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NCT01721733 · ClinicalTrials.gov registry record · Phase 2
Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome
A Phase 2 study of Leigh Syndrome, sponsored by PTC Therapeutics.
- Completed
- Registry status
- Phase 2
- Development phase
- 35
- Enrollment target
- 4
- Study locations
NCT01721733: Completed Phase 2 study of Leigh Syndrome, sponsored by PTC Therapeutics.
NCT01721733 is a Phase 2 study of Leigh Syndrome that has completed, run by PTC Therapeutics. The registered enrollment target is 35 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (74% lower). The trial reports 4 study locations across 4 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01721733, a Phase 2 study of Leigh Syndrome, has completed, sponsored by PTC Therapeutics.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 35 participants
- Enrollment target
- 4
- Study locations
Study Summary
The purpose of this study is to evaluate the effects of EPI-743 in children with Leigh syndrome on disease severity, neuromuscular function, respiratory function, disease morbidity and mortality and disease associated biomarkers.
Primary Outcome
Change from baseline to six months will be compared between subjects in active treatment group and placebo group
Conditions Studied
Interventions
- DRUG Placebo
- DRUG EPI-743 15 mg/kg
- DRUG EPI-743 5 mg/kg
Study Locations (4)
California
- Stanford University - Palo Alto
Ohio
- Akron Children's Hospital - Akron
Texas
- Baylor College of Medicine - Houston
Washington
- Seattle Children's Hospital - Seattle
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 35 participants |
| Start Date | 2012-10-31 |
| Est. Completion | 2015-05-31 |
| Phase | Phase 2 |
What the finished NCT01721733 record still lists
NCT01721733 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 35 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (74% lower).
The record links to 1 condition, with Leigh Syndrome appearing as the primary indexed condition, and to 3 interventions - of which Placebo is the first listed.
NCT01721733 reports a single indexed study location in California, Ohio, Texas.
Frequently Asked Questions
What is clinical trial NCT01721733 about?
NCT01721733 is a clinical study titled "Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome". The purpose of this study is to evaluate the effects of EPI-743 in children with Leigh syndrome on disease severity, neuromuscular function, respiratory function, disease morbidity and mortality and disease associated biomarkers.
What is the current status of trial NCT01721733?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 35 participants. The study started on 2012-10-31. Estimated completion is 2015-05-31.
What conditions does trial NCT01721733 study?
This clinical trial studies the following conditions: Leigh Syndrome.
What interventions are being tested in trial NCT01721733?
The interventions under investigation include: Placebo (DRUG), EPI-743 15 mg/kg (DRUG), EPI-743 5 mg/kg (DRUG).
Who is sponsoring clinical trial NCT01721733?
This trial is sponsored by PTC Therapeutics, which has 52 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT01721733 being conducted?
This trial has 4 study locations across California, Ohio, Texas, Washington. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Leigh Syndrome
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
Where NCT01721733's enrollment target sits among peer trials
35 1524th of 2000 higher than 462 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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