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Active, not recruitingNCT06109181Updated Nov 12, 2025

Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

A Phase 1/2 interventional study of LX2020 in Arrhythmogenic Cardiomyopathy, PKP2-ACM and PKP2-ARVC, sponsored by Lexeo Therapeutics. Active, not recruiting at 5 sites in United States. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2025-11-12.

Sponsored by Lexeo Therapeutics · Phase 1/2, Interventional, and Treatment

Phase
Phase 1/2
Study type
Interventional
Enrollment
10
Allocation
Not applicable
Ages
18 Years to 65 Years
Sex
All
01

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

02

Conditions studied

  • Arrhythmogenic Cardiomyopathy
  • PKP2-ACM
  • PKP2-ARVC

Keywords

  • Arrhythmogenic Cardiomyopathy
  • ACM
  • Cardiomyopathy
  • ARVC
  • Arrhythmogenic Right Ventricular
  • Arrhythmogenic Right Ventricular Dysplasia
  • Genetic cardiomyopathy
  • Gene Therapy
  • PKP2 Gene
  • Plakophilin-2
  • LX2020
  • HEROIC-PKP2
  • Ventricular Arrhythmia
  • PVCs
  • Sudden Cardiac Death
  • Cardiac Arrest
03

In context

Cardiomyopathies

1,176 studies on the registry are indexed under Cardiomyopathies; 287 are open to participants now.

This study's planned enrollment of 10 is below the median of 51 across 609 interventional studies indexed under Cardiomyopathies.

Browse Cardiomyopathies studies →

Lead sponsor

Lexeo Therapeutics is the lead sponsor of 8 studies on the registry; 4 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 65 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility 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
05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential assignment
Masking
None (open label)
Enrollment
10 participants (estimated)

Study arms

  • Experimental
    LX2020

    Single ascending dose of LX2020, with a starting dose of 2.0 x10\^13 gc/kg, in multiple cohorts

    Genetic: LX2020

Interventions

  • GeneticLX2020

    LX2020 is an adeno-associated viral vector encoding the human Plakophilin-2 (PKP2) gene (AAVrh.10hPKP2)

06

What researchers measure

Primary outcomes

  1. Percentage of subjects who experienced at least 1 treatment emergent adverse event (TEAE) and/or 1 treatment emergent serious adverse event (TESAE).

    Evaluation of Safety and Tolerability of LX2020

    Time frame: 12 months

Other outcomes

  1. Selected Exploratory

    Change in the frequency of ventricular arrhythmias from baseline.

    Time frame: 12 months

07

Study locations

5 sites
  • Stanford University
    Stanford, California 94305, United States
  • Johns Hopkins University
    Baltimore, Maryland 21287, United States
  • University of Michigan
    Ann Arbor, Michigan 48109, United States
  • University of Rochester
    Rochester, New York 14642, United States
  • Medical University of South Carolina
    Charleston, South Carolina 29425, United States
08

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Nov 12, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT06109181
Lead sponsor
Lexeo Therapeutics
Responsible party
Sponsor
First posted
Oct 31, 2023
Start date
Feb 29, 2024
Primary completion
Dec 2026 (estimated)
Completion
Mar 2027 (estimated)
Last update
Nov 12, 2025

Study contacts

LEXEO Clinical Trials
study director · Lexeo Therapeutics

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is active, not recruiting, as verified in Nov 2025. You cannot join it, but the record below documents what was studied.

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