Experimental drug shows promise for rare genetic disorder
NCT ID NCT03759678
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.
Disclaimer
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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.
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.
More trials for these conditions
Other studies related to the condition(s) this trial covers.
Contacts and locations
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Locations
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Hospital Universitario La Paz
Madrid, Spain, Spain
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Royal Papworth Hospital NHS Foundation Trust
Cambridge, Cambridgeshire, CB2 0AY, United Kingdom
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University of California - Los Angeles
Los Angeles, California, 90095, United States
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University of Giessen
Giessen, Germany