Phase 1b Study to Assess Safety of Single-Dose VERVE-102 in Adults with Familial Hypercholesterolemia or Early Coronary Artery Disease Needing Lower LDL Cholesterol

What this Trial Is About

Researchers are evaluating the safety and pharmacodynamics of VERVE-102, a drug using base-editing technology to disrupt the PCSK9 gene in the liver. This study focuses on adults with heterozygous familial hypercholesterolemia HeFH or premature coronary artery disease CAD who need further lowering of LDL cholesterol. The trial is an open-label, Phase 1b, single ascending dose study designed to assess safety and drug behavior in this patient group. Participants will receive a single intravenous dose of VERVE-102 in one of several dose-escalation or fixed-dose cohorts. The study uses a sequential design without randomization or blinding. Each participant is administered one dose, and the study monitors responses up to one year after dosing. During the study, participants will be closely observed for treatment-emergent adverse events and serious adverse events for up to 365 days. Researchers will measure drug concentration levels, time to peak concentration, elimination half-life, and changes in plasma PCSK9 and LDL cholesterol from baseline. This extended follow-up helps evaluate the drugs safety profile and pharmacodynamic effects over time.

A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease