Natural History of Duchenne Muscular Dystrophy

Prior treatment

• 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:
• MRI: metal implants in regions of interest for the study
  • Related to the study protocol and ICH/GCP requirements:
• 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

Other illness

• Related to the DMD disease severity:

Lab values

• Cardiomyopathy based on physical/cardiological examination and echocardiography with Left Ventricular Simpson biplane Ejection Fraction (LVEF) below 55%

Other

• Subject will be excluded from enrolment into the study for any of the following reasons:
• Respiratory Assistance: need for either a diurnal and/or a nocturnal ventilation
• Muscle testing: inability to cooperate with
• Unwilling and/or unable to comply with all the study protocol requirements and/or procedures

• 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)