Inherited Mitochondrial Disease
Conditions
Interventions
Vatiquinone (PTC743):
Participants will receive vatiquinone oral solution (100 mg/mL), up to 400 mg, administered orally or via feeding tube 3 times daily (TID).
Sponsors
Vinay Penematsa
Eligibility
Sex/Gender
All
Inclusion criteria
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
Exclusion criteria: - Current participation in any other interventional study. - Pregnancy or breast feeding.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of Participants With Adverse Events (AEs) [Time Frame: Baseline (Day 1) up to end of study (up to approximately 3 years)] | — |
Countries
France, Italy, Japan, Poland, Spain, UK, US
Contacts
Public ContactChikako Rosario
Parexel International Inc.
Outcome results
None listed