Gene therapy trial aims to stop duchenne in its tracks
NCT ID NCT06138639
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
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.
Disclaimer
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This is a summary of
the original study
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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.
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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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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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Ann & Robert H. Lurie Children's Hospital of Chicago
RECRUITINGChicago, Illinois, 60611-2605, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Arkansas Children's Hospital
RECRUITINGLittle Rock, Arkansas, 72202, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital of Philadelphia
RECRUITINGPhiladelphia, Pennsylvania, 19104, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Children's Hospital of the King's Daughters
RECRUITINGNorfolk, Virginia, 23510, United States
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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Fondazione Policlinico Universitario Agostino Gemelli IRCCS
RECRUITINGRome, 00168, Italy
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Great Ormond Street Hospital
RECRUITINGLondon, WC1N 3JH, United Kingdom
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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Nationwide Children's Hospital
RECRUITINGColumbus, Ohio, 43215, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Oregon Health and Sciences University
RECRUITINGPortland, Oregon, 97239, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Rare Disease Research
RECRUITINGAtlanta, Georgia, 30329, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Seattle Children's Hospital
RECRUITINGSeattle, Washington, 98105, United States
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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The Hospital for Sick Children
RECRUITINGToronto, Ontario, M5G 0A4, Canada
Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of California
RECRUITINGSan Diego, California, 92037, United States
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of California, Davis
RECRUITINGSacramento, California, 95817, United States
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Contact Phone: •••-•••-•••• Email: •••••@•••••
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University of California, Los Angeles Medical Center
RECRUITINGLos Angeles, California, 90095, United States
Contact Phone: •••-•••-•••• Email: •••••@•••••
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Washington University in St. Louis
RECRUITINGSt Louis, Missouri, 63110, United States
Contact
Contact Phone: •••-•••-•••• Email: •••••@•••••