Phase 1b study of VERVE-101 administered to patients with heterozygous familial hypercholesterolemia
Research type
Research Study
Full title
Open-label, phase 1b, single-ascending dose and optional re-dosing study to evaluate the safety of VERVE-101 administered to patients with heterozygous familial hypercholesterolemia, atherosclerotic cardiovascular disease, and uncontrolled hypercholesterolemia
IRAS ID
1005362
Contact name
Leslie Stolz
Contact email
Sponsor organisation
Verve Therapeutics, Inc.
Eudract number
2022-000749-33
Clinicaltrials.gov Identifier
Research summary
Verve Therapeutics is funding this research to learn more about VERVE-101 to see if it is safe and whether it lowers low-density lipoprotein cholesterol (LDL-C) in patients with heterozygous familial hypercholesterolemia (HeFH). HeFH is a genetic condition that causes extremely high levels of (LDL-C) in the blood. LDL-C is a type of cholesterol or “fat” that is present in the blood and is sometimes referred to as “bad” cholesterol. High LDL-C can cause a narrowing of the arteries, known as atherosclerosis, and increase the risk of suffering a heart attack or stroke. VERVE-101 is designed to lower the levels of LDL-C in the blood. It does this by making a change to the DNA in a gene called “PCSK9” to stop it from working. Stopping the PCSK9 gene from working is known to lower LDL-C levels. VERVE-101 is an investigational medication and is not yet approved for use by any health authorities. This is the first time VERVE-101 is being tested in humans. The goal of this study is to determine whether VERVE-101 is safe and whether it lowers blood levels of LDL-C. Participants will be in the study for approximately one year, but will be asked to participate in longer-term studies as well. The study is planned to be conducted at research centers in US, Europe, and New Zealand.
REC name
South Central - Oxford A Research Ethics Committee
REC reference
22/SC/0217
Date of REC Opinion
15 Sep 2022
REC opinion
Further Information Favourable Opinion