[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100523162":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":20,"centralContacts":28,"locations":34,"responsibleParty":125,"collaborators":19,"id":127,"slug":128,"hasResults":129,"nctId":130,"briefTitle":131,"officialTitle":132,"acronym":19,"eligibilityCriteria":133,"healthyVolunteers":129,"sex":134,"minAge":135,"maxAge":19,"enrollmentInfo":136,"targetDuration":19,"studyType":139,"phases":140,"briefSummary":142,"conditions":143,"keywords":145,"overallStatus":36,"whyStopped":19,"lastUpdateSubmitDate":155,"lastUpdatePostDateStruct":156,"startDateStruct":159,"completionDateStruct":161,"leadSponsor":163,"locationsCount":164},{"fullName":5,"class":6},"Rocket Pharmaceuticals Inc.","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"RP-A501","EXPERIMENTAL","One planned dose of RP-A501 in cohorts of subjects with a confirmed diagnosis of Danon Disease.",[13],"Genetic: RP-A501",[15],{"type":16,"name":9,"description":17,"armGroupLabels":18,"otherNames":19},"GENETIC","RP-A501 is a gene therapy product consisting of a rAAV9 capsid containing the human LAMP2B transgene which will be administered as a single IV infusion.",[9],null,[21,25],{"name":22,"affiliation":23,"role":24},"Joseph Rossano, MD","Children's Hospital of Philadelphia","PRINCIPAL_INVESTIGATOR",{"name":26,"affiliation":27,"role":24},"Barry Greenberg, MD","University of California, San Diego",[29],{"name":30,"role":31,"phone":32,"phoneExt":19,"email":33},"Clinical Information","CONTACT","646-627-0033","clinicaltrials@rocketpharma.com",[35,52,71,83,97,111],{"facility":27,"status":36,"city":37,"state":38,"zip":39,"country":40,"countryCode":41,"cosmosGeoPoint":42,"geoPoint":47,"contacts":48},"RECRUITING","La Jolla","California","92037","United States","US",{"type":43,"coordinates":44},"Point",[45,46],-117.2742,32.84727,{"lat":46,"lon":45},[49],{"name":26,"role":31,"phone":50,"phoneExt":19,"email":51},"858-246-2987","Bgreenberg@health.ucsd.edu",{"facility":53,"status":36,"city":54,"state":55,"zip":56,"country":40,"countryCode":41,"cosmosGeoPoint":57,"geoPoint":61,"contacts":62},"Boston Children's Hospital","Boston","Massachusetts","02115",{"type":43,"coordinates":58},[59,60],-71.05977,42.35843,{"lat":60,"lon":59},[63,66,69],{"name":64,"role":31,"phone":19,"phoneExt":19,"email":65},"Emily Morris","Emily.Morris@childrens.harvard.edu",{"name":67,"role":31,"phone":19,"phoneExt":19,"email":68},"Bridget Kerwin","BridgetO_Kerwin@DFCI.HARVARD.EDU",{"name":70,"role":24,"phone":19,"phoneExt":19,"email":19},"Dominic Abrams, MD",{"facility":23,"status":36,"city":72,"state":73,"zip":74,"country":40,"countryCode":41,"cosmosGeoPoint":75,"geoPoint":79,"contacts":80},"Philadelphia","Pennsylvania","19104",{"type":43,"coordinates":76},[77,78],-75.16362,39.95238,{"lat":78,"lon":77},[81],{"name":22,"role":31,"phone":19,"phoneExt":19,"email":82},"rossanoj@chop.edu",{"facility":84,"status":36,"city":85,"state":19,"zip":19,"country":86,"countryCode":87,"cosmosGeoPoint":88,"geoPoint":92,"contacts":93},"German Heart Center Munich","Munich","Germany","DE",{"type":43,"coordinates":89},[90,91],11.57549,48.13743,{"lat":91,"lon":90},[94],{"name":95,"role":31,"phone":19,"phoneExt":19,"email":96},"Cordula Wolf, MD","wolf@dhm.mhn.de",{"facility":98,"status":36,"city":99,"state":19,"zip":19,"country":100,"countryCode":101,"cosmosGeoPoint":102,"geoPoint":106,"contacts":107},"Meyer Childrens Hospital","Florence","Italy","IT",{"type":43,"coordinates":103},[104,105],11.24626,43.77925,{"lat":105,"lon":104},[108],{"name":109,"role":31,"phone":19,"phoneExt":19,"email":110},"Iacopo Olivotto, MD","iacopo.olivotto@unifi.it",{"facility":112,"status":36,"city":113,"state":19,"zip":19,"country":114,"countryCode":115,"cosmosGeoPoint":116,"geoPoint":120,"contacts":121},"Great Ormund Street Hospital & UCL Institute of Cardiovascular Science","London","United Kingdom","UK",{"type":43,"coordinates":117},[118,119],-0.12574,51.50853,{"lat":119,"lon":118},[122],{"name":123,"role":31,"phone":19,"phoneExt":19,"email":124},"Juan Kaski, MD","Juan.Kaski@gosh.nhs.uk",{"type":126,"investigatorFullName":19,"investigatorTitle":19,"investigatorAffiliation":19,"oldNameTitle":19,"oldOrganization":19},"SPONSOR","100523162","phase-2-a-gene-therapy-study-of-rp-a501-in-male-patients-with-danon-disease-100523162",false,"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","8 Years",{"count":137,"type":138},14,"ESTIMATED","INTERVENTIONAL",[141],"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.",[144],"Danon Disease",[146,147,148,149,150,151,152,153,154],"Hypertrophic Cardiomyopathy","HCM","X-linked disease","LAMP2","Pediatric","Skeletal Myopathies","Cardiomyopathy","Lysosomal Storage Disorder","Left Ventricular Hypertrophy","2026-04-28",{"date":157,"type":158},"2026-05-04","ACTUAL",{"date":160,"type":158},"2023-09-05",{"date":162,"type":138},"2032-04",{"name":5,"class":6},6]