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
Marcia Felker, MD
Primary Investigator
Overview
This study is being done to see if Ataluren will slow the progression of DMD in boys with DMD who have specific mutations.
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.
Eligibility
You may be eligible for this study if you meet the following criteria:
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Conditions:
Nonsense Mutation Duchenne Muscular Dystrophy
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Age: Between 7 Years - 100 Years
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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
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