[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100507286":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":20,"centralContacts":25,"locations":31,"responsibleParty":76,"collaborators":19,"id":78,"slug":79,"hasResults":80,"nctId":81,"briefTitle":82,"officialTitle":83,"acronym":19,"eligibilityCriteria":84,"healthyVolunteers":80,"sex":85,"minAge":86,"maxAge":19,"enrollmentInfo":87,"targetDuration":19,"studyType":90,"phases":91,"briefSummary":93,"conditions":94,"keywords":96,"overallStatus":33,"whyStopped":19,"lastUpdateSubmitDate":109,"lastUpdatePostDateStruct":110,"startDateStruct":113,"completionDateStruct":115,"leadSponsor":117,"locationsCount":118},{"fullName":5,"class":6},"Rocket Pharmaceuticals Inc.","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"RP-A601","EXPERIMENTAL","Single ascending dose of RP-A601 in 2 consecutive cohorts",[13],"Genetic: RP-A601",[15],{"type":16,"name":9,"description":17,"armGroupLabels":18,"otherNames":19},"GENETIC","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)",[9],null,[21],{"name":22,"affiliation":23,"role":24},"Barry Greenberg, MD","University of California, San Diego","PRINCIPAL_INVESTIGATOR",[26],{"name":27,"role":28,"phone":29,"phoneExt":19,"email":30},"Clinical Information","CONTACT","646-627-0033","clinicaltrials@rocketpharma.com",[32,49,65],{"facility":23,"status":33,"city":34,"state":35,"zip":36,"country":37,"countryCode":38,"cosmosGeoPoint":39,"geoPoint":44,"contacts":45},"RECRUITING","La Jolla","California","92037","United States","US",{"type":40,"coordinates":41},"Point",[42,43],-117.2742,32.84727,{"lat":43,"lon":42},[46],{"name":22,"role":28,"phone":47,"phoneExt":19,"email":48},"858-246-2987","Bgreenberg@health.ucsd.edu",{"facility":50,"status":33,"city":51,"state":52,"zip":53,"country":37,"countryCode":38,"cosmosGeoPoint":54,"geoPoint":58,"contacts":59},"Duke University","Durham","North Carolina","27710",{"type":40,"coordinates":55},[56,57],-78.89862,35.99403,{"lat":57,"lon":56},[60,63],{"name":61,"role":28,"phone":19,"phoneExt":19,"email":62},"Meghan Pergola, MPH","meghan.pergola@duke.edu",{"name":64,"role":24,"phone":19,"phoneExt":19,"email":19},"James Daubert, MD",{"facility":66,"status":67,"city":68,"state":69,"zip":70,"country":37,"countryCode":38,"cosmosGeoPoint":71,"geoPoint":75,"contacts":19},"Children's Hospital of Philadelphia","WITHDRAWN","Philadelphia","Pennsylvania","19104",{"type":40,"coordinates":72},[73,74],-75.16362,39.95238,{"lat":74,"lon":73},{"type":77,"investigatorFullName":19,"investigatorTitle":19,"investigatorAffiliation":19,"oldNameTitle":19,"oldOrganization":19},"SPONSOR","100507286","phase-1-a-phase-1-dose-escalation-trial-of-rp-a601-in-subjects-with-pkp2-variant-mediated-arrhythmogenic-cardiomyopathy-pkp2-acm-100507286",false,"NCT05885412","A Phase 1, Dose Escalation Trial of RP-A601 in Subjects With PKP2 Variant-Mediated Arrhythmogenic Cardiomyopathy (PKP2-ACM)","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)","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.","ALL","18 Years",{"count":88,"type":89},9,"ESTIMATED","INTERVENTIONAL",[92],"PHASE1","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.",[95],"PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)",[97,98,99,100,101,102,103,104,105,106,107,108],"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","2026-04-14",{"date":111,"type":112},"2026-04-16","ACTUAL",{"date":114,"type":112},"2023-08-29",{"date":116,"type":89},"2029-09",{"name":5,"class":6},3]