Phase 1b Study of VERVE-201 in Patients With Refractory Hyperlipidemia

April 22, 2026 updated by: Verve Therapeutics, Inc.

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

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 Hyperlipidemia.

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) and triglycerides. This study is designed to determine the safety and pharmacodynamic profile of VERVE-201 in this patient population.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

36

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

  • Australia
      • Adelaide, Australia
        • Recruiting
        • Clinical Study Center
      • Melbourne, Australia
        • Recruiting
        • Clinical Study Center
  • Canada
      • Chicoutimi, Canada
        • Recruiting
        • Clinical Study Center
      • Montreal, Canada
        • Recruiting
        • Clinical Study Center
      • Toronto, Canada
        • Recruiting
        • Clinical Study Center
  • South Africa
      • Cape Town, South Africa
        • Recruiting
        • Clinical Study Center
      • Johannesburg, South Africa
        • Recruiting
        • Clinical Study Center
  • United Kingdom
      • Liverpool, United Kingdom
        • Recruiting
        • Clinical Study Center
      • London, United Kingdom
        • Recruiting
        • Clinical Study Center
      • Manchester, United Kingdom
        • Recruiting
        • 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

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Refractory hypercholesterolemia
  • Refractory hypertriglyceridemia

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

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: Cohort 1: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-201.
Drug: VERVE-201
Intravenous (IV) infusion
Experimental: Cohort 2: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-201.
Drug: VERVE-201
Intravenous (IV) infusion
Experimental: Cohort 3: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-201.
Drug: VERVE-201
Intravenous (IV) infusion
Experimental: Cohort 4: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-201.
Drug: VERVE-201
Intravenous (IV) infusion
Experimental: Cohort 5: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-201.
Drug: VERVE-201
Intravenous (IV) infusion
Experimental: Cohort 6: Single Ascending Dose Escalation
Participants will receive a single dose of VERVE-201.
Drug: VERVE-201
Intravenous (IV) infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
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
Percent and absolute change from baseline in serum ANGPTL3 concentration
Time Frame: Up to Day 365
Up to Day 365
Percent and absolute change from baseline in serum LDL-C
Time Frame: Up to Day 365
Up to Day 365
Percent and absolute change from baseline in serum triglycerides
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)

October 30, 2024

Primary Completion (Estimated)

December 1, 2027

Study Completion (Estimated)

December 1, 2027

Study Registration Dates

First Submitted

June 4, 2024

First Submitted That Met QC Criteria

June 4, 2024

First Posted (Actual)

June 11, 2024

Study Record Updates

Last Update Posted (Actual)

April 28, 2026

Last Update Submitted That Met QC Criteria

April 22, 2026

Last Verified

November 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • VT-20101

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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