Capricor is enrolling the HOPE-2 clinical trial for Capricor’s lead investigational therapy, CAP-1002. The clinical trial will evaluate the safety and efficacy of repeat, intravenous (IV) doses of allogeneic cardiosphere-derived cells (CDCs), or CAP-1002, in boys and young men in advanced stages of Duchenne muscular dystrophy, a fatal genetic disorder for which there are limited treatment options.
While there are many clinical initiatives in Duchenne muscular dystrophy, HOPE-2 is one of the very few to focus on non-ambulant patients. These boys and young men are looking to maintain what function they have in their arms and hands, and Capricor’s previous study of a single intracoronary dose of CAP-1002 indicates the therapy may be able to help them retain those functions or to slow their loss of those functions.
For HOPE-2, the primary efficacy endpoint will be the relative change in patients’ abilities to perform manual tasks that relate to activities of daily living and are important to their quality of life. These abilities will be measured through a validated test for skeletal muscle function in Duchenne muscular dystrophy, the Performance of the Upper Limb (PUL) test. HOPE-2 will focus on the mid-level dimension of the PUL – or the ability to use muscles from the elbow to the fingers, which are essential for operating wheelchairs and performing other daily functions. HOPE-2 will measure the change from the beginning of the trial, or baseline, to Month 12.
Please visit ClinicalTrials.gov for more information regarding the HOPE-2 clinical trial.
Watch patient testimonial from the HOPE-1 clinical trial here
Watch a video on DMD and CAP-1002’s focus here