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

Sponsor

Pfizer (Industry)

Overall Status

Recruiting

CT.gov ID

NCT05981092

Collaborator

(none)

Enrollment

Locations

30.2

Anticipated Duration (Months)

3.2

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Cardiomyopathy, Dilated Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM)

Study Design

Study Type:

Observational

Anticipated Enrollment :

35 participants

Official Title:

A Prospective Multinational Study of the Natural History of Participants With BAG3 Mutation Associated Dilated Cardiomyopathy

Actual Study Start Date :

Oct 14, 2022

Anticipated Primary Completion Date :

Apr 19, 2025

Anticipated Study Completion Date :

Apr 19, 2025

Arms and Interventions

Arm	Intervention/Treatment
BAG3 DCM A single arm observational trial where all participants will undergo the same schedule of assessments.

Outcome Measures

Primary Outcome Measures

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

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 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 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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Key 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)

Key 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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Brigham & Women's Hospital	Boston	Massachusetts	United States	02115
2	Brigham and Women's Hospital	Boston	Massachusetts	United States	02115
3	Montefiore Medical Center(Hutchinson Metro Center)	Bronx	New York	United States	10461
4	Montefiore Medical Center(Medical Arts Pavilion)	Bronx	New York	United States	10467
5	Montefiore Medical Center	Bronx	New York	United States	10467
6	Narodowy Instytut Kardiologii Stefana Kardynala Wyszynskiego - Panstwowy Instytut Badawczy	Warszawa		Poland	04-628
7	Hospital Universitario Puerta de Hierro Majadahonda	Majadahonda	Madrid	Spain	28222
8	Hospital Clinico Universitario Virgen de La Arrixaca	El Palmar	Murcia	Spain	30120
9	CHUAC-Complejo Hospitalario Universitario A Coruña	A Coruna		Spain	15006
10	Hospital Universitario 12 de Octubre	Madrid		Spain	28041
11	The Royal Brompton and Harefield Hospitals	Harefield	London	United Kingdom	UB9 6JH