Hope for Friedreich's ataxia: new drug trial aims to slow disease progression

NCT ID NCT06447025

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study is testing the long-term safety and effectiveness of an experimental drug called CTI-1601 for people with Friedreich's ataxia, a rare genetic disease that affects movement and heart function. It includes about 85 adolescents and adults who have or have not taken the drug before. Participants receive daily injections, and researchers will monitor side effects, heart health, motor skills, and daily living abilities 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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Conditions

The condition(s) this trial relates to.

Friedreich ataxia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Study contacts

  • Contact

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

Locations

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

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

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

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

  • 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

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

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

  • Ohio State University United States

    ACTIVE_NOT_RECRUITING

    Columbus, Ohio, 43210, United States

  • Uncommon Cures

    RECRUITING

    Chevy Chase, Maryland, 20815, United States

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

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

  • University of California Los Angeles

    RECRUITING

    Los Angeles, California, 90095, United States

  • University of Iowa

    RECRUITING

    Iowa City, Iowa, 52242, United States