HOPE-Duchenne is a phase I/II randomized, controlled, open-label trial designed to evaluate the safety and explore the efficacy of intracoronary CAP-1002, in patients with DMD with cardiomyopathy. Here we report the results of Halt Cardiomyopathy Progression (HOPE)–Duchenne, a clinical trial of allogeneic CDCs (CAP-1002). Cardiac function and structure were assessed by MRI. Given the preclinical observations of improved skeletal muscle function, we also investigated changes in performance of the upper limb (PUL) and other assessments of dystrophic skeletal muscle function.
Unlike most clinical trials in DMD, which target younger ambulatory patients, we studied an older population with more advanced disease. HOPE participants were predominantly nonambulatory and had substantial cardiomyopathy without heart failure; they had severe limitation in shoulder function but preserved, albeit compromised, middle and distal upper limb function. Thus, in the HOPE population, both cardiac and upper limb function were optimally situated between severe loss of function, from which recovery would be extraordinary, and normal function, upon which it is impossible to improve.
The concordant signals of efficacy in the HOPE-Duchenne trial in heart and skeletal muscle suggest not just slowed disease progression, but net improvements from baseline. In brief, we report that a treatment originally targeted at DMD cardiomyopathy could potentially benefit both cardiac and skeletal muscle.