A Study of VERVE-102 in Patients with Familial Hypercholesterolemia or Premature Coronary Artery Disease

Phase 1

Recruiting

• Conditions: Heterozygous Familial Hypercholesterolemia; Premature Coronary Heart Disease
• Interventions: Drug: VERVE-102

• Registration Number: NCT06164730
• Lead Sponsor: Verve Therapeutics, Inc.

Brief Summary

VT-10201 is an Open-label, Phase 1b, Single-ascending Dose Study That Will Evaluate the Safety of VERVE-102 Administered to Patients With Heterozygous Familial Hypercholesterolemia (HeFH) or Premature Coronary Artery Disease (CAD) Who Require Additional Lowering of LDL-C. VERVE-102 Uses Base-editing Technology Designed to Disrupt the Expression of the PCSK9 Gene in the Liver and Lower Circulating PCSK9 and LDL-C. This Study is Designed to Determine the Safety and Pharmacodynamic Profile of VERVE-102 in This Patient Population.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-12-01
• Study First Submitted QC: 2023-12-01
• Study First Posted: 2023-12-11
• Study Start: 2024-04-30
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-07
• Last Update Posted: 2024-11-11
• Primary Completion: 2026-08
• Study Completion: 2026-08

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 36

Inclusion Criteria

• Diagnosis of HeFH or premature CAD
• Females of non-childbearing potential or males

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Exclusion Criteria

• Homozygous familial hypercholesterolemia
• Active or history of chronic liver disease
• Current treatment with PCSK9 inhibitor or prior treatment within specified timeframe
• 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
Cohort 1: Single Ascending Dose Escalation	VERVE-102	Participants will receive a single dose of VERVE-102.
Cohort 2: Single Ascending Dose Escalation	VERVE-102	Participants will receive a single dose of VERVE-102.
Cohort 3: Single Ascending Dose Escalation	VERVE-102	Participants will receive a single dose of VERVE-102.
Cohort 4: Single Ascending Dose Escalation	VERVE-102	Participants will receive a single dose of VERVE-102.

Primary Outcome Measures

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

Secondary Outcome Measures

Name	Time	Method
Evaluation of time to maximum observed concentration (tmax)	up to Day 365
Evaluation of terminal elimination half-life (t1/2)	up to Day 365
Percent and absolute change from baseline in LDL-C	up to Day 365
Evaluation of maximum observed concentration (Cmax)	up to Day 365
Percent and absolute change from baseline in plasma PCSK9 concentration	up to Day 365

Trial Locations

Locations (1)

Clinical Study Center; 🇬🇧 Manchester, United Kingdom