Experimental drug for rare cholesterol disease shows promise but study halted early
NCT ID NCT05217667
First seen Nov 01, 2025 · Last updated May 22, 2026 · Updated 29 times
Summary
This study tested an experimental drug called ARO-ANG3 in 18 people with homozygous familial hypercholesterolemia (HoFH), a rare genetic condition causing extremely high cholesterol and early heart disease. Participants received two doses of the drug and were monitored for 36 weeks, with an optional 24-month extension. The goal was to lower LDL cholesterol levels. However, the study was terminated early, so results are limited.
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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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Contacts and locations
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Locations
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Research Site 1
Québec, Quebec, G1V 4W2, Canada
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Research Site 2
Chicoutimi, Quebec, G7H 7K9, Canada
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Research Site 3
Nedlands, Western Australia, 6009, Australia
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Research Site 4
Mount Sinai, New York, 10029, United States
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Research Site 5
Cincinnati, Ohio, 45227, United States
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Research Site 7
Johannesburg, 2193, South Africa
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Research Site 8
Camperdown, New South Wales, 2050, Australia
Conditions
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