One-Time gene therapy aims to restore muscle in boys with duchenne
NCT ID NCT05693142
First seen Jun 27, 2026 · Last updated Jul 22, 2026 · Updated 4 times
Summary
This study tests a one-time gene therapy called RGX-202 in boys with Duchenne muscular dystrophy (DMD). The therapy delivers a mini version of the missing dystrophin protein to muscle cells. Researchers will check safety and whether it improves muscle function, like standing and walking. The trial enrolls 65 boys aged 1 to 12.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- RGX-202 (a gene therapy that delivers a microdystrophin protein to muscle cells)
- What this could lead to
- If successful, this could provide a one-time treatment that slows or stops muscle decline in Duchenne muscular dystrophy, helping boys maintain strength and mobility longer.
- What could go wrong
- This is an early-to-mid-stage trial with only 65 participants, so results may not apply to everyone. Gene therapies can cause immune reactions or other side effects, and the long-term effects are still unknown.
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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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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BC Children's Hospital
Vancouver, British Columbia, V65 3N1, Canada
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Children's Hospital Colorado
Aurora, Colorado, 80045, United States
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Children's Hospital London Health Science Centre
London, Ontario, Canada
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Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1, Canada
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Children's Hospital of Orange County
Orange, California, 92868, United States
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Children's Hospital of Richmond at Virginia Commonwealth University
Richmond, Virginia, 23298, United States
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Children's Hospital of the King's Daughters
Norfolk, Virginia, 23510, United States
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Cincinnati Children's
Cincinnati, Ohio, 45229, United States
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Columbia University Medical Center
New York, New York, 10032, United States
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Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503, United States
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Monroe Carell Children's Hospital at Vanderbilt
Nashville, Tennessee, 37232, United States
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Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
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Oregon Health & Science University
Portland, Oregon, 97239, United States
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Rare Disease Research
Atlanta, Georgia, 30329, United States
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Stanford School of Medicine /Division of Neuromuscular Medicine
Palo Alto, California, 94304, United States
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The Hospital for Sick Children
Toronto, Ontario, M5G 1X8, Canada
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The University of Texas Southwestern Medical Center
Dallas, Texas, 75390, United States
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University of Florida
Gainesville, Florida, 32610, United States
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University of Iowa
Iowa City, Iowa, 52242, United States
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University of Kansas Medical Center
Kansas City, Kansas, 60160, United States
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University of Massachusetts Chan Medical School
Worcester, Massachusetts, 01608, United States
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can a lower steroid dose preserve strength in young boys with DMD?
- Can a targeted infusion slow muscle decline in duchenne? a new trial aims to find out.
- Can a massive patient database unlock new treatments for muscular dystrophy?
- Umbilical cord stem cells aim to slow muscle loss in duchenne boys
- Experimental drug aims to preserve arm and lung function in boys with advanced duchenne
- Experimental drug KER-065 aims to slow muscle decline in duchenne muscular dystrophy