A Phase 3, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Ataluren in Patients with Nonsense Mutation Duchenne Muscular Dystrophy and Open-Label Extension

Study Overview

This study is being done to see if Ataluren will slow the progression of DMD in boys with DMD who have specific mutations.

Study Description

The main goals of this study are to obtain additional information on whether the actions of ataluren can slow disease progression as assessed by tests for walking, lower-limb muscle function, upper-limb muscle function for children 7 years of age and older, muscle strength for children under 7 years of age, magnetic resonance imaging (MRI) only at participating sites, and health-related quality of life (HRQL) in males with DMD, and provide additional information on the safety of ataluren over 144 weeks.

 

Participants will be compensated for their participation.


  • IRB Number: 1809416902
  • Research Study Identifier: TX10227
  • Principal Investigator: Marcia Felker, MD

Recruitment Status

Open

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