A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease

NCT ID: NCT06164730

Last Updated: 2025-12-17

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

RECRUITING

Clinical Phase

PHASE1

Total Enrollment

85 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-04-30

Study Completion Date

2027-08-31

Brief Summary

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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.

Detailed Description

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Conditions

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Heterozygous Familial Hypercholesterolemia Premature Coronary Heart Disease

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Single ascending dose escalation/adaptive design.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Cohort 1: Single Ascending Dose Escalation

Participants will receive a single dose of VERVE-102.

Group Type EXPERIMENTAL

VERVE-102

Intervention Type DRUG

Intravenous (IV) infusion

Cohort 2: Single Ascending Dose Escalation

Participants will receive a single dose of VERVE-102.

Group Type EXPERIMENTAL

VERVE-102

Intervention Type DRUG

Intravenous (IV) infusion

Cohort 3: Single Ascending Dose Escalation

Participants will receive a single dose of VERVE-102.

Group Type EXPERIMENTAL

VERVE-102

Intervention Type DRUG

Intravenous (IV) infusion

Cohort 4: Single Ascending Dose Escalation

Participants will receive a single dose of VERVE-102.

Group Type EXPERIMENTAL

VERVE-102

Intervention Type DRUG

Intravenous (IV) infusion

Cohort 5: Single Ascending Dose Escalation

Participants will receive a single dose of VERVE-102.

Group Type EXPERIMENTAL

VERVE-102

Intervention Type DRUG

Intravenous (IV) infusion

Cohort 6: Single Ascending Dose Escalation

Participants will receive a single dose of VERVE-102.

Group Type EXPERIMENTAL

VERVE-102

Intervention Type DRUG

Intravenous (IV) infusion

Interventions

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VERVE-102

Intravenous (IV) infusion

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Diagnosis of HeFH or premature CAD

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
Minimum Eligible Age

18 Years

Maximum Eligible Age

70 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

Dothan, Alabama, United States

Site Status ACTIVE_NOT_RECRUITING

Clinical Study Center

Adelaide, , Australia

Site Status RECRUITING

Clinical Study Center

Melbourne, , Australia

Site Status RECRUITING

Clinical Study Center

Sydney, , Australia

Site Status ACTIVE_NOT_RECRUITING

Clinical Study Center

Chicoutimi, , Canada

Site Status RECRUITING

Clinical Study Center

Montreal, , Canada

Site Status RECRUITING

Clinical Study Center

Toronto, , Canada

Site Status RECRUITING

Clinical Study Center

Vancouver, , Canada

Site Status RECRUITING

Clinical Study Center

Rehovot, , Israel

Site Status RECRUITING

Clinical Study Center

Christchurch, , New Zealand

Site Status RECRUITING

Clinical Study Center

Birmingham, , United Kingdom

Site Status ACTIVE_NOT_RECRUITING

Clinical Study Center

Edinburgh, , United Kingdom

Site Status RECRUITING

Clinical Study Center

London, , United Kingdom

Site Status RECRUITING

Clinical Study Center

Manchester, , United Kingdom

Site Status RECRUITING

Clinical Study Center

Nottingham, , United Kingdom

Site Status RECRUITING

Countries

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United States Australia Canada Israel New Zealand United Kingdom

Central Contacts

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Clinical Development

Role: CONTACT

781-970-6833

Other Identifiers

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VT-10201

Identifier Type: -

Identifier Source: org_study_id