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

NCT ID: NCT05398029

Last Updated: 2025-05-08

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

13 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-07-05

Study Completion Date

2025-02-14

Brief Summary

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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.

Detailed Description

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Conditions

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Heterozygous Familial Hypercholesterolemia Atherosclerotic Cardiovascular Disease Hypercholesterolemia

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Single ascending dose escalation/adaptive design followed by single dose expansion.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Part A: Single Ascending Dose Escalation/Adaptive Design

Participants will receive a single dose of VERVE-101 in multiple dose-escalation cohorts.

Group Type EXPERIMENTAL

VERVE-101

Intervention Type DRUG

Intravenous (IV) infusion.

Part B: Single Dose Expansion

Participants will receive a single dose of VERVE-101 selected based on the doses studied in Part A.

Group Type EXPERIMENTAL

VERVE-101

Intervention Type DRUG

Intravenous (IV) infusion.

Interventions

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VERVE-101

Intravenous (IV) infusion.

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Verve Therapeutics, Inc.

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Clinical Study Center

Auckland, , New Zealand

Site Status

Clinical Study Center

Christchurch, , New Zealand

Site Status

Clinical Study Center

London, , United Kingdom

Site Status

Countries

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New Zealand United Kingdom

Other Identifiers

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VT-1001

Identifier Type: -

Identifier Source: org_study_id

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