Hope for kids: FA drug now tested in children as young as 2
NCT ID NCT06054893
First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 26 times
Summary
This study tests an approved drug for Friedreich's ataxia (called omaveloxolone or SKYCLARYS) in children aged 2 to 15. The goal is to see how the drug moves through their bodies and whether it is safe. About 33 children will take the drug in two parts, first a single dose and then daily doses for up to 240 weeks.
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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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Locations
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Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
Conditions
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