Gene editing for high triglycerides in kids with FCS
Treatments studied
Part of Digestive system, Genetic & congenital, Hormones & metabolism clinical trials.
This trial tests a new gene-editing treatment (CS-121) for children and teens with a rare inherited condition called Familial Chylomicronemia Syndrome (FCS). It aims to lower very high triglyceride levels and reduce the risk of pancreatitis.
Summary written for real people, not researchers, by Clin2.
Who can take part
- Age between 4 and 17 years old.
- Very high triglycerides (a type of fat in the blood) — at least 500 mg/dL.
- Diagnosed with Familial Chylomicronemia Syndrome (FCS), either by a genetic test or by a doctor based on symptoms.
- Have not had a bad reaction to other treatments, or treatment hasn't worked well enough.
- No acute pancreatitis in the past month.
- Livers and kidneys are working well enough (your doctor will check).
Quick eligibility check
Answer a few plain-language questions, based on this study's own requirements, to get a preliminary sense of fit.
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Other trials that look related to this one.
This trial tests a new gene therapy called YOLT-101 for people with familial hypercholesterolemia, a genetic condition that causes very high cholesterol. The treatment aims to lower cholesterol levels and reduce heart risks.
This trial tests a new injection called ART002g1 for people with a genetic condition that causes very high cholesterol. It aims to see if the drug is safe and can help lower cholesterol levels.
This study tests a new gene-editing treatment called CS-121 for people with FCS whose triglycerides remain very high despite standard therapy. It aims to lower triglyceride levels by editing the APOC3 gene.
This trial tests an experimental drug called CS-121 for people with very high triglyceride levels that don't improve with standard treatments. It uses a new gene-editing approach to lower triglycerides and aims to reduce the risk of pancreatitis and other complications.
This study tests a new medicine called olezarsen to lower very high triglycerides (fats) in the blood of children and teenagers with familial chylomicronemia syndrome (FCS), a rare genetic condition. The goal is to reduce the risk of pancreatitis and other complications.
This trial tests a one-time gene therapy (NGGT006) to treat a severe form of inherited high cholesterol that doesn't respond well to standard medications. It aims to lower cholesterol and reduce the risk of heart disease.
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