At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 6 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.
An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy
In Brief
A Phase 2 clinical trial evaluating Ataluren for Nonsene Mutation Duchenne Muscular Dystrophy. Completed, enrolled 6 participants across 1 site.
Detailed Summary
This study is designed to evaluate safety, tolerability, and pharmacokinetics (PK) in male children with nmDMD aged ≥6 months to \<2 years treated daily for 24 weeks with orally administered ataluren 10, 10, and 20 milligrams/kilogram (mg/kg) (morning, mid-day, and evening dose, respectively).
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
CountriesUnited States
Collaborators--
Timeline
Phase 2CompletedFinished
202120222023202420252026
First PostedApr 2020
Enrollment StartDec 2021
Primary CompletionAug 2023
TodayJul 2026
First PostedApr 7, 2020
Enrollment StartDec 29, 2021
Primary CompletionAug 7, 2023
TodayJul 2, 2026
Enrollment to primary: 1.6 yearsPosted 6.2 years ago
Interventions
Atalurendrug
Ataluren will be administered as per the dose and schedule specified in the arm.