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A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
Sponsor: PTC Therapeutics
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.
Official title: Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia
Key Details
Gender
All
Age Range
Any - Any
Study Type
INTERVENTIONAL
Enrollment
130
Start Date
2022-12-08
Completion Date
2027-12-31
Last Updated
2025-10-30
Healthy Volunteers
No
Conditions
Interventions
Vatiquinone
Vatiquinone will be administered per dose and schedule specified in the arm.
Locations (14)
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