Participants who meet all eligibility criteria will be randomly assigned to receive intravenous (IV) CAP-1002 or placebo (an inactive substance). The chance of being assigned to the CAP-1002 treatment group is 50/50, like flipping a coin.
The treatment assignment will remain blinded throughout the trial, which means that neither the participant, nor the participant’s family, study doctor, or any researcher will know whether the participant was assigned CAP-1002 or placebo.
Participants will receive an IV infusion of CAP-1002 or placebo every three months for a total of 4 doses. If trial evidence suggests an appropriate risk/benefit profile of CAP-1002, Capricor, upon the recommendation of the Data Safety Monitoring Board (DSMB), may introduce an open-label extension study to offer CAP-1002 to participants who were randomized to placebo and completed the original trial.
Participation in HOPE-2 will last for about 13 months, including the screening period, and includes 6-7 visits to the study center.
HOPE-2 is one of the very few trials to focus on both ambulant and non-ambulant individuals 10 years of age and older. A specific DMD genetic mutation is not required for participation. Eligible participants must be taking steroids regularly for at least 12 months prior to screening and have reduced upper limb strength. Click here for additional eligibility criteria.
The primary efficacy endpoint in the HOPE-2 trial is the change in participants’ abilities to perform the Performance of Upper Limb (PUL) module. The PUL test is a physical test that was developed specifically for, and with, patients with DMD to evaluate their ability to perform manual tasks that relate to activities of daily living and are important to quality of life. As part of the trial, HOPE-2 participants will also complete medical check-ups, blood tests, quality of life questionnaires, cardiac MRI, strength testing, pulmonary function tests, and North Star Ambulatory Assessment.