[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100532569":3},{"organization":4,"armGroups":7,"interventions":16,"overallOfficials":10,"centralContacts":21,"locations":27,"responsibleParty":270,"collaborators":10,"id":272,"slug":273,"hasResults":274,"nctId":275,"briefTitle":276,"officialTitle":277,"acronym":10,"eligibilityCriteria":278,"healthyVolunteers":274,"sex":279,"minAge":280,"maxAge":10,"enrollmentInfo":281,"targetDuration":10,"studyType":284,"phases":10,"briefSummary":285,"conditions":286,"keywords":288,"overallStatus":30,"whyStopped":10,"lastUpdateSubmitDate":293,"lastUpdatePostDateStruct":294,"startDateStruct":297,"completionDateStruct":299,"leadSponsor":301,"locationsCount":302},{"fullName":5,"class":6},"Rocket Pharmaceuticals Inc.","INDUSTRY",[8,13],{"label":9,"type":10,"description":10,"interventionNames":11},"Prospective Cohort",null,[12],"Other: No intervention",{"label":14,"type":10,"description":10,"interventionNames":15},"Retrospective Cohort",[12],[17],{"type":18,"name":19,"description":19,"armGroupLabels":20,"otherNames":10},"OTHER","No intervention",[9,14],[22],{"name":23,"role":24,"phone":25,"phoneExt":10,"email":26},"Clinical Information","CONTACT","646-627-0033","clinicaltrials@rocketpharma.com",[28,53,70,87,103,122,142,162,182,198,216,234,251],{"facility":29,"status":30,"city":31,"state":32,"zip":33,"country":34,"countryCode":35,"cosmosGeoPoint":36,"geoPoint":41,"contacts":42},"Phoenix Children's Hospital","RECRUITING","Phoenix","Arizona","85016","United States","US",{"type":37,"coordinates":38},"Point",[39,40],-112.07404,33.44838,{"lat":40,"lon":39},[43,47,51],{"name":44,"role":24,"phone":45,"phoneExt":10,"email":46},"Andrew Papez, MD","(602) 546-0512","apapez@phoenixchildrens.com",{"name":48,"role":24,"phone":49,"phoneExt":10,"email":50},"Sanjana Khanna","6029334482","skhanna1@phoenixchildrens.com",{"name":44,"role":52,"phone":10,"phoneExt":10,"email":10},"PRINCIPAL_INVESTIGATOR",{"facility":54,"status":30,"city":55,"state":56,"zip":57,"country":34,"countryCode":35,"cosmosGeoPoint":58,"geoPoint":62,"contacts":63},"University of California, San Diego","La Jolla","California","92037",{"type":37,"coordinates":59},[60,61],-117.2742,32.84727,{"lat":61,"lon":60},[64,68],{"name":65,"role":24,"phone":66,"phoneExt":10,"email":67},"Kim Hong, MD","(858) 246-2996","knhong@health.ucsd.edu",{"name":69,"role":52,"phone":10,"phoneExt":10,"email":10},"Kimberly Hong, MD",{"facility":71,"status":30,"city":72,"state":73,"zip":74,"country":34,"countryCode":35,"cosmosGeoPoint":75,"geoPoint":79,"contacts":80},"Children's Hospital Colorado","Aurora","Colorado","80045",{"type":37,"coordinates":76},[77,78],-104.83192,39.72943,{"lat":78,"lon":77},[81,85],{"name":82,"role":24,"phone":83,"phoneExt":10,"email":84},"Matthew Taylor, MD, PhD","(720) 848-1030","matthew.taylor@uchsc.edu",{"name":86,"role":52,"phone":10,"phoneExt":10,"email":10},"Matthew Taylor, MD",{"facility":88,"status":30,"city":89,"state":90,"zip":91,"country":34,"countryCode":35,"cosmosGeoPoint":92,"geoPoint":96,"contacts":97},"University of Florida College of Medicine","Gainesville","Florida","32610",{"type":37,"coordinates":93},[94,95],-82.32483,29.65163,{"lat":95,"lon":94},[98,102],{"name":99,"role":24,"phone":100,"phoneExt":10,"email":101},"Mohammed M Ahmed, MD","(352) 273-7770","Mustafa.Ahmed@medicine.ufl.edu",{"name":99,"role":52,"phone":10,"phoneExt":10,"email":10},{"facility":104,"status":30,"city":105,"state":106,"zip":107,"country":34,"countryCode":35,"cosmosGeoPoint":108,"geoPoint":112,"contacts":113},"University of Michigan","Ann Arbor","Michigan","48109",{"type":37,"coordinates":109},[110,111],-83.74088,42.27756,{"lat":111,"lon":110},[114,117,119],{"name":115,"role":24,"phone":10,"phoneExt":10,"email":116},"Supriya Shore","shores@med.umich.edu",{"name":118,"role":52,"phone":10,"phoneExt":10,"email":10},"Supriya Shore, MD",{"name":120,"role":121,"phone":10,"phoneExt":10,"email":10},"Josh Meisner, MD","SUB_INVESTIGATOR",{"facility":123,"status":30,"city":124,"state":125,"zip":126,"country":34,"countryCode":35,"cosmosGeoPoint":127,"geoPoint":131,"contacts":132},"Washington University","St Louis","Missouri","63110",{"type":37,"coordinates":128},[129,130],-90.19789,38.62727,{"lat":130,"lon":129},[133,137,141],{"name":134,"role":24,"phone":135,"phoneExt":10,"email":136},"Charles Canter, MD","314-286-2404","canter@wustl.edu",{"name":138,"role":24,"phone":139,"phoneExt":10,"email":140},"Teresa Roberson","314-302-7209","t.roberson@wustl.edu",{"name":134,"role":52,"phone":10,"phoneExt":10,"email":10},{"facility":143,"status":30,"city":144,"state":144,"zip":145,"country":34,"countryCode":35,"cosmosGeoPoint":146,"geoPoint":150,"contacts":151},"Columbia University Medical Center\u002FNew York Presbyterian","New York","10032",{"type":37,"coordinates":147},[148,149],-74.00597,40.71427,{"lat":149,"lon":148},[152,156,160],{"name":153,"role":24,"phone":154,"phoneExt":10,"email":155},"Farhana Latif","(212) 305-3172","fl2203@cumc.columbia.edu",{"name":157,"role":24,"phone":10,"phoneExt":158,"email":159},"Jessica Idumonyi","212) 305-1429","joi2102@cumc.columbia.edu",{"name":161,"role":52,"phone":10,"phoneExt":10,"email":10},"Farhana Latif, MD",{"facility":163,"status":30,"city":164,"state":165,"zip":166,"country":34,"countryCode":35,"cosmosGeoPoint":167,"geoPoint":171,"contacts":172},"Texas Children's Hospital","Houston","Texas","77030",{"type":37,"coordinates":168},[169,170],-95.36327,29.76328,{"lat":170,"lon":169},[173,177,181],{"name":174,"role":24,"phone":175,"phoneExt":10,"email":176},"Kyle Hope, MD","(832) 822-4280","kyle.hope@bcm.edu",{"name":178,"role":24,"phone":179,"phoneExt":10,"email":180},"David Garuba","8328265622","odgaruba@texaschildrens.org",{"name":174,"role":52,"phone":10,"phoneExt":10,"email":10},{"facility":183,"status":30,"city":184,"state":10,"zip":10,"country":185,"countryCode":186,"cosmosGeoPoint":187,"geoPoint":191,"contacts":192},"German Heart Center Munich","München","Germany","DE",{"type":37,"coordinates":188},[189,190],13.31243,51.60698,{"lat":190,"lon":189},[193,197],{"name":194,"role":24,"phone":195,"phoneExt":10,"email":196},"Cordula Wolf, MD","+498912182877","wolf@dhm.mhn.de",{"name":194,"role":52,"phone":10,"phoneExt":10,"email":10},{"facility":199,"status":30,"city":200,"state":10,"zip":201,"country":202,"countryCode":203,"cosmosGeoPoint":204,"geoPoint":208,"contacts":209},"Sheba Medical Center","Ramat Gan","5265601","Israel","IL",{"type":37,"coordinates":205},[206,207],34.81065,32.08227,{"lat":207,"lon":206},[210,214],{"name":211,"role":24,"phone":212,"phoneExt":10,"email":213},"Yair Anikster, PhD","+97235305018","yair.anikster@sheba.health.gov.il",{"name":215,"role":52,"phone":10,"phoneExt":10,"email":10},"Yair Anikster, MD",{"facility":217,"status":30,"city":218,"state":10,"zip":219,"country":220,"countryCode":221,"cosmosGeoPoint":222,"geoPoint":226,"contacts":227},"Azienda Ospedaliera Universitaria Meyer IRCCS","Florence","50139","Italy","IT",{"type":37,"coordinates":223},[224,225],11.24626,43.77925,{"lat":225,"lon":224},[228,232],{"name":229,"role":24,"phone":230,"phoneExt":10,"email":231},"Iacopo Olivotto, PhD","+39557945138","Iacopo.olivotto@unifi.it",{"name":233,"role":52,"phone":10,"phoneExt":10,"email":10},"Iacopo Olivotto, MD",{"facility":235,"status":30,"city":236,"state":237,"zip":10,"country":238,"countryCode":239,"cosmosGeoPoint":240,"geoPoint":244,"contacts":245},"Hospital Universitario Puerta de Hierro Majadahonda","Majadahonda","Madrid","Spain","ES",{"type":37,"coordinates":241},[242,243],-3.87182,40.47353,{"lat":243,"lon":242},[246,249],{"name":247,"role":24,"phone":10,"phoneExt":10,"email":248},"Pavia Garcia","pablogpavia@yahoo.es",{"name":250,"role":52,"phone":10,"phoneExt":10,"email":10},"Pavia Garcia, MD",{"facility":252,"status":30,"city":253,"state":10,"zip":254,"country":255,"countryCode":256,"cosmosGeoPoint":257,"geoPoint":261,"contacts":262},"University College London - Great Ormond Street Institute of Child Health","London","WC1N 1DZ","United Kingdom","UK",{"type":37,"coordinates":258},[259,260],-0.12574,51.50853,{"lat":260,"lon":259},[263,267,268],{"name":264,"role":24,"phone":265,"phoneExt":10,"email":266},"Juan Kaski, MD","+4402074059200","j.kaski@ucl.ac.uk",{"name":264,"role":52,"phone":10,"phoneExt":10,"email":10},{"name":269,"role":121,"phone":10,"phoneExt":10,"email":10},"Zhia Lim, MD",{"type":271,"investigatorFullName":10,"investigatorTitle":10,"investigatorAffiliation":10,"oldNameTitle":10,"oldOrganization":10},"SPONSOR","100532569","danon-disease-natural-history-study-100532569",false,"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","ALL","8 Years",{"count":282,"type":283},60,"ESTIMATED","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).",[287],"Danon Disease",[287,289,290,291,292],"LAMP2","Lysosome-associated membrane protein 2","Cardiomyopathy","X-linked","2026-04-29",{"date":295,"type":296},"2026-05-05","ACTUAL",{"date":298,"type":296},"2023-12-20",{"date":300,"type":283},"2030-03",{"name":5,"class":6},13]