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NCT Number: NCT05398029

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

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.

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Key information

Conditions

Heterozygous Familial Hypercholesterolemia Arterial Occlusive Diseases Arteriosclerosis Atherosclerosis Atherosclerotic Cardiovascular Disease Cardiovascular Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dyslipidemias Genetic Diseases, Inborn Hypercholesterolemia Hyperlipidemias Hyperlipoproteinemia Type II Hyperlipoproteinemias Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases Vascular Diseases

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Clinical Study Center, Auckland, New Zealand

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

VERVE-101

Drug

Intravenous (IV) infusion.

Primary outcomes

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

    Time frame: up to Day 365

Secondary outcomes

  1. Evaluation of maximum observed concentration (Cmax)

    Time frame: up to Day 365

  2. Evaluation of time to maximum observed concentration (tmax)

    Time frame: up to Day 365

  3. Evaluation of terminal elimination half-life (t1/2)

    Time frame: up to Day 365

Other outcomes

  1. Percent and absolute change from baseline in plasma PCSK9 concentration.

    Time frame: up to Day 365

  2. Percent and absolute change from baseline in LDL-C.

    Time frame: up to Day 365

Sponsors and collaborators

Lead sponsor

Verve Therapeutics, Inc.

Industry

Registry information

Official study title

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

Important dates

Study start
2022
Primary completion
2025
Study completion
2025
First posted
May 31, 2022
Registry last updated
May 8, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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