At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 15 enrolled
Drug / intervention
Eteplirsendrug
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 Safety, Tolerability, and Pharmacokinetics Study of Eteplirsen in Young Patients With Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping
In Brief
A Phase 2 clinical trial evaluating Eteplirsen for Duchenne Muscular Dystrophy. Completed, enrolled 15 participants across 4 sites in 4 countries.
Detailed Summary
This is a multicenter, open-label, dose-escalation study to evaluate the safety, tolerability, and PK of once-weekly IV infusions of eteplirsen in approximately 12 male participants, ages 6 months to 48 months (inclusive), who have genotypically confirmed DMD with a deletion mutation amenable to exon 51 skipping.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsDuchenne Muscular Dystrophy
CountriesBelgium, France, Italy, United Kingdom
Collaborators--
Timeline
Phase 2CompletedFinished
201820192020202120222023202420252026
First PostedJul 2017
Enrollment StartAug 2017
Primary CompletionMar 2021
TodayJul 2026
First PostedJul 17, 2017
Enrollment StartAug 16, 2017
Primary CompletionMar 10, 2021
TodayJul 2, 2026
Enrollment to primary: 3.6 yearsPosted 9.0 years ago
Interventions
Eteplirsendrug
Infusion for intravenous use.