Observational Study of Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping

Duchenne Muscular Dystrophy Clinical Trial

— pre U7-53  

Official title:

Study of Clinical and Radiological Changes in Patients With Duchenne Muscular Dystrophy Theoretically Treatable With Exon 53 Skipping

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT01385917
• Other study ID #: GHN007.10
• Status: Recruiting
• Phase: N/A
• First received: June 29, 2011
• Last updated: April 11, 2016
• Start date: October 2011
• Est. completion date: December 2018

Study information

• Verified date: March 2016
• Source: Genethon
• Contact: n/a
• Is FDA regulated: No
• Health authority: France: Committee for the Protection of PersonnesFrance: Agence Nationale de Sécurité du Médicament et des produits de santé
• Study type: Observational

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 45
• Est. completion date: December 2018
• Est. primary completion date: December 2018
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 12 Years to 20 Years

Eligibility

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

Study Design

Observational Model: Cohort, Time Perspective: Prospective

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Locations

Country	Name	City	State
France	Myology Institute	Paris
United Kingdom	Great ormond Street Hospital & University College London Hospital	London

Sponsors (2)

Lead Sponsor	Collaborator
Genethon	Institute of Myology

Countries where clinical trial is conducted

France,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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 DMD, potentially treatable with AAV-mediated exon 53 skipping.	Every year	No