At a glance
ClinicalIndex Comparison RecordPhase 2Completed· 39 enrolled
Drug / intervention
Placebo +1 moredrug
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 2-Part, Randomized, Double-Blind, Placebo-Controlled, Dose-Titration, Safety, Tolerability, and Pharmacokinetics Study (Part 1) Followed by an Open-Label Efficacy and Safety Evaluation (Part 2) of SRP-4053 in Patients With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
In Brief
A Phase 2 clinical trial evaluating Placebo and SRP-4053 for Duchenne Muscular Dystrophy. Completed, enrolled 39 participants across 5 sites in 4 countries.
Detailed Summary
This is a first-in-human, multiple-dose 2-part study to assess the safety, tolerability, efficacy, and pharmacokinetics of SRP-4053 in Duchenne muscular dystrophy (DMD) patients with deletions amenable to exon 53 skipping.
Study Details
Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsDuchenne Muscular Dystrophy
CountriesFrance, Italy, United Kingdom, United States
Timeline
Phase 2CompletedFinished
201520162017201820192020202120222023202420252026
First PostedDec 2014
Enrollment StartJan 2015
Primary CompletionMar 2019
TodayJul 2026
First PostedDec 8, 2014
Enrollment StartJan 13, 2015
Primary CompletionMar 25, 2019
TodayJul 2, 2026
Enrollment to primary: 4.2 yearsPosted 11.6 years ago
Interventions
Placebodrug
SRP-4053 placebo-matching solution for IV infusion.
SRP-4053drug
SRP-4053 (golodirsen) solution for IV infusion.