Experimental drug targets stubborn cholesterol in rare disease
NCT ID NCT05217667
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
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. Participants received injections of the drug and were monitored for safety and cholesterol changes over 36 weeks, with an optional longer extension. The trial was terminated early, so its findings 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
ARO-ANG3 injection
What this could lead to
If successful, this could point toward a new way to lower dangerously high cholesterol in people with a rare genetic condition.
What could go wrong
This early trial was terminated, so results are limited. It involved only 18 people, and the drug may not work or may have side effects.
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.
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