A Study to Assess Real-world Patient Characteristics and Clinical Course for Symptomatic Patients With PKP2-ACM

Summary

An observational study to assess real-world patient characteristics and clinical course of disease in participants with PKP2-ACM.

Eligibility

Inclusion Criteria:

* Adults with a clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC)
* Documentation of a pathogenic or likely pathogenic truncating variant in PKP2
* Frequent premature ventricular contractions (PVCs)
* Patients must have an ICD placed prior to enrollment
* Left ventricular ejection fraction (LVEF) ≥ 50% for Part A participants. Left ventricular ejection fraction (LVEF) ≥40% for Part B participants.

Exclusion Criteria:

* Evidence of variant(s) in addition to PKP2 that meet standard criteria to be considered pathogenic or likely pathogenic for an arrhythmogenic cardiomyopathy.
* A history of other cardiac abnormalities as specified in the protocol.
* New York Heart Association symptoms of heart failure of Class IV at the time of consent.
* A history of prior gene transfer therapy.

Conditions4

Locations6 sites

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