At a glance
ClinicalIndex Comparison RecordPhase 3Completed· 360 enrolled
Drug / intervention
Ataluren +1 moredrug
Likely dose
Ataluren 20 mg/kgfrom record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.
Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.
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
In Brief
A Phase 3 clinical trial evaluating Ataluren and PLACEBO for Muscular Dystrophy, Duchenne and 8 related conditions. Completed, enrolled 360 participants across 64 sites in 18 countries.
Detailed Summary
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsMuscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases, Musculoskeletal Disease, Neuromuscular Diseases, Nervous System Diseases, Genetic Diseases, X-Linked, Genetic Diseases, Inborn
CountriesAustralia, Brazil, Bulgaria, Canada, China, Hong Kong, India, Japan, Malaysia, Mexico, Poland, Puerto Rico, Russia, South Korea, Taiwan, Thailand, Turkey (Türkiye), United States
Collaborators--
Timeline
Phase 3CompletedFinished
201820192020202120222023202420252026
First PostedJun 2017
Enrollment StartJul 2017
Primary CompletionMar 2022
Study CompletionJul 2023
TodayJul 2026
First PostedJun 7, 2017
Enrollment StartJul 6, 2017
Primary CompletionMar 5, 2022
Study CompletionJul 25, 2023
TodayJul 2, 2026
Enrollment to primary: 4.7 yearsPosted 9.1 years ago
Interventions
Atalurendrug
10, 20 mg/kg
PLACEBOdrug
10, 20 mg/kg