Ignite Creation Date:
2024-05-06 @ 11:00 AM
Last Modification Date:
2024-10-26 @ 12:38 PM
Study NCT ID:
NCT03406780
Status:
COMPLETED
Last Update Posted:
2020-06-05
First Post:
2018-01-15
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 2 Randomized Double-Blind Placebo-Controlled Trial Evaluating the Safety and Efficacy of Intravenous Delivery of Allogeneic Cardiosphere-Derived Cells in Subjects With Duchenne Muscular Dystrophy
Status:
COMPLETED
Status Verified Date:
2020-06
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-2
Brief Summary:
HOPE-2 is a double-blind clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy DMD 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 a 12-month period
Detailed Description:
Approximately 84 eligible study participants will be randomized to either CAP-1002 or placebo in a 11 ratio
The trial will include visits at Screening BaselineDay 1 Week 4 and Months 3 6 9 and 12 with IV infusions of CAP-1002 or placebo on Day 1 and Months 3 6 and 9
Safety evaluations will include adverse events concomitant medications physical exam vital signs 12-lead ECG and clinical laboratory testing
Efficacy will be evaluated in the Performance of the Upper Limb pulmonary function testing North Star Ambulatory Assessment ambulatory subjects only strength testing cardiac MRI and quality of life
If trial data suggests an appropriate riskbenefit profile of CAP-1002 Capricor upon the recommendation of the Data Safety Monitoring Board DSMB will introduce an open-label extension study to offer CAP-1002 to study participants who were randomized to placebo and completed all trial visits during the 12-month period
The trial will include visits at Screening BaselineDay 1 Week 4 and Months 3 6 9 and 12 with IV infusions of CAP-1002 or placebo on Day 1 and Months 3 6 and 9
Safety evaluations will include adverse events concomitant medications physical exam vital signs 12-lead ECG and clinical laboratory testing
Efficacy will be evaluated in the Performance of the Upper Limb pulmonary function testing North Star Ambulatory Assessment ambulatory subjects only strength testing cardiac MRI and quality of life
If trial data suggests an appropriate riskbenefit profile of CAP-1002 Capricor upon the recommendation of the Data Safety Monitoring Board DSMB will introduce an open-label extension study to offer CAP-1002 to study participants who were randomized to placebo and completed all trial visits during the 12-month period
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