Phase II Study to Explore Doses of EDP167 Injections for Adults with Homozygous Familial Hypercholesterolemia HoFH

What this Trial Is About

This research aims to evaluate the effects of EDP167, a small interfering RNA drug targeting the angiopoietin-like 3 protein ANGPTL3, in adults with Homozygous Familial Hypercholesterolemia HoFH, a genetic condition causing very high cholesterol levels. By inhibiting ANGPTL3, EDP167 may help manage lipid levels in these patients. The study is a phase II, dose-exploration trial designed to assess the drugs safety, effectiveness, and how it behaves in the body pharmacokinetics and pharmacodynamics. Participants will be randomly assigned to receive either 200 mg or 300 mg doses of EDP167 through subcutaneous injections. The main study phase includes a screening period and 24 weeks of treatment, with injections given on Day 1 and Week 12. After this, those who complete the main phase may enter a 6-month extension phase receiving injections at Month 0 and Month 3, followed by a 6-month follow-up to further monitor effects and safety. Throughout the study, participants will have their cholesterol levels and other lipid markers regularly measured, including LDL-C, triglycerides, total cholesterol, and ANGPTL3 levels. Safety and immune response to the drug will also be assessed. Participants are monitored to observe changes from baseline up to 6 months after treatment during the extension phase. The total participation duration includes initial screening, treatment periods, and follow-up visits to evaluate both short- and longer-term study outcomes.

A Dose-exploration Study of EDP167 in HoFH