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 2026-10-19.
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.
255 studies on the registry are indexed under Cardiomyopathy, Dilated; 66 are open to participants now.
This study's planned enrollment of 30 is below the median of 245 across 96 observational studies indexed under Cardiomyopathy, Dilated.
Browse Cardiomyopathy, Dilated studies →Rocket Pharmaceuticals Inc. is the lead sponsor of 19 studies on the registry; 7 are open to participants now.
Counted across the registry records on this site, refreshed daily.
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 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 (HIV). 8. Unwillingness to comply with study procedures, including follow-up as specified by this protocol, or unwillingness to fully cooperate with the investigator.
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
NYHA class (heart failure)
The New York Heart Association’s four classes of how much heart failure limits physical activity.
Studies set their own limit — many outside heart failure leave out class III–IV, while heart failure studies may ask for a particular class. Check the class this study names.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗
This study is not yet recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.
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Rocket Pharmaceuticals Inc.