# Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia

> **NCT01728064** · PHASE2 · COMPLETED · sponsor: **PTC Therapeutics** · enrollment: 60 (estimated)

## Conditions studied

- Friedreich's Ataxia

## Interventions

- **DRUG:** Placebo
- **DRUG:** EPI-743 400 mg
- **DRUG:** EPI-743 200 mg

## Key facts

- **NCT ID:** NCT01728064
- **Lead sponsor:** PTC Therapeutics
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2012-12-31
- **Primary completion:** 2015-10-31
- **Final completion:** 2016-02-29
- **Target enrollment:** 60 (ESTIMATED)
- **Last updated:** 2020-08-31


## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01728064

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01728064, "Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia". Retrieved via AI Analytics 2026-09-04 from https://api.ai-analytics.org/clinical/NCT01728064. Licensed CC0.

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