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

Cardiomyopathy, Dilated Clinical Trial

— BAG3 DCM  

Official title:

A PROSPECTIVE MULTINATIONAL STUDY OF THE NATURAL HISTORY OF PARTICIPANTS WITH BAG3 MUTATION ASSOCIATED DILATED CARDIOMYOPATHY

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05981092
• Other study ID #: C4981001
• Secondary ID: 2022-000398-20
• Status: Recruiting
• Start date: October 14, 2022
• Est. completion date: August 9, 2025

Study information

• Verified date: April 2024
• Source: Pfizer
• Contact: Pfizer CT.gov Call Center
• Phone: 1-800-718-1021
• Email: ClinicalTrials.gov_Inquiries[@]pfizer.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 35
• Est. completion date: August 9, 2025
• Est. primary completion date: August 9, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Cardiomyopathies
• Cardiomyopathy, Dilated
• Heart Diseases

Locations

Country	Name	City	State
Netherlands	Maastricht University Medical Center	Maastricht
Poland	Narodowy Instytut Kardiologii Stefana Kardynala Wyszynskiego - Panstwowy Instytut Badawczy	Warszawa
Spain	CHUAC-Complejo Hospitalario Universitario A Coruña	A Coruna
Spain	Hospital Clinico Universitario Virgen de La Arrixaca	El Palmar	Murcia
Spain	Hospital Universitario 12 de Octubre	Madrid
Spain	Hospital Universitario Puerta de Hierro Majadahonda	Majadahonda	Madrid
United Kingdom	The Royal Brompton and Harefield Hospitals	Harefield	London
United Kingdom	Barts Health NHS Trust, St Bartholomew's Hospital	London
United States	University of Colorado Anschutz Medical Campus, Fitzsimons Building	Aurora	Colorado
United States	University of Colorado Clinical and Translational Research Center	Aurora	Colorado
United States	University of Colorado Denver, Anschutz Medical Campus	Aurora	Colorado
United States	University of Colorado Hospital	Aurora	Colorado
United States	Brigham & Women's Hospital	Boston	Massachusetts
United States	Brigham and Women's Hospital	Boston	Massachusetts
United States	Montefiore Medical Center	Bronx	New York
United States	Montefiore Medical Center(Hutchinson Metro Center)	Bronx	New York
United States	Montefiore Medical Center(Medical Arts Pavilion)	Bronx	New York
United States	Division of Heart Failure and Transplantation(Hospital of the University of Pennsylvania)	Philadelphia	Pennsylvania
United States	Mayo Clinic - Rochester	Rochester	Minnesota
United States	Stanford University Healthcare/Stanford Health Care	Stanford	California

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Countries where clinical trial is conducted

United States,  Netherlands,  Poland,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Determine baseline of cardiac structure and function in BAG3 associated DCM.	Baseline measures of left ventricular volumes at end systole and diastole using imaging at baseline.	Baseline
Primary	Determine changes over time in cardiac structure and function in BAG3 associated DCM.	Changes over time of left ventricular volumes at end systole and diastole using imaging over the course of one year follow up time.	1 year
Secondary	Changes in high sensitivity troponin I (hsTNI) levels	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.	Baseline to 1 year
Secondary	Changes in high sensitivity troponin T (hsTNT) levels	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.	Baseline to 1 year
Secondary	Changes in N-terminal Prohormone of Pro-Brain-Type Natriuretic peptide (NT-proBNP) levels	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.	Baseline to 1 year

External Links

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