Capricor Therapeutics completes enrollment in randomized clinical trial for Duchenne muscular dystrophy

Written by Elena Conroy

The subjects will received an allogeneic cardiac cell therapy for the treatment of heart disease, the most common cause of death in Duchenne muscular dystrophy sufferers.

Capricor Therapeutics, Inc. (CA,USA) a clinical-stage biotechnology company developing biological therapies for a number of medical conditions, announced that the randomized Phase I/II HOPE-Duchenne clinical trial has completed its enrollment with 25 subjects. HOPE (Halt cardiomyOPathy progrEssion in Duchenne) is evaluating CAP-1002, Capricor’s lead investigational cardiac cell therapy, in Duchenne muscular dystrophy-associated cardiomyopathy.

Duchenne muscular dystrophy (DMD) affects approximately 20,000 boys and young men in the U.S. It is caused by a genetic abnormality in the dystrophin complex, a structural element which plays a critical role in muscle fiber integrity. The disease leads to chronic skeletal and cardiac muscle damage and following years of progressive weakness, patients often die in their twenties, most commonly due to heart disease.

“It is accepted that the most common cause of death from DMD relates to the impact of this disease on heart muscle, “ stated John Jefferies, director of the advanced heart failure and cardiomyopathy programs at Cincinnati Children’s Hospital and Principal Investigator of the HOPE-Duchenne clinical trial. “The enthusiasm for HOPE-Duchenne has been remarkable, and we are excited to see the upcoming results of this innovative study.”

The HOPE trial enrolled 25 boys with DMD who were at least 12 years of age at the time of screening and who have DMD-associated cardiomyopathy, defined as the presence of scar tissue in at least four left ventricular segments as determined by magnetic resonance imaging (MRI). The 13 subjects randomized to the active treatment arm received CAP-1002, an allogeneic cardiac cell therapy for the treatment of heart disease, via intracoronary infusion into each of the three main coronary arteries during a single procedure. The 12 subjects randomized to the control arm received usual care and did not receive an infusion. The primary outcome measures of the trial will consist of a broad assessment of safety and tolerability of CAP-1002.

“The rate of patient enrollment into HOPE-Duchenne far surpassed our expectations, signifying the need for therapeutic options as well as the desire of the DMD community to address the heart disease that is highly prevalent in this population,” explained Linda Marbán, president and chief executive officer of Capricor. “We look forward to announcing top-line six-month results from HOPE-Duchenne in the first quarter of next year, in which we will report on the safety as well as the potential efficacy of CAP-1002. In DMD, scar tissue progressively aggregates in the heart, leading to a deterioration of cardiac function for which treatment options are limited. We believe CAP-1002 is the only therapeutic candidate in development for the treatment of DMD that has been clinically shown to reduce scar tissue in the damaged heart.”

HOPE-Duchenne is being funded in part through the support of the California Institute for Regenerative Medicine (CA, USA) and is being conducted at Cincinnati Children’s Hospital Medical Center (OH, USA), Cedars-Sinai Heart Institute (CA, USA) and the University of Florida in Gainesville (FLO, USA). Capricor expects to report top-line six-month data from this trial in the first quarter of 2017.

For more information on the trail, please visit or (NCT02485938).