Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

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

Status

Active, not recruiting

Phases

Phase 1Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT06109181

Enrollment

Registered

2023-10-31

Start date

2024-02-29

Completion date

2027-03-31

Last updated

2025-11-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

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

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

Interventions

GENETICLX2020

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

Study design

Allocation

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to 65 Years

Healthy volunteers

Inclusion 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

Design outcomes

Primary

Measure	Time frame	Description
Percentage of subjects who experienced at least 1 treatment emergent adverse event (TEAE) and/or 1 treatment emergent serious adverse event (TESAE).	12 months	Evaluation of Safety and Tolerability of LX2020

Other

Measure	Time frame	Description
Selected Exploratory	12 months	Change in the frequency of ventricular arrhythmias from baseline.

Countries

United States

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026