CS-121 APOC3 Base Editing in Children and Adolescents With Hyperchylomicronemia
Trial Parameters
Brief Summary
This is a Prospective, Single-center, Open-label, Single-arm Clinical Study to Evaluate the Safety and Efficacy of CS-121, an In Vivo Base Editing Therapy Delivered by Lipid Nanoparticles Targeting APOC3, in Children and Adolescents (4-18 years) With Hyperchylomicronemia
Eligibility Criteria
Inclusion Criteria: * Male or female participants aged 4 years ≤ age \< 18 years. * Severe hypertriglyceridemia (sHTG), defined as a triglyceride (TG) level ≥ 500 mg/dL. * Confirmed diagnosis of genetically inherited FCS via genetic testing, or clinically diagnosed FCS plus persistent chylomicronemia. * Failure to achieve adequate TG control, For participants under 8 years of age, the investigator determine at their discretion whether prior lipidlowering therapy has been administered. * Participants aged 6 years and above must sign the informed consent form themselves; for participants under 18 years of age, their parent/legal guardian must sign the informed consent form. (Participants under 6 years of age are exempt from signing the written informed consent form). * Female participants of childbearing potential must have a negative result on serum pregnancy testing. Exclusion Criteria: * Currently participating in other interventional clinical studies, or having an insufficient washou