BAG3-DCM Natural History Study

NCT07486752 | Not Yet Recruiting DMC

• 1 other identifier: RP-NI-A701-0146
• Study Type: observational
• Target: 30
• Locations: 0 countries
• Sites: N/A

Brief 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.

Conditions

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

Keywords

BAG3; Dilated Cardiomyopathy; BAG3-DCM; Cardiomyopathy; BCL2-associated Athanogene 3

Outcome Measures

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.

  48 months

Secondary Outcomes (9)

• NYHA Classification

  48 months

• Change in arrhythmias or risk factor for ventricular arrhythmias

  48 months

• Heart rhythm and rate monitoring measures

  48 months

• Cardiac biomarkers and blood proteomics

  48 months

• Evaluate patient reported outcomes and quality of life measures

  48 months

Study Arms (1)

Prospective Cohort and Retrospective (Non Interventional)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

You may not qualify if:

• All Cohorts:
• \. 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.
• \. NYHA class IV HF. 5. Presence or requirement for MCS or predicted need for MCS or heart transplantation within 6 months prior to enrollment.
• \. 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.

Sponsors & Collaborators

• Rocket Pharmaceuticals Inc.: lead

MeSH Terms

Conditions

Cardiomyopathy, Dilated; Cardiovascular Diseases; Heart Diseases; Genetic Diseases, Inborn; Cardiomyopathies

Condition Hierarchy (Ancestors)

Cardiomegaly; Laminopathies; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Central Study Contacts

Clinical Information

CONTACT

646-627-0033; clinicaltrials@rocketpharma.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 12, 2026
• First Posted: March 20, 2026
• Study Start: May 1, 2026
• Primary Completion (Estimated): December 1, 2032
• Study Completion (Estimated): March 1, 2033
• Last Updated: March 20, 2026

Record last verified: 2026-03