NCT05515536

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

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

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 130 (actual)
• Sponsor: PTC Therapeutics · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Conditions

• Friedreich Ataxia

Interventions

Type	Name	Description
DRUG	Vatiquinone	Vatiquinone will be administered per dose and schedule specified in the arm.

Timeline

• Start date: 2022-12-08
• Primary completion: 2027-12-31
• Completion: 2027-12-31
• First posted: 2022-08-25
• Last updated: 2025-10-30

Locations

14 sites across 9 countries: United States, Australia, Brazil, Canada, France, Germany, Italy, New Zealand, Spain

Regulatory

• FDA-regulated drug study