First trial for young kids with rare neurological disease

NCT ID NCT06054893

Summary

This study aims to find the right dose and check the safety of the drug omaveloxolone (SKYCLARYS®) in children and teenagers aged 2 to 15 with Friedreich's ataxia. The drug is already approved for people 16 and older, but researchers need to understand how younger bodies process it. The study will involve about 35 participants and will track their health for up to 240 weeks to monitor for side effects and see how the drug works over time.

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

    Email: •••••@•••••

  • Contact

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

Locations

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

    Contact

    Contact Phone: •••-•••-••••

Conditions

Explore the condition pages connected to this study.