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.
13 clinical trials for this condition and its sub-types, 11 tagged with Familial chylomicronemia syndrome itself.
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Sub-types of Familial chylomicronemia syndrome
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Hyperlipoproteinemia, type 1D 0 trials
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Lipase deficiency, combined 0 trials
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Can a new shot tame a rare fat disorder in kids?
Disease control Recruiting nowThis trial tests whether olezarsen, an experimental drug given as a weekly injection, can lower dangerously high blood fat levels in children with familial chylomicronemia syndrome (FCS). FCS is a rare genetic condition that prevents the body from breaking down fats, leading to s…
Phase 3 • Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated Aug 28, 2026 00:00 UTC
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New drug offers hope for rare, severe high triglyceride conditions
Disease control Expanded accessThis program gives adults and adolescents with familial chylomicronemia syndrome (FCS) and adults with severe hypertriglyceridemia (SHTG) access to an experimental drug called plozasiran. Participants receive injections every 3 months for 15 to 21 months. The goal is to lower dan…
Sponsor: Arrowhead Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 09:09 UTC
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Gene editing shot aims to tame deadly fat levels in rare disease
Disease control Recruiting nowThis early-stage trial tests a single infusion of CS-121, a gene editing therapy, in 15 adults with familial chylomicronemia syndrome (FCS), a rare condition causing extremely high blood fat levels. The therapy targets the APOC3 gene to lower triglycerides and reduce disease burd…
Early phase 1 • Sponsor: CorrectSequence Therapeutics Co., Ltd • Aim: Disease control
Last updated Jun 27, 2026 09:04 UTC
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Gene-Editing shot aims to tame deadly fat levels in kids
Disease control Recruiting nowThis early-stage trial tests a new gene-editing treatment called CS-121 in 15 children and teens (ages 4-18) with a rare condition causing extremely high triglycerides. The therapy uses lipid nanoparticles to deliver a base editor that targets the APOC3 gene in the liver. The mai…
Early phase 1 • Sponsor: Shanghai Jiao Tong University School of Medicine • Aim: Disease control
Last updated Jun 27, 2026 09:00 UTC