Ireland
--:--IST
Latest
Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide The €100K Reality Check: Why a Six-Figure Pharma Salary in Ireland Feels Different Than in Switzerland or Germany Ireland's €93.8 Billion Non-EU Pharma Export Engine: 2025 Eurostat Trade Data and Economic Impact Astellas Expands Its 330 Million Euro Tralee Biopharma Facility with a Second Aseptic Filling Line to Double Drug-Product Capacity Xeolas Pharmaceuticals Opens 158,000 Sq Ft State-of-the-Art Baldoyle Facility to Scale Specialty Medicine Manufacturing Priya Life Science Partners with Fleming for the 9th Annual Corporate Compliance & Transparency in Life Sciences Summit in Zurich Ireland Has the Capital and the Lessons: Digital Project Management Is How They Become Delivery Dunbar Pharma Brings First Plant-Derived Dronabinol API to UK Market Through IPS Pharma Leveraging Priya Life Science as a Data Tracker: The Ultimate Use Case & Career Guide The €100K Reality Check: Why a Six-Figure Pharma Salary in Ireland Feels Different Than in Switzerland or Germany Ireland's €93.8 Billion Non-EU Pharma Export Engine: 2025 Eurostat Trade Data and Economic Impact
Recruiting Observational

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

NCT07646600 · tracked via the Priya Life Science China tracker
Phase
Observational
Started
2026-08-18
Last updated
2026-10-22

Condition(s) studied

Dilated Cardiomyopathy

Investigational drug(s) / intervention(s)

Observational, None intervention

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

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
Inclusion Criteria: * Participants must meet all of the following criteria to be eligible for inclusion: 1. Age ≥18 years at screening, male or female; 2. Presence of a BAG3 pathogenic or likely pathogenic mutation interpreted according to the American College of Medical Genetics and Genomics (ACMG) guidelines; 3. Heart failure (HF) Stage B or C, New York Heart Association (NYHA) functional class I-III; 4. 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; 5. Willing and able to comply with all scheduled visits, laboratory tests, and other study procedures; 6. Capable of providing signed informed consent, including compliance with the requirements and restrictions listed in the informed consent form and this protocol. Exclusion Criteria: * Participants meeting any of the following criteria will be excluded: 1. Acute decompensated heart failure within 1 month prior to enrollment; 2. Any of the following within 3 months prior to screening: * Myocardial infarction * Cardiac surgical procedures (excluding pacemaker/implantable cardioverter defibrillator/cardiac resynchronization therapy implantation) * Implantable cardioverter defibrillator (ICD) implantation * Acute coronary syndrome * Hospitalization for cardiac arrhythmia 3. History of heart transplantation; 4. Estimated glomerular filtration rate (eGFR) \<30 mL/min/1.73 m² (calculated using the Chronic Kidney Disease Epidemiology Collaboration formula); 5. Active malignancy or malignancy diagnosed within 3 years prior to screening, except for: * Surgically cured in situ malignancies * Surgically cured early-stage cancers (including breast, prostate, skin \[basal cell carcinoma, squamous cell carcinoma\], thyroid, or cervical cancer) * Other early-stage malignancies with an expected 2-year recurrence rate ≤20%, approved by the Medical Monitor 6. Non-cardiac condition limiting life expectancy to \<1 year; 7. Presence of any of the following cardiac conditions: * Other forms of cardiomyopathy contributing to heart failure (e.g., inflammatory or infiltrative cardiomyopathy) * Clinically significant cardiac anatomic abnormalities (e.g., left ventricular aneurysm) * Significant coronary artery disease (e.g., prior revascularization, exercise-induced angina) * Uncorrected, hemodynamically significant (moderate to severe) primary structural valvular disease not due to heart failure (investigator's judgment) 8. Any severe concurrent disease (e.g., active systemic infection) that, in the investigator's judgment, would make the participant unsuitable for study participation; 9. Any medical or psychiatric condition that may increase study risk, including: * Active or recent (within 1 year) suicidal ideation/behavior * Laboratory abnormalities * Other conditions deemed unsuitable by the investigator 10. Administration of any investigational drug within 30 days prior to screening; 11. Prior gene or cell therapy treatment; 12. Anticipated need for cardiac transplantation, left ventricular assist device implantation, or other cardiac surgery within the next 3 months (investigator's assessment); 13. More than 3 first-degree relatives from the same family enrolled concurrently (excluding the participant).

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)

FacilityCityRegionStatus
Research Site Beijing China Recruiting
Research Site Beijing China Not Yet Recruiting
Research Site Hubei China Not Yet Recruiting
Official registry record

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