Phase
Condition
Muscular Dystrophy
Treatment
N/AClinical Study ID
Ages 4-9 Male
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Male
4 to 9 years old inclusive
Body-weight ≤ 95th percentile or the BMI scale ≤ 95th percentile (according tovalidated scale in force in country site). Related to the DMD disease:
Diagnosis of DMD based upon documented gene testing with detailed genotyping
Able to achieve at inclusion and screening visits:
NSAA (North Star Ambulatory Assessment) scale > 18 or ≥ 16 if participant isbetween 4 and < 5 years old at screening and:
Gowers test < or = 7 sec and/or
6-Minute Walk Test (6MWT): a distance ≥ 350 meters at inclusion visit (M0)
Ongoing corticosteroid therapy or initiation of corticosteroid therapy according tostandard of care prior to Screening visit Related to the study protocol and ICH/GCP (Good Clinical Practice) requirements:
Signed informed consent by at least one parent or both parents or legal guardianrepresentative(s), when applicable and according to the country regulation
Affiliated to or a beneficiary of a Health Care scheme (according to countryregulation)
Exclusion
Exclusion Criteria: Subject will be excluded from enrolment into the study for any of the followingreasons: Related to the DMD disease severity:
Cardiomyopathy based on physical/cardiological examination and echocardiography withLeft 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 mayinterfere with DMD natural evolution and or evaluation of outcomes designed toassess DMD Natural History Related to specific assessments:
Muscle testing: inability to cooperate with
MRI: metal implants in regions of interest for the study Related to the study protocol and ICH/GCP requirements:
Unwilling and/or unable to comply with all the study protocol requirements and/orprocedures
Previous inclusion to another clinical trial with an Investigational MedicinalProduct (IMP), within the 3 months or IMP washout period (whichever is longer) priorto the screening visit of the study
Previously treated with a gene therapy drug for DMD, such as:
any AAV mediated gene transfer products or any gene editing products in aclinical trial or in a clinical setting,
if exons skipping drug was used, the last dose of exon skipping drug within 5half-lives prior to the screening visit
- Concomitant participation to any other interventional clinical trial
Study Design
Study Description
Connect with a study center
Centre Hospitalier Universitaire Brugmann
Brussels 2800866, 1020
BelgiumActive - Recruiting
UZ Leuven
Leuven 2792482, 3000
BelgiumActive - Recruiting
CHR Hôpital de la Citadelle
Liège 2792413, 4000
BelgiumSite Not Available
University Hospital of Bordeaux
Bordeaux, 33076
FranceSite Not Available
University Hospital of Bordeaux
Bordeaux 3031582, 33076
FranceActive - Recruiting
Brest University Hospital Centre
Brest, 29609
FranceSite Not Available
Brest University Hospital Centre
Brest 3030300, 29609
FranceActive - Recruiting
Hopital Femme Mere Enfant
Bron, 69677
FranceSite Not Available
Hopital Femme Mere Enfant
Bron 3029931, 69677
FranceActive - Recruiting
CHU Lille
Lille, 59000
FranceSite Not Available
CHU Lille
Lille 2998324, 59000
FranceActive - Recruiting
Hopital la Timone Enfants
Marseille, 13385
FranceSite Not Available
Hopital la Timone Enfants
Marseille 2995469, 13385
FranceActive - Recruiting
Centre Hospitalier Universitaire - Hôpital Gui de Chauliac
Montpellier 2992166, 34295
FranceSite Not Available
Hôpital Armand Trousseau
Paris, 75011
FranceSite Not Available
Hôpital Armand Trousseau
Paris 2988507, 75011
FranceActive - Recruiting
Hôpital Hautepierre
Strasbourg, 67000
FranceSite Not Available
Hôpital Hautepierre
Strasbourg 2973783, 67000
FranceActive - Recruiting
Hospital Sant Joan de Deu Esplugues de Llobregat
Esplugues de Llobregat 3122826, 8950
SpainActive - Recruiting
Hospital La Fe de Valencia
Valencia 2509954, 46026
SpainSite Not Available
Great Ormond Street Hospital
London,
United KingdomSite Not Available
Great Ormond Street Hospital & University College London Hospital
London, WC1N 1EH
United KingdomActive - Recruiting
Great Ormond Street Hospital & University College London Hospital
London 2643743, WC1N 1EH
United KingdomActive - Recruiting
Institute of Genetic Medicine
Newcastle,
United KingdomSite Not Available
Institute of Genetic Medicine
Newcastle 6695976,
United KingdomActive - Recruiting

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