Clinical trial NCT05515536: A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia
Official title: Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Friedreich Ataxia
Lead sponsor: PTC Therapeutics
Sponsor class: INDUSTRY
First posted: 2022-08-25
Enrollment: 130
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.

Detailed 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.

Eligibility criteria: 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

Primary outcomes:
measure: Number of Participants With Adverse Events (AEs)
timeFrame: Baseline up to 3 years
Secondary outcomes:
measure: Change From Baseline in the Modified Friedreich Ataxia Rating Scale (mFARS) and its 4 Subscales (Upright Stability, Upper Limb, Lower Limb, Bulbar) Scores at Year 3
timeFrame: Baseline, Year 3
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: UCLA
state: California
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Iowa City
country: United States
facility: University of Iowa
state: Iowa
city: Philadelphia
country: United States
facility: The Children's Hospital of Philadelphia
state: Pennsylvania
city: Parkville
country: Australia
facility: Murdoch Children's Research Institute
state: Victoria
city: São Paulo
country: Brazil
facility: University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology
city: Montreal
country: Canada
facility: Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)
state: Quebec
city: Montréal
country: Canada
facility: CHU Sainte-Justine
state: Quebec
city: PARIS cedex
country: France
facility: Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute)
city: Tuebingen
country: Germany
facility: Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE)
city: Roma
country: Italy
facility: Ospedale Pediatrico Bambino Gesu' IRCCS
city: Auckland
country: New Zealand
facility: CBR Neurogenetic Research Clinic, University of Auckland
city: Barcelona
country: Spain
facility: Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares
Study officials:
References: