A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
Public ClinicalTrials.gov record NCT05218655. 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
An Open-Label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
The primary objective of this study is to assess the safety of vatiquinone in participants with inherited mitochondrial disease who had prior exposure to vatiquinone in a PTC/BioElectron sponsored (previously Edison) clinical study or treatment plan. The study will continue until vatiquinone becomes commercially available or the program is terminated.
Study identification
- NCT ID
- NCT05218655
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 101 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- Not listed
- 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
- Jun 22, 2022 (Actual)
- Primary completion
- Apr 15, 2025 (Actual)
- Completion
- Apr 15, 2025 (Actual)
- Last update posted
- Dec 22, 2025
2022 – 2025
United States locations
- U.S. sites
- 14
- U.S. states
- 11
- U.S. cities
- 13
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of California, San Diego Altman Clinical and Translational Research Institute | La Jolla | California | 92037 | — |
| Stanford University | Stanford | California | 94305 | — |
| Yale Medicine | New Haven | Connecticut | 06511 | — |
| Children's National | Washington D.C. | District of Columbia | 20010 | — |
| Child Neurology Center of Northwest Florida | Gulf Breeze | Florida | 32561 | — |
| The Johns Hopkins University | Baltimore | Maryland | 21205 | — |
| Columbia University Medical Center - CUMC | New York | New York | 10032 | — |
| Akron Children's Hospital | Akron | Ohio | 44308 | — |
| Cleveland Clinic | Cleveland | Ohio | 44195 | — |
| Children's Hospital of Philadelphia - CHOP | Philadelphia | Pennsylvania | 19104 | — |
| Medical University of South Carolina - MUSC | Charleston | South Carolina | 29425 | — |
| Baylor College of Medicine | Houston | Texas | 77030 | — |
| UT Health The University of Texas | Houston | Texas | 77030 | — |
| Seattle Children 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 12 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT05218655, 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 Dec 22, 2025 · Synced Oct 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 public registry record, listed eligibility criteria, and available contact information for NCT05218655 live on ClinicalTrials.gov.