An observational study in Dilated Cardiomyopathy, sponsored by AstraZeneca. Recruiting at 3 sites in China. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-10-22.
Sponsored by AstraZeneca · Observational
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.
This is a multicentre, prospective, observational natural history study targeting Chinese adults with BAG3 DCM. Approximately 10 patients with BAG3 DCM will be enrolled from 3 research sites in China. Comprehensive clinical data, including demographic characteristics, medical history, clinical manifestations, treatment records, clinical outcomes, and PROs, will be collected. After completing the informed consent process, eligible patients will undergo screening and enrollment, planned to last 1 year. Patients with a confirmed diagnosis of BAG3 DCM will be directly enrolled; those initially diagnosed only with DCM will undergo genetic testing (e.g., whole-exome sequencing), and carriers of BAG3 mutations will be included upon confirmation. After enrollment, participants will undergo a comprehensive baseline assessment of clinical parameters (including imaging, cardiopulmonary function, and laboratory tests) and enter a subsequent 2-year follow-up evaluation period. Patient follow-up will continue until any of the following events occurs: death, study withdrawal, loss to follow-up (defined as failure to contact via three attempts including telephone, WeChat, medical record review, and contact with family members/treating physicians), early study termination/suspension, or completion of the scheduled 2-year follow-up period.
Medical history and cardiomyopathy treatment history at baseline (e.g., medications, surgical procedures, hospitalizations / resuscitation records) will be retrieved from patients' electronic medical records at enrollment. After enrollment, prospective data collection will include information from routine clinical practice, investigator-solicited clinical outcomes, and Patient-reported outcomes (PROs). An Electronic Data Capture (EDC) system will be used for data collection. All enrolled cardiomyopathy patients will be followed at regular intervals until the end of the observation follow-up period.
This study will collect electronic medical record data from cardiomyopathy patients at 3 research sites in China. Data sources include hospital electronic health records, medical records, and patient-reported outcome questionnaires. Routine clinical data (including but not limited to baseline information, assessments, and treatment records) will be collected during routine clinical visits or via remote questionnaires. Follow-up frequency is set at baseline (Day 1, V1), Month 3 (V2), Month 6 (V3), Month 12 (V4), Month 18 (V5), and Month 24 (V6). All data will be collected and managed using an EDC system.
255 studies on the registry are indexed under Cardiomyopathy, Dilated; 66 are open to participants now.
This study's planned enrollment of 10 is below the median of 245 across 96 observational studies indexed under Cardiomyopathy, Dilated.
Browse Cardiomyopathy, Dilated studies →AstraZeneca is the lead sponsor of 3,499 studies on the registry; 270 are open to participants now.
Of its 368 completed or terminated interventional studies of FDA-regulated products, 178 (48%) have results posted.
Counted across the registry records on this site, refreshed daily.
BAG3 DCM
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Participants must meet all of the following criteria to be eligible for inclusion:
Exclusion Criteria:
Participants meeting any of the following criteria will be excluded:
Any of the following within 3 months prior to screening:
Active or malignancy diagnosed within 3 years prior to screening, except for:
Presence of any of the following cardiac conditions:
Any medical or psychiatric condition that may increase study risk, including:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
LVEF (left ventricular ejection fraction)
The share of blood the heart’s main pumping chamber (the left ventricle) pushes out with each beat, measured by an echocardiogram (heart ultrasound) or a MUGA scan. About 50–70% is normal.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GFR and eGFR (kidney filtering)
GFR (glomerular filtration rate) is how much blood the kidneys filter each minute. It is usually estimated from a blood test — eGFR, by formulas such as CKD-EPI or MDRD — and some studies ask for it to be measured directly instead. Usually given in mL/min/1.73 m².
Check the method, the units and the minimum the study states. A value of 90 or above is in the normal-or-high range, but kidney damage can show in other tests too.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Cancer (malignancy)
Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.
Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Life expectancy
The study doctor’s estimate of how long someone is likely to live. Criteria use it so that people can stay in the study long enough for it to answer its question.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Clinically significant
Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Approximately 10 patients with BAG3 DCM will be enrolled from 3 research sites in China. Comprehensive clinical data, including demographic characteristics, medical history, clinical manifestations, treatment records, clinical outcomes, and PROs, will be collected.
Other: Observational, None intervention
Observational, None intervention
changes in cardiac imaging parameters, left ventricular ejection fraction (LVEF) and global longitudinal strain (GLS)
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.
Time frame: Baseline (at Day 1 post-enrollment), 12 months and 24 months
changes in cardiac imaging parameters, left ventricular end diastolic diameter (LVEDD)
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.
Time frame: 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)
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.
Time frame: Baseline (at Day 1 post-enrollment), 12 months and 24 months
changes in cardiac imaging parameters, left ventricular mass index (LVMi)
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.
Time frame: Baseline (at Day 1 post-enrollment), 12 months and 24 months
Circulating Biomarkers
Descriptive summaries of High-sensitivity cardiac troponin I (hs-cTnI), High-sensitivity cardiac troponin T (hs-cTnT), and N-terminal pro b-type natriuretic peptide (NT-proBNP), and other circulating biomarkers level at baseline and each follow-up visit, including absolute and relative percentage changes from baseline. All of the above test parameters are measured in ng/L, and the changes are treated as continuous numerical variables.
Time frame: Baseline (at Day 1 post-enrollment), 3 months, 6 months, 12 months, 18 months, and 24 months
Peak oxygen capacity (VO₂) measured by cardiopulmonary exercise testing (CPET)
Peak oxygen capacity (VO₂) measured by cardiopulmonary exercise testing (CPET) will be summarized at baseline and each time point, along with absolute and relative changes over time.
Time frame: Baseline (at Day 1 post-enrollment) and 12 months.
Kansas City Cardiomyopathy Questionnaire-23 (KCCQ-23)
Changes from baseline in KCCQ-23 overall and domain scores will be described using absolute and relative changes. Raw summary scores are converted to a 0-100 scale, with higher scores indicating better health status.
Time frame: Baseline (at Day 1 post-enrollment), 3 months, 6 months, 12 months, 18 months, and 24 months
EuroQol 5-Dimension 5-Level Health Scale (EQ-5D-5L)
Changes from baseline in EQ-5D-5L overall and domain scores will be described using absolute and relative changes. The EQ-5D was developed by the EuroQol Group to provide a simple, generic measure of health. The EQ-5D primarily consists of two parts: the EQ-5D descriptive system and the EQ-5D visual analogue scale (VAS). The score ranges from \<0 to 1. Higher scores men a better outcome.
Time frame: Baseline (at Day 1 post-enrollment), 3 months, 6 months, 12 months, 18 months, and 24 months
The medical outcomes study 36-item short-form health survey (SF-36)
Changes from baseline in SF-36 overall and domain scores will be described using absolute and relative changes. The questionnaire comprises 8 health domain scales. The raw scores for each health domain scale are converted to a 0-100 scale score and can then be transformed into norm-based T-scores (Mean = 50, Standard Deviation = 10) using scoring software provided by the developer.
Time frame: Baseline (at Day 1 post-enrollment), 3 months, 6 months, 12 months, 18 months, and 24 months
The Frequency and Rate of Notable Cardiac Status Changes
The cumulative number and frequency of notable cardiac status events will be reported. Notable cardiac status changes include but are not limited to: new or worsening signs/symptoms of fluid overload (e.g., edema, pulmonary congestion), new or worsening renal impairment (e.g., cardiorenal syndrome, acute renal failure), heart failure (HF) hospitalization, need for unscheduled intravenous diuretics, HF exacerbation, cardiovascular death, myocardial infarction (MI), hospitalization for unstable angina, coronary revascularization, stroke, new or worsening ventricular or supraventricular arrhythmias, sudden cardiac death (SCD), or SCD-equivalent events (e.g., appropriate implantable cardioverter-defibrillator \[ICD\] shock, sustained ventricular arrhythmia). Event rates per 100 patient-years with 95% confidence intervals will be calculated.
Time frame: Baseline (at Day 1 post-enrollment), 3 months, 6 months, 12 months, 18 months, and 24 months
Plan to share: Yes — Qualified researchers can request access to anonymized individual patient-level data from AstraZeneca group of companies sponsored clinical trials via the request portal Vivli.org. All requests will be evaluated as per the AZ disclosure commitment: https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure. "Yes", indicates that AZ are accepting requests for IPD, but this does not mean all requests will be approved.
Time frame: AstraZeneca will meet or exceed data availability as per the commitments made to the EFPIA/PhRMA Data-Sharing Principles. For details of our timelines, please refer to our disclosure commitment at https://astrazenecagrouptrials.pharmacm.com/ST/Submission/Disclosure.
Access criteria: When a request has been approved AstraZeneca will provide access to the anonymized individual patient-level data via secure research environment Vivli.org. A Signed Data Usage Agreement (non-negotiable contract for data accessors) must be in place before accessing requested information.
Data sharing details ↗ · vivli.org
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