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

Active, not recruiting

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

• Registration Number: NCT05981092
• Lead Sponsor: Alexion Pharmaceuticals, Inc.

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 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2022-10-14
• Study First Submitted: 2023-06-14
• Study First Submitted QC: 2023-07-31
• Study First Posted: 2023-08-08
• Status Verified: 2024-08
• Last Update Submitted: 2024-08-05
• Last Update Posted: 2024-08-06
• Primary Completion: 2026-09-23
• Study Completion: 2026-09-23

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: All
• Target Recruitment: 35

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)

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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

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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.	1 year	Changes over time of left ventricular volumes at end systole and diastole using imaging over the course of one year follow up time.

Secondary Outcome Measures

Name	Time	Method
Changes in high sensitivity troponin T (hsTNT) levels	Baseline to 1 year	HsTNT will be collected at baseline through 1 year to observe the natural history (baseline and variance) of the biomarker and the correlation of hsTNT to severity of clinical disease progression.
Changes in high sensitivity troponin I (hsTNI) levels	Baseline to 1 year	HsTNI will be collected at baseline through 1 year to observe the natural history (baseline and variance) of the biomarker and the correlation of hsTNI to severity of clinical disease progression.
Changes in N-terminal Prohormone of Pro-Brain-Type Natriuretic peptide (NT-proBNP) levels	Baseline to 1 year	NT-proBNP will be collected at baseline through 1 year to observe the natural history (baseline and variance) of the biomarker and the correlation of NT-proBNP to severity of clinical disease progression.

Trial Locations

Locations (1)

Research Site; 🇬🇧 London, United Kingdom