Long-Term safety of mitochondrial drug confirmed in 101 patients
NCT ID NCT05218655
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study looked at the safety of vatiquinone in 101 people with inherited mitochondrial disease who had already taken the drug in a previous study or treatment plan. The goal was to track any side effects until the drug became commercially available or the program ended. Participants had conditions like Leigh syndrome, MELAS, or other mitochondrial diseases.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Akron Children's Hospital
Akron, Ohio, 44308, United States
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Baylor College of Medicine
Houston, Texas, 77030, United States
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CHU Montpellier - Hopital Saint-Eloi
Montpellier, 34295, France
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Child Neurology Center of Northwest Florida
Gulf Breeze, Florida, 32561, United States
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Children's Hospital of Philadelphia - CHOP
Philadelphia, Pennsylvania, 19104, United States
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Children's National
Washington D.C., District of Columbia, 20010, United States
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Chu Angers
Angers, 49933, France
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Cleveland Clinic
Cleveland, Ohio, 44195, United States
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Columbia University Medical Center - CUMC
New York, New York, 10032, United States
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Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N 3JH, United Kingdom
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Hopital Necker-Enfants Malades
Paris, 75015, France
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Hospital Ruber Internacional
Madrid, 28034, Spain
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Hospital Sant Joan de Déu
Barcelona, 08950, Spain
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Hospital Universitario 12 de Octubre
Madrid, 28003, Spain
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Hôpital de Hautepierre - Hôpitaux Universitaires de Strasbourg
Strasbourg, 67000, France
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Instytut Pomnik - Centrum Zdrowia Dziecka
Warsaw, 04-730, Poland
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Medical University of South Carolina - MUSC
Charleston, South Carolina, 29425, United States
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Ospedale Pediatrico Bambino Gesù
Roma, 00165, Italy
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PTC Clinical Site
Japanese City, Japan
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Seattle Children Hospital
Seattle, Washington, 98105, United States
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Stanford University
Stanford, California, 94305, United States
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The Johns Hopkins University
Baltimore, Maryland, 21205, United States
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The Newcastle Upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, NE1 4LP, United Kingdom
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UT Health The University of Texas
Houston, Texas, 77030, United States
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University of California, San Diego Altman Clinical and Translational Research Institute
La Jolla, California, 92037, United States
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Yale Medicine
New Haven, Connecticut, 06511, United States
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