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

Phase 1

Active, not recruiting

• Conditions: Atherosclerotic Cardiovascular Disease; Hypercholesterolemia; Heterozygous Familial Hypercholesterolemia
• Interventions: Drug: VERVE-101

• Registration Number: NCT05398029
• Lead Sponsor: Verve Therapeutics, Inc.

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2022-05-19
• Study First Submitted QC: 2022-05-25
• Study First Posted: 2022-05-31
• Study Start: 2022-07-05
• Status Verified: 2024-12
• Last Update Submitted: 2024-12-24
• Last Update Posted: 2024-12-30
• Primary Completion: 2025-06
• Study Completion: 2025-06

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 44

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs).	up to Day 365

Secondary Outcome Measures

Name	Time	Method
Evaluation of maximum observed concentration (Cmax)	up to Day 365
Evaluation of time to maximum observed concentration (tmax)	up to Day 365
Evaluation of terminal elimination half-life (t1/2)	up to Day 365

Trial Locations

Locations (1)

Clinical Study Center; 🇬🇧 London, United Kingdom