A Study About the Natural History in Adults With BAG3 Dilated Cardiomyopathy (a Type of Heart Disease) (BAG3 DCM)
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The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT05981092 |
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Recruitment Status :
Recruiting
First Posted : August 8, 2023
Last Update Posted : April 11, 2024
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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 |
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| Cardiomyopathy, Dilated Bcl-2 Anathogene-3 (BAG3) Dilated Cardiomyopathy (DCM) |
| Study Type : | Observational |
| Estimated Enrollment : | 35 participants |
| Observational Model: | Cohort |
| Time Perspective: | Prospective |
| Official Title: | A PROSPECTIVE MULTINATIONAL STUDY OF THE NATURAL HISTORY OF PARTICIPANTS WITH BAG3 MUTATION ASSOCIATED DILATED CARDIOMYOPATHY |
| Actual Study Start Date : | October 14, 2022 |
| Estimated Primary Completion Date : | August 9, 2025 |
| Estimated Study Completion Date : | August 9, 2025 |
| Group/Cohort |
|---|
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BAG3 DCM
A single arm observational trial where all participants will undergo the same schedule of assessments.
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- Determine baseline of cardiac structure and function in BAG3 associated DCM. [ Time Frame: 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. [ Time Frame: 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.
- Changes in high sensitivity troponin I (hsTNI) levels [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 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.
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Non-Probability Sample |
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
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05981092
| Contact: Pfizer CT.gov Call Center | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
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| Study Director: | Pfizer CT.gov Call Center | Pfizer |
| Responsible Party: | Pfizer |
| ClinicalTrials.gov Identifier: | NCT05981092 |
| Other Study ID Numbers: |
C4981001 2022-000398-20 ( EudraCT Number ) |
| First Posted: | August 8, 2023 Key Record Dates |
| Last Update Posted: | April 11, 2024 |
| Last Verified: | April 2024 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | No |
| 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. |
| Studies a U.S. FDA-regulated Drug Product: | No |
| Studies a U.S. FDA-regulated Device Product: | No |
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BAG3 DCM cardiomyopathy |
dilated cardiomyopathy heritable heart disease heart disease |
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Heart Diseases Cardiomyopathies Cardiomyopathy, Dilated Cardiovascular Diseases |
Cardiomegaly Laminopathies Genetic Diseases, Inborn |