The goal of this international observational study is to learn about the natural history of Dilated Cardiomyopathy (DCM) arising from pathogenic BAG3 variants in adult patients ≥18 years of age.
This is an observational study with both retrospective and prospective data collection. The study is designed to describe the natural history of BAG3-DCM including the signs and symptoms, key clinical events, and impact of the disease on quality of life as managed with the current standard of care. A hybrid (retrospective and prospective data collection) approach is being used to generate robust and longitudinal data.
Study Type
OBSERVATIONAL
Enrollment
30
Cardiac structure and function
Evaluate cardiovascular health as assessed by cardiac biomarkers and the occurrence of clinical outcomes related to the cardiovascular system.
Time frame: 48 months
NYHA Classification
Evaluation over duration of follow up
Time frame: 48 months
Change in arrhythmias or risk factor for ventricular arrhythmias
Evaluate changes in health status as assessed by occurrence of clinical outcomes
Time frame: 48 months
Heart rhythm and rate monitoring measures
Evaluation of change over duration of follow up
Time frame: 48 months
Cardiac biomarkers and blood proteomics
Evaluation of change over duration of follow up
Time frame: 48 months
Evaluate patient reported outcomes and quality of life measures
Evaluate patient reported outcomes and quality of life measures using validated questionnaire.
Time frame: 48 months
Evaluate changes in health status
Evaluate changes in health status as assessed by occurrence of clinical outcomes
Time frame: 48 months
Event free survival
Evaluation over duration of follow up
Time frame: 48 months
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Anti-AAV9 titer
Change in antibody assay findings over time
Time frame: 48 months
Tissue expression of BAG3 protein and DCM features
Change in protein expression and histopathologic features
Time frame: 48 months