NCT07050160

Long-term Follow-up Study of Gene Therapy for Arrhythmogenic Cardiomyopathy Due to a Plakophilin-2 Pathogenic Variant

A Phase 1/2 Long-term Follow-up Study of the Safety and Efficacy of LX2020 Gene Therapy in Patients With Arrhythmogenic Cardiomyopathy Due to a Plakophilin-2 Pathogenic Variant

• Status: Enrolling By Invitation
• Phase: —
• Study type: Observational
• Enrollment: 10 (estimated)
• Sponsor: Lexeo Therapeutics · Industry
• Sex: All
• Age: 18 Years – 67 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this Phase 1/2 long-term follow-up (LTFU) study is to assess the long-term safety and tolerability of LX2020 for the treatment of arrhythmogenic cardiomyopathy (ACM) due to a plakophilin-2 gene (PKP2) pathogenic variant (PKP2-ACM).

Detailed description

GRIT-PKP2 is a Phase 1/2 long-term follow-up (LTFU) study designed to evaluate the long-term safety and tolerability of LX2020 gene therapy for participants with PKP2-ACM who have previously received LX2020 in the parent study, LX2020-01. After completion of Study LX2020-01, participants will roll over into this study LX2020-02 for an additional 4-year long-term observation. No gene therapy will be administered in Study LX2020-02. Efficacy will be assessed as an exploratory objective.

Conditions

• Arrhythmogenic Cardiomyopathy
• PKP2-ACM
• PKP2-ARVC

Timeline

• Start date: 2025-08-29
• Primary completion: 2030-08-01
• Completion: 2030-08-01
• First posted: 2025-07-03
• Last updated: 2025-11-12

Locations

1 site across 1 country: United States