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

Phase 1

Recruiting

• Conditions: Hypercholesterolemia
• Interventions: Drug: VERVE-201

• Registration Number: NCT06451770
• Lead Sponsor: Verve Therapeutics, Inc.

Brief Summary

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 Hypercholesterolemia.

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

Detailed Description

Not available

Study Timeline

• Status Verified: 2024-06
• Study First Submitted: 2024-06-04
• Study First Submitted QC: 2024-06-04
• Study First Posted: 2024-06-11
• Study Start: 2024-10-30
• Last Update Submitted: 2024-11-11
• Last Update Posted: 2024-11-14
• Primary Completion: 2027-03
• Study Completion: 2027-03

Recruitment & Eligibility

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

Inclusion Criteria

• Refractory hypercholesterolemia
• Females of non-childbearing potential or males

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
Cohort 1: Single Ascending Dose Escalation	VERVE-201	Participants will receive a single dose of VERVE-201.
Cohort 2: Single Ascending Dose Escalation	VERVE-201	Participants will receive a single dose of VERVE-201.
Cohort 3: Single Ascending Dose Escalation	VERVE-201	Participants will receive a single dose of VERVE-201.
Cohort 4: Single Ascending Dose Escalation	VERVE-201	Participants will receive a single dose of VERVE-201.

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 maximum observed concentration (Cmax)	Up to Day 365
Evaluation of terminal elimination half-life (t1/2)	Up to Day 365
Percent and absolute change from baseline in serum ANGPTL3 concentration	Up to Day 365
Percent and absolute change from baseline in serum LDL-C	Up to Day 365

Trial Locations

Locations (1)

Clinical Study Center; 🇬🇧 London, United Kingdom