Queen Square Centre for Neuromuscular Diseases



A study of SRP-4053 in Patients with Duchenne Muscular Dystrophy (DMD) Amenable to Exon 53 Skipping.

Sponsor Sarepta, EU Grant
CI Professor Francesco Muntoni
sites Newcastle and London GOSH
Contact Newcastle: becky.davis@ncl.ac.uk; London: c.uzowuru@ucl.ac.uk
More information on the trial  

Trial information

This is a first-in-human, multi-centre, multiple-dose study.

Primary objective

To assess the safety, tolerability, efficacy, and pharmacokinetics of once weekly intravenous infusion of the drug SRP-4053 in patients with genotypically-confirmed DMD with a deletion amenable to exon 53 skipping.

Trial design

This is a two part study. Part one is a randomised, double-blind, placebo-controlled, dose-titration trial to assess the safety, tolerability, and pharmacokinetics of four doses of SRP-4053 over approximately 12 weeks.

Part two is a 48-week, open-label evaluation to assess the efficacy and safety of the chosen dose of the drug determined in part one compared with an untreated control group.


12 patients with DMD will be recruited for the part one.

All 12 treated patients from part one plus an additional 12 new patients in part two must have a clinical diagnosis of DMD confirmed by the finding of a genomic deletion amenable to exon 53 skipping.

Untreated control patients in Part 2 (up to 24) will be DMD patients with genotypically-confirmed DMD who are not amenable to treatment by exon 53 skipping but otherwise meet the same eligibility criteria as treated patients new to part two. 

Part one will be about 12 weeks while Part two will last approximately 52 weeks. Including a four to six week screening period, the total study duration (for patients participating in both parts one and two) will be approximately 70 weeks.