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Clinical Trials in the USA / NCT06109181
Active, not recruiting Phase 1/2

Gene Therapy for ACM Due to a PKP2 Pathogenic Variant

NCT06109181 · tracked via the Priya Life Science USA tracker
Sponsor
Lexeo Therapeutics
Phase
Phase 1/2
Started
2024-02-29
Last updated
2025-11-12

Condition(s) studied

Arrhythmogenic CardiomyopathyPKP2-ACMPKP2-ARVC

Investigational drug(s) / intervention(s)

LX2020

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

Study 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

Sex
ALL
Min age
18 Years
Max age
65 Years
Healthy volunteers
No
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

Primary outcome measure(s)

  • 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

Trial sites (5)

FacilityCityRegionStatus
Stanford University Stanford California
Johns Hopkins University Baltimore Maryland
University of Michigan Ann Arbor Michigan
University of Rochester Rochester New York
Medical University of South Carolina Charleston South Carolina

More Lexeo Therapeutics trials in the USA

Long-Term Follow-up of Gene Therapy for APOE4 Homozygote Alzheimer's Disease Active, not recruiting
Clinical Course Of Disease In Participants With FA-CM Recruiting
Study of LX2006 Gene Therapy in Friedreich Ataxia Cardiomyopathy Recruiting
A Study to Assess Real-world Patient Characteristics and Clinical Course for Symptomatic Patients With PKP2-ACM Recruiting
Gene Therapy for Cardiomyopathy Associated With Friedreich's Ataxia Active, not recruiting

Other trials for the same condition

A Study to Assess Real-world Patient Characteristics and Clinical Course for Symptomatic Patients With PKP2-ACM Recruiting
Long-term Follow-up Study of Gene Therapy for Arrhythmogenic Cardiomyopathy Due to a Plakophilin-2 Pathogenic Variant Enrolling by invitation
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT06109181 on ClinicalTrials.gov ↗ ← All trials in the USA