Experimental drug shows promise for rare genetic disorder

NCT ID NCT03759678

Terminated Disease control Sponsor: IntraBio Inc Source: ClinicalTrials.gov ↗

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

Summary

This Phase II trial tested a drug called N-Acetyl-L-Leucine (IB1001) in 17 people with Ataxia-Telangiectasia, a rare genetic disease that affects movement and immunity. The study aimed to see if the drug could improve symptoms and slow the disease over time. The trial was terminated early, so results are limited.

What this could mean

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

Active substance: N-Acetyl-L-Leucine (IB1001)
What this could lead to: If this works, it could point toward a treatment to control symptoms and slow disease progression in Ataxia-Telangiectasia.
What could go wrong: This is a very early, small Phase II trial that was terminated, so results are limited. It may not show clear benefit or be safe for all patients.

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.

ataxia telangiectasia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

ATAXIA TELANGIECTASIA

Contacts and locations

Locations

Hospital Universitario La Paz

Madrid, Spain, Spain
Royal Papworth Hospital NHS Foundation Trust

Cambridge, Cambridgeshire, CB2 0AY, United Kingdom
University of California - Los Angeles

Los Angeles, California, 90095, United States
University of Giessen

Giessen, Germany

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