Cardiac Trial for Ambulatory and Non-Ambulatory Patients with Duchenne Muscular Dystrophy

Did you know that heart failure is very common and often fatal in patients with Duchenne muscular dystrophy?

The Halt cardiOmyopathy ProgrEssion in Duchenne (HOPE-Duchenne) is a clinical trial that will evaluate whether an investigational cardiac cell therapy is safe and potentially effective in minimizing scar and improving heart function in DMD associated heart disease.

CureDuchenne and Capricor Therapeutics recently hosted a webinar that provided an overview of the investigational cardiac cell therapy (CAP-1002) and the HOPE-Duchenne trial, including its study design, eligibility criteria, study procedures and study locations.  To listen to the webinar, click here.

Here are some highlights about the clinical trial:

Eligibility

• Ambulatory or Non-ambulatory
• Age:  12+
• Weight: over 50 kg
• Documented diagnosis of Duchenne by genetic analysis
• Heart disease due to Duchenne

Study overview

• 24 patients enrolled
• Approximately 13 months of participation
• 7 clinical visits
• Study involves
  • Questions about medical history and some measures of muscle function
  • Check-ups including blood tests
  • Heart tests including a short ECG, a heart MRI, and a heart monitor worn at home
  • Breathing tests

Other considerations

• Can be included in other trials at the same time
• Travel expenses covered by Sponsor

Study sites

• Cedars-Sinai Medical Center, Los Angeles (Recruiting)
• Cincinnati Children’s Hospital Medical Center (Not Yet Recruiting)
• University of Florida, Gainesville, FL (Potential)

To find a site, see the list on www.ClinicalTrials.gov (Study Identifier NCT02485938)

Please contact debra@cureduchenne.org for more information.