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

> **NCT04577352** · PHASE2,PHASE3 · COMPLETED · sponsor: **PTC Therapeutics** · enrollment: 146 (actual)

## Conditions studied

- Friedreich Ataxia

## Interventions

- **DRUG:** Vatiquinone
- **DRUG:** Placebo

## Key facts

- **NCT ID:** NCT04577352
- **Lead sponsor:** PTC Therapeutics
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE2,PHASE3
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2020-12-17
- **Primary completion:** 2023-04-04
- **Final completion:** 2023-10-02
- **Target enrollment:** 146 (ACTUAL)
- **Last updated:** 2026-04-13


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT04577352

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT04577352, "A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia". Retrieved via AI Analytics 2026-05-26 from https://api.ai-analytics.org/clinical/NCT04577352. Licensed CC0.

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