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Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

Not Recruiting
18-65 years
All
Phase 1/2

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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

Study details
    Arrhythmogenic Cardiomyopathy
    PKP2-ACM
    PKP2-ARVC

NCT06109181

Lexeo Therapeutics

12 December 2025

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