Gene therapy for arrhythmogenic cardiomyopathy caused by a PKP2 variant
A Phase 1/2 Study of the Safety and Efficacy of LX2020 Gene Therapy in Patients With Arrhythmogenic Cardiomyopathy Due to a Plakophilin-2 Pathogenic Variant
This study is testing a new gene therapy for adults with arrhythmogenic cardiomyopathy caused by a specific gene change to see if it is safe and how well it works.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Enrollment | 10 (estimated) |
| Ages | 18 Years to 65 Years |
| Sex | All |
| Sponsor | Lexeo Therapeutics Industry-sponsored |
| Locations | 5 sites (Stanford, California and 4 other locations) |
| Trial ID | NCT06109181 on ClinicalTrials.gov |
What this trial studies
This Phase 1/2 clinical trial is an open-label, multicenter investigation designed to evaluate the safety and tolerability of LX2020, a gene therapy, in adult patients diagnosed with arrhythmogenic cardiomyopathy (ACM) due to a pathogenic variant in the PKP2 gene. The trial involves intravenous administration of the therapy in a dose-escalating manner. Participants must meet specific clinical and genetic criteria, including having frequent premature ventricular complexes and a history of implantable cardioverter-defibrillator placement. The study aims to gather data on the treatment's effects on this rare cardiac condition.
Who should consider this trial
Good fit: Ideal candidates for this study are adults with a clinical diagnosis of arrhythmogenic cardiomyopathy who have a documented pathogenic variant in the PKP2 gene.
Not a fit: Patients with additional pathogenic variants affecting ACM or those with severe heart failure symptoms may not benefit from this study.
Why it matters
Potential benefit: If successful, this gene therapy could provide a novel treatment option for patients with arrhythmogenic cardiomyopathy due to PKP2 variants, potentially improving heart function and reducing arrhythmia risk.
How similar studies have performed: While gene therapy for cardiac conditions is an emerging field, this specific approach targeting PKP2-related arrhythmogenic cardiomyopathy is novel and has not been extensively tested in prior studies.
Eligibility criteria
Show full inclusion / exclusion criteria
Selected Inclusion Criteria: * Adults with a clinical diagnosis of ACM meeting the 2010 revised Task Force Criteria (TFC) * Genetic testing documenting a pathogenic or likely pathogenic variant in PKP2 * Frequent premature ventricular complexes (PVCs) * Implantable cardioverter-defibrillator (ICD) implantation ≥ 12 weeks prior to the pre-screening MRI * Left ventricular ejection fraction ≥ 40% Selected Exclusion Criteria: * Evidence of variant(s) in addition to PKP2 that meets the standard criteria to be considered pathogenic or likely pathogenic for ACM * Other cardiac abnormalities as specified in the protocol * New York Heart Association Functional Class IV at the time of consent * History of prior gene transfer therapy
Where this trial is running
Stanford, California and 4 other locations
- Stanford University — Stanford, California, United States (Recruiting)
- Johns Hopkins University — Baltimore, Maryland, United States (Recruiting)
- University of Michigan — Ann Arbor, Michigan, United States (Recruiting)
- University of Rochester — Rochester, New York, United States (Recruiting)
- Medical University of South Carolina — Charleston, South Carolina, United States (Recruiting)
Study contacts
- Study coordinator: 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.