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 SRP4053 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping

Trial Profile

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 SRP4053 in Patients with Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping

Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 15 Dec 2017

At a glance

  • Drugs SRP 4053 (Primary)
  • Indications Duchenne muscular dystrophy
  • Focus Adverse reactions; First in man; Therapeutic Use
  • Sponsors Sarepta Therapeutics
  • Most Recent Events

    • 02 Oct 2017 Planned End Date changed from 1 May 2019 to 1 Mar 2019.
    • 02 Oct 2017 Planned primary completion date changed from 1 May 2019 to 1 Mar 2019.
    • 28 Sep 2017 According to a Sarepta Therapeutics media release, data from the study will be presented at the 22nd International Annual Congress of the World Muscle Society (WMS) 2017.
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