CIRM Funded Clinical Trials

Allogeneic Cardiosphere-Derived Cells for Duchenne Muscular Dystrophy Cardiomyopathy


Deborah Ascheim
Investigator:
Institution:
CIRM Grant:
Award Value:
$3,376,259
Trial Sponsor:
Trial Stage: 
Phase 2
Trial Status: 
Active, not recruiting
Targeted Enrollment:
24
ClinicalTrials.gov ID:
Details: 

Heart failure is a leading cause of death for Duchenne muscular dystrophy patients. Cardiosphere-derived cells (CDCs) decrease myocardial fibrosis, improve cardiac function and induce regeneration of heart muscle inĀ  preclinical models of DMD.

Design: 

Randomized, open-label, placebo-controlled. Multi-vessel intra-coronary delivery, single dose.

Goal: 

Primary: Safety and tolerability in DMD patients. Secondary: Structural or functional cardiac benefits, quality of life improvements.

Status: 

Fully enrolled. 6-month interim analysis showed significant improvement in some measures of cardiac and upper limb function. Award end projected Dec. 2017 (early)