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

Locations

Belgium

Centre Hospitalier Universitaire Brugmann1020, Brussels Belgium

UZ Leuven3000, Leuven Belgium

CHR Hôpital de la Citadelle4000, Liège Belgium

France

University Hospital of Bordeaux33076, Bordeaux France

Sponsors and collaborators

Genethon

Lead sponsor

Source ClinicalTrials.gov