Children's trial launches for Friedreich's ataxia drug

NCT ID NCT06054893

Ongoing Disease control Sponsor: Biogen Source: ClinicalTrials.gov ↗

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

Summary

This study tests omaveloxolone, a drug already approved for adults with Friedreich's ataxia, in children aged 2 to 15. Researchers want to see how the drug moves through children's bodies and whether it is safe. The study involves two parts: a single dose followed by daily dosing, with regular check-ups for up to 240 weeks.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance: omaveloxolone (also known as SKYCLARYS)
What this could lead to: If successful, this study could help determine safe dosing for children, potentially leading to a treatment that slows disease progression in younger patients.
What could go wrong: This is an early phase 1 study with only 33 participants, so results may not apply to all children. The drug is already approved for adults, but its effects in growing children are unknown and side effects are possible.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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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.

FRIEDREICH ATAXIA

Contacts and locations

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Locations

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

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