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

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT07050160

Acronym

GRIT-PKP2

Enrollment

Registered

2025-07-03

Start date

2025-08-29

Completion date

2030-08-31

Last updated

2025-11-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Arrhythmogenic Cardiomyopathy, PKP2-ACM, PKP2-ARVC

Keywords

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

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.

Interventions

None listed

Study design

Observational model

OTHER

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

ALL

Age

18 Years to 67 Years

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame	Description
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

Other

Measure	Time frame	Description
Selected Exploratory	4 years	Change in the frequency of ventricular arrhythmias from baseline.

Countries

United States

Outcome results