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

Verve Therapeutics

Status and phase: Completed; Phase 1

Conditions: Atherosclerotic Cardiovascular Disease; Hypercholesterolemia; Heterozygous Familial Hypercholesterolemia

Treatments: Drug: VERVE-101

Study type: Interventional

Funder types: Industry

Identifiers

NCT05398029

VT-1001

Details and patient eligibility

About

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.

Enrollment: 13 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male and/or female participants 18 up to 75 years at time of signing of informed consent
• Female participants not of child-bearing potential
• Diagnosis of HeFH
• Established ASCVD

Exclusion criteria

• 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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: None (Open label)

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13 participants in 2 patient groups

Part A: Single Ascending Dose Escalation/Adaptive Design

Experimental group

Description:

Participants will receive a single dose of VERVE-101 in multiple dose-escalation cohorts.

Treatment:

Drug: VERVE-101

Part B: Single Dose Expansion

Experimental group

Description:

Participants will receive a single dose of VERVE-101 selected based on the doses studied in Part A.

Treatment:

Drug: VERVE-101