Experimental drug targets stubborn cholesterol in rare disease

NCT ID NCT05217667

Terminated Disease control Sponsor: Arrowhead Pharmaceuticals Source: ClinicalTrials.gov ↗

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

homozygous familial hypercholesterolemia

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HOMOZYGOUS FAMILIAL HYPERCHOLESTEROLEMIA

Contacts and locations

Locations

Research Site 1

Québec, Quebec, G1V 4W2, Canada
Research Site 2

Chicoutimi, Quebec, G7H 7K9, Canada
Research Site 3

Nedlands, Western Australia, 6009, Australia
Research Site 4

Mount Sinai, New York, 10029, United States
Research Site 5

Cincinnati, Ohio, 45227, United States
Research Site 7

Johannesburg, 2193, South Africa
Research Site 8

Camperdown, New South Wales, 2050, Australia

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