Status:

ACTIVE_NOT_RECRUITING

Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

Lead Sponsor:

Lexeo Therapeutics

Conditions:

Arrhythmogenic Cardiomyopathy

PKP2-ACM

Eligibility:

All Genders

18-65 years

Phase:

PHASE1

PHASE2

Brief 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
  • 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

  • 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

Key Trial Info

Start Date :

February 29 2024

Trial Type :

INTERVENTIONAL

Allocation :

ESTIMATED

End Date :

March 1 2027

Estimated Enrollment :

10 Patients enrolled

Trial Details

Trial ID

NCT06109181

Start Date

February 29 2024

End Date

March 1 2027

Last Update

November 12 2025

Active Locations (5)

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Page 1 of 2 (5 locations)

1

Stanford University

Stanford, California, United States, 94305

2

Johns Hopkins University

Baltimore, Maryland, United States, 21287

3

University of Michigan

Ann Arbor, Michigan, United States, 48109

4

University of Rochester

Rochester, New York, United States, 14642