[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100387687":3},{"organization":4,"armGroups":7,"interventions":8,"overallOfficials":7,"centralContacts":16,"locations":24,"responsibleParty":70,"collaborators":7,"id":72,"slug":73,"hasResults":74,"nctId":75,"briefTitle":76,"officialTitle":77,"acronym":7,"eligibilityCriteria":78,"healthyVolunteers":74,"sex":79,"minAge":80,"maxAge":81,"enrollmentInfo":7,"targetDuration":7,"studyType":82,"phases":7,"briefSummary":83,"conditions":84,"keywords":86,"overallStatus":27,"whyStopped":7,"lastUpdateSubmitDate":89,"lastUpdatePostDateStruct":90,"startDateStruct":7,"completionDateStruct":7,"leadSponsor":93,"locationsCount":94},{"fullName":5,"class":6},"Amicus Therapeutics","INDUSTRY",null,[9,13],{"type":10,"name":11,"description":12,"armGroupLabels":7,"otherNames":7},"BIOLOGICAL","ATB200","Participants received ATB200 co-administered with AT2221 capsule (Miglustat)",{"type":14,"name":15,"description":12,"armGroupLabels":7,"otherNames":7},"DRUG","AT2221",[17,22],{"name":18,"role":19,"phone":20,"phoneExt":7,"email":21},"For Site","CONTACT","866-9AMICUS","patientadvocacy@amicusrx.com",{"name":23,"role":19,"phone":20,"phoneExt":7,"email":21},"For Patient",[25,39,49,59],{"facility":26,"status":27,"city":28,"state":29,"zip":30,"country":31,"countryCode":32,"cosmosGeoPoint":33,"geoPoint":38,"contacts":7},"UCSF Benioff Children's Hospital Oakland","AVAILABLE","Oakland","California","94609","United States","US",{"type":34,"coordinates":35},"Point",[36,37],-122.2708,37.80437,{"lat":37,"lon":36},{"facility":40,"status":27,"city":41,"state":42,"zip":43,"country":31,"countryCode":32,"cosmosGeoPoint":44,"geoPoint":48,"contacts":7},"UF Health Shands Hospital","Gainesville","Florida","32610",{"type":34,"coordinates":45},[46,47],-82.32483,29.65163,{"lat":47,"lon":46},{"facility":50,"status":27,"city":51,"state":7,"zip":7,"country":52,"countryCode":53,"cosmosGeoPoint":54,"geoPoint":58,"contacts":7},"University Hospital of Padova","Padova","Italy","IT",{"type":34,"coordinates":55},[56,57],11.14261,44.38225,{"lat":57,"lon":56},{"facility":60,"status":27,"city":61,"state":7,"zip":62,"country":63,"countryCode":64,"cosmosGeoPoint":65,"geoPoint":69,"contacts":7},"National Taiwan University Hospital","Taipei","10002","Taiwan","TW",{"type":34,"coordinates":66},[67,68],121.52639,25.05306,{"lat":68,"lon":67},{"type":71,"investigatorFullName":7,"investigatorTitle":7,"investigatorAffiliation":7,"oldNameTitle":7,"oldOrganization":7},"SPONSOR","100387687","expanded-access-for-atb200at2221-for-the-treatment-of-iopd-100387687",false,"NCT04327973","Expanded Access for ATB200\u002FAT2221 for the Treatment of IOPD","Expanded Use Of Recombinant Human Acid Alpha-Glucosidase\u002FN-butyl-deoxynojirimycin (ATB200\u002FAT2221) For Patients With Infantile-Onset Pompe Disease","Inclusion Criteria:\n\n1. Patient is male or female and 0 to \\\u003C 18 years old.\n2. Patient has a diagnosis of Pompe disease, based on documentation of GAA genotyping demonstrating variants associated with IOPD.\n3. Patient does not currently qualify for or is unable to enroll (eg, due to location, etc) in any commercial drug-sponsored ongoing clinical trial.\n4. Patient is currently being treated with an approved ERT and is declining (eg, worsening in motor function, respiratory function\u002Fparameters, cardiac function, etc) based on treating physician's clinical judgement.\n5. The patient's legal guardian or representative has given informed consent (and assent when age appropriate for regional requirements) to treatment prior to administering ATB200\u002FAT2221 in a manner consistent with all national requirements.\n6. If of reproductive potential and sexually active, female subjects agree to use a highly effective method of contraception throughout the duration of the treatment and for up to 90 days after their last dose of ATB200\u002FAT2221.\n\nExclusion Criteria:\n\n1. Patient has a hypersensitivity to any of the excipients in ATB200, alglucosidase alfa, or AT2221\n2. Patient has a medical or any other extenuating condition or circumstance that may, in the opinion of the investigator and\u002For Amicus, pose an undue safety risk to the patient.\n3. Concomitant use of miglitol (eg, Glyset), non-AT2221 form of miglustat (eg, Zavesca), acarbose (eg, Precose or Glucobay), voglibose (eg, Volix, Vocarb,or Volibo)","ALL","0 Years","17 Years","EXPANDED_ACCESS","This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200\u002FAT2221.",[85],"Pompe Disease Infantile-Onset",[87,88],"expanded access","compassionate use","2025-09-09",{"date":91,"type":92},"2025-09-10","ACTUAL",{"name":5,"class":6},4]