Long-term Follow-up Study of Gene Therapy for Arrhythmogenic Cardiomyopathy Due to a Plakophilin-2 Pathogenic Variant (GRIT-PKP2)

Lexeo Therapeutics

Status: Invitation-only

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

Study type: Observational

Funder types: Industry

Identifiers

NCT07050160

LX2020-02

Details and patient eligibility

About

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).

Full 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.

Enrollment: 10 estimated patients

Sex: All

Ages: 18 to 67 years old

Volunteers: No 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.

Trial design

10 participants in 1 patient group

LX2020 treated participants

Description:

Participants who received LX2020 in Study LX2020-01