Experimental drug for rare epilepsy disorders fails to complete trial

NCT ID NCT04378075

First seen Apr 03, 2026 · Last updated May 01, 2026 · Updated 4 times

Summary

This study tested a drug called vatiquinone in 68 people with mitochondrial disease who have hard-to-control seizures. Participants were randomly assigned to receive either the drug or a placebo for 24 weeks, with an option to continue the drug afterward. The trial was stopped early, so results are limited. The goal was to see if vatiquinone could reduce seizure frequency and hospital stays.

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Contacts and locations

Locations

  • A.P.H.P - Hôpital Necker-Enfants Malades - Service de Neurologie pédiatrique

    Paris, 75015, France

  • Akron Children's Hospital

    Akron, Ohio, 44308, United States

  • Alberta Children's Hospital, University of Calgary

    Calgary, T3B 6A8, Canada

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • Boston Children Hospital

    Boston, Massachusetts, 02115, United States

  • CHU d'Angers - Service de génétique

    Angers, 49933, France

  • CHU de Montpellier - Hôpital Gui de Chauliac - Département de neuropédiatrie

    Montpellier, 34295, France

  • CHU de Strasbourg - Hôpital de Hautepierre - Service de Neuropédiatrie

    Strasbourg, 67200, France

  • Children's National Medical Center - Department Of Neurology

    Washington D.C., District of Columbia, 20010, United States

  • Children's of Minnesota

    Minneapolis, Minnesota, 55404, United States

  • Great Ormond Street Hospital for Children NHS Foundation Trust

    London, WC1N 3JH, United Kingdom

  • Hospital Ruber Internacional, Neurology Department, Epilepsy Program

    Madrid, 28034, Spain

  • Hospital Sant Joan de Déu

    Barcelona, 08950, Spain

  • Hospital Universitario 12 de Octubre

    Madrid, 28041, Spain

  • Instytut Pomnik-Centrum Zdrowia Dziecka, Centrum Wsparacia Pediatrycznych Badań Klinicznych

    Warsaw, 04-730, Poland

  • John Hopkins Medicine

    Baltimore, Maryland, 21287, United States

  • Karolinska University hospital, Astrid Lindgrens Children Hospital

    Stockholm, S-171 76, Sweden

  • PTC Clinical Site

    Multiple Locations, Japan

  • Pediatric Genetics Clinic (Main MGH Hospital)

    Boston, Massachusetts, 02114-2696, United States

  • Seattle Children's hospital

    Seattle, Washington, 98105, United States

  • Stanford University

    Stanford, California, 94305, United States

  • The Newcastle Upon Tyne Hospitals NHS Foundation Trust

    Newcastle upon Tyne, NE1 4LP, United Kingdom

  • U.O.C. Malattie Muscolari e Neurodegenerative, Dipartimento di Scienze Neurologiche e Psichiatriche, Ospedale Pediatrico Bambino Gesù

    Roma, 00165, Italy

  • UOC Neuropsichiatria Infantile, Istituto Neurologico Carlo Besta-Fondazione IRCCS

    Milan, 20133, Italy

  • University of California

    San Diego, California, 92123, United States

  • University of Texas Health Science

    Houston, Texas, 77030, United States

  • Yale School of Medicine

    New Haven, Connecticut, 06520, United States

Conditions

Explore the condition pages connected to this study.