[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100637093":3},{"organization":4,"outcomesModule":7,"designInfo":14,"detailedDescription":17,"studyPopulation":18,"armGroups":13,"interventions":13,"overallOfficials":19,"centralContacts":24,"locations":29,"responsibleParty":180,"collaborators":13,"id":182,"slug":183,"hasResults":184,"nctId":185,"briefTitle":186,"officialTitle":187,"acronym":13,"eligibilityCriteria":188,"healthyVolunteers":184,"sex":189,"minAge":13,"maxAge":13,"enrollmentInfo":190,"targetDuration":13,"studyType":193,"phases":13,"briefSummary":194,"conditions":195,"keywords":197,"overallStatus":32,"whyStopped":13,"lastUpdateSubmitDate":208,"lastUpdatePostDateStruct":209,"startDateStruct":212,"completionDateStruct":214,"leadSponsor":216,"locationsCount":217},{"fullName":5,"class":6},"JCR Pharmaceuticals Co., Ltd.","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":13,"otherOutcomes":13},[9],{"measure":10,"description":11,"timeFrame":12},"Evaluate the course of disease progression in individuals with fucosidosis who were\u002Fare untreated with any investigational products","These observations are intended to inform possible future treatment studies.","Baseline to up to 4 years",null,{"allocation":13,"interventionModel":13,"interventionModelDescription":13,"primaryPurpose":13,"observationalModel":15,"timePerspective":16,"maskingInfo":13},"COHORT","OTHER","Time Perspective: Both retrospective (Part A) and prospective (Part B).\n\nEnrollment: Part A is anticipated to enroll up to 57 participants for retrospective data collection. Part B is anticipated to enroll up to 31 participants for prospective data collection, the majority of whom are expected to be participants also enrolled in Part A.","Individuals of any age diagnosed with fucosidosis. Part A: living and deceased participants. Part B: living participants.",[20],{"name":21,"affiliation":22,"role":23},"Karolina M Stepien, MD","Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust","PRINCIPAL_INVESTIGATOR",[25],{"name":5,"role":26,"phone":27,"phoneExt":13,"email":28},"CONTACT","+81 797 328582","clinical_development@jp.jcrpharm.com",[30,44,55,66,77,88,93,103,112,118,127,133,144,153,162,171],{"facility":31,"status":32,"city":33,"state":34,"zip":35,"country":36,"countryCode":37,"cosmosGeoPoint":38,"geoPoint":43,"contacts":13},"Children's Hospital of Orange County","RECRUITING","Orange","California","92868","United States","US",{"type":39,"coordinates":40},"Point",[41,42],-117.85311,33.78779,{"lat":42,"lon":41},{"facility":45,"status":46,"city":47,"state":48,"zip":49,"country":36,"countryCode":37,"cosmosGeoPoint":50,"geoPoint":54,"contacts":13},"University of Minnesota Health","NOT_YET_RECRUITING","Minneapolis","Minnesota","55455",{"type":39,"coordinates":51},[52,53],-93.26384,44.97997,{"lat":53,"lon":52},{"facility":56,"status":46,"city":57,"state":13,"zip":58,"country":59,"countryCode":60,"cosmosGeoPoint":61,"geoPoint":65,"contacts":13},"Indira Gandhi Institute of Child Health, Department of Pediatric Neurology","Bengaluru","560029","India","IN",{"type":39,"coordinates":62},[63,64],77.59369,12.97194,{"lat":64,"lon":63},{"facility":67,"status":46,"city":68,"state":13,"zip":69,"country":70,"countryCode":71,"cosmosGeoPoint":72,"geoPoint":76,"contacts":13},"Erasmus University Medical Center","Rotterdam","3015 AA","Netherlands","NL",{"type":39,"coordinates":73},[74,75],4.47917,51.9225,{"lat":75,"lon":74},{"facility":78,"status":46,"city":79,"state":13,"zip":80,"country":81,"countryCode":82,"cosmosGeoPoint":83,"geoPoint":87,"contacts":13},"La Rabta Hospital","Tunis","1007","Tunisia","TN",{"type":39,"coordinates":84},[85,86],10.16579,36.81897,{"lat":86,"lon":85},{"facility":89,"status":46,"city":79,"state":13,"zip":80,"country":81,"countryCode":82,"cosmosGeoPoint":90,"geoPoint":92,"contacts":13},"National Institute Mongi-Ben Hamida of Neurology of Tunis",{"type":39,"coordinates":91},[85,86],{"lat":86,"lon":85},{"facility":94,"status":46,"city":95,"state":13,"zip":96,"country":97,"countryCode":13,"cosmosGeoPoint":98,"geoPoint":102,"contacts":13},"Cukurova University, Faculty of Medicine","Adana","01339","Turkey (Türkiye)",{"type":39,"coordinates":99},[100,101],35.32531,36.98615,{"lat":101,"lon":100},{"facility":104,"status":46,"city":105,"state":13,"zip":106,"country":97,"countryCode":13,"cosmosGeoPoint":107,"geoPoint":111,"contacts":13},"Istanbul University Cerrahpasa Medical Faculty Hospital","Istanbul","34098",{"type":39,"coordinates":108},[109,110],28.94966,41.01384,{"lat":110,"lon":109},{"facility":113,"status":46,"city":105,"state":13,"zip":114,"country":97,"countryCode":13,"cosmosGeoPoint":115,"geoPoint":117,"contacts":13},"Marmara University Pendik Training and Research Hospital","34899",{"type":39,"coordinates":116},[109,110],{"lat":110,"lon":109},{"facility":119,"status":46,"city":120,"state":13,"zip":121,"country":97,"countryCode":13,"cosmosGeoPoint":122,"geoPoint":126,"contacts":13},"Ege University, Faculty of Medicine","Izmir","35100",{"type":39,"coordinates":123},[124,125],27.13838,38.41273,{"lat":125,"lon":124},{"facility":128,"status":46,"city":120,"state":13,"zip":129,"country":97,"countryCode":13,"cosmosGeoPoint":130,"geoPoint":132,"contacts":13},"Dokuz Eylül University Hospital","35330",{"type":39,"coordinates":131},[124,125],{"lat":125,"lon":124},{"facility":134,"status":46,"city":135,"state":13,"zip":136,"country":137,"countryCode":138,"cosmosGeoPoint":139,"geoPoint":143,"contacts":13},"Royal Belfast Hospital for Sick Children","Belfast","BT12 6BA","United Kingdom","UK",{"type":39,"coordinates":140},[141,142],-5.92541,54.59682,{"lat":142,"lon":141},{"facility":145,"status":46,"city":146,"state":13,"zip":147,"country":137,"countryCode":138,"cosmosGeoPoint":148,"geoPoint":152,"contacts":13},"Birmingham Women's and Children's NHS Foundation Trust","Birmingham","B4 6NH",{"type":39,"coordinates":149},[150,151],-1.89983,52.48142,{"lat":151,"lon":150},{"facility":154,"status":46,"city":155,"state":13,"zip":156,"country":137,"countryCode":138,"cosmosGeoPoint":157,"geoPoint":161,"contacts":13},"National Institute for Health and Care Research Clinical Research Facility, Great Ormond Street Hospital","London","WC1N 3JH",{"type":39,"coordinates":158},[159,160],-0.12574,51.50853,{"lat":160,"lon":159},{"facility":163,"status":46,"city":164,"state":13,"zip":165,"country":137,"countryCode":138,"cosmosGeoPoint":166,"geoPoint":170,"contacts":13},"St. Mary's Hospital, Manchester University NHS Foundation Trust","Manchester","M13 9WL",{"type":39,"coordinates":167},[168,169],-2.23743,53.48095,{"lat":169,"lon":168},{"facility":172,"status":32,"city":173,"state":13,"zip":174,"country":137,"countryCode":138,"cosmosGeoPoint":175,"geoPoint":179,"contacts":13},"Barnes Clinical Research Facility, Salford Royal Hospital, Northern Care Alliance NHS Foundation Trust","Salford","M6 8HD",{"type":39,"coordinates":176},[177,178],-2.29042,53.48771,{"lat":178,"lon":177},{"type":181,"investigatorFullName":13,"investigatorTitle":13,"investigatorAffiliation":13,"oldNameTitle":13,"oldOrganization":13},"SPONSOR","100637093","a-long-term-observational-study-of-patients-with-fucosidosis-100637093",false,"NCT07615400","A Long-Term Observational Study of Patients With Fucosidosis","A Retrospective and Prospective Natural History Study of Patients With Fucosidosis","Inclusion Criteria (Part A):\n\n* Confirmed diagnosis of fucosidosis\n* Patient either with or without previous allogeneic hematopoietic stem cell transplant (HSCT) for treatment of fucosidosis\n\nExclusion Criteria (Part A):\n\n* Patient\u002Fparent\u002Fcaregiver not willing to consent to participate\n* Patient deceased with no availability of appropriate historical consent, and patient's family\u002Fcaregivers are either unable to be contacted, or refuse consent to data sharing\n\nInclusion Criteria (Part B):\n\n* Patient is living\n* Confirmed diagnosis of fucosidosis\n* Patient either with or without previous allogeneic HSCT for treatment of fucosidosis\n\nExclusion Criteria (Part B):\n\n* Patient\u002Fparent\u002Fcaregiver not willing to consent to participate\n* Current participation in any other interventional or therapeutic study (exception: patients who have previously received bone marrow \u002F HSCT as treatment for fucosidosis are not excluded)\n* Patients who, in the opinion of the site investigator, would be unable or unsuitable to participate in the demands of the study","ALL",{"count":191,"type":192},57,"ESTIMATED","OBSERVATIONAL","The purpose of this observational research study is to learn more about the natural history of fucosidosis, its symptoms, and how it develops over time.\n\nThis study intends to collect information from participants diagnosed with fucosidosis; however, this study does not include any medication or treatment other than the usual medical care provided to study participants.\n\nThe information collected in this study will be used to help understand the disease characteristics of fucosidosis; with this information potentially being able to help design future studies and treatments for this disease.\n\nThere is currently no approved treatment for patients with fucosidosis.\n\nThe study consists of 2 parts: a) Part A - retrospective data collection, and b) Part B - prospective data collection.",[196],"Fucosidosis",[198,199,200,201,202,203,204,205,206,207],"fucosidosis","Lysosomal Storage Diseases","alpha-L-Fucosidase","Genetic Diseases, Inborn","Congenital, Hereditary, and Neonatal Diseases and Abnormalities","Carbohydrate Metabolism, Inborn Errors","Metabolism, Inborn Errors","Alpha-Fucosidase Deficiency","Brain Diseases, Metabolic, Inborn","Lysosomal Storage Diseases, Nervous System","2026-08-04",{"date":210,"type":211},"2026-08-05","ACTUAL",{"date":213,"type":211},"2026-03-25",{"date":215,"type":192},"2031-01",{"name":5,"class":6},16]