At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 20 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.
Phase 2 Clinical Pharmacology Study to Assess Dystrophin Levels in Subjects With nmDMD Before and After Treatment With Ataluren
In Brief
A Phase 2 clinical trial evaluating Ataluren for Duchenne Muscular Dystrophy. Completed, enrolled 20 participants across 9 sites.
Detailed Summary
This study is designed to evaluate the ability of ataluren to increase dystrophin protein levels in muscle cells of participants with nmDMD. The study will evaluate the levels of dystrophin before and after 40 weeks of ataluren therapy using muscle biopsies and 2 validated assay methods, electrochemiluminescence (ECL) and immunohistochemistry.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsDuchenne Muscular Dystrophy
CountriesUnited States
Collaborators--
Timeline
Phase 2CompletedFinished
20192020202120222023202420252026
First PostedAug 2018
Enrollment StartDec 2018
Primary CompletionOct 2020
TodayJul 2026
First PostedAug 27, 2018
Enrollment StartDec 21, 2018
Primary CompletionOct 23, 2020
TodayJul 2, 2026
Enrollment to primary: 1.8 yearsPosted 7.8 years ago
Interventions
Atalurendrug
Ataluren will be administered as per the dose and schedule specified in the arm.