Actively Recruiting
Natural History of Duchenne Muscular Dystrophy
Led by Genethon · Updated on 2026-02-12
220
Participants Needed
15
Research Sites
510 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Natural History of Duchenne Muscular Dystrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Male
- Aged 4 to 9 years inclusive
- Body weight or BMI at or below the 95th percentile based on local validated scales
- Confirmed diagnosis of Duchenne Muscular Dystrophy by gene testing with detailed genotyping
- Able to achieve at screening and inclusion visits: NSAA scale >18 or 616 if between 4 and <5 years old, Gowers test 67 seconds, and/or 6-Minute Walk Test distance 350 meters at inclusion
- Ongoing or planned corticosteroid therapy according to standard care before screening
- Signed informed consent by parent(s) or legal guardian
- Affiliated with or beneficiary of a health care scheme as per local regulations
You will not qualify if you...
- Cardiomyopathy with Left Ventricular Ejection Fraction below 55%
- Need for daytime or nighttime respiratory ventilation support
- Any co-morbidities or surgeries that may affect DMD progression or study evaluations
- Inability to cooperate with muscle testing
- Presence of metal implants in areas of interest for MRI
- Unwillingness or inability to comply with study procedures
- Participation in another investigational medicinal product trial within 3 months or IMP washout
- Previous gene therapy treatment for DMD, including AAV-mediated gene transfer or gene editing
- Recent exon skipping drug use within 5 half-lives prior to screening
- Concurrent participation in any other interventional clinical trial
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 15 locations
1
Centre Hospitalier Universitaire Brugmann
Brussels, Belgium, 1020
Actively Recruiting
2
UZ Leuven
Leuven, Belgium, 3000
Actively Recruiting
3
CHR Hôpital de la Citadelle
Liège, Belgium, 4000
Not Yet Recruiting
4
University Hospital of Bordeaux
Bordeaux, France, 33076
Actively Recruiting
5
Brest University Hospital Centre
Brest, France, 29609
Actively Recruiting
6
Hopital Femme Mere Enfant
Bron, France, 69677
Actively Recruiting
7
CHU Lille
Lille, France, 59000
Actively Recruiting
8
Hopital la Timone Enfants
Marseille, France, 13385
Actively Recruiting
9
Centre Hospitalier Universitaire - Hôpital Gui de Chauliac
Montpellier, France, 34295
Not Yet Recruiting
10
Hôpital Armand Trousseau
Paris, France, 75011
Actively Recruiting
11
Hôpital Hautepierre
Strasbourg, France, 67000
Actively Recruiting
12
Hospital Sant Joan de Deu Esplugues de Llobregat
Esplugues de Llobregat, Spain, 8950
Actively Recruiting
13
Hospital La Fe de Valencia
Valencia, Spain, 46026
Active, Not Recruiting
14
Great Ormond Street Hospital & University College London Hospital
London, United Kingdom, WC1N 1EH
Actively Recruiting
15
Institute of Genetic Medicine
Newcastle, United Kingdom
Actively Recruiting
Research Team
F
Francesco MUNTONI, Pr
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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