CAP-1002 (HOPE-2 trial)

Questions and Answers

What is the HOPE-2 trial?

HOPE-2 is a Phase 2 clinical trial that will evaluate the safety and efficacy of repeat doses of CAP-1002 in boys and young men with Duchenne muscular dystrophy and reduced upper limb function.

What is CAP-1002?

CAP-1002 is a biologic product consisting of cardiosphere-derived cells (CDCs) derived from donated heart muscle. These cells have been shown to be potentially immune-modulatory, anti-inflammatory, anti-fibrotic, and regenerative. In a previous clinical trial, CAP-1002 delivered directly into the arteries of the heart of boys and young men with Duchenne-related heart disease was shown to be generally safe and well tolerated and demonstrated signals of significant and sustained improvement in cardiac and skeletal muscle function compared to participants who received usual care only.

At what stage is this research?

HOPE-2 is actively enrolling participants. Approximately 84 participants will be randomized at 10-15 medical centers in the United States. Visit the HOPE-2 page at for a current listing of trial sites.

Who is funding this trial?

This trial is sponsored and funded by Capricor, Inc.

Who is eligible for this trial?

HOPE-2 is one of the very few trials to focus on both individuals with Duchenne who are ambulatory and non-ambulatory and at least 10 years of age or older. Eligible participants must be taking steroids regularly for at least 12 months prior to screening and have reduced upper limb strength.

What happens during this trial?

  • Participants in HOPE-2 must first read and sign an informed consent form before completing any trial assessments.
  • Participants who qualify will be randomly assigned to receive intravenous (IV) infusions of CAP-1002 or placebo (an inactive substance). The chance of being assigned to the CAP-1002 group is 50/50, like flipping a coin. The treatment assignment, which remains blinded to the participant, participant’s family, and study doctor, does not change throughout the course of the trial. Participants will receive an IV infusion of CAP-1002 or placebo every three months for a total of 4 doses.
  • HOPE-2 participants will complete medical check-ups, blood tests, quality of life questionnaires, cardiac MRI, the Performance of Upper Limb module, strength testing, pulmonary function tests, and North Star Ambulatory Assessment.

How long will I be in this trial?

Participation in HOPE-2 will last for about 13 months, including the screening period, requiring about 6-7 visits to the study center.

If the results from the HOPE-2 trial suggest an appropriate risk/benefit profile of CAP-1002, Capricor, upon the recommendation of the Data Safety Monitoring Board (DSMB), plans to introduce an open-label extension study to offer CAP-1002 to participants who were randomized to placebo and completed the original trial.

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