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

April 5, 2024 updated by: Verve Therapeutics, Inc.

Open-label, Phase 1b, Single-ascending Dose and Optional re Dosing Study to Evaluate the Safety of VERVE-101 Administered to Patients With Heterozygous Familial Hypercholesterolemia, Atherosclerotic Cardiovascular Disease, and Uncontrolled Hypercholesterolemia

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.

Study Overview

Status

Active, not recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

44

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • New Zealand
      • Auckland, New Zealand
        • Clinical Study Center
      • Christchurch, New Zealand
        • Clinical Study Center
  • United Kingdom
      • London, United Kingdom
        • Clinical Study Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 75 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Part B: Single Dose Expansion
Participants will receive a single dose of VERVE-101 selected based on the doses studied in Part A.
Drug: VERVE-101
Intravenous (IV) infusion.
Experimental: Part A: Single Ascending Dose Escalation/Adaptive Design
Participants will receive a single dose of VERVE-101 in multiple dose-escalation cohorts.
Drug: VERVE-101
Intravenous (IV) infusion.

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs).
Time Frame: up to Day 365
up to Day 365

Secondary Outcome Measures

Outcome Measure
Time Frame
Evaluation of maximum observed concentration (Cmax)
Time Frame: up to Day 365
up to Day 365
Evaluation of time to maximum observed concentration (tmax)
Time Frame: up to Day 365
up to Day 365
Evaluation of terminal elimination half-life (t1/2)
Time Frame: up to Day 365
up to Day 365

Other Outcome Measures

Outcome Measure
Time Frame
Percent and absolute change from baseline in plasma PCSK9 concentration.
Time Frame: up to Day 365
up to Day 365
Percent and absolute change from baseline in LDL-C.
Time Frame: up to Day 365
up to Day 365

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Verve Therapeutics, Inc.

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 5, 2022

Primary Completion (Estimated)

December 1, 2024

Study Completion (Estimated)

December 1, 2024

Study Registration Dates

First Submitted

May 19, 2022

First Submitted That Met QC Criteria

May 25, 2022

First Posted (Actual)

May 31, 2022

Study Record Updates

Last Update Posted (Actual)

April 8, 2024

Last Update Submitted That Met QC Criteria

April 5, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VT-1001

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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