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Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy A phase I/II/III study with a dose determination part followed by an efficacy and safety evaluation, quadruple blind placebo-controlled part and then by a long term safety follow up part, in ambulant boys

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Trial Profile

Microdystrophin (GNT0004) Gene Therapy Clinical Trial in Duchenne Muscular Dystrophy A phase I/II/III study with a dose determination part followed by an efficacy and safety evaluation, quadruple blind placebo-controlled part and then by a long term safety follow up part, in ambulant boys

Status: Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 26 May 2025

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At a glance

  • Drugs GNT 0004 (Primary)
  • Indications Duchenne muscular dystrophy
  • Focus Therapeutic Use
  • Sponsors Genethon

Most Recent Events

  • 19 May 2025 According to a Genethon media release, The initial part of the clinical trial aimed at selecting the optimal dose (dose escalation phase), evaluating the tolerance and preliminary efficacy of the treatment and determined the effective dose for the pivotal phase of the GNT-016-MDYF trial, which is expected to start in mid-2025 .
  • 19 May 2025 According to a Genethon media release, data from this study presented at meeting of the American Society of Gene & Cell Therapy (ASGCT) in New Orleans, May 13-17 2025.
  • 19 May 2025 Results presented in the Genethon Media Release

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