Status:

ACTIVE_NOT_RECRUITING

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

Lead Sponsor:

Alexion Pharmaceuticals, Inc.

Conditions:

Cardiomyopathy, Dilated

Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM)

Eligibility:

All Genders

18+ years

Brief 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 beco...

Eligibility Criteria

Inclusion

  • 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

  • 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

Key Trial Info

Start Date :

October 14 2022

Trial Type :

OBSERVATIONAL

Allocation :

ACTUAL

End Date :

August 12 2027

Estimated Enrollment :

35 Patients enrolled

Trial Details

Trial ID

NCT05981092

Start Date

October 14 2022

End Date

August 12 2027

Last Update

December 11 2025

Active Locations (15)

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Page 1 of 4 (15 locations)

1

Research Site

Palo Alto, California, United States, 94305

2

Research Site

Aurora, Colorado, United States, 80045

3

Research Site

Boston, Massachusetts, United States, 02115

4

Research Site

Rochester, Minnesota, United States, 55905