Trial Profile

Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia

Status: Active, no longer recruiting

Phase of Trial: Phase III

Latest Information Update: 22 Apr 2025

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At a glance

Drugs Vatiquinone (Primary)
Indications Friedreich's ataxia
Focus Adverse reactions; Registrational
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.
28 Jan 2025 Status changed from recruiting to active, no longer recruiting.
20 Apr 2024 The protocol has been amended to reduce in study time frame for assessing Number of Participants With Adverse Events Baseline up to 3 years.

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