HOPE-Duchenne (Halt cardiomyOPathy progrEssion in Duchenne) — HOPE  

Duchenne Muscular Dystrophy Clinical Trial

Official title: A Randomized, Open-label Study of the Safety and Efficacy of Multi- Vessel Intracoronary Delivery of Allogeneic Cardiosphere-Derived Cells in Patients With Cardiomyopathy Secondary to Duchenne Muscular Dystrophy

Status Completed

Clinical Trial Summary

Male subjects with cardiomyopathy secondary to Duchenne muscular dystrophy (DMD) meeting all inclusion and no exclusion criteria will be randomized. All subjects will be at least 12 years of age. They will be randomized in a 1:1 manner to either intracoronary infusion of CAP-1002 in three coronary arteries supplying the three major cardiac territories of the left ventricle of the heart (anterior, lateral, inferior/posterior) or usual care. In the active treatment arm, all three major cardiac territories will be treated (infused) during a single procedure in an open-label fashion.

Clinical Trial Description

Approximately 24, and not more than 30, subjects will be randomized into the study, in two sequential enrollment groups. Safety data from Group 1 will undergo a Data Safety Monitoring Board (DSMB) review prior to initiation of enrollment for Group 2.

The first 6-8 randomized subjects will comprise Group 1, and will include a minimum of 3 subjects completing intracoronary infusion with CAP-1002. The DSMB will conduct a review of interim safety data through 72 hours post-Day 0 for at least 3 infused subjects and for at least 6 subjects overall.

Enrollment of Group 2 will begin per DSMB recommendations following their review of the 72 hour safety data from Group 1. Group 2 will include approximately 18 subjects. Screening and randomization will continue until at total of 12 subjects are infused with CAP 1002 or 30 subjects are randomized into the study, whichever comes first.

All subjects assigned to the active treatment arm will receive an intended total dose of up to 75 million (M) CAP-1002 cells infused as 25M cells into each of the three left ventricle cardiac territories (anterior, lateral, inferior/posterior).

Subjects randomized to receive usual care will continue to be cared for and treated in whatever manner the investigator deems most appropriate for the subject on an ongoing basis, and will receive no infusion.

Randomization will take place within 30 days of the first screening procedure. After completion of the screening procedures, eligible subjects randomized to active treatment arm will receive CAP-1002 administered via intracoronary infusion on Day 0. Day 0 for eligible subjects randomized to the usual care arm will occur 7 days after the date of randomization. All randomized subjects will have a follow-up telephone call on Study Day 3, and study visits at Weeks 2 and 6, and at Months 3, 6 and 12 post Day 0. ;

Related Conditions & MeSH terms

• Cardiomyopathies
• Cardiomyopathy
• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

• NCT number: NCT02485938
• Study type: Interventional
• Source: Capricor Inc.
• Status: Completed
• Phase: Phase 1/Phase 2
• Start date: January 2016
• Completion date: September 2017