Experimental gene therapy targets rare form of duchenne muscular dystrophy

NCT ID NCT04240314

Completed Disease control Sponsor: Megan Waldrop Source: ClinicalTrials.gov ↗

First seen Jun 26, 2026 · Last updated Jun 26, 2026

Summary

This early-stage trial tested a single-dose gene therapy called scAAV9.U7.ACCA in 3 boys with Duchenne muscular dystrophy caused by a duplication of exon 2. The therapy was given through a vein and aimed to help muscle cells produce dystrophin, a protein missing in this disease. The main goal was to check for serious side effects, with a secondary look at whether dystrophin levels increased in muscle biopsies.

What this could mean

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

Active substance: scAAV9.U7.ACCA (a gene therapy given as a single injection into a vein)
What this could lead to: If successful, this could point toward a treatment that helps boys with Duchenne muscular dystrophy produce a missing protein, potentially slowing muscle damage.
What could go wrong: This is a very early, small trial (only 3 participants) focused on safety, not effectiveness. The therapy may not work or could cause serious side effects.

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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

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

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