Rare condition

Homozygous familial hypercholesterolemia

11

studies recruiting now

as of 7 Sept 2026

85

studies registered in total

as of 7 Sept 2026

15

countries with a recruiting site

as of 7 Sept 2026

15 May 2026

most recent study posted

among recruiting studies

Recruiting trials

Showing the 5 most recently updated recruiting studies, as recorded 7 Sept 2026. Live status on each study page.

See all 11 recruiting studies

Where recruiting studies are running

Countries with at least one recruiting site among the studies above, 7 Sept 2026. Tap a country to search trials there.

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Patient organisations

We do not yet list a dedicated organisation for this condition. The directories below are the best route.

About Homozygous familial hypercholesterolemia

RareTrial does not yet hold a plain-language description of this condition. The most reliable starting point is Orphanet’s expert page, which lists specialist centres, registries and patient organisations, and the genes involved (ABCG5, ABCG8, APOB, LDLRAP1, PCSK9, LDLR).

Treatments being studied

2 approved treatments and 11 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.

Approved: Evinacumab (Evkeeza)Approved: Evolocumab (Repatha)
Phase 3Rosuvastatin
Phase 3Lerodalcibep
Phase 3Zodasiran
Phase 3Inclisiran
Phase 3Alirocumab (Praluent)
Phase 3Lomitapide (Lojuxta)
Phase 3Lomitapide Mesylate (Juxtapid)
Phase 2/3Tafolecimab

+ 3 more in development

Related conditions

Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).