Dilated Cardiomyopathy (DCM)NCT07486752
BAG3-DCM Natural History Study
An observational study of Dilated Cardiomyopathy (DCM) and Cardiovascular Diseases, run by Rocket Pharmaceuticals Inc..
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Dilated Cardiomyopathy (DCM), Cardiovascular Diseases, Heart Diseases
- How many people
- 30 participants
- Where
- No sites listed yet
- Study dates
- Dec 2026to Mar 2033
- Phase
- Observational
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: 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.
Who may be able to join
- Subjects are eligible for inclusion into the study only if all the following criteria apply:
- General:
- Adult patients 18 years or older at the time of providing informed consent (i.e., signing the ICF).
- Capable and willing to provide signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and protocol.
- Diagnosis of DCM as defined by mild to moderate systolic dysfunction performed within 12 months of enrollment and confirmed by the principal investigator that the DCM is predominantly non-ischemic.
- Documentation of a pathogenic or likely pathogenic variant in BAG3 by a CLIA-certified or equivalent genetic testing laboratory.
- NYHA class I-III
Who cannot join
- 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.
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: An Observational Study of Patients With Dilated Cardiomyopathy (DCM) Associated With Pathogenic BAG3 Variants
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.
Read the detailed description
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.
- Keywords
- BAG3, Dilated Cardiomyopathy, BAG3-DCM, Cardiomyopathy, BCL2-associated Athanogene 3
- Medical subject headings
- Cardiomyopathy, Dilated, Cardiovascular Diseases, Heart Diseases, Genetic Diseases, Inborn, Cardiomyopathies
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Prospective Cohort and Retrospective (Non Interventional) | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Cardiac structure and function
Evaluate cardiovascular health as assessed by cardiac biomarkers and the occurrence of clinical outcomes related to the cardiovascular system.
Measured: 48 months
Secondary outcomes (9)
NYHA Classification
Evaluation over duration of follow up
Measured: 48 months
Change in arrhythmias or risk factor for ventricular arrhythmias
Evaluate changes in health status as assessed by occurrence of clinical outcomes
Measured: 48 months
Heart rhythm and rate monitoring measures
Evaluation of change over duration of follow up
Measured: 48 months
Cardiac biomarkers and blood proteomics
Evaluation of change over duration of follow up
Measured: 48 months
Evaluate patient reported outcomes and quality of life measures
Evaluate patient reported outcomes and quality of life measures using validated questionnaire.
Measured: 48 months
Evaluate changes in health status
Evaluate changes in health status as assessed by occurrence of clinical outcomes
Measured: 48 months
Event free survival
Evaluation over duration of follow up
Measured: 48 months
Anti-AAV9 titer
Change in antibody assay findings over time
Measured: 48 months
Tissue expression of BAG3 protein and DCM features
Change in protein expression and histopathologic features
Measured: 48 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Mar 2026
- Study startDec 2026 (estimated)
- Primary completionDec 2032 (estimated)
- Study completionMar 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dilated Cardiomyopathy (DCM)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07486752
- Sponsor's protocol ID
- RP-NI-A701-0146
- Status last verified
- Oct 2026
- First submitted
- 12 Mar 2026
- Last updated
- 19 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No