At a glance
ClinicalIndex Comparison RecordPhase 3Completed· 109 enrolled
Drug / intervention
eteplirsendrug
Likely dose
eteplirsen 30 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.
An Open-Label, Multi-Center, Study With a Concurrent Untreated Control Arm to Evaluate the Efficacy and Safety of Eteplirsen in Duchenne Muscular Dystrophy
In Brief
A Phase 3 clinical trial evaluating eteplirsen for Duchenne Muscular Dystrophy (DMD). Completed, enrolled 109 participants across 37 sites.
Detailed Summary
The main objective of this study is to provide evidence of efficacy of eteplirsen (AVI-4658) in Duchenne muscular dystrophy (DMD) patients that are amenable to skipping exon 51. Additional objectives include evaluation of safety, biomarkers and the long-term effects of eteplirsen up to 96 weeks, followed by a safety extension (not to exceed 48 weeks).
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsDuchenne Muscular Dystrophy (DMD)
CountriesUnited States
Collaborators--
Timeline
Phase 3CompletedFinished
201520162017201820192020202120222023202420252026
First PostedOct 2014
Enrollment StartNov 2014
Primary CompletionJun 2019
TodayJul 2026
First PostedOct 2, 2014
Enrollment StartNov 17, 2014
Primary CompletionJun 14, 2019
TodayJul 2, 2026
Enrollment to primary: 4.6 yearsPosted 11.8 years ago
Interventions
eteplirsendrug
Eteplirsen 30 mg/kg will be administered as an IV infusion once a week for 96 weeks, followed by a safety extension (not to exceed 48 weeks).