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.
Study duration from FPFV: Q1 2019 to LPLV: Q4 2026 Primary Ojectives: * To assess the natural disease course using standardized and disease appropriate evaluations in a cohort of young male subjects aged from 4 to 9 years at inclusion and diagnosed for Duchenne Muscular Dystrophy (DMD). * To record a baseline period prior to rolling over into a gene therapy phase I/II/III clinical study. Secondary Objectives: * To identify clinical, imaging and/or laboratory parameters that could be predictive indicators of the disease course in DMD, within the selected range of age. * To identify the best outcome measure(s) for further clinical trial assessments.
Study Type
OBSERVATIONAL
Enrollment
220
Centre Hospitalier Universitaire Brugmann
Brussels, Belgium
RECRUITINGUZ Leuven
Leuven, Belgium
NSAA scale
NSAA scale (age appropriate modified North Star Ambulatory Assessment)
Time frame: Screening 36 months
10 Meter Walk/ Run test (10MW/RT)
Time function Test
Time frame: Screening 36 months
Raise from floor (RFF)
Time function test
Time frame: Screening - 36 months
6 Minutes Walk Test (6 MWT)
Motor Function Measurement
Time frame: Inclusion 36 months
Myoset : Myo-grip, -pinch
Motor Function Measurement
Time frame: Inclusion 36 months
Stride velocity 95th centile (SV95c)
Motor Function Measurement with wearable device (SYDE)
Time frame: Inclusion 36 months
Muscle Imaging Nuclear Magnetic Resonance Imaging (NMRI)
Muscle Imaging
Time frame: Inclusion 36 months
Pulmonary Function Test (PFT)
Respiratory Function Assessment
Time frame: Inclusion 36 months
ECG - Echocardiography
Cardiac Function Assessment
Time frame: Inclusion 36 months
ACTIVLIM
Patient Reported Outcome
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CHR Hôpital de la Citadelle
Liège, Belgium
NOT_YET_RECRUITINGUniversity Hospital of Bordeaux
Bordeaux, France
RECRUITINGBrest University Hospital Centre
Brest, France
RECRUITINGHopital Femme Mere Enfant
Bron, France
RECRUITINGCHU Lille
Lille, France
RECRUITINGHopital la Timone Enfants
Marseille, France
RECRUITINGCentre Hospitalier Universitaire - Hôpital Gui de Chauliac
Montpellier, France
NOT_YET_RECRUITINGHôpital Armand Trousseau
Paris, France
RECRUITING...and 5 more locations
Time frame: Inclusion 36 months
EQ-5D
Questionnaire of Life
Time frame: Inclusion 36 months