Heterozygous Familial Hypercholesterolemia, Premature Coronary Heart Disease
Conditions
Keywords
VERVE-102, Familial Hypercholesterolemia, Coronary Artery Disease, Dose Escalation, Gene Editing, Base Editing, heart-2
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.
Interventions
DRUGVERVE-102
Intravenous (IV) infusion
Sponsors
Verve Therapeutics, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Single ascending dose escalation/adaptive design.
Eligibility
Sex/Gender
ALL
Age
18 Years to 70 Years
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) | up to Day 365 |
Secondary
| Measure | Time frame |
|---|---|
| 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 |
| Percent and absolute change from baseline in plasma PCSK9 concentration | up to Day 365 |
| Percent and absolute change from baseline in LDL-C | up to Day 365 |
Countries
Australia, Canada, Israel, New Zealand, United Kingdom, United States
Contacts
CONTACTTrial questions or participation questions: 1-877-CTLILLY (1-877-285-4559) or
CONTACTPhysicians interested in becoming principal investigators please contact
STUDY_DIRECTORCall 1-877-CTLILLY (1-877-285-4559) or 1-317-615-4559 Mon - Fri 8 AM - 8 PM Eastern time (UTC/GMT - 5 hours, EST)
Eli Lilly and Company
Outcome results
None listed