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

NCT05981092 · tracked via the Priya Life Science Netherlands tracker

Sponsor

Alexion Pharmaceuticals, Inc

Phase

Observational

Started

2022-10-14

Last updated

2026-06-12

Condition(s) studied

Cardiomyopathy, DilatedBcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM)

Study 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.

Eligibility

Sex

ALL

Min age

18 Years

Max age

—

Healthy volunteers

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

Primary outcome measure(s)

Determine baseline of cardiac structure and function in BAG3 associated DCM. — 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. — 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.

Trial sites (15)

Facility	City	Region
Research Site	Palo Alto	California
Research Site	Aurora	Colorado
Research Site	Boston	Massachusetts
Research Site	Rochester	Minnesota
Research Site	The Bronx	New York
Research Site	Philadelphia	Pennsylvania
Research Site	Amsterdam	Amsterdam
Research Site	Maastricht	Maastricht
Research Site	Warsaw	Warszawa
Research Site	A Coruña	A Coruna
Research Site	Madrid	Madrid
Research Site	Majadahonda	Madrid
Research Site	El Palmar	Murcia
Research Site	Harefield	London
Research Site	London	London

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Study of ALXN2220 Versus Placebo in Adults With ATTR-CM Active, not 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 NCT05981092 on ClinicalTrials.gov ↗ ← All trials in the Netherlands