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Homozygous familial hypercholesterolemia

MONDO:0018328

Also known as: HoFH, homozygous familial hypercholesterolemia

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

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Broader categories

Disease (680) Hyperlipidemia (295) Metabolic disease (233) Inherited lipid metabolism disorder (189) Hereditary disease (176) Familial hypercholesterolemia (56) Inborn errors of metabolism (45) Human disease (14) Familial hyperlipidemia (8) Hyperlipoproteinemia (8)

Trials to join now! 9 Not yet recruiting 1 Not yet finished but already full! 6 Completed 5 Terminated 1

New shot could help teens with rare, severe high cholesterol

Disease control Not yet recruiting

This study tests a new drug called zodasiran in 12 teens aged 12 to 17 with homozygous familial hypercholesterolemia, a rare genetic condition causing very high cholesterol. The drug is given as a shot under the skin. The main goal is to see if it safely lowers bad cholesterol af…

Phase: PHASE3 • Sponsor: Arrowhead Pharmaceuticals • Aim: Disease control

Last updated Jun 27, 2026 13:00 UTC