146
Participants
Start Date
December 17, 2020
Primary Completion Date
April 4, 2023
Study Completion Date
October 2, 2023
Vatiquinone
Vatiquinone will be administered per dose and schedule specified in the arm.
Placebo
Placebo will be administered per schedule specified in the arm.
CBR Neurogenetic Research Clinic, University of Auckland, Auckland
Murdoch Children's Research Institute, Parkville
The Children's Hospital of Philadelphia, Philadelphia
University of Florida, Gainesville
University of South Florida, Tampa
University of Iowa, Iowa City
Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE), Tübingen
Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute), Paris
UCLA, Los Angeles
University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology, São Paulo
Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM), Montreal
CHU Sainte-Justine, Montreal
Ospedale Pediatrico Bambino Gesu' IRCCS, Roma
Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares, Barcelona
Lead Sponsor
PTC Therapeutics
INDUSTRY