Hope for friedreich ataxia: experimental drug vatiquinone put to the test
NCT ID NCT04577352
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tested an experimental drug called vatiquinone in 146 people with Friedreich ataxia, a rare genetic disease that affects movement and coordination. Participants took either the drug or a placebo for 72 weeks to see if it slowed worsening of symptoms, measured by a standard rating scale. The goal was to find a treatment that helps control the disease, not cure it.
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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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CBR Neurogenetic Research Clinic, University of Auckland
Auckland, 1023, New Zealand
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CHU Sainte-Justine
Montreal, Quebec, H3T1C5, Canada
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Centre de Recherche du Centre Hospitalier de l'Université de Montreal (CRCHUM)
Montreal, Quebec, H2X 0A9, Canada
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Department of Neurology and Hertie-Institute for Clinical Brain Research German Center of Neurodegenerative Diseases (DZNE)
Tübingen, 72076, Germany
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Hospital Sant Joan de Déu Barcelona Unidad de Enfermedades Neuromusculares
Barcelona, 08950, Spain
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Hôpital Pitié-Salpêtrière, Institut du Cerveau (Paris Brain Institute)
Paris, 75646, France
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Murdoch Children's Research Institute
Parkville, Victoria, 3052, Australia
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Ospedale Pediatrico Bambino Gesu' IRCCS
Roma, 00165, Italy
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The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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UCLA
Los Angeles, California, 90095, United States
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University of Campinas (UNICAMP) - School of Medical Sciences, Dept of Neurology
São Paulo, 13083-887, Brazil
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University of Florida
Gainesville, Florida, 32608, United States
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University of Iowa
Iowa City, Iowa, 52242, United States
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University of South Florida
Tampa, Florida, 33612, United States