New gene therapy trial hopes to restore muscle protein in duchenne boys

NCT ID NCT04626674

Recruiting now Disease control Sponsor: Sarepta Therapeutics, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study tests a one-time gene therapy called delandistrogene moxeparvovec in 83 people with Duchenne muscular dystrophy. The goal is to see if it is safe and helps the body make dystrophin, a protein missing in Duchenne. The trial is now enrolling non-ambulatory participants (Cohort 8) and will follow them for up to 156 weeks.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: delandistrogene moxeparvovec (gene therapy)
What this could lead to: If successful, this could point toward a treatment that helps boys with Duchenne produce a key muscle protein, potentially slowing disease progression.
What could go wrong: This is an early Phase 1 study, so safety and effectiveness are not yet proven. Risks include liver injury and immune reactions, and long-term benefits are uncertain.

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 MUSCULAR DYSTROPHY, DUCHENNE

Contacts and locations

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Locations

Arkansas Children's Hospital
RECRUITING

Little Rock, Arkansas, 72202, United States
Children's Hospital of The King's Daughters
RECRUITING

Norfolk, Virginia, 23507, United States
Nationwide Children's Hospital
ACTIVE_NOT_RECRUITING

Columbus, Ohio, 43205, United States
Neurology Rare Disease Center
RECRUITING

Flower Mound, Texas, 75028, United States

Contact Email: •••••@•••••
Stanford University
RECRUITING

Palo Alto, California, 94304, United States

Contact Email: •••••@•••••
University of California, Davis
RECRUITING

Sacramento, California, 95616, United States
Washington University in St. Louis
RECRUITING

St Louis, Missouri, 63110, United States

Contact Email: •••••@•••••

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