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Could heart cells help muscles? new trial for duchenne MD

NCT ID NCT05126758

First seen Jan 07, 2026 · Last updated Jun 23, 2026 · Updated 30 times

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 cells or a placebo every 3 months for a year, then all can get the cells for another year. The goal is to see if the treatment improves arm and hand function and protects the heart.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Locations

  • Akron Children's Hospital

    Akron, Ohio, 44308, United States

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    Chicago, Illinois, 60611, United States

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202, United States

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • Children's Health Specialty Care Pavilion

    Dallas, Texas, 75207, United States

  • Children's Hospital Colorado

    Aurora, Colorado, 80045, United States

  • Children's Hospital of Los Angeles, Division of Neurology

    Los Angeles, California, 90027, United States

  • Children's Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Phoenix Children's Hospital

    Phoenix, Arizona, 85016, United States

  • Rare Disease Research NC LLC

    Hillsborough, North Carolina, 27278, United States

  • Rare Disease Research, LLC

    Atlanta, Georgia, 30329, United States

  • Saint Louis Children's Hospital

    St Louis, Missouri, 63110, United States

  • Seattle Children's

    Seattle, Washington, 98105, United States

  • UCSD Altman Clinical and Translational Research Institute

    La Jolla, California, 92037, United States

  • University of California, Davis

    Sacramento, California, 95817, United States

  • University of Iowa Hospitals and Clinics

    Iowa City, Iowa, 52242, United States

  • University of Missouri Health Care

    Columbia, Missouri, 65212, United States

  • University of Utah Hospital

    Salt Lake City, Utah, 84112, United States

  • University of Virginia Children's Hospital

    Charlottesville, Virginia, 22903, United States

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 type of heart-derived cell

What this could lead to

If it works, this could slow the loss of arm and hand function and help preserve heart muscle in boys and young men with Duchenne muscular dystrophy.

What could go wrong

This is an early-stage phase 3 trial with only 106 participants, so results may not apply to everyone. The treatment requires repeated infusions and may not improve function for all patients.

Conditions

The condition(s) this trial relates to.

atrophic muscular disease Duchenne muscular dystrophy hereditary disease muscle tissue disorder Muscular Disorders, Atrophic muscular dystrophy nervous system disorder neuromuscular disease X-linked disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.