Gene therapy trial aims to stop duchenne in its tracks
NCT ID NCT06138639
First seen Jun 26, 2026 · Last updated Jul 09, 2026 · Updated 3 times
Summary
This study tests a single dose of SGT-003, a gene therapy that delivers a working version of the dystrophin gene to muscle cells. About 60 boys with Duchenne muscular dystrophy, aged from infancy to under 18, will receive the treatment and be followed for 5 years. The goal is to see if it is safe and can restore enough dystrophin to improve muscle strength and function.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- SGT-003 (a gene therapy using a modified virus to deliver a shortened dystrophin gene)
- What this could lead to
- If successful, this could provide a one-time treatment that slows or stops muscle decline in Duchenne muscular dystrophy, potentially improving quality of life and mobility.
- What could go wrong
- This is an early-phase trial with only 60 participants, so results may not apply to all. Gene therapies can cause immune reactions or liver issues, and the long-term effects are 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
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Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611-2605, United States
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Arkansas Children's Hospital
RECRUITINGLittle Rock, Arkansas, 72202, United States
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
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Children's Hospital of the King's Daughters
RECRUITINGNorfolk, Virginia, 23510, United States
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Fondazione Policlinico Universitario Agostino Gemelli IRCCS
RECRUITINGRome, 00168, Italy
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Great Ormond Street Hospital
RECRUITINGLondon, WC1N 3JH, United Kingdom
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Nationwide Children's Hospital
RECRUITINGColumbus, Ohio, 43215, United States
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Oregon Health and Sciences University
RECRUITINGPortland, Oregon, 97239, United States
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Rare Disease Research
RECRUITINGAtlanta, Georgia, 30329, United States
Contact Email: •••••@•••••
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Seattle Children's Hospital
RECRUITINGSeattle, Washington, 98105, United States
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The Hospital for Sick Children
RECRUITINGToronto, Ontario, M5G 0A4, Canada
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University of California
RECRUITINGSan Diego, California, 92037, United States
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University of California, Davis
RECRUITINGSacramento, California, 95817, United States
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University of California, Los Angeles Medical Center
RECRUITINGLos Angeles, California, 90095, United States
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Washington University in St. Louis
RECRUITINGSt Louis, Missouri, 63110, United States
Contact Email: •••••@•••••
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- 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