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NCT05981092

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

Active, enrolled Last updated 11 December 2025
What this trial tests

trial in Cardiomyopathy, Dilated in 35 participants. Participants enrolled and being followed up; not accepting new ones.

Timeline
14 October 2022
Primary endpoint
12 August 2027
12 August 2027

Quick facts

Lead sponsorAlexion Pharmaceuticals, Inc.
StatusActive, enrolled
Study typeOBSERVATIONAL
Enrollment35
Start date14 October 2022
Primary completion12 August 2027
Estimated completion12 August 2027
Sites15 locations across Netherlands, United Kingdom, Poland, United States, Spain

Conditions studied

Sponsor

Alexion Pharmaceuticals, Inc. — full company profile →

Who can join

18 and older, any sex, with Cardiomyopathy, Dilated or Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM). Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

  1. Pathophysiology of dilated cardiomyopathy: from mechanisms to precision medicine.
    Gigli M, Stolfo D, Merlo M, Sinagra G, et al · · 2025 · cited 35× · PMID 39394525 · DOI 10.1038/s41569-024-01074-2

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Other recruiting trials for Cardiomyopathy, Dilated

Currently open trials in the same condition.

Other Alexion Pharmaceuticals, Inc. trials

Trials by the same sponsor.

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Data sources for this page

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT05981092.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing