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Researchers track Long-Term effects of duchenne gene therapy

NCT ID NCT05967351

Knowledge-focused 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 about 400 people with Duchenne muscular dystrophy who have already received the gene therapy SRP-9001 in an earlier study. The goal is to monitor their safety and muscle function over time. No new treatment is given in this study.

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

About 400 people

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

Started

Sep 2023

Expected to finish

Oct 2033

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

Children (under 18), adults (18 to 64) and older adults (65 and over)

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: * Received delandistrogene moxeparvovec for Duchenne muscular dystrophy in a previous clinical study. * Has (a) parent(s) or legal caregiver(s) or is ≥18 years of age and able to understand and comply with the study visit schedule and all other protocol requirements. Exclusion Criteria: * Not applicable

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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

    38 sites in 9 countries. 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

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

    Chicago, Illinois, 60611, United States

  • Arkansas Children's Hospital

    Little Rock, Arkansas, 72202, United States

  • Boston Children's Hospital

    Boston, Massachusetts, 02115, United States

  • Chiildren's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Health Specialty Center

    Dallas, Texas, 75207, United States

  • Children's Hospital Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Children's Hospital of Colorado

    Aurora, Colorado, 80045-7106, United States

  • Children's Hospital of The King's Daughters

    Norfolk, Virginia, 23510, United States

  • Columbia University Pediatric Neuromuscular Center

    New York, New York, 10032, United States

  • Great Ormond Street Hospital For Children NHS Foundation Trust

    London, Greater London, WC1N 1EJ, United Kingdom

  • Hong Kong Children's Hospital

    Hong Kong, Hong Kong

  • Hospital Sant Joan de Deu

    Esplugues de Llobregat, Barcelona, 08950, Spain

  • Hospital Universitario y Politecnico La fe. Neurology Department

    Valencia, 46026, Spain

  • Institute of Translational and Clinical Research

    Newcastle upon Tyne, NE1 3BZ, United Kingdom

  • Kaohsiung Medical University Chung-Ho Memorial Hospital

    Kaohsiung City, 807, Taiwan

  • Kobe University Hospital

    Kobe, Hyōgo, 650-0017, Japan

  • Lenox Baker Children's Hospital (Duke University)

    Durham, North Carolina, 27705, United States

  • Lucile Packard Children's Hospital Stanford (LPCH)

    Palo Alto, California, 94304, United States

  • National Center of Neurology and Psychiatry

    Kodaira, Tokyo, 187-8551, Japan

  • National Taiwan University Hospital

    Taipei, 100225, Taiwan

  • Nationwide Children's Hospital

    Columbus, Ohio, 43205, United States

  • Oregon Health and Science University

    Portland, Oregon, 97239, United States

  • Oxford University Hospitals NHS Foundation Trust

    Oxford, Oxfordshire, OX3 9DU, United Kingdom

  • Rady Children's Hospital

    San Diego, California, 92123, United States

  • The Johns Hopkins Hospital, Rubenstein Child Health Bldg

    Baltimore, Maryland, 21287, United States

  • Tokyo Women's Medical University Hospital

    Tokyo, 1628666, Japan

  • UCLA Medical Center

    Los Angeles, California, 90095, United States

  • UOC Neurologia Pediatrica e Malattie Muscolari, Istituto G. Gaslini, Istituto Pediatrico di Ricovero e Cura a Carattere Scientifico

    Genova, 16147, Italy

  • UOC Neurologia, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico

    Milan, 20122, Italy

  • UOC Neuropsichiatria Infantile, Area Salute del Bambino, Fondazione Policlinico Universitario A. Gemelli IRCCS, Universita Cattoclica del Sacro Cuore

    Roma, 00168, Italy

  • Universitair Ziekenhuis Gent, Neuromuscular Reference Centre (NMRC)

    Ghent, East Flanders, 9000, Belgium

  • University of California, Davis

    Sacramento, California, 95817, United States

  • University of Florida Clinical Research Center

    Gainesville, Florida, 32610, United States

  • University of Iowa ICTS Clinical Research Unit

    Iowa City, Iowa, 52242, United States

  • University of Rochester Medical Center

    Rochester, New York, 14642, United States

  • University of Utah Health

    Salt Lake City, Utah, 84132, United States

  • Universitätsklinikum Essen - Klinik für Kinderheilkunde I

    Essen, North Rhine-Westphalia, 45147, Germany

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

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