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Not yet recruitingNCT07486752Updated Mar 20, 2026

BAG3-DCM Natural History Study

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

Study type
Observational
Model
Cohort
Time perspective
Prospective
Enrollment
30
Ages
18 Years and older
Sex
All
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Study 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 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.

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Conditions studied

  • Dilated Cardiomyopathy (DCM)
  • Cardiovascular Diseases
  • Heart Diseases
  • Genetic Diseases

Keywords

  • BAG3
  • Dilated Cardiomyopathy
  • BAG3-DCM
  • Cardiomyopathy
  • BCL2-associated Athanogene 3
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Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

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.

Eligibility criteria

Key Inclusion Criteria:

Subjects are eligible for inclusion into the study only if all the following criteria apply:

General:

  1. Adult patients 18 years or older at the time of providing informed consent (i.e., signing the ICF).
  2. Capable and willing to provide signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and protocol.
  3. 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.
  4. Documentation of a pathogenic or likely pathogenic variant in BAG3 by a CLIA-certified or equivalent genetic testing laboratory.
  5. NYHA class I-III

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.

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Study design

Observational model
Cohort
Time perspective
Prospective
Enrollment
30 participants (estimated)
Patient registry
No

Groups and cohorts

  • Prospective Cohort and Retrospective (Non Interventional)
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What researchers measure

Primary outcomes

  1. 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

Secondary outcomes

  1. NYHA Classification

    Evaluation over duration of follow up

    Time frame: 48 months

  2. 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

  3. Heart rhythm and rate monitoring measures

    Evaluation of change over duration of follow up

    Time frame: 48 months

  4. Cardiac biomarkers and blood proteomics

    Evaluation of change over duration of follow up

    Time frame: 48 months

  5. 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

  6. Evaluate changes in health status

    Evaluate changes in health status as assessed by occurrence of clinical outcomes

    Time frame: 48 months

  7. Event free survival

    Evaluation over duration of follow up

    Time frame: 48 months

  8. Anti-AAV9 titer

    Change in antibody assay findings over time

    Time frame: 48 months

  9. Tissue expression of BAG3 protein and DCM features

    Change in protein expression and histopathologic features

    Time frame: 48 months

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Study locations

No study locations are listed for this record.

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Registry details

Key details

Study ID
NCT07486752
Lead sponsor
Rocket Pharmaceuticals Inc.
Responsible party
Sponsor
First posted
Mar 20, 2026
Start date
May 2026 (estimated)
Primary completion
Dec 2032 (estimated)
Completion
Mar 2033 (estimated)
Last update
Mar 20, 2026

Study contacts

Clinical Information
Contact
clinicaltrials@rocketpharma.com
646-627-0033

Oversight

Data monitoring committee
Yes
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

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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