NCT04577352

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

A Randomized, Parallel-Arm, Double-Blind, Placebo-Controlled Study With Open-Label Extension to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Friedreich Ataxia (MOVE-FA)

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 146 (actual)
• Sponsor: PTC Therapeutics · Industry
• Sex: All
• Age: 7 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of the study is to evaluate the efficacy (using the modified Friedreich Ataxia Rating Scale \[mFARS\]) and safety of vatiquinone in participants with Friedreich ataxia (FA).

Detailed description

During the double-blind, placebo-controlled phase, participants will be stratified by baseline mFARS score (\<40 versus ≥40), age of disease onset (\<14 versus ≥14), and age at screening (≤21 years or \>21 years) and randomized to receive either vatiquinone or placebo using interactive response technology (IRT). Following completion of the randomized, double-blind, placebo-controlled phase (72 weeks), participants will enter into an open-label extension phase (24 weeks) during which they will receive open-label treatment with vatiquinone at the dose they received in the randomized phase of the study (for participants entering the extension phase who initially received placebo, the dose of vatiquinone will be determined based on age and weight) and then a safety follow-up (approximately 30 days \[±5 days\] after last dose or termination visit, whichever is later). The primary efficacy analysis will be based on change from baseline in mFARS score of participants between 7 and 21 years old. In order to explore the treatment efficacy and safety, approximately an additional 20 participants \>21 years of age will be randomized for a total of approximately 126 participants.

Conditions

• Friedreich Ataxia

Interventions

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

Timeline

• Start date: 2020-12-17
• Primary completion: 2023-04-04
• Completion: 2023-10-02
• First posted: 2020-10-06
• Last updated: 2026-04-13
• Results posted: 2026-04-08

Locations

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

Regulatory

• FDA-regulated drug study