Additional Relevant Details
The HOPE trial enrolled 25 boys with DMD who were at least 12 years of age at the time of screening and who have DMD-associated cardiomyopathy, defined as the presence of scar tissue in at least four left ventricular segments as determined by magnetic resonance imaging (MRI). The 13 subjects randomized to the active treatment arm received CAP-1002 via intracoronary infusion into each of the three main coronary arteries during a single procedure. The 12 subjects randomized to the control arm received usual care and did not receive an infusion.The primary outcome measures of the trial will consist of a broad assessment of safety and tolerability of CAP-1002. Efficacy will be evaluated according to pre-specified secondary outcome measures, including absolute and relative changes in cardiac scar tissue and cardiac function as measured by MRI, performance on the Six-Minute Walk Test (6MWT) and the Performance of the Upper Limb (PUL), and scoring on the Pediatric Quality of Life Inventory (PedsQL).