Experimental gene therapy targets duchenne MD in young boys

NCT ID NCT05429372

Terminated Disease control Sponsor: Pfizer Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This Phase 2 trial tested a single dose of gene therapy (fordadistrogene movaparvovec) in 10 boys with early-stage Duchenne muscular dystrophy. The goal was to check safety and whether the therapy could help muscles produce a mini-dystrophin protein. The study was terminated early, so full results are not available.

What this could mean

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

Active substance: Fordadistrogene movaparvovec (a gene therapy)
What this could lead to: If successful, this gene therapy could help boys with Duchenne muscular dystrophy produce a shortened form of dystrophin, potentially slowing muscle damage.
What could go wrong: This early-phase trial was terminated, so results are limited. Gene therapies can cause immune reactions or other serious side effects, and long-term benefits are unproven.

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.

MUSCULAR DYSTROPHY, DUCHENNE

Contacts and locations

Locations

CTSI Clinical Research Center

Salt Lake City, Utah, 84108, United States
Perth Children's Hospital

Nedlands, Western Australia, 6009, Australia
Primary Children's Hospital

Salt Lake City, Utah, 84113, United States
The Children's Hospital at Westmead

Westmead, New South Wales, 2145, Australia
The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States
The Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19146, United States
The Royal Children's Hospital Melbourne

Parkville, Victoria, 3052, Australia
UF Health Shands Hospital

Gainesville, Florida, 32610, United States
University of Florida

Gainesville, Florida, 32610, United States
University of Utah Clinical Neurosciences Center

Salt Lake City, Utah, 84132, United States
University of Utah Hospital

Salt Lake City, Utah, 84132, United States
University of Utah Hospital & Clinics Investigational Drug Services

Salt Lake City, Utah, 84112, United States
University of Utah Imaging and Neurosciences Center

Salt Lake City, Utah, 84108, United States

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