[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"trial:NCT06228924":3,"trial-entities:NCT06228924":185,"trial-summary:NCT06228924":190},{"id":4,"nct_id":4,"org_study_id":5,"brief_title":6,"official_title":7,"overall_status":8,"completion_date":9,"status_verified_date":10,"last_update_date":11,"start_date":12,"sponsor_name":13,"lead_sponsor_class":14,"has_dmc":15,"brief_summary":16,"detailed_description":17,"conditions":18,"keywords":20,"study_type":31,"primary_purpose":32,"phases":33,"enrollment_info":35,"interventions":38,"primary_outcomes":46,"secondary_outcomes":53,"sex":61,"minimum_age":62,"maximum_age":63,"healthy_volunteers":64,"eligibility_criteria":65,"std_ages":82,"locations":85,"central_contacts":165,"overall_officials":173,"references":174,"see_also_links":175},"NCT06228924","TN-401-0012","Open-label, Dose Escalation Study of Safety and Preliminary Efficacy of TN-401 in Adults With PKP2 Mutation-associated ARVC","First-in-Human, Open-Label, Safety, Tolerability, Dose-Finding, Pharmacodynamic and Cardiac Transgene Expression Study of TN-401, a Recombinant Adeno-associated Virus Serotype 9 (AAV9) Containing Plakophilin-2 (PKP2) Transgene, in Adults With PKP2 Mutation-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)","RECRUITING","2029-10-01","2024-09","2025-02-06","2024-03-26","Tenaya Therapeutics","INDUSTRY",true,"This first-in-human study is designed to evaluate the safety, and preliminary efficacy (PD) of TN-401 gene therapy in adult patients with symptomatic PKP2 mutation-associated ARVC.","The RIDGE-1™ open-label Safety, Tolerability, Dose-finding, PD and Cardiac Transgene Expression Study will enroll up to 15 patients in two planned dose cohorts. Patients in each cohort will receive a single intravenous (IV) dose of TN-401. Following Data Safety Monitoring Board (DSMB) review of each dose cohort, the next dose cohort will be initiated. DSMB review will also be needed to expand the dose cohorts. The dose for Cohorts 1\u002F1a will be 3E13 (3 × 1013) vg\u002Fkg and the dose for Cohorts 2\u002F2a will be 6E13 (6 × 1013) vg\u002Fkg.",[19],"Arrhythmogenic Right Ventricular Cardiomyopathy",[21,22,23,24,25,26,27,28,29,30],"PKP2 Mutation Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)","Arrhythmogenic Cardiomyopathy (ACM)","PKP2-associated ARVC","PKP2-ARVC","PKP2-ACM","Adeno Associated Virus (AAV)","Gene Therapy","Gene Transfer","Genetic cardiomyopathy","Heart Failure","INTERVENTIONAL","TREATMENT",[34],"PHASE1",{"count":36,"type":37},15,"ESTIMATED",[39],{"type":40,"name":41,"description":42,"armGroupLabels":43},"GENETIC","TN-401","TN-401 is a recombinant adeno-associated virus serotype 9 (AAV9) gene therapy containing Plakophilin-2 (PKP2) transgene. It is a single (one-time) intravenous dose.",[44,45],"Cohort 1","Cohort 2",[47,50],{"measure":48,"timeFrame":49},"Number and severity of Adverse Events over the course of the study.","52 weeks",{"measure":51,"timeFrame":52},"Number of Serious Adverse Events related to study drug.","5 years",[54,57,59],{"measure":55,"timeFrame":56},"To assess changes in daily PVC and NSVT counts","Week 52",{"measure":58,"timeFrame":56},"To assess frequency of ICD therapy administration",{"measure":60,"timeFrame":56},"To assess frequency of sustained VT","ALL","18 Years","65 Years",false,{"inclusion":66,"exclusion":73,"raw_text":81},[67,68,69,70,71,72],"PKP2 mutation (pathogenic or likely pathogenic)","Arrhythmogenic Right Ventricular Cardiomyopathy as defined by the 2010 revised Task Force Criteria","Left Ventricular Ejection Fraction ≥50%","Functioning Implantable Cardiac Defibrillator with remote integration capabilities at least 9 months prior to Screening","NYHA Functional Class I, II, or III","Frequent premature ventricular contractions (PVCs)",[74,75,76,77,78,79,80],"Ventricular tachycardia (VT) ablation within 6 months of Screening or planned VT ablation within 6 months after Screening","High AAV9 neutralizing antibody titer","Prior myocardial infarction","Right Ventricular Heart Failure","Class IV Heart Failure","Clinically significant renal disease","Clinically significant liver disease","Inclusion Criteria:\n\n* PKP2 mutation (pathogenic or likely pathogenic)\n* Arrhythmogenic Right Ventricular Cardiomyopathy as defined by the 2010 revised Task Force Criteria\n* Left Ventricular Ejection Fraction ≥50%\n* Functioning Implantable Cardiac Defibrillator with remote integration capabilities at least 9 months prior to Screening\n* NYHA Functional Class I, II, or III\n* Frequent premature ventricular contractions (PVCs)\n\nExclusion Criteria:\n\n* Ventricular tachycardia (VT) ablation within 6 months of Screening or planned VT ablation within 6 months after Screening\n* High AAV9 neutralizing antibody titer\n* Prior myocardial infarction\n* Right Ventricular Heart Failure\n* Class IV Heart Failure\n* Clinically significant renal disease\n* Clinically significant liver disease",[83,84],"ADULT","OLDER_ADULT",[86,99,110,122,133,144,154],{"facility":87,"status":8,"city":88,"state":89,"zip":90,"country":91,"contacts":92,"geoPoint":96},"University of California San Francisco","San Francisco","California","94143","United States",[93],{"name":94,"role":95},"Vasanth Vedantham, MD","CONTACT",{"lat":97,"lon":98},37.77493,-122.41942,{"facility":100,"status":8,"city":101,"state":102,"zip":103,"country":91,"contacts":104,"geoPoint":107},"University of Colorado - Anschutz Medical Campus","Aurora","Colorado","80045",[105],{"name":106,"role":95},"Matthew Taylor, MD",{"lat":108,"lon":109},39.72943,-104.83192,{"facility":111,"status":8,"city":112,"state":113,"zip":114,"country":91,"contacts":115,"geoPoint":119},"Johns Hopkins University","Baltimore","Maryland","21287",[116],{"name":117,"role":118},"Andreas Barth, MD","PRINCIPAL_INVESTIGATOR",{"lat":120,"lon":121},39.29038,-76.61219,{"facility":123,"status":8,"city":124,"state":125,"zip":126,"country":91,"contacts":127,"geoPoint":130},"Brigham and Women's Hospital","Boston","Massachusetts","02115",[128],{"name":129,"role":95},"Neal Lakdawala, MD",{"lat":131,"lon":132},42.35843,-71.05977,{"facility":134,"status":8,"city":135,"state":136,"zip":137,"country":91,"contacts":138,"geoPoint":141},"Mayo Clinic","Rochester","Minnesota","55905",[139],{"name":140,"role":118},"John R. Giudicessi, MD",{"lat":142,"lon":143},44.02163,-92.4699,{"facility":145,"status":8,"city":146,"state":146,"zip":147,"country":91,"contacts":148,"geoPoint":151},"NYU Langone Health","New York","10016",[149],{"name":150,"role":95},"Larry Chinitz, MD",{"lat":152,"lon":153},40.71427,-74.00597,{"facility":155,"status":8,"city":156,"state":157,"zip":158,"country":91,"contacts":159,"geoPoint":162},"Cleveland Clinic","Cleveland","Ohio","44195",[160],{"name":161,"role":95},"Milind Desai, MD",{"lat":163,"lon":164},41.4995,-81.69541,[166,170],{"name":167,"role":95,"phone":168,"email":169},"Matthew Pollman, M.D.","(650) 416-1186","mpollman@tenayathera.com",{"name":171,"role":95,"email":172},"Niha Kamat","clinical.trials@tenayathera.com",[],[],[176,179,181,183],{"label":177,"url":178},"Related Info","https:\u002F\u002Fwww.tenayatherapeutics.com\u002Fwp-content\u002Fuploads\u002F2023-BCVS_PKP2-Novel-Model_Xu.pdf",{"label":177,"url":180},"https:\u002F\u002Fwww.tenayatherapeutics.com\u002Fwp-content\u002Fuploads\u002FPKP2-Gene-Therapy-for-Arrhythmogenic-Right-Ventricular-Cardiomyopathy.pdf",{"label":177,"url":182},"https:\u002F\u002Fwww.tenayatherapeutics.com\u002Fwp-content\u002Fuploads\u002F04152022_PKP2-Presentation_HRS2022_v6.pdf",{"label":177,"url":184},"https:\u002F\u002Fwww.tenayatherapeutics.com\u002Fwp-content\u002Fuploads\u002FCardiac-AAV9-PKP2-therapy-Abstract-final.pdf",{"nct_id":4,"conditions":186,"biomarkers":188},[187],"Arrhythmogenic Right Ventricular Dysplasia",[189],"PKP2 Gene",{"nct_id":4,"found":15,"summary":191,"prompt_version":201},{"design":192,"status":193,"heading":194,"summary":195,"follow_up":196,"word_count":197,"commitments":198,"compensation":199,"drugs_mentioned":200},"This is an open-label study, meaning you and your doctors will know which treatment you are receiving. It will involve up to 15 participants in two dose groups, each receiving a single intravenous dose of TN-401.","completed","TN-401 Gene Therapy for PKP2-related ARVC","This study is testing a new gene therapy called TN-401 for adults with Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC), a heart condition caused by a specific genetic change (PKP2 mutation). TN-401 is a one-time intravenous (into the vein) dose designed to deliver a healthy copy of the PKP2 gene. The main goals are to see how safe TN-401 is by tracking any side effects over 52 weeks and serious side effects over 5 years. You might be able to join if you are between 18 and 65 years old, have a confirmed PKP2 mutation and ARVC, and meet other heart health criteria, including having a functioning implanted defibrillator. The study plans to enroll up to 15 participants.","Participants will be followed for adverse events for 52 weeks and for serious adverse events related to the study drug for 5 years.",116,"You would receive a single intravenous dose of TN-401. The study will track your safety for 52 weeks and serious side effects for 5 years.","Not stated in the trial record.",[41],"v2"]