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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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 a new one-time gene-editing treatment (CS-121) for people with very high triglycerides that don't lower enough with standard medicines. The treatment aims to reduce triglycerides and could help prevent pancreatitis and heart problems, but it is an early-stage gene therapy with strict safety requirements.
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.
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