New drug aims to slash stubborn cholesterol in rare genetic disease
NCT ID NCT05217667
First seen Nov 01, 2025 · Last updated May 17, 2026 · Updated 27 times
Summary
This study tests an experimental drug called ARO-ANG3 in 18 adults with homozygous familial hypercholesterolemia (HoFH), a rare genetic condition causing dangerously high cholesterol. Participants receive two doses of the drug and are monitored for 36 weeks to see if it safely lowers their LDL ("bad") cholesterol. Those who complete the main study may continue treatment for up to 24 more months.
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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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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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