Ignite Creation Date:
2025-12-26 @ 11:13 PM
Ignite Modification Date:
2026-03-17 @ 11:45 PM
Study NCT ID:
NCT05885412
Status:
RECRUITING
Last Update Posted:
2024-10-04
First Post:
2023-05-22
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D019571', 'term': 'Arrhythmogenic Right Ventricular Dysplasia'}, {'id': 'D016757', 'term': 'Death, Sudden, Cardiac'}, {'id': 'D006323', 'term': 'Heart Arrest'}], 'ancestors': [{'id': 'D006330', 'term': 'Heart Defects, Congenital'}, {'id': 'D018376', 'term': 'Cardiovascular Abnormalities'}, {'id': 'D002318', 'term': 'Cardiovascular Diseases'}, {'id': 'D009202', 'term': 'Cardiomyopathies'}, {'id': 'D006331', 'term': 'Heart Diseases'}, {'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': {'phases': ['PHASE1'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 9}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2023-08-29', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2024-10', 'completionDateStruct': {'date': '2026-09', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2024-10-02', 'studyFirstSubmitDate': '2023-05-22', 'studyFirstSubmitQcDate': '2023-05-22', 'lastUpdatePostDateStruct': {'date': '2024-10-04', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2023-06-02', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-09', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Evaluation of safety associated with RP-A601', 'timeFrame': '12 months post-infusion', 'description': 'Incidence of treatment emergent adverse events (TEAEs), incidence of Serious Adverse Events (SAEs), and identification of Dose Limiting Toxicities (DLTs)'}], 'secondaryOutcomes': [{'measure': 'Preliminary efficacy of RP-A601 - Myocardial PKP2 protein expression', 'timeFrame': '12 months post-infusion', 'description': 'Assessment of changes in myocardial PKP2 protein expression'}, {'measure': 'Preliminary efficacy of RP-A601 - Ventricular ectopy and arrhythmia', 'timeFrame': '12 months post-infusion', 'description': 'Assessment of changes in levels of ventricular ectopy and arrhythmia on cardiac rhythm monitoring'}, {'measure': 'Preliminary efficacy of RP-A601 - Cardiac biomarkers', 'timeFrame': '12 months post-infusion', 'description': 'Assessment of changes in circulating levels of cardiac biomarkers of injury and stress'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Arrhythmogenic cardiomyopathy', 'Arrhythmogenic Right Ventricular Cardiomyopathy', 'Arrhythmogenic Right Ventricular Dysplasia', 'Sudden Cardiac Death', 'Genetic cardiomyopathy', 'Gene therapy', 'PKP2', 'ARVC', 'ARVD', 'ACM', 'Cardiac Arrest', 'Ventricular Arrhythmia'], 'conditions': ['PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)']}, 'descriptionModule': {'briefSummary': 'This Phase 1 dose escalation trial will assess the safety and preliminary efficacy of a single dose intravenous infusion of RP-A601 in high-risk adult patients with PKP2-ACM.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n1. Male or female ≥18 years at the time of signing the informed consent\n2. Capable and willing to provide signed informed consent\n3. Clinical diagnosis of ACM as defined by the 2010 revised Task Force Criteria (TFC)\n4. Documentation of a pathogenic or likely pathogenic truncating variant in PKP2\n5. History of Implantable Cardioverter-Defibrillator (ICD) implantation ≥6 months prior to enrollment\n6. PVC frequency ≥500 per 24 hours by ambulatory rhythm monitoring\n7. Left ventricular ejection fraction by echocardiogram or CMR ≥50%\n\nKey Exclusion Criteria:\n\n1. Anti-AAVrh.74 capsid neutralizing antibody titer of \\>1:40\n2. Cardiomyopathy related to a genetic etiology other than PKP2 truncating variant\n3. Previous participation in a study of gene transfer or gene editing\n4. Severe Right ventricular (RV) dysfunction\n5. New York Heart Association (NYHA) Class IV heart failure.'}, 'identificationModule': {'nctId': 'NCT05885412', 'briefTitle': 'A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Rocket Pharmaceuticals Inc.'}, 'officialTitle': 'A Phase 1 Dose Escalation Trial Evaluating an Intravenously Administered Recombinant Adeno-Associated Virus Serotype rh.74 (AAVrh.74) Vector Containing the Human Plakophilin-2a (PKP2a) Coding Sequence (RP-A601; AAVrh.74-PKP2a) in Subjects With Arrhythmogenic Cardiomyopathy Arising From Pathogenic PKP2 Variants (PKP2-ACM)', 'orgStudyIdInfo': {'id': 'RP-A601-0323'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'RP-A601', 'description': 'Single ascending dose of RP-A601 in 2 consecutive cohorts', 'interventionNames': ['Genetic: RP-A601']}], 'interventions': [{'name': 'RP-A601', 'type': 'GENETIC', 'description': 'RP-A601 is a recombinant viral vector composed of an AAV serotype rh.74 (AAVrh.74) capsid encapsulating the transgene, human plakophilin 2 (PKP2), transcript variant 2a (PKP2a)', 'armGroupLabels': ['RP-A601']}]}, 'contactsLocationsModule': {'locations': [{'zip': '92037', 'city': 'La Jolla', 'state': 'California', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Barry Greenberg, MD', 'role': 'CONTACT', 'email': 'Bgreenberg@health.ucsd.edu', 'phone': '858-246-2987'}], 'facility': 'University of California, San Diego', 'geoPoint': {'lat': 32.84727, 'lon': -117.2742}}, {'zip': '27710', 'city': 'Durham', 'state': 'North Carolina', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Meghan Pergola, MPH', 'role': 'CONTACT', 'email': 'meghan.pergola@duke.edu'}, {'name': 'James Daubert, MD', 'role': 'PRINCIPAL_INVESTIGATOR'}], 'facility': 'Duke University', 'geoPoint': {'lat': 35.99403, 'lon': -78.89862}}, {'zip': '19104', 'city': 'Philadelphia', 'state': 'Pennsylvania', 'status': 'RECRUITING', 'country': 'United States', 'contacts': [{'name': 'Joseph Rossano, MD', 'role': 'CONTACT', 'email': 'rossanoj@chop.edu'}], 'facility': "Children's Hospital of Philadelphia", 'geoPoint': {'lat': 39.95238, 'lon': -75.16362}}], 'centralContacts': [{'name': 'Clinical Information', 'role': 'CONTACT', 'email': 'clinicaltrials@rocketpharma.com', 'phone': '646-627-0033'}], 'overallOfficials': [{'name': 'Barry Greenberg, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of California, San Diego'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Rocket Pharmaceuticals Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}