An Observational Study to Assess Real-world Patient Characteristics, Clinical Course, and Treatment Patterns for Symptomatic Patients With Arrhythmogenic Cardiomyopathy (ACM) Due To a PlaKoPhilin-2 Pathogenic Variant (PKP2)
Lexeo Therapeutics
Summary
An observational study to assess real-world patient characteristics and clinical course of disease in participants with PKP2-ACM.
Description
SNAPSHOT-PKP2 is an observational, multicenter study that consists of 2 parts, a retrospective EMR review (up to 2 years) and a prospective observational study (1 year), to evaluate the clinical burden of illness of patients with PKP2-ACM, and to prospectively evaluate changes in key cardiac parameters and patient-reported outcome measures (PROMs) associated with PKP2-ACM progression.
Eligibility
- Age range
- 18–65 years
- Sex
- All
- Healthy volunteers
- No
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 f…
Locations (6)
- Leland Stanford Junior UniversityRedwood City, California
- Johns Hopkins UniversityBaltimore, Maryland
- Northshore University Healthsystem Research InstituteColumbia, Maryland
- University of MichiganAnn Arbor, Michigan
- Henry Ford HospitalDetroit, Michigan
- University of RochesterRochester, New York