Homozygous familial hypercholesterolemia
MONDO:0018328Also known as: HoFH, homozygous familial hypercholesterolemia
22 clinical trials for this condition and its sub-types.
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New registry tracks Real-World safety of cholesterol drug lomitapide
Disease control CompletedThis observational registry followed 260 patients with homozygous familial hypercholesterolemia (a severe genetic condition causing very high cholesterol) who were taking lomitapide. The goal was to monitor long-term safety, especially liver problems and gastrointestinal issues, …
Sponsor: Amryt Pharma • Aim: Disease control
Last updated Jun 27, 2026 12:32 UTC
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New shot could slash Sky-High cholesterol in teens with rare gene condition
Disease control CompletedThis study tested a drug called inclisiran in teenagers aged 12 to 18 who have a rare inherited condition that causes extremely high cholesterol. The goal was to see if the drug safely lowers their LDL (bad) cholesterol. Participants received either inclisiran or a placebo for on…
Phase: PHASE3 • Sponsor: Novartis Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 08:10 UTC
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New drug SHR-1918 targets stubborn high cholesterol in rare disease
Disease control CompletedThis Phase 3 trial tested the drug SHR-1918 in 55 people with homozygous familial hypercholesterolemia (HoFH), a rare genetic condition causing extremely high cholesterol. Participants received either the drug or a placebo, and researchers measured how much their LDL (bad) choles…
Phase: PHASE3 • Sponsor: Beijing Suncadia Pharmaceuticals Co., Ltd • Aim: Disease control
Last updated Jun 27, 2026 08:04 UTC
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New drug shows promise for rare High-Cholesterol disease
Disease control CompletedThis phase 2 trial tested an investigational drug called SHR-1918 in 26 people with homozygous familial hypercholesterolemia (HoFH), a rare genetic condition causing extremely high cholesterol. The study measured how much the drug lowered LDL (bad) cholesterol after 12 weeks. All…
Phase: PHASE2 • Sponsor: Beijing Suncadia Pharmaceuticals Co., Ltd • Aim: Disease control
Last updated Jun 26, 2026 13:33 UTC
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Rare cholesterol disease study aims to detect heart risks early
Knowledge-focused CompletedThis study looks at people with a rare inherited condition that causes extremely high cholesterol and early heart disease. Researchers used special tests to measure artery hardening before it becomes life-threatening. The goal was to gather information to help develop future trea…
Sponsor: National Heart, Lung, and Blood Institute (NHLBI) • Aim: Knowledge-focused
Last updated Aug 09, 2026 00:00 UTC