Hope for friedreich ataxia: new drug trial shows promise

NCT ID NCT04577352

First seen Apr 09, 2026 · Last updated May 14, 2026 · Updated 6 times

Summary

This study tested a drug called vatiquinone in 146 people with Friedreich ataxia, a rare nerve disease that affects movement and balance. The goal was to see if the drug could slow down the disease and improve daily function. Participants took the drug or a placebo for 72 weeks, and researchers measured changes using a special rating scale.

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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.