution of all patients included in the trial.
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** FAS
**Title:** Sex: Female, Male
**Unit of Measure:** Participants
### Measure 3
**Description:** Ethnicity of all patients included in the trial.
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** FAS
**Title:** Ethnicity (NIH/OMB)
**Unit of Measure:** Participants
### Measure 4
**Description:** Race of all patients included in the trial.
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** FAS
**Title:** Race (NIH/OMB)
**Unit of Measure:** Participants
**Population Description:** Full analysis set (FAS): All registered patients with informed consent (as applicable with local regulations) and at least one administration of Gi(l)otrif®.
## Results Section - More Information Module
### Certain Agreement
**Other Details:** Boehringer Ingelheim (BI) acknowledges that investigators have the right to publish the study results. Investigators shall provide BI with a copy of any publication or presentation for review prior to any submission. Such review will be done with regard to proprietary information, information related to patentable inventions, medical, scientific, and statistical accuracy within 60 days. BI may request a delay of the publication in order to protect BI's intellectual property rights.
**Restriction Type:** OTHER
**Restrictive Agreement:** True
### Limitations and Caveats
**Description:** Due to non-interventional character there were no predefined examinations, retro- and prospective data could be collected, bias regarding AE documentation, participation of patients on treatment was limited due to one exclusion criterion
### Point of Contact
**Email:** [email protected]
**Organization:** Boehringer Ingelheim
**Phone:** 1-800-243-0127
**Title:** Boehringer Ingelheim, Call Center
## Results Section - Outcome Measures Module
### Outcome Measure 1
### Outcome Measure 2
### Outcome Measure 3
### Outcome Measure 4
### Outcome Measure 5
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 21.02
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 21.13
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 44.59
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 57.75
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 24.84
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 16.90
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 3.18
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0.00
**Title:**
#### Outcome Measure 2
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:** 14.61
- **Spread:**
- **Upper Limit:** 21.51
- **Value:** 18.22
- **Comment:** The upper limit of the Confidence Interval has not being reached.
- **Group ID:** OG001
- **Lower Limit:** 12.93
- **Spread:**
- **Upper Limit:** NA
- **Value:** 19.41
**Title:**
#### Outcome Measure 3
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:** 18.22
- **Spread:**
- **Upper Limit:** 23.98
- **Value:** 20.03
- **Comment:** The upper limit of the Confidence Interval has not being reached.
- **Group ID:** OG001
- **Lower Limit:** 17.3
- **Spread:**
- **Upper Limit:** NA
- **Value:** 25.92
**Title:**
#### Outcome Measure 4
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 2.74
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 94.52
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 2.74
**Title:**
#### Outcome Measure 5
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0.00
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 38.03
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0.00
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 5.63
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 100.00
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 15.49
**Title:**
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 0.00
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 40.85
**Title:**
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Description:** An adverse drug reaction (ADR) is defined as a response to a medicinal product which is noxious and unintended. Grade 1, Grade 2, Grade 3 and Grade 4 ADR severity classes were considered for assessment of this outcome.
**Parameter Type:** NUMBER
**Population Description:** Full Analysis set (FAS): All registered patients with informed consent (as applicable with local regulations) and at least one administration of Gi(l)otrif®.
**Reporting Status:** POSTED
**Time Frame:** From signing the informed consent onwards until the end of the study, up to 104 weeks.
**Title:** Percentage of Patients With Adverse Drug Reactions (ADR) by Severity Class.
**Type:** PRIMARY
**Unit of Measure:** Percentage of patients (%)
##### Group
**Description:** Patients were orally treated with starting dose of greater than or equal to 40 mg Gi(l)otrif tablet once daily.
**ID:** OG000
**Title:** Gi(l)Otrif ≥ 40 mg
##### Group
**Description:** Patients were orally treated with starting dose of less than or equal to 30 mg Gi(l)otrif tablet once daily.
**ID:** OG001
**Title:** Gi(l)Otrif ≤ 30 mg
#### Outcome Measure 2
**Description:** Time on treatment with Gi(l)otrif® in real-world setting has been calculated in this assessment. Time on treatment refers to time to treatment failure with Gi(l)otrif®
**Dispersion Type:** 95% Confidence Interval
**Parameter Type:** MEDIAN
**Population Description:** FAS
**Reporting Status:** POSTED
**Time Frame:** From first dose of Gi(l)otrif® treatment to last dose of Gi(l)otrif® treatment, up to 104 weeks.
**Title:** Time on Treatment With Gi(l)Otrif®
**Type:** PRIMARY
**Unit of Measure:** Months
##### Group
**Description:** Patients were orally treated with starting dose of greater than or equal to 40 mg Gi(l)otrif tablet once daily.
**ID:** OG000
**Title:** Gi(l)Otrif ≥ 40 mg
##### Group
**Description:** Patients were orally treated with starting dose of less than or equal to 30 mg Gi(l)otrif tablet once daily.
**ID:** OG001
**Title:** Gi(l)Otrif ≤ 30 mg
#### Outcome Measure 3
**Description:** Time to progression was calculated from the date of first dose of Gi(l)otrif® treatment to the earliest date of documented progression (clinical, radiographic or both clinical/radiographic progression) or tumour-related death, whatever occurred first.
**Dispersion Type:** 95% Confidence Interval
**Parameter Type:** MEDIAN
**Population Description:** FAS
**Reporting Status:** POSTED
**Time Frame:** From first dose of Gi(l)otrif® treatment to last dose of Gi(l)otrif® treatment, up to 104 weeks.
**Title:** Time to Progression With Gi(l)Otrif®
**Type:** PRIMARY
**Unit of Measure:** Months
##### Group
**Description:** Patients were orally treated with starting dose of greater than or equal to 40 mg Gi(l)otrif tablet once daily.
**ID:** OG000
**Title:** Gi(l)Otrif ≥ 40 mg
##### Group
**Description:** Patients were orally treated with starting dose of less than or equal to 30 mg Gi(l)otrif tablet once daily.
**ID:** OG001
**Title:** Gi(l)Otrif ≤ 30 mg
#### Outcome Measure 4
**Description:** Percentage of patients with a modified starting dose that is dose other than the recommended 40 mg daily of Gi(l)otrif® has been calculated to assess this outcome measure.
**Parameter Type:** NUMBER
**Population Description:** FAS
**Reporting Status:** POSTED
**Time Frame:** From first dose of Gi(l)otrif® treatment to last dose of Gi(l)otrif® treatment, up to 104 weeks.
**Title:** Percentage of Patients With a Modified Starting Dose of Gi(l)Otrif®
**Type:** SECONDARY
**Unit of Measure:** Percentage of patients (%)
##### Group
**Description:** All patients who were treated with starting dose other than recommended dose of 40 mg Gi(l)otrif treatment once daily.
**ID:** OG000
**Title:** Modified Starting Dose of Gi(l)Otrif
#### Outcome Measure 5
**Description:** Different reasons for starting dose with modified dose that is dose other than recommended 40 mg once daily.
**Parameter Type:** NUMBER
**Population Description:** FAS
**Reporting Status:** POSTED
**Time Frame:** From first dose of Gi(l)otrif® treatment to last dose of Gi(l)otrif® treatment, up to 104 weeks.
**Title:** Percentage of Patients With Reasons for Modified Starting Dose of Gi(l)Otrif®
**Type:** SECONDARY
**Unit of Measure:** Percentage of patients (%)
##### Group
**Description:** All patients who were treated with modified dose of 50 mg Gi(l)otrif treatment once daily.
**ID:** OG000
**Title:** Modified Dose of 50 mg Gi(l)Otrif
##### Group
**Description:** All patients who were treated with modified starting dose of less than of equal to 30 mg Gi(l)otrif treatment once daily.
**ID:** OG001
**Title:** Modified Dose of ≤ 30 mg Gi(l)Otrif
### Participant Flow Module
#### Group
**Description:** Patients were orally treated with starting dose of greater than or equal to 40 mg Gi(l)otrif tablet once daily.
**ID:** FG000
**Title:** Gi(l)Otrif ≥ 40 mg
#### Group
**Description:** Patients were orally treated with starting dose of less than or equal to 30 mg Gi(l)otrif tablet once daily.
**ID:** FG001
**Title:** Gi(l)Otrif ≤ 30 mg
#### Period
**Title:** Overall Study
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 157
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 71
##### Milestone
**Type:** On Treatment at Study Completion
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 81
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 41
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 157
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 71
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 0
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 0
**Pre-Assignment Details:** All patients were screened for eligibility to participate in the study. Patients attended specialist sites which would then ensure that all patients met all inclusion/exclusion criteria. Patients were not to be entered to study if any of the specific entry criteria were violated.
**Recruitment Details:** This is a non-interventional, multi-country, multi-site study based on existing data from medical records of patients treated with Gi(l)otrif® tablet once daily as indicated in the approved labels. Between December 2016 and October 2017, 231 patients were screened for study participation and 228 patients were treated.
**Has Results:** True |
## Protocol Section
### Identification Module
**NCT ID:** NCT01752179
**Acronym:** KT
**Brief Title:** Kinesio Taping Technique and Trigger Point
**Official Title:** The Effect of Kinesio Taping Technique on Trigger Point of Piriformis Muscle
#### Organization Study ID Info
**ID:** 90/801/T/1/5055
#### Organization
**Class:** OTHER
**Full Name:** University of Social Welfare and Rehabilitation Science
### Status Module
#### Completion Date
**Date:** 2013-02
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2013-03-01
**Type:** ESTIMATED
**Last Update Submit Date:** 2013-02-28
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2013-02
**Type:** ACTUAL
#### Start Date
**Date:** 2011-05
**Status Verified Date:** 2013-02
#### Study First Post Date
**Date:** 2012-12-19
**Type:** ESTIMATED
**Study First Submit Date:** 2012-11-17
**Study First Submit QC Date:** 2012-12-15
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** University of Social Welfare and Rehabilitation Science
#### Responsible Party
**Investigator Affiliation:** University of Social Welfare and Rehabilitation Science
**Investigator Full Name:** Fahimeh Hashemirad
**Investigator Title:** MSc Physical therapist
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** Kinesio taping is a novel method which recently has emerged as a viable option to treat of various musculoskeletal and neuromuscular deficits. The application of tape to injured soft tissues and joints provides support and protection for these structures. Many different techniques are used for injury prevention, treatment, rehabilitation, proprioception and sport. Elastic adhesive tape may be used to unload Myofacial Trigger Points (MTrPs), A trigger point can be located in fascia, ligaments, muscles, and tendons; however, MTrPs are also found in skeletal muscles and/or their fascia. An MTrP is a hyperirritable spot, associated with a taut band of a skeletal muscle that is painful on compression or stretch, and that can give rise to a typical referred pain pattern as well as autonomic phenomena. The use of tape along muscle to unload affected soft tissue seems to be effective in the treatment of trigger points by inhibiting overactive muscle, changing the orientation of fascia and a proprioceptive effect. The purpose of study was to determine the efficacy of Kinesio taping application on trigger point of piriformis muscle.
**Detailed Description:** Piriformis syndrome is a peripheral neuritis of the sciatic nerve caused by an abnormal condition of the piriformis muscle. Some investigators consider it as a form of Myofacial pain syndrome which defined as the presence of exquisite tenderness at a nodule in a palpable taut band of muscle. Trigger points are able to produce referred pain, either spontaneously or on digital compression. Although myofascial trigger points are a widely recognized phenomenon in clinical practice, there remains much to be elucidated with regards to their pathophysiology. Conservative pharmacotherapy with nonsteroidal anti-inflammatory drugs (NSAID), muscle relaxants, and physical therapy modalities such as heat therapy, cold therapy, ultrasound, electrical current and stretching were traditionally used in the treatment of trigger points.
The utilization of Kinesio taping regarding to the proposed mechanisms including 1) restoring correct muscle function by supporting weakened muscles, (2) reducing congestion by improving the flow of blood and lymphatic fluid, (3) decreasing pain by stimulating neurological system, and (4) correcting misaligned joints by retrieving muscle spasm (5) enhancing proprioception through increased stimulation to cutaneous mechanoreceptors can be helpful in restoring muscle function in patients with Myofacial trigger points . However, there are not many controlled studies that have analyzed the effects of the Kinesio taping in their treatment. Therefore, the purpose of study was to determine the efficacy of KT application as an easy and appropriate method on trigger point of piriformis muscle.
### Conditions Module
**Conditions:**
- Piriformis Syndrome
**Keywords:**
- Kinesio taping
- Piriformis syndrome
- Trigger point
### Design Module
#### Design Info
**Allocation:** NON_RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** SINGLE
**Who Masked:**
- OUTCOMES_ASSESSOR
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 50
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** kinesio Tape : Width 5cm ,Length 35cm Y shape
**Intervention Names:**
- Procedure: kinesio Tape : Width 5cm ,Length 35cm Y shape
**Label:** kinesio tape
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** without using Kinesio tape
**Label:** Control group
**Type:** NO_INTERVENTION
### Interventions
#### Intervention 1
**Arm Group Labels:**
- kinesio tape
**Description:** In the experimental group, Kinesio taping application of piriformis according to Kenzo Kase in 2003 is modified by using unloading technique (Macdolanld, 2004). Size and Shape of Tape is Width 5cm ,Length 35cm Y shape.
Taping method include :
1. stretches the piriformis muscle in side lying position ,the affected leg is upper most with hip in flexion, adduction and internal rotation.
2. puts the base of tape over the contralateral of sacrum with no tension.
3. attaches the superior tail on the buttock over the upper part of piriformis and ends at the greater trochanter of the femur.
4. attaches the lower tail by lifting up the soft tissue and ends at the greater trochanter of the femur.
This is an origin to insertion application.
**Name:** kinesio Tape : Width 5cm ,Length 35cm Y shape
**Type:** PROCEDURE
### Outcomes Module
#### Primary Outcomes
**Description:** Change in pain intensity measured based on visual analogue scale at baseline, immediately after kinesio taping application and three day follow up . To measure the pain intensity in patients, a 100-mm visual analogue scale (VAS) is used. Score 0 corresponds to "no pain at all" and score 100 to "the worst imaginable pain"
**Measure:** Change from baseline in pain intensity immediately after kinesio taping application and Change from baseline in pain intensity at three day follow-up.
**Time Frame:** Three days
#### Secondary Outcomes
**Description:** Hip internal rotation is measured in the prone situation at baseline, immediately after kinesio taping application and three day follow up. Patients was asked to bent knee to 90, the axis of goniometer was placed at center of knee joint and the arms of the goniometer were aligned parallel to long axis of tibia then patient moved her leg outwardly as far as she could without allowing the pelvis movement, the stationary arm was hold in the start point while moving arm was aligned to long axis of tibia at the end range of internal rotation, this range was recorded as internal rotation of hip joint.
**Measure:** Change from baseline in hip internal rotation immediately after kinesio taping application and Change from baseline in hip internal rotation at three day follow-up
**Time Frame:** Three days
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Having trigger point and tenderness in piriformis muscle.
* Having at least three positive physical examination tests from FAIR, Freiberg Lasegue and Beaty test.
Exclusion Criteria:
* spinal surgery
* spinal or pelvic fracture
* disc herniation,
* facet arthropathy
* sacroiliitis
* osteoarthritis or fracture of the lower extremities
* systemic disease, such as arthritis or tuberculosis
**Maximum Age:** 70 Years
**Minimum Age:** 20 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Tehran
**Country:** Iran, Islamic Republic of
**Facility:** Akhavan Spine Physical Therapy Center, University of Social Welfare and Rehabilitation Sciences
**Zip:** 1113813111
#### Overall Officials
**Official 1:**
**Affiliation:** Akhavan Spine Physical Therapy Center, University of Social Welfare and Rehabilitation Sciences, Tehran, Iran.
**Name:** Fahimeh Hashemirad, MSc
**Role:** PRINCIPAL_INVESTIGATOR
**Official 2:**
**Affiliation:** University of Social Welfare and Rehabilitation Sciences, Evin, Tehran, Iran.
**Name:** Fahimeh Hashemirad, MSc
**Role:** STUDY_DIRECTOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000020426
- Term: Sciatic Neuropathy
- ID: D000020422
- Term: Mononeuropathies
- ID: D000010523
- Term: Peripheral Nervous System Diseases
- ID: D000009468
- Term: Neuromuscular Diseases
- ID: D000009422
- Term: Nervous System Diseases
- ID: D000009408
- Term: Nerve Compression Syndromes
- ID: D000009437
- Term: Neuralgia
- ID: D000010146
- Term: Pain
- ID: D000009461
- Term: Neurologic Manifestations
- ID: D000017699
- Term: Pelvic Pain
### Condition Browse Module - Browse Branches
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: All
- Name: All Conditions
- Abbrev: BC10
- Name: Nervous System Diseases
- Abbrev: BC16
- Name: Diseases and Abnormalities at or Before Birth
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M16355
- Name: Syndrome
- Relevance: LOW
- As Found: Unknown
- ID: M28404
- Name: Piriformis Muscle Syndrome
- Relevance: HIGH
- As Found: Piriformis Syndrome
- ID: M22222
- Name: Sciatic Neuropathy
- Relevance: LOW
- As Found: Unknown
- ID: M22218
- Name: Mononeuropathies
- Relevance: LOW
- As Found: Unknown
- ID: M13432
- Name: Peripheral Nervous System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M12411
- Name: Neuromuscular Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M12352
- Name: Nerve Compression Syndromes
- Relevance: LOW
- As Found: Unknown
- ID: M5853
- Name: Charcot-Marie-Tooth Disease
- Relevance: LOW
- As Found: Unknown
- ID: M18092
- Name: Hereditary Sensory and Motor Neuropathy
- Relevance: LOW
- As Found: Unknown
- ID: M12381
- Name: Neuralgia
- Relevance: LOW
- As Found: Unknown
- ID: M13066
- Name: Pain
- Relevance: LOW
- As Found: Unknown
- ID: M12404
- Name: Neurologic Manifestations
- Relevance: LOW
- As Found: Unknown
- ID: M19918
- Name: Pelvic Pain
- Relevance: LOW
- As Found: Unknown
- ID: T4568
- Name: Piriformis Syndrome
- Relevance: HIGH
- As Found: Piriformis Syndrome
- ID: T1081
- Name: Charcot-Marie-Tooth Disease
- Relevance: LOW
- As Found: Unknown
- ID: T2761
- Name: Hereditary Motor and Sensory Neuropathy
- Relevance: LOW
- As Found: Unknown
- ID: T2766
- Name: Hereditary Neuropathy With Liability to Pressure Palsies
- Relevance: LOW
- As Found: Unknown
- ID: T5067
- Name: Roussy Levy Syndrome
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000055958
- Term: Piriformis Muscle Syndrome
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT03767179
**Brief Title:** Research of Serum Procalcitonin, ESR, CRP And Leukocyte Levels in Fertile Missed Abortion Cases
**Official Title:** Research of Serum Procalcitonin, Erythrocyte Sedimentation Rate, C-Reactive Protein And Leukocyte Levels in Fertile Missed Abortion Cases
#### Organization Study ID Info
**ID:** KTU
#### Organization
**Class:** OTHER
**Full Name:** Karadeniz Technical University
### Status Module
#### Completion Date
**Date:** 2018-05-15
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2020-06-26
**Type:** ACTUAL
**Last Update Submit Date:** 2020-06-25
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2018-04-15
**Type:** ACTUAL
#### Start Date
**Date:** 2018-01-01
**Type:** ACTUAL
**Status Verified Date:** 2020-06
#### Study First Post Date
**Date:** 2018-12-06
**Type:** ACTUAL
**Study First Submit Date:** 2018-06-17
**Study First Submit QC Date:** 2018-12-04
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Karadeniz Technical University
#### Responsible Party
**Investigator Affiliation:** Karadeniz Technical University
**Investigator Full Name:** Suleyman Guven
**Investigator Title:** Clinical Professor.
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** Aim: The serum procalcitonin, erythrocyte sedimentation rate, c-reactive protein and leukocyte values will be compared between fertile missed abortion diagnosed pregnants and fertile normal pregnants to determine the presence of subclinical infection in the etiology of missed abortion
**Detailed Description:** The study group will consist of 30 fertile, under 35 years old women having for the first time missed abortus diagnosis in the Obstetrics Department of Obstetrics and Gynecology of Medicine Faculty of Karadeniz Technical University, Medical Faculty of Medicine. As control group, 30 cases with the same trimester, fertile, and under 35 years old who had referred to Obstetrics clinic, who had no systemic disease, will be included in the study. Blood samples will be collected at the time of diagnosis of the patients with missed abortion and in normal pregnancy in the week of 20th gestational week and will send to Biochemistry laboratory to examine procalcitonin, erythrocyte sedimentation rate, c-reactive protein and leukocyte values in their sera. The age, weight, gestational week, gravida (number), parity (number), abortus (number), body mass index, systemic diseases, blood group, smoking information, binary test results, and results of genetic analysis will be collected in both groups. The pathology results of the missed abortion group will also be collected. The results will statistically be compared in the SPSS 13.0 program in both groups.
### Conditions Module
**Conditions:**
- Abortion, Missed
**Keywords:**
- Missed abortion
- Procalcitonin
- Erythrocyte sedimentation rate (ESR)
- C-reactive protein (CRP)
- Leukocyte (WBC)
### Design Module
#### Bio Spec
**Description:** Blood Serum, abortion materials
**Retention:** SAMPLES_WITHOUT_DNA
#### Design Info
**Observational Model:** CASE_CONTROL
**Time Perspective:** PROSPECTIVE
#### Enrollment Info
**Count:** 60
**Type:** ACTUAL
**Study Type:** OBSERVATIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** The study group consisted of 30 fertile, under 35 years old women who were included in the study for the first time missed abortus diagnosis.
**Intervention Names:**
- Diagnostic Test: study
**Label:** Study Group
#### Arm Group 2
**Description:** 30 cases with the same trimester, fertile, and under 35 years old who were referred to Obstetrics clinic, who had no systemic disease, were included in the study.
**Intervention Names:**
- Diagnostic Test: study
**Label:** Control Group
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Control Group
- Study Group
**Description:** procalcitonin, sedimentation, CRP, leukocyte identification
**Name:** study
**Type:** DIAGNOSTIC_TEST
### Outcomes Module
#### Primary Outcomes
**Description:** Serum leukocyte count in number
**Measure:** Serum LC
**Time Frame:** 01.01.2018- 15.02.2019
**Description:** Serum CRP level in microgram
**Measure:** CRP
**Time Frame:** 01.01.2018- 15.02.2019
**Description:** Erythrocyte sedimentation rate
**Measure:** ESR
**Time Frame:** 01.01.2018- 15.02.2019
**Description:** Serum procalcitonin level in microgram
**Measure:** Prokalsitonin
**Time Frame:** 01.01.2018- 15.02.2019
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* fertile, less than 20 weeks of geese who have missed abortus for the first time without systemic disease
Exclusion Criteria:
* Failure to meet the missed abortus diagnostic criteria (for the group of fertile missed abortus)
* Smoking
* Rejection to participate in the study.
**Healthy Volunteers:** True
**Maximum Age:** 35 Years
**Minimum Age:** 18 Years
**Sampling Method:** PROBABILITY_SAMPLE
**Sex:** FEMALE
**Standard Ages:**
- ADULT
**Study Population:** 60 pregnant women
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Trabzon
**Country:** Turkey
**Facility:** Karadeniz Technical University, Medical Faculty of Medicine.
**State:** Ortahisar
**Zip:** 61000
#### Overall Officials
**Official 1:**
**Affiliation:** KaradenizTU Medicine Faculty
**Name:** Suleyman Guven, Prof
**Role:** PRINCIPAL_INVESTIGATOR
### IPD Sharing Statement Module
**IPD Sharing:** NO
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000000022
- Term: Abortion, Spontaneous
- ID: D000011248
- Term: Pregnancy Complications
- ID: D000005261
- Term: Female Urogenital Diseases and Pregnancy Complications
- ID: D000091642
- Term: Urogenital Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BXS
- Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions
- Abbrev: All
- Name: All Conditions
### Condition Browse Module - Browse Leaves
- ID: M29
- Name: Abortion, Missed
- Relevance: HIGH
- As Found: Abortion, Missed
- ID: M21
- Name: Abortion, Spontaneous
- Relevance: LOW
- As Found: Unknown
- ID: M14127
- Name: Pregnancy Complications
- Relevance: LOW
- As Found: Unknown
- ID: M2875
- Name: Urogenital Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M27093
- Name: Female Urogenital Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M8399
- Name: Female Urogenital Diseases and Pregnancy Complications
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000000030
- Term: Abortion, Missed
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT04293679
**Brief Title:** Open Label, Dose Escalation Study for the Safety and Efficacy of STP705 in Adult Patients With isSCC
**Official Title:** An Open Label, Dose Escalation Study to Evaluate the Safety and Efficacy of Intralesional Injection of STP705 in Adult Patients With Cutaneous Squamous Cell Carcinoma in Situ (isSCC)
#### Organization Study ID Info
**ID:** SRN-705-004
#### Organization
**Class:** INDUSTRY
**Full Name:** Sirnaomics
### Status Module
#### Completion Date
**Date:** 2020-10-21
**Type:** ACTUAL
#### Last Update Post Date
**Date:** 2022-06-09
**Type:** ACTUAL
**Last Update Submit Date:** 2022-06-08
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2020-10-21
**Type:** ACTUAL
#### Start Date
**Date:** 2019-03-21
**Type:** ACTUAL
**Status Verified Date:** 2020-12
#### Study First Post Date
**Date:** 2020-03-03
**Type:** ACTUAL
**Study First Submit Date:** 2020-02-26
**Study First Submit QC Date:** 2020-02-28
### Sponsor Collaborators Module
#### Collaborators
**Class:** OTHER
**Name:** Amarex Clinical Research
#### Lead Sponsor
**Class:** INDUSTRY
**Name:** Sirnaomics
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** True
### Description Module
**Brief Summary:** This is an open label, dose escalation study to evaluate the safety and efficacy of intralesional injection of STP705 in adult patients with Cutaneous Squamous Cell Carcinoma in situ (isSCC, Bowen's disease). The purpose of this trial is to evaluate the safety, tolerability and efficacy of various doses of STP705 administered as Intralesional injection in subjects with isSCC.
Goals:
* To determine the safe and effective recommended dose of STP705 for the treatment of isSCC.
* Analysis of biomarkers common to isSCC formation pathway including TGF-β1 and COX-2.
**Detailed Description:** This open label, dose escalation study is designed to evaluate the safety and efficacy of various doses of STP705 administered as an intralesional injection in subjects with cutaneous in situ squamous cell carcinoma (isSCC).
The primary objective of this study is to evaluate the safety, tolerability, and efficacy of various doses of STP705 administered as an Intralesional injection in subjects with cutaneous squamous cell carcinoma in situ skin cancer (isSCC). This study seeks to establish a safe and effective recommended dose of STP705 for the treatment of isSCC. Expression of biomarkers common to the isSCC formation pathway, including TGF-β1 and COX-2, will be evaluated.
The primary endpoint will be the proportion of participants with histological clearance of treated isSCC lesion at the End of Treatment (EOT). Histological clearance (HC) will be defined as the absence of detectable evidence of isSCC tumor cell nests as determined by central pathology review.
Secondary endpoints will include i) time to histological clearance of treated isSCC lesion over the 6 week treatment period, ii) proportion of participants with complete clinical clearance of treated isSCC lesion based on investigator assessment at the End of Treatment (EOT), iii) time to complete clinical clearance of treated isSCC lesion based on investigator assessment over the 6 week treatment period, and iv) the change in size of the treated isSCC lesion over the 6 week treatment period.
Safety and tolerability will be assessed by the number of incidence of adverse events (AEs) and serious adverse events (SAEs); the incidence of AEs and SAEs leading to discontinuation of trial medication; the incidence and severity of Local Skin Response (LSR); hypopigmentation and hyperpigmentation following treatment; and the tolerability of repeated Intralesional administration of STP705 as assessed by investigator-evaluation of injection site reactions for all patients and within each cohort.
In addition, safety measures will include clinically relevant changes or new abnormal findings in laboratory values, vital signs, electrocardiograms (ECGs), and physical examination variables.
25 adult patients are planned to be enrolled in the study. They will be divided equally among 5 cohorts (10, 20, 30, 60 and 120 μg dose levels) of 5 subjects each. Enrollment of the first two subjects in each dosing cohort will be staggered by at least 48 hours. Participants in the first cohort will attend the study center once weekly for an injection of STP705 into the isSCC lesion. The participants will receive injections of STP705 once a week for up to 6 weeks. The clinician will evaluate the tumor for clinical changes and reduction in size at each treatment visit for up to 6 weeks. If during the 6 weeks of treatment there is complete clinical clearance of the tumor, the treatments will end. At the End of Treatment visit, the residual tumor, or former tumor location will be excised for analysis. In the absence of dose limiting toxicities (DLT), the subsequent cohorts will receive increasing doses of STP705, following the same schedule of administration as the first cohort.
If any of the SAEs or dose limiting toxicities outlined above has occurred, the Data Safety Monitoring Board (DSMB) will conduct independent review of the data and will make a final decision for dose escalation to the next cohort.
### Conditions Module
**Conditions:**
- Bowen's Disease
- Cutaneous Squamous Cell Carcinoma in Situ
**Keywords:**
- isSCC
- Cutaneous Squamous Cell Carcinoma
- STP705
- Cutaneous Squamous Cell Carcinoma in situ
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** SEQUENTIAL
**Intervention Model Description:** Participants in the first cohort will attend the study center once weekly for an injection of STP705 into the isSCC lesion. The participants will receive injections of STP705 once a week for up to 6 weeks. The clinician will evaluate the tumor for clinical changes and reduction in size at each treatment visit for up to 6 weeks. If during the 6 weeks of treatment there is Complete Clinical Clearance of the tumor, the treatments will end. At the End of Treatment visit, the residual tumor, or former tumor location, will be excised for analysis. In the absence of dose limiting toxicities (DLT), the subsequent cohorts will receive increasing doses of STP705, following the same schedule of administration as the first cohort.
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 25
**Type:** ACTUAL
**Phases:**
- PHASE1
- PHASE2
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Cohort A: STP705 10 μg dose, intradermal injection, given once a week for up to 6 weeks
**Intervention Names:**
- Drug: STP705
**Label:** Cohort A: STP705 10 μg dose
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Cohort B: STP705 20 μg dose, intradermal injection, given once a week for up to 6 weeks
**Intervention Names:**
- Drug: STP705
**Label:** Cohort B: STP705 20 μg dose
**Type:** EXPERIMENTAL
#### Arm Group 3
**Description:** Cohort C: STP705 30 μg dose, intradermal injection, given once a week for up to 6 weeks
**Intervention Names:**
- Drug: STP705
**Label:** Cohort C: STP705 30 μg dose
**Type:** EXPERIMENTAL
#### Arm Group 4
**Description:** Cohort D: STP705 60 μg dose, intradermal injection, given once a week for up to 6 weeks
**Intervention Names:**
- Drug: STP705
**Label:** Cohort D: STP705 60 μg dose
**Type:** EXPERIMENTAL
#### Arm Group 5
**Description:** Cohort E: STP705 120 μg dose, intradermal injection, given once a week for up to 6 weeks
**Intervention Names:**
- Drug: STP705
**Label:** Cohort E: STP705 120 μg dose
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Cohort A: STP705 10 μg dose
- Cohort B: STP705 20 μg dose
- Cohort C: STP705 30 μg dose
- Cohort D: STP705 60 μg dose
- Cohort E: STP705 120 μg dose
**Description:** Investigational Product
**Name:** STP705
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Description:** Proportion of participants with histological clearance of treated isSCC lesion at the End of Treatment (EOT).
Histological clearance (HC) will be defined as the absence of detectable evidence of isSCC tumor cell nests as determined by central pathology review.
**Measure:** Proportion of participants with histological clearance of treated isSCC lesion at the End of Treatment (EOT).
**Time Frame:** 6 weeks
#### Secondary Outcomes
**Measure:** Time to histological clearance of treated isSCC lesion over the 6 week treatment period.
**Time Frame:** over the 6 week treatment period
**Measure:** Proportion of participants with complete clinical clearance of treated isSCC lesion based on investigator assessment at the End of Treatment (EOT).
**Time Frame:** 6 weeks
**Measure:** Time to complete clinical clearance of treated isSCC lesion based on investigator assessment over the 6 week treatment period.
**Time Frame:** over the 6 week treatment period
**Description:** Change in size of the treated isSCC lesion over the 6 week treatment period. A base line assessment of lesion size will be made by investigator at T1 (first visit, Day 0). The change in size will be assessed every week until the surgical excision of isSCC at the End of Treatment visit (EOT, Day 42) .
**Measure:** Change in size of the treated isSCC lesion over the 6 week treatment period.
**Time Frame:** over the 6 week treatment period
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* 1. Male or female adult ≥ 18 years of age.
* 2. Primary, histologically confirmed trunk or extremity (non-peri-orbital/-anogenital/-facial/-scalp) isSCC lesion suitable for excision with a minimum diameter of 0.5cm and with a maximum diameter of 2.0cm.
* 3. Histological diagnosis made no more than 6 months prior to the screening visit.
* 4. Histological biopsy removed ≤25% of the original area of the target lesion.
* 5. No other dermatological disease in the isSCC target site or surrounding area, which in the opinion of the investigator, could interfere with the study.
* 6. Willing to refrain from using non-approved lotions or creams on the target site and surrounding area during the treatment period.
* 7. Willing to refrain from exposure to excessive direct sunlight or ultraviolet light and to avoid the use of tanning parlors for the duration of the study.
* 8. Laboratory values for the tests (listed in the Study Schedule) within the reference ranges as defined by the central laboratory, or "out of range" test results that is clinically acceptable to the investigator. Acceptable "out of range" values are generally those within 2 standard deviations of the mean or explainable due to concurrent medications or disease processes.
* 9. Ability to follow study instructions and likely to complete all study requirements.
* 10. Written informed consent obtained, including consent for tissue to be examined and stored by the Central Histology Lab.
* 11. Written consent to allow photographs of the target isSCC lesion to be used as part of the study data and documentation.
* 12. For females of childbearing potential, a negative pregnancy test at screening and using an acceptable form of birth control (oral / implant/ injectable/ transdermal contraceptives, intrauterine device, condom, diaphragm, abstinence, or a monogamous relationship with a partner who has had a vasectomy).
Exclusion Criteria:
* 1. Pregnant or lactating.
* 2. Presence of known or suspected systemic cancer.
* 3. Histological evidence of nBCC, sBCC, invasive SCC, or any other non-isSCC tumor in the biopsy specimen.
* 4. Histological evidence of severe squamous metaplasia, infiltrative, desomoplastic or micronodular growth patterns in the biopsy specimen.
* 5. History of recurrence of the target isSCC lesion.
* 6. Prior exposure to STP705.
* 7. Evidence of dermatological disease or confounding skin condition in the treatment area, e.g., BCC, actinic keratosis, rosacea, psoriasis, atopic dermatitis, eczema, xeroderma pigmentosa.
* 8. Concurrent disease or treatment that suppresses the immune system;
* 9. Patients with baseline QTC \> 480 msec using Frederica's formula
* 10. Chronic medical condition that in the judgment of the investigator(s) would interfere with the performance of the study or would place the patient at undue risk.
* 11. Known sensitivity to any of the ingredients in the study medication.
* 12. Use of a tanning beds or other excessive or prolonged exposure to ultraviolet light or direct sunlight during the study.
* 13. Treatment with systemic chemotherapeutic agents within the 6 months prior to the screening visit.
* 14. Use of systemic retinoids within the 6 months prior to the screening period.
* 15. Treatment with systemic immunomodulators or immunosuppressants within the 6 months prior to the screening period.
* 16. Use of topical immunomodulators within 2cm of the target isSCC lesion within the 4 weeks prior to the screening period.
* 17. Treatment with the following topical agents within 2cm of the target isSCC lesion within the 4 weeks prior to the screening visit: amino-levulanic acid, 5-fluorouracil, corticosteroids, retinoids, diclofenac, ingenol mebutate, or imiquimod.
* 18. Treatment with liquid nitrogen, surgical excision (excluding diagnostic incisional biopsy) or curettage within 2cm of the target isSCC lesion during the 4 weeks prior to the screening visit.
* 19. Elective surgery within 4 weeks prior to the screening visit, during the study, or 4 weeks after the study period.
* 20. Evidence of current chronic alcohol or drug abuse.
* 21. Current enrollment in an investigational drug or device study or participation in such a study within 4 weeks of the screening visit.
* 22. In the investigator's opinion, evidence of unwillingness, or inability to follow the restrictions and requirements of the protocol and complete the study.
* 23. Taking any investigational product within 1 month of first dose of STP705.
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Aventura
**Country:** United States
**Facility:** Center for Clinical and Cosmetic Research
**State:** Florida
**Zip:** 33180
#### Overall Officials
**Official 1:**
**Affiliation:** Amarex Clinical Research, LLC (Amarex)
**Name:** Kush Dhody, MBBS, MS, CCRA
**Role:** STUDY_DIRECTOR
**Official 2:**
**Affiliation:** Center for Clinical and Cosmetic Research
**Name:** Brian Berman, MD, PhD
**Role:** PRINCIPAL_INVESTIGATOR
### IPD Sharing Statement Module
**IPD Sharing:** UNDECIDED
### References Module
#### References
**Citation:** Kuzbicki L, Brozyna AA. Expression of Cyclooxygenase-2 in Human Epithelial Skin Lesions: A Systematic Review of Immunohistochemical Studies. Appl Immunohistochem Mol Morphol. 2021 Mar 1;29(3):163-174. doi: 10.1097/PAI.0000000000000871.
**PMID:** 32889812
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000009375
- Term: Neoplasms, Glandular and Epithelial
- ID: D000009370
- Term: Neoplasms by Histologic Type
- ID: D000009369
- Term: Neoplasms
- ID: D000018307
- Term: Neoplasms, Squamous Cell
### Condition Browse Module - Browse Branches
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: All
- Name: All Conditions
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M5534
- Name: Carcinoma
- Relevance: HIGH
- As Found: Carcinoma
- ID: M5550
- Name: Carcinoma, Squamous Cell
- Relevance: HIGH
- As Found: Squamous Cell Carcinoma
- ID: M5535
- Name: Carcinoma in Situ
- Relevance: HIGH
- As Found: Carcinoma in Situ
- ID: M5190
- Name: Bowen's Disease
- Relevance: HIGH
- As Found: Bowen's Disease
- ID: M12320
- Name: Neoplasms, Glandular and Epithelial
- Relevance: LOW
- As Found: Unknown
- ID: M12315
- Name: Neoplasms by Histologic Type
- Relevance: LOW
- As Found: Unknown
- ID: M20451
- Name: Neoplasms, Squamous Cell
- Relevance: LOW
- As Found: Unknown
- ID: T810
- Name: Bowen's Disease
- Relevance: HIGH
- As Found: Bowen's Disease
### Condition Browse Module - Meshes
- ID: D000002277
- Term: Carcinoma
- ID: D000002294
- Term: Carcinoma, Squamous Cell
- ID: D000002278
- Term: Carcinoma in Situ
- ID: D000001913
- Term: Bowen's Disease
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT02571179
**Acronym:** fentaobs
**Brief Title:** Intranasal Fentanyl in Treatment of Labour Pain
**Official Title:** Intranasal Fentanyl in Treatment of Labour Pain - Efficacy and Safety
#### Organization Study ID Info
**ID:** KUH12_04_2010
#### Organization
**Class:** OTHER
**Full Name:** Kuopio University Hospital
### Status Module
#### Completion Date
**Date:** 2021-03
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2023-03-16
**Type:** ACTUAL
**Last Update Submit Date:** 2023-03-14
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2021-03
**Type:** ACTUAL
#### Start Date
**Date:** 2011-01
**Status Verified Date:** 2023-03
#### Study First Post Date
**Date:** 2015-10-08
**Type:** ESTIMATED
**Study First Submit Date:** 2015-10-05
**Study First Submit QC Date:** 2015-10-07
### Sponsor Collaborators Module
#### Collaborators
**Class:** INDUSTRY
**Name:** Admescope Ltd
#### Lead Sponsor
**Class:** OTHER
**Name:** Kuopio University Hospital
#### Responsible Party
**Investigator Affiliation:** Kuopio University Hospital
**Investigator Full Name:** Merja Kokki
**Investigator Title:** MD, PhD
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** Physiological changes during pregnancy are known to affect the pharmacokinetics of many drugs. Intranasal fentanyl is an interesting option for obstetric analgesia, but its use in pregnant patients has not been established. The investigators studied pharmacokinetics of intranasal fentanyl in labouring women and to subsequently evaluate the maternal and fetal safety after administration.
### Conditions Module
**Conditions:**
- Labor Pain
**Keywords:**
- Labor pain
- Fentanyl
- Transmucosal
### Design Module
#### Design Info
**Allocation:** NA
**Intervention Model:** SINGLE_GROUP
**Intervention Model Description:** Intranasal fentanyl
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 20
**Type:** ACTUAL
**Phases:**
- PHASE4
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** patient was given intranasal fentanyl 50 microg/dose up to 250 microg
**Intervention Names:**
- Drug: intranasal fentanyl 50 microg dose up to 250 microg
**Label:** Intranasal fentanyl 50 microg/dose
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Intranasal fentanyl 50 microg/dose
**Description:** When contraction pain was ≥ 5/10 (numerical rating scale 0= no pain, 10= worst pain), the parturient was given a intranasal fentanyl 50 µg dose. After 15 minutes, if contraction pain was still ≥ 5/10, a second 50 µg intranasal dose was administered. Fentanyl was administered every 15 minute until contraction pain decreased to less than 5/10 or until the maximum fentanyl dose of 250 µg was administered.
**Name:** intranasal fentanyl 50 microg dose up to 250 microg
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Measure:** Fentanyl maximum concentration
**Time Frame:** From the first intranasal fentanyl dose to birth of the newborn up to 48 hours
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* healthy parturients with uncomplicated, single gestation pregnancies, full term (38-42 weeks of gestation) pregnancy, agreed to participate
Exclusion Criteria:
* a disease that might affect hepatic or renal function, contraindications to opioid analgesics, fetal growth retardation, signs of fetal asphyxia by cardiotocography, meconium stained amniotic fluid or placental insufficiency. The subjects should not have received fentanyl during the previous 14 days.
Not agreed to participate
**Maximum Age:** 45 Years
**Minimum Age:** 18 Years
**Sex:** FEMALE
**Standard Ages:**
- ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Kuopio
**Country:** Finland
**Facility:** Kuopio University Hospital
**State:** Northern Savo
**Zip:** 70029
#### Overall Officials
**Official 1:**
**Affiliation:** Kuopio University Hospital, Kuopio, Finland
**Name:** Merja Kokki, PhD
**Role:** PRINCIPAL_INVESTIGATOR
### IPD Sharing Statement Module
**IPD Sharing:** UNDECIDED
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000010146
- Term: Pain
- ID: D000009461
- Term: Neurologic Manifestations
### Condition Browse Module - Browse Branches
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: All
- Name: All Conditions
- Abbrev: BC10
- Name: Nervous System Diseases
### Condition Browse Module - Browse Leaves
- ID: M26008
- Name: Labor Pain
- Relevance: HIGH
- As Found: Labor Pain
- ID: M13066
- Name: Pain
- Relevance: LOW
- As Found: Unknown
- ID: M12404
- Name: Neurologic Manifestations
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000048949
- Term: Labor Pain
### Intervention Browse Module - Ancestors
- ID: D000000701
- Term: Analgesics, Opioid
- ID: D000009294
- Term: Narcotics
- ID: D000002492
- Term: Central Nervous System Depressants
- ID: D000045505
- Term: Physiological Effects of Drugs
- ID: D000000700
- Term: Analgesics
- ID: D000018689
- Term: Sensory System Agents
- ID: D000018373
- Term: Peripheral Nervous System Agents
- ID: D000000759
- Term: Adjuvants, Anesthesia
- ID: D000018686
- Term: Anesthetics, Intravenous
- ID: D000018681
- Term: Anesthetics, General
- ID: D000000777
- Term: Anesthetics
### Intervention Browse Module - Browse Branches
- Abbrev: AdjAn
- Name: Adjuvants, Anesthesia
- Abbrev: Analg
- Name: Analgesics
- Abbrev: CNSDep
- Name: Central Nervous System Depressants
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M8418
- Name: Fentanyl
- Relevance: HIGH
- As Found: Moderate
- ID: M4032
- Name: Analgesics
- Relevance: LOW
- As Found: Unknown
- ID: M4033
- Name: Analgesics, Opioid
- Relevance: LOW
- As Found: Unknown
- ID: M12245
- Name: Narcotics
- Relevance: LOW
- As Found: Unknown
- ID: M4107
- Name: Anesthetics
- Relevance: LOW
- As Found: Unknown
- ID: M4089
- Name: Adjuvants, Anesthesia
- Relevance: LOW
- As Found: Unknown
- ID: M20766
- Name: Anesthetics, Intravenous
- Relevance: LOW
- As Found: Unknown
- ID: M20761
- Name: Anesthetics, General
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: D000005283
- Term: Fentanyl
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT02032979
**Brief Title:** Neurological and Psychiatric Comorbidities Patients With FSHD 1 and 2
**Official Title:** Neurological and Psychiatric Comorbidities Patients With FSHD 1 and 2
#### Organization Study ID Info
**ID:** 13-AOI-06
#### Organization
**Class:** OTHER
**Full Name:** Centre Hospitalier Universitaire de Nice
### Status Module
#### Completion Date
**Date:** 2015-07-01
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2023-11-14
**Type:** ACTUAL
**Last Update Submit Date:** 2023-11-10
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2015-07-01
**Type:** ACTUAL
#### Start Date
**Date:** 2014-10-20
**Type:** ACTUAL
**Status Verified Date:** 2023-11
#### Study First Post Date
**Date:** 2014-01-10
**Type:** ESTIMATED
**Study First Submit Date:** 2013-12-10
**Study First Submit QC Date:** 2014-01-09
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Centre Hospitalier Universitaire de Nice
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** The investigators propose to conduct a comparative pilot cognitive and psychiatric profiles of 10 patients Facio-Scapulo-Humeral Dystrophy (= FHSD) type 1 and 10 patients with type 2 FSHD study. For this, the investigators relied on observational components: FSHD2 patients appear more often present with psychiatric comorbidities and seem to have lower cognitive performance compared to FSHD1 patients. This was confirmed by a preliminary study on a small sample population of patients. It seems to exist mainly executive dysfunction associated with attention disorders in patients FSHD2. Moreover, their performance in IQ tests would be low in relation to their socio-educational and compared with patients FSHD1 level.
### Conditions Module
**Conditions:**
- Muscular Dystrophy, Facioscapulohumeral
### Design Module
#### Design Info
**Allocation:** NA
**Intervention Model:** SINGLE_GROUP
##### Masking Info
**Masking:** NONE
**Primary Purpose:** BASIC_SCIENCE
#### Enrollment Info
**Count:** 6
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Intervention Names:**
- Behavioral: Psychiatric test
**Label:** FSHD patient
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- FSHD patient
**Name:** Psychiatric test
**Type:** BEHAVIORAL
### Outcomes Module
#### Primary Outcomes
**Measure:** Montgomery and Asberg Depression Rating Scale
**Time Frame:** One time at the inclusion
**Measure:** Hamilton Depression Rating Scale
**Time Frame:** One time at the inclusion
**Measure:** Hamilton Anxiety Rating Scale
**Time Frame:** One time at the inclusion
**Measure:** Quick inventory of depressive symptomatology Self report
**Time Frame:** One time at the inclusion
**Measure:** Quality of Life Enjoyment and Satisfaction Questionnaire - Short Form
**Time Frame:** One time at the inclusion
**Measure:** Mini Mental Status Evaluation
**Time Frame:** One time at the inclusion
**Measure:** Wechsler Adult Intelligence Scale
**Time Frame:** One time at the inclusion
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* age ≥ 18 years and \<75 years
* FSHD patients 1 or 2 with genetic confirmation
Exclusion Criteria:
* Phosphokinase creatine level \> 5 time of the normal
* Patient as medical history :
* A history or active neurological disease likely to interfere with the interpretation of results
* a history of head trauma
* an infectious disease, hormonal, inflammatory or some deficiency may induce cognitive and / or psychiatric troubles
* Patient with cons-indication for performing a brain MRI
* Pregnant, parturient and lactating (producing a serum pregnancy test)
**Maximum Age:** 75 Years
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Nice
**Country:** France
**Facility:** Hôpital Pasteur
**Zip:** 06002
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000020966
- Term: Muscular Disorders, Atrophic
- ID: D000009135
- Term: Muscular Diseases
- ID: D000009140
- Term: Musculoskeletal Diseases
- ID: D000009468
- Term: Neuromuscular Diseases
- ID: D000009422
- Term: Nervous System Diseases
- ID: D000030342
- Term: Genetic Diseases, Inborn
### Condition Browse Module - Browse Branches
- Abbrev: BC05
- Name: Musculoskeletal Diseases
- Abbrev: BC10
- Name: Nervous System Diseases
- Abbrev: BC16
- Name: Diseases and Abnormalities at or Before Birth
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M12093
- Name: Muscular Dystrophies
- Relevance: HIGH
- As Found: Muscular Dystrophy
- ID: M22188
- Name: Muscular Dystrophy, Facioscapulohumeral
- Relevance: HIGH
- As Found: Muscular Dystrophy, Facioscapulohumeral
- ID: M4589
- Name: Atrophy
- Relevance: LOW
- As Found: Unknown
- ID: M12092
- Name: Muscular Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M22697
- Name: Muscular Disorders, Atrophic
- Relevance: LOW
- As Found: Unknown
- ID: M12097
- Name: Musculoskeletal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M12411
- Name: Neuromuscular Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M23686
- Name: Genetic Diseases, Inborn
- Relevance: LOW
- As Found: Unknown
- ID: T3963
- Name: Muscular Dystrophy
- Relevance: HIGH
- As Found: Muscular Dystrophy
- ID: T2182
- Name: Facioscapulohumeral Muscular Dystrophy
- Relevance: HIGH
- As Found: Muscular Dystrophy, Facioscapulohumeral
### Condition Browse Module - Meshes
- ID: D000009136
- Term: Muscular Dystrophies
- ID: D000020391
- Term: Muscular Dystrophy, Facioscapulohumeral
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT01805479
**Brief Title:** Exercise Training in Depressed Traumatic Brain Injury Survivors
**Official Title:** Exercise Training in Depressed Traumatic Brain Injury Survivors
#### Organization Study ID Info
**ID:** PT107574
#### Organization
**Class:** OTHER
**Full Name:** Virginia Commonwealth University
#### Secondary ID Infos
**ID:** 7K23HD067553
**Link:** https://reporter.nih.gov/quickSearch/7K23HD067553
**Type:** NIH
### Status Module
#### Completion Date
**Date:** 2014-06
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2015-12-21
**Type:** ESTIMATED
**Last Update Submit Date:** 2015-12-18
**Overall Status:** WITHDRAWN
#### Primary Completion Date
**Date:** 2014-06
**Type:** ACTUAL
#### Start Date
**Date:** 2013-02
**Status Verified Date:** 2015-12
#### Study First Post Date
**Date:** 2013-03-06
**Type:** ESTIMATED
**Study First Submit Date:** 2013-03-01
**Study First Submit QC Date:** 2013-03-04
**Why Stopped:** Unable to enroll participants. Sponsor requested study closure.
### Sponsor Collaborators Module
#### Collaborators
**Class:** NIH
**Name:** Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
#### Lead Sponsor
**Class:** OTHER
**Name:** Virginia Commonwealth University
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** The purpose of this study is to determine the feasibility of successfully delivering moderate intensity aerobic exercise training to depressed traumatic brain injury subjects between one and three years after injury.
**Detailed Description:** Traumatic brain injury (TBI) alters the lives of many civilians and those involved in armed conflicts. Sequelae of TBI includes depression in up to 30% of cases. Appearance of depression after TBI impairs recovery and results in sub-optimal re-integration into society. Treatment of post TBI depression relies on oral medications, whose efficacy and side effect profile is sub-optimal. Exercise training is effective in spontaneous depression as a stand alone treatment and in conjunction with oral medications, and this may represent an alternative treatment option in the TBI population. Further, the pathophysiology of depression after TBI has yet to be examined with the full scientific rigor required. This project proposes to use exercise training of moderate intensity as a treatment for depression after TBI, while measuring biological markers as a way to investigate efficacy and provide insight into the pathophysiology.
### Conditions Module
**Conditions:**
- Depression
- Traumatic Brain Injury
**Keywords:**
- exercise
- depression
- traumatic brain injury
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** SINGLE
**Who Masked:**
- PARTICIPANT
#### Enrollment Info
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** This group will use a stretching and flexibility program designed to utilize minimal levels of aerobic capacity. It was chosen in place of a education-based control group due to the high level of personal interaction that is found in the active arm.
**Intervention Names:**
- Other: stretching and flexibility program
**Label:** stretching-flexibility
**Type:** PLACEBO_COMPARATOR
#### Arm Group 2
**Description:** aerobic activity targeting 60% peak heart rate for 12 weeks is the active group.
**Intervention Names:**
- Other: aerobic exercise
**Label:** aerobic exercise group
**Type:** ACTIVE_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- aerobic exercise group
**Description:** the active arm will be asked to perform 60% of maximum heart rate
**Name:** aerobic exercise
**Type:** OTHER
#### Intervention 2
**Arm Group Labels:**
- stretching-flexibility
**Description:** stretching and flexibility program for the Placebo Comparator Arm
**Name:** stretching and flexibility program
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Description:** The primary aim is to measure the rate of completion of the exercise protocol.
**Measure:** completion of exercise protocol
**Time Frame:** 12 weeks
#### Secondary Outcomes
**Description:** cognitive testing battery will be performed to examine the impact of exercise on cognition in depressed traumatic brain injury subjects. Administered to both arms.
**Measure:** neuropsychological testing
**Time Frame:** pre and post intervention, week zero and week 13
**Description:** depression symptom inventories will be performed to examine the impact of exercise on cognition in depressed traumatic brain injury subjects. Administered to both arms.
**Measure:** mood assessment
**Time Frame:** week 0, week 13, and weekly during 12 week intervention
**Description:** Columbia-Suicide Severity Rating Scale was developed to meet the need for tracking changes in a person's suicidal thinking and behavior over time, and to determine who is most at risk. Administered to both arms.
**Measure:** Columbia-Suicide Severity Rating Scale
**Time Frame:** week 0, week 13, and weekly during 12 week intervention
**Description:** MRI will be done before and after exercise protocol on a subset of subjects. Selection of these subjects will be done by randomization, starting once the MRI is available for use. The purpose is to examine the brain for exercise influenced volumetric changes. Administered to a subset of both arms of the study.
**Measure:** magnetic resonance imaging
**Time Frame:** week 0, week 13
**Description:** brain-derived neurotrophic factor, vascular endothelial growth factor, insulin-like growth factor-1. The listed neurotrophic factors have been linked to depression and have been shown to be influenced by exercise. The impact of depression and exercise in the context of traumatic brain injury is unknown. Administered to both study arms.
**Measure:** biochemical assays
**Time Frame:** week 0, week 13
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* CT brain with subdural hematoma, subarachnoid hemorrhage, and/or cortical contusion
* Glasgow outcome scale extended of 4 to 8
* speaks English
* Beck Depression Inventory -II score between 14 and 28
* sedentary life style
* one to three years after traumatic brain injury
Exclusion Criteria:
* prior history of brain process (example, stroke, brain tumor)
* aphasia
* psychosis, mania, bipolar disorder, schizophrenia
* pregnancy
* inability to exercise via cycles or treadmill
**Maximum Age:** 65 Years
**Minimum Age:** 14 Years
**Sex:** ALL
**Standard Ages:**
- CHILD
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Richmond
**Country:** United States
**Facility:** Virginia Commonwealth University
**State:** Virginia
**Zip:** 23298-0568
#### Overall Officials
**Official 1:**
**Affiliation:** Virginia Commonwealth University
**Name:** Anne Hudak, MD
**Role:** PRINCIPAL_INVESTIGATOR
### References Module
#### References
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**Citation:** Dunn AL, Trivedi MH, Kampert JB, Clark CG, Chambliss HO. Exercise treatment for depression: efficacy and dose response. Am J Prev Med. 2005 Jan;28(1):1-8. doi: 10.1016/j.amepre.2004.09.003.
**PMID:** 15626549
**Citation:** Hudak A, Warner M, Marquez de la Plata C, Moore C, Harper C, Diaz-Arrastia R. Brain morphometry changes and depressive symptoms after traumatic brain injury. Psychiatry Res. 2011 Mar 31;191(3):160-5. doi: 10.1016/j.pscychresns.2010.10.003.
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**Citation:** Jorge RE, Robinson RG, Arndt SV, Starkstein SE, Forrester AW, Geisler F. Depression following traumatic brain injury: a 1 year longitudinal study. J Affect Disord. 1993 Apr;27(4):233-43. doi: 10.1016/0165-0327(93)90047-n.
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**PMID:** 10080551
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**PMID:** 14660225
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**PMID:** 16895046
**Citation:** Singh NA, Clements KM, Singh MA. The efficacy of exercise as a long-term antidepressant in elderly subjects: a randomized, controlled trial. J Gerontol A Biol Sci Med Sci. 2001 Aug;56(8):M497-504. doi: 10.1093/gerona/56.8.m497.
**PMID:** 11487602
**Citation:** Gordon WA, Sliwinski M, Echo J, McLoughlin M, Sheerer MS, Meili TE. The benefits of exercise in individuals with traumatic brain injury: a retrospective study. J Head Trauma Rehabil. 1998 Aug;13(4):58-67. doi: 10.1097/00001199-199808000-00006.
**PMID:** 9651240
**Citation:** Driver S, Ede A. Impact of physical activity on mood after TBI. Brain Inj. 2009 Mar;23(3):203-12. doi: 10.1080/02699050802695574.
**PMID:** 19205956
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#### See Also Links
**Label:** Arhinoula A. Martinos Center for Biomedical Imaging, Charlestown, Massachusetts freely available online
**URL:** http://surfer.nmr.mgh.harvard.edu
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000001526
- Term: Behavioral Symptoms
- ID: D000001927
- Term: Brain Diseases
- ID: D000002493
- Term: Central Nervous System Diseases
- ID: D000009422
- Term: Nervous System Diseases
- ID: D000006259
- Term: Craniocerebral Trauma
- ID: D000020196
- Term: Trauma, Nervous System
### Condition Browse Module - Browse Branches
- Abbrev: BC10
- Name: Nervous System Diseases
- Abbrev: BC26
- Name: Wounds and Injuries
- Abbrev: All
- Name: All Conditions
- Abbrev: BXM
- Name: Behaviors and Mental Disorders
### Condition Browse Module - Browse Leaves
- ID: M5207
- Name: Brain Injuries
- Relevance: HIGH
- As Found: Brain Injury
- ID: M7058
- Name: Depression
- Relevance: HIGH
- As Found: Depression
- ID: M7061
- Name: Depressive Disorder
- Relevance: LOW
- As Found: Unknown
- ID: M628
- Name: Brain Injuries, Traumatic
- Relevance: HIGH
- As Found: Traumatic Brain Injury
- ID: M17685
- Name: Wounds and Injuries
- Relevance: HIGH
- As Found: Injury
- ID: M4818
- Name: Behavioral Symptoms
- Relevance: LOW
- As Found: Unknown
- ID: M5204
- Name: Brain Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5742
- Name: Central Nervous System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M9349
- Name: Craniocerebral Trauma
- Relevance: LOW
- As Found: Unknown
- ID: M22023
- Name: Trauma, Nervous System
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000001930
- Term: Brain Injuries
- ID: D000070642
- Term: Brain Injuries, Traumatic
- ID: D000014947
- Term: Wounds and Injuries
- ID: D000003863
- Term: Depression
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT05817279
**Brief Title:** AI-aided Optical Coherence Tomography for the Detection of Basal Cell Carcinoma
**Official Title:** AI-aided Optical Coherence Tomography for the Detection of Basal Cell Carcinoma
#### Organization Study ID Info
**ID:** 2022-3517
#### Organization
**Class:** OTHER
**Full Name:** Maastricht University Medical Center
### Status Module
#### Completion Date
**Date:** 2024-12-31
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2024-02-16
**Type:** ACTUAL
**Last Update Submit Date:** 2024-02-15
**Overall Status:** RECRUITING
#### Primary Completion Date
**Date:** 2024-12-31
**Type:** ESTIMATED
#### Start Date
**Date:** 2023-04-10
**Type:** ACTUAL
**Status Verified Date:** 2024-02
#### Study First Post Date
**Date:** 2023-04-18
**Type:** ACTUAL
**Study First Submit Date:** 2023-04-04
**Study First Submit QC Date:** 2023-04-04
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Maastricht University Medical Center
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** Basal cell carcinoma (BCC) is the most common form of cancer among the Caucasian population. A BCC diagnosis is commonly establish by means of an invasive punch biopsy (golden standard). Optical coherence tomography (OCT) is a safe non-invasive diagnostic modality which may replace biopsy if an OCT assessor is able to establish a high confidence BCC diagnosis. Hence, for clinical implementation of OCT, diagnostic certainty should be as high as possible. Artificial intelligence in the form of a clinical decision support system (CDSS) may improve the diagnostic certainty of newly trained OCT assessors by highlighting suspicious areas on OCT scans and by providing diagnostic suggestions (classification). This study will evaluate the effect of a CDSS on the diagnostic certainty and accuracy of OCT assessors.
**Detailed Description:** In this diagnostic case control design, OCT assessors will retrospectively evaluate OCT scans of equivocal BCC lesions twice (once with, and once without the help of the CDSS). A total of 124 scans (62 BCC/62 non-BCC) will be included in the study. Cases will be shuffled to prevent recall bias. AI-aided OCT scans and unaided OCT scans will be presented in alternating order. The assessors will express their certainty level on a 5-point confidence scale. The diagnostic certainty and diagnostic accuracy of OCT assessment with CDSS and without CDSS will be compared.
Research questions:
1. Does AI-aided OCT assessment result in an increase in high-confidence diagnoses compared to unaided OCT assessment?
2. Does AI-aided OCT assessment result in a significant increase in sensitivity for BCC detection without compromising specificity compared to unaided OCT assessment?
3. Does AI-aided OCT assessment result in more accurate BCC subtyping compared to unaided OCT assessment (explorative)
### Conditions Module
**Conditions:**
- Basal Cell Carcinoma
- Optical Coherence Tomography
**Keywords:**
- Basal cell carcinoma
- BCC
- Optical coherence tomography
- OCT
- Imaging
- Artificial intelligence
- Machine learning
### Design Module
#### Design Info
**Observational Model:** CASE_CONTROL
**Time Perspective:** RETROSPECTIVE
#### Enrollment Info
**Count:** 124
**Type:** ESTIMATED
**Study Type:** OBSERVATIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Group of 124 patients with equivocal BCC lesions. Of these lesions, OCT scans have been obtained in the past. These scans will be evaluated with AI-assistance.
**Intervention Names:**
- Diagnostic Test: Optical coherence tomography
**Label:** AI-OCT
#### Arm Group 2
**Description:** Group of 124 patients with equivocal BCC lesions (same patients as in AI-OCT group). Of these lesions, OCT scans have been obtained in the past. These scans will be evaluated without AI-assistance.
**Intervention Names:**
- Diagnostic Test: Optical coherence tomography
**Label:** Unaided OCT
### Interventions
#### Intervention 1
**Arm Group Labels:**
- AI-OCT
- Unaided OCT
**Description:** Optical coherence tomography: OCT is a non-invasive CE-certified diagnostic modality based on light interferometry. An OCT scan visualizes an area with a diameter of 6mm thereby revealing the skin and adnexal structures with a depth of approximately 1.5mm.
3mm punch biopsy: the patients included in this study underwent a 3mm punch biopsy conform regular care. The subsequent histopathological examination of the biopsy specimen serves as ground truth diagnosis of the lesions (gold standard)
**Name:** Optical coherence tomography
**Other Names:**
- 3mm punch biopsy
**Type:** DIAGNOSTIC_TEST
### Outcomes Module
#### Primary Outcomes
**Description:** The difference in percentage of high-confidence diagnoses will be evaluated between AI-OCT and unaided OCT.
**Measure:** Proportion of high-confidence diagnoses
**Time Frame:** 31-12-2023
#### Secondary Outcomes
**Description:** Diagnostic parameters (sensitivity, specificity, positive predictive value, negative predicted value, diagnostic odds ratio) will be estimated for high-confidence diagnoses made by AI-OCT and unaided OCT.
**Measure:** Diagnostic accuracy of high-confidence diagnoses
**Time Frame:** 31-12-2023
**Description:** Differences in diagnostic parameters for BCC subtyping (sBCC/nBCC/iBCC) will be evaluated (explorative)
**Measure:** Diagnostic parameters for BCC subtyping
**Time Frame:** 31-12-2023
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Patients (18+ years)
* Patient underwent OCT scan and punch biopsy for an equivocal BCC lesion
Exclusion Criteria:
- Patient unable to sign informed consent
**Minimum Age:** 18 Years
**Sampling Method:** PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
**Study Population:** Scans of patients with equivocal BCC lesions will be included. Patients previously underwent an OCT scan and punch biopsy for their lesion. Patients signed informed consent for the use of the OCT scans made and patient information for the sake of answering research questions regarding OCT.
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Maastricht
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Tom Wolswijk, MD, MSc
- **Phone:** +31(0)42-3877295
- **Role:** CONTACT
**Country:** Netherlands
**Facility:** Maastricht University Medical Center+
**State:** Limbrug
**Status:** RECRUITING
**Zip:** 6202AZ
### IPD Sharing Statement Module
**IPD Sharing:** UNDECIDED
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000009375
- Term: Neoplasms, Glandular and Epithelial
- ID: D000009370
- Term: Neoplasms by Histologic Type
- ID: D000009369
- Term: Neoplasms
- ID: D000018295
- Term: Neoplasms, Basal Cell
### Condition Browse Module - Browse Branches
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: All
- Name: All Conditions
### Condition Browse Module - Browse Leaves
- ID: M5534
- Name: Carcinoma
- Relevance: HIGH
- As Found: Carcinoma
- ID: M5537
- Name: Carcinoma, Basal Cell
- Relevance: HIGH
- As Found: Basal Cell Carcinoma
- ID: M12320
- Name: Neoplasms, Glandular and Epithelial
- Relevance: LOW
- As Found: Unknown
- ID: M12315
- Name: Neoplasms by Histologic Type
- Relevance: LOW
- As Found: Unknown
- ID: M20439
- Name: Neoplasms, Basal Cell
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000002277
- Term: Carcinoma
- ID: D000002280
- Term: Carcinoma, Basal Cell
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT02315079
**Brief Title:** Point of Care Testing of Inflammatory Markers in Tears
**Official Title:** Point of Care Testing of Inflammatory Markers in Tears
#### Organization Study ID Info
**ID:** IRB201400775
#### Organization
**Class:** OTHER
**Full Name:** University of Florida
### Status Module
#### Completion Date
**Date:** 2017-01
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2019-07-24
**Type:** ACTUAL
**Last Update Submit Date:** 2019-07-23
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2017-01
**Type:** ACTUAL
#### Start Date
**Date:** 2015-01
**Status Verified Date:** 2019-07
#### Study First Post Date
**Date:** 2014-12-11
**Type:** ESTIMATED
**Study First Submit Date:** 2014-12-09
**Study First Submit QC Date:** 2014-12-10
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** University of Florida
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** To evaluate the use of a point of care device to measure markers of inflammation in various eye conditions. In particular, matrix metalloproteinase-9 (MMP-9) will be measured. MMP-9 is an enzyme that plays a role in inflammation. The value obtained with the point of care device will be correlated with values obtained using gel electrophoresis to measure MMP-9 from the same sample. These data will be compared to clinical exam findings and questionnaires to help the investigators better understand the role of this marker of inflammation in eye diseases and possibly improve diagnostic abilities.
**Detailed Description:** As a participant in the study a sample of tears will be collected in a non-invasive manner by sitting comfortably at the slit lamp (a slit lamp is the standard device used to examine the eye and merely requires the participant to sit upright and place their chin on a chin rest and their forehead up against the slit lamp apparatus). The participant will then be asked to look upwards and while gently lowering the lower eyelid, a sterile blunt glass pipette will be used to collect tears from the natural tear lake (the tear lake is a reservoir of tears that sits on the surface of the eye and above the lower eyelid). Collected tears will then be taken for analysis of inflammatory markers.
In addition, a survey to evaluate the symptoms and the symptoms impact daily life (Ocular Surface Disability Index Survey of National Eye Institute Visual Functioning Questionnaire- 25).
### Conditions Module
**Conditions:**
- Dry Eye Syndrome
**Keywords:**
- Ocular Inflammation
- Matrix Metalloproteinase 9
### Design Module
#### Bio Spec
**Description:** de-identified tear samples
**Retention:** SAMPLES_WITHOUT_DNA
#### Design Info
**Observational Model:** COHORT
**Time Perspective:** PROSPECTIVE
#### Enrollment Info
**Count:** 63
**Type:** ACTUAL
**Study Type:** OBSERVATIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** This group will have a regular eye exam with the collection of tears. In addition, a the Ocular Surface Disability Index Survey of National Eye Institute Visual Functioning Questionnaire- 25 may be administered.
**Intervention Names:**
- Other: Eye inflammation
**Label:** Eye inflammation
#### Arm Group 2
**Description:** This group will have a regular eye exam with the collection of tears.
**Intervention Names:**
- Other: Without eye inflammation
**Label:** Without eye inflammation
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Eye inflammation
**Description:** This group will have a regular eye exam with the collection of tears. In addition, a the Ocular Surface Disability Index Survey of National Eye Institute Visual Functioning Questionnaire- 25 may be administered.
**Name:** Eye inflammation
**Type:** OTHER
#### Intervention 2
**Arm Group Labels:**
- Without eye inflammation
**Description:** This group will have a regular eye exam with the collection of tears.
**Name:** Without eye inflammation
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Measure:** Measurement of matrix metalloproteinase-9 in tears
**Time Frame:** Baseline
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Patients that present to clinic for evaluation. Patients with and without evidence of eye inflammation will be eligible for this study.
Exclusion Criteria:
* Patients outside the age limits will not be eligible for the study
**Healthy Volunteers:** True
**Maximum Age:** 90 Years
**Minimum Age:** 18 Years
**Sampling Method:** NON_PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
**Study Population:** Patients aged 18-90 years of age that present to our North Central Florida Tertiary Referral University Eye Clinics for evaluation. Patients with and without evidence of eye inflammation will be eligible for this study.
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Gainesville
**Country:** United States
**Facility:** University of Florida, Department of Ophthalmology
**State:** Florida
**Zip:** 32610
#### Overall Officials
**Official 1:**
**Affiliation:** University of Florida, Department of Ophthalmology
**Name:** Sonal Tuli, MD
**Role:** PRINCIPAL_INVESTIGATOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000007766
- Term: Lacrimal Apparatus Diseases
- ID: D000005128
- Term: Eye Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: All
- Name: All Conditions
- Abbrev: BC11
- Name: Eye Diseases
- Abbrev: BC26
- Name: Wounds and Injuries
### Condition Browse Module - Browse Leaves
- ID: M10293
- Name: Inflammation
- Relevance: LOW
- As Found: Unknown
- ID: M16355
- Name: Syndrome
- Relevance: LOW
- As Found: Unknown
- ID: M18040
- Name: Dry Eye Syndromes
- Relevance: HIGH
- As Found: Dry Eye Syndrome
- ID: M10664
- Name: Keratoconjunctivitis Sicca
- Relevance: LOW
- As Found: Unknown
- ID: M15241
- Name: Rupture
- Relevance: LOW
- As Found: Unknown
- ID: M22785
- Name: Lacerations
- Relevance: LOW
- As Found: Unknown
- ID: M10786
- Name: Lacrimal Apparatus Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M8271
- Name: Eye Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000015352
- Term: Dry Eye Syndromes
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT04904679
**Brief Title:** The Use of Amniotic Membrane or Internal Limiting Membrane for Large or Refractory Macular Holes: A Prospective Study
**Official Title:** The Use of Amniotic Membrane or Internal Limiting Membrane for Large or Refractory Macular Holes: A Prospective Study
#### Organization Study ID Info
**ID:** AMprotocol
#### Organization
**Class:** OTHER
**Full Name:** Hospital Oftalmologico de Sorocaba
### Status Module
#### Completion Date
**Date:** 2023-12-31
**Type:** ESTIMATED
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2023-10-03
**Type:** ACTUAL
**Last Update Submit Date:** 2023-09-30
**Overall Status:** ACTIVE_NOT_RECRUITING
#### Primary Completion Date
**Date:** 2023-09-30
**Type:** ACTUAL
#### Start Date
**Date:** 2020-06-26
**Type:** ACTUAL
**Status Verified Date:** 2023-09
#### Study First Post Date
**Date:** 2021-05-27
**Type:** ACTUAL
**Study First Submit Date:** 2021-05-17
**Study First Submit QC Date:** 2021-05-24
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Hospital Oftalmologico de Sorocaba
#### Responsible Party
**Investigator Affiliation:** Hospital Oftalmologico de Sorocaba
**Investigator Full Name:** Anna Carolina Carvalho Araujo
**Investigator Title:** Ophthalmologist , MD
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** Prospective, randomized, unmasked interventional study. To evaluate anatomical and functional results through microperimetry in cases of refractory or large macular holes (MH), using amniotic membrane (AM) or internal limiting membrane ( ILM ).
**Detailed Description:** This study will evaluate functional results through microperimetry of patients undergoing macular hole surgery ( for patients with macular holes ≥ 600 microns and in cases refractory to conventional surgical treatment) using amniotic membrane or internal limiting membrane as an adjunct in the closure process of the hole.
The current treatment considered the gold standard for the idiopathic macular holes consists of pars plana vitrectomy (PPV) with removal of the posterior hyaloid (when adhered) and the internal limiting membrane (ILM) with the help of a vital dye and subsequent placement of buffering gas (C3F8 or SF6) in non-expandable concentration. Approximately 44% of the large holes do not close after conventional surgery.
To increase the closure rate in these cases, new techniques have been proposed, such as the inverted ILM flap technique and the free ILM flap technique. Many studies demonstrate the effectiveness of these 2 techniques, but with no visual function improvement.
The amniotic membrane has been used in some cases as an adjunct in the macular holes closure and as a substrate for cell growth and improvement of visual acuity. Amniotic membranes are supposed to promote epithelialization and have anti-fibrotic, anti-inflammatory, anti-angiogenic and anti-microbial properties.
In the present study, the patients who meet the inclusion criteria will be randomized and undergo pars plana vitrectomy surgery with removal of the ILM and a fragment of the ILM itself or an amniotic membrane plug will be put in place approximately 1 -1.5 mm under the adjacent retina, inside the macular hole, with the chorionic face in contact with the retinal pigmented epithelium (RPE). After certifying that the plug is in the correct location, the gas fluid exchange will be performed, with a C3F8 buffering infusion of 12.5% and the patients will perform the face down position for 7 days.
The patients included in the study will undergo a complete eye examination, including corrected Visual Acuity measurement (BCVA), performing the Ocular Coherence Tomography ( OCT) using the Zeiss Cirrus 5000 equipment and microperimetry using the macular integrity assessment (MAIA) equipment in the pre-operative. Patients will undergo surgery after complementary exams and adequate pre-anesthetic evaluation.
Patients will be evaluated on the 1st and 7th postoperative days and at 1, 3, 6 and 12 months after surgery. The patient will undergo a complete eye examination and postoperative follow-up as described above, and during visits on the 7th day, and in the 1st, 3rd, 6th and 12th month of the postoperative period, OCT will be performed and the microperimetry will be performed in the 1st, 3rd, 6th and 12th postoperative month for evaluation and monitoring of anatomical and functional responses, respectively.
The use of the amniotic membrane, due to its anti-inflammatory, anti-fibrotic and substrate properties for cell growth is supposed do increase the rate of closure in the refractory and large holes, as well as promoting a better functional response by providing better reconstitution of the outer retinal layers.The amniotic membrane will be provided for this study by the SorocabaEye Bank (BOS), where it will be prepared and preserved.
### Conditions Module
**Conditions:**
- Macular Holes
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
**Intervention Model Description:** Prospective, randomized, unmasked single center interventional study
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 23
**Type:** ESTIMATED
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Patients who will be treated with a amniotic membrane plug
**Intervention Names:**
- Device: Pars plana Vitrectomy with internal limiting membrane peeling
**Label:** Amniotic Membrane group
**Type:** ACTIVE_COMPARATOR
#### Arm Group 2
**Description:** patients who will be treated with a internal limiting membrane flap
**Intervention Names:**
- Device: Pars plana Vitrectomy with internal limiting membrane peeling
**Label:** Internal Limiting Membrane group
**Type:** ACTIVE_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Amniotic Membrane group
- Internal Limiting Membrane group
**Description:** The vitrectomy technique using pars plana 23-gauge will be performed with removal of the ILM (when present adhered to the macula) and placement of an amniotic membrane plug or a internal limiting membrane flap in the macular hole, with a fluid-air exchange and a buffering gas infusion (C3F8 12, 5%). The amniotic membrane plug will be made with a dermatological punch with a diameter of 1.0 to 2mm depending on the size of the macular hole measured by the OCT, and will be positioned inside de macular hole with the chorionic side facing the RPE ( retinal pigmented epithelium) with a 23 gauge forceps. The chorionic side of the membrane is the sticky one. The internal limiting membrane flap will be created at the same time of peeling and will bem positioned in as inverted flap over and inside the macular hole with a 23 gauge ILM forceps. These technics will be helped with de use of a extra lighting sclerotomy.
**Name:** Pars plana Vitrectomy with internal limiting membrane peeling
**Other Names:**
- Use of Amniotic membrane plug
- Use of a internal limiting membrane inverted flap
- gas tamponade (C3F8 12,5%)
**Type:** DEVICE
### Outcomes Module
#### Primary Outcomes
**Description:** measured by the microperimetry
**Measure:** The retinal sensibility
**Time Frame:** through study completion, an average of 1 year
**Description:** measured by the microperimetry
**Measure:** The fixation stability
**Time Frame:** through study completion, an average of 1 year
#### Secondary Outcomes
**Description:** measured by OCT
**Measure:** the macula hole closure rate
**Time Frame:** through study completion, an average of 1 year
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Patients with a macula hole that falls into the two categories below:
* Patients with idiopathic macular hole (with no history of secondary macular hole) ≥ 600 microns (measured using optical coherence tomography)
* Patients with an idiopathic macular hole submitted to conventional treatment without closing it (of any size).
* History of the disease less than 18 months
* Aged over 18 years old
* Visual acuity less than 20/32
Exclusion Criteria:
* History of any previous macular disease other than idiopathic macular hole
* Macular hole of other causes (secondary)
* Patients with diabetic retinopathy or other retinal vascular diseases
* Eyes subjected to intravitreal injection of any medication
* Visual acuity \<20/400 of any retinal cause in the contralateral eye or absence of the globe \*Aged under 50 years old
* Cataract and anti-glaucoma surgery less than 3 months before the study
* Glaucoma with optic nerve excavation \> 0.7 in the studied eye
* Intraocular pressure \> 24 mmHg with the use of maximum medication in the studied eye
* History of vitreoretinal surgery for a condition other than the idiopathic macular hole (retinal detachment, vitreous hemorrhage)
* History of retinal detachment of any etiology
* A patient who manifests himself not being able to perform the head position in the postoperative period
* A patient with active anterior or posterior uveitis
**Minimum Age:** 50 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Sorocaba
**Country:** Brazil
**Facility:** Sorocaba Eye's Hospital
**State:** Sao Paulo
**Zip:** 18031060
### IPD Sharing Statement Module
**IPD Sharing:** NO
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000012164
- Term: Retinal Diseases
- ID: D000005128
- Term: Eye Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC11
- Name: Eye Diseases
- Abbrev: All
- Name: All Conditions
### Condition Browse Module - Browse Leaves
- ID: M15002
- Name: Retinal Perforations
- Relevance: HIGH
- As Found: Macular Hole
- ID: M14999
- Name: Retinal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M8271
- Name: Eye Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000012167
- Term: Retinal Perforations
### Intervention Browse Module - Browse Branches
- Abbrev: Derm
- Name: Dermatologic Agents
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M7074
- Name: Dermatologic Agents
- Relevance: LOW
- As Found: Unknown
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT02196779
**Brief Title:** Laser Fluorescent Imaging of Abdominal Skin During Abdominoplasty
**Official Title:** Laser Fluorescent Imaging of Abdominal Skin During Abdominoplasty
#### Organization Study ID Info
**ID:** Swanson 003
#### Organization
**Class:** OTHER
**Full Name:** Swanson Center
### Status Module
#### Completion Date
**Date:** 2014-11
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2014-11-20
**Type:** ESTIMATED
**Last Update Submit Date:** 2014-11-18
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2014-11
**Type:** ACTUAL
#### Start Date
**Date:** 2014-07
**Status Verified Date:** 2014-11
#### Study First Post Date
**Date:** 2014-07-22
**Type:** ESTIMATED
**Study First Submit Date:** 2014-07-19
**Study First Submit QC Date:** 2014-07-21
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Swanson Center
#### Responsible Party
**Investigator Affiliation:** Swanson Center
**Investigator Full Name:** Eric Swanson, M.D.
**Investigator Title:** Principal Investigator
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** Abdominoplasty is a common cosmetic operation. Some investigators believe that the risk of complications is reduced by limiting the operative dissection. However, this can compromise the quality of the result. This study was undertaken to investigate the blood supply of the abdominoplasty skin flap during surgery using laser fluorescent imaging. An imaging agent is injected and the fluorescence is recorded using a near-infrared laser. The blood supply is tested after a limited dissection and then after a traditional dissection to determine whether an extended dissection reduces the skin circulation. The study hypothesis is that there is no difference in blood supply and the traditional abdominoplasty does not compromise skin circulation.
**Detailed Description:** Protocol
Laser Fluorescent Imaging of Abdominal Skin During Abdominoplasty.
Background/Purpose
Abdominoplasty (tummy tuck) is a common cosmetic operation. One of the risks of surgery is delayed wound healing caused by impaired circulation to the skin. In an effort to reduce risk, some investigators1 advocate a limited-undermining technique, with preservation of the perforating blood vessels and preservation of the Scarpa fascia of the lower abdomen. However, there is no evidence that this limited-undermining technique improves blood supply compared with a traditional abdominoplasty dissection. Such a limited dissection may compromise the cosmetic result.
This study was undertaken to investigate the blood supply of the abdominal skin during the abdominoplasty procedure. By testing the circulation at two points during the dissection, and a third time after completion of the operation, data may be obtained and used to evaluate the effect of the extent of the dissection on blood supply of the skin flap.
Laser fluorescent imaging represents an objective measurement technique that has been used to quantitate blood supply in skin flaps and other tissues. It has been used for several years to monitor the vascularity of flaps used in breast reconstruction. This technique has also been used previously in patients undergoing abdominoplasty.2
Subjects
Twenty consecutive consenting adult men and women undergoing outpatient elective abdominoplasty will be investigated using laser fluorescent imaging. By testing the blood supply at three time points, the patient serves as his or her own control.
Imaging
The SPY Elite Intraoperative Perfusion Assessment System (Lifecell Corp., Branchburg, NJ) consists of a near-infrared laser that detects fluorescence. The imaging agent, indocyanine green, is absorbed at this region of the spectrum, 800-810 nm. The imaging agent is injected intravenously. Starting within 5-10 seconds of injection, the system records images of the abdomen, showing the fluorescence, which indicates blood supply. The half-life of the imaging agent is 2.5-3 minutes. The imaging agent is metabolized by the liver. The only known risk is an allergic reaction to the imaging agent. Patients with a history of an allergic reaction to iodinated contrast dyes are excluded.
Surgery
The lipoabdominoplasty technique is performed as described by Saldanha et al.1 Following this limited dissection, 3 mL (7.5 mg) of indocyanine green is injected and the abdominal image is recorded, documenting blood supply to the lower abdomen. After further dissection, another 3 mL of imaging agent is injected and the lower abdomen is re-imaged. On completion of surgery, a third injection is administered and a final image is recorded.
Null Hypothesis
A traditional abdominoplasty dissection does not compromise circulation compared with a more limited dissection technique.
Informed Consent
Patients are informed as to the nature of the study and are told that their participation is entirely voluntary and they are free to decline, and that doing so does not in any way prejudice their treatment. Patients are informed of the small risk of an allergic reaction that will be treated if it occurs.
Patient Risk
There is no patient risk apart from a very small risk of allergic reaction, which is further reduced by excluding any patient with a known allergy to iodinated contrast dye. The study does not affect patient treatment. It offers the possibility of detection of reduced skin circulation, information that can be used by the surgeon to limit the dissection if necessary and avoid tissue loss and delayed wound healing, known complications of abdominoplasty.
Sample Size
A sample of 20 patients is anticipated, treated over a period of approximately 6 months.
Disclosure
The author has no financial interest in any of the products, devices, or drugs mentioned in this article. The author has no conflicts of interest to disclose. There was no outside funding for this study.
### Conditions Module
**Conditions:**
- Abdominal Skin Redundancy
**Keywords:**
- Abdominoplasty
- circulation
- skin
- dissection
- laser
- fluorescence
- imaging
### Design Module
#### Design Info
**Observational Model:** CASE_ONLY
**Time Perspective:** PROSPECTIVE
#### Enrollment Info
**Count:** 20
**Type:** ACTUAL
**Patient Registry:** True
**Study Type:** OBSERVATIONAL
**Target Duration:** 6 Months
### Arms Interventions Module
#### Arm Group 1
**Description:** Skin circulation to abdominal flap is evaluated during surgery using laser fluorescent imaging.
**Label:** Patients undergoing abdominoplasty
### Outcomes Module
#### Primary Outcomes
**Description:** The blood supply to the skin is evaluated intraoperatively using laser fluorescent imaging.
**Measure:** Skin circulation during abdominoplasty.
**Time Frame:** 6 months
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Patients undergoing abdominoplasty
Exclusion Criteria:
* Allergy to iodinated contrast agents
* Nonconsenting patients
**Healthy Volunteers:** True
**Maximum Age:** 80 Years
**Minimum Age:** 18 Years
**Sampling Method:** NON_PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
**Study Population:** Adult men and women undergoing abdominoplasty.
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Leawood
**Country:** United States
**Facility:** Swanson Center
**State:** Kansas
**Zip:** 66211
#### Overall Officials
**Official 1:**
**Affiliation:** Swanson Center
**Name:** Eric Swanson, M.D.
**Role:** PRINCIPAL_INVESTIGATOR
### References Module
#### References
**Citation:** Roostaeian J, Harris R, Farkas JP, Barton FE, Kenkel JM. Comparison of Limited-Undermining Lipoabdominoplasty and Traditional Abdominoplasty Using Laser Fluorescence Imaging. Aesthet Surg J. 2014 Jul;34(5):741-7. doi: 10.1177/1090820X14532286. Epub 2014 Jul 1.
**PMID:** 24809360
**Citation:** Swanson E. Comparison of Limited and Full Dissection Abdominoplasty Using Laser Fluorescence Imaging to Evaluate Perfusion of the Abdominal Skin. Plast Reconstr Surg. 2015 Jul;136(1):31e-43e. doi: 10.1097/PRS.0000000000001376.
**PMID:** 26111330
## Derived Section
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT01147679
**Brief Title:** Study of Social Behavior and Emotion in Frontotemporal Dementia, Alzheimer's Disease and Controls
**Official Title:** Study of Social Behavior and Emotion in Frontotemporal Dementia, Alzheimer's Disease and Controls
#### Organization Study ID Info
**ID:** 1R01AG034499
**Link:** https://reporter.nih.gov/quickSearch/1R01AG034499
**Type:** NIH
#### Organization
**Class:** OTHER
**Full Name:** University of California, Los Angeles
#### Secondary ID Infos
**Domain:** UCLA Institutional Research Board
**ID:** UCLA IRB#10-001097
**Type:** OTHER
**ID:** R01AG034499
**Link:** https://reporter.nih.gov/quickSearch/R01AG034499
**Type:** NIH
### Status Module
#### Completion Date
**Date:** 2014-07
**Type:** ESTIMATED
#### Expanded Access Info
**Last Known Status:** RECRUITING
#### Last Update Post Date
**Date:** 2011-10-28
**Type:** ESTIMATED
**Last Update Submit Date:** 2011-10-27
**Overall Status:** UNKNOWN
#### Primary Completion Date
**Date:** 2013-09
**Type:** ESTIMATED
#### Start Date
**Date:** 2010-01
**Status Verified Date:** 2011-10
#### Study First Post Date
**Date:** 2010-06-22
**Type:** ESTIMATED
**Study First Submit Date:** 2010-06-18
**Study First Submit QC Date:** 2010-06-18
### Sponsor Collaborators Module
#### Collaborators
**Class:** NIH
**Name:** National Institute on Aging (NIA)
#### Lead Sponsor
**Class:** OTHER
**Name:** University of California, Los Angeles
#### Responsible Party
**Investigator Affiliation:** University of California, Los Angeles
**Investigator Full Name:** Mario F. Mendez
**Investigator Title:** Professor of Neurology and Psychiatry
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** This study is designed to document the loss of sociomoral emotions (like empathy, guilt, and embarrassment) in patients with behavioral variant frontotemporal dementia. The loss of these emotions, which function as the motivators for social behavior, will manifest in specific interpersonal behaviors. These behaviors will correlate with regional changes in regional changes in medial frontal and anterior temporal lobes. These social and emotional changes will be compared with a young-onset Alzheimer's disease comparison group.
**Detailed Description:** Frontotemporal dementia (FTD) is a devastating disorder and one the most common neurodegenerative diseases in middle age. The most prominent early manifestations of bvFTD ("behavior variant" FTD) are not the memory and other cognitive deficits typical of Alzheimer's disease (AD) but, rather, disturbance in social or interpersonal behavior. A basic manifestation of this disorder is a disturbance in the emotions and motives that drive social and moral behavior. In fact, bvFTD is an incredible window to the neuroscience of social behavior. This study will help clarify the neurobiological substrates of sociomoral emotions and their associated clinical features. The findings of this proposal can have major implications for understanding the interaction between brain and social behavior and for designing future research on the basic mechanisms of social neuroscience. This research aims to document the loss of sociomoral emotions (SME) compared to primary emotions in patients with bvFTD vs. patients with AD and normal controls. We need to show that these findings are specific to bvFTD and not present in Alzheimer's disease or normal controls. The project consists of three integrated parts: 1) behavioral measures that include observations in naturalistic settings, behavioral experiments, and behavioral scales; 2) psychophysiological reactivity (i.e., measures of heart rate, blood pressure changes, galvanic skin response, facial electromyography, and facial temperature) to social and emotional stimuli; and 3) brain localization of changes in sociomoral emotions with magnetic resonance imaging technology.
### Conditions Module
**Conditions:**
- Frontotemporal Dementia
- Frontotemporal Degeneration
- Alzheimer's Disease
- Social Behavior
**Keywords:**
- Frontotemporal dementia
- Alzheimer's disease
- Young-Onset
- Frontotemporal degeneration
- control
- social
- behavior
- observation
- emotion
- morality
- dementia
- brain
- relationships
- caregiver
- MRI
- neurology
### Design Module
#### Design Info
**Observational Model:** CASE_CONTROL
**Time Perspective:** PROSPECTIVE
#### Enrollment Info
**Count:** 99
**Type:** ESTIMATED
**Study Type:** OBSERVATIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** This group will include 33 patients who have been diagnosed with behavioral variant frontotemporal dementia by Dr. Mario Mendez. Patients diagnosed elsewhere must have a secondary evaluation at the UCLA FTD Clinic to confirm their diagnosis before study enrollment.
**Label:** bvFTD
#### Arm Group 2
**Description:** This group will include 33 patients who have been diagnosed with clinically probable Alzheimer's disease by Dr. Mario Mendez. Patients diagnosed elsewhere must have a secondary evaluation at the UCLA FTD Clinic to confirm their diagnosis before study enrollment.
**Label:** Alzheimer's disease
#### Arm Group 3
**Description:** 33 health individuals without clinically significant cognitive impairments will be enrolled in this study.
**Label:** Controls
### Outcomes Module
#### Primary Outcomes
**Description:** We will evaluate autonomic (sympathetic and parasympathetic) nervous system reactivity to sociomoral vs. non-social stimuli using measures of heart rate (and heart rate variability), blood pressure (and baroreflex sensitivity), finger pulse volume, and skin conductance changes. Investigators present social and non-social pictures, videos, and written scenarios to all three groups while recording these psychophysiologic measures.
**Measure:** Psychophysiological Reactivity
**Time Frame:** within three months of study enrollment
**Description:** This proposal will use methods of ethnography to classify the social behavior of bvFTD and AD patients and their caregivers in their homes and during research visits. Four social behavioral experiments or vignettes will be used to cross-validate the results of augmented participant observation. Behavioral scales will record differences between caregiver and patient assessments of behavior and further validate the results of the participant observation.
**Measure:** Behavioral Reports and Observations
**Time Frame:** within three months of study enrollment
**Description:** An MRI scan evaluated with state-of-the-art techniques will yield three dimensional maps of localized structural changes that reflect the regions involved in mediating social, moral, and emotional behaviors.
**Measure:** MRI Brain-Mapping
**Time Frame:** within three months of study enrollment
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria (FTD or AD patients):
* The core diagnostic features of bvFTD or NINCDS-ADRDA criteria for clinically probable AD
* Mild-moderate cognitive and functional severity defined as an MMSE \>/=10 and a CDR \</=2.0
* Able to understand and complete procedures and to take part in the tests by hearing and understanding instructions and by seeing the stimuli to be responded to
* Willingness and ability to provide informed consent; Informed consents from caregiver and patient
* English speaking, having acquired English prior to age 13 and using it as primary language
* Minimally impaired language (language and semantics tests cut-off scores)
* Medically stable (defined as absence of medical illness that would interfere with the subject's ability to understand and participate in study procedures)
* Absence of a neurological or psychiatric illness other than bvFTD or clinically probable ADB
* Absence of cortical infarction, other cortical lesion, or significant subcortical lesion on MRI of brain
* Absence of potentially confounding medications, particularly those with effects on the peripheral nervous system, cardiovascular agents, and β-blockers
* Presence of a caregiver who can facilitate participation in this project. (see below) Where there is more than one caregiver, every effort is made to designate the closest relative as the main caregiver.
Exclusion Criteria (FTD or AD patients):
* Violation of any of the criteria above.
Inclusion Criteria (Study Partners/Caregivers):
* Personally visit and interact with the subject at least one time each week for one hour.
* Accompany the subject to each visit.
* Provide opinions about the subject's thinking (i.e., memory, language, problem-solving ability), daily activities (i.e., dressing, hygiene, mobility, household chores, and hobbies), and behavior (i.e., mood, sleep patterns, appetite, participation in social interactions).
* Share personal information including feelings of distress about the subject's behavior or feelings of burden by caregiving responsibilities.
* Read, understand and speak English fluently in order to ensure comprehension of informed consent form and informant-based assessments of the subject.
* Provide full written informed consent on his/her own behalf prior to the performance of any protocol-specific procedure.
* In the opinion of the investigator, the study partner will be compliant with the protocol and have a high probability of completing the study
Exclusion Criteria (Study Partners):
* Violation of any of the criteria above.
Inclusion Criteria (Control Participants):
* Denies neurological or psychiatric illness.
* Not currently a caregiver for a dementia patient (for at least one year).
* Does not take potentially confounding medications, including most of those with effects on the central nervous system and peripheral nervous system, cardiovascular agents, and β-blockers. The use of these medications will be assessed during a telephone screening.
Exclusion Criteria (Control Participants):
* Violation of any of the criteria above.
**Healthy Volunteers:** True
**Maximum Age:** 75 Years
**Minimum Age:** 40 Years
**Sampling Method:** NON_PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
**Study Population:** Patients evaluated by Dr. Mario Mendez at the UCLA Frontotemporal Dementia Clinic
### Contacts Locations Module
#### Central Contacts
**Contact 1:**
**Email:** [email protected]
**Name:** Jill Shapira, RN, PhD
**Phone:** 3107942550
**Role:** CONTACT
**Contact 2:**
**Email:** [email protected]
**Name:** Michelle Mather, BA
**Phone:** 3107946038
**Role:** CONTACT
#### Locations
**Location 1:**
**City:** Los Angeles
**Contacts:**
***Contact 1:***
- **Email:** [email protected]
- **Name:** Michelle Mather, BA
- **Phone:** 310-794-6038
- **Role:** CONTACT
***Contact 2:***
- **Email:** [email protected]
- **Name:** Jill Shapira, RN, PhD
- **Phone:** 3107942550
- **Role:** CONTACT
***Contact 3:***
- **Name:** Mario F Mendez, MD, PhD
- **Role:** PRINCIPAL_INVESTIGATOR
***Contact 4:***
- **Name:** David Shapiro, PhD
- **Role:** SUB_INVESTIGATOR
***Contact 5:***
- **Name:** Jill Shapira, RN, PhD
- **Role:** SUB_INVESTIGATOR
***Contact 6:***
- **Name:** Po-Haong Lu, PsyD
- **Role:** SUB_INVESTIGATOR
***Contact 7:***
- **Name:** John Heritage, PhD
- **Role:** SUB_INVESTIGATOR
***Contact 8:***
- **Name:** Li-Jung Liang, PhD
- **Role:** SUB_INVESTIGATOR
***Contact 9:***
- **Name:** Natalie K Wolcott, PhD
- **Role:** SUB_INVESTIGATOR
***Contact 10:***
- **Name:** Aditi Joshi, PhD
- **Role:** SUB_INVESTIGATOR
***Contact 11:***
- **Name:** Julia Hsiao, DO
- **Role:** SUB_INVESTIGATOR
***Contact 12:***
- **Name:** Collins Liu, MD
- **Role:** SUB_INVESTIGATOR
**Country:** United States
**Facility:** UCLA Department of Neurology
**State:** California
**Status:** RECRUITING
**Zip:** 90095
#### Overall Officials
**Official 1:**
**Affiliation:** University of California, Los Angeles; Veteran's Health Administration, West Los Angeles
**Name:** Mario F Mendez, MD, PhD
**Role:** PRINCIPAL_INVESTIGATOR
### References Module
#### See Also Links
**Label:** The website of the Association for Frontotemporal Degeneration
**URL:** http://www.theaftd.org
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000001927
- Term: Brain Diseases
- ID: D000002493
- Term: Central Nervous System Diseases
- ID: D000009422
- Term: Nervous System Diseases
- ID: D000024801
- Term: Tauopathies
- ID: D000019636
- Term: Neurodegenerative Diseases
- ID: D000019965
- Term: Neurocognitive Disorders
- ID: D000001523
- Term: Mental Disorders
- ID: D000057174
- Term: Frontotemporal Lobar Degeneration
- ID: D000057177
- Term: TDP-43 Proteinopathies
- ID: D000057165
- Term: Proteostasis Deficiencies
- ID: D000008659
- Term: Metabolic Diseases
- ID: D000001037
- Term: Aphasia
- ID: D000013064
- Term: Speech Disorders
- ID: D000007806
- Term: Language Disorders
- ID: D000003147
- Term: Communication Disorders
- ID: D000019954
- Term: Neurobehavioral Manifestations
- ID: D000009461
- Term: Neurologic Manifestations
### Condition Browse Module - Browse Branches
- Abbrev: BC10
- Name: Nervous System Diseases
- Abbrev: BXM
- Name: Behaviors and Mental Disorders
- Abbrev: All
- Name: All Conditions
- Abbrev: BC18
- Name: Nutritional and Metabolic Diseases
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M6904
- Name: Dementia
- Relevance: HIGH
- As Found: Dementia
- ID: M3885
- Name: Alzheimer Disease
- Relevance: HIGH
- As Found: Alzheimer's Disease
- ID: M28762
- Name: Frontotemporal Dementia
- Relevance: HIGH
- As Found: Frontotemporal Dementia
- ID: M20929
- Name: Aphasia, Primary Progressive
- Relevance: HIGH
- As Found: Frontotemporal Dementia
- ID: M22530
- Name: Pick Disease of the Brain
- Relevance: HIGH
- As Found: Frontotemporal Dementia
- ID: M4352
- Name: Aphasia
- Relevance: LOW
- As Found: Unknown
- ID: M5204
- Name: Brain Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5742
- Name: Central Nervous System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M23002
- Name: Tauopathies
- Relevance: LOW
- As Found: Unknown
- ID: M21558
- Name: Neurodegenerative Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M21836
- Name: Neurocognitive Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M4815
- Name: Mental Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M14473
- Name: Psychotic Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M28756
- Name: Frontotemporal Lobar Degeneration
- Relevance: LOW
- As Found: Unknown
- ID: M28759
- Name: TDP-43 Proteinopathies
- Relevance: LOW
- As Found: Unknown
- ID: M28747
- Name: Proteostasis Deficiencies
- Relevance: LOW
- As Found: Unknown
- ID: M11639
- Name: Metabolic Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M15864
- Name: Speech Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M10823
- Name: Language Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M6374
- Name: Communication Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M21826
- Name: Neurobehavioral Manifestations
- Relevance: LOW
- As Found: Unknown
- ID: M12404
- Name: Neurologic Manifestations
- Relevance: LOW
- As Found: Unknown
- ID: T2192
- Name: Familial Alzheimer Disease
- Relevance: HIGH
- As Found: Alzheimer's Disease
- ID: T2399
- Name: Frontotemporal Dementia
- Relevance: HIGH
- As Found: Frontotemporal Dementia
- ID: T2400
- Name: Frontotemporal Dementia, Ubiquitin-positive
- Relevance: HIGH
- As Found: Frontotemporal Dementia
- ID: T4710
- Name: Primary Progressive Aphasia
- Relevance: HIGH
- As Found: Frontotemporal Dementia
- ID: T5168
- Name: Semantic Dementia
- Relevance: HIGH
- As Found: Frontotemporal Dementia
- ID: T704
- Name: Behavioral Variant of Frontotemporal Dementia
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000000544
- Term: Alzheimer Disease
- ID: D000003704
- Term: Dementia
- ID: D000057180
- Term: Frontotemporal Dementia
- ID: D000018888
- Term: Aphasia, Primary Progressive
- ID: D000020774
- Term: Pick Disease of the Brain
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT02091479
**Acronym:** RATHER
**Brief Title:** Early Versus Late Resumption of Anticoagulation in Patients With Both High Thrombosis Risk and Major HEmoRrhage
**Official Title:** Randomized Assay Evaluating the Risk/Benefit of Early Versus Late Resumption of Anticoagulation in Patients With Major, Non-trauma Related Hemorrhage Occurring While on Anticoagulant Treatment for a High Risk of Thrombosis.
#### Organization Study ID Info
**ID:** 1108097
#### Organization
**Class:** OTHER
**Full Name:** Centre Hospitalier Universitaire de Saint Etienne
#### Secondary ID Infos
**ID:** 2012-000286-21
**Type:** EUDRACT_NUMBER
### Status Module
#### Completion Date
**Date:** 2015-06
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2016-03-10
**Type:** ESTIMATED
**Last Update Submit Date:** 2016-03-09
**Overall Status:** TERMINATED
#### Primary Completion Date
**Date:** 2015-06
**Type:** ACTUAL
#### Start Date
**Date:** 2013-01
**Status Verified Date:** 2016-03
#### Study First Post Date
**Date:** 2014-03-19
**Type:** ESTIMATED
**Study First Submit Date:** 2014-03-07
**Study First Submit QC Date:** 2014-03-17
**Why Stopped:** inclusion rate insufficient
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Centre Hospitalier Universitaire de Saint Etienne
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** In patients with a high thromboembolic risk, withdrawing anticoagulant treatment is recommended in some situations, including when major hæmorrhage occurs. But withdrawing treatment can be risky. In patients on a curative dose of anticoagulant medicine, treatment withdrawal heightens the risk of thromboembolic events occurring, with potentially major consequences. For instance, mechanical valve thrombosis is fatal in 15% of patients. Resumption of anticoagulation is therefore critical in patients at high risk for thromboembolic events.
However, in these patients having presented major hæmorrhage, resumption of anticoagulation heightens the risk of hæmorrhage recurrence. This risk is even higher when the original hæmorrhage was not accessible via surgical, endoscopic or endoluminal hemostasis.
As far as investigators know, there is no data in the literature to rely on when the major hæmorrhage is not accessible via hemostatic intervention and the risk of thrombosis is high. When confronted with patients who need anticoagulation but have a high risk of hæmorrhage recurrence, the question of when treatment should be resumed has not been resolved. This is why investigators propose to conduct a randomised comparative study to evaluate two treatment strategies - early resumption (H48 to H72) versus late resumption (H120 to H144) of anticoagulation.
MAIN OBJECTIVE: The main objective of the present study is to evaluate in terms of bleeding risk, thrombosis risk and mortality at one month, the effect of early vs. late resumption of anticoagulation in patients having presented with serious hæmorrhage while on curative-dose anticoagulants and facing a high thromboembolic risk.
**Detailed Description:** STUDY DESIGN: This is a comparative, randomised, open study assessing after 1 month and 3 months the effect of early (H48 to 72) versus late (H120 to 144) resumption of anticoagulation in patients presenting with serious bleeding while on anticoagulants (excluding intracerebral bleeding) and with a thromboembolic risk evaluated as high (except mitral prostheses). The accumulated frequency of major hæmorrhage, thromboembolic events and deaths should be 26% in case of early resumption and 15% in case of late resumption, i.e. a relative risk reduction of 43%. Based on this hypothesis, to obtain 80% power with two-sided α being 5%, each group should include 208 patients, for a total of 416 patients.
EVALUATION CRITERIA: The main criteria in this study will be the accumulated one-month incidence of hæmorrhage recurrence, thromboembolic complications and deaths. It is a combined criterion associating:
* Fatal hæmorrhage proven by autopsy or sudden deaths in a clinical context strongly suggestive of hæmorrhage
* Fatal thromboembolic events proven by autopsy or imagery or sudden deaths in a clinical context strongly suggestive of thrombosis
* Any clinically significant hæmorrhage leading to temporary (\> 24 hours) or permanent withdrawal of anticoagulant treatment
* Any symptomatic thromboembolic event in any territory, proven by imagery or surgery
These events will be validated by a committee for the validation of critical events blind to the date of anticoagulant treatment resumption. The secondary evaluation criteria will be symptomatic hæmorrhages, fatal or not, symptomatic thromboembolic incidents, fatal or not, and mortality at 1 month and 3 mont
### Conditions Module
**Conditions:**
- Major hæmorrhage
**Keywords:**
- hæmorrhage, thrombosis
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 14
**Type:** ACTUAL
**Phases:**
- PHASE3
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** anticoagulant (UFH or LMWH) reintroduction at 48h to 72h after hemorrhage
**Intervention Names:**
- Drug: UFH Early group
**Label:** UFH Early group
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** anticoagulant (UFH or LMWH) réintroduction 120h to 144h after hemorrhage
**Intervention Names:**
- Drug: UFH Late group
**Label:** UFH Late group
**Type:** ACTIVE_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- UFH Early group
**Description:** Either curative dose, intravenous unfractionated heparin (UFH) so as to reach an anti-Xa activity between 0.3 and 0.7 IU/mL, lengthening the activated prothrombin time as determined by each centre according to the treatment area (depending on the coagulometer and reactants).
Or low-molecular-weight, curative dose, subcutaneous heparin (LMWH) so as to reach an anti-Xa activity corresponding to treatment areas as determined for each type of molecule (about 0.5 to 1 anti-Xa IU for most LMWHs, administered via 2 daily injections, and about 0.5 to 1.5 anti-Xa IU for tinzaparin, 1 injection daily).
**Name:** UFH Early group
**Type:** DRUG
#### Intervention 2
**Arm Group Labels:**
- UFH Late group
**Description:** Either curative dose, intravenous unfractionated heparin (UFH) so as to reach an anti-Xa activity between 0.3 and 0.7 IU/mL, lengthening the activated prothrombin time as determined by each centre according to the treatment area (depending on the coagulometer and reactants).
Or low-molecular-weight, curative dose, subcutaneous heparin (LMWH) so as to reach an anti-Xa activity corresponding to treatment areas as determined for each type of molecule (about 0.5 to 1 anti-Xa IU for most LMWHs, administered via 2 daily injections, and about 0.5 to 1.5 anti-Xa IU for tinzaparin, 1 injection daily).
**Name:** UFH Late group
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Description:** Fatal hæmorrhage proven by autopsy or sudden deaths in a clinical context strongly suggestive of hæmorrhage,
* Fatal thromboembolic events proven by autopsy or imagery or sudden deaths in a clinical context strongly suggestive of thrombosis,
* Any clinically significant hæmorrhage leading to temporary (\> 24 hours) or permanent withdrawal of anticoagulant treatment,
* Any symptomatic thromboembolic event in any territory, proven by imagery or surgery,
* Any heart valve thrombosis or any intracavitary thrombus spotted by transesophageal echocardiogram performed systematically in patients with mechanical prosthesis or valvular rheumatic heart disease or AF.
**Measure:** The main criterion in this study is the cumulated incidence of mortality, hæmorrhage recurrence and thromboembolic complications at 1 month
**Time Frame:** The main criterion in this study is the cumulated incidence of mortality, hæmorrhage recurrence and thromboembolic complications at 1 month
#### Secondary Outcomes
**Description:** * Fatal hæmorrhages proven by autopsy or imagery or sudden deaths in a clinical context strongly suggestive of hæmorrhage,
* Fatal thromboembolic events proven by autopsy or imagery or sudden deaths in a clinical context strongly suggestive of thrombosis,
* Any clinically significant hæmorrhage leading to temporary (\> 24 hours) or permanent withdrawal of anticoagulant treatment,
* Any symptomatic thromboembolic event in any territory, proven by imagery or surgery,
* Any heart valve thrombosis or any intracavitary thrombus spotted by transesophageal echocardiogram performed systematically in patients with mechanical prosthesis or valvular rheumatic heart disease or AF.
**Measure:** -We will evaluate the risk/benefit balance of early versus late resumption of anticoagulant treatment,
**Time Frame:** We will evaluate at 1 month and at 3 monthsthe risk/benefit balance of early versus late resumption of anticoagulant treatment
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:affiliated with or a beneficiary of a social security category
* age \> 18 years old
* with major bleeding (ISTH) and highs thrombosis risk (ACCP 2008)
* having signed the inform consent form
Exclusion Criteria:
* intracranial bleeding
* artificial heart valves
* bleeding with hemostatic surgical
* low and moderate thrombosis risk
* INR\>1.2
* hemodynamic instability contra-indication to HBPM or HNF treatment
* With previous history of HIT (heparin Inducted thrombopenia)
* patient who need antiaggregant treatment before anticoagulant treatment
* Hæmoglobin count \< 8 g/dl or patients with hæmoglobin count \< 10 g/dl combined with acute coronary syndrome or proven heart failure
* pregnant
* Polytraumatism
* with curatif heparin before randomisation
**Maximum Age:** 90 Years
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Saint-etienne
**Country:** France
**Facility:** CHU de Saint-Etienne
**Zip:** 42000
**Location 2:**
**City:** Saint-Etienne
**Country:** France
**Facility:** Service d'Urgences et de Réanimation Medicale, CHU de Saint-Etienne
**Zip:** 42055
#### Overall Officials
**Official 1:**
**Affiliation:** CHU SAINT ETIENNE
**Name:** Bernard Tardy, MD phD
**Role:** PRINCIPAL_INVESTIGATOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000010335
- Term: Pathologic Processes
- ID: D000016769
- Term: Embolism and Thrombosis
- ID: D000014652
- Term: Vascular Diseases
- ID: D000002318
- Term: Cardiovascular Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: All
- Name: All Conditions
- Abbrev: BC14
- Name: Heart and Blood Diseases
### Condition Browse Module - Browse Leaves
- ID: M9556
- Name: Hemorrhage
- Relevance: HIGH
- As Found: Hemorrhage
- ID: M16686
- Name: Thrombosis
- Relevance: HIGH
- As Found: Thrombosis
- ID: M7784
- Name: Embolism
- Relevance: LOW
- As Found: Unknown
- ID: M19128
- Name: Embolism and Thrombosis
- Relevance: LOW
- As Found: Unknown
- ID: M17400
- Name: Vascular Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000013927
- Term: Thrombosis
- ID: D000006470
- Term: Hemorrhage
### Intervention Browse Module - Browse Branches
- Abbrev: FiAg
- Name: Fibrinolytic Agents
- Abbrev: AnCoag
- Name: Anticoagulants
- Abbrev: All
- Name: All Drugs and Chemicals
- Abbrev: Coag
- Name: Coagulants
### Intervention Browse Module - Browse Leaves
- ID: M9579
- Name: Heparin
- Relevance: LOW
- As Found: Unknown
- ID: M4244
- Name: Anticoagulants
- Relevance: LOW
- As Found: Unknown
- ID: M16676
- Name: Thrombin
- Relevance: LOW
- As Found: Unknown
- ID: M46053
- Name: Calcium heparin
- Relevance: LOW
- As Found: Unknown
- ID: M9581
- Name: Heparin, Low-Molecular-Weight
- Relevance: LOW
- As Found: Unknown
- ID: M1943
- Name: Tinzaparin
- Relevance: LOW
- As Found: Unknown
- ID: M20153
- Name: Dalteparin
- Relevance: LOW
- As Found: Unknown
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT04369079
**Acronym:** MYONAMASTE
**Brief Title:** Myofascial Versus Conventional Physiotherapy
**Official Title:** Myofascial Versus Conventional Physiotherapy in Post-mastectomy Patients: a Randomized Controlled Trial
#### Organization Study ID Info
**ID:** MYONAMASTE
#### Organization
**Class:** OTHER
**Full Name:** The Greater Poland Cancer Centre
### Status Module
#### Completion Date
**Date:** 2014-12-31
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2020-09-02
**Type:** ACTUAL
**Last Update Submit Date:** 2020-08-31
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2014-12-31
**Type:** ACTUAL
#### Start Date
**Date:** 2013-01-01
**Type:** ACTUAL
**Status Verified Date:** 2020-04
#### Study First Post Date
**Date:** 2020-04-30
**Type:** ACTUAL
**Study First Submit Date:** 2020-04-27
**Study First Submit QC Date:** 2020-04-27
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** The Greater Poland Cancer Centre
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** A total of 61 patients who underwent total mastectomy for breast cancer with axillar lymph dissection and adjuvant radiotherapy were enrolled in this trial.
The patients were randomized by toss of a coin to the treatment (n=30) or control (n=26) groups; of these, 48 met all study protocol requirements, including the final assessment. Inclusion criteria were: presence of functional difficulties in the shoulder area and/or upper torso on the surgical side, and 2) eligibility for physiotherapy (determined by the treating physician). Patients with recurrent disease and/or inflammatory or acute ailments were excluded. All patients gave their informed consent to participate in the study.
The treatment group received myofascial treatment while patients in the control group received conventional therapy consisting of exercise and massage.
**Detailed Description:** Treatment duration in both groups was a mean of 4 weeks. Therapy was performed daily excluding weekends and consisted of 45 minutes of individual work with an oncological physiotherapist. In the treatment group, manual myofascial techniques were used to decrease muscle tension and increase elasticity of the soft tissues in the surgical area as well as in tissues that could affect range of motion (ROM) and cause pain. Fascial techniques were used together with the following techniques: deep massage of neck and shoulder girdle muscles; trigger point therapy; tissue scar treatment in the vicinity of the scar and directly on the scar, by stretching, breaking, pulling, as well as static and dynamic rolling; post-isometric relaxation (stretching) of shoulder and neck muscles; active release technique of the chest and shoulder; selected fascial distortion model techniques; and fascial manipulation techniques consisting of developing specific CC-center of coordination and CF-center of fusion points in the operated area and the shoulder on the same side. The exact sequence and number of procedures differed in each patient according to need as determined by prior functional examination. The control group underwent kinesiotherapeutic procedures that included various floor gymnastic exercises with gymnastic stick, balls, and/or elastic tapes, conventional massage of neck and shoulder girdle muscles and therapeutic exercises to increase ROM in the upper limb and in the chest area. Both groups before or after every of the treatment procedure underwent ten-minute manual lymphatic drainage in the limb on the mastectomy side.
Patient interviews and diagnostic examinations were performed at baseline (pre-treatment) and after treatment finalization. The following variables were assessed during the diagnostic examination of the muscle length with Janda's protocol:
Pectoralis Major - pars clavicularis - the normal length of these fibers allows the patient's arm (in an extended position close to the body) to rest below the horizontal.
Pectoralis Major - pars sternocostalis - the normal length of these fibers allows the abducted the patient's arm to 90° to rest below the horizontal.
Pectoralis Major - pars abdomen - the normal length of these pectoral fibers allows the abducted the patient's arm to 150° with slight external rotation to rest in a horizontal position.
Latissimus dorsi - the normal length allows the arm to rest horizontally to the table with the lumbar spine flat on the table.
Descending part of trapezius - the length is assessed qualitatively by noting the end-feel resistance. The normal end feel is gradual rather than abrupt.
Levator scapulae - the length is assessed qualitatively by noting the end-feel resistance. The normal end feel is gradual rather than abrupt.
### Conditions Module
**Conditions:**
- Sports
- Therapy
**Keywords:**
- physiotherapy
- Myofascial
- post-mastectomy patients
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
**Intervention Model Description:** A total of 61 patients who underwent total mastectomy for breast cancer with axillar lymph dissection and adjuvant radiotherapy were enrolled in this trial.
The patients were randomized by toss of a coin to the treatment (n=30) or control (n=26) groups; of these, 48 met all study protocol requirements, including the final assessment. Inclusion criteria were: presence of functional difficulties in the shoulder area and/or upper torso on the surgical side, and 2) eligibility for physiotherapy (determined by the treating physician). Patients with recurrent disease and/or inflammatory or acute ailments were excluded. All patients gave their informed consent to participate in the study.
The treatment group received myofascial treatment while patients in the control group received conventional therapy consisting of exercise and massage.
##### Masking Info
**Masking:** NONE
**Primary Purpose:** SUPPORTIVE_CARE
#### Enrollment Info
**Count:** 61
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Fascial techniques were used together with the following techniques: deep massage of neck and shoulder girdle muscles; trigger point therapy; tissue scar treatment in the vicinity of the scar and directly on the scar, by stretching, breaking, pulling, as well as static and dynamic rolling; post-isometric relaxation (stretching) of shoulder and neck muscles; active release technique of the chest and shoulder; selected fascial distortion model techniques; and fascial manipulation techniques consisting of developing specific CC-center of coordination and CF-center of fusion points in the operated area and the shoulder on the same side. The exact sequence and number of procedures differed in each patient according to need as determined by prior functional examination. Before or after every of the treatment procedure treatment group patients underwent ten-minute manual lymphatic drainage in the limb on the mastectomy side.
**Intervention Names:**
- Other: manual myofascial techniques
**Label:** TREATMENT GROUP
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Treatment duration was a mean of 4 weeks. Therapy was performed daily excluding weekends and consisted of 45 minutes of individual work with an oncological physiotherapist. The control group underwent kinesiotherapeutic procedures that included various floor gymnastic exercises with gymnastic stick, balls, and/or elastic tapes, conventional massage of neck and shoulder girdle muscles and therapeutic exercises to increase ROM in the upper limb and in the chest area. Before or after every of the treatment procedure control group patients underwent ten-minute manual lymphatic drainage in the limb on the mastectomy side.
**Intervention Names:**
- Other: conventional therapy consisting of exercise and massage.
**Label:** CONTROL GROUP
**Type:** OTHER
### Interventions
#### Intervention 1
**Arm Group Labels:**
- TREATMENT GROUP
**Description:** Fascial techniques were used together with the following techniques: deep massage of neck and shoulder girdle muscles; trigger point therapy; tissue scar treatment in the vicinity of the scar and directly on the scar, by stretching, breaking, pulling, as well as static and dynamic rolling; post-isometric relaxation (stretching) of shoulder and neck muscles; active release technique of the chest and shoulder; selected fascial distortion model techniques; and fascial manipulation techniques consisting of developing specific CC-center of coordination and CF-center of fusion points in the operated area and the shoulder on the same side.
**Name:** manual myofascial techniques
**Type:** OTHER
#### Intervention 2
**Arm Group Labels:**
- CONTROL GROUP
**Description:** Underwent kinesiotherapeutic procedures that included various floor gymnastic exercises with gymnastic stick, balls, and/or elastic tapes, conventional massage of neck and shoulder girdle muscles and therapeutic exercises to increase ROM in the upper limb and in the chest area.
**Name:** conventional therapy consisting of exercise and massage.
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Description:** muscle length with Janda's protocol
Pectoralis Major - pars clavicularis - the normal length of these fibers allows the patient's arm (in an extended position close to the body) to rest below the horizontal.
Pectoralis Major - pars sternocostalis - the normal length of these fibers allows the abducted the patient's arm to 90° to rest below the horizontal.
Pectoralis Major - pars abdomen - the normal length of these pectoral fibers allows the abducted the patient's arm to 150° with slight external rotation to rest in a horizontal position.
Latissimus dorsi - the normal length allows the arm to rest horizontally to the table with the lumbar spine flat on the table.
Descending part of trapezius - the length is assessed qualitatively by noting the end-feel resistance. The normal end feel is gradual rather than abrupt.
Levator scapulae - the length is assessed qualitatively by noting the end-feel resistance. The normal end feel is gradual rather than abrupt \[Page et al. 2010\].
**Measure:** muscle length
**Time Frame:** 2 years
#### Secondary Outcomes
**Description:** complete pathological response (pCR, pathologic Complete Response)
**Measure:** Active ROM in the shoulder joint on the surgical side
**Time Frame:** 2 years
**Description:** ROM of the shoulder joint on the surgical side (flexion; extension; abduction; internal rotation; external rotation in horizontal flexion
**Measure:** pathological response
**Time Frame:** 2 years
**Description:** VAS - A tool used to help a person rate the intensity of certain sensations and feelings, such as pain. The visual analog scale for pain is a straight line with one end meaning no pain (0) and the other end meaning the worst pain imaginable (10) . A patient marks a point ( 0 to 10) on the line that matches the amount of pain she feels."
**Measure:** Pain intensity
**Time Frame:** 2 years
**Description:** Made by the centimeter measure as difference of distance from the bone point, to the most limited place in the scar. It was measured in directions: cranial (distance from the ridge of the coracoid) and in the transverse direction (the distance from the xiphoidal process). The centimeter measure was the distance from the bone point (xiphoid process and coracoid), to the most limited place in the scar (which was marked on the patient's examination card so that the measurement could be repeated to the same place
**Measure:** Scar mobility
**Time Frame:** 2 years
**Description:** Was observed presence of the visual dysfunction of scar (retraction) and presence of the axillary web syndrome
**Measure:** Presence of the visual dysfunction of scar (retraction) and presence of the axillary web syndrome
**Time Frame:** 2 years
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* presence of functional difficulties in the shoulder area and/or upper torso on the surgical side
* eligibility for physiotherapy (determined by the treating physician)
* signed informed consent to participate in the study.
Exclusion Criteria:
* patients with recurrent disease and/or inflammatory or acute ailments were excluded.
**Minimum Age:** 18 Years
**Sex:** FEMALE
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Poznań
**Country:** Poland
**Facility:** Greater Poland Cancer Centre
**State:** Wielkopolska
**Zip:** 61-866
#### Overall Officials
**Official 1:**
**Affiliation:** Greater Poland Cancer Centre
**Name:** Sławomir Marszałek, PhD
**Role:** PRINCIPAL_INVESTIGATOR
### IPD Sharing Statement Module
**IPD Sharing:** NO
## Derived Section
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT01525979
**Brief Title:** Comparative Efficacy Research of Uni- vs Bi-lateral Arm Training Poststroke
**Official Title:** Comparative Efficacy Research of Uni- vs Bi-lateral Arm Training Poststroke: Task-Related or Robotic
#### Organization Study ID Info
**ID:** 99-0832B
#### Organization
**Class:** OTHER
**Full Name:** Chang Gung Memorial Hospital
#### Secondary ID Infos
**Domain:** National Health Research Institutes
**ID:** NHRI-EX100-10010PI
**Type:** OTHER_GRANT
### Status Module
#### Completion Date
**Date:** 2015-04
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2015-06-08
**Type:** ESTIMATED
**Last Update Submit Date:** 2015-06-04
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2015-04
**Type:** ACTUAL
#### Start Date
**Date:** 2011-01
**Status Verified Date:** 2015-06
#### Study First Post Date
**Date:** 2012-02-03
**Type:** ESTIMATED
**Study First Submit Date:** 2012-01-30
**Study First Submit QC Date:** 2012-02-01
### Sponsor Collaborators Module
#### Collaborators
**Class:** OTHER_GOV
**Name:** National Science Council, Taiwan
**Class:** OTHER
**Name:** National Health Research Institutes, Taiwan
#### Lead Sponsor
**Class:** OTHER
**Name:** Chang Gung Memorial Hospital
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** This proposed project aims to
* compare the effects of dose-matched unilateral vs bilateral vs unilateral combined with bilateral upper limb rehabilitation based on task-related practice
* compare the effects of unilateral vs bilateral training based on robot-assisted devices
* study the predictors of treatment outcomes and clinimetric properties of the biomechanical measures
**Detailed Description:** An estimated 30% to 66 % of stroke victims have poor upper extremity function at 6 months after stroke, which contributes to long-term disability in these patients. Training the affected UE of stroke patients has thus been a mainstay of neurorehabilitation. The goal of the intervention is to restore motor and daily functions. New therapeutic strategies have been developed based on principles of neurorehabilitation and motor learning. The need for comparative effectiveness research of the innovative treatments has been called for to promote evidence-based practice and translational science in stroke motor rehabilitation. Wolf and Whitall called for rigorous comparisons between unilateral and bilateral training. McCombe Waller and Whitall also indicated the importance of combining unilateral with bilateral training. For low-functioning patients, robot-assisted training is a possible approach. Identifying possible determinants for unilateral and bilateral training outcomes may also elucidate the factors that influence treatment outcomes. Outcome measures are required that have good clinimetric properties for measuring the effects of the rehabilitation strategies.
The long-term objective is to conduct comparative efficacy research to identify the possible mechanisms and the relative effectiveness of existing rehabilitation approaches of fully defined-, evidence-based, and theory-grounded approaches. This proposed project aims to (1) compare the effects of dose-matched unilateral vs bilateral vs unilateral combined with bilateral upper limb rehabilitation based on task-related practice; (2) compare the effects of unilateral vs bilateral training based on robot-assisted devices; and (3) study the predictors of treatment outcomes and clinimetric properties of the biomechanical measures. The outcome measures will span the spectrum of health-related functioning, including motor and neural control, movement performance, daily functions, and quality of life. Motor and neural control mechanisms involving movement strategies, muscle and force output, and brain reorganization will be evaluated using kinematic, electromyographic (EMG), and kinetic analyses, and also functional magnetic resonance imaging (fMRI) examinations. Clinical outcomes will include movement performance, daily functions, and quality of life, measured by MYOTON-3, Modified Ashworth Scale, the Fugl-Meyer Assessment, Wolf Motor Function Test, Functional Independence Measure, ABILHAND questionnaire, accelerometers, and Stroke Impact Scale. This 5-year project will recruit an estimated 200 patients with stroke. For Part 1, 120 patients with mild-to-moderate motor impairment will be randomized to the dose-matched unilateral, bilateral, or combined group. For Part 1, 80 stroke patients with moderate motor impairment will be randomized to the dose-matched unilateral or bilateral robot-assisted therapy group. Treatment regimens will be designed to ensure that patients in the 3 groups in Part 1 and 2 groups in Part 2 receive an equivalent intensity of treatment (5 days/week for 1.5 hours/day for 4 consecutive weeks). The intervention will be provided at 4 hospitals. Two certified occupational therapists will be trained in the administration of the 5 types of rehabilitation protocols by the PI and a co-PI and will complete a written competency test before subject treatment.
Biomechanical (kinematic, EMG, and kinetic) and fMRI examinations will be performed before, immediately after the 4-week intervention period, and at the 3-month follow-up period (no fMRI examination at follow-up). Clinical measures will be administered before, at midterm (2 weeks after intervention), immediately after, and 3 months after the intervention. At least 3 examiners (1 research assistant, 1 graduate student, and 1 post-doc fellow) blind to group allocation will be in charge of biomechanical, fMRI, and clinical measures. Each type of measure will require 2 evaluators to collaborate for completing the assessment. Before being allowed to work with participants, the examiner's competence will be assessed by the PI and co-PI, and interrater reliability for clinical tests established.
Multivariate analysis of covariance will be used to examine changes in all outcome measures as a function of treatment while controlling for baseline data. Multiple regression models will be established to determine the potential predictors for different functional outcomes of each intervention (unilateral and bilateral training approach). The responsiveness and validity of the biomechanical measures relevant for clinimetric scrutiny will be examined by the index of standardized response mean and Spearman correlation coefficients. The clinical important differences of the biomechanical parameters will be determined by the anchor-based and the distribution-based clinical important differences estimate.
### Conditions Module
**Conditions:**
- Cerebrovascular Accident
**Keywords:**
- Stroke rehabilitation
- Unilateral arm training
- Bilateral arm training
- Robot-assisted arm training
- Kinematic analysis
- Functional magnetic resonance image
- Motor recovery
- Quality of life
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** FACTORIAL
##### Masking Info
**Masking:** SINGLE
**Who Masked:**
- OUTCOMES_ASSESSOR
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 42
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Therapist conducted unilateral arm training Task-related unilateral arm training
**Intervention Names:**
- Behavioral: Task-related unilateral arm training
**Label:** Task-Related UAT
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Therapist conducted bilateral arm Training Task-related bilateral arm training
**Intervention Names:**
- Behavioral: Task-related bilateral arm training
**Label:** Task-Related BAT
**Type:** EXPERIMENTAL
#### Arm Group 3
**Description:** Therapist conducted task-related unilateral training for 45 minutes, followed by task-related bilateral arm training for another 45 minutes during each training session
**Intervention Names:**
- Behavioral: Task-related unilateral arm training
- Behavioral: Task-related bilateral arm training
**Label:** Task-Related UAT coupling BAT
**Type:** EXPERIMENTAL
#### Arm Group 4
**Description:** Robot-assisted unilateral arm training
**Intervention Names:**
- Behavioral: Robot-assisted unilateral arm training
**Label:** Robot-assisted UAT
**Type:** EXPERIMENTAL
#### Arm Group 5
**Description:** Robot-assisted bilateral arm training
**Intervention Names:**
- Behavioral: Robot-assisted bilateral arm training
**Label:** Robot-assisted BAT
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Task-Related UAT
- Task-Related UAT coupling BAT
**Description:** The training tasks will involve daily activities with unilateral proximal or distal upper extremity movements for 5 days/week for 1.5 hours/day for 4 consecutive weeks.
**Name:** Task-related unilateral arm training
**Other Names:**
- Task-related UAT
**Type:** BEHAVIORAL
#### Intervention 2
**Arm Group Labels:**
- Task-Related BAT
- Task-Related UAT coupling BAT
**Description:** This intervention emphasizes UE movements (gross or fine motor tasks) involved in daily activities but focus on both UEs moving synchronously. The duration and intensity of treatment will also be 5 days/week for 1.5 hours/day for 4 consecutive weeks.
**Name:** Task-related bilateral arm training
**Other Names:**
- Task-related BAT
**Type:** BEHAVIORAL
#### Intervention 3
**Arm Group Labels:**
- Robot-assisted BAT
**Description:** The Bi-Manu-Track used in this project (Reha-Stim Co., Berlin, Germany) enables the symmetric practice of 2 movement patterns: forearm pronation-supination and wrist flexion-extension. Each movement has 3 computer-controlled modes: (1) passive-passive, with both arms being moved by the machine with speed, range of motion, and resistance individually adjustable; (2) active-passive, with the unaffected arm driving the affected arm in a mirror-like fashion; and (3) active-active, with both arms actively moving against resistance.
**Name:** Robot-assisted bilateral arm training
**Other Names:**
- Robot-assisted BAT
**Type:** BEHAVIORAL
#### Intervention 4
**Arm Group Labels:**
- Robot-assisted UAT
**Description:** The robot-assisted unilateral arm training group will use mode 1 and 3: (1) passive, affected arm being moved by the machine with speed and range of motion individually adjustable; and (2) active, with the affected arm actively moving. This training program will add an additional mode: active-resistance, with the affected arm/wrist actively moving against resistance.
**Name:** Robot-assisted unilateral arm training
**Other Names:**
- Robot-assisted UAT
**Type:** BEHAVIORAL
### Outcomes Module
#### Primary Outcomes
**Description:** The movement time is collected by a 7-camera motion analysis system (VICON MX 3-D, Oxford Metrics Inc., Oxford, UK) from unilateral and bilateral reaching tasks of pressing the desk bell. MT is the interval between movement onset and offset.
**Measure:** Movement time
**Time Frame:** Baseline and change from baseline in movement time at 4 weeks
**Description:** The movement time is collected by a 7-camera motion analysis system (VICON MX 3-D, Oxford Metrics Inc., Oxford, UK) from unilateral and bilateral reaching tasks of pressing the desk bell. One MU consists of 1 acceleration and 1 deceleration phase of reaching and can be used to characterize movement smoothness.
**Measure:** Motor units
**Time Frame:** Baseline and change from baseline in motor units at 4 weeks
**Description:** The movement time is collected by a 7-camera motion analysis system (VICON MX 3-D, Oxford Metrics Inc., Oxford, UK) from unilateral and bilateral reaching tasks of pressing the desk bell. The angular degree of elbow extension will be calculated by subtracting the angle at the start of the movement from the angle at the end of the movement
**Measure:** Elbow extension angle
**Time Frame:** Baseline and change from baseline in elbow extension angle at 4 weeks
**Description:** The fMRI will be performed on a 3T Magnetom Vision MRI scanner (Siemens, Erlangen, Germany) before and after intervention. The change of Blood oxygenation level-dependent (BOLD) functional images in regions-of-interest will be collected. The laterality index (LI) will be calculated to provide an estimation of the relative hemispheric activation.
**Measure:** Lateral index
**Time Frame:** Baseline and change from baseline in lateral index at 4 weeks
#### Secondary Outcomes
**Description:** The 2 force plates is adopted to obtain the center of pressure (CoP) displacement representing balance performance during standing reaching.
**Measure:** Test of center of pressure (CoP) displacement
**Time Frame:** Baseline and change from baseline in test of CoP displacement at 4 weeks
**Description:** Hand-held dynamometers will be the devices that measure the hand strength.
**Measure:** Hand strength
**Time Frame:** Baseline and change from baseline in hand strength at 4 weeks
**Description:** Raw EMG activities from the muscles of interest during unilateral and bilateral reaching while the patient is sitting and standing will be recorded using the AcqKnowledge data analysis software.
**Measure:** Electromyography (EMG)
**Time Frame:** Baseline and change from baseline in EMG at 4 weeks
**Description:** The MAS scale is an ordinal scale (0-5 points) assessing spasticity of skeletal muscle in UE. A higher score indicating more spasticity.
**Measure:** Modified Ashworth Scale (MAS)
**Time Frame:** Baseline, change from baseline in MAS at 2 weeks, and change from baseline in muscle tone at 4 weeks
**Description:** The UE subscale of the FMA (max. score 66) uses a 3-point ordinal scale to assess motor impairment.
**Measure:** Fugl-Meyer Assessment (FMA)
**Time Frame:** Baseline, change from baseline in FMA at 2 weeks, and change from baseline in FMA at 4 weeks
**Description:** The WMFT is a function-based motor assessment of 17 tasks, including 15 timed and functional ability tasks (fine and gross motor tasks) and 2 strength tasks (lifting and handgrip).
**Measure:** Wolf Motor Function Test (WMFT)
**Time Frame:** Baseline, change from baseline in WMFT at 2 weeks, and change from baseline in WMFT at 4 weeks
**Description:** The FIM consists of 18 items grouped into 6 subscales. Each item is rated from 1 to 7 (max. score 126) based on the required level of assistance to perform the tasks.
**Measure:** Functional Independence Measure (FIM)
**Time Frame:** Baseline, change from baseline in FIM at 2 weeks, and change from baseline in FIM at 4 weeks
**Description:** ABILHAND questionnaire is an inventory of 56 manual activities that uses a 3-point ordinal scale to measure subjectively perceived difficulty in performing everyday bimanual activity.
**Measure:** ABILHAND Questionnaire
**Time Frame:** Baseline, change from baseline in ABILHAND Questionnaire at 2 weeks, and change from baseline in ABILHAND Questionnaire at 4 weeks
**Description:** Accelerometers quantitatively record the amount of activity in free-living conditions and will be used to reflect the amount of affected arm use over time.
**Measure:** Accelerometers
**Time Frame:** Baseline, change from baseline in accelerometers data at 2 weeks, and change from baseline in accelerometers data at 4 weeks
**Description:** The SIS 3.0 contains 59 items measuring 8 domains. Items are rated on a 5-point Likert scale with lower scores indicating greater difficulty in task completion during the past week.
**Measure:** Stroke Impact Scale Version 3.0 (SIS 3.0)
**Time Frame:** Baseline, change from baseline in SIS 3.0 at 2 weeks, and change from baseline in SIS 3.0 at 4 weeks
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* 40 to 75 years old
* 6 to 24 months after stroke onset from a first-ever unilateral stroke
* An initial 26 to 56 scores on the UE subsection of the
* Premorbid right-hand dominance evaluated by the Edinburgh Handedness Inventory
* No excessive spasticity in the shoulder and elbow joints of the affected UE (Modified Ashworth Scale score ≤ 3 in each joint)
* Sufficient cognitive ability, defined as a score of more than 24 on the Mini Mental State Examination
Exclusion Criteria:
* Physician-determined major medical problems or poor physical condition that would interfere with participation
* Excessive pain in any joint that might limit participation
**Maximum Age:** 75 Years
**Minimum Age:** 40 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Taoyuan County
**Country:** Taiwan
**Facility:** Chang Gung Memorial Hospital
#### Overall Officials
**Official 1:**
**Affiliation:** Chang Gung University
**Name:** Ching-yi Wu, ScD
**Role:** PRINCIPAL_INVESTIGATOR
### References Module
#### References
**Citation:** Wu CY, Yang CL, Chuang LL, Lin KC, Chen HC, Chen MD, Huang WC. Effect of therapist-based versus robot-assisted bilateral arm training on motor control, functional performance, and quality of life after chronic stroke: a clinical trial. Phys Ther. 2012 Aug;92(8):1006-16. doi: 10.2522/ptj.20110282. Epub 2012 Apr 19.
**PMID:** 22517782
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000002561
- Term: Cerebrovascular Disorders
- ID: D000001927
- Term: Brain Diseases
- ID: D000002493
- Term: Central Nervous System Diseases
- ID: D000009422
- Term: Nervous System Diseases
- ID: D000014652
- Term: Vascular Diseases
- ID: D000002318
- Term: Cardiovascular Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC10
- Name: Nervous System Diseases
- Abbrev: BC14
- Name: Heart and Blood Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BXM
- Name: Behaviors and Mental Disorders
### Condition Browse Module - Browse Leaves
- ID: M22306
- Name: Stroke
- Relevance: HIGH
- As Found: Cerebrovascular Accident
- ID: M5810
- Name: Cerebrovascular Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M5204
- Name: Brain Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5742
- Name: Central Nervous System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M17400
- Name: Vascular Diseases
- Relevance: LOW
- As Found: Unknown
- ID: T6034
- Name: Quality of Life
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000020521
- Term: Stroke
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT01676779
**Acronym:** DC-MEL
**Brief Title:** mRNA Electroporated Autologous Dendritic Cells for Stage III/IV Melanoma
**Official Title:** Randomized Controlled Phase II Clinical Trial on mRNA Electroporated Autologous Dendritic Cells for Stage III/IV Melanoma Patients Who Are Disease-free Following the Local Treatment of Macrometastases.
#### Organization Study ID Info
**ID:** DC-MEL
#### Organization
**Class:** OTHER
**Full Name:** Universitair Ziekenhuis Brussel
### Status Module
#### Completion Date
**Date:** 2016-09
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2017-05-24
**Type:** ACTUAL
**Last Update Submit Date:** 2017-05-23
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2015-09
**Type:** ACTUAL
#### Start Date
**Date:** 2012-10
**Type:** ACTUAL
**Status Verified Date:** 2017-05
#### Study First Post Date
**Date:** 2012-08-31
**Type:** ESTIMATED
**Study First Submit Date:** 2012-08-21
**Study First Submit QC Date:** 2012-08-30
### Sponsor Collaborators Module
#### Collaborators
**Class:** UNKNOWN
**Name:** RIZIV
#### Lead Sponsor
**Class:** OTHER
**Name:** Universitair Ziekenhuis Brussel
#### Responsible Party
**Investigator Affiliation:** Universitair Ziekenhuis Brussel
**Investigator Full Name:** Bart Neyns
**Investigator Title:** Head of devision (Medical Oncology)
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** This is an open label, 2-arm, 1-stage, randomized controlled phase II study in patients with AJCC stage IIIB/C \& -IV melanoma. At baseline tumor assessment (using total body FDG-PET/CT), patients should be free from measurable tumor lesions (according to RECISTv1.1 definitions) following prior local therapy (e.g. following surgical resection, isolated limb perfusion, radiofrequency ablation, cryotherapy, radiotherapy, electrochemotherapy, ...). Patients should not have symptomatic non-measurable tumor lesions (e.g. bone metastasis, or pleural effusion), and lesions treated by prior local therapy should be free from progression. Patients should not have received any prior systemic therapy (non-experimental or experimental).
**Detailed Description:** This is an open label, 2-arm, 1-stage, randomized controlled phase II study in patients with AJCC stage IIIB/C \& -IV melanoma. At baseline tumor assessment (using total body FDG-PET/CT), patients should be free from measurable tumor lesions (according to RECISTv1.1 definitions) following prior local therapy (e.g. following surgical resection, isolated limb perfusion, radiofrequency ablation, cryotherapy, radiotherapy, electrochemotherapy, ...). Patients should not have symptomatic non-measurable tumor lesions (e.g. bone metastasis, or pleural effusion), and lesions treated by prior local therapy should be free from progression. Patients should not have received any prior systemic therapy (non-experimental or experimental).
* Patients will be randomized between two treatment arms (Arm-A and -B). In study Arm-A, patients will receive DC-administrations during one year following randomization. Salvage treatment by local therapies will be allowed during the study treatment in Arm-A. In study Arm-B, patients will initiate DC-administrations only after documented recurrence of the melanoma that cannot be salvaged by local therapy.
* The primary endpoint of this clinical trial is to determine the rate (%) of patients who are free from macrometastases (: measurable tumor lesions and symptomatic non-measurable tumor lesions) at 1-year (= 52 weeks) after randomization.
Patients treated on Arm-B will serve as a contemporary control-arm to help interpreting the outcome of patients treated in Arm-A. By design (phase II) this trial will not be powered to statistically prove a predefined difference between the two study arms (this would require a phase III design). Patients treated in Arm-B will be able to initiate immunotherapy with autologous DC at the time of recurrence that can not be salvaged by local therapy. Documentation of the anti-tumor activity and survival following DC-treatment at recurrence in Arm-B patients will be a secondary objective of this clinical trial.
### Conditions Module
**Conditions:**
- Malignant Melanoma Stage III
- Malignant Melanoma Stage IV
**Keywords:**
- melanoma
- no evidence of disease
- resection
- macro-metastases
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 88
**Type:** ESTIMATED
**Phases:**
- PHASE2
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Arm-A, patients will receive Dendritic Cell therapy during one year following randomization.
**Intervention Names:**
- Biological: Dendritic cell therapy
**Label:** Arm A dendritic cell therapy
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Arm-B, patients will initiate Dendritic Cell therapy only after documented recurrence of the melanoma that cannot be salvaged by local therapy.
**Intervention Names:**
- Biological: Dendritic cell therapy
**Label:** Arm B Dendritic cell therapy
**Type:** EXPERIMENTAL
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Arm A dendritic cell therapy
- Arm B Dendritic cell therapy
**Description:** Dendritic cell therapy IV and ID
**Name:** Dendritic cell therapy
**Type:** BIOLOGICAL
### Outcomes Module
#### Primary Outcomes
**Description:** Patients will be evaluated 52 weeks following randomization for their melanoma disease status (= macro-metastases present or absent)
**Measure:** 1-year disease free survival percentage
**Time Frame:** 1-year following recruitment date
#### Secondary Outcomes
**Description:** Patients will be followed continuous during their study participation for adverse events
**Measure:** safety
**Time Frame:** continuous during the study (52weeks after start)
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
1. Able and willing to give written informed consent
2. Histological documentation of AJCC stage III or stage IV melanoma
3. melanoma (melanoma originating in the choroid, iris or ciliar body are not eligible)
4. baseline tumor assessment by whole-body FDG-PET/CT, patients should be free from measurable tumor lesions (RECIST (v1.1)), and free from symptomatic non-measurable tumor lesions
5. Prior local treatment of primary and metastatic tumor lesions is allowed . Treated tumor lesions should be free from progression at baseline assessment
6. Normal organ function and normal hematological parameters;laboratory parameters should be within normal range, except following laboratory parameters:HEMOGLOBIN ≥ 10 G/DL; GRANULOCYTES ≥ 1,500/µL; LYMPHOCYTES ≥ 1000/µL; PLATELETS ≥ 100,000/µL; SERUM CREATININ ≤ 2.0 MG/DL; SERUM BILIRUBIN ≤ 2.0 MG/DL; AST AND ALT ≤ 2 X THE NORMAL UPPER LIMITS; LDH ≤ 1,5X NORMAL UPPER LIMIT; CRP ≤ 1,5X NORMAL UPPER LIMIT; PROTHROMBIN TIME (PT) INTERNATIONAL NORMALIZED RATIO (INR) AND PARTIAL THROMBOPLASTIN TIME (PTT) WITHIN NORMAL LIMITS
7. Negative serology for HCV, and HIV; absence of active infection with HBV, and Syphilis; If positive results for HepB or Syphilis indicate immunity and are not indicative of active infection, the patient can enter the study.
8. Adequate venous access(to undergo leukapheresis)
9. No prior systemic therapy for melanoma
10. Full recovery from all prior therapies. A period of 4 weeks following major surgery, radiation therapy, or ILP, or any other major invasive procedure is required
11. Baseline WHO performance status of 0 or 1
12. Male and female patients ≥ 18 years
13. No need for uninterrupted therapeutic anticoagulation
14. No prior history of a serious autoimmune disorder
15. No concomitant medication with immune suppressive drugs
16. Women of childbearing potential must be using an adequate method of contraception to avoid pregnancy throughout the study and for up to 8 weeks after the study in such a manner that the risk of pregnancy is minimized.
Exclusion Criteria:
1. Evidence of immunodeficiency or autoimmune disease requiring medical treatment (e.g. corticosteroids or other immunosuppressive drugs).Vitiligo is not an exclusion criterion
2. Any serious acute or chronic illnesses (e.g. heart disease NYHA Class III or IV,renal-,liver- or pulmonary insufficiency) or other conditions requiring concurrent medications not allowed during this study (e.g. active chronic infections requiring antibiotics)
3. History of malignancy. Curatively treated cervical carcinoma in situ,or squamous-,or basal cell carcinoma of the skin, or subjects who have been treated and recurrence-free of other malignancies for more than 5 years following the diagnosis are eligible
4. Inability to undergo FDG-PET/CT, or MRI examination
5. Mental impairment that may compromise the ability to give informed consent and comply with the requirements of the study
6. Participation in any other clinical trial involving another investigational agent within 4 weeks prior to enrollment
7. Subject is pregnant (positive serum beta-HCG test at screening) or is currently breast-feeding, anticipates becoming pregnant/impregnating their partner during the study or within 6 months after study participation, or subject does not agree to follow acceptable methods of birth control, to avoid conception during the study and for at least 6 months after receiving the last dose of study treatment
8. Current alcohol dependence or drug abuse
9. Known hypersensitivity to the study treatment
10. Legal incapacity or limited legal capacity
11. Presence of any psychological, familial, sociological, or geographical condition potentially hampering compliance with the study protocol and follow-up schedule.
12. Signs and symptoms suggestive of transmissible spongiform encephalopathy,or family members who suffer(ed) from such.
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Brussel
**Country:** Belgium
**Facility:** UZ Brussel
**Zip:** 1090
#### Overall Officials
**Official 1:**
**Affiliation:** Universitair Ziekenhuis Brussel
**Name:** Bart Neyns, Phd Md
**Role:** PRINCIPAL_INVESTIGATOR
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000018358
- Term: Neuroendocrine Tumors
- ID: D000017599
- Term: Neuroectodermal Tumors
- ID: D000009373
- Term: Neoplasms, Germ Cell and Embryonal
- ID: D000009370
- Term: Neoplasms by Histologic Type
- ID: D000009369
- Term: Neoplasms
- ID: D000009380
- Term: Neoplasms, Nerve Tissue
- ID: D000018326
- Term: Nevi and Melanomas
- ID: D000012878
- Term: Skin Neoplasms
- ID: D000009371
- Term: Neoplasms by Site
- ID: D000012871
- Term: Skin Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC04
- Name: Neoplasms
- Abbrev: BC17
- Name: Skin and Connective Tissue Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M11528
- Name: Melanoma
- Relevance: HIGH
- As Found: Melanoma
- ID: M12307
- Name: Neoplasm Metastasis
- Relevance: LOW
- As Found: Unknown
- ID: M20495
- Name: Neuroendocrine Tumors
- Relevance: LOW
- As Found: Unknown
- ID: M19845
- Name: Neuroectodermal Tumors
- Relevance: LOW
- As Found: Unknown
- ID: M20388
- Name: Neuroectodermal Tumors, Primitive
- Relevance: LOW
- As Found: Unknown
- ID: M12318
- Name: Neoplasms, Germ Cell and Embryonal
- Relevance: LOW
- As Found: Unknown
- ID: M12315
- Name: Neoplasms by Histologic Type
- Relevance: LOW
- As Found: Unknown
- ID: M12325
- Name: Neoplasms, Nerve Tissue
- Relevance: LOW
- As Found: Unknown
- ID: M12448
- Name: Nevus, Pigmented
- Relevance: LOW
- As Found: Unknown
- ID: M12446
- Name: Nevus
- Relevance: LOW
- As Found: Unknown
- ID: M20470
- Name: Nevi and Melanomas
- Relevance: LOW
- As Found: Unknown
- ID: M15681
- Name: Skin Neoplasms
- Relevance: LOW
- As Found: Unknown
- ID: M15674
- Name: Skin Diseases
- Relevance: LOW
- As Found: Unknown
- ID: T4091
- Name: Neuroendocrine Tumor
- Relevance: LOW
- As Found: Unknown
- ID: T4092
- Name: Neuroepithelioma
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000008545
- Term: Melanoma
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT02472379
**Brief Title:** Writing About Experiences and Health in Older Adults
**Official Title:** Writing About Experiences and Health in Older Adults
#### Organization Study ID Info
**ID:** GEN
#### Organization
**Class:** OTHER
**Full Name:** University of California, Los Angeles
#### Secondary ID Infos
**ID:** F31AG048668
**Link:** https://reporter.nih.gov/quickSearch/F31AG048668
**Type:** NIH
**ID:** UL1TR000124
**Link:** https://reporter.nih.gov/quickSearch/UL1TR000124
**Type:** NIH
**ID:** R03AG049254
**Link:** https://reporter.nih.gov/quickSearch/R03AG049254
**Type:** NIH
### Status Module
#### Completion Date
**Date:** 2017-03
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2017-05-02
**Type:** ACTUAL
**Last Update Submit Date:** 2017-05-01
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2017-01
**Type:** ACTUAL
#### Start Date
**Date:** 2015-10
**Status Verified Date:** 2017-05
#### Study First Post Date
**Date:** 2015-06-15
**Type:** ESTIMATED
**Study First Submit Date:** 2015-06-10
**Study First Submit QC Date:** 2015-06-12
### Sponsor Collaborators Module
#### Collaborators
**Class:** NIH
**Name:** National Institute on Aging (NIA)
#### Lead Sponsor
**Class:** OTHER
**Name:** University of California, Los Angeles
#### Responsible Party
**Investigator Affiliation:** University of California, Los Angeles
**Investigator Full Name:** Naomi Eisenberger
**Investigator Title:** Associate Professor
**Type:** PRINCIPAL_INVESTIGATOR
### Description Module
**Brief Summary:** UCLA researchers looking for healthy females (age 60+) to participate in a study investigating how writing about your experiences may be related to your health.
Once a week for 6 weeks, participants will write about their experiences and fill out online questionnaires. Participants will also come to the UCLA campus for blood draws and to fill out questionnaires 3 times: once prior to the 6-week writing period, once immediately after the 6-week period, and once 2-months after the writing period.
### Conditions Module
**Conditions:**
- Social Psychology
**Keywords:**
- Immune System
- Behavioral Sciences
- Women's Health
- Health Psychology
- Writing
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** DOUBLE
**Who Masked:**
- PARTICIPANT
- INVESTIGATOR
**Primary Purpose:** BASIC_SCIENCE
#### Enrollment Info
**Count:** 78
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Subjects will be asked to write about experiences with familiar individuals in their lives.
**Intervention Names:**
- Behavioral: Writing: Group 1
**Label:** Writing: Group 1
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Subjects will be asked to write about experiences with familiar places in their lives.
**Intervention Names:**
- Other: Writing: Group 2
**Label:** Writing: Group 2
**Type:** PLACEBO_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Writing: Group 1
**Description:** Writing about experiences with familiar individuals in their lives, once a week for 6 weeks.
**Name:** Writing: Group 1
**Type:** BEHAVIORAL
#### Intervention 2
**Arm Group Labels:**
- Writing: Group 2
**Description:** Writing about experiences with familiar places, once a week for 6 weeks.
**Name:** Writing: Group 2
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Description:** Changes in proinflammatory cytokine and proinflammatory gene expression from blood draws
**Measure:** Inflammatory activity
**Time Frame:** baseline, post 6-week writing period, 2-months post-writing period
**Description:** Changes in self-report measures of health (e.g., pain, sleep) and well-being (e.g., social support, depression)
**Measure:** Health and well-being (self-report)
**Time Frame:** baseline, post 6-week writing period, 2-months post-writing period
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Healthy adults 60 years and older
Exclusion Criteria:
* Following a structured telephone interview, participants with the following conditions will not be able to participate:
1. current smokers
2. active, uncontrolled medical disorders
3. chronic infection (e.g., Hepatitis C, HIV)
4. use of certain medications (e.g., hypnotic and psychotropic medication, steroid use, opioid use)
5. psychiatric disorders (e.g., current major depression, bipolar disorder)
6. body mass index (BMI) greater than 35
Other exclusion criteria may apply.
**Healthy Volunteers:** True
**Maximum Age:** 100 Years
**Minimum Age:** 60 Years
**Sex:** FEMALE
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Los Angeles
**Country:** United States
**Facility:** UCLA Clinical & Translational Research Center (CTRC)
**State:** California
#### Overall Officials
**Official 1:**
**Affiliation:** University of California, Los Angeles
**Name:** Naomi I. Eisenberger, Ph.D.
**Role:** PRINCIPAL_INVESTIGATOR
**Official 2:**
**Affiliation:** University of California, Los Angeles
**Name:** Mona Moieni, M.A.
**Role:** PRINCIPAL_INVESTIGATOR
## Derived Section
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT00184379
**Brief Title:** Effects of Psychoeducation of Relatives to Patients With Serious Mental Illness
**Official Title:** Effects of Psychoeducation of Relatives to Patients With Serious Mental Illness
#### Organization Study ID Info
**ID:** 4.2005.389
#### Organization
**Class:** OTHER
**Full Name:** Norwegian University of Science and Technology
### Status Module
#### Completion Date
**Date:** 2010-06
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2017-08-28
**Type:** ACTUAL
**Last Update Submit Date:** 2017-08-24
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2010-06
**Type:** ACTUAL
#### Start Date
**Date:** 2005-04
**Status Verified Date:** 2017-08
#### Study First Post Date
**Date:** 2005-09-16
**Type:** ESTIMATED
**Study First Submit Date:** 2005-09-13
**Study First Submit QC Date:** 2005-09-15
### Sponsor Collaborators Module
#### Collaborators
**Class:** OTHER
**Name:** St. Olavs Hospital
#### Lead Sponsor
**Class:** OTHER
**Name:** Norwegian University of Science and Technology
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** The purpose of this study is to determine the effects of psychoeducation for relatives to patients with serious mental illness.
**Detailed Description:** The main aim of the study is to compare the effects of group psychoeducation for relatives to patients with serious mental illness with a waiting list control group. The main hypothesis is whether psychoeducation in groups has effects on the stress experience and coping style in the relatives. The relatives will receive group education in 6 weekly sessions. We will measure stress experience, coping style and the relatives' health condition before the sessions, after the sessions and at one year follow-up.
### Conditions Module
**Conditions:**
- Mental Disorder
- Depressive Disorder
- Schizophrenia
**Keywords:**
- Family
- Coping Skills
- Psychoeducation
- Stress, Psychological
- Health Education
### Design Module
#### Design Info
**Allocation:** NON_RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 50
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Relatives of patients with schizophrenia, who receive education
**Intervention Names:**
- Behavioral: Psychoeducation
**Label:** 1 S+E
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Relatives of patients with schizophrenia, who do not receive education
**Label:** 2 S-E
**Type:** NO_INTERVENTION
#### Arm Group 3
**Description:** Relatives of patients with bipolar disorder, who receive education
**Intervention Names:**
- Behavioral: Psychoeducation
**Label:** 3 B+E
**Type:** EXPERIMENTAL
#### Arm Group 4
**Description:** Relatives of patients with bipolar disorder, who do not receive education
**Label:** 4 B-E
**Type:** NO_INTERVENTION
### Interventions
#### Intervention 1
**Arm Group Labels:**
- 1 S+E
- 3 B+E
**Description:** Structured disorder-related educational program for relatives
**Name:** Psychoeducation
**Type:** BEHAVIORAL
### Outcomes Module
#### Primary Outcomes
**Measure:** ECI (Experience of Caregiving Inventory) (Smuckler et al,1994)
**Time Frame:** 0-6 months
**Measure:** COOP/WONCA (Bowling, 1995)
**Time Frame:** 0-6 months
**Measure:** Selection of questions from COPE (Carver et al. 1989)
**Time Frame:** 0-6 months
**Measure:** Evaluation-questions
**Time Frame:** 0-6 months
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Relatives to patients having a psychotic or bipolar disorder
* Good Norwegian language skills
* Consent to participate
* The patients' consent for their relatives to participate
**Maximum Age:** 70 Years
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Trondheim
**Country:** Norway
**Facility:** Østmarka Psychiatric Department, St. Olavs Hospital, University Hospital of Trondheim
#### Overall Officials
**Official 1:**
**Affiliation:** Norwegian University of Science and Technology
**Name:** Olav M Linaker, MD Prof
**Role:** PRINCIPAL_INVESTIGATOR
### References Module
#### References
**Citation:** Moller T, Gudde CB, Folden GE, Linaker OM. The experience of caring in relatives to patients with serious mental illness: gender differences, health and functioning. Scand J Caring Sci. 2009 Mar;23(1):153-60. doi: 10.1111/j.1471-6712.2008.00605.x. Epub 2009 Jan 21.
**PMID:** 19192243
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000019967
- Term: Schizophrenia Spectrum and Other Psychotic Disorders
- ID: D000019964
- Term: Mood Disorders
### Condition Browse Module - Browse Branches
- Abbrev: BXM
- Name: Behaviors and Mental Disorders
- Abbrev: All
- Name: All Conditions
### Condition Browse Module - Browse Leaves
- ID: M7058
- Name: Depression
- Relevance: LOW
- As Found: Unknown
- ID: M7061
- Name: Depressive Disorder
- Relevance: HIGH
- As Found: Depressive Disorder
- ID: M15376
- Name: Schizophrenia
- Relevance: HIGH
- As Found: Schizophrenia
- ID: M4815
- Name: Mental Disorders
- Relevance: HIGH
- As Found: Mental Disorders
- ID: M14473
- Name: Psychotic Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M16105
- Name: Stress, Psychological
- Relevance: LOW
- As Found: Unknown
- ID: M21838
- Name: Schizophrenia Spectrum and Other Psychotic Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M21835
- Name: Mood Disorders
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000012559
- Term: Schizophrenia
- ID: D000003866
- Term: Depressive Disorder
- ID: D000001523
- Term: Mental Disorders
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT03214679
**Brief Title:** Accessible HCV Care Intervention for People Who Inject Illicit Drugs (PWID)
**Official Title:** Accessible Care Intervention for Engaging People Who Inject Illicit Drugs in HCV Care
#### Organization Study ID Info
**ID:** 1612017838A001
#### Organization
**Class:** OTHER
**Full Name:** City University of New York, School of Public Health
#### Secondary ID Infos
**ID:** R01DA041298
**Link:** https://reporter.nih.gov/quickSearch/R01DA041298
**Type:** NIH
### Status Module
#### Completion Date
**Date:** 2021-06-30
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2023-02-22
**Type:** ACTUAL
**Last Update Submit Date:** 2023-01-26
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2021-06-30
**Type:** ACTUAL
#### Results First Post Date
**Date:** 2023-02-22
**Type:** ACTUAL
**Results First Submit Date:** 2023-01-26
**Results First Submit QC Date:** 2023-01-26
#### Start Date
**Date:** 2017-07-20
**Type:** ACTUAL
**Status Verified Date:** 2021-07
#### Study First Post Date
**Date:** 2017-07-11
**Type:** ACTUAL
**Study First Submit Date:** 2017-06-29
**Study First Submit QC Date:** 2017-07-07
### Sponsor Collaborators Module
#### Collaborators
**Class:** OTHER
**Name:** National Development and Research Institutes, Inc.
**Class:** NIH
**Name:** National Institute on Drug Abuse (NIDA)
**Class:** OTHER
**Name:** Weill Medical College of Cornell University
#### Lead Sponsor
**Class:** OTHER
**Name:** City University of New York, School of Public Health
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Is FDA Regulated Device:** False
**Is FDA Regulated Drug:** False
**Oversight Has DMC:** True
### Description Module
**Brief Summary:** The proposed study will examine the feasibility, acceptability, safety, effectiveness, and cost of an Accessible Care intervention for engaging people who inject illicit drugs (PWID) in hepatitis C care. Accessible Care for PWID is low-threshold care provided in programs designed specifically for PWID where they can comfortably access care without fear of shame or stigma. Accessible Care will be provided by co-locating a hepatitis treatment provider, together with a Hepatitis C Care Coordinator (HCCC), on-site at a collaborating needle exchange program. The proposed study will compare the effectiveness of Accessible Care with Usual Care (referrals to existing services) in facilitating linkage, engagement, and retention of PWID in care for hepatitis C, addiction, and HIV prevention. The primary outcome is sustained virologic response, which constitutes virologic cure. Substance use and HIV and HCV risk behaviors are secondary outcomes.
### Conditions Module
**Conditions:**
- Hepatitis C
- People Who Inject Drugs
- PWID
- HCV Coinfection
**Keywords:**
- Hepatitis C
- HCV
- People who inject drugs
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 167
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** "Accessible Care" for PWID is low-threshold care provided in the needle exchange programs, where they can comfortably access services without fear of the shame or stigma that often attends them in mainstream institutions.It includes features such as an informal, nonjudgmental atmosphere, availability of walk-in appointments, and a harm reduction framework to help them identify and pursue their own personal health goals. Accessible Care will be provided by co-locating a hepatitis treatment provider, together with a Hepatitis C Care Coordinator, on-site at our collaborating needle exchange program.
**Intervention Names:**
- Other: Accessible Care
**Label:** Accessible Care
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Usual care represents the current process after someone tests positive for HCV antibody on site at the syringe exchange program. An on site care coordinator (not provided by study) assists with insurance and linkage to HCV medical provider at sites throughout NYC through the NYC Dept of Health Check Hep C program.
**Intervention Names:**
- Other: Usual Care
**Label:** Usual Care
**Type:** ACTIVE_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Accessible Care
**Description:** Accessible Care will be provided by co-locating a hepatitis treatment provider, together with a Hepatitis C Care Coordinator, on-site at our collaborating needle exchange program.
**Name:** Accessible Care
**Type:** OTHER
#### Intervention 2
**Arm Group Labels:**
- Usual Care
**Description:** Usual care entails referral to an on site HCV care coordinator (not provided by study)
**Name:** Usual Care
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Description:** SVR12 is a sustained virologic response to HCV treatment defined as HCV RNA below the limit of quantification 12 weeks post completion of HCV treatment
**Measure:** Proportion of Patients to Achieve SVR12 at 1 Year
**Time Frame:** each participant will be assessed at 1 year post entry
**Description:** Proportion of patients in each arm referred to hepatitis C treatment provider by 12 months
**Measure:** Proportion of Patients in Each Arm Referred to Hepatitis C Treatment Provider
**Time Frame:** each participant will be assessed at 1 year post entry
**Description:** The proportion of participants in each arm who attend an initial visit with a hepatitis treatment provider post randomization.
**Measure:** The Proportion of Participants With Hepatitis C Treatment Engagement by 12 Months That Attended an Initial Visit
**Time Frame:** end of study (12 months)
**Description:** Proportion of patients in each arm who complete a medical evaluation for antiviral treatment, including a history, physical examination and laboratory evaluation
**Measure:** Proportion of Patients in Each Arm Who Complete a Medical Evaluation for Antiviral Treatment
**Time Frame:** each participant will be assessed at 1 year post entry
**Description:** Proportion of participants in each arm physically receiving the first dose of antiviral medication (without necessarily having confirmed ingestion)
**Measure:** Proportion of Participants in Each Arm Who Initiated Treatment
**Time Frame:** Each participant will be assessed 1 year post entry
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
1. 18 years or older,
2. injected heroin, cocaine, or other drugs in the past 90 days.
3. test HCV Ab and RNA positive
4. provide written consent (including consent for researchers to examine their hepatitis C medical records)
Exclusion Criteria:
Persons already in care for hepatitis C, defined as having had at least 2 visits with a hepatitis treatment provider within the past 6 months, will be excluded.
People with decompensated cirrhosis will be excluded.
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** New York
**Country:** United States
**Facility:** Lower East Side Harm Reduction Center
**State:** New York
**Zip:** 10022
#### Overall Officials
**Official 1:**
**Affiliation:** Weill Medical College of Cornell University
**Name:** Kristen Marks
**Role:** PRINCIPAL_INVESTIGATOR
### References Module
#### References
**Citation:** Eckhardt B, Mateu-Gelabert P, Aponte-Melendez Y, Fong C, Kapadia S, Smith M, Edlin BR, Marks KM. Accessible Hepatitis C Care for People Who Inject Drugs: A Randomized Clinical Trial. JAMA Intern Med. 2022 May 1;182(5):494-502. doi: 10.1001/jamainternmed.2022.0170.
**PMID:** 35285851
**Citation:** Aponte-Melendez Y, Mateu-Gelabert P, Fong C, Eckhardt B, Kapadia S, Marks K. The impact of COVID-19 on people who inject drugs in New York City: increased risk and decreased access to services. Harm Reduct J. 2021 Nov 24;18(1):118. doi: 10.1186/s12954-021-00568-3.
**PMID:** 34819070
## Document Section
### Large Document Module
#### Large Docs
- Date: 2021-04-16
- Filename: Prot_SAP_000.pdf
- Has ICF: False
- Has Protocol: True
- Has SAP: True
- Label: Study Protocol and Statistical Analysis Plan
- Size: 1826627
- Type Abbrev: Prot_SAP
- Upload Date: 2022-03-31T14:09
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000008107
- Term: Liver Diseases
- ID: D000004066
- Term: Digestive System Diseases
- ID: D000006525
- Term: Hepatitis, Viral, Human
- ID: D000014777
- Term: Virus Diseases
- ID: D000007239
- Term: Infections
- ID: D000012327
- Term: RNA Virus Infections
- ID: D000086982
- Term: Blood-Borne Infections
- ID: D000003141
- Term: Communicable Diseases
- ID: D000018178
- Term: Flaviviridae Infections
### Condition Browse Module - Browse Branches
- Abbrev: BC01
- Name: Infections
- Abbrev: BC06
- Name: Digestive System Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
### Condition Browse Module - Browse Leaves
- ID: M9592
- Name: Hepatitis A
- Relevance: LOW
- As Found: Unknown
- ID: M9591
- Name: Hepatitis
- Relevance: HIGH
- As Found: Hepatitis
- ID: M9611
- Name: Hepatitis C
- Relevance: HIGH
- As Found: Hepatitis C
- ID: M29581
- Name: Coinfection
- Relevance: HIGH
- As Found: Coinfection
- ID: M11107
- Name: Liver Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M7255
- Name: Digestive System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M8883
- Name: Gastrointestinal Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M9610
- Name: Hepatitis, Viral, Human
- Relevance: LOW
- As Found: Unknown
- ID: M17522
- Name: Virus Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M10283
- Name: Infections
- Relevance: LOW
- As Found: Unknown
- ID: M6368
- Name: Communicable Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M15149
- Name: RNA Virus Infections
- Relevance: LOW
- As Found: Unknown
- ID: M2593
- Name: Blood-Borne Infections
- Relevance: LOW
- As Found: Unknown
- ID: M20324
- Name: Flaviviridae Infections
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000006526
- Term: Hepatitis C
- ID: D000060085
- Term: Coinfection
- ID: D000006505
- Term: Hepatitis
### Intervention Browse Module - Browse Branches
- Abbrev: All
- Name: All Drugs and Chemicals
### Intervention Browse Module - Browse Leaves
- ID: M4225
- Name: Antibodies
- Relevance: LOW
- As Found: Unknown
- ID: M10184
- Name: Immunoglobulins
- Relevance: LOW
- As Found: Unknown
- ID: M20971
- Name: Hepatitis C Antibodies
- Relevance: LOW
- As Found: Unknown
### Misc Info Module
- Version Holder: 2024-05-24
## Results Section
### Adverse Events Module
**Description:** 2 ineligible participants excluded post-randomization are not included in any of the outcome or AE analysis
#### Event Groups
**Group ID:** EG000
**Title:** Accessible Care
**Deaths Num Affected:** 3
**Deaths Num At Risk:** 82
**Description:** "Accessible Care" for PWID is low-threshold care provided in the needle exchange programs, where they can comfortably access services without fear of the shame or stigma that often attends them in mainstream institutions.It includes features such as an informal, nonjudgmental atmosphere, availability of walk-in appointments, and a harm reduction framework to help them identify and pursue their own personal health goals. Accessible Care will be provided by co-locating a hepatitis treatment provider, together with a Hepatitis C Care Coordinator, on-site at our collaborating needle exchange program.
Accessible Care: Accessible Care will be provided by co-locating a hepatitis treatment provider, together with a Hepatitis C Care Coordinator, on-site at our collaborating needle exchange program.
**ID:** EG000
**Other Num at Risk:** 82
**Serious Number Affected:** 12
**Serious Number At Risk:** 82
**Title:** Accessible Care
**Group ID:** EG001
**Title:** Usual Care
**Deaths Num Affected:** 1
**Deaths Num At Risk:** 83
**Description:** Usual care represents the current process after someone tests positive for HCV antibody on site at the syringe exchange program. An on site care coordinator (not provided by study) assists with insurance and linkage to HCV medical provider at sites throughout NYC through the NYC Dept of Health Check Hep C program.
Usual Care: Usual care entails referral to an on site HCV care coordinator (not provided by study)
**ID:** EG001
**Other Num at Risk:** 83
**Serious Number Affected:** 11
**Serious Number At Risk:** 83
**Title:** Usual Care
**Frequency Threshold:** 5
#### Serious Events
**Term:** Hospitalizations related to overdose
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Injury, poisoning and procedural complications
##### Stats
**Group ID:** EG000
**Num Affected:** 3
**Num At Risk:** 82
**Num Events:** 3
**Group ID:** EG001
**Num Affected:** 3
**Num At Risk:** 83
**Num Events:** 3
**Term:** Skin/soft tissue infection
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
##### Stats
**Group ID:** EG000
**Num Affected:** 2
**Num At Risk:** 82
**Num Events:** 2
**Group ID:** EG001
**Num Affected:** 2
**Num At Risk:** 83
**Num Events:** 3
**Term:** miscarriage
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Pregnancy, puerperium and perinatal conditions
##### Stats
**Group ID:** EG000
**Num At Risk:** 82
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 83
**Num Events:** 1
**Term:** leg surgery
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Musculoskeletal and connective tissue disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 82
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 83
**Num Events:** 1
**Term:** mental health hospitalization
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Psychiatric disorders
##### Stats
**Group ID:** EG000
**Num Affected:** 4
**Num At Risk:** 82
**Num Events:** 4
**Group ID:** EG001
**Num Affected:** 2
**Num At Risk:** 83
**Num Events:** 2
**Term:** gallbladder surgery
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Hepatobiliary disorders
##### Stats
**Group ID:** EG000
**Num At Risk:** 82
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 83
**Num Events:** 1
**Term:** pneumonia
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Infections and infestations
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 82
**Num Events:** 1
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 83
**Num Events:** 1
**Term:** Alcohol withdrawal seizure
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Psychiatric disorders
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 82
**Num Events:** 1
**Group ID:** EG001
**Num Affected:** 1
**Num At Risk:** 83
**Term:** asthma/shortness of breath
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Respiratory, thoracic and mediastinal disorders
##### Stats
**Group ID:** EG000
**Num Affected:** 2
**Num At Risk:** 82
**Num Events:** 2
**Group ID:** EG001
**Num At Risk:** 83
**Term:** chest pain/palpitations
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Cardiac disorders
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 82
**Num Events:** 3
**Group ID:** EG001
**Num At Risk:** 83
**Term:** acute renal insufficiency
**Assessment Type:** SYSTEMATIC_ASSESSMENT
**Organ System:** Renal and urinary disorders
##### Stats
**Group ID:** EG000
**Num Affected:** 1
**Num At Risk:** 82
**Num Events:** 1
**Group ID:** EG001
**Num At Risk:** 83
**Time Frame:** 12 months
## Results Section - Baseline Characteristics Module
### Denomination Counts
**Group ID:** BG000
**Value:** 82
**Group ID:** BG001
**Value:** 83
**Group ID:** BG002
**Value:** 165
**Units:** Participants
### Group
**ID:** BG000
**Title:** Accessible Care
**Description:** "Accessible Care" for PWID is low-threshold care provided in the needle exchange programs, where they can comfortably access services without fear of the shame or stigma that often attends them in mainstream institutions.It includes features such as an informal, nonjudgmental atmosphere, availability of walk-in appointments, and a harm reduction framework to help them identify and pursue their own personal health goals. Accessible Care will be provided by co-locating a hepatitis treatment provider, together with a Hepatitis C Care Coordinator, on-site at our collaborating needle exchange program.
Accessible Care: Accessible Care will be provided by co-locating a hepatitis treatment provider, together with a Hepatitis C Care Coordinator, on-site at our collaborating needle exchange program.
### Group
**ID:** BG001
**Title:** Usual Care
**Description:** Usual care represents the current procedure after someone tests positive for HCV antibody on site at the syringe exchange program. An on site care coordinator (not provided by study) assists with insurance and linkage to HCV medical provider at sites throughout NYC through the NYC Dept of Health Check Hep C program.
Usual Care: Usual care entails referral to an on site HCV care coordinator (not provided by study)
### Group
**ID:** BG002
**Title:** Total
**Description:** Total of all reporting groups
### Measure
#### Measurement
**Group ID:** BG000
**Spread:** 10.7
**Value:** 42.6
#### Measurement
**Group ID:** BG001
**Spread:** 10.6
**Value:** 41.3
#### Measurement
**Group ID:** BG002
**Spread:** 10.6
**Value:** 42.0
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 19
#### Measurement
**Group ID:** BG001
**Value:** 17
#### Measurement
**Group ID:** BG002
**Value:** 36
**Category Title:** Female
#### Measurement
**Group ID:** BG000
**Value:** 63
#### Measurement
**Group ID:** BG001
**Value:** 66
#### Measurement
**Group ID:** BG002
**Value:** 129
**Category Title:** Male
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 45
#### Measurement
**Group ID:** BG001
**Value:** 52
#### Measurement
**Group ID:** BG002
**Value:** 97
**Category Title:** Hispanic or Latino
#### Measurement
**Group ID:** BG000
**Value:** 37
#### Measurement
**Group ID:** BG001
**Value:** 31
#### Measurement
**Group ID:** BG002
**Value:** 68
**Category Title:** Not Hispanic or Latino
#### Measurement
**Group ID:** BG000
**Value:** 0
#### Measurement
**Group ID:** BG001
**Value:** 0
#### Measurement
**Group ID:** BG002
**Value:** 0
**Category Title:** Unknown or Not Reported
**Class Title:**
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 45
#### Measurement
**Group ID:** BG001
**Value:** 52
#### Measurement
**Group ID:** BG002
**Value:** 97
**Category Title:** Hispanic
#### Measurement
**Group ID:** BG000
**Value:** 26
#### Measurement
**Group ID:** BG001
**Value:** 27
#### Measurement
**Group ID:** BG002
**Value:** 53
**Category Title:** Non Hispanic-White
#### Measurement
**Group ID:** BG000
**Value:** 7
#### Measurement
**Group ID:** BG001
**Value:** 1
#### Measurement
**Group ID:** BG002
**Value:** 8
**Category Title:** Non Hispanic-Black
#### Measurement
**Group ID:** BG000
**Value:** 4
#### Measurement
**Group ID:** BG001
**Value:** 3
#### Measurement
**Group ID:** BG002
**Value:** 7
**Category Title:** Other ( Includes American Indian or Alaska Native, Asian, and Native Hawaiian or Pacific Islander)
**Class Title:** Ethnicity/Race
### Measure
#### Measurement
**Group ID:** BG000
**Value:** 82
#### Measurement
**Group ID:** BG001
**Value:** 83
#### Measurement
**Group ID:** BG002
**Value:** 165
**Class Title:** United States
**Denomination Units Selected:**
## Results Section - Baseline Characteristics Module
### Measure 1
**Dispersion Type:** STANDARD_DEVIATION
**Parameter Type:** MEDIAN
**Title:** Age, Continuous
**Unit of Measure:** years
### Measure 2
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Sex: Female, Male
**Unit of Measure:** Participants
### Measure 3
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Ethnicity (NIH/OMB)
**Unit of Measure:** Participants
### Measure 4
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Title:** Race/Ethnicity, Customized
**Unit of Measure:** Participants
### Measure 5
**Parameter Type:** NUMBER
**Title:** Region of Enrollment
**Unit of Measure:** participants
**Population Description:** 2 ineligible participants excluded post-randomization (both accessible care arm) are not included
## Results Section - More Information Module
### Certain Agreement
### Limitations and Caveats
**Description:** he study limitations to generalizability include that the study was conducted at a single site and in an urban environment with a high concentration of harm reduction services and minimal state HCV DAA prescribing restrictions. As observed in this trial, most patients were insured or eligible for insurance, and no participants had their insurance company deny treatment with HCV DAA therapy.
### Point of Contact
**Email:** [email protected]
**Organization:** University School of Medicine, New York
**Phone:** 212-562-1000
**Title:** Ben Eckhardt
## Results Section - Outcome Measures Module
### Outcome Measure 1
#### Analysis
**CI Lower Limit:**
**CI Number of Sides:**
**CI Percentage Value:**
**CI Upper Limit:**
**CI Upper Limit Comment:**
**Dispersion Type:**
**Dispersion Value:**
**Estimate Comment:**
**Group Description:**
**Non-Inferiority Comment:**
**Non-Inferiority Type:** SUPERIORITY
**Other Analysis Description:**
**P-Value:** <.001
**P-Value Comment:**
**Parameter Type:**
**Parameter Value:**
**Statistical Comment:**
**Statistical Method:** Chi-squared
**Tested Non-Inferiority:**
### Outcome Measure 2
### Outcome Measure 3
### Outcome Measure 4
### Outcome Measure 5
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 55
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 19
**Title:**
#### Outcome Measure 2
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 76
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 37
**Title:**
#### Outcome Measure 3
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 71
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 31
**Title:**
#### Outcome Measure 4
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 71
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 26
**Title:**
#### Outcome Measure 5
**Class:**
##### Category
**Measurements:**
- **Comment:**
- **Group ID:** OG000
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 64
- **Comment:**
- **Group ID:** OG001
- **Lower Limit:**
- **Spread:**
- **Upper Limit:**
- **Value:** 22
**Title:**
## Results Section
### Outcome Measures Module
#### Outcome Measure 1
**Description:** SVR12 is a sustained virologic response to HCV treatment defined as HCV RNA below the limit of quantification 12 weeks post completion of HCV treatment
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** 2 ineligible participants excluded post-randomization are not included in any of the outcome or AE analyses
**Reporting Status:** POSTED
**Time Frame:** each participant will be assessed at 1 year post entry
**Title:** Proportion of Patients to Achieve SVR12 at 1 Year
**Type:** PRIMARY
**Unit of Measure:** Participants
##### Group
**Description:** "Accessible Care" for PWID is low-threshold care provided in the needle exchange programs, where they can comfortably access services without fear of the shame or stigma that often attends them in mainstream institutions.It includes features such as an informal, nonjudgmental atmosphere, availability of walk-in appointments, and a harm reduction framework to help them identify and pursue their own personal health goals. Accessible Care will be provided by co-locating a hepatitis treatment provider, together with a Hepatitis C Care Coordinator, on-site at our collaborating needle exchange program.
Accessible Care: Accessible Care will be provided by co-locating a hepatitis treatment provider, together with a Hepatitis C Care Coordinator, on-site at our collaborating needle exchange program.
**ID:** OG000
**Title:** Accessible Care
##### Group
**Description:** Usual care represents the current procedure after someone tests positive for HCV antibody on site at the syringe exchange program. An on site care coordinator (not provided by study) assists with insurance and linkage to HCV medical provider at sites throughout NYC through the NYC Dept of Health Check Hep C program.
Usual Care: Usual care entails referral to an on site HCV care coordinator (not provided by study)
**ID:** OG001
**Title:** Usual Care
#### Outcome Measure 2
**Description:** Proportion of patients in each arm referred to hepatitis C treatment provider by 12 months
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** 2 ineligible participants excluded post-randomization are not included in any of the outcome or AE analysis
**Reporting Status:** POSTED
**Time Frame:** each participant will be assessed at 1 year post entry
**Title:** Proportion of Patients in Each Arm Referred to Hepatitis C Treatment Provider
**Type:** PRIMARY
**Unit of Measure:** Participants
##### Group
**Description:** "Accessible Care" for PWID is low-threshold care provided in the needle exchange programs, where they can comfortably access services without fear of the shame or stigma that often attends them in mainstream institutions.It includes features such as an informal, nonjudgmental atmosphere, availability of walk-in appointments, and a harm reduction framework to help them identify and pursue their own personal health goals. Accessible Care will be provided by co-locating a hepatitis treatment provider, together with a Hepatitis C Care Coordinator, on-site at our collaborating needle exchange program.
Accessible Care: Accessible Care will be provided by co-locating a hepatitis treatment provider, together with a Hepatitis C Care Coordinator, on-site at our collaborating needle exchange program.
**ID:** OG000
**Title:** Accessible Care
##### Group
**Description:** Usual care represents the current procedure after someone tests positive for HCV antibody on site at the syringe exchange program. An on site care coordinator (not provided by study) assists with insurance and linkage to HCV medical provider at sites throughout NYC through the NYC Dept of Health Check Hep C program.
Usual Care: Usual care entails referral to an on site HCV care coordinator (not provided by study)
**ID:** OG001
**Title:** Usual Care
#### Outcome Measure 3
**Description:** The proportion of participants in each arm who attend an initial visit with a hepatitis treatment provider post randomization.
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** 2 ineligible participants excluded post-randomization are not included in any of the outcome or AE analysis
**Reporting Status:** POSTED
**Time Frame:** end of study (12 months)
**Title:** The Proportion of Participants With Hepatitis C Treatment Engagement by 12 Months That Attended an Initial Visit
**Type:** PRIMARY
**Unit of Measure:** Participants
##### Group
**Description:** "Accessible Care" for PWID is low-threshold care provided in the needle exchange programs, where they can comfortably access services without fear of the shame or stigma that often attends them in mainstream institutions.It includes features such as an informal, nonjudgmental atmosphere, availability of walk-in appointments, and a harm reduction framework to help them identify and pursue their own personal health goals. Accessible Care will be provided by co-locating a hepatitis treatment provider, together with a Hepatitis C Care Coordinator, on-site at our collaborating needle exchange program.
Accessible Care: Accessible Care will be provided by co-locating a hepatitis treatment provider, together with a Hepatitis C Care Coordinator, on-site at our collaborating needle exchange program.
**ID:** OG000
**Title:** Accessible Care
##### Group
**Description:** Usual care represents the current procedure after someone tests positive for HCV antibody on site at the syringe exchange program. An on site care coordinator (not provided by study) assists with insurance and linkage to HCV medical provider at sites throughout NYC through the NYC Dept of Health Check Hep C program.
Usual Care: Usual care entails referral to an on site HCV care coordinator (not provided by study)
**ID:** OG001
**Title:** Usual Care
#### Outcome Measure 4
**Description:** Proportion of patients in each arm who complete a medical evaluation for antiviral treatment, including a history, physical examination and laboratory evaluation
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** 2 ineligible participants excluded post-randomization are not included in any of the outcome or AE analysis
**Reporting Status:** POSTED
**Time Frame:** each participant will be assessed at 1 year post entry
**Title:** Proportion of Patients in Each Arm Who Complete a Medical Evaluation for Antiviral Treatment
**Type:** PRIMARY
**Unit of Measure:** Participants
##### Group
**Description:** "Accessible Care" for PWID is low-threshold care provided in the needle exchange programs, where they can comfortably access services without fear of the shame or stigma that often attends them in mainstream institutions.It includes features such as an informal, nonjudgmental atmosphere, availability of walk-in appointments, and a harm reduction framework to help them identify and pursue their own personal health goals. Accessible Care will be provided by co-locating a hepatitis treatment provider, together with a Hepatitis C Care Coordinator, on-site at our collaborating needle exchange program.
Accessible Care: Accessible Care will be provided by co-locating a hepatitis treatment provider, together with a Hepatitis C Care Coordinator, on-site at our collaborating needle exchange program.
**ID:** OG000
**Title:** Accessible Care
##### Group
**Description:** Usual care represents the current procedure after someone tests positive for HCV antibody on site at the syringe exchange program. An on site care coordinator (not provided by study) assists with insurance and linkage to HCV medical provider at sites throughout NYC through the NYC Dept of Health Check Hep C program.
Usual Care: Usual care entails referral to an on site HCV care coordinator (not provided by study)
**ID:** OG001
**Title:** Usual Care
#### Outcome Measure 5
**Description:** Proportion of participants in each arm physically receiving the first dose of antiviral medication (without necessarily having confirmed ingestion)
**Parameter Type:** COUNT_OF_PARTICIPANTS
**Population Description:** 2 ineligible participants excluded post-randomization are not included in any of the outcome or AE analysis
**Reporting Status:** POSTED
**Time Frame:** Each participant will be assessed 1 year post entry
**Title:** Proportion of Participants in Each Arm Who Initiated Treatment
**Type:** PRIMARY
**Unit of Measure:** Participants
##### Group
**Description:** "Accessible Care" for PWID is low-threshold care provided in the needle exchange programs, where they can comfortably access services without fear of the shame or stigma that often attends them in mainstream institutions.It includes features such as an informal, nonjudgmental atmosphere, availability of walk-in appointments, and a harm reduction framework to help them identify and pursue their own personal health goals. Accessible Care will be provided by co-locating a hepatitis treatment provider, together with a Hepatitis C Care Coordinator, on-site at our collaborating needle exchange program.
Accessible Care: Accessible Care will be provided by co-locating a hepatitis treatment provider, together with a Hepatitis C Care Coordinator, on-site at our collaborating needle exchange program.
**ID:** OG000
**Title:** Accessible Care
##### Group
**Description:** Usual care represents the current procedure after someone tests positive for HCV antibody on site at the syringe exchange program. An on site care coordinator (not provided by study) assists with insurance and linkage to HCV medical provider at sites throughout NYC through the NYC Dept of Health Check Hep C program.
Usual Care: Usual care entails referral to an on site HCV care coordinator (not provided by study)
**ID:** OG001
**Title:** Usual Care
### Participant Flow Module
#### Group
**Description:** "Accessible Care" for PWID is low-threshold care provided in the needle exchange programs, where they can comfortably access services without fear of the shame or stigma that often attends them in mainstream institutions.It includes features such as an informal, nonjudgmental atmosphere, availability of walk-in appointments, and a harm reduction framework to help them identify and pursue their own personal health goals. Accessible Care will be provided by co-locating a hepatitis treatment provider, together with a Hepatitis C Care Coordinator, on-site at our collaborating needle exchange program.
Accessible Care: Accessible Care will be provided by co-locating a hepatitis treatment provider, together with a Hepatitis C Care Coordinator, on-site at our collaborating needle exchange program.
**ID:** FG000
**Title:** Accessible Care
#### Group
**Description:** Usual care represents the current process after someone tests positive for HCV antibody on site at the syringe exchange program. An on site care coordinator (not provided by study) assists with insurance and linkage to HCV medical provider at sites throughout NYC through the NYC Dept of Health Check Hep C program.
Usual Care: Usual care entails referral to an on site HCV care coordinator (not provided by study)
**ID:** FG001
**Title:** Usual Care
#### Period
**Title:** Overall Study
##### Withdraw
**Type:** Lost to Follow-up
###### Reason
**Group ID:** FG000
**Number of Subjects:** 12
###### Reason
**Group ID:** FG001
**Number of Subjects:** 14
##### Withdraw
**Type:** not eligible
###### Reason
**Group ID:** FG000
**Number of Subjects:** 2
###### Reason
**Group ID:** FG001
**Number of Subjects:** 0
##### Milestone
**Type:** STARTED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 84
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 83
##### Milestone
**Type:** COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 70
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 69
##### Milestone
**Type:** NOT COMPLETED
###### Achievement
**Group ID:** FG000
**Number of Subjects:** 14
###### Achievement
**Group ID:** FG001
**Number of Subjects:** 14
**Has Results:** True |
## Protocol Section
### Identification Module
**NCT ID:** NCT00000179
**Brief Title:** Agitation in Alzheimer's Disease
#### Organization Study ID Info
**ID:** IA0003
#### Organization
**Class:** NIH
**Full Name:** National Institute on Aging (NIA)
#### Secondary ID Infos
**ID:** 3U01AG010483-08S2
**Link:** https://reporter.nih.gov/quickSearch/3U01AG010483-08S2
**Type:** NIH
### Status Module
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2005-06-24
**Type:** ESTIMATED
**Last Update Submit Date:** 2005-06-23
**Overall Status:** COMPLETED
**Status Verified Date:** 2005-03
#### Study First Post Date
**Date:** 1999-11-01
**Type:** ESTIMATED
**Study First Submit Date:** 1999-10-29
**Study First Submit QC Date:** 1999-10-29
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** NIH
**Name:** National Institute on Aging (NIA)
### Description Module
**Brief Summary:** Agitation affects 70 to 90 percent of patients with AD. Signs of agitation include verbal and physical aggressiveness, irritability, wandering, and restlessness. These behaviors often make caring for patients at home very difficult. Trazodone and haldol are two of the most commonly prescribed drugs for agitation in AD patients. Behavior management, a non drug approach, has been effective in reducing signs of agitation. Researchers have yet to compare the effectiveness of drug versus non drug therapy to treat agitation in AD patients and determine which is the best treatment. The Alzheimer's Disease Cooperative Study, with funding from the National Institute on Aging, is conducting an agitation treatment program at 21 sites in 16 States. This study will assess which of the above treatments is most effective.
### Conditions Module
**Conditions:**
- Alzheimer Disease
**Keywords:**
- Alzheimer's disease
- Psychomotor agitation
- Behavioral symptoms
- Haloperidol
- Trazodone
- Anti-psychotic agents
- Anti-depressant agents
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
##### Masking Info
**Masking:** DOUBLE
**Primary Purpose:** TREATMENT
**Phases:**
- PHASE3
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
### Interventions
#### Intervention 1
**Name:** Trazodone
**Type:** DRUG
#### Intervention 2
**Name:** Haloperidol
**Type:** DRUG
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Memory problem consistent with a probable diagnosis of Alzheimer's disease (AD)
* Agitation symptoms for at least the past 2 weeks
* Patient has caregiver who can participate
* Patient lives in the same household as the caregiver
**Minimum Age:** 50 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Los Angeles
**Country:** United States
**Facility:** University of California, Los Angeles
**State:** California
**Zip:** 90095
**Location 2:**
**City:** San Diego
**Country:** United States
**Facility:** University of California, San Diego
**State:** California
**Zip:** 92093-0949
**Location 3:**
**City:** Miami
**Country:** United States
**Facility:** University of Miami
**State:** Florida
**Zip:** 33140
**Location 4:**
**City:** Tampa
**Country:** United States
**Facility:** University of South Florida
**State:** Florida
**Zip:** 33162
**Location 5:**
**City:** Atlanta
**Country:** United States
**Facility:** Emory University
**State:** Georgia
**Zip:** 30329
**Location 6:**
**City:** Springfield
**Country:** United States
**Facility:** Southern Illinois University
**State:** Illinois
**Zip:** 62702
**Location 7:**
**City:** Kansas City
**Country:** United States
**Facility:** University of Kansas Medical Center
**State:** Kansas
**Zip:** 66160
**Location 8:**
**City:** Lexington
**Country:** United States
**Facility:** University of Kentucky
**State:** Kentucky
**Zip:** 40536
**Location 9:**
**City:** Worcester
**Country:** United States
**Facility:** University of Massachusetts
**State:** Massachusetts
**Zip:** 01665
**Location 10:**
**City:** Ann Arbor
**Country:** United States
**Facility:** University of Michigan
**State:** Michigan
**Zip:** 48109
**Location 11:**
**City:** Minneapolis
**Country:** United States
**Facility:** University of Minnesota
**State:** Minnesota
**Zip:** 55455
**Location 12:**
**City:** New York
**Country:** United States
**Facility:** New York University Medical Center
**State:** New York
**Zip:** 10016
**Location 13:**
**City:** New York
**Country:** United States
**Facility:** Mount Sinai Medical Center
**State:** New York
**Zip:** 10029
**Location 14:**
**City:** Rochester
**Country:** United States
**Facility:** University of Rochester
**State:** New York
**Zip:** 14620
**Location 15:**
**City:** Cleveland
**Country:** United States
**Facility:** University Hospitals of Cleveland
**State:** Ohio
**Zip:** 44120
**Location 16:**
**City:** Portland
**Country:** United States
**Facility:** Oregon Health Sciences University
**State:** Oregon
**Zip:** 97201-3098
#### Overall Officials
**Official 1:**
**Affiliation:** University of California, San Diego
**Name:** Leon Thal, MD.
**Role:** PRINCIPAL_INVESTIGATOR
### References Module
#### References
**Citation:** Teri L, Logsdon RG, Peskind E, Raskind M, Weiner MF, Tractenberg RE, Foster NL, Schneider LS, Sano M, Whitehouse P, Tariot P, Mellow AM, Auchus AP, Grundman M, Thomas RG, Schafer K, Thal LJ; Alzheimer's Disease Cooperative Study. Treatment of agitation in AD: a randomized, placebo-controlled clinical trial. Neurology. 2000 Nov 14;55(9):1271-8. doi: 10.1212/wnl.55.9.1271. Erratum In: Neurology 2001 Feb 13;56(3):426.
**PMID:** 11087767
#### See Also Links
**Label:** The Alzheimer's Disease Education and Referral (ADEAR) Center is a service of the National Institute on Aging (NIA)
**URL:** http://www.alzheimers.org/
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000003704
- Term: Dementia
- ID: D000001927
- Term: Brain Diseases
- ID: D000002493
- Term: Central Nervous System Diseases
- ID: D000009422
- Term: Nervous System Diseases
- ID: D000024801
- Term: Tauopathies
- ID: D000019636
- Term: Neurodegenerative Diseases
- ID: D000019965
- Term: Neurocognitive Disorders
- ID: D000001523
- Term: Mental Disorders
### Condition Browse Module - Browse Branches
- Abbrev: BC10
- Name: Nervous System Diseases
- Abbrev: BXM
- Name: Behaviors and Mental Disorders
- Abbrev: All
- Name: All Conditions
- Abbrev: BC23
- Name: Symptoms and General Pathology
- Abbrev: Rare
- Name: Rare Diseases
### Condition Browse Module - Browse Leaves
- ID: M3885
- Name: Alzheimer Disease
- Relevance: HIGH
- As Found: Alzheimer's Disease
- ID: M4815
- Name: Mental Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M14473
- Name: Psychotic Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M4818
- Name: Behavioral Symptoms
- Relevance: LOW
- As Found: Unknown
- ID: M14452
- Name: Psychomotor Agitation
- Relevance: LOW
- As Found: Unknown
- ID: M6904
- Name: Dementia
- Relevance: LOW
- As Found: Unknown
- ID: M5204
- Name: Brain Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5742
- Name: Central Nervous System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M23002
- Name: Tauopathies
- Relevance: LOW
- As Found: Unknown
- ID: M21558
- Name: Neurodegenerative Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M21836
- Name: Neurocognitive Disorders
- Relevance: LOW
- As Found: Unknown
- ID: T2192
- Name: Familial Alzheimer Disease
- Relevance: HIGH
- As Found: Alzheimer's Disease
### Condition Browse Module - Meshes
- ID: D000000544
- Term: Alzheimer Disease
### Intervention Browse Module - Ancestors
- ID: D000000932
- Term: Antiemetics
- ID: D000001337
- Term: Autonomic Agents
- ID: D000018373
- Term: Peripheral Nervous System Agents
- ID: D000045505
- Term: Physiological Effects of Drugs
- ID: D000005765
- Term: Gastrointestinal Agents
- ID: D000014150
- Term: Antipsychotic Agents
- ID: D000014149
- Term: Tranquilizing Agents
- ID: D000002492
- Term: Central Nervous System Depressants
- ID: D000011619
- Term: Psychotropic Drugs
- ID: D000018492
- Term: Dopamine Antagonists
- ID: D000015259
- Term: Dopamine Agents
- ID: D000018377
- Term: Neurotransmitter Agents
- ID: D000045504
- Term: Molecular Mechanisms of Pharmacological Action
- ID: D000018726
- Term: Anti-Dyskinesia Agents
- ID: D000014151
- Term: Anti-Anxiety Agents
- ID: D000017367
- Term: Selective Serotonin Reuptake Inhibitors
- ID: D000014179
- Term: Neurotransmitter Uptake Inhibitors
- ID: D000049990
- Term: Membrane Transport Modulators
- ID: D000018490
- Term: Serotonin Agents
- ID: D000018687
- Term: Antidepressive Agents, Second-Generation
- ID: D000000928
- Term: Antidepressive Agents
### Intervention Browse Module - Browse Branches
- Abbrev: AnDyAg
- Name: Anti-Dyskinesia Agents
- Abbrev: AnEm
- Name: Antiemetics
- Abbrev: CNSDep
- Name: Central Nervous System Depressants
- Abbrev: PsychDr
- Name: Psychotropic Drugs
- Abbrev: Gast
- Name: Gastrointestinal Agents
- Abbrev: All
- Name: All Drugs and Chemicals
- Abbrev: CaAg
- Name: Cardiotonic Agents
### Intervention Browse Module - Browse Leaves
- ID: M9312
- Name: Haloperidol
- Relevance: HIGH
- As Found: Endometriosis
- ID: M215475
- Name: Haloperidol decanoate
- Relevance: HIGH
- As Found: Endometriosis
- ID: M4247
- Name: Antidepressive Agents
- Relevance: LOW
- As Found: Unknown
- ID: M16950
- Name: Trazodone
- Relevance: HIGH
- As Found: PAH
- ID: M16904
- Name: Antipsychotic Agents
- Relevance: LOW
- As Found: Unknown
- ID: M4251
- Name: Antiemetics
- Relevance: LOW
- As Found: Unknown
- ID: M8881
- Name: Gastrointestinal Agents
- Relevance: LOW
- As Found: Unknown
- ID: M14474
- Name: Psychotropic Drugs
- Relevance: LOW
- As Found: Unknown
- ID: M7473
- Name: Dopamine
- Relevance: LOW
- As Found: Unknown
- ID: M20596
- Name: Dopamine Antagonists
- Relevance: LOW
- As Found: Unknown
- ID: M17962
- Name: Dopamine Agents
- Relevance: LOW
- As Found: Unknown
- ID: M20504
- Name: Neurotransmitter Agents
- Relevance: LOW
- As Found: Unknown
- ID: M16905
- Name: Anti-Anxiety Agents
- Relevance: LOW
- As Found: Unknown
- ID: M19649
- Name: Selective Serotonin Reuptake Inhibitors
- Relevance: LOW
- As Found: Unknown
- ID: M15512
- Name: Serotonin
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: D000006220
- Term: Haloperidol
- ID: C000033563
- Term: Haloperidol decanoate
- ID: D000014196
- Term: Trazodone
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT02393079
**Brief Title:** Analysis of TCLT in TBI Patients: a Prospective, Randomized Controlled Trial
**Official Title:** The Effects of Transcranial LED Therapy (TCLT) in Patients With Traumatic Brain Injury (TBI): a Prospective, Randomized Controlled Trial
#### Organization Study ID Info
**ID:** TCLT/DIP
#### Organization
**Class:** OTHER
**Full Name:** University of Sao Paulo General Hospital
### Status Module
#### Completion Date
**Date:** 2019-11
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2022-04-29
**Type:** ACTUAL
**Last Update Submit Date:** 2022-04-22
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2019-06
**Type:** ACTUAL
#### Start Date
**Date:** 2015-01
**Status Verified Date:** 2022-04
#### Study First Post Date
**Date:** 2015-03-19
**Type:** ESTIMATED
**Study First Submit Date:** 2015-02-20
**Study First Submit QC Date:** 2015-03-13
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** University of Sao Paulo General Hospital
#### Responsible Party
**Investigator Affiliation:** University of Sao Paulo General Hospital
**Investigator Full Name:** Guilherme da Cruz Ribeiro Poiani
**Investigator Title:** Principal Investigator - clinical neuropsychologist
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** The purpose of this study is to evaluate the early and late effects of Transcranial Led Therapy (TLTC) in memory and executive functions in patients with moderate and severe TBI history (TBI time longer than 3 months).
**Detailed Description:** In the current study patients in the outpatient center - Neurotrauma Unit at University of São Paulo (HCFMUSP) diagnosed with moderate / severe blunt head trauma that meet the inclusion criteria will be divided randomly and blindly in two groups: Group A (n = 18) will receive stimulation through active helmet and Group B (n = 18) given placebo stimulation or sham through the inactive helmet. All patients will be subjected to 3 times of neuropsychological assessment: Study 1 (E1) - up to 4 weeks before the start of TLTC sessions; Study 2 (E2) - a week after the end of TLTC; and Study 3 (E3) - three months after the intervention. The evaluations will take in order to verify the early and late effects of TLTC in attention, memory and executive functions in patients with moderate and severe TBI.
### Conditions Module
**Conditions:**
- Brain Injury
- Cognitive Deficits
**Keywords:**
- Traumatic Brain Injury
- Cognitive
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** DOUBLE
**Who Masked:**
- PARTICIPANT
- OUTCOMES_ASSESSOR
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 36
**Type:** ACTUAL
**Phases:**
- NA
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Description of the intervention: 18 patients will undergo 18 sessions of transcranial LED therapy (ACTIVE HELMET) with 30 minutes duration each. The sessions take place over 6 weeks. The therapy will be performed with a helmet that covers the frontal and parietal region.
**Intervention Names:**
- Device: Transcranial LED Therapy (TCLT)
**Label:** Active helmet LED
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Description of the intervention: 18 patients will undergo 18 sessions of transcranial LED therapy (INACTIVE HELMET) with 30 minutes duration each. The sessions take place over 6 weeks. The therapy will be performed with a helmet that covers the frontal and parietal region.
**Intervention Names:**
- Device: Sham
**Label:** Sham group
**Type:** SHAM_COMPARATOR
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Active helmet LED
**Description:** Whereas the geometry of the skull and to uniformly distribute the light radiation, the TLTC is applied using as light source a set of LEDs installed in a helmet, emitting radiation in the range between 600nm and 650nm with a peak at 630nm, so in the red region.
**Name:** Transcranial LED Therapy (TCLT)
**Type:** DEVICE
#### Intervention 2
**Arm Group Labels:**
- Sham group
**Description:** A SHAM identical helmet will be used in the control group.
**Name:** Sham
**Type:** DEVICE
### Outcomes Module
#### Primary Outcomes
**Description:** Measurement of each patient's inhibitory control by means of a test that causes them to cognitively suppress a habitual response in favor of a less habitual one. The results of patients with shorter response times indicate better positions in the tables, according to age. Examples of the average time (in seconds) of best results are between 17-29 years (11.79) and 80+ (19.31), any time longer than these indicates decreasing results.
**Measure:** Evidence of improvement in inhibitory control measured by cognitive assessment (Stroop Test - Victoria Version) in patients undergoing intervention with an active LED helmet.
**Time Frame:** Three times: Up to one week before to start intervention, one week and three months after end the intervention
#### Secondary Outcomes
**Description:** Apply a neuropsychological assessment battery focused on executive functioning, attentional processes and memory to measure the effects of the active led helmet compared to the use of the sham device.
**Measure:** Assess the immediate and late effects of the intervention on victims of moderate or severe TBI in the following cognitive domains: executive functioning, attentional processes and memory.
**Time Frame:** Three times: Up to one week before to start intervention, one week and three months after end the intervention
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Clinical and radiological diagnosis of traumatic brain injury
* Must be able to sign the Informed Consent Form
* Patients with TBI beginning three months after injury
Exclusion Criteria:
* Impairment of language and / or limiting engine
* Extensive Cranial vault defects
* Presence of uncontrolled post-TBI epilepsy
**Maximum Age:** 60 Years
**Minimum Age:** 18 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Sao Paulo
**Country:** Brazil
**Facility:** Clinics Hospital - University of Sao Paulo Medical School
**State:** SP
**Zip:** 054010-000
#### Overall Officials
**Official 1:**
**Affiliation:** Clinics Hospital - University of Sao Paulo Medical School
**Name:** Wellingson S Paiva, MD PhD
**Role:** PRINCIPAL_INVESTIGATOR
### References Module
#### References
**Citation:** Poiani GDCR, Zaninotto AL, Carneiro AMC, Zangaro RA, Salgado ASI, Parreira RB, de Andrade AF, Teixeira MJ, Paiva WS. Photobiomodulation using low-level laser therapy (LLLT) for patients with chronic traumatic brain injury: a randomized controlled trial study protocol. Trials. 2018 Jan 8;19(1):17. doi: 10.1186/s13063-017-2414-5.
**PMID:** 29310710
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000014947
- Term: Wounds and Injuries
- ID: D000001927
- Term: Brain Diseases
- ID: D000002493
- Term: Central Nervous System Diseases
- ID: D000009422
- Term: Nervous System Diseases
- ID: D000006259
- Term: Craniocerebral Trauma
- ID: D000020196
- Term: Trauma, Nervous System
- ID: D000019965
- Term: Neurocognitive Disorders
- ID: D000001523
- Term: Mental Disorders
### Condition Browse Module - Browse Branches
- Abbrev: BXM
- Name: Behaviors and Mental Disorders
- Abbrev: All
- Name: All Conditions
- Abbrev: BC10
- Name: Nervous System Diseases
- Abbrev: BC26
- Name: Wounds and Injuries
### Condition Browse Module - Browse Leaves
- ID: M29705
- Name: Cognitive Dysfunction
- Relevance: HIGH
- As Found: Cognitive Deficits
- ID: M5207
- Name: Brain Injuries
- Relevance: HIGH
- As Found: Brain Injury
- ID: M628
- Name: Brain Injuries, Traumatic
- Relevance: LOW
- As Found: Unknown
- ID: M6301
- Name: Cognition Disorders
- Relevance: HIGH
- As Found: Cognitive Deficits
- ID: M17685
- Name: Wounds and Injuries
- Relevance: LOW
- As Found: Unknown
- ID: M5204
- Name: Brain Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M5742
- Name: Central Nervous System Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M9349
- Name: Craniocerebral Trauma
- Relevance: LOW
- As Found: Unknown
- ID: M22023
- Name: Trauma, Nervous System
- Relevance: LOW
- As Found: Unknown
- ID: M21836
- Name: Neurocognitive Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M4815
- Name: Mental Disorders
- Relevance: LOW
- As Found: Unknown
- ID: M14473
- Name: Psychotic Disorders
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000001930
- Term: Brain Injuries
- ID: D000003072
- Term: Cognition Disorders
- ID: D000060825
- Term: Cognitive Dysfunction
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT02147379
**Brief Title:** Lurasidone and Cognition in Bipolar I Disorder
**Official Title:** Changes in Cognitive Functioning in Euthymic Bipolar Patients Treated With Lurasidone Versus Treatment as Usual; A Randomized, Open-Label Study.
#### Organization Study ID Info
**ID:** H14-00290
#### Organization
**Class:** OTHER
**Full Name:** University of British Columbia
### Status Module
#### Completion Date
**Date:** 2017-01-12
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2017-02-02
**Type:** ESTIMATED
**Last Update Submit Date:** 2017-02-01
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2015-10
**Type:** ACTUAL
#### Start Date
**Date:** 2014-05
**Status Verified Date:** 2017-02
#### Study First Post Date
**Date:** 2014-05-26
**Type:** ESTIMATED
**Study First Submit Date:** 2014-05-08
**Study First Submit QC Date:** 2014-05-21
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** University of British Columbia
#### Responsible Party
**Type:** SPONSOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** This is a 6 week, randomized, open-label, parallel group study in patients with Bipolar-I disorder (manic depression), who are in remission from an episode. Participants who show cognitive impairment at baseline will be randomized to receive open-label Lurasidone added on to their current medication(s) or continue their usual treatment for 6 weeks. Participants will have 3 clinical visits and 2 telephone appointments during the study. Given the preliminary evidence for efficacy of Lurasidone in improving cognition in schizophrenia, we propose to examine the efficacy of Lurasidone in improving cognition in bipolar patients.
### Conditions Module
**Conditions:**
- Bipolar I Disorder
**Keywords:**
- Bipolar Disorder
- Cognition
- Euthymic
- Lurasidone
### Design Module
#### Design Info
**Allocation:** RANDOMIZED
**Intervention Model:** PARALLEL
##### Masking Info
**Masking:** NONE
**Primary Purpose:** TREATMENT
#### Enrollment Info
**Count:** 53
**Type:** ACTUAL
**Phases:**
- PHASE3
**Study Type:** INTERVENTIONAL
### Arms Interventions Module
#### Arm Group 1
**Description:** Lurasidone 20 - 80 mg / day added to current treatment for 6 weeks.
**Intervention Names:**
- Drug: Lurasidone
**Label:** Lurasidone
**Type:** EXPERIMENTAL
#### Arm Group 2
**Description:** Patients randomized to this arm will continue their usual treatment.
**Label:** Treatment as usual
**Type:** NO_INTERVENTION
### Interventions
#### Intervention 1
**Arm Group Labels:**
- Lurasidone
**Name:** Lurasidone
**Other Names:**
- Latuda
**Type:** DRUG
### Outcomes Module
#### Primary Outcomes
**Description:** The International Society for Bipolar Disorders-Battery for Assessment of Neurocognition (ISBD-BANC) will be administered to assess cognitive functioning at baseline and at endpoint ( change in scores on cognitive tests) to evaluate the efficacy of lurasidone on cognitive changes in bipolar disorder.
**Measure:** Cognitive Change in Bipolar I disorder after treatment with Lurasidone
**Time Frame:** 6 weeks
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
1. All patients must be taking either lithium or valproate or one of the atypical antipsychotics for mood stabilization.
2. Patients who show cognitive impairments (-0.25 standard deviations (SD) or below) on either the Trail Making Test- trial B (TMT-B) or California Verbal Learning Test (CVLT) trials 1 to 5, or CVLT long delay free recall, at baseline visit.
3. Males or females aged 19 to 65 years inclusive.
4. Diagnostic and Statistical Manual IV edition (DSM-IV TR) diagnosis of Bipolar Type I Disorder, with or without a history of psychosis.
5. Clinically stable with a Montgomery Asberg Depression Rating Scale (MADRS) and Young Mania Rating Scale (YMRS) score less than or equal to 8.
6. A sufficient level of English using a language screening questionnaire.
7. Capability of understanding, consenting to, and complying with the requirements of the study.
Exclusion Criteria:
1. A history of unstable or inadequately treated medical illnesses, including moderate to severe brain injury, or neurological illnesses impacting cognitive function.
2. Treatment with Electroconvulsive Therapy (ECT) within eight weeks prior to enrolment; or treatment with an experimental drug within 30 days prior to enrolment.
3. Axis I diagnosis of substance abuse or dependence within the past month.
4. Significant risk of harm to self or others, in the opinion of the investigator.
5. Pregnancy or lactation in female subjects.
6. Liver function tests (AST and ALT) three times the upper limit of normal. -
**Maximum Age:** 65 Years
**Minimum Age:** 19 Years
**Sex:** ALL
**Standard Ages:**
- ADULT
- OLDER_ADULT
### Contacts Locations Module
#### Locations
**Location 1:**
**City:** Vancouver
**Country:** Canada
**Facility:** University of British Columbia, Department of Psychiatry
**State:** British Columbia
**Zip:** V6T 2A1
#### Overall Officials
**Official 1:**
**Affiliation:** University of British Columbia
**Name:** Lakshmi N Yatham, MBBS,FRCPC,MRCPsych (UK),MB
**Role:** PRINCIPAL_INVESTIGATOR
### References Module
#### References
**Citation:** Yatham LN, Mackala S, Basivireddy J, Ahn S, Walji N, Hu C, Lam RW, Torres IJ. Lurasidone versus treatment as usual for cognitive impairment in euthymic patients with bipolar I disorder: a randomised, open-label, pilot study. Lancet Psychiatry. 2017 Mar;4(3):208-217. doi: 10.1016/S2215-0366(17)30046-9. Epub 2017 Feb 7. Erratum In: Lancet Psychiatry. 2017 Mar;4(3):185.
**PMID:** 28185899
## Derived Section
### Condition Browse Module - Browse Branches
- Abbrev: BXM
- Name: Behaviors and Mental Disorders
- Abbrev: All
- Name: All Conditions
### Condition Browse Module - Browse Leaves
- ID: M4996
- Name: Bipolar Disorder
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Ancestors
- ID: D000014150
- Term: Antipsychotic Agents
- ID: D000014149
- Term: Tranquilizing Agents
- ID: D000002492
- Term: Central Nervous System Depressants
- ID: D000045505
- Term: Physiological Effects of Drugs
- ID: D000011619
- Term: Psychotropic Drugs
- ID: D000058669
- Term: Adrenergic alpha-2 Receptor Antagonists
- ID: D000000317
- Term: Adrenergic alpha-Antagonists
- ID: D000018674
- Term: Adrenergic Antagonists
- ID: D000018663
- Term: Adrenergic Agents
- ID: D000018377
- Term: Neurotransmitter Agents
- ID: D000045504
- Term: Molecular Mechanisms of Pharmacological Action
- ID: D000058830
- Term: Serotonin 5-HT2 Receptor Antagonists
- ID: D000012702
- Term: Serotonin Antagonists
- ID: D000018490
- Term: Serotonin Agents
- ID: D000065127
- Term: Dopamine D2 Receptor Antagonists
- ID: D000018492
- Term: Dopamine Antagonists
- ID: D000015259
- Term: Dopamine Agents
### Intervention Browse Module - Browse Branches
- Abbrev: CNSDep
- Name: Central Nervous System Depressants
- Abbrev: PsychDr
- Name: Psychotropic Drugs
- Abbrev: All
- Name: All Drugs and Chemicals
- Abbrev: CaAg
- Name: Cardiotonic Agents
### Intervention Browse Module - Browse Leaves
- ID: M348
- Name: Lurasidone Hydrochloride
- Relevance: HIGH
- As Found: Thrombolysis
- ID: M16904
- Name: Antipsychotic Agents
- Relevance: LOW
- As Found: Unknown
- ID: M14474
- Name: Psychotropic Drugs
- Relevance: LOW
- As Found: Unknown
- ID: M20746
- Name: Adrenergic Agents
- Relevance: LOW
- As Found: Unknown
- ID: M29195
- Name: Adrenergic alpha-2 Receptor Antagonists
- Relevance: LOW
- As Found: Unknown
- ID: M3669
- Name: Adrenergic alpha-Antagonists
- Relevance: LOW
- As Found: Unknown
- ID: M20755
- Name: Adrenergic Antagonists
- Relevance: LOW
- As Found: Unknown
- ID: M20504
- Name: Neurotransmitter Agents
- Relevance: LOW
- As Found: Unknown
- ID: M15512
- Name: Serotonin
- Relevance: LOW
- As Found: Unknown
- ID: M7473
- Name: Dopamine
- Relevance: LOW
- As Found: Unknown
- ID: M20596
- Name: Dopamine Antagonists
- Relevance: LOW
- As Found: Unknown
- ID: M17962
- Name: Dopamine Agents
- Relevance: LOW
- As Found: Unknown
### Intervention Browse Module - Meshes
- ID: D000069056
- Term: Lurasidone Hydrochloride
### Misc Info Module
- Version Holder: 2024-05-24
|
## Protocol Section
### Identification Module
**NCT ID:** NCT02552979
**Brief Title:** Atopy Patch Test in Children With Atopic Dermatitis
**Official Title:** Atopy Patch Test in Children With Atopic Dermatitis
#### Organization Study ID Info
**ID:** Si524/2011
#### Organization
**Class:** OTHER
**Full Name:** Mahidol University
### Status Module
#### Completion Date
**Date:** 2015-09
**Type:** ACTUAL
#### Expanded Access Info
#### Last Update Post Date
**Date:** 2016-05-03
**Type:** ESTIMATED
**Last Update Submit Date:** 2016-05-02
**Overall Status:** COMPLETED
#### Primary Completion Date
**Date:** 2014-12
**Type:** ACTUAL
#### Start Date
**Date:** 2012-01
**Status Verified Date:** 2016-05
#### Study First Post Date
**Date:** 2015-09-17
**Type:** ESTIMATED
**Study First Submit Date:** 2015-09-16
**Study First Submit QC Date:** 2015-09-16
### Sponsor Collaborators Module
#### Lead Sponsor
**Class:** OTHER
**Name:** Mahidol University
#### Responsible Party
**Investigator Affiliation:** Mahidol University
**Investigator Full Name:** Nualanong Visitsunthorn
**Investigator Title:** Professor
**Type:** PRINCIPAL_INVESTIGATOR
### Oversight Module
**Oversight Has DMC:** False
### Description Module
**Brief Summary:** Positive reactions in atopy patch test in children with atopic dermatitis.
**Detailed Description:** The prevalence of food allergy seems to be increasing, which might explain the increased demand for reliable evaluation of patients with suspected food-related gastrointestinal symptoms. Little is known about the diagnostic accuracy of atopy patch tests(APT) in the clinical practice. APT seems to have a better specificity than the IgE methods and seems to reflect late-phase clinical reactions.The aims of this study were to evaluate:
(i) The prevalence of positive reaction of APTs for atopic dermatitis (ii)Positive reaction of APT compare with skin prick test. (iii)Positive reaction of APT using lyophilized food vs commercially available food extracts. (iv)Side effect or adverse
### Conditions Module
**Conditions:**
- Atopic Dermatitis
- Food Allergy
**Keywords:**
- atopic dermatitis
- atopic patch test
### Design Module
#### Design Info
**Time Perspective:** PROSPECTIVE
#### Enrollment Info
**Count:** 56
**Type:** ACTUAL
**Study Type:** OBSERVATIONAL
### Arms Interventions Module
### Interventions
#### Intervention 1
**Name:** atopic patch test
**Type:** OTHER
### Outcomes Module
#### Primary Outcomes
**Measure:** atopic patch test in atopic dermatitis children
**Time Frame:** 1 year
### Eligibility Module
**Eligibility Criteria:** Inclusion Criteria:
* Patients who had history of atopic dermatitis
Exclusion Criteria:
* Who have dermographism
* Who have chronic disease eg. autoimmune disease, immune deficiency, cancer or allergic disease
* Pregnant women
* Who receive antihistamine, topical steroid and systemic steroid \> 20 mg/day within 7 days prior study
**Maximum Age:** 18 Years
**Minimum Age:** 1 Month
**Sampling Method:** NON_PROBABILITY_SAMPLE
**Sex:** ALL
**Standard Ages:**
- CHILD
- ADULT
**Study Population:** children with atopic dermatitis
## Derived Section
### Condition Browse Module - Ancestors
- ID: D000012871
- Term: Skin Diseases
- ID: D000012873
- Term: Skin Diseases, Genetic
- ID: D000030342
- Term: Genetic Diseases, Inborn
- ID: D000017443
- Term: Skin Diseases, Eczematous
- ID: D000006969
- Term: Hypersensitivity, Immediate
- ID: D000006967
- Term: Hypersensitivity
- ID: D000007154
- Term: Immune System Diseases
### Condition Browse Module - Browse Branches
- Abbrev: BC20
- Name: Immune System Diseases
- Abbrev: All
- Name: All Conditions
- Abbrev: BC17
- Name: Skin and Connective Tissue Diseases
- Abbrev: BC16
- Name: Diseases and Abnormalities at or Before Birth
### Condition Browse Module - Browse Leaves
- ID: M10018
- Name: Hypersensitivity
- Relevance: LOW
- As Found: Unknown
- ID: M7067
- Name: Dermatitis
- Relevance: HIGH
- As Found: Dermatitis
- ID: M7655
- Name: Eczema
- Relevance: HIGH
- As Found: Atopic Dermatitis
- ID: M7071
- Name: Dermatitis, Atopic
- Relevance: HIGH
- As Found: Atopic Dermatitis
- ID: M8636
- Name: Food Hypersensitivity
- Relevance: HIGH
- As Found: Food Allergy
- ID: M15674
- Name: Skin Diseases
- Relevance: LOW
- As Found: Unknown
- ID: M15676
- Name: Skin Diseases, Genetic
- Relevance: LOW
- As Found: Unknown
- ID: M23686
- Name: Genetic Diseases, Inborn
- Relevance: LOW
- As Found: Unknown
- ID: M19712
- Name: Skin Diseases, Eczematous
- Relevance: LOW
- As Found: Unknown
- ID: M10020
- Name: Hypersensitivity, Immediate
- Relevance: LOW
- As Found: Unknown
- ID: M10200
- Name: Immune System Diseases
- Relevance: LOW
- As Found: Unknown
### Condition Browse Module - Meshes
- ID: D000003876
- Term: Dermatitis, Atopic
- ID: D000003872
- Term: Dermatitis
- ID: D000004485
- Term: Eczema
- ID: D000005512
- Term: Food Hypersensitivity
### Misc Info Module
- Version Holder: 2024-05-24
|