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

Purpose

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.

Conditions

  • Heterozygous Familial Hypercholesterolemia
  • Premature Coronary Heart Disease

Eligibility

Eligible Ages
Between 18 Years and 70 Years
Eligible Sex
All
Accepts Healthy Volunteers
No

Inclusion Criteria

  • Diagnosis of HeFH or premature CAD

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

Study Design

Phase
Phase 1
Study Type
Interventional
Allocation
Non-Randomized
Intervention Model
Sequential Assignment
Intervention Model Description
Single ascending dose escalation/adaptive design.
Primary Purpose
Treatment
Masking
None (Open Label)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Cohort 1: Single Ascending Dose Escalation 		Participants will receive a single dose of VERVE-102.
  • Drug: VERVE-102
    Intravenous (IV) infusion
Experimental
Cohort 2: Single Ascending Dose Escalation 		Participants will receive a single dose of VERVE-102.
  • Drug: VERVE-102
    Intravenous (IV) infusion
Experimental
Cohort 3: Single Ascending Dose Escalation 		Participants will receive a single dose of VERVE-102.
  • Drug: VERVE-102
    Intravenous (IV) infusion
Experimental
Cohort 4: Single Ascending Dose Escalation 		Participants will receive a single dose of VERVE-102.
  • Drug: VERVE-102
    Intravenous (IV) infusion
Experimental
Cohort 5: Single Ascending Dose Escalation 		Participants will receive a single dose of VERVE-102.
  • Drug: VERVE-102
    Intravenous (IV) infusion
Experimental
Cohort 6: Single Ascending Dose Escalation 		Participants will receive a single dose of VERVE-102.
  • Drug: VERVE-102
    Intravenous (IV) infusion
Experimental
Cohort 7: Single Ascending Dose Escalation 		Participants will receive a single dose of VERVE-102.
  • Drug: VERVE-102
    Intravenous (IV) infusion
Experimental
Cohort 8: Single Ascending Dose Escalation 		Participants will receive a single dose of VERVE-102.
  • Drug: VERVE-102
    Intravenous (IV) infusion
Experimental
Cohort 9: Single Fixed Dose 		Participants will receive a single dose of VERVE-102.
  • Drug: VERVE-102
    Intravenous (IV) infusion
Experimental
Cohort 10: Single Fixed Dose 		Participants will receive a single dose of VERVE-102.
  • Drug: VERVE-102
    Intravenous (IV) infusion

Recruiting Locations

Clinical Study Center
Dothan, Alabama 36305

Clinical Study Center
Pomona, California 91768

Clinical Study Center
Boca Raton, Florida 33434

Clinical Study Center
Jacksonville, Florida 32216

Clinical Study Center
Winter Park, Florida 32789

Clinical Study Center
High Point, North Carolina 27260

Clinical Study Center
DeSoto, Texas 75115

Clinical Study Center
Renton, Washington 98057

More Details

Status
Recruiting
Sponsor
Verve Therapeutics, Inc.

Study Contact

Clinical Development
781-970-6833
verve102clinicaltrials@lists.lilly.com