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Gene Therapy for ACM Due to a PKP2 Pathogenic Variant
Sponsor: Lexeo Therapeutics
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
Official 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
Key Details
Gender
All
Age Range
18 Years - 65 Years
Study Type
INTERVENTIONAL
Enrollment
10
Start Date
2024-02-29
Completion Date
2027-03
Last Updated
2025-11-12
Healthy Volunteers
No
Interventions
LX2020
LX2020 is an adeno-associated viral vector encoding the human Plakophilin-2 (PKP2) gene (AAVrh.10hPKP2)
Locations (5)
Stanford University
Stanford, California, United States
Johns Hopkins University
Baltimore, Maryland, United States
University of Michigan
Ann Arbor, Michigan, United States
University of Rochester
Rochester, New York, United States
Medical University of South Carolina
Charleston, South Carolina, United States