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

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Updated on 19 February 2024

Summary

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.

Details
Condition Nonsense Mutation Duchenne Muscular Dystrophy
Age 7years - 100years
Clinical Study IdentifierTX10227
Last Modified on19 February 2024

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Additional screening procedures may be conducted by the study team before you can be confirmed eligible to participate.

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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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