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Gene therapy for duchenne muscular dystrophy under Long-Term watch

NCT ID NCT05689164

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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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

Study facts

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

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

7 people

The number who actually took part.

Started

Mar 2023

Finished

Sep 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

0 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: * Participants who received fordadistrogene movaparvovec in a previous Pfizer interventional study. Exclusion Criteria: * Investigator site staff directly involved in the study and their family members

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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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