Experimental drug trial aims to tame devastating seizures in rare genetic disorders
NCT ID NCT04378075
Summary
This study tested whether vatiquinone could reduce seizure frequency in people with mitochondrial diseases who have epilepsy that doesn't respond well to standard medications. It involved 68 participants who were randomly assigned to receive either the drug or a placebo for 24 weeks, followed by an optional longer-term treatment period. The main goal was to see if vatiquinone could lower the number of observable motor seizures and reduce hospital visits related to the disease.
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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
Conditions
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