A PROSPECTIVE MULTINATIONAL STUDY OF THE NATURAL HISTORY OF PARTICIPANTS WITH BAG3 MUTATION ASSOCIATED DILATED CARDIOMYOPATHY
试验速览
- 阶段
- 不适用
- 状态
- 进行中（未招募）
- 入组人数
- 35
- 试验地点
- 16
- 主要终点
- Determine baseline of cardiac structure and function in BAG3 associated DCM.
研究概览
简要总结
The purpose of this study is to learn about the natural progression of DCM (dilated cardiomyopathy) caused by BAG3 gene mutations. DCM is a condition as the heart muscle is weakened and the heart becomes enlarged. This makes it hard for the heart to pump enough blood for the body.
The study is seeking up to about 35 participants who have:
- BAG3 mutation (change in the gene) that causes or is likely to cause dilated cardiomyopathy
- NYHA (New York Heart Association) Class I-IV at screening (Stage B-D)
- Left Ventricular Ejection Fraction less than or equal to 50% (meaning reduced heart function)
All participants in this study will receive their usual treatment. The investigators will observe the natural progression of people who have BAG3 DCM. This will help the investigators better understand the disease and aid in future research.
Participants will take part in this study for three years. During this time, participants will visit the site at least 8 times (about every 3 months for the first year and annually during year 2 and three). Participants will undergo study procedures and give information about their health. These procedures will include a physical exam, cardiac magnetic resonance imaging, echocardiography, ECG monitoring, activity monitoring, cardiopulmonary exercise testing, and blood tests. Participants will answer questions about health and quality of life. The study team will also call participants about 1 time over the phone.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Documented BAG3 mutation that causes or is likely to cause dilated cardiomyopathy
- •Heart failure Stage B-D, New York Heart Association (NYHA) Class I-IV
- •Left Ventricular Ejection Fraction ≤50% (i.e., Reduced Heart Function)
排除标准
- •Acute decompensated heart failure within 1 month prior to enrollment.(such as hospitalization)
- •Any of the following within 3 months prior to screening: myocardial infarction (MI), cardiac surgical procedures (other than for pacemaker/ICD/CRT-defibrillator [CRT-D] implantation), acute coronary syndrome, or hospitalization for cardiac arrhythmia.
- •History of heart transplantation
- •eGFR <30 mL/min/1.73 m2 (significantly impaired kidney function)
- •Noncardiac condition that limits lifespan to <1 year.
- •Presence of other form(s) of cardiomyopathy contributing to heart failure
- •Previous administration with an investigational drug within 30 days (or as determined by the local requirement).
- •No more than 3 first-degree members of the same family who are already participating in the study
结局指标
主要结局
Determine baseline of cardiac structure and function in BAG3 associated DCM.
时间窗: Baseline
Baseline measures of left ventricular volumes at end systole and diastole using imaging at baseline.
Determine changes over time in cardiac structure and function in BAG3 associated DCM.
时间窗: 3 years
Changes over time of left ventricular volumes at end systole and diastole using imaging over the course of 3 years follow up time.
Determine baseline of cardiac structure and function in BAG3 associated DCM.
时间窗: Baseline
Baseline measures of left ventricular volumes at end systole and diastole using imaging at baseline.
次要结局
- Change from Baseline in high sensitivity troponin I (hsTNI) levels(Baseline to year 3)
- Change from Baseline in high sensitivity troponin T (hsTNT) levels(Baseline to year 3)
- Change from Baseline in N-terminal Prohormone of Pro-Brain-Type Natriuretic peptide (NT-proBNP) levels(Baseline to year 3)
- Change from baseline in Measures of Oxygen Capacity(Baseline to year 3)
- Change from Baseline in Scores of Kansas City Cardiomyopathy Questionnaire (KCCQ) domains(Baseline to year 3)
- Change from Baseline in Scores of Patient Global Impression of Severity (PGI-S)(Baseline to year 3)
- Change from Baseline in Scores of Patient Global Impression of Change (PGI-C)(Baseline to year 3)
- Define the rate of cardiac status changes over time.(3 years)