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Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping (pre U7-53)

This study is currently recruiting participants.
Verified March 2016 by Genethon
Sponsor:
ClinicalTrials.gov Identifier:
NCT01385917
First Posted: June 30, 2011
Last Update Posted: April 12, 2016
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
Institute of Myology
Information provided by (Responsible Party):
Genethon
June 29, 2011
June 30, 2011
April 12, 2016
October 2011
December 2018   (Final data collection date for primary outcome measure)
PreU7-53 is a natural history study [ Time Frame: Every year ]
The objective is to monitor the clinical and radiological course of upper limb muscle impairment in patients with DMD, potentially treatable with AAV-mediated exon 53 skipping.
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Complete list of historical versions of study NCT01385917 on ClinicalTrials.gov Archive Site
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Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping
Study of Clinical and Radiological Changes in Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping
PreU7-53 is a natural history study. The objective is to monitor the clinical and radiological course of upper limb muscle impairment in patients with Duchenne Muscular Dystrophy (DMD), potentially treatable with AAV-mediated exon 53 skipping.
Not Provided
Observational
Observational Model: Cohort
Time Perspective: Prospective
Not Provided
Retention:   None Retained
Description:
Total blood count with differential leukocyte count Assay of immunoglobulins and the various sub-classes (IgG, IgM, IgA, IgE) Test for immunisation against all AAV serotypes Serum urea, creatinine, AST/ALT , GammaGT, bilirubin, CK, alkalin phosphatase
Non-Probability Sample
Patients with theoretically exon 53 skipping-treatable DMD
Duchenne Muscular Dystrophy
Not Provided
Not Provided
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*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
45
December 2018
December 2018   (Final data collection date for primary outcome measure)

Inclusion Criteria:

  • Diagnosis of Duchenne muscular dystrophy confirmed by at least genetic testing, theoretically treatable by exon 53 skipping.
  • 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:

  • Patient incapable of sitting upright in a wheelchair for at least one hour.
  • 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
Sexes Eligible for Study: Male
12 Years to 20 Years   (Child, Adult)
No
France,   United Kingdom
 
 
NCT01385917
GHN007.10
No
Not Provided
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Genethon
Genethon
Institute of Myology
Principal Investigator: Laurent SERVAIS, MD Myology Institute
Genethon
March 2016