A Multicenter, Dose-exploration, Open-label Phase II Study to Evaluate the Efficacy and Safety of EDP167 in Adult Patients With Homozygous Familial Hypercholesterolaemia
EDP167 is a double-stranded small interfering RNA (siRNA) drug targeting angiopoietin-like 3 protein (ANGPTL3), which may bring benefits for patients with dyslipidemia conditions. This is a dose exploration study in Homozygous Familial Hypercholesterolaemia (HoFH) patients to evaluate the efficacy and safety and pharmacokinetics (PK)/pharmacodynamics (PD) profiles of multiple EDP167 injections.
• Age ≥18 years old, male or female, and weight ≥40 kg.
• Genetic diagnosis or clinical diagnosis of HoFH.
• Fasting serum LDL-C ≥2.6 mmol/L.
• Follow a daily low-fat diet during the study.
• Receiving stable and tolerable lipid-lowering treatment or other drugs for chronic diseases treatments for certain periods before the study, and maintain the stable treatments throughout the study.
• The pregnancy test results of Women of Childbearing Potential must be negative.
• Agree to use contraceptive measures that comply with regulations and the protocol requirements during the study, and until 6 months after the last dose.
• Understand the study procedures, voluntarily participate, and sign the informed consent form.