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Major study tracks DMD in boys to map disease course

NCT ID NCT03882827

Knowledge-focused Sponsor: Genethon 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study follows 220 boys aged 4 to 9 with Duchenne muscular dystrophy (DMD) for up to 3 years. Researchers collect data on muscle strength, walking ability, heart and lung function, and quality of life to better understand how the disease progresses. The goal is to gather natural history information, not to test a new treatment.

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 220 people

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

Started

Dec 2019

Expected to finish

Sep 2029

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

* Male * 4 to 9 years old inclusive * Body-weight ≤ 95th percentile or the BMI scale ≤ 95th percentile (according to validated scale in force in country site).

Ages

4 to 9 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: 1. Male 2. 4 to 9 years old inclusive 3. Body-weight ≤ 95th percentile or the BMI scale ≤ 95th percentile (according to validated scale in force in country site). Related to the DMD disease: 4. Diagnosis of DMD based upon documented gene testing with detailed genotyping 5. Able to achieve at inclusion and screening visits: 1. NSAA (North Star Ambulatory Assessment) scale \> 18 or ≥ 16 if participant is between 4 and \< 5 years old at screening and: 2. Gowers test \< or = 7 sec and/or 3. 6-Minute Walk Test (6MWT): a distance ≥ 350 meters at inclusion visit (M0) 6. Ongoing corticosteroid therapy or initiation of corticosteroid therapy according to standard of care prior to Screening visit Related to the study protocol and ICH/GCP (Good Clinical Practice) requirements: 7. Signed informed consent by at least one parent or both parents or legal guardian representative(s), when applicable and according to the country regulation 8. Affiliated to or a beneficiary of a Health Care scheme (according to country regulation) Exclusion Criteria: Subject will be excluded from enrolment into the study for any of the following reasons: Related to the DMD disease severity: 9. Cardiomyopathy based on physical/cardiological examination and echocardiography with Left Ventricular Simpson biplane Ejection Fraction (LVEF) below 55% 10. Respiratory Assistance: need for either a diurnal and/or a nocturnal ventilation 11. Any co-morbidity (ies) and or previous or planned surgical event(s) which may interfere with DMD natural evolution and or evaluation of outcomes designed to assess DMD Natural History Related to specific assessments: 12. Muscle testing: inability to cooperate with 13. MRI: metal implants in regions of interest for the study Related to the study protocol and ICH/GCP requirements: 14. Unwilling and/or unable to comply with all the study protocol requirements and/or procedures 15. Previous inclusion to another clinical trial with an Investigational Medicinal Product (IMP), within the 3 months or IMP washout period (whichever is longer) prior to the screening visit of the study 16. Previously treated with a gene therapy drug for DMD, such as: * any AAV mediated gene transfer products or any gene editing products in a clinical trial or in a clinical setting, * if exons skipping drug was used, the last dose of exon skipping drug within 5 half-lives prior to the screening visit 17. Concomitant participation to any other interventional clinical trial

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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

    14 sites in 4 countries. The list below names each one and where it is.

  2. 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 ↗

  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

  • Brest University Hospital Centre

    RECRUITING

    Brest, 29609, France

  • CHR Hôpital de la Citadelle

    NOT_YET_RECRUITING

    Liège, 4000, Belgium

  • CHU Lille

    RECRUITING

    Lille, 59000, France

  • Centre Hospitalier Universitaire - Hôpital Gui de Chauliac

    NOT_YET_RECRUITING

    Montpellier, 34295, France

  • Centre Hospitalier Universitaire Brugmann

    RECRUITING

    Brussels, 1020, Belgium

  • Great Ormond Street Hospital & University College London Hospital

    RECRUITING

    London, WC1N 1EH, United Kingdom

  • Hopital Femme Mere Enfant

    RECRUITING

    Bron, 69677, France

  • Hopital la Timone Enfants

    RECRUITING

    Marseille, 13385, France

  • Hospital La Fe de Valencia

    ACTIVE_NOT_RECRUITING

    Valencia, 46026, Spain

  • Hospital Sant Joan de Deu Esplugues de Llobregat

    RECRUITING

    Esplugues de Llobregat, 8950, Spain

  • Hôpital Armand Trousseau

    RECRUITING

    Paris, 75011, France

  • Hôpital Hautepierre

    RECRUITING

    Strasbourg, 67000, France

  • Institute of Genetic Medicine

    RECRUITING

    Newcastle, United Kingdom

  • UZ Leuven

    RECRUITING

    Leuven, 3000, Belgium

  • University Hospital of Bordeaux

    RECRUITING

    Bordeaux, 33076, France

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