A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Dis… (NCT05981092) | Clinical Trial Compass
Active — Not RecruitingNot Applicable
A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM)
United States, Netherlands, Poland35 participantsStarted 2022-10-14
Plain-language summary
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.
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:
* 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)
Exclusion Criteria:
* 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
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
1
Determine baseline of cardiac structure and function in BAG3 associated DCM.
Timeframe: Baseline
2
Determine changes over time in cardiac structure and function in BAG3 associated DCM.