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

October 29, 2025 updated by: PTC Therapeutics

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

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

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 Type

Interventional

Enrollment (Actual)

130

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Australia
    • Victoria
      • Parkville, Victoria, Australia, 3052
        • Murdoch Children's Research Institute
  • Brazil
      • São Paulo, Brazil, 13083-887
        • University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology
  • Canada
    • Quebec
      • Montreal, Quebec, Canada, H3T1C5
        • CHU Sainte-Justine
      • Montreal, Quebec, Canada, H2X 0A9
        • Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)
  • France
      • Paris, France, 75646
        • Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute)
  • Germany
      • Tübingen, Germany, 72076
        • Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE)
  • Italy
      • Roma, Italy, 00165
        • Ospedale Pediatrico Bambino Gesu' IRCCS
  • New Zealand
      • Auckland, New Zealand, 1023
        • CBR Neurogenetic Research Clinic, University of Auckland
  • Spain
      • Barcelona, Spain, 08950
        • Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares
  • United States
    • California
      • Los Angeles, California, United States, 90095
        • UCLA
    • Florida
      • Gainesville, Florida, United States, 32608
        • University of Florida
      • Tampa, Florida, United States, 33612
        • University of South Florida
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • The Children's Hospital of Philadelphia

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: 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.
Drug: Vatiquinone
Vatiquinone will be administered per dose and schedule specified in the arm.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Number of Participants With Adverse Events (AEs)
Time Frame: Baseline up to 3 years
Baseline up to 3 years

Secondary Outcome Measures

Outcome Measure
Time Frame
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
Time Frame: Baseline, Year 3
Baseline, Year 3

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

PTC Therapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 8, 2022

Primary Completion (Estimated)

December 31, 2027

Study Completion (Estimated)

December 31, 2027

Study Registration Dates

First Submitted

August 23, 2022

First Submitted That Met QC Criteria

August 23, 2022

First Posted (Actual)

August 25, 2022

Study Record Updates

Last Update Posted (Estimated)

October 30, 2025

Last Update Submitted That Met QC Criteria

October 29, 2025

Last Verified

October 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PTC743-NEU-003e-FA
  • 2022-002668-65 (EudraCT Number)
  • 2024-516505-23-00 (Other Identifier: EU CT Number)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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