Extended hope for rare neurological disease

NCT ID NCT05515536

Summary

This study aims to see if the drug vatiquinone is safe and effective for people with Friedreich ataxia when taken for up to three years. It is for 130 participants who have already been taking vatiquinone in a previous trial. The main goal is to monitor for side effects while also checking if the drug helps slow the progression of the disease.

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Contacts and locations

Locations

  • CBR Neurogenetic Research Clinic, University of Auckland

    Auckland, 1023, New Zealand

  • CHU Sainte-Justine

    Montreal, Quebec, H3T1C5, Canada

  • Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)

    Montreal, Quebec, H2X 0A9, Canada

  • Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE)

    Tübingen, 72076, Germany

  • Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares

    Barcelona, 08950, Spain

  • Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute)

    Paris, 75646, France

  • Murdoch Children's Research Institute

    Parkville, Victoria, 3052, Australia

  • Ospedale Pediatrico Bambino Gesu' IRCCS

    Roma, 00165, Italy

  • The Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • UCLA

    Los Angeles, California, 90095, United States

  • University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology

    São Paulo, 13083-887, Brazil

  • University of Florida

    Gainesville, Florida, 32608, United States

  • University of Iowa

    Iowa City, Iowa, 52242, United States

  • University of South Florida

    Tampa, Florida, 33612, United States

Conditions

Explore the condition pages connected to this study.