New drug aims to tame hard-to-control seizures in rare mitochondrial disorders
NCT ID NCT04378075
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested a drug called vatiquinone in 68 people with mitochondrial disease and epilepsy that doesn't respond to standard treatments. Participants were randomly assigned to receive either vatiquinone or a placebo for 24 weeks to see if the drug could reduce the number of visible seizures. The study was stopped early, but the goal was to find a new way to help control seizures in these rare, serious conditions.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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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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A.P.H.P - Hôpital Necker-Enfants Malades - Service de Neurologie pédiatrique
Paris, 75015, France
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Akron Children's Hospital
Akron, Ohio, 44308, United States
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Alberta Children's Hospital, University of Calgary
Calgary, T3B 6A8, Canada
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Baylor College of Medicine
Houston, Texas, 77030, United States
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Boston Children Hospital
Boston, Massachusetts, 02115, United States
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CHU d'Angers - Service de génétique
Angers, 49933, France
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CHU de Montpellier - Hôpital Gui de Chauliac - Département de neuropédiatrie
Montpellier, 34295, France
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CHU de Strasbourg - Hôpital de Hautepierre - Service de Neuropédiatrie
Strasbourg, 67200, France
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Children's National Medical Center - Department Of Neurology
Washington D.C., District of Columbia, 20010, United States
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Children's of Minnesota
Minneapolis, Minnesota, 55404, United States
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Great Ormond Street Hospital for Children NHS Foundation Trust
London, WC1N 3JH, United Kingdom
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Hospital Ruber Internacional, Neurology Department, Epilepsy Program
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, 28041, Spain
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Instytut Pomnik-Centrum Zdrowia Dziecka, Centrum Wsparacia Pediatrycznych Badań Klinicznych
Warsaw, 04-730, Poland
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John Hopkins Medicine
Baltimore, Maryland, 21287, United States
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Karolinska University hospital, Astrid Lindgrens Children Hospital
Stockholm, S-171 76, Sweden
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PTC Clinical Site
Multiple Locations, Japan
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Pediatric Genetics Clinic (Main MGH Hospital)
Boston, Massachusetts, 02114-2696, United States
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Seattle Children's hospital
Seattle, Washington, 98105, United States
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Stanford University
Stanford, California, 94305, 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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U.O.C. Malattie Muscolari e Neurodegenerative, Dipartimento di Scienze Neurologiche e Psichiatriche, Ospedale Pediatrico Bambino Gesù
Roma, 00165, Italy
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UOC Neuropsichiatria Infantile, Istituto Neurologico Carlo Besta-Fondazione IRCCS
Milan, 20133, Italy
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University of California
San Diego, California, 92123, United States
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University of Texas Health Science
Houston, Texas, 77030, United States
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Yale School of Medicine
New Haven, Connecticut, 06520, United States