at La Jolla, California and other locations
study started
completion around



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.

Official Title

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


Inherited Mitochondrial Disease, Leigh syndrome, Alpers Syndrome, mitochondrial encephalomyopathy, MELAS, MERRF, PCH6, refractory epilepsy, Mitochondrial Diseases, Vatiquinone


You can join 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 CAN'T join if...

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


  • University of California, San Diego Altman Clinical and Translational Research Institute
    La Jolla California 92037 United States
  • Stanford University
    Stanford California 94305 United States


accepting new patients by invitation only
Start Date
Completion Date
PTC Therapeutics
Phase 3 Mitochondrial Disease Research Study
Study Type
Expecting 100 study participants
Last Updated