Gene editing for familial chylomicronemia syndrome (FCS)
Treatments studied
Part of Digestive system, Hormones & metabolism clinical trials.
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.
Summary written for real people, not researchers, by Clin2.
Who can take part
- You are between 18 and 55 years old.
- You take standard therapy for FCS but your triglycerides are still above 10 mmol/L (about 880 mg/dL) on at least three separate tests, or you cannot tolerate standard therapy.
- Your NAFCS score is 45 or higher (this is a way to measure how likely you have FCS).
- You are willing to follow study rules, including diet and contraception requirements.
- You have not had acute pancreatitis in the last 3 months or a heart attack or unstable angina in the last 6 months.
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 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 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.
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