Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

Last updated: November 7, 2025
Sponsor: Lexeo Therapeutics
Overall Status: Active - Not Recruiting

Phase

1/2

Condition

Heart Defect

Circulation Disorders

Arrhythmia

Treatment

LX2020

Clinical Study ID

NCT06109181
LX2020-01
  • Ages 18-65
  • All Genders

Study Summary

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 Criteria

Inclusion

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 thepre-screening MRI

  • Left ventricular ejection fraction ≥ 40%

Exclusion

Selected Exclusion Criteria:

  • Evidence of variant(s) in addition to PKP2 that meets the standard criteria to beconsidered 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 Design

Total Participants: 10
Treatment Group(s): 1
Primary Treatment: LX2020
Phase: 1/2
Study Start date:
February 29, 2024
Estimated Completion Date:
March 31, 2027

Connect with a study center

  • Stanford University

    Stanford, California 94305
    United States

    Site Not Available

  • Stanford University

    Stanford 5398563, California 5332921 94305
    United States

    Site Not Available

  • Johns Hopkins University

    Baltimore, Maryland 21287
    United States

    Site Not Available

  • Johns Hopkins University

    Baltimore 4347778, Maryland 4361885 21287
    United States

    Site Not Available

  • University of Michigan

    Ann Arbor, Michigan 48109
    United States

    Site Not Available

  • University of Michigan

    Ann Arbor 4984247, Michigan 5001836 48109
    United States

    Site Not Available

  • University of Rochester

    Rochester, New York 14642
    United States

    Site Not Available

  • University of Rochester

    Rochester 5134086, New York 5128638 14642
    United States

    Site Not Available

  • Medical University of South Carolina

    Charleston, South Carolina 29425
    United States

    Site Not Available

  • Medical University of South Carolina

    Charleston 4574324, South Carolina 4597040 29425
    United States

    Site Not Available

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