A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

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

Conditions
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Inherited Mitochondrial Disease

Intervention / Treatment
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* Drug: Vatiquinone


Participation Criteria
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Eligibility Criteria
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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: Current participation in any other interventional study. Pregnancy or breast feeding.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Vatiquinone<br>Participants will receive vatiquinone oral solution (100 milligrams [mg]/milliliter [mL]), up to 400 mg, administered orally or via feeding tube 3 times daily (TID). | Drug: Vatiquinone<br>* Vatiquinone will be administered per dose and schedule specified in the arm description.<br>* Other names: PTC743;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Participants With Adverse Events (AEs) |  | Baseline (Day 1) up to end of study (up to approximately 3 years) | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Leigh syndrome, Alpers Syndrome, mitochondrial encephalomyopathy, MELAS, MERRF, PCH6, refractory epilepsy