NCT06109181

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
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Lexeo Therapeutics · Industry
• Sex: All
• Age: 18 Years – 65 Years
• Healthy volunteers: Not accepted

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

Conditions

• Arrhythmogenic Cardiomyopathy
• PKP2-ACM
• PKP2-ARVC

Interventions

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

Timeline

• Start date: 2024-02-29
• Primary completion: 2026-12-01
• Completion: 2027-03-01
• First posted: 2023-10-31
• Last updated: 2025-11-12

Locations

5 sites across 1 country: United States

Regulatory

• FDA-regulated drug study