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

V

Verve Therapeutics

Status and phase

Active, not recruiting
Phase 1

Conditions

Atherosclerotic Cardiovascular Disease
Hypercholesterolemia
Heterozygous Familial Hypercholesterolemia

Treatments

Drug: VERVE-101

Study type

Interventional

Funder types

Industry

Identifiers

NCT05398029
VT-1001

Details and patient eligibility

About

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.

Enrollment

44 estimated patients

Sex

All

Ages

18 to 75 years old

Volunteers

No Healthy Volunteers

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

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Sequential Assignment

Masking

None (Open label)

44 participants in 2 patient groups

Part A: Single Ascending Dose Escalation/Adaptive Design
Experimental group
Description:
Participants will receive a single dose of VERVE-101 in multiple dose-escalation cohorts.
Treatment:
Drug: VERVE-101
Part B: Single Dose Expansion
Experimental group
Description:
Participants will receive a single dose of VERVE-101 selected based on the doses studied in Part A.
Treatment:
Drug: VERVE-101

Trial contacts and locations

3

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Central trial contact

Clinical Operations at Verve Therapeutics

Data sourced from clinicaltrials.gov

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