Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
Public ClinicalTrials.gov record NCT01721733. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 2B Randomized, Placebo Controlled, Double Blind Clinical Trial of EPI-743 in Children With Leigh Syndrome
Study identification
- NCT ID
- NCT01721733
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Lead sponsor
- PTC Therapeutics
- Industry
- Enrollment
- 35 participants
Conditions and interventions
Conditions
Interventions
- Placebo Drug
- EPI-743 15 mg/kg Drug
- EPI-743 5 mg/kg Drug
Drug
Eligibility (public fields only)
- Age range
- 6 Years to 17 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Oct 30, 2012
- Primary completion
- Feb 27, 2015
- Completion
- May 30, 2015
- Last update posted
- Aug 30, 2020
2012 – 2015
United States locations
- U.S. sites
- 4
- U.S. states
- 4
- U.S. cities
- 4
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Stanford University | Palo Alto | California | 94304 | — |
| Akron Children's Hospital | Akron | Ohio | 44308 | — |
| Baylor College of Medicine | Houston | Texas | 77030 | — |
| Seattle Children's Hospital | Seattle | Washington | 98105 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT01721733, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Aug 30, 2020 · Synced Jul 22, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT01721733 live on ClinicalTrials.gov.