Ignite Creation Date:
2025-12-25 @ 2:03 AM
Ignite Modification Date:
2025-12-27 @ 11:48 PM
Study NCT ID:
NCT07050160
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2025-11-12
First Post:
2025-06-11
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Long-term Follow-up Study of Gene Therapy for Arrhythmogenic Cardiomyopathy Due to a Plakophilin-2 Pathogenic Variant
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009202', 'term': 'Cardiomyopathies'}, {'id': 'D019571', 'term': 'Arrhythmogenic Right Ventricular Dysplasia'}, {'id': 'D016757', 'term': 'Death, Sudden, Cardiac'}, {'id': 'D006323', 'term': 'Heart Arrest'}], 'ancestors': [{'id': 'D006331', 'term': 'Heart Diseases'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D006330', 'term': 'Heart Defects, Congenital'}, {'id': 'D018376', 'term': 'Cardiovascular Abnormalities'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D003645', 'term': 'Death, Sudden'}, {'id': 'D003643', 'term': 'Death'}, {'id': 'D010335', 'term': 'Pathologic Processes'}, {'id': 'D013568', 'term': 'Pathological Conditions, Signs and Symptoms'}]}}, 'protocolSection': {'designModule': {'studyType': 'OBSERVATIONAL', 'designInfo': {'timePerspective': 'PROSPECTIVE', 'observationalModel': 'OTHER'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 10}, 'patientRegistry': False}, 'statusModule': {'overallStatus': 'ENROLLING_BY_INVITATION', 'startDateStruct': {'date': '2025-08-29', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2030-08', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-11-10', 'studyFirstSubmitDate': '2025-06-11', 'studyFirstSubmitQcDate': '2025-06-24', 'lastUpdatePostDateStruct': {'date': '2025-11-12', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2025-07-03', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2030-08', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Selected Exploratory', 'timeFrame': '4 years', 'description': 'Change in the frequency of ventricular arrhythmias from baseline.'}], 'primaryOutcomes': [{'measure': 'Percentage of subjects who experienced at least 1 treatment emergent adverse event (TEAE) and/or 1 treatment emergent serious adverse event (TESAE).', 'timeFrame': '4 years', 'description': 'Evaluation of Safety and Tolerability of LX2020'}]}, 'oversightModule': {'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'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'], 'conditions': ['Arrhythmogenic Cardiomyopathy', 'PKP2-ACM', 'PKP2-ARVC']}, 'descriptionModule': {'briefSummary': '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).', 'detailedDescription': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '67 Years', 'minimumAge': '18 Years', 'samplingMethod': 'NON_PROBABILITY_SAMPLE', 'studyPopulation': 'Participants who received LX2020 in Study LX2020-01 will roll over into this study.', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Participants who received LX2020 in study LX2020-01\n\nExclusion Criteria:\n\n* Concurrent enrollment in any other clinical investigation involving use of an investigational agent.'}, 'identificationModule': {'nctId': 'NCT07050160', 'acronym': 'GRIT-PKP2', 'briefTitle': 'Long-term Follow-up Study of Gene Therapy for Arrhythmogenic Cardiomyopathy Due to a Plakophilin-2 Pathogenic Variant', 'organization': {'class': 'INDUSTRY', 'fullName': 'Lexeo Therapeutics'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'LX2020-02'}}, 'armsInterventionsModule': {'armGroups': [{'label': 'LX2020 treated participants', 'description': 'Participants who received LX2020 in Study LX2020-01'}]}, 'contactsLocationsModule': {'locations': [{'zip': '48109', 'city': 'Ann Arbor', 'state': 'Michigan', 'country': 'United States', 'facility': 'University of Michigan', 'geoPoint': {'lat': 42.27756, 'lon': -83.74088}}], 'overallOfficials': [{'name': 'Lexeo Clinical Trials', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Lexeo Therapeutics'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Lexeo Therapeutics', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}