A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

The primary objective of this study is to assess the safety of vatiquinone in participants with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron sponsored (previously Edison) clinical study or treatment plan.

The study will continue until vatiquinone becomes commercially available or the program is terminated.

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Key information

Conditions

Inherited Mitochondrial Disease Autoimmune Diseases Autoimmune Diseases of the Nervous System Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Carbohydrate Metabolism, Inborn Errors Cardiovascular Diseases Central Nervous System Diseases Cerebral Small Vessel Diseases Cerebrovascular Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Demyelinating Autoimmune Diseases, CNS Demyelinating Diseases Diffuse Cerebral Sclerosis of Schilder Drug Resistant Epilepsy Epilepsies, Myoclonic Epilepsy Epilepsy, Generalized Epileptic Syndromes Genetic Diseases, Inborn Immune System Diseases Leigh Disease Leukoencephalopathies MELAS Syndrome MERRF Syndrome Metabolic Diseases Metabolism, Inborn Errors Mitochondrial Diseases Mitochondrial Encephalomyopathies Mitochondrial Myopathies Muscular Diseases Musculoskeletal Diseases Myoclonic Epilepsies, Progressive Nervous System Diseases Neuromuscular Diseases Nutritional and Metabolic Diseases Pyruvate Metabolism, Inborn Errors Vascular Diseases

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Chu Angers, Angers, France

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Participants with inherited mitochondrial disease including Leigh syndrome, Alpers syndrome, mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS), myoclonic epilepsy with ragged-red fibers (MERRF), pontocerebellar hypoplasia type 6 (PCH6), or other mitochondrial disease who participated in a previous vatiquinone clinical study or treatment plan.
Women of childbearing potential must have a negative pregnancy test at screening/baseline and agree to abstinence or the use of at least 1 of the highly effective forms of contraception as specified in the protocol (with a failure rate of <1% per year when used consistently and correctly). Highly effective contraception or abstinence must be continued for the duration of the study, and for up to 30 days after the last dose of study drug.
Fertile men who are sexually active with women of childbearing potential and who have not had a vasectomy, must agree to use a barrier method of birth control during the study and for up to 30 days after the last dose of study drug.

Exclusion criteria

Current participation in any other interventional study.
Pregnancy or breast feeding.

Treatment and study plan

Vatiquinone

Drug

Vatiquinone will be administered per dose and schedule specified in the arm description.

Other names: PTC743

Primary outcomes

Number of Participants With Treatment-emergent Adverse Events (TEAEs)

Time frame: Baseline (Day 1) up to 30 days after last dose of study drug (956 days)

An adverse event (AE) was any untoward medical occurrence in a participant who received study drug without regard to possibility of causal relationship. AEs included both serious adverse events (SAEs) and non-serious AEs. A TEAE was defined as an AE that had an onset date or date of worsening on or after the first dose of study drug and within 30 days of the date of the last dose of treatment. A summary of other non-serious AEs and all SAEs, regardless of causality is located in the 'Reported AE section'.

Sponsors and collaborators

Lead sponsor

PTC Therapeutics

Industry

Registry information

Official study title

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

Important dates

Study start: 2022
Primary completion: 2025
Study completion: 2025
First posted: Feb 1, 2022
Registry last updated: Dec 22, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed