Clinical Trial

Disease: Familial Hypercholesterolemia, FH, (NCT06112327)

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.

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:
Long-term Follow-up Study of Investigational Gene-editing Therapies in Participants With or at High Risk for Cardiovascular Disease


Clinical Operations at Verve Therapeutics

Phone Number: +1 781-970-6833




New Zealand

Auckland, New Zealand
Christchurch, New Zealand

United Kingdom

London, United Kingdom

Study start:
Apr. 1, 2024
44 participants
Gene editing method:
Base editing
Type of edit:
Gene knock-out
Proprotein Convertase Subtilisin-Kexin Type 9 (PCSK9)
Delivery method:
Lipid-based nanoparticle (LNP) - In-vivo
IND Enabling Pre-clinical
Phase I Safety
Phase II Safety and Dosing
Phase III Safety and Efficacy

Status: Not yet recruiting


LTF-001 is a long-term follow-up study of participants who received an investigational gene-editing therapy developed by the sponsor to evaluate the long-term effects of the investigational therapy. Participants will be followed for a total of 15 years after the first administration of the gene-editing therapy, including time in both the interventional study and study LTF-001.

Last updated: Feb. 11, 2024
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