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: 

Capricor is using donor cells derived from the heart to treat patients suffering from Duchenne Muscular Dystrophy (DMD), a genetic disorder that leads to progressive muscle degeneration, including heart muscle. One of the leading causes of death for children with DMD is heart failure and the aim of this treatment is to help improve heart muscle outcomes for these patients.

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.

Updates: 

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