Trial Profile

An Open-Label, Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

Status: Completed

Phase of Trial: Phase III

Latest Information Update: 16 Jan 2026

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At a glance

Drugs Vatiquinone (Primary)
Indications Lactic acidosis; Leigh disease; MELAS syndrome; MERRF syndrome; Mitochondrial disorders; Mitochondrial encephalomyopathies; Olivopontocerebellar atrophies
Focus Adverse reactions; Registrational
Sponsors PTC Therapeutics

Most Recent Events

05 Sep 2025 Status changed from active, no longer recruiting to completed.
04 Mar 2025 Status changed from recruiting to active, no longer recruiting.
23 Sep 2024 Planned number of patients changed from 100 to 204.

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