Early-Stage trial for Friedreich's ataxia drug in kids halted

NCT ID NCT06681766

First seen Jan 28, 2026 · Last updated May 14, 2026 · Updated 15 times

Summary

This early-stage study aimed to check the safety of a drug called nomlabofusp in 18 children and teenagers (ages 2 to 17) with Friedreich's ataxia, a rare genetic disease that affects movement and heart function. The drug was given as a shot under the skin. The study was stopped early, so results are limited.

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Contacts and locations

Locations

  • Uncommon Cures

    Chevy Chase, Maryland, 20815, United States

Conditions

Explore the condition pages connected to this study.