Not Yet RecruitingNot Applicable

Observational Study of Natural History of BAG3 Gene Mutation-Associated Dilated Cardiomyopathy in Chinese Adults

China10 participantsStarted 2026-06-29

Plain-language 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.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Age ≥18 years at screening, male or female;
. Presence of a BAG3 pathogenic or likely pathogenic mutation interpreted according to the American College of Medical Genetics and Genomics (ACMG) guidelines;
. Heart failure (HF) Stage B or C, New York Heart Association (NYHA) functional class I-III;
. LVEF ≤50%. If LVEF is 45%-50% and the participant is classified as Stage B heart failure or NYHA Class I, N-terminal pro-B-type natriuretic peptide (NT-proBNP) must be ≥300 pg/mL. LVEF measurement must be based on echocardiography or cardiac magnetic resonance results within the past 12 months;
. Willing and able to comply with all scheduled visits, laboratory tests, and other study procedures;
. Capable of providing signed informed consent, including compliance with the requirements and restrictions listed in the informed consent form and this protocol.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

changes in cardiac imaging parameters, left ventricular ejection fraction (LVEF) and global longitudinal strain (GLS)

Timeframe: Baseline (at Day 1 post-enrollment), 12 months and 24 months

changes in cardiac imaging parameters, left ventricular end diastolic diameter (LVEDD)

Timeframe: Baseline (at Day 1 post-enrollment), 12 months and 24 months

changes in cardiac imaging parameters, left ventricular end systolic volume index (LVESVi) and left ventricular end diastolic volume index (LVEDVi)

Timeframe: Baseline (at Day 1 post-enrollment), 12 months and 24 months

changes in cardiac imaging parameters, left ventricular mass index (LVMi)

Timeframe: Baseline (at Day 1 post-enrollment), 12 months and 24 months

Trial details

NCT IDNCT07646600

SponsorAstraZeneca

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2029-06-29

View on ClinicalTrials.gov More Dilated Cardiomyopathy trials

Plain-language summary

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Age ≥18 years at screening, male or female;
. Presence of a BAG3 pathogenic or likely pathogenic mutation interpreted according to the American College of Medical Genetics and Genomics (ACMG) guidelines;
. Heart failure (HF) Stage B or C, New York Heart Association (NYHA) functional class I-III;
. LVEF ≤50%. If LVEF is 45%-50% and the participant is classified as Stage B heart failure or NYHA Class I, N-terminal pro-B-type natriuretic peptide (NT-proBNP) must be ≥300 pg/mL. LVEF measurement must be based on echocardiography or cardiac magnetic resonance results within the past 12 months;
. Willing and able to comply with all scheduled visits, laboratory tests, and other study procedures;
. Capable of providing signed informed consent, including compliance with the requirements and restrictions listed in the informed consent form and this protocol.