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Can a missing protein be replaced to slow Friedreich's ataxia?

NCT ID NCT07778836

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

First seen Aug 21, 2026 · Last updated Aug 21, 2026

Summary

This phase 3 trial is testing whether a weekly injection of nomlabofusp can improve balance and slow the progression of Friedreich's ataxia, a genetic condition that damages the nervous system. The study includes about 150 adults and children with the disease, who will receive either the drug or a placebo for 72 weeks. The main goal is to see if the treatment improves scores on a standard scale that measures coordination and stability.

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), a protein designed to replace the frataxin that is missing in Friedreich's ataxia
What this could lead to
If it works, this could become the first treatment to address the underlying protein deficiency in Friedreich's ataxia, potentially slowing disease progression and improving balance.
What could go wrong
This is a phase 3 trial, but success is not guaranteed. The treatment may not improve symptoms enough to matter, and side effects from the subcutaneous injections 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 FRIEDREICH'S ATAXIA

Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

Locations

  • Clinilabs

    RECRUITING

    Eatontown, New Jersey, 07724, United States

  • Northwestern Medical Group, Department of Neurology

    RECRUITING

    Chicago, Illinois, 60611, United States

  • University of Texas Southwestern Medical Center

    RECRUITING

    Dallas, Texas, 75390, United States

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