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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

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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

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 27 Mar 2024

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At a glance

  • Drugs Golodirsen (Primary)
  • Indications Duchenne muscular dystrophy
  • Focus Adverse reactions; First in man; Registrational; Therapeutic Use
  • Sponsors Sarepta Therapeutics

Most Recent Events

  • 14 Sep 2020 According to a Sarepta Therapeutics media release, data from this study will be presented at the WMS25 Virtual Congress, the 25th International Annual Congress of the World Muscle Society. The company will host a webcast and conference call on Sept. 28, 2020.
  • 05 Mar 2020 Results published in the Neurology
  • 12 Dec 2019 According to an Food and Drug Administration media release, if the trial fails to verify clinical benefit, the FDA may initiate proceedings to withdraw approval of the drug.

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