Observational Study of Natural History of BAG3 Gene Mutation-Associated Dilated Cardiomyopathy in Chinese Adults
Condition(s) studied
Investigational drug(s) / intervention(s)
Observational, None intervention: Observational, None intervention
Study summary
This is a multicentre, prospective cohort study designed to elucidate the natural history of dilated cardiomyopathy (DCM) associated with specific gene mutations in Chinese patients. By establishing a cohort of patients with inherited cardiomyopathy, the study will collect comprehensive data, including demographic characteristics, disease history, clinical features, prior treatments, current treatment patterns, clinical outcomes, and patient-reported outcomes (PROs). The objective is to describe the disease profile, current diagnostic and therapeutic landscape, clinical progression, and prognosis of this specific patient population in China. The data generated will provide a foundational basis for deepening the understanding of disease progression, identifying unmet clinical needs, and exploring potential surrogate endpoints for future clinical trials in drug development. The study plans to enroll 10 eligible patients with BCL-2-associated athanogene 3 (BAG3)-associated inherited DCM from 3 research sites across China.
Eligibility
Primary outcome measure(s)
- changes in cardiac imaging parameters, left ventricular ejection fraction (LVEF) and global longitudinal strain (GLS) — Baseline (at Day 1 post-enrollment), 12 months and 24 months
Primary endpoints include changes from baseline in cardiac structure and function parameters, left ventricular ejection fraction (LVEF) and global longitudinal strain (GLS), at Month 12 and subsequent follow-ups. Changes in the above imaging assessment results are presented as continuous numerical variables and the results of changes expressed as percentages. - changes in cardiac imaging parameters, left ventricular end diastolic diameter (LVEDD) — Baseline (at Day 1 post-enrollment), 12 months and 24 months
Primary endpoints include changes from baseline in cardiac structure and function parameters, left ventricular end diastolic diameter (LVEDD), at Month 12 and subsequent follow-ups. Changes in the above imaging assessment results are presented as continuous numerical variables and the results of changes expressed as percentages. - changes in cardiac imaging parameters, left ventricular end systolic volume index (LVESVi) and left ventricular end diastolic volume index (LVEDVi) — Baseline (at Day 1 post-enrollment), 12 months and 24 months
Primary endpoints include changes from baseline in cardiac structure and function parameters, left ventricular end systolic volume index (LVESVi) and left ventricular end diastolic volume index (LVEDVi), at Month 12 and subsequent follow-ups. Changes in the above imaging assessment results are presented as continuous numerical variables and the results of changes expressed as percentages. - changes in cardiac imaging parameters, left ventricular mass index (LVMi) — Baseline (at Day 1 post-enrollment), 12 months and 24 months
Primary endpoints include changes from baseline in cardiac structure and function parameters, left ventricular mass index (LVMi), at Month 12 and subsequent follow-ups. Changes in the above imaging assessment results are presented as continuous numerical variables and the results of changes expressed as percentages.
Trial sites (3)
| Facility | City | Region | Status |
|---|---|---|---|
| Research Site | Beijing | China | Recruiting |
| Research Site | Beijing | China | Not Yet Recruiting |
| Research Site | Hubei | China | Not Yet Recruiting |
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This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT07646600 on ClinicalTrials.gov ↗ ← All trials in China