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

Recruiting
7 years - 100 years
Male
1 Location

Brief description of study

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

Detailed description of study

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.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Nonsense Mutation Duchenne Muscular Dystrophy
  • Age: 7 years - 100 years
  • Gender: Male

Inclusion Criteria

Diagnosed with nonsense mutation DMD confirmed by medical history and genotyping

Able to stand, walk, climb, and descend stairs

Taking corticosteroid treatment for DMD for at least 12 months

Updated on 04 Aug 2024. Study ID: 1809416902

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