New hope for duchenne: major trial tests cell therapy to preserve muscle function
NCT ID NCT05126758
Summary
This study is testing whether a cell therapy called deramiocel can slow the loss of muscle function in boys and young men with Duchenne muscular dystrophy (DMD). About 106 participants will receive either the therapy or a placebo via IV infusion every three months for a year, with the option to continue treatment. The main goal is to see if the treatment helps preserve arm and hand function and heart health over time.
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Contacts and locations
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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
Conditions
Explore the condition pages connected to this study.