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Gene therapy vs. standard care: new study tracks Long-Term outcomes in duchenne patients

NCT ID NCT06270719

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 trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study follows 500 people with Duchenne muscular dystrophy to see how well a gene therapy (ELEVIDYS) works compared to standard steroid treatment over time. Researchers will measure movement abilities, breathing, and safety, including liver problems. Participants must already be on or starting steroids, and those getting gene therapy must be at least 4 years old.

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.

Participants

About 500 people

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

Started

Feb 2024

Expected to finish

Dec 2038

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.

Who is studied

DMD participants within each prescriber's practice who either (1) have been prescribed delandistrogene moxeparvovec or (2) are receiving or have been prescribed to begin chronic glucocorticoid therapy in routine clinical practice, and who meet the study eligibility criteria, will be invited to enroll into the study and will be followed according to the protocol.

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: * Has an established clinical diagnosis of DMD based on documentation of clinical findings and prior confirmatory genetic testing using a clinical diagnostic genetic test. * Is currently receiving or has been prescribed to start chronic glucocorticoid therapy at the time of this observational study enrollment. For ELEVIDYS-treated Participants (Cohorts 1a, 1b, and 1c): * Is at least 4 years of age at the time of infusion * Will either: a) be initiating or has initiated ELEVIDYS within the last 30 days in routine clinical practice at the time of this observational study enrollment, or b) was administered ELEVIDYS in routine clinical practice and has the required minimum dataset for entry into the observational study per Sponsor approval For Standard of Care Comparators (Cohort 2): * Is at least 4 years of age at the time of enrollment * Is unexposed to DMD gene therapy at the time of this observational study enrollment Exclusion Criteria: * Has any deletion of exon 8 and/or exon 9 in the DMD gene. * Is currently participating in any DMD interventional study at the time of this observational study enrollment. * Has any prior exposure to DMD gene therapy other than that described for Cohort 1c (ELEVIDYS Retrospectively Treated Cohort). * Has a medical condition or confounding circumstances (for example, prior traumatic limitation for mobility or significant behavioral comorbidity) that, in the opinion of the Investigator, might compromise: * The participant's ability to comply with the protocol-required procedures, * The participant's wellbeing or safety, and/or * The clinical interpretability of the data collected from the participant. Other inclusion/exclusion criteria may 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

How to take part

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

  1. The places running it

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

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. 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

  • All Children's Research Institute, Inc

    St. Petersburg, Florida, 33701, United States

  • Ann and Robert H Lurie Childrens Hospital of Chicago

    Chicago, Illinois, 60611-2991, United States

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202, United States

  • Children's Hospital Los Angeles - PIN

    Los Angeles, California, 90027, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Hospital of the King's Daughters

    Norfolk, Virginia, 23507, United States

  • Children's National Medical Center

    Washington D.C., District of Columbia, 20010, United States

  • Childrens Hospital Medical Center of Akron

    Akron, Ohio, 44302, United States

  • Connecticut Children's Medical Center - Hartford

    Hartford, Connecticut, 06106, United States

  • Cook Children's Hospital

    Fort Worth, Texas, 76104, United States

  • Duke Lenox Baker Children's Hospital

    Durham, North Carolina, 27704, United States

  • Indiana Clinical and Translational Science Institute

    Indianapolis, Indiana, 46202, United States

  • Le Bonheur Children's Hospital

    Memphis, Tennessee, 38103, United States

  • Nemours Children's Hospital - Orlando

    Orlando, Florida, 32827, United States

  • Nicklaus Children's Hospital

    Miami, Florida, 33155, United States

  • OHSU Healthcare (Oregon Health and Science University)

    Portland, Oregon, 97239, United States

  • Penn State Health Milton S. Hershey Medical Center

    Hershey, Pennsylvania, 17033, United States

  • Seattle Children's Hospital

    Seattle, Washington, 98105, United States

  • The Board of Regents of the University of Wisconsin

    Madison, Wisconsin, 53715-1218, United States

  • The Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226-4874, United States

  • University of Colorado - PPDS

    Aurora, Colorado, 80045, United States

  • University of Kansas Medical Center

    Kansas City, Kansas, 66160, United States

  • University of Michigan

    Ann Arbor, Michigan, 48109, United States

  • University of Virginia

    Charlottesville, Virginia, 22903, United States

  • Vanderbilt University Medical Center

    Nashville, Tennessee, 37232, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.