# Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome

> **NCT01721733** · PHASE2 · COMPLETED · sponsor: **PTC Therapeutics** · enrollment: 35 (actual)

## Conditions studied

- Leigh Syndrome

## Interventions

- **DRUG:** Placebo
- **DRUG:** EPI-743 15 mg/kg
- **DRUG:** EPI-743 5 mg/kg

## Key facts

- **NCT ID:** NCT01721733
- **Lead sponsor:** PTC Therapeutics
- **Sponsor class:** INDUSTRY
- **Phase:** PHASE2
- **Study type:** INTERVENTIONAL
- **Status:** COMPLETED
- **Start date:** 2012-10-31
- **Primary completion:** 2015-02-28
- **Final completion:** 2015-05-31
- **Target enrollment:** 35 (ACTUAL)
- **Last updated:** 2020-08-31

## Collaborators

- [object Object]

## Primary source

ClinicalTrials.gov registry: https://clinicaltrials.gov/study/NCT01721733

## Citation

> US National Library of Medicine, ClinicalTrials.gov registration NCT01721733, "Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome". Retrieved via AI Analytics 2026-09-22 from https://api.ai-analytics.org/clinical/NCT01721733. Licensed CC0.

---

*[Clinical trials dataset](/datasets/clinical-trials) · CC0 1.0*