A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy
Public ClinicalTrials.gov record NCT04378075. 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
Efficacy and Safety Study of Vatiquinone for the Treatment of Mitochondrial Disease Subjects With Refractory Epilepsy
Study identification
- NCT ID
- NCT04378075
- Recruitment status
- Terminated
- Study type
- Interventional
- Phase
- Phase 2, Phase 3
- Lead sponsor
- PTC Therapeutics
- Industry
- Enrollment
- 68 participants
Conditions and interventions
Conditions
Interventions
- Placebo Other
- Vatiquinone Drug
Other · Drug
Eligibility (public fields only)
- Age range
- Up to 20 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
- Sep 27, 2020
- Primary completion
- Mar 17, 2023
- Completion
- Dec 26, 2023
- Last update posted
- Mar 30, 2026
2020 – 2023
United States locations
- U.S. sites
- 12
- U.S. states
- 9
- U.S. cities
- 10
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California | San Diego | California | 92123 | — |
| Stanford University | Stanford | California | 94305 | — |
| Yale School of Medicine | New Haven | Connecticut | 06520 | — |
| Children's National Medical Center - Department Of Neurology | Washington D.C. | District of Columbia | 20010 | — |
| John Hopkins Medicine | Baltimore | Maryland | 21287 | — |
| Pediatric Genetics Clinic (Main MGH Hospital) | Boston | Massachusetts | 02114-2696 | — |
| Boston Children Hospital | Boston | Massachusetts | 02115 | — |
| Children's of Minnesota | Minneapolis | Minnesota | 55404 | — |
| Akron Children's Hospital | Akron | Ohio | 44308 | — |
| Baylor College of Medicine | Houston | Texas | 77030 | — |
| University of Texas Health Science | 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.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 15 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04378075, 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 Mar 30, 2026 · Synced May 20, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04378075 live on ClinicalTrials.gov.