Natural History of Duchenne Muscular Dystrophy

 Becker Muscular Dystrophy / Posted 2 years ago

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 5 to 9 Years over a period of 6 to 36 months using disease appropriate evaluations.

  • Inclusion Criteria : Ages Eligible for Study: 5 Years to 9 Years (Child) Sexes Eligible for Study: Male Accepts Healthy Volunteers: No 1. Male 1. 5 to 9 years old inclusive 3.Body-Weight < or = 75th percentile of BMI body-mass index scale (according to validated scale in force in the country site) 4. Diagnosis of DMD based upon Gene testing positive with detailed genotyping 5. Able to achieve:
    • NSAA (North Star Ambulatory Assessment) scale > or =18 (with a maximum of 2 points 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 at inclusion visit (M0) with the distance being 20% of each other 6. Ongoing corticosteroid therapy or initiation of corticosteroid therapy according to standard of care in the previous 3 months 7. Signed informed consent by at least one parent(s) or both parents or legal guardian representative(s), when applicable and according to the country regulation 8. Affiliated Beneficiary of the National Health Care scheme
  • Exclusion Criteria : 9. Cardiomyopathy based on physical cardiological examination and echocardiography with Left 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 may interfere with DMD natural evolution and or evaluation of outcomes designed to assess DMD Natural History 12. Muscle testing: inability to cooperate with 13. Nuclear Magnetic Resonance Imaging (NMRI): metal implants in regions of interest for the study 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) prior to the screening visit of the study 16.Concomitant participation to any other clinical trial
  • Study end date : 30/06/2023
  • Principal Investigator : Francesco MUNTONI
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