Clinical Trial

Disease: Cardiovascular Disease or Calcific Aortic Valve Stenosis, (ACTRN12623001095651p)

Disease info:

Atherosclerotic heart disease refers to heart diseases caused by atherosclerosis. Atherosclerosis is caused by the buildup of plaque inside arteries. Plaque is made up of deposits of cholesterol and other substances. Over time, plaque can harden and narrow the arteries, limiting oxygen-rich blood flow and causing heart diseases such as coronary artery disease (CAD), carotid artery disease, and peripheral arterial disease.

CAD, also known as ischemic heart disease, is the most common type of heart disease and the leading cause of death in the United States. CAD involves the blockage of arteries that supply blood to heart muscle, leading to heart attacks. CAD may weaken the heart muscle over time and contribute to heart failure and arrhythmia (irregular heartbeat). 

Carotid artery disease involves blockage of the arteries that supply blood to the brain and can result in a stroke.

Peripheral arterial disease involves blockage of arteries in the arms, legs and pelvis, and can lead to numbness, pain and infections.

Aortic valve stenosis (AVS) is a type of heart valve disease caused by narrowing of the valve between the lower left heart chamber and the aorta. The aorta is the main artery that carries oxygen-rich blood out of the heart to the rest of the body. Blood flows out of the heart and into the aorta through the aortic valve. AVS can range from mild to severe.
As the aortic valve narrows, the left ventricle has to work harder to pump blood out through the valve. To do this, the muscles in the ventricle walls become thicker. The blood may sometimes back up into the lungs, and severe aortic stenosis can greatly limit the amount of blood reaching other parts of the body. AVS most commonly occurs later in life, but can sometimes be congenital, affecting children from birth. Narrowing of the aortic valve is most often due to a buildup of calcium deposits. This is called calcific aortic stenosis, or calcific aortic valve stenosis. The risk of calcium buildup is increased in individuals born with abnormal aortic or bicuspid valves.
Rheumatic fever has been linked with causing aortic valve stenosis. 

Many mutations in diverse genes have been linked with cardiovascular diseases. The exact mutations implicated and their impact is disease-dependent. A genetic variation in the LPA gene that increases Lp(a) levels has been implicated in increasing the risk of coronary artery disease, AVS, and other heart diseases.


Every year in the United States, over 650,000 people die from heart disease. It is the leading cause of death in the United States in both men and women. Aortic stenosis occurs in about 2% of people over 65 years of age.
Official title:
A Phase 1 Open-label, Multicenter, First-in-human, Ascending Single Dose Study Evaluating the Safety and Tolerability of a Lipid Nanoparticle Formulation of CRISPR–Guide RNA–Cas9 Nuclease (CTX320) for In Vivo Editing of the Apolipoprotein(a) Gene (LPA) in Subjects with Elevated Lipoprotein(a) and a History of Atherosclerotic Cardiovascular Disease or Calcific Aortic Valve Stenosis


Name: A/Prof David Sullivan
Phone: +61 295158832


South Australia, Australia

Western Australia, Australia

New Zealand

Study start:
Sep. 21, 2023
24 participants
Gene editing method:
Type of edit:
Gene knock-out
The apolipoprotein (a) component of Lp(a)
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: Active not recruiting


This is a single-arm, open-label, multicenter, ascending single-dose Phase 1 study. Subjects will receive a single dose of CTX320 via intravenous (IV) infusion. The duration of the infusion is expected to take 1 hour. Planned ascending doses levels will range from 0.1 mg/kg - 0.8 mg/kg. Participants will receive only one dose.

Last updated: Feb. 9, 2024
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