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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 is a Phase 2 study of Leigh Syndrome that has completed, run by PTC Therapeutics. The registered enrollment target is 35 participants. The trial reports 4 study locations across 4 states.
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.
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 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01721733
The ClinicalTrials.gov registry entry for NCT01721733 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 35 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is PTC Therapeutics, which has 52 total studies on file at ClinicalTrials.gov.
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 4 study locations spanning 4 distinct geographic areas - top geographies include 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.
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Related
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