A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
Phase 3 – large-scale trial before approval
Conditions:
Friedreich Ataxia
Sponsor: PTC Therapeutics
trial.available_in:
БГ
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.
Description
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.
Who can participate
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.
Exclusion Criteria:
* Current participation in any other interventional study
* Pregnancy or breastfeeding
Locations
9
Australia (1)
Murdoch Children's Research Institute
Parkville , Victoria
Brazil (1)
University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology
São Paulo
Canada (1)
Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)
Montreal , Quebec
France (1)
Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute)
Paris
Germany (1)
Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE)
Tübingen
Italy (1)
Ospedale Pediatrico Bambino Gesu' IRCCS
Roma
New Zealand (1)
CBR Neurogenetic Research Clinic, University of Auckland
Auckland
Spain (1)
Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares