One-Time gene infusion aims to fight duchenne muscular dystrophy
NCT ID NCT07673809
First seen Jun 29, 2026 · Last updated Jun 30, 2026 · Updated 1 time
Summary
This study tests a single intravenous dose of a gene therapy called GNR-097 in boys aged 4 to 9 with Duchenne muscular dystrophy (DMD). The therapy uses a harmless virus to deliver a shortened version of the dystrophin gene, which is missing or faulty in DMD. The trial aims to see if the treatment is safe and whether it can improve muscle function. Some participants will receive a placebo first and may get the gene therapy later.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
GNR-097 (a gene therapy using a harmless virus to deliver a shortened dystrophin gene)
What this could lead to
If successful, this could provide a one-time treatment that helps boys with Duchenne muscular dystrophy maintain muscle strength and slow disease progression.
What could go wrong
This is an early-phase trial with only 32 participants, so results may not apply to everyone. There are risks of serious side effects like muscle inflammation, heart inflammation, or blood clotting issues.
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: •••••@•••••
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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National Medical Research Center for Children
RECRUITINGMoscow, 119991, Russia
Contact
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Regional Children's Clinical Hospital
RECRUITINGYekaterinburg, 620149, Russia
Contact
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Republican Scientific and Practical Center Mother and Child
RECRUITINGMinsk, 220053, Belarus
Contact
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Russian Children's Clinical Hospital
RECRUITINGMoscow, 117513, Russia
Contact
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Saint Petersburg State Pediatric Medical University
RECRUITINGSaint Petersburg, 194100, Russia
Contact
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Veltischev Research and Clinical Institute for Pediatrics and Pediatric Surgery of the Pirogov Russian National Research Medical University
RECRUITINGMoscow, 125412, Russia
Contact