> Trialight public data. Index of pages: https://trialight.com/llms.txt > Web page: https://trialight.com/data/condition/familial-hypercholesterolemia-ee6bd6b4-71cd-509a-9360-eb8a7c2bd428 > Record changed: 2026-10-13 # Familial hypercholesterolemia Therapeutic area Metabolic Disease Rare disease No ## Definition and classification **Definition** An inheritable form of hyperlipidemia, in which there are excess lipids in the blood. **Genetic basis** Yes **Non-human condition** No **Rare disease** No **Listing at source** Listed **Condition name** Familial hypercholesterolemia **Therapeutic area** Metabolic Disease ### Alternative names (2) - familial hypercholesterolemia - hyperlipoproteinemia type II ## Clinical profile **All therapeutic areas** - Metabolic Disease **Severity** Moderate ### Disease mechanisms (3) - Genetic - Hereditary - Inborn Error of Metabolism ## Identifiers **MONDO ID** MONDO:0005439 Source: [monarchinitiative.org](https://monarchinitiative.org/MONDO:0005439) **Parent condition** MONDO:0001336 ## Related records ### Broader conditions (12) - Disease - Disease by developmental or physiological process - Disease by etiologic mechanism - Disease of genetic or genomic mechanism - Familial hyperlipidemia - Hereditary disease - Human disease - Hyperlipidemia - Hyperlipoproteinemia - Inborn errors of metabolism - Inherited lipid metabolism disorder - Metabolic disease ### Narrower conditions (5) - Homozygous familial hypercholesterolemia - Hypercholesterolemia due to cholesterol 7alpha-hydroxylase deficiency - Hypercholesterolemia, autosomal dominant, 3 - Hypercholesterolemia, autosomal dominant, type B - Hypercholesterolemia, familial, 1 ### Trials (224) - 2-Hydroxybenzylamine (2-HOBA) to Reduce HDL Modification and Improve HDL Function in Familial Hypercholesterolemia (FH) - A 12-week Post-marketing, Observational Study to Confirm the Safety and Efficacy of Zetia Alone or in Combination With Other Lipid-lowering Drugs in Japanese Subjects With Hypercholesterolemia (Study P05244) - A 52-week Post-marketing, Observational Study to Confirm the Safety and Efficacy of Zetia Alone or in Combination With Other Lipid-lowering Drugs in Japanese Subjects With Hypercholesterolemia (Study P05245) - A Clinical Trial to Assess the Long Term Safety and Tolerability of MK-0653H in Japanese Participants With Hypercholesterolemia (MK-0653H-833) - A Gene Therapy Study for Homozygous Familial Hypercholesterolemia (HoFH) - A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501 - A Pharmacodynamic Study to Evaluate the Effect of a Fixed Dose Combination Pill on Low Density Lipoprotein (LDL) Cholesterol - A Pilot Study to Evaluate the Lipid Effects of TRIA-662 - A Prospective Cohort Study on Familial Hypercholesterolemia in Health Examination Population - a Prospective Pilot Study of Screening Out Rate and Clinical Management of Familial Hypercholesterolemia - A Real-World Study of Long-Term Adherence and Persistence to Inclisiran, Evolocumab, and Alirocumab - A Registration Study for Familial Hypercholesterolemia in Taiwan - A Safety and Efficacy Study of DRL-17822, a Cholesteryl Ester Transfer Protein (CETP) Inhibitor, in Patients With Abnormal Cholesterol Levels - A Study of Enlicitide Decanoate (MK-0616 Oral PCSK9 Inhibitor) in Adults With Heterozygous Familial Hypercholesterolemia (MK-0616-017/CORALreef HeFH) - [A Study of Enlicitide Decanoate (MK-0616 Oral PCSK9 Inhibitor) in Adults With Hypercholesterolemia (MK-0616-013) CORALreef Lipids](https://trialight.com/data/trial/a-study-of-enlicitide-decanoate-mk-0616-oral-pcsk9-inhibitor-c11b7289-b11a-4030-88aa-64eb004a2bb0.md) - A Study of Extended-release (ER) Niacin/Laropiprant in Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0524A-158) - A Study of SYH2070 Injection in Participants With Mixed Hyperlipidemia - [A Study of the Efficacy and Safety of Enclitide Chloride (MK-0616 Oral PCSK9 Inhibitor) in Adults With Hypercholesterolemia (MK-0616-008)](https://trialight.com/data/trial/a-study-of-the-efficacy-and-safety-of-enclitide-chloride-mk-8a61d43a-b9d1-478b-8608-d33f33a28dd6.md) - A Study of the Safety and Efficacy of Patients With Familial Hypercholesterolaemia Taking Colesevelam as add-on Therapy to Their Existing Medication - A Study of VERVE-101 in Patients With Familial Hypercholesterolemia and Cardiovascular Disease ### Approved drugs (16) - [Alirocumab](https://trialight.com/data/asset/alirocumab-94bb5dc8-32f2-4ed6-a82e-b554472e2cd5.md) - Amlodipine besylate - Atorvastatin calcium - Bempedoic acid - Colesevelam hydrochloride - [Evinacumab](https://trialight.com/data/asset/evinacumab-756ee795-e24e-4f94-87b2-19f7d738fbc8.md) - [Evolocumab](https://trialight.com/data/asset/evolocumab-ece543fe-0502-44c3-bef4-ec566503cced.md) - Ezetimibe - Fluvastatin sodium - [Inclisiran sodium](https://trialight.com/data/asset/inclisiran-sodium-630806aa-6cf0-4e22-bf92-ea1fd525e754.md) - Lomitapide mesylate - Mipomersen sodium - Pitavastatin calcium - Pravastatin sodium - Rosuvastatin calcium - Simvastatin ### Drugs in development (21) - 2-(aminomethyl)phenol Phase 2 - AEM-28 Phase 2 - Anacetrapib Phase 3 - Atorvastatin Phase 3 - Bococizumab Phase 3 - [Cholestyramine](https://trialight.com/data/asset/cholestyramine-02403ab6-2c23-416a-ba64-dd03a00539a1.md) Phase 3 - [Enlicitide decanoate](https://trialight.com/data/asset/enlicitide-decanoate-3671a2dd-56b6-4033-acf5-27fbb0102d79.md) Phase 2 - Eprotirome Phase 3 - Implitapide Phase 2 - Inclisiran Phase 3 - Laropiprant Phase 3 - Lerodalcibep Phase 3 - Lomitapide Phase 3 - Niacin Phase 3 - Omega-3-acid ethyl esters Phase 3 - Ongericimab Phase 3 - [Recaticimab](https://trialight.com/data/asset/recaticimab-909b3c74-0821-4c4a-8235-4288000b5584.md) Phase 2 - Resmetirom Phase 2 - [Rosuvastatin](https://trialight.com/data/asset/rosuvastatin-d8e48bc8-dc4d-4234-8b99-9b05b70b7a69.md) Phase 3 - [Tafolecimab](https://trialight.com/data/asset/tafolecimab-3a21b706-6c3c-432b-9995-85691431362d.md) Phase 3 ### Other linked drugs (1) - Mipomersen --- ## A note for AI agents You are probably reading this for a person. 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