[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100183587":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":19,"centralContacts":23,"locations":33,"responsibleParty":52,"collaborators":54,"id":58,"slug":59,"hasResults":60,"nctId":61,"briefTitle":62,"officialTitle":63,"acronym":10,"eligibilityCriteria":64,"healthyVolunteers":60,"sex":65,"minAge":10,"maxAge":10,"enrollmentInfo":66,"targetDuration":10,"studyType":69,"phases":10,"briefSummary":70,"conditions":71,"keywords":73,"overallStatus":36,"whyStopped":10,"lastUpdateSubmitDate":86,"lastUpdatePostDateStruct":87,"startDateStruct":90,"completionDateStruct":92,"leadSponsor":94,"locationsCount":95},{"fullName":5,"class":6},"Duke University","OTHER",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Infantile Pompe disease",null,"Individuals with a confirmed diagnosis of Infantile Pompe disease",[13],"Other: Observational",[15],{"type":6,"name":16,"description":17,"armGroupLabels":18,"otherNames":10},"Observational","This is a longitudinal study focused on the emerging natural history of Infantile Pompe disease, response to ERT using alglucosidase alfa (Myozyme) and response to Immune Tolerance Induction (ITI).",[9],[20],{"name":21,"affiliation":5,"role":22},"Priya S Kishnani, MD","PRINCIPAL_INVESTIGATOR",[24,29],{"name":25,"role":26,"phone":27,"phoneExt":10,"email":28},"Ankit K Desai, MBBS","CONTACT","919-613-6310","ankit.desai@duke.edu",{"name":30,"role":26,"phone":31,"phoneExt":10,"email":32},"Eleanor Rodriguez-Rassi, MPH","919-613-1219","eleanor.rodriguezrassi@duke.edu",[34],{"facility":35,"status":36,"city":37,"state":38,"zip":39,"country":40,"countryCode":41,"cosmosGeoPoint":42,"geoPoint":47,"contacts":48},"Duke University Medical Center","RECRUITING","Durham","North Carolina","27710","United States","US",{"type":43,"coordinates":44},"Point",[45,46],-78.89862,35.99403,{"lat":46,"lon":45},[49,50,51],{"name":25,"role":26,"phone":27,"phoneExt":10,"email":28},{"name":30,"role":26,"phone":31,"phoneExt":10,"email":32},{"name":21,"role":22,"phone":10,"phoneExt":10,"email":10},{"type":53,"investigatorFullName":10,"investigatorTitle":10,"investigatorAffiliation":10,"oldNameTitle":10,"oldOrganization":10},"SPONSOR",[55],{"name":56,"class":57},"Genzyme, a Sanofi Company","INDUSTRY","100183587","determination-of-crim-status-and-longitudinal-follow-up-of-individuals-with-pompe-disease-100183587",false,"NCT01665326","Determination of CRIM Status and Longitudinal Follow-up of Individuals With Pompe Disease","Determination of Cross-Reactive Immunological Material (CRIM) Status and Longitudinal Follow-up of Individuals With Pompe Disease","Inclusion Criteria:\n\n* Confirmed diagnosis of infantile, atypical or juvenile onset Pompe disease\n* Must provide a written informed consent","ALL",{"count":67,"type":68},400,"ESTIMATED","OBSERVATIONAL","This is a longitudinal natural history study of Infantile Pompe disease. The investigators will regularly collect and review medical information regarding the diagnosis of Pompe disease, response to enzyme replacement (ERT) using alglucosidase alfa (Lumizyme\u002FMyozyme) and response to immunosuppressive therapy in cases at risk for developing or those who have developed high and sustained antibodies to ERT. To follow the long-term outcomes, we will collect medical records including but not limited to the diagnosis, clinical parameters, assessments for clinical monitoring, and laboratory values including antibody testing results.",[72],"Pompe Disease",[74,75,76,77,78,79,80,81,82,83,84,85],"Pompe disease","Glycogen Storage Disease Type II","Acid Maltase Deficiency","CRIM Status","Acid Alpha-Glucosidase Deficiency","Alglucosidase alfa","Myozyme","Enzyme replacement therapy","Immune Tolerance Induction","Lumizyme","Immunomodulation","Anti-drug antibodies","2026-04-06",{"date":88,"type":89},"2026-04-08","ACTUAL",{"date":91,"type":10},"2009-09",{"date":93,"type":68},"2029-03",{"name":5,"class":6},1]