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Gene therapy trial for duchenne MD halted early – what we know

NCT ID NCT03362502

Disease control Sponsor: Pfizer Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

23 people

The number who actually took part.

Started

Jan 2018

Finished

Jul 2025

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

4 years and older

Sex

Male participants only

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Age as follows, based on ambulatory status: * FOR AMBULATORY PARTICIPANTS, defined as the ability to walk at least 10 meters unassisted: Between 4 and 12 years, inclusive, * FOR NON-AMBULATORY PARTICIPANTS, defined as the inability to walk at least 10 meters unassisted: No age restrictions so long as loss of ambulation occurs prior to the subject's 17th birthday; * Diagnosis of Duchenne muscular dystrophy confirmed by medical history and genetic testing; * Receipt of glucocorticoids for 6 months and a stable daily dose for at least 3 months prior to study entry; * Ability to tolerate magnetic resonance imaging (MRI) without sedation and with no contraindications to these procedures; * Ability to tolerate muscle biopsies under anesthesia with no contraindications to these procedures; * Body weights as follows, based on ambulatory status: * FOR AMBULATORY PARTICIPANTS: Between 15 kg and 50 kg, * FOR NON-AMBULATORY PARTICIPANTS: Less than 75 kg, but which may be managed or adjusted to a lower limit, especially to ensure participant safety; * Functional performance as follows, based on ambulatory status: * FOR AMBULATORY PARTICIPANTS: Ability to rise from floor within seven (7) seconds, * FOR NON-AMBULATORY PARTICIPANTS: Percent predicted forced vital capacity greater than 40% as part of pulmonary function tests, as well as adequate upper limb function. Exclusion Criteria: * Receipt of live attenuated vaccination within 3 months prior to receiving PF-06939926 or exposure to an influenza (or other inactivated) vaccination or systemic antiviral and/or interferon therapy within 30 days prior to receipt of PF-06939926; * Prior exposure to any gene therapy agent, including exon-skipping agents; * Exposure to other investigational drugs within 30 days or 5 half-lives, whichever is longer; * Neutralizing antibodies (NAb) against adeno-associated virus, serotype 9 (AAV9); * Compromised cardiac function as indicated by left ventricular ejection fraction on cardiac MRI, as follows, based on ambulatory status: * FOR AMBULATORY PARTICIPANTS: Less than 55%, * FOR NON-AMBULATORY PARTICIPANTS: Less than 35%; * Inadequate hepatic or renal function or risk factors for autoimmune disease on screening laboratory assessments. * The following genetic abnormalities in the dystrophin gene as confirmed by the investigator based on the review of the DMD genetic testing: 1. Any mutation (exon deletion, exon duplication, insertion, or point mutation) affecting any exon between exon 9 and exon 13, inclusive; OR 2. A deletion that affects both exon 29 and exon 30. Sirolimus Cohort Inclusion Criteria * \> 8 years of age Exclusion Criteria * Hypersensitivity to sirolimus or intolerance to soy, including a history of angioedema * Concomitant use with strong CYP3A4/P-gp inducers or inhibitors

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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

  • Biospecimen Repository & Processing Core - BPRC

    Durham, North Carolina, 27710, United States

  • CCTS Clinical Research Center

    Salt Lake City, Utah, 84108, United States

  • Duke Cardiovascular Magnetic Resonance Center

    Durham, North Carolina, 27710, United States

  • Duke Children's Hospital & Health Center

    Durham, North Carolina, 27710, United States

  • Duke Neurology

    Durham, North Carolina, 27705, United States

  • Duke University Hospital Investigational Drug Services (IDS) Pharmacy

    Durham, North Carolina, 27710, United States

  • Duke University Medical Center, Lenox Baker Children's Hospital

    Durham, North Carolina, 27705, United States

  • MRI Research Center

    Los Angeles, California, 90095, United States

  • Primary Children's Hospital

    Salt Lake City, Utah, 84113, United States

  • Reed Neurological Research Center

    Los Angeles, California, 90095, United States

  • Ronald Reagan UCLA Medical Center (Investigational Drug Section)

    Los Angeles, California, 90095, United States

  • Ronald Reagan UCLA Medical Center - Interventional Radiology

    Los Angeles, California, 90095, United States

  • Ronald Reagan UCLA Medical Center Drug Information Center

    Los Angeles, California, 90095, United States

  • UCLA (David Geffen School of Medicine)

    Los Angeles, California, 90095, United States

  • UCLA Children's Heart Center

    Los Angeles, California, 90095, United States

  • UCLA Mattel Children's Hospital

    Los Angeles, California, 90095, United States

  • UCLA Medical Center

    Los Angeles, California, 90095, United States

  • UCLA Outpatient Surgery Center

    Los Angeles, California, 90095, United States

  • University of Utah Clinical Neurosciences Center

    Salt Lake City, Utah, 84132, United States

  • University of Utah Hospital

    Salt Lake City, Utah, 84112, 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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Other studies related to the condition(s) this trial covers.