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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ENROLLING_BY_INVITATION

Total Enrollment

10 participants

Study Classification

OBSERVATIONAL

Study Start Date

2025-08-29

Study Completion Date

2030-08-31

Brief Summary

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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

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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

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Arrhythmogenic Cardiomyopathy PKP2-ACM PKP2-ARVC

Keywords

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Arrhythmogenic Cardiomyopathy ACM Cardiomyopathy ARVC Arrhythmogenic Right Ventricular Arrhythmogenic Right Ventricular Dysplasia Genetic cardiomyopathy Gene Therapy PKP2 Gene Plakophilin-2 LX2020 GRIT-PKP2 Ventricular Arrhythmia PVCs Sudden Cardiac Death Cardiac Arrest

Study Design

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Observational Model Type

OTHER

Study Time Perspective

PROSPECTIVE

Study Groups

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LX2020 treated participants

Participants who received LX2020 in Study LX2020-01

No interventions assigned to this group

Eligibility Criteria

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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.

Minimum Eligible Age

18 Years

Maximum Eligible Age

67 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Lexeo Clinical Trials

Role: STUDY_DIRECTOR

Lexeo Therapeutics

Locations

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University of Michigan

Ann Arbor, Michigan, United States

Site Status

Countries

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United States

Other Identifiers

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LX2020-02

Identifier Type: -

Identifier Source: org_study_id