New daily shot aims to slow rare nerve disease

NCT ID NCT06447025

First seen Apr 04, 2026 · Last updated May 07, 2026 · Updated 7 times

Summary

This study is testing the long-term safety and effects of a daily injection called CTI-1601 in people with Friedreich's ataxia, a rare genetic disorder that damages the nervous system. About 85 adults and teens who have or haven't taken the drug before will receive it for an extended period. Researchers will monitor side effects, heart function, movement, and daily living skills to see if the treatment helps control the disease over time.

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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.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Children's Hospital of the University of Pennsylvania (CHOP)

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

  • Clinilabs Drug Development, Corp.

    RECRUITING

    Eatontown, New Jersey, 07724, United States

  • Fixel Institute for Neurological Disease, University of Florida Health

    ACTIVE_NOT_RECRUITING

    Gainesville, Florida, 32608, United States

  • Morsani Center for Advanced Health Care, University of South Florida Health

    RECRUITING

    Tampa, Florida, 33612, United States

  • Ohio State University United States

    ACTIVE_NOT_RECRUITING

    Columbus, Ohio, 43210, United States

  • Uncommon Cures

    RECRUITING

    Chevy Chase, Maryland, 20815, United States

  • University of California Los Angeles

    RECRUITING

    Los Angeles, California, 90095, United States

  • University of Iowa

    RECRUITING

    Iowa City, Iowa, 52242, United States

Conditions

Explore the condition pages connected to this study.