HOPE 2 TRIAL 2018-05-25T00:34:04+00:00

The HOPE-2 Clinical Trial

The HOPE-2 trial is evaluating the safety and efficacy of repeat doses of CAP-1002 in boys and young men with Duchenne muscular dystrophy (DMD) and reduced upper limb function.

HOPE-2 is a Phase 2, randomized, double-blind, placebo-controlled clinical trial that will randomize up to 84 participants at 10-15 medical centers in the United States.

Treatment Groups

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.

Trial Sites

About CAP-1002

CAP-1002 is the code name given to the new experimental therapy. It is a biologic product derived from donated heart muscle and consists of cardiosphere-derived cells, or CDCs, a unique population of cells that contains cardiac progenitor cells. CAP-1002 has been shown to exert potent systemic effects that modulate immunologic activity, reduce inflammation and fibrosis, and stimulate cellular regeneration in both cardiac and skeletal muscle.

In the first trial, called the HOPE-Duchenne trial, a single dose of CAP-1002 delivered directly into the arteries of the heart of boys and young men with DMD-related heart disease was shown to be generally safe and well tolerated and demonstrated significant and sustained improvement in cardiac and skeletal muscle function compared to participants who received usual care only.

The HOPE-2 trial is the next step in evaluating the safety and effectiveness of CAP-1002 in treating patients with DMD.

About Duchenne Muscular Dystrophy

Duchenne Muscular Dystrophy (DMD) is a genetic disorder that is diagnosed in childhood and affects approximately one in every 3,600 live male births. DMD is caused by a mutation in the gene that codes for the dystrophin protein. Without functional dystrophin, muscles suffer progressive damage through normal use, leading to the loss of the ability to walk and perform other activities of daily living.

Over time, the muscles that help patients with DMD breathe and cause their hearts to pump will weaken. DMD is a progressive disease. There is no cure for DMD.