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

NCT05515536 · tracked via the Priya Life Science Canada tracker

Sponsor

PTC Therapeutics

Phase

Phase 3

Started

2022-12-08

Last updated

2025-10-30

Condition(s) studied

Friedreich Ataxia

Investigational drug(s) / intervention(s)

Vatiquinone →

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

Study 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.

Eligibility

Sex

ALL

Min age

—

Max age

—

Healthy volunteers

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

Primary outcome measure(s)

Number of Participants With Adverse Events (AEs) — Baseline up to 3 years

Trial sites (14)

Facility	City	Region
UCLA	Los Angeles	California
University of Florida	Gainesville	Florida
University of South Florida	Tampa	Florida
University of Iowa	Iowa City	Iowa
The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
Murdoch Children's Research Institute	Parkville	Victoria
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
CHU Sainte-Justine	Montreal	Quebec
Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute)	Paris	France
Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE)	Tübingen	Germany
Ospedale Pediatrico Bambino Gesu' IRCCS	Roma	Italy
CBR Neurogenetic Research Clinic, University of Auckland	Auckland	New Zealand
Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares	Barcelona	Spain

More PTC Therapeutics trials in Canada

Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA) Starting soon

Other trials for the same condition

FA Clinical Outcome Measures Active, not recruiting

A Natural History Study to TRACK Brain and Spinal Cord Changes in Individuals with Friedreich Ataxia (TRACK-FA) Active, not recruiting

A Study to Learn More About the Effects and Long-Term Safety of Omaveloxolone (BIIB141) in Children and Teens With Friedreich's Ataxia Recruiting

Clinical Course Of Disease In Participants With FA-CM Recruiting

Friedreich Ataxia Global Clinical Consortium UNIFIED Natural History Study Recruiting

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05515536 on ClinicalTrials.gov ↗ ← All trials in Canada