Gene therapy trial for duchenne MD halted early – what we know
NCT ID NCT03362502
First seen Jun 26, 2026 · Last updated Jun 26, 2026
Summary
This early-stage trial tested a single infusion of gene therapy (PF-06939926) in 23 people with Duchenne muscular dystrophy, both those who could still walk and those who could not. The main goal was to check safety and tolerability, while also measuring dystrophin protein levels and muscle function. The study was terminated early, but the data may still help researchers develop better treatments.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
A single intravenous infusion of a gene therapy (PF-06939926) that delivers a shortened version of the dystrophin gene via a harmless virus.
What this could lead to
If successful, this could point toward a one-time treatment that helps slow or stabilize muscle decline in Duchenne muscular dystrophy.
What could go wrong
This was a very early (Phase 1) safety study with only 23 participants, and it was terminated early. Gene therapies can cause serious immune reactions, and the long-term effects are unknown.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Biospecimen Repository & Processing Core - BPRC
Durham, North Carolina, 27710, United States
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CCTS Clinical Research Center
Salt Lake City, Utah, 84108, United States
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Duke Cardiovascular Magnetic Resonance Center
Durham, North Carolina, 27710, United States
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Duke Children's Hospital & Health Center
Durham, North Carolina, 27710, United States
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Duke Neurology
Durham, North Carolina, 27705, United States
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Duke University Hospital Investigational Drug Services (IDS) Pharmacy
Durham, North Carolina, 27710, United States
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Duke University Medical Center, Lenox Baker Children's Hospital
Durham, North Carolina, 27705, United States
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MRI Research Center
Los Angeles, California, 90095, United States
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Primary Children's Hospital
Salt Lake City, Utah, 84113, United States
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Reed Neurological Research Center
Los Angeles, California, 90095, United States
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Ronald Reagan UCLA Medical Center (Investigational Drug Section)
Los Angeles, California, 90095, United States
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Ronald Reagan UCLA Medical Center - Interventional Radiology
Los Angeles, California, 90095, United States
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Ronald Reagan UCLA Medical Center Drug Information Center
Los Angeles, California, 90095, United States
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UCLA (David Geffen School of Medicine)
Los Angeles, California, 90095, United States
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UCLA Children's Heart Center
Los Angeles, California, 90095, United States
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UCLA Mattel Children's Hospital
Los Angeles, California, 90095, United States
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UCLA Medical Center
Los Angeles, California, 90095, United States
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UCLA Outpatient Surgery Center
Los Angeles, California, 90095, United States
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University of Utah Clinical Neurosciences Center
Salt Lake City, Utah, 84132, United States
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University of Utah Hospital
Salt Lake City, Utah, 84112, United States
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University of Utah Hospital & Clinics Investigational Drug Services
Salt Lake City, Utah, 84112, United States
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University of Utah Imaging and Neurosciences Center
Salt Lake City, Utah, 84108, United States