Personalised care for young people with heart muscle disease
Research type
Research Study
Full title
Personalised care for young people with heart muscle disease
IRAS ID
346530
Contact name
Gabrielle Norrish
Contact email
Sponsor organisation
University College London
Clinicaltrials.gov Identifier
Z6364106/2025/09/38, Data protection number
Duration of Study in the UK
6 years, 0 months, 1 days
Research summary
Dilated cardiomyopathy (DCM) is the most common form of heart muscle disease presenting in childhood and is defined as an enlarged heart with reduced pumping ability. It is associated with an increased risk of death, abnormal heart rhythms and need for heart transplantation. In adults, the underlying cause has been shown to be important for how the heart looks (‘the phenotype’) and long-term prognosis. Adults benefit from specific medicines and prediction tools depending on the underlying
cause allowing personalisation of care. In contrast, during childhood the underlying cause is often unknown, and cause-specific outcomes have not been explored. These evidence gaps prevent personalisation of care for young patients. This study will take advantage of the largest group of children with dilated cardiomyopathy in the world and exciting machine learning approaches to answer 3 important
questions; (1) Can we predict the cause of disease by the way the heart looks?(2) Can we predict how the disease will change over time? (3) Can we predict children’s response to commonly used medicines? Together this work will allow clinicians to personalise care for children with DCM for the first-time improving outcomes.REC name
North West - Greater Manchester South Research Ethics Committee
REC reference
25/NW/0375
Date of REC Opinion
18 Dec 2025
REC opinion
Favourable Opinion