A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM)

Running, not enrolling

Conditions studied: Cardiomyopathy, Dilated, Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM)

In brief

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.

Key facts

Study ID

NCT05981092

Run by

Alexion Pharmaceuticals, Inc.

People needed

35

Starts

2022-10-14

Expected to finish

2027-08-12

Last updated by the study team

2026-06-12

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• 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)

You may not qualify if…

• 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

Where it is running

• Research Site — Palo Alto, California, United States
• Research Site — Aurora, Colorado, United States
• Research Site — Boston, Massachusetts, United States
• Research Site — Rochester, Minnesota, United States
• Research Site — The Bronx, New York, United States
• Research Site — Philadelphia, Pennsylvania, United States
• Research Site — Amsterdam, Amsterdam, Netherlands
• Research Site — Maastricht, Maastricht, Netherlands
• Research Site — Warsaw, Warszawa, Poland
• Research Site — A Coruña, A Coruna, Spain
• Research Site — Madrid, Madrid, Spain
• Research Site — Majadahonda, Madrid, Spain
• Research Site — El Palmar, Murcia, Spain
• Research Site — Harefield, London, United Kingdom
• Research Site — London, London, United Kingdom

Full record on ClinicalTrials.gov

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