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

PHASE3Active, not recruitingINTERVENTIONAL
Enrollment

130

Participants

Timeline

Start Date

December 8, 2022

Primary Completion Date

December 31, 2027

Study Completion Date

December 31, 2027

Conditions
Friedreich Ataxia
Interventions
DRUG

Vatiquinone

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

Trial Locations (14)

1023

CBR Neurogenetic Research Clinic, University of Auckland, Auckland

3052

Murdoch Children's Research Institute, Parkville

19104

The Children's Hospital of Philadelphia, Philadelphia

32608

University of Florida, Gainesville

33612

University of South Florida, Tampa

52242

University of Iowa, Iowa City

72076

Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE), Tübingen

75646

Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute), Paris

90095

UCLA, Los Angeles

13083-887

University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology, São Paulo

H2X 0A9

Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM), Montreal

H3T1C5

CHU Sainte-Justine, Montreal

00165

Ospedale Pediatrico Bambino Gesu' IRCCS, Roma

08950

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

Sponsors

Lead Sponsor

All Listed Sponsors
lead

PTC Therapeutics

INDUSTRY

NCT05515536 - A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia | Biotech Hunter | Biotech Hunter