RECRUITINGOBSERVATIONAL
Natural History of Duchenne Muscular Dystrophy
A Prospective, Interventional, Baseline Study In Young Male Subjects Aged From 4 to 9 Years
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.
Who May Be Eligible (Plain English)
Who May Qualify:
1. Male
2. 4 to 9 years old inclusive
3. Body-weight ≤ 95th percentile or the BMI scale ≤ 95th percentile (according to validated scale in force in country site).
Related to the DMD disease:
4. Diagnosis of DMD based upon documented gene testing with detailed genotyping
5. Able to achieve at inclusion and screening visits:
1. NSAA (North Star Ambulatory Assessment) scale \> 18 or ≥ 16 if participant is between 4 and \< 5 years old at screening and:
2. Gowers test \< or = 7 sec and/or
3. 6-Minute Walk Test (6MWT): a distance ≥ 350 meters at inclusion visit (M0)
6. 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:
7. Signed willing to sign a consent form by at least one parent or both parents or legal guardian representative(s), when applicable and according to the country regulation
8. Affiliated to or a beneficiary of a Health Care scheme (according to country regulation)
Who Should NOT Join This Trial:
Subject will be excluded from enrolment into the study for any of the following reasons:
Related to the DMD disease severity:
9. Cardiomyopathy based on physical/cardiological examination and echocardiography with Left Ventricular Simpson biplane Ejection Fraction (LVEF) below 55%
10. Respiratory Assistance: need for either a diurnal and/or a nocturnal ventilation
11. 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:
12. Muscle testing: inability to cooperate with
13. MRI: metal implants in regions of interest for the study
Related to the study protocol and ICH/GCP requirements:
14. Unwilling and/or unable to comply with all the study protocol requirements and/or procedures
...See full criteria on ClinicalTrials.gov
Always talk to your doctor about whether this trial is right for you.
Original Eligibility Criteria
View original clinical language
Inclusion Criteria:
1. Male
2. 4 to 9 years old inclusive
3. Body-weight ≤ 95th percentile or the BMI scale ≤ 95th percentile (according to validated scale in force in country site).
Related to the DMD disease:
4. Diagnosis of DMD based upon documented gene testing with detailed genotyping
5. Able to achieve at inclusion and screening visits:
1. NSAA (North Star Ambulatory Assessment) scale \> 18 or ≥ 16 if participant is between 4 and \< 5 years old at screening and:
2. Gowers test \< or = 7 sec and/or
3. 6-Minute Walk Test (6MWT): a distance ≥ 350 meters at inclusion visit (M0)
6. 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:
7. Signed informed consent by at least one parent or both parents or legal guardian representative(s), when applicable and according to the country regulation
8. Affiliated to or a beneficiary of a Health Care scheme (according to country regulation)
Exclusion Criteria:
Subject will be excluded from enrolment into the study for any of the following reasons:
Related to the DMD disease severity:
9. Cardiomyopathy based on physical/cardiological examination and echocardiography with Left Ventricular Simpson biplane Ejection Fraction (LVEF) below 55%
10. Respiratory Assistance: need for either a diurnal and/or a nocturnal ventilation
11. 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:
12. Muscle testing: inability to cooperate with
13. MRI: metal implants in regions of interest for the study
Related to the study protocol and ICH/GCP requirements:
14. Unwilling and/or unable to comply with all the study protocol requirements and/or procedures
15. 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
16. 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
17. Concomitant participation to any other interventional clinical trial
Locations (15)
Centre Hospitalier Universitaire Brugmann
Brussels, Belgium
UZ Leuven
Leuven, Belgium
CHR Hôpital de la Citadelle
Liège, Belgium
University Hospital of Bordeaux
Bordeaux, France
Brest University Hospital Centre
Brest, France
Hopital Femme Mere Enfant
Bron, France
CHU Lille
Lille, France
Hopital la Timone Enfants
Marseille, France
Centre Hospitalier Universitaire - Hôpital Gui de Chauliac
Montpellier, France
Hôpital Armand Trousseau
Paris, France
Hôpital Hautepierre
Strasbourg, France
Hospital Sant Joan de Deu Esplugues de Llobregat
Esplugues de Llobregat, Spain
Hospital La Fe de Valencia
Valencia, Spain
Great Ormond Street Hospital & University College London Hospital
London, United Kingdom
Institute of Genetic Medicine
Newcastle, United Kingdom