Allogeneic Cardiosphere-Derived Cells for Duchenne Muscular Dystrophy Cardiomyopathy
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.
Randomized, open-label, placebo-controlled. Multi-vessel intra-coronary delivery, single dose.
Primary: Safety and tolerability in DMD patients. Secondary: Structural or functional cardiac benefits, quality of life improvements.
Fully enrolled. 6-month interim analysis showed significant improvement in some measures of cardiac and upper limb function. Capricor received RMAT designation from the US FDA for this DMD cell-based therapy in February, 2018.