CLARITY
Research type
Research Study
Full title
Cellular and Molecular Insights into Hypertrophic Cardiomyopathy using Advanced Cardiac Magnetic Resonance Imaging and Targeted Therapies
IRAS ID
349049
Contact name
Marc Dweck
Contact email
Sponsor organisation
University of Edinburgh
Duration of Study in the UK
3 years, 0 months, 1 days
Research summary
Hypertrophic cardiomyopathy (HCM) is the most common inherited heart muscle disease. It causes abnormal thickening of the heart muscle. Some patients will remain asymptomatic and live a normal life expectancy, whilst others will experience breathlessness, chest pain and heart rhythm abnormalities. In severe cases, patients can develop progressive heart failure, or die suddenly.
Despite ongoing research, there is no cure yet for this disease and current treatments focus on improving symptoms. Because we do not fully understand the disease process, we lack methods of identifying patients at highest risk of developing severe disease. This is a major unmet clinical need, not only to refine selection for implantable cardio-defibrillator therapy, but also because new drug and gene modifying therapies are emerging. Through our study, we propose to understand the underlying cellular mechanisms in hypertrophic cardiomyopathy using advanced cardiac imaging tools at various disease stages. We will investigate how these processes change over time and how they co-relate with blood markers of heart muscle strain. This will allow us to better identify high-risk patients. As these scans will also be carried out on patients who receive the approved study drug mavacamten, we will be able to observe any other effects on the heart muscle at a cellular level, as a result of how the drug works. This will help improve our overall understanding of this condition and current treatments.
Participants with hypertrophic cardiomyopathy within NHS Lothian will be recruited and undergo a range of investigations during study visits, including cardiac PET-MRI scans, echocardiogram, ECG, walking test and blood tests alongside a clinical assessment by the study doctor. Half of the participants will be allocated to receive mavacamten for 12 months. All participants will then undergo repeat investigations at 12 months and findings will be compared between both groups.
REC name
North of Scotland Research Ethics Committee 2
REC reference
25/NS/0021
Date of REC Opinion
18 Mar 2025
REC opinion
Further Information Favourable Opinion