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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New drug combo aims to tame deadly fat levels in the blood
Disease control OngoingThis Phase 2 trial tests two experimental injections, HRS-7249 and SHR-1918, in 108 adults with severely high triglycerides who are at high risk of acute pancreatitis. The study aims to see if these drugs can safely lower fat levels in the blood and reduce the chance of a pancrea…
Phase 2 • Sponsor: Fujian Shengdi Pharmaceutical Co., Ltd. • Aim: Disease control
Last updated Sep 10, 2026 00:00 UTC
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New shot aims to tame dangerous fat levels in blood
Disease control OngoingThis study tests a single dose of a new drug called RN0361 in 108 adults. It includes healthy volunteers and people with very high triglycerides, a type of fat in the blood that can cause serious health problems. The goal is to see if the drug is safe and lowers fat levels. This …
Phase 1/2 • Sponsor: Ikaria Bioscience Pty Ltd • Aim: Disease control
Last updated Jun 27, 2026 08:07 UTC
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New hope for rare fat disorder: safety trial of olezarsen underway
Disease control OngoingThis study tests the safety of a new drug, olezarsen, in 24 adults with familial chylomicronemia syndrome (FCS), a rare condition causing extremely high blood fats. All participants have previously taken volanesorsen. The study monitors side effects like low platelet counts and k…
Phase 3 • Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC
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New drug shows promise for rare fat disorder in extended trial
Disease control OngoingThis study is for people with familial chylomicronemia syndrome (FCS), a rare condition causing extremely high blood fats. It tests a drug called olezarsen, given as a shot under the skin, to see if it can lower triglycerides over months to years. Participants are 60 patients who…
Phase 3 • Sponsor: Ionis Pharmaceuticals, Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:03 UTC