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

Friedreich Ataxia Clinical Trial

Official title:

Long-Term Open-Label Study to Assess the Safety and Efficacy of Vatiquinone in Patients With Friedreich Ataxia

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT05515536
• Other study ID #: PTC743-NEU-003e-FA
• Secondary ID: 2022-002668-65
• Status: Enrolling by invitation
• Phase: Phase 3
• Start date: December 8, 2022
• Est. completion date: December 31, 2027

Study information

• Verified date: May 2024
• Source: PTC Therapeutics
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 140
• Est. completion date: December 31, 2027
• Est. primary completion date: December 31, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Friedreich Ataxia

Intervention

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

Locations

Country	Name	City	State
Australia	Murdoch Children's Research Institute	Parkville	Victoria
Brazil	University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology	São Paulo
Canada	Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)	Montreal	Quebec
Canada	CHU Sainte-Justine	Montréal	Quebec
France	Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute)	PARIS cedex
Germany	Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE)	Tuebingen
Italy	Ospedale Pediatrico Bambino Gesu' IRCCS	Roma
New Zealand	CBR Neurogenetic Research Clinic, University of Auckland	Auckland
Spain	Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares	Barcelona
United States	University of Florida	Gainesville	Florida
United States	University of Iowa	Iowa City	Iowa
United States	UCLA	Los Angeles	California
United States	The Children's Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	University of South Florida	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
PTC Therapeutics

Countries where clinical trial is conducted

United States,  Australia,  Brazil,  Canada,  France,  Germany,  Italy,  New Zealand,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Participants With Adverse Events (AEs)		Baseline up to 3 years
Secondary	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		Baseline, Year 3