One-Time gene infusion aims to fight duchenne muscular dystrophy

NCT ID NCT07673809

Recruiting now Disease control Sponsor: AO GENERIUM Source: ClinicalTrials.gov ↗

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.

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.

Duchenne muscular dystrophy

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

DUCHENNE MUSCULAR DYSTROPHY

Contacts and locations

Locations

National Medical Research Center for Children
RECRUITING

Moscow, 119991, Russia
Regional Children's Clinical Hospital
RECRUITING

Yekaterinburg, 620149, Russia
Republican Scientific and Practical Center Mother and Child
RECRUITING

Minsk, 220053, Belarus
Russian Children's Clinical Hospital
RECRUITING

Moscow, 117513, Russia
Saint Petersburg State Pediatric Medical University
RECRUITING

Saint Petersburg, 194100, Russia
Veltischev Research and Clinical Institute for Pediatrics and Pediatric Surgery of the Pirogov Russian National Research Medical University
RECRUITING

Moscow, 125412, Russia

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