Experimental Friedreich's ataxia drug tested in kids – but trial halted early
NCT ID NCT06681766
First seen Jun 27, 2026 · Last updated Jun 27, 2026
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.
Disclaimer
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the original study
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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.
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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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Locations
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Uncommon Cures
Chevy Chase, Maryland, 20815, United States