New cell therapy offers hope for slowing duchenne muscular dystrophy

NCT ID NCT05126758

First seen Jan 07, 2026 · Last updated Apr 25, 2026 · Updated 18 times

Summary

This study tests a cell therapy called deramiocel (CAP-1002) in 106 boys and young men with Duchenne muscular dystrophy (DMD), a genetic disease that weakens muscles over time. Participants receive either deramiocel or a placebo every 3 months for a year, then all can get the real treatment for another year. The goal is to see if deramiocel can slow the loss of arm and heart function.

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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

Conditions

Explore the condition pages connected to this study.