Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

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

Active, Not Recruiting

This study is active but is not currently recruiting participants.

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Key information

Conditions

Arrhythmogenic Cardiomyopathy Arrhythmogenic Right Ventricular Dysplasia Cardiomyopathies Cardiovascular Abnormalities Cardiovascular Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Death Death, Sudden Death, Sudden, Cardiac Heart Arrest Heart Defects, Congenital Heart Diseases PKP2-ACM PKP2-ARVC Pathologic Processes Pathological Conditions, Signs and Symptoms

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Stanford University, Stanford, California, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

LX2020

Genetic

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

Primary outcomes

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

Time frame: 12 months

Evaluation of Safety and Tolerability of LX2020

Other outcomes

Selected Exploratory

Time frame: 12 months

Change in the frequency of ventricular arrhythmias from baseline.

Sponsors and collaborators

Lead sponsor

Lexeo Therapeutics

Industry

Registry information

Official study title

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

Important dates

Study start: 2024
Primary completion: 2026
Study completion: 2027
First posted: Oct 31, 2023
Registry last updated: Nov 12, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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Enrolling by Invitation