A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

Study Overview
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Brief Summary
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The primary objective of this study is to assess the long-term safety of vatiquinone in participants with Friedreich ataxia (FA) previously exposed to vatiquinone in Study PTC743-NEU-003-FA (NCT04577352) or Study PTC743-NEU-005-FA.

Detailed Description
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Treatment will continue until the participant withdraw consent, regulatory authority and/or PTC discontinue the study, participant's condition substantially worsens after initiating vatiquinone treatment in this study, investigator withdraws the participant from vatiquinone treatment, if, in the investigator's clinical judgment, it is not in the participant's best interest to continue, or vatiquinone becomes commercially available in a participant's country.

Official Title
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Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia

Conditions
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Friedreich Ataxia

Intervention / Treatment
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* Drug: Vatiquinone


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Participants with FA who completed and directly rolled over from a previous vatiquinone clinical study (PTC743-NEU-003-FA [that is, MOVE-FA] or PTC743-NEU-005-FA). Males and females of childbearing potential must be willing to use an effective method of contraception (for example, implants, injectables, transdermal patches, combined oral contraceptives, barrier methods, and intrauterine devices) from the time consent is signed until 30 days after the last dose of study drug or Early Termination Visit. Exclusion Criteria: Current participation in any other interventional study Pregnancy or breastfeeding
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 with FA who were previously treated with vatiquinone at an investigational site and completed participation in a prior PTC Therapeutics (PTC)-sponsored clinical study (PTC743-NEU-003-FA or PTC743-NEU-005-FA) will continue to receive the same dose/formulation of vatiquinone (unless there has been a change in age and/or weight that meets the criteria for a different dose/formulation as described below; dose/formulation will also be changed if participants meet the criteria for a different dose/formulation during study treatment): If <7 years of age, participants will receive an oral solution (100 milligrams [mg]/milliliter [mL]) 3 times a day (TID) at one of the following doses: 15 mg/kilogram (kg) if body weight <13 kg, or 200 mg if body weight ≥13 kg. If ≥7 years of age, participants will receive a capsule formulation (200 mg) orally TID at one of the following doses: 200 mg if body weight ˂25 kg, or 400 mg if body weight ≥25 kg. | Drug: Vatiquinone<br>* Vatiquinone will be administered per dose and schedule specified in the arm.<br>|

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 up to approximately 5 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change From Baseline in the Modified Friedreich Ataxia Rating Scale (mFARS) at Year 5 |  | Baseline, Year 5 |