[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Tenaya Therapeutics\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":142},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,4,0,[8,54,88,119],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":15,"eligibilityCriteria":16,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":19,"enrollmentInfo":20,"targetDuration":4,"studyType":23,"phases":24,"briefSummary":27,"conditions":28,"keywords":30,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":42,"lastUpdatePostDateStruct":43,"startDateStruct":46,"completionDateStruct":48,"leadSponsor":50,"locationsCount":53},"100503515","phase-1-multi-center-open-label-single-ascending-dose-study-of-safety-and-tolerability-of-tn-201-in-adults-with-symptomatic-mybpc3-mutation-associated-hcm-100503515",false,"NCT05836259","Multi-center, Open-label, Single-ascending Dose Study of Safety and Tolerability of TN-201 in Adults With Symptomatic MYBPC3 Mutation-associated HCM","First-in-Human, Open-Label, Safety, Tolerability, Dose-Finding, Pharmacodynamic and Cardiac Transgene Expression Study of TN-201, a Recombinant Adeno-associated Virus Serotype 9 (AAV9) Containing Myosin Binding Protein C Transgene, in Adults With MYBPC3 Mutation-associated Hypertrophic Cardiomyopathy (HCM)","MyPEAK-1","Inclusion Criteria:\n\n* MYBPC3 mutation\n* Hypertrophic Cardiomyopathy (obstructive and nonobstructive)\n* Left Ventricular Ejection Fraction ≥45%\n* NYHA Functional Class II or III symptoms\n* NT-proBNP ≥160pg\u002Fml\n\nExclusion Criteria:\n\n* High AAV9 neutralizing antibody titer","ALL","18 Years","75 Years",{"count":21,"type":22},30,"ESTIMATED","INTERVENTIONAL",[25,26],"PHASE1","PHASE2","This is a first-in-human, non-randomized, open-label study designed to evaluate the safety, tolerability, and pharmacodynamics (PD) of TN-201 in adult patients with symptomatic hypertrophic cardiomyopathy (HCM) caused by mutations in the MYBPC3 gene.",[29],"Hypertrophic Cardiomyopathy",[31,32,33,34,35,36,37,38,39,40],"Hypertrophic Cardiomyopathy (HCM)","Myosin Binding Protein C3 (MYBPC3)","Nonobstructive HCM","Genetic HCM","Familial HCM","Adenoassociated Virus (AAV)","Gene Therapy","Obstructive HCM","nHCM","oHCM","RECRUITING","2026-03-18",{"date":44,"type":45},"2026-03-19","ACTUAL",{"date":47,"type":45},"2023-08-10",{"date":49,"type":22},"2032-08",{"name":51,"class":52},"Tenaya Therapeutics","INDUSTRY",10,{"id":55,"slug":56,"hasResults":11,"nctId":57,"briefTitle":58,"officialTitle":59,"acronym":60,"eligibilityCriteria":61,"healthyVolunteers":11,"sex":17,"minAge":18,"maxAge":62,"enrollmentInfo":63,"targetDuration":4,"studyType":23,"phases":65,"briefSummary":66,"conditions":67,"keywords":69,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":79,"lastUpdatePostDateStruct":80,"startDateStruct":82,"completionDateStruct":84,"leadSponsor":86,"locationsCount":87},"100533677","phase-1-open-label-dose-escalation-study-of-safety-and-preliminary-efficacy-of-tn-401-in-adults-with-pkp2-mutation-associated-arvc-100533677","NCT06228924","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)","RIDGE-1","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","65 Years",{"count":64,"type":22},15,[25],"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.",[68],"Arrhythmogenic Right Ventricular Cardiomyopathy",[70,71,72,73,74,75,37,76,77,78],"PKP2 Mutation Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)","Arrhythmogenic Cardiomyopathy (ACM)","PKP2-associated ARVC","PKP2-ARVC","PKP2-ACM","Adeno Associated Virus (AAV)","Gene Transfer","Genetic cardiomyopathy","Heart Failure","2025-02-05",{"date":81,"type":45},"2025-02-06",{"date":83,"type":45},"2024-03-26",{"date":85,"type":22},"2029-10-01",{"name":51,"class":52},7,{"id":89,"slug":90,"hasResults":11,"nctId":91,"briefTitle":92,"officialTitle":93,"acronym":94,"eligibilityCriteria":95,"healthyVolunteers":11,"sex":17,"minAge":96,"maxAge":18,"enrollmentInfo":97,"targetDuration":4,"studyType":99,"phases":4,"briefSummary":100,"conditions":101,"keywords":103,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":110,"lastUpdatePostDateStruct":111,"startDateStruct":113,"completionDateStruct":115,"leadSponsor":117,"locationsCount":118},"100447884","natural-history-study-in-pediatric-patients-with-mybpc3-mutation-associated-cardiomyopathy-100447884","NCT05112237","Natural History Study in Pediatric Patients With MYBPC3 Mutation-associated Cardiomyopathy","A Prospective and Retrospective Registry and Biomarker Study to Evaluate the Natural History of Pediatric Patients With Cardiomyopathy Due to MYBPC3 Mutations","MyCLIMB","Retrospective\n\nInclusion Criteria:\n\n* Data is available for patient \\\u003C18 years of age. Patients must be \\\u003C18 years of age at enrollment or at time of death.\n* Documented results of genotyping showing the presence of at least one pathogenic or likely pathogenic MYBPC3 mutation (heterozygous, homozygous, or compound heterozygous).\n\nExclusion Criteria:\n\n* Patient received cardiac transplantation or died \\>10 years before study initiation. For homozygous or biallelic infants, data may be collected beyond this 10-year period.\n\nProspective\n\nInclusion Criteria:\n\nFor Infants:\n\n* Infants who are homozygous or compound heterozygous for the known pathogenic truncating MYBPC3 mutations are eligible.\n\nFor all other participants:\n\n* Age \\\u003C18 at entry into the prospective study.\n* Documented results of genotyping identifying at least one pathogenic or likely pathogenic MYBPC3 mutation (heterozygous, homozygous, or compound heterozygous).\n* Diagnosis of Cardiomyopathy (CM): HCM, DCM, RCM, mixed CM, or LVNC.\n\nExclusion Criteria:\n\n* Concurrent participation in an interventional clinical trial unless approved by the sponsor.\n* Severe noncardiac disease anticipated to significantly reduce life expectancy.","0 Years",{"count":98,"type":22},200,"OBSERVATIONAL","The objective of this study is to collect information on patients with cardiomyopathy (CM) due to mutations in the MYBPC3 gene, to evaluate their disease course, burden of illness, risk factors for this disease, and the quality of life (QoL). This study will also collect information on treatments, procedures and outcome in infants and children up to 18 yrs who have this mutation.",[102],"Cardiomyopathy",[104,105,106,31,107,108,109],"Genetic Cardiomyopathy","Pediatric","MYBPC3","Dilated Cardiomyopathy","Restrictive Cardiomyopathy","Non-Compaction cardiomyopathy","2024-11-11",{"date":112,"type":45},"2024-11-13",{"date":114,"type":45},"2021-11-01",{"date":116,"type":22},"2028-06",{"name":51,"class":52},29,{"id":120,"slug":121,"hasResults":11,"nctId":122,"briefTitle":123,"officialTitle":124,"acronym":125,"eligibilityCriteria":126,"healthyVolunteers":11,"sex":17,"minAge":127,"maxAge":62,"enrollmentInfo":128,"targetDuration":4,"studyType":99,"phases":4,"briefSummary":129,"conditions":130,"keywords":131,"overallStatus":41,"whyStopped":4,"lastUpdateSubmitDate":133,"lastUpdatePostDateStruct":134,"startDateStruct":136,"completionDateStruct":138,"leadSponsor":140,"locationsCount":141},"100540043","non-interventional-study-of-seroprevalence-of-pre-existing-antibodies-against-adenovirus-associated-virus-vector-aav9-and-the-progression-of-disease-in-patients-with-plakophilin-2-pkp2-associated-arrhythmogenic-right-ventricular-cardiomyopathy-arvc-100540043","NCT06311708","Non-interventional Study of Seroprevalence of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) and the Progression of Disease in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)","Seroprevalence Study of Pre-existing Antibodies Against Adenovirus-associated Virus Vector (AAV9) in Patients With Plakophilin 2 (PKP2)-Associated Arrhythmogenic Right Ventricular Cardiomyopathy (ARVC)","RIDGE","Inclusion Criteria:\n\n* Ages 14-65 years, inclusive, at the time of consent\n* Pathogenic or likely pathogenic PKP2 gene mutation\n* Diagnosed with ARVC and meet 2010 Modified Task Force Criteria for ARVC as affected.\n* Functioning ICD\n\nExclusion Criteria:\n\n* Currently receiving systemic immunosuppressive therapy, cytotoxic chemotherapy, immunoglobulin therapy or monoclonal antibody therapy\n* History of clinically significant liver disease, hepatitis B virus, hepatitis C virus, human immunodeficiency virus, or tuberculosis infection\n* Previously dosed with any investigational or approved gene therapy product at any time\n* Concurrent participation in another interventional clinical trial unless approved by the Sponsor. Participation in a noninterventional study may be allowed at the investigator's discretion.\n* History of cardiac transplant.","14 Years",{"count":98,"type":22},"This is a multicenter, non-interventional study to observe the natural progression of the disease and to study the prevalence of pre-existing antibodies to AAV9 used for gene therapy in a population of patients with PKP2 gene-associated ARVC. Participation from all patients is encouraged regardless of interest in or eligibility for gene therapy.",[68],[70,71,72,73,74,75,132,37,76,77,78],"Adeno-Associated Virus Serotype 9 (AAV9)","2024-10-30",{"date":135,"type":45},"2024-11-01",{"date":137,"type":45},"2023-01-31",{"date":139,"type":22},"2030-07-11",{"name":51,"class":52},21,""]