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

Conditions

• Heterozygous Familial Hypercholesterolemia
• Premature Coronary Heart Disease

Eligibility Criteria

Sex

ALL

Age

18 Years - 70 Years

Healthy Volunteers

Not accepted

Interventions

• VERVE-102 — DRUG
  Intravenous (IV) infusion

Study Details

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.

Key Dates

Start date

Apr 30, 2024

Status verified

Jun 2026

Primary completion

Aug 31, 2026

Completion

Aug 31, 2027

Study Design

Enrollment

85 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Cohort 1: Single Ascending Dose Escalation
  Participants will receive a single dose of VERVE-102.
• Experimental: Cohort 2: Single Ascending Dose Escalation
  Participants will receive a single dose of VERVE-102.
• Experimental: Cohort 3: Single Ascending Dose Escalation
  Participants will receive a single dose of VERVE-102.
• Experimental: Cohort 4: Single Ascending Dose Escalation
  Participants will receive a single dose of VERVE-102.
• Experimental: Cohort 5: Single Ascending Dose Escalation
  Participants will receive a single dose of VERVE-102.
• Experimental: Cohort 6: Single Ascending Dose Escalation
  Participants will receive a single dose of VERVE-102.
• Experimental: Cohort 7: Single Ascending Dose Escalation
  Participants will receive a single dose of VERVE-102.
• Experimental: Cohort 8: Single Ascending Dose Escalation
  Participants will receive a single dose of VERVE-102.
• Experimental: Cohort 9: Single Fixed Dose
  Participants will receive a single dose of VERVE-102.
• Experimental: Cohort 10: Single Fixed Dose
  Participants will receive a single dose of VERVE-102.

Primary Outcome Measure

Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) [ Time Frame: up to Day 365 ]

Central Contacts

• Clinical Development
  781-970-6833
  [email protected]

Locations (8)

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