Overview
This is a Phase 1/2, first-in-human, open-label, intravenous, dose-escalating, multicenter trial that is designed to assess the safety and tolerability of LX2020 in adult patients with PKP2-ACM
Eligibility
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


