Gene therapy for duchenne muscular dystrophy under Long-Term watch

NCT ID NCT05689164

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

First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This study follows 7 people with Duchenne muscular dystrophy who previously received an experimental gene therapy called fordadistrogene movaparvovec. Researchers will monitor them for 10 years to check for side effects and see if the treatment continues to help with movement. The goal is to better understand the therapy's long-term safety and effectiveness.

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 (gene therapy)
What this could lead to: If successful, this study could confirm the long-term safety and lasting benefits of this gene therapy for Duchenne muscular dystrophy.
What could go wrong: This is a small, terminated follow-up study with only 7 participants, so results may not apply widely. Gene therapy carries risks like immune reactions or unknown long-term side effects.

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

Duke Children's Health Center

Durham, North Carolina, 27710, United States
Duke Lenox Baker Children's

Durham, North Carolina, 27705, United States
Primary Children's Hospital

Salt Lake City, Utah, 84113, United States
Reed Neurological Research Center

Los Angeles, California, 90095, United States
UCLA Children's Heart Center

Los Angeles, California, 90095, United States
UCLA Clinical Lab Services

Los Angeles, California, 90095, United States
UCLA Kameron Gait and Motion Analysis Laboratory (Westwood Rehabilitation Center)

Los Angeles, California, 90095, United States
UCLA Medical Center

Los Angeles, California, 90095, United States
University of Utah Clinical Neurosciences Center

Salt Lake City, Utah, 84132, United States
University of Utah Craig H. Neilsen Rehabilitation Hospital

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

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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