Natural History Dilated Cardiomyopathy Associated with BAG3 Mutations
Research type
Research Study
Full title
A PROSPECTIVE MULTINATIONAL STUDY OF THE NATURAL HISTORY OF PARTICIPANTS WITH BAG3 MUTATION ASSOCIATED DILATED CARDIOMYOPATHY
IRAS ID
321187
Contact name
Perry Elliott
Contact email
Sponsor organisation
Alexion, AstraZeneca Rare Disease
Duration of Study in the UK
1 years, 3 months, 20 days
Research summary
This is a natural history study in dilated cardiomyopathy associated with a particular type of mutation in the BAG3 gene. BAG3 dilated cardiomyopathy is a chronic, debilitating, and progressive illness. Dilated cardiomyopathy is a type of heart muscle disease that causes the heart chambers (ventricles) to thin and stretch, growing larger, and making it harder for the heart to pump blood to the rest of the body. This can cause fatigue and shortness of breath, and is a common cause of heart failure. This study will explore the natural progression of dilated cardiomyopathy caused by BAG3 gene mutations. This will facilitate future interventional trials with the aim of targeting the defect underlying BAG3 dilated cardiomyopathy, in order to give lasting improvements in functional status and symptoms as well as to reduce overall mortality.
Participants will be asked to take part in the study for about 52 weeks and wear an Actigraph CentrePoint Insight Watch device throughout. Participants will wear an ECG patch for 1-2 weeks at home.
REC name
London - Bloomsbury Research Ethics Committee
REC reference
22/LO/0779
Date of REC Opinion
5 Jan 2023
REC opinion
Further Information Favourable Opinion