Hope for Friedreich's ataxia: new drug shows promise in major trial
NCT ID NCT02255435
First seen Nov 01, 2025 · Last updated May 09, 2026 · Updated 24 times
Summary
This study tested a drug called RTA 408 (omaveloxolone) in 172 people with Friedreich's ataxia, a rare nerve disease that affects movement and coordination. The main goals were to see if the drug is safe and if it can improve exercise ability and daily function. Participants took either the drug or a placebo for up to 48 weeks, and some could continue taking the drug in an extension period.
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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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Contacts and locations
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Locations
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
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Emory University Hospital - Neurology
Atlanta, Georgia, 30329, United States
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Medical University Innsbruck
Innsbruck, 6020, Austria
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Murdoch Childrens Research Institute
Parkville, Victoria, 3052, Australia
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Neurological Institute Carlo Besta
Milan, 20133, Italy
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Ohio State University - Neurology
Columbus, Ohio, 43221, United States
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UCLA
Los Angeles, California, 90095, United States
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USF Ataxia Research Center
Tampa, Florida, 33612, United States
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University College of London
London, WC1E 6BT, United Kingdom
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University of Florida - Neurology
Gainesville, Florida, 32610, United States
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University of Iowa Stead Family Children's Hospital
Iowa City, Iowa, 52242, United States
Conditions
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