This study focuses on Duchenne Muscular Dystrophy (DMD), a genetic disorder that weakens muscles over time. Researchers are testing a new treatment called CAP-1002, a type of cell therapy, to see if it helps improve muscle function. Participants, both boys and young men, will be divided into two groups: one receiving the treatment and the other a placebo (a dummy treatment for comparison). The study lasts for two years, with visits every 3 months. Initially, participants receive 4 doses of their assigned treatment over the first year. In the second year, everyone gets CAP-1002 during an open-label phase, meaning all know they are receiving the active treatment.
- Duration: 24 months, with regular visits.
- Treatment: Participants receive CAP-1002 or placebo initially, then CAP-1002 in the second year.
- Eligibility: Boys and young men with DMD, meeting specific criteria.
This trial involves periodic assessments like muscle and heart function tests to ensure safety and measure effectiveness. Participants will be monitored closely for any side effects or adverse reactions throughout the study. If you have DMD or care for someone who does, this study may offer a chance to access new treatments under close medical supervision.