AN OPEN-LABEL, SAFETY STUDY FOR PREVIOUSLY TREATED VATIQUINONE (PTC743) SUBJECTS WITH INHERITED MITOCHONDRIAL DISEASE
PTC743-LSEP
What is the goal of the study?
This is an open-label, Phase 3 safety study of vatiquinone in participants with inherited mitochondrial disease. These participants have been previously treated with vatiquinone at Seattle Children's or another investigational site in a prior PTC/BioElectron-sponsored clinical study or treatment plan. Currently we have 7 patients in 3 vatiquinone studies that will be eligible to continue long term in this study. About 200 previously treated vatiquinone participants will be enrolled at up to approximately 40 investigational sites worldwide. Participants will receive vatiquinone oral solution (100 mg/mL), up to 400 mg, administered orally or via feeding tube three times a day. The participant will continue the dose from the study they were enrolling from.
Who can participate in the study?
Please contact the study team listed below to learn more.