NO_LONGER_AVAILABLENot Applicable

Vatiquinone Expanded Access Protocol

Plain-language summary

The purpose of this study is to provide participants who successfully completed the study titled "An Open-label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease" with liquid Vatiquinone through an intermediate patient population expanded access protocol.

Who can participate

Age range1 Year

SexALL

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Inclusion Criteria: * Subjects with molecular genetic mitochondrial disease including Leigh syndrome, and Alpers syndrome. * Completed participation in the study titled "An Open-label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease" * In the opinion of the clinical investigator, the continuation to receive Vatiquinone will be of benefit to the participants. * Enrollment into a clinical trial to receive Vatiquinone is not possible Exclusion Criteria: * Current participation in any other interventional study * Pregnant or currently breast feeding.

Trial details

NCT IDNCT07159139

SponsorMedical University of South Carolina

Sponsor typeOTHER

Study typeEXPANDED_ACCESS

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