A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (NCT05126758)
A Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial Evaluating the Efficacy and Safety of Human Allogeneic Cardiosphere-Derived Cells for the Treatment of Duchenne Muscular Dystrophy
🩺 Plain-English Summary (8th-Grade Level)
HOPE-3 is a two cohort, Phase 3, multi-center, randomized, double-blind, placebo-controlled clinical trial evaluating the efficacy and safety of a cell therapy called deramiocel (CAP-1002) in study participants with Duchenne muscular dystrophy (DMD) and impaired skeletal muscle function. Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either deramiocel or placebo every 3 months for a total of 4 doses during the first 12 months of the study. All participants will be eligible to receive 4 doses of deramiocel for an additional 12 months as part of an open-label extended assessment period. After completion of the first open-label extension (Months 12-24), subjects who have completed Month 24 are eligible to continue onto a Long-Term Open-Label Extension period that will provide treatment with deramiocel until commercial availability, or until sponsor's decision to terminate the trial, or the participant withdraws consent.
Who can join: Review the eligibility criteria below.
Location: Phoenix Children's Hospital — Phoenix, Arizona
Age range: 10 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (20 Sites)
- Phoenix Children's Hospital — Phoenix, Arizona
- Arkansas Children's Hospital — Little Rock, Arkansas
- UCSD Altman Clinical and Translational Research Institute — La Jolla, California
- Children's Hospital of Los Angeles, Division of Neurology — Los Angeles, California
- University of California, Davis — Sacramento, California
- Children's Hospital Colorado — Aurora, Colorado
- Rare Disease Research, LLC — Atlanta, Georgia
- Ann & Robert H. Lurie Children's Hospital of Chicago — Chicago, Illinois
- University of Iowa Hospitals and Clinics — Iowa City, Iowa
- Boston Children's Hospital — Boston, Massachusetts
- University of Missouri Health Care — Columbia, Missouri
- Saint Louis Children's Hospital — St Louis, Missouri
- Rare Disease Research NC LLC — Hillsborough, North Carolina
- Akron Children's Hospital — Akron, Ohio
- Cincinnati Children's Hospital Medical Center — Cincinnati, Ohio
- Children's Health Specialty Care Pavilion — Dallas, Texas
- University of Utah Hospital — Salt Lake City, Utah
- University of Virginia Children's Hospital — Charlottesville, Virginia
- Seattle Children's — Seattle, Washington
- Children's Wisconsin — Milwaukee, Wisconsin
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Muscular Dystrophies
- Genetic and Physical Study of Childhood Nerve and Muscle Disorders — Bethesda, Maryland
Explore by condition: All Muscular Dystrophies clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.