Clinical trial

Open-label, Phase 1b, Single Ascending Dose Study to Evaluate the Safety of VERVE-102 Administered to Patients With Heterozygous Familial Hypercholesterolemia or Premature Coronary Artery Disease Who Require Additional Lowering of Low-density Lipoprotein Cholesterol

Name

VT-10201

Description

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.

Trial arms

Trial start

2024-05-01

Estimated PCD

2026-08-01

Trial end

2026-08-01

Status

Not yet recruiting

Phase

Early phase I

Treatment

VERVE-102

Intravenous (IV) infusion

Arms:

Cohort 1: Single Ascending Dose Escalation, Cohort 2: Single Ascending Dose Escalation, Cohort 3: Single Ascending Dose Escalation, Cohort 4: Single Ascending Dose Escalation

Size

Primary endpoint

Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)

up to Day 365

Eligibility criteria

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

Protocol

{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SEQUENTIAL', 'interventionModelDescription': 'Single ascending dose escalation/adaptive design.', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 36, 'type': 'ESTIMATED'}}

Updated at

2023-12-11

1 organization

1 product

2 indications

Organization

Verve Therapeutics

Product

VERVE-102

Indication

Heterozygous Familial Hypercholesterolemia

Indication

Coronary Heart Disease