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: 36 estimated patients

Sex: All

Ages: 18 to 70 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Diagnosis of HeFH or premature CAD
• Females of non-childbearing potential or males

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)

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36 participants in 4 patient groups

Cohort 1: Single Ascending Dose Escalation

Experimental group

Description:

Participants will receive a single dose of VERVE-102.

Treatment:

Drug: VERVE-102

Cohort 2: Single Ascending Dose Escalation

Experimental group

Description:

Participants will receive a single dose of VERVE-102.

Treatment:

Drug: VERVE-102

Cohort 3: Single Ascending Dose Escalation

Experimental group

Description:

Participants will receive a single dose of VERVE-102.

Treatment:

Drug: VERVE-102

Cohort 4: Single Ascending Dose Escalation

Experimental group

Description:

Participants will receive a single dose of VERVE-102.

Treatment:

Drug: VERVE-102