AZacitidine Treatment Effect on hypertrophic Cardiomyopathy (AZTEC)
Research type
Research Study
Full title
A trial to evaluate the safety and efficacy of treatment with azacitadine in patients with symptomatic non-obstructive hypertrophic cardiomyopathy (nHCM)
IRAS ID
1009388
Contact name
Lana Dixon
Contact email
Sponsor organisation
Belfast Health and Social Care Trust
ISRCTN Number
ISRCTN38776134
Research summary
Hypertrophic cardiomyopathy is an inherited condition that affects the hearts ability to pump blood around the body. It affects around 1 in 500 people in the UK and can lead to heart failure. For people with hypertrophic cardiomyopathy everyday tasks like walking upstairs or to the shops becomes impossible. New drugs are needed to improve the lives of people with hypertrophic cardiomyopathy to stop the disease getting worse.
In support of this, we have discovered that a process called DNA methylation, which affects how your genetic code is
expressed, can change in people with heart failure and other forms of heart disease. Pre-clinical studies have shown that
changes in DNA methylation in diseased hearts is reversible using low doses of a drug called azacitidine, which also
improves how the heart functions.This drug is used in other types of patients with certain cancers, but has not been used before to treat hypertrophic cardiomyopathy. The aim of this project is to test the drug in hypertrophic cardiomyopathy patients for the first time, through testing a range of low doses and to select a dose to study in more detail to provide evidence on how well it works to improve patient and heart health. The long
term benefits of this research could lead to the development of a new drug type for hypertrophic cardiomyopathy.The study will take place at the Belfast city Hospital and will involve receiving injections of the drug twice weekly for 16 weeks, blood samples will be taken as well as some physical exams including an Echocardiogram and Cardiac MRI. It is funded by both the British Heart Foundation (BHF) , Innovate UK and the Heart Trust Fund and will take three years to complete, although each participant will only be involved for the 16 weeks of treatment with a 4 week follow up.
REC name
North East - Tyne & Wear South Research Ethics Committee
REC reference
25/NE/0191
Date of REC Opinion
2 Dec 2025
REC opinion
Further Information Favourable Opinion