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

Running, not enrolling · Phase 3

Conditions studied: Friedreich Ataxia

In brief

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.

Key facts

Study ID

NCT05515536

Run by

PTC Therapeutics

People needed

130

Starts

2022-12-08

Expected to finish

2027-12-31

Last updated by the study team

2025-10-30

Who can join

Age: any. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• Current participation in any other interventional study
• Pregnancy or breastfeeding

Where it is running

• 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 — Montreal, Quebec, Canada
• 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

Full record on ClinicalTrials.gov

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