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Homozygous familial hypercholesterolemia

MONDO:0018328

Also known as: HoFH, homozygous familial hypercholesterolemia

56 clinical trials for this condition and its sub-types.

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Broader categories

Disease (618) Hyperlipidemia (264) Metabolic disease (215) Hereditary disease (172) Inherited lipid metabolism disorder (165) Familial hypercholesterolemia (53) Inborn errors of metabolism (42) Human disease (14) Hyperlipoproteinemia (7) Familial hyperlipidemia (5)

Trials to join now! 24 Not yet recruiting 6 Not yet finished but already full! 8 Completed 17 Terminated 1

Experimental drug targets stubborn cholesterol in rare disease

Disease control Terminated

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 change…

Phase: PHASE2 • Sponsor: Arrowhead Pharmaceuticals • Aim: Disease control

Last updated Jun 27, 2026 13:06 UTC