Clinical Trial

Disease: Familial Hypercholesterolemia, HeFH, or Premature Coronary Artery Disease, CAD, (NCT06164730)

Disease info:

Familial hypercholesterolemia (FH) is a condition associated with significantly elevated low-density lipoprotein (LDL) cholesterol (LDL-C) or "bad cholesterol" and an increased risk of early onset of coronary artery disease if not sufficiently treated. Most commonly, individuals have heterozygous familial hypercholesterolemia (HeFH), caused by a single DNA variant (alteration) for FH that they have inherited from one (affected) parent. In rare cases, an individual can have homozygous familial hypercholesterolemia (HoFH), which is caused by having two causal FH DNA variants, where one variant is inherited from each (affected) parent. Individuals with HoFH typically have a more severe form of disease.

Having FH greatly increases the risk of hardening of the arteries (atherosclerosis), which can lead to heart attacks, strokes and other vascular conditions. Untreated individuals with FH have a 20-fold increased risk for coronary artery disease (CAD).

FH is treatable and the associated cardiovascular disease is largely preventable with early and intensive treatment, using statins, additional drugs, and other means. Other non-statin medications include PCSK9 inhibitors, ezetimibe, and bempedoic acid. These are effective treatments for individuals with FH who have a persistently elevated LDL-C despite treatment with maximally tolerated statin therapy.

Early identification and treatment of individuals with FH is key to preventing cardiovascular disease. Underdiagnosis of FH is a problem in most countries as high cholesterol can be an invisible and undetected problem until it leads to coronary artery disease.

Individuals with premature coronary artery disease (CAD) face the early onset of cholesterol-induced blockages in their coronary arteries, placing them at a heightened risk of subsequent complications. Insufficient management of LDL-C levels in both individuals with familial hypercholesterolemia (HeFH) and those with premature CAD significantly elevates their lifetime susceptibility to cardiovascular events, such as heart attacks and sudden death.

Frequency:
The incidence of familial hypercholesterolemia (FH) is estimated at about 1/300 persons worldwide but is 7/100 in persons with premature ischemic heart disease (IHD) and founder effect such as French Canadians, Finns, and Afrikaners.
Official title:
Open-label, Phase 1b, Single Ascending Dose Study to Evaluate the Safety of VERVE-102 Administered to Patients With Heterozygous Familial Hypercholesterolemia or Premature Coronary Artery Disease Who Require Additional Lowering of Low-density Lipoprotein Cholesterol
Who:

Contact

Phone: 781-970-6833

Email: verve102clinicaltrials@vervetx.com 

Partners:
Locations:
Study start:
May. 1, 2024
Enrollment:
36 participants
Gene editing method:
Base editor
Type of edit:
Gene silence
Gene:
PCSK9 gene
Delivery method:
GalNAc-LNP (lipid nanoparticle) delivery technology - In-vivo
Indicator
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Not yet recruiting

Description

VT-10201 is an Open-label, Phase 1b, Single-ascending Dose Study That Will Evaluate the Safety of VERVE-102 Administered to Patients With Heterozygous Familial Hypercholesterolemia (HeFH) or Premature Coronary Artery Disease (CAD) Who Require Additional Lowering of LDL-C. VERVE-102 Uses Base-editing Technology Designed to Disrupt the Expression of the PCSK9 Gene in the Liver and Lower Circulating PCSK9 and LDL-C. This Study is Designed to Determine the Safety and Pharmacodynamic Profile of VERVE-102 in This Patient Population.

Last updated: May. 13, 2024
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