Experimental enzyme therapy targets rare kidney-damaging cholesterol disorder
NCT ID NCT04737720
First seen Nov 01, 2025 · Last updated May 07, 2026 · Updated 21 times
Summary
This study tested an artificial version of a missing enzyme (ACP-501) in one person with familial LCAT deficiency, a rare genetic condition that causes low good cholesterol and kidney failure. The participant received several doses of the drug and was monitored with blood tests and clinic visits. The goal was to see if the treatment could raise good cholesterol and slow kidney damage.
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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.
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Locations
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National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States
Conditions
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