[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100585584":3},{"organization":4,"armGroups":7,"interventions":14,"overallOfficials":23,"centralContacts":24,"locations":30,"responsibleParty":151,"collaborators":23,"id":153,"slug":154,"hasResults":155,"nctId":156,"briefTitle":157,"officialTitle":158,"acronym":23,"eligibilityCriteria":159,"healthyVolunteers":155,"sex":160,"minAge":161,"maxAge":162,"enrollmentInfo":163,"targetDuration":23,"studyType":166,"phases":167,"briefSummary":169,"conditions":170,"keywords":172,"overallStatus":33,"whyStopped":23,"lastUpdateSubmitDate":175,"lastUpdatePostDateStruct":176,"startDateStruct":179,"completionDateStruct":181,"leadSponsor":183,"locationsCount":184},{"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",[31,45,55,65,75,86,96,108,120,131,142],{"facility":32,"status":33,"city":34,"state":35,"zip":36,"country":37,"countryCode":38,"cosmosGeoPoint":39,"geoPoint":44,"contacts":23},"Emory Genetics","RECRUITING","Atlanta","Georgia","30322","United States","US",{"type":40,"coordinates":41},"Point",[42,43],-84.38798,33.749,{"lat":43,"lon":42},{"facility":46,"status":33,"city":47,"state":48,"zip":49,"country":37,"countryCode":38,"cosmosGeoPoint":50,"geoPoint":54,"contacts":23},"University of Minnesota Masonic Children's Hospital","Minneapolis","Minnesota","55455",{"type":40,"coordinates":51},[52,53],-93.26384,44.97997,{"lat":53,"lon":52},{"facility":56,"status":33,"city":57,"state":58,"zip":59,"country":37,"countryCode":38,"cosmosGeoPoint":60,"geoPoint":64,"contacts":23},"Atrium Health Levine Children's Hospital","Charlotte","North Carolina","28203",{"type":40,"coordinates":61},[62,63],-80.84313,35.22709,{"lat":63,"lon":62},{"facility":66,"status":33,"city":67,"state":68,"zip":69,"country":37,"countryCode":38,"cosmosGeoPoint":70,"geoPoint":74,"contacts":23},"Cincinnati Children's Hospital Medical Center","Cincinnati","Ohio","45229",{"type":40,"coordinates":71},[72,73],-84.51439,39.12711,{"lat":73,"lon":72},{"facility":76,"status":77,"city":78,"state":79,"zip":80,"country":37,"countryCode":38,"cosmosGeoPoint":81,"geoPoint":85,"contacts":23},"UPMC Children's Hospital of Pittsburgh","NOT_YET_RECRUITING","Pittsburgh","Pennsylvania","15224",{"type":40,"coordinates":82},[83,84],-79.99589,40.44062,{"lat":84,"lon":83},{"facility":87,"status":33,"city":88,"state":89,"zip":90,"country":37,"countryCode":38,"cosmosGeoPoint":91,"geoPoint":95,"contacts":23},"Lysosomal and Rare Disorders Research and Treatment Center, Inc.","Fairfax","Virginia","22030",{"type":40,"coordinates":92},[93,94],-77.30637,38.84622,{"lat":94,"lon":93},{"facility":97,"status":33,"city":98,"state":99,"zip":100,"country":101,"countryCode":102,"cosmosGeoPoint":103,"geoPoint":107,"contacts":23},"Universitair Ziekenhuis (UZ) Leuven","Leuven","Vlaams-Brabant","3000","Belgium","BE",{"type":40,"coordinates":104},[105,106],4.70093,50.87959,{"lat":106,"lon":105},{"facility":109,"status":33,"city":110,"state":111,"zip":112,"country":113,"countryCode":114,"cosmosGeoPoint":115,"geoPoint":119,"contacts":23},"Universitäetsklinikum Müenster (UKM) Klinik für Kinder- und Jugendmedizin - Allgemeine Paediatrie","Münster","North Rhine-Westphalia","48149","Germany","DE",{"type":40,"coordinates":116},[117,118],7.62571,51.96236,{"lat":118,"lon":117},{"facility":121,"status":33,"city":122,"state":122,"zip":123,"country":124,"countryCode":125,"cosmosGeoPoint":126,"geoPoint":130,"contacts":23},"Hospital Universitario de la Paz","Madrid","28046","Spain","ES",{"type":40,"coordinates":127},[128,129],-3.70256,40.4165,{"lat":129,"lon":128},{"facility":132,"status":77,"city":133,"state":23,"zip":134,"country":135,"countryCode":136,"cosmosGeoPoint":137,"geoPoint":141,"contacts":23},"Great Ormond Street Hospital for Children NHS Foundation Trust","London","WC1N 3JH","United Kingdom","UK",{"type":40,"coordinates":138},[139,140],-0.12574,51.50853,{"lat":140,"lon":139},{"facility":143,"status":33,"city":144,"state":23,"zip":145,"country":135,"countryCode":136,"cosmosGeoPoint":146,"geoPoint":150,"contacts":23},"Manchester University NHS Foundation Trust","Manchester","M13 9WL",{"type":40,"coordinates":147},[148,149],-2.23743,53.48095,{"lat":149,"lon":148},{"type":152,"investigatorFullName":23,"investigatorTitle":23,"investigatorAffiliation":23,"oldNameTitle":23,"oldOrganization":23},"SPONSOR","100585584","phase-3-a-study-of-migalastat-in-pediatric-subjects-2-to-12-yrs-with-fabry-disease-and-amenable-gla-variants-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":164,"type":165},8,"ESTIMATED","INTERVENTIONAL",[168],"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.",[171],"Fabry Disease",[173,21,22,174],"migalastat","lysosomal disease","2026-06-23",{"date":177,"type":178},"2026-06-25","ACTUAL",{"date":180,"type":178},"2026-01-08",{"date":182,"type":165},"2028-12",{"name":5,"class":6},11]