Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

Description

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

Conditions

Arrhythmogenic Cardiomyopathy, PKP2-ACM, PKP2-ARVC

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Study Overview

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Study Details

Study 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

A Phase 1/2 Study of the Safety and Efficacy of LX2020 Gene Therapy in Patients With Arrhythmogenic Cardiomyopathy Due to a Plakophilin-2 Pathogenic Variant

Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

Condition
Arrhythmogenic Cardiomyopathy
Intervention / Treatment

-

Contacts and Locations

Stanford

Stanford University, Stanford, California, United States, 94305

Ann Arbor

University of Michigan, Ann Arbor, Michigan, United States, 48109

Rochester

University of Rochester, Rochester, New York, United States, 14642

Charleston

Medical University of South Carolina, Charleston, South Carolina, United States, 29425

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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%
  • * 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

Ages Eligible for Study

18 Years to 65 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Lexeo Therapeutics,

LEXEO Clinical Trials, STUDY_DIRECTOR, Lexeo Therapeutics

Study Record Dates

2027-02