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
This trial does not accept healthy volunteersQualifiers
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 population
* 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).
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
No trial groups listed
Trial outcomes
Primary outcomes
1
NSAA scale
NSAA scale (age appropriate modified North Star Ambulatory Assessment)
Time frame
Screening 36 months2
10 Meter Walk/ Run test (10MW/RT)
Time function Test
Time frame
Screening 36 months3
Raise from floor (RFF)
Time function test
Time frame
Screening - 36 months4
6 Minutes Walk Test (6 MWT)
Motor Function Measurement
Time frame
Inclusion 36 months Source ClinicalTrials.gov