Natural History of Duchenne Muscular Dystrophy
Recruiting now
Conditions studied: Duchenne Muscular Dystrophy
In brief
Baseline Study on Duchenne Muscular Dystrophy (DMD) in view to collect data on the natural disease course in a cohort in young male subjects aged from 4 to 9 Years over a period of 6 to 36 months using disease appropriate evaluations.
Key facts
- Study ID
- NCT03882827
- Run by
- Genethon
- People needed
- 220
- Starts
- 2019-12-19
- Expected to finish
- 2029-09-30
- Last updated by the study team
- 2026-02-12
Who can join
Age: 4 and older, up to 9. Sex: male. Healthy volunteers: not accepted.
You may qualify if…
- 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).
- Related to the DMD disease:
- Diagnosis of DMD based upon documented gene testing with detailed genotyping
- Able to achieve at inclusion and screening visits:
- NSAA (North Star Ambulatory Assessment) scale > 18 or ≥ 16 if participant is between 4 and < 5 years old at screening and:
- Gowers test < or = 7 sec and/or
- 6-Minute Walk Test (6MWT): a distance ≥ 350 meters at inclusion visit (M0)
- 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:
- Signed informed consent by at least one parent or both parents or legal guardian representative(s), when applicable and according to the country regulation
- Affiliated to or a beneficiary of a Health Care scheme (according to country regulation)
You may not qualify if…
- Subject will be excluded from enrolment into the study for any of the following reasons:
- Related to the DMD disease severity:
- Cardiomyopathy based on physical/cardiological examination and echocardiography with Left Ventricular Simpson biplane Ejection Fraction (LVEF) below 55%
- Respiratory Assistance: need for either a diurnal and/or a nocturnal ventilation
- 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:
- Muscle testing: inability to cooperate with
- MRI: metal implants in regions of interest for the study
- Related to the study protocol and ICH/GCP requirements:
- Unwilling and/or unable to comply with all the study protocol requirements and/or procedures
- 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
- 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
- Concomitant participation to any other interventional clinical trial
Where it is running
- Institute of Genetic Medicine — Newcastle, United Kingdom (enrolling)
- UZ Leuven — Leuven, Belgium (enrolling)
- Hospital Sant Joan de Deu Esplugues de Llobregat — Esplugues de Llobregat, Spain (enrolling)
- Great Ormond Street Hospital & University College London Hospital — London, United Kingdom (enrolling)
- Centre Hospitalier Universitaire Brugmann — Brussels, Belgium (enrolling)
- University Hospital of Bordeaux — Bordeaux, France (enrolling)
- Brest University Hospital Centre — Brest, France (enrolling)
- Hopital Femme Mere Enfant — Bron, France (enrolling)
- CHU Lille — Lille, France (enrolling)
- Hopital la Timone Enfants — Marseille, France (enrolling)
- Hôpital Armand Trousseau — Paris, France (enrolling)
- Hôpital Hautepierre — Strasbourg, France (enrolling)
- CHR Hôpital de la Citadelle — Liège, Belgium
- Hospital La Fe de Valencia — Valencia, Spain
- Centre Hospitalier Universitaire - Hôpital Gui de Chauliac — Montpellier, France
Full record on ClinicalTrials.gov
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