VT-1001 is an open-label, phase 1b, single-ascending dose study that will evaluate the safety of VERVE-101 administered to patients with heterozygous familial hypercholesterolemia (HeFH), atherosclerotic cardiovascular disease (ASCVD), and uncontrolled hypercholesterolemia. VERVE-101 uses base-editing technology designed to disrupt the expression of the PCSK9 gene in the liver and lower circulating PCSK9 and LDL-C in patients with established ASCVD due to HeFH. This study is designed to determine the safety and pharmacodynamic profile of VERVE-101 in this patient population.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
13
Intravenous (IV) infusion.
Clinical Study Center
Auckland, New Zealand
Clinical Study Center
Christchurch, New Zealand
Clinical Study Center
London, United Kingdom
Incidence of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and adverse events of special interest (AESIs).
Time frame: up to Day 365
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
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