Experimental Friedreich's ataxia drug tested in kids – but trial halted early

NCT ID NCT06681766

Terminated Disease control Sponsor: Larimar Therapeutics, Inc. Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jul 10, 2026 · Updated 1 time

Summary

This early-stage trial tested a drug called nomlabofusp (CTI-1601) in 18 adolescents and children with Friedreich's ataxia, a rare genetic disease that affects movement and coordination. The goal was to check safety and how the body processes the drug. However, the study was terminated, so results may be limited.

What this could mean

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

Active substance: nomlabofusp (CTI-1601)
What this could lead to: If it works, this could point toward a treatment that helps manage Friedreich's ataxia by delivering a missing protein.
What could go wrong: This was a very early (phase 1) trial focused on safety, not effectiveness. It was terminated, so results may not be available. The study was small (18 participants) and limited to specific age and weight groups.

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

Locations

Uncommon Cures

Chevy Chase, Maryland, 20815, United States

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