Clinical Trials Directory

Trials / Unknown

UnknownNCT01385917

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

Status
Unknown
Phase
Study type
Observational
Enrollment
45 (estimated)
Sponsor
Genethon · Academic / Other
Sex
Male
Age
12 Years – 20 Years
Healthy volunteers
Not accepted

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.

Conditions

Timeline

Start date
2011-10-01
Primary completion
2018-12-01
Completion
2018-12-01
First posted
2011-06-30
Last updated
2016-04-12

Locations

2 sites across 2 countries: France, United Kingdom

Source: ClinicalTrials.gov record NCT01385917. Inclusion in this directory is not an endorsement.