Natural History of Duchenne Muscular Dystrophy
ConditionDuchenne Muscular Dystrophy
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexMale
Age4-9
SponsorGenethon
About this trial
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.
Eligibility criteria
Qualifiers
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).
Diagnosis of DMD based upon documented gene testing with detailed genotyping
Disqualifiers
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
Muscle testing: inability to cooperate with
Trial design
Treatments tested in this trial
- Not listed
Trial groups
No trial groups listed
Sponsors and collaborators
Source ClinicalTrials.gov