Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia
Public ClinicalTrials.gov record NCT01728064. 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
Safety and Efficacy Study of EPI-743 on Visual Function in Patients With Friedreich's Ataxia
Study identification
- NCT ID
- NCT01728064
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Lead sponsor
- PTC Therapeutics
- Industry
- Enrollment
- 60 participants
Conditions and interventions
Conditions
Interventions
- Placebo Drug
- EPI-743 400 mg Drug
- EPI-743 200 mg Drug
Drug
Eligibility (public fields only)
- Age range
- 18 Years to 45 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
- Dec 30, 2012
- Primary completion
- Oct 30, 2015
- Completion
- Feb 28, 2016
- Last update posted
- Aug 30, 2020
2012 – 2016
United States locations
- U.S. sites
- 3
- U.S. states
- 3
- U.S. cities
- 3
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California Los Angeles | Los Angeles | California | 90095 | — |
| University of South Florida | Tampa | Florida | 33612 | — |
| Childrens Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | — |
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 NCT01728064, 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 21, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT01728064 live on ClinicalTrials.gov.