Heart cell therapy shows promise for duchenne MD in major trial
NCT ID NCT05126758
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This Phase 3 trial tests a cell therapy called deramiocel (CAP-1002) in 106 boys and young men with Duchenne muscular dystrophy. Participants receive either the cell therapy or a placebo every 3 months for a year, then all can receive the therapy for another year. The goal is to see if it improves arm and hand function and protects the heart.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- deramiocel (CAP-1002), a cell therapy made from donor heart cells
- What this could lead to
- If it works, this could slow muscle decline and protect heart function in people with Duchenne muscular dystrophy, offering a new treatment option.
- What could go wrong
- This is a Phase 3 trial, but results are not yet known. The treatment may not improve function or could cause side effects like allergic reactions. It is not a cure.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Akron Children's Hospital
Akron, Ohio, 44308, United States
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Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
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Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
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Boston Children's Hospital
Boston, Massachusetts, 02115, United States
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Children's Health Specialty Care Pavilion
Dallas, Texas, 75207, United States
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital of Los Angeles, Division of Neurology
Los Angeles, California, 90027, United States
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Children's Wisconsin
Milwaukee, Wisconsin, 53226, United States
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Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
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Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
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Rare Disease Research NC LLC
Hillsborough, North Carolina, 27278, United States
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Rare Disease Research, LLC
Atlanta, Georgia, 30329, United States
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Saint Louis Children's Hospital
St Louis, Missouri, 63110, United States
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Seattle Children's
Seattle, Washington, 98105, United States
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UCSD Altman Clinical and Translational Research Institute
La Jolla, California, 92037, United States
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University of California, Davis
Sacramento, California, 95817, United States
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University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242, United States
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University of Missouri Health Care
Columbia, Missouri, 65212, United States
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University of Utah Hospital
Salt Lake City, Utah, 84112, United States
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University of Virginia Children's Hospital
Charlottesville, Virginia, 22903, United States
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