Study Overview:

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.

Anyone considering participating in a clinical trial should discuss the matter with their physician. FARA does not endorse or recommend any particular studies.

Study Details:

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.

Key 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.

Key Exclusion Criteria:

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

Additional inclusion and exclusion criteria may apply and will be evaluated by a study doctor. 

Length of Study Commitment:

3 years

Participating Study Locations

Institution Name and LocationInvestigatorStatus

Children’s Hospital of Philadelphia
Philadelphia, PA

Dr. David Lynch

Active, recruiting closed

University of South Florida
Tampa, FL

Dr. Teresa Zesiewicz

Active, recruiting closed

UCLA
Los Angeles, CA

Dr. Susan Perlman

Active, recruiting closed

University of Iowa

Dr. Kathy Mathews

Active, recruiting closed

University of Florida, Gainesville
Gainesville, FL

Dr. Sub Subramony

Active, recruiting closed

Murdoch Children’s Research Institute, Victoria, Australia

Dr. Martin Delatycki

Active, recruiting closed

Centre Hospitalier de l’Universite de Montreal, Canada

Dr. Antoine Duquette

Active, recruiting closed

CHU Sainte-Justine, Canada
Enrolling children <14 years old

Active, recruiting closed

University of Campinas (UNICAMP) , Brazil

Dr. Marcondes Cavalcante Franca Junior

Active, recruiting closed

France, Institute du Cerveau et de la Molelle epiniere (ICM), Hopital Universitaire Pitie-Salpetriere

Dr. Alexandra Durr

Active, recruiting closed

Auckland City Hospital, New Zealand

Dr. Richard Roxburgh

Active, recruiting closed

Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares, Spain

Dr. Alejandra Darling

Active, recruiting closed

Ospedale Pediatrico Bambino Gesu’ IRCCS, Italy

Dr. Enrico Bertini

Active, recruiting closed

Department of Neurology and Hertie-Institute for Clinical Brain Research German Center for Neurodegenerative Diseases, Germany

Explore the FA Drug Development Pipeline

FARA believes that there are many different approaches to treating Friedreich’s ataxia, and that it will require a cocktail approach of two or more treatments to slow, stop, reverse, and cure FA. Learn more about the approach behind this potential treatment and explore the other approaches that are in the FA Drug Development pipeline.