Phase
Condition
Muscular Dystrophy
Treatment
N/AClinical Study ID
Ages 5-9 Male
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
- Male
- 5 to 9 years old inclusive
- Body-Weight < or = 75th percentile of BMI body-mass index scale (according tovalidated scale in force in the country site)
- Diagnosis of DMD based upon Gene testing positive with detailed genotyping
- Able to achieve:
- NSAA (North Star Ambulatory Assessment) scale > or =18 (with a maximum of 2points difference between inclusion and screening visits) and/or:
- Gowers test < or =7 sec
- 6 Minute Walk Test (6MWT) > or = 350 meters at screening visit (M1) and atinclusion visit (M0) with the distance being 20% of each other
- Ongoing corticosteroid therapy or initiation of corticosteroid therapy according tostandard of care in the previous 3 months
- Signed informed consent by at least one parent(s) or both parents or legal guardianrepresentative(s), when applicable and according to the country regulation
- Affiliated Beneficiary of the National Health Care scheme
Exclusion
Exclusion Criteria: 9. Cardiomyopathy based on physical cardiological examination and echocardiography withLeft Ventricular 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 mayinterfere with DMD natural evolution and or evaluation of outcomes designed to assessDMD Natural History 12. Muscle testing: inability to cooperate with 13. Nuclear Magnetic Resonance Imaging (NMRI): metal implants in regions of interest forthe study 14. Unwilling and/or unable to comply with all the study protocol requirements and orprocedures 15. Previous inclusion to another clinical trial with an Investigational Medicinal Product (IMP), within the 3 months (or IMP washout period) prior to the screening visit of thestudy 16. Concomitant participation to any other clinical trial
Study Design
Study Description
Connect with a study center
University Hospital of Bordeaux
Bordeaux, 33076
FranceActive - Recruiting
Brest University Hospital Centre
Brest, 29609
FranceActive - Recruiting
Hopital Femme Mere Enfant
Bron, 69677
FranceActive - Recruiting
CHU Lille
Lille, 59000
FranceActive - Recruiting
Hopital la Timone Enfants
Marseille, 13385
FranceActive - Recruiting
Hôpital Armand Trousseau
Paris, 75011
FranceActive - Recruiting
Hôpital Hautepierre
Strasbourg, 67000
FranceActive - Recruiting
Great Ormond Street Hospital & University College London Hospital
London, WC1N 1EH
United KingdomActive - Recruiting
Institute of Genetic Medicine
Newcastle,
United KingdomActive - Recruiting
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