LSEP Study
Research type
Research Study
Full title
AN OPEN-LABEL, SAFETY STUDY FOR PREVIOUSLY TREATED VATIQUINONE (PTC743) SUBJECTS WITH INHERITED MITOCHONDRIAL DISEASE
IRAS ID
1006123
Contact name
Vinay Penematsa
Contact email
Sponsor organisation
PTC THERAPEUTICS, INC.
Eudract number
2022-000375-39
Clinicaltrials.gov Identifier
Research summary
This is an open-label, Phase 3 safety study of vatiquinone in subjects with inherited mitochondrial disease who were previously treated with vatiquinone at an investigational site in a prior PTC/BioElectron-sponsored clinical study or treatment plan (parent study). A target of approximately 200 previously treated vatiquinone subjects will be enrolled at up to approximately 40 investigational sites worldwide.
Participants: Subjects 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.
Subjects will receive vatiquinone oral solution (100 mg/mL), up to 400 mg, administered orally or via feeding tube TID. Subjects will continue the dosing regimen from their parent study.
Safety assessments for subjects will be performed at screening/baseline and every 6 months until vatiquinone becomes commercially available or the program is terminated. Safety assessments will also be collected for all subjects at the End-of-Treatment Visit.Primary objective: to assess the safety of vatiquinone in subjects with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron-sponsored (previously Edison) clinical study or treatment plan.
Exploratory: to explore the effects of vatiquinone in total seizure count in subjects previously enrolled in vatiquinone Study MIT-E.
Sponsor: PTC Therapeutics.REC name
Wales REC 3
REC reference
22/WA/0290
Date of REC Opinion
5 Dec 2022
REC opinion
Further Information Favourable Opinion