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.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
36
Intravenous (IV) infusion
Clinical Study Center
Adelaide, Australia
RECRUITINGClinical Study Center
Melbourne, Australia
RECRUITINGClinical Study Center
Chicoutimi, Canada
RECRUITINGClinical Study Center
Montreal, Canada
RECRUITINGClinical Study Center
Toronto, Canada
RECRUITINGClinical Study Center
Cape Town, South Africa
RECRUITINGClinical Study Center
Johannesburg, South Africa
RECRUITINGClinical Study Center
Liverpool, United Kingdom
RECRUITINGClinical Study Center
London, United Kingdom
RECRUITINGClinical Study Center
Manchester, United Kingdom
RECRUITINGIncidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
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
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
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