At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 14 enrolled
Drug / intervention
Atalurendrug
Likely dose
Not stated in 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 2 Study of the Safety, Pharmacokinetics, and Pharmacodynamics of Ataluren (PTC124®) in Patients Aged ≥2 to <5 Years Old With Nonsense Mutation Dystrophinopathy
In Brief
A Phase 2 clinical trial evaluating Ataluren for Duchenne Muscular Dystrophy. Completed, enrolled 14 participants across 6 sites.
Detailed Summary
This is a Phase 2, multiple-dose, open-label study evaluating the safety, pharmacokinetics (PK), and pharmacodynamics (PD) of ataluren in participants aged ≥2 to \<5 years old with Duchenne muscular dystrophy (DMD) caused by a nonsense mutation in the dystrophin gene.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsDuchenne Muscular Dystrophy
CountriesUnited States
Collaborators--
Timeline
Phase 2CompletedFinished
2017201820192020202120222023202420252026
Enrollment StartJun 2016
First PostedJun 2016
Primary CompletionFeb 2018
TodayJul 2026
First PostedJun 30, 2016
Enrollment StartJun 9, 2016
Primary CompletionFeb 9, 2018
TodayJul 2, 2026
Enrollment to primary: 1.7 yearsPosted 10.0 years ago
Interventions
Atalurendrug
White to off-white powder for oral suspension.