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New gene therapy trial hopes to restore muscle protein in duchenne boys

NCT ID NCT04626674

Disease control Sponsor: Sarepta Therapeutics, Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
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 Results may not be published yet.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
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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Sep 11, 2026 · Updated 2 times

Summary

This study tests a one-time gene therapy called delandistrogene moxeparvovec in 83 people with Duchenne muscular dystrophy. The goal is to see if it is safe and helps the body make dystrophin, a protein missing in Duchenne. The trial is now enrolling non-ambulatory participants (Cohort 8) and will follow them for up to 156 weeks.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
delandistrogene moxeparvovec (gene therapy)
What this could lead to
If successful, this could point toward a treatment that helps boys with Duchenne produce a key muscle protein, potentially slowing disease progression.
What could go wrong
This is an early Phase 1 study, so safety and effectiveness are not yet proven. Risks include liver injury and immune reactions, and long-term benefits are uncertain.

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

About 83 people

The number the study aims to enrol. It can still change while the study runs.

Started

Nov 2020

Expected to finish

Feb 2028

An estimate. End dates often move.

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 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: * For Cohorts 1-8: Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing. * Cohort 8: Non-ambulatory per protocol-specified criteria at the time of Screening, has a performance upper limb (PUL) entry item score ≥2 at the Screening visit and has a total PUL score of ≥12 and ≤40 at the time of Screening. * Cohorts 1, 2, 3, 5, 7 and 8 only: Stable dose equivalent of oral glucocorticoids for at least 12 weeks before screening and the dose is expected to remain constant (except for modifications to accommodate changes in weight) throughout the first year of the study. * Cohort 1: Is ambulatory, and ≥4 to \<8 years of age at the time of Screening. * Cohort 2: Is ambulatory, and ≥8 to \<18 years of age at the time of Screening. * Cohort 3: Non-ambulatory per protocol specified criteria at the time of Screening. * Cohort 4: Is ambulatory and ≥3 to \<4 years of age at the time of Screening. * Cohort 5a: Is ambulatory and ≥4 to \<9 years of age with time to rise from the floor ≤7 seconds at the screening visit. * Cohort 5b: Non-ambulatory per protocol specified criteria at the time of Screening. * Cohort 6: Is ambulatory, and ≥2 to \<3 years of age at the time of Screening. * Cohort 7: Non-ambulatory per protocol-specified criteria at the time of Screening. * Cohorts 4 and 6: Do not yet require use of chronic steroids for treatment of their DMD, in the opinion of the Investigator, and are not receiving steroids at the time of Screening. * Genetic mutation inclusion criteria vary by cohort. All Cohorts: * Ability to cooperate with motor assessment testing. * rAAVrh74 antibody titers are not elevated as per protocol-specified requirements. Exclusion Criteria: * Cohort 8: Any confounding factors that would prevent the use of oral sirolimus including a known hypersensitivity to sirolimus or any of its excipients. * Has a concomitant illness, autoimmune disease, chronic drug treatment, and/or cognitive delay/impairment that in the opinion of the Investigator creates unnecessary risks for gene transfer. * Exposure to gene therapy, investigational medication, or any treatment designed to increase dystrophin expression within protocol-specified time limits. * Abnormality in protocol-specified diagnostic evaluations or laboratory tests. Note: Other inclusion/exclusion criteria apply.

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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 Muscular dystrophy, Duchenne

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The places running it

    11 sites. The list below names each one and where it is.

  3. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  4. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Ann & Robert H. Lurie Children's Hospital of Chicago

    RECRUITING

    Chicago, Illinois, 60611, United States

  • Arkansas Children's Hospital

    RECRUITING

    Little Rock, Arkansas, 72202, United States

  • Children's Hospital of The King's Daughters

    RECRUITING

    Norfolk, Virginia, 23507, United States

  • Duke University Medical Center

    RECRUITING

    Durham, North Carolina, 27705, United States

  • Nationwide Children's Hospital

    ACTIVE_NOT_RECRUITING

    Columbus, Ohio, 43205, United States

  • Neurology Rare Disease Center

    RECRUITING

    Flower Mound, Texas, 75028, United States

    Contact Email: •••••@•••••

  • Stanford University

    RECRUITING

    Palo Alto, California, 94304, United States

    Contact Email: •••••@•••••

  • University of California, Davis

    RECRUITING

    Sacramento, California, 95616, United States

  • University of California, Los Angeles

    RECRUITING

    Los Angeles, California, 90095, United States

  • University of California, San Diego

    RECRUITING

    La Jolla, California, 92037, United States

  • University of Florida

    RECRUITING

    Gainesville, Florida, 32610, United States

  • Washington University in St. Louis

    RECRUITING

    St Louis, Missouri, 63110, United States

    Contact Email: •••••@•••••

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