Ignite Creation Date:
2024-05-06 @ 4:53 PM
Last Modification Date:
2024-10-26 @ 2:18 PM
Study NCT ID:
NCT05126758
Status:
RECRUITING
Last Update Posted:
2024-04-01
First Post:
2021-11-04
Brief Title:
A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy
Sponsor:
Capricor Inc
Organization:
Capricor Inc
Study Overview
Official Title:
A Phase 3 Randomized Double-Blind Placebo-Controlled Trial Evaluating the Efficacy and Safety of Human Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy
Status:
RECRUITING
Status Verified Date:
2024-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
HOPE-3
Brief Summary:
HOPE-3 is a two cohort Phase 3 multi-center randomized double-blind placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy DMD and impaired skeletal muscle function Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during the first 12-months of the study All participants will be eligible to receive 4 doses of CAP-1002 for an additional 12 months as part of an open-label extended assessment period
Detailed Description:
Up to 102 eligible study subjects will participate in this two cohort study Cohort A will enroll approximately 58 subjects randomized in a 11 ratio activeplacebo Subjects randomized to the active treatment group in Cohort A will receive CAP-1002A manufactured at Capricors manufacturing facility in Los Angeles CA Once Cohort A enrollment is completed Cohort B enrollment will begin Cohort B will enroll approximately 44 participants randomized in a 11 ratio activeplacebo Subjects randomized to the active treatment group in Cohort B will receive CAP-1002B manufactured at Capricors manufacturing facility in San Diego CA Both cohorts will include visits at Screening BaselineDay 1 Month 1 and Months 3 6 9 and 12 Subjects will receive IV infusions of CAP-1002 or placebo on Day 1 and Months 3 6 and 9 All subjects will then be eligible to receive additional IV infusions of CAP-1002 at Month 12 15 18 and 21 as part of the open-label phase of the study
A primary analysis of efficacy and safety will be performed for each individual cohort at Month 12 following 4 administrations of CAP-1002 or placebo
The primary efficacy endpoint is the mean change from baseline in upper limb function as assessed by Performance of the Upper Limb test version 20 PUL 20 Total Score at the 12-month time point Secondary endpoints evaluated at the 12-month time point include assessment of changes in cardiac muscle function and structure by cardiac magnetic resonance imaging cMRI changes in hand-to-mouth function eat 10-bites assessed by the Duchenne Video Assessment DVA quality of life assessments and biomarker analysis for creatine kinase MB isoenzyme CK-MB
Safety evaluations will include adverse events concomitant medications physical exam vital signs and clinical laboratory testing
Following the initial 4 infusions in Cohort A or Cohort B all subjects will be eligible to participate in a 12-month open label extended assessment period and receive up to 4 additional IV infusions of CAP-1002 once every 3 months An analysis of extended safety and efficacy will be conducted after all subjects have completed the Month 24 visit
A primary analysis of efficacy and safety will be performed for each individual cohort at Month 12 following 4 administrations of CAP-1002 or placebo
The primary efficacy endpoint is the mean change from baseline in upper limb function as assessed by Performance of the Upper Limb test version 20 PUL 20 Total Score at the 12-month time point Secondary endpoints evaluated at the 12-month time point include assessment of changes in cardiac muscle function and structure by cardiac magnetic resonance imaging cMRI changes in hand-to-mouth function eat 10-bites assessed by the Duchenne Video Assessment DVA quality of life assessments and biomarker analysis for creatine kinase MB isoenzyme CK-MB
Safety evaluations will include adverse events concomitant medications physical exam vital signs and clinical laboratory testing
Following the initial 4 infusions in Cohort A or Cohort B all subjects will be eligible to participate in a 12-month open label extended assessment period and receive up to 4 additional IV infusions of CAP-1002 once every 3 months An analysis of extended safety and efficacy will be conducted after all subjects have completed the Month 24 visit
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None