Grant Award Details
To complete a Phase II clinical trial in Duchenne Muscular Dystrophy patients with cardiomyopathy, in which allogeneic Cardiosphere Derived Cells (CDCs) [aka CAP-1002] are infused one time via multi-vessel intracoronary delivery.
Progress Reports
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Grant Application Details
- Allogeneic Cardiosphere-Derived Cells for Duchenne Muscular Dystrophy Cardiomyopathy
Therapeutic Candidate or Device
Allogeneic Cardiosphere-Derived Cells (CDCs, CAP-1002)
Indication
Duchenne Muscular Dystrophy Cardiomyopathy
Therapeutic Mechanism
Secretion of identified bioactive elements will reduce myocardial fibrosis and improve cardiac function.
Unmet Medical Need
Heart failure is the leading cause of death among young men with Duchenne Muscular Dystrophy. No specific therapies exist to treat this element of the disease. CAP-1002 is intended to treat the cardiomyopathy associated with DMD.
Project Objective
Phase 2 study completed
Major Proposed Activities
- manufacture CAP-1002 in quantities sufficient to treat all subjects enrolled in the trial
- enroll and treat all subjects per the clinical protocol
- enroll and treat all subjects per the clinical protocol
The applicant is a California-based organization. At least one clinical site within California will participate in the study.
Publications
- Neurology (2019) Cardiac and skeletal muscle effects in the randomized HOPE-Duchenne trial. (PubMed: 30674601)