Can a new drug slow friedreich ataxia? Long-Term trial underway
NCT ID NCT05515536
First seen Jun 24, 2026 · Last updated Jun 27, 2026 · Updated 2 times
Summary
This study is testing the long-term safety of a drug called vatiquinone in 130 people with Friedreich ataxia, a rare genetic disease that affects movement and coordination. All participants have taken vatiquinone before in earlier studies. Researchers will track side effects and measure changes in disease severity over three years.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
vatiquinone
What this could lead to
If successful, this could show that vatiquinone is safe and may help slow the progression of Friedreich ataxia over years.
What could go wrong
This is an open-label study with no placebo group, so results may be less reliable. It only includes people who already tolerated the drug, which may not reflect the broader population.
Disclaimer
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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.
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