A Study of VERVE-101 in Patients With Familial Hypercholesterolemia and Cardiovascular Disease

• Status: Not yet recruiting
• Trial end date: 2024-12-01
• Target enrollment: 0
• Participant gender: All

Summary

VT-1001 is an open-label, phase 1b, single-ascending dose study that will evaluate the safety of VERVE-101 administered to patients with heterozygous familial hypercholesterolemia (HeFH), atherosclerotic cardiovascular disease (ASCVD), and uncontrolled hypercholesterolemia. VERVE-101 uses base-editing technology designed to disrupt the expression of the PCSK9 gene in the liver and lower circulating PCSK9 and LDL-C in patients with established ASCVD due to HeFH. This study is designed to determine the safety and pharmacodynamic profile of VERVE-101 in this patient population.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Verve Therapeutics, Inc.

Criteria

Inclusion Criteria:

• Male and/or female participants 18 to 65 years at time of signing of informed consent

• Female participants not of child-bearing potential

• Diagnosis of HeFH due to mutation in LDLR

• Established ASCVD

Exclusion Criteria:

• Diagnosis of HeFH due to mutation in PCSK9 or APOB, homozygous FH, compound heterozygous FH, double heterozygous FH

• Active or history of chronic liver disease

• Current treatment with PCSK9 monoclonal antibody therapy

• Current or past treatment with inclisiran

• Clinically significant or abnormal laboratory values as defined by the protocol