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

Primary Purpose

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

Status
Recruiting
Phase
Locations
International
Study Type
Observational
Intervention
Sponsored by
Pfizer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an observational trial for Cardiomyopathy, Dilated focused on measuring BAG3, DCM, cardiomyopathy, dilated cardiomyopathy, heritable heart disease, heart disease

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept 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

Sites / Locations

  • University of Colorado Anschutz Medical Campus, Fitzsimons Building
  • University of Colorado Clinical and Translational Research Center
  • University of Colorado Denver, Anschutz Medical Campus
  • University of Colorado Hospital
  • Brigham & Women's HospitalRecruiting
  • Brigham and Women's HospitalRecruiting
  • Montefiore Medical Center(Hutchinson Metro Center)Recruiting
  • Montefiore Medical Center(Medical Arts Pavilion)Recruiting
  • Montefiore Medical CenterRecruiting
  • Division of Heart Failure and Transplantation(Hospital of the University of Pennsylvania)
  • Narodowy Instytut Kardiologii Stefana Kardynala Wyszynskiego - Panstwowy Instytut BadawczyRecruiting
  • Hospital Universitario Puerta de Hierro MajadahondaRecruiting
  • Hospital Clinico Universitario Virgen de La ArrixacaRecruiting
  • CHUAC-Complejo Hospitalario Universitario A CoruñaRecruiting
  • Hospital Universitario 12 de Octubre
  • The Royal Brompton and Harefield HospitalsRecruiting

Arms of the Study

Arm 1

Arm Type

Arm Label

BAG3 DCM

Arm Description

A single arm observational trial where all participants will undergo the same schedule of assessments.

Outcomes

Primary Outcome Measures

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.
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.

Secondary Outcome Measures

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.
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.
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.

Full Information

First Posted
June 14, 2023
Last Updated
September 26, 2023
Sponsor
Pfizer
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1. Study Identification

Unique Protocol Identification Number
NCT05981092
Brief Title
A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease)
Acronym
BAG3 DCM
Official Title
A Prospective Multinational Study of the Natural History of Participants With BAG3 Mutation Associated Dilated Cardiomyopathy
Study Type
Observational

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Recruiting
Study Start Date
October 14, 2022 (Actual)
Primary Completion Date
April 19, 2025 (Anticipated)
Study Completion Date
April 19, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pfizer

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Cardiomyopathy, Dilated, Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM)
Keywords
BAG3, DCM, cardiomyopathy, dilated cardiomyopathy, heritable heart disease, heart disease

7. Study Design

Enrollment
35 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
BAG3 DCM
Arm Description
A single arm observational trial where all participants will undergo the same schedule of assessments.
Primary Outcome Measure Information:
Title
Determine baseline of cardiac structure and function in BAG3 associated DCM.
Description
Baseline measures of left ventricular volumes at end systole and diastole using imaging at baseline.
Time Frame
Baseline
Title
Determine changes over time in cardiac structure and function in BAG3 associated DCM.
Description
Changes over time of left ventricular volumes at end systole and diastole using imaging over the course of one year follow up time.
Time Frame
1 year
Secondary Outcome Measure Information:
Title
Changes in high sensitivity troponin I (hsTNI) levels
Description
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.
Time Frame
Baseline to 1 year
Title
Changes in high sensitivity troponin T (hsTNT) levels
Description
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.
Time Frame
Baseline to 1 year
Title
Changes in N-terminal Prohormone of Pro-Brain-Type Natriuretic peptide (NT-proBNP) levels
Description
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.
Time Frame
Baseline to 1 year

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Study Population Description
primary care clinic and referrals from primary care clinic to investigational sites
Sampling Method
Non-Probability Sample
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Pfizer CT.gov Call Center
Phone
1-800-718-1021
Email
ClinicalTrials.gov_Inquiries@pfizer.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Pfizer CT.gov Call Center
Organizational Affiliation
Pfizer
Official's Role
Study Director
Facility Information:
Facility Name
University of Colorado Anschutz Medical Campus, Fitzsimons Building
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
University of Colorado Clinical and Translational Research Center
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
University of Colorado Denver, Anschutz Medical Campus
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
University of Colorado Hospital
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
Brigham & Women's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Individual Site Status
Recruiting
Facility Name
Brigham and Women's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Individual Site Status
Recruiting
Facility Name
Montefiore Medical Center(Hutchinson Metro Center)
City
Bronx
State/Province
New York
ZIP/Postal Code
10461
Country
United States
Individual Site Status
Recruiting
Facility Name
Montefiore Medical Center(Medical Arts Pavilion)
City
Bronx
State/Province
New York
ZIP/Postal Code
10467
Country
United States
Individual Site Status
Recruiting
Facility Name
Montefiore Medical Center
City
Bronx
State/Province
New York
ZIP/Postal Code
10467
Country
United States
Individual Site Status
Recruiting
Facility Name
Division of Heart Failure and Transplantation(Hospital of the University of Pennsylvania)
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Individual Site Status
Not yet recruiting
Facility Name
Narodowy Instytut Kardiologii Stefana Kardynala Wyszynskiego - Panstwowy Instytut Badawczy
City
Warszawa
ZIP/Postal Code
04-628
Country
Poland
Individual Site Status
Recruiting
Facility Name
Hospital Universitario Puerta de Hierro Majadahonda
City
Majadahonda
State/Province
Madrid
ZIP/Postal Code
28222
Country
Spain
Individual Site Status
Recruiting
Facility Name
Hospital Clinico Universitario Virgen de La Arrixaca
City
El Palmar
State/Province
Murcia
ZIP/Postal Code
30120
Country
Spain
Individual Site Status
Recruiting
Facility Name
CHUAC-Complejo Hospitalario Universitario A Coruña
City
A Coruna
ZIP/Postal Code
15006
Country
Spain
Individual Site Status
Recruiting
Facility Name
Hospital Universitario 12 de Octubre
City
Madrid
ZIP/Postal Code
28041
Country
Spain
Individual Site Status
Not yet recruiting
Facility Name
The Royal Brompton and Harefield Hospitals
City
Harefield
State/Province
London
ZIP/Postal Code
UB9 6JH
Country
United Kingdom
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No
IPD Sharing Plan Description
Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
Links:
URL
https://pmiform.com/clinical-trial-info-request?StudyID=C4981001
Description
To obtain contact information for a study center near you, click here.

Learn more about this trial

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

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