CompletedPhase 1

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

New Zealand, United Kingdom13 participantsStarted 2022-07-05

Plain-language summary

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.

Who can participate

Age range18 Years – 75 Years

SexALL

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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

What they're measuring

Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs).

Timeframe: up to Day 365

Trial details

NCT IDNCT05398029

SponsorVerve Therapeutics, Inc.

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2025-02-14

View on ClinicalTrials.gov More Heterozygous Familial Hypercholesterolemia trials