Clinical trial

A Phase 1b Single Ascending Dose Study to Evaluate the Safety of VERVE-201 in Patients With Refractory Hyperlipidemia

Name

VT-20101

Description

VT-20101 is an Open-label, Phase 1b, Single-ascending dose study that will evaluate the safety of VERVE-201 administered to patients with Refractory Hypercholesterolemia. VERVE-201 uses base-editing technology designed to inactivate the expression of the ANGPTL3 gene in the liver and lower circulating low-density lipoprotein cholesterol (LDL-C). This study is designed to determine the safety and pharmacodynamic profile of VERVE-201 in this patient population.

Trial arms

Trial start

2024-12-01

Estimated PCD

2027-03-01

Trial end

2027-03-01

Status

Not yet recruiting

Phase

Early phase I

Treatment

VERVE-201

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: * Refractory hypercholesterolemia * Females of non-childbearing potential or males Exclusion Criteria: * Active or history of chronic liver disease * Current treatment with monoclonal antibody targeting ANGPTL3 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

2024-06-11

1 organization

1 product

1 indication

Organization

Verve Therapeutics

Product

VERVE-201

Indication

Hypercholesterolemia