Familial chylomicronemia syndrome
MONDO:0018637A rare autosomal recessive disease characterized by the buildup in the blood of fat particles called chylomicrons (chylomicronemia), severe hypertriglyceridemia, and the risk of recurrent and potentially fatal pancreatitis and other complications. It is caused by mutations in the gene encoding LPL or, less frequently, by mutations in genes encoding other proteins necessary for LPL function.
12 clinical trials for this condition and its sub-types.
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Broader categories
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New shot could tame rare 'Fat Blood' disease and prevent pancreatitis
Disease control CompletedThis Phase 3 study tested a drug called plozasiran (ARO-APOC3) in 75 adults with familial chylomicronemia syndrome, a rare genetic disorder causing extremely high blood fat levels and risk of pancreatitis. Participants received four injections of the drug or a placebo over severa…
Phase: PHASE3 • Sponsor: Arrowhead Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 13:03 UTC
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New injection shows promise for rare High-Fat disease
Disease control CompletedThis phase 3 study tested a drug called Plozasiran in 37 Chinese adults with Familial Chylomicronemia Syndrome (FCS), a rare condition causing extremely high blood fats. The trial compared the drug to a placebo over about two years, measuring how much it lowered triglyceride leve…
Phase: PHASE3 • Sponsor: Visirna Therapeutics HK Limited • Aim: Disease control
Last updated Jun 27, 2026 09:08 UTC
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New drug targets dangerous fat levels in blood
Disease control CompletedThis early-stage study tested a new medicine called ARO-APOC3 in healthy volunteers and people with severely high triglycerides or a rare genetic condition called familial chylomicronemia syndrome. The main goal was to check safety and how the drug moves through the body. The stu…
Phase: PHASE1 • Sponsor: Arrowhead Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 08:06 UTC