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

N/AEnrolling By InvitationNCT07050160

Lexeo Therapeutics10 enrolled

Overview

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).

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 Type

OBSERVATIONAL

Enrollment

Conditions

Arrhythmogenic Cardiomyopathy PKP2-ACM PKP2-ARVC

Eligibility

Sex: ALLMin age: 18 YearsMax age: 67 Years

Medical Language ↔ Plain English

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.

Locations (1)

University of Michigan

Ann Arbor, Michigan, United States

Outcomes

Primary Outcomes

Percentage of subjects who experienced at least 1 treatment emergent adverse event (TEAE) and/or 1 treatment emergent serious adverse event (TESAE).

Evaluation of Safety and Tolerability of LX2020

Time frame: 4 years

Data from ClinicalTrials.gov

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