Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping
NCT ID: NCT01385917
Last Updated: 2016-04-12
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
45 participants
OBSERVATIONAL
2011-10-31
2018-12-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
PROSPECTIVE
Eligibility Criteria
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Inclusion Criteria
* Age between ≥ 12 and \<20 years old.
* Non ambulant patients (i;e; inability to walk more than 10 meters without any of assistance).
* Patients covered by a national health insurance scheme.
* Signed informed consent.
Exclusion Criteria
* Patients with severe intellectual impairment preventing them from fully understanding the exercises to be performed.
* Recent (less than 6 months ago) upper limb surgery or trauma This criteria is however no definitive. Patients who have undergone upper limb surgery or trauma may nonetheless be enrolled once the 6 month period is over.
* Known immune deficiency.
* Contraindications to NMR exams
12 Years
20 Years
MALE
No
Sponsors
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Institute of Myology
OTHER
Genethon
OTHER
Responsible Party
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Principal Investigators
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Laurent SERVAIS, MD
Role: PRINCIPAL_INVESTIGATOR
Myology Institute
Locations
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Myology Institute
Paris, , France
Great ormond Street Hospital & University College London Hospital
London, , United Kingdom
Countries
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Facility Contacts
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Other Identifiers
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GHN007.10
Identifier Type: -
Identifier Source: org_study_id
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