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## Protocol Section ### Identification Module **NCT ID:** NCT01888679 **Brief Title:** Influence of Head Positioning on the Movement of the Endotracheal Tube **Official Title:** Influence of Head Positioning on the Movement of the Endotracheal Tube #### Organization Study ID Info **ID:** 229/12 #### Organization **Class:** OTHER **Full Name:** University of Lausanne Hospitals ### Status Module #### Completion Date **Date:** 2013-03 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2020-03-02 **Type:** ACTUAL **Last Update Submit Date:** 2020-02-27 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2013-03 **Type:** ACTUAL #### Start Date **Date:** 2012-08 **Status Verified Date:** 2020-02 #### Study First Post Date **Date:** 2013-06-28 **Type:** ESTIMATED **Study First Submit Date:** 2013-06-21 **Study First Submit QC Date:** 2013-06-27 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Lausanne Hospitals #### Responsible Party **Investigator Affiliation:** University of Lausanne Hospitals **Investigator Full Name:** Patrick Schoettker,MD PD **Investigator Title:** MD **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to evaluate and measure the movement of the endotracheal tube depending on the type and the amount of movement of the head of the patient intubated using a flexible fiberoptic. **Detailed Description:** We performed a maximal head-neck movement trial on 50 adult patients, American Society of Anaesthesiologists 1 or 2. Patients with body mass index N35 kg · m-2, height b150 cm, airway malformations, pulmonary diseases, difficulties in neck flexion or extension, previous ENT surgery or radiotherapy, gastroesophageal reflux, or dental instability were excluded from the study. We measured the change in distance between the ETT tip and the carina, using a fiberscope through the ETT. ### Conditions Module **Conditions:** - Other Conditions That May Be A Focus of Clinical Attention **Keywords:** - Airway - Endotracheal intubation - selective intubation ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### 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:** head movement in extension, flexion, right and left rotation **Intervention Names:** - Procedure: Head movement **Label:** head movement **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - head movement **Description:** Head movement in extension, flexion, right and left rotation **Name:** Head movement **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Description:** Tube movement related to movement of the head **Measure:** Displacement of the endotracheal tube. **Time Frame:** 24 hours ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Patients older than 16 years * ASA 1-2 * Elective surgery Exclusion Criteria: * Pathological changes of the trachea. * Less than BMI 35 kg/m2. * Patients with rheumatoid arthritis or other limitation to cervical mobility. * Emergency surgery. * Individuals measuring less than 150 cm. **Minimum Age:** 16 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Lausanne **Country:** Switzerland **Facility:** University of Lausanne Hospitals **State:** Vaud **Zip:** 1011 #### Overall Officials **Official 1:** **Affiliation:** University of Lausanne Hospitals **Name:** Patrick Schoettker, MD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Tailleur R, Bathory I, Dolci M, Frascarolo P, Kern C, Schoettker P. Endotracheal tube displacement during head and neck movements. Observational clinical trial. J Clin Anesth. 2016 Aug;32:54-8. doi: 10.1016/j.jclinane.2015.12.043. Epub 2016 Mar 22. **PMID:** 27290945 ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00297479 **Brief Title:** Group Therapy for Nicotine Dependence: Mindfulness and Smoking **Official Title:** Group Therapy for Nicotine Dependence #### Organization Study ID Info **ID:** 2004-0988 #### Organization **Class:** OTHER **Full Name:** M.D. Anderson Cancer Center #### Secondary ID Infos **ID:** R01DA018875 **Link:** https://reporter.nih.gov/quickSearch/R01DA018875 **Type:** NIH **Domain:** NCI CTRP **ID:** NCI-2012-02081 **Type:** REGISTRY ### Status Module #### Completion Date **Date:** 2016-09 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2020-06-16 **Type:** ACTUAL **Last Update Submit Date:** 2020-06-03 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2016-09 **Type:** ACTUAL #### Results First Post Date **Date:** 2020-06-16 **Type:** ACTUAL **Results First Submit Date:** 2020-02-03 **Results First Submit QC Date:** 2020-06-03 #### Start Date **Date:** 2005-04 **Status Verified Date:** 2020-06 #### Study First Post Date **Date:** 2006-02-28 **Type:** ESTIMATED **Study First Submit Date:** 2006-02-24 **Study First Submit QC Date:** 2006-02-24 ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** National Institute on Drug Abuse (NIDA) #### Lead Sponsor **Class:** OTHER **Name:** M.D. Anderson Cancer Center #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The goal of this behavioral research study is to create and study a Mindfulness-Based Addiction Treatment (MBAT) for nicotine dependence. Mindfulness is a method to help focus attention on being in the "here and now." It can be learned through training in how to control one's attention. It is usually taught through meditation. The overarching goals of the study are to evaluate the efficacy of MBAT for nicotine dependence and the mechanisms and effects posited to mediate MBAT's impact on abstinence. **Detailed Description:** This 3-group randomized clinical trial will develop and evaluate a Mindfulness-Based Addiction Treatment (MBAT) for nicotine dependence. Mindfulness reflects a purposeful control of attention and can be learned through training in attentional control procedures. Current cigarette smokers (N=550; 400 in formal study; up to 80-150 pilot) will be randomly assigned to Usual Care (UC), Standard Treatment (ST) or MBAT. UC will be four 5-10 minute counseling sessions following the problem-solving approach in the Treating Tobacco Use and Dependence Clinical Practice Guideline (Guideline). ST is a standard smoking cessation group program using a problem-solving/coping skills approach. MBAT is a group smoking cessation program derived from Mindfulness-Based Stress Reduction (MBSR) and Mindfulness-Based Cognitive Therapy. MBAT will not alter the basic mindfulness approach used in MBCT and MBSR, but will replace depression-related material with smoking cessation strategies from the Guideline. All participants will receive nicotine patches and self-help materials. MBAT mechanisms and effects will be assessed using "implicit" cognitive psychological measures and computer-administered questionnaires. Participants will be tracked from baseline through 4 (UC) or 8 (ST and MBAT) treatment visits and follow-up visits 1 and 23 weeks post-treatment. The overarching goals are to evaluate MBAT's efficacy for nicotine dependence and the mechanisms and effects posited to mediate MBAT's impact on abstinence. Primary specific aims are to: 1. Examine the effects of MBAT on abstinence rates 2. Examine the effects of MBAT on mindfulness/metacognitive awareness, attentional control, smoking automaticity, smoking associations in memory, negative affect, depression, stress, affect regulation expectancies, self-efficacy, withdrawal, and coping across the pre- and post-cessation period, and whether these variables mediate MBAT effects on abstinence. ### Conditions Module **Conditions:** - Smoking - Tobacco Use Cessation **Keywords:** - Tobacco Cessation - Smoking Cessation ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 650 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** MBAT is 6 weeks of nicotine patch therapy; a Self-help guide; and In-person group therapy/counseling (8 sessions over 8 weeks) using a Mindfulness-Based Addiction Treatment for nicotine dependence. **Intervention Names:** - Behavioral: MBAT Group Therapy - Drug: Nicotine **Label:** Mindfulness-Based Treatment Group (MBAT) **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Standard Care Group (ST) is 6 weeks of nicotine patch therapy, a Self-help guide and In-person group therapy/counseling (8 sessions over 8 weeks) based upon Treating Tobacco Use and Dependence Clinical Practice Guideline **Intervention Names:** - Drug: Nicotine - Behavioral: Group Therapy **Label:** Standard Care Group **Type:** ACTIVE_COMPARATOR #### Arm Group 3 **Description:** Usual Care (UC) is 6 weeks of nicotine patch therapy, a Self-help guide and In-person individual counseling (4 sessions over 8 weeks) based upon Treating Tobacco Use and Dependence Clinical Practice Guideline **Intervention Names:** - Drug: Nicotine - Behavioral: Individual Therapy **Label:** Usual Care Group **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Mindfulness-Based Treatment Group (MBAT) **Description:** In-person group therapy/counseling (8 sessions over 8 weeks) using a Mindfulness-Based Addiction Treatment for nicotine dependence **Name:** MBAT Group Therapy **Other Names:** - therapy - counseling **Type:** BEHAVIORAL #### Intervention 2 **Arm Group Labels:** - Mindfulness-Based Treatment Group (MBAT) - Standard Care Group - Usual Care Group **Description:** 6 weeks of nicotine patch therapy **Name:** Nicotine **Other Names:** - nicotine patch therapy **Type:** DRUG #### Intervention 3 **Arm Group Labels:** - Standard Care Group **Description:** In-person group therapy/counseling (8 sessions over 8 weeks) based upon Treating Tobacco Use and Dependence Clinical Practice Guideline **Name:** Group Therapy **Other Names:** - counseling **Type:** BEHAVIORAL #### Intervention 4 **Arm Group Labels:** - Usual Care Group **Description:** In-person individual counseling (4 sessions over 8 weeks) based upon Treating Tobacco Use and Dependence Clinical Practice Guideline **Name:** Individual Therapy **Other Names:** - counseling **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** Biochemically verified 7-day point prevalence abstinence rates based on a completers-only approach. **Measure:** Number of Participants With Smoking Abstinence **Time Frame:** 4 weeks post quit day (one week following the end of treatment) **Description:** Biochemically verified 7-day point prevalence abstinence rates using an intent-to-treat approach **Measure:** Smoking Abstinence **Time Frame:** 26 weeks post quit day **Description:** Biochemically verified 7-day point prevalence abstinence rates using an intent-to-treat approach **Measure:** Smoking Abstinence **Time Frame:** 4 weeks post quit day (one week following the end of treatment) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Age 18 or above 2. Current smoker with a history of at least five cigarettes/day for the past year 3. Motivated to quit within the next 30 days (preparation stage) 4. Participants must provide a viable home address and a functioning home telephone number 5. Can read and write in English 6. Register "8" or more on a carbon monoxide breath test 7. Provide viable collateral contact information Exclusion Criteria: 1. Contraindication for nicotine patch use 2. Regular use of tobacco products other than cigarettes (cigars, pipes, smokeless tobacco) 3. Use of bupropion or nicotine patch replacement products other than the study patches 4. Pregnancy or lactation 5. Another household member enrolled in the study 6. Active substance dependence (exclusive of nicotine dependence) 7. Current psychiatric disorder; current use of psychotropic medication 8. Participation in a smoking cessation program or study during the past 90 days **Healthy Volunteers:** True **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Houston **Country:** United States **Facility:** University of Texas MD Anderson Cancer Center **State:** Texas **Zip:** 77030 #### Overall Officials **Official 1:** **Affiliation:** M.D. Anderson Cancer Center **Name:** David W Wetter, Ph.D. **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Jackson S, Brown J, Norris E, Livingstone-Banks J, Hayes E, Lindson N. Mindfulness for smoking cessation. Cochrane Database Syst Rev. 2022 Apr 14;4(4):CD013696. doi: 10.1002/14651858.CD013696.pub2. **PMID:** 35420700 **Citation:** Vidrine JI, Businelle MS, Reitzel LR, Cao Y, Cinciripini PM, Marcus MT, Li Y, Wetter DW. Coping Mediates the Association of Mindfulness with Psychological Stress, Affect, and Depression Among Smokers Preparing to Quit. Mindfulness (N Y). 2015 Jun;6(3):433-443. doi: 10.1007/s12671-014-0276-4. Epub 2014 Jan 18. **PMID:** 28191263 #### See Also Links **Label:** University of Texas MD Anderson Cancer Center Website **URL:** http://www.mdanderson.org ## Derived Section ### Condition Browse Module - Ancestors - ID: D000019966 - Term: Substance-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: M16785 - Name: Tobacco Use Disorder - Relevance: HIGH - As Found: Nicotine Dependence - ID: M21837 - Name: Substance-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: D000014029 - Term: Tobacco Use Disorder ### Intervention Browse Module - Ancestors - ID: D000005731 - Term: Ganglionic Stimulants - ID: D000001337 - Term: Autonomic Agents - ID: D000018373 - Term: Peripheral Nervous System Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018722 - Term: Nicotinic Agonists - ID: D000018679 - Term: Cholinergic Agonists - ID: D000018678 - Term: Cholinergic Agents - ID: D000018377 - Term: Neurotransmitter Agents - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: CNSSti - Name: Central Nervous System Stimulants ### Intervention Browse Module - Browse Leaves - ID: M12478 - Name: Nicotine - Relevance: HIGH - As Found: Prior to - ID: M4029 - Name: Central Nervous System Stimulants - Relevance: LOW - As Found: Unknown - ID: M20796 - Name: Nicotinic Agonists - Relevance: LOW - As Found: Unknown - ID: M20758 - Name: Cholinergic Agents - Relevance: LOW - As Found: Unknown - ID: M20504 - Name: Neurotransmitter Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000009538 - Term: Nicotine ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** MD Anderson requires that all Protocol PIs and their designees report all Serious Adverse Events.No SAE's nor Adverse Events occurred in this protocol to the MD Anderson IRB on an ongoing bases using a systematic approach with standardized forms. SAE's and AE's are reported as they occur and during each continuation report submission. 238 participants were registered under the pilot phase which were not included in the formal clinical trial and no data collected.participants. #### Event Groups **Group ID:** EG000 **Title:** Usual Care Group **Description:** Usual Care (UC) 6 weeks of nicotine patch therapy, Self-help guide \& In-person individual counseling (4 sessions over 8 weeks) **ID:** EG000 **Other Num at Risk:** 103 **Serious Number At Risk:** 103 **Title:** Usual Care Group **Group ID:** EG001 **Title:** Standard Care Group **Description:** Standard Care Group (ST) 6 weeks of nicotine patch therapy, Self-help guide \& In-person group therapy/counseling (8 sessions over 8 weeks) **ID:** EG001 **Other Num at Risk:** 155 **Serious Number At Risk:** 155 **Title:** Standard Care Group **Group ID:** EG002 **Title:** Mindfulness-Based Treatment Group (MBAT) **Description:** MBAT 6 weeks of nicotine patch therapy; Self-help guide; \& In-person group therapy/counseling (8 sessions over 8 weeks) using a MBAT for nicotine dependence. **ID:** EG002 **Other Num at Risk:** 154 **Serious Number At Risk:** 154 **Title:** Mindfulness-Based Treatment Group (MBAT) **Frequency Threshold:** 0 **Time Frame:** Adverse event data collected up to 32 weeks of treatment. ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 103 **Group ID:** BG001 **Value:** 155 **Group ID:** BG002 **Value:** 154 **Group ID:** BG003 **Value:** 412 **Units:** Participants ### Group **ID:** BG000 **Title:** Usual Care Group (UC) **Description:** 6 wks of nicotine patch therapy, self-help guide and in-person individual counseling (4 sessions in 8 weeks) based on Tobacco Use and Dependence Clinical Practice Guideline ### Group **ID:** BG001 **Title:** Cognitive Behavioral Treatment (CBT) **Description:** 6 wks of nicotine patch therapy, self-help guide and in-person group counseling (8 sessions in 8 weeks) based on Treating Tobacco Use and Dependence Clinical Practice Guideline ### Group **ID:** BG002 **Title:** Mindfulness-Based Treatment Group (MBAT) **Description:** 6 wks of nicotine patch therapy; self-help guide; and in-person group counseling (8 sessions in 8 weeks) using a Mindfulness-Based Addiction Treatment for nicotine dependence ### 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:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 0 **Category Title:** <=18 years #### Measurement **Group ID:** BG000 **Value:** 103 #### Measurement **Group ID:** BG001 **Value:** 155 #### Measurement **Group ID:** BG002 **Value:** 154 #### Measurement **Group ID:** BG003 **Value:** 412 **Category Title:** Between 18 and 65 years #### 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:** >=65 years **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 12.5 **Value:** 49 #### Measurement **Group ID:** BG001 **Spread:** 11.9 **Value:** 48.8 #### Measurement **Group ID:** BG002 **Spread:** 11.7 **Value:** 48.4 #### Measurement **Group ID:** BG003 **Spread:** 11.9 **Value:** 48.7 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 58 #### Measurement **Group ID:** BG001 **Value:** 84 #### Measurement **Group ID:** BG002 **Value:** 84 #### Measurement **Group ID:** BG003 **Value:** 226 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 45 #### Measurement **Group ID:** BG001 **Value:** 71 #### Measurement **Group ID:** BG002 **Value:** 70 #### Measurement **Group ID:** BG003 **Value:** 186 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 103 #### Measurement **Group ID:** BG001 **Value:** 155 #### Measurement **Group ID:** BG002 **Value:** 154 #### Measurement **Group ID:** BG003 **Value:** 412 **Class Title:** United States ### Measure #### Measurement **Group ID:** BG000 **Spread:** 4.0 **Value:** 31.9 #### Measurement **Group ID:** BG001 **Spread:** 3.6 **Value:** 31.9 #### Measurement **Group ID:** BG002 **Spread:** 3.8 **Value:** 32.0 #### Measurement **Group ID:** BG003 **Spread:** 3.8 **Value:** 32.0 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** .9 **Value:** 4.2 #### Measurement **Group ID:** BG001 **Spread:** .9 **Value:** 4.3 #### Measurement **Group ID:** BG002 **Spread:** 1.0 **Value:** 4.3 #### Measurement **Group ID:** BG003 **Spread:** .9 **Value:** 4.3 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 8.6 **Value:** 19.3 #### Measurement **Group ID:** BG001 **Spread:** 9.3 **Value:** 19.9 #### Measurement **Group ID:** BG002 **Spread:** 11.7 **Value:** 20.3 #### Measurement **Group ID:** BG003 **Spread:** 10.1 **Value:** 19.9 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 39 #### Measurement **Group ID:** BG001 **Value:** 61 #### Measurement **Group ID:** BG002 **Value:** 59 #### Measurement **Group ID:** BG003 **Value:** 159 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 10 #### Measurement **Group ID:** BG001 **Value:** 13 #### Measurement **Group ID:** BG002 **Value:** 14 #### Measurement **Group ID:** BG003 **Value:** 37 **Class Title:** < High School #### Measurement **Group ID:** BG000 **Value:** 25 #### Measurement **Group ID:** BG001 **Value:** 37 #### Measurement **Group ID:** BG002 **Value:** 39 #### Measurement **Group ID:** BG003 **Value:** 101 **Class Title:** High School/GED #### Measurement **Group ID:** BG000 **Value:** 52 #### Measurement **Group ID:** BG001 **Value:** 76 #### Measurement **Group ID:** BG002 **Value:** 67 #### Measurement **Group ID:** BG003 **Value:** 195 **Class Title:** Some College #### Measurement **Group ID:** BG000 **Value:** 10 #### Measurement **Group ID:** BG001 **Value:** 17 #### Measurement **Group ID:** BG002 **Value:** 28 #### Measurement **Group ID:** BG003 **Value:** 55 **Class Title:** 4-year College Degree #### Measurement **Group ID:** BG000 **Value:** 6 #### Measurement **Group ID:** BG001 **Value:** 11 #### Measurement **Group ID:** BG002 **Value:** 6 #### Measurement **Group ID:** BG003 **Value:** 23 **Class Title:** > 4-year College Degree ### Measure #### Measurement **Group ID:** BG000 **Value:** 61 #### Measurement **Group ID:** BG001 **Value:** 89 #### Measurement **Group ID:** BG002 **Value:** 87 #### Measurement **Group ID:** BG003 **Value:** 237 **Class Title:** < $30,000/year #### Measurement **Group ID:** BG000 **Value:** 42 #### Measurement **Group ID:** BG001 **Value:** 66 #### Measurement **Group ID:** BG002 **Value:** 67 #### Measurement **Group ID:** BG003 **Value:** 175 **Class Title:** > $30,000/year ### Measure #### Measurement **Group ID:** BG000 **Value:** 76 #### Measurement **Group ID:** BG001 **Value:** 103 #### Measurement **Group ID:** BG002 **Value:** 109 #### Measurement **Group ID:** BG003 **Value:** 288 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 22 #### Measurement **Group ID:** BG001 **Value:** 27 #### Measurement **Group ID:** BG002 **Value:** 22 #### Measurement **Group ID:** BG003 **Value:** 71 **Class Title:** **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 ### Measure 5 **Description:** The Kentucky Inventory of Mindfulness Skills (KIMS) is a self-report inventory for the assessment of mindfulness skills (including subscales for observing (15-59), describing (10-40), acting with awareness (15-48), and accept(13-45) . Items are rated on a 5 point Likert scale ranging from 1 (never or very rarely true) to 5 (almost always or always true). Items reflect either direct descriptions of the mindfulness skills, or they describe the absence of that skill and are reverse scored. The minimum score 10 and maximum score 59. Higher-scores reflect more mindfulness. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Kentucky Inventory of Mindfulness Skills (KIMS) **Unit of Measure:** KIMS Score ### Measure 6 **Description:** The Mindful Attention Awareness Scale (MAAS) is a 15 item self-report questionnaire measuring mindfulness. To score the scale, simply compute a mean (average) of the 15 items. Items are rated on using the 1-6 scale ranging from 1 (almost always) to 6 (almost never). Indicates currently how frequently or infrequently each experience. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Mindful Attention Awareness Scale (MAAS) **Unit of Measure:** MAAS Score ### Measure 7 **Description:** Nicotine Dependence was assessed at baseline using the Heaviness of Smoking Index (HIS). The HSI comprises the two items from the Fagerstrom Test for Nicotine Dependence (FTND) that most strongly predict smoking relapse, cigarettes per day (CPD) and minutes to the first cigarette after waking. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Nicotine Dependence - Cigarettes per day **Unit of Measure:** Cigarettes per day ### Measure 8 **Description:** Nicotine Dependence was assessed at baseline using the Heaviness of Smoking Index (HIS). The HSI comprises the two items from the Fagerstrom Test for Nicotine Dependence (FTND) that most strongly predict smoking relapse, cigarettes per day (CPD) and minutes to the first cigarette after waking. **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Nicotine Dependence - Participants reported smoking their first cigarette with 5 minutes of walking **Unit of Measure:** Participants ### Measure 9 **Description:** Years of Education **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Demographics (Education) **Unit of Measure:** Participants ### Measure 10 **Description:** Income: self-reported household income **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Demographics (Household Income) **Unit of Measure:** Participants ### Measure 11 **Description:** Relationship status: self-report of participants with no partner **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Demographics (Spouse/Partner) **Unit of Measure:** Participants ### Measure 12 **Description:** The Center of Epidemiologic Studies Depression Scale (CES-D) assesses depressive symptoms in community non-clinical populations **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Demographics (History of Depression) **Unit of Measure:** Participants **Population Description:** 238 were registered under the pilot phase which were not included in the formal clinical trial and no data collected for those 238 participants. ## Results Section - More Information Module ### Certain Agreement **PI Sponsor Employee:** True ### Point of Contact **Email:** [email protected] **Organization:** UT MD Anderson Cancer Center **Phone:** 713-563-0020 **Title:** Dr. Sharon Giordano, MD/Chair, Health Svcs Research-Clinical ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ### Outcome Measure 4 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 21 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 39 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 43 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 13 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 24 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 20 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 25 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 50 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 52 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 12 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 24 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 53 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Biochemically verified 7-day point prevalence abstinence rates based on a completers-only approach. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Completers-only **Reporting Status:** POSTED **Time Frame:** 4 weeks post quit day (one week following the end of treatment) **Title:** Number of Participants With Smoking Abstinence **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** 6 wks of nicotine patch therapy, self-help guide and in-person individual counseling (4 sessions in 8 weeks) based on Tobacco Use and Dependence Clinical Practice Guideline **ID:** OG000 **Title:** Usual Care Group (UC) ##### Group **Description:** 6 wks of nicotine patch therapy, self-help guide and in-person group counseling (8 sessions in 8 weeks) based on Treating Tobacco Use and Dependence Clinical Practice Guideline **ID:** OG001 **Title:** Cognitive Behavioral Treatment (CBT) ##### Group **Description:** 6 wks of nicotine patch therapy; self-help guide; and in-person group counseling (8 sessions in 8 weeks) using a Mindfulness-Based Addiction Treatment for nicotine dependence **ID:** OG002 **Title:** Mindfulness-Based Treatment Group (MBAT) #### Outcome Measure 2 **Description:** Biochemically verified 7-day point prevalence abstinence rates using an intent-to-treat approach **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Completers Only **Reporting Status:** POSTED **Time Frame:** 26 weeks post quit day **Title:** Smoking Abstinence **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** 6 wks of nicotine patch therapy, self-help guide and in-person individual counseling (4 sessions in 8 weeks) based on Tobacco Use and Dependence Clinical Practice Guideline **ID:** OG000 **Title:** Usual Care Group (UC) ##### Group **Description:** 6 wks of nicotine patch therapy, self-help guide and in-person group counseling (8 sessions in 8 weeks) based on Treating Tobacco Use and Dependence Clinical Practice Guideline **ID:** OG001 **Title:** Cognitive Behavioral Treatment (CBT) ##### Group **Description:** 6 wks of nicotine patch therapy; self-help guide; and in-person group counseling (8 sessions in 8 weeks) using a Mindfulness-Based Addiction Treatment for nicotine dependence **ID:** OG002 **Title:** Mindfulness-Based Treatment Group (MBAT) #### Outcome Measure 3 **Description:** Biochemically verified 7-day point prevalence abstinence rates using an intent-to-treat approach **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Intent to treat **Reporting Status:** POSTED **Time Frame:** 4 weeks post quit day (one week following the end of treatment) **Title:** Smoking Abstinence **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** 6 wks of nicotine patch therapy, self-help guide and in-person individual counseling (4 sessions in 8 weeks) based on Tobacco Use and Dependence Clinical Practice Guideline **ID:** OG000 **Title:** Usual Care Group (UC) ##### Group **Description:** 6 wks of nicotine patch therapy, self-help guide and in-person group counseling (8 sessions in 8 weeks) based on Treating Tobacco Use and Dependence Clinical Practice Guideline **ID:** OG001 **Title:** Cognitive Behavioral Treatment (CBT) ##### Group **Description:** 6 wks of nicotine patch therapy; self-help guide; and in-person group counseling (8 sessions in 8 weeks) using a Mindfulness-Based Addiction Treatment for nicotine dependence **ID:** OG002 **Title:** Mindfulness-Based Treatment Group (MBAT) #### Outcome Measure 4 **Description:** Biochemically verified 7-day point prevalence abstinence rates using an intent-to-treat approach **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Intent to treat **Reporting Status:** POSTED **Time Frame:** 26 weeks post quit day **Title:** Smoking Abstinence **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** 6 wks of nicotine patch therapy, self-help guide and in-person individual counseling (4 sessions in 8 weeks) based on Tobacco Use and Dependence Clinical Practice Guideline **ID:** OG000 **Title:** Usual Care Group (UC) ##### Group **Description:** 6 wks of nicotine patch therapy, self-help guide and in-person group counseling (8 sessions in 8 weeks) based on Treating Tobacco Use and Dependence Clinical Practice Guideline **ID:** OG001 **Title:** Cognitive Behavioral Treatment (CBT) ##### Group **Description:** 6 wks of nicotine patch therapy; self-help guide; and in-person group counseling (8 sessions in 8 weeks) using a Mindfulness-Based Addiction Treatment for nicotine dependence **ID:** OG002 **Title:** Mindfulness-Based Treatment Group (MBAT) ### Participant Flow Module #### Group **Description:** Usual Care (UC) is 6 weeks of nicotine patch therapy, a Self-help guide and In-person individual counseling (4 sessions over 8 weeks) based upon Treating Tobacco Use and Dependence Clinical Practice Guideline **ID:** FG000 **Title:** Usual Care Group #### Group **Description:** Standard Care Group (ST) is 6 weeks of nicotine patch therapy, a Self-help guide and In-person group therapy/counseling (8 sessions over 8 weeks) based upon Treating Tobacco Use and Dependence Clinical Practice Guideline **ID:** FG001 **Title:** Cognitive Behavioral Treatment (CBT) #### Group **Description:** MBAT is 6 weeks of nicotine patch therapy; a Self-help guide; and In-person group therapy/counseling (8 sessions over 8 weeks) using a Mindfulness-Based Addiction Treatment for nicotine dependence. **ID:** FG002 **Title:** Mindfulness-Based Treatment Group (MBAT) #### Period **Title:** Overall Study ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 37 ###### Reason **Group ID:** FG001 **Number of Subjects:** 54 ###### Reason **Group ID:** FG002 **Number of Subjects:** 51 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 103 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 155 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 154 ##### Milestone **Type:** 4-Week Follow Up ###### Achievement **Group ID:** FG000 **Number of Subjects:** 78 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 132 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 128 ##### Milestone **Type:** 26- Week Follow Up ###### Achievement **Group ID:** FG000 **Number of Subjects:** 66 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 101 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 103 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 66 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 101 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 103 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 37 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 54 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 51 **Pre-Assignment Details:** Out of the 650 participants, 412 were on the formal clinical trial and 238 were registered under the pilot phase which were not included in the formal clinical trial and no data collected for those 238 participants. **Recruitment Details:** Participants were recruited from the Houston metropolitan area via local print media. All data collected between January 2007 to February 2010. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT02753179 **Acronym:** COSQOL **Brief Title:** Covert-saccades, Dynamic Visual Acuity and Quality of Life **Official Title:** Quality of Life and Dynamic Visual Acuity in Patients With Bilateral Vestibulopathy: the Impact of Covert-saccades #### Organization Study ID Info **ID:** 69HCL15_0746 #### Organization **Class:** OTHER **Full Name:** Hospices Civils de Lyon ### Status Module #### Completion Date **Date:** 2017-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-01-16 **Type:** ESTIMATED **Last Update Submit Date:** 2017-01-13 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2017-01 **Type:** ACTUAL #### Start Date **Date:** 2016-05 **Status Verified Date:** 2017-01 #### Study First Post Date **Date:** 2016-04-27 **Type:** ESTIMATED **Study First Submit Date:** 2016-04-25 **Study First Submit QC Date:** 2016-04-26 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Hospices Civils de Lyon #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Patients with chronic bilateral vestibular hypofunction may suffer from a visual instability during head movement called oscillopsia. Visual consequence of vestibular deficit can lead to a severe impairment of their quality of life. However, correcting saccades during rapid head movement, called covert-saccades, have been more recently identified. These saccades, which occur during the head movement in patients with vestibular hypofunction, present a very short latency. They could compensate for the lack of vestibular-ocular reflex and greatly decrease oscillopsia and visual impairment. The objective of this study is to evaluate the potential functional benefice of these compensatory movements in a population of 20 patients with chronic bilateral areflexia, in a cross-sectional study. ### Conditions Module **Conditions:** - Vestibular Diseases **Keywords:** - Bilateral vestibular hypofunction - Video Head Impulse Test - Covert-saccades - Quality of life - Dynamic visual acuity ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** BASIC_SCIENCE #### Enrollment Info **Count:** 20 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients suffering from chronic bilateral vestibular hypofunction. **Intervention Names:** - Other: Head Impulse Tests - Other: Dynamic visual acuity Test (DVAT) - Other: VEMPs & VEMPo - Other: Dizziness Handicap Inventory - Other: Oscillopsia severity questionnaire **Label:** Bilateral vestibular hypofunction **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Bilateral vestibular hypofunction **Description:** Head Impulse Tests are performed by the clinician who holds the patient's head in his hands, while he is looking straight at an earth-fixed target; then by turning the patient's head abruptly and unpredictably to the left or right, up or down through a small angle (only 10-20 degrees - not a large angle). 20 impulses in each directions (6) will be performed. **Name:** Head Impulse Tests **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - Bilateral vestibular hypofunction **Description:** Dynamic visual acuity Test (DVAT) assesses visual acuity during head movement relative to baseline static visual acuity. DVA will be assessed actively during self-generated rotations of the head in different directions. **Name:** Dynamic visual acuity Test (DVAT) **Type:** OTHER #### Intervention 3 **Arm Group Labels:** - Bilateral vestibular hypofunction **Description:** VEMPs \& VEMPo are sound evoked muscular contractions of the neck or eye. They are recorded using an evoked response computer, a sound generator, and surface electrodes to pick up neck or eye muscle activation. **Name:** VEMPs & VEMPo **Type:** OTHER #### Intervention 4 **Arm Group Labels:** - Bilateral vestibular hypofunction **Description:** Dizziness Handicap Inventory is a questionnaire that identify difficulties that patient may be experiencing because of dizziness, yielding to a score ranging from 0 to 100 **Name:** Dizziness Handicap Inventory **Type:** OTHER #### Intervention 5 **Arm Group Labels:** - Bilateral vestibular hypofunction **Description:** Oscillopsia severity questionnaire is a 9 items questionnaires that identify oscillopsia in different circumstances. **Name:** Oscillopsia severity questionnaire **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Frequency of covert saccades corresponds to the total amount of covert-saccades divided by the total amount of head impulse tests multiplied by 100. **Measure:** Frequency of covert-saccades **Time Frame:** Day 0 #### Secondary Outcomes **Description:** Dizziness Handicap Inventory is a questionnaire that identify difficulties that patient may be experiencing because of dizziness, yielding to a score ranging from 0 to 100 **Measure:** Quality of life assessed with the Dizziness Handicap Inventory **Time Frame:** Day 0 **Description:** Dynamic visual acuity is a measure of threshold for binocular reading of letters that are presented on a screen during head impulse test **Measure:** Dynamic visual acuity **Time Frame:** day 0 **Description:** Oscillopsia severity questionnaire is a 9 items questionnaires that identify oscillopsia in different circumstances. A mean item score gives an oscillopsia severity score ranging from 1 to 5, yielding to a total score ranging from 0 to 45 **Measure:** Oscillopsia severity questionnaire score **Time Frame:** day 0 **Description:** Latency of covert saccades correspond to the time between the beginning of head impulse and the initiation of the first covert-saccade **Measure:** Latency of covert-saccades **Time Frame:** Day 0 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Bilateral Vestibular Hypofunction (BVH) with at least two of the tree following criteria * Mean peak slow phase velocity of 5°/s or less in bilateral bithermal (30 and 44°C) caloric irrigations * Pathologic Head-impulse test * VOR gain of \<0.25 on rotatory chair tests * Disorder present for over 6 month * Comprehension of the experiments instructions * Patient consent Exclusion Criteria: * Corrected Visual Acuity lower than 5/10 * Other conditions leading to oscillopsia or ataxia * Oculomotor palsy, ocular instability in primary position * Cervical rachis pathology with instability * Cochlear Implants * Non-stabilized medical disease * Pregnant women * Patients under tutelage **Maximum Age:** 90 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Bron **Country:** France **Facility:** Unité de Neuro-Ophtalmologie Hôpital Neurologique **Zip:** 69500 #### Overall Officials **Official 1:** **Affiliation:** Institut National de la Santé Et de la Recherche Médicale, France **Name:** Caroline TILIKETE, MD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007759 - Term: Labyrinth Diseases - ID: D000004427 - Term: Ear Diseases - ID: D000010038 - Term: Otorhinolaryngologic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC09 - Name: Ear, Nose, and Throat Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BXM - Name: Behaviors and Mental Disorders ### Condition Browse Module - Browse Leaves - ID: M18387 - Name: Vestibular Diseases - Relevance: HIGH - As Found: Vestibular Diseases - ID: M850 - Name: Bilateral Vestibulopathy - Relevance: LOW - As Found: Unknown - ID: M10779 - Name: Labyrinth Diseases - Relevance: LOW - As Found: Unknown - ID: M10782 - Name: Labyrinthitis - Relevance: LOW - As Found: Unknown - ID: M7601 - Name: Ear Diseases - Relevance: LOW - As Found: Unknown - ID: M12961 - Name: Otorhinolaryngologic Diseases - Relevance: LOW - As Found: Unknown - ID: T6034 - Name: Quality of Life - Relevance: HIGH - As Found: Quality of Life ### Condition Browse Module - Meshes - ID: D000015837 - Term: Vestibular Diseases ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01254279 **Brief Title:** Early Access to Cabazitaxel in Patients With Metastatic Hormone Refractory Prostate Cancer Previously Treated With a Docetaxel-containing Regimen **Official Title:** Multicentre, Single-arm, Open Label Clinical Trial Intended to Provide Early Access to Cabazitaxel in Patients With Metastatic Hormone Refractory Prostate Cancer Previously Treated With a Docetaxel-containing Regimen and to Document Safety of Cabazitaxel in These Patients #### Organization Study ID Info **ID:** CABAZ_C_05331 #### Organization **Class:** INDUSTRY **Full Name:** Sanofi #### Secondary ID Infos **ID:** 2010-021128-92 **Type:** EUDRACT_NUMBER **Domain:** UTN **ID:** U1111-1115-2476 **Type:** OTHER ### Status Module #### Completion Date **Date:** 2014-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2015-01-21 **Type:** ESTIMATED **Last Update Submit Date:** 2015-01-19 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2014-12 **Type:** ACTUAL #### Start Date **Date:** 2010-12 **Status Verified Date:** 2015-01 #### Study First Post Date **Date:** 2010-12-06 **Type:** ESTIMATED **Study First Submit Date:** 2010-12-02 **Study First Submit QC Date:** 2010-12-02 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Sanofi #### Responsible Party **Type:** SPONSOR ### Description Module **Brief Summary:** The purpose of this study is to allow patients similar to that evaluated in the TROPIC trial (NCT00417079), and Investigators access to cabazitaxel for the management of metastatic Hormone Refractory Prostate Cancer (mHRPC) in those patients who have progressed during or after docetaxel and to document the overall safety of cabazitaxel in these patients. Please note that in each country, patient recruitment will end when cabazitaxel becomes commercially available. ### Conditions Module **Conditions:** - Prostate Cancer Metastatic ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 984 **Type:** ACTUAL **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Cabazitaxel 25 mg/m² intravenously every 3 weeks, in combination with oral prednisone or prednisolone 10 mg daily **Intervention Names:** - Drug: CABAZITAXEL **Label:** Cabazitaxel **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Cabazitaxel **Description:** Pharmaceutical form: Concentrate For Solution For Infusion Route of administration: Intravenous **Name:** CABAZITAXEL **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** To provide early access to cabazitaxel in patients with metastatic hormone refractory prostate cancer previously treated with a docetaxel-containing regimen **Time Frame:** Up to 30 weeks #### Secondary Outcomes **Measure:** To document safety of cabazitaxel in these patients **Time Frame:** Up to 35 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion criteria: * Metastatic Hormone Refractory Prostate Cancer (mHRPC) previously treated with a docetaxel-containing regimen * Disease Progression during or after docetaxel-containing regimen for mHRPC * Surgical or medical castration * Eastern Cooperative Oncology Group (ECOG) Performance Status (PS): 0-2 * Life-expectancy ≥3 months * Adequate bone marrow, liver, and renal function: Neutrophils\> 1500 /mm3; Hemoglobin \> 10 g/dL; Platelets \> 100 x109/L; Bilirubin \< ULN; SGOT (AST) \< 1.5xULN; SGPT (ALT) \< 1.5xULN; Creatinine \< 1.5xULN. If creatinine 1.0 - 1.5 x ULN, creatinine clearance will be calculated according to CKD-EPI formula and patients with creatinine clearance \< 60 mL/min should be excluded. Exclusion criteria: * Prior radiotherapy to ≥ 40% of bone marrow * Prior radionuclide therapy (samarium-153, strontium-89, P-32...) * Prior surgery, radiation, chemotherapy, or other anti-cancer therapy within 4 weeks prior to enrollment * Active grade ≥2 peripheral neuropathy * Active grade ≥2 stomatitis * Active infection requiring systemic antibiotic or anti-fungal medication * Active cancer (other than mHRPC) including prior malignancy from which the patient has been disease-free for ≤5 years (except superficial basal cell skin cancer) * Known brain or leptomeningeal involvement * History of severe hypersensitivity reaction (≥grade 3) to docetaxel * History of severe hypersensitivity reaction (≥grade 3) to polysorbate 80 containing drugs * History of severe hypersensitivity reaction (≥grade 3) or intolerance to prednisone or prednisolone * Uncontrolled severe illness or medical condition (including uncontrolled cardiac arrhythmias, angina pectoris, hypertension or diabetes mellitus). History of congestive heart failure (NYHA III or IV) or myocardial infarction within last 6 months is also not allowed. * Concurrent or planned treatment with potent inhibitors or inducers of cytochrome P450 3A4/5 * Participation in a clinical trial with any investigational drug * Patient with reproductive potential not implementing accepted and effective method of contraception The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial. **Minimum Age:** 18 Years **Sex:** MALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Box Hill **Country:** Australia **Facility:** Investigational Site Number 036004 **Zip:** 3128 **Location 2:** **City:** Campbelltown **Country:** Australia **Facility:** Investigational Site Number 036020 **Zip:** 2560 **Location 3:** **City:** Camperdown **Country:** Australia **Facility:** Investigational Site Number 036001 **Zip:** 2050 **Location 4:** **City:** Camperdown **Country:** Australia **Facility:** Investigational Site Number 036002 **Zip:** 2050 **Location 5:** **City:** Douglas **Country:** Australia **Facility:** Investigational Site Number 036011 **Zip:** 4814 **Location 6:** **City:** Nambour **Country:** Australia **Facility:** Investigational Site Number 036019 **Zip:** 4560 **Location 7:** **City:** North Adelaide **Country:** Australia **Facility:** Investigational Site Number 036023 **Zip:** 5006 **Location 8:** **City:** Parkville **Country:** Australia **Facility:** Investigational Site Number 036008 **Zip:** 3050 **Location 9:** **City:** Perth **Country:** Australia **Facility:** Investigational Site Number 036005 **Location 10:** **City:** Port Macquarie **Country:** Australia **Facility:** Investigational Site Number 036003 **Location 11:** **City:** Randwick **Country:** Australia **Facility:** Investigational Site Number 036025 **Zip:** 2031 **Location 12:** **City:** South Brisbane **Country:** Australia **Facility:** Investigational Site Number 036009 **Zip:** 4101 **Location 13:** **City:** South Brisbane **Country:** Australia **Facility:** Investigational Site Number 036022 **Zip:** 4101 **Location 14:** **City:** Southport **Country:** Australia **Facility:** Investigational Site Number 036017 **Zip:** 4215 **Location 15:** **City:** St Leonards **Country:** Australia **Facility:** Investigational Site Number 036012 **Zip:** 2065 **Location 16:** **City:** Sydney **Country:** Australia **Facility:** Investigational Site Number 036010 **Zip:** 2076 **Location 17:** **City:** Sydney **Country:** Australia **Facility:** Investigational Site Number 036013 **Location 18:** **City:** Sydney **Country:** Australia **Facility:** Investigational Site Number 036014 **Location 19:** **City:** Tweed Heads **Country:** Australia **Facility:** Investigational Site Number 036016 **Location 20:** **City:** Wendouree **Country:** Australia **Facility:** Investigational Site Number 036024 **Zip:** 3355 **Location 21:** **City:** Westmead **Country:** Australia **Facility:** Investigational Site Number 036007 **Zip:** 2145 **Location 22:** **City:** Wodonga **Country:** Australia **Facility:** Investigational Site Number 036015 **Location 23:** **City:** Linz **Country:** Austria **Facility:** Investigational Site Number 040-004 **Zip:** 4010 **Location 24:** **City:** Salzburg **Country:** Austria **Facility:** Investigational Site Number 040-005 **Location 25:** **City:** Vienna **Country:** Austria **Facility:** Investigational Site Number 040-003 **Location 26:** **City:** Wien **Country:** Austria **Facility:** Investigational Site Number 040-002 **Zip:** 1090 **Location 27:** **City:** Brugge **Country:** Belgium **Facility:** Investigational Site Number 056002 **Zip:** 8000 **Location 28:** **City:** Bruxelles **Country:** Belgium **Facility:** Investigational Site Number 056007 **Zip:** 1020 **Location 29:** **City:** Haine St Paul **Country:** Belgium **Facility:** Investigational Site Number 056003 **Zip:** 7100 **Location 30:** **City:** Liège **Country:** Belgium **Facility:** Investigational Site Number 056005 **Zip:** 4000 **Location 31:** **City:** Ottignies **Country:** Belgium **Facility:** Investigational Site Number 056004 **Zip:** 1340 **Location 32:** **City:** Roeselare **Country:** Belgium **Facility:** Investigational Site Number 056001 **Zip:** 8800 **Location 33:** **City:** Tournai **Country:** Belgium **Facility:** Investigational Site Number 056006 **Zip:** 7500 **Location 34:** **City:** Banja Luka **Country:** Bosnia and Herzegovina **Facility:** Investigational Site Number 070003 **Location 35:** **City:** Sarajevo **Country:** Bosnia and Herzegovina **Facility:** Investigational Site Number 070001 **Location 36:** **City:** Zenica **Country:** Bosnia and Herzegovina **Facility:** Investigational Site Number 070002 **Location 37:** **City:** Plovdiv **Country:** Bulgaria **Facility:** Investigational Site Number 100004 **Zip:** 4000 **Location 38:** **City:** Sofia **Country:** Bulgaria **Facility:** Investigational Site Number 100001 **Zip:** 1527 **Location 39:** **City:** Sofia **Country:** Bulgaria **Facility:** Investigational Site Number 100003 **Zip:** 1756 **Location 40:** **City:** Sofia **Country:** Bulgaria **Facility:** Investigational Site Number 100002 **Location 41:** **City:** Kitchener **Country:** Canada **Facility:** Investigational Site Number 124012 **Zip:** N2G1G3 **Location 42:** **City:** Laval **Country:** Canada **Facility:** Investigational Site Number 124009 **Zip:** H7M3L9 **Location 43:** **City:** London **Country:** Canada **Facility:** Investigational Site Number 124004 **Zip:** N6A 4L6 **Location 44:** **City:** Moncton **Country:** Canada **Facility:** Investigational Site Number 124007 **Zip:** E1C6Z8 **Location 45:** **City:** Montreal **Country:** Canada **Facility:** Investigational Site Number 124001 **Zip:** H2L 4M1 **Location 46:** **City:** Montreal **Country:** Canada **Facility:** Investigational Site Number 124011 **Zip:** H3G1A4 **Location 47:** **City:** Quebec **Country:** Canada **Facility:** Investigational Site Number 124010 **Zip:** G1R 2J6 **Location 48:** **City:** Toronto **Country:** Canada **Facility:** Investigational Site Number 124003 **Zip:** M4N3M5 **Location 49:** **City:** Toronto **Country:** Canada **Facility:** Investigational Site Number 124002 **Zip:** M5G2M9 **Location 50:** **City:** Winnipeg **Country:** Canada **Facility:** Investigational Site Number 124006 **Zip:** R3E0V9 **Location 51:** **City:** Zagreb **Country:** Croatia **Facility:** Investigational Site Number 191-001 **Zip:** 10000 **Location 52:** **City:** Brno **Country:** Czech Republic **Facility:** Investigational Site Number 203001 **Zip:** 65653 **Location 53:** **City:** Cph Ø **Country:** Denmark **Facility:** Investigational Site Number 208-001 **Zip:** 2100 **Location 54:** **City:** Herlev **Country:** Denmark **Facility:** Investigational Site Number 208-002 **Zip:** 2730 **Location 55:** **City:** Odense **Country:** Denmark **Facility:** Investigational Site Number 208-003 **Zip:** 5000 **Location 56:** **City:** Helsinki **Country:** Finland **Facility:** Investigational Site Number 246002 **Zip:** 00180 **Location 57:** **City:** Seinäjoki **Country:** Finland **Facility:** Investigational Site Number 246001 **Zip:** 60220 **Location 58:** **City:** Budapest **Country:** Hungary **Facility:** Investigational Site Number 348001 **Zip:** 1032 **Location 59:** **City:** Szolnok **Country:** Hungary **Facility:** Investigational Site Number 348002 **Zip:** 5000 **Location 60:** **City:** Szombathely **Country:** Hungary **Facility:** Investigational Site Number 348003 **Zip:** 9700 **Location 61:** **City:** Veszprém **Country:** Hungary **Facility:** Investigational Site Number 348004 **Zip:** 8200 **Location 62:** **City:** Chennai **Country:** India **Facility:** Investigational Site Number 356004 **Zip:** 600035 **Location 63:** **City:** Gurgaon **Country:** India **Facility:** Investigational Site Number 356006 **Zip:** 12201 **Location 64:** **City:** New Delhi **Country:** India **Facility:** Investigational Site Number 356001 **Zip:** 110029 **Location 65:** **City:** New Delhi **Country:** India **Facility:** Investigational Site Number 356002 **Zip:** 110085 **Location 66:** **City:** Trivandrum **Country:** India **Facility:** Investigational Site Number 356005 **Zip:** 695011 **Location 67:** **City:** Cork **Country:** Ireland **Facility:** Investigational Site Number 372004 **Location 68:** **City:** Dublin 24 **Country:** Ireland **Facility:** Investigational Site Number 372003 **Location 69:** **City:** Dublin 7 **Country:** Ireland **Facility:** Investigational Site Number 372001 **Location 70:** **City:** Dublin 7 **Country:** Ireland **Facility:** Investigational Site Number 372002 **Location 71:** **City:** Arezzo **Country:** 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Cruz de Tenerife **Country:** Spain **Facility:** Investigational Site Number 724005 **Zip:** 38320 **Location 140:** **City:** Santander **Country:** Spain **Facility:** Investigational Site Number 724016 **Zip:** 39008 **Location 141:** **City:** Santiago de Compostela **Country:** Spain **Facility:** Investigational Site Number 724015 **Zip:** 15706 **Location 142:** **City:** Sevilla **Country:** Spain **Facility:** Investigational Site Number 724024 **Zip:** 41013 **Location 143:** **City:** Terrassa **Country:** Spain **Facility:** Investigational Site Number 724004 **Zip:** 08221 **Location 144:** **City:** Valencia **Country:** Spain **Facility:** Investigational Site Number 724013 **Zip:** 46009 **Location 145:** **City:** Valencia **Country:** Spain **Facility:** Investigational Site Number 724014 **Zip:** 46015 **Location 146:** **City:** Zaragoza **Country:** Spain **Facility:** Investigational Site Number 724001 **Zip:** 50009 **Location 147:** **City:** Umeå **Country:** Sweden **Facility:** Investigational Site Number 752-002 **Zip:** 90185 **Location 148:** **City:** Uppsala **Country:** Sweden **Facility:** Investigational Site Number 752-001 **Zip:** 75185 **Location 149:** **City:** Kaohsiung Hsien, **Country:** Taiwan **Facility:** Investigational Site Number 005 **Location 150:** **City:** Taichung **Country:** Taiwan **Facility:** Investigational Site Number 004 **Zip:** 407 **Location 151:** **City:** Taipei **Country:** Taiwan **Facility:** Investigational Site Number 002 **Zip:** 100 **Location 152:** **City:** Taipei **Country:** Taiwan **Facility:** Investigational Site Number 003 **Location 153:** **City:** Tao Yuan Hsien **Country:** Taiwan **Facility:** Investigational Site Number 001 **Location 154:** **City:** Birmingham **Country:** United Kingdom **Facility:** Investigational Site Number 826011 **Zip:** B152TH **Location 155:** **City:** Bristol **Country:** United Kingdom **Facility:** Investigational Site Number 826002 **Zip:** BS28ED **Location 156:** **City:** Glasgow **Country:** United Kingdom **Facility:** Investigational Site Number 826003 **Zip:** G116NT **Location 157:** **City:** Leeds **Country:** United Kingdom **Facility:** Investigational Site Number 826013 **Zip:** LS97TF **Location 158:** **City:** London **Country:** United Kingdom **Facility:** Investigational Site Number 826009 **Zip:** SE19RT **Location 159:** **City:** Manchester **Country:** United Kingdom **Facility:** Investigational Site Number 826012 **Zip:** M204BX **Location 160:** **City:** Nottingham **Country:** United Kingdom **Facility:** Investigational Site Number 826005 **Zip:** NG51PB **Location 161:** **City:** Preston **Country:** United Kingdom **Facility:** Investigational Site Number 826008 **Zip:** PR24QF **Location 162:** **City:** Sutton **Country:** United Kingdom **Facility:** Investigational Site Number 826001 **Zip:** SM25PT **Location 163:** **City:** Torquay **Country:** United Kingdom **Facility:** Investigational Site Number 826007 **Zip:** TQ27AA **Location 164:** **City:** Whitechurch **Country:** United Kingdom **Facility:** Investigational Site Number 826006 **Zip:** CF142TL **Location 165:** **City:** Wirral **Country:** United Kingdom **Facility:** Investigational Site Number 826004 **Zip:** CH634JY #### Overall Officials **Official 1:** **Affiliation:** Sanofi **Name:** Clinical Sciences & Operations **Role:** STUDY_DIRECTOR ### References Module #### References **Citation:** Bahl A, Masson S, Malik Z, Birtle AJ, Sundar S, Jones RJ, James ND, Mason MD, Kumar S, Bottomley D, Lydon A, Chowdhury S, Wylie J, de Bono JS. Final quality of life and safety data for patients with metastatic castration-resistant prostate cancer treated with cabazitaxel in the UK Early Access Programme (EAP) (NCT01254279). BJU Int. 2015 Dec;116(6):880-7. doi: 10.1111/bju.13069. Epub 2015 Jun 16. **PMID:** 25639506 **Citation:** Castellano D, Anton Aparicio LM, Esteban E, Sanchez-Hernandez A, Germa JR, Batista N, Maroto P, Perez-Valderrama B, Luque R, Mendez-Vidal MJ; cabazitaxel EAP study. Cabazitaxel for metastatic castration-resistant prostate cancer: safety data from the Spanish expanded access program. Expert Opin Drug Saf. 2014 Sep;13(9):1165-73. doi: 10.1517/14740338.2014.939583. Epub 2014 Jul 7. **PMID:** 25001524 ## 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 ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M14335 - Name: Prostatic Neoplasms - Relevance: HIGH - As Found: Prostate Cancer - 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 ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Infl - Name: Anti-Inflammatory Agents - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: PhSol - Name: Pharmaceutical Solutions - Abbrev: AnEm - Name: Antiemetics - Abbrev: NeuroAg - Name: Neuroprotective Agents - Abbrev: Gast - Name: Gastrointestinal Agents ### Intervention Browse Module - Browse Leaves - ID: M9789 - Name: Hormones - Relevance: LOW - As Found: Unknown - ID: M14121 - Name: Prednisone - Relevance: LOW - As Found: Unknown - ID: M21860 - Name: Pharmaceutical Solutions - Relevance: LOW - As Found: Unknown - ID: M14120 - Name: Prednisolone - Relevance: LOW - As Found: Unknown - ID: M1668 - Name: Docetaxel - Relevance: LOW - As Found: Unknown - ID: M11749 - Name: Methylprednisolone - Relevance: LOW - As Found: Unknown - ID: M1833 - Name: Methylprednisolone Acetate - Relevance: LOW - As Found: Unknown - ID: M11750 - Name: Methylprednisolone Hemisuccinate - Relevance: LOW - As Found: Unknown - ID: M229449 - Name: Prednisolone acetate - Relevance: LOW - As Found: Unknown - ID: M211887 - Name: Prednisolone hemisuccinate - Relevance: LOW - As Found: Unknown - ID: M248881 - Name: Prednisolone phosphate - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02664779 **Brief Title:** Determination and Comparison of Short-term Effectiveness of Three Methods Used for Recognition of Arrhythmias in People With Different Degrees of Medical Training (Advanced Life Support Workshop Participants-ALS): Randomized Controlled Educational Experiment. #### Organization Study ID Info **ID:** ALS1 #### Organization **Class:** OTHER **Full Name:** Universidad Nacional de Colombia ### Status Module #### Completion Date **Date:** 2017-06 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-06-01 **Type:** ACTUAL **Last Update Submit Date:** 2021-05-26 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2017-01 **Type:** ACTUAL #### Start Date **Date:** 2016-01 **Type:** ACTUAL **Status Verified Date:** 2021-05 #### Study First Post Date **Date:** 2016-01-27 **Type:** ESTIMATED **Study First Submit Date:** 2016-01-22 **Study First Submit QC Date:** 2016-01-22 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Universidad Nacional de Colombia #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** Background: Arrhythmia recognition is a fundamental skill for the provider of advanced life support (ALS). Acquire it is difficult, leading to the birth of systematic methods in an attempt to simplify and optimize, however, it has not compared the effectiveness among the three methods with more evidence among professionals with varying degrees of medical training (ALS Workshop participants). Objective: To determine and compare the effectiveness of the three most widespread and with more evidence systematic methods (10, 6 and 4 steps) for the recognition of arrhythmias in a short-term and its perceived easiness among ALS workshop participants. Methods / design: Educational Cuasi experimental trial with pre and post intervention measurement, blind, with randomized allocation, in 84 ALS workshop participants. Three systematic methods to recognize arrhythmias will be taught and their effectiveness to diagnose in a short-term and its perceived easiness will be measured and compared. ### Conditions Module **Conditions:** - Arrhythmias ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - PARTICIPANT **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 76 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Of participants whom attending the workshop, we will take 84 whom agree to participate and sign the informed consent. They will be divided into subgroups according to their degree of medical training (nursing technicians, university nursing students, university medical students, nurse practitioners graduates, Professional medical graduates, residents of medical specialties and specialists) and randomly assigned one by one from each educational subgroup to 3 intervention groups A, B or C (A = 10 STEPS method, B = 6 STEPS method, C = 4 STEPS method ) so that each group has the third participants of each level of education ensuring matched groups on the level of training of participants in each group. Following this, an equal theoretical test will be performed for all groups to determine the knowledge base in arrhythmias. **Intervention Names:** - Other: Arrhythmia diagnosis with the 10 steps method **Label:** Arrhythmia diagnosis with the 10 steps method **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Of participants whom attending the workshop, we will take 84 whom agree to participate and sign the informed consent. They will be divided into subgroups according to their degree of medical training (nursing technicians, university nursing students, university medical students, nurse practitioners graduates, Professional medical graduates, residents of medical specialties and specialists) and randomly assigned one by one from each educational subgroup to 3 intervention groups A, B or C (A = 10 STEPS method, B = 6 STEPS method, C = 4 STEPS method ) so that each group has the third participants of each level of education ensuring matched groups on the level of training of participants in each group. Following this, an equal theoretical test will be performed for all groups to determine the knowledge base in arrhythmias **Intervention Names:** - Other: Arrhythmia diagnosis with the 6 steps method **Label:** Arrhythmia diagnosis with the 6 steps method **Type:** ACTIVE_COMPARATOR #### Arm Group 3 **Description:** Of participants whom attending the workshop, we will take 84 whom agree to participate and sign the informed consent. They will be divided into subgroups according to their degree of medical training (nursing technicians, university nursing students, university medical students, nurse practitioners graduates, Professional medical graduates, residents of medical specialties and specialists) and randomly assigned one by one from each educational subgroup to 3 intervention groups A, B or C (A = 10 STEPS method, B = 6 STEPS method, C = 4 STEPS method ) so that each group has the third participants of each level of education ensuring matched groups on the level of training of participants in each group. Following this, an equal theoretical test will be performed for all groups to determine the knowledge base in arrhythmias **Intervention Names:** - Other: Arrhythmia diagnosis with the 4 steps method **Label:** Arrhythmia diagnosis with the 4 steps method **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Arrhythmia diagnosis with the 10 steps method **Name:** Arrhythmia diagnosis with the 10 steps method **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - Arrhythmia diagnosis with the 6 steps method **Name:** Arrhythmia diagnosis with the 6 steps method **Type:** OTHER #### Intervention 3 **Arm Group Labels:** - Arrhythmia diagnosis with the 4 steps method **Name:** Arrhythmia diagnosis with the 4 steps method **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** For comparison of the percentage of subjects who achieve scores greater than or equal to 30 (approving minimum score) in the practical test of arrhythmia's diagnosis, the test will be the Chi-square (X2), which allows to compare proportions in more than two groups; or Fisher's exact test, submitted that one of the expected values is less than 5. **Measure:** percentage of subjects who achieve scores greater than or equal to 30 **Time Frame:** 15 minutes after delivered the intervention #### Secondary Outcomes **Description:** In order to compare the magnitude of the change in the average scores in the groups trained with each of the 3 methods, both in the the theoretical and practical test of diagnosis of arrhythmias; ANOVA or Mann-Whitney's U, according to the distribution of variables was performed. These comparisons are made to the post intervention measurements and Bonferroni correction for alpha according to the number of comparisons will perform. The analysis of the collected data will be using STATA 10.0 software. **Measure:** change in the average scores in the groups **Time Frame:** 15 minutes after delivered the intervention ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * • Health professionals * Attend a course in Advanced Life Support in Cali (Colombia), during the study period. * Voluntarily accept to participate in the study Exclusion Criteria: * No **Healthy Volunteers:** True **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Cali **Country:** Colombia **Facility:** Cruz Roja Colombiana **State:** Valle Del Cauca ## Derived Section ### Condition Browse Module - Ancestors - ID: D000006331 - Term: Heart Diseases - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M4453 - Name: Arrhythmias, Cardiac - Relevance: HIGH - As Found: Arrhythmia - ID: M9419 - Name: Heart Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001145 - Term: Arrhythmias, Cardiac ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03388879 **Acronym:** NAFTI **Brief Title:** Reamed Nailing Versus Taylor Spatial Frame in Tibia Shaft Fractures **Official Title:** Reamed Nailing Versus Taylor Spatial Frame in Tibia Shaft Fractures #### Organization Study ID Info **ID:** REKC 2010/1706 #### Organization **Class:** OTHER **Full Name:** Oslo University Hospital ### Status Module #### Completion Date **Date:** 2016-06-21 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-01-03 **Type:** ACTUAL **Last Update Submit Date:** 2017-12-30 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2015-06-16 **Type:** ACTUAL #### Start Date **Date:** 2010-10-31 **Type:** ACTUAL **Status Verified Date:** 2017-12 #### Study First Post Date **Date:** 2018-01-03 **Type:** ACTUAL **Study First Submit Date:** 2017-12-17 **Study First Submit QC Date:** 2017-12-30 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Sahlgrenska University Hospital, Sweden #### Lead Sponsor **Class:** OTHER **Name:** Oslo University Hospital #### Responsible Party **Investigator Affiliation:** Oslo University Hospital **Investigator Full Name:** Frede Frihagen **Investigator Title:** Consultant **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** This is a randomised, bi-centre, prospective, clinical trial in patients with closed tibia shaft fractures. The fracture should be fresh/acute and seen within 3 weeks after the injury. Patients will be randomised to surgery with either a Taylor Spatial Frame (Smith \& Nephew, England) or a reamed intramedullar nail (according to local choice) with locking screws. Primary outcome measure is the physical component summary (PCS) of RAND Short form 36 (SF-36) after 2 years. Among secondary outcomes: Visual Analogue Scale (VAS) for pain, complications, healing, malunion, and resource use. **Detailed Description:** Fractures of the lower leg (fractures of the tibia shaft with or without concurrent fracture of the fibula) are a common injury. According to our fracture register 95 patients with closed tibia fractures were operated the last 3 years at our department. Fractures with moderate or no displacement can be successfully treated with a cast and subsequent Sarmiento brace. Displaced fractures are commonly treated with an intramedullary nail. Intramedullary nailing yields a high rate of union. More than 50 % of operated patients do, however, develop chronic anterior knee pain and one third of the patients have pain at rest. This contributes a big problem for many patients both at spare time and at work. Another problem is significant rates of malunion. The use of ring fixators utilizing rings and 1,8 mm. wires was introduced by Gavril Ilizarov more than 50 years ago, and the technique has been further developed through the introduction of six adjustable struts (Taylor Spatial Frame). This hexapod circular frame allows accurate reduction as well as a high stability. The ring fixator is less invasive and allows early weight bearing, but may be cumbersome to the patient. There is also concern about pin-tract infection, osteomyelitis and joint contracture. Only one prior study has compared ring fixator (Ilizarov) and intramedullar nail in closed tibia fractures. The results showed significant less anterior knee pain in the patients operated with ring fixator, but the study design did not allow clear conclusion. ### Conditions Module **Conditions:** - Tibial Fractures ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** Prospective randomized two-group clinical trial with block randomization. ##### Masking Info **Masking:** SINGLE **Masking Description:** The researcher doing the statistical analyses will be masked for treatment Group (i.e. Group 1 or 2) in a databse blinded for treatment grioups and without variables indirectly revealing treatmnet arm. These will be analyzed later. **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 65 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** A Taylor Spatial Frame should consist of 2 rings with 4 half pins/K-wire attached to each ring. If possible 3, not hydroxyapatite-coated, half pins and one K-wire should be attached to each ring. The half pins/K-wire should be spread in distance and direction for optimum stability. **Intervention Names:** - Device: Taylor Spatial Frame **Label:** Circular frame external fixator **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Nailing technique according to Karladani and Styf published technique (ref: Karladani AH, Styf J. Percutaneous intramedullary nailing of tibial shaft fratures: a new approach for prevention of anterior knee pain. Injury, Int. J. Care Injury 32 (2001) 736-39) **Intervention Names:** - Device: Intramedullary nail **Label:** Intramedullary nail **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Circular frame external fixator **Description:** Circular external fixator **Name:** Taylor Spatial Frame **Other Names:** - Circular frame **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - Intramedullary nail **Description:** Antegrade intramedullary nail **Name:** Intramedullary nail **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Generic Health Related Quality of Life. Mean value 50, standard deviation 10. Higher score better. **Measure:** Physical Component summary of RAND SF 36 (Short Form 36) **Time Frame:** 24 months #### Secondary Outcomes **Description:** Generic Health Related Quality of Life. Range 0 (worst) to 100 (best). **Measure:** Vitality Subscore of RAND (SF) 36 **Time Frame:** 6, 12, 24 months **Description:** Generic Health Related Quality of Life. Range 0 (worst) to 100 (best). **Measure:** Physical functioning, subscore of RAND (SF) 36 **Time Frame:** 6, 12, 24 months **Description:** Generic Health Related Quality of Life. Range 0 (worst) to 100 (best). **Measure:** Bodily pain, subscore of RAND (SF) 36 **Time Frame:** 6, 12, 24 months **Description:** Generic Health Related Quality of Life. Range 0 (worst) to 100 (best). **Measure:** General health perceptions, subscore of RAND (SF) 36 **Time Frame:** 6, 12, 24 months **Description:** Generic Health Related Quality of Life. Range 0 (worst) to 100 (best). **Measure:** Physical role functioning, subscore of RAND (SF) 36 **Time Frame:** 6, 12, 24 months **Description:** Generic Health Related Quality of Life. Range 0 (worst) to 100 (best). **Measure:** Emotional role functioning, subscore of RAND (SF) 36 **Time Frame:** 6, 12, 24 months **Description:** Generic Health Related Quality of Life. Range 0 (worst) to 100 (best). **Measure:** Social role functioning, subscore of RAND (SF) 36 **Time Frame:** 6, 12, 24 months **Description:** Generic Health Related Quality of Life. Range 0 (worst) to 100 (best). **Measure:** Mental health, subscore of RAND (SF) 36 **Time Frame:** 6, 12, 24 months **Description:** Generic Health Related Quality of Life. Mean value 50, standard deviation 10. Higher score better. **Measure:** Physical Component summary of RAND (SF) 36 **Time Frame:** 6, 12 months **Description:** VAS scale 0-10 **Measure:** Pain around the knee **Time Frame:** 6, 12, 24 months **Description:** VAS scale 0-10 **Measure:** Pain around the fracture site **Time Frame:** 6, 12, 24 months **Description:** VAS scale 0-10 **Measure:** Pain around the ankle **Time Frame:** 6, 12, 24 months **Description:** Compartment syndrome, sequela compartment syndrome (e.g. short foot, clawing, neurological disorder), infection that needs operation, any unexpected reoperation (except removal of single pins or screws) **Measure:** Complications major (composite) **Time Frame:** 24 months **Description:** pin tract infection that needs antibiotics, wound complication that don't need reoperation, unexpected minor reoperations (i.e. removal of single pins or screws) **Measure:** Complications minor (composite) **Time Frame:** 24 months **Description:** Minor reoperation (e.g. remove/exchange pins, remove/exchange screws) **Measure:** Reoperations minor (composite) **Time Frame:** 6, 12, 24 months **Description:** Major reoperation (e.g. fasciotomy, exchange nail, surgery for refracture, revision for infection, surgery for non-union) **Measure:** Reoperations major (composite) **Time Frame:** 6, 12, 24 months **Description:** Time to fracture union in days. We require both radiographical union defined by callus bridging 3 of 4 cortices AND clinical union defined by full, pain free and unaided weight bearing. **Measure:** Time to union (composite) **Time Frame:** 6, 12, 24 months **Description:** Number of days away from work for employed patients **Measure:** Resource use; Away from work **Time Frame:** 24 months **Description:** Number of unscheduled contacts with hospital regarding tibia fracture **Measure:** Resource use; Emergency contacts **Time Frame:** 24 months **Description:** Hospital stay in days for index stay **Measure:** Resource use; Length of stay **Time Frame:** 24 months **Description:** Surgery time in minutes for index surgery **Measure:** Resource use; Operation time **Time Frame:** 24 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Closed tibia shaft fractures suited for both study treatments. * A patient who is informed of the purpose of the investigation and who has given informed consent and willingness to accept randomisation either to Taylor Spatial Frame or intramedullary nailing. * Willingness and ability to comply with all investigation procedures * Age between 18 to 70 years * Skeletally mature * Previous unaided walking Exclusion Criteria: * Participation in other clinical investigations that will interfere with this study * Mental illness or other conditions that preclude ring fixator in the judgment of the investigator * Any other concurrent condition(s) that, in the judgment of the investigator, would prohibit the patient from participation in the study * No other injury or previous disease that would be likely to seriously influence the long term outcome (this will exclude e.g. osteomyelitis, vascular or neurological disorder of the lower extremities, rheumatoid artist, malignancy that could influence on bone healing) * Compartment syndrome before randomisation * Pathologic fracture * Ongoing or previous use the last year of drugs that can be bone anabolic (e.g. anabolic steroids, growth hormone, parathyroid hormone) **Maximum Age:** 70 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Oslo **Country:** Norway **Facility:** Orthopedic Center, Ulleval University Hospital **Zip:** 0408 #### Overall Officials **Official 1:** **Affiliation:** Professor, head of research group **Name:** Jan E Madsen, PhD **Role:** STUDY_CHAIR ### IPD Sharing Statement Module **Description:** Depending on demand. No plan as of yet. **IPD Sharing:** UNDECIDED ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014947 - Term: Wounds and Injuries - ID: D000007869 - Term: Leg Injuries ### Condition Browse Module - Browse Branches - Abbrev: BC26 - Name: Wounds and Injuries - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M26370 - Name: Fractures, Bone - Relevance: HIGH - As Found: Fracture - ID: M16737 - Name: Tibial Fractures - Relevance: HIGH - As Found: Tibial Fractures - ID: M17685 - Name: Wounds and 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: D000013978 - Term: Tibial Fractures ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05016479 **Acronym:** arTMSinGD **Brief Title:** Accelerated rTMS in Gambling Disorder: a Multicentric, Randomized, Sham-controlled Trial **Official Title:** Accelerated rTMS in Gambling Disorder: a Multicentric, Randomized, Sham-controlled Trial #### Organization Study ID Info **ID:** richt1ie1 #### Organization **Class:** OTHER **Full Name:** ITAB - Institute for Advanced Biomedical Technologies ### Status Module #### Completion Date **Date:** 2026-02-28 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2021-10-26 **Type:** ACTUAL **Last Update Submit Date:** 2021-10-25 **Overall Status:** NOT_YET_RECRUITING #### Primary Completion Date **Date:** 2025-11-30 **Type:** ESTIMATED #### Start Date **Date:** 2021-12-01 **Type:** ESTIMATED **Status Verified Date:** 2021-10 #### Study First Post Date **Date:** 2021-08-23 **Type:** ACTUAL **Study First Submit Date:** 2021-08-17 **Study First Submit QC Date:** 2021-08-17 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** ITAB - Institute for Advanced Biomedical Technologies #### Responsible Party **Investigator Affiliation:** ITAB - Institute for Advanced Biomedical Technologies **Investigator Full Name:** Mauro Pettorruso **Investigator Title:** Dr **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** Background: Gambling disorder (GD), is a behavioral addiction based on keeping play despite medical, economic and social consequences. GD is characterized by progressive and persistent brain circuits alterations (reward, stress, memory, impulse control and cognitive functions), so a possible treatment could be based on neuromodulation of specific brain areas. Repetitive transcranial magnetic stimulation (rTMS) is a non-invasive brain stimulation, which provides magnetic stimuli on certain brain areas parts with short and long-term effects. rTMS has the FDA approval for some neurological (headache) and psychiatric (treatment-resistant depression, obsessive-compulsive disorder) disease. Nowadays several evidence in scientific literature lead to a promise use of rTMS also in addiction field with a possible indication also for GD. Objectives: the main outcome is to assess symptoms related to GD (craving, play frequencies, money lost) before and after rTMS stimulation on left dorsolateral prefrontal cortex (DLPFC). Eligibility: Healthy, right-handed adults ages 18-65 with a diagnosis of GD. Design: This is a randomized, sham-controlled study. The study includes two phases:1) a rTMS continued treatment phase and 2) a follow-up without rTMS stimulation (30 days). In order to be enrolled, participants will be screened with: * Questionnaires * Medical history * Physical exam * f-MRI After being enrolled, baseline behavioral and imaging data will be collected. In particular, participants will submit: * Questionnaires * Functional MRI * Cognitive tasks During the continued rTMS phase, participants with gambling disorder will be randomized to receive real or sham rTMS. RTMS will be delivered during 5 outpatient treatment days, (3 times/die). After the last stimulation and at the end of the 30-days of follow-up period, subjects will undergo the neurocognitive and psychometric evaluation. Twenty randomized patients of whole enrolled group will undergo fMRI at baseline and at the end of arTMS treatment phase. Treatment includes: * rTMS: A weak electrical current passes through a coil placed on the head. During each stimulation day, participants will receive three rTMS sessions (13 min), with a 50 min of interval. * fMRI: Participants lie on a table that slides into a cylinder that takes pictures of the brain. They respond to images while in the scanner. * Repeat of screening tests and questionnaires **Detailed Description:** Repetitive Transcranial Magnetic Stimulation (rTMS) is a neurostimulation technique that consists in the application of magnetic pulses in order to modulate local brain activity, opened up the possibility of interacting with dysfunctional brain circuits, selectively targeting gambling-related cognitive dysfunction. The main outcome of the present study is to evaluate, in a population of GD patients, the effects of accelerated rTMS (arTMS) applied on Left Dorsolateral Prefrontal Cortex (LDLPFC) in terms of variations in gambling-related symptoms. In order to investigate the possible effects of arTMS on brain connectivity, a subsample of patients will undergo a functional neuroimaging study based on fMRI. The study includes 3 psychiatric assessments with psychometric testing: V1 (enrollment), V2 (day 5), V3 (week 4, follow-up). At Visit 1 (enrollment) the researcher will fully inform the patient about the study, obtaining the patient's informed consent to participate in the study, and will determine the patient's eligibility. Patients will also undergo a battery of cognitive tasks and psychometric evaluation. The same neurocognitive and psychiatric assessment will be repeated during Visit 2 (day 5) and Visit 3 (4 Weeks). The analysis of the neuroimaging data will allow to evaluate the effect of the active arTMS on brain connectivity (reward system, attention and executive control networks) investigate the association between these evidence and clinical variables. ### Conditions Module **Conditions:** - Gambling - Gambling Disorder - Gambling, Pathological - Gambling Problem **Keywords:** - arTMS - Transcranial Magnetic Stimulation - TMS - Gambling Disorder ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** double blind, randomized, sham-controlled with a 1:1 allocation into 2 parallel arms ##### Masking Info **Masking:** TRIPLE **Masking Description:** With regard to the treatment, to ensure that both participants and investigators are blind to the condition (active or sham), the selection of the operation mode (15 Hz, sham) will be pre-programmed by member of the that will not be involved in data collection and analysis. Study personnel will not know which mode is being activated. Sham stimulation will use the same coil placement as that used for active stimulation. Outcomes Assessors will not be present during the rTMS sessions. **Who Masked:** - PARTICIPANT - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 60 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** The treatment involves 15 Repetitive Transcranial Magnetic Stimlation sessions (3/daily for 5 consecutive days, each session lasts 13 min with an interval of 50 min). Coil is placed on the left dorsolateral prefrontal cortex (LDLPFC).The stimulation has a frequency of 15 Hz and a intensity of 120% of the individual resting motor threshold. **Intervention Names:** - Device: Accelerated repetitive Transcranial Magnetic Stimulation **Label:** Active arTMS (15 Hz) **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Sham group receives the same Repetitive Transcranial Magnetic Stimlation sessions of active compactors. However the interventions in the place group are with a superficial stimulation of scalp muscles only, in order to induce a sensation close to the one experienced with the real rTMS stimulation. **Intervention Names:** - Device: Accelerated repetitive Transcranial Magnetic Stimulation **Label:** Sham arTMS **Type:** SHAM_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Active arTMS (15 Hz) - Sham arTMS **Description:** arTMS is a non-invasive brain stimulation technique. It will be used a MagPro R30 with a Cool-B80 figure-of-eight coil (MagVenture, Falun, Denmark). **Name:** Accelerated repetitive Transcranial Magnetic Stimulation **Other Names:** - arTMS **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** To assess the severity of gambling-related symptoms: Yale Brown Obsessive Compulsive Scale adapted for Pathological Gambling (PG-YBOCS), 10-item clinician-administered questionnaire that assess gambling symptoms over a recent time interval **Measure:** Change in gambling behavior assessed by Yale Brown Obsessive Compulsive Scale adapted for Pathological Gambling (PG-YBOCS) **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** a self-rated 5-point Likert scale with total score ranges from 0 to 48 **Measure:** Change in gambling behavior assessed by Gambling Symptom Assessment Scale (G-SAS) **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** in a multidimension retrospective interview focused on the quantitative assessment of gambling behavior **Measure:** Change in gambling behavior assessed by TimeLine Follow Back (TLFB) - Gambling Version **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** an instrument applied to assess continuum variables, using a horizontal line on which the patient shell point his current state from left vertex (no gambling craving) to right vertex (maximum gambling craving). **Measure:** Change in gambling behavior assessed by Visual Analogue Scale **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks #### Secondary Outcomes **Description:** 5-Likert questionnaire to assess the presence of disturbing symptoms attention / hyperactivity **Measure:** Change in attention / hyperactivity symptoms assessed by Adult ADHD Self-Report Scale (ASRS) **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** a self-report 5 point Likert scale with 20 items to assess emotional awareness. The total score goes from 20 to 100: non-alexithymia when the score is less than 51, borderline from 51 to 60 and alexithymia if the score is over 60 **Measure:** Change in emotional awareness assessed by Toronto Alexithymia Scale (TAS-20) **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** a self-report, 5-point Likert scale to measure the current mood state through six dimensions of affect, including tension-anxiety, depression-dejection, anger-hostility, vigor-activity, fatigue-inertia, and confusion-bewilderment **Measure:** Change in mood state assessed by Profile of Mood States (POMS) **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** a scale created with the purpose of understanding traits as neuroticism, antisocial behavior and psychopathy. There are four different subscales: Thrill and Adventure Seeking, Disinhibition,Experience Seeking, Boredom Susceptibility. Each subscale contains 10 items, making a total of 40 items **Measure:** Change in search for sensations assessed by Sensation Seeking Scale V (SSS-V) **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** a 14-item 4-point scale with a total score from 0 to 42 **Measure:** Change in hedonic tone assessed by Snaith-Hamilton Pleasure Scale (SHAPS) **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** a 18-item 6-point scale with a range from 20 to 108 **Measure:** Change in hedonic tone assessed by Temporal Experience of Pleasure Scale (TEPS) **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** to measure the social bonds of the subject with family members, friends, etc; this questionnaire is designed to help assess the degree of enjoyment and satisfaction experienced during the past week. There are 8 subscales : Phisical health, feelings, work, household duties, school/cpurse work, leisure time activities, social relations, general activities. **Measure:** Change in quality of life assessed by Quality of Life Enjoyment and Satisfaction Questionnaire - Short Form (Q-LES-Q-SF) **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** The scale identifies specific behaviors which may be indicative of an individual's intent to complete suicide. An individual exhibiting even a single behavior identified by the scale was 8 to 10 times more likely to complete suicide **Measure:** Change in suicidal behaviours assessed by Columbia-Suicide Severity Rating Scale (C-SSRS) **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** measuring both internalizing symptoms (depression, somatization, anxiety) and externalizing ones (aggression, hostility, impulsiveness) of psychiatric patients, general medicine and non-clinical subjects **Measure:** Change in psychopathological symptoms assessed by SCL-90 **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** a self-report 5-point Likert scale assess to evaluate food craving. The questionnaire consists of 28 items collected in 4 subscale: "high fat foods", "sweets", "carbohydrates", "fast foods" **Measure:** Change in food craving assessed by Food craving questionnaire (FCI) **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** a self-report 5-point Likert questionnaire to estimate sleep quality in the last month. The total score range is from 0 to 28, in particular: absence of insomnia (0-7); sub-threshold insomnia (8-14); moderate insomnia (15-21); and severe insomnia (22-28) **Measure:** Change in sleep quality assessed by Insomnia severity index (ISI) **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** a Likert based scale (1-7 score) to evaluate nicotine craving. The questionnaire consisted in 4 domines (emotionally, expectancy, compulsivity, purposufullness), each with 3 items **Measure:** Change in nicotine craving assessed by Short Tobacco Craving Questionnaire (sTCQ) **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** a 14-item 5-point scale with a total range score from 0 (min ) to 56 (max), that assesses anxiety symptoms exploring different domains "anxious mood", "tension" or "fears" **Measure:** Change in anxiety level assessed by Hamilton Anxiety Scale (HAM-A) **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** a interview with 20 items and total score is considered normal with range 0-7 **Measure:** Change in depressive symptoms assessed by Hamilton Depression Scale (HAM-D) **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** a 11-item interview with total range score form 0 to 60 **Measure:** Change in manic symptoms assessed by Young Mania Rating Scale (YMRS) **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** to measure any possible Collateral effect. PANAS is a 20-items 5-poin Likert questionnaire consisted in 2 subscale: Positive Affect Score and Negative Affect Score, with total range from 10 to 50 for each domains **Measure:** Change in positive and negative symptoms assessed by Positive Affect and Negative Affect Scales (PANAS) **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** a 11-items self-report questionnaire to evaluate temperamental impulsive traits. BIS-11 consists in 3 subscale: "Attentional Impulsivity", "Motor Impulsivity" and "Nonplanning Impulsivity" **Measure:** Change in impulsivness assessed by Barratt Impulsiveness Scale (BIS-11) **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** a psychological task thought to simulate real-life decision making. Participants are presented with four virtual decks of cards on a computer screen. They are told that each deck holds cards that will either reward or penalize them, using game money. The goal of the game is to win as much money as possible. The decks differ from each other in the balance of reward versus penalty cards **Measure:** Change in cognitive performance by Iowa gambling task (IGT) **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** used to measure a participants capacity for sustained attention and response control. For example, a go/no-go test that requires a participant to perform an action given certain stimuli (e.g., press a button - Go) and inhibit that action under a different set of stimuli (e.g., not press that same button - No-Go). **Measure:** Change in cognitive performance by Go / No-Go Task **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** a neuropsychological test extensively used to assess the ability to inhibit cognitive interference that occurs when the processing of a specific stimulus feature impedes the simultaneous processing of a second stimulus attribute, well-known as the Stroop Effect **Measure:** Change in cognitive performance by Stroop color-word task **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks **Description:** a neuropsychological test of "set-shifting", i.e. the ability to display flexibility in the face of changing schedules of reinforcement. A number of stimulus cards are presented to the participant. The original WCST used paper cards and was carried out with the experimenter on one side of the desk facing the participant on the other **Measure:** Change in cognitive performance by Wisconsin card-sorting task **Time Frame:** Baseline, after rTMS treatment (5 day), 4 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Current diagnosis of Gambling Disorder, based on the Diagnostic and Statistical Manual of Mental Disorder - Fifth Edition (DSM-5); Exclusion Criteria: * Current or pre-existing DSM-5 diagnosis of schizophrenia, bipolar disorder, or other psychotic disorder; * Use in the past 4 weeks of any medication with known pro-convulsant action, including antipsychotic medications, tricyclic antidepressants, and antihistamines drugs; * Medical history with significant neurological disorder, including organic brain disease, epilepsy, stroke, brain lesions and multiple sclerosis, previous neurosurgery, or personal history of head trauma that resulted in loss of consciousness for \> 5 minutes and retrograde amnesia for \> 30 minutes; * Any personal or family history (1st degree relatives) of seizures other than febrile childhood seizures; * Any psychiatric, medical or social condition whether or not listed above, due to which, according to the judgment of the PI and after any consults if indicated, participation in the study is not in the best interest of the patient; * For female patients: Pregnancy/breastfeeding. **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:** Mauro Pettorruso, MD, PhD **Phone:** +39 0871 355 6901 **Role:** CONTACT #### Overall Officials **Official 1:** **Affiliation:** ITAB - Institute for Advanced Biomedical **Name:** Massimo di Giannantonio, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007174 - Term: Disruptive, Impulse Control, and Conduct 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: M8832 - Name: Gambling - Relevance: HIGH - As Found: Gambling - ID: M21827 - Name: Conduct Disorder - Relevance: LOW - As Found: Unknown - ID: M10219 - Name: Disruptive, Impulse Control, and Conduct 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: D000005715 - Term: Gambling ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02165579 **Brief Title:** Osteomyelitis: Procalcitonin to Diagnose and Monitor Osteomyelitis **Official Title:** Osteomyelitis: Procalcitonin to Diagnose and Monitor Osteomyelitis #### Organization Study ID Info **ID:** Biomarkers for Osteomyelitis #### Organization **Class:** OTHER **Full Name:** University of Texas Southwestern Medical Center ### Status Module #### Completion Date **Date:** 2014-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-06-08 **Type:** ACTUAL **Last Update Submit Date:** 2018-06-06 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2014-08 **Type:** ACTUAL #### Start Date **Date:** 2014-06 **Type:** ACTUAL **Status Verified Date:** 2018-06 #### Study First Post Date **Date:** 2014-06-17 **Type:** ESTIMATED **Study First Submit Date:** 2014-05-12 **Study First Submit QC Date:** 2014-06-16 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Texas Southwestern Medical Center #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The investigator plans a cohort study of 80 subjects admitted to hospital with a diabetic foot infection in order to compare serial bone biopsies (the current "gold standard") and procalcitonin to diagnose and monitor the effectiveness of therapy for osteomyelitis. The investigator will collect specimens as part of an existing trial to evaluate negative pressure wound therapy in diabetic infected wounds. A high proportion of these patients have osteomyelitis and will receive standard therapy including repeat bone biopsy and parenteral antibiotics. The investigator expects repeated measurement of procalcitonin will be highly correlated with repeated bone biopsy after antibiotic treatment has been completed to determine if therapy has been successful or if additional antibiotic therapy is needed. **Detailed Description:** There is a world-wide epidemic of diabetes. As part of the epidemic lower extremity amputations are dramatically increasing. Soft tissue and bone infections are one of the most common reasons for amputation. It is often difficult to determine if diabetic foot ulcers have an underlying bone infection. Inaccurate diagnosis of osteomyelitis leads to unnecessary antibiotic treatment, surgery, and amputation. In addition, we do not have good diagnostic tools to determine when osteomyelitis has been treated successfully. The role of biomarkers specific to bone turnover (resorption and formation) in relation to bone infections is poorly understood. We know that remodeling is an essential function in bone physiology with increased osteoclast production leading to resorption of old bone coupled with increased osteoblast production associated with new bone formation. The balance between these two functions is known to be disrupted in disease states including osteoporosis, but has not been examined specifically in infected bone. Procalcitonin has been suggeasted as a tool to both diagnose and monitor the effectiveness of therapy for various infections, but there is very little work in diabetic foot osteomyelitis. Aim 1. To evaluate the role of procalcitonin as a screening tool to diagnose diabetic foot osteomyelitis using bone culture and histopathology as the "gold standard" to establish the diagnosis. Aim 2. To determine the role of procalcitonin as a management tool to determine osteomyelitis treatment success versus treatment failure (indicated by bone biopsy) after completing a standard course of antibiotics for 6 weeks. ### Conditions Module **Conditions:** - Diabetes - Foot Ulcer - Osteomyelitis **Keywords:** - procalcitonin - biomarkers - Osteomyelitis - diabetes - foot ulcer(s) - Infectious Disease Society of America stage 3 infection ### Design Module #### Design Info **Observational Model:** CASE_ONLY **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 36 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 1 Cohort, standard care, observational patients are: Diagnosis of diabetes mellitus Age ≥ 21 years Infectious Disease Society of America stage 3 infection **Intervention Names:** - Other: 1 cohort, standard care, observational **Label:** Age > 21, diabetes, osteomyelitis ### Interventions #### Intervention 1 **Arm Group Labels:** - Age > 21, diabetes, osteomyelitis **Description:** This is one cohort standard care observational study. 40 diabetic patients admitted to Parkland hospital with a foot infection. We will enroll a representative cross section of subjects with diabetic foot infection. Patient will be consented and will receive therapy based on standard protocols. Currently patients with suspected osteomyelitis have bone biopsies to identify bacterial pathogens and verify MRI diagnosis. Repeat bone biopsies are performed at the end of therapy to verify that osteomyelitis has been successfully treated. Serum to measure procalcitonin will be obtained at baseline, week 3 and week 6. Tissue samples will be obtained at Baseline, Week 3 and Week 6 for tissue culture. **Name:** 1 cohort, standard care, observational **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Clinical examination. Per current standards, wound healing will be defined as complete granulation over the wound bed (primary intention) or wounds closed by surgical intervention. 80 subjects will be followed during the course of the study and the wound healing dates recorded for those subjects who heal. **Measure:** (1.) wound healing, **Time Frame:** 6 months #### Secondary Outcomes **Description:** Clinical examination. This outcome will be measured by the number of subjects not requiring amputation during the time frame of the study. **Measure:** (2.) limb salvage **Time Frame:** 6 months **Description:** The medical record will be reviewed on each subject and dates of hospitalizations for diabetic foot infection will be recorded. **Measure:** (3.) hospitalizations for recurrent diabetic foot infection **Time Frame:** 6 months **Description:** The medical record will be reviewed for each subject, and the dates and surgical procedures will be recorded. **Measure:** (4.) surgical procedures **Time Frame:** 6 months **Description:** The medical record will be reviewed for each subject and any recurrence of diabetic foot ulcers during the time frame of the study will be recorded. Data will include the date(s) of recurrence, location and size of the ulcer(s), and the presence or absence of infection. **Measure:** (5.) recurrent ulcers. **Time Frame:** 6 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Diagnosis of diabetes mellitus * Age ≥ 21 years * Infectious Disease Society of America stage 3 infection Exclusion Criteria: * History of previous bone infection in the study foot * Unable to provide informed consent * HIV, Hepatitis, osteomyelitis at other sites **Minimum Age:** 21 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Age \> 21, diabetes, Infectious Disease Society of America stage 3 infection (osteomyelitis) ### Contacts Locations Module #### Locations **Location 1:** **City:** Dallas **Country:** United States **Facility:** UT Southwestern Medical Center **State:** Texas **Zip:** 75390-9132 #### Overall Officials **Official 1:** **Affiliation:** UT Southwestern Medical Center **Name:** Lawrence Lavery **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000005534 - Term: Foot Diseases - ID: D000012871 - Term: Skin Diseases - ID: D000007871 - Term: Leg Ulcer - ID: D000012883 - Term: Skin Ulcer - ID: D000001850 - Term: Bone Diseases, Infectious - ID: D000007239 - Term: Infections - ID: D000001847 - Term: Bone Diseases - ID: D000009140 - Term: Musculoskeletal 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: BC01 - Name: Infections - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M7115 - Name: Diabetes Mellitus - 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: M17206 - Name: Ulcer - Relevance: LOW - As Found: Unknown - ID: M18919 - Name: Foot Ulcer - Relevance: HIGH - As Found: Foot Ulcer - ID: M12942 - Name: Osteomyelitis - Relevance: HIGH - As Found: Osteomyelitis - ID: M8658 - Name: Foot Diseases - Relevance: LOW - As Found: Unknown - ID: M15674 - Name: Skin Diseases - Relevance: LOW - As Found: Unknown - ID: M10883 - Name: Leg Ulcer - Relevance: LOW - As Found: Unknown - ID: M15686 - Name: Skin Ulcer - Relevance: LOW - As Found: Unknown - ID: M5126 - Name: Bone Diseases - Relevance: LOW - As Found: Unknown - ID: M5129 - Name: Bone Diseases, Infectious - Relevance: LOW - As Found: Unknown - ID: M12097 - Name: Musculoskeletal Diseases - Relevance: LOW - As Found: Unknown - ID: T4321 - Name: Osteomyelitis - Relevance: HIGH - As Found: Osteomyelitis ### Condition Browse Module - Meshes - ID: D000010019 - Term: Osteomyelitis - ID: D000016523 - Term: Foot Ulcer ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04925479 **Brief Title:** Study to Determine the Dose and Safety of Asciminib in Pediatric Patients With Chronic Myeloid Leukemia **Official Title:** A Multi-center, Open-label Study to Determine the Dose and Safety of Oral Asciminib in Pediatric Patients With Philadelphia Chromosome Positive Chronic Myeloid Leukemia in Chronic Phase (Ph+ CML-CP), Previously Treated With One or More Tyrosine Kinase Inhibitors #### Organization Study ID Info **ID:** CABL001I12201 #### Organization **Class:** INDUSTRY **Full Name:** Novartis #### Secondary ID Infos **ID:** 2021-001286-20 **Type:** EUDRACT_NUMBER ### Status Module #### Completion Date **Date:** 2031-11-01 **Type:** ESTIMATED #### Expanded Access Info **Has Expanded Access:** True **Status For NCT ID:** AVAILABLE #### Last Update Post Date **Date:** 2024-05-03 **Type:** ACTUAL **Last Update Submit Date:** 2024-05-01 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2026-06-30 **Type:** ESTIMATED #### Start Date **Date:** 2021-12-27 **Type:** ACTUAL **Status Verified Date:** 2024-05 #### Study First Post Date **Date:** 2021-06-14 **Type:** ACTUAL **Study First Submit Date:** 2021-05-18 **Study First Submit QC Date:** 2021-06-07 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Novartis Pharmaceuticals #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Oversight Has DMC:** True ### Description Module **Brief Summary:** The aim of this study is to support development of asciminib in the pediatric population (1 to \<18 years) previously treated with one or more TKIs. Full extrapolation of the efficacy of asciminib from adult to pediatric patients will be conducted. Full extrapolation is based on the concept that CML in the pediatric population has the same pathogenesis, similar clinical characteristics and progression pattern as in adults. **Detailed Description:** The aim of this study is to support development of asciminib in the pediatric population (1 to \<18 years) with Philadelphia chromosome positive chronic myeloid leukemia in chronic phase (PH+ CML-CP) previously treated with one or more Tyrosine kinase inhibitor (TKIs). The primary objective of this study is to characterize the pharmacokinetic (PK) profile of asciminib in pediatric patients with the goal of identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted). The pediatric formulation group will include at least 15 participants in each of the following two age categories: 1 to \<12 years and 12 to \<18 years; leading to at least 30 participants enrolled treated with the pediatric formulation. It will consist of a dose determination part (Part 1) and a cohort expansion (Part 2 BID regimen and Part 3 QD regimen). In Part 1, 4-6 participants will be enrolled in order to obtain at least 4 participants evaluable for PK (these participants may be from either of the age categories described above). The initial starting dose will be based on body weight and will be administered BID with food. Once the body weight adjusted dose has been determined in Part 1 of the study, the patients will be enrolled in Part 2 until at least 20 participants, including those who were included in Part 1, have been enrolled (10 per age group) in the pediatric formulation group. Once the interim safety and PK analysis 2 is completed for one of the age groups, the Part 3 QD regimen will open for the respective age group to enroll 10 patients (5 patients by age group). Due to the fact that the pediatric formulation was in development and was not available, this study started with the recruitment of adolescent patients. These participants aged 14 to \<18 years, weighing at least 40 kg receive the adult formulation at a flat dose of 40 mg BID under fasted conditions. The total duration of the treatment period of the study will be 5 years (260 weeks). Participants who, according to Investigator's judgement, are benefiting from study treatment will remain on treatment up to the completion of the treatment period (Week 260/5 years). The primary analysis for the BID regimen is planned after all participants in Part 1 and 2 have completed at least 52 weeks of study treatment or discontinued earlier. The primary analysis for combined regimen (BID+QD) is planned after all participants in Part 1, 2 and 3 have completed at least 52 weeks of study treatment or discontinued earlier. ### Conditions Module **Conditions:** - Myeloid Leukemia, Philadelphia Positive **Keywords:** - Asciminib - pediatric patients - Philadelphia chromosome positive chronic myeloid leukemia in chronic phase - Ph+ CML-CP - ABL001 ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 34 **Type:** ESTIMATED **Phases:** - PHASE1 - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** This arm consists of 2 groups: * The pediatric formulation group where the dose is based on body weight (1.3mg/kg) * The adult formulation group where participants will receive a flat dose of 40mg BID **Intervention Names:** - Drug: Asciminib Pediatric formulation group - Drug: Asciminib Adult formulation group **Label:** Asciminib **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Asciminib **Description:** Asciminib Pediatric formulation group: Mini-tablets will be supplied as size 0 capsules containing 1 mg mini-tablets, taken orally: 10 mg (10x 1 mg tablets in capsule) 15 mg (15x 1 mg tablets in capsule) 20 mg (20x 1 mg tablets in capsule) 30 mg (30x 1 mg tablets in capsule) **Name:** Asciminib Pediatric formulation group **Other Names:** - ABL001 **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Asciminib **Description:** Asciminib Adult formulation group: 40 mg tablets BID, taken orally. 20 mg tablets BID, taken orally. **Name:** Asciminib Adult formulation group **Other Names:** - ABL001 **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted). **Measure:** Primary Pharmacokinetic (PK) parameter: AUClast **Time Frame:** 52 weeks **Description:** Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted). **Measure:** Primary PK parameter: AUCtau **Time Frame:** 52 weeks **Description:** Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted). **Measure:** Secondary PK parameter: Cmax **Time Frame:** 52 weeks **Description:** Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted). **Measure:** Secondary PK parameter: Tmax **Time Frame:** 52 weeks **Description:** Goal: identifying the pediatric formulation dose (fed) leading to asciminib exposure comparable to 40 mg BID in adult patients (fasted). **Measure:** Secondary PK parameter: Ctrough **Time Frame:** 52 weeks #### Secondary Outcomes **Description:** Complete hematological response will be defined as all of the following present for ≥ 4 weeks: * WBC count \< 10 x 10\^9/L * Platelet count \< 450 x 10\^9/L * Basophils \< 5% * No blasts and promyelocytes in peripheral blood * Myelocytes + metamyelocytes \< 5% in peripheral blood * No evidence of extramedullary disease, including spleen and liver **Measure:** Hematologic responses **Time Frame:** 52 weeks **Description:** To assess pharmacodynamic markers of asciminib's anti-leukemic activity. Molecular response will be assessed by Breakpoint Cluster Region gene-Abelson proto-oncogene (BCR-ABL) 1 level. **Measure:** Molecular responses **Time Frame:** 52 weeks **Description:** To assess acceptability and palatability of the pediatric formulation **Measure:** Questionnaire on acceptability and palatability after first dose, 4 and 52 weeks **Time Frame:** after first dose at Week 1 Day 1, 4 weeks, 52 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: - Male or female participants: Pediatric formulation group: ≥ 1 and less than 18 years of age at study entry. Adult formulation group: ≥ 14 and less than 18 years of age and body weight of ≥ 40 kg at study entry. * Participants with Ph+ CML-CP must meet all of the following laboratory values at the screening visit. In the case where bone marrow blast and promyelocyte counts are available, these will be accepted if done within 56 days prior to the screening visit, to avoid unnecessary repetition of this test. 1. \< 15% blasts in peripheral blood and bone marrow 2. \< 30% combined blasts plus promyelocytes in peripheral blood and bone marrow 3. \< 20% basophils in the peripheral blood 4. Neutrophils ≥ 1.5 x 10\^9/L (or WBC ≥ 3 x 10\^9/L if neutrophils are not available) and platelet count ≥ 100 x 10\^9/L 5. No evidence of extramedullary leukemic involvement, with the exception of hepatosplenomegaly * Prior treatment with a minimum of one TKI * Failure (adapted from the 2020 European Leukemia Net (ELN) Guidelines Hochhaus et al 2020 and 2013 ELN Guidelines Baccarani et al 2013) or intolerance to the most recent TKI therapy at the time of screening. * Performance status: Karnofsky ≥ 50% for patients ≥ 16 years of age, and Lansky ≥ 50 for patients \< 16 years of age at the time of screening * Participants must have adequate renal, hepatic, pancreatic and cardiac function * Participants must have electrolyte values within normal limits or corrected to be within normal limits with supplements prior to first dose of study medication: * Evidence of typical BCR-ABL1 transcript \[e14a2 and/or e13a2\] at the time of screening which are amenable to standardized RQ-PCR quantification. Exclusion Criteria: * Known presence of the T315I mutation prior to study entry. * Known second chronic phase of CML after previous progression to AP/BC. * Previous treatment with a hematopoietic stem-cell transplantation. * Patient planning to undergo allogeneic hematopoietic stem cell transplantation. * Cardiac or cardiac repolarization abnormality * Severe and/or uncontrolled concurrent medical disease that in the opinion of the Investigator could cause unacceptable safety risks or compromise compliance with the protocol * History of acute pancreatitis within 1 year of study entry or past medical history of chronic pancreatitis. * History of acute or chronic liver disease. * Impairment of gastrointestinal (GI) function or GI disease that may significantly alter the absorption of study drug * Pregnant or nursing (lactating) females. Other protocol-defined inclusion/exclusion may apply. **Maximum Age:** 18 Years **Minimum Age:** 1 Year **Sex:** ALL **Standard Ages:** - CHILD - ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Novartis Pharmaceuticals **Phone:** 1-888-669-6682 **Role:** CONTACT **Contact 2:** **Name:** Novartis Pharmaceuticals **Phone:** +41613241111 **Role:** CONTACT #### Locations **Location 1:** **City:** Boston **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Taylor Sorrentino - **Role:** CONTACT ***Contact 2:*** - **Name:** Jessica Pollard - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Dana Farber Cancer Institute Dept.of DFCI **State:** Massachusetts **Status:** RECRUITING **Zip:** 02215 **Location 2:** **City:** New York **Contacts:** ***Contact 1:*** - **Phone:** 212-305-9770 - **Role:** CONTACT ***Contact 2:*** - **Name:** Nobuko Hijiya - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Columbia University Medical Center- New York Presbyterian Herbert Irving Cancer Center **State:** New York **Status:** RECRUITING **Zip:** 10032 **Location 3:** **City:** Cincinnati **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Jenni Ho - **Phone:** 800-344-2462 - **Role:** CONTACT ***Contact 2:*** - **Name:** Benjamin Mizukawa - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Cinn Children Hosp Medical Center **State:** Ohio **Status:** RECRUITING **Zip:** 45229-3039 **Location 4:** **City:** Salt Lake City **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Keeley Best - **Phone:** 801-213-3599 - **Role:** CONTACT ***Contact 2:*** - **Name:** Mallorie Heneghan - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** University of Utah Primary Childrens Hospital **State:** Utah **Status:** RECRUITING **Zip:** 84132 **Location 5:** **City:** Hangzhou **Country:** China **Facility:** Novartis Investigative Site **State:** Zhejiang **Status:** RECRUITING **Zip:** 310005 **Location 6:** **City:** Beijing **Country:** China **Facility:** Novartis Investigative Site **Status:** RECRUITING **Zip:** 100044 **Location 7:** **City:** Shanghai **Country:** China **Facility:** Novartis Investigative Site **Status:** RECRUITING **Zip:** 200127 **Location 8:** **City:** Tianjin **Country:** China **Facility:** Novartis Investigative Site **Status:** RECRUITING **Zip:** 300020 **Location 9:** **City:** Bordeaux Cedex **Country:** France **Facility:** Novartis Investigative Site **Status:** RECRUITING **Zip:** 33076 **Location 10:** **City:** Lille **Country:** France **Facility:** Novartis Investigative Site **Status:** RECRUITING **Zip:** 59000 **Location 11:** **City:** Paris **Country:** France **Facility:** Novartis Investigative Site **Status:** RECRUITING **Zip:** 75019 **Location 12:** **City:** Poitiers **Country:** France **Facility:** Novartis Investigative Site **Status:** RECRUITING **Zip:** 86021 **Location 13:** **City:** Erlangen **Country:** Germany **Facility:** Novartis Investigative Site **Status:** RECRUITING **Zip:** 91054 **Location 14:** **City:** Essen **Country:** Germany **Facility:** Novartis Investigative Site **Status:** RECRUITING **Zip:** 45147 **Location 15:** **City:** Hamburg **Country:** Germany **Facility:** Novartis Investigative Site **Status:** RECRUITING **Zip:** 20246 **Location 16:** **City:** Athens **Country:** Greece **Facility:** Novartis Investigative Site **Status:** RECRUITING **Zip:** 115 27 **Location 17:** **City:** Budapest **Country:** Hungary **Facility:** Novartis Investigative Site **Status:** RECRUITING **Zip:** 1094 **Location 18:** **City:** Genova **Country:** Italy **Facility:** Novartis Investigative Site **State:** GE **Status:** RECRUITING **Zip:** 16147 **Location 19:** **City:** Monza **Country:** Italy **Facility:** Novartis Investigative Site **State:** MB **Status:** RECRUITING **Zip:** 20900 **Location 20:** **City:** Torino **Country:** Italy **Facility:** Novartis Investigative Site **State:** TO **Status:** RECRUITING **Zip:** 10126 **Location 21:** **City:** Yokohama-city **Country:** Japan **Facility:** Novartis Investigative Site **State:** Kanagawa **Status:** RECRUITING **Zip:** 232-8555 **Location 22:** **City:** Shinjuku-ku **Country:** Japan **Facility:** Novartis Investigative Site **State:** Tokyo **Status:** RECRUITING **Zip:** 160 8582 **Location 23:** **City:** Osaka **Country:** Japan **Facility:** Novartis Investigative Site **Status:** RECRUITING **Zip:** 534-0021 **Location 24:** **City:** Seoul **Country:** Korea, Republic of **Facility:** Novartis Investigative Site **State:** Korea **Status:** RECRUITING **Zip:** 05505 **Location 25:** **City:** Seoul **Country:** Korea, Republic of **Facility:** Novartis Investigative Site **Status:** RECRUITING **Zip:** 03080 **Location 26:** **City:** Utrecht **Country:** Netherlands **Facility:** Novartis Investigative Site **State:** CS **Status:** RECRUITING **Zip:** 3584 **Location 27:** **City:** Wrocław **Country:** Poland **Facility:** Novartis Investigative Site **Status:** RECRUITING **Zip:** 50367 **Location 28:** **City:** Moscow **Country:** Russian Federation **Facility:** Novartis Investigative Site **Status:** RECRUITING **Zip:** 117198 **Location 29:** **City:** Saint Petersburg **Country:** Russian Federation **Facility:** Novartis Investigative Site **Status:** RECRUITING **Zip:** 197022 **Location 30:** **City:** Muang **Country:** Thailand **Facility:** Novartis Investigative Site **State:** Chiangmai **Status:** RECRUITING **Zip:** 50200 **Location 31:** **City:** Bangkok **Country:** Thailand **Facility:** Novartis Investigative Site **Status:** RECRUITING **Zip:** 10400 **Location 32:** **City:** Khon Kaen **Country:** Thailand **Facility:** Novartis Investigative Site **Status:** RECRUITING **Zip:** 40000 **Location 33:** **City:** Bursa **Country:** Turkey **Facility:** Novartis Investigative Site **State:** Gorukle **Status:** RECRUITING **Zip:** 16059 **Location 34:** **City:** Istanbul **Country:** Turkey **Facility:** Novartis Investigative Site **Status:** RECRUITING **Zip:** 34093 #### Overall Officials **Official 1:** **Affiliation:** Novartis Pharmaceuticals **Name:** Novartis Pharmaceuticals **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **Description:** Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent expert panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data is currently available according to the process described on www.clinicalstudydatarequest.com. **IPD Sharing:** YES **URL:** https://www.clinicalstudydatarequest.com ## 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: D000014178 - Term: Translocation, Genetic - ID: D000002869 - Term: Chromosome Aberrations ### 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: Myeloid Leukemia - ID: M18123 - Name: Leukemia, Myelogenous, Chronic, BCR-ABL Positive - Relevance: HIGH - As Found: Chronic Myeloid Leukemia - ID: M13582 - Name: Philadelphia Chromosome - Relevance: HIGH - As Found: Philadelphia Chromosome - 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: M16932 - Name: Translocation, Genetic - Relevance: LOW - As Found: Unknown - ID: M6109 - Name: Chromosome Aberrations - 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: LOW - As Found: Unknown - 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: D000010677 - Term: Philadelphia Chromosome ### Intervention Browse Module - Ancestors - ID: D000092004 - Term: Tyrosine Kinase Inhibitors - ID: D000047428 - Term: Protein Kinase Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000014803 - Term: Vitamin B Complex - ID: D000014815 - Term: Vitamins - ID: D000018977 - Term: Micronutrients - ID: D000045505 - Term: Physiological Effects of Drugs ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Micro - Name: Micronutrients - Abbrev: VaDiAg - Name: Vasodilator Agents - Abbrev: Lipd - Name: Lipid Regulating Agents - Abbrev: Hemat - Name: Hematinics - Abbrev: AA - Name: Amino Acids - Abbrev: Vi - Name: Vitamins ### Intervention Browse Module - Browse Leaves - ID: M81032 - Name: Asciminib - Relevance: HIGH - As Found: Factorial - ID: M2889 - Name: Tyrosine Kinase Inhibitors - Relevance: LOW - As Found: Unknown - ID: M12476 - Name: Niacinamide - Relevance: HIGH - As Found: Factorial - ID: M12465 - Name: Niacin - Relevance: LOW - As Found: Unknown - ID: M12479 - Name: Nicotinic Acids - 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: M8618 - Name: Folic Acid - 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: M21009 - Name: Micronutrients - Relevance: LOW - As Found: Unknown - ID: M16885 - Name: Trace Elements - Relevance: LOW - As Found: Unknown - ID: T22 - Name: Tyrosine - Relevance: LOW - As Found: Unknown - ID: T455 - Name: Nicotinamide - Relevance: LOW - As Found: Unknown - ID: T453 - Name: Niacin - Relevance: LOW - As Found: Unknown - ID: T454 - Name: Niacinamide - Relevance: LOW - As Found: Unknown - ID: T456 - Name: Nicotinic Acid - Relevance: LOW - As Found: Unknown - ID: T471 - Name: Vitamin B3 - 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 ### Intervention Browse Module - Meshes - ID: D000009536 - Term: Niacinamide - ID: C000621806 - Term: Asciminib ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00924079 **Acronym:** NAL **Brief Title:** The Development of Oral Nalbuphine Dosage Form **Official Title:** Pharmacokinetic Study of Oral Nalbuphine in Normal Healthy Subjects #### Organization Study ID Info **ID:** NAL001 #### Organization **Class:** OTHER **Full Name:** Tri-Service General Hospital #### Secondary ID Infos **ID:** TSGHIRB096-02-010-I ### Status Module #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2009-06-19 **Type:** ESTIMATED **Last Update Submit Date:** 2009-06-18 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2009-11 **Type:** ESTIMATED #### Start Date **Date:** 2008-09 **Status Verified Date:** 2009-06 #### Study First Post Date **Date:** 2009-06-18 **Type:** ESTIMATED **Study First Submit Date:** 2009-06-17 **Study First Submit QC Date:** 2009-06-17 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Tri-Service General Hospital #### Responsible Party **Old Name Title:** Ho Shung-Tai/Professor of the department of anesthsiology **Old Organization:** Department of anesthsiology of Tri-service General Hospitial ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of this study is to investigate possible responses to pharmacokinetic properties for nalbuphine oral formulations in healthy volunteers. ### Conditions Module **Conditions:** - Healthy **Keywords:** - Analysis of plasma concentration of nalbuphine ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** CROSSOVER ##### Masking Info **Masking:** NONE #### Enrollment Info **Count:** 60 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Drug: nalbuphine **Label:** nalbuphine **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - nalbuphine **Description:** oral dosage form, 66 mg, single dose **Name:** nalbuphine **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Pharmacokinetic parameter eq Cmax, AUC, Cl, T1/2 **Time Frame:** 24 hours ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Normal healthy adult subjects between 20-40 years of age. * Body weight within 80-120% of ideal body weight. Ideal body weight = (height-80)0.7 * Acceptable medical history and physical examination including: * Normal chest X-ray and ECG results within six months prior to Period I dosing. * No particular clinical significance in general disease history within two months prior to Period I dosing. * Acceptable clinical laboratory determinations without significant deviation from normal values within two months prior to Period I dosing, which includes: * AST (SGOT) * ALT (SGPT) * Gama-GT * alkaline phosphatase * total bilirubin * albumin * glucose * BUN * uric acid * creatinine * total cholesterol * triglyceride(TG) * Acceptable hematology within two months prior to the study, which includes hemoglobin, hematocrit, red blood cells, MCV, MCH, MCHC, white blood cells, differential white blood cells and platelets. * Acceptable urinalysis within two months prior to the study, which includes pH, blood, glucose and protein. * Signed the written informed consent to participate in this study. Exclusion Criteria: * Recent history of drug or alcohol addiction or abuse. * A clinically significant disorder involving the cardiovascular, respiratory, renal, gastrointestinal, immunologic, hematologic, endocrine or neurologic system(s) or psychiatric disease (as determined by the clinical investigator). * History of allergic response(s) to nalbuphine or related drugs. * History of clinically significant allergies including drug allergies or allergic bronchial asthma. * Evidence of chronic or acute infectious diseases. * Any clinically significant illness or surgery during the four weeks prior to Period I dosing (as determined by the clinical investigator). * Taking any drug known to induce or inhibit hepatic drug metabolism within one month prior to the beginning of the study. * Receiving any investigational drug within one month prior to Period I dosing. * Taking any prescription medication or any nonprescription medication within two weeks prior to Period I doing. * Donating greater than 150 ml of blood within two months prior to Period I dosing or donating plasma (e.g., plasmapheresis) within 14 days prior to Period I dosing. All subjects will be advised not to donate blood for four weeks after completing the study. * Consumption of caffeine, xanthine-containing products (i.e., coffee, tea, caffeine-containing sodas, colas and chocolate, etc.) and/or alcohol at least 48 hours prior to days on which dosing is scheduled and during the periods when blood samples are being collected. * Any other medical reason as determined by the clinical investigator. * Patient is pregnant or breastfeeding. Women of childbearing potential must have a negative urine pregnancy test at Baseline. **Healthy Volunteers:** True **Maximum Age:** 40 Years **Minimum Age:** 20 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Taipei **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Ho Shung-Tai, MD - **Phone:** 886-2-87927125 - **Role:** CONTACT ***Contact 2:*** - **Name:** Ho Shung-Tai, MD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Taiwan **Facility:** Tri-Service General Hospitial **Status:** RECRUITING **Zip:** 11490 ## Derived Section ### 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 ### 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: M12217 - Name: Nalbuphine - Relevance: HIGH - As Found: Cabozantinib - 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 ### Intervention Browse Module - Meshes - ID: D000009266 - Term: Nalbuphine ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00723879 **Brief Title:** Adherence in Patients Receiving PegIntron and Rebetol for Hepatitis C in Conjunction With a Patient Assistance Program (Study P04206) **Official Title:** Evaluation of Adherence Rate in Patients Receiving PegIntron / Rebetol (Weight Based) for Hepatitis C in Conjunction With a Patient Assistance Program. #### Organization Study ID Info **ID:** P04206 #### Organization **Class:** INDUSTRY **Full Name:** Merck Sharp & Dohme LLC ### Status Module #### Completion Date **Date:** 2008-07 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2015-11-03 **Type:** ESTIMATED **Last Update Submit Date:** 2015-11-02 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2008-07 **Type:** ACTUAL #### Start Date **Date:** 2004-10 **Status Verified Date:** 2015-11 #### Study First Post Date **Date:** 2008-07-29 **Type:** ESTIMATED **Study First Submit Date:** 2008-07-25 **Study First Submit QC Date:** 2008-07-25 **Why Stopped:** Because of slow recruitment; Despite the study was started long time ago (Oct 2004), we have not reached the projected number of patients (150 patients) ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Merck Sharp & Dohme LLC #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Patients receiving a patient assistance program during therapy for Hepatitis C will be enrolled into this study. All patients will receive PegIntron and Rebetol (according to the label) and the patient assistance program, which includes (1) medications used for treatment (psychiatric medications); (2) other interventions (nurse support); and (3) educational literature. This study will be compared to similar studies from other clinics using various patient support programs for the purpose of designing future comparative phase IV studies. ### Conditions Module **Conditions:** - Hepatitis C, Chronic - Hepacivirus ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 115 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients receiving a patient assistance program during therapy for hepatitis C will be enrolled into this study. All patients will receive PegIntron plus Rebetol (according to the label) and the patient assistance program. **Intervention Names:** - Biological: Peginterferon alfa-2b (SCH 54031) - Drug: Ribavirin (SCH 18908) - Behavioral: Patient assistance program **Label:** Patients with hepatitis C ### Interventions #### Intervention 1 **Arm Group Labels:** - Patients with hepatitis C **Description:** Peginterferon alfa-2b and ribavirin will be administered according to the products' labeling. **Name:** Peginterferon alfa-2b (SCH 54031) **Other Names:** - PegIntron **Type:** BIOLOGICAL #### Intervention 2 **Arm Group Labels:** - Patients with hepatitis C **Description:** Peginterferon alfa-2b and ribavirin will be administered according to the products' labeling. **Name:** Ribavirin (SCH 18908) **Other Names:** - Rebetol **Type:** DRUG #### Intervention 3 **Arm Group Labels:** - Patients with hepatitis C **Description:** Patient assistance programs will be classified as follows: 1. medications, including psychiatric medications (antidepressants); 2. other interventions, including nurse telephone calls and nurse support in the office: and 3. educational literature about hepatitis C containing information about the disease, mode of transmission, complications, and treatment and treatment drawbacks that will be given to each patient at the start of treatment. **Name:** Patient assistance program **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Measure:** Proportion of patients who complete treatment with PegIntron plus Rebetol therapy for hepatitis C when administered with a patient assistance program. **Time Frame:** NA (i.e. the study was terminated) **Measure:** Average length of treatment for patients receiving a patient assistance program as part of their treatment for hepatitis C with PegIntron plus Rebetol. **Time Frame:** NA (i.e. the study was terminated) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Patients with chronic hepatitis C naive to treatment Exclusion Criteria: * None **Minimum Age:** 18 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Patients receiving a patient assistance program during therapy for Hepatitis C will be enrolled into this study. All patients will receive PegIntron and Rebetol (according to the label) and the patient assistance program, which includes (1) medications used for treatment (psychiatric medications); (2) other interventions (nurse support); and (3) educational literature. ## 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: D000006521 - Term: Hepatitis, Chronic - ID: D000002908 - Term: Chronic Disease - 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: BC01 - Name: Infections - Abbrev: BC06 - Name: Digestive System Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M5114 - Name: Body Weight - Relevance: LOW - As Found: Unknown - ID: M9592 - Name: Hepatitis A - Relevance: HIGH - As Found: Hepatitis - ID: M9591 - Name: Hepatitis - Relevance: HIGH - As Found: Hepatitis - ID: M9611 - Name: Hepatitis C - Relevance: HIGH - As Found: Hepatitis C - ID: M21613 - Name: Hepatitis C, Chronic - Relevance: HIGH - As Found: Hepatitis C, Chronic - 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: M9607 - Name: Hepatitis, Chronic - 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 ### Condition Browse Module - Meshes - ID: D000006506 - Term: Hepatitis A - ID: D000006526 - Term: Hepatitis C - ID: D000019698 - Term: Hepatitis C, Chronic - ID: D000006505 - Term: Hepatitis ### Intervention Browse Module - Ancestors - ID: D000000963 - Term: Antimetabolites - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000000998 - Term: Antiviral Agents - ID: D000000890 - Term: Anti-Infective Agents ### Intervention Browse Module - Browse Branches - Abbrev: PsychDr - Name: Psychotropic Drugs - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Infe - Name: Anti-Infective Agents ### Intervention Browse Module - Browse Leaves - ID: M4247 - Name: Antidepressive Agents - Relevance: LOW - As Found: Unknown - ID: M254669 - Name: Peginterferon alfa-2b - Relevance: HIGH - As Found: Detected - ID: M15083 - Name: Ribavirin - Relevance: HIGH - As Found: Bevacizumab - ID: M4281 - Name: Antimetabolites - Relevance: LOW - As Found: Unknown - ID: M4314 - Name: Antiviral Agents - Relevance: LOW - As Found: Unknown - ID: M4214 - Name: Anti-Infective Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000012254 - Term: Ribavirin - ID: C000417083 - Term: Peginterferon alfa-2b ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00982579 **Acronym:** PedVacc001 **Brief Title:** Safety and Immunogenicity Study of Candidate HIV-1 Vaccine Given to Healthy Infants Born to HIV-1/2-uninfected Mothers **Official Title:** An Open Randomized Phase I Study Evaluating Safety and Immunogenicity of a Candidate HIV-1 Vaccine, MVA.HIVA, Administered to Healthy Infants Born to HIV-1/2-uninfected Mothers #### Organization Study ID Info **ID:** PV001 #### Organization **Class:** OTHER_GOV **Full Name:** Medical Research Council ### Status Module #### Completion Date **Date:** 2011-09 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2012-02-03 **Type:** ESTIMATED **Last Update Submit Date:** 2012-02-02 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2011-06 **Type:** ACTUAL #### Start Date **Date:** 2009-11 **Status Verified Date:** 2009-09 #### Study First Post Date **Date:** 2009-09-23 **Type:** ESTIMATED **Study First Submit Date:** 2009-09-22 **Study First Submit QC Date:** 2009-09-22 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER_GOV **Name:** European and Developing Countries Clinical Trials Partnership (EDCTP) #### Lead Sponsor **Class:** OTHER_GOV **Name:** Medical Research Council #### Responsible Party **Old Name Title:** Dr. Tomas Hanke **Old Organization:** Medical Research Council, UK ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** Objectives: Safety and immunogenicity of MVA.HIVA vaccine in 20-week-old healthy Gambian infants born to HIV-1/2-uninfected mothers. Gross impact of MVA.HIVA on the immunogenicity of EPI vaccines (DTwPHib, HepB, PCV-7 and OPV) when administered at 20 weeks (4 weeks after the last EPI vaccines), who have had BCG vaccine within the first 4 weeks of life. ### Conditions Module **Conditions:** - HIV-1 - HIV Infections **Keywords:** - HIV preventive vaccine ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 48 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Vaccinated at 20 weeks of age (n=24) **Intervention Names:** - Biological: MVA.HIVA **Label:** Vaccinees **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** No experimental vaccine (n=24) **Label:** Controls **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Vaccinees **Description:** 1 dose of 5 x 10\^7 pfu of MVA.HIVA administered intramuscularly **Name:** MVA.HIVA **Type:** BIOLOGICAL ### Outcomes Module #### Primary Outcomes **Measure:** For safety and reactogenicity: Actively and passively collected data on adverse events **Time Frame:** Up to 16 weeks after vaccination #### Secondary Outcomes **Measure:** For immunity to EPI vaccines: Antibody levels to specific vaccines. **Time Frame:** 1 week before and 1 week after vaccination **Measure:** For immunogenicity: Frequency of IFN-γ producing cells determined in ex-vivo (effector) and 10-day cultured (memory) ELISPOT assays after overnight stimulation with pools of HIVA-derived peptides **Time Frame:** Up to 16 weeks after vaccination ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Healthy infants, 19 weeks of age, with weight for age z-scores within 2 standard deviations of normal. * Have received all standard EPI immunizations according to national immunization programme. * Written informed consent by parent. * Mother HIV-1/2-uninfected. Exclusion Criteria: * Acute disease at the time of vaccination (acute disease is defined as the presence of a moderate or severe illness with or without fever). All vaccines can be administered to persons with a minor illness such as diarrhoea, mild upper respiratory tract infection with or without low-grade febrile illness, i.e. axillary temperature of \<37.5 °C ). * Axillary temperature of ≥ 37.5 °C at the time of vaccination. * Any clinically significant abnormal finding on screening from biochemistry or haematology at 19 weeks. * History of allergic disease or reactions likely to be exacerbated by any component of the vaccine, e.g. egg products. * Presence of any underlying disease that compromises the diagnosis and evaluation of response to the vaccine. * Invasive bacterial infections (pneumonia, meningitis). * Any other on-going chronic illness requiring hospital specialist supervision. * Administration of immunoglobulins and/or any blood products within one month preceding the planned administration of the vaccine candidate. * Any history of anaphylaxis in reaction to vaccination. * Research physician's assessment of lack of willingness by parents to participate and comply with all requirements of the protocol, or identification of any factor felt to significantly increase the infant's risk of suffering an adverse outcome. * Likelihood of travel away from the study area. * Untreated malaria infection. * Any other clinical evidence of infection. **Healthy Volunteers:** True **Maximum Age:** 3 Days **Sex:** ALL **Standard Ages:** - CHILD ### Contacts Locations Module #### Locations **Location 1:** **City:** Banjul **Country:** Gambia **Facility:** Medical Research Council Laboratories, The Gambia **State:** Fajara #### Overall Officials **Official 1:** **Affiliation:** Medical Research Council **Name:** Tomas Hanke **Role:** STUDY_DIRECTOR **Official 2:** **Affiliation:** Medical Research Council, The Gambia **Name:** Katie Flanagan **Role:** PRINCIPAL_INVESTIGATOR **Official 3:** **Affiliation:** Karolinska Institutet **Name:** Marie Reilly **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Afolabi MO, Ndure J, Drammeh A, Darboe F, Mehedi SR, Rowland-Jones SL, Borthwick N, Black A, Ambler G, John-Stewart GC, Reilly M, Hanke T, Flanagan KL. A phase I randomized clinical trial of candidate human immunodeficiency virus type 1 vaccine MVA.HIVA administered to Gambian infants. PLoS One. 2013 Oct 24;8(10):e78289. doi: 10.1371/journal.pone.0078289. eCollection 2013. **PMID:** 24205185 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000086982 - Term: Blood-Borne Infections - ID: D000003141 - Term: Communicable Diseases - ID: D000007239 - Term: Infections - ID: D000015229 - Term: Sexually Transmitted Diseases, Viral - ID: D000012749 - Term: Sexually Transmitted Diseases - ID: D000016180 - Term: Lentivirus Infections - ID: D000012192 - Term: Retroviridae Infections - ID: D000012327 - Term: RNA Virus Infections - ID: D000014777 - Term: Virus Diseases - ID: D000091662 - Term: Genital Diseases - ID: D000091642 - Term: Urogenital Diseases - ID: D000007153 - Term: Immunologic Deficiency Syndromes - ID: D000007154 - Term: Immune System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC01 - Name: Infections - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: BC20 - Name: Immune System Diseases ### Condition Browse Module - Browse Leaves - ID: M10283 - Name: Infections - Relevance: LOW - As Found: Unknown - ID: M6368 - Name: Communicable Diseases - Relevance: LOW - As Found: Unknown - ID: M3522 - Name: Acquired Immunodeficiency Syndrome - Relevance: LOW - As Found: Unknown - ID: M18250 - Name: HIV Infections - Relevance: HIGH - As Found: HIV Infections - ID: M2593 - Name: Blood-Borne Infections - Relevance: LOW - As Found: Unknown - ID: M15558 - Name: Sexually Transmitted Diseases - Relevance: LOW - As Found: Unknown - ID: M17933 - Name: Sexually Transmitted Diseases, Viral - Relevance: LOW - As Found: Unknown - ID: M18640 - Name: Lentivirus Infections - Relevance: LOW - As Found: Unknown - ID: M15026 - Name: Retroviridae 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: M2876 - Name: Genital Diseases - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M16355 - Name: Syndrome - Relevance: LOW - As Found: Unknown - ID: M10199 - Name: Immunologic Deficiency Syndromes - Relevance: LOW - As Found: Unknown - ID: M10200 - Name: Immune System Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000015658 - Term: HIV Infections ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M17360 - Name: Vaccines - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03897179 **Brief Title:** INVSENSOR00032 and INVSENSOR00033 Respiration Rate Clinical Performance Study **Official Title:** INVSENSOR00032 and INVSENSOR00033 Respiration Rate Clinical Performance Study #### Organization Study ID Info **ID:** TP-19608 #### Organization **Class:** INDUSTRY **Full Name:** Masimo Corporation ### Status Module #### Completion Date **Date:** 2019-02-25 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2020-03-27 **Type:** ACTUAL **Last Update Submit Date:** 2020-03-13 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2019-02-25 **Type:** ACTUAL #### Results First Post Date **Date:** 2020-03-27 **Type:** ACTUAL **Results First Submit Date:** 2020-03-13 **Results First Submit QC Date:** 2020-03-13 #### Start Date **Date:** 2019-02-13 **Type:** ACTUAL **Status Verified Date:** 2020-03 #### Study First Post Date **Date:** 2019-04-01 **Type:** ACTUAL **Study First Submit Date:** 2019-03-20 **Study First Submit QC Date:** 2019-03-28 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Masimo Corporation #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** True **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** This study compares the performance of respiration rate from pleth measured prospectively with either INVSENSOR00032 and/or INVSENSOR00033 devices against the respiration rate derived from the manual scoring of the capnography waveform in healthy adult subjects. ### Conditions Module **Conditions:** - Healthy ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** OTHER #### Enrollment Info **Count:** 27 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** All subjects will be enrolled into the test group and will receive the INVSENSOR00032 and/or INVSENSOR00033 device. **Intervention Names:** - Device: INVSENSOR00032 and INVSENSOR00033 **Label:** INVSENSOR00032 and INVSENSOR00033 test group **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - INVSENSOR00032 and INVSENSOR00033 test group **Description:** Investigational pulse oximeter device **Name:** INVSENSOR00032 and INVSENSOR00033 **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Accuracy will be determined by comparing the noninvasive respiratory rate by pleth (RRp) measurement of the INVSENSOR00032 and/or INVSENSOR00033 to the respiratory rate reference (RRef) and calculating the arithmetic root mean square (Arms) value. In order to obtain the Arms value, the RR measurement from the reference is subtracted from the INVSENSOR00032 and/or INVSENSOR00033 RRp measurement for a number of samples. The average of this difference is computed as the bias. The standard deviation of the differences is computed as the precision. The square root of the sum of the squares of bias and precision is computed as the Arms value. **Measure:** RRp Arms of Sensor Accuracy **Time Frame:** 1-5 hours ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Physical status of ASA I or II * Must be able to read and communicate in English * Has signed all necessary related documents, e.g. written informed consent, confidentiality agreement. * Passed health assessment screening * Negative pregnancy test for female subjects of child bearing potential. Exclusion Criteria: * Physical status of ASA III, IV, or V * Subject has any medical condition which in the judgment of the investigator, renders them inappropriate for participation in this study * Inability to tolerate sitting still or minimal movement for at least 30 minutes * Positive pregnancy test for female subjects * Refusal to take pregnancy test for women of child bearing potential * Nursing female subjects * Subjects wearing acrylic nails or subjects refusing to remove nail polish * Subjects who have a nail deformity on the measurement finger * Subjects who do not have adequate skin integrity on the measurement finger * Excluded at the Principal Investigator's discretion **Healthy Volunteers:** True **Maximum Age:** 70 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Irvine **Country:** United States **Facility:** Masimo Corporation **State:** California **Zip:** 92618 ## Document Section ### Large Document Module #### Large Docs - Date: 2018-12-18 - Filename: Prot_SAP_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: True - Label: Study Protocol and Statistical Analysis Plan - Size: 10638711 - Type Abbrev: Prot_SAP - Upload Date: 2020-03-13T18:20 ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** INVSENSOR00032 and INVSENSOR00033 Test Group **Deaths Num At Risk:** 27 **Description:** All subjects will be enrolled into the test group and will receive the INVSENSOR00032 and/or INVSENSOR00033 device. INVSENSOR00032 and INVSENSOR00033: Investigational pulse oximeter device **ID:** EG000 **Other Num at Risk:** 27 **Serious Number At Risk:** 27 **Title:** INVSENSOR00032 and INVSENSOR00033 Test Group **Frequency Threshold:** 0 **Time Frame:** Adverse event data was collected over the course of the study (13 days). ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 27 **Units:** Participants ### Group **ID:** BG000 **Title:** INVSENSOR00032 and INVSENSOR00033 Test Group **Description:** All subjects will be enrolled into the test group and will receive the INVSENSOR00032 and/or INVSENSOR00033 device. INVSENSOR00032 and INVSENSOR00033: Investigational pulse oximeter device ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** <=18 years #### Measurement **Group ID:** BG000 **Value:** 26 **Category Title:** Between 18 and 65 years #### Measurement **Group ID:** BG000 **Value:** 1 **Category Title:** >=65 years **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 13 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 14 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 8 **Class Title:** African American #### Measurement **Group ID:** BG000 **Value:** 4 **Class Title:** Asian or Pacific Islander #### Measurement **Group ID:** BG000 **Value:** 6 **Class Title:** Caucasian #### Measurement **Group ID:** BG000 **Value:** 9 **Class Title:** Hispanic ### Measure #### Measurement **Group ID:** BG000 **Value:** 27 **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 **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:** COUNT_OF_PARTICIPANTS **Title:** Region of Enrollment **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement **PI Sponsor Employee:** True ### Point of Contact **Email:** [email protected] **Organization:** Masimo **Phone:** 949-297-7000 **Title:** Ahmed Alghazi ## Results Section - Outcome Measures Module ### Outcome Measure 1 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2.1 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Accuracy will be determined by comparing the noninvasive respiratory rate by pleth (RRp) measurement of the INVSENSOR00032 and/or INVSENSOR00033 to the respiratory rate reference (RRef) and calculating the arithmetic root mean square (Arms) value. In order to obtain the Arms value, the RR measurement from the reference is subtracted from the INVSENSOR00032 and/or INVSENSOR00033 RRp measurement for a number of samples. The average of this difference is computed as the bias. The standard deviation of the differences is computed as the precision. The square root of the sum of the squares of bias and precision is computed as the Arms value. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** 1-5 hours **Title:** RRp Arms of Sensor Accuracy **Type:** PRIMARY **Unit of Measure:** respiration per minute (RPM) ##### Group **Description:** All subjects will be enrolled into the test group and will receive the INVSENSOR00032 and/or INVSENSOR00033 device. INVSENSOR00032 and INVSENSOR00033: Investigational pulse oximeter device **ID:** OG000 **Title:** INVSENSOR00032 and INVSENSOR00033 Test Group ### Participant Flow Module #### Group **Description:** All subjects will be enrolled into the test group and will receive the INVSENSOR00032 and/or INVSENSOR00033 device. INVSENSOR00032 and INVSENSOR00033: Investigational pulse oximeter device **ID:** FG000 **Title:** INVSENSOR00032 and INVSENSOR00033 Test Group #### Period **Title:** Overall Study ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 27 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 26 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 1 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT00445679 **Brief Title:** Paroxetine-referenced Study Evaluating Three Doses of DVS SR in Outpatients With MDD **Official Title:** A Multicenter, Randomized, Double-blind, Paroxetine-referenced, Parallel-group Study to Evaluate the Safety, Efficacy, and Tolerability of 3 Fixed Doses (50mg, 100mg, AND 200mg) of Desvenlafaxine Succinate Sustained-release Tablets in Adult Outpatients With Major Depressive Disorder #### Organization Study ID Info **ID:** 3151A1-336 #### Organization **Class:** INDUSTRY **Full Name:** Wyeth is now a wholly owned subsidiary of Pfizer ### Status Module #### Completion Date **Date:** 2009-02 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2013-11-03 **Type:** ESTIMATED **Last Update Submit Date:** 2013-10-08 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2009-02 **Type:** ACTUAL #### Results First Post Date **Date:** 2013-11-03 **Type:** ESTIMATED **Results First Submit Date:** 2010-02-26 **Results First Submit QC Date:** 2013-10-08 #### Start Date **Date:** 2007-07 **Status Verified Date:** 2013-10 #### Study First Post Date **Date:** 2007-03-09 **Type:** ESTIMATED **Study First Submit Date:** 2007-03-06 **Study First Submit QC Date:** 2007-03-08 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Wyeth is now a wholly owned subsidiary of Pfizer #### Responsible Party **Type:** SPONSOR ### Description Module **Brief Summary:** This study will assess the safety, tolerability and efficacy of desvenlafaxine succinate sustained release (DVS SR) in subjects with major depressive disorder. **Detailed Description:** The primary objective of this study is to investigate the efficacy, safety and tolerability of desvenlafaxine succinate sustained release (DVS SR) in Chinese, Taiwanese, South Korean, and Indian subjects with major depressive disorder (MDD) receiving daily doses of 50 mg, 100 mg, or 200 mg. The secondary objective is to obtain additional information regarding the efficacy of DVS SR in subjects with MDD receiving daily doses of 50 mg, 100 mg, or 200 mg. Additional objectives include obtaining general and functional quality of life outcome data. ### Conditions Module **Conditions:** - Depressive Disorder, Major **Keywords:** - major depressive disorder - MDD - depression ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** TRIPLE **Who Masked:** - PARTICIPANT - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 807 **Type:** ACTUAL **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** DVS SR 50mg/day **Intervention Names:** - Drug: DVS SR **Label:** A **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** DVS SR 100mg/day **Intervention Names:** - Drug: DVS SR **Label:** B **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** DVS SR 200mg/day **Intervention Names:** - Drug: DVS SR **Label:** C **Type:** EXPERIMENTAL #### Arm Group 4 **Description:** Paroxetine 20mg/day **Intervention Names:** - Drug: Paroxetine **Label:** D **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - A - B - C **Description:** Arm 1: 50mg DVS SR tablet, QD, 8 weeks treatment with 2 week taper Arm 2: 100mg DVS SR tablet, QD, 8 weeks treatment with 2 week taper Arm 3: 200mg DVS SR tablet, QD, 8 weeks treatment with 2 week taper **Name:** DVS SR **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - D **Description:** 20 mg Paroxetine capsule, QD, 8 weeks treatment with 2 week taper **Name:** Paroxetine **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** HAM-D17 is a standardized, clinician-administered rating scale that assesses 17 items characteristically associated with major depression. Items are scored on either a 3 point (0 to 2) or a 5 point scale (0 to 4), with 0=none/absent and 4=most severe, for a maximum total score of 50. **Measure:** Percentage of Responders With a 50% or Greater Decrease From Baseline on the Hamilton Rating Scale for Depression, 17-item (HAM-D17) **Time Frame:** 8 weeks #### Secondary Outcomes **Description:** CGI-I is a global rating scale that measures disease improvement. Using a 7-point scale, the clinician rates how much the subject's illness has improved or worsened relative to the baseline status (1= very much improved; 7= very much worse). **Measure:** Clinical Global Impressions Scale-Improvement (CGI-I) Scores **Time Frame:** 8 weeks **Description:** CGI-S is a global rating scale that measures the severity of a subject's disease. Using a 7-point scale, the clinician rates the severity of the patient's mental illness at the time of the assessment, relative to the clinician's experience with subjects who have the same diagnosis (1= normal, not at all ill; 7= among the most extremely ill). **Measure:** Clinical Global Impressions Scale-Severity of Illness (CGI-S) Scores **Time Frame:** 8 weeks **Description:** Measures the overall severity of depressive symptoms. The MADRS has a 10-item checklist. Items are rated on a scale of 0-6, for a total score range of 0 (low severity of depressive symptoms) to 60 (high severity of depressive symptoms). **Measure:** Montgomery and Asberg Depression Rating Scale (MADRS) Total Score Mean Change From Baseline **Time Frame:** Baseline and 8 weeks **Description:** The VAS-PI is a self-rated visual analog scale for the assessment of pain. Scores on the VAS-PI range from 0 (no pain) to 10 (worst possible pain). A decrease in VAS-PI overall scores indicates a subject's assessment of an improvement in pain. **Measure:** Visual Analog Scale-pain Intensity (VAS-PI) Score Mean Change From Baseline **Time Frame:** 8 weeks **Description:** HAM-D6: standardized, clinician-administered rating scale is a subset of the HAM-D17 that assesses 6 items associated with major depression. The scale uses HAM-D17 items 1, 2, 7, 8, 10 and 13. Item 13 is scored 0 to 2 (0=none/absent to 2=most severe) and all others are scored 0 to 4 (0=none/absent to 4=most severe). Total score ranges from 0 to 22; higher score indicates more depression. **Measure:** Hamilton Rating Scale for Depression, 6-item (HAM-D6) Score Mean Change From Baseline **Time Frame:** 8 weeks **Description:** Covi anxiety scale measures the severity of anxiety symptoms on 3 items: verbal report, behavior and somatic complaints. Each dimension is assessed using a 5-point scale: 1 = not at all, 2 = somewhat, 3 = moderately, 4 = considerably, 5 = Very much. Total score ranges from 3 to 15; higher score indicates more anxiety. **Measure:** Covi Anxiety Scale Score Mean Change From Baseline **Time Frame:** 8 weeks ### Eligibility Module **Eligibility Criteria:** Primary Inclusion Criteria: 1. Outpatient men and women at least 18 years of age. 2. Have a primary diagnosis of MDD based on the criteria in the Diagnostic and Statistical Manual of Mental Disorders, 4th edition (DSM IV), single or recurrent episode, without psychotic features. 3. Have a HAM D17 total score ≥20 at the screening and baseline (study day 1) visit. Primary Exclusion Criteria: 1. Treatment with DVS SR at any time in the past. 2. Significant risk of suicide based on clinical judgment, including common suicidal thoughts and suicide having been considered as a possible solution even without specific plans or intent. 3. Any unstable hepatic, renal, pulmonary, cardiovascular (including uncontrolled hypertension), ophthalmologic, neurologic, or any other medical condition that might confound the study or put the subject at greater risk during study participation. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Beijing **Country:** China **Zip:** 100083 **Location 2:** **City:** Beijing **Country:** China **Zip:** 100088 **Location 3:** **City:** Guangdong Province **Country:** China **Zip:** 510370 **Location 4:** **City:** Hunan Province **Country:** China **Zip:** 410011 **Location 5:** **City:** Jiangsu Province **Country:** China **Zip:** 210029 **Location 6:** **City:** Shanghai **Country:** China **Zip:** 200030 **Location 7:** **City:** Shanghai **Country:** China **Zip:** 200065 **Location 8:** **City:** Shanxi Province **Country:** China **Zip:** 710032 **Location 9:** **City:** Sichuan Province **Country:** China **Zip:** 610041 **Location 10:** **City:** Yunnan Province **Country:** China **Zip:** 650032 **Location 11:** **City:** Zhejiang Province **Country:** China **Zip:** 310003 **Location 12:** **City:** Andhra Pradesh **Country:** India **Zip:** 500 038 **Location 13:** **City:** Andhra Pradesh **Country:** India **Zip:** 500034 **Location 14:** **City:** Andhra Pradesh **Country:** India **Zip:** 517 507 **Location 15:** **City:** Andhra Pradesh **Country:** India **Zip:** 530 002 **Location 16:** **City:** Chandigarh **Country:** India **Zip:** 160012 **Location 17:** **City:** Gujarat **Country:** India **Zip:** 380006 **Location 18:** **City:** Gujarat **Country:** India **Zip:** 380013 **Location 19:** **City:** Karnataka **Country:** India **Zip:** 575 001 **Location 20:** **City:** Karnataka **Country:** India **Zip:** 575018 **Location 21:** **City:** Maharashtra **Country:** India **Zip:** 400 012 **Location 22:** **City:** Maharashtra **Country:** India **Zip:** 400 026 **Location 23:** **City:** Maharashtra **Country:** India **Zip:** 411001 **Location 24:** **City:** Mumbai Maharashtra **Country:** India **Zip:** H19400 022 **Location 25:** **City:** New Delhi **Country:** India **Zip:** 110002 **Location 26:** **City:** Punjab **Country:** India **Zip:** 141001 **Location 27:** **City:** Uttar Pradesh **Country:** India **Zip:** 226003 **Location 28:** **City:** Incheon **Country:** Korea, Republic of **Zip:** 400-711 **Location 29:** **City:** Seoul **Country:** Korea, Republic of **Zip:** 110-744 **Location 30:** **City:** Seoul **Country:** Korea, Republic of **Zip:** 135-710 **Location 31:** **City:** Seoul **Country:** Korea, Republic of **Zip:** 135-720 **Location 32:** **City:** Seoul **Country:** Korea, Republic of **Zip:** 137-701 **Location 33:** **City:** Seoul **Country:** Korea, Republic of **Zip:** 138-736 **Location 34:** **City:** Seoul **Country:** Korea, Republic of **Zip:** 140-757 **Location 35:** **City:** Seoul **Country:** Korea, Republic of **Zip:** 150-719 **Location 36:** **City:** Seoul **Country:** Korea, Republic of **Zip:** 158-710 **Location 37:** **City:** Chang-Hua **Country:** Taiwan **Zip:** ROC 500 **Location 38:** **City:** KaoHsiung **Country:** Taiwan **Zip:** ROC 80708 **Location 39:** **City:** Taipei **Country:** Taiwan **Zip:** ROC 100 **Location 40:** **City:** Taipei **Country:** Taiwan **Zip:** ROC 111 **Location 41:** **City:** Taipei **Country:** Taiwan **Zip:** ROC 112 **Location 42:** **City:** Taipei **Country:** Taiwan **Zip:** ROC 114 **Location 43:** **City:** Taipei **Country:** Taiwan **Zip:** ROC 220 #### Overall Officials **Official 1:** **Affiliation:** Wyeth is now a wholly owned subsidiary of Pfizer **Name:** Medical Monitor **Role:** STUDY_DIRECTOR **Official 2:** **Affiliation:** For China: [email protected] **Name:** Trial Manager **Role:** PRINCIPAL_INVESTIGATOR ## 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: Depressive Disorder, Major - 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: D000017367 - Term: Selective Serotonin 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: D000018490 - Term: Serotonin Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018687 - Term: Antidepressive Agents, Second-Generation - ID: D000000928 - Term: Antidepressive Agents - ID: D000011619 - Term: Psychotropic Drugs - ID: D000065690 - Term: Cytochrome P-450 CYP2D6 Inhibitors - ID: D000065607 - Term: Cytochrome P-450 Enzyme Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors ### Intervention Browse Module - Browse Branches - Abbrev: PsychDr - Name: Psychotropic Drugs - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M19654 - Name: Paroxetine - Relevance: HIGH - As Found: Interleukin- - ID: M437 - Name: Desvenlafaxine Succinate - 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 - ID: M20504 - Name: Neurotransmitter Agents - 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 - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown - ID: M30537 - Name: Cytochrome P-450 Enzyme Inhibitors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000017374 - Term: Paroxetine ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** DVS SR 50 **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 50 mg/day **ID:** EG000 **Other Num Affected:** 128 **Other Num at Risk:** 203 **Serious Number Affected:** 4 **Serious Number At Risk:** 203 **Title:** DVS SR 50 **Group ID:** EG001 **Title:** DVS SR 100 **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 100 mg/day **ID:** EG001 **Other Num Affected:** 128 **Other Num at Risk:** 203 **Serious Number Affected:** 1 **Serious Number At Risk:** 203 **Title:** DVS SR 100 **Group ID:** EG002 **Title:** DVS SR 200 **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 200 mg/day **ID:** EG002 **Other Num Affected:** 140 **Other Num at Risk:** 205 **Serious Number Affected:** 5 **Serious Number At Risk:** 205 **Title:** DVS SR 200 **Group ID:** EG003 **Title:** Paroxetine 20 **Description:** Paroxetine 20 mg/day **ID:** EG003 **Other Num Affected:** 134 **Other Num at Risk:** 196 **Serious Number Affected:** 2 **Serious Number At Risk:** 196 **Title:** Paroxetine 20 **Frequency Threshold:** 5 #### Other Events **Term:** Palpitations **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** **Term:** Constipation **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Dry mouth **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Nausea **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Asthenia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** **Term:** Nasopharyngitis **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** **Term:** Anorexia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** **Term:** Dizziness **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** **Term:** Headache **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** **Term:** Somnolence **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** **Term:** Insomnia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** **Term:** Dyspepsia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Sedation **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** #### Serious Events **Term:** Cholelithiasis **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 203 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 203 **Group ID:** EG002 **Num At Risk:** 205 **Group ID:** EG003 **Num At Risk:** 196 **Term:** Pituitary tumor benign **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 203 **Group ID:** EG001 **Num At Risk:** 203 **Group ID:** EG002 **Num At Risk:** 205 **Group ID:** EG003 **Num At Risk:** 196 **Term:** Intentional overdose **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications ##### Stats **Group ID:** EG000 **Num At Risk:** 203 **Group ID:** EG001 **Num At Risk:** 203 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 205 **Group ID:** EG003 **Num At Risk:** 196 **Term:** Suicide attempt **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 203 **Group ID:** EG001 **Num At Risk:** 203 **Group ID:** EG002 **Num Affected:** 2 **Num At Risk:** 205 **Group ID:** EG003 **Num Affected:** 1 **Num At Risk:** 196 **Term:** Mania **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 203 **Group ID:** EG001 **Num At Risk:** 203 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 205 **Group ID:** EG003 **Num At Risk:** 196 **Term:** Suicidal behavior **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 203 **Group ID:** EG001 **Num At Risk:** 203 **Group ID:** EG002 **Num Affected:** 2 **Num At Risk:** 205 **Group ID:** EG003 **Num At Risk:** 196 **Term:** Suicidal ideation **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 203 **Group ID:** EG001 **Num At Risk:** 203 **Group ID:** EG002 **Num At Risk:** 205 **Group ID:** EG003 **Num Affected:** 2 **Num At Risk:** 196 **Term:** Drug abuse **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 203 **Group ID:** EG001 **Num At Risk:** 203 **Group ID:** EG002 **Num At Risk:** 205 **Group ID:** EG003 **Num Affected:** 1 **Num At Risk:** 196 **Term:** Major Depressive Disorder worsening **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 203 **Group ID:** EG001 **Num At Risk:** 203 **Group ID:** EG002 **Num At Risk:** 205 **Group ID:** EG003 **Num Affected:** 1 **Num At Risk:** 196 ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 203 **Group ID:** BG001 **Value:** 203 **Group ID:** BG002 **Value:** 205 **Group ID:** BG003 **Value:** 196 **Group ID:** BG004 **Value:** 807 **Units:** Participants ### Group **ID:** BG000 **Title:** DVS SR 50 **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 50 mg/day ### Group **ID:** BG001 **Title:** DVS SR 100 **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 100 mg/day ### Group **ID:** BG002 **Title:** DVS SR 200 **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 200 mg/day ### Group **ID:** BG003 **Title:** Paroxetine 20 **Description:** Paroxetine 20 mg/day ### Group **ID:** BG004 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 12.96 **Value:** 38.63 #### Measurement **Group ID:** BG001 **Spread:** 13.22 **Value:** 39.26 #### Measurement **Group ID:** BG002 **Spread:** 14.69 **Value:** 39.26 #### Measurement **Group ID:** BG003 **Spread:** 13.22 **Value:** 38.27 #### Measurement **Group ID:** BG004 **Spread:** 13.53 **Value:** 38.86 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 125 #### Measurement **Group ID:** BG001 **Value:** 129 #### Measurement **Group ID:** BG002 **Value:** 118 #### Measurement **Group ID:** BG003 **Value:** 117 #### Measurement **Group ID:** BG004 **Value:** 489 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 78 #### Measurement **Group ID:** BG001 **Value:** 74 #### Measurement **Group ID:** BG002 **Value:** 87 #### Measurement **Group ID:** BG003 **Value:** 79 #### Measurement **Group ID:** BG004 **Value:** 318 **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:** The PIs agreed to allow the sponsor 60 days to review and require changes to presentations or publications but only to protect confidential information and intellectual property, and for the sponsor to file a patent application, as applicable. The PIs also agreed for data to be presented first as a joint, multi-center publication. **Restriction Type:** OTHER **Restrictive Agreement:** True ### Point of Contact **Email:** [email protected] **Organization:** Wyeth **Title:** U. S. Contact Center ## Results Section - Outcome Measures Module ### Outcome Measure 1 #### Analysis **CI Lower Limit:** -12.62 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 6.37 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** Noninferiority to paroxetine was declared if the lower limit of the 95% two-sided confidence interval for the difference in responders is greater than or equal to -9 percentage points. **Non-Inferiority Type:** NON_INFERIORITY_OR_EQUIVALENCE **Other Analysis Description:** **P-Value:** **P-Value Comment:** **Parameter Type:** Difference **Parameter Value:** -3.13 **Statistical Comment:** **Statistical Method:** **Tested Non-Inferiority:** True #### Analysis **CI Lower Limit:** -8.50 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 10.25 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** Noninferiority to paroxetine was declared if the lower limit of the 95% two-sided confidence interval for the difference in responders is greater than or equal to -9 percentage points. **Non-Inferiority Type:** NON_INFERIORITY_OR_EQUIVALENCE **Other Analysis Description:** **P-Value:** **P-Value Comment:** **Parameter Type:** Difference **Parameter Value:** 0.88 **Statistical Comment:** **Statistical Method:** **Tested Non-Inferiority:** True #### Analysis **CI Lower Limit:** -11.68 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 7.33 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** **Non-Inferiority Comment:** Noninferiority to paroxetine was declared if the lower limit of the 95% two-sided confidence interval for the difference in responders is greater than or equal to -9 percentage points. **Non-Inferiority Type:** NON_INFERIORITY_OR_EQUIVALENCE **Other Analysis Description:** **P-Value:** **P-Value Comment:** **Parameter Type:** Difference **Parameter Value:** -2.17 **Statistical Comment:** **Statistical Method:** **Tested Non-Inferiority:** True ### Outcome Measure 2 #### Analysis **CI Lower Limit:** **CI Number of Sides:** **CI Percentage Value:** 95 **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Final on-therapy population **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.714 **P-Value Comment:** **Parameter Type:** **Parameter Value:** **Statistical Comment:** **Statistical Method:** Cochran-Mantel-Haenszel **Tested Non-Inferiority:** False #### Analysis **CI Lower Limit:** **CI Number of Sides:** **CI Percentage Value:** 95 **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Final on-therapy population **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.653 **P-Value Comment:** **Parameter Type:** **Parameter Value:** **Statistical Comment:** **Statistical Method:** Cochran-Mantel-Haenszel **Tested Non-Inferiority:** False #### Analysis **CI Lower Limit:** **CI Number of Sides:** **CI Percentage Value:** 95 **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Final on-therapy population **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.881 **P-Value Comment:** **Parameter Type:** **Parameter Value:** **Statistical Comment:** **Statistical Method:** Cochran-Mantel-Haenszel **Tested Non-Inferiority:** False ### Outcome Measure 3 #### Analysis **CI Lower Limit:** **CI Number of Sides:** **CI Percentage Value:** 95 **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Final on-therapy population **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.450 **P-Value Comment:** **Parameter Type:** **Parameter Value:** **Statistical Comment:** **Statistical Method:** Cochran-Mantel-Haenszel **Tested Non-Inferiority:** False #### Analysis **CI Lower Limit:** **CI Number of Sides:** **CI Percentage Value:** 95 **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Final on-therapy population **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.452 **P-Value Comment:** **Parameter Type:** **Parameter Value:** **Statistical Comment:** **Statistical Method:** Cochran-Mantel-Haenszel **Tested Non-Inferiority:** False #### Analysis **CI Lower Limit:** **CI Number of Sides:** **CI Percentage Value:** 95 **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Final on-therapy population **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** 0.850 **P-Value Comment:** **Parameter Type:** **Parameter Value:** **Statistical Comment:** **Statistical Method:** Cochran-Mantel-Haenszel **Tested Non-Inferiority:** False ### Outcome Measure 4 ### Outcome Measure 5 ### Outcome Measure 6 ### Outcome Measure 7 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Anticipated Posting Date:** 2010-02 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 63 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 67 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 63 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 66 **Title:** #### Outcome Measure 2 **Anticipated Posting Date:** 2010-02 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 72 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 78 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 79 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 72 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 64 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 64 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 53 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 59 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 36 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 34 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 28 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 37 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 16 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 17 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 26 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 17 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 8 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 6 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 10 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 **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 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** #### Outcome Measure 3 **Anticipated Posting Date:** 2010-02 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 40 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 50 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 53 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 42 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 49 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 53 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 47 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 49 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 53 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 46 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 34 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 51 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 29 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 30 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 38 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 27 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 24 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 18 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 19 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 15 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 6 - **Comment:** - **Group ID:** OG003 - **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:** 1 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** #### Outcome Measure 4 **Anticipated Posting Date:** 2010-02 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** -18.37 - **Spread:** - **Upper Limit:** -15.31 - **Value:** -16.84 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** -19.37 - **Spread:** - **Upper Limit:** -16.31 - **Value:** -17.84 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** -18.43 - **Spread:** - **Upper Limit:** -15.20 - **Value:** -16.81 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** -18.08 - **Spread:** - **Upper Limit:** -15.00 - **Value:** -16.54 **Title:** #### Outcome Measure 5 **Anticipated Posting Date:** 2010-02 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** -15.10 - **Spread:** - **Upper Limit:** -8.27 - **Value:** -11.69 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** -13.75 - **Spread:** - **Upper Limit:** -6.26 - **Value:** -10.00 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** -12.75 - **Spread:** - **Upper Limit:** -5.45 - **Value:** -9.10 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** -12.40 - **Spread:** - **Upper Limit:** -6.63 - **Value:** -9.52 **Title:** #### Outcome Measure 6 **Anticipated Posting Date:** 2010-02 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** -7.08 - **Spread:** - **Upper Limit:** -5.92 - **Value:** -6.50 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** -7.06 - **Spread:** - **Upper Limit:** -5.95 - **Value:** -6.51 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** -7.03 - **Spread:** - **Upper Limit:** -5.82 - **Value:** -6.42 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** -7.15 - **Spread:** - **Upper Limit:** -6.01 - **Value:** -6.58 **Title:** #### Outcome Measure 7 **Anticipated Posting Date:** 2010-02 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** -1.47 - **Spread:** - **Upper Limit:** -0.99 - **Value:** -1.23 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** -1.54 - **Spread:** - **Upper Limit:** -1.03 - **Value:** -1.28 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** -1.44 - **Spread:** - **Upper Limit:** -0.89 - **Value:** -1.17 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** -1.49 - **Spread:** - **Upper Limit:** -1.00 - **Value:** -1.24 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** HAM-D17 is a standardized, clinician-administered rating scale that assesses 17 items characteristically associated with major depression. Items are scored on either a 3 point (0 to 2) or a 5 point scale (0 to 4), with 0=none/absent and 4=most severe, for a maximum total score of 50. **Parameter Type:** NUMBER **Population Description:** The intent-to-treat (ITT) population included all subjects randomly assigned to treatment who had a baseline HAM-D17 evaluation, took at least 1 dose of double-blind test article, and had at least 1 postbaseline HAM-D17 evaluation. **Reporting Status:** POSTED **Time Frame:** 8 weeks **Title:** Percentage of Responders With a 50% or Greater Decrease From Baseline on the Hamilton Rating Scale for Depression, 17-item (HAM-D17) **Type:** PRIMARY **Unit of Measure:** percentage of responders ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 50 mg/day **ID:** OG000 **Title:** DVS SR 50 ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 100 mg/day **ID:** OG001 **Title:** DVS SR 100 ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 200 mg/day **ID:** OG002 **Title:** DVS SR 200 ##### Group **Description:** Paroxetine 20 mg/day **ID:** OG003 **Title:** Paroxetine 20 #### Outcome Measure 2 **Description:** CGI-I is a global rating scale that measures disease improvement. Using a 7-point scale, the clinician rates how much the subject's illness has improved or worsened relative to the baseline status (1= very much improved; 7= very much worse). **Parameter Type:** NUMBER **Population Description:** The intent-to-treat (ITT) population included all subjects randomly assigned to treatment who had a baseline HAM-D17 evaluation, took at least 1 dose of double-blind test article, and had at least 1 postbaseline HAM-D17 evaluation. Number of participants analyzed reflects the final on-therapy population. **Reporting Status:** POSTED **Time Frame:** 8 weeks **Title:** Clinical Global Impressions Scale-Improvement (CGI-I) Scores **Type:** SECONDARY **Unit of Measure:** subjects ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 50 mg/day **ID:** OG000 **Title:** DVS SR 50 ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 100 mg/day **ID:** OG001 **Title:** DVS SR 100 ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 200 mg/day **ID:** OG002 **Title:** DVS SR 200 ##### Group **Description:** Paroxetine 20 mg/day **ID:** OG003 **Title:** Paroxetine 20 #### Outcome Measure 3 **Description:** CGI-S is a global rating scale that measures the severity of a subject's disease. Using a 7-point scale, the clinician rates the severity of the patient's mental illness at the time of the assessment, relative to the clinician's experience with subjects who have the same diagnosis (1= normal, not at all ill; 7= among the most extremely ill). **Parameter Type:** NUMBER **Population Description:** The intent-to-treat (ITT) population included all subjects randomly assigned to treatment who had a baseline HAM-D17 evaluation, took at least 1 dose of double-blind test article, and had at least 1 postbaseline HAM-D17 evaluation. Number of participants analyzed reflects the final on-therapy population. **Reporting Status:** POSTED **Time Frame:** 8 weeks **Title:** Clinical Global Impressions Scale-Severity of Illness (CGI-S) Scores **Type:** SECONDARY **Unit of Measure:** subjects ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 50 mg/day **ID:** OG000 **Title:** DVS SR 50 ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 100 mg/day **ID:** OG001 **Title:** DVS SR 100 ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 200 mg/day **ID:** OG002 **Title:** DVS SR 200 ##### Group **Description:** Paroxetine 20 mg/day **ID:** OG003 **Title:** Paroxetine 20 #### Outcome Measure 4 **Description:** Measures the overall severity of depressive symptoms. The MADRS has a 10-item checklist. Items are rated on a scale of 0-6, for a total score range of 0 (low severity of depressive symptoms) to 60 (high severity of depressive symptoms). **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** The intent-to-treat (ITT) population included all subjects randomly assigned to treatment who had a baseline HAM-D17 evaluation, took at least 1 dose of double-blind test article, and had at least 1 postbaseline HAM-D17 evaluation. Number of participants analyzed reflects the final on-therapy population. **Reporting Status:** POSTED **Time Frame:** Baseline and 8 weeks **Title:** Montgomery and Asberg Depression Rating Scale (MADRS) Total Score Mean Change From Baseline **Type:** SECONDARY **Unit of Measure:** units on a scale ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 50 mg/day **ID:** OG000 **Title:** DVS SR 50 ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 100 mg/day **ID:** OG001 **Title:** DVS SR 100 ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 200 mg/day **ID:** OG002 **Title:** DVS SR 200 ##### Group **Description:** Paroxetine 20 mg/day **ID:** OG003 **Title:** Paroxetine 20 #### Outcome Measure 5 **Description:** The VAS-PI is a self-rated visual analog scale for the assessment of pain. Scores on the VAS-PI range from 0 (no pain) to 10 (worst possible pain). A decrease in VAS-PI overall scores indicates a subject's assessment of an improvement in pain. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** The intent-to-treat (ITT) population included all subjects randomly assigned to treatment who had a baseline HAM-D17 evaluation, took at least 1 dose of double-blind test article, and had at least 1 postbaseline HAM-D17 evaluation. Number of participants analyzed reflects the final on-therapy population. **Reporting Status:** POSTED **Time Frame:** 8 weeks **Title:** Visual Analog Scale-pain Intensity (VAS-PI) Score Mean Change From Baseline **Type:** SECONDARY **Unit of Measure:** scores on a scale ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 50 mg/day **ID:** OG000 **Title:** DVS SR 50 ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 100 mg/day **ID:** OG001 **Title:** DVS SR 100 ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 200 mg/day **ID:** OG002 **Title:** DVS SR 200 ##### Group **Description:** Paroxetine 20 mg/day **ID:** OG003 **Title:** Paroxetine 20 #### Outcome Measure 6 **Description:** HAM-D6: standardized, clinician-administered rating scale is a subset of the HAM-D17 that assesses 6 items associated with major depression. The scale uses HAM-D17 items 1, 2, 7, 8, 10 and 13. Item 13 is scored 0 to 2 (0=none/absent to 2=most severe) and all others are scored 0 to 4 (0=none/absent to 4=most severe). Total score ranges from 0 to 22; higher score indicates more depression. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** The intent-to-treat (ITT) population included all subjects randomly assigned to treatment who had a baseline HAM-D17 evaluation, took at least 1 dose of double-blind test article, and had at least 1 postbaseline HAM-D17 evaluation. Number of participants analyzed reflects the final on-therapy population. **Reporting Status:** POSTED **Time Frame:** 8 weeks **Title:** Hamilton Rating Scale for Depression, 6-item (HAM-D6) Score Mean Change From Baseline **Type:** SECONDARY **Unit of Measure:** scores on a scale ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 50 mg/day **ID:** OG000 **Title:** DVS SR 50 ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 100 mg/day **ID:** OG001 **Title:** DVS SR 100 ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 200 mg/day **ID:** OG002 **Title:** DVS SR 200 ##### Group **Description:** Paroxetine 20 mg/day **ID:** OG003 **Title:** Paroxetine 20 #### Outcome Measure 7 **Description:** Covi anxiety scale measures the severity of anxiety symptoms on 3 items: verbal report, behavior and somatic complaints. Each dimension is assessed using a 5-point scale: 1 = not at all, 2 = somewhat, 3 = moderately, 4 = considerably, 5 = Very much. Total score ranges from 3 to 15; higher score indicates more anxiety. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** The intent-to-treat (ITT) population included all subjects randomly assigned to treatment who had a baseline HAM-D17 evaluation, took at least 1 dose of double-blind test article, and had at least 1 postbaseline HAM-D17 evaluation. Number of participants analyzed reflects the final on-therapy population. **Reporting Status:** POSTED **Time Frame:** 8 weeks **Title:** Covi Anxiety Scale Score Mean Change From Baseline **Type:** SECONDARY **Unit of Measure:** scores on a scale ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 50 mg/day **ID:** OG000 **Title:** DVS SR 50 ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 100 mg/day **ID:** OG001 **Title:** DVS SR 100 ##### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 200 mg/day **ID:** OG002 **Title:** DVS SR 200 ##### Group **Description:** Paroxetine 20 mg/day **ID:** OG003 **Title:** Paroxetine 20 ### Participant Flow Module #### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 50 mg/day **ID:** FG000 **Title:** DVS SR 50 #### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 100 mg/day **ID:** FG001 **Title:** DVS SR 100 #### Group **Description:** Desvenlafaxine Succinate Sustained-Release (DVS SR) 200 mg/day **ID:** FG002 **Title:** DVS SR 200 #### Group **Description:** Paroxetine 20 mg/day **ID:** FG003 **Title:** Paroxetine 20 #### Period **Title:** Overall Study ##### Withdraw **Type:** Adverse Event ###### Reason **Group ID:** FG000 **Number of Subjects:** 22 ###### Reason **Group ID:** FG001 **Number of Subjects:** 12 ###### Reason **Group ID:** FG002 **Number of Subjects:** 30 ###### Reason **Group ID:** FG003 **Number of Subjects:** 19 ##### Withdraw **Type:** Failed to return ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 3 ###### Reason **Group ID:** FG002 **Number of Subjects:** 2 ###### Reason **Group ID:** FG003 **Number of Subjects:** 2 ##### Withdraw **Type:** Physician Decision ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ###### Reason **Group ID:** FG002 **Number of Subjects:** 1 ###### Reason **Group ID:** FG003 **Number of Subjects:** 0 ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 10 ###### Reason **Group ID:** FG001 **Number of Subjects:** 6 ###### Reason **Group ID:** FG002 **Number of Subjects:** 6 ###### Reason **Group ID:** FG003 **Number of Subjects:** 5 ##### Withdraw **Type:** Compliance ###### Reason **Group ID:** FG000 **Number of Subjects:** 3 ###### Reason **Group ID:** FG001 **Number of Subjects:** 2 ###### Reason **Group ID:** FG002 **Number of Subjects:** 1 ###### Reason **Group ID:** FG003 **Number of Subjects:** 2 ##### Withdraw **Type:** Withdrawal of Informed Consent ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ###### Reason **Group ID:** FG002 **Number of Subjects:** 1 ###### Reason **Group ID:** FG003 **Number of Subjects:** 0 ##### Withdraw **Type:** Protocol Violation ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ###### Reason **Group ID:** FG001 **Number of Subjects:** 3 ###### Reason **Group ID:** FG002 **Number of Subjects:** 3 ###### Reason **Group ID:** FG003 **Number of Subjects:** 1 ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 8 ###### Reason **Group ID:** FG001 **Number of Subjects:** 14 ###### Reason **Group ID:** FG002 **Number of Subjects:** 20 ###### Reason **Group ID:** FG003 **Number of Subjects:** 18 ##### Withdraw **Type:** Lack of Efficacy ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ###### Reason **Group ID:** FG001 **Number of Subjects:** 6 ###### Reason **Group ID:** FG002 **Number of Subjects:** 2 ###### Reason **Group ID:** FG003 **Number of Subjects:** 0 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 203 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 203 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 205 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 196 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 155 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 157 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 139 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 149 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 48 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 46 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 66 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 47 **Pre-Assignment Details:** After a 4 to 21 day screening period, eligible subjects were treated for up to 8 weeks. **Recruitment Details:** Subjects were recruited in China, India, South Korea and Taiwan from July 2007 to December 2008. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT00972179 **Brief Title:** Safety Study of Tezepelumab (AMG 157) in Healthy Adults **Official Title:** A Randomized, Double-Blind, Placebo-Controlled, Ascending Multiple Dose Study to Evaluate the Safety, Tolerability and Pharmacokinetics of AMG 157 in Healthy Subjects #### Organization Study ID Info **ID:** 20080390 #### Organization **Class:** INDUSTRY **Full Name:** Amgen ### Status Module #### Completion Date **Date:** 2011-01-09 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2022-09-21 **Type:** ACTUAL **Last Update Submit Date:** 2022-09-08 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2011-01-09 **Type:** ACTUAL #### Results First Post Date **Date:** 2022-05-23 **Type:** ACTUAL **Results First Submit Date:** 2022-03-09 **Results First Submit QC Date:** 2022-03-09 #### Start Date **Date:** 2009-09-15 **Type:** ACTUAL **Status Verified Date:** 2022-09 #### Study First Post Date **Date:** 2009-09-04 **Type:** ESTIMATED **Study First Submit Date:** 2009-09-03 **Study First Submit QC Date:** 2009-09-03 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Amgen #### Responsible Party **Type:** SPONSOR ### Description Module **Brief Summary:** The primary objective is to evaluate the safety, tolerability, and immunogenicity of multiple-dose administration of tezepelumab in healthy adults. **Detailed Description:** This study will follow a randomized, multiple-dose, double-blind, placebo-controlled, sequential dose-escalation study design. The study will consist of five subcutaneous (SC) cohorts and one intravenous (IV) cohort. Each dose cohort is planned to enroll 8 participants, randomized such that 6 participants will receive tezepelumab and 2 will receive placebo (3:1 ratio). ### Conditions Module **Conditions:** - Healthy Volunteers **Keywords:** - healthy volunteers ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** SEQUENTIAL **Intervention Model Description:** The cohorts will enroll sequentially: enrollment to the subsequent cohort (ie, next higher dose) will proceed only after the previous dose is determined to be safe and well tolerated by a blinded review of available safety data conducted on day 43 of the previous cohort. Within each dose cohort, participants will be randomized 3:1 to receive tezepelumab or placebo. ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - INVESTIGATOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 49 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Tezepelumab will be administered subcutaneously (SC) at doses from 35 mg once every 28 days (Q28D) (cohort 1) up to 210 mg once every 7 days (Q7D) (cohort 5) and an intravenous (IV) dose cohort of 700 mg Q28D (cohort 6). **Intervention Names:** - Drug: Tezepelumab **Label:** Tezepelumab **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Two participants in each cohort (cohorts 1 to 6) will receive matching placebo administered subcutaneously (cohorts 1-5) or intravenously (cohort 6), matching the treatment regiment of tezepelumab. **Intervention Names:** - Drug: Placebo **Label:** Placebo **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Placebo **Description:** Administered by subcutaneous or intravenous injection. **Name:** Placebo **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Tezepelumab **Description:** Administered by subcutaneous or intravenous injection **Name:** Tezepelumab **Other Names:** - AMG 157 - Tezspire **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Adverse events (AEs) include any untoward medical occurrence in a trial participant administered a study drug and does not necessarily have a causal relationship with this treatment. AEs include worsening of a pre-existing medical condition and laboratory value changes requiring therapy or adjustment in prior therapy. AEs were assessed for severity according to the Common Terminology Criteria for Adverse Events (CTCAE), Version 3, where Grade 1 = Mild AE, Grade 2 = Moderate AE, Grade 3 = severe AE, Grade 4 = life-threatening AE and Grade 5 = death due to AE. Relationship to study treatment was determined by the investigator. A serious adverse event (SAE) is defined as an AE that met 1 or more of below criteria: * was fatal; * was life threatening; * required in-patient hospitalization or prolongation of existing hospitalization; * resulted in persistent or significant disability/incapacity; * was a congenital anomaly/birth defect; * other significant medical hazard. **Measure:** Number of Participants With Treatment-emergent Adverse Events **Time Frame:** From first dose of study drug up to day 169 **Description:** All study samples (tezepelumab and placebo) were tested using an electrochemiluminescence (ECL) based immunoassay to detect and confirm the presence of antibodies capable of binding to tezepelumab. Samples identified as positive in the immunoassay were tested in a receptor-binding ECL-based assay to detect neutralizing or inhibitory effects toward tezepelumab. The number of participants with positive anti-tezepelumab binding antibodies / neutralizing antibodies at any time post-baseline with a negative or no result at baseline is reported. **Measure:** Number of Participants Who Developed Anti-tezepelumab Antibodies After Initiation of Treatment **Time Frame:** For Q28D groups: Days 28, 56, 85, 113, and 169; For Q14D and Q7D groups: Days 29, 57, 85, 113, 141, and 169 #### Secondary Outcomes **Description:** The pharmacokinetic (PK) parameter Tmax was estimated based on the serum concentrations of tezepelumab using noncompartmental methods. The concentration of tezepelumab in human serum was measured using a validated enzyme-linked immunosorbent assay (ELISA). The lower limit of quantification of the assay was 10 ng/ml. **Measure:** Time of Maximum Observed Concentration (Tmax) of Tezepelumab **Time Frame:** First dose: Day 1 predose, end of infusion (EOI; IV cohort only), 4 hours, 3, 7, 14 (Q14D & Q28D only) and 28 days (Q28D only) postdose. Last Dose: Day 57 (Q28D), Day 71 (Q14D) and Day 78 (Q7D) predose, EOI (IV cohort), 4 hours, 3, 7, 14, 28 days postdose **Description:** The PK parameter Cmax was estimated based on the serum concentrations of tezepelumab using noncompartmental methods. The concentration of tezepelumab in human serum was measured using a validated ELISA. The lower limit of quantification of the assay was 10 ng/ml. **Measure:** Maximum Observed Concentration (Cmax) of Tezepelumab **Time Frame:** First dose: Day 1 predose, end of infusion (EOI; IV cohort only), 4 hours, 3, 7, 14 (Q14D & Q28D only) and 28 days (Q28D only) postdose. Last Dose: Day 57 (Q28D), Day 71 (Q14D) and Day 78 (Q7D) predose, EOI (IV cohort), 4 hours, 3, 7, 14, 28 days postdose **Description:** The PK parameter AUCtau was estimated based on the serum concentrations of tezepelumab using noncompartmental methods. The dosing interval (tau) was 28 days, 14 days or 7 days depending on the treatment arm. The concentration of tezepelumab in human serum was measured using a validated ELISA. The lower limit of quantification of the assay was 10 ng/ml. **Measure:** Area Under the Concentration-time Curve Over the Dosing Interval (AUCtau) for Tezepelumab **Time Frame:** First dose: Day 1 predose, end of infusion (EOI; IV cohort only), 4 hours, 3, 7, 14 (Q14D & Q28D only) and 28 days (Q28D only) postdose. Last Dose: Day 57 (Q28D), Day 71 (Q14D) and Day 78 (Q7D) predose, EOI (IV cohort), 4 hours, 3, 7, 14, 28 days postdose **Description:** Accumulation ratio (AR) based on AUCtau was calculated as AUCtau after last dose / AUCtau after first dose, except for the Q7D cohort where AR was calculated as AUCtau after last dose / area under the concentration-time curve from time 0 to time of last measurable concentration (AUC0-t) after first dose. **Measure:** Accumulation Ratio Based on AUCtau **Time Frame:** First dose: Day 1 predose, end of infusion (EOI; IV cohort only), 4 hours, 3, 7, 14 (Q14D & Q28D only) and 28 days (Q28D only) postdose. Last Dose: Day 57 (Q28D), Day 71 (Q14D) and Day 78 (Q7D) predose, EOI (IV cohort), 4 hours, 3, 7, 14, 28 days postdose **Description:** Accumulation ratio based on Cmax calculated as Cmax after last dose / Cmax after first dose **Measure:** Accumulation Ratio Based on Cmax **Time Frame:** First dose: Day 1 predose, end of infusion (EOI; IV cohort only), 4 hours, 3, 7, 14 (Q14D & Q28D only) and 28 days (Q28D only) postdose. Last Dose: Day 57 (Q28D), Day 71 (Q14D) and Day 78 (Q7D) predose, EOI (IV cohort), 4 hours, 3, 7, 14, 28 days postdose ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Subjects must sign an Institutional Review Board (IRB) approved informed consent form before any study-specific procedures; * Healthy subject, aged between 18 and 45 years, inclusive; * Female subject must be of non-reproductive potential (ie, postmenopausal by history - no menses for ≥ 1 year and by follicle-stimulating hormone (FSH) \[using local reference ranges\]; OR history of hysterectomy; OR history of bilateral tubal ligation; OR history of bilateral oophorectomy); * Male subjects with female partner of childbearing potential who agrees to inform their female partner of their participation in this clinical study and use highly effective methods of birth control during the study. (Highly effective methods of birth control may include abstinence, vasectomy, or a condom with spermicide in combination with either hormonal birth control, intra-uterine device, or barrier methods used by the woman); * Male subject who agrees to use birth control for five months after last dose of study medication, male subject who agrees not to donate sperm during the study and for five months after last dose of study medication; * Healthy subject with a body mass index (BMI) between 18 and 32 kg/m\^2, inclusive at screening; * Subject must have normal or clinically acceptable physical examination and electrocardiogram (ECG) results prior to Day 1 based on the opinion of the investigator; * Subject must have normal or clinically acceptable clinical laboratory tests at screening as determined by Amgen and the investigator; * Subject must have adequate renal function (defined as creatinine clearance \> 80 mL/min using the Cockcroft Gault equation). Exclusion Criteria: * Subject who has history or evidence of a clinically significant disorder, condition or disease (including but not limited to cardiopulmonary, oncologic, immunologic, autoimmune, collagen vascular, renal, metabolic, hematologic or psychiatric), that, in the opinion of the Investigator in consultation with the Amgen physician, would pose a risk to subject safety or interfere with the study evaluation, procedures or completion; * Subject who has evidence of any active or suspected bacterial, viral, fungal or parasitic infections within the past 30 days prior to randomization (eg, common cold, viral syndrome, flu-like symptoms). Subject who, in the opinion of the investigator, has a high risk of parasitic disease is also excluded; * Subject who has known positive tuberculin skin test (if not treated with appropriate chemoprophylaxis) or recent (within six months from randomization) exposure to an individual with active tuberculosis; * Subject who has history of malignancy of any type, other than in situ cervical cancer or surgically excised non-melanomatous skin cancers within five years before randomization of the study; * Subject who has known type I/II diabetes; * Subject who uses nonprescription drugs within 14 days prior to randomization and for the entire duration of the study. All herbal supplements, vitamins, and nutritional supplements taken within the last 30 days prior to dosing on Day 1 (and continued use, if appropriate), must be reviewed and approved by the PI and Amgen Medical Monitor; * Subject who has used any systemic cytotoxic or systemic immunosuppressive medications (other than corticosteroids) within 6 months prior to randomization and for the entire duration of the study or has used any corticosteroid, topical cytotoxic or topical immunosuppressive medications within 30 days or five half-lives (whichever is longer) prior to randomization and for the entire duration of the study; * Subject who has previously received any other therapeutic monoclonal antibody; * Subject who has previously received any investigational drug (or is currently using an investigational device) within 30 days or five half-lives (whichever is longer) prior to randomization; * Subject who has tested positive for drugs and/or alcohol use at screening or before randomization, subject who has consumed alcohol within 48 hours prior to any study visit including screening, and subject with alcohol intake of \> 2 drinks/day on average during the study (one drink being equivalent to 12 ounces of regular beer, 8 to 9 ounces of malt liquor, 5 ounces of wine or 1.5 ounces of 80 proof distilled spirits); * Female subject who is pregnant or lactating; female subject who is of child-bearing potential; * Subject who has donated blood (including blood products) or experienced loss of blood ≥ 500 mL within two months of study screening; * Subject who is positive for human immunodeficiency virus (HIV) antibodies, hepatitis B surface antigen, or hepatitis C antibodies; * Subject who has regularly used nicotine or tobacco containing products (including but not limited to: snuff, chewing tobacco, cigars, cigarettes, pipes, or nicotine patches) during six months before randomization and during the study; * Subject who has any other condition that might reduce the chance of obtaining data (eg, known poor compliance) required by the protocol or that might compromise the ability to give truly informed consent. **Healthy Volunteers:** True **Maximum Age:** 45 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Overall Officials **Official 1:** **Affiliation:** Amgen **Name:** MD **Role:** STUDY_DIRECTOR ### References Module #### References **Citation:** Parnes JR, Sullivan JT, Chen L, Dias C. Pharmacokinetics, Safety, and Tolerability of Tezepelumab (AMG 157) in Healthy and Atopic Dermatitis Adult Subjects. Clin Pharmacol Ther. 2019 Aug;106(2):441-449. doi: 10.1002/cpt.1401. Epub 2019 Mar 23. **PMID:** 30779339 #### See Also Links **Label:** AmgenTrials clinical trials website **URL:** http://www.amgentrials.com ## Derived Section ### Misc Info Module #### Removed Countries - Country: United States - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Tezepelumab 35 mg Q28D **Description:** Participants received 35 mg tezepelumab by subcutaneous injection once every 28 days (Q28D) for 3 doses. **ID:** EG000 **Other Num Affected:** 4 **Other Num at Risk:** 6 **Serious Number Affected:** 1 **Serious Number At Risk:** 6 **Title:** Tezepelumab 35 mg Q28D **Group ID:** EG001 **Title:** Tezepelumab 105 mg Q28D **Description:** Participants received 105 mg tezepelumab by subcutaneous injection once every 28 days for 3 doses. **ID:** EG001 **Other Num Affected:** 4 **Other Num at Risk:** 6 **Serious Number At Risk:** 6 **Title:** Tezepelumab 105 mg Q28D **Group ID:** EG002 **Title:** Tezepelumab 210 mg Q28D **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 28 days for 3 doses. **ID:** EG002 **Other Num Affected:** 2 **Other Num at Risk:** 6 **Serious Number At Risk:** 6 **Title:** Tezepelumab 210 mg Q28D **Group ID:** EG003 **Title:** Tezepelumab 210 mg Q14D **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 14 days (Q14D) for 6 doses. **ID:** EG003 **Other Num Affected:** 6 **Other Num at Risk:** 6 **Serious Number At Risk:** 6 **Title:** Tezepelumab 210 mg Q14D **Group ID:** EG004 **Title:** Tezepelumab 210 mg Q7D **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 7 days (Q7D) for 12 doses. **ID:** EG004 **Other Num Affected:** 5 **Other Num at Risk:** 7 **Serious Number At Risk:** 7 **Title:** Tezepelumab 210 mg Q7D **Group ID:** EG005 **Title:** Tezepelumab 700 mg Q28D IV **Description:** Participants received 700 mg tezepelumab by intravenous injection once every 28 days for 3 doses. **ID:** EG005 **Other Num Affected:** 3 **Other Num at Risk:** 6 **Serious Number At Risk:** 6 **Title:** Tezepelumab 700 mg Q28D IV **Group ID:** EG006 **Title:** Tezepelumab Total **Description:** All participants who received tezepelumab. **ID:** EG006 **Other Num Affected:** 24 **Other Num at Risk:** 37 **Serious Number Affected:** 1 **Serious Number At Risk:** 37 **Title:** Tezepelumab Total **Group ID:** EG007 **Title:** Placebo **Description:** Participants received matching placebo administered subcutaneously (Cohorts 1-5) or intravenously (Cohort 6), matching the treatment regimen of tezepelumab. **ID:** EG007 **Other Num Affected:** 10 **Other Num at Risk:** 12 **Serious Number At Risk:** 12 **Title:** Placebo **Frequency Threshold:** 5 #### Other Events **Term:** Eye irritation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Abdominal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Flatulence **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Nausea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Application site dermatitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Fatigue **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Influenza like illness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Injection site haematoma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Injection site haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Injection site irritation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Injection site pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Injection site pruritus **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Injection site swelling **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Injection site urticaria **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Pyrexia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Hypersensitivity **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Immune system disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Bronchitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 14.1 **Term:** Hepatitis C **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 14.1 **Term:** Influenza **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 14.1 **Term:** Upper respiratory tract infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 14.1 **Term:** Viral infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 14.1 **Term:** Burns first degree **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 14.1 **Term:** Corneal abrasion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 14.1 **Term:** Eye injury **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 14.1 **Term:** Joint injury **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 14.1 **Term:** Skin laceration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 14.1 **Term:** Sunburn **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 14.1 **Term:** Aspartate aminotransferase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 14.1 **Term:** Blood creatine phosphokinase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 14.1 **Term:** Body temperature increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 14.1 **Term:** Dehydration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Hyponatraemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Increased appetite **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Polydipsia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Back pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Muscle spasms **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Muscular weakness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Musculoskeletal stiffness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Myalgia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Dizziness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Syncope **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Insomnia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Cough **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Epistaxis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Oropharyngeal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Sinus congestion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Dermatitis contact **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Ecchymosis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Pruritus **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 14.1 **Term:** Haematoma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 14.1 #### Serious Events **Term:** Hepatitis C **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 14.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 6 **Group ID:** EG001 **Num At Risk:** 6 **Group ID:** EG002 **Num At Risk:** 6 **Group ID:** EG003 **Num At Risk:** 6 **Group ID:** EG004 **Num At Risk:** 7 **Group ID:** EG005 **Num At Risk:** 6 **Group ID:** EG006 **Num Affected:** 1 **Num At Risk:** 37 **Group ID:** EG007 **Num At Risk:** 12 **Time Frame:** From first dose of study drug up to day 169 ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 6 **Group ID:** BG001 **Value:** 6 **Group ID:** BG002 **Value:** 6 **Group ID:** BG003 **Value:** 6 **Group ID:** BG004 **Value:** 7 **Group ID:** BG005 **Value:** 6 **Group ID:** BG006 **Value:** 12 **Group ID:** BG007 **Value:** 49 **Units:** Participants ### Group **ID:** BG000 **Title:** Tezepelumab 35 mg Q28D **Description:** Participants received 35 mg tezepelumab by subcutaneous injection once every 28 days (Q28D) for 3 doses. ### Group **ID:** BG001 **Title:** Tezepelumab 105 mg Q28D **Description:** Participants received 105 mg tezepelumab by subcutaneous injection once every 28 days for 3 doses. ### Group **ID:** BG002 **Title:** Tezepelumab 210 mg Q28D **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 28 days for 3 doses. ### Group **ID:** BG003 **Title:** Tezepelumab 210 mg Q14D **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 14 days (Q14D) for 6 doses. ### Group **ID:** BG004 **Title:** Tezepelumab 210 mg Q7D **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 7 days (Q7D) for 12 doses. ### Group **ID:** BG005 **Title:** Tezepelumab 700 mg Q28D IV **Description:** Participants received 700 mg tezepelumab by intravenous injection once every 28 days for 3 doses. ### Group **ID:** BG006 **Title:** Placebo **Description:** Participants received matching placebo administered subcutaneously (Cohorts 1-5) or intravenously (Cohort 6), matching the treatment regimen of tezepelumab. ### Group **ID:** BG007 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 9.3 **Value:** 29.8 #### Measurement **Group ID:** BG001 **Spread:** 5.8 **Value:** 34.8 #### Measurement **Group ID:** BG002 **Spread:** 5.3 **Value:** 32.3 #### Measurement **Group ID:** BG003 **Spread:** 5.6 **Value:** 31.5 #### Measurement **Group ID:** BG004 **Spread:** 7.7 **Value:** 36.1 #### Measurement **Group ID:** BG005 **Spread:** 4.1 **Value:** 27.7 #### Measurement **Group ID:** BG006 **Spread:** 5.2 **Value:** 34.1 #### Measurement **Group ID:** BG007 **Spread:** 6.4 **Value:** 32.6 **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:** 4 #### Measurement **Group ID:** BG004 **Value:** 1 #### Measurement **Group ID:** BG005 **Value:** 0 #### Measurement **Group ID:** BG006 **Value:** 2 #### Measurement **Group ID:** BG007 **Value:** 7 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 6 #### Measurement **Group ID:** BG001 **Value:** 6 #### Measurement **Group ID:** BG002 **Value:** 6 #### Measurement **Group ID:** BG003 **Value:** 2 #### Measurement **Group ID:** BG004 **Value:** 6 #### Measurement **Group ID:** BG005 **Value:** 6 #### Measurement **Group ID:** BG006 **Value:** 10 #### Measurement **Group ID:** BG007 **Value:** 42 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 2 #### Measurement **Group ID:** BG003 **Value:** 1 #### Measurement **Group ID:** BG004 **Value:** 4 #### Measurement **Group ID:** BG005 **Value:** 3 #### Measurement **Group ID:** BG006 **Value:** 4 #### Measurement **Group ID:** BG007 **Value:** 17 **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 #### Measurement **Group ID:** BG004 **Value:** 0 #### Measurement **Group ID:** BG005 **Value:** 2 #### Measurement **Group ID:** BG006 **Value:** 1 #### Measurement **Group ID:** BG007 **Value:** 3 **Category Title:** Black or African American #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 6 #### Measurement **Group ID:** BG002 **Value:** 4 #### Measurement **Group ID:** BG003 **Value:** 5 #### Measurement **Group ID:** BG004 **Value:** 3 #### Measurement **Group ID:** BG005 **Value:** 1 #### Measurement **Group ID:** BG006 **Value:** 7 #### Measurement **Group ID:** BG007 **Value:** 29 **Category Title:** Hispanic or Latino **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 **Population Description:** Participants who received at least 1 dose of study drug (Safety population). ## Results Section - More Information Module ### Certain Agreement **Other Details:** The Clinical Trial Agreement generally does not restrict an investigator's discussion of trial results after completion. The Agreement permits Amgen a limited period of time to review material discussing trial results (typically up to 45 days and possible extension). Amgen may remove confidential information, but authors have final control and approval of publication content. For multicenter studies, the investigator agrees not to publish any results before the first multi-center publication. **Restriction Type:** OTHER **Restrictive Agreement:** True ### Point of Contact **Email:** [email protected] **Organization:** Amgen Inc. **Phone:** 866-572-6436 **Title:** Study Director ## 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:** - **Upper Limit:** - **Value:** 4 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 6 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 - **Comment:** - **Group ID:** OG006 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 10 **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 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG006 - **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 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG006 - **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 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG006 - **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 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG006 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 **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 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG006 - **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 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG006 - **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:** 0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG006 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 69.7 - **Spread:** - **Upper Limit:** 167 - **Value:** 160 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 70.4 - **Spread:** - **Upper Limit:** 167 - **Value:** 71.5 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** 68.7 - **Spread:** - **Upper Limit:** 166 - **Value:** 118 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** 69.9 - **Spread:** - **Upper Limit:** 168 - **Value:** 70.7 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** 66.9 - **Spread:** - **Upper Limit:** 165 - **Value:** 164 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** 1.13 - **Spread:** - **Upper Limit:** 4.17 - **Value:** 4.00 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 6 - **Group ID:** OG001 - **Value:** 6 - **Group ID:** OG002 - **Value:** 6 - **Group ID:** OG003 - **Value:** 6 - **Group ID:** OG004 - **Value:** 7 - **Group ID:** OG005 - **Value:** 6 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 68.6 - **Spread:** - **Upper Limit:** 176 - **Value:** 166 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 70.7 - **Spread:** - **Upper Limit:** 167 - **Value:** 71.8 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** 69.3 - **Spread:** - **Upper Limit:** 168 - **Value:** 167 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** 65.1 - **Spread:** - **Upper Limit:** 162 - **Value:** 66.5 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** 65.9 - **Spread:** - **Upper Limit:** 164 - **Value:** 74.4 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** 1.17 - **Spread:** - **Upper Limit:** 4.03 - **Value:** 3.97 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 5 - **Group ID:** OG001 - **Value:** 3 - **Group ID:** OG002 - **Value:** 5 - **Group ID:** OG003 - **Value:** 6 - **Group ID:** OG004 - **Value:** 5 - **Group ID:** OG005 - **Value:** 5 **Units:** Participants #### 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:** 0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG006 - **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 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG006 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 1.16 - **Upper Limit:** - **Value:** 3.70 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 3.13 - **Upper Limit:** - **Value:** 10.7 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** 9.50 - **Upper Limit:** - **Value:** 23.6 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** 5.40 - **Upper Limit:** - **Value:** 23.8 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** 4.58 - **Upper Limit:** - **Value:** 18.0 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** 48.2 - **Upper Limit:** - **Value:** 294 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 6 - **Group ID:** OG001 - **Value:** 6 - **Group ID:** OG002 - **Value:** 6 - **Group ID:** OG003 - **Value:** 6 - **Group ID:** OG004 - **Value:** 7 - **Group ID:** OG005 - **Value:** 6 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 2.02 - **Upper Limit:** - **Value:** 6.29 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 2.02 - **Upper Limit:** - **Value:** 16.6 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** 17.5 - **Upper Limit:** - **Value:** 37.4 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** 13.5 - **Upper Limit:** - **Value:** 63.8 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** 45.6 - **Upper Limit:** - **Value:** 117 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** 74.9 - **Upper Limit:** - **Value:** 370 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 5 - **Group ID:** OG001 - **Value:** 3 - **Group ID:** OG002 - **Value:** 5 - **Group ID:** OG003 - **Value:** 6 - **Group ID:** OG004 - **Value:** 5 - **Group ID:** OG005 - **Value:** 5 **Units:** Participants #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 23.4 - **Upper Limit:** - **Value:** 78.9 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 50.5 - **Upper Limit:** - **Value:** 237 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** 171 - **Upper Limit:** - **Value:** 481 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** 49.2 - **Upper Limit:** - **Value:** 263 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** 34.9 - **Upper Limit:** - **Value:** 84.3 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** 395 - **Upper Limit:** - **Value:** 2980 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 6 - **Group ID:** OG001 - **Value:** 5 - **Group ID:** OG002 - **Value:** 6 - **Group ID:** OG003 - **Value:** 6 - **Group ID:** OG004 - **Value:** 7 - **Group ID:** OG005 - **Value:** 6 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 35.9 - **Upper Limit:** - **Value:** 136 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 28.8 - **Upper Limit:** - **Value:** 333 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** 338 - **Upper Limit:** - **Value:** 799 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** 180 - **Upper Limit:** - **Value:** 787 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** 224 - **Upper Limit:** - **Value:** 732 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** 1270 - **Upper Limit:** - **Value:** 4050 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 5 - **Group ID:** OG001 - **Value:** 3 - **Group ID:** OG002 - **Value:** 5 - **Group ID:** OG003 - **Value:** 6 - **Group ID:** OG004 - **Value:** 5 - **Group ID:** OG005 - **Value:** 5 **Units:** Participants #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.262 - **Upper Limit:** - **Value:** 1.82 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.0883 - **Upper Limit:** - **Value:** 1.64 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** 0.242 - **Upper Limit:** - **Value:** 1.59 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** 0.892 - **Upper Limit:** - **Value:** 2.89 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** 1.93 - **Upper Limit:** - **Value:** 8.23 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** 0.314 - **Upper Limit:** - **Value:** 1.34 **Title:** #### Outcome Measure 7 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.392 - **Upper Limit:** - **Value:** 1.79 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.0901 - **Upper Limit:** - **Value:** 1.66 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** 0.288 - **Upper Limit:** - **Value:** 1.59 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** 0.965 - **Upper Limit:** - **Value:** 2.84 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** 1.69 - **Upper Limit:** - **Value:** 6.74 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** 0.176 - **Upper Limit:** - **Value:** 1.30 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Adverse events (AEs) include any untoward medical occurrence in a trial participant administered a study drug and does not necessarily have a causal relationship with this treatment. AEs include worsening of a pre-existing medical condition and laboratory value changes requiring therapy or adjustment in prior therapy. AEs were assessed for severity according to the Common Terminology Criteria for Adverse Events (CTCAE), Version 3, where Grade 1 = Mild AE, Grade 2 = Moderate AE, Grade 3 = severe AE, Grade 4 = life-threatening AE and Grade 5 = death due to AE. Relationship to study treatment was determined by the investigator. A serious adverse event (SAE) is defined as an AE that met 1 or more of below criteria: * was fatal; * was life threatening; * required in-patient hospitalization or prolongation of existing hospitalization; * resulted in persistent or significant disability/incapacity; * was a congenital anomaly/birth defect; * other significant medical hazard. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** All participants who received at least 1 dose of study drug **Reporting Status:** POSTED **Time Frame:** From first dose of study drug up to day 169 **Title:** Number of Participants With Treatment-emergent Adverse Events **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Participants received 35 mg tezepelumab by subcutaneous injection once every 28 days (Q28D) for three doses. **ID:** OG000 **Title:** Tezepelumab 35 mg Q28D ##### Group **Description:** Participants received 105 mg tezepelumab by subcutaneous injection once every 28 days for 3 doses. **ID:** OG001 **Title:** Tezepelumab 105 mg Q28D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 28 days for 3 doses. **ID:** OG002 **Title:** Tezepelumab 210 mg Q28D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 14 days (Q14D) for 6 doses. **ID:** OG003 **Title:** Tezepelumab 210 mg Q14D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 7 days (Q7D) for 12 doses. **ID:** OG004 **Title:** Tezepelumab 210 mg Q7D ##### Group **Description:** Participants received 700 mg tezepelumab by intravenous injection once every 28 days for 3 doses. **ID:** OG005 **Title:** Tezepelumab 700 mg Q28D IV ##### Group **Description:** Participants received matching placebo administered subcutaneously (Cohorts 1-5) or intravenously (Cohort 6), matching the treatment regimen of tezepelumab. **ID:** OG006 **Title:** Placebo #### Outcome Measure 2 **Description:** The pharmacokinetic (PK) parameter Tmax was estimated based on the serum concentrations of tezepelumab using noncompartmental methods. The concentration of tezepelumab in human serum was measured using a validated enzyme-linked immunosorbent assay (ELISA). The lower limit of quantification of the assay was 10 ng/ml. **Dispersion Type:** Full Range **Parameter Type:** MEDIAN **Population Description:** Participants who received tezepelumab and had a sufficient number of serum concentration measurements for computing the PK parameter after first dose and after last dose. **Reporting Status:** POSTED **Time Frame:** First dose: Day 1 predose, end of infusion (EOI; IV cohort only), 4 hours, 3, 7, 14 (Q14D & Q28D only) and 28 days (Q28D only) postdose. Last Dose: Day 57 (Q28D), Day 71 (Q14D) and Day 78 (Q7D) predose, EOI (IV cohort), 4 hours, 3, 7, 14, 28 days postdose **Title:** Time of Maximum Observed Concentration (Tmax) of Tezepelumab **Type:** SECONDARY **Unit of Measure:** hours ##### Group **Description:** Participants received 35 mg tezepelumab by subcutaneous injection once every 28 days (Q28D) for 3 doses. **ID:** OG000 **Title:** Tezepelumab 35 mg Q28D ##### Group **Description:** Participants received 105 mg tezepelumab by subcutaneous injection once every 28 days for three doses. **ID:** OG001 **Title:** Tezepelumab 105 mg Q28D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 28 days for three doses. **ID:** OG002 **Title:** Tezepelumab 210 mg Q28D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 14 days (Q14D) for 6 doses. **ID:** OG003 **Title:** Tezepelumab 210 mg Q14D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 7 days (Q7D) for 12 doses. **ID:** OG004 **Title:** Tezepelumab 210 mg Q7D ##### Group **Description:** Participants received 700 mg tezepelumab by intravenous injection once every 28 days for 3 doses. **ID:** OG005 **Title:** Tezepelumab 700 mg Q28D IV #### Outcome Measure 3 **Description:** All study samples (tezepelumab and placebo) were tested using an electrochemiluminescence (ECL) based immunoassay to detect and confirm the presence of antibodies capable of binding to tezepelumab. Samples identified as positive in the immunoassay were tested in a receptor-binding ECL-based assay to detect neutralizing or inhibitory effects toward tezepelumab. The number of participants with positive anti-tezepelumab binding antibodies / neutralizing antibodies at any time post-baseline with a negative or no result at baseline is reported. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** All participants who received at least 1 dose of study drug **Reporting Status:** POSTED **Time Frame:** For Q28D groups: Days 28, 56, 85, 113, and 169; For Q14D and Q7D groups: Days 29, 57, 85, 113, 141, and 169 **Title:** Number of Participants Who Developed Anti-tezepelumab Antibodies After Initiation of Treatment **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Participants received 35 mg tezepelumab by subcutaneous injection once every 28 days (Q28D) for three doses. **ID:** OG000 **Title:** Tezepelumab 35 mg Q28D ##### Group **Description:** Participants received 105 mg tezepelumab by subcutaneous injection once every 28 days for three doses. **ID:** OG001 **Title:** Tezepelumab 105 mg Q28D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 28 days for three doses. **ID:** OG002 **Title:** Tezepelumab 210 mg Q28D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 14 days (Q14D) for 6 doses. **ID:** OG003 **Title:** Tezepelumab 210 mg Q14D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 7 days (Q7D) for 12 doses. **ID:** OG004 **Title:** Tezepelumab 210 mg Q7D ##### Group **Description:** Participants received 700 mg tezepelumab by intravenous injection once every 28 days for 3 doses. **ID:** OG005 **Title:** Tezepelumab 700 mg Q28D IV ##### Group **Description:** Participants received matching placebo administered subcutaneously (Cohorts 1-5) or intravenously (Cohort 6), matching the treatment regimen of tezepelumab. **ID:** OG006 **Title:** Placebo #### Outcome Measure 4 **Description:** The PK parameter Cmax was estimated based on the serum concentrations of tezepelumab using noncompartmental methods. The concentration of tezepelumab in human serum was measured using a validated ELISA. The lower limit of quantification of the assay was 10 ng/ml. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Participants who received tezepelumab and had a sufficient number of serum concentration measurements for computing the PK parameter after first dose and after last dose. **Reporting Status:** POSTED **Time Frame:** First dose: Day 1 predose, end of infusion (EOI; IV cohort only), 4 hours, 3, 7, 14 (Q14D & Q28D only) and 28 days (Q28D only) postdose. Last Dose: Day 57 (Q28D), Day 71 (Q14D) and Day 78 (Q7D) predose, EOI (IV cohort), 4 hours, 3, 7, 14, 28 days postdose **Title:** Maximum Observed Concentration (Cmax) of Tezepelumab **Type:** SECONDARY **Unit of Measure:** µg/mL ##### Group **Description:** Participants received 35 mg tezepelumab by subcutaneous injection once every 28 days (Q28D) for three doses. **ID:** OG000 **Title:** Tezepelumab 35 mg Q28D ##### Group **Description:** Participants received 105 mg tezepelumab by subcutaneous injection once every 28 days for three doses. **ID:** OG001 **Title:** Tezepelumab 105 mg Q28D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 28 days for three doses. **ID:** OG002 **Title:** Tezepelumab 210 mg Q28D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 14 days (Q14D) for 6 doses. **ID:** OG003 **Title:** Tezepelumab 210 mg Q14D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 7 days (Q7D) for 12 doses. **ID:** OG004 **Title:** Tezepelumab 210 mg Q7D ##### Group **Description:** Participants received 700 mg tezepelumab by intravenous injection once every 28 days for 3 doses. **ID:** OG005 **Title:** Tezepelumab 700 mg Q28D IV #### Outcome Measure 5 **Description:** The PK parameter AUCtau was estimated based on the serum concentrations of tezepelumab using noncompartmental methods. The dosing interval (tau) was 28 days, 14 days or 7 days depending on the treatment arm. The concentration of tezepelumab in human serum was measured using a validated ELISA. The lower limit of quantification of the assay was 10 ng/ml. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Participants who received tezepelumab and had a sufficient number of serum concentration measurements for computing the PK parameter after first dose and after last dose. **Reporting Status:** POSTED **Time Frame:** First dose: Day 1 predose, end of infusion (EOI; IV cohort only), 4 hours, 3, 7, 14 (Q14D & Q28D only) and 28 days (Q28D only) postdose. Last Dose: Day 57 (Q28D), Day 71 (Q14D) and Day 78 (Q7D) predose, EOI (IV cohort), 4 hours, 3, 7, 14, 28 days postdose **Title:** Area Under the Concentration-time Curve Over the Dosing Interval (AUCtau) for Tezepelumab **Type:** SECONDARY **Unit of Measure:** days*µg/mL ##### Group **Description:** Participants received 35 mg tezepelumab by subcutaneous injection once every 28 days (Q28D) for three doses. **ID:** OG000 **Title:** Tezepelumab 35 mg Q28D ##### Group **Description:** Participants received 105 mg tezepelumab by subcutaneous injection once every 28 days for three doses. **ID:** OG001 **Title:** Tezepelumab 105 mg Q28D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 28 days for three doses. **ID:** OG002 **Title:** Tezepelumab 210 mg Q28D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 14 days (Q14D) for 6 doses. **ID:** OG003 **Title:** Tezepelumab 210 mg Q14D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 7 days (Q7D) for 12 doses. **ID:** OG004 **Title:** Tezepelumab 210 mg Q7D ##### Group **Description:** Participants received 700 mg tezepelumab by intravenous injection once every 28 days for 3 doses. **ID:** OG005 **Title:** Tezepelumab 700 mg Q28D IV #### Outcome Measure 6 **Description:** Accumulation ratio (AR) based on AUCtau was calculated as AUCtau after last dose / AUCtau after first dose, except for the Q7D cohort where AR was calculated as AUCtau after last dose / area under the concentration-time curve from time 0 to time of last measurable concentration (AUC0-t) after first dose. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Participants who received tezepelumab and had a sufficient number of serum concentration measurements for computing the PK parameter after first dose and after last dose. **Reporting Status:** POSTED **Time Frame:** First dose: Day 1 predose, end of infusion (EOI; IV cohort only), 4 hours, 3, 7, 14 (Q14D & Q28D only) and 28 days (Q28D only) postdose. Last Dose: Day 57 (Q28D), Day 71 (Q14D) and Day 78 (Q7D) predose, EOI (IV cohort), 4 hours, 3, 7, 14, 28 days postdose **Title:** Accumulation Ratio Based on AUCtau **Type:** SECONDARY **Unit of Measure:** ratio ##### Group **Description:** Participants received 35 mg tezepelumab by subcutaneous injection once every 28 days (Q28D) for three doses. **ID:** OG000 **Title:** Tezepelumab 35 mg Q28D ##### Group **Description:** Participants received 105 mg tezepelumab by subcutaneous injection once every 28 days for three doses. **ID:** OG001 **Title:** Tezepelumab 105 mg Q28D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 28 days for three doses. **ID:** OG002 **Title:** Tezepelumab 210 mg Q28D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 14 days (Q14D) for 6 doses. **ID:** OG003 **Title:** Tezepelumab 210 mg Q14D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 7 days (Q7D) for 12 doses. **ID:** OG004 **Title:** Tezepelumab 210 mg Q7D ##### Group **Description:** Participants received 700 mg tezepelumab by intravenous injection once every 28 days for 3 doses. **ID:** OG005 **Title:** Tezepelumab 700 mg Q28D IV #### Outcome Measure 7 **Description:** Accumulation ratio based on Cmax calculated as Cmax after last dose / Cmax after first dose **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Participants who received tezepelumab and had a sufficient number of serum concentration measurements for computing the PK parameter after first dose and after last dose. **Reporting Status:** POSTED **Time Frame:** First dose: Day 1 predose, end of infusion (EOI; IV cohort only), 4 hours, 3, 7, 14 (Q14D & Q28D only) and 28 days (Q28D only) postdose. Last Dose: Day 57 (Q28D), Day 71 (Q14D) and Day 78 (Q7D) predose, EOI (IV cohort), 4 hours, 3, 7, 14, 28 days postdose **Title:** Accumulation Ratio Based on Cmax **Type:** SECONDARY **Unit of Measure:** ratio ##### Group **Description:** Participants received 35 mg tezepelumab by subcutaneous injection once every 28 days (Q28D) for three doses. **ID:** OG000 **Title:** Tezepelumab 35 mg Q28D ##### Group **Description:** Participants received 105 mg tezepelumab by subcutaneous injection once every 28 days for three doses. **ID:** OG001 **Title:** Tezepelumab 105 mg Q28D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 28 days for three doses. **ID:** OG002 **Title:** Tezepelumab 210 mg Q28D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 14 days (Q14D) for 6 doses. **ID:** OG003 **Title:** Tezepelumab 210 mg Q14D ##### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 7 days (Q7D) for 12 doses. **ID:** OG004 **Title:** Tezepelumab 210 mg Q7D ##### Group **Description:** Participants received 700 mg tezepelumab by intravenous injection once every 28 days for 3 doses. **ID:** OG005 **Title:** Tezepelumab 700 mg Q28D IV ### Participant Flow Module #### Group **Description:** Participants received 35 mg tezepelumab by subcutaneous injection once every 28 days (Q28D) for 3 doses. **ID:** FG000 **Title:** Tezepelumab 35 mg Q28D #### Group **Description:** Participants received 105 mg tezepelumab by subcutaneous injection once every 28 days for 3 doses. **ID:** FG001 **Title:** Tezepelumab 105 mg Q28D #### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 28 days for 3 doses. **ID:** FG002 **Title:** Tezepelumab 210 mg Q28D #### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 14 days (Q14D) for 6 doses. **ID:** FG003 **Title:** Tezepelumab 210 mg Q14D #### Group **Description:** Participants received 210 mg tezepelumab by subcutaneous injection once every 7 days (Q7D) for 12 doses. **ID:** FG004 **Title:** Tezepelumab 210 mg Q7D #### Group **Description:** Participants received 700 mg tezepelumab by intravenous injection once every 28 days for 3 doses. **ID:** FG005 **Title:** Tezepelumab 700 mg Q28D IV #### Group **Description:** Participants received matching placebo administered subcutaneously (Cohorts 1-5) or intravenously (Cohort 6), matching the treatment regimen of tezepelumab. **ID:** FG006 **Title:** Placebo #### Period **Title:** Overall Study ##### Withdraw **Type:** Noncompliance ###### 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:** 1 ###### Reason **Group ID:** FG005 **Number of Subjects:** 0 ###### Reason **Group ID:** FG006 **Number of Subjects:** 1 ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 3 ###### 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:** 2 ##### Withdraw **Type:** Administrative Decision ###### 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 ##### 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:** 1 ###### Reason **Group ID:** FG003 **Number of Subjects:** 0 ###### Reason **Group ID:** FG004 **Number of Subjects:** 0 ###### Reason **Group ID:** FG005 **Number of Subjects:** 3 ###### Reason **Group ID:** FG006 **Number of Subjects:** 1 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 6 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 6 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 6 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 6 ###### Achievement **Group ID:** FG004 **Number of Subjects:** 7 ###### Achievement **Group ID:** FG005 **Number of Subjects:** 6 ###### Achievement **Group ID:** FG006 **Number of Subjects:** 12 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 5 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 3 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 5 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 5 ###### Achievement **Group ID:** FG004 **Number of Subjects:** 5 ###### Achievement **Group ID:** FG005 **Number of Subjects:** 3 ###### Achievement **Group ID:** FG006 **Number of Subjects:** 8 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 1 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 3 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 1 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 1 ###### Achievement **Group ID:** FG004 **Number of Subjects:** 2 ###### Achievement **Group ID:** FG005 **Number of Subjects:** 3 ###### Achievement **Group ID:** FG006 **Number of Subjects:** 4 **Pre-Assignment Details:** Participants were enrolled into 1 of 6 cohorts. In the first 5 cohorts escalating subcutaneous doses of tezepelumab were compared with placebo and in cohort 6 a regimen of intravenous tezepelumab was compared with placebo. Within each cohort, healthy participants were randomized at a 6:2 ratio to receive either tezepelumab or placebo. **Recruitment Details:** This study was conducted at a single center in the United States. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT04379479 **Acronym:** FUTURE-T **Brief Title:** Clinical Effect of Dialyzable Leukocyte Extract in Suspected or Confirmed Cases of COVID-19 (FUTURE-T) **Official Title:** Assessment of the Clinical Effect of Dialyzable Leukocyte Extracts in Individuals With Acute Respiratory Infection (Suspected or Confirmed Cases of COVID-19) (FUTURE-T) #### Organization Study ID Info **ID:** IC-20-001 #### Organization **Class:** OTHER **Full Name:** National Polytechnic Institute, Mexico #### Secondary ID Infos **Domain:** Instituto Politécnico Nacional **ID:** SH-001-2020 **Type:** OTHER ### Status Module #### Completion Date **Date:** 2020-12 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** NOT_YET_RECRUITING #### Last Update Post Date **Date:** 2020-05-07 **Type:** ACTUAL **Last Update Submit Date:** 2020-05-06 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2020-08 **Type:** ESTIMATED #### Start Date **Date:** 2020-05 **Type:** ESTIMATED **Status Verified Date:** 2020-05 #### Study First Post Date **Date:** 2020-05-07 **Type:** ACTUAL **Study First Submit Date:** 2020-05-01 **Study First Submit QC Date:** 2020-05-06 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** National Polytechnic Institute, Mexico #### Responsible Party **Investigator Affiliation:** National Polytechnic Institute, Mexico **Investigator Full Name:** Toni Homberg von Thaden **Investigator Title:** Principal Investigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** Main goal: To generate information on the efficacy and safety of Dialyzable Leukocyte Extract (DLE) as an aid in the treatment of patients with acute respiratory infection (suspected or confirmed cases of COVID-19). Primary goal: To generate information on the efficacy of DLE as an aid in symptomatic treatment, by reducing the signs and symptoms of acute respiratory infection (suspected/confirmed cases of COVID-19). Secondary goals: 1. To evaluate clinical deterioration and respiratory alarm data. 2. To evaluate the duration of the clinical picture. 3. To explore cytokine changes associated with the therapeutic effect induced by DLE. 4. To obtain data on the safety of DLE as an aid in the symptomatic treatment of acute respiratory infection (suspected/confirmed cases of COVID-19). 5. To generate information to validate the contingency scale to assess the severity of acute respiratory disease (suspected/confirmed cases of COVID-19). Justification The systemic inflammatory response has been recognized as being responsible for COVID-19 complications. Immunomodulation strategies to control it are currently being considered, including the use of systemic steroids to down-regulate the systemic inflammatory response, the use of human immunoglobulin and even chloroquine given its anti-inflammatory and antiviral qualities; however, none of these treatments has been sufficiently studied or has shown any significant change in the clinical course of infected patients. Due to the importance of the COVID-19 pandemic and in the absence of specific treatment, it is important to implement new treatments that allow modulating the immune response, and one strategy may be the addition of DLE to symptomatic and supportive treatment. Hypotheses by goals. 1. The addition of DLE to the symptomatic treatment could decrease the severity of the clinical outcome (signs and symptoms) in individuals with an acute respiratory infection (cases suspected/confirmed by COVID-19). 2. The addition of DLE to the symptomatic treatment could decrease the clinical deterioration due to the acute respiratory infectious process (suspected/confirmed cases of COVID-19). 3. The addition of DLE to the symptomatic treatment could decrease the duration of the clinical outcome (suspected/confirmed cases of COVID-19). **Detailed Description:** Dialyzable Leukocyte Extract (DLE) (Transferon oral®) is a dialyzed extract obtained from human leukocytes. Its active ingredient is a complex mixture of low molecular weight peptides obtained from dialysis (12 kDa), and subsequent ultrafiltration (10 kDa) of lysed leukocyte and platelet concentrates. This product is manufactured by Laboratory of Scientific Research (Pharma-FT) at the Nacional School of Biological Sciences (ENCB), National Polytechnic Institute (IPN) using a patented method in Mexico (MX/a/2011/013852), USA (US9328152B2), EU (EPA-126128), Canada (2860260), Peru (000969-2014/DIN) and Colombia (14138326). DLE is a complex drug, the active principle is multiple peptide units. Thus, DLE cannot be fully characterized, requiring the application of analytical techniques that can describe the overall behavior of all of its components (peptide polydispersity). Two orthogonal techniques have been used to determine the molecular weight of the peptide components of DLE based on their migration through a polymer matrix. The molecular weight of 10 kDa was determined by polyacrylamide gels electrophoresis under denaturing conditions (SDS-PAGE), while a molecular weight lower than 15 kDa was determined by molecular exclusion chromatography. The peptide nature of DLE has been demonstrated using an aminogram method. This method includes the acidic hydrolysis of the peptide components until free amino acids are obtained, which are derivatized and identified based on the retention time of an amino acid standard using reverse phase chromatography (RP-UPLC). Additionally, the peptides of this drug were determined to be highly soluble by RP-UPLC coupled to UV, while mass spectometry, coupled to RP-UPLC, exhibited consistent ionization patterns. Non-clinical and clinical studies relevant to this research. Experimentally, in a murine model of virus infection, administration of DLE showed a decrease in serum concentrations for TNF and IL-6, accompanied by an increase of IFN. In humans, viral etiology is responsible for 90-95% of cases of respiratory tract infections. The common viruses involved in respiratory diseases are rhinoviruses (∼50%), coronaviruses (∼10%), adenoviruses, respiratory syncytial virus, influenza, and parainfluenza viruses (which generally add up between 10% to 15%), human metapneumovirus (\<5 %). The first evidence that DLE might have a therapeutic effect in respiratory infections arose from the analysis of a series of cases (36 adults and 63 children) with allergic rhinitis who received DLE in addition to the standard treatment. 11% of adults and 25.4% of children reported a decrease in the frequency of respiratory infections. Similarly, in a series of 70 adults and 54 children with persistent moderate asthma who received treatment with DLE as adjuvant therapy, 44% of adults and 31% of children reported a decrease in the frequency of respiratory infections, in addition to the improvement of allergic symptoms. In a study of 52 individuals, the effectiveness of DLE was evaluated by reducing the severity and frequency of infectious symptoms. That study showed a decrease in the frequency and in the severity of the infectious respiratory symptoms (77.1%) in pediatric patients. In comparison, 82.4% of adults decrease the frequency and/or diminish the severity of infectious respiratory symptoms. In a clinical follow-up of pediatric patients with sepsis to whom DLE was added to the standard treatment decrease in C-reactive protein levels, reduction in the neutrophil count at 72 hours after hospitalization, and an increase in the survival rate of 30% was reported. SARS-CoV-2 induces significant lung damage due to uncontrolled and dysregulated inflammatory activity, then immunomodulatory drugs have been suggested as treatment. The findings related to DLE, support their immunomodulatory capacity as a potential therapeutic tool in infectious respiratory diseases with a prominent inflammatory component. Primary goal. To generate information on the efficacy of DLE as an aid in symptomatic treatment, by reducing the signs and symptoms of acute respiratory infection (suspected/confirmed cases of COVID-19). Secondary goals. 1. To evaluate clinical deterioration and respiratory alarm data. 2. To assess the duration of the clinical manifestations. 3. To explore cytokine changes 4. To obtain data on the safety of DLE as an aid in the symptomatic treatment of acute respiratory infection (suspected/confirmed cases of COVID-19). 5. To generate information to validate the contingency scale (CS) to assess the severity of acute respiratory disease (suspected/confirmed cases of COVID-19). Hypothesis. The addition of DLE to the symptomatic treatment will reduce the signs and symptoms of acute respiratory infection. The recruitment method will be achieved electronically. The acute respiratory infection will be classified according to its clinical symptoms, following the Mexican Clinical Guideline in which COVID-19 disease is divided into non-serious and severe cases. The clinical evaluation of the patient will be assessed by a CS designed for this study, and includes three major items subdivided into minor items (range 0 to 4 in each one): General symptoms: Fever, Headache Superior airway respiratory symptoms: Sneezing, nasal congestion, runny nose Lower airway respiratory symptoms: Cough, Dyspnea, and Pain or Sense of constriction or oppression in the chest. Statistics. Four populations will be considered for the analysis: randomized individuals in the study or full analysis set (FAS), safety population (SP), the population of the intention-to-treat (ITT), and population per-protocol (PP). The FAS population will consist of all individuals who were randomized into the study (they are assigned a random number). The ITT population will consist of all patients who were randomized, who received at least one dose of the study drug, and who had at least one assessment of the primary or secondary endpoints after the baseline assessment. Following the principle of the intention of treatment, patients will be analyzed according to the treatment assigned during randomization. The SP will consist of all patients who received at least one dose of the study drug and who have at least one safety assessment after the baseline assessment. The patients will be analyzed according to the treatment they received. As a clarifying note, the statement that the patient has no adverse events constitutes a safety assessment. The PP will consist of all patients in the ITT population who complete all visits, who have an accurate evaluation of the primary efficacy variable, and who do not contain significant protocol violations. Statistical methods. Demographic data, clinical history, and key baseline efficacy variables for all randomized patients will be summarized by treatment group in tables (number and percentage) for qualitative variables; and in tables containing the average, standard deviation, median, minimum and maximum, by treatment group, for the quantitative variables. A comparison of groups at the baseline visit will be analyzed using Chi-square tests for qualitative variables and two-sample student t-tests for quantitative variables (p-values will be provided for descriptive purposes only). Additionally, the medical history, concomitant medications, comorbidities will be summarized using frequency tables. Primary outcome. The primary result of efficacy will be the measurement of the score of the CS at the end of treatment or early termination, in the ITT population. The algorithm of the imputation of the last observation carried forward (LOCF) will be applied in the case of patients who withdraw, discontinue therapy, or withdraw from the study. The primary analysis will be performed using the ITT population. This analysis will also be performed in the PP population to assess the robustness of the results. The primary outcome will be analyzed with a covariance model that includes the treatment group, baseline value, and center. The exploratory hypothesis of the primary objective is to establish whether there could be a difference in the decrease in the score of the CS at the end of treatment, under a superiority hypothesis approach described below: Ho: µPlacebo - µDLE\>0 Ha: µPlacebo - µDLE\<0 Possible superiority of the DLE addition as an aid to symptomatic treatment relative to symptomatic/placebo treatment will be established if the upper limit of the 2-sided 95% confidence interval for the difference µPlacebo-µDLE is less than 0. Secondary outcomes. The appearance of respiratory alarm data will be identified in the study groups, presenting the results in a frequency chart with the proportion of patients who presented alarm data by group. It will be analyzed using the Chi-Square test. The duration of clinical manifestations corresponds to the number of days with any of the signs and symptoms described in the CS. Descriptive statistics will be performed in each treatment group; the comparison will be made using the student's t-test. Immunological changes are defined as changes in the total leukocyte count, total lymphocytes, ESR, and serum concentration of CRP, procalcitonin, D-dimer, ferritin, CK, myoglobin, IL-6, TNF, interferons, and specific antibodies for the virus. Descriptive statistics will be presented by treatment group vs. visit, as well as the proportion of patients who show normalization of values during treatment. Sample size. Due to the lack of information on the efficacy of the study drug in cases of acute respiratory infection with COVID-19, the statistical calculation of the sample size does not apply. However, as a descriptive exercise, given the current contingency by COVID-19 pandemic, an estimation of the sample size is proposed in this study based on the CS that has been specifically designed for this study. Contingency score ranges from 0 to 32, considering that the study population is non-severe patients, the scoring average will correspond to the order of 16 points with a standard deviation of 4. It has also been assumed that the scoring behavior is that of a continuous variable with a normal distribution. The sample size calculation procedure is by means of the Julious 2004/FARTSSIE22 equation for superiority designs in parallel with two groups (DLE vs. Placebo). According to the following values: a. Type I error: 0.05 (5%); b. Type II error: 0.2 (20%). Power: 1-b: 0.8 (80%) Proposed standard deviation: 4 Difference to detect between groups: 1 The sample size relationship between groups: 1:1 (balanced) The sample size calculated is 253 per group. An additional 10% (q) will be included to offset withdrawals from the study or treatment; therefore, 562 patients (nt) 281 will be randomized by treatment in order to have the estimated number of patients for the hypothesis test. nt=n/1-q =506/0.9=562 Procedures to explain missing data. Missing data for the variables that are measured on a single occasion cannot be substituted, and that individual cannot be included in the analysis. For data that is measured on more than one time, in case of missing data, the last value measured after the baseline will be taken into account. (LOCF) Procedures for reporting any deviation from the statistical plan. If there are deviations from the original statistical plan, these will be described and justified in the final report. Selection of individuals to be included in the analysis. All randomized individuals who have received at least one dose of the investigational product under the maxim of ITT will be included in the analysis. Access to information. The study will be monitored by specific personnel; internal and external inspections and audits will be allowed and facilitated, allowing access to information while maintaining the confidentiality of the study individuals. ### Conditions Module **Conditions:** - COVID-19 - SARS-CoV-2 Infection **Keywords:** - Dialyzable leukocyte extract - Treatment COVID-19 - Treatment SARS-CoV-2 infection ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** Exploratory clinical trial, prospective, longitudinal, placebo-controlled, double-blinded, in patients with symptoms of non-severe acute respiratory infection (suspected/confirmed cases of COVID-19). ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - INVESTIGATOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 562 **Type:** ESTIMATED **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Oral administration 2 mg/5 mL every 24 hours for 14 days, following by 2 mg/5 mL twice a week for 3 weeks. All patients will receive paracetamol as symptomatic treatment, 500 mg oral administration 3 times per day. **Intervention Names:** - Drug: Dialyzable Leukocyte Extract **Label:** Dialyzable Leukocyte Extract **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Oral administration 5 mL every 24 hours for 14 days, following by 5 mL twice a week for 3 weeks. All patients will receive paracetamol as symptomatic treatment, 500 mg oral administration 3 times per day. **Intervention Names:** - Drug: Placebo oral **Label:** Placebo **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Dialyzable Leukocyte Extract **Description:** Experimental Intervention. Dialyzable Leukocyte Extract **Name:** Dialyzable Leukocyte Extract **Other Names:** - Transferon oral **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Placebo **Description:** Placebo **Name:** Placebo oral **Other Names:** - Placebo comparator **Type:** DRUG ### Outcomes Module #### Other Outcomes **Description:** Defined as the changes in the total concentration of the serum cytokines IL-6, TNF-α and type I and II Interferons due to DLE treatment. **Measure:** Cytokine concentration **Time Frame:** 35 days #### Primary Outcomes **Description:** Change in the score of the "Contingency scale to assess the severity of acute respiratory disease in cases suspected/confirmed by COVID-19" at the end of treatment concerning the baseline value. The clinical effect will be daily evaluated with the patient's diary in the cell app. Minimum value 0, Maximum value 32 points. Higher scores mean worse outcome. **Measure:** Change in the score of the "Contingency scale to assess the severity of acute respiratory disease in cases suspected/confirmed by COVID-19" **Time Frame:** 35 days #### Secondary Outcomes **Description:** Defined as the presence of signs and symptoms related with respiratory alarm symptoms. The respiratory alarm data will be daily evaluated with the patient's diary in the cell app. **Measure:** Clinical deterioration **Time Frame:** 35 days **Description:** Defined as the number of days with any of the symptoms mentioned in contingency table during the visits and follow-up through the patient's diary in the cell app. **Measure:** Duration of the clinical status **Time Frame:** 35 days ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Adults who agree to participate and sign informed consent. 2. Suspected case, according to the operational definition (CONAVE).\* 3. In the case of confirmed cases, will be those individuals who meet the operational definition of a suspected case and have a confirmed diagnosis by molecular biology, according to the operational definition (CONAVE).\*\* 4. The time of acute respiratory symptoms should be no longer than 72h. 5. Negative to the rapid test for influenza A/B. 6. Live in an urban area with easy access for visits. * Person of any age that has presented at least two of the following signs and symptoms: cough, fever or headache Accompanied by at least one of the following signs or symptoms: Dyspnea (signal of severity) Arthralgia Myalgia Odynophagia / pharyngeal burning Rhinorrhea Conjunctivitis Chest pain \*\*SARS-CoV2 infection confirmed by molecular diagnostic by one laboratory from the National Network of Public Health Laboratories recognized by InDRE. Exclusion Criteria: 1. Pregnancy. 2. Evidence of severe acute respiratory infection, even if it meets the criteria for a suspected or confirmed case. 3. Hepatic insufficiency 4. Diseases that occur with immunosuppression or therapeutic immunosuppression. 5. Heart diseases; controlled hypertension is allowed. 6. Metabolic diseases; controlled diabetes mellitus is allowed. 7. Individuals who have been treated with DLE in the last 6 months. CONAVE: National Committee for Epidemiological Surveillance. InDRE: Institute of Epidemiological Diagnosis and Reference. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Melissa I Espinosa-Navarro, MD **Phone:** +525557296000 **Phone Ext:** 62369 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Maricarmen Sánchez-Leon, MD **Phone:** +525557296000 **Phone Ext:** 62531 **Role:** CONTACT #### Locations **Location 1:** **City:** Mexico City **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Melissa I Espinosa-Navarro, MD - **Phone:** +525557296000 - **Phone Ext:** 62359 - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Maricarmen Sánchez-León, MD - **Phone:** +525557296000 - **Phone Ext:** 52531 - **Role:** CONTACT ***Contact 3:*** - **Name:** Toni A Homberg, MD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Mexico **Facility:** Escuela Nacional de Ciencias Biológicas, Instituto Politécnico Nacional **Zip:** 11 340 #### Overall Officials **Official 1:** **Affiliation:** Instituto Politecnico Nacional **Name:** Sonia Mayra Pérez-Tapia, PhD **Role:** STUDY_DIRECTOR **Official 2:** **Affiliation:** Universidad Nacional Autonoma de Mexico **Name:** Maria C Jimenez-Martínez, MD PhD **Role:** STUDY_DIRECTOR **Official 3:** **Affiliation:** National Polytechnic Institute, Mexico **Name:** Toni A Homberg von Thaden, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Access Criteria:** All data will be published in a scientific journal. **Description:** All data will be shared that underlies results in a publication **Info Types:** - STUDY_PROTOCOL - SAP - ICF - CSR - ANALYTIC_CODE **IPD Sharing:** YES **Time Frame:** The data will become available immediately at the end of study. ### References Module #### References **Citation:** Carballo-Uicab G, Linares-Trejo JE, Mellado-Sanchez G, Lopez-Morales CA, Velasco-Velazquez M, Pavon L, Estrada-Parra S, Perez-Tapia SM, Medina-Rivero E. Validation of a Cell Proliferation Assay to Assess the Potency of a Dialyzable Leukocyte Extract Intended for Batch Release. Molecules. 2019 Sep 20;24(19):3426. doi: 10.3390/molecules24193426. **PMID:** 31547184 **Citation:** Avila S, Munoz-Garcia L, Vazquez-Leyva S, Salinas-Jazmin N, Medina-Rivero E, Pavon L, Mellado-Sanchez G, Chacon-Salinas R, Estrada-Parra S, Vallejo-Castillo L, Perez-Tapia SM. Transferon, a peptide mixture with immunomodulatory properties is not immunogenic when administered with various adjuvants. J Immunotoxicol. 2017 Dec;14(1):169-177. doi: 10.1080/1547691X.2017.1346009. **PMID:** 28707490 **Citation:** Ramirez-Ramirez D, Vadillo E, Arriaga-Pizano LA, Mayani H, Estrada-Parra S, Velasco-Velazquez MA, Perez-Tapia SM, Pelayo R. Early Differentiation of Human CD11c+NK Cells with gammadelta T Cell Activation Properties Is Promoted by Dialyzable Leukocyte Extracts. J Immunol Res. 2016;2016:4097642. doi: 10.1155/2016/4097642. Epub 2016 Oct 25. **PMID:** 27847830 **Citation:** Medina-Rivero E, Vallejo-Castillo L, Vazquez-Leyva S, Perez-Sanchez G, Favari L, Velasco-Velazquez M, Estrada-Parra S, Pavon L, Perez-Tapia SM. Physicochemical Characteristics of Transferon Batches. Biomed Res Int. 2016;2016:7935181. doi: 10.1155/2016/7935181. Epub 2016 Jul 20. **PMID:** 27525277 **Citation:** Salinas-Jazmin N, Estrada-Parra S, Becerril-Garcia MA, Limon-Flores AY, Vazquez-Leyva S, Medina-Rivero E, Pavon L, Velasco-Velazquez MA, Perez-Tapia SM. Herpes murine model as a biological assay to test dialyzable leukocyte extracts activity. J Immunol Res. 2015;2015:146305. doi: 10.1155/2015/146305. Epub 2015 Apr 23. **PMID:** 25984538 **Citation:** Medina-Rivero E, Merchand-Reyes G, Pavon L, Vazquez-Leyva S, Perez-Sanchez G, Salinas-Jazmin N, Estrada-Parra S, Velasco-Velazquez M, Perez-Tapia SM. Batch-to-batch reproducibility of Transferon. J Pharm Biomed Anal. 2014 Jan;88:289-94. doi: 10.1016/j.jpba.2013.09.004. Epub 2013 Sep 17. **PMID:** 24099727 **Citation:** Homberg T, Sáenz V, Galicia-Carreón J, Lara I, Cervantes-Trujano E, Andaluz MC, Vera E, Pineda O, Ayala-Balboa J, Estrada-García A, Estrada-Parra S, Pérez-Tapia M, Jiménez-Martínez MC.The adverse event profile in patients treated with Transferon TM (Dialyzable leukocyte extracts): A preliminary report. Pharmacology & Pharmacy. 2015; 6(02): 65. #### See Also Links **Label:** Official site **URL:** https://www.encb.ipn.mx ## Derived Section ### Condition Browse Module - Ancestors - 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 ### 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 - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M2561 - Name: COVID-19 - Relevance: HIGH - As Found: COVID-19 - ID: M10283 - Name: Infections - Relevance: HIGH - As Found: Infection - ID: M6368 - Name: Communicable Diseases - Relevance: LOW - As Found: Unknown - ID: M14978 - Name: Respiratory Tract 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: 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: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000007239 - Term: Infections - ID: D000086382 - Term: COVID-19 ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03436979 **Brief Title:** The NeuGuide™ System for Vaginal Colpopexy in the Treatment of Uterine Prolapse **Official Title:** The NeuGuide™ System for Vaginal Colpopexy in the Treatment of Uterine Prolapse Post-Market Surveillance Clinical Study #### Organization Study ID Info **ID:** 06-CLP-0098 #### Organization **Class:** INDUSTRY **Full Name:** Pop Medical Solutions ### Status Module #### Completion Date **Date:** 2024-08-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-11-22 **Type:** ACTUAL **Last Update Submit Date:** 2023-11-21 **Overall Status:** ACTIVE_NOT_RECRUITING #### Primary Completion Date **Date:** 2023-10-30 **Type:** ACTUAL #### Start Date **Date:** 2018-01-01 **Type:** ACTUAL **Status Verified Date:** 2023-11 #### Study First Post Date **Date:** 2018-02-19 **Type:** ACTUAL **Study First Submit Date:** 2018-01-23 **Study First Submit QC Date:** 2018-02-08 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Pop Medical Solutions #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** True **Is FDA Regulated Drug:** False **Is US Export:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** The objective of the study is to assess the long term safety, durability of clinical effectiveness and cost effectiveness of the NeuGuide™ system when used for vaginal colpopexy in the treatment of uterine prolapse. ### Conditions Module **Conditions:** - Uterine Prolapse Without Vaginal Wall Prolapse **Keywords:** - prolapse - sacrospinous fixation - minimally invasive ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 60 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Subjects who are undergoing surgical treatment for uterine prolapse will be included in the study and will be treated using the NeuGuide™ System. **Intervention Names:** - Device: NeuGuide™ System **Label:** Subjects ### Interventions #### Intervention 1 **Arm Group Labels:** - Subjects **Description:** The NeuGuide™ device is indicated for anchoring sutures to ligaments of the pelvic floor. **Name:** NeuGuide™ System **Other Names:** - transvaginal sacrospinous ligament fixation **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Primary composite performance outcome **Measure:** A composite of the Pelvic Organ Prolapse Quantification (POP-Q) C point score, patient-rated symptom of vaginal bulging after vaginal colpopexy performed using the NeuGuide™ and the absence of further surgical therapy for uterine prolapse. **Time Frame:** 12 months **Description:** Primary safety of surgical implantation as reflected by adverse events **Measure:** The number of device / procedure related Serious Adverse Events (SAE). **Time Frame:** 30 days **Description:** Primary safety of NeuGuide treatment as reflected by adverse events **Measure:** The number of Serious Adverse Events and Adverse Events. **Time Frame:** 12 months #### Secondary Outcomes **Description:** Secondary anatomical performance of the NeuGuide treatment **Measure:** Change in mean POP-Q C point score from baseline (cm). **Time Frame:** one month **Description:** Secondary anatomical performance of the NeuGuide treatment **Measure:** Change in mean POP-Q C point score from baseline (cm). **Time Frame:** 6 months **Description:** Secondary anatomical of the NeuGuide treatment **Measure:** Change in mean POP-Q C point score from baseline (cm). **Time Frame:** 12 months **Description:** Secondary anatomical performance of the NeuGuide treatment outcome **Measure:** Change in mean POP-Q C point score from baseline (cm). **Time Frame:** 24 months **Description:** Secondary anatomical performance of the NeuGuide treatment **Measure:** Change in mean POP-Q C point score from baseline (cm). **Time Frame:** 36 months **Description:** Secondary performance: durability of the NeuGuide treatment **Measure:** Number of subjects who require surgical therapy of uterine prolapse or urinary incontinence. **Time Frame:** 36 months **Description:** Secondary symptomatic performance of the NeuGuide treatment **Measure:** Number of subjects in whom the primary symptomatic reason for repair of prolapse persists. **Time Frame:** 12 months **Description:** Secondary outcome: Cost performance of the procedure **Measure:** The time utilization of staff performing the NeuGuide procedure. **Time Frame:** one month **Description:** Secondary outcome: POP-Q Stage Score. **Measure:** The POP-Q Stage Score calculated from POP-Q measurements, where the Stages of POP-Q system measurement are as follows: Stage 0 no prolapse is demonstrated Stage 1 the most distal portion of the prolapse is more than 1 cm above the level of the hymen **Time Frame:** Baseline **Description:** Secondary outcome: POP-Q Stage Score. **Measure:** The POP-Q Stage Score calculated from POP-Q measurements, where the Stages of POP-Q system measurement are as follows: Stage 0 no prolapse is demonstrated Stage 1 the most distal portion of the prolapse is more than 1 cm above the level of the hymen **Time Frame:** one month **Description:** Secondary outcome: POP-Q Stage Score. **Measure:** The POP-Q Stage Score calculated from POP-Q measurements, where the Stages of POP-Q system measurement are as follows: Stage 0 no prolapse is demonstrated Stage 1 the most distal portion of the prolapse is more than 1 cm above the level of the hymen **Time Frame:** 12 months **Description:** Secondary outcome: POP-Q Stage Score. **Measure:** The POP-Q Stage Score calculated from POP-Q measurements, where the Stages of POP-Q system measurement are as follows: Stage 0 no prolapse is demonstrated Stage 1 the most distal portion of the prolapse is more than 1 cm above the level of the hymen **Time Frame:** 24 months **Description:** Secondary outcome: POP-Q Stage Score. **Measure:** The POP-Q Stage Score calculated from POP-Q measurements, where the Stages of POP-Q system measurement are as follows: Stage 0 no prolapse is demonstrated Stage 1 the most distal portion of the prolapse is more than 1 cm above the level of the hymen **Time Frame:** 36 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Patient with POP-Q C point greater than (-) 1 cm. 2. Patients who have previously had a partial hysterectomy in which the cervix is intact may be included in this study. 3. Non-pregnant female (female of child bearing potential must have a negative pregnancy test). 4. Patient understands the nature and potential hazards of the procedure and provides written informed consent prior to any study specific assessments. 5. Patient is able to complete written questionnaires. 6. Patient is willing and able to comply with the specified study requirements and follow-up assessments, and can be contacted by telephone. Exclusion Criteria: 1. Known diagnosis of reproductive tract abnormalities. 2. Prior pelvic radiation therapy, any malignancy or active pelvic inflammatory disease. 3. Known history of severe Pelvic Inflammatory Disease (PID). 4. Prior total hysterectomy. 5. Prior pelvic prolapse surgery using synthetic mesh. 6. Pathological PAP in the past year. 7. Moderate or severe bacterial cervicitis. 8. Moderate or severe pelvic pain (\> 3 on VAS). 9. Severe morbid obesity (BMI \>45). 10. Temperature \> 38°C (oral or equivalent), sepsis, or active infection requiring IV anti-microbial treatment. 11. Significant cognitive impairment. 12. Active malignancy other than non-melanoma skin cancer. 13. Planned surgery (more than a minor one) in the next 30 days. 14. Patient has a known hypersensitivity to device materials (Nickel, suture material). 15. Moribund patient or patient with severe or deteriorating damage in critical body systems. 16. Any condition that in the judgment of the investigators would interfere with the subject's ability to provide informed consent, comply with study instructions, place the subject at increased risk, or which might confound interpretation of study results. 17. Concurrent enrollment in another device or drug trial that has not completed the primary endpoint or clinically interferes with the current study endpoints. **Gender Based:** True **Gender Description:** Must have uterine prolapse **Maximum Age:** 84 Years **Minimum Age:** 36 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Subjects who are undergoing surgical treatment for uterine prolapse. ### Contacts Locations Module #### Locations **Location 1:** **City:** Washington **Country:** United States **Facility:** MedStar Health Research Institute **State:** District of Columbia **Zip:** 20010 **Location 2:** **City:** Fort Lauderdale **Country:** United States **Facility:** Holy Cross Hospital **State:** Florida **Zip:** 33364 **Location 3:** **City:** Weston **Country:** United States **Facility:** Cleveland Clinic Florida **State:** Florida **Zip:** 33331 **Location 4:** **City:** Cambridge **Country:** United States **Facility:** Mount Auburn Hospital **State:** Massachusetts **Zip:** 02138 **Location 5:** **City:** Brooklyn **Country:** United States **Facility:** Maimonides Medical Center **State:** New York **Zip:** 11220 **Location 6:** **City:** Valley Stream **Country:** United States **Facility:** South Nassau Community Hospital Cancer Center **State:** New York **Zip:** 11580 **Location 7:** **City:** Cleveland **Country:** United States **Facility:** Cleveland Clinic Cleveland **State:** Ohio **Zip:** 44195 **Location 8:** **City:** Allentown **Country:** United States **Facility:** The Institute for Female Pelvic Medicine and Reconstructive Surgery **State:** Pennsylvania **Zip:** 18103 **Location 9:** **City:** Newtown **Country:** United States **Facility:** Female Pelvic Health Center **State:** Pennsylvania **Zip:** 18940 **Location 10:** **City:** Dallas **Country:** United States **Facility:** Walnut Hill OB/GYN Associates **State:** Texas **Zip:** 75231 **Location 11:** **City:** Falls Church **Country:** United States **Facility:** INOVA Women's Hospital **State:** Virginia **Zip:** 22046 **Location 12:** **City:** Berlin-Tempelhof **Country:** Germany **Facility:** St. Joseph Krankenhaus **Location 13:** **City:** Berlin-Zehlendorf **Country:** Germany **Facility:** Krankenhaus Waldfriede **Zip:** 14163 **Location 14:** **City:** München **Country:** Germany **Facility:** Isar Kliniken GmbH **Location 15:** **City:** Be'er Sheva **Country:** Israel **Facility:** Soroka Medical Center #### Overall Officials **Official 1:** **Affiliation:** MAXIS Medical **Name:** James C Leiter, M.D. **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **Description:** There is no plan to make individual participant data a available to other researchers. **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020763 - Term: Pathological Conditions, Anatomical - 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 - ID: D000056887 - Term: Pelvic Organ Prolapse ### 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: M14261 - Name: Prolapse - Relevance: HIGH - As Found: Prolapse - ID: M17344 - Name: Uterine Prolapse - Relevance: HIGH - As Found: Uterine Prolapse - ID: M22519 - Name: Pathological Conditions, Anatomical - 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: M28618 - Name: Pelvic Organ Prolapse - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000014596 - Term: Uterine Prolapse - ID: D000011391 - Term: Prolapse ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00991679 **Brief Title:** CXCL9, CXCL10, CXCL11, and CXCR3 in Dry Eye Syndrome **Official Title:** Expression of CXCL9, CXCL10, CXCL11, and CXCR3 in the Tear Film and Ocular Surface of Patients With Dry Eye Syndrome #### Organization Study ID Info **ID:** I-2007-12-111 #### Organization **Class:** OTHER **Full Name:** Chonnam National University Hospital ### Status Module #### Completion Date **Date:** 2008-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2009-10-08 **Type:** ESTIMATED **Last Update Submit Date:** 2009-10-07 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2008-10 **Type:** ACTUAL #### Start Date **Date:** 2008-02 **Status Verified Date:** 2009-10 #### Study First Post Date **Date:** 2009-10-08 **Type:** ESTIMATED **Study First Submit Date:** 2009-10-07 **Study First Submit QC Date:** 2009-10-07 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Chonnam National University Hospital #### Responsible Party **Old Name Title:** Kyung-Chul Yoon, MD/ Assisted professor **Old Organization:** Department of Ophthalmology, Chonnam National University Medical School and Hospital, Gwangju, South Korea ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The aim of this study is to investigate the expression of CXCL9, CXCL10, CXCL11, and CXCR3 in the tear film and ocular surface of patients with dry eye syndrome. Thirty-three patients with dry eye (16 Sjögren's syndrome and 17 non-Sjögren's syndrome patients) and 15 control subjects were recruited. The concentrations of CXCL9, CXCL10, and CXCL11 in tears were measured using enzyme-linked immunosorbent assay. The correlation between chemokine levels and tear film and ocular surface parameters was analyzed. Expression of CXCL9, CXCL10, CXCL11, and CXCR3 in the conjunctiva was evaluated using immunohistochemistry. Flow cytometry was performed to count CXCR3+ cells and CXCR3+CD4+ cells in the conjunctiva. ### Conditions Module **Conditions:** - Dry Eye Syndrome **Keywords:** - Dry eye syndrome - CXCL9 chemokine - CXCL10 chemokine - CXCL11 chemokine - CXCR3 receptor ### Design Module #### Bio Spec **Description:** tears, conjunctiva **Retention:** SAMPLES_WITHOUT_DNA #### Design Info **Observational Model:** CASE_CONTROL **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 48 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Normal control subjects who did not show the clinical symptom and findings of dry eye syndrome. **Label:** Normal control subjects #### Arm Group 2 **Description:** Patients with dry eye syndrome who had symptoms of dry eye for more than 3 months, low tear film break up time (BUT, ≤7 sec), low Schirmer test (\<10 mm), low tear clearance rate (\<8X), and positive fluorescein or rose bengal vital staining (≥3) and were not treated with anti-inflammatory agents such as topical cyclosporine or steroids were included in the study. **Label:** dry eye patients ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * clinical diagnosis of dry eye syndrome Exclusion Criteria: * active ocular infection or inflammation not associated with dry eye * drug toxicity * contact lens wear * ocular allergy * ocular surgery within the last 3 months * lid or lash abnormalities **Healthy Volunteers:** True **Maximum Age:** 77 Years **Minimum Age:** 24 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Primary care clinic ### Contacts Locations Module #### Locations **Location 1:** **City:** Gwangju **Country:** Korea, Republic of **Facility:** Chonnam National University Hospital **State:** Chonnam **Zip:** 501-757 #### Overall Officials **Official 1:** **Affiliation:** Department of Ophthalmology, Chonnam National University Medical School and Hospital, Gwangju, South Korea **Name:** Kyung-Chul Yoon, MD, PhD **Role:** STUDY_DIRECTOR ### References Module #### References **Citation:** Yoon KC, De Paiva CS, Qi H, Chen Z, Farley WJ, Li DQ, Pflugfelder SC. Expression of Th-1 chemokines and chemokine receptors on the ocular surface of C57BL/6 mice: effects of desiccating stress. Invest Ophthalmol Vis Sci. 2007 Jun;48(6):2561-9. doi: 10.1167/iovs.07-0002. **PMID:** 17525185 **Citation:** Wallace GR, John Curnow S, Wloka K, Salmon M, Murray PI. The role of chemokines and their receptors in ocular disease. Prog Retin Eye Res. 2004 Jul;23(4):435-48. doi: 10.1016/j.preteyeres.2004.04.004. **PMID:** 15219876 **Citation:** Pflugfelder SC, Jones D, Ji Z, Afonso A, Monroy D. Altered cytokine balance in the tear fluid and conjunctiva of patients with Sjogren's syndrome keratoconjunctivitis sicca. Curr Eye Res. 1999 Sep;19(3):201-11. doi: 10.1076/ceyr.19.3.201.5309. **PMID:** 10487957 **Citation:** Yoon KC, Park CS, You IC, Choi HJ, Lee KH, Im SK, Park HY, Pflugfelder SC. Expression of CXCL9, -10, -11, and CXCR3 in the tear film and ocular surface of patients with dry eye syndrome. Invest Ophthalmol Vis Sci. 2010 Feb;51(2):643-50. doi: 10.1167/iovs.09-3425. Epub 2009 Oct 22. **PMID:** 19850844 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000004194 - Term: Disease - ID: D000010335 - Term: Pathologic Processes - ID: D000007766 - Term: Lacrimal Apparatus Diseases - ID: D000005128 - Term: Eye Diseases - ID: D000007637 - Term: Keratoconjunctivitis - ID: D000003231 - Term: Conjunctivitis - ID: D000003229 - Term: Conjunctival Diseases - ID: D000007634 - Term: Keratitis - ID: D000003316 - Term: Corneal 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: M16355 - Name: Syndrome - Relevance: HIGH - As Found: Syndrome - ID: M18040 - Name: Dry Eye Syndromes - Relevance: HIGH - As Found: Dry Eye Syndrome - ID: M10664 - Name: Keratoconjunctivitis Sicca - Relevance: HIGH - As Found: Dry Eye Syndrome - ID: M15241 - Name: Rupture - Relevance: LOW - As Found: Unknown - ID: M22785 - Name: Lacerations - Relevance: LOW - As Found: Unknown - ID: M10663 - Name: Keratoconjunctivitis - 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 - ID: M6455 - Name: Conjunctivitis - Relevance: LOW - As Found: Unknown - ID: M6453 - Name: Conjunctival Diseases - Relevance: LOW - As Found: Unknown - ID: M10660 - Name: Keratitis - Relevance: LOW - As Found: Unknown - ID: M6539 - Name: Corneal Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000015352 - Term: Dry Eye Syndromes - ID: D000007638 - Term: Keratoconjunctivitis Sicca - ID: D000013577 - Term: Syndrome ### Intervention Browse Module - Browse Branches - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: ARhu - Name: Antirheumatic Agents - Abbrev: Derm - Name: Dermatologic Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Infl - Name: Anti-Inflammatory Agents ### Intervention Browse Module - Browse Leaves - ID: M18961 - Name: Cyclosporine - Relevance: LOW - As Found: Unknown - ID: M6730 - Name: Cyclosporins - Relevance: LOW - As Found: Unknown - ID: M4217 - Name: Anti-Inflammatory Agents - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04743479 **Acronym:** ESPRIT-AI **Brief Title:** Artificial Intelligence-based Early Screening of Pancreatic Cancer and High Risk Tracing (ESPRIT-AI) **Official Title:** Artificial Intelligence-based Health Information Management System and Key Technology Study of Early Screening and Hierarchical Diagnosis and Treatment of Pancreatic Cancer #### Organization Study ID Info **ID:** ChanghaiH-PP07 #### Organization **Class:** OTHER **Full Name:** Changhai Hospital ### Status Module #### Completion Date **Date:** 2030-12-30 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-09-01 **Type:** ACTUAL **Last Update Submit Date:** 2023-08-31 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2025-12-30 **Type:** ESTIMATED #### Start Date **Date:** 2020-12-01 **Type:** ACTUAL **Status Verified Date:** 2023-08 #### Study First Post Date **Date:** 2021-02-08 **Type:** ACTUAL **Study First Submit Date:** 2021-02-03 **Study First Submit QC Date:** 2021-02-03 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Changhai Hospital #### Responsible Party **Investigator Affiliation:** Changhai Hospital **Investigator Full Name:** Guo ShiWei **Investigator Title:** Associated Professor at the Institute of Pancreatic Surgery **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** Pancreatic cancer is one of the most fatal malignancies with a 5-year survival rate of only \~6%\[1\]. The reasons for this high mortality rate can be attributed to several factors, of which perhaps the most important is delayed diagnosis due to vague symptoms and consequently missed opportunities for surgical resection. Therefore, the ability to detect pancreatic cancer at an early, more curable stage is urgently needed. Identifying risk factors and biomarkers of early pancreatic cancer could facilitate screening for individuals at higher than average risk and expedite the diagnosis in individuals with symptoms and substantially improve an individual's chance of surviving the disease. Thus, the investigators propose this longitudinal study entitled, "Artificial Intelligence-based Early Screening of Pancreatic Cancer and High Risk Tracing (ESPRIT-AI)" in order to generate clinical data sets and bank serial blood specimens of high risk individuals. **Detailed Description:** The study is being run by a team of dedicated physicians and researchers, led by Jin Gang, MD, Director of Department of general surgery of Shanghai Changhai Hospital. The trial will include individuals with new-onset diabetes (diagnosed within the past 3 year), familial pancreatic cancer, inherited syndromes associated with pancreatic cancer (including hereditary pancreatitis, familial atypical multiple mole and melanoma syndrome, hereditary nonpolyposis colon cancer, Peutz-Jeghers syndrome, hereditary breast and ovarian cancer syndromes, etc), pancreatic cystic neoplasm (including IPMN, MCN) as well as chronic pancreatitis. Participants will undergo annual laboratory tests and high-resolution MRI/CT examinations of the pancreas. Any suspicious lesions will be further examined by endoscopic ultrasound (EUS). If pancreatic cancer or a pre-cancerous lesion is identified, the individual will be referred for surgery. We will also be collecting a blood sample from all participants for DNA isolation. Clinical data and biological specimens contained in this study may be used for a wide variety of future related studies to the cause, diagnosis, outcome and treatment of pancreatic cancer. ### Conditions Module **Conditions:** - Pancreatic Cancer - Diabetes - Familial Pancreatic Cancer - Pancreatic Cystic Neoplasm - Chronic Pancreatitis - Hereditary Pancreatitis **Keywords:** - Screening - Early Diagnosis - Artificial Intelligence - Pancreatic Cancer ### Design Module #### Bio Spec **Description:** Patients in this study will have the option of donating blood for biobanking, approximately 10cc of plasma and 10cc of serum. Further, samples that are routinely collected for diagnostic purposes may be collected and banked at the time of their clinically routine procedure. If a patient consents to the use of extra material for research purposes, the biological samples will be banked and the blood, and/or pancreatic juice/cystic fluid, tissues, and saliva will be used for identification and characterization of potential biomarkers from de-identified samples. **Retention:** SAMPLES_WITH_DNA #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 5000 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** New Onset Diabetes must meet one of the following criteria: 1. Documented diabetes diagnosed within the past 3 years. 2. Definite new-onset diabetes based on recent fasting blood glucose (FBG) values ≥126 mg/dl (7.0 mmol/L) or Hemoglobin A1c (HbA1c) ≥ 6.5%. All glycemic parameters must be measured in an outpatient setting. **Intervention Names:** - Diagnostic Test: high-resolution MRI/CT examinations **Label:** New Onset Diabetes #### Arm Group 2 **Description:** Familial pancreatic cancer must meet one of the following criteria: 1. ≥ 2 blood relatives with pancreatic cancer (includes 1st-3rd degree relatives) 2. One 1st degree relative with PDAC diagnosed before age 60 **Intervention Names:** - Diagnostic Test: high-resolution MRI/CT examinations **Label:** Familial pancreatic cancer #### Arm Group 3 **Description:** Family history includes with inherited syndromes associated with pancreatic cancer ( ≥ 2 blood relative, includes 1st-3rd degree relatives). Inherited syndromes must meet one of the following criteria: 1. Hereditary pancreatitis 2. Familial atypical multiple mole and melanoma syndrome 3. Hereditary nonpolyposis colon cancer 4. Peutz-Jeghers syndrome 5. Hereditary breast and ovarian cancer syndromes **Intervention Names:** - Diagnostic Test: high-resolution MRI/CT examinations **Label:** Inherited syndromes associated with pancreatic cancer #### Arm Group 4 **Description:** Pancreatic Cystic Neoplasm, including intraductal papillary mucinous neoplasms (IPMN) and mucinous cystic neoplasms (MCN), which are defined by endoscopic ultrasound or serial imaging. **Intervention Names:** - Diagnostic Test: high-resolution MRI/CT examinations **Label:** Pancreatic Cystic Neoplasm #### Arm Group 5 **Description:** Chronic pancreatitis, defined by cross-sectional imaging, endoscopic ultrasound, functional testing abnormalities OR as diagnosed by a gastroenterologist. **Intervention Names:** - Diagnostic Test: high-resolution MRI/CT examinations **Label:** Chronic pancreatitis ### Interventions #### Intervention 1 **Arm Group Labels:** - Chronic pancreatitis - Familial pancreatic cancer - Inherited syndromes associated with pancreatic cancer - New Onset Diabetes - Pancreatic Cystic Neoplasm **Description:** Participants will undergo annual questionnaire survey, laboratory tests and high-resolution MRI/CT examinations of the pancreas. Any suspicious lesions will be further examined by endoscopic ultrasound (EUS). **Name:** high-resolution MRI/CT examinations **Other Names:** - questionnaire survey - laboratory tests **Type:** DIAGNOSTIC_TEST ### Outcomes Module #### Primary Outcomes **Description:** Determine incidence of pancreatic cancer or precursor lesions among high risk individuals. **Measure:** Incidence **Time Frame:** 5 years **Description:** Assesses the influence of risk factors on the incidence of pancreatic cancer or precursor lesions among high risk individuals. **Measure:** Hazard ratio (HR) **Time Frame:** 5 years #### Secondary Outcomes **Description:** Calculate survival time from point of diagnosis and treatment among the identified patients with pancreatic cancer. **Measure:** Survival time **Time Frame:** 5 years **Description:** Assesses the influence of risk factors on survival time among the identified patients with pancreatic cancer. **Measure:** HR **Time Frame:** 5 years **Description:** Determine diagnostic yield (sensitivity, specificity, positive/negative predictive value and accuracy) of AI-based surveillance program to predict early stage pancreatic cancer. **Measure:** Diagnostic yield **Time Frame:** 5 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Subject is able and willing to provide informed consent and sign an informed consent form. * Subject or authorized representative must be willing to complete a detailed questionnaire. * Subject must meet one of the following criteria: 1. New onset diabetes (diagnosed within the past 3 years) 2. Familial pancreatic cancer 3. Inherited syndromes associated with pancreatic cancer (including Hereditary pancreatitis, Familial atypical multiple mole and melanoma syndrome, Hereditary nonpolyposis colon cancer, Peutz-Jeghers syndrome, Hereditary breast and ovarian cancer syndromes, etc) 4. Pancreatic cystic neoplasm (including IPMN, MCN) 5. Chronic pancreatitis Exclusion Criteria: * Subject has been diagnosed with pancreatic cancer or other malignant tumors in the last 5 years; * Subject has any medical condition that contraindicates high-resolution MRI or CT; * Subject cannot be followed up or is participating in other clinical trials. **Maximum Age:** 75 Years **Minimum Age:** 50 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** High risk individuals of pancreatic cancer ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Beilei Wang, M.D. **Phone:** 13774238083 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Shiwei Guo, M.D. **Phone:** 18621500666 **Role:** CONTACT #### Locations **Location 1:** **City:** Shanghai **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Beilei Wang, M.D. - **Phone:** 13774238083 - **Role:** CONTACT ***Contact 2:*** - **Name:** Gang Jin, M.D. - **Role:** PRINCIPAL_INVESTIGATOR ***Contact 3:*** - **Name:** Bimeng Zhang, M.D. - **Role:** SUB_INVESTIGATOR **Country:** China **Facility:** Shanghai Changhai Hospital **Status:** RECRUITING **Zip:** 200433 #### Overall Officials **Official 1:** **Affiliation:** Department of general surgery, Changhai Hospital **Name:** Gang Jin, M.D. **Role:** STUDY_CHAIR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Kamisawa T, Wood LD, Itoi T, Takaori K. Pancreatic cancer. Lancet. 2016 Jul 2;388(10039):73-85. doi: 10.1016/S0140-6736(16)00141-0. Epub 2016 Jan 30. **PMID:** 26830752 **Citation:** Lin QJ, Yang F, Jin C, Fu DL. Current status and progress of pancreatic cancer in China. World J Gastroenterol. 2015 Jul 14;21(26):7988-8003. doi: 10.3748/wjg.v21.i26.7988. **PMID:** 26185370 **Citation:** Henrikson NB, Aiello Bowles EJ, Blasi PR, Morrison CC, Nguyen M, Pillarisetty VG, Lin JS. Screening for Pancreatic Cancer: Updated Evidence Report and Systematic Review for the US Preventive Services Task Force. JAMA. 2019 Aug 6;322(5):445-454. doi: 10.1001/jama.2019.6190. **PMID:** 31386140 **Citation:** Singhi AD, Koay EJ, Chari ST, Maitra A. Early Detection of Pancreatic Cancer: Opportunities and Challenges. Gastroenterology. 2019 May;156(7):2024-2040. doi: 10.1053/j.gastro.2019.01.259. Epub 2019 Feb 2. **PMID:** 30721664 **Citation:** Pereira SP, Oldfield L, Ney A, Hart PA, Keane MG, Pandol SJ, Li D, Greenhalf W, Jeon CY, Koay EJ, Almario CV, Halloran C, Lennon AM, Costello E. Early detection of pancreatic cancer. Lancet Gastroenterol Hepatol. 2020 Jul;5(7):698-710. doi: 10.1016/S2468-1253(19)30416-9. Epub 2020 Mar 2. **PMID:** 32135127 **Citation:** Maitra A, Sharma A, Brand RE, Van Den Eeden SK, Fisher WE, Hart PA, Hughes SJ, Mather KJ, Pandol SJ, Park WG, Feng Z, Serrano J, Rinaudo JAS, Srivastava S, Chari ST; Consortium for the Study of Chronic Pancreatitis, Diabetes, and Pancreatic Cancer (CPDPC). A Prospective Study to Establish a New-Onset Diabetes Cohort: From the Consortium for the Study of Chronic Pancreatitis, Diabetes, and Pancreatic Cancer. Pancreas. 2018 Nov/Dec;47(10):1244-1248. doi: 10.1097/MPA.0000000000001169. **PMID:** 30325864 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000004067 - Term: Digestive System Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000004701 - Term: Endocrine Gland Neoplasms - ID: D000004066 - Term: Digestive System Diseases - ID: D000010182 - Term: Pancreatic Diseases - ID: D000004700 - Term: Endocrine System Diseases - ID: D000002908 - Term: Chronic Disease - ID: D000020969 - Term: Disease Attributes - ID: D000010335 - Term: Pathologic Processes - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009370 - Term: Neoplasms by Histologic Type ### 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: BC04 - Name: Neoplasms - Abbrev: BC06 - Name: Digestive System Diseases - 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: M13110 - Name: Pancreatic Neoplasms - Relevance: HIGH - As Found: Pancreatic Cancer - ID: M13115 - Name: Pancreatitis - Relevance: HIGH - As Found: Pancreatitis - ID: M26260 - Name: Pancreatitis, Chronic - Relevance: HIGH - As Found: Chronic Pancreatitis - ID: M20441 - Name: Neoplasms, Cystic, Mucinous, and Serous - Relevance: HIGH - As Found: Cystic Neoplasms - ID: M8886 - Name: Gastrointestinal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7256 - Name: Digestive System Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7863 - Name: Endocrine Gland 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: M13102 - Name: Pancreatic Diseases - Relevance: LOW - As Found: Unknown - ID: M7862 - Name: Endocrine 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: M12320 - Name: Neoplasms, Glandular and Epithelial - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - 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 - ID: T2768 - Name: Hereditary Pancreatitis - Relevance: HIGH - As Found: Hereditary Pancreatitis - ID: T2243 - Name: Familial Pancreatic Cancer - Relevance: HIGH - As Found: Familial Pancreatic Cancer ### Condition Browse Module - Meshes - ID: D000010190 - Term: Pancreatic Neoplasms - ID: D000018297 - Term: Neoplasms, Cystic, Mucinous, and Serous - ID: D000010195 - Term: Pancreatitis - ID: D000050500 - Term: Pancreatitis, Chronic ### 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: Gast - Name: Gastrointestinal Agents - Abbrev: HB - Name: Herbal and Botanical ### Intervention Browse Module - Browse Leaves - ID: M207501 - Name: Chrysarobin - Relevance: LOW - As Found: Unknown - ID: M22554 - Name: Pancrelipase - Relevance: LOW - As Found: Unknown - ID: M13114 - Name: Pancreatin - Relevance: LOW - As Found: Unknown - ID: T120 - Name: Cola - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03176979 **Brief Title:** Contrast Enhanced Spectral Mammography With Digital Breast Tomosynthesis For Patients With Newly Diagnosed Breast Cancer **Official Title:** Emerging Tools in the Detection of Breast Cancer: Comparison of Contrast Enhanced Spectral Mammography With Digital Breast Tomosynthesis to Conventional Imaging Techniques Including Contrast Enhanced Magnetic Resonance Imaging and 2D Mammography With or Without Targeted Ultrasound #### Organization Study ID Info **ID:** 1B-16-3 #### Organization **Class:** OTHER **Full Name:** University of Southern California #### Secondary ID Infos **Domain:** CTRP (Clinical Trial Reporting Program) **ID:** NCI-2017-00778 **Type:** REGISTRY **Domain:** USC / Norris Comprehensive Cancer Center **ID:** 1B-16-3 **Type:** OTHER **ID:** P30CA014089 **Link:** https://reporter.nih.gov/quickSearch/P30CA014089 **Type:** NIH ### Status Module #### Completion Date **Date:** 2021-07-08 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-11-18 **Type:** ACTUAL **Last Update Submit Date:** 2021-11-16 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2021-07-08 **Type:** ACTUAL #### Start Date **Date:** 2017-04-21 **Type:** ACTUAL **Status Verified Date:** 2021-11 #### Study First Post Date **Date:** 2017-06-06 **Type:** ACTUAL **Study First Submit Date:** 2017-06-02 **Study First Submit QC Date:** 2017-06-02 ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** National Cancer Institute (NCI) #### Lead Sponsor **Class:** OTHER **Name:** University of Southern California #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Is US Export:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** This pilot clinical trial studies how well contrast enhanced spectral mammography works with digital breast tomosynthesis in imaging patients with newly diagnosed breast cancer. Contrast enhanced spectral mammography uses a special dye that is injected into the veins before mammogram images are taken. Digital breast tomosynthesis uses multiple x-ray pictures to produce a 3-dimensional rendering of the entire breast. Contrast enhanced spectral mammography with digital breast tomosynthesis may highlight areas of concern within the breast in more detail than a standard mammogram and improve the accuracy of tumor size. **Detailed Description:** PRIMARY OBJECTIVES: I. To compare the index lesion size (the largest diameter) from each of the four readings (standard of care 2 dimensional \[D\], magnetic resonance imaging \[MRI\], contrast enhanced spectral mammography \[CESM\], 3D) to gold standard index lesion size from surgical pathology (the largest diameter). II. To document the additional ipsilateral and contralateral breast cancer lesions detected by the MRI, CESM, and 3D readings listed above. OUTLINE: Patients undergo a clinical breast examination and a diagnostic mammogram with or without targeted breast ultrasound to the index cancer as part of their standard of care preoperative work-up. As part of the research study, patients receive contrast agent intravenously (IV) and then undergo a CESM with digital breast tomosynthesis (DBT) over 30 minutes. Patients also receive a contrast agent, gadolinium, IV and undergo bilateral breast contrast enhanced (CE)-MRI over 10 minutes. After completion of study, patients are followed up within 24-96 hours. ### Conditions Module **Conditions:** - Breast Carcinoma ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** DIAGNOSTIC #### Enrollment Info **Count:** 43 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients undergo a clinical breast examination and a diagnostic mammogram with or without targeted breast ultrasound to the index cancer as part of their standard of care preoperative work-up. As part of the research study, patients receive contrast agent IV and then undergo a CESM with DBT over 30 minutes. Patients also receive a contrast agent, gadolinium, IV and undergo bilateral breast CE-MRI over 10 minutes. **Intervention Names:** - Other: Contrast Agent - Procedure: Digital Tomosynthesis Mammography - Procedure: Dual-Energy Contrast-Enhanced Digital Spectral Mammography **Label:** Diagnostic (CESM with DBT) **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Diagnostic (CESM with DBT) **Description:** Given IV **Name:** Contrast Agent **Other Names:** - Contrast - Contrast Drugs - contrast material - Contrast Medium **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - Diagnostic (CESM with DBT) **Description:** Undergo CESM with DBT **Name:** Digital Tomosynthesis Mammography **Other Names:** - DBT - Digital Breast Tomosynthesis - Digital Tomosynthesis of the Breast **Type:** PROCEDURE #### Intervention 3 **Arm Group Labels:** - Diagnostic (CESM with DBT) **Description:** Undergo CESM with DBT **Name:** Dual-Energy Contrast-Enhanced Digital Spectral Mammography **Other Names:** - Dual-Energy Contrast-Enhanced Digital Mammography (CEDM) - Dual-Energy Contrast-Enhanced Digital Subtraction Mammography **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Description:** Intra-class correlation will be used to assess the agreement of index lesion size from each imaging reading to the gold standard (histopathology at surgery). Bland-Altman plot will be used to illustrate the pattern of difference between each reading and the gold standard. **Measure:** Index lesion size using the largest diameter **Time Frame:** Baseline ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Newly diagnosed breast cancer patients Exclusion Criteria: * Women with history of surgical, medical, or radiation therapy for breast cancer * Women with renal failure or insufficiency * Women with iodine contrast allergy * Women with gadolinium contrast allergy * Women who are pregnant, possibly pregnant, or lactating * Women undergoing neoadjuvant chemotherapy **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Los Angeles **Country:** United States **Facility:** USC / Norris Comprehensive Cancer Center **State:** California **Zip:** 90033 #### Overall Officials **Official 1:** **Affiliation:** University of Southern California **Name:** Mary Yamashita, MD **Role:** PRINCIPAL_INVESTIGATOR ## 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 Carcinoma - ID: M5534 - Name: Carcinoma - Relevance: LOW - As Found: Unknown - 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:** NCT01636479 **Brief Title:** Phase 1 Safety Testing of SAR405838 **Official Title:** A Phase 1 Study to Assess the Safety, Tolerability, Pharmacokinetics, and Biological Activity of SAR405838 in Patients With Advanced Cancer #### Organization Study ID Info **ID:** TED12318 #### Organization **Class:** INDUSTRY **Full Name:** Sanofi #### Secondary ID Infos **ID:** 2012-000733-39 **Domain:** UTN **ID:** U1111-1127-2911 **Type:** OTHER ### Status Module #### Completion Date **Date:** 2018-03-05 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-05-17 **Type:** ACTUAL **Last Update Submit Date:** 2018-05-16 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2018-03-05 **Type:** ACTUAL #### Start Date **Date:** 2012-07-13 **Status Verified Date:** 2018-05 #### Study First Post Date **Date:** 2012-07-10 **Type:** ESTIMATED **Study First Submit Date:** 2012-07-02 **Study First Submit QC Date:** 2012-07-05 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Sanofi #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Primary Objectives: * To determine safety and the maximum tolerated dose (MTD) of SAR405838 through the characterization of dose-limiting toxicities (DLTs). * To assess biological activities in patients with dedifferentiated liposarcoma during MTD cohort expansion. Secondary Objectives: * Pharmacokinetic (PK) profile of SAR405838. * Biomarkers in association with SAR405838. * Anti-tumor activity in response to SAR405838. * Food effect on SAR405838 PK. * Compliance with SAR405838 treatment. * Cytochrome P450 3A4/5 (CYP3A4/5) activity. **Detailed Description:** Total duration of study participation for each patient will be one month screening followed by treatment until precluded by toxicity, noncompliance, progression, or death. ### Conditions Module **Conditions:** - Neoplasm Malignant ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 77 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** SAR405838 in escalating doses **Intervention Names:** - Drug: SAR405838 **Label:** SAR405838 **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - SAR405838 **Description:** Pharmaceutical form: Capsule Route of administration: Oral **Name:** SAR405838 **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** SAR405838 Maximum tolerated dose (MTD) **Time Frame:** Cycle 1 (21 days) or 2 Cycles (42 days) dependent on dosing schedule **Measure:** In MTD cohort, clinical benefit **Time Frame:** Until disease progression #### Secondary Outcomes **Measure:** Adverse events (eg, number of patients experiencing AEs) **Time Frame:** Baseline to end of study **Measure:** PK parameters (Cmax, Tmax, AUC) **Time Frame:** Baseline to end of study **Measure:** Biomarkers **Time Frame:** Baseline to end of study **Measure:** Clinical response **Time Frame:** Baseline to end of study **Measure:** Drug administration compliance **Time Frame:** Baseline to end of study ### Eligibility Module **Eligibility Criteria:** Inclusion criteria: * Histologically or cytologically confirmed diagnosis of a solid tumor for which no further effective standard treatment is available. Patients with lymphomas may be enrolled. * For dose escalation, tumor type that has high biomarker prevalence without molecular confirmation of biomarker status, or any tumor type with molecular confirmation of biomarker status; For MTD cohort expansion, only dedifferentiated liposarcoma will be included. * Presence of locally advanced or metastatic disease with at least one measurable lesion. Exclusion criteria: * Age \<18 years. * Eastern Cooperative Oncology Group (ECOG) performance status of \>1. * Life expectancy \<12 weeks. * Unstable brain or leptomeningeal disease based on history and physical examination. * Inadequate organ functions, positive pregnancy test. * Pregnancy or breast-feeding. * Any anti-cancer drug therapy within 2 weeks (8 weeks for mitomycin C or nitrosoureas) or 5 half-lives of the drug prior to study treatment, whichever is shorter, prior to study treatment. * Unwillingness, if not postmenopausal or surgically sterile, to abstain from sexual intercourse or employ an effective barrier or medical method of contraception during the study drug administration and follow-up periods. * Recent (3 months) history of acute pancreatitis. The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Boston **Country:** United States **Facility:** Investigational Site Number 840101 **State:** Massachusetts **Zip:** 02114 **Location 2:** **City:** Boston **Country:** United States **Facility:** Investigational Site Number 840001 **State:** Massachusetts **Zip:** 02115 **Location 3:** **City:** New York **Country:** United States **Facility:** Investigational Site Number 840002 **State:** New York **Zip:** 10021 **Location 4:** **City:** Villejuif **Country:** France **Facility:** Investigational Site Number 250001 **Zip:** 94805 **Location 5:** **City:** Amsterdam **Country:** Netherlands **Facility:** Investigational Site Number 528001 **Zip:** 1066 CX **Location 6:** **City:** Rotterdam **Country:** Netherlands **Facility:** Investigational Site Number 528003 **Zip:** 3075 EA **Location 7:** **City:** Utrecht **Country:** Netherlands **Facility:** Investigational Site Number 528002 **Zip:** 3584 CX #### Overall Officials **Official 1:** **Affiliation:** Sanofi **Name:** Clinical Sciences & Operations **Role:** STUDY_DIRECTOR ### References Module #### References **Citation:** de Jonge M, de Weger VA, Dickson MA, Langenberg M, Le Cesne A, Wagner AJ, Hsu K, Zheng W, Mace S, Tuffal G, Thomas K, Schellens JH. A phase I study of SAR405838, a novel human double minute 2 (HDM2) antagonist, in patients with solid tumours. Eur J Cancer. 2017 May;76:144-151. doi: 10.1016/j.ejca.2017.02.005. Epub 2017 Mar 17. **PMID:** 28324749 ## Derived Section ### Condition Browse Module - Meshes - ID: D000009369 - Term: Neoplasms ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02110979 **Brief Title:** Validation of a Patient Decision Aid for Type 2 Diabetes **Official Title:** Validation of a Patient Decision Aid for Type 2 Diabetes #### Organization Study ID Info **ID:** 70-1036-015 #### Organization **Class:** OTHER **Full Name:** EPI-Q ### Status Module #### Completion Date **Date:** 2015-05 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2015-08-07 **Type:** ESTIMATED **Last Update Submit Date:** 2015-08-05 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2015-05 **Type:** ACTUAL #### Start Date **Date:** 2014-04 **Status Verified Date:** 2015-08 #### Study First Post Date **Date:** 2014-04-10 **Type:** ESTIMATED **Study First Submit Date:** 2014-04-04 **Study First Submit QC Date:** 2014-04-07 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** EPI-Q #### Responsible Party **Investigator Affiliation:** EPI-Q **Investigator Full Name:** Alicia Shillington **Investigator Title:** Executive Vice President **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The objectives of this study are to compare measurements in knowledge of decision options, support for decision making, uncertainly in decision making, and clarity of values important to decision making among two groups of type 2 diabetes patients, those who receive a Patient Decision Aid and those who receive usual care. **Detailed Description:** This study uses a randomized, controlled trial design to compare knowledge of decision options, support for decision making, uncertainly in decision making, and clarity of values important to decision making among patients with uncontrolled type 2 diabetes who are only taking metformin medication. The intervention is a Patient Decision Aid (PDA) video delivered via the internet. ### Conditions Module **Conditions:** - Diabetes Mellitus, Type 2 **Keywords:** - Patient Decision Aid - Shared Decision Making ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE #### Enrollment Info **Count:** 200 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Subjects receive an internet-based patient decision aid video. The PDA is viewed outside of the doctor's office via a personal computer in preparation for regularly scheduled face to face interaction between patients and clinicians. **Intervention Names:** - Behavioral: Use of a PDA to assist shared decision making **Label:** PDA **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Patients receive usual care as determined by their clinician. **Label:** Usual care **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - PDA **Name:** Use of a PDA to assist shared decision making **Other Names:** - Patient Decision Aid **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** The primary endpoint for this investigation is the knowledge total score at final followup 4- 6 weeks after randomization between the PDA versus usual care groups. Questions are about available treatments and specific to the information on medication options available to people with type 2 diabetes when metformin alone does not achieve adequate glycemic control. **Measure:** Knowledge score **Time Frame:** 6 weeks #### Secondary Outcomes **Description:** Measured by using the Decisional Conflict Scale (DCS). Scale will measure domains including: (1) uncertainty in choosing among alternatives; (2) modifiable factors contributing to uncertainty such as feeling uninformed, unclear about values and unsupported in decision making; and (3) perceived quality of decision making. Global decision conflict score and perceived effective decision sub-score will be summarized descriptively following decision and will be compared between arms. Sub-scores are: Uncertainty, Informed, Values clarity, and Support. **Measure:** Decisional conflict **Time Frame:** 6 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Type 2 diabetes 2. Age 18 or older 3. English speaking 4. Currently taking metformin 5. Inadequate glycemic control in the opinion of the investigator 6. Is considering additional medication options on the advice of their physician 7. Can provide a valid email address 8. Access to the internet and able to read and respond to internet questionnaires Exclusion Criteria: 1. Participation in a clinical trial of a diabetes medication within 1 year 2. Currently taking more than two (2) medications for diabetes 3. Has been exposed to diabetes medications from more than three (3) drug classes 4. Adults unable to consent 5. Individuals who are not yet adults (infants, children, teenagers) 6. Pregnant women 7. Prisoners **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Oak Brook **Country:** United States **Facility:** EPI-Q Inc **State:** Illinois **Zip:** 60523 **Location 2:** **City:** Oak Brook **Country:** United States **Facility:** EPI-Q **State:** Illinois **Zip:** 60523 #### Overall Officials **Official 1:** **Affiliation:** EPI-Q **Name:** Alicia Shillington, PhD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Bailey RA, Pfeifer M, Shillington AC, Harshaw Q, Funnell MM, VanWingen J, Col N. Effect of a patient decision aid (PDA) for type 2 diabetes on knowledge, decisional self-efficacy, and decisional conflict. BMC Health Serv Res. 2016 Jan 14;16:10. doi: 10.1186/s12913-016-1262-4. **PMID:** 26762150 ## Derived Section ### Condition Browse Module - Ancestors - 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 ### Condition Browse Module - Browse Leaves - ID: M7115 - Name: Diabetes Mellitus - Relevance: HIGH - As Found: Diabetes - ID: M7119 - Name: Diabetes Mellitus, Type 2 - Relevance: HIGH - As Found: Diabetes Mellitus, Type 2 - 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: D000003920 - Term: Diabetes Mellitus - ID: D000003924 - Term: Diabetes Mellitus, Type 2 ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04366479 **Brief Title:** Endurance Exercise on nrf2 mRNA Expression Gene and VO2max **Official Title:** Effect of Endurance Exercise on nrf2 mRNA Expression Gene and Physical Fitness (VO2max) of Indonesian Hajj Health Officers #### Organization Study ID Info **ID:** HasanuddinU #### Organization **Class:** OTHER **Full Name:** Hasanuddin University ### Status Module #### Completion Date **Date:** 2019-10-08 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2020-04-30 **Type:** ACTUAL **Last Update Submit Date:** 2020-04-28 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2019-10-08 **Type:** ACTUAL #### Start Date **Date:** 2019-05-08 **Type:** ACTUAL **Status Verified Date:** 2020-04 #### Study First Post Date **Date:** 2020-04-29 **Type:** ACTUAL **Study First Submit Date:** 2020-04-17 **Study First Submit QC Date:** 2020-04-24 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER_GOV **Name:** Indonesia-MoH #### Lead Sponsor **Class:** OTHER **Name:** Hasanuddin University #### Responsible Party **Investigator Affiliation:** Hasanuddin University **Investigator Full Name:** Ismail Ahmad **Investigator Title:** Associate Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** Indonesian Hajj Health Officers have the responsibility and very important role in providing health services for Indonesian pilgrims during embarkation and debarkation. One of the main indicators of success is they have excellent physical fitness. This study aims to determine the effectiveness of an intervention program by comparing the results of (intervention) intervention groups, namely the effect of physical exercise interventions (endurance exercise) measured before and after the intervention. This research is an intervention study, and I understand that the process of taking blood can occur with fear, pain due to being pricked by a needle, can faint or be infected. However, previous blood pressure tests, sterile collection techniques and performed by experts, are very unlikely to cause side effects. **Detailed Description:** The investigators invite all Indonesian Hajj Health Officers candidates to participate in research on physical fitness (VO2max) which is an important part of providing Hajj health services in Saudi Arabia while they are on duty. I was involved in the RT-PCR examination and physical fitness test (VO2max) that was carried out before the intervention program and was briefed on the examination protocol before being tested. The consent form was given to me first to be handled. I was informed about the intervention process and the purpose of the research, as well as the confidentiality of the data, collected. And it was also stated that I would be allowed to withdraw from the research at any time without pressure and coercion from any party. The procedure investigators will do is take samples from prospective Indonesian Hajj Health Officers undergoing a series of anamneses and physical examinations by doctors, in the form of personal data, physical activities, general physical examinations including TB, BB, HR, RR, then performed the physical activity eligibility forms. Next, a 1 ml blood sample is taken into a purple test tube containing EDTA to prevent lysis. The blood sample is taken to the HUM-RC Makassar Biomolecular Biology laboratory and stored in a frozen cupboard, after which an initial physical fitness measurement (VO2max) test is carried out using the Multi Fitness Test (MFT) or Bleep Test to PRE TEST at the Health Training Center (Health Training Center) BBPK) Makassar, followed by running exercises intervention as far as 1600 meters 3 times a week duration of 20-30 each training session with a frequency of exercise 16 times 5 weeks according to a predetermined schedule, then conducted a physical fitness test (VO2max) using the same method when PRE TEST to POST TEST I, followed by 1 ml blood sampling at Sudiang Embarkation Hajj Makassar, then 1 week later physical fitness test (VO2max) to POST TEST II without blood sampling is taken again. Participants (n = 30), measured physical fitness level (VO2max) using the Multistage Fitness Test (MFT) Bleep Test (level and feedback) method before being given intervention (pre-exercise), after that given a running intervention 1600 meters duration 20 -30 minutes each training session 3 times a week with a frequency of exercise16 times for 5 weeks, then physical fitness measurements (VO2max) → (post-exercise 1), then intermittent 1-week duration of physical fitness measurements (VO2max) again without intervention and blood sampling (post-exercise 2). Participants only joined in one group will be given intervention for 5 weeks in the form of running 1600 meters duration of 20-30 minutes 3 times a week. It is important for the participants as well as for us to know which training methods are given. this information will be documented in our file, but the investigators will not see these files until after the research is complete. this is the best way the investigators have for testing without being influenced by what the investigators think or expect might happen. The doctor in charge of research will always look after the participants very carefully during the study. If the investigators are worried about what is done, the investigators will find out how the level of physical fitness that participants get and make changes. If there is something the participant is worried about or is disturbing the Participant about the research, please talk to me or one of the other researchers. In this study, the investigators did not use a placebo term, but the investigators still explained to participants in the world of research that the term placebo is known as clinical trials or interventions given to participants that are not genuine but fake. For good research, participants mustn't know whether the participants have been properly measured according to the criteria or not. This is one of the best ways the investigators have to find out what level of physical fitness the investigators are testing. No side effects might occur because the previous sample was very rigorously selected through analysis, physical examination and physical activity feasibility testing. If Participants find that Our interventions are very uncomfortable for participants, the investigators can use other exercise options more comfortably according to the available exercise choices. The possibility of danger, risk, or side effects in this study is very small because the selection of samples is done very closely starting with anamnesis and head-to-toe physical examination and continued with a physical activity feasibility test (PAR-Q). If the physical fitness test occurs when there is suffocation, the researcher has prepared 2 medical staff who accompanied the subjects during the physical fitness test and also in this study the investigators prepared 2 clinics namely the Makassar BBPK Health Clinic which was used during the physical fitness pre-test and the Clinic Musytasyfa Health Makassar Haji Sudiang Dormitory that was used at the time of physical fitness post-test. During the study, the investigators carried out a series of research processes as follows: Pre-exercise * In the first stage: Conducting anamneses and physical examinations by doctors, in the form of personal data, physical activity, general physical examination including Height, Weight, Heart Rate, Respiratory Rate, Blood Pressure (BP) * In the second stage: Completion of physical activity eligibility forms. * In the third step: a 1 ml blood sample is taken into a purple test tube containing EDTA to prevent lysis. The blood samples were taken to the Makassar HUM-RC Biomolecular Biology laboratory and stored in a frozen cupboard. * In the fourth step: Measurement of the initial physical fitness test (VO2max) using the Multi Fitness Test (MFT) or Bleep Test to PRE TEST at the Makassar Health Training Center (BBPK) Endurance Exercise * In the first stage: Selecting an exercise schedule according to the provisions of the study * In the second stage: Exercise choice I: Monday-Wednesday-Friday; Exercise choice II: Tuesday-Thursday-Saturday; Exercise choice III: Wednesday-Friday-Sunday * In the third stage: Document endurance exercises in the form of a video exercise Post Exercise I * In the first stage: Initial physical fitness test (VO2max) measurement using the Multi Fitness Test (MFT) or Bleep Test to POST TEST I at the Haji Sudiang Makassar Dormitory In the second stage: Recovery 1 hour after the physical fitness test In the third stage: Taking 1 ml of blood sample inserted in a purple test tube containing EDTA to prevent lysis. The blood samples were taken to the Makassar HUM-RC Biomolecular Biology laboratory and stored in a frozen cupboard. Post Exercise II Measurement of the initial physical fitness test (VO2max) using the Multi Fitness Test (MFT) or Bleep Test to POST TEST II at the Makassar Sports Hall ### Conditions Module **Conditions:** - Physical Fitness **Keywords:** - Endurance Exercise - Nuclear Respiratory Factor - Physical Fitness (VO2max) ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP **Intervention Model Description:** This type of quasi-experimental research (quasy experiment) with pre-test and post-test research design with only control design. ##### Masking Info **Masking:** NONE **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Count:** 30 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** The study lasted for 5 weeks (1 month 7 days). During the research process, we monitor closely, especially the training schedule, implementation, and evaluation, directly and indirectly. Directly assisting participants to do endurance training activities, not directly monitoring via telephone or WhatsApp. **Intervention Names:** - Behavioral: Multistage Fitness Test **Label:** intervention group **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - intervention group **Description:** Participants only joined in one group will be given intervention for 5 weeks in the form of running 1600 meters duration of 20-30 minutes 3 times a week. It is important for the participants as well as for us to know which training methods are given. this information will be documented in our file, but we will not see these files until after the research is complete. this is the best way we have for testing without being influenced by what we think or expect might happen. **Name:** Multistage Fitness Test **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** Aerobic capacity is also known as maximal oxygen uptake or maximum oxygen consumption (VO2max) **Measure:** VO2max Measurement **Time Frame:** 5 weeks **Description:** RT-PCR analyzes human RNA in the nrf2 RNA sample as the target gene and GAPDH is selected as the housekeeping gene **Measure:** Measurement of mRNA NRF2 Gene Expression **Time Frame:** 5 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Participants from the Nurse profession aged 30 - 39 years * Pass the selection of candidates for Indonesian Hajj Health Officers * Understand the physical training program instructions in the rockport method * Willing to participate in activities and all research provisions Exclusion Criteria: * Had a history of coronary heart disease,asthma * Not approved by the doctor to participate in the study due to a medical condition **Healthy Volunteers:** True **Maximum Age:** 39 Years **Minimum Age:** 30 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Makassar **Country:** Indonesia **Facility:** Makassar Center for Health Training **State:** South Sulawesi **Zip:** 90245 #### Overall Officials **Official 1:** **Affiliation:** Department of Biomolecular Medicine, Hasanuddin University Makassar **Name:** Mochammad Hatta, Profesor **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Vivian H. Heyward. (2016). Aerobic Capacity, Physical Fitness and VO2 Maxium Measurement. Advance Fitness Assessment & Exercise Prescription, 252(805), 2-5. https://www.biopac.com/wp-content/uploads/app252.pdf **Citation:** Lockie RG, Dawes JJ, Moreno MR, Cesario KA, Balfany K, Stierli M, Dulla JM, Orr RM. Relationship Between the 20-m Multistage Fitness Test and 2.4-km Run in Law Enforcement Recruits. J Strength Cond Res. 2021 Oct 1;35(10):2756-2761. doi: 10.1519/JSC.0000000000003217. **PMID:** 31268997 **Citation:** Zhao XQ, Zhang YF, Xia YF, Zhou ZM, Cao YQ. Promoter demethylation of nuclear factor-erythroid 2-related factor 2 gene in drug-resistant colon cancer cells. Oncol Lett. 2015 Sep;10(3):1287-1292. doi: 10.3892/ol.2015.3468. Epub 2015 Jul 8. **PMID:** 26622665 ## Document Section ### Large Document Module #### Large Docs - Date: 2019-05-15 - Filename: Prot_SAP_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: True - Label: Study Protocol and Statistical Analysis Plan - Size: 356479 - Type Abbrev: Prot_SAP - Upload Date: 2020-04-21T05:15 - Date: 2019-10-08 - Filename: ICF_001.pdf - Has ICF: True - Has Protocol: False - Has SAP: False - Label: Informed Consent Form - Size: 440968 - Type Abbrev: ICF - Upload Date: 2020-04-24T17:29 ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01772979 **Acronym:** MITO15 **Brief Title:** Study With Trabectedin in BRCA1 and BRCA2 Mutation Carrier and BRCAness Phenotype Ovarian Cancer **Official Title:** Phase II Study With Trabectedin (Yondelis®) in BRCA1 and BRCA2 Mutation Carrier and BRCAness Phenotype Advanced Ovarian Cancer Patients #### Organization Study ID Info **ID:** MITO15 #### Organization **Class:** OTHER **Full Name:** Catholic University of the Sacred Heart ### Status Module #### Completion Date **Date:** 2014-06 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2013-01-21 **Type:** ESTIMATED **Last Update Submit Date:** 2013-01-17 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2014-06 **Type:** ESTIMATED #### Start Date **Date:** 2011-06 **Status Verified Date:** 2012-12 #### Study First Post Date **Date:** 2013-01-21 **Type:** ESTIMATED **Study First Submit Date:** 2012-11-17 **Study First Submit QC Date:** 2013-01-17 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Catholic University of the Sacred Heart #### Responsible Party **Investigator Affiliation:** Catholic University of the Sacred Heart **Investigator Full Name:** Prof. Giovanni Scambia **Investigator Title:** Professor Giovanni Scambia **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** This is a multicenter phase II study on trabectedin in advanced or recurrent ovarian cancer patients with BRCA mutation and BRCAness phenotype. The purpose of this study is to determine the feasibility in terms of objective response rate by RECIST version 1.1 (Complete and Partial Response \[CR + PR\]) with trabectedin in patients with BRCA1 or BRCA2 mutation carrier or BRCAness phenotype advanced ovarian cancer patients. **Detailed Description:** The main contribution to hereditary ovarian cancer comes from breast cancer (BRCA) genes mutations, which are responsible of 90% of hereditary ovarian cancer. The two susceptibility genes associated with epithelial-type OC are BRCA1 and BRCA2. The BRCA proteins play an important role in the DNA repair mechanisms and are also involved in the control of the cell cycle checkpoints, in protein ubiquitinization and chromatin remodelling. Mutations in the BRCA genes have been extensively described in families affected by breast and/or OC; mutated BRCA1 has been found in up to 75% of families with hereditary OC - Recent data suggest that dysfunction of BRCA1andBRCA2, so-called BRCAness, maybe more prevalent than originally assumed. Both genetic and epigenetic mechanisms can create the BRCAness phenotype in at least a third of all epithelial ovarian cancers. The definition of BRCAness ovarian cancer is: high-grade serous cancers, high initial sensitivity to platinum drugs and retention of platinum-sensitivity through multiple relapses, longer history of disease, longer survival, longer TFIs between relapses. Yondelis® (trabectedin) is proposed to block the transcriptional activation of a subset of inducible genes without affecting their constitutive expression. Trabectedin binds to the minor groove of DNA, bending the helix to the major groove. This binding to DNA triggers a cascade of events affecting several transcription factors, DNA binding proteins, and DNA repair pathways, resulting in perturbation of the cell cycle. Cell cycle studies of the action of trabectedin on tumor cells in vitro reveal that it decreases the rate of progression of the cells through S phase towards G2 and causes a prolonged blockade in G2/M at biologically relevant concentrations (20-80 nM). These cell cycle blocks are p53-independent and lead to a strong apoptopic response. Cells in G1 are more sensitive to the cytotoxic effects of trabectedin. These effects appear to be related to the unique 3-subunit structure, where two of the subunits or rings are involved in binding to the minor groove of DNA in guanine-cytosine rich sequences and alkylation N2 of guanine forming adducts that distorted the DNA helix structure and they are recognized by the TC-NER mechanism. DNA repair proficiency is a major determinant for the cytotoxicity of trabectedin: human cell lines deficient for genes essential for TC-NER activity as XPA, XPB, XPD, XPF, XPG, ERCC1, CSA and CSB are resistant to trabectedin, and this resistance is reverted by complementation of the cells with the corresponding gene. Trabectedin induces double strand breaks and that the BRCA1-/- human cell line HCC1937 and BRCA2Δ22/Δ22 mice cells are more sensitive to trabectedin and this hypersensitivity is reverted by complementation by the BRCA1 or BRCA2 gene. Based in these observations it was hypothesized that the NER machinery trapped in the DNA lesion induced by trabectedin was resolved by the cells producing double strand breaks that were repaired by the HRR machinery, and synergistic action of TC-NER and HRR machinery would be necessary for maximal trabectedin cytotoxicity. ### Conditions Module **Conditions:** - BRCA1 and BRCA2 Mutation Carrier and BRCAness Phenotype **Keywords:** - Trabectedin - BRCA1 and BRCA2 - BRCAness - Ovarian cancer ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 100 **Type:** ESTIMATED **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Trabectedin 1.3 mg/m2 q 21 days Patients will receive trabectedin until disease progression or unacceptable toxicity **Intervention Names:** - Drug: Trabectedin **Label:** Trabectedin **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Trabectedin **Name:** Trabectedin **Other Names:** - Yondelis **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** To evaluate the feasibility (in terms of objective response rate by RECIST version 1.1) of Yondelis treatment in recurrent ovarian cancer population selected for BRCA mutation or BRCAness phenotype. The response rate will be compared with an hystorical control arm of recurrent ovarian cancer patients unselected for BRCA mutation or BRCAness phenotype. **Measure:** objective response **Time Frame:** 24 months #### Secondary Outcomes **Description:** -Duration of response **Measure:** Response **Time Frame:** 36 months **Description:** -Progression-free survival \[the diagnosis of progression will be assessed by radiological criteria; CA 125 increases alone (GCIG criteria of progression) will not be considered as progression of disease without a radiological confirmation of progression\]. **Measure:** Progression-free survival **Time Frame:** 36 months **Description:** Safety profile of trabectedin in this patient population **Measure:** safety profile **Time Frame:** 36 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Patients with partially platinum sensitive ovarian cancer (platinum-free interval 6-12 months) who have previously received at least two platinum based chemotherapy lines, BRCA mutated or with BRCAness phenotype. * Definition of BRCAness phenotype: high-grade serous cancers, great initial sensitivity to platinum drugs and retention of platinum-sensitivity through multiple relapses, long history of disease, long survival, long TFIs between relapses (patients with high personal risk factors will be included after doing the analysis for BRCA 1-2 mutation before knowing the results). * BRCA 1 and/or BRCA 2 mutation carriers (patients with established mutation will be included, patients with high personal risk factors will be included after doing the analysis before knowing the results) 2. Patients with platinum resistant ovarian cancer, BRCA mutated or with BRCAness phenotype who have previously received at least two previous chemotherapy lines (including platinum rechallenge). Definition of platinum resistant: Tumor progression within 6 months of completion of platinum-based therapy (after platinum re-challenge for platinum sensitive recurrence). 3. Patient's written informed consent before any clinical trial-specific procedure. 4. 18 years-of-age or older. 5. Measurable disease as defined in the Response Evaluation Criteria in Solid Tumors (RECIST) Guidelines 6. Eastern Cooperative Oncology Group (ECOG) Performance Status (PS) 0 or 1. 7. Hematologic variables: 1. Hemoglobin ≥9 g/dL 2. Absolute neutrophil count (ANC) ≥1,500/μL, and 3. Platelet count ≥100,000/μL. 8. Serum creatinine ≤ 1.5 mg/dL or creatinine clearance ≥ 30 mL/min 9. Creatinine phosphokinase (CPK) ≤ 2.5 ULN. 10. Hepatic function variables 1. Total bilirubin ≤ ULN. 2. Total alkaline phosphatase ≤ 2.5 ULN 3. AST (serum aspartate transaminase \[SGOT\]) and ALT (serum alanine transaminase \[SGPT\]) must be ≤2.5 x ULN. 11. Albumin ≥ 25 g/l. 12. Adequately recovered from the acute toxicity of any prior treatment. - Exclusion Criteria: * 1. Prior exposure to trabectedin. 2. Known hypersensitivity to any of the components of the trabectedin i.v. formulation or dexamethasone. 3. Less than 2 prior chemotherapy lines given in patients with partially platinum sensitive, BRCA mutated or BRCAness phenotype, ovarian cancer recurrences (including platinum rechallenge). 4. Patients with platinum refractory, BRCA mutated or with BRCAness phenotype, ovarian cancer. 5. Less than 4 weeks from last dose of therapy with any investigational agent, or chemotherapy. 6. History of another neoplastic disease (except basal cell carcinoma or cervical carcinoma in situ adequately treated) unless in remission for 3 years or longer. 7. Known clinically relevant CNS metastases. 8. Other serious illnesses, such as: • Congestive heart failure or angina pectoris; myocardial infarction within 1 year before enrollment; uncontrolled arterial hypertension or arrhythmias * Psychiatric disorder that prevents compliance with protocol * Active viral hepatitis; or chronic liver disease * Active infection * Any other unstable medical conditions **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Name:** Catholic University of Sacred Heart . **Phone:** +39 0630156279 **Role:** CONTACT **Contact 2:** **Name:** Catholic University of Sacred Heart, . **Phone:** +39 0630156279 **Role:** CONTACT #### Locations **Location 1:** **City:** Rome **Contacts:** ***Contact 1:*** - **Name:** Catholic University of Sacred Heart - **Role:** CONTACT **Country:** Italy **Facility:** Catholic University of Sacred Heart **Status:** RECRUITING **Zip:** 00100 #### Overall Officials **Official 1:** **Affiliation:** Catholic University of Sacred Heart **Name:** Giovanni Scambia, Prof **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** National Cancer Institute, Milan **Name:** Domenica Lorusso **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000004701 - Term: Endocrine Gland Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000010049 - Term: Ovarian Diseases - ID: D000000291 - Term: Adnexal 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: D000005833 - Term: Genital Neoplasms, Female - ID: D000014565 - Term: Urogenital Neoplasms - ID: D000091662 - Term: Genital Diseases - ID: D000004700 - Term: Endocrine System Diseases - ID: D000006058 - Term: Gonadal Disorders - ID: D000002277 - Term: Carcinoma - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009370 - Term: Neoplasms by Histologic Type ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: BC19 - Name: Gland and Hormone Related Diseases - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M12974 - Name: Ovarian Neoplasms - Relevance: HIGH - As Found: Ovarian Cancer - ID: M1704 - Name: Carcinoma, Ovarian Epithelial - Relevance: HIGH - As Found: Ovarian Cancer - ID: M5534 - Name: Carcinoma - Relevance: LOW - As Found: Unknown - ID: M7863 - Name: Endocrine Gland Neoplasms - Relevance: LOW - As Found: Unknown - ID: M12972 - Name: Ovarian Diseases - Relevance: LOW - As Found: Unknown - ID: M3643 - Name: Adnexal 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: M8945 - Name: Genital Neoplasms, Female - Relevance: LOW - As Found: Unknown - ID: M17315 - Name: Urogenital Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7862 - Name: Endocrine System Diseases - Relevance: LOW - As Found: Unknown - ID: M9163 - Name: Gonadal Disorders - 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: T4352 - Name: Ovarian Cancer - Relevance: HIGH - As Found: Ovarian Cancer - ID: T4354 - Name: Ovarian Epithelial Cancer - Relevance: HIGH - As Found: Ovarian Cancer ### Condition Browse Module - Meshes - ID: D000010051 - Term: Ovarian Neoplasms - ID: D000077216 - Term: Carcinoma, Ovarian Epithelial ### Intervention Browse Module - Ancestors - ID: D000018906 - Term: Antineoplastic Agents, Alkylating - ID: D000000477 - Term: Alkylating Agents - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000000970 - Term: Antineoplastic Agents ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M1862 - Name: Trabectedin - Relevance: HIGH - As Found: Excluded - ID: M20942 - Name: Antineoplastic Agents, Alkylating - Relevance: LOW - As Found: Unknown - ID: M3820 - Name: Alkylating Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000077606 - Term: Trabectedin ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01692379 **Acronym:** REVASCAT **Brief Title:** Endovascular Revascularization With Solitaire Device Versus Best Medical Therapy in Anterior Circulation Stroke Within 8 Hours **Official Title:** RandomizEd Trial of reVascularizAtion With Solitaire FR® Device Versus Best mediCal Therapy in the Treatment of Acute Stroke Due to anTerior Circulation Large Vessel Occlusion Presenting Within 8 Hours of Symptom Onset #### Organization Study ID Info **ID:** REVASCAT #### Organization **Class:** OTHER **Full Name:** Fundacio Ictus Malaltia Vascular ### Status Module #### Completion Date **Date:** 2015-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2016-02-03 **Type:** ESTIMATED **Last Update Submit Date:** 2016-02-02 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2015-03 **Type:** ACTUAL #### Start Date **Date:** 2012-11 **Status Verified Date:** 2016-02 #### Study First Post Date **Date:** 2012-09-25 **Type:** ESTIMATED **Study First Submit Date:** 2012-09-20 **Study First Submit QC Date:** 2012-09-21 **Why Stopped:** Following DSMB advice after first interim analysis (n=174) ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Medtronic - MITG #### Lead Sponsor **Class:** OTHER **Name:** Fundacio Ictus Malaltia Vascular #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** To evaluate the hypothesis that mechanical embolectomy with the Solitaire FR device is superior to medical management alone in achieving favorable outcome in the distribution of the modified Rankin Scale scores at 90 days in subjects presenting with acute large vessel ischemic stroke \< 8 hours from symptom onset. **Detailed Description:** Prospective, multi-center, randomized, controlled, open, blinded-endpoint trial with a sequential design. The randomization employs a 1:1 ratio of mechanical embolectomy with the CE MARK approved stentriever Solitaire FR® versus medical management alone. Randomization will be done under a minimization process using age, baseline NIHSS, therapeutic window and vessel occlusion site. For the primary endpoint, subjects will be followed for 90 days post-randomization. Interim analysis will be performed as preplanned and interpreted by the Data Safety Monitoring Board (DSMB) after the first 174, 346 and 518 patients representing 25%, 50% and 75% of the planned sample size have completed their 90-day follow-up. The DSMB will advise the executive committee (EC) on recommendations to stop the trial early either for reasons of safety, efficacy, futility or to adjust the sample size. The latter may be necessary as given the paucity of data regarding natural history of the non-treated patients assumptions regarding rates of favorable outcomes in this group of patients may be incorrect. Of note, sample size adjustment based on different than expected outcomes rates is permitted, but it is not permitted to adjust the sample size based on change in the pre-specified treatment effect which is set at 10%. Subject population: Subjects presenting with acute ischemic stroke within 8 hours from symptom onset and whose strokes are attributable to an occlusion of the internal carotid or proximal MCA (M1) arteries. Subjects are either ineligible for IV alteplase or have received IV alteplase therapy without recanalization. ### Conditions Module **Conditions:** - Acute Stroke **Keywords:** - Acute ischemic stroke - endovascular treatment - clinical trial ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 206 **Type:** ACTUAL **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Mechanical embolectomy with Solitaire FR device **Intervention Names:** - Device: Solitaire FR device **Label:** Endovascular treatment **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Medical treatment (standard of care in acute ischemic stroke)including intravenous thrombolysis **Intervention Names:** - Other: Medical treatment **Label:** Medical treatment **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Endovascular treatment **Description:** Mechanical embolectomy in anterior large vessel occlusion **Name:** Solitaire FR device **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - Medical treatment **Description:** Standard of care in acute ischemic stroke including intravenous rTPA **Name:** Medical treatment **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** evaluated by two separate assessors who are blinded to treatment **Measure:** Between-group comparison of the distribution of the modified Rankin Scale scores (shift analysis) **Time Frame:** 90 days after enrollment #### Secondary Outcomes **Measure:** Mortality at 90 days **Time Frame:** 90 days after enrollment **Description:** Deterioration in NIHSS score of ≥4 points within 24 hours from treatment and evidence of intraparenchymal hemorrhage type 2 in the 22 to 36 hours follow-up imaging scans evaluated by independent CT/MR Core Lab and Clinical Events Committee **Measure:** Symptomatic Intracerebral Hemorrhage **Time Frame:** 24h (-2/+12 hours) after enrollment **Description:** Infarct volume evaluated in a second neuroimaging after treatment evaluated by independent CT/MR Core Lab **Measure:** Infarct Volume **Time Frame:** 24h (-2/+12h) post treatment **Description:** Vessel recanalization evaluated by CT angiography or MRA at 24 hours in both treatment groups evaluated by independent CT/MR Core Lab **Measure:** Vessel recanalization **Time Frame:** 24h post treatment **Description:** Procedural related complications in the endovascular treatment arm: arterial perforation, arterial dissection, and embolization in a previously uninvolved vascular territory evaluated by the Angiography Core Lab and the Clinical Events Committee **Measure:** Intraprocedural related complications in endovascular arm **Time Frame:** During endovascular treatment ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * 1. Acute ischemic stroke where patient is ineligible for IV thrombolytic treatment or the treatment is contraindicated (e.g., subject presents beyond recommended time from symptom onset), or where patient has received IV thrombolytic therapy without recanalization after a minimum of 30 min from start of iv tPA infusion * 2. No significant pre-stroke functional disability (mRS ≤ 1) * 3. Baseline NIHSS score obtained prior to randomization must be equal or higher than 6 points * 4. Age ≥18 and ≤ 85. * 5. Occlusion (TICI 0-1) of the intracranial ICA (distal ICA or T occlusions), MCA-M1 segment or tandem proximal ICA/MCA-M1 suitable for endovascular treatment, as evidenced by CTA, MRA or angiogram, with or without concomitant cervical carotid occlusion or stenosis * 6. Patient treatable within eight hours of symptom onset. Symptoms onset is defined as point in time the patient was last seen well (at baseline). Treatment start is defined as groin puncture. * 7. Informed consent obtained from patient or acceptable patient surrogate Exclusion Criteria: * Clinical criteria * 1. Known hemorrhagic diathesis, coagulation factor deficiency, or oral anticoagulant therapy with INR \> 3.0 * 2. Baseline platelet count \< 30.000/µL * 3. Baseline blood glucose of \< 50mg/dL or \>400mg/dl * 4. Severe, sustained hypertension (SBP \> 185 mm Hg or DBP \> 110 mm Hg) * 5. Patients in coma (NIHSS item of consciousness \>1) (Intubated patients for transfer could be randomized only in case an NIHSS is obtained by a neurologist prior transportation). * 6. Seizures at stroke onset which would preclude obtaining a baseline NIHSS * 7. Serious, advanced, or terminal illness with anticipated life expectancy of less than one year. * 8. History of life threatening allergy (more than rash) to contrast medium * 9. Subjects who has received iv t-PA treatment beyond 4,5 hours from the beginning of the symptoms * 10. Renal insufficiency with creatinine ≥ 3 mg/dl * 11. Woman of childbearing potential who is known to be pregnant or lactating or who has a positive pregnancy test on admission. * 12. Subject participating in a study involving an investigational drug or device that would impact this study. * 13. Cerebral vasculitis * 14. Patients with a pre-existing neurological or psychiatric disease that would confound the neurological or functional evaluations, mRS score at baseline must be ≤ 1. This excludes patients who are severely demented, require constant assistance in a nursing home type setting or who live at home but are not fully independent in activities of daily living (toileting, dressing, eating, cooking and preparing meals, etc.) * 15. Unlikely to be available for 90-day follow-up (e.g. no fixed home address, visitor from overseas). Neuroimaging criteria: * 16. Hypodensity on CT or restricted diffusion amounting to an ASPECTS score of \<7 on NCCT or \<6 on DWI MRI. Patients 81 to 85 years old with ASPECTS score on non-contrast CT or DWI MRI \<9 must be excluded. ASPECTS must be evaluated by CBV maps of CT Perfusion, CTA source imaging (CTA-SI) or DWI-MR in patients whose vascular occlusion study (CTA/MRA) confirming qualifying occlusion, is performed beyond 4.5 hours of last seen well. * 17. CT or MR evidence of hemorrhage (the presence of microbleeds is allowed). * 18. Significant mass effect with midline shift. * 19. Evidence of ipsilateral carotid occlusion, high grade stenosis or arterial dissection in the extracranial or petrous segment of the internal carotid artery that cannot be treated or will prevent access to the intracranial clot or excessive tortuosity of cervical vessels precluding device delivery/deployment * 20. Subjects with occlusions in multiple vascular territories (e.g., bilateral anterior circulation, or anterior/posterior circulation) * 21. Evidence of intracranial tumor (except small meningioma). **Maximum Age:** 85 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Badalona **Country:** Spain **Facility:** Hospital Germans Trias i Pujol **State:** Barcelona **Zip:** 08916 **Location 2:** **City:** Hospitalet de Llobregat **Country:** Spain **Facility:** Hospital Universitari Bellvitge **State:** Barcelona **Zip:** 08907 **Location 3:** **City:** Barcelona **Country:** Spain **Facility:** Hospital Universitario Vall D'Hebron **Zip:** 08035 **Location 4:** **City:** Barcelona **Country:** Spain **Facility:** Hospital Clinic **Zip:** 08036 #### Overall Officials **Official 1:** **Affiliation:** Hospital Universitario Germans Trias i Pujol, Barcelona, Spain **Name:** Antoni Dávalos, MD **Role:** STUDY_CHAIR **Official 2:** **Affiliation:** UPMC Stroke Institute, Pittsburgh, PA, USA **Name:** Tudor Jovin, MD **Role:** STUDY_CHAIR **Official 3:** **Affiliation:** Hospital Clinic Barcelona, Barcelona, Spain **Name:** Angel Chamorro, MD **Role:** STUDY_DIRECTOR **Official 4:** **Affiliation:** Hospital Josep Trueta, Girona, Spain **Name:** Joaquin Serena, MD **Role:** STUDY_DIRECTOR **Official 5:** **Affiliation:** Hospital Vall D'Hebron, Barcelona, Spain **Name:** Alex Rovira, MD **Role:** STUDY_DIRECTOR **Official 6:** **Affiliation:** Hospital de Bellvitge, Barcelona, Spain **Name:** Maria A De Miquel, MD **Role:** STUDY_DIRECTOR **Official 7:** **Affiliation:** Hospital Clinic, Barcelona, Spain **Name:** Luis Sanromán, MD **Role:** STUDY_DIRECTOR **Official 8:** **Affiliation:** UPC, Barcelona, Spain **Name:** Erik Cobo, MD **Role:** STUDY_DIRECTOR **Official 9:** **Affiliation:** Hospital Vall D'Hebron, Barcelona, Spain **Name:** Carlos Molina, MD **Role:** STUDY_DIRECTOR ### References Module #### References **Citation:** Molina CA, Chamorro A, Rovira A, de Miquel A, Serena J, Roman LS, Jovin TG, Davalos A, Cobo E. REVASCAT: a randomized trial of revascularization with SOLITAIRE FR device vs. best medical therapy in the treatment of acute stroke due to anterior circulation large vessel occlusion presenting within eight-hours of symptom onset. Int J Stroke. 2015 Jun;10(4):619-26. doi: 10.1111/ijs.12157. Epub 2013 Nov 10. **PMID:** 24206399 **Citation:** Kaneko N, Komuro Y, Yokota H, Tateshima S. Stent retrievers with segmented design improve the efficacy of thrombectomy in tortuous vessels. J Neurointerv Surg. 2019 Feb;11(2):119-122. doi: 10.1136/neurintsurg-2018-014061. Epub 2018 Jul 24. **PMID:** 30045949 **Citation:** Al-Ajlan FS, Al Sultan AS, Minhas P, Assis Z, de Miquel MA, Millan M, San Roman L, Tomassello A, Demchuk AM, Jovin TG, Cuadras P, Davalos A, Goyal M, Menon BK; REVASCAT Investigators. Posttreatment Infarct Volumes when Compared with 24-Hour and 90-Day Clinical Outcomes: Insights from the REVASCAT Randomized Controlled Trial. AJNR Am J Neuroradiol. 2018 Jan;39(1):107-110. doi: 10.3174/ajnr.A5463. Epub 2017 Nov 23. **PMID:** 29170266 **Citation:** Davalos A, Cobo E, Molina CA, Chamorro A, de Miquel MA, Roman LS, Serena J, Lopez-Cancio E, Ribo M, Millan M, Urra X, Cardona P, Tomasello A, Castano C, Blasco J, Aja L, Rubiera M, Gomis M, Renu A, Lara B, Marti-Fabregas J, Jankowitz B, Cerda N, Jovin TG; REVASCAT Trial Investigators. Safety and efficacy of thrombectomy in acute ischaemic stroke (REVASCAT): 1-year follow-up of a randomised open-label trial. Lancet Neurol. 2017 May;16(5):369-376. doi: 10.1016/S1474-4422(17)30047-9. Epub 2017 Mar 16. **PMID:** 28318984 **Citation:** Millan M, Remollo S, Quesada H, Renu A, Tomasello A, Minhas P, Perez de la Ossa N, Rubiera M, Llull L, Cardona P, Al-Ajlan F, Hernandez M, Assis Z, Demchuk AM, Jovin T, Davalos A; REVASCAT Trial Investigators. Vessel Patency at 24 Hours and Its Relationship With Clinical Outcomes and Infarct Volume in REVASCAT Trial (Randomized Trial of Revascularization With Solitaire FR Device Versus Best Medical Therapy in the Treatment of Acute Stroke Due to Anterior Circulation Large Vessel Occlusion Presenting Within Eight Hours of Symptom Onset). Stroke. 2017 Apr;48(4):983-989. doi: 10.1161/STROKEAHA.116.015455. Epub 2017 Mar 14. **PMID:** 28292867 **Citation:** Lopez-Cancio E, Jovin TG, Cobo E, Cerda N, Jimenez M, Gomis M, Hernandez-Perez M, Caceres C, Cardona P, Lara B, Renu A, Llull L, Boned S, Muchada M, Davalos A. Endovascular treatment improves cognition after stroke: A secondary analysis of REVASCAT trial. Neurology. 2017 Jan 17;88(3):245-251. doi: 10.1212/WNL.0000000000003517. Epub 2016 Dec 9. **PMID:** 27940648 **Citation:** Ribo M, Molina CA, Cobo E, Cerda N, Tomasello A, Quesada H, De Miquel MA, Millan M, Castano C, Urra X, Sanroman L, Davalos A, Jovin T; REVASCAT Trial Investigators. Association Between Time to Reperfusion and Outcome Is Primarily Driven by the Time From Imaging to Reperfusion. Stroke. 2016 Apr;47(4):999-1004. doi: 10.1161/STROKEAHA.115.011721. Epub 2016 Mar 8. **PMID:** 26956258 **Citation:** Lopez-Cancio E, Salvat M, Cerda N, Jimenez M, Codas J, Llull L, Boned S, Cano LM, Lara B, Molina C, Cobo E, Davalos A, Jovin TG, Serena J; REVASCAT investigators. Phone and Video-Based Modalities of Central Blinded Adjudication of Modified Rankin Scores in an Endovascular Stroke Trial. Stroke. 2015 Dec;46(12):3405-10. doi: 10.1161/STROKEAHA.115.010909. Epub 2015 Nov 5. **PMID:** 26542697 **Citation:** Urra X, Abilleira S, Dorado L, Ribo M, Cardona P, Millan M, Chamorro A, Molina C, Cobo E, Davalos A, Jovin TG, Gallofre M; Catalan Stroke Code and Reperfusion Consortium. Mechanical Thrombectomy in and Outside the REVASCAT Trial: Insights From a Concurrent Population-Based Stroke Registry. Stroke. 2015 Dec;46(12):3437-42. doi: 10.1161/STROKEAHA.115.011050. Epub 2015 Oct 27. **PMID:** 26508752 **Citation:** Jovin TG, Chamorro A, Cobo E, de Miquel MA, Molina CA, Rovira A, San Roman L, Serena J, Abilleira S, Ribo M, Millan M, Urra X, Cardona P, Lopez-Cancio E, Tomasello A, Castano C, Blasco J, Aja L, Dorado L, Quesada H, Rubiera M, Hernandez-Perez M, Goyal M, Demchuk AM, von Kummer R, Gallofre M, Davalos A; REVASCAT Trial Investigators. Thrombectomy within 8 hours after symptom onset in ischemic stroke. N Engl J Med. 2015 Jun 11;372(24):2296-306. doi: 10.1056/NEJMoa1503780. Epub 2015 Apr 17. **PMID:** 25882510 **Citation:** Abilleira S, Ribera A, Davalos A, Ribo M, Chamorro A, Cardona P, Molina CA, Martinez-Yelamos A, Urra X, Dorado L, Roquer J, Marti-Fabregas J, Aja L, Tomasello A, Castano C, Blasco J, Canovas D, Castellanos M, Krupinski J, Guimaraens L, Perendreu J, Ustrell X, Purroy F, Gomez-Choco M, Baiges JJ, Cocho D, Saura J, Gallofre M; Catalan Stroke Code and Reperfusion Consortium. Functional outcome after primary endovascular therapy or IV thrombolysis alone for stroke. An observational, comparative effectiveness study. Cerebrovasc Dis. 2014;38(5):328-36. doi: 10.1159/000368433. Epub 2014 Nov 21. **PMID:** 25428822 #### See Also Links **Label:** Fundacio Ictus is a non-profit organization sponsoring REVASCAT study **URL:** http://fundacioictus.com/ ## 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: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M10543 - Name: Ischemia - Relevance: LOW - As Found: Unknown - ID: M22306 - Name: Stroke - Relevance: HIGH - As Found: Acute Stroke - ID: M2400 - Name: Ischemic Stroke - Relevance: LOW - As Found: Unknown - 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: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000020521 - Term: Stroke ### 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: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03483779 **Brief Title:** Comparing Ginkgo Biloba Pills and Placebo in the Treatment of Coronary Heart Disease With Impaired Glucose Regulation **Official Title:** A Series of N-of-1 Trials of Comparing Ginkgo Biloba Pills and Placebo in the Treatment of Coronary Heart Disease With Impaired Glucose Regulation #### Organization Study ID Info **ID:** YXY20180226 #### Organization **Class:** OTHER **Full Name:** Xiyuan Hospital of China Academy of Chinese Medical Sciences ### Status Module #### Completion Date **Date:** 2021-07-30 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2018-04-27 **Type:** ACTUAL **Last Update Submit Date:** 2018-04-25 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2020-12-30 **Type:** ESTIMATED #### Start Date **Date:** 2018-04-25 **Type:** ESTIMATED **Status Verified Date:** 2018-02 #### Study First Post Date **Date:** 2018-03-30 **Type:** ACTUAL **Study First Submit Date:** 2018-03-25 **Study First Submit QC Date:** 2018-03-29 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Wanbangde Pharmaceutical Group Co., LTD #### Lead Sponsor **Class:** OTHER **Name:** Xiyuan Hospital of China Academy of Chinese Medical Sciences #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** Background Coronary heart disease has become a serious challenge to China with its high prevalence and mortality. The impaired glucose regulation is prevalent in patients with cardiovascular disease. However, there are few drugs that interfere early with impaired glucose regulation. Ginkgo biloba extract is not only a commonly used drug for cardiovascular diseases, but also has a significant effect in reducing blood sugar. Therefore, this study used a single case randomized controlled trial to explore the efficacy of Ginkgo biloba pills in the treatment of coronary heart disease patients with impaired glucose regulation. Methods This is a randomized, double-blind, placebo-controlled, three-period crossover trial for a single subject.A total of 12 subjects will be recruited in this trial. The trial is divided into three cycles, one cycle has two treatment periods. Ginkgo biloba pills and placebo will be randomized during the treatment period. The test period will be lasted 58 weeks and subjects will take 48 weeks. Subjects will be selected by the researcher strictly in accordance with the inclusion and exclusion criteria. ### Conditions Module **Conditions:** - Randomized Controlled Trial - Ginkgo Biloba Extract ### 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:** 12 **Type:** ESTIMATED **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Five Ginkgo biloba pills a time and three times a day. One treatment period including 8 weeks. **Intervention Names:** - Drug: Ginkgo biloba pills **Label:** Experimental group:Ginkgo biloba pills **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Five placebo pills a time and three times a day. One treatment period including 8 weeks. **Intervention Names:** - Drug: placebo pills **Label:** Control group:placebo pills **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Experimental group:Ginkgo biloba pills **Description:** Each N-of-1 trial will consist of 6 treatment periods, of which 2 treatment periods are in one group, including 8 weeks of placebo treatment and 8 weeks of ginkgo biloba pills. **Name:** Ginkgo biloba pills **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Control group:placebo pills **Description:** Each N-of-1 trial will consist of 6 treatment periods, of which 2 treatment periods are in one group, including 8 weeks of placebo treatment and 8 weeks of ginkgo biloba pills. **Name:** placebo pills **Type:** DRUG ### Outcomes Module #### Other Outcomes **Measure:** HbA1c **Time Frame:** 8 weeks **Measure:** fasting insulin **Time Frame:** 8 weeks **Measure:** lipids **Time Frame:** 8 weeks **Description:** incidence of composite endpoints of major adverse cardiovascular and cerebrovascular events **Measure:** main adverse cardiovascular and cerebrovascular events(MACCE) **Time Frame:** 58 weeks #### Primary Outcomes **Description:** changes in blood glucose **Measure:** fasting plasma glucose (FPG) **Time Frame:** 8 weeks **Description:** changes in blood glucose **Measure:** postprandial 2h blood glucose (2hPG) **Time Frame:** 8 weeks **Description:** Questionnaires will be completed (SAQ - Seattle Angina Questionnaire) at the end of each treatment period. The Seattle Angina Questionnaire (SAQ) is a self-administered, 19-item questionnaire, a cardiac disease-related quality-of-life measure. The SAQ is well validated and sensitive to clinical changes. It has five subscales: physical limitation, angina stability, angina frequency, treatment satisfaction, and quality of life. The possible range of scores for each of the five subscales is 0 to 100, with higher scores indicating better quality of life. **Measure:** Seattle Angina Questionnaire **Time Frame:** 8 weeks #### Secondary Outcomes **Description:** Symptoms of angina pectoris, including frequency, duration, attack severity, and the doses of nitroglycerin were recorded, and an angina pectoris symptom score will be calculated. The effect index is determined according to the following formula: Effect index (n) = \[(symptom score before treatment - symptom score after treatment) / symptom score before treatment\] × 100%. A value of n ≥ 70% suggested a significant effect; 70% \>n ≥ 30% suggested an effect; 30% \>n ≥ 0 suggested no effect; n \< 0 suggested a worsening effect. **Measure:** Symptoms of angina pectoris **Time Frame:** 8 weeks **Measure:** C-reactive protein **Time Frame:** 8 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. male and female patients with clear history of previous myocardial infarction or history of percutaneous coronary intervention（PCI） or history of coronary artery bypass grafting(CABG) (at least 3 months or more),or who have coronary angiography or coronary CT angiography（CTA） results suggested at least one coronary artery stenosis and lumen stenosis ≥50%, 2. in line with the criteria for stable angina, and the number of episodes of angina pectoris ≥ 2 times per week, 3. comply with the diagnostic criteria of blood stasis syndrome of coronary heart disease（CHD）, 4. comply with the 2016 Diabetes Association (ADA) published criteria for impaired diagnosis of glucose regulation， 5. aged between 18 and 75 years， 6. participants voluntarily participated in this study, signed informed consent and had good compliance. Exclusion Criteria: 1. with congenital or rheumatic heart disease or severe cardiopulmonary insufficiency (grade 3 and 4 of cardiac function),or uncontrolled severe arrhythmias (including ventricular tachycardia, supraventricular tachycardia),or not controlled hypertension (systolic blood pressure ≥ 160 mmHg or diastolic blood pressure≥ 100 mmHg), 2. with cerebrovascular disease,or with severe liver and kidney dysfunction, or with endocrine, urinary, blood system and other serious primary diseases, 3. within 4 weeks, there was history of major organ surgery such as head, chest or abdomen or bleeding tendency, 4. those who have taken hypoglycaemic agents or glucocorticoids, thiazide diuretics and other drugs that affect blood sugar levels within 3 months, 5. people with diseases affecting blood glucose metabolism, such as thyroid glands and adrenal diseases, or those with previous history of the aforementioned diseases, 6. allergies or persons allergic to known ingredients of the study drug, 7. pregnancy and lactation women or those with a pregnancy plan, 8. subjects who participated in other clinical trials in the last 3 months, 9. researchers consider that subjects should not participate in clinical trials. **Maximum Age:** 75 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Mingyue Sun, PhD **Phone:** 008601062835652 **Role:** CONTACT #### Locations **Location 1:** **City:** Beijing **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Yue Liu, PhD - **Phone:** +86 13701167815 - **Role:** CONTACT ***Contact 2:*** - **Name:** Rui Gao, PhD - **Role:** PRINCIPAL_INVESTIGATOR ***Contact 3:*** - **Name:** Yue Liu, PhD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** China **Facility:** Xiyuan Hospital **State:** Beijing **Status:** RECRUITING **Zip:** 100091 #### Overall Officials **Official 1:** **Affiliation:** Xiyuan Hospital of China Academy of Chinese Medical Sciences **Name:** Rui Gao, PhD **Role:** STUDY_CHAIR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ### References Module #### References **Citation:** Sun M, Chai L, Lu F, Zhao Y, Li Q, Cui B, Gao R, Liu Y. Efficacy and Safety of Ginkgo Biloba Pills for Coronary Heart Disease with Impaired Glucose Regulation: Study Protocol for a Series of N-of-1 Randomized, Double-Blind, Placebo-Controlled Trials. Evid Based Complement Alternat Med. 2018 Oct 14;2018:7571629. doi: 10.1155/2018/7571629. eCollection 2018. **PMID:** 30405743 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000014652 - Term: Vascular Diseases - ID: D000001161 - Term: Arteriosclerosis - ID: D000001157 - Term: Arterial Occlusive 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: M9419 - Name: Heart Diseases - Relevance: HIGH - As Found: Heart Disease - ID: M6549 - Name: Coronary Disease - Relevance: HIGH - As Found: Coronary Heart Disease - ID: M6546 - Name: Coronary Artery Disease - Relevance: HIGH - As Found: Coronary Heart Disease - ID: M19506 - Name: Myocardial Ischemia - Relevance: HIGH - As Found: Coronary Heart Disease - ID: M10543 - Name: Ischemia - Relevance: LOW - As Found: Unknown - ID: M17400 - Name: Vascular 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 ### Condition Browse Module - Meshes - ID: D000006331 - Term: Heart Diseases - ID: D000003327 - Term: Coronary Disease - ID: D000003324 - Term: Coronary Artery Disease - ID: D000017202 - Term: Myocardial Ischemia ### Intervention Browse Module - Browse Branches - Abbrev: HB - Name: Herbal and Botanical - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: T167 - Name: Ginkgo - Relevance: HIGH - As Found: Bimatoprost ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01580579 **Brief Title:** Predictors of Radiation Pneumonitis in Locally Advanced Lung Cancer Treated With Chemoradiation **Official Title:** Clinical, Biochemical, Dosimetric and Functional Respiratory Predictors of Radiation Pneumonitis in Locally Advanced Lung Cancer (Stages IIIa and IIIb) Treated With Chemotherapy and Radiotherapy #### Organization Study ID Info **ID:** INCANOGAR2012-JA2 #### Organization **Class:** OTHER **Full Name:** Instituto Nacional de Cancerologia de Mexico ### Status Module #### Completion Date **Date:** 2016-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-03-03 **Type:** ACTUAL **Last Update Submit Date:** 2017-03-01 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2015-12 **Type:** ACTUAL #### Start Date **Date:** 2012-07 **Status Verified Date:** 2017-03 #### Study First Post Date **Date:** 2012-04-19 **Type:** ESTIMATED **Study First Submit Date:** 2012-04-17 **Study First Submit QC Date:** 2012-04-18 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** National Council of Science and Technology, Mexico #### Lead Sponsor **Class:** OTHER **Name:** Instituto Nacional de Cancerologia de Mexico #### Responsible Party **Investigator Affiliation:** Instituto Nacional de Cancerologia de Mexico **Investigator Full Name:** Oscar Gerardo Arrieta Rodríguez MD **Investigator Title:** Chief of Thoracic Oncology Department **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Lung cancer \[LC\] is the leading cause of cancer death worldwide. The standard treatment of locally advanced lung cancer unresectable or marginally resectable is combination therapy with radical or preoperative chemoradiation. The local control rates and survival with this treatment modality have increased by more than 30%. Radiotherapy \[RT\] with technical molded 3D \[3D-CRT, Three-Dimensional Conformal Radiation Therapy\] or IMRT \[intensity-modulated radiation therapy\] has allowed that the total dose of radiation has increased which leads to a direct benefit on the results treatment. Between 17-30% of patients are susceptible to pneumonitis due to radiation \[NR\]. This complication may appear at the end of the RT or up to 6 months after the treatment. In severe cases, mortality can reach 50%. It's well known that in various diseases, functional abnormalities precede the clinical manifestations. The degree of pulmonary failure secondary to RT is measured following the standards of the Radiation Therapy Oncology Group who ranks in degrees \[0 to 4\]. Not precisely known factors that influence the development of NR. **Detailed Description:** Objectives: To evaluate the effect of chemotherapy and thoracic radiotherapy on pulmonary function and identify predictors of radiation pneumonitis in locally advanced lung cancer \[stages IIIA and IIIB\]. Hypothesis: Respiratory function tests may predict the development of radiation pneumonitis in patients with locally advanced lung cancer who receive radical treatment with chemoradiation. Methods Prospective cohort study with patients with locally advanced lung cancer of the Lung Cancer Clinic of the National Cancer Institute \[INCAN\]. Patients will receive weekly paclitaxel 50 mg and carboplatin AUC 2 with concomitant radiotherapy 44-63 Gy (22-33 fractions). Followup of lung function tests at baseline, during treatment with radiotherapy and will be carried out on 4 more occasions. ### Conditions Module **Conditions:** - Radiation; Adverse Effect, Pneumonitis **Keywords:** - Radiation pneumonitis - locally advanced lung cancer - Respiratory function tests ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 62 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients with locally advanced lung cancer who are candidates to chemoradiation **Label:** locally advanced lung cancer ### Outcomes Module #### Primary Outcomes **Measure:** Evaluate pulmonary function after chemoradiation treatment in locally advanced NSCLC patients. **Time Frame:** January 2012 to December 2014 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Candidates must have understood and signed informed consent * Histopathological diagnosis of locally advanced lung cancer \[IIIA-cT2N1-2, cT3N1-2, cT4N0, M0o IIIB: cT2N3, cT3N3, cT4N1-3, M0\]. They may also include patients with oligometastatic disease\[M1\] candidates for chemoradiation * Any histology * Medical tests: white blood cell count ≥ plasma 3,000 / mm3, platelets ≥ 100,000 / mm 3, hemoglobin ≥ 12 g / dl, serum creatinine ≤ 1.5 mg / dl, total bilirubin ≤ 1.5, transaminases \[ ≤ 2.5 times the upper limit of normal \[ULN\], alkaline phosphatase \<5 ULN. * Age ≥ 18 years. * General condition score according to ECOG 0 to 2 or a ≥ 60% Karnofsky. * Estimated life expectancy with treatment of at least 24 weeks. Exclusion criteria: * Uncontrolled concurrent diseases. * History of previous radiotherapy to the primary site. * Pregnant or breast-feeding. * Use of anticoagulants in therapeutic doses * Intercurrent Malignancies, except dormant basal cell carcinoma in skin, carcinoma in situ of the cervix * Invasive cancer unless the background was at least 5 years and the disease-free status. **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Patients with locally advanced lung cancer candidates to receive treatment with chemoradiation that will receive treatment in the National Cancer Institute in Mexico City. ### Contacts Locations Module #### Locations **Location 1:** **City:** Mexico city **Country:** Mexico **Facility:** Instituto Nacional de Cancerología de México **Zip:** 14000 #### Overall Officials **Official 1:** **Affiliation:** Instituto de Cancerología **Name:** Oscar Arrieta, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** YES ### References Module #### References **Citation:** Kim M, Lee J, Ha B, Lee R, Lee KJ, Suh HS. Factors predicting radiation pneumonitis in locally advanced non-small cell lung cancer. Radiat Oncol J. 2011 Sep;29(3):181-90. doi: 10.3857/roj.2011.29.3.181. Epub 2011 Sep 30. **PMID:** 22984669 **Citation:** Noble PW, Barkauskas CE, Jiang D. Pulmonary fibrosis: patterns and perpetrators. J Clin Invest. 2012 Aug;122(8):2756-62. doi: 10.1172/JCI60323. Epub 2012 Aug 1. **PMID:** 22850886 **Citation:** Kong FM, Hayman JA, Griffith KA, Kalemkerian GP, Arenberg D, Lyons S, Turrisi A, Lichter A, Fraass B, Eisbruch A, Lawrence TS, Ten Haken RK. Final toxicity results of a radiation-dose escalation study in patients with non-small-cell lung cancer (NSCLC): predictors for radiation pneumonitis and fibrosis. Int J Radiat Oncol Biol Phys. 2006 Jul 15;65(4):1075-86. doi: 10.1016/j.ijrobp.2006.01.051. Epub 2006 May 2. **PMID:** 16647222 **Citation:** Torre-Bouscoulet L, Arroyo-Hernandez M, Martinez-Briseno D, Munoz-Montano WR, Gochicoa-Rangel L, Bacon-Fonseca L, Perez-Padilla R, Vergara E, Garcia-Sancho C, Lozano-Ruiz F, Fernandez-Plata R, Guzman-Barragan A, Arrieta O. Longitudinal Evaluation of Lung Function in Patients With Advanced Non-Small Cell Lung Cancer Treated With Concurrent Chemoradiation Therapy. Int J Radiat Oncol Biol Phys. 2018 Jul 15;101(4):910-918. doi: 10.1016/j.ijrobp.2018.04.014. Epub 2018 Apr 12. **PMID:** 29976503 **Citation:** Torre-Bouscoulet L, Munoz-Montano WR, Martinez-Briseno D, Lozano-Ruiz FJ, Fernandez-Plata R, Beck-Magana JA, Garcia-Sancho C, Guzman-Barragan A, Vergara E, Blake-Cerda M, Gochicoa-Rangel L, Maldonado F, Arroyo-Hernandez M, Arrieta O. Abnormal pulmonary function tests predict the development of radiation-induced pneumonitis in advanced non-small cell lung Cancer. Respir Res. 2018 Apr 24;19(1):72. doi: 10.1186/s12931-018-0775-2. **PMID:** 29690880 ## 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: D000012141 - Term: Respiratory Tract Infections - ID: D000007239 - Term: Infections - ID: D000017563 - Term: Lung Diseases, Interstitial - ID: D000055370 - Term: Lung Injury - ID: D000011832 - Term: Radiation Injuries - ID: D000014947 - Term: Wounds and Injuries ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC01 - Name: Infections - Abbrev: BC26 - Name: Wounds and Injuries - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M11172 - Name: Lung Neoplasms - Relevance: HIGH - As Found: Lung Cancer - ID: M13904 - Name: Pneumonia - Relevance: HIGH - As Found: Pneumonitis - ID: M19814 - Name: Radiation Pneumonitis - Relevance: HIGH - As Found: Radiation Pneumonitis - 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: 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: M19813 - Name: Lung Diseases, Interstitial - Relevance: LOW - As Found: Unknown - ID: M28143 - Name: Lung Injury - Relevance: LOW - As Found: Unknown - ID: M14679 - Name: Radiation Injuries - Relevance: LOW - As Found: Unknown - ID: M17685 - Name: Wounds and Injuries - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000011014 - Term: Pneumonia - ID: D000008175 - Term: Lung Neoplasms - ID: D000017564 - Term: Radiation Pneumonitis ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02018679 **Brief Title:** Er:YAG Ablative Fractional Laser Assisted-Photodynamic Therapy Versus Photodynamic Therapy for Basal Cell Carcinoma **Official Title:** Er:YAG Ablative Fractional Laser Assisted-Photodynamic Therapy Versus Photodynamic Therapy for Nodular Basal Cell Carcinoma in Asian: A Prospective, Randomized Study With 12 Months Follow-up #### Organization Study ID Info **ID:** DAUDerma-02 #### Organization **Class:** OTHER **Full Name:** Dong-A University ### Status Module #### Completion Date **Date:** 2013-09 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2013-12-23 **Type:** ESTIMATED **Last Update Submit Date:** 2013-12-17 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2013-02 **Type:** ACTUAL #### Start Date **Date:** 2011-03 **Status Verified Date:** 2013-12 #### Study First Post Date **Date:** 2013-12-23 **Type:** ESTIMATED **Study First Submit Date:** 2013-11-29 **Study First Submit QC Date:** 2013-12-17 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Dong-A University #### Responsible Party **Investigator Affiliation:** Dong-A University **Investigator Full Name:** Song Ki-Hoon **Investigator Title:** Assistant professor and chairman, Department of dermatology Dong-A University, College of medicine **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Topical photodynamic therapy with methyl-aminolaevulinate (MAL-PDT) has been introduced as an alternatively attractive procedure for BCC. Er:YAG ablative fractional laser (AFL) treatment removes the stratum corneum to increase MAL uptake and may improve efficacy. However, no studies have directly compared the efficacy of Er:YAG AFL-PDT and MAL-PDT in treating nodular BCC in Asians. **Detailed Description:** Basal cell carcinoma (BCC) is the most common cancer in the Caucasian population, with an incidence rising worldwide. there is an increasing trend in the incidence rates of BCC in asian and greater percentage of pigmented BCCs is found to be the most characteristic clinical feature of BCC in Asian compared to BCC in Caucasians. Topical photodynamic therapy with methyl-aminolaevulinate (MAL-PDT) has been introduced as an alternatively attractive procedure for BCC. PDT facilitates the light activation of a photosensitizer in the presence of oxygen. The oxygen generates reactive oxygen species leading to selective and highly localized destruction of abnormal cells. MAL is an efficient photosensitizer as a result of improved lesion penetration attributed to enhanced lipophilicity, decreased charge and also has a greater specificity for neoplastic cells, compared with 5-aminolevulinic acid. Because histologic features of nBCC include down-growth of epithelial buds into the dermis, palisading basal cell and separation of epidermis from the underlying dermis, it is generally treated twice within an interval of 1 week.But, MAL-PDT shows the lower efficacy for the treatment of pigmented BCC because melanin disturbs the absorption of the MAL. Also, a significantly higher proportions of BCC in the Asian population were pigmented BCC compared with pigmented BCC of Caucasian. Consequently, additional techniques are needed to enhance the penetration and accumulation of MAL in order to improve PDT efficacy and decrease treatment duration in darker-skinned patients. Er:YAG ablative fractional laser therapy (AFL) can ablate the epidermis and dermis without significant thermal injury. This approach creates microscopic ablation zones (MAZ) in laser-applied portion of the skin. The tissue with MAZ is surrounded by thin layers of coagulated tissue. Since the Er:YAG AFL resurfaces 5-20% of the skin at one time and does not injure the entire thickness of the epidermis, healing times are minimized. Recent studies have demonstrated that Er:YAG AFL facilitates delivery and uptake of topical MAL deep into the skin, enhancing porphyrin synthesis and photodynamic activation. We have compared the efficacy, recurrence rate, cosmetic outcomes and safety of Er:YAG AFL-PDT with standard MAL-PDT in the treatment of nBCC among Korean populations. ### Conditions Module **Conditions:** - Nodular Basal Cell Carcinoma **Keywords:** - Nodular basal cell carcinoma - Er:YAG - AFL-assisted - MAL-PDT ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** FACTORIAL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 34 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** AFL was performed using a 2940-nm Er:YAG ablative fractional laser (Joule, Sciton Inc., CA, UA) at 550-600 µm ablation in depth, level 1 coagulation, 22% treatment density, and a single pulse. Immediately afterwards, a 1-mm thick layer of MAL (16% Metvix® cream, PhotoCure ASA, Oslo, Norway) was applied to the lesion and to 5 mm of surrounding healthy tissue. The area was covered with an occlusive dressing (Tegaderm, 3M, Saint Paul, MN, US) for 3 hours, after which the remaining cream was removed with saline gauze, and the red fluorescence of porphyrins was visualized with Wood's light. Each treatment area was then separately illuminated with red light-emitting diode (LED) lamps (Aktilite CL128; Galderma, Bruchsal, Germany) with peak emission at 632 nm and total light dose of 37 J cm2. **Intervention Names:** - Procedure: Er:YAG AFL-PDT **Label:** Er:YAG AFL-PDT **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Immediately afterwards, a 1-mm thick layer of MAL (16% Metvix® cream, PhotoCure ASA, Oslo, Norway) was applied to the lesion and to 5 mm of surrounding healthy tissue. The area was covered with an occlusive dressing (Tegaderm, 3M, Saint Paul, MN, US) for 3 hours, after which the remaining cream was removed with saline gauze, and the red fluorescence of porphyrins was visualized with Wood's light. Each treatment area was then separately illuminated with red light-emitting diode (LED) lamps (Aktilite CL128; Galderma, Bruchsal, Germany) with peak emission at 632 nm and total light dose of 37 J cm2. Areas which were scheduled to receive MAL-PDT received the second treatment 7 days later. **Intervention Names:** - Procedure: MAL-PDT **Label:** MAL-PDT **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Er:YAG AFL-PDT **Description:** AFL was performed using a 2940-nm Er:YAG ablative fractional laser (Joule, Sciton Inc., CA, UA) at 550-600 µm ablation in depth, level 1 coagulation, 22% treatment density, and a single pulse. Immediately afterwards, a 1-mm thick layer of MAL (16% Metvix® cream, PhotoCure ASA, Oslo, Norway) was applied to the lesion and to 5 mm of surrounding healthy tissue. The area was covered with an occlusive dressing (Tegaderm, 3M, Saint Paul, MN, US) for 3 hours, after which the remaining cream was removed with saline gauze, and the red fluorescence of porphyrins was visualized with Wood's light. Each treatment area was then separately illuminated with red light-emitting diode (LED) lamps (Aktilite CL128; Galderma, Bruchsal, Germany) with peak emission at 632 nm and total light dose of 37 J cm2. **Name:** Er:YAG AFL-PDT **Type:** PROCEDURE #### Intervention 2 **Arm Group Labels:** - MAL-PDT **Description:** a 1-mm thick layer of MAL (16% Metvix® cream, PhotoCure ASA, Oslo, Norway) was applied to the lesion and to 5 mm of surrounding healthy tissue. The area was covered with an occlusive dressing (Tegaderm, 3M, Saint Paul, MN, US) for 3 hours, after which the remaining cream was removed with saline gauze, and the red fluorescence of porphyrins was visualized with Wood's light. Each treatment area was then separately illuminated with red light-emitting diode (LED) lamps (Aktilite CL128; Galderma, Bruchsal, Germany) with peak emission at 632 nm and total light dose of 37 J cm2. Areas which were scheduled to receive MAL-PDT received the second treatment 7 days later. **Name:** MAL-PDT **Type:** PROCEDURE ### Outcomes Module #### Other Outcomes **Description:** In all cases of complete response, the patients were reviewed at 12 months to check for recurrence. Recurrence was assessed by inspection, dermoscopy, photography, palpation, and histologic findings. **Measure:** Difference of the recurrence rates between Er:YAG AFL-PDT and MAL-PDT **Time Frame:** recurrence rates were evaluated at 12 months after the last treatment. **Description:** Adverse events reported by the patient were noted at each follow-up visit, including severity, duration, and need for additional treatment. The severity of the adverse event was assessed as follows: mild (transient and easily tolerated); moderate (caused the patient discomfort and interrupted usual activities); and severe (caused considerable interference with usual activities and may have been incapacitating or life threatening). All adverse events due to PDT were described as phototoxic reactions (i.e. erythema, postinflammatory hyperpigmentation, edema, itching, oozing, bleeding, etc.). **Measure:** Difference of the safety between Er:YAG AFL-PDT and MAL-PDT **Time Frame:** Safety assessments were performed at the end of the 3-hour cream application; after the illumination during each treatment session; and at 1 week, 3 months, and 12 months after the last treatment #### Primary Outcomes **Description:** Lesion responses were classified as either a complete response (complete disappearance of the lesion) or a non-complete response (incomplete disappearance) **Measure:** Difference the efficacy between Er:YAG AFL-PDT and MAL-PDT **Time Frame:** Efficacy was evaluated at 3 months and 12 months after treatment #### Secondary Outcomes **Description:** I was graded using a 4-point scale: excellent (only slight occurrence of redness or change in pigmentation), good (moderate redness or change in pigmentation), fair (slight to moderate scarring, atrophy, or induration), or poor (extensive scarring, atrophy, or induration) **Measure:** Difference of the cosmetic outcomes between Er:YAG AFL-PDT and MAL-PDT treatment **Time Frame:** Cosmetic outcome was assessed by each investigator for all lesions that achieved a complete response at 3 or 12 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * patient's request for alternative treatment due to the lower cosmetic outcomes of surgery * difficulty to surgical excision due to bleeding abnormalities or cardiac problems Exclusion Criteria: * patients with more than 5 eligible lesions * lesions deeper than 2mm in depth * lesions located in the midface region, nose, orbital areas, and ears * lesions with a longest diameter of less than 6 mm or more than 15mm * infiltrative BCC * morpheaform BCC * known allergies to the MAL cream or lidocaine * pregnancy * lactation * any active systemic infectious disease * immunosuppressive treatment * personal history of malignant melanoma * tendency towards melasma or keloid formation * prior treatment of the lesions within 4 weeks * any indication of poor compliance **Healthy Volunteers:** True **Maximum Age:** 78 Years **Minimum Age:** 38 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Busan **Country:** Korea, Republic of **Facility:** Dong-A University **State:** Seo-gu **Zip:** 602-715 #### Overall Officials **Official 1:** **Affiliation:** Dong-A University **Name:** Ki-Hoon Song, M.D., Ph.D. **Role:** STUDY_CHAIR ## 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 ### Intervention Browse Module - Browse Branches - Abbrev: Derm - Name: Dermatologic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M245552 - Name: Methyl 5-aminolevulinate - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02469779 **Brief Title:** Middle and High School Students' Reactions to a Health Interactive Website: ASPIRE Reactions **Official Title:** Middle and High School Students' Reactions to a Health Interactive Website: ASPIRE Reactions #### Organization Study ID Info **ID:** 2013-0296 #### Organization **Class:** OTHER **Full Name:** M.D. Anderson Cancer Center ### Status Module #### Completion Date **Date:** 2021-02-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-02-08 **Type:** ACTUAL **Last Update Submit Date:** 2021-02-05 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2021-02-01 **Type:** ACTUAL #### Start Date **Date:** 2013-12 **Type:** ACTUAL **Status Verified Date:** 2021-02 #### Study First Post Date **Date:** 2015-06-11 **Type:** ESTIMATED **Study First Submit Date:** 2015-06-09 **Study First Submit QC Date:** 2015-06-10 ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** National Institutes of Health (NIH) **Class:** NIH **Name:** National Cancer Institute (NCI) #### Lead Sponsor **Class:** OTHER **Name:** M.D. Anderson Cancer 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:** The goal of this research study is to learn how middle and high school students react to an interactive website called ASPIRE ( A Smoking Prevention Interactive Experience) and health information online and what their preferences are when using ASPIRE. Researchers hope to use the results of this study to design more effective online health programs to provide middle and high school students with up-to-date information about tobacco use, meditation, and general health and well-being. **Detailed Description:** Participants complete up to 2 separate parts. Part 1: For Part 1, participants complete a survey about their mental and physical health and their opinions concerning health information and the internet. The survey takes about 15-20 minutes to complete. Part 2: If the results of the survey show eligibility, participants complete Part 2 of the study. For Part 2 participants are randomly assigned (as in a roll of dice) to 1 of 2 study groups. Participants have a 1 in 3 chance of being assigned to receive standard care. Participants have a 2 in 3 chance of being assigned to use the ASPIRE website. This is done because no one knows if one study group is better, the same, or worse than the other group. If participant assigned to receive standard care, he or she is shown a website that has the same information presented in ASPIRE, but in text form. Participants in both groups sit in front of a computer and go onto a website. Both the ASPIRE website and the text-based website have been designed for middle and high school students. The ASPIRE website has videos, activities, and health information facts about the effects of smoking, the benefits of meditation, and healthy living. The text-based website only has health information facts about smoking, the benefits of meditation, and healthy living. Participants complete 3 to 4 sessions using either the ASPIRE website or the text-based website throughout their first semester. Each session lasts about 40 minutes. The use of the websites will be videotaped. Follow-Up Surveys: Participants complete a follow-up survey about their opinions concerning health information and the internet immediately after they complete their last session on the website. The follow-up surveys take about 15 minutes to complete. Length of Study: Active participation on this study is over after participants complete the follow-up survey. If participant does not continue onto Part 2 of the study, their active participation is over after they complete Part 1 of the study. Other Information: Participants take part in this study at their school during their after school program. During the use of the website, each study participant is individually videotaped and audio recorded. The videotapes and audio recordings are used to help the research staff analyze each participant's use of the websites. The video tapes and audio recordings are stored on a password-protected encrypted hard drive that is stored in a locked office at MD Anderson. Only the study researcher and authorized members of the research staff are allowed to use the videotapes and audio recordings (for research purposes). ### Conditions Module **Conditions:** - Tobacco Use Cessation - Tobacco Prevention **Keywords:** - Tobacco Prevention - ASPIRE Website - Surveys - Questionnaires - Focus Groups - Health Interactive Website ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 700 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants complete baseline survey. Participants view the ASPIRE website containing videos, activities, and health information facts about the effects of smoking, the benefits of meditation, and healthy living. This viewing will be videotaped and audio recorded. 3 to 4 sessions will be completed. After each website session, survey will be completed. **Intervention Names:** - Behavioral: Surveys - Behavioral: ASPIRE Website - Behavioral: Follow Up Survey **Label:** ASPIRE Group **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants complete baseline survey. Participants view the ASPIRE text-based website containing health information facts about smoking, the benefits of meditation, and healthy living. This viewing will be videotaped and audio recorded. 3 to 4 sessions will be completed. After each website session, survey will be completed. **Intervention Names:** - Behavioral: Surveys - Behavioral: ASPIRE Text-Based Website - Behavioral: Follow Up Survey **Label:** Control Group **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - ASPIRE Group - Control Group **Description:** At baseline participants complete survey about their mental and physical health and their opinions concerning health information and the internet. The survey will take about 15-20 minutes to complete. **Name:** Surveys **Other Names:** - Questionnaires **Type:** BEHAVIORAL #### Intervention 2 **Arm Group Labels:** - ASPIRE Group **Description:** Participants view the ASPIRE website containing videos, activities, and health information facts about the effects of smoking, the benefits of meditation, and healthy living. This viewing will be videotaped **Name:** ASPIRE Website **Type:** BEHAVIORAL #### Intervention 3 **Arm Group Labels:** - Control Group **Description:** Participants view the ASPIRE text-based website containing health information facts about smoking, the benefits of meditation, and healthy living. **Name:** ASPIRE Text-Based Website **Type:** BEHAVIORAL #### Intervention 4 **Arm Group Labels:** - ASPIRE Group - Control Group **Description:** Participants complete a follow-up survey about their opinions concerning health information and the internet immediately after they have completed their last session on the website. **Name:** Follow Up Survey **Other Names:** - Questionnaire **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** This outcome is measured using the validated susceptibility to smoke scale in a Likert scale format and baseline and immediate follow up. **Measure:** Intention to Smoke **Time Frame:** 1 day #### Secondary Outcomes **Description:** Outcome measured using validated Likert scales. In addition, facial expressions of happiness, sadness, disgust, fear, anger, and surprise are measured using a software program of emotion recognition for facial movement. The software captures the extent of expression of each of the emotions on a scale from 0 to 1. **Measure:** Emotional Response **Time Frame:** 4 days **Description:** Participants' reported qualitatively attributes (i.e. features) in ASPIRE that they recall to be emotionally involving. This report conducted using open-ended questions. **Measure:** Participants' Reported Qualitatively Attributes in ASPIRE **Time Frame:** 1 day **Description:** Generation of emotional expression during use of ASPIRE is dependent on data for use of ASPIRE features, and data for emotional expression. Recorded videos of participant's online behavior analyzed via manual content analysis. Recorded videos of online behavior (i.e. mouse movement on the screen, clicking, dragging behaviors, and what the webpage presents) also analyzed using N-vivo. **Measure:** Relationship Between Emotional Involvement During Use of ASPIRE Website and Change in Susceptibility to Smoking **Time Frame:** 1 day ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Ages 11 through 18 (11 and 18 included) 2. High school and Middle School students are both eligible 3. All 150 are nonsmokers. Nonsmokers are defined as individuals who have not smoked, not even part of a cigarette in the past year. 4. All will score above the median on smoking susceptibility 5. 10 will be White/Caucasian participants, 5 will be African American participants, 10 will be Hispanic participants, and 5 will be Asian participants. 6. English speaking Exclusion Criteria: 1)None **Healthy Volunteers:** True **Maximum Age:** 18 Years **Minimum Age:** 11 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Houston **Country:** United States **Facility:** Mt. Calvary Baptist Church **State:** Texas **Zip:** 77008 **Location 2:** **City:** Houston **Country:** United States **Facility:** Young Men's Christian Association (YMCA) **State:** Texas **Zip:** 77253 #### Overall Officials **Official 1:** **Affiliation:** M.D. Anderson Cancer Center **Name:** Alex Prokhorov, MD, PHD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Khalil GE, Wang H, Calabro KS, Mitra N, Shegog R, Prokhorov AV. From the Experience of Interactivity and Entertainment to Lower Intention to Smoke: A Randomized Controlled Trial and Path Analysis of a Web-Based Smoking Prevention Program for Adolescents. J Med Internet Res. 2017 Feb 16;19(2):e44. doi: 10.2196/jmir.7174. **PMID:** 28209560 #### See Also Links **Label:** University of Texas MD Anderson Cancer Center Website **URL:** http://www.mdanderson.org ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06299579 **Brief Title:** GD-11 for Injection in the Treatment of Acute Ischemic Stroke **Official Title:** Phase III Clinical Trial of GD-11 for Injection in the Treatment of Acute Ischemic Stroke #### Organization Study ID Info **ID:** WG-GD11-III-01 #### Organization **Class:** OTHER **Full Name:** Beijing Tiantan Hospital ### Status Module #### Completion Date **Date:** 2025-12-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-03-15 **Type:** ACTUAL **Last Update Submit Date:** 2024-03-12 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2025-02-22 **Type:** ESTIMATED #### Start Date **Date:** 2024-02-29 **Type:** ACTUAL **Status Verified Date:** 2024-03 #### Study First Post Date **Date:** 2024-03-08 **Type:** ACTUAL **Study First Submit Date:** 2024-02-27 **Study First Submit QC Date:** 2024-03-06 ### Sponsor Collaborators Module #### Collaborators **Class:** UNKNOWN **Name:** Jiangsu Wangao Pharmaceutical Co. ltd #### Lead Sponsor **Class:** OTHER **Name:** Beijing Tiantan Hospital #### Responsible Party **Investigator Affiliation:** Beijing Tiantan Hospital **Investigator Full Name:** Yongjun Wang **Investigator Title:** Chief Physician **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Phase III Clinical Trial of GD-11 for Injection in the Treatment of Acute Ischemic Stroke - A Multi-Center, Randomized, Double-Blind, Parallel, Placebo-Controlled Phase III Clinical Study with the primary objective of evaluation of the efficacy and safety of GD-11 for injection in the treatment of acute ischemic stroke patients within 48 hours. The subject has a clinical diagnosis of acute ischemic stroke, within 48 hours from stroke onset to start of study treatment, with a National Institutes of Health Stroke Scale (NIHSS) between 6 and 20, had a total score of upper and lower limbs on motor deficits ≥ 2. The primary outcome is the proportion of subjects with mRS score ≤ 1 at 90 days after treatment. **Detailed Description:** A multicenter, randomized, double-blind, parallel, placebo-controlled trial design was used. Subjects were randomly assigned in a 1:1 ratio to the experimental group (GD-11 for injection treatment group) and the control group (GD-11 for injection placebo group). Randomization stratification factors included onset time (≤24 hours, \>24 hours) and center.Continuous treatment was performed for 10 days (20 times), followed up to 90 days after the first administration. The trial was divided into three phases: screening/baseline phase, treatment phase, and follow-up phase. Screening/baseline phase: Subjects entered the screening/baseline phase after signing the informed consent for screening examinations. Treatment phase: Eligible subjects were randomly assigned in a 1:1 ratio to receive GD-11 for injection or placebo for injection for 10 days (20 times). During the treatment, protocol-required examinations were performed and safety was evaluated. Follow-up phase: Subjects who completed the treatment entered the follow-up phase and were followed up to 90 days after the first administration. Stroke-related scale scores were performed on the 10th, 30th, and 90th days after the first use of the test drug. Adverse events were recorded during the treatment and follow-up phases to further evaluate safety. ### Conditions Module **Conditions:** - Acute Ischemic Stroke **Keywords:** - Subjects within 48 hours ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** This trial is designed as a multicenter, randomized, double-blind, placebo-parallel controlled trial using a multicenter, randomized, double-blind, placebo-parallel controlled trial design. Subjects are randomly assigned in a 1:1 ratio and random number tables are generated using SAS（Statistics Analysis System）software. ##### Masking Info **Masking:** TRIPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 980 **Type:** ESTIMATED **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** GD-11 for injection, freeze-dried powder, 80mg, 160mg/dose Before the test drug is used, from the specification of 100 ml saline infusion bag, use a sterile syringe to extract about 15 ml saline into the test drug, by the oscillator or artificial vibration for about 5min, completely dissolved and then injected back to the administration of the infusion bag with a sterile syringe, gently shaking and mixing, and then intravenously titrated for 30min ± 10min. The first dose should be completed as soon as possible after randomization; the second dose should not be less than 6 hours from the start of the first dose, but not more than 12 + 1h; each subsequent dose interval of 12 ± 1h (calculated using the fixed time of administration as the baseline point and each dose should not be less than 6 hours from the start of the last dose); 10 consecutive days of treatment, a total of 20 times. **Intervention Names:** - Drug: GD-11 for injection test group **Label:** GD-11 for injection test group **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Placebo, freeze-dried powder, 80mg, 160mg/dose Before the test drug is used, from the specification of 100 ml saline infusion bag, use a sterile syringe to extract about 15 ml saline into the test drug, by the oscillator or artificial vibration for about 5min, completely dissolved and then injected back to the administration of the infusion bag with a sterile syringe, gently shaking and mixing, and then intravenously titrated for 30min ± 10min. The first dose should be completed as soon as possible after randomization; the second dose should not be less than 6 hours from the start of the first dose, but not more than 12 + 1h; each subsequent dose interval of 12 ± 1h (calculated using the fixed time of administration as the baseline point and each dose should not be less than 6 hours from the start of the last dose); 10 consecutive days of treatment, a total of 20 times. **Intervention Names:** - Drug: Placebo control group **Label:** Placebo control group **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - GD-11 for injection test group **Description:** The first dose of GD-11 was administered as soon as possible after randomization and then every 12±1 hour. A total of 20 doses were required. **Name:** GD-11 for injection test group **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Placebo control group **Description:** The first dose of placebo was administered as soon as possible after randomization and then every 12±1 hour. A total of 20 doses were required. **Name:** Placebo control group **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Proportion of subjects with mRS score ≤1 on 90th day of treatment **Measure:** Proportion of subjects with mRS score ≤1 on 90th day of treatment **Time Frame:** on 90th day of treatment ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: Only those who meet all of the following items will be enrolled: 1. age ≥18 years and \<81 years, male or female; 2. National Institutes of Health Stroke Scale (NIHSS) score: 6 ≤ NIHSS ≤ 20, and the sum of item 5 Upper Extremity and item 6 Lower Extremity scores ≥ 2 after the onset of this event; 3. The onset of the disease is within 48 hours (including 48 hours); 4. Patients who are diagnosed with ischemic stroke according to the requirements of the latest guidelines such as "Diagnostic Points of Various Major Cerebrovascular Diseases in China 2019" or "Clinical Management Guidelines of Cerebrovascular Diseases in China (2nd edition)", and who have a good healing after the first onset or the last onset (mRS score ≤1 before this onset); 5. Obtaining informed consent approved by the Ethics Committee voluntarily signed by the patients or their legal representatives. Exclusion Criteria: Those who met one of the following items at screening will not be eligible for enrollment: 1. intracranial hemorrhagic disease as seen on cranial imaging: hemorrhagic stroke, epidural hematoma, intracranial hematoma, ventricular hemorrhage, subarachnoid hemorrhage, etc.; if blood seepage only, the suitability for enrollment may be based on the investigator's judgment; 2. severe impairment of consciousness: item score \>1 on the 1a level of consciousness of the NIHSS; 3. thrombolysis, thrombolysis or intervention has been applied or is planned to be applied after this episode; 4. transient ischemic attack (TIA); 5. the patient's blood pressure remains ≥ 220 mmHg systolic or ≥ 120 mmHg diastolic after control; 6. patients with a previous diagnosis of severe mental disorders as well as patients with dementia; 7. patients who have been diagnosed with severe active liver disease, such as acute hepatitis, chronic active hepatitis, cirrhosis, etc.; or ALT (Alanine amino Transferase) or AST (Aspartate amino Transferase) \> 2.0 x ULN (Upper Limit of Normal Value); 8. patients who have been diagnosed with severe active renal disease, renal insufficiency; or serum creatinine \> 1.5 × ULN; 9. after the onset of the disease, the drug with neuroprotective effect has been used in the marketing, such as commercially available edaravone, edaravone dextran edaravone/(+)-2-Decanol injection concentrated solution, nimodipine, gangliosides, cytidine diphosphate, piracetam, oxiracetam, butylphenyl peptide, human urinary kallidinogenase (Urinary Kallidinogenase), cinepazide, murine nerve growth factor, cerebral vivax (hydrolysate of cerebral proteins), calf's blood deprivation of protein injection, calf's blood deprivation of protein extract injection and so on. 10. previously diagnosed with concurrent malignant tumors and undergoing anti-tumor therapy; for subjects diagnosed with malignant tumors after enrollment, continued participation in the study may be based on the investigator's judgment and the subject's wishes; 11. previously diagnosed with a serious systemic disease with an expected survival of \<90 days; 12. the patient is pregnant, breastfeeding and the patient/patient's partner has the potential for pregnancy and plans to become pregnant during the trial period 13. patients with a previously known hypersensitivity to GD-11 for Injection and its excipients; 14. history of major surgery within 4 weeks prior to enrollment that in the investigator's assessment affects neurologic function scores or affects 90-day survival; 15. participation in another clinical study within 30 days prior to randomization or ongoing participation in another clinical study; Not considered by the investigator to be suitable for participation in this clinical study. **Maximum Age:** 81 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Yongjun Wang **Phone:** +86 10 5997 8538 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Shuya Li **Phone:** 13601367028 **Role:** CONTACT #### Locations **Location 1:** **City:** Beijing **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Yongjun Wang - **Role:** CONTACT ***Contact 2:*** - **Name:** Yongjun Wang - **Role:** PRINCIPAL_INVESTIGATOR **Country:** China **Facility:** Beijing Tiantan Hospital Capital Medical University Beijing **State:** Beijing **Status:** NOT_YET_RECRUITING **Zip:** 100000 **Location 2:** **City:** Shangxi **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** hongguo Dai - **Role:** CONTACT **Country:** China **Facility:** Linfen Central Hospital **State:** Linfen City **Status:** RECRUITING **Zip:** 041099 #### Overall Officials **Official 1:** **Affiliation:** IRB of Beijing Tiantan Hospital Capital Medical University Beijing **Name:** Shuya Li **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Motwani JG, Lipworth BJ. Clinical pharmacokinetics of drug administered buccally and sublingually. Clin Pharmacokinet. 1991 Aug;21(2):83-94. doi: 10.2165/00003088-199121020-00001. No abstract available. **PMID:** 1884569 **Citation:** Sato T, Mizuno K, Ishii F. A novel administration route of edaravone--II: mucosal absorption of edaravone from edaravone/hydroxypropyl-beta-cyclodextrin complex solution including L-cysteine and sodium hydrogen sulfite. Pharmacology. 2010;85(2):88-94. doi: 10.1159/000276548. Epub 2010 Jan 21. **PMID:** 20110753 **Citation:** Enlimomab Acute Stroke Trial Investigators. Use of anti-ICAM-1 therapy in ischemic stroke: results of the Enlimomab Acute Stroke Trial. Neurology. 2001 Oct 23;57(8):1428-34. doi: 10.1212/wnl.57.8.1428. **PMID:** 11673584 **Citation:** Cui L, Hung HM, Wang SJ. Modification of sample size in group sequential clinical trials. Biometrics. 1999 Sep;55(3):853-7. doi: 10.1111/j.0006-341x.1999.00853.x. **PMID:** 11315017 **Citation:** Mehta CR, Pocock SJ. Adaptive increase in sample size when interim results are promising: a practical guide with examples. Stat Med. 2011 Dec 10;30(28):3267-84. doi: 10.1002/sim.4102. Epub 2010 Nov 30. **PMID:** 22105690 ## 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 - ID: D000010335 - Term: Pathologic Processes - ID: D000020520 - Term: Brain Infarction - ID: D000002545 - Term: Brain Ischemia - ID: D000007238 - Term: Infarction - ID: D000009336 - Term: Necrosis ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M10543 - Name: Ischemia - Relevance: HIGH - As Found: Ischemic - ID: M22306 - Name: Stroke - Relevance: HIGH - As Found: Stroke - ID: M2400 - Name: Ischemic Stroke - Relevance: HIGH - As Found: Ischemic Stroke - ID: M5793 - Name: Cerebral Infarction - Relevance: HIGH - As Found: Ischemic Stroke - ID: M10282 - Name: Infarction - Relevance: LOW - As Found: Unknown - 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: M22305 - Name: Brain Infarction - Relevance: LOW - As Found: Unknown - ID: M5794 - Name: Brain Ischemia - Relevance: LOW - As Found: Unknown - ID: M12284 - Name: Necrosis - Relevance: LOW - As Found: Unknown - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000020521 - Term: Stroke - ID: D000083242 - Term: Ischemic Stroke - ID: D000002544 - Term: Cerebral Infarction - ID: D000007511 - Term: Ischemia ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01424579 **Brief Title:** Diacutaneous Fibrolysis and Subacromial Syndrome **Official Title:** Effectiveness of Diacutaneous Fibrolysis for the Treatment of Subacromial Impingement Syndrome: a Randomised Controlled Trial #### Organization Study ID Info **ID:** CEIC-P07/22 #### Organization **Class:** OTHER **Full Name:** Fundacio d'Investigacio en Atencio Primaria Jordi Gol i Gurina ### Status Module #### Completion Date **Date:** 2011-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2013-04-05 **Type:** ESTIMATED **Last Update Submit Date:** 2013-04-03 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2011-08 **Type:** ACTUAL #### Start Date **Date:** 2008-02 **Status Verified Date:** 2013-04 #### Study First Post Date **Date:** 2011-08-29 **Type:** ESTIMATED **Study First Submit Date:** 2011-08-24 **Study First Submit QC Date:** 2011-08-26 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Fundacio d'Investigacio en Atencio Primaria Jordi Gol i Gurina #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Subacromial Impingement Syndrome (SIS) is the most common cause of shoulder pain with high lifetime prevalence (one in three) in general population. In occupational population is the most common upper extremity disorder. Symptoms include pain, a variable degree of mobility limitation and a more or less pronounced functional impairment. Conservative treatment is usually the first therapeutic option and some physiotherapeutic techniques have proved its efficacy but nevertheless treatment remains challenging. According to the investigators clinical experience, Diacutaneous Fibrolysis has a beneficial effect on patients suffering from SIS, but no one published clinical trial has evaluated this manual technique previously. The investigators hypothesis is that adding Diacutaneous Fibrolysis to a protocolized physiotherapeutic treatment can provide better outcomes. The investigators objective was to assess the effect of Diacutaneous Fibrolysis on pain, mobility and functional status in patients suffering from SIS. A double-blind (patient and evaluator) randomized clinical trial was carried out in two public centres of Primary Health Care of the Spanish National Health System. The study protocol was approved by the Clinical Research Ethics Committee from the Jordi Gol Institute of Research in Primary Health Care and all the patients provided written consent. A hundred and twenty patients with clinical diagnosis of SIS were included and randomly allocated to one of three groups. All groups received the same daily protocolized treatment based on therapeutic exercises, analgesic electrotherapy and cryotherapy during three weeks. Additionally, intervention group received six sessions (two a week) of actual Diacutaneous Fibrolysis; placebo group received six sessions (two a week) of placebo Diacutaneous Fibrolysis, while control group received only the protocolized treatment. Pain intensity (VAS), active range of motion (flexion, abduction, extension, external and internal rotation) and functional status (Constant-Murley score) were measured in baseline, after the three weeks of treatment and three months after the end of treatment. ### Conditions Module **Conditions:** - Subacromial Impingement Syndrome ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - INVESTIGATOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 120 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** The group received tree weeks of a daily protocolized treatment and additionally six sessions (two a week) of actual Diacutaneous Fibrolysis. **Intervention Names:** - Other: Actual Diacutaneous Fibrolysis - Other: Protocolized physiotherapeutic Treatment **Label:** Actual Diacutaneous Fibrolysis **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** This group received tree weeks of a daily protocolized treatment and additionally six sessions (two a week) of placebo Diacutaneous Fibrolysis. **Intervention Names:** - Other: Placebo Diactuaneous Fibrolysis - Other: Protocolized physiotherapeutic Treatment **Label:** Placebo Diacutaneous Fybrolisis **Type:** PLACEBO_COMPARATOR #### Arm Group 3 **Description:** This group received only tree weeks of a daily protocolized treatment. **Intervention Names:** - Other: Protocolized physiotherapeutic Treatment **Label:** No Diacutaneous Fibrolysis **Type:** OTHER ### Interventions #### Intervention 1 **Arm Group Labels:** - Actual Diacutaneous Fibrolysis **Description:** Diacutaneous Fibrolysis is a non-invasive physiotherapeutic technique applied by means of a set of metallic hooks ending in a spatula with bevelled edges that allow a deeper and more precise application, which could not be achieved manually. Appropriate hook is applied following the intermuscular septum between the muscles with an anatomical or functional relationship with the painful structure, in a centripetal direction towards the pain location, in order to release adherences between musculoskeletal structures. **Name:** Actual Diacutaneous Fibrolysis **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - Placebo Diacutaneous Fybrolisis **Description:** Placebo Diacutaneous Fibrolysis was applied at a superficial level and, instead of fibrolysis, a pinch of skin was held with the thumb of the palpatory hand and the tip of the spatula, without any action taking place on the deep tissular levels. **Name:** Placebo Diactuaneous Fibrolysis **Type:** OTHER #### Intervention 3 **Arm Group Labels:** - Actual Diacutaneous Fibrolysis - No Diacutaneous Fibrolysis - Placebo Diacutaneous Fybrolisis **Description:** Tree weeks of daily therapeutic exercises, analgesic electrotherapy and cryotherapy. **Name:** Protocolized physiotherapeutic Treatment **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Measure instrument: Visual Analogue Scale **Measure:** Changes from Baseline in Pain intensity at 3 weeks and 3 months **Time Frame:** Baseline -3 weeks - 3 months. #### Secondary Outcomes **Description:** Flexion, extension, abduction and external rotation movement were measured with a universal double-armed goniometer, and results are expressed in degrees. For Internal rotation the distance between the position achieved by the tip of the thumb in the hand-behind-back test and the inferior tip of the spinous process of C7 was measured with a flexible metric tape and results are expressed in centimeters. **Measure:** Changes from Baseline in Active Range of Motion at 3 weeks and 3 months **Time Frame:** Baseline - 3 weeks - 3 months **Description:** Measure instrument: Constant-Murley score **Measure:** Changes from Baseline in Functional status at 3 weeks and 3 months **Time Frame:** Baseline - 3 weeks - 3 months ### Eligibility Module **Eligibility Criteria:** Inclusion criteria: * Over 18 years * Diagnosed of Subacromial Impingement Syndrome * Signed a written consent form. Exclusion criteria: * Damaged skin and/or cutaneous lesions in the shoulder area, * A concomitant treatment with platelet antiaggregant agents * Acute inflammatory conditions in the shoulder * Previous shoulder surgery * A pending litigation or court claim **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Cornellà de Llobregat **Country:** Spain **Facility:** ICS Servei de Rehabilitació Sant Ildefons **State:** Barcelona **Zip:** 08940 #### Overall Officials **Official 1:** **Affiliation:** Institut Català de la Salut **Name:** Martín Barra-López, PT **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** Institut Català de la Salut **Name:** Carlos López-de-Celis, DO, PT **Role:** STUDY_CHAIR **Official 3:** **Affiliation:** Servicio Gallego de Salud **Name:** Gabriela Fernández-Jentsch, PT **Role:** STUDY_CHAIR ### References Module #### See Also Links **Label:** Related Info **URL:** http://www.ncbi.nlm.nih.gov/pubmed/23523255 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000004194 - Term: Disease - ID: D000010335 - Term: Pathologic Processes - ID: D000014947 - Term: Wounds and Injuries - ID: D000070599 - Term: Shoulder Injuries - ID: D000007592 - Term: Joint Diseases - ID: D000009140 - Term: Musculoskeletal Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: BC26 - Name: Wounds and Injuries ### Condition Browse Module - Browse Leaves - ID: M16355 - Name: Syndrome - Relevance: HIGH - As Found: Syndrome - ID: M21476 - Name: Shoulder Impingement Syndrome - Relevance: HIGH - As Found: Subacromial Impingement Syndrome - ID: M17685 - Name: Wounds and Injuries - Relevance: LOW - As Found: Unknown - ID: M602 - Name: Shoulder Injuries - 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: D000019534 - Term: Shoulder Impingement Syndrome - ID: D000013577 - Term: Syndrome ### Intervention Browse Module - Browse Branches - Abbrev: Analg - Name: Analgesics - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M4032 - Name: Analgesics - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00242879 **Brief Title:** A Dose Ranging Study Of GW640385 Boosted With Ritonavir (Rtv) In Comparison To A RTV-Boosted Protease Inhibitor (PI) In HIV-1 Infected PI-Experienced Adults **Official Title:** Phase IIB, Randomized, Multicenter, Parallel Group, Study to Evaluate the Safety, Pharmacokinetics and Antiviral Effect of Four Blinded Dosing Regimens of GW640385X/Ritonavir Compared to Open-label Current PI Therapy in HIV-1 Infected, Protease Inhibitor Experienced Adults for 2 Weeks With Long-term Assessment (>48 Weeks) of Safety, Pharmacokinetic and Antiviral Activity of Selected 385/RTV Dosing Regimen(s) vs. a Ritonavir-boosted, Protease Inhibitor Containing Regimen #### Organization Study ID Info **ID:** HPR20001 #### Organization **Class:** INDUSTRY **Full Name:** ViiV Healthcare ### Status Module #### Completion Date **Date:** 2007-06 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-05-30 **Type:** ACTUAL **Last Update Submit Date:** 2017-05-25 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2007-06 **Type:** ACTUAL #### Start Date **Date:** 2005-08 **Status Verified Date:** 2017-05 #### Study First Post Date **Date:** 2005-10-21 **Type:** ESTIMATED **Study First Submit Date:** 2005-10-19 **Study First Submit QC Date:** 2005-10-19 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** ViiV Healthcare #### Responsible Party **Type:** SPONSOR ### Description Module **Brief Summary:** This is a two phase study (randomised and non-randomised phase). The randomised phase will initially examine 4 blinded doses of GW640385 boosted with rtv (with continuation of current background therapy) in comparison to an ongoing, open-labeled rtv-boosted protease inhibitor (PI) regimen for 15 days. At the Day 15 visit, all subjects will optimize background therapy. Additionally, subjects receiving the lowest dose of GW640385 will be re-randomised to one of the higher doses and subjects in the control arm will receive a new rtv-boosted PI based on resistance testing at screening. Subjects will remain in the randomized phase on one of these 4 continuing treatment arms for at least 48 weeks. An interim analysis will occur during the randomised phase to select for a dose of GW640385 to evaluate further in Phase III studies. After dose selection subjects will move to the non-randomised phase of the study. In the non-randomised phase subjects who are receiving GW640385 will be assigned to final selected dose for assessment of long term safety, tolerability, pharmacokinetics, and antiviral activity. **Detailed Description:** A Phase IIB, Randomized, Multicenter, Parallel Group Study to Evaluate the Short-Term Safety, PK and Antiviral Activity of Four Dosing Regimens of GW640385/rtv Therapy Compared to Open-label Current Protease Inhibitor (PI) Therapy in HIV-1, PI-Experienced Adults for 2 wks with Long-Term Evaluation (\>48 wks) of Safety, PK and Antiviral Activity of Selected GW640385/rtv Dosing Regimen(s) vs. a RTV-boosted, PI Containing Regimen ### Conditions Module **Conditions:** - Infection, Human Immunodeficiency Virus I - HIV-1 Infection **Keywords:** - treatment-experienced - RTV - protease inhibitor - ritonavir - GW640385 - HIV-1 ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 130 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Name:** Physician determined comparator PI + ritonavir **Type:** DRUG #### Intervention 2 **Name:** GW640385 + ritonavir **Other Names:** - Physician determined comparator PI + ritonavir **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Time averaged change in plasma HIV-1 RNA over 16 wks **Measure:** Proportion of subjects achieving the target pharmacokinetic (PK) GW640385 drug levels **Measure:** Change in laboratory parameters #### Secondary Outcomes **Measure:** Assessments of HIV viral load changes **Measure:** GW640385 and RTV pharmacokinetic measurements **Measure:** The incidence of adverse events **Measure:** Changes in laboratory measurements **Measure:** ECG measurements **Measure:** HIV viral resistance assessment **Measure:** Immunologic measures ### Eligibility Module **Eligibility Criteria:** Inclusion criteria: * 18+ years of age (or =16 years of age for non-EU countries, according to local requirements). * HIV-1 infected subjects. * Females must be of either non-childbearing potential or have a negative pregnancy test at Screening and agree to use a protocol approved method of contraception. * Plasma HIV-1 RNA (viral load) =1,000 copies/mL at Screening. * Evidence of at least 2 multi-PI resistant mutations at Screening or within 3 months of Screening. * Subjects must have been receiving the same anti-HIV medicines that they are on currently for at least 8 weeks prior to Screening; these anti-HIV medicines will include a single protease inhibitor (PI) in combination with a low dose of ritonavir (i.e., a ritonavir-boosted PI). However, the current PI cannot be tipranavir. * Able to understand and follow protocol requirements, instructions and protocol-stated restrictions. * Be willing and able to provide signed and dated written informed consent prior to study entry. Exclusion criteria: * Subjects cannot change their anti-HIV medicines between Screening and Day 1 Visit. * Subjects can not be receiving dual ritonavir-boosted PIs, non-nucleoside reverse transcriptase inhibitors (NNRTIs) or Tipranavir at Screening. * Active CDC Class C disease at screening. * Pregnant or breastfeeding women. * Protocol-specified laboratory abnormalities at Screening. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Phoenix **Country:** United States **Facility:** GSK Investigational Site **State:** Arizona **Zip:** 85006 **Location 2:** **City:** Bakersfield **Country:** United States **Facility:** GSK Investigational Site **State:** California **Zip:** 93301 **Location 3:** **City:** Fountain Valley **Country:** United States **Facility:** GSK Investigational Site **State:** California **Zip:** 92708 **Location 4:** **City:** Los Angeles **Country:** United States **Facility:** GSK Investigational Site **State:** California **Zip:** 90046 **Location 5:** **City:** San Francisco **Country:** United States **Facility:** GSK Investigational Site **State:** California **Zip:** 94115 **Location 6:** **City:** San Francisco **Country:** United States **Facility:** GSK Investigational Site **State:** California **Zip:** 94121 **Location 7:** **City:** Denver **Country:** United States **Facility:** GSK Investigational Site **State:** Colorado **Zip:** 80204 **Location 8:** **City:** Denver **Country:** United States **Facility:** GSK Investigational Site **State:** Colorado **Zip:** 80220 **Location 9:** **City:** Norwalk **Country:** United States **Facility:** GSK Investigational Site **State:** Connecticut **Zip:** 06851 **Location 10:** **City:** Washington, D.C. **Country:** United States **Facility:** GSK Investigational Site **State:** District of Columbia **Zip:** 20007 **Location 11:** **City:** Washington, D.C. **Country:** United States **Facility:** GSK Investigational Site **State:** District of Columbia **Zip:** 20009 **Location 12:** **City:** Bradenton **Country:** United States **Facility:** GSK Investigational Site **State:** Florida **Zip:** 34205 **Location 13:** **City:** Fort Lauderdale **Country:** United States **Facility:** GSK Investigational Site **State:** Florida **Zip:** 33306 **Location 14:** **City:** Fort Lauderdale **Country:** United States **Facility:** GSK Investigational Site **State:** Florida **Zip:** 33308 **Location 15:** **City:** Fort Lauderdale **Country:** United States **Facility:** GSK Investigational Site **State:** Florida **Zip:** 33316 **Location 16:** **City:** Miami Beach **Country:** United States **Facility:** GSK Investigational Site **State:** Florida **Zip:** 33140 **Location 17:** **City:** Chicago **Country:** United States **Facility:** GSK Investigational Site **State:** Illinois **Zip:** 60612-7230 **Location 18:** **City:** Chicago **Country:** United States **Facility:** GSK Investigational Site **State:** Illinois **Zip:** 60613 **Location 19:** **City:** Chicago **Country:** United States **Facility:** GSK Investigational Site **State:** Illinois **Zip:** 60657 **Location 20:** **City:** Indianapolis **Country:** United States **Facility:** GSK Investigational Site **State:** Indiana **Zip:** 46202 **Location 21:** **City:** Louisville **Country:** United States **Facility:** GSK Investigational Site **State:** Kentucky **Zip:** 40202 **Location 22:** **City:** Baltimore **Country:** United States **Facility:** GSK Investigational Site **State:** Maryland **Zip:** 21201 **Location 23:** **City:** Boston **Country:** United States **Facility:** GSK Investigational Site **State:** Massachusetts **Zip:** 02118 **Location 24:** **City:** Boston **Country:** United States **Facility:** GSK Investigational Site **State:** Massachusetts **Zip:** 02215 **Location 25:** **City:** Las Vegas **Country:** United States **Facility:** GSK Investigational Site **State:** Nevada **Zip:** 89102 **Location 26:** **City:** Newark **Country:** United States **Facility:** GSK Investigational Site **State:** New Jersey **Zip:** 7102 **Location 27:** **City:** Rochester **Country:** United States **Facility:** GSK Investigational Site **State:** New York **Zip:** 14604 **Location 28:** **City:** Greenville **Country:** United States **Facility:** GSK Investigational Site **State:** South Carolina **Zip:** 29605 **Location 29:** **City:** Dallas **Country:** United States **Facility:** GSK Investigational Site **State:** Texas **Zip:** 75246 **Location 30:** **City:** Houston **Country:** United States **Facility:** GSK Investigational Site **State:** Texas **Zip:** 77027 **Location 31:** **City:** Hampton **Country:** United States **Facility:** GSK Investigational Site **State:** Virginia **Zip:** 23666 **Location 32:** **City:** Darlinghurst **Country:** Australia **Facility:** GSK Investigational Site **State:** New South Wales **Zip:** 2010 **Location 33:** **City:** Liverpool **Country:** Australia **Facility:** GSK Investigational Site **State:** New South Wales **Zip:** 2170 **Location 34:** **City:** South Yarra **Country:** Australia **Facility:** GSK Investigational Site **State:** Victoria **Zip:** 3141 **Location 35:** **City:** Bruxelles **Country:** Belgium **Facility:** GSK Investigational Site **Zip:** 1000 **Location 36:** **City:** Vancouver **Country:** Canada **Facility:** GSK Investigational Site **State:** British Columbia **Zip:** V6Z 2C7 **Location 37:** **City:** Toronto **Country:** Canada **Facility:** GSK Investigational Site **State:** Ontario **Zip:** M4N 3M5 **Location 38:** **City:** Toronto **Country:** Canada **Facility:** GSK Investigational Site **State:** Ontario **Zip:** M5B 1L6 **Location 39:** **City:** Montreal **Country:** Canada **Facility:** GSK Investigational Site **State:** Quebec **Zip:** H2L 4P9 **Location 40:** **City:** Montreal **Country:** Canada **Facility:** GSK Investigational Site **State:** Quebec **Zip:** H2X 2P4 **Location 41:** **City:** Sainte-Foy **Country:** Canada **Facility:** GSK Investigational Site **State:** Quebec **Zip:** G1V 4G2 **Location 42:** **City:** Caen **Country:** France **Facility:** GSK Investigational Site **Zip:** 14000 **Location 43:** **City:** La Roche Sur Yon cedex 9 **Country:** France **Facility:** GSK Investigational Site **Zip:** 85025 **Location 44:** **City:** Lyon Cedex 02 **Country:** France **Facility:** GSK Investigational Site **Zip:** 69288 **Location 45:** **City:** Lyon Cedex 03 **Country:** France **Facility:** GSK Investigational Site **Zip:** 69437 **Location 46:** **City:** Nantes **Country:** France **Facility:** GSK Investigational Site **Zip:** 44093 **Location 47:** **City:** Paris Cedex 10 **Country:** France **Facility:** GSK Investigational Site **Zip:** 75475 **Location 48:** **City:** Paris Cedex 12 **Country:** France **Facility:** GSK Investigational Site **Zip:** 75571 **Location 49:** **City:** Muenchen **Country:** Germany **Facility:** GSK Investigational Site **State:** Bayern **Zip:** 80335 **Location 50:** **City:** Frankfurt **Country:** Germany **Facility:** GSK Investigational Site **State:** Hessen **Zip:** 60590 **Location 51:** **City:** Bonn **Country:** Germany **Facility:** GSK Investigational Site **State:** Nordrhein-Westfalen **Zip:** 53127 **Location 52:** **City:** Essen **Country:** Germany **Facility:** GSK Investigational Site **State:** Nordrhein-Westfalen **Zip:** 45122 **Location 53:** **City:** Berlin **Country:** Germany **Facility:** GSK Investigational Site **Zip:** 13353 **Location 54:** **City:** Hamburg **Country:** Germany **Facility:** GSK Investigational Site **Zip:** 20099 **Location 55:** **City:** Ferrara **Country:** Italy **Facility:** GSK Investigational Site **State:** Emilia-Romagna **Zip:** 44100 **Location 56:** **City:** Rimini **Country:** Italy **Facility:** GSK Investigational Site **State:** Emilia-Romagna **Zip:** 47900 **Location 57:** **City:** Milano **Country:** Italy **Facility:** GSK Investigational Site **State:** Lombardia **Zip:** 20127 **Location 58:** **City:** Pavia **Country:** Italy **Facility:** GSK Investigational Site **State:** Lombardia **Zip:** 27100 **Location 59:** **City:** Torino **Country:** Italy **Facility:** GSK Investigational Site **State:** Piemonte **Zip:** 10149 **Location 60:** **City:** Bari **Country:** Italy **Facility:** GSK Investigational Site **State:** Puglia **Zip:** 70124 **Location 61:** **City:** Bagno a Ripoli (FI) **Country:** Italy **Facility:** GSK Investigational Site **State:** Toscana **Zip:** 50126 **Location 62:** **City:** Cascais **Country:** Portugal **Facility:** GSK Investigational Site **Zip:** 2750 **Location 63:** **City:** Lisboa **Country:** Portugal **Facility:** GSK Investigational Site **Zip:** 1150 **Location 64:** **City:** Ponce **Country:** Puerto Rico **Facility:** GSK Investigational Site **Zip:** 00731 **Location 65:** **City:** San Juan **Country:** Puerto Rico **Facility:** GSK Investigational Site **Zip:** 00909-1711 **Location 66:** **City:** Bucharest **Country:** Romania **Facility:** GSK Investigational Site **Zip:** 021105 **Location 67:** **City:** Constanta **Country:** Romania **Facility:** GSK Investigational Site **Zip:** 900709 **Location 68:** **City:** Iasi **Country:** Romania **Facility:** GSK Investigational Site **Zip:** 700116 **Location 69:** **City:** London **Country:** United Kingdom **Facility:** GSK Investigational Site **Zip:** EC1 7BE **Location 70:** **City:** London **Country:** United Kingdom **Facility:** GSK Investigational Site **Zip:** SW10 9TH #### Overall Officials **Official 1:** **Affiliation:** GlaxoSmithKline **Name:** GSK Clinical Trials, MD **Role:** STUDY_DIRECTOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020969 - Term: Disease Attributes - ID: D000010335 - Term: Pathologic Processes - ID: D000007153 - Term: Immunologic Deficiency Syndromes - ID: D000007154 - Term: Immune System Diseases - ID: D000086982 - Term: Blood-Borne Infections - ID: D000015229 - Term: Sexually Transmitted Diseases, Viral - ID: D000012749 - Term: Sexually Transmitted Diseases - ID: D000016180 - Term: Lentivirus Infections - ID: D000012192 - Term: Retroviridae Infections - ID: D000012327 - Term: RNA Virus Infections - ID: D000014777 - Term: Virus Diseases - ID: D000012897 - Term: Slow Virus Diseases - ID: D000091662 - Term: Genital Diseases - ID: D000091642 - Term: Urogenital Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC01 - Name: Infections - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: BC20 - Name: Immune System Diseases ### Condition Browse Module - Browse Leaves - ID: M10283 - Name: Infections - Relevance: HIGH - As Found: Infection - ID: M6368 - Name: Communicable Diseases - Relevance: HIGH - As Found: Infection - ID: M3522 - Name: Acquired Immunodeficiency Syndrome - Relevance: HIGH - As Found: Human Immunodeficiency Virus - ID: M18250 - Name: HIV Infections - Relevance: HIGH - As Found: Human Immunodeficiency Virus - ID: M17522 - Name: Virus Diseases - Relevance: LOW - As Found: Unknown - ID: M10199 - Name: Immunologic Deficiency Syndromes - Relevance: LOW - As Found: Unknown - ID: M16355 - Name: Syndrome - Relevance: LOW - As Found: Unknown - ID: M22700 - Name: Disease Attributes - Relevance: LOW - As Found: Unknown - ID: M10200 - Name: Immune System Diseases - Relevance: LOW - As Found: Unknown - ID: M2593 - Name: Blood-Borne Infections - Relevance: LOW - As Found: Unknown - ID: M15558 - Name: Sexually Transmitted Diseases - Relevance: LOW - As Found: Unknown - ID: M17933 - Name: Sexually Transmitted Diseases, Viral - Relevance: LOW - As Found: Unknown - ID: M18640 - Name: Lentivirus Infections - Relevance: LOW - As Found: Unknown - ID: M15026 - Name: Retroviridae Infections - Relevance: LOW - As Found: Unknown - ID: M15149 - Name: RNA Virus Infections - Relevance: LOW - As Found: Unknown - ID: M15700 - Name: Slow Virus Diseases - 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 ### Condition Browse Module - Meshes - ID: D000007239 - Term: Infections - ID: D000003141 - Term: Communicable Diseases - ID: D000000163 - Term: Acquired Immunodeficiency Syndrome - ID: D000015658 - Term: HIV Infections ### Intervention Browse Module - Ancestors - ID: D000017320 - Term: HIV Protease Inhibitors - ID: D000084762 - Term: Viral Protease Inhibitors - ID: D000011480 - Term: Protease Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000019380 - Term: Anti-HIV Agents - ID: D000044966 - Term: Anti-Retroviral Agents - ID: D000000998 - Term: Antiviral Agents - ID: D000000890 - Term: Anti-Infective Agents - ID: D000065692 - Term: Cytochrome P-450 CYP3A Inhibitors - ID: D000065607 - Term: Cytochrome P-450 Enzyme Inhibitors ### Intervention Browse Module - Browse Branches - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M21394 - Name: Ritonavir - Relevance: HIGH - As Found: Left - ID: M4314 - Name: Antiviral Agents - Relevance: LOW - As Found: Unknown - ID: M19609 - Name: HIV Protease Inhibitors - Relevance: LOW - As Found: Unknown - ID: M14343 - Name: Protease Inhibitors - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown - ID: M21350 - Name: Anti-HIV Agents - Relevance: LOW - As Found: Unknown - ID: M25428 - Name: Anti-Retroviral Agents - Relevance: LOW - As Found: Unknown - ID: M4214 - Name: Anti-Infective Agents - Relevance: LOW - As Found: Unknown - ID: M30564 - Name: Cytochrome P-450 CYP3A Inhibitors - Relevance: LOW - As Found: Unknown - ID: M30537 - Name: Cytochrome P-450 Enzyme Inhibitors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000019438 - Term: Ritonavir ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03963479 **Brief Title:** Vitamin B6 and Magnesium- A Clinical Trial on ASD Patients **Official Title:** Vitamin B6 and Magnesium on Neurobehavioral Status of Autism Spectrum Disorder: A Randomized, Double-Blind, Placebo Controlled Study #### Organization Study ID Info **ID:** BSMMU/2018/14607 #### Organization **Class:** OTHER **Full Name:** Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh ### Status Module #### Completion Date **Date:** 2019-07-31 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2019-07-31 **Type:** ACTUAL **Last Update Submit Date:** 2019-07-30 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2019-07-31 **Type:** ESTIMATED #### Start Date **Date:** 2019-01-01 **Type:** ACTUAL **Status Verified Date:** 2019-05 #### Study First Post Date **Date:** 2019-05-24 **Type:** ACTUAL **Study First Submit Date:** 2019-05-15 **Study First Submit QC Date:** 2019-05-23 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh #### Responsible Party **Investigator Affiliation:** Bangabandhu Sheikh Mujib Medical University, Dhaka, Bangladesh **Investigator Full Name:** Dr. Farhana Khan **Investigator Title:** Resident **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** To determine whether Vitamin B6 and Magnesium improve neurobehavioral status in terms of General observation, Cognitive working, Socialization, Communication and Sensory Dysfunction in patient with Autism Spectrum Disorder. ### Conditions Module **Conditions:** - Autism Spectrum Disorder ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** TRIPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - OUTCOMES_ASSESSOR **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Count:** 50 **Type:** ESTIMATED **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Magnesium (50mg) for ages 1-3 years (100mg) for ages 4-8 years (200mg) for ages 9-12 years Vitamin B6 (25mg) for ages 1-3 years (50mg) for ages 4-8 years (100mg) for ages 9-12years **Intervention Names:** - Combination Product: Magnesium and Vitamin B6 **Label:** Magnesium-Vitamin B6 **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Magnesium (50mg) for ages 1-3 years (100mg) for ages 4-8 years (200mg) for ages 9-12 years Vitamin B6 (25mg) for ages 1-3 years (50mg) for ages 4-8 years (100mg) for ages 9-12years **Intervention Names:** - Combination Product: Magnesium and Vitamin B6 **Label:** Placebo **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Magnesium-Vitamin B6 - Placebo **Description:** Magnesium and Vitamin B6 will be given to the enrolled patients for 3 months **Name:** Magnesium and Vitamin B6 **Type:** COMBINATION_PRODUCT ### Outcomes Module #### Primary Outcomes **Description:** General observation, Cognitive Working, socialization, Communication, Sensory Dysfunction. At first a autism will be diagnosed by DSM-5 .Then the above mention criterias along with the severity will be diagnosed by Autism Diagnostic Checklist tool by a psychologist. **Measure:** Autism Spectrum Disorder **Time Frame:** 3 months #### Secondary Outcomes **Description:** The Autism Diagnostic Checklist will be repeated to compare the outcomes in terms of General observation, Cognitive Working, socialization, Communication, Sensory along with the severity of the Autism. **Measure:** Autism Spectrum Disorder **Time Frame:** 0 month ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Children between 2 to 12 years of age suspected ASD assessed by DSM-5 and ADCL tool 2. Each patient will be free of psychoactive medication for atleast 3 months prior to the entry into the trial 3. Newly diagnosed patient 4. Co-morbid neurological disorder like hyperactivity Exclusion Criteria: 1. Patients with chronic diseases or any known metabolic or hormonal diseases 2. Patients with any known chromosomal or genetic syndromes 3. Patients unable to give informed consent 4. Patients unable to travel to clinical visits or non co-operative **Maximum Age:** 12 Years **Minimum Age:** 2 Years **Sex:** ALL **Standard Ages:** - CHILD ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Farhana Khan, MBBS **Phone:** 8801674255083 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Md Sayedur Rahman, MBBS, MPhil **Phone:** 8801971840757 **Role:** CONTACT #### Locations **Location 1:** **City:** Dhaka **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Md. Sayedur Rahman, MBBS, MPhil - **Phone:** 8801971840757 - **Role:** CONTACT **Country:** Bangladesh **Facility:** Farhana Khan **Status:** RECRUITING **Zip:** 02 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000002659 - Term: Child Development Disorders, Pervasive - ID: D000065886 - Term: Neurodevelopmental 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: M4623 - Name: Autistic Disorder - Relevance: LOW - As Found: Unknown - ID: M206 - Name: Autism Spectrum Disorder - Relevance: HIGH - As Found: Autism Spectrum Disorder - ID: M5903 - Name: Child Development Disorders, Pervasive - Relevance: LOW - As Found: Unknown - ID: M5902 - Name: Developmental Disabilities - Relevance: LOW - As Found: Unknown - ID: M30644 - Name: Neurodevelopmental 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: D000067877 - Term: Autism Spectrum Disorder ### Intervention Browse Module - Ancestors - ID: D000014815 - Term: Vitamins - ID: D000018977 - Term: Micronutrients - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000014803 - Term: Vitamin B Complex ### Intervention Browse Module - Browse Branches - Abbrev: Micro - Name: Micronutrients - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Hemat - Name: Hematinics - Abbrev: Vi - Name: Vitamins ### Intervention Browse Module - Browse Leaves - ID: M23026 - Name: Vitamin B 6 - Relevance: HIGH - As Found: Health Outcomes - ID: M14583 - Name: Pyridoxal - Relevance: HIGH - As Found: Health Outcomes - ID: M14589 - Name: Pyridoxine - Relevance: HIGH - As Found: Health Outcomes - ID: M17558 - Name: Vitamins - Relevance: LOW - As Found: Unknown - ID: M17546 - Name: Vitamin B Complex - Relevance: LOW - As Found: Unknown - ID: M21009 - Name: Micronutrients - Relevance: LOW - As Found: Unknown - ID: M16885 - Name: Trace Elements - Relevance: LOW - As Found: Unknown - ID: M8618 - Name: Folic Acid - Relevance: LOW - As Found: Unknown - ID: T474 - Name: Vitamin B6 - Relevance: HIGH - As Found: Health Outcomes - ID: T459 - Name: Pyridoxal - Relevance: HIGH - As Found: Health Outcomes - ID: T461 - Name: Pyridoxine - Relevance: HIGH - As Found: Health Outcomes - 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 ### Intervention Browse Module - Meshes - ID: D000025101 - Term: Vitamin B 6 - ID: D000011730 - Term: Pyridoxal - ID: D000011736 - Term: Pyridoxine ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02101879 **Brief Title:** Cardiotoxicity in Metastatic Her 2 Positive Patients Treated With Trastuzumab ,Pertuzumab and Taxanes **Official Title:** Cardiotoxicity in Metastatic Her 2 Positive Patients Treated With First Line Trastuzumab, Pertuzumab and Taxanes Based Regimen #### Organization Study ID Info **ID:** Cardiotoxicity #### Organization **Class:** OTHER **Full Name:** Rambam Health Care Campus ### Status Module #### Completion Date **Date:** 2016-08 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2014-05-02 **Type:** ESTIMATED **Last Update Submit Date:** 2014-05-01 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2016-03 **Type:** ESTIMATED #### Start Date **Date:** 2014-05 **Status Verified Date:** 2014-05 #### Study First Post Date **Date:** 2014-04-02 **Type:** ESTIMATED **Study First Submit Date:** 2014-03-05 **Study First Submit QC Date:** 2014-03-30 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Rambam Health Care Campus #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Approximately 15-25% of all breast cancers are human epidermal growth factor receptor 2 (HER2) positive and it has been well known that HER2 overexpression is associated with more aggressive phenotype and poor prognosis with resistance to certain chemotherapeutic agents. Trastuzumab administration as an adjuvant and in metastatic HER2 positive breast cancer is associated with both symptomatic and asymptomatic cardiotoxicity. The incidence of trastuzumab-mediated cardiotoxicity were 27% with antracycline combination and 13% when it was administered with paclitaxel . Pertuzumab, a recombinant humanized monoclonal antibody binding to the HER2 dimerization domain, prevents dimerization of HER2 with other HER receptors (HER3,HER1, and HER4) especially with HER3. Blocking HER2-HER3 dimerization is postulated to be the most clinically relevant action of pertuzumab and this can effectively block her2-mediated cell signaling. Pertuzumab is indicated in combination with trastuzumab and docetaxel for the treatment of patients with HER2-positive metastatic breast cancer who have not received prior anti-HER2 therapy or chemotherapy for metastatic disease. Treatment of breast cancer with pertuzumab plus trastuzumab plus docetaxel as first line treatment until disease progression might be complicated by cardiotoxicity in up to 14.5% of the Patients. Cardinale et al showed that troponin I (TNI) positive identifies trastuzumab-treated patients who are at risk for cardiotoxicity and are unlikely to recover from cardiac dysfunction despite HF therapy. There is very little data about the reversibility and identification of patients at risk for cardiotoxicity of the pertuzumab plus trastuzumab plus docetaxel regimen and of those who will not recover from cardiac dysfunction,this information is crucial. The usefulness of troponin I (TNI) and Brain natriuretic peptide (BNP) in the identification of patients at risk for PT cardiotoxicity and in the prediction of LVEF recovery has never been investigated. based on this background , this study aim is to evaluate the cardiotoxicity of pertuzumab plus trastuzumab plus docetaxel regimen and the application of troponin I (TNI) and Brain natriuretic peptide (BNP) in this setting. **Detailed Description:** Breast cancer is the leading cancer in women in Israel with 4000 new cases every year. Approximately 15-25% of all breast cancers are human epidermal growth factor receptor 2 (HER2) positive and it has been well known that HER2 overexpression is associated with more aggressive phenotype and poor prognosis with resistance to certain chemotherapeutic agents. Trastuzumab, an anti-HER2 humanized monoclonal antibody, is the standard treatment for both early and metastatic HER2-positive breast cancer. In addition to other chemotherapeutic agents, trastuzumab significantly improves response rate and survival in HER2-positive early and metastatic breast cancer. Although it is well known that trastuzumab therapy is closely associated with both symptomatic and asymptomatic cardiotoxicity. Addition of trastuzumab to chemotherapy significantly improved response rate, time to disease progression and reduction of death compared to chemotherapy alone in HER2-positive metastatic breast cancer (MBC). Thus, anti-HER2 treatment is a standard therapeutic approach for HER2-positive MBC patients with visceral crisis. Trastuzumab administration as an adjuvant and in metastatic HER2 positive breast cancer is associated with both symptomatic and asymptomatic cardiotoxicity. The incidence of trastuzumab-mediated cardiotoxicity were 27% with antracycline combination and 13% when it was administered with paclitaxel . Pertuzumab, a recombinant humanized monoclonal antibody binding to the HER2 dimerization domain, prevents dimerization of HER2 with other HER receptors (HER3,HER1, and HER4) especially with HER3. Pertuzumab differs from trastuzumab in the epitope binding regions of the light chain; pertuzumab binds to domain 2 ofHER2 essential for dimerization, whereas trastuzumab binds to domain 4 of HER2. Blocking HER2-HER3 dimerization is postulated to be the most clinically relevant action of pertuzumab and this can effectively block her2-mediated cell signaling. Pertuzumab is indicated in combination with trastuzumab and docetaxel for the treatment of patients with HER2-positive metastatic breast cancer who have not received prior anti-HER2 therapy or chemotherapy for metastatic disease. The randomized, double-blind, placebo controlled,phase III CLEOPATRA (the CLinical Evaluation OfPertuzumab And Trastuzumab) study involved 808 patients with HER2-positive MBC who had not received prior chemotherapy or biologic therapy, who were randomized to placebo plus trastuzumab plus docetaxel or pertuzumab plus trastuzumab plus docetaxel as first line treatment until disease progression. No additional cardiotoxicity was observed when adding pertuzumab compared to placebo. The incidence of any cardiac disorder as assessed by the investigators was similar in the placebo (16.4%) and pertuzumab (14.5%) arms. Left ventricular systolic dysfunction (LVSD) was observed in 8.3% and 4.4% of placebo and pertuzumab patients, respectively. Left ventricular systolic dysfunction of grade III or higher was reported in 7 (1.8%) patients in the placebo arm and 4 (1.0%) in the pertuzumab arm. Treatment of breast cancer with pertuzumab plus trastuzumab plus docetaxel as first line treatment until disease progression might be complicated by cardiotoxicity in up to 14.5% of the Patients. Cardinale et al showed that troponin I (TNI) positive identifies trastuzumab-treated patients who are at risk for cardiotoxicity and are unlikely to recover from cardiac dysfunction despite HF therapy. There is very little data about the reversibility and identification of patients at risk for cardiotoxicity of the pertuzumab plus trastuzumab plus docetaxel regimen and of those who will not recover from cardiac dysfunction,this information is crucial. The usefulness of troponin I (TNI) and Brain natriuretic peptide (BNP) in the identification of patients at risk for PT cardiotoxicity and in the prediction of LVEF recovery has never been investigated. based on this background , this study aim is to evaluate the cardiotoxicity of pertuzumab plus trastuzumab plus docetaxel regimen and the application of troponin I (TNI) and Brain natriuretic peptide (BNP) in this setting. ### Conditions Module **Conditions:** - Cardiotoxicity. - Anti Her2 Therapy. - Metastatic Breast Cancer ### Design Module #### Bio Spec **Description:** Blood samples for TNI \& BNP will be sent before every cycle for the first 5 cycles of treatment **Retention:** SAMPLES_WITHOUT_DNA #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 20 **Type:** ESTIMATED **Patient Registry:** True **Study Type:** OBSERVATIONAL **Target Duration:** 12 Months ### Arms Interventions Module #### Arm Group 1 **Description:** Trastuzumab \& Pertuzumab \& Taxanes **Label:** Breast cancer Her2 positive ### Outcomes Module #### Primary Outcomes **Description:** Prior to every treatment cycle, blood samples will be taken for TNI \& BNP. Patients with elevated levels will be sent for LEVF evaluation. In cases with LEVF reduction of 15% or more from the baseline or LEVF less than 50% will be sent for Cardiological consult in order to consider ACEI or BB treatment **Measure:** To assesses blood levels of TNI and BNP during the first four cycles Trastuzumab&Pertuzumab and Taxanes treatment **Time Frame:** The patients will be followed until the end of therapy (an expected average of 18 months). **Description:** Prior to every treatment cycle, blood samples will be taken for TNI \& BNP. Patients with elevated levels will be sent for LEVF evaluation. In cases with LEVF reduction of 15% or more from the baseline or LEVF less than 50% will be sent for Cardiological consult in order to consider ACEI or BB treatment **Measure:** To evaluate the correlation between elevated TNI&BNP and decline of LVEF on echocardiography until end of treatment. **Time Frame:** The patients will be followed until the end of therapy (an expected average of 18 months). ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Metastatic Breast Cancer patients with Her2 Positive Disease. * No prior treatment Exclusion Criteria: * LEVF less than 50% **Minimum Age:** 18 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Patients with Metastatic Breast Cancer Her2 Positive ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Georgeta Fried, MD **Phone:** +972-4-854-3018 **Role:** CONTACT #### Locations **Location 1:** **City:** Haifa **Contacts:** ***Contact 1:*** - **Name:** Georgeta Fried, MD - **Role:** PRINCIPAL_INVESTIGATOR ***Contact 2:*** - **Name:** Shlomit Shachar-Strulov, MD - **Role:** SUB_INVESTIGATOR **Country:** Israel **Facility:** Rambam MC **Status:** RECRUITING ## Derived Section ### Condition Browse Module - Ancestors - ID: D000006331 - Term: Heart Diseases - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000010335 - Term: Pathologic Processes - ID: D000064420 - Term: Drug-Related Side Effects and Adverse Reactions - ID: D000064419 - Term: Chemically-Induced Disorders - ID: D000011832 - Term: Radiation Injuries - ID: D000014947 - Term: Wounds and Injuries ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC25 - Name: Substance Related Disorders - Abbrev: BC26 - Name: Wounds and Injuries ### Condition Browse Module - Browse Leaves - ID: M5220 - Name: Breast Neoplasms - Relevance: LOW - As Found: Unknown - ID: M30670 - Name: Cardiotoxicity - Relevance: HIGH - As Found: Cardiotoxicity - ID: M9419 - Name: Heart Diseases - Relevance: LOW - As Found: Unknown - ID: M30303 - Name: Drug-Related Side Effects and Adverse Reactions - Relevance: LOW - As Found: Unknown - ID: M30302 - Name: Chemically-Induced Disorders - Relevance: LOW - As Found: Unknown - ID: M14679 - Name: Radiation Injuries - Relevance: LOW - As Found: Unknown - ID: M17685 - Name: Wounds and Injuries - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000066126 - Term: Cardiotoxicity ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M325 - Name: Trastuzumab - Relevance: LOW - As Found: Unknown - ID: M147959 - Name: Taxane - Relevance: LOW - As Found: Unknown - ID: M289243 - Name: Pertuzumab - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04809779 **Acronym:** NeoPD1TNBC **Brief Title:** PD-1 Inhibitor Concurrent With Chemotherapy as Neoadjuvant Therapy for TNBC **Official Title:** PD-1 Inhibitor Sintilimab Concurrent With Epirubicin Cyclophosphamide and Nab-paclitaxel as Neoadjuvant Therapy for Triple Negative Breast Cancer #### Organization Study ID Info **ID:** 2020466 #### Organization **Class:** OTHER **Full Name:** First Affiliated Hospital, Sun Yat-Sen University ### Status Module #### Completion Date **Date:** 2024-03 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** NOT_YET_RECRUITING #### Last Update Post Date **Date:** 2021-03-22 **Type:** ACTUAL **Last Update Submit Date:** 2021-03-19 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2023-12 **Type:** ESTIMATED #### Start Date **Date:** 2021-03 **Type:** ESTIMATED **Status Verified Date:** 2021-03 #### Study First Post Date **Date:** 2021-03-22 **Type:** ACTUAL **Study First Submit Date:** 2021-03-19 **Study First Submit QC Date:** 2021-03-19 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** First Affiliated Hospital, Sun Yat-Sen University #### Responsible Party **Investigator Affiliation:** First Affiliated Hospital, Sun Yat-Sen University **Investigator Full Name:** Ying Lin **Investigator Title:** Vice Director **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Is US Export:** False ### Description Module **Brief Summary:** The purpose of the study is to address the following hypotheses: the PD-1 inhibitor Sintilimab 200mg for intravenous (IV) administration will be given together with three-week epirubincin, cyclophosphamide (EC) × 4 treatments from the second cycle followed by weekly nab-paclitaxel x12 treatments or three-week nab-paclitaxel x4 treatments. This regimen will induce higher pathologic complete response (pCR) rate in triple negative breast cancer than historical pCR rates (30-40%) observed with chemotherapy alone. ### Conditions Module **Conditions:** - Triple Negative Breast Cancer ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 49 **Type:** ESTIMATED **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Drug: Sintilimab **Label:** Sintilimab **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Sintilimab **Description:** The PD-1 inhibitor Sintilimab 200mg for intravenous (IV) administration will be given together with three-week epirubincin, cyclophosphamide (EC) × 4 treatments from the second cycle followed by weekly nab-paclitaxel x12 treatments or three-week nab-paclitaxel x4 treatments. **Name:** Sintilimab **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Pathologic response will be assessed in the surgically resected cancer and lymph nodes after completion of all chemotherapy by the local pathologist as part of routine care. Pathologic complete response is defined as no invasive cancer in the resected breast tissue and lymph nodes (ypT0/Tis, ypN0). **Measure:** Pathologic Complete Response (pCR) **Time Frame:** 21 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Newly diagnosed histologically confirmed stage I-III, ER, PR and HER2 negative invasive breast cancer as defined by the ASCO CAP guidelines for whom systemic chemotherapy would be indicated based on physician judgment following standard NCCN practice guidelines. Exclusion Criteria: * Patients for whom anthracycline, nab-paclitaxel or antibody therapies are contraindicated. **Maximum Age:** 60 Years **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Nan Shao **Phone:** 02087755766 **Phone Ext:** 8198 **Role:** CONTACT #### Locations **Location 1:** **City:** Guangzhou **Country:** China **Facility:** The First Affiliated Hospital of Sun Yat-sen University ## 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: M30373 - Name: Triple Negative Breast Neoplasms - Relevance: HIGH - As Found: Triple Negative 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 - ID: D000064726 - Term: Triple Negative Breast Neoplasms ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: ARhu - Name: Antirheumatic Agents ### Intervention Browse Module - Browse Leaves - ID: M19537 - Name: Paclitaxel - Relevance: LOW - As Found: Unknown - ID: M231 - Name: Albumin-Bound Paclitaxel - Relevance: LOW - As Found: Unknown - ID: M6727 - Name: Cyclophosphamide - Relevance: LOW - As Found: Unknown - ID: M17954 - Name: Epirubicin - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05658679 **Brief Title:** Radiomics in Pancreatic Cancer **Official Title:** Radiomics-based Detection and Outcome Prediction for Pancreatic Cancer #### Organization Study ID Info **ID:** Retro-001 #### Organization **Class:** OTHER **Full Name:** Ruijin Hospital ### Status Module #### Completion Date **Date:** 2024-12 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2022-12-21 **Type:** ACTUAL **Last Update Submit Date:** 2022-12-13 **Overall Status:** ACTIVE_NOT_RECRUITING #### Primary Completion Date **Date:** 2023-12 **Type:** ESTIMATED #### Start Date **Date:** 2016-01 **Type:** ACTUAL **Status Verified Date:** 2022-12 #### Study First Post Date **Date:** 2022-12-21 **Type:** ACTUAL **Study First Submit Date:** 2022-12-05 **Study First Submit QC Date:** 2022-12-13 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Ruijin Hospital #### Responsible Party **Investigator Affiliation:** Ruijin Hospital **Investigator Full Name:** Weishen WANG **Investigator Title:** Principal Investigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** The images of patients with pancreatic cancer were collected and analyzed based on the methodes of radiomics ### Conditions Module **Conditions:** - Pancreatic Cancer ### Design Module #### Design Info **Observational Model:** CASE_CONTROL **Time Perspective:** RETROSPECTIVE #### Enrollment Info **Count:** 2000 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients with pancreatic tumors diagnosed clinically or pathologically **Label:** patients with pancreatic cancer #### Arm Group 2 **Description:** Participants who judged the pancreas to be completely healthy through medical examination **Label:** Participants without pancreatic cancer ### Outcomes Module #### Primary Outcomes **Measure:** 5-year survival rate **Time Frame:** 1 week ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Gender unlimited, 18-80 years old; * Received abdominal imaging; * informed consent signed. Exclusion Criteria: * Pregnant / lactating women； * Contraindications of imaging examination。 **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 participants willing to receive abdominal imaging could be included in this study ## Derived Section ### Condition Browse Module - Ancestors - ID: D000004067 - Term: Digestive System Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000004701 - Term: Endocrine Gland Neoplasms - ID: D000004066 - Term: Digestive System Diseases - ID: D000010182 - Term: Pancreatic Diseases - ID: D000004700 - Term: Endocrine System Diseases ### 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: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M13110 - Name: Pancreatic Neoplasms - Relevance: HIGH - As Found: Pancreatic Cancer - ID: M8886 - Name: Gastrointestinal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7256 - Name: Digestive System Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7863 - Name: Endocrine Gland 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: M13102 - Name: Pancreatic Diseases - Relevance: LOW - As Found: Unknown - ID: M7862 - Name: Endocrine System Diseases - Relevance: LOW - As Found: Unknown - ID: T4387 - Name: Pancreatic Cancer - Relevance: HIGH - As Found: Pancreatic Cancer ### Condition Browse Module - Meshes - ID: D000010190 - Term: Pancreatic Neoplasms ### Intervention Browse Module - Browse Branches - Abbrev: Gast - Name: Gastrointestinal Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M22554 - Name: Pancrelipase - Relevance: LOW - As Found: Unknown - ID: M13114 - Name: Pancreatin - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01135979 **Brief Title:** Assessment of the Sympathetic Nervous System Blockade of the Upper Limb After a Brachial Plexus Block in Patients With End Stage Renal Failure **Official Title:** Assessment of the Sympathetic Nervous System Blockade of the Upper Limb After a Brachial Plexus Block in Patients With End Stage Renal Failure #### Organization Study ID Info **ID:** 07AN007 #### Organization **Class:** OTHER **Full Name:** University of Nottingham ### Status Module #### Completion Date **Date:** 2012-12 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2013-03-06 **Type:** ESTIMATED **Last Update Submit Date:** 2013-03-05 **Overall Status:** WITHDRAWN #### Primary Completion Date **Date:** 2012-12 **Type:** ESTIMATED #### Start Date **Date:** 2010-02 **Status Verified Date:** 2013-03 #### Study First Post Date **Date:** 2010-06-03 **Type:** ESTIMATED **Study First Submit Date:** 2010-06-02 **Study First Submit QC Date:** 2010-06-02 **Why Stopped:** Unable to recruit ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Nottingham #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to investigate what effect a local anesthetic nerve block of the arm in patients with end stage renal failure has upon blood flow in the skin of the arm. **Detailed Description:** Aim To quantify the degree of sympathetic blockade in the upper limb in patients with end stage renal failure produced during a regional anaesthetic technique, using laser Doppler flowmetry. Hypothesis Regional anaesthesia of the upper limb results in blockade of motor and sensory nerves, providing surgical and post-operative analgesia. Sympathetic nerves are also blocked resulting in local vasodilatation. This may be of benefit in surgery where enhanced blood flow is beneficial such as arterio-venous fistula creation. The degree and duration of sympathetic blockade produced by regional blocks however, has never been quantified. This study aims to quantify sympathetic blockade by measuring the changes in skin blood flow and skin temperature after placement of a brachial plexus block. ### Conditions Module **Conditions:** - Renal Failure ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Label:** Arterio-venous fistulae creation ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Patients with end stage renal failure * Over 18 years of age * Written consent obtained * Due to undergo surgery for forearm fistula creation under a regional block Exclusion Criteria: * Smokers * Damaged skin on the arm * Circulatory disorders such as Raynaud's disease, systemic sclerosis, sickle cell trait or disease * Current use of beta blockers * undergoing haemodialysis for more than 12 months **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Patients with end stage renal failure who are due to have a forearm fistula created ### Contacts Locations Module #### Locations **Location 1:** **City:** Nottingham **Country:** United Kingdom **Facility:** Nottingham University Hospitals NHS Trust **State:** Nottinghamshire **Zip:** NG7 2UH ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007674 - Term: Kidney Diseases - 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 - ID: D000051436 - Term: Renal Insufficiency, Chronic - ID: D000002908 - Term: Chronic Disease - ID: D000020969 - Term: Disease Attributes - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M10699 - Name: Kidney Failure, Chronic - Relevance: HIGH - As Found: End Stage Renal Failure - ID: M26718 - Name: Renal Insufficiency - Relevance: HIGH - As Found: Renal Failure - ID: M10698 - Name: Kidney Diseases - 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 - ID: M26717 - Name: Renal Insufficiency, Chronic - 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 ### Condition Browse Module - Meshes - ID: D000051437 - Term: Renal Insufficiency - ID: D000007676 - Term: Kidney Failure, Chronic ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01883479 **Acronym:** HM2 **Brief Title:** Effect of Exercise and Wellness Interventions on Preventing Postpartum Depression **Official Title:** Effect of Exercise and Wellness Interventions on Preventing Postpartum Depression. #### Organization Study ID Info **ID:** R01MH096748 **Link:** https://reporter.nih.gov/quickSearch/R01MH096748 **Type:** NIH #### Organization **Class:** OTHER **Full Name:** University of Minnesota #### Secondary ID Infos **ID:** R01MH096748 **Link:** https://reporter.nih.gov/quickSearch/R01MH096748 **Type:** NIH ### Status Module #### Completion Date **Date:** 2017-05-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-06-29 **Type:** ACTUAL **Last Update Submit Date:** 2017-06-28 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2017-05-01 **Type:** ACTUAL #### Start Date **Date:** 2012-12 **Status Verified Date:** 2017-06 #### Study First Post Date **Date:** 2013-06-21 **Type:** ESTIMATED **Study First Submit Date:** 2013-04-24 **Study First Submit QC Date:** 2013-06-18 ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** National Institute of Mental Health (NIMH) #### Lead Sponsor **Class:** OTHER **Name:** University of Minnesota #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to examine the efficacy of the active interventions (exercise or wellness/support intervention) compared to usual care on the prevention of postpartum depression among women at risk for depression. The investigators will also examine the efficacy of an exercise intervention compared to a wellness/support intervention on postpartum depressive symptoms among women at risk for depression. **Detailed Description:** The purpose of this study is to examine the efficacy of exercise and wellness/support interventions for preventing postpartum depression. Specifically, 450 sedentary postpartum women (2-6 weeks postpartum) with a history of depression prior to pregnancy will be randomized to one of three groups each lasting six months: (1) telephone-based exercise intervention; (2) telephone-based wellness/support intervention; or (3) usual care. Participants will also complete a follow-up assessment session at 9 months. Participants will be recruited via online, email, and print advertisements. The investigators will obtain healthcare provider consent for each participant prior to randomization. The exercise intervention will consist of a theory-based telephone intervention shown to increase exercise among postpartum women in a previous study (Lewis et al., 2011). The wellness/support condition will be on the same schedule as the exercise intervention and will address several topics related to wellness. The usual care condition will receive their choice of the exercise or wellness/support condition upon completion of their final assessment. The primary dependent variable will be depression as measured by the Structured Clinical Interview for DSM-IV Axis I Disorders (SCID-I) and the Edinburgh Postnatal Depression Scale (EPDS). Exercise adherence will be assessed using the 7-Day Physical Activity Recall Interview and the ActiGraph (i.e., an accelerometer, an objective measure of exercise). ### Conditions Module **Conditions:** - Postpartum Depression ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 450 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Telephone-based intervention designed to increase exercise among postpartum women. **Intervention Names:** - Behavioral: Exercise **Label:** Exercise **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Telephone-based intervention designed to provide support to postpartum women. **Intervention Names:** - Behavioral: Wellness/Support **Label:** Wellness/Support **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Participants receive usual care and will receive their choice of the interventions at 9 months. **Label:** Usual care **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Exercise **Description:** Telephone-based intervention designed to increase exercise among postpartum women. **Name:** Exercise **Type:** BEHAVIORAL #### Intervention 2 **Arm Group Labels:** - Wellness/Support **Description:** Telephone-based intervention designed to provide support to postpartum women. **Name:** Wellness/Support **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** Depression assessed by the The Structured Clinical Interview for DSM-IV Axis I Disorders; SCID-I **Measure:** Depression **Time Frame:** 6 months #### Secondary Outcomes **Description:** Depressive symptoms as assessed by the PHQ-9 **Measure:** Depressive Symptoms **Time Frame:** 6 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Generally healthy * Low active * Currently pregnant (who will wait to be randomized until postpartum) or less than six weeks postpartum * History of depression Exclusion Criteria: * Less than 18 years of age * Pre-existing hypertension or diabetes * Currently exercising (defined as exercising more than 60 minutes per week) * Enrolled in another exercise or weight management study * Another member of the household participating in the study * Unable to exercise for 20 minutes continuously * Musculoskeletal problems such as arthritis, gout, osteoporosis, or back, hip or knee pain that may interfere with exercising * Exercise induced asthma * Any condition that would make exercise unsafe or unwise * Taking medication that interferes with heart rate response to exercise such as beta blockers * Hospitalization for a psychiatric disorder in the past six months * Current depressive episode (based on the SCID) and/or currently receiving antidepressant medication or psychotherapy for depression (participants who are depressed prior to randomization will be referred to their primary physician or psychiatrist) **Healthy Volunteers:** True **Maximum Age:** 45 Years **Minimum Age:** 21 Years **Sex:** FEMALE **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Minneapolis **Country:** United States **Facility:** University of Minnesota **State:** Minnesota **Zip:** 55455 #### Overall Officials **Official 1:** **Affiliation:** University of Minnesota **Name:** Beth A Lewis, PhD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Lewis BA, Schuver K, Dunsiger S, Samson L, Frayeh AL, Terrell CA, Ciccolo JT, Fischer J, Avery MD. Randomized trial examining the effect of exercise and wellness interventions on preventing postpartum depression and perceived stress. BMC Pregnancy Childbirth. 2021 Nov 22;21(1):785. doi: 10.1186/s12884-021-04257-8. **PMID:** 34802425 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000001526 - Term: Behavioral Symptoms - ID: D000019964 - Term: Mood Disorders - ID: D000001523 - Term: Mental Disorders - ID: D000011644 - Term: Puerperal Disorders - 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: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions ### Condition Browse Module - Browse Leaves - ID: M7058 - Name: Depression - Relevance: HIGH - As Found: Depression - ID: M7061 - Name: Depressive Disorder - Relevance: HIGH - As Found: Depression - ID: M21076 - Name: Depression, Postpartum - Relevance: HIGH - As Found: Postpartum Depression - ID: M4818 - Name: Behavioral Symptoms - Relevance: LOW - As Found: Unknown - 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: M14499 - Name: Puerperal Disorders - 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: D000019052 - Term: Depression, Postpartum - ID: D000003863 - Term: Depression - ID: D000003866 - Term: Depressive Disorder ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02914379 **Brief Title:** A Study of LY3337641 in Healthy Male Participants **Official Title:** Disposition of [¹⁴C]-LY3337641 Following Oral Administration in Healthy Male Subjects #### Organization Study ID Info **ID:** 16297 #### Organization **Class:** INDUSTRY **Full Name:** Eli Lilly and Company #### Secondary ID Infos **Domain:** Eli Lilly and Company **ID:** I8K-MC-JPDE **Type:** OTHER ### Status Module #### Completion Date **Date:** 2016-11 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-08-25 **Type:** ACTUAL **Last Update Submit Date:** 2022-10-11 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2016-11 **Type:** ACTUAL #### Results First Post Date **Date:** 2023-08-25 **Type:** ACTUAL **Results First Submit Date:** 2022-10-11 **Results First Submit QC Date:** 2022-10-11 #### Start Date **Date:** 2016-09 **Status Verified Date:** 2022-10 #### Study First Post Date **Date:** 2016-09-26 **Type:** ESTIMATED **Study First Submit Date:** 2016-09-21 **Study First Submit QC Date:** 2016-09-22 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Eli Lilly and Company #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to measure how much LY3337641 gets into the bloodstream and how long it takes the body to get rid of it. In addition, the safety and tolerability of the study drug will be evaluated. Participants will be admitted to a clinical research unit (CRU) the day before dosing. Participants remain confined to the CRU for at least 7 days. Participants may be discharged from the CRU any time after 7 days post-dose (Day 8), and up to a maximum of 21 days post-dose (Day 22). This study will last approximately 30 days for each participant, not including screening. Screening is required within 28 days prior to the start of the study. ### Conditions Module **Conditions:** - Healthy ### 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:** Participants received 20 milligrams (mg) oral dose of LY3337641 containing 120 microcuries of radioactivity. **Intervention Names:** - Drug: [¹⁴C]-LY3337641 **Label:** 20mg [¹⁴C]-LY3337641 **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - 20mg [¹⁴C]-LY3337641 **Description:** Administered orally **Name:** [¹⁴C]-LY3337641 **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Urinary excretion samples from each participant were measured by liquid scintillation counting. The radioactive counts detected in urine samples were each divided by the theoretical radioactive count in the total radioactive dose administered and multiplied by 100% to arrive at a percentage of total radioactive dose excreted in urine and feces. **Measure:** Urinary Excretion of Radioactivity Over Time Expressed as a Percentage of the Total Radioactive Dose Administered **Time Frame:** Baseline up to 22 days **Description:** Fecal excretion samples from each participant were measured by liquid scintillation counting. The radioactive counts detected in fecal samples were each divided by the theoretical radioactive count in the total radioactive dose administered and multiplied by 100% to arrive at a percentage of total radioactive dose excreted in feces. **Measure:** Fecal Excretion of Radioactivity Over Time Expressed as a Percentage of the Total Radioactive Dose Administered **Time Frame:** Baseline up to 22 days #### Secondary Outcomes **Measure:** Pharmacokinetics: Maximum Observed Concentration (Cmax) of Plasma LY3337641 **Time Frame:** Predose, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 16, 24, 36, 48, 72, 96, 120, 144, 168, 216, 264 and 312 hours post dose **Measure:** Pharmacokinetics: Maximum Observed Concentration (Cmax) of Plasma Radioactivity **Time Frame:** Predose, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 16, 24, 36, 48, 72, 96, 120, 144, 168, 216, 264 and 312 hours post dose **Measure:** Pharmacokinetics: Maximum Observed Concentration (Cmax) of Whole Blood Radioactivity **Time Frame:** Predose, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 16, 24, 36, 48, 72, 96, 120, 144, 168, 216, 264 and 312 hours post dose **Measure:** Pharmacokinetics: Area Under the Concentration-Time Curve From Time Zero to Infinity (AUC 0 to ∞) of Plasma LY3337641 **Time Frame:** Predose, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 16, 24, 36, 48, 72, 96, 120, 144, 168, 216, 264 and 312 hours post dose **Measure:** Pharmacokinetics: Area Under the Concentration-Time Curve From Time Zero to Infinity (AUC 0 to ∞) of Plasma Radioactivity **Time Frame:** Predose, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 16, 24, 36, 48, 72, 96, 120, 144, 168, 216, 264 and 312 hours post dose **Measure:** Pharmacokinetics: Area Under the Concentration-Time Curve From Time Zero to Infinity (AUC 0 to ∞) of Whole Blood Radioactivity **Time Frame:** Predose, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 16, 24, 36, 48, 72, 96, 120, 144, 168, 216, 264 and 312 hours post dose **Measure:** Pharmacokinetics: Total Number of Measurable Metabolites of LY3337641 in Plasma, Urine and Feces **Time Frame:** Predose, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 16, 24, 36, 48, 72, 96, 120, 144, 168, 216, 264, and 312 hours postdose ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Overtly healthy males, as determined by medical history and physical examination * Have a body mass index (BMI) between 18.5 and 32.0 kilograms per meter squared (kg/m²) * Have clinical laboratory test results within normal reference range for the population or investigative site, or results with acceptable deviations that are judged to be not clinically significant by the investigator * Have venous access sufficient to allow for blood sampling as per the protocol * Are able and willing to give signed informed consent * Generally have a minimum of 1 bowel movement per day Exclusion Criteria: * Have participated, within the last 30 days, in a clinical trial involving an investigational product. If the previous investigational product has a long half-life, 3 months or 5 half-lives (whichever is longer, if known) should have passed * Have a significant history of or current cardiovascular, respiratory, hepatic, renal, gastrointestinal (GI) (cholecystectomy not acceptable), endocrine, hematological, dermatologic, or neurological disorders capable of significantly altering the absorption, metabolism, or elimination of drugs; of constituting a risk when taking the investigational product; or of interfering with the interpretation of data * Regularly use known drugs of abuse and/or show positive findings on urinary drug screening * Have used or intend to use over-the-counter or prescription medication, including herbal medications, within 14 days prior to dosing * Have donated blood of more than 500 milliliter (mL) within the month prior to screening * Have an average weekly alcohol intake that exceeds 21 units per week, and are unwilling to stop alcohol consumption for 48 hours prior to admission, and while resident in the clinical research unit (CRU) * In the opinion of the investigator or sponsor, are unsuitable for inclusion in the study * Have had exposure to significant diagnostic, therapeutic, or employment related radiation within 12 months prior to dosing (serial x-ray or computed tomography scans, barium meal, current employment in a job requiring radiation exposure monitoring) * Have participated in a \[¹⁴C\] study within the last 6 months prior to admission for this study. The total 12-month exposure from this study and a maximum of one other previous \[¹⁴C\]-study within 6 to 12 months of this study must be within the Code of Federal Regulations (CFR) recommended levels considered safe (per 21 CFR 361.1): less than 5000 Milli-rem (mrem) /year whole body annual exposure **Healthy Volunteers:** True **Maximum Age:** 65 Years **Minimum Age:** 18 Years **Sex:** MALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Madison **Country:** United States **Facility:** Covance Clinical Research Unit **State:** Wisconsin **Zip:** 53704 #### Overall Officials **Official 1:** **Affiliation:** Eli Lilly and Company **Name:** Call 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 9 AM - 5 PM Eastern time (UTC/GMT - 5 hours, EST) **Role:** STUDY_DIRECTOR ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** 20 mg [¹⁴C]-LY3337641 **Deaths Num At Risk:** 6 **Description:** Participants received 20 mg oral dose of LY3337641 containing 120 microcuries of radioactivity. **ID:** EG000 **Other Num Affected:** 1 **Other Num at Risk:** 6 **Serious Number At Risk:** 6 **Title:** 20 mg [¹⁴C]-LY3337641 **Frequency Threshold:** 5 #### Other Events **Term:** Abdominal discomfort **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 19.0 **Term:** Change of bowel habit **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 19.0 **Term:** Diarrhoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 19.0 **Term:** Ecchymosis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 19.0 **Time Frame:** From Baseline to End of Study (Up to 29 Days) ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 6 **Units:** Participants ### Group **ID:** BG000 **Title:** 20mg [¹⁴C]-LY3337641 **Description:** Participants received 20 mg oral dose of LY3337641 containing 120 microcuries of radioactivity. ### Measure #### Measurement **Group ID:** BG000 **Spread:** 11.4 **Value:** 34.3 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 6 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 6 **Category Title:** Not Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** American Indian or Alaska Native #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Asian #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Native Hawaiian or Other Pacific Islander #### Measurement **Group ID:** BG000 **Value:** 1 **Category Title:** Black or African American #### Measurement **Group ID:** BG000 **Value:** 5 **Category Title:** White #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** More than one race #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 6 **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:** Ethnicity (NIH/OMB) **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Race (NIH/OMB) **Unit of Measure:** Participants ### Measure 5 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Region of Enrollment **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement **Restriction Type:** GT60 **Restrictive Agreement:** True ### Point of Contact **Email:** [email protected] **Organization:** Eli Lilly and Company **Phone:** 800-545-5979 **Title:** Chief Medical Officer ## 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 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 1.0 - **Upper Limit:** - **Value:** 2.9 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 5.1 - **Upper Limit:** - **Value:** 86.4 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 26 - **Upper Limit:** - **Value:** 77.9 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 25 - **Upper Limit:** - **Value:** 122 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 24 - **Upper Limit:** - **Value:** 67.5 **Title:** #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 25 - **Upper Limit:** - **Value:** 370 **Title:** #### Outcome Measure 7 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 51 - **Upper Limit:** - **Value:** 1240 **Title:** #### Outcome Measure 8 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 105 - **Upper Limit:** - **Value:** 5840 **Title:** #### Outcome Measure 9 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 6 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 8 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Urinary excretion samples from each participant were measured by liquid scintillation counting. The radioactive counts detected in urine samples were each divided by the theoretical radioactive count in the total radioactive dose administered and multiplied by 100% to arrive at a percentage of total radioactive dose excreted in urine and feces. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** All participants who received the study drug and have evaluable pharmacokinetic (PK) data. **Reporting Status:** POSTED **Time Frame:** Baseline up to 22 days **Title:** Urinary Excretion of Radioactivity Over Time Expressed as a Percentage of the Total Radioactive Dose Administered **Type:** PRIMARY **Unit of Measure:** Percentage of total dose ##### Group **Description:** Participants received 20 mg oral dose of LY3337641 containing 120 microcuries of radioactivity. **ID:** OG000 **Title:** 20 mg [¹⁴C]-LY3337641 #### Outcome Measure 2 **Description:** Fecal excretion samples from each participant were measured by liquid scintillation counting. The radioactive counts detected in fecal samples were each divided by the theoretical radioactive count in the total radioactive dose administered and multiplied by 100% to arrive at a percentage of total radioactive dose excreted in feces. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** All participants who received the study drug and have evaluable PK data. **Reporting Status:** POSTED **Time Frame:** Baseline up to 22 days **Title:** Fecal Excretion of Radioactivity Over Time Expressed as a Percentage of the Total Radioactive Dose Administered **Type:** PRIMARY **Unit of Measure:** Percentage of total dose ##### Group **Description:** Participants received 20 mg oral dose of LY3337641 containing 120 microcuries of radioactivity. **ID:** OG000 **Title:** 20 mg [¹⁴C]-LY3337641 #### Outcome Measure 3 **Dispersion Type:** Geometric Coefficient of Variation **Parameter Type:** GEOMETRIC_MEAN **Population Description:** All participants who received the study drug and have evaluable PK data. **Reporting Status:** POSTED **Time Frame:** Predose, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 16, 24, 36, 48, 72, 96, 120, 144, 168, 216, 264 and 312 hours post dose **Title:** Pharmacokinetics: Maximum Observed Concentration (Cmax) of Plasma LY3337641 **Type:** SECONDARY **Unit of Measure:** Nanograms/milliliter (ng/mL) ##### Group **Description:** Participants received 20 mg oral dose of LY3337641 containing 120 microcuries of radioactivity. **ID:** OG000 **Title:** 20 mg [¹⁴C]-LY3337641 #### Outcome Measure 4 **Dispersion Type:** Geometric Coefficient of Variation **Parameter Type:** GEOMETRIC_MEAN **Population Description:** All participants who received the study drug and have evaluable PK data. **Reporting Status:** POSTED **Time Frame:** Predose, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 16, 24, 36, 48, 72, 96, 120, 144, 168, 216, 264 and 312 hours post dose **Title:** Pharmacokinetics: Maximum Observed Concentration (Cmax) of Plasma Radioactivity **Type:** SECONDARY **Unit of Measure:** nanogram equivalents/g (ng-equiv/g) ##### Group **Description:** Participants received 20 mg oral dose of LY3337641 containing 120 microcuries of radioactivity. **ID:** OG000 **Title:** 20 mg [¹⁴C]-LY3337641 #### Outcome Measure 5 **Dispersion Type:** Geometric Coefficient of Variation **Parameter Type:** GEOMETRIC_MEAN **Population Description:** All participants who received the study drug and have evaluable PK data. **Reporting Status:** POSTED **Time Frame:** Predose, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 16, 24, 36, 48, 72, 96, 120, 144, 168, 216, 264 and 312 hours post dose **Title:** Pharmacokinetics: Maximum Observed Concentration (Cmax) of Whole Blood Radioactivity **Type:** SECONDARY **Unit of Measure:** ng/mL ##### Group **Description:** Participants received 20 mg oral dose of LY3337641 containing 120 microcuries of radioactivity. **ID:** OG000 **Title:** 20 mg [¹⁴C]-LY3337641 #### Outcome Measure 6 **Dispersion Type:** Geometric Coefficient of Variation **Parameter Type:** GEOMETRIC_MEAN **Population Description:** All participants who received the study drug and have evaluable PK data. **Reporting Status:** POSTED **Time Frame:** Predose, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 16, 24, 36, 48, 72, 96, 120, 144, 168, 216, 264 and 312 hours post dose **Title:** Pharmacokinetics: Area Under the Concentration-Time Curve From Time Zero to Infinity (AUC 0 to ∞) of Plasma LY3337641 **Type:** SECONDARY **Unit of Measure:** Nanograms*hours per milliliter (ng*h/mL) ##### Group **Description:** Participants received 20 mg oral dose of LY3337641 containing 120 microcuries of radioactivity. **ID:** OG000 **Title:** 20 mg [¹⁴C]-LY3337641 #### Outcome Measure 7 **Dispersion Type:** Geometric Coefficient of Variation **Parameter Type:** GEOMETRIC_MEAN **Population Description:** All participants who received the study drug and have evaluable PK data. **Reporting Status:** POSTED **Time Frame:** Predose, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 16, 24, 36, 48, 72, 96, 120, 144, 168, 216, 264 and 312 hours post dose **Title:** Pharmacokinetics: Area Under the Concentration-Time Curve From Time Zero to Infinity (AUC 0 to ∞) of Plasma Radioactivity **Type:** SECONDARY **Unit of Measure:** ng-equivalents*h/gram (ng-equiv*h/g) ##### Group **Description:** Participants received 20 mg oral dose of LY3337641 containing 120 microcuries of radioactivity. **ID:** OG000 **Title:** 20 mg [¹⁴C]-LY3337641 #### Outcome Measure 8 **Dispersion Type:** Geometric Coefficient of Variation **Parameter Type:** GEOMETRIC_MEAN **Population Description:** All participants who received the study drug and have evaluable PK data. **Reporting Status:** POSTED **Time Frame:** Predose, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 16, 24, 36, 48, 72, 96, 120, 144, 168, 216, 264 and 312 hours post dose **Title:** Pharmacokinetics: Area Under the Concentration-Time Curve From Time Zero to Infinity (AUC 0 to ∞) of Whole Blood Radioactivity **Type:** SECONDARY **Unit of Measure:** ng*h/mL ##### Group **Description:** Participants received 20 mg oral dose of LY3337641 containing 120 microcuries of radioactivity. **ID:** OG000 **Title:** 20 mg [¹⁴C]-LY3337641 #### Outcome Measure 9 **Parameter Type:** NUMBER **Population Description:** All participants who received the study drug and have evaluable PK data. **Reporting Status:** POSTED **Time Frame:** Predose, 0.5, 1, 2, 3, 4, 6, 8, 10, 12, 16, 24, 36, 48, 72, 96, 120, 144, 168, 216, 264, and 312 hours postdose **Title:** Pharmacokinetics: Total Number of Measurable Metabolites of LY3337641 in Plasma, Urine and Feces **Type:** SECONDARY **Unit of Measure:** Number of Metabolites ##### Group **Description:** Participants received 20 mg oral dose of LY3337641 containing 120 microcuries of radioactivity. **ID:** OG000 **Title:** 20 mg [¹⁴C]-LY3337641 ### Participant Flow Module #### Group **Description:** Participants received 20 milligrams (mg) oral dose of LY3337641 containing 120 microcuries of radioactivity. **ID:** FG000 **Title:** 20mg [¹⁴C]-LY3337641 #### Period **Title:** Overall Study ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 6 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 6 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 0 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT06040879 **Brief Title:** PENG Block and Lateral Femoral Cutaneous Nerve Block For Hip Replacement Surgery **Official Title:** Postoperative Analgesia Outcomes Of Pericapsular Nerve Group Block (PENG Block) Combined With Lateral Femoral Cutaneous Nerve Block After Hip Replacement Surgery: A Randomized Controlled Study #### Organization Study ID Info **ID:** HMU09.2023 #### Organization **Class:** OTHER **Full Name:** Hanoi Medical University ### Status Module #### Completion Date **Date:** 2022-09-03 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-09-18 **Type:** ACTUAL **Last Update Submit Date:** 2023-09-14 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2022-08-31 **Type:** ACTUAL #### Start Date **Date:** 2021-08-01 **Type:** ACTUAL **Status Verified Date:** 2023-09 #### Study First Post Date **Date:** 2023-09-18 **Type:** ACTUAL **Study First Submit Date:** 2023-09-07 **Study First Submit QC Date:** 2023-09-14 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Hanoi 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:** Background: this study aimed to describe the pain relief outcomes after hip replacement surgery by continuous Pericapsular Nerve Group Block (PENG Block) in combination with lateral femoral cutaneous nerve (LFCN) block under the guidance of ultrasound. Methods: patients who had hip surgery at E University hospital, Hanoi, Vietnam from August 2021 to August 2022 belonged to two groups: group of patients with pain relief with PENG block in combination with LFCN block (PENG BLOCK group) and group of patients with patient-controlled intravenous analgesia (PCA group). Outcomes regarding clinical and pain score from initiation of insertion or PCA insertion (H0) to after 72 hours (H72) were recorded. **Detailed Description:** First, patients were explained about the study and asked to sign written informed consent if they agreed to participate in the study. Next, the patient was instructed to use the visual analogue scale (VAS) for pain score, as well as how to press the button to request pain relief. Then, a monitor was installed and operated to assess the clinical parameters (electrocardiogram, blood pressure, SpO2, arterial blood pressure, temperature). The patient was oxygenated 3-5 liters/minute and performed a peripheral intravenous line, with an 18G catheter, infused with 0.9% NaCl solution. Spinal anesthesia was administered with ropivacaine 0.5% and fentanyl. During and after the surgery, a group of patients received intravenous morphine analgesia via PCA (concentration 1mg/ml, bolus dose of 1mg, lock time 10 minutes, maximum dose 10mg/4 hours). The remaining group of patients receive pain relief by PENG block and LFCN block. In this group, the ultrasound probe was placed horizontally from the anterior superior iliac spine, and was moved along the femoral arc defining the pubic spine. Then, the transducer was rotated 45 degrees, moved parallel to the femoral arch identifying the anterior inferior iliac spine (AIIS), iliopubic eminence (IPE) and inferior lumbosacral head. The ultrasound probe was moved lightly until the upper end of the femoral head was identified. Next, a 120 mm Tuohy 18G anesthetic needle was used under ultrasound guidance, which was moved lateral to medial in the plane between the ultrasound transducer and the superior tip of the femoral head. Then, 10 mL of ropivacaine 0.25% was injected through the anesthetic needle tip. Data were collected at different times including: before surgery, before anesthesia, during surgery and after surgery. The information was recorded in the medical record. Preoperative data: the collected information included the general characteristics of the study patients including age, gender, weight, height, history of smoking/motion sickness, diagnosis, health classification according to ASA, liver and kidney function tests. With pre-anesthesia data, information was collected including pulse, blood pressure, respiratory rate, SpO2. Intraoperative data: the information collected includes information about anesthesia and surgical procedure. Post-operative data: the collected information includes blood test results (urea, creatinine, liver enzymes), pain score (Visual analogue scale - VAS - scale from 0 to 10, the higher the score, the higher the pain level. more), analgesic drugs consumption status, respiratory and circulatory changes, adverse events, and analgesic satisfaction. Time was recorded from initiation of catheterization or PCA insertion (H0) to after 72 hours (H72). ### Conditions Module **Conditions:** - POSTOPERATIVE ANALGESIA **Keywords:** - Pericapsular Nerve Group Block - lateral femoral cutaneous nerve - multimodal analgesia - ultrasound-guided analgesia - patient-controlled analgesia ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** PCA group and PENG BLOCK group ##### Masking Info **Masking:** SINGLE **Who Masked:** - PARTICIPANT **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Count:** 60 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** receiving pain relief through PENG and LFCN block **Intervention Names:** - Procedure: PERICAPSULAR NERVE GROUP BLOCK COMBINED WITH LATERAL FEMORAL CUTANEOUS NERVE BLOCK **Label:** PENG BLOCK **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** receiving morphine PCA analgesia **Intervention Names:** - Procedure: PERICAPSULAR NERVE GROUP BLOCK COMBINED WITH LATERAL FEMORAL CUTANEOUS NERVE BLOCK **Label:** PCA **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - PCA - PENG BLOCK **Description:** During and after the surgery, a group of patients received intravenous morphine analgesia via PCA (concentration 1mg/ml, bolus dose of 1mg, lock time 10 minutes, maximum dose 10mg/4 hours). The remaining group of patients receive pain relief by PENG block and LFCN block. In this group, the ultrasound probe was placed horizontally from the anterior superior iliac spine, and was moved along the femoral arc defining the pubic spine. Then, the transducer was rotated 45 degrees, moved parallel to the femoral arch identifying the anterior inferior iliac spine (AIIS), iliopubic eminence (IPE) and inferior lumbosacral head. The ultrasound probe was moved lightly until the upper end of the femoral head was identified. Next, a 120 mm Tuohy 18G anesthetic needle was used under ultrasound guidance, which was moved lateral to medial in the plane between the ultrasound transducer and the superior tip of the femoral head. Then, 10 mL of ropivacaine 0.25% was injected through the anesthetic needle tip **Name:** PERICAPSULAR NERVE GROUP BLOCK COMBINED WITH LATERAL FEMORAL CUTANEOUS NERVE BLOCK **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Description:** the average VAS score (Visual Analogue Scale) at rest and on movement of both groups. The Visual Analogue Scale (VAS) measures pain intensity. The VAS consists of a 10cm line, with two end points representing 0 ('no pain') and 10 ('pain as bad as it could possibly be'). Ask the patient to rate their current level of pain by placing a mark on the line. Use a ruler to measure the distance in centimetres from the 'no pain marker' (or zero) to the current pain mark. **Measure:** VAS score at rest and on movement **Time Frame:** Time was recorded from initiation of catheterization or PCA insertion (H0) to after 72 hours (H72) **Description:** Rate of vomitting/nausea, itchy, urinary retention, respiratory failure postoperative of both groups. **Measure:** Side effects **Time Frame:** Time was recorded from initiation of catheterization or PCA insertion (H0) to after 72 hours (H72) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * American Society of Anesthesiologists (ASA) physical status I-III. * Patients had hip replacement surgery. Exclusion Criteria: * infection of the anesthetized area. * coagulation disorders * organs dysfunction * allergy to anesthetics * did not cooperate with physicians * history of opioid dependence * inability to participate in the study **Maximum Age:** 80 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Hanoi **Country:** Vietnam **Facility:** Hanoi Medical University #### Overall Officials **Official 1:** **Affiliation:** Hanoi Medical University **Name:** Tu Nguyen, Professor **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **Access Criteria:** anesthesiologists, surgeons all over the world **Description:** We agree that all results and conclusion of this research will be used fully for other researchers **Info Types:** - STUDY_PROTOCOL - SAP - ICF - CSR - ANALYTIC_CODE **IPD Sharing:** YES **Time Frame:** from September 2023 to the unidentified time ### References Module #### References **Citation:** Ferguson RJ, Palmer AJ, Taylor A, Porter ML, Malchau H, Glyn-Jones S. Hip replacement. Lancet. 2018 Nov 3;392(10158):1662-1671. doi: 10.1016/S0140-6736(18)31777-X. **PMID:** 30496081 **Citation:** Ferrata P, Carta S, Fortina M, Scipio D, Riva A, Di Giacinto S. Painful hip arthroplasty: definition. Clin Cases Miner Bone Metab. 2011 May;8(2):19-22. **PMID:** 22461810 **Citation:** YaDeau JT, Tedore T, Goytizolo EA, Kim DH, Green DS, Westrick A, Fan R, Rade MC, Ranawat AS, Coleman SH, Kelly BT. Lumbar plexus blockade reduces pain after hip arthroscopy: a prospective randomized controlled trial. Anesth Analg. 2012 Oct;115(4):968-72. doi: 10.1213/ANE.0b013e318265bacd. Epub 2012 Jul 19. **PMID:** 22822195 **Citation:** Duarte LT, Beraldo PS, Saraiva RA. [Effects of epidural analgesia and continuous lumbar plexus block on functional rehabilitation after total hip arthroplasty]. Rev Bras Anestesiol. 2009 Sep-Oct;59(5):531-44. doi: 10.1016/s0034-7094(09)70078-9. Portuguese. **PMID:** 19784509 **Citation:** Singelyn FJ, Ferrant T, Malisse MF, Joris D. Effects of intravenous patient-controlled analgesia with morphine, continuous epidural analgesia, and continuous femoral nerve sheath block on rehabilitation after unilateral total-hip arthroplasty. Reg Anesth Pain Med. 2005 Sep-Oct;30(5):452-7. doi: 10.1016/j.rapm.2005.05.008. **PMID:** 16135349 **Citation:** Lin DY, Morrison C, Brown B, Saies AA, Pawar R, Vermeulen M, Anderson SR, Lee TS, Doornberg J, Kroon HM, Jaarsma RL. Pericapsular nerve group (PENG) block provides improved short-term analgesia compared with the femoral nerve block in hip fracture surgery: a single-center double-blinded randomized comparative trial. Reg Anesth Pain Med. 2021 May;46(5):398-403. doi: 10.1136/rapm-2020-102315. Epub 2021 Feb 26. **PMID:** 33637625 **Citation:** Shafiq F, Hamid M, Samad K. Complications and interventions associated with epidural analgesia for postoperative pain relief in a tertiary care hospital. Middle East J Anaesthesiol. 2010 Oct;20(6):827-32. **PMID:** 21526668 **Citation:** Rasouli MR, Viscusi ER. Adductor Canal Block for Knee Surgeries: An Emerging Analgesic Technique. Arch Bone Jt Surg. 2017 May;5(3):131-132. No abstract available. **PMID:** 28656158 **Citation:** Kamel I, Ahmed MF, Sethi A. Regional anesthesia for orthopedic procedures: What orthopedic surgeons need to know. World J Orthop. 2022 Jan 18;13(1):11-35. doi: 10.5312/wjo.v13.i1.11. eCollection 2022 Jan 18. **PMID:** 35096534 #### See Also Links **Label:** Related Info **URL:** https://www.who.int/news-room/fact-sheets/detail/musculoskeletal-conditions ## Derived Section ### Condition Browse Module - Ancestors - ID: D000010468 - Term: Perceptual Disorders - ID: D000019954 - Term: Neurobehavioral Manifestations - ID: D000009461 - Term: Neurologic Manifestations - ID: D000009422 - Term: Nervous System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M3727 - Name: Agnosia - Relevance: HIGH - As Found: Analgesia - ID: M13379 - Name: Perceptual 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: T241 - Name: Agnosia - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000000377 - Term: Agnosia ### Intervention Browse Module - Browse Branches - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Analg - Name: Analgesics ### Intervention Browse Module - Browse Leaves - ID: M1700 - Name: Ropivacaine - Relevance: LOW - As Found: Unknown - ID: M4107 - Name: Anesthetics - Relevance: LOW - As Found: Unknown - ID: M11982 - Name: Morphine - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00521079 **Acronym:** EMPOWER **Brief Title:** EMPOWER Clinical Trial: Vagal Blocking for Obesity Control **Official Title:** EMPOWER Clinical Trial: Vagal Blocking for Obesity Control #### Organization Study ID Info **ID:** D00343-000 #### Organization **Class:** INDUSTRY **Full Name:** ReShape Lifesciences ### Status Module #### Completion Date **Date:** 2018-05 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-08-24 **Type:** ACTUAL **Last Update Submit Date:** 2018-07-26 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2009-10 **Type:** ACTUAL #### Results First Post Date **Date:** 2017-02-20 **Type:** ACTUAL **Results First Submit Date:** 2016-08-23 **Results First Submit QC Date:** 2016-12-28 #### Start Date **Date:** 2007-08 **Status Verified Date:** 2018-05 #### Study First Post Date **Date:** 2007-08-27 **Type:** ESTIMATED **Study First Submit Date:** 2007-08-23 **Study First Submit QC Date:** 2007-08-23 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** ReShape Lifesciences #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** This is a randomized multi-center study being done to measure the ability of a new medical device, Maestro System, to safely reduce body weight over five years in people who are considered obese. **Detailed Description:** The Maestro System is a neuromodulation system that consists of implantable and external components. Implantable components: two leads (one electrode each for the anterior and posterior intra-abdominal vagal nerve trunks) that are connected to an implantable neuroregulator. External components: one programmable, battery-powered, ambulatory external controller connected via a small, flexible cable to a cutaneous transmit coil that is positioned externally over the neuroregulator. A clinician programmer that transmits information to the controller and uploads data from the controller. All non-diabetic subjects will be randomized in a 2:1 allocation to therapy ON or therapy OFF groups. All type 2 diabetes mellitus subjects will be randomized in a 1:1 allocation to therapy ON or therapy OFF groups. All subjects will receive blinded therapy through the 12-month follow-up visit. All subjects will participate in a medical weight management program. ### Conditions Module **Conditions:** - Obesity **Keywords:** - Obesity - Bariatric surgery - Excess weight loss - Vagus nerve - Vagal blocking - vBloc(TM) therapy ### 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:** 294 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Subjects implanted with a functional Maestro System device that delivers therapy (Therapy ON). **Intervention Names:** - Device: Therapy ON **Label:** vBloc **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Subjects implanted with a functional Maestro System device that does NOT deliver therapy (Therapy OFF). **Intervention Names:** - Device: Therapy OFF **Label:** Placebo **Type:** SHAM_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - vBloc **Description:** Intermittent, programmable, intra-abdominal vagal blocking device that delivers therapy (Therapy ON) **Name:** Therapy ON **Other Names:** - Maestro System **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - Placebo **Description:** Active intra-abdominal placebo device that delivers no therapy (Therapy OFF) **Name:** Therapy OFF **Other Names:** - Maestro System **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Observe at least a 10% greater percentage excess weight loss (%EWL) with vBloc therapy delivered by the Maestro System compared to sham 12 months following randomization using MetLife Method (ideal body weight is calculated based on Metropolitan Height and Weight Tables) **Measure:** Percentage of Excess Weight Loss (EWL) With the Maestro System **Time Frame:** Baseline and 1 Year **Description:** To estimate the rate of serious, system- and procedure-related adverse events associated with the Maestro System. **Measure:** Rate of System and Procedure-related Serious Adverse Events (SAEs). **Time Frame:** 1 Year #### Secondary Outcomes **Description:** To evaluate the percentage of participants achieving 25% excess weight loss from baseline (implant) to 12 months between treatment groups. **Measure:** Percentage of Participants Achieving 25% Excess Weight Loss (%EWL) **Time Frame:** Baseline and 1 Year ### Eligibility Module **Eligibility Criteria:** Inclusion criteria 1. Informed consent. 2. Body mass index (BMI) ≥ 40 kg/m2 to 45 kg/m2 or BMI ≥ 35 kg/m2 to 39.9 kg/m2 with one or more obesity related co-morbid condition. Co-morbid conditions may include one or more of the following: * Type 2 diabetes mellitus (note: type 2 diabetics are allowed at selected centers only, see Inclusion criterion #5) * Hypertension as defined by systolic pressure ≥140 mmHg and/or diastolic pressure ≥90 mmHg a) treated or untreated with systolic ≥140 mmHg or diastolic ≥90 mmHg or b) treated with systolic \<140 mmHg and diastolic \<90 mmHg * Dyslipidemia as defined by total cholesterol ≥200 or LDL ≥130 a) treated or untreated with total cholesterol ≥200 or LDL ≥130 or b) treated with total cholesterol \<200 or LDL \<130 * Sleep apnea syndrome (confirmed by overnight p02 studies) * Obesity related cardiomyopathy 3. Females or males Note: females of child-bearing potential must have a negative urine pregnancy test at Screen and also within 14 days of implant procedure followed by physician-approved contraceptive regimen for the duration of the study period. 4. 18-65 years of age inclusive. 5. Type 2 diabetes mellitus subjects (at selected centers, limited to approximately 34 subjects) with: * Glycosylated hemoglobin (Hb A1c) 6.5 - 9 % inclusive at screening visit. * Onset: 10 years or less since initial diagnosis. * Stable treatment regimen: no change in oral hypoglycemic treatment regimen within past 3 months. * Currently not using insulin therapy, GLP-1 receptor agonists (e.g., exenatide), or DPP-4 inhibitors (e.g., sitagliptin) for diabetes treatment and have not been on these treatments in the past 6 months. * Creatinine within normal reference range. * No history of proliferative retinopathy. * No history of peripheral neuropathy. * No history of autonomic neuropathy. * No history of coronary artery disease, with or without angina pectoris. * No history of peripheral vascular disease. 6. Failure to respond to supervised diet/exercise program(s) in which the subject was engaged for at least 6 months within the last five years. 7. Ability to complete all study visits and procedures. Exclusion criteria 1. Concurrent chronic pancreatic disease. 2. History of Crohn's disease and/or ulcerative colitis. 3. History of bariatric surgery, fundoplication, gastric resection or major upper-abdominal surgery (acceptable surgeries include cholecystectomy, hysterectomy). 4. History of pulmonary embolism or blood coagulation disorders. 5. Clinically significant hiatal hernias known from subject's medical record as or determined by upper endoscopy prior to implant if they have not had one done during the previous 6 months that specifically reported on the presence or absence of hiatal hernia making reference to the Z-line and/or diaphragmatic notch, in order to rule out subjects with hiatal hernia that may require surgical repair (to support exclusion criterion #7). 6. Current portal hypertension and/or esophageal varices. 7. Intra-operative exclusion: hiatal hernia requiring surgical repair or extensive dissection at esophagogastric junction at time of surgery. 8. Treatment with weight-loss prescription drug therapy within the prior three months and the use of prescription drug therapy or the use over-the-counter weight loss preparations for the duration of the trial. 9. Smoking cessation within the prior six months. 10. Known genetic cause of obesity (e.g., Prader-Willi Syndrome). 11. Overall sustained reduction of more than 10% of body weight in the previous 12 months. 12. Physician-prescribed pre-operative diet with intent to lose weight prior to surgery (note: a) study subject may continue any personal diet they were on prior to study enrollment \[see exclusion criterion #24\] b) standardized EMPOWER weight management program to be initiated in all subjects at time of activation, approximately two weeks after implant) 13. Current type 1 diabetes mellitus (DM). 14. Current or recent history (within 12 months) of ongoing bulimia. 15. Current alterations in treatment for thyroid disorders (stable treatment regimen for prior three months acceptable). 16. Current alterations in treatment for epilepsy (stable treatment regimen for prior six months acceptable). 17. Current treatment for peptic ulcer disease (previous history acceptable). 18. Chronic (more than 4 weeks of daily use) treatment with narcotic analgesic drug regimens (treatment with non-steroidal anti-inflammatory drugs acceptable). 19. Current alterations in treatment regimens of anti-cholinergic drugs, including tricyclic antidepressants (stable treatment regimen for prior six months acceptable). 20. Current medical condition that, in the opinion of the investigator, would make subject unfit for surgery under general anesthesia or that would be exacerbated by intentional weight loss. Some examples include diagnosis of cancer, recent heart attack, recent stroke or recent serious trauma. 21. Presence of permanently implanted electrical powered medical device or implanted gastrointestinal device or prosthesis (e.g., pacemakers, implanted defibrillators, neurostimulators etc.). 22. Planned or contemplated use of Magnetic Resonance Imaging (MRI) or oncological radiation during the course of the trial. 23. Significant psychiatric disorders that, in the opinion of the investigator, may interfere with subject's ability to follow study procedures and/or instructions. 24. Current, active member of an organized weight loss program (e.g., Weight Watchers, TOPS). 25. Current participant in another weight loss study or other clinical trials. 26. Have a friend or family member who is currently participating or is planning to participate in this clinical trial. 27. Patient reported: * inability to walk for about 10 minutes without stopping, * feeling of pain in chest when doing physical activity, * feeling of pain in chest when not doing physical activity. Note: unless pain in chest in known to be related to upper gastrointestinal disorders such as gastroesophageal reflux disease or heartburn. 28. Clinically significant cardiac rhythm disorder that requires either medical and/or surgical intervention (e.g., paroxysmal or chronic atrial fibrillation). **Maximum Age:** 65 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Scottsdale **Country:** United States **Facility:** HonorHealth (formerly Scottsdale Bariatric Center) **State:** Arizona **Zip:** 85258 **Location 2:** **City:** La Jolla **Country:** United States **Facility:** Scripps Clinic **State:** California **Zip:** 92037 **Location 3:** **City:** Orange **Country:** United States **Facility:** University of California, Irvine Medical Center **State:** California **Zip:** 92868 **Location 4:** **City:** Stanford **Country:** United States **Facility:** Stanford University Medical Center **State:** California **Zip:** 94305 **Location 5:** **City:** Weston **Country:** United States **Facility:** Cleveland Clinic - Florida **State:** Florida **Zip:** 33331 **Location 6:** **City:** Baltimore **Country:** United States **Facility:** Johns Hopkins **State:** Maryland **Zip:** 21224 **Location 7:** **City:** Boston **Country:** United States **Facility:** Tufts New England Medical Center **State:** Massachusetts **Zip:** 02111 **Location 8:** **City:** Minneapolis **Country:** United States **Facility:** University of Minnesota **State:** Minnesota **Zip:** 55455 **Location 9:** **City:** Rochester **Country:** United States **Facility:** Mayo Clinic **State:** Minnesota **Zip:** 55905 **Location 10:** **City:** Saint Louis **Country:** United States **Facility:** Washington University School of Medicine **State:** Missouri **Zip:** 63110 **Location 11:** **City:** Cleveland **Country:** United States **Facility:** Cleveland Clinic - Ohio **State:** Ohio **Zip:** 44195 **Location 12:** **City:** Portland **Country:** United States **Facility:** Oregon Health & Science University **State:** Oregon **Zip:** 97239-3098 **Location 13:** **City:** Richmond **Country:** United States **Facility:** Virginia Commonwealth University **State:** Virginia **Zip:** 23298 **Location 14:** **City:** Sydney **Country:** Australia **Facility:** Institute of Weight Control **State:** New South Wales **Zip:** 2153 **Location 15:** **City:** Bedford Park **Country:** Australia **Facility:** Adelaide Bariatric Center - Flinders Private Hospital **State:** South Australia **Zip:** 5042 #### Overall Officials **Official 1:** **Affiliation:** VA Medical Center, Minneapolis, MN / University of Minnesota **Name:** Charles J Billington, MD **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** Mayo Clinic **Name:** Michael Sarr, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000050177 - Term: Overweight - ID: D000044343 - Term: Overnutrition - ID: D000009748 - Term: Nutrition Disorders - ID: D000001835 - Term: Body Weight ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases ### Condition Browse Module - Browse Leaves - ID: M5114 - Name: Body Weight - Relevance: LOW - As Found: Unknown - ID: M18102 - Name: Weight Loss - Relevance: LOW - As Found: Unknown - 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 ### Condition Browse Module - Meshes - ID: D000009765 - Term: Obesity ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** Includes roll-in subjects. #### Event Groups **Group ID:** EG000 **Title:** vBloc **Description:** Subjects implanted with a functional Maestro System device that delivers therapy (Therapy ON). **ID:** EG000 **Other Num Affected:** 146 **Other Num at Risk:** 192 **Serious Number Affected:** 23 **Serious Number At Risk:** 192 **Title:** vBloc **Group ID:** EG001 **Title:** Placebo **Description:** Subjects implanted with a functional Maestro System device that does NOT delivers therapy (Therapy OFF). **ID:** EG001 **Other Num Affected:** 70 **Other Num at Risk:** 102 **Serious Number Affected:** 12 **Serious Number At Risk:** 102 **Title:** Placebo **Frequency Threshold:** 5 #### Other Events **Term:** Bloating Abdominal **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Chest Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Primarily related to the sensation of therapy. Generally occurred early with mild to moderate severity. **Organ System:** General disorders **Source Vocabulary:** **Term:** Constipation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Diarrhea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Eructation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Heartburn/Dyspepsia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Incision pain incision site **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Surgical and medical procedures **Source Vocabulary:** **Term:** Nausea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Pain Abdominal **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Pain neuroregulator site **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** **Term:** Skin reaction to coil/coil adhesion method **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** **Term:** Wound redness or irritation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** **Term:** Cold/flu/respiratory tract infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** **Term:** Depression **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** **Term:** Energy decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** **Term:** Headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** **Term:** Edema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** **Term:** Emesis (vomiting) **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Out of range lab values **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** **Term:** Vitamin or mineral insufficiency **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** **Term:** Cramps abdominal **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Reaction to medicines **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** **Term:** Urinary tract infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** **Term:** Anxiety **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** **Term:** Trauma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** **Term:** Paresthesia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** #### Serious Events **Term:** Incision pain incision site **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Surgical and medical procedures ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 192 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 102 **Term:** Infection neuroregulator site **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 192 **Num Events:** 2 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 102 **Num Events:** 1 **Term:** Lead impedance high **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Surgical and medical procedures ##### Stats **Group ID:** EG000 **Num At Risk:** 192 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 102 **Num Events:** 1 **Term:** Neuroregulator malfunction **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Surgical and medical procedures ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 192 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 102 **Term:** Pain abdominal **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 192 **Num Events:** 3 **Group ID:** EG001 **Num At Risk:** 102 **Term:** Pain neuroregulator site **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 192 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 102 **Num Events:** 1 **Term:** Bleeding other **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Reproductive system and breast disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 192 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 102 **Term:** Cardiac abnormality **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 192 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 102 **Term:** Cold/flu/respiratory tract infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 192 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 102 **Term:** Gallbladder disease **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 192 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 102 **Term:** Hypertension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 192 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 102 **Num Events:** 1 **Term:** Infection other **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 192 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 102 **Num Events:** 1 **Term:** Large bowel dysfunction **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 192 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 102 **Num Events:** 1 **Term:** Other **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders ##### Stats **Group ID:** EG000 **Num Affected:** 8 **Num At Risk:** 192 **Num Events:** 8 **Group ID:** EG001 **Num Affected:** 6 **Num At Risk:** 102 **Num Events:** 6 **Term:** Pain other **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 192 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 102 **Term:** Reaction to medicines **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 192 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 102 ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 183 **Group ID:** BG001 **Value:** 97 **Group ID:** BG002 **Value:** 280 **Units:** Participants ### Group **ID:** BG000 **Title:** vBloc **Description:** Subjects implanted with a functional Maestro System device that delivers therapy (Therapy ON). ### Group **ID:** BG001 **Title:** Placebo **Description:** Subjects implanted with a functional Maestro System device that does NOT deliver therapy (Therapy OFF). ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** <=18 years #### Measurement **Group ID:** BG000 **Value:** 183 #### Measurement **Group ID:** BG001 **Value:** 97 #### Measurement **Group ID:** BG002 **Value:** 280 **Category Title:** Between 18 and 65 years #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** >=65 years **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 9.7 **Value:** 45.7 #### Measurement **Group ID:** BG001 **Spread:** 10.6 **Value:** 45.6 #### Measurement **Group ID:** BG002 **Spread:** 10 **Value:** 45.7 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 165 #### Measurement **Group ID:** BG001 **Value:** 83 #### Measurement **Group ID:** BG002 **Value:** 248 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 18 #### Measurement **Group ID:** BG001 **Value:** 14 #### Measurement **Group ID:** BG002 **Value:** 32 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 142 #### Measurement **Group ID:** BG001 **Value:** 77 #### Measurement **Group ID:** BG002 **Value:** 219 **Class Title:** United States #### Measurement **Group ID:** BG000 **Value:** 41 #### Measurement **Group ID:** BG001 **Value:** 20 #### Measurement **Group ID:** BG002 **Value:** 61 **Class Title:** Australia **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 ## Results Section - More Information Module ### Certain Agreement **Restriction Type:** LTE60 **Restrictive Agreement:** True ### Limitations and Caveats **Description:** Two confounding efficacy factors: Variable hours of therapy in both arms due to inconsistent external power source use and altered vagal excitability in the control arm due to small electrical inputs delivered to vagal nerves for safety checks. ### Point of Contact **Email:** [email protected] **Organization:** EnteroMedics Inc **Phone:** 651-634-3209 **Title:** Senior Vice President of Clinical ## 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:** 2 - **Upper Limit:** - **Value:** 17 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 2 - **Upper Limit:** - **Value:** 16 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 6 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 22 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 25 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Observe at least a 10% greater percentage excess weight loss (%EWL) with vBloc therapy delivered by the Maestro System compared to sham 12 months following randomization using MetLife Method (ideal body weight is calculated based on Metropolitan Height and Weight Tables) **Dispersion Type:** Standard Error **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** Baseline and 1 Year **Title:** Percentage of Excess Weight Loss (EWL) With the Maestro System **Type:** PRIMARY **Unit of Measure:** Percentage of excess weight loss ##### Group **Description:** Subjects implanted with a functional Maestro System device that delivers therapy (Therapy ON). **ID:** OG000 **Title:** vBloc ##### Group **Description:** Subjects implanted with a functional Maestro System device that does NOT deliver therapy (Therapy OFF). **ID:** OG001 **Title:** Placebo #### Outcome Measure 2 **Description:** To estimate the rate of serious, system- and procedure-related adverse events associated with the Maestro System. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** 1 Year **Title:** Rate of System and Procedure-related Serious Adverse Events (SAEs). **Type:** PRIMARY **Unit of Measure:** Events ##### Group **Description:** Subjects implanted with a functional Maestro System device that delivers therapy (Therapy ON). **ID:** OG000 **Title:** vBloc ##### Group **Description:** Subjects implanted with a functional Maestro System device that does NOT deliver therapy (Therapy OFF). **ID:** OG001 **Title:** Placebo #### Outcome Measure 3 **Description:** To evaluate the percentage of participants achieving 25% excess weight loss from baseline (implant) to 12 months between treatment groups. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** Baseline and 1 Year **Title:** Percentage of Participants Achieving 25% Excess Weight Loss (%EWL) **Type:** SECONDARY **Unit of Measure:** Percentage of subjects ##### Group **Description:** Subjects implanted with a functional Maestro System device that delivers therapy (Therapy ON). **ID:** OG000 **Title:** vBloc ##### Group **Description:** Subjects implanted with a functional Maestro System device that does NOT deliver therapy (Therapy OFF). **ID:** OG001 **Title:** Placebo ### Participant Flow Module #### Group **Description:** Subjects implanted with a functional Maestro System device that delivers therapy (Therapy ON). **ID:** FG000 **Title:** vBloc #### Group **Description:** Subjects implanted with a functional Maestro System device that does NOT deliver therapy (Therapy OFF). **ID:** FG001 **Title:** Placebo #### Period **Title:** Overall Study ##### Withdraw **Type:** Adverse Event ###### Reason **Group ID:** FG000 **Number of Subjects:** 7 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 5 ###### Reason **Group ID:** FG001 **Number of Subjects:** 3 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 192 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 102 ##### Milestone **Type:** Surgical Roll-In ###### Achievement **Group ID:** FG000 **Number of Subjects:** 9 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 5 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 178 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 97 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 14 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 5 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT05858879 **Acronym:** NOTIFY-ASCVD **Brief Title:** Notification of Incidental Coronary Artery Calcium in Patients With Atherosclerotic Cardiovascular Disease (NOTIFY-ASCVD) **Official Title:** Notification of Incidental Coronary Artery Calcium in Patients With Atherosclerotic Cardiovascular Disease (NOTIFY-ASCVD) #### Organization Study ID Info **ID:** 70362 #### Organization **Class:** OTHER **Full Name:** Stanford University ### Status Module #### Completion Date **Date:** 2024-12-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-03-12 **Type:** ACTUAL **Last Update Submit Date:** 2024-03-07 **Overall Status:** NOT_YET_RECRUITING #### Primary Completion Date **Date:** 2024-06-30 **Type:** ESTIMATED #### Start Date **Date:** 2024-04-01 **Type:** ESTIMATED **Status Verified Date:** 2024-03 #### Study First Post Date **Date:** 2023-05-15 **Type:** ACTUAL **Study First Submit Date:** 2023-05-05 **Study First Submit QC Date:** 2023-05-05 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Stanford University #### Responsible Party **Investigator Affiliation:** Stanford University **Investigator Full Name:** Fatima Rodriguez **Investigator Title:** Associate Professor of Medicine (Cardiovascular Medicine) **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Estimate the impact of notifying both patients and their clinicians of the presence of incidental coronary artery calcium (CAC) on initiation of lipid-lowering therapy in patients with ASCVD who are not receiving lipid-lowering therapy. **Detailed Description:** This is a randomized quality improvement (QI) project evaluating the impact of notifying patients and their clinicians (primary care, cardiologists, neurologists, or vascular surgeon) of incidental CAC detected on a prior chest CT scan. Patients will be identified by screening previous non-gated chest CT scans and electronic health records. The presence of CAC will be confirmed by a radiologist. Eligible patients will be randomized to: 1) notification of presence of CAC with a CT scan image; 2) notification of presence of CAC without a CT scan image; 3) or usual care. ### Conditions Module **Conditions:** - ASCVD - Coronary Artery Calcification ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** Eligible patients will be randomized to: 1) notification of presence of CAC with a CT scan image; 2) notification of presence of CAC without a CT scan image; 3) or usual care. ##### Masking Info **Masking:** DOUBLE **Masking Description:** In the usual care arm, patients or clinicians will not be notified. **Who Masked:** - PARTICIPANT - CARE_PROVIDER **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 1000 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Notification of presence of CAC with a CT scan image **Intervention Names:** - Other: Notification of patients and clinicians **Label:** Notification with a CAC image **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Notification of presence of CAC without a CT scan image **Intervention Names:** - Other: Notification of patients and clinicians **Label:** Notification without a CAC image **Type:** ACTIVE_COMPARATOR #### Arm Group 3 **Description:** Usual care **Intervention Names:** - Other: Notification of patients and clinicians **Label:** Usual care **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Notification with a CAC image - Notification without a CAC image - Usual care **Description:** Notification Patients randomized to notification will receive a message sent by either the electronic health record (EHR) patient portal or US mail that will inform them of the CAC identified on their previous chest CT scan and a recommendation that they discuss this finding and initiation of lipid-lowering therapy with their clinician. The clinicians will be notified of these findings by an earlier EHR message that will be sent 2 weeks before the patient notification. Usual Care The usual care arm will not receive any additional notification beyond this standard of care. **Name:** Notification of patients and clinicians **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Number of patients who had initiation of lipid-lowering therapy **Measure:** Initiation of lipid-lowering therapy **Time Frame:** Month 6 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Presence of CAC on non-gated chest CT scans performed from 2021 to 2023 2. Age \<85 years 3. Diagnosis of coronary artery disease, peripheral artery disease, or cerebrovascular disease 4. Visit to Stanford affiliated clinician since 2021 at one of the following Stanford clinics including University affiliated clinics: 1. Internal Medicine 2. Family Medicine 3. Cardiology 4. Neurology 5. Vascular surgery Exclusion Criteria: 1. No diagnosis of ASCVD 2. Patients receiving lipid-lowering therapy 3. Dementia 4. Metastatic cancer or active cancer undergoing chemotherapy **Maximum Age:** 84 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Fahim Abbasi, MD **Phone:** 650-624-0954 **Role:** CONTACT **Contact 2:** **Name:** Sarah Magee **Role:** CONTACT #### Overall Officials **Official 1:** **Affiliation:** Stanford University **Name:** Fatima Rodriguez, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - 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: BC18 - Name: Nutritional and Metabolic Diseases ### Condition Browse Module - Browse Leaves - ID: M26188 - Name: Atherosclerosis - Relevance: HIGH - As Found: Atherosclerotic Cardiovascular Disease - ID: M5377 - Name: Calcinosis - 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: D000002318 - Term: Cardiovascular Diseases - ID: D000050197 - Term: Atherosclerosis ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: BDCA - Name: Bone Density Conservation Agents ### Intervention Browse Module - Browse Leaves - ID: M5381 - Name: Calcium - Relevance: LOW - As Found: Unknown - ID: M5398 - Name: Calcium, Dietary - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03221179 **Brief Title:** Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of RO7049665 in Healthy Volunteers **Official Title:** A Randomized, Adaptive, Investigator/ Subject Blind, Single Ascending Dose, Placebo-Controlled Phase I Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered RO7049665 in Healthy Volunteers #### Organization Study ID Info **ID:** WP39826 #### Organization **Class:** INDUSTRY **Full Name:** Hoffmann-La Roche ### Status Module #### Completion Date **Date:** 2019-07-05 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2019-08-13 **Type:** ACTUAL **Last Update Submit Date:** 2019-08-09 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2019-07-05 **Type:** ACTUAL #### Start Date **Date:** 2017-07-10 **Type:** ACTUAL **Status Verified Date:** 2019-08 #### Study First Post Date **Date:** 2017-07-18 **Type:** ACTUAL **Study First Submit Date:** 2017-07-07 **Study First Submit QC Date:** 2017-07-14 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Hoffmann-La Roche #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** The primary objective of this study is to evaluate the safety and tolerability of single ascending doses of subcutaneous (SC) injections of RO7049665 in healthy volunteers. In addition, pharmacokinetics (PK) of RO7049665, the effects of single doses of RO7049665 on regulatory T-cells as well as the single dose immunogenicity of RO7049665 will be evaluated. This trial plans to evaluate approximately seven single dose-levels of RO7049665 or matching-placebo during dose-escalation in approximately 40 participants. ### Conditions Module **Conditions:** - Healthy Volunteers ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** SEQUENTIAL ##### Masking Info **Masking:** TRIPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 49 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants will be administered a single SC dose of RO7049665 administered in up to 4 SC injection. **Intervention Names:** - Biological: RO7049665 **Label:** RO7049665 **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants will be administered a single SC dose of matching placebo formulation administered in up to 4 SC injections. **Intervention Names:** - Biological: Placebo **Label:** Placebo **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - RO7049665 **Description:** A single ascending dose (starting dose 1.5 micrograms \[mcg\])of RO7049665 will be administered SC. **Name:** RO7049665 **Type:** BIOLOGICAL #### Intervention 2 **Arm Group Labels:** - Placebo **Description:** Matching placebo will be administered SC once. **Name:** Placebo **Type:** BIOLOGICAL ### Outcomes Module #### Primary Outcomes **Description:** An adverse event is any untoward medical occurrence in a participant administered a pharmaceutical product and which does not necessarily have to have a causal relationship with the treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding, for example), symptom, or disease temporally associated with the use of a pharmaceutical product, whether or not considered related to the pharmaceutical product. Preexisting conditions which worsen during a study are also considered as adverse events. **Measure:** Percentage of Participants with Adverse Events **Time Frame:** From baseline up to approximately 8 weeks #### Secondary Outcomes **Description:** Blood samples will be taken pre-dose and at multiple time points after dose administration. RO7049665 concentration will be analyzed in serum and the time from SC injection to maximum concentration of RO7049665 will be determined. **Measure:** PK: Time to Maximum Concentration (Tmax) of RO7049665 **Time Frame:** Pre-dose on Day 1, post-dose on Day 1 at 2 hours (h), 6 h and 12 h, once daily on Days 2-8, Day 12, Day 15, Day 21, Day 29 and Day 43 **Description:** Blood samples will be taken pre-dose and at multiple time points after dose administration. RO7049665 concentration will be analyzed in serum and the maximum concentration of RO7049665 will be determined. **Measure:** PK: Maximum Serum Concentration Observed (Cmax) of RO7049665 **Time Frame:** Pre-dose on Day 1, post-dose on Day 1 at 2 hours (h), 6 h and 12 h, once daily on Days 2-8, Day 12, Day 15, Day 21, Day 29 and Day 43 **Description:** Blood samples will be taken pre-dose and at multiple time points after dose administration. RO7049665 concentration will be analyzed in serum. AUC from time of drug administration to last blood sampling (AUClast) will be determined in a plot of RO7049665 serum concentration versus time. **Measure:** PK: Area Under the Concentration-Time Curve (AUC) from Time 0 to Time of Last Sampling of RO7049665 **Time Frame:** Pre-dose on Day 1, post-dose on Day 1 at 2 hours (h), 6 h and 12 h, once daily on Days 2-8, Day 12, Day 15, Day 21, Day 29 and Day 43 **Description:** Blood samples will be taken pre-dose and at multiple time points after dose administration. RO7049665 concentration will be analyzed in serum. AUC from time of drug administration extrapolated to infinity (AUCinf) will be determined in a plot of RO7049665 serum concentration versus time. **Measure:** PK: AUC from Time 0 to infinity (AUCinf) of RO7049665 **Time Frame:** Pre-dose on Day 1, post-dose on Day 1 at 2 hours (h), 6 h and 12 h, once daily on Days 2-8, Day 12, Day 15, Day 21, Day 29 and Day 43 **Description:** Blood samples will be taken pre-dose and at multiple time points after dose administration. RO7049665 concentration will be analyzed in serum. AUC from time of drug administration to time tau (AUC0-t), which is defined as the time of last measurable serum concentration, will be determined in a plot of RO7049665 serum concentration versus time. **Measure:** PK: AUC from Time 0 to Time tau (AUC0-t) of RO7049665 **Time Frame:** Pre-dose on Day 1, post-dose on Day 1 at 2 hours (h), 6 h and 12 h, once daily on Days 2-8, Day 12, Day 15, Day 21, Day 29 and Day 43 **Description:** Blood samples will be taken pre-dose and at multiple time points after dose administration. RO7049665 concentration will be analyzed in serum. Clearance, which is a measure of the rate at which a drug is metabolized or eliminated, will be determined. **Measure:** PK: Apparent Clearance (CL/F) of RO7049665 **Time Frame:** Pre-dose on Day 1, post-dose on Day 1 at 2 hours (h), 6 h and 12 h, once daily on Days 2-8, Day 12, Day 15, Day 21, Day 29 and Day 43 **Description:** Blood samples will be taken pre-dose and at multiple time points after dose administration. RO7049665 concentration will be analyzed in serum. Volume of distribution of RO7049665 will be determined. **Measure:** PK: Apparent Volume of Distribution (Vz/F) of RO7049665 **Time Frame:** Pre-dose on Day 1, post-dose on Day 1 at 2 hours (h), 6 h and 12 h, once daily on Days 2-8, Day 12, Day 15, Day 21, Day 29 and Day 43 **Description:** Blood samples will be taken pre-dose and at multiple time points after dose administration. RO7049665 concentration will be analyzed in serum. t1/2 is the time required for the serum concentration of RO7049665 to be reduced to half. **Measure:** PK: Half-life (t1/2) of RO7049665 **Time Frame:** Pre-dose on Day 1, post-dose on Day 1 at 2 hours (h), 6 h and 12 h, once daily on Days 2-8, Day 12, Day 15, Day 21, Day 29 and Day 43 **Description:** Blood samples will be taken for flow cytometry and peripheral blood mononuclear cell (PBMC) isolation. Markers for quantification of Tregs will be assessed. **Measure:** Change from Baseline in Regulatory T Lymphocyte (Tregs) Count **Time Frame:** Day -1, Pre-dose Day 1; post-dose Day 2, 3, 4, 6, 8, 12, 15, 29, up to follow-up visit (Day 57) **Description:** Anti-drug antibody assays will be used to detect anti-drug antibodies against RO7049665. Samples which are positive for anti-drug antibodies will be further assessed using a neutralizing antibody assay. **Measure:** Percentage of Participants with Anti-Drug Antibodies **Time Frame:** Pre-dose Day 1, post-dose Day 8, 15, 29, up to follow-up visit (Day 57) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Male healthy volunteers, 18 to 45 years of age, inclusive; * Absence of evidence of any active or chronic disease; * Body mass index (BMI) of 18-30 kilograms per square meter (kg/m\^2), inclusive; * Contraception requirements: refrain from heterosexual intercourse or use contraceptive measures, and agreement to refrain from donating sperm. Exclusion Criteria: * History of any clinically significant gastrointestinal, renal, hepatic, broncho-pulmonary, neurological, psychiatric, cardio-vascular, endocrinological, hematological or allergic disease, metabolic disorder, cancer or cirrhosis; * Clinically significant abnormalities (as judged by the Investigator) in laboratory test results; * Concomitant disease or condition that could interfere with, or treatment of which might interfere with, the conduct of the study, or that would, in the opinion of the Investigator, pose an unacceptable risk to the participant in this study; * History of hypersensitivity to biologic agents or any of the excipients in the formulation; * Any abnormal skin conditions or potentially obscuring tattoos, pigmentation or lesions in the area intended for subcutaneous injection; * Prior administration of aldesleukin, or interleukin-2 (IL-2) derivatives. **Healthy Volunteers:** True **Maximum Age:** 45 Years **Minimum Age:** 18 Years **Sex:** MALE **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Zuidlaren **Country:** Netherlands **Facility:** PRA Health Sciences Early Development Services **Zip:** 9471 GP #### Overall Officials **Official 1:** **Affiliation:** Hoffmann-La Roche **Name:** Clinical Trials **Role:** STUDY_DIRECTOR ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05493579 **Brief Title:** Impact of Implant Supported Overdenture on Changes of Electromyographic and Brain Activity **Official Title:** "Impact of Mandibular Implant Supported Overdenture on Changes of Electromyographic Activity, Brain Activity, Cognitive Function, Nutrition and Depression Status." #### Organization Study ID Info **ID:** REC-PR-21-08 #### Organization **Class:** OTHER **Full Name:** Al-Azhar University ### Status Module #### Completion Date **Date:** 2021-07-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2022-08-09 **Type:** ACTUAL **Last Update Submit Date:** 2022-08-07 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2021-04-01 **Type:** ACTUAL #### Start Date **Date:** 2020-10-01 **Type:** ACTUAL **Status Verified Date:** 2022-01 #### Study First Post Date **Date:** 2022-08-09 **Type:** ACTUAL **Study First Submit Date:** 2022-01-09 **Study First Submit QC Date:** 2022-08-07 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Al-Azhar University #### Responsible Party **Investigator Affiliation:** Misr International University **Investigator Full Name:** Noha Taha Kamel Taha Alloush **Investigator Title:** Assistant Lecturer **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of this study is to evaluate and compare the effects of a mandibular implant-supported overdenture with a mandibular complete denture on electromyographic activity, brain activity, and cognitive performance in edentulous patients. Ten patients who are entirely edentulous and did not wear dentures will be chosen. To assess brain activity and cognitive function, electroencephalograms, the Mini-Mental State Examination (MMSE), and electromyographic examinations of the temporalis and masseter muscles will be performed prior to complete denture construction, one and three months after complete denture construction, and one and three months after implant supported overdenture insertion, respectively. ### Conditions Module **Conditions:** - Implant Supported Overdenture ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### 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:** - Other: Implant supported overdenture **Label:** Complete denture then complete implant supported overdenture **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Complete denture then complete implant supported overdenture **Description:** Implant supported overdenture compared to complete denture **Name:** Implant supported overdenture **Type:** OTHER ### Outcomes Module #### Other Outcomes **Description:** The highest EMG activity in the masseter muscle and the anterior temporalis muscle will be calculated during the maximum clenching, maximal voluntary contraction (MVC) in the intercuspal position. The EMG signals will be stored and analyzed as root mean square (RMS) values expressed in microvolts (μV) and the mean amplitude of the motor unit potential (MUP) will be calculated in microvolts (μV). **Measure:** Muscle Activity (EMG) **Time Frame:** 3 months #### Primary Outcomes **Description:** An assessment of the patient's brain activity will be done. The waves obtained during all phases of assessment will be analyzed, and the software will separate the alpha waves, which occur in the frequency range of 8-12 Hz, evaluating their amplitude. The mean amplitude (in microvolts) of alpha waves was obtained. **Measure:** Brain Activity (EEG) **Time Frame:** 3 months #### Secondary Outcomes **Description:** Cognitive function will be assessed using the Mini-Mental State Examination (MMSE) questionnaire. The MMSE set, with a total score of 30, assesses different domains of cognitive function. The MMSE comprises 30 questions, with 10 devoted to orientation; three items requiring registration of new information; five questions addressing attention and calculation; three recall items; eight items assessing language skills; and one construction question. Any score of 24 or more (out of 30) indicates normal cognition. Scores below this indicate severe (9 points), moderate (10-18 points), or mild (19-23 points) cognitive impairment. **Measure:** Cognitive Function (MMSE) **Time Frame:** 3 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * All patients' ridges should be covered with firm mucosa which is free from any signs of inflammation or ulceration. * Patients should be free from any bone disorder and exhibit adequate height and width of the residual alveolar ridge. * All patients must have sufficient inter arch space. Exclusion Criteria: * Patients with oral or systemic diseases. * Patients with xerostomia or excessive salivation. * Patients with parafunctional habits (bruxism or clenching). * Heavy smoker or alcoholic patients. * Patients with history of temporo-mandibular dysfunction. * Patients with neurological or psychiatric disorders. **Healthy Volunteers:** True **Maximum Age:** 69 Years **Minimum Age:** 65 Years **Sex:** ALL **Standard Ages:** - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Cairo **Country:** Egypt **Facility:** Faculty of Dentistry. AL Azhar University for Girls, Cairo, Egypt. ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ## 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: M7058 - Name: Depression - Relevance: LOW - As Found: Unknown - ID: M7061 - Name: Depressive Disorder - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05511779 **Brief Title:** Study to Confirm of the Safety and Tolerability of Brincidofovir in Subjects With BK Virus Infection (Viremia) After Kidney Transplantation **Official Title:** Phase II, Open-label, Randomized, Multiple Ascending Dose Confirmation of the Safety and Tolerability of Brincidofovir in Subjects With BK Virus Infection (Viremia) After Kidney Transplantation (BASTION) #### Organization Study ID Info **ID:** BCV-BN01 #### Organization **Class:** INDUSTRY **Full Name:** SymBio Pharmaceuticals ### Status Module #### Completion Date **Date:** 2025-02-16 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-02-28 **Type:** ACTUAL **Last Update Submit Date:** 2023-02-26 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-12-16 **Type:** ESTIMATED #### Start Date **Date:** 2022-10-14 **Type:** ACTUAL **Status Verified Date:** 2022-11 #### Study First Post Date **Date:** 2022-08-23 **Type:** ACTUAL **Study First Submit Date:** 2022-08-16 **Study First Submit QC Date:** 2022-08-22 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** SymBio Pharmaceuticals #### 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 is a Phase II, multicenter, open-label, randomized, standard of care (SOC) controlled, multiple ascending dose study to assess the safety and tolerability of IV Brincidofovir (BCV) in subjects with BKV infection after kidney transplantation. The study will be conducted at multiple study sites in several countries including Australia and Japan. Subjects who meet eligibility criteria will be enrolled in the study and will be randomized and assigned to BCV or SOC (defined as use of the same immunosuppressant administered during prescreening) before receipt of the first dose of study drug in both the Dose Escalation Phase and the Expansion Phase. ### Conditions Module **Conditions:** - BK Virus Infection - Nephropathy - Kidney Transplantation **Keywords:** - BK Virus - Nephropathy - Kidney transplantation ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** SEQUENTIAL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 36 **Type:** ESTIMATED **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** BCV: 0.3 mg/kg administered as a continuous IV infusion over 2 hours on Day1 and Day4 for 8 weeks (up to a maximum of 14 weeks). **Intervention Names:** - Drug: Brincidofovir **Label:** Dose Escalation Phase: Cohort 1: BCV 0.3 mg/kg BIW **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** BCV: 0.4 mg/kg administered as a continuous IV infusion over 2 hours on Day1 and Day4 for 8 weeks (up to a maximum of 14 weeks). **Intervention Names:** - Drug: Brincidofovir **Label:** Dose Escalation Phase: Cohort 2: BCV 0.4 mg/kg BIW **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** BCV: Recommended dosage administered as a continuous IV infusion over 2 hours on Day1 and Day4 for 8 weeks (up to a maximum of 14 weeks). **Intervention Names:** - Drug: Brincidofovir **Label:** Expansion Phase: BCV Recommended dosage regimen in the Dose Escalation Phase **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Dose Escalation Phase: Cohort 1: BCV 0.3 mg/kg BIW - Dose Escalation Phase: Cohort 2: BCV 0.4 mg/kg BIW - Expansion Phase: BCV Recommended dosage regimen in the Dose Escalation Phase **Description:** BCV 0.3 mg/kg BIW or 0.4 mg/kg BIW administered as a continuous IV infusion over 2 hours **Name:** Brincidofovir **Other Names:** - SyB V-1901 - BCV **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** * Incidence of TEAEs of the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Grade 3 severity and serious adverse events * Incidence of treatment-related TEAEs * Incidence of adverse events (AEs) requiring permanent discontinuation of BCV * Absolute and changes over time in safety laboratory parameters (ie, hematology, blood chemistry, and urinalysis) **Measure:** Incidence of treatment-emergent adverse events (TEAEs) **Time Frame:** from the time of administration of the first dose of study drug through the follow-up visit(up to 14 weeks (treatment period) and 30 days (follow-up period)) **Description:** Change from baseline in BK viral load in plasma measured through follow-up for each subject. Change from baseline in BK viral load in urine measured through follow-up for each subject. Peak BK viral load in plasma from Week 2 Day 1 through follow-up for each subject. Time-averaged area under the viremia-time curve for BK viral load in plasma from baseline through follow-up for each subject. **Measure:** Antiviral Effects **Time Frame:** From baseline to follow-up visit(up to 14 weeks (treatment period) and 30 days (follow-up period)) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Male or female, at least 18 years of age at the time of signing the informed consent at screening. * Kidney transplant recipient. "BK viral load increase and ≥ 3.6 log IU/mL" at 2 weeks post immunosuppression reduction or "BK viral load does not decrease by ≥ 0.3 log IU/mL" at 4 weeks post immunosuppression reduction during prescreening. (Note: Immunosuppressant reduction needs to be continued during the screening period). * eGFR ≥ 30 mL/min. * Subjects under immunosuppression with tacrolimus, MMF/Myfortic, and/or corticosteroid. Exclusion Criteria: * Subjects who weigh ≥ 120 kg. * National Institutes of Health/NCI CTCAE Grade 2 or higher diarrhea (ie, increase of ≥ 4 stools per day over usual pretransplant stool output) within 7 days before Day 1. * Poor clinical prognosis, including active malignancy or use of vasopressors other than low dose (eg, ≤ 5 μg/kg/min) dopamine for renal perfusion within 7 days before Day 1. * Use of renal replacement therapy within 7 days before Day 1. * History of intolerance to cidofovir or related compounds (ie, other nucleotide derivatives \[adefovir or tenofovir\]) **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Yuji Hoshino **Phone:** +81-3-6684-6616 **Role:** CONTACT #### Locations **Location 1:** **City:** Adelaide **Country:** Australia **Facility:** Royal Adelaide Hospital **Status:** RECRUITING **Location 2:** **City:** Melbourne **Country:** Australia **Facility:** Austin Health **Status:** RECRUITING **Location 3:** **City:** Melbourne **Country:** Australia **Facility:** The Royal Melbourne Hospital **Status:** NOT_YET_RECRUITING **Location 4:** **City:** Hachiōji **Country:** Japan **Facility:** Tokyo Medical University Hachioji Medical Center **Status:** NOT_YET_RECRUITING **Location 5:** **City:** Nagoya **Country:** Japan **Facility:** Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital **Status:** RECRUITING **Location 6:** **City:** Osaka **Country:** Japan **Facility:** Osaka General Medical Center **Status:** RECRUITING **Location 7:** **City:** Osaka **Country:** Japan **Facility:** Osaka Metropolitan University Hospital **Status:** NOT_YET_RECRUITING **Location 8:** **City:** Sapporo **Country:** Japan **Facility:** Hokkaido University Hospital **Status:** RECRUITING **Location 9:** **City:** Sapporo **Country:** Japan **Facility:** Sapporo City General Hospital **Status:** NOT_YET_RECRUITING **Location 10:** **City:** Shimotsuke **Country:** Japan **Facility:** Jichi Medical University Hospital **Status:** NOT_YET_RECRUITING **Location 11:** **City:** Shinjuku-ku **Country:** Japan **Facility:** Tokyo Women's Medical University Hospital **Status:** NOT_YET_RECRUITING **Location 12:** **City:** Suita **Country:** Japan **Facility:** Osaka University Hospital **Status:** NOT_YET_RECRUITING **Location 13:** **City:** Toyoake **Country:** Japan **Facility:** Fujita Health University Hospital **Status:** RECRUITING **Location 14:** **City:** Yokohama **Country:** Japan **Facility:** Yokohama City University Medical Center **Status:** RECRUITING #### Overall Officials **Official 1:** **Affiliation:** SymBio Pharmaceuticals Limited **Name:** Carolyn Yanavich **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020969 - Term: Disease Attributes - ID: D000010335 - Term: Pathologic Processes - 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 - ID: D000018805 - Term: Sepsis - ID: D000018746 - Term: Systemic Inflammatory Response Syndrome - ID: D000007249 - Term: Inflammation ### Condition Browse Module - Browse Branches - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: All - Name: All Conditions - Abbrev: BC01 - Name: Infections - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M10698 - Name: Kidney Diseases - Relevance: HIGH - As Found: Nephropathy - ID: M10283 - Name: Infections - Relevance: HIGH - As Found: Infection - ID: M6368 - Name: Communicable Diseases - Relevance: HIGH - As Found: Infection - ID: M17522 - Name: Virus Diseases - Relevance: HIGH - As Found: Virus Infection - ID: M17511 - Name: Viremia - Relevance: HIGH - As Found: Viremia - ID: M22700 - Name: Disease Attributes - 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 - ID: M20864 - Name: Sepsis - Relevance: LOW - As Found: Unknown - ID: M16869 - Name: Toxemia - 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: D000007239 - Term: Infections - ID: D000003141 - Term: Communicable Diseases - ID: D000014777 - Term: Virus Diseases - ID: D000014766 - Term: Viremia - ID: D000007674 - Term: Kidney Diseases ### Intervention Browse Module - Ancestors - ID: D000000998 - Term: Antiviral Agents - 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: M351625 - Name: Brincidofovir - Relevance: HIGH - As Found: Renal Stones - ID: M4314 - Name: Antiviral Agents - Relevance: LOW - As Found: Unknown - ID: M4214 - Name: Anti-Infective Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: C000525733 - Term: Brincidofovir ### Misc Info Module #### Removed Countries - Country: Korea, Republic of - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02341079 **Brief Title:** Intraoperative Liposomal Bupivacaine Injection in Primary Total Knee Arthroplasty **Official Title:** Prospective Randomized Trial Comparing Intraoperative Liposomal Bupivacaine Injection With Indwelling Femoral Nerve Blockade in Early Postoperative Pain Control for Primary Total Knee Arthroplasty #### Organization Study ID Info **ID:** NMCSD.2014.0101 #### Organization **Class:** FED **Full Name:** United States Naval Medical Center, San Diego ### Status Module #### Completion Date **Date:** 2015-10 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2017-03-10 **Type:** ACTUAL **Last Update Submit Date:** 2017-03-07 **Overall Status:** WITHDRAWN #### Primary Completion Date **Date:** 2015-10 **Type:** ESTIMATED #### Start Date **Date:** 2015-03 **Status Verified Date:** 2017-03 #### Study First Post Date **Date:** 2015-01-19 **Type:** ESTIMATED **Study First Submit Date:** 2015-01-14 **Study First Submit QC Date:** 2015-01-16 **Why Stopped:** Principal no longer interested in conducting study ### Sponsor Collaborators Module #### Lead Sponsor **Class:** FED **Name:** United States Naval Medical Center, San Diego #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of the study is to evaluate the efficacy of local infiltration of liposomal bupivacaine versus use of an indwelling femoral peripheral nerve block in controlling early postoperative pain in primary total knee arthroplasty. The investigators hope to demonstrate equivalency of treatment modalities to provide an alternative to the commonly used treatment of femoral nerve blockade. **Detailed Description:** Postoperative pain control in total knee arthroplasty is generally managed by a multimodal approach of premedication, epidural and/or peripheral nerve blocks, narcotic medications, and anti-inflammatories. Multiple studies have evaluated the efficacy of anesthetic "cocktails" of various medications for local tissue infiltration to aide in controlling postoperative pain. A recent systematic review supported the use of local anesthetic infiltration in a single intraoperative dose as an adjunct to femoral nerve block in the relief of pain; with combinations of ropivacaine, ketorolac, and adrenaline providing the best results. Several studies noted decreases in opioid consumption and overall pain scores in the perioperative period. A novel compound, liposomal bupivacaine (EXPAREL®, Pacira Pharmaceuticals, Inc., Parsippany, NJ, USA), has been proposed for use in total knee arthroplasty. To our knowledge, only one randomized controlled trial involving use of this compound in total knee arthroplasty has been published and was performed as a phase two dose ranging trial comparing liposomal bupivacaine to bupivacaine HCl. No statistically significant difference between the two compounds was shown, but a trend towards benefit in short term pain control was shown with EXPAREL 532mg. Our current pain management regimen utilizes the multimodal approach of neuraxial anesthesia, indwelling femoral nerve catheter, opioids, and anti-inflammatory medications. The regional anesthesia service manages postoperative pain while the indwelling femoral nerve catheter is in place. The epidural is utilized for early postoperative analgesia and thromboprophylaxis. The epidural is discontinued on post-operative day 1 and the femoral nerve catheter is removed by the morning of postoperative day 3. Although femoral nerve catheters provide excellent pain control following TKA, there are associated disadvantages. Most notably there is inherent quadriceps weakness which places the patient at increased fall risk. To reduce the risk of falling, patients must wear a knee immobilizer with ambulation until the catheter is removed and quadriceps function has returned. This may delay the ability to actively participate in physical therapy. The purpose of our present study is to evaluate the efficacy of local infiltration of liposomal bupivacaine versus use of an indwelling femoral peripheral nerve block in controlling early postoperative pain. The investigators aim to show equivalency between the two treatment modalities. Our hypothesis is that a systematic local infiltration of liposomal bupivacaine provided intraoperatively is equally efficacious as femoral indwelling peripheral nerve blockade in immediate postoperative pain control with more rapid progression with physical therapy. ### Conditions Module **Conditions:** - Osteoarthritis - Osteoarthritis, Knee - Arthritis, Degenerative - Osteoarthrosis - Osteoarthrosis Deformans **Keywords:** - Total Knee Arthroplasty - Osteoarthritis - Femoral Nerve Blockade - Liposomal Bupivacaine ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Type:** ACTUAL **Phases:** - PHASE2 - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Femoral nerve block delivered via indwelling femoral nerve catheter **Intervention Names:** - Drug: Indwelling femoral nerve block **Label:** Femoral Nerve Blockade **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Intraoperative intracapsular injection of bupivacaine liposome **Intervention Names:** - Drug: Bupivacaine Liposome Injection **Label:** Bupivacaine Liposome Injection **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Bupivacaine Liposome Injection **Description:** Intraoperative intracapsular injection of 266mg of bupivacaine liposome injection diluted to 60mL with 0.9% normal saline. **Name:** Bupivacaine Liposome Injection **Other Names:** - EXPAREL **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Femoral Nerve Blockade **Description:** Femoral nerve block delivered via indwelling femoral nerve catheter with 0.125% bupivacaine. **Name:** Indwelling femoral nerve block **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** 10cm VAS scale completed by the patient at set intervals postoperatively **Measure:** Visual Analog Pain Scale **Time Frame:** 0-3 months **Description:** Oral and intravenous opioid use calculated in terms of morphine equivalents **Measure:** Oral and Intravenous Opioid Consumption **Time Frame:** 0-5 days (during hospitalization) **Description:** Standardized functional clinical score for knee symptoms **Measure:** Hospital for Special Surgery (HSS) Knee Scores **Time Frame:** 0-3 months #### Secondary Outcomes **Measure:** Length of Hospital Stay **Time Frame:** 0-5 days **Measure:** Distance ambulated with physical therapy **Time Frame:** 0-5 days (during hospitalization) **Description:** Time to return of quadriceps function **Measure:** Time to straight leg raise with physical therapy **Time Frame:** 0-5 days (during hospitalization) **Description:** Knee range of motion measure by physical therapy and in clinic **Measure:** Postoperative range of motion **Time Frame:** 0-3 months **Description:** All intervention related complications for each arm of the study **Measure:** Postoperative complication rate **Time Frame:** 0-3 months **Description:** Plasma levels of bupivacaine monitored throughout hospital stay **Measure:** Plasma bupivacaine concentrations **Time Frame:** 0-5 days (during hospitalization) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Age over 18 years old * Undergoing unilateral primary total knee arthroplasty Exclusion Criteria: * Revision total knee arthroplasty * Bilateral total knee arthroplasty * Prior allergy or adverse reaction to local anesthetic * Hepatic dysfunction **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** San Diego **Country:** United States **Facility:** Naval Medical Center San Diego **State:** California **Zip:** 92134 #### Overall Officials **Official 1:** **Affiliation:** Naval Medical Center San Diego Department of Orthopadic Surgery **Name:** Patrick B Morrissey, M.D. **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000001168 - Term: Arthritis - ID: D000007592 - Term: Joint Diseases - ID: D000009140 - Term: Musculoskeletal Diseases - ID: D000012216 - Term: Rheumatic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases ### Condition Browse Module - Browse Leaves - ID: M12926 - Name: Osteoarthritis - Relevance: HIGH - As Found: Osteoarthritis - ID: M22168 - Name: Osteoarthritis, Knee - Relevance: HIGH - As Found: Osteoarthritis, Knee - ID: M13069 - Name: Pain, Postoperative - Relevance: LOW - As Found: Unknown - ID: M13066 - Name: Pain - Relevance: LOW - As Found: Unknown - ID: M4476 - Name: Arthritis - 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 - ID: M15045 - Name: Rheumatic Diseases - Relevance: LOW - As Found: Unknown - ID: M6323 - Name: Collagen Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000010003 - Term: Osteoarthritis - ID: D000020370 - Term: Osteoarthritis, Knee ### Intervention Browse Module - Ancestors - ID: D000000779 - Term: Anesthetics, Local - ID: D000000777 - Term: Anesthetics - ID: D000002492 - Term: Central Nervous System Depressants - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018689 - Term: Sensory System Agents - ID: D000018373 - Term: Peripheral Nervous System Agents ### 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: M5315 - Name: Bupivacaine - Relevance: HIGH - As Found: Following - ID: M4107 - Name: Anesthetics - Relevance: LOW - As Found: Unknown - ID: M4109 - Name: Anesthetics, Local - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000002045 - Term: Bupivacaine ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06185179 **Brief Title:** Metformin and Muscle Recovery **Official Title:** Use of Metformin to Improve Muscle Regrowth in Older Adults #### Organization Study ID Info **ID:** 086328 #### Organization **Class:** OTHER **Full Name:** University of Utah ### Status Module #### Completion Date **Date:** 2030-07-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-01-03 **Type:** ACTUAL **Last Update Submit Date:** 2023-12-28 **Overall Status:** NOT_YET_RECRUITING #### Primary Completion Date **Date:** 2029-07-31 **Type:** ESTIMATED #### Start Date **Date:** 2024-09-01 **Type:** ESTIMATED **Status Verified Date:** 2023-12 #### Study First Post Date **Date:** 2023-12-29 **Type:** ACTUAL **Study First Submit Date:** 2023-12-13 **Study First Submit QC Date:** 2023-12-14 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Utah #### Responsible Party **Investigator Affiliation:** University of Utah **Investigator Full Name:** Micah Drummond **Investigator Title:** Professor, Physical Therapy and Athletic Training **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True ### Description Module **Brief Summary:** A hallmark of aging is an impaired ability to adequately recover following a stressor, such as muscle disuse, resulting in muscle fibrosis and weakness thereby increasing the risk for falls and loss of independence. Mechanistic-based therapeutic strategies to enhance muscle recovery in older adults do not exist. Metformin has been implicated to have positive effects on muscle size and function through non-glycemic mechanisms. Metformin has been shown to enhance macrophage function and lessen cellular senescence burden by targeting SASP in a variety of muscle interstitial cells. However, the role of metformin to improve muscle recovery in older adults following disuse atrophy through immunomodulating and senomorphic mechanisms have not been examined. Therefore, the purpose of this study is to conduct a randomized, double blind, placebo-controlled clinical trial in older adult participants to determine if short-term metformin delivery (vs placebo) during the recovery phase following disuse atrophy can improve muscle regrowth. **Detailed Description:** Aging is associated with impaired muscle recovery following disuse atrophy. If not addressed, restricted muscle regrowth and function may lead to a cascade of health crisis events for the aged individual (falls, disability, metabolic diseases). Clinically adopted treatments that promote muscle recovery, particularly in elderly patients, do not exist. The long-term goal of this project is to develop mechanistic-based therapeutic approaches to accelerate muscle recovery following disuse atrophy in older adults. Macrophages are well understood to play a requisite role in myofiber remodeling including removal of debris and senescent cells, promotion of stem/satellite cell proliferation and differentiation, and regulation fibro-adipogenic progenitor cell (FAP) dynamics and fate. It is known that aged muscle during recovery have an impaired macrophage inflammatory and functional response complimented with poor muscle recovery. The investigators posit that age-related local immune dysfunction disrupts extracellular matrix (ECM) remodeling following muscle disuse by reducing the removal of senescence cells resulting in their accumulation. Therefore, treatments are needed to improve macrophage function and mitigate cellular senescence to facilitate muscle cellular remodeling events (e.g., macrophage, satellite cells, FAPs) during recovery following disuse in aging. Metformin has gained traction to be repurposed as a treatment for a broad range of age-related diseases but its role in regulating muscle regrowth following disuse atrophy in humans is not known. Metformin has shown to modulate inflammatory profiles, mitigate cellular senescence and SASP (e.g., macrophages, FAPs, and myoblasts) while also improving muscle regeneration and satellite cell function in injured mice. Therefore, metformin treatment could have therapeutic value to temper unwanted collagen deposition and promote muscle regrowth and function when strategically delivered during the muscle regrowth phase in aging by targeting immune cell function and cellular senescence and SASP. The overarching hypothesis is that metformin would mitigate cellular senescence and SASP specifically in macrophages, FAPs and satellite cells and would correspond to enhanced muscle function following disuse atrophy. The investigators will conduct a randomized, placebo-controlled clinical trial in healthy older participants to test if metformin provided during a 14d recovery phase following unilateral limb immobilization would improve muscle macrophage, FAP and satellite cell function, reduce cellular senescence and muscle fibrosis. ### Conditions Module **Conditions:** - Muscle Atrophy or Weakness ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** 2-weeks single leg immobilization followed by 2-weeks of recovery ##### Masking Info **Masking:** DOUBLE **Masking Description:** Metformin and Placebo pills will be encapsulated by the pharmacy in order to protect treatment assignment **Who Masked:** - PARTICIPANT - INVESTIGATOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 50 **Type:** ESTIMATED **Phases:** - EARLY_PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Metformin pills will be given orally and daily during the 2-week recovery period following single leg immobilization Metformin will be titrated up to a dose 2g/day. 1g dose (two 500 mg pills) will be taken in the morning and a 1g dose will be taken in the evening. This will take place over each day for 14 days during the recovery period. **Intervention Names:** - Drug: Metformin **Label:** Metformin **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Placebo pills will be given orally and daily during the 2-week recovery period following single leg immobilization. Two pills will be taken in the morning and two pills will be taken in the evening. This will take place over each day for 14 days during the recovery period. **Intervention Names:** - Other: Placebo **Label:** Placebo **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Metformin **Description:** Metformin will be distributed in 500mg pills. **Name:** Metformin **Other Names:** - Glucophage - Fortamet - Glumetza - Riomet **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Placebo **Description:** Placebo will be distributed in pill form **Name:** Placebo **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** recovery of thigh muscle volume will be defined as the percentage of TMV at recovery when comparing to the TMV immobilization time point ((TMV recovery - TMV immobilization)/TMV immobilization)\*100. Thigh muscle volume will be determined by MRI. **Measure:** % recovery of thigh muscle volume (TMV) **Time Frame:** Thigh muscle volume will be determined at 14 days of single leg immobilization and at 14 days of recovery. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Age between 60y and older 2. BMI: \<30 kg/m2 3. Good general medical health, ambulatory and in independent living setting 4. Adequate upper body strength to use assistive walking device (crutches, walker, etc) as assessed by PI/staff during screening 5. Clinical Frailty Scale score \< 3 6. Mini-Cog score \> 3 Exclusion Criteria: 1. History of cardiovascular disease (e.g., CHF, CAD, MI, CVA) 2. History of endocrine or metabolic disease such as hypo/hyperthyroidism and diabetes (Treated hypo/hyperthyroid for at least 6 months will be permitted) 3. History of kidney disease or failure (CKD \> stage 4; serum creatinine \>1.5mg/dL) 4. History of vascular disease 5. Risk of DVT including family history of thrombophilia, DVT, pulmonary emboli, myeloproliferative diseases including polycythemia (Hb\>18 g/dL) or thrombocytosis (platelets\>400x103/mL), and connective tissue diseases (positive lupus anticoagulant), hyperhomocystinemia, deficiencies of factor V Leiden, proteins S and C, and antithrombine III 6. Use of anticoagulant therapy (e.g., Coumadin, heparin) 7. Uncontrolled hypertension - Elevated systolic pressure \>150 or a diastolic blood pressure \> 100 8. Implanted electronic devices (e.g., pacemakers, electronic infusion pumps, stimulators) 9. Cancer or history of successfully treated cancer (less than 1 year) other than basal cell carcinoma 10. Chronic systemic corticosteroid use (≥ 2 weeks) within 4 weeks of enrollment and for study duration (intra-articular/topical/inhaled therapeutic or physiologic doses of corticosteroids will be permitted) 11. Androgens or growth hormone within 6 months of enrollment and for study duration (topical physiologic androgen replacement will be permitted) 12. Inability to abstain from smoking or vaping for duration of study 13. Currently taking estrogen products (topical estrogen products will be permitted) 14. Currently on weight loss diet or medication 15. History of stroke with motor disability 16. A recent history (\<12 months) of GI bleed 17. History of liver disease or AST/ALT 2 times above the normal limit 18. History of respiratory disease (acute upper respiratory infection, history of chronic lung disease) 19. Any staff members who report directly to the principal investigators **Healthy Volunteers:** True **Minimum Age:** 60 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Micah Drummond **Phone:** 801-213-2737 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Rebekah Drummond **Phone:** 801-839-4534 **Role:** CONTACT #### Overall Officials **Official 1:** **Affiliation:** University of Utah **Name:** Micah Drummond **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Description:** IPD can be requested by the PI, Dr. Drummond. **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000001284 - Term: Atrophy - ID: D000020763 - Term: Pathological Conditions, Anatomical - ID: D000020879 - Term: Neuromuscular Manifestations - ID: D000009461 - Term: Neurologic Manifestations - ID: D000009422 - Term: Nervous System Diseases ### 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: M12090 - Name: Muscular Atrophy - Relevance: HIGH - As Found: Muscle Atrophy - ID: M4589 - Name: Atrophy - Relevance: LOW - As Found: Unknown - ID: M4554 - Name: Asthenia - Relevance: LOW - As Found: Unknown - ID: M22519 - Name: Pathological Conditions, Anatomical - 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 ### Condition Browse Module - Meshes - ID: D000009133 - Term: Muscular Atrophy ### 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:** NCT02331979 **Brief Title:** Improving Bladder Function in SCI by Neuromodulation **Official Title:** Improving Bladder Function in SCI by Neuromodulation #### Organization Study ID Info **ID:** 14-000932 #### Organization **Class:** OTHER **Full Name:** University of California, Los Angeles ### Status Module #### Completion Date **Date:** 2024-03-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-05-19 **Type:** ACTUAL **Last Update Submit Date:** 2023-05-17 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-03-31 **Type:** ESTIMATED #### Start Date **Date:** 2015-09 **Status Verified Date:** 2023-05 #### Study First Post Date **Date:** 2015-01-06 **Type:** ESTIMATED **Study First Submit Date:** 2015-01-03 **Study First Submit QC Date:** 2015-01-03 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of California, Los Angeles #### Responsible Party **Investigator Affiliation:** University of California, Los Angeles **Investigator Full Name:** Daniel Lu, MD, PhD **Investigator Title:** Associate Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** This trial will investigate the safety and utility of spinal cord neuromodulation to improve urinary bladder function in the context of spinal cord injury. **Detailed Description:** This trial will investigate the safety and utility of spinal cord neuromodulation to improve urinary bladder function in the context of spinal cord injury. Neuromodulation will be in the form of transcutaneous electrical stimulation and/or magnetic stimulation. ### Conditions Module **Conditions:** - Spinal Cord Injury ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 24 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Evaluate neuromodulation in 6 subjects with prior motor training. **Intervention Names:** - Device: Electromagnetic Neuromodulation **Label:** Stimulation of Non-Naive **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Evaluate neuromodulation in 6 naive subjects. **Intervention Names:** - Device: Electromagnetic Neuromodulation **Label:** Stimulation of Naive **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Apply parameters discovered in Arm 1 and Arm 2 to evaluate neuromodulation in 12 naive subjects. **Intervention Names:** - Device: Electromagnetic Neuromodulation **Label:** Stimulation **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Stimulation - Stimulation of Naive - Stimulation of Non-Naive **Description:** Electromagnetic stimulation to neuromodulate the spinal cord in the context of spinal cord injury. **Name:** Electromagnetic Neuromodulation **Other Names:** - Transcutaneous Electrical Stimulation - Magnetic Stimulation **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Measure:** Urine flow and volume **Time Frame:** Months 1-48 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Male 18-45 years; 2. At least 1 year post-injury; 3. Non-progressive SCI at C2-T8 (non-conus injury); 4. Motor Complete ASIA (A or B); 5. Neurogenic bladder requiring clean intermittent straight catheterization; 6. Able to attend twice weekly testing sessions for 6 months. 7. Have intact lower extremity anatomy and able to use lower extremity for assistive standing and stepping. Exclusion Criteria: 1. History of autonomic dysreflexia; 2. Ventilator dependency; 3. Musculoskeletal dysfunction, unhealed fracture, pressure ulcer, active infection; 4. Clinically significant depression or ongoing drug abuse; 5. Received botox injection, or bladder surgery (suprapubic access, Brindley procedure, etc.); 6. Prostatic hypertrophy or bladder outlet disorder; 7. Cardiopulmonary disease that precludes lower extremity training or rehabilitation. **Maximum Age:** 45 Years **Minimum Age:** 18 Years **Sex:** MALE **Standard Ages:** - ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Daniel Lu, MD PhD **Phone:** 310-825-4321 **Role:** CONTACT #### Locations **Location 1:** **City:** Los Angeles **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Daniel C Lu, MD PhD - **Phone:** 310-267-2975 - **Role:** CONTACT **Country:** United States **Facility:** University of California, Los Angeles **State:** California **Status:** RECRUITING **Zip:** 90095 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000013118 - Term: Spinal Cord Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000009422 - Term: Nervous System Diseases - 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: M15916 - Name: Spinal Cord Injuries - Relevance: HIGH - As Found: Spinal Cord Injury - ID: M15915 - Name: Spinal Cord 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: M22023 - Name: Trauma, Nervous System - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000013119 - Term: Spinal Cord Injuries ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02671279 **Acronym:** PreBar **Brief Title:** Low Calorie Diet in Morbidly Obese Patients **Official Title:** Effect of 6 Days Low Calorie Diet Before Bariatric Surgery #### Organization Study ID Info **ID:** 2015/361 #### Organization **Class:** OTHER **Full Name:** Haukeland University Hospital ### Status Module #### Completion Date **Date:** 2019-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2020-02-13 **Type:** ACTUAL **Last Update Submit Date:** 2020-02-11 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2018-08 **Type:** ACTUAL #### Start Date **Date:** 2016-08 **Type:** ACTUAL **Status Verified Date:** 2017-12 #### Study First Post Date **Date:** 2016-02-02 **Type:** ESTIMATED **Study First Submit Date:** 2015-07-13 **Study First Submit QC Date:** 2016-01-28 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Helse Fonna #### Lead Sponsor **Class:** OTHER **Name:** Haukeland University Hospital #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** This is a prospective study of morbidly obese patients that are planned to conduct bariatric surgery. The patients included in the study will conduct a low-calorie diet for 6 days prior to the operation and be admitted to hospital for this period. On admission and after finishing the diet a number of tests will be performed: Weigh, height, oral glucose tolerance test, bioimpedance measurement, and samples of blood, urine and a biopsy of subcutaneous adipose tissue. **Detailed Description:** The study will include 14 patients planned for bariatric surgery with BMI 40-46. The patients will be admitted to hospital 8 days prior to planned surgery and conduct a diet based mostly on fluids (yogurt, soups). Estimated daily calorie intake will be 400-600 kcal. At the day of admission and at day 7 a number of tests will be performed including blood tests, urine test and biometric measurement and a biopsy of subcutaneous adipose tissue. Weight will be measured every morning for the study period. During the operation fatty tissue samples will be taken subcutaneous and from the omentum major. This study is planned as a control group to another study where blood tests and subcutanous adipose tissue was sampled before and 6 days after bariatric surgery. The main aim of this study is to compare changes in adipose tissue and blood samples for patients before and 6 days after surgery to before and 6 days after a low-calorie diet. ### Conditions Module **Conditions:** - Obesity ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** BASIC_SCIENCE #### Enrollment Info **Count:** 15 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Dietary intervention group **Intervention Names:** - Dietary Supplement: Low calorie diet **Label:** Low calorie diet **Type:** OTHER ### Interventions #### Intervention 1 **Arm Group Labels:** - Low calorie diet **Description:** 6 days of low calorie diet, estimated kcal 400-600/day. **Name:** Low calorie diet **Type:** DIETARY_SUPPLEMENT ### Outcomes Module #### Primary Outcomes **Measure:** Changes in adipose gene expression signatures in obese subjects after 6 days of low-calorie diet **Time Frame:** 8 days #### Secondary Outcomes **Measure:** Changes in metabolomic signatures in obese subjects after 6 days of low-calorie diet **Time Frame:** 8 days ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * BMI 40-46 kg/m2 * The patients must not conduct a diet before admission. * Fasting blood glucose \<-7 nmol/l Exclusion Criteria: * Diabetes mellitus * Treatment with antidiabetics * Allergy for substances in the planned study-diet **Maximum Age:** 60 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Bergen **Country:** Norway **Facility:** Haukeland University Hospital **State:** Hordaland **Zip:** 5020 **Location 2:** **City:** Haugesund **Country:** Norway **Facility:** Haugesund Sjukehus, Helse Fonna #### Overall Officials **Official 1:** **Affiliation:** Haukeland University Hospital **Name:** Gunnar Mellgren, MD, PhD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M12701 - Name: Obesity - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01486979 **Brief Title:** Pharmacokinetic Assessment of the Absorption of Estradiol in Postmenopausal Women With Atrophic Vaginitis **Official Title:** A Pharmacokinetic Randomized Study With a Parallel Group Design to Assess the Extent of Systemic Absorption of Estradiol During Treatment With a 10 µg or 25 µg Estradiol Vaginal Tablet Administered Once Daily for 2 Weeks Followed by 10 Weeks of Twice-Weekly Maintenance Therapy in Postmenopausal Women With Atrophic Vaginitis #### Organization Study ID Info **ID:** VAG-1850 #### Organization **Class:** INDUSTRY **Full Name:** Novo Nordisk A/S #### Secondary ID Infos **ID:** 2006-005957-31 **Type:** EUDRACT_NUMBER ### Status Module #### Completion Date **Date:** 2007-05 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-02-08 **Type:** ESTIMATED **Last Update Submit Date:** 2017-02-06 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2007-05 **Type:** ACTUAL #### Start Date **Date:** 2007-01 **Status Verified Date:** 2017-02 #### Study First Post Date **Date:** 2011-12-07 **Type:** ESTIMATED **Study First Submit Date:** 2011-12-05 **Study First Submit QC Date:** 2011-12-06 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Novo Nordisk A/S #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** This trial is conducted in Europe. The aim of this trial is to to evaluate the extent of systemic absorption of estradiol during treatment with two different doses of estradiol in postmenopausal women with atrophic vaginitis. ### Conditions Module **Conditions:** - Menopause - Postmenopausal Vaginal Atrophy ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 58 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Drug: estradiol, 10 mcg **Label:** Low dose **Type:** EXPERIMENTAL #### Arm Group 2 **Intervention Names:** - Drug: estradiol, 25 mcg **Label:** High dose **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Low dose **Description:** One vaginal tablet once daily in the morning during the first two weeks of treatment followed by one vaginal tablet twice weekly in the morning for 10 weeks **Name:** estradiol, 10 mcg **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - High dose **Description:** One vaginal tablet once daily in the morning during the first two weeks of treatment followed by one vaginal tablet twice weekly in the morning for 10 weeks **Name:** estradiol, 25 mcg **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Area under the curve (AUC) #### Secondary Outcomes **Measure:** Average plasma concentration (C average) **Measure:** Maximal concentration (Cmax) **Measure:** Minimal concentration (Cmin) **Measure:** Time of maximal concentration (tmax) **Measure:** Incidence of adverse events ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Subjects who are able to use German language in speaking and writing * Postmenopausal women with at least 5 years after last menstruation, or bilateral oophorectomy performed two years or more prior to the time of screening * Serum FSH (Follicle Stimulating Hormone) levels above 40 mIU/ml and estradiol below 20 pg/ml * Maximum 5% superficial cells as assessed by evaluation of vaginal cytology * Endometrial thickness below 4.0 mm (double layer), as measured by transvaginal ultrasound (if applicable) * Availability of a normal mammogram within one year prior to trial start * Good general health as assessed by the Investigator and based on medical history, and physical and laboratory examinations Exclusion Criteria: * Known or suspected allergy to trial product or related products * Known, suspected or past history of breast cancer * Known, suspected or past estrogen dependent neoplasia e.g. endometrial cancer * Endometrial hyperplasia or endometrial polyps diagnosed during the screening period * Abnormal genital bleeding of unknown etiology * Previous estrogen and/ or progestin hormone replacement therapy **Maximum Age:** 70 Years **Minimum Age:** 60 Years **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Neu-Ulm **Country:** Germany **Facility:** Novo Nordisk Investigational Site **Zip:** 89231 #### Overall Officials **Official 1:** **Affiliation:** Novo Nordisk A/S **Name:** Global Clinical Registry (GCR, 1452) **Role:** STUDY_DIRECTOR ### References Module #### References **Citation:** Eugster-Hausmann M, Waitzinger J, Lehnick D. Minimized estradiol absorption with ultra-low-dose 10 microg 17beta-estradiol vaginal tablets. Climacteric. 2010 Jun;13(3):219-27. doi: 10.3109/13697137.2010.483297. **PMID:** 20423242 #### See Also Links **Label:** Clinical Trials at Novo Nordisk **URL:** http://novonordisk-trials.com ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014623 - Term: Vaginal 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: 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: M4589 - Name: Atrophy - Relevance: LOW - As Found: Unknown - ID: M17375 - Name: Vaginitis - Relevance: HIGH - As Found: Vaginitis - ID: M29419 - Name: Atrophic Vaginitis - Relevance: HIGH - As Found: Atrophic Vaginitis - ID: M17371 - Name: Vaginal 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: T6036 - Name: Menopause - Relevance: HIGH - As Found: Menopause ### Condition Browse Module - Meshes - ID: D000014627 - Term: Vaginitis - ID: D000059268 - Term: Atrophic Vaginitis ### Intervention Browse Module - Ancestors - ID: D000004967 - Term: Estrogens - ID: D000006728 - Term: Hormones - ID: D000006730 - Term: Hormones, Hormone Substitutes, and Hormone Antagonists - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000080066 - Term: Contraceptive Agents, Hormonal - ID: D000003270 - Term: Contraceptive Agents - ID: D000012102 - Term: Reproductive Control Agents - ID: D000003271 - Term: Contraceptive Agents, Female ### Intervention Browse Module - Browse Branches - Abbrev: Repr - Name: Reproductive Control Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M266279 - Name: Estradiol 17 beta-cypionate - Relevance: HIGH - As Found: Cell lymphoma - ID: M266280 - Name: Estradiol 3-benzoate - Relevance: HIGH - As Found: Cell lymphoma - ID: M8108 - Name: Estradiol - Relevance: HIGH - As Found: Cell lymphoma - ID: M234941 - Name: Polyestradiol phosphate - Relevance: HIGH - As Found: Cell lymphoma - ID: M8116 - Name: Estrogens - 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: M6494 - Name: Contraceptive Agents - Relevance: LOW - As Found: Unknown - ID: M2116 - Name: Contraceptive Agents, Hormonal - Relevance: LOW - As Found: Unknown - ID: M6495 - Name: Contraceptive Agents, Female - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: C000007630 - Term: Estradiol 17 beta-cypionate - ID: C000074283 - Term: Estradiol 3-benzoate - ID: D000004958 - Term: Estradiol - ID: C000008958 - Term: Polyestradiol phosphate ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01381679 **Acronym:** ACT II **Brief Title:** Effect of Ezetimibe Treatment on Low-density Lipoprotein Cholesterol (LDL-C) Levels in Participants With Coronary Heart Disease (CHD) Already Treated With a Statin (MK-0653A-205 AM1) **Official Title:** Austrian Cholesterol Screening And Treatment II (ACT II) #### Organization Study ID Info **ID:** 0653A-205 #### Organization **Class:** INDUSTRY **Full Name:** Organon and Co ### Status Module #### Completion Date **Date:** 2010-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2022-02-09 **Type:** ACTUAL **Last Update Submit Date:** 2022-02-07 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2010-12 **Type:** ACTUAL #### Results First Post Date **Date:** 2012-03-22 **Type:** ESTIMATED **Results First Submit Date:** 2011-11-23 **Results First Submit QC Date:** 2012-02-17 #### Start Date **Date:** 2009-05 **Status Verified Date:** 2022-02 #### Study First Post Date **Date:** 2011-06-27 **Type:** ESTIMATED **Study First Submit Date:** 2011-06-23 **Study First Submit QC Date:** 2011-06-24 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Organon and Co #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** This non-interventional longitudinal study is a follow-up of the Austrian Cholesterol Screening and Treatment project (ACT I), which assessed the cholesterol status, including achievement of the target levels applicable at that time (LDL levels \<100 mg/dL), in participants with coronary heart disease (CHD) already being treated with a statin. In this study, participants without adequate LDL-cholesterol reduction with a statin underwent extension of therapy with ezetimibe with the goal of achieving target levels. ### Conditions Module **Conditions:** - Hypercholesterolemia ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 1682 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants in whom LDL-C target levels have not been achieved and for whom ezetimibe therapy has been chosen. **Intervention Names:** - Drug: Ezetimibe **Label:** All participants ### Interventions #### Intervention 1 **Arm Group Labels:** - All participants **Description:** Ezetimibe was not mandatory and was prescribed as per routine prescription by physician. **Name:** Ezetimibe **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Individual LDL-C target values were set according to the Austrian Cholesterol Consensus (ACC) 2007 for patients for patients suffering from coronary heart disease (CHD) or CHD equivalent in an office-based, routine medical care setting. Participants were categorized as either high-risk or very high-risk based on ACC criteria. The LDL-C target levels for each category were 100 mg/dL and 70 mg/dL, respectively **Measure:** Number of Participants Achieving Individual LDL Cholesterol (LDL-C) Target Level **Time Frame:** Up to 12 months #### Secondary Outcomes **Measure:** Change From Baseline in Total Cholesterol (TC) at Month 3 **Time Frame:** Baseline and Month 3 **Measure:** Change From Baseline in TC at Month 12 **Time Frame:** Baseline and Month 12 **Measure:** Change From Baseline in LDL-C at Month 3 **Time Frame:** Baseline and Month 3 **Measure:** Change From Baseline in LDL-C at Month 12 **Time Frame:** Baseline and Month 12 **Measure:** Change From Baseline in High-density Lipoprotein Cholesterol (HDL-C) at Month 3 **Time Frame:** Baseline and Month 3 **Measure:** Change From Baseline in HDL-C at Month 12 **Time Frame:** Baseline and Month 12 **Measure:** Change From Baseline in Triglycerides (TG) at Month 3 **Time Frame:** Baseline and Month 3 **Measure:** Change From Baseline in TG at Month 12 **Time Frame:** Baseline and Month 12 ### Eligibility Module **Eligibility Criteria:** Inclusion criteria: * Participants in whom LDL-cholesterol target levels have not been achieved. * Participants in whom a decision has been made by the physician to initiate treatment with ezetimibe (longitudinal analyses). The treatment decision will be made prior to and independent from inclusion of participants into this study. - Participants with LDL cholesterol levels ≤113 mg/dl and a very high risk, which led to case-by-case approval of medication may be documented. Exclusion criteria: * Any condition which, in the opinion of the treating physician, precludes treatment with ezetimibe. * In order not to interfere with data collection it is recommended not to include participants currently in a clinical trial. * Previous and ongoing treatment with ezetimibe. **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT **Study Population:** Participants without adequate LDL-cholesterol reduction with a statin, representative cross-section across all regions/parts of Austria (federal provinces, urban/rural areas, office-based internists/general practitioners) ### References Module #### References **Citation:** Pichler M, Lautsch D, Adler C, Bogl K, Drexel H, Eber B, Fauer C, Fochterle J, Foger B, Gansch K, Grafinger P, Lechleitner M, Ludvik B, Maurer G, Morz R, Paulweber B, Pfeiffer KP, Prager R, Stark G, Toplak H, Traindl O, Weitgasser R. Are there differences in LDL-C target value attainment in Austrian federal states? Yes! Wien Med Wochenschr. 2013 Dec;163(23-24):528-35. doi: 10.1007/s10354-013-0219-z. Epub 2013 Aug 27. **PMID:** 23979353 **Citation:** Eber B, Lautsch D, Fauer C, Drexel H, Pfeiffer KP, Traindl O, Pichler M. Can LDL-cholesterol targets be achieved in a population at high risk? Results of the non-interventional study ACT II. Curr Med Res Opin. 2012 Sep;28(9):1447-54. doi: 10.1185/03007995.2012.717919. Epub 2012 Aug 16. **PMID:** 22856551 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000006949 - Term: Hyperlipidemias - ID: D000050171 - Term: Dyslipidemias - ID: D000052439 - Term: Lipid Metabolism Disorders - ID: D000008659 - Term: Metabolic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases ### Condition Browse Module - Browse Leaves - ID: M9419 - Name: Heart Diseases - Relevance: LOW - As Found: Unknown - ID: M6549 - Name: Coronary Disease - 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: M9988 - Name: Hypercholesterolemia - Relevance: HIGH - As Found: Hypercholesterolemia - ID: M10000 - Name: Hyperlipidemias - Relevance: LOW - As Found: Unknown - ID: M10002 - Name: Hyperlipoproteinemias - Relevance: LOW - As Found: Unknown - ID: M26181 - Name: Dyslipidemias - Relevance: LOW - As Found: Unknown - ID: M11639 - Name: Metabolic Diseases - Relevance: LOW - As Found: Unknown - ID: M27029 - Name: Lipid Metabolism Disorders - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000006937 - Term: Hypercholesterolemia ### 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 ### 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: LOW - As Found: Unknown - 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 ### Intervention Browse Module - Meshes - ID: D000069438 - Term: Ezetimibe ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** All Participants **Description:** Participants in whom low density lipoprotein cholesterol (LDL-C) target levels have not been achieved and for whom ezetimibe therapy has been chosen. **ID:** EG000 **Other Num at Risk:** 1682 **Serious Number Affected:** 3 **Serious Number At Risk:** 1682 **Title:** All Participants **Frequency Threshold:** 5 #### Serious Events **Term:** Cholecystitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA 13.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 1682 **Num Events:** 1 **Term:** Basal Cell Carcinoma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 13.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 1682 **Num Events:** 1 **Term:** Bronchial Neoplasm **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 13.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 1682 **Num Events:** 1 **Time Frame:** 12 months ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 1682 **Units:** Participants ### Group **ID:** BG000 **Title:** All Participants **Description:** Participants in whom low density lipoprotein cholesterol (LDL-C) target levels have not been achieved and for whom ezetimibe therapy has been chosen. ### Measure #### Measurement **Group ID:** BG000 **Spread:** 10.4 **Value:** 64.58 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 1003 **Class Title:** Males #### Measurement **Group ID:** BG000 **Value:** 655 **Class Title:** Females #### Measurement **Group ID:** BG000 **Value:** 24 **Class Title:** Not Reported ### Measure #### Measurement **Group ID:** BG000 **Value:** 1682 **Class Title:** Austria **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:** Sex/Gender, Customized **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** NUMBER **Title:** Region of Enrollment **Unit of Measure:** participants ## Results Section - More Information Module ### Certain Agreement **Restriction Type:** LTE60 **Restrictive Agreement:** True ### Point of Contact **Email:** [email protected] **Organization:** Merck Sharp & Dohme Corp **Phone:** 1-800-672-6372 **Title:** Senior Vice President, Global Clinical Development ## 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 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 678 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** -49.4 - **Spread:** 40.5 - **Upper Limit:** -43.8 - **Value:** -46.6 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** -57.7 - **Spread:** 37.62 - **Upper Limit:** -51.3 - **Value:** -54.5 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** -44.2 - **Spread:** 31.81 - **Upper Limit:** -39.3 - **Value:** -41.7 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** -51.9 - **Spread:** 26.64 - **Upper Limit:** -46.7 - **Value:** -49.3 **Title:** #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 2.0 - **Spread:** 14.17 - **Upper Limit:** 3.6 - **Value:** 2.8 **Title:** #### Outcome Measure 7 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 3.1 - **Spread:** 15.05 - **Upper Limit:** 5.0 - **Value:** 4.0 **Title:** #### Outcome Measure 8 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** -41.1 - **Spread:** 82.51 - **Upper Limit:** -26.6 - **Value:** -33.8 **Title:** #### Outcome Measure 9 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** -51.5 - **Spread:** 75.06 - **Upper Limit:** -36.4 - **Value:** -43.9 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Individual LDL-C target values were set according to the Austrian Cholesterol Consensus (ACC) 2007 for patients for patients suffering from coronary heart disease (CHD) or CHD equivalent in an office-based, routine medical care setting. Participants were categorized as either high-risk or very high-risk based on ACC criteria. The LDL-C target levels for each category were 100 mg/dL and 70 mg/dL, respectively **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** Up to 12 months **Title:** Number of Participants Achieving Individual LDL Cholesterol (LDL-C) Target Level **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Participants in whom low density lipoprotein cholesterol (LDL-C) target levels have not been achieved and for whom ezetimibe therapy has been chosen. **ID:** OG000 **Title:** All Participants #### Outcome Measure 2 **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** Participants with baseline, follow-up visit 1 (3 months), and follow-up visit 2 (12 months) measurements for TC. **Reporting Status:** POSTED **Time Frame:** Baseline and Month 3 **Title:** Change From Baseline in Total Cholesterol (TC) at Month 3 **Type:** SECONDARY **Unit of Measure:** mg/dL ##### Group **Description:** Participants in whom low density lipoprotein cholesterol (LDL-C) target levels have not been achieved and for whom ezetimibe therapy has been chosen. **ID:** OG000 **Title:** All Participants #### Outcome Measure 3 **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** Participants with baseline, follow-up visit 1 (3 months), and follow-up visit 2 (12 months) measurements for TC. **Reporting Status:** POSTED **Time Frame:** Baseline and Month 12 **Title:** Change From Baseline in TC at Month 12 **Type:** SECONDARY **Unit of Measure:** mg/dL ##### Group **Description:** Participants in whom low density lipoprotein cholesterol (LDL-C) target levels have not been achieved and for whom ezetimibe therapy has been chosen. **ID:** OG000 **Title:** All Participants #### Outcome Measure 4 **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** Participants with baseline, follow-up visit 1 (3 months), and follow-up visit 2 (12 months) measurements for LDL-C. **Reporting Status:** POSTED **Time Frame:** Baseline and Month 3 **Title:** Change From Baseline in LDL-C at Month 3 **Type:** SECONDARY **Unit of Measure:** mg/dL ##### Group **Description:** Participants in whom low density lipoprotein cholesterol (LDL-C) target levels have not been achieved and for whom ezetimibe therapy has been chosen. **ID:** OG000 **Title:** All Participants #### Outcome Measure 5 **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** Participants with baseline, follow-up visit 1 (3 months), and follow-up visit 2 (12 months) measurements for LDL-C. **Reporting Status:** POSTED **Time Frame:** Baseline and Month 12 **Title:** Change From Baseline in LDL-C at Month 12 **Type:** SECONDARY **Unit of Measure:** mg/dL ##### Group **Description:** Participants in whom low density lipoprotein cholesterol (LDL-C) target levels have not been achieved and for whom ezetimibe therapy has been chosen. **ID:** OG000 **Title:** All Participants #### Outcome Measure 6 **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** Participants with baseline, follow-up visit 1 (3 months), and follow-up visit 2 (12 months) measurements for HDL-C. **Reporting Status:** POSTED **Time Frame:** Baseline and Month 3 **Title:** Change From Baseline in High-density Lipoprotein Cholesterol (HDL-C) at Month 3 **Type:** SECONDARY **Unit of Measure:** mg/dL ##### Group **Description:** Participants in whom low density lipoprotein cholesterol (LDL-C) target levels have not been achieved and for whom ezetimibe therapy has been chosen. **ID:** OG000 **Title:** All Participants #### Outcome Measure 7 **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** Participants with baseline, follow-up visit 1 (3 months), and follow-up visit 2 (12 months) measurements for HDL-C. **Reporting Status:** POSTED **Time Frame:** Baseline and Month 12 **Title:** Change From Baseline in HDL-C at Month 12 **Type:** SECONDARY **Unit of Measure:** mg/dL ##### Group **Description:** Participants in whom low density lipoprotein cholesterol (LDL-C) target levels have not been achieved and for whom ezetimibe therapy has been chosen. **ID:** OG000 **Title:** All Participants #### Outcome Measure 8 **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** Participants with baseline, follow-up visit 1 (3 months), and follow-up visit 2 (12 months) measurements for TG. **Reporting Status:** POSTED **Time Frame:** Baseline and Month 3 **Title:** Change From Baseline in Triglycerides (TG) at Month 3 **Type:** SECONDARY **Unit of Measure:** mg/dL ##### Group **Description:** Participants in whom low density lipoprotein cholesterol (LDL-C) target levels have not been achieved and for whom ezetimibe therapy has been chosen. **ID:** OG000 **Title:** All Participants #### Outcome Measure 9 **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEAN **Population Description:** Participants with baseline, follow-up visit 1 (3 months), and follow-up visit 2 (12 months) measurements for TG. **Reporting Status:** POSTED **Time Frame:** Baseline and Month 12 **Title:** Change From Baseline in TG at Month 12 **Type:** SECONDARY **Unit of Measure:** mg/dL ##### Group **Description:** Participants in whom low density lipoprotein cholesterol (LDL-C) target levels have not been achieved and for whom ezetimibe therapy has been chosen. **ID:** OG000 **Title:** All Participants ### Participant Flow Module #### Group **Description:** Participants in whom low density lipoprotein cholesterol (LDL-C) target levels have not been achieved and for whom ezetimibe therapy has been chosen. **ID:** FG000 **Title:** All Participants #### Period **Title:** Overall Study ##### Withdraw **Type:** Adverse Event ###### Reason **Group ID:** FG000 **Number of Subjects:** 3 ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 885 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 1682 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 794 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 888 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT01735279 **Brief Title:** Studying the Effects of Administration of Polyunsaturated Fatty Acids (PUFAS) of Omega-3 Series in Nicotine Dependence **Official Title:** "Studying the Effects of Administration of Polyunsaturated Fatty Acids (PUFAS) of Omega-3 Series in Nicotine Dependence" #### Organization Study ID Info **ID:** Tobacco&Omega #### Organization **Class:** OTHER **Full Name:** Federal University of São Paulo ### Status Module #### Completion Date **Date:** 2014-03 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2020-10-14 **Type:** ACTUAL **Last Update Submit Date:** 2020-09-18 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2013-12 **Type:** ACTUAL #### Results First Post Date **Date:** 2020-10-14 **Type:** ACTUAL **Results First Submit Date:** 2016-01-06 **Results First Submit QC Date:** 2020-09-18 #### Start Date **Date:** 2013-01 **Type:** ACTUAL **Status Verified Date:** 2020-09 #### Study First Post Date **Date:** 2012-11-28 **Type:** ESTIMATED **Study First Submit Date:** 2012-11-23 **Study First Submit QC Date:** 2012-11-27 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Federal University of São Paulo #### Responsible Party **Investigator Affiliation:** Federal University of São Paulo **Investigator Full Name:** Jose Carlos Fernandes Galduroz MD **Investigator Title:** Md PhD **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Nicotine dependence may prolong the exposure to toxic substances that cause various diseases. The Central Nervous System (CNS) is consisted by a large amount of Polyunsaturated Fatty Acids (PUFAS) from omega-3 serie. Omega-3 takes part in several actions, including the modulation of dopaminergic neurotransmission. In its deficiency is detected a hypofunctioning of the mesolimbic and mesocortical pathway, related to the reward system, involved on the context of nicotine dependence. Treatment using dietary supplementation with omega-3 shows improvements in several diseases, including mood disorders such as anxiety and depression. The investigators hypothesis is that supplementation with these fatty acids can restore the levels of omega-3 and could decrease nicotine dependence. The investigators objective is to investigate a possible association between increased serum levels of omega-3 and the reduction in nicotine dependence. **Detailed Description:** Introduction: A cigarette has more than 6,000 toxic substances that can cause various diseases. Nicotine dependence may prolong the exposure to these toxic substances. The Central Nervous System (CNS) is consisted by a large amount of Polyunsaturated Fatty Acids (PUFAS) from omega-3 serie. Omega-3 takes part in several actions, including the modulation of dopaminergic neurotransmission. In its deficiency is detected a hypofunctioning of the mesolimbic and mesocortical pathway, related to the reward system, involved on the context of nicotine dependence. Treatment using dietary supplementation with omega-3 shows improvements in several diseases, including mood disorders such as anxiety and depression. The investigators hypothesis is that supplementation with these fatty acids can restore the levels of omega-3 and could decrease nicotine dependence. Objective: Investigate a possible association between increased serum levels of omega-3 and the reduction in nicotine dependence. Material and Methods: In the clinical study, placebo controlled, double-blind, parallel, randomized, will be administered to 60 volunteers: placebo or fish oil for 90 days. Psychometric assessments will be carried out, measurements of serum levels of PUFAS, levels of carbon monoxide (CO) and cotinine in plasma will be done for monitoring the clinical course. Data Analysis: Repeated measures (ANOVA) for the dependent variables (dependency, anxiety, depression, motivation, compulsion, dosage of PUFAS, exhaled CO and cotinine) and independent (groups and time) to check for significant differences. If so, a second ANOVA with covariates will be conducted. Significance is p \<0.05 in all analyzes. ### Conditions Module **Conditions:** - Tobacco Dependence - Nicotine Dependence **Keywords:** - tobacco dependence - tobacco treatment - omega 3 series - polyunsaturated fatty acids ### 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:** 58 **Type:** ACTUAL **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** The placebo group will receive 3g per day of mineral oil during 90 days treatment **Intervention Names:** - Drug: Placebo **Label:** Placebo **Type:** PLACEBO_COMPARATOR #### Arm Group 2 **Description:** The omega 3 group will receive 3g per day of fish oil during 90 days treatment **Intervention Names:** - Drug: Omega 3 **Label:** Omega3 **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Omega3 **Description:** Each fish oil capsule contained 210.99 mg of EPA and 129.84 mg of DHA. **Name:** Omega 3 **Other Names:** - fish oil - omega 3 fatty acid **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Placebo **Description:** mineral oil + food dye #2 (simulating the colour of essential fatty acids); 1000 mg of mineral oil per capsule **Name:** Placebo **Other Names:** - mineral oil **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** The Fageström Test for Nicotine Dependence is a widely used test to measure the nicotine dependence. This test was chosen to evaluate the possible changes on the nicotine dependence during the clinical trial. The range for the scale is: very low (dependence) 0-2 low (dependence) 3-4 moderate (dependence) 5 high (dependence) 6-7 very high (dependence) 8-10 So, lower levels (low scores on the scale) of the FTND measure indicates lower nicotine dependence, and higher levels (high scores on the scales) of the FTND measure indicates higher nicotine dependence. **Measure:** Fageström Test for Nicotine Dependence Score at Baseline and After 90 Days of the Treatment **Time Frame:** Baseline (prior to the beginning) and after 90 days of the treatment #### Secondary Outcomes **Description:** The polyunsaturated fatty acid Docosahexaenoic acid (DHA) is one of the main fatty acids omega-3 series. The dosage of these analyte was achieved by means of Liquid Chromatography - Mass Spectrometry Mass Spectrometry - LS-MS-MS. **Measure:** Concentration of Docosahexaenoic Acid (DHA) at Baseline and After 90 Days of Treatment **Time Frame:** At baseline (prior to the beginning of the treatment) and after 90 days of treatment **Description:** The polyunsaturated fatty acid Eicosapentaenoic acid (EPA) is one of the main fatty acids omega-3 series. The dosage of these analyte was achieved by means of Liquid Chromatography - Mass Spectrometry Mass Spectrometry - LS-MS-MS. **Measure:** Concentration of Eicosapentaenoic Acid (EPA) **Time Frame:** At baseline (prior to the beginning of the treatment) and after 90 days of treatment ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * healthy smokers; age between 20 and 60 years; score in Fagerström Test for Nicotine Dependence (FTND) up to 5 points (FTND \> 5); high motivation to stop smoking (accessed by Richmond Test) Exclusion Criteria: * psychiatric disorders; taking psychoactive medications; history of alcohol and/or other drugs abuse or dependence; **Healthy Volunteers:** True **Maximum Age:** 60 Years **Minimum Age:** 20 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** São Paulo **Country:** Brazil **Facility:** Unidade de Dependência de Drogas (UDED) **Zip:** 55 #### Overall Officials **Official 1:** **Affiliation:** Universidade Federal de São Paulo (UNIFESP) **Name:** José Carlos F. Galduróz, Md PhD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Description:** We do not plan to make individual participant data available. The data obtained was analysed and published **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000019966 - Term: Substance-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: M16785 - Name: Tobacco Use Disorder - Relevance: HIGH - As Found: Nicotine Dependence - ID: M21837 - Name: Substance-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: D000014029 - Term: Tobacco Use Disorder ### Intervention Browse Module - Ancestors - ID: D000004643 - Term: Emollients - ID: D000003879 - Term: Dermatologic Agents ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Derm - Name: Dermatologic Agents - Abbrev: Ot - Name: Other Dietary Supplements ### Intervention Browse Module - Browse Leaves - ID: M12478 - Name: Nicotine - Relevance: LOW - As Found: Unknown - ID: M11869 - Name: Mineral Oil - Relevance: HIGH - As Found: Hypoparathyroidism - ID: M7809 - Name: Emollients - Relevance: LOW - As Found: Unknown - ID: M7074 - Name: Dermatologic Agents - Relevance: LOW - As Found: Unknown - ID: T415 - Name: Omega 3 Fatty Acid - Relevance: HIGH - As Found: Machine ### Intervention Browse Module - Meshes - ID: D000008899 - Term: Mineral Oil ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** No adverse event were reported by the participants #### Event Groups **Group ID:** EG000 **Title:** Placebo Group n= 29 **Deaths Num At Risk:** 29 **Description:** placebo capsules 1,000 mg - mineral oil + food dye #2 (simulating the colour of essential fatty acids), taken in 3 daily doses, for 90 days. **ID:** EG000 **Other Num at Risk:** 29 **Serious Number At Risk:** 29 **Title:** Placebo Group n= 29 **Group ID:** EG001 **Title:** Omega3 Group n= 29 **Deaths Num At Risk:** 29 **Description:** omega 3 capsules 1,000 mg fish oil taken as 3 daily doses, for 90 days. Each fish oil capsule contained 210.99 mg of EPA and 129.84 mg of DHA **ID:** EG001 **Other Num at Risk:** 29 **Serious Number At Risk:** 29 **Title:** Omega3 Group n= 29 **Frequency Threshold:** 5 **Time Frame:** The adverse event were collected monthly (after 30, 60 and 90 days of the treatment) during the 90 days (treatment time) ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 29 **Group ID:** BG001 **Value:** 29 **Group ID:** BG002 **Value:** 58 **Units:** Participants ### Group **ID:** BG000 **Title:** Placebo **Description:** placebo capsules 1,000 mg - mineral oil + food dye #2 (simulating the colour of essential fatty acids), taken in 3 daily doses, for 90 days. ### Group **ID:** BG001 **Title:** Omega3 **Description:** omega 3 capsules 1,000 mg fish oil taken as 3 daily doses, for 90 days. Each fish oil capsule contained 210.99 mg of EPA and 129.84 mg of DHA ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 11.5 **Value:** 47.8 #### Measurement **Group ID:** BG001 **Spread:** 11.1 **Value:** 46.2 #### Measurement **Group ID:** BG002 **Spread:** 11.3 **Value:** 47 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 15 #### Measurement **Group ID:** BG001 **Value:** 13 #### Measurement **Group ID:** BG002 **Value:** 28 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 14 #### Measurement **Group ID:** BG001 **Value:** 16 #### Measurement **Group ID:** BG002 **Value:** 30 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 4.7 **Value:** 24.3 #### Measurement **Group ID:** BG001 **Spread:** 3.9 **Value:** 24.7 #### Measurement **Group ID:** BG002 **Spread:** 4.3 **Value:** 24.5 **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 ### Measure 3 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Body Mass Index (BMI) **Unit of Measure:** kg/m^2 ## Results Section - More Information Module ### Certain Agreement ### Point of Contact **Email:** [email protected] **Organization:** Department of Psychobiology, Universidade Federal de São Paulo (UNIFESP), Rua Napoleão de Barros, São Paulo , Brazil **Phone:** +55 11 2149 0000 **Title:** Md PhD José Carlos F. Galduróz ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ### 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:** Test t Student **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** <0.05 **P-Value Comment:** **Parameter Type:** Mean Difference (Final Values) **Parameter Value:** 0.05 **Statistical Comment:** **Statistical Method:** ANOVA **Tested Non-Inferiority:** #### Analysis **CI Lower Limit:** **CI Number of Sides:** ONE_SIDED **CI Percentage Value:** 95 **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:** <0.01 **P-Value Comment:** **Parameter Type:** Mean Difference (Final Values) **Parameter Value:** 30.84 **Statistical Comment:** **Statistical Method:** ANOVA **Tested Non-Inferiority:** #### 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 **Other Analysis Description:** **P-Value:** <0.01 **P-Value Comment:** **Parameter Type:** Mean Difference (Final Values) **Parameter Value:** 0.002 **Statistical Comment:** **Statistical Method:** t-test, 1 sided **Tested Non-Inferiority:** #### Analysis **CI Lower Limit:** **CI Number of Sides:** ONE_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 **Other Analysis Description:** **P-Value:** <0.01 **P-Value Comment:** **Parameter Type:** Mean Difference (Final Values) **Parameter Value:** 4.4 **Statistical Comment:** **Statistical Method:** t-test, 1 sided **Tested Non-Inferiority:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 2.01 - **Upper Limit:** - **Value:** 6.53 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 1.52 - **Upper Limit:** - **Value:** 6.56 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 1.85 - **Upper Limit:** - **Value:** 5.96 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 2.15 - **Upper Limit:** - **Value:** 5.22 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 794.00 - **Upper Limit:** - **Value:** 747.71 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 757.49 - **Upper Limit:** - **Value:** 645.38 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 767.41 - **Upper Limit:** - **Value:** 663.93 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 1,484.63 - **Upper Limit:** - **Value:** 1,004.94 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 67.72 - **Upper Limit:** - **Value:** 77.00 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 111.79 - **Upper Limit:** - **Value:** 86.76 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 73.22 - **Upper Limit:** - **Value:** 77.39 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 285.37 - **Upper Limit:** - **Value:** 174.87 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 65.10 - **Upper Limit:** - **Value:** 101.20 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 85.15 - **Upper Limit:** - **Value:** 129.38 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 63.26 - **Upper Limit:** - **Value:** 97.79 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 61.94 - **Upper Limit:** - **Value:** 98.54 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** The Fageström Test for Nicotine Dependence is a widely used test to measure the nicotine dependence. This test was chosen to evaluate the possible changes on the nicotine dependence during the clinical trial. The range for the scale is: very low (dependence) 0-2 low (dependence) 3-4 moderate (dependence) 5 high (dependence) 6-7 very high (dependence) 8-10 So, lower levels (low scores on the scale) of the FTND measure indicates lower nicotine dependence, and higher levels (high scores on the scales) of the FTND measure indicates higher nicotine dependence. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** Baseline (prior to the beginning) and after 90 days of the treatment **Title:** Fageström Test for Nicotine Dependence Score at Baseline and After 90 Days of the Treatment **Type:** PRIMARY **Unit of Measure:** units on a scale ##### Group **Description:** placebo capsules 1,000 mg - mineral oil + food dye #2 (simulating the colour of essential fatty acids), taken in 3 daily doses, for 90 days. **ID:** OG000 **Title:** Placebo ##### Group **Description:** omega 3 capsules 1,000 mg fish oil taken as 3 daily doses, for 90 days. Each fish oil capsule contained 210.99 mg of EPA and 129.84 mg of DHA **ID:** OG001 **Title:** Omega3 #### Outcome Measure 2 **Description:** The polyunsaturated fatty acid Docosahexaenoic acid (DHA) is one of the main fatty acids omega-3 series. The dosage of these analyte was achieved by means of Liquid Chromatography - Mass Spectrometry Mass Spectrometry - LS-MS-MS. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** At baseline (prior to the beginning of the treatment) and after 90 days of treatment **Title:** Concentration of Docosahexaenoic Acid (DHA) at Baseline and After 90 Days of Treatment **Type:** SECONDARY **Unit of Measure:** ng/ml ##### Group **Description:** placebo capsules 1,000 mg - mineral oil + food dye #2 (simulating the colour of essential fatty acids), taken in 3 daily doses, for 90 days. **ID:** OG000 **Title:** Placebo ##### Group **Description:** omega 3 capsules 1,000 mg fish oil taken as 3 daily doses, for 90 days. Each fish oil capsule contained 210.99 mg of EPA and 129.84 mg of DHA **ID:** OG001 **Title:** Omega3 #### Outcome Measure 3 **Description:** The polyunsaturated fatty acid Eicosapentaenoic acid (EPA) is one of the main fatty acids omega-3 series. The dosage of these analyte was achieved by means of Liquid Chromatography - Mass Spectrometry Mass Spectrometry - LS-MS-MS. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** At baseline (prior to the beginning of the treatment) and after 90 days of treatment **Title:** Concentration of Eicosapentaenoic Acid (EPA) **Type:** SECONDARY **Unit of Measure:** ng/ml ##### Group **Description:** placebo capsules 1,000 mg - mineral oil + food dye #2 (simulating the colour of essential fatty acids), taken in 3 daily doses, for 90 days. **ID:** OG000 **Title:** Placebo ##### Group **Description:** omega 3 capsules 1,000 mg fish oil taken as 3 daily doses, for 90 days. Each fish oil capsule contained 210.99 mg of EPA and 129.84 mg of DHA **ID:** OG001 **Title:** Omega3 #### Outcome Measure 4 **Description:** Cotinine is the major metabolite of nicotine, making it its primary metabolite. Cotinine dosage is considered one of the best parameters to distinguish smokers from non-smokers as well as for the nicotine graduation consumption. The traditional cut off value used to distinguish between smokers and non-smokers is 15 ng/mL for dosages from plasma sample **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** At baseline (prior to the beginning of the treatment) and after 90 days of treatment **Title:** Concentration of Serum Cotinine at Baseline and After 90 Days of Treatment **Type:** POST_HOC **Unit of Measure:** ng/ml ##### Group **Description:** placebo capsules 1,000 mg - mineral oil + food dye #2 (simulating the colour of essential fatty acids), taken in 3 daily doses, for 90 days. **ID:** OG000 **Title:** Placebo ##### Group **Description:** omega 3 capsules 1,000 mg fish oil taken as 3 daily doses, for 90 days. Each fish oil capsule contained 210.99 mg of EPA and 129.84 mg of DHA **ID:** OG001 **Title:** Omega3 ### Participant Flow Module #### Group **Description:** placebo capsules 1,000 mg - mineral oil + food dye #2 (simulating the colour of essential fatty acids), taken in 3 daily doses. **ID:** FG000 **Title:** Placebo #### Group **Description:** omega 3 capsules 1,000 mg fish oil taken as 3 daily doses. Each fish oil capsule contained 210.99 mg of EPA and 129.84 mg of DHA **ID:** FG001 **Title:** Omega3 #### Period **Title:** Overall Study ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 11 ###### Reason **Group ID:** FG001 **Number of Subjects:** 8 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 29 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 29 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 18 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 21 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 11 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 8 **Pre-Assignment Details:** No participants were excluded. **Recruitment Details:** Data were collected in São Paulo/Brazil between January 2013 and August 2013. All participants signed an informed consent prior to the beginning of the study. The participants did not receive any kind of compensation for participation in the study. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT05188079 **Brief Title:** Effect of Acute Nitrate Supplementation on Vascular Function in Hispanic, Black and White Population **Official Title:** Effect of Acute Nitrate Supplementation on Vascular Function in Hispanic, Black and White Population #### Organization Study ID Info **ID:** STUDY00001163 #### Organization **Class:** OTHER **Full Name:** University of Texas at Austin ### Status Module #### Completion Date **Date:** 2026-01-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2022-01-12 **Type:** ACTUAL **Last Update Submit Date:** 2022-01-07 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2025-08-01 **Type:** ESTIMATED #### Start Date **Date:** 2021-10-01 **Type:** ACTUAL **Status Verified Date:** 2022-01 #### Study First Post Date **Date:** 2022-01-12 **Type:** ACTUAL **Study First Submit Date:** 2021-12-08 **Study First Submit QC Date:** 2022-01-07 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Texas at Austin #### 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 purpose of the study is to assess vascular function in African Americans, Hispanic and Caucasian Americans to better understand racial differences in cardiovascular health. The investigators will further investigate the affect of Beetroot juice on the vascular function of all participants. **Detailed Description:** African Americans and Hispanic individuals have the highest incidence of type 2 diabetes, which increases their risk of all cardiovascular diseases. One of the hallmarks of type 2 diabetes is vascular dysfunction, caused by a lack of nitric oxide bioavailability. Vascular function and blood flow responses to exercise have been shown to improve after nitrate supplementation (beetroot juice), However, whether nitrate supplementation will improve vascular function in African American and Hispanic individuals remains unknown. The investigators will perform a randomized, placebo-controlled study in healthy African American, Hispanic and Caucasian subjects. They will be studied before and 90 minutes after consuming Beetroot juice (high nitrate supplement) or Placebo, with at least 1-week washout period between the two visits (Kenjale AA et al., J Appl Physiol 2011). ### Conditions Module **Conditions:** - Healthy Adults ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER **Intervention Model Description:** randomized, cross-over, placebo-controlled trial ##### Masking Info **Masking:** TRIPLE **Masking Description:** it will be double blinded **Who Masked:** - PARTICIPANT - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** BASIC_SCIENCE #### Enrollment Info **Count:** 90 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 140 ml of beetroot juice (high nitrate dietary supplement) **Intervention Names:** - Dietary Supplement: Beetroot juice **Label:** Beetroot Juice **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Placebo drink looks and tastes like the beetroot juice but has the nitrate removed from the juice. **Intervention Names:** - Dietary Supplement: Placebo drink **Label:** Placebo **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Beetroot Juice **Description:** high nitrate supplement **Name:** Beetroot juice **Type:** DIETARY_SUPPLEMENT #### Intervention 2 **Arm Group Labels:** - Placebo **Description:** placebo **Name:** Placebo drink **Type:** DIETARY_SUPPLEMENT ### Outcomes Module #### Primary Outcomes **Description:** Blood flow of the brachial artery will be measured continuously at rest, during 3 min of handgrip exercise and for up to 1 min after the cessation of handgrip exercise. **Measure:** Forearm blood flow **Time Frame:** through study completion, an average of 2 year #### Secondary Outcomes **Description:** The subjects will lay supine with arm extended on a side table. Resting brachial artery diameter and velocity will be measured at rest for 2 min. Following this, a pneumatic cuff will be inflated on the forearm to supersystolic pressure (220-240 Torr) for 5 min and once the cuff is released, brachial artery diameter and velocity will be measured for up to 3 minutes after cuff deflation. FMD% will be calculated as (highest diameter after cuff deflation - baseline brachial artery diameter)/ baseline diameter \* 100 **Measure:** Flow mediated vasodilation **Time Frame:** through study completion, an average of 2 year ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Healthy men and women who are either Caucasian American, Hispanic, or non-Hispanic African American Exclusion Criteria: * Blood pressure higher than or equal to 140/90 mmHg * BMI \> 35kg/m2 * Have a history of cardiovascular, neurological or vascular diseases * Take prescription medications * Are smokers * Are pregnant **Healthy Volunteers:** True **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:** jasdeep kaur, PhD **Phone:** 5124718595 **Role:** CONTACT #### Locations **Location 1:** **City:** Austin **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Jasdeep Kaur, PhD - **Phone:** 512-471-8595 - **Role:** CONTACT **Country:** United States **Facility:** University of Texas at Austin **State:** Texas **Status:** RECRUITING **Zip:** 78712 ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: Rare - Name: Rare Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06131879 **Acronym:** RCS **Brief Title:** Laser Versus Physiotherapy in Management of Bruxism in Children: RCS **Official Title:** Evaluation of Laser Acupuncture Versus Modified Physical Therapy Intervention in Management of Bruxism in Children, Study Protocol for a Randomized Controlled Study #### Organization Study ID Info **ID:** 4-4-3 #### Organization **Class:** OTHER **Full Name:** National Research Centre, Egypt ### Status Module #### Completion Date **Date:** 2023-10-19 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-11-14 **Type:** ACTUAL **Last Update Submit Date:** 2023-11-12 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2023-09-15 **Type:** ACTUAL #### Start Date **Date:** 2023-08-22 **Type:** ACTUAL **Status Verified Date:** 2023-11 #### Study First Post Date **Date:** 2023-11-14 **Type:** ACTUAL **Study First Submit Date:** 2023-10-20 **Study First Submit QC Date:** 2023-11-12 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** National Research Centre, Egypt #### Responsible Party **Investigator Affiliation:** National Research Centre, Egypt **Investigator Full Name:** Mohamed Farouk Rashed **Investigator Title:** Researcher **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Is US Export:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** The goal of this randomized controlled study is to compare laser acupuncture versus modified physical therapy in controlling bruxism in children. The main questions aim to answer are: * Laser will decrease tempro-mandibular joint pain from bruxism more than modified physical therapy? * Laser will decrease muscles activity from bruxism more than modified physical therapy? Children took 6 sessions of either laser acupuncture or modified physical therapy Researchers compared laser acupuncture versus modified physical therapy to see if any decrease in tempro-mandibular joint pain and muscles activity from bruxism **Detailed Description:** The children participated in the study as bruxers according to the American Association of Sleep Medicine (AASM) diagnostic criteria \[ Sixteen children will be randomly allocated to two groups of 8 individuals each: Group one: laser acupuncture, Group two: electromyography biofeedback training. Pain will be measured by Visual Analogue scale; VAS (primary outcome)and muscle activity will be measured by Electromyography (Secondary outcome) of temporalis and masseter muscles. Measurements will be taken preoperatively and after 8 weeks. ### Conditions Module **Conditions:** - Bruxism **Keywords:** - bruxism - laser - children - Physiotherapy ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** Randomized double arm controlled study ##### Masking Info **Masking:** DOUBLE **Masking Description:** Investigator and outcomes assessor were blinded to groups **Who Masked:** - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 16 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Laser acupuncture biostimulation for temporalis and masseter muscles **Intervention Names:** - Device: Laser acupuncture **Label:** Laser acupuncture **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Muscle relaxation and biofeedback for temporalis and masseter muscles **Intervention Names:** - Behavioral: Modified physical therapy **Label:** Modified physical therapy **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Laser acupuncture **Description:** Biostimulation of temporalis and masseter muscles **Name:** Laser acupuncture **Other Names:** - Doctor smile low level laser device **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - Modified physical therapy **Description:** Progressive muscle relaxation of temporalis and masseter muscles **Name:** Modified physical therapy **Other Names:** - Relaxation and biofeedback of muscles **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** Tenderness and pain of temporalis and masseter muscles by visual analogue scale (VAS) of pain before and after intervention either no pain, mild pain, moderate or severe pain. Better outcome means no or mild pain **Measure:** Tempro-mandibular joint pain **Time Frame:** 2 months #### Secondary Outcomes **Description:** Activity of temporalis and masseter muscles by surface electromyography (EMG) at rest and during function (Decrease in activity by small values bymicrons means better outcome and vice versa) **Measure:** Activity of temporalis and masseter muscles **Time Frame:** 2 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Six to twelve years children 2. Medically free 3. Normal occlusion 4. No or low caries experience 5. Clinical dental wear 6. Clenching or grinding reported by the parents Exclusion Criteria: 1. Children with any physical or psychological disease. 2. Children received any previous treatment for bruxism. 3. Children with tempro-mandibular joint disorders **Healthy Volunteers:** True **Maximum Age:** 12 Years **Minimum Age:** 6 Years **Sex:** ALL **Standard Ages:** - CHILD ### Contacts Locations Module #### Locations **Location 1:** **City:** Dokki **Country:** Egypt **Facility:** National Research Centre **State:** Giza **Zip:** 12622 #### Overall Officials **Official 1:** **Affiliation:** National Research Centre, Egypt **Name:** Mohamed F Rashed, Researcher **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Description:** No plan to share results **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014076 - Term: Tooth Diseases - ID: D000009057 - Term: Stomatognathic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC07 - Name: Mouth and Tooth Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M5286 - Name: Bruxism - Relevance: HIGH - As Found: Bruxism - ID: M16831 - Name: Tooth Diseases - Relevance: LOW - As Found: Unknown - ID: M12017 - Name: Stomatognathic Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000002012 - Term: Bruxism ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06179979 **Brief Title:** PET Study With 11 C-PIB to Evaluate Amyloid Protein Deposits in Mild Cognitive Disorder, Alzheimer's Disease and Early Frontotemporal Degeneration. **Official Title:** PET Study With 11 C-PIB to Evaluate Amyloid Protein Deposits in Mild Cognitive Disorder, Alzheimer's Disease and Early Frontotemporal Degeneration. #### Organization Study ID Info **ID:** PET-PIB-1 #### Organization **Class:** OTHER **Full Name:** IRCCS San Raffaele ### Status Module #### Completion Date **Date:** 2008-07-15 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-12-22 **Type:** ACTUAL **Last Update Submit Date:** 2023-12-12 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2008-07-15 **Type:** ACTUAL #### Start Date **Date:** 2008-05-17 **Type:** ACTUAL **Status Verified Date:** 2023-12 #### Study First Post Date **Date:** 2023-12-22 **Type:** ACTUAL **Study First Submit Date:** 2023-12-12 **Study First Submit QC Date:** 2023-12-12 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** IRCCS San Raffaele #### Responsible Party **Investigator Affiliation:** IRCCS San Raffaele **Investigator Full Name:** Chiti Arturo **Investigator Title:** Professor in Diagnostic Imaging and Radiotherapy Faculty of Medicine and Surgery, Vita-Salute San Raffaele University Director, Department of Nuclear Medicine, IRCCS Ospedale San Raffaele **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** Preclinical and clinical data have demonstrated the ability of the 11C-PIB tracer to selectively bind accumulations of amyloid protein, a neuropathological marker characteristic of the neurodegenerative pathologies covered by this study. The validation in larger groups of patients, and the comparison between the different clinical syndromes included in the spectrum, will allow the diagnostic and prognostic potential of the tracer to be evaluated, with important consequences for the clinical management of patients. In particular, the tracer could play a central role in the clinical management of patients with neurodegenerative diseases and cognitive impairment. Numerous pharmacological trials are currently underway, worldwide, for the validation of anti-amyloid drugs. In the future we could think about early monitoring with imaging of the effectiveness of the treatment. T he FDG PET technique can be of great help in obtaining relationships between radiation damage to the brain and possibly neurological and neuropsychological deficits associates. ### Conditions Module **Conditions:** - Alzheimer Disease ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 60 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Description:** Subjects considered suitable will undergo a clinical and neuropsychological evaluation and a PET/CT study with intravenous administration of 11C-PIB. **Name:** PET/CT with 11C-PiB **Type:** DIAGNOSTIC_TEST ### Outcomes Module #### Primary Outcomes **Description:** Comparative evaluation of the accumulation of amyloid protein in the various neurodegenerative conditions included with attention to the specificity of the tracer as an element of differential diagnosis. **Measure:** PET/CT with 11C-PiB to characterize amyloid protein deposits in patients with neurodegenerative diseases. **Time Frame:** 2 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Alzheimer's diagnosis according to the diagnostic criteria in use. Exclusion Criteria: * current or previous treatments with neuroactive drugs; * pregnancy or breastfeeding. **Maximum Age:** 80 Years **Minimum Age:** 40 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Adult patients with neurodegenerative diseases will be included: with mild cognitive disorder, Alzheimer's dementia and early temporal frontal degeneration. ### IPD Sharing Statement Module **IPD Sharing:** NO ## 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: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M3885 - Name: Alzheimer Disease - Relevance: HIGH - As Found: Alzheimer's Disease - ID: M4021 - Name: Amyloidosis - Relevance: LOW - As Found: Unknown - ID: M6301 - Name: Cognition Disorders - Relevance: HIGH - As Found: Cognitive Disorders - ID: M29705 - Name: Cognitive Dysfunction - Relevance: HIGH - As Found: Cognitive Disorders - 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: M4815 - Name: Mental Disorders - Relevance: LOW - As Found: Unknown - ID: M14473 - Name: Psychotic 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 - ID: D000060825 - Term: Cognitive Dysfunction - ID: D000003072 - Term: Cognition Disorders ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02213679 **Brief Title:** Guanidinoacetic Acid Loading for Chronic Fatigue Syndrome **Official Title:** Guanidinoacetic Acid Loading for Chronic Fatigue Syndrome #### Organization Study ID Info **ID:** 878/13-EUTC:03 #### Organization **Class:** OTHER **Full Name:** Center for Health, Exercise and Sport Sciences, Serbia ### Status Module #### Completion Date **Date:** 2015-07 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-02-01 **Type:** ESTIMATED **Last Update Submit Date:** 2017-01-30 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2015-06 **Type:** ACTUAL #### Start Date **Date:** 2014-08 **Status Verified Date:** 2017-01 #### Study First Post Date **Date:** 2014-08-11 **Type:** ESTIMATED **Study First Submit Date:** 2014-08-05 **Study First Submit QC Date:** 2014-08-07 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Center for Health, Exercise and Sport Sciences, Serbia #### Responsible Party **Investigator Affiliation:** Center for Health, Exercise and Sport Sciences, Serbia **Investigator Full Name:** Assoc. Prof. Sergej M. Ostojic, MD, PhD **Investigator Title:** Associate Professor of Biomedical Sciences **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** Chronic fatigue syndrome (CFS) is a debilitating condition of unknown etiology. Recent studies have shown that CFS is associated with impaired cellular energetics and low levels of phosphocreatine. Since guanidinoacetic acid (GAA) acts as a highly bioavailable precursor of creatine it may provide an ideal dietary supplement to facilitate treatment and perhaps prevention of CFS. The overall hypothesis to be evaluated is that medium-term supplementation with GAA will improve clinical outcomes in well-defined adult CFS patients via augmented provision of creatine. Specific aims: (1) To determine the effects of GAA on CFS symptomatology using a fatigue severity inventory, soreness of locomotive apparatus scales, and a health-related quality of life survey; (2) To determine the effect of GAA on creatine metabolism using laboratory studies and magnetic resonance spectroscopy; (3) To characterize the physiological effects of GAA on work capacity via actigraphy and exercise performance tests; and (4); To determine the prevalence of subjectively reported side-effects and biochemical adverse events associated with GAA intervention. **Detailed Description:** A variety of dietary interventions have been used in the management of CFS, yet no therapeutic modality demonstrated overall positive results in terms of effectiveness (Whiting et al. 2001). Previous studies have evaluated the effects of essential fatty acids, vitamins, minerals and/or enzymes, with findings do not support the use of a broad-spectrum nutritional supplement in treating CFS-related symptoms (Brouwers et al. 2002). Considering the fact that patients with CFS have lower levels of high-energy compounds (e.g. phosphocreatine, adenosine triphosphate) (Block et al. 1998), effective dietary treatment of CFS should be focused on providing compounds that facilitates cellular bioenergetics. Besides other candidate agents, guanidinoacetic acid (GAA) could be of particular interest since it occurs naturally in the human body and acts as an immediate precursor of creatine (Wyss and Kaddurah-Daouk, 2000). Due to its low cost and high bioavailability (Baker 2009), if proven effective dietary GAA may be suitable for use in broad CFS population. ### Conditions Module **Conditions:** - Chronic Fatigue Syndrome **Keywords:** - Creatine - Guanidinoacetic acid - Intervention - Fatigue - Muscle strength ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** QUADRUPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Count:** 20 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Supplementation with dietary guanidinoacetic acid **Intervention Names:** - Dietary Supplement: Guanidinoacetic acid **Label:** Guanidinoacetic acid **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Supplementation with cellulose **Intervention Names:** - Other: Placebo **Label:** Placebo **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Guanidinoacetic acid **Description:** Dietary supplement **Name:** Guanidinoacetic acid **Type:** DIETARY_SUPPLEMENT #### Intervention 2 **Arm Group Labels:** - Placebo **Description:** Placebo **Name:** Placebo **Type:** OTHER ### Outcomes Module #### Other Outcomes **Measure:** Health-related quality of life **Time Frame:** Baseline and after 3 months **Description:** Measurement of duration, frequency, and intensity of various types of human physical activity (exercise and nonexercise physical activity) **Measure:** Daily physical activity **Time Frame:** Baseline and after 3 months **Description:** For muscular performance, maximal voluntary strength of knee extensor muscles will be measured bilaterally using an isometric dynamometer during static knee joint movement with leg at 165º of flexion (180º = leg fully extended). The better of two efforts for each leg will be recorded with cumulative value presented as total isometric strength. **Measure:** Muscular strength **Time Frame:** Baseline and after 3 months **Measure:** Serum creatine **Time Frame:** Baseline and after 3 months **Measure:** Side-effects prevalence **Time Frame:** During 3 months of intervention #### Primary Outcomes **Measure:** Change in the Multidimensional Fatigue Inventory (MFI) score **Time Frame:** Baseline and afetr 3 months #### Secondary Outcomes **Measure:** Pain in the locomotive apparatus **Time Frame:** Baseline and after 3 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Adults who fulfilled the 1994 CDC criteria for CFS * Older than 18 years of age will be candidates for inclusion in the study. Exclusion Criteria: * Psychiatric comorbidity * Use of any dietary supplement within 4-weeks prior to the study commencing * Pregnant **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Belgrade **Country:** Serbia **Facility:** Center for Health, Exercise and Sport Sciences **Zip:** 11000 #### Overall Officials **Official 1:** **Affiliation:** Faculty of Sport and Physical Education, Novi Sad **Name:** Sergej M Ostojic, MD, PhD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ### References Module #### References **Citation:** Ostojic SM, Niess B, Stojanovic M, Obrenovic M. Creatine metabolism and safety profiles after six-week oral guanidinoacetic acid administration in healthy humans. Int J Med Sci. 2013;10(2):141-7. doi: 10.7150/ijms.5125. Epub 2013 Jan 3. **PMID:** 23329885 **Citation:** Ostojic SM, Niess B, Stojanovic M, Obrenovic M. Co-administration of methyl donors along with guanidinoacetic acid reduces the incidence of hyperhomocysteinaemia compared with guanidinoacetic acid administration alone. Br J Nutr. 2013 Sep 14;110(5):865-70. doi: 10.1017/S0007114512005879. Epub 2013 Jan 28. **PMID:** 23351309 **Citation:** Ostojic SM, Stojanovic M, Drid P, Hoffman JR. Dose-response effects of oral guanidinoacetic acid on serum creatine, homocysteine and B vitamins levels. Eur J Nutr. 2014 Dec;53(8):1637-43. doi: 10.1007/s00394-014-0669-0. Epub 2014 Feb 18. **PMID:** 24535415 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000004194 - Term: Disease - ID: D000010335 - Term: Pathologic Processes - ID: D000009135 - Term: Muscular Diseases - ID: D000009140 - Term: Musculoskeletal Diseases - ID: D000004679 - Term: Encephalomyelitis - ID: D000090862 - Term: Neuroinflammatory Diseases - ID: D000009422 - Term: Nervous System Diseases - ID: D000009468 - Term: Neuromuscular Diseases - ID: D000002908 - Term: Chronic Disease - ID: D000020969 - Term: Disease Attributes ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC01 - Name: Infections - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M16355 - Name: Syndrome - Relevance: HIGH - As Found: Syndrome - ID: M8364 - Name: Fatigue - Relevance: HIGH - As Found: Fatigue - ID: M18260 - Name: Fatigue Syndrome, Chronic - Relevance: HIGH - As Found: Chronic Fatigue Syndrome - ID: M12092 - Name: Muscular Diseases - Relevance: LOW - As Found: Unknown - ID: M12097 - Name: Musculoskeletal Diseases - Relevance: LOW - As Found: Unknown - ID: M7842 - Name: Encephalomyelitis - Relevance: LOW - As Found: Unknown - ID: M2803 - Name: Neuroinflammatory Diseases - Relevance: LOW - As Found: Unknown - ID: M12411 - Name: Neuromuscular 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 ### Condition Browse Module - Meshes - ID: D000015673 - Term: Fatigue Syndrome, Chronic - ID: D000013577 - Term: Syndrome - ID: D000005221 - Term: Fatigue ### Intervention Browse Module - Browse Branches - Abbrev: Ot - Name: Other Dietary Supplements - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: T385 - Name: Creatine - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05210179 **Brief Title:** Phase 2b Booster Vaccination (TURKOVAC) Against COVID-19 **Official Title:** Open-Label, Two Arms, Multi-Centered, Phase 2b Clinical Trial to Determine the Safety, Efficacy, and Immunogenicity of Booster Vaccination (TURKOVAC) Against SARS-CoV-2 #### Organization Study ID Info **ID:** TSB-VAC-COV-TUR-RF2B.05 #### Organization **Class:** OTHER_GOV **Full Name:** Health Institutes of Turkey ### Status Module #### Completion Date **Date:** 2023-05-02 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-11-29 **Type:** ACTUAL **Last Update Submit Date:** 2023-11-28 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2023-02-25 **Type:** ACTUAL #### Start Date **Date:** 2022-01-24 **Type:** ACTUAL **Status Verified Date:** 2023-02 #### Study First Post Date **Date:** 2022-01-27 **Type:** ACTUAL **Study First Submit Date:** 2022-01-26 **Study First Submit QC Date:** 2022-01-26 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER_GOV **Name:** Health Institutes of Turkey #### 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 is open-label, two arms, multi-centered, phase 2b clinical trial to determine the efficacy, safety, and immunogenicity of booster vaccination (TURKOVAC) against Covid-19. The primary aim of the study is to evaluate the efficacy of a booster dose of TURKOVAC vaccine administered to subjects who have passed at least 90 days and at most 240 days after the second dose of the first course of Comirnaty (Code name: BNT162b2) vaccine. **Detailed Description:** This phase 2b study aims to determine the efficacy, safety, and immunogenicity of a booster dose of TURKOVAC vaccine administered to subjects who have passed at least 90 days and at most 240 days after the second dose of the first course of Comirnaty (Code name: BNT162b2) vaccine. Efficacy will be evaluated by spike-specific antibody response and neutralizing antibody levels on days 0, 28 (all subjects), 48, 84 and 168 days (50% of subjects). For the booster dose, subjects will be assigned open-label according to randomization (1:1) for 2 different arms. Comparing the efficacy, safety, and immunogenicity results of different series of TURKOVAC vaccines (TURKOVAC-Koçak and TURKOVAC-Dollvet) produced in different production facilities are the secondary objectives of the study. The booster dose vaccine arms are as follows: * TURKOVAC-Koçak * TURKOVAC-Dollvet ### Conditions Module **Conditions:** - COVID-19 - Sars-CoV-2 Infection **Keywords:** - COVID-19 - SARS-CoV-2 Vaccine - Booster - Efficacy - Immunogenicity - Safety ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** National, Open-Label, Two Arms, Multi-Centered, Phase 2b Clinical Trial to Determine the Safety, Efficacy, and Immunogenicity of Booster Vaccination (TURKOVAC) Against SARS-CoV-2 ##### Masking Info **Masking:** NONE **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 65 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** The dose of the TURKOVAC vaccine will be 3 μg/0.5 mL and will be administered by injection into the deltoid muscle. **Intervention Names:** - Biological: TURKOVAC-Koçak **Label:** TURKOVAC-Koçak **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** The dose of the TURKOVAC vaccine will be 3 μg/0.5 mL and will be administered by injection into the deltoid muscle. **Intervention Names:** - Biological: TURKOVAC-Dollvet **Label:** TURKOVAC-Dollvet **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - TURKOVAC-Dollvet **Description:** TURKOVAC-Dollvet vaccines produced by Dollvet Veterinary Vaccine Pharmaceutical Biological Substance Production Industry Co., Ltd. **Name:** TURKOVAC-Dollvet **Type:** BIOLOGICAL #### Intervention 2 **Arm Group Labels:** - TURKOVAC-Koçak **Description:** TURKOVAC-Koçak vaccines produced by Koçak Farma Pharmaceutical and Chemical Industry Co., Ltd. **Name:** TURKOVAC-Koçak **Type:** BIOLOGICAL ### Outcomes Module #### Primary Outcomes **Description:** The change in the level of SARS-CoV2 Neutralizing Antibodies in the 28th day compared to the baseline **Measure:** Neutralizing Antibodies **Time Frame:** On days 0 and 28 **Description:** The change in the level of SARS-CoV2 Pseudo-Neutralizing Antibodies in the 28th day compared to the baseline **Measure:** Pseudo-Neutralizing Antibodies **Time Frame:** On days 0 and 28 **Description:** The change in the level of SARS-CoV2 anti-spike protein immunoglobulin G in the 28th day compared to the baseline **Measure:** Anti-spike protein immunoglobulin G **Time Frame:** On days 0 and 28 **Description:** The change in the level of T Cell Responses in the 28th day compared to the baseline **Measure:** T Cell Responses **Time Frame:** On days 0 and 28 #### Secondary Outcomes **Description:** Rates of subjects who have been found to be positive for COVID-19 by RT-PCR (real time polymerase chain reaction) test after administration of TURKOVAC vaccine. (On day 0 and it will be repeated if the subject develops symptoms during the follow-up period) **Measure:** Rates of subjects who have been found to be positive for COVID-19 **Time Frame:** On day 0 **Description:** Incidence of adverse reactions within 7 days of vaccination in all subjects. (Within 7 days of booster vaccination) **Measure:** Incidence of Adverse Reaction **Time Frame:** Within 7 days of booster vaccination **Description:** Incidence of Serious Adverse Events (SAE) up to day 168 after vaccination in all subjects. **Measure:** Incidence of Serious Adverse Events (SAE) **Time Frame:** Up to day 168 after booster vaccination ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Subjects willing and able to give signed informed consent to participate in study, 2. Healthy male or female aged 18 - 59 years (including both groups), 3. Subjects who were vaccinated with Comirnaty (Code name: BNT162b2) for 2 doses and who had a minimum of 90 days and a maximum of 240 days after the second dose, 4. Subjects with a minimum of 28 days and a maximum of 42 days between 1st and 2nd dose of Comirnaty (Code name: BNT162b2) vaccines, 5. Female subjects of childbearing potential and male subjects of to have child potential who are willing to ensure that they or their partner use effective contraception continuously from 1 month before vaccination to 3 months after booster vaccination, 6. In the opinion of the investigator, subjects capable and willing to comply with all study requirements, 7. Subjects are willing to agree to abstain from donating blood during the study. Exclusion Criteria: 1. Administration of any vaccine (registered or investigational) other than study intervention within 30 days before and after each study vaccine (one week for authorized seasonal flu vaccine or pneumococcal vaccine), 2. Known history of SARS-CoV-2 infection, 3. Pre-or planned use of another vaccine or product likely to affect the study (e.g. adenovirus vectored vaccines, any coronavirus vaccine), 4. Subjects who were pregnant at the time of enrollment or who plan to become pregnant within the first 3 months following vaccination and who are breastfeeding, 5. Subjects with fever (above 38°C) at the time of vaccination and/or up to 72 hours before (subjects may be screened again after acute condition has resolved), 6. Administration of immunoglobulins and/or any blood product within 3 months prior to vaccination, 7. Any confirmed or suspected immunosuppressive or immunodeficiency state; asplenia; recurrent severe infections and use of immunosuppressants (less than ≤14 days) in the last 6 months, excluding topical steroids or short-term oral steroids, 8. Possible history of allergic disease or reaction (e.g. to the active substance) by any component of the study vaccines, 9. Any history of anaphylaxis, 10. Current cancer diagnosis or treatment (excluding basal cell carcinoma of the skin and cervical carcinoma in situ), 11. History of bleeding disorder (e.g. factor deficiency, coagulopathy or platelet disorder), or prior history of significant bleeding or bruising following intramuscular injections or venipuncture, 12. Continued use of anticoagulants such as coumarins and related anticoagulants (i.e. warfarin) or new oral anticoagulants (e.g. apixaban, rivaroxaban, dabigatran and edoxaban), 13. Cerebral venous sinus thrombosis, antiphospholipid syndrome, or a history of heparin-induced thrombocytopenia and thrombosis (HITT or HIT type 2), 14. Suspected or known current alcohol or drug addiction, 15. Any other significant disease, disorder or finding that could significantly increase the subject's risk for participation in the study, affect the subject's ability to participate in the study, or impair the interpretation of study data; severe and/or uncontrolled cardiovascular disease, respiratory disease, gastrointestinal disease, liver disease, kidney disease, endocrine disorder, and neurological disease (mild/moderate well-controlled comorbidities are permitted), 16. History of active or previous autoimmune neurological disorders (e.g. multiple sclerosis, Guillain-Barre syndrome, transverse myelitis) (Bell's palsy will not be an exclusion criterion), 17. Subjects with severe renal impairment or liver failure, 18. Subjects who will undergo scheduled elective surgery during the study, 19. Subjects with a life expectancy of less than 6 months, 20. Subject who participated in another clinical trial study involving an investigational product in the past 12 weeks, 21. In case of clinical necessity, a COVID-19 PCR (polymerase chain reaction) test will be requested from the subjects, and subjects who are positive will be excluded from the study, 22. Known history of SARS-CoV-2 infection, 23. Acute respiratory disease (moderate or severe illness with or without fever). (Subjects may be screened again after acute condition has resolved), 24. Fever (greater than 37.8°C as measured by ear) (Subjects can be enrolled again after acute condition improves), 25. Insufficient level of Turkish to perform the informed consent, except where briefing by an independent witness can be provided and is available. **Healthy Volunteers:** True **Maximum Age:** 59 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Ankara **Country:** Turkey **Facility:** T.R. Ministry of Health Ankara City Hospital, Infectious Diseases and Clinical Microbiology Clinic **Zip:** 06800 **Location 2:** **City:** Antalya **Country:** Turkey **Facility:** University of Health Sciences, Antalya Training and Research Hospital, Family Medicine Clinic **Location 3:** **City:** Bolu **Country:** Turkey **Facility:** Abant İzzet Baysal University Izzet Baysal Training and Research Hospital **Location 4:** **City:** Istanbul **Country:** Turkey **Facility:** İstanbul University Cerrahpaşa, Cerrahpaşa Faculty of Medicine, Department of Internal Medicine - Department of Infectious Diseases and Clinical Microbiology **Location 5:** **City:** Istanbul **Country:** Turkey **Facility:** T.R. Ministry of Health Başakşehir Çam ve Sakura City Hospital, Clinical Microbiology Clinic **Location 6:** **City:** Istanbul **Country:** Turkey **Facility:** T.R. Ministry of Health Istanbul Provincial Health Directorate Prof. Dr. Cemil Taşçıoğlu City Hospital, Infectious Diseases and Clinical Microbiology Clinic **Location 7:** **City:** İzmir **Country:** Turkey **Facility:** T.R. Ministry of Health İzmir Provincial Health Directorate İzmir Health Sciences University Tepecik Training and Research Hospital, Infectious Diseases **Zip:** 35180 **Location 8:** **City:** Kayseri **Country:** Turkey **Facility:** T.R. Ministry of Health Kayseri City Training and Research Hospital, Infectious Diseases and Clinical Microbiology Department **Location 9:** **City:** Kocaeli **Country:** Turkey **Facility:** University of Health Sciences, Derince Training and Research Hospital, Department of Infectious Diseases and Clinical Microbiology #### Overall Officials **Official 1:** **Affiliation:** Faculty Member **Name:** Bedia Dinç, Assoc. Prof. **Role:** PRINCIPAL_INVESTIGATOR ## 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 ### 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: COVID-19 - ID: M10283 - Name: Infections - Relevance: LOW - As Found: Unknown - ID: M6368 - Name: Communicable Diseases - 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: 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 ### Condition Browse Module - Meshes - ID: D000086382 - Term: COVID-19 ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M17360 - Name: Vaccines - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05372679 **Acronym:** REDUCED **Brief Title:** REDUCED 1 - Renal Denervation Using Ultrasonic Catheter EmitteD Energy Study / **Official Title:** Clinical Evaluation of the Therapeutic Intra-Vascular Ultrasound (TIVUS™) System for Renal Denervation in Patients With Uncontrolled Stage 2 Hypertension #### Organization Study ID Info **ID:** CLNS05-001 #### Organization **Class:** INDUSTRY **Full Name:** SoniVie Inc. ### Status Module #### Completion Date **Date:** 2025-12 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-02-07 **Type:** ACTUAL **Last Update Submit Date:** 2024-02-05 **Overall Status:** ACTIVE_NOT_RECRUITING #### Primary Completion Date **Date:** 2025-12 **Type:** ESTIMATED #### Start Date **Date:** 2022-11-11 **Type:** ACTUAL **Status Verified Date:** 2024-02 #### Study First Post Date **Date:** 2022-05-13 **Type:** ACTUAL **Study First Submit Date:** 2022-05-09 **Study First Submit QC Date:** 2022-05-09 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** SoniVie Inc. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** True **Is FDA Regulated Drug:** False **Is Unapproved Device:** True **Oversight Has DMC:** True ### Description Module **Brief Summary:** This is a prospective, multicenter, non-randomized, open-label clinical study of the safety and performance of the TIVUS™ System in subjects with uncontrolled stage 2 hypertension in order to assess the safety and effectiveness of the TIVUS™ System when used for renal artery denervation. The study will assess the acute and chronic safety of the procedure as well as the reduction in 24-hour ambulatory mean systolic blood pressure from baseline to 3 months after the procedure. **Detailed Description:** Nearly half of adults in the United States (108 million, or 45%) have hypertension defined as a systolic blood pressure ≥ 130 mm Hg or a diastolic blood pressure ≥ 80 mm Hg or are taking medication for hypertension. Hypertension is associated with cardiovascular and renal pathologies and leads to death if not treated appropriately. The cardiovascular risk factor doubles with each 20/10 mm Hg elevation in BP values above 115/75 mm Hg. The current first-line therapy for hypertension involves change in life-style (i.e., diet and exercise) and various medications. However, only about 1 in 4 treated adults (24%) with hypertension have their condition under control. Thus, they are at increased risk for the major side effects of chronically elevated blood pressure, myocardial infarction, stroke, renal disease and heart failure. High blood pressure was a primary or contributing cause of death for more than 494,873 people in the United States in 2018, and was estimated to affect around 1.13 billion people worldwide and resulted in approximately 10 million deaths worldwide, in 2015. About half of adults (45%) with uncontrolled hypertension have a blood pressure of 140/90 mm Hg or higher, now defined as Stage 2 hypertension. This includes 37 million U.S. adults. About 30 million adults are recommended to take medication. Almost two out of three of this group (19 million) have a blood pressure of 140/90 mm Hg or higher. The other 17 million adults are taking medications but are unable to lower their blood pressure below 140/90 mm Hg and are thus at increased risk for the risks associated with uncontrolled high blood pressure9. High blood pressure costs the United States about $131 billion each year, averaged over 12 years from 2003 to 2014. In the past decade, several devices were developed in order to target the autonomic nervous system and lower BP in patients with uncontrolled hypertension. These devices aim to provide additional treatment option for patients who do not respond to antihypertensive medication. These devices target other BP regulating mechanisms such as baroreflex activation, deep brain stimulation carotid body ablation, direct vagus nerve stimulation, cardiac neuromodulation, central iliac arteriovenous coupler as well as other devices are currently under research, however, there is no medical device that has been approved so far. The Therapeutic Intra Vascular UltraSound (TIVUS™) System is designed for renal artery nerve ablation using transluminal ultrasound (US). The TIVUS™ System is a high intensity, non-focused, ultrasound catheter system, which enables remote, localized, controlled, and repeatable thermal modulation of nerves adjacent to arterial vessel wall for performing safe and effective therapeutic artery sympathetic denervation. The ultrasonic energy is transmitted from a catheter positioned within the artery lumen, while avoiding direct contact with the artery wall. ### Conditions Module **Conditions:** - Uncontrolled Stage 2 Hypertension ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP **Intervention Model Description:** Prospective, multicenter, non-randomized, open-label clinical study ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 25 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Denervating the sympathetic nerves surrounding the renal vasculature using unfocused ultrasound **Intervention Names:** - Device: Renal denervation **Label:** Renal denervation **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Renal denervation **Description:** The TIVUS system will be used for renal denervation **Name:** Renal denervation **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** acute treatment related adverse events including including all-cause mortality, acute kidney injury, significant embolic event, renal artery perforation or dissection requiring intervention, vascular complications, stroke, myocardial infarction, or transient ischemic attack, deterioration in renal function end-stage renal failure, hospital admission for hypertensive crisis not related to confirmed nonadherence with medications or the protocol, new renal artery stenosis of more than 70% and major access site complications or cardiovascular complications **Measure:** Incidence of Adverse Events **Time Frame:** from baseline to 1 month post procedure **Description:** chronic treatment related adverse events including including all-cause mortality, acute kidney injury, significant embolic event, renal artery perforation or dissection requiring intervention, vascular complications, stroke, myocardial infarction, or transient ischemic attack, deterioration in renal function end-stage renal failure, hospital admission for hypertensive crisis not related to confirmed nonadherence with medications or the protocol, new renal artery stenosis of more than 70% and major access site complications or cardiovascular complications **Measure:** Incidence of Adverse Events **Time Frame:** from baseline to 12 month post procedure **Description:** Reduction in daytime ambulatory mean systolic blood pressure from baseline to 3 months after the procedure. **Measure:** Change in mean daytime ambulatory systolic BP **Time Frame:** From baseline to 3 months post procedure #### Secondary Outcomes **Description:** Reduction of 24-hour ambulatory mean systolic and diastolic blood pressure from baseline **Measure:** Change in mean 24-hr ambulatory systolic and diastolic bp **Time Frame:** 3, 6, 12, 24, 36 months **Description:** Reduction of 24-hour ambulatory mean diastolic blood pressure from baseline **Measure:** Change in mean 24-hr ambulatory diastolic bp **Time Frame:** 3 months **Description:** Reduction of daylight ambulatory mean systolic blood pressure from baseline **Measure:** Change in mean daytime ambulatory systolic bp **Time Frame:** 6, 12, 24, 36 months **Description:** Reduction of nighttime ambulatory mean systolic and diastolic blood pressure from baseline **Measure:** Change in mean nighttime ambulatory systolic and diastolic bp **Time Frame:** 3, 6, 12, 24, 36 months **Description:** Reduction of office mean systolic and diastolic blood pressure from baseline **Measure:** Change in mean office systolic and diastolic BP **Time Frame:** 3, 6, 12, 24, 36 months **Description:** Reduction in mean 24-hour ambulatory pulse pressure from baseline **Measure:** Change in mean 24-hr ambulatory pulse **Time Frame:** 3, 6, 12, 24, 36 months **Description:** Number of patients achieving target 24-hour ambulatory mean systolic blood pressure (\<130 mm Hg) **Measure:** Rate of patients achieving target 24-hr ambulatory mean systolic BP **Time Frame:** 3, 6, 12, 24, 36 months **Description:** Number of patients achieving target office systolic blood pressure (\<140 mm Hg) **Measure:** Rate of patients achieving target office systolic BP **Time Frame:** 3, 6, 12, 24, 36 months **Description:** Reduction in the number of antihypertensive medications taken and/or the dose taken **Measure:** Change in number of antihypertensive medications taken **Time Frame:** 6, 12 months **Description:** Percentage of patients who respond to the treatment, with response defined as a reduction of at least 10mm in mean 24-hour ambulatory systolic blood pressure **Measure:** Percentage of patients who respond to treatment **Time Frame:** 3, 6, 12 months **Description:** Incidence of hypertensive or hypotensive emergency episodes resulting in hospitalization **Measure:** Incidence of hypertensive or hypotensive emergency episodes **Time Frame:** 3, 6, 12 months **Description:** Changes in renal function, based on laboratory parameters such as eGFR (CKD-EPI) and serum creatinine **Measure:** Change in renal function based on laboratory parameters **Time Frame:** 3, 6, 12 months **Description:** Changes in renal function, based on artery stenosis (\>70%) **Measure:** Change in renal function based on artery stenosis **Time Frame:** 6, 12 months **Description:** Episodes of heart failure events **Measure:** Incidence of heart failure **Time Frame:** 3, 6, 12 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Written informed consent to participate in the study obtained from the patient, according to local regulations, prior to initiation of any study mandated procedure. 2. Male or female, ≥ 18 years of age and ≤ 80 years of age at the time of screening. 3. Individual has office systolic blood pressure (SBP) ≥ 140 mm Hg but \< 180 mm Hg, and a diastolic blood pressure (DBP) ≥ 90 mm Hg but \< 110 mm Hg based on an average of 3 office seated blood pressure readings measured by a manual, automated or semi-automated validated BP monitor, on current medications. 4. Documented mean daytime ABPM systolic blood pressure (SBP) ≥ 135 mm Hg but \< 180 mm Hg, after a 4 week stabilization period. 5. Patient is either on at least one anti-hypertensive medication at maximal tolerated dose with a medically documented intolerance to one or more medications, or on three medications where one is a diuretic. 6. Patient is willing and expected to maintain their anti-hypertensive medication regimen for at least 3 months. 7. Patient is able and willing to comply with all study procedures. Exclusion Criteria: 1. Patient has been previously diagnosed with abnormal renal artery anatomy and/or renal anatomy such as single kidney, other renal development anomaly such as ectopic or horseshoe kidney, or polycystic kidney disease precluding renal denervation therapy as detailed in the angiographic exclusion criteria. 2. Pregnant or breastfeeding women or women planning a pregnancy within 12 months of study enrollment. 3. Patient has an estimated glomerular filtration rate (eGFR) of \< 40mL/min/1.73m2 CKD-EPI as calculated using the CKD-EPI 2021 equation. 4. Patients with uncontrolled rapid AF. 5. Patient has had a previous renal denervation procedure. 6. Patients with daytime ABPM mean systolic blood pressure (SBP) \> 180 mm Hg. 7. Patient has Type 1 diabetes or poorly controlled Type 2 diabetes (HbA1c \> 9%). 8. Patients with history of myocardial infarction, unstable angina pectoris, heart failure, cerebrovascular accident, or widespread atherosclerosis, with documented intravascular thrombosis or unstable plaques. 9. Patient has a planned major surgery or cardiovascular intervention in the next 6 months. 10. Patient who has undergone a major surgery or cardiovascular intervention in the previous 3 months. 11. Patient has frequent intermittent or chronic pain that results in treatment with nonsteroidal anti-inflammatory drugs (NSAIDs) for two or more days per week over the month prior to enrollment. 12. Patient is taking immunosuppressive therapy for diseases featuring vasculitis. 13. Patient on anticoagulant therapy that cannot be temporarily withheld for study procedure. 14. Contraindication to recommended denervation procedure medications or intravascular contrast that cannot be adequately controlled with pre-medication. 15. Concurrent enrollment in another device or drug trial that has not completed the primary endpoint or clinically interferes with the current study endpoints. For patient previously participating in a drug trial, allow a wash-off period of at least 5 half-lives of the investigational drug. 16. Patient with significant co-morbid condition(s) which, at the discretion of the PI, are deemed to prohibit study entry. 17. Patient works the night shift. 18. Patient has valvular stenosis with risk of cardiac event with significant and/or abrupt decline in systolic blood pressure. 19. Untreated secondary cause of hypertension. 20. Device therapy within the past five years for the treatment of hypertension. 21. Prior renal stent or angioplasty. 22. Life expectancy \<1 year. 23. Patients with history of renal transplant or planned renal transplantation within the next year. 24. History of one or more episodes of severe orthostatic hypotension within the past year. 25. Patient has unstable cardiac or pulmonary disease (including pulmonary hypertension) requiring chronic oxygen support. 26. Uncontrolled or inadequately treated bleeding diathesis. 27. Evidence of active infection within 7 days of procedure Angiographic Exclusion Criteria The following characteristics identified either on the renal artery CT scan or on the Eligibility II Renal artery Angiogram will prevent the patient from being included: 1. Main renal arteries \< 4 mm in lumen diameter or \< 20 mm in length; accessory renal arteries, if present, \< 4 mm in lumen diameter or \<10 mm in length. 2. Aorto-renal angle that in the operator's opinion prevents a safe cannulation of the renal artery. 3. Severe common femoral artery, common and/or external iliac artery, renal, iliac or aortic calcification or tortuosity that may compromise the safe performance and completion of the TIVUS™ procedure. 4. Hemodynamically or anatomically significant renal artery abnormality or stenosis in either renal artery which, in the operator's opinion, would interfere with safe cannulation of the renal artery or meets local standards for surgical repair or interventional dilation (NOTE: vessel areas with calcification and fibromuscular dysplasia (FMD) should be avoided as intended treatment areas). 5. Any renal artery stenosis \> 30% by visual assessment. 6. Any renal artery aneurysm (\>50% of the main renal artery reference vessel diameter by visual estimate). 7. Presence of fibromuscular dysplasia 8. Significant renal artery atheroma, aneurysm, calcification in the target vessel identified on CT Angiogram **Maximum Age:** 80 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Birmingham **Country:** United States **Facility:** Cardiology P.C. **State:** Alabama **Zip:** 35211 **Location 2:** **City:** Jonesboro **Country:** United States **Facility:** ST Bernards Medical Center **State:** Arkansas **Zip:** 72401 **Location 3:** **City:** Little Rock **Country:** United States **Facility:** Arkansas Heart Hospital **State:** Arkansas **Zip:** 72211 **Location 4:** **City:** Bridgeport **Country:** United States **Facility:** Bridgeport Hospital **State:** Connecticut **Zip:** 06610 **Location 5:** **City:** Springfield **Country:** United States **Facility:** Prairie Education and Research Cooperative **State:** Illinois **Zip:** 62701 **Location 6:** **City:** Minneapolis **Country:** United States **Facility:** Minneapolis Heart Institution Foundation **State:** Minnesota **Zip:** 55407 **Location 7:** **City:** New York **Country:** United States **Facility:** Columbia University/NYPH **State:** New York **Zip:** 10032 **Location 8:** **City:** Raleigh **Country:** United States **Facility:** NC Heart and Vascular Research, LLC **State:** North Carolina **Zip:** 27607 **Location 9:** **City:** Charleston **Country:** United States **Facility:** Medical University South Carolina **State:** South Carolina **Zip:** 29425 #### Overall Officials **Official 1:** **Affiliation:** Columbia University/NYPH **Name:** Ajay Kirtane, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - 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: M10024 - Name: Hypertension - Relevance: HIGH - As Found: Hypertension - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000006973 - Term: Hypertension ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02619279 **Brief Title:** A Livelihood Intervention for Impoverished Women and Children in Rural Uganda **Official Title:** A Livelihood Intervention for Impoverished Women and Children in Rural Uganda: Randomized Controlled Trial #### Organization Study ID Info **ID:** MH096620-S4 #### Organization **Class:** OTHER **Full Name:** Massachusetts General Hospital ### Status Module #### Completion Date **Date:** 2016-12 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2015-12-02 **Type:** ESTIMATED **Last Update Submit Date:** 2015-12-01 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2016-12 **Type:** ESTIMATED #### Start Date **Date:** 2015-11 **Status Verified Date:** 2015-12 #### Study First Post Date **Date:** 2015-12-02 **Type:** ESTIMATED **Study First Submit Date:** 2015-08-15 **Study First Submit QC Date:** 2015-12-01 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Mbarara University of Science and Technology #### Lead Sponsor **Class:** OTHER **Name:** Massachusetts General Hospital #### Responsible Party **Investigator Affiliation:** Massachusetts General Hospital **Investigator Full Name:** Alexander Tsai **Investigator Title:** Assistant Professor of Psychiatry **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The objective of the study is to determine whether a livelihood intervention can improve health and schooling outcomes of the children of woman participating in a livelihood intervention in rural Uganda. ### Conditions Module **Conditions:** - Indigency ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE #### Enrollment Info **Count:** 150 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants' mothers will receive a livelihood intervention package consisting of a orientation and training and a loan package of chickens and associated implements to create poultry microenterprises **Intervention Names:** - Other: Livelihood intervention **Label:** Immediate treatment **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants' mothers will receive no intervention but will be placed on a waitlist to receive the intervention after a 12-month delay. **Label:** Delayed treatment **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Immediate treatment **Description:** Participants' mothers will receive a livelihood intervention package consisting of a orientation and training and a loan package of chickens and associated implements to create poultry microenterprises. **Name:** Livelihood intervention **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Height-for-age calculated based on child height and child age, measured for children under 5 years of age **Measure:** Stunting based on Anthropometric Measurements **Time Frame:** 12 months **Description:** Weight-for-height calculated based on child height and child weight, measured for children under 5 years of age **Measure:** Weight-for-Height based on Anthropometric Measurements **Time Frame:** 12 months **Description:** Raven's Colored Progressive Matrices test score (range, 0-18), measured for children aged 5-8 years **Measure:** Raven's Colored Progressive Matrices test **Time Frame:** 12 months **Description:** Mullen Early Learning Scales: 144 items total, divided across 4 subtests in visual receptive organization, visual expressive organization, language expressive organization, and language receptive organization **Measure:** Mullen Early Learning Scales **Time Frame:** 12 months **Description:** Home Observation of the Measurement of Environment, modified for the Ugandan context (58 items distributed across the following domains: Responsivity, Encouragement of maturity, Emotional climate, Learning materials and opportunities, Enrichment, Family companionship, Family integration and Physical environment) **Measure:** Home Observation of the Measurement of Environment Scale **Time Frame:** 12 months #### Secondary Outcomes **Description:** Average time spent on school and schoolwork in the past 7 days (number of hours) **Measure:** Child Educational Investment Scale **Time Frame:** 12 months **Description:** Average time spent on household chores and playing in the past 7 days (number of hours) **Measure:** Child Non-Educational Investment Scale **Time Frame:** 12 months **Description:** Average time spent on reading to children or helping with homework in past 7 days (number of hours) **Measure:** Mother's Educational Investment Scale **Time Frame:** 12 months **Description:** Mother's aspiration for child's educational attainment, ranging from 1 (finish P7 primary school) to 5 (finish graduate school) **Measure:** Mother's Aspirations for Child Education Scale **Time Frame:** 12 months **Description:** Mother's aspirations for child's adult employment by age 25 (with the option to select one of 33 different blue-collar and white-collar occupational categories) **Measure:** Mother's Aspirations for Child Career Scale **Time Frame:** 12 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * All children under 18 years Exclusion Criteria: * Adults older than 18 years **Healthy Volunteers:** True **Maximum Age:** 18 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Alexander C Tsai, MD, PhD **Phone:** 617-724-1120 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Bernard Kakuhikire, MBA **Phone:** 256-075-250-4030 **Role:** CONTACT #### Locations **Location 1:** **City:** Mbarara **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Alexander C Tsai, MD, PhD - **Phone:** 617-724-1120 - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Bernard Kakuhikire, MBA - **Phone:** 256-075-250-4030 - **Role:** CONTACT ***Contact 3:*** - **Name:** Alexander C Tsai, MD, PhD - **Role:** PRINCIPAL_INVESTIGATOR ***Contact 4:*** - **Name:** Bernard Kakuhikire, MBA - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Uganda **Facility:** Mbarara University of Science and Technology **Status:** RECRUITING #### Overall Officials **Official 1:** **Affiliation:** Massachusetts General Hospital **Name:** Alexander C Tsai, MD, PhD **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** Mbarara University of Science and Technology **Name:** Bernard Kakuhikire, MBA **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00541879 **Acronym:** DKENERGY **Brief Title:** Obesity and Diabetes Prevention Through Science Enrichment **Official Title:** Type 2 Diabetes Knowledge: Obesity and Diabetes Prevention Through Science Enrichment #### Organization Study ID Info **ID:** H 04-257 RR25 RR 020469 #### Organization **Class:** OTHER **Full Name:** Colorado State University #### Secondary ID Infos **ID:** R 25 RR 020469 **ID:** R 25 RR 015646 ### Status Module #### Completion Date **Date:** 2011-09 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2014-11-07 **Type:** ESTIMATED **Last Update Submit Date:** 2014-11-05 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2011-09 **Type:** ACTUAL #### Start Date **Date:** 2001-08 **Status Verified Date:** 2014-11 #### Study First Post Date **Date:** 2007-10-10 **Type:** ESTIMATED **Study First Submit Date:** 2007-10-09 **Study First Submit QC Date:** 2007-10-09 ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** National Center for Research Resources (NCRR) **Class:** UNKNOWN **Name:** Poudre School District, Fort Collins, CO #### Lead Sponsor **Class:** OTHER **Name:** Colorado State University #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Type 2 diabetes and obesity in children continue to increase at alarming rates with devastating results. However, both these metabolic diseases are largely preventable through adoption of a healthy lifestyle, an understanding of what happens to food in the body, energy balance and some simple aspects of glucose regulation. Can elementary school children be taught the knowledge and skills necessary to prevent type 2 diabetes and obesity? Children need to learn this essential knowledge and practice these important health behavior skills. Elementary school may be an ideal place to master this subject that is a direct and logical extension of current health curricula including nutrition and physical activity blended with science and math. **Detailed Description:** Biomedical scientists lead a classroom series of interactive, inquiry-based science and health explorations directed at type 2 diabetes, obesity and their prevention in three elementary schools serving children at high risk for type 2 diabetes. Diabetes knowledge was measured in children and parents at the beginning, end and 9 months after the end of the intervention using validated questionnaires. ### Conditions Module **Conditions:** - Type 2 Diabetes - Obesity **Keywords:** - Childhood - Prevention - Energy balance - Glucose regulation - Healthy lifestyle - Healthy eating - Active living ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 2909 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Elementary school children -grades 2-6 participating in Program ENERGY **Intervention Names:** - Behavioral: Program ENERGY **Label:** I **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Elementary school children in grades 2-6 matched to the intervention classes not receiving any intervention **Intervention Names:** - Other: comparison **Label:** II **Type:** SHAM_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - I **Description:** Weekly or biweekly classroom and gym based science enrichment focused on how the body works including blood glucose regulation, healthy eating and physical activity, diabetes abd how it can be prevented **Name:** Program ENERGY **Type:** BEHAVIORAL #### Intervention 2 **Arm Group Labels:** - II **Name:** comparison **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Measure:** Diabetes knowledge and prevention behaviors in elementary school children **Time Frame:** one-three school year(s) #### Secondary Outcomes **Measure:** Parental diabetes knowledge, self-reported physical activity **Time Frame:** one -three school year(s) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Elementary school child in selected intervention or comparison classes/schools **Healthy Volunteers:** True **Maximum Age:** 13 Years **Minimum Age:** 6 Years **Sex:** ALL **Standard Ages:** - CHILD ### Contacts Locations Module #### Locations **Location 1:** **City:** Fort Collins **Country:** United States **Facility:** Putnam, Tavelli, Bennett and Dunn Elementary Schools **State:** Colorado **Zip:** 80524 #### Overall Officials **Official 1:** **Affiliation:** Colorado State University **Name:** L. Arthur Campfield, PhD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### See Also Links **Label:** Program ENERGY website **URL:** http://www.ProgramENERGY.org ## Derived Section ### Condition Browse Module - Ancestors - ID: D000044882 - Term: Glucose Metabolism Disorders - ID: D000008659 - Term: Metabolic Diseases - ID: D000004700 - Term: Endocrine System Diseases - ID: D000050177 - Term: Overweight - ID: D000044343 - Term: Overnutrition - ID: D000009748 - Term: Nutrition Disorders - ID: D000001835 - Term: Body Weight ### 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 - ID: M7119 - Name: Diabetes Mellitus, Type 2 - Relevance: HIGH - As Found: Type 2 Diabetes - ID: M12701 - Name: Obesity - Relevance: HIGH - As Found: Obesity - 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 - 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 ### Condition Browse Module - Meshes - ID: D000003920 - Term: Diabetes Mellitus - ID: D000009765 - Term: Obesity - ID: D000003924 - Term: Diabetes Mellitus, Type 2 ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03648879 **Brief Title:** Confocal Endoscopic Microscopy for Detection of Early Stage Gastric Cancer in Subjects With Hereditary Diffuse Gastric Cancer Syndrome **Official Title:** Phase II Study Evaluating Confocal Endoscopic Microscopy for Detection of Early Stage Gastric Cancer in Subjects With Hereditary Diffuse Gastric Cancer Syndrome #### Organization Study ID Info **ID:** 180141 #### Organization **Class:** NIH **Full Name:** National Institutes of Health Clinical Center (CC) #### Secondary ID Infos **ID:** 18-C-0141 ### Status Module #### Completion Date **Date:** 2020-05-05 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-07-12 **Type:** ACTUAL **Last Update Submit Date:** 2021-06-17 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2020-04-20 **Type:** ACTUAL #### Results First Post Date **Date:** 2021-03-25 **Type:** ACTUAL **Results First Submit Date:** 2021-03-02 **Results First Submit QC Date:** 2021-03-02 #### Start Date **Date:** 2019-02-11 **Type:** ACTUAL **Status Verified Date:** 2021-06 #### Study First Post Date **Date:** 2018-08-28 **Type:** ACTUAL **Study First Submit Date:** 2018-08-24 **Study First Submit QC Date:** 2018-08-24 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** NIH **Name:** National Cancer Institute (NCI) #### Responsible Party **Investigator Affiliation:** National Cancer Institute (NCI) **Investigator Full Name:** Jeremy Davis, M.D. **Investigator Title:** Principal Investigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** True **Is FDA Regulated Drug:** False **Is US Export:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Background: People with hereditary gastric cancer syndrome are at increased risk of getting cancer in their stomach. These people should have regular endoscopies and biopsies to check for cancer if they are choosing to keep their stomach. Researchers want to see if they can improve the detection of cancer by endoscopy. Improved endoscopies could better detect early signs of cancer in people with this syndrome. Objective: To see if a small microscope attached to an endoscope to inspect the stomach lining is better than regular endoscopy to find the first signs of cancer in the stomach. Eligibility: People ages 18 and older who have a personal or family history of a hereditary gastric cancer syndrome or have a mutation that is known to lead to gastric cancer Design: Participants will be screened over the phone or in person with: * Personal and family medical history * Review of their medical records Participants will have a physical exam. Then they will be put under general anesthesia. They will have an endoscopy. A lighted tube will be inserted into the mouth and go down to the stomach. First, the standard device will be used. Then participants will be injected with fluorescein. This is a contrast agent. Then the microscope will be added to the tube and the endoscopic evaluation of the stomach will be repeated. During the procedure, biopsies will be taken from different areas of the stomach. Participants will be observed for a few hours after the procedure. About 14 days after the endoscopy, participants will be asked to return to the clinic for a follow-up visit. This visit can also be conducted over the phone. **Detailed Description:** Background: Hereditary Diffuse Gastric Cancer (HDGC) syndrome is caused by a germline mutation in the Cadherin 1 (CDH1) gene. Carriers of this mutation have a 56-70% lifetime risk of developing gastric adenocarcinoma. Current international guidelines recommend endoscopic screening of CDH1 mutation carriers that consists of systematic biopsies of an otherwise normal appearing stomach. However, this approach lacks sufficient sensitivity for detecting intramucosal foci of signet ring cells (SRC), which are pathognomonic of HDGC syndrome. The goal of the current study is to utilize confocal endoscopic microscopy (CEM) for screening the gastric mucosa in this high-risk population. Objective: Determine if confocal endoscopic microscopy (CEM) will afford greater sensitivity for detection of signet ring cells (SRC) foci in CDH1 germline mutation carriers. Eligibility: CDH1 germline mutation carriers, or those who meet clinical criteria for HDGC testing but have tested negative for a CDH1 gene mutation or those who have other germline mutations suspected to be, or reported to be, associated with HDGC (e.g. Catenin Alpha 1 (CTNNA1). Design: Phase II, single-institution study of CEM for detection of intramucosal SRC foci compared to current systematic gastric mapping procedure. ### Conditions Module **Conditions:** - Gastric Cancer - Gastric Neoplasms **Keywords:** - Diagnostic - Diagnosis - EGD - Examination of Stomach ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** DIAGNOSTIC #### Enrollment Info **Count:** 37 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Upper white-light endoscopy and confocal endoscopic microscopy **Intervention Names:** - Device: Endoscope+Cellvizio(R) 100 microscope **Label:** 1/Arm 1 - Upper white-light endoscopy and confocal endoscopic microscopy **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - 1/Arm 1 - Upper white-light endoscopy and confocal endoscopic microscopy **Description:** Patients will undergo white-light, upper endoscopy. In addition, during this endoscopy patients will undergo Confocal Endoscopic Microscopy (CEM) using the Cellvizio probe (Mauna Kea Technologies) to scan the same anatomic zones. **Name:** Endoscope+Cellvizio(R) 100 microscope **Type:** DEVICE ### Outcomes Module #### Other Outcomes **Description:** Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. **Measure:** Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0) **Time Frame:** Date of enrollment to date off study, approximately 11 months and 19 days. #### Primary Outcomes **Description:** Sensitivity for detection of SRC foci in CDH1 germline mutation carriers was assessed by confocal endoscopic microscopy (CEM) compared to the current method of and standard white light endoscopy. Sensitivity in CEM and WLE is defined as the percentage of participants with detectable cancer on endoscopic biopsy. **Measure:** Percentage of Participants With Detectable Confocal Endoscopic Microscopy (CEM) w/Greater Sensitivity for Detection of Signet Ring Cells (SRC)Foci in Cadherin-1 (CDH1) Germline Mutation Carriers Compared to Current Method of Standard White Light Endoscopy **Time Frame:** 14 days #### Secondary Outcomes **Description:** In participants who choose to undergo prophylactic total gastrectomy with permanent pathologic analysis, the false negative rate (the fraction of participants who have SRC foci not identified by CEM and White Light Endoscopy techniques) will be determined and reported. The fraction percentage of patients who underwent prophylactic total gastrectomy with findings of SRC foci in the gastrectomy specimen will represent the denominator (number) and the number of patients with negative findings by CEM and White Light Endoscopy (WLE) will represent the numerator (number) to generate the false negative detection rate (fraction) for CEM and WLE, respectively. **Measure:** Percentage of Participants Who Have Signet Ring Cells (SRC) Foci Not Identified by Confocal Endoscopic Microscopy (CEM) **Time Frame:** Date of enrollment to date of prophylactic gastrectomy, approximately 6 months or an average of 1 month up to 12 months. ### Eligibility Module **Eligibility Criteria:** * INCLUSION CRITERIA: * Patients with Cadherin-1 (CDH1) germline mutation known to be pathogenic or likely pathogenic, which may also be classified as "significant" or "likely significant" (patients with variants of "uncertain significance " are excluded) or -Patients with Catenin Alpha 1 (CTNNA1) and partner and localizer of breast cancer 2 (BRCA2) (PALB2) germline mutations suspected to be, or reported to be, associated with hereditary diffuse gastric cancer (HDGC) syndrome. or * In the absence of a germline CDH1 mutation, patients must meet clinical criteria for genetic testing due to a history suggestive of Hereditary Diffuse Gastric Cancer (HDGC) syndrome * Age greater than or equal to 18 years. * Physiologically able to undergo upper endoscopy. * Ability to understand and the willingness to sign a written informed consent document. * Pregnant women are eligible during second trimester of pregnancy if clinically indicated for evaluation of cancer. EXCLUSION CRITERIA: * Current use of therapeutic anticoagulation medication * Known bleeding disorder or thrombocytopenia. * Unstable angina or recent (within 3 months) myocardial infarction * Any clinical contraindication to general anesthesia **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Bethesda **Country:** United States **Facility:** National Institutes of Health Clinical Center **State:** Maryland **Zip:** 20892 #### Overall Officials **Official 1:** **Affiliation:** National Cancer Institute (NCI) **Name:** Jeremy L Davis, M.D. **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Access Criteria:** Requests must be made by clinical investigators with interest and/or expertise in gastrointestinal cancers or endoscopic surveillance techniques. **Description:** We will share the study protocol, statistical analysis plan, and clinical study report. **Info Types:** - STUDY_PROTOCOL - SAP - CSR **IPD Sharing:** YES **Time Frame:** For 2 years from date of study completion. ### References Module #### References **Citation:** Schueler SA, Gamble LA, Curtin BF, Ruff SM, Connolly M, Hannah C, Quezado M, Miettinen M, George M, Blakely AM, Hernandez JM, Heller T, Koh C, Davis JL. Evaluation of confocal laser endomicroscopy for detection of occult gastric carcinoma in CDH1 variant carriers. J Gastrointest Oncol. 2021 Apr;12(2):216-225. doi: 10.21037/jgo-20-430. **PMID:** 34012620 **Citation:** Ruff S, Curtin B, Quezado M, Heller T, Koh C, Steinberg SM, Connolly M, Hernandez JM, Davis JL. Evaluation of confocal endoscopic microscopy for detection of early-stage gastric cancer in hereditary diffuse gastric cancer (HDGC) syndrome. J Gastrointest Oncol. 2019 Jun;10(3):407-411. doi: 10.21037/jgo.2019.01.04. **PMID:** 31183189 #### See Also Links **Label:** NIH Clinical Center Detailed Web Page **URL:** https://clinicalstudies.info.nih.gov/cgi/detail.cgi?B_2018-C-0141.html ## Document Section ### Large Document Module #### Large Docs - Date: 2019-06-05 - Filename: Prot_SAP_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: True - Label: Study Protocol and Statistical Analysis Plan - Size: 643239 - Type Abbrev: Prot_SAP - Upload Date: 2021-01-12T11:59 - Date: 2019-07-22 - Filename: ICF_001.pdf - Has ICF: True - Has Protocol: False - Has SAP: False - Label: Informed Consent Form - Size: 201265 - Type Abbrev: ICF - Upload Date: 2021-01-12T12:01 ## 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 ### 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: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M16064 - Name: Stomach Neoplasms - Relevance: HIGH - As Found: Gastric Cancer - ID: M16355 - Name: Syndrome - 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: M16062 - Name: Stomach Diseases - Relevance: LOW - As Found: Unknown - ID: T5486 - Name: Stomach Cancer - Relevance: HIGH - As Found: Gastric Cancer - ID: T1863 - Name: Diffuse Gastric Cancer - Relevance: HIGH - As Found: Diffuse Gastric Cancer - ID: T2744 - Name: Hereditary Diffuse Gastric Cancer - Relevance: HIGH - As Found: Hereditary Diffuse Gastric Cancer ### Condition Browse Module - Meshes - ID: D000013274 - Term: Stomach Neoplasms ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** 36/37 were evaluable for adverse events. One participant was determined to have Cadherin-1 (CDH1) variant of uncertain significance. #### Event Groups **Group ID:** EG000 **Title:** 1/Arm 1 - Upper White-light Endoscopy and Confocal Endoscopic Microscopy **Deaths Num At Risk:** 36 **Description:** Upper white-light endoscopy and confocal endoscopic microscopy Endoscope+Cellvizio(R) 100 microscope: Patients will undergo white-light, upper endoscopy. In addition, during this endoscopy patients will undergo Confocal Endoscopic Microscopy (CEM) using the Cellvizio probe (Mauna Kea Technologies) to scan the same anatomic zones. **ID:** EG000 **Other Num Affected:** 1 **Other Num at Risk:** 36 **Serious Number At Risk:** 36 **Title:** 1/Arm 1 - Upper White-light Endoscopy and Confocal Endoscopic Microscopy **Frequency Threshold:** 0 #### Other Events **Term:** Fever **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE (5.0) **Term:** Urinary Tract Infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** CTCAE (5.0) **Time Frame:** Date of enrollment to date off study, approximately 11 months and 19 days. ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 37 **Units:** Participants ### Group **ID:** BG000 **Title:** 1/Arm 1 - Upper White-light Endoscopy and Confocal Endoscopic Microscopy **Description:** Upper white-light endoscopy and confocal endoscopic microscopy Endoscope+Cellvizio(R) 100 microscope: Patients will undergo white-light, upper endoscopy. In addition, during this endoscopy patients will undergo Confocal Endoscopic Microscopy (CEM) using the Cellvizio probe (Mauna Kea Technologies) to scan the same anatomic zones. ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** <=18 years #### Measurement **Group ID:** BG000 **Value:** 30 **Category Title:** Between 18 and 65 years #### Measurement **Group ID:** BG000 **Value:** 7 **Category Title:** >=65 years **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 14.02 **Value:** 50.51 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 28 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 9 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 4 **Class Title:** Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 31 **Class Title:** Not Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 2 **Class Title:** Ethnicity Unknown or Not Reported #### Measurement **Group ID:** BG000 **Value:** 33 **Class Title:** White #### Measurement **Group ID:** BG000 **Value:** 2 **Class Title:** Race Other #### Measurement **Group ID:** BG000 **Value:** 2 **Class Title:** Race Unknown ### Measure #### Measurement **Group ID:** BG000 **Value:** 37 **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:** COUNT_OF_PARTICIPANTS **Title:** Race/Ethnicity, Customized **Unit of Measure:** Participants ### Measure 5 **Parameter Type:** NUMBER **Title:** Region of Enrollment **Unit of Measure:** participants ## Results Section - More Information Module ### Certain Agreement ### Point of Contact **Email:** [email protected] **Organization:** National Cancer Institute **Phone:** 240-760-6229 **Title:** Dr. Jeremy L. Davis ## 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:** 16.7 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 11.1 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 67 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 87 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Sensitivity for detection of SRC foci in CDH1 germline mutation carriers was assessed by confocal endoscopic microscopy (CEM) compared to the current method of and standard white light endoscopy. Sensitivity in CEM and WLE is defined as the percentage of participants with detectable cancer on endoscopic biopsy. **Parameter Type:** NUMBER **Population Description:** 36/37 were evaluable for this outcome measure. One participant was determined to have Cadherin-1 (CDH1) variant of uncertain significance. **Reporting Status:** POSTED **Time Frame:** 14 days **Title:** Percentage of Participants With Detectable Confocal Endoscopic Microscopy (CEM) w/Greater Sensitivity for Detection of Signet Ring Cells (SRC)Foci in Cadherin-1 (CDH1) Germline Mutation Carriers Compared to Current Method of Standard White Light Endoscopy **Type:** PRIMARY **Unit of Measure:** Percentage of participants ##### Group **Description:** Upper white-light endoscopy and confocal endoscopic microscopy Endoscope+Cellvizio(R) 100 microscope: Patients will undergo white-light, upper endoscopy. In addition, during this endoscopy patients will undergo Confocal Endoscopic Microscopy (CEM) using the Cellvizio probe (Mauna Kea Technologies) to scan the same anatomic zones. **ID:** OG000 **Title:** 1/Arm 1 - Upper White-light Endoscopy and Confocal Endoscopic Microscopy #### Outcome Measure 2 **Description:** In participants who choose to undergo prophylactic total gastrectomy with permanent pathologic analysis, the false negative rate (the fraction of participants who have SRC foci not identified by CEM and White Light Endoscopy techniques) will be determined and reported. The fraction percentage of patients who underwent prophylactic total gastrectomy with findings of SRC foci in the gastrectomy specimen will represent the denominator (number) and the number of patients with negative findings by CEM and White Light Endoscopy (WLE) will represent the numerator (number) to generate the false negative detection rate (fraction) for CEM and WLE, respectively. **Parameter Type:** NUMBER **Population Description:** 36/37 were evaluable for this outcome measure. One participant was determined to have Cadherin-1 (CDH1) variant of uncertain significance. **Reporting Status:** POSTED **Time Frame:** Date of enrollment to date of prophylactic gastrectomy, approximately 6 months or an average of 1 month up to 12 months. **Title:** Percentage of Participants Who Have Signet Ring Cells (SRC) Foci Not Identified by Confocal Endoscopic Microscopy (CEM) **Type:** SECONDARY **Unit of Measure:** Percentage of participants ##### Group **Description:** Upper white-light endoscopy and confocal endoscopic microscopy Endoscope+Cellvizio(R) 100 microscope: Patients will undergo white-light, upper endoscopy. In addition, during this endoscopy patients will undergo Confocal Endoscopic Microscopy (CEM) using the Cellvizio probe (Mauna Kea Technologies) to scan the same anatomic zones. **ID:** OG000 **Title:** 1/Arm 1 - Upper White-light Endoscopy and Confocal Endoscopic Microscopy #### Outcome Measure 3 **Description:** Here is the number of participants with serious and non-serious adverse events assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0). A non-serious adverse event is any untoward medical occurrence. A serious adverse event is an adverse event or suspected adverse reaction that results in death, a life-threatening adverse drug experience, hospitalization, disruption of the ability to conduct normal life functions, congenital anomaly/birth defect or important medical events that jeopardize the patient or subject and may require medical or surgical intervention to prevent one of the previous outcomes mentioned. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** 36/37 were evaluable for this outcome measure. One participant was determined to have Cadherin-1 (CDH1) variant of uncertain significance. **Reporting Status:** POSTED **Time Frame:** Date of enrollment to date off study, approximately 11 months and 19 days. **Title:** Number of Participants With Serious and Non-serious Adverse Events Assessed by the Common Terminology Criteria for Adverse Events (CTCAE v5.0) **Type:** OTHER_PRE_SPECIFIED **Unit of Measure:** Participants ##### Group **Description:** Upper white-light endoscopy and confocal endoscopic microscopy Endoscope+Cellvizio(R) 100 microscope: Patients will undergo white-light, upper endoscopy. In addition, during this endoscopy patients will undergo Confocal Endoscopic Microscopy (CEM) using the Cellvizio probe (Mauna Kea Technologies) to scan the same anatomic zones. **ID:** OG000 **Title:** 1/Arm 1 - Upper White-light Endoscopy and Confocal Endoscopic Microscopy ### Participant Flow Module #### Group **Description:** Upper white-light endoscopy and confocal endoscopic microscopy Endoscope+Cellvizio(R) 100 microscope: Patients will undergo white-light, upper endoscopy. In addition, during this endoscopy patients will undergo Confocal Endoscopic Microscopy (CEM) using the Cellvizio probe (Mauna Kea Technologies) to scan the same anatomic zones. **ID:** FG000 **Title:** 1/Arm 1 - Upper White-light Endoscopy and Confocal Endoscopic Microscopy #### Period **Title:** Overall Study ##### Withdraw **Type:** Subject determined to have Cadherin-1 (CDH1) variant of uncertain significance. ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 37 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 36 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 1 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT00281879 **Brief Title:** Donor Stem Cell Transplant or Donor White Blood Cell Infusions in Treating Patients With Hematologic Cancer **Official Title:** Transplantation of Unrelated Donor Hematopoietic Stem Cells for the Treatment of Hematological Malignancies #### Organization Study ID Info **ID:** CDR0000452794 #### Organization **Class:** OTHER **Full Name:** OHSU Knight Cancer Institute #### Secondary ID Infos **ID:** P30CA016058 **Link:** https://reporter.nih.gov/quickSearch/P30CA016058 **Type:** NIH **ID:** OHSU-TPI-9695-L **ID:** OHSU-540 ### Status Module #### Completion Date **Date:** 2008-03 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-09-27 **Type:** ACTUAL **Last Update Submit Date:** 2017-09-25 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2008-02 **Type:** ACTUAL #### Results First Post Date **Date:** 2012-06-19 **Type:** ESTIMATED **Results First Submit Date:** 2012-05-17 **Results First Submit QC Date:** 2012-05-17 #### Start Date **Date:** 2006-02 **Status Verified Date:** 2017-09 #### Study First Post Date **Date:** 2006-01-25 **Type:** ESTIMATED **Study First Submit Date:** 2006-01-24 **Study First Submit QC Date:** 2006-01-24 ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** National Cancer Institute (NCI) #### Lead Sponsor **Class:** OTHER **Name:** OHSU Knight Cancer Institute #### Responsible Party **Investigator Affiliation:** OHSU Knight Cancer Institute **Investigator Full Name:** Richard Maziarz **Investigator Title:** Principal Investigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** RATIONALE: A peripheral stem cell transplant or an umbilical cord blood transplant from a donor may be able to replace blood-forming cells that were destroyed by chemotherapy or radiation therapy. Giving an infusion of the donor's white blood cells (donor lymphocyte infusion) after the transplant may help destroy any remaining cancer cells (graft-versus-tumor effect). Sometimes the transplanted cells can make an immune response against the body's normal cells. Methotrexate, cyclosporine, tacrolimus, or methylprednisolone may stop this from happening. PURPOSE: This clinical trial is studying how well a donor stem cell transplant or donor white blood cell infusions work in treating patients with hematologic cancer. **Detailed Description:** OBJECTIVES: Primary * Determine the effectiveness of unrelated donor allogeneic hematopoietic stem cells for transplantation after conditioning for the treatment of patients with high-risk hematologic malignancies. * Compare survival, disease-free survival (DFS), response rate, and toxicity rates in these patients with historical controls. * Compare the rate and severity of acute and chronic GVHD after allogeneic hematopoietic stem cell transplantation in patients with hematopoietic malignancies with historical controls transplanted with stem cells from related sibling donors * Assess engraftment, long-term hematopoietic recovery, relapse rate, and disease-free survival when allogeneic hematopoietic stem cells are used as a source of stem cells for transplantation in patients with high-risk hematological malignancies. * Assess engraftment, long-term hematopoietic recovery, and overall survival when allogeneic hematopoietic stem cells are used as a source of stem cells for transplantation in patients who have graft failure or graft rejection. * Compare engraftment, long-term hematopoietic recovery, rate of GVHD, rate of relapse, toxicity rates, overall disease-free survival and overall survival when donor leukocyte infusions (DLI) are given for patients who have disease recurrence, progression, or low donor chimerisms after unrelated stem cell transplantation or before DLI with historical controls of other donor leukocyte infusions. Secondary * Determine the quality of life of patients undergoing hematopoietic stem cell transplantation or donor leukocyte infusions from unrelated HLA genotypically-identical donors. OUTLINE: Patients are assigned to 1 of 8 treatment groups. * Group 1\*: Patients undergo total body irradiation (TBI) twice a day on days -7 to -4. Patients then receive cyclophosphamide IV over 1 hour on days -3 and -2. On day 0 patients undergo stem cell transplantation (SCT). Beginning on day 7, patients receive filgrastim (G-CSF) IV once daily until blood counts recover. * Group 2 (patients who have previously experienced dose-limiting radiotherapy): Patients receive oral busulfan 4 times daily on days -7 to -4 and cyclophosphamide IV over 1 hour on days -3 and -2. On day 0 patients undergo SCT. Beginning on day 7, patients receive G-CSF IV once daily until blood counts recover. * Group 3 (pediatric patients only): Patients receive busulfan IV 4 times daily on days -9 to -6 and cyclophosphamide IV over 1 hour and fludarabine IV over 30 minutes on days -5 to -2. On day 0 patients undergo SCT. Beginning on day 7, patients may receive G-CSF IV once daily until blood counts recover. * Group 4 (second SCT for patients who have experienced graft rejection or failure)\*: Patients receive low-dose fludarabine IV over 30 minutes on days -4 to -2. Patients then undergo low-dose TBI once followed by SCT on day 0. Beginning on day 7, patients may receive G-CSF IV once daily until blood counts recover. * Group 5 (patients who developed grade 3 cystitis after prior cyclophosphamide-containing therapy): Patients receive carmustine IV over 2 hours on day -6, etoposide IV over 2 hours and cytarabine IV over 30 minutes on days -5 to -2, and melphalan IV over 30 minutes on day -1 (BEAM). On day 0, patients undergo SCT. Beginning on day 7, patients receive G-CSF IV once daily until blood counts recover. * Group 6 (cord blood transplantation): Patients receive anti-thymocyte globulin IV once daily and methylprednisolone IV twice daily on days -3 to -1. On day 0, patients undergo an umbilical cord blood SCT. * Group 7 (patients with relapsing or progressive disease after prior transplants or low donor chimerisms)\*: Patients must not have existing graft-versus-host disease (GVHD). Patients receive donor lymphocyte infusions with conditioning chemotherapy and/or radiotherapy at the discretion of the investigator. * Group 8 (pediatric patients only)\*: Patients undergo TBI twice a day on days -7 to -5. Patients also receive etoposide IV over 24 hours on day -4 and cyclophosphamide IV over 1 hour on days -3 and -2. On day 0, patients undergo SCT. Beginning on day 7, patients may receive G-CSF IV once daily until blood counts recover. NOTE: \*Patients who have received \> 3000 cGy to the central nervous system or \> 2000 cGy to the lung or liver may not receive any regimen containing total body irradiation (TBI) All patients receive GVHD prophylaxis comprising methotrexate IV on days 1, 3, 6, and 11; cyclosporine and/or tacrolimus on days -2 to 100; and/or methylprednisolone IV on days 7 to 100. Patients with an unrelated donor who experience a relapse prior to transplantation, may proceed directly to transplantation. However, if immediate transplantation from the unrelated donor is not possible, the patient must be re-induced into a complete hematological remission. Patients who experience graft failure or graft rejection after allogeneic transplantation are eligible for a second stem cell infusion from the original donor. Quality of life is assessed at baseline and at 7 days, 3 months, and 1 year after transplantation. After completion of study treatment, patients are followed periodically for survival. PROJECTED ACCRUAL: A total of 43 patients will be accrued for this study. ### Conditions Module **Conditions:** - Chronic Myeloproliferative Disorders - Leukemia - Lymphoma - Multiple Myeloma and Plasma Cell Neoplasm - Myelodysplastic Syndromes - Myelodysplastic/Myeloproliferative Neoplasms - Unusual Cancers of Childhood **Keywords:** - adult acute lymphoblastic leukemia in remission - L1 adult acute lymphoblastic leukemia - L2 adult acute lymphoblastic leukemia - L3 adult acute lymphoblastic leukemia - recurrent adult acute lymphoblastic leukemia - accelerated phase chronic myelogenous leukemia - blastic phase chronic myelogenous leukemia - chronic phase chronic myelogenous leukemia - relapsing chronic myelogenous leukemia - childhood acute lymphoblastic leukemia in remission - L1 childhood acute lymphoblastic leukemia - L2 childhood acute lymphoblastic leukemia - L3 childhood acute lymphoblastic leukemia - recurrent childhood acute lymphoblastic leukemia - recurrent adult Hodgkin lymphoma - recurrent/refractory childhood Hodgkin lymphoma - stage IV adult Hodgkin lymphoma - stage IV childhood Hodgkin lymphoma - adult acute myeloid leukemia in remission - adult acute myeloid leukemia with 11q23 (MLL) abnormalities - adult acute myeloid leukemia with t(15;17)(q22;q12) - adult acute myeloid leukemia with t(16;16)(p13;q22) - childhood acute myeloid leukemia in remission - recurrent adult acute myeloid leukemia - recurrent childhood acute myeloid leukemia - myelodysplastic/myeloproliferative neoplasm, unclassifiable - previously treated myelodysplastic syndromes - secondary myelodysplastic syndromes - secondary acute myeloid leukemia - refractory anemia with excess blasts in transformation - refractory anemia with excess blasts - refractory anemia - chronic myelomonocytic leukemia - childhood chronic myelogenous leukemia - chronic eosinophilic leukemia - primary myelofibrosis - chronic neutrophilic leukemia - de novo myelodysplastic syndromes - atypical chronic myeloid leukemia, BCR-ABL negative - extranodal marginal zone B-cell lymphoma of mucosa-associated lymphoid tissue - juvenile myelomonocytic leukemia - nodal marginal zone B-cell lymphoma - noncontiguous stage II adult Burkitt lymphoma - noncontiguous stage II adult diffuse large cell lymphoma - noncontiguous stage II adult diffuse mixed cell lymphoma - noncontiguous stage II adult diffuse small cleaved cell lymphoma - noncontiguous stage II adult immunoblastic large cell lymphoma - noncontiguous stage II adult lymphoblastic lymphoma - noncontiguous stage II grade 1 follicular lymphoma - noncontiguous stage II grade 2 follicular lymphoma - noncontiguous stage II grade 3 follicular lymphoma - noncontiguous stage II mantle cell lymphoma - noncontiguous stage II marginal zone lymphoma - noncontiguous stage II small lymphocytic lymphoma - recurrent adult Burkitt lymphoma - recurrent adult diffuse large cell lymphoma - recurrent adult diffuse mixed cell lymphoma - recurrent adult diffuse small cleaved cell lymphoma - recurrent adult immunoblastic large cell lymphoma - recurrent adult lymphoblastic lymphoma - recurrent childhood large cell lymphoma - recurrent childhood lymphoblastic lymphoma - recurrent childhood small noncleaved cell lymphoma - recurrent grade 1 follicular lymphoma - recurrent grade 2 follicular lymphoma - recurrent grade 3 follicular lymphoma - recurrent mantle cell lymphoma - recurrent marginal zone lymphoma - recurrent small lymphocytic lymphoma - refractory chronic lymphocytic leukemia - refractory hairy cell leukemia - refractory multiple myeloma - splenic marginal zone lymphoma - stage I multiple myeloma - stage II multiple myeloma - stage III adult Burkitt lymphoma - stage III adult Hodgkin lymphoma - stage III adult diffuse large cell lymphoma - stage III adult diffuse mixed cell lymphoma - stage III adult diffuse small cleaved cell lymphoma - stage III adult immunoblastic large cell lymphoma - stage III adult lymphoblastic lymphoma - stage III chronic lymphocytic leukemia - stage III grade 1 follicular lymphoma - stage III grade 2 follicular lymphoma - stage III grade 3 follicular lymphoma - stage III mantle cell lymphoma - stage III marginal zone lymphoma - stage III multiple myeloma - stage III small lymphocytic lymphoma - stage IV adult Burkitt lymphoma - stage IV adult diffuse large cell lymphoma - stage IV adult diffuse mixed cell lymphoma - stage IV adult diffuse small cleaved cell lymphoma - stage IV adult immunoblastic large cell lymphoma - stage IV adult lymphoblastic lymphoma - stage IV chronic lymphocytic leukemia - stage IV grade 1 follicular lymphoma - stage IV grade 2 follicular lymphoma - stage IV grade 3 follicular lymphoma - stage IV mantle cell lymphoma - stage IV marginal zone lymphoma - stage IV small lymphocytic lymphoma - stage III childhood Hodgkin lymphoma - stage III childhood large cell lymphoma - stage III childhood lymphoblastic lymphoma - stage III childhood small noncleaved cell lymphoma - stage IV childhood large cell lymphoma - stage IV childhood lymphoblastic lymphoma - stage IV childhood small noncleaved cell lymphoma - unusual cancers of childhood - childhood myelodysplastic syndromes ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 200 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Biological: filgrastim - Drug: cyclophosphamide - Drug: cyclosporine - Drug: methotrexate - Drug: mycophenolate mofetil - Drug: tacrolimus - Procedure: peripheral blood stem cell transplantation - Radiation: radiation therapy **Label:** Cyclophosphamide (Cytoxan) and Total Body Irradiation (TBI) **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Intervention Names:** - Biological: filgrastim - Drug: busulfan - Drug: cyclophosphamide - Drug: cyclosporine - Drug: methotrexate - Drug: mycophenolate mofetil - Drug: tacrolimus - Procedure: peripheral blood stem cell transplantation **Label:** Busulfan and Cyclophosphamide (Cytoxan) **Type:** ACTIVE_COMPARATOR #### Arm Group 3 **Description:** On the day of your admission, you will start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Chemotherapy will begin on Day -6 with carmustine (BCNU), followed by etoposide (VP-16), cytosine arabinoside (ARA-C), and melphalan. This conditioning regimen is known as the BEAM regimen. The dose of this therapy is high enough to kill cancer cells but will also kill all of your normal blood forming cells. Subjects undergoing the BEAM regimen will not have total body irradiation (TBI). **Intervention Names:** - Biological: filgrastim - Drug: carmustine - Drug: cyclosporine - Drug: cytarabine - Drug: etoposide - Drug: melphalan - Drug: methotrexate - Drug: mycophenolate mofetil - Drug: tacrolimus - Procedure: peripheral blood stem cell transplantation **Label:** BEAM Regimen **Type:** ACTIVE_COMPARATOR #### Arm Group 4 **Description:** A conditioning regimen of low-dose fludarabine and TBI is used in the event that a second donation of hematopoietic stem cells is necessary. Chemotherapy with Fludarabine will begin 4 days prior to your transplant. This drug will be given through the catheter in your chest daily for 3 days. TBI (radiation) will be given to you on the day of your transplant. After your TBI, your donor's stem cells / bone marrow will be given to you through your catheter. The drugs cyclosporine and mycophenolate mofetil (MMF) will be given orally to help you accept your donor's cells. **Intervention Names:** - Biological: filgrastim - Drug: cyclosporine - Drug: fludarabine phosphate - Drug: methotrexate - Drug: mycophenolate mofetil - Drug: tacrolimus - Procedure: peripheral blood stem cell transplantation - Radiation: radiation therapy **Label:** Low-Dose Fludarabine and TBI(for second stem cell donation) **Type:** ACTIVE_COMPARATOR #### Arm Group 5 **Description:** On the day of your admission you will start to take a drug called Dilantin which is used to help prevent seizures while you receive your chemotherapy drugs. You will also start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. On the next day, you will then begin your conditioning therapy with a drug called busulfan. This medicine will be given to you by an infusion into your bloodstream through a small tube in the vein of your arm four times per day for four days. After the busulfan treatment, you will receive 4 doses each of two drugs, cyclophosphamide (also known as Cytoxan) and fludarabine, over 2 hours into your vein. **Intervention Names:** - Biological: filgrastim - Drug: busulfan - Drug: cyclophosphamide - Drug: cyclosporine - Drug: fludarabine phosphate - Drug: methotrexate - Drug: mycophenolate mofetil - Drug: tacrolimus - Procedure: peripheral blood stem cell transplantation **Label:** Busulfan, Cyclophosphamide, and Fludarabine (Pediatric only) **Type:** ACTIVE_COMPARATOR #### Arm Group 6 **Description:** If you are undergoing a pre-transplant conditioning regimen prior to undergoing a cord blood transplant, you will receive a drug called ATG to improve your chances of engraftment and decrease your risk of graft versus host disease. You may receive ATG 3 times during your transplant regimen on days -3 through days -1 in addition to your pre-transplant conditioning therapy. Methylprednisolone will also be given during each dose of ATG to help reduce any reactions during infusion. **Intervention Names:** - Biological: anti-thymocyte globulin - Biological: filgrastim - Drug: cyclosporine - Drug: methotrexate - Drug: methylprednisolone - Drug: mycophenolate mofetil - Drug: tacrolimus - Procedure: peripheral blood stem cell transplantation - Procedure: umbilical cord blood transplantation **Label:** ATG For Cord Blood Transplants **Type:** ACTIVE_COMPARATOR #### Arm Group 7 **Description:** Donor Leukocyte Infusions: You will receive DLI from your original transplant donor. This will be given through a vein , usually in your arm. It will be similar to getting a platelet or blood transfusion. You may require more than one DLI. The decision to give you another infusion will be determined by your condition, relapse status, GVHD and how much DLI you were given before. You may need chemotherapy and/or radiation to improve your disease status prior to additional DLI's. **Intervention Names:** - Biological: filgrastim - Drug: cyclosporine - Drug: methotrexate - Drug: mycophenolate mofetil - Drug: tacrolimus - Procedure: peripheral blood stem cell transplantation **Label:** DLI (Donor Leukocyte Infusion) **Type:** ACTIVE_COMPARATOR #### Arm Group 8 **Description:** On the day after your admission, you will start receiving radiation therapy (TBI). Radiation will be given to you 2 times a day for 3 days. On the next day, you will then begin your chemotherapy with a drug called etoposide. This medicine will be given to you by an infusion into your bloodstream through a small tube in the vein of your arm for one day. After the etoposide treatment, on the next day you will receive cyclophosphamide (also known as Cytoxan) for 2 days. When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. After you have completed the cyclophosphamide you will rest one day without any anti-cancer therapy. This allows your body time to remove and inactivate the chemotherapy. After a day of rest, you will be given your donor's cells. **Intervention Names:** - Biological: filgrastim - Drug: cyclophosphamide - Drug: cyclosporine - Drug: etoposide - Drug: methotrexate - Drug: mycophenolate mofetil - Drug: tacrolimus - Procedure: peripheral blood stem cell transplantation - Radiation: radiation therapy **Label:** Cyclophosphamide, Etoposide (VP16) and TBI (Pediatric only) **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - ATG For Cord Blood Transplants **Description:** Intravenous, 1.5 mg/kg of body weight daily for 7 to 14 days The first dose should be administered over a minimum of 6 hours and over at least 4 hours on subsequent doses through a high-flow vein. **Name:** anti-thymocyte globulin **Other Names:** - ATG **Type:** BIOLOGICAL #### Intervention 2 **Arm Group Labels:** - ATG For Cord Blood Transplants - BEAM Regimen - Busulfan and Cyclophosphamide (Cytoxan) - Busulfan, Cyclophosphamide, and Fludarabine (Pediatric only) - Cyclophosphamide (Cytoxan) and Total Body Irradiation (TBI) - Cyclophosphamide, Etoposide (VP16) and TBI (Pediatric only) - DLI (Donor Leukocyte Infusion) - Low-Dose Fludarabine and TBI(for second stem cell donation) **Description:** Will be given IV at 5 µg/kg/day. The first injection will be administered on day +7, i.e. 7 days after the hematopoietic stem cells are infused. Will be administered until the ANC is 1500 / µl for 2 days. Dose and schedule of G-CSF administration is left to each center's discretion. **Name:** filgrastim **Other Names:** - G-CSF **Type:** BIOLOGICAL #### Intervention 3 **Arm Group Labels:** - Busulfan and Cyclophosphamide (Cytoxan) - Busulfan, Cyclophosphamide, and Fludarabine (Pediatric only) **Description:** Patients who take the drug PO, busulfan will be administered at 1 mg/kg/ dose given by mouth every 6 hours for 16 consecutive doses. Pediatric patients who receive busulfan IV continuous infusion will receive a dose of 3.0 mg/kg/IBW if under the age of 2.Pediatric patients over the age of 2 will receive busulfan at a dose of 0.8 mg/kg/dose. **Name:** busulfan **Type:** DRUG #### Intervention 4 **Arm Group Labels:** - BEAM Regimen **Description:** 300mg/m2 IV dissolved in 500 cc NS infused over 2 hours into right atrial catheter on day -6. **Name:** carmustine **Type:** DRUG #### Intervention 5 **Arm Group Labels:** - Busulfan and Cyclophosphamide (Cytoxan) - Busulfan, Cyclophosphamide, and Fludarabine (Pediatric only) - Cyclophosphamide (Cytoxan) and Total Body Irradiation (TBI) - Cyclophosphamide, Etoposide (VP16) and TBI (Pediatric only) **Description:** For transplantation, the drug is diluted in 250 to 500 cc of NS or D5W and administered IV over 2 hours. **Name:** cyclophosphamide **Type:** DRUG #### Intervention 6 **Arm Group Labels:** - ATG For Cord Blood Transplants - BEAM Regimen - Busulfan and Cyclophosphamide (Cytoxan) - Busulfan, Cyclophosphamide, and Fludarabine (Pediatric only) - Cyclophosphamide (Cytoxan) and Total Body Irradiation (TBI) - Cyclophosphamide, Etoposide (VP16) and TBI (Pediatric only) - DLI (Donor Leukocyte Infusion) - Low-Dose Fludarabine and TBI(for second stem cell donation) **Description:** Initial doses will be administered IV at a starting dose of 1.5 mg/kg BID. The infusion will vary from 2-24hr depending on the incidence of side-effects. **Name:** cyclosporine **Type:** DRUG #### Intervention 7 **Arm Group Labels:** - BEAM Regimen **Description:** 400 mg/m2 dissolved in 200cc D5W and infused over 30 minutes into right atrial catheter on days -5, -4, -3, -2. **Name:** cytarabine **Type:** DRUG #### Intervention 8 **Arm Group Labels:** - BEAM Regimen - Cyclophosphamide, Etoposide (VP16) and TBI (Pediatric only) **Description:** Etoposide administration 200 mg /m2 dissolved in 1 liter NS and infused over 2 hours into right atrial catheter. Infusion to begin after cytarabine administration on days -5, -4, -3, -2. Etoposide administration 50 mg/kg IV over 24 hours, divided into 3 doses. Dilute in normal saline at a concentration of 0.4 mg/ml (Observe for precipitation). Administered IV with continuous infusion over 24 hours. Diuretics may be given for fluid overload. **Name:** etoposide **Type:** DRUG #### Intervention 9 **Arm Group Labels:** - Busulfan, Cyclophosphamide, and Fludarabine (Pediatric only) - Low-Dose Fludarabine and TBI(for second stem cell donation) **Description:** Fludarabine administered at 30 mg/m2 IVPB infused over 30 minutes into right atrial catheter on days -4, -3, -2. Fludarabine administered at 40 mg/m2 IVPB infused over 30 into the right atrial catheter on days -5, -4, -3, and -2. **Name:** fludarabine phosphate **Type:** DRUG #### Intervention 10 **Arm Group Labels:** - BEAM Regimen **Description:** 140 mg /m2 in concentration of 0.45 mg/ml of NS infused over 30 minutes into right atrial catheter on day -1. **Name:** melphalan **Type:** DRUG #### Intervention 11 **Arm Group Labels:** - ATG For Cord Blood Transplants - BEAM Regimen - Busulfan and Cyclophosphamide (Cytoxan) - Busulfan, Cyclophosphamide, and Fludarabine (Pediatric only) - Cyclophosphamide (Cytoxan) and Total Body Irradiation (TBI) - Cyclophosphamide, Etoposide (VP16) and TBI (Pediatric only) - DLI (Donor Leukocyte Infusion) - Low-Dose Fludarabine and TBI(for second stem cell donation) **Description:** Administered on days +1, +3, and +7. **Name:** methotrexate **Type:** DRUG #### Intervention 12 **Arm Group Labels:** - ATG For Cord Blood Transplants **Description:** Methyl-prednisolone is administered IV as a rapid infusion. **Name:** methylprednisolone **Type:** DRUG #### Intervention 13 **Arm Group Labels:** - ATG For Cord Blood Transplants - BEAM Regimen - Busulfan and Cyclophosphamide (Cytoxan) - Busulfan, Cyclophosphamide, and Fludarabine (Pediatric only) - Cyclophosphamide (Cytoxan) and Total Body Irradiation (TBI) - Cyclophosphamide, Etoposide (VP16) and TBI (Pediatric only) - DLI (Donor Leukocyte Infusion) - Low-Dose Fludarabine and TBI(for second stem cell donation) **Description:** Mycophenolate may be used as a substitute for Methotrexate **Name:** mycophenolate mofetil **Type:** DRUG #### Intervention 14 **Arm Group Labels:** - ATG For Cord Blood Transplants - BEAM Regimen - Busulfan and Cyclophosphamide (Cytoxan) - Busulfan, Cyclophosphamide, and Fludarabine (Pediatric only) - Cyclophosphamide (Cytoxan) and Total Body Irradiation (TBI) - Cyclophosphamide, Etoposide (VP16) and TBI (Pediatric only) - DLI (Donor Leukocyte Infusion) - Low-Dose Fludarabine and TBI(for second stem cell donation) **Description:** A drug used to decrease the risk of graft versus host disease (GvHD). **Name:** tacrolimus **Other Names:** - FK-506 **Type:** DRUG #### Intervention 15 **Arm Group Labels:** - ATG For Cord Blood Transplants - BEAM Regimen - Busulfan and Cyclophosphamide (Cytoxan) - Busulfan, Cyclophosphamide, and Fludarabine (Pediatric only) - Cyclophosphamide (Cytoxan) and Total Body Irradiation (TBI) - Cyclophosphamide, Etoposide (VP16) and TBI (Pediatric only) - DLI (Donor Leukocyte Infusion) - Low-Dose Fludarabine and TBI(for second stem cell donation) **Description:** The stem cells will be given to you by intravenous injection (through your vein) using a catheter that was placed prior to beginning chemotherapy. The stem cell infusion takes 1-6 hours. **Name:** peripheral blood stem cell transplantation **Type:** PROCEDURE #### Intervention 16 **Arm Group Labels:** - ATG For Cord Blood Transplants **Description:** The patient will receive ATG to improve the changes of engraftment and decrease their risk of graft versus host disease. The patient may receive ATG 3 times during their transplant regimen on days -3 through days -1 **Name:** umbilical cord blood transplantation **Type:** PROCEDURE #### Intervention 17 **Arm Group Labels:** - Cyclophosphamide (Cytoxan) and Total Body Irradiation (TBI) - Cyclophosphamide, Etoposide (VP16) and TBI (Pediatric only) - Low-Dose Fludarabine and TBI(for second stem cell donation) **Description:** Radiation will be given to you 2 times a day for 3 or 4 days. **Name:** radiation therapy **Type:** RADIATION ### Outcomes Module #### Primary Outcomes **Description:** Determine the effectiveness of unrelated donor allogeneic hematopoietic stem cells for transplantation after conditioning for the treatment of high-risk hematopoietic malignancies. Disease-free survival: The length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. **Measure:** Number of Participants With Disease Free Survival (DFS). **Time Frame:** Duration of the study; Up to 2 years ### Eligibility Module **Eligibility Criteria:** DISEASE CHARACTERISTICS: * Diagnosis of 1 of the following\*: * Acute lymphoblastic leukemia in any disease phase * Patients with any of the following high-risk features are encouraged to enroll: * Philadelphia chromosome positive disease * L3 morphology, especially in the presence of t(8;14), t(8;22), or t(2;8) * Patients not in remission at day 28 of first induction * High LDH (i.e., ≥ 300 IU/mL at presentation) * Pre-B-cell, mixed lineage, or Burkitt's markers * Relapsed in the marrow while receiving continuous chemotherapy * Within 6 months after stopping chemotherapy * Relapse in one organ or extramedullary relapses in more than one organ while still receiving chemotherapy * Hodgkin's or non-Hodgkin's lymphoma beyond first complete remission (CR) or in first CR with features of high-risk disease, including, but not limited to: * Lymphoma not in CR after 3 courses of primary therapy * Patients with bulky disease at presentation, especially bulky mediastinal disease * Patients with LDH ≥ 300 IU/mL at presentation * Patients with extranodal disease * Patients with first remission within less than 1 year * Stage IV disease at presentation, especially with marrow involvement * Patients with high-intermediate or high International Index Scores * Acute myeloid leukemia (AML) meeting the following criteria: * Beyond first remission or high-risk disease in first CR * Required multiple courses of induction therapy to achieve a remission * Had residual leukemia on day 14-28 bone marrow examination after initial induction * Patients with any cytogenetic abnormality except inv 16 or t(8;21) * Chronic myelogenous leukemia in the chronic or early accelerated phase of the disease * Patients with blast crisis that can be induced back into chronic phase may be transplanted in second chronic phase * Myelodysplastic syndromes (MDS) meeting the following requirements: * Transfusion-dependent refractory anemia (RA), RA with excess blasts (RAEB), RAEB in transformation, or chronic myelomonocytic leukemia (CMML) * Patients with MDS that present with or evolve to AML must be re-induced back to remission prior to initiating a search for an unrelated donor NOTE: \*Patients with other hematologic malignancies not listed above, including diseases such as chronic lymphocytic leukemia (CLL), multiple myeloma, or rare pediatric malignancies, or patients who are felt to be at high-risk for relapse but who do not have features listed, may be allowed at the discretion of the investigator. * Must have failed prior stem cell transplantation * Must have a suitable unrelated allogeneic hematopoietic stem cell donor * A 5/6 match degree is acceptable for unrelated bone marrow donors * A 4/6 match degree is acceptable for unrelated cord blood units PATIENT CHARACTERISTICS: * SWOG performance status (PS) 0-2 OR * Karnofsky PS 50-100% OR * Lansky PS 50-100% * Creatinine clearance ≥ 45 mL/min * Creatinine ≤ 2.5 mg/dL * Bilirubin ≤ 2 mg/dL (abnormally high liver function tests allowed if the only source for the elevation is due to lymphoma of the liver) * AST or ALT ≤ 2 times normal (abnormally high liver function tests allowed if the only source for the elevation is due to lymphoma of the liver) * No patients at high risk of veno-occlusive disease * Not pregnant or nursing * Negative serum pregnancy test * Fertile patients must use an effective contraceptive method * DLCO ≥ 50% of predicted * FEV_1/FVC ≥ 65% of predicted * No current congestive heart failure (CHF) and/or LVEF ≥ 45% * No myocardial infarction within the past 6 months * No unstable angina within the past 6 months * HIV negative * Life expectancy must not be limited by disease other than malignancy * No allergy to any chemotherapeutic agent included in the regimen PRIOR CONCURRENT THERAPY: * See Disease Characteristics **Maximum Age:** 60 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Portland **Country:** United States **Facility:** OHSU Knight Cancer Institute **State:** Oregon **Zip:** 97239-3098 #### Overall Officials **Official 1:** **Affiliation:** OHSU Knight Cancer Institute **Name:** Richard Maziarz, MD **Role:** STUDY_CHAIR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000004194 - Term: Disease - ID: D000010335 - Term: Pathologic Processes - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000008232 - Term: Lymphoproliferative Disorders - ID: D000008206 - Term: Lymphatic Diseases - ID: D000007160 - Term: Immunoproliferative Disorders - ID: D000007154 - Term: Immune System Diseases - ID: D000006402 - Term: Hematologic Diseases - 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: D000006474 - Term: Hemorrhagic Disorders - ID: D000001855 - Term: Bone Marrow Diseases - ID: D000011230 - Term: Precancerous Conditions ### 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: BC23 - Name: Symptoms and General Pathology - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth - Abbrev: BC01 - Name: Infections - 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: Plasma Cell Neoplasm - ID: M13844 - Name: Plasmacytoma - Relevance: HIGH - As Found: Plasma Cell Neoplasm - ID: M10945 - Name: Leukemia - Relevance: HIGH - As Found: Leukemia - ID: M10955 - Name: Leukemia, Myeloid - Relevance: LOW - As Found: Unknown - ID: M18127 - Name: Leukemia, Myeloid, Acute - Relevance: LOW - As Found: Unknown - ID: M10951 - Name: Leukemia, Lymphoid - Relevance: LOW - As Found: Unknown - ID: M18116 - Name: Leukemia, Lymphocytic, Chronic, B-Cell - Relevance: LOW - As Found: Unknown - ID: M18123 - Name: Leukemia, Myelogenous, Chronic, BCR-ABL Positive - Relevance: LOW - As Found: Unknown - ID: M16355 - Name: Syndrome - Relevance: HIGH - As Found: Syndrome - ID: M27585 - Name: Precursor Cell Lymphoblastic Leukemia-Lymphoma - 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: M11222 - Name: Lymphoma, Non-Hodgkin - Relevance: LOW - As Found: Unknown - ID: M4070 - Name: Anemia - Relevance: LOW - As Found: Unknown - ID: M11221 - Name: Lymphoma, Follicular - Relevance: LOW - As Found: Unknown - ID: M12 - Name: Congenital Abnormalities - Relevance: LOW - As Found: Unknown - ID: M18831 - Name: Lymphoma, Large B-Cell, Diffuse - Relevance: LOW - As Found: Unknown - ID: M20554 - Name: Lymphoma, B-Cell, Marginal Zone - Relevance: LOW - As Found: Unknown - ID: M12307 - Name: Neoplasm Metastasis - Relevance: LOW - As Found: Unknown - ID: M18125 - Name: Leukemia, Myeloid, Chronic-Phase - Relevance: LOW - As Found: Unknown - ID: M21314 - Name: Hematologic Neoplasms - Relevance: LOW - As Found: Unknown - ID: M9751 - Name: Hodgkin Disease - Relevance: LOW - As Found: Unknown - ID: M12149 - Name: Myeloproliferative Disorders - Relevance: HIGH - As Found: Myeloproliferative Disorders - ID: M14850 - Name: Recurrence - Relevance: LOW - As Found: Unknown - ID: M14164 - Name: Preleukemia - Relevance: HIGH - As Found: Myelodysplastic Syndrome - ID: M12145 - Name: Myelodysplastic Syndromes - Relevance: HIGH - As Found: Myelodysplastic Syndrome - ID: M18828 - Name: Lymphoma, B-Cell - Relevance: LOW - As Found: Unknown - ID: M28312 - Name: Primary Myelofibrosis - Relevance: LOW - As Found: Unknown - ID: M5321 - Name: Burkitt Lymphoma - Relevance: LOW - As Found: Unknown - ID: M4084 - Name: Anemia, Refractory, with Excess of Blasts - Relevance: LOW - As Found: Unknown - ID: M18132 - Name: Leukemia, Myelomonocytic, Chronic - Relevance: LOW - As Found: Unknown - ID: M27706 - Name: Leukemia, Myelomonocytic, Juvenile - Relevance: LOW - As Found: Unknown - ID: M10950 - Name: Leukemia, Hairy Cell - Relevance: LOW - As Found: Unknown - ID: M4083 - Name: Anemia, Refractory - Relevance: LOW - As Found: Unknown - ID: M18124 - Name: Leukemia, Myeloid, Accelerated Phase - Relevance: LOW - As Found: Unknown - ID: M381 - Name: Plasmablastic Lymphoma - Relevance: LOW - As Found: Unknown - ID: M18830 - Name: Lymphoma, Large-Cell, Immunoblastic - Relevance: LOW - As Found: Unknown - ID: M5033 - Name: Blast Crisis - Relevance: LOW - As Found: Unknown - ID: M18126 - Name: Leukemia, Neutrophilic, Chronic - Relevance: LOW - As Found: Unknown - ID: M27707 - Name: Myelodysplastic-Myeloproliferative Diseases - Relevance: HIGH - As Found: Myelodysplastic/Myeloproliferative Neoplasms - 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: M9490 - Name: Hematologic Diseases - 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: M9560 - Name: Hemorrhagic Disorders - Relevance: LOW - As Found: Unknown - ID: M5134 - Name: Bone Marrow Diseases - Relevance: LOW - As Found: Unknown - ID: M14111 - Name: Precancerous Conditions - Relevance: LOW - As Found: Unknown - ID: T3947 - Name: Multiple Myeloma - Relevance: HIGH - As Found: Multiple Myeloma - ID: T4587 - Name: Plasmacytoma - Relevance: HIGH - As Found: Plasma Cell Neoplasm - ID: T3995 - Name: Myeloid Leukemia - Relevance: LOW - As Found: Unknown - ID: T182 - Name: Acute Myeloid Leukemia - Relevance: LOW - As Found: Unknown - ID: T188 - Name: Acute Non Lymphoblastic Leukemia - Relevance: LOW - As Found: Unknown - ID: T1308 - Name: Chronic Lymphocytic Leukemia - Relevance: LOW - As Found: Unknown - ID: T1303 - Name: Chronic Graft Versus Host Disease - Relevance: LOW - As Found: Unknown - ID: T1309 - Name: Chronic Myeloid Leukemia - Relevance: LOW - As Found: Unknown - ID: T175 - Name: Acute Lymphoblastic Leukemia - Relevance: LOW - As Found: Unknown - ID: T3533 - Name: Lymphoblastic Lymphoma - Relevance: LOW - As Found: Unknown - ID: T3543 - Name: Lymphosarcoma - Relevance: LOW - As Found: Unknown - 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: T1866 - Name: Diffuse Large B-Cell Lymphoma - Relevance: LOW - As Found: Unknown - ID: T3612 - Name: Marginal Zone Lymphoma - Relevance: LOW - As Found: Unknown - ID: T2817 - Name: Hodgkin Lymphoma - Relevance: LOW - As Found: Unknown - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown - ID: T1311 - Name: Chronic Myeloproliferative Disorders - Relevance: HIGH - As Found: Chronic Myeloproliferative Disorders - ID: T1134 - Name: Childhood Acute Lymphoblastic Leukemia - Relevance: LOW - As Found: Unknown - ID: T3993 - Name: Myelodysplastic Syndromes - Relevance: HIGH - As Found: Myelodysplastic Syndrome - ID: T4705 - Name: Primary Myelofibrosis - Relevance: LOW - As Found: Unknown - ID: T892 - Name: Burkitt Lymphoma - Relevance: LOW - As Found: Unknown - ID: T3991 - Name: Myelodysplastic Syndrome With Excess Blasts - Relevance: LOW - As Found: Unknown - ID: T1310 - Name: Chronic Myelomonocytic Leukemia - Relevance: LOW - As Found: Unknown - ID: T3174 - Name: Juvenile Myelomonocytic Leukemia - Relevance: LOW - As Found: Unknown - ID: T2650 - Name: Hairy Cell Leukemia - Relevance: LOW - As Found: Unknown - ID: T3994 - Name: Myelodysplastic/myeloproliferative Disease - Relevance: HIGH - As Found: Myelodysplastic/Myeloproliferative Neoplasms - ID: T4586 - Name: Plasmablastic Lymphoma - Relevance: LOW - As Found: Unknown - ID: T3539 - Name: Lymphoma, Large-cell, Immunoblastic - Relevance: LOW - As Found: Unknown - ID: T1312 - Name: Chronic Neutrophilic Leukemia - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000008223 - Term: Lymphoma - ID: D000007938 - Term: Leukemia - ID: D000009369 - Term: Neoplasms - ID: D000009101 - Term: Multiple Myeloma - ID: D000054219 - Term: Neoplasms, Plasma Cell - ID: D000011289 - Term: Preleukemia - ID: D000010954 - Term: Plasmacytoma - ID: D000009190 - Term: Myelodysplastic Syndromes - ID: D000009196 - Term: Myeloproliferative Disorders - ID: D000054437 - Term: Myelodysplastic-Myeloproliferative Diseases - ID: D000013577 - Term: Syndrome ### Intervention Browse Module - Ancestors - ID: D000007166 - Term: Immunosuppressive Agents - ID: D000007155 - Term: Immunologic Factors - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018501 - Term: Antirheumatic Agents - ID: D000018906 - Term: Antineoplastic Agents, Alkylating - ID: D000000477 - Term: Alkylating Agents - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000000970 - Term: Antineoplastic Agents - ID: D000019653 - Term: Myeloablative Agonists - ID: D000000020 - Term: Abortifacient Agents, Nonsteroidal - ID: D000000019 - Term: Abortifacient Agents - ID: D000012102 - Term: Reproductive Control Agents - ID: D000000964 - Term: Antimetabolites, Antineoplastic - ID: D000000963 - Term: Antimetabolites - ID: D000003879 - Term: Dermatologic Agents - ID: D000004791 - Term: Enzyme Inhibitors - ID: D000005493 - Term: Folic Acid Antagonists - ID: D000019384 - Term: Nucleic Acid Synthesis Inhibitors - ID: D000000972 - Term: Antineoplastic Agents, Phytogenic - ID: D000059005 - Term: Topoisomerase II Inhibitors - ID: D000059003 - Term: Topoisomerase Inhibitors - ID: D000000998 - Term: Antiviral Agents - ID: D000000890 - Term: Anti-Infective Agents - ID: D000065095 - Term: Calcineurin Inhibitors - ID: D000000935 - Term: Antifungal Agents - ID: D000000903 - Term: Antibiotics, Antineoplastic - ID: D000000904 - Term: Antibiotics, Antitubercular - ID: D000000995 - Term: Antitubercular Agents - ID: D000000900 - Term: Anti-Bacterial Agents - ID: D000000893 - Term: Anti-Inflammatory Agents - ID: D000000932 - Term: Antiemetics - ID: D000001337 - Term: Autonomic Agents - ID: D000018373 - Term: Peripheral Nervous System Agents - ID: D000005765 - Term: Gastrointestinal Agents - ID: D000005938 - Term: Glucocorticoids - ID: D000006728 - Term: Hormones - ID: D000006730 - Term: Hormones, Hormone Substitutes, and Hormone Antagonists - ID: D000018696 - Term: Neuroprotective Agents - ID: D000020011 - Term: Protective Agents ### Intervention Browse Module - Browse Branches - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: ARhu - Name: Antirheumatic Agents - Abbrev: Derm - Name: Dermatologic Agents - Abbrev: Repr - Name: Reproductive Control Agents - Abbrev: Infl - Name: Anti-Inflammatory Agents - Abbrev: AnEm - Name: Antiemetics - Abbrev: NeuroAg - Name: Neuroprotective Agents - Abbrev: Gast - Name: Gastrointestinal Agents - Abbrev: ChanBlk - Name: Channel Blockers - Abbrev: AntiConv - Name: Anticonvulsants - Abbrev: Micro - Name: Micronutrients - Abbrev: Hemat - Name: Hematinics - Abbrev: Vi - Name: Vitamins ### Intervention Browse Module - Browse Leaves - ID: M6766 - Name: Cytarabine - Relevance: HIGH - As Found: Infants - ID: M11703 - Name: Methotrexate - Relevance: HIGH - As Found: Breast Cancer - ID: M6727 - Name: Cyclophosphamide - Relevance: HIGH - As Found: Cycle - ID: M11541 - Name: Melphalan - Relevance: HIGH - As Found: Modified - ID: M18961 - Name: Cyclosporine - Relevance: HIGH - As Found: New - ID: M6730 - Name: Cyclosporins - Relevance: HIGH - As Found: New - ID: M14120 - Name: Prednisolone - Relevance: LOW - As Found: Unknown - ID: M8191 - Name: Etoposide - Relevance: HIGH - As Found: Tolerability - ID: M283230 - Name: Fludarabine - Relevance: HIGH - As Found: Comparison - ID: M1945 - Name: Lenograstim - Relevance: LOW - As Found: Unknown - ID: M7411 - Name: Diuretics - Relevance: LOW - As Found: Unknown - ID: M12128 - Name: Mycophenolic Acid - Relevance: HIGH - As Found: Mindfulness - ID: M18950 - Name: Tacrolimus - Relevance: HIGH - As Found: 3 weeks - ID: M341643 - Name: Etoposide phosphate - Relevance: LOW - As Found: Unknown - ID: M5336 - Name: Busulfan - Relevance: HIGH - As Found: Full - ID: M11749 - Name: Methylprednisolone - Relevance: HIGH - As Found: Smoking - ID: M225513 - Name: Fludarabine phosphate - Relevance: HIGH - As Found: Intubation - ID: M13577 - Name: Phenytoin - Relevance: LOW - As Found: Unknown - ID: M5585 - Name: Carmustine - Relevance: HIGH - As Found: Needling - ID: M1833 - Name: Methylprednisolone Acetate - Relevance: LOW - As Found: Unknown - ID: M11750 - Name: Methylprednisolone Hemisuccinate - Relevance: LOW - As Found: Unknown - ID: M229449 - Name: Prednisolone acetate - Relevance: LOW - As Found: Unknown - ID: M211887 - Name: Prednisolone hemisuccinate - Relevance: LOW - As Found: Unknown - ID: M248881 - Name: Prednisolone phosphate - Relevance: LOW - As Found: Unknown - ID: M3834 - Name: Allopurinol - Relevance: LOW - As Found: Unknown - ID: M4279 - Name: Antilymphocyte Serum - Relevance: HIGH - As Found: Ibuprofen - ID: M17811 - Name: Mesna - 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: M20604 - Name: Antirheumatic Agents - Relevance: LOW - As Found: Unknown - ID: M20942 - Name: Antineoplastic Agents, Alkylating - Relevance: LOW - As Found: Unknown - ID: M3820 - Name: Alkylating Agents - Relevance: LOW - As Found: Unknown - ID: M4281 - Name: Antimetabolites - Relevance: LOW - As Found: Unknown - ID: M7074 - Name: Dermatologic Agents - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown - ID: M8618 - Name: Folic Acid - Relevance: LOW - As Found: Unknown - ID: M17546 - Name: Vitamin B Complex - Relevance: LOW - As Found: Unknown - ID: M8619 - Name: Folic Acid Antagonists - Relevance: LOW - As Found: Unknown - ID: M4314 - Name: Antiviral Agents - Relevance: LOW - As Found: Unknown - ID: M4214 - Name: Anti-Infective Agents - Relevance: LOW - As Found: Unknown - ID: M30452 - Name: Calcineurin Inhibitors - Relevance: LOW - As Found: Unknown - ID: M6252 - Name: Clotrimazole - Relevance: LOW - As Found: Unknown - ID: M11796 - Name: Miconazole - Relevance: LOW - As Found: Unknown - ID: M4254 - Name: Antifungal Agents - Relevance: LOW - As Found: Unknown - ID: M4222 - Name: Anti-Bacterial Agents - Relevance: LOW - As Found: Unknown - ID: M4224 - Name: Antibiotics, Antitubercular - Relevance: LOW - As Found: Unknown - ID: M4311 - Name: Antitubercular Agents - Relevance: LOW - As Found: Unknown - 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: M20773 - Name: Neuroprotective Agents - Relevance: LOW - As Found: Unknown - ID: M21869 - Name: Protective Agents - 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 ### Intervention Browse Module - Meshes - ID: D000003561 - Term: Cytarabine - ID: D000016572 - Term: Cyclosporine - ID: D000009173 - Term: Mycophenolic Acid - ID: D000008775 - Term: Methylprednisolone - ID: D000003520 - Term: Cyclophosphamide - ID: D000008558 - Term: Melphalan - ID: D000002066 - Term: Busulfan - ID: D000002330 - Term: Carmustine - ID: D000008727 - Term: Methotrexate - ID: C000024352 - Term: Fludarabine - ID: C000042382 - Term: Fludarabine phosphate - ID: D000005047 - Term: Etoposide - ID: D000016559 - Term: Tacrolimus - ID: D000003524 - Term: Cyclosporins - ID: D000000961 - Term: Antilymphocyte Serum ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Cyclophosphamide (Cytoxan) and Total Body Irradiation (TBI) **Description:** On admission day, you will start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Allopurinol tablets will be given to you by mouth up to three times a day for 7 days. You will begin radiation therapy to your entire body at the start of your transplant treatment. This procedure is called TBI (total body irradiation). Radiation will be given to you 2 times a day for 3 or 4 days. After the radiation treatment, you will receive two doses of cyclophosphamide by vein (through a small plastic tube leading into the bloodstream). Each dose takes about 2 hours to administer. When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. After you have completed the cyclophosphamide you will rest one day without any anti-cancer therapy. **ID:** EG000 **Other Num Affected:** 19 **Other Num at Risk:** 128 **Serious Number Affected:** 101 **Serious Number At Risk:** 128 **Title:** Cyclophosphamide (Cytoxan) and Total Body Irradiation (TBI) **Group ID:** EG001 **Title:** Busulfan and Cyclophosphamide (Cytoxan) **Description:** On admission day, you will start to take a drug called Dilantin, which is used to help prevent seizures while you receive your chemotherapy drugs. You will also start to take a drug called allopurinol,which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Allopurinol tablets will be given to you by mouth up to three times a day for 7 days. You will begin the therapy with a drug called busulfan. This medicine is take by mouth four times per day for four days. After the oral busulfan treatment, you will receive two doses of cyclophosphamide over 2 hours by vein (through a small tube leading into the bloodstream). When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. Subjects undergoing the Busulfan and Cyclophosphamide regimen will not have total body irradiation (TBI). **ID:** EG001 **Other Num at Risk:** 36 **Serious Number Affected:** 25 **Serious Number At Risk:** 36 **Title:** Busulfan and Cyclophosphamide (Cytoxan) **Group ID:** EG002 **Title:** BEAM Regimen **Description:** On the day of your admission, you will start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Chemotherapy will begin on Day -6 with carmustine (BCNU), followed by etoposide (VP-16), cytosine arabinoside (ARA-C), and melphalan. This conditioning regimen is known as the BEAM regimen. The dose of this therapy is high enough to kill cancer cells but will also kill all of your normal blood forming cells. Subjects undergoing the BEAM regimen will not have total body irradiation (TBI). **ID:** EG002 **Other Num Affected:** 2 **Other Num at Risk:** 20 **Serious Number Affected:** 17 **Serious Number At Risk:** 20 **Title:** BEAM Regimen **Group ID:** EG003 **Title:** Low-Dose Fludarabine and TBI(for Second Stem Cell Donation) **Description:** A conditioning regimen of low-dose fludarabine and TBI is used in the event that a second donation of hematopoietic stem cells is necessary. Chemotherapy with Fludarabine will begin 4 days prior to your transplant. This drug will be given through the catheter in your chest daily for 3 days. TBI (radiation) will be given to you on the day of your transplant. After your TBI, your donor's stem cells / bone marrow will be given to you through your catheter. The drugs cyclosporine and mycophenolate mofetil (MMF) will be given orally to help you accept your donor's cells. **ID:** EG003 **Title:** Low-Dose Fludarabine and TBI(for Second Stem Cell Donation) **Group ID:** EG004 **Title:** Busulfan, Cyclophosphamide, and Fludarabine (Pediatric Only) **Description:** On the day of your admission you will start to take a drug called Dilantin which is used to help prevent seizures while you receive your chemotherapy drugs. You will also start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. On the next day, you will then begin your conditioning therapy with a drug called busulfan. This medicine will be given to you by an infusion into your bloodstream through a small tube in the vein of your arm four times per day for four days. After the busulfan treatment, you will receive 4 doses each of two drugs, cyclophosphamide (also known as Cytoxan) and fludarabine, over 2 hours into your vein. **ID:** EG004 **Other Num at Risk:** 6 **Serious Number Affected:** 5 **Serious Number At Risk:** 6 **Title:** Busulfan, Cyclophosphamide, and Fludarabine (Pediatric Only) **Group ID:** EG005 **Title:** ATG For Cord Blood Transplants **Description:** If you are undergoing a pre-transplant conditioning regimen prior to undergoing a cord blood transplant, you will receive a drug called ATG to improve your chances of engraftment and decrease your risk of graft versus host disease. You may receive ATG 3 times during your transplant regimen on days -3 through days -1 in addition to your pre-transplant conditioning therapy. Methylprednisolone will also be given during each dose of ATG to help reduce any reactions during infusion. **ID:** EG005 **Other Num at Risk:** 9 **Serious Number Affected:** 6 **Serious Number At Risk:** 9 **Title:** ATG For Cord Blood Transplants **Group ID:** EG006 **Title:** DLI (Donor Leukocyte Infusion) **Description:** Donor Leukocyte Infusions: You will receive DLI from your original transplant donor. This will be given through a vein , usually in your arm. It will be similar to getting a platelet or blood transfusion. You may require more than one DLI. The decision to give you another infusion will be determined by your condition, relapse status, GVHD and how much DLI you were given before. You may need chemotherapy and/or radiation to improve your disease status prior to additional DLI's. **ID:** EG006 **Title:** DLI (Donor Leukocyte Infusion) **Group ID:** EG007 **Title:** Cyclophosphamide, Etoposide (VP16) and TBI (Pediatric Only) **Description:** On the day after your admission, you will start receiving radiation therapy (TBI). Radiation will be given to you 2 times a day for 3 days. On the next day, you will then begin your chemotherapy with a drug called etoposide. This medicine will be given to you by an infusion into your bloodstream through a small tube in the vein of your arm for one day. After the etoposide treatment, on the next day you will receive cyclophosphamide (also known as Cytoxan) for 2 days. When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. After you have completed the cyclophosphamide you will rest one day without any anti-cancer therapy. This allows your body time to remove and inactivate the chemotherapy. After a day of rest, you will be given your donor's cells. **ID:** EG007 **Other Num at Risk:** 1 **Serious Number Affected:** 1 **Serious Number At Risk:** 1 **Title:** Cyclophosphamide, Etoposide (VP16) and TBI (Pediatric Only) **Frequency Threshold:** 5 #### Other Events **Term:** Headache **Organ System:** General disorders **Source Vocabulary:** **Term:** Nausea **Organ System:** General disorders **Source Vocabulary:** #### Serious Events **Term:** Staph Sepsis **Organ System:** Blood and lymphatic system disorders ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 128 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Altered mental status **Organ System:** Psychiatric disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 128 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Pneumonia **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 5 **Num At Risk:** 128 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Hypoxia **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 128 **Group ID:** EG001 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Death **Organ System:** General disorders ##### Stats **Group ID:** EG000 **Num Affected:** 101 **Num At Risk:** 128 **Group ID:** EG001 **Num Affected:** 25 **Num At Risk:** 36 **Group ID:** EG002 **Num Affected:** 17 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num Affected:** 5 **Num At Risk:** 6 **Group ID:** EG005 **Num Affected:** 6 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num Affected:** 1 **Num At Risk:** 1 **Num Events:** 1 **Term:** Fever **Organ System:** Immune system disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 128 **Group ID:** EG001 **Num At Risk:** 36 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Graft versus host disease (GVHD) **Organ System:** Gastrointestinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 17 **Num At Risk:** 128 **Group ID:** EG001 **Num Affected:** 13 **Num At Risk:** 36 **Group ID:** EG002 **Num Affected:** 3 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num Affected:** 1 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Acute Respiratory Distress Syndrome (ARDS) **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 128 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 36 **Group ID:** EG002 **Num Affected:** 2 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num Affected:** 1 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num Affected:** 1 **Num At Risk:** 1 **Term:** Cardiac Problems **Organ System:** Cardiac disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 128 **Group ID:** EG001 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Thrombocytopenia **Organ System:** Blood and lymphatic system disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 128 **Group ID:** EG001 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Diarrhea **Organ System:** General disorders ##### Stats **Group ID:** EG000 **Num Affected:** 4 **Num At Risk:** 128 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Orthostatic Hypertension **Organ System:** Blood and lymphatic system disorders ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 128 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** acute renal failure **Organ System:** Renal and urinary disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 128 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 36 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num Affected:** 1 **Num At Risk:** 1 **Term:** Intraparenchymal Hemorrhage **Organ System:** Vascular disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 128 **Group ID:** EG001 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Pulmonary Hemorrhage **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 128 **Group ID:** EG001 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num Affected:** 1 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Rigors **Organ System:** Immune system disorders ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 128 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Pneutropenic Fever **Organ System:** Immune system disorders ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 128 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Back pain **Organ System:** Musculoskeletal and connective tissue disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 128 **Group ID:** EG001 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Kidney Stones **Organ System:** Renal and urinary disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 128 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 36 **Group ID:** EG002 **Num Affected:** 2 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Encephalopathy **Organ System:** Infections and infestations ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 128 **Group ID:** EG001 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Viral encephalitis **Organ System:** Infections and infestations ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 128 **Group ID:** EG001 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Pulmonary aspergillus **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 128 **Group ID:** EG001 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Avascular necrosis **Organ System:** Musculoskeletal and connective tissue disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 128 **Group ID:** EG001 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Anorexia **Organ System:** Psychiatric disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 128 **Group ID:** EG001 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Dehydration **Organ System:** General disorders ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 128 **Group ID:** EG001 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Alveolar hemorrhage **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 15 **Num At Risk:** 128 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 36 **Group ID:** EG002 **Num Affected:** 2 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Bronchopneumonia **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 4 **Num At Risk:** 128 **Group ID:** EG001 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Bacterial pneumonia **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 128 **Group ID:** EG001 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Seizures **Organ System:** General disorders ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 128 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Cytomegalovirus pneumonitis **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 128 **Group ID:** EG001 **Num At Risk:** 36 **Group ID:** EG002 **Num Affected:** 1 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Tachycardia **Organ System:** Cardiac disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 128 **Group ID:** EG001 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Positive blood cultures **Organ System:** Blood and lymphatic system disorders ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 128 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Gastroenteritis **Organ System:** Gastrointestinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 128 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Liver failure **Organ System:** Hepatobiliary disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 128 **Group ID:** EG001 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Hyperbilirubinemia **Organ System:** Hepatobiliary disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 128 **Group ID:** EG001 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Fungal infection **Organ System:** Infections and infestations ##### Stats **Group ID:** EG000 **Num Affected:** 11 **Num At Risk:** 128 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 36 **Group ID:** EG002 **Num Affected:** 2 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num Affected:** 1 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 **Term:** Steroid-induced psychoses **Organ System:** Psychiatric disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 128 **Group ID:** EG001 **Num At Risk:** 36 **Group ID:** EG002 **Num At Risk:** 20 **Group ID:** EG003 **Group ID:** EG004 **Num At Risk:** 6 **Group ID:** EG005 **Num Affected:** 1 **Num At Risk:** 9 **Group ID:** EG006 **Group ID:** EG007 **Num At Risk:** 1 ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 128 **Group ID:** BG001 **Value:** 36 **Group ID:** BG002 **Value:** 20 **Group ID:** BG003 **Value:** 0 **Group ID:** BG004 **Value:** 6 **Group ID:** BG005 **Value:** 9 **Group ID:** BG006 **Value:** 0 **Group ID:** BG007 **Value:** 1 **Group ID:** BG008 **Value:** 200 **Units:** Participants ### Group **ID:** BG000 **Title:** Cyclophosphamide (Cytoxan) and Total Body Irradiation (TBI) **Description:** On admission day, you will start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Allopurinol tablets will be given to you by mouth up to three times a day for 7 days. You will begin radiation therapy to your entire body at the start of your transplant treatment. This procedure is called TBI (total body irradiation). Radiation will be given to you 2 times a day for 3 or 4 days. After the radiation treatment, you will receive two doses of cyclophosphamide by vein (through a small plastic tube leading into the bloodstream). Each dose takes about 2 hours to administer. When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. After you have completed the cyclophosphamide you will rest one day without any anti-cancer therapy. ### Group **ID:** BG001 **Title:** Busulfan and Cyclophosphamide (Cytoxan) **Description:** On admission day, you will start to take a drug called Dilantin, which is used to help prevent seizures while you receive your chemotherapy drugs. You will also start to take a drug called allopurinol,which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Allopurinol tablets will be given to you by mouth up to three times a day for 7 days. You will begin the therapy with a drug called busulfan. This medicine is take by mouth four times per day for four days. After the oral busulfan treatment, you will receive two doses of cyclophosphamide over 2 hours by vein (through a small tube leading into the bloodstream). When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. Subjects undergoing the Busulfan and Cyclophosphamide regimen will not have total body irradiation (TBI). ### Group **ID:** BG002 **Title:** BEAM Regimen **Description:** On the day of your admission, you will start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Chemotherapy will begin on Day -6 with carmustine (BCNU), followed by etoposide (VP-16), cytosine arabinoside (ARA-C), and melphalan. This conditioning regimen is known as the BEAM regimen. The dose of this therapy is high enough to kill cancer cells but will also kill all of your normal blood forming cells. Subjects undergoing the BEAM regimen will not have total body irradiation (TBI). ### Group **ID:** BG003 **Title:** Low-Dose Fludarabine and TBI(for Second Stem Cell Donation) **Description:** A conditioning regimen of low-dose fludarabine and TBI is used in the event that a second donation of hematopoietic stem cells is necessary. Chemotherapy with Fludarabine will begin 4 days prior to your transplant. This drug will be given through the catheter in your chest daily for 3 days. TBI (radiation) will be given to you on the day of your transplant. After your TBI, your donor's stem cells / bone marrow will be given to you through your catheter. The drugs cyclosporine and mycophenolate mofetil (MMF) will be given orally to help you accept your donor's cells. ### Group **ID:** BG004 **Title:** Busulfan, Cyclophosphamide, and Fludarabine (Pediatric Only) **Description:** On the day of your admission you will start to take a drug called Dilantin which is used to help prevent seizures while you receive your chemotherapy drugs. You will also start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. On the next day, you will then begin your conditioning therapy with a drug called busulfan. This medicine will be given to you by an infusion into your bloodstream through a small tube in the vein of your arm four times per day for four days. After the busulfan treatment, you will receive 4 doses each of two drugs, cyclophosphamide (also known as Cytoxan) and fludarabine, over 2 hours into your vein. ### Group **ID:** BG005 **Title:** ATG For Cord Blood Transplants **Description:** If you are undergoing a pre-transplant conditioning regimen prior to undergoing a cord blood transplant, you will receive a drug called ATG to improve your chances of engraftment and decrease your risk of graft versus host disease. You may receive ATG 3 times during your transplant regimen on days -3 through days -1 in addition to your pre-transplant conditioning therapy. Methylprednisolone will also be given during each dose of ATG to help reduce any reactions during infusion. ### Group **ID:** BG006 **Title:** DLI (Donor Leukocyte Infusion) **Description:** Donor Leukocyte Infusions: You will receive DLI from your original transplant donor. This will be given through a vein , usually in your arm. It will be similar to getting a platelet or blood transfusion. You may require more than one DLI. The decision to give you another infusion will be determined by your condition, relapse status, GVHD and how much DLI you were given before. You may need chemotherapy and/or radiation to improve your disease status prior to additional DLI's. ### Group **ID:** BG007 **Title:** Cyclophosphamide, Etoposide (VP16) and TBI (Pediatric Only) **Description:** On the day after your admission, you will start receiving radiation therapy (TBI). Radiation will be given to you 2 times a day for 3 days. On the next day, you will then begin your chemotherapy with a drug called etoposide. This medicine will be given to you by an infusion into your bloodstream through a small tube in the vein of your arm for one day. After the etoposide treatment, on the next day you will receive cyclophosphamide (also known as Cytoxan) for 2 days. When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. After you have completed the cyclophosphamide you will rest one day without any anti-cancer therapy. This allows your body time to remove and inactivate the chemotherapy. After a day of rest, you will be given your donor's cells. ### Group **ID:** BG008 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Value:** 27 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG004 **Value:** 6 #### Measurement **Group ID:** BG005 **Value:** 7 #### Measurement **Group ID:** BG007 **Value:** 1 #### Measurement **Group ID:** BG008 **Value:** 42 **Category Title:** <=18 years #### Measurement **Group ID:** BG000 **Value:** 101 #### Measurement **Group ID:** BG001 **Value:** 35 #### Measurement **Group ID:** BG002 **Value:** 20 #### Measurement **Group ID:** BG004 **Value:** 0 #### Measurement **Group ID:** BG005 **Value:** 0 #### Measurement **Group ID:** BG007 **Value:** 0 #### Measurement **Group ID:** BG008 **Value:** 156 **Category Title:** Between 18 and 65 years #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG004 **Value:** 0 #### Measurement **Group ID:** BG005 **Value:** 2 #### Measurement **Group ID:** BG007 **Value:** 0 #### Measurement **Group ID:** BG008 **Value:** 2 **Category Title:** >=65 years **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 14.923 **Value:** 32.033 #### Measurement **Group ID:** BG001 **Spread:** 11.67 **Value:** 48.33 #### Measurement **Group ID:** BG002 **Spread:** 12.87 **Value:** 46.187 #### Measurement **Group ID:** BG004 **Spread:** 7.90 **Value:** 12.22 #### Measurement **Group ID:** BG005 **Spread:** 10.725 **Value:** 11.77 #### Measurement **Group ID:** BG007 **Spread:** 0 **Value:** 5.42 #### Measurement **Group ID:** BG008 **Spread:** 16.818 **Value:** 34.743 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 56 #### Measurement **Group ID:** BG001 **Value:** 13 #### Measurement **Group ID:** BG002 **Value:** 3 #### Measurement **Group ID:** BG004 **Value:** 4 #### Measurement **Group ID:** BG005 **Value:** 5 #### Measurement **Group ID:** BG007 **Value:** 1 #### Measurement **Group ID:** BG008 **Value:** 82 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 72 #### Measurement **Group ID:** BG001 **Value:** 23 #### Measurement **Group ID:** BG002 **Value:** 17 #### Measurement **Group ID:** BG004 **Value:** 2 #### Measurement **Group ID:** BG005 **Value:** 4 #### Measurement **Group ID:** BG007 **Value:** 0 #### Measurement **Group ID:** BG008 **Value:** 118 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 128 #### Measurement **Group ID:** BG001 **Value:** 36 #### Measurement **Group ID:** BG002 **Value:** 20 #### Measurement **Group ID:** BG004 **Value:** 6 #### Measurement **Group ID:** BG005 **Value:** 9 #### Measurement **Group ID:** BG007 **Value:** 1 #### Measurement **Group ID:** BG008 **Value:** 200 **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 ## Results Section - More Information Module ### Certain Agreement ### Limitations and Caveats **Description:** Early termination ### Point of Contact **Email:** [email protected] **Organization:** OHSU Knight Cancer Institute **Phone:** 503-494-1551 **Title:** Dr. Richard Maziarz ## Results Section - Outcome Measures Module ### Outcome Measure 1 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Determine the effectiveness of unrelated donor allogeneic hematopoietic stem cells for transplantation after conditioning for the treatment of high-risk hematopoietic malignancies. Disease-free survival: The length of time after treatment ends that a patient survives without any signs or symptoms of that cancer or any other type of cancer. **Reporting Status:** POSTED **Time Frame:** Duration of the study; Up to 2 years **Title:** Number of Participants With Disease Free Survival (DFS). **Type:** PRIMARY ##### Group **ID:** OG000 **Title:** Cyclophosphamide (Cytoxan) and Total Body Irradiation (TBI) ##### Group **ID:** OG001 **Title:** Busulfan and Cyclophosphamide (Cytoxan) ##### Group **Description:** On the day of your admission, you will start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Chemotherapy will begin on Day -6 with carmustine (BCNU), followed by etoposide (VP-16), cytosine arabinoside (ARA-C), and melphalan. This conditioning regimen is known as the BEAM regimen. The dose of this therapy is high enough to kill cancer cells but will also kill all of your normal blood forming cells. Subjects undergoing the BEAM regimen will not have total body irradiation (TBI). **ID:** OG002 **Title:** BEAM Regimen ##### Group **Description:** A conditioning regimen of low-dose fludarabine and TBI is used in the event that a second donation of hematopoietic stem cells is necessary. Chemotherapy with Fludarabine will begin 4 days prior to your transplant. This drug will be given through the catheter in your chest daily for 3 days. TBI (radiation) will be given to you on the day of your transplant. After your TBI, your donor's stem cells / bone marrow will be given to you through your catheter. The drugs cyclosporine and mycophenolate mofetil (MMF) will be given orally to help you accept your donor's cells. **ID:** OG003 **Title:** Low-Dose Fludarabine and TBI(for Second Stem Cell Donation) ##### Group **Description:** On the day of your admission you will start to take a drug called Dilantin which is used to help prevent seizures while you receive your chemotherapy drugs. You will also start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. On the next day, you will then begin your conditioning therapy with a drug called busulfan. This medicine will be given to you by an infusion into your bloodstream through a small tube in the vein of your arm four times per day for four days. After the busulfan treatment, you will receive 4 doses each of two drugs, cyclophosphamide (also known as Cytoxan) and fludarabine, over 2 hours into your vein. **ID:** OG004 **Title:** Busulfan, Cyclophosphamide, and Fludarabine (Pediatric Only) ##### Group **Description:** If you are undergoing a pre-transplant conditioning regimen prior to undergoing a cord blood transplant, you will receive a drug called ATG to improve your chances of engraftment and decrease your risk of graft versus host disease. You may receive ATG 3 times during your transplant regimen on days -3 through days -1 in addition to your pre-transplant conditioning therapy. Methylprednisolone will also be given during each dose of ATG to help reduce any reactions during infusion. **ID:** OG005 **Title:** ATG For Cord Blood Transplants ##### Group **Description:** Donor Leukocyte Infusions: You will receive DLI from your original transplant donor. This will be given through a vein , usually in your arm. It will be similar to getting a platelet or blood transfusion. You may require more than one DLI. The decision to give you another infusion will be determined by your condition, relapse status, GVHD and how much DLI you were given before. You may need chemotherapy and/or radiation to improve your disease status prior to additional DLI's. **ID:** OG006 **Title:** DLI (Donor Leukocyte Infusion) ##### Group **Description:** On the day after your admission, you will start receiving radiation therapy (TBI). Radiation will be given to you 2 times a day for 3 days. On the next day, you will then begin your chemotherapy with a drug called etoposide. This medicine will be given to you by an infusion into your bloodstream through a small tube in the vein of your arm for one day. After the etoposide treatment, on the next day you will receive cyclophosphamide (also known as Cytoxan) for 2 days. When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. After you have completed the cyclophosphamide you will rest one day without any anti-cancer therapy. This allows your body time to remove and inactivate the chemotherapy. After a day of rest, you will be given your donor's cells. **ID:** OG007 **Title:** Cyclophosphamide, Etoposide (VP16) and TBI (Pediatric Only) ### Participant Flow Module #### Group **Description:** On admission day, you will start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Allopurinol tablets will be given to you by mouth up to three times a day for 7 days. You will begin radiation therapy to your entire body at the start of your transplant treatment. This procedure is called TBI (total body irradiation). Radiation will be given to you 2 times a day for 3 or 4 days. After the radiation treatment, you will receive two doses of cyclophosphamide by vein (through a small plastic tube leading into the bloodstream). Each dose takes about 2 hours to administer. When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. After you have completed the cyclophosphamide you will rest one day without any anti-cancer therapy. **ID:** FG000 **Title:** Cyclophosphamide (Cytoxan) and Total Body Irradiation (TBI) #### Group **Description:** On admission day, you will start to take a drug called Dilantin, which is used to help prevent seizures while you receive your chemotherapy drugs. You will also start to take a drug called allopurinol,which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Allopurinol tablets will be given to you by mouth up to three times a day for 7 days. You will begin the therapy with a drug called busulfan. This medicine is take by mouth four times per day for four days. After the oral busulfan treatment, you will receive two doses of cyclophosphamide over 2 hours by vein (through a small tube leading into the bloodstream). When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. Subjects undergoing the Busulfan and Cyclophosphamide regimen will not have total body irradiation (TBI). **ID:** FG001 **Title:** Busulfan and Cyclophosphamide (Cytoxan) #### Group **Description:** On the day of your admission, you will start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. Chemotherapy will begin on Day -6 with carmustine (BCNU), followed by etoposide (VP-16), cytosine arabinoside (ARA-C), and melphalan. This conditioning regimen is known as the BEAM regimen. The dose of this therapy is high enough to kill cancer cells but will also kill all of your normal blood forming cells. Subjects undergoing the BEAM regimen will not have total body irradiation (TBI). **ID:** FG002 **Title:** BEAM Regimen #### Group **Description:** A conditioning regimen of low-dose fludarabine and TBI is used in the event that a second donation of hematopoietic stem cells is necessary. Chemotherapy with Fludarabine will begin 4 days prior to your transplant. This drug will be given through the catheter in your chest daily for 3 days. TBI (radiation) will be given to you on the day of your transplant. After your TBI, your donor's stem cells / bone marrow will be given to you through your catheter. The drugs cyclosporine and mycophenolate mofetil (MMF) will be given orally to help you accept your donor's cells. **ID:** FG003 **Title:** Low-Dose Fludarabine and TBI(for Second Stem Cell Donation) #### Group **Description:** On the day of your admission you will start to take a drug called Dilantin which is used to help prevent seizures while you receive your chemotherapy drugs. You will also start to take a drug called allopurinol which helps to protect your kidneys as your body works to discharge cells killed off by your chemotherapy and TBI. On the next day, you will then begin your conditioning therapy with a drug called busulfan. This medicine will be given to you by an infusion into your bloodstream through a small tube in the vein of your arm four times per day for four days. After the busulfan treatment, you will receive 4 doses each of two drugs, cyclophosphamide (also known as Cytoxan) and fludarabine, over 2 hours into your vein. **ID:** FG004 **Title:** Busulfan, Cyclophosphamide, and Fludarabine (Pediatric Only) #### Group **Description:** If you are undergoing a pre-transplant conditioning regimen prior to undergoing a cord blood transplant, you will receive a drug called ATG to improve your chances of engraftment and decrease your risk of graft versus host disease. You may receive ATG 3 times during your transplant regimen on days -3 through days -1 in addition to your pre-transplant conditioning therapy. Methylprednisolone will also be given during each dose of ATG to help reduce any reactions during infusion. **ID:** FG005 **Title:** ATG For Cord Blood Transplants #### Group **Description:** Donor Leukocyte Infusions: You will receive DLI from your original transplant donor. This will be given through a vein , usually in your arm. It will be similar to getting a platelet or blood transfusion. You may require more than one DLI. The decision to give you another infusion will be determined by your condition, relapse status, GVHD and how much DLI you were given before. You may need chemotherapy and/or radiation to improve your disease status prior to additional DLI's. **ID:** FG006 **Title:** DLI (Donor Leukocyte Infusion) #### Group **Description:** On the day after your admission, you will start receiving radiation therapy (TBI). Radiation will be given to you 2 times a day for 3 days. On the next day, you will then begin your chemotherapy with a drug called etoposide. This medicine will be given to you by an infusion into your bloodstream through a small tube in the vein of your arm for one day. After the etoposide treatment, on the next day you will receive cyclophosphamide (also known as Cytoxan) for 2 days. When you are given cyclophosphamide, you will also be given a medication called MESNA to help protect your bladder from damage. After you have completed the cyclophosphamide you will rest one day without any anti-cancer therapy. This allows your body time to remove and inactivate the chemotherapy. After a day of rest, you will be given your donor's cells. **ID:** FG007 **Title:** Cyclophosphamide, Etoposide (VP16) and TBI (Pediatric Only) #### Period **Title:** Overall Study ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 128 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 36 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 20 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG004 **Number of Subjects:** 6 ###### Achievement **Group ID:** FG005 **Number of Subjects:** 9 ###### Achievement **Group ID:** FG006 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG007 **Number of Subjects:** 1 ##### 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 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 128 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 36 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 20 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG004 **Number of Subjects:** 6 ###### Achievement **Group ID:** FG005 **Number of Subjects:** 9 ###### Achievement **Group ID:** FG006 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG007 **Number of Subjects:** 1 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT02477579 **Brief Title:** A Prospective, Multi-Center, Pivotal Study to Evaluate the Safety and Effectiveness of the NovaCross™ Micro-catheter in Facilitating Crossing Chronic Total Occlusion (CTO) Coronary Lesions **Official Title:** A Prospective, Multi-Center, Non-randomized, Single Arm, Open Label, Pivotal Study to Evaluate the Safety and Effectiveness of the NovaCross™ Micro-catheter in Facilitating Crossing Chronic Total Occlusion (CTO) Coronary Lesions #### Organization Study ID Info **ID:** NT-CLP-01 #### Organization **Class:** INDUSTRY **Full Name:** Nitiloop Ltd. ### Status Module #### Completion Date **Date:** 2017-08-10 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-08-17 **Type:** ACTUAL **Last Update Submit Date:** 2017-08-14 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2017-08-10 **Type:** ACTUAL #### Start Date **Date:** 2015-05-17 **Type:** ACTUAL **Status Verified Date:** 2017-08 #### Study First Post Date **Date:** 2015-06-23 **Type:** ESTIMATED **Study First Submit Date:** 2015-06-17 **Study First Submit QC Date:** 2015-06-22 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Nitiloop Ltd. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** This study evaluates the safety and effectiveness of the a novel device called NovaCross to help cross Chronic Total Occlusion (CTO) lesions in coronary arteries. **Detailed Description:** The purpose of this trial is to evaluate the safety and effectiveness of the NovaCross™ micro-catheter when used to facilitate crossing of Chronic Total Occlusion (CTO) lesions in coronary arteries. The procedure will be conducted on consenting patients diagnosed with a CTO in a coronary vessel that requires revascularization after a previously failed attempt to cross or refractory to 10 minutes of conventional guidewire attempt. ### Conditions Module **Conditions:** - Chronic Total Occlusion of Coronary Artery ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 145 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** NovaCross microcatheter will be used. **Intervention Names:** - Device: NovaCross **Label:** NovaCross **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - NovaCross **Description:** The NovaCross™ is a guidewire positioning and support microcatheter for improving chronic total occlusion (CTO) crossability. The NovaCross™ gains its supportive characteristics through the use of a unique operator-controlled Nitinol scaffold and an extendable segment, both at its distal tip. **Name:** NovaCross **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** 30 day MACE, defined as the composite of death, myocardial infarction (MI), or urgent revascularization (target vessel revascularization (TVR) or urgent coronary artery bypass surgery (CABG)). **Measure:** MACE **Time Frame:** 30 days **Description:** Intra-procedural technical success, defined as the ability of the NovaCross™ micro-catheter to successfully facilitate placement of a guidewire beyond a native coronary chronic total occlusion (CTO) in the true vessel lumen. **Measure:** Intra-procedural technical success **Time Frame:** Intra-procedure #### Secondary Outcomes **Description:** 1. The ability to cross the lesion with a guidewire in the true lumen, effectively dilate the CTO lesion, and place a coronary stent with residual lumen stenosis of less than 30% while restoring antegrade TIMI 3 flow. **Measure:** 1. The ability to cross the lesion with a guidewire in the true lumen, effectively dilate the CTO lesion, and place a coronary stent with residual lumen stenosis of less than 30% while restoring antegrade TIMI 3 flow. **Time Frame:** Procedure **Description:** 2. The ability of the NovaCross™ micro-catheter to facilitate a guidewire successfully penetrating the proximal cap of the CTO. **Measure:** Proximal cap (ability of the NovaCross™ micro-catheter to facilitate a guidewire successfully penetrating the proximal cap of the CTO.) **Time Frame:** Procedure **Description:** 3. The effectiveness of the extendable portion in intra-CTO microcatheter crossability. **Measure:** Effectiveness of extendable portion (effectiveness of the extendable portion in intra-CTO microcatheter crossability) **Time Frame:** Procedure **Description:** 4. The ability to have full visualization of the NovaCross during the CTO procedure. **Measure:** Visualization (ability to have full visualization of the NovaCross during the CTO procedure) **Time Frame:** Procedure **Description:** 5. Assess the usability of the NovaCross™ by the operator. **Measure:** Usability (Assess the usability of the NovaCross™ by the operator) **Time Frame:** Procedure **Description:** 6. Device-related perforation at the site of target coronary lesion and/or its proximal reference segment. **Measure:** Device-related perforation (at the site of target coronary lesion and/or its proximal reference segment) **Time Frame:** Procedure ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Adult aged 25-80 * Patient understands and has signed the study informed consent form. * Patient has an angiographic documented Chronic Total Occlusion (i.e. \>3 months occlusion duration) showing distal TIMI flow 0. * Suitable candidate for non-emergent, coronary angioplasty * Documented coronary angiography preceding the PCI reveals at least one CTO lesion situated in a non-infarct related coronary artery or its side branches * Body Mass Index (BMI) \< 40 * Left ventricle ejection fraction \> 25% Exclusion Criteria: * Patient unable to give informed consent. * Current participation in another study with any investigational drug or device. * Patient is known or suspected not to tolerate the contrast agent. * Aorta-ostial CTO location (Ostial bifurcation origins may be considered), SVG CTO, in-stent CTO. * Intolerance to Aspirin and/or inability to tolerate a second antiplatelet agent (Clopidogrel and Prasugrel and Ticagrelor). * Appearance of a fresh thrombus or intraluminal filling defects. * Recent major cerebrovascular event (history of stroke or TIA within 1 month) * Cardiac intervention within 4 weeks of the procedure * Renal insufficiency (serum creatinine of \> 2.3mg/dl or 203μmol/L) * Active gastrointestinal bleeding * Active infection or fever that may be due to infection * Life expectancy \< 2 years due to other illnesses * Significant anemia (hemoglobin \< 8.0 mg / dl) * Severe uncontrolled systemic hypertension (\> 240 mmHg within 1 month of procedure) * Severe electrolyte imbalance * Congestive heart failure \[New York Heart Association (NYHA) Class III\\IV\] CSA Class IV. * Unstable angina requiring emergent percutaneous trans-luminal coronary angioplasty (PTCA) or coronary artery bypass graft (CABG) * Recent myocardial infarction (MI) (within the past two weeks) * Uncontrolled diabetes \>2 serum glucose concentrations of \>350 mg/dl within 7 days. * Unwillingness or inability to comply with any protocol requirements * Pregnant or nursing * Extensive prior dissection from a coronary guidewire use * Drug abuse or alcoholism. * Patients under custodial care. * Bleeding diathesis or coagulation disorder; * Kawasaki's disease or other vasculitis **Maximum Age:** 80 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:** Central Arkansas Veterans Healthcare System **State:** Arkansas **Zip:** 72205 **Location 2:** **City:** Naperville **Country:** United States **Facility:** Edward Hospital **State:** Illinois **Zip:** 60540 **Location 3:** **City:** New York **Country:** United States **Facility:** Columbia University Medical Center/New York Presbyterian Hospital **State:** New York **Zip:** 10032 **Location 4:** **City:** York **Country:** United States **Facility:** York Hospital **State:** Pennsylvania **Zip:** 17403 **Location 5:** **City:** Krakow **Country:** Poland **Facility:** SPZOZ University Hospital in Krakow #### Overall Officials **Official 1:** **Affiliation:** Belfast Health and Social Care Trust **Name:** Simon Walsh, MD **Role:** PRINCIPAL_INVESTIGATOR ## Annotation Section ### Unposted Annotation #### Event: RELEASE - Date: 2019-12-04 - Date Unknown: Unknown #### Event: RESET - Date: 2019-12-18 - Date Unknown: Unknown ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: Rare - Name: Rare Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: T1303 - Name: Chronic Graft Versus Host Disease - Relevance: HIGH - As Found: Chronic ### Misc Info Module #### Submission Tracking - Estimated Results First Submit Date: 2019-12-04 ##### Submission Infos - MCP Release N: Unknown - Release Date: 2019-12-04 - Reset Date: 2019-12-18 - Unrelease Date: Unknown - Unrelease Date Unknown: Unknown - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06295679 **Brief Title:** A Study Assessing Repatha® in Combination With Standard of Care (SOC) Compared With SOC on Major Cardiovascular Events in Chinese Participants With Atherosclerotic Cardiovascular Disease **Official Title:** A Real-world, Prospective, Observational Study Assessing the Effectiveness of Repatha® Used in Combination With Standard of Care Compared With Standard of Care Alone on Major Cardiovascular Events in Chinese Patients With Established Atherosclerotic Cardiovascular Disease #### Organization Study ID Info **ID:** 20180442 #### Organization **Class:** INDUSTRY **Full Name:** Amgen ### Status Module #### Completion Date **Date:** 2028-12-19 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-05-16 **Type:** ACTUAL **Last Update Submit Date:** 2024-05-15 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2028-12-19 **Type:** ESTIMATED #### Start Date **Date:** 2022-12-19 **Type:** ACTUAL **Status Verified Date:** 2024-05 #### Study First Post Date **Date:** 2024-03-06 **Type:** ACTUAL **Study First Submit Date:** 2024-02-26 **Study First Submit QC Date:** 2024-03-04 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Amgen #### 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 primary objective of the study is to evaluate real-world effectiveness of treatment with Repatha® in combination with SOC, compared with SOC alone, on the risk for cardiovascular (CV) death, myocardial infarction (MI), stroke, hospitalization for unstable angina, or coronary revascularization, whichever occurs first, in participants with established atherosclerotic CV disease (ASCVD) treated with SOC, according to local clinical practice. ### Conditions Module **Conditions:** - Major Cardiovascular Event - Established Atherosclerotic Cardiovascular Disease **Keywords:** - Cardiovascular death - Myocardial infarction - Stroke - Unstable angina - Coronary revascularization - Atherosclerotic Cardiovascular Disease - Repatha ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 7000 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants with clinically evident ASCVD treated with Repatha® in combination with SOC in a clinical setting. To ensure that the recruitment strategy has as little impact on routine practice as possible, the decision to treat the participant with Repatha® with SOC will be made independently of, and before, enrollment in the study. **Label:** Repatha® with Standard of Care Exposure #### Arm Group 2 **Description:** Participants with clinically evident ASCVD treated with SOC alone in a clinical setting. To ensure that the recruitment strategy has as little impact on routine practice as possible, the decision to treat the participant with SOC only will be made independently of, and before, enrollment in the study. **Label:** Standard of Care Exposure ### Outcomes Module #### Primary Outcomes **Measure:** Time to CV Death, MI, Hospitalization for Unstable Angina, Stroke, or Coronary Revascularization, Whichever Occurs First **Time Frame:** Up to 72 months #### Secondary Outcomes **Measure:** Time to CV Death, MI, or Stroke, Whichever Occurs First **Time Frame:** Up to 72 months **Measure:** Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline **Time Frame:** Baseline and end of follow-up (up to 72 months) **Measure:** Percent Change in LDL-C From Baseline **Time Frame:** Baseline and end of follow-up (up to 72 months) **Measure:** Number of Participants Who Experienced Adverse Events **Time Frame:** Up to 72 months **Measure:** Number of Participants Who Experienced Adverse Drug Reactions **Time Frame:** Up to 72 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Adult participants ≥ 18 years of age. * Participants or participant's legally authorized representative has provided informed consent to participate in this study. * Participants who meet one of the following: * Prescribed Repatha® in addition to an existing SOC treatment according to local guidelines and approved label. OR * Already received SOC treatment prior to enrollment. * Participants with ANY of the following. * Diagnosis of MI OR stroke within 2 years before enrollment. * 2 MIs OR ≥ 2 strokes OR (≥ 1 MI AND ≥ 1 stroke) any time before enrollment. * Diagnosis of (MI OR stroke) AND diabetes. * Diagnosis of (MI OR stroke) AND documented multivessel disease (defined as \> 50% stenosis of ≥ 2 major coronary arteries on coronary angiography or coronary artery contrast enhanced computed tomography). * Diagnosis of symptomatic peripheral arterial disease. * Most recent fasting LDL-C ≥ 70 mg/dL (≥ 1.8 mmol/L) or nonhigh-density lipoprotein cholesterol (non-HDL-C) ≥ 100 mg/dL (≥ 2.6 mmol/L) within 6 months prior to enrollment. * Most recent fasting triglycerides ≤ 400 mg/dL (≤ 4.5 mmol/L) within 6 months prior to enrollment. Exclusion Criteria: * Stroke within past 1 month. * Known hemorrhagic stroke at any time. * Stroke due to thromboembolic event. * Any prior use of Repatha® or other proprotein convertase subtilisin/kexin type 9 inhibition treatments within past 6 months prior to enrollment. * Participants currently enrolled in another study involving any investigational procedure, device or drug. * Participants prescribed Repatha® with a history of severe hypersensitivity or allergy to any subsidiary. **Maximum Age:** 150 Years **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** The study population comprises Chinese participants with established ASCVD treated with Repatha® in combination with SOC or with SOC alone in a clinical setting which includes any primary through tertiary healthcare setting where Repatha® is prescribed. ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Amgen Call Center **Phone:** 866-572-6436 **Role:** CONTACT #### Locations **Location 1:** **City:** Beijing **Country:** China **Facility:** China Japan Friendship Hospital **State:** Beijing **Status:** RECRUITING **Zip:** 100029 **Location 2:** **City:** Beijing **Country:** China **Facility:** Peking University First Hospital **State:** Beijing **Status:** RECRUITING **Zip:** 100034 **Location 3:** **City:** Beijing **Country:** China **Facility:** Beijing Haidian Hospital **State:** Beijing **Status:** RECRUITING **Zip:** 100086 **Location 4:** **City:** Beijing **Country:** China **Facility:** Peking University Third Hospital **State:** Beijing **Status:** RECRUITING **Zip:** 100191 **Location 5:** **City:** Beijing **Country:** China **Facility:** Beijing Hospital **State:** Beijing **Status:** RECRUITING **Zip:** 100730 **Location 6:** **City:** Chongqing **Country:** China **Facility:** Chongqing Emergency Medical Center **State:** Chongqing **Status:** RECRUITING **Zip:** 400014 **Location 7:** **City:** Chongqing **Country:** China **Facility:** Chongqing General Hospital **State:** Chongqing **Status:** RECRUITING **Zip:** 401121 **Location 8:** **City:** Dong Guan **Country:** China **Facility:** Dongguan Songshan Lake Central Hospital **State:** Guangdong **Status:** RECRUITING **Zip:** 523127 **Location 9:** **City:** Foshan **Country:** China **Facility:** The First Peoples Hospital of Foshan **State:** Guangdong **Status:** RECRUITING **Zip:** 528000 **Location 10:** **City:** Guangzhou **Country:** China **Facility:** The First Affiliated Hospital of Shantou University Medical College **State:** Guangdong **Status:** RECRUITING **Zip:** 510280 **Location 11:** **City:** Guangzhou **Country:** China **Facility:** Zhujiang Hospital of Southern Medical University **State:** Guangdong **Status:** RECRUITING **Zip:** 510280 **Location 12:** **City:** Guangzhou **Country:** China **Facility:** Nanfang Hospital, Southern Medical University **State:** Guangdong **Status:** RECRUITING **Zip:** 510515 **Location 13:** **City:** Guangzhou **Country:** China **Facility:** The First Affiliated Hospital of Jinan University **State:** Guangdong **Status:** RECRUITING **Zip:** 510630 **Location 14:** **City:** Guangzhou **Country:** China **Facility:** The Third Affiliated Hospital of Sun Yat-sen University **State:** Guangdong **Status:** RECRUITING **Zip:** 510630 **Location 15:** **City:** Guangzhou **Country:** China **Facility:** The Sixth Affiliated Hospital Sun Yat-sen University **State:** Guangdong **Status:** RECRUITING **Zip:** 510655 **Location 16:** **City:** Huizhou **Country:** China **Facility:** The Third Peoples Hospital of Huizhou City **State:** Guangdong **Status:** RECRUITING **Zip:** 516002 **Location 17:** **City:** Zhuhai **Country:** China **Facility:** Zhuhai Peoples Hospital **State:** Guangdong **Status:** RECRUITING **Zip:** 519050 **Location 18:** **City:** Yulin **Country:** China **Facility:** The First Peoples Hospital of Yulin **State:** Guangxi **Status:** RECRUITING **Zip:** 537006 **Location 19:** **City:** Yulin **Country:** China **Facility:** The Second Affiliated Hospital of Guilin Medical University **State:** Guangxi **Status:** RECRUITING **Zip:** 541199 **Location 20:** **City:** Guiyang **Country:** China **Facility:** The Affiliated Hospital of Guizhou Medical University **State:** Guizhou **Status:** RECRUITING **Zip:** 550001 **Location 21:** **City:** Cangzhou **Country:** China **Facility:** Cangzhou Central Hospital **State:** Hebei **Status:** RECRUITING **Zip:** 061001 **Location 22:** **City:** Tangshan **Country:** China **Facility:** Tangshan Gongren Hosipital **State:** Hebei **Status:** RECRUITING **Zip:** 063000 **Location 23:** **City:** Haerbin **Country:** China **Facility:** Heilongjiang Provincial Hospital **State:** Heilongjiang **Status:** RECRUITING **Zip:** 150036 **Location 24:** **City:** Kaifeng **Country:** China **Facility:** Huaihe Hospital of Henan University **State:** Henan **Status:** RECRUITING **Zip:** 475000 **Location 25:** **City:** Nanyang **Country:** China **Facility:** Nanyang Central Hospital **State:** Henan **Status:** RECRUITING **Zip:** 473000 **Location 26:** **City:** Zhengzhou **Country:** China **Facility:** The Seventh Peoples Hospital of Zhengzhou **State:** Henan **Status:** RECRUITING **Zip:** 450016 **Location 27:** **City:** Zhengzhou **Country:** China **Facility:** The First Affiliated hospital of Zhengzhou University **State:** Henan **Status:** RECRUITING **Zip:** 450052 **Location 28:** **City:** Wuhan **Country:** China **Facility:** The Central Hospital of Wuhan **State:** Hubei **Status:** RECRUITING **Zip:** 430014 **Location 29:** **City:** Wuhan **Country:** China **Facility:** Union Hospital Tongji Medical College Huazhong University of science and Technology **State:** Hubei **Status:** RECRUITING **Zip:** 430022 **Location 30:** **City:** Wuhan **Country:** China **Facility:** Wuhan No 1 Hospital **State:** Hubei **Status:** RECRUITING **Zip:** 430022 **Location 31:** **City:** Wuhan **Country:** China **Facility:** Tongji Hospital, Tongji Medical college, Huazhong University of Science and Technology **State:** Hubei **Status:** RECRUITING **Zip:** 430030 **Location 32:** **City:** Wuhan **Country:** China **Facility:** Wuhan Third Hospital-Tongren Hospital Of Wuhan University **State:** Hubei **Status:** RECRUITING **Zip:** 430060 **Location 33:** **City:** Changsha **Country:** China **Facility:** Hunan Provincial Peoples Hospital **State:** Hunan **Status:** RECRUITING **Zip:** 410005 **Location 34:** **City:** Changshu **Country:** China **Facility:** Changshu No1 Peoples Hospital **State:** Jiangsu **Status:** RECRUITING **Zip:** 215500 **Location 35:** **City:** Changshu **Country:** China **Facility:** Changshu NO2 Peoples Hospital **State:** Jiangsu **Status:** RECRUITING **Zip:** 215523 **Location 36:** **City:** Nantong **Country:** China **Facility:** Nantong First Peoples Hospital **State:** Jiangsu **Status:** RECRUITING **Zip:** 226000 **Location 37:** **City:** Suzhou **Country:** China **Facility:** The first peoples hospital of Kunshan **State:** Jiangsu **Status:** RECRUITING **Zip:** 215300 **Location 38:** **City:** Wuxi **Country:** China **Facility:** Wuxi Peoples Hospital **State:** Jiangsu **Status:** RECRUITING **Zip:** 214023 **Location 39:** **City:** Xuzhou **Country:** China **Facility:** The Affiliated Hospital of Xuzhou Medical University **State:** Jiangsu **Status:** RECRUITING **Zip:** 221006 **Location 40:** **City:** Nanchang **Country:** China **Facility:** Jiangxi Provincial People Hospital **State:** Jiangxi **Status:** RECRUITING **Zip:** 330006 **Location 41:** **City:** Nanchang **Country:** China **Facility:** The first affiliated Hospital of Nanchang University **State:** Jiangxi **Status:** RECRUITING **Zip:** 330006 **Location 42:** **City:** Nanchang **Country:** China **Facility:** The First Hospital of Nanchang **State:** Jiangxi **Status:** RECRUITING **Zip:** 330008 **Location 43:** **City:** Changchun **Country:** China **Facility:** The Second Hospital of Jilin University **State:** Jilin **Status:** RECRUITING **Zip:** 13041 **Location 44:** **City:** Dalian **Country:** China **Facility:** Dalian Municipal Central Hospital Affiliated Of Dalian Medical University **State:** Liaoning **Status:** RECRUITING **Zip:** 116000 **Location 45:** **City:** Dalian **Country:** China **Facility:** The First Affiliated Hospital of Dalian Medical University **State:** Liaoning **Status:** RECRUITING **Zip:** 116000 **Location 46:** **City:** Dalian **Country:** China **Facility:** The Second Hospital of Dalian Medical University **State:** Liaoning **Status:** RECRUITING **Zip:** 116027 **Location 47:** **City:** Jinzhou **Country:** China **Facility:** The First Affiliated Hospital of Jinzhou Medical University **State:** Liaoning **Status:** RECRUITING **Zip:** 121000 **Location 48:** **City:** Shenyang **Country:** China **Facility:** General Hospital of Northern Theater Command **State:** Liaoning **Status:** RECRUITING **Zip:** 110016 **Location 49:** **City:** Shenyang **Country:** China **Facility:** The Forth Affiliated Hospital of China Medical University **State:** Liaoning **Status:** RECRUITING **Zip:** 110032 **Location 50:** **City:** Xi'an **Country:** China **Facility:** Tangdu Hospital,Air Force Military Medical University **State:** Shaanxi **Status:** RECRUITING **Zip:** 710038 **Location 51:** **City:** Xi'an **Country:** China **Facility:** Shaanxi Provincial Peoples Hospital **State:** Shaanxi **Status:** RECRUITING **Zip:** 710068 **Location 52:** **City:** Jinan **Country:** China **Facility:** Qilu Hospital of Shandong University **State:** Shandong **Status:** RECRUITING **Zip:** 250012 **Location 53:** **City:** Jinan **Country:** China **Facility:** Jinan Central Hospital **State:** Shandong **Status:** RECRUITING **Zip:** 250013 **Location 54:** **City:** Jinan **Country:** China **Facility:** Shandong Qianfoshan Hospital **State:** Shandong **Status:** RECRUITING **Zip:** 250014 **Location 55:** **City:** Jinan **Country:** China **Facility:** Shandong Provincial Hospital **State:** Shandong **Status:** RECRUITING **Zip:** 250021 **Location 56:** **City:** Jining **Country:** China **Facility:** Affiliated hospital of Jining Medical University **State:** Shandong **Status:** RECRUITING **Zip:** 272009 **Location 57:** **City:** Zibo **Country:** China **Facility:** Zibo central Hospital **State:** Shandong **Status:** RECRUITING **Zip:** 255030 **Location 58:** **City:** Shanghai **Country:** China **Facility:** Xinhua Hospital Affiliated To Shanghai Jiaotong University School of Medicine **State:** Shanghai **Status:** RECRUITING **Zip:** 200092 **Location 59:** **City:** Taiyuan **Country:** China **Facility:** The Second Hospital of Shanxi Medical University **State:** Shanxi **Status:** RECRUITING **Zip:** 030001 **Location 60:** **City:** XI An **Country:** China **Facility:** The First Affiliated Hospital of Xi An Jiaotong University **State:** Shanxi **Status:** RECRUITING **Zip:** 710061 **Location 61:** **City:** Chengdu **Country:** China **Facility:** Chengdu First Peoples Hospital **State:** Sichuan **Status:** RECRUITING **Zip:** 610041 **Location 62:** **City:** Chengdu **Country:** China **Facility:** Chengdu Third Peoples Hospital **State:** Sichuan **Status:** RECRUITING **Zip:** 61031 **Location 63:** **City:** Tianjin **Country:** China **Facility:** Tianjin Peoples Hospital **State:** Tianjin **Status:** RECRUITING **Zip:** 300120 **Location 64:** **City:** Tianjin **Country:** China **Facility:** Tianjin Chest Hospital **State:** Tianjin **Status:** RECRUITING **Zip:** 300222 **Location 65:** **City:** Urumqi **Country:** China **Facility:** The First Affiliated Hospital of Xinjiang Medical University **State:** Xinjiang **Status:** RECRUITING **Zip:** 830054 **Location 66:** **City:** Kunming **Country:** China **Facility:** Yanan Hospital of Kunming City **State:** Yunnan **Status:** RECRUITING **Zip:** 473009 **Location 67:** **City:** Kunming **Country:** China **Facility:** First Affiliated Hospital of Kunming Medical University **State:** Yunnan **Status:** RECRUITING **Zip:** 650032 **Location 68:** **City:** Kunming **Country:** China **Facility:** Yunnan First Peoples Hospital **State:** Yunnan **Status:** RECRUITING **Zip:** 650032 **Location 69:** **City:** Hangzhou **Country:** China **Facility:** Sir Run Run Shaw Hospital Affiliated to Zhejiang University School of Medicine **State:** Zhejiang **Status:** RECRUITING **Zip:** 310016 **Location 70:** **City:** Hangzhou **Country:** China **Facility:** the First Peoples Hospital of Xiaoshan District Hangzhou **State:** Zhejiang **Status:** RECRUITING **Zip:** 311201 **Location 71:** **City:** Huzhou **Country:** China **Facility:** Huzhou Central Hospital **State:** Zhejiang **Status:** RECRUITING **Zip:** 310005 **Location 72:** **City:** Huzhou **Country:** China **Facility:** The First Peoples Hospital of Huzhou **State:** Zhejiang **Status:** RECRUITING **Zip:** 313000 **Location 73:** **City:** Jiaxing **Country:** China **Facility:** Jiaxing Second Hospital **State:** Zhejiang **Status:** RECRUITING **Zip:** 314000 **Location 74:** **City:** Ningbo **Country:** China **Facility:** The first affiliated hospital of Ningbo university **State:** Zhejiang **Status:** RECRUITING **Zip:** 315010 **Location 75:** **City:** Ningbo **Country:** China **Facility:** Ningbo medical center lihuili hospital **State:** Zhejiang **Status:** RECRUITING **Zip:** 315046 **Location 76:** **City:** Shaoxing **Country:** China **Facility:** Shaoxing Second Hospital **State:** Zhejiang **Status:** RECRUITING **Zip:** 312000 **Location 77:** **City:** Beijing **Country:** China **Facility:** Beijing Anzhen Hospital, Capital Medical University **Status:** RECRUITING **Zip:** 100029 **Location 78:** **City:** Shanghai **Country:** China **Facility:** Tongji Hospital of Tongji University **Status:** RECRUITING **Zip:** 200065 #### Overall Officials **Official 1:** **Affiliation:** Amgen **Name:** MD **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **Access Criteria:** Qualified researchers may submit a request containing the research objectives, the Amgen product(s) and Amgen study/studies in scope, endpoints/outcomes of interest, statistical analysis plan, data requirements, publication plan, and qualifications of the researcher(s). In general, Amgen does not grant external requests for individual patient data for the purpose of re-evaluating safety and efficacy issues already addressed in the product labelling. Requests are reviewed by a committee of internal advisors. If not approved, a Data Sharing Independent Review Panel will arbitrate and make the final decision. Upon approval, information necessary to address the research question will be provided under the terms of a data sharing agreement. This may include anonymized individual patient data and/or available supporting documents, containing fragments of analysis code where provided in analysis specifications. Further details are available at the URL below. **Description:** De-identified individual patient data for variables necessary to address the specific research question in an approved data sharing request. **Info Types:** - STUDY_PROTOCOL - SAP - ICF - CSR **IPD Sharing:** YES **Time Frame:** Data sharing requests relating to this study will be considered beginning 18 months after the study has ended and either 1) the product and indication have been granted marketing authorization in both the US and Europe or 2) clinical development for the product and/or indication discontinues and the data will not be submitted to regulatory authorities. There is no end date for eligibility to submit a data sharing request for this study. **URL:** http://www.amgen.com/datasharing ### References Module #### See Also Links **Label:** AmgenTrials clinical trials website **URL:** http://www.amgentrials.com ## Derived Section ### Condition Browse Module - Ancestors - ID: D000001161 - Term: Arteriosclerosis - ID: D000001157 - Term: Arterial Occlusive Diseases - ID: D000014652 - Term: Vascular 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: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M22306 - Name: Stroke - Relevance: LOW - As Found: Unknown - ID: M12155 - Name: Myocardial Infarction - Relevance: LOW - As Found: Unknown - ID: M10282 - Name: Infarction - Relevance: LOW - As Found: Unknown - ID: M26188 - Name: Atherosclerosis - Relevance: HIGH - As Found: Atherosclerotic Cardiovascular Disease - ID: M4117 - Name: Angina Pectoris - Relevance: LOW - As Found: Unknown - ID: M6845 - Name: Death - Relevance: LOW - As Found: Unknown - ID: M4119 - Name: Angina, Unstable - 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: D000002318 - Term: Cardiovascular Diseases - ID: D000050197 - Term: Atherosclerosis ### Intervention Browse Module - Browse Branches - Abbrev: Lipd - Name: Lipid Regulating Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M348166 - Name: Evolocumab - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04213079 **Brief Title:** Treatments of Mal de Debarquement Syndrome (MdDS) by Habituation of Velocity Storage **Official Title:** Treatments of Mal de Debarquement Syndrome (MdDS) by Habituation of Velocity Storage #### Organization Study ID Info **ID:** GCO-19-0348 #### Organization **Class:** OTHER **Full Name:** Icahn School of Medicine at Mount Sinai #### Secondary ID Infos **ID:** 1R21DC018390-01 **Link:** https://reporter.nih.gov/quickSearch/1R21DC018390-01 **Type:** NIH ### Status Module #### Completion Date **Date:** 2022-11-30 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2024-01-03 **Type:** ACTUAL **Last Update Submit Date:** 2023-12-12 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2022-11-30 **Type:** ACTUAL #### Results First Post Date **Date:** 2024-01-03 **Type:** ACTUAL **Results First Submit Date:** 2023-11-15 **Results First Submit QC Date:** 2023-12-12 #### Start Date **Date:** 2020-06-15 **Type:** ACTUAL **Status Verified Date:** 2023-12 #### Study First Post Date **Date:** 2019-12-30 **Type:** ACTUAL **Study First Submit Date:** 2019-12-23 **Study First Submit QC Date:** 2019-12-27 ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** National Institute on Deafness and Other Communication Disorders (NIDCD) #### Lead Sponsor **Class:** OTHER **Name:** Icahn School of Medicine at Mount Sinai #### Responsible Party **Investigator Affiliation:** Icahn School of Medicine at Mount Sinai **Investigator Full Name:** Sergei Yakushin **Investigator Title:** Associate Professor, Neurology **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** True **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Mal de Debarquement Syndrome (MdDS) is an under-recognized but nevertheless common balance disorder, which in most cases occurs after exposure to prolonged passive motion. The current treatment approaches focus on reducing symptoms, but they can be retriggered. This project aims to shift the focus of MdDS treatment to permanently eliminating the symptom trigger while also minimizing symptoms. **Detailed Description:** Mal de Debarquement Syndrome (MdDS) is an under-recognized but nevertheless common balance disorder, primarily manifested by constant self-motion sensations consisting of rocking/swaying or gravitational pull of the body, which are accompanied by fatigue, migraine, hypersensitivity to light/noise/crowds, visually induced dizziness, and cognitive dysfunctions. As the name implies ("disembarkation sickness"), in most cases MdDS occurs after exposure to prolonged passive motion, specified as motion-triggered (MT) MdDS. However, the symptoms of MdDS can also occur without a motion trigger, termed as spontaneous MdDS. MdDS is debilitating and entails various mental health issues, such as suicidal thoughts, depression, and anxiety. Treatments for this disorder are still limited, as the specific underlying pathophysiology remains unclear. Recently, the team developed the first treatment method that can safely and effectively ease MdDS symptoms in the majority of patients via readaptation of the vestibulo-ocular reflex (VOR). The hypothesis underlying this treatment is that MdDS is caused by maladaptation of the functional component of the VOR called velocity storage, whose readaptation can be stimulated by exposure to whole-field visual motion coupled with head tilts. Over the past several years, more than 500 patients from around the world have been treated with this method. The success rate immediately after this treatment is 75% for MT MdDS, but some patients report return of symptoms after subsequent flights or prolonged car rides. Thus, the effectiveness of the current MdDS treatment protocol can depend on a serious practical limitation of needing to permanently avoid transportation. Building on the previous hypothesis of velocity storage maladaptation, the study team currently hypothesizes that another method, based on the reduction (habituation) of the velocity storage, can also resolve MdDS symptoms. Velocity storage can be greatly habituated within 4-5 days using a protocol previously developed in the study team's laboratory to reduce susceptibility to motion sickness. Preliminary data support the application of this protocol to MdDS. Moreover, since animal-based research suggests that velocity storage habituation is permanently retained, the study team further hypothesizes that this new treatment method yields robust long-term outcomes. In this project, 50 MT MdDS patients with otherwise normal vestibular and neurological functions will be randomly assigned into two groups, one to be treated by velocity storage habituation and the other by readaptation. Patients will be followed up for 6 months. Based on the preliminary data, the study team expects both groups to yield similar initial success rates for symptom improvement. However, the study team expects the group undergoing the habituation protocol to better retain the initial treatment impact in the long term. This project will significantly impact the MdDS treatment practice. The current approach focuses on reducing symptoms, but they can be retriggered by another prolonged exposure to passive motion. The habituation approach on the other hand focuses on permanently minimizing the symptom trigger while also minimizing symptoms. This project will also increase the current understanding of recurrent MdDS. ### Conditions Module **Conditions:** - Mal de Debarquement Syndrome (MdDS) **Keywords:** - Mal de Debarquement Syndrome - Motion Sickness - body rocking - body swaying - Habituation of velocity storage ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** In this project, 50 motion triggered MdDS patients with otherwise normal vestibular and neurological functions will be randomly assigned into two groups, one to be treated by velocity storage habituation and the other by readaptation. Patients will be followed up for 6 months. Based on the preliminary data, we expect both groups to yield similar initial success rates for symptom improvement. ##### Masking Info **Masking:** SINGLE **Who Masked:** - PARTICIPANT **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 47 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Treatment by re-adaptation of the vestibulo-ocular reflex (VOR) for participants with motion triggered MdDS **Intervention Names:** - Device: re-adaptation of the vestibulo-ocular reflex **Label:** Vestibulo-ocular reflex (VOR) **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants with motion triggered MdDS **Intervention Names:** - Device: Habituation of velocity storage of the vestibulo-ocular reflex **Label:** Habituation of velocity storage **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Vestibulo-ocular reflex (VOR) **Description:** The VOR will be readapted by activating velocity storage with full-field optokinetic motion at 5°/s in a set direction while the head is oscillated with a set frequency and direction. The readaptation training will be conducted in repeated modules, each lasting for 1-5 min. The expected duration of daily sessions varies from 30 to 90 min. A day's session will be terminated if patient no longer feel symptoms of MdDS. **Name:** re-adaptation of the vestibulo-ocular reflex **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - Habituation of velocity storage **Description:** The central (velocity storage) time constant will be reduced by inducing cancellation of two velocity storage-mediated responses: OKN and the VOR. Sinusoidal rotation at 0.017 Hz (1 revolution/min) in darkness advances the slow phase eye velocity of the VOR by 32º. In contrast, the OKN at this frequency has no phase advancement. Thus, to counteract the VOR by OKN, the optokinetic stimulus should be set to 32º phase advance the out of phased head rotation stimulus. Since the conflict stimulus is expected to be overwhelming to patients at higher chair velocities, subjects will be first trained with a 10°/s stimulus. In a previous study, no complaints were reported when subjects were tested at such low velocities. Preliminary testing show signs of symptom improvement when the peak velocity reached 30°/s to 40°/s. **Name:** Habituation of velocity storage of the vestibulo-ocular reflex **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** The overall severity of MdDS-related symptoms was subjectively reported on a single 11-point scale of 0-10, where the score 0 indicated no symptoms and 10 the most difficult of combined symptoms that the patient subject could imagine. Higher score indicates poorer health outcomes. Among the symptoms to consider were: brain fog, head pressure, fullness of ear, heavy head, headache, nausea, blurry vision, fatigue, sensitivity to fluorescent lights, scrolling of computer screen, sensitivity to smell, sensitivity to noise, walking on trampoline, sensation of gravitational pull up or down. Subjects were trained to estimate the level of symptoms to minimize inconsistency. **Measure:** Subjective Symptoms Self-report of Overall Severity **Time Frame:** During treatment (Day 1), Day 5, and 6 month follow up #### Secondary Outcomes **Description:** Visual Vertigo Analogue Scale. There are 9 separate visual analogue scales to rate intensity of visual vertigo provoking situation. Each scale is on a 0-10 cm line. Full scale from 0-10. Higher score represents more dizziness. **Measure:** Visual Vertigo Analogue Scale (VVAS) **Time Frame:** Baseline and 6 month follow up **Description:** Physical, emotional, and functional disability related to MdDS will be assessed with DHI. DHI is a 25-item self report questionnaire, total score range from 0 to 100, with higher score indicating more perceived disability. **Measure:** Dizziness Handicap Inventory (DHI) Questionnaire **Time Frame:** Baseline and 6 month follow up **Description:** The vestibulo-ocular reflex (VOR) is a class of reflex eye movement that counters head movement to stabilize vision. A perfect stabilization occurs when the velocity of the retinal image slip is zero, i.e. when the ratio, or gain, of the eye rotation speed to the head rotation speed is one. The VOR is a fast reflex whose direct pathway consists of a three-neuron arc, but also has parallel, indirect pathways that allow integration of signals from the peripheral vestibular organs with those of other sensory modalities such as vision and proprioception to modulate the eye movement response. The gain of the direct VOR pathway is the ratio of the eye rotation speed to the head rotation speed at the onset of head rotation, and is a unitless measure. **Measure:** VOR Direct Pathway Gain **Time Frame:** Baseline and Day 5 **Description:** The velocity storage mechanism is an indirect component of the VOR that facilitates the reflex by storing and releasing signals related to head rotation, for example by prolonging the eye movement response beyond the peripheral vestibular activity during head movement and generating similar eye movement response to rotational cues provided by other sensory modalities. The time constant of this indirect VOR pathway is the rate of charging/discharging in the exponential ideation of its behavior, measured in seconds, estimated from the profile of eye rotation speed during prolonged whole-body rotation that is the combination of the contributions from the direct and indirect pathways. **Measure:** VOR Indirect Pathway Time Constant **Time Frame:** Baseline and Day 5 **Description:** The gain of the indirect VOR pathway is the term that determines the contribution of velocity storage to the profile of eye rotation speed during prolonged whole-body rotation. The measure is normalized to the head rotation velocity and is thus unitless. **Measure:** VOR Indirect Pathway Coupling Gain **Time Frame:** Baseline and Day 5 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: - Age 18-78. Exclusion Criteria: - Patient with serious spinal, neck and legs injuries will be excluded, since postural ability is essential for both treatments. **Maximum Age:** 78 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:** Vestibular Testing Center **State:** New York **Zip:** 10029 #### Overall Officials **Official 1:** **Affiliation:** Icahn School of Medicine at Mount Sinai **Name:** Sergei Yakushin, PhD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Access Criteria:** Researchers who provide a methodologically sound proposal to achieve aims in the approved proposal. **Description:** Individual participant data that underlie the results reported in this article, after deidentification (text, tables, figures, and appendices). **Info Types:** - STUDY_PROTOCOL **IPD Sharing:** YES **Time Frame:** Beginning 3 months and ending 5 years following article publication. ### References Module #### Available IPDs **Type:** Study Protocol **URL:** http://mdds.nyc/ #### References **Citation:** Dai M, Cohen B, Cho C, Shin S, Yakushin SB. Treatment of the Mal de Debarquement Syndrome: A 1-Year Follow-up. Front Neurol. 2017 May 5;8:175. doi: 10.3389/fneur.2017.00175. eCollection 2017. **PMID:** 28529496 **Citation:** Yakushin SB, Palla A, Haslwanter T, Bockisch CJ, Straumann D. Dependence of adaptation of the human vertical angular vestibulo-ocular reflex on gravity. Exp Brain Res. 2003 Sep;152(1):137-42. doi: 10.1007/s00221-003-1543-0. Epub 2003 Jul 17. **PMID:** 12879171 **Citation:** Eron JN, Cohen B, Raphan T, Yakushin SB. Adaptation of orientation of central otolith-only neurons. Ann N Y Acad Sci. 2009 May;1164:367-71. doi: 10.1111/j.1749-6632.2009.03848.x. **PMID:** 19645928 **Citation:** Yakushin SB, Xiang Y, Cohen B, Raphan T. Dependence of the roll angular vestibuloocular reflex (aVOR) on gravity. J Neurophysiol. 2009 Nov;102(5):2616-26. doi: 10.1152/jn.00245.2009. Epub 2009 Aug 19. **PMID:** 19692515 **Citation:** Kolesnikova OV, Raphan T, Cohen B, Yakushin SB. Orientation adaptation of eye movement-related vestibular neurons due to prolonged head tilt. Ann N Y Acad Sci. 2011 Sep;1233:214-8. doi: 10.1111/j.1749-6632.2011.06176.x. **PMID:** 21950996 **Citation:** Mucci V, Canceri JM, Brown R, Dai M, Yakushin SB, Watson S, Van Ombergen A, Jacquemyn Y, Fahey P, Van de Heyning PH, Wuyts F, Browne CJ. Mal de Debarquement Syndrome: A Retrospective Online Questionnaire on the Influences of Gonadal Hormones in Relation to Onset and Symptom Fluctuation. Front Neurol. 2018 May 24;9:362. doi: 10.3389/fneur.2018.00362. eCollection 2018. **PMID:** 29910765 **Citation:** Mucci V, Canceri JM, Brown R, Dai M, Yakushin S, Watson S, Van Ombergen A, Topsakal V, Van de Heyning PH, Wuyts FL, Browne CJ. Mal de Debarquement Syndrome: a survey on subtypes, misdiagnoses, onset and associated psychological features. J Neurol. 2018 Mar;265(3):486-499. doi: 10.1007/s00415-017-8725-3. Epub 2018 Jan 5. **PMID:** 29305644 **Citation:** Dai M, Cohen B, Smouha E, Cho C. Readaptation of the vestibulo-ocular reflex relieves the mal de debarquement syndrome. Front Neurol. 2014 Jul 15;5:124. doi: 10.3389/fneur.2014.00124. eCollection 2014. **PMID:** 25076935 **Citation:** Cohen B, Dai M, Yakushin SB, Cho C. The neural basis of motion sickness. J Neurophysiol. 2019 Mar 1;121(3):973-982. doi: 10.1152/jn.00674.2018. Epub 2019 Jan 30. **PMID:** 30699041 **Citation:** Dai M, Raphan T, Cohen B. Prolonged reduction of motion sickness sensitivity by visual-vestibular interaction. Exp Brain Res. 2011 May;210(3-4):503-13. doi: 10.1007/s00221-011-2548-8. Epub 2011 Feb 2. **PMID:** 21287155 **Citation:** Cohen B, Dai M, Yakushin SB, Raphan T. Baclofen, motion sickness susceptibility and the neural basis for velocity storage. Prog Brain Res. 2008;171:543-53. doi: 10.1016/S0079-6123(08)00677-8. **PMID:** 18718351 **Citation:** Cohen B, Yakushin SB, Cho C. Hypothesis: The Vestibular and Cerebellar Basis of the Mal de Debarquement Syndrome. Front Neurol. 2018 Feb 5;9:28. doi: 10.3389/fneur.2018.00028. eCollection 2018. **PMID:** 29459843 **Citation:** Yakushin SB, Raphan T, Cohen B. Coding of Velocity Storage in the Vestibular Nuclei. Front Neurol. 2017 Aug 16;8:386. doi: 10.3389/fneur.2017.00386. eCollection 2017. **PMID:** 28861030 **Citation:** Eron JN, Ogorodnikov D, Horn AKE, Yakushin SB. Adaptation of spatio-temporal convergent properties in central vestibular neurons in monkeys. Physiol Rep. 2018 Sep;6(17):e13750. doi: 10.14814/phy2.13750. **PMID:** 30178612 **Citation:** Eron JN, Cohen B, Raphan T, Yakushin SB. Adaptation of orientation vectors of otolith-related central vestibular neurons to gravity. J Neurophysiol. 2008 Sep;100(3):1686-90. doi: 10.1152/jn.90289.2008. Epub 2008 May 21. **PMID:** 18497367 #### See Also Links **Label:** Guidelines for patients coming for the treatment. Available treatment announcements **URL:** http://mdds.nyc/ ## Document Section ### Large Document Module #### Large Docs - Date: 2022-07-26 - Filename: Prot_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: False - Label: Study Protocol - Size: 333888 - Type Abbrev: Prot - Upload Date: 2023-11-15T15:23 - Date: 2021-08-24 - Filename: ICF_001.pdf - Has ICF: True - Has Protocol: False - Has SAP: False - Label: Informed Consent Form - Size: 390268 - Type Abbrev: ICF - Upload Date: 2023-11-15T15:24 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000004194 - Term: Disease - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M16355 - Name: Syndrome - Relevance: HIGH - As Found: Syndrome - ID: M12002 - Name: Motion Sickness - Relevance: LOW - As Found: Unknown - ID: T3569 - Name: Mal De Debarquement Syndrome - Relevance: HIGH - As Found: Mal de Debarquement Syndrome ### Condition Browse Module - Meshes - ID: D000013577 - Term: Syndrome ### Misc Info Module #### Submission Tracking ##### First MCP Info ###### Post Date - Date: 2023-12-07 - Type: ACTUAL - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Vestibulo-ocular Reflex (VOR) **Deaths Num At Risk:** 24 **Description:** Treatment by re-adaptation of the vestibulo-ocular reflex (VOR) for participants with motion triggered MdDS Re-adaptation of the vestibulo-ocular reflex: The VOR will be readapted by activating velocity storage with full-field optokinetic motion at 5°/s in a set direction while the head is oscillated with a set frequency and direction. The readaptation training will be conducted in repeated modules, each lasting for 1-5 min. The expected duration of daily sessions varies from 30 to 90 min. A day's session will be terminated if patient no longer feel symptoms of MdDS. **ID:** EG000 **Other Num at Risk:** 24 **Serious Number At Risk:** 24 **Title:** Vestibulo-ocular Reflex (VOR) **Group ID:** EG001 **Title:** Habituation of Velocity Storage **Deaths Num At Risk:** 21 **Description:** Participants with motion triggered MdDS Habituation of velocity storage of the vestibulo-ocular reflex: The central (velocity storage) time constant will be reduced by inducing cancellation of two velocity storage-mediated responses: OKN and the VOR. Sinusoidal rotation at 0.017 Hz (1 revolution/min) in darkness advances the slow phase eye velocity of the VOR by 32º. In contrast, the OKN at this frequency has no phase advancement. Thus, to counteract the VOR by OKN, the optokinetic stimulus should be set to 32º phase advance the out of phased head rotation stimulus. Since the conflict stimulus is expected to be overwhelming to patients at higher chair velocities, subjects will be first trained with a 10°/s stimulus. In a previous study, no complaints were reported when subjects were tested at such low velocities. Preliminary testing show signs of symptom improvement when the peak velocity reached 30°/s to 40°/s. **ID:** EG001 **Other Num at Risk:** 21 **Serious Number At Risk:** 21 **Title:** Habituation of Velocity Storage **Frequency Threshold:** 0 **Time Frame:** 6 months ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 24 **Group ID:** BG001 **Value:** 21 **Group ID:** BG002 **Value:** 45 **Units:** Participants ### Group **ID:** BG000 **Title:** Vestibulo-ocular Reflex (VOR) **Description:** Treatment by re-adaptation of the vestibulo-ocular reflex (VOR) for participants with motion triggered MdDS Re-adaptation of the vestibulo-ocular reflex: The VOR will be readapted by activating velocity storage with full-field optokinetic motion at 5°/s in a set direction while the head is oscillated with a set frequency and direction. The readaptation training will be conducted in repeated modules, each lasting for 1-5 min. The expected duration of daily sessions varies from 30 to 90 min. A day's session will be terminated if patient no longer feel symptoms of MdDS. ### Group **ID:** BG001 **Title:** Habituation of Velocity Storage **Description:** Participants with motion triggered MdDS Habituation of velocity storage of the vestibulo-ocular reflex: The central (velocity storage) time constant will be reduced by inducing cancellation of two velocity storage-mediated responses: OKN and the VOR. Sinusoidal rotation at 0.017 Hz (1 revolution/min) in darkness advances the slow phase eye velocity of the VOR by 32º. In contrast, the OKN at this frequency has no phase advancement. Thus, to counteract the VOR by OKN, the optokinetic stimulus should be set to 32º phase advance the out of phased head rotation stimulus. Since the conflict stimulus is expected to be overwhelming to patients at higher chair velocities, subjects will be first trained with a 10°/s stimulus. In a previous study, no complaints were reported when subjects were tested at such low velocities. Preliminary testing show signs of symptom improvement when the peak velocity reached 30°/s to 40°/s. ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 13.9 **Value:** 47.4 #### Measurement **Group ID:** BG001 **Spread:** 14.2 **Value:** 46.7 #### Measurement **Group ID:** BG002 **Spread:** 14.0 **Value:** 47.1 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 24 **Group ID:** BG001 **Value:** 21 **Group ID:** BG002 **Value:** 45 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 22 #### Measurement **Group ID:** BG001 **Value:** 16 #### Measurement **Group ID:** BG002 **Value:** 38 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 2 #### Measurement **Group ID:** BG001 **Value:** 5 #### Measurement **Group ID:** BG002 **Value:** 7 **Category Title:** Male #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 24 **Group ID:** BG001 **Value:** 21 **Group ID:** BG002 **Value:** 45 **Class Title:** ### Measure #### Measurement **Group ID:** BG002 **Value:** 0 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 0 **Group ID:** BG001 **Value:** 0 **Group ID:** BG002 **Value:** 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:** years ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Race and Ethnicity were not collected from any participant. **Title:** Race and Ethnicity Not Collected **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement **PI Sponsor Employee:** True ### Point of Contact **Email:** [email protected] **Organization:** Icahn School of Medicine at Mount Sinai **Phone:** 212-241-9349 **Title:** Sergei Yakushin ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ### Outcome Measure 4 ### Outcome Measure 5 ### Outcome Measure 6 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 1.9 - **Upper Limit:** - **Value:** 5.2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 1.9 - **Upper Limit:** - **Value:** 5.2 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 2.1 - **Upper Limit:** - **Value:** 3.2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 1.7 - **Upper Limit:** - **Value:** 2.1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 2.0 - **Upper Limit:** - **Value:** 2.9 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 2.2 - **Upper Limit:** - **Value:** 3.4 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 2.0 - **Upper Limit:** - **Value:** 4.8 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 2.3 - **Upper Limit:** - **Value:** 4.4 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 2.1 - **Upper Limit:** - **Value:** 2.5 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 2.2 - **Upper Limit:** - **Value:** 2.6 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 17.3 - **Upper Limit:** - **Value:** 52.1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 16.1 - **Upper Limit:** - **Value:** 47.2 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 19.5 - **Upper Limit:** - **Value:** 35.3 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 18.6 - **Upper Limit:** - **Value:** 32.1 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.13 - **Upper Limit:** - **Value:** 0.53 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.10 - **Upper Limit:** - **Value:** 0.42 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.14 - **Upper Limit:** - **Value:** 0.45 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.08 - **Upper Limit:** - **Value:** 0.47 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 3.9 - **Upper Limit:** - **Value:** 16.6 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 4.0 - **Upper Limit:** - **Value:** 15.0 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 5.4 - **Upper Limit:** - **Value:** 16.0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 4.5 - **Upper Limit:** - **Value:** 15.6 **Title:** #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.022 - **Upper Limit:** - **Value:** 0.102 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.030 - **Upper Limit:** - **Value:** 0.093 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.033 - **Upper Limit:** - **Value:** 0.080 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.027 - **Upper Limit:** - **Value:** 0.099 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** The overall severity of MdDS-related symptoms was subjectively reported on a single 11-point scale of 0-10, where the score 0 indicated no symptoms and 10 the most difficult of combined symptoms that the patient subject could imagine. Higher score indicates poorer health outcomes. Among the symptoms to consider were: brain fog, head pressure, fullness of ear, heavy head, headache, nausea, blurry vision, fatigue, sensitivity to fluorescent lights, scrolling of computer screen, sensitivity to smell, sensitivity to noise, walking on trampoline, sensation of gravitational pull up or down. Subjects were trained to estimate the level of symptoms to minimize inconsistency. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** During treatment (Day 1), Day 5, and 6 month follow up **Title:** Subjective Symptoms Self-report of Overall Severity **Type:** PRIMARY **Unit of Measure:** score on a scale ##### Group **Description:** Treatment by re-adaptation of the vestibulo-ocular reflex (VOR) for participants with motion triggered MdDS Re-adaptation of the vestibulo-ocular reflex: The VOR will be readapted by activating velocity storage with full-field optokinetic motion at 5°/s in a set direction while the head is oscillated with a set frequency and direction. The readaptation training will be conducted in repeated modules, each lasting for 1-5 min. The expected duration of daily sessions varies from 30 to 90 min. A day's session will be terminated if patient no longer feel symptoms of MdDS. **ID:** OG000 **Title:** Vestibulo-ocular Reflex (VOR) ##### Group **Description:** Participants with motion triggered MdDS Habituation of velocity storage of the vestibulo-ocular reflex: The central (velocity storage) time constant will be reduced by inducing cancellation of two velocity storage-mediated responses: OKN and the VOR. Sinusoidal rotation at 0.017 Hz (1 revolution/min) in darkness advances the slow phase eye velocity of the VOR by 32º. In contrast, the OKN at this frequency has no phase advancement. Thus, to counteract the VOR by OKN, the optokinetic stimulus should be set to 32º phase advance the out of phased head rotation stimulus. Since the conflict stimulus is expected to be overwhelming to patients at higher chair velocities, subjects will be first trained with a 10°/s stimulus. In a previous study, no complaints were reported when subjects were tested at such low velocities. Preliminary testing show signs of symptom improvement when the peak velocity reached 30°/s to 40°/s. **ID:** OG001 **Title:** Habituation of Velocity Storage #### Outcome Measure 2 **Description:** Visual Vertigo Analogue Scale. There are 9 separate visual analogue scales to rate intensity of visual vertigo provoking situation. Each scale is on a 0-10 cm line. Full scale from 0-10. Higher score represents more dizziness. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** Baseline and 6 month follow up **Title:** Visual Vertigo Analogue Scale (VVAS) **Type:** SECONDARY **Unit of Measure:** score on a scale ##### Group **Description:** Treatment by re-adaptation of the vestibulo-ocular reflex (VOR) for participants with motion triggered MdDS Re-adaptation of the vestibulo-ocular reflex: The VOR will be readapted by activating velocity storage with full-field optokinetic motion at 5°/s in a set direction while the head is oscillated with a set frequency and direction. The readaptation training will be conducted in repeated modules, each lasting for 1-5 min. The expected duration of daily sessions varies from 30 to 90 min. A day's session will be terminated if patient no longer feel symptoms of MdDS. **ID:** OG000 **Title:** Vestibulo-ocular Reflex (VOR) ##### Group **Description:** Participants with motion triggered MdDS Habituation of velocity storage of the vestibulo-ocular reflex: The central (velocity storage) time constant will be reduced by inducing cancellation of two velocity storage-mediated responses: OKN and the VOR. Sinusoidal rotation at 0.017 Hz (1 revolution/min) in darkness advances the slow phase eye velocity of the VOR by 32º. In contrast, the OKN at this frequency has no phase advancement. Thus, to counteract the VOR by OKN, the optokinetic stimulus should be set to 32º phase advance the out of phased head rotation stimulus. Since the conflict stimulus is expected to be overwhelming to patients at higher chair velocities, subjects will be first trained with a 10°/s stimulus. In a previous study, no complaints were reported when subjects were tested at such low velocities. Preliminary testing show signs of symptom improvement when the peak velocity reached 30°/s to 40°/s. **ID:** OG001 **Title:** Habituation of Velocity Storage #### Outcome Measure 3 **Description:** Physical, emotional, and functional disability related to MdDS will be assessed with DHI. DHI is a 25-item self report questionnaire, total score range from 0 to 100, with higher score indicating more perceived disability. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** Baseline and 6 month follow up **Title:** Dizziness Handicap Inventory (DHI) Questionnaire **Type:** SECONDARY **Unit of Measure:** score on a scale ##### Group **Description:** Treatment by re-adaptation of the vestibulo-ocular reflex (VOR) for participants with motion triggered MdDS Re-adaptation of the vestibulo-ocular reflex: The VOR will be readapted by activating velocity storage with full-field optokinetic motion at 5°/s in a set direction while the head is oscillated with a set frequency and direction. The readaptation training will be conducted in repeated modules, each lasting for 1-5 min. The expected duration of daily sessions varies from 30 to 90 min. A day's session will be terminated if patient no longer feel symptoms of MdDS. **ID:** OG000 **Title:** Vestibulo-ocular Reflex (VOR) ##### Group **Description:** Participants with motion triggered MdDS Habituation of velocity storage of the vestibulo-ocular reflex: The central (velocity storage) time constant will be reduced by inducing cancellation of two velocity storage-mediated responses: OKN and the VOR. Sinusoidal rotation at 0.017 Hz (1 revolution/min) in darkness advances the slow phase eye velocity of the VOR by 32º. In contrast, the OKN at this frequency has no phase advancement. Thus, to counteract the VOR by OKN, the optokinetic stimulus should be set to 32º phase advance the out of phased head rotation stimulus. Since the conflict stimulus is expected to be overwhelming to patients at higher chair velocities, subjects will be first trained with a 10°/s stimulus. In a previous study, no complaints were reported when subjects were tested at such low velocities. Preliminary testing show signs of symptom improvement when the peak velocity reached 30°/s to 40°/s. **ID:** OG001 **Title:** Habituation of Velocity Storage #### Outcome Measure 4 **Description:** The vestibulo-ocular reflex (VOR) is a class of reflex eye movement that counters head movement to stabilize vision. A perfect stabilization occurs when the velocity of the retinal image slip is zero, i.e. when the ratio, or gain, of the eye rotation speed to the head rotation speed is one. The VOR is a fast reflex whose direct pathway consists of a three-neuron arc, but also has parallel, indirect pathways that allow integration of signals from the peripheral vestibular organs with those of other sensory modalities such as vision and proprioception to modulate the eye movement response. The gain of the direct VOR pathway is the ratio of the eye rotation speed to the head rotation speed at the onset of head rotation, and is a unitless measure. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** Baseline and Day 5 **Title:** VOR Direct Pathway Gain **Type:** SECONDARY **Unit of Measure:** ratio ##### Group **Description:** Treatment by re-adaptation of the vestibulo-ocular reflex (VOR) for participants with motion triggered MdDS Re-adaptation of the vestibulo-ocular reflex: The VOR will be readapted by activating velocity storage with full-field optokinetic motion at 5°/s in a set direction while the head is oscillated with a set frequency and direction. The readaptation training will be conducted in repeated modules, each lasting for 1-5 min. The expected duration of daily sessions varies from 30 to 90 min. A day's session will be terminated if patient no longer feel symptoms of MdDS. **ID:** OG000 **Title:** Vestibulo-ocular Reflex (VOR) ##### Group **Description:** Participants with motion triggered MdDS Habituation of velocity storage of the vestibulo-ocular reflex: The central (velocity storage) time constant will be reduced by inducing cancellation of two velocity storage-mediated responses: OKN and the VOR. Sinusoidal rotation at 0.017 Hz (1 revolution/min) in darkness advances the slow phase eye velocity of the VOR by 32º. In contrast, the OKN at this frequency has no phase advancement. Thus, to counteract the VOR by OKN, the optokinetic stimulus should be set to 32º phase advance the out of phased head rotation stimulus. Since the conflict stimulus is expected to be overwhelming to patients at higher chair velocities, subjects will be first trained with a 10°/s stimulus. In a previous study, no complaints were reported when subjects were tested at such low velocities. Preliminary testing show signs of symptom improvement when the peak velocity reached 30°/s to 40°/s. **ID:** OG001 **Title:** Habituation of Velocity Storage #### Outcome Measure 5 **Description:** The velocity storage mechanism is an indirect component of the VOR that facilitates the reflex by storing and releasing signals related to head rotation, for example by prolonging the eye movement response beyond the peripheral vestibular activity during head movement and generating similar eye movement response to rotational cues provided by other sensory modalities. The time constant of this indirect VOR pathway is the rate of charging/discharging in the exponential ideation of its behavior, measured in seconds, estimated from the profile of eye rotation speed during prolonged whole-body rotation that is the combination of the contributions from the direct and indirect pathways. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** Baseline and Day 5 **Title:** VOR Indirect Pathway Time Constant **Type:** SECONDARY **Unit of Measure:** seconds ##### Group **Description:** Treatment by re-adaptation of the vestibulo-ocular reflex (VOR) for participants with motion triggered MdDS Re-adaptation of the vestibulo-ocular reflex: The VOR will be readapted by activating velocity storage with full-field optokinetic motion at 5°/s in a set direction while the head is oscillated with a set frequency and direction. The readaptation training will be conducted in repeated modules, each lasting for 1-5 min. The expected duration of daily sessions varies from 30 to 90 min. A day's session will be terminated if patient no longer feel symptoms of MdDS. **ID:** OG000 **Title:** Vestibulo-ocular Reflex (VOR) ##### Group **Description:** Participants with motion triggered MdDS Habituation of velocity storage of the vestibulo-ocular reflex: The central (velocity storage) time constant will be reduced by inducing cancellation of two velocity storage-mediated responses: OKN and the VOR. Sinusoidal rotation at 0.017 Hz (1 revolution/min) in darkness advances the slow phase eye velocity of the VOR by 32º. In contrast, the OKN at this frequency has no phase advancement. Thus, to counteract the VOR by OKN, the optokinetic stimulus should be set to 32º phase advance the out of phased head rotation stimulus. Since the conflict stimulus is expected to be overwhelming to patients at higher chair velocities, subjects will be first trained with a 10°/s stimulus. In a previous study, no complaints were reported when subjects were tested at such low velocities. Preliminary testing show signs of symptom improvement when the peak velocity reached 30°/s to 40°/s. **ID:** OG001 **Title:** Habituation of Velocity Storage #### Outcome Measure 6 **Description:** The gain of the indirect VOR pathway is the term that determines the contribution of velocity storage to the profile of eye rotation speed during prolonged whole-body rotation. The measure is normalized to the head rotation velocity and is thus unitless. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Reporting Status:** POSTED **Time Frame:** Baseline and Day 5 **Title:** VOR Indirect Pathway Coupling Gain **Type:** SECONDARY **Unit of Measure:** ratio ##### Group **Description:** Treatment by re-adaptation of the vestibulo-ocular reflex (VOR) for participants with motion triggered MdDS Re-adaptation of the vestibulo-ocular reflex: The VOR will be readapted by activating velocity storage with full-field optokinetic motion at 5°/s in a set direction while the head is oscillated with a set frequency and direction. The readaptation training will be conducted in repeated modules, each lasting for 1-5 min. The expected duration of daily sessions varies from 30 to 90 min. A day's session will be terminated if patient no longer feel symptoms of MdDS. **ID:** OG000 **Title:** Vestibulo-ocular Reflex (VOR) ##### Group **Description:** Participants with motion triggered MdDS Habituation of velocity storage of the vestibulo-ocular reflex: The central (velocity storage) time constant will be reduced by inducing cancellation of two velocity storage-mediated responses: OKN and the VOR. Sinusoidal rotation at 0.017 Hz (1 revolution/min) in darkness advances the slow phase eye velocity of the VOR by 32º. In contrast, the OKN at this frequency has no phase advancement. Thus, to counteract the VOR by OKN, the optokinetic stimulus should be set to 32º phase advance the out of phased head rotation stimulus. Since the conflict stimulus is expected to be overwhelming to patients at higher chair velocities, subjects will be first trained with a 10°/s stimulus. In a previous study, no complaints were reported when subjects were tested at such low velocities. Preliminary testing show signs of symptom improvement when the peak velocity reached 30°/s to 40°/s. **ID:** OG001 **Title:** Habituation of Velocity Storage ### Participant Flow Module #### Group **Description:** Treatment by re-adaptation of the vestibulo-ocular reflex (VOR) for participants with motion triggered MdDS Re-adaptation of the vestibulo-ocular reflex: The VOR will be readapted by activating velocity storage with full-field optokinetic motion at 5°/s in a set direction while the head is oscillated with a set frequency and direction. The readaptation training will be conducted in repeated modules, each lasting for 1-5 min. The expected duration of daily sessions varies from 30 to 90 min. A day's session will be terminated if patient no longer feel symptoms of MdDS. **ID:** FG000 **Title:** Vestibulo-ocular Reflex (VOR) #### Group **Description:** Participants with motion triggered MdDS Habituation of velocity storage of the vestibulo-ocular reflex: The central (velocity storage) time constant will be reduced by inducing cancellation of two velocity storage-mediated responses: OKN and the VOR. Sinusoidal rotation at 0.017 Hz (1 revolution/min) in darkness advances the slow phase eye velocity of the VOR by 32º. In contrast, the OKN at this frequency has no phase advancement. Thus, to counteract the VOR by OKN, the optokinetic stimulus should be set to 32º phase advance the out of phased head rotation stimulus. Since the conflict stimulus is expected to be overwhelming to patients at higher chair velocities, subjects will be first trained with a 10°/s stimulus. In a previous study, no complaints were reported when subjects were tested at such low velocities. Preliminary testing show signs of symptom improvement when the peak velocity reached 30°/s to 40°/s. **ID:** FG001 **Title:** Habituation of Velocity Storage #### Period **Title:** Overall Study ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 2 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 24 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 23 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 24 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 21 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 2 **Recruitment Details:** Recruitment from April 2020 -July 2022 with first enrollment in June 2020; Patient volunteers with MdDS were recruited through various sources of referral and announcements posted on the Internet, including ClinicalTrials.gov (NCT04213079). Applicants seeking treatment were screened with an intake form, and each candidate's diagnosis of MdDS with an associable motion trigger was confirmed by a board-certified physician through a telephone interview when necessary. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT01982279 **Brief Title:** Longitudinal Measurement of Caloric Compensation and Eating in the Absence of Hunger in French Preschool Children **Official Title:** One Year Change in Caloric Compensation and Eating in the Absence of Hunger in French Preschool Children #### Organization Study ID Info **ID:** HabEat WP31 Follow up #### Organization **Class:** OTHER **Full Name:** Centre des Sciences du Goût et de l'Alimentation #### Secondary ID Infos **ID:** INRA WP31 FU **Domain:** INRA **ID:** WP31.FU. INRA **Type:** OTHER ### Status Module #### Completion Date **Date:** 2012-06 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2013-11-13 **Type:** ESTIMATED **Last Update Submit Date:** 2013-11-12 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2011-03 **Type:** ACTUAL #### Start Date **Date:** 2011-02 **Status Verified Date:** 2013-11 #### Study First Post Date **Date:** 2013-11-13 **Type:** ESTIMATED **Study First Submit Date:** 2013-10-30 **Study First Submit QC Date:** 2013-11-12 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** European Union #### Lead Sponsor **Class:** OTHER **Name:** Centre des Sciences du Goût et de l'Alimentation #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** Objectives: The aims were to measure one year change in caloric compensation (CC) and eating in the absence of hunger (EAH) in 3 to 6 year-old French children in their regular eating context and to link these change with individual characteristics (adiposity, age, gender) and maternal feeding practices. Three series of measurements of caloric compensation (CC) and eating in the absence of hunger (EAH) were conducted: at baseline, and 3 (FU3M) and 15 (FU15M) months later. Each serie of measurement was composed of three consecutive weeks an identical lunch was served in four preschool canteens reaching 236 children. The first lunch was a control session. For the CC situation (week 2), thirty minutes before the lunch children were offered a preload (137 kcal). For the EAH situation (week 3), ten minutes after lunch children were exposed to palatable foods (430 kcal). Food intake was measured at the individual level. Maternal eating behaviour and feeding practices were measured by questionnaires. Child's height and weight were measured by a medical doctor. ### Conditions Module **Conditions:** - Healthy **Keywords:** - Caloric compensation - Eating in the absence of hunger - Preschool children - Maternal feeding practices - Maternal eating behavior - one year - children ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** CROSS_SECTIONAL #### Enrollment Info **Count:** 236 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Outcomes Module #### Primary Outcomes **Description:** To evaluate the change in caloric compensation across time, caloric compensation was measured at baseline, 3 and 15 months after baseline, in the same conditions. At each time point to assess caloric compensation, children were offered a chocolate bun 30 minutes before the lunch. The food intake at lunch was recorded. We calculated if children compensated the chocolate bun, by comparing energy intake at this lunch with energy intake from a control lunch which took place one week before. **Measure:** Change in caloric compensation in 15 months **Time Frame:** one week **Description:** We conducted a control lunch and recorded food intake of each child. This measurement was conducted a week before measuring caloric compensation. **Measure:** Control measurement of energy intake at baseline **Time Frame:** One week **Description:** In order to evaluate the change in eating in the absence of hunger across time, a measurement was conducted at baseline, 3 months and 15 months after baseline. To collect the measurement of eating in the absence of hunger, we offered palatable foods to children 10 minutes after their lunch. We recorded the energy intake from these post-meal foods. At each time point, this measurement was conducted one week after the measurement of caloric compensation. **Measure:** Change in eating in the absence of hunger in 15 months **Time Frame:** one week. **Description:** We conducted a control lunch and recorded food intake of each child. This measurement was conducted a week before measuring caloric compensation. **Measure:** Control measurement of energy intake three months after baseline **Time Frame:** One week **Description:** We conducted a control lunch and recorded food intake of each child. This measurement was conducted a week before measuring caloric compensation. **Measure:** Control measurement of energy intake fifteen months after baseline **Time Frame:** One week #### Secondary Outcomes **Description:** Using a validated questionnaire, we collected information about maternal feeding practices (Comprehensive Feeding Practices Questionnaire, Musher-Eizenman \& Holub, 2007) and eating behaviour (Dutch Eating Behaviour Questionnaire, Van Strien, 1986). **Measure:** Maternal feeding practices and eating behavior **Time Frame:** one month after inclusion ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * To be registered to the school canteen, to provide parental approval at inclusion and at the FU15M Exclusion Criteria: * Chronic disease or food allergies **Healthy Volunteers:** True **Maximum Age:** 7 Years **Minimum Age:** 2 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - CHILD **Study Population:** The population was composed of 236 children at inclusion. Among them, 98 participated in the three time of measurements of caloric compensation or eating in the absence of hunger. ### Contacts Locations Module #### Locations **Location 1:** **City:** Dijon **Country:** France **Facility:** Centre des Sciences du Goût et de l'Alimentation **Zip:** 21000 #### Overall Officials **Official 1:** **Affiliation:** Centre des Sciences du Goût et de l'Alimentation **Name:** Nicklaus Sophie, phD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### See Also Links **Label:** Web site for the main research project funded by the European Union **URL:** http://www.habeat.eu/ ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03212079 **Acronym:** PATH **Brief Title:** Physical Activities by Technology Help (PATH) **Official Title:** Novel Individualized Intervention for Behavioral Change Among High-Risk Group Cancer Survivors : Physical Activities by Technology Help (PATH) #### Organization Study ID Info **ID:** IRB00113882 #### Organization **Class:** OTHER **Full Name:** Johns Hopkins University ### Status Module #### Completion Date **Date:** 2018-06-30 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-08-07 **Type:** ACTUAL **Last Update Submit Date:** 2018-08-06 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2018-03-15 **Type:** ACTUAL #### Start Date **Date:** 2017-04-03 **Type:** ACTUAL **Status Verified Date:** 2018-08 #### Study First Post Date **Date:** 2017-07-11 **Type:** ACTUAL **Study First Submit Date:** 2017-04-04 **Study First Submit QC Date:** 2017-07-06 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Johns Hopkins 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:** PATH is a research study for cancer survivors to help participants to become more active. Studies suggested an association between inactivity and cancer. The investigators created new novel ways and technologies that may help participants to become more active. The three methods the investigators are studying are: 1) participant become active on her/his own; educational material will be provided; 2) working with a programmed health coach over the phone via text messages; and 3) using digital voice assist to help participant become more active. The digital voice assist will be delivered via Amazon Alexa on Echo speaker (it is the famous intelligent voice that you see in superball commercial by Alec Baldwin). This study is funded by the State of Maryland. **Detailed Description:** If the participant agree to be in this study, the investigators will ask the participant to do the following things: The participant will be asked questions about his/her physical activity (walking, running or other exercise) to determine if he/she are eligible to participate. If the participant is eligible and interested in participating, the investigators will ask hi/her to read and sign the consent form. After that, the investigators will randomly assign the participant to a research group (this method would be similar to drawing numbers from a hat). The result will determine which group the participant will belong to. The study would have three different groups: 1. Group one will be self-motivated to be physically active (control group) 2. The second group will receive smart daily text messages only (text group); 3. The last group would have the Amazon Echo smart device installed in participants' homes and they will interact with a digital voice assist that will help them be active (Alexa group). * All study participants will receive a Fitbit device which you can keep after the study. * When the investigators start the study, the participant will be asked to start wearing wrist device immediately. For the next one week the investigators will monitor the participant daily number of steps to establish how active he/she is. The participant will not be required to change his/her daily routine in the first week. * After the end of the first week, the investigators will ask the participant to increase his/her daily steps to at least 10 thousand steps per day for the next four weeks. In total the participant will have one week of hi/her normal daily routine and four weeks of trying to do 10 thousand steps per day or more. * If the participant is in the control group, the investigators will ask you to try to do 10 thousand steps per day on his/her own. * If the participant are in the text group, he/she will get smart text messages with healthy tips to help track his/her activities and reach his/her daily goal. * If the participant is in Alexa group, a study member will visit the participant at his/her home before the end of the first week to install the Echo smart speaker and to explain to the participant how to use the voice assist to help him/her become more active for the next four weeks. ### Conditions Module **Conditions:** - Breast Cancer - Prostate Cancer - Lung Cancer - Colorectal Cancer - Cervical Cancer - Oral Cancer **Keywords:** - Cancer Survivors - Physical Activities - Baltimore - State of Maryland ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** This is a single center three arms randomized trial. ##### Masking Info **Masking:** SINGLE **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** OTHER #### Enrollment Info **Count:** 42 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** The participant will self motivate hi/herself to increase physical activities. **Label:** Control **Type:** NO_INTERVENTION #### Arm Group 2 **Description:** The participant will receive personalized smart text messages to encourage him/her to increase physical activities **Intervention Names:** - Behavioral: Mycoach Smart Text **Label:** Mycoach Smart Text **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** The participant will interact with intelligent coach on Amazon Alexa (a digital voice assist) to help him/her become more active **Intervention Names:** - Behavioral: MyCoach on Amazon Alexa **Label:** MyCoach via Amazon Alexa **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Mycoach Smart Text **Description:** Personalized text messages to you cellphone to help you become more active **Name:** Mycoach Smart Text **Other Names:** - Smart text **Type:** BEHAVIORAL #### Intervention 2 **Arm Group Labels:** - MyCoach via Amazon Alexa **Description:** This is an intelligent voice that you can communicate with via Amazon echo speaker **Name:** MyCoach on Amazon Alexa **Other Names:** - Digital Voice Assist **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** The investigators will measure your physical activity by number of steps before and after intervention via wearable activity tracker. **Measure:** Average number of steps. Wearable sensor based **Time Frame:** 5 weeks #### Secondary Outcomes **Description:** Defined as 3 min. or more of uninterrupted activity measured by wearable sensor. **Measure:** Total number and duration of activity bouts. Wearable sensor based **Time Frame:** 5 weeks **Description:** Time spent walking vs time spent sitting measured by all in one sensor. **Measure:** Transitions between active/inactive periods. Wearable sensor based **Time Frame:** 5 weeks **Description:** Distribution parameter of number of steps per/minute within 24 hours period. Measured by all in one sensor. **Measure:** Daily patterns of activity. Wearable sensor based **Time Frame:** 5 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * A cancer survivor of breast, prostate, lung, colorectal, cervical or oral cancer survivor and live within the Baltimore Maryland area. * Finished your active cancer treatment at least three months ago. * Overweight or obese and do not exercise daily. * Do not have any physical limitation to do mild to moderate physical activities. * Have a smart phone (iPhone or Android device) with Wi-Fi internet connection at home. * Actively using an email account * Willing to accept the random study assignment. * Willing to wear a Fitbit band 'a physical activity tracking device' on your wrist for five weeks every single day. * Willing to have an Echo speaker 'a smart home speaker with voice assistant' installed in your home and use the digital voice assist for four weeks. * Willing to receive daily text messages on your phone for four weeks. * Willing to provide us with access to your Fitbit physical activities data. * Willing to sign the consent form. Exclusion Criteria: * Already doing moderate to high physical activities in their daily life (rapid screener). * Planning to relocate within the next 4-5 weeks. * Stage 4 cancer. * Already using physical activity tracker or part of a physical activity program. * Part of another study that may interfere with our outcome of interest, unstable mental condition. * Mental condition that prevents patient from performing the study activities and requirements. * Pregnancy. **Maximum Age:** 80 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Baltimore **Country:** United States **Facility:** Johns Hopkins Bloomberg School of Public Health **State:** Maryland **Zip:** 21205 #### Overall Officials **Official 1:** **Affiliation:** Johns Hopkins Bloomberg School of Public Health **Name:** Ahmed Hassoon, MD,MPH,PMP **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ### References Module #### References **Citation:** Hassoon A, Baig Y, Naiman DQ, Celentano DD, Lansey D, Stearns V, Coresh J, Schrack J, Martin SS, Yeh HC, Zeilberger H, Appel LJ. Randomized trial of two artificial intelligence coaching interventions to increase physical activity in cancer survivors. NPJ Digit Med. 2021 Dec 9;4(1):168. doi: 10.1038/s41746-021-00539-9. **PMID:** 34887491 **Citation:** Hassoon A, Schrack J, Naiman D, Lansey D, Baig Y, Stearns V, Celentano D, Martin S, Appel L. Increasing Physical Activity Amongst Overweight and Obese Cancer Survivors Using an Alexa-Based Intelligent Agent for Patient Coaching: Protocol for the Physical Activity by Technology Help (PATH) Trial. JMIR Res Protoc. 2018 Feb 12;7(2):e27. doi: 10.2196/resprot.9096. **PMID:** 29434016 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000006258 - Term: Head and Neck Neoplasms - ID: D000009059 - Term: Mouth Diseases - ID: D000009057 - Term: Stomatognathic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: All - Name: All Conditions - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: BC07 - Name: Mouth and Tooth Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M5220 - Name: Breast Neoplasms - Relevance: LOW - As Found: Unknown - ID: M14335 - Name: Prostatic Neoplasms - Relevance: LOW - As Found: Unknown - ID: M11172 - Name: Lung Neoplasms - Relevance: LOW - As Found: Unknown - ID: M5830 - Name: Uterine Cervical Neoplasms - Relevance: LOW - As Found: Unknown - ID: M12022 - Name: Mouth Neoplasms - Relevance: HIGH - As Found: Oral Cancer - ID: M9348 - Name: Head and Neck Neoplasms - Relevance: LOW - As Found: Unknown - ID: M12019 - Name: Mouth Diseases - Relevance: LOW - As Found: Unknown - ID: M12017 - Name: Stomatognathic Diseases - Relevance: LOW - As Found: Unknown - ID: T4265 - Name: Oral Cancer - Relevance: HIGH - As Found: Oral Cancer - ID: T3466 - Name: Lip and Oral Cavity Cancer - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000009062 - Term: Mouth Neoplasms ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02796079 **Brief Title:** A Clinical Study Using Autologous Bone Marrow Stem Cell for Diabetes Related Vascular Complications **Official Title:** A Clinical Study Using Autologous Bone Marrow Stem Cell for Diabetes Related Vascular Complications #### Organization Study ID Info **ID:** ThirdSouthernMedical #### Organization **Class:** OTHER_GOV **Full Name:** The Third Affiliated Hospital of Southern Medical University ### Status Module #### Completion Date **Date:** 2018-12 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2017-04-06 **Type:** ACTUAL **Last Update Submit Date:** 2017-04-04 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2017-12 **Type:** ESTIMATED #### Start Date **Date:** 2015-01 **Status Verified Date:** 2017-04 #### Study First Post Date **Date:** 2016-06-10 **Type:** ESTIMATED **Study First Submit Date:** 2016-05-30 **Study First Submit QC Date:** 2016-06-06 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Nanfang Hospital, Southern Medical University **Class:** INDUSTRY **Name:** Academy Military Medical Science, China **Class:** OTHER **Name:** The Fifth Affiliated Hospital of Southern Medical University **Class:** OTHER **Name:** Southern Medical University, China #### Lead Sponsor **Class:** OTHER_GOV **Name:** Jie Shen #### Responsible Party **Investigator Affiliation:** The Third Affiliated Hospital of Southern Medical University **Investigator Full Name:** Jie Shen **Investigator Title:** doctor **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** Stem cell therapy has been a new and effective therapy in recent years for diabetic foot.This study intends to establish an optimal clinical research program, and attempts to break the technical bottleneck in the stem cell therapy for treating diabetes related vascular complications. **Detailed Description:** Diabetic foot is one of the most serious chronic complications of diabetic patients, and still lacking effective treatments. Stem cell therapy has been a new and effective therapy in recent years for diabetic foot. Combined with the previous studies of our research group, this study intends to transform part of the results of this research, establish an optimal clinical research program, and attempts to break the technical bottleneck in the stem cell therapy for treating diabetes related vascular complications. ### Conditions Module **Conditions:** - Peripheral Vascular Disease - Ischemia - Diabetic Foot ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 240 **Type:** ESTIMATED **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Mesenchymal stem cells derived from bone marrow infusion **Intervention Names:** - Biological: mesenchymal stem cells **Label:** Autologous Bone Marrow Stem Cell **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** saline injections **Intervention Names:** - Biological: saline **Label:** saline **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Autologous Bone Marrow Stem Cell **Description:** stem cell acquisition, processing and reinfection, to evaluate the efficacy by using autologous bone marrow stem cell **Name:** mesenchymal stem cells **Type:** BIOLOGICAL #### Intervention 2 **Arm Group Labels:** - saline **Description:** saline injections **Name:** saline **Type:** BIOLOGICAL ### Outcomes Module #### Primary Outcomes **Description:** To determine the ability of MSC to facilitate and accelerate diabetic foot ulcers healing. **Measure:** Area of diabetic foot ulcers **Time Frame:** 3 months #### Secondary Outcomes **Description:** Improvement of local perfusion. **Measure:** Improvement of transcutaneous oxygen partial pressure (TcPO2) **Time Frame:** 3 months **Measure:** Improvement of microvascular cutaneous reactivity by laser Doppler perfusion monitoring (LDPM) **Time Frame:** 3 months **Description:** Measure of the subjective symptom of pain. **Measure:** Pain (Visual-Analog Scale) **Time Frame:** 3 months **Measure:** Walking distance (treadmill) if possible **Time Frame:** 3 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Diabetes mellitus Type 2 or Type 1 * Age between 18-80 years * Chronic foot ulcer more than 6 weeks * No sufficient response to best standard care delivered for six weeks. * PAD up to Fontaine stage III or IV period * CLI with the ankle brachial index (index ankle-brachial, ABI) \<0.7 and (or) the percutaneous oxygen partial pressure (oxygen tension transcutaneous, TcPO2) \<30mmHg Exclusion Criteria: * HbA1c \>12% * Hemoglobin \<10 mg/dl * Creatinine clearance rate \<30ml/min * Systemic bacterial, viral infections (Mei Du, hepatitis, cytomegalovirus infection, HIV, B19 infection, herpes virus infection) and sepsis * Have accepted the treatment of stem cells or growth factors * Have a history of malignant disease * Pregnancy * Mental illness history * Abnormal coagulation function * Allergic reaction * Severe cardiac insufficiency (III-IV NYHA) * Using vasoactive substances **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:** Jie Shen **Phone:** +86 13808893818 **Role:** CONTACT #### Locations **Location 1:** **City:** Guangzhou **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Zhang Qun - **Phone:** 020-62784060 - **Role:** CONTACT ***Contact 2:*** - **Name:** Jie Shen - **Role:** PRINCIPAL_INVESTIGATOR **Country:** China **Facility:** the Third Affiliated Hospital of Southern Medical University **State:** Guangdong **Status:** RECRUITING **Zip:** 510515 #### Overall Officials **Official 1:** **Affiliation:** The Third Affiliated Hospital of Southern Medical University **Name:** Jie Shen **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ## Derived Section ### Condition Browse Module - Ancestors - ID: D000003925 - Term: Diabetic Angiopathies - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000016523 - Term: Foot Ulcer - ID: D000007871 - Term: Leg Ulcer - ID: D000012883 - Term: Skin Ulcer - ID: D000012871 - Term: Skin Diseases - ID: D000048909 - Term: Diabetes Complications - ID: D000003920 - Term: Diabetes Mellitus - ID: D000004700 - Term: Endocrine System Diseases - ID: D000003929 - Term: Diabetic Neuropathies - ID: D000050197 - Term: Atherosclerosis - ID: D000001161 - Term: Arteriosclerosis - ID: D000001157 - Term: Arterial Occlusive 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: BC14 - Name: Heart and Blood Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: BC10 - Name: Nervous System Diseases ### Condition Browse Module - Browse Leaves - ID: M7115 - Name: Diabetes Mellitus - Relevance: LOW - As Found: Unknown - ID: M17400 - Name: Vascular Diseases - Relevance: HIGH - As Found: Vascular Disease - ID: M10543 - Name: Ischemia - Relevance: LOW - As Found: Unknown - ID: M29213 - Name: Peripheral Arterial Disease - Relevance: HIGH - As Found: Peripheral Vascular Disease - ID: M18894 - Name: Peripheral Vascular Diseases - Relevance: HIGH - As Found: Peripheral Vascular Disease - ID: M19933 - Name: Diabetic Foot - Relevance: HIGH - As Found: Diabetic Foot - ID: M7120 - Name: Diabetic Angiopathies - Relevance: LOW - As Found: Unknown - ID: M17206 - Name: Ulcer - Relevance: LOW - As Found: Unknown - ID: M18919 - Name: Foot Ulcer - Relevance: LOW - As Found: Unknown - ID: M10883 - Name: Leg Ulcer - Relevance: LOW - As Found: Unknown - ID: M15686 - Name: Skin Ulcer - Relevance: LOW - As Found: Unknown - ID: M15674 - Name: Skin Diseases - Relevance: LOW - As Found: Unknown - ID: M26004 - Name: Diabetes Complications - Relevance: LOW - As Found: Unknown - ID: M7862 - Name: Endocrine System Diseases - Relevance: LOW - As Found: Unknown - ID: M7124 - Name: Diabetic Neuropathies - Relevance: LOW - As Found: Unknown - ID: M26188 - Name: Atherosclerosis - 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 ### Condition Browse Module - Meshes - ID: D000017719 - Term: Diabetic Foot - ID: D000014652 - Term: Vascular Diseases - ID: D000016491 - Term: Peripheral Vascular Diseases - ID: D000058729 - Term: Peripheral Arterial Disease ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05490979 **Brief Title:** The Impact of Dyad Exercises on Well-being and Connection in Young Adults **Official Title:** Effectiveness and Physiological Mechanisms of Contemplative Dyad Meditation to Increase Social Connection in Young Adults in the Aftermath of the Pandemic #### Organization Study ID Info **ID:** #10085952 #### Organization **Class:** OTHER **Full Name:** University of Pennsylvania ### Status Module #### Completion Date **Date:** 2023-10-03 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-10-05 **Type:** ACTUAL **Last Update Submit Date:** 2023-10-03 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2023-06-22 **Type:** ACTUAL #### Start Date **Date:** 2022-09-06 **Type:** ACTUAL **Status Verified Date:** 2023-10 #### Study First Post Date **Date:** 2022-08-08 **Type:** ACTUAL **Study First Submit Date:** 2022-08-04 **Study First Submit QC Date:** 2022-08-05 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Pennsylvania #### Responsible Party **Investigator Affiliation:** University of Pennsylvania **Investigator Full Name:** Michael Platt **Investigator Title:** James S. Riepe University Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** Many people are experiencing low well-being and loneliness, particularly due to the COVID-19 pandemic. As the world is opening back up, it is crucial to determine methods to help people grow closer again and boost subjective well-being. One promising method is contemplative dyad meditation, which has hardly been studied. This is a method in which two people have a structured dialogue with each other while contemplating a prompt, as they alternate between listening and speaking. It is related to but different from other methods that have previously been shown to increase connection, such as the "fast friends" exercise. In "fast friends", two people answer a series of increasingly personal questions in a dialogue. Here, 180 participants between 18-35 years will be randomly allocated to three conditions (stratified by gender): (a) contemplative dyad meditation training, (b) "fast friends", or (c) no-intervention. Participants in the dyad meditation group will receive professional meditation training followed by 2 weeks of regular meditation practice. Participants in the "fast friends" group will meet regularly during 2 weeks to practice "fast friends" exercises. The impact of the interventions on well-being, loneliness, mindfulness, and related measures will be investigated. After the interventions have finished, participants' physiology (heart rate) and brain waves (using electroencephalography \[EEG\]) during the respective exercises will also be measured to explore potential biological mechanisms. Of particular interest are heart rate variability (HRV, often linked with higher well-being), frontal alpha asymmetry in the EEG (linked with positive affect and approach), and biological synchrony in these variables between the two interacting individuals. Both dyad meditations and "fast friends" exercises are predicted to improve closeness, thriving, loneliness, affect, depression, anxiety, and social interaction anxiety compared to no-intervention. Moreover, dyad meditation is predicted to have stronger effects than "fast friends" in terms of increasing mindfulness, self-compassion, and empathy. Dyad meditation and fast friends will show differential physiological signatures (e.g., lower heart rate and higher averaged alpha power for meditation). This study may reveal effective methods to improve well-being and connection and provide insights into their biological mechanisms. ### Conditions Module **Conditions:** - Mental Health Wellness 1 - Loneliness ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Masking Description:** Participants only receive information about the condition they have been assigned to and are not explicitly informed about the other conditions or the overall purpose of the study. All participants will be debriefed when the study has ended. **Who Masked:** - PARTICIPANT **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 120 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Behavioral: Contemplative dyad meditation **Label:** Contemplative dyad meditation **Type:** EXPERIMENTAL #### Arm Group 2 **Intervention Names:** - Behavioral: Fast friends **Label:** Fast friends **Type:** ACTIVE_COMPARATOR #### Arm Group 3 **Label:** No intervention **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Contemplative dyad meditation **Description:** Participants take part in a 3-hour group meditation training led by a professional meditation teacher. They receive detailed instructions and also practice contemplative dyad meditation for at least 30 minutes with another participant. During the 2 weeks following the training, participants meet in supervised group settings to practice the meditation method with alternating partners for up to 6 times. **Name:** Contemplative dyad meditation **Type:** BEHAVIORAL #### Intervention 2 **Arm Group Labels:** - Fast friends **Description:** During 2 weeks, participants meet in supervised group settings to practice the 'fast friends' exercise with alternating partners for up to 6 times. **Name:** Fast friends **Type:** BEHAVIORAL ### Outcomes Module #### Other Outcomes **Description:** measured using electrocardiogram, by Bitalino **Measure:** Heart rate **Time Frame:** 3 weeks **Description:** measured using electrocardiogram, by Bitalino, calculated as Root Mean Square of Successive Differences (RMSSD) **Measure:** Heart rate variability **Time Frame:** 3 weeks **Description:** measured using respiration belts by Bitalino **Measure:** Respiration **Time Frame:** 3 weeks **Description:** measured using Emotiv-EEG **Measure:** EEG **Time Frame:** 3 weeks #### Primary Outcomes **Description:** The Brief Inventory of Thriving (Su, Tay, \& Diener, 2014) is a questionnaire consisting of 10 items, measuring psychological well-being. Scores range from 1 to 5 with a higher score indicating more thriving (i.e., higher well-being). **Measure:** Brief Inventory of Thriving **Time Frame:** 3 weeks **Description:** The Loneliness sub-scale from the Comprehensive Inventory of Thriving (Su, Tay, \& Diener, 2014) consists of 3 items. Scores range from 1 to 5 with a higher score indicating higher loneliness. **Measure:** Loneliness Scores on the Comprehensive Inventory of Thriving **Time Frame:** 3 weeks **Description:** The Five Facet Mindfulness Questionnaire (Baer et al., 2006) consists of 39 items. Scores for mindfulness range between 1 and 5 with a higher score indicating more mindfulness. **Measure:** Mindfulness Score on the Five Facet Mindfulness Questionnaire **Time Frame:** 3 weeks **Description:** The Inclusion of Other in the Self Scale (Aron, Aron, \& Smollan, 1992) consists of 7 response options measuring closeness to another person (here: the meditation or exercise partner). The response options each show two circles labeled as" "Self" and "Other", which vary in terms of overlap, from barely touching (1, i.e., not close at all) to almost completely overlapping (7, i.e., extremely close). **Measure:** Inclusion of Other in the Self Scale **Time Frame:** Directly after each individual meditation or "fast friends" exercise #### Secondary Outcomes **Description:** The 10-item version of the Negative And Positive Affect Scale by Joshanloo (2017) contains 5 items measuring positive affect. Scores for positive affect from these 5 items range from 5 to 25 with 25 indicating higher positive affect. The scale is used here with the following adaptation: it asks about the previous 14 days rather than 30 days. **Measure:** Positive Affect Scores on the Negative And Positive Affect Scale **Time Frame:** 3 weeks **Description:** The 10-item version of the Negative And Positive Affect Scale (NAPAS) by Joshanloo (2017) contains 5 items measuring negative affect. Scores for negative affect from these 5 items range from 5 to 25 with 25 indicating higher negative affect. The scale is used here with the following adaptation: it asks about the previous 14 days rather than 30 days. **Measure:** Negative Affect Scores on the Negative And Positive Affect Scale **Time Frame:** 3 weeks **Description:** The Patient Health Questionnaire-9 consists of 9 items and measures the severity of depression. Scores range from 0 to 27 with a higher score indicating more severe depression. **Measure:** Depression Scores on the Patient Health Questionnaire-9 **Time Frame:** 3 weeks **Description:** The Generalized Anxiety Disorder 7-item questionnaire screens for general anxiety disorder. Scores range from 0 to 21 with higher scores indicating more severe anxiety. **Measure:** Generalized Anxiety Disorder-7 questionnaire **Time Frame:** 3 weeks **Description:** The Social Interaction Anxiety Scale-6 (Peters et al., 2012) is a 6-item measure assessing social interaction anxiety. Scores range from 0 to 24 with 24 indicating more severe social anxiety. **Measure:** Social Interaction Anxiety Scale-6 **Time Frame:** 3 weeks **Description:** The short form of the Self-Compassion Scale (Raes et al., 2011) includes 12 items. Scores range from 1 to 5 with higher scores indicating more self-compassion. Change = (Week 3 Score - Baseline Score). **Measure:** Self-Compassion Scale Short-Form **Time Frame:** 3 weeks **Description:** The Toronto Empathy Questionnaire consists of 16 items. Scores range from 0 to 64 with higher scores indicating higher empathy. **Measure:** Toronto Empathy Questionnaire **Time Frame:** 3 weeks **Description:** The 10-item version of the Negative And Positive Affect Scale by Joshanloo (2017) contains 5 items measuring positive affect. Scores for positive affect from these 5 items range from 5 to 25 with 25 indicating higher positive affect. The scale is used here with the following adaptation: it asks about the current moment rather than the past 30 days. Response options are adapted to vary from 1 (not at all) to 5 (extremely). **Measure:** Positive Affect Scores on the Negative And Positive Affect Scale **Time Frame:** Directly after each individual meditation or "fast friends" exercise **Description:** The 10-item version of the Negative And Positive Affect Scale (NAPAS) by Joshanloo (2017) contains 5 items measuring negative affect. Scores for negative affect from these 5 items range from 5 to 25 with 25 indicating higher negative affect. The scale is used here with the following adaptation: it asks about the current moment rather than the past 30 days. Response options are adapted to vary from 1 (not at all) to 5 (extremely). **Measure:** Negative Affect Scores on the Negative And Positive Affect Scale **Time Frame:** Directly after each individual meditation or "fast friends" exercise **Description:** Average correlation between emotions of the 2 people doing the exercise together, as reported with regards to the last 5 minutes of the meditation or the "fast friends" exercise. The following emotions (taken from the short version of the Negative and Positive Affect scale by Joshanloo) will be rated by both participants: restless/fidgety, hopeless, that everything was an effort, in good spirits, calm and peaceful, full of life. **Measure:** Emotional synchrony **Time Frame:** Directly after each individual meditation or "fast friends" exercise ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Current student, staff, or employee at the University of Pennsylvania (for safety reasons) Exclusion Criteria: * none **Healthy Volunteers:** True **Maximum Age:** 35 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Philadelphia **Country:** United States **Facility:** University of Pennsylvania **State:** Pennsylvania **Zip:** 19104 #### Overall Officials **Official 1:** **Affiliation:** University of Pennsylvania **Name:** Michael L Platt, PhD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02348879 **Brief Title:** A Study to Examine the Safety, Pharmacokinetics and Pharmacodynamics of AMG 403 in Healthy Subject **Official Title:** A Placebo Controlled, Randomized, Double-blind, Sequential, Rising, Single Dose Study to Examine the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Intravenous and Subcutaneous AMG 403 in Healthy Young and Older Adult Subjects #### Organization Study ID Info **ID:** 20040195 #### Organization **Class:** INDUSTRY **Full Name:** Amgen ### Status Module #### Completion Date **Date:** 2006-03 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2015-01-30 **Type:** ESTIMATED **Last Update Submit Date:** 2015-01-28 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2006-01 **Type:** ACTUAL #### Start Date **Date:** 2005-01 **Status Verified Date:** 2015-01 #### Study First Post Date **Date:** 2015-01-28 **Type:** ESTIMATED **Study First Submit Date:** 2014-12-23 **Study First Submit QC Date:** 2015-01-27 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Amgen #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** This study is to evaluate the safety of AMG 403 in healthy adult subjects. The study consists of a 21 day screening period followed by administration of the investigational product and up to 154 day evaluation period. ### Conditions Module **Conditions:** - Chronic Pain ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** QUADRUPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 51 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** AMG 403 administered as subcutaneous and intravenous doses **Intervention Names:** - Drug: AMG 403 **Label:** AMG 403 **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** No active drug **Intervention Names:** - Drug: Placebo **Label:** Placebo **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - AMG 403 **Description:** AMG 403 is for treatment of subjects with chronic pain **Name:** AMG 403 **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Placebo **Description:** contains no active drug **Name:** Placebo **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Incidence of treatment emergent adverse events **Time Frame:** up to 112 days **Description:** Vital signs to be assessed included temperature, respiratory rate, pulse rate and rhythm (regular/irregular), and blood pressure. Generally, abnormal vital signs were only reported as adverse events if they required treatment or were associated with an adverse event. **Measure:** Incidence of abnormal clinically significant vital signs **Time Frame:** up to 112 days **Description:** Laboratory abnormalities were defined by laboratory normal ranges and were not reported as adverse events unless symptomatic or associated with an adverse event. **Measure:** Incidence of abnormal clinically significant chemistry, hematology and urinalysis test results **Time Frame:** up to 112 days **Description:** ECG abnormalities were reported as adverse events if they represented a change from baseline or if associated with symptoms or an adverse event. **Measure:** Incidence of abnormal clinically significant ECG results **Time Frame:** up to 112 days #### Secondary Outcomes **Measure:** Pharmacokinetics profile of AMG 403 including Tmax, AUClast and Cmax **Time Frame:** up to 112 days ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Healthy men and women of non-child bearing potential, * Between the ages of 18 and 55 inclusive, * Body mass index from 18 to 33 kg/m2, * Skin type compatible with the study assessments, and without significant skin allergies, pigmentary disorders, tattoos, or any active dermatologic conditions that might interfere with the study conduct. Exclusion Criteria: * Prior or current history of peripheral neuropathy, paraesthesias, dysesthesias, herpes zoster, post-herpetic neuralgia, * Evidence of any current illness such as a common cold, viral syndrome, or flu-like symptoms, any disturbance of the autonomic nervous system, * History of Raynaud's phenomenon; Know allergy or intolerance to capsaicin or hot peppers. **Healthy Volunteers:** True **Standard Ages:** - CHILD - ADULT - OLDER_ADULT ### Contacts Locations Module #### Overall Officials **Official 1:** **Affiliation:** Amgen **Name:** MD **Role:** STUDY_DIRECTOR ### References Module #### References **Citation:** Gow JM, Tsuji WH, Williams GJ, Mytych D, Sciberras D, Searle SL, Mant T, Gibbs JP. Safety, tolerability, pharmacokinetics, and efficacy of AMG 403, a human anti-nerve growth factor monoclonal antibody, in two phase I studies with healthy volunteers and knee osteoarthritis subjects. Arthritis Res Ther. 2015 Oct 8;17:282. doi: 10.1186/s13075-015-0797-9. **PMID:** 26449617 #### See Also Links **Label:** AmgenTrials clinical trials website **URL:** http://www.amgentrials.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 - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M29442 - Name: Chronic Pain - Relevance: HIGH - As Found: Chronic Pain - ID: M13066 - Name: Pain - Relevance: LOW - As Found: Unknown - ID: M12404 - Name: Neurologic Manifestations - Relevance: LOW - As Found: Unknown - ID: T1303 - Name: Chronic Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000059350 - Term: Chronic Pain ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05082779 **Brief Title:** Study in Healthy Volunteers to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of CS0159 **Official Title:** A Phase I, Randomized, Double-Blind, Placebo-Contralled, Single Asending Dose / Multiple Ascending Dose Study of CS0159 to to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics, and Effect Food in Healthy Subject #### Organization Study ID Info **ID:** CS0159-001 #### Organization **Class:** OTHER **Full Name:** Cascade Pharmaceuticals, Inc ### Status Module #### Completion Date **Date:** 2022-10-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2022-10-18 **Type:** ACTUAL **Last Update Submit Date:** 2022-10-15 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2022-09-16 **Type:** ACTUAL #### Start Date **Date:** 2021-10-26 **Type:** ACTUAL **Status Verified Date:** 2021-10 #### Study First Post Date **Date:** 2021-10-19 **Type:** ACTUAL **Study First Submit Date:** 2021-09-25 **Study First Submit QC Date:** 2021-10-05 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Covance #### Lead Sponsor **Class:** OTHER **Name:** Cascade Pharmaceuticals, Inc #### 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 whole study includes 2 parts. Both the SAD study and MAD study are randomized, double-blinded, and placebo-controlled studies, conducted in healthy subjects, to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics profiles of CS0159. The SAD part also involves a pilot food effect (FE) study, designed to assess the food effect on single-dose PK profile in healthy subjects. **Detailed Description:** A total of 48 healthy subjects will be allocated to 1 of 6 cohorts (cohort A1\~A6) in the SAD study, each cohort including 8 subjects (6 subjects will receive investigational new drug (IND) product and 2 receive placebo). Each subject in fasted state will be randomly assigned to receive a single oral dose of CS0159 or placebo.To ensure the safety for all SAD cohorts (including A3 in both treatment periods). The MAD study will enroll 32 healthy subjects, allocated to 1 of 4 cohorts (cohort B1\~B4) and each cohort including 8 participants (6 subjects will receive IND products and 2 receive placebo). Subjects will be randomly assigned to orally receive the IND product or placebo. ### Conditions Module **Conditions:** - Primary Sclerosing Cholangitis (PSC) ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - INVESTIGATOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 79 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants in fasted state will receive CS0159 0.2 mg or placebo once on Day 1. **Intervention Names:** - Drug: CS0159 **Label:** Cohort A1: 0.2 mg **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants in fasted state will receive CS0159 0.6 mg or placebo once on Day 1. **Intervention Names:** - Drug: CS0159 **Label:** Cohort A2: 0.6 mg **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Participants in fasted state will receive CS0159 1 mg or placebo once on Day 1 followed by a 7-day washout period then given in 1 mg tablet (in fed state) on Day 8. **Intervention Names:** - Drug: CS0159 **Label:** Cohort A3: 1 mg **Type:** EXPERIMENTAL #### Arm Group 4 **Description:** Participants in fasted state will receive CS0159 2 mg or placebo once on Day 1. **Intervention Names:** - Drug: CS0159 **Label:** Cohort A4: 2 mg **Type:** EXPERIMENTAL #### Arm Group 5 **Description:** Participants in fasted state will receive CS0159 4 mg or placebo once on Day 1. **Intervention Names:** - Drug: CS0159 **Label:** Cohort A5: 4 mg **Type:** EXPERIMENTAL #### Arm Group 6 **Description:** Participants in fasted state will receive CS0159 8 mg or placebo once on Day 1. **Intervention Names:** - Drug: CS0159 **Label:** Cohort A6: 8 mg **Type:** EXPERIMENTAL #### Arm Group 7 **Description:** Participants in fasted state will receive CS0159 0.4 mg or placebo once daily for a consecutive 14 days. **Intervention Names:** - Drug: CS0159 **Label:** Cohort B1: 0.4 mg **Type:** EXPERIMENTAL #### Arm Group 8 **Description:** Participants in fasted state will receive CS0159 1 mg or placebo once daily for a consecutive 14 days. **Intervention Names:** - Drug: CS0159 **Label:** Cohort B2: 1 mg **Type:** EXPERIMENTAL #### Arm Group 9 **Description:** Participants in fasted state will receive CS0159 2 mg or placebo once daily for a consecutive 14 days. **Intervention Names:** - Drug: CS0159 **Label:** Cohort B3: 2 mg **Type:** EXPERIMENTAL #### Arm Group 10 **Description:** Participants in fasted state will receive CS0159 4 mg or placebo once daily for a consecutive 14 days. **Intervention Names:** - Drug: CS0159 **Label:** Cohort B4: 4mg **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Cohort A1: 0.2 mg - Cohort A2: 0.6 mg - Cohort A3: 1 mg - Cohort A4: 2 mg - Cohort A5: 4 mg - Cohort A6: 8 mg - Cohort B1: 0.4 mg - Cohort B2: 1 mg - Cohort B3: 2 mg - Cohort B4: 4mg **Description:** Tablets administered orally **Name:** CS0159 **Other Names:** - Placebo **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Area under the concentration-time curve (AUC) from time zero to infinity (AUC0-∞) **Measure:** Single-Dose Pharmacokinetic (PK) Parameter **Time Frame:** Day 1 after dosing **Description:** AUC from time zero to the time of the last measured concentration **Measure:** Single-Dose Pharmacokinetic (PK) Parameter: (AUC0-last) **Time Frame:** Day 1 after dosing **Description:** Maximum observed plasma concentration **Measure:** Single-Dose Pharmacokinetic (PK) Parameter: (Cmax) **Time Frame:** Day 1 after dosing **Description:** Time of the maximum observed plasma concentration **Measure:** Single-Dose Pharmacokinetic (PK) Parameter: (Tmax) **Time Frame:** Day 1 after dosing **Description:** Maximum concentration during a dosing interval Ct_max **Measure:** Multiple-Dose PK Parameter **Time Frame:** Day 1 after dosing; day 14 **Description:** Minimum concentration during a dosing interval **Measure:** Multiple-Dose PK Parameter: (Ct_min, Day 14) **Time Frame:** Day 1 after dosing; day 14 **Description:** AUC over one dosing interval **Measure:** Multiple-Dose PK Parameter: (AUCtau) **Time Frame:** Day 1 after dosing; day 14 **Description:** Incidence and severity of adverse events **Measure:** To characterize the safety and tolerability of single dose of CS0159 **Time Frame:** up to Day 31 **Description:** Incidence and severity of adverse events **Measure:** To characterize the safety and tolerability of multiple doses of CS0159 **Time Frame:** up to Day 44 #### Secondary Outcomes **Description:** fibroblast growth factor 19 **Measure:** Pharmacodynamic (PD) Parameter: FGF19 **Time Frame:** Day -1; day 1 **Description:** serum concentration **Measure:** Pharmacodynamic (PD) Parameter: C4 **Time Frame:** Day -1; day 1 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Healthy male and non-pregnant female volunteers 2. In good health, determined by having no clinically significant findings from medical history, physical examination, 12-lead ECG, vital signs measurements, and clinical laboratory evaluations Exclusion Criteria: 1. Subjects with special dietary requirements and cannot follow a uniform diet. 2. Pregnant or nursing females or females who have pregnancy plans during the trial or within 3 months after the trial. 3. Any subject with SARS-CoV-2 infection, based on a positive polymerase chain reaction for SARS-CoV-2. 4. History or evidence of clinically significant disorder, condition, or disease that, in the opinion of the investigator, would pose a risk to subject safety or interfere with the study evaluations, procedures, or completion. **Healthy Volunteers:** True **Maximum Age:** 55 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Daytona Beach **Country:** United States **Facility:** Labcorp Clinical Research Unit, Inc. **State:** Florida **Zip:** 32117 #### Overall Officials **Official 1:** **Affiliation:** Labcorp Clinical Research Unit, Inc. **Name:** Kathleen Doisy, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000001649 - Term: Bile Duct Diseases - ID: D000001660 - Term: Biliary Tract Diseases - ID: D000004066 - Term: Digestive System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC06 - Name: Digestive System Diseases - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M6002 - Name: Cholangitis - Relevance: HIGH - As Found: Cholangitis - ID: M17914 - Name: Cholangitis, Sclerosing - Relevance: HIGH - As Found: Primary Sclerosing Cholangitis - ID: M4935 - Name: Bile Duct Diseases - Relevance: LOW - As Found: Unknown - ID: M4946 - Name: Biliary Tract 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: T4712 - Name: Primary Sclerosing Cholangitis - Relevance: HIGH - As Found: Primary Sclerosing Cholangitis ### Condition Browse Module - Meshes - ID: D000002761 - Term: Cholangitis - ID: D000015209 - Term: Cholangitis, Sclerosing ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02302079 **Acronym:** VIDI **Brief Title:** A Study to Evaluate ASP8232 in Reducing Central Retinal Thickness in Subjects With Diabetic Macular Edema (DME) **Official Title:** A Phase 2, Double-Masked, Randomized, Active Controlled Study to Evaluate the Efficacy and Safety of ASP8232 in Reducing Central Retinal Thickness in Subjects With Diabetic Macular Edema #### Organization Study ID Info **ID:** 8232-CL-3001 #### Organization **Class:** INDUSTRY **Full Name:** Astellas Pharma Inc ### Status Module #### Completion Date **Date:** 2016-08-12 **Type:** ACTUAL #### Disp First Post Date **Date:** 2017-08-08 **Type:** ACTUAL **Disp First Submit Date:** 2017-08-01 **Disp First Submit QC Date:** 2017-08-01 #### Expanded Access Info #### Last Update Post Date **Date:** 2019-03-06 **Type:** ACTUAL **Last Update Submit Date:** 2019-03-04 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2016-08-12 **Type:** ACTUAL #### Start Date **Date:** 2015-01-12 **Type:** ACTUAL **Status Verified Date:** 2019-03 #### Study First Post Date **Date:** 2014-11-26 **Type:** ESTIMATED **Study First Submit Date:** 2014-11-24 **Study First Submit QC Date:** 2014-11-24 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Astellas Pharma Europe B.V. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to evaluate efficacy and safety of ASP8232 in subjects with diabetic macular edema (DME). This study will evaluate the percent change from baseline in excess central subfield thickness (CST) in the study eye as assessed by spectral domain-optical coherence Tomography (SD-OCT) for ASP8232 monotherapy at Month 3. ### Conditions Module **Conditions:** - Diabetes Mellitus - Diabetic Macular Edema **Keywords:** - ASP8232 - Diabetes Mellitus - ranibizumab - Diabetic Macular Edema ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** TRIPLE **Who Masked:** - PARTICIPANT - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 96 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** ASP8232 will be given orally once daily and sham injections 3 times with 1 month intervals **Intervention Names:** - Drug: ASP8232 - Other: Sham intravitreal (IVT) injection **Label:** ASP8232 + sham intravitreal (IVT) injections **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** ASP8232 will be given orally once daily and ranibizumab injections 3 times with 1 month intervals **Intervention Names:** - Drug: ASP8232 - Drug: ranibizumab **Label:** ASP8232 + ranibizumab intravitreal (IVT) injections **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Placebo will be given orally once daily and ranibizumab injections 3 times with 1 month intervals **Intervention Names:** - Drug: ranibizumab - Drug: Placebo **Label:** Placebo + ranibizumab intravitreal (IVT) injections **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - ASP8232 + ranibizumab intravitreal (IVT) injections - ASP8232 + sham intravitreal (IVT) injections **Description:** oral capsule **Name:** ASP8232 **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - ASP8232 + ranibizumab intravitreal (IVT) injections - Placebo + ranibizumab intravitreal (IVT) injections **Description:** intravitreal (IVT) injection **Name:** ranibizumab **Other Names:** - Lucentis **Type:** DRUG #### Intervention 3 **Arm Group Labels:** - Placebo + ranibizumab intravitreal (IVT) injections **Description:** oral capsule **Name:** Placebo **Type:** DRUG #### Intervention 4 **Arm Group Labels:** - ASP8232 + sham intravitreal (IVT) injections **Description:** intravitreal (IVT) injection **Name:** Sham intravitreal (IVT) injection **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Measure:** Percent change from baseline in excess central subfield thickness (CST) in the study eye as assessed by spectral domain-optical coherence tomography (SD-OCT) at Month 3 **Time Frame:** Baseline and Month 3 #### Secondary Outcomes **Measure:** Absolute change from baseline in CST in the study eye as assessed by SD-OCT at Month 3 **Time Frame:** Baseline and Month 3 **Measure:** Change from baseline in early treatment diabetic retinopathy study (ETDRS) best corrected visual acuity (BCVA) score in the study eye at Month 3 **Time Frame:** Baseline and Month 3 **Measure:** Absolute and percent change from baseline in excess CST in the study eye as assessed by SD-OCT at Months 1 and 2 **Time Frame:** Baseline and Months 1, 2 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Subject must have a documented diagnosis of type 1 or type 2 diabetes mellitus and a glycosylated hemoglobin A1c (HbA1c) of ≤ 12.0% at Screening * Subject has definite retinal thickening due to diffuse diabetic macular edema (DME) involving the central macula based on evaluating investigator's clinical evaluation and demonstrated by spectral domain-optical coherence tomography (SD-OCT) * Subject has central subfield thickness (CST) of at least 375 μm by SD-OCT with presence of intraretinal and/or subretinal fluid at screening visit and at the randomization visit * Subject has early treatment diabetic retinopathy study (ETDRS) best corrected visual acuity (BCVA) letter score ≤ 73 (Snellen 20/40) and ≥ 24 (Snellen 20/320) at screening visit Exclusion Criteria: * Subject's study eye has macular edema considered to be due to a cause other than DME * Subject's study eye has a decrease in BCVA due to causes other than DME that is likely to be decreasing BCVA by 3 lines or more * Subject's study eye has significant macular ischemia as shown on angiography * Subject's study eye has any other ocular disease that may cause substantial reduction in BCVA * Subject has active peri-ocular or ocular infection * Subject's study eye has a history of non-infectious uveitis * Subject's study eye has high myopia (-8 diopter or more correction) * Subject's study eye has a history of prior pars plana vitrectomy * Subject's study eye has a history of any ocular surgery within 3 months prior to Day 1 * Subject's study eye has a history of YAG capsulotomy within 3 months prior to Day 1 * Subject's study eye has a history of panretinal scatter photocoagulation (PRP) or focal laser within 3 months prior to Day 1 or anticipated need for PRP during the course of the study through the Week 12 visit * Subject's study eye has a history of prior IVT, subtenon, or periocular, non-sustained release, steroid therapy within 3 months prior to Day 1 * Subject's study eye has a history of intravitreal sustained release dexamethasone therapy within 6 months prior to Day 1. * Subject's study eye has a history of intravitreal sustained release fluocinolone within 3 years prior to Day 1. * Subject's study eye has a history of prior treatment for DME with IVT anti-vascular endothelial growth factor (VEGF) treatment within 8 weeks prior to Day 1 * Subject has a history of prior treatment with any other (than previously listed) approved treatment which is not labeled for DME within 1 year prior to Day 1 * Subject's study eye has high-risk proliferative diabetic retinopathy (PDR) * Subject has uncontrolled glaucoma * Subject has media clarity, papillary constriction (i.e., senile miosis), or subject lacks cooperation that would interfere with any study procedures, evaluations or interpretation of data **Maximum Age:** 85 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Phoenix **Country:** United States **Facility:** Site US10021 **State:** Arizona **Zip:** 85104 **Location 2:** **City:** Tucson **Country:** United States **Facility:** Site US10025 **State:** Arizona **Zip:** 85704 **Location 3:** **City:** Arcadia **Country:** United States **Facility:** Site US10006 **State:** California **Zip:** 91007 **Location 4:** **City:** Beverly Hills **Country:** United States **Facility:** Site US10004 **State:** California **Zip:** 90211 **Location 5:** **City:** Palm Desert **Country:** United States **Facility:** Site US10007 **State:** California **Zip:** 92260 **Location 6:** **City:** Sacramento **Country:** United States **Facility:** Site US10011 **State:** California **Zip:** 95819 **Location 7:** **City:** Santa Ana **Country:** United States **Facility:** Site US10031 **State:** California **Zip:** 92705 **Location 8:** **City:** Golden **Country:** United States **Facility:** Site US10029 **State:** Colorado **Zip:** 80401 **Location 9:** **City:** Miami **Country:** United States **Facility:** Site US10016 **State:** Florida **Zip:** 33126 **Location 10:** **City:** Winter Haven **Country:** United States **Facility:** Site US10005 **State:** Florida **Zip:** 33880 **Location 11:** **City:** Augusta **Country:** United States **Facility:** Site US10036 **State:** Georgia **Zip:** 30909 **Location 12:** **City:** Boston **Country:** United States **Facility:** Site US10002 **State:** Massachusetts **Zip:** 02114 **Location 13:** **City:** Omaha **Country:** United States **Facility:** Site US10001 **State:** Nebraska **Zip:** 985540 **Location 14:** **City:** Reno **Country:** United States **Facility:** Site US10027 **State:** Nevada **Zip:** 89511 **Location 15:** **City:** Nashville **Country:** United States **Facility:** Site US10012 **State:** Tennessee **Zip:** 37203 **Location 16:** **City:** Abilene **Country:** United States **Facility:** Site US10010 **State:** Texas **Zip:** 79606 **Location 17:** **City:** Austin **Country:** United States **Facility:** Site US10015 **State:** Texas **Zip:** 78705 **Location 18:** **City:** Houston **Country:** United States **Facility:** Site US10013 **State:** Texas **Zip:** 77030 **Location 19:** **City:** McAllen **Country:** United States **Facility:** Site US10030 **State:** Texas **Zip:** 78503 **Location 20:** **City:** San Antonio **Country:** United States **Facility:** Site US10009 **State:** Texas **Zip:** 78240-1502 **Location 21:** **City:** Charlottesville **Country:** United States **Facility:** Site US10017 **State:** Virginia **Zip:** 22903 #### Overall Officials **Official 1:** **Affiliation:** Astellas Pharma Europe B.V. **Name:** Global Medical Lead **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **Access Criteria:** Researchers must submit a proposal to conduct a scientifically relevant analysis of the study data. The research proposal is reviewed by an Independent Research Panel. If the proposal is approved, access to the study data is provided in a secure data sharing environment after receipt of a signed Data Sharing Agreement. **Description:** Access to anonymized individual participant level data collected during the trial, in addition to study-related supporting documentation, is planned for trials conducted with approved product indications and formulations, as well as compounds terminated during development. Conditions and exceptions are described under the Sponsor Specific Details for Astellas on www.clinicalstudydatarequest.com. **Info Types:** - STUDY_PROTOCOL - SAP - CSR **IPD Sharing:** YES **Time Frame:** Access to participant level data is offered to researchers after publication of the primary manuscript (if applicable) and is available as long as Astellas has legal authority to provide the data. **URL:** https://www.clinicalstudydatarequest.com/ ### References Module #### References **Citation:** Nguyen QD, Sepah YJ, Berger B, Brown D, Do DV, Garcia-Hernandez A, Patel S, Rahhal FM, Shildkrot Y, Renfurm RW; VIDI Research Group. Primary outcomes of the VIDI study: phase 2, double-masked, randomized, active-controlled study of ASP8232 for diabetic macular edema. Int J Retina Vitreous. 2019 Aug 1;5:28. doi: 10.1186/s40942-019-0178-7. eCollection 2019. **PMID:** 31388454 #### See Also Links **Label:** Link to results on the Astellas Clinical Study Results website **URL:** https://astellasclinicalstudyresults.com/study.aspx?ID=298 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000044882 - Term: Glucose Metabolism Disorders - ID: D000008659 - Term: Metabolic Diseases - ID: D000004700 - Term: Endocrine System Diseases - ID: D000008268 - Term: Macular Degeneration - ID: D000012162 - Term: Retinal Degeneration - ID: D000012164 - Term: Retinal Diseases - ID: D000005128 - Term: Eye 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: BC11 - Name: Eye Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M7115 - Name: Diabetes Mellitus - Relevance: HIGH - As Found: Diabetes Mellitus - ID: M11261 - Name: Macular Edema - Relevance: HIGH - As Found: Macular Edema - ID: M7657 - Name: Edema - Relevance: HIGH - As Found: Edema - 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 - ID: M11260 - Name: Macular Degeneration - Relevance: LOW - As Found: Unknown - ID: M14997 - Name: Retinal Degeneration - Relevance: LOW - As Found: Unknown - 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: D000008269 - Term: Macular Edema - ID: D000003920 - Term: Diabetes Mellitus - ID: D000004487 - Term: Edema ### Intervention Browse Module - Ancestors - 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: D000000970 - Term: Antineoplastic Agents ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M472 - Name: Ranibizumab - Relevance: HIGH - As Found: Dysfunction - 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: D000069579 - Term: Ranibizumab ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05891379 **Brief Title:** Inebilizumab in Acute Neuromyelitis Optica Spectrum Disorders **Official Title:** Effectiveness and Safety of Inebilizumab in the Acute Phase of Neuromyelitis Optica Spectrum Disorders-a Multicentric, Prospective, Real Word Study #### Organization Study ID Info **ID:** XMEC-2023-003 #### Organization **Class:** OTHER **Full Name:** Xuanwu Hospital, Beijing ### Status Module #### Completion Date **Date:** 2025-07-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-06-29 **Type:** ACTUAL **Last Update Submit Date:** 2023-06-26 **Overall Status:** NOT_YET_RECRUITING #### Primary Completion Date **Date:** 2025-07-31 **Type:** ESTIMATED #### Start Date **Date:** 2023-07-20 **Type:** ESTIMATED **Status Verified Date:** 2023-05 #### Study First Post Date **Date:** 2023-06-06 **Type:** ACTUAL **Study First Submit Date:** 2023-05-24 **Study First Submit QC Date:** 2023-06-05 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Xuanwu Hospital, Beijing #### 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 aimed to observe the effectiveness and safety of inebilizumab in the acute phase of neuromyelitis optica spectrum disorders. **Detailed Description:** This is a a multicentric, prospective, real word study of inebilizumab in NMOSD acute attack compared with oral immunosuppressant. A total of 50 patients will be enrolled at approximately 10 centers around China. ### Conditions Module **Conditions:** - Neuromyelitis Optica Spectrum Disorder **Keywords:** - Neuromyelitis Optica Spectrum Disorder - inebilizumab - observational study ### Design Module #### Bio Spec **Description:** Blood samples are collected before the treatment and at the last visit. **Retention:** SAMPLES_WITH_DNA #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 50 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** intravenous Intravenous methylprednisolone (IVMP) plus intravenous inebilizumab **Intervention Names:** - Drug: Inebilizumab **Label:** Exposed group #### Arm Group 2 **Description:** IVMP plus oral immunosuppressant (Mycophenolate Mofetil or Azathioprine) **Intervention Names:** - Drug: oral immunosuppressant **Label:** Non-exposed group ### Interventions #### Intervention 1 **Arm Group Labels:** - Exposed group **Description:** Inebilizumab: 300mg IV on Day1 and Day 15. The first dose of inelizumab is given during IVMP. **Name:** Inebilizumab **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Non-exposed group **Description:** Oral immunosuppressants (azathioprine or mycolate mofetil) are initiated during IVMP. **Name:** oral immunosuppressant **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Change in Expanded Disability Status Scale (EDSS) score from baseline at the last visit(EDSS : Minimum Score 1, Maximum score 10, higher scores mean a worse outcome). **Measure:** Change in Expanded Disability Status Scale (EDSS) score from baseline **Time Frame:** 6 months #### Secondary Outcomes **Description:** Change in Expanded Disability Status Scale (EDSS) score from baseline at month 1, month 3(EDSS : Minimum Score 1, Maximum score 10, higher scores mean a worse outcome). **Measure:** Change in Expanded Disability Status Scale (EDSS) score from baseline **Time Frame:** 1 months, 3 months **Description:** Disability improvement is defined as a reduction in EDSS score of: A) \>=1.0 from the baseline EDSS score when the baseline score was \<=5.5 B) \>= 0.5 when the baseline EDSS score \> 5.5(EDSS : Minimum Score 1, Maximum score 10, higher scores mean a worse outcome). **Measure:** Percentage of Participants With Disability Improvement **Time Frame:** 6 months **Description:** Change in modified Rankin score (mRS) from baseline at month 1, month 3, month 6(mRS : Minimum Score 0, Maximum score 6, higher scores mean a worse outcome). **Measure:** Change in modified Rankin score (mRS) from baseline **Time Frame:** 1 months, 3 months, 6 months **Measure:** Time to first relapse **Time Frame:** 6 months **Description:** Number of New, and/or Enlarging T2 Hyperintense Lesions Detected by Magnetic Resonance Imaging (MRI) at the last visit **Measure:** Number of New, and/or Enlarging T2 Hyperintense Lesions Detected by Magnetic Resonance Imaging (MRI) **Time Frame:** 6 months **Description:** Change in time taken to complete the timed 25 foot walk test from baseline **Measure:** Change in Timed 25 Foot Walk Test from baseline **Time Frame:** 1 months, 3 months , 6 months **Measure:** Number of NMOSD attacked related rescue treatment **Time Frame:** 6 months **Description:** Change in serum GFAP levels from baseline at the last visit **Measure:** Change in serum GFAP levels from baseline **Time Frame:** 6 months **Description:** Change in AQP4-ab titers from baseline at the last visit **Measure:** Change in AQP4-ab titers from baseline **Time Frame:** 6 months **Description:** Change in Low-contrast Visual Acuity (LCVA) at month 3, month 6)(The LCVA test is used to determine the number of letters that can be read on a standardized low-contrast Landolt C Broken Rings Chart held at a distance of 3 meters). **Measure:** Change in Low-contrast Visual Acuity (LCVA) from baseline **Time Frame:** 3 months, 6 months **Description:** Changes in EQ-5D scores from baseline at month 1 month, month 3 , month 6(EQ-5D-5L: Minimum Score 5, Maximum score 25, lower scores mean a better quality of life). **Measure:** Changes in EQ-5D-5L scores from baseline **Time Frame:** 1 month, 3 months ,6 months **Description:** Change in retinal nerve fiber layer (RNFL) loss measured by optical coherence tomography (OCT) from baseline at month 1, month 3，month 6. **Measure:** Change in retinal nerve fiber layer (RNFL) loss from baseline **Time Frame:** 3 months ,6 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * 1. Age ≥ 18 years with anti-AQP4-IgG seropositive NMOSD as defined by 2015 NMOSD diagnostic criteria by IPND (International Panel for NMO Diagnosis); 2. In the acute phase of NMOSD (definition of acute phase: new neurological symptoms or aggravation of existing symptoms within 30 days before screening, lasting at least 24 hours without with fever); 3. Patients who plan to receive or are receiving intravenous methylprednisolone therapy; 4. Expanded disability status scale (EDSS) score ≤ 8 and ≥ 2.5 during the screening period; 5. Able and willing to give written informed consent; 6. Women of childbearing potential who agree to use adequate contraception during the study. Exclusion Criteria: * 1. Lactating and pregnant females; 2. Participate in other interventional studies within 30 days before screening or within 5 half-lives of the investigational agent before enrollment; 3. Combined with severe mental disorders and other conditions and unable to cooperate with follow-up; 4. History of malignancies; 5. Received plasma exchange, immunoadsorption or intravenous immunoglobulin therapy within 1 month before screening; 6. Patients with negative hepatitis B surface antibody (HBsAg) and positive hepatitis B core antibody (HBcAb), or HBsAg-positive virus carriers, or patients with active tuberculosis or positive tuberculosis screening without appropriate treatment history; 7. Other situations that researchers think are not suitable to participate in this study. **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** NMOSD patients with acute attacks ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Junwei Hao, MD **Phone:** 01083198277 **Role:** CONTACT #### Overall Officials **Official 1:** **Affiliation:** Xuanwu Hospital, Beijing **Name:** Junwei Hao, MD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009188 - Term: Myelitis, Transverse - ID: D000020278 - Term: Demyelinating Autoimmune Diseases, CNS - ID: D000020274 - Term: Autoimmune Diseases of the Nervous System - ID: D000009422 - Term: Nervous System Diseases - ID: D000009902 - Term: Optic Neuritis - ID: D000009901 - Term: Optic Nerve Diseases - ID: D000003389 - Term: Cranial Nerve Diseases - ID: D000003711 - Term: Demyelinating Diseases - ID: D000005128 - Term: Eye Diseases - ID: D000001327 - Term: Autoimmune Diseases - ID: D000007154 - Term: Immune System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC11 - Name: Eye Diseases - Abbrev: BC20 - Name: Immune System Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC01 - Name: Infections - Abbrev: BC04 - Name: Neoplasms - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M12414 - Name: Neuromyelitis Optica - Relevance: HIGH - As Found: Neuromyelitis Optica - ID: M12142 - Name: Myelitis - Relevance: LOW - As Found: Unknown - ID: M12143 - Name: Myelitis, Transverse - Relevance: LOW - As Found: Unknown - ID: M4629 - Name: Autoimmune Diseases - Relevance: LOW - As Found: Unknown - ID: M22098 - Name: Demyelinating Autoimmune Diseases, CNS - Relevance: LOW - As Found: Unknown - ID: M22094 - Name: Autoimmune Diseases of the Nervous System - Relevance: LOW - As Found: Unknown - ID: M12387 - Name: Neuritis - Relevance: LOW - As Found: Unknown - ID: M12833 - Name: Optic Neuritis - Relevance: LOW - As Found: Unknown - ID: M12832 - Name: Optic Nerve Diseases - Relevance: LOW - As Found: Unknown - ID: M6605 - Name: Cranial Nerve Diseases - Relevance: LOW - As Found: Unknown - ID: M6909 - Name: Demyelinating Diseases - Relevance: LOW - As Found: Unknown - ID: M8271 - Name: Eye Diseases - Relevance: LOW - As Found: Unknown - ID: M10200 - Name: Immune System Diseases - Relevance: LOW - As Found: Unknown - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown - ID: T4103 - Name: Neuromyelitis Optica Spectrum Disorder - Relevance: HIGH - As Found: Neuromyelitis Optica Spectrum Disorder - ID: T3988 - Name: Myelitis - Relevance: LOW - As Found: Unknown - ID: T4263 - Name: Optic Neuritis - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000009471 - Term: Neuromyelitis Optica ### Intervention Browse Module - Ancestors - ID: D000007155 - Term: Immunologic Factors - ID: D000045505 - Term: Physiological Effects of Drugs ### Intervention Browse Module - Browse Branches - Abbrev: Infl - Name: Anti-Inflammatory Agents - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: AnEm - Name: Antiemetics - Abbrev: NeuroAg - Name: Neuroprotective Agents - Abbrev: Gast - Name: Gastrointestinal Agents - Abbrev: ARhu - Name: Antirheumatic Agents ### Intervention Browse Module - Browse Leaves - ID: M14120 - Name: Prednisolone - Relevance: LOW - As Found: Unknown - ID: M12128 - Name: Mycophenolic Acid - Relevance: LOW - As Found: Unknown - ID: M11749 - Name: Methylprednisolone - Relevance: LOW - As Found: Unknown - ID: M1833 - Name: Methylprednisolone Acetate - Relevance: LOW - As Found: Unknown - ID: M11750 - Name: Methylprednisolone Hemisuccinate - Relevance: LOW - As Found: Unknown - ID: M229449 - Name: Prednisolone acetate - Relevance: LOW - As Found: Unknown - ID: M211887 - Name: Prednisolone hemisuccinate - Relevance: LOW - As Found: Unknown - ID: M248881 - Name: Prednisolone phosphate - Relevance: LOW - As Found: Unknown - ID: M10212 - Name: Immunosuppressive Agents - Relevance: HIGH - As Found: Acne Scars - ID: M4678 - Name: Azathioprine - Relevance: LOW - As Found: Unknown - ID: M10201 - Name: Immunologic Factors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000007166 - Term: Immunosuppressive Agents ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01148979 **Brief Title:** Lisdexamfetamine Dimesylate in Residual Symptoms and Cognitive Impairment in Major Depressive Disorder. **Official Title:** Double-Blind, Placebo-Controlled, Randomized Trial of Adjunctive Lisdexamfetamine Dimesylate in Residual Symptoms of Major Depressive Disorder Partially Responsive to Selective Serotonin or Norepinephrine Reuptake Inhibitor Monotherapy #### Organization Study ID Info **ID:** 2010-P-000871 #### Organization **Class:** OTHER **Full Name:** Mclean Hospital ### Status Module #### Completion Date **Date:** 2014-10 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-05-23 **Type:** ACTUAL **Last Update Submit Date:** 2017-04-19 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2014-09 **Type:** ACTUAL #### Results First Post Date **Date:** 2017-05-23 **Type:** ACTUAL **Results First Submit Date:** 2017-03-08 **Results First Submit QC Date:** 2017-04-19 #### Start Date **Date:** 2010-09 **Status Verified Date:** 2017-04 #### Study First Post Date **Date:** 2010-06-23 **Type:** ESTIMATED **Study First Submit Date:** 2010-06-21 **Study First Submit QC Date:** 2010-06-22 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Shire #### Lead Sponsor **Class:** OTHER **Name:** Mclean Hospital #### Responsible Party **Investigator Affiliation:** Mclean Hospital **Investigator Full Name:** J. Alexander Bodkin **Investigator Title:** Director, Clinical Psychopharmacology Research Program **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Is US Export:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** This study aims to test the effect of a newly-approved stimulant medication, lisdexamfetamine dimesylate (Vyvanse), on specific residual symptoms of depression found in some patients who are undergoing treatment with, but have only partially responded to, a selective-serotonin reuptake inhibitor (SSRI) or selective-norepinephrine reuptake inhibitor (SNRI) antidepressant. Specifically, the investigators hypothesize that symptoms potentially related to deficient dopaminergic activity, such as lassitude, apathy, reduced positive affect and impaired executive function, in particular, will improve. This protocol is designed to test the hypothesis that this cluster of co-occurring residual symptoms sometimes found in treated depression will respond as a group to adjunctive psychostimulant therapy. The investigators propose to demonstrate this cluster of residual depressive symptoms and to measure the effect of stimulant therapy on it. The investigators hope to better understand the specific symptoms in this clinical population that are likely to improve with stimulant therapy. **Detailed Description:** This protocol is designed to test the hypothesis that a cluster of co-occurring residual symptoms sometimes found in treated depression will respond as a group to psychostimulant therapy. This cluster includes apathy, reduced drive, fatigue, impaired executive function and other cognitive measures, and low positive affect. We propose to measure this cluster of symptoms in a population of residually depressed subjects demonstrating them, and then to measure the effect of stimulant therapy on this cluster, and each constituent symptom, as well as to measure its effect on subjects' overall functional impairment, and to document treatment emergent adverse effects. The goal is to gather data about the response of these residual symptoms to stimulant therapy. Since each subject will be exposed to active treatment and matched placebo, a benefit to individual participants will be to learn if their specific symptom burden is ameliorated by stimulant therapy, and what adverse effects may emerge for them. We hope to develop an understanding of the specific symptoms in this clinical population that are likely to improve with stimulant therapy. We also hope to be able to characterize the side effect burden of stimulant therapy in this clinical population. ### Conditions Module **Conditions:** - Major Depressive Disorder **Keywords:** - Major Depressive Disorder - Cognitive Impairment - Partial Response - Residual Symptoms ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER **Intervention Model Description:** This is a double-blind, placebo-controlled, crossover study in which each subject will act as their own control after being randomly assigned to placebo or Lisdexamfetamine Dimesylate (Vyvanse) for the first 4 weeks of study treatment, then receiving the other for the second 4 weeks of treatment. The two 4-week treatment periods are separated by a washout of 2 weeks. ##### Masking Info **Masking:** DOUBLE **Masking Description:** Both the study participant and the study investigators making evaluations are blinded to which treatment arm the participants are assigned to (i.e., whether they are receiving placebo or Vyvanse at any given point). Placebo and active medication capsules look identical. **Who Masked:** - PARTICIPANT - INVESTIGATOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 35 **Type:** ACTUAL **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants receive Lisdexamfetamine Dimesylate 20-50 mg capsule each morning for 4 weeks. After a washout period of 2 weeks, they receive Placebo capsule (matching Lisdexamfetamine Dimesylate (Vyvanse) capsule) each morning for 4 weeks. **Intervention Names:** - Drug: Lisdexamfetamine Dimesylate (Vyvanse) **Label:** Adjunct Lisdexamfetamine (Vyvanse) **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants receive Placebo capsule (matching Lisdexamfetamine Dimesylate (Vyvanse) 20-50 mg capsule) each morning for 4 weeks. After a washout period of 2 weeks, they receive Lisdexamfetamine Dimesylate capsule each morning for 4 weeks. **Intervention Names:** - Drug: Placebo **Label:** Adjunct Placebo **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Adjunct Lisdexamfetamine (Vyvanse) **Description:** Lisdexamfetamine Dimesylate (Vyvanse) capsules dose ranging from 20mg to 50 mg. **Name:** Lisdexamfetamine Dimesylate (Vyvanse) **Other Names:** - Vyvanse **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Adjunct Placebo **Description:** Lisdexamfetamine Dimesylate (Vyvanse)-matched placebo capsules. **Name:** Placebo **Other Names:** - Sugar pill **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** The Montgomery-Asberg Depression Rating Scale Dysphoric Apathy Retardation subfactor (MDAR) is a 5-item subscale of the clinician-administered 10-item Montgomery-Asberg Depression Rating Scale (MADRS). MDAR score can range from 0-30 with a higher score representing a greater severity of depressive symptoms. **Measure:** Change From Baseline in the Dysphoric Apathy/Retardation Sub-factor (MDAR) of Montgomery-Asberg Depression Rating Scale (MADRS) at 4 Weeks. **Time Frame:** Baseline to 4 weeks of treatment ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Meeting diagnostic criteria for major depression during the present episode of illness, currently with at least mild improvement by report (CGI-I ≥3) but with continuing residual symptoms. 2. A score of ≥10 on MADRS items 1,2,6,7 and 8, (the MADRS Dysphoric Apathy/Retardation factor, Parker et al., 2003) at screening and randomization. These 5 items are: Apparent sadness, Reported sadness, Concentration difficulties, Lassitude, and Inability to feel. A score of ≥3 will be required for at least 2 of these items. 3. A score of 3 or 4 on the Clinical Global Impression of Severity (CGI-S) at screening and randomization. 4. At randomization subjects must have received therapeutic dosages of approved SSRI or SNRI agents for at least 8 weeks, with the last 4 weeks at a constant dosage. 5. Females of Child-bearing Potential (FOCP) must have a negative serum beta Human Chorionic Gonadotropin (B-hCG) pregnancy test at the screening visit and a negative urine pregnancy test at the baseline visit, and agree to use one of the following methods of birth control: oral contraceptives, contraceptive implants, injectable contraceptives, IUDs, double-barrier contraception, sexual abstinence or vasectomized partner(s). Exclusion Criteria: 1. Treatment within 4 weeks of randomization with any non-SSRI/SNRI antidepressant (trazodone is permitted up to 100 mg at night for sleep); any antipsychotic agent; any mood stabilizer; any standing benzodiazepine regimen other than at low doses for sleep (≤ 1 mg lorazepam HS or the equivalent); or a standing regimen of any other agent that may affect cognitive function (e.g., psychostimulants, including modafinil or R-modafinil). 2. Any current or past psychotic disorder, Bipolar I or II disorder, Current panic disorder, History of ADHD, Antisocial Personality Disorder or Borderline Personality Disorder, Mental retardation or any dementing disorder. 3. Covi Anxiety Scale score greater than Raskin Depression Scale score at Screening, to exclude subjects with more prominent anxiety than depression 4. MADRS Sleep (item 4), or Appetite (item 5) \>3 at screening or randomization 5. Initial insomnia at screening that is not adequately controlled by sleep medication (trazodone up to 100 mg HS for sleep and/or ≤ 1 mg lorazepam HS or the equivalent). 6. Medical conditions that might be exacerbated by Vyvanse treatment, such as uncontrolled hypertension or angina; or conditions that would make study findings hard to interpret, such as hyperthyroidism 7. History of seizure (other than infantile febrile seizures), any tic disorder, or a current diagnosis and/or family history of Tourette's Disorder. 8. Known cardiac structural abnormality or any other condition that may affect cardiac performance 9. Any clinically significant ECG or laboratory abnormality at Screening 10. Current abnormal thyroid function, as defined by abnormal TSH at Screening (Treatment with a stable dose of thyroid medication for at least 3 months is permitted if TSH is normal at screening). 11. Suicide attempt within the past 2 years or a history of any homicidal behavior. 12. MADRS Suicidal thoughts (item 10) \>4 at any study visit, or if a patient is considered by the investigator to be at clinical risk of suicide at any time in the course of the study. 13. resting sitting systolic blood pressure \>149mmHg or diastolic blood pressure \> 95mmHg. Subjects may be on monotherapy with anti-hypertensive medication. 14. documented allergy, hypersensitivity, intolerance, or non-responsivity to methylphenidate or amphetamines. 15. Subject has a history of a substance use disorder (abuse or dependence, as defined by DSM-IV-TR™), with the exception of nicotine dependence, within 6 months prior to screening. 16. Subject has glaucoma 17. Subject is taking other medications that have central nervous system (CNS) effects or affect performance, such as sedating antihistamines and decongestant **Maximum Age:** 65 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Belmont **Country:** United States **Facility:** McLean Hospital **State:** Massachusetts **Zip:** 02478 #### Overall Officials **Official 1:** **Affiliation:** Mclean Hospital **Name:** J. Alexander Bodkin Bodkin, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ### References Module #### References **Citation:** Bodkin JA, Lasser RA, Wines JD Jr, Gardner DM, Baldessarini RJ. Combining serotonin reuptake inhibitors and bupropion in partial responders to antidepressant monotherapy. J Clin Psychiatry. 1997 Apr;58(4):137-45. doi: 10.4088/jcp.v58n0401. **PMID:** 9164423 **Citation:** Candy M, Jones L, Williams R, Tookman A, King M. Psychostimulants for depression. Cochrane Database Syst Rev. 2008 Apr 16;(2):CD006722. doi: 10.1002/14651858.CD006722.pub2. **PMID:** 18425966 **Citation:** Dunkin JJ, Leuchter AF, Cook IA, Kasl-Godley JE, Abrams M, Rosenberg-Thompson S. Executive dysfunction predicts nonresponse to fluoxetine in major depression. J Affect Disord. 2000 Oct;60(1):13-23. doi: 10.1016/s0165-0327(99)00157-3. **PMID:** 10940443 **Citation:** Edgar CJ, Pace-Schott EF, Wesnes KA. Approaches to measuring the effects of wake-promoting drugs: a focus on cognitive function. Hum Psychopharmacol. 2009 Jul;24(5):371-89. doi: 10.1002/hup.1034. **PMID:** 19565524 **Citation:** Fava M, Graves LM, Benazzi F, Scalia MJ, Iosifescu DV, Alpert JE, Papakostas GI. A cross-sectional study of the prevalence of cognitive and physical symptoms during long-term antidepressant treatment. J Clin Psychiatry. 2006 Nov;67(11):1754-9. doi: 10.4088/jcp.v67n1113. **PMID:** 17196056 **Citation:** Fava M, Thase ME, DeBattista C. A multicenter, placebo-controlled study of modafinil augmentation in partial responders to selective serotonin reuptake inhibitors with persistent fatigue and sleepiness. J Clin Psychiatry. 2005 Jan;66(1):85-93. doi: 10.4088/jcp.v66n0112. **PMID:** 15669893 **Citation:** Fleurence R, Williamson R, Jing Y, Kim E, Tran QV, Pikalov AS, Thase ME. A systematic review of augmentation strategies for patients with major depressive disorder. Psychopharmacol Bull. 2009;42(3):57-90. **PMID:** 19752841 **Citation:** Gorlyn M, Keilp JG, Grunebaum MF, Taylor BP, Oquendo MA, Bruder GE, Stewart JW, Zalsman G, Mann JJ. Neuropsychological characteristics as predictors of SSRI treatment response in depressed subjects. J Neural Transm (Vienna). 2008 Aug;115(8):1213-9. doi: 10.1007/s00702-008-0084-x. Epub 2008 Jul 16. **PMID:** 18629432 **Citation:** Parker RD, Flint EP, Bosworth HB, Pieper CF, Steffens DC. A three-factor analytic model of the MADRS in geriatric depression. Int J Geriatr Psychiatry. 2003 Jan;18(1):73-7. doi: 10.1002/gps.776. **PMID:** 12497559 **Citation:** Ravindran AV, Kennedy SH, O'Donovan MC, Fallu A, Camacho F, Binder CE. Osmotic-release oral system methylphenidate augmentation of antidepressant monotherapy in major depressive disorder: results of a double-blind, randomized, placebo-controlled trial. J Clin Psychiatry. 2008 Jan;69(1):87-94. doi: 10.4088/jcp.v69n0112. **PMID:** 18312042 **Citation:** Taylor BP, Bruder GE, Stewart JW, McGrath PJ, Halperin J, Ehrlichman H, Quitkin FM. Psychomotor slowing as a predictor of fluoxetine nonresponse in depressed outpatients. Am J Psychiatry. 2006 Jan;163(1):73-8. doi: 10.1176/appi.ajp.163.1.73. **PMID:** 16390892 **Citation:** Trivedi MH, Rush AJ, Wisniewski SR, Nierenberg AA, Warden D, Ritz L, Norquist G, Howland RH, Lebowitz B, McGrath PJ, Shores-Wilson K, Biggs MM, Balasubramani GK, Fava M; STAR*D Study Team. Evaluation of outcomes with citalopram for depression using measurement-based care in STAR*D: implications for clinical practice. Am J Psychiatry. 2006 Jan;163(1):28-40. doi: 10.1176/appi.ajp.163.1.28. **PMID:** 16390886 ## 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: M29705 - Name: Cognitive Dysfunction - Relevance: LOW - As Found: Unknown - 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: D000000697 - Term: Central Nervous System Stimulants - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018765 - Term: Dopamine Uptake Inhibitors - ID: D000014179 - Term: Neurotransmitter Uptake Inhibitors - ID: D000049990 - Term: Membrane Transport Modulators - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000015259 - Term: Dopamine Agents - ID: D000018377 - Term: Neurotransmitter Agents ### Intervention Browse Module - Browse Branches - Abbrev: VaCoAg - Name: Vasoconstrictor Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: CNSSti - Name: Central Nervous System Stimulants - Abbrev: CaAg - Name: Cardiotonic Agents ### Intervention Browse Module - Browse Leaves - ID: M12575 - Name: Norepinephrine - Relevance: LOW - As Found: Unknown - ID: M15512 - Name: Serotonin - Relevance: LOW - As Found: Unknown - ID: M446 - Name: Lisdexamfetamine Dimesylate - Relevance: HIGH - As Found: 124 - ID: M4029 - Name: Central Nervous System Stimulants - Relevance: LOW - As Found: Unknown - ID: M7473 - Name: Dopamine - Relevance: LOW - As Found: Unknown - ID: M20832 - Name: Dopamine Uptake Inhibitors - 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 ### Intervention Browse Module - Meshes - ID: D000069478 - Term: Lisdexamfetamine Dimesylate ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Adjunct Lisdexamfetamine (Vyvanse) **Deaths Num At Risk:** 28 **Description:** Participants receive Lisdexamfetamine Dimesylate 20-50 mg capsule each morning for 4 weeks. After a washout period of 2 weeks, they receive Placebo capsule (matching Lisdexamfetamine Dimesylate (Vyvanse) capsule) each morning for 4 weeks. Lisdexamfetamine Dimesylate (Vyvanse): Lisdexamfetamine Dimesylate (Vyvanse) capsules dose ranging from 20mg to 50 mg. **ID:** EG000 **Other Num Affected:** 23 **Other Num at Risk:** 28 **Serious Number At Risk:** 28 **Title:** Adjunct Lisdexamfetamine (Vyvanse) **Group ID:** EG001 **Title:** Adjunct Placebo **Deaths Num At Risk:** 28 **Description:** Participants receive Placebo capsule (matching Lisdexamfetamine Dimesylate (Vyvanse) 20-50 mg capsule) each morning for 4 weeks. After a washout period of 2 weeks, they receive Lisdexamfetamine Dimesylate capsule each morning for 4 weeks. Placebo: Lisdexamfetamine Dimesylate (Vyvanse)-matched placebo capsules. **ID:** EG001 **Other Num Affected:** 18 **Other Num at Risk:** 28 **Serious Number At Risk:** 28 **Title:** Adjunct Placebo **Frequency Threshold:** 5 #### Other Events **Term:** decreased appetite **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** **Term:** headache **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** **Term:** dry mouth **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** **Term:** insomnia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** **Term:** irritability **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** **Term:** anxiety **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** **Term:** upper respiratory infection **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** **Term:** fatigue **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** **Term:** gastrointesinal disturbance **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** increased activation **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** **Term:** diaphoresis **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Notes:** increased sweating **Organ System:** General disorders **Source Vocabulary:** **Term:** decreased libido **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** **Term:** stomach virus **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** **Term:** tinnitus **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** **Term:** muscle tension **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** **Term:** tachycardia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** **Term:** paresthesia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** **Time Frame:** Adverse event data were collected over the 4 years of active subject participation. ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 13 **Group ID:** BG001 **Value:** 16 **Group ID:** BG002 **Value:** 29 **Units:** Participants ### Group **ID:** BG000 **Title:** Placebo, Then Vyvanse **Description:** Participants first received Placebo capsule (matching Lisdexamfetamine Dimesylate(Vyvanse) 20-50 mg capsule) each morning for 4 weeks. After a washout period of 2 weeks, they then received Lisdexamfetamine Dimesylate capsule each morning for 4 weeks. Lisdexamfetamine Dimesylate (Vyvanse): Lisdexamfetamine Dimesylate (Vyvanse) capsules dose ranging from 20mg to 50 mg. Placebo: Lisdexamfetamine Dimesylate (Vyvanse)-matched placebo capsules. ### Group **ID:** BG001 **Title:** Vyvanse, Then Placebo **Description:** Participants first received Lisdexamfetamine Dimesylate 20-50 mg capsule each morning for 4 weeks. After a washout period of 2 weeks, they then received Placebo capsule (matching Lisdexamfetamine Dimesylate (Vyvanse) capsule) each morning for 4 weeks. Lisdexamfetamine Dimesylate (Vyvanse): Lisdexamfetamine Dimesylate (Vyvanse) capsules dose ranging from 20mg to 50 mg. Placebo: Lisdexamfetamine Dimesylate (Vyvanse)-matched placebo capsules. ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 10.6 **Value:** 52.8 #### Measurement **Group ID:** BG001 **Spread:** 11.4 **Value:** 48.9 #### Measurement **Group ID:** BG002 **Spread:** 11.0 **Value:** 50.6 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 5 #### Measurement **Group ID:** BG001 **Value:** 7 #### Measurement **Group ID:** BG002 **Value:** 12 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 8 #### Measurement **Group ID:** BG001 **Value:** 9 #### Measurement **Group ID:** BG002 **Value:** 17 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 13 #### Measurement **Group ID:** BG001 **Value:** 16 #### Measurement **Group ID:** BG002 **Value:** 29 **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:** Region of Enrollment **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement ### Limitations and Caveats **Description:** The small sample size and crossover design both weaken group differences. ### Point of Contact **Email:** [email protected] **Organization:** McLean Hospital **Phone:** 617-855-3186 **Title:** J. Alexander Bodkin, MD ## Results Section - Outcome Measures Module ### Outcome Measure 1 #### Analysis **CI Lower Limit:** **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** The statistical analysis represents a within-subject comparison of change under treatment with Vyvanse versus change under treatment with placebo, using paired t-tests with each subject as their own control. All tests reported are two-tailed. **Non-Inferiority Comment:** A Paired sample t-test was used to test the null hypothesis of no difference in MDAR score change after 4 weeks of treatment with Vyvanse versus placebo. **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** <0.05 **P-Value Comment:** **Parameter Type:** **Parameter Value:** **Statistical Comment:** **Statistical Method:** t-test, 2 sided **Tested Non-Inferiority:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 3.98 - **Upper Limit:** - **Value:** 12.57 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 3.94 - **Upper Limit:** - **Value:** 13.46 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 5.51 - **Upper Limit:** - **Value:** 9.08 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 5.54 - **Upper Limit:** - **Value:** 6.36 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 4.66 - **Upper Limit:** - **Value:** -3.49 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 5.03 - **Upper Limit:** - **Value:** -7.08 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** The Montgomery-Asberg Depression Rating Scale Dysphoric Apathy Retardation subfactor (MDAR) is a 5-item subscale of the clinician-administered 10-item Montgomery-Asberg Depression Rating Scale (MADRS). MDAR score can range from 0-30 with a higher score representing a greater severity of depressive symptoms. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** All participants who completed all 4 weeks on both treatments (i.e., Placebo and Vyvanse) were included in the analysis. **Reporting Status:** POSTED **Time Frame:** Baseline to 4 weeks of treatment **Title:** Change From Baseline in the Dysphoric Apathy/Retardation Sub-factor (MDAR) of Montgomery-Asberg Depression Rating Scale (MADRS) at 4 Weeks. **Type:** PRIMARY **Unit of Measure:** scores on a scale ##### Group **Description:** Participants first received Placebo capsule (matching Lisdexamfetamine Dimesylate(Vyvanse) 20-50 mg capsule) each morning for 4 weeks. After a washout period of 2 weeks, they then received Lisdexamfetamine Dimesylate (Vyvanse) capsule each morning for 4 weeks, starting with initial dose 30 mg/d. Lisdexamfetamine Dimesylate (Vyvanse): Lisdexamfetamine Dimesylate (Vyvanse) capsules dose ranging from 20mg to 50 mg. Placebo: Lisdexamfetamine Dimesylate (Vyvanse)-matched placebo capsules. **ID:** OG000 **Title:** Placebo Adjunct ##### Group **Description:** Participants first received Lisdexamfetamine Dimesylate (Vyvanse) 20-50 mg capsule each morning for 4 weeks, staring with initial dose 30 mg/d. After a washout period of 2 weeks, they then received Placebo capsule (matching Lisdexamfetamine Dimesylate (Vyvanse) capsule) each morning for 4 weeks. Lisdexamfetamine Dimesylate (Vyvanse): Lisdexamfetamine Dimesylate (Vyvanse) capsules dose ranging from 20mg to 50 mg. Placebo: Lisdexamfetamine Dimesylate (Vyvanse)-matched placebo capsules. **ID:** OG001 **Title:** Lisdexamfetamine Dimesylate (Vyvanse) ### Participant Flow Module #### Group **Description:** Participants first received Placebo capsule (matching Lisdexamfetamine Dimesylate(Vyvanse) 20-50 mg capsule) each morning for 4 weeks. After a washout period of 2 weeks, they then received Lisdexamfetamine Dimesylate (Vyvanse) capsule each morning for 4 weeks, starting with initial dose 30 mg/d. Lisdexamfetamine Dimesylate (Vyvanse): Lisdexamfetamine Dimesylate (Vyvanse) capsules dose ranging from 20mg to 50 mg. Placebo: Lisdexamfetamine Dimesylate (Vyvanse)-matched placebo capsules. **ID:** FG000 **Title:** Placebo, Then Vyvanse #### Group **Description:** Participants first received Lisdexamfetamine Dimesylate (Vyvanse) 20-50 mg capsule each morning for 4 weeks, staring with initial dose 30 mg/d. After a washout period of 2 weeks, they then received Placebo capsule (matching Lisdexamfetamine Dimesylate (Vyvanse) capsule) each morning for 4 weeks. Lisdexamfetamine Dimesylate (Vyvanse): Lisdexamfetamine Dimesylate (Vyvanse) capsules dose ranging from 20mg to 50 mg. Placebo: Lisdexamfetamine Dimesylate (Vyvanse)-matched placebo capsules. **ID:** FG001 **Title:** Vyvanse, Then Placebo #### Period **Title:** Overall Study ##### Withdraw **Type:** Lost to Follow-up ###### 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:** 13 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 16 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 13 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 15 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 1 **Pre-Assignment Details:** Of the 35 subjects screened, 29 were randomized. Of the 6 not randomized, 3 did not meet inclusion criteria, 1 withdrew consent, and 2 were lost to follow post screening. Of the 29 randomized, 28 completed both parts of the study. One subject was lost to follow after 3 weeks in the first half. Data were analyzed for the 28 completers only. **Recruitment Details:** 35 potential subjects were screened for eligibility between September 2010 and April 2014 at McLean Hospital in Belmont, MA. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT04668079 **Brief Title:** Modified Cross Body Stretch Verses Modified Sleeper Stretch in Basketball Players for Posterior Capsule Tightness **Official Title:** Comparison of Modified Cross Body Stretch and Modified Sleeper Stretch in Basketball Players for Posterior Capsule Tightness in Basketball Players #### Organization Study ID Info **ID:** REC/00720 Rubina Naz #### Organization **Class:** OTHER **Full Name:** Riphah International University ### Status Module #### Completion Date **Date:** 2020-12-08 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2020-12-17 **Type:** ACTUAL **Last Update Submit Date:** 2020-12-15 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2020-11-01 **Type:** ACTUAL #### Start Date **Date:** 2020-04-01 **Type:** ACTUAL **Status Verified Date:** 2020-12 #### Study First Post Date **Date:** 2020-12-16 **Type:** ACTUAL **Study First Submit Date:** 2020-12-09 **Study First Submit QC Date:** 2020-12-14 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Riphah International University #### 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 of this research is to compare the effect of modified cross body stretch and modified sleeper stretch on pain, range of motion, disability and throwing ability in athletes with posterior capsule tightness. Randomized controlled trials done at International Islamic University. The sample size was 32. The subjects were divided in two groups, 16 subjects in modified cross body stretch group and 16 in modified sleeper stretch group. Study duration was of 6 months. Sampling technique applied was non probability connivance sampling technique. Tools used in the study are PENN shoulder score, gonoiometer, thumb up back(TUB) and seated basketball throw test(SBBT). Data was be analyzed through SPSS 21. **Detailed Description:** Basketball is considered as the highly demanding sports due to high demand and complexity of the game. The overhead throwing activities makes it more vulnerable to the injuries. In repetitive throwing there are different type of violet forces placed on shoulder joint It causes adaptation in soft tissue anatomically which in turn limits ROM of shoulder joint and Posterior shoulder tightness. Posterior capsule tightness can result due to abnormal humeral head motion which decreases subacromial space during overhead activities leading to compression of tissues causing limited shoulder flexion, internal rotation and horizontal adduction. Posterior capsule tightening occurs when the capsular tissue and musculature of the shoulder tighten, usually due to "repeated overload in the eccentric portion of arm deceleration. This loss of internal rotation results from contracture and tightening of the posterior inferior portion of the glenohumeral joint capsule, which occurs from the repetitive microtrauma imparted during the deceleration phase of the throwing motion. Repetitive throwing motion creates adaptive increased external rotation and decreased internal rotation in the dominant shoulder joint which is termed as GIRD (Glenohumeral internal rotation deficit).Posterior capsule tightness is often treated without surgery. It can be treated by strengthening, stretching, neuromuscular control exercises. Strengthening protocol can perform specifically on Weak muscles by using resistance and weight. The neuromuscular training helps the body to act and react on different pattern of stress demand on it through neural pathways. The mainstay of the posterior capsule tightness treatment for athlete is the stretching of the posterior capsule. One author proposed the modified forms, the modified sleeper stretch and the modified cross body stretch. And found these modified stretching more effective and beneficial than the sleeper and cross body stretch Literature review: Kevin et al investigated the acute effects of sleeper stretch on posterior shoulder tightness and internal rotation of shoulder. There was significant improvement in results. A study conducted in 2018 to determine whether posterior shoulder stretch was effective in increasing internal rotation and horizontal adduction ROM in volleyball and tennis players with internal rotation deficit \>¬¬15 degrees. The intervention group performs the sleeper stretch daily for 8 weeks. Results showed significant improvement in internal rotation and horizontal adduction. Another article was published in 2014, with the aim to compare the effects of horizontal adduction stretch with scapular stabilization versus horizontal adduction stretch without scapular stabilization on posterior shoulder tightness and passive internal rotation. Results showed significant improvement among the scapular stabilization group. A RCT conducted in which each subject completed stretching interventions for two days. Purpose of the study was to investigate acute effects of modified sleeper stretch and modified cross body stretch on posterior shoulder tightness and glenohumral internal rotation deficit with more than 10-degree loss. ### Conditions Module **Conditions:** - Shoulder Capsulitis **Keywords:** - Modified cross body stretch - Modified sleeper stretch - Posterior capsule tightness - Range of motion - Seated basketball throw - Thumb up back ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 32 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** modified cross body stretch **Intervention Names:** - Other: Modified cross body stretch **Label:** Modified cross body stretch **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** modified sleeper stretch **Intervention Names:** - Other: Modified sleeper stretch **Label:** Modified sleeper stretch **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Modified cross body stretch **Description:** Athlete was in side lying positioned. trunk was moved posteriorly 200 to 300 and shoulder is raised to 900 and semi flex both knees. Athlete was ask to passively pull the humerus across the body into horizontal adduction with the opposite hand. 5 reps once daily 3 sets for 4 weeks was given **Name:** Modified cross body stretch **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - Modified sleeper stretch **Description:** Athlete was in side lying positioned on the involved side, trunk was rolled posteriorly 200 to 300 and shoulder is raised to 900 and elbow is flexed to 900 and semiflex both knees. athlete was ask to perform internal rotation passively by using the opposite arm. 5 reps once daily 3 sets for 4 weeks was given **Name:** Modified sleeper stretch **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** The disability of shoulder conditions was measured by Penn score. Which consists of 3 categories i.e.; pain, satisfaction and functional ability. Pain consists of 3 questionnaires that consists of 10 scores each (total 30) and then satisfaction, it consists of 1 questionnaire consists of 10 scores, functional ability consists of 20 questionnaires with 10 scores each for the questionnaire (total 60) **Measure:** PENN shoulder score **Time Frame:** 4th week **Description:** SBBT (seated basketball throws) was used to test the explosiveness of the upper extremity. **Measure:** Seated basketball throw **Time Frame:** 4th week **Description:** (Thumb-up-the back- measurement) was used to measure the ability to actively move thumb up the back with the help of inch tape. **Measure:** Thumb up back(TUB) **Time Frame:** 4th week #### Secondary Outcomes **Description:** Changes from the Baseline ROM range of Motion of Shoulder flexion was taken with the Help of Goniometer **Measure:** Shoulder ROM (flexion) **Time Frame:** 4th week **Description:** Changes from the Baseline ROM range of Motion of Shoulder extension was taken with the Help of Goniometer **Measure:** Shoulder ROM Extension **Time Frame:** 4th week **Description:** Changes from the Baseline ROM range of Motion of shoulder abduction was taken with the Help of Goniometer **Measure:** Shoulder ROM abduction **Time Frame:** 4th week **Description:** Changes from the Baseline ROM range of Motion of shoulder adduction was taken with the Help of Goniometer **Measure:** Shoulder ROM adduction **Time Frame:** 4th week **Description:** Changes from the Baseline ROM range of Motion of Shoulder external rotation was taken with the Help of Goniometer **Measure:** Shoulder ROM external rotation **Time Frame:** 4th week **Description:** Changes from the Baseline ROM range of Motion of Shoulder internal rotation was taken with the Help of Goniometer **Measure:** Shoulder ROM internal rotation **Time Frame:** 4th week ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Basketball Players * Internal rotation deficit greater than 20° degrees * BMI 18.5-24.9 Exclusion Criteria: * Having a systemic pathology including inflammatory joint disease * Having musculoskeletal or neurological disease * Having taken anti-inflammatory medication **Maximum Age:** 30 Years **Minimum Age:** 15 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Islamabad **Country:** Pakistan **Facility:** Islamic international medical college **State:** Punjab **Zip:** 46000 #### Overall Officials **Official 1:** **Affiliation:** Riphah International University **Name:** Aisha Razzaq, MSPT-OMPT **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Vukasevic V, Mitrovic M, Masanovic B. A comparative study of motor ability between elite basketball players from different regions. Sport Mont. 2020;18(1):Ahead of Print **Citation:** Ekhtiari S, Khan M, Burrus T, Madden K, Gagnier J, Rogowski JP, Maerz T, Bedi A. Hip and Groin Injuries in Professional Basketball Players: Impact on Playing Career and Quality of Life After Retirement. Sports Health. 2019 May/Jun;11(3):218-222. doi: 10.1177/1941738119838274. Epub 2019 Apr 23. **PMID:** 31013191 **Citation:** Drakos MC, Domb B, Starkey C, Callahan L, Allen AA. Injury in the national basketball association: a 17-year overview. Sports Health. 2010 Jul;2(4):284-90. doi: 10.1177/1941738109357303. **PMID:** 23015949 **Citation:** Quartey J, Davor SF, Kwakye SK. An injury profile of basketball players in Accra, Ghana. S Afr J Physiother. 2019 Mar 27;75(1):467. doi: 10.4102/sajp.v75i1.467. eCollection 2019. **PMID:** 31061911 **Citation:** Grow K. The Sleeper Stretch: Effects on Range of Motion and Injury in Baseball Players. 2010 **Citation:** McClure P, Balaicuis J, Heiland D, Broersma ME, Thorndike CK, Wood A. A randomized controlled comparison of stretching procedures for posterior shoulder tightness. J Orthop Sports Phys Ther. 2007 Mar;37(3):108-14. doi: 10.2519/jospt.2007.2337. **PMID:** 17416125 **Citation:** Rao MS, Tejitha S. COMPARISON OF 3 STRETCHING PROTOCOLS FOR POSTERIOR SHOULDER TIGHTNESS IN THROWERS. Int J Physiother Res. 2016;4(2):1429-35 **Citation:** Myers JB, Laudner KG, Pasquale MR, Bradley JP, Lephart SM. Glenohumeral range of motion deficits and posterior shoulder tightness in throwers with pathologic internal impingement. Am J Sports Med. 2006 Mar;34(3):385-91. doi: 10.1177/0363546505281804. Epub 2005 Nov 22. **PMID:** 16303877 **Citation:** Laudner KG, Sipes RC, Wilson JT. The acute effects of sleeper stretches on shoulder range of motion. J Athl Train. 2008 Jul-Aug;43(4):359-63. doi: 10.4085/1062-6050-43.4.359. **PMID:** 18668168 **Citation:** Chepeha JC, Magee DJ, Bouliane M, Sheps D, Beaupre L. Effectiveness of a Posterior Shoulder Stretching Program on University-Level Overhead Athletes: Randomized Controlled Trial. Clin J Sport Med. 2018 Mar;28(2):146-152. doi: 10.1097/JSM.0000000000000434. **PMID:** 28731885 **Citation:** Salamh PA, Kolber MJ, Hanney WJ. Effect of scapular stabilization during horizontal adduction stretching on passive internal rotation and posterior shoulder tightness in young women volleyball athletes: a randomized controlled trial. Arch Phys Med Rehabil. 2015 Feb;96(2):349-56. doi: 10.1016/j.apmr.2014.09.038. Epub 2014 Oct 23. **PMID:** 25450120 **Citation:** Immediate effects of two types of stretching techniques on glenohumeral internal rotation deficit and posterior shoulder tightness; a crossover randomised controlled trial ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007592 - Term: Joint Diseases - ID: D000009140 - Term: Musculoskeletal Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M5332 - Name: Bursitis - Relevance: HIGH - As Found: Capsulitis - ID: M10621 - Name: Joint Diseases - Relevance: LOW - As Found: Unknown - ID: M12097 - Name: Musculoskeletal Diseases - Relevance: LOW - As Found: Unknown - ID: T4202 - Name: Oculocerebral Syndrome With Hypopigmentation - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000002062 - Term: Bursitis ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03932279 **Brief Title:** Characterization of the Microbiome in Cutaneous T Cell Lymphoma **Official Title:** Characterization of the Microbiome in Cutaneous T Cell Lymphoma #### Organization Study ID Info **ID:** XZ05212018 #### Organization **Class:** OTHER **Full Name:** Northwestern University ### Status Module #### Completion Date **Date:** 2028-08-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-03-12 **Type:** ACTUAL **Last Update Submit Date:** 2024-03-08 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2028-02-28 **Type:** ESTIMATED #### Start Date **Date:** 2019-01-30 **Type:** ACTUAL **Status Verified Date:** 2024-03 #### Study First Post Date **Date:** 2019-04-30 **Type:** ACTUAL **Study First Submit Date:** 2019-02-18 **Study First Submit QC Date:** 2019-04-28 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Northwestern University #### Responsible Party **Investigator Affiliation:** Northwestern University **Investigator Full Name:** Xiaolong (Alan) Zhou **Investigator Title:** Principal Investigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Investigators plan to perform a pilot study that aims to characterize the microbiome of human cutaneous T cell lymphoma patients and compare this to the microbiome of age and sex matched controls. ### Conditions Module **Conditions:** - Cutaneous T Cell Lymphoma ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 300 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Label:** Stage IA-IIA cutaneous T cell lymphoma #### Arm Group 2 **Label:** Stage IIB and above cutaneous T cell lymphoma #### Arm Group 3 **Label:** CD30+ lymphoproliferative disorders #### Arm Group 4 **Label:** Plaque psoriasis with BSA>5% on routine phototherapy #### Arm Group 5 **Label:** Moderate to severe atopic dermatitis on routine bleach bath #### Arm Group 6 **Label:** Healthy controls ### Outcomes Module #### Primary Outcomes **Description:** Diversity analysis of microbiome samples (measured by number of bacteria species/sample) **Measure:** Bacterial diversity index **Time Frame:** 3 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Group 1: Patients with stage IA-IIA cutaneous T cell lymphoma * Group 2: Patients with stage IIB and above cutaneous T cell lymphoma * Group 3: Patients with CD30+ lymphoproliferative disorder including lymphomatoid papulosis and cutaneous anaplastic large cell lymphoma * Group 4: Patients with plaque psoriasis with BSA\>5% on routine phototherapy per standard of care * Group 5: Patients with moderate to severe atopic dermatitis on routine bleach bath therapy per standard of care * Group 6: Healthy individuals without the above skin conditions, similar age and sex distribution to the patients with cutaneous T cell lymphoma * All Groups: subjects who are age 18-89 years of age at time of enrollment * All Groups: Subjects who are able and willing to give informed consent for this study and the Dermatology Tissue Acquisition and Biorepository (STU00009443). Exclusion Criteria: * All Groups: Subjects who are younger than 18 years of age or older than 90 years of age * All Groups: Subjects who are unable to give consent * Patients currently on systemic antibiotics or recent (within past 4 weeks) exposure to systemic antibiotics * We will not recruit the following populations: adults unable to consent, individuals who are not yet adults (infants, children, teenagers), pregnant women, prisoners and other vulnerable populations. **Healthy Volunteers:** True **Maximum Age:** 89 Years **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Patients with cutaneous T cell lymphoma, atopic dermatitis, and psoriasis and healthy individuals without any of these conditions will be enrolled at Northwestern Memorial Hospital Dermatology outpatient clinic. ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Dermatology CTU **Phone:** 312-503-5944 **Role:** CONTACT #### Locations **Location 1:** **City:** Chicago **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Dermatology CTU - **Phone:** 312-503-5944 - **Role:** CONTACT ***Contact 2:*** - **Name:** Alan Zhou, MD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Northwestern University **State:** Illinois **Status:** RECRUITING **Zip:** 60611 #### Overall Officials **Official 1:** **Affiliation:** Northwestern University **Name:** Alan Zhou, MD **Role:** PRINCIPAL_INVESTIGATOR ## 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 - ID: D000008228 - Term: Lymphoma, Non-Hodgkin ### 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: HIGH - As Found: Lymphoma - ID: M18829 - Name: Lymphoma, T-Cell - Relevance: HIGH - As Found: T-cell Lymphoma - ID: M18833 - Name: Lymphoma, T-Cell, Peripheral - Relevance: HIGH - As Found: T-cell Lymphoma - ID: M18832 - Name: Lymphoma, T-Cell, Cutaneous - Relevance: HIGH - As Found: Cutaneous T-Cell 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: M11222 - Name: Lymphoma, Non-Hodgkin - Relevance: LOW - As Found: Unknown - ID: T3543 - Name: Lymphosarcoma - Relevance: HIGH - As Found: Lymphoma - ID: T4496 - Name: Peripheral T-cell Lymphoma - Relevance: HIGH - As Found: T-cell Lymphoma - ID: T1689 - Name: Cutaneous T-cell Lymphoma - Relevance: HIGH - As Found: Cutaneous T-Cell Lymphoma ### Condition Browse Module - Meshes - ID: D000008223 - Term: Lymphoma - ID: D000016399 - Term: Lymphoma, T-Cell - ID: D000016411 - Term: Lymphoma, T-Cell, Peripheral - ID: D000016410 - Term: Lymphoma, T-Cell, Cutaneous ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02819479 **Acronym:** LIBERTI **Brief Title:** Low-dose Intra-arterial Bevacizumab for Edema and Radiation Necrosis Therapeutic Intervention (LIBERTI) **Official Title:** Low-dose Intra-arterial Bevacizumab for Edema and Radiation Necrosis Therapeutic Intervention (LIBERTI) #### Organization Study ID Info **ID:** 14-N0229 #### Organization **Class:** OTHER **Full Name:** Norton Healthcare ### Status Module #### Completion Date **Date:** 2019-06-06 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2022-05-24 **Type:** ACTUAL **Last Update Submit Date:** 2022-05-02 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2019-06-06 **Type:** ACTUAL #### Results First Post Date **Date:** 2021-06-28 **Type:** ACTUAL **Results First Submit Date:** 2020-10-16 **Results First Submit QC Date:** 2021-06-25 #### Start Date **Date:** 2016-11-23 **Type:** ACTUAL **Status Verified Date:** 2022-05 #### Study First Post Date **Date:** 2016-06-30 **Type:** ESTIMATED **Study First Submit Date:** 2016-05-09 **Study First Submit QC Date:** 2016-06-27 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** University of Kentucky #### Lead Sponsor **Class:** OTHER **Name:** Norton Healthcare #### Responsible Party **Investigator Affiliation:** Norton Healthcare **Investigator Full Name:** Shervin Dashti, MD **Investigator Title:** Principal Investigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** To assess the overall safety and efficacy of intra-arterial (IA) bevacizumab for the treatment of radiation necrosis. A single 2.5 mg/kg dose of bevacizumab will be given intra-arterially after osmotic blood-brain-barrier disruption. **Detailed Description:** BACKGROUND Radiation Necrosis: Stereotactic radiosurgery has become integral in treatment of brain tumors and arteriovenous malformations (AVM). In up to 10% of cases, this can lead to radiation necrosis (RN) with significant surrounding vasogenic edema and mass effect. Medical treatment for RN includes steroids, vitamin E, pentoxiphylline, and hyperbaric oxygen. Up to 20% of cases however, are medically refractory and experience progressive neurological decline and disabling headaches. Surgical resection and laser interstitial thermal therapy (LITT) are sometimes used for treatment of medically refractory radiation necrosis. These invasive options carry risks of serious complications such as infection, seizures, neurological deficits, and hemorrhage, and may have a failure rate as high as 39% in patients with radiation necrosis of the brain. Bevacizumab: Bevacizumab (Avastin, Genentech BioOncology, South San Francisco, CA) is a recombinant humanized version of a murine anti-human vascular endothelial growth factor (VEGF) monoclonal antibody. Recently, bevacizumab was shown in a small randomized controlled trial (n=14) to be effective in treatment of refractory radiation necrosis after radiation therapy in brain tumors1. Patients received 7.5 mg/kg IV-Bevacizumab every 3 weeks for 4 cycles. All patients receiving Bevacizumab and none of the patients receiving placebo had significant clinical and radiographic improvement. PRE-CLINICAL DATA Role of vascular endothelial growth factor (VEGF) in radiation necrosis VEGF has been implicated in the pathophysiology of radiation necrosis. Reactive astrocytes immediately surrounding the necrotic core in RN are strongly VEGF-positive by immunohistochemistry. It is postulated that radiation causes microvascular injury leading to hypoxia. Hypoxia-induced VEGF up-regulation then drives an increase in vascular permeability, leading to the extensive vasogenic edema seen in RN. Bevacizumab binds circulating VEGF receptors with high specificity, blocking the down-stream signaling cascade. CLINICAL DATA: Bevacizumab was originally developed and tested as an anti-angiogenic treatment for various solid tumors. More recently, IV-Bevacizumab was shown in a small, randomized controlled trial (n=14) to be very effective in treatment of refractory radiation necrosis after radiation therapy in brain tumors1. Patients received 7.5 mg/kg IV-Bevacizumab every 3 weeks for 4 cycles. All patients receiving Bevacizumab and none of the patients receiving placebo had significant clinical and radiographic improvement. This improvement was durable at 10 months in 8 of 11 patients (4 patients crossed over from the control group). There was however, a very high rate of adverse events (60%), and major adverse events (30%). Major adverse events included venous sinus thrombosis, pulmonary embolus, and aspiration pneumonia. The investigators of the present study recently published a case series of two pediatric patients with highly symptomatic steroid refractory radiation necrosis in the brain after stereotactic radiosurgery for treatment of cerebral arteriovenous malformations3. Both patients were refractory to all accepted medical therapies. Both were steroid dependent for a prolonged period and severely cushingoid. Both had suffered a significant decline in quality of life with severe headache and needed to withdraw from school. In both instances, the patients made a remarkable progressive clinical and radiographic improvement after receiving a single 2.5 mg/kg dose of intra-arterial bevacizumab, which was durable one-year later. To increase bevacizumab penetration into the brain, the investigators used intra-arterial Mannitol to disrupt the blood-brain barrier immediately prior to targeted intra-arterial drug administration. RATIONALE: RATIONALE: CURRENT IV BEVACIZUMAB REGIMEN FOR RADIATION NECROSIS AND ITS ASSOCIATED MORBIDITY: Current IV-bevacizumab regimens use a dose of 7.5 mg/kg every 3 weeks for 4 cycles. There are significant known side effects of bevacizumab including gastrointestinal perforation, deep venous thrombosis, venous sinus thrombosis, pulmonary embolus, intracranial hemorrhage, wound dehiscence, and severe hypertension. These complications are common to the anti-angiogenic class of drugs and reflect systemic exposure to bevacizumab. In our initial clinical experience, the investigators utilized a combination of intra-arterial (IA) route of delivery and BBB disruption to reduce bevacizumab dose while maintaining efficacy. This is supported by the durable clinical and radiographic response in our patients after a single 2.5 mg/kg dose of bevacizumab. The investigators believed that this approach would reduce the incidence of serious systemic toxicities compared to the IV-bevacizumab regimens (7.5-15 mg/kg every 2-3 weeks for several weeks to months). There are multiple recent reports of patients with radiation necrosis who improved with IV-bevacizumab, only to relapse months later. In fact 3/11 patients in the randomized controlled trial discussed above required repeat treatment with IV-bevacizumab because of RN symptom progression1. In contrast, the two patients in our series who received IA-bevacizumab continue to show progressive clinical and radiographic improvement more than one year later. The investigators believe that the increased penetration of bevacizumab into the brain because of the intra-arterial administration after blood-brain barrier disruption results in binding of virtually all VEGF molecules. The fact that the results are durable and progressively improving suggests that massive blocking of VEGF activity could have stopped a positive feedback loop of inflammation. Therefore, IA-bevacizumab may result in more effective and durable control of radiation necrosis compared to traditional IV-bevacizumab treatment. RATIONALE: INTRA-ARTERIAL (IA) ROUTE OF BEVACIZUMAB ADMINISTRATION SIGNIFICANTLY INCREASES DRUG DELIVERY TO THE BRAIN: IA-therapy decreases volume dilution of the drug in the circulation and reduces first-pass degradation via proteolytic catabolism, resulting in higher drug delivery to target brain tissue. Super-selective IA-injection of 99mTc-HMPAO (Ceretec®) into human cerebral arteries achieves a concentration of radiotracer in brain tissue 50 times higher than with IV injection. In clinical studies of cerebral chemotherapy, the concentration delivered to the tumor by using intra-arterial injection versus intravenous administration of chemotherapeutic agents was five times higher with hydrosoluble drugs and up to 50 times higher with liposoluble drugs. The investigators will infuse bevacizumab in the artery that supplies the territory affected by RN, such as cervical internal carotid artery and/or cervical vertebral artery. RATIONALE: BLOOD-BRAIN-BARRIER BREAKDOWN PRIOR TO INTRA-ARTERIAL THERAPY FURTHER ENHANCES DRUG DELIVERY TO THE BRAIN: The blood-brain-barrier is a selective permeability barrier that block entry of many drugs into the brain. Bevacizumab is a monoclonal antibody with a high molecular weight (149 kDa). There is convincing evidence in the literature that the concentration in the brain of high molecular weight molecules can be significantly increased after osmotic BBB disruption. Several tumor clinical trials have shown that localization of monoclonal antibodies to the brain is poor without BBB disruption (0.0006%-0.0043% of the injected dose/g of tumor). There is also evidence of a 20-fold increase in permeability to immunoreactive IgM Mab with BBB disruption in rats. The investigators believe that using blood-brain-barrier disruption significantly increases delivery of Bevacizumab to the affected brain. The investigators will use the protocol described by Neuwelt and colleagues, using infusion of 25% Mannitol over 30 seconds. This protocol has been shown to temporarily disrupt the blood brain barrier, peaking at 15 minutes and dissipating in 4 hours. IA-chemotherapy following BBBD has been shown to be feasible and safe across multiple centers with low incidence of complications27. The efficacy and safety profile was reproducible across multiple centers. In fact, safety of this protocol has been established in more than 6000 patients treated worldwide with BBBD for intra-arterial chemotherapy infusion. The main possible complication is seizure, which occurs in \<6% of cases. It is important to note that these seizures generally occurred in patients with widespread malignant pathology such as Glioblastoma and CNS lymphoma who were treated with very toxic chemotherapy agents immediately after BBBD. Recent refinements to the osmotic BBBD protocol have incorporated the use of general anesthesia, as well as prophylaxis with an anti-epileptic agent and Valium to reduce seizure threshold and the chance of seizures. SAFETY OF CEREBRAL INTRA-ARTERIAL BEVACIZUMAB TREATMENT: Safety of IA-Bevacizumab treatment after hyperosmotic BBBD was recently established in a series of malignant glioma patients. This was done through super-selective injection of intracranial tumor arterial pedicles for purpose of anti-tumor effects. Dose-escalation was performed from 2 mg/kg to 15 mg/kg without reaching maximal tolerated dose. There was a significant decrease in the contrast enhancing and FLAIR signal characteristics of the tumor and surrounding brain at one month after treatment. Overall toxicity for this cohort was comparable to previous reports for IV Bevacizumab therapy. Specifically, hyperosmotic BBB-breakdown followed by IA-Bevacizumab administration did not cause any direct neurotoxicity; there were no cases of intracranial hemorrhage. Multiple other reports of BBBD followed by intra-arterial bevacizumab treatment for other pathologies such as vestibular schwannoma, ependymoma, and malignant brainstem glioma have also demonstrated good safety profile with no obvious neurotoxicity. TREATMENT PLAN: VASCULAR ACCESS, CEREBRAL ANGIOGRAM, AND OSMOTIC BLOOD-BRAIN-BARRIER DISRUPTION: The investigators will use the protocol described by Neuwelt and colleagues, using infusion of 25% Mannitol over 30 seconds. The safety of this protocol has been established in more than 6000 patients treated worldwide with BBBD for intra-arterial chemotherapy infusion. The patients are to be premedicated with 6 mg Dexamethasone and 1000 mg Keppra. General endotracheal anesthesia will be induced. The femoral artery will be accessed using the Seldinger technique. A 5-French diagnostic catheter will be used to catheterize the cervical internal carotid artery ipsilateral to the area of radiation necrosis. Baseline internal carotid angiogram will be performed. The anesthesiologist will be instructed to maintain SBP \>120 or at pre-operative baseline, whichever value is higher. This is important for efficient bulk flow of drug through the blood brain barrier opening. The catheter is positioned at C1-2 level in the cervical internal carotid artery and C6-7 for a vertebral artery infusion. Optimal rate of Mannitol infusion will be determined by performing injection of contrast at 4 ml/sec for 3 seconds into vessel. If there is no reflux of contrast into the external carotid artery, the injection rate injection will be increased by 2 ml/sec to maximum of 12 ml/sec. The lowest rate at which there is reflux into the external carotid artery will be chosen (the rate to just exceed cerebral blood flow. Next, 5 mg IV Valium and 0.2 mg IV Atropine are to be administered. Warm (37 degrees Co) 25% Mannitol is filtered through a 5-micron filter, and then infused into the ipsilateral cervical carotid artery at the rate determined above for a total of 30 seconds. INTRA-ARTERIAL BEVACIZUMAB ADMINISTRATION: Test injection of contrast will be done in the artery. If there is any evidence of catheter-induced vasospasm, the catheter may be withdrawn more proximally within the artery. Repeat test injection of contrast will be done to document resolution of vasospasm. Within 5 minutes of Mannitol infusion, 2.5 mg/kg bevacizumab in a volume of 100 ml will be administered into the artery over 10 minutes. Repeat angiogram will be performed to document BBBD, as well as to rule out thromboembolic phenomenon. ### Conditions Module **Conditions:** - Radiation Necrosis **Keywords:** - Radiation necrosis - Radiation adverse event - Intra-arterial chemotherapy - Bevacizumab - Blood brain barrier disruption - Blood brain barrier breakdown - Arteriovenous malformation - Stereotactic radiosurgery ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 10 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** A single intra-arterial targeted dose of 2.5 mg/kg bevacizumab will be administered after osmotic blood-brain-barrier disruption with intra-arterial 25% mannitol at rate of 4-12 ml/sec for 30 seconds. **Intervention Names:** - Drug: 25% Mannitol - Drug: Low-dose Intra-arterial Bevacizumab **Label:** Low-dose Intra-arterial Bevacizumab **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Low-dose Intra-arterial Bevacizumab **Description:** Route of administration: In this study, the first step of the treatment will be performing osmotic blood-brain-barrier disruption with administration of intra-arterial 25% Mannitol into the appropriate cervical artery. **Name:** 25% Mannitol **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Low-dose Intra-arterial Bevacizumab **Description:** Route of administration: In this study, the second step of the treatment will be administering intra-arterial targeted bevacizumab into the appropriate cervical artery. **Name:** Low-dose Intra-arterial Bevacizumab **Other Names:** - Avastin **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Imaging response to therapy will be quantitatively assessed on MRI using volumetric analysis. Regions of T2 and FLAIR prolongation above contralateral white matter will be calculated and quantified in cubic centimeters. Region of interest (ROI) will be created using a semi-automated, thresholding and region-growing technique. Enhancement of the lesion will be calculated using similar volumetric ROI analysis with a contrast threshold of 40% above background and measured in cubic centimeters. **Measure:** Change in Radiation Necrosis and Cerebral Edema After a Single Treatment of Low Dose Intra-arterial Avastin (Bevacizumab) **Time Frame:** Baseline (before treatment), and 3 months and 12 months after single dose intra-arterial Avastin (bevacizumab) #### Secondary Outcomes **Description:** We will determine how severely headache affect the patient's life before and after treatment by performing the Migraine Disability Assessment (MIDAS) questionnaire. MIDAS TOTAL SCORE is the sum of Items 1 through 5. A Total Score of 0-5 signifies little or no disability, score of 6-10 signifies mild disability, score of 11-20 signifies moderate disability, and score of 21+ signifies severe disability. The theoretical maximum score would be 450. **Measure:** Change in Headache Associated Morbidity Measured With MIDAS TOTAL SCORE After a Single Treatment of Low Dose Intra-arterial Avastin (Bevacizumab) Will be Measured. **Time Frame:** Baseline (before treatment), and 6 weeks, 3 months, 6 months, 9 months, and 12 months after single dose intra-arterial Avastin (bevacizumab) **Description:** We will determine how severely headache affect the patient's life before and after treatment by performing the Migraine Disability Assessment (MIDAS) questionnaire. MIDAS DAYS of HEADACHE is Item 6 of the questionnaire which sums total number of days the patient had headache symptoms over the past 3 months regardless of headache severity or resultant disability. Min score of 0 days is the best possible score. Max Score of 90 days is worse possible score, meaning the patient experienced headache pain every day over past 3 months. **Measure:** Change in Headache Associated Morbidity Measured With MIDAS DAYS of HEADACHE After a Single Treatment of Low Dose Intra-arterial Avastin (Bevacizumab) Will be Measured. **Time Frame:** Baseline (before treatment), and 6 weeks, 3 months, 6 months, 9 months, and 12 months after single dose intra-arterial Avastin (bevacizumab) **Description:** We will determine how severely headache affect the patient's life before and after treatment by performing the Migraine Disability Assessment (MIDAS) questionnaire. MIDAS PAIN LEVEL is Item 7 which rates the 3-month average headache PAIN LEVEL on scale from 0 to 10 where 0 = no pain at all, and 10 = pain as bad as it can be. **Measure:** Change in Headache Associated Morbidity Measured With MIDAS PAIN LEVEL After a Single Treatment of Low Dose Intra-arterial Avastin (Bevacizumab) Will be Measured. **Time Frame:** Baseline (before treatment), and 6 weeks, 3 months, 6 months, 9 months, and 12 months after single dose intra-arterial Avastin (bevacizumab) **Description:** Quantitative change in headache will be assessed by performing the Headache Impact Test (HIT-6), which is a fixed-length 6-item questionnaire. The score for this questionnaire can range from 36 to 78, with 36 indicating minimum headache impact and the max score of 78 indicating worst possible headache impact. **Measure:** Change in Headache Measured With Headache Impact Test (HIT-6) After a Single Treatment of Low Dose Intra-arterial Avastin (Bevacizumab) **Time Frame:** Baseline (before treatment), and 6 weeks, 3 months, 6 months, 9 months, and 12 months after single dose intra-arterial Avastin (bevacizumab) **Description:** Quantitative change in functional status will be assessed by performing Karnofsky Performance Status Scale (KPS). The KPS score can range from 0 to 100 where 0 is dead and 100 is fully alive and normal with no complaints and no evidence of disease. **Measure:** Change in Functional Status After a Single Treatment of Low Dose Intra-arterial Bevacizumab **Time Frame:** Baseline (before treatment), Day 1, and at 3 months, and 12 months after single dose intra-arterial Avastin (bevacizumab) **Description:** To assess the utility of intra-arterial (IA) bevacizumab treatment in allowing decreased steroid usage, total cumulative days of steroid usage were compared between the 12 months PRIOR TO and the 12 months IMMEDIATELY FOLLOWING IA bevacizumab. Days of steroid usage were tabulated via medical history and chart review at the baseline visit for the 12 months prior to IA bevacizumab and post-treatment on days 0 and 1, week 6, and months 3, 6, 9 and 12 for the 12 month total after IA bevacizumab treatment. Excluding topical steroid preparations, all days of enteral or parenteral steroid intake of any dose were included in the cumulative summation. **Measure:** Change in Steroid Usage After a Single Treatment of Low Dose Intra-arterial Avastin (Bevacizumab) **Time Frame:** 12 months prior to single dose of IA bevacizumab; 12 months following single dose of IA bevacizumab **Description:** In order to investigate post-operative changes in Neurocognitive performance after intra-arterial bevacizumab, patients who consented underwent formal neuropsychological battery testing. Sixteen subtests from the Neuropsychological Assessment Battery (Stern \& White, PAR Inc.) were chosen for brevity, sensitivity, and due to the wide range of available normative data (ages 18-97). For each subtest, T-score = 50 is the normative mean with SD = 10. Thus T-scores of 40-60 are within one standard deviation of the mean and are considered generally normal. Scores increasingly below 40 indicate decreased neurocognitive performance compared to healthy demographically matched persons. Scores significantly above 60 indicate increase neurocognitive performance compared to healthy demographically matched persons. **Measure:** Post-operative Neurocognitive Change After Intra-arterial Bevacizumab **Time Frame:** Baseline (before bevacizumab), and 3 months and 12 months after single dose intra-arterial Avastin (bevacizumab) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: Patients must have radiation necrosis based on radiographic evidence defined as: * Increased T1 contrast enhancement in the radiated area with central hypointensity * Increased surrounding vasogenic edema on FLAIR MRI images * The underlying lesion prompting the radiation can include: Benign lesions such as AVM, Meningioma, schwannoma, trigeminal neuralgia: No biopsy is necessary * Radiation necrosis must be symptomatic, including either severe headache, seizures, or neurological deficits. * Radiation necrosis must be refractory to steroid treatment; defined as failing a 3-week steroid regiment or not tolerating steroids because of side effects. Beyond 3 weeks, the side effects of steroid therapy worsen rapidly. The patient may receive other therapies such as Vitamine E, Pentoxyfylline, and hyperbaric oxygen during the trial. Other inclusion criteria include: * Age \>18 years. * Ability to understand and the willingness to sign a written informed consent document. * Both men and women and members of all races and ethnic groups are eligible for this trial. * Karnofsky Performance Status \> or = 70%. * Life expectancy of greater than 3 months. * Patients must have normal organ and marrow function as defined below: leukocytes greater than equal to1,500/mcL platelets greater than equal to 85,000/mcL creatinine less than equal to 1.8 mg/dl •Birth Control: The effects of Bevacizumab on the developing human fetus are unknown. For this reason, women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control; abstinence) prior to study entry and for the duration of study participation. Should a woman become pregnant or suspect she is pregnant while participating in this study, she should inform her treating physician immediately. Women of childbearing age will have a urine pregnancy test immediately before each IA Bevacizumab treatment. Exclusion Criteria: * Patients may not be started on any other investigational agents during the course of this trial. They may however continue previous medical regiments aimed for treatment of radiation necrosis. These include steroids, vitamin E, pentoxiphylline, and hyperbaric oxygen. We feel that these treatments are generally ineffectual and would not confound the results. * Malignant brain tumor * Concomitant use of anticoagulation agents including Coumadin, anticoagulation dose Lovenox or Arixtra. Aspirin is acceptable. * Active bleeding or pathological condition that carries high risk of bleeding. * Abdominal fistula, abscess, or gastrointestinal tract perforation 28 days of study entry. * Uncontrolled intercurrent illness including, but not limited to ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements. * Any major surgery in the prior 4 weeks. Also any major surgery expected to be performed in the ensuing 4 weeks after treatment. * Pregnant women are excluded from this study because Bevacizumab is expected to disrupt angiogenesis during pregnancy with the potential for teratogenic or abortive effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with Bevacizumab, breastfeeding should be discontinued if the mother is treated with Bevacizumab. * HIV-positive patients on combination antiretroviral therapy are ineligible because of the potential for pharmacokinetic interactions with Bevacizumab. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Lexington **Country:** United States **Facility:** University of Kentucky Medical Center **State:** Kentucky **Zip:** 40536 **Location 2:** **City:** Louisville **Country:** United States **Facility:** Norton Brownsboro Hospital **State:** Kentucky **Zip:** 40242 #### Overall Officials **Official 1:** **Affiliation:** Norton Healthcare **Name:** Shervin R Dashti, MD,PhD **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** University of Kentucky **Name:** Justin Fraser, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Dashti SR, Kadner RJ, Folley BS, Sheehan JP, Han DY, Kryscio RJ, Carter MB, Shields LBE, Plato BM, La Rocca RV, Spalding AC, Yao TL, Fraser JF. Single low-dose targeted bevacizumab infusion in adult patients with steroid-refractory radiation necrosis of the brain: a phase II open-label prospective clinical trial. J Neurosurg. 2022 Apr 15;137(6):1676-1686. doi: 10.3171/2022.2.JNS212006. Print 2022 Dec 1. **PMID:** 35426830 ## Document Section ### Large Document Module #### Large Docs - Date: 2016-06-21 - Filename: Prot_SAP_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: True - Label: Study Protocol and Statistical Analysis Plan - Size: 2553241 - Type Abbrev: Prot_SAP - Upload Date: 2020-09-01T11:23 - Date: 2018-06-08 - Filename: ICF_001.pdf - Has ICF: True - Has Protocol: False - Has SAP: False - Label: Informed Consent Form - Size: 1012745 - Type Abbrev: ICF - Upload Date: 2020-09-01T11:24 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC14 - Name: Heart and Blood Diseases ### Condition Browse Module - Browse Leaves - ID: M12 - Name: Congenital Abnormalities - Relevance: LOW - As Found: Unknown - ID: M7657 - Name: Edema - Relevance: LOW - As Found: Unknown - ID: M12284 - Name: Necrosis - Relevance: HIGH - As Found: Necrosis - ID: M4473 - Name: Arteriovenous Malformations - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000009336 - Term: Necrosis ### Intervention Browse Module - Ancestors - ID: D000074322 - Term: Antineoplastic Agents, Immunological - ID: D000000970 - Term: Antineoplastic Agents - 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: D000004234 - Term: Diuretics, Osmotic - ID: D000004232 - Term: Diuretics - ID: D000045283 - Term: Natriuretic Agents ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: NaAg - Name: Natriuretic Agents ### Intervention Browse Module - Browse Leaves - ID: M246 - Name: Bevacizumab - Relevance: HIGH - As Found: Non- - ID: M11342 - Name: Mannitol - Relevance: HIGH - As Found: Abuse - ID: M1346 - Name: Antineoplastic Agents, Immunological - 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 - ID: M7411 - Name: Diuretics - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000068258 - Term: Bevacizumab - ID: D000008353 - Term: Mannitol ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** Adverse Event Monitoring performed on Day 0, Day 1, Week 6, Month 3, and Month 12 #### Event Groups **Group ID:** EG000 **Title:** Low-dose Intra-arterial Bevacizumab **Deaths Num At Risk:** 10 **Description:** A single intra-arterial dose of 2.5 mg/kg bevacizumab will be administered after osmotic blood-brain-barrier disruption with intra-arterial 25% mannitol at rate of 4-12 ml/sec for 30 seconds. 25% Mannitol: Route of administration: In this study, the first step of the treatment will be performing osmotic blood-brain-barrier disruption with administration of intra-arterial 25% Mannitol into the appropriate cervical artery. Low-dose Intra-arterial Bevacizumab: Route of administration: In this study, the second step of the treatment will be administering intra-arterial bevacizumab into the appropriate cervical artery. **ID:** EG000 **Other Num Affected:** 9 **Other Num at Risk:** 10 **Serious Number Affected:** 6 **Serious Number At Risk:** 10 **Title:** Low-dose Intra-arterial Bevacizumab **Frequency Threshold:** 0 #### Other Events **Term:** 1st degree AV block **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Acne **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Acne Related to Decadron **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Altered Mental Status **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Amnesia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Anxiety **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Asthma episode **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Bilateral lower leg edema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Blurred vision **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Cervical strain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Cervical strain due to MVA **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Confusion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Decreased appetite **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Dehydration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Diplopia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Expressive aphasia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Fall **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Fracture Left Hand **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** CTCAE v3.0 Term **Term:** Headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** 1 episode Frontal Headache from MVA; 1 episode Headache; 1 episode Right sided headache; 1 episode Headaches with vertigo; 3 episodes worsening headache **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Generalized Anxiety **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Generalized Malaise **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** hair loss **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Hallucinations **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Head Trauma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** CTCAE v3.0 Term **Term:** Hypercholesterolemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Hypersomnia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Hypertension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Hypertriglyceridemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Inability to focus **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Inability to focus attention **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Incomplete Spontaneous Abortion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Pregnancy, puerperium and perinatal conditions **Source Vocabulary:** CTCAE v3.0 Term **Term:** Dizziness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Increased dizziness **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Eye twitches **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Left eye twitches **Organ System:** Eye disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Homonymous Hemianopia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Left Homonymous Hemianopia **Organ System:** Eye disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Leg skin tear non-healing wound **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Left Leg skin tear non-healing wound **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Leg traumatic injury **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Left leg traumatic injury **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** CTCAE v3.0 Term **Term:** Loss of appetite **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Loss of vision **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** loss of vision X 30 seconds **Organ System:** Eye disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Lower Back pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Major Depressive Disorder **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Abdominal Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Mild Abdominal Pain **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Nausea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Neck pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Neck stiffness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Nosebleeds **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Panic attacks **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Personality Changes **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Personality Changes including anger **Organ System:** Psychiatric disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Pregnancy **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Pregnancy, puerperium and perinatal conditions **Source Vocabulary:** CTCAE v3.0 Term **Term:** Numbness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** 1 episode Intermittent Numbness in the bottom; 1 episode Whole body numbness **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Scalp Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Left vertex scalp soreness **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Ankle sprain due to fall **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Right ankle sprain due to fall **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** CTCAE v3.0 Term **Term:** Hip Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Right Hip Radiating Pain **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Inner Ear pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Right Inner Ear pain **Organ System:** Ear and labyrinth disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Knee pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** right knee pain from MVA **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Cold sensation leg **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Right leg cold sensation knee to foot **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Leg cramps **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Leg Muscle aches **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Right Leg Muscle aches **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Partial toenail ingrown **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Right partial toenail ingrown **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Calf muscle loss of tone **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Rt Calf muscle loss of tone **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Seizures **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Generalized Seizures **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Tonic/clonic Seizures **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Shot term memory problems **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Steroid Induced Hyperglycemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Endocrine disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Vision loss in left eye **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** temporary vision loss in left eye **Organ System:** Eye disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Thrombocytopenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Traumatic Fall **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** CTCAE v3.0 Term **Term:** Trouble performing simple tasks **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Unsteadiness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Upper Respiratory Infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Urinary incontinence **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Visual seizure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Vomiting **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Weight Loss **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Whole body shaking associated with seizures **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Worsening Acne on extremities **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Worsening chest pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Worsening of left sided weakness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term **Term:** Worsening of striae bilateral arms **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** CTCAE v3.0 Term #### Serious Events **Term:** Seizure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Month 6 Pt 20 min non-responsive but appeared awake. Ambulance. Brain MRI decr edema no acute infarct or hemorrh. D/C home. Another Pt month 6 seizure activity w dizziness, chest pain, left jaw pain and left arm pain and numbness, double vision **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 10 **Num Events:** 2 **Term:** Acute encephalopathy **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Pt admitted month 9 for 1-Confusion 2-Intermittent diaphoresis 3-Heat/cold intolerance 4-Mild abd Pain 5-Vomiting 6-Unsteadiness. Brain CT/MRI no sig change target area, age related ischemic changes. Workup neg. Meds held pt improved D/Ced home. **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 10 **Num Events:** 1 **Term:** Double Vision **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Double vision on Day 1 with dizziness reported after transferred into TCU per protocol. Investigator notified, patient transferred to ICU, symptoms resolved 2 hours after onset. **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 10 **Num Events:** 1 **Term:** Chest Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Pt has history of chest pain but symptoms worsened and were associated with Month 6 episode of seizures **Organ System:** Cardiac disorders **Source Vocabulary:** CTCAE v3.0 Term ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 10 **Num Events:** 1 **Term:** Headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Month 9 Pt having recurrent worsening headaches, Brain MRI showed peri-lesion radiation necrosis with residual AVM. Pt opted for surgical resection of simple supra-tentorial AVM and Radiation necrosis in the Perisylvian and insular region. **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 10 **Num Events:** 1 **Term:** Dizziness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Day 1 Double vision with dizziness reported after transferred into TCU per protocol. Investigator notified, patient transferred to ICU, symptoms resolved 2 hours after onset. **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 10 **Num Events:** 1 **Term:** Dysarthria **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Day 1 Pt developed acute onset Dysarthria followed by Lt. UE and LE Hemiparesis. Evaluated in ED, Brain CT/MRI and EEG revealed no sig changes, Hospitalized x 8 d started on steroids and Keppra (for poss seizure) weakness improved to LUE 5/5, LLE 3/5 **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 10 **Num Events:** 1 **Term:** Left Lower Extremity Hemiparesis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Day 1 Pt developed acute onset Dysarthria followed by Lt. UE and LE Hemiparesis. Evaluated in ED, Brain CT/MRI and EEG revealed no sig changes, Hospitalized x 8 d started on steroids and Keppra (for poss seizure) weakness improved to LUE 5/5, LLE 3/5 **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 10 **Num Events:** 1 **Term:** Left Upper Extremity Hemiparesis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Day 1 Pt developed acute onset Dysarthria followed by Lt. UE and LE Hemiparesis. Evaluated in ED, Brain CT/MRI and EEG revealed no sig changes, Hospitalized x 8 d started on steroids and Keppra (for poss seizure) weakness improved to LUE 5/5, LLE 3/5 **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 10 **Num Events:** 1 **Term:** Cerebral Edema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Notes:** Month 8 Pt having symptoms of cerebral edema, Brain MRI showed interval increase in edema and radiation necrosis. Pt opted for 2 cycles of IV Avastin with subsequent improvement in symptoms and MRI findings. **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE v3.0 Term ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 10 **Num Events:** 1 **Time Frame:** 1 year ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 10 **Units:** Participants ### Group **ID:** BG000 **Title:** Low-dose Intra-arterial Avastin (Bevacizumab) **Description:** A single intra-arterial dose of 2.5 mg/kg bevacizumab will be administered after osmotic blood-brain-barrier disruption with intra-arterial 25% mannitol at rate of 4-12 ml/sec for 30 seconds. 25% Mannitol: Route of administration: In this study, the first step of the treatment will be performing osmotic blood-brain-barrier disruption with administration of intra-arterial 25% Mannitol into the appropriate cervical artery. Low-dose Intra-arterial Bevacizumab: Route of administration: In this study, the second step of the treatment will be administering intra-arterial bevacizumab into the appropriate cervical artery. ### Measure #### Measurement **Group ID:** BG000 **Spread:** 14.8 **Value:** 35.1 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 8 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 2 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 1 **Category Title:** Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 9 **Category Title:** Not Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** American Indian or Alaska Native #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Asian #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Native Hawaiian or Other Pacific Islander #### Measurement **Group ID:** BG000 **Value:** 1 **Category Title:** Black or African American #### Measurement **Group ID:** BG000 **Value:** 9 **Category Title:** White #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** More than one race #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 10 **Class Title:** United States ### Measure #### Measurement **Group ID:** BG000 **Spread:** 12.1 **Value:** 14.5 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 95.9 **Value:** 86.8 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 110.1 **Value:** 81.7 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 29.8 **Value:** 33.3 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 2.2 **Value:** 6.9 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 7.5 **Value:** 62.4 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 0 **Upper Limit:** 100 **Value:** 52.5 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 9.9 **Value:** 79.0 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 11.8 **Value:** 41.1 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 8.3 **Value:** 44.9 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 10.7 **Value:** 38.2 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 3.8 **Value:** 44.6 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 14.6 **Value:** 41.0 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 3.4 **Value:** 47.6 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 2.4 **Value:** 44.6 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 3.5 **Value:** 44.6 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 3.0 **Value:** 42.9 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 3.6 **Value:** 41.3 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 2.9 **Value:** 43.2 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 2.3 **Value:** 43.7 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 3.5 **Value:** 41.4 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 2.9 **Value:** 39.8 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 2.2 **Value:** 41.8 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 3.7 **Value:** 45.0 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 4.1 **Value:** 95.8 **Class Title:** **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Description:** Age at enrollment, years **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:** Ethnicity (NIH/OMB) **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:** Region of Enrollment **Unit of Measure:** participants ### Measure 6 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** MRI of Brain: volume of radiation necrosis **Unit of Measure:** cubic centimeters, or cm^3 ### Measure 7 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** MRI of Brain: volume of edema **Unit of Measure:** cubic centimeters, or cm^3 ### Measure 8 **Description:** MIDAS (Migraine Disability Assessment) is a 7-item tool that measures how severely headaches prevented or interfered with normal activity over the past 3 months. For each item 1 through 5 the min score=0 days, max score=90 days. The MIDAS TOTAL SCORE sums items 1 - 5 only. TOTAL SCORE of 0-5 indicates little or no disability. Total Score of 6-10 indicates mild disability. Total Score of 11-20 indicates moderate disability. Total score of 21+ indicates severe disability. Theoretical Max Total Score is 450 days. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Headache severity via Migraine Disability Assessment (MIDAS) TOTAL SCORE **Unit of Measure:** units on a scale from 0 to 450 ### Measure 9 **Description:** MIDAS (Migraine Disability Assessment) questionnaire is a 7-item tool that measures how severely headaches prevented or interfered with normal activity over the past 3 months. MIDAS DAYS of HEADACHE is Item 6 of the questionnaire which sums total number of days the patient had headache symptoms over the past 3 months regardless of headache severity or resultant disability. Min score of 0 days is the best possible score. Max Score of 90 days is worse possible score, meaning the patient experienced headache pain every day over past 3 months. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** DAYS of Headache via Migraine Disability Assessment (MIDAS) **Unit of Measure:** Days of headache over past 3 months ### Measure 10 **Description:** MIDAS (Migraine Disability Assessment) is a 7-item tool that measures how severely headaches prevented or interfered with normal activity over the past 3 months. Item 7 rates the 3-month average headache PAIN LEVEL on scale from 0 to 10 where 0 = no pain at all, and 10 = pain as bad as it can be. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Headache PAIN LEVEL via Migraine Disability Assessment (MIDAS) **Unit of Measure:** units on a scale from 0 to 10 ### Measure 11 **Description:** The Headache Impact Test-6 is a tool used to measure the impact headaches have on a patient's ability to function in their normal daily life. This 6-item questionnaire was designed to help patients describe and communicate the way they feel and what they cannot do because of their headaches. For each item, patients must select one answer: never, rarely, sometimes, very often, or always. Scores range from min of 36 indicating minimum headache impact, to a max of 78 indicating worst possible headache impact. A score of 60 or more indicates headaches having a very severe impact on daily life. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Headache severity via Headache Impact Test (HIT-6) **Unit of Measure:** units on a scale from 36 - 78 ### Measure 12 **Description:** Total cumulative days of steroid usage were compared between the 12 months prior to and the 12 months immediately following targeted IA-bevacizumab. Days of steroid usage was tabulated via medical history and chart review at the baseline visit, and post-treatment on days 0 and 1, week 6, and months 3, 6, 9 and 12. Excluding topical steroid preparations, all days of enteral or parenteral steroid intake of any dose were included in the cumulative summation. **Dispersion Type:** INTER_QUARTILE_RANGE **Parameter Type:** MEDIAN **Title:** Cumulative Days of Steroid use during 12 months Pre-Bevacizumab **Unit of Measure:** Days ### Measure 13 **Description:** The Karnofsky Performance Status Scale allows patients to be classified as to their functional impairment. The lower the score, the worse the survival for most serious illnesses. Scores range from 0% to 100% where 100%=Normal with no complaints, no evidence of disease. 90%=Able to carry on normal activity; minor signs or symptoms of disease. 80%=Normal activity with effort; some signs or symptoms of disease. 70% = cares for self; unable to carry on normal activity or to do active work. 60%=Requires occasional assistance, but is able to care for most of his personal needs and 0%=Dead. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Karnofsky Performance Status Scale **Unit of Measure:** % on Karnofsky scale ### Measure 14 **Description:** The Neuropsychological Assessment Battery is a comprehensive, demographically adjusted, normalized battery of tests which measure neurocognitive function. The Digits Forward module generates T scores which indicate the degree to which immediate auditory attention is higher or lower than that of healthy demographically matched individuals in the normative sample. T-score of 50 is the mean and is considered normal. Scores significantly below 40 indicate decreased auditory attention. Scores significantly above 60 indicate better auditory attention than healthy demographically matched persons. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Digits Forward via Neuropsychological Assessment Battery **Unit of Measure:** T score with Mean=50, SD=10 ### Measure 15 **Description:** The Neuropsychological Assessment Battery is a comprehensive, demographically adjusted, normalized battery of tests which measure neurocognitive function. The Digits Backward module generates T scores which indicate the degree to which auditory working memory is stronger or weaker than that of a healthy demographically matched individual in the normative sample. T-score of 50 is equal to the mean and is considered normal. Scores significantly below 40 indicate decreased performance. Scores significantly above 60 indicate stronger ability compared to healthy demographically matched persons. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Digits Backward via Neuropsychological Assessment Battery **Unit of Measure:** T score with Mean=50, SD=10 ### Measure 16 **Description:** The Neuropsychological Assessment Battery is a comprehensive, demographically adjusted normalized battery of tests which measure neurocognitive function. Numbers \& Letters Speed module is an excellent general indicator of speed and efficiency of information processing. T scores indicate the degree to which info processing speed differs from that of healthy demographically matched individuals. T-scores of 40-60 are within 1 SD of the mean (50) and are considered normal. Scores below 40 indicate decreased processing speed. Scores above 60 indicate more rapid processing speed. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Numbers and Letters Speed via Neuropsychological Assessment Battery **Unit of Measure:** T score with Mean=50, SD=10 ### Measure 17 **Description:** The Neuropsychological Assessment Battery is a comprehensive, demographically normalized battery of tests of neurocognitive function. Numbers \& Letters ERRORS module measures the extent of errorful processing. Errorful processing is considered "inefficient". T scores indicate the degree to which processing efficiency differs from that of healthy demographically matched individuals. T-score of 50 is the mean. Scores significantly below 40 indicate decreased performance. Scores significantly above 60 indicate stronger ability compared to healthy demographically matched persons. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Numbers and Letters Errors via Neuropsychological Assessment Battery **Unit of Measure:** T score with Mean=50, SD=10 ### Measure 18 **Description:** The Neuropsychological Assessment Battery is a comprehensive, demographically normalized battery of tests of neurocognitive function. Numbers \& Letters EFFICIENCY module compares error detection to time. Greater "efficiency" defined as minimal errors during rapid processing. Slow, errorful processing is considered "inefficient". T scores indicate the degree to which processing efficiency differs from that of healthy demographically matched individuals. T-score of 50 is the mean. Scores below 40 indicate decreased info processing efficiency. Scores above 60 indicate more efficient processing. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Numbers and Letters Efficiency via Neuropsychological Assessment Battery **Unit of Measure:** T score with Mean=50, SD=10 ### Measure 19 **Description:** The Neuropsychological Assessment Battery is a comprehensive, demographically normalized battery of tests of neurocognitive function. Naming module measures language processing. Errorful naming is considered inefficient language processing. T scores indicate the degree to which language processing differs from that of healthy demographically matched individuals. T-score of 50 is the mean. Scores significantly below 40 indicate decreased performance. Scores significantly above 60 indicate stronger ability compared to healthy demographically matched persons. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Naming via Neuropsychological Assessment Battery **Unit of Measure:** T score with Mean=50, SD=10 ### Measure 20 **Description:** The Neuropsychological Assessment Battery is a comprehensive, demographically normalized battery of tests of neurocognitive function. The List Learning List Immediate Recall module is one measure of short term memory. T scores indicate the degree to which short term memory differs from that of healthy demographically matched individuals. T-score of 50 is the mean. Scores significantly below 40 indicate decreased performance. Scores significantly above 60 indicate stronger ability compared to healthy demographically matched persons. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** List Learning List Immediate Recall via Neuropsychological Assessment Battery **Unit of Measure:** T score with Mean=50, SD=10 ### Measure 21 **Description:** The Neuropsychological Assessment Battery is a comprehensive, demographically normalized battery of tests of neurocognitive function. The List Learning List Long Delayed Recall module is one measure of long term memory. T scores indicate the degree to which long term memory differs from that of healthy demographically matched individuals. T-score of 50 is the mean. Scores significantly below 40 indicate decreased performance. Scores significantly above 60 indicate stronger ability compared to healthy demographically matched persons. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** List Learning List Long Delayed Recall via Neuropsychological Assessment Battery **Unit of Measure:** T score with Mean=50, SD=10 ### Measure 22 **Description:** The Neuropsychological Assessment Battery is a comprehensive, demographically normalized battery of tests of neurocognitive function. The Shape Learning Immediate Recognition module is another measure of short term memory. T scores indicate the degree to which short term memory differs from that of healthy demographically matched individuals. T-score of 50 is the mean. Scores significantly below 40 indicate decreased performance. Scores significantly above 60 indicate stronger ability compared to healthy demographically matched persons. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Shape Learning Immediate Recognition via Neuropsychological Assessment Battery **Unit of Measure:** T score with Mean=50, SD=10 ### Measure 23 **Description:** The Neuropsychological Assessment Battery is a comprehensive, demographically normalized battery of tests of neurocognitive function. The Shape Learning Delayed Recognition module is another measure of long term memory. T scores indicate the degree to which long term memory differs from that of healthy demographically matched individuals. T-score of 50 is the mean. Scores significantly below 40 indicate decreased performance. Scores significantly above 60 indicate stronger ability compared to healthy demographically matched persons. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Shape Learning Delayed Recognition via Neuropsychological Assessment Battery **Unit of Measure:** T score with Mean=50, SD=10 ### Measure 24 **Description:** The Neuropsychological Assessment Battery is a comprehensive, demographically normalized battery of tests of neurocognitive function. The Story Learning Phrase Unit Immediate Recall module is another measure of short term memory. T scores indicate the degree to which short term memory differs from that of healthy demographically matched individuals. T-score of 50 is the mean. Scores significantly below 40 indicate decreased performance. Scores significantly above 60 indicate stronger ability compared to healthy demographically matched persons. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Story Learning Phrase Unit Immediate Recall via Neuropsychological Assessment Battery **Unit of Measure:** T score with Mean=50, SD=10 ### Measure 25 **Description:** The Neuropsychological Assessment Battery is a comprehensive, demographically normalized battery of tests of neurocognitive function. The Story Learning Phrase Unit Delayed Recall module is another measure of long term memory. T scores indicate the degree to which long term memory differs from that of healthy demographically matched individuals. T-score of 50 is the mean. Scores significantly below 40 indicate decreased performance. Scores significantly above 60 indicate stronger ability compared to healthy demographically matched persons. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Story Learning Phrase Unit Delayed Recall via Neuropsychological Assessment Battery **Unit of Measure:** T score with Mean=50, SD=10 ### Measure 26 **Description:** The Neuropsychological Assessment Battery is a comprehensive, demographically normalized battery of tests of neurocognitive function. Design Construction module is one measure of visuospatial processing. T scores indicate the degree to which visuospatial processing differs from that of healthy demographically matched individuals. T-score of 50 is the mean. Scores significantly below 40 indicate decreased performance. Scores significantly above 60 indicate stronger ability compared to healthy demographically matched persons. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Design Construction via Neuropsychological Assessment Battery **Unit of Measure:** T score with Mean=50, SD=10 ### Measure 27 **Description:** The Neuropsychological Assessment Battery is a comprehensive, demographically normalized battery of tests of neurocognitive function. Mazes module is one measure of executive functioning. T scores indicate the degree to which executive functioning differs from that of healthy demographically matched individuals. T-score of 50 is the mean. Scores significantly below 40 indicate decreased performance. Scores significantly above 60 indicate stronger ability compared to healthy demographically matched persons. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Mazes via Neuropsychological Assessment Battery **Unit of Measure:** T score with Mean=50, SD=10 ### Measure 28 **Description:** The Neuropsychological Assessment Battery is a comprehensive, demographically normalized battery of tests of neurocognitive function. Categories module is another measure of executive functioning. T scores indicate the degree to which executive functioning differs from that of healthy demographically matched individuals. T-score of 50 is the mean. Scores significantly below 40 indicate decreased performance. Scores significantly above 60 indicate stronger ability compared to healthy demographically matched persons. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Categories via Neuropsychological Assessment Battery **Unit of Measure:** T score with Mean=50, SD=10 ### Measure 29 **Description:** The Neuropsychological Assessment Battery is a comprehensive, demographically normalized battery of tests of neurocognitive function. The Word Generation module is another measure of executive functioning. T scores indicate the degree to which executive functioning differs from that of healthy demographically matched individuals. T-score of 50 is the mean. Scores significantly below 40 indicate decreased performance. Scores significantly above 60 indicate stronger ability compared to healthy demographically matched persons. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Word Generation via Neuropsychological Assessment Battery **Unit of Measure:** T score with Mean=50, SD=10 ### Measure 30 **Description:** The Wechsler Test of Adult Reading is a test of cognitive ability. The full scale intelligence quotient (FSIQ) estimate was obtained at Baseline to provide an estimate of premorbid cognitive functioning and to serve as a covariate for neurocognitive analyses. FSIQ population mean =100, SD = 15. By convention scores 130- 210 are very superior; 120-129 superior; 110-119 high average. Scores 90-109 are considered to be in the "average" range. Scores 80-89 are low average; 70-79 borderline; 28-69 are classified as extremely low. **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Full Scale Intelligence Quotient (FSIQ) via Wechsler Test of Adult Reading **Unit of Measure:** units on a scale from 28 to 210 ## Results Section - More Information Module ### Certain Agreement ### Limitations and Caveats **Description:** Pilot Phase 2 Single Arm Clinical Trial with small number of patients. Not powered sufficiently to provide definitive proof for change in current standard of care of medically refractory radiation necrosis of the brain. ### Point of Contact **Email:** [email protected] **Organization:** Norton Neuroscience Institute **Phone:** 502-394-6390 **Title:** Shervin R. Dashti, MD, PhD, Principal Investigator ## 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:** CHANGE IN VOLUME OF RADIATION NECROSIS: To explore durability of radiographic responses, we invoked a linear mixed model in which we addressed heterogeneity of time points by fitting the most appropriate model among compound symmetry, Huyhn-Felt structure, and an unstructured covariance matrix (multivariate analysis of variance). **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.012 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** eta^2 **Parameter Value:** 0.37 **Statistical Comment:** F(1,14) = 8.29 **Statistical Method:** Mixed Models Analysis **Tested Non-Inferiority:** #### 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:** CHANGE IN VOLUME OF CEREBRAL EDEMA: To explore durability of radiographic responses, we invoked a linear mixed model in which we addressed heterogeneity of time points by fitting the most appropriate model among compound symmetry, Huyhn-Felt structure, and an unstructured covariance matrix (multivariate analysis of variance). **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.09 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** eta^2 **Parameter Value:** 0.19 **Statistical Comment:** F(1,14) = 3.29 **Statistical Method:** Mixed Models Analysis **Tested Non-Inferiority:** ### 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:** To explore durability of clinical response, Migraine Disability Assessment (MIDAS) TOTAL SCORES were analyzed using a linear mixed model in which we addressed heterogeneity of time points by fitting the most appropriate model among compound symmetry, Huyhn Felt structure, and an unstructured covariance matrix (multivariate analysis of variance) **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.02 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** eta^2 **Parameter Value:** 0.29 **Statistical Comment:** F(5,34) = 2.74 **Statistical Method:** Mixed Models Analysis **Tested Non-Inferiority:** ### 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:** To explore durability of clinical response, Migraine Disability Assessment (MIDAS) DAYS OF HEADACHE were analyzed using a linear mixed model in which we addressed heterogeneity of time points by fitting the most appropriate model among compound symmetry, Huyhn Felt structure, and an unstructured covariance matrix (multivariate analysis of variance). **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.019 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** eta^2 **Parameter Value:** 0.37 **Statistical Comment:** F(5,34) = 3.17 **Statistical Method:** Mixed Models Analysis **Tested Non-Inferiority:** ### 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:** To explore durability of clinical response, Migraine Disability Assessment (MIDAS) MIDAS PAIN LEVEL data were analyzed using a linear mixed model in which we addressed heterogeneity of time points by fitting the most appropriate model among compound symmetry, Huyhn Felt structure, and an unstructured covariance matrix (multivariate analysis of variance). **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.0006 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** eta^2 **Parameter Value:** 0.3 **Statistical Comment:** F(5,35) = 3.96 **Statistical Method:** Mixed Models Analysis **Tested Non-Inferiority:** ### 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:** To explore durability of clinical response, Headache Impact Test (HIT-6™) scores were analyzed using a linear mixed model in which we addressed heterogeneity of time points by fitting the most appropriate model among compound symmetry, Huyhn-Felt structure, and an unstructured covariance matrix (multivariate analysis of variance). **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.02 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** eta^2 **Parameter Value:** 0.30 **Statistical Comment:** F(5, 35) = 2.98 **Statistical Method:** Mixed Models Analysis **Tested Non-Inferiority:** ### 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:** To explore durability of clinical response, Karnofsky Performance Status Scale (KPS) data were analyzed using a linear mixed model in which we addressed heterogeneity of time points by fitting the most appropriate model among compound symmetry, Huyhn-Felt structure, and an unstructured covariance matrix (multivariate analysis of variance). **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.23 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** eta^2 **Parameter Value:** 0.19 **Statistical Comment:** F(2,14) = 1.62 **Statistical Method:** Mixed Models Analysis **Tested Non-Inferiority:** ### Outcome Measure 7 #### 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:** Wilcoxon signed rank test (2-sided) was used to compare the total number of days on steroids in the 12 months prior versus the 12 months immediately following single dose IA Avastin (bevacizumab). **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.374 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** Pearson's r **Parameter Value:** -0.199 **Statistical Comment:** Z = -0.889 **Statistical Method:** Wilcoxon (Mann-Whitney) **Tested Non-Inferiority:** ### Outcome Measure 8 #### 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:** DIGITS FORWARD: To explore post-operative change in Neurocognitive performance after intra-arterial bevacizumab, repeated measures ANOVA was performed for the DIGITS FORWARD variable across 3 time points (BL, 3 months, 12 months) where IQ was placed in the model as covariate. **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.660 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** partial eta^2 **Parameter Value:** 0.153 **Statistical Comment:** F(2,5) = 0.451 **Statistical Method:** One way Repeat Meas ANOVA **Tested Non-Inferiority:** #### 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:** DIGITS BACKWARD: To explore post-operative change in Neurocognitive performance after intra-arterial bevacizumab, repeated measures ANOVA was performed for the DIGITS BACKWARD variable across 3 time points (BL, 3 months, 12 months) where IQ was placed in the model as covariate. **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.100 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** partial eta^2 **Parameter Value:** 0.602 **Statistical Comment:** F(2,5) = 3.78 **Statistical Method:** One way Repeat Meas ANOVA **Tested Non-Inferiority:** #### 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:** NUMBERS \& LETTERS SPEED: To explore post-operative change in Neurocognitive performance after intra-arterial bevacizumab, repeated measures ANOVA was performed for the NUMBERS \& LETTERS SPEED variable across 3 time points (BL, 3 months, 12 months) where IQ was placed in the model as covariate. **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.149 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** partial eta^2 **Parameter Value:** 0.532 **Statistical Comment:** F(2,5) = 2.847 **Statistical Method:** One way Repeat Meas ANOVA **Tested Non-Inferiority:** #### 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:** NUMBERS \& LETTERS ERRORS: To explore post-operative change in Neurocognitive performance after intra-arterial bevacizumab, repeated measures ANOVA was performed for the NUMBERS \& LETTERS ERRORS variable across 3 time points (BL, 3 months, 12 months) where IQ was placed in the model as covariate. **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.041 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** partial eta^2 **Parameter Value:** 0.721 **Statistical Comment:** F(2,5) = 6.470 **Statistical Method:** One way Repeat Meas ANOVA **Tested Non-Inferiority:** #### 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:** NUMBERS \& LETTERS EFFICIENCY: To explore post-operative change in Neurocognitive performance after intra-arterial bevacizumab, repeated measures ANOVA was performed for the NUMBERS \& LETTERS EFFICIENCY variable across 3 time points (BL, 3 months, 12 months) where IQ was placed in the model as covariate. **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.124 **P-Value Comment:** **Parameter Type:** partial eta^2 **Parameter Value:** 0.566 **Statistical Comment:** F (2,5) = 3.256 **Statistical Method:** One way Repeat Meas ANOVA **Tested Non-Inferiority:** #### 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:** NAMING: To explore post-operative change in Neurocognitive performance after intra-arterial bevacizumab, repeated measures ANOVA was performed for the NAMING variable across 3 time points (BL, 3 months, 12 months) where IQ was placed in the model as covariate. **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.898 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** partial eta^2 **Parameter Value:** 0.042 **Statistical Comment:** F(2,5) = 0.110 **Statistical Method:** One way Repeat Meas ANOVA **Tested Non-Inferiority:** #### 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:** LIST LEARNING LIST IMMEDIATE RECALL: To explore post-operative change in Neurocognitive performance after intra-arterial bevacizumab, repeated measures ANOVA was performed for the LIST LEARNING LIST IMMEDIATE RECALL variable across 3 time points (BL, 3 months, 12 months) where IQ was placed in the model as covariate. **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.754 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** partial eta^2 **Parameter Value:** 0.107 **Statistical Comment:** F(2,5) = 0.299 **Statistical Method:** One way Repeat Meas ANOVA **Tested Non-Inferiority:** #### 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:** LIST LEARNING LIST LONG DELAYED RECALL: To explore post-operative change in Neurocognitive performance after intra-arterial bevacizumab, repeated measures ANOVA was performed for the LIST LEARNING LIST LONG DELAYED RECALL variable across 3 time points (BL, 3 months, 12 months) where IQ was placed in the model as covariate. **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.031 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** partial eta^2 **Parameter Value:** 0.751 **Statistical Comment:** F(2,5) = 7.556 **Statistical Method:** One way Repeat Meas ANOVA **Tested Non-Inferiority:** #### 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:** SHAPE LEARNING IMMEDIATE RECOGNITION: To explore post-operative change in Neurocognitive performance after intra-arterial bevacizumab, repeated measures ANOVA was performed for the SHAPE LEARNING IMMEDIATE RECOGNITION variable across 3 time points (BL, 3 months, 12 months) where IQ was placed in the model as covariate. **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.426 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** partial eta^2 **Parameter Value:** 0.289 **Statistical Comment:** F(2,5) = 1.018 **Statistical Method:** One way Repeat Meas ANOVA **Tested Non-Inferiority:** #### 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:** SHAPE LEARNING DELAYED RECOGNITION: To explore post-operative change in Neurocognitive performance after intra-arterial bevacizumab, repeated measures ANOVA was performed for the SHAPE LEARNING DELAYED RECOGNITION variable across 3 time points (BL, 3 months, 12 months) where IQ was placed in the model as covariate. **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.326 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** partial eta^2 **Parameter Value:** 0.361 **Statistical Comment:** F(2,5) = 1.412 **Statistical Method:** One way Repeat Meas ANOVA **Tested Non-Inferiority:** #### 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:** STORY LEARNING PHRASE UNIT IMMEDIATE RECALL: To explore post-operative change in Neurocognitive performance after intra-arterial bevacizumab, repeated measures ANOVA was performed for the STORY LEARNING PHRASE UNIT IMMEDIATE RECALL variable across 3 time points (BL, 3 months, 12 months) where IQ was placed in the model as covariate. **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.790 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** partial eta^2 **Parameter Value:** 0.090 **Statistical Comment:** F(2,5) = 0.247 **Statistical Method:** One way Repeat Meas ANOVA **Tested Non-Inferiority:** #### 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:** STORY LEARNING PHRASE UNIT DELAYED RECALL: To explore post-operative change in Neurocognitive performance after intra-arterial bevacizumab, repeated measures ANOVA was performed for the STORY LEARNING PHRASE UNIT DELAYED RECALL variable across 3 time points (BL, 3 months, 12 months) where IQ was placed in the model as covariate. **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.505 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** partial eta^2 **Parameter Value:** 0.239 **Statistical Comment:** F(2,5) = 0.786 **Statistical Method:** One way Repeat Meas ANOVA **Tested Non-Inferiority:** #### 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:** DESIGN CONSTRUCTION: To explore post-operative change in Neurocognitive performance after intra-arterial bevacizumab, repeated measures ANOVA was performed for the DESIGN CONSTRUCTION variable across 3 time points (BL, 3 months, 12 months) where IQ was placed in the model as covariate. **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.080 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** partial eta^2 **Parameter Value:** 0.636 **Statistical Comment:** F(2,5) = 4.362 **Statistical Method:** One way Repeat Meas ANOVA **Tested Non-Inferiority:** #### 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:** MAZES: To explore post-operative change in Neurocognitive performance after intra-arterial bevacizumab, repeated measures ANOVA was performed for the MAZES variable across 3 time points (BL, 3 months, 12 months) where IQ was placed in the model as covariate. **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.052 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** partial eta^2 **Parameter Value:** 0.693 **Statistical Comment:** F(2,5) = 5.654 **Statistical Method:** One way Repeat Meas ANOVA **Tested Non-Inferiority:** #### 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:** CATEGORIES: To explore post-operative change in Neurocognitive performance after intra-arterial bevacizumab, repeated measures ANOVA was performed for the CATEGORIES variable across 3 time points (BL, 3 months, 12 months) where IQ was placed in the model as covariate. **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.968 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** partial eta^2 **Parameter Value:** 0.013 **Statistical Comment:** F(2,5) = 0.033 **Statistical Method:** One way Repeat Meas ANOVA **Tested Non-Inferiority:** #### 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:** WORD GENERATION: To explore post-operative change in Neurocognitive performance after intra-arterial bevacizumab, repeated measures ANOVA was performed for the WORD GENERATION variable across 3 time points (BL, 3 months, 12 months) where IQ was placed in the model as covariate. **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** 0.469 **P-Value Comment:** The threshold for statistical significance was p \< 0.05. **Parameter Type:** partial eta^2 **Parameter Value:** 0.261 **Statistical Comment:** F(2,5) = 0.884 **Statistical Method:** One way Repeat Meas ANOVA **Tested Non-Inferiority:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 95.9 - **Upper Limit:** - **Value:** 86.8 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 37.5 - **Upper Limit:** - **Value:** 38.6 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 33.7 - **Upper Limit:** - **Value:** 32.6 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 8 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 12.1 - **Upper Limit:** - **Value:** 14.5 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 7.5 - **Upper Limit:** - **Value:** 6.6 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 3.3 - **Upper Limit:** - **Value:** 3.2 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 8 **Units:** Participants #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 110.1 - **Upper Limit:** - **Value:** 81.7 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 66.6 - **Upper Limit:** - **Value:** 49.7 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 30.3 - **Upper Limit:** - **Value:** 22.1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 46.2 - **Upper Limit:** - **Value:** 28.3 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 33.7 - **Upper Limit:** - **Value:** 24.4 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 26.5 - **Upper Limit:** - **Value:** 15.6 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 29.8 - **Upper Limit:** - **Value:** 33.3 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 35.0 - **Upper Limit:** - **Value:** 25.8 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 9.3 - **Upper Limit:** - **Value:** 9.0 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 14.1 - **Upper Limit:** - **Value:** 14.6 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 17.6 - **Upper Limit:** - **Value:** 12.4 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 17.0 - **Upper Limit:** - **Value:** 11.5 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 2.2 - **Upper Limit:** - **Value:** 6.9 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 1.8 - **Upper Limit:** - **Value:** 6.4 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 2.9 - **Upper Limit:** - **Value:** 4.7 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 2.9 - **Upper Limit:** - **Value:** 5.0 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 3.0 - **Upper Limit:** - **Value:** 4.8 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 3.5 - **Upper Limit:** - **Value:** 4.8 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 7.5 - **Upper Limit:** - **Value:** 62.4 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 11.4 - **Upper Limit:** - **Value:** 54.5 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 13.0 - **Upper Limit:** - **Value:** 52.3 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 13.6 - **Upper Limit:** - **Value:** 53.3 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 15.4 - **Upper Limit:** - **Value:** 53.2 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 14.4 - **Upper Limit:** - **Value:** 52.1 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 8 **Units:** Participants #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 9.9 - **Upper Limit:** - **Value:** 79.0 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 7.4 - **Upper Limit:** - **Value:** 81.0 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 11.6 - **Upper Limit:** - **Value:** 83.0 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 7.4 - **Upper Limit:** - **Value:** 86.3 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 8 **Units:** Participants #### Outcome Measure 7 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0 - **Spread:** - **Upper Limit:** 156.25 - **Value:** 61.5 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0 - **Spread:** - **Upper Limit:** 87.25 - **Value:** 13.0 **Title:** #### Outcome Measure 8 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 11.8 - **Upper Limit:** - **Value:** 41.1 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 12.2 - **Upper Limit:** - **Value:** 44.1 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 11.7 - **Upper Limit:** - **Value:** 42.3 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 8 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 8.3 - **Upper Limit:** - **Value:** 44.9 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 11.9 - **Upper Limit:** - **Value:** 44.9 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 13.1 - **Upper Limit:** - **Value:** 40.3 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 8 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 10.7 - **Upper Limit:** - **Value:** 38.2 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 10.8 - **Upper Limit:** - **Value:** 36.7 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 10.7 - **Upper Limit:** - **Value:** 36.8 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 8 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 12.0 - **Upper Limit:** - **Value:** 44.6 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 12.6 - **Upper Limit:** - **Value:** 47.4 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 10.1 - **Upper Limit:** - **Value:** 52.9 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 8 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 14.6 - **Upper Limit:** - **Value:** 41.0 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 11.8 - **Upper Limit:** - **Value:** 36.6 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 11.1 - **Upper Limit:** - **Value:** 36.6 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 8 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 10.6 - **Upper Limit:** - **Value:** 47.6 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 7.3 - **Upper Limit:** - **Value:** 49.9 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 10.9 - **Upper Limit:** - **Value:** 49.1 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 8 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 7.7 - **Upper Limit:** - **Value:** 44.6 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 13.1 - **Upper Limit:** - **Value:** 44.2 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 7.8 - **Upper Limit:** - **Value:** 46.4 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 8 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 11.1 - **Upper Limit:** - **Value:** 44.6 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 11.0 - **Upper Limit:** - **Value:** 42.6 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 12.1 - **Upper Limit:** - **Value:** 51.3 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 8 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 9.5 - **Upper Limit:** - **Value:** 42.9 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 10.7 - **Upper Limit:** - **Value:** 48.6 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 11.3 - **Upper Limit:** - **Value:** 50.0 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 8 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 11.3 - **Upper Limit:** - **Value:** 41.3 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 12.2 - **Upper Limit:** - **Value:** 46.0 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 9.1 - **Upper Limit:** - **Value:** 47.3 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 8 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 9.2 - **Upper Limit:** - **Value:** 43.2 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 12.7 - **Upper Limit:** - **Value:** 47.1 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 12.8 - **Upper Limit:** - **Value:** 49.6 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 8 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 7.3 - **Upper Limit:** - **Value:** 43.7 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 9.2 - **Upper Limit:** - **Value:** 48.5 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 8.0 - **Upper Limit:** - **Value:** 47.1 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 8 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 11.1 - **Upper Limit:** - **Value:** 41.4 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 14.3 - **Upper Limit:** - **Value:** 41.6 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 9.6 - **Upper Limit:** - **Value:** 45.8 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 8 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 9.2 - **Upper Limit:** - **Value:** 39.8 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 12.3 - **Upper Limit:** - **Value:** 43.3 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 11.7 - **Upper Limit:** - **Value:** 43.0 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 8 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 6.8 - **Upper Limit:** - **Value:** 41.8 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 10.4 - **Upper Limit:** - **Value:** 44.5 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 8.2 - **Upper Limit:** - **Value:** 39.9 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 8 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 11.8 - **Upper Limit:** - **Value:** 45.0 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 9.8 - **Upper Limit:** - **Value:** 45.6 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 10 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 11.3 - **Upper Limit:** - **Value:** 49.0 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 8 **Units:** Participants ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Imaging response to therapy will be quantitatively assessed on MRI using volumetric analysis. Regions of T2 and FLAIR prolongation above contralateral white matter will be calculated and quantified in cubic centimeters. Region of interest (ROI) will be created using a semi-automated, thresholding and region-growing technique. Enhancement of the lesion will be calculated using similar volumetric ROI analysis with a contrast threshold of 40% above background and measured in cubic centimeters. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** 10 adult subjects presented with medically refractory radiation necrosis of the brain and disabling headache. All were treated with steroids prior to study enrollment. **Reporting Status:** POSTED **Time Frame:** Baseline (before treatment), and 3 months and 12 months after single dose intra-arterial Avastin (bevacizumab) **Title:** Change in Radiation Necrosis and Cerebral Edema After a Single Treatment of Low Dose Intra-arterial Avastin (Bevacizumab) **Type:** PRIMARY **Unit of Measure:** cm^3 ##### Group **Description:** A single intra-arterial dose of 2.5 mg/kg bevacizumab will be administered after osmotic blood-brain-barrier disruption with intra-arterial 25% mannitol at rate of 4-12 ml/sec for 30 seconds. 25% Mannitol: Route of administration: In this study, the first step of the treatment will be performing osmotic blood-brain-barrier disruption with administration of intra-arterial 25% Mannitol into the appropriate cervical artery. Low-dose Intra-arterial bevacizumab: Route of administration: In this study, the second step of the treatment will be administering intra-arterial bevacizumab into the appropriate cervical artery. **ID:** OG000 **Title:** Low-dose Intra-arterial Avastin (Bevacizumab) #### Outcome Measure 2 **Description:** We will determine how severely headache affect the patient's life before and after treatment by performing the Migraine Disability Assessment (MIDAS) questionnaire. MIDAS TOTAL SCORE is the sum of Items 1 through 5. A Total Score of 0-5 signifies little or no disability, score of 6-10 signifies mild disability, score of 11-20 signifies moderate disability, and score of 21+ signifies severe disability. The theoretical maximum score would be 450. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** 10 adult subjects presented with medically refractory radiation necrosis of the brain and disabling headache. **Reporting Status:** POSTED **Time Frame:** Baseline (before treatment), and 6 weeks, 3 months, 6 months, 9 months, and 12 months after single dose intra-arterial Avastin (bevacizumab) **Title:** Change in Headache Associated Morbidity Measured With MIDAS TOTAL SCORE After a Single Treatment of Low Dose Intra-arterial Avastin (Bevacizumab) Will be Measured. **Type:** SECONDARY **Unit of Measure:** score on a scale ##### Group **Description:** A single intra-arterial dose of 2.5 mg/kg bevacizumab will be administered after osmotic blood-brain-barrier disruption with intra-arterial 25% mannitol at rate of 4-12 ml/sec for 30 seconds. 25% Mannitol: Route of administration: In this study, the first step of the treatment will be performing osmotic blood-brain-barrier disruption with administration of intra-arterial 25% Mannitol into the appropriate cervical artery. Low-dose Intra-arterial bevacizumab: Route of administration: In this study, the second step of the treatment will be administering intra-arterial bevacizumab into the appropriate cervical artery. **ID:** OG000 **Title:** Low-dose Intra-arterial Avastin (Bevacizumab) #### Outcome Measure 3 **Description:** We will determine how severely headache affect the patient's life before and after treatment by performing the Migraine Disability Assessment (MIDAS) questionnaire. MIDAS DAYS of HEADACHE is Item 6 of the questionnaire which sums total number of days the patient had headache symptoms over the past 3 months regardless of headache severity or resultant disability. Min score of 0 days is the best possible score. Max Score of 90 days is worse possible score, meaning the patient experienced headache pain every day over past 3 months. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** 10 adult subjects presented with medically refractory radiation necrosis of the brain and disabling headache. **Reporting Status:** POSTED **Time Frame:** Baseline (before treatment), and 6 weeks, 3 months, 6 months, 9 months, and 12 months after single dose intra-arterial Avastin (bevacizumab) **Title:** Change in Headache Associated Morbidity Measured With MIDAS DAYS of HEADACHE After a Single Treatment of Low Dose Intra-arterial Avastin (Bevacizumab) Will be Measured. **Type:** SECONDARY **Unit of Measure:** Days ##### Group **Description:** A single intra-arterial dose of 2.5 mg/kg bevacizumab will be administered after osmotic blood-brain-barrier disruption with intra-arterial 25% mannitol at rate of 4-12 ml/sec for 30 seconds. 25% Mannitol: Route of administration: In this study, the first step of the treatment will be performing osmotic blood-brain-barrier disruption with administration of intra-arterial 25% Mannitol into the appropriate cervical artery. Low-dose Intra-arterial bevacizumab: Route of administration: In this study, the second step of the treatment will be administering intra-arterial bevacizumab into the appropriate cervical artery. **ID:** OG000 **Title:** Low-dose Intra-arterial Avastin (Bevacizumab) #### Outcome Measure 4 **Description:** We will determine how severely headache affect the patient's life before and after treatment by performing the Migraine Disability Assessment (MIDAS) questionnaire. MIDAS PAIN LEVEL is Item 7 which rates the 3-month average headache PAIN LEVEL on scale from 0 to 10 where 0 = no pain at all, and 10 = pain as bad as it can be. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** 10 adult subjects presented with medically refractory radiation necrosis of the brain and disabling headache. **Reporting Status:** POSTED **Time Frame:** Baseline (before treatment), and 6 weeks, 3 months, 6 months, 9 months, and 12 months after single dose intra-arterial Avastin (bevacizumab) **Title:** Change in Headache Associated Morbidity Measured With MIDAS PAIN LEVEL After a Single Treatment of Low Dose Intra-arterial Avastin (Bevacizumab) Will be Measured. **Type:** SECONDARY **Unit of Measure:** score on a scale ##### Group **Description:** A single intra-arterial dose of 2.5 mg/kg bevacizumab will be administered after osmotic blood-brain-barrier disruption with intra-arterial 25% mannitol at rate of 4-12 ml/sec for 30 seconds. 25% Mannitol: Route of administration: In this study, the first step of the treatment will be performing osmotic blood-brain-barrier disruption with administration of intra-arterial 25% Mannitol into the appropriate cervical artery. Low-dose Intra-arterial bevacizumab: Route of administration: In this study, the second step of the treatment will be administering intra-arterial bevacizumab into the appropriate cervical artery. **ID:** OG000 **Title:** Low-dose Intra-arterial Avastin (Bevacizumab) #### Outcome Measure 5 **Description:** Quantitative change in headache will be assessed by performing the Headache Impact Test (HIT-6), which is a fixed-length 6-item questionnaire. The score for this questionnaire can range from 36 to 78, with 36 indicating minimum headache impact and the max score of 78 indicating worst possible headache impact. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** 10 adult subjects presented with medically refractory radiation necrosis of the brain and disabling headache. All 10 received single treatment of low dose intra-arterial bevacizumab. **Reporting Status:** POSTED **Time Frame:** Baseline (before treatment), and 6 weeks, 3 months, 6 months, 9 months, and 12 months after single dose intra-arterial Avastin (bevacizumab) **Title:** Change in Headache Measured With Headache Impact Test (HIT-6) After a Single Treatment of Low Dose Intra-arterial Avastin (Bevacizumab) **Type:** SECONDARY **Unit of Measure:** score on a scale ##### Group **Description:** A single intra-arterial dose of 2.5 mg/kg bevacizumab will be administered after osmotic blood-brain-barrier disruption with intra-arterial 25% mannitol at rate of 4-12 ml/sec for 30 seconds. 25% Mannitol: Route of administration: In this study, the first step of the treatment will be performing osmotic blood-brain-barrier disruption with administration of intra-arterial 25% Mannitol into the appropriate cervical artery. Low-dose Intra-arterial bevacizumab: Route of administration: In this study, the second step of the treatment will be administering intra-arterial bevacizumab into the appropriate cervical artery. **ID:** OG000 **Title:** Low-dose Intra-arterial Avastin (Bevacizumab) #### Outcome Measure 6 **Description:** Quantitative change in functional status will be assessed by performing Karnofsky Performance Status Scale (KPS). The KPS score can range from 0 to 100 where 0 is dead and 100 is fully alive and normal with no complaints and no evidence of disease. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** 10 adult subjects presented with medically refractory radiation necrosis of the brain and disabling headache. All were treated with steroids prior to study enrollment. **Reporting Status:** POSTED **Time Frame:** Baseline (before treatment), Day 1, and at 3 months, and 12 months after single dose intra-arterial Avastin (bevacizumab) **Title:** Change in Functional Status After a Single Treatment of Low Dose Intra-arterial Bevacizumab **Type:** SECONDARY **Unit of Measure:** units on a scale ##### Group **Description:** A single intra-arterial dose of 2.5 mg/kg bevacizumab will be administered after osmotic blood-brain-barrier disruption with intra-arterial 25% mannitol at rate of 4-12 ml/sec for 30 seconds. 25% Mannitol: Route of administration: In this study, the first step of the treatment will be performing osmotic blood-brain-barrier disruption with administration of intra-arterial 25% Mannitol into the appropriate cervical artery. Low-dose Intra-arterial Bevacizumab: Route of administration: In this study, the second step of the treatment will be administering intra-arterial bevacizumab into the appropriate cervical artery. **ID:** OG000 **Title:** Low-dose Intra-arterial Bevacizumab #### Outcome Measure 7 **Description:** To assess the utility of intra-arterial (IA) bevacizumab treatment in allowing decreased steroid usage, total cumulative days of steroid usage were compared between the 12 months PRIOR TO and the 12 months IMMEDIATELY FOLLOWING IA bevacizumab. Days of steroid usage were tabulated via medical history and chart review at the baseline visit for the 12 months prior to IA bevacizumab and post-treatment on days 0 and 1, week 6, and months 3, 6, 9 and 12 for the 12 month total after IA bevacizumab treatment. Excluding topical steroid preparations, all days of enteral or parenteral steroid intake of any dose were included in the cumulative summation. **Dispersion Type:** Inter-Quartile Range **Parameter Type:** MEDIAN **Population Description:** 10 adult patients age 31.0 (IQR 24.3, 42.8) years, including 8 (80%) women, 2 (20%) men, 9 (90%) white, 1 (10%) black, 9 (90%) Non-Hispanic, and 1 (10%) Hispanic **Reporting Status:** POSTED **Time Frame:** 12 months prior to single dose of IA bevacizumab; 12 months following single dose of IA bevacizumab **Title:** Change in Steroid Usage After a Single Treatment of Low Dose Intra-arterial Avastin (Bevacizumab) **Type:** SECONDARY **Unit of Measure:** Days ##### Group **Description:** A single intra-arterial dose of 2.5 mg/kg bevacizumab will be administered after osmotic blood-brain-barrier disruption with intra-arterial 25% mannitol at rate of 4-12 ml/sec for 30 seconds. 25% Mannitol: Route of administration: In this study, the first step of the treatment will be performing osmotic blood-brain-barrier disruption with administration of intra-arterial 25% Mannitol into the appropriate cervical artery. Low-dose Intra-arterial bevacizumab: Route of administration: In this study, the second step of the treatment will be administering intra-arterial bevacizumab into the appropriate cervical artery. **ID:** OG000 **Title:** Low-dose Intra-arterial Avastin (Bevacizumab) #### Outcome Measure 8 **Description:** In order to investigate post-operative changes in Neurocognitive performance after intra-arterial bevacizumab, patients who consented underwent formal neuropsychological battery testing. Sixteen subtests from the Neuropsychological Assessment Battery (Stern \& White, PAR Inc.) were chosen for brevity, sensitivity, and due to the wide range of available normative data (ages 18-97). For each subtest, T-score = 50 is the normative mean with SD = 10. Thus T-scores of 40-60 are within one standard deviation of the mean and are considered generally normal. Scores increasingly below 40 indicate decreased neurocognitive performance compared to healthy demographically matched persons. Scores significantly above 60 indicate increase neurocognitive performance compared to healthy demographically matched persons. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** 10 adult subjects, 2 male and 8 female, 1 black and 9 white, 1 Hispanic and 9 non-Hispanic, recruited from outpatient medical clinics at 2 academic medical centers (5 subjects per center) between Nov 2016 and Jan 2018. 2 subjects experienced progression of radiation necrosis requiring intervention at approx. month 11. Their 12-month data are not included in the Neurocognitive reporting. **Reporting Status:** POSTED **Time Frame:** Baseline (before bevacizumab), and 3 months and 12 months after single dose intra-arterial Avastin (bevacizumab) **Title:** Post-operative Neurocognitive Change After Intra-arterial Bevacizumab **Type:** SECONDARY **Unit of Measure:** T score where mean T=50, SD=10 ##### Group **Description:** A single intra-arterial dose of 2.5 mg/kg bevacizumab will be administered after osmotic blood-brain-barrier disruption with intra-arterial 25% mannitol at rate of 4-12 ml/sec for 30 seconds. 25% Mannitol: Route of administration: In this study, the first step of the treatment will be performing osmotic blood-brain-barrier disruption with administration of intra-arterial 25% Mannitol into the appropriate cervical artery. Low-dose Intra-arterial Bevacizumab: Route of administration: In this study, the second step of the treatment will be administering intra-arterial bevacizumab into the appropriate cervical artery. **ID:** OG000 **Title:** Low-dose Intra-arterial Bevacizumab ### Participant Flow Module #### Group **Description:** A single intra-arterial dose of 2.5 mg/kg bevacizumab will be administered after osmotic blood-brain-barrier disruption with intra-arterial 25% mannitol at rate of 4-12 ml/sec for 30 seconds. 25% Mannitol: Route of administration: In this study, the first step of the treatment will be performing osmotic blood-brain-barrier disruption with administration of intra-arterial 25% Mannitol into the appropriate cervical artery. Low-dose Intra-arterial Bevacizumab: Route of administration: In this study, the second step of the treatment will be administering intra-arterial bevacizumab into the appropriate cervical artery. **ID:** FG000 **Title:** Low-dose Intra-arterial Bevacizumab #### Period **Title:** Overall Study ##### Withdraw **Type:** Adverse Event ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 10 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 8 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 2 **Pre-Assignment Details:** This was a single-arm study. There was no wash out or run-in period after enrollment. There was no assignment to groups. **Recruitment Details:** Adults recruited from outpatient medical clinics at 2 academic medical centers (5 subjects per center) between Nov 2016 and Jan 2018. Enrollment was closed once 10 adult subjects were enrolled. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT04626479 **Brief Title:** Substudy 03A: A Study of Immune and Targeted Combination Therapies in Participants With First Line (1L) Renal Cell Carcinoma (MK-3475-03A) **Official Title:** A Phase 1b/2 Study of Immune and Targeted Combination Therapies in Participants With RCC (U03): Substudy 03A #### Organization Study ID Info **ID:** 3475-03A #### Organization **Class:** INDUSTRY **Full Name:** Merck Sharp & Dohme LLC #### Secondary ID Infos **Domain:** Merck **ID:** MK-3475-03A **Type:** OTHER **Domain:** EU CT **ID:** 2023-506838-68-00 **Type:** REGISTRY **ID:** 2019-003609-84 **Type:** EUDRACT_NUMBER ### Status Module #### Completion Date **Date:** 2026-05-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-04-18 **Type:** ACTUAL **Last Update Submit Date:** 2024-04-17 **Overall Status:** ACTIVE_NOT_RECRUITING #### Primary Completion Date **Date:** 2026-05-31 **Type:** ESTIMATED #### Start Date **Date:** 2020-12-16 **Type:** ACTUAL **Status Verified Date:** 2024-04 #### Study First Post Date **Date:** 2020-11-12 **Type:** ACTUAL **Study First Submit Date:** 2020-11-10 **Study First Submit QC Date:** 2020-11-10 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Merck Sharp & Dohme LLC #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Oversight Has DMC:** False ### Description Module **Brief Summary:** Substudy 03A is part of a larger research study that is testing experimental treatments for renal cell carcinoma (RCC). The larger study is the umbrella study (U03). The goal of substudy 03A is to evaluate the safety and efficacy of experimental combinations of investigational agents in participants with advanced first line (1L) clear cell renal cell carcinoma (ccRCC). This substudy will have two phases: a safety lead-in phase and an efficacy phase. The safety lead-in phase will be used to demonstrate a tolerable safety profile for the combination of investigational agents. There will be no hypothesis testing in this study. ### Conditions Module **Conditions:** - Carcinoma, Renal Cell **Keywords:** - receptor tyrosine kinase inhibitor - programmed cell death 1 (PD-1, PD1) - programmed cell death ligand 1 (PD-L1, PDL1) ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 400 **Type:** ESTIMATED **Phases:** - PHASE1 - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants will receive pembrolizumab/quavonlimab (coformulation of pembrolizumab 400 mg and quavonlimab 25 mg) PLUS lenvatinib 20 mg. Pembrolizumab/quavonlimab will be administered intravenously (IV) once every 6 weeks (Q6W) for up to 17 administrations (up to \~2 years). Lenvatinib will be administered orally once-daily (QD) until progressive disease or discontinuation. **Intervention Names:** - Drug: Lenvatinib - Biological: Pembrolizumab/Quavonlimab **Label:** Coformulation Pembrolizumab/Quavonlimab + Lenvatinib **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants will receive favezelimab/pembrolizumab (coformulation of favezelimab 800 mg and pembrolizumab 200 mg) PLUS lenvatinib 20 mg. Favezelimab/Pembrolizumab will be administered IV once every 3 weeks (Q3W) for up to 35 administrations (up to \~2 years). Lenvatinib will be administered orally QD until progressive disease or discontinuation. **Intervention Names:** - Biological: Favezelimab/Pembrolizumab - Drug: Lenvatinib **Label:** Coformulation Favezelimab/Pembrolizumab+ Lenvatinib **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Participants will receive pembrolizumab 400 mg PLUS belzutifan 120 mg PLUS lenvatinib 20 mg. Pembrolizumab will be administered IV Q6W for up to 17 administrations (up to \~2 years). Both belzutifan and lenvatinib will be administered orally QD until progressive disease or discontinuation. **Intervention Names:** - Biological: Pembrolizumab - Drug: Belzutifan - Drug: Lenvatinib **Label:** Pembrolizumab + Belzutifan + Lenvatinib **Type:** EXPERIMENTAL #### Arm Group 4 **Description:** Participants will receive pembrolizumab 400 mg PLUS lenvatinib 20 mg. Pembrolizumab will be administered IV Q6W for up to 17 administrations (up to \~ 2 years). Lenvatinib will be administered orally QD until progressive disease or discontinuation. **Intervention Names:** - Biological: Pembrolizumab - Drug: Lenvatinib **Label:** Pembrolizumab + Lenvatinib **Type:** EXPERIMENTAL #### Arm Group 5 **Description:** Participants will receive vibostolimab/pembrolizumab (coformulation of 200 mg vibostolimab and pembrolizumab 200 mg). Vibostolimab/pembrolizumab will be administered IV once every 3 weeks (Q3W) for up to 35 administrations (up to \~2 years). Belzutifan will be administered orally QD until progressive disease or discontinuation. **Intervention Names:** - Drug: Belzutifan - Drug: Vibostolimab/Pembrolizumab **Label:** Coformulation Vibostolimab/Pembrolizumab+Belzutifan **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Pembrolizumab + Belzutifan + Lenvatinib - Pembrolizumab + Lenvatinib **Description:** Administered via IV infusion at a dose of 400 mg Q6W **Name:** Pembrolizumab **Other Names:** - MK-3475 - KEYTRUDA® **Type:** BIOLOGICAL #### Intervention 2 **Arm Group Labels:** - Coformulation Favezelimab/Pembrolizumab+ Lenvatinib **Description:** Administered via IV infusion at a dose of 800 mg/200 mg Q3W **Name:** Favezelimab/Pembrolizumab **Other Names:** - MK-4280A **Type:** BIOLOGICAL #### Intervention 3 **Arm Group Labels:** - Coformulation Vibostolimab/Pembrolizumab+Belzutifan - Pembrolizumab + Belzutifan + Lenvatinib **Description:** Administered via oral tablet at a dose of 120 mg QD **Name:** Belzutifan **Other Names:** - MK-6482 - WELIREG™ **Type:** DRUG #### Intervention 4 **Arm Group Labels:** - Coformulation Favezelimab/Pembrolizumab+ Lenvatinib - Coformulation Pembrolizumab/Quavonlimab + Lenvatinib - Pembrolizumab + Belzutifan + Lenvatinib - Pembrolizumab + Lenvatinib **Description:** Administered via oral capsule at a dose of 20 mg QD **Name:** Lenvatinib **Other Names:** - MK-7902 - E7080 - LENVIMA® **Type:** DRUG #### Intervention 5 **Arm Group Labels:** - Coformulation Pembrolizumab/Quavonlimab + Lenvatinib **Description:** Administered via IV infusion at a dose of 400 mg/25 mg Q6W **Name:** Pembrolizumab/Quavonlimab **Other Names:** - MK-1308A **Type:** BIOLOGICAL #### Intervention 6 **Arm Group Labels:** - Coformulation Vibostolimab/Pembrolizumab+Belzutifan **Description:** Administered via IV infusion at a dose of 200 mg/200 mg Q6W **Name:** Vibostolimab/Pembrolizumab **Other Names:** - MK-7684A **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** DLTs are defined as one or more of the following toxicities: (1) grade (gr) 4 nonhematologic toxicity (2) gr 4 hematologic toxicity lasting \>7 days OR gr 4 platelet count decreased of any duration OR gr 3 platelet count decreased if associated with bleeding (3) gr 3 nonhematologic toxicity except gr 3 fatigue (lasting ≤3 days), diarrhea, nausea, vomiting or rash without standard of care (4) gr 3 or 4 nonhematologic abnormality if medical intervention is required or if it leads to hospitalization or persists for \>1 week (5) gr 3 or 4 febrile neutropenia (6) gr 3 or 4 alanine aminotransferase, aspartate aminotransferase and/or bilirubin laboratory value (7) treatment related adverse event (AE) causing study intervention discontinuation during the first 21 days (8) treatment related AE causing intervention administration delay for \>14 days during the first 21 days (9) gr 5 toxicity. The number of participants who experience one or more DLTs in the safety lead-in phase will be presented. **Measure:** Safety Lead-in Phase: Number of participants who experience one or more dose-limiting toxicities (DLTs) **Time Frame:** Up to ~21 days **Description:** An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. The number of participants who experience one or more AEs in the safety lead-in phase will be presented. **Measure:** Safety Lead-in Phase: Number of participants who experience one or more adverse events (AEs) **Time Frame:** Up to ~21 days **Description:** An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. The number of participants who discontinue study treatment due to an AE in the safety lead-in phase will be presented. **Measure:** Safety Lead-in Phase: Number of participants who discontinue study treatment due to an AE **Time Frame:** Up to ~21 days **Description:** DLTs are defined as one or more of the following toxicities: (1) grade (gr) 4 nonhematologic toxicity (2) gr 4 hematologic toxicity lasting \>7 days OR gr 4 platelet count decreased of any duration OR gr 3 platelet count decreased if associated with bleeding (3) gr 3 nonhematologic toxicity except gr 3 fatigue (lasting ≤3 days), diarrhea, nausea, vomiting or rash without standard of care (4) gr 3 or 4 nonhematologic abnormality if medical intervention is required or if it leads to hospitalization or persists for \>1 week (5) gr 3 or 4 febrile neutropenia (6) gr 3 or 4 alanine aminotransferase, aspartate aminotransferase and/or bilirubin laboratory value (7) treatment related adverse event (AE) causing study intervention discontinuation during the first 21 days (8) treatment related AE causing intervention administration delay for \>14 days during the first 21 days (9) gr 5 toxicity. The number of participants who experience one or more DLTs in the efficacy phase will be presented. **Measure:** Efficacy Phase: Number of participants who experience one or more DLTs **Time Frame:** Up to ~21 days **Description:** An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. The number of participants who experience one or more AEs in the efficacy phase will be presented. **Measure:** Efficacy Phase: Number of participants who experience one or more AEs **Time Frame:** Up to ~43 months **Description:** An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. The number of participants who discontinue study treatment due to an AE in the efficacy phase will be presented. **Measure:** Efficacy Phase: Number of participants who discontinue study treatment due to an AE **Time Frame:** Up to ~43 months **Description:** ORR is defined as the percentage of participants in the analysis population who have a complete response (CR: disappearance of all target lesions) or partial response (PR: at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum of diameters). Responses are according to Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST 1.1) by blinded independent central review (BICR). **Measure:** Efficacy Phase: Objective response rate (ORR) **Time Frame:** Up to ~43 months #### Secondary Outcomes **Description:** For participants in the analysis population who demonstrate a confirmed CR (disappearance of all target lesions) or confirmed PR (at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum of diameters), DOR is defined as the time from first documented evidence of CR or PR until progressive disease or death due to any cause, whichever occurs first. Responses are according to RECIST 1.1 by BICR. **Measure:** Efficacy Phase: Duration of response (DOR) **Time Frame:** Up to ~43 months **Description:** PFS is defined as the time from randomization to the first documented progressive disease or death due to any cause, whichever occurs first. Responses are according to RECIST 1.1 by BICR. **Measure:** Efficacy Phase: Progression-free survival (PFS) **Time Frame:** Up to ~43 months **Description:** OS is defined as the time from randomization to death due to any cause. **Measure:** Efficacy Phase: Overall survival (OS) **Time Frame:** Up to ~43 months **Description:** CBR is defined as the percentage of participants who have achieved CR (disappearance of all target lesions) or PR (at least a 30% decrease in the sum of diameters of target lesions, taking as reference the baseline sum of diameters) or stable disease (SD: neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease) of ≥6 months. Responses are according to RECIST 1.1 by BICR. **Measure:** Efficacy Phase: Clinical benefit rate (CBR) **Time Frame:** Up to ~43 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Has a histologically confirmed diagnosis of locally advanced/metastatic ccRCC * Has received no prior systemic therapy for advanced RCC; prior neoadjuvant/adjuvant therapy for RCC is acceptable if completed ≥12 months before randomization/allocation. * Is able to swallow oral medication * Has adequate organ function * Participants receiving bone resorptive therapy must have therapy initiated at least 2 weeks before randomization/allocation * Has resolution of toxic effects of the most recent prior therapy to ≤Grade 1 * Has adequately controlled blood pressure (BP ≤150/90 mm Hg) with no change in hypertensive medications within 1 week before randomization/allocation * Male participants are abstinent from heterosexual intercourse or agree to use contraception during treatment with and for at least 7 days after the last dose of lenvatinib and /or belzutifan; 7 days after lenvatinib and/or belzutifan is stopped, if the participant is only receiving pembrolizumab, pembrolizumab/quavonlimab, favezelimab/pembrolizumab or a combination of the aforementioned drugs, no contraception is needed * Female participants must not be pregnant and not be a woman of childbearing potential (WOCBP) or is a WOCBP abstinent from heterosexual intercourse or using contraception during the intervention period and for at least 120 days after the last dose of pembrolizumab, pembrolizumab/quavonlimab, favezelimab/pembrolizumab for 30 days after the last dose of lenvatinib or belzutifan, whichever occurs last and must abstain from breastfeeding during the study intervention period and for at least 120 days after study intervention Exclusion Criteria: * Has urine protein ≥1 g/24 hours and has any of the following: (a) a pulse oximeter reading \<92% at rest, or (b) requires intermittent supplemental oxygen, or (c) requires chronic supplemental oxygen (d) active hemoptysis within 3 weeks prior to the first dose of study intervention * Has clinically significant cardiovascular disease within 12 months from the first dose of study intervention administration * Has had major surgery within 3 weeks before first dose of study interventions * Has a history of lung disease * Has a history of inflammatory bowel disease * Has preexisting gastrointestinal (GI) or non-GI fistula * Has malabsorption due to prior GI surgery or disease * Has received prior radiotherapy within 2 weeks of start of study intervention * Has received a live or live attenuated vaccine within 30 days before the first dose of study drug; killed vaccines are allowed * Has received more than 4 previous systemic anticancer treatment regimens * Has a diagnosis of immunodeficiency or is receiving any form of immunosuppressive therapy within 7 days prior to the first dose of study intervention * Has known additional malignancy that is progressing or has required active treatment within the past 3 years * Has known central nervous system (CNS) metastases and/or carcinomatous meningitis * Has an active autoimmune disease that has required systemic treatment in the past 2 years; replacement therapy is not considered a form of systemic treatment and is allowed * Has an active infection requiring systemic therapy * Has a known history of human immunodeficiency virus (HIV) infection * Has a known history of Hepatitis B * Has had an allogenic tissue/solid organ transplant **Maximum Age:** 120 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** San Francisco **Country:** United States **Facility:** University of California at San Francisco ( Site 1008) **State:** California **Zip:** 94158 **Location 2:** **City:** New Haven **Country:** United States **Facility:** Yale-New Haven Hospital-Yale Cancer Center ( Site 1011) **State:** Connecticut **Zip:** 06510 **Location 3:** **City:** Chicago **Country:** United States **Facility:** University of Chicago ( Site 1013) **State:** Illinois **Zip:** 60637 **Location 4:** **City:** Iowa City **Country:** United States **Facility:** University of Iowa ( Site 1012) **State:** Iowa **Zip:** 52242 **Location 5:** **City:** Detroit **Country:** United States **Facility:** Henry Ford Health System ( Site 1014) **State:** Michigan **Zip:** 48202 **Location 6:** **City:** New York **Country:** United States **Facility:** Laura and Isaac Perlmutter Cancer Center ( Site 1016) **State:** New York **Zip:** 10016 **Location 7:** **City:** New York **Country:** United States **Facility:** Memorial Sloan Kettering Cancer Center ( Site 1002) **State:** New York **Zip:** 10065 **Location 8:** **City:** Durham **Country:** United States **Facility:** Duke Cancer Institute ( Site 1015) **State:** North Carolina **Zip:** 27710 **Location 9:** **City:** Pittsburgh **Country:** United States **Facility:** UPMC Cancer Center/Hillman Cancer Center ( Site 1017) **State:** Pennsylvania **Zip:** 15232 **Location 10:** **City:** Dallas **Country:** United States **Facility:** UTSW Medical Center ( Site 1003) **State:** Texas **Zip:** 75390 **Location 11:** **City:** Blacktown **Country:** Australia **Facility:** Western Sydney Local Health District ( Site 1601) **State:** New South Wales **Zip:** 2148 **Location 12:** **City:** Kogarah **Country:** Australia **Facility:** St George Hospital ( Site 1602) **State:** New South Wales **Zip:** 2217 **Location 13:** **City:** Herston **Country:** Australia **Facility:** Royal Brisbane and Women s Hospital ( Site 1603) **State:** Queensland **Zip:** 4029 **Location 14:** **City:** Heidelberg **Country:** Australia **Facility:** Austin Health ( Site 1600) **State:** Victoria **Zip:** 3084 **Location 15:** **City:** Toronto **Country:** Canada **Facility:** Princess Margaret Cancer Centre ( Site 1101) **State:** Ontario **Zip:** M5G 1Z5 **Location 16:** **City:** Montreal **Country:** Canada **Facility:** Jewish General Hospital ( Site 1100) **State:** Quebec **Zip:** H3T 1E2 **Location 17:** **City:** Temuco **Country:** Chile **Facility:** James Lind Centro de Investigación del Cáncer ( Site 2108) **State:** Araucania **Zip:** 4800827 **Location 18:** **City:** Temuco **Country:** Chile **Facility:** CIDO SpA-Oncology ( Site 2106) **State:** Araucania **Zip:** 4810148 **Location 19:** **City:** Santiago **Country:** Chile **Facility:** FALP-UIDO ( Site 2100) **State:** Region M. De Santiago **Zip:** 7500921 **Location 20:** **City:** Santiago **Country:** Chile **Facility:** Oncovida ( Site 2107) **State:** Region M. De Santiago **Zip:** 7510032 **Location 21:** **City:** Santiago **Country:** Chile **Facility:** Bradfordhill-Clinical Area ( Site 2101) **State:** Region M. De Santiago **Zip:** 8420383 **Location 22:** **City:** Viña del Mar **Country:** Chile **Facility:** ONCOCENTRO APYS-ACEREY ( Site 2103) **State:** Valparaiso **Zip:** 2520598 **Location 23:** **City:** Valledupar **Country:** Colombia **Facility:** Sociedad De Oncologia Y Hematologia Del Cesar-Oncology ( Site 1905) **State:** Cesar **Zip:** 200001 **Location 24:** **City:** Montería **Country:** Colombia **Facility:** Oncomédica S.A.S ( Site 1904) **State:** Cordoba **Zip:** 230002 **Location 25:** **City:** Bogota **Country:** Colombia **Facility:** Clinica Colsanitas S.A, Sede Clínica Universitaria Colombia-Center Investigator ( Site 1900) **State:** Distrito Capital De Bogota **Zip:** 111321 **Location 26:** **City:** Cali **Country:** Colombia **Facility:** Fundación Valle del Lili ( Site 1901) **State:** Valle Del Cauca **Zip:** 760032 **Location 27:** **City:** Vandoeuvre les Nancy **Country:** France **Facility:** Institut De Cancerologie De Lorraine ( Site 1204) **State:** Ain **Zip:** 54519 **Location 28:** **City:** Strasbourg **Country:** France **Facility:** Institut de cancérologie Strasbourg Europe (ICANS) ( Site 1203) **State:** Alsace **Zip:** 67200 **Location 29:** **City:** Toulouse Cedex 9 **Country:** France **Facility:** Institut Claudius Regaud ( Site 1200) **State:** Haute-Garonne **Zip:** 31059 **Location 30:** **City:** Villejuif **Country:** France **Facility:** Gustave Roussy ( Site 1202) **State:** Val-de-Marne **Zip:** 94800 **Location 31:** **City:** Budapest **Country:** Hungary **Facility:** Országos Onkológiai Intézet-Urogenitális Tumorok és Klinikai Farmakológiai Osztály ( Site 2301) **State:** Pest **Zip:** 1122 **Location 32:** **City:** Haifa **Country:** Israel **Facility:** Rambam MC ( Site 1500) **Zip:** 3525408 **Location 33:** **City:** Jerusalem **Country:** Israel **Facility:** Hadassah Medical Center-Oncology ( Site 1504) **Zip:** 9112001 **Location 34:** **City:** Petah Tiqwa **Country:** Israel **Facility:** Rabin Medical Center ( Site 1502) **Zip:** 4941492 **Location 35:** **City:** Ramat Gan **Country:** Israel **Facility:** Sheba Medical Center - Oncology Division ( Site 1501) **Zip:** 52621 **Location 36:** **City:** Tel Aviv **Country:** Israel **Facility:** Sourasky Medical Center ( Site 1503) **Zip:** 6423906 **Location 37:** **City:** Songpagu **Country:** Korea, Republic of **Facility:** Asan Medical Center ( Site 1800) **State:** Seoul **Zip:** 05505 **Location 38:** **City:** Seoul **Country:** Korea, Republic of **Facility:** Severance Hospital ( Site 1802) **Zip:** 03722 **Location 39:** **City:** Seoul **Country:** Korea, Republic of **Facility:** Samsung Medical Center ( Site 1801) **Zip:** 06351 **Location 40:** **City:** Amsterdam **Country:** Netherlands **Facility:** Nederlands Kanker Instituut - Antoni van Leeuwenhoek (NKI-AVL)-medical oncology ( Site 2402) **State:** Noord-Holland **Zip:** 1066CX **Location 41:** **City:** Rotterdam **Country:** Netherlands **Facility:** Erasmus Medisch Centrum ( Site 2401) **State:** Zuid-Holland **Zip:** 3015 GD **Location 42:** **City:** Auckland **Country:** New Zealand **Facility:** Auckland City Hospital ( Site 1700) **Zip:** 1023 **Location 43:** **City:** Bydgoszcz **Country:** Poland **Facility:** Centrum Onkologii im. Prof. Franciszka Lukaszczyka-Ambulatorium Chemioterapii ( Site 2201) **State:** Kujawsko-pomorskie **Zip:** 85-796 **Location 44:** **City:** Warszawa **Country:** Poland **Facility:** Narodowy Instytut Onkologii im. Marii Sklodowskiej-Curie - P-Oddzial Badan Wczesnych Faz ( Site 2200 **State:** Mazowieckie **Zip:** 02-781 **Location 45:** **City:** Gdańsk **Country:** Poland **Facility:** Uniwersyteckie Centrum Kliniczne-Early Clinical Trials Unit ( Site 2202) **State:** Pomorskie **Zip:** 80-952 **Location 46:** **City:** Barcelona **Country:** Spain **Facility:** Hospital Universitari Vall d Hebron ( Site 1300) **State:** Cataluna **Zip:** 08035 **Location 47:** **City:** Madrid **Country:** Spain **Facility:** Hospital Universitario Ramon y Cajal ( Site 1301) **Zip:** 28034 **Location 48:** **City:** Southampton **Country:** United Kingdom **Facility:** Southampton General Hospital ( Site 1403) **State:** England **Zip:** SO16 6YD **Location 49:** **City:** Glasgow **Country:** United Kingdom **Facility:** The Beatson West of Scotland Cancer Centre ( Site 1405) **State:** Glasgow City **Zip:** G12 0YN **Location 50:** **City:** Preston **Country:** United Kingdom **Facility:** Royal Preston Hospital ( Site 1406) **State:** Lancashire **Zip:** PR2 9HT **Location 51:** **City:** Leicester **Country:** United Kingdom **Facility:** Leicester Royal Infirmary ( Site 1408) **State:** Leicestershire **Zip:** LE1 5WW **Location 52:** **City:** London **Country:** United Kingdom **Facility:** Barts Health NHS Trust ( Site 1401) **State:** London, City Of **Zip:** EC1A 7BE **Location 53:** **City:** Edinburgh **Country:** United Kingdom **Facility:** Western General Hospital ( Site 1402) **State:** Midlothian **Zip:** EH4 2XU **Location 54:** **City:** Cardiff **Country:** United Kingdom **Facility:** Velindre Cancer Centre Hospital ( Site 1407) **State:** Wales **Zip:** CF14 2TL **Location 55:** **City:** Manchester **Country:** United Kingdom **Facility:** The Christie NHS Foundation Trust ( Site 1400) **Zip:** M20 4BX #### Overall Officials **Official 1:** **Affiliation:** Merck Sharp & Dohme LLC **Name:** Medical Director **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **Description:** http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf **IPD Sharing:** YES **URL:** http://engagezone.msd.com/ds_documentation.php ### References Module #### See Also Links **Label:** Merck Oncology Clinical Trials Information **URL:** http://merckoncologyclinicaltrials.com **Label:** Plain Language Summary **URL:** https://trialstransparency.merckclinicaltrials.com/Study.aspx?id=3475-03A&kw=3475-03A ## 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: BC23 - Name: Symptoms and General Pathology - 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: Carcinoma, Renal Cell - ID: M6845 - Name: Death - 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: 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: Carcinoma, Renal Cell - ID: T1341 - Name: Clear Cell Renal Cell Carcinoma - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000002277 - Term: Carcinoma - ID: D000002292 - Term: Carcinoma, Renal Cell ### 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: 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: M348353 - Name: Belzutifan - Relevance: HIGH - As Found: Time before - ID: M349416 - Name: Pembrolizumab - Relevance: HIGH - As Found: Blind - ID: M353738 - Name: Lenvatinib - Relevance: HIGH - As Found: Fibrillation - ID: M2889 - Name: Tyrosine Kinase Inhibitors - Relevance: LOW - As Found: Unknown - 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: C000531958 - Term: Lenvatinib - ID: C000720612 - Term: Belzutifan ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02616679 **Brief Title:** Cognitive Detection of Preclinical AD: Validation Using Biomarkers **Official Title:** Cognitive Detection of Preclinical AD: Validation Using Biomarkers #### Organization Study ID Info **ID:** 13-00706 #### Organization **Class:** OTHER **Full Name:** NYU Langone Health ### Status Module #### Completion Date **Date:** 2020-08-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-01-15 **Type:** ACTUAL **Last Update Submit Date:** 2021-01-13 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2020-08-12 **Type:** ACTUAL #### Start Date **Date:** 2016-01-05 **Type:** ACTUAL **Status Verified Date:** 2021-01 #### Study First Post Date **Date:** 2015-11-30 **Type:** ESTIMATED **Study First Submit Date:** 2015-11-25 **Study First Submit QC Date:** 2015-11-25 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Janssen Scientific Affairs, LLC #### Lead Sponsor **Class:** OTHER **Name:** NYU Langone Health #### 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 current study aims to validate several novel cognitive tasks expected to be sensitive to brain impairment in specific anatomic regions affected in preclinical Alzheimer's disease(pAD). The tasks are validated in 60 cognitively and clinically normal participants ages 60 - 85, inclusive, against reasonably well-established biomarkers of Alzheimer's disease, including 1) simultaneous positron emission tomography (PET) \[18F\]Flutemetamol amyloid and CT imaging and 2) to the extent data is available from other studies, participants' brain MRI and cerebral spinal fluid (CSF) amyloid and tau. **Detailed Description:** Biomarkers, such as amyloid deposition, and Hipp volume loss, and low Aβ and high pTau in CSF, are useful for identifying cognitively normal (CN) elderly who are likely have early AD pathology ("preclinical AD"). However, they are invasive and/or expensive. The goal of the current study is to develop and validate cognitive proxies of AD biomarkers by using cognitive tasks that are dependent on brain regions impaired by very early AD pathology. If successful, these tasks will provide a non-invasive and cost-effective way to identify and track change in CN individuals at high risk for progressing to mild cognitive impairment (MCI) and dementia stages of AD and thus will facilitate future prevention trials in pAD. Subjects will attend three study visits. During the first study visit, subjects will have eligibility criteria confirmed, have a blood sample drawn, and complete about half of the cognitive tasks. The second visit, which will occur within one week of visit one, will involve completion of the remaining cognitive tasks. Subjects will also be asked to have a PET-CT scan during visit three (to occur within 3 months of visits 1 and 2). ### Conditions Module **Conditions:** - Mild Cognitive Impairment ### Design Module #### Bio Spec **Description:** Any remaining blood specimens will be retained indefinitely for future use by the Center for Cognitive Neurology (CCN) at NYU Langone Medical Center. Some samples may also be sent to Janssen Research \& Development, LLC for potential future research use. Potential future research use by study investigators or collaborators will include possible discovery of novel biomarkers associated with increased AD risk, and study of validity of the use of such markers in preclinical AD. True genetic testing will not be done on these samples. Subjects may decline to have their samples stored for future use by checking the applicable box on the informed consent form. Subjects who agreed to have their samples stored for future use may revoke this permission. **Retention:** SAMPLES_WITH_DNA #### Design Info **Observational Model:** COHORT **Time Perspective:** CROSS_SECTIONAL #### Enrollment Info **Count:** 81 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants will be deemed cognitively normal based on criteria set forth by the National Alzheimer's Disease Coordinating Center/Alzheimer's Disease Centers (ADCC/ADC). **Label:** Cognitively Normal #### Arm Group 2 **Description:** Participants will be deemed aMCI based on criteria set forth by the National Alzheimer's Disease Coordinating Center/Alzheimer's Disease Centers (ADCC/ADC). **Label:** Amnestic Mild Cognitive Impairment (aMCI) ### Outcomes Module #### Primary Outcomes **Description:** The overall score on the cognitive tests will be correlated via a linear regression with biomarker data collected during the study. Each biomarker will be regressed individually. Biomarker data includes: MRI hippocampus (hipp) volume, entorhinal cortex (EC) volume, EC standard uptake value ratios (SUVRs), Hipp SUVR, precuneus (PCu) SUVR, PET-\[18F\] Flutemetamol SUVR, CSF Aβ and tau levels, levels of diffusion tensor imaging mean diffusivity, and levels of fractional anisotropy. **Measure:** Correlation of composite score on cognitive tests with biomarkers **Time Frame:** 3 months #### Secondary Outcomes **Measure:** Presence of ApoE allele **Time Frame:** 3 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Prior enrollment as a participant in the NYU Alzheimer's Disease Center (ADC) and completion of the ADC Clinical Evaluation within the past year. * Clinical diagnosis of "cognitively normal" or "amnestic mild cognitive impairment" based on recent (within 1 year) consensus meeting cross-referenced with standard neuropsychological scores. * Normal or corrected-to-normal vision and hearing (able to see images on computer screen and hear auditory events delivered through the computer speaker). Exclusion Criteria: * Significant history of mental illness, drug or alcohol abuse; severe trauma preventing normal use of dominant hand (needed to move the mouse cursor); clinical depression (unless medically controlled); other neurologic conditions (i.e. stroke), or learning disability; ophthalmologic/visual problems that prevent viewing a computer screen at a normal distance (such as legal blindness, detached retinas, occlusive cataracts). * Lack of capacity to give informed consent and no legally authorized representative to provide consent. * Having pacemakers, aneurysm clips, cochlear implants, or metal/foreign objects in body and therefore, unable to receive MRI. * Pregnancy, breastfeeding or planning to have a baby. **Healthy Volunteers:** True **Maximum Age:** 85 Years **Minimum Age:** 60 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Participants will be elderly individuals who are participating in ongoing clinical research at the NYU CCN, including at the ADC and the Center for Brain Health (CBH). Participants may also be recruited through community outreach or referrals from other centers. Cognitively normal (CN) participants and participants with amnestic mild cognitive impairment (aMCI) will be enrolled. ### Contacts Locations Module #### Locations **Location 1:** **City:** New York **Country:** United States **Facility:** NYU Langone Medical Center **State:** New York **Zip:** 10016 #### Overall Officials **Official 1:** **Affiliation:** NYU Langone Medical Center **Name:** Martin Sadowski, PhD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000003072 - Term: Cognition Disorders - 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 ### Condition Browse Module - Browse Leaves - ID: M29705 - Name: Cognitive Dysfunction - Relevance: HIGH - As Found: Mild Cognitive Impairment - ID: M6301 - Name: Cognition Disorders - 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: D000060825 - Term: Cognitive Dysfunction ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00803179 **Brief Title:** Growth Hormone Therapy for Wasting in Cystic Fibrosis **Official Title:** Growth Hormone Therapy for Wasting in Cystic Fibrosis #### Organization Study ID Info **ID:** 900005 #### Organization **Class:** OTHER **Full Name:** University of Massachusetts, Worcester ### Status Module #### Completion Date **Date:** 2012-03 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2013-01-15 **Type:** ESTIMATED **Last Update Submit Date:** 2012-12-10 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2012-03 **Type:** ACTUAL #### Results First Post Date **Date:** 2013-01-15 **Type:** ESTIMATED **Results First Submit Date:** 2012-11-13 **Results First Submit QC Date:** 2012-12-10 #### Start Date **Date:** 2008-11 **Status Verified Date:** 2012-12 #### Study First Post Date **Date:** 2008-12-05 **Type:** ESTIMATED **Study First Submit Date:** 2008-12-04 **Study First Submit QC Date:** 2008-12-04 **Why Stopped:** Poor enrollment, patients were lost to follow up ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Massachusetts, Worcester #### Responsible Party **Investigator Affiliation:** University of Massachusetts, Worcester **Investigator Full Name:** Michael Stalvey **Investigator Title:** Study Principle Investigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Our hypothesis is that Growth Hormone (GH) will not only target the peripheral tissue to stimulate weight and muscle growth which will maximize nutritional potential and improve overall quality of life. We theorize that this will occur through a multitude of factors: increased appetite, more constructive utilization of caloric intake and decreased catabolic signaling. The first aim will address changes in weight and lean body mass following the institution of GH therapy in adults with Cystic Fibrosis (CF) related wasting. The second aim will measure impact on quality of life of these individuals. Additionally, the third aim will monitor effects of GH therapy on diabetes and insulin sensitivity. Finally, the fourth aim will observe changes in the subjects underlying diagnosis of CF, specifically lung function, muscle strength and inflammatory state. **Detailed Description:** The following is a more detailed description of the aims listed above: Specific Aim 1: Measure change in weight in adults with CF related wasting following GH therapy. 1.1) Monitor weight gained or loss from baseline. 1.2) Assess changes in fat free mass from baseline by bioelectrical impedence analysis. Specific Aim 2: Evaluate overall quality of life (QOL) in adults with CF related wasting treated with GH therapy. 2.1) Perform CF disease-specific and general QOL analysis via CF QOL questionnaires. 2.2) Monitor compliance with therapy via subject report. Specific Aim 3: Monitor impact of GH therapy in relation to CF related diabetes onset or control. 3.1) Measure impact on insulin sensitivity in non-diabetes subjects 3.2) Observe change in exogenous insulin requirements and glycemic control in subjects with diabetes. Specific Aim 4: Quantify impact of anabolic therapy on manifestations of underlying diagnosis associated with CF. 4.1) Observe changes in lung function from baseline during GH therapy. 4.2) Determine changes in overall muscle strength via hand grip and six minute walk. 4.3) Evaluate changes in serum markers. ### Conditions Module **Conditions:** - Cystic Fibrosis **Keywords:** - CF ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 5 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Nutropin Aqueous (AQ): Initiation treatment for adult males is 0.2mg/d and for women 0.4mg/d **Intervention Names:** - Drug: Nutropin AQ **Label:** Growth Hormone Therapy **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Growth Hormone Therapy **Description:** Based on recommendations from the 2007 GH Deficiency Consensus Workshop on adult GH deficiency, the recommended initiation of treatment for adult males is 0.2mg/d and for women 0.4mg/d, with a titration upwards based on insulin-like growth factor (IGF-1) (product of GH stimulation at target tissues) levels and patient response. IGF-1 will be monitored at the 3,4,5 and 11 month intervals. For subjects under the age of 25 with an open epiphysis of the hand and/or wrist we will treat with the dose of 0.3mg/kg/week. Subjects will be on growth hormone for 8 months with a baseline visit prior to initiation of therapy and a 3 month follow-up visit after stopping therapy. **Name:** Nutropin AQ **Other Names:** - Growth Hormone (GH) **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Measure Change in Weight in Adults With Cystic Fibrosis (CF) Related Wasting Following Growth Hormone (GH) Therapy **Time Frame:** 14 months #### Secondary Outcomes **Measure:** Evaluate Overall Quality of Life (QOL) in Adults With CF Related Wasting Treated With GH Therapy **Time Frame:** 14 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Ability to provide written informed consent and comply with study assessments for the full duration of the study. * Age \> 18 years * Cystic fibrosis, diagnosed by either sweat chloride or genetic testing * Less than 92% ideal body weight based on body mass index (BMI) of 22 for women and 23 for men * Moderate or better pulmonary function (Forced Expiratory Volume (FEV1) \>40% of predicted). * Agree to use an effective method of birth control to prevent pregnancy during the research study. Women should not nurse (breast feed) a baby while on this study because Nutropin AQ may enter breast milk and possibly harm the child. Exclusion Criteria: * Pregnancy (positive pregnancy test) prior enrollment in the study * Any other condition that the investigator believes would pose a significant hazard to the subject if the investigational therapy were initiated * Participation in another simultaneous medical investigation or trial * Pediatric patients * Active neoplasm * History of organ transplantation * Prader Willi Syndrome who are severely obese or have severe respiratory impairment * Patients with hepatic impairment resulting in abnormal coagulation studies (\>1.5 times normal reference range) * Poorly controlled diabetes as determined by a Hemoglobin A1c greater than or equal to 9.0%. * Individuals with electrocardiogram abnormality or cardiac pacing. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Worcester **Country:** United States **Facility:** Umms/Ummhc **State:** Massachusetts **Zip:** 01655 #### Overall Officials **Official 1:** **Affiliation:** Unversity of Massachusetts Medical School **Name:** Michael Stalvey, MD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Thaker V, Carter B, Putman M. Recombinant growth hormone therapy for cystic fibrosis in children and young adults. Cochrane Database Syst Rev. 2021 Aug 23;8(8):CD008901. doi: 10.1002/14651858.CD008901.pub5. **PMID:** 34424546 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000010335 - Term: Pathologic Processes - ID: D000010182 - Term: Pancreatic Diseases - ID: D000004066 - Term: Digestive System Diseases - ID: D000008171 - Term: Lung Diseases - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000030342 - Term: Genetic Diseases, Inborn - ID: D000007232 - Term: Infant, Newborn, Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC06 - Name: Digestive System Diseases - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth - 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: M6755 - Name: Cystic Fibrosis - Relevance: HIGH - As Found: Cystic Fibrosis - ID: M8485 - Name: Fibrosis - Relevance: HIGH - As Found: Fibrosis - ID: M21265 - Name: Wasting Syndrome - Relevance: LOW - As Found: Unknown - ID: M5363 - Name: Cachexia - Relevance: LOW - As Found: Unknown - ID: M13102 - Name: Pancreatic 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: M11168 - Name: Lung Diseases - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown - ID: M23686 - Name: Genetic Diseases, Inborn - Relevance: LOW - As Found: Unknown - ID: M10276 - Name: Infant, Newborn, Diseases - Relevance: LOW - As Found: Unknown - ID: T1710 - Name: Cystic Fibrosis - Relevance: HIGH - As Found: Cystic Fibrosis ### Condition Browse Module - Meshes - ID: D000003550 - Term: Cystic Fibrosis - ID: D000005355 - Term: Fibrosis ### Intervention Browse Module - Ancestors - ID: D000006730 - Term: Hormones, Hormone Substitutes, and Hormone Antagonists - 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: LOW - As Found: Unknown - ID: M16759 - Name: Tin Fluorides - Relevance: LOW - As Found: Unknown - ID: M9789 - Name: Hormones - Relevance: HIGH - As Found: Loss - ID: M173166 - Name: Insulin, Globin Zinc - Relevance: LOW - As Found: Unknown - ID: M11900 - Name: Mitogens - Relevance: LOW - As Found: Unknown - ID: M9788 - Name: Hormone Antagonists - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000006728 - Term: Hormones ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Growth Hormone Therapy **Description:** Nutropin AQ: Initiation treatment for adult males is 0.2mg/d and for women 0.4mg/d **ID:** EG000 **Other Num at Risk:** 5 **Serious Number At Risk:** 5 **Title:** Growth Hormone Therapy **Frequency Threshold:** 0 ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 5 **Units:** Participants ### Group **ID:** BG000 **Title:** Growth Hormone Therapy **Description:** Nutropin AQ: Initiation treatment for adult males is 0.2mg/d and for women 0.4mg/d ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** <=18 years #### Measurement **Group ID:** BG000 **Value:** 5 **Category Title:** Between 18 and 65 years #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** >=65 years **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 5 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 5 **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 **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 ## Results Section - More Information Module ### Certain Agreement **PI Sponsor Employee:** True ### Point of Contact **Email:** [email protected] **Organization:** UMass Medical School **Phone:** 508-856-4280 **Title:** Michael Stalvey, MD, Principal Investigator ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 #### Outcome Measure 2 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Reporting Status:** POSTED **Time Frame:** 14 months **Title:** Measure Change in Weight in Adults With Cystic Fibrosis (CF) Related Wasting Following Growth Hormone (GH) Therapy **Type:** PRIMARY ##### Group **Description:** Nutropin Aqueous (AQ): Initiation treatment for adult males is 0.2mg/d and for women 0.4mg/d **ID:** OG000 **Title:** Growth Hormone Therapy #### Outcome Measure 2 **Reporting Status:** NOT_POSTED **Time Frame:** 14 months **Title:** Evaluate Overall Quality of Life (QOL) in Adults With CF Related Wasting Treated With GH Therapy **Type:** SECONDARY ### Participant Flow Module #### Group **Description:** Nutropin AQ: Initiation treatment for adult males is 0.2mg/d and for women 0.4mg/d **ID:** FG000 **Title:** Growth Hormone Therapy #### Period **Title:** Overall Study ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 3 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 5 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 2 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 3 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT06077279 **Acronym:** HCT **Brief Title:** HCT Effect Among Patients With Psychotic Disorders **Official Title:** Effect of Horticultural Therapy Program on Psychological Wellbeing, Hope, and Social Adjustment Among Patients With Psychotic Disorders #### Organization Study ID Info **ID:** IRB00013620(9/19/2025) #### Organization **Class:** OTHER **Full Name:** Alexandria University ### Status Module #### Completion Date **Date:** 2023-08-30 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-10-11 **Type:** ACTUAL **Last Update Submit Date:** 2023-10-04 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2023-06-30 **Type:** ACTUAL #### Start Date **Date:** 2022-11-01 **Type:** ACTUAL **Status Verified Date:** 2023-10 #### Study First Post Date **Date:** 2023-10-11 **Type:** ACTUAL **Study First Submit Date:** 2023-08-29 **Study First Submit QC Date:** 2023-10-04 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Alexandria University #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** Horticulture therapy has been found to have positive effects on individuals with psychotic disorders, promoting social interaction, reducing social isolation, and improving cognitive function. Effectiveness of horticulture therapy in enhancing well-being, reducing symptoms, and facilitating social adjustment in this population **Detailed Description:** Horticulture therapy involves gardening and plant-related activities as a form of therapeutic intervention, enhance patient to be mindful to current environment and promote relaxation which can reduce symptoms such as anxiety, depression, and social isolation, improve cognitive function, and promote overall well-being .With this respect, there is an urgent need to find a sustainable and eco-friendly solution for this complex issue by returned to nature and utilizing the low cost and current resources at the same time to make a signification reduction in the psychotic disorder's symptoms ### Conditions Module **Conditions:** - Psychotic Disorders **Keywords:** - Hope - Psychological Adjustment - Psychological Wellbeing - Horticulture Therapy ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Count:** 120 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** receive horticulture therapy by seven sessions within two months **Intervention Names:** - Behavioral: Horticultural Therapy Program **Label:** Horticultural Therapy **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** doesn't receive horticulture therapy but receive routine care by hospital within conducting the research **Label:** Routine hospital care **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Horticultural Therapy **Description:** Horticultural Therapy Program Is selected plant therapy **Name:** Horticultural Therapy Program **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** Effect of Horticultural Therapy Program may enhance hope by Herth Hope Index among patients with psychotic disorders as Herth Hope Index score ranged from 12 to 48, with higher score indicated higher hope. **Measure:** Effect of Horticultural Therapy Program on hope level among patients with psychotic disorders **Time Frame:** three months **Description:** Effect of Horticultural Therapy Program may enhance psychological wellbeing by Ryff psychological wellbeing scale among patients with psychotic disorders a Ryff psychological wellbeing scale score ranged from 18 to 126 with Higher scores mean higher levels of psychological well-being. **Measure:** Effect of Horticultural Therapy Program on psychological wellbeing level among patients with psychotic **Time Frame:** three months **Description:** Effect of Horticultural Therapy Program may enhance social adjustment by The Modified Social Adjustment Score ranged from 45 to 225 with Higher scores mean higher levels of social adjustment. **Measure:** Effect of Horticultural Therapy Program on social adjustment level among patients with psychotic **Time Frame:** three month ### Eligibility Module **Eligibility Criteria:** Inclusion and exclusionCriteria: did not undergo any other intervention during the HT program used in the study, just routine care in hospital - **Healthy Volunteers:** True **Maximum Age:** 60 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Alexandria **Country:** Egypt **Facility:** Faculty of Nursing ### IPD Sharing Statement Module **IPD Sharing:** NO ## Document Section ### Large Document Module #### Large Docs - Date: 2023-10-03 - Filename: Prot_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: False - Label: Study Protocol - Size: 773518 - Type Abbrev: Prot - Upload Date: 2023-10-03T08:40 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000019967 - Term: Schizophrenia Spectrum and Other Psychotic Disorders ### Condition Browse Module - Browse Branches - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M4815 - Name: Mental Disorders - Relevance: HIGH - As Found: Psychotic Disorders - ID: M14473 - Name: Psychotic Disorders - Relevance: HIGH - As Found: Psychotic Disorders - ID: M15376 - Name: Schizophrenia - Relevance: LOW - As Found: Unknown - ID: M21838 - Name: Schizophrenia Spectrum and Other Psychotic Disorders - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001523 - Term: Mental Disorders - ID: D000011618 - Term: Psychotic Disorders ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00638079 **Brief Title:** Evaluating the Effect of Food on Absorption of Megace ES **Official Title:** Single-center, Randomized, Open-label, 2-way Crossover Bioavailability Study, Evaluating the Effect of Food on Megace ES (Megestrol Acetate 625 mg/5 mL Oral Suspension) Following a 625 mg Dose in Healthy Subjects #### Organization Study ID Info **ID:** PAR 100.1.C.003 #### Organization **Class:** INDUSTRY **Full Name:** Endo Pharmaceuticals ### Status Module #### Completion Date **Date:** 2006-07 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2016-04-19 **Type:** ESTIMATED **Last Update Submit Date:** 2016-04-18 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2006-07 **Type:** ACTUAL #### Start Date **Date:** 2006-06 **Status Verified Date:** 2015-08 #### Study First Post Date **Date:** 2008-03-18 **Type:** ESTIMATED **Study First Submit Date:** 2008-03-11 **Study First Submit QC Date:** 2008-03-11 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** SFBC Anapharm #### Lead Sponsor **Class:** INDUSTRY **Name:** Endo Pharmaceuticals #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** To evaluate the effect of food on the rate and extent of absorption of megestrol acetate 625 mg/5 mL , and determine the safety and tolerability of megestrol acetate 625 mg/5 mL in healthy individuals. ### Conditions Module **Conditions:** - Pharmacokinetics - Bioavailability - Absorption **Keywords:** - Pharmacokinetics - Food Effect - Bioavailability - Megace ES ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER ##### Masking Info **Masking:** NONE #### Enrollment Info **Count:** 24 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Megestrol acetate 625 mg/5 mL oral suspension (Megace ES) with a high fat meal **Intervention Names:** - Drug: Megestrol acetate oral suspension 625 mg/5 mL **Label:** A **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Megestrol acetate 625 mg/5 mL oral suspension (Megace ES) following an overnight fast **Intervention Names:** - Drug: Megestrol acetate oral suspension 625 mg/5 mL **Label:** B **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - A **Description:** Megestrol acetate oral suspension 625 mg/5 mL. Single dose (5 mL) administered with a high fat meal **Name:** Megestrol acetate oral suspension 625 mg/5 mL **Other Names:** - Megace ES **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - B **Description:** Megestrol acetate oral suspension 625 mg/5 mL. Single dose (5 mL) administered following an overnight fast **Name:** Megestrol acetate oral suspension 625 mg/5 mL **Other Names:** - Megace ES **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Rate and extent of absorption **Time Frame:** 120 hours (5 days) **Measure:** Safety assessed using adverse events (AEs), clinical laboratory results, vital signs, physical examinations, and ECGs. **Time Frame:** signing of informed consent to 30 days after last study visit ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Body weight ranging from 60-100 kg (132-220 lbs) and body mass index ≥18 and ≤32 * Healthy Exclusion Criteria: * History of or any current medical conditions that could interfere with drug consumption, absorption, distribution, metabolism (eg. CYP450 inducers or inhibitors), or excretion of study drug * History of or any current medical conditions that could affect subject safety * History of frequent nausea or emesis, regardless of etiology * Participation in a clinical drug study during the 30 days preceding the initial dose * Significant illness during the 4 weeks preceding study entry * Use of any medication, including vitamins/herbal/mineral supplements, during the 7 days preceding the initial dose * Refusal or inability to abstain from food 10 hours proceeding and 4 hours following study drug administration, to consume the FDA high fat meal as directed, and to abstain from caffeine- or xanthine-containing beverages entirely during each confinement * Any history of or current drug or alcohol abuse * Prior alcohol intake exceeding the equivalent of 14 units/week (12 oz beer = 4 oz wine = 1.5 oz shot = 1 unit) on average, or consumption of any alcoholic beverages within 48 hours of study drug administration * History of smoking\>25 cigarettes/day within 45 days of study drug administration * Blood or blood products donated within 30 days prior to study drug administration, or anytime during the study, except as required by this protocol * Positive results of urine drug screen, blood alcohol by a Breathalyzer test, hepatitis B surface antigen, hepatitis B surface antibody (unless immunized), or anti-HCV **Healthy Volunteers:** True **Maximum Age:** 55 Years **Minimum Age:** 18 Years **Sex:** MALE **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Montreal (Quebec) **Country:** Canada **Facility:** SFBC Anapharm **Zip:** H3X 2H9 #### Overall Officials **Official 1:** **Affiliation:** SFBC Anapharm **Name:** Benoit Deschamps, MD **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** Endo Pharmaceuticals **Name:** Todd Kirby, PhD **Role:** STUDY_DIRECTOR ### References Module #### References **Citation:** Deschamps B, Musaji N, Gillespie JA. Food effect on the bioavailability of two distinct formulations of megestrol acetate oral suspension. Int J Nanomedicine. 2009;4:185-92. doi: 10.2147/ijn.s6308. Epub 2009 Sep 10. **PMID:** 19774117 #### See Also Links **Label:** International Journal of Nanomedicine **URL:** http://www.dovepress.com/international-journal-of-nanomedicine-journal ## Derived Section ### Intervention Browse Module - Ancestors - ID: D000003278 - Term: Contraceptives, Oral, Hormonal - ID: D000003276 - Term: Contraceptives, Oral - ID: D000003271 - Term: Contraceptive Agents, Female - ID: D000003270 - Term: Contraceptive Agents - ID: D000012102 - Term: Reproductive Control Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000080066 - Term: Contraceptive Agents, Hormonal - ID: D000003280 - Term: Contraceptives, Oral, Synthetic - ID: D000018931 - Term: Antineoplastic Agents, Hormonal - ID: D000000970 - Term: Antineoplastic Agents - ID: D000019167 - Term: Appetite Stimulants - ID: D000000697 - Term: Central Nervous System Stimulants ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: Repr - Name: Reproductive Control Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: CNSSti - Name: Central Nervous System Stimulants ### Intervention Browse Module - Browse Leaves - ID: M11518 - Name: Megestrol - Relevance: HIGH - As Found: January - ID: M21272 - Name: Megestrol Acetate - Relevance: HIGH - As Found: Caucasian - ID: M6494 - Name: Contraceptive Agents - Relevance: LOW - As Found: Unknown - ID: M6500 - Name: Contraceptives, Oral - Relevance: LOW - As Found: Unknown - ID: M6502 - Name: Contraceptives, Oral, Hormonal - Relevance: LOW - As Found: Unknown - ID: M6495 - Name: Contraceptive Agents, Female - Relevance: LOW - As Found: Unknown - ID: M2116 - Name: Contraceptive Agents, Hormonal - Relevance: LOW - As Found: Unknown - ID: M20966 - Name: Antineoplastic Agents, Hormonal - Relevance: LOW - As Found: Unknown - ID: M4029 - Name: Central Nervous System Stimulants - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000008535 - Term: Megestrol - ID: D000019290 - Term: Megestrol Acetate ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04328779 **Brief Title:** Multi-task Gait Training Mode to Enhance Walking Function in Patients With Chronic Stroke **Official Title:** Multi-task Gait Training Mode to Enhance Walking Function, Cognitive Performance, Task Coordination, and Transfer to Community Ambulation in Patients With Chronic Stroke #### Organization Study ID Info **ID:** ChangGungU #### Organization **Class:** OTHER **Full Name:** Chang Gung University ### Status Module #### Completion Date **Date:** 2023-10-31 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2024-02-07 **Type:** ACTUAL **Last Update Submit Date:** 2024-02-05 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2023-10-31 **Type:** ACTUAL #### Start Date **Date:** 2020-08-14 **Type:** ACTUAL **Status Verified Date:** 2024-02 #### Study First Post Date **Date:** 2020-03-31 **Type:** ACTUAL **Study First Submit Date:** 2020-03-30 **Study First Submit QC Date:** 2020-03-30 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Chang Gung University #### Responsible Party **Investigator Affiliation:** Chang Gung University **Investigator Full Name:** Li-Ling Chuang **Investigator Title:** Associated Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The objective of this study is to investigate psychometric properties of dual-task walking assessments and compare effects of multi-task walking interventions on walking for patients with chronic stroke. Specifically, we will investigate psychometric properties (i.e. reliability, validity, and responsiveness) of dual-task walking assessments with the Stroop task for chronic stroke individuals (Aim 1). The second aim of this study is to compare the effects of multi-task walking training mode to traditional rehabilitation in patients with chronic stroke (Aim 2). The third aim of this study is to compare the immediate, retained, and transfer effect of multi-task overground walking training to multi-task treadmill walking training on walking function, cognitive performance, task coordination, and community ambulation in patients with chronic stroke (Aim 3). **Detailed Description:** Only 7% patients can walk safely in the community. Functional community ambulation requires the ability of walking while performing other tasks. It is emergent to establish dual-task walking assessment with good psychometric properties and to identify the most effective approach that enhances dual-task walking performance for stroke patients. Our ongoing study found that motor tasks combined with cognitive dual-task training (multi-tasking ) have more improvements on cognitive performance of the Stroop task while walking than cognitive dual-task training alone. Therefore, combined motor and cognitive elements to walking training might be considered as a novel therapeutic strategy to promote functional ambulation and recovery of stroke patients. This combined strategy is based on attentional capacity sharing theory and the research advances on dual-task to enhance active participation and cognitive involvement, strengthen dual-task walking ability, then transfer to promote community ambulation and participation. However, lacking prospective, controlled trials quantify gait and cognition under dual-task conditions after multi-task walking training. Specifically, we will investigate psychometric properties of dual-task walking assessments for chronic stroke individuals. The second aim is to compare the immediate, retained, and transfer effects of of multi-task overground walking training to multi-task treadmill walking and traditional rehabilitation on walking function, cognitive performance, task coordination, and community ambulation in patients with chronic stroke. A metric analysis and comparative efficacy research will be conducted. Sixty chronic stroke patients will receive dual-task walking assessments twice at pretreatment with a 1-week interval for test-retest assessment and investigation of the reliability and validity of outcome measures. The primary outcome measure of the dual-task walking assessments will include walking at preferred speed and fast speed and simultaneously perform the Stroop task. Concurrent validity will be studied to validate the dual-task walking measures with each other and with the item 14 of the mini-Balance Evaluation Systems test (Mini-BESTest), dual-task Timed-up-and-Go test (dual-TUG), and 6-min walk test obtain concurrently for assessing dual-task ability. In addition, we will compare dual-task walking performance between fallers and non-fallers to examine discriminant validity of dual-task walking assessments. A comparative efficacy research is a single-blind, randomized controlled trial, which will be conducted at medical centers. Sixty ambulatory stroke patients will be randomized to multi-task overground walking training or multi-task treadmill walking training or traditional rehabilitation. All three groups will receive interventions 3 times a week for 4 weeks. The multi-task overground walking training group will undertake overground walking training while concurrently perform motor and cognitive tasks. The multi-task treadmill walking training group will train the same set of motor and cognitive tasks while walking on the treadmill. Traditional rehabilitation will train strength, balance, and gait. A blinded assessor will administer three assessments All participants will be examined gait and cognitive performance under single-task (walking only, cognitive tasks only) and dual-task conditions (walking while performing the Stroop task) at baseline, post intervention, and 1-month follow-up. The primary outcome measure of gait and cognition is gait speed and composite score of accuracy and reaction time of the cognitive tasks under single- and dual-task conditions. The secondary outcome measures will be the Mini-BESTest, single- and dual-TUG, Functional Gait Assessment, 6-minute Walk Test, physical activity monitor, and Stroke Impact Scale. Repeated measure ANOVA will be used to compare measurements at baseline, after training, and follow-up among the groups. ### Conditions Module **Conditions:** - Stroke **Keywords:** - multi-task training - community ambulation - cognition ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 68 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** The multi-task overground walking training group will undertake overground walking training while concurrently perform motor and cognitive tasks. **Intervention Names:** - Other: The multi-task overground walking training **Label:** multi-task overground walking training **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** The multi-task treadmill walking training group will train the same set of motor and cognitive tasks while walking on the treadmill. **Intervention Names:** - Device: The multi-task treadmill walking training **Label:** multi-task treadmill walking **Type:** ACTIVE_COMPARATOR #### Arm Group 3 **Description:** Traditional rehabilitation group will train strength, balance, and gait. **Intervention Names:** - Other: Traditional rehabilitation **Label:** traditional rehabilitation **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - multi-task overground walking training **Description:** The multi-task overground walking training group will undertake overground walking training while concurrently perform motor and cognitive tasks for 30 minutes per session, 3 times a week for 4 weeks. **Name:** The multi-task overground walking training **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - multi-task treadmill walking **Description:** The multi-task treadmill walking training group will train the same set of motor and cognitive tasks while walking on the treadmill for 30 minutes per session, 3 times a week for 4 weeks. **Name:** The multi-task treadmill walking training **Type:** DEVICE #### Intervention 3 **Arm Group Labels:** - traditional rehabilitation **Description:** Traditional rehabilitation will train strength, balance, and gait for 30 minutes per session, 3 times a week for 4 weeks. **Name:** Traditional rehabilitation **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Participants will walk 10m at their preferred speed and at fast speed. A 12-meter walkway will be used for walking testing. In order to allow the subjects to have enough distance to accelerate and decelerate, only the time taken to walk the middle 10 meters will be recorded by a stopwatch or Physilog® sensors (Gait Up, Switzerland). The primary gait parameter is gait speed (cm/s) under single-task and dual-task walking conditions using the 10 Meter Walking Test. **Measure:** gait speed **Time Frame:** 5 minutes **Description:** For the Stroop task, we will calculate the composite score for the Stroop task under single-task and dual-task walking conditions by dividing the accuracy (% correct responses) with the reaction time of correct answers (milliseconds), which accounts for speed-accuracy tradeoffs. **Measure:** composite score **Time Frame:** 5 minutes #### Secondary Outcomes **Description:** The Mini-BESTest consists of 14 items and.includes four subscales: anticipatory postural adjustments, reactive postural control, sensory orientation, and dynamic gait. Each item is rated on a three-point ordinal scale (0 = severe, 1=moderate, and 2 = normal), with a maximum score of 28 points. **Measure:** Mini-Balance Evaluation Systems Test (Mini-BESTest) **Time Frame:** 10 minutes **Description:** The TUG test will be used as an index of dynamic balance of the elderly and stroke patients. At the signal, participants stand up, walk 3 m, turn, walk back, and sit down again. The score is the time to complete the test measured using a stopwatch. The TUG test will be administered under the single-task (preferred speed and maximum fast) and dual-task conditions (tray carrying and counting backward by 3s). In dual-task condition, participants will be asked to perform the TUG test while carrying a tray with glasses (dual-TUG manual) or counting backward by 3s (dual-TUG cognition). The instruction for dual-TUG tests is to walk with your comfortable speed and concurrently perform a secondary task (carry the tray in front of you with both hands without dropping glasses on the tray or counting backward by 3s). **Measure:** Timed Up and Go Test (TUG) **Time Frame:** 2 minutes **Description:** The FGA is comprised of 10-item that contains 7 of 8 items (except walking around obstacles) from the Dynamic Gait Index and 3 additional tasks, including walking with a narrow base of support, walking with the eyes closed, and walking backward. Subjects' performance of each test item was rated on a 4-point scale (0-3), with the total score ranging between 0 and 30. **Measure:** Functional Gait Assessment (FGA) **Time Frame:** 10 minutes **Description:** The BBS is a 14-item scale quantitatively assesses both static and dynamic balance with psychometrically sound measure of balance impairment after stroke. The items are scored from 0 to 4, with a score of 0 representing independent item completion. Scores of the BBS range from 0 to 56, with higher scores suggest better balance. **Measure:** Berg Balance Scale (BBS) **Time Frame:** 5-10 minutes **Description:** Participants walk for 6-minute at comfortable speed to examine their walking endurance. **Measure:** 6-minute Walk Test **Time Frame:** 6 minutes **Description:** Physical activity monitor will be examined by StepWatch TM Activity Monitor (Modus Health, Washington, DC) to objectively measure total walking step numbers of continually three days (except for sleep and bath). **Measure:** Physical activity monitor **Time Frame:** 3 days **Description:** Health-related quality of life will be measured using the SIS 3.0, which is specific to the stroke population. The SIS 3.0 contains 59 items measuring 8 domains (i.e., strength, hand function, Activities of Daily Living/Instrumental Activities of Daily Living \[ADL/IADL\], mobility, communication, emotion. memory and thinking and participation) with a single item assessing perceived overall recovery from stroke. Items are rated on a 5-point Likert scale with lower scores indicating greater difficulty in task completion during the past week. Aggregate scores, ranges from 0 to 100, are generated for each domain. **Measure:** Stroke Impact Scale Version 3.0 (SIS 3.0) **Time Frame:** 10 minutes **Description:** Participants will be evaluated regarding the participants' perception with the change in walking related to the intervention. The PGIC is a transition scale that is a single question asking the patients to rate their walking function now, as compared with how it was prior to before beginning treatment on a scale from 1 (very much better ) to 7 (very much worse). **Measure:** Patient Global Impression of Change Scale (PGIC) **Time Frame:** 1 minute ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. first-ever stroke with onset duration more than 3 months 2. able to walk 10 m 3. no severe vision, hearing, and language problems. Exclusion Criteria: 1. orthopedic and other neurological disorders that affect walking 2. other treatments that could influence the effects of the interventions (e.g., recent Botulin toxin treatment of the lower extremity) 3. moderate or severe cognitive impairments (score \< 24 on Mini-Mental State Examination) 4. severe uncorrected visual deficits. **Maximum Age:** 85 Years **Minimum Age:** 20 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Taipei **Country:** Taiwan **Facility:** Mackay Memory Hospital #### Overall Officials **Official 1:** **Affiliation:** Chang Gung University **Name:** Li-Ling Chuang, Ph.D. **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## 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:** NCT03351179 **Brief Title:** Predictors and Outcomes of In-hospital HFpEF in AMI Patients **Official Title:** Predictors and Outcomes of Hospitalized Heart Failure With Preserved Ejection Fraction Among Patients With First Acute Myocardial Infarction After Reperfusion Treatments #### Organization Study ID Info **ID:** FirstSoochowU #### Organization **Class:** OTHER **Full Name:** The First Affiliated Hospital of Soochow University ### Status Module #### Completion Date **Date:** 2018-12-30 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** ENROLLING_BY_INVITATION #### Last Update Post Date **Date:** 2019-01-02 **Type:** ACTUAL **Last Update Submit Date:** 2018-12-30 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2018-12-30 **Type:** ESTIMATED #### Start Date **Date:** 2013-01-01 **Type:** ACTUAL **Status Verified Date:** 2018-12 #### Study First Post Date **Date:** 2017-11-22 **Type:** ACTUAL **Study First Submit Date:** 2017-11-04 **Study First Submit QC Date:** 2017-11-18 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Xiangjun Yang #### Responsible Party **Investigator Affiliation:** The First Affiliated Hospital of Soochow University **Investigator Full Name:** Xiangjun Yang **Investigator Title:** Director, department of cardiology **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** This retrospective observation is to investigate the incidence,clinical outcomes and prognosis of hospitalized heart failure with preserved ejection fraction (HFpEF) in patients with acute myocardial infarction(AMI). **Detailed Description:** 1. Patients with first AMI undergoing primary percutaneous coronary intervention (PCI) in our hospital since January 2013 were retrospectively evaluated. The enrolled subjects were divided into two groups (AMI patients with HFpEF and AMI patients without HF). 2. The investigator collected the clinical data of participants' demographics (age, sex), previous history,risk factors for AMI (hypertension, hyperlipidemia, diabetes mellitus, smoking and stroke), laboratory biomarkers, echocardiographic measurements, clinical characteristics,medical procedures and treatments, as well as in-hospital complications. 3. Two sub-investigators independently analyzed these data by using SPSS software. ### Conditions Module **Conditions:** - Acute Myocardial Infarction - Heart Failure With Preserved Ejection Fraction - Predictor - Prognosis **Keywords:** - Heart Failure with Preserved Ejection Fraction - Acute MyocAcute Myocardial Infarction - Predictive factors ### Design Module #### Design Info **Observational Model:** CASE_CONTROL **Time Perspective:** RETROSPECTIVE #### Enrollment Info **Count:** 500 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Combination Product: clinical characteristics, previous history, laboratory bio-markers, echocardiographic measurements, angiographic findings,clinical outcomes **Label:** AMI patients with HFpEF #### Arm Group 2 **Intervention Names:** - Combination Product: clinical characteristics, previous history, laboratory bio-markers, echocardiographic measurements, angiographic findings,clinical outcomes **Label:** AMI patients without HF ### Interventions #### Intervention 1 **Arm Group Labels:** - AMI patients with HFpEF - AMI patients without HF **Description:** The differences of data(clinical characteristics, history, laboratory biomarkers, echocardiographic measurements, angiographic characteristics and clinical outcomes) in two groups were conducted. The differences between two groups were compared (clinical characteristics, history, laboratory biomarkers, echocardiographic measurements, angiographic characteristics and clinical outcomes) in two groups were compared, and then found the risk factors. Following,the incidence of clinical outcomes and mortality were compared. Then, univariate logistic regression and multivariate logistic regression analysis adjusted for significant risk factors were performed to find out the independent predictive factors. Finally, the ROC was constructed, and the area was evaluated to assess the predicted probability of regression model. **Name:** clinical characteristics, previous history, laboratory bio-markers, echocardiographic measurements, angiographic findings,clinical outcomes **Type:** COMBINATION_PRODUCT ### Outcomes Module #### Primary Outcomes **Measure:** Risk factors of incident in-hospital HFpEF **Time Frame:** Outcome measure will be assessed at discharge, and data will be reported through study completion, an average of 1 year. #### Secondary Outcomes **Measure:** Clinical prognosis in patients with HFpEF **Time Frame:** Outcome measure will be assessed at discharge, and data will be reported through study completion, an average of 1 year. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * First AMI patients undergoing PCI with HFpEF or without HF. Exclusion Criteria: * First AMI patients with heart failure with reduced ejection fraction. * First AMI patients with severe inflammatory diseases, valvular heart disease, non-cardiac caused symptoms, serious hepatic and renal failure, congenital cardiomyopathy,or pericardial diseases. **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT **Study Population:** AMI patients with HFpEF: First AMI subjects undergoing PCI with a diagnosis of HFpEF. AMI patients without HF: First AMI subjects undergoing PCI without a diagnosis of HF. ### Contacts Locations Module #### Locations **Location 1:** **City:** Suzhou **Country:** China **Facility:** the First Affiliated Hospital of Soochow University **State:** Jiangsu **Zip:** 215006 #### Overall Officials **Official 1:** **Affiliation:** First Affiliated Hospital of Soochow University **Name:** Xiangjun Yang, PhD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ## Derived Section ### Condition Browse Module - Ancestors - ID: D000006331 - Term: Heart Diseases - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000007511 - Term: Ischemia - ID: D000010335 - Term: Pathologic Processes - ID: D000009336 - Term: Necrosis - ID: D000017202 - Term: Myocardial Ischemia - 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 - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M9421 - Name: Heart Failure - Relevance: HIGH - As Found: Heart Failure - ID: M12155 - Name: Myocardial Infarction - Relevance: HIGH - As Found: Myocardial Infarction - ID: M10282 - Name: Infarction - Relevance: HIGH - As Found: Infarction - ID: M9419 - Name: Heart Diseases - Relevance: LOW - As Found: Unknown - 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: 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: D000006333 - Term: Heart Failure - ID: D000009203 - Term: Myocardial Infarction - ID: D000007238 - Term: Infarction ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01795079 **Brief Title:** Effects of Transcranial Direct Current Stimulation (tDCS) on Neuropathic Symptoms Following Burn Injury **Official Title:** Boston-Harvard Burn Injury Model System: Cortical Modulation With Transcranial Direct Current Stimulation (tDCS) for Neuropathic Symptoms Following Burn Injury #### Organization Study ID Info **ID:** 2012-p-001996 #### Organization **Class:** OTHER **Full Name:** Spaulding Rehabilitation Hospital ### Status Module #### Completion Date **Date:** 2020-04-15 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-03-17 **Type:** ACTUAL **Last Update Submit Date:** 2021-02-23 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2019-07 **Type:** ACTUAL #### Results First Post Date **Date:** 2021-03-17 **Type:** ACTUAL **Results First Submit Date:** 2020-12-02 **Results First Submit QC Date:** 2021-02-23 #### Start Date **Date:** 2013-01 **Status Verified Date:** 2021-02 #### Study First Post Date **Date:** 2013-02-20 **Type:** ESTIMATED **Study First Submit Date:** 2013-02-15 **Study First Submit QC Date:** 2013-02-15 ### Sponsor Collaborators Module #### Collaborators **Class:** FED **Name:** U.S. Department of Education #### Lead Sponsor **Class:** OTHER **Name:** Spaulding Rehabilitation Hospital #### Responsible Party **Investigator Affiliation:** Spaulding Rehabilitation Hospital **Investigator Full Name:** Felipe Fregni **Investigator Title:** Associate Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of this study is to see the effects of transcranial direct current stimulation (tDCS) on the pain and itching associated with burn injury. This study is part of the Boston-Harvard Burn Model System. The investigators hypothesize that there will be a decrease in pain levels with active stimulation, when compared to sham stimulation, using a 3 week stimulation schedule- 2 weeks of stimulation (10 consecutive days) followed by 1 week of stimulation (5 consecutive days) after three follow up visits at 2, 4 and 8 weeks after initial course of stimulation. The subject will also have follow ups at 2, 4 and 8 weeks after the second course of stimulation. If a subject receives sham during the experiment, he/she may enroll in an open-label portion of the study and receive 10 days of active stimulation. ### Conditions Module **Conditions:** - Burn Injury - Chronic Pain - Pruritus - Itching **Keywords:** - transcranial stimulation - direct current ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 34 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Subjects will undergo 20 minutes active tDCS. **Intervention Names:** - Device: Transcranial direct current stimulation (tDCS) **Label:** Active tDCS **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Subjects will undergo 20 minutes of sham stimulation. **Intervention Names:** - Device: Transcranial direct current stimulation (tDCS) **Label:** Sham tDCS **Type:** SHAM_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Active tDCS - Sham tDCS **Description:** Subjects will undergo 15 sessions of tDCS stimulation (either active or sham), 1x per day at 20 minutes per session. **Name:** Transcranial direct current stimulation (tDCS) **Other Names:** - 1x1 low-intensity direct current stimulator - Soterix Medical **Type:** DEVICE ### Outcomes Module #### Other Outcomes **Description:** Determine whether anodal transcranial direct current stimulation is effective in increasing quality of life in subjects with neuropathic pain and itching due to burn injury, as measured by changes in the Veterans RAND 36 Item Health Survey (VR-36). There are eight domains total including: bodily pain, role limitations due to physical problems, physical functioning, general health perception, vitality, social functioning, and role limitations due to mental health issues. Scores for each domain are from 0-100 with a higher score defining a more favorable health outcome. **Measure:** Change in Quality of Life Scale **Time Frame:** 2 weeks **Description:** Determine whether anodal transcranial direct current stimulation is effective in increasing community integration functional outcomes in subjects with neuropathic pain and itching due to burn injury, as measured by changes in the Community Integration Questionnaire (CIQ). Total CIQ scores were used as the outcome measure. Contains 15 itms assessing community integrations across three domains (Home integration, social integration, productive activity) . The total score can range from 0 to 29 points (0 - minimal integration to 29 -maximal integration). A positive change indicates an improvement in integration. **Measure:** Change in Community Integration Scale **Time Frame:** 2 weeks #### Primary Outcomes **Description:** Determine whether anodal transcranial direct current stimulation is effective in reducing pain in subjects with neuropathic pain due to burn injury, as measured by changes in the Brief Pain Inventory (BPI). Brief Pain Inventory (BPI) consist consists of a 9 part questionnaire. The questions include the severity of pain levels (worst, least, average, \& current), the impact of pain on daily functioning in different areas (mood, walking, relationships, sleep, normal work, \& general activity), current treatments and perceived effectiveness of current treatments. The VAS Pain scale is a simple 10- point scale (0 = ''no pain'', 10 = ''pain as bad as you can imagine'') measuring patients' worst pain and least pain, on average and at present time. **Measure:** Change in Pain Scale **Time Frame:** 2 weeks **Description:** Determine whether anodal transcranial direct current stimulation is effective in reducing itch severity/activity in subjects with neuropathic itching due to burn injury, as measured by changes in the Visual Analog Scale (VAS) .This is a 0 to 10 scale, where 0 indicates no intensity and a 10 indicates unbearable intensity of itching. **Measure:** Change in Itch Severity/Activity Scale **Time Frame:** 2 weeks #### Secondary Outcomes **Description:** Determine whether anodal transcranial direct current stimulation is effective in decreasing severity of depression in subjects with neuropathic pain and itching due to burn injury, as measured by changes in the Beck Depression Inventory (BDI). The Beck Depression Inventory (BDI) contains 21 questions, each answer being scored on a scale value of 0 to 3 (total from 0-63). Higher total scores indicate more severe depressive symptoms. **Measure:** Change in Depression Scale **Time Frame:** 2 weeks **Description:** Determine whether anodal transcranial direct current stimulation is effective in decreasing post-traumatic stress symptoms in subjects with neuropathic pain and itching due to burn injury, as measured by changes in the Impact of Event Scale Revised (IES-R).This 22-item scale is designed to measure severity of PTS symptoms associated with a traumatic event. Subjects rate their level of distress associated with the event on a 0-4 scale (0 means not at all distressed, 4 means extremely distressed). The IES-R yields a total score (ranging from 0 to 88) where higher scores represent higher stress **Measure:** Change in Post-Traumatic Stress Symptoms Scale **Time Frame:** 2 weeks **Description:** Determine whether anodal transcranial direct current stimulation is effective in decreasing severity of anxiety in subjects with neuropathic pain and itching due to burn injury, as measured by changes in the Visual Analog Scale (VAS).This is a self-evaluation scale that ranges from 0 to 10, where 0 means no anxiety and 10 means the worst anxiety ever. **Measure:** Change in Anxiety Scale **Time Frame:** 2 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Providing informed consent to participate in the study * Age 18 or older * Burn injury with pain and/or itch that is moderate to severe * Burn injury occurring at least 3 weeks prior to enrollment Exclusion Criteria: * Subjects with burns in scalp in the area of electrode placement * Psychiatric disorders that have led to hospitalization within the past 6 months or signs of suicidality * Learning disorders that may prevent patient's ability to complete assessments * Unstable conditions preventing travel to study site * Current use of any of the following anti-epileptic medications or dopaminergic medications known to reduce or inhibit the benefits of tDCS treatment: carbamazepine, oxcarbazepine, phenytoin * Contraindications to tDCS including implanted metal plates in the head or implanted brain medical devices * Pregnancy at time of enrollment * History of other neurological conditions associated with structural anatomical changes (i.e. stroke, brain injury, Parkinson's) **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Boston **Country:** United States **Facility:** Spaulding Rehabilitation Hospital **State:** Massachusetts **Zip:** 02129 #### Overall Officials **Official 1:** **Affiliation:** Spaulding Rehabilitation Hospital **Name:** Felipe Fregni, MD PhD MPH **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### See Also Links **Label:** Boston-Harvard Burn Injury Model System Website **URL:** http://www.bh-bims.org ## Document Section ### Large Document Module #### Large Docs - Date: 2016-03-21 - Filename: Prot_SAP_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: True - Label: Study Protocol and Statistical Analysis Plan - Size: 1036371 - Type Abbrev: Prot_SAP - Upload Date: 2021-02-02T17:04 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000010146 - Term: Pain - ID: D000009461 - Term: Neurologic Manifestations - ID: D000012871 - Term: Skin Diseases - ID: D000012877 - Term: Skin Manifestations ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC26 - Name: Wounds and Injuries - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M29442 - Name: Chronic Pain - Relevance: HIGH - As Found: Chronic Pain - ID: M5326 - Name: Burns - Relevance: HIGH - As Found: Burn Injury - ID: M13066 - Name: Pain - Relevance: LOW - As Found: Unknown - ID: M14396 - Name: Pruritus - Relevance: HIGH - As Found: Pruritus - ID: M17685 - Name: Wounds and Injuries - Relevance: HIGH - As Found: Injury - ID: M12404 - Name: Neurologic Manifestations - Relevance: LOW - As Found: Unknown - ID: M15674 - Name: Skin Diseases - Relevance: LOW - As Found: Unknown - ID: M15680 - Name: Skin Manifestations - Relevance: LOW - As Found: Unknown - ID: T1303 - Name: Chronic Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000011537 - Term: Pruritus - ID: D000059350 - Term: Chronic Pain - ID: D000014947 - Term: Wounds and Injuries - ID: D000002056 - Term: Burns ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Active tDCS **Deaths Num At Risk:** 16 **Description:** Subjects will undergo 20 minutes active tDCS. Transcranial direct current stimulation (tDCS): Subjects will undergo 15 sessions of tDCS stimulation (either active or sham), 1x per day at 20 minutes per session. **ID:** EG000 **Other Num Affected:** 2 **Other Num at Risk:** 16 **Serious Number At Risk:** 16 **Title:** Active tDCS **Group ID:** EG001 **Title:** Sham tDCS **Deaths Num At Risk:** 15 **Description:** Subjects will undergo 20 minutes of sham stimulation. Transcranial direct current stimulation (tDCS): Subjects will undergo 15 sessions of tDCS stimulation (either active or sham), 1x per day at 20 minutes per session. **ID:** EG001 **Other Num Affected:** 5 **Other Num at Risk:** 15 **Serious Number At Risk:** 15 **Title:** Sham tDCS **Frequency Threshold:** 0 #### Other Events **Term:** Hedache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** **Time Frame:** Period of stimulation (3 weeks) ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 16 **Group ID:** BG001 **Value:** 15 **Group ID:** BG002 **Value:** 31 **Units:** Participants ### Group **ID:** BG000 **Title:** Active tDCS **Description:** Subjects will undergo 20 minutes active tDCS. Transcranial direct current stimulation (tDCS): Subjects will undergo 15 sessions of tDCS stimulation (either active or sham), 1x per day at 20 minutes per session. ### Group **ID:** BG001 **Title:** Sham tDCS **Description:** Subjects will undergo 20 minutes of sham stimulation. Transcranial direct current stimulation (tDCS): Subjects will undergo 15 sessions of tDCS stimulation (either active or sham), 1x per day at 20 minutes per session. ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 14 **Value:** 49 #### Measurement **Group ID:** BG001 **Spread:** 14 **Value:** 48 #### Measurement **Group ID:** BG002 **Spread:** 14 **Value:** 48.5 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 9 #### Measurement **Group ID:** BG001 **Value:** 7 #### Measurement **Group ID:** BG002 **Value:** 16 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 7 #### Measurement **Group ID:** BG001 **Value:** 8 #### Measurement **Group ID:** BG002 **Value:** 15 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 2 **Category Title:** Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 15 #### Measurement **Group ID:** BG001 **Value:** 14 #### Measurement **Group ID:** BG002 **Value:** 29 **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:** **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:** Ethnicity (NIH/OMB) **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement ### Point of Contact **Email:** [email protected] **Organization:** Spaulding Rehabilitation Hospital **Phone:** 6179526158 **Title:** Felipe Fregni ## 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:** 1.94 - **Upper Limit:** - **Value:** 4.45 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 1.45 - **Upper Limit:** - **Value:** 4.19 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 2.91 - **Upper Limit:** - **Value:** 3.65 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 1.76 - **Upper Limit:** - **Value:** 1.83 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 7.1 - **Upper Limit:** - **Value:** 9.1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 7.8 - **Upper Limit:** - **Value:** 9.2 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 10.6 - **Upper Limit:** - **Value:** 10.8 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 16.5 - **Upper Limit:** - **Value:** 36.3 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 3.62 - **Upper Limit:** - **Value:** 3.25 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 3.37 - **Upper Limit:** - **Value:** 2.92 **Title:** #### Outcome Measure 6 #### Outcome Measure 7 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Determine whether anodal transcranial direct current stimulation is effective in reducing pain in subjects with neuropathic pain due to burn injury, as measured by changes in the Brief Pain Inventory (BPI). Brief Pain Inventory (BPI) consist consists of a 9 part questionnaire. The questions include the severity of pain levels (worst, least, average, \& current), the impact of pain on daily functioning in different areas (mood, walking, relationships, sleep, normal work, \& general activity), current treatments and perceived effectiveness of current treatments. The VAS Pain scale is a simple 10- point scale (0 = ''no pain'', 10 = ''pain as bad as you can imagine'') measuring patients' worst pain and least pain, on average and at present time. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** blinded assessment of scores at the primary endpoint (2 weeks) **Reporting Status:** POSTED **Time Frame:** 2 weeks **Title:** Change in Pain Scale **Type:** PRIMARY **Unit of Measure:** units on a scale ##### Group **Description:** Subjects will undergo 20 minutes active tDCS. Transcranial direct current stimulation (tDCS): Subjects will undergo 15 sessions of tDCS stimulation (either active or sham), 1x per day at 20 minutes per session. **ID:** OG000 **Title:** Active tDCS ##### Group **Description:** Subjects will undergo 20 minutes of sham stimulation. Transcranial direct current stimulation (tDCS): Subjects will undergo 15 sessions of tDCS stimulation (either active or sham), 1x per day at 20 minutes per session. **ID:** OG001 **Title:** Sham tDCS #### Outcome Measure 2 **Description:** Determine whether anodal transcranial direct current stimulation is effective in reducing itch severity/activity in subjects with neuropathic itching due to burn injury, as measured by changes in the Visual Analog Scale (VAS) .This is a 0 to 10 scale, where 0 indicates no intensity and a 10 indicates unbearable intensity of itching. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** blinded assessment at primary endpoint (2 weeks) **Reporting Status:** POSTED **Time Frame:** 2 weeks **Title:** Change in Itch Severity/Activity Scale **Type:** PRIMARY **Unit of Measure:** units on a scale ##### Group **Description:** Subjects will undergo 20 minutes active tDCS. Transcranial direct current stimulation (tDCS): Subjects will undergo 15 sessions of tDCS stimulation (either active or sham), 1x per day at 20 minutes per session. **ID:** OG000 **Title:** Active tDCS ##### Group **Description:** Subjects will undergo 20 minutes of sham stimulation. Transcranial direct current stimulation (tDCS): Subjects will undergo 15 sessions of tDCS stimulation (either active or sham), 1x per day at 20 minutes per session. **ID:** OG001 **Title:** Sham tDCS #### Outcome Measure 3 **Description:** Determine whether anodal transcranial direct current stimulation is effective in decreasing severity of depression in subjects with neuropathic pain and itching due to burn injury, as measured by changes in the Beck Depression Inventory (BDI). The Beck Depression Inventory (BDI) contains 21 questions, each answer being scored on a scale value of 0 to 3 (total from 0-63). Higher total scores indicate more severe depressive symptoms. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** blinded assessment of scores at the primary endpoint (2 weeks) **Reporting Status:** POSTED **Time Frame:** 2 weeks **Title:** Change in Depression Scale **Type:** SECONDARY **Unit of Measure:** score on a scale ##### Group **Description:** Subjects will undergo 20 minutes active tDCS. Transcranial direct current stimulation (tDCS): Subjects will undergo 15 sessions of tDCS stimulation (either active or sham), 1x per day at 20 minutes per session. **ID:** OG000 **Title:** Active tDCS ##### Group **Description:** Subjects will undergo 20 minutes of sham stimulation. Transcranial direct current stimulation (tDCS): Subjects will undergo 15 sessions of tDCS stimulation (either active or sham), 1x per day at 20 minutes per session. **ID:** OG001 **Title:** Sham tDCS #### Outcome Measure 4 **Description:** Determine whether anodal transcranial direct current stimulation is effective in decreasing post-traumatic stress symptoms in subjects with neuropathic pain and itching due to burn injury, as measured by changes in the Impact of Event Scale Revised (IES-R).This 22-item scale is designed to measure severity of PTS symptoms associated with a traumatic event. Subjects rate their level of distress associated with the event on a 0-4 scale (0 means not at all distressed, 4 means extremely distressed). The IES-R yields a total score (ranging from 0 to 88) where higher scores represent higher stress **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** blinded assessment of scores at the primary endpoint(2 weeks) **Reporting Status:** POSTED **Time Frame:** 2 weeks **Title:** Change in Post-Traumatic Stress Symptoms Scale **Type:** SECONDARY **Unit of Measure:** score on a scale ##### Group **Description:** Subjects will undergo 20 minutes active tDCS. Transcranial direct current stimulation (tDCS): Subjects will undergo 15 sessions of tDCS stimulation (either active or sham), 1x per day at 20 minutes per session. **ID:** OG000 **Title:** Active tDCS ##### Group **Description:** Subjects will undergo 20 minutes of sham stimulation. Transcranial direct current stimulation (tDCS): Subjects will undergo 15 sessions of tDCS stimulation (either active or sham), 1x per day at 20 minutes per session. **ID:** OG001 **Title:** Sham tDCS #### Outcome Measure 5 **Description:** Determine whether anodal transcranial direct current stimulation is effective in decreasing severity of anxiety in subjects with neuropathic pain and itching due to burn injury, as measured by changes in the Visual Analog Scale (VAS).This is a self-evaluation scale that ranges from 0 to 10, where 0 means no anxiety and 10 means the worst anxiety ever. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** blinded assessment at the primary endpoint (2 weeks) **Reporting Status:** POSTED **Time Frame:** 2 weeks **Title:** Change in Anxiety Scale **Type:** SECONDARY **Unit of Measure:** units on a scale ##### Group **Description:** Subjects will undergo 20 minutes active tDCS. Transcranial direct current stimulation (tDCS): Subjects will undergo 15 sessions of tDCS stimulation (either active or sham), 1x per day at 20 minutes per session. **ID:** OG000 **Title:** Active tDCS ##### Group **Description:** Subjects will undergo 20 minutes of sham stimulation. Transcranial direct current stimulation (tDCS): Subjects will undergo 15 sessions of tDCS stimulation (either active or sham), 1x per day at 20 minutes per session. **ID:** OG001 **Title:** Sham tDCS #### Outcome Measure 6 **Description:** Determine whether anodal transcranial direct current stimulation is effective in increasing quality of life in subjects with neuropathic pain and itching due to burn injury, as measured by changes in the Veterans RAND 36 Item Health Survey (VR-36). There are eight domains total including: bodily pain, role limitations due to physical problems, physical functioning, general health perception, vitality, social functioning, and role limitations due to mental health issues. Scores for each domain are from 0-100 with a higher score defining a more favorable health outcome. **Reporting Status:** NOT_POSTED **Time Frame:** 2 weeks **Title:** Change in Quality of Life Scale **Type:** OTHER_PRE_SPECIFIED #### Outcome Measure 7 **Description:** Determine whether anodal transcranial direct current stimulation is effective in increasing community integration functional outcomes in subjects with neuropathic pain and itching due to burn injury, as measured by changes in the Community Integration Questionnaire (CIQ). Total CIQ scores were used as the outcome measure. Contains 15 itms assessing community integrations across three domains (Home integration, social integration, productive activity) . The total score can range from 0 to 29 points (0 - minimal integration to 29 -maximal integration). A positive change indicates an improvement in integration. **Reporting Status:** NOT_POSTED **Time Frame:** 2 weeks **Title:** Change in Community Integration Scale **Type:** OTHER_PRE_SPECIFIED ### Participant Flow Module #### Group **Description:** Subjects will undergo 20 minutes active tDCS. Transcranial direct current stimulation (tDCS): Subjects will undergo 15 sessions of tDCS stimulation (either active or sham), 1x per day at 20 minutes per session. **ID:** FG000 **Title:** Active tDCS #### Group **Description:** Subjects will undergo 20 minutes of sham stimulation. Transcranial direct current stimulation (tDCS): Subjects will undergo 15 sessions of tDCS stimulation (either active or sham), 1x per day at 20 minutes per session. **ID:** FG001 **Title:** Sham tDCS #### Period **Title:** Overall Study ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 16 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 15 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 13 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 12 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 3 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 3 **Pre-Assignment Details:** Please note that 34 subjects consented to participated but 3 were screened out (after consent, there are additional tests to determine eligibility) and/or they dropped out before randomization (therefore, only 31 subjects were randomized). **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT00374179 **Brief Title:** CT-322 in Treating Patients With Advanced Solid Tumors and Non-Hodgkin's Lymphoma **Official Title:** A Phase 1, Escalating Dose Study of CT-322, a VEGFR-2 Antagonist, as Monotherapy in Patients With Advanced Solid Tumors and Non-Hodgkin's Lymphoma #### Organization Study ID Info **ID:** CT-322.001 #### Organization **Class:** INDUSTRY **Full Name:** Adnexus, A Bristol-Myers Squibb R&D Company ### Status Module #### Completion Date **Date:** 2009-02 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2009-02-24 **Type:** ESTIMATED **Last Update Submit Date:** 2009-02-23 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2008-11 **Type:** ACTUAL #### Start Date **Date:** 2006-08 **Status Verified Date:** 2009-02 #### Study First Post Date **Date:** 2006-09-11 **Type:** ESTIMATED **Study First Submit Date:** 2006-09-08 **Study First Submit QC Date:** 2006-09-08 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Adnexus, A Bristol-Myers Squibb R&D Company #### Responsible Party **Old Name Title:** Medical Director, Adnexus, A Bristol-Myers Squibb R&D Company **Old Organization:** Adnexus, A Bristol-Myers Squibb R&D Company ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** RATIONALE: CT-322 may stop the growth of solid tumors or non-Hodgkin's lymphoma by stopping blood flow to the cancer. PURPOSE: This phase I trial is studying the side effects of CT-322 in treating patients with advanced solid tumors or non-Hodgkin's lymphoma. ### Conditions Module **Conditions:** - Cancer ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 40 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Description:** IV solution, weekly or bi-weekly **Name:** CT-322 **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Safety and tolerability of CT-322 **Time Frame:** Throughout the study #### Secondary Outcomes **Measure:** To evaluate the pharmacokinetics of CT-322 in these patients; **Time Frame:** Throughout the study **Measure:** to assess whether antibodies to this drug develop in these patients; and **Time Frame:** Throughout the study **Measure:** to make a preliminary assessment of the biological activity of CT-322 to alter tumor growth. **Time Frame:** Throughout the study ### Eligibility Module **Eligibility Criteria:** DISEASE CHARACTERISTICS * Histologically proven advanced solid malignancy or NHL for which no standard therapy exists or for which standard therapy had failed * No known brain or leptomeningeal disease * No prior bone marrow transplant or stem cell rescue * No histologically confirmed squamous non-small cell lung cancer (NSCLC) with central chest tumor(s) still in place PATIENT CHARACTERISTICS Age: \* 18 and over Performance status: \* ECOG performance status ≤ 2 Life expectancy: \* \> 3 months Hematopoietic: * ANC ≥ 1500/mL * Platelets ≥ 100,000/mL * Hemoglobin ≥ 9.0 g/dL; and not requiring transfusion \> 1 unit/month Hepatic: * AST and ALT ≤ 2.5 x ULN; if liver function abnormalities are due to the underlying malignancy, then AST and ALT may be ≤ 5 x the ULN * Bilirubin ≤ 1.5 x ULN * aPTT and PT \< 1.5 x ULN Renal: * Creatinine ≤ 1.5 x ULN; patients with serum creatinine \> 1 x ULN must also have creatinine clearance (based on a 24-hour urine collection) ≤ 60 mL/min * No proteinuria \> 1+ on dipstick analysis; in the case of \> 1+ dipstick proteinuria, a 24-hour urine collection for protein must be \< 500 mg/24 hours * Urinary protein/creatinine ratio \< 1 * No glomerulonephritis Cardiovascular: * No coronary artery bypass graft, angioplasty, vascular stent, myocardial infarction, unstable angina, symptomatic congestive heart failure, severe uncontrolled hypertension, hemorrhagic or thrombotic stroke or any other CNS bleeding within the preceding 12 months * LVEF normal by echocardiogram or MUGA within the past 12 months if there was prior exposure to anthracyclines or radiotherapy encompassing the heart Immunologic: \* Not known to have human immunodeficiency virus (HIV), active hepatitis virus C (HVC), or active hepatitis virus B (HVB) Other: * Negative pregnancy test within 7 days prior to enrollment * Not pregnant or breast feeding * Fertile patients must agree to use effective contraception or commit to abstinence during the study period, or be surgically sterile * No serious nonhealing wound, ulcer, or bone fracture * Have the ability to understand and sign an informed consent document * Be willing and able to comply with scheduled visits, treatment plan, laboratory tests, and other study procedures PRIOR CONCURRENT THERAPY: Biologic therapy: * See Disease Characteristics * At least 4 weeks since prior biological or immunotherapy and recovered Chemotherapy: * See Disease Characteristics * At least 4 weeks since prior chemotherapy and recovered * At least 6 weeks for mitomycin C and nitrosoureas prior to study entry and recovered Radiotherapy: \* At least 4 weeks since prior radiotherapy to a visceral organ and recovered Surgery: * At least 4 weeks since prior major or laparoscopic surgery and recovered * At least 1 week since prior minor surgery Other: * No other concurrent anticancer therapy * Not concurrently enrolled in another therapeutic clinical trial involving ongoing therapy * No concurrent full dose, therapeutic anti-coagulation with warfarin or related oral anti-coagulants or unfractionated or low molecular weight heparins; low dose warfarin for catheter prophylaxis or acetylsalicylic acid ≤ 325 mg/day is acceptable **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Indianapolis **Country:** United States **Facility:** Indiana University Cancer Center **State:** Indiana **Zip:** 46202 **Location 2:** **City:** San Antonio **Country:** United States **Facility:** South Texas Accelerated Research Therapeutics **State:** Texas **Zip:** 78229 **Location 3:** **City:** San Antonio **Country:** United States **Facility:** Institute for Drug Development **State:** Texas **Zip:** 78245-3217 ### References Module #### References **Citation:** Tolcher AW, Sweeney CJ, Papadopoulos K, Patnaik A, Chiorean EG, Mita AC, Sankhala K, Furfine E, Gokemeijer J, Iacono L, Eaton C, Silver BA, Mita M. Phase I and pharmacokinetic study of CT-322 (BMS-844203), a targeted Adnectin inhibitor of VEGFR-2 based on a domain of human fibronectin. Clin Cancer Res. 2011 Jan 15;17(2):363-71. doi: 10.1158/1078-0432.CCR-10-1411. Epub 2011 Jan 11. **PMID:** 21224368 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000008223 - Term: Lymphoma - 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: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M11220 - Name: Lymphoma - 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: T3543 - Name: Lymphosarcoma - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000008228 - Term: Lymphoma, Non-Hodgkin ### Intervention Browse Module - Browse Branches - Abbrev: PhSol - Name: Pharmaceutical Solutions - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M21860 - Name: Pharmaceutical Solutions - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05341479 **Brief Title:** An Observational Study of Clinical Treatments for Patients With Oropharyngeal Carcinoma **Official Title:** Department of Otorhinolaryngology, Eye & ENT Hospital #### Organization Study ID Info **ID:** FD-EENT-2022036 #### Organization **Class:** OTHER **Full Name:** Eye & ENT Hospital of Fudan University ### Status Module #### Completion Date **Date:** 2025-07-01 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2022-04-22 **Type:** ACTUAL **Last Update Submit Date:** 2022-04-18 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2025-03-31 **Type:** ESTIMATED #### Start Date **Date:** 2022-04-01 **Type:** ACTUAL **Status Verified Date:** 2022-04 #### Study First Post Date **Date:** 2022-04-22 **Type:** ACTUAL **Study First Submit Date:** 2022-04-18 **Study First Submit QC Date:** 2022-04-18 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Eye & ENT Hospital of Fudan University #### Responsible Party **Investigator Affiliation:** Eye & ENT Hospital of Fudan University **Investigator Full Name:** LeiTao **Investigator Title:** Director **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** This is a prospective, observational study evaluating the effect and efficiency of different clinical treatments for patients with oropharyngeal carcinoma (OPC). The selection of treatment for individual patient is based on tumor characteristics (tumor size and tumor location), a complete assessment of therapeutic effects (treatment effectiveness, possible dysfunction after operation, function maintenance, complications, etc.), and the preferences of doctors and patients. **Detailed Description:** For early-stage oropharyngeal carcinoma (T1-2,N0), radiotherapy or resection of the primary tumor (transoral laser microsurgery, transoral robotic surgery, conventional transoral surgery, and open surgery) with ipsilateral or bilateral neck dissection are performed according to the guidelines of Chinese society of clinical oncology (CSCO, version 2021) and the NCCN guidelines (version 2021). Postoperative radiotherapy (RT) or chemoradiotherapy (CRT) should be considered for patients with adverse features (e.g., extranodal extension, positive margins, perineural invasion or vascular invasion). For advanced oropharyngeal carcinoma (T1-2,N1-3/T3-4,N0-3), chemoradiotherapy (CRT), resection of the primary tumor (transoral robotic surgery, conventional transoral surgery, transoral laser microsurgery, and open surgery) with ipsilateral or bilateral neck dissection or neoadjuvant therapy with appropriate therapy (surgical resection, RT or CRT) are performed according to the CSCO guidelines (version 2021) and the NCCN guidelines (version 2021). Postoperative radiotherapy (RT) or chemoradiotherapy (CRT) should be considered for patients with adverse features (e.g., pT3 or pT4 primary, pN2 or pN3 nodal disease, extranodal extension, positive margins, perineural invasion or vascular invasion). This study aims to evaluate and compare the clinical outcomes of different medical treatments in patients with same TNM stage disease, overall and stratified by HPV expression. Demographic and clinicopathological characteristics of patients enrolled were also collected to assess their associations with treatment strategies and prognoses. The rates of overall survival, disease specific survival, disease free survival, local control, regional control, and progress-free survival are analyzed in the present study. ### Conditions Module **Conditions:** - Oropharynx Carcinoma **Keywords:** - Oropharynx carcinoma - Treatment - Survival outcomes ### Design Module #### Bio Spec **Description:** Whole blood, Tumor tissue (If available), Para-tumor tissue (If available) **Retention:** SAMPLES_WITH_DNA #### Design Info **Observational Model:** OTHER **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 200 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Early-stage OPC patients treated with surgery according to proper indications (CSCO 2021 and NCCN 2021). **Intervention Names:** - Procedure: Surgery for early-stage OPC **Label:** Surgical treatment for early-stage OPC #### Arm Group 2 **Description:** Early-stage OPC patients treated with radiotherapy (RT) according to proper indications (CSCO 2021 and NCCN 2021). **Intervention Names:** - Radiation: RT for early-stage OPC **Label:** RT treatment for early-stage OPC #### Arm Group 3 **Description:** Advanced OPC patients treated with chemoradiotherapy (CRT) according to proper indications (CSCO 2021 and NCCN 2021). **Intervention Names:** - Radiation: CRT for advanced OPC **Label:** CRT treatment for advanced OPC #### Arm Group 4 **Description:** Advanced OPC patients treated with surgery according to proper indications (CSCO 2021 and NCCN 2021). **Intervention Names:** - Other: Surgery for advanced OPC **Label:** Surgical treatment for advanced OPC #### Arm Group 5 **Description:** Advanced OPC patients treated with neoadjuvant treatment according to proper indications (CSCO 2021 and NCCN 2021). **Intervention Names:** - Procedure: Neoadjuvant treatment for advanced OPC **Label:** Neoadjuvant treatment for advanced OPC ### Interventions #### Intervention 1 **Arm Group Labels:** - Surgical treatment for early-stage OPC **Description:** The treatment strategy for patients should be discussed by the multidisciplinary team with the goal of maximizing survival with preservation of appearance and function and planned based on the tumor extension as ascertained by clinical evaluation and careful interpretation of appropriate imaging examinations. Early-stage OPC patients (T1-2,N0) enrolled in this group will be treated with surgical resection. The surgical techniques of the primary tumor include transoral robotic surgery, conventional transoral surgery, transoral laser microsurgery (e.g., CO2 laser resection), and open surgery. Ipsilateral neck dissection should be performed for patients enrolled in this group. For patients with OPC at or approaching the midline, bilateral neck dissection should be strongly considered. Postoperative radiotherapy (RT) or system therapy/RT should be considered for patients with adverse features (e.g., extranodal extension, perineural invasion or vascular invasion). **Name:** Surgery for early-stage OPC **Type:** PROCEDURE #### Intervention 2 **Arm Group Labels:** - RT treatment for early-stage OPC **Description:** The treatment strategy for patients should be discussed by the multidisciplinary team with the goal of maximizing survival with preservation of appearance and function and planned based on the tumor extension as ascertained by clinical evaluation and careful interpretation of appropriate imaging examinations. Early-stage OPC patients (T1-2,N0) enrolled in this group will be treated with RT. **Name:** RT for early-stage OPC **Type:** RADIATION #### Intervention 3 **Arm Group Labels:** - CRT treatment for advanced OPC **Description:** The treatment strategy for patients should be discussed by the multidisciplinary team with the goal of maximizing survival with preservation of appearance and function and planned based on the tumor extension as ascertained by clinical evaluation and careful interpretation of appropriate imaging examinations. Advanced OPC patients (T1-2,N1-3/T3-4,N0-3) enrolled in this group will be treated with CRT. **Name:** CRT for advanced OPC **Type:** RADIATION #### Intervention 4 **Arm Group Labels:** - Surgical treatment for advanced OPC **Description:** The treatment strategy for patients should be discussed by the multidisciplinary team with the goal of maximizing survival with preservation of appearance and function and planned based on the tumor extension as ascertained by clinical evaluation and careful interpretation of appropriate imaging examinations. Advanced OPC patients (T1-2,N1-3/T3-4,N0-3) enrolled in this group will be treated with surgical resection. The surgical techniques of the primary tumor include transoral robotic surgery, conventional transoral surgery, transoral laser microsurgery, and open surgery. Ipsilateral neck dissection should be performed for patients enrolled in this group. For patients with OPC at or approaching the midline, bilateral neck dissection should be strongly considered. Postoperative radiotherapy (RT) or system therapy/RT should be considered for patients with adverse features (e.g., pT3 or pT4 primary, pN2 or pN3 nodal disease, extranodal extension, or vascular invasion). **Name:** Surgery for advanced OPC **Type:** OTHER #### Intervention 5 **Arm Group Labels:** - Neoadjuvant treatment for advanced OPC **Description:** The treatment strategy for patients should be discussed by the multidisciplinary team with the goal of maximizing survival with preservation of appearance and function and planned based on the tumor extension as ascertained by clinical evaluation and careful interpretation of appropriate imaging examinations. Advanced OPC patients (T1-2,N1-3/T3-4,N0-3) enrolled in this group will be treated with neoadjuvant treatment. The subsequent treatments include RT, system therapy/RT, and surgical resection with or without postoperative adjuvant therapy. **Name:** Neoadjuvant treatment for advanced OPC **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Description:** The time between the treatment and the date of death from any causes **Measure:** 3-year overall survival **Time Frame:** Three years #### Secondary Outcomes **Description:** The time between the treatment and the first evidence of local recurrence **Measure:** 3-year local control survival **Time Frame:** Three years **Description:** The time between the treatment and the first evidence of regional recurrence **Measure:** 3-year regional control **Time Frame:** Three years **Description:** The time between the treatment and the date of death from oropharyngeal cancer **Measure:** 3-year disease specific survival **Time Frame:** Three years **Description:** The time between the treatment and the first evidence of disease recurrence, metastasis or death from any cause **Measure:** 3-year disease free survival **Time Frame:** Three years **Description:** The time between the treatment and the date of first documented disease progression or death from any cause **Measure:** 3-year progress-free survival **Time Frame:** Three years **Description:** Quality of life as assessed with the MD Anderson Dysphagia Inventory (MDADI) **Measure:** Quality of Life post treatment **Time Frame:** One year and three years post treatment ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Oropharyngeal cancer: Base of Tongue/Tonsil/Posterior Pharyngeal Wall/Soft Palate; * T1, T2, T3, and T4 stage. * Age 18 - 90. * Male or female. * Good compliance. * No other severe related diseases that may impact the treatment (such as other tumors, severe heart, lung and central nervous system diseases, etc.). * Negative pregnancy test (for female patients with fertility). * Male patients with fertility and female patients with fertility and pregnancy risk must agree to use contraceptive methods throughout the study period, and continued until at least 6 months after the last dose of cisplatin. Female patients do not have fertility. Female patients with postmenopausal status. Exclusion Criteria: * Patients who have previously been diagnosed with immunodeficiency or known human immunodeficiency virus (HIV) or acquired immunodeficiency syndrome (AIDS)-related diseases. * Patients with a known history of active tuberculosis (TB). * Pregnant women or lactating women. * The doctors believes that it is inappropriate for patients to participate in the trial: having, for example, severe acute or chronic medical conditions (including immune colitis, inflammatory bowel disease, non-infectious pneumonia, pulmonary fibrosis) or mental illness (including recent time \[within the past year\] or active suicidal ideation or behavior). **Maximum Age:** 90 Years **Minimum Age:** 18 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** This prospective, observational study involves patients with T1-T4 stages oropharyngeal carcinoma, and we aim to evaluate and compare the oncological outcomes of different clinical treatments in patients with same TNM stage disease, overall and stratified by HPV expression. Demographic and clinicopathological characteristics of patients enrolled are also collected to assess their associations with treatment strategies and prognoses. ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Lei Tao, Dr. **Phone:** +86 2164377134 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Xiaoke Zhu **Phone:** +86 18221615406 **Role:** CONTACT #### Locations **Location 1:** **City:** Shanghai **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Lei Tao, Dr. - **Phone:** +86 2164377134 - **Role:** CONTACT **Country:** China **Facility:** Department of Otorhinolaryngology, Eye & ENT Hospital **State:** Shanghai **Status:** RECRUITING **Zip:** 200031 #### Overall Officials **Official 1:** **Affiliation:** Department of Otorhinolaryngology, Eye & ENT Hospital, Fudan University, Shanghai, China **Name:** Lei Tao, Dr. **Role:** STUDY_CHAIR ## Derived Section ### Condition Browse Module - Ancestors - 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: M5534 - Name: Carcinoma - Relevance: HIGH - As Found: 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 ### Condition Browse Module - Meshes - ID: D000002277 - Term: Carcinoma ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03639779 **Brief Title:** Efficacy of Intralesional Sodium Thiosulfate Versus Intralesional Saline for Dystrophic and Idiopathic Calcinosis Cutis **Official Title:** A Comparative Study of the Efficacy of Intralesional Sodium Thiosulfate Versus Intralesional Normal Saline for the Treatment of Dystrophic and Idiopathic Calcinosis Cutis, A Double-Blind Randomized Placebo-Controlled Trial #### Organization Study ID Info **ID:** BIO-18-14095 #### Organization **Class:** OTHER **Full Name:** University of Central Florida ### Status Module #### Completion Date **Date:** 2019-12-31 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-07-01 **Type:** ACTUAL **Last Update Submit Date:** 2021-06-29 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2019-12-31 **Type:** ACTUAL #### Results First Post Date **Date:** 2021-07-01 **Type:** ACTUAL **Results First Submit Date:** 2021-05-15 **Results First Submit QC Date:** 2021-06-29 #### Start Date **Date:** 2018-11-02 **Type:** ACTUAL **Status Verified Date:** 2021-06 #### Study First Post Date **Date:** 2018-08-21 **Type:** ACTUAL **Study First Submit Date:** 2018-08-17 **Study First Submit QC Date:** 2018-08-17 **Why Stopped:** Time and resource constraints ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Central Florida #### Responsible Party **Investigator Affiliation:** University of Central Florida **Investigator Full Name:** David Weinstein **Investigator Title:** Assistant Professor of Dermatology **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Is US Export:** True **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of our research is to compare the effectiveness of 125mg/50ml sodium thiosulfate (STS) solution to normal saline (0.9% sodium chloride) when injected intralesionally for the treatment of calcinosis cutis. Our specific aim is to assess the response of dystrophic and idiopathic calcinosis cutis to the injections of sodium thiosulfate in our patients. **Detailed Description:** Our specific aim is to assess the response of dystrophic and idiopathic calcinosis cutis to the injections of sodium thiosulfate in our patients. ### Conditions Module **Conditions:** - Calcinosis Cutis **Keywords:** - Calcinosis cutis - sodium thiosulfate - sodium chloride ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** This is a parallel two-arm study comparing sodium thiosulfate and saline control. ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - INVESTIGATOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 5 **Type:** ACTUAL **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 50 ml vials of sodium thiosulfate (250mg/ml) will be used for treatment. **Intervention Names:** - Drug: Sodium Thiosulfate **Label:** Sodium thiosulfate **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** 30 ml vials of sodium chloride 0.9% will be used for the control treatment. **Intervention Names:** - Other: Saline Solution **Label:** Saline solution **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Sodium thiosulfate **Description:** A volume of 0.1 ml/cm2 of STS will be injected into each lesion. **Name:** Sodium Thiosulfate **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Saline solution **Description:** A volume of 0.1 ml/cm2 of normal saline will be injected into the control lesion. **Name:** Saline Solution **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** The lesion size is measured in square centimeters. **Measure:** Lesion Size **Time Frame:** 3 months **Description:** The PGA will be done by the physician to assess appearance and changes in the lesion. The lesion will be assigned a score from 0 (clear) to 4 (severe). **Measure:** Physician Global Assessment (PGA) **Time Frame:** 3 months **Description:** The VAS for pain is used to assess the pain associated with the lesion. The pain will be recorded as a number from 1 (no pain) to 10 (severe pain). **Measure:** Visual Analog Scale (VAS) for Pain **Time Frame:** 3 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Male or female adult 18 years of age or older * Must have health insurance will be eligible to participate * Must have a current diagnosis of dystrophic or idiopathic calcinosis cutis * Subjects must have at least 2 lesions of at least 2mm in size Exclusion Criteria: * Unable to read and speak English * Allergy to any component of the sodium thiosulfate solution * Adults unable to consent * Individuals who are not yet adults (infants, children, teenagers) * Pregnant women * Women who are breastfeeding * Prisoners **Healthy Volunteers:** True **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Orlando **Country:** United States **Facility:** UCF Health Lake Nona Office **State:** Florida **Zip:** 32832 ### IPD Sharing Statement Module **IPD Sharing:** NO ## Document Section ### Large Document Module #### Large Docs - Date: 2019-05-15 - Filename: Prot_SAP_002.pdf - Has ICF: False - Has Protocol: True - Has SAP: True - Label: Study Protocol and Statistical Analysis Plan - Size: 301394 - Type Abbrev: Prot_SAP - Upload Date: 2021-06-09T10:45 - Date: 2019-05-21 - Filename: ICF_003.pdf - Has ICF: True - Has Protocol: False - Has SAP: False - Label: Informed Consent Form - Size: 251745 - Type Abbrev: ICF - Upload Date: 2021-06-09T10:46 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000002128 - Term: Calcium Metabolism Disorders - ID: D000008659 - Term: Metabolic Diseases - ID: D000012871 - Term: Skin Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases ### Condition Browse Module - Browse Leaves - ID: M5377 - Name: Calcinosis - Relevance: HIGH - As Found: Calcinosis - ID: M2903 - Name: Calcinosis Cutis - Relevance: HIGH - As Found: Calcinosis Cutis - ID: M11639 - Name: Metabolic Diseases - Relevance: LOW - As Found: Unknown - ID: M5391 - Name: Calcium Metabolism Disorders - Relevance: LOW - As Found: Unknown - ID: M15674 - Name: Skin Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000092182 - Term: Calcinosis Cutis - ID: D000002114 - Term: Calcinosis ### Intervention Browse Module - Ancestors - ID: D000000931 - Term: Antidotes - ID: D000020011 - Term: Protective Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000000975 - Term: Antioxidants - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000000995 - Term: Antitubercular Agents - ID: D000000900 - Term: Anti-Bacterial Agents - ID: D000000890 - Term: Anti-Infective Agents - ID: D000002614 - Term: Chelating Agents - ID: D000064449 - Term: Sequestering Agents ### Intervention Browse Module - Browse Branches - Abbrev: PhSol - Name: Pharmaceutical Solutions - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Infe - Name: Anti-Infective Agents ### Intervention Browse Module - Browse Leaves - ID: M21860 - Name: Pharmaceutical Solutions - Relevance: LOW - As Found: Unknown - ID: M256801 - Name: Sodium thiosulfate - Relevance: HIGH - As Found: C-reactive protein - ID: M4250 - Name: Antidotes - Relevance: LOW - As Found: Unknown - ID: M21869 - Name: Protective Agents - Relevance: LOW - As Found: Unknown - ID: M4292 - Name: Antioxidants - Relevance: LOW - As Found: Unknown - ID: M4311 - Name: Antitubercular 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: M5860 - Name: Chelating Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: C000017717 - Term: Sodium thiosulfate ### Misc Info Module #### Submission Tracking ##### First MCP Info ###### Post Date - Date: 2021-06-10 - Type: ACTUAL - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Sodium Thiosulfate **Deaths Num At Risk:** 5 **Description:** 50 ml vials of sodium thiosulfate (250mg/ml) will be used for treatment. Sodium Thiosulfate: A volume of 0.1 ml/cm2 of STS will be injected into each lesion. **ID:** EG000 **Other Num at Risk:** 5 **Serious Number At Risk:** 5 **Title:** Sodium Thiosulfate **Group ID:** EG001 **Title:** Saline Solution **Deaths Num At Risk:** 5 **Description:** 30 ml vials of sodium chloride 0.9% will be used for the control treatment. Saline Solution: A volume of 0.1 ml/cm2 of normal saline will be injected into the control lesion. **ID:** EG001 **Other Num at Risk:** 5 **Serious Number At Risk:** 5 **Title:** Saline Solution **Frequency Threshold:** 0 **Time Frame:** 3 months ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 5 **Units:** Participants ### Group **ID:** BG000 **Title:** All Participants **Description:** All study participants received an injection of Sodium Thiosulfate into one lesion. They received an injection of saline in the control lesion. ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** <=18 years #### Measurement **Group ID:** BG000 **Value:** 3 **Category Title:** Between 18 and 65 years #### Measurement **Group ID:** BG000 **Value:** 2 **Category Title:** >=65 years **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 4 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 1 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** American Indian or Alaska Native #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Asian #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Native Hawaiian or Other Pacific Islander #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Black or African American #### Measurement **Group ID:** BG000 **Value:** 5 **Category Title:** White #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** More than one race #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Unknown or Not Reported **Class Title:** **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Age, Categorical **Unit of Measure:** Participants ### 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 ## Results Section - More Information Module ### Certain Agreement ### Limitations and Caveats **Description:** The study had to be closed early due to lack up support/resources. ### Point of Contact **Email:** [email protected] **Organization:** University of Central Florida College of Medicine **Phone:** (407)266-4900 **Title:** Dr. David Weinstein ## 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:** 0.1 - **Spread:** - **Upper Limit:** 4 - **Value:** 2.8 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 0.4 - **Spread:** - **Upper Limit:** 4 - **Value:** 1.8 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 5 - **Group ID:** OG001 - **Value:** 5 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0 - **Spread:** - **Upper Limit:** 4 - **Value:** 3 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 0.1 - **Spread:** - **Upper Limit:** 4 - **Value:** 3.0 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 4 - **Group ID:** OG001 - **Value:** 4 **Units:** Participants #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 4 - **Spread:** - **Upper Limit:** 4 - **Value:** 4 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 4 - **Spread:** - **Upper Limit:** 4 - **Value:** 4 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 5 - **Group ID:** OG001 - **Value:** 5 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0 - **Spread:** - **Upper Limit:** 4 - **Value:** 3 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 1 - **Spread:** - **Upper Limit:** 4 - **Value:** 3.25 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 4 - **Group ID:** OG001 - **Value:** 4 **Units:** Participants #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.4 - **Spread:** - **Upper Limit:** 10 - **Value:** 3.6 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** 0 - **Spread:** - **Upper Limit:** 6.6 - **Value:** 2.6 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** The lesion size is measured in square centimeters. **Dispersion Type:** Full Range **Parameter Type:** MEAN **Population Description:** One participant was lost to follow up **Reporting Status:** POSTED **Time Frame:** 3 months **Title:** Lesion Size **Type:** PRIMARY **Unit of Measure:** cm^2 ##### Group **Description:** 50 ml vials of sodium thiosulfate (250mg/ml) will be used for treatment. Sodium Thiosulfate: A volume of 0.1 ml/cm2 of STS will be injected into each lesion. **ID:** OG000 **Title:** Sodium Thiosulfate ##### Group **Description:** 30 ml vials of sodium chloride 0.9% will be used for the control treatment. Saline Solution: A volume of 0.1 ml/cm2 of normal saline will be injected into the control lesion. **ID:** OG001 **Title:** Saline Solution #### Outcome Measure 2 **Description:** The PGA will be done by the physician to assess appearance and changes in the lesion. The lesion will be assigned a score from 0 (clear) to 4 (severe). **Dispersion Type:** Full Range **Parameter Type:** MEAN **Population Description:** One participant was lost to follow up. **Reporting Status:** POSTED **Time Frame:** 3 months **Title:** Physician Global Assessment (PGA) **Type:** PRIMARY **Unit of Measure:** score on a scale ##### Group **Description:** 50 ml vials of sodium thiosulfate (250mg/ml) will be used for treatment. Sodium Thiosulfate: A volume of 0.1 ml/cm2 of STS will be injected into each lesion. **ID:** OG000 **Title:** Sodium Thiosulfate ##### Group **Description:** 30 ml vials of sodium chloride 0.9% will be used for the control treatment. Saline Solution: A volume of 0.1 ml/cm2 of normal saline will be injected into the control lesion. **ID:** OG001 **Title:** Saline Solution #### Outcome Measure 3 **Description:** The VAS for pain is used to assess the pain associated with the lesion. The pain will be recorded as a number from 1 (no pain) to 10 (severe pain). **Dispersion Type:** Full Range **Parameter Type:** MEAN **Population Description:** One participant was lost to follow up. **Reporting Status:** POSTED **Time Frame:** 3 months **Title:** Visual Analog Scale (VAS) for Pain **Type:** PRIMARY **Unit of Measure:** score on a scale ##### Group **Description:** 50 ml vials of sodium thiosulfate (250mg/ml) will be used for treatment. Sodium Thiosulfate: A volume of 0.1 ml/cm2 of STS will be injected into each lesion. **ID:** OG000 **Title:** Sodium Thiosulfate ##### Group **Description:** 30 ml vials of sodium chloride 0.9% will be used for the control treatment. Saline Solution: A volume of 0.1 ml/cm2 of normal saline will be injected into the control lesion. **ID:** OG001 **Title:** Saline Solution ### Participant Flow Module #### Group **Description:** All study participants received an injection of Sodium Thiosulfate into one lesion. They received an injection of saline in the control lesion 50 ml vials of sodium thiosulfate (250mg/ml) were used for treatment. 30 ml vials of sodium chloride 0.9% will be used for the control treatment. A volume of 0.1 ml/cm2 of STS or normal will be injected into each lesion. **ID:** FG000 **Title:** All Paticipants #### Period **Title:** Overall Study ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 5 **Number of Units:** 10 ##### Milestone **Type:** Baseline ###### Achievement **Group ID:** FG000 **Number of Subjects:** 5 **Number of Units:** 10 ##### Milestone **Type:** Month 1 ###### Achievement **Group ID:** FG000 **Number of Subjects:** 5 **Number of Units:** 10 ##### Milestone **Type:** Month 2 ###### Achievement **Group ID:** FG000 **Number of Subjects:** 4 **Number of Units:** 8 ##### Milestone **Type:** Month 3 ###### Achievement **Group ID:** FG000 **Number of Subjects:** 4 **Number of Units:** 8 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 4 **Number of Units:** 8 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 1 **Number of Units:** 2 **Pre-Assignment Details:** The study had to be close early due to lack of support and time resulting in a lower number of participants than hoped for. 5 participants began the study, 1 was lost to follow-up. Each participant had 2 study lesions, each assigned to a different study arm. **Type Units Analyzed:** Lesions **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT00328679 **Brief Title:** Evaluation of Food Hypersensitivity in Children/Adolescents With Functional Dyspepsia **Official Title:** Food-Specific IgE, IgG, IgG4, Skin Prick Testing and Atopy Patch Testing in Children/Adolescents With Functional Dyspepsia: A Pilot Study #### Organization Study ID Info **ID:** 0604-068 #### Organization **Class:** OTHER **Full Name:** Children's Mercy Hospital Kansas City ### Status Module #### Completion Date **Date:** 2008-09 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2008-10-08 **Type:** ESTIMATED **Last Update Submit Date:** 2008-10-06 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2008-09 **Type:** ACTUAL #### Start Date **Date:** 2006-06 **Status Verified Date:** 2008-10 #### Study First Post Date **Date:** 2006-05-22 **Type:** ESTIMATED **Study First Submit Date:** 2006-05-18 **Study First Submit QC Date:** 2006-05-18 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Children's Mercy Hospital Kansas City #### Responsible Party **Old Name Title:** Nancy Neilan, MT (ASCP)/ Research Coordinator **Old Organization:** Children's Mercy Hospitals and Clinics ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The main purpose of this study is to determine if standard and investigational tests used to help diagnose and treat food allergies can provide information that will be useful in determining the cause of dyspepsia and helpful in designing a treatment plan. The study will also determine if there is a connection between positive allergy tests and inflammation in the upper abdomen. **Detailed Description:** Recurrent abdominal pain is the most common type of pain in school age children and young adolescents. Over 80% of these children have pain in the upper abdomen which is diagnosed as functional dyspepsia (FD). Many of these children are also found to have eosinophilic duodenitis (ED). ED is a type of inflammation in the lining of the gastrointestinal tract characterized by an increase in eosinophils. An increase in intestinal eosinophils is a finding also seen with food allergy upon exposure to the offending antigen. The presence of intestinal eosinophilia in ED would suggest an allergic mechanism may be involved in the generation of pain and other symptoms associated with ED. Endoscopy and biopsy are used to aid in the identification of ED, which often is followed by elimination diets and food challenges to identify the offending allergen. This approach is both invasive (due to endoscopy) and cumbersome (due to the complexity and restrictiveness of the elimination diet). The value of screening for food hypersensitivities in children with ED has not been well characterized despite the theoretical links between food hypersensitivities, gut inflammation, and symptoms of dyspepsia. The current study will determine if standard and investigational tests used to evaluate food hypersensitivity have the potential to be used as biomarkers to direct treatment of children with ED. ### Conditions Module **Conditions:** - Dyspepsia **Keywords:** - abdominal - stomach - dyspepsia - food allergy ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** SINGLE **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Count:** 41 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Device: in vitro and in vivo allergy testing **Label:** A **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - A **Description:** Patch Test: food to be tested is suspended in sterile saline, placed on the skin of the back using a Finn Chamber secured with surgical tape and left in place for 48 hours. **Name:** in vitro and in vivo allergy testing **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Measure:** measure specific IgE, IgG total, IgG subclass 4, skin prick tests and atopy patch tests to milk, egg, soy, corn, peanut and wheat **Time Frame:** 48 hrs and 72 hrs after patch placement #### Secondary Outcomes **Measure:** Determine T-lymphocytes, eosinophils and mast cell densities on duodenal biopsy samples demonstrating eosinophilia **Time Frame:** collected at time of biopsy, patient group only ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Age of 8-17 years, inclusive * Diagnosis of functional dyspepsia (FD) by physician based on Rome II criteria (patient group only) * Undergoing endoscopy to evaluate FD following demonstration of a lack of clinical response to acid reduction therapy (patient group only) * Informed permission/assent Exclusion Criteria (patient goup): * Previous testing for food-specific IgE, IgG, IgG4, skin prick or allergy patch tests within the past 12 months or any previous positive result(s) for food-specific IgE, IgG, IgG4, SPT or APT to milk, egg, soy, corn, peanut or wheat; * Any use of steroids or leukotriene receptor antagonists within one month prior to the study * Any use of antihistamines, antihistamine-like drugs or topical steroid within two weeks prior to the study * Any chronic non-gastrointestinal illness requiring regular medical care Exclusion Criteria (healthy control group) * In addition to patient exclusion criteria as defined above * Any current or chronic history within the previous 6 months of gastrointestinal symptoms including abdominal pain or discomfort, nausea, vomiting, bloating, diarrhea or constipation * History of asthma or chronic respiratory symptoms * History of allergic rhinitis or chronic sinusitis * History of allergic reactions attributed to food **Maximum Age:** 17 Years **Minimum Age:** 8 Years **Sex:** ALL **Standard Ages:** - CHILD ### Contacts Locations Module #### Locations **Location 1:** **City:** Kansas City **Country:** United States **Facility:** The Children's Mercy Hospital and Clinics **State:** Missouri **Zip:** 64108 #### Overall Officials **Official 1:** **Affiliation:** The Children's Mercy Hospital and Clinics **Name:** Nancy A Neilan, MT (ASCP) **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Neilan NA, Dowling PJ, Taylor DL, Ryan P, Schurman JV, Friesen CA. Useful biomarkers in pediatric eosinophilic duodenitis and their existence: a case-control, single-blind, observational pilot study. J Pediatr Gastroenterol Nutr. 2010 Apr;50(4):377-84. doi: 10.1097/MPG.0b013e3181c2c28a. **PMID:** 20216101 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012817 - Term: Signs and Symptoms, Digestive ### Condition Browse Module - Browse Branches - Abbrev: BC20 - Name: Immune System Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M10018 - Name: Hypersensitivity - Relevance: LOW - As Found: Unknown - ID: M7589 - Name: Dyspepsia - Relevance: HIGH - As Found: Dyspepsia - ID: M8636 - Name: Food Hypersensitivity - Relevance: LOW - As Found: Unknown - ID: M15622 - Name: Signs and Symptoms, Digestive - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000004415 - Term: Dyspepsia ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02372279 **Acronym:** PROdE **Brief Title:** Prospective Randomized Study to Evaluate the Effect of Embryonic Observation on the Gestation (PROdE) **Official Title:** Prospective Randomized Study to Evaluate the Effect of Embryonic Observation on the Live Birth Rate (LBR) #### Organization Study ID Info **ID:** InstitutoBernabeu #### Organization **Class:** OTHER **Full Name:** Instituto Bernabeu ### Status Module #### Completion Date **Date:** 2021-03 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-06-23 **Type:** ACTUAL **Last Update Submit Date:** 2021-06-21 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2021-03 **Type:** ACTUAL #### Start Date **Date:** 2015-02 **Type:** ACTUAL **Status Verified Date:** 2021-06 #### Study First Post Date **Date:** 2015-02-26 **Type:** ESTIMATED **Study First Submit Date:** 2015-02-20 **Study First Submit QC Date:** 2015-02-20 **Why Stopped:** The forecast regarding the number of patients that could enter the study were not met in the designated period ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Instituto Bernabeu #### Responsible Party **Investigator Affiliation:** Instituto Bernabeu **Investigator Full Name:** Jorge Ten Morro **Investigator Title:** Ph.D. Embryologist **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** In a conventional in vitro fertilization (IVF) cycle, daily microscopic observation of embryos outside the incubator to assess their morphology and establish a selection process is performed. In this way it is possible to know which embryo or embryos have greater implantantion capacity and will be transferred to the uterus to obtain a viable pregnancy. However, these observations can produce deleterious effects on embryo development due to changes in temperature, pH and osmolarity of the culture media, as well as a negative effect of direct light microscope for observation. This project aims to test the hypothesis that non-embryonic observation produces a beneficial effect on embryo quality until day 5 of development (blastocyst stage) and, therefore, on rates of implantation and ongoing gestation, compared with the conventional protocol of observations under the inverted microscope on days two, three and five of development. **Detailed Description:** The present study is a prospective double-blind randomised controlled trial (RCT) approved by a local Ethics Committe. Summarily, in the control group we did three embryo observations outside the incubator as we usually do in our conventional protocol: day 2, day 3 and day 5, just before ET; in the study group we performed a unique embryo observation on day 5 before ET. All the subjects that participated in the study were informed and gave us their consent to take part on it. The inclusion criteria of the study were: first cycle of ART treatment with conventional IVF or ICSI with donated eggs, normal uterine cavity and a single or double embryo transfer, always performed on day 5 at blastocyst stage. The exclusion criteria were the following: patients above 50 years old, patients that were diagnosed with recurrent implantation failure (RIF) and/or recurrent pregnancy loss (RPL) or uterine pathologies, body mass index \>30 kg/m2, the use of seminal samples coming from donors or testicular origin and cycles that included preimplantational genetic testing (PGT). LBR was the main outcome of our study, defined as the number of deliveries that resulted in a live born neonate, expressed per 100 embryo transfers (Zegers- Hochschild et al., 2009). Secondary efficacy endpoints were positive hCG rate (\>5 mUI/mL, assessed in serum 14 days after oocyte retrieval), implantation rate (number of gestational sacs observed divided by the number of embryos transferred, expressed as a percentage, %), ongoing pregnancy rate, defined as a pregnancy with a detectable heart rate at 12 weeks of gestation or beyond and miscarriage (loss of a pregnancy after 12 weeks of gestation). In addition, we assessed the next IVF laboratory parameters: fertilization rate, blastocyst formation rate on day 5, blastocyst quality, number of transferred embryos and number of usable blastocysts (number of blastocysts transferred and frozen). The sample size calculation was based on the primary outcome. We assumed a live birth rate of 40% in the control group, compared to 50% in the non-observational group deduced from previous studies. By applicating the sample size calculation program, 776 patients (388 per group) were required in order to detect a risk difference (RD) of 10% between the two groups in the final analysis, with a power of 80% at a two-sided, adjusted alpha-level of 0.05. A follow-up loss rate of 10% was estimated. ### Conditions Module **Conditions:** - Placenta; Implantation **Keywords:** - Embryo development - Implantation - In vitro fertilization ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER **Primary Purpose:** OTHER #### Enrollment Info **Count:** 193 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Study group. No embryonic observation from day one to day five of embryo development. **Label:** No embryo observation (NEO) **Type:** NO_INTERVENTION #### Arm Group 2 **Description:** Control group. Conventional embryonic observations performed in day two, three and five of embryo development. **Intervention Names:** - Other: Embryo observation **Label:** Embryo observation (EO) **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Embryo observation (EO) **Description:** In EO group, conventional observations will be made. **Name:** Embryo observation **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** The number of deliveries that resulted in a live born neonate, expressed per 100 embryos **Measure:** Live Birth Rate **Time Frame:** Nine months after treatment. #### Secondary Outcomes **Description:** Number of treatments with a level of BhCG \>5 mUI/mL, assessed in serum. **Measure:** Positive hCG rate **Time Frame:** 14 days after oocyte retrieval. **Description:** Number of gestational sacs observed divided by the number of embryos transferred. expressed as a percentage, %. **Measure:** Implantation rate **Time Frame:** 6-7 weeks after oocyte retrieval. **Description:** Defined as a pregnancy with a detectable heart rate at 12 weeks of gestation or beyond. **Measure:** Ongoing pregnancy rate **Time Frame:** 12 weeks after B-hCG positive test **Description:** Loss of a pregnancy after 12 weeks of gestation. **Measure:** Miscarriage rate **Time Frame:** From 12 weeks after gestation to delivery **Description:** Number of fertilized oocytes out of the total microinjected or inseminated. **Measure:** Fertilization rate **Time Frame:** 16-18 hours after microinjection or insemination **Description:** Number of embryos that reach the blastocyst stage at day 5 **Measure:** Blastocyst formation rate on day 5 **Time Frame:** 5 days after microinjection or insemination. **Description:** The assessment of blastocyst morphology in several grades **Measure:** Blastocyst quality **Time Frame:** 5 days after microinjection or insemination. **Description:** Number of blastocysts transferred and frozen. **Measure:** Number of usable blastocysts **Time Frame:** 5 days after microinjection or insemination. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * First cycle of ART treatment with conventional IVF or ICSI with donated eggs. * Normal uterine cavity and a single or double embryo transfer, always performed on day 5 at blastocyst stage. Exclusion Criteria: * Patients above 50 years old, patients that were diagnosed with recurrent implantation failure (RIF) and/or recurrent pregnancy loss (RPL) or uterine pathologies, * Body mass index \>30 kg/m2. * The use of seminal samples coming from donors or testicular origin. * Cycles that included preimplantational genetic testing (PGT). **Maximum Age:** 50 Years **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Alicante **Country:** Spain **Facility:** Instituto Bernabeu **Zip:** 03016 #### Overall Officials **Official 1:** **Affiliation:** Instituto Bernabeu **Name:** Jorge Ten Morro, Ph.D. **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Alpha Scientists in Reproductive Medicine and ESHRE Special Interest Group of Embryology. The Istanbul consensus workshop on embryo assessment: proceedings of an expert meeting. Hum Reprod. 2011 Jun;26(6):1270-83. doi: 10.1093/humrep/der037. Epub 2011 Apr 18. **PMID:** 21502182 **Citation:** Fujiwara M, Takahashi K, Izuno M, Duan YR, Kazono M, Kimura F, Noda Y. Effect of micro-environment maintenance on embryo culture after in-vitro fertilization: comparison of top-load mini incubator and conventional front-load incubator. J Assist Reprod Genet. 2007 Jan;24(1):5-9. doi: 10.1007/s10815-006-9088-3. Epub 2006 Dec 13. **PMID:** 17160731 **Citation:** Gardner DK, Lane M, Stevens J, Schlenker T, Schoolcraft WB. Blastocyst score affects implantation and pregnancy outcome: towards a single blastocyst transfer. Fertil Steril. 2000 Jun;73(6):1155-8. doi: 10.1016/s0015-0282(00)00518-5. **PMID:** 10856474 **Citation:** Bernabeu R, Roca M, Torres A, Ten J. Indomethacin effect on implantation rates in oocyte recipients. Hum Reprod. 2006 Feb;21(2):364-9. doi: 10.1093/humrep/dei343. Epub 2005 Nov 10. **PMID:** 16284067 **Citation:** Herrero J, Tejera A, Albert C, Vidal C, de los Santos MJ, Meseguer M. A time to look back: analysis of morphokinetic characteristics of human embryo development. Fertil Steril. 2013 Dec;100(6):1602-9.e1-4. doi: 10.1016/j.fertnstert.2013.08.033. Epub 2013 Sep 29. **PMID:** 24083877 **Citation:** Zhang JQ, Li XL, Peng Y, Guo X, Heng BC, Tong GQ. Reduction in exposure of human embryos outside the incubator enhances embryo quality and blastulation rate. Reprod Biomed Online. 2010 Apr;20(4):510-5. doi: 10.1016/j.rbmo.2009.12.027. Epub 2009 Dec 28. **PMID:** 20129824 **Citation:** Kirkegaard K, Kesmodel US, Hindkjaer JJ, Ingerslev HJ. Time-lapse parameters as predictors of blastocyst development and pregnancy outcome in embryos from good prognosis patients: a prospective cohort study. Hum Reprod. 2013 Oct;28(10):2643-51. doi: 10.1093/humrep/det300. Epub 2013 Jul 30. **PMID:** 23900207 **Citation:** Steptoe PC, Edwards RG. Birth after the reimplantation of a human embryo. Lancet. 1978 Aug 12;2(8085):366. doi: 10.1016/s0140-6736(78)92957-4. No abstract available. **PMID:** 79723 **Citation:** Bognar Z, Csabai TJ, Pallinger E, Balassa T, Farkas N, Schmidt J, Gorgey E, Berta G, Szekeres-Bartho J, Bodis J. The effect of light exposure on the cleavage rate and implantation capacity of preimplantation murine embryos. J Reprod Immunol. 2019 Apr;132:21-28. doi: 10.1016/j.jri.2019.02.003. Epub 2019 Mar 4. **PMID:** 30852462 **Citation:** Barberet J, Chammas J, Bruno C, Valot E, Vuillemin C, Jonval L, Choux C, Sagot P, Soudry A, Fauque P. Randomized controlled trial comparing embryo culture in two incubator systems: G185 K-System versus EmbryoScope. Fertil Steril. 2018 Feb;109(2):302-309.e1. doi: 10.1016/j.fertnstert.2017.10.008. Epub 2017 Nov 23. **PMID:** 29175066 **Citation:** Armstrong S, Bhide P, Jordan V, Pacey A, Marjoribanks J, Farquhar C. Time-lapse systems for embryo incubation and assessment in assisted reproduction. Cochrane Database Syst Rev. 2019 May 29;5(5):CD011320. doi: 10.1002/14651858.CD011320.pub4. **PMID:** 31140578 **Citation:** Kalleas D, McEvoy K, Horne G, Roberts SA, Brison DR. Live birth rate following undisturbed embryo culture at low oxygen in a time-lapse incubator compared to a high-quality benchtop incubator. Hum Fertil (Camb). 2022 Feb;25(1):147-153. doi: 10.1080/14647273.2020.1729423. Epub 2020 Feb 26. **PMID:** 32098536 ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03275779 **Acronym:** EXFREQ **Brief Title:** Investigating the Role of Resistance Exercise Frequency in the Regulation of Skeletal Muscle Mass **Official Title:** Investigating the Role of Resistance Exercise Frequency in the Regulation of Skeletal Muscle Mass #### Organization Study ID Info **ID:** ERN_17-0997 #### Organization **Class:** OTHER **Full Name:** University of Birmingham ### Status Module #### Completion Date **Date:** 2018-12-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-01-05 **Type:** ACTUAL **Last Update Submit Date:** 2023-01-03 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2018-12-01 **Type:** ACTUAL #### Start Date **Date:** 2018-01-01 **Type:** ACTUAL **Status Verified Date:** 2022-12 #### Study First Post Date **Date:** 2017-09-08 **Type:** ACTUAL **Study First Submit Date:** 2017-08-31 **Study First Submit QC Date:** 2017-09-05 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Maastricht University #### Lead Sponsor **Class:** OTHER **Name:** University of Birmingham #### 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 will investigate whether manipulating resistance exercise frequency impacts muscle protein synthesis rates. The investigators will test the hypothesise that a higher resistance exercise frequency will result in greater muscle protein synthesis rates than a lower resistance exercise frequency. **Detailed Description:** Resistance exercise is currently the most effective means of building or maintaining muscle mass. Resistance exercise guidelines generally suggest that those looking to increase muscle mass should train each muscle group once to twice per week. However, it has been proposed that it may be of greater benefit to train a muscle group with a higher frequency (i.e., four to six times per week) than currently suggested. If true, completing the same total volume of resistance exercise in more frequent, smaller bouts could prove to be a more beneficial strategy to optimally build or maintain muscle mass. Therefore, this study will investigate whether manipulating resistance exercise frequency impacts cumulative muscle protein synthesis rates in young individuals. Participants will undergo a 7 day period of habitual activity before completing the same total volume of resistance exercise as either; i) one isolated bout (low frequency) or ii) five smaller bouts (high frequency) over a period of 7 days. ### Conditions Module **Conditions:** - Physical Activity ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER ##### Masking Info **Masking:** NONE **Primary Purpose:** BASIC_SCIENCE #### Enrollment Info **Count:** 9 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants complete 7 days of habitual physical activity followed by a 7 day period where participants complete a single bout of unilateral resistance exercise. **Intervention Names:** - Behavioral: Low Frequency Condition **Label:** Low Frequency Condition **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants complete 7 days of habitual physical activity followed by a 7 day period where participants complete the same total volume of resistance exercise as the low frequency condition as five smaller bouts of unilateral resistance exercise. **Intervention Names:** - Behavioral: High Frequency Condition **Label:** High Frequency Condition **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Low Frequency Condition **Description:** Young, untrained participants complete 7 days of habitual physical activity followed by a 7 day period where participants complete a single bout of unilateral resistance exercise. **Name:** Low Frequency Condition **Type:** BEHAVIORAL #### Intervention 2 **Arm Group Labels:** - High Frequency Condition **Description:** Young, untrained participants complete 7 days of habitual physical activity followed by a 7 day period where participants complete the same total volume of resistance exercise as the low frequency condition as five smaller bouts of unilateral resistance exercise. **Name:** High Frequency Condition **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** The change in muscle protein synthesis rates (FSR %/day) from baseline will be determined between the low and high frequency conditions at days 10 and 15 using deuterium oxide (D2O). **Measure:** Change in muscle protein synthesis (FSR %/day) **Time Frame:** At day 10 and 15. #### Secondary Outcomes **Description:** The change in acute satellite cell response will be determined in muscle samples following low and high frequency resistance exercise at days 10 and 15. **Measure:** Change in satellite cell response **Time Frame:** At day 10 and 15. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Body mass index (18.5-29.99 kg/m2) * Untrained - defined as: Perform activities of daily living and recreation but have completed no regular lower body resistance-type exercise (e.g., weight training) activity in the last year. * Good general health Exclusion Criteria: * Lidocaine allergy * Hypertension (≥140/90 mmHg) * Current participation in another clinical study * Previous participation in this study * Bleeding disorder/s * Current or recent smoker * Vegetarian or vegan * Past history of substance abuse and/or taking prescription or non-prescription medication (e.g., beta-blockers, insulin or thyroxine) or supplements that may influence normal metabolic responses. **Gender Based:** True **Gender Description:** Participants must be male. **Healthy Volunteers:** True **Maximum Age:** 35 Years **Minimum Age:** 18 Years **Sex:** MALE **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Birmingham **Country:** United Kingdom **Facility:** School of Sport Exercise and Rehabilitation Sciences, University of Birmingham **State:** West Midlands **Zip:** B15 2TT #### Overall Officials **Official 1:** **Affiliation:** University of Birmingham **Name:** Gareth Wallis, 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:** NCT03901079 **Brief Title:** Trial for A Novel Facilitated Antegrade Steering Technique in Revascularization of Coronary Chronic Total Occlusions **Official Title:** A Prospective, Multi-center, Non-Randomized Controled Trial for A Novel Facilitated Antegrade Steering Technique in Revascularization of Coronary Chronic Total Occlusions In Chinese Population--FAST CTO China #### Organization Study ID Info **ID:** S2362 #### Organization **Class:** INDUSTRY **Full Name:** Boston Scientific Corporation ### Status Module #### Completion Date **Date:** 2021-03-24 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-12-08 **Type:** ACTUAL **Last Update Submit Date:** 2023-12-06 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2020-01-10 **Type:** ACTUAL #### Results First Post Date **Date:** 2023-12-08 **Type:** ACTUAL **Results First Submit Date:** 2021-03-25 **Results First Submit QC Date:** 2023-12-06 #### Start Date **Date:** 2018-10-08 **Type:** ACTUAL **Status Verified Date:** 2023-12 #### Study First Post Date **Date:** 2019-04-03 **Type:** ACTUAL **Study First Submit Date:** 2018-08-10 **Study First Submit QC Date:** 2019-04-02 **Why Stopped:** HGRAC approval letter had expired. The enrollment speed of FAST CTO is much slower than planning and the scientific value becomes limited due to the delayed data readout anticipation. ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Boston Scientific Corporation #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** To assess the safety and efficacy of the BridgePoint CTO system in recanalization of CTO lesions which are resistant to a conventional wire approach in a multicenter study in Chinese population **Detailed Description:** All subjects who are candidates for percutaneous coronary intervention (PCI), signed the informed consent form and had chronic total coronary occlusion (CTO) lesion will be evaluated for enrollment in this study.At least 100 subjects will be enrolled. Each site will be allowed to enroll up to a maximum of 25 subjects. Primary Effectiveness Endpoint: Technical Success: the ability of the BridgePoint Medical System to successfully facilitate placement of a guidewire beyond a CTO lesion in the true vessel lumen/within the collaterals in those cases that were otherwise refractory to treatment with a currently marketed guidewire Primary Safety Endpoint: 30-day MACE rate for CTO cases in which the BridgePoint Medical System was used. MACE is defined as the composite of cardiac death, Q-wave and non-Q-wave myocardial infarction(MI), and any ischemia-driven target lesion revascularization(TLR). ### Conditions Module **Conditions:** - CAD ### Design Module #### Design Info **Observational Model:** CASE_ONLY **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 20 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** BridgePoint CTO System: * CrossBoss Catheter * Stingray LP Catheter * Stingray Guidewire and Extension Wire **Intervention Names:** - Other: single-arm study **Label:** CTO BridgePoint system ### Interventions #### Intervention 1 **Arm Group Labels:** - CTO BridgePoint system **Description:** single-arm study **Name:** single-arm study **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** 6 subjects have the results data, and 5 of them (83.3%) reported technical success. One (16.7%) failed. **Measure:** Technical Success: Successfully Facilitate Placement of a Guidewire Beyond a CTO Lesion in the True Vessel Lumen/Within the Collaterals **Time Frame:** Subject follow up will occur via telephone contact or clinic visit at 30 days,6 and 12 months post index procedure. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Subject must be ≥ 18 but \< 80 years of age * Subject (or legal guardian) understands the trial requirements and the treatment procedures and provides written informed consent before any trial-specific tests or procedures are performed * Subject is eligible for percutaneous coronary intervention (PCI) * Subject has symptomatic coronary artery disease or myocardial infraction (MI) with objective evidence of ischemia or silent ischemia * Subject is an acceptable candidate for coronary artery bypass grafting (CABG) * Subject is willing to comply with all protocol-required follow-up evaluation * Subject has a left ventricular ejection fraction (LVEF) ≥45% as measured within 60 days prior to enrollment Angiographic Inclusion Criteria: AI1. A maximum of one de novo CTO lesion in a native coronary artery with thrombolysis in Myocardial Infarction (TIMI) flow grade 0 AI2. Non-acute CTO lesion with an estimated duration of at least 3 months by clinical history and/or comparison with previous angiogram or electrocardiogram(ECG) AI3. The CTO lesion must have an angiographic landing zone≥ 10 mm proximal to any major bifurcation without severe calcification. AI4. Lesion length \< 40mm without excessive tortuosity and angulation(\>45°) Exclusion Criteria: * Subject has clinical symptoms and/or ECG changes consistent with Acute MI (include STEMI and Non- STEMI) within 1 week * Subject has cardiogenic shock, hemodynamic instability requiring inotropic or mechanical circulatory support, intractable ventricular arrhythmias, or ongoing intractable angina * Subject has received an organ transplant or is on a waiting list for an organ transplant * Subject is receiving or scheduled to receive chemotherapy within 30 days before or after the index procedure * Planned PCI (including staged procedures) or CABG after the index procedure * Subject has a known allergy to contrast (that cannot be adequately pre-medicated) and/or protocol-required concomitant medications (e.g., aspirin or all thienopyridines) * Subject has one of the following (as assessed prior to the index procedure): Other serious medical illness (e.g., cancer, congestive heart failure) with estimated life expectancy of less than 12 months / Current problems with substance abuse (e.g., alcohol, cocaine, heroin, etc.) / Planned procedure that may cause non-compliance with the protocol or confound data interpretation * Subject is receiving chronic (≥72 hours) anticoagulation therapy (i.e., heparin, coumadin) * Subject with out of range complete blood count (CBC) values that are determined by the study physician to be clinically significant. * Subject is on dialysis or has baseline serum creatinine level \>2.0 mg/dL (177µmol/L) * Subject has a history of bleeding diathesis or coagulopathy or will refuse blood transfusions * Subject has had a history of cerebrovascular accident (CVA) or transient ischemic attack (TIA) within the past 6 months * Subject has an active peptic ulcer or active gastrointestinal (GI) bleeding * Subject has signs or symptoms of active heart failure (i.e., NYHA class IV) or LVEF \< 45% at the time of the index procedure * Subject is participating in another investigational drug or device clinical trial that has not reached its primary endpoint or intends to participate in another investigational drug or device clinical trial within 12 months after the index procedure * Subject had PCI within the previous 2 weeks * Subject with known intention to procreate within 12 months after the index procedure (women of child-bearing potential who are sexually active must agree to use a reliable method of contraception from the time of screening through 12 months after the index procedure) * Subject is a woman who is pregnant or nursing (a pregnancy test must be performed within 7 days prior to the index procedure in women of child-bearing potential) Angiographic Exclusion Criteria: AE1. Target lesion is an aorto-ostial lesion or located in left main coronary artery, previous venous or arterial bypass grafts AE2. Target lesion involving a segment of previous stent AE3. Target vessel has excessive tortuosity and/or angulation proximal to the target lesion(\>45°) AE4. Target lesion and/or the target vessel proximal to the target lesion is moderately to severely calcified by visual estimate AE5. Target lesion is located within 5 mm of the origin of the left anterior descending (LAD) coronary artery or left circumflex (LCx) coronary artery by visual estimate AE6. Target lesion will be accessed via a saphenous vein graft or arterial graft AE7. Subject has unprotected left main coronary artery disease (\>50% diameter stenosis) AE8. Thrombus, or possible thrombus, present in the target vessel (by visual estimate) AE9. Target vessel has a dissection greater than National Heart, Lung, Blood Institute (NHLBI) type C **Maximum Age:** 80 Years **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** All subjects who are candidates for percutaneous coronary intervention (PCI), signed the informed consent form and had chronic total coronary occlusion (CTO) lesion will be evaluated for enrollment in this study. ### Contacts Locations Module #### Locations **Location 1:** **City:** Chengdu **Country:** China **Facility:** West China Hospital Sichuan University **State:** Sichuan **Zip:** 610041 #### Overall Officials **Official 1:** **Affiliation:** Fuwai Hospital, CAMS & PUMC **Name:** Yuejin Yang, Doc. **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Document Section ### Large Document Module #### Large Docs - Date: 2019-11-12 - Filename: Prot_001.pdf - Has ICF: False - Has Protocol: True - Has SAP: False - Label: Study Protocol - Size: 689627 - Type Abbrev: Prot - Upload Date: 2021-03-22T23:08 - Date: 2020-01-20 - Filename: SAP_002.pdf - Has ICF: False - Has Protocol: False - Has SAP: True - Label: Statistical Analysis Plan - Size: 377857 - Type Abbrev: SAP - Upload Date: 2022-03-01T01:20 ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: Rare - Name: Rare Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: T1303 - Name: Chronic Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** CTO BridgePoint System **Deaths Num At Risk:** 20 **Description:** BridgePoint CTO System: * CrossBoss Catheter * Stingray LP Catheter * Stingray Guidewire and Extension Wire single-arm study: single-arm study **ID:** EG000 **Other Num at Risk:** 20 **Serious Number Affected:** 4 **Serious Number At Risk:** 20 **Title:** CTO BridgePoint System **Frequency Threshold:** 0 #### Serious Events **Term:** Coronary artery atherosclerotic heart disease **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 10.0 ##### Stats **Group ID:** EG000 **Num Affected:** 4 **Num At Risk:** 20 **Num Events:** 4 **Time Frame:** Baseline,30 days, 6 months and 12 months ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 20 **Units:** Participants ### Group **ID:** BG000 **Title:** CTO BridgePoint System **Description:** BridgePoint CTO System: * CrossBoss Catheter * Stingray LP Catheter * Stingray Guidewire and Extension Wire single-arm study: single-arm study ### Measure #### Measurement **Group ID:** BG000 **Spread:** 9.4 **Value:** 57.5 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 20 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 3 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 17 **Category Title:** Male #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 20 **Class Title:** ### Measure #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 0 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 4 **Category Title:** Yes #### Measurement **Group ID:** BG000 **Value:** 16 **Category Title:** No #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 20 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 18 **Category Title:** Yes #### Measurement **Group ID:** BG000 **Value:** 2 **Category Title:** No #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 20 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 9 **Category Title:** Yes #### Measurement **Group ID:** BG000 **Value:** 11 **Category Title:** No #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 20 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 3 **Category Title:** Yes #### Measurement **Group ID:** BG000 **Value:** 16 **Category Title:** No #### Measurement **Group ID:** BG000 **Value:** 1 **Category Title:** Unknown #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 20 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 3 **Category Title:** Yes #### Measurement **Group ID:** BG000 **Value:** 17 **Category Title:** No #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 20 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Yes #### Measurement **Group ID:** BG000 **Value:** 20 **Category Title:** No #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 20 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 6 **Category Title:** None #### Measurement **Group ID:** BG000 **Value:** 5 **Category Title:** Stable angina #### Measurement **Group ID:** BG000 **Value:** 8 **Category Title:** Unstable angina #### Measurement **Group ID:** BG000 **Value:** 1 **Category Title:** Unknown #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 20 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Yes #### Measurement **Group ID:** BG000 **Value:** 20 **Category Title:** No #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 20 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 8 **Category Title:** Yes #### Measurement **Group ID:** BG000 **Value:** 12 **Category Title:** No #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 20 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Yes #### Measurement **Group ID:** BG000 **Value:** 19 **Category Title:** No #### Measurement **Group ID:** BG000 **Value:** 1 **Category Title:** Unknown #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 20 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 1 **Category Title:** Yes #### Measurement **Group ID:** BG000 **Value:** 19 **Category Title:** No #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 20 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 19 **Category Title:** Yes #### Measurement **Group ID:** BG000 **Value:** 1 **Category Title:** No #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 20 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Yes #### Measurement **Group ID:** BG000 **Value:** 20 **Category Title:** No #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 20 **Class Title:** **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 **Population Description:** Race and Ethnicity were not collected from any participant. **Title:** Race and Ethnicity Not Collected **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Current diabetes mellitus **Unit of Measure:** Participants ### Measure 5 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** History of hyperlipidemia requiring medication **Unit of Measure:** Participants ### Measure 6 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** History of hypertension requiring medication **Unit of Measure:** Participants ### Measure 7 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Family history of coronary artery disease **Unit of Measure:** Participants ### Measure 8 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** History of Myocardial Infarction (MI) **Unit of Measure:** Participants ### Measure 9 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** History of congestive heart failure **Unit of Measure:** Participants ### Measure 10 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Current anginal status **Unit of Measure:** Participants ### Measure 11 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Does Subject have silent ischemia **Unit of Measure:** Participants ### Measure 12 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** History of Percutaneous Coronary Intervention (PCI) **Unit of Measure:** Participants ### Measure 13 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** History of Coronary Artery Bypass Graft (CABG) surgery **Unit of Measure:** Participants ### Measure 14 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** History of arrhythmia **Unit of Measure:** Participants ### Measure 15 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Left Ventricular Ejection Fraction (LVEF) measured **Unit of Measure:** Participants ### Measure 16 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** History of cardiogenic shock **Unit of Measure:** Participants **Population Description:** Subject demographics, Physical assessment, Medical history, Angina assessment, LVEF, Cardiac medication, risk factors, and pre-procedure lesion characteristics will be summarized using descriptive statistics for continuous variables and frequency tables for discrete variables. No formal statistical testing will be done since this is a single-arm trial. ## Results Section - More Information Module ### Certain Agreement ### Limitations and Caveats **Description:** HGRAC rejected all initiated applications from the leading site without any comments. Only leading site can submit the HGRAC application for multi-center studies and the ban lifting date of leading site is still unclear, it's not allowed to recruit or collect any data from the subjects after Aug 2020 (the expiry date of the approval ). The enrollment speed of FAST CTO is much slower than planning and the scientific value becomes limited due to the delayed data readout anticipation. ### Point of Contact **Email:** [email protected] **Organization:** BSC **Phone:** +861085216427 **Title:** Chenchen FAN ## Results Section - Outcome Measures Module ### Outcome Measure 1 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 **Title:** Success ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** Failed ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** 6 subjects have the results data, and 5 of them (83.3%) reported technical success. One (16.7%) failed. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Only 6 subjects have the results of technical success or not. **Reporting Status:** POSTED **Time Frame:** Subject follow up will occur via telephone contact or clinic visit at 30 days,6 and 12 months post index procedure. **Title:** Technical Success: Successfully Facilitate Placement of a Guidewire Beyond a CTO Lesion in the True Vessel Lumen/Within the Collaterals **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** BridgePoint CTO System: * CrossBoss Catheter * Stingray LP Catheter * Stingray Guidewire and Extension Wire single-arm study: single-arm study **ID:** OG000 **Title:** CTO BridgePoint System ### Participant Flow Module #### Group **Description:** BridgePoint CTO System: * CrossBoss Catheter * Stingray LP Catheter * Stingray Guidewire and Extension Wire single-arm study: single-arm study **ID:** FG000 **Title:** CTO BridgePoint System #### Period **Title:** Overall Study ##### Withdraw **Type:** 8 of the 20 subjects already enrolled were unable to complete the the 12-month follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 8 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 20 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 12 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 8 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT05639179 **Brief Title:** Novel Anti-CD19 Universal CAR-T Cells for r/r CD19+ B-ALL **Official Title:** An Investigator-initiated Trial to Evaluate the Efficacy and Safety of Anti-CD19 Universal CAR-T Cells in the Treatment of Relapsed/Refractory(r/r) CD19+ B-cell Acute Lymphoblastic Leukemia(B-ALL) #### Organization Study ID Info **ID:** KM-010 #### Organization **Class:** OTHER **Full Name:** 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China ### Status Module #### Completion Date **Date:** 2025-12-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2022-12-08 **Type:** ACTUAL **Last Update Submit Date:** 2022-12-07 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-12-31 **Type:** ESTIMATED #### Start Date **Date:** 2022-12-05 **Type:** ACTUAL **Status Verified Date:** 2022-12 #### Study First Post Date **Date:** 2022-12-06 **Type:** ACTUAL **Study First Submit Date:** 2022-11-25 **Study First Submit QC Date:** 2022-11-25 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China #### 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 is a single-arm, single-center, open-labeled clinical study to evaluate the safety and efficacy of UCAR-T Cells injection for patients with relapsed/refractory(r/r) B-cell Acute Lymphoblastic Leukemia(B-ALL). **Detailed Description:** Although the anti-CD19 CAR-T cell therapies have gained significant clinical outcome in patients with r/r B-ALL，autologous CAR-T is not feasible for some patients. To make further improvement, the investigators are going to conduct a clinical trial using universal CAR-T(UCAR-T) cells targeting CD19 for r/r B-ALL patients. After enrollment, patients will get a 3-5 days lymphodepletion therapy, then the UCAR-T Cells will be infused by vein. Subjects will be followed for safety and efficacy up to 12 weeks. For those with a durable remission 12 weeks after infusion, the follow-up will last for at least 12 months for disease control. ### Conditions Module **Conditions:** - B-cell Acute Lymphoblastic Leukemia - B-ALL **Keywords:** - universal CAR-T(UCAR-T) - CD19 ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 30 **Type:** ESTIMATED **Phases:** - PHASE1 - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Subjects who meet the enrollment conditions will receive intravenous infusion of UCAR-T Cells after lymphodepletion. **Intervention Names:** - Biological: UCAR-T Cells **Label:** Assigned Interventions **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Assigned Interventions **Description:** UCAR-T Cellswill be administered by vein. The trial includes two portions. The first portion is a"3+3"dose escalation study, in which three dose groups are set:Dose level one:1×10\^6 cells/kg;Dose level two:2×10\^6 cells/kg;Dose level three:5×10\^6 cells/kg. Each dose group requires at least three subjects. The trial will start from dose level one. The second portion includes a dosage extended cohort and will start after the finish of the"3+3"dose escalation study. Twelve subjects will get infusion of UCAR-T Cells at the best dose verified in the first portion. **Name:** UCAR-T Cells **Type:** BIOLOGICAL ### Outcomes Module #### Primary Outcomes **Description:** Neurotoxicity and/or CRS≥G3. **Measure:** Dose-limiting toxicity (DLT) **Time Frame:** Up to 28 days after infusion **Description:** The frequency, severity, and laboratory findings of all adverse events/serious adverse events are included. **Measure:** Incidence of Treatment Related adverse events (AEs) **Time Frame:** Up to 12 months after infusion #### Secondary Outcomes **Description:** The persistence over time of CAR-T cells in the peripheral blood as determined by flow cytometry and qPCR. **Measure:** Persistence of CAR-T cells **Time Frame:** Up to 24 weeks after infusion **Description:** Patients who achieve CR(complete response) or CRi after infusion **Measure:** Objective response rate (ORR) **Time Frame:** At 4,8,12 weeks after infusion **Description:** Progression-free survival (PFS) is the time between the time a patient with tumor disease receives treatment and the time between the observation of disease progression or death from any cause. **Measure:** Progression-free survival (PFS) **Time Frame:** Up to 24 weeks after infusion **Description:** Overall survival (OS) is the time from randomization to death from any cause. **Measure:** Overall survival (OS) **Time Frame:** Up to 24 weeks after infusion **Description:** Duration of remission (DOR) is the time from the first detection of CR or PR to the discovery of PD. **Measure:** Duration of remission (DOR) **Time Frame:** Up to 24 weeks after infusion ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Male or female, aged 2-75 years; 2. A definite diagnosis of relapsed/refractory B-ALL and a percentage of primitive/naive lymphocytes \>5% in bone marrow at baseline (flow cytometry); 3. CD19 expression was positive in bone marrow or peripheral blood tumor cells; 4. ECOG score 0-2 points; 5. Expected survival time ≥3 months; 6. Adequate liver, kidney, heart and lung function; 7. Patients who have recovered from acute toxic effects of prior chemotherapy should be excluded from the trial at least one week apart; 8. Women of childbearing age have negative blood pregnancy test before the start of the trial, and agree to take effective contraceptive measures during the trial until the last follow-up; male subjects with partners of childbearing potential agree to take effective contraceptive measures during the trial until the last follow-up; 9. Voluntarily sign the informed consent. Exclusion Criteria: 1. Presence of other concurrent active malignancy; 2. People with severe mental disorders; 3. A history of any of the following genetic disorders, such as Fanconi anemia, Schu-Day syndrome, Gerstmann syndrome, or any other known bone marrow failure syndrome; 4. Acute GVHD of grade II-IV or extensive chronic GVHD; 5. Had grade III-IV heart failure or myocardial infarction, cardiac angioplasty or stenting, unstable angina pectoris, or other clinically prominent heart disease within one year prior to enrollment; 6. The presence of any indwelling catheter or drainage (e.g., percutaneous nephrostomy, indwelling catheter, bile drainage, or pleural/peritoneal/pericardial catheter), except for patients who are permitted to use dedicated central venous catheters; 7. A history or disease of the central nervous system(CNS), such as seizure disease, cerebrovascular ischemia/bleeding, dementia, cerebellar disease, or any autoimmune disease involving the CNS; 8. Human immunodeficiency virus (HIV) seropositivity; Hepatitis B surface antigen positive or hepatitis B core antibody positive, and HBV-DNA positive; Patients with hepatitis C (HCV-RNA quantitative test results positive); Or the presence of other serious active viral or bacterial infections or uncontrolled systemic fungal infections; 9. Patients with severe history of allergy or allergic constitution; 10. A history of autoimmune diseases (e.g., Crohn's disease, rheumatoid arthritis, systemic lupus erythematosus) leading to end-organ damage or requiring systemic immunosuppressive/systemic disease modulating drugs within the past 2 years; 11. Had or is suffering from interstitial lung disease (e.g., pneumonia, pulmonary fibrosis); 12. Had undergone other clinical trials in the 4 weeks prior to participating in this trial; 13. Poor compliance due to physiological, family, social, geographical and other factors, unable to cooperate with the study protocol and follow-up plan; 14. For patients contraindicated with cyclophosphamide and fludarabine chemotherapy; 15. Subjects requiring systemic corticosteroid therapy (prednisone ≥5mg/ day or equivalent dose of another corticosteroid) or other immunosuppressive agents within 1 month after UCAR-T cell reinfusion, except for adverse events; 16. Receiving donor lymphocyte infusion within 6 weeks before enrollment; 17. Pregnant and lactating women; 18. Any other condition that the investigator deemed inappropriate for inclusion. **Maximum Age:** 75 Years **Minimum Age:** 2 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Wang Sanbin, Doctor **Phone:** 13187424131 **Phone Ext:** +86 **Role:** CONTACT #### Locations **Location 1:** **City:** Kunming **Contacts:** ***Contact 1:*** - **Name:** Chen Li - **Phone:** 64774206 - **Phone Ext:** 0871 - **Role:** CONTACT **Country:** China **Facility:** 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China **State:** Yunnan **Status:** RECRUITING **Zip:** 650000 #### Overall Officials **Official 1:** **Affiliation:** 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China **Name:** Wang Sanbin, Doctor **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: 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: All - Name: All Conditions - Abbrev: BC20 - Name: Immune System Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M10945 - Name: Leukemia - Relevance: HIGH - As Found: Leukemia - ID: M10951 - Name: Leukemia, Lymphoid - Relevance: HIGH - As Found: Lymphoblastic Leukemia - ID: M27585 - Name: Precursor Cell Lymphoblastic Leukemia-Lymphoma - Relevance: HIGH - As Found: Acute Lymphoblastic Leukemia - ID: M11220 - Name: Lymphoma - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown - ID: M9490 - Name: Hematologic Diseases - 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: T175 - Name: Acute Lymphoblastic Leukemia - Relevance: HIGH - As Found: Acute Lymphoblastic Leukemia - ID: T3533 - Name: Lymphoblastic Lymphoma - Relevance: HIGH - As Found: Acute Lymphoblastic Leukemia - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown - ID: T3543 - Name: Lymphosarcoma - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000007938 - Term: Leukemia - ID: D000054198 - Term: Precursor Cell Lymphoblastic Leukemia-Lymphoma - ID: D000007945 - Term: Leukemia, Lymphoid ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01743079 **Brief Title:** Study of Telbivudine and Lamivudine to Prevent Vertical Transmission of Hepatitis B **Official Title:** The Efficacy and Safety of Telbivudine and Lamivudine Use in Highly Viremic Mothers to Prevent Hepatitis B Transmission #### Organization Study ID Info **ID:** 20080810 #### Organization **Class:** OTHER **Full Name:** Beijing YouAn Hospital ### Status Module #### Completion Date **Date:** 2013-07 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2013-07-30 **Type:** ESTIMATED **Last Update Submit Date:** 2013-07-29 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2013-07 **Type:** ACTUAL #### Start Date **Date:** 2009-01 **Status Verified Date:** 2013-07 #### Study First Post Date **Date:** 2012-12-06 **Type:** ESTIMATED **Study First Submit Date:** 2012-12-03 **Study First Submit QC Date:** 2012-12-04 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** New Discovery LLC #### Lead Sponsor **Class:** OTHER **Name:** Beijing YouAn Hospital #### Responsible Party **Investigator Affiliation:** Beijing YouAn Hospital **Investigator Full Name:** Hua Zhang **Investigator Title:** Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of this study is to evaluate the efficacy and safety of telbivudine and lamivudine use during late pregnancy for the prevention of HBV perinatal transmission in highly viraemic mothers. **Detailed Description:** This study enrolls HBV mono-infected pregnant women cohorts managed in outpatient clinics/delivery unit at YouAn Hospital in Beijing. Subjects are prospectively followed from gestation week 26 to postpartum week 52. Treatment naïve mothers with HBV DNA \> 6 log10 c/mL and normal ALT are eligible. Mothers with abnormal fetus, cirrhosis or evidence of hepatocellular carcinoma (HCC) are excluded. At gestation week 28, mothers will receive telbivudine (LdT) 600 mg per day or lamivudine (Lam) 100 mg per day until postpartum 4 or no treatment as per their preference. All infants will receive standard immunoprophylaxis. Data is collected from patient records using data extraction forms. Primary endpoints are vertical transmission rates at infants' age of 52 week and the safety of telbivudine or lamivudine use. ### Conditions Module **Conditions:** - Chronic Hepatitis B - Late Pregnancy - Transmission - Complication **Keywords:** - Telbivudine - Lamivudine - safety - efficacy - pregnancy ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 700 **Type:** ACTUAL **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Mother receives telbivudine 600mg per day. Infant receives standard immunoprophylaxis **Intervention Names:** - Drug: Telbivudine **Label:** Telbivudine **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Mother receives lamivudine 100mg per day. Infant receives standard immunoprophylaxis. **Intervention Names:** - Drug: Lamivudine **Label:** Lamivudine **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Mother receives no antiviral treatment. Infant receives standard immunoprophylaxis **Label:** No antiviral treatment **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Telbivudine **Description:** LdT 600mg QD **Name:** Telbivudine **Other Names:** - LdT **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Lamivudine **Description:** LAM 100mg QD **Name:** Lamivudine **Other Names:** - LAM **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Safety on fetal exposure of telbivudine and lamivudine and vertical transmission rate from mother to child **Time Frame:** From gestation week 26 to postpartum week 52 #### Secondary Outcomes **Measure:** percentage of mothers with serum HBV DNA level reduction, ALT within normal range and HBeAg and/or HBsAg negativity with or without seroconversion **Time Frame:** From gestation week 26 to pastpartume week 52 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Age: 20-40 years old 2. HBsAg, HBeAg positive and HBV DNA \>6 log10 copies/ml 3. Gestational age: 26-28 weeks with normal fetus 4. Willing to consent for the study Exclusion Criteria: 1. Elevated ALT 2. Antiviral treatment experience patients 3. Co-infection with HAV, HCV,HDV, HIV 4. Concurrent treatment with immune modulators, cytotoxic drugs, or steroids 5. Clinical signs of threatened miscarriage in early pregnancy 6. Clinical evidence of cirrhosis and/or hepatocellular carcinoma **Maximum Age:** 40 Years **Minimum Age:** 20 Years **Sex:** FEMALE **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Beijing **Country:** China **Facility:** Beijing YouAn Hospital **State:** Beijing **Zip:** 100069 #### Overall Officials **Official 1:** **Affiliation:** Division of Liver Diseases, Mount Sinai School of Medicine, Flushing, NY **Name:** Calvin Pan, MD **Role:** STUDY_DIRECTOR **Official 2:** **Affiliation:** Beijing YouAn Hospital **Name:** Hua Zhang, MD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Zhang H, Pan CQ, Pang Q, Tian R, Yan M, Liu X. Telbivudine or lamivudine use in late pregnancy safely reduces perinatal transmission of hepatitis B virus in real-life practice. Hepatology. 2014 Aug;60(2):468-76. doi: 10.1002/hep.27034. **PMID:** 25187919 ## 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: D000018347 - Term: Hepadnaviridae Infections - ID: D000004266 - Term: DNA Virus Infections - ID: D000006521 - Term: Hepatitis, Chronic - 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: M9595 - Name: Hepatitis B - Relevance: HIGH - As Found: Hepatitis B - ID: M21609 - Name: Hepatitis B, Chronic - Relevance: HIGH - As Found: Chronic Hepatitis B - ID: M9591 - Name: Hepatitis - Relevance: HIGH - As Found: Hepatitis - ID: M9607 - Name: Hepatitis, Chronic - Relevance: LOW - As Found: Unknown - 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: M20487 - Name: Hepadnaviridae Infections - Relevance: LOW - As Found: Unknown - ID: M7442 - Name: DNA Virus 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: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000006506 - Term: Hepatitis A - ID: D000006509 - Term: Hepatitis B - ID: D000019694 - Term: Hepatitis B, Chronic - ID: D000006505 - Term: Hepatitis ### Intervention Browse Module - Ancestors - ID: D000018894 - Term: Reverse Transcriptase Inhibitors - ID: D000019384 - Term: Nucleic Acid Synthesis Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000000998 - Term: Antiviral Agents - ID: D000000890 - Term: Anti-Infective Agents - ID: D000019380 - Term: Anti-HIV Agents - ID: D000044966 - Term: Anti-Retroviral 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: M21243 - Name: Lamivudine - Relevance: HIGH - As Found: Plane - ID: M4314 - Name: Antiviral Agents - Relevance: LOW - As Found: Unknown - ID: M1879 - Name: Telbivudine - Relevance: HIGH - As Found: 3 times per day - ID: M20935 - Name: Reverse Transcriptase Inhibitors - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown - ID: M4214 - Name: Anti-Infective Agents - Relevance: LOW - As Found: Unknown - ID: M21350 - Name: Anti-HIV Agents - Relevance: LOW - As Found: Unknown - ID: M25428 - Name: Anti-Retroviral Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000019259 - Term: Lamivudine - ID: D000077712 - Term: Telbivudine ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02224079 **Brief Title:** Safety, Tolerability and Pharmacokinetics of Single Rising Doses of BIIB 722 CL and HPβCD in Young Healthy Male Volunteers **Official Title:** Safety, Tolerability and Preliminary Pharmacokinetics of Single Rising Doses of 1 mg, 2.5 mg, 5 mg, 10 mg, 15 mg, 20 mg, 25 mg, 37.5 mg, 50 mg, 75 mg, 100 mg, 125 mg and 150 mg BIIB 722 CL (Calculated as 'Free Base') and HPβCD Given as Intravenous Infusion Over 30 Minutes to Young Healthy Male Subjects. A Single-centre, Single-blind, Placebo-controlled, Randomised Study. #### Organization Study ID Info **ID:** 1180.3 #### Organization **Class:** INDUSTRY **Full Name:** Boehringer Ingelheim ### Status Module #### Expanded Access Info #### Last Update Post Date **Date:** 2014-08-25 **Type:** ESTIMATED **Last Update Submit Date:** 2014-08-21 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2002-06 **Type:** ACTUAL #### Start Date **Date:** 2002-04 **Status Verified Date:** 2014-08 #### Study First Post Date **Date:** 2014-08-25 **Type:** ESTIMATED **Study First Submit Date:** 2014-08-21 **Study First Submit QC Date:** 2014-08-21 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Boehringer Ingelheim #### Responsible Party **Type:** SPONSOR ### Description Module **Brief Summary:** Study to assess safety, tolerability and pharmacokinetics (PK) of single intravenous (i.v.) doses of BIIB 722 CL ### Conditions Module **Conditions:** - Healthy ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 100 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Drug: BIIB 722 CL - Drug: Placebo **Label:** BIIB 722 CL **Type:** EXPERIMENTAL #### Arm Group 2 **Intervention Names:** - Drug: Placebo **Label:** Placebo **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - BIIB 722 CL **Name:** BIIB 722 CL **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - BIIB 722 CL - Placebo **Description:** Hydroxypropyl-beta-cyclodextrin (HPβCD) **Name:** Placebo **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Number of subjects with adverse events **Time Frame:** up to 12 days after drug administration **Description:** blood pressure, pulse rate, respiratory rate, oral body temperature **Measure:** Number of subjects with clinically significant findings in vital signs **Time Frame:** up to 12 days after drug administration **Measure:** Number of subjects with clinically significant findings in ECG **Time Frame:** up to 12 days after drug administration **Measure:** Number of subjects with clinically significant findings in laboratory tests **Time Frame:** up to 12 days after drug administration #### Secondary Outcomes **Measure:** Plasma concentration time profiles **Time Frame:** up to 96 hours after drug administration **Measure:** Maximum measured concentration of the analyte in plasma (Cmax) **Time Frame:** up to 96 hours after drug administration **Measure:** Time from dosing to the maximum concentration of the analyte in plasma (tmax) **Time Frame:** up to 96 hours after drug administration **Measure:** Area under the concentration-time curve of the analyte in plasma from time zero to a specified point in time (AUC0-t) **Time Frame:** up to 96 hours after drug administration **Measure:** Terminal half-life of the analyte in plasma (t1/2) **Time Frame:** up to 96 hours after drug administration **Measure:** Mean residence time of the analyte in the body (MRT) **Time Frame:** up to 96 hours after drug administration **Measure:** Total clearance of the analyte in plasma (CL) **Time Frame:** up to 96 hours after drug administration **Measure:** Apparent volume of distribution at steady state (Vss) **Time Frame:** up to 96 hours after drug administration **Measure:** Amount of drug excreted into urine (Ae) **Time Frame:** up to 72 hours after drug administration ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Healthy males * 21 to 50 years of age * Broca index \>= -20% and \<= +20% * Written informed consent according to Good Clinical Practice (GCP) and local legislation Exclusion Criteria: * Any finding of the medical examination (including blood pressure, pulse rate and Electrocardiogram (ECG)) deviating from normal and of clinical relevance * History or current gastrointestinal, hepatic, renal, respiratory, cardiovascular, metabolic, immunologic or hormonal disorders * Diseases of the central nervous system or psychiatric disorders or neurological disorders * History of orthostatic hypotension, fainting spells or blackouts * Chronic or relevant acute infections * History of allergy/hypersensitivity (including drug allergy) which is deemed relevant to the trial as judged by the investigator * Intake of drugs with a long half-life (\> 24 hours) within at least one month or less than ten half-lives of the respective drug before enrolment in the study * Use of any drugs, which might influence the results of the trial within two weeks prior to administration or during the trial * Participation in another trial with an investigational drug (\<= two months prior to administration or during trial) * Smoker (\> 10 cigarettes or \> 3 cigars or \> 3 pipes/day) * Inability to refrain from smoking on study days * Alcohol abuse (\> 60 g/day) * Drug abuse * Blood donation (\>= 100 mL within four weeks prior to administration or during the trial) * Excessive physical activities (within the last week before the study) * Any laboratory value outside the reference range of clinical relevance **Healthy Volunteers:** True **Maximum Age:** 50 Years **Minimum Age:** 21 Years **Sex:** MALE **Standard Ages:** - ADULT ## Derived Section ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M107438 - Name: Betadex - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00383279 **Brief Title:** Improving Employment in Patients Who Have Survived Cervical Cancer, Uterine Cancer, or Ovarian Cancer **Official Title:** Improving Employment Among Gynecologic Cancer Survivors #### Organization Study ID Info **ID:** CCCWFU-99106 #### Organization **Class:** OTHER **Full Name:** Wake Forest University Health Sciences #### Secondary ID Infos **ID:** CCCWFU-99106 **ID:** CCCWFU-BG06-014 ### Status Module #### Completion Date **Date:** 2007-02 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-08-09 **Type:** ACTUAL **Last Update Submit Date:** 2018-08-07 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2007-02 **Type:** ACTUAL #### Start Date **Date:** 2006-02 **Type:** ACTUAL **Status Verified Date:** 2018-08 #### Study First Post Date **Date:** 2006-10-03 **Type:** ESTIMATED **Study First Submit Date:** 2006-09-29 **Study First Submit QC Date:** 2006-09-29 ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** National Cancer Institute (NCI) #### 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:** RATIONALE: Finding out which problems affect employment in survivors of gynecologic cancer may help in planning cancer treatment and improve the quality of life of future cancer survivors. PURPOSE: This research study is looking at ways to improve employment in patients who have survived cervical cancer, uterine cancer, or ovarian cancer. **Detailed Description:** OBJECTIVES: * Conduct formative work, including 4 to 5 focus groups, leading to development of a structured interview instrument assessing factors related to employment outcomes among women with gynecologic cancers. * Pilot a recruitment strategy and administer the interview among a sample of 60 to 70 gynecologic cancer survivors; and, with each woman's permission, a family member, a key health care provider, and her supervisor will be interviewed. OUTLINE: This is a multicenter, pilot study. Patients complete a survey, meet with a focus group, and undergo a short interview. Patient-designated participants are also interviewed; these individuals may include a health care provider, a significant other, and/or a work supervisor. PROJECTED ACCRUAL: A total of 70 patients will be accrued for this study. ### Conditions Module **Conditions:** - Cervical Cancer - Ovarian Cancer - Sarcoma **Keywords:** - recurrent cervical cancer - stage 0 cervical cancer - stage IA cervical cancer - stage IB cervical cancer - stage IIA cervical cancer - stage IIB cervical cancer - stage III cervical cancer - stage IVA cervical cancer - stage IVB cervical cancer - recurrent uterine sarcoma - stage I uterine sarcoma - stage II uterine sarcoma - stage III uterine sarcoma - stage IV uterine sarcoma - borderline ovarian surface epithelial-stromal tumor - ovarian sarcoma - ovarian stromal cancer - recurrent ovarian epithelial cancer - recurrent ovarian germ cell tumor - stage I ovarian epithelial cancer - stage I ovarian germ cell tumor - stage II ovarian epithelial cancer - stage II ovarian germ cell tumor - stage III ovarian epithelial cancer - stage III ovarian germ cell tumor - stage IV ovarian epithelial cancer - stage IV ovarian germ cell tumor ### Design Module #### Design Info **Primary Purpose:** OTHER #### Enrollment Info **Count:** 70 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Name:** counseling intervention **Type:** OTHER #### Intervention 2 **Name:** study of socioeconomic and demographic variables **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Measure:** Development of a structured interview instrument **Measure:** Recruitment strategy ### Eligibility Module **Eligibility Criteria:** DISEASE CHARACTERISTICS: * Diagnosis of 1 of the following invasive cancers: * Cervical cancer * Uterine cancer * Ovarian cancer * Diagnosed ≥ 6 months ago * Any stage disease * Employed at the time of diagnosis, defined as working for pay ≥ 20 hours/week PATIENT CHARACTERISTICS: * Not specified PRIOR CONCURRENT THERAPY: * Not specified **Maximum Age:** 120 Years **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Winston-Salem **Country:** United States **Facility:** Wake Forest University Comprehensive Cancer Center **State:** North Carolina **Zip:** 27157-1096 #### Overall Officials **Official 1:** **Affiliation:** Wake Forest University Health Sciences **Name:** Brigitte E. Miller, MD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000004701 - Term: Endocrine Gland Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000010049 - Term: Ovarian Diseases - ID: D000000291 - Term: Adnexal 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: D000005833 - Term: Genital Neoplasms, Female - ID: D000014565 - Term: Urogenital Neoplasms - ID: D000091662 - Term: Genital Diseases - ID: D000004700 - Term: Endocrine System Diseases - ID: D000006058 - Term: Gonadal Disorders - ID: D000002277 - Term: Carcinoma - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000018204 - Term: Neoplasms, Connective and Soft Tissue - ID: D000014594 - Term: Uterine Neoplasms - ID: D000002577 - Term: Uterine Cervical Diseases - ID: D000014591 - Term: Uterine Diseases ### 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 - Abbrev: BC19 - Name: Gland and Hormone Related Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M5830 - Name: Uterine Cervical Neoplasms - Relevance: HIGH - As Found: Cervical Cancer - ID: M15327 - Name: Sarcoma - Relevance: HIGH - As Found: Sarcoma - ID: M14850 - Name: Recurrence - Relevance: LOW - As Found: Unknown - ID: M12974 - Name: Ovarian Neoplasms - Relevance: HIGH - As Found: Ovarian Cancer - ID: M1704 - Name: Carcinoma, Ovarian Epithelial - Relevance: HIGH - As Found: Ovarian Cancer - ID: M17342 - Name: Uterine Neoplasms - Relevance: LOW - As Found: Unknown - ID: M5225 - Name: Brenner Tumor - Relevance: LOW - As Found: Unknown - ID: M5534 - Name: Carcinoma - Relevance: LOW - As Found: Unknown - ID: M7863 - Name: Endocrine Gland Neoplasms - Relevance: LOW - As Found: Unknown - ID: M12972 - Name: Ovarian Diseases - Relevance: LOW - As Found: Unknown - ID: M3643 - Name: Adnexal 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: M8945 - Name: Genital Neoplasms, Female - Relevance: LOW - As Found: Unknown - ID: M17315 - Name: Urogenital Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7862 - Name: Endocrine System Diseases - Relevance: LOW - As Found: Unknown - ID: M9163 - Name: Gonadal Disorders - 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: M20350 - Name: Neoplasms, Connective and Soft Tissue - Relevance: LOW - As Found: Unknown - ID: M5825 - Name: Uterine Cervical Diseases - Relevance: LOW - As Found: Unknown - ID: M17339 - Name: Uterine Diseases - Relevance: LOW - As Found: Unknown - ID: T5284 - Name: Soft Tissue Sarcoma - Relevance: LOW - As Found: Unknown - ID: T2475 - Name: Germ Cells Tumors - Relevance: LOW - As Found: Unknown - ID: T4352 - Name: Ovarian Cancer - Relevance: HIGH - As Found: Ovarian Cancer - ID: T4354 - Name: Ovarian Epithelial Cancer - Relevance: HIGH - As Found: Ovarian Cancer - ID: T4355 - Name: Ovarian Germ Cell Tumor - Relevance: LOW - As Found: Unknown - ID: T5821 - Name: Uterine Sarcoma - Relevance: LOW - As Found: Unknown - ID: T4357 - Name: Ovarian Low Malignant Potential Tumor - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000010051 - Term: Ovarian Neoplasms - ID: D000077216 - Term: Carcinoma, Ovarian Epithelial - ID: D000012509 - Term: Sarcoma - ID: D000002583 - Term: Uterine Cervical Neoplasms ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00613379 **Brief Title:** PRO 140 by IV Administration in Adults With HIV-1 Infection **Official Title:** A Phase 2a, Randomized, Double-Blind, Placebo-Controlled Study of PRO 140 by Intravenous Administration in Adult Subjects With Human Immunodeficiency Virus Type 1 Infection #### Organization Study ID Info **ID:** PRO 140 2301 #### Organization **Class:** INDUSTRY **Full Name:** CytoDyn, Inc. #### Secondary ID Infos **ID:** 1U19AI066329 **Link:** https://reporter.nih.gov/quickSearch/1U19AI066329 **Type:** NIH ### Status Module #### Completion Date **Date:** 2008-08 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2016-07-15 **Type:** ESTIMATED **Last Update Submit Date:** 2016-06-16 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2008-08 **Type:** ACTUAL #### Results First Post Date **Date:** 2013-05-31 **Type:** ESTIMATED **Results First Submit Date:** 2013-04-12 **Results First Submit QC Date:** 2013-04-12 #### Start Date **Date:** 2007-12 **Status Verified Date:** 2016-06 #### Study First Post Date **Date:** 2008-02-13 **Type:** ESTIMATED **Study First Submit Date:** 2008-01-30 **Study First Submit QC Date:** 2008-02-12 ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** National Institute of Allergy and Infectious Diseases (NIAID) #### Lead Sponsor **Class:** INDUSTRY **Name:** CytoDyn, Inc. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is: 1. To assess and characterize the PK and PD of PRO 140 administered IV 2. To assess the antiviral activity of PRO 140 3. To assess the safety and tolerability of PRO 140 ### Conditions Module **Conditions:** - HIV Infections **Keywords:** - HIV - treatment naïve ### 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:** 30 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 10 mg/kg PRO 140, one IV dose (N=10) **Intervention Names:** - Drug: PRO 140 **Label:** Arm 1 **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** 5 mg/kg PRO 140, one IV dose (N=10) **Intervention Names:** - Drug: PRO 140 **Label:** Arm 2 **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Placebo, one IV dose (N=10) **Intervention Names:** - Drug: Placebo **Label:** Arm 3 **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Arm 1 **Description:** 10 mg/kg PRO 140, one IV dose (N=10) **Name:** PRO 140 **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Arm 2 **Description:** 5 mg/kg PRO 140, one IV dose (N=10) **Name:** PRO 140 **Type:** DRUG #### Intervention 3 **Arm Group Labels:** - Arm 3 **Description:** PBO, one IV dose (N=10) **Name:** Placebo **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** The primary end point was the maximum change from baseline in viral load following initiation of treatment, defined as HIV-1 copies/mL, measured by the Roche Amplicor HIV-1 Monitor UltraSensitive™ Test (lower limit of detection \[LLD\] = 48 copies/mL). **Measure:** Maximum Change in Viral Load Following Initiation of Treatment. **Time Frame:** 59 days ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Males \& females, age ≥ 18 years (or minimum adult age as determined by local regulatory authorities) 2. Screening plasma HIV-1 RNA ≥ 5,000 copies/mL 3. CD4+ T-lymphocyte cell count ≥ 300 cells/mm3 and no documented count \< or = 250 cells/mm3 4. Has not taken any anti-retroviral therapy (ART) w/in 12 wks of Early Screening Visit 5. Exclusive CCR5-tropic virus as determined by Trofile™ Assay at Early Screening Visit 6. Clinically normal or "not clinically significant (NCS)" resting electrocardiogram 7. Women of reproductive potential must have a negative serum pregnancy test at Late Screening Visit \& a negative urine pregnancy test w/in 72 hrs prior to first dose of study medication, \& be non-lactating. Male \& female subjects must agree not to participate in a conception process from Early Screening Visit through Day 59. Exclusion Criteria: 1. CXCR4 tropic virus or dual/mixed tropic (R5X4) virus determined by the Trofile™ Assay. 2. Females who are pregnant, lactating or breastfeeding, or who plan to become pregnant during the study. 3. History of active hepatitis within the previous 24 wks 4. Prior use of any entry, attachment, CCR5 co-receptor or fusion inhibitor, experimental or approved. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Tarrytown **Country:** United States **Facility:** Progenics Pharmaceuticals, Inc **State:** New York **Zip:** 10591 #### Overall Officials **Official 1:** **Affiliation:** Progenics Pharmaceuticals, Inc. **Name:** Stephen Morris, MD, PhD **Role:** STUDY_DIRECTOR ### References Module #### See Also Links **Label:** Former Sponsor website **URL:** http://www.progenics.com **Label:** Sponsor Website **URL:** http://www.cytodyn.com/ ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020969 - Term: Disease Attributes - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC01 - Name: Infections - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: BC20 - Name: Immune System Diseases ### Condition Browse Module - Browse Leaves - ID: M10283 - Name: Infections - Relevance: HIGH - As Found: Infection - ID: M6368 - Name: Communicable Diseases - Relevance: HIGH - As Found: Infection - ID: M3522 - Name: Acquired Immunodeficiency Syndrome - Relevance: LOW - As Found: Unknown - ID: M18250 - Name: HIV Infections - Relevance: LOW - As Found: Unknown - ID: M17522 - Name: Virus Diseases - Relevance: LOW - As Found: Unknown - ID: M10199 - Name: Immunologic Deficiency Syndromes - Relevance: LOW - As Found: Unknown - ID: M22700 - Name: Disease Attributes - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000007239 - Term: Infections - ID: D000003141 - Term: Communicable Diseases ### Intervention Browse Module - Ancestors - ID: D000023581 - Term: HIV Fusion Inhibitors - ID: D000065147 - Term: Viral Fusion Protein Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000019380 - Term: Anti-HIV Agents - ID: D000044966 - Term: Anti-Retroviral Agents - ID: D000000998 - Term: Antiviral Agents - ID: D000000890 - Term: Anti-Infective Agents - ID: D000007155 - Term: Immunologic Factors - ID: D000045505 - Term: Physiological Effects of Drugs ### Intervention Browse Module - Browse Branches - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M289514 - Name: Leronlimab - Relevance: HIGH - As Found: Cutaneous T-Cell Lymphoma - ID: M4225 - Name: Antibodies - Relevance: LOW - As Found: Unknown - ID: M10184 - Name: Immunoglobulins - Relevance: LOW - As Found: Unknown - ID: M18138 - Name: HIV Antibodies - Relevance: HIGH - As Found: Cutaneous T-Cell Lymphoma - ID: M21350 - Name: Anti-HIV Agents - Relevance: LOW - As Found: Unknown - ID: M25428 - Name: Anti-Retroviral Agents - Relevance: LOW - As Found: Unknown - ID: M4314 - Name: Antiviral Agents - Relevance: LOW - As Found: Unknown - ID: M4214 - Name: Anti-Infective Agents - Relevance: LOW - As Found: Unknown - ID: M10201 - Name: Immunologic Factors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: C000420063 - Term: Leronlimab - ID: D000015483 - Term: HIV Antibodies ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Arm 1 **Description:** 10 mg/kg PRO 140, one IV dose (N=10) **ID:** EG000 **Other Num Affected:** 8 **Other Num at Risk:** 10 **Serious Number At Risk:** 10 **Title:** Arm 1 **Group ID:** EG001 **Title:** Arm 2 **Description:** 5 mg/kg PRO 140, one IV dose (N=10) **ID:** EG001 **Other Num Affected:** 7 **Other Num at Risk:** 10 **Serious Number At Risk:** 10 **Title:** Arm 2 **Group ID:** EG002 **Title:** Arm 3 **Description:** PBO, one IV dose (N=10) **ID:** EG002 **Other Num Affected:** 11 **Other Num at Risk:** 11 **Serious Number At Risk:** 11 **Title:** Arm 3 **Frequency Threshold:** 5 #### Other Events **Term:** Lymphadenopathy **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Bundle branch block left **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Lacrimation increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Retinal haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Vision blurred **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Anorectal discomfort **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Dental caries **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Diarrhoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Dry mouth **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Gastrooesophageal reflux disease **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Haemorrhoids **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Mouth ulceration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Nausea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Catheter site haematoma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Chest discomfort **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Chills **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Fatigue **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Feeling hot **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Malaise **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Oedema peripheral **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Bronchiectasis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (11.0) **Term:** Bronchitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (11.0) **Term:** Candidiasis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (11.0) **Term:** Hepatitis B **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (11.0) **Term:** Hordeolum **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (11.0) **Term:** Oral hairy leukoplakia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (11.0) **Term:** Upper respiratory tract infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (11.0) **Term:** Arthropod bite **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (11.0) **Term:** Back injury **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (11.0) **Term:** Contusion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (11.0) **Term:** Skin laceration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (11.0) **Term:** Sunburn **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (11.0) **Term:** Blood amylase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (11.0) **Term:** Blood bicarbonate decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (11.0) **Term:** Blood glucose increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (11.0) **Term:** Blood lactate dehydrogenase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (11.0) **Term:** Echocardiogram normal **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (11.0) **Term:** Electrocardiogram ST segment elevation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (11.0) **Term:** Electrocardiogram abnormal **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (11.0) **Term:** Lipase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (11.0) **Term:** Neutrophil count decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (11.0) **Term:** QRS axis abnormal **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (11.0) **Term:** Syphilis test positive **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (11.0) **Term:** Weight increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (11.0) **Term:** White blood cell count decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (11.0) **Term:** Anorexia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Obesity **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Arthralgia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Arthropathy **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Back pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Joint crepitation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Musculoskeletal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Myalgia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Dizziness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Hypersomnia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Paraesthesia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Anxiety **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Depression **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Insomnia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Proteinuria **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Erectile dysfunction **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Reproductive system and breast disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Gynaecomastia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Reproductive system and breast disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Dyspnoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Nasal congestion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Pharyngeal erythema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Pharyngolaryngeal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Productive cough **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Upper respiratory tract congestion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Erythema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Night sweats **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Pruritus **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Rash **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Skin exfoliation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA (11.0) **Term:** Physical assault **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Social circumstances **Source Vocabulary:** MedDRA (11.0) ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 10 **Group ID:** BG001 **Value:** 10 **Group ID:** BG002 **Value:** 11 **Group ID:** BG003 **Value:** 31 **Units:** Participants ### Group **ID:** BG000 **Title:** Arm 1 **Description:** 10 mg/kg PRO 140, one IV dose (N=10) ### Group **ID:** BG001 **Title:** Arm 2 **Description:** 5 mg/kg PRO 140, one IV dose (N=10) ### Group **ID:** BG002 **Title:** Arm 3 **Description:** PBO, one IV dose (N=10) ### 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:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 0 **Category Title:** <=18 years #### Measurement **Group ID:** BG000 **Value:** 10 #### Measurement **Group ID:** BG001 **Value:** 10 #### Measurement **Group ID:** BG002 **Value:** 11 #### Measurement **Group ID:** BG003 **Value:** 31 **Category Title:** Between 18 and 65 years #### 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:** >=65 years **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 10.17 **Value:** 42.5 #### Measurement **Group ID:** BG001 **Spread:** 9.88 **Value:** 42 #### Measurement **Group ID:** BG002 **Spread:** 11.65 **Value:** 38 #### Measurement **Group ID:** BG003 **Spread:** 10.48 **Value:** 40.7 **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:** Female #### Measurement **Group ID:** BG000 **Value:** 10 #### Measurement **Group ID:** BG001 **Value:** 10 #### Measurement **Group ID:** BG002 **Value:** 11 #### Measurement **Group ID:** BG003 **Value:** 31 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 10 #### Measurement **Group ID:** BG001 **Value:** 10 #### Measurement **Group ID:** BG002 **Value:** 11 #### Measurement **Group ID:** BG003 **Value:** 31 **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 ## Results Section - More Information Module ### Certain Agreement **Other Details:** Because the study is a multicenter study, the PI(s) can only publish after the aggregate results of all investigators and institutions participating in the study have been published, and the proposed publication is reviewed by Progenics. In the event that Progenics does not publish the results of the study within eighteen months from availability of final study analysis, the PI can publish provided that the proposed publication is reviewed by Progenics. **Restriction Type:** OTHER **Restrictive Agreement:** True ### Point of Contact **Email:** [email protected] **Organization:** Cytodyn, Inc. **Phone:** 360-980-8524 **Title:** Dr. Nader Pourhassan ## Results Section - Outcome Measures Module ### Outcome Measure 1 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.639 - **Upper Limit:** - **Value:** -1.67 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.229 - **Upper Limit:** - **Value:** -1.83 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** 0.244 - **Upper Limit:** - **Value:** -0.32 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** The primary end point was the maximum change from baseline in viral load following initiation of treatment, defined as HIV-1 copies/mL, measured by the Roche Amplicor HIV-1 Monitor UltraSensitive™ Test (lower limit of detection \[LLD\] = 48 copies/mL). **Dispersion Type:** Standard Error **Parameter Type:** MEAN **Population Description:** All randomized subjects who received one dose of study drug were considered intent-to-treat (ITT) subjects and were analyzed for efficacy. **Reporting Status:** POSTED **Time Frame:** 59 days **Title:** Maximum Change in Viral Load Following Initiation of Treatment. **Type:** PRIMARY **Unit of Measure:** Log10copies HIV-1 RNA/mL ##### Group **Description:** 10 mg/kg PRO 140, one IV dose (N=10) **ID:** OG000 **Title:** Arm 1 ##### Group **Description:** 5 mg/kg PRO 140, one IV dose (N=10) **ID:** OG001 **Title:** Arm 2 ##### Group **Description:** PBO, one IV dose (N=10) **ID:** OG002 **Title:** Arm 3 ### Participant Flow Module #### Group **Description:** 10 mg/kg PRO 140, one IV dose (N=10) **ID:** FG000 **Title:** Arm 1 #### Group **Description:** 5 mg/kg PRO 140, one IV dose (N=10) **ID:** FG001 **Title:** Arm 2 #### Group **Description:** PBO, one IV dose (N=10) **ID:** FG002 **Title:** Arm 3 #### Period **Title:** Overall Study ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 10 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 10 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 11 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 10 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 10 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 11 ##### 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 **Pre-Assignment Details:** Subjects screened up to 12 weeks **Recruitment Details:** Recruitment was from December 2007 to August 2008 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT00801879 **Brief Title:** Mupirocin Ointment to Eliminate Nasal Carriage of Staphylococcus Aureus in HIV Infection **Official Title:** Intranasal Mupirocin to Eliminate Carriage of Staphylococcus Aureus in HIV Infection #### Organization Study ID Info **ID:** AAAB0129 #### Organization **Class:** OTHER **Full Name:** Columbia University #### Secondary ID Infos **ID:** K08AI072043 **Link:** https://reporter.nih.gov/quickSearch/K08AI072043 **Type:** NIH ### Status Module #### Completion Date **Date:** 2006-04 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2014-10-29 **Type:** ESTIMATED **Last Update Submit Date:** 2014-10-28 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2006-04 **Type:** ACTUAL #### Start Date **Date:** 2003-09 **Status Verified Date:** 2014-10 #### Study First Post Date **Date:** 2008-12-04 **Type:** ESTIMATED **Study First Submit Date:** 2008-12-03 **Study First Submit QC Date:** 2008-12-03 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** GlaxoSmithKline **Class:** NIH **Name:** National Institute of Allergy and Infectious Diseases (NIAID) #### Lead Sponsor **Class:** OTHER **Name:** Columbia University #### Responsible Party **Investigator Affiliation:** Columbia University **Investigator Full Name:** Franklin D. Lowy **Investigator Title:** Professor of Medicine and Pathology at the New York-Presbyterian Hospital at the Columbia University Medical Center **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Staphylococcus aureus is a bacteria that causes serious, often life threatening infections including pneumonia, wound, and bloodstream infections. Persons with AIDS are at high risk for S. aureus infections. They are also at high risk for nasal carriage of S. aureus. In fact, nasal carriage is a known risk factor for subsequent S. aureus infection. Topical mupirocin, an antibiotic when applied to the anterior nares, is a safe, effective way to eliminate S. aureus colonization. Some studies have shown that mupirocin can also decrease the risk of S. aureus infection, but many of those studies utilized historical controls and none were rigorously tested among AIDS patients over an extended period of time. The main purpose of this randomized, double-blinded, placebo controlled study is to determine if mupirocin can eliminate S. aureus nasal colonization in residents at PSI (inpatient, drug rehabilitation facility for AIDS patients in the Bronx.) PSI residents currently have a high incidence of S. aureus nasal colonization and infection. Nasal cultures followed by twice daily application of mupirocin vs. placebo for five days will be performed on a monthly basis for 8 months. the study will examine whether mupirocin decreases the incidence of S. aureus infections and prevents S. aureus nasal colonization. The study is important because it may show that mupirocin is an effective way to eliminate nasal colonization and prevent S. aureus infections in AIDS patients, among those at highest risk for serious S. aureus infection. Hypothesis: Monthly application of mupirocin will reduce nasal colonization with S.aureus **Detailed Description:** This was a randomized double-blinded placebo controlled trial. Subjects were treated with intranasal mupirocin or placebo on a monthly basis and tested for nasal colonization a month after each treatment. This was performed up to 8 months. ### Conditions Module **Conditions:** - Staphylococcus Aureus - HIV Infections **Keywords:** - mupirocin - Staphylococcus aureus - colonization - infection - HIV ### 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:** 100 **Type:** ACTUAL **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 0.25g mupirocin calcium ointment, 2% in each nostril twice daily for 5 days (repeated monthly for up to 8 months) **Intervention Names:** - Drug: Mupirocin calcium ointment, 2% **Label:** Mupirocin ointment **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** 0.25g in each nostril twice daily for 5 days (repeated monthly for up to 8 months) **Intervention Names:** - Drug: Placebo ointment **Label:** Placebo ointment **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Mupirocin ointment **Description:** 0.25 g in each nostril twice daily for 5 days (given monthly for up to 8 months) **Name:** Mupirocin calcium ointment, 2% **Other Names:** - Bactroban Nasal 2% **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Placebo ointment **Description:** Placebo ointment 0.25g in each nostril twice daily for 5 days (repeated monthly for up to 8 months) **Name:** Placebo ointment **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Nasal colonization with Staphylococcus aureus **Time Frame:** monthly assessment of colonization (~1 month after each treatment) #### Secondary Outcomes **Measure:** Infection with Staphylococcus aureus **Time Frame:** monthly ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Resident at Project Samaritan Inc. (PSI) Exclusion Criteria: * Past hypersensitivity to mupirocin or glycerol * Pregnancy * Lactation * Expected discharge from PSI in the following month * Treatment with intranasal mupirocin within the preceding two months **Healthy Volunteers:** True **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Bronx **Country:** United States **Facility:** Project Samaritan Inc. **State:** New York **Zip:** 10452 **Location 2:** **City:** New York **Country:** United States **Facility:** Columbia University **State:** New York **Zip:** 10032 #### Overall Officials **Official 1:** **Affiliation:** Columbia University **Name:** Rachel J Gordon, MD, MPH **Role:** STUDY_DIRECTOR **Official 2:** **Affiliation:** Columbia University **Name:** Franklin D Lowy, MD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Gordon RJ, Chez N, Jia H, Zeller B, Sobieszczyk M, Brennan C, Hisert KB, Lee MH, Vavagiakis P, Lowy FD. The NOSE study (nasal ointment for Staphylococcus aureus eradication): a randomized controlled trial of monthly mupirocin in HIV-infected individuals. J Acquir Immune Defic Syndr. 2010 Dec;55(4):466-72. doi: 10.1097/QAI.0b013e3181ec2a68. **PMID:** 20686410 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020969 - Term: Disease Attributes - ID: D000010335 - Term: Pathologic Processes - ID: D000086982 - Term: Blood-Borne Infections - ID: D000015229 - Term: Sexually Transmitted Diseases, Viral - ID: D000012749 - Term: Sexually Transmitted Diseases - ID: D000016180 - Term: Lentivirus Infections - ID: D000012192 - Term: Retroviridae Infections - ID: D000012327 - Term: RNA Virus Infections - ID: D000014777 - Term: Virus Diseases - ID: D000091662 - Term: Genital Diseases - ID: D000091642 - Term: Urogenital Diseases - ID: D000007153 - Term: Immunologic Deficiency Syndromes - ID: D000007154 - Term: Immune System Diseases - ID: D000012897 - Term: Slow Virus Diseases - ID: D000016908 - Term: Gram-Positive Bacterial Infections - ID: D000001424 - Term: Bacterial Infections - ID: D000001423 - Term: Bacterial Infections and Mycoses ### Condition Browse Module - Browse Branches - Abbrev: BC01 - Name: Infections - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: BC20 - Name: Immune System Diseases ### Condition Browse Module - Browse Leaves - ID: M10283 - Name: Infections - Relevance: HIGH - As Found: Infection - ID: M6368 - Name: Communicable Diseases - Relevance: HIGH - As Found: Infection - ID: M3522 - Name: Acquired Immunodeficiency Syndrome - Relevance: HIGH - As Found: HIV Infections - ID: M18250 - Name: HIV Infections - Relevance: HIGH - As Found: HIV Infections - ID: M16355 - Name: Syndrome - Relevance: LOW - As Found: Unknown - ID: M10199 - Name: Immunologic Deficiency Syndromes - Relevance: LOW - As Found: Unknown - ID: M15996 - Name: Staphylococcal Infections - Relevance: HIGH - As Found: Staphylococcus Aureus - ID: M22700 - Name: Disease Attributes - Relevance: LOW - As Found: Unknown - ID: M2593 - Name: Blood-Borne Infections - Relevance: LOW - As Found: Unknown - ID: M15558 - Name: Sexually Transmitted Diseases - Relevance: LOW - As Found: Unknown - ID: M17933 - Name: Sexually Transmitted Diseases, Viral - Relevance: LOW - As Found: Unknown - ID: M18640 - Name: Lentivirus Infections - Relevance: LOW - As Found: Unknown - ID: M15026 - Name: Retroviridae 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: M2876 - Name: Genital Diseases - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M10200 - Name: Immune System Diseases - Relevance: LOW - As Found: Unknown - ID: M15700 - Name: Slow Virus Diseases - Relevance: LOW - As Found: Unknown - ID: M4722 - Name: Bacterial Infections - Relevance: LOW - As Found: Unknown - ID: M19252 - Name: Gram-Positive 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 ### Condition Browse Module - Meshes - ID: D000007239 - Term: Infections - ID: D000003141 - Term: Communicable Diseases - ID: D000015658 - Term: HIV Infections - ID: D000000163 - Term: Acquired Immunodeficiency Syndrome - ID: D000013203 - Term: Staphylococcal Infections ### Intervention Browse Module - Ancestors - ID: D000077264 - Term: Calcium-Regulating Hormones and Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000000900 - Term: Anti-Bacterial Agents - ID: D000000890 - Term: Anti-Infective Agents - ID: D000011500 - Term: Protein Synthesis Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: BDCA - Name: Bone Density Conservation Agents - Abbrev: Infe - Name: Anti-Infective Agents ### Intervention Browse Module - Browse Leaves - ID: M5381 - Name: Calcium - Relevance: HIGH - As Found: Radiation - ID: M5398 - Name: Calcium, Dietary - Relevance: LOW - As Found: Unknown - ID: M19075 - Name: Mupirocin - Relevance: HIGH - As Found: Bispectral Index - ID: M9789 - Name: Hormones - 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: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000016712 - Term: Mupirocin - ID: D000002118 - Term: Calcium ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01093079 **Acronym:** LapVsOpen prtn **Brief Title:** Laparoscopic Versus Open Partial Nephrectomy - Surgical and Oncological Outcomes #### Organization Study ID Info **ID:** 5489 #### Organization **Class:** OTHER **Full Name:** Rabin Medical Center ### Status Module #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2010-03-25 **Type:** ESTIMATED **Last Update Submit Date:** 2010-03-24 **Overall Status:** UNKNOWN #### Start Date **Date:** 2009-09 **Status Verified Date:** 2009-07 #### Study First Post Date **Date:** 2010-03-25 **Type:** ESTIMATED **Study First Submit Date:** 2010-03-24 **Study First Submit QC Date:** 2010-03-24 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Rabin Medical Center #### Responsible Party **Old Name Title:** Golan Shay, MD **Old Organization:** Rabin Medical Center ### Description Module **Brief Summary:** Partial Nephrectomy is the standard care for small (\<4 cm) renal tumors. Despite the expanding use of laparoscopic approach, debate exist regarding the short and long term outcomes compared to the open approach. Our goal is to perform a prospective randomized trial to compare these methods ### Conditions Module **Conditions:** - Tumor - Nephrectomy - Laparoscopy **Keywords:** - Renal tumor, RCC, Partial nephrectomy, Laparoscopy ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 100 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Procedure: Laparoscopic partial nephrectomy **Label:** Laparoscopic partial nephrectomy #### Arm Group 2 **Intervention Names:** - Procedure: Open partial nephrectomy **Label:** Open Partial nephrectomy ### Interventions #### Intervention 1 **Arm Group Labels:** - Laparoscopic partial nephrectomy **Description:** Laparoscopic partial nephrectomy **Name:** Laparoscopic partial nephrectomy **Type:** PROCEDURE #### Intervention 2 **Arm Group Labels:** - Open Partial nephrectomy **Description:** Open partial nephrectomy **Name:** Open partial nephrectomy **Type:** PROCEDURE ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * patients referred to partial nephrectomy Exclusion Criteria: * Contra-indication for laparocopic surgery * Complexed , large tumors, single kidney, chronic renal failure **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Patients referred to partial nephrectomy due to renal lesion , suspected to be a tumor ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Shay Golan, MD **Phone:** 972-50-7447573 **Role:** CONTACT #### Locations **Location 1:** **City:** Petach Tikva **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** shay golan, MD - **Phone:** 972-50-3447573 - **Role:** CONTACT **Country:** Israel **Facility:** Rabin medical center **Status:** RECRUITING ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M10703 - Name: Kidney Neoplasms - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05913479 **Acronym:** HYDRAGYNE **Brief Title:** Evaluation of the Efficacy and Safety of Mucogye® Gel as a Moisturizer **Official Title:** HYDRAGYNE (MUCG234) - Evaluation of the Efficacy and Safety of Mucogye® Gel as a Moisturizer #### Organization Study ID Info **ID:** MUCG234 #### Organization **Class:** INDUSTRY **Full Name:** Biocodex #### Secondary ID Infos **Domain:** ID-RCB **ID:** 2022-A02088-35 **Type:** OTHER ### Status Module #### Completion Date **Date:** 2024-07 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-01-18 **Type:** ACTUAL **Last Update Submit Date:** 2024-01-17 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-07 **Type:** ESTIMATED #### Start Date **Date:** 2023-10-10 **Type:** ACTUAL **Status Verified Date:** 2024-01 #### Study First Post Date **Date:** 2023-06-22 **Type:** ACTUAL **Study First Submit Date:** 2023-05-31 **Study First Submit QC Date:** 2023-06-12 ### Sponsor Collaborators Module #### Collaborators **Class:** UNKNOWN **Name:** Voisin Consulting Life Science (VCLS) #### Lead Sponsor **Class:** INDUSTRY **Name:** Biocodex #### 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 of this post-Market Clinical Follow-up (PMCF) study for a class IIb medical device is to confirm th efficacy and safety of Mucogyne Gel as a moisturizer in women with vaginal dryness irrespective of the cause; when used in accordance with its approved labelling. ### Conditions Module **Conditions:** - Vaginal Dryness ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP **Intervention Model Description:** Prospective, multicenter (France), open label without comparator study ##### Masking Info **Masking:** NONE **Primary Purpose:** OTHER #### Enrollment Info **Count:** 50 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 3 planned visits for each eligible patient: * Screening/Baseline visit: V0 at Day 0. Patient receive a box of Mucogyne Gel. * Phone call: V1 phone call at Day 10 ± 3 * End-of-study visit: V2 at Day 35 ± 3 **Intervention Names:** - Device: Mucogyne Gel **Label:** Mucogyne treatment **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Mucogyne treatment **Description:** At inclusion visit (V0), the Investigator will ask the subject to apply MUCOGYNE® Gel as described in the Instructions For Use, i.e., internally, one application 2 to 3 times a week until symptoms improve during 5 weeks (D35 +/- 3) **Name:** Mucogyne Gel **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Primary efficacy criterion: Clinical scoring, by the investigator, of the Vaginal Health Index Score (VHIS), including evaluation of elasticity, fluid volume, pH, epithelial integrity, and moisture. **Measure:** Assessment of the efficacy of MUCOGYNE® Gel intra vaginal applications on subjects' vaginal dryness irrespective of the cause over a 35-day period of use. **Time Frame:** Day 0 to Day 35 +/- 3 #### Secondary Outcomes **Description:** Secondary efficacy criteria - Auto-evaluation, by the subjects, of pain and/or dyspareunia on Visual Analogical Scales (VAS) from 0 to 10 **Measure:** Assessment of the local performance of MUCOGYNE® Gel in reducing vulva irritation **Time Frame:** Day 0 to Day 35 +/- 3 **Description:** Secondary efficacy criteria - Clinical scoring of vulvo-vaginal dryness by the Investigator on a Visual Analogical Scale from 0 to 10 **Measure:** Assessment of the local performance of MUCOGYNE® Gel in reducing vulvo-vaginal dryness **Time Frame:** Day 0 to Day 35 +/- 3 **Description:** Secondary efficacy criteria - Completion of the CGI-I (Clinical Global Impressions-Improvement) by the Investigator. **Measure:** Assessment by investigator of changes in patient's clinical status **Time Frame:** Day 0 to Day 35 +/- 3 **Description:** Secondary efficacy criteria - Completion of the Patients' Global Impression of Change (PGIC) Scale to assess the changes perceived by the patient following the treatment taken. **Measure:** Assessment of changes in patient's clinical status **Time Frame:** Day 0 to Day 35 +/- 3 **Description:** Secondary efficacy criteria - Completion of the intra vaginal treatment satisfaction questionnaire (Likert questionnaire) to assess the level of satisfaction of the treatment on the vaginal symptoms. **Measure:** Assessment of the patient's satisfaction **Time Frame:** Day 0 to Day 35 +/- 3 **Description:** Secondary efficacy criteria - Completion of the self-reported FSFI (Female Sexual Function Index) to investigate sexual function (for women having sexual relations). **Measure:** Assessment of the patient's sexual function **Time Frame:** Day 0 to Day 35 +/- 3 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: Women with the following conditions: * ≥ 18 years of age at inclusion with vaginal dryness, irrespective of the cause (which may or may not be accompanied by irritation of the vulva, discomfort, or itchiness). * Having a Vaginal Health Index Score (VHIS) \<15 associated to pain and/or dyspareunia feeling. * Subject agrees to not use any lubricant, local estrogens, or other vaginal product during the study. * Subject agrees to not modify their intimate hygiene products. * Able to understand and sign the informed consent form for study enrolment. * Subject able to comply with study requirements, as defined in the protocol. * Subject affiliated to a health social security system. Exclusion Criteria: Women with the following conditions: * General: * Pregnancy (subject of childbearing potential must not be pregnant and must agree to avoid pregnancy during the study by using an effective birth control method from at least one month before D0 (V0) and throughout the duration of the study). * Participating at the same time in another interventional trial within the four previous weeks and during the study period, being in an exclusion period for a previous study. * Deprived of freedom by administrative or legal decision or under guardianship. * Subject in a social or sanitary establishment. * Subject suspected to be non-compliant according to the investigator's judgment. * Subject in an emergency situation. * Linked to subject's status: * Known hypersensitivity to one of MUCOGYNE® Gel components. * Subject with a known vaginal pathology (clinical diagnosis only) other than vaginal dryness/atrophy. * Linked to previous or ongoing treatments: * Subject with a condition or receiving a medication which, in the investigator's judgment, put the subject at undue risk. * Subject suffering from systemic diseases and/or using concurrent therapy that may interfere with the evaluation of the study results. * Subject undergoing a topical treatment on the test area or a systemic treatment: corticosteroids during the 2 previous weeks and during the study; retinoids and/or immunosuppressors during the 1,5 previous months and during the study; subject having started or changed her oral contraceptive or any other hormonal treatment during the one previous month. **Gender Based:** True **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Roxane NOEL **Phone:** +33 3 44 86 75 79 **Phone Ext:** +33 **Role:** CONTACT #### Locations **Location 1:** **City:** Caen **Contacts:** ***Contact 1:*** - **Name:** Paul LEFEVRE, md - **Role:** CONTACT ***Contact 2:*** - **Name:** Paul LEFEVRE, MD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** France **Facility:** Dr Paul Lefevre's medical office **Status:** RECRUITING **Zip:** 14000 **Location 2:** **City:** Colmar **Contacts:** ***Contact 1:*** - **Name:** Thierry KELLER, MD - **Role:** CONTACT ***Contact 2:*** - **Name:** Thierry KELLER, MD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** France **Facility:** Dr Thierry KELLER's medical office **Status:** RECRUITING **Zip:** 68000 **Location 3:** **City:** Paris **Contacts:** ***Contact 1:*** - **Name:** Sihame MOKHBAT, midwife - **Role:** CONTACT ***Contact 2:*** - **Name:** Sihame MOKHBAT, Midwife - **Role:** PRINCIPAL_INVESTIGATOR **Country:** France **Facility:** Sihame MOKHBAT's office **Status:** RECRUITING **Zip:** 75018 **Location 4:** **City:** Écully **Contacts:** ***Contact 1:*** - **Name:** Christiane ARMAND, MD - **Role:** CONTACT ***Contact 2:*** - **Name:** Christiane ARMAND, MD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** France **Facility:** Dr Christiane ARMAND's medical office **Status:** RECRUITING **Zip:** 69130 #### Overall Officials **Official 1:** **Affiliation:** Chief Scientific officer **Name:** Oana BERNARD, MD **Role:** STUDY_DIRECTOR ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24