Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
100 participants
OBSERVATIONAL
2019-12-19
2023-06-30
Brief Summary
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Detailed Description
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Primary Ojectives:
* To assess the natural disease course using standardized and disease appropriate evaluations over a period of 6 to 36 months in a cohort of young male subjects aged from 5 to 9 years at inclusion and diagnosed for Duchenne Muscular Dystrophy (DMD).
* To record a baseline period prior to the setup of an AAV gene therapy dose escalation phase I/II First in Man 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.
Conditions
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Study Design
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COHORT
PROSPECTIVE
Eligibility Criteria
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Inclusion Criteria
2. 5 to 9 years old inclusive
3. Body-Weight \< or = 75th percentile of BMI body-mass index scale (according to validated scale in force in the country site)
4. Diagnosis of DMD based upon Gene testing positive with detailed genotyping
5. Able to achieve:
* NSAA (North Star Ambulatory Assessment) scale \> or =18 (with a maximum of 2 points difference between inclusion and screening visits) and/or:
* Gowers test \< or =7 sec
* 6 Minute Walk Test (6MWT) \> or = 350 meters at screening visit (M1) and at inclusion visit (M0) with the distance being 20% of each other
6. Ongoing corticosteroid therapy or initiation of corticosteroid therapy according to standard of care in the previous 3 months
7. Signed informed consent by at least one parent(s) or both parents or legal guardian representative(s), when applicable and according to the country regulation
8. Affiliated Beneficiary of the National Health Care scheme
Exclusion Criteria
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
12. Muscle testing: inability to cooperate with
13. Nuclear Magnetic Resonance Imaging (NMRI): metal implants in regions of interest for the study
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) prior to the screening visit of the study
16. Concomitant participation to any other clinical trial
5 Years
9 Years
MALE
No
Sponsors
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Genethon
OTHER
Responsible Party
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Principal Investigators
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Francesco MUNTONI, Pr
Role: PRINCIPAL_INVESTIGATOR
GOSH LONDON
Locations
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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
Hôpital Armand Trousseau
Paris, , France
Hôpital Hautepierre
Strasbourg, , France
Great Ormond Street Hospital & University College London Hospital
London, , United Kingdom
Institute of Genetic Medicine
Newcastle, , United Kingdom
Countries
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Central Contacts
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Other Identifiers
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GNT-014-MDYF
Identifier Type: -
Identifier Source: org_study_id
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