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

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).

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Key information

Conditions

Arrhythmogenic Cardiomyopathy Arrhythmogenic Right Ventricular Dysplasia Cardiomyopathies Cardiovascular Abnormalities Cardiovascular Diseases Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Death Death, Sudden Death, Sudden, Cardiac Heart Arrest Heart Defects, Congenital Heart Diseases PKP2-ACM PKP2-ARVC Pathologic Processes Pathological Conditions, Signs and Symptoms

Age range

18 year–67 year

Sex eligibility

All sexes

Study type

Observational

Primary location

University of Michigan

Ann Arbor, Michigan, 48109, United States

About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Primary outcomes

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

Time frame: 4 years

Evaluation of Safety and Tolerability of LX2020

Other outcomes

Selected Exploratory

Time frame: 4 years

Change in the frequency of ventricular arrhythmias from baseline.

Sponsors and collaborators

Lead sponsor

Lexeo Therapeutics

Industry

Registry information

Official study title

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

Acronym: GRIT-PKP2

Important dates

Study start: 2025
Primary completion: 2030
Study completion: 2030
First posted: Jul 3, 2025
Registry last updated: Nov 12, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

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