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An Open-Label Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Vatiquinone in Children With Friedreich Ataxia Younger Than 7 Years of Age

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Trial Profile

An Open-Label Study to Evaluate Pharmacokinetics, Safety, and Efficacy of Vatiquinone in Children With Friedreich Ataxia Younger Than 7 Years of Age

Status: Completed
Phase of Trial: Phase II

Latest Information Update: 04 Mar 2025

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At a glance

  • Drugs Vatiquinone (Primary)
  • Indications Friedreich's ataxia
  • Focus Adverse reactions; Pharmacokinetics
  • Sponsors PTC Therapeutics

Most Recent Events

  • 27 Feb 2025 According to a PTC Therapeutics media release, Vatiquinone for children and adults with Friedreich's ataxia, granted priority review with a target regulatory action date of August 19, 2025.
  • 08 Oct 2024 Status changed from active, no longer recruiting to completed.
  • 24 Aug 2023 Planned End Date changed from 31 Aug 2024 to 5 Oct 2024.

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