HOPE-Duchenne Phase I/II Clinical Trial
The HOPE-Duchenne clinical trial evaluated the safety and efficacy of a single intracoronary dose of Capricor Therapeutics’ novel cell therapy, CAP-1002, in boys and young men in advanced stages of Duchenne muscular dystrophy.
The Phase I/II, randomized, controlled, open-label trial found that CAP-1002 was generally safe, well-tolerated and demonstrated significant and sustained signals of improvement in cardiac and skeletal muscle function in patients with Duchenne muscular dystrophy, a fatal genetic disease with limited treatment options.
The HOPE-Duchenne clinical trial enrolled 25 patients and was conducted at three U.S. centers. All participants had significant cardiac scarring and approximately two-thirds were wheelchair-dependent at the time they began the trial. During the 12-month course of the trial, all patients received the standard-of-care for Duchenne muscular dystrophy, including oral steroids, and 13 also received one dose of intracoronary CAP-1002 upon randomization.
Using the Performance of the Upper Limb (PUL) test, which is a validated functional assessment of upper limb activities of daily living, Capricor found significant and sustained improvement in the mid-distal PUL, which measures from the elbow, in eight of the nine lower functioning CAP-1002 patients, while the control patients had no improvement.
We also reported that magnetic resonance imaging (MRI) found the participants who received CAP-1002 had significant scar reduction and improvement in inferior wall systolic thickening compared to the usual care group. Progressive cardiac scarring eventually impairs the heart’s pumping ability and is the leading cause of death in Duchenne muscular dystrophy.
The trial was funded, in part, through the support of the California Institute for Regenerative Medicine (CIRM).
For more information please click here for the 12-month Results of the HOPE Clinical Trial presented at the AHA Scientific Sessions 2017.
Please visit ClinicalTrials.gov for more information regarding the HOPE clinical trial.