HOPE - Halt cardiOmyopathy ProgrEssion in Duchenne

 

Frequently Asked Questions (FAQs) about this study:

 

What is the HOPE-Duchenne trial?

  • HOPE-Duchenne is a Phase II clinical trial that evaluates if CAP-1002, an investigational cardiac stem cell therapy, is safe and potentially effective in treating boys and men with heart disease related to Duchenne muscular dystrophy. This trial includes an assessment of the amount of scar tissue in the heart muscle before and after infusion of the investigational cardiac stem cell therapy compared to scar remaining after usual care.
  • Enrollment in the HOPE-Duchenne trial closed in September 2016. Final results are anticipated in November 2017.

 

What is CAP-1002?

  • CAP-1002 is a biologic product consisting largely of adult heart stem cells grown from donated tissue from adult heart muscle. These cells have been shown to be potentially anti-inflammatory, anti-fibrotic, and reduce cell death. When studied in the setting of a heart attack in adults, CAP-1002 has been shown to reduce the size of scar in the heart and increase viable muscle.

 

Who funded this trial?

  • This trial is funded by Capricor, Inc with support from the California Institute for Regenerative Medicine (CIRM).

 

 What happens in this trial?

  • Participants that qualified for the trial were randomly assigned to one of two treatment groups: 1) those who received CAP-1002 and usual care that you would normally get from your doctor, or 2) those who receive usual care alone.
  • Participants randomly assigned to receive CAP-1002 visited the study hospital to have the cells infused into the arteries of their heart. The whole treatment took about 1 hour, and an overnight hospital stay was needed for observation.
  • This trial involves questions about medical history, and regular check-ups including blood tests, ECGs, heart MRIs, a heart monitor worn at home, simple measures of muscle function, and breathing tests.
  • Participants will participate in this trial for approximately 13 months. HOPE is being conducted at three U.S. sites.

 

Are any results available?

Capricor recently completed the 6-month interim analysis of the HOPE-Duchenne trial. Below is a summary of the results presented in a public webinar on April 25th, 2017, which can be access on Capricor’s website: http://capricor.com/news/events/

  • 25 participants were randomized in the trial. 13 were assigned to the CAP-1002 treatment group, and 12 were assigned to the usual care treatment group.
  • CAP-1002 has been well-tolerated by participants in the HOPE trial. The most common adverse events were atrial fibrillation (20%) and the common cold (16%). The atrial fibrillation is an irregular heartbeat that occurred during the delivery of CAP-1002 in the arteries of the heart. The atrial fibrillation resolved on its own, and participants did not experience any other symptoms. No serious adverse events were evaluated as related to the CAP-1002 investigational product.
  • We observed approximately a 5% reduction in measured scar size in the hearts of those who received CAP-1002 when compared to a negligible change in the usual care group, which appears consistent with the natural history of DMD Glossary Link cardiomyopathy. Although this did not achieve statistical significance (p=0.09), this is a notable observation, since scar progressively increases over time in DMD.
  • Improvements were observed in the heart’s wall thickening in those who received CAP-1002 compared to the usual care group. Wall thickening is very important to the heart’s ability to contract correctly and effectively push out blood to your body. Statistically-significant increases were seen in thickening of the inferior wall of the left ventricle (p<0.05). Wall thickening of the anterior and later walls in the CAP-1002 group also trended towards improvement, but did not meet statistical significance.
  • Significant improvements in ejection fraction as well as in end-systolic and end-diastolic volumes were not observed. This was not an unexpected finding after a single dose of CAP-1002 and short term heart function monitoring.
  • In the Performance of Upper Limb (PUL), there was a significant improvement in upper limb function in the middle (i.e., elbow) and distal (i.e., hands) levels at Week 6 (p<0.05) in the CAP-1002 treatment group compared to the usual care group via responder analysis. However, this effect became less pronounced by Month 3, and disappeared by Month 6.

 

 What are future plans for CAP-1002 in DMD?

  • The HOPE-Duchenne trial will continue throughout 2017 until the last Month 12 visit is complete. After which, if the one-year results of the study demonstrate that CAP-1002 is safe according to an independent data review committee, participants randomly assigned to usual care group will be eligible to receive the stem cell infusion after completing the trial.
  • Finally, Capricor is evaluating repeat dosing of CAP-1002 via intravenous infusion in patients with DMD. Additional details on this next trial will be provided as plans mature.

 

Where can I learn more?

  • For information about Capricor and HOPE-Duchenne trial, please visit www.capricor.com and our ClinicalTrials.gov site at NCT02485938.

 

Updated 5/11/2017