[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"Rocket Pharmaceuticals Inc.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":197},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,6,0,[8,59,86,116,149,173],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":28,"overallStatus":46,"whyStopped":4,"lastUpdateSubmitDate":47,"lastUpdatePostDateStruct":48,"startDateStruct":51,"completionDateStruct":53,"leadSponsor":55,"locationsCount":58},"100603501","phase-1-a-phase-1-aav-gene-therapy-trial-evaluating-safety-and-preliminary-efficacy-of-rp-a701-in-subjects-with-bag3-dilated-cardiomyopathy-100603501",false,"NCT07137338","A Phase 1 AAV Gene Therapy Trial Evaluating Safety and Preliminary Efficacy of RP-A701 in Subjects With BAG3 Dilated Cardiomyopathy","A Phase 1 Dose Escalation Trial Evaluating an Intravenously Administered Recombinant Adeno-associated Virus Serotype rh.74 (AAVrh.74) Vector Containing the Human BCL2-associated Athanogene 3 (BAG3) Gene Coding Sequence (RP-A701) in Subjects With Dilated Cardiomyopathy Arising From Pathogenic BAG3 Variants (BAG3-DCM)","Inclusion Criteria:\n\nSubjects are eligible for inclusion into the study only if all the following criteria apply:\n\n1. Male or female between 18 and 65 years of age at the time of signing the informed consent\n2. Capable of and willing to provide signed informed consent\n3. Clinical diagnosis of DCM defined as and requiring each of the following:\n\n   1. Mild to moderate systolic dysfunction (LVEF ≥ 25% and ≤ 45%) by echocardiography or CMR performed within 3 months of enrollment.\n   2. Absence of severe coronary artery disease (\\>70% stenosis) or active myocardial ischemia as the etiology of LV systolic dysfunction\n   3. Absence of uncontrolled hypertension, significant cardiac valve disease (i.e., greater than moderate in severity), infiltrative disorder, or systemic disease known to cause cardiomyopathy.\n4. Documentation of a pathogenic or likely pathogenic variant in BAG3\n5. History of ICD implantation ≥ 3 months prior to enrollment\n6. NYHA Class II or III HF symptoms with stable HF therapeutic guideline-directed medical regimen for 30 days prior to enrollment\n\nExclusion Criteria:\n\n1. CV disease that may be related to a genetic etiology other than a BAG3 pathogenic or likely pathogenic variant.\n2. Previous participation in a study of gene transfer or gene editing.\n3. I.V. inotropic, vasodilator, or diuretic therapy ≤ 30 days prior to enrollment.\n4. History of intracardiac thrombosis or arterial thromboembolic events\n5. Severe RV dysfunction assessed by echocardiogram or CMR ≤ 12 months prior to screening\n6. LVEF \\\u003C 25% by echocardiogram or CMR at ≤ 3 months prior to screening\n7. NYHA Class I or IV HF","ALL","18 Years","65 Years",{"count":20,"type":21},8,"ESTIMATED","INTERVENTIONAL",[24],"PHASE1","This is a Phase 1, open-label, dose-escalation trial to characterize the safety, tolerability, and preliminary efficacy of RP-A701 following a single IV administration in high-risk adult patients with BAG3-DCM.",[27],"Dilated Cardiomyopathy (DCM)",[29,30,31,32,33,34,35,36,37,38,39,40,41,42,43,44,45],"Cardiomyopathy","Dilated","BAG3 Protein","human","dilated cardiomyopathy","DCM","BAG3","BCL2-associated Athanogene 3","Genetic Cardiomyopathy","Gene Therapy","Heart Failure","Ventricular Arrhythmia","Cardiovascular Diseases","Cardiomyopathies","Heart Diseases","Inherited heart disease","AAVrh.74","RECRUITING","2026-06-26",{"date":49,"type":50},"2026-06-30","ACTUAL",{"date":52,"type":21},"2026-06",{"date":54,"type":21},"2029-06",{"name":56,"class":57},"Rocket Pharmaceuticals Inc.","INDUSTRY",3,{"id":60,"slug":61,"hasResults":11,"nctId":62,"briefTitle":63,"officialTitle":64,"acronym":4,"eligibilityCriteria":65,"healthyVolunteers":11,"sex":16,"minAge":66,"maxAge":4,"enrollmentInfo":67,"targetDuration":4,"studyType":69,"phases":4,"briefSummary":70,"conditions":71,"keywords":73,"overallStatus":46,"whyStopped":4,"lastUpdateSubmitDate":77,"lastUpdatePostDateStruct":78,"startDateStruct":80,"completionDateStruct":82,"leadSponsor":84,"locationsCount":85},"100532569","danon-disease-natural-history-study-100532569","NCT06214507","Danon Disease Natural History Study","An Observational Study of Genetic Cardiomyopathy, Danon Disease","Prospective Cohort:\n\n1. Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene by a CLIA-certified genetic testing laboratory\n2. Patient or parent\u002Flegal guardian are capable and willing to provide signed informed consent\n3. Age ≥ 8 years at enrollment\n\n   Female Prospective Cohort:\n4. Evidence of left ventricular hypertrophy in the 12 months prior to or at enrollment.\n\n   Retrospective (only) Cohort:\n5. Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene by a CLIA-certified genetic testing laboratory\n6. Patient or parent\u002Flegal guardian are capable and willing to provide signed informed consent, as required by local regulations\n7. Age ≥ 8 years at enrollment\n8. Prior cardiac transplantation or prior mechanical circulatory support\n9. At least 30 days of retrospective medical records available prior to cardiac transplantation or mechanical circulatory support\n\n   Female Retrospective (only) Cohort:\n10. Prior evidence of left ventricular hypertrophy.\n\nKey Exclusion Criteria:\n\nAll Cohorts:\n\n1. Concurrent enrollment in any other clinical investigation involving use of an investigational agent for any condition at time of enrollment to this study that could confound interpretation of this study\n2. Previous treatment with a gene therapy\n\n   Prospective Cohort:\n3. Prior mechanical circulatory support at time of enrollment to this study\n4. Prior cardiac transplantation at time of enrollment to this study\n\n   Female patients:\n5. Age \\>51 years at enrollment","8 Years",{"count":68,"type":21},60,"OBSERVATIONAL","The goal of this international observational study is to learn about the natural history of Danon disease in male patients (≥8 years of age) and female patients (8 to 50 years of age).",[72],"Danon Disease",[72,74,75,29,76],"LAMP2","Lysosome-associated membrane protein 2","X-linked","2026-04-29",{"date":79,"type":50},"2026-05-05",{"date":81,"type":50},"2023-12-20",{"date":83,"type":21},"2030-03",{"name":56,"class":57},13,{"id":87,"slug":88,"hasResults":11,"nctId":89,"briefTitle":90,"officialTitle":91,"acronym":4,"eligibilityCriteria":92,"healthyVolunteers":11,"sex":93,"minAge":66,"maxAge":4,"enrollmentInfo":94,"targetDuration":4,"studyType":22,"phases":96,"briefSummary":98,"conditions":99,"keywords":100,"overallStatus":46,"whyStopped":4,"lastUpdateSubmitDate":108,"lastUpdatePostDateStruct":109,"startDateStruct":111,"completionDateStruct":113,"leadSponsor":115,"locationsCount":5},"100523162","phase-2-a-gene-therapy-study-of-rp-a501-in-male-patients-with-danon-disease-100523162","NCT06092034","A Gene Therapy Study of RP-A501 in Male Patients With Danon Disease","Gene Therapy for Danon Disease: A Phase 2 Study Evaluating the Efficacy and Safety of Intravenously Administered Adeno-Associated Virus Serotype 9 (rAAV9) Vector Containing the Human LAMP2 Isoform B Transgene (RP-A501; AAV9.LAMP2B) in Male Patients With Danon Disease","Key Inclusion Criteria:\n\n1. Documentation of a pathogenic or likely pathogenic variant of the LAMP2 gene.\n2. Male.\n3. Age ≥8 years.\n4. Evidence of left ventricular hypertrophy with preserved systolic function phenotype as defined by each of the following:\n\n   1. Abnormal thickening of Left ventricular wall,\n   2. Left ventricular ejection fraction (LVEF) ≥ 50%.\n5. New York Heart Association (NYHA) Class II to III.\n6. High sensitivity Troponin I (hsTnI) ≥20% above the upper limit of normal (ULN)\n7. Ability to comply with study procedures including investigational therapy and follow-up evaluations.\n\nKey Exclusion Criteria:\n\n1. Anti-AAV9 neutralizing antibody titer \\>1:40.\n2. Severe heart failure or requirement for advanced therapies.\n3. History of intracardiac thrombosis or arterial thromboembolic events including stroke, transient ischemic attack (TIA), acute coronary syndrome, myocardial infarction or unstable angina.\n4. Prior cardiac or other organ (lung, liver, other) transplantation.","MALE",{"count":95,"type":21},14,[97],"PHASE2","This is a single arm Phase 2 trial to evaluate the efficacy and safety of RP-A501, a recombinant adeno-associated virus serotype 9 (AAV9) containing the human lysosome-associated membrane protein 2 isoform B (LAMP2B) transgene, in male patients with Danon Disease.",[72],[101,102,103,74,104,105,29,106,107],"Hypertrophic Cardiomyopathy","HCM","X-linked disease","Pediatric","Skeletal Myopathies","Lysosomal Storage Disorder","Left Ventricular Hypertrophy","2026-04-28",{"date":110,"type":50},"2026-05-04",{"date":112,"type":50},"2023-09-05",{"date":114,"type":21},"2032-04",{"name":56,"class":57},{"id":117,"slug":118,"hasResults":11,"nctId":119,"briefTitle":120,"officialTitle":121,"acronym":4,"eligibilityCriteria":122,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":123,"targetDuration":4,"studyType":22,"phases":125,"briefSummary":126,"conditions":127,"keywords":129,"overallStatus":46,"whyStopped":4,"lastUpdateSubmitDate":141,"lastUpdatePostDateStruct":142,"startDateStruct":144,"completionDateStruct":146,"leadSponsor":148,"locationsCount":58},"100507286","phase-1-a-phase-1-dose-escalation-trial-of-rp-a601-in-subjects-with-pkp2-variant-mediated-arrhythmogenic-cardiomyopathy-pkp2-acm-100507286","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.",{"count":124,"type":21},9,[24],"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.",[128],"PKP2 Arrhythmogenic Cardiomyopathy (PKP2-ACM)",[130,131,132,133,134,135,136,137,138,139,140,40],"Arrhythmogenic cardiomyopathy","Arrhythmogenic Right Ventricular Cardiomyopathy","Arrhythmogenic Right Ventricular Dysplasia","Sudden Cardiac Death","Genetic cardiomyopathy","Gene therapy","PKP2","ARVC","ARVD","ACM","Cardiac Arrest","2026-04-14",{"date":143,"type":50},"2026-04-16",{"date":145,"type":50},"2023-08-29",{"date":147,"type":21},"2029-09",{"name":56,"class":57},{"id":150,"slug":151,"hasResults":11,"nctId":152,"briefTitle":153,"officialTitle":154,"acronym":4,"eligibilityCriteria":155,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":4,"enrollmentInfo":156,"targetDuration":4,"studyType":69,"phases":4,"briefSummary":158,"conditions":159,"keywords":161,"overallStatus":164,"whyStopped":4,"lastUpdateSubmitDate":165,"lastUpdatePostDateStruct":166,"startDateStruct":168,"completionDateStruct":170,"leadSponsor":172,"locationsCount":4},"100630367","bag3-dcm-natural-history-study-100630367","NCT07486752","BAG3-DCM Natural History Study","An Observational Study of Patients With Dilated Cardiomyopathy (DCM) Associated With Pathogenic BAG3 Variants","Key Inclusion Criteria:\n\nSubjects are eligible for inclusion into the study only if all the following criteria apply:\n\nGeneral:\n\n1. Adult patients 18 years or older at the time of providing informed consent (i.e., signing the ICF).\n2. Capable and willing to provide signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and protocol.\n3. Diagnosis of DCM as defined by mild to moderate systolic dysfunction performed within 12 months of enrollment and confirmed by the principal investigator that the DCM is predominantly non-ischemic.\n4. Documentation of a pathogenic or likely pathogenic variant in BAG3 by a CLIA-certified or equivalent genetic testing laboratory.\n5. NYHA class I-III\n\nKey Exclusion Criteria:\n\nAll Cohorts:\n\n1\\. Concurrent enrollment in any other clinical investigation involving use of an investigational agent for any condition at time of enrollment to this study that could confound interpretation of this study results 2. Previous treatment with gene therapy 2. Gene testing indicates that the patient's arrhythmia or cardiomyopathy may be related to a genetic etiology other than BAG3 variant.\n\n4\\. NYHA class IV HF. 5. Presence or requirement for MCS or predicted need for MCS or heart transplantation within 6 months prior to enrollment.\n\n6\\. Prior heart transplantation. 7. Known infection with human immunodeficiency virus (HIV). 8. Unwillingness to comply with study procedures, including follow-up as specified by this protocol, or unwillingness to fully cooperate with the investigator.",{"count":157,"type":21},30,"The goal of this international observational study is to learn about the natural history of Dilated Cardiomyopathy (DCM) arising from pathogenic BAG3 variants in adult patients ≥18 years of age.",[27,41,43,160],"Genetic Diseases",[35,162,163,29,36],"Dilated Cardiomyopathy","BAG3-DCM","NOT_YET_RECRUITING","2026-03-17",{"date":167,"type":50},"2026-03-20",{"date":169,"type":21},"2026-05",{"date":171,"type":21},"2033-03",{"name":56,"class":57},{"id":174,"slug":175,"hasResults":11,"nctId":176,"briefTitle":177,"officialTitle":178,"acronym":4,"eligibilityCriteria":179,"healthyVolunteers":11,"sex":16,"minAge":180,"maxAge":4,"enrollmentInfo":181,"targetDuration":4,"studyType":69,"phases":4,"briefSummary":183,"conditions":184,"keywords":185,"overallStatus":46,"whyStopped":4,"lastUpdateSubmitDate":189,"lastUpdatePostDateStruct":190,"startDateStruct":192,"completionDateStruct":194,"leadSponsor":196,"locationsCount":58},"100565635","pkp2-acm-natural-history-study-100565635","NCT06644742","PKP2-ACM Natural History Study","The Natural History of Arrhythmogenic Cardiomyopathy With Pathogenic Plakophilin-2 Variants (PKP2-ACM): An Observational Cohort Study","Inclusion Criteria\n\nPatients must meet all the following criteria (and none of the exclusion criteria) to be eligible for study participation:\n\n1. Male or female age 12 years or older at the time of providing informed consent (i.e., ICF provision).\n2. Capable and willing to provide signed informed consent and\u002For assent, which includes compliance with the requirements and restrictions listed in the ICF and protocol.\n3. Clinical diagnosis of arrhythmogenic cardio myopathy (ACM)\n4. Documentation of a pathogenic or likely pathogenic variant in PKP2 by a CLIA-certified genetic testing laboratory\n5. History of ICD implantation ≥6 months prior to ICF provision\n6. Left ventricular ejection fraction by echocardiogram or cardiac magnetic resonance (CMR) ≥50% at ≤12 months prior to ICF provision\n\nExclusion Criteria\n\nPatients meeting any of the following criteria are excluded from study participation:\n\n1. Gene testing indicates that the subject's arrhythmia or cardiomyopathy may be related to a genetic etiology other than PKP2 truncating variant.\n2. Concurrent participation in any other clinical investigation involving use of an investigational agent that could confound results of this study.\n3. Previous participation in a study of gene transfer or gene editing.\n4. NYHA Class IV heart failure.\n5. Presence or requirement for mechanical circulatory support (MCS) or predicted need for MCS or heart transplantation within 6 months of enrollment.\n6. Prior cardiac or other organ (lung, liver, other) transplantation.\n7. Pacemaker dependent rhythm documented, as assessed by the principal investigator ≤12 months prior to enrollment.\n8. Positive human immunodeficiency virus (HIV) antibody test.\n9. Unwillingness to comply with study procedures, including follow-up as specified by this protocol, or unwillingness to fully cooperate with the investigator.","12 Years",{"count":182,"type":21},36,"The goal of this study is to describe the natural history and clinical events for patients who have Arrhythmogenic Cardiomyopathy with Pathogenic Plakophilin-2 Variants (PKP2-ACM) managed with standard of care.",[42,43,41,160],[186,187,188,136],"Arrhythmogenic Cardiomyopathy","Plakophilin-2","Arrhythmogenic Cardiomyopathy (AC, ARVD\u002FC)","2026-02-24",{"date":191,"type":50},"2026-02-27",{"date":193,"type":21},"2026-03",{"date":195,"type":21},"2031-04",{"name":56,"class":57},""]