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

Not yet recruiting

• Conditions: Arrhythmogenic Cardiomyopathy; PKP2-ARVC; PKP2-ACM

• Registration Number: NCT07050160
• Lead Sponsor: Lexeo Therapeutics

Brief 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.

Study Timeline

• Status Verified: 2025-06
• Study First Submitted: 2025-06-11
• Study First Submitted QC: 2025-06-24
• Last Update Submitted: 2025-06-24
• Study First Posted: 2025-07-03
• Last Update Posted: 2025-07-03
• Study Start: 2025-08-01
• Primary Completion: 2030-08
• Study Completion: 2030-08-01

Recruitment & Eligibility

• Status: NOT_YET_RECRUITING
• Sex: All
• Target Recruitment: 10

Inclusion Criteria

• Participants who received LX2020 in study LX2020-01

Exclusion Criteria

• Concurrent enrollment in any other clinical investigation involving use of an investigational agent.

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Percentage of subjects who experienced at least 1 treatment emergent adverse event (TEAE) and/or 1 treatment emergent serious adverse event (TESAE).	4 years	Evaluation of Safety and Tolerability of LX2020