New drug shows promise for rare movement disorder

NCT ID NCT02255435

First seen Nov 01, 2025 · Last updated May 20, 2026 · Updated 26 times

Summary

This study tested a drug called RTA 408 (omaveloxolone) in 172 people with Friedreich's ataxia, a rare genetic disease that affects movement and coordination. The goal was to see if the drug is safe and can improve exercise ability and daily function. Participants took the drug or a placebo daily for up to 48 weeks, with some continuing in an extension period.

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Contacts and locations

Locations

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Emory University Hospital - Neurology

    Atlanta, Georgia, 30329, United States

  • Medical University Innsbruck

    Innsbruck, 6020, Austria

  • Murdoch Childrens Research Institute

    Parkville, Victoria, 3052, Australia

  • Neurological Institute Carlo Besta

    Milan, 20133, Italy

  • Ohio State University - Neurology

    Columbus, Ohio, 43221, United States

  • UCLA

    Los Angeles, California, 90095, United States

  • USF Ataxia Research Center

    Tampa, Florida, 33612, United States

  • University College of London

    London, WC1E 6BT, United Kingdom

  • University of Florida - Neurology

    Gainesville, Florida, 32610, United States

  • University of Iowa Stead Family Children's Hospital

    Iowa City, Iowa, 52242, United States

Conditions

Explore the condition pages connected to this study.