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NCT Number: NCT05515536

A Study to Assess the Safety and Efficacy of Vatiquinone in Participants 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.

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This study is active but is not currently recruiting participants.

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Key information

Conditions

Friedreich Ataxia Brain Diseases Central Nervous System Diseases Cerebellar Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Metabolic Diseases Mitochondrial Diseases Nervous System Diseases Neurodegenerative Diseases Nutritional and Metabolic Diseases Spinal Cord Diseases Spinocerebellar Degenerations

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Murdoch Children's Research Institute, Parkville, Victoria, Australia

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Vatiquinone

Drug

Vatiquinone will be administered per dose and schedule specified in the arm.

Primary outcomes

  1. Number of Participants With Adverse Events (AEs)

    Time frame: Baseline up to 3 years

Secondary outcomes

  1. 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

Sponsors and collaborators

Lead sponsor

PTC Therapeutics

Industry

Registry information

Official study title

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

Important dates

Study start
2022
Primary completion
2027
Study completion
2027
First posted
Aug 25, 2022
Registry last updated
Oct 30, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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