BAG3-DCM Natural History Study
Trial statusNot yet recruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age18+
SponsorRocket Pharmaceuticals Inc.
About this trial
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.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Disqualifiers
None
Trial 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.
Trial design
Design model
Cohort
Time perspective
Prospective
Treatments tested in this trial
Not listed
Trial groups
30 Participants
are grouped into 1 trial groupGroup A: Prospective Cohort and Retrospective (Non Interventional)
Trial outcomes
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 monthsSecondary outcomes
1
NYHA Classification
Evaluation over duration of follow up
Time frame
48 months2
Change in arrhythmias or risk factor for ventricular arrhythmias
Evaluate changes in health status as assessed by occurrence of clinical outcomes
Time frame
48 months3
Heart rhythm and rate monitoring measures
Evaluation of change over duration of follow up
Time frame
48 months4
Cardiac biomarkers and blood proteomics
Evaluation of change over duration of follow up
Time frame
48 monthsLocations
Sponsors and contacts
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Source ClinicalTrials.gov
This trial is not recruiting at the moment. You can still explore other options: