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

Completed · Phase 3

Conditions studied: Inherited Mitochondrial Disease

In brief

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.

Key facts

Study ID

NCT05218655

Run by

PTC Therapeutics

People needed

101

Starts

2022-06-22

Expected to finish

2025-04-15

Last updated by the study team

2025-12-22

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

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

Where it is running

• University of California, San Diego Altman Clinical and Translational Research Institute — La Jolla, California, United States
• Stanford University — Stanford, California, United States
• Yale Medicine — New Haven, Connecticut, United States
• Children's National — Washington D.C., District of Columbia, United States
• Child Neurology Center of Northwest Florida — Gulf Breeze, Florida, United States
• The Johns Hopkins University — Baltimore, Maryland, United States
• Columbia University Medical Center - CUMC — New York, New York, United States
• Akron Children's Hospital — Akron, Ohio, United States
• Cleveland Clinic — Cleveland, Ohio, United States
• Children's Hospital of Philadelphia - CHOP — Philadelphia, Pennsylvania, United States
• Medical University of South Carolina - MUSC — Charleston, South Carolina, United States
• Baylor College of Medicine — Houston, Texas, United States
• UT Health The University of Texas — Houston, Texas, United States
• Seattle Children Hospital — Seattle, Washington, United States
• Chu Angers — Angers, France
• CHU Montpellier - Hopital Saint-Eloi — Montpellier, France
• Hopital Necker-Enfants Malades — Paris, France
• Hôpital de Hautepierre - Hôpitaux Universitaires de Strasbourg — Strasbourg, France
• Ospedale Pediatrico Bambino Gesù — Roma, Italy
• PTC Clinical Site — Japanese City, Japan
• Instytut Pomnik - Centrum Zdrowia Dziecka — Warsaw, Poland
• Hospital Sant Joan de Déu — Barcelona, Spain
• Hospital Universitario 12 de Octubre — Madrid, Spain
• Hospital Ruber Internacional — Madrid, Spain
• Great Ormond Street Hospital for Children NHS Foundation Trust — London, United Kingdom

Full record on ClinicalTrials.gov

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