Trials / Completed
CompletedNCT02352896
Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Syndrome
Long-Term Safety and Efficacy Evaluation of Vatiquinone (EPI-743) in Children With Leigh Syndrome
- Status
- Completed
- Phase
- Phase 2
- Study type
- Interventional
- Enrollment
- 30 (actual)
- Sponsor
- PTC Therapeutics · Industry
- Sex
- All
- Age
- 1 Year – 18 Years
- Healthy volunteers
- Not accepted
Summary
EPI-743 in Leigh syndrome participants that participated in previous EPI743-12-002 (NCT01721733) study.
Detailed description
To monitor the long-term safety and neurodevelopmental effects of EPI-743 on children who complete the EPI743-12-002 placebo-controlled trial.
Conditions
Interventions
| Type | Name | Description |
|---|---|---|
| DRUG | EPI-743 | EPI-743 100 milligrams/milliliter (mg/mL) oral solution will be administered per dose and schedule specified in the arm. |
Timeline
- Start date
- 2014-01-31
- Primary completion
- 2023-10-30
- Completion
- 2023-10-30
- First posted
- 2015-02-02
- Last updated
- 2024-10-23
Locations
4 sites across 1 country: United States
Source: ClinicalTrials.gov record NCT02352896. Inclusion in this directory is not an endorsement.