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

Phase 3

Active, not recruiting

• Conditions: Friedreich Ataxia
• Interventions: Drug: Vatiquinone

• Registration Number: NCT05515536
• Lead Sponsor: PTC Therapeutics

Brief Summary

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.

Detailed Description

This long-term, open-label study will serve as a continued access study for participants with FA who have previously participated in a vatiquinone study. The purpose of this study is to assess continued safety and efficacy of vatiquinone dosing in previously treated participants. This study addresses the medical need for participants to continue vatiquinone with a planned study duration of 3 years.

Study Timeline

• Study First Submitted: 2022-08-23
• Study First Submitted QC: 2022-08-23
• Study First Posted: 2022-08-25
• Study Start: 2022-12-08
• Status Verified: 2025-07
• Last Update Submitted: 2025-07-15
• Last Update Posted: 2025-07-16
• Primary Completion: 2027-12-31
• Study Completion: 2027-12-31

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 130

Inclusion Criteria

• Participants with FA who completed and directly rolled over from a previous vatiquinone clinical study.
• 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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Vatiquinone	Vatiquinone	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 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.

Primary Outcome Measures

Name	Time	Method
Number of Participants With Adverse Events (AEs)	Baseline up to 3 years

Secondary Outcome Measures

Name	Time	Method
Change From Baseline in the Modified Friedreich Ataxia Rating Scale (mFARS) and its 4 Subscales (Upright Stability, Upper Limb, Lower Limb, Bulbar) Scores at Year 3	Baseline, Year 3

Trial Locations

Locations (14)

UCLA; 🇺🇸 Los Angeles, California, United States

University of Florida; 🇺🇸 Gainesville, Florida, United States

University of South Florida; 🇺🇸 Tampa, Florida, United States

University of Iowa; 🇺🇸 Iowa City, Iowa, United States

The Children's Hospital of Philadelphia; 🇺🇸 Philadelphia, Pennsylvania, United States

Murdoch Children's Research Institute; 🇦🇺 Parkville, Victoria, Australia

University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology; 🇧🇷 São Paulo, Brazil

Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM); 🇨🇦 Montreal, Quebec, Canada

CHU Sainte-Justine; 🇨🇦 Montréal, Quebec, Canada

Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute); 🇫🇷 PARIS cedex, France

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