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## Protocol Section ### Identification Module **NCT ID:** NCT03041779 **Acronym:** SuPPerP **Brief Title:** Comparison Between Rectal Suppository Acetaminophen and Diclofenac Sodium as Analgesia for Postpartum Perineal Tear **Official Title:** A Single Blinded, Open-labelled, Randomized Control Trial Comparing Acetaminophen Rectal Suppository With Diclofenac Rectal Suppository as Analgesia for Perineal Injury Following Childbirth #### Organization Study ID Info **ID:** NMRR-15-868-26140 #### Organization **Class:** OTHER **Full Name:** Clinical Research Centre, Malaysia ### Status Module #### Completion Date **Date:** 2016-07 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-02-03 **Type:** ESTIMATED **Last Update Submit Date:** 2017-02-01 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2016-07 **Type:** ACTUAL #### Start Date **Date:** 2015-10 **Status Verified Date:** 2017-02 #### Study First Post Date **Date:** 2017-02-03 **Type:** ESTIMATED **Study First Submit Date:** 2016-12-12 **Study First Submit QC Date:** 2017-02-01 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Clinical Research Centre, Malaysia #### Responsible Party **Investigator Affiliation:** Clinical Research Centre, Malaysia **Investigator Full Name:** Woon Shu Yuan **Investigator Title:** Doctor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** To assess the prevalence of pain score for perineum pain following childbirth followed by phase 2 study to assess the analgesic effectiveness of acetaminophen and diclofenac rectal suppository in postpartum perineum pain secondary to perineal trauma. **Detailed Description:** Studies has demonstrated that non-steroidal anti-inflammatory drugs (NSAIDs) rectal suppositories are associated with less pain up to 24 hours after birth, and less additional analgesia is required. Therefore, In view of rectal route of analgesic administration is better in local action and systemic paracetamol also proven to be effective in controlling post-partum perineal pain with unknown effectiveness in its suppository form; the investigators would like to conduct this study to investigate the effectiveness of acetaminophen rectal suppository versus diclofenac rectal suppository in controlling postpartum perineal pain. This study will be conducted in 2 stages. Stage 1 is a 3 months prospective observational study which aims to determine the prevalence and severity of perineal pain following childbirth in Hospital Sultanah Aminah, Johor Bahru; while Stage 2 is a single blinded, open-labelled, randomized control trial study design which will determine if acetaminophen rectal suppository is as equivalence as diclofenac rectal suppository in reducing postpartum perineal pain secondary to perineal trauma. ### Conditions Module **Conditions:** - Perineal Tear - Perineal Laceration (Obstetric) ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - PARTICIPANT **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 909 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Paracetamol 500Mg Suppository **Intervention Names:** - Drug: Diclofenac Sodium 50Mg Suppository **Label:** Paracetamol **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Diclofenac Sodium 50Mg Suppository **Intervention Names:** - Drug: Paracetamol 500Mg Suppository **Label:** Voltaren **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Paracetamol **Name:** Diclofenac Sodium 50Mg Suppository **Other Names:** - Voltaren 50Mg Suppository **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Voltaren **Name:** Paracetamol 500Mg Suppository **Other Names:** - Acetaminophen 500Mg Suppository **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** perineum pain score following childbirth **Time Frame:** at 2nd to 3rd hour post repair #### Secondary Outcomes **Measure:** perineum pain score following childbirth **Time Frame:** immediate after delivery **Measure:** perineum pain score following childbirth **Time Frame:** immediate post perineum repair **Measure:** perineum pain score following childbirth **Time Frame:** at 5th to 6th hour post repair **Measure:** perineum pain score following childbirth **Time Frame:** prior to discharge ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: Stage 1: a) All pregnant women who sustained perineal trauma (either 1st degree tear, 2nd degree tear or episiotomy) post vaginal delivery Stage 2: 1. All pregnant women who have planned vaginal delivery in HSAJB from 1st January 2016 till 30th June 2016. 2. All pregnant women who sustained 1st degree/ 2nd degree perineal tear or episiotomy tear post vaginal delivery. 3. All pregnant women who have consented to involve in the study. Exclusion Criteria: Stage 1: 1. Patient who sustained additional perineal tear (eg. Labial tear or periurethral tear) following childbirth. 2. Patient who developed post-partum complications. Stage 2: 1. Patient who is allergic to paracetamol or voltaren. 2. Patient who is unable to or unwilling to give consent. 3. Patient who is ended up with caesarean section. 4. Patient who sustained additional perineal tear including labial tear or periurethral tear. 5. Patient who developed postpartum complications (eg. Retained placenta, uterine atony, postpartum haemorrhage, multiple vaginal wall tear etc. ) **Sex:** FEMALE **Standard Ages:** - CHILD - ADULT - OLDER_ADULT ### Contacts Locations Module #### Overall Officials **Official 1:** **Affiliation:** O&G department, Hospital Sultanah Aminah Johor Bahru, Malaysia **Name:** Shu Yuan Woon, MBBS **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ### References Module #### References **Citation:** Cunningham G, Leveno K, Bloom S, et al. Maternal Anatomy. Williams obstetrics. 22nd Edition. New York: McGraw-Hill, 2005:21. **Citation:** Albers L, Garcia J, Renfrew M, McCandlish R, Elbourne D. Distribution of genital tract trauma in childbirth and related postnatal pain. Birth. 1999 Mar;26(1):11-7. doi: 10.1046/j.1523-536x.1999.00011.x. **PMID:** 10352050 **Citation:** Macarthur AJ, Macarthur C. Incidence, severity, and determinants of perineal pain after vaginal delivery: a prospective cohort study. Am J Obstet Gynecol. 2004 Oct;191(4):1199-204. doi: 10.1016/j.ajog.2004.02.064. **PMID:** 15507941 **Citation:** Williams A, Herron-Marx S, Carolyn H. The prevalence of enduring postnatal perineal morbidity and its relationship to perineal trauma. Midwifery. 2007 Dec;23(4):392-403. doi: 10.1016/j.midw.2005.12.006. Epub 2006 Dec 29. **PMID:** 17196714 **Citation:** Hedayati H, Parsons J, Crowther CA. Rectal analgesia for pain from perineal trauma following childbirth. Cochrane Database Syst Rev. 2003;(3):CD003931. doi: 10.1002/14651858.CD003931. **PMID:** 12917995 **Citation:** Chou D, Abalos E, Gyte GM, Gulmezoglu AM. Paracetamol/acetaminophen (single administration) for perineal pain in the early postpartum period. Cochrane Database Syst Rev. 2013 Jan 31;(1):CD008407. doi: 10.1002/14651858.CD008407.pub2. **PMID:** 23440827 **Citation:** Gilman G. Drug absorption, bioavailability, and routes of administration. The pharmacological basis of therapeutics. 8th Edition. New York: Macmillan Publishing Co. 1990:7. ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014947 - Term: Wounds and Injuries ### Condition Browse Module - Browse Branches - Abbrev: BC26 - Name: Wounds and Injuries - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M15241 - Name: Rupture - Relevance: LOW - As Found: Unknown - ID: M22785 - Name: Lacerations - Relevance: HIGH - As Found: Laceration - ID: M17685 - Name: Wounds and Injuries - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000022125 - Term: Lacerations ### Intervention Browse Module - Ancestors - ID: D000018712 - Term: Analgesics, Non-Narcotic - ID: D000000700 - Term: Analgesics - ID: D000018689 - Term: Sensory System Agents - ID: D000018373 - Term: Peripheral Nervous System Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000058633 - Term: Antipyretics - ID: D000000894 - Term: Anti-Inflammatory Agents, Non-Steroidal - ID: D000000893 - Term: Anti-Inflammatory Agents - ID: D000018501 - Term: Antirheumatic Agents - ID: D000016861 - Term: Cyclooxygenase Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action ### Intervention Browse Module - Browse Branches - Abbrev: Antipy - Name: Antipyretics - Abbrev: Analg - Name: Analgesics - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Infl - Name: Anti-Inflammatory Agents - Abbrev: ARhu - Name: Antirheumatic Agents ### Intervention Browse Module - Browse Leaves - ID: M2340 - Name: Acetaminophen - Relevance: HIGH - As Found: Compared - ID: M7197 - Name: Diclofenac - Relevance: HIGH - As Found: Cartilage - ID: M4032 - Name: Analgesics - Relevance: LOW - As Found: Unknown - ID: M20786 - Name: Analgesics, Non-Narcotic - Relevance: LOW - As Found: Unknown - ID: M29176 - Name: Antipyretics - Relevance: LOW - As Found: Unknown - ID: M4217 - Name: Anti-Inflammatory Agents - Relevance: LOW - As Found: Unknown - ID: M4218 - Name: Anti-Inflammatory Agents, Non-Steroidal - Relevance: LOW - As Found: Unknown - ID: M20604 - Name: Antirheumatic Agents - Relevance: LOW - As Found: Unknown - ID: M19209 - Name: Cyclooxygenase Inhibitors - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000000082 - Term: Acetaminophen - ID: D000004008 - Term: Diclofenac ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03646279 **Brief Title:** Thoracolumbar Brace for Individuals With Parkinson's Disease **Official Title:** Pilot Investigation of a Thoracolumbar Brace for Individuals With Parkinson's Disease #### Organization Study ID Info **ID:** 2015/06/7 #### Organization **Class:** OTHER **Full Name:** Duquesne University ### Status Module #### Completion Date **Date:** 2016-03-31 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-04-27 **Type:** ACTUAL **Last Update Submit Date:** 2021-04-23 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2016-03-31 **Type:** ACTUAL #### Start Date **Date:** 2015-09-17 **Type:** ACTUAL **Status Verified Date:** 2021-04 #### Study First Post Date **Date:** 2018-08-24 **Type:** ACTUAL **Study First Submit Date:** 2018-08-20 **Study First Submit QC Date:** 2018-08-22 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** AbiliLife #### Lead Sponsor **Class:** OTHER **Name:** Duquesne University #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Is US Export:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** This pilot study looked to see if a new brace would change the way people with Parkinson's Disease positioned their bodies. It also gathered information on the experience of wearing the brace. ### Conditions Module **Conditions:** - Parkinson Disease ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP **Intervention Model Description:** Single-subject A-B design ##### Masking Info **Masking:** NONE **Primary Purpose:** DEVICE_FEASIBILITY #### Enrollment Info **Count:** 10 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Description:** The CALIBRACE is a new product designed to improve posture using a front-to-back tensioning system that lifts shoulders up and back. **Name:** Calibrace **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Measured using PostureScreen Mobile Application and all measured bilaterally Neck position measured from (EAM to Acrominom) * angulation in degrees (flexion values positive/extension values negative) * translation in inches (anterior values positive and posterior values negative) **Measure:** Postural Alignment - Neck Position **Time Frame:** In a single session, 10 data points per phase were collected **Description:** Measured using PostureScreen Mobile Application and all measured bilaterally Trunk position measure form Acrominom to Greater Trochanter of femur, * angulation in degrees (flexion values positive/extension values negative) * translation in inches (anterior values positive and posterior values negative) **Measure:** Postural Alignment - Trunk Position **Time Frame:** In a single session, 10 data points per phase were collected **Description:** Measured using PostureScreen Mobile Application and all measured bilaterally Hip/knee position measured from greater trochanter of femur to lateral epicondyle of femur * angulation in degrees (flexion values positive/extension values negative) * translation in inches (anterior values positive and posterior values negative) **Measure:** Postural Alignment - Hip/Knee Position **Time Frame:** In a single session, 10 data points per phase were collected #### Secondary Outcomes **Description:** The mean center of pressure location measured in root mean squared through the average of three trials of eyes open and the average of three trials of eyes closed. **Measure:** Postural Sway - Center of pressure position **Time Frame:** Single session **Description:** The mean center of pressure velocity measured in meters/second through the average of three trials of eyes open and the average of three trials of eyes closed. **Measure:** Postural Sway - Center of pressure velocity **Time Frame:** Single session **Description:** Survey/interview with seven 4 points Likert-type rating scale (Strongly Disagree, Disagree, Agree, Strongly Agree). High numbers represent more agreement. and three open-ended questions There are no total or subscale scores. The frequency of ratings by participants, by the question is what is reported. **Measure:** Researcher Developed Survey on the Experience of the Brace - No Formal Name or Abbreviation **Time Frame:** Single Session ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * 50-80 years of age * Diagnosed with Parkinson's Disease * Participant self-reports mild difficulty with posture * Independent in ambulation without an assistive device (i.e., no walker or cane) * Able to give consent determined via telephone screen assessment or at minimum can follow simple directions * Able to tolerate 5 minutes of standing or walking at a time per self-report Exclusion Criteria: * Rigid or fixed spine, as determined by self-report and use of thoracolumbar flexibility screen * Symptoms present before the age of 50 * Other neurologic diagnosis, as determined by self-report * Spinal surgery * Lives in a nursing home or skilled facility * Uncorrected vision loss * Skin that is sensitive to tape or extremely fragile * Recent surgery (within the last 3 months) that affects mobility, as determined by self-report * Chest pain at rest or with activity per self report * Shortness of breath with daily activity per self report * Severe orthostatic hypotension assessed during in-person screen with sitting and standing blood pressure, evidenced by a 20-point drop in systolic blood pressure or 10-point drop in diastolic blood pressure and symptoms (e.g., dizziness) that don't resolve in 5 minutes. * High blood pressure 180/110 resting or systolic blood pressure \<90 mmHG * Resting heart rate \> 100 bpm or \<50 bpm * Waist circumference \< 31" or \> 51" that would prevent proper fitting of CALIBRACE. **Maximum Age:** 80 Years **Minimum Age:** 50 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### IPD Sharing Statement Module **Description:** This is not currently a plan to share these data with other researchers. **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020734 - Term: Parkinsonian Disorders - ID: D000001480 - Term: Basal Ganglia Diseases - ID: D000001927 - Term: Brain Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000009422 - Term: Nervous System Diseases - ID: D000009069 - Term: Movement Disorders - ID: D000080874 - Term: Synucleinopathies - ID: D000019636 - Term: Neurodegenerative Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases ### Condition Browse Module - Browse Leaves - ID: M13213 - Name: Parkinson Disease - Relevance: HIGH - As Found: Parkinson's Disease - ID: M22494 - Name: Parkinsonian Disorders - Relevance: LOW - As Found: Unknown - ID: M25603 - Name: Ganglion Cysts - Relevance: LOW - As Found: Unknown - ID: M16358 - Name: Synovial Cyst - Relevance: LOW - As Found: Unknown - ID: M4774 - Name: Basal Ganglia Diseases - Relevance: LOW - As Found: Unknown - ID: M5204 - Name: Brain Diseases - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: M12029 - Name: Movement Disorders - Relevance: LOW - As Found: Unknown - ID: M2217 - Name: Synucleinopathies - Relevance: LOW - As Found: Unknown - ID: M21558 - Name: Neurodegenerative Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000010300 - Term: Parkinson Disease ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03607279 **Brief Title:** Comparison of Plasma Neutrophil Gelatinase - Associated Lipocalin (NGAL) Levels **Official Title:** Comparison of Plasma Neutrophil Gelatinase - Associated Lipocalin (NGAL) Levels After Robot-assisted Laparoscopic Versus Retropubic Radical Prostatectomy #### Organization Study ID Info **ID:** 2016-118 #### Organization **Class:** OTHER_GOV **Full Name:** Antalya Training and Research Hospital ### Status Module #### Completion Date **Date:** 2017-08 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-12-19 **Type:** ACTUAL **Last Update Submit Date:** 2018-12-18 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2017-06 **Type:** ACTUAL #### Start Date **Date:** 2017-01 **Type:** ACTUAL **Status Verified Date:** 2018-12 #### Study First Post Date **Date:** 2018-07-31 **Type:** ACTUAL **Study First Submit Date:** 2018-07-23 **Study First Submit QC Date:** 2018-07-30 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER_GOV **Name:** Antalya Training and Research Hospital #### Responsible Party **Investigator Affiliation:** Antalya Training and Research Hospital **Investigator Full Name:** Arzu Karaveli **Investigator Title:** Study director **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of the study is to compare the effect of two surgical techniques (open vs robotic assisted) on plasma levels of NGAL (neutrophil gelatinase-associated lipocalin) after radical prostatectomy. **Detailed Description:** Patients undergoing radical prostatectomy are at increased risk of acute kidney injury (AKI) because of intraoperative bleeding, obstructive uropathy and older age. In particular robot-assisted laparoscopic radical prostatectomy (RALP) which is in increasing demand as an alternative surgical option for retropubic radical prostatectomy (RRP) is associated with postoperative renal dysfunction because pneumoperitoneum during RALP can decrease cardiac output and renal perfusion. NGAL may become important for diagnosis of postoperative AKI after urogenital oncosurgery. ### Conditions Module **Conditions:** - Prostatectomy **Keywords:** - NGAL - Prostatectomy - Acute kidney injury ### Design Module #### Design Info **Observational Model:** CASE_CONTROL **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 66 **Type:** ACTUAL **Patient Registry:** True **Study Type:** OBSERVATIONAL **Target Duration:** 6 Months ### Arms Interventions Module #### Arm Group 1 **Description:** Plasma NGAL was determined at baseline, 6 and 12 hrs after induction of anaesthesia **Intervention Names:** - Procedure: NGAL **Label:** NGAL - retropubic radical prostatectomy #### Arm Group 2 **Description:** Plasma NGAL was determined at baseline, 6 and 12 hrs after induction of anaesthesia **Intervention Names:** - Procedure: NGAL **Label:** NGAL - robotic radical prostatectomy ### Interventions #### Intervention 1 **Arm Group Labels:** - NGAL - retropubic radical prostatectomy - NGAL - robotic radical prostatectomy **Name:** NGAL **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Description:** Plasma NGAL was determined at baseline, 6 hours and 12 hours after surgery, ng/ml **Measure:** Plasma NGAL **Time Frame:** 0 hours, 6 hours and 12 hours after surgery ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * patients who undergo prostatectomy * American Society of Anesthesiology (ASA) I-III Exclusion Criteria: * preexisting renal disease * emergency surgery * heart failure * peripheral vascular disease **Maximum Age:** 80 Years **Minimum Age:** 18 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** MALE **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** prostatectomy ### Contacts Locations Module #### Locations **Location 1:** **City:** Antalya **Country:** Turkey **Facility:** Antalya Training and Research Hospital, Department of Anesthesiology and Reanimation **Zip:** 07100 #### Overall Officials **Official 1:** **Affiliation:** Netherlands: Ministry of Health, Welfare and Sports **Name:** Arzu Karaveli **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Orsolya M, Attila-Zoltan M, Gherman V, Zaharie F, Bolboaca S, Chira C, Bodolea C, Tomuleasa C, Irimie A, Coman I, Ionescu D. The effect of anaesthetic management on neutrophil gelatinase associated lipocalin (NGAL) levels after robotic surgical oncology. J BUON. 2015 Jan-Feb;20(1):317-24. **PMID:** 25778333 **Citation:** Joo EY, Moon YJ, Yoon SH, Chin JH, Hwang JH, Kim YK. Comparison of Acute Kidney Injury After Robot-Assisted Laparoscopic Radical Prostatectomy Versus Retropubic Radical Prostatectomy: A Propensity Score Matching Analysis. Medicine (Baltimore). 2016 Feb;95(5):e2650. doi: 10.1097/MD.0000000000002650. **PMID:** 26844486 ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M28998 - Name: Acute Kidney Injury - Relevance: LOW - As Found: Unknown - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Browse Branches - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M4107 - Name: Anesthetics - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02829879 **Brief Title:** Effectiveness of Arginin and Potassium Nitrate Dentifrices in Dentin Hypersensitivity Therapy **Official Title:** Comparison of the Clinical Effectiveness of 8% Arginine/1450 Ppm Sodium Monofluorophosphate Versus 5% Potassium Nitrate/2500 Ppm Sodium Fluoride in Dentin Hypersensitivity Therapy: A Randomized Controlled Clinical Trial #### Organization Study ID Info **ID:** FIOUCh 13-103 #### Organization **Class:** OTHER **Full Name:** University of Chile ### Status Module #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2016-07-12 **Type:** ESTIMATED **Last Update Submit Date:** 2016-07-11 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2016-09 **Type:** ESTIMATED #### Start Date **Date:** 2015-12 **Status Verified Date:** 2016-07 #### Study First Post Date **Date:** 2016-07-12 **Type:** ESTIMATED **Study First Submit Date:** 2016-07-08 **Study First Submit QC Date:** 2016-07-11 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Chile #### Responsible Party **Investigator Affiliation:** University of Chile **Investigator Full Name:** Patricia Hernández-Ríos **Investigator Title:** Professor **Type:** PRINCIPAL_INVESTIGATOR ### Description Module **Brief Summary:** Introduction: Dentin hypersensitivity (DH) could be defined as a brief and sharp pain in response to thermical, chemical, tactile and osmotic stimuli, which cannot be attributed to any dental condition or pathology. Multiple therapies for the treatment of DH have been studied, including equivocal evidence about the efficacy of potassium nitrate salts and recent investigations a promising new 8% Arginin dentifrice. Aim: The aim of this study is to compare the clinical effectiveness of 8% arginine/1450ppm sodium monofluorophosphate (D1) and 5% potassium nitrate/2500ppm sodium fluoride (D2) dentifrices in the treatment of dentin hypersensitivity. Methods: Parallel-design, double-masked, randomized controlled clinical trial. Fifty healthy volunteers aged 18 to 70 years who attend at the Faculty of Dentistry , University of Chile, with DH and a visual analog scale (VAS) score ≥4 at least in two anterior and/or premolar teeth, will be selected and randomized into two treatment groups: T1 (n=25): 8% arginine/1450 ppm sodium monofluorophosphate dentifrice (D1); and T2 (n=25): 5% potassium nitrate/2500 ppm sodium fluoride dentifrice (D2). Environmental, dietary and oral hygiene habits will be recorded in a clinical chart, while O´Leary plaque index and dentin hypersensitivity (DH) measurements will be clinically assessed, at baseline, 4 and 8 weeks. DH will be evaluated through the mean VAS value in response to evaporative and thermal stimuli at baseline, 4 and 8 week follow-up. The data will be analysed through Stata® V11 program. ### Conditions Module **Conditions:** - Dentin Sensitivity **Keywords:** - Dentin Sensitivity - Arginin - Potassium Nitrate - Dentifrice - RCT ### 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:** 50 **Type:** ESTIMATED **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 25 volunteers with dentin sensitivity **Intervention Names:** - Drug: 8% arginine/1450ppm sodium monofluorophosphate dentifrice **Label:** arginine **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** 25 volunteers with dentin sensitivity **Intervention Names:** - Drug: 5% potassium nitrate/2500ppm sodium fluoride dentifrice **Label:** potassium nitrate **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - arginine **Name:** 8% arginine/1450ppm sodium monofluorophosphate dentifrice **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - potassium nitrate **Name:** 5% potassium nitrate/2500ppm sodium fluoride dentifrice **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Differences in mean VAS score per subject **Time Frame:** Baseline, 4 and 8 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Age between 18 and 70 years * Dentin sensitivity in at least 2 teeth (incisors, cuspids and/or bicuspids) with a VAS score ≥4 * Good general health Exclusion Criteria: * Defective or extensive restorations, deep dental caries, pulpitis, chipped teeth, * Bridgework, dentures or crowned teeth that could interfere with the evaluation of hypersensitivity * Pregnancy or lactation * Ongoing treatment with anti-inflammatory or sedative drugs * Systemic conditions that are etiologic or predisposing to dentine hypersensitivity, like chronic acid regurgitation * Ongoing periodontal therapy or periodontal surgery in the preceding 3 months * Allergic responses to the dentifrices * Hypersensitivity treatment in the preceding 3 months **Maximum Age:** 70 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Patricia Hernández-Ríos, Professor **Phone:** +56229781839 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Johanna Contreras, Professor **Phone:** +56229781839 **Role:** CONTACT #### Locations **Location 1:** **City:** Santiago **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Patricia Hernández-Ríos, Professor - **Phone:** +56229781839 - **Role:** CONTACT **Country:** Chile **Facility:** Faculty of Dentistry, University of Chile **State:** Región Metropolitana **Status:** RECRUITING ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007154 - Term: Immune System Diseases - ID: D000014076 - Term: Tooth Diseases - ID: D000009057 - Term: Stomatognathic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC20 - Name: Immune System Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC07 - Name: Mouth and Tooth Diseases ### Condition Browse Module - Browse Leaves - ID: M10018 - Name: Hypersensitivity - Relevance: HIGH - As Found: Hypersensitivity - ID: M7003 - Name: Dentin Sensitivity - Relevance: HIGH - As Found: Dentin Sensitivity - ID: M10200 - Name: Immune System Diseases - Relevance: LOW - As Found: Unknown - 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: D000003807 - Term: Dentin Sensitivity - ID: D000006967 - Term: Hypersensitivity ### Intervention Browse Module - Ancestors - ID: D000002327 - Term: Cariostatic Agents - ID: D000020011 - Term: Protective Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000000891 - Term: Anti-Infective Agents, Local - ID: D000000890 - Term: Anti-Infective Agents ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: AA - Name: Amino Acids ### Intervention Browse Module - Browse Leaves - ID: M8587 - Name: Fluorides - Relevance: HIGH - As Found: Concentration - ID: M140080 - Name: Listerine - Relevance: HIGH - As Found: 1.2 - ID: M15771 - Name: Sodium Fluoride - Relevance: HIGH - As Found: 1.2 - ID: M247884 - Name: Fluorophosphate - Relevance: HIGH - As Found: 5-Azacytidine - ID: M21869 - Name: Protective Agents - Relevance: LOW - As Found: Unknown - ID: M4214 - Name: Anti-Infective Agents - Relevance: LOW - As Found: Unknown - ID: M4215 - Name: Anti-Infective Agents, Local - Relevance: LOW - As Found: Unknown - ID: T1 - Name: Arginine - Relevance: HIGH - As Found: Radical ### Intervention Browse Module - Meshes - ID: C000057473 - Term: Listerine - ID: D000005459 - Term: Fluorides - ID: D000012969 - Term: Sodium Fluoride - ID: C000012980 - Term: Fluorophosphate ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04799379 **Acronym:** EPHAS **Brief Title:** Microporous Polysaccharide Hemospheres (MPH) Improving Outcome After Rectal Surgery **Official Title:** Evaluation of Microporous Polysaccharide Hemospheres (MPH) Agent in Preventing Postoperative Complications After Rectal Surgery (EPHAS Study). #### Organization Study ID Info **ID:** GERM-EPHAS #### Organization **Class:** OTHER **Full Name:** Grupo Español de Rehabilitación Multimodal ### Status Module #### Completion Date **Date:** 2023-06-30 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2021-12-29 **Type:** ACTUAL **Last Update Submit Date:** 2021-12-28 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2021-12-31 **Type:** ESTIMATED #### Start Date **Date:** 2021-04-01 **Type:** ACTUAL **Status Verified Date:** 2021-12 #### Study First Post Date **Date:** 2021-03-16 **Type:** ACTUAL **Study First Submit Date:** 2021-02-26 **Study First Submit QC Date:** 2021-03-11 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Universidad de Zaragoza **Class:** OTHER_GOV **Name:** Aragon Health Science Institute #### Lead Sponsor **Class:** OTHER **Name:** Grupo Español de Rehabilitación Multimodal #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** Multi-center, prospective, cohort study, of patients scheduled for elective rectal surgery who received absorbable hemostatic powder to assess whether it can improve patient postoperative outcomes, reduce usage of drains and prove to be cost-effective. This cohort will be compared to a previously studied cohort using drains (Power and IMPRICA study) with comparable patient characteristics. The primary endpoint of the study is: postoperative pelvic sepsis within 60 postoperative days including anastomotic leakage, pelvic abscess and peritonitis. Secondary endpoints: postoperative morbidities, rate of reoperation and length of hospitalization. **Detailed Description:** Multimodal surgical rehabilitation, also known as Enhanced Recovery After Surgery, entails the application of a series of perioperative procedure measures and strategies aimed at patients who are going to undergo a surgical procedure with the objective of reducing secondary stress caused by the surgical intervention and thus achieve enhanced recovery of the patient and decrease complications and mortality. ERAS protocols are care programs based on scientific evidence, encompassing all aspects of patient care and requiring multidisciplinary management, with the participation of diverse specialists1. Starting at the diagnosis, their aim is to recognize patients' individual needs to optimize their treatment before, during and after surgery. The close collaboration of all specialists participating in the process, as well as of the actual patients and their relatives has proved to be essential. The Multimodal Rehabilitation Programmes (MRP) or Enhanced Recovery Programmes (ERAS) review traditional perioperative procedure practices, evaluating the specific key points of each type of surgery and analysing their scientific evidence. MRPs have shown, in cents that have routinely adopted them, a significant improvement in the patient's quality of life. Furthermore, MRPs significantly reducing the hospital stay and potential complications associated with hospitalisation2, being the anastomotic leak (AL) the most serious of them. Total mesorectal excision (TME) and bowel restoration is currently the standard treatment for middle to low rectal cancer. However, TME has been shown to be associated with high anastomotic leakage with a reported incidence of up to 24%, reaching 50% when clinically silent radiographic leaks are considered3. Despite a large number of studies in the literature that have investigated risk factors, the fundamental causes of AL remain unclear. In this sense, according to enhanced recovery after surgery (ERAS) protocols4, pelvic drain should not be used routinely as it may cause patient discomfort and prolong hospitalization. Moreover, drain itself is also a potential site of infection especially if open or passive drainage system is used. Even based in the best available evidence, ERAS protocols have important implementation problems because they have to put up with traditional attitudes. In this sense, many surgeons still advocate the use of a prophylactic pelvic drain because they believe that fluid collection in the pelvis could be a potential source of contamination and thereby weakening anastomotic integrity and healing. In a recent study from the Spanish group GERM2, avoidance of drains was achieved only in 34.7% of patients undergoing elective colorectal surgery. ERAS centres had a greater avoidance of drains vs non-ERAS centres (38.6% vs 28.3%), although most patients still received drains. More relevant, avoidance of drainage was associated with a significant reduction in moderate to severe complications. We believe that it is more than justified to try new options that help surgeons reduce the use of drains. One of these, is the use of hemostatic agents. Some topical hemostats may theoretically be of benefit due to its claimed lymphostatic properties. Published data for this is sparse, but some studies, have shown statistically significant reduction of fluid collection after using Arista®AH5. Arista is a plant-based, flowable powder engineered to rapidly dehydrate blood, enhancing clotting on contact. Arista facilitates the formation of a highly resilient, natural clot within just a few minutes regardless of the patient's coagulation status. Arista® is fully absorbable within 24 to 48 hours of application, and because Arista degrades rapidly, it does not promote infection. This study has been designed to support the working hypothesis that Arista® placed intraoperatively into the dissected pelvic area could reduce sepsis and postoperative anastomotic leakage. We choose to study Arista® for a theoretical reason: it contains microporous polysaccharide hemospheres (MPHs). MPHs not only activate the coagulation cascade but they cause tissue desiccation which presumably seals capillaries and could theoretically also seal small lymph vessels left open by electrosurgical devices. Study assumptions: Arista spread on the pelvic floor at the end of surgery may allow to 1. close small holes and prevent fluid from penetrating into the pelvic floor, thereby avoiding AL 2. facilitate the avoidance of drains ### Conditions Module **Conditions:** - Postoperative Sepsis **Keywords:** - Rectal surgery - Pelvic drain - microporous polysaccharide hemospheres ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 300 **Type:** ESTIMATED **Patient Registry:** True **Study Type:** OBSERVATIONAL **Target Duration:** 60 Days ### Arms Interventions Module ### Interventions #### Intervention 1 **Description:** Use of microporous polysaccharide hemospheres (MPH) agent in patients after rectal surgery to avoid pelvic collections and the use of any drain **Name:** microporous polysaccharide hemospheres (MPH) agent in rectal surgery **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** % patients with organ-space infection or anastomotic leak **Measure:** postoperative pelvic sepsis including anastomotic leakage, pelvic abscess and peritonitis. **Time Frame:** 60 days #### Secondary Outcomes **Description:** % Patients with at least one complication related to the surgical intervention **Measure:** postoperative morbidities **Time Frame:** 60 days **Description:** % patients requiring reoperation for any cause **Measure:** rate of reoperation **Time Frame:** 60 days **Description:** Total number of days of stay included pre and postoperative. **Measure:** Hospital stay **Time Frame:** 60 days ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * All adult patients (aged \>18 years) with a diagnosis of malignant rectal cancer who are scheduled for radical surgery (anterior resection). Informed consent will be obtained from all subjects, who will participate in the study voluntarily. Exclusion Criteria: * Patient refusal, patients undergoing emergency surgery, patients under 18 years of age, existence of other concomitant surgical processes. Previous chemo-radiotherapy. Ileostomy or colostomy. Patients that have receive a drain. **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Adult patients (aged \>18 years) with a diagnosis of malignant rectal cancer who are scheduled for radical surgery. This study will be conducted in 12 Spanish general hospitals, which are selected on the basis of having established an enhanced recovery protocol that complies with the recommendations of the Aragon Health Sciences Institute (IACS) and Spanish National Health Service (https://portal.guiasalud.es/wpcontent/uploads/2019/10/viaclinica-rica_english.pdf) ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Alejandro Bona, Mr. **Phone:** (+34) 601 102 673 **Role:** CONTACT #### Locations **Location 1:** **City:** Elche **Contacts:** ***Contact 1:*** - **Name:** Antonio Arroyo, PhD - **Role:** CONTACT ***Contact 2:*** - **Name:** Luis Sanchez - **Role:** SUB_INVESTIGATOR ***Contact 3:*** - **Name:** Jose Luis Muñoz - **Role:** SUB_INVESTIGATOR **Country:** Spain **Facility:** Hospital General Universitario de Elche **State:** Alicante **Status:** RECRUITING **Location 2:** **City:** Alzira **Contacts:** ***Contact 1:*** - **Name:** Javier Blanco - **Role:** CONTACT **Country:** Spain **Facility:** Hospital de la RIBERA **State:** Valencia **Status:** NOT_YET_RECRUITING **Location 3:** **City:** Madrid **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Miguel Leon Arellano - **Role:** CONTACT ***Contact 2:*** - **Name:** Damian Garcia Olmo - **Role:** SUB_INVESTIGATOR ***Contact 3:*** - **Name:** Hector Guadalajara - **Role:** SUB_INVESTIGATOR **Country:** Spain **Facility:** Hospital Jimenz diaz **Status:** RECRUITING **Location 4:** **City:** Madrid **Contacts:** ***Contact 1:*** - **Name:** Alicia Ruiz de la Hermosa, MD - **Phone:** 649337762 - **Role:** CONTACT ***Contact 2:*** - **Name:** Maria Luisa Fuenmayor, MD - **Role:** SUB_INVESTIGATOR **Country:** Spain **Facility:** Hospital Universitario Infanta Leonor **Status:** RECRUITING **Location 5:** **City:** Zaragoza **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** José Manuel Ramírez Rodriguez, Prof - **Role:** CONTACT ***Contact 2:*** - **Name:** Vicente Aguilella - **Role:** SUB_INVESTIGATOR ***Contact 3:*** - **Name:** Azucena Gonzalo - **Role:** SUB_INVESTIGATOR ***Contact 4:*** - **Name:** Luigi Antinolfi - **Role:** SUB_INVESTIGATOR ***Contact 5:*** - **Name:** Blanca Martinez - **Role:** SUB_INVESTIGATOR **Country:** Spain **Facility:** Hospital Clínico Universitario Lozano Blesa **Status:** RECRUITING #### Overall Officials **Official 1:** **Affiliation:** Hospital de la Ribera **Name:** Javier Blanco, PhD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ### References Module #### References **Citation:** Ruiz-Tovar J, Sanchez-Santos R, Martin-Garcia-Almenta E, Garcia Villabona E, Hernandez AM, Hernandez-Matias A, Ramirez JM; grupo de Trabajo de Cirugia Bariatrica del Grupo Espanol de Rehabilitacion Multimodal (GERM). Enhanced recovery after bariatric surgery. Cir Esp (Engl Ed). 2019 Dec;97(10):551-559. doi: 10.1016/j.ciresp.2019.05.003. Epub 2019 Jun 18. English, Spanish. **PMID:** 31221424 **Citation:** Ripolles-Melchor J, Ramirez-Rodriguez JM, Casans-Frances R, Aldecoa C, Abad-Motos A, Logrono-Egea M, Garcia-Erce JA, Camps-Cervantes A, Ferrando-Ortola C, Suarez de la Rica A, Cuellar-Martinez A, Marmana-Mezquita S, Abad-Gurumeta A, Calvo-Vecino JM; POWER Study Investigators Group for the Spanish Perioperative Audit and Research Network (REDGERM). Association Between Use of Enhanced Recovery After Surgery Protocol and Postoperative Complications in Colorectal Surgery: The Postoperative Outcomes Within Enhanced Recovery After Surgery Protocol (POWER) Study. JAMA Surg. 2019 Aug 1;154(8):725-736. doi: 10.1001/jamasurg.2019.0995. Erratum In: JAMA Surg. 2022 May 1;157(5):460. **PMID:** 31066889 **Citation:** Cavaliere D, Popivanov G, Cassini D, Cirocchi R, Henry BM, Vettoretto N, Ercolani G, Solaini L, Gerardi C, Tabakov M, Tomaszewski KA. Is a drain necessary after anterior resection of the rectum? A systematic review and meta-analysis. Int J Colorectal Dis. 2019 Jun;34(6):973-981. doi: 10.1007/s00384-019-03276-4. Epub 2019 Apr 25. **PMID:** 31025093 **Citation:** Luong J, Milanese E, Fortino J, Vetto JT. Reduction of lymphocele rate in patients undergoing sentinel node biopsy for melanoma by intraoperative placement of plant-based hemostatic powder: Results of a prospective trial. Am J Surg. 2019 May;217(5):878-881. doi: 10.1016/j.amjsurg.2019.02.016. Epub 2019 Feb 14. **PMID:** 30799018 **Citation:** Grupo de trabajo. Vía Clínica de Recuperación Intensificada en Cirugía Abdominal (RICA). Vía clínica de recuperación intensificada en cirugía abdominal (RICA) Ministerio de Sanidad, Servicios Sociales e Igualdad. Instituto Aragonés de Ciencias de la Salud. 2014 Available from: http://portal.guiasalud.es/contenidos/iframes/documentos/ opbe/2015-07/ViaClinica-RICA.pdf ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC01 - Name: Infections ### Condition Browse Module - Browse Leaves - ID: M14065 - Name: Postoperative Complications - Relevance: LOW - As Found: Unknown - ID: M20864 - Name: Sepsis - Relevance: LOW - As Found: Unknown - ID: M16869 - Name: Toxemia - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03520179 **Acronym:** SMA-REACH **Brief Title:** Improving Standards of Care and Translational Research in Spinal Muscular Atrophy (SMA) **Official Title:** Improving Standards of Care and Translational Research in Spinal Muscular Atrophy (SMA) #### Organization Study ID Info **ID:** 11DN15 #### Organization **Class:** OTHER **Full Name:** Great Ormond Street Hospital for Children NHS Foundation Trust ### Status Module #### Completion Date **Date:** 2023-08-12 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2022-08-17 **Type:** ACTUAL **Last Update Submit Date:** 2022-08-16 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2023-08-12 **Type:** ESTIMATED #### Start Date **Date:** 2013-12-17 **Type:** ACTUAL **Status Verified Date:** 2022-08 #### Study First Post Date **Date:** 2018-05-09 **Type:** ACTUAL **Study First Submit Date:** 2018-01-30 **Study First Submit QC Date:** 2018-04-26 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Great Ormond Street Hospital for Children NHS Foundation Trust #### 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 SMA REACH UK Network is a national and international partnership between doctors and therapists involved in the care of children and adults with Spinal Muscular Atrophy. This network is supported by Biogen and SMA UK. **Detailed Description:** The investigators aim is to establish a Spinal Muscular Atrophy (SMA) National Platform to improve UK standards of care, manage national and International clinical trials and facilitate translational research for this common neuromuscular disease. To achieve this purpose investigators will start to systematically collect longitudinal validated outcome measures for SMA children followed at GOSH, the largest cohort followed in UK, and pilot and update novel outcome measures. This will be done ensuring that data collected are not only clinically meaningful but also robust for subsequent use in clinical trials. In collaboration with the MRC Neuromuscular Centre in London and Newcastle, investigators will link the existing registries and the longitudinal data collection of outcome measures and develop a hub and bespoke platform model linking the other paediatric UK centres involved in the clinical management of SMA patients. This UK SMA Platform (SMA REACH UK) will be a unique infrastructure containing the largest comprehensive longitudinal series of SMA patient data in the UK; the data collected will be agreed between the relevant other UK centres stakeholders and will take into consideration other international initiatives with historical success in SMA treatment and research. Ongoing analysis including modern psychometric techniques will ensure that the functional data collected in the UK SMA population meets the high statistical standards required for the data to inform natural history studies and be usable as an outcome measure for clinical trials. In addition SMA REACH UK is in the position to be involved in an international initiative called ISMAC (International SMA Consortium) with two prestigious Networks: the PNCRN in the United States (Principal Investigator Richard Finkel) and the Italian SMA Network (Principle Investigator Eugenio Mercuri). The Consortium has been contacted by the Biotechnology Company; Biogen with strong interest in collecting anonymised natural history data on the entire spectrum of SMA severity from routine clinical visits. The main data to be collected, including medical information and physiotherapy assessments, were agreed across the three Networks and will be slightly more extended than the current dataset collected at each Centre. The data collected with the new dataset will be collated on a separate IT platform which will contain anonymised clinical and physiotherapy data from patients who have consented to take part, and will be accessible to Biogen and can be shared with third parties (pharmaceuticals, academic and government institutions) in a strictly anonymised form. The ownership of the data will remain with the PIs at each centre. ### Conditions Module **Conditions:** - Spinal Muscular Atrophy ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** CROSS_SECTIONAL #### Enrollment Info **Count:** 600 **Type:** ESTIMATED **Patient Registry:** True **Study Type:** OBSERVATIONAL **Target Duration:** 18 Months ### Arms Interventions Module #### Arm Group 1 **Description:** genetically confirmed SMA **Label:** SMA TYPE 1 #### Arm Group 2 **Description:** genetically confirmed SMA **Label:** SMA TYPE 2 #### Arm Group 3 **Description:** genetically confirmed SMA, Ambulant and non-ambulant **Label:** SMA TYPE 3 ### Outcomes Module #### Primary Outcomes **Description:** This is a scale used for the assessment of movement and function of very weak infants with SMA type 1. It consists of 16 items of motor function graded 0-4 with a maximum achievable score of 64. **Measure:** Physiotherapy assessment using The Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP INTEND) **Time Frame:** 1 year **Description:** This scale assesses motor performance in infants born pre-term to 4 months of age. It consists of 29 items, with 3 item sets (screening, easy and hard sets). **Measure:** Physiotherapy assessment using the Test of Infant Motor Performance Screening Items (TIMPSI) **Time Frame:** 1 year #### Secondary Outcomes **Description:** The patients will be selected depending on availability and willingness to participate.The interviews will be performed at the most convenient time for the patient. They will be done either face to face, through a phone call or videoconference. **Measure:** Patient´s perception about the condition, interventions performed and Standards of Care assessed by patient interviews **Time Frame:** 1 year **Description:** This is a questionnaire which assesses overall health, pain and ability to participate in activities of daily living. It can be scored relating to eight scales - upper extremity and physical function, transfers and basic mobility, sports/physical functioning, pain/comfort, treatment expectations, happiness, satisfaction with symptoms, global functioning. **Measure:** Patients perception about the condition, interventions performed and Standards of Care assessed using the Paediatric Outcomes Data Collection Instrument (PODCI) **Time Frame:** 1 year **Description:** This is a questionnaire to be used for non-ambulant patients and consists of a series of 17 questions reporting on physical function including ability to transfer, cough, swallowing, fatigue and arm function. **Measure:** Patients perception about the condition, interventions performed and Standards of Care assessed using the Egan Klassifikation Scale (EK2) **Time Frame:** 1 year ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * It will be genetically confirmed SMA Exclusion Criteria: * Involvement in clinical trials is not an exclusion criterion nor having had surgical procedures. Patients who are participating in clinical trials with novel treatments will also be included in the database although the data from this subgroup won't be analysed in the natural history study, nor shared with pharmaceutical companies and other third parties as part of the ISMAC collaboration. **Maximum Age:** 99 Years **Minimum Age:** 0 Months **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT **Study Population:** Patients with genetically confirmed SMA types 0, I, II and III (ambulant and non-ambulant) followed at each open and recruiting neuromuscular centre. ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Salma Samsuddin **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** MariaCristina Scoto **Role:** CONTACT #### Locations **Location 1:** **City:** London **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Salma Samsuddin - **Role:** CONTACT ***Contact 2:*** - **Name:** Francesco Muntoni - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United Kingdom **Facility:** Dubowitz Neuromuscular Centre **Status:** RECRUITING **Zip:** WC1N 1EH ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020763 - Term: Pathological Conditions, Anatomical - ID: D000020879 - Term: Neuromuscular Manifestations - ID: D000009461 - Term: Neurologic Manifestations - ID: D000009422 - Term: Nervous System Diseases - ID: D000013118 - Term: Spinal Cord Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000016472 - Term: Motor Neuron Disease - ID: D000019636 - Term: Neurodegenerative Diseases - ID: D000009468 - Term: Neuromuscular Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M12090 - Name: Muscular Atrophy - Relevance: HIGH - As Found: Muscular Atrophy - ID: M12091 - Name: Muscular Atrophy, Spinal - Relevance: HIGH - As Found: Spinal Muscular Atrophy - ID: M4589 - Name: Atrophy - Relevance: HIGH - As Found: Atrophy - 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 - ID: M15915 - Name: Spinal Cord Diseases - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: M18879 - Name: Motor Neuron Disease - Relevance: LOW - As Found: Unknown - ID: M4024 - Name: Amyotrophic Lateral Sclerosis - Relevance: LOW - As Found: Unknown - ID: M21558 - Name: Neurodegenerative Diseases - Relevance: LOW - As Found: Unknown - ID: M12411 - Name: Neuromuscular Diseases - Relevance: LOW - As Found: Unknown - ID: T5342 - Name: Spinal Muscular Atrophy - Relevance: HIGH - As Found: Spinal Muscular Atrophy - ID: T349 - Name: Amyotrophic Lateral Sclerosis - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000009133 - Term: Muscular Atrophy - ID: D000009134 - Term: Muscular Atrophy, Spinal - ID: D000001284 - Term: Atrophy ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03933579 **Acronym:** PHOENIKS **Brief Title:** The PAH Platform for Deep Phenotyping in Korean Subjects **Official Title:** A Nation-wide Multicenter Registry and Biobank Program for Deep Phenotyping of Idiopathic and Hereditary Pulmonary Arterial Hypertension in Korea: the PAH Platform for Deep Phenotyping in Korean Subjects (PHOENIKS) Cohort #### Organization Study ID Info **ID:** WJC-IIT-1002 #### Organization **Class:** OTHER **Full Name:** Gachon University Gil Medical Center ### Status Module #### Completion Date **Date:** 2020-12-31 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2019-05-01 **Type:** ACTUAL **Last Update Submit Date:** 2019-04-28 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2020-12-31 **Type:** ESTIMATED #### Start Date **Date:** 2018-03-01 **Type:** ACTUAL **Status Verified Date:** 2019-04 #### Study First Post Date **Date:** 2019-05-01 **Type:** ACTUAL **Study First Submit Date:** 2019-04-10 **Study First Submit QC Date:** 2019-04-28 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Sejong General Hospital **Class:** OTHER **Name:** Chonnam National University Hospital **Class:** OTHER **Name:** Keimyung University Dongsan Medical Center **Class:** OTHER **Name:** Saint Vincent's Hospital, Korea **Class:** OTHER **Name:** Seoul National University **Class:** OTHER **Name:** Chungnam National University Hospital **Class:** OTHER **Name:** Wonju Severance Christian Hospital **Class:** OTHER **Name:** Asan Medical Center **Class:** OTHER **Name:** Wonkwang University Hospital **Class:** OTHER **Name:** Chungbuk National University Hospital **Class:** OTHER **Name:** Samsung Medical Center **Class:** OTHER **Name:** Severance Hospital **Class:** OTHER **Name:** The Catholic University of Korea **Class:** OTHER **Name:** Seoul National University Bundang Hospital **Class:** OTHER **Name:** Pusan National University Hospital **Class:** OTHER **Name:** Chonbuk National University Hospital **Class:** OTHER **Name:** Pusan National University Yangsan Hospital #### Lead Sponsor **Class:** OTHER **Name:** Gachon University Gil Medical Center #### Responsible Party **Investigator Affiliation:** Gachon University Gil Medical Center **Investigator Full Name:** Wook-Jin Chung **Investigator Title:** Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** A total of 16 regional hospitals will be registering clinical data and biological specimens of idiopathic pulmonary arterial hypertension (IPAH)/heritable pulmonary arterial hypertension (HPAH) patients across Korea. The diagnosis of pulmonary arterial hypertension(PAH) will be based on right heart catheterization, where PAH caused by etiology other than HPAH or IPAH will be excluded. All clinical data will be stored to a government-based online database. Each participating hospitals will be collecting whole blood from each patient, through which DNA, RNA, serum, plasma, and peripheral blood mononuclear cells will be extracted from the buffy coat layer for further multi-omics analysis. **Detailed Description:** Study Objectives The current study is a multicenter registry and biobank based on South Korean populations to construct a database for the elucidation the molecular and genetic modifiers of PAH. The ultimate goal is to utilize deep phenotypic data for prognosis prediction and discovery of biomarkers and targeted therapies. Study Sample The current study will initially exclude the most frequent form of PAH in Korea, the connective tissue disease (CTD)-related PAH, and focus on the diagnosis and deep phenotyping of idiopathic PAH (IPAH) and heritable PAH (HPAH) patients. The inclusion criteria are as follows: (1) over 18 years of age, (2) mean pulmonary arterial pressure of 25mmHg or higher confirmed by right heart catheterization (RHC), (3) pulmonary vascular resistance ≥ 240 dynes∙s∙cm-5, (4) left ventricle diastolic pressure (LVDEP) or pulmonary capillary wedge pressure (PCWP) ≤ 15mmHg. Exclusion criteria are: (1) patients with drug-induced-PAH, (2) CTD, human immunodeficiency virus (HIV) infection, portal hypertension, congenital heart disease, or Schistosomiasis associated-PAH, (3) long-term responders to calcium channel blockers, (4) PAH patients with overt features of venous capillaries involvement, leaving IPAH and HPAH patients among group 1 of PH to be evaluated. HPAH will be diagnosed by identifying patients with heterozygous pathogenic variants of predetermined genes such as BMPR2, ACVRL1, ENG, CAV1, SMAD1, SMAD4, SMAD9, KCNK3, and EIF2AK4. Patients without a specific genetic mutation will then be categorized as IPAH patients. The genomic data of family members across 3 pedigrees of an HPAH patient will also be analyzed. All clinical and biological data will be reported to a customized web-based case report form called the iCReaT system managed by the Korean Center for Disease Control. All collected biospecimens will be stored at the Korean National Institute of Health main storage. Although the current analysis is planned to be limited to IPAH and HPAH patients, our steering committee plans to expand to all types of PAH in subsequent studies. This study was approved by the institutional ethics committee of each participating institutions and complied with the Declaration of Helsinki (6th revision). An informed consent will be received by each patient. Baseline Data and Biospecimen Collection The baseline data from the registered patients across the 16 regional hospitals will include the following: WHO functional classification, 6-minute walking test, blood sample, electrocardiogram, chest X-ray, echocardiography, optional pulmonary-cardio exercise test, optional Cardiac MRI, and RHC. Detailed information of each exams are specified in Table 2. The registered patient will be followed up on a regular basis for further data and biospecimen collection. With the patient's blood sample, DNA, RNA, serum, plasma, and peripheral blood mononuclear cells (PBMC) from the buffy coat will be separated and be extracted for further storage and studies. For the DNA sample, 2.5ml of the whole blood will be stored in a PAX gene DNA tube at the collecting site, and it will be carried to the main center at 4\~10 C. At the main center, it will, then, be transferred to a 2ml cryotube to be stored at -70\~80C. For patient's RNA collection, 2.5 mL of whole blood will be collected at PAX gene RNA tube, and it will be sent to the main center at 4\~10 C, which will also be further transferred to a cryotube to be stored at -70\~80C. Patient's serum, plasma and the buffy coat will also be collected. At the respective collection sites, serum will be collected at a serum separation tube, and after 30 minutes of venipuncture, it will be centrifuged and stored at -20C. For patient's plasma, cell preparation tube will be used within 2 hours of blood collection. Then, each sample will be ready to be stored at -20C by treating the samples with human serum type antibody, DMSO, and freezing medium. To separate and extract PBMC, white buffy coat layer will be separated and be stored at a 1.5ml tube with a freezing medium at -20C. The collected baseline data will then go through a clean-up process and further evaluation of its quality will be done. In addition, five patients will be selected to do a whole-genome sequencing. All the collected biospecimen will be donated to NIH center storage, and will be further used for a multi-omics studies in order to deep phenotype the patient's specimens. Patient Follow Up All patients will be followed up twice or more a year, with expected 80% follow up rate. During the second and third year, patient registry and data collection will be continued with the same protocol. To maintain the credibility of collected data, the steering committee will continuously monitor and audit the collected data. Protocol may be further crafted after evaluating the previous year's data. In addition, effective data management strategies, including a guideline to standardize patient's body measurement and blood sample will be developed. Ten patient samples will be selected for a next generation sequencing, and we will be utilized in discovering novel genetic mutations. Genetic Mutation Analysis across Three-Pedigrees for HPAH Patients In order to determine patients with HPAH, a familial genetic study will be performed. A three-generation pedigree for each PAH patient with with or without the existence of a BMPR2, ACVRL1, ENG, CAV1, SMAD1, SMAD4, SMAD9, KCNK3, and/or EIF2AK4 mutation will be evaluated. Blood samples from family members of patients with a HPAH will be collected for genetic screening. ### Conditions Module **Conditions:** - Pulmonary Arterial Hypertension - Deep Phenotyping - Idiopathic Pulmonary Arterial Hypertension - Heritable Pulmonary Arterial Hypertension ### Design Module #### Bio Spec **Description:** Each participating hospitals will be collecting whole blood from each patient, through which DNA, RNA, serum, plasma, and peripheral blood mononuclear cells will be extracted from the buffy coat layer for further multi-omics analysis. **Retention:** SAMPLES_WITH_DNA #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 500 **Type:** ESTIMATED **Patient Registry:** True **Study Type:** OBSERVATIONAL **Target Duration:** 3 Years ### Arms Interventions Module ### Interventions #### Intervention 1 **Description:** Each participating hospitals will be collecting whole blood from each patient, through which DNA, RNA, serum, plasma, and peripheral blood mononuclear cells will be extracted from the buffy coat layer for further multi-omics analysis. **Name:** Deep phenotyping **Type:** DIAGNOSTIC_TEST ### Outcomes Module #### Primary Outcomes **Description:** A composite of death and hospitalization **Measure:** Composite outcomes of death and hospitalization **Time Frame:** 3 years #### Secondary Outcomes **Description:** 6 minute walk test **Measure:** 6 minute walk test **Time Frame:** 3 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Over 18 years * Mean pulmonary arterial pressure of 25mmHg or higher confirmed by right heart catheterization (RHC) * Pulmonary vascular resistance ≥ 240 dynes∙s∙cm-5 * Left ventricle diastolic pressure (LVDEP) or pulmonary capillary wedge pressure (PCWP) ≤ 15mmHg Exclusion Criteria: * Patients with drug-induced-PAH * Patients with CTD, human immunodeficiency virus (HIV) infection, portal hypertension, congenital heart disease, or Schistosomiasis associated- PAH * Long-term responders to calcium channel blockers * PAH patients with overt features of venous capillaries involvement **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Idiopathic PAH and heritable PAH patients in Korea ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Wook-Jin Chung, MD **Phone:** 82-32-460-3663 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Albert Y Jang, MD **Phone:** 82-32-460-3663 **Role:** CONTACT #### Locations **Location 1:** **City:** Incheon **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Mi Ju Yu, BS - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Gyeong-Lim Hyun, BS - **Role:** CONTACT **Country:** Korea, Republic of **Facility:** Department of Cardiovascular Medicine, Gachon University, Gil Medical Center **Status:** RECRUITING ### IPD Sharing Statement Module **Description:** We are planning to share data **IPD Sharing:** UNDECIDED ### References Module #### References **Citation:** Jang AY, Kim S, Park SJ, Choi H, Oh PC, Oh S, Kim KH, Kim KH, Byun K, Chung WJ; PHOENIKS Investigators. A Nationwide multicenter registry and biobank program for deep phenotyping of idiopathic and hereditary pulmonary arterial hypertension in Korea: the PAH platform for deep phenotyping in Korean subjects (PHOENIKS) cohort. Clin Hypertens. 2019 Sep 15;25:21. doi: 10.1186/s40885-019-0126-8. eCollection 2019. **PMID:** 31534782 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014652 - Term: Vascular Diseases - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000006976 - Term: Hypertension, Pulmonary - ID: D000008171 - Term: Lung Diseases - ID: D000012140 - Term: Respiratory Tract Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M10024 - Name: Hypertension - Relevance: HIGH - As Found: Hypertension - ID: M2261 - Name: Pulmonary Arterial Hypertension - Relevance: HIGH - As Found: Pulmonary Arterial Hypertension - ID: M30541 - Name: Familial Primary Pulmonary Hypertension - Relevance: HIGH - As Found: Idiopathic Pulmonary Arterial Hypertension - ID: M10027 - Name: Hypertension, Pulmonary - Relevance: LOW - As Found: Unknown - ID: M17400 - Name: Vascular 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: T4807 - Name: Pulmonary Arterial Hypertension - Relevance: HIGH - As Found: Pulmonary Arterial Hypertension ### Condition Browse Module - Meshes - ID: D000081029 - Term: Pulmonary Arterial Hypertension - ID: D000065627 - Term: Familial Primary Pulmonary Hypertension - ID: D000006973 - Term: Hypertension ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02126579 **Acronym:** MEL60 **Brief Title:** Phase I/II Trial of a Long Peptide Vaccine (LPV7) Plus TLR Agonists **Official Title:** Open Label, Randomized, Phase I/II Study of a Long Peptide Vaccine Plus TLR Agonists for Resected Stage IIb-IV Melanoma. (MEL60) #### Organization Study ID Info **ID:** 15931 #### Organization **Class:** OTHER **Full Name:** University of Virginia #### Secondary ID Infos **Domain:** UVA Human Immune Therapy Center **ID:** MEL60 **Type:** OTHER ### Status Module #### Completion Date **Date:** 2021-05-05 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2024-02-28 **Type:** ACTUAL **Last Update Submit Date:** 2024-02-02 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2019-11-20 **Type:** ACTUAL #### Results First Post Date **Date:** 2023-11-03 **Type:** ACTUAL **Results First Submit Date:** 2022-04-28 **Results First Submit QC Date:** 2023-10-12 #### Start Date **Date:** 2014-05-01 **Type:** ACTUAL **Status Verified Date:** 2024-02 #### Study First Post Date **Date:** 2014-04-30 **Type:** ESTIMATED **Study First Submit Date:** 2014-04-24 **Study First Submit QC Date:** 2014-04-28 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** University of Virginia #### Lead Sponsor **Class:** OTHER **Name:** Craig L Slingluff, Jr #### Responsible Party **Investigator Affiliation:** University of Virginia **Investigator Full Name:** Craig L Slingluff, Jr **Investigator Title:** Professor, Department of Surgery **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of this study is to learn what effects (good and bad) an experimental vaccine (LPV7) plus tetanus peptide and other substances called polyICLC, resiquimod, and Montanide ISA-51 have on you and your melanoma. We will also look at whether the experimental vaccine and these drugs cause any changes in your immune system. ### Conditions Module **Conditions:** - Melanoma - Metastatic Melanoma - Mucosal Melanoma **Keywords:** - melanoma - neoplasms - Poly ICLC - Freund's Adjuvant - Metastatic melanoma - resiquimod - adjuvants - peptide vaccine ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 50 **Type:** ACTUAL **Phases:** - PHASE1 - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA administered in one skin location rotated to different sites on an extremity clinically uninvolved with melanoma. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. **Intervention Names:** - Biological: Peptide Vaccine (LPV7) + Tetanus peptide - Other: IFA **Label:** Arm A (Part 1) **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC vaccine administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. **Intervention Names:** - Biological: Peptide Vaccine (LPV7) + Tetanus peptide - Other: PolyICLC **Label:** Arm B (Part 1) **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Peptide Vaccine (LPV7) + Tetanus peptide vaccine administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after the vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. **Intervention Names:** - Biological: Peptide Vaccine (LPV7) + Tetanus peptide - Other: Resiquimod **Label:** Arm C (Part 1) **Type:** EXPERIMENTAL #### Arm Group 4 **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. **Intervention Names:** - Biological: Peptide Vaccine (LPV7) + Tetanus peptide - Other: PolyICLC - Other: Resiquimod **Label:** Arm D (Part 1) **Type:** EXPERIMENTAL #### Arm Group 5 **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA + PolyICLC vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. **Intervention Names:** - Biological: Peptide Vaccine (LPV7) + Tetanus peptide - Other: PolyICLC - Other: IFA **Label:** Arm E (Part 1) **Type:** EXPERIMENTAL #### Arm Group 6 **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. **Intervention Names:** - Biological: Peptide Vaccine (LPV7) + Tetanus peptide - Other: Resiquimod - Other: IFA **Label:** Arm F (Part 1) **Type:** EXPERIMENTAL #### Arm Group 7 **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC + IFA vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. **Intervention Names:** - Biological: Peptide Vaccine (LPV7) + Tetanus peptide - Other: PolyICLC - Other: Resiquimod - Other: IFA **Label:** Arm G(Part 1) **Type:** EXPERIMENTAL #### Arm Group 8 **Description:** Peptide Vaccine (LPV7) + IFA + PolyICLC vaccines administered in one skin location. Each vaccine will be administered in the same skin site for all 6 vaccines. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. **Intervention Names:** - Biological: Peptide Vaccine (LPV7) + Tetanus peptide - Other: PolyICLC - Other: IFA **Label:** Arm E2 **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Arm A (Part 1) - Arm B (Part 1) - Arm C (Part 1) - Arm D (Part 1) - Arm E (Part 1) - Arm E2 - Arm F (Part 1) - Arm G(Part 1) **Description:** 1.5 mL administered half intradermally and half subcutaneously. **Name:** Peptide Vaccine (LPV7) + Tetanus peptide **Type:** BIOLOGICAL #### Intervention 2 **Arm Group Labels:** - Arm B (Part 1) - Arm D (Part 1) - Arm E (Part 1) - Arm E2 - Arm G(Part 1) **Description:** 1 mL administered half intradermally and half subcutaneously **Name:** PolyICLC **Type:** OTHER #### Intervention 3 **Arm Group Labels:** - Arm C (Part 1) - Arm D (Part 1) - Arm F (Part 1) - Arm G(Part 1) **Description:** 500 mg applied to vaccine site after vaccine administration **Name:** Resiquimod **Type:** OTHER #### Intervention 4 **Arm Group Labels:** - Arm A (Part 1) - Arm E (Part 1) - Arm E2 - Arm F (Part 1) - Arm G(Part 1) **Description:** 2 mL administered half intradermally and half subcutaneously **Name:** IFA **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Safety and toxicity following vaccination with 7 long peptides in melanoma patients with and without TLR agonists. Patients are evaluated by safety labs and physical exams to assess for toxicity. **Measure:** Number of Participants With Treatment-related Adverse Events Per Study Arm **Time Frame:** 6 months **Description:** Levels of peptide-reactive CD8+ T cells in the peripheral blood: number of participants with T cell response to minimal epitope for CD8 T cells. This was assessed by direct (ex vivo) IFN-gamma ELIspot assay for reactivity to known minimal epitopes. To be considered positive, there had to be an increase compared to the maximum negative control target by at least 2-fold and by at least 20 IFN-gamma secreting cells per 100,000 CD8 T cells evaluated. **Measure:** T Cell Response in Peripheral Blood Over Duration of Study Participation **Time Frame:** 6 months #### Secondary Outcomes **Description:** CD4+ T cell responses to peptides in the vaccine, and their function **Measure:** T Cell Response and Function in Peripheral Blood **Time Frame:** 6 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Histologically or cytologically proven Stage IIB - IV melanoma rendered clinically free of disease by surgery, other therapy, or spontaneous remission within 6 months prior to registration. * Patients may have had melanoma from a cutaneous, mucosal or unknown primary site * Patients with small radiologic or clinical findings may be eligible * Patients with treated brain metastases may be eligible if the following are true: * Total number of brain metastases ever is less than or equal to 3 * The brain metastases have been completely removed by surgery or have been treated completely with stereotactic radiotherapy * There has been no evident growth of any brain metastases since treatment * No treated brain metastases is greater than 2 cm at the time of protocol entry * Patients must have at least 1 intact axillary and/or inguinal lymph node basin * ECOG performance status of 0-1 * Lab parameters as follows: * HLA-A1, A2, A3, B35, or B51 * ANC \> 1000/mm3 and Platelets \> 100,000/mm3 and Hemoglobin \> 9 g/dL * AST and ALT up to 2.5 x ULN * Bilirubin up to 2.5 x ULN * Alkaline Phosphatase up to 2.5 x ULN * Creatinine up to 1.5 x ULN * HGBA1C level ≤ 7.5% Exclusion Criteria: * Patients with melanoma from a uveal or ocular primary site * Patients currently receiving any systemic therapy within 4 weeks of study registration. Gamma knife or stereotactic radiosurgery must not be administered within 1 week prior to study registration. Patients who are currently receiving nitrosoureas within the preceding 6 weeks. * Patients who have received CTLA-4, PD-1, PD-L1, CD137, or CD27 within the prior 12 months. * Patients with known or suspected allergy to any component of the vaccine * HIV positive or active Hepatitis C virus * Patients receiving any of the following medications within 4 weeks are excluded: * Agents with immunomodulating activity (with the exception of non-steroidal anti-inflammatory agents and topical steroids) * Allergy desensitization injections * Systemic corticosteroids, administered parenterally or orally. Inhaled steroids (e.g. Advair, Flovent, Azmacort) are not permitted. Topical corticosteroids are acceptable including steroids with very low solubility administered nasally for local effects only (e.g. Nasonex) * Any growth factors (e.g. GM-CSF, G-CSF, erythropoietin). * Interferon therapy * Interleukin-2 or other interleukins * Other investigational drugs or investigational therapy if currently receiving or have received within 1 month * Pregnancy or the possibility of becoming pregnant during the study. And women who are breastfeeding. * Must not have had prior autoimmune disorders requiring cytotoxic or immunosuppressive therapy, or autoimmune disorders with visceral involvement. The following are not exclusionary: * Presence of laboratory evidence of autoimmune disease (e.g. positive ANA titer) without symptoms * Clinical evidence of vitiligo * Other forms of depigmenting illness * Mild arthritis requiring NSAID medications * Patients with a medical contradiction or potential problem with complying with the protocol, in the opinion of the investigator * Patients with Class III or IV heart disease (according to NYHA classification) * Patients with a body weight \< 110 lbs. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Houston **Country:** United States **Facility:** MDAnderson Cancer Center **State:** Texas **Zip:** 77030 **Location 2:** **City:** Charlottesville **Country:** United States **Facility:** University of Virginia **State:** Virginia **Zip:** 22908 #### Overall Officials **Official 1:** **Affiliation:** University of Virginia **Name:** Craig L Slingluff, Jr., M.D. **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Patel SP, Petroni GR, Roszik J, Olson WC, Wages NA, Chianese-Bullock KA, Smolkin M, Varhegyi N, Gaughan E, Smith KT, Haden K, Hall EH, Gnjatic S, Hwu P, Slingluff CL. Phase I/II trial of a long peptide vaccine (LPV7) plus toll-like receptor (TLR) agonists with or without incomplete Freund's adjuvant (IFA) for resected high-risk melanoma. J Immunother Cancer. 2021 Aug;9(8):e003220. doi: 10.1136/jitc-2021-003220. **PMID:** 34413169 #### See Also Links **Label:** NCI website **URL:** http://www.cancer.gov/cancertopics/types/melanoma ## Document Section ### Large Document Module #### Large Docs - Date: 2017-07-31 - Filename: Prot_SAP_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: True - Label: Study Protocol and Statistical Analysis Plan - Size: 1124978 - Type Abbrev: Prot_SAP - Upload Date: 2021-10-03T22:52 - Date: 2019-06-11 - Filename: ICF_001.pdf - Has ICF: True - Has Protocol: False - Has SAP: False - Label: Informed Consent Form - Size: 1153234 - Type Abbrev: ICF - Upload Date: 2021-10-03T22:55 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000018358 - Term: Neuroendocrine Tumors - ID: D000017599 - Term: Neuroectodermal Tumors - ID: D000009373 - Term: Neoplasms, Germ Cell and Embryonal - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000009369 - Term: Neoplasms - ID: D000009380 - Term: Neoplasms, Nerve Tissue - ID: D000018326 - Term: Nevi and Melanomas - ID: D000012878 - Term: Skin Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000012871 - Term: Skin Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M11528 - Name: Melanoma - Relevance: HIGH - As Found: Melanoma - ID: M20495 - Name: Neuroendocrine Tumors - Relevance: LOW - As Found: Unknown - ID: M19845 - Name: Neuroectodermal Tumors - Relevance: LOW - As Found: Unknown - ID: M20388 - Name: Neuroectodermal Tumors, Primitive - Relevance: LOW - As Found: Unknown - ID: M12318 - Name: Neoplasms, Germ Cell and Embryonal - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown - ID: M12325 - Name: Neoplasms, Nerve Tissue - Relevance: LOW - As Found: Unknown - ID: M12448 - Name: Nevus, Pigmented - Relevance: LOW - As Found: Unknown - ID: M12446 - Name: Nevus - Relevance: LOW - As Found: Unknown - ID: M20470 - Name: Nevi and Melanomas - Relevance: LOW - As Found: Unknown - ID: M15681 - Name: Skin Neoplasms - Relevance: LOW - As Found: Unknown - ID: M15674 - Name: Skin Diseases - Relevance: LOW - As Found: Unknown - ID: T4091 - Name: Neuroendocrine Tumor - Relevance: LOW - As Found: Unknown - ID: T4092 - Name: Neuroepithelioma - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000008545 - Term: Melanoma ### Intervention Browse Module - Ancestors - ID: D000097912 - Term: Toll-Like Receptor Agonists - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000091369 - Term: Immunomodulating Agents - ID: D000007155 - Term: Immunologic Factors - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000007369 - Term: Interferon Inducers ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: ANeo - Name: Antineoplastic Agents ### Intervention Browse Module - Browse Leaves - ID: M17360 - Name: Vaccines - Relevance: LOW - As Found: Unknown - ID: M280480 - Name: Poly ICLC - Relevance: HIGH - As Found: Activity scheduling - ID: M8740 - Name: Freund's Adjuvant - Relevance: LOW - As Found: Unknown - ID: M354039 - Name: Resiquimod - Relevance: HIGH - As Found: Pulse Pressure - ID: M2853 - Name: Immunomodulating Agents - Relevance: LOW - As Found: Unknown - ID: M10201 - Name: Immunologic Factors - Relevance: LOW - As Found: Unknown - ID: M10407 - Name: Interferons - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: C000402365 - Term: Resiquimod - ID: C000019531 - Term: Poly ICLC ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** All adverse events (treatment-related or not), All serious adverse events (treatment-related or not), and All Cause Mortality on study #### Event Groups **Group ID:** EG000 **Title:** Arm A (Part 1) **Deaths Num At Risk:** 5 **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA administered in one skin location rotated to different sites on an extremity clinically uninvolved with melanoma. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. IFA: 2 mL administered half intradermally and half subcutaneously **ID:** EG000 **Other Num Affected:** 5 **Other Num at Risk:** 5 **Serious Number At Risk:** 5 **Title:** Arm A (Part 1) **Group ID:** EG001 **Title:** Arm B (Part 1) **Deaths Num Affected:** 1 **Deaths Num At Risk:** 7 **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC vaccine administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously **ID:** EG001 **Other Num Affected:** 7 **Other Num at Risk:** 7 **Serious Number At Risk:** 7 **Title:** Arm B (Part 1) **Group ID:** EG002 **Title:** Arm C (Part 1) **Deaths Num Affected:** 1 **Deaths Num At Risk:** 4 **Description:** Peptide Vaccine (LPV7) + Tetanus peptide vaccine administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after the vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. Resiquimod: 500 mg applied to vaccine site after vaccine administration **ID:** EG002 **Other Num Affected:** 4 **Other Num at Risk:** 4 **Serious Number At Risk:** 4 **Title:** Arm C (Part 1) **Group ID:** EG003 **Title:** Arm D (Part 1) **Deaths Num Affected:** 2 **Deaths Num At Risk:** 6 **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously Resiquimod: 500 mg applied to vaccine site after vaccine administration **ID:** EG003 **Other Num Affected:** 6 **Other Num at Risk:** 6 **Serious Number At Risk:** 6 **Title:** Arm D (Part 1) **Group ID:** EG004 **Title:** Arm E (Part 1 + 2) **Deaths Num Affected:** 1 **Deaths Num At Risk:** 16 **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA + PolyICLC vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously IFA: 2 mL administered half intradermally and half subcutaneously Except that one participant on Part 2 did not have tetanus peptide in the vaccine, and that participants vaccines were all administered at the same vaccine site. **ID:** EG004 **Other Num Affected:** 16 **Other Num at Risk:** 16 **Serious Number At Risk:** 16 **Title:** Arm E (Part 1 + 2) **Group ID:** EG005 **Title:** Arm F (Part 1) **Deaths Num At Risk:** 6 **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. Resiquimod: 500 mg applied to vaccine site after vaccine administration IFA: 2 mL administered half intradermally and half subcutaneously **ID:** EG005 **Other Num Affected:** 6 **Other Num at Risk:** 6 **Serious Number At Risk:** 6 **Title:** Arm F (Part 1) **Group ID:** EG006 **Title:** Arm G(Part 1) **Deaths Num Affected:** 2 **Deaths Num At Risk:** 6 **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC + IFA vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously Resiquimod: 500 mg applied to vaccine site after vaccine administration IFA: 2 mL administered half intradermally and half subcutaneously **ID:** EG006 **Other Num Affected:** 6 **Other Num at Risk:** 6 **Serious Number At Risk:** 6 **Title:** Arm G(Part 1) **Frequency Threshold:** 0 #### Other Events **Term:** Other **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Tinnitus **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Ear and labyrinth disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Constipation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Diarrhea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Mucositis Oral **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Nausea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Vomiting **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Other **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Chills **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Fatigue **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Fever **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Flu-like symptoms **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Injection site reaction **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Localized edema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Other **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Autoimmune disorder **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Immune system disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Bruising **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** CTCAE (4.0) **Term:** lymphocyte count decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** CTCAE (4.0) **Term:** weight loss **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** CTCAE (4.0) **Term:** white blood cell decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** CTCAE (4.0) **Term:** Anorexia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Arthralgia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Generalized muscle weakness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Myalgia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Other **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Pain in extremity **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Dizziness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Dysgeusia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Anxiety **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Allergic rhinitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Cough **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Dyspnea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Sore throat **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Hyperhidrosis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Pain of skin **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Pruritis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Rash - maculopapular **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** CTCAE (4.0) **Term:** scalp pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Skin hyperpigmentation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Hypopigmentation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Skin induration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Skin ulceration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Flushing **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Hot flashes **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Hematoma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Hypertension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Anemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Chest pain - cardiac **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Other **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Blurred vision **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Flashing lights **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Floaters **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Night blindness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Other **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Watering eyes **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Dry mouth **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Dysphagia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Edema limbs **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Edema trunk **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Other **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** CTCAE (4.0) **Term:** Sepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** CTCAE (4.0) **Term:** Skin infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** CTCAE (4.0) **Term:** Venous Injury **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** CTCAE (4.0) **Term:** Aspartate aminotransferase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** CTCAE (4.0) **Term:** Weight gain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** CTCAE (4.0) **Term:** Hyperglycemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Hyperkalemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Hypoglycemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Hyponatremia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Bone pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Paresthesia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Peripheral sensory neuropathy **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Presyncope **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Agitation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Depression **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Insomnia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Other **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Renal colic **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Other **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Nasal congestion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Dry skin **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Urticaria **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** CTCAE (4.0) **Term:** Other **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** CTCAE (4.0) **Time Frame:** 6 months ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 5 **Group ID:** BG001 **Value:** 7 **Group ID:** BG002 **Value:** 4 **Group ID:** BG003 **Value:** 6 **Group ID:** BG004 **Value:** 16 **Group ID:** BG005 **Value:** 6 **Group ID:** BG006 **Value:** 6 **Group ID:** BG007 **Value:** 50 **Units:** Participants ### Group **ID:** BG000 **Title:** Arm A (Part 1) **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA administered in one skin location rotated to different sites on an extremity clinically uninvolved with melanoma. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. IFA: 2 mL administered half intradermally and half subcutaneously ### Group **ID:** BG001 **Title:** Arm B (Part 1) **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC vaccine administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously ### Group **ID:** BG002 **Title:** Arm C (Part 1) **Description:** Peptide Vaccine (LPV7) + Tetanus peptide vaccine administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after the vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. Resiquimod: 500 mg applied to vaccine site after vaccine administration ### Group **ID:** BG003 **Title:** Arm D (Part 1) **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously Resiquimod: 500 mg applied to vaccine site after vaccine administration ### Group **ID:** BG004 **Title:** Arm E (Part 1 + 2) **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA + PolyICLC vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously IFA: 2 mL administered half intradermally and half subcutaneously. Except that the last patient, on Part 2 received all 6 vaccines in the same site and did not receive tetanus peptide. ### Group **ID:** BG005 **Title:** Arm F (Part 1) **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. Resiquimod: 500 mg applied to vaccine site after vaccine administration IFA: 2 mL administered half intradermally and half subcutaneously ### Group **ID:** BG006 **Title:** Arm G(Part 1) **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC + IFA vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously Resiquimod: 500 mg applied to vaccine site after vaccine administration IFA: 2 mL administered half intradermally and half subcutaneously ### Group **ID:** BG007 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 33 **Upper Limit:** 62 **Value:** 52 #### Measurement **Group ID:** BG001 **Lower Limit:** 21 **Upper Limit:** 69 **Value:** 64 #### Measurement **Group ID:** BG002 **Lower Limit:** 44 **Upper Limit:** 76 **Value:** 64 #### Measurement **Group ID:** BG003 **Lower Limit:** 19 **Upper Limit:** 67 **Value:** 53 #### Measurement **Group ID:** BG004 **Lower Limit:** 24 **Upper Limit:** 71 **Value:** 60 #### Measurement **Group ID:** BG005 **Lower Limit:** 37 **Upper Limit:** 65 **Value:** 56 #### Measurement **Group ID:** BG006 **Lower Limit:** 49 **Upper Limit:** 71 **Value:** 59 #### Measurement **Group ID:** BG007 **Lower Limit:** 19 **Upper Limit:** 76 **Value:** 58 **Class Title:** Median Age ### Measure #### Measurement **Group ID:** BG000 **Value:** 2 #### Measurement **Group ID:** BG001 **Value:** 3 #### Measurement **Group ID:** BG002 **Value:** 2 #### Measurement **Group ID:** BG003 **Value:** 1 #### Measurement **Group ID:** BG004 **Value:** 9 #### Measurement **Group ID:** BG005 **Value:** 0 #### Measurement **Group ID:** BG006 **Value:** 3 #### Measurement **Group ID:** BG007 **Value:** 20 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 4 #### Measurement **Group ID:** BG002 **Value:** 2 #### Measurement **Group ID:** BG003 **Value:** 5 #### Measurement **Group ID:** BG004 **Value:** 7 #### Measurement **Group ID:** BG005 **Value:** 6 #### Measurement **Group ID:** BG006 **Value:** 3 #### Measurement **Group ID:** BG007 **Value:** 30 **Category Title:** Male **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 #### Measurement **Group ID:** BG004 **Value:** 0 #### Measurement **Group ID:** BG005 **Value:** 0 #### Measurement **Group ID:** BG006 **Value:** 0 #### Measurement **Group ID:** BG007 **Value:** 0 **Category Title:** Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 5 #### Measurement **Group ID:** BG001 **Value:** 7 #### Measurement **Group ID:** BG002 **Value:** 4 #### Measurement **Group ID:** BG003 **Value:** 6 #### Measurement **Group ID:** BG004 **Value:** 16 #### Measurement **Group ID:** BG005 **Value:** 6 #### Measurement **Group ID:** BG006 **Value:** 6 #### Measurement **Group ID:** BG007 **Value:** 50 **Category Title:** Not Hispanic or Latino #### 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:** 0 #### Measurement **Group ID:** BG006 **Value:** 0 #### Measurement **Group ID:** BG007 **Value:** 0 **Category Title:** Unknown or Not Reported **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 #### Measurement **Group ID:** BG004 **Value:** 1 #### Measurement **Group ID:** BG005 **Value:** 0 #### Measurement **Group ID:** BG006 **Value:** 0 #### Measurement **Group ID:** BG007 **Value:** 1 **Category Title:** American Indian or Alaska Native #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 0 #### Measurement **Group ID:** BG004 **Value:** 0 #### Measurement **Group ID:** BG005 **Value:** 0 #### Measurement **Group ID:** BG006 **Value:** 0 #### Measurement **Group ID:** BG007 **Value:** 0 **Category Title:** Asian #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 0 #### Measurement **Group ID:** BG004 **Value:** 0 #### Measurement **Group ID:** BG005 **Value:** 0 #### Measurement **Group ID:** BG006 **Value:** 0 #### Measurement **Group ID:** BG007 **Value:** 0 **Category Title:** Native Hawaiian or Other Pacific Islander #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 0 #### Measurement **Group ID:** BG004 **Value:** 0 #### Measurement **Group ID:** BG005 **Value:** 0 #### Measurement **Group ID:** BG006 **Value:** 0 #### Measurement **Group ID:** BG007 **Value:** 0 **Category Title:** Black or African American #### Measurement **Group ID:** BG000 **Value:** 5 #### Measurement **Group ID:** BG001 **Value:** 7 #### Measurement **Group ID:** BG002 **Value:** 4 #### Measurement **Group ID:** BG003 **Value:** 6 #### Measurement **Group ID:** BG004 **Value:** 15 #### Measurement **Group ID:** BG005 **Value:** 6 #### Measurement **Group ID:** BG006 **Value:** 6 #### Measurement **Group ID:** BG007 **Value:** 49 **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:** 0 #### Measurement **Group ID:** BG006 **Value:** 0 #### Measurement **Group ID:** BG007 **Value:** 0 **Category Title:** More than one race #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 #### Measurement **Group ID:** BG003 **Value:** 0 #### Measurement **Group ID:** BG004 **Value:** 0 #### Measurement **Group ID:** BG005 **Value:** 0 #### Measurement **Group ID:** BG006 **Value:** 0 #### Measurement **Group ID:** BG007 **Value:** 0 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 5 #### Measurement **Group ID:** BG001 **Value:** 7 #### Measurement **Group ID:** BG002 **Value:** 4 #### Measurement **Group ID:** BG003 **Value:** 6 #### Measurement **Group ID:** BG004 **Value:** 16 #### Measurement **Group ID:** BG005 **Value:** 6 #### Measurement **Group ID:** BG006 **Value:** 6 #### Measurement **Group ID:** BG007 **Value:** 50 **Class Title:** United States **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** FULL_RANGE **Parameter Type:** MEDIAN **Title:** Age, Customized **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 ## Results Section - More Information Module ### Certain Agreement **PI Sponsor Employee:** True ### Point of Contact **Email:** [email protected] **Organization:** University of Virginia **Phone:** 4349249311 **Title:** Craig Slingluff, MD; Professor, Department of Surgery; Program Director, UVA Cancer Center ## 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:** 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:** 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:** 1 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **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:** 1 - **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:** 2 - **Comment:** - **Group ID:** OG006 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 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:** 1 - **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:** 2 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **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:** 0 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG006 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 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:** 1 - **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:** 2 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 12 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 - **Comment:** - **Group ID:** OG006 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 **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:** 5 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 12 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 - **Comment:** - **Group ID:** OG006 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 **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:** 2 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 8 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **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:** 1 - **Comment:** - **Group ID:** OG002 - **Lower 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- **Spread:** - **Upper Limit:** - **Value:** 15 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 6 - **Comment:** - **Group ID:** OG006 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 6 **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:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **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:** 6 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 - **Comment:** - **Group ID:** OG006 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 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:** 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:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 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:** 1 - **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:** 2 - **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:** 0 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **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:** 2 - **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:** 6 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG006 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 **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:** 3 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 7 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **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:** 1 - **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:** 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:** 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:** 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:** 2 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG006 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 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:** 2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 6 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG006 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 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:** 1 - **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:** 1 - **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:** 2 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **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:** 1 - **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:** 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:** 1 - **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:** 1 - **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:** 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:** 1 - **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:** 7 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG006 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 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:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **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:** 1 - **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:** 1 - **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:** 0 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG006 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **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:** 2 - **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:** 1 - **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:** 4 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 14 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG006 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 **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:** 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:** 5 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG006 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **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:** 1 - **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 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG003 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG004 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG006 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 - **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:** 8 - **Comment:** - **Group ID:** OG005 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG006 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Safety and toxicity following vaccination with 7 long peptides in melanoma patients with and without TLR agonists. Patients are evaluated by safety labs and physical exams to assess for toxicity. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** All participants enrolled and treated. **Reporting Status:** POSTED **Time Frame:** 6 months **Title:** Number of Participants With Treatment-related Adverse Events Per Study Arm **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA administered in one skin location rotated to different sites on an extremity clinically uninvolved with melanoma. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. IFA: 2 mL administered half intradermally and half subcutaneously **ID:** OG000 **Title:** Arm A (Part 1) ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC vaccine administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously **ID:** OG001 **Title:** Arm B (Part 1) ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide vaccine administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after the vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. Resiquimod: 500 mg applied to vaccine site after vaccine administration **ID:** OG002 **Title:** Arm C (Part 1) ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously Resiquimod: 500 mg applied to vaccine site after vaccine administration **ID:** OG003 **Title:** Arm D (Part 1) ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA + PolyICLC vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma (except that the vaccines were administered to the same skin site for the one patient from cohort 2) Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. (except that the tetanus peptide was not included in the vaccine for the one patient on this arm from cohort 2. PolyICLC: 1 mL administered half intradermally and half subcutaneously IFA: 2 mL administered half intradermally and half subcutaneously **ID:** OG004 **Title:** Arm E (Part 1 + Part 2) ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. Resiquimod: 500 mg applied to vaccine site after vaccine administration IFA: 2 mL administered half intradermally and half subcutaneously **ID:** OG005 **Title:** Arm F (Part 1) ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC + IFA vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously Resiquimod: 500 mg applied to vaccine site after vaccine administration IFA: 2 mL administered half intradermally and half subcutaneously **ID:** OG006 **Title:** Arm G(Part 1) #### Outcome Measure 2 **Description:** Levels of peptide-reactive CD8+ T cells in the peripheral blood: number of participants with T cell response to minimal epitope for CD8 T cells. This was assessed by direct (ex vivo) IFN-gamma ELIspot assay for reactivity to known minimal epitopes. To be considered positive, there had to be an increase compared to the maximum negative control target by at least 2-fold and by at least 20 IFN-gamma secreting cells per 100,000 CD8 T cells evaluated. **Parameter Type:** COUNT_OF_PARTICIPANTS **Reporting Status:** POSTED **Time Frame:** 6 months **Title:** T Cell Response in Peripheral Blood Over Duration of Study Participation **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA administered in one skin location rotated to different sites on an extremity clinically uninvolved with melanoma. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. IFA: 2 mL administered half intradermally and half subcutaneously **ID:** OG000 **Title:** Arm A (Part 1) ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC vaccine administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously **ID:** OG001 **Title:** Arm B (Part 1) ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide vaccine administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after the vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. Resiquimod: 500 mg applied to vaccine site after vaccine administration **ID:** OG002 **Title:** Arm C (Part 1) ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously Resiquimod: 500 mg applied to vaccine site after vaccine administration **ID:** OG003 **Title:** Arm D (Part 1) ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA + PolyICLC vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously IFA: 2 mL administered half intradermally and half subcutaneously Except that one patient, on Part 2, received all 6 vaccines in the same skin site, and did not have tetanus peptide in the vaccine. **ID:** OG004 **Title:** Arm E (Part 1) ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. Resiquimod: 500 mg applied to vaccine site after vaccine administration IFA: 2 mL administered half intradermally and half subcutaneously **ID:** OG005 **Title:** Arm F (Part 1) ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC + IFA vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously Resiquimod: 500 mg applied to vaccine site after vaccine administration IFA: 2 mL administered half intradermally and half subcutaneously **ID:** OG006 **Title:** Arm G(Part 1) #### Outcome Measure 3 **Description:** CD4+ T cell responses to peptides in the vaccine, and their function **Parameter Type:** COUNT_OF_PARTICIPANTS **Reporting Status:** POSTED **Time Frame:** 6 months **Title:** T Cell Response and Function in Peripheral Blood **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA administered in one skin location rotated to different sites on an extremity clinically uninvolved with melanoma. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. IFA: 2 mL administered half intradermally and half subcutaneously **ID:** OG000 **Title:** Arm A (Part 1) ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC vaccine administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously **ID:** OG001 **Title:** Arm B (Part 1) ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide vaccine administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after the vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. Resiquimod: 500 mg applied to vaccine site after vaccine administration **ID:** OG002 **Title:** Arm C (Part 1) ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously Resiquimod: 500 mg applied to vaccine site after vaccine administration **ID:** OG003 **Title:** Arm D (Part 1) ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA + PolyICLC vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously IFA: 2 mL administered half intradermally and half subcutaneously Except that one participant on Arm E was on Part 2, where all vaccines were administered at the same vaccine site, and the tetanus peptide was not included in the vaccines. **ID:** OG004 **Title:** Arm E (Part 1 + 2) ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. Resiquimod: 500 mg applied to vaccine site after vaccine administration IFA: 2 mL administered half intradermally and half subcutaneously **ID:** OG005 **Title:** Arm F (Part 1) ##### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC + IFA vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously Resiquimod: 500 mg applied to vaccine site after vaccine administration IFA: 2 mL administered half intradermally and half subcutaneously **ID:** OG006 **Title:** Arm G(Part 1) ### Participant Flow Module #### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA administered in one skin location rotated to different sites on an extremity clinically uninvolved with melanoma. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. IFA: 2 mL administered half intradermally and half subcutaneously **ID:** FG000 **Title:** Arm A (Part 1) #### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC vaccine administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously **ID:** FG001 **Title:** Arm B (Part 1) #### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide vaccine administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after the vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. Resiquimod: 500 mg applied to vaccine site after vaccine administration **ID:** FG002 **Title:** Arm C (Part 1) #### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously Resiquimod: 500 mg applied to vaccine site after vaccine administration **ID:** FG003 **Title:** Arm D (Part 1) #### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA + PolyICLC vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma (except that the vaccines were administered to the same skin site for the one patient from cohort 2) Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. (except that the tetanus peptide was not included in the vaccine for the one patient on this arm from cohort 2. PolyICLC: 1 mL administered half intradermally and half subcutaneously IFA: 2 mL administered half intradermally and half subcutaneously **ID:** FG004 **Title:** Arm E (Part 1 + Part 2) #### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + IFA vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. Resiquimod: 500 mg applied to vaccine site after vaccine administration IFA: 2 mL administered half intradermally and half subcutaneously **ID:** FG005 **Title:** Arm F (Part 1) #### Group **Description:** Peptide Vaccine (LPV7) + Tetanus peptide + PolyICLC + IFA vaccines administered in one skin location that is rotated to different sites on an extremity clinically uninvolved with melanoma. Resiquimod will be applied to the vaccine site immediately after vaccine administration. Vaccines will be administered on Days 1, 8, 15, 36, 57, and 78. Peptide Vaccine (LPV7) + Tetanus peptide: 1.5 mL administered half intradermally and half subcutaneously. PolyICLC: 1 mL administered half intradermally and half subcutaneously Resiquimod: 500 mg applied to vaccine site after vaccine administration IFA: 2 mL administered half intradermally and half subcutaneously **ID:** FG006 **Title:** Arm G(Part 1) #### Period **Title:** Overall Study ##### Withdraw **Type:** disease progression ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ###### Reason **Group ID:** FG002 **Number of Subjects:** 0 ###### Reason **Group ID:** FG003 **Number of Subjects:** 1 ###### Reason **Group ID:** FG004 **Number of Subjects:** 0 ###### Reason **Group ID:** FG005 **Number of Subjects:** 0 ###### Reason **Group ID:** FG006 **Number of Subjects:** 1 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 5 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 7 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 4 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 6 ###### Achievement **Group ID:** FG004 **Number of Subjects:** 16 ###### Achievement **Group ID:** FG005 **Number of Subjects:** 6 ###### Achievement **Group ID:** FG006 **Number of Subjects:** 6 ##### Milestone **Type:** Received at Least 3 Vaccines ###### Achievement **Group ID:** FG000 **Number of Subjects:** 5 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 7 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 4 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 6 ###### Achievement **Group ID:** FG004 **Number of Subjects:** 16 ###### Achievement **Group ID:** FG005 **Number of Subjects:** 6 ###### Achievement **Group ID:** FG006 **Number of Subjects:** 6 ##### Milestone **Type:** COMPLETED **Comment:** Completed all 6 vaccines ###### Achievement **Group ID:** FG000 **Number of Subjects:** 5 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 7 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 4 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 5 ###### Achievement **Group ID:** FG004 **Number of Subjects:** 16 ###### Achievement **Group ID:** FG005 **Number of Subjects:** 6 ###### Achievement **Group ID:** FG006 **Number of Subjects:** 5 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG003 **Number of Subjects:** 1 ###### Achievement **Group ID:** FG004 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG005 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG006 **Number of Subjects:** 1 **Pre-Assignment Details:** After enrolling 49 participants across all 7 arms in accord with the study objectives, the study was amended to allow enrollment of an additional cohort on arm E (part 2) as a neoadjuvant vaccine with biopsies pre- and post-vaccine. However, only 1 patient enrolled on part 2 arm E; thus, we closed the study. Analysis of 1 patient would not satisfy the goals of that neoadjuvant component; so, we include data for the part 2 Arm E participant in Arm E for reporting purposes. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT02160379 **Brief Title:** Effects of Roux-en-Y Gastric Bypass on Gastro-intestinal Hormone Secretion and Brain Responses Food Pictures **Official Title:** Dynamic Effect of Gastrointestinal Hormones on Cerebral Mechanisms Involved in Brain Perception of Food Pictures After Bariatric Surgery #### Organization Study ID Info **ID:** 219/09 #### Organization **Class:** OTHER **Full Name:** University of Lausanne ### Status Module #### Completion Date **Date:** 2012-03 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2014-06-10 **Type:** ESTIMATED **Last Update Submit Date:** 2014-06-06 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2012-03 **Type:** ACTUAL #### Start Date **Date:** 2011-01 **Status Verified Date:** 2014-06 #### Study First Post Date **Date:** 2014-06-10 **Type:** ESTIMATED **Study First Submit Date:** 2014-06-04 **Study First Submit QC Date:** 2014-06-06 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Lausanne #### Responsible Party **Investigator Affiliation:** University of Lausanne **Investigator Full Name:** Luc Tappy, MD **Investigator Title:** Professor of Physiology **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** This is a cross-sectional study enrolling * 11 weight-stable, formerly obese female patients 1-5 years after Roux-en-Y gastric Bypass (RYGB) * 11 age- and weight-matched female subjects * 10 normal weight (Body Mass Index 19-25 kg/m2) subjects The following measurements will be performed in each participants in basal conditions (after an overnight fast) and after ingestion of a standard meal * electroencephalographic recordings of visually evoked potential after presentation of food- or non-food pictures * plasma concentration of metabolites (glucose, fatty acids), glucoregulatory hormones (insulin, glucagon), gastro-intestinal hormones (Gastric Inhibitory Polypeptide (GIP), Glucagon-like Peptide-1 (GLP1), ghrelin,Cholecystokinin (CCK), and bile acids Aims of this observational study are * to evaluate the effects of RYGB on brain perception of food related visual stimulation * to search for relationships between RYGB effects on brain responses and gastro-intestinal hormone secretion ### Conditions Module **Conditions:** - Obesity **Keywords:** - roux-en-Y-gastric bypass - brain responses to food - gastrointestinal hormones - bile acids ### Design Module #### Design Info **Observational Model:** CASE_CONTROL **Time Perspective:** CROSS_SECTIONAL #### Enrollment Info **Count:** 31 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Formerly obese females 1-5 years after gastric bypass **Intervention Names:** - Other: standard test meal **Label:** post Roux-en-Y-gastric bypass #### Arm Group 2 **Description:** non-operated females age-and weight-matched to post Roux-en-Y-gastric bypass subjects **Intervention Names:** - Other: standard test meal **Label:** matched controls #### Arm Group 3 **Description:** non overweight (BMI between 19 and 25 kg/m2) healthy females **Intervention Names:** - Other: standard test meal **Label:** healthy young controls ### Interventions #### Intervention 1 **Arm Group Labels:** - healthy young controls - matched controls - post Roux-en-Y-gastric bypass **Name:** standard test meal **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Recording and analysis of visual evoked potentials (VEPs) while patients categorize photographs of foods and non-food kitchen utensils **Measure:** Changes of visually-evoked EEG potentials after ingestion of a meal **Time Frame:** basal, 120 min postprandial and 240 postprandial #### Secondary Outcomes **Description:** Measurement of triglyceride concentrations at 30-60 min intervals in basal conditions (after an overnight fast) and over 6 hours after ingestion of a standard meal) **Measure:** changes in plasma triglyceride concentration after ingestion of a meal **Time Frame:** basal, 90 min postprandial and 180 postprandial **Description:** Measurement of GLP1 and GIP concentrations at 30-60 min intervals in basal conditions (after an overnight fast) and over 6 hours after ingestion of a standard meal) **Measure:** Changes in plasma glucoincretins (GLP1 and GIP) concentrations after ingestion of a meal **Time Frame:** basal, 90 min postprandial and 180 postprandial **Description:** Measurement of cholecystokinin concentrations at 30-60 min intervals in basal conditions (after an overnight fast) and over 6 hours after ingestion of a standard meal) **Measure:** Changes in plasma cholecystokinin concentrations after ingestion of a meal **Time Frame:** basal, 90 min postprandial and 180 postprandial **Description:** Measurement of bile acids concentrations at 30-60 min intervals in basal conditions (after an overnight fast) and over 6 hours after ingestion of a standard meal) **Measure:** Changes in plasma bile acids concentrations after ingestion of a meal **Time Frame:** basal, 90 min postprandial and 180 postprandial **Description:** Measurement of glucose, insulin, glucagon and non-esterified fatty acid concentrations at 30-60 min intervals in basal conditions (after an overnight fast) and over 6 hours after ingestion of a standard meal) **Measure:** changes in plasma glucoregulatory status after ingestion of a meal **Time Frame:** basal, 90 min postprandial and 180 postprandial ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * good apparent health * gender: female * age 18-50 * non-smoker Exclusion Criteria: * diabetes or antidiabetic medication * consumption of more than 50g alcohol per week * vegetarian or any special diet **Healthy Volunteers:** True **Maximum Age:** 50 Years **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** FEMALE **Standard Ages:** - ADULT **Study Population:** Formerly obese patients attending the obesity clinics of Lausanne University Hospital and healthy subjects resident in the Lausanne area ### Contacts Locations Module #### Locations **Location 1:** **City:** Lausanne **Country:** Switzerland **Facility:** CHUV-clinical research center **State:** VD **Zip:** CH-1001 #### Overall Officials **Official 1:** **Affiliation:** University of Lausanne **Name:** Luc Tappy, MD **Role:** PRINCIPAL_INVESTIGATOR ## 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 ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Gast - Name: Gastrointestinal Agents ### Intervention Browse Module - Browse Leaves - ID: M9789 - Name: Hormones - Relevance: LOW - As Found: Unknown - ID: M4933 - Name: Bile Acids and Salts - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00725179 **Brief Title:** Treatment of Acetaminophen Toxicity With N-acetylcysteine **Official Title:** Treatment of Acetaminophen Toxicity With Intravenous vs Oral N-acetylcysteine: A Retrospective Review #### Organization Study ID Info **ID:** APAP & NAC #### Organization **Class:** OTHER **Full Name:** Akron Children's Hospital ### Status Module #### Completion Date **Date:** 2008-10 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2011-09-09 **Type:** ESTIMATED **Last Update Submit Date:** 2011-09-08 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2008-09 **Type:** ACTUAL #### Start Date **Date:** 2008-08 **Status Verified Date:** 2011-09 #### Study First Post Date **Date:** 2008-07-30 **Type:** ESTIMATED **Study First Submit Date:** 2008-07-25 **Study First Submit QC Date:** 2008-07-29 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Akron Children's Hospital #### Responsible Party **Investigator Affiliation:** Akron Children's Hospital **Investigator Full Name:** Martha Blackford **Investigator Title:** PharmD **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Retrospective chart review of patients who received N-acetylcysteine for APAP toxicity to assess length of oral vs IV treatment and overall healthcare costs. ### Conditions Module **Conditions:** - Acetaminophen Toxicity **Keywords:** - acetaminophen toxicity - n-acetylcysteine - liver toxicity ### Design Module #### Design Info **Observational Model:** CASE_ONLY **Time Perspective:** RETROSPECTIVE #### Enrollment Info **Count:** 130 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients receiving oral NAC treatment **Label:** 1 #### Arm Group 2 **Description:** Patients receiving IV NAC treatment **Label:** 2 ### Outcomes Module #### Primary Outcomes **Measure:** Duration of oral vs IV NAC treatment in hours **Time Frame:** From admission to discharge #### Secondary Outcomes **Measure:** Overall healthcare costs associated with IV vs Oral NAC treatment **Time Frame:** From admission to discharge ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Admitted to CHMCA between June 1, 2004 to May 31, 2008 with a qualifying ICD-9 diagnosis code for the following APAP overdose situations; poisoning, accidental, suicide attempt, therapeutic use, assault, and undetermined (965.4, E850.4, E950.0, E935.4, E962.0, E980.0) * Serum APAP concentrations ≥ 150 g/mL at 4 hours or a serum concentration above the possible hepatotoxic line on the Rumack-Matthew's nomogram if obtained \> 4 hours post ingestion * Between the ages of 0-21 years Exclusion Criteria: * Serum APAP concentrations are not actually documented * Patient did not receive oral or IV NAC treatment * Patient has a preexisting liver disease such as cirrhosis or hepatitis C * Patient \> 21 years of age **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT **Study Population:** Pediatric and adolescent patients with acetaminophen toxicity ### Contacts Locations Module #### Locations **Location 1:** **City:** Akron **Country:** United States **Facility:** Children's Hospital Medical Center of Akron **State:** Ohio **Zip:** 44703 #### Overall Officials **Official 1:** **Affiliation:** CHMCA **Name:** Martha Blackford, PharmD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Heard KJ. Acetylcysteine for acetaminophen poisoning. N Engl J Med. 2008 Jul 17;359(3):285-92. doi: 10.1056/NEJMct0708278. **PMID:** 18635433 **Citation:** Culley CM, Krenzelok EP. A clinical and pharmacoeconomic justification for intravenous acetylcysteine: a US perspective. Toxicol Rev. 2005;24(2):131-43. doi: 10.2165/00139709-200524020-00007. **PMID:** 16180933 **Citation:** Kanter MZ. Comparison of oral and i.v. acetylcysteine in the treatment of acetaminophen poisoning. Am J Health Syst Pharm. 2006 Oct 1;63(19):1821-7. doi: 10.2146/ajhp060050. **PMID:** 16990628 **Citation:** Blackford MG, Felter T, Gothard MD, Reed MD. Assessment of the clinical use of intravenous and oral N-acetylcysteine in the treatment of acute acetaminophen poisoning in children: a retrospective review. Clin Ther. 2011 Sep;33(9):1322-30. doi: 10.1016/j.clinthera.2011.08.005. Epub 2011 Sep 3. **PMID:** 21890206 ## Derived Section ### Intervention Browse Module - Browse Branches - Abbrev: Antipy - Name: Antipyretics - Abbrev: Analg - Name: Analgesics - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Hemat - Name: Hematinics - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: Resp - Name: Respiratory System Agents ### Intervention Browse Module - Browse Leaves - ID: M2340 - Name: Acetaminophen - Relevance: LOW - As Found: Unknown - ID: M11110 - Name: Liver Extracts - Relevance: LOW - As Found: Unknown - ID: M3475 - Name: Acetylcysteine - Relevance: LOW - As Found: Unknown - ID: M244107 - Name: N-monoacetylcystine - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02991079 **Brief Title:** Effectiveness of a Multifactorial Intervention to Improve Diet and Physical Activity in Diabetics From Primary Care **Official Title:** Effectiveness of a Multifactorial Intervention to Improve Diet and Physical Activity in Diabetic Patients From Primary Care: A Randomised Controlled Trial #### Organization Study ID Info **ID:** GRS 1276/B/16 #### Organization **Class:** OTHER **Full Name:** Fundacion para la Investigacion y Formacion en Ciencias de la Salud ### Status Module #### Completion Date **Date:** 2018-03-28 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-04-24 **Type:** ACTUAL **Last Update Submit Date:** 2018-04-23 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2018-03-17 **Type:** ACTUAL #### Start Date **Date:** 2017-02-01 **Type:** ACTUAL **Status Verified Date:** 2018-04 #### Study First Post Date **Date:** 2016-12-13 **Type:** ESTIMATED **Study First Submit Date:** 2016-12-09 **Study First Submit QC Date:** 2016-12-12 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Castilla-León Health Service #### Lead Sponsor **Class:** OTHER **Name:** Fundacion para la Investigacion y Formacion en Ciencias de la Salud #### 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 randomized, clinical trial aimed at diabetics between 25-70 years (with no cardiovascular disease) selected at urban primary care health clinics in Salamanca (Spain). It is aimed at assessing the effects of adding an ICT (information and communication technology) tool, developed for the Smartphone application and group activities of healthy food and cardio-health rides in support of behavioral and educational recommendations in the increased physical activity and adaption to the Mediterranean dietary pattern. **Detailed Description:** Objective: The main objective of this study is to evaluate the effect of adding a multifactorial intervention (ICT tool for Smartphone, cardio-health rides and a standardized advice about eating habits) to the usual care in the increase of the physical activity, in order to reach the international recommendations and in the Increased adherence to the Mediterranean diet in type 2 diabetic patients. Secondary objectives will be to evaluate the effect of intervention in improving dietary patterns, cardiovascular risk factors and metabolic control. Design and setting: A randomised controlled clinical trial, with two parallel groups, aimed at assessing the effects of adding an ICT tool and group activities on healthy food and cardio-health rides (intervention), in support of behavioral and educational recommendations (control) in the increased physical activity and adaption to the Mediterranean dietary pattern. Study setting: Primary Care Health Area of Salamanca in the Research Unit of La Alamedilla, belonging to the Spanish Network for Preventive Activities and Health Promotion (redIAPP) and Salamanca Institute for Biomedical Research (IBSAL). Study population: 200 patients with Diabetes Mellitus Type 2, aged between 25-70 years, of both sexes, who meet selection criteria and signed informed consent. Each participant will make three visits: baseline, three and twelve months post-intervention. Measuring the effect of the intervention: The validated 14-point Mediterranean Diet Adherence Screener, the International Physical Activity Questionnaire (IPAQ), pedometer, laboratory values including glucose and glycosylated hemoglobin, and quality of life questionnaire (SF -12). ### Conditions Module **Conditions:** - Healthy **Keywords:** - Food Habits - Health education - Physical activity - Information and Communication Technologies ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 200 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Counseling on physical activity and Mediterranean diet. **Intervention Names:** - Other: Control: Lifestyle counseling **Label:** Control: Lifestyle counseling **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Add for three months a smartphone with an app (EVIDENT) to improve alimentation and physical activity, five cardio-health rides and feeding workshop. **Intervention Names:** - Other: Intervention group - Other: Control: Lifestyle counseling **Label:** Intervention group **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Intervention group **Description:** Smartphone with APP (EVIDENT) for 3 months, cardio-health rides and feeding workshop. Counseling on physical activity and Mediterranean diet. **Name:** Intervention group **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - Control: Lifestyle counseling - Intervention group **Description:** Conseling on physical activity and nutrition (intervention) **Name:** Control: Lifestyle counseling **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Principal endpoint of alimentation, will be measured using the validated 14-point Mediterranean Diet Adherence Screener. **Measure:** Adherence to the Mediterranean diet using the validated 14-point Mediterranean Diet Adherence Screener **Time Frame:** 1 year **Description:** Measured by the International Physical Activity Questionary - Short Form **Measure:** Self-reported physical activity measured by the International Physical Activity Questionary - Short Form **Time Frame:** 1 year #### Secondary Outcomes **Description:** Measurement by pedometer. **Measure:** Increase physical activity measurement by pedometer **Time Frame:** 1 year **Description:** Measurement by laboratory test sampling **Measure:** HbA1c **Time Frame:** 1 year **Description:** Measurement by laboratory test sampling **Measure:** Fasting Plasma Glucose **Time Frame:** 1 year **Description:** Measurement by SF-12 test **Measure:** Quality of life measured by SF-12 test **Time Frame:** 1 year **Description:** Measurement by Framingham Risk Score **Measure:** Cardiovascular risk factors measured by Framingham Risk Score **Time Frame:** 1 year ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Subjects with Diabetes Mellitus Type 2 since 25 to 70 years. Exclusion Criteria: * Older than 70 years are excluded, due to difficulties in the use of ICTs. * History of cardiovascular events (acute myocardial infarction, stroke, etc). * Diagnosis of clinically demonstrable neurological and/or neuropsychological disease. * Muscular-skeletal pathology that inhibit mobility. * Those with any other circumstance that the investigators consider could interfere with the study procedures. **Maximum Age:** 70 Years **Minimum Age:** 25 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Salamanca **Country:** Spain **Facility:** Primary Care Research Unit - The Alamedilla Center for Health **Zip:** 37003 ### References Module #### References **Citation:** Alonso-Dominguez R, Recio-Rodriguez JI, Patino-Alonso MC, Sanchez-Aguadero N, Garcia-Ortiz L, Gomez-Marcos MA. Acute effect of healthy walking on arterial stiffness in patients with type 2 diabetes and differences by age and sex: a pre-post intervention study. BMC Cardiovasc Disord. 2019 Mar 8;19(1):56. doi: 10.1186/s12872-019-1039-x. **PMID:** 30849947 **Citation:** Alonso-Dominguez R, Patino-Alonso MC, Sanchez-Aguadero N, Garcia-Ortiz L, Recio-Rodriguez JI, Gomez-Marcos MA. Effect of a multifactorial intervention on the increase in physical activity in subjects with type 2 diabetes mellitus: a randomized clinical trial (EMID Study). Eur J Cardiovasc Nurs. 2019 Jun;18(5):399-409. doi: 10.1177/1474515119835048. Epub 2019 Feb 26. **PMID:** 30808196 **Citation:** Alonso-Dominguez R, Gomez-Marcos MA, Patino-Alonso MC, Sanchez-Aguadero N, Agudo-Conde C, Castano-Sanchez C, Garcia-Ortiz L, Recio-Rodriguez JI. Effectiveness of a multifactorial intervention based on an application for smartphones, heart-healthy walks and a nutritional workshop in patients with type 2 diabetes mellitus in primary care (EMID): study protocol for a randomised controlled trial. BMJ Open. 2017 Sep 14;7(9):e016191. doi: 10.1136/bmjopen-2017-016191. **PMID:** 28912193 ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03989479 **Brief Title:** Effectiveness of T-shaped Toothbrush **Official Title:** The Effectiveness of T-shaped Toothbrush in Plaque Removal and Maintaining Gingival Health Among Children #### Organization Study ID Info **ID:** T1 #### Organization **Class:** OTHER **Full Name:** University of Malaya ### Status Module #### Completion Date **Date:** 2016-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2019-06-21 **Type:** ACTUAL **Last Update Submit Date:** 2019-06-19 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2015-09 **Type:** ACTUAL #### Start Date **Date:** 2015-04 **Type:** ACTUAL **Status Verified Date:** 2019-06 #### Study First Post Date **Date:** 2019-06-18 **Type:** ACTUAL **Study First Submit Date:** 2019-06-13 **Study First Submit QC Date:** 2019-06-13 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Malaya #### 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 evaluated the effectiveness of two manual toothbrushes, the T-shaped toothbrush (Denson™, Malaysia) and a conventional toothbrush (Kid's Soft Toothbrush, Colgate) in removing plaque and maintaining gingival health among 8-10-year old children. Brushing demonstration was provided at baseline and reinforced at 2 weeks and 1 month interval.Plaque scores and gingival scores were measured at 2 weeks and 1 month and 3 months **Detailed Description:** T-shaped toothbrush (Denson™, Malaysia) was introduced that is designed to efficiently clean and reduce gingival inflammation. Unlike most common conventional manual toothbrush, this new toothbrush was designed to employ a vertical motion on all tooth surfaces which simulates the natural up and down movement of the hand, suggesting that this makes the brushing process to be more controlled and stable. One hundred and ten participants were assigned randomly to conventional toothbrush (Kid's Soft Toothbrush, Colgate) as a control group, while another half were assigned the T-shaped toothbrush (Denson™, Malaysia) as an experimental group. Gingival status was scored using the Loe and Silness Gingival Index 25. The amount of plaque was scored using the modified Quigley and Hein Plaque Index (TQHI) 26. Both indices were recorded at 6 sites around all the teeth (mesiobuccal, midbuccal, distobuccal, mesiopalatal, midpalatal, distopalatal). All teeth were included except teeth with prosthetic crowns or cervical restorations. One hundred children completed the study. ### Conditions Module **Conditions:** - Oral Hygiene **Keywords:** - toothbrush - plaque removal - gingival health ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - INVESTIGATOR **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 100 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Brushing with conventional Kid's Soft Toothbrush, Colgate (5-9-year-old) **Intervention Names:** - Behavioral: Brushing demonstration using conventional toothbrush **Label:** Conventional Toothbrush **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Brushing with T-shaped toothbrush (Denson™, Malaysia) **Intervention Names:** - Behavioral: Brushing demonstration using T shaped toothbrush **Label:** T-shaped Toothbrush **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - T-shaped Toothbrush **Description:** Brushing demonstration given at baseline and reinforced at 2 weeks and 1 month **Name:** Brushing demonstration using T shaped toothbrush **Type:** BEHAVIORAL #### Intervention 2 **Arm Group Labels:** - Conventional Toothbrush **Description:** Brushing demonstration given at baseline and reinforced at 2 weeks and 1 month **Name:** Brushing demonstration using conventional toothbrush **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** Plaque score measurement performed at baseline, after 2 weeks, 1 month and 3 months. Plaque score measurement (amount of plaque) was recorded on both the buccal and lingual sides of all teeth using the modified Quigley and Hein Plaque Index (Turesky et al., 1970) {0-no plaque present, 1- separate flecks of plaque at the cervical margin, 2- a thin, continous band of plaque at the cervical margin, 3- a band of plaque wider than 1mm but covering less than 1/3 of the surface, 4- plaque covering at least 1/3 but less than 2/3 of the surface, 5- plaque covering more than 2/3 of the surface}. Each tooth was scored in six areas: mesiofacial, midfacial, distofacial, mesiolingual, midlingual and distolingual. The minimum and maximum score per tooth was 0 and 30. The scores for each participant was summed and divided by the total number of measurements (number of teeth scored multiplied by six). Higher scores indicated worse outcome. **Measure:** Plaque score **Time Frame:** 3 months **Description:** Gingival status measurement performed at baseline, after 2 weeks, 1 month and 3 months. Gingival status (gingival inflammation) was scored using the Loe and Silness (Loe \& Silness, 1963) {0-absence of inflammation, 1- mild inflammation, 2- moderate inflammation, 3- severe inflammation}. Each tooth was scored in six areas: mesiofacial, midfacial, distofacial, mesiolingual, midlingual and distolingual. The minimum and maximum score per tooth was 0 and 18. The scores for each participant was calculated by adding all the individual plaque scores and dividing this sum by the total number of measurements (number of teeth scored multiplied by six). Higher scores indicated worse outcome **Measure:** Gingival status **Time Frame:** 3 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * good systemic health * normal motor and cognitive development Exclusion Criteria: * acute intraoral lesion * history of antibiotic and/ or antiseptic therapy in the past one month * interproximal caries or restorations * 3 or more missing teeth in one quadrant **Healthy Volunteers:** True **Maximum Age:** 10 Years **Minimum Age:** 8 Years **Sex:** ALL **Standard Ages:** - CHILD ### Contacts Locations Module #### Overall Officials **Official 1:** **Affiliation:** Faculty of Dentistry, University of Malaya **Name:** Shani Ann Mani, MDS **Role:** STUDY_CHAIR ### References Module #### References **Citation:** Mamat N, Mani SA, Danaee M. T-shaped toothbrush for plaque removal and gingival health in children: a randomized controlled trial. BMC Oral Health. 2022 Apr 7;22(1):113. doi: 10.1186/s12903-022-02137-x. **PMID:** 35392881 ## Document Section ### Large Document Module #### Large Docs - Date: 2014-11-26 - Filename: ICF_000.pdf - Has ICF: True - Has Protocol: False - Has SAP: False - Label: Informed Consent Form - Size: 263270 - Type Abbrev: ICF - Upload Date: 2019-06-11T00:52 ## Derived Section ### Intervention Browse Module - Browse Branches - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M140080 - Name: Listerine - Relevance: LOW - As Found: Unknown - ID: M15771 - Name: Sodium Fluoride - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02168179 **Brief Title:** KeraStat Skin Therapy in Treating Radiation Dermatitis in Patients With Newly Diagnosed Stage 0-IIIA Breast Cancer **Official Title:** Safety of Treatment With KeraStat Skin Therapy in Breast Cancer Patients Developing Radiation Dermatitis #### Organization Study ID Info **ID:** IRB00028009 #### Organization **Class:** OTHER **Full Name:** Wake Forest University Health Sciences #### Secondary ID Infos **Domain:** CTRP (Clinical Trial Reporting Program) **ID:** NCI-2014-01274 **Type:** REGISTRY **ID:** CCCWFU 98114 **ID:** P30CA012197 **Link:** https://reporter.nih.gov/quickSearch/P30CA012197 **Type:** NIH ### Status Module #### Completion Date **Date:** 2016-07 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-07-05 **Type:** ACTUAL **Last Update Submit Date:** 2018-07-02 **Overall Status:** WITHDRAWN #### Primary Completion Date **Date:** 2016-07 **Type:** ACTUAL #### Start Date **Date:** 2014-12 **Type:** ACTUAL **Status Verified Date:** 2018-07 #### Study First Post Date **Date:** 2014-06-20 **Type:** ESTIMATED **Study First Submit Date:** 2014-06-18 **Study First Submit QC Date:** 2014-06-18 **Why Stopped:** No longer pursuing study at our site. ### 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:** This pilot clinical trial studies KeraStat Skin Therapy in treating radiation dermatitis in patients with newly diagnosed stage 0-IIIA breast cancer. Radiation dermatitis is an itchy, painful skin rash that can occur following treatment with radiation. KeraStat Skin Therapy may be a better treatment for radiation dermatitis. **Detailed Description:** PRIMARY OBJECTIVES: I. To obtain a preliminary estimate the incidence of early adverse skin reaction (EASR) during radiation therapy (RT) and up to two months post RT after the application of the cosmetic cream KeraStat Skin Therapy during RT in breast cancer patients in a pilot study. SECONDARY OBJECTIVES: I. To associate personal characteristics (e.g., race/ethnicity, age, hormone therapy, smoking status, comorbidities, breast size) and treatment characteristics (e.g., RT dose) to incidence of EASR at any time point. OUTLINE: Patients apply KeraStat Skin Therapy topically twice daily (BID) during radiation therapy. After completion of study treatment, patients are followed up at 1 and 2 months. ### Conditions Module **Conditions:** - Ductal Breast Carcinoma in Situ - Skin Reactions Secondary to Radiation Therapy - Stage IA Breast Cancer - Stage IB Breast Cancer - Stage IIA Breast Cancer - Stage IIB Breast Cancer - Stage IIIA Breast Cancer ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients apply KeraStat Skin Therapy topically BID during radiation therapy. **Intervention Names:** - Procedure: dermatologic complications management/prevention - Other: questionnaire administration **Label:** Supportive Care (KeraStat Skin Therapy) **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Supportive Care (KeraStat Skin Therapy) **Description:** Apply KeraStat Skin Therapy topically **Name:** dermatologic complications management/prevention **Other Names:** - complications management/prevention, dermatologic - management/prevention, dermatologic complications **Type:** PROCEDURE #### Intervention 2 **Arm Group Labels:** - Supportive Care (KeraStat Skin Therapy) **Description:** Ancillary studies **Name:** questionnaire administration **Type:** OTHER ### Outcomes Module #### Other Outcomes **Description:** A series of 2xr tables will be examined to determine the relationship between each of the categorical confounders and the primary endpoint. Chi-squared statistics will be estimated for each of these tables to give some preliminary descriptive data to identify potential variables that may be associated with the primary outcome. **Measure:** Categorical confounders, such as race/ethnicity, age, hormone therapy, smoking history/status, diabetes, high blood pressure, breast size, RT characteristics, and RT dosimetry characteristics, using the Baseline Study Risk Questionnaire **Time Frame:** Baseline #### Primary Outcomes **Description:** At each time point the proportion of women who have RT-induced EASR present will be estimated and a 95% confidence interval will be calculated around this estimate. **Measure:** Occurrence of any RT-induced EASR defined as a grade 4 or higher toxicity using the Modified Oncology Nursing Society Criteria for Radiation-Induced Acute Skin Toxicity **Time Frame:** Up to 2 months after completion of radiation therapy ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Newly diagnosed with breast carcinoma, stage 0-IIIA (including ductal carcinoma in situ \[DCIS\]) * Status post-lumpectomy, -quadrantectomy, or -mastectomy * Plan to receive adjuvant radiation to the whole breast or chest wall +/- regional lymph nodes * Total dose \>= 40Gy * Dose per fraction \>= 1.8 use of 2-dimensional (2D), 3-dimensional (3D) conformal, or intensity-modulated radiation therapy (IMRT) treatment techniques allowed; skin sparing IMRT patients excluded; a daily fraction of 2.7 Gy to the whole breast is suggested for hypofractionated regimens * Concurrent and sequential boost techniques are allowed for both standard and hypofractionated regimens * Adjuvant hormonal therapy will be allowed prior to, during and/or after RT at the discretion of a medical oncologist * Targeted therapies such as Herceptin will be allowed prior to, during, and/or after RT at the discretion of the medical oncologist * Patients who are able and willing to sign protocol consent form Exclusion Criteria: * Prior radiation to the involved breast or chest wall * Concurrent chemotherapy; (patients may receive chemotherapy prior to radiation or following radiation at the treating physician's discretion) * Patients who underwent breast reconstruction following mastectomy (placement of tissue expanders and implants are not allowed) * Patients undergoing partial breast irradiation * Patients who have undergone MammoSite® or any other form of brachytherapy * Patients may not be concurrently enrolled in a protocol that involves treatment of the skin ie: applying lotions /moisturizers; protocols that do not involve treatment of the skin are allowed * Patients who are pregnant or breastfeeding **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Winston-Salem **Country:** United States **Facility:** Comprehensive Cancer Center of Wake Forest University **State:** North Carolina **Zip:** 27157 #### Overall Officials **Official 1:** **Affiliation:** Wake Forest University Health Sciences **Name:** Arthur Blackstock **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 - ID: D000002277 - Term: Carcinoma - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000044584 - Term: Carcinoma, Ductal - ID: D000000230 - Term: Adenocarcinoma - ID: D000018299 - Term: Neoplasms, Ductal, Lobular, and Medullary - 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: BC23 - Name: Symptoms and General Pathology - Abbrev: BC26 - Name: Wounds and Injuries ### Condition Browse Module - Browse Leaves - ID: M5220 - Name: Breast Neoplasms - Relevance: HIGH - As Found: Breast Cancer - ID: M5534 - Name: Carcinoma - Relevance: LOW - As Found: Unknown - ID: M7067 - Name: Dermatitis - Relevance: HIGH - As Found: Dermatitis - ID: M12307 - Name: Neoplasm Metastasis - Relevance: LOW - As Found: Unknown - ID: M5535 - Name: Carcinoma in Situ - Relevance: HIGH - As Found: Carcinoma in Situ - ID: M895 - Name: Breast Carcinoma In Situ - Relevance: HIGH - As Found: Breast carcinoma in situ - ID: M5542 - Name: Carcinoma, Intraductal, Noninfiltrating - Relevance: HIGH - As Found: Ductal breast carcinoma in situ - ID: M20415 - Name: Carcinoma, Ductal, Breast - Relevance: HIGH - As Found: Ductal breast carcinoma - ID: M14701 - Name: Radiodermatitis - Relevance: HIGH - As Found: Radiation Dermatitis - ID: M25356 - Name: Carcinoma, Ductal - 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 - 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: 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: D000001943 - Term: Breast Neoplasms - ID: D000002278 - Term: Carcinoma in Situ - ID: D000018270 - Term: Carcinoma, Ductal, Breast - ID: D000071960 - Term: Breast Carcinoma In Situ - ID: D000002285 - Term: Carcinoma, Intraductal, Noninfiltrating - ID: D000003872 - Term: Dermatitis - ID: D000011855 - Term: Radiodermatitis ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05528679 **Brief Title:** A Phase II Study of 610 in Participants With Severe Eosinophilic Asthma **Official Title:** A Multicenter, Randomized, Double-blind, Placebo-controlled Phase II, Efficacy and Safety Study of Recombinant Anti-IL-5 Humanized Monoclonal Antibody Therapy in Adult Subjects With Severe Eosinophilic Asthma #### Organization Study ID Info **ID:** SSGJ-610-BA-II-01 #### Organization **Class:** INDUSTRY **Full Name:** Sunshine Guojian Pharmaceutical (Shanghai) Co., Ltd. ### Status Module #### Completion Date **Date:** 2023-09 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2022-09-06 **Type:** ACTUAL **Last Update Submit Date:** 2022-09-01 **Overall Status:** NOT_YET_RECRUITING #### Primary Completion Date **Date:** 2023-09 **Type:** ESTIMATED #### Start Date **Date:** 2022-09 **Type:** ESTIMATED **Status Verified Date:** 2022-09 #### Study First Post Date **Date:** 2022-09-06 **Type:** ACTUAL **Study First Submit Date:** 2022-09-01 **Study First Submit QC Date:** 2022-09-01 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Sunshine Guojian Pharmaceutical (Shanghai) Co., Ltd. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** This study will assess the efficacy and safety of 610 as an adjunctive therapy in adult subjects with severe eosinophilic asthma. **Detailed Description:** This is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of 610 in adults with severe eosinophilic asthma. Plan to recruit 120 subjects, and the subjects divided into 3 groups: 610 100mg group, 610 300mg group and placebo group. The study is divided into screening period of 4 weeks, treatment period of 16 weeks and follow-up period of 8 weeks. ### Conditions Module **Conditions:** - Asthma **Keywords:** - Asthma - Eosinophilic asthma - Exacerbations - 610 - Placebo ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** TRIPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 120 **Type:** ESTIMATED **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 610 100 mg administered subcutaneously every 4 weeks **Intervention Names:** - Drug: 610 100mg **Label:** Treatment group A **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** 610 300 mg administered subcutaneously every 4 weeks **Intervention Names:** - Drug: 610 300mg **Label:** Treatment group B **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** placebo administered subcutaneously every 4 weeks **Intervention Names:** - Drug: placebo **Label:** placebo Arm Type：no inter **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Treatment group A **Description:** 100mg administered subcutaneously Q4W **Name:** 610 100mg **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Treatment group B **Description:** 300mg administered subcutaneously Q4W **Name:** 610 300mg **Type:** DRUG #### Intervention 3 **Arm Group Labels:** - placebo Arm Type：no inter **Description:** administered subcutaneously Q4W **Name:** placebo **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** FEV1 is defined as the volume of air expelled from the lungs in 1 second. Pre-bronchodilator FEV1 measurements were taken by spirometry. **Measure:** Change from baseline in pre-bronchodilator forced expiratory volume in one second (FEV1) at week 16 **Time Frame:** Baseline (Day 1) and at week 16 #### Secondary Outcomes **Description:** FEV1 is defined as the volume of air expelled from the lungs in 1 second. Pre-bronchodilator FEV1 measurements were taken by spirometry. **Measure:** Changes from baseline in pre-bronchodilator forced expiratory volume in one second (FEV1) at weeks 4, 8, and 12 **Time Frame:** Baseline (Day 1) and at week 4,8,12 **Description:** Percentage of FEV1 will be measured using spirometry. **Measure:** Percentage change from baseline in pre-bronchodilator forced expiratory volume in one second (FEV1) at weeks 4, 8, 12, 16 **Time Frame:** Baseline (Day 1) and at week 4,8,12 **Description:** Asthma exacerbation are defined as worsening of asthma which required use of systemic corticosteroids (≥3 days. For maintenance of systemic corticosteroids, at least double the existing maintenance dose for at least 3 days was required) and/or hospitalization and/or emergency department (ED) visits. **Measure:** Number of asthma exacerbation through study week 16 **Time Frame:** From baseline (Day 1) to week 16 **Description:** Asthma exacerbation are defined as worsening of asthma which required use of systemic corticosteroids (≥3 days. For maintenance of systemic corticosteroids, at least double the existing maintenance dose for at least 3 days was required) and/or hospitalization and/or emergency department (ED) visits. **Measure:** Time to first asthma exacerbation event **Time Frame:** From baseline (Day 1) to week 16 **Description:** Asthma exacerbations that are associated with a hospitalization or an emergency room visit. **Measure:** Number of asthma exacerbations requiring hospitalization (including intubation and ICU admission) or emergency room visits (not conversion to hospitalization) **Time Frame:** From baseline (Day 1) to week 16 **Description:** Asthma exacerbations that are associated with a hospitalization. **Measure:** Number of asthma exacerbations requiring hospitalization (including intubation and ICU admission) **Time Frame:** From baseline (Day 1) to week 16 **Description:** Albuterol or levalbuterol for an asthma exacerbation is considered rescue medication. **Measure:** Puffs of rescue medication for asthma exacerbations **Time Frame:** From baseline (Day 1) to week 16 **Description:** The ACQ has 7 questions- the first 5 items assess the most common asthma symptoms plus 6. short-acting bronchodilator use and 7. FEV1 (pre-bronchodilator use, % and % predicted use). Patients are asked to recall how their asthma has been during the previous week and to respond to the symptom questions on a 7-point scale (0=no impairment, 6= maximum impairment). **Measure:** Change from baseline in Asthma Control Questionnaire score at week 4,8,12,16 **Time Frame:** Baseline (Day 1) and at week 4,8,12,16 **Description:** The St. George's Respiratory Questionnaire is an established instrument, comprising 50 questions, evaluating symptoms, activity, and impacts; to measure Quality of Life in participants with diseases of airway obstruction and to elicit the participant's opinion of his/her health. **Measure:** Change From Baseline in the St. George's Respiratory Questionnaire Total Score at week 16 **Time Frame:** Baseline (Day 1) and at week 16 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Able to understand and follow the protocol requirements, and give written informed consent prior to participation voluntarily in the study * Female and male aged 18 to 75 years * Diagnosed with asthma for ≥12 months that meet GINA * Within 6 months before screening, treatment with medium to high dose inhaled corticosteroid（ICS，inhaled fluticasone at a dosage of at least 500 μg, or equivalent, daily. ICS can be included in In the ICS/LABA combination preparation）and at least one other additional controller medication, such as long-acting β₂ receptor agonist (LABA), leukotriene receptor antagonist (LTRA), theophylline, long-acting Anticholinergic drugs (LAMA), etc. Those medicine must be stable for ≥ 28 days prior to screening and baseline and must continue without dosage changes throughout the study * Current treatment with maintenance oral corticosteroids (OCS), prednisone dosage must be ≤10 mg, or equivalent, daily, and stable for ≥ 28 days prior to screening and baseline and must continue without dosage changes throughout the study * In the past 12 months prior to screening， two or more asthma exacerbations history, or at least one time emergency department (ED) visits and/or ICU and/or hospitalization * Pre-bronchodilator FEV1 \<80% predicted value * Positive of bronchodilator test or positive of bronchial provocation test * Asthma Control Questionnaire score ≥1.5 * Asthma-related blood eosinophils ≥ 300 cells/μL within 6 months before screening, or asthma-related blood eosinophils ≥ 150 cells/μL at screening * Male and their partners or female must commit to correct use of one or more effective contraceptive measures of the duration of the trial and for 6 months after the last study drug administration. No fertility, sperm donation, or egg donation plans for at least 6 months after the last study drug administration Exclusion Criteria: * With clinically important lung diseases other than asthma that may affect safety or efficacy and evaluated by investigator. This includes lung infection, chronic obstructive pulmonary disease, bronchiectasis, hypersensitivity pneumonitis, pulmonary fibrosis, Allergic bronchopulmonary aspergillosis, etc. * With other conditions that could lead to elevated eosinophils such as hypereosinophilic syndromes, eosinophilic granulomatosis with polyangiitis (EGPA), or eosinophilic esophagitis * In past 12 months prior to screening，patients has done bronchial thermoplasty or radiotherapy or plan to do it during of the trial * with severe cardiac disease or uncontrolled or severe cardiac arrhythmia * poorly controlled systemic disease * Active infection that requiring systemic treatment at screening * Parasitic infection without adequate treatment within 6 months before screening * Lymphoproliferative disease or any malignancy history within past 5 years prior to screening (Except for received treatment and no recurrence in the past 3 months include basal cell carcinoma, actinic keratosis, carcinoma in situ of cervix, or resected non-invasive malignant colonic polyps.) * Liver function meets one of the following criteria at screening or before randomization: a) Serum aspartate aminotransferase (AST) or serum alanine aminotransferase (ALT) ≥ 2.0×ULN (upper limit of normal); b) Total bilirubin≥1.5×ULN * At screening, HBsAg or HCV Ab or HIV Ab or TP Ab positive; HBsAg or HCV Ab positive need to be further tested of HBV DNA titer detection or HCV RNA detection (More than normal value range needs to be excluded) * Subjects who have received any monoclonal antibody treatment within 3 months or 5 half-lives (whichever is longer) before screening, or with poor treatment effect of anti-IL-5/5R * Vaccination history with live vaccines (including live attenuated vaccines) within 4 weeks before screening, or plan to receive during of the trial * Participated in any interventional clinical trial and received intervention within 3 months before screening * Allergy/intolerance to investigational medicinal product. * Current smokers with average monthly smoking of ≥10 cigarettes within 6 months before screening, or former smokers with a smoking history of ≥10 pack years (number of pack years = (number of cigarettes per day / 20) x number of years smoked) * Plan to pregnant during of the trial or pregnant or breastfeeding * Any other things that are not suitable for participating in this study **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:** Qinghong Zhou, MD **Phone:** 18911301578 **Role:** CONTACT #### Locations **Location 1:** **City:** Shanghai **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Min Zhang, Doctor - **Phone:** 13482345145 - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Xin Zhou, Bachelor - **Phone:** 13701756821 - **Role:** CONTACT ***Contact 3:*** - **Name:** Min Zhang - **Role:** PRINCIPAL_INVESTIGATOR ***Contact 4:*** - **Name:** Xin Zhou - **Role:** PRINCIPAL_INVESTIGATOR **Country:** China **Facility:** Shanghai General Hospital **State:** Shanghai **Zip:** 200080 #### Overall Officials **Official 1:** **Affiliation:** Sunshine Guojian Pharmaceutical (Shanghai) Co., Ltd. **Name:** Qinghong Zhou, MD **Role:** STUDY_DIRECTOR **Official 2:** **Affiliation:** Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine **Name:** Min Zhang, MD **Role:** PRINCIPAL_INVESTIGATOR **Official 3:** **Affiliation:** Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine **Name:** Xin Zhou, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ## Derived Section ### Condition Browse Module - Ancestors - ID: D000001982 - Term: Bronchial Diseases - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000008173 - Term: Lung Diseases, Obstructive - ID: D000008171 - Term: Lung Diseases - ID: D000012130 - Term: Respiratory Hypersensitivity - ID: D000006969 - Term: Hypersensitivity, Immediate - ID: D000006967 - Term: Hypersensitivity - ID: D000007154 - Term: Immune System Diseases - ID: D000017681 - Term: Hypereosinophilic Syndrome - ID: D000004802 - Term: Eosinophilia - ID: D000007960 - Term: Leukocyte Disorders - ID: D000006402 - Term: Hematologic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: BC20 - Name: Immune System Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC15 - Name: Blood and Lymph Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M4556 - Name: Asthma - Relevance: HIGH - As Found: Asthma - ID: M14511 - Name: Pulmonary Eosinophilia - Relevance: HIGH - As Found: Eosinophilic Asthma - ID: M7961 - Name: Eosinophilia - Relevance: LOW - As Found: Unknown - ID: M5258 - Name: Bronchial Diseases - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown - ID: M11168 - Name: Lung Diseases - Relevance: LOW - As Found: Unknown - ID: M11170 - Name: Lung Diseases, Obstructive - Relevance: LOW - As Found: Unknown - ID: M10018 - Name: Hypersensitivity - Relevance: LOW - As Found: Unknown - ID: M14967 - Name: Respiratory Hypersensitivity - Relevance: LOW - As Found: Unknown - ID: M10020 - Name: Hypersensitivity, Immediate - Relevance: LOW - As Found: Unknown - ID: M10200 - Name: Immune System Diseases - Relevance: LOW - As Found: Unknown - ID: M16355 - Name: Syndrome - Relevance: LOW - As Found: Unknown - ID: M19901 - Name: Hypereosinophilic Syndrome - Relevance: LOW - As Found: Unknown - ID: M10963 - Name: Leukocyte Disorders - Relevance: LOW - As Found: Unknown - ID: M9490 - Name: Hematologic Diseases - Relevance: LOW - As Found: Unknown - ID: T2886 - Name: Hypereosinophilic Syndrome - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001249 - Term: Asthma - ID: D000011657 - Term: Pulmonary Eosinophilia ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M4225 - Name: Antibodies - Relevance: LOW - As Found: Unknown - ID: M4230 - Name: Antibodies, Monoclonal - Relevance: LOW - As Found: Unknown - ID: M10184 - Name: Immunoglobulins - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02476279 **Acronym:** SVI **Brief Title:** Stent vs. Indomethacin for Preventing Post-ERCP Pancreatitis **Official Title:** Stent vs. Indomethacin for Preventing Post-ERCP Pancreatitis: The SVI Trial #### Organization Study ID Info **ID:** U01DK104833-01 **Link:** https://reporter.nih.gov/quickSearch/U01DK104833-01 **Type:** NIH #### Organization **Class:** OTHER **Full Name:** Medical University of South Carolina #### Secondary ID Infos **ID:** U01DK104833-01 **Link:** https://reporter.nih.gov/quickSearch/U01DK104833-01 **Type:** NIH ### Status Module #### Completion Date **Date:** 2023-01-25 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2024-05-08 **Type:** ACTUAL **Last Update Submit Date:** 2024-04-15 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2023-01-25 **Type:** ACTUAL #### Results First Post Date **Date:** 2024-05-08 **Type:** ACTUAL **Results First Submit Date:** 2024-04-15 **Results First Submit QC Date:** 2024-04-15 #### Start Date **Date:** 2015-09 **Type:** ACTUAL **Status Verified Date:** 2024-04 #### Study First Post Date **Date:** 2015-06-19 **Type:** ESTIMATED **Study First Submit Date:** 2015-06-15 **Study First Submit QC Date:** 2015-06-16 ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) #### Lead Sponsor **Class:** OTHER **Name:** Medical University of South Carolina #### Responsible Party **Investigator Affiliation:** Medical University of South Carolina **Investigator Full Name:** Badih Joseph Elmunzer **Investigator Title:** Professor of Medicine **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** Background: Pancreatitis is the most frequent complication of endoscopic retrograde cholangiopancreatography (ERCP), accounting for substantial morbidity, occasional mortality, and increased health care expenditures. Until recently, the only effective method of preventing post-ERCP pancreatitis (PEP) had been prophylactic pancreatic stent placement (PSP), an intervention that is costly, time consuming, technically challenging, and potentially dangerous. The investigators recently reported the results of a large randomized controlled trial demonstrating that rectal indomethacin, a non-steroidal anti-inflammatory drug, reduced the risk of pancreatitis after ERCP in high-risk patients, most of whom (\>80%) had received a pancreatic stent. Secondary analysis of this RCT suggested that subjects who received indomethacin alone were less likely to develop PEP than those who received a pancreatic stent alone or the combination of indomethacin and stent, even after adjusting for underlying differences in subject risk. If indomethacin were to obviate the need for PSP, major clinical and cost benefits in ERCP practice could be realized. Objective: To assess whether rectal indomethacin alone is non-inferior to the combination of rectal indomethacin and prophylactic pancreatic stent placement for preventing post-ERCP pancreatitis in high-risk cases. Methods: Comparative effectiveness multi-center non-inferiority trial of rectal indomethacin alone vs. the combination of rectal indomethacin and prophylactic pancreatic stent placement for the prevention of post-ERCP pancreatitis in high-risk patients. One thousand four hundred and thirty subjects at elevated risk for PEP who would normally receive a pancreatic stent for prophylaxis will be randomized to indomethacin alone or the combination of indomethacin and PSP. The proportion of patients developing PEP and moderate-severe PEP will be compared. In addition, the investigators will establish a quality-assured central repository of biological specimens obtained from study participants, permitting future translational research elucidating the molecular and genetic mechanisms of PEP, as well as the mechanisms by which non-steroidal anti-inflammatory drugs prevent this complication. **Detailed Description:** The purpose of the SVI study is to determine whether or not rectal indomethacin has no important loss of efficacy as compared to the combination of rectal indomethacin and prophylactic pancreatic stent placement in patients undergoing high-risk ERCP who require pancreatic stent placement (PSP) for the sole purpose of pancreatitis prevention. The primary efficacy endpoint is defined as post-ERCP pancreatitis defined per consensus (Altanta) criteria. Another way of stating the trial's purpose is that the proportion of subjects with post-ERCP pancreatitis on rectal indomethacin alone is not more than that of the combination of rectal indomethacin and prophylactic PSP by more than a pre-specified absolute amount (i.e., the non-inferiority margin). This is a blinded, two-armed non-inferiority trial where eligible patients will be randomized to either the combination treatment or indomethacin alone. Participants will be randomized during the ERCP procedure after eligibility is confirmed, and receive indomethacin at the time of randomization. The primary efficacy endpoint of post-ERCP pancreatitis within 2 days from randomization will be assessed by an independent adjudication panel. The participant follow-up period is 30 days from randomization. ### Conditions Module **Conditions:** - Post-ERCP Pancreatitis ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** QUADRUPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 1950 **Type:** ACTUAL **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Indomethacin 100 mg rectally immediately after ERCP **Intervention Names:** - Other: Indomethacin 100 mg rectally immediately after ERCP, NO prophylactic pancreatic stent placement **Label:** Indomethacin alone **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Indomethacin 100 mg rectally immediately after ERCP AND prophylactic pancreatic stent placement **Intervention Names:** - Other: Indomethacin 100 mg rectally immediately after ERCP AND prophylactic pancreatic stent placement **Label:** Indomethacin+pancreatic stent **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Indomethacin alone **Name:** Indomethacin 100 mg rectally immediately after ERCP, NO prophylactic pancreatic stent placement **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - Indomethacin+pancreatic stent **Name:** Indomethacin 100 mg rectally immediately after ERCP AND prophylactic pancreatic stent placement **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Post-ERCP pancreatitis (PEP) was based on a widely validated consensus definition that was applied as a diagnostic framework. In this consensus definition, PEP is diagnosed if there was new onset (or increase) of pain in the upper abdomen, elevation in pancreatic enzymes of at least three times the upper limit of normal 24 h after the procedure, and hospitalization for at least two nights. The outcome was independently adjudicated by 3 ERCP experts at non-enrolling centers based on review of the medical records for study participants who were hospitalized with any adverse event within 2 days of the ERCP. Medical records were redacted of all information that could potentially reveal study group assignment, including radiology reports. The consensus definition was applied as a diagnostic framework so that adjudicators could use their best judgment in cases that did not strictly satisfy the criteria. PEP was declared if there was agreement between at least two of the three adjudicators. **Measure:** The Proportion of Subjects in Each Study Group With Post-ERCP Pancreatitis **Time Frame:** Within 48 hours after ERCP #### Secondary Outcomes **Description:** Moderate or severe post-ERCP pancreatitis was based on the consensus definition as a diagnostic framework. For the severity assessment, radiographic information was made available to the adjudicators. The severity was defined as mild post-ERCP pancreatitis resulting in a hospitalization of ≤3 days, moderate post-ERCP pancreatitis resulting in a hospitalization of 4-10 days, and severe post-ERCP pancreatitis resulting in a hospitalization of \> 10 days, or leading to the development of pancreatic necrosis or pseudocyst, or requiring percutaneous or surgical intervention. The outcome was declared if there was agreement between at least two of the three adjudicators. **Measure:** The Proportion of Subjects in Each Study Group With Moderate-severe Post-ERCP Pancreatitis **Time Frame:** Within one month of ERCP ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: Any patient undergoing ERCP in whom pancreatic stent placement is planned for post-ERCP pancreatitis prevention, is ≥ 18 years old, who provides informed consent, AND: Has one of the following: 1. Clinical suspicion of or known sphincter of Oddi dysfunction 2. History of post-ERCP pancreatitis (at least one prior episode of pancreatitis after ERCP) 3. Pancreatic sphincterotomy 4. Pre-cut (access) sphincterotomy (freehand pre-cut and septotomy) 5. Difficult cannulation: cannulation duration ≥ 6 minutes (starting at time of initial papillary engagement with at least 25% of the time in contact with the papilla) AND/OR ≥ 6 cannulation attempts (defined as sustained contact with papilla lasting at least 1 second). 6. Short-duration (≤ 1 min) balloon dilation of an intact biliary sphincter. Or has at least 2 of the following: 7. Age \< 50 years old \& female gender 8. History of recurrent pancreatitis (at least 2 episodes) 9. ≥3 pancreatic injections 10. Pancreatic acinarization 11. Pancreatic brush cytology Exclusion Criteria: 1. Ampullectomy 2. Cases in which a pancreatic stent must be placed for therapeutic intent 3. Unwillingness or inability to consent for the study 4. Pregnancy 5. Breast feeding mother 6. Standard contraindications to ERCP 7. Allergy to Aspirin or NSAIDs 8. Known renal failure (Cr \> 1.4 mg/dl) 9. Ongoing or recent (within 2 weeks) hospitalization for gastrointestinal hemorrhage 10. Ongoing or recent (within 1 week) hospitalization for acute pancreatitis 11. Known chronic calcific pancreatitis 12. Pancreatic head malignancy 13. Procedure performed on major papilla/ventral pancreatic duct in patient with pancreas divisum (no manipulation of minor papilla) 14. ERCP for biliary stent removal or exchange without anticipated pancreatogram 15. Subjects with prior biliary sphincterotomy now scheduled for repeat biliary therapy without anticipated pancreatogram 16. Anticipated inability to follow protocol 17. Absence of rectum **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Los Angeles **Country:** United States **Facility:** Univesrity of Southern California **State:** California **Location 2:** **City:** Denver **Country:** United States **Facility:** University of Colorado **State:** Colorado **Location 3:** **City:** Orlando **Country:** United States **Facility:** The Florida Hospital **State:** Florida **Location 4:** **City:** Atlanta **Country:** United States **Facility:** Emory University **State:** Georgia **Location 5:** **City:** Chicago **Country:** United States **Facility:** Northwestern University **State:** Illinois **Location 6:** **City:** Kansas City **Country:** United States **Facility:** University of Kansas **State:** Kansas **Location 7:** **City:** Baltimore **Country:** United States **Facility:** Johns Hopkins University **State:** Maryland **Location 8:** **City:** Ann Arbor **Country:** United States **Facility:** University of Michigan **State:** Michigan **Location 9:** **City:** Saint Louis **Country:** United States **Facility:** Washington University **State:** Missouri **Location 10:** **City:** Lebanon **Country:** United States **Facility:** Dartmouth University **State:** New Hampshire **Location 11:** **City:** Cleveland **Country:** United States **Facility:** Case Western Reserve University **State:** Ohio **Location 12:** **City:** Columbus **Country:** United States **Facility:** The Ohio State University Wexner Medical Center **State:** Ohio **Location 13:** **City:** Portland **Country:** United States **Facility:** Oregon Health & Science University **State:** Oregon **Location 14:** **City:** Pittsburgh **Country:** United States **Facility:** University of Pittsburgh **State:** Pennsylvania **Location 15:** **City:** Charleston **Country:** United States **Facility:** Medical University of South Carolina **State:** South Carolina **Location 16:** **City:** Nashville **Country:** United States **Facility:** Vanderbilt University **State:** Tennessee **Location 17:** **City:** Seattle **Country:** United States **Facility:** Virginia Mason Medical Center **State:** Washington **Location 18:** **City:** Milwaukee **Country:** United States **Facility:** Medical College of Wisconsin **State:** Wisconsin **Location 19:** **City:** Calgary **Country:** Canada **Facility:** University of Calgary **Location 20:** **City:** Montreal **Country:** Canada **Facility:** McGill University ### References Module #### References **Citation:** Elmunzer BJ, Serrano J, Chak A, Edmundowicz SA, Papachristou GI, Scheiman JM, Singh VK, Varadarajulu S, Vargo JJ, Willingham FF, Baron TH, Cote GA, Romagnuolo J, Wood-Williams A, Depue EK, Spitzer RL, Spino C, Foster LD, Durkalski V; SVI study group and the United States Cooperative for Outcomes Research in Endoscopy (USCORE). Rectal indomethacin alone versus indomethacin and prophylactic pancreatic stent placement for preventing pancreatitis after ERCP: study protocol for a randomized controlled trial. Trials. 2016 Mar 3;17(1):120. doi: 10.1186/s13063-016-1251-2. Erratum In: Trials. 2020 Jun 3;21(1):471. **PMID:** 26941086 ## Document Section ### Large Document Module #### Large Docs - Date: 2021-02-15 - Filename: Prot_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: False - Label: Study Protocol - Size: 418360 - Type Abbrev: Prot - Upload Date: 2024-03-18T13:07 - Date: 2020-01-03 - Filename: SAP_001.pdf - Has ICF: False - Has Protocol: False - Has SAP: True - Label: Statistical Analysis Plan - Size: 585440 - Type Abbrev: SAP - Upload Date: 2024-03-18T13:11 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000010182 - Term: Pancreatic Diseases - ID: D000004066 - Term: Digestive System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC06 - Name: Digestive System Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M13115 - Name: Pancreatitis - Relevance: HIGH - As Found: Pancreatitis - 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 ### Condition Browse Module - Meshes - ID: D000010195 - Term: Pancreatitis ### Intervention Browse Module - Ancestors - ID: D000000894 - Term: Anti-Inflammatory Agents, Non-Steroidal - ID: D000018712 - Term: Analgesics, Non-Narcotic - ID: D000000700 - Term: Analgesics - ID: D000018689 - Term: Sensory System Agents - ID: D000018373 - Term: Peripheral Nervous System Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000000893 - Term: Anti-Inflammatory Agents - ID: D000018501 - Term: Antirheumatic Agents - ID: D000006074 - Term: Gout Suppressants - ID: D000015149 - Term: Tocolytic Agents - ID: D000012102 - Term: Reproductive Control Agents - ID: D000016861 - Term: Cyclooxygenase Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action ### Intervention Browse Module - Browse Branches - Abbrev: Infl - Name: Anti-Inflammatory Agents - Abbrev: ARhu - Name: Antirheumatic Agents - Abbrev: Analg - Name: Analgesics - Abbrev: Repr - Name: Reproductive Control Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M10257 - Name: Indomethacin - Relevance: HIGH - As Found: Noise - ID: M4217 - Name: Anti-Inflammatory Agents - Relevance: LOW - As Found: Unknown - ID: M4218 - Name: Anti-Inflammatory Agents, Non-Steroidal - Relevance: LOW - As Found: Unknown - ID: M4032 - Name: Analgesics - Relevance: LOW - As Found: Unknown - ID: M20786 - Name: Analgesics, Non-Narcotic - Relevance: LOW - As Found: Unknown - ID: M20604 - Name: Antirheumatic Agents - Relevance: LOW - As Found: Unknown - ID: M17869 - Name: Tocolytic Agents - Relevance: LOW - As Found: Unknown - ID: M19209 - Name: Cyclooxygenase Inhibitors - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000007213 - Term: Indomethacin ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** Adverse events were assessed at two follow up visits post-randomization: (1) 3-5 days after ERCP and (2) 30 (+/-5) days after ERCP. The follow-up was accomplished by directly contacting the subject or their treating healthcare provider in the event he or she had been hospitalized. #### Event Groups **Group ID:** EG000 **Title:** Indomethacin Alone **Deaths Num Affected:** 24 **Deaths Num At Risk:** 975 **Description:** Indomethacin 100 mg rectally immediately after ERCP, NO prophylactic pancreatic stent placement **ID:** EG000 **Other Num Affected:** 233 **Other Num at Risk:** 975 **Serious Number Affected:** 355 **Serious Number At Risk:** 975 **Title:** Indomethacin Alone **Group ID:** EG001 **Title:** Indomethacin+Pancreatic Stent **Deaths Num Affected:** 26 **Deaths Num At Risk:** 975 **Description:** Indomethacin 100 mg rectally immediately after ERCP AND prophylactic pancreatic stent placement **ID:** EG001 **Other Num Affected:** 228 **Other Num at Risk:** 975 **Serious Number Affected:** 352 **Serious Number At Risk:** 975 **Title:** Indomethacin+Pancreatic Stent **Frequency Threshold:** 0.5 #### Other Events **Term:** Abdominal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Abdominal pain upper **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Constipation **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:** Nausea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Vomiting **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Pyrexia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Lipase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (19.0) **Term:** Headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Dyspnoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) **Term:** Oropharyngeal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) #### Serious Events **Term:** Anaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 4 **Num At Risk:** 975 **Num Events:** 4 **Group ID:** EG001 **Num Affected:** 6 **Num At Risk:** 975 **Num Events:** 6 **Term:** Coagulopathy **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Leukocytosis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 6 **Num At Risk:** 975 **Num Events:** 6 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 4 **Term:** Neutropenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Sickle cell anaemia with crisis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Splenic haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Acute left ventricular failure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Atrial fibrillation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Bundle branch block left **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Cardiac arrest **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Term:** Cardiac failure congestive **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Sinus bradycardia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Sinus tachycardia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Tachycardia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Abdominal discomfort **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Term:** Abdominal distension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Abdominal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 140 **Num At Risk:** 975 **Num Events:** 163 **Group ID:** EG001 **Num Affected:** 166 **Num At Risk:** 975 **Num Events:** 177 **Term:** Abdominal pain upper **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Term:** Ascites **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Term:** Colitis ulcerative **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Constipation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Diarrhoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Term:** Diverticulum **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Duodenal perforation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 3 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 3 **Term:** Duodenal ulcer **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Dysphagia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Faecaloma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Gastric haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Gastritis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Gastrointestinal haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 4 **Num At Risk:** 975 **Num Events:** 4 **Group ID:** EG001 **Num Affected:** 8 **Num At Risk:** 975 **Num Events:** 8 **Term:** Gastrointestinal perforation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Haematemesis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Haematochezia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Term:** Haemorrhagic necrotic pancreatitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Haemorrhoids **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Ileus **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Intestinal perforation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Intra-abdominal haematoma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Intra-abdominal haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Localised intraabdominal fluid collection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Melaena **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 9 **Num At Risk:** 975 **Num Events:** 9 **Group ID:** EG001 **Num Affected:** 4 **Num At Risk:** 975 **Num Events:** 4 **Term:** Nausea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 16 **Num At Risk:** 975 **Num Events:** 16 **Group ID:** EG001 **Num Affected:** 9 **Num At Risk:** 975 **Num Events:** 9 **Term:** Pancreatic fistula **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Pancreatic haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Pancreatic mass **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Pancreatic necrosis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Pancreatitis haemorrhagic **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Pancreatitis necrotising **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Pancreatitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 90 **Num At Risk:** 975 **Num Events:** 92 **Group ID:** EG001 **Num Affected:** 76 **Num At Risk:** 975 **Num Events:** 80 **Term:** Peritoneal haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Proctalgia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Rectal haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Retroperitoneal haematoma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Small intestinal obstruction **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 3 **Group ID:** EG001 **Num Affected:** 5 **Num At Risk:** 975 **Num Events:** 5 **Term:** Vomiting **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 3 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Asthenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Chest pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 4 **Num At Risk:** 975 **Num Events:** 4 **Term:** Chills **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Death **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Generalised oedema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Hypothermia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Multiple organ dysfunction syndrome **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Oedema peripheral **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Term:** Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Term:** Perforation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Pyrexia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 6 **Num At Risk:** 975 **Num Events:** 6 **Group ID:** EG001 **Num Affected:** 6 **Num At Risk:** 975 **Num Events:** 6 **Term:** Systemic inflammatory response syndrome **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Bile duct obstruction **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num Affected:** 4 **Num At Risk:** 975 **Num Events:** 4 **Term:** Bile duct stone **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 3 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Biliary colic **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Biloma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Cholangitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 11 **Num At Risk:** 975 **Num Events:** 11 **Group ID:** EG001 **Num Affected:** 6 **Num At Risk:** 975 **Num Events:** 6 **Term:** Cholecystitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Cholecystitis acute **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Cholelithiasis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Cholestasis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Cryptogenic cirrhosis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Haemobilia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Hepatic failure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Hepatic haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Hepatorenal syndrome **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Hepatotoxicity **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Hyperbilirubinaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 4 **Num At Risk:** 975 **Num Events:** 6 **Term:** Jaundice **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Perforation bile duct **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Hepatobiliary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Hypersensitivity **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Immune system disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Transplant rejection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Immune system disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Abdominal abscess **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Abdominal infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Term:** Abscess **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Bacteraemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 3 **Term:** Biliary tract infection bacterial **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Clostridium difficile colitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 3 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Corona virus infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Cytomegalovirus colitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Gallbladder abscess **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Gangrene **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Gastrointestinal infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Hepatitis C **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Influenza **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Liver abscess **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Pancreas infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Pneumonia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 3 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Term:** Pneumonia staphylococcal **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Postoperative wound infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Pyelonephritis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Term:** Sepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 10 **Num At Risk:** 975 **Num Events:** 10 **Group ID:** EG001 **Num Affected:** 5 **Num At Risk:** 975 **Num Events:** 5 **Term:** Septic shock **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 3 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 3 **Term:** Streptococcal bacteraemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Urinary tract infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 3 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Term:** Anaemia postoperative **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Burns second degree **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Clavicle fracture **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Incisional hernia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Laceration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Peripancreatic fluid collection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Post procedural bile leak **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 5 **Num At Risk:** 975 **Num Events:** 5 **Group ID:** EG001 **Num Affected:** 4 **Num At Risk:** 975 **Num Events:** 4 **Term:** Post procedural haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 4 **Num At Risk:** 975 **Num Events:** 4 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Term:** Postoperative ileus **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Procedural pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Vascular pseudoaneurysm **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Vena cava injury **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Blood bilirubin **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Blood bilirubin increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Blood creatinine increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Coronavirus test positive **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Haemoglobin decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Hepatic enzyme increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 4 **Num At Risk:** 975 **Num Events:** 4 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Lipase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 3 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Term:** Liver function test increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Term:** Transaminases increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num At Risk:** 975 **Term:** White blood cell count increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Dehydration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 3 **Term:** Diabetes mellitus inadequate control **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Failure to thrive **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Hyperkalaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Term:** Hypoglycaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Hypokalaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Lactic acidosis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Metabolic alkalosis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Arthralgia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Back pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Flank pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Muscle spasms **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Adenocarcinoma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Term:** Adenocarcinoma pancreas **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** B-cell lymphoma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Bladder cancer **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Cholangiocarcinoma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 3 **Group ID:** EG001 **Num Affected:** 6 **Num At Risk:** 975 **Num Events:** 6 **Term:** Colon cancer **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Colon cancer metastatic **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 3 **Term:** Colorectal adenocarcinoma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Colorectal cancer metastatic **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Gastric cancer **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Hepatic cancer recurrent **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Hepatocellular carcinoma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 4 **Num At Risk:** 975 **Num Events:** 4 **Term:** Lung neoplasm malignant **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Malignant peritoneal neoplasm **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Metastases to central nervous system **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Metastatic neoplasm **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Term:** Neoplasm malignant **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Oesophageal carcinoma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Pancreatic carcinoma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 3 **Term:** Pancreatic carcinoma metastatic **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Tongue cancer metastatic **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Encephalopathy **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Term:** Ischaemic stroke **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Metabolic encephalopathy **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Seizure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Somnolence **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Syncope **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Tremor **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Device dislocation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Product Issues **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Confusional state **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 3 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 3 **Term:** Delirium **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Major depression **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Psychogenic seizure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Acute kidney injury **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 8 **Num At Risk:** 975 **Num Events:** 8 **Group ID:** EG001 **Num Affected:** 5 **Num At Risk:** 975 **Num Events:** 6 **Term:** Chronic kidney disease **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Dysuria **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Renal failure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Renal impairment **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Renal vein compression **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Urinary retention **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Female genital tract fistula **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Reproductive system and breast disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Acute pulmonary oedema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Acute respiratory distress syndrome **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Acute respiratory failure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num Affected:** 4 **Num At Risk:** 975 **Num Events:** 4 **Term:** Cough **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Dyspnoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Haemoptysis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Hypoxia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 3 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Pleural effusion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Pneumonia aspiration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Pulmonary oedema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Respiratory arrest **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Respiratory distress **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Respiratory failure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 3 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 3 **Term:** Tachypnoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Pruritus **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Pruritus generalised **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Rash **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Skin ulcer **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Cholecystectomy **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Surgical and medical procedures **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Term:** Extubation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Surgical and medical procedures **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Hepatectomy **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Surgical and medical procedures **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 975 **Num Events:** 2 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Flushing **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Hypertension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Hypotension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num Affected:** 3 **Num At Risk:** 975 **Num Events:** 4 **Term:** Hypovolaemic shock **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Term:** Peripheral ischaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 975 **Term:** Shock haemorrhagic **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA (19.0) ##### Stats **Group ID:** EG000 **Num At Risk:** 975 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 975 **Num Events:** 1 **Time Frame:** Non-serious adverse events were collected from randomization through the Day 5 follow up visit. Serious adverse events were collected from randomization through the end of study (Day 30). ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 975 **Group ID:** BG001 **Value:** 975 **Group ID:** BG002 **Value:** 1950 **Units:** Participants ### Group **ID:** BG000 **Title:** Indomethacin Alone **Description:** Indomethacin 100 mg rectally immediately after ERCP, NO prophylactic pancreatic stent placement ### Group **ID:** BG001 **Title:** Indomethacin+Pancreatic Stent **Description:** Indomethacin 100 mg rectally immediately after ERCP AND prophylactic pancreatic stent placement ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 16.4 **Value:** 55.6 #### Measurement **Group ID:** BG001 **Spread:** 16.3 **Value:** 55.8 #### Measurement **Group ID:** BG002 **Spread:** 16.4 **Value:** 55.7 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 975 **Group ID:** BG001 **Value:** 975 **Group ID:** BG002 **Value:** 1950 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 599 #### Measurement **Group ID:** BG001 **Value:** 596 #### Measurement **Group ID:** BG002 **Value:** 1195 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 376 #### Measurement **Group ID:** BG001 **Value:** 379 #### Measurement **Group ID:** BG002 **Value:** 755 **Category Title:** Male #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 975 **Group ID:** BG001 **Value:** 975 **Group ID:** BG002 **Value:** 1950 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 101 #### Measurement **Group ID:** BG001 **Value:** 99 #### Measurement **Group ID:** BG002 **Value:** 200 **Category Title:** Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 866 #### Measurement **Group ID:** BG001 **Value:** 871 #### Measurement **Group ID:** BG002 **Value:** 1737 **Category Title:** Not Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 8 #### Measurement **Group ID:** BG001 **Value:** 5 #### Measurement **Group ID:** BG002 **Value:** 13 **Category Title:** Unknown or Not Reported #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 975 **Group ID:** BG001 **Value:** 975 **Group ID:** BG002 **Value:** 1950 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 4 #### Measurement **Group ID:** BG001 **Value:** 3 #### Measurement **Group ID:** BG002 **Value:** 7 **Category Title:** American Indian or Alaska Native #### Measurement **Group ID:** BG000 **Value:** 17 #### Measurement **Group ID:** BG001 **Value:** 19 #### Measurement **Group ID:** BG002 **Value:** 36 **Category Title:** Asian #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 2 #### Measurement **Group ID:** BG002 **Value:** 3 **Category Title:** Native Hawaiian or Other Pacific Islander #### Measurement **Group ID:** BG000 **Value:** 115 #### Measurement **Group ID:** BG001 **Value:** 102 #### Measurement **Group ID:** BG002 **Value:** 217 **Category Title:** Black or African American #### Measurement **Group ID:** BG000 **Value:** 809 #### Measurement **Group ID:** BG001 **Value:** 825 #### Measurement **Group ID:** BG002 **Value:** 1634 **Category Title:** White #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 7 #### Measurement **Group ID:** BG002 **Value:** 10 **Category Title:** More than one race #### Measurement **Group ID:** BG000 **Value:** 26 #### Measurement **Group ID:** BG001 **Value:** 17 #### Measurement **Group ID:** BG002 **Value:** 43 **Category Title:** Unknown or Not Reported #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 975 **Group ID:** BG001 **Value:** 975 **Group ID:** BG002 **Value:** 1950 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 6.8 **Value:** 28.6 #### Measurement **Group ID:** BG001 **Spread:** 7.2 **Value:** 29.6 #### Measurement **Group ID:** BG002 **Spread:** 7.0 **Value:** 29.1 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 972 **Group ID:** BG001 **Value:** 975 **Group ID:** BG002 **Value:** 1947 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 363 #### Measurement **Group ID:** BG001 **Value:** 366 #### Measurement **Group ID:** BG002 **Value:** 729 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 868 **Group ID:** BG001 **Value:** 875 **Group ID:** BG002 **Value:** 1743 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 262 #### Measurement **Group ID:** BG001 **Value:** 252 #### Measurement **Group ID:** BG002 **Value:** 514 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 975 **Group ID:** BG001 **Value:** 975 **Group ID:** BG002 **Value:** 1950 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 36 #### Measurement **Group ID:** BG001 **Value:** 24 #### Measurement **Group ID:** BG002 **Value:** 60 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 975 **Group ID:** BG001 **Value:** 975 **Group ID:** BG002 **Value:** 1950 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 128 #### Measurement **Group ID:** BG001 **Value:** 126 #### Measurement **Group ID:** BG002 **Value:** 254 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 975 **Group ID:** BG001 **Value:** 975 **Group ID:** BG002 **Value:** 1950 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 795 #### Measurement **Group ID:** BG001 **Value:** 823 #### Measurement **Group ID:** BG002 **Value:** 1618 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 975 **Group ID:** BG001 **Value:** 975 **Group ID:** BG002 **Value:** 1950 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 112 #### Measurement **Group ID:** BG001 **Value:** 100 #### Measurement **Group ID:** BG002 **Value:** 212 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 975 **Group ID:** BG001 **Value:** 975 **Group ID:** BG002 **Value:** 1950 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 0 **Upper Limit:** 1 **Value:** 0 #### Measurement **Group ID:** BG001 **Lower Limit:** 0 **Upper Limit:** 2 **Value:** 1 #### Measurement **Group ID:** BG002 **Lower Limit:** 0 **Upper Limit:** 2 **Value:** 1 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 973 **Group ID:** BG001 **Value:** 974 **Group ID:** BG002 **Value:** 1947 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 58 #### Measurement **Group ID:** BG001 **Value:** 66 #### Measurement **Group ID:** BG002 **Value:** 124 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 975 **Group ID:** BG001 **Value:** 975 **Group ID:** BG002 **Value:** 1950 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 6 #### Measurement **Group ID:** BG001 **Value:** 8 #### Measurement **Group ID:** BG002 **Value:** 14 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 975 **Group ID:** BG001 **Value:** 975 **Group ID:** BG002 **Value:** 1950 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 864 #### Measurement **Group ID:** BG001 **Value:** 869 #### Measurement **Group ID:** BG002 **Value:** 1733 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 975 **Group ID:** BG001 **Value:** 975 **Group ID:** BG002 **Value:** 1950 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 555 #### Measurement **Group ID:** BG001 **Value:** 546 #### Measurement **Group ID:** BG002 **Value:** 1101 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 975 **Group ID:** BG001 **Value:** 975 **Group ID:** BG002 **Value:** 1950 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 935 **Value:** 1796 #### Measurement **Group ID:** BG001 **Spread:** 938 **Value:** 1852 #### Measurement **Group ID:** BG002 **Spread:** 937 **Value:** 1824 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 974 **Group ID:** BG001 **Value:** 974 **Group ID:** BG002 **Value:** 1948 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 1052 **Value:** 1554 #### Measurement **Group ID:** BG001 **Spread:** 1085 **Value:** 1606 #### Measurement **Group ID:** BG002 **Spread:** 1069 **Value:** 1581 #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 972 **Group ID:** BG001 **Value:** 973 **Group ID:** BG002 **Value:** 1945 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 18 #### Measurement **Group ID:** BG002 **Value:** 18 **Category Title:** 3 French #### Measurement **Group ID:** BG000 **Value:** 4 #### Measurement **Group ID:** BG001 **Value:** 165 #### Measurement **Group ID:** BG002 **Value:** 169 **Category Title:** 4 French #### Measurement **Group ID:** BG000 **Value:** 9 #### Measurement **Group ID:** BG001 **Value:** 586 #### Measurement **Group ID:** BG002 **Value:** 595 **Category Title:** 5 French #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 18 #### Measurement **Group ID:** BG002 **Value:** 18 **Category Title:** >5 French #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 3 **Category Title:** Missing #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 16 **Group ID:** BG001 **Value:** 787 **Group ID:** BG002 **Value:** 803 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 7 #### Measurement **Group ID:** BG001 **Value:** 455 #### Measurement **Group ID:** BG002 **Value:** 462 **Category Title:** 2-5cm #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 95 #### Measurement **Group ID:** BG002 **Value:** 98 **Category Title:** 6-8cm #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 237 #### Measurement **Group ID:** BG002 **Value:** 240 **Category Title:** >8cm #### Measurement **Group ID:** BG000 **Value:** 3 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 3 **Category Title:** Missing #### Denomination **Units:** Participants ##### Denomination Counts **Group ID:** BG000 **Value:** 16 **Group ID:** BG001 **Value:** 787 **Group ID:** BG002 **Value:** 803 **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 ### Measure 4 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Race (NIH/OMB) **Unit of Measure:** Participants ### Measure 5 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Population Description:** BMI missing for 3 participants **Title:** BMI **Unit of Measure:** kg/m^2 ### Measure 6 **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Missing in 207 patients (100 in indomethacin plus stent group, 107 in indomethacin alone group) due to question being added in August, 2016. **Title:** Antibiotic use in past 3 months **Unit of Measure:** Participants ### Measure 7 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Clinical suspicion or known sphincter of Oddi dysfunction **Unit of Measure:** Participants ### Measure 8 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** History of post-ERCP pancreatitis **Unit of Measure:** Participants ### Measure 9 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** History of recurrent pancreatitis **Unit of Measure:** Participants ### Measure 10 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Difficult cannulation **Unit of Measure:** Participants ### Measure 11 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Precut (access) sphincterotomy **Unit of Measure:** Participants ### Measure 12 **Dispersion Type:** INTER_QUARTILE_RANGE **Parameter Type:** MEDIAN **Population Description:** Missing in three patients (one in indomethacin plus stent group, two in indomethacin alone group). **Title:** Number of pancreatic injections **Unit of Measure:** injections ### Measure 13 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Pancreatic sphincterotomy **Unit of Measure:** Participants ### Measure 14 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Pancreatic acinarisation **Unit of Measure:** Participants ### Measure 15 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Biliary sphincterotomy **Unit of Measure:** Participants ### Measure 16 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Trainee involvement **Unit of Measure:** Participants ### Measure 17 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Population Description:** Missing in two patients (one in indomethacin plus stent group, one in indomethacin alone group). **Title:** Total intravenous fluid received during periprocedural period, mL **Unit of Measure:** mL ### Measure 18 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Population Description:** Missing in five patients (two in indomethacin plus stent group, three in indomethacin alone group). **Title:** Total intravenous lactated Ringer's fluid received during periprocedural period, mL **Unit of Measure:** mL ### Measure 19 **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Analysis population is those who received a stent **Title:** Prophylactic pancreatic stent calibre in those who received a stent **Unit of Measure:** Participants ### Measure 20 **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Analysis population is those who received a stent **Title:** Prophylactic pancreatic stent length in those who received a stent **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement ### Limitations and Caveats **Description:** Potential unmasking events occurred and could have influenced care. Decay in secondary outcome blinding, as unblinded clinician could resume care of patient after 48 h. Adjudicators provided radiographic information when assessing PEP severity, which may have influenced decision making. Unblinded endoscopists made procedural decisions, possibly altered according to study arm. Findings may not be applicable to community endoscopists since all sites were tertiary referral centers. ### Point of Contact **Email:** [email protected] **Organization:** Division of Gastroenterology & Hepatology, Medical University of South Carolina **Phone:** 8437926982 **Title:** Dr. B. Joseph Elmunzer ## Results Section - Outcome Measures Module ### Outcome Measure 1 #### Analysis **CI Lower Limit:** 0.006 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 0.066 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** These numbers are in the intention-to-treat analysis population. Risk difference in post-ERCP pancreatitis (PEP) is calculated as risk of PEP in indomethacin alone arm minus risk of PEP in indomethacin plus stent arm. **Group Description:** **Non-Inferiority Comment:** To declare noninferiority, the upper bound of the two-sided 95% CI for the risk difference in post-ERCP pancreatitis (indomethacin alone minus indomethacin plus stent) needed to be less than 5% in both the intention-to-treat and per protocol analysis populations. **Non-Inferiority Type:** NON_INFERIORITY **Other Analysis Description:** **P-Value:** **P-Value Comment:** **Parameter Type:** Risk Difference (RD) **Parameter Value:** 0.036 **Statistical Comment:** **Statistical Method:** **Tested Non-Inferiority:** #### Analysis **CI Lower Limit:** -0.003 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 0.060 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** These numbers are in the per protocol analysis population. Risk difference in post-ERCP pancreatitis (PEP) is calculated as risk of PEP in indomethacin alone arm minus risk of PEP in indomethacin plus stent arm. **Group Description:** **Non-Inferiority Comment:** To declare noninferiority, the upper bound of the two-sided 95% CI for the risk difference in post-ERCP pancreatitis (indomethacin alone minus indomethacin plus stent) needed to be less than 5% in both the intention-to-treat and per protocol analysis populations. **Non-Inferiority Type:** NON_INFERIORITY **Other Analysis Description:** **P-Value:** **P-Value Comment:** **Parameter Type:** Risk Difference (RD) **Parameter Value:** 0.028 **Statistical Comment:** **Statistical Method:** **Tested Non-Inferiority:** ### Outcome Measure 2 #### Analysis **CI Lower Limit:** -0.002 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 0.043 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** These numbers are in the intention-to-treat analysis population. Risk difference is calculated as risk of moderate-severe PEP in indomethacin alone arm minus risk of moderate-severe PEP in indomethacin plus stent arm. **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** **P-Value Comment:** **Parameter Type:** Risk Difference (RD) **Parameter Value:** 0.021 **Statistical Comment:** **Statistical Method:** **Tested Non-Inferiority:** #### Analysis **CI Lower Limit:** -0.004 **CI Number of Sides:** TWO_SIDED **CI Percentage Value:** 95 **CI Upper Limit:** 0.044 **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** These numbers are in the per protocol analysis population. Risk difference is calculated as risk of moderate-severe PEP in indomethacin alone arm minus risk of moderate-severe PEP in indomethacin plus stent arm. **Group Description:** **Non-Inferiority Comment:** **Non-Inferiority Type:** OTHER **Other Analysis Description:** **P-Value:** **P-Value Comment:** **Parameter Type:** Risk Difference (RD) **Parameter Value:** 0.020 **Statistical Comment:** **Statistical Method:** **Tested Non-Inferiority:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 145 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 110 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 975 - **Group ID:** OG001 - **Value:** 975 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 137 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 90 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 951 - **Group ID:** OG001 - **Value:** 777 **Units:** Participants #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 78 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 58 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 974 - **Group ID:** OG001 - **Value:** 974 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 74 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 45 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 951 - **Group ID:** OG001 - **Value:** 777 **Units:** Participants ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Post-ERCP pancreatitis (PEP) was based on a widely validated consensus definition that was applied as a diagnostic framework. In this consensus definition, PEP is diagnosed if there was new onset (or increase) of pain in the upper abdomen, elevation in pancreatic enzymes of at least three times the upper limit of normal 24 h after the procedure, and hospitalization for at least two nights. The outcome was independently adjudicated by 3 ERCP experts at non-enrolling centers based on review of the medical records for study participants who were hospitalized with any adverse event within 2 days of the ERCP. Medical records were redacted of all information that could potentially reveal study group assignment, including radiology reports. The consensus definition was applied as a diagnostic framework so that adjudicators could use their best judgment in cases that did not strictly satisfy the criteria. PEP was declared if there was agreement between at least two of the three adjudicators. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Intention-To-Treat and Per Protocol populations **Reporting Status:** POSTED **Time Frame:** Within 48 hours after ERCP **Title:** The Proportion of Subjects in Each Study Group With Post-ERCP Pancreatitis **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Indomethacin 100 mg rectally immediately after ERCP, NO prophylactic pancreatic stent placement **ID:** OG000 **Title:** Indomethacin Alone ##### Group **Description:** Indomethacin 100 mg rectally immediately after ERCP AND prophylactic pancreatic stent placement **ID:** OG001 **Title:** Indomethacin+Pancreatic Stent #### Outcome Measure 2 **Description:** Moderate or severe post-ERCP pancreatitis was based on the consensus definition as a diagnostic framework. For the severity assessment, radiographic information was made available to the adjudicators. The severity was defined as mild post-ERCP pancreatitis resulting in a hospitalization of ≤3 days, moderate post-ERCP pancreatitis resulting in a hospitalization of 4-10 days, and severe post-ERCP pancreatitis resulting in a hospitalization of \> 10 days, or leading to the development of pancreatic necrosis or pseudocyst, or requiring percutaneous or surgical intervention. The outcome was declared if there was agreement between at least two of the three adjudicators. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Intention-to-treat and per protocol. Outcome data missing for two participants (one in each treatment arm) and excluded from percentage denominator. **Reporting Status:** POSTED **Time Frame:** Within one month of ERCP **Title:** The Proportion of Subjects in Each Study Group With Moderate-severe Post-ERCP Pancreatitis **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Indomethacin 100 mg rectally immediately after ERCP, NO prophylactic pancreatic stent placement **ID:** OG000 **Title:** Indomethacin Alone ##### Group **Description:** Indomethacin 100 mg rectally immediately after ERCP AND prophylactic pancreatic stent placement **ID:** OG001 **Title:** Indomethacin+Pancreatic Stent ### Participant Flow Module #### Group **Description:** Indomethacin 100 mg rectally immediately after ERCP, NO prophylactic pancreatic stent placement **ID:** FG000 **Title:** Indomethacin Alone #### Group **Description:** Indomethacin 100 mg rectally immediately after ERCP AND prophylactic pancreatic stent placement **ID:** FG001 **Title:** Indomethacin+Pancreatic Stent #### Period **Title:** Overall Study ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 975 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 975 ##### Milestone **Type:** COMPLETED **Comment:** Completed follow-up for the primary and secondary endpoints ###### Achievement **Group ID:** FG000 **Number of Subjects:** 974 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 974 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 1 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 1 **Pre-Assignment Details:** Of 7243 patients assessed for eligibility, 1950 met eligibility criteria and were randomly assigned to treatment. **Recruitment Details:** Patients were recruited from 20 referral centers in the USA and Canada between September 2015 and January 2023. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT04483479 **Acronym:** Rollover **Brief Title:** Orally Administered ENT-01 for Parkinson's Disease-Related Constipation Follow-on Safety "Roll-over" Study (Rollover) **Official Title:** A Multicenter, Non-Randomized, Open-Label Study to Evaluate the Safety and Efficacy of Orally Administered ENT-01 in Improving Constipation and Neurologic Symptoms in Patients With Parkinson's Disease and Constipation Over a 14-week Period #### Organization Study ID Info **ID:** ENT-01-2b-20-02 #### Organization **Class:** INDUSTRY **Full Name:** Enterin Inc. ### Status Module #### Completion Date **Date:** 2022-02-17 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2024-03-19 **Type:** ACTUAL **Last Update Submit Date:** 2024-02-20 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2022-02-17 **Type:** ACTUAL #### Results First Post Date **Date:** 2024-03-19 **Type:** ACTUAL **Results First Submit Date:** 2023-10-03 **Results First Submit QC Date:** 2024-02-20 #### Start Date **Date:** 2020-07-30 **Type:** ACTUAL **Status Verified Date:** 2024-02 #### Study First Post Date **Date:** 2020-07-23 **Type:** ACTUAL **Study First Submit Date:** 2020-07-20 **Study First Submit QC Date:** 2020-07-22 **Why Stopped:** Poor recruitment/retention due to the pandemic. Data was not analyzed due to early termination. ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Enterin Inc. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Oversight Has DMC:** True ### Description Module **Brief Summary:** This study will be conducted as an open-label safety follow-on to a multi-center, double-blind, randomized study. All subjects who participated in the randomized study will be offered participation in this unblinded, single-arm, safety study. Approximately 50 subjects will be entered into the study and ENT-01 will be administered daily in escalating doses followed by a fixed dose for 12 weeks. Each subject will participate for approximately 20 weeks; dosing duration will be approximately 14 weeks. **Detailed Description:** The study will be conducted on an out-patient or in-patient basis with visits performed at the screening visit, at 6 and 12 weeks, and at the end of the 6th week of the wash-out period (end of study). The dose escalation period will last up to 20 days, the fixed dose period will last 12 weeks, and the wash-out period will last 6 weeks. Subjects will begin dosing at the same dose level they were stratified to in the ENT-01-030 randomized study (i.e., starting at either 3 tablets or 6 tablets,) and escalate up to a pro-kinetic dose or a maximum dose of 250mg. ### Conditions Module **Conditions:** - Parkinson Disease - Constipation ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP **Intervention Model Description:** This is a Phase 2b, non-randomized, open-label study. ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 27 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Orally Administered ENT-01 25mg Tablet Once Daily (Dose dependent on ENT-01-030 dose level stratification) **Intervention Names:** - Drug: Active Investigational Treatment ENT-01 **Label:** Active Treatment **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Active Treatment **Description:** ENT-01 will be administered in tablet form, once daily in escalating doses followed by a fixed-dose for 12 weeks. **Name:** Active Investigational Treatment ENT-01 **Other Names:** - ENT-01 **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Number of participants with Treatment Related Adverse Events **Measure:** Number of Participants With Treatment Related Adverse Events **Time Frame:** Through study treatment and completion up to 14 weeks **Description:** The number of participants with treatment related episodes of recurrent vomiting defined as 3-5 episodes of vomiting within 24 hours **Measure:** Number of Participants With Treatment Related Recurrent Vomiting **Time Frame:** Through study treatment and completion up to 14 weeks **Description:** The number of participants with treatment related episodes of recurrent diarrhea defined as 7 episodes of diarrhea within 24 hours for 2 consecutive days **Measure:** Number of Participants With Treatment Related Recurrent Diarrhea **Time Frame:** Through study treatment and completion up to 14 weeks **Description:** The number of participants with treatment related episodes of dizziness defined as lightheadedness or fainting on rising from lying to sitting or standing and severe enough to require non-urgent medical intervention within 24 hours **Measure:** Number of Participants With Treatment Related Dizziness **Time Frame:** Through study treatment and completion up to 14 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. All subjects who participated in the randomized study ENT-01-030 (KARMET Stage 2 Extension subjects) and who completed the dosing period. 2. Subjects aged 30-90 years at the time of screening for the ENT-01-030 study, both genders 3. Subjects must provide informed consent and be willing and able to comply with study procedures. 4. Subjects must be able to read, understand, and accurately record data into the diary to guarantee full participation in the study. 5. Female subjects must have negative serum or urine pregnancy tests and must not be lactating. For females able to bear children, a hormonal (i.e., oral, implantable, or injectable) and single-barrier method, or a double-barrier method of birth control must be used throughout the study. A vasectomized partner will be allowed as one in conjunction with another single-barrier method. 6. Female subjects unable to bear children must have this documented in the CRF (i.e., tubal ligation, hysterectomy, or postmenopausal \[defined as a minimum of one year since the last menstrual period\]). Post-menopausal status will be confirmed by follicle stimulating hormone (FSH) in women less than 60 years of age. Exclusion Criteria: 1. Unable or unwilling to provide informed consent or to comply with study procedures. 2. Unable to withdraw proton pump inhibitors. 3. Unable or unwilling to withdraw from laxatives, opiates, clonazepam, or any medications which may cause constipation 2 weeks prior to the dose adjustment period and throughout the rest of the study. 4. Diagnosis of secondary constipation beyond that of Parkinson's disease. 5. A compromised gastrointestinal system which includes: * Structural, metabolic, or functional GI diseases or disorders. * Acute GI illness within 2 weeks of the screening visit. * History of major GI surgery within 30 days of the screening visit (a history of cholecystectomy, polypectomy, hernia repair or appendicectomy are not exclusionary as long as they were performed more than 30 days before the screening visit). 6. Neurological disorder other than Parkinson's disease that in the opinion of the investigator might interfere with the conduct of the study. 7. On treatment with intra-jejunal dopamine or carbidopa/levodopa (i.e. Duopa). 8. Subjects starting a new Parkinson's disease medication or modifying an existing medication within 2 weeks prior to enrollment. 9. Unable to maintain a stable diet regimen. 10. Subjects with a cognitive impairment that preclude them from understanding the informed consent. 11. Subjects placed under legal guardianship. 12. History of excessive alcohol use or substance abuse. 13. Any clinically significant abnormalities on screening laboratories or physical examination requiring further evaluation or treatment. 14. Females who are pregnant or breastfeeding. 15. Subject or caregiver unable to administer daily oral dosing of study drug. 16. Participation in a non-Enterin investigational drug trial within the month prior to dosing in the present study. 17. Any other reason, which in the opinion of the investigator would confound proper interpretation of the study. **Maximum Age:** 90 Years **Minimum Age:** 30 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Fountain Valley **Country:** United States **Facility:** The Parkinson's and Movement Disorder Institute **State:** California **Zip:** 92708 **Location 2:** **City:** Pasadena **Country:** United States **Facility:** SC3 Research - Pasadena **State:** California **Zip:** 91105 **Location 3:** **City:** Englewood **Country:** United States **Facility:** Rocky Mountain Movement Disorders Center **State:** Colorado **Zip:** 80113 **Location 4:** **City:** Washington **Country:** United States **Facility:** Georgetown Universtiy, Department of Neurology **State:** District of Columbia **Zip:** 20007 **Location 5:** **City:** Atlantis **Country:** United States **Facility:** JEM Research Institute **State:** Florida **Zip:** 33462 **Location 6:** **City:** Boca Raton **Country:** United States **Facility:** Parkinson's Disease and Movement Disorders Center of Boca Raton **State:** Florida **Zip:** 33486 **Location 7:** **City:** Port Charlotte **Country:** United States **Facility:** Parkinson's Disease Treatment Center of SWFL **State:** Florida **Zip:** 33980 **Location 8:** **City:** Sarasota **Country:** United States **Facility:** Intercoastal Medical Group **State:** Florida **Zip:** 34239 **Location 9:** **City:** Tampa **Country:** United States **Facility:** USF Health Byrd Parkinson's Disease Movement Disorders Center of Excellence **State:** Florida **Zip:** 33613 **Location 10:** **City:** West Palm Beach **Country:** United States **Facility:** Premiere Research Institute at Palm Beach Neurology **State:** Florida **Zip:** 33407 **Location 11:** **City:** Lebanon **Country:** United States **Facility:** Dartmouth Hitchcock Medical Center **State:** New Hampshire **Zip:** 13756 **Location 12:** **City:** Albany **Country:** United States **Facility:** Albany Medical College **State:** New York **Zip:** 12208 **Location 13:** **City:** New York **Country:** United States **Facility:** Icahn School of Medicine at Mount Sinai **State:** New York **Zip:** 10029 **Location 14:** **City:** Cleveland **Country:** United States **Facility:** Cleveland Clinic **State:** Ohio **Zip:** 44095 **Location 15:** **City:** Toledo **Country:** United States **Facility:** University of Toledo Medical Center **State:** Ohio **Zip:** 43614 **Location 16:** **City:** Hershey **Country:** United States **Facility:** Penn State University **State:** Pennsylvania **Zip:** 17033 **Location 17:** **City:** Kirkland **Country:** United States **Facility:** Evergreen Health - Booth Gardner Parkinson's Care Center **State:** Washington **Zip:** 98034 **Location 18:** **City:** Huntington **Country:** United States **Facility:** University Physicians & Surgeons, Inc. dba Marshall Health **State:** West Virginia **Zip:** 25701 #### Overall Officials **Official 1:** **Affiliation:** Enterin Inc. **Name:** Michael Zasloff, MD, PhD **Role:** STUDY_CHAIR **Official 2:** **Affiliation:** Enterin Inc. **Name:** Denise Barbut, MD, FRCP **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Document Section ### Large Document Module #### Large Docs - Date: 2020-06-30 - Filename: Prot_SAP_001.pdf - Has ICF: False - Has Protocol: True - Has SAP: True - Label: Study Protocol and Statistical Analysis Plan - Size: 598378 - Type Abbrev: Prot_SAP - Upload Date: 2023-11-07T10:40 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020734 - Term: Parkinsonian Disorders - ID: D000001480 - Term: Basal Ganglia Diseases - ID: D000001927 - Term: Brain Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000009422 - Term: Nervous System Diseases - ID: D000009069 - Term: Movement Disorders - ID: D000080874 - Term: Synucleinopathies - ID: D000019636 - Term: Neurodegenerative Diseases - ID: D000012817 - Term: Signs and Symptoms, Digestive ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases ### Condition Browse Module - Browse Leaves - ID: M6472 - Name: Constipation - Relevance: HIGH - As Found: Constipation - ID: M13213 - Name: Parkinson Disease - Relevance: HIGH - As Found: Parkinson's Disease - ID: M22494 - Name: Parkinsonian Disorders - Relevance: LOW - As Found: Unknown - ID: M25603 - Name: Ganglion Cysts - Relevance: LOW - As Found: Unknown - ID: M16358 - Name: Synovial Cyst - Relevance: LOW - As Found: Unknown - ID: M4774 - Name: Basal Ganglia Diseases - Relevance: LOW - As Found: Unknown - ID: M5204 - Name: Brain Diseases - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: M12029 - Name: Movement Disorders - Relevance: LOW - As Found: Unknown - ID: M2217 - Name: Synucleinopathies - Relevance: LOW - As Found: Unknown - ID: M21558 - Name: Neurodegenerative Diseases - Relevance: LOW - As Found: Unknown - ID: M15622 - Name: Signs and Symptoms, Digestive - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000010300 - Term: Parkinson Disease - ID: D000003248 - Term: Constipation ### Misc Info Module #### Submission Tracking ##### First MCP Info ###### Post Date - Date: 2023-10-27 - Type: ACTUAL - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Active Treatment **Deaths Num At Risk:** 27 **Description:** Orally Administered ENT-01 25mg Tablet Once Daily (Dose dependent on ENT-01-030 dose level stratification) Active Investigational Treatment ENT-01: ENT-01 will be administered in tablet form, once daily in escalating doses followed by a fixed-dose for 12 weeks. **ID:** EG000 **Other Num Affected:** 24 **Other Num at Risk:** 27 **Serious Number At Risk:** 27 **Title:** Active Treatment **Frequency Threshold:** 5 #### Other Events **Term:** Nausea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (Unspecified) **Term:** Vomiting **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (Unspecified) **Term:** Diarrhoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (Unspecified) **Term:** Tremors **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (Unspecified) **Term:** Weight Decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (Unspecified) **Term:** Dizziness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA (Unspecified) **Time Frame:** 20 weeks ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 27 **Units:** Participants ### Group **ID:** BG000 **Title:** Active Treatment **Description:** Orally Administered ENT-01 25mg Tablet Once Daily (Dose dependent on ENT-01-030 dose level stratification) Active Investigational Treatment ENT-01: ENT-01 will be administered in tablet form, once daily in escalating doses followed by a fixed-dose for 12 weeks. ### 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:** 24 **Category Title:** >=65 years **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 9 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 18 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 3 **Category Title:** Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 23 **Category Title:** Not Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 1 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 27 **Class Title:** United States ### Measure #### Measurement **Group ID:** BG000 **Spread:** 4.63 **Value:** 26.7 **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:** Ethnicity (NIH/OMB) **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Region of Enrollment **Unit of Measure:** Participants ### Measure 5 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** BMI **Unit of Measure:** KG/m^2 ## Results Section - More Information Module ### Certain Agreement **Restrictive Agreement:** True ### Limitations and Caveats **Description:** Study was terminated early due to poor recruitment/retention due to the pandemic. ### Point of Contact **Email:** [email protected] **Organization:** Enterin **Phone:** 5054692670 **Title:** Dr. Richard Larson, Chief Medical Officer ## 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:** 23 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 8 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 8 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Number of participants with Treatment Related Adverse Events **Parameter Type:** COUNT_OF_PARTICIPANTS **Reporting Status:** POSTED **Time Frame:** Through study treatment and completion up to 14 weeks **Title:** Number of Participants With Treatment Related Adverse Events **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Orally Administered ENT-01 25mg Tablet Once Daily (Dose dependent on ENT-01-030 dose level stratification) Active Investigational Treatment ENT-01: ENT-01 will be administered in tablet form, once daily in escalating doses followed by a fixed-dose for 12 weeks. **ID:** OG000 **Title:** Active Treatment #### Outcome Measure 2 **Description:** The number of participants with treatment related episodes of recurrent vomiting defined as 3-5 episodes of vomiting within 24 hours **Parameter Type:** COUNT_OF_PARTICIPANTS **Reporting Status:** POSTED **Time Frame:** Through study treatment and completion up to 14 weeks **Title:** Number of Participants With Treatment Related Recurrent Vomiting **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Orally Administered ENT-01 25mg Tablet Once Daily (Dose dependent on ENT-01-030 dose level stratification) Active Investigational Treatment ENT-01: ENT-01 will be administered in tablet form, once daily in escalating doses followed by a fixed-dose for 12 weeks. **ID:** OG000 **Title:** Active Treatment #### Outcome Measure 3 **Description:** The number of participants with treatment related episodes of recurrent diarrhea defined as 7 episodes of diarrhea within 24 hours for 2 consecutive days **Parameter Type:** COUNT_OF_PARTICIPANTS **Reporting Status:** POSTED **Time Frame:** Through study treatment and completion up to 14 weeks **Title:** Number of Participants With Treatment Related Recurrent Diarrhea **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Orally Administered ENT-01 25mg Tablet Once Daily (Dose dependent on ENT-01-030 dose level stratification) Active Investigational Treatment ENT-01: ENT-01 will be administered in tablet form, once daily in escalating doses followed by a fixed-dose for 12 weeks. **ID:** OG000 **Title:** Active Treatment #### Outcome Measure 4 **Description:** The number of participants with treatment related episodes of dizziness defined as lightheadedness or fainting on rising from lying to sitting or standing and severe enough to require non-urgent medical intervention within 24 hours **Parameter Type:** COUNT_OF_PARTICIPANTS **Reporting Status:** POSTED **Time Frame:** Through study treatment and completion up to 14 weeks **Title:** Number of Participants With Treatment Related Dizziness **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Orally Administered ENT-01 25mg Tablet Once Daily (Dose dependent on ENT-01-030 dose level stratification) Active Investigational Treatment ENT-01: ENT-01 will be administered in tablet form, once daily in escalating doses followed by a fixed-dose for 12 weeks. **ID:** OG000 **Title:** Active Treatment ### Participant Flow Module #### Group **Description:** Orally Administered ENT-01 25mg Tablet Once Daily (Dose dependent on ENT-01-030 dose level stratification) Active Investigational Treatment ENT-01: ENT-01 will be administered in tablet form, once daily in escalating doses followed by a fixed-dose for 12 weeks. **ID:** FG000 **Title:** Active Treatment #### Period **Title:** Overall Study ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 27 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 12 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 15 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT04622579 **Acronym:** R2DLBCL80 **Brief Title:** Lenalidomide Combined With Rituximab as Front-line Therapy for DLBCL Patients Aged Over 80 Years **Official Title:** Lenalidomide Combined With Rituximab as Front-line Therapy in Elderly Patients Aged Over 80 Years With Diffuse Large B Cell Lymphoma #### Organization Study ID Info **ID:** 2020099H #### Organization **Class:** OTHER **Full Name:** Guangdong Provincial People's Hospital ### Status Module #### Completion Date **Date:** 2023-03-31 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2020-11-10 **Type:** ACTUAL **Last Update Submit Date:** 2020-11-05 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2022-12-31 **Type:** ESTIMATED #### Start Date **Date:** 2020-10-23 **Type:** ACTUAL **Status Verified Date:** 2020-11 #### Study First Post Date **Date:** 2020-11-10 **Type:** ACTUAL **Study First Submit Date:** 2020-11-05 **Study First Submit QC Date:** 2020-11-05 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Guangzhou First People's Hospital **Class:** OTHER **Name:** First People's Hospital of Foshan **Class:** OTHER **Name:** Huizhou Municipal Central Hospital **Class:** OTHER **Name:** Shantou Central Hospital #### Lead Sponsor **Class:** OTHER **Name:** Guangdong Provincial People's Hospital #### Responsible Party **Investigator Affiliation:** Guangdong Provincial People's Hospital **Investigator Full Name:** Wenyu Li **Investigator Title:** professor of medicine **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** About 40% of Diffuse large B cell lymphoma relapse or is refractory, age is a prognostic factor of DLBCL, as elderly patients are not capable to received standard treatment, the prognosis of elderly patients is poor especially those aged over 80 years. In this study,we aimed to investigate the safety and efficacy of the combination of lenalidomide and rituximab in elderly patients aged ≥ 80 years with untreated DLBCL. ### Conditions Module **Conditions:** - Diffuse Large B Cell Lymphoma - Age Over 80 Years **Keywords:** - lenalidomide ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 20 **Type:** ESTIMATED **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Rituximab 375 mg/m2 i.v d1 q28d； Lenalidomide 10mg Po. d1-21 q28d. After 6 cycles, patients obtained CR or PR will continue with Lenalidomide maintenance till the 24th month. **Intervention Names:** - Drug: lenalidomide combined with rituximab **Label:** lenalidomide combined with rituximab **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - lenalidomide combined with rituximab **Description:** lenalidomide and rituximab are dilivered as described, parameters concerning efficacy and safety are also obtained. **Name:** lenalidomide combined with rituximab **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Overall response rate **Measure:** ORR **Time Frame:** 24 months #### Secondary Outcomes **Description:** Complete response **Measure:** CR **Time Frame:** 24 months **Description:** Overall survival **Measure:** OS **Time Frame:** from date of treatment until the date of death from any cause, assessed up to 5 years **Description:** Progression-free survival **Measure:** PFS **Time Frame:** from date of treatment until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 5 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Age≧80 years ; 2. ECOG score 0-2； 3. untreated with pathologically confirmed CD20+ DLBCL ; 4. expected life expectancy of ≥ 12 weeks; 5. capable of swallowing tablets; 6. GFR(by Cockcroft- Gault)≥30 ml/min; 7. can sign written informed consent to participate in the study. Exclusion Criteria: 1. with CNS involvement; 2. with other malignancy (not including adequate-treated non-melanoma cutaneum carcinoma); patients with other cancer but disease-free for ≥5 years can enter this study; 3. with ≥ grade 2 peripheral neurophathy; 4. with cardiopathy including unstable angia or myocardial infarction over the past 8 weeks; 5. received live vaccine within 28 days.; 6. HIV-positive; 7. thrombosis ; 8. GFR\<30 mL/min; 9. other conditions not suitable for rituximab or lenalidomide application. **Minimum Age:** 80 Years **Sex:** ALL **Standard Ages:** - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Xinmiao Jiang **Phone:** （+86）20-83827812 **Role:** CONTACT #### Locations **Location 1:** **City:** Guangzhou **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Xinmiao Jiang - **Phone:** +86 20 81884713-80631 - **Role:** CONTACT **Country:** China **Facility:** Guangdong provincial people's hospital **State:** Guangdong **Status:** RECRUITING **Zip:** 510080 ## 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: M18831 - Name: Lymphoma, Large B-Cell, Diffuse - Relevance: HIGH - As Found: Diffuse Large B-Cell Lymphoma - ID: M18828 - Name: Lymphoma, B-Cell - Relevance: HIGH - As Found: B-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: LOW - As Found: Unknown - ID: T640 - Name: B-cell Lymphoma - Relevance: HIGH - As Found: B-cell Lymphoma - ID: T1866 - Name: Diffuse Large B-Cell Lymphoma - Relevance: HIGH - As Found: Diffuse Large B-Cell Lymphoma ### Condition Browse Module - Meshes - ID: D000008223 - Term: Lymphoma - ID: D000016393 - Term: Lymphoma, B-Cell - ID: D000016403 - Term: Lymphoma, Large B-Cell, Diffuse ### Intervention Browse Module - Ancestors - ID: D000074322 - Term: Antineoplastic Agents, Immunological - ID: D000000970 - Term: Antineoplastic Agents - ID: D000007155 - Term: Immunologic Factors - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018501 - Term: Antirheumatic Agents - ID: D000020533 - Term: Angiogenesis Inhibitors - ID: D000043924 - Term: Angiogenesis Modulating Agents - ID: D000006133 - Term: Growth Substances - ID: D000006131 - Term: Growth Inhibitors ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: ARhu - Name: Antirheumatic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M373 - Name: Rituximab - Relevance: HIGH - As Found: Healthy - ID: M1725 - Name: Lenalidomide - Relevance: HIGH - As Found: Respiratory - ID: M1346 - Name: Antineoplastic Agents, Immunological - 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: M22318 - Name: Angiogenesis Inhibitors - Relevance: LOW - As Found: Unknown - ID: M9231 - Name: Growth Inhibitors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000069283 - Term: Rituximab - ID: D000077269 - Term: Lenalidomide ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04110379 **Brief Title:** Effect of Virtual Reality Glasses as a Distraction Method on Child's Anxiety During Dental Treatment **Official Title:** Effect of Virtual Reality Glasses as a Distraction Method on Child's Anxiety During Dental Treatment(Randomized Controlled Clinical Trial) #### Organization Study ID Info **ID:** VR for anxiety #### Organization **Class:** OTHER **Full Name:** University of Alexandria ### Status Module #### Completion Date **Date:** 2020-10-07 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-12-04 **Type:** ACTUAL **Last Update Submit Date:** 2023-12-01 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2020-10-07 **Type:** ACTUAL #### Start Date **Date:** 2019-07-20 **Type:** ACTUAL **Status Verified Date:** 2023-12 #### Study First Post Date **Date:** 2019-10-01 **Type:** ACTUAL **Study First Submit Date:** 2019-09-24 **Study First Submit QC Date:** 2019-09-27 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Alexandria University #### Lead Sponsor **Class:** OTHER **Name:** Nourhan M.Aly #### Responsible Party **Investigator Affiliation:** University of Alexandria **Investigator Full Name:** Nourhan M.Aly **Investigator Title:** Instructor of Dental Public Health **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** The aim of the study is to evaluate and compare the effect of virtual reality glasses (VR) to conventional behavior management techniques as a distraction method on child's dental anxiety during dental treatment **Detailed Description:** The study to be conducted will be a randomized controlled clinical trial .The sample will consist of forty children presented to the Department of Pediatric Dentistry and Dental Public Health, 20 preschoolers of age ranging from 4-5 years old and 20 schoolers of age ranging from 6-8 years old. The eligible participants will be randomly divided into a study group where virtual reality glasses distraction will be used for child behavior management and a control group where conventional behavior management techniques (tell-show-do, distraction, and positive reinforcement) will be used. ### Conditions Module **Conditions:** - Dental Anxiety **Keywords:** - virtual reality - distraction - salivary cortisol ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** The eligible participants will be randomly and equally divided into group I (study) and group II (control). Each group will be further subdivided according to age into subgroup A (preschool aged children) and subgroup B (school aged children). ##### Masking Info **Masking:** NONE **Primary Purpose:** OTHER #### Enrollment Info **Count:** 40 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Child behavior management will be done using virtual reality glasses distraction (Remax Fantasy Land virtual reality glasses (Schenzen Remax Co.,Ltd)) **Intervention Names:** - Behavioral: Virtual Reality (VR) **Label:** Virtual Reality (VR) **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Child behavior management will be done using conventional behavior management techniques **Intervention Names:** - Behavioral: Conventional Behavior Management Techniques **Label:** Conventional Behavior Management **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Virtual Reality (VR) **Description:** All the dental procedures that will be done will be explained to the child using tell-show-do technique. VR glasses will be introduced to the child using tell-show-do technique and he will be given a choice of cartoon episodes to select from according to his own interest and age appropriate to view during the dental treatment. The child will be given five minutes to get familiar with the VR glasses before starting the dental treatment. **Name:** Virtual Reality (VR) **Type:** BEHAVIORAL #### Intervention 2 **Arm Group Labels:** - Conventional Behavior Management **Description:** Conventional behavior management techniques will be done to relieve the child's dental anxiety during the dental treatment such as : tell-show-do technique, distraction, and positive reinforcement, according to the child's behavior. **Name:** Conventional Behavior Management Techniques **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** This scale consists of 6 categories (range from 0 to 5) where 0= relaxed, 1=uneasy, 2= tense, 3= reluctant, 4= interference, 5= out of contact **Measure:** Preoperative Venham clinical anxiety rating scale **Time Frame:** Baseline; at the beginning of the visit **Description:** This scale consists of 6 categories (range from 0 to 5) where 0= relaxed, 1=uneasy, 2= tense, 3= reluctant, 4= interference, 5= out of contact **Measure:** Postoperative Venham clinical anxiety rating scale **Time Frame:** post dental treatment; after 2 hours **Description:** Each child will be asked to pool saliva in his/her mouth for 5 minutes and then passively drool it in the receiving vessel. The whole unstimulated salivary samples obtained will be centrifuged and 2 ml of each sample will be stored at -20º until being processed. The salivary samples will be analyzed for salivary cortisol using solid phase enzyme linked immunosorbent assay using DRG salivary cortisol ELISA kit. **Measure:** Preoperative evaluation of salivary cortisol level **Time Frame:** Baseline; at the beginning of the visit **Description:** Each child will be asked to pool saliva in his/her mouth for 5 minutes and then passively drool it in the receiving vessel. The whole unstimulated salivary samples obtained will be centrifuged and 2 ml of each sample will be stored at -20º until being processed. The salivary samples will be analyzed for salivary cortisol using solid phase enzyme linked immunosorbent assay using DRG salivary cortisol ELISA kit. **Measure:** Postoperative evaluation of salivary cortisol level **Time Frame:** post dental treatment; after 2 hours ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Healthy children (ASA category I). * Frankl behavior rating score 2 or 3. * Requiring pulpotomy in one of their primary molars. Exclusion Criteria: * Children taking medications that interfere with measures of salivary cortisol. * Presence of any systemic or mental disease. **Maximum Age:** 8 Years **Minimum Age:** 4 Years **Sex:** ALL **Standard Ages:** - CHILD ### Contacts Locations Module #### Locations **Location 1:** **City:** Alexandria **Country:** Egypt **Facility:** Faculty of Dentistry, Alexandria University **Zip:** 21512 #### Overall Officials **Official 1:** **Affiliation:** Faculty of Dentistry, Alexandria University, Egypt **Name:** Yomna Alaa Eldin, BDS **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** Faculty of Dentistry, Alexandria University, Egypt **Name:** Karin M.L. Dowidar, PhD **Role:** STUDY_DIRECTOR **Official 3:** **Affiliation:** Faculty of Dentistry, Alexandria University, Egypt **Name:** Laila El Habashy, PhD **Role:** STUDY_DIRECTOR **Official 4:** **Affiliation:** Faculty of Medicine, Alexandria University, Egypt **Name:** Akram Deghady, PhD **Role:** STUDY_DIRECTOR ### References Module #### References **Citation:** Al-Khotani A, Bello LA, Christidis N. Effects of audiovisual distraction on children's behaviour during dental treatment: a randomized controlled clinical trial. Acta Odontol Scand. 2016 Aug;74(6):494-501. doi: 10.1080/00016357.2016.1206211. Epub 2016 Jul 13. **PMID:** 27409593 **Citation:** Appukuttan DP. Strategies to manage patients with dental anxiety and dental phobia: literature review. Clin Cosmet Investig Dent. 2016 Mar 10;8:35-50. doi: 10.2147/CCIDE.S63626. eCollection 2016. **PMID:** 27022303 **Citation:** Asl Aminabadi N, Erfanparast L, Sohrabi A, Ghertasi Oskouei S, Naghili A. The Impact of Virtual Reality Distraction on Pain and Anxiety during Dental Treatment in 4-6 Year-Old Children: a Randomized Controlled Clinical Trial. J Dent Res Dent Clin Dent Prospects. 2012 Fall;6(4):117-24. doi: 10.5681/joddd.2012.025. Epub 2012 Nov 12. **PMID:** 23277857 **Citation:** Fakhruddin KS, Hisham EB, Gorduysus MO. Effectiveness of audiovisual distraction eyewear and computerized delivery of anesthesia during pulp therapy of primary molars in phobic child patients. Eur J Dent. 2015 Oct-Dec;9(4):470-475. doi: 10.4103/1305-7456.172637. **PMID:** 26929683 **Citation:** Gadicherla S, Shenoy RP, Patel B, Ray M, Naik B, Pentapati KC. Estimation of salivary cortisol among subjects undergoing dental extraction. J Clin Exp Dent. 2018 Feb 1;10(2):e116-e119. doi: 10.4317/jced.54369. eCollection 2018 Feb. **PMID:** 29670727 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000001523 - Term: Mental Disorders ### Condition Browse Module - Browse Branches - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M4324 - Name: Anxiety Disorders - Relevance: HIGH - As Found: Anxiety - ID: M4815 - Name: Mental Disorders - Relevance: LOW - As Found: Unknown - ID: M14473 - Name: Psychotic Disorders - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001008 - Term: Anxiety Disorders ### Intervention Browse Module - Browse Branches - Abbrev: Infl - Name: Anti-Inflammatory Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M9912 - Name: Hydrocortisone - Relevance: LOW - As Found: Unknown - ID: M155245 - Name: Hydrocortisone 17-butyrate 21-propionate - Relevance: LOW - As Found: Unknown - ID: M228609 - Name: Hydrocortisone acetate - Relevance: LOW - As Found: Unknown - ID: M263259 - Name: Hydrocortisone hemisuccinate - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02976779 **Brief Title:** A Phase I, Double Blind, Randomized, Placebo Controlled, Maximal Dose Study to Determine the Safety, Tolerability of Topical Cream Containing MGC (Medical Grade Cannabis) in Healthy Volunteers **Official Title:** A Phase I, Double Blind, Randomized, Placebo Controlled, Maximal Dose Study to Determine the Safety, Tolerability of Topical Cream Containing MGC (Medical Grade Cannabis) in Healthy Volunteers #### Organization Study ID Info **ID:** OWC-PSO-01 #### Organization **Class:** INDUSTRY **Full Name:** One World Cannabis Ltd. ### Status Module #### Completion Date **Date:** 2019-01-25 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2019-03-13 **Type:** ACTUAL **Last Update Submit Date:** 2019-03-11 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2019-01-25 **Type:** ACTUAL #### Start Date **Date:** 2017-02-03 **Type:** ACTUAL **Status Verified Date:** 2019-01 #### Study First Post Date **Date:** 2016-11-29 **Type:** ESTIMATED **Study First Submit Date:** 2016-11-25 **Study First Submit QC Date:** 2016-11-28 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** One World Cannabis Ltd. #### 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 single center, prospective, double-blind, placebo-controlled, randomized two parts study (part I and part II) will assess the safety of topical cream (3% CBD and 3% THC), applied twice daily (bid), in healthy subjects, for up to 6 treatment weeks and additional 2 follow-up weeks. **Detailed Description:** The study is divided into 2 stages. Healthy subjects will be consented to participate in the study. Part I is designed to evaluate the single dose safety assessment of single application of MGC cream in 26 healthy volunteers (intact skin) for up to 24 consecutive hours under admission conditions in the clinical site. In this stage, the subjects will be divided into 3 application doses. The following procedures will be performed: * Time point 0- IV line, BP, Heart Rate, Blood testing (chemistry, blood count, Cannabinoids) and clinician evaluation of cannabis related psychological aspect (such as evaluation the cannabis abuse screening test). * Time point 6 Hours- BP, Heart Rate Blood testing (chemistry, blood count, Cannabinoids) * Time point 12- BP, Heart Rate, Blood testing (chemistry, blood count, Cannabinoids) * Time point 24- BP, Heart Rate, Blood testing (chemistry, blood count, Cannabinoids) and and clinician evaluation of cannabis related psychological aspect (such as evaluation the cannabis abuse screening test). Part II that will be followed the 24 hours (of part I) is designed to evaluate the repeated applications safety of MGC cream in healthy volunteers for up to 6 weeks, both in the clinic and by subjects at home. 20 healthy subjects that will be treated twice daily with: * Maximal dose of 30 mg CBD : 30 mg THC, 1:1 ratio * Placebo Control - 0 mg CBD : 0 mg THC, 1:1 ratio (vehicle) This stage will be initiated at least 96 hour after completion part I of the study (washout period) .The placebo will be applied on one arm and the control on the second arm. Both, subject and investigator will be blinded as to the product/placebo allocation. Application at home will be performed twice a day. Subjects will be asked to attend the clinic on day 0 of the part II treatment, once a week during the 6 weeks of daily application and 2 weeks post application. In each visit the following evaluations will be performed: BP, Heart Rate, Blood testing (chemistry, blood count, Cannabinoids) and clinician of cannabis related psychological aspect (such as evaluation the cannabis abuse screening test). ### Conditions Module **Conditions:** - Safety Study for Future Treatment of Psoriasis ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 26 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Cannabis based topical cream 3% CBD and 3% THC. The cream was designed to treat Psoriasis . The cream will be applied twice daily. **Intervention Names:** - Drug: OWC MGC cream **Label:** OWC MGC cream **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Carrier cream. CBD and THC were taken out from the formulation. The cream will be applied twice daily. **Intervention Names:** - Drug: OWC Control Cream **Label:** OWC Control Cream **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - OWC MGC cream **Description:** Topical cream containing 3% CBD and 3% THC for Psoriasis treatment **Name:** OWC MGC cream **Other Names:** - OWC psoriasis cream **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - OWC Control Cream **Description:** Topical Cream without active ingredients **Name:** OWC Control Cream **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Collection of Adverse events and Serious Adverse Events **Measure:** Incidence rate of AEs/ SAEs **Time Frame:** 24 hours **Description:** Collection of Adverse events and Serious Adverse Events **Measure:** Incidence rate of AEs/ SAEs **Time Frame:** 6 weeks #### Secondary Outcomes **Description:** Erythema Edema Swelling Itching Heat Pain Cellulitis Other **Measure:** Specific AEs/ SAEs **Time Frame:** Baseline to 24 hours **Description:** Erythema Edema Swelling Itching Heat Pain Cellulitis Other **Measure:** Specific AEs/ SAEs **Time Frame:** baseline to 6 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Alanine aminotransferase (ALT), alkaline phosphatase and bilirubin \<=1.5xupper limit of normal (ULN) (isolated bilirubin \>1.5xULN is acceptable if bilirubin is fractionated and direct bilirubin \<35%). * Healthy as determined by a responsible and experienced physician, based on a medical evaluation including medical history, physical examination, laboratory tests and electrocardiogram (ECGs). * A subject with a clinical abnormality or laboratory parameters outside the reference range for the population being studied may be included only if the Investigator and the OWC Medical Monitor agree that the finding is unlikely to introduce additional risk factors and will not interfere with the study procedures. * Subjects with hemoglobin (Hb) values higher than ULN the normal range should always be excluded from enrollment. * A female subject is eligible to participate if she is of non-childbearing potential (postmenopausal or pre-menopausal females with a documented tubal ligation or hysterectomy). Females on hormone replacement therapy (HRT) and whose menopausal status is in doubt will be required to use one of the contraception methods in the protocol. * Male subjects with female partners of child-bearing potential must agree to use one of the contraception methods listed in the protocol. * Agree not to participate in any other interventional clinical trials during the study * Agree to follow study instructions meticulously Exclusion Criteria: * A positive pre-study Hepatitis B surface antigen or positive Hepatitis C antibody result within 3 months of screening. * Current or chronic history of liver disease, or known hepatic or biliary abnormalities (with the exception of Gilbert's syndrome or asymptomatic gallstones). * Have evidence of significant, uncontrolled hematological, endocrine, cerebrovascular, cardiovascular, coronary, pulmonary, gastrointestinal, or neurological disease. (Participants with hypothyroidism who are on adequate and stable thyroid replacement will not be excluded). * History of malignancy within 5 years of Screening or those with a strong family history of cancer (e.g., familial cancer disorders), with the exception of squamous cell or basal cell carcinoma of the skin that has been definitively treated. * A history of drug or alcohol abuse, or a history of regular alcohol consumption within 6 months of the study defined as an average weekly intake of \>14 drinks for males or \>7 drinks for females. One drink is equivalent to 12 gram of alcohol: 12 ounces (360 mililiter \[mL\]) of beer, 5 ounces (150 mL) of wine or 1.5 ounces (45 mL) of 80 proof distilled spirits. * Consumption of: Barbiturates, Cocaine, Ethanol, SSRI's, Protease inhibitors, Warfarin, Sildenafil, Theophilline, Tricyclic antidepressants * A positive test for human immunodeficiency virus (HIV) antibody. * The subject has participated in a clinical trial and has received an investigational product within the following time period prior to the first dosing day in the current study: 30 days, 5 half-lives (whichever is longer). * Unable to refrain from the use of prescription or non-prescription drugs, including vitamins, herbal and dietary supplements within 7 days (or 14 days if the drug is a potential enzyme inducer) or 5 half-lives (whichever is longer) prior to the first dose of study medication, unless in the opinion of the Investigator and OWC Medical Monitor the medication will not interfere with the study procedures or compromise subject safety. * History of sensitivity to any of the study medications, or components thereof or a history of drug or other allergy that, in the opinion of the investigator or OWC Medical Monitor, contraindicates their participation. * Pregnant females as determined by positive urine human chorionic gonadotropin test at screening or prior to dosing. * Are pregnant or nursing, or of child bearing potential and not practicing an effective means of birth control. * Are not able to give adequate informed consent. * Have any current problem or a history of substance abuse which, in the opinion of the investigator, might interfere with participation in the protocol. * Have used marijuana within a month of starting the study. * Fail the initial urine drug screen and blood test which tests for illicit drug use within the prior month. **Healthy Volunteers:** True **Maximum Age:** 60 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Ramat Gan **Country:** Israel **Facility:** Sheba Medical Center ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000017444 - Term: Skin Diseases, Papulosquamous - ID: D000012871 - Term: Skin Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC25 - Name: Substance Related Disorders - Abbrev: BXM - Name: Behaviors and Mental Disorders ### Condition Browse Module - Browse Leaves - ID: M14422 - Name: Psoriasis - Relevance: HIGH - As Found: Psoriasis - ID: M5449 - Name: Marijuana Abuse - Relevance: LOW - As Found: Unknown - ID: M15674 - Name: Skin Diseases - Relevance: LOW - As Found: Unknown - ID: M19713 - Name: Skin Diseases, Papulosquamous - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000011565 - Term: Psoriasis ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05021679 **Acronym:** AMP SWT **Brief Title:** AMP Step Wedge Trial **Official Title:** Activity and Mobility Promotion (AMP): Implementation and Impact of a Multifaceted Intervention to Increase Mobility in Hospital Patients #### Organization Study ID Info **ID:** IRB00270215 #### Organization **Class:** OTHER **Full Name:** Johns Hopkins University ### Status Module #### Completion Date **Date:** 2024-04-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2024-04-25 **Type:** ACTUAL **Last Update Submit Date:** 2024-04-23 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2024-04-01 **Type:** ACTUAL #### Start Date **Date:** 2021-01-01 **Type:** ACTUAL **Status Verified Date:** 2024-04 #### Study First Post Date **Date:** 2021-08-25 **Type:** ACTUAL **Study First Submit Date:** 2021-08-16 **Study First Submit QC Date:** 2021-08-24 ### 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:** False ### Description Module **Brief Summary:** Annually, more than 35 million patients are hospitalized in the United States. Many of these will experience hospital-acquired loss of physical functioning due to a lack of mobility during their in-patient stay. Such loss includes difficulties performing basic activities, such as rising from a chair, toileting, or ambulating. This loss of function may increase hospital length of stay (LOS), nursing home placement, and decrease mobility and participation in community activities even years after hospitalization. Prevention of this hospital-acquired functional loss is critical. Even the sickest hospitalized patients (e.g., those in the intensive care unit \[ICU\]), can safely and feasibly benefit from early mobilization. In the non-ICU setting there is evidence that patient mobilization reduces LOS and hospital costs, while improving patient satisfaction and physical and psychological outcomes. The overall objective of this proposed project is to evaluate the implementation and impact of a transdisciplinary and multifaceted mobility program (Johns Hopkins Activity and Mobility Promotion - AMP) on clinical outcomes among hospitalized adults. In addition to clinical outcomes, we will identify barriers and facilitators to high-performance program adoption. Results of this project will provide critical new insights on the effectiveness of AMP and inform dissemination and implementation nationwide. ### Conditions Module **Conditions:** - Immobility - Disability **Keywords:** - Activity - Mobility - Hospital-acquired disability ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP **Intervention Model Description:** Step-wedge clinical trial ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 16676 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Other: AMP Implementation **Label:** AMP Implementation **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - AMP Implementation **Description:** Each study site will implement the Johns Hopkins AMP program at different times. The AMP program includes nursing staff training on the goals of AMP, how to complete mobility-focused outcome measures, set mobility goals, and to safely mobilize/ambulate patients. The AMP program also includes embedding these outcome measures and mobility goals into electronic medical records and producing weekly/monthly reports that show how often nursing staff score patient mobility and help patients meet daily activity goals. **Name:** AMP Implementation **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Mobility goal is set and measured using the Johns Hopkins Highest Level of Mobility (JH-HLM) Scale **Measure:** % of patients meeting daily mobility goal **Time Frame:** 39 months **Description:** Mobility documentation to include Activity Measure for Post-Acute Care (AM-PAC) and JH-HLM **Measure:** % of patients with mobility measurements documented daily **Time Frame:** 39 months **Description:** To be extracted from electronic medical record **Measure:** % of patients receiving physical and/or occupational therapy consults **Time Frame:** 39 months #### Secondary Outcomes **Description:** To be extracted from electronic medical record **Measure:** Hospital length of stay (days) **Time Frame:** 39 months **Description:** Count of where patients are discharged to (e.g,. home, inpatient rehab unit) assessed by extraction from electronic medical record. **Measure:** Discharge disposition status **Time Frame:** 39 months **Description:** To be extracted from electronic medical record **Measure:** Number of physical and occupational therapy visits received during inpatient stay **Time Frame:** 39 months **Description:** Includes falls, pressure injury, and venous thromboembolism. To be extracted from electronic medical record **Measure:** Number of hospital-acquired morbidities **Time Frame:** 39 months **Description:** To be extracted from electronic medical record **Measure:** Number of patients with 30-day readmissions **Time Frame:** 39 months **Description:** Number of employee injuries from facilitating patient mobility as assessed by medical record extraction **Measure:** Employee injuries resulting from patient mobilization **Time Frame:** 39 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * All adult patients admitted to study hospital unit * Analysis will only include those with lengths of stay \>=3 days Exclusion Criteria: * Patients with active do-not-resuscitate (DNR) order * \<18 years old **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 Hospital **State:** Maryland **Zip:** 21287 #### Overall Officials **Official 1:** **Affiliation:** Johns Hopkins University **Name:** Sapna Kudchadkar, MD **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** Johns Hopkins University **Name:** Erik Hoyer, PhD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01442779 **Brief Title:** Clinical Trial of Low Dose Oral Interferon Alpha in Idiopathic Pulmonary Fibrosis **Official Title:** Clinical Trial of Low Dose Oral Interferon Alpha in Idiopathic Pulmonary Fibrosis #### Organization Study ID Info **ID:** 00190 #### Organization **Class:** OTHER **Full Name:** Texas Tech University Health Sciences Center ### Status Module #### Completion Date **Date:** 2007-05 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2012-04-20 **Type:** ESTIMATED **Last Update Submit Date:** 2012-04-18 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2007-05 **Type:** ACTUAL #### Results First Post Date **Date:** 2011-09-29 **Type:** ESTIMATED **Results First Submit Date:** 2009-09-23 **Results First Submit QC Date:** 2011-08-23 #### Start Date **Date:** 2000-09 **Status Verified Date:** 2012-04 #### Study First Post Date **Date:** 2011-09-29 **Type:** ESTIMATED **Study First Submit Date:** 2009-09-23 **Study First Submit QC Date:** 2011-08-23 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Ainos, Inc. (f/k/a Amarillo Biosciences Inc. #### Lead Sponsor **Class:** OTHER **Name:** Texas Tech University Health Sciences Center #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to determine the possible efficacy of low dose, orally administered interferon alpha in subjects with Idiopathic Pulmonary Fibrosis (IPF). **Detailed Description:** This is a pilot study to determine if oral administration of low doses of Interferon alpha might be effective in treating Idiopathic Pulmonary Fibrosis (IPF). This is a disease that damages the lungs leading to marked decreases in the quality of life and death within 3-5 years after diagnosis. The cause is unknown. The standard treatment has for some time been steroids such as prednisone or prednisolone because of their anti-inflammatory actions, but there is little evidence that steroids either improve the condition, prevent further deterioration or improve life expectancy. Additionally, they have many side effects. In this disease, normal cells are damaged for unknown reasons and replaced by a type of scar. This scar tissue prevents the easy movement of oxygen from the lungs into the blood, making it difficult for the patient to perform normal activities. With progression, which usually occurs rapidly, patients require supplemental oxygen to perform even simple tasks. Interferons are chemicals normally produced in the body and the rate of their production has been shown to be reduced in the lungs of patients with IPF. They are involved in regulating the activity of the immune system which may play a role in initiating the damage to the lungs in IPF and they also can inhibit the activity of the cells that form the scar tissue. Our hypothesis is that treating patients with interferon might prevent damage to additional normal tissue and prevent the formation of additional scar tissue. This would prevent progression, improve the quality of life and extend the expected life span if successful. Another study has been ongoing in which IPF patients have been given injections of large doses of another type of interferon. This treatment regimen is expensive and side effects have been fairly frequent. In contrast, we are treating IPF patients with low doses of interferon administered orally. The interferon is taken three times per day by letting a lozenge dissolve in the mouth. These low doses have been shown to produce effects in patients with other diseases and they produce very few side effects. If side effects occur, they usually are not severe and go away quickly. Those reported most commonly by other subjects have been headaches, nausea, rashes, respiratory infections, sore throat or diarrhea. No one has had to stop taking the medicine because of the side effects. The medicine is provided free of charge. This study has been going on for about 5 years. The subjects are given the same tests that they receive as part of their standard of care. These include chest x-rays, High Resolution CT scans, pulmonary function tests and some blood tests. They are done before starting interferon alpha, and, depending on the test, are repeated at 3-, 6-, 9- or 12 month intervals. In addition subjects are asked to complete questionnaires on the quality of life, cough history and a dyspnea index at each visit. ### Conditions Module **Conditions:** - Respiratory Tract Diseases - Lung Diseases - Lung Diseases, Interstitial - Pulmonary Fibrosis **Keywords:** - Idiopathic Pulmonary Fibrosis - Interferon alpha ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 18 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Description:** dose form - oral lozenge dose - 150 International Units (IU) frequency - 3 times a day duration - at least 1 year **Name:** Interferon alpha oral lozenge **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Disease progression was determined by comparing results of the High Resolution Computed Tomography(HRCT) and pulmonary function at one year to the baseline HRCT \& pulmonary function. The same radiologist did the comparsion for all subjects. **Measure:** Minimal/no Progression (1 yr) by High Resolution Computed Tomography (HRCT) & Pulmonary Function **Time Frame:** 1 yr **Measure:** Minimal/no Change in Quality of Life **Time Frame:** 12 months #### Secondary Outcomes **Description:** changes in cough status after treatment for 1 month. **Measure:** Participants With Change in Cough **Time Frame:** 1 month ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * The only subjects to be included in this study are those diagnosed with Idiopathic Pulmonary Fibrosis with diagnosis based on the criteria published by the American Thoracic Society in the International Consensus Statement. 1. Exclusion of other known causes of interstitial lung disease. 2. Abnormal pulmonary function studies. 3. Bibasilar reticular abnormalities with minimal ground glass opacities on HRCT scan. 4. Biopsy or lavage showing no features supporting alternative diagnosis. 5. Patient older than 50 years of age. 6. Insidious onset of otherwise unexplained dyspnea on exertion. 7. Duration greater than 3 months. 8. Bibasilar, inspiratory crackles. Exclusion Criteria: * under the age of 50 * history of hypersensitivity to interferons * history of hypersensitivity to biological products such as vaccines * pregnant or lactating women * women of child bearing age not pregnancy protected during the study * unresolved serious cardiovascular disease **Minimum Age:** 50 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Lubbock **Country:** United States **Facility:** Texas Tech University Health Sciences Center **State:** Texas **Zip:** 79430 #### Overall Officials **Official 1:** **Affiliation:** Texas Tech University Health Sciences Center **Name:** Cynthia Jumper, MD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M8485 - Name: Fibrosis - Relevance: HIGH - As Found: Fibrosis - ID: M11168 - Name: Lung Diseases - Relevance: HIGH - As Found: Lung Disease - ID: M14512 - Name: Pulmonary Fibrosis - Relevance: HIGH - As Found: Pulmonary Fibrosis - ID: M27989 - Name: Idiopathic Pulmonary Fibrosis - Relevance: HIGH - As Found: Idiopathic Pulmonary Fibrosis - ID: M19813 - Name: Lung Diseases, Interstitial - Relevance: HIGH - As Found: Lung Diseases, Interstitial - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: HIGH - As Found: Respiratory Tract Diseases - ID: T3003 - Name: Idiopathic Pulmonary Fibrosis - Relevance: HIGH - As Found: Idiopathic Pulmonary Fibrosis ### Condition Browse Module - Meshes - ID: D000008171 - Term: Lung Diseases - ID: D000011658 - Term: Pulmonary Fibrosis - ID: D000054990 - Term: Idiopathic Pulmonary Fibrosis - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000017563 - Term: Lung Diseases, Interstitial - ID: D000005355 - Term: Fibrosis ### Intervention Browse Module - Ancestors - ID: D000000970 - Term: Antineoplastic 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: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M10407 - Name: Interferons - Relevance: HIGH - As Found: Recurrent - ID: M19243 - Name: Interferon-alpha - Relevance: HIGH - As Found: Status - 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: D000007372 - Term: Interferons - ID: D000016898 - Term: Interferon-alpha ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Interferon Alpha **Description:** Treatment with low dose oral interferon alpha lozenges taken 3 times daily (approximately 6 hours apart). Lozenge is to be dissolved under tongue or by moving around in mouth. **ID:** EG000 **Other Num at Risk:** 18 **Serious Number Affected:** 4 **Serious Number At Risk:** 18 **Title:** Interferon Alpha **Frequency Threshold:** 0 #### Serious Events **Term:** Death **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Notes:** The deaths that occurred were complications of the disease (IPF), not related to the study drug **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 4 **Num At Risk:** 18 **Num Events:** 4 ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 18 **Units:** Participants ### Group **ID:** BG000 **Title:** Interferon Alpha **Description:** Treatment with low dose oral interferon alpha lozenges taken 3 times daily (approximately 6 hours apart). Lozenge is to be dissolved under tongue or by moving around in mouth. ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** <=18 years #### Measurement **Group ID:** BG000 **Value:** 7 **Category Title:** Between 18 and 65 years #### Measurement **Group ID:** BG000 **Value:** 11 **Category Title:** >=65 years **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Spread:** 8.8 **Value:** 67 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 9 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 9 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 18 **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 **PI Sponsor Employee:** True ### Point of Contact **Email:** [email protected] **Organization:** Texas Tech University Health Sciences Center **Phone:** 806-743-2532 **Title:** Lorenz Lutherer, MD, PhD ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 12 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 12 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Disease progression was determined by comparing results of the High Resolution Computed Tomography(HRCT) and pulmonary function at one year to the baseline HRCT \& pulmonary function. The same radiologist did the comparsion for all subjects. **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** 1 yr **Title:** Minimal/no Progression (1 yr) by High Resolution Computed Tomography (HRCT) & Pulmonary Function **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Treatment with low dose oral interferon alpha lozenges taken 3 times daily (approximately 6 hours apart). Lozenge is to be dissolved under tongue or by moving around in mouth. **ID:** OG000 **Title:** Interferon Alpha #### Outcome Measure 2 **Parameter Type:** NUMBER **Population Description:** Analysis was performed only on those subject who continue on medication for at least one year. **Reporting Status:** POSTED **Time Frame:** 12 months **Title:** Minimal/no Change in Quality of Life **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Treatment with low dose oral interferon alpha lozenges taken 3 times daily (approximately 6 hours apart). Lozenge is to be dissolved under tongue or by moving around in mouth. **ID:** OG000 **Title:** Interferon Alpha #### Outcome Measure 3 **Description:** changes in cough status after treatment for 1 month. **Parameter Type:** NUMBER **Population Description:** During the course of the study it was noted that subjects experienced a change in the cough that is sometimes associated with IPF. The 6 subjects still enrolled in the study were asked to complete a questionnare regarding the status of the cough. **Reporting Status:** POSTED **Time Frame:** 1 month **Title:** Participants With Change in Cough **Type:** SECONDARY **Unit of Measure:** Participants ##### Group **Description:** Treatment with low dose oral interferon alpha lozenges taken 3 times daily (approximately 6 hours apart). Lozenge is to be dissolved under tongue or by moving around in mouth. **ID:** OG000 **Title:** Interferon Alpha ### Participant Flow Module #### Group **Description:** Treatment with low dose oral interferon alpha lozenges taken 3 times daily (approximately 6 hours apart). Lozenge is to be dissolved under tongue or by moving around in mouth. **ID:** FG000 **Title:** Interferon Alpha #### Period **Title:** Overall Study ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 3 ##### Withdraw **Type:** Death ###### Reason **Group ID:** FG000 **Number of Subjects:** 3 ##### Withdraw **Type:** Non-compliance ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 18 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 11 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 7 **Recruitment Details:** Medical clinic **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT02658279 **Brief Title:** Pembrolizumab (MK-3475) in Patients With Recurrent Malignant Glioma With a Hypermutator Phenotype **Official Title:** A Proof-of-Concept, Pilot Study of Pembrolizumab (MK-3475) in Patients With Recurrent Malignant Glioma With a Hypermutator Phenotype #### Organization Study ID Info **ID:** 15-227 #### Organization **Class:** OTHER **Full Name:** Memorial Sloan Kettering Cancer Center ### Status Module #### Completion Date **Date:** 2025-01 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-04-11 **Type:** ACTUAL **Last Update Submit Date:** 2024-04-10 **Overall Status:** ACTIVE_NOT_RECRUITING #### Primary Completion Date **Date:** 2025-01 **Type:** ESTIMATED #### Start Date **Date:** 2016-01-22 **Type:** ACTUAL **Status Verified Date:** 2024-04 #### Study First Post Date **Date:** 2016-01-18 **Type:** ESTIMATED **Study First Submit Date:** 2016-01-12 **Study First Submit QC Date:** 2016-01-14 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Merck Sharp & Dohme LLC **Class:** OTHER **Name:** M.D. Anderson Cancer Center **Class:** OTHER **Name:** Dana-Farber Cancer Institute **Class:** OTHER **Name:** University of California, San Francisco **Class:** UNKNOWN **Name:** Huntsman Cancer Institute/ University of Utah **Class:** OTHER **Name:** University of California, Los Angeles **Class:** OTHER **Name:** University of Miami #### Lead Sponsor **Class:** OTHER **Name:** Memorial Sloan Kettering Cancer Center #### Responsible Party **Type:** SPONSOR ### Description Module **Brief Summary:** The purpose of this study is to test if the study drug called pembrolizumab could control the growth or shrink the cancer but it could also cause side effects. Researchers hope to learn if the study drug will shrink the cancer by half, or prevent it from growing for at least 6 months. Pembrolizumab is an antibody that targets the immune system and activates it to stop cancer growth and/or kill cancer cells. ### Conditions Module **Conditions:** - Glioma - Recurrent Malignant Glioma **Keywords:** - Pembrolizumab (MK-3475) - 15-227 - Hypermutator Phenotype ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 27 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Pembrolizumab 200 mg will be administered as an approximately 30 minute (-5/+10 mins) IV infusion every 3 weeks. Sites should make every effort to target infusion timing to be as close to 30 minutes as possible. Patients will be followed with DCE perfusion MRI done at baseline and every 9 weeks (+/- 7 days). Patients will be allowed to stay on treatment in spite of increase in tumor size, provided the patient has no, or manageable, new neurologic symptoms, and at the discretion of treating physician. In that situation, tumor resection should be encouraged for the distinction between tumor progression and immunologic reactions. Patients undergoing surgical resection will have tissue collected for collateral studies. All collected tissue will be stored in the Pathology Core Tissue bank at MSK. **Intervention Names:** - Drug: Pembrolizumab **Label:** Pembrolizumab (MK-3475) **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Pembrolizumab (MK-3475) **Name:** Pembrolizumab **Other Names:** - (MK-3475) **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** patients with stable disease will be considered responders if disease is stable for 27 weeks or more. The regimen will be considered worthy of further study if responses as defined above are observed in at least 3 of the 12 subjects. All subjects will undergo DCE-MRI of the head at the time points specified in Study Flow Chart . Local radiologic assessment of tumor measurements will be used during the study for clinical management and investigator-assessed disease progression. Additional MRIs may be obtained at the discretion of the investigators, as clinically indicated. Radiologic response (complete response or partial response) will be assessed by comparing on-treatment MRI scans with the pretreatment baseline MRI scans. Radiologic progression will be determined by using the smallest tumor measurement of either the pretreatment baseline or after initiation of study medication. The RANO criteria will be used for determining disease status. **Measure:** response rate **Time Frame:** 1 year ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Histologically confirmed diagnosis of malignant glioma by enrolling institution: * WHO grade IV tumors (GBM or its variants) * WHO grade III anaplastic astrocytoma or oligodendroglial tumors or * WHO grade II gliomas, if MRI shows contrast enhancement * Tumor recurrence after previous treatment, which must have included at least radiation therapy and one cytotoxic chemotherapy. There is no limit on number of previous recurrences or lines of treatment. * Previously obtained tumor sample exhibits a hypermutator phenotype. For the purposes of this trial, a hypermutator phenotype is defined as tumors harboring 30 mutations (non-synonymous somatic point or indel mutations) detected by the MSK-IMPACT or comparable next generation sequencing performed in a CLIA environment. Contingent to approval by the MSK Principal Investigator, patients with less than 30 mutations may be eligible if they display a mutation in a mismatch repair gene or other mutations in genes known to be associated with hypermutator phenotypes or microsatellite instability, including but not limited to MLH1, MSH2, MSH6, PMS2, POLE, POLD as determined by validated methods, or if microsatellite instability is present, as identified by polymerase chain reaction (PCR) or other validated methods. \*Note: The MSK-IMPACT (Integrated Mutation Profiling for Actionable Cancer Targets) assay is a next generation genomic profiling performed on formalin-fixed, paraffin-embedded (FFPE) tumor tissue in a CLIA-certified Molecular Diagnostic Service laboratory. IMPACT provides full exon coverage of 410 cancer related genes and can detect base substitutions, small indels, copy number alterations and selected gene re-rearrangements. In some cases, additional assays such as Sanger Sequencing or fluorescence in situ hybridization (FISH) may be required to confirm specific results detected on IMPACT. Patients at MSK will have this assay to determine eligibility. Use of other validated next-generation sequencing techniques for eligibility may be considered, provided they are performed in a CLIA-certified laboratory and are approved by the MSK Principal Investigator. * Be willing and able to provide written informed consent/assent for the trial. * Be ≥ 18 years of age on day of signing informed consent. * An interval of ≥ 12 weeks from the end of prior radiation therapy is required unless there is either: i) histopathologic confirmation of recurrent tumor, or ii) new enhancement on MRI outside of the radiation treatment field * An interval of ≥ 4 weeks after the last administration of any investigational agent or any other treatment prior to first dose of pembrolizumab * Must have recovered (i.e., ≤ Grade 1 or at baseline) from adverse events of any previous treatment. Note: Surgical resection for recurrent tumor prior to enrollment is allowed. * Karnofsky performance status of ≥ 70 * Demonstrate adequate organ function as per below. All screening labs should be performed within 14 days of treatment initiation. * Absolute neutrophil count (ANC) ≥1,500 /mcL * Platelets ≥100,000 / mcL * Hemoglobin ≥9 g/dL or ≥ 5.6 mmol/L without transfusion or EPO dependency (within 7 days of assessment) * Serum creatinine ≤1.5 X upper limit of normal (ULN) OR measured or calculated creatinine clearance (GFR can also be used in place of creatinine or CrCl) ≥ 60 mL/min for subject with creatinine levels \> 1.5 X institutional ULN * Serum total bilirubin ≤ 1.5 X ULN OR direct bilirubin ≤ ULN for subjects with total bilirubin levels \> 1.5 ULN * AST (SGOT) and ALT (SGPT) ≤ 2.5 X ULN * Albumin \>2.5 mg/dL * International Normalized Ratio (INR) or Prothrombin Time (PT) ≤1.5 X ULN unless subject is receiving anticoagulant therapy as long as PT or PTT is within therapeutic range of intended use of anticoagulants * Activated Partial Thromboplastin Time (aPTT) ≤1.5 X ULN unless subject is receiving anticoagulant therapy as long as PT or PTT is within therapeutic range of intended use of anticoagulants * Female subject of childbearing potential should have a negative urine or serum pregnancy test. If the urine test is positive or cannot be confirmed as negative, a serum pregnancy test will be required. * Female subjects of childbearing potential should be willing to use 2 methods of birth control or be surgically sterile, or abstain from heterosexual activity for the course of the study through 120 days after the last dose of study medication (Reference Section 9.7.2). Subjects of childbearing potential are those who have not been surgically sterilized or have not been free from menses for \> 1 year. * Male subjects should agree to use an adequate method of contraception starting with the first dose of study therapy through 120 days after the last dose of study therapy. Exclusion Criteria: * Subjects requiring escalating or chronic supraphysiologic doses of corticosteroids (\> 10 mg/d of prednisone equivalent) for control of disease at the time of registration * Previous or current treatment with an anti-CTLA-4, anti-PD-1, anti-PD-L1, or anti-PD-L2 agent. * Hypersensitivity to pembrolizumab or any of its excipients. * Has a diagnosis of immunodeficiency, including Human Immunodeficiency Virus (HIV) or acquired immunodeficiency syndrome (AIDS) * Has known active Hepatitis B (e.g., HBsAg reactive) or Hepatitis C (e.g., HCV RNA \[qualitative\] is detected). * Has a known history of active TB (Bacillus Tuberculosis) * Has a known additional malignancy that is progressing or requires active treatment. Exceptions include basal cell carcinoma of the skin or squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer. * Has active autoimmune disease that has required systemic treatment in the past 2 years (i.e. with use of disease modifying agents, corticosteroids or immunosuppressive drugs). Replacement therapy (eg., thyroxine, insulin, or physiologic corticosteroid replacement therapy for adrenal or pituitary insufficiency, etc.) is not considered a form of systemic treatment. * Has known history of, or any evidence of active, non-infectious pneumonitis. * Has an active infection requiring systemic therapy. * Has a history or current evidence of any condition, therapy, or laboratory abnormality that might confound the results of the trial, interfere with the subject's participation for the full duration of the trial, or is not in the best interest of the subject to participate, in the opinion of the treating investigator. * Has known psychiatric or substance abuse disorders that would interfere with cooperation with the requirements of the trial. * Is pregnant or breastfeeding, or expecting to conceive or father children within the projected duration of the trial, starting with the pre-screening or screening visit through 120 days after the last dose of trial treatment. * Unable to undergo MRI of the brain (i.e. pacemaker or any other contraindication for MRIs). * Has received a live vaccine within 30 days of planned start of study therapy. Note: Seasonal influenza vaccines for injection are generally inactivated flu vaccines and are allowed; however intranasal influenza vaccines (e.g., Flu-Mist®) are live attenuated vaccines, and are not allowed. * Has previously received treatment with bevacizumab **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Los Angeles **Country:** United States **Facility:** University of California, Los Angeles **State:** California **Zip:** 90095 **Location 2:** **City:** San Francisco **Country:** United States **Facility:** University of California San Francisco **State:** California **Zip:** 94143 **Location 3:** **City:** Miami **Country:** United States **Facility:** University of Miami **State:** Florida **Location 4:** **City:** Boston **Country:** United States **Facility:** Massachusetts General Hospital **State:** Massachusetts **Zip:** 02114 **Location 5:** **City:** Boston **Country:** United States **Facility:** Brigham and Women's Hospital **State:** Massachusetts **Zip:** 02115 **Location 6:** **City:** Boston **Country:** United States **Facility:** Dana Farber Cancer Institute **State:** Massachusetts **Zip:** 02115 **Location 7:** **City:** Basking Ridge **Country:** United States **Facility:** Memorial Sloan Kettering Cancer Center **State:** New Jersey **Location 8:** **City:** Middletown **Country:** United States **Facility:** Memorial Sloan Kettering Monmouth **State:** New Jersey **Zip:** 07748 **Location 9:** **City:** Montvale **Country:** United States **Facility:** Memorial Sloan Kettering Bergen **State:** New Jersey **Zip:** 07645 **Location 10:** **City:** Commack **Country:** United States **Facility:** Memorial Sloan Kettering Commack **State:** New York **Zip:** 11725 **Location 11:** **City:** Harrison **Country:** United States **Facility:** Memorial Sloan Kettering Westchester **State:** New York **Zip:** 10604 **Location 12:** **City:** New York **Country:** United States **Facility:** Memorial Sloan Kettering Cancer Center **State:** New York **Zip:** 10065 **Location 13:** **City:** Uniondale **Country:** United States **Facility:** Memorial Sloan Kettering Nassau **State:** New York **Zip:** 11553 **Location 14:** **City:** Cleveland **Country:** United States **Facility:** Cleveland Clinic **State:** Ohio **Zip:** 44195 **Location 15:** **City:** Houston **Country:** United States **Facility:** Md Anderson Cancer Center **State:** Texas **Zip:** 77030 **Location 16:** **City:** Salt Lake City **Country:** United States **Facility:** University of Utah **State:** Utah **Zip:** 84112 #### Overall Officials **Official 1:** **Affiliation:** Memorial Sloan Kettering Cancer Center **Name:** Thomas Kaley, MD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### See Also Links **Label:** Memorial Sloan Kettering Cancer Center **URL:** https://www.mskcc.org/ ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020969 - Term: Disease Attributes - ID: D000010335 - Term: Pathologic Processes - ID: D000018302 - Term: Neoplasms, Neuroepithelial - ID: D000017599 - Term: Neuroectodermal Tumors - ID: D000009373 - Term: Neoplasms, Germ Cell and Embryonal - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000009369 - Term: Neoplasms - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009380 - Term: Neoplasms, Nerve Tissue ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M9020 - Name: Glioma - Relevance: HIGH - As Found: Malignant Glioma - ID: M14850 - Name: Recurrence - Relevance: HIGH - As Found: Recurrent - ID: M22700 - Name: Disease Attributes - Relevance: LOW - As Found: Unknown - ID: M20446 - Name: Neoplasms, Neuroepithelial - Relevance: LOW - As Found: Unknown - ID: M19845 - Name: Neuroectodermal Tumors - Relevance: LOW - As Found: Unknown - ID: M20388 - Name: Neuroectodermal Tumors, Primitive - Relevance: LOW - As Found: Unknown - ID: M12318 - Name: Neoplasms, Germ Cell and Embryonal - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown - ID: M12320 - Name: Neoplasms, Glandular and Epithelial - Relevance: LOW - As Found: Unknown - ID: M12325 - Name: Neoplasms, Nerve Tissue - Relevance: LOW - As Found: Unknown - ID: T2519 - Name: Glioma - Relevance: HIGH - As Found: Glioma - ID: T4092 - Name: Neuroepithelioma - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000005910 - Term: Glioma - ID: D000012008 - Term: Recurrence ### 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 ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M349416 - Name: Pembrolizumab - Relevance: HIGH - As Found: Blind - ID: M1346 - Name: Antineoplastic Agents, Immunological - Relevance: LOW - As Found: Unknown - ID: M2342 - Name: Immune Checkpoint Inhibitors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: C000582435 - Term: Pembrolizumab ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05516979 **Acronym:** EXPAND **Brief Title:** A Safety & Efficacy Study of Treatment With AP1189 in Rheumatoid Arthritis Patients naïve to DMARD Treatment **Official Title:** A Double-blind, Multi-center, Randomized, Placebo-controlled Study of the Safety and Efficacy of 12 Weeks Extended Treatment With AP1189 in Early Rheumatoid Arthritis (RA) Patients naïve to DMARD Treatment #### Organization Study ID Info **ID:** SynAct-CS007 #### Organization **Class:** INDUSTRY **Full Name:** SynAct Pharma Aps ### Status Module #### Completion Date **Date:** 2023-07-20 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-11-07 **Type:** ACTUAL **Last Update Submit Date:** 2023-11-03 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2023-07-20 **Type:** ACTUAL #### Start Date **Date:** 2022-09-26 **Type:** ACTUAL **Status Verified Date:** 2023-11 #### Study First Post Date **Date:** 2022-08-26 **Type:** ACTUAL **Study First Submit Date:** 2022-08-24 **Study First Submit QC Date:** 2022-08-25 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** NBCD A/S #### Lead Sponsor **Class:** INDUSTRY **Name:** SynAct Pharma Aps #### 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 study is a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety and efficacy of 12 weeks daily treatment with 100 mg AP1189 in RA patients who are to start up-titration with methotrexate (MTX). ### Conditions Module **Conditions:** - Rheumatoid Arthritis ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** Multi-center, randomized, double-blind, placebo-controlled study with 12 weeks of treatment with AP1189 or placebo ##### Masking Info **Masking:** TRIPLE **Who Masked:** - PARTICIPANT - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 127 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Treatment period of 12 weeks given as 1 tablet daily **Intervention Names:** - Drug: 100 mg AP1189 **Label:** 100 mg AP1189 **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Treatment period of 12 weeks given as 1 tablet daily **Intervention Names:** - Drug: Placebo **Label:** Placebo **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - 100 mg AP1189 **Description:** AP1189 tablets for oral use **Name:** 100 mg AP1189 **Other Names:** - AP1189 tablets for oral use **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Placebo **Description:** Matching placebo for oral use **Name:** Placebo **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Evaluation of the safety and tolerability of AP1189 on the number and severity of reported Adverse Events, compared with placebo **Measure:** Number of reported AEs **Time Frame:** 12 weeks **Description:** The change in American College of Rheumatology 20% (ACR20) compared to baseline **Measure:** Change in ACR20 **Time Frame:** 12 weeks #### Secondary Outcomes **Description:** The change in American College of Rheumatology 50% (ACR50) compared to baseline **Measure:** Change in ACR50 **Time Frame:** 12 weeks **Description:** The change in American College of Rheumatology 70% (ACR70) compared to baseline **Measure:** Change in ACR70 **Time Frame:** 12 weeks **Description:** The change Clinical Disease Activity Index (CDAI) compared to baseline **Measure:** Change in (CDAI) **Time Frame:** 12 weeks **Description:** The change in DAS-28, based on a CRP value, compare to baseline **Measure:** Change in DAS-28 **Time Frame:** 12 weeks ### Eligibility Module **Eligibility Criteria:** Main Inclusion Criteria: * Confirmed diagnosis of RA according to the 2010 ACR/EULAR RA classification criteria and are ACR class I-III * ≥ 6 swollen joints (based on 66 joint counts) and ≥ 6 tender joints (based on 68 joint counts). * Candidate for MTX treatment * Is about to begin treatment with MTX * Must meet at least one of the following parameters at Screening: 1. positive result for anti-CCP or RF 2. Serum CRP ≥ 6 mg/L * Highly active RA (CDAI \> 22) at screening and baseline * Negative QuantiFERON-in-Tube test (QFG-IT) * Females of child-bearing potential must use of highly effective birth control method Main Exclusion Criteria: * Major surgery (including joint operation) within 8 weeks prior to screening or planned surgery within 1 month following randomization * Rheumatic autoimmune disease other than RA, including systemic lupus erythematosus (SLE), mixed connective tissue disease (MCTD), scleroderma, polymyositis, or significant systemic involvement secondary to RA. Sjögren's syndrome with RA is allowable * Prior history of or current inflammatory joint disease other than RA * Subjects with fibromyalgia * Use of hydroxychloroquine within 4 weeks prior the Screening Visit * Initiation of, or change in existing NSAID treatment within 2 weeks prior to the baseline visit * Corticosteroids except inhaled or nasal formulations for seasonal allergy or asthma are prohibited within 2 weeks prior to screening * Evidence of serious uncontrolled concomitant cardiovascular, nervous system, pulmonary, renal, hepatic, endocrine, or gastrointestinal disease * Have prior renal transplant, current renal dialysis, or severe renal insufficiency * Uncontrolled disease states, such as asthma, psoriasis, or inflammatory bowel disease where flares are commonly treated with oral or parenteral corticosteroids * Evidence of active malignant disease (except basal cell carcinoma of the skin that has been excised and cured) * Neuropathies or other painful conditions that might interfere with pain evaluation * Body weight of \>150 kg * HBsAg positive and/or Anti-HBc with sign of current infection. **Maximum Age:** 85 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Chișinău **Country:** Moldova, Republic of **Facility:** Timofei Mosneaga Republican Clinical Hospital ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007592 - Term: Joint Diseases - ID: D000009140 - Term: Musculoskeletal Diseases - ID: D000012216 - Term: Rheumatic Diseases - ID: D000003240 - Term: Connective Tissue Diseases - ID: D000001327 - Term: Autoimmune Diseases - ID: D000007154 - Term: Immune System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: BC20 - Name: Immune System Diseases ### Condition Browse Module - Browse Leaves - ID: M4476 - Name: Arthritis - Relevance: HIGH - As Found: Arthritis - ID: M4480 - Name: Arthritis, Rheumatoid - Relevance: HIGH - As Found: Rheumatoid Arthritis - ID: M10621 - Name: Joint Diseases - Relevance: LOW - As Found: Unknown - ID: M12097 - Name: Musculoskeletal Diseases - Relevance: LOW - As Found: Unknown - ID: M15045 - Name: Rheumatic Diseases - Relevance: LOW - As Found: Unknown - ID: M6323 - Name: Collagen Diseases - Relevance: LOW - As Found: Unknown - ID: M6464 - Name: Connective Tissue Diseases - Relevance: LOW - As Found: Unknown - ID: M4629 - Name: Autoimmune Diseases - Relevance: LOW - As Found: Unknown - ID: M10200 - Name: Immune System Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001168 - Term: Arthritis - ID: D000001172 - Term: Arthritis, Rheumatoid ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00579579 **Brief Title:** Functional Outcomes and Quality of Life in Patients Undergoing Surgery for Rectal Cancer **Official Title:** Functional Outcomes and Quality of Life in Patients Undergoing Surgery for Rectal Cancer #### Organization Study ID Info **ID:** 06-151 #### Organization **Class:** OTHER **Full Name:** Memorial Sloan Kettering Cancer Center ### Status Module #### Completion Date **Date:** 2023-02-15 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-02-17 **Type:** ACTUAL **Last Update Submit Date:** 2023-02-15 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2023-02-15 **Type:** ACTUAL #### Start Date **Date:** 2006-11-28 **Type:** ACTUAL **Status Verified Date:** 2023-02 #### Study First Post Date **Date:** 2007-12-24 **Type:** ESTIMATED **Study First Submit Date:** 2007-12-20 **Study First Submit QC Date:** 2007-12-20 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Memorial Sloan Kettering Cancer Center #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of this study is to find out what happens to patients after they have surgery for rectal cancer. After being treated for rectal cancer, patients tell us that their bowel, bladder and sexual function have changed. We are trying to understand how these changes affect your quality of life. The research that we have now does not explain these changes or problems very well. The patients will be asked questions about bowel function, bladder function, sexual function, and quality of life so we can understand these changes better. This will help us take better care of our patients in the future, before and after their treatment for rectal cancer. **Detailed Description:** Rectal cancer is the second most common cancer in North America. Therapy has rapidly improved over the last 20 years, and the surgical technique of total mesorectal excision, as well as advances in radiation and chemotherapy, have resulted in improved survival and decreased local recurrence. As a result, survivorship issues become increasingly important for patients with rectal cancer. Patients uniformly demonstrate a strong desire to avoid a permanent stoma and show strong preferences for sphincter preserving surgery (SPS). With the introduction of the circular stapler, SPS is technically possible in a higher proportion of patients. Additionally, even tumors at the anorectal ring are considered amenable to SPS in select patients with ultra-low rectal cancers. At present, long-term outcomes after rectal cancer surgery are poorly understood. Bowel, bladder and sexual function appear to be negatively affected by multi-modality therapy. However, function has been poorly studied, and it is difficult to translate the data into clinically meaningful information for patients. Clinically, bowel, bladder and sexual dysfunction seem to affect quality of life (QOL), although this has never been well studied. It is important to quantify the extent of impairment so that it can be used to educate patients preoperatively. However, translating these data to clinicians and patients remains challenging, and efforts to convey the data in a meaningful manner preoperatively constitute an important element in managing patient expectations. By understanding patients' baseline needs, expectations and satisfaction at the time of the preoperative consent, we can begin to develop novel preoperative strategies for educating patients about postoperative function and quality of life in a meaningful manner, so that they may better adapt after surgery. We ultimately plan to use data from this study to develop and subsequently evaluate the role of an educational tool outlining functional outcomes after rectal cancer surgery. ### Conditions Module **Conditions:** - Rectal Cancer **Keywords:** - Bowel function - Bladder function - Sexual function - Quality of life ### Design Module #### Design Info **Observational Model:** CASE_ONLY **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 229 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients Undergoing Surgery for Rectal Cancer **Intervention Names:** - Behavioral: Questionaires **Label:** 1 ### Interventions #### Intervention 1 **Arm Group Labels:** - 1 **Description:** Prior to surgery, patients will be asked to complete baseline surveys. In addition, a random subsample of approximately 30 patients will be asked to participate in a short qualitative interview to explore expectations regarding bowel function and quality of life following surgery. Follow-up surveys will be completed at 6, 12 and 24 months after bowel continuity has been restored (defined by their last surgical procedure). Patients with a permanent stoma will receive the Stoma-specific QOL questions in place of the MSKCC Bowel Function Instrument at 6 and 12 months after initial surgery. **Name:** Questionaires **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Measure:** To prospectively evaluate bowel, sexual and bladder function in patients who undergo resection for stage I-III rectal cancer. **Time Frame:** Two sets of surveys before surgery. After surgery, the participant will be asked to fill out some or all of a series of surveys at 6, 12, and 24 months. #### Secondary Outcomes **Measure:** To assess the impact of bowel, bladder and sexual function on quality of life over time in patients undergoing resection for stage I-III rectal cancer and to compare QOL in patients with and without a stoma. **Time Frame:** Two sets of surveys before surgery. After surgery, the participant will be asked to fill out some or all of a series of surveys at 6, 12, and 24 months. **Measure:** To evaluate the expectations, informational needs and satisfaction with the surgical consent process in patients undergoing resection for stage I-III rectal cancer. **Time Frame:** Two sets of surveys before surgery. After surgery, the participant will be asked to fill out some or all of a series of surveys at 6, 12, and 24 months. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Stage I-III rectal adenocarcinoma based on preoperative testing * Surgery (Sphincter preserving - transanal (TAE),transanal endoscopic microsurgery (TEM), low anterior resection (LAR), coloanal resection (CAA), OR Permanent stoma - abdominal perineal resection (APR)) planned at MSKCC * Age \> or = to 18 * Speak English Exclusion Criteria: * Stage IV disease at time of pre-operative consult * History of other malignancies (besides squamous cell or basal cell cancer of skin) less than five years ago **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Participants are being asked to take part in this study because they have rectal cancer and are planning on having surgery done at MSKCC. ### Contacts Locations Module #### Locations **Location 1:** **City:** New York **Country:** United States **Facility:** Memorial Sloan-Kettering Cancer Center **State:** New York **Zip:** 10065 #### Overall Officials **Official 1:** **Affiliation:** Memorial Sloan Kettering Cancer Center **Name:** Julio Garcia-Aguilar, MD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### See Also Links **Label:** Memorial Sloan-Kettering Cancer Center **URL:** http://www.mskcc.org ## Derived Section ### Condition Browse Module - Ancestors - ID: D000015179 - Term: Colorectal Neoplasms - ID: D000007414 - Term: Intestinal Neoplasms - 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: D000007410 - Term: Intestinal Diseases - ID: D000012002 - Term: Rectal Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC06 - Name: Digestive System Diseases - Abbrev: All - Name: All Conditions - Abbrev: BXM - Name: Behaviors and Mental Disorders ### Condition Browse Module - Browse Leaves - ID: M14846 - Name: Rectal Neoplasms - Relevance: HIGH - As Found: Rectal Cancer - ID: M17890 - Name: Colorectal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M10448 - Name: Intestinal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M8886 - Name: Gastrointestinal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7256 - Name: Digestive System Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7255 - Name: Digestive System Diseases - Relevance: LOW - As Found: Unknown - ID: M8883 - Name: Gastrointestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M10444 - Name: Intestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M14844 - Name: Rectal Diseases - Relevance: LOW - As Found: Unknown - ID: T6034 - Name: Quality of Life - Relevance: HIGH - As Found: Quality of Life ### Condition Browse Module - Meshes - ID: D000012004 - Term: Rectal Neoplasms ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03637179 **Brief Title:** Acute Exposure to High Altitude on Pulmonary Artery Pressure and Right Heart Function (Echo) Under Exercise **Official Title:** Acute Exposure to Hypoxia in Precapillary Pulmonary Hypertension: Physiological and Clinical Effects at Rest and During Exercise #### Organization Study ID Info **ID:** 2018-00455_B4 #### Organization **Class:** OTHER **Full Name:** University of Zurich ### Status Module #### Completion Date **Date:** 2019-01-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2020-01-07 **Type:** ACTUAL **Last Update Submit Date:** 2020-01-06 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2019-01-01 **Type:** ACTUAL #### Start Date **Date:** 2018-07-01 **Type:** ACTUAL **Status Verified Date:** 2020-01 #### Study First Post Date **Date:** 2018-08-17 **Type:** ACTUAL **Study First Submit Date:** 2018-07-09 **Study First Submit QC Date:** 2018-08-16 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Zurich #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Randomized crossover Trial in patients with Pulmonary Hypertension (PAH, CTEPH) to assess the acute response to High Altitude (2500m above sea level) on pulmonary artery pressure and right heart function (Echo) under exercise. **Detailed Description:** Low altitude baseline measurements will be performed in Zurich (460m asl) including Echocardiography, Right heart catheterization, six-minute walk test (6MWT), pulmonary function test, clinical assessment and blood gas Analysis. Randomly assigned to the order of testing, the participants will be tested in Low Altitude (Zurich, 470m) and at High Altitude (2500m). Several times within the exposure, the pulmonary artery pressure and the right heart function will be assessed by echo under exercise. ### Conditions Module **Conditions:** - Pulmonary Hypertension **Keywords:** - High Altitude - Pulmonary artery pressure - Echocardiography - Right heart function - exercise ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER **Intervention Model Description:** Crossover Assignment Including a baseline assessment and assessments at Low altitude and High altitude ##### Masking Info **Masking:** NONE **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 28 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** The participants will be tested in Zurich (Low altitude: 470m above sea level) and consecutively at High Altitude(Säntis; 2500m above sea Level) **Intervention Names:** - Other: Assessment at Low Altitude (470m above sea level) - Other: Exposure to High Altitude (2500m above sea level) **Label:** Order A **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** The participants will be tested at High Altitude (Säntis; 2500m above sea level) and consecutively in Zurich (Low altitude; 470m above sea level). **Intervention Names:** - Other: Assessment at Low Altitude (470m above sea level) - Other: Exposure to High Altitude (2500m above sea level) **Label:** Order B **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Order A - Order B **Description:** Assessment at Low Altitude (in Zurich; 470m above sea level) in order to compare this data with High altitude exposure **Name:** Assessment at Low Altitude (470m above sea level) **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - Order A - Order B **Description:** Exposure to High Altitude (Säntis; 2500m above sea level) for approximately 5 hours **Name:** Exposure to High Altitude (2500m above sea level) **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Pulmonary artery pressure will be measured by echo (TTE) under exercise. **Measure:** Echocardiographic Assessment at High Altitude (2500m) during exercise **Time Frame:** 1 day at altitude #### Secondary Outcomes **Description:** The right heart function (fac, D-Shaping, etc.) will be assessed by echocardiography (TTE) under exercise **Measure:** Echocardiographic right heart assessment at High Altitude (2500m) during exercise **Time Frame:** 1 day at altitude ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria * Informed consent * PH diagnosed according to internation Guidelines: mPAP ≥ 25 mmHg along with a PAWP ≤15 mmHg during right heart catheterization at the time of Initial diagnosis * PH class 1 (PAH) or 4 (CTEPH) * Stable condition, on the same medication for \> 4 weeks * Patient live permanently at an altitude \< 1000m asl. Exclusion Criteria: * Resting PaO2 ≤7.3 kPA corresponding to the requirement of long-term oxygen therapy \> 16hour daily (nocturnal oxygen therapy alone is allowed) * Severe daytime hypercapnia (pCO2 \> 6.5 kPa) * Susceptibility to high altitude related diseases (AMS, HAPE, etc.) based on previous experienced discomfort at altitudes. * Exposure to an altitude \>1500m for ≥3 nights during the last 4 weeks before the study participation * Residence \> 1000m above sea level * Inability to follow the procedures of the study, e.g. due to language problems, psychological disorders, neurological or orthopedic problems with Walking disability * Women who are pregnant or breast feeding **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Zurich **Country:** Switzerland **Facility:** Respiratory Clinic, University Hospital of Zurich **Zip:** 8091 #### Overall Officials **Official 1:** **Affiliation:** University of Zurich **Name:** Silvia Ulrich, Prof. **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014652 - Term: Vascular Diseases - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000008171 - Term: Lung Diseases - ID: D000012140 - Term: Respiratory Tract 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: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M10024 - Name: Hypertension - Relevance: HIGH - As Found: Hypertension - ID: M4185 - Name: Hypoxia - Relevance: LOW - As Found: Unknown - ID: M10027 - Name: Hypertension, Pulmonary - Relevance: HIGH - As Found: Pulmonary Hypertension - ID: M17400 - Name: Vascular 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: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000006976 - Term: Hypertension, Pulmonary - ID: D000006973 - Term: Hypertension ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05208879 **Acronym:** CaCo **Brief Title:** CArotenoid in hypoChOlesterolemia **Official Title:** Characterization of the Density of Macular Pigment in Patients With Primary Intestinal Hypocholesterolemia and Its Relation to Their Carotenoid and Anti-oxidant Status. #### Organization Study ID Info **ID:** 69HCL21_0720 #### Organization **Class:** OTHER **Full Name:** Hospices Civils de Lyon ### Status Module #### Completion Date **Date:** 2026-06-30 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-09-21 **Type:** ACTUAL **Last Update Submit Date:** 2023-09-20 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2026-06-30 **Type:** ESTIMATED #### Start Date **Date:** 2022-06-30 **Type:** ACTUAL **Status Verified Date:** 2023-09 #### Study First Post Date **Date:** 2022-01-26 **Type:** ACTUAL **Study First Submit Date:** 2022-01-12 **Study First Submit QC Date:** 2022-01-12 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Hospices Civils de Lyon #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Hypobetalipoproteinemias (HBL) represent a heterogeneous group of disorders characterized by reduced plasma levels of plasmatic lipids (mainly triglycerides, total cholesterol (TC), LDL-cholesterol (LDL-C), and apolipoprotein B (apoB)) below the 5th percentile of the general population adjusted for age, gender. HBL may be attributed to inherited disorders caused by mutations in several known genes. Intestinal recessive HBL includes abetalipoproteinemia (ABL) (OMIM 200100) and Chylomicron Retention Disease (CMRD) (OMIM 246700) - also called Anderson's disease. Those two recessives form of HBL are the ones considered in this study. ABL is due to mutations in the Microsomal Triglyceride Transfer Protein (MTTP) gene which is required for the assembly and secretion of apoB-containing lipoproteins: Low-Density Lipoprotein (LDL) and chylomicrons (CM) in both liver and intestine. Similarly, CMRD is due to mutations in the Sar1b gene encoding the Sar1b protein involved in the control of the intracellular trafficking of CMs in COPII-coated vesicles. Due to a defect in Apolipoprotein B-containing lipoproteins these diseases are characterized by dietary lipids and fat-soluble vitamins (A, D, E, K) malabsorption inducing digestive and growth disorders from birth. In parallel, neurological manifestations may appear, mainly as a consequence of vitamin E and A deficiencies. Ophthalmological disorders are inconstant, with many patients being asymptomatic until adulthood. Loss of night or color vision are the first symptoms associated with retinal degeneration. Without treatment with high doses of vitamins, retinal degeneration can lead to blindness. The exact biological mechanism still remains unknown. Indeed, cases described in the scientific literature demonstrate that early treatment with high doses of vitamin E and A can stop or prevent neurological complications in the vast majority of patients; however, ophthalmic complications have a more versatile response. Thus, despite early vitamin supplementation, several cases of adolescent or adult patients with vision impairment in the form of retinitis pigmentosa have been reported. This so-called secondary retinitis pigmentosa is characterized by a progressive loss of photoreceptors and a dysfunction of the pigmentary epithelium resulting in a progressive and gradual loss of vision, usually leading to blindness. Interestingly, primary (i.e., genetic) retinitis pigmentosa are characterized by "macula lutea" atrophy composed of two lipophilic molecules from the carotenoid xanthophyll family lutein and zeaxanthin, also known as macular pigments. Moreover, preliminary data seem to show that the patients considered for this study, present decreased plasmatic carotene concentrations as well as plasmatic vitamin E concentrations largely lower than the threshold of normality. Thus, even if early treatment seems to prevent major ophthalmic complications, it does not provide total ophthalmic protection, which suggests the involvement of other factors among which carotenoids could occupy a prominent place given their essential role in maintaining the integrity of the macula. ### Conditions Module **Conditions:** - Primary Intestinal Hypocholesterolemia - Abetalipoproteinemia - Chylomicron Retention Disease **Keywords:** - Abetalipoproteinemia, - Chylomicron Retention Disease - Hypocholesterolemia - Macula Pigment - Retinitis Pigmentosa - Carotenoids ### Design Module #### Design Info **Observational Model:** CASE_CONTROL **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 40 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients with genetically proven familial hypocholesterolemia, who will accept to participate in this study and have been treated for this pathology since 1990 in the Department of Gastroenterology and Paediatric Nutrition (Pr Peretti) and continue their follow-up into adulthood at the GHE (Groupement Hospitalier Est) in Lyon in the endocrinology-nutrition service (Pr Moulin). They may be girl/woman or boy/man over 6 years of age and over 12 kg at the time of inclusion (age required for cooperation on macular pigment measurement), agreeing to participate in the study with clear and informed consent. These patients are covered by social security. **Intervention Names:** - Biological: Characterization of carotenoid status and plasma levels of oxidative stress markers in patients (case group only) **Label:** Case group. #### Arm Group 2 **Description:** The control group consists of children over 6 years old or adult patients, followed routinely in the ophthalmology department of the Edouard Herriot Hospital, Lyon (Pr Kodjikian) not suffering from genetic hypocholesterolemia and requiring a fundus examination as part of the usual follow-up of their ocular pathology, if this pathology does not interfere with the macular pigment density. An additional measurement of the macular pigment density will be made during this examination. The control group is only needed for the macular pigment analysis. No control group is considered for the characterization of plasma lutein and zeaxanthin deficiency and for the analysis of oxidative stress, so there will be no additional blood sampling for control patients. **Intervention Names:** - Other: Characterization, evaluation and comparison of macular pigment density **Label:** Control group. ### Interventions #### Intervention 1 **Arm Group Labels:** - Case group. **Description:** Carotenoid status will be determined by measuring plasma and erythrocyte concentrations of lutein and zeaxanthin obtained during an annual blood draw. These molecules will be analyzed by high performance liquid chromatography. The oxidative stress markers will be measured, in the blood sample already collected, thanks to specific assay kits: Erythrocyte reduced glutathione, Superoxide dismutase (SOD), Glutathione peroxidase (GPx), Plasma and erythrocyte malondialdehyde, Plasma vitamin C, Plasma oxidized cholesterol, F2-isoprostanes. This study does not result in any change in patient management, but requires the collection of an additional volume of blood (14 ml of study-specific blood) during the annual blood draw performed as part of routine patient follow-up. **Name:** Characterization of carotenoid status and plasma levels of oxidative stress markers in patients (case group only) **Type:** BIOLOGICAL #### Intervention 2 **Arm Group Labels:** - Control group. **Description:** The macular pigment optical density (OD) will be determined by an additional photograph during the fundus usually performed for annual follow-up through two-wavelength autofluorescence imaging. Indeed, the measurement of the optical density consists of an additional post-examination analysis of additional retinal images obtained during the fundus performed for the patients' ophthalmologic follow-up. **Name:** Characterization, evaluation and comparison of macular pigment density **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** The macular pigment optical density (OD) will be determined by an additional photograph during the fundus usually performed for annual follow-up through two-wavelength autofluorescence imaging. This imaging method provides a profile of autofluorescence distribution calculated using a gray intensity scale in the center of the macula and along the horizontal and vertical meridians. The optical density of the macular pigment is then expressed as the logarithm of the ratio of the peri-oval/foveal excitation spectra symbolized by the acronym "DU" ("Density Unit"). Indeed, in autofluorescence, the lipofuscin chromophores are generally excited with two wavelengths: a first one which is located in the blue spectral region where the absorption band of the lipofuscin overlaps that of the macular pigments, and a second one which is always located in the absorption region of the lipofuscin but outside the absorption range of the macular pigments. Thus, excitation by the first (green) light causes **Measure:** The principal outcome measure is the macular pigment optical density (OD) **Time Frame:** The optical density measurement will be performed, in addition to the usual patient follow-up tests, during a single visit to the ophthalmology department on a date chosen by the patient. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Genetically proven family hypocholesterolemia patients, * Patients systematically monitored in the Gastroenterology and Paediatric Nutrition department of the Women's Hospital Mother Child of Lyon or in the adult endocrinology department of the GHE (Louis Pradel Hospital), * Girl/woman or boy/man over 6 years and over 12 kg at the time of inclusion (age required for cooperation on macular pigment measurement), * No objection from the patient or their parents/legal tutors in the case of a minor patient, * Patient covered by social security. Exclusion Criteria: * Allergy to local anesthetics (especially xylocaine) * Mydriatic allergy * Person participating in another research with an exclusion period still in progress at pre-inclusion * Person subject to a safeguard measure. **Maximum Age:** 50 Years **Minimum Age:** 6 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - CHILD - ADULT **Study Population:** This study relates to adults or children over 6 years of age (cooperation necessary to perform the fundus) with primary intestinal hypocholesterolemia. The control population comprising major control patients under the age of 50 (limit the risk of age-related macular degeneration - AMD) or under age over 6 (cooperation necessary to perform OF) routinely monitored in the service of ophthalmology requiring a fundus examination as part of their usual follow-up. ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Peretti Noël, Pr **Phone:** +33 472357050 **Role:** CONTACT #### Locations **Location 1:** **City:** Bron **Contacts:** ***Contact 1:*** - **Name:** PERETTI Noel, Pr - **Role:** CONTACT ***Contact 2:*** - **Name:** PERETTI Noel - **Role:** PRINCIPAL_INVESTIGATOR **Country:** France **Facility:** UF nutrition pédiatrique, Service hépatologie, gastroentérologie et nutrition pédiatrique Hôpital Femme Mère Enfant de Lyon (GHE-HFME) **Status:** RECRUITING **Zip:** 69500 **Location 2:** **City:** Bron **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Moulin Philippe, Pr - **Phone:** +33 472681304 - **Role:** CONTACT **Country:** France **Facility:** Fédération d'endocrinologie, maladies métaboliques, diabète et nutrition Hôpital cardiovasculaire et pneumologique Louis Pradel **Status:** NOT_YET_RECRUITING **Zip:** 69677 **Location 3:** **City:** Bron **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Peretti Noël, Pr - **Phone:** +33 472357050 - **Role:** CONTACT **Country:** France **Facility:** UF nutrition pédiatrique, Service hépatologie, gastroentérologie et nutrition pédiatrique Hôpital Femme Mère Enfant de Lyon (GHE-HFME) **Status:** RECRUITING **Zip:** 69677 #### Overall Officials **Official 1:** **Affiliation:** Hospices Civils de Lyon **Name:** Peretti Noël, Pr **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000006995 - Term: Hypobetalipoproteinemias - ID: D000007009 - Term: Hypolipoproteinemias - ID: D000008052 - Term: Lipid Metabolism, Inborn Errors - ID: D000008661 - Term: Metabolism, Inborn Errors - ID: D000030342 - Term: Genetic Diseases, Inborn - ID: D000050171 - Term: Dyslipidemias - ID: D000052439 - Term: Lipid Metabolism Disorders - ID: D000008659 - Term: Metabolic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC11 - Name: Eye Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M15008 - Name: Retinitis - Relevance: LOW - As Found: Unknown - ID: M15009 - Name: Retinitis Pigmentosa - Relevance: LOW - As Found: Unknown - ID: M11 - Name: Abetalipoproteinemia - Relevance: HIGH - As Found: Abetalipoproteinemia - ID: M10045 - Name: Hypobetalipoproteinemias - Relevance: LOW - As Found: Unknown - ID: M10059 - Name: Hypolipoproteinemias - Relevance: LOW - As Found: Unknown - ID: M11641 - Name: Metabolism, Inborn Errors - Relevance: LOW - As Found: Unknown - ID: M11054 - Name: Lipid Metabolism, Inborn Errors - Relevance: LOW - As Found: Unknown - ID: M23686 - Name: Genetic Diseases, Inborn - 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 - ID: T4945 - Name: Retinitis Pigmentosa - Relevance: LOW - As Found: Unknown - ID: T71 - Name: Abetalipoproteinemia - Relevance: HIGH - As Found: Abetalipoproteinemia - ID: T1320 - Name: Chylomicron Retention Disease - Relevance: HIGH - As Found: Chylomicron Retention Disease - ID: T2225 - Name: Familial Hypobetalipoproteinemia - Relevance: LOW - As Found: Unknown - ID: T2942 - Name: Hypolipoproteinemia - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000000012 - Term: Abetalipoproteinemia ### Intervention Browse Module - Ancestors - ID: D000000975 - Term: Antioxidants - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000020011 - Term: Protective Agents - ID: D000045505 - Term: Physiological Effects of Drugs ### Intervention Browse Module - Browse Branches - Abbrev: Micro - Name: Micronutrients - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Vi - Name: Vitamins - Abbrev: Ot - Name: Other Dietary Supplements ### Intervention Browse Module - Browse Leaves - ID: M4513 - Name: Ascorbic Acid - Relevance: LOW - As Found: Unknown - ID: M17558 - Name: Vitamins - Relevance: LOW - As Found: Unknown - ID: M4292 - Name: Antioxidants - Relevance: LOW - As Found: Unknown - ID: M16264 - Name: Superoxide Dismutase - Relevance: LOW - As Found: Unknown - ID: M5592 - Name: Carotenoids - Relevance: HIGH - As Found: Mupirocin - ID: M21869 - Name: Protective Agents - Relevance: LOW - As Found: Unknown - ID: T477 - Name: Vitamin C - Relevance: LOW - As Found: Unknown - ID: T437 - Name: Ascorbic Acid - Relevance: LOW - As Found: Unknown - ID: T407 - Name: Lutein - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000002338 - Term: Carotenoids ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02208479 **Brief Title:** Cardiac Surgery and Diaphragm Function **Official Title:** Diaphragmatic Dysfunction in Cardiac Surgery: Observational Study #### Organization Study ID Info **ID:** 2014-13 #### Organization **Class:** OTHER **Full Name:** University Hospital, Grenoble ### Status Module #### Completion Date **Date:** 2014-11 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2015-09-23 **Type:** ESTIMATED **Last Update Submit Date:** 2015-09-22 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2014-11 **Type:** ACTUAL #### Start Date **Date:** 2014-06 **Status Verified Date:** 2015-09 #### Study First Post Date **Date:** 2014-08-05 **Type:** ESTIMATED **Study First Submit Date:** 2014-08-01 **Study First Submit QC Date:** 2014-08-04 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University Hospital, Grenoble #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Diaphragm dysfunction is common after cardiac surgery and may delay weaning from mechanical ventilation and cause respiratory distress. The investigators' main objective is to determine the incidence of diaphragm dysfunction ( using the non-invasive ultrasonic method by calculating the inspiratory diaphragmatic thickening fraction) in a selected population of cardiac surgery patients during weaning from mechanical ventilation. The second endpoints are to determine the associated risk factors to post-operative diaphragm weakness and the consequence on the patient outcome. ### Conditions Module **Conditions:** - Cardiac Surgery **Keywords:** - cardiac surgery - diaphragm dysfunction - mechanical ventilation weaning - respiratory paralysis - muscle weakness - cardiac bypass - valvular surgery - respiratory insufficiency ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 66 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Other: Non invasive ultrasound measurement of the diaphragm thickness during breathing **Label:** cardiac surgery ### Interventions #### Intervention 1 **Arm Group Labels:** - cardiac surgery **Name:** Non invasive ultrasound measurement of the diaphragm thickness during breathing **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Measure:** Diaphragm inspiratory thickening fraction **Time Frame:** At the begining of the spontaneous breathing trial (around 4hours after surgery) #### Secondary Outcomes **Measure:** Diaphragm dysfunction before surgery **Time Frame:** the day before surgery **Measure:** Severity score (Euroscore) **Time Frame:** the day before surgery **Measure:** extracorporeal circulation duration **Time Frame:** peroperative **Measure:** left ventricular ejection fraction **Time Frame:** the day before surgery ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * 18 years old and older * planned surgery * ready for weaning from mechanical ventilation Exclusion Criteria: * protected patient * patient refusal **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** patient admitted in Intensive Care Unit (ICU) after planned cardiac surgery ### Contacts Locations Module #### Locations **Location 1:** **City:** Grenoble **Country:** France **Facility:** Unité de Réanimation Cardio-vasculaire et Thoracique **Zip:** 38043 ### References Module #### References **Citation:** DiNino E, Gartman EJ, Sethi JM, McCool FD. Diaphragm ultrasound as a predictor of successful extubation from mechanical ventilation. Thorax. 2014 May;69(5):423-7. doi: 10.1136/thoraxjnl-2013-204111. Epub 2013 Dec 23. **PMID:** 24365607 **Citation:** McCool FD, Tzelepis GE. Dysfunction of the diaphragm. N Engl J Med. 2012 Mar 8;366(10):932-42. doi: 10.1056/NEJMra1007236. No abstract available. Erratum In: N Engl J Med. 2012 May 31;366(22):2138. **PMID:** 22397655 **Citation:** Jaber S, Petrof BJ, Jung B, Chanques G, Berthet JP, Rabuel C, Bouyabrine H, Courouble P, Koechlin-Ramonatxo C, Sebbane M, Similowski T, Scheuermann V, Mebazaa A, Capdevila X, Mornet D, Mercier J, Lacampagne A, Philips A, Matecki S. Rapidly progressive diaphragmatic weakness and injury during mechanical ventilation in humans. Am J Respir Crit Care Med. 2011 Feb 1;183(3):364-71. doi: 10.1164/rccm.201004-0670OC. Epub 2010 Sep 2. **PMID:** 20813887 **Citation:** Moury PH, Cuisinier A, Durand M, Bosson JL, Chavanon O, Payen JF, Jaber S, Albaladejo P. Diaphragm thickening in cardiac surgery: a perioperative prospective ultrasound study. Ann Intensive Care. 2019 Apr 24;9(1):50. doi: 10.1186/s13613-019-0521-z. **PMID:** 31016412 ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M14968 - Name: Respiratory Insufficiency - Relevance: LOW - As Found: Unknown - ID: M20944 - Name: Muscle Weakness - Relevance: LOW - As Found: Unknown - ID: M13204 - Name: Paresis - Relevance: LOW - As Found: Unknown - ID: M4554 - Name: Asthenia - Relevance: LOW - As Found: Unknown - ID: M13157 - Name: Paralysis - Relevance: LOW - As Found: Unknown - ID: M14970 - Name: Respiratory Paralysis - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04757779 **Brief Title:** A Single-arm, Phase Ⅱ Clinical Trial of Anlotinib Hydrochloride Combined With Irinotecan or Docetaxel for Second Line Treatment of Nonsensitive Relapsed Small-cell Lung Cancer **Official Title:** A Single-arm, Phase Ⅱ Clinical Trial of Anlotinib Hydrochloride Combined With Irinotecan or Docetaxel for Second Line Treatment of Nonsensitive Relapsed Small-cell Lung Cancer #### Organization Study ID Info **ID:** HZCH-2019-004 #### Organization **Class:** OTHER **Full Name:** First People's Hospital of Hangzhou ### Status Module #### Completion Date **Date:** 2024-12-30 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2021-02-17 **Type:** ACTUAL **Last Update Submit Date:** 2021-02-14 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2022-12-30 **Type:** ESTIMATED #### Start Date **Date:** 2019-12-30 **Type:** ACTUAL **Status Verified Date:** 2021-02 #### Study First Post Date **Date:** 2021-02-17 **Type:** ACTUAL **Study First Submit Date:** 2021-02-14 **Study First Submit QC Date:** 2021-02-14 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** First People's Hospital of Hangzhou #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Is US Export:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Anlotinib hydrochloride is a multi-target antiangiogenic drug. It was recommended by Chinese Society of Clinical Oncology(CSCO) guideline as a third-line treatment for advanced small-cell lung cancer. This study intends to assess the efficacy and safety of anlotinib hydrochloride combined with irinotecan or docetaxel for second line treatment of nonsensitive relapsed small-cell lung cancer. ### Conditions Module **Conditions:** - Relapsed Small Cell Lung Cancer - Anlotinib ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP **Intervention Model Description:** From the start of the study, the subjects are orally administered with anlotinib 12mg on empty stomach. Subjects need to take anlotinib 2 weeks continuously and stop for 1 week(every 3 weeks is a cycle). On Day1 and Day8, subjects are required to inject irinotecan (65mg/m2) or docetaxel(60mg/m2) of a cycle,until disease progression or intolerable toxicity, for 4 cycles at most. ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 40 **Type:** ESTIMATED **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** From the start of the study, the subjects are orally administered with anlotinib 12mg on empty stomach. Subjects need to take anlotinib 2 weeks continuously and stop for 1 week(every 3 weeks is a cycle). On Day1 and Day8, subjects are required to inject irinotecan(65mg/m2) or docetaxel(60mg/m2) of a cycle,until disease progression or intolerable toxicity, for 4 cycles at most. **Intervention Names:** - Drug: anlotinib hydrochloride combined with irinotecan or docetaxel **Label:** anlotinib hydrochloride combined with irinotecan or docetaxel **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - anlotinib hydrochloride combined with irinotecan or docetaxel **Description:** From the start of the study, the subjects are orally administered with anlotinib 12mg on empty stomach.Subjects need to take anlotinib 2 weeks continuously and stop for 1 week(every 3 weeks is a cycle),the dose of anlotinib can be adjusted as 12mg,10mg or 8mg according to adverse effects.On Day1 and Day8, subjects are required to inject irinotecan (65mg/m2)or docetaxel (60mg/m2) of a cycle,until disease progression or intolerable toxicity, for 4 cycles at most. **Name:** anlotinib hydrochloride combined with irinotecan or docetaxel **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** the proportion of patients assessed with complete response and partial response **Measure:** objective response rate（ORR） **Time Frame:** 2 years #### Secondary Outcomes **Description:** the time from date of randomization to disease progression or death **Measure:** progression-free survival time（PFS） **Time Frame:** 2 years **Description:** the proportion of patients assessed with complete response，partial response and stable disease **Measure:** disease control rate（DCR） **Time Frame:** 2 years **Description:** the time from date of randomization to death from any cause **Measure:** overall survival（OS） **Time Frame:** 2 years **Description:** Quality of Life assessed by The European Organization for Research and Treatment of Cancer Quality of Life Questionnaire core 30(EORTC QLQ-C30) **Measure:** quality of life（QoF）assessed by EORTC QLQ-C30 **Time Frame:** 2 years **Description:** Quality of Life assessed by The European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Lung Cancer (EORTC QLQ LC-13) **Measure:** quality of life（QoF）assessed by EORTC QLQ LC-13 **Time Frame:** 2 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. The subject volunteered to participate in the study with informed consent signed. 2. Histologically or pathologically confirmed small-cell lung cancer (whether limited or advanced stage). 3. Have received at least first-line platinum-based chemotherapy for small-cell lung cancer and comfirmed disease relapse with imaging material. Disease progression during previous chemotherapy or less than 6 month after last chemotherapy. 4. Relapsed advanced small-cell lung cancer patients with symptom-controlled brain metastasis or leptomeningeal metastasis (subjects with symptomatic brain metastasis are allowed to receive radiotherapy, whether brain lesions can be deemed as target lesions is decided by investigators.); or patients with newly- discovered brain metastasis or leptomeningeal metastasis diagnosed by CT/MRI. Symptomatic or asymptomatic serosal cavity effusion (pleural effusion, ascites, pericardial effusion, local therapy is allowed). Radiotherapy for symptomatic bone metastasis or elsewhere is allowed as long as response evaluation is not affected. 5. Age:18-75 years old. 6. Eastern Cooperative Oncology Group (ECOG) performance status(PS) score ≤ 2. 7. Survival is expected to be ≥ 6 months. 8. At least one non-irradiated target lesion confirmed by CT/MRI scan less than 28 days before first dose of the study drug. 9. Male and women must use contraception within first dose to 24 weeks after last dose. 10. Major organ functions meet the following criteria within 7 days prior to treatment: blood routine examination and coagulation function (no blood transfusion within 14 days): hemoglobin≥90g/L; Absolute Neutrophil Count(ANC)≥1.5×109/L; Platelet (PLT)≥80×109/L; International normalized ratio(INR)≤1.5，Activated partial thromboplastin time(APTT)≤1.5 × upper limit of normal value(ULN); biochemical test standards: Total bilirubin(TBIL)≤1.5× ULN; ALT/AST≤2.5×ULN without liver metastasis, ALT/AST≤5×ULN with liver metastasis; Creatinine ≤1.25× ULN or endogenous creatinine clearance rate(Ccr)\>45ml/min. Exclusion Criteria: 1. Non-small-cell lung cancer (including a mixture of small-cell and non-small cell lung cancer). 2. Patients with small-cell lung cancer who relapsed more than 6 months after first- line treatment. 3. Medical imaging shows that the distance between the tumor and large vessels is less than 5mm; or lesions invade major blood vessels; or patients who are at risk of severe bleeding during the following treatment which is determined by investigators. 4. Medical imaging shows significant pulmonary cavity or necrotic tumor. 5. Uncontrolled hypertension (systolic blood pressure≥140mmHg or diastolic blood pressure≥90mmHg, even with optimal medication treatment). 6. Subjects with ≥grade Ⅱmyocardial ischemia or myocardial infarction, uncontrolled arrhythmia (include QT interval≥450ms for males, ≥470ms for females). 7. Heart function of NYHA grade Ⅲ-Ⅳ or left ventricle ejection fraction(LVEF)\<50% confirmed by echocardiography. 8. Coagulant function abnormality (INR\>1.5 or PT\>ULN+4 seconds or APTT\> 1.5ULN), with a bleeding tendency or patients is receiving thrombolytic or anticoagulant therapy. 9. For subjects who are using an anticoagulant or vitamin K antagonist (e.g. warfarin or heparin or other similar drugs), low dose heparin (6000-12000U daily for an adult) or aspirin (≤100mg daily) is allowed for preventive purposes when INR≤1.5. 10. Symptoms or propensity to bleed within 3 months prior to screening (include gastrointestinal hemorrhage, ulcerative gastric bleeding, fecal occult blood 2+ or above, vasculitis). 11. Arterial/venous thrombosis within 12 months prior to screening, e.g. cerebrovascular accident (include temporary ischemic attack, cerebral hemorrhage, cerebral infarction), deep venous thrombosis, pulmonary embolism. 12. Inherited or acquired bleeding and thrombus propensity (hemophilia, coagulation dysfunction, thrombocytopenia and hypersplenism). 13. Unhealed wound or fracture for a long time. 14. Major surgical operation or severe traumatic injury, bone fracture or ulcer within 4 weeks prior to screening, which affect drug absorption e.g. inability to swallow, chronic diarrhea and intestinal obstruction. 15. Abdominal fistula, gastrointestinal perforation, intraperitoneal abscess within 6 months prior to screening; routine urine test indicate urine protein≥++ or 24- hours proteinuria≥1.0g. 16. History of psychotropic drug abuse and cannot abstain from it or with mental disorders. 17. Participation in other clinical trials of anti-tumor drugs within 4 weeks prior to screening. 18. Previous or concurrent with other types of uncured malignancies, with the exception of cured basal cell carcinoma of the skin, carcinoma in situ of cervix and superficial bladder cancer. 19. Pregnant or lactating women, fertile patients who are unwilling or unable to use effective contraceptives. **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:** Bing Xia, MD **Phone:** 86 571 56006388 **Role:** CONTACT #### Locations **Location 1:** **City:** Hangzhou **Contacts:** ***Contact 1:*** - **Name:** Bing Xia, MD - **Role:** CONTACT **Country:** China **Facility:** Affiliated Hangzhou Cancer Hospital, Zhejiang University School of Medicine **State:** Zhejiang **Status:** RECRUITING **Zip:** 310002 ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012142 - Term: Respiratory Tract Neoplasms - ID: D000013899 - Term: Thoracic Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000008171 - Term: Lung Diseases - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000002283 - Term: Carcinoma, Bronchogenic - ID: D000001984 - Term: Bronchial Neoplasms ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M11172 - Name: Lung Neoplasms - Relevance: HIGH - As Found: Lung Cancer - ID: M28323 - Name: Small Cell Lung Carcinoma - Relevance: HIGH - As Found: Small Cell Lung Cancer - ID: M5534 - Name: Carcinoma - Relevance: LOW - As Found: Unknown - ID: M14979 - Name: Respiratory Tract Neoplasms - Relevance: LOW - As Found: Unknown - ID: M16658 - Name: Thoracic Neoplasms - Relevance: LOW - As Found: Unknown - ID: M11168 - Name: Lung Diseases - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown - ID: M5540 - Name: Carcinoma, Bronchogenic - Relevance: LOW - As Found: Unknown - ID: M5260 - Name: Bronchial Neoplasms - Relevance: LOW - As Found: Unknown - ID: T5271 - Name: Small Cell Lung Cancer - Relevance: HIGH - As Found: Small Cell Lung Cancer ### Condition Browse Module - Meshes - ID: D000008175 - Term: Lung Neoplasms - ID: D000055752 - Term: Small Cell Lung Carcinoma ### Intervention Browse Module - Ancestors - ID: D000000970 - Term: Antineoplastic Agents - ID: D000050257 - Term: Tubulin Modulators - ID: D000050256 - Term: Antimitotic Agents - ID: D000050258 - Term: Mitosis Modulators - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000059004 - Term: Topoisomerase I Inhibitors - ID: D000059003 - Term: Topoisomerase Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M1668 - Name: Docetaxel - Relevance: HIGH - As Found: Physical - ID: M1671 - Name: Irinotecan - Relevance: HIGH - As Found: Infection - ID: M26197 - Name: Tubulin Modulators - Relevance: LOW - As Found: Unknown - ID: M26196 - Name: Antimitotic Agents - Relevance: LOW - As Found: Unknown - ID: M29349 - Name: Topoisomerase I Inhibitors - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000077143 - Term: Docetaxel - ID: D000077146 - Term: Irinotecan ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05801679 **Brief Title:** Evaluating Outcomes in Cardiac Surgery Patients Who Receive Sugammadex vs. Placebo **Official Title:** A Prospective Randomized Blinded Controlled Trial Comparing Clinical Outcomes in Cardiac Surgical Patients Who Receive Sugammadex vs. Placebo #### Organization Study ID Info **ID:** EH23-005 #### Organization **Class:** OTHER **Full Name:** NorthShore University HealthSystem ### Status Module #### Completion Date **Date:** 2025-06 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-04-12 **Type:** ACTUAL **Last Update Submit Date:** 2024-04-11 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2025-06 **Type:** ESTIMATED #### Start Date **Date:** 2023-07-03 **Type:** ACTUAL **Status Verified Date:** 2024-04 #### Study First Post Date **Date:** 2023-04-06 **Type:** ACTUAL **Study First Submit Date:** 2023-01-30 **Study First Submit QC Date:** 2023-04-05 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** NorthShore University HealthSystem #### Responsible Party **Investigator Affiliation:** NorthShore University HealthSystem **Investigator Full Name:** Steven Greenberg **Investigator Title:** Jeffery S. Vender Endowed Chair of Anesthesiology Research and Education **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:** This is a prospective randomized blinded controlled trial that will enroll 175 subjects undergoing cardiopulmonary bypass at NorthShore University HealthSystem. The purpose of this study is to compare clinical outcomes in elective and urgent cardiac surgical patients at NorthShore University HealthSystem when receiving sugammadex, a common neuromuscular blockade reversal drug given after surgery and before the breathing tube is removed vs. those patients who do not receive sugammadex (placebo) group. The Investigators will compare the following outcomes in both the sugammadex and placebo groups during patients hospital stay: # of patients who have the breathing tube removed within 6 hour of the end of surgery, time it takes to remove the breathing tube after surgery, ICU and hospital length of stay, cost of the ICU stay, time to achieve a train of four ratio of \> or equal to 0.9, whether patients develop pneumonia or not, whether they require the breathing tube to be replaced during their hospital stay and to compare the nursing perception of patients recovery within first 24 hours of their ICU stay. **Detailed Description:** At the conclusion of many cardiac surgical cases requiring cardiopulmonary bypass, patients are typically transferred to the intensive care unit (ICU) with the endotracheal tube remaining in the airway postoperatively without routine reversal of neuromuscular blockade (NMB). This blockade is typically metabolized by the liver/kidney and then patients are liberated from the ventilator in the ICU afterwards. The proposed reason for this strategy is to reduce the potential risk of rebleeding or arrhythmias due to a sympathetic response from patients. A survey among 495 cardiac anesthesiologists in the U.S. in 2002 suggested that only 9% of anesthesiologists routinely reverse NMB in these patients prior to extubation. However, the lack of reversal drug use among any surgical patient population could result in residual neuromuscular blockade, which is defined by a train of four ratio ≥0.9. Patients who do not meet this level of neuromuscular recovery are at risk for a number of adverse outcomes including hypoxemia, airway obstruction, impaired swallowing function, increased risk for aspiration, prolonged length of stay, postoperative respiratory complications, and need for reintubation. The data regarding residual neuromuscular blockade in cardiac surgical patients is limited. A prospective observational cohort of 50 cardiac surgical patients, suggested that 66% of patients had significant residual neuromuscular blockade within 1 hour postoperatively. Prolonged intubation can lead to unwanted adverse outcomes such as pneumonia. Our clinical practice at NorthShore University HealthSystem for cardiac surgical patients changed as it relates to managing neuromuscular blockade. Prior to 2019, the clinical care team (cardiac surgery, intensive care, nursing, and anesthesia) did not routinely discuss dosing or reversal of neuromuscular blockade during the ICU handoff of patients. Anesthesia professionals also did not routinely reverse neuromuscular blockade in post-cardiac surgical patients. In the latter half of 2019, the care team developed a multidisciplinary handoff checklist, which includes discussion regarding the last dose of NMB, and whether the patient was given reversal. The anesthesia professionals changed practice to meet or exceed the Society of Thoracic Surgeons (STS) early extubation national benchmark within 6 hours of the end of surgery. Therefore, the investigators hypothesize that by reversing cardiac surgery patients with sugammadex in the ICU, the investigators will be able to achieve the STS early extubation criteria more frequently and it will also result in reduced ICU, hospital length of stay and cost of ICU stay. The investigators also believe it will result in less reintubation and pneumonia. ### Conditions Module **Conditions:** - Surgery **Keywords:** - Sugammadex - Cardiac Surgical Patients - Anesthesia ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER **Primary Purpose:** OTHER #### Enrollment Info **Count:** 175 **Type:** ESTIMATED **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Single intravenous (IV) bolus of sugammadex at 2 mg/kg (Twitch count 2-4 of 4) or 4 mg/kg (twitch count less than 2 of 4). **Intervention Names:** - Drug: Sugammadex **Label:** Sugammadex **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Single intravenous (IV) bolus of Placebo at 2 mg/kg (Twitch count 2-4 of 4) or 4 mg/kg (twitch count less than 2 of 4). **Intervention Names:** - Other: Placebo **Label:** Placebo **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Sugammadex **Description:** Fifteen minutes after ICU arrival, subjects will be administered sugammadex (2 mg/kg assuming a twitch count of 2-4 of 4 or 4mg/kg assuming a twitch count less than 2 of 4) by the anesthesia provider. Five minutes after administration, a quantitative neuromuscular monitor (TetraGraph, Senzime, Uppsala, Sweden) will be applied and the TOF ratio will be recorded. **Name:** Sugammadex **Other Names:** - MK-8616 **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Placebo **Description:** Fifteen minutes after ICU arrival, subjects will be administered placebo (2 mg/kg assuming a twitch count of 2-4 of 4 or 4mg/kg assuming a twitch count less than 2 of 4) by the anesthesia provider. Five minutes after administration, a quantitative neuromuscular monitor (TetraGraph, Senzime, Uppsala, Sweden) will be applied and the TOF ratio will be recorded. **Name:** Placebo **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Will compare the difference in the number of subjects who receive sugammadex and meet the STS 6-hour extubation criteria from the end of surgery vs. those that do not receive neuromuscular blockade reversal. **Measure:** Society of Thoracic Surgery (STS) quality benchmark of early extubation criteria **Time Frame:** within 6 hours of end of surgery #### Secondary Outcomes **Description:** Time to first extubation from end of surgery in each group will be recorded. **Measure:** Time to first extubation **Time Frame:** Intraoperative (The time (in hours) it takes to remove the breathing tube immediately at the end of surgery.) **Description:** Time from administration of sugammadex vs. placebo to achieve a TOF ratio ≥0.9 prior to extubation will be recorded. **Measure:** Time from administration of sugammadex vs. placebo **Time Frame:** Within 6 hours of administration of sugammadex or placebo **Description:** ICU length of stay (hours) in each group will be recorded. **Measure:** ICU length of stay **Time Frame:** up to 168 hours (7days) **Description:** Hospital length of stay (days) in each group will be recorded. **Measure:** Hospital length of stay **Time Frame:** up to 7 days **Description:** The incidence of reintubation post-extubation in each group will be collected during the current hospital stay. **Measure:** Incidence of reintubation post-extubation **Time Frame:** up to 1 week **Description:** The incidence of post-extubation pneumonia in each group will be collected during the current hospital stay **Measure:** Incidence of post-extubation pneumonia **Time Frame:** up to 1 week **Description:** Cost of ICU stay in each group will be collected from hospital billing data. **Measure:** Cost of ICU Stay **Time Frame:** up to 1 week **Description:** The nursing perception questionnaire of cardiac surgical subjects' ICU quality of recovery within first 24 hours of ICU length of stay will be collected. (A scale from 1-5; 1=Very dissatisfied, 2=Somewhat dissatisfied, 3=Neutral, 4= Somewhat satisfied, 5= Very satisfied) **Measure:** Nursing perception questionnaire of cardiac surgical subjects' ICU quality of recovery **Time Frame:** Within first 24 hours of ICU length of stay ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Subject must be an elective or urgent cardiac surgical patient undergoing cardiopulmonary bypass at NorthShore University HealthSystem. 2. Male or female subject aged 21 to 90 years, at the time of consent. 3. Subject who can consent in English. 4. Subjects who are eligible for fast track extubation as defined by those patients who plan on being extubated within 24 hours of the end of surgery and optimally within the 6-hour STS benchmark time from end of surgery. Exclusion Criteria: 1. Subjects having emergency cardiac surgery. 2. Subjects who cannot consent in English. 3. Subjects who are not eligible to be extubated within 24 hours of the end of surgery. 4. Subjects with neuromuscular disorders. 5. Subjects on home oxygen. 6. Subjects who have known allergies or reactions to rocuronium or sugammadex. 7. Subjects with anticipated need for prolonged intubation by the clinical treating team. 8. Subjects with a history of opioid abuse. 9. Subjects on mechanical circulatory support. 10. Subjects who have end stage renal disease requiring dialysis. **Maximum Age:** 90 Years **Minimum Age:** 21 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Steven Greenberg, MD **Phone:** 847-570-2760 **Role:** CONTACT #### Locations **Location 1:** **City:** Evanston **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Steven Greenberg, MD - **Phone:** 847-570-2760 - **Role:** CONTACT **Country:** United States **Facility:** NorthShore University HealthSystem **State:** Illinois **Status:** RECRUITING **Zip:** 60201 #### Overall Officials **Official 1:** **Affiliation:** NorthShore University HealthSystem **Name:** Steven Greenberg, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Description:** We do not plan to make IPD available to other researchers. **IPD Sharing:** NO ### References Module #### References **Citation:** Chacon M, Markin NW. Early is Good, But is Immediate Better? Considerations in Fast-Track Extubation After Cardiac Surgery. J Cardiothorac Vasc Anesth. 2022 May;36(5):1265-1267. doi: 10.1053/j.jvca.2022.01.031. Epub 2022 Jan 25. No abstract available. **PMID:** 35193778 **Citation:** Kotfis K, Szylinska A, Listewnik M, Lechowicz K, Kosiorowska M, Drozdzal S, Brykczynski M, Rotter I, Zukowski M. Balancing intubation time with postoperative risk in cardiac surgery patients - a retrospective cohort analysis. Ther Clin Risk Manag. 2018 Nov 5;14:2203-2212. doi: 10.2147/TCRM.S182333. eCollection 2018. **PMID:** 30464493 **Citation:** Cove ME, Ying C, Taculod JM, Oon SE, Oh P, Kollengode R, MacLaren G, Tan CS. Multidisciplinary Extubation Protocol in Cardiac Surgical Patients Reduces Ventilation Time and Length of Stay in the Intensive Care Unit. Ann Thorac Surg. 2016 Jul;102(1):28-34. doi: 10.1016/j.athoracsur.2016.02.071. Epub 2016 May 4. **PMID:** 27154151 **Citation:** Goeddel LA, Hollander KN, Evans AS. Early Extubation After Cardiac Surgery: A Better Predictor of Outcome than Metric of Quality? J Cardiothorac Vasc Anesth. 2018 Apr;32(2):745-747. doi: 10.1053/j.jvca.2017.12.037. Epub 2018 Jan 2. No abstract available. **PMID:** 29395821 **Citation:** Murphy GS, Szokol JW, Vender JS, Marymont JH, Avram MJ. The use of neuromuscular blocking drugs in adult cardiac surgery: results of a national postal survey. Anesth Analg. 2002 Dec;95(6):1534-9, table of contents. doi: 10.1097/00000539-200212000-00012. **PMID:** 12456412 **Citation:** Murphy GS, Brull SJ. Residual neuromuscular block: lessons unlearned. Part I: definitions, incidence, and adverse physiologic effects of residual neuromuscular block. Anesth Analg. 2010 Jul;111(1):120-8. doi: 10.1213/ANE.0b013e3181da832d. Epub 2010 May 4. **PMID:** 20442260 **Citation:** Maybauer DM, Geldner G, Blobner M, Puhringer F, Hofmockel R, Rex C, Wulf HF, Eberhart L, Arndt C, Eikermann M. Incidence and duration of residual paralysis at the end of surgery after multiple administrations of cisatracurium and rocuronium. Anaesthesia. 2007 Jan;62(1):12-7. doi: 10.1111/j.1365-2044.2006.04862.x. **PMID:** 17156221 **Citation:** Roy M, Morissette N, Girard M, Robillard N, Beaulieu P. Postoperative awake paralysis in the intensive care unit after cardiac surgery due to residual neuromuscular blockade: a case report and prospective observational study. Can J Anaesth. 2016 Jun;63(6):725-30. doi: 10.1007/s12630-016-0606-1. Epub 2016 Mar 2. **PMID:** 26936365 **Citation:** Carron M, Zarantonello F, Tellaroli P, Ori C. Efficacy and safety of sugammadex compared to neostigmine for reversal of neuromuscular blockade: a meta-analysis of randomized controlled trials. J Clin Anesth. 2016 Dec;35:1-12. doi: 10.1016/j.jclinane.2016.06.018. Epub 2016 Aug 4. **PMID:** 27871504 **Citation:** Ebert TJ, Cumming CE, Roberts CJ, Anglin MF, Gandhi S, Anderson CJ, Stekiel TA, Gliniecki R, Dugan SM, Abdelrahim MT, Klinewski VB, Sherman K. Characterizing the Heart Rate Effects From Administration of Sugammadex to Reverse Neuromuscular Blockade: An Observational Study in Patients. Anesth Analg. 2022 Oct 1;135(4):807-814. doi: 10.1213/ANE.0000000000006131. Epub 2022 Sep 15. **PMID:** 35759402 **Citation:** Fischer MO, Brotons F, Briant AR, Suehiro K, Gozdzik W, Sponholz C, Kirkeby-Garstad I, Joosten A, Nigro Neto C, Kunstyr J, Parienti JJ, Abou-Arab O, Ouattara A; VENICE study group. Postoperative Pulmonary Complications After Cardiac Surgery: The VENICE International Cohort Study. J Cardiothorac Vasc Anesth. 2022 Aug;36(8 Pt A):2344-2351. doi: 10.1053/j.jvca.2021.12.024. Epub 2021 Dec 25. **PMID:** 35094928 **Citation:** Li L, Jiang Y, Zhang W. Sugammadex for Fast-Track Surgery in Children Undergoing Cardiac Surgery: A Randomized Controlled Study. J Cardiothorac Vasc Anesth. 2021 May;35(5):1388-1392. doi: 10.1053/j.jvca.2020.08.069. Epub 2020 Sep 3. **PMID:** 32962936 **Citation:** Yan P, Wu X, Cai F, Chen Y, Huang Y, Li G, Lai K. Efficacy and safety of sugammadex in anesthesia of cardiac surgery: A retrospective study. J Clin Anesth. 2020 Oct;65:109845. doi: 10.1016/j.jclinane.2020.109845. Epub 2020 May 26. No abstract available. **PMID:** 32464476 ## Derived Section ### Intervention Browse Module - Browse Branches - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M4107 - Name: Anesthetics - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06055179 **Brief Title:** XCHT for Irinotecan-Induced Gut Toxicities (Randomized Controlled Trial) **Official Title:** Mechanistic and Pharmacokinetic Studies of Classical Chinese Formula Xiao Chai Hu Tang (XCHT) Against Irinotecan-Induced Gut Toxicities(Clinical Study Part:Randomized Controlled Trial) #### Organization Study ID Info **ID:** 2021KT1005-2 #### Organization **Class:** OTHER **Full Name:** Guangzhou University of Traditional Chinese Medicine #### Secondary ID Infos **Domain:** National Nature Science Fundation of China **ID:** 81961128028 **Type:** OTHER_GRANT ### Status Module #### Completion Date **Date:** 2025-02-01 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-03-22 **Type:** ACTUAL **Last Update Submit Date:** 2024-03-21 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-12-01 **Type:** ESTIMATED #### Start Date **Date:** 2024-03-08 **Type:** ACTUAL **Status Verified Date:** 2023-09 #### Study First Post Date **Date:** 2023-09-26 **Type:** ACTUAL **Study First Submit Date:** 2023-09-09 **Study First Submit QC Date:** 2023-09-24 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** University of Houston #### Lead Sponsor **Class:** OTHER **Name:** Guangzhou University of Traditional Chinese Medicine #### Responsible Party **Investigator Affiliation:** Guangzhou University of Traditional Chinese Medicine **Investigator Full Name:** Haibo Zhang **Investigator Title:** Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** Randomized double-blind placebo-controlled trial (RCT) study, to determine the impact of XCHT on irinotecan-induced severe delayed-onset diarrhea (SDOD), and to determine the feasibility of using plasma raloxifene-4'-glucuronide as a probe for intestinal UGT activity. **Detailed Description:** A total of 98 patients, who are planning to recieve at least 3 cycles of FOLFIRI/mXELIRI chemotherapy, will be randomly assigned, at a 1:1 ratio, to XCHT group or placebo group, using a central randomization system. Patients will be administered with XCHT/placebo (9 g, qd, po) for 5 days each cycle of chemotherapy for 3 cycles. The XCHT/placebo administration begins 3 days before chemotherapy in each cycle, that is the chemotherapy begins on the 4th day of XCHT/placebo administration. Plasma will be collected for pharmacokinetic testing (using raloxifene 60mg po as probe), on the day before chemotherapy, that is on the 3rd day of XCHT/placebo administration in each cycle. The purpose of this study includes 1) to determine the safety and efficacy of XCHT for prevention of irinotecan-induced diarrhea; 2) to determine the PK profile of SN-38, SN-38G, raloxifene, raloxifene-glucuronide, and XCHT components; and 3) to validate the use of raloxifene-4'G as a probe for irinotecan-induced diarrhea. ### Conditions Module **Conditions:** - Xiao Chai Hu Tang - Irinotecan-induced Diarrhea **Keywords:** - Malignant tumor - Irinotecan-induced diarrhea - Xiao Chai Hu Tang - Classical Chinese Formula - pharmacokinetic mechanism ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** QUADRUPLE **Masking Description:** Patients will be randomly assigned to XCHT/placebo groups, at a 1:1 ratio, using a central randomization system provided by the TCM clinical study methodology group of Guangdong Provincial Hospital of Chinese Medicine. This group is also responsible for blinding, including keeping the blinding codes, supervision, and quality control of blinding, by allocating drug codes for each patient. Investigators will dispense the drug according to the drug codes. The investigators, the study subjects, the care givers, and the outcome assessors will be blinded to the assigned group of subjects. **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 98 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients will be administered with XCHT (9 g, qd, po) for 5 days each cycle of FOLFIRI/mXELIRI chemotherapy for 3 cycles. The XCHT administration begins 3 days before chemotherapy in each cycle, that is the chemotherapy begins on the 4th day of XCHT administration. Plasma will be collected for pharmacokinetic testing (using raloxifene 60mg po as probe), on the day before chemotherapy, that is on the 3rd day of XCHT administration in each cycle. **Intervention Names:** - Drug: Xiao Chai Hu Tang (XCHT) - Drug: FOLFIRI/mXELIRI regimen - Other: Raloxifene **Label:** XCHT group **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Patients will be administered with placebo (9 g, qd, po) for 5 days each cycle of FOLFIRI/mXELIRI chemotherapy for 3 cycles. The placebo administration begins 3 days before chemotherapy in each cycle, that is the chemotherapy begins on the 4th day of placebo administration. Plasma will be collected for pharmacokinetic testing (using raloxifene 60mg po as probe), on the day before chemotherapy, that is on the 3rd day of placebo administration in each cycle. **Intervention Names:** - Drug: Placebo - Drug: FOLFIRI/mXELIRI regimen - Other: Raloxifene **Label:** Placebo group **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - XCHT group **Description:** XCHT 9g, po qd, 3 days before each cycle of chemotherapy for 5 days, for 3 cycles of chemotherapy. **Name:** Xiao Chai Hu Tang (XCHT) **Other Names:** - Xiao Chai Hu Tang granules from Nin Jiom Medicine Manufactory (Hong Kong) Ltd **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Placebo group **Description:** Placebo 9g, po qd, 3 days before each cycle of chemotherapy for 5 days, for 3 cycles of chemotherapy. **Name:** Placebo **Other Names:** - Placebo provided by Nin Jiom Medicine Manufactory (Hong Kong) Ltd **Type:** DRUG #### Intervention 3 **Arm Group Labels:** - Placebo group - XCHT group **Description:** Patients will receive 3 cycles of FOLFIRI/mXELIRI chemotherapy. FOLFIRI regimen:irinotecan (180 mg/m2) intravenous (IV) for over 90 minutes on Day 1. Folinic acid (400 mg/m2) IV over 2 hours on Day 1. 5-fluorouracil (5-FU) IV bolus (400 mg/m2) over 5 minutes on Day 1, followed by 5-FU (2400 mg/m2) IV continuously for 46-48 hours. Every 2 weeks. mXELIRI regimen:irinotecan (200 mg/m2) intravenous (IV) for over 90 minutes on Day 1. Capecitabine (800 mg/m²) PO bid on day 1-14. Every 3 weeks. **Name:** FOLFIRI/mXELIRI regimen **Type:** DRUG #### Intervention 4 **Arm Group Labels:** - Placebo group - XCHT group **Description:** Raloxifene 60mg po, used as probe for pharmacokinetic testing, on Day 3 (the day before chemotherapy), for 3 cycles of chemotherapy. **Name:** Raloxifene **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** The diarrhea severity will be evaluated following standard criteria in NCI-CTC AE 5.0 Grade 2 is defined as Stool is increased by 4-6 times each day relative to baseline; discharge from stoma moderately increased. **Measure:** Incidence of diarrhea (grade ≥2) **Time Frame:** Through study completion, an average of 2 months #### Secondary Outcomes **Description:** The diarrhea severity will be evaluated following standard criteria in NCI-CTC AE 5.0 **Measure:** Incidence of diarrhea (grade ≥3) **Time Frame:** Through study completion, an average of 2 months **Description:** Other adverse reactions will be evaluated following standard criteria in NCI-CTC AE 5.0 **Measure:** Incidence of other chemo-related adverse effects **Time Frame:** Through study completion, an average of 2 months **Description:** Occult blood test for stool, reported as negative, weak positive, and positive. **Measure:** Occult blood test for stool **Time Frame:** Through study completion, an average of 2 months **Description:** Cmax ,if with complete data, in each cycle of Cmax for XCHT and their metabolites (14 compounds) and intestinal UGT enzyme probe (raloxifene and metabolites) **Measure:** PK parameters(Cmax) **Time Frame:** The blood samples (2.0 ml) will be collected at 4 points for each cycle(hour 0, hour 1, hour 2, hour 4 after raloxifene administration) **Description:** AUC 0-24hr, if with complete data, in each cycle of AUC0-24hr for XCHT and their metabolites (14 compounds) and intestinal UGT enzyme probe (raloxifene and metabolites) **Measure:** PK parameters(AUC) **Time Frame:** The blood samples (2.0 ml) will be collected at 4 points for each cycle(hour 0, hour 1, hour 2, hour 4 after raloxifene administration) **Description:** T1/2, if with complete data, in each cycle of T1/2 for XCHT and their metabolites (14 compounds) and intestinal UGT enzyme probe (raloxifene and metabolites) **Measure:** PK parameters(T1/2) **Time Frame:** The blood samples (2.0 ml) will be collected at 4 points for each cycle(hour 0, hour 1, hour 2, hour 4 after raloxifene administration) ### Eligibility Module **Eligibility Criteria:** Inclusion criteria: 1. Malignant tumor confirmed by histology or cytology; 2. Age ≥ 18 years old, ≤ 75 years old; 3. ECOG score of the patient ≤ 2 points; 4. Patients who have diarrhea worse than grade 2 due to irinotecan chemotherapy (the last dose of irinotecan is administered within 1 month); 5. Patients who plan to receive 3 cycles of FOLFIRI/mXELIRI chemotherapy; 6. Normal organ functions which can meet the requirements for systemic chemotherapy: * Normal bone marrow function: absolute neutrophil count (ANC) ≥ 1.5×109/L, PLT ≥ 100×109/L, hemoglobin ≥ 90g/L; * Normal renal functions: serum creatinine ≤ 1.5mg/dl (133μmol/L) and/or creatinine clearance ≥ 60ml/min; * Normal hepatic functions: total serum bilirubin level ≤ 1.5 times of the upper limit of normal value (ULN), serum aspartate aminotransferase (AST) \& alanine aminotransferase (ALT) ≤ 2.5× ULN; AST \& ALT ≤ 5 × ULN if abnormal hepatic functions are caused by a potentially malignant tumor. 7. Patients who can understand and complete the questionnaires in the case report form; 8. Patients who can understand and sign the informed consent form, is well compliant, and can be followed up. Exclusion Criteria: 1. Patients with diagnosed depression, obsession or/and schizophrenia; 2. Patients with diagnosed inflammatory bowel diseases (including Crohn's disease, ulcerative colitis) 3. Patient with active tuberculosis and other uncontrolled infections; 4. Patient who has previously received radiotherapy on the abdominal cavity or pelvic cavity; 5. Pregnant or lactating women; 6. Patient who previously had or is now having thromboembolic events. **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:** Yanjuan Zhu, Dr. **Phone:** 86 20 81887233 **Phone Ext:** 34830 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Yadong Chen, Dr. **Phone:** 86 20 81887233 **Phone Ext:** 34830 **Role:** CONTACT #### Locations **Location 1:** **City:** Guangzhou **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Haibo Zhang, Prof - **Phone:** 86-020-81887233 - **Phone Ext:** 34830 - **Role:** CONTACT **Country:** China **Facility:** Guangdong Provincial Hospital of Traditional Chinese Medicine **State:** Guangdong **Status:** RECRUITING **Zip:** 510120 #### Overall Officials **Official 1:** **Affiliation:** Guangdong Provincial Hospital of Traditional Chinese Medicine **Name:** Haibo Zhang, Prof. **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012817 - Term: Signs and Symptoms, Digestive ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M7159 - Name: Diarrhea - Relevance: HIGH - As Found: Diarrhea - ID: M15622 - Name: Signs and Symptoms, Digestive - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000003967 - Term: Diarrhea ### Intervention Browse Module - Ancestors - ID: D000004965 - Term: Estrogen Antagonists - ID: D000006727 - Term: Hormone Antagonists - ID: D000006730 - Term: Hormones, Hormone Substitutes, and Hormone Antagonists - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000020845 - Term: Selective Estrogen Receptor Modulators - ID: D000020847 - Term: Estrogen Receptor Modulators - ID: D000050071 - Term: Bone Density Conservation Agents ### Intervention Browse Module - Browse Branches - Abbrev: Micro - Name: Micronutrients - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: Hemat - Name: Hematinics - Abbrev: BDCA - Name: Bone Density Conservation Agents - Abbrev: Vi - Name: Vitamins ### Intervention Browse Module - Browse Leaves - ID: M6191 - Name: Leucovorin - Relevance: LOW - As Found: Unknown - ID: M8600 - Name: Fluorouracil - Relevance: LOW - As Found: Unknown - ID: M29233 - Name: Levoleucovorin - Relevance: LOW - As Found: Unknown - ID: M377 - Name: Capecitabine - Relevance: LOW - As Found: Unknown - ID: M1671 - Name: Irinotecan - Relevance: LOW - As Found: Unknown - ID: M8618 - Name: Folic Acid - Relevance: LOW - As Found: Unknown - ID: M22601 - Name: Raloxifene Hydrochloride - Relevance: HIGH - As Found: 1 mm - ID: M8116 - Name: Estrogens - Relevance: LOW - As Found: Unknown - ID: M8114 - Name: Estrogen Antagonists - Relevance: LOW - As Found: Unknown - ID: M30483 - Name: Estrogen Receptor Antagonists - 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: M22599 - Name: Estrogen Receptor Modulators - Relevance: LOW - As Found: Unknown - ID: M22597 - Name: Selective Estrogen Receptor Modulators - 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: D000020849 - Term: Raloxifene Hydrochloride ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03264079 **Brief Title:** Effect of Itraconazole on the Pharmacokinetics of Bardoxolone Methyl in Healthy Adults **Official Title:** An Open-Label Study of the Effect of a Strong CYP3A4 Inhibitor (Itraconazole) on the Pharmacokinetics of Bardoxolone Methyl in Healthy Volunteers #### Organization Study ID Info **ID:** 402-C-1701 #### Organization **Class:** INDUSTRY **Full Name:** Biogen ### Status Module #### Completion Date **Date:** 2017-11-13 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2024-02-02 **Type:** ACTUAL **Last Update Submit Date:** 2024-02-01 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2017-11-13 **Type:** ACTUAL #### Start Date **Date:** 2017-10-16 **Type:** ACTUAL **Status Verified Date:** 2024-02 #### Study First Post Date **Date:** 2017-08-28 **Type:** ACTUAL **Study First Submit Date:** 2017-08-23 **Study First Submit QC Date:** 2017-08-24 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Reata, a wholly owned subsidiary of Biogen #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Oversight Has DMC:** False ### Description Module **Brief Summary:** This trial will study the impact of a strong CYP3A4 inhibitor (itraconazole) on the pharmacokinetics of bardoxolone methyl (RTA 402) in healthy adult subjects. This is an open-label, fixed-sequence crossover pharmacokinetic (PK) study in healthy volunteers. Subjects will complete a screening visit within 28 days of Study Day 1. Subjects must qualify for the study based on inclusion/exclusion criteria. For Period 1, all qualified subjects shall receive a single oral dose of bardoxolone methyl (10 mg) on Day 1. For Period 2, following a washout of 14 days, itraconazole (SPORANOX®) capsules (100 mg) will be administered as a 200-mg single daily dose on Study Days 15 through 27, with bardoxolone methyl (10 mg) administered on Day 18 (1 hour after itraconazole administration). Bardoxolone methyl doses will be administered under fasted conditions. Itraconazole will be administered under fed conditions on Study Days 15-17, and Study Days 19-27. On study Day 18, itraconazole will be administered under fasted conditions. Subjects will be confined beginning on Study Day -1 through the last blood sample collection on Study Day 9 during Period 1, and from Study Day 14 through the last PK blood draw on Study Day 28 during Period 2. **Detailed Description:** Study Sponsor, originally Reata Pharmaceuticals, Inc., is now Reata Pharmaceuticals, Inc., a wholly owned subsidiary of Biogen. ### Conditions Module **Conditions:** - Healthy Volunteers **Keywords:** - RTA 402 - Bardoxolone methyl - Itraconazole - Sporanox - CYP3A4 ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP **Intervention Model Description:** All participants are placed into a one group to receive two interventions: one intervention during Period 1 and a second intervention during Period 2. For Period 1, all qualified subjects shall receive a single oral dose of bardoxolone methyl (10 mg) on Day 1. For Period 2, following a washout of 14 days, itraconazole (SPORANOX®) capsules (100 mg) will be administered as a 200-mg single daily dose on Study Days 15 through 27, with bardoxolone methyl (10 mg) administered on Day 18 (1 hour after itraconazole administration) ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 16 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Period 1: Bardoxolone methyl capsules 10 mg Period 2: two Itraconazole capsules 100 mg **Intervention Names:** - Drug: Bardoxolone methyl capsules 10 mg - Drug: Itraconazole capsules 100 mg **Label:** Bardoxolone methyl 10 mg and Itraconazole 200 mg **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Bardoxolone methyl 10 mg and Itraconazole 200 mg **Description:** One 10 mg capsule of bardoxolone methyl administered on Study Day 1 (Period 1) of the trial and one 10 mg capsule of bardoxolone methyl administered on Study Day 18 (Period 2) of the trial **Name:** Bardoxolone methyl capsules 10 mg **Other Names:** - RTA 402 **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Bardoxolone methyl 10 mg and Itraconazole 200 mg **Description:** Two 100 mg capsules of itraconazole administered once daily during Study Days 15 - 27 (Period 2) of the trial **Name:** Itraconazole capsules 100 mg **Other Names:** - Sporanox **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Area under the plasma concentration-time curve of bardoxolone methyl from time 0 to the last measurable time point (AUC0-t). **Time Frame:** 28 days **Measure:** Area under the plasma concentration-time curve of bardoxolone methyl extrapolated to infinity (AUC0-inf) **Time Frame:** 28 days **Measure:** Maximum observed drug concentration (Cmax) in plasma of bardoxolone methyl **Time Frame:** 28 days ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Male or female and age is between 18 and 55 years, inclusive; * Female subjects of childbearing potential must not be planning a pregnancy, pregnant, or lactating, and must have a negative serum pregnancy test result before enrollment into the study, and must be willing to use contraception as specified or abstain from sexual activity for the duration of the study; * If male, subject must be surgically sterile or practicing an approved method of contraception, from initial study drug administration through 90 days after administration of the last dose of study drug; * If male, subject agrees to abstain from sperm donation through 90 days after administration of the last dose of study drug; * Body Mass Index (BMI) is ≥ 18 to ≤31 kg/m2, inclusive; * A condition of general good health, based upon the results of a medical history, physical examination, vital signs, laboratory profile, and a 12-lead electrocardiogram (ECG), as judged by the investigator; * Must voluntarily sign and date each informed consent, approved by an Institutional Review Board (IRB), prior to the initiation of any screening or study-specific procedures. Exclusion Criteria: * History of clinically significant drug allergies, including allergies to any of the components of the investigational product and/or clinically significant food allergies as determined by the investigator; * Presence or history of any significant cardiovascular, gastrointestinal, hepatic, renal, pulmonary, hematologic, endocrine, immunologic, dermatologic, neurologic, or psychiatric disease, as determined by the investigator; * Presence of any other condition (including surgery) known to interfere with the absorption, distribution, metabolism, or excretion of medicines; * Known hypersensitivity to any component in the formulations of bardoxolone methyl, or SPORANOX®; * Requirement for any over-the-counter and/or prescription medication, vitamins, and/or herbal supplements on a regular basis; * Use of any medications (over-the-counter and/or prescription medication), vitamins, and/or herbal supplements, within the 30-day period prior to study drug administration or within 5 half-lives (if known), whichever is longer; * Recent (6-month) history of drug or alcohol abuse; * B-type natriuretic peptide (BNP) level \>200 pg/mL at screening; * Positive test result for hepatitis B surface antigen (HBsAg), hepatitis C virus antibody (HCV Ab), or HIV antibodies (HIV Ab) at screening; * Donation or loss of 550 mL or more blood volume (including plasmapheresis) or receipt of a transfusion of any blood product within 8 weeks prior to study drug administration; * Receipt of any investigational product within a time period equal to 10 half-lives of the product, if known, or a minimum of 30 days prior to study drug administration; * Positive screen results for drugs of abuse, alcohol, or cotinine at screening or Day -1; * Consumption of alcohol within 72 hours prior to study drug administration; * Consumption of grapefruit, grapefruit products, star fruit, star fruit products, or Seville oranges within the 72-hour period prior to study drug administration; * Use of tobacco or nicotine-containing products within the 6-month period preceding study drug administration; * Current enrollment in another clinical study; * Screening laboratory analyses that show any of the following abnormal laboratory results: 1. Alanine transaminase (ALT) or aspartate aminotransferase (AST) levels above the upper limit of normal (ULN); 2. Clinically significant abnormal ECG; ECG with QTc using Fridericia's correction formula (QTcF) \> 450 msec is exclusionary; 3. Consideration by the investigator, for any reason, that the subject is an unsuitable candidate to receive either bardoxolone methyl or itraconazole. **Healthy Volunteers:** True **Maximum Age:** 55 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Cincinnati **Country:** United States **Facility:** Medpace Clinical Pharmacology Unit **State:** Ohio **Zip:** 45227 ### IPD Sharing Statement Module **Description:** In accordance with Biogen's Clinical Trial Transparency and Data Sharing Policy on https://www.biogentrialtransparency.com/ **IPD Sharing:** YES **URL:** https://vivli.org/ ## Derived Section ### Intervention Browse Module - Ancestors - ID: D000000935 - Term: Antifungal Agents - ID: D000000890 - Term: Anti-Infective Agents - ID: D000058888 - Term: 14-alpha Demethylase Inhibitors - ID: D000065607 - Term: Cytochrome P-450 Enzyme Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000065088 - Term: Steroid Synthesis Inhibitors - ID: D000006727 - Term: Hormone Antagonists - ID: D000006730 - Term: Hormones, Hormone Substitutes, and Hormone Antagonists - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000065692 - Term: Cytochrome P-450 CYP3A 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: M20133 - Name: Itraconazole - Relevance: HIGH - As Found: Self-management - ID: M256158 - Name: Hydroxyitraconazole - 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: M4214 - Name: Anti-Infective Agents - 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 - ID: M9789 - Name: Hormones - Relevance: LOW - As Found: Unknown - ID: M9788 - Name: Hormone Antagonists - Relevance: LOW - As Found: Unknown - ID: M30564 - Name: Cytochrome P-450 CYP3A Inhibitors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000017964 - Term: Itraconazole ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00289679 **Brief Title:** Cytokine Profile and Metal Ion Concentrations at Patients Undergoing a Revision THR #### Organization Study ID Info **ID:** kf 01059/04 #### Organization **Class:** OTHER **Full Name:** Frederiksberg University Hospital ### Status Module #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2015-03-18 **Type:** ESTIMATED **Last Update Submit Date:** 2015-03-17 **Overall Status:** UNKNOWN **Status Verified Date:** 2007-07 #### Study First Post Date **Date:** 2006-02-10 **Type:** ESTIMATED **Study First Submit Date:** 2006-02-09 **Study First Submit QC Date:** 2006-02-09 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Frederiksberg University Hospital ### Description Module **Brief Summary:** To evaluate the concentration of cytokines and metal ions in blod samples and biopsies from patients undergoing a hip revision. ### Conditions Module **Conditions:** - Hip Revision ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Name:** Hip prosthesis revision **Type:** DEVICE ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * THR revision **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** arne borgwardt, md **Phone:** 004538163450 **Role:** CONTACT #### Locations **Location 1:** **City:** Frederiksberg **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** arne borgwardt, md - **Phone:** 004538163450 - **Role:** CONTACT **Country:** Denmark **Facility:** Frederiksberg University Hospital **Status:** RECRUITING **Zip:** 2000 #### Overall Officials **Official 1:** **Affiliation:** Frederiksberg University Hospital **Name:** arne borgwardt, md **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00527579 **Brief Title:** PET Imaging of Peripheral Benzodiazepine Receptors in Patients With Neurocysticercosis Using [F-18]FB **Official Title:** PET Imaging of Peripheral Benzodiazepine Receptors in Patients With Neurocysticercosis Using [F-18]FBR #### Organization Study ID Info **ID:** 070209 #### Organization **Class:** NIH **Full Name:** National Institutes of Health Clinical Center (CC) #### Secondary ID Infos **ID:** 07-M-0209 ### Status Module #### Completion Date **Date:** 2013-11-26 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-07-05 **Type:** ACTUAL **Last Update Submit Date:** 2018-07-03 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2013-11-26 **Type:** ACTUAL #### Start Date **Date:** 2007-09-04 **Status Verified Date:** 2013-11-26 #### Study First Post Date **Date:** 2007-09-11 **Type:** ESTIMATED **Study First Submit Date:** 2007-09-08 **Study First Submit QC Date:** 2007-09-08 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** NIH **Name:** National Institute of Mental Health (NIMH) #### Responsible Party **Type:** SPONSOR ### Description Module **Brief Summary:** The purpose of this protocol is to measure peripheral benzodiazepine receptors in the brain using positron emission tomography (PET) and compare the imaging results between patients and healthy people. **Detailed Description:** Objective In endemic regions neurocysticercosis is the most common cause of adult acquired epilepsy and thus an important public health problem. The disease is caused by infection with the larval form of the tapeworm, Taenia solium. Although neurocysticercosis is common only in many developing regions, an increased number of patients are diagnosed in developed countries mostly due to immigration of infected individuals. The peripheral benzodiazepine receptor (PBR) can be a clinically useful marker to detect neuroinflammation because activated microglia in inflammatory areas expresses high levels of PBR. PBR has been imaged with positron emission tomography (PET) using \[(11) C\]1-(2-chlorophenyl-N-methylpropyl)-3-isoquinoline carboxamide (PK11195), which provides low levels of specific signal. Recently we developed a new ligand, N-fluroacetyl-N-(2,5-dimethoxybenzyl)-2-phenoxyaniline (\[(18)F\]FBR), which showed much greater specific signals than \[(11) C\]PK11195 in non-human primates. The major objective of this protocol is to assess the utility of \[(18) F\]FBR PET to detect neuroinflammation in patients with neurocysticercosis. A secondary objective is to study whether some healthy subjects do not show binding of \[(18)F\]FBR by performing whole body imaging using \[18F\]FBR and binding assays using blood cells. In other protocols using a PET ligand with similar structure, \[(11)C\]PBR28, approximately 8% of subjects (9/\~ 118 ) did not show binding. In protocols 07-N-0035 and 08-M-0158, we compared binding of \[11C\]PBR28 and \[(11)C\]PK 11195 in approximately ten healthy subjects including five who did not show binding of \[(11)C\]PBR28 in prior whole body imaging. We found differences in organs with regard to sensitivity to the phenomenon of non-binding. In the non-binders, PBR28 showed no binding in all five organs with high PBR density. However, PK 11195 showed significantly reduced binding in only two organs with PBR. We now wish to determine whether PBR06 is more similar to PBR28 or to PK 11195 in terms of the non-binding phenomenon. In the current protocol, in addition to whole body imaging using \[(18)F\]FBR, we will do in vitro binding assays using blood cells as another tissue to examine the effect of non-binding. Study population For \[(18)F\]FBR brain scans, ten patients will be recruited and clinically followed under protocol 85-I-0127, Treatment of Cysticercosis including Neurocysticercosis with Praziquantel or Albendazole (PI: Theodore E. Nash, MD, NIAID). Fifteen healthy subjects will be recruited. For whole body scan using \[(18)F\]FBR, additional 30 healthy subjects will be recruited. Therefore, total accrual numbers are 10 patients with neurocysticercosis and 45 healthy subjects (15 for brain \[(18)F\]FBR and 30 for whole body \[(18)F\]FBR scans. Design Ten patients with neurocysticercosis and 15 age-matched healthy subjects will have brain PET scans. In addition, we will also perform a whole body PET scan on 30 healthy subjects to study the radiation-absorbed doses and study whether some healthy subjects do not show binding of \[(18)F\]FBR. Patients will have up to three \[(18)F\]FBR PET scans during the follow-up and the treatment under 85-I-0127, typically a few weeks apart. Outcome measures \<TAB\> In brain PET scans, \[(18)F\]FBR binding will be compared with clinical symptoms and MRI findings. In addition, the binding will be compared between patients and age-matched control subjects. We have calculated radiation absorbed doses in approximately seven healthy subjects who showed normal distribution (i.e., binders) of activity in organs. If we found subjects who appear to have no binding to \[(18)F\]FBR, we will calculate radiation-absorbed doses of the non-binders. ### Conditions Module **Conditions:** - Neurocysticercosis - Healthy **Keywords:** - Epilepsy - Taenia Solium - Microglia - Neuroinflammation - Compartment Model - Neurocysticercosis - Healthy Volunteer - HV ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** DIAGNOSTIC #### Enrollment Info **Count:** 24 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Name:** [F-18]FBR **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Binding of [F-18]FBR at peripheral benzodiazepine receptor. **Time Frame:** 3 years #### Secondary Outcomes **Measure:** MRI **Time Frame:** 3 years ### Eligibility Module **Eligibility Criteria:** * INCLUSION CRITERIA: COMMON TO PATIENTS WITH NEUROCYSTICERCOSIS AND HEALTHY SUBJECTS: Ages between 18 and 65, inclusive. \<TAB\> In addition, patients must meet the inclusion criteria of protocol 85-I-0127. Control subjects are healthy based on history, physical exams, ECG, and lab tests. EXCLUSION CRITERIA: COMMON TO ALL SUBJECTS: Current psychiatric illness, substance abuse or severe systemic disease based on history and physical exam. ECG with clinically significant abnormalities. Any existing physical exam and ECG within one year will be reviewed and if none already exists in the chart, these will be obtained and reviewed. Prior participation in other research protocols or clinical care in the last year such that radiation exposure would exceed the annual guideline of RSC. Pregnancy or breast feeding. Claustrophobia. Positive HIV test. Cannot lie on back for a few hours for the PET scans. ADDITIONAL EXCLUSION CRITERIA TO BRAIN SCANS: Presence of ferromagnetic metal in the body or heart pacemaker. \[(18)F\]FBR did not show binding in a whole body PET \[(18)F\]FBR scan in the past. ADDITIONAL EXCLUSION CRITERIA FOR PATIENTS: Medically unstable. Seizures are not well controlled with medications. A history of brain disease other than neurocysticercosis. Laboratory tests with clinically significant abnormalities unrelated to neurocysticercosis or its treatment. ADDITIONAL EXCLUSION CRITERIA FOR HEALTHY SUBJECTS: Laboratory tests with clinically significant abnormalities. A history of brain disease. **Maximum Age:** 65 Years **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, 9000 Rockville Pike **State:** Maryland **Zip:** 20892 #### Overall Officials **Official 1:** **Affiliation:** National Institute of Mental Health (NIMH) **Name:** Masahiro Fujita, M.D. **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Anholt RR, De Souza EB, Oster-Granite ML, Snyder SH. Peripheral-type benzodiazepine receptors: autoradiographic localization in whole-body sections of neonatal rats. J Pharmacol Exp Ther. 1985 May;233(2):517-26. **PMID:** 2987488 **Citation:** Anholt RR, Murphy KM, Mack GE, Snyder SH. Peripheral-type benzodiazepine receptors in the central nervous system: localization to olfactory nerves. J Neurosci. 1984 Feb;4(2):593-603. doi: 10.1523/JNEUROSCI.04-02-00593.1984. **PMID:** 6321699 **Citation:** Anholt RR, Pedersen PL, De Souza EB, Snyder SH. The peripheral-type benzodiazepine receptor. Localization to the mitochondrial outer membrane. J Biol Chem. 1986 Jan 15;261(2):576-83. **PMID:** 3001071 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020809 - Term: Central Nervous System Helminthiasis - ID: D000020807 - Term: Central Nervous System Parasitic Infections - ID: D000002494 - Term: Central Nervous System Infections - ID: D000007239 - Term: Infections - ID: D000010272 - Term: Parasitic Diseases - ID: D000002590 - Term: Cestode Infections - ID: D000006373 - Term: Helminthiasis - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000009422 - Term: Nervous System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC01 - Name: Infections - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M7983 - Name: Epilepsy - Relevance: LOW - As Found: Unknown - ID: M21874 - Name: Neurocysticercosis - Relevance: HIGH - As Found: Neurocysticercosis - ID: M6756 - Name: Cysticercosis - Relevance: HIGH - As Found: Neurocysticercosis - ID: M16396 - Name: Taeniasis - Relevance: HIGH - As Found: Neurocysticercosis - ID: M9461 - Name: Helminthiasis - 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: M13185 - Name: Parasitic Diseases - Relevance: LOW - As Found: Unknown - ID: M5743 - Name: Central Nervous System Infections - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: T1713 - Name: Cysticercosis - Relevance: HIGH - As Found: Neurocysticercosis - ID: T2684 - Name: Helminthiasis - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000020019 - Term: Neurocysticercosis - ID: D000003551 - Term: Cysticercosis - ID: D000013622 - Term: Taeniasis ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05347979 **Brief Title:** Effect of Relacorilant on the Pharmacokinetics of the Sensitive P-glycoprotein Substrate Dabigatran Etexilate in Healthy Participants **Official Title:** An Open-Label, Drug-Drug Interaction Study Designed to Evaluate the Effect of Relacorilant on the Pharmacokinetics of the Sensitive P-glycoprotein Substrate Dabigatran Etexilate in Healthy Subjects #### Organization Study ID Info **ID:** CORT125134-132 #### Organization **Class:** INDUSTRY **Full Name:** Corcept Therapeutics ### Status Module #### Completion Date **Date:** 2022-07-19 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-02-09 **Type:** ACTUAL **Last Update Submit Date:** 2023-02-07 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2022-07-19 **Type:** ACTUAL #### Start Date **Date:** 2022-05-25 **Type:** ACTUAL **Status Verified Date:** 2023-02 #### Study First Post Date **Date:** 2022-04-27 **Type:** ACTUAL **Study First Submit Date:** 2022-04-18 **Study First Submit QC Date:** 2022-04-22 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Corcept Therapeutics #### 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 is to determine the effect of relacorilant on the pharmacokinetics (PK) of the sensitive P-glycoprotein (P-gp) substrate dabigatran etexilate. **Detailed Description:** The investigational medicinal product (IMP), relacorilant, and the non-investigational medicinal product (NIMP), dabigatran etexilate, will be used to evaluate the effect of relacorilant on the PK of the sensitive P-gp substrate, dabigatran etexilate in healthy participants. Participants will receive a single dose of dabigatran etexilate before and after administration of daily (QD) doses of relacorilant for 11 days. As all participants will receive the same treatments, the study will be open-label and no randomization is required. ### Conditions Module **Conditions:** - Cushing Syndrome - Neoplasms ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 30 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Following an overnight fast, participants will receive 75 mg dabigatran etexilate on Day 1, 400 mg dose of relacorilant QD on Days 3 to 13, and 75 mg dabigatran etexilate on Day 12. On Day 12, dabigatran etexilate will be dosed at approximately the same time as the relacorilant dose. **Intervention Names:** - Drug: Dabigatran Etexilate - Drug: Relacorilant **Label:** Dabigatran Etexilate (NIMP) and Relacorilant (IMP) **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Dabigatran Etexilate (NIMP) and Relacorilant (IMP) **Description:** Dabigatran will be administered orally as a 75 mg capsule on Day 1 and Day 12. **Name:** Dabigatran Etexilate **Other Names:** - Pradaxa® **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Dabigatran Etexilate (NIMP) and Relacorilant (IMP) **Description:** Relacorilant will be administered orally as 4 X 100 mg capsules (400 mg) on Days 3 through 13. **Name:** Relacorilant **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Maximum Observed Plasma Concentration (Cmax) of Dabigatran When Administered With and Without Relacorilant **Time Frame:** Up to Day 14 **Measure:** Area Under the Curve from Time 0 to the Time of Last Measurable Concentration (AUC0-last) of Dabigatran When Administered With and Without Relacorilant **Time Frame:** Up to Day 14 **Measure:** Area Under the Curve from Time 0 Extrapolated to Infinity (AUC 0-inf) of Dabigatran When Administered With and Without Relacorilant **Time Frame:** Up to Day 14 #### Secondary Outcomes **Measure:** Plasma Concentrations of Relacorilant **Time Frame:** Up to Day 6 **Measure:** Number of Participants with Adverse Events (AEs) and Serious Adverse Events (SAEs) **Time Frame:** Up to 30 days post final dose **Measure:** Number of Participants with Clinically Significant Abnormalities in Blood Pressure and Heart Rate **Time Frame:** Up to Day 14 **Measure:** Number of Participants with Clinically Significant Abnormalities in Electrocardiogram (ECG) Measurements **Time Frame:** Up to Day 14 **Measure:** Number of Participants with Clinically Significant Abnormalities in Laboratory Safety Tests (Clinical Chemistry, Hematology, Urinalysis) **Time Frame:** Up to Day 14 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Must agree to use an adequate method of contraception * Healthy men or non-pregnant, non-lactating healthy women of non-childbearing potential * Body mass index (BMI) of 19.0 to 32.0 kg/m\^2 as measured at screening * Weight ≥50 kg at screening Exclusion Criteria: * Serious adverse reaction or serious hypersensitivity to any drug or the formulation excipients * Presence or history of clinically significant allergy requiring treatment, as judged by the Investigator. * Significant serious skin disease, including rash, food allergy, eczema, psoriasis, or urticaria * History of clinically significant cardiovascular, renal, hepatic, chronic respiratory or gastrointestinal disease, bleeding disorder or abnormal bleeding, or clinically significant active bleeding, congenital or acquired clotting disorders, neurological or psychiatric disorder * History of esophagitis, gastritis, gastroesophageal reflux surgery, or significant trauma or surgery within 1 month of IMP/NIMP administration * Have poor venous access that limits phlebotomy * Evidence of current severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) infection * Clinically significant abnormal clinical chemistry, hematology or thrombocytopenia, coagulation or urinalysis * Positive hepatitis B surface antigen (HBsAg), hepatitis C virus antibody (HCV Ab) or human immunodeficiency virus (HIV) antibody results * Evidence of renal impairment at screening * Pregnant or lactating women * Women of childbearing potential. A woman is considered of childbearing potential unless she is permanently sterile or is postmenopausal * Participants who have received any IMP in a clinical research study within 5 half-lives or within 30 days prior to first dose. * Participants who are taking, or have taken, any prescribed or over-the-counter drug or herbal remedies in the 14 days before IMP/NIMP administration. * Participants who are currently using glucocorticoids or have a history of systemic glucocorticoid use at any dose within the last 12 months before IMP/NIMP administration, or 3 months for inhaled products * Participants who are taking, or have taken, heparin, vitamin K antagonists or anti-platelet agents within 1 month before IMP/NIMP administration * Participants who are taking, or have taken, selective serotonin re-uptake inhibitors, serotonin and norepinephrine re-uptake inhibitors within 3 months before IMP/NIMP administration * History of any drug or alcohol abuse in the past 2 years * A confirmed positive alcohol urine test at screening or admission * Current smokers and those who have smoked within the last 12 months * Current users of e-cigarettes and nicotine replacement products and those who have used these products within the last 12 months * Positive drugs of abuse test result * Male participants with pregnant or lactating partners * Donation of blood within 2 months or donation of plasma within 7 days prior to first dose of study medication **Healthy Volunteers:** True **Maximum Age:** 55 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Miami **Country:** United States **Facility:** Site 01 **State:** Florida **Zip:** 33126 #### Overall Officials **Official 1:** **Affiliation:** Corcept Therapeutics **Name:** Joseph Custodio, PhD **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000000308 - Term: Adrenocortical Hyperfunction - ID: D000000307 - Term: Adrenal Gland Diseases - ID: D000004700 - Term: Endocrine System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC20 - Name: Immune System Diseases - 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: M10018 - Name: Hypersensitivity - Relevance: LOW - As Found: Unknown - ID: M16355 - Name: Syndrome - Relevance: LOW - As Found: Unknown - ID: M6689 - Name: Cushing Syndrome - Relevance: HIGH - As Found: Cushing's Syndrome - ID: M3660 - Name: Adrenocortical Hyperfunction - Relevance: LOW - As Found: Unknown - ID: M3659 - Name: Adrenal Gland Diseases - Relevance: LOW - As Found: Unknown - ID: M7862 - Name: Endocrine System Diseases - Relevance: LOW - As Found: Unknown - ID: T1679 - Name: Cushing's Syndrome - Relevance: HIGH - As Found: Cushing's Syndrome - ID: T2879 - Name: Hyperadrenalism - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000003480 - Term: Cushing Syndrome ### Intervention Browse Module - Ancestors - ID: D000000991 - Term: Antithrombins - ID: D000015842 - Term: Serine Proteinase Inhibitors - ID: D000011480 - Term: Protease Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000000925 - Term: Anticoagulants ### Intervention Browse Module - Browse Branches - Abbrev: AnCoag - Name: Anticoagulants - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: AA - Name: Amino Acids ### Intervention Browse Module - Browse Leaves - ID: M490 - Name: Dabigatran - Relevance: HIGH - As Found: Intraocular Lens - ID: M340819 - Name: polysaccharide-K - Relevance: LOW - As Found: Unknown - ID: M4307 - Name: Antithrombins - Relevance: LOW - As Found: Unknown - ID: M4306 - Name: Antithrombin III - Relevance: LOW - As Found: Unknown - ID: M14343 - Name: Protease Inhibitors - Relevance: LOW - As Found: Unknown - ID: M18391 - Name: Serine Proteinase Inhibitors - Relevance: LOW - As Found: Unknown - ID: M19609 - Name: HIV Protease Inhibitors - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown - ID: M4244 - Name: Anticoagulants - Relevance: LOW - As Found: Unknown - ID: T18 - Name: Serine - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000069604 - Term: Dabigatran ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01503879 **Brief Title:** Evaluation of Gas Exchange by the Measurement of Lung Diffusion for Carbon Monoxide During General Anaesthesia **Official Title:** Effects of General Anaesthesia and Invasive Mechanical Ventilation on Alveolo-capillary Membrane: Evaluation of Gas Exchange by the Measurement of Lung Diffusion for Carbon Monoxide (DLCO) and Plasma Dosage of Surfactant Protein-B (SPB). #### Organization Study ID Info **ID:** ARHSG 10 2010 DLCO1 #### Organization **Class:** OTHER **Full Name:** San Gerardo Hospital ### Status Module #### Completion Date **Date:** 2012-10 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2012-08-29 **Type:** ESTIMATED **Last Update Submit Date:** 2012-08-28 **Overall Status:** UNKNOWN #### Start Date **Date:** 2011-10 **Status Verified Date:** 2012-08 #### Study First Post Date **Date:** 2012-01-04 **Type:** ESTIMATED **Study First Submit Date:** 2011-12-31 **Study First Submit QC Date:** 2012-01-03 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** San Gerardo Hospital #### Responsible Party **Investigator Affiliation:** San Gerardo Hospital **Investigator Full Name:** Prof. Roberto Fumagalli's **Investigator Title:** MD **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Mechanical ventilation is a therapeutic method used in order to keep gas exchange adequate to cell metabolism in patients with acute respiratory failure. It is currently proved that, although on one hand the use of this method keeps gas exchange, on the other hand it promotes and supports pulmonary inflammatory processes (VILI). A recent study about the effect of positive end-expiratory pressure (PEEP) on DLCO (diffusing capacity of the lung for carbon monoxide) in patients undergoing invasive mechanical ventilation has proved that patients without any evident pulmonary disease (negative medical history, negative chest clinical examination, normal chest X-ray radiography and normal arterial oxygen tension \[PaO2\]) after 24 hours of invasive mechanical ventilation show a significant worsening of pulmonary gas exchange properties. The authors have supposed that this worsening may be caused by an early alteration of alveolar-capillary membrane caused by mechanical ventilation itself. This hypothesis finds support in some studies carried out on animal models which founds that mechanical ventilation, even when low tidal volumes (Vt) are set for a few hours, is able to induce lung injury (as shown by histologic findings). The most sensitive and specific tools the investigators can currently rely on for the study of alveolar-capillary membrane are the measurement of diffusing capacity of the lung for carbon monoxide (DLCO) and the evaluation of plasmatic levels of pulmonary surfactant protein B (SPB). DLCO is a standard, widely diffused technique for the evaluation of functional alterations of alveolar-capillary membrane and it is currently available also for patients undergoing invasive mechanical ventilation. SBP is produced by type II pneumocytes in the alveoli. An increase of its plasmatic levels is correlated to a decay of pulmonary gas exchange; SPB thus can be considered an alveolar-capillary membrane anatomical damage marker. The primary end-point of this study is to evaluate the changes of anatomical (SPB) and functional (DLCO) features of alveolar-capillary membrane between the spontaneous breathing and mechanical ventilation as well as the progressive changes affecting DLCO and SPB over time during general anaesthesia and mechanical ventilation in patients with otherwise healthy lung undergoing elective surgery. This in order to check the timing of the observed worsening of alveolar-capillary membrane function, and to find out if the process is progressive in time. The secondary end point is to check if the alterations of functional features of alveolar membrane (DLCO) are proportionate to the increase of alveolar injury marker (SPB), in order to understand if the worsening of alveolar-capillary membrane function is to be attributable to an anatomical damage or to a physiologic change of the ventilation-perfusion matching. ### Conditions Module **Conditions:** - Lung Diffusion - Acute Respiratory Failure **Keywords:** - general anaesthesia - invasive mechanical ventilation - DLCO - SPB ### Design Module #### Bio Spec **Description:** surfactant protein B **Retention:** SAMPLES_WITHOUT_DNA #### Design Info **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 20 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * over 18 years of age * undergoing a non-thoracic, non-laparoscopic surgery, lasting more than three hours and requiring general anaesthesia and invasive mechanical ventilation Exclusion Criteria: * COPD 3 Gold stage or above * ASA physical status classification system 4 or above * heart failure NYHA 2 or above * chronic kidney disease * axillary temperature over 38 °C * BMI over 30 kg/m\^2 * pregnancy or breastfeeding **Healthy Volunteers:** True **Minimum Age:** 18 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Patients undergoing elective surgery lasting more than three hours, requiring general anaesthesia and invasive mechanical ventilation. ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Roberto Fumagalli, MD **Phone:** +390392339269 **Role:** CONTACT #### Locations **Location 1:** **City:** Monza **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Roberto Fumagalli, MD - **Phone:** +390392339269 - **Role:** CONTACT ***Contact 2:*** - **Name:** Roberto Fumagalli, MD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Italy **Facility:** Ospedale San Gerardo **State:** MB **Status:** RECRUITING #### Overall Officials **Official 1:** **Affiliation:** Milano Bicocca University **Name:** Roberto MD Fumagalli **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012120 - Term: Respiration Disorders - ID: D000012140 - Term: Respiratory Tract Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M14968 - Name: Respiratory Insufficiency - Relevance: HIGH - As Found: Respiratory Failure - ID: M14957 - Name: Respiration Disorders - 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: D000012131 - Term: Respiratory Insufficiency ### Intervention Browse Module - Browse Branches - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Resp - Name: Respiratory System Agents ### Intervention Browse Module - Browse Leaves - ID: M4107 - Name: Anesthetics - Relevance: LOW - As Found: Unknown - ID: M14517 - Name: Pulmonary Surfactants - Relevance: LOW - As Found: Unknown - ID: M5506 - Name: Carbon Monoxide - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05762679 **Acronym:** Myofascial **Brief Title:** Quantifying Myofascial Dysfunction in Post-Stroke Pain **Official Title:** Developing Quantitative Imaging and Other Relevant Biomarkers of Myofascial Tissues for Clinical Pain Management #### Organization Study ID Info **ID:** IRB00354876 #### Organization **Class:** OTHER **Full Name:** Johns Hopkins University #### Secondary ID Infos **ID:** 1R61AT012279-01 **Link:** https://reporter.nih.gov/quickSearch/1R61AT012279-01 **Type:** NIH ### Status Module #### Completion Date **Date:** 2024-05-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-05-03 **Type:** ACTUAL **Last Update Submit Date:** 2024-05-02 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-05-31 **Type:** ESTIMATED #### Start Date **Date:** 2023-02-28 **Type:** ACTUAL **Status Verified Date:** 2024-05 #### Study First Post Date **Date:** 2023-03-10 **Type:** ACTUAL **Study First Submit Date:** 2023-02-28 **Study First Submit QC Date:** 2023-02-28 ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** National Center for Complementary and Integrative Health (NCCIH) **Class:** OTHER **Name:** New York University **Class:** OTHER **Name:** George Mason University #### 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:** The purpose of this study is to quantify the extent of GlycosAminoGlycan/Hyaluronic Acid (GAG/HA) accumulation using T1rho (T1ρ) MRI in the paretic versus non-paretic shoulder rotator muscles, and correlate the T1ρ Magnetic Resonance Imaging (MRI) measurements with US echo texture measurements to develop a clinic-friendly tool to infer the extent of HA accumulation; and to distinguish between latent versus active Post Stroke Shoulder Pain (PSSP) using ultrasound (US) shear strain mapping of the same muscles on the paretic side compared with the non-paretic side. **Detailed Description:** Shoulder pain is extremely common after stroke and occurs in 30-70% of patients. Chronic post stroke shoulder pain (PSSP) contributes to depression, interferes with motor recovery, and decreases quality of life. Although PSSP is thought to be caused by damage to the myofascial tissues around the shoulder joint, the pathophysiology of myofascial dysfunction and pain in PSSP has not been elucidated, leading to missed opportunities for early diagnosis, and variable success with pain management. The accumulation of HA in muscle and its fascia can cause myofascial dysfunction. HA is a GAG and a chief constituent of the extracellular matrix of muscle. In physiologic quantities, it functions as a lubricant and a viscoelastic shock absorber, enabling force transmission during muscle contraction and stretch. Reduced joint mobility and spasticity can result in focal accumulation and alteration of HA in muscle, leading to the development of taut bands, dysfunctional gliding of deep fascia and muscle layers, Reduced Range of Motion (ROM), and pain. Muscle HA concentrations can be imaged using T1ρ MRI, and myofascial dysfunction can be assessed using echo texture analysis and shear strain mapping on quantitative US, which may serve as useful biomarkers to elucidate the pathophysiology of myofascial dysfunction in PSSP. ### Conditions Module **Conditions:** - Myofascial Dysfunction **Keywords:** - Glycosaminoglycan - Hyaluronic acid - Post stroke shoulder pain ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** CROSS_SECTIONAL #### Enrollment Info **Count:** 40 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Latent PSSP defined as focal palpable nodules that may be tender on palpation with pain rating of \< 5/10 when combined with the hand-behind-neck (HBN) maneuver. **Label:** Latent PSSP #### Arm Group 2 **Description:** Active PSSP is defined as focal palpable nodules that are tender on palpation, reproducing the pain, and eliciting a pain rating of \>= 5/10 when combined with the hand-behind-neck (HBN) maneuver. **Label:** Active PSSP ### Outcomes Module #### Primary Outcomes **Description:** Comparison of T1rho (T1ρ) MRI of pectoralis major and infraspinatus muscles in paretic and non-paretic shoulders **Measure:** To quantify the extent of HA accumulation in shoulder muscles using quantitative MRI and US. **Time Frame:** Baseline **Description:** Comparison of peak lateral shear strain of pectoralis major and infraspinatus muscles in paretic latent vs active PSSP compared to non-paretic shoulders **Measure:** To distinguish between latent versus active PSSP using US shear strain mapping of the same muscles on the paretic side compared with the non-paretic side. **Time Frame:** Baseline ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * 18 years or older * Hemiparesis from Ischemic or Hemorrhagic Stroke * 4-120 months post-stroke with Hemiparesis since the incidence and intensity of PSSP * Show a difference of more than 10 degrees of passive ER-ROM between non-paretic and paretic shoulders with or without pain * Able to provide informed consent and comply with testing protocols Exclusion Criteria: * Received treatment for spasticity with Botulinum Toxin or Intrathecal Baclofen within the past three months * Have another neurologic condition that may affect motor response (e.g. Parkinson's disease, Amyotrophic Lateral Sclerosis (ALS), Multiple Sclerosis (MS)) * Have a contraindication to MRI (claustrophobia, magnetic pacemakers and clips) * Have non-musculoskeletal PSSP such as only central pain or Chronic Regional Pain Syndrome (CRPS) * Have a complicated medical condition, or significant injury to either upper limb. **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** We will enroll 20 patients each with latent PSSP and active PSSP group, repectively. ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Preeti Raghavan, MD **Phone:** 410-955-0703 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Jonny Huang, DPT **Phone:** (410) 614-1347 **Role:** CONTACT #### Locations **Location 1:** **City:** Baltimore **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Jonny Huang, DPT - **Phone:** 410-614-1347 - **Role:** CONTACT ***Contact 2:*** - **Name:** Preeti Raghavan, MD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Johns Hopkins University School of Medicine **State:** Maryland **Status:** RECRUITING **Zip:** 21287 #### Overall Officials **Official 1:** **Affiliation:** Johns Hopkins University **Name:** Preeti Raghavan, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Browse Branches - Abbrev: BC05 - Name: Musculoskeletal Diseases - 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 ### Condition Browse Module - Browse Leaves - ID: M21907 - Name: Shoulder Pain - Relevance: LOW - As Found: Unknown - ID: M22306 - Name: Stroke - Relevance: LOW - As Found: Unknown - ID: M13066 - Name: Pain - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M9878 - Name: Hyaluronic Acid - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02429479 **Brief Title:** Preparing Family Caregivers to Make Medical Decisions for Their Loved Ones **Official Title:** Preparing Family Caregivers of Very Ill Patients for End-of-Life Decision Making #### Organization Study ID Info **ID:** 37476 #### Organization **Class:** OTHER **Full Name:** Milton S. Hershey Medical Center #### Secondary ID Infos **ID:** 1R01NR012757-01A1 **Link:** https://reporter.nih.gov/quickSearch/1R01NR012757-01A1 **Type:** NIH ### Status Module #### Completion Date **Date:** 2021-06-30 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2024-01-26 **Type:** ACTUAL **Last Update Submit Date:** 2024-01-25 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2017-06-30 **Type:** ACTUAL #### Start Date **Date:** 2013-06-01 **Type:** ACTUAL **Status Verified Date:** 2021-08 #### Study First Post Date **Date:** 2015-04-29 **Type:** ESTIMATED **Study First Submit Date:** 2015-04-24 **Study First Submit QC Date:** 2015-04-24 ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** National Institute of Nursing Research (NINR) **Class:** OTHER **Name:** Brigham and Women's Hospital #### Lead Sponsor **Class:** OTHER **Name:** Milton S. Hershey Medical Center #### Responsible Party **Investigator Affiliation:** Milton S. Hershey Medical Center **Investigator Full Name:** Benjamin H. Levi **Investigator Title:** Co-PI **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The overarching goal of the project is to improve the process and experience of surrogate decision-making by family caregivers. Since feeling unprepared to make surrogate decisions is a major contributor to caregiver stress, the primary outcome is caregiver self-efficacy --i.e., caregivers' assessment of how well prepared they feel to serve effectively as a surrogate decision-maker. Through follow-on Renewal funding, we are now also qualitatively examining family caregivers' experience with surrogate decision-making. **Detailed Description:** The long-term goal is to help family caregivers of seriously ill patients be better prepared to serve as surrogate decision-makers when their loved ones can no longer make medical decisions for themselves. Research shows that family caregivers find surrogate decision-making highly stressful and emotionally burdensome, in part because they feel unprepared for surrogate decision-making. To date, no studies have determined which advance care planning (ACP) process best prepares caregivers for this role. The investigators' prior work shows that a computer-based decision aid can help patients make more informed decisions and communicate their wishes more effectively. The investigators now propose to determine if family caregivers of patients with life-threatening illnesses are better prepared for surrogate decision-making: 1) when they engage in a structured ACP process together with patients; and 2) when they use this online decision aid for ACP. This will be accomplished via a randomized, controlled trial with a 2 x 2 factorial design comprising 4 groups: Standard ACP/Patient Alone (Group 1), Decision Aid/Patient Alone (Group 2), Standard ACP/Patients and Caregivers Together (Group 3), and Decision Aid/Patients and Caregivers Together (Group 4). ### Conditions Module **Conditions:** - Neoplasms - Heart Failure - Kidney Diseases - Lung Diseases **Keywords:** - Advance Care Planning - Living Will - Family Caregiver - Advance Directive - Surrogate Decision Maker - Proxy - Self-Efficacy ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** FACTORIAL ##### Masking Info **Masking:** SINGLE **Who Masked:** - PARTICIPANT **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Count:** 285 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients (without their family caregiver) complete a standard living will form online. **Intervention Names:** - Behavioral: Standard advance care planning **Label:** Standard ACP/Patient Alone **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Patients (without their family caregiver) complete Making Your Wishes Known, an online decision aid for advance care planning. **Intervention Names:** - Behavioral: Making Your Wishes Known **Label:** Decision Aid/Patient Alone **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Patients and their family caregiver together complete a standard living will form online. **Intervention Names:** - Behavioral: Standard advance care planning **Label:** Standard ACP/Together **Type:** ACTIVE_COMPARATOR #### Arm Group 4 **Description:** Patients and their family caregiver together complete Making Your Wishes Known, an online decision aid for advance care planning. **Intervention Names:** - Behavioral: Making Your Wishes Known **Label:** Decision Aid/Together **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Decision Aid/Patient Alone - Decision Aid/Together **Description:** Making Your Wishes Known provides tailored education, values clarification exercises, and a sophisticated decision aid that translates an individual's goals and preferences into a specific medical plan that can be implemented by a healthcare team. **Name:** Making Your Wishes Known **Type:** BEHAVIORAL #### Intervention 2 **Arm Group Labels:** - Standard ACP/Patient Alone - Standard ACP/Together **Description:** This is a online simple living will form. **Name:** Standard advance care planning **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** Family caregiver self-efficacy is measured using a validated questionnaire to determine if they feel better prepared to serve as surrogates for their loved one. **Measure:** Self-efficacy **Time Frame:** 6 weeks #### Secondary Outcomes **Description:** Family caregiver responses to treatment decisions hypothetical clinical vignettes will be compared to the decisions for the same vignettes made by their loved one. Each vignette has 6-8 associated treatment decisions; the family caregiver's response for each item will be compared with the loved one's (i.e., patient's) response, and a total concordance (i.e., number of items for which there is agreement) will be calculated. **Measure:** Accuracy of medical decisions **Time Frame:** 6 weeks **Description:** Using validated instruments and semi-structured interviews, family caregivers who have made a major medical decision on behalf of their loved one will report their level of distress, decisional conflict, satisfaction with decision, and experience with surrogate decision-making. **Measure:** Family caregivers' stress associated with actual (i.e., real-life) surrogate decision-making **Time Frame:** 1-2 years **Description:** Family caregivers will complete a questionnaire that assess their knowledge of surrogate responsibilities and end-of-life medical conditions and treatments **Measure:** Family caregiver knowledge **Time Frame:** 6 weeks - 2 years **Description:** Family Caregivers are interviewed about the depth of communication with their loved one (frequency, content, helpfulness of discussions) regarding advance care planning issues. **Measure:** Depth of communication **Time Frame:** 2 years **Description:** Participants who complete the advance care planning interventions fill out an evaluation of the intervention using a 16-item questionnaire. This instrument comprises: Twelve 5-point Likert-style questions on how the program presented various kinds of information; helped the user clarify values, choose a spokesperson, etc.; and helped the user document or be prepared communicate their wishes to others. Three 10-point Likert-style questions on user overall satisfaction, with the advance directive created by the intervention, and the amount of information provided. One open-ended item asking how the intervention was helpful. **Measure:** Satisfaction with advance care planning **Time Frame:** 1st study visit ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. 18 years of age or older 2. Diagnosis of kidney disease (e.g. chronic kidney disease, end stage renal disease ) OR advanced cancer (Stage IV disease or having an estimated survival of \<2 years) OR severe heart failure (e.g. New York Heart Assoc. Class III or Class IV) OR severe lung disease (e.g. Stage III or Stage IV COPD by modified GOLD Spirometric Classification, Idiopathic Pulmonary Fibrosis). 3. Able to read and understand English at an 8th grade level (word 26 on either blue or tan version of the WRAT-3 reading subtest) 4. Neuro-cognitively able to engage in ACP (Mini Mental State Exam (MMSE) score \>23) 5. No active suicidal ideations (i.e., score of 0 or 1 on item 9 of the BDI-II). Exclusion Criteria: * Failure on any of the above inclusion criteria. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Boston **Country:** United States **Facility:** Brigham & Women's Hospital **State:** Massachusetts **Zip:** 02120 **Location 2:** **City:** Hershey **Country:** United States **Facility:** Penn State Milton S. Hershey Medical Center / Penn State College of Medicine **State:** Pennsylvania **Zip:** 17033 #### Overall Officials **Official 1:** **Affiliation:** Penn State Milton S. Hershey Medical Center / Penn State College of Medicine **Name:** Benjamin H Levi, MD PhD **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** Penn State Milton S. Hershey Medical Center / Penn State College of Medicine **Name:** Michael J Green, MD MS **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Access Criteria:** Researchers who provide a methodologically sound proposal/request for these data. **Description:** We will present our findings at conferences and in peer-reviewed publications, and will share our study protocol, consent forms, measures, and statistical plan with other researchers upon request. **Info Types:** - STUDY_PROTOCOL - SAP - ICF **IPD Sharing:** YES **Time Frame:** July 1, 2023 - June 30, 2026 ### References Module #### References **Citation:** Thiede E, Levi BH, Lipnick D, Johnson R, Seo La I, Lehman EB, Smith T, Wiegand D, Green M, Van Scoy LJ. Effect of Advance Care Planning on Surrogate Decision Makers' Preparedness for Decision Making: Results of a Mixed-Methods Randomized Controlled Trial. J Palliat Med. 2021 Jul;24(7):982-993. doi: 10.1089/jpm.2020.0238. Epub 2020 Dec 29. **PMID:** 33373538 **Citation:** Lipnick D, Green M, Thiede E, Smith TJ, Lehman EB, Johnson R, La IS, Wiegand D, Levi BH, Van Scoy LJ. Surrogate Decision Maker Stress in Advance Care Planning Conversations: A Mixed-Methods Analysis From a Randomized Controlled Trial. J Pain Symptom Manage. 2020 Dec;60(6):1117-1126. doi: 10.1016/j.jpainsymman.2020.07.001. Epub 2020 Jul 6. **PMID:** 32645452 **Citation:** Foy AJ, Levi BH, Van Scoy LJ, Bucher A, Dimmock A, Green MJ. Patient Preference to Accept Medical Treatment Is Associated with Spokesperson Agreement. Ann Am Thorac Soc. 2019 Apr;16(4):518-521. doi: 10.1513/AnnalsATS.201806-428RL. No abstract available. **PMID:** 30714833 #### See Also Links **Label:** This is the website for the intervention arm advance care planning decision aid. **URL:** https://www.makingyourwishesknown.com ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014570 - Term: Urologic Diseases - ID: D000052776 - Term: Female Urogenital Diseases - ID: D000005261 - Term: Female Urogenital Diseases and Pregnancy Complications - ID: D000091642 - Term: Urogenital Diseases - ID: D000052801 - Term: Male Urogenital Diseases - ID: D000012140 - Term: Respiratory Tract Diseases ### Condition Browse Module - Browse Branches - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: All - Name: All Conditions - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M10698 - Name: Kidney Diseases - Relevance: HIGH - As Found: Kidney Disease - ID: M11168 - Name: Lung Diseases - Relevance: HIGH - As Found: Lung Disease - ID: M9421 - Name: Heart Failure - Relevance: LOW - As Found: Unknown - ID: M6845 - Name: Death - 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: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000008171 - Term: Lung Diseases - ID: D000007674 - Term: Kidney Diseases ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00710879 **Brief Title:** Evaluation of a Multi-Purpose Solution **Official Title:** A Study to Evaluate the Clinical Performance of a Multi-Purpose Solution. #### Organization Study ID Info **ID:** 568 #### Organization **Class:** INDUSTRY **Full Name:** Bausch & Lomb Incorporated ### Status Module #### Completion Date **Date:** 2009-02 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2011-12-13 **Type:** ESTIMATED **Last Update Submit Date:** 2011-12-07 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2009-02 **Type:** ACTUAL #### Results First Post Date **Date:** 2011-02-08 **Type:** ESTIMATED **Results First Submit Date:** 2011-01-14 **Results First Submit QC Date:** 2011-01-14 #### Start Date **Date:** 2008-07 **Status Verified Date:** 2011-12 #### Study First Post Date **Date:** 2008-07-08 **Type:** ESTIMATED **Study First Submit Date:** 2008-06-30 **Study First Submit QC Date:** 2008-07-07 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Bausch & Lomb Incorporated #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** This study evaluates a multi-purpose solution when used on a daily wear basis by currently adapted soft contact lens wearers. ### Conditions Module **Conditions:** - Contact Lens Solutions **Keywords:** - Efficacy ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** SUPPORTIVE_CARE #### Enrollment Info **Count:** 180 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Multi-purpose solution administered to adapted FDA group I soft contact lens wearers and FDA group IV soft contact lens wearers. **Intervention Names:** - Device: Bausch & Lomb Multi-Purpose Solution **Label:** Multipurpose Solution **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Multipurpose Solution **Description:** Daily care for contact lenses. **Name:** Bausch & Lomb Multi-Purpose Solution **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Excellent = No bacterial infection suspected and no ocular pathogens detected and bacteria of normal flora \<0-103 CFU/mL. Good = No bacterial infection suspected and no ocular pathogens detected and bacteria of normal flora \<103-105 CFU/mL. Skeptical = Bacterial infection suspected and ocular pathogens detected and bacteria of normal flora ≥ 105 CFU/mL. No Efficacy = Bacterial infection definite and ocular pathogens detected and bacteria of normal flora ≥ 105 CFU/mL. Pathogens were H. aegyptius, H. influenzae, Moraxella spp., P. aeruginosa, S. pneumoniae, S. aureus, N. gonorrhoeae **Measure:** Antimicrobial Efficacy **Time Frame:** 2 weeks, 3 months #### Secondary Outcomes **Description:** Very Safe = No solution related AEs and no changes in lens properties related to the solution. Safe = No solution related AEs and slight change in lens properties related to the solution, but lens wear was continued. Skeptical = Solution related AEs were suspected and lens properties changed due to the solution and lens wear was discontinued. Not Safe = Solution related AEs were present and lens properties changed due to the solution and lens wear was discontinued. **Measure:** Solution Related AE's and Lens Changes **Time Frame:** 3 months, 6 months **Description:** The Utility was determined based on the results of the efficacy and safety evaluations. **Measure:** Solution Utlility **Time Frame:** 3 months, 6 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Is an adapted Group I or Group IV soft contact lens wearer and agrees to wear the study lenses on a daily wear basis. * Must be able and willing to comply with all treatment and follow-up study procedures. * Must have a clear central cornea. * VA correctable to 0.3 LogMar or better (driving vision) Exclusion Criteria: * Systemic disease affecting ocular health. * Using systemic or topical medications. * Wear monovision, multifocal or toric contact lenses. * Any grade 2 or greater slit lamp findings. * Pregnancy **Healthy Volunteers:** True **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Torrance **Country:** United States **Facility:** Dr. Nicholas Marsico **State:** California **Zip:** 90505 #### Overall Officials **Official 1:** **Affiliation:** Bausch & Lomb Incorporated **Name:** Bev Barna **Role:** STUDY_DIRECTOR ## Derived Section ### 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 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Multi-purpose Solution and Optima 38 Lenses **Description:** Multi-purpose solution when used on a daily wear basis with United States (US) Food and Drug Administration (FDA) Group I contact lenses (Optima 38) with no scheduled replacement. Participants were followed for 3 months. **ID:** EG000 **Other Num at Risk:** 120 **Serious Number At Risk:** 120 **Title:** Multi-purpose Solution and Optima 38 Lenses **Group ID:** EG001 **Title:** Multi-Purpose Solution and Acuvue 2 Lenses **Description:** B\&L Multi-Purpose Solution when used on a daily wear basis with United States (US) Food and Drug Administration (FDA) Group IV contact lenses (Acuvue 2) with scheduled replacements every 2 weeks. Participants were followed for 6 months. **ID:** EG001 **Other Num at Risk:** 240 **Serious Number At Risk:** 240 **Title:** Multi-Purpose Solution and Acuvue 2 Lenses **Frequency Threshold:** 5 **Time Frame:** Adverse events were evaluated at every visit. Group 1, 3 months and group 4, 6 months. ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 60 **Group ID:** BG001 **Value:** 117 **Group ID:** BG002 **Value:** 177 **Units:** Participants ### Group **ID:** BG000 **Title:** Multi-purpose Solution and Optima 38 Lenses **Description:** Multi-purpose solution when used on a daily wear basis with United States (US) Food and Drug Administration (FDA) Group I contact lenses (Optima 38) with no scheduled replacement. Participants were followed for 3 months. ### Group **ID:** BG001 **Title:** Multi-Purpose Solution and Acuvue 2 Lenses **Description:** B\&L Multi-Purpose Solution when used on a daily wear basis with United States (US) Food and Drug Administration (FDA) Group IV contact lenses (Acuvue 2) with scheduled replacements every 2 weeks. Participants were followed for 6 months. ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 11.0 **Value:** 60 #### Measurement **Group ID:** BG001 **Spread:** 10.3 **Value:** 117 #### Measurement **Group ID:** BG002 **Value:** 177 **Class Title:** Aged 18-64 ### Measure #### Measurement **Group ID:** BG000 **Value:** 37 #### Measurement **Group ID:** BG001 **Value:** 75 #### Measurement **Group ID:** BG002 **Value:** 112 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 23 #### Measurement **Group ID:** BG001 **Value:** 42 #### Measurement **Group ID:** BG002 **Value:** 65 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 11 #### Measurement **Group ID:** BG002 **Value:** 11 **Class Title:** 1 Grandparent #### Measurement **Group ID:** BG000 **Value:** 7 #### Measurement **Group ID:** BG001 **Value:** 18 #### Measurement **Group ID:** BG002 **Value:** 25 **Class Title:** 2 Grandparents #### Measurement **Group ID:** BG000 **Value:** 4 #### Measurement **Group ID:** BG001 **Value:** 6 #### Measurement **Group ID:** BG002 **Value:** 10 **Class Title:** 3 Grandparents #### Measurement **Group ID:** BG000 **Value:** 49 #### Measurement **Group ID:** BG001 **Value:** 82 #### Measurement **Group ID:** BG002 **Value:** 131 **Class Title:** 4 Grandparents **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Parameter Type:** NUMBER **Title:** Age, Customized **Unit of Measure:** Participants ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 3 **Description:** Participants with Grandparents Born in Japan **Parameter Type:** NUMBER **Title:** Participants with Grandparents Born in Japan **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement **Other Details:** All study data generated as a result of this study will be regarded as confidential, until appropriate analysis and review by the Sponsor or its designee are completed. The results of the study may be published or presented by the Investigator(s) after the review by, and in consultation and agreement with the Sponsor, and such that confidential or proprietary information is not disclosed. **Restriction Type:** OTHER **Restrictive Agreement:** True ### Point of Contact **Email:** [email protected] **Organization:** Bausch & Lomb Incorporated **Phone:** (585) 338-6439 **Title:** Joseph Barr, OD, MS ## 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:** 114 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 219 **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:** 3 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 118 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 238 **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:** 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 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 112 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 225 **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:** 5 **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:** 4 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Excellent = No bacterial infection suspected and no ocular pathogens detected and bacteria of normal flora \<0-103 CFU/mL. Good = No bacterial infection suspected and no ocular pathogens detected and bacteria of normal flora \<103-105 CFU/mL. Skeptical = Bacterial infection suspected and ocular pathogens detected and bacteria of normal flora ≥ 105 CFU/mL. No Efficacy = Bacterial infection definite and ocular pathogens detected and bacteria of normal flora ≥ 105 CFU/mL. Pathogens were H. aegyptius, H. influenzae, Moraxella spp., P. aeruginosa, S. pneumoniae, S. aureus, N. gonorrhoeae **Parameter Type:** NUMBER **Population Description:** All eligible, dispensed eyes with cultures taken within window. Group I subjects were cultured at the 3-Month Visit. Group IV subjects were cultured at the 2-week Follow-up visit. **Reporting Status:** POSTED **Time Frame:** 2 weeks, 3 months **Title:** Antimicrobial Efficacy **Type:** PRIMARY **Type Units Analyzed:** eyes **Unit of Measure:** Eyes ##### Group **Description:** Multi-purpose solution when used on a daily wear basis with United States (US) Food and Drug Administration (FDA) Group I contact lenses (Optima 38) with no scheduled replacement. Participants were followed for 3 months. **ID:** OG000 **Title:** Multi-purpose Solution and Optima 38 Lenses ##### Group **Description:** B\&L Multi-Purpose Solution when used on a daily wear basis with United States (US) Food and Drug Administration (FDA) Group IV contact lenses (Acuvue 2) with scheduled replacements every 2 weeks. Participants were followed for 6 months. **ID:** OG001 **Title:** Multi-Purpose Solution and Acuvue 2 Lenses #### Outcome Measure 2 **Description:** Very Safe = No solution related AEs and no changes in lens properties related to the solution. Safe = No solution related AEs and slight change in lens properties related to the solution, but lens wear was continued. Skeptical = Solution related AEs were suspected and lens properties changed due to the solution and lens wear was discontinued. Not Safe = Solution related AEs were present and lens properties changed due to the solution and lens wear was discontinued. **Parameter Type:** NUMBER **Population Description:** All Eligible, Dispensed Eyes **Reporting Status:** POSTED **Time Frame:** 3 months, 6 months **Title:** Solution Related AE's and Lens Changes **Type:** SECONDARY **Type Units Analyzed:** eyes **Unit of Measure:** Eyes ##### Group **Description:** Multi-purpose solution when used on a daily wear basis with United States (US) Food and Drug Administration (FDA) Group I contact lenses (Optima 38) with no scheduled replacement. Participants were followed for 3 months. **ID:** OG000 **Title:** Multi-purpose Solution and Optima 38 Lenses ##### Group **Description:** B\&L Multi-Purpose Solution when used on a daily wear basis with United States (US) Food and Drug Administration (FDA) Group IV contact lenses (Acuvue 2) with scheduled replacements every 2 weeks. Participants were followed for 6 months. **ID:** OG001 **Title:** Multi-Purpose Solution and Acuvue 2 Lenses #### Outcome Measure 3 **Description:** The Utility was determined based on the results of the efficacy and safety evaluations. **Parameter Type:** NUMBER **Population Description:** All Eligible, Dispensed Eyes **Reporting Status:** POSTED **Time Frame:** 3 months, 6 months **Title:** Solution Utlility **Type:** SECONDARY **Type Units Analyzed:** Eyes **Unit of Measure:** Eyes ##### Group **Description:** Multi-purpose solution when used on a daily wear basis with United States (US) Food and Drug Administration (FDA) Group I contact lenses (Optima 38) with no scheduled replacement. Participants were followed for 3 months. **ID:** OG000 **Title:** Multi-purpose Solution and Optima 38 Lenses ##### Group **Description:** B\&L Multi-Purpose Solution when used on a daily wear basis with United States (US) Food and Drug Administration (FDA) Group IV contact lenses (Acuvue 2) with scheduled replacements every 2 weeks. Participants were followed for 6 months. **ID:** OG001 **Title:** Multi-Purpose Solution and Acuvue 2 Lenses ### Participant Flow Module #### Group **Description:** Multi-purpose solution when used on a daily wear basis with United States (US) Food and Drug Administration (FDA) Group I contact lenses (Optima 38) with no scheduled replacement. Participants were followed for 3 months. **ID:** FG000 **Title:** Multi-purpose Solution and Optima 38 Lenses #### Group **Description:** B\&L Multi-Purpose Solution when used on a daily wear basis with United States (US) Food and Drug Administration (FDA) Group IV contact lenses (Acuvue 2) with scheduled replacements every 2 weeks. Participants were followed for 6 months. **ID:** FG001 **Title:** Multi-Purpose Solution and Acuvue 2 Lenses #### Period **Title:** Overall Study ##### Withdraw **Type:** Lost to Follow-up ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ##### Withdraw **Type:** Protocol Violation ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 3 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 60 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 117 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 58 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 113 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 2 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 4 **Pre-Assignment Details:** This study focused on participants of Japanese descent (at least one grandparent born in Japan) for a minimum of 20% of total enrollment. 180 participants were enrolled, 3 participants were ineligible at screening, 177 participants were eligible at the start of the study. **Recruitment Details:** First participant was enrolled in this study on 07/07/2008 and last subject exited on 02/02/2009. 180 participants were enrolled at 9 investigative sites in the United States. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT05874479 **Acronym:** AirPressureNYC **Brief Title:** Reducing AIR Pollution Exposure to Lower Blood PRESSURE Among New York City Public Housing Residents **Official Title:** Reducing AIR Pollution Exposure to Lower Blood PRESSURE Among New York City Public Housing Residents #### Organization Study ID Info **ID:** 23-00517 #### Organization **Class:** OTHER **Full Name:** NYU Langone Health ### Status Module #### Completion Date **Date:** 2028-03 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-12-27 **Type:** ACTUAL **Last Update Submit Date:** 2023-12-20 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2027-09 **Type:** ESTIMATED #### Start Date **Date:** 2023-12-20 **Type:** ACTUAL **Status Verified Date:** 2023-12 #### Study First Post Date **Date:** 2023-05-25 **Type:** ACTUAL **Study First Submit Date:** 2023-05-12 **Study First Submit QC Date:** 2023-05-12 ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** National Heart, Lung, and Blood Institute (NHLBI) #### 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:** True ### Description Module **Brief Summary:** Fine particulate matter \<2.5 µm (PM2.5) air pollution is the fifth leading risk factor for global mortality, with the largest portion of deaths due to cardiovascular disease (CVD). While several mechanisms are responsible, PM2.5-induced elevations in blood pressure (BP) may be relevant. Indoor portable air cleaners (PACs) are a novel approach to reduce exposure to PM2.5 and potentially lower blood pressure. The current study is being conducted to provide evidence that PACs reduce PM2.5 exposure and lower systolic blood pressure (SBP) in key patient populations. ### Conditions Module **Conditions:** - Blood Pressure **Keywords:** - self-measured morning home systolic blood pressure (AM H-SBP) ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - INVESTIGATOR **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 440 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Using a double-blind randomized process, active PACs (with HEPA filters inside) will be assembled and placed in the bedroom of study participants. Initiation of the PAC devices will occur at baseline on Day 0. After completion of enrollment, consent and the placement of PACs, electricity monitors, and PurpleAir monitoring, the PAC will be turned on. Participants will be instructed to keep the PACs on in bedrooms during the study duration, closing bedroom doors at night and during the day, when possible. The PAC will run for 24 hours on Day 0 of the study protocol before the 30-day treatment period begins. A kilowatt meter will be installed with the PAC to monitor electricity usage as a measure of adherence. **Intervention Names:** - Device: Active PAC **Label:** Active Portable Air Cleaner (PAC) **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Using a double-blind randomized process, sham PACs (with no filters inside) will be assembled and placed in the bedroom of study participants. Initiation of the PAC devices will occur at baseline on Day 0. After completion of enrollment, consent and the placement of PACs, electricity monitors, and PurpleAir monitoring, the PAC will be turned on. Participants will be instructed to keep PACs on in bedrooms during the study duration, closing bedroom doors at night and during the day, when possible. The PAC will run for 24 hours on Day 0 of the study protocol before the 30-day treatment period begins. A kilowatt meter will be installed with the PAC to monitor electricity usage as a measure of adherence. **Intervention Names:** - Device: Sham PAC **Label:** Sham PAC **Type:** SHAM_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Active Portable Air Cleaner (PAC) **Description:** The active PAC will contain HEPA filters inside the device. **Name:** Active PAC **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - Sham PAC **Description:** The sham PAC will contain no HEPA filters inside the device. **Name:** Sham PAC **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Description:** Participants measure HBP every day between 6-9 am. **Measure:** Average Self-Measured Morning Home Systolic Blood Pressure (AM H-SBP) over 30 Days **Time Frame:** Up to Day 30 #### Secondary Outcomes **Description:** Participants measure HBP every day between 6-9 am. **Measure:** Average Self-Measured Morning Home Systolic Blood Pressure (AM H-SBP) over 90 Days **Time Frame:** Up to Day 90 **Description:** Participants measure HBP every day between 6-9 am. **Measure:** Average Self-Measured Morning Home Systolic Blood Pressure (AM H-SBP) over 180 Days **Time Frame:** Up to Day 180 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * self-reported nonsmokers living in a nonsmoking household. * adults living with hypertension (HTN) from NYC public housing. Exclusion Criteria: * History of major known arrhythmias (e.g. atrial flutter or fibrillation, ventricular tachycardia). * Screening systolic BP ≥160 mm Hg or diastolic BP ≥100 mm Hg (i.e. severe hypertension by the 2017 ACC/AHA BP guideline). * A change in drug regimen in the prior 2 weeks or a planned change in drug regimen during the first 30 days for those taking antihypertensive medication. * Current smoking or living with an active smoker who smokes indoors * Planned travel out of NYC for ≥2 weeks in next 6 months * Incarcerated * Pregnant * Unable/unwilling to consent * Established cardiovascular disease * End-stage renal disease (chronic kidney disease stage IV or on dialysis) * Barrier to technology use (e.g., visual or hearing impairment) * Lung disease requiring oxygen * Cancer receiving treatment **Maximum Age:** 100 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Jonathan Newman **Phone:** (212) 263-9393 **Role:** CONTACT #### Locations **Location 1:** **City:** New York **Country:** United States **Facility:** NYU Langone Health **State:** New York **Status:** RECRUITING **Zip:** 10016 #### Overall Officials **Official 1:** **Affiliation:** NYU Langone Health **Name:** Jonathan Newman **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Access Criteria:** The investigator who proposed to use the data will be granted access upon reasonable request. Requests should be directed to [email protected]. To gain access, data requestors will need to sign a data access agreement. **Description:** The de-identified participant data from the final research dataset used in the published manuscript will be shared upon reasonable request beginning 9 months and ending 36 months following article publication or as required by a condition of awards and agreements supporting the research provided the investigator who proposes to use the data executes a data use agreement with NYU Langone Health. Requests may be directed to: [email protected]. The protocol and statistical analysis plan will be made available on Clinicaltrials.gov only as required by federal regulation or as a condition of awards and agreements supporting the research. **Info Types:** - STUDY_PROTOCOL - SAP **IPD Sharing:** YES **Time Frame:** Beginning 9 months and ending 36 months following article publication or as required by a condition of awards and agreements supporting the research. ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014652 - Term: Vascular Diseases - ID: D000002318 - Term: Cardiovascular Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC14 - Name: Heart and Blood Diseases ### Condition Browse Module - Browse Leaves - ID: M27583 - Name: Systolic Murmurs - Relevance: LOW - As Found: Unknown - ID: M10072 - Name: Hypotension - Relevance: HIGH - As Found: Lower Blood Pressure - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000007022 - Term: Hypotension ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04474379 **Acronym:** PMT2 **Brief Title:** Everyday Memory Impairment in PD-related Cognitive Decline **Official Title:** Prospective Memory Impairment in Parkinson Disease-related Cognitive Decline: Intervention and Mechanisms #### Organization Study ID Info **ID:** 202007054 #### Organization **Class:** OTHER **Full Name:** Washington University School of Medicine ### Status Module #### Completion Date **Date:** 2026-09-01 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-05-10 **Type:** ACTUAL **Last Update Submit Date:** 2024-05-08 **Overall Status:** ENROLLING_BY_INVITATION #### Primary Completion Date **Date:** 2026-09-01 **Type:** ESTIMATED #### Start Date **Date:** 2021-11-29 **Type:** ACTUAL **Status Verified Date:** 2024-05 #### Study First Post Date **Date:** 2020-07-16 **Type:** ACTUAL **Study First Submit Date:** 2020-07-10 **Study First Submit QC Date:** 2020-07-14 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Washington University School of Medicine #### Responsible Party **Investigator Affiliation:** Washington University School of Medicine **Investigator Full Name:** Erin Foster **Investigator Title:** Assistant Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** The investigators will aim enroll participants into our study within 3-6 months after their parent study visit so the investigators can utilize some key data points (e.g. PD-MCI diagnosis, rs-fcMRI data) from that study. PD participants will participate in a single-blind RCT with two treatment arms: process training and strategy training (Fig 4). They will complete pre-training assessment (Pre), be randomized to treatment arm (1:1 ratio stratified by sex), and then complete 8 training sessions over an 8-week period. They will return within 1 week for post-training assessment (Post) and then will complete Follow-up (FU) assessments via web or mailed survey 3 and 6 months after training ends. They will complete a 12mo FU assessment in person in conjunction with their annual parent study visit. HC participants will complete prospective memory assessment at one time point coinciding with (or within 3-6 months of) their parent study visit to determine whether any relationships observed between rs-fcMRI data and prospective memory are specific to PD. **Detailed Description:** Parkinson disease (PD) causes cognitive deficits that can impair instrumental activities of daily living function and quality of life (QOL) even in the absence of dementia. Currently, there are no treatments to address this pressing burden and, even worse, some medical treatments for PD can exacerbate cognitive deficits. For these reasons, behavioral interventions that attenuate the negative functional consequences of cognitive decline in PD and thus potentially delay the onset of dementia are in high demand. An intervention that enables people with PD to remember to perform necessary and meaningful daily activities could improve or maintain their independence, disease self-management, participation in society, and reduce caregiver burden. The primary goal of this study is to determine the efficacy of such an intervention. Importantly, in doing so, the investigators will directly address a critical point of contention in cognitive intervention research: Which approach (process or strategy training) is the best option for producing functionally-relevant benefits for people with PD? Additionally, the study team will investigate behavioral and neurobiological correlates of prospective memory in PD, including individual characteristics that may predict treatment response. This work will lead to a targeted and tailored biobehavioral intervention that optimizes function, promotes QOL, and improves the long-term management of a common chronic neurological condition. It will move the field of rehabilitation forward by providing valuable theoretical and practical information related to the type of cognitive intervention approach that is most appropriate for people with PD. This then will stimulate and justify the channeling of future research efforts and funding toward further development, testing, dissemination, and implementation of said approach. Primary Outcomes: Aim 1. Laboratory prospective memory performance - The Virtual Week test will be administered to PD participants at Pre and Post to assess near transfer of training objectively (primary endpoint). It will also be administered to PD participants at 12mo FU to explore long term training effects (secondary endpoint) and to HC participants at Pre to investigate neural mechanisms of prospective memory performance (Aim 3). It is a computerized board game that simulates daily life and real-world prospective memory challenges. Each circuit represents one day in which the participant completes time-appropriate activities and makes choices about them. Embedded in each day are 8 prospective memory tasks (4 event, 4 time). Participants complete a practice day and then 4 test days; equivalent versions are counterbalanced across testing sessions. The main outcome variable is prospective memory accuracy, defined as the proportion of correct prospective memory responses (32 total: 16 event, 16 time). To further explore the effects of time-based training, the investigators will record strategic clock-checking behavior by requiring participants to click a button to reveal the virtual time of day. Aim 2. Reported everyday prospective memory: (2a) General everyday prospective memory function - The Prospective and Retrospective Memory Questionnaire (PRMQ)will be administered via web-based or mailed survey to PD participants and informants at Pre, Post and 3mo FU to assess far transfer of training (primary endpoint), to PD participants and informants at 6mo and 12mo FU to explore long term training effects (secondary endpoint), and to HC participants at Pre to determine neural mechanisms of everyday prospective memory (Aim 3). The PRMQ is the most widely used questionnaire for everyday prospective memory. Participants rate the frequency of 8 everyday prospective memory failures (1=Never, 5=Very Often), item scores are summed, and higher scores indicate worse everyday prospective memory. The PRMQ includes environment- and self-cued subscales (4 items each) that parallel the event- and time-based task distinction, respectively. (2b) Personalized real-life prospective memory tasks - The Bangor Goal-Setting Interview (BGSI)offers a standardized means of eliciting individual goals and rating goal attainment over time and has been successfully used in cognitive rehabilitation RCTs with older adults, including those with mild to moderate dementia. During the first two training sessions, after an explanation of the prospective memory task types (session 1: event, session 2: time), participants will complete the BGSI with the trainer to identify and set goals for 3-6 real-life prospective memory tasks they anticipate having to complete over the training period. They and their informants will rate their Pre and Post attainment of these goals on a 10-point scale (1=never remember to do; 10=always remember to do). Goal attainment ratings are averaged to yield mean attainment scores. Additional measures: Aims 3 \& 4. Neural correlates of prospective memory and treatment response - Rs-fcMRI data will be collected and managed under the auspices of the parent study at Pre. Briefly, MRIs will be completed OFF PD medications on a 3T Siemens Prisma scanner with a 20 channel head coil and include up to 6 BOLD rs-fcMRI scans (416 volumes/run, TE=26.6ms, TR=800ms, FOV= 213mm, flip angle=61°, 3mm3 voxels; multiband factor=4; 5:39min) during fixation (eyes open). More details, including rigorous quality control methods, are in references. The investigators will use a network-level analysis approach using standard nodes from canonical networks. Our primary variables of interest will be intranetwork integrity scores, calculated based on the cross-correlations of each node within the network, for the following cognitive networks: DMN, CON, FPN, PMN, and MTL. Alternate approaches may include seed-based analyses (e.g. DLPFC) and Object Oriented Data Analysis followed by post-hoc identification of significant networks. Aim 4. Behavioral predictors of treatment response - Based on our pilot RCT (Prelim Data 2)1, The investigators will administer the Credibility and Expectancy Questionnaire (CEQ) and Beck Depression Inventory-II (BDI-II) at Pre as potential motivational predictors of treatment response. The CEQ will be administered at the end of training session 2, so participants have some knowledge of the intervention on which to base their perceptions of credibility and expectancy. The investigators will utilize the extensive neuropsychological data collected at Pre in the parent study to assess potential cognitive predictors of treatment response. Our primary cognitive variables will be the domain z-scores for Memory and Executive Function, computed as the average of standardized scores from measures assessing that domain. Other - PD (and normal cognition for HCs) will be determined via MDS Level II diagnostic criteria in the parent study based on a comprehensive neuropsychological assessment battery and in-depth clinical interviews (CDR). In addition to the main measures of interest described above, the investigators will collect other data to characterize participants, as covariates, or as secondary outcomes (Table 3). This will permit exploration of the influence of sex or other relevant biological variables (e.g. co-morbidities, medication) and broader functional outcomes. The investigators will obtain much of this data from the parent study. ### Conditions Module **Conditions:** - Parkinson Disease - Mild Cognitive Impairment ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** Participants will be randomized to treatment arm (1:1 ratio, stratified by sex and Clinical Dementia Rating (CDR) Score 0 or 0.5): the strategy training or the process training. ##### Masking Info **Masking:** DOUBLE **Masking Description:** The participant and outcomes assessor will be blind to which cognitive training a participant will be randomized. **Who Masked:** - PARTICIPANT - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 120 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Consists of 8-90 minute sessions over 8 weeks. In sessions 1 and 2, in addition to teaching about event- and time-based tasks, the therapist teaches the participant specific strategies for each type of task (implementation intentions for event-based and strategic clock-checking for time-based) and instructs in their use before and during the training games. In sessions 3-8, the tester tells the participant s/he will be practicing both types of tasks in the training games and can support the participant's strategy use if needed. Feedback on accuracy and strategy use are provided after each training game. After completing the training games, the therapist and participant discuss how the strategies can be applied to the participant's real-life prospective memory goals, and the therapist helps the participant develop written action plans to do so. Plans and goals are reviewed and modified, if necessary, at each session. **Intervention Names:** - Behavioral: Strategy Training (Time- and Event- Based) **Label:** Strategy Training **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Consists of 8, 90 minute sessions over 8 weeks. In sessions 1 and 2, the therapist teaches the participant about event- and time-based prospective memory tasks, respectively. In sessions 3-8, the tester tells the participant that s/he will be practicing both types of tasks in the training games. In all sessions, the participant completes the training games with no strategy instruction from the therapist. Feedback on accuracy is provided after each training game. This is typical of a process training approach and expects that practice of the training tasks will improve prospective memory ability per se or that participants will develop effective strategies for completing prospective memory tasks on their own. At the end of each session, the therapist reminds the participant of his/her real-life prospective memory goals, provides a handout that lists the goals, and instructs the participant to try to complete them as intended. Goals are reviewed and modified if necessary. **Label:** Process Training **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Strategy Training **Description:** In time-based training, strategic clock-checking behavior is taught by requiring participants to click a button to reveal the virtual time of day. Event-based training involves implementation intention methods. **Name:** Strategy Training (Time- and Event- Based) **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** A computer-based prospective memory test that simulates a real day where a person has to remember to do tasks. **Measure:** Change in Virtual Week Computer-based Memory Test performance after 5-weeks and 12-month post-intervention. **Time Frame:** Administered at baseline, pre-treatment session; One week post-treatment (approximately five weeks after the baseline test); and 12-months post-strategy training sessions. **Description:** Participant-reported everyday prospective memory **Measure:** Change in The Prospective and Retrospective Memory Questionnaire scores after 5-weeks, 3-months, 6-months, and 12-month post-intervention. **Time Frame:** Administered at baseline, pre-treatment session; One week post-treatment (approximately five weeks after the baseline test); then 3-, 6- and 12-months post-completion of strategy training sessions. **Description:** A standardized measure that elicits individual goals and rating goal attainment over time **Measure:** Change in Bangor Goal Setting Interview goal attainment after 5-weeks and 12-month post-intervention. **Time Frame:** Administered at baseline, pre-treatment session; One week post-treatment (approximately five weeks after the baseline test); and 12-months post-strategy training sessions. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Males and females over age 50 who meet criteria for typical idiopathic PD * Hoehn \& Yahr stage I-III, * Treated with levodopa/carbidopa * Have subjective memory complaints (as identified in phone screen), * Have an informant to complete relevant ratings, * Medications should be stable for 4 weeks prior with no changes planned during the treatment portion of the study (Pre to Post); changes over the follow-up period will be tracked and accounted for as appropriate. Exclusion Criteria: * Dementia according to MDS criteria or MoCA score \<21. * Other neurological disorders (e.g. stroke, seizures), brain surgery, severe systemic diseases, major psychiatric disorder or history of psychotic symptoms (e.g. schizophrenia, bipolar disorder, delusions, hallucinations), or drug abuse. * Treatment with medications that interfere with cognition (e.g. anticholinergics). * Any other condition that would interfere with participation (e.g., non-English speaking, significant current depression). * Psychiatric conditions/ symptoms that are common in PD (e.g. anxiety, depression) are allowed if they are deemed insufficient to interfere with participation. **Minimum Age:** 50 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Saint Louis **Country:** United States **Facility:** Washington University School of Medicine **State:** Missouri **Zip:** 63110 #### Overall Officials **Official 1:** **Affiliation:** Washington University School of Medicine **Name:** Erin Foster, PhD, OTD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Access Criteria:** Per individual request to PI. **Description:** All collected IPD that underlie results in a publication. Unpublished data may be shared at the PI's discretion. All shared data will be deidentified. **Info Types:** - STUDY_PROTOCOL - SAP - ICF **IPD Sharing:** YES **Time Frame:** Starting 3 months after publication ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020734 - Term: Parkinsonian Disorders - ID: D000001480 - Term: Basal Ganglia Diseases - ID: D000001927 - Term: Brain Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000009422 - Term: Nervous System Diseases - ID: D000009069 - Term: Movement Disorders - ID: D000080874 - Term: Synucleinopathies - ID: D000019636 - Term: Neurodegenerative Diseases - ID: 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 - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases ### Condition Browse Module - Browse Leaves - ID: M29705 - Name: Cognitive Dysfunction - Relevance: HIGH - As Found: Cognitive Decline - ID: M13213 - Name: Parkinson Disease - Relevance: HIGH - As Found: Parkinson's Disease - ID: M22494 - Name: Parkinsonian Disorders - Relevance: LOW - As Found: Unknown - ID: M25603 - Name: Ganglion Cysts - Relevance: LOW - As Found: Unknown - ID: M16358 - Name: Synovial Cyst - Relevance: LOW - As Found: Unknown - ID: M4774 - Name: Basal Ganglia Diseases - Relevance: LOW - As Found: Unknown - ID: M5204 - Name: Brain Diseases - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: M12029 - Name: Movement Disorders - Relevance: LOW - As Found: Unknown - ID: M2217 - Name: Synucleinopathies - Relevance: LOW - As Found: Unknown - ID: M21558 - Name: Neurodegenerative Diseases - Relevance: LOW - As Found: Unknown - ID: 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: D000010300 - Term: Parkinson Disease - ID: D000060825 - Term: Cognitive Dysfunction ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01720979 **Brief Title:** TBI Project Amsterdam **Official Title:** Attention, Learning and Behaviour Following Traumatic Brain Injury in Children #### Organization Study ID Info **ID:** NL37226.029.11 #### Organization **Class:** OTHER **Full Name:** VU University of Amsterdam ### Status Module #### Completion Date **Date:** 2015-09 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2015-12-03 **Type:** ESTIMATED **Last Update Submit Date:** 2015-12-02 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2015-09 **Type:** ACTUAL #### Start Date **Date:** 2011-11 **Status Verified Date:** 2015-12 #### Study First Post Date **Date:** 2012-11-02 **Type:** ESTIMATED **Study First Submit Date:** 2012-10-25 **Study First Submit QC Date:** 2012-11-01 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Amsterdam UMC, location VUmc **Class:** OTHER **Name:** Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA) #### Lead Sponsor **Class:** OTHER **Name:** Marsh Königs #### Responsible Party **Investigator Affiliation:** VU University of Amsterdam **Investigator Full Name:** Marsh Königs **Investigator Title:** MSc **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Traumatic Brain Injury (TBI) is the world leading cause of acquired brain injury. Literature suggests a pivotal role for attentional functioning in neurocognitive and behavioural consequences of paediatric TBI. Limitations of traditional neuropsychological measures of attentional functioning have interfered with identification of the effect of paediatric TBI on attentional networks so far. Moreover, the associations between attentional networks, learning abilities, academic performance and behavioural and emotional problems following paediatric TBI are yet to be explored. **Detailed Description:** Background: Traumatic brain injury (TBI) is the world leading cause of disability in children (Winslade, 1998), causing deficits in motor function, neurocognition and adaptive behaviour (Anderson, 2001). Literature shows that age at injury is inversely related to the magnitude of deficits following TBI, highlighting the vulnerability of children for the effects of TBI. The neurocognitive consequences of paediatric TBI have primarily been characterized by impairments in speed of information processing, attentional functioning and learning (Babikian \& Asarnow, 2009; Catroppa \& Anderson, 2009), interfering with typical neurocognitive development. We aim at elucidating the effects of TBI on neurocognitive function and investigate the relations between neurocognitive deficits, academic achievement and emotional and behavioural function, in order to improve our understanding of the post-injury functioning of children that have suffered TBI. Methods: Patients with TBI will be compared to a control group consisting of orthopedically injured patients. Orthopaedic control (OC) groups offer a better comparison to TBI patients than typically developing children by controlling for TBI risk factors related to neurocognition (e.g. Attention Deficit Hyperactivity Disorder, socioeconomic status), hospitalisation and the type of injuries other than brain injuries. Measures: Child's Orientation and Amnesia Test, Attention Network Test, Probabilistic Learning Test, Child Behaviour Checklist, Strengths \& Difficulties Questionnaire, Experimental Neurocognitive Test developed at the VU University and Pupil Monitoring System. ### Conditions Module **Conditions:** - Traumatic Brain Injury **Keywords:** - TBI - Traumatic Brain Injury - Children - Prognosis - Neurocognition ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** RETROSPECTIVE #### Enrollment Info **Count:** 90 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Children that were admitted to the hospital after traumatic injuries to body parts below the clavicles (traumatic control injury) and children that were admitted to the hospital after traumatic brain injury as diagnosed by a physician (TBI). **Label:** Patients with traumatic injuries ### Outcomes Module #### Primary Outcomes **Description:** FA/ADC values will be reported for selected region's of interest in children with TBI and children with traumatic control injuries. **Measure:** The white-matter integrity of children with TBI will be compared to children with traumatic control injuries **Time Frame:** on average 1-year post-TBI **Description:** Mean reaction time and accuracy will be reported for children with TBI and children with traumatic control injuries **Measure:** Performance on tests of attention will be compared between children with TBI and children with traumatic control injuries **Time Frame:** on average 1-year post-TBI #### Secondary Outcomes **Description:** Problem scores on several types of behavioural problems will be reported for children with TBI and children with traumatic control injuries **Measure:** Parental reports of behavioural regulation will be compared between children with TBI and children with traumatic control injuries **Time Frame:** on average 1-year post-TBI **Description:** Scores on standardised tests of academic achievement will be reported for children with TBI and children with traumatic control injuries **Measure:** Academic achievement will be compared between children with TBI and children with traumatic control injuries **Time Frame:** on average 1-year post-TBI **Description:** Reaction time and accuracy on computerised tests of reinforced learning will be reported for children with TBI and children with traumatic control injuries **Measure:** Performance on test of reinforced learning will be compared between children with TBI and children with traumatic control injuries **Time Frame:** on average 1-year post-TBI ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: TBI patients will be included if they * have parental written informed consent * provide written informed consent if aged over 11 years * are Dutch speaking * have a clinical diagnosis of TBI (closed head injury) * have a time post-injury that is longer than 1 month * are aged between 6-12 years. Trauma control patients will be included if they: * have parental written informed consent * provide written consent if aged over 11 years * are Dutch speaking * have suffered an orthopaedic injury * have no history of TBI * and are aged between 6-12 years. **Maximum Age:** 12 Years **Minimum Age:** 6 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - CHILD **Study Population:** Children that have been admitted to the hospital after sustaining trauma. ### Contacts Locations Module #### Locations **Location 1:** **City:** Eindhoven - Tilburg **Country:** Netherlands **Facility:** Libra rehabilitation centers Blixembosch Leijpark **State:** Brabant **Location 2:** **City:** Amsterdam **Country:** Netherlands **Facility:** Academic Medical Center **State:** Noord Holland **Zip:** 1081 **Location 3:** **City:** Amsterdam **Country:** Netherlands **Facility:** VU University of Amsterdam **State:** Noord Holland **Zip:** 1081 **Location 4:** **City:** Huizen **Country:** Netherlands **Facility:** Merem revalidatiecentra de Trappenberg **State:** Noord Holland **Zip:** 1081 **Location 5:** **City:** Rotterdam **Country:** Netherlands **Facility:** Erasmus MC **State:** Zuid Holland #### Overall Officials **Official 1:** **Affiliation:** VU University of Amsterdam **Name:** Jaap Oosterlaan, PhD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014947 - Term: Wounds and Injuries - ID: D000001927 - Term: Brain Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000009422 - Term: Nervous System Diseases - ID: D000006259 - Term: Craniocerebral Trauma - ID: D000020196 - Term: Trauma, Nervous System ### Condition Browse Module - Browse Branches - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC26 - Name: Wounds and Injuries - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M5207 - Name: Brain Injuries - Relevance: HIGH - As Found: Brain Injury - ID: M628 - Name: Brain Injuries, Traumatic - Relevance: HIGH - As Found: Traumatic Brain Injury - ID: M17685 - Name: Wounds and Injuries - Relevance: LOW - As Found: Unknown - ID: M5204 - Name: Brain Diseases - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: M9349 - Name: Craniocerebral Trauma - Relevance: LOW - As Found: Unknown - ID: M22023 - Name: Trauma, Nervous System - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001930 - Term: Brain Injuries - ID: D000070642 - Term: Brain Injuries, Traumatic ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05598879 **Acronym:** G-COR **Brief Title:** Global Cardio Oncology Registry **Official Title:** Global Cardio Oncology Registry #### Organization Study ID Info **ID:** IRB 22-211 #### Organization **Class:** OTHER **Full Name:** The Cleveland Clinic ### Status Module #### Completion Date **Date:** 2027-07-01 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2022-10-28 **Type:** ACTUAL **Last Update Submit Date:** 2022-10-27 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2025-07-01 **Type:** ESTIMATED #### Start Date **Date:** 2022-07-01 **Type:** ACTUAL **Status Verified Date:** 2022-10 #### Study First Post Date **Date:** 2022-10-28 **Type:** ACTUAL **Study First Submit Date:** 2022-10-25 **Study First Submit QC Date:** 2022-10-27 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** The Cleveland Clinic #### Responsible Party **Investigator Affiliation:** The Cleveland Clinic **Investigator Full Name:** Sadler,Diego **Investigator Title:** MD FACC Associate Professor of Medicine. CCLCM CWRU **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** G-COR is the first Global Prospective Cardio-Oncology Registry. It is a multinational, multicenter prospective observational cohort registry, with the goal of collecting clinical, laboratory, imaging, demographic, and socioeconomic data to identify risk factors associated with increased incidence of cancer therapy related cardiovascular toxicity (CTR-CVT) in different settings and to derive and validate risk scores for cardio oncology patients treated in different geographic locations throughout the world. **Detailed Description:** G-COR is the first Global Prospective Cardio-Oncology Registry. It is a multinational, multicenter prospective observational cohort registry, with the goal of collecting clinical, laboratory, imaging, demographic, and socioeconomic data to identify risk factors associated with increased incidence of cancer therapy related cardiovascular toxicity (CTR-CVT) in different settings and to derive and validate risk scores for cardio oncology patients treated in different geographic locations throughout the world. G-COR will involve the collaboration from 124 hospitals from 24 countries that completed survey with sites demographics. It will evaluate cardiovascular disease in three distinct populations of cancer patients (hematological malignancies: lymphomas, leukemias, multiple myeloma; breast cancer patients; and patients treated with check point inhibitors immunotherapy). G-COR will evaluate the cardiovascular impact of different cancer treatments in the above-described patients, and similarities and differences in diagnostic and treatment modalities as well as outcomes and the impact of socioeconomic factors and risk factors for toxicities in a large worldwide population. G-COR will study the impact of cancer in CV disease in cancer patients treated at academic centers as well as in patients treated at community hospitals, through a systematic prospective data collection in a global digital platform. G-COR is an IRB approved prospective registry, conducted with the logistical support of C5 Clinical Research Division and the Cardiovascular Outcomes Registries and Research (CORR) group at the Cleveland Clinic and have developed eCRFs with an extensive Red Cap Cloud platform. G-COR Executive, Scientific and topic committees are led by North American, European, Latin American, Australian and Asian representatives from both academic and community centers. The pilot phase of G-COR enrolls breast cancer patients only, and the global phase will include all three cohorts of patients (breast, Hem and ICIs). The investigators have started enrolling patients for G-COR pilot phase with US centers, and will start the global international phase in 2023. ### Conditions Module **Conditions:** - Breast Cancer - Hematologic Malignancy - Immune Checkpoint Inhibitor-Related Myocarditis - Cardiotoxicity - Cardiovascular Diseases **Keywords:** - cardiotoxicity - Cancer related cardiovascular diseases - prospective registry - international collaboration - social determinants of health - health care disparities - real world data - cardiology-oncology team work ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 5000 **Type:** ESTIMATED **Patient Registry:** True **Study Type:** OBSERVATIONAL **Target Duration:** 18 Months ### Arms Interventions Module #### Arm Group 1 **Description:** Patients with breast cancer who present for initial cardio-oncology consultation. Clinical follow up for 18 months. **Intervention Names:** - Other: anonymized data collection during programmed surveillance clinical follow up **Label:** Breast cancer #### Arm Group 2 **Description:** Patients with lymphomas, leukemias, multiple myeloma, and AL amyloidosis who present for initial cardio-oncology consultation. Clinical follow up for 18 months. **Intervention Names:** - Other: anonymized data collection during programmed surveillance clinical follow up **Label:** Hematological malignancies #### Arm Group 3 **Description:** Patients with any type of cancer treated with immune check point inhibitors who present for initial cardio-oncology consultation. Clinical follow for 18 months **Intervention Names:** - Other: anonymized data collection during programmed surveillance clinical follow up **Label:** Immune check point inhibitors ### Interventions #### Intervention 1 **Arm Group Labels:** - Breast cancer - Hematological malignancies - Immune check point inhibitors **Description:** anonymized data entry of demographic, clinical, imaging, laboratory, cancer treatment, and cardiovascular events into a RedCap Cloud platform **Name:** anonymized data collection during programmed surveillance clinical follow up **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Any new cardiac event occurring during or after cancer treatment **Measure:** Cardiotoxicity **Time Frame:** 18 months of prospective follow up **Description:** Heart failure, myocardial infarction, cardiac arrhythmias, syncope, coronary revascularization, heart transplant, cerebrovascular accident, peripheral arterial disease, hypertension, pulmonary hypertension. All events will be adjudicated according to standard clinical definitions. **Measure:** New cardiovascular events **Time Frame:** 18 months of prospective follow up **Description:** Death during or after cancer treatment, adjudicated to cardiovascular causes by treating physicians. **Measure:** Cardiovascular death. **Time Frame:** 18 months of prospective follow up ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * New cardio-oncology consultation for breast cancer patients, or * New cardio-oncology consultation for Hodgkin's or non-Hodgkin's lymphoma patients, or * New cardio-oncology consultation for acute or chronic leukemia patients, or * New cardio-oncology consultation for multiple myeloma or AL amyloidosis, or * New cardio-oncology consultations for immune check-point inhibitors cardiac evaluation. * All patients have to be 18 years old or older Exclusion Criteria: * Cardio-oncology patients who have previously had cardio-oncology evaluation and follow up by the investigators. * Minors less than 18 years old. * Inability or unwillingness to consent to participate **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** As above ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Diego Sadler, MD FACC **Phone:** 5613898833 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Rohit Moudgil, MD PhD **Phone:** 216-445-1932 **Role:** CONTACT #### Locations **Location 1:** **City:** Weston **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Diego Sadler, MD FACC - **Phone:** 561-389-8833 - **Role:** CONTACT **Country:** United States **Facility:** Cleveland Clinic Florida **State:** Florida **Status:** RECRUITING **Zip:** 33331 #### Overall Officials **Official 1:** **Affiliation:** The Cleveland Clinic **Name:** Diego Sadler, MD FACC **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Description:** To be established by the Scientific committee **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009369 - Term: Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000006402 - Term: Hematologic Diseases - ID: D000006331 - Term: Heart 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 - ID: D000009202 - Term: Cardiomyopathies ### 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 - Abbrev: BC15 - Name: Blood and Lymph Conditions - Abbrev: Rare - Name: Rare Diseases ### 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: M21314 - Name: Hematologic Neoplasms - Relevance: HIGH - As Found: Hematologic Malignancies - ID: M12157 - Name: Myocarditis - Relevance: HIGH - As Found: Myocarditis - ID: M9490 - Name: Hematologic Diseases - Relevance: LOW - As Found: Unknown - 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 - ID: M12154 - Name: Cardiomyopathies - Relevance: LOW - As Found: Unknown - ID: T4005 - Name: Myocarditis - Relevance: HIGH - As Found: Myocarditis ### Condition Browse Module - Meshes - ID: D000019337 - Term: Hematologic Neoplasms - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000066126 - Term: Cardiotoxicity - ID: D000009205 - Term: Myocarditis ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M2342 - Name: Immune Checkpoint Inhibitors - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06034379 **Brief Title:** Electronic Spectacles Versus Low Dose Atropine in Young Myopes **Official Title:** Kubota Corrective Spectacles and Low Dose Atropine for Slowing Myopic Progression in Taiwanese Children #### Organization Study ID Info **ID:** CP1:73200-TW #### Organization **Class:** INDUSTRY **Full Name:** Kubota Vision Inc. ### Status Module #### Completion Date **Date:** 2025-03 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-09-21 **Type:** ACTUAL **Last Update Submit Date:** 2023-09-18 **Overall Status:** NOT_YET_RECRUITING #### Primary Completion Date **Date:** 2025-03 **Type:** ESTIMATED #### Start Date **Date:** 2023-10 **Type:** ESTIMATED **Status Verified Date:** 2023-09 #### Study First Post Date **Date:** 2023-09-13 **Type:** ACTUAL **Study First Submit Date:** 2023-08-26 **Study First Submit QC Date:** 2023-09-11 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** China Medical University Hospital #### Lead Sponsor **Class:** INDUSTRY **Name:** Kubota Vision Inc. #### 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 is a bilateral, dispensing, masked, randomized clinical trial. Myopic children will be randomly assigned to one of the following: (1) Investigational clinical prototype (CP1) device without atropine, (2) Investigational CP1 device with daily instillation of 0.01% atropine, or (3) daily instillation of 0.01% atropine without use of the investigational CP1 device. Primary endpoint: Difference in the 12-month change of cycloplegic spherical refractive error and axial length between each of the three treatment groups. **Detailed Description:** The eSpectacle clinical prototype (CP1) device consists of a clear 15° central aperture and projects +9.00D defocused micro-LED lights onto the peripheral retina. In this pilot investigation, we aim to observe the ocular biometric and refractive changes following part-time wear of the clinical prototype device over a course of 12 months. This trial is bilateral, dispensing, masked, and randomized (stratified by age). Myopic children will be randomly assigned to one of the following: (1) CP1 device without atropine, (2) CP1 device with daily instillation of 0.01% atropine, or (3) daily instillation of 0.01% atropine without use of the CP1 device. A total of 45 subjects (15 per subgroup) are targeted to complete the study, which will consist of 8 visits: Screening, baseline/dispense, 1 week, 1 month, 3 months, 6 months, 9 months, and 12 months. Subjects will be enrolled from the patient population at the investigator site. The primary objective is to evaluate the effectiveness of the eSpectacle clinical prototype device with and without the use of 0.01 percent atropine for slowing the progression of myopia in Taiwanese children, by assessing changes in central axial length and cycloplegic autorefraction following at least 12 hours per week of wear over 12 months. To minimize bias, the primary outcome variables of cycloplegic autorefraction and axial length will be measured by a Masked Investigator. ### Conditions Module **Conditions:** - Myopia ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - INVESTIGATOR - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 45 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 15 subjects will be randomly assigned to use the eSpectacle Clinical Prototype (CP1) device 2 hours per day, at least 6 days per week. Standard single vision correction will be used during other waking hours. The eSpectacle clinical prototype (CP1) device consists of a clear 15° central aperture and projects +9.00D defocused micro-LED lights onto the peripheral retina. **Intervention Names:** - Device: eSpectacle Clinical prototype (CP1) device **Label:** Clinical prototype (CP1) device **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** 15 subjects will be randomly assigned to use the eSpectacle Clinical Prototype (CP1) device 2 hours per day, at least 6 days per week, in addition to nightly instillation of one drop of 0.01% atropine. Standard single vision correction will be used during other waking hours. The eSpectacle clinical prototype (CP1) device consists of a clear 15° central aperture and projects +9.00D defocused micro-LED lights onto the peripheral retina. Atropine is an anticholinergic medication which can be used for dilation and cycloplegia and has been evaluated for slowing the progression of myopia. **Intervention Names:** - Device: eSpectacle Clinical prototype (CP1) device - Drug: 0.01% atropine **Label:** CP1 and 0.01% atropine **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** 15 subjects will be randomly assigned to nightly instillation of one drop of 0.01% atropine without use of the eSpectacle clinical prototype (CP1) device. Standard single vision correction will be used during waking hours. Atropine is an anticholinergic medication which can be used for dilation and cycloplegia and has been evaluated for slowing the progression of myopia. **Intervention Names:** - Drug: 0.01% atropine **Label:** 0.01% atropine **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - CP1 and 0.01% atropine - Clinical prototype (CP1) device **Description:** The CP1 device consists of a clear 15° central aperture and projects +9.00D defocused micro-LED lights of approximately 4800 nits illumination to the peripheral retina. **Name:** eSpectacle Clinical prototype (CP1) device **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - 0.01% atropine - CP1 and 0.01% atropine **Description:** Atropine is a and anticholinergic medication which can be used for dilation and cycloplegia. **Name:** 0.01% atropine **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Difference in the changes of cycloplegic spherical refractive error between each of the three treatment groups. **Measure:** CSER **Time Frame:** 12 months **Description:** Difference in the changes of axial length between each of the three treatment groups. **Measure:** AL **Time Frame:** 12 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. The subject must be between 6 and 13 years of age (inclusive). 2. The subject must appear able and willing to adhere to the instructions set forth in the clinical protocol. 3. The guardian of a minor under 18 years old must read and sign the STATEMENT OF INFORMED CONSENT and be provided a copy of the form. The subject under 18 years old must be willing to assent to the STATEMENT OF INFORMED CONSENT. 4. Spherical component of refraction in the range of -0.50 to -10.00 DS in each eye. 5. Refractive cylinder less than or equal to 2.00 DC in each eye (minus cylinder format). 6. Best corrected distance VA of at least 20/25 in each eye. Exclusion Criteria: 1. Currently pregnant or lactating 2. History of severe dry eye, strabismus, or amblyopia 3. Any systemic disease (e.g., Sjögren's Syndrome), allergies, infectious disease (e.g., hepatitis, tuberculosis), contagious immunosuppressive diseases (e.g., HIV), autoimmune disease (e.g., diabetes, rheumatoid arthritis), or other diseases, by self-report, which are known to interfere with the participation in the study at the investigator's discretion. 4. Use of systemic or ocular medications (e.g., chronic steroid use) that are known to interfere with vision and ocular accommodation at the investigator's discretion. 5. Any previous, or planned (during the course of the study) ocular surgery (e.g., radial keratotomy, PRK, LASIK, strabismus surgery etc.) or orthokeratology 6. Any active ocular infection. 7. Any accommodative or binocular anomalies including amblyopia. 8. Any physical or mental developmental delay. 9. Any relevant ocular or systemic condition deemed unacceptable by the investigator that may negatively impact the subject's performance or ability to successfully complete the study (at the investigator's discretion) 10. Anisometropia greater than 1.50D **Healthy Volunteers:** True **Maximum Age:** 13 Years **Minimum Age:** 6 Years **Sex:** ALL **Standard Ages:** - CHILD ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012030 - Term: Refractive Errors - ID: D000005128 - Term: Eye Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC11 - Name: Eye Diseases ### Condition Browse Module - Browse Leaves - ID: M20559 - Name: Disease Progression - Relevance: LOW - As Found: Unknown - ID: M12168 - Name: Myopia - Relevance: HIGH - As Found: Myopia - ID: M14872 - Name: Refractive Errors - Relevance: LOW - As Found: Unknown - ID: M8271 - Name: Eye Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000009216 - Term: Myopia ### Intervention Browse Module - Ancestors - ID: D000000759 - Term: Adjuvants, Anesthesia - ID: D000000889 - Term: Anti-Arrhythmia Agents - ID: D000001993 - Term: Bronchodilator Agents - ID: D000001337 - Term: Autonomic Agents - ID: D000018373 - Term: Peripheral Nervous System Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018927 - Term: Anti-Asthmatic Agents - ID: D000019141 - Term: Respiratory System Agents - ID: D000009184 - Term: Mydriatics - ID: D000010276 - Term: Parasympatholytics - ID: D000018727 - Term: Muscarinic Antagonists - ID: D000018680 - Term: Cholinergic Antagonists - ID: D000018678 - Term: Cholinergic Agents - ID: D000018377 - Term: Neurotransmitter Agents - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action ### Intervention Browse Module - Browse Branches - Abbrev: AnArAg - Name: Anti-Arrhythmia Agents - Abbrev: AdjAn - Name: Adjuvants, Anesthesia - Abbrev: Resp - Name: Respiratory System Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: CNSDep - Name: Central Nervous System Depressants ### Intervention Browse Module - Browse Leaves - ID: M4590 - Name: Atropine - Relevance: HIGH - As Found: Autism - ID: M20760 - Name: Cholinergic Antagonists - Relevance: LOW - As Found: Unknown - ID: M4107 - Name: Anesthetics - Relevance: LOW - As Found: Unknown - ID: M4089 - Name: Adjuvants, Anesthesia - Relevance: LOW - As Found: Unknown - ID: M4213 - Name: Anti-Arrhythmia Agents - Relevance: LOW - As Found: Unknown - ID: M5269 - Name: Bronchodilator Agents - Relevance: LOW - As Found: Unknown - ID: M20963 - Name: Anti-Asthmatic Agents - Relevance: LOW - As Found: Unknown - ID: M21137 - Name: Respiratory System Agents - Relevance: LOW - As Found: Unknown - ID: M12139 - Name: Mydriatics - Relevance: LOW - As Found: Unknown - ID: M13189 - Name: Parasympatholytics - Relevance: LOW - As Found: Unknown - ID: M20801 - Name: Muscarinic Antagonists - Relevance: LOW - As Found: Unknown - ID: M20758 - Name: Cholinergic Agents - Relevance: LOW - As Found: Unknown - ID: M20504 - Name: Neurotransmitter Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000001285 - Term: Atropine ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06111079 **Brief Title:** Aspirin Discontinuation in High-Risk Pregnant Women of Preeclampsia **Official Title:** Aspirin Discontinuation at 28 or 36 Weeks' Gestation in High-Risk Pregnant Women of Preeclampsia A Randomized Clinical Trial #### Organization Study ID Info **ID:** [2023] Ethics Review NO.118 #### Organization **Class:** OTHER **Full Name:** The Third Affiliated Hospital of Guangzhou Medical University ### Status Module #### Completion Date **Date:** 2024-12-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-12-19 **Type:** ACTUAL **Last Update Submit Date:** 2023-12-18 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-06-01 **Type:** ESTIMATED #### Start Date **Date:** 2023-11-01 **Type:** ACTUAL **Status Verified Date:** 2023-12 #### Study First Post Date **Date:** 2023-11-01 **Type:** ACTUAL **Study First Submit Date:** 2023-10-23 **Study First Submit QC Date:** 2023-10-26 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** FANG HE #### Responsible Party **Investigator Affiliation:** The Third Affiliated Hospital of Guangzhou Medical University **Investigator Full Name:** FANG HE **Investigator Title:** Chief Physician/Professor of the Obstetrics Department **Type:** SPONSOR_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:** Low-dose aspirin (LDA) is considered to be the most effective agent to prevent preeclampsia (PE). At present, there is little exploration about the timing of aspirin discontinuation. Most international guidelines default until 36 weeks of gestation or delivery. China Guideline (2020) recommended that aspirin should be preventively used until 26-28 weeks of gestation, but there was little direct evidence. According to the two-stage theory, placental dysplasia before 28 weeks of gestation is the key to developing PE, the significance of aspirin use after 28 weeks of gestation is debatable. If aspirin discontinuation at 28 weeks of gestation is proven to be feasible for preventing preterm PE, it will not only reduce the risk of Perinatal Haemorrhage but also save medical expenses. **Detailed Description:** This study aimed to optimize the strategy of aspirin to prevent preeclampsia, by exploring whether aspirin discontinuation at 28 weeks of gestation to prevent preterm PE is not inferior to 36 weeks of gestation. We will include pregnant women with normal NT scan who meet at least one high-risk factor or at least two medium-risk factors. During 12-16 weeks of gestation, the mean arterial pressure (MAP) and placental growth factor (PlGF) are measured, then initiating aspirin 100mg qn. At 24-27+6 weeks of gestation, the patients were 1:1 randomly divided into two groups, sFlt-1 and PlGF were detected at the same time. The experimental group discontinue aspirin at 28 weeks of gestation, and the control group receive aspirin until 36 weeks of gestation. The incidence of preterm PE in the two groups was compared by non-inferiority test, and the non-inferiority threshold was 2%. ### Conditions Module **Conditions:** - Preeclampsia - Perinatal Haemorrhage **Keywords:** - low dose aspirin - prevention - preeclampsia - pregnancy outcomes - randomized controlled trial - aspirin discontinuation ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** Pregnant women with normal NT scan who meet at least one high-risk factor or at least two medium-risk factors are included. Initiating aspirin 100mg qn during 12-16 weeks of gestation. At 24-27+6 weeks of gestation, the patients were 1:1 randomly divided into two groups. The experimental group discontinue aspirin at 28 weeks of gestation, and the control group receive aspirin until 36 weeks of gestation. ##### Masking Info **Masking:** NONE **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 1800 **Type:** ESTIMATED **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** initiation of aspirin 100mg qn from 12 to 16 weeks of gestation, receiving 1:1 randomized grouping at 24-27+6 weeks of gestation, discontinuing aspirin at 28 weeks of gestation. **Intervention Names:** - Drug: aspirin discontinuation **Label:** Aspirin discontinuation group **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** initiation of aspirin 100mg qn from 12 to 16 weeks of gestation, receiving 1:1 randomized grouping at 24-27+6 weeks of gestation, continuing aspirin until 36 weeks of gestation. **Intervention Names:** - Drug: aspirin continuation **Label:** Control group **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Aspirin discontinuation group **Description:** Initiation of aspirin 100mg qn from 12 to 16 weeks of gestation, discontinuing aspirin at 28 weeks of gestation according to randomization at 24-27+6 weeks of gestation. **Name:** aspirin discontinuation **Other Names:** - initiation of aspirin in early pregnancy **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Control group **Description:** Initiation of aspirin 100mg qn from 12 to 16 weeks of gestation, continuing aspirin until 36 weeks of gestation according to randomization at 24-27+6 weeks of gestation. **Name:** aspirin continuation **Other Names:** - initiation of aspirin in early pregnancy **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** delivery with preeclampsia before 37 weeks of gestation **Measure:** Incidence of preterm preeclampsia **Time Frame:** Within one week after delivery #### Secondary Outcomes **Description:** delivery with preeclampsia before 34 weeks of gestation **Measure:** Incidence of early-onset preeclampsia **Time Frame:** Within one week after delivery **Description:** delivery with preeclampsia after 37 weeks of gestation **Measure:** Incidence of term preeclampsia **Time Frame:** Within one week after delivery **Description:** new onset of high blood pressure after 20 weeks of gestation (systolic blood pressure ≥140 mm Hg and/or diastolic blood pressure ≥90 mm Hg) **Measure:** Incidence of gestational hypertension **Time Frame:** Within one week after delivery **Description:** birth weight below the 10th percentile **Measure:** Incidence of small for gestational age **Time Frame:** Within one week after delivery ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. At \<16 weeks of gestation, normal NT scan 2. At least 1 high risk factor or at least 2 moderate risk factors 3. Intend to receive prenatal examination and deliver in this institution 4. Signed a written informed consent for participation in the study Exclusion Criteria: 1. Aspirin initiated after 16 week 2. Intolerant or allergic to aspirin 3. Aspirin adherence was \<80% 4. Miscarriage or termination of pregnancy before randomization 5. drop out (do not return to the hospital for delivery). 6. Lost to follow-up **Maximum Age:** 45 Years **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Fang He, M.D **Phone:** +86 13724831279 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Qingwen Nie, Master **Phone:** +86 15622149953 **Role:** CONTACT #### Locations **Location 1:** **City:** Guangzhou **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** FANG HE, M.D - **Phone:** 13724831279 - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Qingwen Nie, Master - **Phone:** 15622149953 - **Role:** CONTACT **Country:** China **Facility:** FANG HE **State:** Guangdong **Status:** RECRUITING **Zip:** 510150 #### Overall Officials **Official 1:** **Affiliation:** The Third Affiliated Hospital of Guangzhou Medical University **Name:** Fang He, M.D **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000010335 - Term: Pathologic Processes - ID: D000046110 - Term: Hypertension, Pregnancy-Induced - 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: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M9556 - Name: Hemorrhage - Relevance: HIGH - As Found: Hemorrhage - ID: M14106 - Name: Pre-Eclampsia - Relevance: HIGH - As Found: Preeclampsia - ID: M7633 - Name: Eclampsia - Relevance: LOW - As Found: Unknown - ID: M10024 - Name: Hypertension - Relevance: LOW - As Found: Unknown - ID: M25635 - Name: Hypertension, Pregnancy-Induced - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: T2019 - Name: Eclampsia - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000011225 - Term: Pre-Eclampsia - ID: D000006470 - Term: Hemorrhage ### Intervention Browse Module - Ancestors - ID: D000000894 - Term: Anti-Inflammatory Agents, Non-Steroidal - ID: D000018712 - Term: Analgesics, Non-Narcotic - ID: D000000700 - Term: Analgesics - ID: D000018689 - Term: Sensory System Agents - ID: D000018373 - Term: Peripheral Nervous System Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000000893 - Term: Anti-Inflammatory Agents - ID: D000018501 - Term: Antirheumatic Agents - ID: D000005343 - Term: Fibrinolytic Agents - ID: D000050299 - Term: Fibrin Modulating Agents - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000010975 - Term: Platelet Aggregation Inhibitors - ID: D000016861 - Term: Cyclooxygenase Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors - ID: D000058633 - Term: Antipyretics ### Intervention Browse Module - Browse Branches - Abbrev: Antipy - Name: Antipyretics - Abbrev: Infl - Name: Anti-Inflammatory Agents - Abbrev: ARhu - Name: Antirheumatic Agents - Abbrev: FiAg - Name: Fibrinolytic Agents - Abbrev: Analg - Name: Analgesics - Abbrev: PlAggInh - Name: Platelet Aggregation Inhibitors - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M4548 - Name: Aspirin - Relevance: HIGH - As Found: Progression - ID: M4217 - Name: Anti-Inflammatory Agents - Relevance: LOW - As Found: Unknown - ID: M4218 - Name: Anti-Inflammatory Agents, Non-Steroidal - Relevance: LOW - As Found: Unknown - ID: M4032 - Name: Analgesics - Relevance: LOW - As Found: Unknown - ID: M20786 - Name: Analgesics, Non-Narcotic - Relevance: LOW - As Found: Unknown - ID: M20604 - Name: Antirheumatic Agents - Relevance: LOW - As Found: Unknown - ID: M8473 - Name: Fibrinolytic Agents - Relevance: LOW - As Found: Unknown - ID: M13865 - Name: Platelet Aggregation Inhibitors - Relevance: LOW - As Found: Unknown - ID: M19209 - Name: Cyclooxygenase Inhibitors - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown - ID: M29176 - Name: Antipyretics - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000001241 - Term: Aspirin ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04747379 **Acronym:** PEERS **Brief Title:** Psychological Effect of Explicit Recall After Sedation (PEERS) **Official Title:** Psychological Effect of Explicit Recall After Sedation #### Organization Study ID Info **ID:** 114966 #### Organization **Class:** OTHER **Full Name:** Lawson Health Research Institute ### Status Module #### Completion Date **Date:** 2025-12 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-03-01 **Type:** ACTUAL **Last Update Submit Date:** 2024-02-29 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2025-12 **Type:** ESTIMATED #### Start Date **Date:** 2021-09-16 **Type:** ACTUAL **Status Verified Date:** 2024-02 #### Study First Post Date **Date:** 2021-02-10 **Type:** ACTUAL **Study First Submit Date:** 2020-10-27 **Study First Submit QC Date:** 2021-02-09 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Lawson Health Research Institute #### Responsible Party **Investigator Affiliation:** Lawson Health Research Institute **Investigator Full Name:** Jason Chui **Investigator Title:** Anesthesiologist/Associate Professor, Western University, Department of Anesthesia and Perioperative Medicine **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Is US Export:** True **Oversight Has DMC:** False ### Description Module **Brief Summary:** Awareness, or explicit recall, under sedation is often intended and expected by anesthesiologists and is assumed that does not associate with any sequelae. Thus, awareness under sedation is a common event and is estimated to occur in around one-quarter of patients in our population. However, two recent registry studies suggested some patients with awareness under sedation have comparable psychological sequelae to those patients with awareness during general anesthesia. As such, we plan to conduct a a single center, prospective cohort study to evaluate the incidence, experience and psychological consequence of awareness under sedation. In this study, we will prospectively include 2500 patients who will be scheduled to have major joint replacement surgery under regional anesthesia and sedation at University Hospital, London Health Sciences Center. All participants will be assessed at four separate time points including: 1. Enrollment/Surgical Preparatory Area (\~ 2 hours before surgery) 2. Post Anesthesia Care Unit (or up to 6 hours after surgery) 3. Postoperative day one (in hospital) 4. Postoperative 3 months (expected to be after discharge via telephone) ### Conditions Module **Conditions:** - Awareness, Anesthesia - Post Traumatic Stress Disorder - Depression ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 2500 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Outcomes Module #### Primary Outcomes **Description:** Explicit recall will be assessed by Modified Brice Questionnaire **Measure:** Explicit recall **Time Frame:** Up to 6 hours after surgery #### Secondary Outcomes **Description:** Explicit recall will be assessed by Modified Brice Questionnaire The Michigan Awareness Classification is used to report the levels of sensation during awareness. 5 Levels of sensation are classified as auditory, tactile, pain, paralysis, or pain and paralysis. A designation of 'D' for distress is used for patient reports of fear, anxiety, suffocation, sense of doom, sense of impending death, or similar reports that indicated emotional distress during the explicit recall experience. **Measure:** Explicit Recall **Time Frame:** Postoperative Day 1 **Description:** The Michigan Awareness Classification - Michigan Score The Michigan Awareness Classification is used to report the levels of sensation during awareness. 5 Levels of sensation are classified as auditory, tactile, pain, paralysis, or pain and paralysis. A designation of 'D' for distress is used for patient reports of fear, anxiety, suffocation, sense of doom, sense of impending death, or similar reports that indicated emotional distress during the explicit recall experience. **Measure:** Sensation During Awareness **Time Frame:** Postoperative Day 1 **Description:** Light, smell, and other feelings The Michigan Awareness Classification is used to report the levels of sensation during awareness. 5 Levels of sensation are classified as auditory, tactile, pain, paralysis, or pain and paralysis. A designation of 'D' for distress is used for patient reports of fear, anxiety, suffocation, sense of doom, sense of impending death, or similar reports that indicated emotional distress during the explicit recall experience. **Measure:** Details of recall experience **Time Frame:** Postoperative Day 1 **Description:** PCL-S Questionnaire **Measure:** Post Traumatic Stress Disorder **Time Frame:** Postoperative 3 Months **Description:** PHQ-9 Questionnaire **Measure:** Depression **Time Frame:** Postoperative 3 Months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Adult patient (\>18 years old) 2. Scheduled to have orthopedic surgery or joint replacement therapy under sedation/regional anesthesia 3. Patient is able to communicate in English 4. ASA I-V Exclusion Criteria: 1. Unable to obtain informed consent 2. Patient received General Anesthesia (GA) or conversion to GA during their surgical procedures 3. Unable to conduct the survey such as language barrier, overt psychiatric disorder thought to interfere with the reliability of the interview (e.g. dementia etc) 4. Patient who is expected to require mechanical ventilation at postoperative period (i.e. unable to perform surveys at Post-Anesthesia Care Unit (PACU) and postoperative day one) 5. Unable to follow up via phone or attend a postoperative follow-up visit at postoperative 3 months **Minimum Age:** 18 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Patients scheduled to undergo orthopedic or joint replacement surgeries under sedation/regional anesthesia at University Hospital located in London, Ontario, Canada. ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Jason Chui, MD **Phone:** 519-685-8500 **Phone Ext:** 34435 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** LeeAnne Fochesato, MD **Phone:** 519-663-3384 **Role:** CONTACT #### Locations **Location 1:** **City:** London **Contacts:** ***Contact 1:*** - **Name:** Jason Chui, MBChB - **Role:** CONTACT ***Contact 2:*** - **Name:** LeeAnne Fochesato, MSc - **Role:** CONTACT **Country:** Canada **Facility:** London Health Science Centre **State:** Ontario **Status:** RECRUITING **Zip:** N6A 5A5 #### Overall Officials **Official 1:** **Affiliation:** University of Western Ontario, Canada **Name:** Jason Chui, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ## Derived Section ### Condition Browse Module - Ancestors - ID: D000068099 - Term: Trauma and Stressor Related Disorders - ID: D000001523 - Term: Mental Disorders - ID: D000007431 - Term: Intraoperative Complications - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC26 - Name: Wounds and Injuries ### Condition Browse Module - Browse Leaves - ID: M16103 - Name: Stress Disorders, Post-Traumatic - Relevance: HIGH - As Found: Post Traumatic Stress Disorder - ID: M7058 - Name: Depression - Relevance: LOW - As Found: Unknown - ID: M7061 - Name: Depressive Disorder - Relevance: LOW - As Found: Unknown - ID: M24916 - Name: Stress Disorders, Traumatic - Relevance: HIGH - As Found: Traumatic Stress Disorder - ID: M29299 - Name: Intraoperative Awareness - Relevance: HIGH - As Found: Awareness, Anesthesia - ID: M17685 - Name: Wounds and Injuries - Relevance: LOW - As Found: Unknown - ID: M222 - Name: Trauma and Stressor Related 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: M10465 - Name: Intraoperative Complications - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000058926 - Term: Intraoperative Awareness - ID: D000040921 - Term: Stress Disorders, Traumatic - ID: D000013313 - Term: Stress Disorders, Post-Traumatic ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03206879 **Brief Title:** Fecal ESBL Resurgence During Antibiotic Treatment **Official Title:** Fecal ESBL Resurgence During Antibiotic Treatment #### Organization Study ID Info **ID:** 16-086 #### Organization **Class:** OTHER **Full Name:** University Hospital, Akershus ### Status Module #### Completion Date **Date:** 2018-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2024-02-06 **Type:** ACTUAL **Last Update Submit Date:** 2024-02-05 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2018-08 **Type:** ACTUAL #### Start Date **Date:** 2016-11 **Type:** ACTUAL **Status Verified Date:** 2024-02 #### Study First Post Date **Date:** 2017-07-02 **Type:** ACTUAL **Study First Submit Date:** 2017-06-30 **Study First Submit QC Date:** 2017-06-30 **Why Stopped:** Several sites did not manage to recruit patients. ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER_GOV **Name:** Sorlandet Hospital HF **Class:** OTHER_GOV **Name:** Helse Stavanger HF **Class:** OTHER_GOV **Name:** Sykehuset Telemark **Class:** OTHER **Name:** Oslo University Hospital **Class:** OTHER **Name:** Sykehuset Ostfold #### Lead Sponsor **Class:** OTHER **Name:** University Hospital, Akershus #### Responsible Party **Investigator Affiliation:** University Hospital, Akershus **Investigator Full Name:** Silje Bakken Jørgensen **Investigator Title:** Head of section, infection control officer **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Norwegian hospitals are struggling to control spread of extended-spectrum beta-lactamase producing Enterobacteriaceae (ESBL-E) in health care settings. Strict screening and isolation criteria are enforced in hospital, and patients previously infected by ESBL-E are regarded as possible ESBL-E carriers indefinitely. This observational multi-centre cross-sectional study aims to estimate the duration of fecal ESBL-E carriage and to assess the risk of ESBL-E resurgence during hospital stay for patients previously infected by ESBL-E. Through better knowledge of these risk factors, infection control procedures may be better tailored to rhe individual patient. ### Conditions Module **Conditions:** - Antibiotic Drug Resistance **Keywords:** - ESBL - Enterobacteriaceae - multidrug-resistance ### Design Module #### Bio Spec **Description:** Bacterial fecal isolates. **Retention:** SAMPLES_WITH_DNA #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 50 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Outcomes Module #### Primary Outcomes **Measure:** fecal carriage of ESBL-E **Time Frame:** Baseline #### Secondary Outcomes **Measure:** fecal ESBL-E carriage developed during admission **Time Frame:** Baseline (admission) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: Patients with previous infection or colonization with ESBL-E admitted to one of the participating wards. - Exclusion Criteria: * Not able to give informed consent. **Maximum Age:** 120 Years **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Adult, hospitalized patients previously infected by ESBL-E. ### Contacts Locations Module #### Overall Officials **Official 1:** **Affiliation:** Akershus univversitetssykehus HF **Name:** Silje B Jørgensen, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Intervention Browse Module - Browse Branches - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M4222 - Name: Anti-Bacterial Agents - Relevance: LOW - As Found: Unknown - ID: M4224 - Name: Antibiotics, Antitubercular - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03381079 **Brief Title:** Efficacy and Safety of Posterior Scleral Reinforcement on Controlling Myopia in Adults With High Myopia **Official Title:** Posterior Scleral Reinforcement for Adults With High Myopia #### Organization Study ID Info **ID:** TRECKY2017-049 #### Organization **Class:** OTHER **Full Name:** Beijing Tongren Hospital ### Status Module #### Completion Date **Date:** 2020-12-31 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** NOT_YET_RECRUITING #### Last Update Post Date **Date:** 2017-12-22 **Type:** ACTUAL **Last Update Submit Date:** 2017-12-20 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2020-10-01 **Type:** ESTIMATED #### Start Date **Date:** 2018-04-01 **Type:** ESTIMATED **Status Verified Date:** 2017-12 #### Study First Post Date **Date:** 2017-12-21 **Type:** ACTUAL **Study First Submit Date:** 2017-12-12 **Study First Submit QC Date:** 2017-12-17 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Beijing Tongren Hospital #### Responsible Party **Investigator Affiliation:** Beijing Tongren Hospital **Investigator Full Name:** Shi-Ming Li **Investigator Title:** Vice director of Ophthalmology clinical research center **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** This study will evaluate the efficacy and safety of posterior scleral reinforcement on controlling myopia progression, including change in refraction, axial elongation as well as sight-threatening complications, in adults with high myopia. Half the adults will receive posterior scleral reinforcement, while the other half will receive no surgerical treatment. ### Conditions Module **Conditions:** - High Myopia - Posterior Scleral Reinforcement - Adults - Axial Elongation - Myopia Progression ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 120 **Type:** ESTIMATED **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** In this arm, the adults with high myopia will be given posterior scleral reinforcement. **Intervention Names:** - Procedure: Posterior scleral reinforcement **Label:** Surgical group **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** In this arm, the adults with high myopia will not be given any surgical treatment. **Label:** Control group **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Surgical group **Description:** Posterior scleral reinforcement (pPSR) is a procedure applied with autologous or biological material or synthetic materials reinforcing pole of the sclera after eyeball, in order to prevent or alleviate myopic development. **Name:** Posterior scleral reinforcement **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Description:** Level of myopia and myopia progression as assessed by autorefraction **Measure:** Cycloplegic autorefraction **Time Frame:** 2 years **Description:** Longth of the eyeball as assessed by IOL Master or Lenstar **Measure:** Axial length **Time Frame:** 2 years #### Secondary Outcomes **Description:** Bleeding, inflammation or retinal detachment as assessed by B scan, slitlamp, etc. **Measure:** Number of participants with treatment-related adverse events **Time Frame:** 2 years **Description:** An index for visual function measured by visual acuity chart **Measure:** Visual acuity **Time Frame:** 2 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Cycloplegic spherical equivalent less than -6.0 D * Myopia progression greater than 1.0 D per year * Normal IOP, no strabismus or any other ocular pathological changes * no any other ocular or systematic diseases that may affect refractive development Exclusion Criteria: * Currently or history using other interventions to control myopia progression (acupuncture, drugs, contact lens, ear needles and so on) * Unable to cooperate with the ocular examination,questionnaire survey,or orthokeratology wearing **Maximum Age:** 40 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Shi-Ming Li, PhD **Phone:** +86-10-58269920 **Role:** CONTACT #### Overall Officials **Official 1:** **Affiliation:** Beijing Tongren Eye Center, Beijing Tongren Hospital **Name:** Ningli Wang, PhD **Role:** STUDY_CHAIR ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012030 - Term: Refractive Errors - ID: D000005128 - Term: Eye Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC11 - Name: Eye Diseases ### Condition Browse Module - Browse Leaves - ID: M20559 - Name: Disease Progression - Relevance: LOW - As Found: Unknown - ID: M12168 - Name: Myopia - Relevance: HIGH - As Found: Myopia - ID: M14872 - Name: Refractive Errors - Relevance: LOW - As Found: Unknown - ID: M8271 - Name: Eye Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000009216 - Term: Myopia ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02998879 **Acronym:** rhLaman-08 **Brief Title:** Trial on Safety and Efficacy of Velmanase Alfa Treatment in Pediatric Patients With Alpha-Mannosidosis **Official Title:** A 24-month Multicenter, Open-label Phase II Trial Investigating the Safety and Efficacy of Repeated Velmanase Alfa (Recombinant Human Alpha-mannosidase) Treatment in Pediatric Patients Below 6 Years of Age With Alpha-Mannosidosis #### Organization Study ID Info **ID:** CCD-LMZYMAA1-08 #### Organization **Class:** INDUSTRY **Full Name:** Chiesi Farmaceutici S.p.A. #### Secondary ID Infos **ID:** 2016-001988-36 **Type:** EUDRACT_NUMBER ### Status Module #### Completion Date **Date:** 2020-07 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-10-26 **Type:** ACTUAL **Last Update Submit Date:** 2021-10-18 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2020-07 **Type:** ACTUAL #### Start Date **Date:** 2016-12 **Status Verified Date:** 2021-10 #### Study First Post Date **Date:** 2016-12-21 **Type:** ESTIMATED **Study First Submit Date:** 2016-11-25 **Study First Submit QC Date:** 2016-12-16 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Cromsource #### Lead Sponsor **Class:** INDUSTRY **Name:** Chiesi Farmaceutici S.p.A. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The main objectives of the study are to evaluate safety and efficacy of repeated treatment with recombinant human alfa-mannosidase of patients with alfa-mannosidosis aged less than 6 years **Detailed Description:** The Primary endpoints of the study include: * Safety and tolerability of velmanase alfa as per Adverse events (AEs, including IRR), vital signs, laboratory parameters (hematology, biochemistry and urinanalysis) * Detection of anti-velmanase alfa antibodies and neutralizing/inhibitory antibodies The Secondary endpoints include changes from baseline to 24 months for the following parameters. Efficacy outcomes: * Serum oligosaccharides * Functional capacity: Peabody Developmental Motor Scale - 2nd edition (PDMS-2) scores, Mullen's Scale of Early Learning (MSEL) scores, Bruininks-Oseretsky Test Of Motor Proficiency-2nd Edition (BOT-2), when applicable by age (from 4 years) or upon the judgment of the physician * Endurance: 3-Minute Stair Climb Test (3MSCT) and 6-Minute Walk Test (6MWT) in pediatric patients from 4 years of age, or when applicable according to the judgment of the physician, 2-Minute Walk Test (2MWT) in pediatric patients below 4 years of age, or when applicable according to the judgment of the physician * Hearing evaluation: Otoacoustic Emissions (OAE) testing, Automatic Auditory Brainstem Response (A-ABR) audiometry * Immunological profile, when applicable upon the judgment of the physician: * CSF biomarkers: Tau protein (Tau), Neurofilament Protein Light (NFL), Glial Fibrillary Acidic Protein (GFAp), Oligosaccharides * Assessment of quality of life via Questionnaire to parents * Assessment of mannose-rich oligosaccharides in brain tissue, MRI * Pharmacokinetic parameters ### Conditions Module **Conditions:** - Alpha-Mannosidosis ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 5 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** velmanase alfa 1mg/kg body weight infusion **Intervention Names:** - Drug: Velmanase Alfa (e.g. Lamazym) **Label:** Velmanase Alfa **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Velmanase Alfa **Description:** iv infusion treatment **Name:** Velmanase Alfa (e.g. Lamazym) **Other Names:** - Lamazym **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Safety and tolerability assessed as per AEs including infusion-related reactions \[IRRs\] **Measure:** Safety and tolerability of velmanase alfa as per Adverse events **Time Frame:** From baseline throughout study completion, at least of 2 years **Measure:** Safety and tolerability of velmanase alfa as per vital signs **Time Frame:** From baseline throughout study completion, at least of 2 years **Measure:** Safety and tolerability of velmanase alfa as per clinical laboratory parameters as per hematology **Time Frame:** From baseline throughout study completion, at least of 2 years **Measure:** Safety and tolerability of velmanase alfa as per clinical laboratory parameters as per blood biochemistry **Time Frame:** From baseline throughout study completion, at least of 2 years **Measure:** Safety and tolerability of velmanase alfa as per clinical laboratory parameters as per urinalysis **Time Frame:** From baseline throughout study completion, at least of 2 years **Description:** Serum samples for anti-velmanase alfa-IgG antibody (ADA) testing will be obtained **Measure:** Detection of anti-velmanase alfa-IgG antibodies (ADA) and neutralizing/inhibitory antibodies **Time Frame:** From baseline throughout study completion, at least of 2 years #### Secondary Outcomes **Description:** Assessment of change from baseline in levels of Serum oligosaccharides **Measure:** Evaluation of levels of Serum oligosaccharides **Time Frame:** From baseline throughout study completion, at least for 2 years **Description:** Serum samples for anti-velmanase alfa-IgG antibody (ADA) testing will be obtained **Measure:** Functional capacity: The Peabody Developmental Motor Scale test (PDMS-2) **Time Frame:** From baseline throughout study completion, at least for 2 years **Measure:** Functional capacity: Bruininks-Oseretsky test of Motor Proficiency (BOT-2) when applicable by age (from 4 years) or upon the judgment of the physician **Time Frame:** From baseline throughout study completion, at least for 2 years **Measure:** Functional capacity: Mullen Scales of Early Learning (MSEL) **Time Frame:** From baseline throughout study completion, at least for 2 years **Measure:** Endurance: 3-Minute Stair Climb Test (3MSCT) in pediatric patients from 4 years of age, or when applicable according to the judgment of the physician **Time Frame:** From baseline throughout study completion, at least for 2 years **Measure:** Endurance: 6-Minute Walk Test (6MWT) in pediatric patients from 4 years of age, or when applicable according to the judgment of the physician 2-Minute Walk Test (2MWT) in pediatric patients below 4 years of age **Time Frame:** From baseline throughout study completion, at least for 2 years **Measure:** Hearing evaluation: Otoacoustic Emissions (OAE) testing **Time Frame:** From baseline throughout study completion, at least for 2 years **Measure:** Hearing evaluation: Automatic Auditory Brainstem Response (A-ABR) audiometry **Time Frame:** From baseline throughout study completion, at least for 2 years **Measure:** Immunological profile when applicable upon the judgement of the physician (Serum IgG, IgA, IgM; in vitro synthesis of IgG; in vitro proliferative response and Immunophenotype) **Time Frame:** From baseline throughout study completion, at least for 2 years **Measure:** CSF biomarkers: Tau protein (Tau) § Neurofilament Protein Light (NFL) § Glial Fibrillary Acidic Protein (GFAp) § Oligosaccharides **Time Frame:** From baseline throughout study completion, at least for 2 years **Measure:** Assessment of quality of life via Questionnaire **Time Frame:** From baseline throughout study completion, at least for 2 years **Measure:** Assessment of mannose-rich oligosaccharides in brain tissue, as measured by Magnetic Resonance Spectroscopy (MRS) **Time Frame:** From baseline throughout study completion, at least for 2 years **Measure:** Magnetic Resonance Imaging (MRI) in white matter, gray matter and in centrum semi ovale, and diffusion-MRI of the brain, **Time Frame:** From baseline throughout study completion, at least for 2 years **Measure:** Pharmacokinetic parameters to determine Cmax (Peak Concentration) **Time Frame:** At first dose (visit 1) and after 6 months (visit 26) **Measure:** Pharmacokinetic parameters to determine Ctrough (Trough Plasma Concentration) **Time Frame:** At first dose (visit 1) and after 6 months (visit 26) **Measure:** Pharmacokinetic parameters to determine Area Under Curve (AUC24) **Time Frame:** At first dose (visit 1) and after 6 months (visit 26) **Measure:** Pharmacokinetic parameters to determine AUClast (Area Under Curve After The Last Count) **Time Frame:** At first dose (visit 1) and after 6 months (visit 26) **Measure:** Pharmacokinetic parameters to determine AUCinf (Area Under Curve From Time Zero To Infinity) **Time Frame:** At first dose (visit 1) and after 6 months (visit 26) **Measure:** Pharmacokinetic parameters to determine tmax (Time To Peak Concentration) **Time Frame:** At first dose (visit 1) and after 6 months (visit 26) **Measure:** Pharmacokinetic parameters to determine CL (Clearance) **Time Frame:** At first dose (visit 1) and after 6 months (visit 26) **Measure:** Pharmacokinetic parameters to determine t1/2 (Elimination Half-Life) **Time Frame:** At first dose (visit 1) and after 6 months (visit 26) **Measure:** Pharmacokinetic parameters to determine Rac (Obs) Observed Accumulation Ratio **Time Frame:** At first dose (visit 1) and after 6 months (visit 26) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Patient's custodial parent(s) must provide signed ICF prior to the involvement of the patient in any trial-related activities 2. The subject's custodial parent(s) must have the ability to comply with the protocol 3. The subject must have a confirmed diagnosis of alpha-mannosidosis as defined by alpha-mannosidase activity in leukocytes or fibroblasts \< 10% of normal activity (historical data) 4. The subject must have an age at the time of screening \< 6 years. Exclusion Criteria: 1. The subject's diagnosis cannot be confirmed by alpha-mannosidase activity \< 10% of normal activity 2. Presence of known chromosomal abnormality and syndromes affecting psychomotor development, other than alpha-mannosidosis 3. History of BMT 4. Presence of known clinically significant cardiovascular, hepatic, pulmonary, or renal disease or other medical conditions that, in the opinion of the Investigator, would preclude participation in the trial 5. Any other medical condition or serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial 6. Planned major surgery that, in the opinion of the Investigator, would preclude participation in the trial 7. Participation in other interventional trials testing the IMP within the last 3 months. **Maximum Age:** 6 Years **Sex:** ALL **Standard Ages:** - CHILD ### Contacts Locations Module #### Locations **Location 1:** **City:** Wien **Country:** Austria **Location 2:** **City:** Copenhagen **Country:** Denmark **Location 3:** **City:** Lyon **Country:** France **Location 4:** **City:** Hamburg **Country:** Germany **Location 5:** **City:** Mainz **Country:** Germany **Location 6:** **City:** Trieste **Country:** Italy ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### See Also Links **Label:** Study Record on EU Clinical Trials Register including results **URL:** https://www.clinicaltrialsregister.eu/ctr-search/trial/2016-001988-36/results ## Derived Section ### Condition Browse Module - Ancestors - ID: D000002239 - Term: Carbohydrate Metabolism, Inborn Errors - ID: D000008661 - Term: Metabolism, Inborn Errors - ID: D000030342 - Term: Genetic Diseases, Inborn - ID: D000016464 - Term: Lysosomal Storage Diseases - ID: D000008659 - Term: Metabolic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M25409 - Name: Mannosidase Deficiency Diseases - Relevance: HIGH - As Found: Mannosidosis - ID: M11352 - Name: alpha-Mannosidosis - Relevance: HIGH - As Found: Alpha-Mannosidosis - ID: M6879 - Name: Deficiency Diseases - Relevance: LOW - As Found: Unknown - ID: M11641 - Name: Metabolism, Inborn Errors - Relevance: LOW - As Found: Unknown - ID: M5498 - Name: Carbohydrate Metabolism, Inborn Errors - Relevance: LOW - As Found: Unknown - ID: M23686 - Name: Genetic Diseases, Inborn - Relevance: LOW - As Found: Unknown - ID: M18871 - Name: Lysosomal Storage Diseases - Relevance: LOW - As Found: Unknown - ID: M11639 - Name: Metabolic Diseases - Relevance: LOW - As Found: Unknown - ID: T299 - Name: Alpha-mannosidosis - Relevance: HIGH - As Found: Alpha-Mannosidosis ### Condition Browse Module - Meshes - ID: D000044904 - Term: Mannosidase Deficiency Diseases - ID: D000008363 - Term: alpha-Mannosidosis ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05838079 **Acronym:** CBHS-I **Brief Title:** Copenhagen Baby Heart Study - Impact **Official Title:** Copenhagen Baby Heart Study - Impact #### Organization Study ID Info **ID:** H-19038069 #### Organization **Class:** OTHER **Full Name:** Rigshospitalet, Denmark ### Status Module #### Completion Date **Date:** 2029-07 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-05-01 **Type:** ACTUAL **Last Update Submit Date:** 2023-04-20 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2029-07 **Type:** ESTIMATED #### Start Date **Date:** 2018-11-01 **Type:** ACTUAL **Status Verified Date:** 2023-04 #### Study First Post Date **Date:** 2023-05-01 **Type:** ACTUAL **Study First Submit Date:** 2023-04-20 **Study First Submit QC Date:** 2023-04-20 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Copenhagen University Hospital at Herlev **Class:** OTHER **Name:** Copenhagen University Hospital, Hvidovre #### Lead Sponsor **Class:** OTHER **Name:** Henning Bundgaard #### Responsible Party **Investigator Affiliation:** Rigshospitalet, Denmark **Investigator Full Name:** Henning Bundgaard **Investigator Title:** Professor **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** Copenhagen Baby Heart Study - Impact (CBHS-I) is an extension to Copenhagen Baby Heart (CBH) which included over 25.000 new-borns in the Copenhagen area between 2016-2018. Based on clinical and subclinical deviations in the examinations in CBH, subgroups of participants will be invited to clinical examinations (echocardiography and electrocardiogram) in early childhood. There will also be a new, targeted inclusion based on certain exposures during pregnancy. The main objectives are to assess the prevalence of congenital and inherited heart disease and, and the development of these during early childhood; examining the association between pre- and postnatal exposure, disease, lifestyle, environmental and genetic factors; continue to establish reference values for echocardiography in Danish neonates and children. **Detailed Description:** Copenhagen Baby Heart Study - Impact (CBHS-I) is an extension to Copenhagen Baby Heart (CBH) which included over 25.000 new-borns in the Copenhagen area between 2016-2018. Purpose: 1. To assess the prevalence of congenital and inherited heart disease not detected prenatally, and through follow-up asses the consequence of these, e.g., bicuspid aortic valve and septal defects. 2. To assess the prevalence of structural and functional anomalies detected by echocardiography and electrocardiography shortly after birth and, by follow-up examination, assess the consequence of these in early childhood. 3. To establish reference values for echocardiography and ECG for Danish children. 4. To assess the association between high-risk pregnancies and complications during pregnancy (e.g., twin/multiple pregnancy, assisted human reproduction, preeclampsia, and gestational diabetes) and the risk for congenital heart disease in the offspring and early childhood. 5. To assess the association between maternal chronic diseases (e.g., thyroid disorders or pre-existing diabetes) and the risk for congenital heart disease in the offspring and early childhood. 6. To assess the life-long development of cardiovascular disease as well as other conditions and to study associations between both pre- and postnatal exposure and disease, including lifestyle, environmental and genetic factors. Methods The birth cohort of CBH constitute over 25.000 new-borns. At birth an umbilical cord blood sample was taken and immediately analysed for routine biochemical markers, and samples were also stored for possibility of future analyses and DNA sequencing. All clinical examinations (echocardiography, electrocardiography, and measurement of oxygen saturation) took place within the first 2nd month of life, the vast majority within the 1st month, median age at examination was 11 days (interquartile range 7-15). In addition to this CBH has built a unique and extended database which include information about the maternal and paternal health, the pregnancy and birth. As part of CBHS-I subgroups of the CBH cohort (e.g., children born to mothers with diabetes or preeclampsia, or children with abnormal findings at baseline examinations in CBH) will be invited for follow-up examinations (echocardiography and electrocardiography). There will also be a new inclusion of new-borns from the same geographical area and Hospitals as in CBH in order to expand some of the sub cohorts, e.g., children born to mothers with diabetes. ### Conditions Module **Conditions:** - Congenital Heart Disease ### Design Module #### Bio Spec **Description:** Venous and/or umbilical cord blood sample for DNA extraction and analysis of biomarkers **Retention:** SAMPLES_WITH_DNA #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 35000 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** New-borns, within the first 30 days of birth. **Intervention Names:** - Other: Clinical examination **Label:** New-borns (CBHS-I) #### Arm Group 2 **Description:** Children, 3-13 years of age. The children from the original inclusion in CBH, and those included in CBHS-I. **Intervention Names:** - Other: Clinical examination **Label:** Children (CBH and CBHS-I) #### Arm Group 3 **Description:** Children and adults, age 1-99 years of age. Some subprojects will invite the family members of the participating child for family examinations. **Intervention Names:** - Other: Clinical examination **Label:** Family members ### Interventions #### Intervention 1 **Arm Group Labels:** - Children (CBH and CBHS-I) - Family members - New-borns (CBHS-I) **Description:** Echocardiography Electrocardiography Analysis of umbilical and/or venous blood. Peripheral measurement of oxygen saturation **Name:** Clinical examination **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Measure:** Prevalence of congenital heart disease **Time Frame:** 10 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Participant in CBH or * Family member of a participant in CBH or * Born to mothers with pre-existing or gestational diabetes at either Rigshospitalet, Hvidovre Hospital, or Herlev Hospital during the period July 2020 through December 2023. Exclusion Criteria: * Faliure of providing concent (parents) **Maximum Age:** 99 Years **Minimum Age:** 1 Day **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT **Study Population:** Participants from CBH and in some cases their family members. New-borns of mothers with pre-existing or gestational diabetes, included during CBHS-I. ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Henning Bundgaard, MD, DMSc **Phone:** +4535450512 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Kasper Iversen, MD, DMSc **Phone:** +4538686009 **Role:** CONTACT #### Locations **Location 1:** **City:** Copenhagen Ø **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Henning Bundgaard, MD, DMSc - **Phone:** +4535450512 - **Role:** CONTACT **Country:** Denmark **Facility:** Rigshospitalet Copenhagen University Hospital. **Status:** RECRUITING **Zip:** 2100 **Location 2:** **City:** Herlev **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Kasper Iversen, MD, DMSc - **Phone:** +4538686009 - **Role:** CONTACT **Country:** Denmark **Facility:** Herlev and Gentofte Hospital **Status:** RECRUITING **Zip:** 2730 **Location 3:** **City:** Hvidovre **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Christina Rørbye, MD, PhD, Associate Professor - **Phone:** +4538625520 - **Role:** CONTACT **Country:** Denmark **Facility:** Hvidovre Hospital **Status:** RECRUITING **Zip:** 2650 #### Overall Officials **Official 1:** **Affiliation:** Department of Cardiology, The Heart Center, Rigshospitalet, Copenhagen University Hospital, Denmark **Name:** Henning Bundgaard, MD, DMSc **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** Department of Cardiology, Herlev Hospital, Denmark. **Name:** Kasper Iversen, MD, DMSc **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000018376 - Term: Cardiovascular Abnormalities - ID: D000000013 - Term: Congenital Abnormalities ### Condition Browse Module - Browse Branches - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth ### Condition Browse Module - Browse Leaves - ID: M9419 - Name: Heart Diseases - Relevance: HIGH - As Found: Heart Disease - ID: M9418 - Name: Heart Defects, Congenital - Relevance: HIGH - As Found: Congenital Heart Disease - ID: M12 - Name: Congenital Abnormalities - Relevance: LOW - As Found: Unknown - ID: M20503 - Name: Cardiovascular Abnormalities - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000006331 - Term: Heart Diseases - ID: D000006330 - Term: Heart Defects, Congenital ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04709679 **Brief Title:** Laser Dosage in MicroPulse TransScleral CycloPhotocoagulation **Official Title:** Clinical Outcome of Varying Doses of Micropulse TransScleral CycloPhotocoagulation Laser in Patients With Glaucoma. #### Organization Study ID Info **ID:** STUDY00003149 #### Organization **Class:** OTHER **Full Name:** State University of New York at Buffalo ### Status Module #### Completion Date **Date:** 2021-10-11 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2022-07-01 **Type:** ACTUAL **Last Update Submit Date:** 2022-06-28 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2021-10-11 **Type:** ACTUAL #### Start Date **Date:** 2021-01-01 **Type:** ACTUAL **Status Verified Date:** 2022-06 #### Study First Post Date **Date:** 2021-01-14 **Type:** ACTUAL **Study First Submit Date:** 2021-01-12 **Study First Submit QC Date:** 2021-01-12 **Why Stopped:** Dose was not therapeudic ### Sponsor Collaborators Module #### Collaborators **Class:** UNKNOWN **Name:** IRIDEX Corporation #### Lead Sponsor **Class:** OTHER **Name:** State University of New York at Buffalo #### Responsible Party **Investigator Affiliation:** State University of New York at Buffalo **Investigator Full Name:** Sandra Sieminski **Investigator Title:** Principal Investigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Is US Export:** True **Oversight Has DMC:** False ### Description Module **Brief Summary:** This is a prospective study to evaluate the clinical outcome of varying doses of Micropulse TransScleral CycloPhotocoagulation (MP-TSCPC) laser in patients with glaucoma. The study will assess documented complication rates, changes in visual acuity after the procedure, changes in intraocular pressures and medication drop usage, and need for subsequent treatments (including further laser or surgery). **Detailed Description:** Glaucoma is a condition where an increase in the accumulation of aqueous humor inside the anterior chamber of the eye results in raised intraocular pressure (IOP), visual field defects and cupping of optic disc. Typically, patients who have glaucoma are treated with topical medications or incisional surgery to lower the IOP. Micropulse transscleral cyclophotocoagulation (MP-TSCPC) is a non-incisional laser surgery used in the treatment of glaucoma. MP-TSCPC powered by the Cyclo G6 (Iridex Corporation, Mountain View, CA, USA) causes mild thermal damage to the ciliary body (gland that produces aqueous fluid in the eye), which, in turn, causes a decrease in intraocular pressure. MP-TSCPC is a variation of a longstanding older treatment for glaucoma, traditional diode TSCPC, which caused a significant amount of tissue damage and therefore was reserved for the treatment of end stage glaucoma. The micropulse laser application includes an on-off cycle. In an on cycle, the micropulse probe administers a short pulse of laser energy followed by an off cycle which is a rest period for the tissue to cool off before the next on-off cycle begins. This corresponds to 0.5 ms duration of "on-time" and 1.1 ms interval of "off-time" during the laser application. This on-off cycle allows for the laser to treat the affected area without evidence of tissue damage, as opposed to the traditional diode TSCPC. Because only 31.3% of the total laser energy is applied to the ciliary body, there is a less tissue damage, less post-operative inflammation, and decreased complication rates. Therefore, MP-TSCPC is currently being utilized for milder cases of glaucoma, and in patients with viable vision. The laser probe is usually applied perpendicular to the limbus in a continuous sweeping motion, for an average of 10 seconds of sweeping time per hemifield, referred to as "dwell time". Therefore, the three parameters that can be varied in the treatment with MP-TSCPC are total laser duration (seconds), laser power (mW) and dwell time (seconds/hemifield). Most of the studies used a laser power of 2000 - 2500 mW. The laser duration also has varied between studies. A duration time of 50 to 180 sec per hemisphere was used in different studies. Most studies have utilized a dwell time of 10 seconds per hemifield, and one cited an application of a "stop and go" method, applying the laser in discrete spots at 10 seconds each. In all of the prior studies, despite the variable applications of power, dwell time, and duration, there have been similar rates of complications such as vision loss, macular edema, and hypotony. Generally, the laser duration is tailor-made and is adjusted based on the iris color and severity of glaucoma. However, there is a gap in knowledge on systematic comparison of different doses of MP-TSCPC for a safe and effective treatment of glaucoma. This study will systematically evaluate the clinical outcome of glaucoma surgery based on a logical variation of MP-TSCPC laser dosing parameters. ### Conditions Module **Conditions:** - Glaucoma **Keywords:** - MP-TSCPC - Laser Duration - Laser Dwell time - Laser Power ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 6 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** The laser dwell time will be constant but the laser power and duration will be varied for patients **Intervention Names:** - Procedure: MicroPulse TransScleral CycloPhotoCoagulation **Label:** Constant dwell time but varying power and duration **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** The laser power will be constant but the laser dwell time and duration will be varied for patients **Intervention Names:** - Procedure: MicroPulse TransScleral CycloPhotoCoagulation **Label:** Constant power but varying dwell time and duration **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** The laser duration will be constant but the laser dwell time and power will be varied for patients **Intervention Names:** - Procedure: MicroPulse TransScleral CycloPhotoCoagulation **Label:** Constant duration but varying dwell time and power **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Constant duration but varying dwell time and power - Constant dwell time but varying power and duration - Constant power but varying dwell time and duration **Description:** A laser power of 2000- 2500 mW, a laser duration time of 50 to 80 sec per hemifield and varying da number of sweeps per hemifield 3, 4 or 5 will be randomized between patients **Name:** MicroPulse TransScleral CycloPhotoCoagulation **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Description:** Baseline (pre-operative condition) and post operative intraocular pressure will be measured on Day1, Week1, Months 1, 3, 6, 12, 18 and 24. **Measure:** Changes in intraocular pressure measurements between baseline and postoperative followup visits **Time Frame:** 2years #### Secondary Outcomes **Description:** Baseline (pre-operative condition) and post operative visual acuity will be measured on Day1, Week1, Months 1, 3, 6, 12, 18 and 24. **Measure:** Changes in visual acuity measurements measured using a Snellen Vision Chart between baseline and postoperative followup visits **Time Frame:** 2 years **Description:** Baseline (pre-operative condition) and post operative number of medications list will be measured on Day1, Week1, Months 1, 3, 6, 12, 18 and 24. **Measure:** Changes in number of medications between baseline and postoperative followup visits **Time Frame:** 2 years **Description:** Rate of post surgical complications such as Hypotony, Corneal Edema, Hyphema, Iritis, Vitreous Hemorrhage and Cystoid Macular Edema. **Measure:** Rate of post surgical complications **Time Frame:** 2 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Decision to treat by MP-TSCPC Laser * Patients diagnosed with Glaucoma * Patients aged 18 years old and above * Glaucoma that is inadequately controlled on medical therapy * Patients with primary open angle glaucoma with or without previously failed trabeculectomy or other aqueous drainage surgical procedures Exclusion Criteria: * Patients age less than 18 years * Patients unable or unwilling to provide informed consent to participate in the study * Patients potentially unavailable for follow up visits * Patients with significant scleral thinning * Patients with ocular infection, inflammation or intraocular surgery in the study eye 2 months prior to enrollment in the study * Albino patients that have no iris pigmentation **Maximum Age:** 89 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Buffalo **Country:** United States **Facility:** The Ira G. Ross Eye Institute **State:** New York **Zip:** 14209 #### Overall Officials **Official 1:** **Affiliation:** Ross Eye Institute **Name:** Sandra F Sieminski, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009798 - Term: Ocular Hypertension - ID: D000005128 - Term: Eye Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC11 - Name: Eye Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC14 - Name: Heart and Blood Diseases ### Condition Browse Module - Browse Leaves - ID: M9013 - Name: Glaucoma - Relevance: HIGH - As Found: Glaucoma - ID: M10024 - Name: Hypertension - Relevance: LOW - As Found: Unknown - ID: M12731 - Name: Ocular Hypertension - Relevance: LOW - As Found: Unknown - ID: M8271 - Name: Eye Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000005901 - Term: Glaucoma ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06356779 **Acronym:** OLIGO-DK **Brief Title:** Longitudinal Study of Local Ablative Therapy in Oligometastatic Disease **Official Title:** A National Longitudinal Study of Metastases-directed Local Ablative Therapy for Patients With Oligometastatic Disease - a Combined Interventional and Observational Trial #### Organization Study ID Info **ID:** H-23066725 #### Organization **Class:** OTHER **Full Name:** Herlev Hospital ### Status Module #### Completion Date **Date:** 2035-12-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-04-16 **Type:** ACTUAL **Last Update Submit Date:** 2024-04-15 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2030-12-31 **Type:** ESTIMATED #### Start Date **Date:** 2024-04-15 **Type:** ACTUAL **Status Verified Date:** 2024-04 #### Study First Post Date **Date:** 2024-04-10 **Type:** ACTUAL **Study First Submit Date:** 2024-03-25 **Study First Submit QC Date:** 2024-04-04 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Gitte Fredberg Persson MD PhD #### Responsible Party **Investigator Affiliation:** Herlev Hospital **Investigator Full Name:** Gitte Fredberg Persson MD PhD **Investigator Title:** Associate Professor, Chief PhysicianGitte Fredberg Persson MD PhD **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** This prospective national multicenter observational and interventional study aims to assess the longitudinal disease trajectory of patients with oligometastatic disease (OMD) who receive local metastasis-directed therapy. Patients with any category of OMD from any non-hematological cancer are eligible for inclusion. Local ablative therapy (LAT) includes surgical metastasectomy, radiotherapy, thermal ablation, and electroporations. The primary objective is to assess the time to failure of LAT strategy in patients with OMD from any primary cancer treated with all LAT modalities. **Detailed Description:** Patients with oligometastatic disease (OMD) are often treated with a combination of surgery, stereotactic radiotherapy, thermal ablations, or electroporation, either concurrently or in succession, however, most studies are focused on a single modality. In addition, local differences in the use of local ablative therapy (LAT) in different metastatic sites and diseases exist and may impact outcomes for patients with OMD. OLIGO-DK is designed to address these shortcomings. The aim is to offer LAT with any modality to all patients with OMD from all primary cancer histologies and in all metastatic sites, where it is deemed clinically relevant, within the framework of a national prospective multicenter study, combining both standard and non-standard LAT of OMD in an observational and an interventional cohort, respectively. At the same time, we aim to assess the longitudinal treatment trajectory of oligometastatic patients and create a national network for radiotherapy of oligometastases. Finally, we aim to create a clinically applicable prediction model for patient selection. The trial is a national, prospective, multicentre trial. Patients with both genuine and induced non-hematological OMD who are receiving metastases-directed local ablative therapy are included, and all LAT modalities of all metastatic sites from all primary cancers are included. The trial will include both an observational cohort and an interventional cohort. The observational cohort will include patients with OMD who are treated with LAT, which is considered standard-of-care according to national guidelines. The interventional cohort will include patients who are treated with implemented LAT techniques but for indications that are not considered standard-of-care. The final decision on treatment choice is made by the treating physician in consultation with the patient, and the patient may be referred across regional borders for specific treatments. This trial is not on its own designed for the evaluation of novel or experimental LAT techniques, where safety is a primary concern. In these cases, a separate ethical approval protocol is necessary. Patients can still be included in the OLIGO-DK protocol for prospective data collection. In addition, inclusion in this protocol does not impede patients from inclusion in other oligometastatic protocols. Patients are prospectively included, followed, and evaluated by the Centralised Trial Unit and remain included for follow-up until death or patient preference. Due to the nature of oligometastatic disease, patients may receive LAT more than once in the protocol, if the disease is amenable to further local ablative therapy. The trial will initiate accrual in the Capital Region of Denmark, with subsequent expansion after the first interim analysis. A national OMD MDT conference and a nationwide overview of LAT options will be established during the trial. All departments of oncology, and their associated departments of surgery and interventional radiology performing LAT will be able to include patients. ### Conditions Module **Conditions:** - Oligometastatic Disease - Metastases - Ablation Techniques - Radiotherapy - Stereotactic Radiation - Surgery **Keywords:** - oligometastatic disease - metastases-directed therapy - metastasectomy - thermal ablation - stereotactic ablative radiotherapy - stereotactic radiosurgery - endpoints ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 1200 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Discussion at multidisciplinary team conferences Lesion-specific treatment plan with allocation to * surgical metastasectomy * stereotactic radiotherapy * thermal ablation * electroporation **Intervention Names:** - Procedure: Local ablative therapy (LAT) **Label:** Local ablative therapy **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Local ablative therapy **Description:** Surgical metastasectomy, stereotactic ablative radiotherapy, thermal ablation, or electroporation to all oligometastatic lesions **Name:** Local ablative therapy (LAT) **Other Names:** - Metastases-directed therapy **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Description:** Defined as time from first day of LAT to progression of disease, locally or metastatically, not amenable to new LAT or progression of disease leading to initiation of or change in systemic treatment **Measure:** Time to failure of local ablative therapy (LAT) strategy **Time Frame:** Assessed every 3-6 months for 5 years or life-long #### Secondary Outcomes **Description:** Defined as time from first day of LAT to disease progression at any site or death **Measure:** Progression-free survival **Time Frame:** Assessed every 3-6 months for 5 years or life-long **Description:** Defined as time from first day of LAT to disease progression not amenable to new LAT. Deaths from any cause are censored. Initiation of or change in systemic treatment is ignored **Measure:** Time to widespread progression **Time Frame:** Assessed every 3-6 months for 5 years or life-long **Description:** Defined as time from first day of LAT to initiation of systemic treatment, change in systemic treatment, or end of systemic treatment due to progression. Change in or end of systemic treatment due to toxicity is ignored **Measure:** Freedom from systemic treatment **Time Frame:** Assessed every 3-6 months for 5 years or life-long **Description:** Defined as time from first day of first LAT to death from any cause **Measure:** Overall survival **Time Frame:** Assessed every 3-6 months for 5 years or life-long **Description:** Defined as time from first day of LAT to progression of the disease. Deaths from any cause are censored **Measure:** Time to progression **Time Frame:** Assessed every 3-6 months for 5 years or life-long **Description:** Defined as time from first day of LAT to progression within the treated area. In case of doubt or disagreement, the case is reviewed at the local MDT conference. Deaths from any cause are censored **Measure:** Time to local progression **Time Frame:** 5 years or life-long **Description:** Fractions of treated lesions which have not locally progressed at 1- and 3-years after local ablative therapy. Analysed per lesion, per patient, per treatment modality and per organ **Measure:** Local lesion control rate at 1- and 3-years post-local ablative therapy **Time Frame:** 3 years **Description:** Defined as time from first day of LAT to progression outside the treatment field. Deaths from any cause are censored. **Measure:** Time to distant progression **Time Frame:** Assessed every 3-6 months for 5 years or life-long **Description:** LAT related toxicity is defined as adverse events which are categorized by the local investigator as suspected expected or suspected unexpected due to LAT **Measure:** Investigator reported grade 3-5 CTCAE (v.5.0) LAT related toxicity **Time Frame:** 2 years **Description:** Defined as LAT-related toxicity which occurs or is worsened within 3- months of end-of-treatment (EOT). LAT-related toxicity, which occurs or is worsened after the commencement of the LAT course but before EOT, is also registered as early toxicity. **Measure:** Harms **Time Frame:** Actively every 3-months for 2 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Histology or cytology proven non-haematological cancer * Stage IV disease * ECOG performance status ≤ 2 * Life expectancy \> 6 months * A baseline scan within 42 days of inclusion (PET-CT or CT or MRI scan) is required, preferably within 28 days for optimal prospective evaluation * Primary tumor must be controlled, defined by the radiographical response of the primary tumor by systemic or local treatment. If progressing, it is planned to be treated with local ablative therapy (LAT) * Oligometastatic disease according to the ESTRO-EORTC classification, both de-novo and induced, including oligoprogression * A maximum of five oligometastases or oligopersistent/oligoprogressive lesions. More than five metastases are allowed in the following cases, 1) location in a defined anatomical entity or 2) location in immediate proximity and as such, cannot be treated separately * All oligometastatic lesions must be planned for definitive LAT. If all visible/progressive/persistent disease is not treated, the patient cannot be included * Local ablative therapy must be deemed clinically relevant for the individual patient by the treating team of physicians, or a multidisciplinary team and discussion must be documented in the patient chart * Ability to understand and the willingness to sign a written informed consent document Exclusion Criteria: * Pregnancy * Diffuse cancer disease, which cannot be locally ablated, i.e., leptomeningeal carcinomatosis, malignant pleural effusions, lymphangitic carcinomatosis, or peritoneal carcinomatosis * If LAT is deemed unsafe by the MDT (e.g., tumor perforation of hollow organs) In addition, the patients receiving SBRT to oligometastatic sites should comply with the following criteria. * The size of the target is limited by the ability to safely deliver locally ablative doses to the metastatic lesions. Generally, an upper limit of 5 cm is recommended * If the patient has received previous radiotherapy, the combined dose at the radiation site must not exceed the dose constraints according to Appendix 2 - Radiotherapy Recommendations **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Michael RT Laursen, MD **Phone:** +45 3868 9202 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Gitte F Persson **Phone:** +453868 9299 **Role:** CONTACT #### Locations **Location 1:** **City:** Copenhagen **Contacts:** ***Contact 1:*** - **Name:** Mette Pøhl, MD PhD - **Role:** CONTACT ***Contact 2:*** - **Name:** Mette Pøhl, MD PhD - **Role:** PRINCIPAL_INVESTIGATOR ***Contact 3:*** - **Name:** Maja Maraldo, MD PhD - **Role:** SUB_INVESTIGATOR ***Contact 4:*** - **Name:** Morten Suppli, MD PhD - **Role:** SUB_INVESTIGATOR ***Contact 5:*** - **Name:** Rene H Petersen, MD PhD - **Role:** SUB_INVESTIGATOR ***Contact 6:*** - **Name:** Hans-Christian Pommergaard, MD PhD - **Role:** SUB_INVESTIGATOR ***Contact 7:*** - **Name:** Søren Møller, MD PhD - **Role:** SUB_INVESTIGATOR ***Contact 8:*** - **Name:** Michael Achiam, MD PhD - **Role:** SUB_INVESTIGATOR ***Contact 9:*** - **Name:** Mikkel Rosendahl, MD PhD - **Role:** SUB_INVESTIGATOR **Country:** Denmark **Facility:** Copenhagen University Hospital Rigshospitalet **State:** Capital Region Of Denmark **Status:** NOT_YET_RECRUITING **Zip:** 2100 **Location 2:** **City:** Herlev **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Michael RT Laursen, MD - **Phone:** +453868 9202 - **Role:** CONTACT ***Contact 2:*** - **Name:** Mette Felter, MD PhD - **Role:** SUB_INVESTIGATOR ***Contact 3:*** - **Name:** Henriette Lindberg, MD PhD - **Role:** PRINCIPAL_INVESTIGATOR ***Contact 4:*** - **Name:** Eva Serup-Hansen, MD PhD - **Role:** SUB_INVESTIGATOR ***Contact 5:*** - **Name:** Sebastian Krog, MD - **Role:** SUB_INVESTIGATOR ***Contact 6:*** - **Name:** Jesper Palshof, MD PhD - **Role:** SUB_INVESTIGATOR ***Contact 7:*** - **Name:** Bodil Engelmann, MD PhD - **Role:** SUB_INVESTIGATOR ***Contact 8:*** - **Name:** Eva Ellebæk, MD PhD - **Role:** SUB_INVESTIGATOR ***Contact 9:*** - **Name:** Lisbet Hölmich, MD PhD - **Role:** SUB_INVESTIGATOR **Country:** Denmark **Facility:** Copenhagen University Hospital Herlev and Gentofte **State:** Capital Region Of Denmark **Status:** RECRUITING **Zip:** 2730 **Location 3:** **City:** Hillerød **Contacts:** ***Contact 1:*** - **Name:** Maria Lendorf - **Role:** CONTACT ***Contact 2:*** - **Name:** Maria Lendorf, MD PD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Denmark **Facility:** Hillerød Hospital **State:** Capital Region Of Denmark **Status:** NOT_YET_RECRUITING **Zip:** 3400 **Location 4:** **City:** Aarhus **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Mette Marie Fode, MD PhD - **Role:** CONTACT ***Contact 2:*** - **Name:** Mette Marie Fode, MD PhD - **Role:** PRINCIPAL_INVESTIGATOR ***Contact 3:*** - **Name:** Azza Khalil, MD PhD - **Role:** SUB_INVESTIGATOR ***Contact 4:*** - **Name:** Thomas Decker Christensen, MD PhD - **Role:** SUB_INVESTIGATOR ***Contact 5:*** - **Name:** Ole Graumann, MD PhD - **Role:** SUB_INVESTIGATOR ***Contact 6:*** - **Name:** Jørgen Bjerggaard Jensen, MD PhD - **Role:** SUB_INVESTIGATOR **Country:** Denmark **Facility:** Aarhus University Hospital **State:** Central Denmark Region **Status:** NOT_YET_RECRUITING **Zip:** 8200 **Location 5:** **City:** Herning **Contacts:** ***Contact 1:*** - **Name:** Trine Øllegaard - **Role:** CONTACT ***Contact 2:*** - **Name:** Trine Øllegaard, MD PhD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Denmark **Facility:** Gødstrup Hospital **State:** Central Denmark Region **Status:** NOT_YET_RECRUITING **Zip:** 7400 **Location 6:** **City:** Aarhus **Contacts:** ***Contact 1:*** - **Name:** Britte Weber, MD PhD - **Role:** CONTACT ***Contact 2:*** - **Name:** Britta Weber, MD PhD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Denmark **Facility:** Danish Center for Particle Therapy **State:** Central Region Denmark **Status:** NOT_YET_RECRUITING **Zip:** 8200 **Location 7:** **City:** Aalborg **Contacts:** ***Contact 1:*** - **Name:** Jimmy Søndergaard, MD PhD - **Role:** CONTACT ***Contact 2:*** - **Name:** Laurids Ø Poulsen, MD PhD - **Role:** CONTACT ***Contact 3:*** - **Name:** Jimmi Søndergaard, MD PhD - **Role:** PRINCIPAL_INVESTIGATOR ***Contact 4:*** - **Name:** Laurids Ø Poulsen, MD PhD - **Role:** SUB_INVESTIGATOR ***Contact 5:*** - **Name:** Hella Sand, MSc - **Role:** SUB_INVESTIGATOR **Country:** Denmark **Facility:** Aalborg University Hospital **State:** Northern Region Of Denmark **Status:** NOT_YET_RECRUITING **Zip:** 9000 **Location 8:** **City:** Odense **Contacts:** ***Contact 1:*** - **Name:** Tine Schytte, MD PhD - **Role:** CONTACT ***Contact 2:*** - **Name:** Tine Schytte, MD PhD - **Role:** PRINCIPAL_INVESTIGATOR ***Contact 3:*** - **Name:** Jørgen Johansen, MD PhD - **Role:** SUB_INVESTIGATOR ***Contact 4:*** - **Name:** Christina Nyborg, MD PhD - **Role:** SUB_INVESTIGATOR **Country:** Denmark **Facility:** Odense University Hospital **State:** Southern Denmark Region **Status:** NOT_YET_RECRUITING **Zip:** 5000 **Location 9:** **City:** Sønderborg **Country:** Denmark **Facility:** Sønderborg Hospital **State:** Southern Denmark Region **Status:** NOT_YET_RECRUITING **Zip:** 6400 **Location 10:** **City:** Vejle **Contacts:** ***Contact 1:*** - **Name:** Charlotte Kristiansen, MD - **Role:** CONTACT ***Contact 2:*** - **Name:** Charlotte Kristiansen, MD - **Role:** PRINCIPAL_INVESTIGATOR ***Contact 3:*** - **Name:** Lars Fokdal, MD PhD - **Role:** SUB_INVESTIGATOR ***Contact 4:*** - **Name:** Lise Bentzen, MD PhD - **Role:** SUB_INVESTIGATOR **Country:** Denmark **Facility:** Vejle Hospital **State:** Southern Denmark Region **Status:** NOT_YET_RECRUITING **Zip:** 7100 **Location 11:** **City:** Roskilde **Contacts:** ***Contact 1:*** - **Name:** Julie Gehl, MD DMSc - **Role:** CONTACT ***Contact 2:*** - **Name:** Julie Gehl, MD DMSc - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Denmark **Facility:** Zealand University Hospital, Roskilde and Næstved **State:** Zealand Region **Status:** NOT_YET_RECRUITING **Zip:** 4000 #### Overall Officials **Official 1:** **Affiliation:** Copenhagen University Hospital Herlev and Gentofte **Name:** Gitte F Persson, MD PhD **Role:** STUDY_CHAIR **Official 2:** **Affiliation:** Copenhagen University Hospital Herlev and Gentofte **Name:** Michael RT Laursen, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Access Criteria:** No specific access criteria **Description:** Disease specific individual participant data will be available to the Danish Multidisciplinary Cancer Groups. Modality specific individual participant data will be available for separate analysis. **Info Types:** - STUDY_PROTOCOL - SAP **IPD Sharing:** YES **Time Frame:** The study protocol and the first draft of the SAP will be shared and available after the publication of the protocol article. ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009385 - Term: Neoplastic Processes - ID: D000009369 - Term: Neoplasms - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M12307 - Name: Neoplasm Metastasis - Relevance: HIGH - As Found: Metastases - ID: M12330 - Name: Neoplastic Processes - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000009362 - Term: Neoplasm Metastasis ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05251779 **Brief Title:** A Clinical Study on the Effects of Inhalation of Volatile Oil of Cang-Ai Via the Nose on Patients With Depression **Official Title:** A Clinical Study on the Effects of Inhalation of Volatile Oil of Cang-Ai Via the Nose on Patients With Depression #### Organization Study ID Info **ID:** YunnanUCM #### Organization **Class:** OTHER **Full Name:** Yunnan University of Chinese Medicine ### Status Module #### Completion Date **Date:** 2022-12-01 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2022-10-20 **Type:** ACTUAL **Last Update Submit Date:** 2022-10-19 **Overall Status:** NOT_YET_RECRUITING #### Primary Completion Date **Date:** 2022-12-01 **Type:** ESTIMATED #### Start Date **Date:** 2022-11-01 **Type:** ESTIMATED **Status Verified Date:** 2022-10 #### Study First Post Date **Date:** 2022-02-23 **Type:** ACTUAL **Study First Submit Date:** 2022-01-24 **Study First Submit QC Date:** 2022-02-22 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Jieqiong Cui #### Responsible Party **Investigator Affiliation:** Yunnan University of Chinese Medicine **Investigator Full Name:** Jieqiong Cui **Investigator Title:** Doctoral students **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** In this study, 60 depressed patients who meet the criteria will randomly be divided into a test group and a control group, with 30 patients in each group. The test group will be given Volatile oil of Cang-Ai (hereafter referred to as CAVO) for inhalation and the control group will be given Bergamot for inhalation. The observation indicators are that after one and two sessions of the intervention, the patients' blood pressure, heart rate, depression scale scores, and changes in functional near-infrared spectroscopy(fNIRS). This randomized controlled trial will be used to look at the clinical efficacy of CAVO in patients with depression. **Detailed Description:** Depression is a common psychiatric disorder characterised by persistent depressed mood, loss of interest and feelings of helplessness. Depression has the highest burden of illness of any mental illness and is the leading cause of disability. Currently, depression is usually treated with antidepressants as the first line of treatment, but because the pathophysiological mechanisms of depression are still unclear, the mechanisms of antidepressant treatment are unknown, and there is no objective way to predict efficacy, depression often requires multiple "trial and error treatments" before an effective treatment plan can be determined. Chemically synthesised drugs are widely used to treat depression, but side effects have become a bottleneck to their long-term use. As a result, natural products from medicinal plants, such as Essential oils or Volatile oils, have become research targets for the development of new drugs. Volatile oil of Cang-Ai is a component extracted from aromatic Chinese herbs such as Atractlodis Rhizoma, Herba Agastaches, Flos Caryophylli, which is commonly used clinically in the treatment of mood disorders. This study will use a randomised controlled study method. 60 patients with depression who attended the First Affiliated Hospital of Yunnan University of Traditional Chinese Medicine from March 2022 to September 2022 will be recruited. They will be randomly divided into a trial group and a control group, 30 patients in each group. The test group will be given Volatile oil of Cang-Ai for inhalation and the control group will be given Bergamot for inhalation. Ultimately, a number of indicators will be tested to assess the clinical efficacy of the volatile oil of Cang-Ai. ### Conditions Module **Conditions:** - Depressive Disorder - Mental Disorder in Adolescence **Keywords:** - Aromatic Herbs - Volatile oils - Inhalation via the nose ### 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:** 60 **Type:** ESTIMATED **Phases:** - EARLY_PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** The test group inhaled the volatile oil of Cang-Ai at a concentration of 1% by inhalation once a day for 30 minutes **Intervention Names:** - Drug: Volatile Oil of Cang-Ai **Label:** Cang-Ai Group **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** The control group inhaled the bergamot essential oils at a concentration of 1% by inhalation once a day for 30 minutes **Intervention Names:** - Drug: Bergamot essential oils **Label:** Bergamot group **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Cang-Ai Group **Description:** The test group will be given Volatile oil of Cang-Ai (hereafter referred to as CAVO) for inhalation **Name:** Volatile Oil of Cang-Ai **Other Names:** - CAVO **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Bergamot group **Description:** The control group will be given Bergamot essential oils for inhalation. **Name:** Bergamot essential oils **Other Names:** - Citrus bergamia **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Functional near-infrared spectroscopy to measure changes in the relative concentrations of Oxy-Hb and Deoxy-Hb in the cerebral cortex (prefrontal and temporal lobes) **Measure:** Change from functional near-infrared spectroscopy（fNIRS) at 28 days **Time Frame:** At the end of Cycle 1 (each cycle is 14 days) **Description:** Functional near-infrared spectroscopy to measure changes in the relative concentrations of Oxy-Hb and Deoxy-Hb in the cerebral cortex (prefrontal and temporal lobes) **Measure:** Change from functional near-infrared spectroscopy（fNIRS) at 28 days **Time Frame:** At the end of Cycle 2 (each cycle is 14 days) **Description:** The following severity ranges for the HAMD: no depression (0-7); mild depression (8-16); moderate depression (17-23); and severe depression (≥24) **Measure:** 24-item Hamilton Depression Inventory scores **Time Frame:** At the end of Cycle 1 (each cycle is 14 days) **Description:** The following severity ranges for the HAMD: no depression (0-7); mild depression (8-16); moderate depression (17-23); and severe depression (≥24) **Measure:** 24-item Hamilton Depression Inventory scores **Time Frame:** At the end of Cycle 2 (each cycle is 14 days) #### Secondary Outcomes **Description:** Patients' blood pressure will be measured by a blood pressure monitor **Measure:** Change from blood pressure **Time Frame:** At the end of Cycle 1 (each cycle is 14 days) **Description:** Patients' blood pressure will be measured by a blood pressure monitor **Measure:** Change from blood pressure at 28 days **Time Frame:** At the end of Cycle 2 (each cycle is 14 days) **Description:** Patients' heart rate will be measured by a heart rate monitor **Measure:** Change from heart rate **Time Frame:** At the end of Cycle 1 (each cycle is 14 days) **Description:** Patients' heart rate will be measured by a heart rate monitor **Measure:** Change from heart rate at 28 days **Time Frame:** At the end of Cycle 2 (each cycle is 14 days) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Meeting the Diagnostic and Statistical Manual of Mental Disorders (DSM-5) diagnostic criteria for depressive episodes. * Beck Depression Scale \>10; 24-item Hamilton Depression Scale (HAMD) score \>20 * Healthy sense of smell, no allergic diseases, respiratory diseases, age 16-40 years, male or female. * No use of antidepressants and other psychiatric drugs or physiotherapy such as electroconvulsive shock or transcranial magnetic stimulation (TMS) for at least 1 week prior to the intervention ⑤All subjects volunteered to participate and signed an informed consent form after approval by the hospital's ethics committee Exclusion Criteria: * History of schizophrenia, alcohol and drug dependence strictly excluded ②Depression with a history of organic brain disease and endocrine disorders or secondary to other psychiatric disorders * Score \>3 on the Hamilton Depression Inventory for Suicide ④Pregnant and breastfeeding women with a history of manic or hypomanic episodes ⑤Family history of monophasic or bipolar disorder **Maximum Age:** 28 Years **Minimum Age:** 6 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT ### IPD Sharing Statement Module **Access Criteria:** Sharing with researcher for Meta-analysis **Description:** The original data from each eligible study. including de-identified demographic information for each participant such as age, sex, nature of their health condition, as well as information about treatments or tests received and outcomes observed **Info Types:** - STUDY_PROTOCOL - SAP - ICF - CSR **IPD Sharing:** YES **Time Frame:** After Dec 2022 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000019964 - Term: Mood Disorders - ID: D000012120 - Term: Respiration Disorders - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: All - Name: All Conditions - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M7058 - Name: Depression - Relevance: LOW - As Found: Unknown - ID: M7061 - Name: Depressive Disorder - Relevance: HIGH - As Found: Depressive Disorder - ID: M27137 - Name: Respiratory Aspiration - Relevance: HIGH - As Found: Inhalation - ID: M4815 - Name: Mental Disorders - Relevance: HIGH - As Found: Mental Disorders - ID: M14473 - Name: Psychotic Disorders - Relevance: LOW - As Found: Unknown - ID: M21835 - Name: Mood Disorders - Relevance: LOW - As Found: Unknown - ID: M14957 - Name: Respiration Disorders - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000053120 - Term: Respiratory Aspiration - ID: D000003866 - Term: Depressive Disorder - ID: D000001523 - Term: Mental Disorders ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01340079 **Brief Title:** Virtual World Health Behavior Counseling for Patients With Diabetes **Official Title:** Virtual World Health Behavior Counseling for Patients With Diabetes #### Organization Study ID Info **ID:** 1RC1LM010412-01 **Link:** https://reporter.nih.gov/quickSearch/1RC1LM010412-01 **Type:** NIH #### Organization **Class:** OTHER **Full Name:** Boston Medical Center ### Status Module #### Completion Date **Date:** 2012-07 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2012-09-24 **Type:** ESTIMATED **Last Update Submit Date:** 2012-09-20 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2012-07 **Type:** ACTUAL #### Start Date **Date:** 2011-04 **Status Verified Date:** 2012-05 #### Study First Post Date **Date:** 2011-04-22 **Type:** ESTIMATED **Study First Submit Date:** 2011-03-16 **Study First Submit QC Date:** 2011-04-20 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** University of Massachusetts, Worcester #### Lead Sponsor **Class:** OTHER **Name:** Boston Medical Center #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** Comparison of 2 methods of delivery health education for African-American women with Type 2 Diabetes. The two methods are: face to face education in small groups, as compared to delivering the same curriculum in the virtual world, Second Life. **Detailed Description:** This study will provide health education to African-American women with type 2 diabetes either face to face, or using the Internet. The study seeks to determine how feasible the internet method is for this type of health education, and how it compares to the face to face method. Patients will be recruited from Boston Medical Center. They will do baseline surveys and have blood drawn. They will then be randomized into receiving their diabetes education in a face to face group at BMC, or receiving it online while at home. The online group will receive a computer to access the Internet program and Internet access. Both groups will participate in 8 diabetes education groups, and 4 individual counseling sessions. Subjects will complete surveys before and after the study to measure changes in physical activity, diet, and use of medications, and will have blood tests drawn at BMC before and after the study to measure changes in diabetes control, cholesterol. They will also have blood pressure measured before and after the study period. ### Conditions Module **Conditions:** - Type 2 Diabetes **Keywords:** - diabetes mellitus, non-insulin dependent ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** HEALTH_SERVICES_RESEARCH #### Enrollment Info **Count:** 89 **Type:** ACTUAL **Phases:** - PHASE1 - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Virtual world delivery method **Intervention Names:** - Behavioral: virtual world **Label:** Virtual world **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** face to face method of health education **Intervention Names:** - Behavioral: face to face **Label:** face to face **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Virtual world **Description:** health education using virtual world Second Life **Name:** virtual world **Type:** BEHAVIORAL #### Intervention 2 **Arm Group Labels:** - face to face **Description:** health education delivered in face to face groups **Name:** face to face **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Measure:** Change from Baseline in hemoglobin a1c at 8 weeks after completion of the intervention. **Time Frame:** Baseline and 8 weeks after completion of the intervention #### Secondary Outcomes **Measure:** Change from Baseline in dietary patterns at 8 weeks after completion of the intervention. **Time Frame:** Baseline and 8 weeks after completion of the intervention **Measure:** Change from Baseline in physical activity at 8 weeks after completion of the intervention. **Time Frame:** Baseline and 8 weeks after completion of the intervention ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Diagnosed with T2DM 2. HbA1c level \>= 8.0 3. Currently treated with diet, oral hypoglycemics or insulin. If currently on insulin, must have a history of prior therapy with diet alone or oral hypoglycemic agents 4. African-American origin 5. \>= 18 years old 6. Telephone in home or easy access to one 7. Able to understand and participate in the study protocol 8. Functionally capable of meeting the activity goals 9. Understands and can provide informed consent 10. Physician approval to participate in the study. Exclusion Criteria: 1. History of diabetic ketoacidosis 2. Gestational diabetes 3. Unable or unwilling to provide informed consent 4. Plans to move out of the area within the 12-month study period 5. Required intermittent glucocorticoid therapy within the past 3 months 6. Experienced an acute coronary event (myocardial infarction or unstable angina) within the previous 6 months 7. Has a medical condition that precludes adherence to study dietary recommendations (e.g., Crohn's disease, ulcerative colitis, end-stage renal disease) 8. Has a medical or psychiatric illness (i.e., dementia, psychiatric hospitalization or suicidality within past 5 years or takes an neuroleptic medication). We will not exclude based on history of depression or anxiety or taking anti-depressants or anti-anxiety medications. **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Boston **Country:** United States **Facility:** Boston Medical Center **State:** Massachusetts **Zip:** 02118 #### Overall Officials **Official 1:** **Affiliation:** Boston Medical Center/Boston University Medical Campus **Name:** John M. Wiecha, MD, MPH **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** University of Massachusetts, Worcester, MA **Name:** Milagros Rosal, PhD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Rosal MC, Heyden R, Mejilla R, Capelson R, Chalmers KA, Rizzo DePaoli M, Veerappa C, Wiecha JM. A Virtual World Versus Face-to-Face Intervention Format to Promote Diabetes Self-Management Among African American Women: A Pilot Randomized Clinical Trial. JMIR Res Protoc. 2014 Oct 24;3(4):e54. doi: 10.2196/resprot.3412. **PMID:** 25344620 ## 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: LOW - As Found: Unknown - ID: M11639 - Name: Metabolic Diseases - Relevance: LOW - As Found: Unknown - ID: M25403 - Name: Glucose Metabolism Disorders - Relevance: LOW - As Found: Unknown - ID: M7862 - Name: Endocrine System Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000003920 - Term: Diabetes Mellitus ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01466179 **Brief Title:** Clinical Study With Blinatumomab in Patients With Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia (ALL) **Official Title:** An Open Label, Multicenter, Phase II Study to Evaluate Efficacy and Safety of the BiTE® Antibody Blinatumomab in Adult Patients With Relapsed/Refractory B-precursor Acute Lymphoblastic Leukemia (ALL) #### Organization Study ID Info **ID:** MT103-211 #### Organization **Class:** INDUSTRY **Full Name:** Amgen Research (Munich) GmbH #### Secondary ID Infos **ID:** 2011-002257-61 **Type:** EUDRACT_NUMBER ### Status Module #### Completion Date **Date:** 2017-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-08-18 **Type:** ACTUAL **Last Update Submit Date:** 2017-07-18 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2013-10 **Type:** ACTUAL #### Results First Post Date **Date:** 2015-01-22 **Type:** ESTIMATED **Results First Submit Date:** 2014-12-21 **Results First Submit QC Date:** 2015-01-20 #### Start Date **Date:** 2011-12 **Status Verified Date:** 2017-07 #### Study First Post Date **Date:** 2011-11-06 **Type:** ESTIMATED **Study First Submit Date:** 2011-10-28 **Study First Submit QC Date:** 2011-11-04 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Amgen Research (Munich) GmbH #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of this study is to confirm whether the bispecific T cell engager antibody blinatumomab (MT103) is effective and safe in the treatment of patients with relapsed or refractory Acute Lymphoblastic Leukemia (ALL). **Detailed Description:** Relapsed/refractory B-precursor ALL in adult patients is an aggressive malignant disease with dismal prognosis. Several studies have reported long term survival to be below 10%. Major prognostic factors are duration of first complete remission (CR1) and age. With current salvage chemotherapy, complete remission (CR) rate is low (20 to 30%) in patients in first salvage with short duration (\< one year) of first remission, patients relapsed after first salvage, or patients aged 60 years and older. Duration of CR is usually very short (median disease free survival \[DFS\]: 2.0-7.5 months). Allogeneic hematopoietic stem cell transplantation (HSCT) may provide a curative treatment option for patients in CR with a satisfactory donor and appropriate clinical status including age, organ function, and remission status. Allogeneic HSCT is not an option in most elderly patients with relapsed ALL. Additional therapeutic approaches are urgently needed. Blinatumomab is a bispecific single-chain antibody derivative against CD (cluster of differentiation)19 and CD3, designed to link B cells and T cells resulting in T cell activation and a cytotoxic T cell response against CD19-expressing cells. In vitro data indicate CD19+ lymphoma and leukemia cell lines to be extremely sensitive to blinatumomab-mediated cytotoxicity. Blinatumomab has the potential to provide meaningful therapeutic benefits to patients compared with existing treatments for this patient population. This study consists of a screening period, a treatment period and a follow-up period. Participants receive one to five treatment cycles of blinatumomab at a target dose of 28 μg/day. In the first cycle, the initial dose is 9 μg/day for the first seven days of treatment, escalated to 28 μg/day starting from Week 2 of treatment. Participants who achieve remission within two cycles of treatment can receive up to three additional cycles of consolidation treatment or proceed to allogeneic HSCT. In the event of progression or relapse within the treatment period, treatment will be terminated. Participants with hematological relapse during the efficacy or safety follow-up period may receive up to three additional cycles of blinatumomab (retreatment) for a maximal total of eight cycles at the investigator´s discretion. Thirty days after end of the last treatment, participants have an end-of-core-study visit. Following this, there are efficacy follow-up visits at 3, 6, 9, 12, 18 and 24 months at the most after treatment start. Once efficacy follow-up is complete, information on survival collected at least every six months until death or at least until three years after treatment start, whichever occurs earlier (survival follow-up). ### Conditions Module **Conditions:** - Acute Lymphoblastic Leukemia **Keywords:** - B-ALL - relapsed ALL - refractory ALL - adult ALL - Leukemia - Leukemia, Lymphoid - precursor cell lymphoblastic leukemia-lymphoma - Lymphatic diseases - Lymphoproliferative disorders - bispecific antibody - anti-CD19 - Immunotherapeutic treatment - immunoproliferative disorders ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 225 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants received blinatumomab by continuous intravenous (CIV) infusion over 4 weeks followed by a treatment-free interval of 2 weeks for up to 5 consecutive cycles. The initial dose was 9 μg/day for the first seven days of treatment, escalated to 28 μg/day starting from Week 2 of treatment. **Intervention Names:** - Biological: Blinatumomab **Label:** Blinatumomab **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Blinatumomab **Description:** Continuous intravenous infusion over four weeks per treatment cycle **Name:** Blinatumomab **Other Names:** - AMG103 - MT103 - BLINCYTO™ **Type:** BIOLOGICAL ### Outcomes Module #### Primary Outcomes **Description:** Hematological assessments were performed from bone marrow biopsy samples. All hematological assessments of bone marrow were reviewed in a central reference laboratory. Hematological remissions were defined by the following criteria: Complete Remission (CR): * bone marrow blasts ≤ 5% * no evidence of disease * full recovery of peripheral blood counts: * platelets \> 100,000/μL, and * absolute neutrophil count (ANC) \> 1,000/μL Complete Remission With Partial Hematological Recovery (CRh\*): * bone marrow blasts ≤ 5% * no evidence of disease * partial recovery of peripheral blood counts: * platelets \> 50,000/μL, and * ANC \> 500/μL. **Measure:** Percentage of Participants With a Best Response of Complete Remission or Complete Remission With Only Partial Hematological Recovery Within 2 Cycles of Treatment **Time Frame:** Within the first 2 cycles of treatment, 12 weeks #### Secondary Outcomes **Description:** Time to hematological relapse was measured for participants in remission during the core study (the time from the first infusion through 30 days after the last infusion), from the time the participant first achieved remission until first documented relapse or death due to disease progression. Participants without documented relapse (hematological or extramedullary) and who did not die were censored at the time of their last bone marrow assessment or their last survival follow-up visit confirming remission. Participants who died without having reported hematological relapse or without showing any clinical sign of disease progression were censored on their date of death. Hematological relapse is defined as: * proportion of blasts in bone marrow \> 5% after documented CR/CRh\* or * blasts in peripheral blood after documented CR/CRh\*. Time to hematological relapse was analyzed by Kaplan-Meier methods and the median observation time was calculated by the reverse Kaplan Meier method. **Measure:** Time to Hematological Relapse (Duration of Response) **Time Frame:** Up to the data cut-off date of 10 October 2013; median observation time was 8.0 months. **Description:** Participants who were eligible for allogeneic HSCT were those who achieved remission (complete response or complete response with partial recovery of peripheral blood counts) after 2 cycles of blinatumomab treatment, and no further anti-leukemic medication was given before HSCT. **Measure:** Percentage of Participants Who Received an Allogeneic Hematopoietic Stem Cell Transplant (HSCT) During Blinatumomab Induced Remission **Time Frame:** Up to the data cut-off date of 10 October 2013. Maximum duration on study was 17.8 months. **Description:** Complete Remission was defined by the following criteria: * bone marrow blasts ≤ 5% * no evidence of disease * full recovery of peripheral blood counts: * platelets \> 100,000/μL, and * absolute neutrophil count (ANC) \> 1,000/μL **Measure:** Percentage of Participants With a Best Response of Complete Remission Within 2 Cycles of Treatment **Time Frame:** Within the first 2 cycles of treatment, 12 weeks **Description:** Complete Remission With Partial Hematological Recovery was defined by the following criteria: * bone marrow blasts ≤ 5% * no evidence of disease * partial recovery of peripheral blood counts: * platelets \> 50,000/μL, and * ANC \> 500/μL. **Measure:** Percentage of Participants With a Best Response of Complete Remission With Only Partial Hematological Recovery Within 2 Cycles of Treatment **Time Frame:** Within the first 2 cycles of treatment, 12 weeks **Description:** Partial Remission is defined as bone marrow blasts 6% to 25% with at least a 50% reduction from baseline. **Measure:** Percentage of Participants With a Best Response of Partial Remission Within 2 Cycles of Treatment **Time Frame:** Within the first 2 cycles of treatment, 12 weeks **Description:** Relapse-free survival was assessed for participants who achieved a complete remission or complete remission with partial hematological recovery during the core study and was measured from the time the participant first achieved remission until first documented relapse or death due to any cause. Participants without a documented relapse (hematological or extramedullary) or who did not die were censored at the time of their last bone marrow assessment or their last survival follow-up visit confirming remission. Relapse free survival was estimated using Kaplan-Meier methods and the median observation time was calculated by the reverse Kaplan Meier method. **Measure:** Relapse-free Survival **Time Frame:** Up to the data cut-off date of 10 October 2013; median observation time was 8.9 months. **Description:** Event-free survival was calculated from the start date of blinatumomab infusion until the date of bone marrow aspiration at which hematological relapse was first detected, or the date of diagnosis on which the hematological or extramedullary relapse was documented or the date of start of any new therapy for ALL (excluding HSCT), or the date of death, whichever was earlier. Participants who did not achieve complete remission or complete remission with partial hematological recovery during the core study were evaluated as having an event on Day 1. Participants in remission who did not experience hematological relapse, did not receive a new therapy for ALL (excluding HSCT), and did not die were censored on the date of the last available bone marrow aspiration or on the last date of survival follow-up visit, whichever was later. Event free survival was estimated using Kaplan-Meier methods and the median observation time was calculated by the reverse Kaplan Meier method. **Measure:** Event-free Survival **Time Frame:** Up to the data cut-off date of 10 October 2013; median observation time was 9.8 months. **Description:** Overall survival was measured for all participants from the time the participant received the first treatment of blinatumomab until death due to any cause or the date of the last follow-up. Participants who did not die were censored on the last documented visit date or the date of the last phone contact when the patient was last known to have been alive. Overall survival was estimated using Kaplan-Meier methods. The median follow-up time with respect to overall survival was calculated by the reverse Kaplan Meier method. **Measure:** Overall Survival **Time Frame:** Up to the data cut-off date of 10 October 2013; median observation time was 9.8 months. **Description:** Adverse events (AEs) were evaluated for severity according to the the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 4, as follows: Grade 1 - Mild AE; Grade 2 - Moderate AE; Grade 3 - Severe AE; Grade 4 - Life-threatening or disabling AE; Grade 5 - Death. The investigator used medical judgment to determine if there was a causal relationship (ie, related, unrelated) between an adverse event and blinatumomab. An AE was considered "serious" if it resulted in death, was life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant incapacity or substantial disruption to conduct normal life functions, is a congenital anomaly or birth defect or is a medically important condition. Progressive disease was not an adverse event, per the protocol, unless it was more severe than expected for the patient. Therefore, many deaths due to progressive disease were not counted as adverse events. **Measure:** Number of Participants With Treatment-emergent Adverse Events **Time Frame:** From the start of the first infusion to 30 days after the end of the last infusion in the core study or from the start of the first retreatment cycle infusion to 30 days after the end of the last retreatment cycle, median treatment duration was 42.2 days. **Description:** The analysis of 100-day mortality after allogeneic HSCT was assessed for all participants who received an allogeneic HSCT while in remission (CR/CRh\*) following treatment with blinatumomab. 100-day mortality after allogeneic HSCT was calculated relative to the date of allogeneic HSCT. Patients alive were censored on the last documented visit date or the date of the last phone contact when the patient was last known to have been alive. The 100-day mortality rate after allogeneic HSCT was defined as the percentage of patients having died up to 100 days after allogeneic HSCT estimated using the estimated time to death in percent calculated by Kaplan-Meier methods. **Measure:** 100-Day Mortality After Allogeneic Hematopoietic Stem Cell Transplant **Time Frame:** From the date of allogeneic HSCT until the data cut-off date of 10 October 2013; median observation time was 7.4 months. **Description:** The steady state concentration of blinatumomab was summarized as the observed concentrations collected at least 10 hours after the start of the IV infusion or dose step for cycle 1 and cycle 2, respectively. Serum concentrations of blinatumomab were measured using a validated bioassay. The lower limit of quantitation (LLOQ) = 50.0 pg/mL. **Measure:** Serum Blinatumomab Concentration at Steady State **Time Frame:** Samples were taken before treatment start and on Days 3, 8, 10, 15, 22, and 29 after infusion start during Cycles 1 and 2. **Description:** The activation of immune effector cells was monitored by the measurement of peripheral blood cytokine levels including interleukin (IL)-2, IL-4, IL-6, IL-10, tumor necrosis factor (TNF)-α and interferon gamma (IFN)-γ using enzyme-linked immunosorbent assays or cytometric bead assays. The limit of detection of the assay (LOD) was 20 pg/mL and the limit of quantification (LOQ) was 125 pg/mL. Data below LOD were set to 10 pg/mL while data \< LOQ and \> LOD were reported as measured. Serum IL-4 levels were below detection limit (\< 20 pg/mL) at all time points in all participants studied. **Measure:** Serum Cytokine Peak Levels **Time Frame:** Serum samples were collected on Days 1 and 8 at 2 hours and 6 hours after treatment start, and on Day 2 (24 hours) and Day 3 (48 hours) of each treatment cycle and on Days 9 and 10 after dose step. **Description:** Blast Free Hypoplastic or Aplastic Bone Marrow was defined as: * bone marrow blasts ≤ 5% * no evidence of disease * insufficient recovery of peripheral counts: platelets ≤ 50,000/μL and/or absolute neutrophil count (ANC) ≤ 500/μL **Measure:** Percentage of Participants With a Best Response of Blast Free Hypoplastic or Aplastic Bone Marrow Within 2 Cycles of Treatment **Time Frame:** Within the first 2 cycles of treatment, 12 weeks **Description:** Complete Remission (CR): * bone marrow blasts ≤ 5% * no evidence of disease * full recovery of peripheral blood counts: * platelets \> 100,000/μL, and * absolute neutrophil count (ANC) \> 1,000/μL Complete Remission With Partial Hematological Recovery (CRh\*): * bone marrow blasts ≤ 5% * no evidence of disease * partial recovery of peripheral blood counts: * platelets \> 50,000/μL, and * ANC \> 500/μL Blast Free Hypoplastic or Aplastic Bone Marrow: * bone marrow blasts ≤ 5% * no evidence of disease * insufficient recovery of peripheral counts: platelets ≤ 50,000/μL and/or ANC ≤ 500/μL Partial Remission: • bone marrow blasts 6% to 25% with at least a 50% reduction from Baseline. **Measure:** Best Response During the Core Study **Time Frame:** From the first dose of blinatumomab until 30 days after the end of the last infusion during the core study, or until the data cut-off date of 10 October 2013; a maximum of 7.5 months. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Patients with Philadelphia chromosome (Ph)-negative B-precursor ALL, with any of the following: * relapsed or refractory with first remission duration less than or equal to 12 months in first salvage or * relapsed or refractory after first salvage therapy or * relapsed or refractory within 12 months of allogeneic hematopoietic stem cell transplantation (HSCT) * 10% or more blasts in bone marrow * In case of clinical signs of additional extramedullary disease: measurable disease * Eastern Cooperative Oncology Group (ECOG) performance status ≤ 2 * Age ≥ 18 years Exclusion Criteria: * Patients with Ph-positive ALL * Patients with Burkitt's Leukemia according to World Health organization (WHO) classification * History or presence of clinically relevant central nervous system (CNS) pathology * Active ALL in the CNS or testes * Current autoimmune disease or history of autoimmune disease with potential CNS involvement * Autologous HSCT within six weeks prior to start of blinatumomab treatment * Allogeneic HSCT within three months prior to start of blinatumomab treatment * Any active acute graft versus-host disease (GvHD), or active chronic GvHD Grade 2 - 4 * Any systemic therapy against GvHD within two weeks prior to start of blinatumomab treatment * Cancer chemotherapy within two weeks prior to start of blinatumomab treatment * Radiotherapy within two weeks prior to start of blinatumomab treatment * Immunotherapy (e.g., rituximab) within four weeks prior to start of blinatumomab treat-ment * Any investigational anti-leukemic product within four weeks prior to start of blinatumomab treatment * Treatment with any other investigational medicinal product (IMP) after signature of informed consent * Eligibility for allogeneic HSCT at the time of enrollment * Known hypersensitivity to immunoglobulins or to any other component of the IMP formulation * Abnormal laboratory values indicative of inadequate renal or liver function * History of malignancy requiring treatment other than ALL within five years prior to start of blinatumomab treatment with the exception of basal cell or squamous cell carcinoma of the skin, or carcinoma "in situ" of the cervix * Any concurrent disease or medical condition that is deemed to interfere with the conduct of the study * Infection with human immunodeficiency virus (HIV) or chronic infection with hepatitis B virus or hepatitis C virus * Pregnant or nursing women * Women of childbearing potential not willing to use an effective form of contraception. Male patients not willing to ensure not to beget a child * Previous treatment with blinatumomab **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Duarte **Country:** United States **Facility:** City of Hope **State:** California **Zip:** 91010-3000 **Location 2:** **City:** Los Angeles **Country:** United States **Facility:** University of California Los Angeles **State:** California **Zip:** 90095-1678 **Location 3:** **City:** Atlanta **Country:** United States **Facility:** Winship Cancer Institute of Emory University **State:** Georgia **Zip:** 30322 **Location 4:** **City:** Chicago **Country:** United States **Facility:** Rush University Medical Center **State:** Illinois **Zip:** 60612 **Location 5:** **City:** Chicago **Country:** United States **Facility:** University of Chicago **State:** Illinois **Zip:** 60637 **Location 6:** **City:** Boston **Country:** United States **Facility:** Dana Farber Institute **State:** Massachusetts **Zip:** 02215 **Location 7:** **City:** Detroit **Country:** United States **Facility:** Barbara Ann Karmanos Cancer Institute **State:** Michigan **Zip:** 48201 **Location 8:** **City:** Rochester **Country:** United States **Facility:** Mayo Clinic **State:** Minnesota **Zip:** 55905 **Location 9:** **City:** Saint Louis **Country:** United States **Facility:** Washington University School of Medicine **State:** Missouri **Zip:** 63110 **Location 10:** **City:** Buffalo **Country:** United States **Facility:** Roswell Park Cancer Streets **State:** New York **Zip:** 14263 **Location 11:** **City:** Philadelphia **Country:** United States **Facility:** University of Pennsylvania **State:** Pennsylvania **Zip:** 19104 **Location 12:** **City:** Houston **Country:** United States **Facility:** University of Texas MD Anderson Cancer Center **State:** Texas **Zip:** 77030 **Location 13:** **City:** Angers **Country:** France **Facility:** CHU d'Angers **Zip:** 49933 **Location 14:** **City:** Nantes **Country:** France **Facility:** Hôpital de l'hôtel Dieu **Zip:** 44000 **Location 15:** **City:** Paris **Country:** France **Facility:** Hôpital Saint Louis **Zip:** 75475 **Location 16:** **City:** Toulouse **Country:** France **Facility:** CHU de Purpan **Zip:** 31059 **Location 17:** **City:** Berlin **Country:** Germany **Facility:** Charité - Campus Benjamin Franklin **Zip:** 12200 **Location 18:** **City:** Frankfurt **Country:** Germany **Facility:** Klinikum der Goethe Universität, Medizinische Klinik II **Zip:** 60590 **Location 19:** **City:** Freiburg **Country:** Germany **Facility:** Universitätsklinikum Freiburg **Zip:** 79106 **Location 20:** **City:** Hannover **Country:** Germany **Facility:** Medizinische Hochschule Hannover **Zip:** 30625 **Location 21:** **City:** Kiel **Country:** Germany **Facility:** Universitätsklinikum Schleswig-Holstein **Zip:** 24116 **Location 22:** **City:** Münster **Country:** Germany **Facility:** Universitätsklinikum Münster **Zip:** 48149 **Location 23:** **City:** Tübingen **Country:** Germany **Facility:** Universitätsklinikum Tübingen **Zip:** 72076 **Location 24:** **City:** Ulm **Country:** Germany **Facility:** Universitätsklinikum Ulm **Zip:** 89081 **Location 25:** **City:** Würzburg **Country:** Germany **Facility:** Julius-Maximilians-Universität, Medizinische Klinik und Poliklinik II **Zip:** 97080 **Location 26:** **City:** Bergamo **Country:** Italy **Facility:** Ospedali Riuniti di Bergamo **Zip:** 24128 **Location 27:** **City:** Naples **Country:** Italy **Facility:** Azienda Ospedaliera Antonio Cardarelli **Zip:** 80131 **Location 28:** **City:** Palermo **Country:** Italy **Facility:** Ospedali Riuniti "Villa Sofia-Cervello" **Zip:** 90146 **Location 29:** **City:** Rome **Country:** Italy **Facility:** Università La Sapienza di Roma **Zip:** 00161 **Location 30:** **City:** Turin **Country:** Italy **Facility:** Azienda Ospedaliero-Universitaria **Zip:** 10126 **Location 31:** **City:** Verona **Country:** Italy **Facility:** Azienda Ospedaliera di Verona **Zip:** 37134 **Location 32:** **City:** Badalona **Country:** Spain **Facility:** ICO Hospital Germans Trias I Pujol **Zip:** 08916 **Location 33:** **City:** Barcelona **Country:** Spain **Facility:** Hospital Clínic Servei d´Hematologia **Zip:** 08036 **Location 34:** **City:** Madrid **Country:** Spain **Facility:** Hospital 12 de Octubre **Zip:** 28041 **Location 35:** **City:** Salamanca **Country:** Spain **Facility:** Hospital universitario de Salamanca **Zip:** 37007 **Location 36:** **City:** Sevilla **Country:** Spain **Facility:** Hospital Universitario Virgen Del Rocio **Zip:** 41013 **Location 37:** **City:** Bristol **Country:** United Kingdom **Facility:** University Hospitals Bristol NHS **Zip:** BS2 8ED **Location 38:** **City:** London **Country:** United Kingdom **Facility:** Royal Free Hampstead NHS Trust **Zip:** NW3 2QG **Location 39:** **City:** Manchester **Country:** United Kingdom **Facility:** The Christie NHS Foundation Trust **Zip:** M20 4BX #### Overall Officials **Official 1:** **Affiliation:** Klinikum der Goethe Universität Frankfurt **Name:** Nicola Gökbuget, MD **Role:** PRINCIPAL_INVESTIGATOR **Official 2:** **Affiliation:** Julius-Maximilians-Universität, Medizinische Klinik und Poliklinik II, Würzburg **Name:** Max Topp, MD **Role:** PRINCIPAL_INVESTIGATOR **Official 3:** **Affiliation:** MD Anderson Cancer Center, Houston, Texas **Name:** Hagop Kantarjian, MD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Shi Z, Zhu Y, Zhang J, Chen B. Monoclonal antibodies: new chance in the management of B-cell acute lymphoblastic leukemia. Hematology. 2022 Dec;27(1):642-652. doi: 10.1080/16078454.2022.2074704. **PMID:** 35622074 **Citation:** Gokbuget N, Kantarjian HM, Bruggemann M, Stein AS, Bargou RC, Dombret H, Fielding AK, Heffner L, Rigal-Huguet F, Litzow M, O'Brien S, Zugmaier G, Gao S, Nagorsen D, Forman SJ, Topp MS. Molecular response with blinatumomab in relapsed/refractory B-cell precursor acute lymphoblastic leukemia. Blood Adv. 2019 Oct 22;3(20):3033-3037. doi: 10.1182/bloodadvances.2019000457. **PMID:** 31648325 **Citation:** Zhu M, Kratzer A, Johnson J, Holland C, Brandl C, Singh I, Wolf A, Doshi S. Blinatumomab Pharmacodynamics and Exposure-Response Relationships in Relapsed/Refractory Acute Lymphoblastic Leukemia. J Clin Pharmacol. 2018 Feb;58(2):168-179. doi: 10.1002/jcph.1006. Epub 2017 Sep 18. **PMID:** 28922466 **Citation:** Barlev A, Lin VW, Katz A, Hu K, Cong Z, Barber B. Estimating Long-Term Survival of Adults with Philadelphia Chromosome-Negative Relapsed/Refractory B-Precursor Acute Lymphoblastic Leukemia Treated with Blinatumomab Using Historical Data. Adv Ther. 2017 Jan;34(1):148-155. doi: 10.1007/s12325-016-0447-x. Epub 2016 Nov 21. **PMID:** 27873237 **Citation:** Zhu M, Wu B, Brandl C, Johnson J, Wolf A, Chow A, Doshi S. Blinatumomab, a Bispecific T-cell Engager (BiTE((R))) for CD-19 Targeted Cancer Immunotherapy: Clinical Pharmacology and Its Implications. Clin Pharmacokinet. 2016 Oct;55(10):1271-1288. doi: 10.1007/s40262-016-0405-4. **PMID:** 27209293 **Citation:** Topp MS, Gokbuget N, Stein AS, Zugmaier G, O'Brien S, Bargou RC, Dombret H, Fielding AK, Heffner L, Larson RA, Neumann S, Foa R, Litzow M, Ribera JM, Rambaldi A, Schiller G, Bruggemann M, Horst HA, Holland C, Jia C, Maniar T, Huber B, Nagorsen D, Forman SJ, Kantarjian HM. Safety and activity of blinatumomab for adult patients with relapsed or refractory B-precursor acute lymphoblastic leukaemia: a multicentre, single-arm, phase 2 study. Lancet Oncol. 2015 Jan;16(1):57-66. doi: 10.1016/S1470-2045(14)71170-2. Epub 2014 Dec 16. Erratum In: Lancet Oncol. 2015 Apr;16(4):e158. **PMID:** 25524800 ## 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: M11225 - Name: Lymphoproliferative Disorders - Relevance: LOW - As Found: Unknown - ID: M10206 - Name: Immunoproliferative Disorders - Relevance: LOW - As Found: Unknown - ID: M11203 - Name: Lymphatic Diseases - 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: 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: T3543 - Name: Lymphosarcoma - Relevance: LOW - As Found: Unknown - ID: T170 - Name: Acute Graft Versus Host Disease - 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 ### Intervention Browse Module - Ancestors - ID: D000000970 - Term: Antineoplastic Agents ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: ANeo - Name: Antineoplastic Agents ### Intervention Browse Module - Browse Leaves - ID: M4225 - Name: Antibodies - Relevance: LOW - As Found: Unknown - ID: M20194 - Name: Antibodies, Bispecific - Relevance: LOW - As Found: Unknown - ID: M10184 - Name: Immunoglobulins - Relevance: LOW - As Found: Unknown - ID: M287547 - Name: Blinatumomab - Relevance: HIGH - As Found: Inguinal Hernia ### Intervention Browse Module - Meshes - ID: C000510808 - Term: Blinatumomab ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold. #### Event Groups **Group ID:** EG000 **Title:** Blinatumomab **Description:** Participants received blinatumomab by continuous intravenous (CIV) infusion over 4 weeks followed by a treatment-free interval of 2 weeks for up to 5 consecutive cycles. The the initial dose was 9 μg/day for the first seven days of treatment, escalated to 28 μg/day starting from Week 2 of treatment. **ID:** EG000 **Other Num Affected:** 186 **Other Num at Risk:** 189 **Serious Number Affected:** 121 **Serious Number At Risk:** 189 **Title:** Blinatumomab **Frequency Threshold:** 5 #### Other Events **Term:** Anaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Febrile neutropenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Leukopenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Neutropenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Thrombocytopenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Sinus tachycardia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Tachycardia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Vision blurred **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Abdominal distension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Abdominal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Abdominal pain upper **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Constipation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Diarrhoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Nausea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Vomiting **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Asthenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Chest pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Chills **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Fatigue **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Oedema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Oedema peripheral **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Pyrexia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Cytokine release syndrome **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Immune system disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Upper respiratory tract infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 **Term:** Alanine aminotransferase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 16.1 **Term:** Aspartate aminotransferase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 16.1 **Term:** Blood bilirubin increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 16.1 **Term:** Immunoglobulins decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 16.1 **Term:** Weight increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 16.1 **Term:** Decreased appetite **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Hyperglycaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Hypokalaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Hypomagnesaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Hypophosphataemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Arthralgia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Back pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Bone pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Muscle spasms **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Muscular weakness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Myalgia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Pain in extremity **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Dizziness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Tremor **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Anxiety **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Insomnia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Cough **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Dyspnoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Epistaxis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Petechiae **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Rash **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Hypertension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 16.1 **Term:** Hypotension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 16.1 #### Serious Events **Term:** Anaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Disseminated intravascular coagulation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Febrile neutropenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 16 **Num At Risk:** 189 **Term:** Leukocytosis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Lymphopenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Neutropenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 7 **Num At Risk:** 189 **Term:** Pancytopenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Atrial fibrillation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Bradycardia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Cardiac failure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Cardiac failure congestive **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Sinus bradycardia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Supraventricular tachycardia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Ventricular fibrillation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Aplasia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Congenital, familial and genetic disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Diplopia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Abdominal pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Colitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Diarrhoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Enteritis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Gastrointestinal haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Pancreatitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Asthenia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Chest pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Device occlusion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Disease progression **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 189 **Term:** Fatigue **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Hypothermia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Medical device complication **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 189 **Term:** Mucosal inflammation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Multi-organ failure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Oedema peripheral **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Pyrexia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 11 **Num At Risk:** 189 **Term:** Cytokine release syndrome **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Immune system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Hypersensitivity **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Immune system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Abdominal infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Aspergillus infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** BK virus infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Bacterial sepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Bronchopneumonia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Bronchopulmonary aspergillosis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Candida infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Cellulitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Cholecystitis infective **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Device related infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 7 **Num At Risk:** 189 **Term:** Enterobacter infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Enterococcal bacteraemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Enterococcal infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Enterococcal sepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Epididymal infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Erysipelas **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Escherichia sepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Fungal infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Fusarium infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Gastrointestinal infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Herpes zoster **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 4 **Num At Risk:** 189 **Term:** Kidney infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Klebsiella infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Leuconostoc infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Lower respiratory tract infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Lung infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Neutropenic sepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Pathogen resistance **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Pilonidal cyst **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Pneumonia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 9 **Num At Risk:** 189 **Term:** Pneumonia fungal **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Pneumonia klebsiella **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Pneumonia respiratory syncytial viral **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Pseudomonal bacteraemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Pseudomonas infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Pulmonary sepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Rhinitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Rhinovirus infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Sepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 9 **Num At Risk:** 189 **Term:** Septic shock **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 189 **Term:** Sinusitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Sinusitis fungal **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Staphylococcal bacteraemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 4 **Num At Risk:** 189 **Term:** Staphylococcal infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Staphylococcal sepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Streptococcal sepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Urinary tract infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Urosepsis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Viral haemorrhagic cystitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Viral infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Accidental overdose **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Femoral neck fracture **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Hip fracture **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Overdose **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 5 **Num At Risk:** 189 **Term:** Post lumbar puncture syndrome **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Subdural haematoma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Toxicity to various agents **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Alanine aminotransferase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Aspartate aminotransferase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Blood alkaline phosphatase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Blood bilirubin increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 189 **Term:** Blood creatinine increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Blood lactate dehydrogenase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** C-reactive protein increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Neutrophil count decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Platelet count decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** White blood cell count decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Fluid overload **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Hyperglycaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Hypokalaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Tumour lysis syndrome **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Arthralgia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Arthritis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Back pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Bone pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Fistula **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Muscular weakness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Myopathy **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Pain in extremity **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Acute leukaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Acute lymphocytic leukaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** B precursor type acute leukaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Chloroma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Lymphoma **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Aphasia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Ataxia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 189 **Term:** Cerebral haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Cognitive disorder **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 189 **Term:** Convulsion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Dizziness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Dysaesthesia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Dysarthria **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Encephalopathy **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 5 **Num At Risk:** 189 **Term:** Headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 4 **Num At Risk:** 189 **Term:** Hemiparesis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Nervous system disorder **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Neurological symptom **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Neurotoxicity **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 189 **Term:** Paraesthesia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Poor quality sleep **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Tremor **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 5 **Num At Risk:** 189 **Term:** Trigeminal nerve disorder **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Agitation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Confusional state **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 5 **Num At Risk:** 189 **Term:** Delirium febrile **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Mental status changes **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Restlessness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Bladder perforation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Renal failure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Renal failure acute **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Scrotal oedema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Reproductive system and breast disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Respiratory distress **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Respiratory failure **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 189 **Term:** Erythema multiforme **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Hyperhidrosis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Rash vesicular **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Skin lesion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Catheter placement **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Surgical and medical procedures **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Central venous catheter removal **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Surgical and medical procedures **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Resuscitation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Surgical and medical procedures **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Capillary leak syndrome **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Embolism **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Femoral artery occlusion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Hypotension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 189 **Term:** Hypovolaemic shock **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Term:** Subclavian vein thrombosis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 16.1 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 189 **Time Frame:** From the start of the first infusion to 30 days after the end of the last infusion in the core study or from the start of the first retreatment cycle infusion to 30 days after the end of the last retreatment cycle, median treatment duration was 42.2 days. ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 189 **Units:** Participants ### Group **ID:** BG000 **Title:** Blinatumomab **Description:** Participants received blinatumomab by continuous intravenous (CIV) infusion over 4 weeks followed by a treatment-free interval of 2 weeks for up to 5 consecutive cycles. The initial dose was 9 μg/day for the first seven days of treatment, escalated to 28 μg/day starting from Week 2 of treatment. ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 18 **Upper Limit:** 79 **Value:** 39.0 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 90 **Class Title:** 18 to < 35 years #### Measurement **Group ID:** BG000 **Value:** 46 **Class Title:** 35 to < 55 years #### Measurement **Group ID:** BG000 **Value:** 28 **Class Title:** 55 to < 65 years #### Measurement **Group ID:** BG000 **Value:** 25 **Class Title:** ≥ 65 years ### Measure #### Measurement **Group ID:** BG000 **Value:** 70 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 119 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 145 **Class Title:** White #### Measurement **Group ID:** BG000 **Value:** 6 **Class Title:** Asian #### Measurement **Group ID:** BG000 **Value:** 7 **Class Title:** Black or African American #### Measurement **Group ID:** BG000 **Value:** 1 **Class Title:** American Indian or Alaska native #### Measurement **Group ID:** BG000 **Value:** 1 **Class Title:** Native Hawaiian or other Pacific Islander #### Measurement **Group ID:** BG000 **Value:** 9 **Class Title:** Other #### Measurement **Group ID:** BG000 **Value:** 20 **Class Title:** Not recorded ### Measure #### Measurement **Group ID:** BG000 **Value:** 16 **Class Title:** Primary refractory #### Measurement **Group ID:** BG000 **Value:** 39 **Class Title:** Relapse ≤ 12 months of allogeneic HSCT #### Measurement **Group ID:** BG000 **Value:** 23 **Class Title:** Entering first salvage; first remission ≤ 12 mo #### Measurement **Group ID:** BG000 **Value:** 108 **Class Title:** Entering second or greater salvage therapies #### Measurement **Group ID:** BG000 **Value:** 3 **Class Title:** No criteria met ### Measure #### Measurement **Group ID:** BG000 **Value:** 16 **Class Title:** 0 #### Measurement **Group ID:** BG000 **Value:** 107 **Class Title:** 1 #### Measurement **Group ID:** BG000 **Value:** 46 **Class Title:** 2 #### Measurement **Group ID:** BG000 **Value:** 20 **Class Title:** >2 ### Measure #### Measurement **Group ID:** BG000 **Value:** 64 **Class Title:** Prior allogeneic HSCT #### Measurement **Group ID:** BG000 **Value:** 16 **Class Title:** No prior alloHSCT, no prior relapse #### Measurement **Group ID:** BG000 **Value:** 84 **Class Title:** No prior alloHSCT, 1 prior relapse #### Measurement **Group ID:** BG000 **Value:** 22 **Class Title:** No prior alloHSCT, 2 prior relapses #### Measurement **Group ID:** BG000 **Value:** 3 **Class Title:** No prior alloHSCT, > 2 prior relapses ### Measure #### Measurement **Group ID:** BG000 **Value:** 38 **Class Title:** No prior salvage therapy #### Measurement **Group ID:** BG000 **Value:** 77 **Class Title:** 1 prior salvage therapy #### Measurement **Group ID:** BG000 **Value:** 42 **Class Title:** 2 prior salvage therapies #### Measurement **Group ID:** BG000 **Value:** 32 **Class Title:** > 2 prior salvage therapies ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 1.9 **Upper Limit:** 249.0 **Value:** 16.59 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 0.1 **Upper Limit:** 56.8 **Value:** 1.38 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 1 **Class Title:** < 10% #### Measurement **Group ID:** BG000 **Value:** 43 **Class Title:** 10% - < 50% #### Measurement **Group ID:** BG000 **Value:** 145 **Class Title:** ≥ 50% **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** FULL_RANGE **Parameter Type:** MEDIAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 2 **Parameter Type:** NUMBER **Title:** Age, Customized **Unit of Measure:** participants ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 4 **Description:** Race was not permitted to be collected in France **Parameter Type:** NUMBER **Title:** Race/Ethnicity, Customized **Unit of Measure:** participants ### Measure 5 **Description:** HSCT = hematopoietic stem cell transplantation **Parameter Type:** NUMBER **Title:** Disease stage entry criteria met **Unit of Measure:** participants ### Measure 6 **Parameter Type:** NUMBER **Title:** Number of prior relapses **Unit of Measure:** participants ### Measure 7 **Description:** alloHSCT = allogeneic hematopoietic stem cell transplantation **Parameter Type:** NUMBER **Title:** Prior allogeneic HSCT and prior relapses **Unit of Measure:** participants ### Measure 8 **Parameter Type:** NUMBER **Title:** Number of prior salvage therapies **Unit of Measure:** participants ### Measure 9 **Dispersion Type:** FULL_RANGE **Parameter Type:** MEDIAN **Title:** Time since initial diagnosis **Unit of Measure:** months ### Measure 10 **Description:** Reported for 173 participants with a prior relapse (the other16 participants were primary refractory with no prior relapses). **Dispersion Type:** FULL_RANGE **Parameter Type:** MEDIAN **Title:** Time since last relapse **Unit of Measure:** months ### Measure 11 **Description:** Bone marrow blasts were assessed by local and central laboratories; reported data are based on maximum central and local laboratory assessments. **Parameter Type:** NUMBER **Title:** Baseline bone marrow blast category **Unit of Measure:** participants **Population Description:** Primary Analysis Set ## 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 **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 ### Outcome Measure 8 ### Outcome Measure 9 ### Outcome Measure 10 ### Outcome Measure 11 ### Outcome Measure 12 ### Outcome Measure 13 ### Outcome Measure 14 ### Outcome Measure 15 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 35.7 - **Spread:** - **Upper Limit:** 50.2 - **Value:** 42.9 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** Not estimable due to the number of events during the observation period. - **Group ID:** OG000 - **Lower Limit:** 5.1 - **Spread:** - **Upper Limit:** NA - **Value:** 6.7 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 28.8 - **Spread:** - **Upper Limit:** 51.0 - **Value:** 39.5 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 26.7 - **Spread:** - **Upper Limit:** 40.5 - **Value:** 33.3 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 5.7 - **Spread:** - **Upper Limit:** 14.6 - **Value:** 9.5 **Title:** #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.9 - **Spread:** - **Upper Limit:** 6.1 - **Value:** 2.6 **Title:** #### Outcome Measure 7 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 4.8 - **Spread:** - **Upper Limit:** 8.3 - **Value:** 5.9 **Title:** #### Outcome Measure 8 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.0 - **Spread:** - **Upper Limit:** 1.0 - **Value:** 0.0 **Title:** #### Outcome Measure 9 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 4.2 - **Spread:** - **Upper Limit:** 7.5 - **Value:** 6.1 **Title:** #### Outcome Measure 10 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 188 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 155 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 166 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 105 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 121 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 105 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 69 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 63 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 34 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 18 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 28 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 **Title:** #### Outcome Measure 11 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.0 - **Spread:** - **Upper Limit:** 23.4 - **Value:** 11.3 **Title:** #### Outcome Measure 12 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 258 - **Upper Limit:** - **Value:** 211 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 502 - **Upper Limit:** - **Value:** 621 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** 444 - **Upper Limit:** - **Value:** 731 **Title:** #### Outcome Measure 13 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 409 - **Upper Limit:** - **Value:** 93.1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 83.1 - **Upper Limit:** - **Value:** 27.4 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 45.8 - **Upper Limit:** - **Value:** 22.8 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 27.6 - **Upper Limit:** - **Value:** 21.6 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 822 - **Upper Limit:** - **Value:** 589 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 136 - **Upper Limit:** - **Value:** 95.7 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 633 - **Upper Limit:** - **Value:** 397 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 941 - **Upper Limit:** - **Value:** 428 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 44.6 - **Upper Limit:** - **Value:** 24.7 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 5.21 - **Upper Limit:** - **Value:** 10.8 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 5.17 - **Upper Limit:** - **Value:** 11 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 2.03 - **Upper Limit:** - **Value:** 10.3 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 2390 - **Upper Limit:** - **Value:** 826 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 681 - **Upper Limit:** - **Value:** 234 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 952 - **Upper Limit:** - **Value:** 315 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 114 - **Upper Limit:** - **Value:** 69.2 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 125 - **Upper Limit:** - **Value:** 30 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 3.33 - **Upper Limit:** - **Value:** 10.3 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 15 - **Upper Limit:** - **Value:** 12.1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 7.79 - **Upper Limit:** - **Value:** 12 **Title:** #### Outcome Measure 14 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 5.3 - **Spread:** - **Upper Limit:** 14.0 - **Value:** 9.0 **Title:** #### Outcome Measure 15 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 36.2 - **Spread:** - **Upper Limit:** 50.8 - **Value:** 43.4 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 28.6 - **Spread:** - **Upper Limit:** 42.7 - **Value:** 35.4 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 4.5 - **Spread:** - **Upper Limit:** 12.8 - **Value:** 7.9 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 5.3 - **Spread:** - **Upper Limit:** 14.0 - **Value:** 9.0 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 0.9 - **Spread:** - **Upper Limit:** 6.1 - **Value:** 2.6 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Hematological assessments were performed from bone marrow biopsy samples. All hematological assessments of bone marrow were reviewed in a central reference laboratory. Hematological remissions were defined by the following criteria: Complete Remission (CR): * bone marrow blasts ≤ 5% * no evidence of disease * full recovery of peripheral blood counts: * platelets \> 100,000/μL, and * absolute neutrophil count (ANC) \> 1,000/μL Complete Remission With Partial Hematological Recovery (CRh\*): * bone marrow blasts ≤ 5% * no evidence of disease * partial recovery of peripheral blood counts: * platelets \> 50,000/μL, and * ANC \> 500/μL. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** The Primary Analysis Set (PAS), defined as participants from the first 3 stages of the study who received any infusion of blinatumomab. **Reporting Status:** POSTED **Time Frame:** Within the first 2 cycles of treatment, 12 weeks **Title:** Percentage of Participants With a Best Response of Complete Remission or Complete Remission With Only Partial Hematological Recovery Within 2 Cycles of Treatment **Type:** PRIMARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants received blinatumomab by continuous intravenous (CIV) infusion over 4 weeks followed by a treatment-free interval of 2 weeks for up to 5 consecutive cycles. The initial dose was 9 μg/day for the first seven days of treatment, escalated to 28 μg/day starting from Week 2 of treatment. **ID:** OG000 **Title:** Blinatumomab #### Outcome Measure 2 **Description:** Time to hematological relapse was measured for participants in remission during the core study (the time from the first infusion through 30 days after the last infusion), from the time the participant first achieved remission until first documented relapse or death due to disease progression. Participants without documented relapse (hematological or extramedullary) and who did not die were censored at the time of their last bone marrow assessment or their last survival follow-up visit confirming remission. Participants who died without having reported hematological relapse or without showing any clinical sign of disease progression were censored on their date of death. Hematological relapse is defined as: * proportion of blasts in bone marrow \> 5% after documented CR/CRh\* or * blasts in peripheral blood after documented CR/CRh\*. Time to hematological relapse was analyzed by Kaplan-Meier methods and the median observation time was calculated by the reverse Kaplan Meier method. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEDIAN **Population Description:** Participants who reached complete remission or complete remission with partial hematological recovery during the core study. **Reporting Status:** POSTED **Time Frame:** Up to the data cut-off date of 10 October 2013; median observation time was 8.0 months. **Title:** Time to Hematological Relapse (Duration of Response) **Type:** SECONDARY **Unit of Measure:** months ##### Group **Description:** Participants received blinatumomab by continuous intravenous (CIV) infusion over 4 weeks followed by a treatment-free interval of 2 weeks for up to 5 consecutive cycles. The initial dose was 9 μg/day for the first seven days of treatment, escalated to 28 μg/day starting from Week 2 of treatment. **ID:** OG000 **Title:** Blinatumomab #### Outcome Measure 3 **Description:** Participants who were eligible for allogeneic HSCT were those who achieved remission (complete response or complete response with partial recovery of peripheral blood counts) after 2 cycles of blinatumomab treatment, and no further anti-leukemic medication was given before HSCT. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** Participants who reached complete remission or complete remission with partial hematological recovery during the first 2 cycles of treatment. **Reporting Status:** POSTED **Time Frame:** Up to the data cut-off date of 10 October 2013. Maximum duration on study was 17.8 months. **Title:** Percentage of Participants Who Received an Allogeneic Hematopoietic Stem Cell Transplant (HSCT) During Blinatumomab Induced Remission **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants received blinatumomab by continuous intravenous (CIV) infusion over 4 weeks followed by a treatment-free interval of 2 weeks for up to 5 consecutive cycles. The initial dose was 9 μg/day for the first seven days of treatment, escalated to 28 μg/day starting from Week 2 of treatment. **ID:** OG000 **Title:** Blinatumomab #### Outcome Measure 4 **Description:** Complete Remission was defined by the following criteria: * bone marrow blasts ≤ 5% * no evidence of disease * full recovery of peripheral blood counts: * platelets \> 100,000/μL, and * absolute neutrophil count (ANC) \> 1,000/μL **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** Primary analysis set **Reporting Status:** POSTED **Time Frame:** Within the first 2 cycles of treatment, 12 weeks **Title:** Percentage of Participants With a Best Response of Complete Remission Within 2 Cycles of Treatment **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants received blinatumomab by continuous intravenous (CIV) infusion over 4 weeks followed by a treatment-free interval of 2 weeks for up to 5 consecutive cycles. The initial dose was 9 μg/day for the first seven days of treatment, escalated to 28 μg/day starting from Week 2 of treatment. **ID:** OG000 **Title:** Blinatumomab #### Outcome Measure 5 **Description:** Complete Remission With Partial Hematological Recovery was defined by the following criteria: * bone marrow blasts ≤ 5% * no evidence of disease * partial recovery of peripheral blood counts: * platelets \> 50,000/μL, and * ANC \> 500/μL. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** Primary analysis set **Reporting Status:** POSTED **Time Frame:** Within the first 2 cycles of treatment, 12 weeks **Title:** Percentage of Participants With a Best Response of Complete Remission With Only Partial Hematological Recovery Within 2 Cycles of Treatment **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants received blinatumomab by continuous intravenous (CIV) infusion over 4 weeks followed by a treatment-free interval of 2 weeks for up to 5 consecutive cycles. The initial dose was 9 μg/day for the first seven days of treatment, escalated to 28 μg/day starting from Week 2 of treatment. **ID:** OG000 **Title:** Blinatumomab #### Outcome Measure 6 **Description:** Partial Remission is defined as bone marrow blasts 6% to 25% with at least a 50% reduction from baseline. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** Primary analysis set **Reporting Status:** POSTED **Time Frame:** Within the first 2 cycles of treatment, 12 weeks **Title:** Percentage of Participants With a Best Response of Partial Remission Within 2 Cycles of Treatment **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants received blinatumomab by continuous intravenous (CIV) infusion over 4 weeks followed by a treatment-free interval of 2 weeks for up to 5 consecutive cycles. The initial dose was 9 μg/day for the first seven days of treatment, escalated to 28 μg/day starting from Week 2 of treatment. **ID:** OG000 **Title:** Blinatumomab #### Outcome Measure 7 **Description:** Relapse-free survival was assessed for participants who achieved a complete remission or complete remission with partial hematological recovery during the core study and was measured from the time the participant first achieved remission until first documented relapse or death due to any cause. Participants without a documented relapse (hematological or extramedullary) or who did not die were censored at the time of their last bone marrow assessment or their last survival follow-up visit confirming remission. Relapse free survival was estimated using Kaplan-Meier methods and the median observation time was calculated by the reverse Kaplan Meier method. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEDIAN **Population Description:** Participants who reached complete remission or complete remission with partial hematological recovery during the core study **Reporting Status:** POSTED **Time Frame:** Up to the data cut-off date of 10 October 2013; median observation time was 8.9 months. **Title:** Relapse-free Survival **Type:** SECONDARY **Unit of Measure:** months ##### Group **Description:** Participants received blinatumomab by continuous intravenous (CIV) infusion over 4 weeks followed by a treatment-free interval of 2 weeks for up to 5 consecutive cycles. The initial dose was 9 μg/day for the first seven days of treatment, escalated to 28 μg/day starting from Week 2 of treatment. **ID:** OG000 **Title:** Blinatumomab #### Outcome Measure 8 **Description:** Event-free survival was calculated from the start date of blinatumomab infusion until the date of bone marrow aspiration at which hematological relapse was first detected, or the date of diagnosis on which the hematological or extramedullary relapse was documented or the date of start of any new therapy for ALL (excluding HSCT), or the date of death, whichever was earlier. Participants who did not achieve complete remission or complete remission with partial hematological recovery during the core study were evaluated as having an event on Day 1. Participants in remission who did not experience hematological relapse, did not receive a new therapy for ALL (excluding HSCT), and did not die were censored on the date of the last available bone marrow aspiration or on the last date of survival follow-up visit, whichever was later. Event free survival was estimated using Kaplan-Meier methods and the median observation time was calculated by the reverse Kaplan Meier method. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEDIAN **Population Description:** Primary analysis set **Reporting Status:** POSTED **Time Frame:** Up to the data cut-off date of 10 October 2013; median observation time was 9.8 months. **Title:** Event-free Survival **Type:** SECONDARY **Unit of Measure:** months ##### Group **Description:** Participants received blinatumomab by continuous intravenous (CIV) infusion over 4 weeks followed by a treatment-free interval of 2 weeks for up to 5 consecutive cycles. The initial dose was 9 μg/day for the first seven days of treatment, escalated to 28 μg/day starting from Week 2 of treatment. **ID:** OG000 **Title:** Blinatumomab #### Outcome Measure 9 **Description:** Overall survival was measured for all participants from the time the participant received the first treatment of blinatumomab until death due to any cause or the date of the last follow-up. Participants who did not die were censored on the last documented visit date or the date of the last phone contact when the patient was last known to have been alive. Overall survival was estimated using Kaplan-Meier methods. The median follow-up time with respect to overall survival was calculated by the reverse Kaplan Meier method. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEDIAN **Population Description:** Primary analysis set **Reporting Status:** POSTED **Time Frame:** Up to the data cut-off date of 10 October 2013; median observation time was 9.8 months. **Title:** Overall Survival **Type:** SECONDARY **Unit of Measure:** months ##### Group **Description:** Participants received blinatumomab by continuous intravenous (CIV) infusion over 4 weeks followed by a treatment-free interval of 2 weeks for up to 5 consecutive cycles. The initial dose was 9 μg/day for the first seven days of treatment, escalated to 28 μg/day starting from Week 2 of treatment. **ID:** OG000 **Title:** Blinatumomab #### Outcome Measure 10 **Description:** Adverse events (AEs) were evaluated for severity according to the the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE), version 4, as follows: Grade 1 - Mild AE; Grade 2 - Moderate AE; Grade 3 - Severe AE; Grade 4 - Life-threatening or disabling AE; Grade 5 - Death. The investigator used medical judgment to determine if there was a causal relationship (ie, related, unrelated) between an adverse event and blinatumomab. An AE was considered "serious" if it resulted in death, was life-threatening, requires or prolongs inpatient hospitalization, results in persistent or significant incapacity or substantial disruption to conduct normal life functions, is a congenital anomaly or birth defect or is a medically important condition. Progressive disease was not an adverse event, per the protocol, unless it was more severe than expected for the patient. Therefore, many deaths due to progressive disease were not counted as adverse events. **Parameter Type:** NUMBER **Population Description:** Full analysis set (FAS), defined as all patients who received any infusion of blinatumomab. **Reporting Status:** POSTED **Time Frame:** From the start of the first infusion to 30 days after the end of the last infusion in the core study or from the start of the first retreatment cycle infusion to 30 days after the end of the last retreatment cycle, median treatment duration was 42.2 days. **Title:** Number of Participants With Treatment-emergent Adverse Events **Type:** SECONDARY **Unit of Measure:** participants ##### Group **Description:** Participants received blinatumomab by continuous intravenous (CIV) infusion over 4 weeks followed by a treatment-free interval of 2 weeks for up to 5 consecutive cycles. The initial dose was 9 μg/day for the first seven days of treatment, escalated to 28 μg/day starting from Week 2 of treatment. **ID:** OG000 **Title:** Blinatumomab #### Outcome Measure 11 **Description:** The analysis of 100-day mortality after allogeneic HSCT was assessed for all participants who received an allogeneic HSCT while in remission (CR/CRh\*) following treatment with blinatumomab. 100-day mortality after allogeneic HSCT was calculated relative to the date of allogeneic HSCT. Patients alive were censored on the last documented visit date or the date of the last phone contact when the patient was last known to have been alive. The 100-day mortality rate after allogeneic HSCT was defined as the percentage of patients having died up to 100 days after allogeneic HSCT estimated using the estimated time to death in percent calculated by Kaplan-Meier methods. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** Participants who received an allogeneic HSCT while in remission induced by blinatumomab treatment. **Reporting Status:** POSTED **Time Frame:** From the date of allogeneic HSCT until the data cut-off date of 10 October 2013; median observation time was 7.4 months. **Title:** 100-Day Mortality After Allogeneic Hematopoietic Stem Cell Transplant **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants received blinatumomab by continuous intravenous (CIV) infusion over 4 weeks followed by a treatment-free interval of 2 weeks for up to 5 consecutive cycles. The initial dose was 9 μg/day for the first seven days of treatment, escalated to 28 μg/day starting from Week 2 of treatment. **ID:** OG000 **Title:** Blinatumomab #### Outcome Measure 12 **Description:** The steady state concentration of blinatumomab was summarized as the observed concentrations collected at least 10 hours after the start of the IV infusion or dose step for cycle 1 and cycle 2, respectively. Serum concentrations of blinatumomab were measured using a validated bioassay. The lower limit of quantitation (LLOQ) = 50.0 pg/mL. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Pharmacokinetic Data Set (PKS) defined as all patients who received any infusion of blinatumomab and had at least one PK sample collected unless significant protocol deviations affected the data analysis or if key dosing, dosing interruption or sampling information was missing. **Reporting Status:** POSTED **Time Frame:** Samples were taken before treatment start and on Days 3, 8, 10, 15, 22, and 29 after infusion start during Cycles 1 and 2. **Title:** Serum Blinatumomab Concentration at Steady State **Type:** SECONDARY **Unit of Measure:** pg/mL ##### Group **Description:** Participants receiving 9 μg/day blinatumomab by continuous intravenous (CIV) infusion during Cycle 1. **ID:** OG000 **Title:** Cycle 1: Blinatumomab 9 μg/Day ##### Group **Description:** Participants receiving 28 μg/day blinatumomab by continuous intravenous (CIV) infusion during Cycle 1. **ID:** OG001 **Title:** Cycle 1: Blinatumomab 28 μg/Day ##### Group **Description:** Participants receiving 28 μg/day blinatumomab by continuous intravenous (CIV) infusion during Cycle 2. **ID:** OG002 **Title:** Cycle 2: Blinatumomab 28 μg/Day #### Outcome Measure 13 **Description:** The activation of immune effector cells was monitored by the measurement of peripheral blood cytokine levels including interleukin (IL)-2, IL-4, IL-6, IL-10, tumor necrosis factor (TNF)-α and interferon gamma (IFN)-γ using enzyme-linked immunosorbent assays or cytometric bead assays. The limit of detection of the assay (LOD) was 20 pg/mL and the limit of quantification (LOQ) was 125 pg/mL. Data below LOD were set to 10 pg/mL while data \< LOQ and \> LOD were reported as measured. Serum IL-4 levels were below detection limit (\< 20 pg/mL) at all time points in all participants studied. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Pharmacodynamic Data Set (PDS): All patients who received any infusion of blinatumomab and had at least one pharmacodynamic sample collected. N indicates the number of participants with available data at each time point. **Reporting Status:** POSTED **Time Frame:** Serum samples were collected on Days 1 and 8 at 2 hours and 6 hours after treatment start, and on Day 2 (24 hours) and Day 3 (48 hours) of each treatment cycle and on Days 9 and 10 after dose step. **Title:** Serum Cytokine Peak Levels **Type:** SECONDARY **Unit of Measure:** pg/mL ##### Group **Description:** Participants received blinatumomab by continuous intravenous (CIV) infusion over 4 weeks followed by a treatment-free interval of 2 weeks for up to 5 consecutive cycles. The initial dose was 9 μg/day for the first seven days of treatment, escalated to 28 μg/day starting from Week 2 of treatment. **ID:** OG000 **Title:** Blinatumomab #### Outcome Measure 14 **Description:** Blast Free Hypoplastic or Aplastic Bone Marrow was defined as: * bone marrow blasts ≤ 5% * no evidence of disease * insufficient recovery of peripheral counts: platelets ≤ 50,000/μL and/or absolute neutrophil count (ANC) ≤ 500/μL **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** Primary analysis set **Reporting Status:** POSTED **Time Frame:** Within the first 2 cycles of treatment, 12 weeks **Title:** Percentage of Participants With a Best Response of Blast Free Hypoplastic or Aplastic Bone Marrow Within 2 Cycles of Treatment **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants received blinatumomab by continuous intravenous (CIV) infusion over 4 weeks followed by a treatment-free interval of 2 weeks for up to 5 consecutive cycles. The initial dose was 9 μg/day for the first seven days of treatment, escalated to 28 μg/day starting from Week 2 of treatment. **ID:** OG000 **Title:** Blinatumomab #### Outcome Measure 15 **Description:** Complete Remission (CR): * bone marrow blasts ≤ 5% * no evidence of disease * full recovery of peripheral blood counts: * platelets \> 100,000/μL, and * absolute neutrophil count (ANC) \> 1,000/μL Complete Remission With Partial Hematological Recovery (CRh\*): * bone marrow blasts ≤ 5% * no evidence of disease * partial recovery of peripheral blood counts: * platelets \> 50,000/μL, and * ANC \> 500/μL Blast Free Hypoplastic or Aplastic Bone Marrow: * bone marrow blasts ≤ 5% * no evidence of disease * insufficient recovery of peripheral counts: platelets ≤ 50,000/μL and/or ANC ≤ 500/μL Partial Remission: • bone marrow blasts 6% to 25% with at least a 50% reduction from Baseline. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** Primary analysis set **Reporting Status:** POSTED **Time Frame:** From the first dose of blinatumomab until 30 days after the end of the last infusion during the core study, or until the data cut-off date of 10 October 2013; a maximum of 7.5 months. **Title:** Best Response During the Core Study **Type:** SECONDARY **Unit of Measure:** percentage of participants ##### Group **Description:** Participants received blinatumomab by continuous intravenous (CIV) infusion over 4 weeks followed by a treatment-free interval of 2 weeks for up to 5 consecutive cycles. The initial dose was 9 μg/day for the first seven days of treatment, escalated to 28 μg/day starting from Week 2 of treatment. **ID:** OG000 **Title:** Blinatumomab ### Participant Flow Module #### Group **Description:** Participants received blinatumomab by continuous intravenous (CIV) infusion over 4 weeks followed by a treatment-free interval of 2 weeks for up to 5 consecutive cycles. The initial dose was 9 μg/day for the first seven days of treatment, escalated to 28 μg/day starting from Week 2 of treatment. **ID:** FG000 **Title:** Blinatumomab #### Period **Title:** Overall Study ##### Withdraw **Type:** Ongoing in core study ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ##### Withdraw **Type:** Physician Decision ###### Reason **Group ID:** FG000 **Number of Subjects:** 46 ##### Withdraw **Type:** Progressive disease ###### Reason **Group ID:** FG000 **Number of Subjects:** 43 ##### Withdraw **Type:** Adverse Event ###### Reason **Group ID:** FG000 **Number of Subjects:** 32 ##### Withdraw **Type:** Disease relapse ###### Reason **Group ID:** FG000 **Number of Subjects:** 23 ##### Withdraw **Type:** Lack of Efficacy ###### Reason **Group ID:** FG000 **Number of Subjects:** 14 ##### Withdraw **Type:** Death ###### Reason **Group ID:** FG000 **Number of Subjects:** 7 ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 7 ##### Withdraw **Type:** Protocol Violation ###### Reason **Group ID:** FG000 **Number of Subjects:** 2 ##### Withdraw **Type:** Other ###### Reason **Group ID:** FG000 **Number of Subjects:** 3 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 189 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 10 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 179 **Pre-Assignment Details:** Two hundred twenty-five participants enrolled in the study overall. Results below include data for 189 participants enrolled in the first 3 stages of the study (the primary analysis set). An additional 36 participants enrolled in the Additional Evaluation Cohort are not reported here as the study is ongoing and data collection has not completed. **Recruitment Details:** This study was open to adult patients with relapsed / refractory B-precursor acute lymphoblastic leukemia (ALL). The study protocol originally used a Simon 2-stage design and was subsequently expanded to include a third stage. Protocol amendment 4 added an additional cohort of participants for central nervous system evaluations. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT05189379 **Acronym:** TEDDS_OCRT **Brief Title:** Targeted Extinction of Drug Cues During Sleep - Olfactory Cue Reactivity Task **Official Title:** Targeted Extinction of Drug Cues During Sleep - Olfactory Cue Reactivity Task #### Organization Study ID Info **ID:** tedds_ocrt_1 #### Organization **Class:** OTHER **Full Name:** Central Institute of Mental Health, Mannheim ### Status Module #### Completion Date **Date:** 2023-05-25 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2024-01-31 **Type:** ACTUAL **Last Update Submit Date:** 2024-01-30 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2023-05-25 **Type:** ACTUAL #### Start Date **Date:** 2022-03-10 **Type:** ACTUAL **Status Verified Date:** 2024-01 #### Study First Post Date **Date:** 2022-01-12 **Type:** ACTUAL **Study First Submit Date:** 2021-12-27 **Study First Submit QC Date:** 2021-12-27 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Central Institute of Mental Health, Mannheim #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** The study aims to utilise olfactory stimulation in addition to the visual stimulation in cue reactivity tasks to enhance precision in measuring alcohol based cues in individuals diagnosed with heavy drinking. The study consists of one cue reactivity task with visual stimuli, another cue reactivity task with matching odour/visual stimuli and lastly a monetary incentive delay task. **Detailed Description:** The intention of the study is to enhance the measurement precision in image-based cue reactivity task by introducing a new level of stimuli in the form of odours. During the course of the functional Magnetic Resonance Imaging (fMRI) screening, participants are required to finish three tasks, namely; odour-based cue reactivity task (OCRT), image-based cue reactivity task(ICRT) and monetary incentive delay task (MID). Participants will be randomly divided into two groups, one group will receive tasks in the following order: OCRT-MID-ICRT and the other group will receive them in the following order: ICRT-MID-OCRT. With this, the habituation effect from receiving odours during OCRT is expected to be balanced. ### Conditions Module **Conditions:** - Heavy Drinking **Keywords:** - Cue Reactivity Task - Heavy Drinking - Olfactory Cue Reactivity Task ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** A two group study is constructed with the sole intention to balance the amount of participants who would undergo the same experiment with a different order. Half of the participants will receive odour based Cue Reactivity Task first, other half will complete the image based Cue Reactivity Task first. Before the MRI screening participants would be asked about their preference between beer, wine and schnapps then during the odour based Cue Reactivity Task only their preferred beverage odour would be delivered. ##### Masking Info **Masking:** NONE **Primary Purpose:** BASIC_SCIENCE #### Enrollment Info **Count:** 46 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants assigned to this arm will undergo the experiment in the following order: Odour based Cue Reactivity Task, Monetary Incentive Delay Task, Image based Cue Reactivity Task **Intervention Names:** - Other: Non-Alcoholic Odour - Other: Alcoholic Odour - Other: Non-Alcoholic Images - Other: Alcoholic Images **Label:** Olfaction First **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants assigned to this arm will undergo the experiment in the following order: Image based Cue Reactivity Task, Monetary Incentive Delay Task, Odour based Cue Reactivity Task **Intervention Names:** - Other: Non-Alcoholic Odour - Other: Alcoholic Odour - Other: Non-Alcoholic Images - Other: Alcoholic Images **Label:** Image First **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Image First - Olfaction First **Description:** A chemical used in fragrances and flavouring with a smell close to a nutty, buttery structure. Used as a 1:100 deionised water dilution. **Name:** Non-Alcoholic Odour **Other Names:** - Coffee furanone - 2-Methyltetrahydrofuran-3-one **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - Image First - Olfaction First **Description:** Alcoholic beverage beer / Alcoholic beverage red wine / Alcoholic beverage brandy / The type of the Alcoholic Odour intervention will be presented in a participant-preference basis. **Name:** Alcoholic Odour **Other Names:** - Beer - Wine - Schnapps **Type:** OTHER #### Intervention 3 **Arm Group Labels:** - Image First - Olfaction First **Description:** Images of non-alcoholic items, namely; broom, duster, clothing iron, lightbulb, mat, mulch, penlight, rack, rock, kitchen scissors, trough, vent are presented in parallel with the non-alcoholic odour **Name:** Non-Alcoholic Images **Type:** OTHER #### Intervention 4 **Arm Group Labels:** - Image First - Olfaction First **Description:** Images of alcoholic items, namely, beer, wine, schnapps are presented in parallel with the alcoholic odour **Name:** Alcoholic Images **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Change in brain activation in the form of BOLD signals within 3 groups of ROIs: * Memory related ROIs: Hippocampus, Dorsolateral Prefrontal Cortex * Cognitive Control related ROIs: Anterior Cingulate Cortex, Ventrolateral Prefrontal Cortex * Reward related ROIs: Striatum (ventral and dorsal) and Insula **Measure:** Change in Blood Oxygen Level Dependent (BOLD) signal within selected Regions of Interest (ROI) **Time Frame:** Once during the fMRI experiment **Description:** A cue reactivity task based on the odours and images of alcoholic and non-alcoholic items. Adapted from Vollstädt-Klein et al., 2011. Subjects are presented with odours and images of alcoholic (beer, wine, schnapps) and non-alcoholic (odour: 2-Methyltetrahydrofuran-3-one / images: broom, duster, clothing iron, lightbulb, mat, mulch, penlight, rack, rock, kitchen scissors, trough, vent) items during an fMRI session in blocks. For non-alcoholic items, in each block 5 images from the same stimuli group are presented with the non-alcoholic odour. For alcoholic items, in each block there are 5 images from the selected stimuli group with the congruent odour. After each block subjects are asked to rate their liking and wanting towards the items that have been presented to them in two 1-5 scales. Blocks are pseudorandomised to maximise balance between groups and minimise the effect of habituation. Minimum 1: "not at all", maximum 5: "very much". **Measure:** Odour based cue reactivity task **Time Frame:** Once during the fMRI screening **Description:** A cue reactivity task based on the images of alcoholic and non-alcoholic items. Adapted from Vollstädt-Klein et al., 2011. Subjects are presented with images of the alcoholic (beer, wine, schnapps) and the non-alcoholic (broom, duster, clothing iron, lightbulb, mat, mulch, penlight, rack, rock, kitchen scissors, trough, vent) items during an fMRI session in blocks. In each block there are 5 images from the same stimuli group. After each block subjects are asked to rate their liking and wanting towards the items that have been presented to them in two 1-5 scales. Blocks are pseudorandomised to maximise balance between groups and minimise the effect of habituation. Minimum 1: "not at all", maximum 5: "very much". **Measure:** Image based cue reactivity task **Time Frame:** Once during the fMRI screening **Description:** A task to invoke reward based activations in the subjects. Adapted from Kirsch et al., 2003. Subjects are presented 3 different stimulus conditions, each linked either to a positive monetary reward (a vertically oriented arrow pointing upward), a positive verbal feedback (a vertically oriented double-sided arrow) or a neutral no reward or feedback (a horizontally oriented double-sided arrow). After presentation of the two positive stimuli types, a flashing screen is presented and during the flashing screen if the subjects can press the button fast enough they will either win 1€ or they will be shown a positive verbal feedback depending on the positive stimuli type. The negative stimuli is followed by a 3 second black screen to include a control condition. Each stimulus condition is presented 10 times in a pseudorandom schedule with no more than two equal conditions in succession. The inter-trial interval randomly varies between 6-9 seconds. **Measure:** Monetary incentive delay task **Time Frame:** Once during the fMRI screening #### Secondary Outcomes **Description:** Task measuring the objective vigilance of subjects. To distinguish vigilance between the wake and the sleep conditions, following measures will be compared: Median reaction speed (1/reaction time) and percentage of lapses (number of lapses divided by the number of all valid stimuli, excluding false starts. Lapse \>= 500 ms, false start \< 100ms). The task is prepared in JsPsych, and administered with using a tablet connected to internet. Participant data is securely stored in an encrypted server. **Measure:** Psychomotor Vigilance Test (Roach et al. 2006) **Time Frame:** Once before the fMRI screening **Description:** Test measuring current level of alertness of subjects on a seven point scale. A high test score indicates a high level of sleepiness and conversely a low test score yields a low level of sleepiness. Test scores will be utilized as a pre-measurement alertness indicator for participants. The task is prepared in JsPsych, and administered with using a tablet connected to internet. Participant data is securely stored in an encrypted server. **Measure:** Stanford Sleepiness Scale (Hoddes et al. 1973) **Time Frame:** Once before the fMRI screening **Description:** Questionnaire on self-assessment of current craving of subjects. Consists of 8 items on a Likert-type scale from 1-7 where higher scores represent stronger alcohol urges. The task is prepared in JsPsych, and administered with using a tablet connected to internet. Participant data is securely stored in an encrypted server. **Measure:** Alcohol Urge Questionnaire (Bohn et al. 1995) **Time Frame:** Once before the fMRI screening **Description:** A psychophysical test that is developed for assessing individuals reactions to the olfactory stimuli (Hummel et al., 1997). The 12-stick test from Burghardt is chosen for the study and it consists of identifying one of the four items that are presented with each of the twelve odourant sticks. At the end, with the aid of the identification score, the participant's olfaction capability is assessed. **Measure:** Sniffin' Sticks Test (Burghardt®, Wedel, Germany)(Hummel et al., 1997) **Time Frame:** Once before the fMRI screening ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * An Heavy Drinking diagnosis, confirmed by Alcohol Use Disorders Identification Test * Ability to provide fully informed consent and to use self-rating scales in fMRI * Understanding of the German language Exclusion Criteria: * Severe mental or physical illnesses * Insomnia * Any metal parts and pieces in the body * Claustrophobia; fear of confined spaces **Maximum Age:** 65 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Mannheim **Country:** Germany **Facility:** Zentralinstitut für Seelische Gesundheit Mannheim **State:** Baden-Württemberg **Zip:** 68159 #### Overall Officials **Official 1:** **Affiliation:** ZI Mannheim, Department of Clinical Psychology, RG Psychology and Neurobiology of Sleep and Memory **Name:** Gordon B Feld, Dr **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Description:** Due to data protection rules, individual data cannot be shared. Cumulated data on the group level can be made available for meta-analyses on request. **IPD Sharing:** NO ### References Module #### References **Citation:** Vollstadt-Klein S, Loeber S, Kirsch M, Bach P, Richter A, Buhler M, von der Goltz C, Hermann D, Mann K, Kiefer F. Effects of cue-exposure treatment on neural cue reactivity in alcohol dependence: a randomized trial. Biol Psychiatry. 2011 Jun 1;69(11):1060-6. doi: 10.1016/j.biopsych.2010.12.016. Epub 2011 Feb 3. **PMID:** 21292243 **Citation:** Roach GD, Dawson D, Lamond N. Can a shorter psychomotor vigilance task be used as a reasonable substitute for the ten-minute psychomotor vigilance task? Chronobiol Int. 2006;23(6):1379-87. doi: 10.1080/07420520601067931. **PMID:** 17190720 **Citation:** Hoddes E, Zarcone V, Smythe H, Phillips R, Dement WC. Quantification of sleepiness: a new approach. Psychophysiology. 1973 Jul;10(4):431-6. doi: 10.1111/j.1469-8986.1973.tb00801.x. No abstract available. **PMID:** 4719486 **Citation:** Bohn MJ, Krahn DD, Staehler BA. Development and initial validation of a measure of drinking urges in abstinent alcoholics. Alcohol Clin Exp Res. 1995 Jun;19(3):600-6. doi: 10.1111/j.1530-0277.1995.tb01554.x. **PMID:** 7573780 **Citation:** Hummel T, Sekinger B, Wolf SR, Pauli E, Kobal G. 'Sniffin' sticks': olfactory performance assessed by the combined testing of odor identification, odor discrimination and olfactory threshold. Chem Senses. 1997 Feb;22(1):39-52. doi: 10.1093/chemse/22.1.39. **PMID:** 9056084 **Citation:** Kirsch P, Schienle A, Stark R, Sammer G, Blecker C, Walter B, Ott U, Burkart J, Vaitl D. Anticipation of reward in a nonaversive differential conditioning paradigm and the brain reward system: an event-related fMRI study. Neuroimage. 2003 Oct;20(2):1086-95. doi: 10.1016/S1053-8119(03)00381-1. **PMID:** 14568478 ## Derived Section ### Intervention Browse Module - Browse Branches - Abbrev: Micro - Name: Micronutrients - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M10533 - Name: Iron - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00738179 **Acronym:** SAVE **Brief Title:** Continuous Positive Airway Pressure Treatment of Obstructive Sleep Apnea to Prevent Cardiovascular Disease **Official Title:** Sleep Apnea cardioVascular Endpoints Study - Investigating the Effectiveness of Treatment With CPAP vs Standard Care in Reducing CV Morbidity and Mortality in Patients With Co-existing CV Disease and Moderate-severe Obstructive Sleep Apnea. #### Organization Study ID Info **ID:** SAVE001 #### Organization **Class:** OTHER **Full Name:** Adelaide Institute for Sleep Health #### Secondary ID Infos **ID:** ANZCTR 12608000409370 ### Status Module #### Completion Date **Date:** 2015-12 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** ACTIVE_NOT_RECRUITING #### Last Update Post Date **Date:** 2015-02-06 **Type:** ESTIMATED **Last Update Submit Date:** 2015-02-04 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2015-12 **Type:** ESTIMATED #### Start Date **Date:** 2008-09 **Status Verified Date:** 2015-02 #### Study First Post Date **Date:** 2008-08-20 **Type:** ESTIMATED **Study First Submit Date:** 2008-08-19 **Study First Submit QC Date:** 2008-08-19 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Philips Respironics **Class:** OTHER **Name:** National Health and Medical Research Council, Australia **Class:** INDUSTRY **Name:** ResMed **Class:** INDUSTRY **Name:** Fisher and Paykel Healthcare **Class:** OTHER **Name:** The George Institute **Class:** OTHER **Name:** Health Research Council, New Zealand #### Lead Sponsor **Class:** OTHER **Name:** Adelaide Institute for Sleep Health #### Responsible Party **Investigator Affiliation:** Adelaide Institute for Sleep Health **Investigator Full Name:** Professor R. Doug McEvoy **Investigator Title:** MD, Director **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** Obstructive Sleep Apnea (OSA) is a condition in which a person stops breathing for several seconds at a time due to relaxation of the throat muscles. This can occur many times during sleep. It is known to cause sleepiness and poor concentration during the day. Research indicates that OSA may be a modifiable risk factor for cardiovascular disease due to its association with hypertension, stroke, heart attack and sudden death. The standard therapy for symptomatic OSA is continuous positive airway pressure (CPAP). CPAP has been shown to effectively reduce snoring, obstructive episodes and daytime sleepiness and to modestly reduce blood pressure and other risk factors for cardiovascular disease. The overall aim of SAVE is to determine if CPAP can reduce the risk of heart attack, stroke or heart failure for people with OSA. **Detailed Description:** There is increasing evidence to indicate that OSA is an important modifiable risk factor for CV disease including stroke, MI, and heart failure. Increased nocturnal arterial blood pressure (BP), hypercoagulability, oxidative stress, inflammation, insulin resistance and cardiac arrhythmias are all associated with OSA. These effects are presumed to accelerate the progression of atheromatous disease, particularly within the coronary or cerebral vasculature. Moreover, OSA also appears to increase the risk of sudden death during sleep, which is different from the circadian pattern of sudden death in those without OSA, suggesting that episodes of apnea may have a direct triggering effect for cardiac arrhythmias or MI. CPAP is now standard therapy for symptomatic OSA, with adherence to treatment comparable to that of other therapies for common chronic diseases. CPAP can eliminate apneas and improve daytime sleepiness, mood and quality of life. Furthermore, short term (1-3 months) randomised controlled trials of CPAP have shown modest reductions in blood pressure (BP) and other markers of CV disease, including C-reactive protein (CRP) and coagulation. However, the epidemiological data is complicated by potential residual confounding factors and the randomised evidence is limited. Thus, a direct causal link between OSA and CV disease remains inconclusive. The management of OSA, therefore, remains principally directed towards symptom control rather than CV risk modification. The present trial aims to test whether long-term use of CPAP can reduce the incidence of CV events. If the trial shows that CPAP treatment of OSA reduces the incidence of CV events it will influence clinical practice toward the early detection and management of OSA, and add CPAP to the range of strategies available for the prevention of CV disease. ### Conditions Module **Conditions:** - Sleep Apnea - Cardiovascular Disease **Keywords:** - Continuous Positive Airway Pressure (CPAP) - Obstructive Sleep Apnea (OSA) - Cardiovascular (CV) - Cardiovascular Disease - Clinical Trial ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 2500 **Type:** ESTIMATED **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** CPAP plus standard care of cardiovascular risk factors **Intervention Names:** - Device: Continuous Positive Airway Pressure (CPAP) **Label:** 1 **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Standard care alone **Intervention Names:** - Other: Standard care **Label:** 2 **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - 1 **Description:** CPAP worn nightly **Name:** Continuous Positive Airway Pressure (CPAP) **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - 2 **Description:** Standard care of cardiovascular risk factors **Name:** Standard care **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Measure:** A composite of the CV endpoints of CV death, non-fatal acute myocardial infarction, non-fatal stroke, hospital admission for heart failure, and new hospitalisation for unstable angina or transient ischaemic attack. **Time Frame:** Reviewed 6-monthly; average patient follow up, 4.5 years #### Secondary Outcomes **Measure:** Composite of CV death, MI & ischaemic stroke; components of primary composite endpoint; re-vascularisation procedures; all-cause death; new onset atrial fibrillation; new onset diabetes; OSA symptom scores; mood; health-related quality of life. **Time Frame:** Reviewed 6-monthly; average patient follow up, 4.5 years. **Measure:** In a sub-sample of 600 subjects pathophysiological mechanisms of CPAP-induced CV event reduction will be explored by assessing various intermediate markers of CV risk **Time Frame:** baseline and at 6-months, 2 and 4 years following randomisation **Description:** In a sub-sample of 150 participants (75 from the CPAP plus standard treatment and 75 from the standard treatment arms) the effect of CPAP on cardiac and vascular function using cardiac MRI will be investigated. The sub-study will evaluate left and right ventricular mass, volume and systolic/diastolic function and compliance of the aorta. **Measure:** Cardiac MRI to assess effects of CPAP on cardiac structure and function. **Time Frame:** Randomisation and at 6 months follow-up ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Males and females, any race, and aged between 45 and 75 years 2. Evidence of established coronary or cerebrovascular disease as evident by: * Coronary artery disease * Previous MI (equal to or greater than 90 days prior to ApneaLinkTM assessment) * Stable angina or unstable angina (Clinical event equal to or greater than 30 days and confirmatory test equal to or greater than 7 days prior to ApneaLinkTM assessment) defined as either ≥70% diameter stenosis of at least one major epicardial artery segment, or ≥50% diameter stenosis of the left main coronary artery, or \>50% stenosis in at least two major epicardial arteries.; or positive stress test (ST depression equal to or greater than 2 mm or a positive nuclear perfusion scintigram) * Multi-vessel percutaneous angioplasty (PTCA) and/or stent equal to or greater than 90 days prior to ApneaLinkTM assessment * Multi-vessel coronary artery bypass surgery (CABG) \>1 year prior to ApneaLinkTM assessment * Cerebrovascular disease * Previous stroke (includes definite or presumed cerebral ischaemia/infarction and intracerebral but not subarachnoid haemorrhage) equal to or greater than 90 days prior to ApneaLinkTM assessment or minor disabling stroke with minimal residual neurological disability (modified Rankin Score of '0 = no symptoms' or '1 = No significant disability despite symptoms, able to carry out all usual duties and activities' within 7 days of stroke onset) ≥7 days prior to ApneaLinkTM assessment. * Previous transient ischaemic event (TIA) of the brain or retina (symptoms \<24 hours) but not of presumed vertebrobasilar system ischemia. The TIA diagnosis must be confirmed by a suitably qualified clinician (≥7 days but \<1year prior to ApneaLinkTM assessment) 3. Patients have moderate-severe OSA (equivalent to apnea plus hypopneas index \[AHI\] \>30 per hour of sleep) as determined by a ≥ 4% oxygen dip rate \> 12/ h on overnight testing using the ApneaLinkTM device and confirmed by the SAVE core lab in Adelaide upon receipt of the ApneaLinkTM data 4. Patients are able and willing to give appropriate informed consent Exclusion Criteria: Patients will be excluded from entry if ANY of the criteria listed below are met: 1. Any condition that in the opinion of the responsible physician or investigator makes the potential participant unsuitable for the study. For example, * co-morbid disease with severe disability or likelihood of death * significant memory, perceptual, or behavioural disorder * neurological deficit (e.g. limb paresis) preventing self administration of the CPAP mask * contraindication to CPAP use e.g. pneumothorax * residence sufficiently remote from the clinic to preclude follow-up clinic visits 2. Any planned coronary or carotid revascularisation procedure in the next 6 months 3. Severe respiratory disease defined as * severe chronic obstructive pulmonary disease (FEV1/FVC \< 70% and FEV1 \< 50% predicted), or * resting, awake SaO2 \< 90% by ApneaLinkTM device 4. New York Heart Association (NYHA) categories III-IV of heart failure 5. Other household member enrolled in SAVE trial or using CPAP 6. Prior use of CPAP treatment for OSA 7. Increased risk of a sleep-related accident and/or excessive daytime sleepiness, defined by any one of the following: * driver occupation (eg truck, taxi) * 'fall-asleep' accident or 'near miss' accident in previous 12 months * high (\> 15) score on the Epworth Sleepiness Scale 8. Severe nocturnal desaturation documented on the ApneaLinkTM device as \> 10% overnight recording time with arterial oxygen saturation of \< 80% 9. Cheyne-Stokes Respiration (CSResp) * CSResp identified on ApneaLinkTM nasal pressure recording by typical crescendo-decrescendo pattern of respiration with associated apneas and/or hypopneas in the absence of inspiratory flow limitation. * patients excluded if \> 50% of nasal pressure - defined apneas and hypopneas judged to be due to CSResp. **Maximum Age:** 75 Years **Minimum Age:** 45 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Adelaide **Country:** Australia **Facility:** Adelaide Institute for Sleep Health, Repatriation General Hospital **State:** South Australia **Zip:** 5041 **Location 2:** **City:** São Paulo **Country:** Brazil **Facility:** Brazil Principal Investigator: Geraldo Lorenzi Filho, Heart Institute, University of São Paulo **Location 3:** **City:** Beijing **Country:** China **Facility:** Regional Coordinating Centre China: The George Institute China Beijing **State:** Beijing **Zip:** 100088 **Location 4:** **City:** Hyderabad **Country:** India **Facility:** Regional Coordinating Centre India: The George Institute India 839C, Road No. 44A Jubilee Hills **State:** Andhra Pradesh **Zip:** 500 033 **Location 5:** **City:** Barcelona **Country:** Spain **Facility:** Regional Coordinating Centre Spain: Spanish Respiratory Society (Sociedad Española de Neumología y Cirugía Torácica) (SEPAR) **Zip:** 08029 #### Overall Officials **Official 1:** **Affiliation:** Adelaide Institute for Sleep Health **Name:** R D McEvoy **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Cheng Y, Ou Q, Chen B, Loffler KA, Doug McEvoy R, Xu Y, Wang Q, Lao M. The changes of AHI after long-term CPAP in patients with comorbid OSA and cardiovascular disease. Sleep Breath. 2023 May;27(2):511-518. doi: 10.1007/s11325-022-02633-y. Epub 2022 May 14. **PMID:** 35562555 **Citation:** Lao M, Cheng Y, Gao X, Ou Q. The interaction among OSA, CPAP, and medications in patients with comorbid OSA and cardiovascular/cerebrovascular disease: a randomized controlled trial. BMC Pulm Med. 2022 Mar 21;22(1):99. doi: 10.1186/s12890-022-01879-2. **PMID:** 35313858 **Citation:** Stevens D, Loffler KA, Buman MP, Dunstan DW, Luo Y, Lorenzi-Filho G, Barbe FE, Anderson CS, McEvoy RD; SAVE investigators. CPAP increases physical activity in obstructive sleep apnea with cardiovascular disease. J Clin Sleep Med. 2021 Feb 1;17(2):141-148. doi: 10.5664/jcsm.8792. **PMID:** 32951632 **Citation:** Li J, McEvoy RD, Zheng D, Loffler KA, Wang X, Redline S, Woodman RJ, Anderson CS. Self-reported Snoring Patterns Predict Stroke Events in High-Risk Patients With OSA: Post Hoc Analyses of the SAVE Study. Chest. 2020 Nov;158(5):2146-2154. doi: 10.1016/j.chest.2020.05.615. Epub 2020 Jul 15. **PMID:** 32679238 **Citation:** Loffler KA, Heeley E, Freed R, Meng R, Bittencourt LR, Gonzaga Carvalho CC, Chen R, Hlavac M, Liu Z, Lorenzi-Filho G, Luo Y, McArdle N, Mukherjee S, Yap HS, Zhang X, Palmer LJ, Anderson CS, McEvoy RD, Drager LF; SAVE Substudy Investigators. Continuous Positive Airway Pressure Treatment, Glycemia, and Diabetes Risk in Obstructive Sleep Apnea and Comorbid Cardiovascular Disease. Diabetes Care. 2020 Aug;43(8):1859-1867. doi: 10.2337/dc19-2006. Epub 2020 Apr 14. **PMID:** 32291275 **Citation:** Li J, Zheng D, Loffler KA, Wang X, McEvoy RD, Woodman RJ, Luo Y, Lorenzi-Filho G, Barbe F, Tripathi M, Anderson CS; SAVE Investigators. Sleep duration and risk of cardiovascular events: The SAVE study. Int J Stroke. 2020 Oct;15(8):858-865. doi: 10.1177/1747493020904913. Epub 2020 Feb 3. **PMID:** 32013799 **Citation:** Van Ryswyk E, Anderson CS, Antic NA, Barbe F, Bittencourt L, Freed R, Heeley E, Liu Z, Loffler KA, Lorenzi-Filho G, Luo Y, Margalef MJM, McEvoy RD, Mediano O, Mukherjee S, Ou Q, Woodman R, Zhang X, Chai-Coetzer CL. Predictors of long-term adherence to continuous positive airway pressure in patients with obstructive sleep apnea and cardiovascular disease. Sleep. 2019 Oct 9;42(10):zsz152. doi: 10.1093/sleep/zsz152. **PMID:** 31587046 **Citation:** Ou Q, Chen B, Loffler KA, Luo Y, Zhang X, Chen R, Wang Q, Drager LF, Lorenzi-Filho G, Hlavac M, McArdle N, Mukherjee S, Mediano O, Barbe F, Anderson CS, McEvoy RD, Woodman RJ; SAVE investigators. The Effects of Long-term CPAP on Weight Change in Patients With Comorbid OSA and Cardiovascular Disease: Data From the SAVE Trial. Chest. 2019 Apr;155(4):720-729. doi: 10.1016/j.chest.2018.08.1082. Epub 2018 Sep 27. **PMID:** 30268694 **Citation:** Loffler KA, Heeley E, Freed R, Anderson CS, Brockway B, Corbett A, Chang CL, Douglas JA, Ferrier K, Graham N, Hamilton GS, Hlavac M, McArdle N, McLachlan J, Mukherjee S, Naughton MT, Thien F, Young A, Grunstein RR, Palmer LJ, Woodman RJ, Hanly PJ, McEvoy RD; SAVE (Sleep Apnea Cardiovascular Endpoints) Investigators. Effect of Obstructive Sleep Apnea Treatment on Renal Function in Patients with Cardiovascular Disease. Am J Respir Crit Care Med. 2017 Dec 1;196(11):1456-1462. doi: 10.1164/rccm.201703-0603OC. **PMID:** 28743190 **Citation:** McEvoy RD, Antic NA, Heeley E, Luo Y, Ou Q, Zhang X, Mediano O, Chen R, Drager LF, Liu Z, Chen G, Du B, McArdle N, Mukherjee S, Tripathi M, Billot L, Li Q, Lorenzi-Filho G, Barbe F, Redline S, Wang J, Arima H, Neal B, White DP, Grunstein RR, Zhong N, Anderson CS; SAVE Investigators and Coordinators. CPAP for Prevention of Cardiovascular Events in Obstructive Sleep Apnea. N Engl J Med. 2016 Sep 8;375(10):919-31. doi: 10.1056/NEJMoa1606599. Epub 2016 Aug 28. **PMID:** 27571048 **Citation:** Antic NA, Heeley E, Anderson CS, Luo Y, Wang J, Neal B, Grunstein R, Barbe F, Lorenzi-Filho G, Huang S, Redline S, Zhong N, McEvoy RD. The Sleep Apnea cardioVascular Endpoints (SAVE) Trial: Rationale, Ethics, Design, and Progress. Sleep. 2015 Aug 1;38(8):1247-57. doi: 10.5665/sleep.4902. **PMID:** 25669180 **Citation:** Chai-Coetzer CL, Luo YM, Antic NA, Zhang XL, Chen BY, He QY, Heeley E, Huang SG, Anderson C, Zhong NS, McEvoy RD. Predictors of long-term adherence to continuous positive airway pressure therapy in patients with obstructive sleep apnea and cardiovascular disease in the SAVE study. Sleep. 2013 Dec 1;36(12):1929-37. doi: 10.5665/sleep.3232. **PMID:** 24293768 #### See Also Links **Label:** Related Info **URL:** http://www.savetrial.org ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012120 - Term: Respiration Disorders - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000012818 - Term: Signs and Symptoms, Respiratory - ID: D000020919 - Term: Sleep Disorders, Intrinsic - ID: D000020920 - Term: Dyssomnias - ID: D000012893 - Term: Sleep Wake Disorders - ID: D000009422 - Term: Nervous System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BXM - Name: Behaviors and Mental Disorders ### Condition Browse Module - Browse Leaves - ID: M4361 - Name: Apnea - Relevance: HIGH - As Found: Apnea - ID: M15694 - Name: Sleep Apnea Syndromes - Relevance: HIGH - As Found: Sleep Apnea - ID: M22010 - Name: Sleep Apnea, Obstructive - Relevance: HIGH - As Found: Obstructive Sleep Apnea - ID: M16355 - Name: Syndrome - Relevance: LOW - As Found: Unknown - ID: M14957 - Name: Respiration Disorders - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown - ID: M15623 - Name: Signs and Symptoms, Respiratory - Relevance: LOW - As Found: Unknown - ID: M22242 - Name: Parasomnias - Relevance: LOW - As Found: Unknown - ID: M22654 - Name: Sleep Disorders, Intrinsic - Relevance: LOW - As Found: Unknown - ID: M22655 - Name: Dyssomnias - Relevance: LOW - As Found: Unknown - ID: M15696 - Name: Sleep Wake Disorders - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001049 - Term: Apnea - ID: D000012891 - Term: Sleep Apnea Syndromes - ID: D000020181 - Term: Sleep Apnea, Obstructive - ID: D000002318 - Term: Cardiovascular Diseases ### Misc Info Module #### Removed Countries - Country: United Kingdom - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03430479 **Brief Title:** Anti PD-1 Antibody With Radiation Therapy in Patients With HER2-negative Metastatic Breast Cancer **Official Title:** Phase Ib/II Study to Assess Efficacy, Safety & Immunological Biomarker of Anti PD-1 Antibody With Radiation Therapy in Patients With HER2-negative Metastatic Breast Cancer #### Organization Study ID Info **ID:** kbcrnb002 #### Organization **Class:** OTHER **Full Name:** Kyoto Breast Cancer Research Network ### Status Module #### Completion Date **Date:** 2021-04-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-01-19 **Type:** ACTUAL **Last Update Submit Date:** 2023-01-18 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2018-11-30 **Type:** ACTUAL #### Start Date **Date:** 2017-06-22 **Type:** ACTUAL **Status Verified Date:** 2023-01 #### Study First Post Date **Date:** 2018-02-13 **Type:** ACTUAL **Study First Submit Date:** 2018-02-06 **Study First Submit QC Date:** 2018-02-06 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Kyoto Breast Cancer Research Network #### 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 aim of this study is to evaluate the safety and efficacy of anti PD-1 antibody with radiation therapy in patients with HER2-negative metastatic breast cancer. ### Conditions Module **Conditions:** - Breast Cancer ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 32 **Type:** ACTUAL **Phases:** - PHASE1 - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Drug: Cohort A **Label:** Cohort A **Type:** EXPERIMENTAL #### Arm Group 2 **Intervention Names:** - Drug: Cohort A **Label:** Cohort B **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Cohort A - Cohort B **Description:** Radiation + Nivolumab + hormone therapy **Name:** Cohort A **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Phase Ib : dose-limiting toxicity rate **Time Frame:** 2 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: - Cohort A 1. Documentation of ER-positive and/or PR-positive tumor (\>=1% positive stained cells) . 2. Patients must satisfy the following criteria for prior therapy: - Progressed during treatment or within 12 months of completion of adjuvant hormone therapy. or Progressed while prior hormone therapy for advanced/metastatic breast cancer. Two previous line of hormone therapy for advanced/metastatic disease is allowed. 3. Patients who have hormone therapy that can be expected for advanced /metastatic disease. Cohort B 4. Patients who have come to be non-responsive more than two line of chemotherapy 5. Prior chemotherapy with anthracycline and taxane agent Cohort A and B 6. Female patients who are histologically or cytologically confirmed to have breast cancer 7. Patients who have distant metastatic lesion as follow - More than one bone lesion for radiation therapy 8. Patients with cancer confirmed to be HER2-negative.（ 9. Patients with a measurable lesion based on RECIST 1.1. 10. Patients aged \>= 20 years at informed consent 11. Patients with ECOG PS of 0 to 1. 12. Patients without any severe disorder in the major organs. 13. Patients expected to survive for ≥ 90 days. 14. Patients of childbearing potential must be using an acceptable method of contraception to avoid pregnancy and must not be breastfeeding for 18 weeks after the last dose of investigational product 15. Patients who have provided written informed consent themselves. Exclusion Criteria: - Exclusion Criteria: 1. Patients who have neuropathy (more than Grade 2) 2. Patients with any active autoimmune disease or a history of known autoimmune disease. 3. Patients who has a history of pneumonitis or interstitial lung disease. 4. Active, untreated central nervous system metastasis. 5. Patients with pericardial effusion, pleural effusion or ascites requiring treatment 6. Patients with uncontrolled diabetes mellitus 7. Patients with a condition requiring systemic treatment with either corticosteroids or other immunosuppressive medications within 28 days of the enrollment. 8. Patients who has received radiotherapy within 28 days of study registration, or radiotherapy for thorax within 56 days of the enrollment. 9. Pregnant or breast-feeding women. 10. Prior therapy with Nivolumab, anti CTLA-4 antibody therapies, any other antibody or drug specifically targeting T-cell co-stimulation or checkpoint pathways 11. Patients considered ineligible for participation in this study by their attending physicians **Minimum Age:** 20 Years **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Kyoto **Country:** Japan **Facility:** Kyoto University Hospital **Zip:** 606-8507 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000001941 - Term: Breast Diseases - ID: D000012871 - Term: Skin Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M5220 - Name: Breast Neoplasms - Relevance: HIGH - As Found: Breast Cancer - ID: M5218 - Name: Breast Diseases - Relevance: LOW - As Found: Unknown - ID: M15674 - Name: Skin Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001943 - Term: Breast Neoplasms ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: ANeo - Name: Antineoplastic Agents ### Intervention Browse Module - Browse Leaves - ID: M9789 - Name: Hormones - Relevance: LOW - As Found: Unknown - ID: M4225 - Name: Antibodies - Relevance: LOW - As Found: Unknown - ID: M1854 - Name: Nivolumab - Relevance: LOW - As Found: Unknown - ID: M10184 - Name: Immunoglobulins - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01189279 **Brief Title:** Safety and Pharmacokinetics Study of New Formulation of Bimatoprost in Patients With Alopecia #### Organization Study ID Info **ID:** 192024-053 #### Organization **Class:** INDUSTRY **Full Name:** Allergan ### Status Module #### Completion Date **Date:** 2011-02 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2013-08-30 **Type:** ESTIMATED **Last Update Submit Date:** 2013-06-24 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2011-01 **Type:** ACTUAL #### Results First Post Date **Date:** 2013-08-30 **Type:** ESTIMATED **Results First Submit Date:** 2013-06-24 **Results First Submit QC Date:** 2013-06-24 #### Start Date **Date:** 2010-10 **Status Verified Date:** 2013-06 #### Study First Post Date **Date:** 2010-08-26 **Type:** ESTIMATED **Study First Submit Date:** 2010-08-25 **Study First Submit QC Date:** 2010-08-25 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Allergan #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** This study will investigate the safety, tolerability, and pharmacokinetics of new formulation of bimatoprost following topical application in patients with alopecia. Two formulations of bimatoprost will be investigated in Part 1 and a third formulation of bimatoprost will be investigated in Part 2. Part 2 will begin after Part 1 has completed. ### Conditions Module **Conditions:** - Alopecia - Alopecia, Androgenetic - Baldness ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - INVESTIGATOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 42 **Type:** ACTUAL **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** bimatoprost Formulation A applied topically to the scalp once daily on Day 1 and Days 4-17. **Intervention Names:** - Drug: bimatoprost Formulation A **Label:** Part 1: bimatoprost Formulation A **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** bimatoprost Formulation B applied topically to the scalp once daily on Day 1 and Days 4-17. **Intervention Names:** - Drug: bimatoprost Formulation B **Label:** Part 1: bimatoprost Formulation B **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** bimatoprost Formulation C applied topically to the scalp once daily on Day 1 and Days 4-17. **Intervention Names:** - Drug: bimatoprost Formulation C **Label:** Part 2: bimatoprost Formulation C **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Part 1: bimatoprost Formulation A **Description:** bimatoprost Formulation A applied topically to the scalp once daily on Day 1 and Days 4-17. **Name:** bimatoprost Formulation A **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Part 1: bimatoprost Formulation B **Description:** bimatoprost Formulation B applied topically to the scalp once daily on Day 1 and Days 4-17. **Name:** bimatoprost Formulation B **Type:** DRUG #### Intervention 3 **Arm Group Labels:** - Part 2: bimatoprost Formulation C **Description:** bimatoprost Formulation C applied topically to the scalp once daily on Day 1 and Days 4-17. **Name:** bimatoprost Formulation C **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Cmax is the maximum plasma level following a single dose of bimatoprost. Plasma is the fluid portion of the blood in which the cells are suspended. **Measure:** Maximum Plasma Level (Cmax) Following a Single Dose of Bimatoprost **Time Frame:** Day 1 **Description:** Cmax is the maximum plasma level following multiple doses of bimatoprost. Plasma is the fluid portion of the blood in which the cells are suspended. **Measure:** Maximum Plasma Level (Cmax) Following Multiple Doses of Bimatoprost **Time Frame:** 17 Days #### Secondary Outcomes **Description:** An ECG is a tracing of the heart's electrical activity over time in waves with points identified at P, Q, R, S, and T \[measured in milliseconds (ms)\], as well as the heart rate \[measured in beats per minute (bpm)\]. Clinically significant abnormal results include maximum post-treatment QTcB\>500 ms, maximum post-treatment QTcF\>500 ms, maximum post-treatment QT interval \>500 ms, PR interval 25% increase from baseline and \>200 ms, QRS interval 25% increase from baseline and \>100 ms, heart rate 25% increase from baseline and \>100 bpm, and heart rate 25% decrease from baseline and \<50 bpm. **Measure:** Percentage of Patients With Clinically Significant Electrocardiogram (ECG) Findings **Time Frame:** 17 Days **Description:** Local scalp tolerability by patient assessment is based on a 4-point scale (0=None, 1=Mild, 2=Moderate, and 3=Severe) for 3 symptoms (burning, itching, and stinging). An at least 1-grade increase from baseline at any timepoint indicates a worsening of symptoms. **Measure:** Number of Patients With an at Least 1-Grade Severity Increase in Local Scalp Tolerability by Patient Assessment **Time Frame:** Baseline, 20 Days **Description:** Local scalp tolerability by dermatologist assessment is based on a 4-point scale (0=None, 1=Mild, 2=Moderate, and 3=Severe) for 5 symptoms (dryness/scaling, edema, erythema, folliculitis, and pigmentation). An at least 1-grade increase at any timepoint from baseline indicates a worsening of symptoms. **Measure:** Number of Patients With an at Least 1-Grade Severity Increase in Local Scalp Tolerability by Dermatologist Assessment **Time Frame:** Baseline, 20 Days ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Males with moderate male-pattern baldness (androgenic alopecia) * Females with moderate female pattern hair loss * Non-smoker or smoker with at least 30 days abstinence from smoking/using nicotine-containing products Exclusion Criteria: * Any dermatological condition of the scalp other than androgenic alopecia (males) or female pattern hair loss (females) * Use of bimatoprost or other prostaglandin analogs within 3 months * Prior use of scalp hair growth treatment (eg, finasteride, minoxidil) within 6 months * Any prior hair growth procedures (eg, hair transplant or laser) * Blood donation or equivalent blood loss within 90 days * History of alcohol or drug addiction **Maximum Age:** 64 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Tempe **Country:** United States **State:** Arizona #### Overall Officials **Official 1:** **Affiliation:** Allergan **Name:** Medical Director **Role:** STUDY_DIRECTOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000007039 - Term: Hypotrichosis - ID: D000006201 - Term: Hair Diseases - ID: D000012871 - Term: Skin Diseases - ID: D000020763 - Term: Pathological Conditions, Anatomical ### Condition Browse Module - Browse Branches - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M3846 - Name: Alopecia - Relevance: HIGH - As Found: Alopecia - ID: M3847 - Name: Alopecia Areata - Relevance: HIGH - As Found: Alopecia - ID: M10089 - Name: Hypotrichosis - Relevance: LOW - As Found: Unknown - ID: M9293 - Name: Hair Diseases - Relevance: LOW - As Found: Unknown - ID: M15674 - Name: Skin Diseases - Relevance: LOW - As Found: Unknown - ID: M22519 - Name: Pathological Conditions, Anatomical - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000000505 - Term: Alopecia - ID: D000000506 - Term: Alopecia Areata ### Intervention Browse Module - Ancestors - ID: D000000959 - Term: Antihypertensive Agents ### Intervention Browse Module - Browse Branches - Abbrev: AnAg - Name: Antihypertensive Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M473 - Name: Bimatoprost - Relevance: HIGH - As Found: Port - ID: M4277 - Name: Antihypertensive Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000069580 - Term: Bimatoprost ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Part 1: Bimatoprost Formulation A **Description:** bimatoprost Formulation A applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** EG000 **Other Num Affected:** 7 **Other Num at Risk:** 14 **Serious Number At Risk:** 14 **Title:** Part 1: Bimatoprost Formulation A **Group ID:** EG001 **Title:** Part 1: Bimatoprost Formulation B **Description:** bimatoprost Formulation B applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** EG001 **Other Num Affected:** 6 **Other Num at Risk:** 14 **Serious Number At Risk:** 14 **Title:** Part 1: Bimatoprost Formulation B **Group ID:** EG002 **Title:** Part 2: Bimatoprost Formulation C **Description:** bimatoprost Formulation C applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** EG002 **Other Num Affected:** 1 **Other Num at Risk:** 14 **Serious Number At Risk:** 14 **Title:** Part 2: Bimatoprost Formulation C **Frequency Threshold:** 5 #### Other Events **Term:** Headache **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA version 13.1 **Term:** Feeling Hot **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA version 13.1 **Term:** Myalgia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA version 13.1 **Term:** Neck Pain **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA version 13.1 **Term:** Rhinorrhoea **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA version 13.1 **Term:** Somnolence **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA version 13.1 **Term:** Application Site Folliculitis **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA version 13.1 **Term:** Papule **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA version 13.1 **Term:** Procedural Hypotension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA version 13.1 **Term:** Pruritus **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA version 13.1 ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 14 **Group ID:** BG001 **Value:** 14 **Group ID:** BG002 **Value:** 14 **Group ID:** BG003 **Value:** 42 **Units:** Participants ### Group **ID:** BG000 **Title:** Part 1: Bimatoprost Formulation A **Description:** bimatoprost Formulation A applied topically to the scalp once daily on Day 1 and Days 4-17. ### Group **ID:** BG001 **Title:** Part 1: Bimatoprost Formulation B **Description:** bimatoprost Formulation B applied topically to the scalp once daily on Day 1 and Days 4-17. ### Group **ID:** BG002 **Title:** Part 2: Bimatoprost Formulation C **Description:** bimatoprost Formulation C applied topically to the scalp once daily on Day 1 and Days 4-17. ### Group **ID:** BG003 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Value:** 5 #### Measurement **Group ID:** BG001 **Value:** 4 #### Measurement **Group ID:** BG002 **Value:** 8 #### Measurement **Group ID:** BG003 **Value:** 17 **Class Title:** <45 years #### Measurement **Group ID:** BG000 **Value:** 9 #### Measurement **Group ID:** BG001 **Value:** 10 #### Measurement **Group ID:** BG002 **Value:** 6 #### Measurement **Group ID:** BG003 **Value:** 25 **Class Title:** 45 to 64 years ### Measure #### Measurement **Group ID:** BG000 **Value:** 7 #### Measurement **Group ID:** BG001 **Value:** 7 #### Measurement **Group ID:** BG002 **Value:** 7 #### Measurement **Group ID:** BG003 **Value:** 21 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 7 #### Measurement **Group ID:** BG001 **Value:** 7 #### Measurement **Group ID:** BG002 **Value:** 7 #### Measurement **Group ID:** BG003 **Value:** 21 **Category Title:** Male **Class Title:** **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Parameter Type:** NUMBER **Title:** Age, Customized **Unit of Measure:** Participants ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement **Other Details:** A disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 90 days from the time submitted to the sponsor for review. The sponsor cannot require changes to the communication and cannot extend the embargo. **Restriction Type:** OTHER **Restrictive Agreement:** True ### Point of Contact **Email:** [email protected] **Organization:** Allergan, Inc **Phone:** 714-246-4500 **Title:** Therapeutic Area Head, ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ### Outcome Measure 3 ### Outcome Measure 4 ### Outcome Measure 5 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.575 - **Upper Limit:** - **Value:** 0.552 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 1.11 - **Upper Limit:** - **Value:** 1.77 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** 3.66 - **Upper Limit:** - **Value:** 5.58 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.60 - **Upper Limit:** - **Value:** 1.26 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 1.66 - **Upper Limit:** - **Value:** 3.01 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** 7.0 - **Upper Limit:** - **Value:** 10.1 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 20.238 - **Upper Limit:** - **Value:** 0.0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 23.460 - **Upper Limit:** - **Value:** 0.0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** 19.667 - **Upper Limit:** - **Value:** 0.0 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG002 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Cmax is the maximum plasma level following a single dose of bimatoprost. Plasma is the fluid portion of the blood in which the cells are suspended. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Per Protocol: all subjects with no major protocol deviations **Reporting Status:** POSTED **Time Frame:** Day 1 **Title:** Maximum Plasma Level (Cmax) Following a Single Dose of Bimatoprost **Type:** PRIMARY **Unit of Measure:** Picograms/Milliliter (pg/mL) ##### Group **Description:** bimatoprost Formulation A applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** OG000 **Title:** Part 1: Bimatoprost Formulation A ##### Group **Description:** bimatoprost Formulation B applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** OG001 **Title:** Part 1: Bimatoprost Formulation B ##### Group **Description:** bimatoprost Formulation C applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** OG002 **Title:** Part 2: Bimatoprost Formulation C #### Outcome Measure 2 **Description:** Cmax is the maximum plasma level following multiple doses of bimatoprost. Plasma is the fluid portion of the blood in which the cells are suspended. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Per Protocol: all subjects with no major protocol deviations **Reporting Status:** POSTED **Time Frame:** 17 Days **Title:** Maximum Plasma Level (Cmax) Following Multiple Doses of Bimatoprost **Type:** PRIMARY **Unit of Measure:** Picograms/Milliliter (pg/mL) ##### Group **Description:** bimatoprost Formulation A applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** OG000 **Title:** Part 1: Bimatoprost Formulation A ##### Group **Description:** bimatoprost Formulation B applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** OG001 **Title:** Part 1: Bimatoprost Formulation B ##### Group **Description:** bimatoprost Formulation C applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** OG002 **Title:** Part 2: Bimatoprost Formulation C #### Outcome Measure 3 **Description:** An ECG is a tracing of the heart's electrical activity over time in waves with points identified at P, Q, R, S, and T \[measured in milliseconds (ms)\], as well as the heart rate \[measured in beats per minute (bpm)\]. Clinically significant abnormal results include maximum post-treatment QTcB\>500 ms, maximum post-treatment QTcF\>500 ms, maximum post-treatment QT interval \>500 ms, PR interval 25% increase from baseline and \>200 ms, QRS interval 25% increase from baseline and \>100 ms, heart rate 25% increase from baseline and \>100 bpm, and heart rate 25% decrease from baseline and \<50 bpm. **Parameter Type:** NUMBER **Population Description:** Safety Population: all subjects who received at least 1 dose of study medication **Reporting Status:** POSTED **Time Frame:** 17 Days **Title:** Percentage of Patients With Clinically Significant Electrocardiogram (ECG) Findings **Type:** SECONDARY **Unit of Measure:** Percentage of Patients ##### Group **Description:** bimatoprost Formulation A applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** OG000 **Title:** Part 1: Bimatoprost Formulation A ##### Group **Description:** bimatoprost Formulation B applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** OG001 **Title:** Part 1: Bimatoprost Formulation B ##### Group **Description:** bimatoprost Formulation C applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** OG002 **Title:** Part 2: Bimatoprost Formulation C #### Outcome Measure 4 **Description:** Local scalp tolerability by patient assessment is based on a 4-point scale (0=None, 1=Mild, 2=Moderate, and 3=Severe) for 3 symptoms (burning, itching, and stinging). An at least 1-grade increase from baseline at any timepoint indicates a worsening of symptoms. **Parameter Type:** NUMBER **Population Description:** Safety Population: all subjects who received at least 1 dose of study medication **Reporting Status:** POSTED **Time Frame:** Baseline, 20 Days **Title:** Number of Patients With an at Least 1-Grade Severity Increase in Local Scalp Tolerability by Patient Assessment **Type:** SECONDARY **Unit of Measure:** Patients ##### Group **Description:** bimatoprost Formulation A applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** OG000 **Title:** Part 1: Bimatoprost Formulation A ##### Group **Description:** bimatoprost Formulation B applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** OG001 **Title:** Part 1: Bimatoprost Formulation B ##### Group **Description:** bimatoprost Formulation C applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** OG002 **Title:** Part 2: Bimatoprost Formulation C #### Outcome Measure 5 **Description:** Local scalp tolerability by dermatologist assessment is based on a 4-point scale (0=None, 1=Mild, 2=Moderate, and 3=Severe) for 5 symptoms (dryness/scaling, edema, erythema, folliculitis, and pigmentation). An at least 1-grade increase at any timepoint from baseline indicates a worsening of symptoms. **Parameter Type:** NUMBER **Population Description:** Safety Population: all subjects who received at least 1 dose of study medication **Reporting Status:** POSTED **Time Frame:** Baseline, 20 Days **Title:** Number of Patients With an at Least 1-Grade Severity Increase in Local Scalp Tolerability by Dermatologist Assessment **Type:** SECONDARY **Unit of Measure:** Patients ##### Group **Description:** bimatoprost Formulation A applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** OG000 **Title:** Part 1: Bimatoprost Formulation A ##### Group **Description:** bimatoprost Formulation B applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** OG001 **Title:** Part 1: Bimatoprost Formulation B ##### Group **Description:** bimatoprost Formulation C applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** OG002 **Title:** Part 2: Bimatoprost Formulation C ### Participant Flow Module #### Group **Description:** bimatoprost Formulation A applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** FG000 **Title:** Part 1: Bimatoprost Formulation A #### Group **Description:** bimatoprost Formulation B applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** FG001 **Title:** Part 1: Bimatoprost Formulation B #### Group **Description:** bimatoprost Formulation C applied topically to the scalp once daily on Day 1 and Days 4-17. **ID:** FG002 **Title:** Part 2: Bimatoprost Formulation C #### Period **Title:** Overall Study ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 14 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 14 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 14 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 13 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 14 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 13 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 1 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 0 ###### Achievement **Group ID:** FG002 **Number of Subjects:** 1 **Pre-Assignment Details:** Part 1 of the study was double-blind, followed by Part 2 which was open-label. No patients enrolled in Part 1 were enrolled in Part 2 of the study. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT04187079 **Brief Title:** Immunopathologic Profiles and Blood Biomarkers in Patients With IPF **Official Title:** Immunopathologic Profiles of the Lung Micro- Environment Using Cryobiopsies and Identification of Blood Biomarkers in Patients With IPF #### Organization Study ID Info **ID:** Cryobiopsy in IPF #### Organization **Class:** OTHER **Full Name:** University of Aarhus ### Status Module #### Completion Date **Date:** 2021-07-27 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-08-10 **Type:** ACTUAL **Last Update Submit Date:** 2021-08-09 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2019-05-01 **Type:** ACTUAL #### Start Date **Date:** 2017-09-18 **Type:** ACTUAL **Status Verified Date:** 2021-07 #### Study First Post Date **Date:** 2019-12-05 **Type:** ACTUAL **Study First Submit Date:** 2019-12-02 **Study First Submit QC Date:** 2019-12-02 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Aarhus University Hospital #### Lead Sponsor **Class:** OTHER **Name:** University of Aarhus #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** Examination of expression of PD-L1, PD-L2, Beta- catenin, B-cell follicles and Tenascin- C in patients with IPF compared with other interstitial lung diseases. Examination of anti HSP 70, p-ANCA, c-ANCA, CD4+/CD28- and CD8+/CD28- cells in patients with IPF compared with other interstitial lung diseases. Compare the above mentioned findings with changes in pulmonary function tests, 6 minute walking test, exacerbation and mortality over a 2 year follow-up period. **Detailed Description:** Examination of expression of PD-L1, PD-L2, Beta- catenin, B-cell follicles and Tenascin- C in cryobiopsies from the lungs inpatients with IPF compared with other interstitial lung diseases. Examination of anti HSP 70, p-ANCA, c-ANCA, CD4+/CD28- and CD8+/CD28- cells in blood samples in patients with IPF compared with other interstitial lung diseases. Compare the above mentioned findings with changes in pulmonary function tests, 6 minute walking test, exacerbation and mortality over a 2 year follow-up period. ### Conditions Module **Conditions:** - Idiopathic Pulmonary Fibrosis ### Design Module #### Bio Spec **Description:** Plasma Cryobiopsies **Retention:** SAMPLES_WITHOUT_DNA #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 100 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients diagnosed with idiopathic pulmonary fibrosis after multidisciplinary team discussion using medical history, HRCT pattern, laboratory findings, pulmonary function tests and cryobiopsy specimens. **Label:** IPF #### Arm Group 2 **Description:** Patients diagnosed with other interstitial diseases other than IPF after multidisciplinary team discussion using medical history, HRCT pattern, laboratory findings, pulmonary function tests and cryobiopsy specimens. **Label:** Other ILDs ### Outcomes Module #### Primary Outcomes **Description:** Expression of PD-L1 in the epithelial cells in lungs **Measure:** PD-L1 **Time Frame:** At diagnosis ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Patients suspected of having an ILD who are having cryobiopsies performed Exclusion Criteria: * BMI \> 35 * DLCO \< 35% * FVC \< 45% * Pulmonary hypertension * Cardiac or other severe comorbidity that will increase the risk of complications **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT **Study Population:** Patients suspected of having an ILD who are having cryobiopsies performed to get a histological specimen for the diagnoses. ### Contacts Locations Module #### Locations **Location 1:** **City:** Aarhus **Country:** Denmark **Facility:** Aarhus University Hospital, Department of Respiratory Diseases and Allergy **Zip:** 8000 #### Overall Officials **Official 1:** **Affiliation:** Department of Respiratory Diaseses, Aarhus University Hospital **Name:** Venerino Poletti, Prof **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000005355 - Term: Fibrosis - ID: D000010335 - Term: Pathologic Processes - ID: D000017563 - Term: Lung Diseases, Interstitial - ID: D000008171 - Term: Lung Diseases - ID: D000012140 - Term: Respiratory Tract Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M8485 - Name: Fibrosis - Relevance: LOW - As Found: Unknown - ID: M14512 - Name: Pulmonary Fibrosis - Relevance: HIGH - As Found: Pulmonary Fibrosis - ID: M27989 - Name: Idiopathic Pulmonary Fibrosis - Relevance: HIGH - As Found: Idiopathic Pulmonary Fibrosis - ID: M11168 - Name: Lung Diseases - Relevance: LOW - As Found: Unknown - ID: M19813 - Name: Lung Diseases, Interstitial - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown - ID: T3003 - Name: Idiopathic Pulmonary Fibrosis - Relevance: HIGH - As Found: Idiopathic Pulmonary Fibrosis ### Condition Browse Module - Meshes - ID: D000011658 - Term: Pulmonary Fibrosis - ID: D000054990 - Term: Idiopathic Pulmonary Fibrosis ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00635479 **Brief Title:** Role of Vacuum Assisted Closure (VAC) Device in Postoperative Management of Pelvic and Acetabular Fractures **Official Title:** Role of Vacuum Assisted Closure (VAC) Device in Postoperative Management of Pelvic and Acetabular Fractures #### Organization Study ID Info **ID:** IRB 1096320 #### Organization **Class:** OTHER **Full Name:** University of Missouri-Columbia #### Secondary ID Infos **ID:** 1138438 ### Status Module #### Completion Date **Date:** 2013-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-12-11 **Type:** ACTUAL **Last Update Submit Date:** 2017-11-08 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2013-09 **Type:** ACTUAL #### Results First Post Date **Date:** 2014-02-14 **Type:** ESTIMATED **Results First Submit Date:** 2014-01-01 **Results First Submit QC Date:** 2014-01-01 #### Start Date **Date:** 2008-03 **Status Verified Date:** 2017-11 #### Study First Post Date **Date:** 2008-03-13 **Type:** ESTIMATED **Study First Submit Date:** 2008-03-06 **Study First Submit QC Date:** 2008-03-12 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** University of California, Davis **Class:** OTHER **Name:** University of Alabama at Birmingham **Class:** OTHER **Name:** Medical College of Wisconsin #### Lead Sponsor **Class:** OTHER **Name:** University of Missouri-Columbia #### Responsible Party **Investigator Affiliation:** University of Missouri-Columbia **Investigator Full Name:** Brett Crist **Investigator Title:** Assistant Professor, Co-Director of Trauma Services, Co-Director Trauma Fellowship, Department of Orthopaedic Surgery **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this research is to study the efficacy and cost effectiveness of the VAC device in comparison to traditional gauze wound dressing in pelvic, acetabular and hip fractures, specifically to see if there is a reduction in the incidence of post operative surgical wound drainage, infections, and hospital stay. **Detailed Description:** Soft tissue injuries are commonly associated with pelvic and acetabular injuries and additional tissue injury occurs during surgery. Post operative wound drainage, infections and prolonged hospital stay are a common problem during postoperative care. Traditional treatment is dressing of the surgical wound with different conventional dressings. Use of negative pressure wound therapy has been shown to be beneficial in significantly decreasing wound drainage. Stannard et al. reported the results of randomizing 44 patients with lower extremity fractures (including 4 pilon fractures) into either receiving standard post operative dressing versus NPWT (negative pressure wound therapy). His results showed no difference in infection rate or wound breakdown, but did show a significant difference in the drainage time. The NPWT group stopped draining 3 days earlier than the standard dressing group. The use of NPWT has greatly increased over the years and has been an important adjunct to wound management. These results and anecdotal clinical experience with the use of NPWT (wound VAC) has led us to develop our research question; Does the use of incisional VAC following pelvic \&/or acetabular surgery decrease wound complications. The VAC (KCI USA) device is relatively new device that utilizes negative pressure as a treatment modality for soft tissue injuries following high velocity injuries. VAC device exerts intermittent or constant negative pressure and removes excess fluid from the interstitial space and increases perfusion through vessels. Previous VAC studies showed decreased bacterial load after applying VAC device to the infected wounds. There have been no randomized studies to prove the cost effectiveness and efficacy of VAC device in reducing wound drainage, infections, and prolonged hospital stays in comparison to traditional gauze dressing wound management during post operative management of pelvic and acetabular fractures. In examining the incidence of wound complications/infections, we can determine if the incisional VAC decreases the need for additional intervention and if there are any patient related factors (i.e. obesity) related to increased risk of wound complications. ### Conditions Module **Conditions:** - Pelvic Fractures - Acetabular Fractures - Hip Fractures **Keywords:** - Fractures - Pelvic bones - Acetabulum - Hip bones ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 115 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** will have the VAC device used for post-operative management of acetabular fractures and pelvic fractures. **Intervention Names:** - Device: VAC device **Label:** VAC Device placement **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** will receive current traditional surgical wound management with daily dressing changes in post operative management of acetabular fractures and pelvic fractures. **Intervention Names:** - Other: Gauze dressing **Label:** Gauze dressing **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - VAC Device placement **Description:** Vacuum Assisted Closure (VAC) device for surgical incision **Name:** VAC device **Other Names:** - Wound Vac - Negative pressure wound therapy (NPWT) - Incisional Vac **Type:** DEVICE #### Intervention 2 **Arm Group Labels:** - Gauze dressing **Description:** Gauze dressing for surgical incision **Name:** Gauze dressing **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Measure:** Number of Participants With Wound Infections **Time Frame:** Until wound healed, up to 1 year ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * 18 years or older * Scheduled for surgical repair of pelvic and/or acetabular fracture * Subject/guardian able to provide informed consent Exclusion Criteria: * Less than 18 years of age * Subject/guardian unable to provide informed consent **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Columbia **Country:** United States **Facility:** University of Missouri **State:** Missouri **Zip:** 65212 #### Overall Officials **Official 1:** **Affiliation:** University of Missouri-Columbia **Name:** Brett D Crist, MD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014947 - Term: Wounds and Injuries - ID: D000005264 - Term: Femoral Fractures - ID: D000025981 - Term: Hip 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: M9696 - Name: Hip Fractures - Relevance: HIGH - As Found: Hip Fracture - ID: M17685 - Name: Wounds and Injuries - Relevance: LOW - As Found: Unknown - ID: M8402 - Name: Femoral Fractures - Relevance: LOW - As Found: Unknown - ID: M23105 - Name: Hip 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: D000006620 - Term: Hip Fractures ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** VAC Device **Description:** Vacuum Assisted Closure (VAC) device Wound Vac: Vacuum Assisted Closure (VAC) device for surgical incision **ID:** EG000 **Other Num Affected:** 3 **Other Num at Risk:** 49 **Serious Number Affected:** 15 **Serious Number At Risk:** 49 **Title:** VAC Device **Group ID:** EG001 **Title:** Gauze Dressing **Description:** Gauze Dressing Gauze dressing: Gauze dressing for surgical incision **ID:** EG001 **Other Num Affected:** 5 **Other Num at Risk:** 42 **Serious Number Affected:** 9 **Serious Number At Risk:** 42 **Title:** Gauze Dressing **Frequency Threshold:** 0 #### Other Events **Term:** urinary tract infection **Organ System:** Renal and urinary disorders **Source Vocabulary:** **Term:** ileus **Organ System:** Gastrointestinal disorders **Source Vocabulary:** **Term:** Regional Pain Syndrome **Organ System:** Nervous system disorders **Source Vocabulary:** **Term:** pneumomediastinum **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** **Term:** hematoma **Organ System:** Vascular disorders **Source Vocabulary:** **Term:** malignancy, unrelated **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** **Term:** fall, no injury **Organ System:** General disorders **Source Vocabulary:** #### Serious Events **Term:** infection **Organ System:** Infections and infestations ##### Stats **Group ID:** EG000 **Num Affected:** 10 **Num At Risk:** 49 **Group ID:** EG001 **Num Affected:** 4 **Num At Risk:** 42 **Term:** Perforated colon **Organ System:** Gastrointestinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 49 **Group ID:** EG001 **Num At Risk:** 42 **Term:** Pneumonia **Organ System:** Respiratory, thoracic and mediastinal disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 49 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 42 **Term:** CVA **Organ System:** Vascular disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 49 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 42 **Term:** implant failure **Organ System:** Surgical and medical procedures ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 49 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 42 **Term:** prescription overdose **Organ System:** General disorders ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 49 **Group ID:** EG001 **Num At Risk:** 42 **Term:** DVT **Organ System:** Vascular disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 49 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 42 **Term:** PE **Organ System:** Vascular disorders ##### Stats **Group ID:** EG000 **Num At Risk:** 49 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 42 ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 55 **Group ID:** BG001 **Value:** 60 **Group ID:** BG002 **Value:** 115 **Units:** Participants ### Group **ID:** BG000 **Title:** VAC Device **Description:** Vacuum Assisted Closure (VAC) device Wound Vac: Vacuum Assisted Closure (VAC) device for surgical incision ### Group **ID:** BG001 **Title:** Gauze Dressing **Description:** Gauze Dressing Gauze dressing: Gauze dressing for surgical incision ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 19.6 **Value:** 47.2 #### Measurement **Group ID:** BG001 **Spread:** 20.1 **Value:** 48.3 #### Measurement **Group ID:** BG002 **Spread:** 19.9 **Value:** 47.6 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 12 #### Measurement **Group ID:** BG001 **Value:** 19 #### Measurement **Group ID:** BG002 **Value:** 31 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 43 #### Measurement **Group ID:** BG001 **Value:** 41 #### Measurement **Group ID:** BG002 **Value:** 84 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 55 #### Measurement **Group ID:** BG001 **Value:** 60 #### Measurement **Group ID:** BG002 **Value:** 115 **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:** 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:** University of Missouri, Department of Orthopaedics **Phone:** 573-882-6562 **Title:** Brett Crist, MD ## 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 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 2 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** Until wound healed, up to 1 year **Title:** Number of Participants With Wound Infections **Type:** PRIMARY **Unit of Measure:** participants ##### Group **Description:** Vacuum Assisted Closure (VAC) device Wound Vac: Vacuum Assisted Closure (VAC) device for surgical incision **ID:** OG000 **Title:** VAC Device ##### Group **Description:** Gauze Dressing Gauze dressing: Gauze dressing for surgical incision **ID:** OG001 **Title:** Gauze Dressing ### Participant Flow Module #### Group **Description:** Vacuum Assisted Closure (VAC) device Wound Vac: Vacuum Assisted Closure (VAC) device for surgical incision **ID:** FG000 **Title:** VAC Device #### Group **Description:** Gauze Dressing Gauze dressing: Gauze dressing for surgical incision **ID:** FG001 **Title:** Gauze Dressing #### Period **Title:** Overall Study ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 55 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 60 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 49 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 42 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 6 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 18 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT02049879 **Brief Title:** Daily Activity After Corticosteroids Injection Among Knee Osteoarthritis Patients **Official Title:** Quantification of Daily Physical Activity After an Intra-articular Corticosteroid Injection in Patients With Knee Osteoarthritis #### Organization Study ID Info **ID:** INF-2010 #### Organization **Class:** OTHER **Full Name:** Laval University ### Status Module #### Completion Date **Date:** 2012-08 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2014-03-03 **Type:** ESTIMATED **Last Update Submit Date:** 2014-02-28 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2012-08 **Type:** ACTUAL #### Start Date **Date:** 2008-11 **Status Verified Date:** 2014-02 #### Study First Post Date **Date:** 2014-01-30 **Type:** ESTIMATED **Study First Submit Date:** 2014-01-28 **Study First Submit QC Date:** 2014-01-29 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Laval University #### Responsible Party **Investigator Affiliation:** Laval University **Investigator Full Name:** Philippe Corbeil **Investigator Title:** Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The objective of this study was to objectively measure daily physical activity and spatiotemporal gait pattern, as well as improvements in self reported symptoms and quality of life, before and six weeks after an intra-articular corticosteroids injection in patients suffering from knee osteoarthritis. Fourteen patients with unilateral knee osteoarthritis were recruited. The intra-articular corticosteroid injection was given at the end of the second week. Physical activity was objectively measured by an accelerometer worn by the participants for eight weeks. In addition, the Western Ontario and McMaster Universities Arthritis Index (WOMAC), the Medical Outcome Study Short Form-36 (MOS-SF36) and gait trials were completed every two weeks to assess symptoms, quality of life and spatiotemporal parameters of gait. ### Conditions Module **Conditions:** - Knee Osteoarthritis ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 14 **Type:** ACTUAL **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Corticosteroids injection **Intervention Names:** - Drug: Corticosteroids injection **Label:** Injection **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Injection **Description:** A solution of triamcinolone 40 mg (Kenalog) mixed with 3 cc of 2% xylocaine without epinephrine. **Name:** Corticosteroids injection **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Change in Knee pain **Time Frame:** Change from Baseline at 2, 4 and 6 weeks **Measure:** Change in physical activity **Time Frame:** Change from baseline at 2, 4 and 6 weeks #### Secondary Outcomes **Description:** Assessment with WOMAC and MOS-SF36 questionnaires **Measure:** Change in function and quality of life **Time Frame:** Change from baseline at 2, 4 and 6 weeks **Description:** Gait symmetry was verified with regards to the swing and stance phase durations and step length **Measure:** Change in Spatiotemporal gait parameters **Time Frame:** Change from Baseline at 2, 4 and 6 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * 50 years old or more * Medial knee osteoarthritis (Kellgren \& Lawrence Grade I to III) Exclusion Criteria: * Intra-articular injection during the last six months * Isolated femoro-patellar arthritis * Rheumatoid arthritis * Knee instability * Spinal stenosis * Lower limb fracture over the last year * Lower limb surgery in the last three months **Minimum Age:** 50 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Quebec **Country:** Canada **Facility:** Pavillon de l'Éducation Physique et des Sports - Université Laval **Zip:** G1V 0A6 #### Overall Officials **Official 1:** **Affiliation:** Laval University **Name:** Philippe Corbeil, PhD **Role:** STUDY_DIRECTOR **Official 2:** **Affiliation:** Laval University **Name:** Yoann Dessery, MSc **Role:** PRINCIPAL_INVESTIGATOR **Official 3:** **Affiliation:** Laval University **Name:** Étienne Belzile, MD **Role:** STUDY_DIRECTOR ### References Module #### References **Citation:** Dessery Y, Belzile EL, Turmel S, Dore J, Diallo B, Corbeil P. Modulation of physical activity to optimize pain sensation following an intra-articular corticosteroid injection in patients with knee osteoarthritis. ScientificWorldJournal. 2014;2014:209165. doi: 10.1155/2014/209165. Epub 2014 Nov 16. **PMID:** 25478585 ## 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: 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: Knee Osteoarthritis - 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 - Browse Branches - Abbrev: AnArAg - Name: Anti-Arrhythmia Agents - Abbrev: ChanBlk - Name: Channel Blockers - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: PhSol - Name: Pharmaceutical Solutions - Abbrev: Infl - Name: Anti-Inflammatory Agents - Abbrev: VaCoAg - Name: Vasoconstrictor Agents - Abbrev: Resp - Name: Respiratory System Agents ### Intervention Browse Module - Browse Leaves - ID: M11014 - Name: Lidocaine - Relevance: LOW - As Found: Unknown - ID: M21860 - Name: Pharmaceutical Solutions - Relevance: LOW - As Found: Unknown - ID: M16975 - Name: Triamcinolone Acetonide - Relevance: LOW - As Found: Unknown - ID: M16974 - Name: Triamcinolone - Relevance: LOW - As Found: Unknown - ID: M237966 - Name: Triamcinolone hexacetonide - Relevance: LOW - As Found: Unknown - ID: M209573 - Name: Triamcinolone diacetate - Relevance: LOW - As Found: Unknown - ID: M7992 - Name: Epinephrine - Relevance: LOW - As Found: Unknown - ID: M30371 - Name: Racepinephrine - Relevance: LOW - As Found: Unknown - ID: M211043 - Name: Epinephryl borate - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02420379 **Brief Title:** Safety Study of Eteplirsen to Treat Early Stage Duchenne Muscular Dystrophy **Official Title:** An Open-Label, Multi-Center Study to Evaluate the Safety, Efficacy and Tolerability of Eteplirsen in Early Stage Duchenne Muscular Dystrophy #### Organization Study ID Info **ID:** 4658-203 #### Organization **Class:** INDUSTRY **Full Name:** Sarepta Therapeutics, Inc. ### Status Module #### Completion Date **Date:** 2018-12-17 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-01-25 **Type:** ACTUAL **Last Update Submit Date:** 2020-12-31 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2018-12-17 **Type:** ACTUAL #### Results First Post Date **Date:** 2020-07-22 **Type:** ACTUAL **Results First Submit Date:** 2020-07-06 **Results First Submit QC Date:** 2020-07-06 #### Start Date **Date:** 2015-06-30 **Type:** ACTUAL **Status Verified Date:** 2020-12 #### Study First Post Date **Date:** 2015-04-17 **Type:** ESTIMATED **Study First Submit Date:** 2015-04-10 **Study First Submit QC Date:** 2015-04-14 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Sarepta Therapeutics, 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:** This is an open-label study to assess the safety, tolerability, efficacy and pharmacokinetics of eteplirsen in patients with early stage Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping. **Detailed Description:** Safety, including adverse event monitoring and routine laboratory assessments, will be followed on an ongoing basis for all patients. Clinical efficacy, including functional tests and MRI, will be assessed at regularly scheduled study visits. Patients will undergo one baseline and one follow-up muscle biopsy. Population and serial PK will be collected. ### Conditions Module **Conditions:** - Duchenne Muscular Dystrophy (DMD) **Keywords:** - DMD - Duchenne muscular dystrophy - eteplirsen - Dystrophin - exon 51 ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 33 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Approximately 20 patients will receive weekly infusions of eteplirsen 30 mg/kg . **Intervention Names:** - Drug: eteplirsen **Label:** Open-Label **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Approximately 20 patients with DMD not amenable to exon 51 skipping will be observed for 96 weeks. **Label:** Control Group **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Open-Label **Description:** Eteplirsen 30 mg/kg will be administered as an IV infusion once a week for 96 weeks. **Name:** eteplirsen **Other Names:** - AVI-4658 - EXONDYS 51® **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Adverse Event (AE) was any untoward medical occurrence in a participant that did not necessarily have a causal relationship with the study drug. Serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; Life-threatening event; required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. Treatment emergent adverse events were events that developed or worsened during the on-treatment period (defined as time from first dose of study drug and up to 28 days after last dose of study drug \[up to 100 weeks\]) that were absent before treatment or that worsened relative to pre-treatment state. AEs included both serious and non-serious adverse events. **Measure:** Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation **Time Frame:** Baseline up to 100 weeks **Description:** Laboratory parameters included hematology, serum chemistry (SC), urinalysis and coagulation. Number of participants with at least one potentially clinically significant abnormal findings were reported as TEAEs. The Investigator determined whether abnormal assessment results were clinically significant or not clinically significant. Clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care. **Measure:** Number of Participants With Potentially Clinically Significant Laboratory Abnormalities Reported as TEAEs **Time Frame:** Baseline up to 100 weeks **Description:** Vital sign parameters included systolic blood pressure (SBP), diastolic blood pressure (DBP), heart rate (HR), and body temperature. Number of participants with at least one potentially clinically significant abnormal vital signs findings were reported as TEAEs. The Investigator determined whether abnormal assessment results were clinically significant or not clinically significant. Clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care. **Measure:** Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs Reported as TEAEs **Time Frame:** Baseline up to 100 weeks **Description:** Physical examinations, full and brief, were performed by the Investigator, a physician Sub-Investigator, or a Nurse Practitioner (if licensed in the state or province to perform physical examinations). Full physical examinations included examination of general appearance; head, ears, eyes, nose, and throat; heart; lungs; chest; abdomen; skin; lymph nodes; and musculoskeletal and neurological systems. Number of participants with at least one abnormal physical examination findings were reported. Abnormality in physical examinations was based on Investigator's discretion. **Measure:** Number of Participants With at Least One Abnormal Physical Examination Finding **Time Frame:** Baseline up to 100 weeks **Description:** Twelve-lead ECGs and Holter ECGs were performed at a consistent time of day throughout the study. Electrocardiograms were performed only after the participant was in the supine position, resting, and quiet for a minimum of 15 minutes. The ECG was manually reviewed and interpreted by medically qualified personnel. Number of participants with at least one abnormalities in ECGs were reported as TEAEs. **Measure:** Number of Participants With Abnormalities in Electrocardiograms (ECGs) Reported as TEAEs **Time Frame:** Baseline up to 96 weeks **Description:** Standard, 2-dimensional ECHOs were performed at a consistent time of day throughout the study. Cardiac function events included cardiomegaly, tachycardia, and dyspnoea. The ECHO was reviewed and interpreted by medically qualified personnel. Number of participants with at least one abnormalities in ECHO were reported as TEAEs. **Measure:** Number of Participants With Abnormalities in Echocardiograms (ECHO) Reported as TEAEs **Time Frame:** Baseline up to 96 weeks #### Secondary Outcomes **Description:** Change from baseline in dystrophin protein levels (in muscle biopsy samples) were determined by Western blot at Week 48 and 96 was reported. For each time point, 2 blocks of tissues were analyzed by Western blot, each with 2 replicates of gels to determine the dystrophin level as compared to a healthy individual (Percent Normal). The block average value from 2 replicate gels was computed. The overall average was calculated as the mean of the block average values. The overall average values were used for all analyses. In case only 1 gel was available for a block, then that value was used as the block average value. **Measure:** Change From Baseline in Dystrophin Protein Levels Quantified by Western Blot at Week 48 and 96 **Time Frame:** Baseline, Week 48 and 96 **Description:** Change from baseline in dystrophin intensity levels (in muscle biopsy samples) was determined by Immunohistochemistry at Week 48 and 96 was reported. **Measure:** Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 48 and 96 **Time Frame:** Baseline, Week 48 and 96 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Male 4-6 years of age. * Diagnosis of DMD, genotypically confirmed. * Stable dose of oral corticosteroids for at least 12 weeks or has not received corticosteroids for at least 12 weeks. * Intact right and left biceps muscles or two alternative upper arm muscle groups. * Parent that is willing to provide consent and comply with study procedures. Exclusion Criteria: * Use of any pharmacologic treatment (other than corticosteroids) within 12 weeks that may have an effect on muscle strength or function (e.g., growth hormone, anabolic steroids). * Previous or current treatment with any other experimental treatments within 12 weeks or participation in any other clinical trial within 6 months. * Major surgery within 3 months prior to the first dose of study drug, or planned surgery during this study which would interfere with the ability to perform study activities. * Presence of other clinically significant illness. **Maximum Age:** 6 Years **Minimum Age:** 4 Years **Sex:** MALE **Standard Ages:** - CHILD ### Contacts Locations Module #### Locations **Location 1:** **City:** Phoenix **Country:** United States **Facility:** Neuromuscular Research Center of Arizona **State:** Arizona **Zip:** 85028 **Location 2:** **City:** Los Angeles **Country:** United States **Facility:** Ronald Reagan UCLA Medical Center **State:** California **Zip:** 90095 **Location 3:** **City:** Sacramento **Country:** United States **Facility:** University of California, Davis Medical Center **State:** California **Zip:** 95817 **Location 4:** **City:** Stanford **Country:** United States **Facility:** Stanford University Medical Center **State:** California **Zip:** 94305 **Location 5:** **City:** Gainesville **Country:** United States **Facility:** University of Florida, Shands Hospital **State:** Florida **Zip:** 32610 **Location 6:** **City:** Atlanta **Country:** United States **Facility:** Rare Disease Research Center **State:** Georgia **Zip:** 30318 **Location 7:** **City:** Atlanta **Country:** United States **Facility:** Children's Hospital of Atlanta **State:** Georgia **Zip:** 30324 **Location 8:** **City:** Iowa City **Country:** United States **Facility:** University of Iowa Children's Hospital **State:** Iowa **Zip:** 52242 **Location 9:** **City:** Saint Louis **Country:** United States **Facility:** St. Louis Children's Hospital **State:** Missouri **Zip:** 63110 **Location 10:** **City:** Columbus **Country:** United States **Facility:** Nationwide Children's Hospital **State:** Ohio **Zip:** 43205 **Location 11:** **City:** Portland **Country:** United States **Facility:** Shriners Hospital for Children **State:** Oregon **Zip:** 97239 **Location 12:** **City:** Philadelphia **Country:** United States **Facility:** Children's Hospital of Philadelphia **State:** Pennsylvania **Zip:** 19104 **Location 13:** **City:** Seattle **Country:** United States **Facility:** Seattle Children's Hospital **State:** Washington **Zip:** 98105 #### Overall Officials **Official 1:** **Affiliation:** Sarepta Therapeutics, Inc. **Name:** Medical Director **Role:** STUDY_DIRECTOR ## Document Section ### Large Document Module #### Large Docs - Date: 2019-02-11 - Filename: SAP_000.pdf - Has ICF: False - Has Protocol: False - Has SAP: True - Label: Statistical Analysis Plan - Size: 913431 - Type Abbrev: SAP - Upload Date: 2020-07-03T08:46 - Date: 2017-06-08 - Filename: Prot_001.pdf - Has ICF: False - Has Protocol: True - Has SAP: False - Label: Study Protocol - Size: 1820998 - Type Abbrev: Prot - Upload Date: 2020-07-03T08:49 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020966 - Term: Muscular Disorders, Atrophic - ID: D000009135 - Term: Muscular Diseases - ID: D000009140 - Term: Musculoskeletal Diseases - ID: D000009468 - Term: Neuromuscular Diseases - ID: D000009422 - Term: Nervous System Diseases - ID: D000030342 - Term: Genetic Diseases, Inborn - ID: D000040181 - Term: Genetic Diseases, X-Linked ### Condition Browse Module - Browse Branches - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M12093 - Name: Muscular Dystrophies - Relevance: HIGH - As Found: Muscular Dystrophy - ID: M22185 - Name: Muscular Dystrophy, Duchenne - Relevance: HIGH - As Found: Duchenne Muscular Dystrophy - ID: M4589 - Name: Atrophy - Relevance: LOW - As Found: Unknown - ID: M12092 - Name: Muscular Diseases - Relevance: LOW - As Found: Unknown - ID: M22697 - Name: Muscular Disorders, Atrophic - Relevance: LOW - As Found: Unknown - ID: M12097 - Name: Musculoskeletal Diseases - Relevance: LOW - As Found: Unknown - ID: M12411 - Name: Neuromuscular Diseases - Relevance: LOW - As Found: Unknown - ID: M23686 - Name: Genetic Diseases, Inborn - Relevance: LOW - As Found: Unknown - ID: M24877 - Name: Genetic Diseases, X-Linked - Relevance: LOW - As Found: Unknown - ID: T3963 - Name: Muscular Dystrophy - Relevance: HIGH - As Found: Muscular Dystrophy - ID: T698 - Name: Becker Muscular Dystrophy - Relevance: HIGH - As Found: Duchenne Muscular Dystrophy - ID: T1945 - Name: Duchenne Muscular Dystrophy - Relevance: HIGH - As Found: Duchenne Muscular Dystrophy ### Condition Browse Module - Meshes - ID: D000009136 - Term: Muscular Dystrophies - ID: D000020388 - Term: Muscular Dystrophy, Duchenne ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Eteplirsen 30 mg/kg **Deaths Num At Risk:** 26 **Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks. **ID:** EG000 **Other Num Affected:** 26 **Other Num at Risk:** 26 **Serious Number Affected:** 4 **Serious Number At Risk:** 26 **Title:** Eteplirsen 30 mg/kg **Group ID:** EG001 **Title:** Control Group (Untreated) (Non-exon 51 Amenable Participants) **Deaths Num At Risk:** 7 **Description:** Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks. **ID:** EG001 **Other Num Affected:** 5 **Other Num at Risk:** 7 **Serious Number At Risk:** 7 **Title:** Control Group (Untreated) (Non-exon 51 Amenable Participants) **Frequency Threshold:** 5 #### Other Events **Term:** Nasopharyngitis **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (17.1) **Term:** Upper respiratory tract infection **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (17.1) **Term:** Ear infection **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (17.1) **Term:** Influenza **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (17.1) **Term:** Otitis media **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (17.1) **Term:** Pharyngitis streptococcal **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (17.1) **Term:** Rhinitis **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (17.1) **Term:** Conjunctivitis **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (17.1) **Term:** Gastroenteritis viral **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (17.1) **Term:** Staphylococcal infection **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (17.1) **Term:** Impetigo **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (17.1) **Term:** Cough **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Rhinorrhoea **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Nasal congestion **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Epistaxis **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Oropharyngeal pain **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Productive cough **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Upper respiratory tract congestion **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Vomiting **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Diarrhoea **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Abdominal pain **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Abdominal pain upper **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Constipation **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Nausea **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Dental caries **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Fall **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (17.1) **Term:** Contusion **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (17.1) **Term:** Procedural pain **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (17.1) **Term:** Skin abrasion **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (17.1) **Term:** Scratch **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (17.1) **Term:** Head injury **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (17.1) **Term:** Sunburn **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (17.1) **Term:** Ligament sprain **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (17.1) **Term:** Pain in extremity **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Arthralgia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Back pain **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Muscle spasms **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Muscular weakness **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Musculoskeletal pain **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Scoliosis **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Rash **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Dermatitis contact **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Keloid scar **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Rash pruritic **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Pyrexia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Catheter site pain **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Catheter site bruise **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Headache **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Migraine **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Ear pain **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Ear and labyrinth disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Cardiac murmur **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA (17.1) **Term:** Aggression **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Hypersensitivity **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Immune system disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Chromaturia **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Decreased appetite **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA (17.1) **Term:** Skin papilloma **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Neoplasms benign, malignant and unspecified (incl cysts and polyps) **Source Vocabulary:** MedDRA (17.1) **Term:** Adenoidectomy **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Surgical and medical procedures **Source Vocabulary:** MedDRA (17.1) **Term:** Myringotomy **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Surgical and medical procedures **Source Vocabulary:** MedDRA (17.1) **Term:** Orchidopexy **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Surgical and medical procedures **Source Vocabulary:** MedDRA (17.1) #### Serious Events **Term:** Foreign body **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (17.1) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 26 **Group ID:** EG001 **Num At Risk:** 7 **Term:** Coxsackie viral infection **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (17.1) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 26 **Group ID:** EG001 **Num At Risk:** 7 **Term:** Influenza **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (17.1) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 26 **Group ID:** EG001 **Num At Risk:** 7 **Term:** Rhinovirus infection **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA (17.1) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 26 **Group ID:** EG001 **Num At Risk:** 7 **Term:** Rhabdomyolysis **Assessment Type:** NON_SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (17.1) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 26 **Group ID:** EG001 **Num At Risk:** 7 **Time Frame:** Baseline up to 100 weeks ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 26 **Group ID:** BG001 **Value:** 7 **Group ID:** BG002 **Value:** 33 **Units:** Participants ### Group **ID:** BG000 **Title:** Eteplirsen 30 mg/kg **Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks. ### Group **ID:** BG001 **Title:** Control Group (Untreated) (Non-exon 51 Amenable Participants) **Description:** Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks. ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 0.82 **Value:** 5.0 #### Measurement **Group ID:** BG001 **Spread:** 1.00 **Value:** 5.0 #### Measurement **Group ID:** BG002 **Spread:** 0.85 **Value:** 5.0 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 26 #### Measurement **Group ID:** BG001 **Value:** 7 #### Measurement **Group ID:** BG002 **Value:** 33 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 5 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 5 **Category Title:** Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 21 #### Measurement **Group ID:** BG001 **Value:** 7 #### Measurement **Group ID:** BG002 **Value:** 28 **Category Title:** Not Hispanic or Latino #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** American Indian or Alaska Native #### Measurement **Group ID:** BG000 **Value:** 2 #### Measurement **Group ID:** BG001 **Value:** 1 #### Measurement **Group ID:** BG002 **Value:** 3 **Category Title:** Asian #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Native Hawaiian or Other Pacific Islander #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 1 **Category Title:** Black or African American #### Measurement **Group ID:** BG000 **Value:** 22 #### Measurement **Group ID:** BG001 **Value:** 6 #### Measurement **Group ID:** BG002 **Value:** 28 **Category Title:** White #### Measurement **Group ID:** BG000 **Value:** 1 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 1 **Category Title:** More than one race #### Measurement **Group ID:** BG000 **Value:** 0 #### Measurement **Group ID:** BG001 **Value:** 0 #### Measurement **Group ID:** BG002 **Value:** 0 **Category Title:** Unknown or Not Reported **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 26 #### Measurement **Group ID:** BG001 **Value:** 7 #### Measurement **Group ID:** BG002 **Value:** 33 **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:** NUMBER **Title:** Region of Enrollment **Unit of Measure:** participants **Population Description:** Full set included all participants who were enrolled in the eteplirsen group and received at least 1 dose of eteplirsen as well as all participants who were enrolled in the untreated group and had at least 1 assessment post-enrollment assessment. ## Results Section - More Information Module ### Certain Agreement **Other Details:** The most restrictive relevant agreement provides that the PI can only publish the study results with the approval of Sponsor. **Restriction Type:** OTHER **Restrictive Agreement:** True ### Point of Contact **Email:** [email protected] **Organization:** Sarepta Therapeutics, Inc. **Phone:** +1-888-727-3782 **Title:** Medical 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 ### Outcome Measure 8 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 26 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 5 **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:** 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 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 3 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 11 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 23 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 4 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 1 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 0 **Title:** #### Outcome Measure 7 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.0896 - **Upper Limit:** - **Value:** 0.102 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 14 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.4863 - **Upper Limit:** - **Value:** 0.321 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 11 **Units:** Participants #### Outcome Measure 8 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.0096 - **Upper Limit:** - **Value:** 0.004 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 14 **Units:** Participants **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.0175 - **Upper Limit:** - **Value:** 0.015 **Title:** **Denomination:** - **Group ID:** OG000 - **Value:** 11 **Units:** Participants ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Adverse Event (AE) was any untoward medical occurrence in a participant that did not necessarily have a causal relationship with the study drug. Serious adverse event (SAE) was an AE resulting in any of the following outcomes: death; Life-threatening event; required or prolonged inpatient hospitalization; persistent or significant disability/incapacity; congenital anomaly. Treatment emergent adverse events were events that developed or worsened during the on-treatment period (defined as time from first dose of study drug and up to 28 days after last dose of study drug \[up to 100 weeks\]) that were absent before treatment or that worsened relative to pre-treatment state. AEs included both serious and non-serious adverse events. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Full set included all participants who were enrolled in the eteplirsen group and received at least 1 dose of eteplirsen as well as all participants who were enrolled in the untreated group and had at least 1 assessment post-enrollment assessment. **Reporting Status:** POSTED **Time Frame:** Baseline up to 100 weeks **Title:** Number of Participants With Treatment Emergent Adverse Events (TEAEs), Serious TEAEs, and TEAEs Leading to Discontinuation **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks. **ID:** OG000 **Title:** Eteplirsen 30 mg/kg ##### Group **Description:** Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks. **ID:** OG001 **Title:** Control Group (Untreated) (Non-exon 51 Amenable Participants) #### Outcome Measure 2 **Description:** Laboratory parameters included hematology, serum chemistry (SC), urinalysis and coagulation. Number of participants with at least one potentially clinically significant abnormal findings were reported as TEAEs. The Investigator determined whether abnormal assessment results were clinically significant or not clinically significant. Clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Full set included all participants who are enrolled in the eteplirsen group and receive at least 1 dose of eteplirsen as well as all participants who are enrolled in the untreated group who have at least 1 assessment post-enrollment assessment. **Reporting Status:** POSTED **Time Frame:** Baseline up to 100 weeks **Title:** Number of Participants With Potentially Clinically Significant Laboratory Abnormalities Reported as TEAEs **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks. **ID:** OG000 **Title:** Eteplirsen 30 mg/kg ##### Group **Description:** Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks. **ID:** OG001 **Title:** Control Group (Untreated) (Non-exon 51 Amenable Participants) #### Outcome Measure 3 **Description:** Vital sign parameters included systolic blood pressure (SBP), diastolic blood pressure (DBP), heart rate (HR), and body temperature. Number of participants with at least one potentially clinically significant abnormal vital signs findings were reported as TEAEs. The Investigator determined whether abnormal assessment results were clinically significant or not clinically significant. Clinical significance was defined as any variation in assessment results that had medical relevance resulting in an alteration in medical care. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Full set included all participants who were enrolled in the eteplirsen group and receive at least 1 dose of eteplirsen as well as all participants who are enrolled in the untreated group who have at least 1 assessment post-enrollment assessment. **Reporting Status:** POSTED **Time Frame:** Baseline up to 100 weeks **Title:** Number of Participants With Potentially Clinically Significant Abnormalities in Vital Signs Reported as TEAEs **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks. **ID:** OG000 **Title:** Eteplirsen 30 mg/kg ##### Group **Description:** Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks. **ID:** OG001 **Title:** Control Group (Untreated) (Non-exon 51 Amenable Participants) #### Outcome Measure 4 **Description:** Physical examinations, full and brief, were performed by the Investigator, a physician Sub-Investigator, or a Nurse Practitioner (if licensed in the state or province to perform physical examinations). Full physical examinations included examination of general appearance; head, ears, eyes, nose, and throat; heart; lungs; chest; abdomen; skin; lymph nodes; and musculoskeletal and neurological systems. Number of participants with at least one abnormal physical examination findings were reported. Abnormality in physical examinations was based on Investigator's discretion. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Full set included all participants who are enrolled in the eteplirsen group and receive at least 1 dose of eteplirsen as well as all participants who are enrolled in the untreated group who have at least 1 assessment postenrollment assessment. **Reporting Status:** POSTED **Time Frame:** Baseline up to 100 weeks **Title:** Number of Participants With at Least One Abnormal Physical Examination Finding **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks. **ID:** OG000 **Title:** Eteplirsen 30 mg/kg ##### Group **Description:** Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks. **ID:** OG001 **Title:** Control Group (Untreated) (Non-exon 51 Amenable Participants) #### Outcome Measure 5 **Description:** Twelve-lead ECGs and Holter ECGs were performed at a consistent time of day throughout the study. Electrocardiograms were performed only after the participant was in the supine position, resting, and quiet for a minimum of 15 minutes. The ECG was manually reviewed and interpreted by medically qualified personnel. Number of participants with at least one abnormalities in ECGs were reported as TEAEs. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Full set included all participants who are enrolled in the eteplirsen group and receive at least 1 dose of eteplirsen as well as all participants who are enrolled in the untreated group who have at 1 assessment post-enrollment assessment. **Reporting Status:** POSTED **Time Frame:** Baseline up to 96 weeks **Title:** Number of Participants With Abnormalities in Electrocardiograms (ECGs) Reported as TEAEs **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks. **ID:** OG000 **Title:** Eteplirsen 30 mg/kg ##### Group **Description:** Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks. **ID:** OG001 **Title:** Control Group (Untreated) (Non-exon 51 Amenable Participants) #### Outcome Measure 6 **Description:** Standard, 2-dimensional ECHOs were performed at a consistent time of day throughout the study. Cardiac function events included cardiomegaly, tachycardia, and dyspnoea. The ECHO was reviewed and interpreted by medically qualified personnel. Number of participants with at least one abnormalities in ECHO were reported as TEAEs. **Parameter Type:** COUNT_OF_PARTICIPANTS **Population Description:** Full set included all participants who are enrolled in the eteplirsen group and receive at least 1 dose of eteplirsen as well as all participants who are enrolled in the untreated group who have at least 1 assessment post-enrollment assessment. **Reporting Status:** POSTED **Time Frame:** Baseline up to 96 weeks **Title:** Number of Participants With Abnormalities in Echocardiograms (ECHO) Reported as TEAEs **Type:** PRIMARY **Unit of Measure:** Participants ##### Group **Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks. **ID:** OG000 **Title:** Eteplirsen 30 mg/kg ##### Group **Description:** Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks. **ID:** OG001 **Title:** Control Group (Untreated) (Non-exon 51 Amenable Participants) #### Outcome Measure 7 **Description:** Change from baseline in dystrophin protein levels (in muscle biopsy samples) were determined by Western blot at Week 48 and 96 was reported. For each time point, 2 blocks of tissues were analyzed by Western blot, each with 2 replicates of gels to determine the dystrophin level as compared to a healthy individual (Percent Normal). The block average value from 2 replicate gels was computed. The overall average was calculated as the mean of the block average values. The overall average values were used for all analyses. In case only 1 gel was available for a block, then that value was used as the block average value. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Muscle Biopsy Set included all participants who received at least 1 dose of eteplirsen and who had data from both baseline (pre-treatment) and Week 48 or 96 (on-treatment) muscle biopsy samples. Data for this outcome was not planned to be collected and analyzed for control group (untreated) (non-exon 51 amenable participants). Here, number of participants analyzed signifies participants who were evaluable at specific time point. **Reporting Status:** POSTED **Time Frame:** Baseline, Week 48 and 96 **Title:** Change From Baseline in Dystrophin Protein Levels Quantified by Western Blot at Week 48 and 96 **Type:** SECONDARY **Unit of Measure:** Percent Normal Dystrophin Protein Level ##### Group **Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks. **ID:** OG000 **Title:** Eteplirsen 30 mg/kg #### Outcome Measure 8 **Description:** Change from baseline in dystrophin intensity levels (in muscle biopsy samples) was determined by Immunohistochemistry at Week 48 and 96 was reported. **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Muscle Biopsy Set included all participants who received at least 1 dose of eteplirsen and who had data from both baseline (pre-treatment) and Week 48 or 96 (on-treatment) muscle biopsy samples. Data for this outcome was not planned to be collected and analyzed for control group (untreated) (non-exon 51 amenable participants). Here, number of participants analyzed signifies participants who were evaluable at specific time point. **Reporting Status:** POSTED **Time Frame:** Baseline, Week 48 and 96 **Title:** Change From Baseline in Dystrophin Intensity Levels Determined by Immunohistochemistry (IHC) at Week 48 and 96 **Type:** SECONDARY **Unit of Measure:** Percent dystrophin positive fibers ##### Group **Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks. **ID:** OG000 **Title:** Eteplirsen 30 mg/kg ### Participant Flow Module #### Group **Description:** Participants with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping received eteplirsen 30 milligram per kilogram (mg/kg) intravenous (IV) infusions, once weekly, for 96 weeks. **ID:** FG000 **Title:** Eteplirsen 30 mg/kg #### Group **Description:** Participants with DMD not amenable to exon 51 skipping were observed for 96 weeks. **ID:** FG001 **Title:** Control Group (Untreated) (Non-exon 51 Amenable Participants) #### Period **Title:** Overall Study ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 3 ##### Withdraw **Type:** Unable to complete due to schooling ###### Reason **Group ID:** FG000 **Number of Subjects:** 0 ###### Reason **Group ID:** FG001 **Number of Subjects:** 1 ##### Withdraw **Type:** Transitioned to commercial drug ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ###### Reason **Group ID:** FG001 **Number of Subjects:** 0 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 26 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 7 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 25 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 3 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 1 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 4 **Pre-Assignment Details:** A total of 33 participants were enrolled in the study treatment. **Recruitment Details:** The study was conducted at 13 sites in the United States. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT05405179 **Brief Title:** Simultaneous Dental Implant in Free Vascularized Bone Flaps for Jaw Reconstruction **Official Title:** Simultaneous Dental Implant in Free Vascularized Bone Flaps for Jaw Reconstruction Using Patient-specific Surgical Guides and Titanium Plates: a Pilot Study #### Organization Study ID Info **ID:** UW21-341 #### Organization **Class:** OTHER **Full Name:** The University of Hong Kong ### Status Module #### Completion Date **Date:** 2024-08-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2022-07-19 **Type:** ACTUAL **Last Update Submit Date:** 2022-07-14 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-02-28 **Type:** ESTIMATED #### Start Date **Date:** 2021-09-01 **Type:** ACTUAL **Status Verified Date:** 2022-05 #### Study First Post Date **Date:** 2022-06-06 **Type:** ACTUAL **Study First Submit Date:** 2022-05-31 **Study First Submit QC Date:** 2022-05-31 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** The University of Hong Kong #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** After jaw resection, free vascularized bone flaps are frequently used for repairing maxillofacial defects. Simultaneous dental implantation is more preferable to secondary implantation as it saves the patients from going through a second surgery after recovering from the first one. In this study, the aim is to preliminarily evaluate the clinical outcome of simultaneous dental implants in vascularized bone flaps in jaw reconstruction using patient-specific surgical plates and 3-in-1-PSSG. The objectives of this study were: 1) to investigate the intraoperative success rate; 2) to measure the accuracy of dental implants position; and 3) to assess implant survival rate and postoperative adverse events. **Detailed Description:** After jaw resection, free vascularized bone flaps are frequently used for repairing maxillofacial defects. Owing to the extensive loss of dentition and soft tissues after the ablative surgery, the use of conventional fixed and removable prostheses is often limited in such cases. Implant-supported fixed prosthesis could be a better alternative which is free-standing and does not rely on tooth or soft tissue support. Dental implants on bone flaps restore the patient's oral function and comfort. Simultaneous dental implantation is more preferable to secondary implantation as it saves the patients from going through a second surgery after recovering from the first one. However, conventional freehand immediate placement of dental implants in jaw reconstructive surgery is technically challenging as the position has to be highly precise to support an aesthetic and functional prosthesis. The current development of computer-aided surgery has been able to plan the inset of a vascularized bone flap in the maxillomandibular complex using digitally planned and printed surgical guides based on the patient's computed tomography (CT) data. The computer-aided surgery also facilitates the placement of dental implants at the optimal position and the modification of the bony reconstruction according to the implant position. Previously, the investigators have designed and fabricated novel patient-specific surgical plates by metal three- dimensional (3D) printing technology for jaw reconstruction, which led to precise reconstructive outcomes. In this study, the investigators propose a novel three-in-one patient-specific surgical guide (3-in-1-PSSG) which includes bone segmentation, surgical plate and implant placement positioning functions, to facilitate simultaneous dental implants in vascularized bone flap jaw reconstruction. It is hypothesized that the 3-in-1-PSSG simplifies the surgical procedure. Together with the patient-specific titanium plate, the clinical outcomes for simultaneous dental implants in jaw reconstruction with vascularized bone flap will be improved. In this study, the aim is to preliminarily evaluate the clinical outcome of simultaneous dental implants in vascularized bone flaps in jaw reconstruction using patient-specific surgical plates and 3-in-1-PSSG. The objectives of this study were: 1) to investigate the intraoperative success rate; 2) to measure the accuracy of dental implants position; and 3) to assess implant survival rate and postoperative adverse events. ### Conditions Module **Conditions:** - Mandibular Reconstruction - Dental Implant - Free Tissue Flaps - Jaw Neoplasms **Keywords:** - Maxillo-mandibular reconstruction - jaw neoplasms - free tissue flaps - dental implant - Patient-specific titanium plates - virtual surgical planning ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### 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:** To perform dental implantation simultaneously in free vascularized flap during jaw reconstruction **Intervention Names:** - Procedure: Simultaneous dental implantation **Label:** Simultaneous dental implantation **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Simultaneous dental implantation **Description:** To perform dental implantation simultaneously in free vascularized bone flaps for jaw reconstruction using patient-specific surgical guides and titanium plates **Name:** Simultaneous dental implantation **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Description:** Success is defined as the uneventful placement of dental implant with adequate stability and correct angulation (Implant insertion torque value greater than 15Ncm and no implant thread exposure) **Measure:** Intraoperative success rate **Time Frame:** Intraoperative #### Secondary Outcomes **Description:** Position of dental implant on CT scan compared to planned position **Measure:** Accuracy of dental implant placements **Time Frame:** One month after surgery **Description:** Implant that is retained in the reconstructed jaw at the time of examination, regardless of the state of the prosthesis. or patient satisfaction. **Measure:** Implant survival **Time Frame:** 6 months after surgery **Description:** OHIP-14 questionnaire **Measure:** Impact of oral health condition on quality of life **Time Frame:** 6 months after surgery ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Age greater than 18 years, of both gender; 2. Diagnosed with maxillofacial benign or malignant pathology and indicated for jaw surgery with immediate dental implants; 3. Titanium plates and dental implants will be used in the surgery; 4. Agree to comply with follow-up procedures; 5. Provision of signed and dated informed consent form. Exclusion Criteria: 1. Patients who are pregnant; 2. Patients who need jaw reconstruction for the management of post-traumatic, congenital or developmental jaw deformities; 3. Patients who have medically compromised conditions and cannot tolerate the surgery; 4. Patients who are unable to have a two-week period prior to surgery, for virtual surgery simulation, patient-specific plates designing and fabrication; 5. Patients whose systemic conditions or diseases that violate the normal bone healing; 6. Patients who are unable to take the preoperative and postoperative CT/CBCT scans. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Wing Shan Choi, PhD **Phone:** +85228590363 **Role:** CONTACT #### Locations **Location 1:** **City:** Hong Kong **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Winnie Wing Shan Choi - **Phone:** 28590266 - **Role:** CONTACT **Country:** Hong Kong **Facility:** Prince Philip Dental Hospital **Status:** RECRUITING ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009369 - Term: Neoplasms - ID: D000012888 - Term: Skull Neoplasms - ID: D000001859 - Term: Bone Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000001847 - Term: Bone Diseases - ID: D000009140 - Term: Musculoskeletal Diseases - ID: D000007571 - Term: Jaw Diseases - ID: D000009057 - Term: Stomatognathic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: BC07 - Name: Mouth and Tooth Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M10603 - Name: Jaw Neoplasms - Relevance: HIGH - As Found: Jaw Neoplasms - ID: M15691 - Name: Skull Neoplasms - Relevance: LOW - As Found: Unknown - ID: M5138 - Name: Bone Neoplasms - Relevance: LOW - As Found: Unknown - ID: M5126 - Name: Bone Diseases - Relevance: LOW - As Found: Unknown - ID: M12097 - Name: Musculoskeletal Diseases - Relevance: LOW - As Found: Unknown - ID: M10601 - Name: Jaw Diseases - Relevance: LOW - As Found: Unknown - ID: M12017 - Name: Stomatognathic Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000007573 - Term: Jaw Neoplasms ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01940679 **Brief Title:** Bioavailability of Encapsulated Omega-3 Fatty Acids **Official Title:** Bioavailability of Encapsulated Omega-3 Fatty Acids #### Organization Study ID Info **ID:** 13-13-H #### Organization **Class:** FED **Full Name:** United States Army Research Institute of Environmental Medicine ### Status Module #### Completion Date **Date:** 2013-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2014-03-04 **Type:** ESTIMATED **Last Update Submit Date:** 2014-03-03 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2013-12 **Type:** ACTUAL #### Start Date **Date:** 2013-09 **Status Verified Date:** 2014-03 #### Study First Post Date **Date:** 2013-09-12 **Type:** ESTIMATED **Study First Submit Date:** 2013-09-09 **Study First Submit QC Date:** 2013-09-09 ### Sponsor Collaborators Module #### Collaborators **Class:** UNKNOWN **Name:** Combat Feeding Directorate at Natick Soldier, Research, Development and Engineering Center #### Lead Sponsor **Class:** FED **Name:** United States Army Research Institute of Environmental Medicine #### Responsible Party **Investigator Affiliation:** United States Army Research Institute of Environmental Medicine **Investigator Full Name:** Tracey Smith **Investigator Title:** Research Dietitian **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Omega-3 fatty acids (n-3s), particularly eicosapentaenoic acid (EPA) and docosahexaenoic acid (DHA), have anti-inflammatory properties and other documented beneficial health effects that could warrant n-3 fortification of combat rations. However, military combat rations must meet rigorous shelf-stability guidelines (e.g.,, 3 years at 80°F or 6 months at 100°F), which is problematic for natural food sources of n-3s (e.g., oil and fish), which degrade when exposed to high temperatures and prolonged shelf-storage. Encapsulation, e.g., coating freeze dried n-3s with gelatin, can prevent this degradation, and Natick Soldier Research, Development and Engineering Center's (NSRDEC) Combat Feeding Directorate used encapsulated n-3 technology to produce n-3 enriched ration items that withstand environmental stressors and meet shelf-life specifications for military rations. However, the bioavailability of the encapsulated n-3s in these rations is unknown, particularly when they are incorporated into high-protein food items and exposed to high temperatures and prolonged storage. In this randomized, cross-over study, civilian and/or military personnel will be asked to consume: 1) a high protein food item enriched with encapsulated n-3s (600 mg) and previously stored for 6 months at 100°F; 2) a low-protein food item enriched with encapsulated n-3s (600 mg) and previously stored for 6 months at 100°F; 3) a high protein food item with encapsulated n-3s (600 mg) that was not subjected to high-temperature and prolonged storage; and, 4) a low-protein food item with encapsulated n-3s (600 mg) that was not subjected to high temperature and prolonged storage. Serial blood sampling to measure acute changes in the circulating fatty acid profile will occur in the hour before and 6 hours after consumption of each ration component. The only known risks, which this study presents to participants, are those associated with venous catheter placement. The results will help military ration developers determine the ration components best suited for n-3 fortification. ### Conditions Module **Conditions:** - Bioavailability of Omega-3s in Military Rations ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER ##### Masking Info **Masking:** TRIPLE **Who Masked:** - PARTICIPANT - INVESTIGATOR - OUTCOMES_ASSESSOR #### Enrollment Info **Count:** 14 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Fresh meat stick w/ encapsulated 600 mg EPA/DHA; product frozen immediately after production. **Intervention Names:** - Dietary Supplement: encapsulated 600 mg EPA/DHA **Label:** Fresh meat stick **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Stored meat stick with encapsulated 600 mg EPA/DHA; stored at 100F for 3 months after production **Intervention Names:** - Dietary Supplement: encapsulated 600 mg EPA/DHA **Label:** Stored meat stick **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Fresh pound cake w/ encapsulated 600 mg EPA/DHA; product frozen immediately after production. **Intervention Names:** - Dietary Supplement: encapsulated 600 mg EPA/DHA **Label:** Fresh pound cake **Type:** EXPERIMENTAL #### Arm Group 4 **Description:** Stored pound cake with encapsulated 600 mg EPA/DHA; stored at 100F for 3 months after production. **Intervention Names:** - Dietary Supplement: encapsulated 600 mg EPA/DHA **Label:** Stored pound cake **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Fresh meat stick - Fresh pound cake - Stored meat stick - Stored pound cake **Description:** encapsulated 600 mg EPA/DHA **Name:** encapsulated 600 mg EPA/DHA **Type:** DIETARY_SUPPLEMENT ### Outcomes Module #### Primary Outcomes **Measure:** Plasma n-3 concentrations **Time Frame:** 15 minute, 2 hrs, 4 hrs and 6 hrs after consumption of n-3 rich food item ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * between 18 and 45 years old * leave status and have approval from your supervisor (for DoD Civilians) Exclusion Criteria: * under the age of 18 or over the age of 45 * bleeding disorder (von Willebrand disease, hemophilia) or taking medications that impair ability to stop bleeding (heparin) * consumed ≥1 serving (equivalent to a deck of cards) of salmon, herring, mackerel, anchovies, sardines, halibut or tuna or omega-3 enriched eggs per week over the previous past month * regularly consume dietary supplements (for example, capsules) containing omega-3 fatty acids **Healthy Volunteers:** True **Maximum Age:** 45 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Natick **Country:** United States **Facility:** U.S. Army Research Institute of Environmental Medicine **State:** Massachusetts **Zip:** 01760 #### Overall Officials **Official 1:** **Affiliation:** U.S. Army Research Institute of Environmental Medicine **Name:** Tracey J Smith, PhD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Intervention Browse Module - Browse Branches - Abbrev: Ot - Name: Other Dietary Supplements - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: T415 - Name: Omega 3 Fatty Acid - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06273579 **Brief Title:** Efficiency of Verbal Intelligent Tutor Instruction in Neurosurgical Simulation **Official Title:** Efficiency of Verbal Intelligent Tutor Instruction in Neurosurgical Simulation: A Randomized Controlled Trial #### Organization Study ID Info **ID:** 2010-270, NEU-09-042-Trial 5 #### Organization **Class:** OTHER **Full Name:** McGill University ### Status Module #### Completion Date **Date:** 2024-12 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-03-05 **Type:** ACTUAL **Last Update Submit Date:** 2024-03-03 **Overall Status:** NOT_YET_RECRUITING #### Primary Completion Date **Date:** 2024-10 **Type:** ESTIMATED #### Start Date **Date:** 2024-03 **Type:** ESTIMATED **Status Verified Date:** 2024-03 #### Study First Post Date **Date:** 2024-02-22 **Type:** ACTUAL **Study First Submit Date:** 2024-02-07 **Study First Submit QC Date:** 2024-02-16 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** McGill University #### Responsible Party **Investigator Affiliation:** McGill University **Investigator Full Name:** Rolando Del Maestro **Investigator Title:** Director of the Neurosurgical Simulation and Artificial Intelligence Learning Centre **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** At the Neurosurgical Simulation and Artificial Intelligence Learning Centre, we seek to provide surgical trainees with innovative technologies that allow them to improve their surgical technical skills in risk-free environments, potentially improving patient operative outcomes. The Intelligent Continuous Expertise Monitoring System (ICEMS), a deep learning application that assesses and trains neurosurgical technical skill and provides continuous intraoperative feedback, is one such technology that may improve surgical education. In this randomized controlled trial, medical students from four Quebec universities will be blinded and randomized to one of three groups (one control and two experimental). Group 1 (control) will be provided with verbal AI tutor feedback based on the ICEMS error detection. Group 2 will be tutored by a human instructor who will receive ICEMS error data and deliver verbal instruction identical to that which the AI tutor delivers. Group 3 will be tutored by a human instructor who will be provided with ICEMS data but may deliver feedback as they feel is appropriate to correct the error. The aim of this study is to determine how the method of delivery of verbal surgical error instruction influences trainee response to instruction and overall surgical performance. Evaluating trainee responses to AI instructor verbal feedback as compared to feedback from human instructors will allow for further development, testing, and optimization of the ICEMS and other AI tutoring systems. **Detailed Description:** Background: Expert surgical technical skill is linked with improved patient outcomes; however, training novices to master these skills remains challenging. The Intelligent Continuous Expertise Monitoring System (ICEMS) is a deep learning application that was developed at the Neurosurgical Simulation and Artificial Intelligence Learning Centre to improve neurosurgical education. The ICEMS assesses and trains bimanual surgical performance by providing continuous feedback via verbal instructions in order to improve trainee performance and mitigate errors. Rationale: A previous randomized controlled trial (RCT) performed at our centre demonstrated that intelligent tutoring is more effective than expert tutoring in a simulated neurosurgical procedure (NCT05168150). Another RCT revealed that medical students' performance in response to ICEMS instruction to decrease bipolar force application was variable (NCT04700384). An agglomerative clustering algorithm classified these variable student responses into 3 groups: 53% successfully obeyed the instruction to correct the error, 36% did not obey the instruction, and 11% over-responded to the instruction. This response variability could significantly limit the utility of the ICEMS and may be attributed to different learning styles, stress levels, or misinterpretation of AI instruction. During this study, expert trainers were not provided with ICEMS error data. Conducting a new RCT in which expert trainers are provided with ICEMS error data will clarify the reason many trainees did not respond to the AI instruction. This report follows the Consolidated Standards of Reporting Trials-Artificial Intelligence (CONSORT-AI) as well as the Machine Learning to Assess Surgical Expertise (MLASE) checklist. Hypotheses: 1. Verbal AI feedback will yield significantly lower success response rates among trainees than identical error feedback provided by human instructors. 2. Trainee performance assessment scores will be significantly higher in the two different human instruction groups assessed. 3. Instruction delivered by the AI tutor will result in increased stress levels and cognitive load as compared to verbal error feedback delivered by human instructors. Primary Objectives: To determine how the method of delivery of surgical error instruction influences: 1. Trainee response to instruction, i.e., whether they corrected, did not correct, or over-corrected the error (data collected by the ICEMS). 2. Trainee overall surgical performance (average expertise score on practice scenarios calculated by the ICEMS, Objective Structured Assessment of Technical Skills (OSATS) score on realistic scenario determined by two blinded expert raters). Secondary Objective: To determine how the method of delivery of surgical error instruction influences trainee affective cognitive responses (self-reported via questionnaires on 5-point Likert scales). Setting: McGill University's Neurosurgical Simulation and Artificial Intelligence Learning Centre. Participants: Students enrolled in their preparatory, first, or second year at one of four Quebec medical schools. Design: A three-arm randomized controlled trial. Intervention: Participants will undergo a training session of approximately 90 minutes on the NeuroVR (CAE Healthcare), a virtual reality (VR) surgical simulator that simulates a subpial brain tumor resection. The NeuroVR has two possible scenarios: a simple practice scenario and a complex realistic scenario. Participants will perform six repetitions of the practice scenario (5 minutes each) followed by the realistic scenario (13 minutes). The ICEMS will continuously assess performance throughout the trial. All participants will receive verbal feedback when the ICEMS detects an error in their performance; however, the method of delivery of this verbal feedback will differ between groups. * Group 1 (control) will receive verbal feedback directly from the ICEMS when an error is detected. * Group 2 (experimental) will receive verbal feedback from an expert instructor delivered in the same words as the ICEMS. * Group 3 (experimental) will receive verbal feedback from an expert instructor delivered in their own words. Verbal feedback will be based on the following six metrics: 1. Tissue injury risk: When a trainee receives feedback on this metric, the healthy brain tissue has been damaged. 2. Bleeding risk: When a trainee receives feedback on this metric, there is bleeding that must be cauterized. 3. Instrument tip separation distance: Refers to the distance between the tip of the ultrasonic aspirator and the tips of the bipolar forceps. When a trainee receives feedback on this metric, their instruments are too far apart. 4. High bipolar force: Refers to the amount of force applied to the tissue by the bipolar forceps. When a trainee receives feedback on this metric, they are applying too much force with the bipolar. 5. Low bipolar force: Refers to the amount of force applied to the tissue by the bipolar forceps. When a trainee receives feedback on this metric, they are not applying enough force with the bipolar. 6. High aspirator force: Refers to the amount of force applied to the tissue by the ultrasonic aspirator. When a trainee receives feedback on this metric, they are applying too much force with the aspirator. These metrics will continuously be evaluated by the ICEMS. The ICEMS will only detect an error on one metric at a time according to a predetermined hierarchy (in the order listed above). For example, if a trainee makes an error on both bleeding risk (2) and high aspirator force (6) at the same time, the ICEMS will only detect an error for bleeding risk since this metric is above high aspirator force in the hierarchy. The first practice scenario will serve as a baseline; thus, no feedback will be given. In the second, third, fourth, and fifth repetitions, feedback will be given according to ICEMS error detection. In the sixth repetition as well as the realistic scenario, no feedback will be provided. Significance: With surgical education approaches beginning to shift towards competency-based frameworks, the implementation of effective AI educational feedback into surgical training becomes crucial for optimizing surgical learning. The results of this RCT will allow for the evaluation and reengineering of the ICEMS and other AI tutoring systems, which may advance the development of not only standardized competency-based surgical education training curricula, but any AI tutor technology dependent on verbal instruction. ### Conditions Module **Conditions:** - Surgical Education **Keywords:** - Virtual Reality - Artificial Intelligence - Surgical Training - Surgical Education - Surgical Simulation - Neurosurgery ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** Randomized Controlled Trial ##### Masking Info **Masking:** DOUBLE **Masking Description:** Double (Participant, Expert Instructor) Both study participants and expert instructors are unaware of the intervention (varying the method of delivery of verbal error instruction). Moreover, when rating performance in the realistic scenario using the OSATS, experts are unaware of which group any given trainee is part of. **Who Masked:** - PARTICIPANT - OUTCOMES_ASSESSOR **Primary Purpose:** HEALTH_SERVICES_RESEARCH #### Enrollment Info **Count:** 78 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 26 participants allocated. During their second, third, fourth, and fifth repetition of the practice subpial brain tumor resection scenario, participants will receive verbal ICEMS feedback when the system detects an error on their performance. **Label:** Tutored by AI **Type:** NO_INTERVENTION #### Arm Group 2 **Description:** 26 participants allocated. During their second, third, fourth, and fifth repetition of the practice subpial brain tumor resection scenario, participants will receive verbal feedback from an expert instructor. The expert instructor will deliver this feedback using the same words as the ICEMS. **Intervention Names:** - Behavioral: Experimental Group - Verbal expert instructor feedback in AI's words **Label:** Tutored by human instructor using AI's words **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** 26 participants allocated. During their second, third, fourth, and fifth repetition of the practice subpial brain tumor resection scenario, participants will receive verbal feedback from an expert instructor. The expert instructor will deliver this feedback using any wording they feel is appropriate to correct the error. **Intervention Names:** - Behavioral: Experimental Group - Verbal expert instructor feedback in expert's own words **Label:** Tutored by human instructor using wording of choice **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Tutored by human instructor using AI's words **Description:** Expert instructor assigned to tutor this group will receive error detection data from the ICEMS. They will also be provided with a list of commands that the ICEMS uses. When the system detects an error in a student's performance for a given metric, the expert instructor must deliver this command in the same words as the ICEMS. **Name:** Experimental Group - Verbal expert instructor feedback in AI's words **Type:** BEHAVIORAL #### Intervention 2 **Arm Group Labels:** - Tutored by human instructor using wording of choice **Description:** Expert instructor assigned to tutor this group will receive error detection data from the ICEMS. When the system detects an error in a student's performance for a given metric, the expert will deliver feedback in their own words. **Name:** Experimental Group - Verbal expert instructor feedback in expert's own words **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** After a trainee receives verbal feedback on a specific metric, the ICEMS will record their response to this instruction, i.e., whether they corrected, did not correct, or over-corrected the error. **Measure:** Response to instruction **Time Frame:** 1 day of study **Description:** The ICEMS will continuously assess the trainee's performance and calculate an average expertise score between -1.00 (novice) and 1.00 (expert). **Measure:** Average Intelligent Continuous Expertise Monitoring System (ICEMS) expertise score **Time Frame:** 1 day of study **Description:** While performing the complex realistic scenario, participants will be video recorded. Two blinded expert raters will evaluate these videos using the OSATS global rating scale between 1 (novice) and 7 (expert). **Measure:** Objective Structured Assessment of Technical Skills (OSATS) global rating **Time Frame:** Approximately 5 months after start of study #### Secondary Outcomes **Description:** Measured using Duffy's Medical Emotions Scale (MES) before, during, and after the intervention (self-reported via questionnaires on 5-point Likert scales). **Measure:** Difference in the strength of emotions elicited **Time Frame:** 1 day of study **Description:** Measured using Leppink's Cognitive Load Index (CLI) after the intervention (self-reported via questionnaire on 5-point Likert scales). **Measure:** Difference in cognitive load **Time Frame:** 1 day of study ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Medical students who are actively enrolled in medical school at any Quebec institution who do not fit the exclusion criteria. * Premedical students who are actively enrolled in medical school at any Quebec institution who do not fit the exclusion criteria. Exclusion Criteria: * Prior use of the NeuroVR (CAE Healthcare) simulator. **Healthy Volunteers:** True **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Rolando F Del Maestro, MD, PhD **Phone:** (519) 708-0346 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Bianca Giglio, BSc **Phone:** (514) 802-1608 **Role:** CONTACT #### Locations **Location 1:** **City:** Montréal **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Rolando F Del Maestro, MD, PhD - **Phone:** (519) 708-0346 - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Bianca Giglio, BSc - **Phone:** (514) 802-1608 - **Role:** CONTACT **Country:** Canada **Facility:** Neurosurgical Simulation and Artificial Intelligence Learning Centre **State:** Quebec **Zip:** H2X 4B3 ### IPD Sharing Statement Module **Access Criteria:** Researchers who wish to access the data must contact the principal investigator of the trial, Dr. Rolando F. Del Maestro. **Description:** Data obtained from primary and secondary outcomes may be shared if other researchers have an interest in this data. **Info Types:** - STUDY_PROTOCOL - SAP - ICF - CSR - ANALYTIC_CODE **IPD Sharing:** YES **Time Frame:** Data will be available for 5 years following the completion of the trial. ### References Module #### References **Citation:** Fazlollahi AM, Bakhaidar M, Alsayegh A, Yilmaz R, Winkler-Schwartz A, Mirchi N, Langleben I, Ledwos N, Sabbagh AJ, Bajunaid K, Harley JM, Del Maestro RF. Effect of Artificial Intelligence Tutoring vs Expert Instruction on Learning Simulated Surgical Skills Among Medical Students: A Randomized Clinical Trial. JAMA Netw Open. 2022 Feb 1;5(2):e2149008. doi: 10.1001/jamanetworkopen.2021.49008. **PMID:** 35191972 **Citation:** Yilmaz R, Fazlollahi AM, Winkler-Schwartz A, Wang A, Makhani HH, Alsayegh A, Bakhaidar M, Tran DH, Santaguida C, Del Maestro RF. Effect of Feedback Modality on Simulated Surgical Skills Learning Using Automated Educational Systems- A Four-Arm Randomized Control Trial. J Surg Educ. 2024 Feb;81(2):275-287. doi: 10.1016/j.jsurg.2023.11.001. Epub 2023 Dec 29. **PMID:** 38160107 **Citation:** Fazlollahi AM, Yilmaz R, Winkler-Schwartz A, Mirchi N, Ledwos N, Bakhaidar M, Alsayegh A, Del Maestro RF. AI in Surgical Curriculum Design and Unintended Outcomes for Technical Competencies in Simulation Training. JAMA Netw Open. 2023 Sep 5;6(9):e2334658. doi: 10.1001/jamanetworkopen.2023.34658. **PMID:** 37725373 **Citation:** Mirchi N, Bissonnette V, Yilmaz R, Ledwos N, Winkler-Schwartz A, Del Maestro RF. The Virtual Operative Assistant: An explainable artificial intelligence tool for simulation-based training in surgery and medicine. PLoS One. 2020 Feb 27;15(2):e0229596. doi: 10.1371/journal.pone.0229596. eCollection 2020. **PMID:** 32106247 **Citation:** Winkler-Schwartz A, Yilmaz R, Mirchi N, Bissonnette V, Ledwos N, Siyar S, Azarnoush H, Karlik B, Del Maestro R. Machine Learning Identification of Surgical and Operative Factors Associated With Surgical Expertise in Virtual Reality Simulation. JAMA Netw Open. 2019 Aug 2;2(8):e198363. doi: 10.1001/jamanetworkopen.2019.8363. **PMID:** 31373651 **Citation:** Sioufi J, Hall B, Antel R, Moussa S, Subasri M, Fakih M, Islam N, Hamdy RC, Chopra S, Harley JM, Keuhl A, Bassilious E, Sherbino J, Bilgic E, Bondok MS, Bondok M, Martel L, Law C, Posel N, Fleiszer D, Daud A, Hauer T, Carr-Pries N, Hali K, Wolfstadt J, Ferguson P, Ghasroddashti A, Sorefan-Mangou F, Del Fernandes R, Williams E, Choi K, Zevin B, Patterson ED, Kirupaharan S, Mann S, Winthrop A, Zevin B, Bondok M, Ghanmi N, Etherington C, Saddiki Y, Lefebvre I, Berthelot P, Dion PM, Raymond B, Seguin J, Sekhavati P, Islam S, Boet S, Tee T, Pachchigar P, Tarabay B, Yilmaz R, Hamdan NA, Agu C, Almansouri A, Harley J, Del Maestro R, Bondok M, Bondok MS, Nguyen AX, Law C, Nathoo N, Bakshi N, Ahuja N, Damji KF, Grewal K, Azher S, Moreno M, Pekrun R, Wiseman J, Fried GM, Lajoie S, Brydges R, Hadwin A, Sun NZ, Khalil E, Harley JM, Nguyen EL, Patel P, Muaddi H, Rukavina N, Bucur R, Shwaartz C, Islam N, Moussa S, Subasri M, Fakih M, Hamdy RC, Wong E, Tewari A, Brydges R, Louridas M, Balaji S, Patel P, Muaddi H, Gaebe K, Luzzi C, Kay A, Rukavina N, Selzner M, Reichman T, Shwaartz C, Balaji S, Muaddi H, Shahabinezhad A, Patel P, Rukavina N, Reichman T, Jayaraman S, Shwaartz C, Nashed J, Ramelli L, Kolasky O, Dickenson T, Dullege M, Kang A, Winthrop A, Mann S, Lau D, Henkelman E, Jacob J, Watson I, Haji F, McEwen CC, Jaffer I, Sibbald M, Blouin V, Benard F, Pelletier F, Abdo S, Meloche-Dumas L, Kapralos B, Dubrowski A, Patocskai E, Pachchigar P, Agu C, Yilmaz R, Tee T, Maestro RD, Adedipe I, Stephens C, Ghebretatios M, Laplante S, Patel P, Balaji S, Muaddi H, Rukavina N, Shwaartz C, Brodovsky M, Lai C, Behzadi A, Blair G, Almansouri A, Hamdan NA, Yilmaz R, Tee T, Pachchigar P, Eskandari M, Agu C, Giglio B, Balasubramaniam N, Bierbrier J, Collins DL, Gueziri HE, Del Maestro RF, Koonar E, Ramazani F, Hart R, Henley J, Roberts S, Chandarana S, Matthews W, Schrag C, Matthews J, Mackenzie D, Cutting C, Lui J, Delisle E, Cordoba T, Cordoba C, Giglio B, Lacroix A, Cairns J, Alsayegh A, Alhantoobi M, Balasubramaniam N, Safih W, Hamel M, Del Maestro R, Francis G, Moise A, Omar Y, Hathi K, Mavedatnia D, Grose E, Philips T, Schneider C, Corbin D, Lesage F, Pellerin M, Ben-Ali W, Tamani Z, Joly-Chevrier M, Benard F, Meloche-Dumas L, Laflamme L, Boulva K, Younan R, Dubrowski A, Patocskai E, Sticca G, Petruccelli J, Dorion D, Osman Y, Benard F, Habti M, Meloche-Dumas L, Duranleau X, Boulva K, Kaviani A, Younan R, Dubrowski A, Vessella K, Patocskai E, Valji R, Turner S, Lam T, Mobilio MH, Hirsh J, Lising D, Cil T, Marcon E, Moulton CA, D'Souza A, Milazzo T, Datta S, Valiquette C, Avery E, Voineskos S, Musgrave M, Wanzel K, Schneidman J, Armstrong N, Gerardis G, Silver J, Azzam MA, Fisher R, Banks I, Young M, Nguyen LH, Skakum M, Hancock BJ, Min SL, Youssef F, Keijzer R, Morris M, Shawyer A, Retrosi G. C-CASE 2023: Promoting Excellence in Surgical Education: Canadian Conference for the Advancement of Surgical Education, Oct. 12-13, 2023, Montreal, Quebec. Can J Surg. 2023 Dec 8;66(6 Suppl 2):S137-S150. doi: 10.1503/cjs.014523. Print 2023 Nov-Dec. No abstract available. **PMID:** 38065582 ## Derived Section ### Intervention Browse Module - Browse Branches - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M11767 - Name: Metronidazole - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01886079 **Brief Title:** The Effects of Dexmedetomidine on Postoperative Renal Function in Valvular Heart Surgery #### Organization Study ID Info **ID:** 4-2013-0194 #### Organization **Class:** OTHER **Full Name:** Yonsei University ### Status Module #### Completion Date **Date:** 2015-05 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2013-06-25 **Type:** ESTIMATED **Last Update Submit Date:** 2013-06-20 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2015-05 **Type:** ESTIMATED #### Start Date **Date:** 2013-05 **Status Verified Date:** 2013-06 #### Study First Post Date **Date:** 2013-06-25 **Type:** ESTIMATED **Study First Submit Date:** 2013-06-18 **Study First Submit QC Date:** 2013-06-20 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Yonsei University #### Responsible Party **Type:** SPONSOR ### Description Module **Brief Summary:** The purpose of study is to compare the incidence of acute kidney injury in patients receiving perioperative dexmedetomidine or placebo undergoing valvular heart surgery. ### Conditions Module **Conditions:** - Acute Kidney Injury ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** TRIPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - INVESTIGATOR **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 200 **Type:** ESTIMATED **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Drug: dexmedetomidine **Label:** Dexmedetomidine group **Type:** EXPERIMENTAL #### Arm Group 2 **Intervention Names:** - Drug: Saline **Label:** Saline group **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Dexmedetomidine group **Description:** 0.4 mcg/kg/h, IV, The infusion of study drug is started after anesthesia induction and continued until 24 hours after surgery **Name:** dexmedetomidine **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Saline group **Description:** 0.4 mcg/kg/h, IV **Name:** Saline **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** BUN/Creatinine, cystatin C, eGFR, urine output Incidence of acute kidney injury (based on the AKIN criteria) **Measure:** Comparison of postoperative renal function in patients with or without dexmedetomidine undergoing valvular heart surgery: **Time Frame:** 24 hours after surgery. ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * adult patients over the age of 20 scheduled for valvular heart surgery Exclusion Criteria: * Left ventricular-ejection fraction \< 30% * Preexisting congestive heart failure * Severe coronary artery disease * Hemodynamically unstable arrhythmia * Cardiogenic shock during perioperative period * Ventricular assist device * Severe renal dysfunction (eGFR \< 15ml/min per 1.73m2) **Maximum Age:** 90 Years **Minimum Age:** 20 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Jong Wook Song, MD **Phone:** 82-2-2228-2420 **Role:** CONTACT #### Locations **Location 1:** **City:** Seoul **Country:** Korea, Republic of **Facility:** Department of Anesthesiology and Pain Medicine, Yonsei University College of Medicine **Status:** RECRUITING **Zip:** 120-752 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000051437 - Term: Renal Insufficiency - 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 ### Condition Browse Module - Browse Branches - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M28998 - Name: Acute Kidney Injury - Relevance: HIGH - As Found: Acute Kidney Injury - ID: M26718 - Name: Renal Insufficiency - 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: 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: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000058186 - Term: Acute Kidney Injury ### Intervention Browse Module - Ancestors - ID: D000006993 - Term: Hypnotics and Sedatives - ID: D000002492 - Term: Central Nervous System Depressants - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000018712 - Term: Analgesics, Non-Narcotic - ID: D000000700 - Term: Analgesics - ID: D000018689 - Term: Sensory System Agents - ID: D000018373 - Term: Peripheral Nervous System Agents - ID: D000058647 - Term: Adrenergic alpha-2 Receptor Agonists - ID: D000000316 - Term: Adrenergic alpha-Agonists - ID: D000000322 - Term: Adrenergic Agonists - ID: D000018663 - Term: Adrenergic Agents - ID: D000018377 - Term: Neurotransmitter Agents - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action ### 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: M22662 - Name: Dexmedetomidine - Relevance: HIGH - As Found: Twice daily - ID: M4107 - Name: Anesthetics - Relevance: LOW - As Found: Unknown - ID: M10043 - Name: Hypnotics and Sedatives - Relevance: LOW - As Found: Unknown - ID: M4032 - Name: Analgesics - Relevance: LOW - As Found: Unknown - ID: M20786 - Name: Analgesics, Non-Narcotic - Relevance: LOW - As Found: Unknown - ID: M20746 - Name: Adrenergic Agents - Relevance: LOW - As Found: Unknown - ID: M3668 - Name: Adrenergic alpha-Agonists - Relevance: LOW - As Found: Unknown - ID: M3673 - Name: Adrenergic Agonists - Relevance: LOW - As Found: Unknown - ID: M20504 - Name: Neurotransmitter Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000020927 - Term: Dexmedetomidine ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00751179 **Brief Title:** Rocuronium Plus Sugammadex Versus Succinylcholine Alone in Participants Undergoing Short Surgical Procedures (19.4.319)(P05700 AM2)(COMPLETED) **Official Title:** A Multi-center, Randomized, Parallel Group, Comparative, Active Controlled, Safety Assessor Blinded Trial in Adult Subjects Comparing Rocuronium Plus Sugammadex Versus Succinylcholine Alone in Subjects Undergoing Short Surgical Procedures in Out-patient Surgicenters #### Organization Study ID Info **ID:** P05700 #### Organization **Class:** INDUSTRY **Full Name:** Merck Sharp & Dohme LLC #### Secondary ID Infos **ID:** 19.4.319 ### Status Module #### Completion Date **Date:** 2009-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2015-06-30 **Type:** ESTIMATED **Last Update Submit Date:** 2015-06-03 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2009-12 **Type:** ACTUAL #### Results First Post Date **Date:** 2012-03-20 **Type:** ESTIMATED **Results First Submit Date:** 2012-02-23 **Results First Submit QC Date:** 2012-02-23 #### Start Date **Date:** 2008-11 **Status Verified Date:** 2015-06 #### Study First Post Date **Date:** 2008-09-11 **Type:** ESTIMATED **Study First Submit Date:** 2008-09-10 **Study First Submit QC Date:** 2008-09-10 ### 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:** A multi-center, randomized, parallel group, comparative, active controlled, safety assessor blinded trial in adult subjects comparing rocuronium plus sugammadex versus succinylcholine alone in subjects undergoing short surgical procedures in out-patient surgicenters. The primary objective was to evaluate changes in plasma potassium levels after treatment with rocuronium, sugammadex, or succinylcholine in adult subjects scheduled for short surgical procedures in out-patient surgicenters. ### Conditions Module **Conditions:** - Neuromuscular Blockade ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 161 **Type:** ACTUAL **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Rocuronium - Sugammadex 4.0 mg/kg **Intervention Names:** - Drug: rocuronium - Drug: sugammadex **Label:** Rocuronium - Sugammadex **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Succinylcholine 1.0 mg/kg **Intervention Names:** - Drug: succinylcholine **Label:** Succinylcholine **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Rocuronium - Sugammadex **Description:** A single bolus intubation dose of 0.6 mg/kg rocuronium will be administered following induction of anesthesia and if required, single bolus dose(s) of 0.15 mg/kg rocuronium will be administered to maintain the neuromuscular block. **Name:** rocuronium **Other Names:** - Zemuron® Injection (rocuronium bromide) **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Rocuronium - Sugammadex **Description:** At the end of the surgical procedure at a target depth of neuromuscular blockade of at least 1-2 Post-Tetanic Count (PTC), 4.0 mg/kg of sugammadex will be administered. **Name:** sugammadex **Other Names:** - sugammadex sodium injection - SCH 900916 - Org 25969 - Bridion **Type:** DRUG #### Intervention 3 **Arm Group Labels:** - Succinylcholine **Description:** A single bolus intubation dose of 1.0 mg/kg succinylcholine will be administered following induction of anesthesia and the subject is allowed to recover spontaneously from the neuromuscular blockade. **Name:** succinylcholine **Other Names:** - Quelicin® (succinylcholine chloride injection United States Pharmacopeia [USP]) **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Change from baseline = 5 minutes post dose value - baseline value. Baseline levels were obtained prior to rocuronium or succinylcholine dose. Only data post rocuronium dose are included for "rocuronium - sugammadex" group. The change from baseline interval included most or all of the intubation/surgical period for sugammadex analysis (since sugammadex was administered at the end of the surgical procedure) but not for succinylcholine or rocuronium analyses (since these were administered immediately after the baseline measurement just prior to intubation/commencement of the surgical period). **Measure:** Change From Baseline in Plasma Potassium Levels at 5 Minutes After Treatment With Rocuronium or Succinylcholine **Time Frame:** Baseline and 5 minutes post dose **Description:** Change from baseline = 5 minutes post dose value - baseline value. Baseline levels were obtained prior to rocuronium dose. Only data post sugammadex dose are included for "rocuronium - sugammadex" group. The change from baseline interval included most or all of the intubation/surgical period for sugammadex analysis (since sugammadex was administered at the end of the surgical procedure) but not for succinylcholine or rocuronium analyses (since these were administered immediately after the baseline measurement just prior to intubation/commencement of the surgical period). **Measure:** Change From Baseline in Plasma Potassium Levels at 5 Minutes After Treatment With Sugammadex **Time Frame:** Baseline and 5 minutes post dose #### Secondary Outcomes **Description:** Change from baseline = 2 minutes post dose value - baseline value. Baseline levels were obtained prior to rocuronium or succinylcholine dose. Only data post rocuronium dose are included for "rocuronium - sugammadex" group. The change from baseline interval included most or all of the intubation/surgical period for sugammadex analysis (since sugammadex was administered at the end of the surgical procedure) but not for succinylcholine or rocuronium analyses (since these were administered immediately after the baseline measurement just prior to intubation/commencement of the surgical period). **Measure:** Change From Baseline in Plasma Potassium Levels at 2 Minutes After Treatment With Rocuronium or Succinylcholine **Time Frame:** Baseline and 2 minutes post dose **Description:** Change from baseline = 2 minutes post dose value - baseline value. Baseline levels were obtained prior to rocuronium dose. Only data post sugammadex dose are included for "rocuronium - sugammadex" group. The change from baseline interval included most or all of the intubation/surgical period for sugammadex analysis (since sugammadex was administered at the end of the surgical procedure) but not for succinylcholine or rocuronium analyses (since these were administered immediately after the baseline measurement just prior to intubation/commencement of the surgical period). **Measure:** Change From Baseline in Plasma Potassium Levels at 2 Minutes After Treatment With Sugammadex **Time Frame:** Baseline and 2 minutes post dose **Description:** Change from baseline = 10 minutes post dose value - baseline value. Baseline levels were obtained prior to rocuronium or succinylcholine dose. Only data post rocuronium dose are included for "rocuronium - sugammadex" group. The change from baseline interval included most or all of the intubation/surgical period for sugammadex analysis (since sugammadex was administered at the end of the surgical procedure) but not for succinylcholine or rocuronium analyses (since these were administered immediately after the baseline measurement just prior to intubation/commencement of the surgical period). **Measure:** Change From Baseline in Plasma Potassium Levels at 10 Minutes After Treatment With Rocuronium or Succinylcholine **Time Frame:** Baseline and 10 minutes post dose **Description:** Change from baseline = 10 minutes post dose value - baseline value. Baseline levels were obtained prior to rocuronium dose. Only data post sugammadex dose are included for "rocuronium - sugammadex" group. The change from baseline interval included most or all of the intubation/surgical period for sugammadex analysis (since sugammadex was administered at the end of the surgical procedure) but not for succinylcholine or rocuronium analyses (since these were administered immediately after the baseline measurement just prior to intubation/commencement of the surgical period). **Measure:** Change From Baseline in Plasma Potassium Levels at 10 Minutes After Treatment With Sugammadex **Time Frame:** Baseline and 10 minutes post dose **Description:** Change from baseline = 15 minutes post dose value - baseline value. Baseline levels were obtained prior to rocuronium or succinylcholine dose. Only data post rocuronium dose are included for "rocuronium - sugammadex" group. The change from baseline interval included most or all of the intubation/surgical period for sugammadex analysis (since sugammadex was administered at the end of the surgical procedure) but not for succinylcholine or rocuronium analyses (since these were administered immediately after the baseline measurement just prior to intubation/commencement of the surgical period). **Measure:** Change From Baseline in Plasma Potassium Levels at 15 Minutes After Treatment With Rocuronium or Succinylcholine **Time Frame:** Baseline and 15 minutes post dose **Description:** Change from baseline = 15 minutes post dose value - baseline value. Baseline levels were obtained prior to rocuronium dose. Only data post sugammadex dose are included for "rocuronium - sugammadex" group. The change from baseline interval included most or all of the intubation/surgical period for sugammadex analysis (since sugammadex was administered at the end of the surgical procedure) but not for succinylcholine or rocuronium analyses (since these were administered immediately after the baseline measurement just prior to intubation/commencement of the surgical period). **Measure:** Change From Baseline in Plasma Potassium Levels at 15 Minutes After Treatment With Sugammadex **Time Frame:** Baseline and 15 minutes post dose **Description:** Only AEs which occurred following administration of sugammadex or succinylcholine are included. AEs in the rocuronium - sugammadex group occurring after rocuronium but before sugammadex administration are considered "pretreatment" events and are not included. The AE reporting interval included the entire intubation/surgical period for the succinylcholine group (since succinylcholine was administered just prior to intubation/commencement of surgery) but not for the rocuronium - sugammadex group (since sugammadex was administered at the end of the surgical procedure). **Measure:** Number of Participants With at Least One Adverse Event (AE) in Rocuronium - Sugammadex and Succinylcholine Treatment Groups **Time Frame:** Up to 7 days post dose **Description:** Neuromuscular functioning was monitored by applying repetitive train of four (TOF) electrical stimulations to the ulnar nerve every 15 seconds and assessing twitch response at the adductor pollicis muscle. Nerve stimulation continued until the ratio of the magnitude of the fourth twitch (T4) to first twitch (T1) reached at least 0.9. The greater the T4/T1 ratio the greater the recovery from neuromuscular blockade, with a value of 1.0 representing full recovery. **Measure:** Time to Recovery of the Fourth Twitch/First Twitch (T4/T1) Ratio to 0.9 Following Administration of 4.0 mg/kg of Sugammadex After Neuromuscular Blockade Induced by Rocuronium **Time Frame:** Start of administration of sugammadex to recovery from neuromuscular blockade (Up to approximately 6 minutes) **Description:** Neuromuscular functioning was monitored by applying repetitive train of four (TOF) electrical stimulations to the ulnar nerve every 15 seconds and assessing twitch response at the adductor pollicis muscle. Nerve stimulation continued until recovery of T1 of 90% of baseline and full recovery of neuromuscular function occurred as determined by the anesthesiologist as per routine clinical practice. **Measure:** Time to Recovery of T1 to 90% of Baseline Following Neuromuscular Blockade Induced by Succinylcholine **Time Frame:** Start of administration of succinylcholine to recovery from neuromuscular blockade (Up to approximately 18 minutes) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Male or female subjects \>=18 years of age; * ASA Class 1-3; * Subjects with a Body Mass Index (BMI) of \< 35 kg/m\^2 ; * Subjects scheduled to undergo an elective short procedure with general anesthesia requiring neuromuscular relaxation and endotracheal intubation in outpatient surgicenters; * Subjects scheduled to undergo an elective surgical procedure expected to last 1.5 hours or less (from end of intubation to end of suturing/stapling of skin); * Subjects who are scheduled to undergo an elective surgical procedures that allows access to the arm for TOF-Watch® SX monitoring; * Subjects who have given written informed consent. Exclusion Criteria: * Subjects known to have ischemic heart disease or a history of myocardial infarction; * Subjects in whom a difficult intubation is expected because of anatomical malformations; * Subjects with medical conditions and/or undergoing surgical procedures that are not compatible with the use of the TOF-Watch SX (e.g., injuries to the thumbs/distal forearms, bilateral ulnar nerve damage or subjects with cardiac pacemakers); * Subjects known or suspected to have neuromuscular disorders impairing neuromuscular blockade (e.g., subjects with myasthenia gravis); * Subjects who would require the use of pneumatic tourniquet during the surgical procedure; * Subjects known or suspected to have significant renal dysfunction (e.g., creatinine clearance \< 30 mL per min); * Subjects known or suspected to have significant hepatic dysfunction; * Subjects known or suspected to have a (family) history of malignant hyperthermia; * Subjects known or suspected to be hypersensitive to sugammadex or other cyclodextrins or rocuronium or any of its excipients; * Subjects known or suspected to have an allergy to opiates/opioids, muscle relaxants, or other medications used during general anesthesia; * Subjects for whom a pre-established need for post operative intensive care admission and/or hospital admission is expected; * Subjects for whom an intra operative IV administration of fluids that contain potassium is expected; * Female subjects who are pregnant; * Female subjects who are breast-feeding; * Subjects who have participated in a previous sugammadex trial; * Subjects who have participated in another investigational drug trial within 30 days before entering into clinical trial (CT) 19.4.319 (P05700) unless pre-approved by the sponsor. **Healthy Volunteers:** True **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### References Module #### References **Citation:** Soto R, Jahr JS, Pavlin J, Sabo D, Philip BK, Egan TD, Rowe E, de Bie J, Woo T. Safety and Efficacy of Rocuronium With Sugammadex Reversal Versus Succinylcholine in Outpatient Surgery-A Multicenter, Randomized, Safety Assessor-Blinded Trial. Am J Ther. 2016 Nov/Dec;23(6):e1654-e1662. doi: 10.1097/MJT.0000000000000206. **PMID:** 25768376 **Citation:** Sabo D, Jahr J, Pavlin J, Philip B, Shimode N, Rowe E, Woo T, Soto R. The increases in potassium concentrations are greater with succinylcholine than with rocuronium-sugammadex in outpatient surgery: a randomized, multicentre trial. Can J Anaesth. 2014 May;61(5):423-32. doi: 10.1007/s12630-014-0128-7. Epub 2014 Apr 8. **PMID:** 24710957 ## Derived Section ### Intervention Browse Module - Ancestors - ID: D000000927 - Term: Anticonvulsants - ID: D000003473 - Term: Neuromuscular Nondepolarizing Agents - ID: D000009466 - Term: Neuromuscular Blocking Agents - ID: D000009465 - Term: Neuromuscular Agents - ID: D000018373 - Term: Peripheral Nervous System Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000009467 - Term: Neuromuscular Depolarizing Agents ### Intervention Browse Module - Browse Branches - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: AntiConv - Name: Anticonvulsants - Abbrev: Ot - Name: Other Dietary Supplements ### Intervention Browse Module - Browse Leaves - ID: M4107 - Name: Anesthetics - Relevance: LOW - As Found: Unknown - ID: M5241 - Name: Bromides - Relevance: HIGH - As Found: Half - ID: M1666 - Name: Rocuronium - Relevance: HIGH - As Found: Sub- - ID: M4854 - Name: Benzocaine - Relevance: LOW - As Found: Unknown - ID: M16177 - Name: Succinylcholine - Relevance: HIGH - As Found: Alpelisib - ID: M4246 - Name: Anticonvulsants - Relevance: LOW - As Found: Unknown - ID: M6684 - Name: Neuromuscular Nondepolarizing Agents - Relevance: LOW - As Found: Unknown - ID: M12409 - Name: Neuromuscular Blocking Agents - Relevance: LOW - As Found: Unknown - ID: T433 - Name: Tannic Acid - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000001965 - Term: Bromides - ID: D000077123 - Term: Rocuronium - ID: D000013390 - Term: Succinylcholine ### Misc Info Module #### Removed Countries - Country: Canada - Country: United States - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module **Description:** Includes only AEs post sugammadex or succinylcholine. AE reporting interval included entire intubation/surgical period for succinylcholine group (since succinylcholine was administered just prior to intubation/commencement of surgery) but not for rocuronium - sugammadex group (since sugammadex was administered at the end of the surgical procedure). #### Event Groups **Group ID:** EG000 **Title:** Rocuronium - Sugammadex **Description:** An intubation dose of rocuronium was administered following induction of anesthesia and, if required, maintenance doses were administered to maintain the neuromuscular block. At the end of the surgical procedure sugammadex was administered for reversal of neuromuscular blockade. **ID:** EG000 **Other Num Affected:** 57 **Other Num at Risk:** 66 **Serious Number Affected:** 1 **Serious Number At Risk:** 66 **Title:** Rocuronium - Sugammadex **Group ID:** EG001 **Title:** Succinylcholine **Description:** An intubation dose of succinylcholine was administered following induction of anesthesia and the subject was allowed to recover spontaneously from the neuromuscular blockade. **ID:** EG001 **Other Num Affected:** 67 **Other Num at Risk:** 80 **Serious Number Affected:** 3 **Serious Number At Risk:** 80 **Title:** Succinylcholine **Frequency Threshold:** 5 #### Other Events **Term:** Abdominal pain **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Nausea **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Vomiting **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (12.1) **Term:** Incision site pain **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (12.1) **Term:** Procedural hypotension **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (12.1) **Term:** Procedural nausea **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (12.1) **Term:** Procedural pain **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (12.1) **Term:** Myalgia **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA (12.1) #### Serious Events **Term:** Rectal hemorrhage **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA (12.1) ##### Stats **Group ID:** EG000 **Num At Risk:** 66 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 80 **Num Events:** 1 **Term:** Procedural nausea **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA (12.1) ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 66 **Num Events:** 1 **Group ID:** EG001 **Num At Risk:** 80 **Term:** Medical observation **Organ System:** Investigations **Source Vocabulary:** MedDRA (12.1) ##### Stats **Group ID:** EG000 **Num At Risk:** 66 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 80 **Num Events:** 1 **Term:** Oxygen saturation decreased **Organ System:** Investigations **Source Vocabulary:** MedDRA (12.1) ##### Stats **Group ID:** EG000 **Num At Risk:** 66 **Group ID:** EG001 **Num Affected:** 1 **Num At Risk:** 80 **Num Events:** 1 **Time Frame:** Up to 7 days post dose ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 66 **Group ID:** BG001 **Value:** 80 **Group ID:** BG002 **Value:** 146 **Units:** Participants ### Group **ID:** BG000 **Title:** Rocuronium - Sugammadex **Description:** An intubation dose of rocuronium was administered following induction of anesthesia and, if required, maintenance doses were administered to maintain the neuromuscular block. At the end of the surgical procedure sugammadex was administered for reversal of neuromuscular blockade. ### Group **ID:** BG001 **Title:** Succinylcholine **Description:** An intubation dose of succinylcholine was administered following induction of anesthesia and the subject was allowed to recover spontaneously from the neuromuscular blockade. ### Group **ID:** BG002 **Title:** Total **Description:** Total of all reporting groups ### Measure #### Measurement **Group ID:** BG000 **Spread:** 13 **Value:** 40 #### Measurement **Group ID:** BG001 **Spread:** 14 **Value:** 45 #### Measurement **Group ID:** BG002 **Spread:** 14 **Value:** 43 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 48 #### Measurement **Group ID:** BG001 **Value:** 53 #### Measurement **Group ID:** BG002 **Value:** 101 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 18 #### Measurement **Group ID:** BG001 **Value:** 27 #### Measurement **Group ID:** BG002 **Value:** 45 **Category Title:** Male **Class Title:** **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Description:** Includes only participants who were randomized and treated with sugammadex or succinylcholine **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 2 **Description:** Includes only participants who were randomized and treated with sugammadex or succinylcholine **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement **Other Details:** If a proposed publication contains reference to an invention owned by the Sponsor or to which the Sponsor otherwise has rights, the Sponsor may request a reasonable suspension of the publication in order to be able to file a patent application protecting such invention. **Restriction Type:** OTHER **Restrictive Agreement:** True ### Limitations and Caveats **Description:** The potassium change from baseline interval included most/all of the intubation/surgical period for the sug but not the suc or roc analysis. AE reporting interval included the entire intubation/surgery period for the suc but not the roc-sug group. ### Point of Contact **Email:** [email protected] **Organization:** Merck Sharp & Dohme Corp **Title:** Senior Vice President, Global Clinical Development ## Results Section - Outcome Measures Module ### Outcome Measure 1 #### Analysis **CI Lower Limit:** **CI Number of Sides:** **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Treatment comparison was between the change from baseline in potassium level post-rocuronium dose versus post-succinylcholine dose, using a one way ANOVA model, with treatment as the term in the model. **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** <0.0001 **P-Value Comment:** Significance level was set at 0.05. **Parameter Type:** **Parameter Value:** **Statistical Comment:** **Statistical Method:** ANOVA **Tested Non-Inferiority:** False ### Outcome Measure 2 #### Analysis **CI Lower Limit:** **CI Number of Sides:** **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Treatment comparison was between the change from baseline in potassium level post-rocuronium dose versus post-succinylcholine dose, using a one way ANOVA model, with treatment as the term in the model. **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** <0.0001 **P-Value Comment:** Significance level was set at 0.05. **Parameter Type:** **Parameter Value:** **Statistical Comment:** **Statistical Method:** ANOVA **Tested Non-Inferiority:** False ### Outcome Measure 3 ### Outcome Measure 4 #### Analysis **CI Lower Limit:** **CI Number of Sides:** **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Treatment comparison was between the change from baseline in potassium level post-rocuronium dose versus post-succinylcholine dose, using a one way ANOVA model, with treatment as the term in the model. **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** <0.0001 **P-Value Comment:** Significance level was set at 0.05. **Parameter Type:** **Parameter Value:** **Statistical Comment:** **Statistical Method:** ANOVA **Tested Non-Inferiority:** False ### Outcome Measure 5 ### Outcome Measure 6 #### Analysis **CI Lower Limit:** **CI Number of Sides:** **CI Percentage Value:** **CI Upper Limit:** **CI Upper Limit Comment:** **Dispersion Type:** **Dispersion Value:** **Estimate Comment:** **Group Description:** Treatment comparison was between the change from baseline in potassium level post-rocuronium dose versus post-succinylcholine dose, using a one way ANOVA model, with treatment as the term in the model. **Non-Inferiority Comment:** **Non-Inferiority Type:** SUPERIORITY_OR_OTHER **Other Analysis Description:** **P-Value:** <0.0001 **P-Value Comment:** Significance level was set at 0.05. **Parameter Type:** **Parameter Value:** **Statistical Comment:** **Statistical Method:** ANOVA **Tested Non-Inferiority:** False ### Outcome Measure 7 ### Outcome Measure 8 ### Outcome Measure 9 ### Outcome Measure 10 ### Outcome Measure 11 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.32 - **Upper Limit:** - **Value:** -0.06 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.34 - **Upper Limit:** - **Value:** 0.30 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.30 - **Upper Limit:** - **Value:** -0.09 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.36 - **Upper Limit:** - **Value:** 0.19 **Title:** #### Outcome Measure 3 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.39 - **Upper Limit:** - **Value:** -0.02 **Title:** #### Outcome Measure 4 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.27 - **Upper Limit:** - **Value:** -0.01 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.41 - **Upper Limit:** - **Value:** 0.32 **Title:** #### Outcome Measure 5 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.38 - **Upper Limit:** - **Value:** 0.07 **Title:** #### Outcome Measure 6 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.26 - **Upper Limit:** - **Value:** 0.02 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** 0.42 - **Upper Limit:** - **Value:** 0.33 **Title:** #### Outcome Measure 7 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.39 - **Upper Limit:** - **Value:** 0.07 **Title:** #### Outcome Measure 8 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** 0.42 - **Upper Limit:** - **Value:** 0.02 **Title:** #### Outcome Measure 9 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 60 - **Comment:** - **Group ID:** OG001 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 75 **Title:** #### Outcome Measure 10 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 1.59 - **Spread:** - **Upper Limit:** 2.03 - **Value:** 1.79 **Title:** #### Outcome Measure 11 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 10.06 - **Spread:** - **Upper Limit:** 11.52 - **Value:** 10.76 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Change from baseline = 5 minutes post dose value - baseline value. Baseline levels were obtained prior to rocuronium or succinylcholine dose. Only data post rocuronium dose are included for "rocuronium - sugammadex" group. The change from baseline interval included most or all of the intubation/surgical period for sugammadex analysis (since sugammadex was administered at the end of the surgical procedure) but not for succinylcholine or rocuronium analyses (since these were administered immediately after the baseline measurement just prior to intubation/commencement of the surgical period). **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Participants who received rocuronium or succinylcholine with evaluable (i.e., not missing or hemolyzed) blood samples for potassium measurement at baseline and at the 5 minute post-dose time point. **Reporting Status:** POSTED **Time Frame:** Baseline and 5 minutes post dose **Title:** Change From Baseline in Plasma Potassium Levels at 5 Minutes After Treatment With Rocuronium or Succinylcholine **Type:** PRIMARY **Unit of Measure:** mmol/L ##### Group **Description:** An intubation dose of rocuronium was administered following induction of anesthesia and, if required, maintenance doses were administered to maintain the neuromuscular block. At the end of the surgical procedure sugammadex was administered for reversal of neuromuscular blockade. **ID:** OG000 **Title:** Rocuronium - Sugammadex ##### Group **Description:** An intubation dose of succinylcholine was administered following induction of anesthesia and the subject was allowed to recover spontaneously from the neuromuscular blockade. **ID:** OG001 **Title:** Succinylcholine #### Outcome Measure 2 **Description:** Change from baseline = 2 minutes post dose value - baseline value. Baseline levels were obtained prior to rocuronium or succinylcholine dose. Only data post rocuronium dose are included for "rocuronium - sugammadex" group. The change from baseline interval included most or all of the intubation/surgical period for sugammadex analysis (since sugammadex was administered at the end of the surgical procedure) but not for succinylcholine or rocuronium analyses (since these were administered immediately after the baseline measurement just prior to intubation/commencement of the surgical period). **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Participants who received rocuronium or succinylcholine with evaluable (i.e., not missing or hemolyzed) blood samples for potassium measurement at baseline and at the 2 minute post-dose time point. **Reporting Status:** POSTED **Time Frame:** Baseline and 2 minutes post dose **Title:** Change From Baseline in Plasma Potassium Levels at 2 Minutes After Treatment With Rocuronium or Succinylcholine **Type:** SECONDARY **Unit of Measure:** mmol/L ##### Group **Description:** An intubation dose of rocuronium was administered following induction of anesthesia and, if required, maintenance doses were administered to maintain the neuromuscular block. At the end of the surgical procedure sugammadex was administered for reversal of neuromuscular blockade. **ID:** OG000 **Title:** Rocuronium - Sugammadex ##### Group **Description:** An intubation dose of succinylcholine was administered following induction of anesthesia and the subject was allowed to recover spontaneously from the neuromuscular blockade. **ID:** OG001 **Title:** Succinylcholine #### Outcome Measure 3 **Description:** Change from baseline = 2 minutes post dose value - baseline value. Baseline levels were obtained prior to rocuronium dose. Only data post sugammadex dose are included for "rocuronium - sugammadex" group. The change from baseline interval included most or all of the intubation/surgical period for sugammadex analysis (since sugammadex was administered at the end of the surgical procedure) but not for succinylcholine or rocuronium analyses (since these were administered immediately after the baseline measurement just prior to intubation/commencement of the surgical period). **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Participants who received sugammadex with evaluable (i.e., not missing or hemolyzed) blood samples for potassium measurement at baseline and at the 2 minute post-dose time point. **Reporting Status:** POSTED **Time Frame:** Baseline and 2 minutes post dose **Title:** Change From Baseline in Plasma Potassium Levels at 2 Minutes After Treatment With Sugammadex **Type:** SECONDARY **Unit of Measure:** mmol/L ##### Group **Description:** An intubation dose of rocuronium was administered following induction of anesthesia and, if required, maintenance doses were administered to maintain the neuromuscular block. At the end of the surgical procedure sugammadex was administered for reversal of neuromuscular blockade. **ID:** OG000 **Title:** Rocuronium - Sugammadex #### Outcome Measure 4 **Description:** Change from baseline = 10 minutes post dose value - baseline value. Baseline levels were obtained prior to rocuronium or succinylcholine dose. Only data post rocuronium dose are included for "rocuronium - sugammadex" group. The change from baseline interval included most or all of the intubation/surgical period for sugammadex analysis (since sugammadex was administered at the end of the surgical procedure) but not for succinylcholine or rocuronium analyses (since these were administered immediately after the baseline measurement just prior to intubation/commencement of the surgical period). **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Participants who received rocuronium or succinylcholine with evaluable (i.e., not missing or hemolyzed) blood samples for potassium measurement at baseline and at the 10 minute post-dose time point. **Reporting Status:** POSTED **Time Frame:** Baseline and 10 minutes post dose **Title:** Change From Baseline in Plasma Potassium Levels at 10 Minutes After Treatment With Rocuronium or Succinylcholine **Type:** SECONDARY **Unit of Measure:** mmol/L ##### Group **Description:** An intubation dose of rocuronium was administered following induction of anesthesia and, if required, maintenance doses were administered to maintain the neuromuscular block. At the end of the surgical procedure sugammadex was administered for reversal of neuromuscular blockade. **ID:** OG000 **Title:** Rocuronium - Sugammadex ##### Group **Description:** An intubation dose of succinylcholine was administered following induction of anesthesia and the subject was allowed to recover spontaneously from the neuromuscular blockade. **ID:** OG001 **Title:** Succinylcholine #### Outcome Measure 5 **Description:** Change from baseline = 10 minutes post dose value - baseline value. Baseline levels were obtained prior to rocuronium dose. Only data post sugammadex dose are included for "rocuronium - sugammadex" group. The change from baseline interval included most or all of the intubation/surgical period for sugammadex analysis (since sugammadex was administered at the end of the surgical procedure) but not for succinylcholine or rocuronium analyses (since these were administered immediately after the baseline measurement just prior to intubation/commencement of the surgical period). **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Participants who received sugammadex with evaluable (i.e., not missing or hemolyzed) blood samples for potassium measurement at baseline and at the 10 minute post-dose time point. **Reporting Status:** POSTED **Time Frame:** Baseline and 10 minutes post dose **Title:** Change From Baseline in Plasma Potassium Levels at 10 Minutes After Treatment With Sugammadex **Type:** SECONDARY **Unit of Measure:** mmol/L ##### Group **Description:** An intubation dose of rocuronium was administered following induction of anesthesia and, if required, maintenance doses were administered to maintain the neuromuscular block. At the end of the surgical procedure sugammadex was administered for reversal of neuromuscular blockade. **ID:** OG000 **Title:** Rocuronium - Sugammadex #### Outcome Measure 6 **Description:** Change from baseline = 15 minutes post dose value - baseline value. Baseline levels were obtained prior to rocuronium or succinylcholine dose. Only data post rocuronium dose are included for "rocuronium - sugammadex" group. The change from baseline interval included most or all of the intubation/surgical period for sugammadex analysis (since sugammadex was administered at the end of the surgical procedure) but not for succinylcholine or rocuronium analyses (since these were administered immediately after the baseline measurement just prior to intubation/commencement of the surgical period). **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Participants who received rocuronium or succinylcholine with evaluable (i.e., not missing or hemolyzed) blood samples for potassium measurement at baseline and at the 15 minute post-dose time point. **Reporting Status:** POSTED **Time Frame:** Baseline and 15 minutes post dose **Title:** Change From Baseline in Plasma Potassium Levels at 15 Minutes After Treatment With Rocuronium or Succinylcholine **Type:** SECONDARY **Unit of Measure:** mmol/L ##### Group **Description:** An intubation dose of rocuronium was administered following induction of anesthesia and, if required, maintenance doses were administered to maintain the neuromuscular block. At the end of the surgical procedure sugammadex was administered for reversal of neuromuscular blockade. **ID:** OG000 **Title:** Rocuronium - Sugammadex ##### Group **Description:** An intubation dose of succinylcholine was administered following induction of anesthesia and the subject was allowed to recover spontaneously from the neuromuscular blockade. **ID:** OG001 **Title:** Succinylcholine #### Outcome Measure 7 **Description:** Change from baseline = 15 minutes post dose value - baseline value. Baseline levels were obtained prior to rocuronium dose. Only data post sugammadex dose are included for "rocuronium - sugammadex" group. The change from baseline interval included most or all of the intubation/surgical period for sugammadex analysis (since sugammadex was administered at the end of the surgical procedure) but not for succinylcholine or rocuronium analyses (since these were administered immediately after the baseline measurement just prior to intubation/commencement of the surgical period). **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Participants who received sugammadex with evaluable (i.e., not missing or hemolyzed) blood samples for potassium measurement at baseline and at the 15 minute post-dose time point. **Reporting Status:** POSTED **Time Frame:** Baseline and 15 minutes post dose **Title:** Change From Baseline in Plasma Potassium Levels at 15 Minutes After Treatment With Sugammadex **Type:** SECONDARY **Unit of Measure:** mmol/L ##### Group **Description:** An intubation dose of rocuronium was administered following induction of anesthesia and, if required, maintenance doses were administered to maintain the neuromuscular block. At the end of the surgical procedure sugammadex was administered for reversal of neuromuscular blockade. **ID:** OG000 **Title:** Rocuronium - Sugammadex #### Outcome Measure 8 **Description:** Change from baseline = 5 minutes post dose value - baseline value. Baseline levels were obtained prior to rocuronium dose. Only data post sugammadex dose are included for "rocuronium - sugammadex" group. The change from baseline interval included most or all of the intubation/surgical period for sugammadex analysis (since sugammadex was administered at the end of the surgical procedure) but not for succinylcholine or rocuronium analyses (since these were administered immediately after the baseline measurement just prior to intubation/commencement of the surgical period). **Dispersion Type:** Standard Deviation **Parameter Type:** MEAN **Population Description:** Participants who received sugammadex with evaluable (i.e., not missing or hemolyzed) blood samples for potassium measurement at baseline and at the 5 minute post-dose time point. **Reporting Status:** POSTED **Time Frame:** Baseline and 5 minutes post dose **Title:** Change From Baseline in Plasma Potassium Levels at 5 Minutes After Treatment With Sugammadex **Type:** PRIMARY **Unit of Measure:** mmol/L ##### Group **Description:** An intubation dose of rocuronium was administered following induction of anesthesia and, if required, maintenance doses were administered to maintain the neuromuscular block. At the end of the surgical procedure sugammadex was administered for reversal of neuromuscular blockade. **ID:** OG000 **Title:** Rocuronium - Sugammadex #### Outcome Measure 9 **Description:** Only AEs which occurred following administration of sugammadex or succinylcholine are included. AEs in the rocuronium - sugammadex group occurring after rocuronium but before sugammadex administration are considered "pretreatment" events and are not included. The AE reporting interval included the entire intubation/surgical period for the succinylcholine group (since succinylcholine was administered just prior to intubation/commencement of surgery) but not for the rocuronium - sugammadex group (since sugammadex was administered at the end of the surgical procedure). **Parameter Type:** NUMBER **Population Description:** Participants who received sugammadex or succinylcholine. **Reporting Status:** POSTED **Time Frame:** Up to 7 days post dose **Title:** Number of Participants With at Least One Adverse Event (AE) in Rocuronium - Sugammadex and Succinylcholine Treatment Groups **Type:** SECONDARY **Unit of Measure:** participants ##### Group **Description:** An intubation dose of rocuronium was administered following induction of anesthesia and, if required, maintenance doses were administered to maintain the neuromuscular block. At the end of the surgical procedure sugammadex was administered for reversal of neuromuscular blockade. **ID:** OG000 **Title:** Rocuronium - Sugammadex ##### Group **Description:** An intubation dose of succinylcholine was administered following induction of anesthesia and the subject was allowed to recover spontaneously from the neuromuscular blockade. **ID:** OG001 **Title:** Succinylcholine #### Outcome Measure 10 **Description:** Neuromuscular functioning was monitored by applying repetitive train of four (TOF) electrical stimulations to the ulnar nerve every 15 seconds and assessing twitch response at the adductor pollicis muscle. Nerve stimulation continued until the ratio of the magnitude of the fourth twitch (T4) to first twitch (T1) reached at least 0.9. The greater the T4/T1 ratio the greater the recovery from neuromuscular blockade, with a value of 1.0 representing full recovery. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** GEOMETRIC_MEAN **Population Description:** Participants who received both rocuronium and sugammadex and had evaluable neuromuscular function data determined to be reliable by a central independent adjudication committee. **Reporting Status:** POSTED **Time Frame:** Start of administration of sugammadex to recovery from neuromuscular blockade (Up to approximately 6 minutes) **Title:** Time to Recovery of the Fourth Twitch/First Twitch (T4/T1) Ratio to 0.9 Following Administration of 4.0 mg/kg of Sugammadex After Neuromuscular Blockade Induced by Rocuronium **Type:** SECONDARY **Unit of Measure:** Minutes ##### Group **Description:** An intubation dose of rocuronium was administered following induction of anesthesia and, if required, maintenance doses were administered to maintain the neuromuscular block. At the end of the surgical procedure sugammadex was administered for reversal of neuromuscular blockade. **ID:** OG000 **Title:** Rocuronium - Sugammadex #### Outcome Measure 11 **Description:** Neuromuscular functioning was monitored by applying repetitive train of four (TOF) electrical stimulations to the ulnar nerve every 15 seconds and assessing twitch response at the adductor pollicis muscle. Nerve stimulation continued until recovery of T1 of 90% of baseline and full recovery of neuromuscular function occurred as determined by the anesthesiologist as per routine clinical practice. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** GEOMETRIC_MEAN **Population Description:** Participants who received succinylcholine and had evaluable neuromuscular function data determined to be reliable by a central independent adjudication committee. **Reporting Status:** POSTED **Time Frame:** Start of administration of succinylcholine to recovery from neuromuscular blockade (Up to approximately 18 minutes) **Title:** Time to Recovery of T1 to 90% of Baseline Following Neuromuscular Blockade Induced by Succinylcholine **Type:** SECONDARY **Unit of Measure:** Minutes ##### Group **Description:** An intubation dose of succinylcholine was administered following induction of anesthesia and the subject was allowed to recover spontaneously from the neuromuscular blockade. **ID:** OG000 **Title:** Succinylcholine ### Participant Flow Module #### Group **Description:** An intubation dose of rocuronium (roc) was administered following induction of anesthesia and, if required, maintenance doses were administered to maintain the neuromuscular block. At the end of the surgical procedure sugammadex (sug) was administered for reversal of neuromuscular blockade. **ID:** FG000 **Title:** Rocuronium - Sugammadex #### Group **Description:** An intubation dose of succinylcholine (suc) was administered following induction of anesthesia and the subject was allowed to recover spontaneously from the neuromuscular blockade. **ID:** FG001 **Title:** Succinylcholine #### Period **Title:** Overall Study ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 76 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 85 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 62 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 70 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 14 ###### Achievement **Group ID:** FG001 **Number of Subjects:** 15 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT00158379 **Brief Title:** Taxol Carboplatin and Erythropoetin **Official Title:** Carboplatin With Following Taxol® Therapy Under Additional Application of Epoetin Alfa (ERYPO ®) With Female Patients With Advanced Ovarian Cancer FIGO IA/G3 - IV #### Organization Study ID Info **ID:** 3002000 #### Organization **Class:** OTHER **Full Name:** North Eastern German Society of Gynaecological Oncology ### Status Module #### Completion Date **Date:** 2008-06 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2017-02-06 **Type:** ESTIMATED **Last Update Submit Date:** 2016-12-12 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2008-04 **Type:** ACTUAL #### Results First Post Date **Date:** 2016-11-25 **Type:** ESTIMATED **Results First Submit Date:** 2016-10-04 **Results First Submit QC Date:** 2016-10-04 #### Start Date **Date:** 2003-07 **Status Verified Date:** 2016-12 #### Study First Post Date **Date:** 2005-09-12 **Type:** ESTIMATED **Study First Submit Date:** 2005-09-09 **Study First Submit QC Date:** 2005-09-10 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** North Eastern German Society of Gynaecological Oncology #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Time to progression (physical examination and radiologic imaging ### Conditions Module **Conditions:** - Ovarian Cancer ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 105 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Drug: Paclitaxel **Label:** Paclitaxel **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Paclitaxel **Description:** 4 cycles of Carboplatin AUC 5 every 3 weeks. 12 weekly infusions of 80 mg/m² Taxol® **Name:** Paclitaxel **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Time to progression **Measure:** Progression-free Survival. Progression is Defined According WHO-criteria as Appearance of Any New Lesion or Increase of Existing Lesions by at Least 25% **Time Frame:** every 3 months for up to 3 years #### Secondary Outcomes **Description:** defined as hematological and non-hematological adverse events of grade \>= grade 1 **Measure:** Toxicity **Time Frame:** after every cycle during therapy phase and after every 3 months during follow-up, for up to 3 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * patients with primary ovarian cancer * ECOG- 0-2 * Age \>= 18 * no chemotherapy, radiation or immunotherapy in medical history for ovarian cancer * adequate bone marrow, liver and kidney reserve: leukocytes ≥ 2.0 x 109/l, platelets ≥ 100 x 109/l, bilirubin \<= 2,0 mg%, creatinine \<= 1,5 mg% or creatinine clearance ≥ 60 ml/ min, hemoglobin ≥ 9 g/ dl SGOT, SGPT an AP within 3 fold of the reference laboratory's normal range * written informed consent Exclusion Criteria: * before-existing heart illness, Cardiac infarct within last 6 months * Radiotherapy within 4 weeks for study entry * Patients in pregnancy or breast feeding (in premenopausal women anticonception has to be assured: intrauterine devices, surgical methods of sterilization, or, in hormone insensitive tumors only, oral, subcutaneous or transvaginal hormonal, non-estrogen containing contraceptives) **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Overall Officials **Official 1:** **Affiliation:** Charite University, Berlin, Germany **Name:** Jalid Sehouli **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### See Also Links **Label:** Related Info **URL:** http://www.noggo.de ## 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: D000000972 - Term: Antineoplastic Agents, Phytogenic - ID: D000000970 - Term: Antineoplastic Agents - ID: D000050257 - Term: Tubulin Modulators - ID: D000050256 - Term: Antimitotic Agents - ID: D000050258 - Term: Mitosis Modulators - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Hemat - Name: Hematinics ### Intervention Browse Module - Browse Leaves - ID: M19537 - Name: Paclitaxel - Relevance: HIGH - As Found: Surgery - ID: M231 - Name: Albumin-Bound Paclitaxel - Relevance: LOW - As Found: Unknown - ID: M18650 - Name: Carboplatin - Relevance: LOW - As Found: Unknown - ID: M314 - Name: Epoetin Alfa - Relevance: LOW - As Found: Unknown - ID: M26197 - Name: Tubulin Modulators - Relevance: LOW - As Found: Unknown - ID: M26196 - Name: Antimitotic Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000017239 - Term: Paclitaxel ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Paclitaxel **Description:** Paclitaxel: 4 cycles of Carboplatin AUC 5 every 3 weeks. 12 weekly infusions of 80 mg/m² Taxol® **ID:** EG000 **Other Num Affected:** 105 **Other Num at Risk:** 105 **Serious Number Affected:** 13 **Serious Number At Risk:** 105 **Title:** Paclitaxel **Frequency Threshold:** 0 #### Other Events **Term:** Anemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** Medra #### Serious Events **Term:** Nausea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** Medra ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 105 **Num Events:** 3 **Term:** Death **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** Medra ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 105 **Num Events:** 1 **Term:** Disease progression **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** Medra ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 105 **Num Events:** 1 **Term:** Suspected thrombosis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** Medra ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 105 **Num Events:** 2 **Term:** Ileus **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** Medra ##### Stats **Group ID:** EG000 **Num Affected:** 3 **Num At Risk:** 105 **Num Events:** 3 **Term:** deterioration of general condition **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** Medra ##### Stats **Group ID:** EG000 **Num Affected:** 5 **Num At Risk:** 105 **Num Events:** 5 **Term:** embolism **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** Medra ##### Stats **Group ID:** EG000 **Num Affected:** 2 **Num At Risk:** 105 **Num Events:** 4 ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 105 **Units:** Participants ### Group **ID:** BG000 **Title:** Paclitaxel **Description:** Paclitaxel: 4 cycles of Carboplatin AUC 5 every 3 weeks. 12 weekly infusions of 80 mg/m² Taxol® ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 23.4 **Upper Limit:** 80.3 **Value:** 60.4 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 105 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 0 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 105 **Class Title:** Germany **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** FULL_RANGE **Parameter Type:** MEDIAN **Title:** Age, Continuous **Unit of Measure:** years ### Measure 2 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Gender **Unit of Measure:** Participants ### Measure 3 **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:** Charite Campus Vichow Klinikum **Phone:** +49 30-450564052 **Title:** Prof. Dr. Jalid Sehouli ## Results Section - Outcome Measures Module ### Outcome Measure 1 ### Outcome Measure 2 ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** 25.4 - **Spread:** - **Upper Limit:** 40.0 - **Value:** 28.8 **Title:** #### Outcome Measure 2 **Class:** ##### Category **Measurements:** - **Comment:** - **Group ID:** OG000 - **Lower Limit:** - **Spread:** - **Upper Limit:** - **Value:** 105 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** Time to progression **Dispersion Type:** 95% Confidence Interval **Parameter Type:** MEDIAN **Reporting Status:** POSTED **Time Frame:** every 3 months for up to 3 years **Title:** Progression-free Survival. Progression is Defined According WHO-criteria as Appearance of Any New Lesion or Increase of Existing Lesions by at Least 25% **Type:** PRIMARY **Unit of Measure:** months ##### Group **Description:** Paclitaxel: 4 cycles of Carboplatin AUC 5 every 3 weeks. 12 weekly infusions of 80 mg/m² Taxol® **ID:** OG000 **Title:** Paclitaxel #### Outcome Measure 2 **Description:** defined as hematological and non-hematological adverse events of grade \>= grade 1 **Parameter Type:** NUMBER **Reporting Status:** POSTED **Time Frame:** after every cycle during therapy phase and after every 3 months during follow-up, for up to 3 years **Title:** Toxicity **Type:** SECONDARY **Unit of Measure:** participants ##### Group **Description:** Paclitaxel: 4 cycles of Carboplatin AUC 5 every 3 weeks. 12 weekly infusions of 80 mg/m² Taxol® **ID:** OG000 **Title:** Paclitaxel ### Participant Flow Module #### Group **Description:** Paclitaxel: 4 cycles of Carboplatin AUC 5 every 3 weeks. 12 weekly infusions of 80 mg/m² Taxol® **ID:** FG000 **Title:** Paclitaxel #### Period **Title:** Overall Study ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 105 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 105 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 0 **Recruitment Details:** recruitment period: July 2003 - December 2004 **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT01652079 **Brief Title:** CRLX101 in Combination With Bevacizumab for Recurrent Ovarian/Tubal/Peritoneal Cancer **Official Title:** A Phase II, 2-stage Trial of CRLX101 in Combination With Bevacizumab in Recurrent Platinum-Resistant Ovarian, Tubal and Peritoneal Cancer #### Organization Study ID Info **ID:** 11-485 #### Organization **Class:** OTHER **Full Name:** Massachusetts General Hospital ### Status Module #### Completion Date **Date:** 2018-03 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2018-07-02 **Type:** ACTUAL **Last Update Submit Date:** 2018-06-28 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2018-03 **Type:** ACTUAL #### Start Date **Date:** 2012-04 **Status Verified Date:** 2018-06 #### Study First Post Date **Date:** 2012-07-27 **Type:** ESTIMATED **Study First Submit Date:** 2012-07-25 **Study First Submit QC Date:** 2012-07-25 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Massachusetts General Hospital #### Responsible Party **Investigator Affiliation:** Massachusetts General Hospital **Investigator Full Name:** Richard Thomas Penson **Investigator Title:** Principal Investigator **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** This research study is a Phase II clinical trial. In addition to studying safety, Phase II clinical trials test if the investigational drug is effective and whether the drug works in treating a specific cancer. "Investigational" means that the drug is still being studied and that research doctors are trying to find out more about it-such as the safest dose to use, the side effects it may cause, and if the drug is effective for treating different types of cancer. It also means that the FDA (the U.S. Food and Drug Administration) has not yet approved CRLX101 for your type of cancer. Camptothecin is a chemical extracted from plants that is the basis for the standard FDA-approved chemotherapy drugs irinotecan and topotecan. Camptothecin works by interfering with the way cells divide and multiply. The investigational drug CRLX101 is a formulation of camptothecin and a large molecule (nanoparticle)that appears to allow more of the camptothecin to get into tumors and stay in tumors. The persistence of the CRLX101 in the tumor may increase the probability that the tumor cells will be damaged. CRLX101 has been well tolerated in the laboratory and in participants with different kinds of cancer. Bevacizumab (Avastin) is a VEGF inhibitor which has activity in many kinds of cancer. Bevacizumab has been successfully combined with many chemotherapy partners. It has been hypothesized that the combination of bevacizumab with CRLX101 might have unique clinical activity in combination in the treatment of this disease due to the simultaneous inhibition of distinct steps along the HIF → (CAIX) → VEGF → VEGFR2 pathway. Specifically, it is hypothesized that CRLX101-mediated inhibition of HIF-1α carries with it the potential to interrupt hypoxia and HIF-1α-associated resistance to VEGFR inhibitors. It is hoped that this combination will work to treat your type of cancer. **Detailed Description:** You will receive CRLX101 and bevacizumab through an intravenous (IV) infusion once every 14 days. Each cycle is 28 days. You will continue to receive both drugs until you and/or the research doctor decides it may not be in your best interest to continue. You will receive premedication including decadron, zantac and benadryl to help prevent an allergic reaction and nausea prior to your CRLX101 infusion.You will also receive IV fluid before and after the study drug administration to keep you hydrated. It will be important for you to drink water regularly in between study visits.You will be treated as an outpatient. At every clinic visit, you will undergo the following assessments: Medical history, physical examination, vital signs, performance status, routine blood tests, urine tests, assessment for any new side effects, CT evaluation (every 8 weeks). You will have an end of study visit within 30 days of your last dose. ### Conditions Module **Conditions:** - Ovarian Cancer - Fallopian Tube Cancer - Primary Peritoneal Cancer **Keywords:** - Epithelial - Primary ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 63 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** CRLX101 **Intervention Names:** - Drug: CRLX101 **Label:** Treatment Arm **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Treatment Arm **Description:** q 14 days **Name:** CRLX101 **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Progression free survival at 6 months (PFS6) using RECIST 1.1 **Measure:** Progression Free Survival **Time Frame:** 6 months #### Secondary Outcomes **Description:** Response Rate (CR+PR) using RECIST 1.1 **Measure:** Response Rate **Time Frame:** 2 years **Description:** Assessment of toxicity **Measure:** Assessment of Toxicity **Time Frame:** 2 years **Description:** Analysis of ovarian tumor biopsies and ascites of the presence or absence of CRLX101 or the active drug, camptothecin **Measure:** Analysis of biopsies **Time Frame:** 2 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Histologically or cytologically confirmed epithelial ovarian, tubal or primary peritoneal cancer * Measurable disease * May have received up to 2 prior cytotoxic chemotherapy * Life expectancy of greater than 3 months Exclusion Criteria: * Pregnant or breastfeeding * Prior camptothecin, prior VEFG inhibitors * Gross hematuria * Chemotherapy or radiotherapy within 4 weeks of study entry * uncontrolled HTN * Receiving other study agents * History of allergic reaction to compounds of similar chemical or biologic composition to topotecan or irinotecan * Known brain metastases * History of a different malignancy within the previous 2 years * Intercurrent illness * HIV positive on combination antiretroviral therapy **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Boston **Country:** United States **Facility:** Massachusetts General Hospital **State:** Massachusetts **Zip:** 02114 **Location 2:** **City:** Boston **Country:** United States **Facility:** Beth Israel Deaconess Medical Center **State:** Massachusetts **Zip:** 02115 **Location 3:** **City:** Boston **Country:** United States **Facility:** Brigham and Women's Hospital **State:** Massachusetts **Zip:** 02215 **Location 4:** **City:** Boston **Country:** United States **Facility:** Dana-Farber Cancer Institute **State:** Massachusetts **Zip:** 02215 #### Overall Officials **Official 1:** **Affiliation:** Massachusetts General Hospital **Name:** Richard Penson, MD, MRCP **Role:** PRINCIPAL_INVESTIGATOR ## Annotation Section ### Unposted Annotation #### Event: RELEASE - Date: 2022-12-14 - Date Unknown: Unknown #### Event: RESET - Date: 2023-01-05 - Date Unknown: Unknown #### Event: RELEASE - Date: 2023-01-05 - Date Unknown: Unknown #### Event: RESET - Date: 2023-01-31 - Date Unknown: Unknown #### Event: RELEASE - Date: 2023-03-06 - Date Unknown: Unknown #### Event: RESET - Date: 2023-03-28 - Date Unknown: Unknown #### Event: RELEASE - Date: 2023-06-07 - Date Unknown: Unknown #### Event: RESET - Date: 2023-06-27 - Date Unknown: Unknown ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - 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: D000005184 - Term: Fallopian Tube Diseases - ID: D000000008 - Term: Abdominal Neoplasms - ID: D000004067 - Term: Digestive System Neoplasms - ID: D000004066 - Term: Digestive System Diseases - ID: D000010532 - Term: Peritoneal Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC04 - Name: Neoplasms - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: BC19 - Name: Gland and Hormone Related Diseases - Abbrev: BC06 - Name: Digestive System Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M14850 - Name: Recurrence - Relevance: LOW - As Found: Unknown - ID: M12974 - Name: Ovarian Neoplasms - Relevance: LOW - As Found: Unknown - ID: M1704 - Name: Carcinoma, Ovarian Epithelial - Relevance: LOW - As Found: Unknown - ID: M8328 - Name: Fallopian Tube Neoplasms - Relevance: HIGH - As Found: Fallopian Tube Cancer - ID: M13443 - Name: Peritoneal Neoplasms - Relevance: HIGH - As Found: Peritoneal Cancer - 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: M8327 - Name: Fallopian Tube Diseases - Relevance: LOW - As Found: Unknown - ID: M7 - Name: Abdominal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M8886 - Name: Gastrointestinal Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7256 - Name: Digestive System Neoplasms - Relevance: LOW - As Found: Unknown - ID: M7255 - Name: Digestive System Diseases - Relevance: LOW - As Found: Unknown - ID: M8883 - Name: Gastrointestinal Diseases - Relevance: LOW - As Found: Unknown - ID: M13441 - Name: Peritoneal Diseases - Relevance: LOW - As Found: Unknown - ID: T4352 - Name: Ovarian Cancer - Relevance: HIGH - As Found: Ovarian Cancer - ID: T2189 - Name: Fallopian Tube Cancer - Relevance: HIGH - As Found: Fallopian Tube Cancer - ID: T4354 - Name: Ovarian Epithelial Cancer - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000005185 - Term: Fallopian Tube Neoplasms - ID: D000010534 - Term: Peritoneal Neoplasms ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M246 - Name: Bevacizumab - Relevance: LOW - As Found: Unknown ### Misc Info Module #### Submission Tracking - Estimated Results First Submit Date: 2022-12-14 ##### Submission Infos - MCP Release N: Unknown - Release Date: 2022-12-14 - Reset Date: 2023-01-05 - Unrelease Date: Unknown - Unrelease Date Unknown: Unknown - MCP Release N: Unknown - Release Date: 2023-01-05 - Reset Date: 2023-01-31 - Unrelease Date: Unknown - Unrelease Date Unknown: Unknown - MCP Release N: Unknown - Release Date: 2023-03-06 - Reset Date: 2023-03-28 - Unrelease Date: Unknown - Unrelease Date Unknown: Unknown - MCP Release N: Unknown - Release Date: 2023-06-07 - Reset Date: 2023-06-27 - Unrelease Date: Unknown - Unrelease Date Unknown: Unknown - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04848779 **Brief Title:** A Prospective Study to Observe & Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD) **Official Title:** A Prospective Observational Study to Describe Clinical Outcomes of Alglucosidase Alfa Treatment in Patients ≤6 Months of Age With Infantile-onset Pompe Disease (IOPD) #### Organization Study ID Info **ID:** OBS17003 #### Organization **Class:** INDUSTRY **Full Name:** Sanofi #### Secondary ID Infos **Domain:** ICTRP **ID:** U1111-1266-4848 **Type:** REGISTRY ### Status Module #### Completion Date **Date:** 2026-03-30 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-02-09 **Type:** ACTUAL **Last Update Submit Date:** 2024-02-08 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2026-03-30 **Type:** ESTIMATED #### Start Date **Date:** 2021-06-10 **Type:** ACTUAL **Status Verified Date:** 2024-02 #### Study First Post Date **Date:** 2021-04-19 **Type:** ACTUAL **Study First Submit Date:** 2021-03-29 **Study First Submit QC Date:** 2021-04-13 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Sanofi #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Oversight Has DMC:** False ### Description Module **Brief Summary:** Primary Objective: To describe the effect of routine practice with alglucosidase alfa in patients with IOPD ≤6 months of age, on invasive ventilation-free survival after 52 weeks of treatment. Secondary Objectives: * To describe the effect of routine practice with alglucosidase alfa on invasive ventilation-free survival and survival at 12 and 18 months of age, as well as on change in left ventricular mass (LVM) Z score, Alberta Infant Motor Scale (AIMS) score, body weight, body length, and head circumference Z scores, and urinary glucose tetrasaccharide (Hex4), at Week 52 of treatment. * To describe the safety, tolerability, and immunogenicity of alglucosidase alfa in the routine practice of IOPD treatment. **Detailed Description:** The planned duration of observation for each participant will be 104 weeks after enrollment, to determine secondary outcomes at 18 months (approximately 78 weeks) of age. ### Conditions Module **Conditions:** - Glycogen Storage Disease Type II ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 16 **Type:** ESTIMATED **Patient Registry:** True **Study Type:** OBSERVATIONAL **Target Duration:** 104 Weeks ### Arms Interventions Module #### Arm Group 1 **Intervention Names:** - Drug: Alglucosidase alfa GZ419829 **Label:** Cohort 1 ### Interventions #### Intervention 1 **Arm Group Labels:** - Cohort 1 **Description:** Pharmaceutical form: Lyophilized powder for solution Route of administration: intravenous **Name:** Alglucosidase alfa GZ419829 **Other Names:** - Myozyme **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Proportion of participants alive and free of invasive ventilation at Week 52 of treatment **Time Frame:** Week 52 #### Secondary Outcomes **Measure:** Proportion of participants alive and free of invasive ventilation at 12 and 18 months of age **Time Frame:** at 12 and 18 months of age **Measure:** Proportion of participants alive at Week 52 of treatment **Time Frame:** Week 52 **Measure:** Proportion of participants alive at 12 months and 18 months of age **Time Frame:** at 12 and 18 months of age **Measure:** Proportion of participants free of ventilator use and free of supplemental oxygen use at Week 52 **Time Frame:** Week 52 **Measure:** Change from baseline to Week 52 in LVM Z score **Time Frame:** from baseline to Week 52 **Measure:** Change from baseline to Week 52 in AIMS score **Time Frame:** from baseline to Week 52 **Measure:** Change from baseline to Week 52 in body length Z-scores **Time Frame:** from baseline to Week 52 **Measure:** Change from baseline to Week 52 in body weight Z-scores **Time Frame:** from baseline to Week 52 **Measure:** Change from baseline to Week 52 in head circumference Z-scores **Time Frame:** from baseline to Week 52 **Measure:** Change from baseline to Week 52 in body length percentiles **Time Frame:** from baseline to Week 52 **Measure:** Change from baseline to Week 52 in body weight percentiles **Time Frame:** from baseline to Week 52 **Measure:** Change from baseline to Week 52 in head circumference percentiles **Time Frame:** from baseline to Week 52 **Measure:** Change from baseline to Week 52 in urinary Hex4 **Time Frame:** from baseline to Week 52 **Measure:** Number of participants experiencing at least 1 treatment-emergent adverse events (TEAE), including infusion-associated reactions (IAR) **Time Frame:** From inclusion for 104 weeks **Measure:** Number of participants with abnormalities in physical examinations **Time Frame:** From inclusion for 104 weeks **Measure:** Number of participants with abnormalities in clinical laboratory results **Time Frame:** From inclusion for 104 weeks **Measure:** Number of participants with abnormalities in vital signs measurements **Time Frame:** From inclusion for 104 weeks **Measure:** Number of participants with abnormalities in 12-lead electrocardiogram (ECG) **Time Frame:** From inclusion for 104 weeks **Measure:** Incidence of treatment-emergent antidrug antibodies (ADA) **Time Frame:** From inclusion for 104 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * At the time of informed consent, participants must be ≤6 months of age, corrected for gestation if necessary. Gestational age \<40 weeks will be adjusted to a full-term gestational age of 40 weeks. * Participants must have alglucosidase alfa enzyme replacement therapy (ERT) planned or initiated for IOPD treatment irrespective of study participation, according to the treating physician's decision regarding participants' routine disease management. * Participants must have available and accessible medical records from the time of IOPD diagnosis and from subsequent follow-up. * Participants must have a confirmed diagnosis of IOPD, defined as presence of 2 pathogenic acid alpha glucosidase (GAA) variants and documented GAA deficiency in blood (dried blood spot \[DBS\] accepted), skin, or muscle tissue, or presence of 1 pathogenic GAA variant and documented GAA deficiency in blood, skin, or muscle tissue from separate samples (either from 2 different tissues or from the same tissue but at 2 different sampling dates.) (DBS and leukocytes are acceptable as 2 different samples from blood). * Participants must have established cross-reacting immunologic material (CRIM) status available prior to enrollment. CRIM status may be provided by historical CRIM testing results or prediction of CRIM status based on genotyping performed at a Clinical Laboratory Improvement Amendments (CLIA) or other appropriately certified genetic laboratory. * Participants must have cardiomyopathy at the time of diagnosis (LVMI equivalent to mean age-specific LVMI): * LVMI +1 standard deviation (SD) in participants diagnosed by newborn or sibling screening, * LVMI +2 SD in participants diagnosed by clinical evaluation. * Participants must have informed consent provided by parent(s)/legally acceptable representatives (LARs). Exclusion Criteria: * Participants with respiratory insufficiency, defined as: * Oxygen saturation \<90% on room air as determined by pulse oximetry, * Venous partial pressure of carbon dioxide (pCO2) \>55 mmHg or arterial pCO2 \>40 mmHg on room air, * Use of invasive (with intubation or tracheostomy) or noninvasive (no intubation or tracheostomy) ventilation at enrollment, for participants not having started ERT at enrollment, * Use of invasive or noninvasive ventilation at the time of ERT initiation, for participants having started ERT before enrollment. * Participants with major congenital abnormality including heart defect, neural tube defect, or Down syndrome that, in the opinion of the investigator, would preclude participation in the study or potentially decrease survival. * Participants with clinically significant organic disease other than signs/symptoms related to Pompe disease, including clinically significant cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or circumstance that, in the opinion of the investigator, would preclude participation or potentially decrease survival. * Previous or ongoing treatment in any clinical trial of, or managed access program for, avalglucosidase alfa or any other Pompe disease-specific therapy. The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial. **Maximum Age:** 6 Months **Minimum Age:** 0 Days **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - CHILD **Study Population:** Confirmly diagnosed IOPD patients 6 months or less of age (corrected for gestational age if needed), treated or planned to be treated with alglucosidase alfa, at the time of study inclusion. ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Trial Transparency email recommended (Toll free number for US & Canada) **Phone:** 800-633-1610 **Phone Ext:** option 6 **Role:** CONTACT #### Locations **Location 1:** **City:** Valhalla **Country:** United States **Facility:** Boston Children's Health Physicians Site Number : 8400002 **State:** New York **Status:** RECRUITING **Zip:** 10595 **Location 2:** **City:** Durham **Country:** United States **Facility:** Duke University Medical Center Site Number : 8400004 **State:** North Carolina **Status:** RECRUITING **Zip:** 27710 **Location 3:** **City:** Cincinnati **Country:** United States **Facility:** Cincinnati Children's Hospital Medical Center - PIN Site Number : 8400001 **State:** Ohio **Status:** RECRUITING **Zip:** 45229-3039 **Location 4:** **City:** Seattle **Country:** United States **Facility:** Seattle Childrens Hospital and Regional Medical Center Site Number : 8400003 **State:** Washington **Status:** RECRUITING **Zip:** 98040 **Location 5:** **City:** Leuven **Country:** Belgium **Facility:** Investigational Site Number : 0560001 **Status:** RECRUITING **Zip:** 3000 **Location 6:** **City:** Tours **Country:** France **Facility:** Investigational Site Number : 2500001 **Status:** RECRUITING **Zip:** 37044 **Location 7:** **City:** Gießen **Country:** Germany **Facility:** Investigational Site Number : 2760001 **Status:** RECRUITING **Zip:** 35392 **Location 8:** **City:** Monza **Country:** Italy **Facility:** Investigational Site Number : 3800002 **State:** Monza E Brianza **Status:** RECRUITING **Zip:** 20052 **Location 9:** **City:** Firenze **Country:** Italy **Facility:** Investigational Site Number : 3800001 **Status:** RECRUITING **Zip:** 50139 **Location 10:** **City:** Rotterdam **Country:** Netherlands **Facility:** Investigational Site Number : 5280001 **Status:** RECRUITING **Zip:** 3015 GD **Location 11:** **City:** Esplugues de Llobregat **Country:** Spain **Facility:** Investigational Site Number : 7240001 **State:** Catalunya [Cataluña] **Status:** RECRUITING **Zip:** 08950 **Location 12:** **City:** Taipei **Country:** Taiwan **Facility:** Investigational Site Number : 1580001 **Status:** RECRUITING **Zip:** 100 **Location 13:** **City:** London **Country:** United Kingdom **Facility:** Investigational Site Number : 8260001 **State:** London, City Of **Status:** RECRUITING **Zip:** WC1N 3JH **Location 14:** **City:** Manchester **Country:** United Kingdom **Facility:** Investigational Site Number : 8260002 **Status:** RECRUITING **Zip:** M13 9WL #### Overall Officials **Official 1:** **Affiliation:** Sanofi **Name:** Clinical Sciences & Operations **Role:** STUDY_DIRECTOR ### IPD Sharing Statement Module **Description:** Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org **IPD Sharing:** YES ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020140 - Term: Lysosomal Storage Diseases, Nervous System - ID: D000020739 - Term: Brain Diseases, Metabolic, Inborn - ID: D000001928 - Term: Brain Diseases, Metabolic - ID: D000001927 - Term: Brain Diseases - ID: D000002493 - Term: Central Nervous System Diseases - ID: D000009422 - Term: Nervous System Diseases - ID: D000008661 - Term: Metabolism, Inborn Errors - ID: D000030342 - Term: Genetic Diseases, Inborn - ID: D000002239 - Term: Carbohydrate Metabolism, Inborn Errors - ID: D000016464 - Term: Lysosomal Storage Diseases - ID: D000008659 - Term: Metabolic Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC16 - Name: Diseases and Abnormalities at or Before Birth - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M9114 - Name: Glycogen Storage Disease - Relevance: HIGH - As Found: Glycogen Storage Disease - ID: M9115 - Name: Glycogen Storage Disease Type II - Relevance: HIGH - As Found: Glycogen Storage Disease Type II - ID: M18871 - Name: Lysosomal Storage Diseases - Relevance: LOW - As Found: Unknown - ID: M5204 - Name: Brain Diseases - Relevance: LOW - As Found: Unknown - ID: M5205 - Name: Brain Diseases, Metabolic - Relevance: LOW - As Found: Unknown - ID: M22498 - Name: Brain Diseases, Metabolic, Inborn - Relevance: LOW - As Found: Unknown - ID: M5742 - Name: Central Nervous System Diseases - Relevance: LOW - As Found: Unknown - ID: M11641 - Name: Metabolism, Inborn Errors - Relevance: LOW - As Found: Unknown - ID: M23686 - Name: Genetic Diseases, Inborn - Relevance: LOW - As Found: Unknown - ID: M5498 - Name: Carbohydrate Metabolism, Inborn Errors - Relevance: LOW - As Found: Unknown - ID: M11639 - Name: Metabolic Diseases - Relevance: LOW - As Found: Unknown - ID: T2562 - Name: Glycogen Storage Disease Type 2 - Relevance: HIGH - As Found: Glycogen Storage Disease Type II ### Condition Browse Module - Meshes - ID: D000006009 - Term: Glycogen Storage Disease Type II - ID: D000006008 - Term: Glycogen Storage Disease ### 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:** NCT03525379 **Acronym:** REV-HF **Brief Title:** Evaluating the Clinical Efficacy of Resveratrol in Improving Metabolic and Skeletal Muscle Function in Patients With Heart Failure **Official Title:** Evaluating the Clinical Efficacy of Resveratrol in Improving Metabolic and Skeletal Muscle Function in Patients With Heart Failure #### Organization Study ID Info **ID:** REV-HF-2015-1 #### Organization **Class:** OTHER **Full Name:** University of Alberta ### Status Module #### Completion Date **Date:** 2019-10-08 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-01-12 **Type:** ACTUAL **Last Update Submit Date:** 2023-01-11 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2019-10-08 **Type:** ACTUAL #### Start Date **Date:** 2017-10-26 **Type:** ACTUAL **Status Verified Date:** 2022-05 #### Study First Post Date **Date:** 2018-05-15 **Type:** ACTUAL **Study First Submit Date:** 2018-02-01 **Study First Submit QC Date:** 2018-05-02 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Alberta #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** This is a randomized, double-blind, placebo-controlled trial evaluating the effect of resveratrol on metabolic and skeletal muscle function. Patients will be randomized and allocated to either resveratrol or placebo, for 8 weeks with a 2-week run-in period before the intervention period (to ensure they are not on any nutritional supplement that contains resveratrol). **Detailed Description:** Trial Objectives Primary outcome- Change in Isolated Skeletal Muscle Blood Flow, Oxygen Extraction and Consumption and Metabolic Rate of Recovery with Exercise by Cardiac MRI (CMR). In order to evaluate potential changes in skeletal muscle vascular function and oxygen extraction with resveratrol therapy, the investigators will use a lower limb (calf muscle) targeted exercise protocol in conjunction with magnetic resonance imaging (MRI)32. MRI will be used to measure, simultaneously, the peak rate of blood flow in the calf muscle along with the whole calf muscle extraction of oxygen, which together are used to determine oxygen consumption. This method allows the determinants of oxygen consumption (blood flow and oxygen extraction) to be evaluated, which is necessary to understand the mechanisms of therapy action. Secondary outcomes- 1. Change in Vascular Function by CMR. Measurement of aortic distensibility will be used to assess changes in the compliance of vascular system with therapy. Compliance will be defined as the fractional change in cross-sectional area of the aorta over the cardiac cycle (measured at both ascending and descending locations) divided by the corresponding change in pressure (pulse pressure = systolic blood pressure - diastolic blood pressure, measured with arm cuff at time of aorta imaging). 2. Change in Body Composition by CMR. Assessment of adipose and visceral fat content in abdomen and skeletal muscle will be made using multi-echo DIXON magnetic resonance image acquisition with automated fat/water decomposition using VARPO post-processing. 3. Phosphocreatinine (PCr) uptake/recovery on skeletal muscle by CMR. Skeletal muscle metabolism will be measured on a 3T PRISMA MRI system (Peter S Allen MRI Centre) equipped with a plantar-flexion exercise setup and phosphorous imaging capabilities. Supine subjects will perform exercise (toe-push on one leg) until exhaustion. P NMR spectra will be acquired continuously during exercise to measure PCr utilization, change in muscle pH, and most importantly, the recovery rate following exercise. PCr recovery following exercise is a direct measure of substrate utilization and oxygen supply, which is measured as the rate of PCr replenishment. The rate constant (in seconds) characterizes the exponential recovery of PCr to resting values (providing a quantitative measure of oxidative phosphorylation. Normal values from healthy controls are 30-35 seconds with prior studies of patients with HF show values of 76 seconds. A study of reproducibility showed a coefficient of variability of 4.6% for this rate constant (from repeated studies on separate days). 4. Change in LVEF, LVEDV and longitudinal strain by CMR. CMR remains the most precise measure of cardiac structure and function and allows for very small sample sizes to detect changes in important variables such as left ventricular ejection fraction (LVEF), left ventricular end diastolic volume (LVEDV) and longitudinal strain. Longitudinal strain is complimentary to volume and ejection fraction and is increasingly used to detect subclinical changes in heart function even in cases of preserved ejection fraction, and is predictive of HF outcomes. The investigators have reported a coefficient of variability of 2.6% for measurement of ventricular volumes with MRI, similar or better than previous studies, which would enable the measurement of a change of 5 ml in LVEDV with therapy, for example, with only 10 subjects (α=0.05, power=0.95). 5. Distance walked on 6-minute walk test (6-MWT). The 6-MWT is a validated and reproducible clinical endpoint that has been used to demonstrate differences in early phase work of medications such as ACE inhibitors, cardiac resynchronization therapy, and exercise interventions. 6. Change in the Kansas City Cardiomyopathy Questionnaire (KCCQ). The KCCQ has been used for multiple large RCT and is sensitive to change, validated in patients with heart failure and linked to prognosis in the short and long-term. A minimal clinically important difference is 5 points. 7. Change in Functional Assessment of Chronic Illness Therapy (FACIT-F). The FACIT-F was developed as a quality of life tool to evaluate small changes in fatigue for patients undergoing anemia or cancer therapy. There are no other validated tools specific to heart failure that address fatigue, despite the principal importance of this symptom. Therefore, the FACIT-F will be used to measure fatigue. 8. Change in Sleep Quality. The Medical Outcomes Study (MOS) Sleep Scale is a patient-reported, non-disease-specific instrument for evaluating sleep outcomes. The MOS Sleep Scale measures subjective experiences of sleep across several different domains. ### Conditions Module **Conditions:** - Congestive Heart Failure Chronic **Keywords:** - Skeletal Muscle Blood Flow - (calf muscle) targeted exercise ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** SEQUENTIAL **Intervention Model Description:** After providing written informed consent, patients who qualify will be randomized via the web-based system coordinated by Research Electronic Data Capture (REDCap) at the University of Alberta. A patient's eligibility will be confirmed and a unique identifier will be assigned. Blinding of the patients and study personnel is preserved by using matching placebo capsules. Clinical outcomes will be adjudicated by a Clinical Events Committee, and core laboratories will assess the CMR measures and biomarkers independently and blinded to treatment assignment. ##### Masking Info **Masking:** DOUBLE **Masking Description:** The randomization codes will be maintained in REDCap. The principal investigator will have authority to unblind patients if safety concerns arise. **Who Masked:** - PARTICIPANT - CARE_PROVIDER **Primary Purpose:** DIAGNOSTIC #### Enrollment Info **Count:** 13 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 1) Resveratrol- (Transmax) trans- resveratrol (Biotivia Longevity Bioceuticals, LLC, New York, USA) in cellulose capsules. One capsule will be taken orally 2 times per day (BID) for 8 weeks. **Intervention Names:** - Drug: Resveratrol - Drug: Placebo **Label:** Resveratrol **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** 2) Placebo- 500mg (Biotivia Longevity Bioceuticals, LLC, New York, USA) in cellulose capsules. One capsule will be taken orally 2 times per day (BID) for 8 weeks. **Intervention Names:** - Drug: Resveratrol - Drug: Placebo **Label:** Placebo **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Placebo - Resveratrol **Description:** Subject participation will be approximately 10 weeks in duration. Participants will be randomized to receive one treatment: either resveratrol for 8 weeks OR placebo for 8 weeks. There will be a 2-week run-in period prior to the treatment period. The schedule for the study is: Week 0 Screening and randomization Weeks 1-2 Run-in; no treatment Weeks 3-10 Treatment Phase (resveratrol or placebo) **Name:** Resveratrol **Other Names:** - (Transmax) trans- resveratrol **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Placebo - Resveratrol **Description:** Participants will be randomized to receive one treatment: either resveratrol for 8 weeks OR placebo for 8 weeks. There will be a 2-week run-in period prior to the treatment period. **Name:** Placebo **Other Names:** - Subject participation will be approximately 10 weeks in duration. Participants will be randomized to receive one treatment: either resveratrol for 8 weeks OR placebo for 8 weeks **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** In order to evaluate potential changes in skeletal muscle vascular function and oxygen extraction with resveratrol therapy, the investigators will use a lower limb (calf muscle) targeted exercise protocol in conjunction with magnetic resonance imaging (MRI). MRI will be used to measure, simultaneously, the peak rate of blood flow in the calf muscle along with the whole calf muscle extraction of oxygen, which together is used to determine oxygen consumption. This method allows the determinants of oxygen consumption (blood flow and oxygen extraction) to be evaluated, which is necessary to understand the mechanisms of therapy action. **Measure:** Change in Isolated Skeletal Muscle Blood Flow **Time Frame:** 10 weeks **Description:** In order to evaluate potential changes in oxygen extraction with resveratrol therapy, the investigators will use a lower limb (calf muscle) targeted exercise protocol in conjunction with magnetic resonance imaging (MRI). MRI will be used to measure, simultaneously, the peak rate of blood flow in the calf muscle along with the whole calf muscle extraction of oxygen, which together is used to determine oxygen consumption. This method allows the determinants of oxygen consumption (blood flow and oxygen extraction) to be evaluated, which is necessary to understand the mechanisms of therapy action. **Measure:** Change in Oxygen extraction and consumption **Time Frame:** 10 weeks #### Secondary Outcomes **Description:** Measurement of aortic distensibility will be used to assess changes in the compliance of vascular system with therapy. Compliance will be defined as the fractional change in cross-sectional area of the aorta over the cardiac cycle (measured at both ascending and descending locations) **Measure:** Vascular Function by CMR **Time Frame:** 10 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Male: Given the nature of the project and the need to have normative data for variables that may vary by sex, male patients only will be included. 2. Age: Patients should be 50 to 75 years of age. Patients who are younger or older may have variations in skeletal muscle or activity levels that would increase the variation in functional testing. 3. Clinically diagnosed heart failure with reduced ejection fraction (HFrEF; left ventricular ejection fraction \<45%) or heart failure with preserved ejection fraction (HFpEF; left ventricular ejection fraction \>45%) as defined by the Alberta HEART study. Exclusion Criteria: 1. Unable to undergo CMR imaging. 2. CMR exclusions: renal failure \[a glomerular filtration rate \<30 mL/min)\], implantable cardiac device (ICD or CRT), uncontrolled atrial fibrillation or recurrent ventricular arrhythmias). 3. General medical conditions: uncontrolled thyroid disorders, hepatic failure, or myocardial revascularization procedures \[coronary angioplasty and/or surgical revascularization in the previous 3 months\], cancer/malignancy, or with moderate-severe dementia). 4. Patients taking any of the following: oral anticoagulants, insulin, dihydropyridine calcium channel blockers, sildenafil or midazolam. 5. Patients with allergies to the study products. 6. Patients with hormonal disorders. 7. Unwilling to stop regular use of natural health products or dietary supplements containing resveratrol for 14 days prior to study entry and for the duration of the study. **Gender Based:** True **Gender Description:** Male: Given the nature of the project and the need to have normative data for variables that may vary by sex, male patients only will be included. **Maximum Age:** 75 Years **Minimum Age:** 50 Years **Sex:** MALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Edmonton **Country:** Canada **Facility:** Alberta Cardiovascular and Stroke Research Centre **State:** Alberta **Zip:** T6G 2B7 ### IPD Sharing Statement Module **IPD Sharing:** UNDECIDED ## Derived Section ### Condition Browse Module - Ancestors - ID: D000006331 - Term: Heart Diseases - ID: D000002318 - Term: Cardiovascular Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M9421 - Name: Heart Failure - Relevance: HIGH - As Found: Heart Failure - ID: M9419 - Name: Heart Diseases - Relevance: LOW - As Found: Unknown - ID: T1303 - Name: Chronic Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000006333 - Term: Heart Failure ### Intervention Browse Module - Ancestors - ID: D000000975 - Term: Antioxidants - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000020011 - Term: Protective Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000004791 - Term: Enzyme Inhibitors - ID: D000010975 - Term: Platelet Aggregation Inhibitors ### Intervention Browse Module - Browse Branches - Abbrev: PlAggInh - Name: Platelet Aggregation Inhibitors - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M1684 - Name: Resveratrol - Relevance: HIGH - As Found: Avastin - ID: M4292 - Name: Antioxidants - Relevance: LOW - As Found: Unknown - ID: M21869 - Name: Protective Agents - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown - ID: M13865 - Name: Platelet Aggregation Inhibitors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000077185 - Term: Resveratrol ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02161679 **Brief Title:** Phase II Study of IMMU-132 Alone or in Combination With Carboplatin in Patients With Triple-Negative Breast Cancer **Official Title:** Randomized Phase II Study of IMMU-132 Alone or in Combination With Carboplatin in Patients With Relapsed/Refractory Triple-Negative Breast Cancer #### Organization Study ID Info **ID:** IMMU-132-02 #### Organization **Class:** INDUSTRY **Full Name:** Gilead Sciences ### Status Module #### Completion Date **Date:** 2016-08 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-08-19 **Type:** ACTUAL **Last Update Submit Date:** 2021-08-12 **Overall Status:** WITHDRAWN #### Primary Completion Date **Date:** 2016-08 **Type:** ACTUAL #### Start Date **Date:** 2016-08 **Type:** ACTUAL **Status Verified Date:** 2020-09 #### Study First Post Date **Date:** 2014-06-12 **Type:** ESTIMATED **Study First Submit Date:** 2014-06-10 **Study First Submit QC Date:** 2014-06-11 **Why Stopped:** FDA asked to administratively split from IND115621- to open a new IND you need to file a protocol we only drafted it to get the IND open - never initiated ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Gilead Sciences #### Responsible Party **Type:** SPONSOR ### Description Module **Brief Summary:** This is a Phase II, open-label study that evaluates the safety and efficacy of IMMU-132 alone and in combination with carboplatin in patients with triple-negative breast cancer. IMMU132 will be administered once-weekly for the first 2 weeks of 3-week treatment cycles. For those patients assigned to also receive carboplatin, will receive it on the same schedules starting 30 minutes after the completion of IMMU-132 administration. Patients may receive up to a maximum total of 8 cycles. **Detailed Description:** The primary objective is to evaluate the safety and efficacy of IMMU-132 alone and in combination with carboplatin administered in 3-week treatment cycles for up to 8 cycles, in patients with triple-negative breast cancer that have received at least two prior treatments. The secondary objectives are to obtain data concerning pharmacokinetics, and immunogenicity. This is a multi-center study. Eighty patients are planned to be enrolled, with an equal distribution between the two groups. All patients will receive a starting dose of IMMU-132administered once-weekly for the first 2 weeks of 3-week treatment cycles. For those patients assigned to also receive carboplatin, carboplatin will also be administered on the same schedules starting 30 minutes after the completion of IMMU-132 administration. Patients may receive up to a maximum total of 8 cycles (16 doses), but patients with a complete response, partial response or stable disease at that time, or patients who had achieved an objective response, but relapsed after discontinuing treatment, may continue to be treated based on physician discretion. Follow up is then required until resolution or stabilization of any treatment-related toxicity, and patients with stable disease or objective responses must also continue evaluations until survived. ### Conditions Module **Conditions:** - Triple-negative Breast Cancer **Keywords:** - Antibody drug conjugate - IMMU-132 - Triple-negative breast cancer - Safety and tolerability - Progression Free Survival (PFS) ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** IMMU-132 infusion is administered **Intervention Names:** - Drug: IMMU-132 infusion is administered to participants in one arm for the study - Drug: IMMU-132 plus Carboplatin infusion **Label:** IMMU-132 **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** IMMU-132 infusion and Carboplatin infusion are administered to the participants in this arm of study. **Intervention Names:** - Drug: IMMU-132 infusion is administered to participants in one arm for the study - Drug: IMMU-132 plus Carboplatin infusion **Label:** IMMU-132 plus Carboplatin **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - IMMU-132 - IMMU-132 plus Carboplatin **Name:** IMMU-132 infusion is administered to participants in one arm for the study **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - IMMU-132 - IMMU-132 plus Carboplatin **Name:** IMMU-132 plus Carboplatin infusion **Other Names:** - IMMU-132 and Carboplatin infusions are administered to the participants in this arm of study **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Progression Free Survival **Time Frame:** 2 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Female subjects with triple negative metastatic breast cancer, age 18 years or older, pathologically confirmed metastatic adenocarcinoma of the breast. Pathologically confirmed as triple-negative, measurable disease, defined by (RECIST 1.1) guidelines; * Two or more prior chemotherapy, immunotherapy and/or monoclonal antibody therapy for the treatment of the subjects' metastatic breast cancer; * Prior neoadjuvant or adjuvant chemotherapy must have been completed at least 4 weeks before start of study treatment with all related toxicities resolved; * Prior radiotherapy must have completed at least 2 weeks before randomization, with full recovery; * At least 4 weeks from major surgery, ECOG performance status 0-1. * Hematology parameters (ANC) ≥ 1500/mm2; * Platelets ≥ 100,000/mm2; * Hemoglobin (Hgb) ≥ 9 g/dL AST \& ALT ≤ 2.5 x ULN); * If hepatic metastases present ≤ 5.0 x ULN Total bilirubin ≤ ULN ; * Subjects with Gilbert's syndrome can have bilirubin of up to 1.5 x ULN; * Alkaline phosphatase ≤ 2.5 x ULN (unless bone metastases are present in the absence of liver metastasis); * Creatinine clearance \> 60 mL/min Exclusion Criteria: * Male subjects with triple negative metastatic breast cancer; * Concurrent chemotherapy, immunotherapy or monoclonal antibody or any other anti-tumor therapy for breast cancer, * Concurrent or prior anticoagulation therapy within 7 days of first dose of study treatment, * History of, or known current evidence of brain metastasis, including leptomeningeal involvement; * Subjects with bone as the only site of metastatic disease. **Maximum Age:** 90 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ## 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 - Abbrev: BC23 - Name: Symptoms and General Pathology ### 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: M20559 - Name: Disease Progression - 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 - ID: D000064726 - Term: Triple Negative Breast Neoplasms ### Intervention Browse Module - Ancestors - ID: D000000970 - Term: Antineoplastic Agents - ID: D000007155 - Term: Immunologic Factors - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000000972 - Term: Antineoplastic Agents, Phytogenic - ID: D000059004 - Term: Topoisomerase I Inhibitors - ID: D000059003 - Term: Topoisomerase 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: ANeo - Name: Antineoplastic Agents ### Intervention Browse Module - Browse Leaves - ID: M4225 - Name: Antibodies - Relevance: LOW - As Found: Unknown - ID: M18650 - Name: Carboplatin - Relevance: HIGH - As Found: System - ID: M287620 - Name: Sacituzumab govitecan - Relevance: HIGH - As Found: Myo- - ID: M10184 - Name: Immunoglobulins - Relevance: LOW - As Found: Unknown - ID: M5426 - Name: Camptothecin - Relevance: HIGH - As Found: Myo- - ID: M20855 - Name: Immunoconjugates - Relevance: HIGH - As Found: Myo- - ID: M10201 - Name: Immunologic Factors - Relevance: LOW - As Found: Unknown - ID: M29349 - Name: Topoisomerase I Inhibitors - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000016190 - Term: Carboplatin - ID: D000002166 - Term: Camptothecin - ID: C000608132 - Term: Sacituzumab govitecan - ID: D000018796 - Term: Immunoconjugates ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00067379 **Brief Title:** Early Orthodontic Intervention Under Medicaid **Official Title:** Early Orthodontic Intervention Under Medicaid #### Organization Study ID Info **ID:** NIDCR-142542 #### Organization **Class:** OTHER **Full Name:** University of Washington #### Secondary ID Infos **ID:** U54DE014254 **Link:** https://reporter.nih.gov/quickSearch/U54DE014254 **Type:** NIH ### Status Module #### Completion Date **Date:** 2009-06 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** ACTIVE_NOT_RECRUITING #### Last Update Post Date **Date:** 2010-06-10 **Type:** ESTIMATED **Last Update Submit Date:** 2010-06-09 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2009-06 **Type:** ESTIMATED #### Start Date **Date:** 2003-04 **Status Verified Date:** 2010-06 #### Study First Post Date **Date:** 2003-08-19 **Type:** ESTIMATED **Study First Submit Date:** 2003-08-15 **Study First Submit QC Date:** 2003-08-18 ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** National Institute of Dental and Craniofacial Research (NIDCR) **Class:** OTHER **Name:** Northwest and Alaska Center on Oral Health Disparities **Class:** OTHER **Name:** Washington State Department of Social and Health Services **Class:** OTHER **Name:** Seattle Children's Hospital #### Lead Sponsor **Class:** OTHER **Name:** University of Washington #### Responsible Party **Old Name Title:** Gregory King, DMD, PhD **Old Organization:** University of Washington ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The primary objective of this study is to examine the usefulness of early orthodontic intervention as a means of increasing access to orthodontic services for children of low-income families. **Detailed Description:** Orthodontic treatment has become a widely accepted procedure in dentistry. The benefits include improved oral health, function, esthetics and quality of life. Significant disparities exist among income strata regarding access to orthodontic services. The sources of these disparities are complex and may reflect differences in the disease prevalence, gender, cultural biases, perception of problems by this population, economic imperatives and negative perceptions of these patients by orthodontists. The primary objective of this study is to examine the usefulness of early orthodontic intervention as a means of increasing access to orthodontic services for children of low-income families. Aim 1. To compare orthodontic outcomes, facial body image, and quality of life between Medicaid participants who receive early orthodontic treatment and those who do not. 1.a To compare the level of understanding and compliance between early treatment subjects given information about the goals, risks and benefits of the planned treatment one-on-one by an orthodontist, with subjects who also use an interactive CD-ROM to provide this information. This will be referred to as the Informed Consent Study and should not be confused with the routine informed consenting process used to enroll subjects into the overall study.Aim 2. To compare orthodontic outcomes, facial body image, and quality of life between Medicaid-funded and private-pay patients who receive full orthodontic treatment at adolescence.Aim 3. To compare orthodontic outcomes, facial body image, and quality of life between Medicaid-funded patients who receive early orthodontic treatment only and Medicaid-funded participants who receive full orthodontic treatment at adolescence. Relationship of this project with the Disparity Center theme. Two of the goals of the Northwest and Alaska Center for Oral Health Disparity are met by this study. The first is to conduct clinical research to evaluate the efficacy of interventions to prevent and treat oral diseases and conditions in children. The second is to develop community-based research that translates existing knowledge and new information regarding children and their caretakers into new technologies and interventions that hold promise for reducing disparities. Today, children of low-income families have very limited access to orthodontic treatment. We also know that some relatively simple interventions done during the mixed dentition can be effective at reducing the severity of malocclusion. This study is designed to examine how effective these early orthodontic interventions are in a Medicaid population. It will also examine how outcomes from early treatment in Medicaid patients compare to the more complex approach of complete orthodontic treatment in the permanent dentition. Many orthodontists perceive that Medicaid patients are at risk for poor outcomes, and limit access as a consequence. The follow-up component of this study will address this issue by making a comparison between Medicaid and private-pay patients treated in similar environments. We expect that significantly greater access to orthodontic services could be provided for Medicaid patients by the more widespread use of simpler, more timely interventions. This study will provide data on the trade-off between simple, timely partial treatments, versus complete full treatments. In response to the second theme of the Disparity Center, the use of interactive CD-ROMs that provide treatment information at the comprehension level of young patients and their parents, and in a culturally appropriate manner, will be examined. We expect this approach will improve patient and parent understanding of the proposed treatment, as well as enhancing compliance with treatment procedures in order to assure successful outcomes. ### Conditions Module **Conditions:** - Malocclusion ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 150 **Type:** ESTIMATED **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Medicaid patients with medically necessary malocclusions treated during the mixed dentition with limited goals followed by observation **Intervention Names:** - Procedure: interceptive orthodontic treatments **Label:** 1 **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - 1 **Description:** orthodontic procedures **Name:** interceptive orthodontic treatments **Type:** PROCEDURE ### Outcomes Module #### Primary Outcomes **Measure:** Peer Assessment Rating **Time Frame:** Pre-study, post - early treatment or observation, post full treatment or observation ### Eligibility Module **Eligibility Criteria:** "Inclusion Criteria:" * Enrolled in Medicaid * Fluency in English, Spanish, Vietnamese, Chinese, Somalian or Ethiopian * Acceptable malocclusion * Free of oral disease * Current immunizations and record of dental care * Acceptable oral hygiene * Anticipate living in the area for 4 years * Absence of craniofacial anomalies * Absence of unilateral posterior crossbite with facial asymmetry * No prior orthodontic treatment * Agree to be randomized to early orthodontic treatment or late full treatment * Signs assent/consent **Maximum Age:** 11 Years **Minimum Age:** 8 Years **Sex:** ALL **Standard Ages:** - CHILD ### Contacts Locations Module #### Locations **Location 1:** **City:** Seattle **Country:** United States **Facility:** Odessa Brown Children's Clinic **State:** Washington **Zip:** 98122 #### Overall Officials **Official 1:** **Affiliation:** University of Washington **Name:** Gregory J King, DDS **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** King GJ, Spiekerman CF, Greenlee GM, Huang GJ. Randomized clinical trial of interceptive and comprehensive orthodontics. J Dent Res. 2012 Jul;91(7 Suppl):59S-64S. doi: 10.1177/0022034512448663. **PMID:** 22699670 ## 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: M11301 - Name: Malocclusion - Relevance: HIGH - As Found: Malocclusion - 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: D000008310 - Term: Malocclusion ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04405479 **Brief Title:** Resting Postural Tremor in Multiple Sclerosis **Official Title:** Spectral Analyses of Resting Postural Tremor Measured by Accelerometer and Gyroscope as a Predictive Tool of Multiple Sclerosis #### Organization Study ID Info **ID:** EK-VP/23/0/2014 #### Organization **Class:** OTHER **Full Name:** Charles University, Czech Republic ### Status Module #### Completion Date **Date:** 2024-12-30 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-02-06 **Type:** ACTUAL **Last Update Submit Date:** 2024-02-05 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-10-28 **Type:** ESTIMATED #### Start Date **Date:** 2021-05-01 **Type:** ACTUAL **Status Verified Date:** 2024-02 #### Study First Post Date **Date:** 2020-05-28 **Type:** ACTUAL **Study First Submit Date:** 2020-05-20 **Study First Submit QC Date:** 2020-05-27 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Charles University, Czech Republic #### Responsible Party **Investigator Affiliation:** Charles University, Czech Republic **Investigator Full Name:** Kamila Řasová **Investigator Title:** associate professor PhDr. Kamila Řasová, Ph.D. **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** Early diagnosis is a key factor for effective treatment of multiple sclerosis. Accelerometer and gyroscope measurement of tremor could screen potential motor control dysfunction and predict a risk for development of multiple sclerosis. **Detailed Description:** The aim of this study is to test whether specially developed accelerometer and gyroscope can differentiate pattern of resting postural tremor in healthy and people with multiple sclerosis. Spectral analysis and the correlation of the average signal spectra will be computed. Moreover, categorization based on the tremor component according to the maximum likelihood estimation will be done. ### Conditions Module **Conditions:** - Multiple Sclerosis **Keywords:** - Tremor - Accelerometer ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 30 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** MS patients will be chosen by an independent neurologist. **Intervention Names:** - Behavioral: Examination of tremor and upper hand functions **Label:** Patients with multiple sclerosis #### Arm Group 2 **Description:** Healthy controls will be paired by age and sex with MS patients. **Intervention Names:** - Behavioral: Examination of tremor and upper hand functions **Label:** Healthy volunteers ### Interventions #### Intervention 1 **Arm Group Labels:** - Healthy volunteers - Patients with multiple sclerosis **Description:** Participants will be only examined. They will not undergo any intervention. **Name:** Examination of tremor and upper hand functions **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Description:** accelerometer (1 m·s-2) **Measure:** Tremor **Time Frame:** baseline #### Secondary Outcomes **Description:** Hand dynamometer JAMA **Measure:** Hand grip strength **Time Frame:** baseline **Description:** Nine Hole Peg Test **Measure:** Hand and arm function **Time Frame:** baseline **Description:** Coin Rotation Task **Measure:** Psychomotor processing speed **Time Frame:** baseline ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: Group 1: * Definite diagnosis of MS * prevailed motor deficit of upper extremities, but without visible tremor * stable clinical status in the preceding 3 months * Expanded Disability Status Scale score (EDSS) ≥ 2 ≤ 7.5, and * no corticosteroid therapy in the preceding month Group 2: * Healthy volunteers in the age of 20-70 years will be employed * There will be defined as persons without any neurological disease diagnose at the time of the measurement. Exclusion Criteria (both groups): Patients with factors disturbing mobility (e.g. stroke, pregnancy, fractures) were excluded from the study. **Healthy Volunteers:** True **Maximum Age:** 70 Years **Minimum Age:** 20 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** People with MS and healthy volunteers ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Kamila Řasová, Ph.D. **Phone:** +420604511416 **Role:** CONTACT #### Locations **Location 1:** **City:** Prague **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Ivana Stetkarova, prof MD PhD - **Phone:** +420197170 - **Role:** CONTACT **Country:** Czechia **Facility:** Department of neurology **Status:** RECRUITING **Zip:** 100 00 **Location 2:** **City:** Prague **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Tom Philipp, M.D., Ph.D. - **Phone:** +420737273589 - **Role:** CONTACT **Country:** Czechia **Facility:** Deparment of revmatology and rehabilitation **Status:** RECRUITING **Zip:** 140 59 **Location 3:** **City:** Praha **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Kamila Řasová, Ph.D. - **Phone:** +420604511416 - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Natalia Hrušková - **Phone:** +420702836959 - **Role:** CONTACT **Country:** Czechia **Facility:** Kamila Řasová **Status:** RECRUITING **Zip:** 128 00 #### Overall Officials **Official 1:** **Affiliation:** Third Faculty of Mecicine, Charles Univerzity **Name:** Kamila Řasová, Ph.D. **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000010335 - Term: Pathologic Processes - ID: D000020278 - Term: Demyelinating Autoimmune Diseases, CNS - ID: D000020274 - Term: Autoimmune Diseases of the Nervous System - ID: D000009422 - Term: Nervous System Diseases - ID: D000003711 - Term: Demyelinating Diseases - ID: D000001327 - Term: Autoimmune Diseases - ID: D000007154 - Term: Immune System Diseases - ID: D000020820 - Term: Dyskinesias - ID: D000009461 - Term: Neurologic Manifestations ### Condition Browse Module - Browse Branches - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC20 - Name: Immune System Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M12060 - Name: Multiple Sclerosis - Relevance: HIGH - As Found: Multiple Sclerosis - ID: M15415 - Name: Sclerosis - Relevance: HIGH - As Found: Sclerosis - ID: M16956 - Name: Tremor - Relevance: HIGH - As Found: Tremor - 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: M6909 - Name: Demyelinating Diseases - Relevance: LOW - As Found: Unknown - ID: M10200 - Name: Immune System Diseases - Relevance: LOW - As Found: Unknown - ID: M22574 - Name: Dyskinesias - Relevance: LOW - As Found: Unknown - ID: M12404 - Name: Neurologic Manifestations - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000009103 - Term: Multiple Sclerosis - ID: D000014202 - Term: Tremor - ID: D000012598 - Term: Sclerosis ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00619879 **Brief Title:** Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies **Official Title:** Myeloablative Hematopoietic Progenitor Cell Transplantation (HPCT) for Pediatric Malignancies #### Organization Study ID Info **ID:** SCT 0307 #### Organization **Class:** OTHER **Full Name:** Ann & Robert H Lurie Children's Hospital of Chicago ### Status Module #### Completion Date **Date:** 2020-01 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2011-03-30 **Type:** ESTIMATED **Last Update Submit Date:** 2011-03-29 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2020-01 **Type:** ESTIMATED #### Start Date **Date:** 2007-03 **Status Verified Date:** 2011-03 #### Study First Post Date **Date:** 2008-02-21 **Type:** ESTIMATED **Study First Submit Date:** 2008-01-08 **Study First Submit QC Date:** 2008-02-07 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Ann & Robert H Lurie Children's Hospital of Chicago #### Responsible Party **Old Name Title:** Morris Kletzel, MD **Old Organization:** Children's Memorial Hospital ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to show that myeloablative hematopoietic progenitor cell transplantation (HPCT) continues to offer acceptable disease-free survival for select patients requiring HPCT. **Detailed Description:** Myeloablative hematopoietic progenitor cell transplantation (HPCT) remains the standard of care for patients requiring HPCT. The purpose of this study is to evaluate the morbidity and mortality of myeloablative HPCT at Children's Memorial Hospital. It will also look to determine the toxicity of a single conditioning regimen consisting of total body irradiation (TBI), etoposide (VP-16), and Cyclophosphamide for patients with transplant eligible lymphoid malignant conditions or with transplant eligible myeloid malignant conditions who are receiving cord blood units, or to determine the toxicity of a single conditioning regimen consisting of Busulfan and Cyclophosphamide for patients with transplant eligible myeloid malignant conditions who are not receiving cord blood units. ### Conditions Module **Conditions:** - Leukemia, Myelogenous, Chronic - Leukemia, Lymphoblastic, Acute - Leukemia, Myelogenous, Acute - Myeloproliferative-Myelodysplastic Diseases - Lymphoma, Malignant **Keywords:** - Leukemia, Myelogenous, Chronic - Leukemia, Lymphoblastic, Acute - Leukemia, Myelogenous, Acute - Myeloproliferative-Myelodysplastic Diseases - Juvenile Myelomonocytic Leukemia - Dysmyelopoietic Syndromes - Lymphoma, Malignant - Stem Cell Transplantation, Hematopoietic - Allogeneic Transplantation - Human Leukocyte Antigens - Busulfan - Total Body Irradiation - VP-16 - Etoposide - Cyclophosphamide - Graft-Versus-Host Disease ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 200 **Type:** ESTIMATED **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Description:** Total Body Irradiation (TBI) 1200 cGy will be given on days -8,-7,-6 and -5 in eight sessions, delivering 150cGy in each session. Etoposide 1000 mg/m2 as a 24 hour continuous infusion started on day -4. Cyclophosphamide 60 mg/kg/day IV given over 1 hour daily on days -3, -2. **Name:** Myeloablative Chemotherapy Regimen for Lymphoid Malignancies or Cord Blood Unit Recipients **Type:** DRUG #### Intervention 2 **Description:** Busulfan administration: * For children \>/= 4 years of age, Busulfan 0.8 mg/kg/dose will be given every 6 hours over days -8,-7, -6, and -5 for a total of 16 doses. * For children \< 4 years of age, Busulfan 1 mg/kg/dose will be given every 6 hours over days -8, -7, -6, -5 for a total of 16 doses. * Pharmacokinetic analysis will guide dose modifications targeted to receive an average AUC of 800-1200 microMols\*min for the 16 doses. Lorazepam (0.05 mg/kg) IV will be administered one half hour before the initial dose of Busulfan is given and every 6 hours through day -4. Etoposide 1000 mg/m2 as a 24 hour continuous infusion started on day -4. Cyclophosphamide 60 mg/kg/day IV given over 1 hour daily on days -3 and -2. **Name:** Myeloablative Chemotherapy Regimen for Non-Cord Blood Unit Recipients with Myeloid Malignancies **Type:** DRUG #### Intervention 3 **Description:** Hematopoietic progenitor cells (HPCs) will be infused on day 0. Source of cells may be bone marrow, peripheral blood cells, or cord blood units, from matched related or unrelated donors. **Name:** Hematopoietic Progenitor Cell Transplantation (HPCT) **Type:** OTHER #### Intervention 4 **Description:** Patients with prior CNS disease over the age of 1 year will be treated with 600 cGy of cranial irradiation in addition to 1200 cGy of TBI. Patients diagnosed with ALL with CNS disease (at the time of diagnosis or relapse) \< 1 year of age will receive CNS treatment as Intrathecal Methotrexate as follows: * Infants ≤ 1 year of age at the time of Intrathecal Therapy will receive a dosing of 7.5 mg once a month for 6 months after transplant beginning at day +30 with an adequate white count * Children 1-2 years of age at the time of Intrathecal Therapy will receive 8 mg once a month for 6 months after transplant beginning at day +30 with an adequate white count **Name:** CNS radiation treatment for ALL with prior CNS disease patients **Type:** RADIATION ### Outcomes Module #### Primary Outcomes **Measure:** Evaluate the morbidity and mortality of hematopoietic progenitor cell transplantation (HPCT) at Children's Memorial Hospital. **Time Frame:** To study end #### Secondary Outcomes **Measure:** Evaluate the effectiveness of graft versus host disease prevention with a combination of anti-thymocyte globulin, continuous infusion cyclosporine, and short course methotrexate for transplants. **Time Frame:** To study end **Measure:** Determine the toxicity of a single conditioning regimen consisting of total body irradiation, etoposide, and Cyclophosphamide for patients with transplant eligible lymphoid malignant conditions or myeloid malignant conditions receiving cord blood units. **Time Frame:** To study end **Measure:** Determine the toxicity of a single conditioning regimen consisting of Busulfan and Cyclophosphamide for patients with transplant eligible myeloid malignant conditions who are not receiving cord blood units. **Time Frame:** To study end ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Malignant Disease * Chronic myleogenous leukemia in chronic or accelerated phase * Acute lymphoblastic leukemia (ALL) * First remission high-risk ALL (Ph+, t( 4-11) infants). * Second remission ALL, after a short first remission (\<36 mos from Dx). * 3rd or greater remission ALL. * Acute myelogenous leukemia (AML) * First remission high risk acute nonlymphoblastic (ANLL) (as defined by cytogenetics), if a matched sibling donor is available. * Initial partial remission AML (\<20% blasts in the bone marrow). * AML that is refractory to two cycles of induction therapy. * Second or greater remission AML * Myelodysplastic/Myeloproliferative Disease * Juvenile Myelomonocytic Leukemia (JMML) * Myelosplastic syndrome and/or pre-leukemia at any stage * Lymphoma * Relapsed lymphoma with residual disease that appears to be chemo-sensitive and non-bulky (\<5 cm at largest diameter) * Venous Access: Three lumens of central vascular access will be required for all patients entered on protocol due to the need for a dedicated line for continuous infusion cyclosporine. * Informed Consent: The patient and/or the patient's legally authorized guardian must acknowledge in writing that consent to become a study subject has been obtained in accordance with the institutional policies approved by the U.S. Department of Health and Human Services. * Patient organ function requirements: * Adequate renal function: Serum Creatinine \<\~1.5 x normal, or Creatinine clearance of 70 mL/min/1.73 mE2 or an equivalent GFR as determined by the institutional normal range * Adequate liver function: Total bilirubin \<1.5 x normal; and SGOT (AST) or SGPT (ALT) \<\~2.5 x normal * Adequate cardiac function: Shortening fraction of \>/=27% by echocardiogram * Adequate pulmonary function: FEV1/FVC \>/=60% by pulmonary function test; for children who are uncooperative, no evidence of dysnpea at rest, or exercise intolerance, and must have a pulse oximetry \>94% in room air * Performance status: Lansky for children \</= 16 years \>/= 60; Karnofsky status for those \> 16 years of age \>/= 70 * Effective Contraceptive Use: Women of childbearing potential and sexually active males should use effective contraception while on study. Exclusion Criteria: * Patients who are pregnant or lactating * Inability to find a suitable donor for the patient * Patient is HIV-positive * Patient has active Hepatitis B * Disease progression or relapse prior to HPC infusion **Maximum Age:** 21 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Morris Kletzel, MD **Phone:** 773-880-4562 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Terriss Conterato **Phone:** 773-880-8153 **Role:** CONTACT #### Locations **Location 1:** **City:** Chicago **Contacts:** ***Contact 1:*** - **Name:** Morris Kletzel, MD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Children's Memorial Hospital **State:** Illinois **Status:** RECRUITING **Zip:** 60614 #### Overall Officials **Official 1:** **Affiliation:** Ann & Robert H Lurie Children's Hospital of Chicago **Name:** Morris Kletzel, 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: D000006402 - Term: Hematologic Diseases - ID: D000007945 - Term: Leukemia, Lymphoid - 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 ### 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: 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: M16355 - Name: Syndrome - Relevance: LOW - As Found: Unknown - ID: M11220 - Name: Lymphoma - Relevance: HIGH - As Found: Lymphoma, Malignant - ID: M9189 - Name: Graft vs Host Disease - Relevance: LOW - As Found: Unknown - ID: M14164 - Name: Preleukemia - Relevance: LOW - As Found: Unknown - ID: M12145 - Name: Myelodysplastic Syndromes - Relevance: LOW - As Found: Unknown - ID: M27706 - Name: Leukemia, Myelomonocytic, Juvenile - Relevance: LOW - As Found: Unknown - ID: M10951 - Name: Leukemia, Lymphoid - Relevance: LOW - As Found: Unknown - ID: M27585 - Name: Precursor Cell Lymphoblastic Leukemia-Lymphoma - Relevance: HIGH - As Found: Leukemia, Lymphoblastic, Acute - ID: M10955 - Name: Leukemia, Myeloid - Relevance: HIGH - As Found: Leukemia, Myelogenous - ID: M18123 - Name: Leukemia, Myelogenous, Chronic, BCR-ABL Positive - Relevance: HIGH - As Found: Leukemia, Myelogenous, Chronic - ID: M18127 - Name: Leukemia, Myeloid, Acute - Relevance: HIGH - As Found: Leukemia, Myelogenous, Acute - 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: 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: T1303 - Name: Chronic Graft Versus Host Disease - Relevance: LOW - As Found: Unknown - ID: T3543 - Name: Lymphosarcoma - Relevance: HIGH - As Found: Lymphoma, Malignant - ID: T2832 - Name: Homologous Wasting Disease - Relevance: LOW - As Found: Unknown - ID: T170 - Name: Acute Graft Versus Host Disease - Relevance: LOW - As Found: Unknown - ID: T3993 - Name: Myelodysplastic Syndromes - 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: T175 - Name: Acute Lymphoblastic Leukemia - Relevance: HIGH - As Found: Leukemia, Lymphoblastic, Acute - ID: T3533 - Name: Lymphoblastic Lymphoma - Relevance: HIGH - As Found: Leukemia, Lymphoblastic, Acute - ID: T1311 - Name: Chronic Myeloproliferative Disorders - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000008223 - Term: Lymphoma - ID: D000007938 - Term: Leukemia - ID: D000054198 - Term: Precursor Cell Lymphoblastic Leukemia-Lymphoma - ID: D000007951 - Term: Leukemia, Myeloid - ID: D000015464 - Term: Leukemia, Myelogenous, Chronic, BCR-ABL Positive - ID: D000015470 - Term: Leukemia, Myeloid, Acute ### Intervention Browse Module - Browse Branches - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: ARhu - Name: Antirheumatic Agents - Abbrev: Derm - Name: Dermatologic Agents - Abbrev: Repr - Name: Reproductive Control Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: AntiConv - Name: Anticonvulsants - Abbrev: AnEm - Name: Antiemetics - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: PsychDr - Name: Psychotropic Drugs - Abbrev: Gast - Name: Gastrointestinal Agents ### Intervention Browse Module - Browse Leaves - ID: M11703 - Name: Methotrexate - Relevance: LOW - As Found: Unknown - ID: M6727 - Name: Cyclophosphamide - Relevance: LOW - As Found: Unknown - ID: M8191 - Name: Etoposide - Relevance: LOW - As Found: Unknown - ID: M341643 - Name: Etoposide phosphate - Relevance: LOW - As Found: Unknown - ID: M5336 - Name: Busulfan - Relevance: LOW - As Found: Unknown - ID: M11140 - Name: Lorazepam - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT04700579 **Acronym:** TB-LUNG **Brief Title:** Pre- and Post-treatment Lung Microbiota, Metabolome and Immune Signatures at the Site of Disease in Patients With Active Pulmonary Tuberculosis **Official Title:** Pre- and Post-treatment Lung Microbiota, Metabolome and Immune Signatures at the Site of Disease in Patients With Active Pulmonary Tuberculosis #### Organization Study ID Info **ID:** N19/09/126 #### Organization **Class:** OTHER **Full Name:** University of Stellenbosch ### Status Module #### Completion Date **Date:** 2026-01 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-03-28 **Type:** ACTUAL **Last Update Submit Date:** 2024-03-27 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-12-31 **Type:** ESTIMATED #### Start Date **Date:** 2021-03-04 **Type:** ACTUAL **Status Verified Date:** 2024-03 #### Study First Post Date **Date:** 2021-01-08 **Type:** ACTUAL **Study First Submit Date:** 2020-12-28 **Study First Submit QC Date:** 2021-01-06 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** New York University #### Lead Sponsor **Class:** OTHER **Name:** University of Stellenbosch #### Responsible Party **Investigator Affiliation:** University of Stellenbosch **Investigator Full Name:** Grant Theron **Investigator Title:** 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 diverse microbial communities in different parts of the human body (microbiome) are important for health but understudied in pulmonary tuberculosis (TB), which is the single biggest infectious cause of death in the world. The investigators will study the site-of-disease microbiome (in the lung bronchoalveolar space) in TB cases to investigate how, before TB treatment, metabolic compounds made by microbes affect host biomarkers important for TB control. The investigators will ask this question again at the end-of-treatment and one year later. Specifically, the investigators will sample the lung at the active TB hotspot identified by imaging and compare this to a non-involved lung segment usually in the opposite lung. The investigators will compare the lung microbiome to other sites in the body (i.e. oral cavity, nasopharynx, supraglottis, and gut). A small amount of blood (\~15 ml) will be collected to assess peripheral immunological correlates of the host microbiome. Protected specimen brushings of the lung will be used to explore transcriptomic signatures and how these relate to the lung microbiome. The investigators will also apply these questions to the same number of controls (healthy patients and patients with an alternative diagnoses). This will lay the foundation for clinical trials to evaluate if specific bacteria have diagnostic (e.g., PCR) or therapeutic potential (e.g., antibiotics, prebiotics, probiotics, vaccines) where targeting the microbiome could improve clinical outcomes. **Detailed Description:** The human body is host to complex microbial communities at different anatomical sites such as the gut, oral cavity, vagina, skin, and the lower respiratory tract - a site previously thought to be sterile. Growing evidence has implicated the role of the human microbiome in various diseases for example, Prevotella-enriched lung communities in HIV-positive pneumonia patients independently predict 70-day mortality, and Lactobacillus enriched murine gut microbiome alleviates asthma-like symptoms. However, despite the scale and severity of TB, there are limited studies on the microbiome in TB cases, the site of disease, and the effect of treatment, especially in the context of HIV. These key knowledge gaps preclude the design and evaluation of interventions that could target the microbiome and avert poor treatment outcomes in TB. To date the few microbiome studies in TB have focused on the upper respiratory tract (using specimens such as sputum) and gut rather than the site of disease which, in TB, is typically the lung. These studies have shown associations between the microbiome and state of disease. For example, mice colonized with Helicobacter hepaticus in the gut demonstrate poor control of mycobacterial growth, heightened inflammation, and severe tissue pathology in the lungs. The lung which is the site of disease in pulmonary TB has been widely considered sterile until recently and the lung microbiome remains widely understudied in TB regardless of the potential impact it might have in TB pathogenesis. One of the major reasons why the lung is understudied is the difficult in sampling the lung. The investigators will implement a modified bronchoscopy procedure to avoid microbial cross-contamination from neighbouring anatomical sites (including from diseased to healthy parts of the lungs) and to accurately sample the low biomass in the bronchoalveolar space. The investigators hypothesize that TB cases have a distinct site-of-disease lung microbiota compared to non-diseased contralateral tissue, characterized by an enrichment of oral anaerobic fermenters, SCFAs, and impaired inflammation and tissue repair biomarkers. They also expect microbial and host biomarkers to be altered by TB treatment. A study by one of the investigators has already demonstrated lung microbiomes enriched with anaerobic oral taxa are associated with lung inflammation of the Th17 phenotype. The products of microbial anaerobic metabolism have also been shown to modulate immune response to diseases. The investigators will correlate the complex microbial communities at the site-of-disease in TB with the microbial and host biomarkers at the site-of-disease. The study will recruit self-reporting patients with their first TB episode and Xpert MTB/RIF Ultra-confirmed TB from Scottsdene and Wallacedene primary care clinics in Cape Town. A total of 50 TB cases equally stratified by HIV status and 50 healthy household contacts (HHC) also stratified by HIV will be recruited. In addition to HIV-negatives, the study is recruiting an equal number of ART-treated HIV-positive TB cases, because there an epidemiologically important subpopulation with impaired pulmonary immunity. An additional 50 sick controls with other pulmonary diseases (Asthma, Chronic obstructive pulmonary disease (COPD), Cancer, Bronchiectasis (including post-TB) and Pneumonia) will recruited as comparator groups. ### Conditions Module **Conditions:** - TB - Tuberculosis - HIV/AIDS **Keywords:** - Microbiome - Short Chain Fatty Acids - Immunological Biomarkers ### Design Module #### Bio Spec **Description:** Bronchoalveolar lavage fluid, Protected specimen brushings, Sputum, Oro- Nasopharyngeal swabs, Urine, Blood and Stool. **Retention:** SAMPLES_WITH_DNA #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 150 **Type:** ESTIMATED **Patient Registry:** True **Study Type:** OBSERVATIONAL **Target Duration:** 18 Months ### Arms Interventions Module #### Arm Group 1 **Description:** * n= 50 (25 HIV positive and 25 HIV negative) * Xpert MTB/RIF Ultra-confirmed TB **Label:** TB Cases #### Arm Group 2 **Description:** * n= (25 HIV positive and 25 HIV negative) * Culture negative TB result **Label:** Healthy Household Contacts #### Arm Group 3 **Description:** * n=50 * Diseases: Asthma, Chronic obstructive pulmonary disease (COPD), Cancer, Bronchiectasis (including post-TB) and Pneumonia **Label:** Sick controls ### Outcomes Module #### Primary Outcomes **Description:** Lung microbiome in diseased and non-diseased segments determined by 16S rRNA gene sequencing. **Measure:** Characterization of changes in microbiota in diseased vs. non-diseased lung segments, stratified by HIV status. **Time Frame:** Up to 18 months **Description:** Correlation analysis of specific cytokines profiled using commercial multiplexed Luminex panels and SCFAs measured using Gas chromatography-mass spectrometry (GC-MS) assays. **Measure:** Association of specific microbial taxa in diseased segments with elevated SCFAs and impaired host inflammation and tissue repair biomarkers. **Time Frame:** Up to 18 months **Description:** Characterize bacterial community resilience alongside changes in microbial and host biomarkers. **Measure:** Evaluate the impact of treatment on the lung microbiome. **Time Frame:** Up to 18 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * 18-60 years old. * Agree to undergo CXR and/or CT scan. * Has unilateral TB disease defined as one lung with extensive evidence of TB disease (non-applicable to healthy controls; sick controls will require an alternative diagnosis). * No evidence of prior TB treatment and/or CXR/CT does not have obvious evidence of prior TB. * Willing to undergo a research bronchoscopy at baseline, 6 months and 18 months and likely to remain in the area for the study period. * If HIV-positive, must be stable on antiretroviral therapy (ART) for ≥1 year. * Able and willing to return for follow-up visits, with no plans to move in the near future. * Willing to comply with study requirements i.e. provision of contact details and written, informed consent prior to enrolment. Exclusion Criteria: * Less than 18 years or older than 60 years of age. * Has already initiated TB treatment. * Rifampicin resistant. * Has a previous history of TB. * Bilateral TB disease defined as both lungs with extensive TB disease * Has received probiotics, antibiotics or inhaled steroids within three months prior to enrolment (not applicable to sick controls) * Has diabetes mellitus, which affects TB disease, treatment response, and the microbiome * Has a contraindication for bronchoscopy (e.g., FEV1 \<70%), as determined by bronchoscopists according to best practice guidelines * Has a daily alcohol intake of more than 6 beers or 4 mixed drinks * Is pregnant (a commercial human chorionic gonadotropin determination assay will be performed in accordance with manufacturer's guidance on urine) or pregnancy planned for follow-up period * Recent hospitalization for any reason **Healthy Volunteers:** True **Maximum Age:** 60 Years **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT **Study Population:** Self-presenting patients with Xpert MTB/RIF Ultra-confirmed TB (n=50, equally stratified by HIV) and culture negative HHCs (n=50, equally stratified by HIV) will be recruited from primary care clinics (Scottsdene and Wallacedene) in Cape Town, South Africa. An additional 50 sick controls with other lung diseases will be referred from Tygerberg Academic Hospital. ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Grant Theron, PhD **Phone:** (+27) 021 938 9693 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Charissa Naidoo, PhD **Phone:** (+27) 0219389955 **Role:** CONTACT #### Locations **Location 1:** **City:** Cape Town **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Grant Theron, PhD - **Phone:** (+27) 21 938 9693 - **Phone Ext:** 9693 - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Byron WP Reeve, PhD - **Phone:** (+27) 21 938 9954 - **Phone Ext:** 9954 - **Role:** CONTACT **Country:** South Africa **Facility:** Kraaifontein Community Health Centre **State:** Western Cape **Status:** RECRUITING **Zip:** 7570 **Location 2:** **City:** Cape Town **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Grant Theron, PhD - **Phone:** (+27) 21 938 9693 - **Phone Ext:** 9693 - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Byron WP Reeve, PhD - **Phone:** (+27) 21 938 9954 - **Phone Ext:** 9954 - **Role:** CONTACT **Country:** South Africa **Facility:** Scottsdene Clinic **State:** Western Cape **Status:** RECRUITING **Zip:** 7570 **Location 3:** **City:** Cape Town **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Grant Theron, PhD - **Phone:** (+27) 21 938 9693 - **Phone Ext:** 9693 - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Byron WP Reeve, PhD - **Phone:** (+27) 21 938 9954 - **Phone Ext:** 9954 - **Role:** CONTACT **Country:** South Africa **Facility:** Wallacedene Clinic **State:** Western Cape **Status:** RECRUITING **Zip:** 7570 #### Overall Officials **Official 1:** **Affiliation:** University of Stellenbosch **Name:** Grant Theron, PhD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Access Criteria:** Data access will be granted to interested collaborators and parties through sharing of data files or login information for the study's secure RedCap electronic database during the course of patient recruitment. **Description:** Collaborators will have access to de-identified IPD data. Other researchers wishing to access the data will be required to sign a data sharing agreement as per institutional policies. **Info Types:** - STUDY_PROTOCOL - SAP - ICF **IPD Sharing:** YES **Time Frame:** The data will be available once the findings are published. ### References Module #### References **Citation:** Yatsunenko T, Rey FE, Manary MJ, Trehan I, Dominguez-Bello MG, Contreras M, Magris M, Hidalgo G, Baldassano RN, Anokhin AP, Heath AC, Warner B, Reeder J, Kuczynski J, Caporaso JG, Lozupone CA, Lauber C, Clemente JC, Knights D, Knight R, Gordon JI. Human gut microbiome viewed across age and geography. Nature. 2012 May 9;486(7402):222-7. doi: 10.1038/nature11053. **PMID:** 22699611 **Citation:** Wade WG. The oral microbiome in health and disease. Pharmacol Res. 2013 Mar;69(1):137-43. doi: 10.1016/j.phrs.2012.11.006. Epub 2012 Nov 28. **PMID:** 23201354 **Citation:** Weiner J 3rd, Parida SK, Maertzdorf J, Black GF, Repsilber D, Telaar A, Mohney RP, Arndt-Sullivan C, Ganoza CA, Fae KC, Walzl G, Kaufmann SH. Biomarkers of inflammation, immunosuppression and stress with active disease are revealed by metabolomic profiling of tuberculosis patients. PLoS One. 2012;7(7):e40221. doi: 10.1371/journal.pone.0040221. Epub 2012 Jul 23. Erratum In: PLoS One. 2016;11(3):e0153050. **PMID:** 22844400 **Citation:** Grice EA, Kong HH, Renaud G, Young AC; NISC Comparative Sequencing Program; Bouffard GG, Blakesley RW, Wolfsberg TG, Turner ML, Segre JA. A diversity profile of the human skin microbiota. Genome Res. 2008 Jul;18(7):1043-50. doi: 10.1101/gr.075549.107. Epub 2008 May 23. **PMID:** 18502944 **Citation:** Shenoy MK, Iwai S, Lin DL, Worodria W, Ayakaka I, Byanyima P, Kaswabuli S, Fong S, Stone S, Chang E, Davis JL, Faruqi AA, Segal MR, Huang L, Lynch SV. Immune Response and Mortality Risk Relate to Distinct Lung Microbiomes in Patients with HIV and Pneumonia. Am J Respir Crit Care Med. 2017 Jan 1;195(1):104-114. doi: 10.1164/rccm.201603-0523OC. **PMID:** 27447987 **Citation:** Wipperman MF, Fitzgerald DW, Juste MAJ, Taur Y, Namasivayam S, Sher A, Bean JM, Bucci V, Glickman MS. Antibiotic treatment for Tuberculosis induces a profound dysbiosis of the microbiome that persists long after therapy is completed. Sci Rep. 2017 Sep 7;7(1):10767. doi: 10.1038/s41598-017-10346-6. **PMID:** 28883399 **Citation:** Botero LE, Delgado-Serrano L, Cepeda ML, Bustos JR, Anzola JM, Del Portillo P, Robledo J, Zambrano MM. Respiratory tract clinical sample selection for microbiota analysis in patients with pulmonary tuberculosis. Microbiome. 2014 Aug 25;2:29. doi: 10.1186/2049-2618-2-29. eCollection 2014. **PMID:** 25225609 **Citation:** Cheung MK, Lam WY, Fung WY, Law PT, Au CH, Nong W, Kam KM, Kwan HS, Tsui SK. Sputum microbiota in tuberculosis as revealed by 16S rRNA pyrosequencing. PLoS One. 2013;8(1):e54574. doi: 10.1371/journal.pone.0054574. Epub 2013 Jan 24. **PMID:** 23365674 **Citation:** Cui Z, Zhou Y, Li H, Zhang Y, Zhang S, Tang S, Guo X. Complex sputum microbial composition in patients with pulmonary tuberculosis. BMC Microbiol. 2012 Nov 23;12:276. doi: 10.1186/1471-2180-12-276. **PMID:** 23176186 **Citation:** Segal LN, Clemente JC, Tsay JC, Koralov SB, Keller BC, Wu BG, Li Y, Shen N, Ghedin E, Morris A, Diaz P, Huang L, Wikoff WR, Ubeda C, Artacho A, Rom WN, Sterman DH, Collman RG, Blaser MJ, Weiden MD. Enrichment of the lung microbiome with oral taxa is associated with lung inflammation of a Th17 phenotype. Nat Microbiol. 2016 Apr 4;1:16031. doi: 10.1038/nmicrobiol.2016.31. **PMID:** 27572644 **Citation:** Segal LN, Alekseyenko AV, Clemente JC, Kulkarni R, Wu B, Gao Z, Chen H, Berger KI, Goldring RM, Rom WN, Blaser MJ, Weiden MD. Enrichment of lung microbiome with supraglottic taxa is associated with increased pulmonary inflammation. Microbiome. 2013 Jul 1;1(1):19. doi: 10.1186/2049-2618-1-19. Erratum In: Microbiome. 2014;2:21. Gao, Zhan [added]. **PMID:** 24450871 **Citation:** Naidoo CC, Nyawo GR, Wu BG, Walzl G, Warren RM, Segal LN, Theron G. The microbiome and tuberculosis: state of the art, potential applications, and defining the clinical research agenda. Lancet Respir Med. 2019 Oct;7(10):892-906. doi: 10.1016/S2213-2600(18)30501-0. Epub 2019 Mar 22. **PMID:** 30910543 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009164 - Term: Mycobacterium Infections - ID: D000000193 - Term: Actinomycetales Infections - ID: D000016908 - Term: Gram-Positive Bacterial Infections - ID: D000001424 - Term: Bacterial Infections - ID: D000001423 - Term: Bacterial Infections and Mycoses - ID: D000007239 - Term: Infections - ID: D000012141 - Term: Respiratory Tract Infections - ID: D000008171 - Term: Lung Diseases - ID: D000012140 - Term: Respiratory Tract Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC01 - Name: Infections - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: BC20 - Name: Immune System Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M3522 - Name: Acquired Immunodeficiency Syndrome - Relevance: LOW - As Found: Unknown - ID: M18250 - Name: HIV Infections - Relevance: LOW - As Found: Unknown - ID: M17127 - Name: Tuberculosis - Relevance: HIGH - As Found: Tuberculosis - ID: M17147 - Name: Tuberculosis, Pulmonary - Relevance: HIGH - As Found: Pulmonary Tuberculosis - ID: M10283 - Name: Infections - Relevance: LOW - As Found: Unknown - ID: M6368 - Name: Communicable Diseases - Relevance: LOW - As Found: Unknown - ID: M12119 - Name: Mycobacterium Infections - 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 - ID: M14978 - Name: Respiratory Tract Infections - Relevance: LOW - As Found: Unknown - ID: M11168 - Name: Lung Diseases - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000014376 - Term: Tuberculosis - ID: D000014397 - Term: Tuberculosis, Pulmonary ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05151679 **Brief Title:** Chelated Oral Iron Versus Intravenous Iron Sucrose for Treatment of Iron Deficiency Anemia Late in Pregnancy **Official Title:** Chelated Oral Iron Versus Intravenous Iron Sucrose for Treatment of Iron Deficiency Anemia Late in Pregnancy ( Randomized Controlled Trial ) #### Organization Study ID Info **ID:** oral iron versus I.V iron #### Organization **Class:** OTHER **Full Name:** Assiut University ### Status Module #### Expanded Access Info #### Last Update Post Date **Date:** 2021-12-09 **Type:** ACTUAL **Last Update Submit Date:** 2021-11-28 **Overall Status:** AVAILABLE **Status Verified Date:** 2021-11 #### Study First Post Date **Date:** 2021-12-09 **Type:** ACTUAL **Study First Submit Date:** 2021-11-28 **Study First Submit QC Date:** 2021-11-28 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Assiut University #### Responsible Party **Investigator Affiliation:** Assiut University **Investigator Full Name:** Mahmoud Shaban Mohammed Farghaly **Investigator Title:** resident doctor **Type:** PRINCIPAL_INVESTIGATOR ### Description Module **Brief Summary:** compare the efficacy and safety of intravenous iron sucrose versus chelated oral iron in the treatment of iron deficiency anemia late in pregnancy. **Detailed Description:** patients will be recruited from the pregnant women who will attend the outpatient clinic at Women's Health Hospital Assuit University with the diagnosis of iron deficiency anaemia between 26-34 weeks with Hb level 8-10 in the initial visit the patients will be counselled about the iron therapy, its benefits and risk of discontinuation written consent will be obtained. All women will be subjected to the following: 1. History taking 1. Personal history: demographic factors, age and dietary habits. 2. Obstetric history: parity, EDD, last delivery, gestational age determined from last menstrual period or previous early ultrasound scanning report. 3. Past history: history of chronic diseases such as diabetes or hypertension, blood transfusion and either oral or i.v iron treatment. 2. Clinical examination General examination including pulse, blood pressure, BMI, pallor, temperature and auscultation of lungs and heart. Abdominal examination for fundal level. The patients of the study will be randomly divided into two groups Group 1 will be treated by I.V ferrous sucrose Group 2 will be treated by chelated oral iron 3. Invetigations * Ultrasonography in the initial visit to assess fetal well being. * Blood sample will be taken at recruitment on day 0, day 14, day 28, day 40 of therapy.these time points were chosen on the basis of previous studies and to minimize inconvenience to women. The time points were also chosen to detect any difference in the speed of restoration of Hb% and iron stores. The blood sample will be examined for the following: 1. Complete blood picture. A sample of 2 ml venous blood will be collected in EDTA vacutainers. 2. Serum ferritin evaluation. A sample of 2 ml venous blood will be collected in EDTA vacutainers and separated sera will be stored at 20 C until the time of assay by turbidimetric technique. 3. Total iron binding capacity. A sample of 2 ml venous blood will be collected in EDTA vacutainers 4. Doses and administration Treatment will be started 24rh after initial visit women will be randomized used random sequence computer generated list in such a way that every patient had equal chance to be among any of the two groups either to group 1 , where they will receive I.V ferrous sucrose in the E.U in Women's Health Hospital Assuit University as an i.v infusion in 250 ml 0.9% sodium chloride slowly over 30 minutes, and then will be discontinued for another 30 minutes to detect any hypersensitivity reactions with monitoring of vital signs during infusion, antishock measures will be prepared beside the patient during administration ( corticosteroids, antihistaminic, calcium and oxygen ). Patients will be asked to note any symptoms or adverse effects of treatment such as facial flushing nausea, metallic taste, dyspepsia, and burning at the site of injection. The dose in mg will be calculated from the following formula: 2.4 × weight × ( target - actual Hb ) I g/dl + 500 Target Hb 12 g% The total required dose of iron will be divided into three doses, which will be given every 3 days, and the maximum daily dose is 200mg ( 2 ampoules ). This group will not receive further iron supplementation. They will be asked to note any symptoms or adverse effects of treatment. or to group 2 where they will receive chelated oral iron, they will be given iron chelated amino acid containing 15 mg of elemental iron once daily for 6 weeks. the women will be advised to take 1 tab of iron chelated amino acid containing 15mg of elemental iron once daily with meals for 6 weeks from the day of recruitment. Date will be given when to stop oral supplementation after 6 weeks. This group will be advised to note side effects such as nausea, metallic taste, dyspepsia and constipation. We will add 500µg of folic acid to patients of group 1 once daily for 6 weeks to eliminate the differences in the results between the groups as ferrotron ( chelated oral iron for second group ) contains 400µg folic acid. ### Conditions Module **Conditions:** - Iron Deficiency Anemia of Pregnancy ### Design Module #### Expanded Access Types **Individual:** True **Study Type:** EXPANDED_ACCESS ### Arms Interventions Module ### Interventions #### Intervention 1 **Description:** Treatment will be started 24rh after initial visit women will be randomized used random sequence computer generated list in such a way that every patient had equal chance to be among any of the two groups either to group 1 , where they will receive I.V ferrous sucrose in the E.U in Women's Health Hospital Assuit University as an i.v infusion in 250 ml 0.9% sodium chloride slowly over 30 minutes. The total required dose of iron will be divided into three doses, which will be given every 3 days, and the maximum daily dose is 200mg ( 2 ampoules ). **Name:** Chelated Iron **Other Names:** - ferrotron **Type:** DRUG #### Intervention 2 **Description:** to group 2 where they will receive chelated oral iron, they will be given iron chelated amino acid containing 15 mg of elemental iron once daily for 6 weeks. the women will be advised to take 1 tab of iron chelated amino acid containing 15mg of elemental iron once daily with meals for 6 weeks from the day of recruitment. **Name:** Iron sucrose **Other Names:** - sacrofer **Type:** DRUG ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Women aged 18 years or more with established iron deficiency anemia ( Hb: 8-10 ) 2. Pregnancy 26-34 weeks Exclusion Criteria: 1. Anemia due to other causes than iron deficiency anemia. 2. Recent blood transfusion (in the last 3 months ). 3. Allergy to iron. 4. Multiple pregnancy. **Healthy Volunteers:** True **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** mahmoud farghaly, resident **Phone:** +201005450572 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** mohammed sayed, prof **Phone:** +201006801036 **Role:** CONTACT ### References Module #### References **Citation:** Bencaiova G, von Mandach U, Zimmermann R. Iron prophylaxis in pregnancy: intravenous route versus oral route. Eur J Obstet Gynecol Reprod Biol. 2009 Jun;144(2):135-9. doi: 10.1016/j.ejogrb.2009.03.006. Epub 2009 Apr 29. **PMID:** 19406557 **Citation:** Means RT. Iron Deficiency and Iron Deficiency Anemia: Implications and Impact in Pregnancy, Fetal Development, and Early Childhood Parameters. Nutrients. 2020 Feb 11;12(2):447. doi: 10.3390/nu12020447. **PMID:** 32053933 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000006402 - Term: Hematologic Diseases - ID: D000019189 - Term: Iron Metabolism Disorders - ID: D000008659 - Term: Metabolic Diseases - ID: D000000747 - Term: Anemia, Hypochromic - ID: D000044342 - Term: Malnutrition - ID: D000009748 - Term: Nutrition Disorders ### Condition Browse Module - Browse Branches - Abbrev: BC15 - Name: Blood and Lymph Conditions - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M20857 - Name: Anemia, Iron-Deficiency - Relevance: HIGH - As Found: Iron Deficiency Anemia - ID: M2781 - Name: Iron Deficiencies - Relevance: HIGH - As Found: Iron Deficiency - ID: M4070 - Name: Anemia - Relevance: HIGH - As Found: Anemia - ID: M6879 - Name: Deficiency Diseases - Relevance: HIGH - As Found: Deficiency Anemia - ID: M9490 - Name: Hematologic Diseases - Relevance: LOW - As Found: Unknown - ID: M11639 - Name: Metabolic Diseases - Relevance: LOW - As Found: Unknown - ID: M21177 - Name: Iron Metabolism Disorders - Relevance: LOW - As Found: Unknown - ID: M4077 - Name: Anemia, Hypochromic - Relevance: LOW - As Found: Unknown - ID: M25306 - Name: Malnutrition - Relevance: LOW - As Found: Unknown - ID: M12684 - Name: Nutrition Disorders - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000000740 - Term: Anemia - ID: D000018798 - Term: Anemia, Iron-Deficiency - ID: D000090463 - Term: Iron Deficiencies - ID: D000003677 - Term: Deficiency Diseases ### Intervention Browse Module - Ancestors - ID: D000014131 - Term: Trace Elements - ID: D000018977 - Term: Micronutrients - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000006397 - Term: Hematinics - ID: D000002614 - Term: Chelating Agents - ID: D000064449 - Term: Sequestering Agents - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action ### Intervention Browse Module - Browse Branches - Abbrev: Micro - Name: Micronutrients - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Hemat - Name: Hematinics ### Intervention Browse Module - Browse Leaves - ID: M10533 - Name: Iron - Relevance: HIGH - As Found: Autologous - ID: M1861 - Name: Ferric Oxide, Saccharated - Relevance: HIGH - As Found: HIV Prevention - ID: M10534 - Name: Iron Chelating Agents - Relevance: HIGH - As Found: Forodesine - ID: M5860 - Name: Chelating Agents - 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: M9485 - Name: Hematinics - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000007501 - Term: Iron - ID: D000077605 - Term: Ferric Oxide, Saccharated - ID: D000007502 - Term: Iron Chelating Agents ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05253079 **Brief Title:** Erector Spinae Plane Block Versus Subcostal Transversus Abdominis Plane Block in Open Liver Resection Surgery **Official Title:** Erector Spinae Plane Block Versus Subcostal Transversus Abdominis Plane Block in Patients Undergoing Open Liver Resection Surgery: A Randomized Controlled Trial #### Organization Study ID Info **ID:** MS-481-2021 #### Organization **Class:** OTHER **Full Name:** Kasr El Aini Hospital ### Status Module #### Completion Date **Date:** 2022-05 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2022-03-11 **Type:** ACTUAL **Last Update Submit Date:** 2022-03-10 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2022-05 **Type:** ESTIMATED #### Start Date **Date:** 2022-03-01 **Type:** ACTUAL **Status Verified Date:** 2022-03 #### Study First Post Date **Date:** 2022-02-23 **Type:** ACTUAL **Study First Submit Date:** 2022-02-14 **Study First Submit QC Date:** 2022-02-14 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Kasr El Aini Hospital #### Responsible Party **Investigator Affiliation:** Kasr El Aini Hospital **Investigator Full Name:** Maha Mostafa Ahmad, MD **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:** Liver resection surgery is a common surgical procedure which is performed on patients with benign, malignant or metastatic hepatic tumor as well as for living liver donor. Liver resection surgery is usually performed through either right subcostal or inversed L-shaped incision; both approaches are associated with a significant postoperative pain which requires intensive analgesic plan to facilitate early mobilization and minimize complications. There are various lines for pain management in liver resection surgery such as systemic analgesic drugs, neuraxial blocks (e.g., thoracic epidural analgesia) and transversus abdominis plane \[TAP\] block). Systemic analgesic drugs are nearly constantly used in liver resection. However, being systemically administered, these drugs have many side effects on many organs and cannot totally eliminate postoperative pain. Thoracic epidural block is commonly associated with hypotension; furthermore, its use has other limitations such as delaying postoperative mobilization and possible hematoma and cord compression in patients with coagulopathy which is expected following liver resection. Therefore, there had been an increased interest in the use of abdominal field blocks to avoid disadvantages of neuraxial blocks and minimize the use of parenteral analgesic drugs. TAP block is one of the classic field blocks which is extensively used in laparotomies including liver resection. However, the lack of visceral pain control TAP block influences the quality of its analgesic effect in this type of patients. Nevertheless, TAP block, namely the subcostal approach, is still the recommended field block in the latest procedure-specific recommendations for pain management in liver resection as it is the only block which showed good evidence. In recent years, there has been increased interest in a newer field block, the erector spinae plane block (ESPB), due its easy performance and the possible coverage of visceral pain in addition to the somatic pain. ESPB showed promising results in liver resection surgery. ESBP was superior to TAP block in various abdominal surgeries. However, its analgesic efficacy had not been previously compared in relation to TAP in patients undergoing open liver resection surgery. **Detailed Description:** An independent research assistant will be responsible for opening the envelope and drug preparation with no further involvement in the study. The local anesthetic solution preparation will be as follow; 2 syringes of 20 ml of 0.25% isobaric bupivacaine. Upon arrival to the operating room, routine monitors (electrocardiogram, pulse oximetry, and non-invasive blood pressure monitor) will be applied; intravenous line will be secured, and pre-medication drugs will be delivered (metoclopramide 10 mg, and omeprazole 40 mg). General anesthesia will be induced by 2-3 mg/kg propofol and 1-2 mcg/kg fentanyl. Tracheal intubation by direct laryngoscopy will be facilitated by atracurium 0.5 mg/kg. Anesthesia will be maintained by 2-2.5% sevoflurane and 0.1 mg/kg/20min atracurium. After induction of anesthesia, patients will receive their assigned intervention. Intraoperative analgesic management Morphine boluses (titrated 0.05 mg/kg boluses till response) will be given in case of inadequate analgesia (heart rate/mean blood pressure increase by 20% from the baseline) Intraoperative fluid and hemodynamic management will be according to the discretion of the attending anesthetist. At the end of the surgery, all patients will receive intravenous paracetamol (1 g) and ketorolac (30 mg) before the extubation. Postoperative care All patients will receive regular intravenous paracetamol 1 g/6hours and ketorolac 30 mg/8hours. Pain assessments using Numerical Rating Scale (NRS) will be performed at rest and during cough at 0.5, 1, 2, 4, 6, 18, 24 hours after leaving the operating room. If NRS score is \> 3 intravenous titration of 2 mg morphine is given slowly to be repeated after 30 minutes if pain persisted. If other opioids are given, morphine equivalent dose will be calculated using opioids conversion chart. Intravenous ondansetron 4 mg will be given to treat nausea or vomiting if occurs. the following data will be recorded Complications: hematoma, nausea, vomiting, itching, urine retention, constipation, Initial pathology, type and length of skin incision, duration of surgery, blood loss, need for blood transfusion, vasoactive drugs Age, sex, American society of anesthesiologist-physical status (ASA), comorbidity, weight, height and body mass index ### Conditions Module **Conditions:** - Analgesia - Hepatic Resection Surgery ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** TRIPLE **Who Masked:** - PARTICIPANT - CARE_PROVIDER - OUTCOMES_ASSESSOR **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 60 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** subcostal transversus abdominis plane block **Intervention Names:** - Other: subcostal transversus plane block **Label:** subcostal transversus abdominis plane block group **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** erector spinae plane block **Intervention Names:** - Other: erector spinae plane block **Label:** erector spinae plane block group **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - subcostal transversus abdominis plane block group **Description:** Patient in TAP block group will receive oblique subcostal TAP while in supine position using a linear 6-13 MHz ultrasound transducer. The operator will place the ultrasound probe obliquely on the upper abdominal wall along the subcostal margin near the xiphisternum of the sternum in the midline of the abdomen. The landmarks, which included the rectus abdominis muscle and underlying transversus abdominis muscle, will be identified near the costal margin and xyphoid. The probe will be then moved laterally until the aponeurosis of the external, internal oblique, and transversus abdominis are seen, and then the probe will be moved further laterally until the transversus abdominis muscle is identified. The operator will direct the needle toward the transversus abdominis, and the local anesthetic solution will be injected after negative aspiration between the rectus abdominis and transversus abdominis muscles along the subcostal line (in-plane superomedial to inferolateral) **Name:** subcostal transversus plane block **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - erector spinae plane block group **Description:** Patients in ESPB group will be turned to the lateral position and receive ESPB at the level of the 7th thoracic (T7) transverse process using a linear 6-13 MHz ultrasound transducer. The transducer will be positioned vertically 3 cm to the side of the midline to visualize the muscles of the back, the transverse process, and the pleura between the two transverse processes. Then, a 22G 10-mm needle will be introduced in the cranial-caudal direction toward the transverse process (T7) using the in-plane method till the needle tip crosses all the muscles. The tip of needle should be in the plane between the transverse process and the erector spinae muscle. After ensuring negative aspiration, the local anesthetic solution will be injected below the muscle. the procedure will be repeated on the other side **Name:** erector spinae plane block **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** mg **Measure:** total morphine requirement in the first 24 hours **Time Frame:** 0.5 hour after extubation till 24 hour after extubation #### Secondary Outcomes **Description:** mg **Measure:** intraoperative morphine requirement **Time Frame:** 15 minutes after block administration till 1 minutes after extubation **Description:** hours **Measure:** time to first morphine requirement **Time Frame:** 1 minute after block administration till 24 hours postoperatively **Description:** 11-points scale in which the patients are asked to circle the number between 0 and 10 that best describe their pain intensity. Zero represents 'no pain at all' whereas 10 represents 'the worst pain ever possible **Measure:** NRS **Time Frame:** 0.5, 1, 2, 4, 6, 18, 24 hours after leaving the operating room **Description:** time in hours of being able to be independently mobile e.g. using the bathroom **Measure:** Time to independent movement **Time Frame:** 0.5 hour after extubation till 24 hour after extubation **Description:** beat per minutes **Measure:** heart rate **Time Frame:** 15 minutes before induction of anesthesia, 15 minutes after induction of anesthesia, every 15 minutes intraoperatively and 0.5, 1, 2, 4, 6, 18, 24 hours postoperatively **Description:** mmHg **Measure:** mean arterial pressure **Time Frame:** 15 minutes before induction of anesthesia, 15 minutes after induction of anesthesia, every 15 minutes intraoperatively and 0.5, 1, 2, 4, 6, 18, 24 hours postoperatively **Description:** 1 = Awake and alert 2 = Slightly sedated, 3 = moderately sedated follows simple commands, 4 = deeply sedated, responds to nonpainful stimuli, 5 = deeply sedated, responds to painful stimuli, 6 = deeply sedated, unresponsive to painful stimuli **Measure:** Modified Ramsay Sedation Score **Time Frame:** 0.5, 1, 2, 4, 6, 18, 24 hours after leaving the operating room ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * adult patients (\>18 years), * ASA-physical status I-III * undergoing open liver resection surgery for either primary/metastatic hepatic malignancy, biliary tract malignancy, or benign hepatic tumor Exclusion Criteria: * history of allergy to any of the study drugs, * a body mass index (BMI) \<18 or ≥ 35 kg/m2, * coagulopathy (INR \>1.5 and/or platelet count \<70000/µL), * local infection, * history of chronic pain or regular opioid use; * inability to comprehend the Numeric Rating Scale (NRS), * liver resection combined with a second surgical procedure, laparoscopic resections, * pregnant or lactating women. * Patients with complicated procedures * those needing postoperative ventilation will be excluded from the study **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** ahmed hasanin **Phone:** 01095076954 **Role:** CONTACT #### Locations **Location 1:** **City:** Cairo **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Anesthesia, Pain Management and Surgical ICU Department - **Phone:** 00201222224057 - **Role:** CONTACT **Country:** Egypt **Facility:** Kasr Alaini Hospital **Status:** RECRUITING **Zip:** 11562 ### IPD Sharing Statement Module **Description:** data can be available from the principle investigator upon reasonable request **IPD Sharing:** NO ### References Module #### References **Citation:** Mostafa M, Mousa MS, Hasanin A, Arafa AS, Raafat H, Ragab AS. Erector spinae plane block versus subcostal transversus abdominis plane block in patients undergoing open liver resection surgery: A randomized controlled trial. Anaesth Crit Care Pain Med. 2023 Feb;42(1):101161. doi: 10.1016/j.accpm.2022.101161. Epub 2022 Sep 22. **PMID:** 36154912 ## Derived Section ### Intervention Browse Module - Browse Branches - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: PhSol - Name: Pharmaceutical Solutions - Abbrev: Hemat - Name: Hematinics ### Intervention Browse Module - Browse Leaves - ID: M4107 - Name: Anesthetics - Relevance: LOW - As Found: Unknown - ID: M21860 - Name: Pharmaceutical Solutions - Relevance: LOW - As Found: Unknown - ID: M4109 - Name: Anesthetics, Local - Relevance: LOW - As Found: Unknown - ID: M11110 - Name: Liver Extracts - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01661179 **Brief Title:** Evaluate the Safety and Tolerability of Vandetanib in Japanese Patients With Medullary Thyroid Carcinoma **Official Title:** A Phase I/II, Open-label Study to Evaluate the Safety and Tolerability of Vandetanib 300 mg/Day in Japanese Patients With Unresectable Locally Advanced or Metastatic Medullary Thyroid Carcinoma #### Organization Study ID Info **ID:** D4200C00098 #### Organization **Class:** INDUSTRY **Full Name:** Sanofi ### Status Module #### Completion Date **Date:** 2014-07 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2016-12-05 **Type:** ESTIMATED **Last Update Submit Date:** 2016-10-11 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2013-12 **Type:** ACTUAL #### Results First Post Date **Date:** 2015-05-19 **Type:** ESTIMATED **Results First Submit Date:** 2014-12-08 **Results First Submit QC Date:** 2015-05-18 #### Start Date **Date:** 2012-11 **Status Verified Date:** 2016-10 #### Study First Post Date **Date:** 2012-08-09 **Type:** ESTIMATED **Study First Submit Date:** 2012-07-17 **Study First Submit QC Date:** 2012-08-06 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Genzyme, a Sanofi Company #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Open-label Study to Evaluate the Safety and Tolerability of Vandetanib 300 mg/day in Japanese Patients with Unresectable Locally Advanced or Metastatic Medullary Thyroid Carcinoma. **Detailed Description:** A Phase I/II, Open-label Study to Evaluate the Safety and Tolerability of Vandetanib 300 mg/day in Japanese Patients with Unresectable Locally Advanced or Metastatic Medullary Thyroid Carcinoma ### Conditions Module **Conditions:** - Unresectable Locally Advanced or Metastatic, Medullary Thyroid Carcinoma **Keywords:** - Medullary Thyroid Carcinoma - Medullary Thyroid Cancer - Vandetanib ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 14 **Type:** ACTUAL **Phases:** - PHASE1 - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 300 mg/day vandetanib **Intervention Names:** - Drug: Vandetanib 300mg **Label:** Vandetanib 300mg **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Vandetanib 300mg **Description:** 300 mg oral dose once daily (100 mg x 3 tablets) **Name:** Vandetanib 300mg **Other Names:** - ZD6474 **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** ORR is defined as the percentage of patients who have a confirmed CR (Disappearance of all target lesions) or PR (\>=30% decrease in the sum of diameters of target lesions) prior to any evidence of progression as defined by RECIST V1.1. This percentage is calculated with only patients who had at least measurable lesion in the efficacy analysis set. **Measure:** Objective Response Rate Within the First 56 Weeks After the First Dose of Vandetanib **Time Frame:** Sept 2012 to May 2014 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Written consent from female or male Japanese patients aged 20 years and over. Evidence of non-childbearing status for women of childbearing potential, or postmenopausal status. * Previous diagnosis of unresectable, locally advanced or metastatic, hereditary or sporadic Medullary Thyroid Carcinoma(MTC). * Patients who have a good overall health status(World Health Organization (WHO) Performance status 0-2). * Patients who have appropriate renal conditions confirmed by test results for taking part in the study. * For patients with measurable disease(at least one lesion, not irradiated within 12 weeks of study registration, with longest diameter more or equal 10mm (lymph nodes minimum more or equal 15 mm) with CT or MRI). Exclusion Criteria: * Patients with brain metastases or spinal cord compression. * Patients with significant abnormal ECG (QTcB correction unmeasurable or more than 480 ms)findings and /or significant cardiac conditions or events, uncontrolled hypertension and evidence of severe lung disease. * Abnormal electrolytes such as potassium, magnesium and calcium, or abnormal organ functions such as decreased creatinine clearance. * Patients with significant abnormal laboratory findings (to include abnormal liver function tests (bilirubin more than 1.5xULRR, and ALT, AST, or ALP more than 2.5xULRR or 5.0xULRR if related to liver metastases). * Prior treatment (major surgery, radiation therapy, chemotherapy, or other investigational drugs) received within 28 days before registration. **Minimum Age:** 20 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Fukuoka-shi **Country:** Japan **Facility:** Research Site **Location 2:** **City:** Kobe-shi **Country:** Japan **Facility:** Research Site **Location 3:** **City:** Koto-ku **Country:** Japan **Facility:** Research Site **Location 4:** **City:** Shinjuku-ku **Country:** Japan **Facility:** Research Site #### Overall Officials **Official 1:** **Affiliation:** Sanofi **Name:** Clinical Sciences & Operations, MD **Role:** STUDY_DIRECTOR ### References Module #### See Also Links **Label:** CSR Synopsis **URL:** http://filehosting.pharmacm.com/DownloadService.ashx?client=CTR_MED_7111&studyid=356&filename=D4200C00098_Clinical_Trial_Disclosure_Synopsis.pdf ## 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: D000004700 - Term: Endocrine System Diseases - ID: D000004701 - Term: Endocrine Gland Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000006258 - Term: Head and Neck Neoplasms - ID: D000018358 - Term: Neuroendocrine Tumors - ID: D000017599 - Term: Neuroectodermal Tumors - ID: D000009373 - Term: Neoplasms, Germ Cell and Embryonal - ID: D000000230 - Term: Adenocarcinoma - ID: D000009380 - Term: Neoplasms, Nerve Tissue ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: All - Name: All Conditions - Abbrev: BC19 - Name: Gland and Hormone Related Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M5534 - Name: Carcinoma - Relevance: HIGH - As Found: Carcinoma - ID: M16718 - Name: Thyroid Diseases - Relevance: HIGH - As Found: Thyroid - ID: M16723 - Name: Thyroid Neoplasms - Relevance: HIGH - As Found: Thyroid Carcinoma - ID: M20423 - Name: Carcinoma, Neuroendocrine - Relevance: HIGH - As Found: Medullary Thyroid 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: M7862 - Name: Endocrine System Diseases - Relevance: LOW - As Found: Unknown - ID: M7863 - Name: Endocrine Gland Neoplasms - Relevance: LOW - As Found: Unknown - ID: M9348 - Name: Head and Neck Neoplasms - Relevance: LOW - As Found: Unknown - ID: M20495 - Name: Neuroendocrine Tumors - Relevance: LOW - As Found: Unknown - ID: M19845 - Name: Neuroectodermal Tumors - Relevance: LOW - As Found: Unknown - ID: M20388 - Name: Neuroectodermal Tumors, Primitive - Relevance: LOW - As Found: Unknown - ID: M12318 - Name: Neoplasms, Germ Cell and Embryonal - Relevance: LOW - As Found: Unknown - ID: M3585 - Name: Adenocarcinoma - Relevance: LOW - As Found: Unknown - ID: M12325 - Name: Neoplasms, Nerve Tissue - Relevance: LOW - As Found: Unknown - ID: T5651 - Name: Thyroid Cancer, Medullary - Relevance: HIGH - As Found: Medullary Thyroid Carcinoma - ID: T4091 - Name: Neuroendocrine Tumor - Relevance: LOW - As Found: Unknown - ID: T4092 - Name: Neuroepithelioma - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000002277 - Term: Carcinoma - ID: D000013964 - Term: Thyroid Neoplasms - ID: D000018278 - Term: Carcinoma, Neuroendocrine - ID: D000013959 - Term: Thyroid Diseases ### Misc Info Module - Version Holder: 2024-05-24 ## Results Section ### Adverse Events Module #### Event Groups **Group ID:** EG000 **Title:** Vandetanib 300 mg **Description:** Vandetanib at 300 mg using 3 x 100 mg vandetanib tablets were dosed orally, once daily **ID:** EG000 **Other Num Affected:** 14 **Other Num at Risk:** 14 **Serious Number Affected:** 4 **Serious Number At Risk:** 14 **Title:** Vandetanib 300 mg **Frequency Threshold:** 5 #### Other Events **Term:** Chalazion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Corneal opacity **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Abdominal pain upper **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Dyspepsia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Gastritis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Haemorrhoidal haemorrhage **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Nausea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Fatigue **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Non-cardiac chest pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Pyrexia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Anisakiasis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 **Term:** Tinea pedis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 **Term:** Alanine aminotransferase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 17.0 **Term:** Aspartate aminotransferase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 17.0 **Term:** Blood alkaline phosphatase increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 17.0 **Term:** Electrocardiogram QT prolonged **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 17.0 **Term:** Haemoglobin increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 17.0 **Term:** Weight decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 17.0 **Term:** Arthralgia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Arthritis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Muscle spasms **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Musculoskeletal chest pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Dysgeusia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Headache **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Somnolence **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Syncope **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Tremor **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Anxiety **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Insomnia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Haematuria **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Eosinophilia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Blood and lymphatic system disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Palpitations **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Supraventricular extrasystoles **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Cardiac disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Vertigo **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Ear and labyrinth disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Dry eye **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Eyelid function disorder **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Keratitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Keratopathy **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Meibomian gland dysfunction **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Vision blurred **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Cheilitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Constipation **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Diarrhoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Dry mouth **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Glossitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Stomatitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Vomiting **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Malaise **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Mucosal erosion **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Oedema **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Oedema mucosal **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Bronchitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 **Term:** Cystitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 **Term:** Dermatitis infected **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 **Term:** Gastroenteritis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 **Term:** Herpes zoster **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 **Term:** Laryngitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 **Term:** Nasopharyngitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 **Term:** Paronychia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 **Term:** Rash pustular **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 **Term:** Upper respiratory tract infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 **Term:** Urinary tract infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 **Term:** Vaginitis bacterial **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 **Term:** Vulvovaginal candidiasis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 **Term:** Wound infection **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 **Term:** Thermal burn **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Injury, poisoning and procedural complications **Source Vocabulary:** MedDRA 17.0 **Term:** Blood calcium increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 17.0 **Term:** Blood creatinine increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 17.0 **Term:** Blood magnesium decreased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 17.0 **Term:** Red blood cell count increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 17.0 **Term:** Weight increased **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Investigations **Source Vocabulary:** MedDRA 17.0 **Term:** Decreased appetite **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Dehydration **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Hypocalcaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Hypokalaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Hypomagnesaemia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Metabolism and nutrition disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Neck pain **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Musculoskeletal and connective tissue disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Peripheral sensory neuropathy **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Restlessness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Psychiatric disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Punctate keratitis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Eye disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Dizziness **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Nervous system disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Nephrolithiasis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Proteinuria **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Renal impairment **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Renal and urinary disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Cough **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Dysphonia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Dyspnoea **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Dyspnoea exertional **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Epistaxis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Hiccups **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Alopecia **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Dermatitis acneiform **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Dry skin **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Ingrowing nail **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Onychomadesis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Palmar-plantar erythrodysaesthesia syndrome **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Pruritus **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Rash **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Rash maculo-papular **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Skin and subcutaneous tissue disorders **Source Vocabulary:** MedDRA 17.0 **Term:** Hypertension **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Vascular disorders **Source Vocabulary:** MedDRA 17.0 #### Serious Events **Term:** Ascites **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Gastrointestinal disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 14 **Num Events:** 1 **Term:** Fatigue **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 14 **Num Events:** 1 **Term:** Malaise **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** General disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 14 **Num Events:** 1 **Term:** Pyelonephritis **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Infections and infestations **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 14 **Num Events:** 1 **Term:** Interstitial lung disease **Assessment Type:** SYSTEMATIC_ASSESSMENT **Organ System:** Respiratory, thoracic and mediastinal disorders **Source Vocabulary:** MedDRA 17.0 ##### Stats **Group ID:** EG000 **Num Affected:** 1 **Num At Risk:** 14 **Num Events:** 1 ## Results Section - Baseline Characteristics Module ### Denomination Counts **Group ID:** BG000 **Value:** 14 **Units:** Participants ### Group **ID:** BG000 **Title:** Vandetanib 300 mg **Description:** Vandetanib at 300 mg using 3 x 100 mg vandetanib tablets were dosed orally, once daily ### Measure #### Measurement **Group ID:** BG000 **Lower Limit:** 37 **Spread:** 10.21 **Upper Limit:** 71 **Value:** 52.6 **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 6 **Class Title:** >=20 - <50 years #### Measurement **Group ID:** BG000 **Value:** 7 **Class Title:** >=50 - <65 years #### Measurement **Group ID:** BG000 **Value:** 1 **Class Title:** >=65 years ### Measure #### Measurement **Group ID:** BG000 **Value:** 7 **Category Title:** Female #### Measurement **Group ID:** BG000 **Value:** 7 **Category Title:** Male **Class Title:** ### Measure #### Measurement **Group ID:** BG000 **Value:** 14 **Class Title:** Asian **Denomination Units Selected:** ## Results Section - Baseline Characteristics Module ### Measure 1 **Dispersion Type:** STANDARD_DEVIATION **Parameter Type:** MEAN **Title:** Age, Continuous **Unit of Measure:** Years ### Measure 2 **Parameter Type:** NUMBER **Title:** Age, Customized **Unit of Measure:** Participants ### Measure 3 **Parameter Type:** COUNT_OF_PARTICIPANTS **Title:** Sex: Female, Male **Unit of Measure:** Participants ### Measure 4 **Parameter Type:** NUMBER **Title:** Race/Ethnicity, Customized **Unit of Measure:** Participants ## Results Section - More Information Module ### Certain Agreement **Other Details:** If no publication has occurred within 12 months of the completion of the study, the Investigator shall have the right to publish/present independently the results of the study. The Investigator shall provide the Sponsor with a copy of any such presentation/publication for comment at least 30 days before any presentation/submission for publication. If requested by the Sponsor, any presentation/submission shall be delayed up to 90 days, to allow the Sponsor to preserve its proprietary rights. **Restriction Type:** OTHER **Restrictive Agreement:** True ### Point of Contact **Email:** [email protected] **Organization:** Sanofi **Title:** Trial Transparency Team ## 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:** 13.86 - **Spread:** - **Upper Limit:** 68.42 - **Value:** 38.5 **Title:** ## Results Section ### Outcome Measures Module #### Outcome Measure 1 **Description:** ORR is defined as the percentage of patients who have a confirmed CR (Disappearance of all target lesions) or PR (\>=30% decrease in the sum of diameters of target lesions) prior to any evidence of progression as defined by RECIST V1.1. This percentage is calculated with only patients who had at least measurable lesion in the efficacy analysis set. **Dispersion Type:** 95% Confidence Interval **Parameter Type:** NUMBER **Population Description:** All patients with post-baseline efficacy assessments **Reporting Status:** POSTED **Time Frame:** Sept 2012 to May 2014 **Title:** Objective Response Rate Within the First 56 Weeks After the First Dose of Vandetanib **Type:** PRIMARY **Unit of Measure:** percentage of participants ##### Group **Description:** Vandetanib at 300 mg using 3 x 100 mg vandetanib tablets were dosed orally, once daily **ID:** OG000 **Title:** Vandetanib 300 mg ### Participant Flow Module #### Group **Description:** Vandetanib at 300 mg using 3 x 100 mg vandetanib tablets were dosed orally, once daily **ID:** FG000 **Title:** Vandetanib 300 mg #### Period **Title:** Overall Study ##### Withdraw **Type:** Death ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ##### Withdraw **Type:** Withdrawal by Subject ###### Reason **Group ID:** FG000 **Number of Subjects:** 1 ##### Withdraw **Type:** Other Eligibility criteria ###### Reason **Group ID:** FG000 **Number of Subjects:** 4 ##### Milestone **Type:** STARTED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 14 ##### Milestone **Type:** COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 8 ##### Milestone **Type:** NOT COMPLETED ###### Achievement **Group ID:** FG000 **Number of Subjects:** 6 **Pre-Assignment Details:** 16 participants were screened; 14 participants were treated. **Recruitment Details:** From 12 November 2012 to 18 June 2013, 14 participants were treated by 4 centers in Japan to receive vandetanib. **Has Results:** True

## Protocol Section ### Identification Module **NCT ID:** NCT00179179 **Brief Title:** The Effects of Nutrition Supplementation and Resistance Exercise During Hemodialysis **Official Title:** The Effects of Nutrition Supplementation and Resistance Exercise During Hemodialysis #### Organization Study ID Info **ID:** 50189 #### Organization **Class:** OTHER **Full Name:** Vanderbilt University #### Secondary ID Infos **ID:** R01DK045604 **Link:** https://reporter.nih.gov/quickSearch/R01DK045604 **Type:** NIH ### Status Module #### Completion Date **Date:** 2007-05 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2009-07-09 **Type:** ESTIMATED **Last Update Submit Date:** 2009-07-08 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2007-05 **Type:** ACTUAL #### Start Date **Date:** 2005-04 **Status Verified Date:** 2009-07 #### Study First Post Date **Date:** 2005-09-15 **Type:** ESTIMATED **Study First Submit Date:** 2005-09-13 **Study First Submit QC Date:** 2005-09-13 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Vanderbilt University #### Responsible Party **Old Name Title:** Alp Ikizler, MD **Old Organization:** Vanderbilt University Medical Center ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** To test the hypothesis that an exercise session combined with adequate nutritional supplementation improves skeletal muscle protein accretion during a hemodialysis session. ### Conditions Module **Conditions:** - End Stage Renal Disease ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 11 **Type:** ACTUAL **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** nutritional supplement plus resistance exercise **Intervention Names:** - Drug: nutritional supplementation - Behavioral: exercise **Label:** 1 **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** nutritional supplement only (resistance exercise will not be performed) **Intervention Names:** - Drug: nutritional supplementation **Label:** 2 **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - 1 - 2 **Description:** 2 oral ingestions per study visit of protein supplement containing a total of 960 kilocalories: 132.8 kilocalories from protein, 412.8 kilocalories from carbohydrates, and 412.8 kilocalories from fat; 6 study visits over a 12-month period **Name:** nutritional supplementation **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - 1 **Description:** resistance training on a duel leg press consisting of 3 sets of 8-12 repetitions; the first 5 study visits over a 12-month period **Name:** exercise **Type:** BEHAVIORAL ### Outcomes Module #### Primary Outcomes **Measure:** improvement in net protein muscle balance **Time Frame:** 10 hours #### Secondary Outcomes **Measure:** improvement in net whole body protein balance **Time Frame:** 10 hours ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * On hemodialysis for more than 3 months, on a thrice weekly hemodialysis program. * Adequately dialyzed (Kt/V \> 1.2). * Age 18-75 Exclusion Criteria: * Pregnant women. * Patients unable to perform exercise * Severe unstable underlying disease besides commonly associated with ESRD. Cardiac patients that are stable will be included. * Patients hospitalized within the last month prior to the study. * Patients with malfunctioning arterial-venous access (recirculation and/or blood flow \< 750 ml/min) * Patients receiving steroids and/or other immunosuppressive agents. **Maximum Age:** 75 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Nashville **Country:** United States **Facility:** Vanderbilt University Medical Center **State:** Tennessee **Zip:** 37232 #### Overall Officials **Official 1:** **Affiliation:** Vanderbilt University Medical Center **Name:** Alp Ikizler, MD **Role:** PRINCIPAL_INVESTIGATOR ## 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: D000051437 - Term: Renal Insufficiency - 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: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M10698 - Name: Kidney Diseases - Relevance: LOW - As Found: Unknown - ID: M10699 - Name: Kidney Failure, Chronic - Relevance: HIGH - As Found: End Stage Renal Disease - ID: M26718 - Name: Renal Insufficiency - Relevance: LOW - As Found: Unknown - ID: M17319 - Name: Urologic Diseases - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M27095 - Name: Male Urogenital Diseases - Relevance: LOW - As Found: Unknown - 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: D000007676 - Term: Kidney Failure, Chronic ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03712579 **Brief Title:** Impact of High-fat Meals Varying in Fatty Acid Composition on Adipose and Systemic Metabolic-inflammatory Responses **Official Title:** Impact of High-fat Meals Varying in Fatty Acid Composition on Adipose and Systemic Metabolic-inflammatory Responses: a Randomized Controlled Postprandial Study #### Organization Study ID Info **ID:** R18-P124 #### Organization **Class:** OTHER **Full Name:** Loughborough University ### Status Module #### Completion Date **Date:** 2020-10-15 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2022-05-17 **Type:** ACTUAL **Last Update Submit Date:** 2022-05-15 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2020-10-15 **Type:** ACTUAL #### Start Date **Date:** 2019-01-21 **Type:** ACTUAL **Status Verified Date:** 2022-05 #### Study First Post Date **Date:** 2018-10-19 **Type:** ACTUAL **Study First Submit Date:** 2018-06-06 **Study First Submit QC Date:** 2018-10-17 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Loughborough University #### Responsible Party **Investigator Affiliation:** Loughborough University **Investigator Full Name:** Dr Oonagh Markey **Investigator Title:** Vice-Chancellor's Research Fellow **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** Cardiometabolic disorders are a leading cause of death worldwide. Replacing saturated fatty acids (SFA) with unsaturated fatty acids is recommended as a way of lowering cardiometabolic disease risk. Consuming a diet rich in SFA may lead to a greater metabolic-inflammatory response in white adipose tissue during the fasting state, when compared to eating a diet rich in monounsaturated fatty acids (MUFA). Since individuals spend most of the day in the fed (or postprandial) state, it is important to see how different types of dietary fatty acids affect postprandial white adipose tissue and systemic metabolic-inflammatory responses. This study will investigate the effect of a SFA-rich meal on markers of white adipose tissue and systemic metabolic-inflammation, compared to a MUFA-rich meal in overweight adults. In a randomised, single blind controlled, cross-over manner participants will consume either a SFA- or MUFA-rich meal and sequential blood and white adipose tissue samples will be collected before and until 6 hours postprandially. ### Conditions Module **Conditions:** - Inflammation - Obesity - Inflammatory Response - Cardiovascular Diseases - Overweight ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** CROSSOVER ##### Masking Info **Masking:** SINGLE **Who Masked:** - PARTICIPANT **Primary Purpose:** BASIC_SCIENCE #### Enrollment Info **Count:** 8 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants will consume a SFA-rich test meal (75g test fat) and sequential blood and white adipose tissue samples will be collected **Intervention Names:** - Dietary Supplement: SFA-Rich Meal **Label:** SFA-Rich Meal **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants will consume a MUFA-rich test meal (75g test fat) and sequential blood and white adipose tissue samples will be collected **Intervention Names:** - Dietary Supplement: MUFA-Rich Meal **Label:** MUFA-Rich Meal **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - SFA-Rich Meal **Description:** Saturated fatty acid-rich test meal, containing 75g test fat **Name:** SFA-Rich Meal **Type:** DIETARY_SUPPLEMENT #### Intervention 2 **Arm Group Labels:** - MUFA-Rich Meal **Description:** Monounsaturated fatty acid-rich test meal, containing 75g test fat **Name:** MUFA-Rich Meal **Type:** DIETARY_SUPPLEMENT ### Outcomes Module #### Primary Outcomes **Description:** This will be assessed following the collection of white adipose tissue samples across the postprandial period **Measure:** Protein expression (content and phosphorylation) of key markers of metabolic inflammation in white adipose tissue (for example assessment of NFKB/IKBa total protein and phosphorylation by western blot analysis) **Time Frame:** White adipose tissue samples will be collected at -0.5 (fasted), 1, 4 and 6 hours postprandially #### Secondary Outcomes **Description:** This will be assessed following the collection of blood samples **Measure:** Systemic Markers of Inflammation (for example TNFa and IL-6 concentrations, determined using an ELISA) **Time Frame:** Blood samples will be collected at -0.5 (fasted) until 6 hours postprandially **Description:** This will be assessed following the collection of white adipose tissue samples **Measure:** Gene expression of key markers of metabolic inflammation in white adipose tissue **Time Frame:** White adipose tissue samples will be collected at -0.5 (fasted), 1, 4 and 6 hours postprandially **Description:** Assessed following the collection of blood samples **Measure:** Characterisation of immune cell populations (monocyte subsets) from peripheral blood mononuclear cells (measured using flow cytometry analysis) **Time Frame:** Blood samples will be collected at -0.5 (fasted) and 4 hours postprandially **Description:** Assessed following the collection of blood samples **Measure:** Serum Markers of Insulin Resistance **Time Frame:** Blood samples will be collected at -0.5 (fasted) until 6 hours postprandially **Description:** Assessed following the collection of blood samples **Measure:** Serum Lipid Profile (primarily triacylglycerol and non-esterified fatty acid concentrations, measured using a spectrophotometric assay) **Time Frame:** Blood samples will be collected at -0.5 (fasted) until 6 hours postprandially ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * 18-50 years * BMI = 25-40 kg/m2 * Male or Female * Waist circumference \>94 cm (men) and \>80cm (women)\* * Physically active (\> 3 x 30 min moderate intensity exercise per week) * Systolic blood pressure \< 160 mmHg and diastolic blood pressure \< 100 mmHg * No cardiometabolic (e.g. heart disease, high blood pressure, type 2 diabetes) or inflammatory illness * NOTE: If waist circumference falls below 94 cm for men or 80cm for women but BMI is \>25 kg/m2 volunteers may still be recruited at the PI's discretion. Exclusion Criteria: * Smoker * Previous diagnosis of anaemia * Women who are pregnant or lactating * Taking medication known to interfere with study outcomes (e.g. treatment for hyperlidaemia, hypertension, inflammation or hypercoagulation) or prescribed antibiotics within the last 3 months * Taking nutritional supplements known to interfere with study outcomes (e.g. fish oil or evening primrose oil) * Unstable weight history (\>3 kg loss or gain in the previous 3 months) * An allergy to lidocaine * Those with known or suspected food intolerances, allergies or hypersensitivity to any components of the meal (e.g. lactose/wheat intolerance) * Alcohol consumption \>28 units per week for a man or \>21 units per week for a woman * Any other unusual medical history or diet and lifestyle habits or practices that would preclude volunteers from participating in a dietary intervention or metabolic study * Parallel participation in another intervention study **Healthy Volunteers:** True **Maximum Age:** 50 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Loughborough **Country:** United Kingdom **Facility:** Loughborough University **State:** Leicestershire **Zip:** LE11 3TU #### Overall Officials **Official 1:** **Affiliation:** Loughborough University **Name:** Oonagh Markey, BSc, PhD **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000010335 - Term: Pathologic Processes - 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: M10293 - Name: Inflammation - Relevance: HIGH - As Found: Inflammation - ID: M12701 - Name: Obesity - Relevance: LOW - As Found: Unknown - ID: M26186 - Name: Overweight - Relevance: HIGH - As Found: Overweight - 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: D000002318 - Term: Cardiovascular Diseases - ID: D000050177 - Term: Overweight - ID: D000007249 - Term: Inflammation ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00000879 **Brief Title:** A Study of the Effects of Giving Two Anti-HIV Vaccines to Babies of HIV-Positive Mothers **Official Title:** A Phase I/II Study to Evaluate the Safety and Immunogenicity of ALVAC HIV Vaccines Alone and With AIDSVAX B/B in Children Born to HIV-Infected Mothers #### Organization Study ID Info **ID:** ACTG 326 #### Organization **Class:** NIH **Full Name:** National Institute of Allergy and Infectious Diseases (NIAID) #### Secondary ID Infos **ID:** PACTG 326 **Domain:** DAIDS ES Registry Number **ID:** 10601 **Type:** REGISTRY ### Status Module #### Completion Date **Date:** 2005-04 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-10-29 **Type:** ACTUAL **Last Update Submit Date:** 2021-10-27 **Overall Status:** COMPLETED **Status Verified Date:** 2021-10 #### Study First Post Date **Date:** 2001-08-31 **Type:** ESTIMATED **Study First Submit Date:** 1999-11-02 **Study First Submit QC Date:** 2001-08-30 ### Sponsor Collaborators Module #### Collaborators **Class:** NIH **Name:** Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) #### Lead Sponsor **Class:** NIH **Name:** National Institute of Allergy and Infectious Diseases (NIAID) #### Responsible Party **Type:** SPONSOR ### Description Module **Brief Summary:** The purpose of this study is to see if giving the ALVAC vCP1452 anti-HIV vaccine alone or with another vaccine called AIDSVAX B/B to babies of HIV-positive mothers is safe. The study will also look at how these vaccines affect a baby's immune system. Most HIV-positive children get HIV from their mothers during pregnancy or birth. Treatment with anti-HIV drugs can reduce the baby's risk of getting HIV. Vaccines also may help prevent HIV infection. This study will look at whether the ALVAC vCP1452 vaccine and the AIDSVAX B/B vaccine can help the body fight off HIV infection. There is no chance of getting HIV infection from the vaccines. (This study has been changed. In earlier versions, ALVAC vCP205 and AIDSVAX B/E were going to be used.) **Detailed Description:** Transmission of HIV from an untreated infected mother to her offspring is thought to occur to some infants perinatally and others at parturition. It is possible that administration of an immunogenic vaccine can reduce the vertical transmission of HIV-1 or moderate its course in infected infants. Successful early sensitization to HIV epitopes might succeed in preventing HIV infection. Alternately, the enhancement of HIV-specific immune function might also succeed in modifying HIV replication and affecting disease progression. Sixty infants are treated in this randomized, double-blind study; 45 infants receive recombinant Canarypox virus, ALVAC-HIV vCP205, and 15 receive placebo. Mothers serve as proxy for their infants. All infants receive a minimum of four immunizations, at Weeks 0 (within 72 hours of birth), 4, 8, and 12. Initially, 24 patients are randomized to receive one of two doses of vCP205 or a saline placebo. When a suitable subunit vaccine is available, the protocol will be amended and 36 additional infants will be randomized to receive vCP205 alone or with a subunit vaccine at Weeks 4 and 8 (or vaccine placebo with or without subunit placebo). \[AS PER AMENDMENT 11/5/97: 18 infants receive ALVAC-HIV vCP205 at one of two doses and 6 receive placebo.\] \[AS PER AMENDMENT 9/9/99: Cohort 1 received vCP205. Cohort 2 received a higher dose of vCP205. Cohort A received vCP205 placebo (saline). Cohorts 1, 2, and A were double-blinded and closed to accrual in March 1999. As of September 1999, infants are randomized to one of four new cohorts. Cohort 3 receives vCP1452 at Weeks 0, 4, 8, and 12. Cohort 4 receives vCP1452 at Weeks 0 and 4, then receives vCP1452 plus AIDSVAX B/E gp120 at Weeks 8 and 12. Cohort B receives vCP1452 placebo at Weeks 0, 4, 8, and 12. Cohort C receives vCP1452 placebo at Weeks 0 and 4, then receives vCP1452 placebo plus AIDSVAX B/E placebo at Weeks 8 and 12. All infants are followed every 2 weeks for the first 14 weeks of life, and then every 6 months until age 2. Cord blood is used to establish autologous B cell lines, and CTL assays are performed to characterize the immune response to HIV. In addition, CD4 count, viral load, and mucosal antibody responses are measured. Immunized infants who are not infected with HIV serve as controls for the immunogenicity of the vaccines in the infected infants.\] \[AS PER AMENDMENT 1/24/00: AIDSVAX B/E has been replaced with AIDSVAX B/B.\] ### Conditions Module **Conditions:** - HIV Infections - HIV Seronegativity **Keywords:** - Pregnancy - Pregnancy Complications, Infectious - AIDS Vaccines - Disease Transmission, Vertical - Avipoxvirus - HIV Preventive Vaccine - HIV Therapeutic Vaccine ### Design Module #### Design Info ##### Masking Info **Masking:** DOUBLE **Primary Purpose:** PREVENTION #### Enrollment Info **Count:** 48 **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Name:** ALVAC(2)120(B,MN)GNP (vCP1452) **Type:** BIOLOGICAL #### Intervention 2 **Name:** MN rgp120/HIV-1 and GNE8 rgp120/HIV-1 **Type:** BIOLOGICAL #### Intervention 3 **Name:** ALVAC-HIV MN120TMG (vCP205) **Type:** BIOLOGICAL ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria The infant may be eligible if the mother: * Is HIV-positive. * Is willing to follow the study guidelines. * Had her baby at Week 37 of pregnancy or later. Exclusion Criteria The infant will not be eligible if the mother: * Has hepatitis B. * Is breast-feeding her baby. * Used certain medications during pregnancy. The infant will not be eligible if he/she: * Is more than 3 days old at study entry. * Has a serious infection or life-threatening illness. **Maximum Age:** 3 Days **Minimum Age:** 0 Days **Sex:** ALL **Standard Ages:** - CHILD ### Contacts Locations Module #### Locations **Location 1:** **City:** Long Beach **Country:** United States **Facility:** Long Beach Memorial Med. Ctr., Miller Children's Hosp. **State:** California **Zip:** 90801 **Location 2:** **City:** Orange **Country:** United States **Facility:** Children's Hosp. of Orange County **State:** California **Zip:** 92868 **Location 3:** **City:** San Francisco **Country:** United States **Facility:** UCSF Pediatric AIDS CRS **State:** California **Location 4:** **City:** Torrance **Country:** United States **Facility:** Harbor - UCLA Med. Ctr. - Dept. of Peds., Div. of Infectious Diseases **State:** California **Location 5:** **City:** Chicago **Country:** United States **Facility:** Mt. Sinai Hosp. Med. Ctr. - Chicago, Womens & Childrens HIV Program **State:** Illinois **Zip:** 60608 **Location 6:** **City:** Chicago **Country:** United States **Facility:** Chicago Children's CRS **State:** Illinois **Location 7:** **City:** New Orleans **Country:** United States **Facility:** Tulane Univ. Health Science Ctr., Tulane Univ. Hosp. & Clinic **State:** Louisiana **Location 8:** **City:** Baltimore **Country:** United States **Facility:** Univ. of Maryland Med. Ctr., Div. of Ped. Immunology & Rheumatology **State:** Maryland **Zip:** 21201 **Location 9:** **City:** Boston **Country:** United States **Facility:** HMS - Children's Hosp. Boston, Div. of Infectious Diseases **State:** Massachusetts **Location 10:** **City:** Worcester **Country:** United States **Facility:** WNE Maternal Pediatric Adolescent AIDS CRS **State:** Massachusetts **Location 11:** **City:** Bronx **Country:** United States **Facility:** Jacobi Med. Ctr. Bronx NICHD CRS **State:** New York **Zip:** 10461 **Location 12:** **City:** New York **Country:** United States **Facility:** Nyu Ny Nichd Crs **State:** New York **Zip:** 10016 **Location 13:** **City:** New York **Country:** United States **Facility:** Columbia IMPAACT CRS **State:** New York **Zip:** 10032 **Location 14:** **City:** Syracuse **Country:** United States **Facility:** SUNY Upstate Med. Univ., Dept. of Peds. **State:** New York **Zip:** 13210 **Location 15:** **City:** Philadelphia **Country:** United States **Facility:** Univ. of Pennsylvania Health System, Hosp. of the Univ. of Pennsylvania **State:** Pennsylvania **Zip:** 19104 **Location 16:** **City:** Philadelphia **Country:** United States **Facility:** The Children's Hosp. of Philadelphia IMPAACT CRS **State:** Pennsylvania **Location 17:** **City:** Seattle **Country:** United States **Facility:** Seattle Children's Hospital CRS **State:** Washington #### Overall Officials **Official 1:** **Name:** John Lambert **Role:** STUDY_CHAIR **Official 2:** **Name:** Daniel Johnson **Role:** STUDY_CHAIR **Official 3:** **Name:** Stuart Starr **Role:** STUDY_CHAIR ### References Module #### References **Citation:** Johnson D, McFarland E, Muresan P, Fenton T, Lambert J, McNamara J, Hawkins E, Bouquin P, Read J, Estep S, Gunurathan S, Gurwith M, PACTG 326 Protocol Team. PACTG 326: A Phase I/II Study to Evaluate the Safety and Immunogenicity of Alvac HIV Vaccines Alone and with AIDSVax B/B in Children Born to HIV-infected Mothers: Preliminary Results. 10th Conference on Retroviruses and Oppurtunistic Infections. Feb 2003. Abstract 404. **Citation:** Lambert JS. HIV vaccines in infants and children. Paediatr Drugs. 2005;7(5):267-76. doi: 10.2165/00148581-200507050-00001. **PMID:** 16220994 ## 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: 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 - Abbrev: BC20 - Name: Immune System Diseases ### Condition Browse Module - Browse Leaves - ID: M14127 - Name: Pregnancy Complications - 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: M3522 - Name: Acquired Immunodeficiency Syndrome - Relevance: LOW - As Found: Unknown - ID: M18250 - Name: HIV Infections - Relevance: HIGH - As Found: HIV Infections - ID: M9742 - Name: HIV Seropositivity - Relevance: LOW - As Found: Unknown - ID: M14130 - Name: Pregnancy Complications, Infectious - 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: 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:** NCT05116579 **Brief Title:** Circulating Tumor DNA (ctDNA) Monitoring in the Assessment and Prediction of the Efficacy of PARP Inhibitors (PARPi) **Official Title:** Circulating Tumor DNA (ctDNA) Monitoring in the Assessment and Prediction of the Efficacy of PARP Inhibitors (PARPi) #### Organization Study ID Info **ID:** 2021-FXY-171 #### Organization **Class:** OTHER **Full Name:** Sun Yat-sen University ### Status Module #### Completion Date **Date:** 2023-08-31 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2021-11-11 **Type:** ACTUAL **Last Update Submit Date:** 2021-11-01 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2023-03-31 **Type:** ESTIMATED #### Start Date **Date:** 2021-10-31 **Type:** ESTIMATED **Status Verified Date:** 2021-11 #### Study First Post Date **Date:** 2021-11-11 **Type:** ACTUAL **Study First Submit Date:** 2021-11-01 **Study First Submit QC Date:** 2021-11-01 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Sun Yat-sen University #### Responsible Party **Investigator Affiliation:** Sun Yat-sen University **Investigator Full Name:** Yonghong Li **Investigator Title:** Deputy Director of the Department of Urology **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False ### Description Module **Brief Summary:** To evaluate the application value of customized ctDNA monitoring in efficacy assessment and prediction during PARPi treatment **Detailed Description:** This clinical study is an open-label, single-center, observational study to evaluate the application value of customized ctDNA monitoring in efficacy assessment and prediction during PARPi treatment in mCRPC patients. A total of 30 participants with second-line treatment failure will be registered in this study. Whole blood collection will be conducted during the treatment for ctDNA detection, homologous recombination repair (HRR) genes testing, personalized panel customization and whole exome sequencing. ### Conditions Module **Conditions:** - Metastatic Castration-resistant Prostate Cancer ### Design Module #### Bio Spec **Description:** Whole blood and tumor tissue **Retention:** SAMPLES_WITH_DNA #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 30 **Type:** ESTIMATED **Patient Registry:** True **Study Type:** OBSERVATIONAL **Target Duration:** 12 Months ### Outcomes Module #### Primary Outcomes **Description:** Radiological progression free survival (rPFS) - defined as the time from randomisation to radiological progression, assessed by investigator per Response Evaluation Criteria in Solid Tumours (RECIST) 1.1 (soft tissue) and Prostate Cancer Working Group-3 (PCWG-3) criteria (bone), or death from any cause, or the last follow-up, whichever occurs first **Measure:** Radiological progression free survival (rPFS) **Time Frame:** From date of randomisation to until radiographic progression (up to 1 year) **Description:** ORR is the percentage of patients with at least one visit response of Complete response (CR) or Partial response (PR), in their soft tissue disease assessed by Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1), in the absence of progression on bone scan assessed by Prostate Cancer Working Group 3 (PCWG3)). Per RECIST v1.1, CR=Disappearance of all target lesions; PR = \>=30% decrease in the sum of diameters of target lesions; For each treatment group, ORR is the number of patients with a CR and PR. **Measure:** Objective Response Rate (ORR) **Time Frame:** From randomisation until radiographic progression (up to 1 year) **Description:** PSA response rate is defined as the proportion of patients with a PSA decline (defined as a ≥30%, ≥50% and other declines in PSA from baseline). **Measure:** PSA response rate **Time Frame:** From randomisation until radiographic progression (up to 1 year) #### Secondary Outcomes **Description:** OS is defined as time from treatment commencement to death of any cause **Measure:** Overall Survival (OS) **Time Frame:** 2 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: Patients must meet ALL of the following criteria: 1. Willing and able to provide informed consent. 2. Adult males from 18 to 75 years age. 3. History of histologically or cytologically confirmed adenocarcinoma of the prostate with DDR genes mutation (BRCA1/2, ATM, BARD1, BRIP1, CDK12, CHEK1, CHEK2, FANCL, PALB2, PPP2R2A, RAD51B, RAD51C, RAD51D, RAD54L) detected by high throughput sequencing 4. Documented evidence of metastatic castration resistant prostate cancer (mCRPC) and proposed treatment of PARP inhibitors. 5. Evidence of measurable target lesion in imaging studies. 6. Participants can provide adequate formalin fixed paraffin-embedded (FFPE) tumor tissue collected before any treatment: tumor cell content\>30% and necrotic cells\<10%. 7. ECOG performance status 0-1 8. Estimated survival≥12 weeks Exclusion Criteria: Patients must NOT meet any of the following criteria: 1. Do not meet the inclusion criteria. 2. Under any other anti-tumor therapy like chemotherapy and/or immunotherapy. 3. Receiving organ transplantation in the last 3 months. 4. Participants with autoimmune diseases or history of HBV, HCV or HIV infection (acute or chronic). 5. Participants with pneumonia. 6. Severe concurrent illness or co-morbid disease that would make the subject unsuitable for enrolment 7. Unwilling and unable to provide informed consent. 8. Patients who are judged unsuitable for clinical trial participation by the investigators. Elimination Criteria: Violation of the prescribed rule of medication that may influence the judgment of curative effect and safety. **Gender Based:** True **Maximum Age:** 75 Years **Minimum Age:** 18 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** MALE **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** clinic-based sample ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Yonghong Li, M.D. **Phone:** +020-87343656 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Jun Wang, M.D. **Phone:** +020-87343656 **Role:** CONTACT #### Locations **Location 1:** **City:** Guangzhou **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Yonghong Li, M.D. - **Phone:** +020-87343656 - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Fangjian Zhou, M.D. - **Phone:** +020-87343656 - **Role:** CONTACT **Country:** China **Facility:** Sun Yat-sen University Cancer Center **State:** Guangdong **Status:** RECRUITING **Zip:** 510060 #### Overall Officials **Official 1:** **Affiliation:** Sun Yat-sen University **Name:** Yonghong Li, M.D. **Role:** PRINCIPAL_INVESTIGATOR ## 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: Castration-Resistant 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: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M205 - Name: Poly(ADP-ribose) Polymerase Inhibitors - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT06198179 **Brief Title:** Effects of UP-CAT With and Without Visual Feedback in Children With CP **Official Title:** Effects of Upper Limb Children Action-observation Training (UP-CAT) With and Without Visual Feedback in Children With Cerebral Palsy #### Organization Study ID Info **ID:** REC/RCR&AHS/23/0753 #### Organization **Class:** OTHER **Full Name:** Riphah International University ### Status Module #### Completion Date **Date:** 2024-02-05 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-01-30 **Type:** ACTUAL **Last Update Submit Date:** 2024-01-28 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-02-05 **Type:** ESTIMATED #### Start Date **Date:** 2023-12-01 **Type:** ACTUAL **Status Verified Date:** 2024-01 #### Study First Post Date **Date:** 2024-01-10 **Type:** ACTUAL **Study First Submit Date:** 2023-12-26 **Study First Submit QC Date:** 2023-12-26 ### 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:** True ### Description Module **Brief Summary:** Cerebral palsy (CP) is the most common neurological disorder of movement and/or posture and of motor function, which are due to a non-progressive interference or abnormality of the developing brain. In hemiplegic cerebral palsy (HCP), one side of the body is involved with the upper extremity more affected than the lower. HCP is treated by addressing the underlying cause and by various form of therapy to recover motor function. Motor function in hemi paretic limb particularly fine motor skill may be improved by upper limb children action-observation therapy (UP-CAT). **Detailed Description:** This will be randomized controlled trial. Non-probability purposive sampling technique will be used for recruiting samples for study. Children will be assessed by House Functional Classification System for hemiplegia to include in the study. Computerized randomization will be used to locate subjects in two groups, and 28 hemiplegic cerebral palsy children will be divided into 2 groups; experimental and control groups. In experimental group hemiplegic cerebral palsy children will perform upper limb exercises with visual feedback for 5 times a week, 15 sets of daily life upper limb activities, each action will be observed for 2 minutes and executed for 2 minutes. In control group hemiplegic cerebral palsy children will perform upper limb activities for 5 times a week, 15 sets of daily life upper limb activities, each action will be observed for 2 minutes and executed for 2 minutes. Both groups will be assessed at the time of recruitment and at the end of 4 weeks. Data will be collected by Melbourne unilateral upper limb (MUUL) and ABILHAND-kids questionnaire. After collection of data, data will be analyzed by SPSS-25. ### Conditions Module **Conditions:** - Cerebral Palsy **Keywords:** - Cerebral palsy (CP), - Rehabilitation - Upper extremity - Activities of daily living ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL **Intervention Model Description:** This study will be randomized controlled trial (RCT) used to measure the effects of upper limb action-observation training (UP-CAT) with and without visual feedback in children with cerebral palsy. subjects with cerebral palsy meeting pre determined inclusion and exclusion criteria will be be divided into two groups using non-probability sampling techniques. ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 28 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** these individuals will receive the upper limb children action-observation training (UP-CAT) with visual feedback UP-CAT: * Individual tasks like modelling compound,board games,cards,magnets,glass bead to do bracelets,painting and crafting (making a grass man,making musical instruments with cardboard),cooking (making juice cocktails,fruit skewers,chocolate fondue),building towers, making puzzles \& water games including filling a bucket with a sponge/bottle,filling \& throwing water balloons. * To maximize the variety of the actions we included different objects (e.g.coins,bottles, stamps,modelling compound) to be grasped with different grasp types (whole hand,pinch, tripod grasp) and in different orientations of the wrist. Visual feedback: Mirror in front of patient will be used. After the baseline assessment,the patient in the experimental group will receive the upper limb children action-observation training with visual feedback Time of rehabilitation will be 1 hour/subject. **Intervention Names:** - Other: upper limb children action-observation training (UP-CAT) with visual feedback **Label:** Experimental group **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** This will receive the upper limb children action-observation training (UP-CAT). UP-CAT: Following tasks will be performed by children * Individual tasks like modelling compound,board games,cards,magnets,glass bead to do bracelets,painting and crafting (making a grass man,making musical instruments with cardboard),cooking (making juice cocktails,fruit skewers,chocolate fondue),building towers, making puzzles \& water games including filling a bucket with a sponge/bottle,filling \& throwing water balloons * To maximize the variety of the actions we included different objects (e.g. coins,bottles, stamps,modelling compound) to be grasped with different grasp types (whole hand,pinch, tripod grasp) \& in different orientations of the wrist.After the baseline assessment,the patient in the experimental group will receive the UL children action-observation training with visual feedback Time of rehabilitation will be 1 hour per subject. **Intervention Names:** - Other: upper limb children action-observation training (UPCAT) **Label:** Control group **Type:** OTHER ### Interventions #### Intervention 1 **Arm Group Labels:** - Control group **Description:** upper limb children action-observation training (UPCAT) without visual Feedback **Name:** upper limb children action-observation training (UPCAT) **Type:** OTHER #### Intervention 2 **Arm Group Labels:** - Experimental group **Description:** upper limb children action-observation training (UP-CAT) with visual feedback: Mirror therapy as visual feedback **Name:** upper limb children action-observation training (UP-CAT) with visual feedback **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** The MUUL is an evaluative tool that measures unilateral upper extremity quality of movement in children with neurological impairments aged from 5 to 15 years. MUUL is a criterion-referenced test based on 16 items scored on a 3- to 5-point ordinal scale comprising tasks that are representative of the most important components of unilateral UL function **Measure:** Melbourne Assessment of Unilateral Upper Limb Function (MUUL) **Time Frame:** 4 weeks **Description:** The ABILHAND-Kids is a short questionnaire that measures 21 mainly bimanual daily activities referred to the activity domain of the ICF. The difficulty experienced by the child to perform the required tasks is scored by a parent on a 3-point ordinal scale (impossible, difficult, and easy). **Measure:** ABILHAND-Kids **Time Frame:** 4 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Cerebral palsy children aged 5 years to 10 years at the time of recruitment. * Both male and female * According to Modified Ashworth scale of spasticity; score should be +1. * Mild or moderate UL disability i.e. active use of affected UL from poor active assist use to complete spontaneous use according to House Functional Classification System grade between 4 and 8. Exclusion Criteria: * Individuals with the other neurological deficits * Visual impairment. * Children with upper limb disabilities other than hemiplegic cerebral palsy. * Children with uncontrolled epileptic seizures in last 3 years * Non co-operative patients. * Previous orthopedic surgery or botulinum toxin A (BoNT-A) injection in the UL within 6 months prior to the enrolment of this study. **Healthy Volunteers:** True **Maximum Age:** 10 Years **Minimum Age:** 5 Years **Sex:** ALL **Standard Ages:** - CHILD ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Imran Amjad, PhD **Phone:** 9233224390125 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Muhammad Asif Javed, MS **Phone:** 923224209422 **Role:** CONTACT #### Locations **Location 1:** **City:** Lahore **Country:** Pakistan **Facility:** Riphah International University **State:** Punjab **Status:** RECRUITING #### Overall Officials **Official 1:** **Affiliation:** Riphah International University **Name:** IQRA Muhammad Afzal, MS* **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Sgandurra G, Cecchi F, Beani E, Mannari I, Maselli M, Falotico FP, Inguaggiato E, Perazza S, Sicola E, Feys H, Klingels K, Ferrari A, Dario P, Boyd RN, Cioni G. Tele-UPCAT: study protocol of a randomised controlled trial of a home-based Tele-monitored UPper limb Children Action observation Training for participants with unilateral cerebral palsy. BMJ Open. 2018 May 14;8(5):e017819. doi: 10.1136/bmjopen-2017-017819. **PMID:** 29764869 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009422 - Term: Nervous System Diseases - ID: D000001925 - Term: Brain Damage, Chronic - ID: D000001927 - Term: Brain Diseases - ID: D000002493 - Term: Central Nervous System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC26 - Name: Wounds and Injuries - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M5796 - Name: Cerebral Palsy - Relevance: HIGH - As Found: Cerebral Palsy - ID: M13157 - Name: Paralysis - Relevance: LOW - As Found: Unknown - ID: M5207 - Name: Brain Injuries - Relevance: LOW - As Found: Unknown - ID: M5202 - Name: Brain Damage, Chronic - 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: T1303 - Name: Chronic Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000002547 - Term: Cerebral Palsy ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00675779 **Acronym:** EndoStatin **Brief Title:** Efficacy Study of Atorvastatin in Pelvic Pain Relief in Women With Endometriosis **Official Title:** Comparison of Pain Relief and Inflammatory Status in Women With Surgically Confirmed Endometriosis Treated With Atorvastatin, Oral Contraceptive or Combined Oral Contraceptives and Atorvastatin: Open Randomized Controlled Trial #### Organization Study ID Info **ID:** 204-08 #### Organization **Class:** OTHER **Full Name:** Poznan University of Medical Sciences ### Status Module #### Completion Date **Date:** 2011-03 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** ACTIVE_NOT_RECRUITING #### Last Update Post Date **Date:** 2008-05-12 **Type:** ESTIMATED **Last Update Submit Date:** 2008-05-09 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2010-11 **Type:** ESTIMATED #### Start Date **Date:** 2008-04 **Status Verified Date:** 2008-05 #### Study First Post Date **Date:** 2008-05-12 **Type:** ESTIMATED **Study First Submit Date:** 2008-05-05 **Study First Submit QC Date:** 2008-05-09 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** University of California, Davis **Class:** INDUSTRY **Name:** Biomet Polska Sp. z.o.o. #### Lead Sponsor **Class:** OTHER **Name:** Poznan University of Medical Sciences #### Responsible Party **Old Name Title:** Robert Z. Spaczynski, MD PhD **Old Organization:** Poznan University of Medical Sciences ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is to determine whether atorvastatin (alone or in combination with oral contraceptive) is effective in treatment of pelvic pain and inflammatory response in women with endometriosis. ### Conditions Module **Conditions:** - Endometriosis - Pain **Keywords:** - endometriosis - atorvastatin - oral - contraception - pain - relief - pelvic pain in women with endometriosis ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 44 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** oral contraceptive + atorvastatin **Intervention Names:** - Drug: atorvastatin + oral contraceptive **Label:** 2 **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** oral contraceptive **Intervention Names:** - Drug: oral contraceptive (Mercilon) **Label:** 1 **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - 1 **Description:** oral contraceptive (20ug EE, 150ug DSG) 1 tablet a day p.o. for 21 days with 7 day break for 6 months **Name:** oral contraceptive (Mercilon) **Other Names:** - Mercilon (Organon Schering-Plough, Poland) **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - 2 **Description:** atorvastatin 20mg po a day for 6 months + oral contraceptive (20ug EE, 150ug DSG) 1 tablet po a day for 21 days with 7 day break for 6 months **Name:** atorvastatin + oral contraceptive **Other Names:** - Atrox 20 (Biofarm sp. z o.o. , Poznan, Poland) **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** pain relief **Time Frame:** 3,6,12 months #### Secondary Outcomes **Measure:** inflammatory status **Time Frame:** 6 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * informed written consent * premenopausal women aged 18-45 * clinical signs and symptoms of endometriosis for minimum 3 months with endometriosis confirmed at laparoscopy or laparotomy within last 4 months - 5 years (preferably with histological confirmation) * pain symptoms of moderate intensity (dysmenorrhoea + dyspareunia + nonmenstrual pelvic pain \> 6 points on Biberoglu \& Behrman scale \[1981\]) * no clinical signs of sexually transmitted disease Exclusion Criteria: * cancer of the ovary, adrenals, endometrium, uterine cervix, breasts * pregnancy or lactation * unexplained uterine/cervical bleeding * hormonal therapy within last 3 months (for GnRH analogs 6 months) * irregular menses (\> 35 days) or secondary amenorrhoea (\>3 months) * other chronic disease affecting pelvic or abdominal cavity (including PID, ulcerating colitis , Crohn's disease, recurrent interstitial cystitis) * sexually transmitted disease (gonorrhoea, Chlamydia) * uncontrolled diabetes mellitus type I or II, VTE or other contraindications to medicine used in the study * chronic therapy with CYP3A4 inhibitors (including ketoconazole, erythromycin and others) **Maximum Age:** 45 Years **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Poznan **Country:** Poland **Facility:** Poznan University of Medical Sciences, Department of Gynecology and Obstetrics **Zip:** 60-535 #### Overall Officials **Official 1:** **Affiliation:** University of California, Davies, USA **Name:** Antoni J Duleba, MD **Role:** STUDY_CHAIR **Official 2:** **Affiliation:** Poznan University of Medical Sciences, Poland **Name:** Leszek Pawelczyk, MD PhD **Role:** STUDY_DIRECTOR ## Derived Section ### Condition Browse Module - Ancestors - 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: M19918 - Name: Pelvic Pain - Relevance: LOW - As Found: Unknown - ID: M7877 - Name: Endometriosis - Relevance: HIGH - As Found: Endometriosis - ID: M13066 - Name: Pain - 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 ### Condition Browse Module - Meshes - ID: D000004715 - Term: Endometriosis ### Intervention Browse Module - Ancestors - ID: D000000924 - Term: Anticholesteremic Agents - ID: D000000960 - Term: Hypolipidemic Agents - ID: D000000963 - Term: Antimetabolites - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action - ID: D000057847 - Term: Lipid Regulating Agents - ID: D000019161 - Term: Hydroxymethylglutaryl-CoA Reductase Inhibitors - ID: D000004791 - Term: Enzyme Inhibitors - ID: D000012102 - Term: Reproductive Control Agents - ID: D000045505 - Term: Physiological Effects of Drugs - ID: D000003271 - Term: Contraceptive Agents, Female - ID: D000003473 - Term: Neuromuscular Nondepolarizing Agents - ID: D000009466 - Term: Neuromuscular Blocking Agents - ID: D000009465 - Term: Neuromuscular Agents - ID: D000018373 - Term: Peripheral Nervous System Agents ### Intervention Browse Module - Browse Branches - Abbrev: Lipd - Name: Lipid Regulating Agents - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: Repr - Name: Reproductive Control Agents ### Intervention Browse Module - Browse Leaves - ID: M351 - Name: Atorvastatin - Relevance: HIGH - As Found: Of each - ID: M6494 - Name: Contraceptive Agents - Relevance: HIGH - As Found: Veterans - ID: M6500 - Name: Contraceptives, Oral - Relevance: HIGH - As Found: Ovary - ID: M6501 - Name: Contraceptives, Oral, Combined - Relevance: LOW - As Found: Unknown - ID: M216393 - Name: Dacuronium - Relevance: HIGH - As Found: Proflavine - ID: M4243 - Name: Anticholesteremic Agents - Relevance: LOW - As Found: Unknown - ID: M4278 - Name: Hypolipidemic Agents - Relevance: LOW - As Found: Unknown - ID: M4281 - Name: Antimetabolites - Relevance: LOW - As Found: Unknown - ID: M28883 - Name: Lipid Regulating Agents - Relevance: LOW - As Found: Unknown - ID: M21155 - Name: Hydroxymethylglutaryl-CoA Reductase Inhibitors - Relevance: LOW - As Found: Unknown - ID: M7951 - Name: Enzyme Inhibitors - Relevance: LOW - As Found: Unknown - ID: M6495 - Name: Contraceptive Agents, Female - Relevance: LOW - As Found: Unknown - ID: M6684 - Name: Neuromuscular Nondepolarizing Agents - Relevance: LOW - As Found: Unknown - ID: M12409 - Name: Neuromuscular Blocking Agents - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000069059 - Term: Atorvastatin - ID: D000003270 - Term: Contraceptive Agents - ID: D000003276 - Term: Contraceptives, Oral - ID: C000003549 - Term: Dacuronium ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03927079 **Acronym:** ABBA **Brief Title:** Predictive Factors of Good Pulmonary Penetration of Antibiotics : AntiBiotics Dosage in Broncho-Alveolar Lavage **Official Title:** Predictive Factors of Good Pulmonary Penetration of Antibiotics : AntiBiotics Dosage in Broncho-Alveolar Lavage #### Organization Study ID Info **ID:** PI2018_843_0032 #### Organization **Class:** OTHER **Full Name:** Centre Hospitalier Universitaire, Amiens ### Status Module #### Completion Date **Date:** 2023-06-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-09-06 **Type:** ACTUAL **Last Update Submit Date:** 2023-09-05 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2022-04-01 **Type:** ACTUAL #### Start Date **Date:** 2019-04-23 **Type:** ACTUAL **Status Verified Date:** 2023-09 #### Study First Post Date **Date:** 2019-04-25 **Type:** ACTUAL **Study First Submit Date:** 2019-04-23 **Study First Submit QC Date:** 2019-04-23 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Centre Hospitalier Universitaire, Amiens #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Respiratory infections are common and sometimes very severe. An insufficient dosage of the antibiotic could lead to a treatment failure A correct plasmatic antibiotic concentration is not a guarantee of a clinical success as it could not be a reflect of pulmonary concentration. The aim of this study is to determinate the predictive factors of pulmonary penetration of antibiotics in patients with a beta lactamines failure and who undergoes a flexible bronchoscopy. **Detailed Description:** To check if pulmonary concentrations of antibiotic are enough we will measure antibiotic concentration in the broncho-alveolar lavage (BAL). This technique which is clinically relevant and reliable could determinate the pulmonary diffusion level for antibiotics by calculating the ratio between plasmatic and intra-alveolar antibiotic concentration. This ratio will be correlated with potential limitation factors of pulmonary diffusion as respiratory diseases (COPD, cystic fibrosis, fibrosis...), sepsis, hypoalbuminemia. We have chosen to study the beta lactamin antibiotics because they are the most frequently used in pneumonia. Moreover, the beta lactamins pulmonary diffusion is likely to be the lowest. Finally, for patients with a known pathogen, we will divide this pulmonary concentration with minimal inhibitory concentration (MIC). Indeed, in severe pneumonia, to be sure of bactericidal activity, a pulmonary concentration of beta lactamines should be always higher than 4 to 5 times MIC. ### Conditions Module **Conditions:** - Antibiotics - Pneumonia **Keywords:** - Antibiotics - Pneumonia - broncho-alveolar lavage ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** DIAGNOSTIC #### Enrollment Info **Count:** 101 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Description:** included patients will have a broncho-alveolar lavage (BAL) to measure intra-alveolar antibiotic concentration, and a blood test to measure plasmatic concentration **Name:** measure of intra-alveolar antibiotic concentration in µg/ml **Type:** BIOLOGICAL #### Intervention 2 **Description:** included patients will have a broncho-alveolar lavage (BAL) to measure intra-alveolar antibiotic concentration, and a blood test to measure plasmatic concentration **Name:** broncho-alveolar lavage (BAL) **Type:** PROCEDURE #### Intervention 3 **Description:** included patients will have a broncho-alveolar lavage (BAL) to measure intra-alveolar antibiotic concentration, and a blood test to measure plasmatic **Name:** blood test to measure plasmatic antibiotic concentration in µg/ml **Type:** BIOLOGICAL ### Outcomes Module #### Primary Outcomes **Description:** pulmonary diffusion level for beta lactamins is determined by ratio between bronchoalveolar concentration and plasmatic concentration of tested antibiotics **Measure:** pulmonary diffusion level for beta lactamins **Time Frame:** on the day of the bronco-alveolar lavage #### Secondary Outcomes **Description:** measure of apyrexia duration in days **Measure:** measure of apyrexia duration in days **Time Frame:** from day of inclusion to 15 days after inclusion **Description:** duration in days for regression of the biological inflammatory syndrome (CRP concentration in mg/l divided by 2) **Measure:** duration in days for regression of the biological inflammatory syndrome **Time Frame:** from day of inclusion to 15 days after inclusion **Description:** measure of length of hospitalisation in days **Measure:** measure of length of hospitalisation **Time Frame:** from day of inclusion to 15 days after inclusion **Description:** 28-day mortality will be measured **Measure:** Number of deaths at 28-day **Time Frame:** 28 days after inclusion **Description:** virus presence will be detected in bronchoalveolar lavage (BAL) **Measure:** virus presence in BAL **Time Frame:** day of bronchoalveolar lavage (BAL) ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * patients who undergo a flexible bronchoscopic lavage and with a beta lactamines treatment as cephalosporin of 3rd generation (CEFTRIAXONE / CEFOTAXIME, CEFTAZIDIME), of 4th generation (CEFEPIM), or AMOXICILLIN-CLAVULANIC ACID, or PIPERACILLIN-TAZOBACTAM * patient major * informed and signed consent form Exclusion Criteria: * patient under chronic dialysis * patient placed under judicial protection **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Amiens **Country:** France **Facility:** CHU Amiens **Zip:** 80480 #### Overall Officials **Official 1:** **Affiliation:** CHU Amiens **Name:** Claire Andrejak, Pr **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000012141 - Term: Respiratory Tract Infections - ID: D000007239 - Term: 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: M13904 - Name: Pneumonia - Relevance: HIGH - As Found: Pneumonia - 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: 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: D000011014 - Term: Pneumonia ### Intervention Browse Module - Ancestors - ID: D000000890 - Term: Anti-Infective Agents - ID: D000000995 - Term: Antitubercular Agents - ID: D000002614 - Term: Chelating Agents - ID: D000064449 - Term: Sequestering Agents - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action ### Intervention Browse Module - Browse Branches - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M4222 - Name: Anti-Bacterial Agents - Relevance: HIGH - As Found: Initial - ID: M4224 - Name: Antibiotics, Antitubercular - Relevance: HIGH - As Found: Initial - ID: M7296 - Name: Dimercaprol - Relevance: HIGH - As Found: Isavuconazonium - ID: M4311 - Name: Antitubercular 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: D000000900 - Term: Anti-Bacterial Agents - ID: D000000904 - Term: Antibiotics, Antitubercular - ID: D000004112 - Term: Dimercaprol ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01880879 **Acronym:** Pro-HOPE **Brief Title:** A Prospective Multicenter Trial Evaluating Helios Biodegradable Polymer SES Safety and Effectiveness in CAD Treatment **Official Title:** A Prospective Multicenter Trial Evaluating Helios Biodegradable Polymer Sirolimus-eluting Stent Safety and Effectiveness in Treatment of Coronary Artery Disease #### Organization Study ID Info **ID:** Pro-HOPE #### Organization **Class:** INDUSTRY **Full Name:** Kinhely Bio-tech Co., Ltd. ### Status Module #### Completion Date **Date:** 2015-01 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2013-06-24 **Type:** ESTIMATED **Last Update Submit Date:** 2013-06-20 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2015-01 **Type:** ESTIMATED #### Start Date **Date:** 2013-01 **Status Verified Date:** 2013-01 #### Study First Post Date **Date:** 2013-06-19 **Type:** ESTIMATED **Study First Submit Date:** 2013-05-30 **Study First Submit QC Date:** 2013-06-16 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** RenJi Hospital **Class:** OTHER **Name:** Peking University #### Lead Sponsor **Class:** INDUSTRY **Name:** Kinhely Bio-tech Co., Ltd. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** research topic:helios After registration of clinical research bidders:kinhely bio-tech Co.Ltd study design:A prospective single treatment group multicenter clinical study Number of patients:Included in the subjects of 800 cases Main research purpose:Evaluation of safety and effectiveness Mainly studies the finish:1 year target lesion of the failure Inclusion criteria : one:Gender not limited at the age of 18 to 80 two:myocardial ischemia or angina symptoms of coronary artery disease three:A narrow is less than 50% treated with stents four:Comply with the instructions five:Voluntarily signed the informed consent form exclusion criteria: one:Pregnant women two:cardiac shock three:Against a suppository medicines or allergies four:Participated in other test research in the first 6 months five:Within 6 months after PCI plans to accept non cardiac surgery six:Non-compliant patient Follow-up time:A month for 6 months and 9 months to 1 year to 2 years follow-up progress plan:All center within 6 months after start of complete into the group **Detailed Description:** no desired ### Conditions Module **Conditions:** - Coronary Artery Disease **Keywords:** - coronary artery disease - PCI - stent ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 800 **Type:** ESTIMATED **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** the group with helios stent implanted **Intervention Names:** - Device: helios stent **Label:** helios stent **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - helios stent **Description:** the group with helios stent implanted **Name:** helios stent **Type:** DEVICE ### Outcomes Module #### Other Outcomes **Description:** Stent placement in patients with the incidence of hospitalized again **Measure:** Rehospitalization rate **Time Frame:** 2 years #### Primary Outcomes **Description:** Target blood vessels caused by cardiac death and myocardial infarction and ischemia driven overall incidence of target lesion revascularization **Measure:** 1 year incidence of target lesion **Time Frame:** 1year #### Secondary Outcomes **Description:** Death and myocardial infarction and thromboembolism events and hospitalizations and the incidence of major adverse cardiac events as the standard for safety evaluation **Measure:** cardiac death Target blood vessels caused by myocardial infarction Ischemia driven target lesion revascularization of 1 year and 2 years,MACE, Stent thrombosis events,clinical success. **Time Frame:** 2 years **Description:** Cardiac death drive target blood vessels lead to myocardial infarction and ischemia of the incidence of target lesion revascularization **Measure:** 2 years, the incidence of various combination of the pathological changes of failure **Time Frame:** 2 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Any age 18 to 80 sex Myocardial ischemia or angina pectoris of coronary artery disease An application stents diameter stenosis greater than 50% Comply with the instructions Voluntarily signed the informed consent form Exclusion Criteria: * Pregnant women cardiac shock Intolerance to antithrombotic therapy or allergies 6 months prior to the start of the study involved in other studies Within 6 months after PCI plans to accept non cardiac surgery Non-compliant patient **Maximum Age:** 80 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Shanghai **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** qi lu, chairman - **Phone:** +86-021-68383364 - **Phone Ext:** 68383006 - **Role:** CONTACT ***Contact 2:*** - **Name:** ben he, chairman - **Role:** PRINCIPAL_INVESTIGATOR **Country:** China **Facility:** Yan chai hospital affiliated to Shanghai jiaotong university school of medicine **State:** Shanghai **Status:** RECRUITING **Zip:** 200127 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000006331 - Term: Heart Diseases - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000001161 - Term: Arteriosclerosis - ID: D000001157 - Term: Arterial Occlusive Diseases - ID: D000014652 - Term: Vascular Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology ### Condition Browse Module - Browse Leaves - ID: M6549 - Name: Coronary Disease - Relevance: HIGH - As Found: Coronary Artery Disease - ID: M6546 - Name: Coronary Artery Disease - Relevance: HIGH - As Found: Coronary Artery Disease - ID: M19506 - Name: Myocardial Ischemia - Relevance: HIGH - As Found: Coronary Artery Disease - ID: M10543 - Name: Ischemia - Relevance: LOW - As Found: Unknown - ID: M9419 - Name: Heart Diseases - Relevance: LOW - As Found: Unknown - ID: M4469 - Name: Arteriosclerosis - Relevance: LOW - As Found: Unknown - ID: M4465 - Name: Arterial Occlusive Diseases - Relevance: LOW - As Found: Unknown - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000003324 - Term: Coronary Artery Disease - ID: D000017202 - Term: Myocardial Ischemia - ID: D000003327 - Term: Coronary Disease ### Intervention Browse Module - Browse Branches - Abbrev: Infe - Name: Anti-Infective Agents - Abbrev: ANeo - Name: Antineoplastic Agents - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M21960 - Name: Sirolimus - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02329379 **Acronym:** TGAUS **Brief Title:** The Role of Levothyroxine on Goiter With Atypia of Undetermined Significance **Official Title:** Therapeutic Role of Levothyroxine on the Patients With Atypia of Undetermined Significance (AUS) Goiter #### Organization Study ID Info **ID:** 1-103-05-081 #### Organization **Class:** OTHER **Full Name:** National Defense Medical Center, Taiwan ### Status Module #### Completion Date **Date:** 2015-12-31 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2019-08-21 **Type:** ACTUAL **Last Update Submit Date:** 2019-08-19 **Overall Status:** WITHDRAWN #### Primary Completion Date **Date:** 2015-12-31 **Type:** ACTUAL #### Start Date **Date:** 2015-05 **Type:** ESTIMATED **Status Verified Date:** 2019-08 #### Study First Post Date **Date:** 2014-12-31 **Type:** ESTIMATED **Study First Submit Date:** 2014-12-29 **Study First Submit QC Date:** 2014-12-29 **Why Stopped:** No participants enrolled ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Sheng-Chiang Su #### Responsible Party **Investigator Affiliation:** National Defense Medical Center, Taiwan **Investigator Full Name:** Sheng-Chiang Su **Investigator Title:** Attending Physician **Type:** SPONSOR_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** 1. To elucidate the therapeutic role of levothyroxine on the patient with atypia of undetermined significance (AUS) goiter, we would design a prospective, open label and non-randomized trial to verify the therapeutic effects on goiter with AUS by means of TSH (Thyrotropin; Thyroid-Stimulating Hormone) suppression related reduction of goiter volume with subsequent alleviation of previous cytological malignant tendency. During following for 1-2 years after therapy with simultaneous monitor of possible adverse effects of levothyroxine (eltroxin), we collected blood samples and gathered all necessary data as well as performed thyroid sonography with fine-needle aspiration (FNA) for the subjects. Furthermore, we would finish our individual study for each enrolled subject if his/her condition exhibited the criteria of primary end point: reduced goiter volume under sono \>15% or cytology: benign for 2 times; operation for thyroidectomy, CAD attack, refractory arrhythmia, newly DM (Diabetes Mellitus), etc. 2. To re-evaluate the current prevalence of goiter within Taiwanese adults, we recruited all patients who presented possible thyroid related symptoms, such as unexplained body weight loss with increased appetite, palpitation, hand tremors, neck swelling, hoarseness and abnormal sensation over throat. Only the patients with goiter proven by thyroid sono in euthyroid status without other preexisting major disorders can be enrolled in our trial to undergo open label, non-randomized study. **Detailed Description:** The prevalence of goiter in Taiwanese adults was found to be 19.4% in males, 33.6% in females and 25% in total about 20 years ago even though there was no iodine-deficient area reported in Taiwan. The result implied that goiter not only became a quite common disorder, but also deserved further investigation, particularly since 0.7% of goiter would be diagnosed as thyroid cancers by means of fine-needle aspiration (FNA). However, the recent research indicated 5-15% of results after fine-needle aspiration directly disclosed malignant cytology. On the other hand, about 80% of initial results of FNA would appear non-malignant cytology. The most common feature of them was found to be indeterminate cytology, which refers to 15-30% of FNA specimens. The indeterminate cytology included follicular or Hurthle cell neoplasm as well as atypia of undetermined significance (AUS). Based on the revised American Thyroid Association Management Guidelines for Patients with Thyroid Nodules, people would be referred to visit surgeons for surgical intervention if their reports of FNA demonstrated either Hurthle cell neoplasm or follicular neoplasm without autonomously functioning nodules; instead, population with AUS goiter only received observation for a period of time for following. Otherwise, they would be arranged to undergo surgical manipulations if any evidence of malignancy was suspected. There has been lack of sufficient studies to offer therapeutic options for the patient with AUS goiter around the world up to the present, especially in Taiwan. Therefore, in order to avoid unnecessary operation performed upon the patient with AUS goiter and provide another effective treatment for them, a well-designed, prospective clinical trial will be warranted on the patient with AUS goiter. To achieve the goal, the pathophysiology of goiter required to be understood at first. Although the actual etiology of goiter was uncertain, TSH (thyrotropin) has been regarded as a major factor to induce its formation up to now. To attenuate the growth-tropic effects of TSH, levothyroxine suppression therapy had been administered in the past to treat goiter, particularly about the goiter with colloid nodules. Successful reduction of goiter volume was found in some published papers while others did not verify the therapeutic effects of levothyroxine. However, the fact that goiter with size under thyroid sonogram more than 1cm required to be further examined indicated reduced volume of goiter may offer benefits including amelioration of previous malignant tendency especially in AUS goiter through TSH suppression therapy though some studies indicated routine suppression therapy of benign thyroid nodules in iodine sufficient populations is not recommended At first, patients who aged 20 to 70 with thyroid dysfunction related manifestations as goiter, palpitation or hand tremors would be recruited while they visited MET OPD (Out-Patient Department) and then they would be arranged to undergo thyroid sono as well as receive thyroid functional tests during screening periods before the study. The patients would be eligible for the interventional trial if their thyroid sonography demonstrated goiter and their thyroidal function revealed euthyroid status. After careful investigations of subjects' associated histories as family histories, drug histories and diet habits, the patients would be actually recruited for the clinical trial with the signed informed consent. Initially, the enrolled subjects would be classified into two major groups as solitary nodular goiter and multiple nodular goiter. Subsequently, two subgroups would be identified from each major group by presence of autoantibody of thyroglobulin or not. Thereafter, the patients would be asked to comply with the guidelines of ATA (American Thyroid Association) for advanced evaluation , fine needle aspiration and following if they indeed present with either goiter with more than 1 cm in size or less than 1 cm with regard to typical malignant characteristics under thyroid sonography, such as heterogeneous, hypoechoic, increased peri-vascularization, tall than wide. Furthermore, patients with history of family MTC (Medullary Thyroid Cancer) or contact of radiation or radiation therapy would be arranged to under FNA, too. The patients with first FNA disclosing AUS (atypia of undetermined significance) would be arranged to receive FNA again for confirmatory examination one month later and soon later those patients with positive findings of AUS twice would be allowed to take eltroxin for TSH suppression therapy. On the contrary, the patients would be assigned to the groups who will receive traditional observation and following of thyroid sonography as well as thyroid functional tests. These observed patients would receive surgical interventions if their FNA showed malignant potentials as NUCLEAR GROOVING， MICROFOLLICULAR FOCI，Hurthle cell, etc. As to the studied patients with consecutive FNA reports indicating AUS twice would be arranged to take levothyroxine (eltroxin, 0.1 mg) for therapy and its dosage would be adjusted carefully after 2 weeks per each OPD visiting. Optimal therapeutic goals would be regarded as subclinical hyperthyroidism and constant concentrations or dosages of eltroxin would be achieved in 2 months. Thereafter, the studied subjects would be arranged to receive thyroid sonography and FNA 3 months later and other necessary blood biochemistry studies for following. All following information would be recorded well and completely. The primary endpoint means: reduced goiter volume under sono \>15% or cytology: benign\*2 times; operation for thyroidectomy; CAD attack; refractory arrhythmia; newly DM. The pathology of all FNA during the clinical trial would be judged by the Bethesda System for Reporting Thyroid Cytopathology. ### Conditions Module **Conditions:** - Goiter ### Design Module #### Design Info **Allocation:** NON_RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Type:** ACTUAL **Phases:** - PHASE4 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** The patients who have uninodular goiter demonstrated by thyroid sono and positive autoantibody of thyroglobulin with pathological reports of fine needle aspiration showing atypical undetermined of significance will take eltroxin for TSH suppression therapy to ameliorate of goiter **Intervention Names:** - Drug: Levothyroxine **Label:** Unigoiter, ATA (+), AUS (+) on Tx **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** The patients who have uninodular goiter demonstrated by thyroid sono and negative autoantibody of thyroglobulin with pathological reports of fine needle aspiration showing atypical undetermined of significance will take eltroxin for TSH suppression therapy to ameliorate of goiter **Intervention Names:** - Drug: Levothyroxine **Label:** Unigoiter, ATA (-), AUS (+) on Tx **Type:** ACTIVE_COMPARATOR #### Arm Group 3 **Description:** The patients who have uninodular goiter demonstrated by thyroid sono and positive autoantibody of thyroglobulin with pathological reports of fine needle aspiration showing atypical undetermined of significance decide not to take eltroxin for TSH suppression therapy; instead, only observation with following will be conducted **Label:** Unigoiter, ATA (+), AUS (+) without Tx **Type:** NO_INTERVENTION #### Arm Group 4 **Description:** The patients who have uninodular goiter demonstrated by thyroid sono and negative autoantibody of thyroglobulin with pathological reports of fine needle aspiration showing atypical undetermined of significance decide not to take eltroxin for TSH suppression therapy; instead, only observation with following will be conducted **Label:** Unigoiter, ATA (-), AUS (+) without Tx **Type:** NO_INTERVENTION #### Arm Group 5 **Description:** The patients who have multinodular goiter demonstrated by thyroid sono and positive autoantibody of thyroglobulin with pathological reports of fine needle aspiration showing atypical undetermined of significance will take eltroxin for TSH suppression therapy to ameliorate of goiter **Intervention Names:** - Drug: Levothyroxine **Label:** Multigoiter, ATA (+), AUS (+) on Tx **Type:** EXPERIMENTAL #### Arm Group 6 **Description:** The patients who have multinodular goiter demonstrated by thyroid sono and negative autoantibody of thyroglobulin with pathological reports of fine needle aspiration showing atypical undetermined of significance will take eltroxin for TSH suppression therapy to ameliorate of goiter **Intervention Names:** - Drug: Levothyroxine **Label:** Multigoiter, ATA (-), AUS (+) on Tx **Type:** ACTIVE_COMPARATOR #### Arm Group 7 **Description:** The patients who have multinodular goiter demonstrated by thyroid sono and positive autoantibody of thyroglobulin with pathological reports of fine needle aspiration showing atypical undetermined of significance decide not to take eltroxin for TSH suppression therapy; instead, only observation with following will be conducted **Label:** Multigoiter, ATA (+), AUS (+) without Tx **Type:** NO_INTERVENTION #### Arm Group 8 **Description:** The patients who have multinodular goiter demonstrated by thyroid sono and negative autoantibody of thyroglobulin with pathological reports of fine needle aspiration showing atypical undetermined of significance decide not to take eltroxin for TSH suppression therapy; instead, only observation with following will be conducted **Label:** Multigoiter, ATA (-), AUS (+) without Tx **Type:** NO_INTERVENTION ### Interventions #### Intervention 1 **Arm Group Labels:** - Multigoiter, ATA (+), AUS (+) on Tx - Multigoiter, ATA (-), AUS (+) on Tx - Unigoiter, ATA (+), AUS (+) on Tx - Unigoiter, ATA (-), AUS (+) on Tx **Name:** Levothyroxine **Other Names:** - Eltroxin **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Number of participants with reduced goiter volume under sono >15% **Time Frame:** 2 years **Measure:** Number of participants with fine-needle aspiration cytology: benign*2 times **Time Frame:** 2 years #### Secondary Outcomes **Measure:** Number of participants need to receive operation for thyroidectomy due to malignant potentials **Time Frame:** 2 years ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * all patients who presented with goiter proven by thyroid sono in euthyroid status without other preexisting major disorders and possible thyroid related symptoms, such as unexplained body weight loss with increased appetite, palpitation, hand tremors, neck swelling, hoarseness and abnormal sensation over throat. Exclusion Criteria: * all patients without definite goiter * all patients with major illness as CAD, DM or CVA (CerebroVascular Accident) * all patients with pregnancy or malignancy **Maximum Age:** 70 Years **Minimum Age:** 20 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Overall Officials **Official 1:** **Affiliation:** Tri-Service General Hospital National Defense Medical Center **Name:** Sheng-Chiang Su **Role:** PRINCIPAL_INVESTIGATOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000013959 - Term: Thyroid Diseases - ID: D000004700 - Term: Endocrine System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC19 - Name: Gland and Hormone Related Diseases - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M9147 - Name: Goiter - Relevance: HIGH - As Found: Goiter - ID: M16718 - Name: Thyroid Diseases - Relevance: LOW - As Found: Unknown - ID: M7862 - Name: Endocrine System Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000006042 - Term: Goiter ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M4625 - Name: Autoantibodies - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02959879 **Acronym:** PANACHE01 **Brief Title:** Neo-adjuvant FOLF(IRIN)OX for Resectable Pancreatic Adenocarcinoma **Official Title:** Resectable Pancreatic Adenocarcinoma Neo-Adjuvant FOLF(IRIN)OX-based CHEmotherapy - A Multicenter, Randomised Phase II Trial (PANACHE01-PRODIGE48 Study) #### Organization Study ID Info **ID:** 2014/210/HP #### Organization **Class:** OTHER **Full Name:** University Hospital, Rouen ### Status Module #### Completion Date **Date:** 2021-12 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2018-04-26 **Type:** ACTUAL **Last Update Submit Date:** 2018-04-24 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2021-12 **Type:** ESTIMATED #### Start Date **Date:** 2017-03-01 **Type:** ACTUAL **Status Verified Date:** 2018-04 #### Study First Post Date **Date:** 2016-11-09 **Type:** ESTIMATED **Study First Submit Date:** 2016-11-03 **Study First Submit QC Date:** 2016-11-07 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** UNICANCER **Class:** OTHER **Name:** Federation Francophone de Cancerologie Digestive **Class:** OTHER **Name:** Federation of Research in Surgery (FRENCH) **Class:** OTHER **Name:** GERCOR - Multidisciplinary Oncology Cooperative Group #### Lead Sponsor **Class:** OTHER **Name:** University Hospital, Rouen #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** In patients with resectable pancreatic duct adenocarcinoma (PDAC), curative surgery followed by adjuvant chemotherapy is currently the standard of care. However, the long-term results are still poor, with median disease-free and overall survival of 14 months and 23 months. The corresponding 5-year overall survival rate is 20%. Chemotherapy before surgery (neoadjuvant chemotherapy) allows identification of patients with rapidly progressive metastatic disease at time of preoperative restaging (surgery is then avoided in these patients), and may increase the rate of free margin resection (R0) and reduce the risk of local recurrence. Even though single-agent gemcitabine and 5-FU have been validated in adjuvant and metastatic settings, the objective response was low (at around 10%), whereas combination chemotherapy exceeds a response rate of 30% in advanced disease. In metastatic PDAC, palliative FOLFIRINOX chemotherapy has been demonstrated to be effective (in terms of response rates and progression-free survival) and well tolerated. Interestingly, the response rate is increased by using more than two chemotherapeutic agents in advanced pancreatic cancer, justifying the use of an alternative neoadjuvant FOLFOX-based chemotherapy arm. PANACHE-01 is an open, non-comparative, randomised, multicentre Phase II study designed to assess the safety and efficacy of two modes of neo-adjuvant chemotherapy (FOLFIRINOX \& FOLFOX) relative to the current reference treatment (surgery and then adjuvant chemotherapy) for resectable PDAC. Patients with immediately resectable PDAC (definition based on the NCCN's (American National Comprehensive Cancer Network 2014) latest guidelines) will be randomised to either pancreatectomy and adjuvant chemotherapy or 4 cycles of neoadjuvant chemotherapy with either FOLFOX or FOLFIRINOX. The patients in the neoadjuvant chemotherapy arms will receive postoperative chemotherapy for 4 months (8 cycles). ### Conditions Module **Conditions:** - Resectable Pancreatic Duct Adenocarcinoma **Keywords:** - FOLFOX - FOLFIRINOX ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 160 **Type:** ESTIMATED **Phases:** - PHASE2 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** 4 cycles of FOLFOX neoadjuvant chemotherapy are administrated to patient. Curative surgery for resectable pancreatic duct adenocarcinoma will be done after neoadjuvant chemotherapy. 8 cycles of adjuvant chemotherapy are administrated following the surgery **Intervention Names:** - Drug: FOLFOX neoadjuvant chemotherapy - Procedure: curative surgery for resectable pancreatic duct adenocarcinoma - Drug: adjuvant chemotherapy **Label:** FOLFOX neoadjuvant chemotherapy **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** 4 cycles of FOLFIRINOX neoadjuvant chemotherapy are administrated to patient. Curative surgery for resectable pancreatic duct adenocarcinoma will be done after neoadjuvant chemotherapy. 8 cycles of adjuvant chemotherapy are administrated following the surgery **Intervention Names:** - Drug: FOLFIRINOX neoadjuvant chemotherapy - Procedure: curative surgery for resectable pancreatic duct adenocarcinoma - Drug: adjuvant chemotherapy **Label:** FOLFIRINOX neoadjuvant chemotherapy **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Curative surgery for resectable pancreatic duct adenocarcinoma will be done after randomization. 12 cycles of standard adjuvant chemotherapy are administrated following the surgery **Intervention Names:** - Procedure: curative surgery for resectable pancreatic duct adenocarcinoma - Drug: Standard adjuvant chemotherapy **Label:** standard adjuvant chemotherapy **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - FOLFOX neoadjuvant chemotherapy **Description:** 4 cycles of FOLFOX neoadjuvant chemotherapy are administrated to patient **Name:** FOLFOX neoadjuvant chemotherapy **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - FOLFIRINOX neoadjuvant chemotherapy **Description:** 4 cycles of FOLFIRINOX neoadjuvant chemotherapy are administrated to patient **Name:** FOLFIRINOX neoadjuvant chemotherapy **Type:** DRUG #### Intervention 3 **Arm Group Labels:** - FOLFIRINOX neoadjuvant chemotherapy - FOLFOX neoadjuvant chemotherapy - standard adjuvant chemotherapy **Description:** curative surgery for resectable pancreatic duct adenocarcinoma **Name:** curative surgery for resectable pancreatic duct adenocarcinoma **Type:** PROCEDURE #### Intervention 4 **Arm Group Labels:** - standard adjuvant chemotherapy **Description:** 12 cycles of standard adjuvant chemotherapy are administrated **Name:** Standard adjuvant chemotherapy **Type:** DRUG #### Intervention 5 **Arm Group Labels:** - FOLFIRINOX neoadjuvant chemotherapy - FOLFOX neoadjuvant chemotherapy **Description:** 8 cycles of standard adjuvant chemotherapy are administrated **Name:** adjuvant chemotherapy **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Number of patients alive is evaluated 12 months after the surgery **Measure:** Number of patients alive **Time Frame:** 12 months **Description:** The number of patients who achieved the complete chemotherapy treatment sequences is evaluated 12 months after the surgery **Measure:** Number of patients who achieved the complete chemotherapy treatment sequences **Time Frame:** 12 months #### Secondary Outcomes **Description:** Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 **Measure:** Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 **Time Frame:** through end of treatment, an average of 12 months **Description:** Evaluation of post-operative complications is assessed using Dindo Clavien classification **Measure:** Number of post-operative complications **Time Frame:** 1 month after surgery **Description:** Number of patients alive is evaluated 36 months after the surgery **Measure:** Number of patients alive and without recurrence **Time Frame:** 36 months **Description:** Number of accomplished R0 resection surgery is evaluated by pathologists **Measure:** Number of accomplished R0 resection surgery **Time Frame:** Surgery day **Description:** Evaluation of quality of life is done using EORTC QLQ C30 **Measure:** Evaluation of quality of life **Time Frame:** 4 weeks after the end of chemotherapy treatment **Description:** Evaluation of quality of life is done using EORTC QLQ-PAN26 **Measure:** Evaluation of quality of life **Time Frame:** 4 weeks after the end of chemotherapy treatment ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Histology-proven, adenocarcinoma of the pancreas. * Resectable adenocarcinoma (according to the NCCN classification 2014): absence of distant organ or distal lymph node metastases, absence of evidence of superior mesenteric vein (SMV) and portal vein distortion, tumour thrombus, or venous encasement, the existence of clear fat planes around the celiac axis, hepatic artery and superior mesenteric artery (SMA). Resectability is evaluated on arterial-phase and portal-phase IV contrast-enhanced multislice CT-scan of the pancreas (slice thickness: 2.5 mm), as evaluated in a multidisciplinary staff meeting including at least one radiologist and one expert surgeon. * No prior chemotherapy. * Age 18 years or over. * Ability to understand and willingness to consent to formal requirements for study participation * Provision of written informed consent prior to any study-specific screening procedures. Exclusion Criteria: * PDAC defined as "borderline", locally advanced, non-resectable or metastatic. * Prior cancer therapy for PDAC * Surgical or anaesthesiological contra-indications: non-controlled congestive heart failure - non-treated angina - recent myocardial infarction (in the previous year) - non-controlled arterial hypertension (SBP \>160 mm or DBP \> 100 mm, despite optimal drug treatment), long QT, major non-controlled infection, severe liver failure. * Any medical, psychological or social situation that (in the investigator's opinion) could limit (i) the patient's compliance with the protocol or (ii) the ability to obtain or interpret data. * Pregnant or breastfeeding women and women of child-bearing age not using effective means of contraception. * History or current evidence on physical examination of central nervous system disease or peripheral neuropathy ≥ grade 1, according to according to Common Terminology Criteria for Adverse Events (CTCAE) v.4.0. * Known hypersensitivity reaction to any of the components of study treatments. * Pregnancy (the absence of which must be confirmed in a ß-hCG test) or breast-feeding. * Any significant disease which, in the investigator's opinion, would exclude the patient from the study. * Patients having been included in a clinical trial within the previous 4 weeks or participating in another trial. **Maximum Age:** 85 Years **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Lilian SCHWARZ, MD **Phone:** +3323288 **Phone Ext:** 8265 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Julien BLOT **Role:** CONTACT #### Locations **Location 1:** **City:** Rouen **Contacts:** ***Contact 1:*** - **Name:** Lilian SCHWARZ, MD - **Role:** CONTACT ***Contact 2:*** - **Name:** Lilian SCHWARZ, MD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** France **Facility:** Rouen University Hospital **Status:** RECRUITING #### Overall Officials **Official 1:** **Affiliation:** Rouen University Hospital **Name:** Lilian SCHWARZ, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Schwarz L, Vernerey D, Bachet JB, Tuech JJ, Portales F, Michel P, Cunha AS. Resectable pancreatic adenocarcinoma neo-adjuvant FOLF(IRIN)OX-based chemotherapy - a multicenter, non-comparative, randomized, phase II trial (PANACHE01-PRODIGE48 study). BMC Cancer. 2018 Jul 24;18(1):762. doi: 10.1186/s12885-018-4663-4. Erratum In: BMC Cancer. 2020 Mar 3;20(1):168. **PMID:** 30041614 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000002277 - Term: Carcinoma - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000009369 - Term: Neoplasms - ID: D000018299 - Term: Neoplasms, Ductal, Lobular, and Medullary ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M25356 - Name: Carcinoma, Ductal - Relevance: HIGH - As Found: Duct Adenocarcinoma - ID: M3585 - Name: Adenocarcinoma - Relevance: HIGH - As Found: Adenocarcinoma - ID: M5534 - Name: Carcinoma - Relevance: LOW - As Found: Unknown - ID: M12320 - Name: Neoplasms, Glandular and Epithelial - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000000230 - Term: Adenocarcinoma - ID: D000044584 - Term: Carcinoma, Ductal ### Intervention Browse Module - Ancestors - 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: M271136 - Name: Folfirinox - Relevance: HIGH - As Found: Indocyanine Green ### Intervention Browse Module - Meshes - ID: C000627770 - Term: Folfirinox ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT01410279 **Brief Title:** Inspiratory Muscle Training in Pulmonary Hypertension **Official Title:** Inspiratory Muscle Training Effect on the Functional Capacity and the Quality of Life in Chronic Pulmonary Hypertension #### Organization Study ID Info **ID:** 09032 #### Organization **Class:** OTHER **Full Name:** Hospital de Clinicas de Porto Alegre ### Status Module #### Completion Date **Date:** 2011-12 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2011-08-05 **Type:** ESTIMATED **Last Update Submit Date:** 2011-08-04 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2011-09 **Type:** ESTIMATED #### Start Date **Date:** 2009-12 **Status Verified Date:** 2011-08 #### Study First Post Date **Date:** 2011-08-05 **Type:** ESTIMATED **Study First Submit Date:** 2011-04-20 **Study First Submit QC Date:** 2011-08-04 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Associação Fundo de Incentivo à Pesquisa **Class:** OTHER_GOV **Name:** Conselho Nacional de Desenvolvimento Científico e Tecnológico #### Lead Sponsor **Class:** OTHER **Name:** Hospital de Clinicas de Porto Alegre #### Responsible Party **Old Name Title:** Pedro Dal'Lago **Old Organization:** UFCSPA ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The purpose of this study is evaluate the effect the inspiratory muscle training on the functional capacity of patients with pulmonary hypertension. **Detailed Description:** Pulmonary hypertension (PH) is a syndrome, in the case of hemodynamic alteration of a number of diseases and processes, such as the progressive increase in pulmonary vascular resistance, right heart failure and early death. Frequently patients with PH have reduced functional capacity, quality of life and survival. It was demonstrated recently that patients with HP also have inspiratory muscle weakness, possibly further increase the fatigue and dyspnea during exercise. To meet this need, inspiratory muscle training can lead to significant improvement in respiratory muscle strength, exercise tolerance and dyspnea, thus resulting in reduction of discomfort during activities of daily living, increase in exercise capacity and improved quality of life in different situations. ### Conditions Module **Conditions:** - Pulmonary Hypertension **Keywords:** - pulmonary hypertension - inspiratory muscle training - inspiratory muscle weakness - pulmonary functional - functional capacity - quality of life ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 34 **Type:** ESTIMATED **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Description:** The load is adjusted weekly in thirty percent of maximal inspiratory pressure of the subject. **Name:** Inspiratory muscle training **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Measure:** The distance traveled in meters in the six minutes walk test after 8 weeks the inspiratory muscle training **Time Frame:** patients will be monitored throughout the training protocol for 8 weeks #### Secondary Outcomes **Measure:** Increase in score of quality of life questionnaire after 8 weeks the inspiratory muscle training **Time Frame:** patients will be monitored throughout the training protocol for 8 weeks ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Clinical diagnosis pulmonary hypertension * World Health Organization (WHO) functional class II to IV * inspiratory muscle weakness Exclusion Criteria: * obesity * chronic obstructive pulmonary disease * orthopedic and neurological disease **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:** Pedro Dal'Lago **Phone:** 55(51)3303-8796 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Glória M Ferreira **Phone:** 55(51)9284.0703 **Role:** CONTACT #### Locations **Location 1:** **City:** Porto Alegre **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Pedro Dal'Lago - **Phone:** +55(51)3303-8796 - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Glória M Ferreira - **Phone:** +55(51)9284-0703 - **Role:** CONTACT ***Contact 3:*** - **Name:** Pedro Dal'Lago - **Role:** PRINCIPAL_INVESTIGATOR ***Contact 4:*** - **Name:** Glória M Ferreira - **Role:** SUB_INVESTIGATOR ***Contact 5:*** - **Name:** Sérgio M Barreto - **Role:** SUB_INVESTIGATOR **Country:** Brazil **Facility:** Hospital de Clinica de Porto Alegre **State:** Rio Grande do Sul **Status:** RECRUITING **Zip:** 90035003 #### Overall Officials **Official 1:** **Affiliation:** Hospital de Clínicas de Porto Alegre **Name:** Pedro Dal'Lago **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Dall'Ago P, Chiappa GR, Guths H, Stein R, Ribeiro JP. Inspiratory muscle training in patients with heart failure and inspiratory muscle weakness: a randomized trial. J Am Coll Cardiol. 2006 Feb 21;47(4):757-63. doi: 10.1016/j.jacc.2005.09.052. Epub 2006 Jan 26. **PMID:** 16487841 **Citation:** Mereles D, Ehlken N, Kreuscher S, Ghofrani S, Hoeper MM, Halank M, Meyer FJ, Karger G, Buss J, Juenger J, Holzapfel N, Opitz C, Winkler J, Herth FF, Wilkens H, Katus HA, Olschewski H, Grunig E. Exercise and respiratory training improve exercise capacity and quality of life in patients with severe chronic pulmonary hypertension. Circulation. 2006 Oct 3;114(14):1482-9. doi: 10.1161/CIRCULATIONAHA.106.618397. Epub 2006 Sep 18. **PMID:** 16982941 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014652 - Term: Vascular Diseases - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000008171 - Term: Lung Diseases - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000012120 - Term: Respiration Disorders - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC14 - Name: Heart and Blood Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC05 - Name: Musculoskeletal Diseases - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: BXM - Name: Behaviors and Mental Disorders - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M10024 - Name: Hypertension - Relevance: HIGH - As Found: Hypertension - ID: M20944 - Name: Muscle Weakness - Relevance: LOW - As Found: Unknown - ID: M13204 - Name: Paresis - Relevance: LOW - As Found: Unknown - ID: M27137 - Name: Respiratory Aspiration - Relevance: HIGH - As Found: Inspiratory - ID: M4554 - Name: Asthenia - Relevance: LOW - As Found: Unknown - ID: M10027 - Name: Hypertension, Pulmonary - Relevance: HIGH - As Found: Pulmonary Hypertension - ID: M17400 - Name: Vascular 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: M14957 - Name: Respiration Disorders - Relevance: LOW - As Found: Unknown - ID: T6034 - Name: Quality of Life - Relevance: LOW - As Found: Unknown - ID: T1303 - Name: Chronic Graft Versus Host Disease - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000006976 - Term: Hypertension, Pulmonary - ID: D000053120 - Term: Respiratory Aspiration - ID: D000006973 - Term: Hypertension ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT00051779 **Brief Title:** An Investigational Drug (CAL) Versus Zoledronic Acid (Zometa®) in Patients With Breast Cancer **Official Title:** A 24-Week Blinded Study Conducted at Multiple Centers, Evaluating the Safety and Effectiveness of Various Doses of the Investigational Drug (CAL) Versus Zoledronic Acid (Zometa®) When Randomly Assigned to Patients With Breast Cancer That Has Metastasized to Bone #### Organization Study ID Info **ID:** CAL-03 #### Organization **Class:** INDUSTRY **Full Name:** Chugai Pharma USA ### Status Module #### Last Update Post Date **Date:** 2005-06-24 **Type:** ESTIMATED **Last Update Submit Date:** 2005-06-23 **Overall Status:** COMPLETED **Status Verified Date:** 2004-02 #### Study First Post Date **Date:** 2003-01-17 **Type:** ESTIMATED **Study First Submit Date:** 2003-01-16 **Study First Submit QC Date:** 2003-01-16 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Chugai Pharma USA ### Description Module **Brief Summary:** This study is intended to evaluate the safety, tolerability, and possible effectiveness of an investigational humanized monoclonal antibody (CAL) to the parathyroid hormone-related protein (PTHrP) when compared to zoledronic acid in patients with breast cancer metastatic to bone. The study will also evaluate the possible effects of both study drugs on performance status, markers of bone metabolism, and skeletal events related to bone metastasis including elevated blood calcium levels, bone pain, metastatic lesions, complications and interventions. The levels of CAL in the blood will also be evaluated. ### Conditions Module **Conditions:** - Breast Neoplasms - Breast Cancer - Metastasis **Keywords:** - CAL - Parathyroid hormone-related protein (PTHrP) - Breast Cancer - Bone Metastasis - Hypercalcemia ### Design Module #### Design Info **Primary Purpose:** TREATMENT **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module ### Interventions #### Intervention 1 **Name:** CAL **Type:** DRUG ### Eligibility Module **Eligibility Criteria:** Major Inclusion Criteria - Others Stipulated within the Protocol The study physician must assure you have/are: * Must be a female at least 18 years of age and be using an effective form of birth control. * A documented history of breast cancer and at least one bone metastasis that has not been previously treated by radiation or surgery, and is not anticipated to be treated within the next 24 weeks. * A total body bone scan and other radiographic scan(s) performed on you within 4 weeks prior to or during the screening period sufficient to image all sites of bone metastases. * You must be willing to perform a daily telephone diary and be willing to keep a paper diary and provide voluntary consent to participate in this study. Major Exclusion Criteria - Others Stipulated within the Protocol The study physician must assure you do not have/are not: * A change in analgesic (pain relief) type medication during the screening period (example, non-narcotic to narcotic). * Received radiation therapy to any bone metastasis or started a new course of chemotherapy within 3 weeks prior to the screening visit or during the screening period. * Used any bisphosphonate type drug during the 30 days prior to the anticipated first dose of study drug * Vertebral spine or weight-bearing bone metastasis that would place you at imminent risk for fracture or surgical intervention. * Evidence of active infection or immune deficiency, renal failure, abnormal liver function, or a serum calcium level \> 10.1 mg/dL. * Use of any investigational drug within 30 days prior to screening. **Minimum Age:** 18 Years **Sex:** FEMALE **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Little Rock **Country:** United States **Facility:** University of Arkansas for Medical Sciences **State:** Arkansas **Zip:** 72205 **Location 2:** **City:** Springdale **Country:** United States **Facility:** Highlands Oncology Group **State:** Arkansas **Zip:** 72764 **Location 3:** **City:** Concord **Country:** United States **Facility:** Bay Area Cancer Research Group **State:** California **Zip:** 94520 **Location 4:** **City:** Greenbrae **Country:** United States **Facility:** California Cancer Care, Inc. **State:** California **Zip:** 94904 **Location 5:** **City:** Los Angeles **Country:** United States **Facility:** Institute of Cancer Therapies **State:** California **Location 6:** **City:** Vista **Country:** United States **Facility:** San Diego Cancer Research Institute **State:** California **Zip:** 92083 **Location 7:** **City:** Aurora **Country:** United States **Facility:** Anschutz Cancer Pavilion at the University of Colorado Cancer Center **State:** Colorado **Zip:** 80010-0510 **Location 8:** **City:** New Haven **Country:** United States **Facility:** Yale University **State:** Connecticut **Zip:** 06520-8028 **Location 9:** **City:** Ft. Lauderdale **Country:** United States **Facility:** Holy Cross Hospital **State:** Florida **Zip:** 33308 **Location 10:** **City:** Chicago **Country:** United States **Facility:** Rush Cancer Institute **State:** Illinois **Zip:** 60612-3824 **Location 11:** **City:** Lexington **Country:** United States **Facility:** University of Kentucky Medical Center **State:** Kentucky **Zip:** 40536-0098 **Location 12:** **City:** New Orleans **Country:** United States **Facility:** Louisiana State University **State:** Louisiana **Zip:** 70112-2278 **Location 13:** **City:** Frederick **Country:** United States **Facility:** Frederick Memorial Hospital **State:** Maryland **Zip:** 21701 **Location 14:** **City:** Boston **Country:** United States **Facility:** Dana-Farber/Harvard Cancer Center **State:** Massachusetts **Zip:** 02115 **Location 15:** **City:** Detroit **Country:** United States **Facility:** Josephine Ford Cancer Center **State:** Michigan **Zip:** 48202 **Location 16:** **City:** Grand Rapids **Country:** United States **Facility:** Spectrum Health **State:** Michigan **Zip:** 49503 **Location 17:** **City:** Southfield **Country:** United States **Facility:** Southfield Oncology Institute, Inc. **State:** Michigan **Zip:** 48076 **Location 18:** **City:** St. Louis **Country:** United States **Facility:** St. Louis Center for Clinical Research **State:** Missouri **Zip:** 63128 **Location 19:** **City:** Las Vegas **Country:** United States **Facility:** Nevada Cancer Center **State:** Nevada **Zip:** 89109 **Location 20:** **City:** Lebanon **Country:** United States **Facility:** Dartmouth Hitchcock Medical Center **State:** New Hampshire **Zip:** 03756 **Location 21:** **City:** Santa Fe **Country:** United States **Facility:** New Mexico Cancer Care Associates **State:** New Mexico **Zip:** 87505 **Location 22:** **City:** Bronx **Country:** United States **Facility:** Montefiore Medical Center **State:** New York **Zip:** 10467-2490 **Location 23:** **City:** Brooklyn **Country:** United States **Facility:** HemOnCare, P.C. **State:** New York **Zip:** 11235 **Location 24:** **City:** New York **Country:** United States **Facility:** Memorial Sloan Kettering Cancer Center **State:** New York **Zip:** 10021 **Location 25:** **City:** Syracuse **Country:** United States **Facility:** SUNY Upstate Medical University **State:** New York **Zip:** 12310 **Location 26:** **City:** Durham **Country:** United States **Facility:** Duke University Medical Center **State:** North Carolina **Zip:** 27710 **Location 27:** **City:** Cleveland **Country:** United States **Facility:** Ireland Cancer Center **State:** Ohio **Zip:** 44106 **Location 28:** **City:** Cleveland **Country:** United States **Facility:** University Hospitals of Cleveland **State:** Ohio **Zip:** 44106 **Location 29:** **City:** Columbus **Country:** United States **Facility:** Hematology Oncology Consultants, Inc. **State:** Ohio **Zip:** 43235 **Location 30:** **City:** Hershey **Country:** United States **Facility:** Penn State Hershey Medical Center **State:** Pennsylvania **Zip:** 17033 **Location 31:** **City:** Columbia **Country:** United States **Facility:** Palmetto Health **State:** South Carolina **Zip:** 29203 **Location 32:** **City:** Memphis **Country:** United States **Facility:** Boston Baskin Cancer Group **State:** Tennessee **Zip:** 38104 **Location 33:** **City:** Memphis **Country:** United States **Facility:** The Boston Baskin Cancer Group **State:** Tennessee **Zip:** 38104 **Location 34:** **City:** Memphis **Country:** United States **Facility:** The West Clinic **State:** Tennessee **Zip:** 38120 **Location 35:** **City:** Corpus Christi **Country:** United States **Facility:** Cancer Specialists of South Texas, PA **State:** Texas **Zip:** 78412 **Location 36:** **City:** Dallas **Country:** United States **Facility:** Center for Oncology Research & Treatment **State:** Texas **Zip:** 75230 **Location 37:** **City:** Houston **Country:** United States **Facility:** Baylor College of Medicine **State:** Texas **Zip:** 77030 **Location 38:** **City:** Milwaukee **Country:** United States **Facility:** Medical College of Wisconsin-FMLH East Neoplastic Diseases and Related Disorders **State:** Wisconsin **Zip:** 53226 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009371 - Term: Neoplasms by Site - ID: D000009369 - Term: Neoplasms - ID: D000001941 - Term: Breast Diseases - ID: D000012871 - Term: Skin Diseases - ID: D000009385 - Term: Neoplastic Processes - ID: D000010335 - Term: Pathologic Processes ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC17 - Name: Skin and Connective Tissue Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases ### Condition Browse Module - Browse Leaves - ID: M5220 - Name: Breast Neoplasms - Relevance: HIGH - As Found: Breast Cancer - ID: M12307 - Name: Neoplasm Metastasis - Relevance: HIGH - As Found: Metastases - ID: M9985 - Name: Hypercalcemia - 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 - ID: M12330 - Name: Neoplastic Processes - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000001943 - Term: Breast Neoplasms - ID: D000009362 - Term: Neoplasm Metastasis ### 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: M9789 - Name: Hormones - Relevance: LOW - As Found: Unknown - ID: M1699 - Name: Zoledronic Acid - Relevance: LOW - As Found: Unknown - ID: M13194 - Name: Parathyroid Hormone - Relevance: LOW - As Found: Unknown - ID: M25279 - Name: Parathyroid Hormone-Related Protein - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05366179 **Brief Title:** Autologous CAR-T Cells Targeting B7-H3 in Recurrent or Refractory GBM CAR.B7-H3Tc **Official Title:** Phase I Study of Intraventricular Infusion of T Cells Expressing B7-H3 Specific Chimeric Antigen Receptors (CAR) in Subjects With Recurrent or Refractory Glioblastoma #### Organization Study ID Info **ID:** LCCC2059-ATL #### Organization **Class:** OTHER **Full Name:** UNC Lineberger Comprehensive Cancer Center ### Status Module #### Completion Date **Date:** 2030-05 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-04-23 **Type:** ACTUAL **Last Update Submit Date:** 2024-04-22 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2024-10-31 **Type:** ESTIMATED #### Start Date **Date:** 2022-09-02 **Type:** ACTUAL **Status Verified Date:** 2024-04 #### Study First Post Date **Date:** 2022-05-09 **Type:** ACTUAL **Study First Submit Date:** 2022-05-04 **Study First Submit QC Date:** 2022-05-04 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** UNC Lineberger Comprehensive Cancer Center #### 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 purpose of this study is to test the safety of using T lymphocyte chimeric antigen receptor cells against the B7-H3 antigen (CAR.B7-H3T cells) in patients with glioblastoma. CAR.B7-H3T cells treatment has not been tested in humans and is not an approved treatment by the Food and Drug Administration for glioblastoma. **Detailed Description:** This is a phase 1, single center, open-label study aims to determine the safety of escalating doses of chimeric antigen receptor T cells (CAR-T) cells targeting the B7-H3 antigen administered via intraventricular infusion to adult subjects with relapsed or refractory glioblastoma (GBM). Subjects with GBM who meet procurement eligibility criteria will have cells collected following their initial surgical resection to manufacture CAR.B7-H3T cells, preferably prior to initiation of adjuvant chemoradiation. At the time that the subjects have confirmed refractory or recurrent GBM, CAR.B7-H3T cells will be manufactured for subjects who likely to be eligible for cell infusion. Eligible subjects will receive up to 3 weekly infusions of CAR.B7-H3 cells. To receive the additional cycles of CAR.B7-H3 cells, all treatment-related toxicities must have resolved to grade \< 3, the subject must not have experienced a dose limiting toxicity (DLT) and the subject must not have progressive disease that has been confirmed by magnetic resonance imaging per Immunotherapy response assessment in neuro-oncology criteria. The data from the dose escalation will be used to determine a recommended phase 2 dose (RP2D), which will be decided on based on the maximum tolerated dose (MTD) and additional factors such as the feasibility of administering infusions. ### Conditions Module **Conditions:** - Glioblastoma Multiforme **Keywords:** - Brain Tumor - Glioblastoma Multiforme - Cell therapy - Relapse - Recurrent ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 36 **Type:** ESTIMATED **Phases:** - PHASE1 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** CAR.B7-H3 T cells: Subjects with refractory or recurrent glioblastoma multiforme, have cells collected following their initial surgical resection to manufacture CAR.B7-H3 T cells, preferably before initiation of adjuvant chemoradiation. **Intervention Names:** - Drug: CAR.B7-H3T cells infusion **Label:** Single Arm **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Single Arm **Description:** The Chimeric Antigen Receptors (CAR).B7-H3T cells will be administered via intraventricular infusion up to 3 weekly infusions. A 0.5 mL suspension of T cells infusion is given, over 5-10 minutes, via a Rickham catheter and will be followed by a normal-saline flush of 3-5 mL over 5-10 minutes. Dose escalation will be performed considering the dose limiting toxicities (DLTs) listed in the protocol. Six doses will be explored. The starting dose will be 2 × 10\^6 transduced cells/infusion (Dose Level (DL) 1) and will enroll at least 3 subjects. If there are no dose DLTs within 4 weeks of the third cellular product administration in the first 3 subjects, then the next cohort will evaluate 5 × 10\^6 transduced cells/infusion (DL2). **Name:** CAR.B7-H3T cells infusion **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Number of participants with adverse event (AE)s as a measure of safety and tolerability of intraventricular administration CAR.B7-H3 T cells in subjects with progressive recurrent or refractory glioblastoma multiforme. AEs will be classified and graded according to the National Cancer Institute's Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0. Dose Limiting Toxicities (DLTs) are defined as at least possibly related to CAR.B7-H3T cell product administration. **Measure:** Number of participants with adverse event **Time Frame:** Up to 10 weeks **Description:** Cytokine Release Syndrome (CRS) will be graded according to American Society for Transplantation and Cellular Therapy (ASTCT) CRS Consensus Grading. Grade 1 - Mild (Symptomatic Management): Fever ≥38\^ o C, No hypotension, No hypoxia, Grade 2 - Moderate (Moderate Intervention): Fever ≥38\^ o C, Hypotension not requiring vasopressors, Hypoxia requiring low-flow nasal cannula (≤6 L/minute) or blow-by, Grade 3 - Severe (Aggressive Intervention): Fever ≥ 38\^ o C , Hypotension requiring a vasopressor with or without vasopressin, Hypoxia requiring high-flow nasal cannula (\>6 L/minute), facemask, nonrebreather mask, or Venturi mask, Grade 4 - Life-threatening (Life-sustaining intervention): Fever ≥38\^oC, Hypotension requiring multiple vasopressors (excluding vasopressin),Hypoxia requiring positive pressure (e.g. Continuous positive airway pressure, BiPAP, intubation, mechanical ventilation), Grade 5 - Death: Death. **Measure:** Cytokine Release Syndrome **Time Frame:** Up to 10 weeks **Description:** Neurotoxicity will be graded according to the Central Nervous System (CNS) Toxicity criteria. Grade 0: Normal or no change from baseline exam at start of therapy, Grade 1: Mild lethargy and/or irritability or visual, motor, or sensory symptoms without change in neurological exam, Grade 2: Moderate lethargy, disorientation, or psychosis lasting \< 48 hours or mild increase in pre-existing neurological deficit, Grade 3: \>48hours of severe lethargy, but responsive to verbal stimuli or disorientation or psychosis lasting \>48 hours, Grade 4: Coma, unresponsive to verbal stimuli, increasing neurological deficit above grade 3, evidence of herniation, development of uncontrolled seizures, intracerebral hemorrhage. **Measure:** Neurotoxicity **Time Frame:** Up to 10 weeks #### Secondary Outcomes **Description:** RP2D for intraventricular administration of CAR.B7-H3 T cells is determined based on the modified 3+3 dose-finding rule. Dose escalation will be performed considering the dose-limiting toxicities (DLTs), dose level (DL) will be increased unless there is no DLT. **Measure:** Identification of Recommended phase 2 dose (RP2D) **Time Frame:** Up to 4 weeks **Description:** ORR is defined as the percentage of subjects with \[compete response (CR) + partial response (PR) + stable disease\] per Immunotherapy response assessment in neuro-oncology (iRANO) criteria. Complete response (CR): Disappearance of all enhancing disease for ≥ 4 weeks; no new lesions; stable or improved T2-weighted-fluid-attenuated inversion recovery (T2/FLAIR); no more than physiological steroids; clinically stable or improved. Partial response (PR): ≥ 50% decrease in the sum of biperpendicular diameters of enhancing disease for ≥ 4 weeks; no new lesions; stable or improved T2/FLAIR; stable or decreased steroid dose, clinically stable or improved. Stable disease (SD): Does not qualify for CR, PR or progressive disease; no new lesions; stable or improved T2/FLAIR; stable or decreased steroid dose; clinically stable or improved. **Measure:** Objective Response Rate (ORR) **Time Frame:** Up to 4 weeks **Description:** PFS is defined as the time from with the initial CAR.B7-H3 T cell infusion until disease progression per iRANO criteria or death as a result of any cause. Subjects who do not meet criteria for progression by the analysis cut-off date will be censored at their last evaluable disease assessment date. Immunotherapy response assessment in neuro-oncology (iRANO) criteria. Progressive disease (PD): ≥ 25% increase in the sum of biperpendicular diameters of enhancing disease; or new lesions; or substantial worsened T2/FLAIR; or substantial clinical decline. **Measure:** Progression Free Survival (PFS) **Time Frame:** Up to 12 months **Description:** OS is defined as the time from initial intraventricular infusion of CAR.B7-H3 T cells infusion to date of death for any cause **Measure:** Overall Survival (OS) **Time Frame:** Up to 5 years **Description:** DOR is defined as the time from documentation of PR or better to progressive disease (PD). **Measure:** Duration of Response (DOR) **Time Frame:** Up to 12 months ### Eligibility Module **Eligibility Criteria:** INCLUSION CRITERIA 1. Karnofsky score of \> 60% 2. Diagnosis of recurrent supratentorial- or infra-tentorial glioblastoma multiforme (GBM) (World Health Organization 2016 or 2021), based on Response assessment in neuro-oncology criteria (RANO) magnetic resonance imaging (MRI) criteria. Disseminated GBM down the spinal cord is not allowed. Must have previously undergone resection or biopsy at initial diagnosis. 3. Must have undergone at least 4005 cGy of radiation with concurrent temozolomide. 4. No current or previous exposure to antiangiogenic agents, such as bevacizumab. 5. Female subjects of childbearing potential must be willing to abstain from heterosexual activity or to use 2 forms of effective methods of contraception from the time of informed consent until 6 months after study treatment discontinuation. 6. Male subjects with female partners must have had a prior vasectomy or agree to use an adequate method of contraception starting with the first dose of study therapy through 3 months after the cell infusion therapy. If a male subject receives multiple infusions, they must remain on contraception throughout the duration and 3 months after the last cell infusion therapy. 7. The subject is willing and able to comply with study procedures based on the judgment of the investigator. EXCLUSION CRITERIA 1. Subject is pregnant or lactating (Note: Breast milk cannot be stored for future use while the mother is being treated on study). 2. Subjects with a prior or concurrent malignancy whose natural history or treatment has the potential to interfere with the safety or efficacy assessment of the investigational regimen. 3. Active infection with HIV, hepatitis B virus, hepatitis C virus (HCV). Note: To meet eligibility subjects are required to be negative for HIV antibody, negative for HTLV1 and 2 antibodies, negative for Hepatitis B surface antigen, and negative for HCV antibody and viral load. 4. Contraindication to MRI contrast agents or an inability to undergo MRI scans due to MRI non-compatible implanted materials. 5. Prior exposure to chimeric antigen receptor T cell therapy for treatment of glioblastoma. 6. Evidence of disseminated disease involving the brainstem, cerebellum or spinal cord. 7. Previously implanted carmustine wafers or brachytherapy for the treatment of glioma. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Catherine Cheng **Phone:** 919-445-4208 **Role:** CONTACT **Contact 2:** **Email:** [email protected]@med.unc.edu **Name:** Caroline Babinec **Phone:** 919-962-7426 **Role:** CONTACT #### Locations **Location 1:** **City:** Chapel Hill **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Catherine Cheng - **Phone:** 919-445-4208 - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Caroline Babinec - **Phone:** 919-962-7426 - **Role:** CONTACT ***Contact 3:*** - **Name:** Yasmeen Rauf, MD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Lineberger Comprehensive Cancer Center **State:** North Carolina **Status:** RECRUITING **Zip:** 27514 #### Overall Officials **Official 1:** **Affiliation:** UNC Lineberger Comprehensive Cancer Center **Name:** Yasmeen Rauf, MD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### See Also Links **Label:** University of North Carolina Lineberger Comprehensive Cancer Center Clinical Trials **URL:** http://unclineberger.org/patientcare/clinical-trials/clinical-trials ## Derived Section ### Condition Browse Module - Ancestors - ID: D000001254 - Term: Astrocytoma - ID: D000005910 - Term: Glioma - ID: D000018302 - Term: Neoplasms, Neuroepithelial - ID: D000017599 - Term: Neuroectodermal Tumors - ID: D000009373 - Term: Neoplasms, Germ Cell and Embryonal - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000009369 - Term: Neoplasms - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009380 - Term: Neoplasms, Nerve Tissue ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC04 - Name: Neoplasms - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M14850 - Name: Recurrence - Relevance: LOW - As Found: Unknown - ID: M9019 - Name: Glioblastoma - Relevance: HIGH - As Found: Glioblastoma - ID: M5209 - Name: Brain Neoplasms - Relevance: LOW - As Found: Unknown - ID: M4561 - Name: Astrocytoma - Relevance: LOW - As Found: Unknown - ID: M9020 - Name: Glioma - Relevance: LOW - As Found: Unknown - ID: M20446 - Name: Neoplasms, Neuroepithelial - Relevance: LOW - As Found: Unknown - ID: M19845 - Name: Neuroectodermal Tumors - Relevance: LOW - As Found: Unknown - ID: M20388 - Name: Neuroectodermal Tumors, Primitive - Relevance: LOW - As Found: Unknown - ID: M12318 - Name: Neoplasms, Germ Cell and Embryonal - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown - ID: M12320 - Name: Neoplasms, Glandular and Epithelial - Relevance: LOW - As Found: Unknown - ID: M12325 - Name: Neoplasms, Nerve Tissue - Relevance: LOW - As Found: Unknown - ID: T2518 - Name: Glioblastoma - Relevance: HIGH - As Found: Glioblastoma - ID: T2519 - Name: Glioma - Relevance: LOW - As Found: Unknown - ID: T4092 - Name: Neuroepithelioma - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000005909 - Term: Glioblastoma ### 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: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05285579 **Acronym:** ANGIOCOR **Brief Title:** Biomarkers of Angiogenesis for Response to Therapeutic Combination in Advanced or Metastatic Kidney Cancer **Official Title:** Predictive Role of Circulating Biomarkers Involved in Angiogenesis in Metastatic Kidney Cancer in the Era of New Therapeutic Associations: Immunotherapies, Anti-angiogenic #### Organization Study ID Info **ID:** APHP211213 #### Organization **Class:** OTHER **Full Name:** Assistance Publique - Hôpitaux de Paris #### Secondary ID Infos **Domain:** Agence nationale de sécurité du médicament et des produits de santé **ID:** IDRCB2021-A02030-41 **Type:** REGISTRY ### Status Module #### Completion Date **Date:** 2025-08 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2023-04-19 **Type:** ACTUAL **Last Update Submit Date:** 2023-04-14 **Overall Status:** RECRUITING #### Primary Completion Date **Date:** 2025-08 **Type:** ESTIMATED #### Start Date **Date:** 2022-05-05 **Type:** ACTUAL **Status Verified Date:** 2023-04 #### Study First Post Date **Date:** 2022-03-17 **Type:** ACTUAL **Study First Submit Date:** 2022-03-09 **Study First Submit QC Date:** 2022-03-09 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER_GOV **Name:** Institut National de la Santé Et de la Recherche Médicale, France **Class:** UNKNOWN **Name:** FONCER contre le cancer #### Lead Sponsor **Class:** OTHER **Name:** Assistance Publique - Hôpitaux de Paris #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** This is a multicenter, exploratory, prospective study to identify angiogenesis and immune-related biomarkers predictive of progression free survival in patients with metastatic or advanced renal cell carcinoma treated by a combination of immunotherapy and antiangiogenic. **Detailed Description:** Recently, the management of renal cell carcinoma has undergone major changes with the emergence of combined therapies associating tyrosine kinase inhibitors (TKI) and immune checkpoint inhibitors (ICI) as first line treatments. However, there are no criteria to guide the choice between the different combinations validated and or between ICI combinations. Angiogenesis and immunity are intimately linked and some markers related have could be interesting to predict the efficacy of these combinations. Angiogenesis and immunity are highly related. This link may lead to new biomarkers to be explored to predict the response to TKI + ICI therapy combinations. On this basis, the investigators propose to conduct an open-label exploratory, multicenter prospective trial to study the association between angiogenesis and immune markers and the effect of combined TKI+ICI treatments. ### Conditions Module **Conditions:** - Renal Cell Carcinoma - Renal Cancer - Renal Cancer Metastatic **Keywords:** - renal cell carcinoma - blood biomarkers - predictive factor - tyrosine kinase inhibitor - immune checkpoint inhibitor ### Design Module #### Bio Spec **Description:** Plasma + tumour tissue **Retention:** SAMPLES_WITHOUT_DNA #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 100 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Therapeutic combination tyrosine kinase inhibitor (TKI) + immune checkpoint inhibitors (ICI) **Intervention Names:** - Biological: Blood collection - Other: Tumour samples **Label:** TKI+ICI #### Arm Group 2 **Description:** Therapeutic combination with different immune checkpoint inhibitors (ICI) **Intervention Names:** - Biological: Blood collection - Other: Tumour samples **Label:** ICI+ICI ### Interventions #### Intervention 1 **Arm Group Labels:** - ICI+ICI - TKI+ICI **Description:** Systematic extra blood sampling at inclusion, 6 weeks after inclusion and in case of progression of the cancer **Name:** Blood collection **Type:** BIOLOGICAL #### Intervention 2 **Arm Group Labels:** - ICI+ICI - TKI+ICI **Description:** reuse of tumour tissue collect in usual patient care **Name:** Tumour samples **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Description:** Time from inclusion to progression documented by imaging and based on RECIST 1.1 and iRECIST criteria or patient death. The iRECIST criteria use the same methods of monitoring tumor lesions as the RECIST 1.1 criteria but a confirmation 4-6 weeks after suspicion of progression is required to confirm or rule out progression because patients undergoing immunotherapy may present pseudo-progressions. **Measure:** Progression-free survival **Time Frame:** 24 months #### Secondary Outcomes **Description:** Observation of a partial or complete response according to RECIST 1.1 criteria during the follow-up **Measure:** Objective response rate **Time Frame:** 24 months **Description:** Time between the observation of an objective response (partial or complete according to RECIST 1.1 criteria) and the progression **Measure:** Response duration **Time Frame:** 24 months ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Histologically proven advanced or metastatic renal carcinoma * treated in first line with an ICI-ICI or ITK-ICI combination (following current recommendations at inclusion) Exclusion Criteria: * Previous systemic treatment for renal cell carcinoma * Other cancer developed in the last 5 years except local forms apparently healed as basal cell cancer. * Contraindication for ICI-ICI or TKI-ICI combinations recommended on 1st line * Refusal to participate in the study * No affiliation to a social security regime (beneficiary or entitled) * Vulnerable patients as defined by french law (Public Heath Code sections L1121 -5 to L1121-8) : * Major patient subjected to legal protection (guardianship, curatorship, protection of justice) * Pregnant or breastfeeding woman **Minimum Age:** 18 Years **Sampling Method:** PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Patients with a metastatic or advanced renal cell carcinoma who will benefit of an ICI-TKI or ICI-ICI combination as a first-line treatment (according to the current guidelines at the time of inclusion). ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Natacha Nohilé **Phone:** 33156095982 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Laetitia MAUGE, PharmD, PhD **Phone:** 33156093905 **Role:** CONTACT #### Locations **Location 1:** **City:** Paris **Contacts:** ***Contact 1:*** - **Name:** Marie AUVRAY-KUNITZ, MD - **Role:** CONTACT ***Contact 2:*** - **Name:** Marie AUVRAY-KUNITZ, MD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** France **Facility:** Hôpital européen Georges-Pompidou AP-HP **Status:** RECRUITING **Zip:** 75015 **Location 2:** **City:** Paris **Contacts:** ***Contact 1:*** - **Name:** Olivier HUILLARD, MD - **Role:** CONTACT ***Contact 2:*** - **Name:** Olivier HUILLARD, MD, PhD - **Role:** PRINCIPAL_INVESTIGATOR **Country:** France **Facility:** Hôpital Cochin - AP-HP **Status:** RECRUITING #### Overall Officials **Official 1:** **Affiliation:** Assitance Puplique - Hôpitaux de Paris **Name:** Laetitia MAUGE, PharmD, PhD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Access Criteria:** Data sharing must be accepted by the sponsor and the PI based on a scientific project and scientific involvement of the PI team. Collaboration will be fostered. Data sharing must respect the agreements made with funders. Teams wishing obtain IPD must meet the sponsor and IP team to present scientific (and commercial) purpose, IPD needed, format of data transmission, and timeframe. Technical feasibility and financial support will be discussed before mandatory contractual agreement. Processing of shared data must comply with European General Data Protection Regulation (GDPR). **Description:** Individual participant data (IPD) that underlie results in publication could be shared. IPD detailed in the protocol of a planned metaanalysis could be shared **Info Types:** - STUDY_PROTOCOL - ICF **IPD Sharing:** YES **Time Frame:** Two years after the last publication ## Derived Section ### Condition Browse Module - Ancestors - ID: D000002277 - Term: Carcinoma - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009370 - Term: Neoplasms by Histologic Type - ID: D000009369 - Term: Neoplasms - ID: D000000230 - Term: Adenocarcinoma - ID: D000014571 - Term: Urologic Neoplasms - ID: D000014565 - Term: Urogenital Neoplasms - ID: D000009371 - Term: Neoplasms by Site - ID: D000052776 - Term: Female Urogenital Diseases - ID: D000005261 - Term: Female Urogenital Diseases and Pregnancy Complications - ID: D000091642 - Term: Urogenital Diseases - ID: D000007674 - Term: Kidney Diseases - ID: D000014570 - Term: Urologic Diseases - ID: D000052801 - Term: Male Urogenital Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC04 - Name: Neoplasms - Abbrev: All - Name: All Conditions - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M5534 - Name: Carcinoma - Relevance: LOW - As Found: Unknown - ID: M5548 - Name: Carcinoma, Renal Cell - Relevance: HIGH - As Found: Renal Cancer - ID: M10703 - Name: Kidney Neoplasms - Relevance: HIGH - As Found: Renal Cancer - ID: M12320 - Name: Neoplasms, Glandular and Epithelial - Relevance: LOW - As Found: Unknown - ID: M12315 - Name: Neoplasms by Histologic Type - Relevance: LOW - As Found: Unknown - ID: M3585 - Name: Adenocarcinoma - Relevance: LOW - As Found: Unknown - ID: M17320 - Name: Urologic Neoplasms - Relevance: LOW - As Found: Unknown - ID: M17315 - Name: Urogenital Neoplasms - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M10698 - Name: Kidney Diseases - Relevance: LOW - As Found: Unknown - ID: M17319 - Name: Urologic Diseases - Relevance: LOW - As Found: Unknown - ID: M27095 - Name: Male Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: T4906 - Name: Renal Cell Carcinoma - Relevance: HIGH - As Found: Renal Cell Carcinoma - ID: T1341 - Name: Clear Cell Renal Cell Carcinoma - Relevance: HIGH - As Found: Renal Cell Carcinoma ### Condition Browse Module - Meshes - ID: D000002292 - Term: Carcinoma, Renal Cell - ID: D000007680 - Term: Kidney Neoplasms ### 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: M22318 - Name: Angiogenesis Inhibitors - Relevance: LOW - As Found: Unknown - ID: M2853 - Name: Immunomodulating Agents - Relevance: LOW - As Found: Unknown - ID: M2342 - Name: Immune Checkpoint Inhibitors - Relevance: LOW - As Found: Unknown - ID: M2889 - Name: Tyrosine Kinase Inhibitors - Relevance: LOW - As Found: Unknown - ID: T22 - Name: Tyrosine - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT05929079 **Acronym:** TRIUMPH-2 **Brief Title:** A Study of Retatrutide (LY3437943) in Participants With Type 2 Diabetes Mellitus Who Have Obesity or Overweight **Official Title:** A Master Protocol to Investigate the Efficacy and Safety of LY3437943 Once Weekly in Participants With Type 2 Diabetes Mellitus Who Have Obesity or Overweight: A Randomized Double-Blind, Placebo-Controlled Trial #### Organization Study ID Info **ID:** 18558 #### Organization **Class:** INDUSTRY **Full Name:** Eli Lilly and Company #### Secondary ID Infos **Domain:** Master Protocol Eli Lilly and Company **ID:** J1I-MC-GZBK **Type:** OTHER **Domain:** ISA Eli Lilly and Company **ID:** J1I-MC-GSA2 **Type:** OTHER **Domain:** EU Trial Number **ID:** 2023-503658-11-00 **Type:** OTHER ### 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:** RECRUITING #### Primary Completion Date **Date:** 2026-05-03 **Type:** ESTIMATED #### Start Date **Date:** 2023-07-11 **Type:** ACTUAL **Status Verified Date:** 2024-04-01 #### Study First Post Date **Date:** 2023-07-03 **Type:** ACTUAL **Study First Submit Date:** 2023-06-26 **Study First Submit QC Date:** 2023-06-26 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** INDUSTRY **Name:** Eli Lilly and Company #### 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 purpose of this study is to is to evaluate the efficacy and safety of retatrutide in participants with type 2 diabetes in participants who have obesity or overweight (J1I-MC-GZBK master protocol) including a subset of participants who have obstructive sleep apnea (OSA) (J1I-MC-GSA2). The study will last about 89 weeks and will include up to 24 visits. ### Conditions Module **Conditions:** - Type 2 Diabetes - Obesity - Overweight - Obstructive Sleep Apnea ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** DOUBLE **Who Masked:** - PARTICIPANT - INVESTIGATOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 1000 **Type:** ESTIMATED **Phases:** - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants will receive retatrutide subcutaneously (SC). **Intervention Names:** - Drug: Retatrutide **Label:** Retatrutide Dose 1 **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants will receive retatrutide SC. **Intervention Names:** - Drug: Retatrutide **Label:** Retatrutide Dose 2 **Type:** EXPERIMENTAL #### Arm Group 3 **Description:** Participants will receive retatrutide SC. **Intervention Names:** - Drug: Retatrutide **Label:** Retatrutide Dose 3 **Type:** EXPERIMENTAL #### Arm Group 4 **Description:** Participants will receive placebo. **Intervention Names:** - Drug: Placebo **Label:** Placebo **Type:** PLACEBO_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Retatrutide Dose 1 - Retatrutide Dose 2 - Retatrutide Dose 3 **Description:** Administered SC **Name:** Retatrutide **Other Names:** - LY3437943 **Type:** DRUG #### Intervention 2 **Arm Group Labels:** - Placebo **Description:** Administered SC **Name:** Placebo **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Measure:** Percent Change from Baseline in Body Weight **Time Frame:** Baseline, Week 80 **Measure:** Change from Baseline in Apnea-Hypopnea Index (AHI) for GSA2 Subset **Time Frame:** Baseline, Week 80 #### Secondary Outcomes **Measure:** Change from Baseline in Body Mass Index (BMI) **Time Frame:** Baseline, Week 80 **Measure:** Change from Baseline in Waist Circumference **Time Frame:** Baseline, Week 80 **Measure:** Change from Baseline in Systolic Blood Pressure (SBP) **Time Frame:** Baseline, Week 80 **Measure:** Change from Baseline in Diastolic Blood Pressure (DBP) **Time Frame:** Baseline, Week 80 **Measure:** Percent Change from Baseline in Total Cholesterol **Time Frame:** Baseline, Week 80 **Measure:** Percent Change from Baseline in Triglycerides **Time Frame:** Baseline, Week 80 **Measure:** Change from Baseline in Hemoglobin (A1c) HbA1c % **Time Frame:** Baseline, Week 80 **Measure:** Change from Baseline in Short Form 36 Version 2 (SF-36v2) Acute Form Physical Functioning Domain Score **Time Frame:** Baseline, Week 80 **Measure:** Change from Baseline in Fasting Glucose **Time Frame:** Baseline, Week 80 **Description:** AUC is presented as a single average measure of AUC across the study duration. **Measure:** Pharmacokinetics (PK): Steady State Area Under the Concentration Time Curve (AUC) **Time Frame:** Baseline through Week 80 **Measure:** Percent Change from Baseline in Apnea-Hypopnea Index (AHI) for GSA2 Subset **Time Frame:** Baseline, Week 80 **Description:** A hierarchical combination of change from baseline in the FOSQ 10 score, FOSQ Vigilance Domain Score, and FOSQ Activity Level Domain Score will be assessed by the win ratio. The reported unit will be the total "wins" for each treatment group from performing a hierarchical comparison of the component. **Measure:** A Hierarchical Combination of Functional Outcomes of Sleep Questionnaire (FOSQ) 10 score, FOSQ Vigilance Domain Score, and FOSQ Activity Level Domain Score for GSA2 Subset **Time Frame:** Baseline to Week 80 **Measure:** Percentage of Participants with ≥50% AHI Reduction from Baseline for GSA2 Subset **Time Frame:** Baseline to Week 80 **Measure:** Percentage of Participants with AHI <5 or with AHI 5-14 with Epworth Sleepiness Scale (ESS) ≤10 for GSA2 Subset **Time Frame:** Week 80 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Have a body mass index (BMI) greater than or equal to 27.0 kilogram/square meter (kg/m ²) * Have Type 2 Diabetes (T2D) * Are on stable treatment for T2D for at least 90 days * Have a history of at least one unsuccessful dietary effort to lose body weight. GSA2 Inclusion Criteria * Previously diagnosed with OSA * Have AHI ≥15 on polysomnography at screening (definition of moderate-to-severe OSA) * For participants not on positive airway pressure (PAP) therapy: unable or unwilling to use PAP therapy and have not used PAP for at least 4 weeks prior to screening. * If on PAP therapy, have been on PAP therapy for at least 3 consecutive months prior to screening, and willing to temporarily stop using PAP therapy for approximately 7 days prior to each of the sleep study (PSG) visits. Exclusion Criteria: * Have a self-reported or documented change in body weight \>5 kg (11 pounds) within 90 days. * Have taken weight loss drugs, including over-the-counter medications, within 90 days prior to screening. * Have a prior or planned surgical treatment for obesity. * Have Type 1 diabetes * Have a family or personal history of medullary thyroid carcinoma (MTC) or multiple endocrine neoplasia syndrome type 2 (MEN-2) * Have had pancreatitis GSA2 Exclusion Criteria * Use stimulants (for example, modafinil, armodafinil, solriamfetol, pitolisant, amphetamine) less than 3 months prior to screening. * Use hypnotics, mirtazapine, opioids, trazodone, and zonisamide less than 3 months prior to screening. * Use a dental appliance or other device to treat OSA other than PAP therapy. **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** There may be multiple sites in this clinical trial. 1-877-CTLILLY (1-877-285-4559) or **Phone:** 1-317-615-4559 **Role:** CONTACT #### Locations **Location 1:** **City:** Anniston **Contacts:** ***Contact 1:*** - **Name:** Almena Free - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Pinnacle Research Group, LLC **State:** Alabama **Status:** NOT_YET_RECRUITING **Zip:** 36207 **Location 2:** **City:** Los Angeles **Contacts:** ***Contact 1:*** - **Phone:** 213-483-1800 - **Role:** CONTACT ***Contact 2:*** - **Name:** David Guzman - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** National Research Institute - Wilshire **State:** California **Status:** RECRUITING **Zip:** 90057 **Location 3:** **City:** Northridge **Contacts:** ***Contact 1:*** - **Name:** Christopher Chow - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Valley Clinical Trials, Inc. **State:** California **Status:** RECRUITING **Zip:** 91325 **Location 4:** **City:** Walnut Creek **Contacts:** ***Contact 1:*** - **Phone:** 925-930-7267 - **Role:** CONTACT ***Contact 2:*** - **Name:** Helen L Stacey - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Diablo Clinical Research, Inc. **State:** California **Status:** RECRUITING **Zip:** 94598 **Location 5:** **City:** Waterbury **Contacts:** ***Contact 1:*** - **Phone:** 203-419-4420 - **Role:** CONTACT ***Contact 2:*** - **Name:** Joseph Soufer - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Chase Medical Research, LLC **State:** Connecticut **Status:** RECRUITING **Zip:** 06708 **Location 6:** **City:** Brandon **Contacts:** ***Contact 1:*** - **Phone:** 813-758-6613 - **Role:** CONTACT ***Contact 2:*** - **Name:** Daniel G. Lorch - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Teradan Clinical Trials, LLC **State:** Florida **Status:** RECRUITING **Zip:** 33511 **Location 7:** **City:** Fleming Island **Contacts:** ***Contact 1:*** - **Name:** Mitchell Rothstein - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Fleming Island Center for Clinical Research **State:** Florida **Status:** NOT_YET_RECRUITING **Zip:** 32003 **Location 8:** **City:** Ocala **Contacts:** ***Contact 1:*** - **Phone:** 352-629-5800 - **Role:** CONTACT ***Contact 2:*** - **Name:** Raj Karunakara - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Renstar Medical Research **State:** Florida **Status:** RECRUITING **Zip:** 34470 **Location 9:** **City:** West Palm Beach **Contacts:** ***Contact 1:*** - **Phone:** 561-689-0606 - **Role:** CONTACT ***Contact 2:*** - **Name:** Mira Baron - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Palm Beach Research Center **State:** Florida **Status:** RECRUITING **Zip:** 33409 **Location 10:** **City:** Atlanta **Contacts:** ***Contact 1:*** - **Phone:** 4048519934 - **Role:** CONTACT ***Contact 2:*** - **Name:** Dennis Michael Lacey - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** NeuroTrials Research Inc **State:** Georgia **Status:** RECRUITING **Zip:** 30328 **Location 11:** **City:** Honolulu **Contacts:** ***Contact 1:*** - **Phone:** 8085316886 - **Role:** CONTACT ***Contact 2:*** - **Name:** Cindy H. T. Pau - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** East-West Medical Research Institute **State:** Hawaii **Status:** RECRUITING **Zip:** 96814 **Location 12:** **City:** Indianapolis **Contacts:** ***Contact 1:*** - **Name:** Blair Brengle - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Brengle Family Medicine **State:** Indiana **Status:** RECRUITING **Zip:** 46260 **Location 13:** **City:** Waltham **Contacts:** ***Contact 1:*** - **Phone:** 617-744-1310 - **Role:** CONTACT ***Contact 2:*** - **Name:** David DiBenedetto - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** MedVadis Research Corporation **State:** Massachusetts **Status:** RECRUITING **Zip:** 02451 **Location 14:** **City:** Saint Peters **Contacts:** ***Contact 1:*** - **Phone:** 636-387-5100 - **Role:** CONTACT ***Contact 2:*** - **Name:** Timothy R Smith - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** StudyMetrix Research **State:** Missouri **Status:** RECRUITING **Zip:** 63303 **Location 15:** **City:** Las Vegas **Contacts:** ***Contact 1:*** - **Phone:** 702-750-0222 - **Role:** CONTACT ***Contact 2:*** - **Name:** Cornell Calinescu - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Las Vegas Medical Research **State:** Nevada **Status:** RECRUITING **Zip:** 89113 **Location 16:** **City:** Monroe **Contacts:** ***Contact 1:*** - **Name:** Awawu Igbinadolor - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Monroe Biomedical Research **State:** North Carolina **Status:** NOT_YET_RECRUITING **Zip:** 28112 **Location 17:** **City:** Morehead City **Contacts:** ***Contact 1:*** - **Phone:** 2524998623 - **Role:** CONTACT ***Contact 2:*** - **Name:** Mary Katherine Lawrence - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Carteret Medical Group **State:** North Carolina **Status:** RECRUITING **Zip:** 28557 **Location 18:** **City:** Fargo **Contacts:** ***Contact 1:*** - **Name:** Michael J Lillestol - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Lillestol Research **State:** North Dakota **Status:** RECRUITING **Zip:** 58104 **Location 19:** **City:** Cincinnati **Contacts:** ***Contact 1:*** - **Phone:** 5135336325 - **Role:** CONTACT ***Contact 2:*** - **Name:** James Paul Maynard - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** CTI-CRC **State:** Ohio **Status:** RECRUITING **Zip:** 45212 **Location 20:** **City:** Cincinnati **Contacts:** ***Contact 1:*** - **Name:** Bruce Corser - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Intrepid Research **State:** Ohio **Status:** NOT_YET_RECRUITING **Zip:** 45245 **Location 21:** **City:** Pittsburgh **Contacts:** ***Contact 1:*** - **Phone:** 412-650-6155 - **Role:** CONTACT ***Contact 2:*** - **Name:** Bryce A. Palchick - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Office 18 **State:** Pennsylvania **Status:** RECRUITING **Zip:** 15236 **Location 22:** **City:** Uniontown **Contacts:** ***Contact 1:*** - **Name:** Paul Hartley - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Preferred Primary Care Physicians **State:** Pennsylvania **Status:** RECRUITING **Zip:** 15401 **Location 23:** **City:** Greenville **Contacts:** ***Contact 1:*** - **Phone:** 864-334-0141 - **Role:** CONTACT ***Contact 2:*** - **Name:** Ronald Mayfield - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Tribe Clinical Research, LLC **State:** South Carolina **Status:** RECRUITING **Zip:** 29607 **Location 24:** **City:** Myrtle Beach **Contacts:** ***Contact 1:*** - **Name:** Donald Charles Eagerton - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Trial Management Associates **State:** South Carolina **Status:** RECRUITING **Zip:** 29572 **Location 25:** **City:** Spartanburg **Contacts:** ***Contact 1:*** - **Name:** Christopher Michael Davis - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Tribe Clinical Research - Spartanburg **State:** South Carolina **Status:** RECRUITING **Zip:** 29301 **Location 26:** **City:** Austin **Contacts:** ***Contact 1:*** - **Name:** John Douglas Hudson - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** FutureSearch Trials of Neurology **State:** Texas **Status:** RECRUITING **Zip:** 78731 **Location 27:** **City:** Dallas **Contacts:** ***Contact 1:*** - **Phone:** 972-566-7799 - **Role:** CONTACT ***Contact 2:*** - **Name:** Julio Rosenstock - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Dallas Diabetes Research Center **State:** Texas **Status:** RECRUITING **Zip:** 75230 **Location 28:** **City:** Ogden **Contacts:** ***Contact 1:*** - **Phone:** 801-409-2040 - **Role:** CONTACT ***Contact 2:*** - **Name:** Michael D Woolman - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Advanced Research Institute **State:** Utah **Status:** RECRUITING **Zip:** 84405 **Location 29:** **City:** Bellevue **Contacts:** ***Contact 1:*** - **Name:** Nazia Rahman - **Role:** PRINCIPAL_INVESTIGATOR **Country:** United States **Facility:** Northwest Clinical Research Center **State:** Washington **Status:** RECRUITING **Zip:** 98007 **Location 30:** **City:** Buenos Aires **Contacts:** ***Contact 1:*** - **Name:** Diego Aizenberg - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Argentina **Facility:** Centro Médico Viamonte **State:** Ciudad Autónoma De Buenos Aires **Status:** RECRUITING **Zip:** C1120AAC **Location 31:** **City:** Ciudad Autonoma de Buenos Aires **Contacts:** ***Contact 1:*** - **Name:** Pablo Rene Costanzo - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Argentina **Facility:** Consultorio de Investigación Clínica EMO SRL **State:** Ciudad Autónoma De Buenos Aires **Status:** RECRUITING **Zip:** 1405 **Location 32:** **City:** Ciudad Autonoma de Buenos Aires **Contacts:** ***Contact 1:*** - **Name:** Maria Jimena Coronel - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Argentina **Facility:** Centro Medico Dra. Laura Maffei- Investigacion Clinica Aplicada **State:** Ciudad Autónoma De Buenos Aires **Status:** RECRUITING **Zip:** C1425AGC **Location 33:** **City:** Santa Rosa **Contacts:** ***Contact 1:*** - **Name:** Marcos Alejandro Mayer - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Argentina **Facility:** Centro de Salud e Investigaciones Médicas **State:** La Pampa **Status:** RECRUITING **Zip:** 6300 **Location 34:** **City:** Botany **Contacts:** ***Contact 1:*** - **Name:** Paul Bird - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Australia **Facility:** Optimus Clinical Research **State:** New South Wales **Status:** NOT_YET_RECRUITING **Zip:** 2019 **Location 35:** **City:** Merewether **Contacts:** ***Contact 1:*** - **Phone:** 61249632323 - **Role:** CONTACT ***Contact 2:*** - **Name:** Claire Morbey - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Australia **Facility:** The AIM Centre / Hunter Diabetes Centre **State:** New South Wales **Status:** RECRUITING **Zip:** 2291 **Location 36:** **City:** Sydney **Contacts:** ***Contact 1:*** - **Phone:** 61298053248 - **Role:** CONTACT ***Contact 2:*** - **Name:** Ron Grunstein - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Australia **Facility:** Woolcock Institute of Medical Research **State:** New South Wales **Status:** RECRUITING **Zip:** 2037 **Location 37:** **City:** Brisbane **Contacts:** ***Contact 1:*** - **Phone:** 61736468111 - **Role:** CONTACT ***Contact 2:*** - **Name:** Donald Soutter Alexander McLeod - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Australia **Facility:** Royal Brisbane and Women's Hospital **State:** Queensland **Status:** RECRUITING **Zip:** 4029 **Location 38:** **City:** Brisbane **Contacts:** ***Contact 1:*** - **Phone:** 61738765688 - **Role:** CONTACT ***Contact 2:*** - **Name:** David Martin Colquhoun - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Australia **Facility:** Core Research Group **State:** Queensland **Status:** RECRUITING **Zip:** 4064 **Location 39:** **City:** Adelaide **Contacts:** ***Contact 1:*** - **Name:** Andrew Hamilton - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Australia **Facility:** Nightingale Research **State:** South Australia **Status:** RECRUITING **Zip:** 5000 **Location 40:** **City:** Bedford Park **Contacts:** ***Contact 1:*** - **Phone:** 61874219780 - **Role:** CONTACT ***Contact 2:*** - **Name:** Danny J. Eckert - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Australia **Facility:** Flinders University **State:** South Australia **Status:** RECRUITING **Zip:** 5042 **Location 41:** **City:** Oaklands Park **Contacts:** ***Contact 1:*** - **Phone:** 610874258690 - **Role:** CONTACT ***Contact 2:*** - **Name:** Stephen Neil Stranks - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Australia **Facility:** Southern Adelaide Diabetes & Endocrine Services **State:** South Australia **Status:** RECRUITING **Zip:** 5046 **Location 42:** **City:** Box Hill **Contacts:** ***Contact 1:*** - **Name:** Christopher Gilfillan - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Australia **Facility:** Box Hill Hospital **State:** Victoria **Status:** RECRUITING **Zip:** 3128 **Location 43:** **City:** Camberwell **Contacts:** ***Contact 1:*** - **Name:** King Cheung - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Australia **Facility:** Emeritus Research **State:** Victoria **Status:** NOT_YET_RECRUITING **Zip:** 3124 **Location 44:** **City:** Heidelberg West **Contacts:** ***Contact 1:*** - **Phone:** 0394962160 - **Role:** CONTACT ***Contact 2:*** - **Name:** Elif Ekinci - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Australia **Facility:** Austin Health - Repatriation Hospital **State:** Victoria **Status:** RECRUITING **Zip:** 3081 **Location 45:** **City:** Joondalup **Contacts:** ***Contact 1:*** - **Phone:** 61894006133 - **Role:** CONTACT ***Contact 2:*** - **Name:** Peter Walter Purnell - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Australia **Facility:** GenesisCare **State:** Western Australia **Status:** RECRUITING **Zip:** 6027 **Location 46:** **City:** Sao Paulo **Contacts:** ***Contact 1:*** - **Name:** Bruno Halpern - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Brazil **Facility:** BR Trials - Ensaios Clinicos e Consultoria **Status:** NOT_YET_RECRUITING **Zip:** 01236030 **Location 47:** **City:** São Paulo **Contacts:** ***Contact 1:*** - **Name:** Gustavo Augusto - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Brazil **Facility:** CPQuali Pesquisa Clínica **Status:** NOT_YET_RECRUITING **Zip:** 01228-000 **Location 48:** **City:** São Paulo **Contacts:** ***Contact 1:*** - **Name:** Breno Balabem Alves - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Brazil **Facility:** CEPIC - Centro Paulista de Investigação Clínica **Status:** NOT_YET_RECRUITING **Zip:** 04266-010 **Location 49:** **City:** Raipur **Contacts:** ***Contact 1:*** - **Name:** Sabah Siddiqui - **Role:** PRINCIPAL_INVESTIGATOR **Country:** India **Facility:** All India Institute of Medical Sciences **State:** Chhattisgarh **Status:** RECRUITING **Zip:** 492099 **Location 50:** **City:** Ahmedabad **Contacts:** ***Contact 1:*** - **Name:** DHAIWAT MRUGESH SHUKLA - **Role:** PRINCIPAL_INVESTIGATOR **Country:** India **Facility:** V.S. General Hospital **State:** Gujarat **Status:** RECRUITING **Zip:** 380006 **Location 51:** **City:** Ahmedabad **Contacts:** ***Contact 1:*** - **Name:** Tiven Marwah - **Role:** PRINCIPAL_INVESTIGATOR **Country:** India **Facility:** Avron Hospitals **State:** Gujarat **Status:** RECRUITING **Zip:** 380013 **Location 52:** **City:** Ahmedabad **Contacts:** ***Contact 1:*** - **Name:** Vitthaldas N Shah - **Role:** PRINCIPAL_INVESTIGATOR **Country:** India **Facility:** Zydus Hospitals & Healthcare Research Pvt.Ltd. **State:** Gujarat **Status:** RECRUITING **Zip:** 380054 **Location 53:** **City:** Bangalore **Contacts:** ***Contact 1:*** - **Name:** Sreenivasa Murthy - **Role:** PRINCIPAL_INVESTIGATOR **Country:** India **Facility:** Life Care Hospital and Research Centre **State:** Karnataka **Status:** RECRUITING **Zip:** 560092 **Location 54:** **City:** Hubli **Contacts:** ***Contact 1:*** - **Name:** Raghavendra Belgaonkar - **Role:** PRINCIPAL_INVESTIGATOR **Country:** India **Facility:** Sushruta Multispeciality Hospital & Research Centre **State:** Karnataka **Status:** RECRUITING **Zip:** 580021 **Location 55:** **City:** Mysore **Contacts:** ***Contact 1:*** - **Name:** Sumaiya Anjum - **Role:** PRINCIPAL_INVESTIGATOR **Country:** India **Facility:** Mysore Medical College **State:** Karnataka **Status:** RECRUITING **Zip:** 570001 **Location 56:** **City:** Indore **Contacts:** ***Contact 1:*** - **Phone:** 919977999687 - **Role:** CONTACT ***Contact 2:*** - **Name:** Sandeep Julka - **Role:** PRINCIPAL_INVESTIGATOR **Country:** India **Facility:** CARE CHL-Hospitals **State:** Madhya Pradesh **Status:** RECRUITING **Zip:** 452008 **Location 57:** **City:** Jaipur **Contacts:** ***Contact 1:*** - **Phone:** 09414048666 - **Role:** CONTACT ***Contact 2:*** - **Name:** SANDEEP KUMAR MATHUR - **Role:** PRINCIPAL_INVESTIGATOR **Country:** India **Facility:** Sawai Man Singh Medical College Hospital (SMS Hospital) **State:** Rajasthan **Status:** RECRUITING **Zip:** 302004 **Location 58:** **City:** Chennai **Contacts:** ***Contact 1:*** - **Name:** VISWANATHAN MOHAN - **Role:** PRINCIPAL_INVESTIGATOR **Country:** India **Facility:** Madras Diabetes Research Foundation **State:** Tamil Nadu **Status:** RECRUITING **Zip:** 600086 **Location 59:** **City:** Vellore **Contacts:** ***Contact 1:*** - **Name:** Nihal Thomas - **Role:** PRINCIPAL_INVESTIGATOR **Country:** India **Facility:** Christian Medical College Vellore **State:** Tamil Nadu **Status:** RECRUITING **Zip:** 632004 **Location 60:** **City:** Hyderabad **Contacts:** ***Contact 1:*** - **Name:** Bipin Kumar Sethi - **Role:** PRINCIPAL_INVESTIGATOR **Country:** India **Facility:** Care Hospitals Hyderabad- Banjara Hills **State:** Telangana **Status:** RECRUITING **Zip:** 500034 **Location 61:** **City:** Hyderabad **Contacts:** ***Contact 1:*** - **Phone:** 917842041355 - **Role:** CONTACT ***Contact 2:*** - **Name:** Vishnupriya Rao Paturi - **Role:** PRINCIPAL_INVESTIGATOR **Country:** India **Facility:** Kumudini Devi Diabetes Research Center **State:** Telangana **Status:** RECRUITING **Zip:** 500072 **Location 62:** **City:** Kolkata **Contacts:** ***Contact 1:*** - **Name:** Sujoy Ghosh - **Role:** PRINCIPAL_INVESTIGATOR **Country:** India **Facility:** Institute of Post Graduate Medical Education and Research and Seth Sukhlal Karnani Memorial Hospital **State:** West Bengal **Status:** RECRUITING **Zip:** 700020 **Location 63:** **City:** Mexicali **Contacts:** ***Contact 1:*** - **Phone:** 6865534121 - **Role:** CONTACT ***Contact 2:*** - **Name:** Francisco Javier Lopez Maldonado - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Mexico **Facility:** Centro de Investigacion en Artritis y Osteoporosis SC **State:** Baja California **Status:** RECRUITING **Zip:** 21200 **Location 64:** **City:** Mexico City **Contacts:** ***Contact 1:*** - **Phone:** 525565501629 - **Role:** CONTACT ***Contact 2:*** - **Name:** Mariana Zolandi Crespo - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Mexico **Facility:** RM Pharma Specialists **State:** Distrito Federal **Status:** RECRUITING **Zip:** 03100 **Location 65:** **City:** Mexico City **Contacts:** ***Contact 1:*** - **Name:** Jose Hector Sanchez Mijangos - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Mexico **Facility:** Clinica Omega **State:** Distrito Federal **Status:** NOT_YET_RECRUITING **Zip:** 06700 **Location 66:** **City:** Mexico City **Contacts:** ***Contact 1:*** - **Phone:** 525585340943 - **Role:** CONTACT ***Contact 2:*** - **Name:** Melchor Alpizar Salazar - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Mexico **Facility:** Centro Especializado En Diabetes, Obesidad Y Prevencion De Enfermedades Cardiovasculares **State:** Distrito Federal **Status:** RECRUITING **Zip:** 11650 **Location 67:** **City:** Guadalajara **Contacts:** ***Contact 1:*** - **Phone:** 5233305493 - **Role:** CONTACT ***Contact 2:*** - **Name:** Cesar Gonzalo Calvo - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Mexico **Facility:** Diseno y Planeacion en Investigacion Medica **State:** Jalisco **Status:** RECRUITING **Zip:** 44130 **Location 68:** **City:** Guadalajara **Contacts:** ***Contact 1:*** - **Phone:** 5233361654104 - **Role:** CONTACT ***Contact 2:*** - **Name:** Maria Del Rosario Arechavaleta Granell - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Mexico **Facility:** Private Practice - Dr. Arechavaleta Granell Maria del Rosario **State:** Jalisco **Status:** RECRUITING **Zip:** 44670 **Location 69:** **City:** Guadalajara **Contacts:** ***Contact 1:*** - **Phone:** 523336422886 - **Role:** CONTACT ***Contact 2:*** - **Name:** Marisol Herrera Marmolejo - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Mexico **Facility:** Unidad de Investigación Clínica y Atención Médica HEPA **State:** Jalisco **Status:** RECRUITING **Zip:** 44670 **Location 70:** **City:** Cuernavaca **Contacts:** ***Contact 1:*** - **Name:** Leobardo Sauque Reyna - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Mexico **Facility:** Instituto de Diabetes, Obesidad y Nutricion **State:** Morelos **Status:** NOT_YET_RECRUITING **Zip:** 62250 **Location 71:** **City:** Monterrey **Contacts:** ***Contact 1:*** - **Phone:** 8140401554 - **Role:** CONTACT ***Contact 2:*** - **Name:** Elías García - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Mexico **Facility:** Cardiolink Clin Trials **State:** Nuevo León **Status:** RECRUITING **Zip:** 64060 **Location 72:** **City:** Monterrey **Contacts:** ***Contact 1:*** - **Name:** Raymundo Garcia Reza - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Mexico **Facility:** Raymundo Garcia Reza **State:** Nuevo León **Status:** NOT_YET_RECRUITING **Zip:** 64460 **Location 73:** **City:** Monterrey **Contacts:** ***Contact 1:*** - **Name:** Jose Gerardo Gonzalez Gonzalez - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Mexico **Facility:** Hospital Universitario "Dr. Jose Eleuterio Gonzalez" **State:** Nuevo León **Status:** NOT_YET_RECRUITING **Zip:** 66460 **Location 74:** **City:** Mérida **Contacts:** ***Contact 1:*** - **Name:** Jesus Simon Campos - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Mexico **Facility:** Kohler and Milstein Research S.A. de C.V. **State:** Yucatán **Status:** NOT_YET_RECRUITING **Zip:** 97070 **Location 75:** **City:** Chihuahua **Contacts:** ***Contact 1:*** - **Phone:** 526144398618 - **Role:** CONTACT ***Contact 2:*** - **Name:** Luis Alejandro Nevarez - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Mexico **Facility:** Investigacion En Salud Y Metabolismo S.C / Nutricion Clinica / Unidad de Base de Datos **Status:** RECRUITING **Zip:** 31110 **Location 76:** **City:** Veracruz **Contacts:** ***Contact 1:*** - **Phone:** 522299327808 - **Role:** CONTACT ***Contact 2:*** - **Name:** Alejandro Quintin Barrat Hernandez - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Mexico **Facility:** FAICIC S. de R.L. de C.V. **Status:** RECRUITING **Zip:** 91900 **Location 77:** **City:** Veracruz **Contacts:** ***Contact 1:*** - **Phone:** 2299314102 - **Role:** CONTACT ***Contact 2:*** - **Name:** Luis Horacio Aguilar Espinoza - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Mexico **Facility:** Arké SMO S.A de C.V **Status:** RECRUITING **Zip:** 91910 **Location 78:** **City:** Oradea **Contacts:** ***Contact 1:*** - **Phone:** 004072248022 - **Role:** CONTACT ***Contact 2:*** - **Name:** Dana Cosma - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Romania **Facility:** Diabdana **State:** Bihor **Status:** RECRUITING **Zip:** 410147 **Location 79:** **City:** Bucharest **Contacts:** ***Contact 1:*** - **Phone:** 40314288633 - **Role:** CONTACT ***Contact 2:*** - **Name:** Georgeta Vacaru - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Romania **Facility:** Geea Medical Easy Diet **State:** București **Status:** RECRUITING **Zip:** 010627 **Location 80:** **City:** Bucharest **Contacts:** ***Contact 1:*** - **Phone:** 0040212323445 - **Role:** CONTACT ***Contact 2:*** - **Name:** Marlena Pascu - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Romania **Facility:** Centrul Medical NutriLife **State:** București **Status:** RECRUITING **Zip:** 013761 **Location 81:** **City:** Mangalia **Contacts:** ***Contact 1:*** - **Phone:** 0040241740581 - **Role:** CONTACT ***Contact 2:*** - **Name:** Amalia Drobeta Thury-Burileanu - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Romania **Facility:** Gama Diamed **State:** Constanța **Status:** RECRUITING **Zip:** 905500 **Location 82:** **City:** Târgu-Mureș **Contacts:** ***Contact 1:*** - **Phone:** 400744572303 - **Role:** CONTACT ***Contact 2:*** - **Name:** Carmen Crisan - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Romania **Facility:** Centrul Medical Mediab **State:** Mureș **Status:** RECRUITING **Zip:** 540142 **Location 83:** **City:** Bacău **Contacts:** ***Contact 1:*** - **Phone:** 40744775762 - **Role:** CONTACT ***Contact 2:*** - **Name:** NOEMI NICOLETA PLETEA - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Romania **Facility:** CMI Dr.Pletea Noemi SRL **Status:** RECRUITING **Zip:** 600154 **Location 84:** **City:** Almeria **Contacts:** ***Contact 1:*** - **Phone:** 0034678510067 - **Role:** CONTACT ***Contact 2:*** - **Name:** Pedro Mezquita-Raya - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Spain **Facility:** Centro Periférico de Especialidades Bola Azul **State:** Almería **Status:** RECRUITING **Zip:** 04009 **Location 85:** **City:** Malaga **Contacts:** ***Contact 1:*** - **Name:** Francisco Tinahones Madueno - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Spain **Facility:** Hospital Universitario Virgen de la Victoria **State:** Andalucía **Status:** RECRUITING **Zip:** 29010 **Location 86:** **City:** Málaga **Contacts:** ***Contact 1:*** - **Name:** GABRIEL OLVEIRA - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Spain **Facility:** H.R.U Málaga - Hospital Civil **State:** Andalucía **Status:** RECRUITING **Zip:** 29009 **Location 87:** **City:** León **Contacts:** ***Contact 1:*** - **Phone:** 34987237400 - **Role:** CONTACT ***Contact 2:*** - **Name:** Maria Dolores Ballesteros Pomar - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Spain **Facility:** Complejo Asistencial Universitario de León - Hospital de León **State:** Castilla Y León **Status:** RECRUITING **Zip:** 24071 **Location 88:** **City:** Madrid **Contacts:** ***Contact 1:*** - **Phone:** 0034913369082 - **Role:** CONTACT ***Contact 2:*** - **Name:** Jose Maria Fernandez Rodriguez - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Spain **Facility:** Hospital Universitario Ramón y Cajal **State:** Madrid, Comunidad De **Status:** RECRUITING **Zip:** 28034 **Location 89:** **City:** Seville **Contacts:** ***Contact 1:*** - **Phone:** 658011529 - **Role:** CONTACT ***Contact 2:*** - **Name:** Cristobal Jesus Morales Portillo - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Spain **Facility:** Vithas Hospital Sevilla **State:** Sevilla **Status:** RECRUITING **Zip:** 41950 **Location 90:** **City:** Sevilla **Contacts:** ***Contact 1:*** - **Phone:** 955012000 - **Role:** CONTACT ***Contact 2:*** - **Name:** Virginia Bellido - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Spain **Facility:** Hospital Universitario Virgen Del Rocio **Status:** RECRUITING **Zip:** 41013 **Location 91:** **City:** Kaohsiung Niao Sung Dist **Contacts:** ***Contact 1:*** - **Phone:** 886975056106 - **Role:** CONTACT ***Contact 2:*** - **Name:** Chih-Yuan Fang - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Taiwan **Facility:** Chang Gung Memorial Hospital at Kaohsiung **State:** Kaohsiung **Status:** NOT_YET_RECRUITING **Zip:** 83301 **Location 92:** **City:** Taichung City **Contacts:** ***Contact 1:*** - **Phone:** 886424739595 - **Role:** CONTACT ***Contact 2:*** - **Name:** Chien-Ning Huang - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Taiwan **Facility:** Chung Shan Medical University Hospital **State:** Taichung **Status:** NOT_YET_RECRUITING **Zip:** 402 **Location 93:** **City:** Taipei City **Contacts:** ***Contact 1:*** - **Phone:** 886228712121 - **Role:** CONTACT ***Contact 2:*** - **Name:** Chern-En Chiang - **Role:** PRINCIPAL_INVESTIGATOR **Country:** Taiwan **Facility:** Taipei Veterans General Hospital **State:** Taipei **Status:** NOT_YET_RECRUITING **Zip:** 11217 #### 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 ### IPD Sharing Statement Module **Access Criteria:** A research proposal must be approved by an independent review panel and researchers must sign a data sharing agreement. **Description:** Anonymized individual participant level data will be provided in a secure access environment upon approval of a research proposal and a signed data sharing agreement. **Info Types:** - STUDY_PROTOCOL - SAP - CSR **IPD Sharing:** YES **Time Frame:** a are available 6 months after the primary publication and approval of the indication studied in the US and European Union (EU), whichever is later. Data will be indefinitely available for requesting. **URL:** http://vivli.org/ ### References Module #### See Also Links **Label:** A Study of Retatrutide (LY3437943) in Participants With Type 2 Diabetes Mellitus Who Have Obesity or Overweight (TRIUMPH-2) **URL:** https://trials.lilly.com/en-US/trial/408215 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000044882 - Term: Glucose Metabolism Disorders - ID: D000008659 - Term: Metabolic Diseases - ID: D000004700 - Term: Endocrine System Diseases - ID: D000044343 - Term: Overnutrition - ID: D000009748 - Term: Nutrition Disorders - ID: D000001835 - Term: Body Weight - ID: D000012891 - Term: Sleep Apnea Syndromes - ID: D000001049 - Term: Apnea - ID: D000012120 - Term: Respiration Disorders - ID: D000012140 - Term: Respiratory Tract Diseases - ID: D000020919 - Term: Sleep Disorders, Intrinsic - ID: D000020920 - Term: Dyssomnias - ID: D000012893 - Term: Sleep Wake Disorders - ID: D000009422 - Term: Nervous System Diseases ### Condition Browse Module - Browse Branches - Abbrev: BC18 - Name: Nutritional and Metabolic Diseases - Abbrev: BC19 - Name: Gland and Hormone Related Diseases - Abbrev: All - Name: All Conditions - Abbrev: BC08 - Name: Respiratory Tract (Lung and Bronchial) Diseases - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: BC10 - Name: Nervous System Diseases - Abbrev: BXM - Name: Behaviors and Mental Disorders ### Condition Browse Module - Browse Leaves - ID: M7115 - Name: Diabetes Mellitus - Relevance: HIGH - As Found: Diabetes Mellitus - ID: M7119 - Name: Diabetes Mellitus, Type 2 - Relevance: HIGH - As Found: Type 2 Diabetes - ID: M4361 - Name: Apnea - Relevance: LOW - As Found: Unknown - ID: M15694 - Name: Sleep Apnea Syndromes - Relevance: LOW - As Found: Unknown - ID: M22010 - Name: Sleep Apnea, Obstructive - Relevance: HIGH - As Found: Obstructive Sleep Apnea - ID: M12701 - Name: Obesity - Relevance: HIGH - As Found: Obesity - ID: M26186 - Name: Overweight - Relevance: HIGH - As Found: Overweight - 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: M25307 - Name: Overnutrition - Relevance: LOW - As Found: Unknown - ID: M12684 - Name: Nutrition Disorders - Relevance: LOW - As Found: Unknown - ID: M5114 - Name: Body Weight - Relevance: LOW - As Found: Unknown - ID: M16355 - Name: Syndrome - Relevance: LOW - As Found: Unknown - ID: M14957 - Name: Respiration Disorders - Relevance: LOW - As Found: Unknown - ID: M14977 - Name: Respiratory Tract Diseases - Relevance: LOW - As Found: Unknown - ID: M22242 - Name: Parasomnias - Relevance: LOW - As Found: Unknown - ID: M22654 - Name: Sleep Disorders, Intrinsic - Relevance: LOW - As Found: Unknown - ID: M22655 - Name: Dyssomnias - Relevance: LOW - As Found: Unknown - ID: M15696 - Name: Sleep Wake Disorders - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000020181 - Term: Sleep Apnea, Obstructive - ID: D000003920 - Term: Diabetes Mellitus - ID: D000009765 - Term: Obesity - ID: D000003924 - Term: Diabetes Mellitus, Type 2 - ID: D000050177 - Term: Overweight ### Misc Info Module #### Removed Countries - Country: China - Country: Korea, Republic of - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02286479 **Brief Title:** Comparison of Loop and Primary Incision&Drainage Techniques in the Emergency Department **Official Title:** Comparison of Loop Drainage and Primary Incision and Drainage Techniques in Patients With Cutaneous Abscess in the Emergency Department #### Organization Study ID Info **ID:** KOU KAEK 2014/259 #### Organization **Class:** OTHER **Full Name:** Kocaeli University ### Status Module #### Completion Date **Date:** 2016-08 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2016-08-15 **Type:** ESTIMATED **Last Update Submit Date:** 2016-08-12 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2016-08 **Type:** ACTUAL #### Start Date **Date:** 2014-10 **Status Verified Date:** 2016-08 #### Study First Post Date **Date:** 2014-11-07 **Type:** ESTIMATED **Study First Submit Date:** 2014-11-05 **Study First Submit QC Date:** 2014-11-06 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Kocaeli University #### Responsible Party **Investigator Affiliation:** Kocaeli University **Investigator Full Name:** Ibrahim Ulas Ozturan **Investigator Title:** MD **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** Skin abscesses are among the most common soft tissue infections cause emergency room visits frequently. Management of abscess drainage and prevent further complications are important entities for emergency physicians. Historically primary incision and drainage (I\&D) technique has found very effective method of abscess drainage, however a novel technique loop drainage holds promising. The purpose of our study is comparison efficacy of I\&D and loop drainage techniques in patients with cutaneous abscess. ### Conditions Module **Conditions:** - Skin Abscess ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 50 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** In the incision and drainage group, the abscesses are incised, irrigated by sterile solutions and drained conventionally. **Intervention Names:** - Procedure: Incision and Drainage **Label:** Incision and Drainage **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** In the loop drainage group, two small incision are made on each side of abscess. The pus are drained and septations are seperated by a forceps. Abscess cavitary irrigated by sterile solution. Sterile, non-powder, non-latex surgical gloves cuff is inserted in one incision and taken out from the other insicion. Then two tips of cuff are tied loosely. **Intervention Names:** - Procedure: Loop drainage **Label:** Loop drainage **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Incision and Drainage **Description:** In the incision and drainage group, the abscesses are incised, irrigated by sterile solutions and drained conventionally. **Name:** Incision and Drainage **Type:** PROCEDURE #### Intervention 2 **Arm Group Labels:** - Loop drainage **Description:** In the loop drainage group, two small incision are made on each side of abscess. The pus are drained and septations are seperated by a forceps. Abscess cavitary irrigated by sterile solution. Sterile, non-powder, non-latex surgical gloves cuff is inserted in one incision and taken out from the other insicion. Then two tips of cuff are tied loosely. **Name:** Loop drainage **Type:** PROCEDURE ### Outcomes Module #### Other Outcomes **Measure:** Procedure Pain Intensity **Time Frame:** intraoperative #### Primary Outcomes **Measure:** Abscess Resolution **Time Frame:** 7 days #### Secondary Outcomes **Measure:** Procedure Time **Time Frame:** intraoperative ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Adult patients presenting to Kocaeli University Emergency Department with cutaneous abscess. * Providing written informed consent. Exclusion Criteria: * Under 18 years of age. * Immunosuppressive patients. * Using medications have effects on wound healing. * Abscess is not recognizable by bedside ultrasound. * Lidocaine allergy **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Kocaeli **Country:** Turkey **Facility:** Kocaeli University Medical Faculty Emergency Medicine Department **Zip:** 41730 ### IPD Sharing Statement Module **IPD Sharing:** YES ### References Module #### References **Citation:** Ozturan IU, Dogan NO, Karakayali O, Ozbek AE, Yilmaz S, Pekdemir M, Suner S. Comparison of loop and primary incision & drainage techniques in adult patients with cutaneous abscess: A preliminary, randomized clinical trial. Am J Emerg Med. 2017 Jun;35(6):830-834. doi: 10.1016/j.ajem.2017.01.036. Epub 2017 Jan 22. **PMID:** 28162873 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000020969 - Term: Disease Attributes - ID: D000010335 - Term: Pathologic Processes - ID: D000013492 - Term: Suppuration - ID: D000007239 - Term: Infections - ID: D000007249 - Term: Inflammation ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC26 - Name: Wounds and Injuries - Abbrev: BC01 - Name: Infections ### Condition Browse Module - Browse Leaves - ID: M7796 - Name: Emergencies - Relevance: HIGH - As Found: Emergency - ID: M1112 - Name: Surgical Wound - Relevance: LOW - As Found: Unknown - ID: M37 - Name: Abscess - Relevance: HIGH - As Found: Abscess - ID: M22700 - Name: Disease Attributes - Relevance: LOW - As Found: Unknown - ID: M16273 - Name: Suppuration - 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: M10293 - Name: Inflammation - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000000038 - Term: Abscess - ID: D000004630 - Term: Emergencies ### 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:** NCT06264479 **Acronym:** PEAR-TREE2 **Brief Title:** Prospective Evaluation of AI R&D Tool for Patient Stratification: a Trial for Renal Immuno-oncology Model Experimental Evaluation 2 **Official Title:** Prospective Evaluation of AI R&D Tool for Patient Stratification: a Trial for Renal Immuno-oncology Model Experimental Evaluation 2 #### Organization Study ID Info **ID:** PEAR-TREE2 #### Organization **Class:** INDUSTRY **Full Name:** Ourotech, Inc. ### Status Module #### Completion Date **Date:** 2029-03-31 **Type:** ESTIMATED #### Expanded Access Info #### Last Update Post Date **Date:** 2024-04-02 **Type:** ACTUAL **Last Update Submit Date:** 2024-04-01 **Overall Status:** NOT_YET_RECRUITING #### Primary Completion Date **Date:** 2028-09-30 **Type:** ESTIMATED #### Start Date **Date:** 2024-05-01 **Type:** ESTIMATED **Status Verified Date:** 2024-04 #### Study First Post Date **Date:** 2024-02-20 **Type:** ACTUAL **Study First Submit Date:** 2024-02-09 **Study First Submit QC Date:** 2024-02-09 ### Sponsor Collaborators Module #### Collaborators **Class:** OTHER **Name:** Royal Free Hospital NHS Foundation Trust **Class:** UNKNOWN **Name:** Cambridge University Hospitals NHS Trust **Class:** UNKNOWN **Name:** East & North Herts NHS Trust **Class:** OTHER **Name:** Imperial College Healthcare NHS Trust #### Lead Sponsor **Class:** INDUSTRY **Name:** Ourotech, Inc. #### Responsible Party **Type:** SPONSOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** True ### Description Module **Brief Summary:** Pear Bio has developed a predictive biomarker technology that combines 3D cell culture, microscopy and computer vision to measure the response of an individual patient's tumor sample to different systemic therapy regimens that are tested simultaneously ex vivo. This study will recruit patients with advanced or metastatic kidney cancer who are due to start a clinically-indicated new line of therapy. The oncologist will be blinded to the response on the Pear Bio test (the test will be run in parallel with the patient's treatment). The primary objective of this study is to establish the sensitivity and specificity of Pear Bio's test results against patient outcomes (objective response, progression-free survival, depth and duration of response, overall survival). **Detailed Description:** This is a multicenter, international, observational pilot study that aims to determine the accuracy of a new assay, the Pear Bio test, in predicting response rate (ORR) in patients receiving standard of care systemic therapies for kidney cancer. Patients will undergo an additional, mandatory core needle biopsy of a lesion before commencing their next line of therapy. 40mL of blood will also be collected from each patient. The biopsy sample will be run on the Pear Bio test while the patient receives their standard of care treatment. As such, for this study, the result from the Pear Bio test will not be used to inform the choice of treatment and the treating oncologist will be blinded to the test results. The investigators will measure objective response rate (ORR) and other outcome metrics (PFS, DoR, OS, etc.). The Pear Bio test results will be compared to the actual patient outcomes to determine the test's sensitivity, specificity, positive predictive value and negative predictive value. ### Conditions Module **Conditions:** - Kidney Cancer - Renal Cell Cancer Metastatic **Keywords:** - computer vision - cell culture - microtumor - functional testing - predictive biomarker - immuno-oncology ### Design Module #### Bio Spec **Description:** Tumor cores and blood **Retention:** SAMPLES_WITH_DNA #### Design Info **Observational Model:** COHORT **Time Perspective:** PROSPECTIVE #### Enrollment Info **Count:** 200 **Type:** ESTIMATED **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Patients with advanced or metastatic kidney cancer, due to start a new line of systemic therapy (targeted drug, immunotherapy, etc.) **Intervention Names:** - Procedure: Biopsy **Label:** Trial Cohort ### Interventions #### Intervention 1 **Arm Group Labels:** - Trial Cohort **Description:** Patients undergo a biopsy from a lesion, and give 40ml of blood **Name:** Biopsy **Type:** PROCEDURE ### Outcomes Module #### Other Outcomes **Description:** The percentage of patient samples successfully arriving at central lab with \>100k live cells isolated and maintaining 70% cell viability after 4 days in culture with no treatment **Measure:** Culture success rate **Time Frame:** 4 days **Description:** The hazard ratio for PFS and OS between Pear-assay biomarker-high and biomarker-low groups **Measure:** Hazard ratio by biomarker group **Time Frame:** 2 years **Description:** The performance of Pear image-based biomarkers are established against patients overall survival. **Measure:** OS prediction accuracy **Time Frame:** 2 years #### Primary Outcomes **Description:** The sensitivity, specificity PPV and NPV of Pear image-based biomarkers are established against patient ORR (evaluated on imaging). **Measure:** Objective Response Rate correlation accuracy (sensitivity & specificity) **Time Frame:** 6 months #### Secondary Outcomes **Description:** The sensitivity, specificity PPV and NPV of Pear image-based biomarkers are established against patients achieving a complete response (evaluated by imaging). **Measure:** Complete Response rate correlation accuracy (sensitivity & specificity) **Time Frame:** 2 years **Description:** The sensitivity, specificity PPV and NPV of Pear image-based biomarkers are established against patients achieving a deep response (evaluated by imaging). **Measure:** Deep Response rate correlation accuracy (sensitivity & specificity) **Time Frame:** 2 years **Description:** The sensitivity, specificity PPV and NPV of Pear image-based biomarkers are established against patients achieving a durable response at 6, 12 and 24 months (evaluated by imaging). **Measure:** Durable Response rate correlation accuracy (sensitivity & specificity) **Time Frame:** 2 years **Description:** The performance of Pear image-based biomarkers are established against patients progression-free survival at 6, 12 and 24 months (evaluated by imaging). **Measure:** PFS prediction accuracy **Time Frame:** 2 years ### Eligibility Module **Eligibility Criteria:** Inclusion: 1. Able to give written informed consent prior to admission to this study. 2. Female or male aged ≥18 years. 3. Evidence of advanced RCC with intention to receive systemic therapy, defined as: * Clinical suspicion of advanced RCC with intention to undergo a clinically-mandated biopsy and subsequent systemic therapy OR * Histological evidence of advanced RCC with intention to undergo subsequent systemic therapy and willing to undergo additional research biopsy 4. At least one lesion evaluable under RECIST 1.1 criteria 5. Willing to donate at least two additional core biopsy samples prior to starting subsequent systemic therapy. 6. Willing to undergo venous sampling for 40mL of blood Exclusion: 1. Early stage kidney cancer 2. Patients who do not have kidney cancer 3. Patients with RCC that do not intend to receive systemic therapy 4. Patients who have already commenced systemic therapy with no plans of changing the systemic therapy after the collection of the core needle biopsy. 5. Any other disease, metabolic dysfunction, physical examination finding, or clinical laboratory finding that, in the investigator's opinion, gives reasonable suspicion of a disease or condition that may affect the interpretation of the results, render the patient at high risk from treatment complications or interferes with obtaining informed consent. 6. Previous diagnosis of other cancer. Previously treated cancer may be acceptable in some circumstances (e.g. surgery for an unrelated cancer \> 5 years ago) after discussion with the Sponsor. 7. No lesions are amenable to biopsy **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Patients with advanced or metastatic kidney cancer, due to start a new line of therapy ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Duleek Ranatunga **Phone:** +44 7716558079 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Elli Tham **Role:** CONTACT #### Locations **Location 1:** **City:** Cambridge **Contacts:** ***Contact 1:*** - **Name:** Kate Fife - **Role:** CONTACT **Country:** United Kingdom **Facility:** Addenbrooke's Hospital **State:** Cambridgeshire **Zip:** CB2 0QQ **Location 2:** **City:** London **Contacts:** ***Contact 1:*** - **Name:** Anand Sharma - **Role:** CONTACT **Country:** United Kingdom **Facility:** Mount Vernon Cancer Centre **Zip:** HA6 2RN **Location 3:** **City:** London **Country:** United Kingdom **Facility:** Imperial College Healthcare NHS Trust **Zip:** W6 8RF **Location 4:** **City:** London **Contacts:** ***Contact 1:*** - **Name:** Ekaterini Boleti, MD PhD MRCP - **Phone:** 020 7830 2396 - **Phone Ext:** 34476 - **Role:** CONTACT **Country:** United Kingdom **Facility:** Royal Free NHS Foundation Trust #### Overall Officials **Official 1:** **Affiliation:** Royal Free Hospital NHS Foundation Trust **Name:** Ekaterini Boleti **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **Description:** Pseudonymised data will be shared between study sites, such as treatment and outcome data for each patient. No publication will be made revealing individual patient data; only group-level data will be published. **IPD Sharing:** NO ### References Module #### Available IPDs **Type:** Study Protocol **URL:** https://www.pearbio.com/peartree2 ## Document Section ### Large Document Module #### Large Docs - Date: 2023-07-28 - Filename: Prot_SAP_000.pdf - Has ICF: False - Has Protocol: True - Has SAP: True - Label: Study Protocol and Statistical Analysis Plan - Size: 424039 - Type Abbrev: Prot_SAP - Upload Date: 2023-07-28T07:18 - Date: 2023-07-28 - Filename: ICF_001.pdf - Has ICF: True - Has Protocol: False - Has SAP: False - Label: Informed Consent Form - Size: 197013 - Type Abbrev: ICF - Upload Date: 2023-07-28T07:19 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000009369 - Term: Neoplasms - ID: D000000230 - Term: Adenocarcinoma - ID: D000002277 - Term: Carcinoma - ID: D000009375 - Term: Neoplasms, Glandular and Epithelial - ID: D000009370 - Term: Neoplasms by Histologic Type - 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: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: All - Name: All Conditions - Abbrev: Rare - Name: Rare Diseases ### Condition Browse Module - Browse Leaves - ID: M5548 - Name: Carcinoma, Renal Cell - Relevance: HIGH - As Found: Renal Cell Cancer - ID: M10703 - Name: Kidney Neoplasms - Relevance: HIGH - As Found: Kidney Cancer - ID: M5534 - Name: Carcinoma - Relevance: LOW - As Found: Unknown - ID: M3585 - Name: Adenocarcinoma - 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: 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: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000002292 - Term: Carcinoma, Renal Cell - ID: D000007680 - Term: Kidney Neoplasms ### Intervention Browse Module - Browse Branches - Abbrev: All - Name: All Drugs and Chemicals ### Intervention Browse Module - Browse Leaves - ID: M2853 - Name: Immunomodulating Agents - Relevance: LOW - As Found: Unknown ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02075879 **Brief Title:** The Effects of a Nurse-led Case Management Programme on Home Exercise Training for Haemodialysis Patients **Official Title:** The Effects of a Nurse-led Case Management Programme on Home Exercise Training for Haemodialysis Patients #### Organization Study ID Info **ID:** HSEARS20121105002 #### Organization **Class:** OTHER **Full Name:** The Hong Kong Polytechnic University ### Status Module #### Completion Date **Date:** 2013-12 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2014-03-03 **Type:** ESTIMATED **Last Update Submit Date:** 2014-02-27 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2013-12 **Type:** ACTUAL #### Start Date **Date:** 2013-02 **Status Verified Date:** 2014-02 #### Study First Post Date **Date:** 2014-03-03 **Type:** ESTIMATED **Study First Submit Date:** 2014-02-27 **Study First Submit QC Date:** 2014-02-27 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** The Hong Kong Polytechnic University #### Responsible Party **Investigator Affiliation:** The Hong Kong Polytechnic University **Investigator Full Name:** Susan Ka Yee Chow **Investigator Title:** Assistant Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Oversight Has DMC:** True ### Description Module **Brief Summary:** The purpose of this study is to examine the effects of a nurse-led case management programme on home exercise training for haemodialysis patients. We hypothesised that: a. There is no significant difference in physical functioning between participants receiving the nurse-led home exercise training programme and those receiving the comparison care. b. There is no difference in depression between participants receiving the nurse-led home exercise training programme and those receiving the comparison care. c. There is no difference in quality of life and health perception between participants receiving the nurse-led home exercise training programme and those receiving the comparison care. d. There is no difference in physical activity levels between participants receiving the nurse-led home exercise training programme and those receiving the comparison care. e. There is no difference in perceived benefits and barriers of exercise between participants receiving the nurse-led home exercise training programme and those receiving the comparison care. The study was a randomized controlled trial conducted in two haemodialysis units of two tertiary hospitals in Nanjing, China. Participants were randomly assigned to either study group or comparison group. Participants in both groups received the in-center exercise training (20 minutes) before haemodialysis sessions weekly for 6 weeks and were instructed to perform exercise at home. The in-center training was focused on flexibility and strengthening exercise. Patients were encouraged to have cardiovascular exercises at home which will improve their cardiovascular conditions and endurance. The list of cardiovascular exercise included brisk walking, bicycling, jogging. Participants in the study group were instructed to start walking or brisk walking at low duration and gradually progress to a maximum of 30 minutes daily per week. To facilitate exercise progression, the nurse case managers discussed exercise benefits, explored exercise barriers and developed mutual goals with patients. The nurse motivated them and checked the exercise behaviors to ensure adherence to the recommended exercise regime. The nurse case managers interviewed the study group patients weekly for six weeks and biweekly for another six weeks. Participants in the comparison group only participated in the in-center exercise training. The comparison group patients received usual care from the nurse without the interviews and mutual goals developed. **Detailed Description:** The duration of interventions was 12 weeks. In-center exercise training sessions were available for all participants once a week for 6 weeks. Each session lasted about 20 minutes. Participants were instructed to perform flexibility and strength exercise in centres prior to the haemodialysis session. The researcher performed exercises with the participants and emphasised the importance of self-monitoring during the sessions. Symptoms (such as chest pain, dyspnea, dizziness, and leg cramps) and vital signs (blood pressure, heart rate, and pulse) were checked before exercise and monitored during the entire exercise session. Participants were instructed to self-monitor exercise intensity. The exercise programme was pause immediately if the patient was feeling short of breath, chest pain or pressure, irregular heartbeats, leg cramps, dizziness or lightheadedness, blurring of vision, or any discomfort. Intervention group During the study period, participants in the intervention group received additional face-to-face interviews conducted by the designated case managers every week for six weeks and biweekly for another six weeks. During interviews, patients were instructed to start aerobic exercise, such as walking, jogging, cycling, or brisk walking at home at low duration and gradually progress to a maximum of 30 minutes daily per week. Apart from aerobic exercise, patients were recommended to perform strength exercises twice a week and flexibility exercises for every day. The key elements of the interview included: 1) assessment of patients' knowledge and exercise behaviour; examining patients' attitudes and feelings about being physical activity; 2) explore patients' barriers to exercise and perceived benefits; 3) patient education on the benefits of exercise if needed; 4) development of mutual goals in an exercise plan; 5) explain the purpose of a recording exercise log and instruction in correct way to keep an exercise diary; 6) discuss the available supports and resources that could motivate patients to initiate and maintain exercise, e.g., centre-based exercise programme, support from family members, health care providers, peer patients, and so forth. In the follow-up interviews, case managers reviewed the exercise log with participants, discussed unpleasant experiences related to exercise, exercise progress, and provided encouragement. According to the progress participants made, the exercise goals and plan were renewed or revised. Referrals were made as needed. Comparison group Participants in the comparison group were advised to participate in the in-centre exercise training programme which was available before haemodialysis sessions once a week for different shifts of patients. The training session lasted about 20 minutes and only flexibility and strength training were provided. During the training sessions, patients were advised to increase their daily activity levels. ### Conditions Module **Conditions:** - Kidney Disease ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** SINGLE **Who Masked:** - OUTCOMES_ASSESSOR **Primary Purpose:** HEALTH_SERVICES_RESEARCH #### Enrollment Info **Count:** 113 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Participants in experimental arm received the in-center exercise training (20 minutes) before hemodialysis sessions weekly for 6 weeks and were instructed to perform exercise at home. They were additionally instructed to start walking or brisk walking at low duration and gradually progress to a maximum of 30 minutes daily per week. To facilitate exercise progression, the nurse case managers discussed exercise benefits, explored exercise barriers and developed mutual goals with patients. The nurse motivated them and checked the exercise behaviors to ensure adherence to the recommended exercise regime. The nurse case managers interviewed the patients weekly for six weeks and biweekly for another six weeks. **Intervention Names:** - Behavioral: Nurse-led interviewing **Label:** Nurse-led interviewing **Type:** EXPERIMENTAL #### Arm Group 2 **Description:** Participants received the in-center exercise training (20 minutes) before hemodialysis sessions weekly for 6 weeks and were instructed to perform exercise at home. The in-center training was conducted by the researcher with a group of four-to six participants focusing on flexibility and strengthening exercise only. **Intervention Names:** - Other: Brief group exercise **Label:** Brief group exercise **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - Nurse-led interviewing **Description:** Participants in experimental arm received regular face-to-face interviews during dialysis sessions conducted by the designated case managers every week for 6 weeks and biweekly for another 6 weeks. **Name:** Nurse-led interviewing **Type:** BEHAVIORAL #### Intervention 2 **Arm Group Labels:** - Brief group exercise **Description:** Participants in this arm only participated in the brief in-center exercise training. They received usual care from the nurse without the interviews and mutual goals developed. **Name:** Brief group exercise **Type:** OTHER ### Outcomes Module #### Primary Outcomes **Measure:** Change from baseline in walking speed on the 10-meter walking test at week 6 and change from baseline in walking speed on 10-meter walking test at week 12 **Time Frame:** Baseline, Week 6, and Week 12 #### Secondary Outcomes **Measure:** Change from baseline in time in completing the 10-repetition sit-to-stand test (10-STS) at week 6, and change from baseline in time in completing 10-STS at week 12 **Time Frame:** Baseline, Week 6, and Week 12 **Measure:** Change from baseline in perceived exercise benefits and barriers on the Dialysis Patient-perceived Exercise Benefits and Barriers Scale (DPEBBS) at week 6 and change from baseline in the DPEBBS score at week 12 **Time Frame:** Baseline, Week 6, and Week 12 **Measure:** Change from baseline in health-related quality of life on the Kidney Disease Quality of Life Questionnaire (KDQOL-36) at week 6, and change from baseline in KDQOL-36 score at week 12 **Time Frame:** Baseline, Week 6, and Week 12 **Measure:** Change from baseline in depressive symptoms on the Beck Depression Inventory (BDI) at week 6, and change from baseline in BDI score at week 12 **Time Frame:** Baseline, Week 6, and Week 12 **Measure:** Change from baseline in self-perceived health in the first item in the Kidney Disease Quality of Life Questionnaire at week 6, and change from baseline in self-perceived health score at week 12 **Time Frame:** Baseline, Week 6, and Week 12 **Measure:** Change from week 6 in physical activity levels measured by exercise log at week 12 **Time Frame:** Week 6 and Week 12 ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Dialysis for \>3 months; * Age \> 18 years; * Kt/V (dialysis adequacy) \>1.2; * Hemoglobin level \> 8g/dl; * Ambulatory without assistance ; * Able to communicate in Chinese; * Able to and willing to provide consent. Exclusion Criteria: * Unstable physical conditions or severe musculoskeletal diseases that might hinder exercise training; * Severe hearing impairment; * Diagnosed with mental diseases; * Meeting or exceeding the exercise recommendation. **Maximum Age:** 75 Years **Minimum Age:** 19 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Nanjing **Country:** China **Facility:** The First Affiliated Hospital with Nanjing Medical University **State:** Jiangsu **Zip:** 210029 **Location 2:** **City:** Nanjing **Country:** China **Facility:** The Second Affiliated Hospital with Nanjing Medical University **State:** Jiangsu **Zip:** 210029 #### Overall Officials **Official 1:** **Affiliation:** School of Nursing, The Hong Kong Polytechnic University **Name:** Susan KY Chow, PhD **Role:** PRINCIPAL_INVESTIGATOR ### References Module #### References **Citation:** Bernier-Jean A, Beruni NA, Bondonno NP, Williams G, Teixeira-Pinto A, Craig JC, Wong G. Exercise training for adults undergoing maintenance dialysis. Cochrane Database Syst Rev. 2022 Jan 12;1(1):CD014653. doi: 10.1002/14651858.CD014653. **PMID:** 35018639 **Citation:** Tao X, Chow SK, Wong FK. A nurse-led case management program on home exercise training for hemodialysis patients: A randomized controlled trial. Int J Nurs Stud. 2015 Jun;52(6):1029-41. doi: 10.1016/j.ijnurstu.2015.03.013. Epub 2015 Mar 26. **PMID:** 25840898 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000014570 - Term: Urologic Diseases - ID: D000052776 - Term: Female Urogenital Diseases - ID: D000005261 - Term: Female Urogenital Diseases and Pregnancy Complications - ID: D000091642 - Term: Urogenital Diseases - ID: D000052801 - Term: Male Urogenital Diseases ### Condition Browse Module - Browse Branches - Abbrev: BXS - Name: Urinary Tract, Sexual Organs, and Pregnancy Conditions - Abbrev: All - Name: All Conditions ### Condition Browse Module - Browse Leaves - ID: M10698 - Name: Kidney Diseases - Relevance: HIGH - As Found: Kidney Disease - ID: M17319 - Name: Urologic Diseases - Relevance: LOW - As Found: Unknown - ID: M2875 - Name: Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M27093 - Name: Female Urogenital Diseases - Relevance: LOW - As Found: Unknown - ID: M14127 - Name: Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M8399 - Name: Female Urogenital Diseases and Pregnancy Complications - Relevance: LOW - As Found: Unknown - ID: M27095 - Name: Male Urogenital Diseases - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000007674 - Term: Kidney Diseases ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03843879 **Brief Title:** TAP Blocks vs. IV Lidocaine for Kidney Transplants **Official Title:** A Comparison of Transversus Abdominis Plane Blocks Versus Continuous Intravenous Lidocaine for Kidney Transplant Surgery #### Organization Study ID Info **ID:** IRB18-109 #### Organization **Class:** OTHER **Full Name:** Benaroya Research Institute ### Status Module #### Completion Date **Date:** 2021-09-01 **Type:** ESTIMATED #### Expanded Access Info **Last Known Status:** RECRUITING #### Last Update Post Date **Date:** 2019-07-17 **Type:** ACTUAL **Last Update Submit Date:** 2019-07-15 **Overall Status:** UNKNOWN #### Primary Completion Date **Date:** 2021-03-01 **Type:** ESTIMATED #### Start Date **Date:** 2019-04-01 **Type:** ACTUAL **Status Verified Date:** 2019-07 #### Study First Post Date **Date:** 2019-02-18 **Type:** ACTUAL **Study First Submit Date:** 2019-02-14 **Study First Submit QC Date:** 2019-02-15 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** Benaroya Research Institute #### Responsible Party **Investigator Affiliation:** Benaroya Research Institute **Investigator Full Name:** Neil Hanson **Investigator Title:** Anesthesiologist **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** True **Is US Export:** True **Oversight Has DMC:** False ### Description Module **Brief Summary:** This study will compare continuous intravenous lidocaine against single-injection transversus abdominis plane (TAP) block as a modality for postoperative analgesia in kidney transplant surgery. **Detailed Description:** This study is a non-inferiority trial designed to assess the analgesic efficacy of an intravenous (IV) lidocaine infusion against single-injection transversus abdominis plane (TAP) block in patients undergoing kidney transplant surgery. The investigators propose a study of 124 subjects randomized into two groups. The control group will receive a TAP block. The study group will receive a continuous IV lidocaine infusion. The investigators hypothesize that there will be no statistically significant difference in postoperative opioid consumption between the two groups in the first 24 hours. ### Conditions Module **Conditions:** - Postoperative Pain ### Design Module #### Design Info **Allocation:** RANDOMIZED **Intervention Model:** PARALLEL ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 124 **Type:** ESTIMATED **Phases:** - PHASE2 - PHASE3 **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Single-injection transversus abdominis plane block **Intervention Names:** - Procedure: Transversus Abdominis Plane Block **Label:** TAP Block **Type:** ACTIVE_COMPARATOR #### Arm Group 2 **Description:** Continuous intravenous lidocaine infusion **Intervention Names:** - Drug: Intravenous Lidocaine **Label:** IV Lidocaine **Type:** ACTIVE_COMPARATOR ### Interventions #### Intervention 1 **Arm Group Labels:** - TAP Block **Description:** Single-injection transversus abdominis plane block with 30 mL of 0.25% Bupivacaine with 1:400,000 epinephrine **Name:** Transversus Abdominis Plane Block **Other Names:** - TAP Block **Type:** PROCEDURE #### Intervention 2 **Arm Group Labels:** - IV Lidocaine **Description:** Continuous intravenous lidocaine infusion **Name:** Intravenous Lidocaine **Other Names:** - Continuous intravenous lidocaine **Type:** DRUG ### Outcomes Module #### Primary Outcomes **Description:** Total opioid utilization **Measure:** Opioid Consumption **Time Frame:** 0-24 Hours #### Secondary Outcomes **Description:** Numerical Rating Scale Pain Scores (Range: 0-10, where 0 is no pain and 10 is the worst pain) **Measure:** Pain Scores **Time Frame:** 0-48 Hours **Description:** Total opioid utilization **Measure:** Opioid Consumption **Time Frame:** 24-48 Hours **Description:** Nausea, Vomiting, Pruritis, Respiratory Depression, Constipation **Measure:** Opioid-Related Adverse Events **Time Frame:** 0-48 Hours **Description:** Local Anesthetic Systemic Toxicity **Measure:** Block/Infusion-Related Adverse Events **Time Frame:** 0-48 Hours **Description:** Use of prescribed opioids **Measure:** Opioid Usage **Time Frame:** 30 days from discharge ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: * Kidney transplant recipient * \>18 years old * Consent to participate Exclusion Criteria: * \<18 years old * Refusal to participate * Chronic opioid use * Seizure disorder * Allergy to local anesthestics * Severe hepatic disease **Healthy Volunteers:** True **Minimum Age:** 18 Years **Sex:** ALL **Standard Ages:** - ADULT - OLDER_ADULT ### Contacts Locations Module #### Central Contacts **Contact 1:** **Email:** [email protected] **Name:** Neil A Hanson, MD **Phone:** 206-223-6980 **Role:** CONTACT **Contact 2:** **Email:** [email protected] **Name:** Wyndam M Strodtbeck, MD **Phone:** 206-223-6980 **Role:** CONTACT #### Locations **Location 1:** **City:** Seattle **Contacts:** ***Contact 1:*** - **Email:** [email protected] - **Name:** Neil A Hanson, MD - **Role:** CONTACT ***Contact 2:*** - **Email:** [email protected] - **Name:** Wyndam M Strodtbeck, MD - **Role:** CONTACT **Country:** United States **Facility:** Virginia Mason Medical Center **State:** Washington **Status:** RECRUITING **Zip:** 98101 #### Overall Officials **Official 1:** **Affiliation:** Virginia Mason Medical Center **Name:** Neil A Hanson, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ### References Module #### References **Citation:** Hanson NA, Strunk J, Saunders G, Cowan NG, Brandenberger J, Kuhr CS, Oryhan C, Warren DT, Slee AE, Strodtbeck W. Comparison of continuous intravenous lidocaine versus transversus abdominis plane block for kidney transplant surgery: a randomized, non-inferiority trial. Reg Anesth Pain Med. 2021 Nov;46(11):955-959. doi: 10.1136/rapm-2021-102973. Epub 2021 Aug 20. **PMID:** 34417343 ## Derived Section ### Condition Browse Module - Ancestors - ID: D000011183 - Term: Postoperative Complications - ID: D000010335 - Term: Pathologic Processes - ID: D000010146 - Term: Pain - ID: D000009461 - Term: Neurologic Manifestations ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC10 - Name: Nervous System Diseases ### Condition Browse Module - Browse Leaves - ID: M13069 - Name: Pain, Postoperative - Relevance: HIGH - As Found: Postoperative Pain - ID: M13066 - Name: Pain - Relevance: LOW - As Found: Unknown - ID: M14065 - Name: Postoperative Complications - Relevance: LOW - As Found: Unknown - ID: M12404 - Name: Neurologic Manifestations - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000010149 - Term: Pain, Postoperative ### 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 - ID: D000000889 - Term: Anti-Arrhythmia Agents - ID: D000061567 - Term: Voltage-Gated Sodium Channel Blockers - ID: D000026941 - Term: Sodium Channel Blockers - ID: D000049990 - Term: Membrane Transport Modulators - ID: D000045504 - Term: Molecular Mechanisms of Pharmacological Action ### Intervention Browse Module - Browse Branches - Abbrev: AnArAg - Name: Anti-Arrhythmia Agents - Abbrev: ChanBlk - Name: Channel Blockers - Abbrev: CNSDep - Name: Central Nervous System Depressants - Abbrev: All - Name: All Drugs and Chemicals - Abbrev: VaCoAg - Name: Vasoconstrictor Agents - Abbrev: Resp - Name: Respiratory System Agents ### Intervention Browse Module - Browse Leaves - ID: M11014 - Name: Lidocaine - Relevance: HIGH - As Found: Solution - ID: M5315 - Name: Bupivacaine - Relevance: LOW - As Found: Unknown - ID: M7992 - Name: Epinephrine - Relevance: LOW - As Found: Unknown - ID: M30371 - Name: Racepinephrine - Relevance: LOW - As Found: Unknown - ID: M211043 - Name: Epinephryl borate - Relevance: LOW - As Found: Unknown - ID: M4107 - Name: Anesthetics - Relevance: LOW - As Found: Unknown - ID: M4109 - Name: Anesthetics, Local - Relevance: LOW - As Found: Unknown - ID: M4213 - Name: Anti-Arrhythmia Agents - Relevance: LOW - As Found: Unknown - ID: M23177 - Name: Sodium Channel Blockers - Relevance: LOW - As Found: Unknown - ID: M30025 - Name: Diuretics, Potassium Sparing - Relevance: LOW - As Found: Unknown ### Intervention Browse Module - Meshes - ID: D000008012 - Term: Lidocaine ### Misc Info Module - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT02279979 **Brief Title:** Thoratec Corporation HeartMate PHP™ Cardiogenic Shock Trial **Official Title:** Thoratec Corporation HeartMate PHP™ Cardiogenic Shock Trial #### Organization Study ID Info **ID:** Thoratec HeartMate PHP™ CS #### Organization **Class:** INDUSTRY **Full Name:** Abbott Medical Devices ### Status Module #### Completion Date **Date:** 2017-01 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2023-07-21 **Type:** ACTUAL **Last Update Submit Date:** 2023-07-20 **Overall Status:** TERMINATED #### Primary Completion Date **Date:** 2017-01 **Type:** ACTUAL #### Start Date **Date:** 2014-10 **Type:** ACTUAL **Status Verified Date:** 2023-07 #### Study First Post Date **Date:** 2014-10-31 **Type:** ESTIMATED **Study First Submit Date:** 2014-10-29 **Study First Submit QC Date:** 2014-10-30 ### Sponsor Collaborators Module #### Collaborators **Class:** INDUSTRY **Name:** Thoratec Corporation #### Lead Sponsor **Class:** INDUSTRY **Name:** Abbott Medical Devices #### Responsible Party **Type:** SPONSOR ### Oversight Module **Oversight Has DMC:** False ### Description Module **Brief Summary:** The primary objective of this study is to assess reasonable safety and performance of the HeartMate PHP to provide hemodynamic support for up to 72 hours in patients with cardiogenic shock requiring stabilization. **Detailed Description:** The HeartMate PHP is a catheter-based axial flow pump designed to provide partial left ventricular circulatory support. This prospective, nonrandomized, controlled, single-arm, multi-center, open-label trial will evaluate the safety and performance of the device in patients with cardiogenic shock. ### Conditions Module **Conditions:** - Cardiogenic Shock **Keywords:** - PHP - Cardiogenic Shock - Percutaneous - Ventricular Assist - Heart Pump - Catheter - Thoratec Corporation ### Design Module #### Design Info **Allocation:** NA **Intervention Model:** SINGLE_GROUP ##### Masking Info **Masking:** NONE **Primary Purpose:** TREATMENT #### Enrollment Info **Count:** 9 **Type:** ACTUAL **Phases:** - NA **Study Type:** INTERVENTIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** All enrolled patients will be treated with the HeartMate PHP device **Intervention Names:** - Device: HeartMate PHP **Label:** Treatment Arm **Type:** EXPERIMENTAL ### Interventions #### Intervention 1 **Arm Group Labels:** - Treatment Arm **Description:** The HeartMate PHP system is a catheter-based heart pump and console designed to provide hemodynamic left ventricular support for up to 3 days to stabilize patients by maintaining adequate systemic cardiac output. **Name:** HeartMate PHP **Type:** DEVICE ### Outcomes Module #### Primary Outcomes **Measure:** Clinical stabilization defined as improvement of Cardiac Index to > 2.2 L/min/m2 **Time Frame:** 72 hours #### Secondary Outcomes **Measure:** An assessment of major adverse events **Time Frame:** 72 hours ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: 1. Patient is indicated for left ventricular support according to current medical guidelines ("current medical guidelines" applies only to patients enrolled in Germany) 2. Patient has a cardiac index of \< 2.2 L/min/m2 and is being treated with at least one moderate dose inotrope or at least one moderate dose of vasopressor (e.g., milrinone ≥0.3 mcg/kg/min, dopamine \> 5 mcg/kg/min, dobutamine \> 5 mcg/kg/min) AND: * PCWP \> 18 mmHg, AND * Systolic blood pressure \< 100 mmHg, AND * Decreased organ perfusion as evidenced by urine output of 50 mL/hr OR increased creatinine of 0.3 mg/dl from baseline obtained within 2 weeks, OR cool extremities 3. Written, signed, and dated informed consent Exclusion Criteria: 1. Patient is \>85 years of age 2. Right ventricular failure requiring mechanical circulatory support 3. ST elevation myocardial infarction (STEMI) within 30 days of procedure 4. Cardiac arrest within 7 days of procedure requiring CPR 5. Current treatment with mechanical circulatory device such as IABP, ECMO, centrifugal pump, etc. 6. Documented acute myocarditis 7. Active major cardiac rhythm disturbances (e.g., ventricular tachycardia, ventricular fibrillation). 8. Hypertrophic disease or any left ventricular outflow tract obstruction 9. Active sepsis defined as bacteremia, fever ≥ 101.5 degrees F 10. Mural thrombus in the left ventricle 11. History of aortic valve replacement 12. End-stage renal disease requiring dialysis 13. Documented presence of aortic stenosis (orifice area of 1.5 cm2 or less) 14. Moderate to severe aortic insufficiency (echocardiographic assessment of aortic insufficiency graded as 2 or higher) 15. Platelet count \< 100,000 16. Allergy or Intolerance to heparin, aspirin, clopidogrel, ionic and nonionic contrast media, or any other potentially required anticoagulants or antiplatelet therapy drugs 17. Known coagulopathies 18. Presence of risk factors for severe liver and/or renal dysfunction 19. Stroke within 90 days of enrollment 20. Significant peripheral vascular disease 21. History of heparin induced thrombocytopenia 22. Patient is pregnant or planning to become pregnant during the study period 23. Patient is breastfeeding. 24. Any active disease (e.g., active cancer) which could affect the patient's participation in the study, or if life expectancy is less than 1 year. 25. Any active disease in which the investigator determines would make the patient an inappropriate candidate for the study. 26. Participation in another clinical study of an investigational drug or device that has not met its primary endpoint **Maximum Age:** 85 Years **Sex:** ALL **Standard Ages:** - CHILD - ADULT - OLDER_ADULT ### Contacts Locations Module #### Locations **Location 1:** **City:** Prague **Country:** Czechia **Facility:** Na Homolce Hospital **Zip:** 130 50 **Location 2:** **City:** Prague **Country:** Czechia **Facility:** Institute for Clinical and Experimental Medicine (IKEM) **Zip:** 140 21 **Location 3:** **City:** Düsseldorf **Country:** Germany **Facility:** Universitätskliniken Düsseldorf **Zip:** 40225 **Location 4:** **City:** Hannover **Country:** Germany **Facility:** Medizinische Hochschule Hannover **Zip:** 30625 **Location 5:** **City:** Budapest **Country:** Hungary **Facility:** Semmelweis University Heart and Vascular Center **Zip:** 1122 **Location 6:** **City:** Rotterdam **Country:** Netherlands **Facility:** Erasmus Medical Center **Zip:** 3000-CA #### Overall Officials **Official 1:** **Affiliation:** Thoratec LLC, St. Jude Medical, Abbott **Name:** Pooja Chatterjee **Role:** STUDY_DIRECTOR ## Derived Section ### Condition Browse Module - Ancestors - ID: D000010335 - Term: Pathologic Processes - ID: D000009203 - Term: Myocardial Infarction - ID: D000017202 - Term: Myocardial Ischemia - ID: D000006331 - Term: Heart Diseases - ID: D000002318 - Term: Cardiovascular Diseases - ID: D000014652 - Term: Vascular Diseases - ID: D000007238 - Term: Infarction - ID: D000007511 - Term: Ischemia - ID: D000009336 - Term: Necrosis ### Condition Browse Module - Browse Branches - Abbrev: BC23 - Name: Symptoms and General Pathology - Abbrev: All - Name: All Conditions - Abbrev: BC14 - Name: Heart and Blood Diseases ### Condition Browse Module - Browse Leaves - ID: M15577 - Name: Shock - Relevance: HIGH - As Found: Shock - ID: M15578 - Name: Shock, Cardiogenic - Relevance: HIGH - As Found: Cardiogenic Shock - ID: M12155 - Name: Myocardial Infarction - Relevance: LOW - As Found: Unknown - ID: M10282 - Name: Infarction - Relevance: LOW - As Found: Unknown - ID: M10543 - Name: Ischemia - Relevance: LOW - As Found: Unknown - ID: M6546 - Name: Coronary Artery Disease - Relevance: LOW - As Found: Unknown - ID: M19506 - Name: Myocardial Ischemia - Relevance: LOW - As Found: Unknown - ID: M9419 - Name: Heart Diseases - Relevance: LOW - As Found: Unknown - ID: M17400 - Name: Vascular Diseases - Relevance: LOW - As Found: Unknown - ID: M12284 - Name: Necrosis - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000012770 - Term: Shock, Cardiogenic - ID: D000012769 - Term: Shock ### Misc Info Module #### Removed Countries - Country: Austria - Country: Czech Republic - Country: Slovenia - Version Holder: 2024-05-24

## Protocol Section ### Identification Module **NCT ID:** NCT03195179 **Brief Title:** Primary Urethral Realignment Versus Suprapubic Cystostomy After Pelvic Fracture Urethral Injury **Official Title:** The Outcomes of Primary Urethral Realignment Versus Suprapubic Cystostomy After Pelvic Fracture Urethral Injury #### Organization Study ID Info **ID:** 00094160 #### Organization **Class:** OTHER **Full Name:** University of Utah ### Status Module #### Completion Date **Date:** 2021-08-31 **Type:** ACTUAL #### Expanded Access Info #### Last Update Post Date **Date:** 2021-10-27 **Type:** ACTUAL **Last Update Submit Date:** 2021-10-25 **Overall Status:** COMPLETED #### Primary Completion Date **Date:** 2021-08-31 **Type:** ACTUAL #### Start Date **Date:** 2016-01-01 **Type:** ACTUAL **Status Verified Date:** 2021-10 #### Study First Post Date **Date:** 2017-06-22 **Type:** ACTUAL **Study First Submit Date:** 2017-06-16 **Study First Submit QC Date:** 2017-06-19 ### Sponsor Collaborators Module #### Lead Sponsor **Class:** OTHER **Name:** University of Utah #### Responsible Party **Investigator Affiliation:** University of Utah **Investigator Full Name:** Jeremy Myers **Investigator Title:** Associate Professor **Type:** PRINCIPAL_INVESTIGATOR ### Oversight Module **Is FDA Regulated Device:** False **Is FDA Regulated Drug:** False **Oversight Has DMC:** False ### Description Module **Brief Summary:** Pelvic fracture urethral injuries (PFUI) occur in up to 10% of pelvic fractures. It remains controversial whether initial urethral realignment after PFUI decreases rates of urethral obstruction and the need for subsequent urethral procedures. The retrospective record review should determine the utility of acute urethral realignment after PFUI. **Detailed Description:** A retrospective chart review to compare outcomes between urethral realignment (group 1) and suprapubic tube (SPT) placement (group 2). The comparison will be between two routinely practiced management approaches of urethral injury after pelvic fracture. Prior studies demonstrate urethral realignment is associated with a 15% to 50% reduction in urethral obstruction, however, it has also been associated with higher rates of incontinence and erectile dysfunction. Our hypothesis is that early realignment of traumatic urethral injuries after pelvic fracture lowers the incidence of complications like urethral strictures and subsequent need for surgeries. ### Conditions Module **Conditions:** - Pelvic Fracture - Urethra Tear - Urethra Injury Male - Trauma - Surgery **Keywords:** - pelvic fracture urethral injury - urethra - disruption - trauma ### Design Module #### Design Info **Observational Model:** COHORT **Time Perspective:** RETROSPECTIVE #### Enrollment Info **Count:** 200 **Type:** ACTUAL **Study Type:** OBSERVATIONAL ### Arms Interventions Module #### Arm Group 1 **Description:** Standard of care management for men with complete urethral injuries where a Foley catheter fails to be placed will have a suprapubic tube placed to manage the acute urethral injury. This is a standard of care approach and a retrospective review will be done on the patient record to determine outcomes. **Label:** Suprapubic tube placement #### Arm Group 2 **Description:** Standard of care management for men with complete urethral injuries where a Foley catheter fails to be placed will undergo urethral realignment with a combined antegrade / retrograde approach within 7 days of injury. This is a standard of care approach and a retrospective review will be done on the patient record to determine outcomes. **Label:** Urethral realignment ### Outcomes Module #### Primary Outcomes **Description:** Rates of urethral obstruction identified by urethrogram or cystoscopy **Measure:** Urethral obstruction **Time Frame:** Through study completion, an average of 1 year #### Secondary Outcomes **Description:** The rate of interventions for urethral obstruction after injury **Measure:** Treatment rate for urethral obstruction **Time Frame:** Through study completion, an average of 1 year **Description:** The gap between the 2 severed ends of the urethra **Measure:** Urethroplasty complexity - gap length during urethroplasty **Time Frame:** Through study completion, an average of 1 year **Description:** The length of bulbar mobilization **Measure:** Urethroplasty complexity - bulbar mobilization length during urethroplasty **Time Frame:** Through study completion, an average of 1 year **Description:** The need to split the 2 corporal bodies **Measure:** Urethroplasty complexity - corporal splitting during urethroplasty **Time Frame:** Through study completion, an average of 1 year **Description:** Finding the urethra was completely obstructed **Measure:** Urethroplasty complexity - total obstruction of the urethra during urethroplasty **Time Frame:** Through study completion, an average of 1 year **Description:** Finding a urethral diverticulum **Measure:** Urethroplasty complexity - urethral diverticulum discovered during urethroplasty **Time Frame:** Through study completion, an average of 1 year **Description:** Finding a urethral fistula **Measure:** Urethroplasty complexity - urethral fistula present **Time Frame:** Through study completion, an average of 1 year **Description:** The need to remove the inferior portion of the symphysis pubis **Measure:** Urethroplasty complexity - inferior pubectomy during urethroplasty **Time Frame:** Through study completion, an average of 1 year **Description:** The need to remove the complete symphysis pubis **Measure:** Urethroplasty complexity - total pubectomy during urethroplasty **Time Frame:** Through study completion, an average of 1 year **Description:** Erectile function measured by the Sexual Health Inventory for Men (SHIM) **Measure:** Erectile function- SHIM score **Time Frame:** Through study completion, an average of 1 year **Description:** Measured by the need for pharmacologic treatment of erectile dysfunction **Measure:** Erectile function - medical treatment rates **Time Frame:** Through study completion, an average of 1 year **Description:** Rates of surgical treatment of erectile dysfunction **Measure:** Erectile function - surgical treatment rates **Time Frame:** Through study completion, an average of 1 year **Description:** Rates of surgical treatment of incontinence **Measure:** Incontinence **Time Frame:** Through study completion, an average of 1 year **Description:** Calvien-Dindo grading **Measure:** Post-injury complications **Time Frame:** 3 month period post acute urethral injury ### Eligibility Module **Eligibility Criteria:** Inclusion Criteria: Men \> 18 years old Blunt force trauma Presence of pelvic fracture Urethral injury Inability to pass a Foley catheter retrograde through the injury into the bladder Exclusion Criteria: Straddle type urethral injuries without a pelvic fracture Passage of a catheter successfully in a retrograde fashion **Gender Based:** True **Gender Description:** Pelvic fracture urethral injuries are only treated by this method in men. Female urethral injuries are very rare and most often treated with immediate surgical repair. **Minimum Age:** 18 Years **Sampling Method:** NON_PROBABILITY_SAMPLE **Sex:** MALE **Standard Ages:** - ADULT - OLDER_ADULT **Study Population:** Men experiencing a traumatic pelvic fracture urethral injury where there is a major urethral disruption that prevents passage of a catheter or a retrograde cystoscope. ### Contacts Locations Module #### Locations **Location 1:** **City:** Salt Lake City **Country:** United States **Facility:** University of Utah **State:** Utah **Zip:** 84132 #### Overall Officials **Official 1:** **Affiliation:** University of Utah **Name:** Jeremy Myers, MD **Role:** PRINCIPAL_INVESTIGATOR ### IPD Sharing Statement Module **IPD Sharing:** NO ## Derived Section ### Condition Browse Module - Ancestors - ID: D000005264 - Term: Femoral Fractures - ID: D000025981 - Term: Hip 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: M17685 - Name: Wounds and Injuries - Relevance: HIGH - As Found: Injury - ID: M26370 - Name: Fractures, Bone - Relevance: HIGH - As Found: Fracture - ID: M9696 - Name: Hip Fractures - Relevance: HIGH - As Found: Pelvic Fracture - ID: M15241 - Name: Rupture - Relevance: LOW - As Found: Unknown - ID: M22785 - Name: Lacerations - Relevance: LOW - As Found: Unknown - ID: M8402 - Name: Femoral Fractures - Relevance: LOW - As Found: Unknown - ID: M23105 - Name: Hip Injuries - Relevance: LOW - As Found: Unknown - ID: M10881 - Name: Leg Injuries - Relevance: LOW - As Found: Unknown ### Condition Browse Module - Meshes - ID: D000014947 - Term: Wounds and Injuries - ID: D000050723 - Term: Fractures, Bone - ID: D000006620 - Term: Hip Fractures ### Misc Info Module - Version Holder: 2024-05-24