A Study About The Natural History In Adults With BAG3 Dilated Cardiomyopathy (a Type Of Heart Disease) (BAG3 DCM)

Overview

About this study

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 one year. During this time, participants will visit the site at least 4 times (about every 3 months). 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.

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. There is no guarantee that every individual who qualifies and wants to participate in a trial will be enrolled. Contact the study team to discuss study eligibility and potential participation.

Inclusion Criteria:

* Documented BAG3 mutation that causes or is likely to cause dilated cardiomyopathy
* New York Heart Association (NYHA) Class I-IV at screening (Stage B-D)
* 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

Note: Other protocol defined Inclusion/Exclusion Criteria may apply.

Eligibility last updated 12/4/2025. Questions regarding updates should be directed to the study team contact.

Participating Mayo Clinic locations

Study statuses change often. Please contact the study team for the most up-to-date information regarding possible participation.

Mayo Clinic Location	Status	Contact
Rochester, Minn. Mayo Clinic principal investigator Naveen Pereira, M.D.	Closed for enrollment	Contact information: Brendan Mark (507) 284-4284 Mark.Brendan@mayo.edu

Mayo Clinic Location

Status

Contact

Rochester, Minn.

Mayo Clinic principal investigator

Naveen Pereira, M.D.

Closed for enrollment

Contact information:

Brendan Mark

(507) 284-4284

Mark.Brendan@mayo.edu

More information

Publications

Publications are currently not available

Clinical Trials