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

Verve Therapeutics

Status and phase

Enrolling

Phase 1

Conditions

Premature Coronary Heart Disease

Heterozygous Familial Hypercholesterolemia

Treatments

Drug: VERVE-102

Study type

Interventional

Funder types

Industry

Identifiers

NCT06164730

VT-10201

Details and patient eligibility

About

VT-10201 is an Open-label, Phase 1b, Single-ascending Dose Study That Will Evaluate the Safety of VERVE-102 Administered to Patients With Heterozygous Familial Hypercholesterolemia (HeFH) or Premature Coronary Artery Disease (CAD) Who Require Additional Lowering of LDL-C. VERVE-102 Uses Base-editing Technology Designed to Disrupt the Expression of the PCSK9 Gene in the Liver and Lower Circulating PCSK9 and LDL-C. This Study is Designed to Determine the Safety and Pharmacodynamic Profile of VERVE-102 in This Patient Population.

Enrollment

85 estimated patients

Sex

All

Ages

18 to 70 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Diagnosis of HeFH or premature CAD

Exclusion criteria

Homozygous familial hypercholesterolemia
Active or history of chronic liver disease
Current treatment with PCSK9 inhibitor or prior treatment within specified timeframe
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)

85 participants in 10 patient groups

Cohort 1: Single Ascending Dose Escalation

Experimental group

Description: