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Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

NCT ID: NCT06109181

Last Updated: 2025-11-12

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

10 participants

Study Classification

INTERVENTIONAL

Study Start Date

2024-02-29

Study Completion Date

2027-03-31

Brief Summary

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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

Detailed Description

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Conditions

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Arrhythmogenic Cardiomyopathy PKP2-ACM PKP2-ARVC

Keywords

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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

Study Design

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Allocation Method

NA

Intervention Model

SEQUENTIAL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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LX2020

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

Group Type EXPERIMENTAL

LX2020

Intervention Type GENETIC

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

Interventions

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LX2020

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

Intervention Type GENETIC

Eligibility Criteria

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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%

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
Minimum Eligible Age

18 Years

Maximum Eligible Age

65 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Lexeo Therapeutics

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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LEXEO Clinical Trials

Role: STUDY_DIRECTOR

Lexeo Therapeutics

Locations

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Stanford University

Stanford, California, United States

Site Status

Johns Hopkins University

Baltimore, Maryland, United States

Site Status

University of Michigan

Ann Arbor, Michigan, United States

Site Status

University of Rochester

Rochester, New York, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

Countries

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United States

Other Identifiers

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LX2020-01

Identifier Type: -

Identifier Source: org_study_id