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Experimental gene therapy targets duchenne MD in young boys

NCT ID NCT05429372

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 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.

Study facts

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

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

10 people

The number who actually took part.

Started

Aug 2022

Finished

Oct 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

2 to 3 years

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: * Confirmed diagnosis of DMD by prior genetic testing. Exclusion Criteria: * Any of the following genetic abnormalities in the dystrophin gene: a. Any mutation (exon deletion, exon duplication, insertion, or point mutation) affecting any exon between exon 9 and exon 13, inclusive; OR b. A deletion that affects both exon 29 and exon 30; OR c. A deletion that affects any exons between 56-71, inclusive. * Positive test performed by Pfizer for neutralizing antibodies to AAV9. * Any prior treatment with gene therapy. * Any treatment designed to increase dystrophin expression within 6 months prior to screening (including, but not limited to, exon-skipping and nonsense read through). * Previous or current treatment with oral glucocorticoids or other immunosuppressive agents for the indication of DMD. * Abnormality in specified laboratory tests, including blood counts, liver and kidney function.

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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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