NCT05218655CompletedPhase 3
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease
An Open-Label, Safety Study for Previously Treated Vatiquinone (PTC743) Subjects With Inherited Mitochondrial Disease
Source: ClinicalTrials.gov · Last updated 22 Dec 2025
Brief summary (registry, in English)
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.
- Status (registry)
- Completed
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 101 (actual)
- First submitted
- 19 Jan 2022
- Start date
- 22 Jun 2022
- Primary completion date
- 15 Apr 2025
- Lead sponsor
- PTC Therapeutics
- Diseases
- Epilepsy
- NCT number
- NCT05218655
- EudraCT number
- 2022-000375-39
What you commit to
- Study type: Interventional · Phase 3
- Participants enrolled: 101
- From 22 Jun 2022 to the primary completion date, 15 Apr 2025
- 26 sites in 7 countries
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other
- Current participation in any other interventional study.
- Pregnancy or breast feeding.
Who the study is looking for
- Participants with inherited mitochondrial disease including Leigh syndrome, Alpers syndrome, mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS), myoclonic epilepsy with ragged-red fibers (MERRF), pontocerebellar hypoplasia type 6 (PCH6), or other mitochondrial disease who participated in a previous vatiquinone clinical study or treatment plan.
- Women of childbearing potential must have a negative pregnancy test at screening/baseline and agree to abstinence or the use of at least 1 of the highly effective forms of contraception as specified in the protocol (with a failure rate of \<1% per year when used consistently and correctly). Highly effective contraception or abstinence must be continued for the duration of the study, and for up to 30 days after the last dose of study drug.
- Fertile men who are sexually active with women of childbearing potential and who have not had a vasectomy, must agree to use a barrier method of birth control during the study and for up to 30 days after the last dose of study drug.
Sex: All · Does not accept healthy volunteers
Sites (26)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Hospital Sant Joan de Déu Coordinator of this site? Confirm its status | Barcelona | Spain | Unknown status |
| Hospital Universitario 12 de Octubre Coordinator of this site? Confirm its status | Madrid | Spain | Unknown status |
| Hospital Ruber Internacional Coordinator of this site? Confirm its status | Madrid | Spain | Unknown status |
| CHU Angers Coordinator of this site? Confirm its status | Angers | France | Unknown status |
| CHU Montpellier - Hôpital Saint Eloi Coordinator of this site? Confirm its status | Montpellier | France | Unknown status |
| Hopital Necker Enfants Malades Coordinator of this site? Confirm its status | Paris | France | Unknown status |
| Hôpital de Hautepierre - Hôpitaux Universitaires de Strasbourg Coordinator of this site? Confirm its status | Strasbourg | France | Unknown status |
| Great Ormond Street Hospital For Children NHS Foundation Trust Coordinator of this site? Confirm its status | London | United Kingdom | Unknown status |
| The Newcastle Upon Tyne Hospitals NHS Foundation Trust Coordinator of this site? Confirm its status | Newcastle upon Tyne | United Kingdom | Unknown status |
| Ospedale Pediatrico Bambino Gesù Coordinator of this site? Confirm its status | Roma | Italy | Unknown status |
| PTC Clinical Site Coordinator of this site? Confirm its status | Japanese City | Japan | Unknown status |
| Instytut Pomnik Centrum Zdrowia Dziecka Coordinator of this site? Confirm its status | Warsaw | Poland | Unknown status |
| Akron Children's Hospital Coordinator of this site? Confirm its status | Akron | United States | Unknown status |
| The Johns Hopkins University Coordinator of this site? Confirm its status | Baltimore | United States | Unknown status |
| Medical University of South Carolina (MUSC) Coordinator of this site? Confirm its status | Charleston | United States | Unknown status |
| Cleveland Clinic Coordinator of this site? Confirm its status | Cleveland | United States | Unknown status |
| Child Neurology Center of Northwest Florida Coordinator of this site? Confirm its status | Gulf Breeze | United States | Unknown status |
| UT Health The University of Texas Coordinator of this site? Confirm its status | Houston | United States | Unknown status |
| Baylor College of Medicine Coordinator of this site? Confirm its status | Houston | United States | Unknown status |
| University of California, San Diego Altman Clinical and Translational Research Institute Coordinator of this site? Confirm its status | La Jolla | United States | Unknown status |
| Yale Medicine Coordinator of this site? Confirm its status | New Haven | United States | Unknown status |
| Columbia University Medical Center - CUMC Coordinator of this site? Confirm its status | New York | United States | Unknown status |
| Children's Hospital of Philadelphia (CHOP) Coordinator of this site? Confirm its status | Philadelphia | United States | Unknown status |
| Seattle Children Hospital Coordinator of this site? Confirm its status | Seattle | United States | Unknown status |
| Stanford University Coordinator of this site? Confirm its status | Stanford | United States | Unknown status |
| Children's National Coordinator of this site? Confirm its status | Washington D.C. | United States | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.