[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100585584":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":23,"centralContacts":24,"locations":30,"responsibleParty":146,"collaborators":23,"id":148,"slug":23,"hasResults":149,"nctId":150,"briefTitle":151,"officialTitle":152,"acronym":23,"eligibilityCriteria":153,"healthyVolunteers":149,"sex":154,"minAge":155,"maxAge":156,"enrollmentInfo":157,"targetDuration":23,"studyType":160,"phases":161,"briefSummary":163,"conditions":164,"keywords":166,"overallStatus":33,"whyStopped":23,"lastUpdateSubmitDate":169,"lastUpdatePostDateStruct":170,"startDateStruct":173,"completionDateStruct":175,"leadSponsor":177,"locationsCount":178},{"fullName":5,"class":6},"Amicus Therapeutics","INDUSTRY",[8],{"label":9,"type":10,"description":11,"interventionNames":12},"Migalastat HCl 20 mg Dispersible Tablets","EXPERIMENTAL","Migalastat will be administered every other day (QOD). The initial dose will be based on body weight at baseline.",[13],"Drug: Migalastat HCl 20 mg",[15],{"type":16,"name":17,"description":18,"armGroupLabels":19,"otherNames":20},"DRUG","Migalastat HCl 20 mg","Migalastat will be supplied as 20-mg dispersible tablets. Migalastat 20-mg dispersible tablets contain 16 mg migalastat free base.",[9],[21,22],"AT1001","Galafold",null,[25],{"name":26,"role":27,"phone":28,"phoneExt":23,"email":29},"Amicus Therapeutics Patient Advocacy","CONTACT","609-662-2000","patientadvocacy@amicusrx.com",[31,44,54,64,74,85,95,106,117,127,137],{"facility":32,"status":33,"city":34,"state":35,"zip":36,"country":37,"cosmosGeoPoint":38,"geoPoint":43,"contacts":23},"Emory Genetics","RECRUITING","Atlanta","Georgia","30322","United States",{"type":39,"coordinates":40},"Point",[41,42],-84.38798,33.749,{"lat":42,"lon":41},{"facility":45,"status":33,"city":46,"state":47,"zip":48,"country":37,"cosmosGeoPoint":49,"geoPoint":53,"contacts":23},"University of Minnesota Masonic Children's Hospital","Minneapolis","Minnesota","55455",{"type":39,"coordinates":50},[51,52],-93.26384,44.97997,{"lat":52,"lon":51},{"facility":55,"status":33,"city":56,"state":57,"zip":58,"country":37,"cosmosGeoPoint":59,"geoPoint":63,"contacts":23},"Atrium Health Levine Children's Hospital","Charlotte","North Carolina","28203",{"type":39,"coordinates":60},[61,62],-80.84313,35.22709,{"lat":62,"lon":61},{"facility":65,"status":33,"city":66,"state":67,"zip":68,"country":37,"cosmosGeoPoint":69,"geoPoint":73,"contacts":23},"Cincinnati Children's Hospital Medical Center","Cincinnati","Ohio","45229",{"type":39,"coordinates":70},[71,72],-84.51439,39.12711,{"lat":72,"lon":71},{"facility":75,"status":76,"city":77,"state":78,"zip":79,"country":37,"cosmosGeoPoint":80,"geoPoint":84,"contacts":23},"UPMC Children's Hospital of Pittsburgh","NOT_YET_RECRUITING","Pittsburgh","Pennsylvania","15224",{"type":39,"coordinates":81},[82,83],-79.99589,40.44062,{"lat":83,"lon":82},{"facility":86,"status":33,"city":87,"state":88,"zip":89,"country":37,"cosmosGeoPoint":90,"geoPoint":94,"contacts":23},"Lysosomal and Rare Disorders Research and Treatment Center, Inc.","Fairfax","Virginia","22030",{"type":39,"coordinates":91},[92,93],-77.30637,38.84622,{"lat":93,"lon":92},{"facility":96,"status":33,"city":97,"state":98,"zip":99,"country":100,"cosmosGeoPoint":101,"geoPoint":105,"contacts":23},"Universitair Ziekenhuis (UZ) Leuven","Leuven","Vlaams-Brabant","3000","Belgium",{"type":39,"coordinates":102},[103,104],4.70093,50.87959,{"lat":104,"lon":103},{"facility":107,"status":33,"city":108,"state":109,"zip":110,"country":111,"cosmosGeoPoint":112,"geoPoint":116,"contacts":23},"Universitäetsklinikum Müenster (UKM) Klinik für Kinder- und Jugendmedizin - Allgemeine Paediatrie","Münster","North Rhine-Westphalia","48149","Germany",{"type":39,"coordinates":113},[114,115],7.62571,51.96236,{"lat":115,"lon":114},{"facility":118,"status":33,"city":119,"state":119,"zip":120,"country":121,"cosmosGeoPoint":122,"geoPoint":126,"contacts":23},"Hospital Universitario de la Paz","Madrid","28046","Spain",{"type":39,"coordinates":123},[124,125],-3.70256,40.4165,{"lat":125,"lon":124},{"facility":128,"status":76,"city":129,"state":23,"zip":130,"country":131,"cosmosGeoPoint":132,"geoPoint":136,"contacts":23},"Great Ormond Street Hospital for Children NHS Foundation Trust","London","WC1N 3JH","United Kingdom",{"type":39,"coordinates":133},[134,135],-0.12574,51.50853,{"lat":135,"lon":134},{"facility":138,"status":33,"city":139,"state":23,"zip":140,"country":131,"cosmosGeoPoint":141,"geoPoint":145,"contacts":23},"Manchester University NHS Foundation Trust","Manchester","M13 9WL",{"type":39,"coordinates":142},[143,144],-2.23743,53.48095,{"lat":144,"lon":143},{"type":147,"investigatorFullName":23,"investigatorTitle":23,"investigatorAffiliation":23,"oldNameTitle":23,"oldOrganization":23},"SPONSOR","100585584",false,"NCT06904261","A Study of Migalastat in Pediatric Subjects (2 to \u003C12 Yrs) With Fabry Disease and Amenable GLA Variants","An Open-label Study of the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of 12 Month Treatment With Migalastat in Pediatric Subjects (Aged 2 to \u003C 12 Years) With Fabry Disease and Amenable GLA Variants","Inclusion Criteria\n\n* Male or female subjects, diagnosed with Fabry disease who are between ages 2 and \\\u003C 12 years at randomization (subjects aged 11 years must have birthdays \\> 30 days after randomization)\n* Subject's parent or legally authorized representative is willing and able to provide written informed consent and authorization for use and disclosure of personal health information or research-related health information, and subject provides assent, if applicable.\n* Subject has a GLA variant documented in his\u002Fher medical record that is amenable to migalastat prior to Visit 2.\n* Subject has not received ERT (eg, Replagal® \\[agalsidase alfa\\] or Fabrazyme® \\[agalsidase beta\\]) for at least 14 days prior to Baseline visit.\n* Subject has at least 1 documented complication (ie, historical or current laboratory abnormality or sign\u002Fsymptom) of Fabry disease\n* If of reproductive potential, both male and female subjects agree to use a medically accepted method of contraception throughout the duration of the study and for up to 30 days after their last dose of migalastat.\n\nExclusion Criteria\n\n* Has moderate or severe renal impairment (eGFR \\\u003C 60 mL\u002Fmin\u002F1.73 m2 at Visit 1 \\[screening\\]).\n* Has advanced kidney disease requiring dialysis or kidney transplantation.\n* History of allergy or sensitivity to migalastat (including excipients) or other iminosugars (eg, miglustat, miglitol).\n* Has received any investigational\u002Fexperimental drug, biologic, or device within 30 days or 5 half-lives of the investigational product (whichever is longer) before Visit 1 (screening).\n* Has received any gene therapy at any time or anticipates starting gene therapy during the study period.\n* Requires treatment with Glyset (miglitol) or Zavesca (miglustat), within 6 months before Visit 1(screening) or throughout the study.\n* Has any intercurrent illness or condition at Visit 1 (screening) or Visit 2 (baseline) that may preclude the subject from fulfilling the protocol requirements or suggests to the investigator that the potential subject may have an unacceptable risk by participating in this study.\n* Pregnant or breastfeeding\n* Otherwise unsuitable for the study in the opinion of the investigator","ALL","2 Years","11 Years",{"count":158,"type":159},8,"ESTIMATED","INTERVENTIONAL",[162],"PHASE3","An open-label study to evaluate the safety, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of migalastat treatment in pediatric subjects 2 to \\\u003C 12 years of age with Fabry disease and with amenable GLA variants.",[165],"Fabry Disease",[167,21,22,168],"migalastat","lysosomal disease","2026-06-23",{"date":171,"type":172},"2026-06-25","ACTUAL",{"date":174,"type":172},"2026-01-08",{"date":176,"type":159},"2028-12",{"name":5,"class":6},11]