Familial chylomicronemia syndrome

This 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…

This study tests whether orlistat, a drug that blocks fat absorption, can safely lower very high blood triglycerides in people with type 1 hyperlipoproteinemia, a rare genetic disorder. About 28 participants aged 8 and older will take orlistat or a placebo for several months. The…

This 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…

This 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…

This 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…

This study looks at how people with lipoprotein lipase (LPL) deficiency, a rare condition that causes extremely high blood fat levels, handle dietary fats. Researchers will use special imaging and breath tests to see where fat goes in the body and how it's used for energy. The go…