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 on Investigational Medication for Slowing DMD Progression

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 19 Feb 2024. Study ID: 1809416902

This study investigates the effects of an investigational medication on slowing the progression of Duchenne muscular dystrophy (DMD) in boys with specific genetic mutations. Duchenne muscular dystrophy is a genetic disorder characterized by progressive muscle degeneration and weakness. The study aims to assess how the investigational medication affects walking, muscle function, and quality of life in children with DMD.

Participants will undergo various tests to evaluate their muscle function, including walking tests and assessments of upper and lower limb strength. Some participants will also have magnetic resonance imaging (MRI) at certain locations to further evaluate muscle health. The study will also monitor the safety of the investigational medication over a period of time.

  • Who can participate: Boys diagnosed with nonsense mutation DMD confirmed by medical history and genotyping are eligible. Participants must be able to stand, walk, climb, and descend stairs, and have been on corticosteroid treatment for at least 12 months.
  • Study details: Participants will take part in tests to measure walking and muscle function. Some may have MRIs if they are at participating sites. A placebo is not used in this study. A placebo is an inactive substance that looks like the investigational medicine/vaccine but does not contain any medicine.
  • Study Timelines: The study will last 144 weeks.

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