Phase 1b Study of VERVE-201 in Patients With Refractory Hyperlipidemia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: VERVE-201.
- Who it may be relevant to
- Registry conditions: Hypercholesterolemia, Hypertriglyceridemia, Refractory Hyperlipidemia. Basic parameters: 18 years — 70 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Australia, Canada, Netherlands, South Africa +1
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Phase 1b Single Ascending Dose Study to Evaluate the Safety of VERVE-201 in Patients With Refractory Hyperlipidemia
Overview
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
- Drug VERVE-201
Intravenous (IV) infusion
Primary outcome measures
- Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) [Time frame: Up to Day 365]
Secondary outcome measures (6)
- Evaluation of maximum observed concentration (Cmax) [Time frame: Up to Day 365]
- Evaluation of time to maximum observed concentration (tmax) [Time frame: Up to Day 365]
- Evaluation of terminal elimination half-life (t1/2) [Time frame: Up to Day 365]
- Percent and absolute change from baseline in serum ANGPTL3 concentration [Time frame: Up to Day 365]
- Percent and absolute change from baseline in serum LDL-C [Time frame: Up to Day 365]
- Percent and absolute change from baseline in serum triglycerides [Time frame: Up to Day 365]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Non-randomized
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 4 centers
- Clinical Study Center — Clearwater
- Clinical Study Center — Miami Lakes
- Clinical Study Center — Port Orange
- Clinical Study Center — New York
United Kingdom · 4 centers
- Clinical Study Center — Liverpool
- Clinical Study Center — London
- Clinical Study Center 2 — Manchester
- Clinical Study Center — Manchester
Canada · 3 centers
- Clinical Study Center — Chicoutimi
- Clinical Study Center — Montreal
- Clinical Study Center — Toronto
Australia · 2 centers
- Clinical Study Center — Adelaide
- Clinical Study Center — Melbourne
South Africa · 2 centers
- Clinical Study Center — Cape Town
- Clinical Study Center — Johannesburg
Netherlands · 1 center
- Clinical Study Center — Amsterdam
Identifiers
NCT: NCT06451770 · VT-20101