An observational study in Dilated Cardiomyopathy (DCM), Cardiovascular Diseases and Heart Diseases, sponsored by Rocket Pharmaceuticals Inc.. Not yet recruiting. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-03-20.
Sponsored by Rocket Pharmaceuticals Inc. · Observational
The goal of this international observational study is to learn about the natural history of Dilated Cardiomyopathy (DCM) arising from pathogenic BAG3 variants in adult patients ≥18 years of age.
This is an observational study with both retrospective and prospective data collection. The study is designed to describe the natural history of BAG3-DCM including the signs and symptoms, key clinical events, and impact of the disease on quality of life as managed with the current standard of care. A hybrid (retrospective and prospective data collection) approach is being used to generate robust and longitudinal data.
Adult patients 18 years and over (at the point of consenting) with confirmed Dilated Cardiomyopathy (DCM) arising from pathogenic BAG3 Variants recruited from a range of applicable care settings.
Key Inclusion Criteria:
Subjects are eligible for inclusion into the study only if all the following criteria apply:
General:
Key Exclusion Criteria:
All Cohorts:
1. Concurrent enrollment in any other clinical investigation involving use of an investigational agent for any condition at time of enrollment to this study that could confound interpretation of this study results 2. Previous treatment with gene therapy 2. Gene testing indicates that the patient's arrhythmia or cardiomyopathy may be related to a genetic etiology other than BAG3 variant.
4. NYHA class IV HF. 5. Presence or requirement for MCS or predicted need for MCS or heart transplantation within 6 months prior to enrollment.
6. Prior heart transplantation. 7. Known infection with human immunodeficiency virus (HIV). 8. Unwillingness to comply with study procedures, including follow-up as specified by this protocol, or unwillingness to fully cooperate with the investigator.
Cardiac structure and function
Evaluate cardiovascular health as assessed by cardiac biomarkers and the occurrence of clinical outcomes related to the cardiovascular system.
Time frame: 48 months
NYHA Classification
Evaluation over duration of follow up
Time frame: 48 months
Change in arrhythmias or risk factor for ventricular arrhythmias
Evaluate changes in health status as assessed by occurrence of clinical outcomes
Time frame: 48 months
Heart rhythm and rate monitoring measures
Evaluation of change over duration of follow up
Time frame: 48 months
Cardiac biomarkers and blood proteomics
Evaluation of change over duration of follow up
Time frame: 48 months
Evaluate patient reported outcomes and quality of life measures
Evaluate patient reported outcomes and quality of life measures using validated questionnaire.
Time frame: 48 months
Evaluate changes in health status
Evaluate changes in health status as assessed by occurrence of clinical outcomes
Time frame: 48 months
Event free survival
Evaluation over duration of follow up
Time frame: 48 months
Anti-AAV9 titer
Change in antibody assay findings over time
Time frame: 48 months
Tissue expression of BAG3 protein and DCM features
Change in protein expression and histopathologic features
Time frame: 48 months
No study locations are listed for this record.
This study is not yet recruiting, as verified in Mar 2026. You cannot join it, but the record below documents what was studied.
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Rocket Pharmaceuticals Inc.