Hypercholesterolemia, Hypertriglyceridemia, Refractory Hyperlipidemia
Conditions
Keywords
VERVE-201, Gene Editing, Base Editing, Refractory Hypercholesterolemia, ANGPTL3, ASCVD, Refractory Hypertriglyceridemia, Refractory Hyperlipidemia, Familial Hypercholesterolemia
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 Hyperlipidemia. 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) and triglycerides. This study is designed to determine the safety and pharmacodynamic profile of VERVE-201 in this patient population.
Interventions
DRUGVERVE-201
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
* Refractory hypercholesterolemia * Refractory hypertriglyceridemia
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
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 serum ANGPTL3 concentration | Up to Day 365 |
| Percent and absolute change from baseline in serum LDL-C | Up to Day 365 |
| Percent and absolute change from baseline in serum triglycerides | Up to Day 365 |
Countries
Australia, Canada, Netherlands, South Africa, 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