Long-term Follow-up Study of Gene Therapy for Arrhythmoge... | NCT07050160

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

NCT07050160

Sponsor

Lexeo Therapeutics

Status

Enrolling by invitation

Last Update Posted

Nov 12, 2025Actual

Enrollment

10Estimated

Phase

Not provided

Conditions

Arrhythmogenic Cardiomyopathy
PKP2-ACM
PKP2-ARVC

Interventions

Not provided

Countries

United States

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

No data available

No data is available for this block.

Derived Section

Miscellaneous Info Module

Version Holder

Status Module

Record Verification Date

Nov 2025

Overall Recruitment Status or Expanded Access Status

Enrolling by invitation

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Aug 29, 2025Actual

Primary Completion Date

Aug 2030Estimated

Completion Date

Aug 2030Estimated

First Submitted Date

Jun 11, 2025

First Submission Date that Met QC Criteria

Jun 24, 2025

First Posted Date

Jul 3, 2025Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Nov 10, 2025

Last Update Posted Date

Nov 12, 2025Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Not provided

Is FDA Regulated Drug

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Conditions Module

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

Design Module

Study Type

Observational

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Target Follow-Up Duration

Not provided

Phases

Not provided

Interventional Study Design

Allocation

Not provided

Intervention Model

Biospecimen

No data available

No data is available for this block.

Enrollment

10Estimated

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
LX2020 treated participants		Participants who received LX2020 in Study LX2020-01

Interventions

Not provided

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
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	4 years

Secondary Outcomes

Not provided

Other Outcomes

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

Eligibility Module

Eligibility Criteria

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.

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

18 Years

Maximum Age

67 Years

Standard Ages

AdultOlder Adult

Study Population

Participants who received LX2020 in Study LX2020-01 will roll over into this study.

Sampling Method

Non-Probability Sample

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Lexeo Clinical Trials	Lexeo Therapeutics	Study Director

Locations

Facility	Status	City	State	ZIP	Country	Contacts
University of Michigan		Ann Arbor	Michigan	48109	United States

Condition Browse Module

MeSH Terms

ID	Term
D009202	Cardiomyopathies
D019571	Arrhythmogenic Right Ventricular Dysplasia
D016757	Death, Sudden, Cardiac
D006323	Heart Arrest

Ancestor Terms

ID	Term
D006331	Heart Diseases
D002318	Cardiovascular Diseases
D006330	Heart Defects, Congenital
D018376	Cardiovascular Abnormalities

Browse Leaves

Not provided

Browse Branches

Not provided