Assessing patient characteristics and clinical course for those with PKP2-ACM
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 · NCT06976606
This study looks at the experiences and health changes of people with PKP2-ACM over time to better understand the condition and its impact on their lives.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 40 (estimated) |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Sponsor | Lexeo Therapeutics (industry) |
| Locations | 6 sites (Redwood City, California and 5 other locations) |
| Trial ID | NCT06976606 on ClinicalTrials.gov |
What this trial studies
This observational study, known as SNAPSHOT-PKP2, aims to evaluate the real-world characteristics and clinical progression of patients diagnosed with PKP2-ACM. It consists of two parts: a retrospective review of electronic medical records over the past two years and a prospective observational phase lasting one year. The study will assess the clinical burden of illness and monitor changes in key cardiac parameters and patient-reported outcomes associated with the progression of PKP2-ACM.
Who should consider this trial
Good fit: Ideal candidates include adults diagnosed with arrhythmogenic cardiomyopathy who have a documented pathogenic variant in PKP2 and have an ICD placed.
Not a fit: Patients with additional pathogenic variants or severe heart failure symptoms may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into the clinical course of PKP2-ACM, potentially leading to improved management strategies for patients.
How similar studies have performed: While this study focuses on a specific genetic variant, similar observational studies have shown success in understanding the clinical characteristics of other forms of cardiomyopathy.
Eligibility criteria
Show full inclusion / exclusion criteria
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.
Where this trial is running
Redwood City, California and 5 other locations
- Leland Stanford Junior University — Redwood City, California, United States (RECRUITING)
- Johns Hopkins University — Baltimore, Maryland, United States (RECRUITING)
- Northshore University Healthsystem Research Institute — Columbia, Maryland, United States (RECRUITING)
- University of Michigan — Ann Arbor, Michigan, United States (RECRUITING)
- Henry Ford Hospital — Detroit, Michigan, United States (RECRUITING)
- University of Rochester — Rochester, New York, United States (RECRUITING)
Study contacts
- Study coordinator: Person*: Lexeo Clinical Trials
- Email: clinicaltrials@lexeotx.com
- Phone: 212-547-9879
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
Conditions: Arrhythmogenic Cardiomyopathy, PKP2-ACM, PKP2-ARVC, ACM, Cardiomyopathy, ARVC, Arrhythmogenic Right Ventricular, Arrhythmogenic Right Ventricular Dysplasia