[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"JCR Pharmaceuticals Co., Ltd.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":96},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,3,0,[8,48,75],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":4,"enrollmentInfo":17,"targetDuration":4,"studyType":20,"phases":4,"briefSummary":21,"conditions":22,"keywords":24,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":36,"lastUpdatePostDateStruct":37,"startDateStruct":40,"completionDateStruct":42,"leadSponsor":44,"locationsCount":47},"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":18,"type":19},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.",[23],"Fucosidosis",[25,26,27,28,29,30,31,32,33,34],"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","RECRUITING","2026-08-04",{"date":38,"type":39},"2026-08-05","ACTUAL",{"date":41,"type":39},"2026-03-25",{"date":43,"type":19},"2031-01",{"name":45,"class":46},"JCR Pharmaceuticals Co., Ltd.","INDUSTRY",16,{"id":49,"slug":50,"hasResults":11,"nctId":51,"briefTitle":52,"officialTitle":53,"acronym":4,"eligibilityCriteria":54,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":55,"enrollmentInfo":56,"targetDuration":4,"studyType":58,"phases":59,"briefSummary":62,"conditions":63,"keywords":4,"overallStatus":65,"whyStopped":4,"lastUpdateSubmitDate":66,"lastUpdatePostDateStruct":67,"startDateStruct":69,"completionDateStruct":71,"leadSponsor":73,"locationsCount":74},"100642331","phase-1-a-phase-iii-trial-of-jr-446-in-mucopolysaccharidosis-type-iiib-mps-iiib-100642331","NCT07640984","A Phase I\u002FII Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)","A Global, Open-Label, Phase I\u002FII Trial, to Evaluate Safety, Tolerability, Pharmacodynamic and Preliminary Efficacy of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB)","Inclusion Criteria:\n\n1. Patients with confirmed MPS IIIB with a phenotype of MPS IIIB that is not classified as slowly progressive (i.e., not attenuated).\n2. A chronological age \\\u003C6 years.\n3. Ability to complete and achieve a Standard score ≥55, using either Bailey Scale of Infant Development-4 (BSID-4) Cognitive domain or Kaufman Assessment Battery for Children-II (KABC-II) Non-Verbal Index, whichever tool is most appropriate for the participant's chronological age.\n\nExclusion Criteria:\n\n1. Contraindication for lumbar puncture or MRI.\n2. A participant who has a medical condition or extenuating circumstance that, in the opinion of the principal investigator or sub-investigator, might compromise the participant's ability to comply with protocol requirements, the participant's well-being or safety, or the interpretability of the participant's clinical data.\n3. A participant who has received any other investigational drug product (including but not limited to, tralesinidase alfa enzyme replacement therapy \\[TA-ERT\\], Genistein, KINERET \\[anakinra\\], ambroxol, miglustat) within 4 months (or 5 half-lives, whichever is longer) before the time of providing informed consent.\n4. A participant who has received gene therapy treatment or hematopoietic stem cell transplantation (HSCT) with successful engraftment.\n5. Serious drug allergy or hypersensitivity to any components of JR-446 or medications likely prescribed during the trial.\n6. A participant has a history of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following lumbar puncture.\n7. A patient with recurrent epileptic seizures not adequately controlled with anti-seizure medication, and which, in the clinical judgment of the principal investigator, would preclude safe participation in the trial.\n8. Serology consistent with human immunodeficiency virus (HIV) exposure or consistent with active hepatitis B (HepB) or hepatitis C (HepC) infection.\n9. A participant\u002Ffamily, who, in the opinion of the investigator, may not be able to comply with protocol requirements and cooperate fully with the trial assessments, procedures, and scheduling for JR-446 IMP dose administrations.\n10. Study participants for whom informed consent is unable to be provided by a parent or legal guardian; or when applicable for a study participant who is unable to provide assent with respect to study participation in conjunction with parental or legal guardian consent for participation on study.\n\nThe above information is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial.","5 Years",{"count":57,"type":19},12,"INTERVENTIONAL",[60,61],"PHASE1","PHASE2","This is a global, open-label, Phase I\u002FII, interventional trial in participants younger than 6 years of age with Mucopolysaccharidosis Type IIIB (MPS IIIB), designed to assess the safety and tolerability of JR-446, determine its pharmacodynamic effects, and explore its potential to demonstrate early clinical effects on disease-relevant outcomes.",[64],"Mucopolysaccharidosis IIIB","NOT_YET_RECRUITING","2026-07-02",{"date":68,"type":39},"2026-07-07",{"date":70,"type":19},"2026-06-18",{"date":72,"type":19},"2028-09-30",{"name":45,"class":46},5,{"id":76,"slug":77,"hasResults":11,"nctId":78,"briefTitle":79,"officialTitle":79,"acronym":4,"eligibilityCriteria":80,"healthyVolunteers":11,"sex":16,"minAge":4,"maxAge":81,"enrollmentInfo":82,"targetDuration":4,"studyType":58,"phases":84,"briefSummary":85,"conditions":86,"keywords":4,"overallStatus":35,"whyStopped":4,"lastUpdateSubmitDate":88,"lastUpdatePostDateStruct":89,"startDateStruct":91,"completionDateStruct":93,"leadSponsor":95,"locationsCount":5},"100553656","phase-1-an-open-label-phase-iii-study-of-jr-446-in-mucopolysaccharidosis-type-iiib-100553656","NCT06488924","An Open-label Phase I\u002FII Study of JR-446 in Mucopolysaccharidosis Type IIIB","Inclusion Criteria:\n\n* Chronological age of \\\u003C18 years\n* Confirmed diagnosis of MPS IIIB\n\nExclusion Criteria:\n\n* Prior experience to gene therapy or HSCT with successful engraftment\n* Past use of another investigational drug or product in last 4 months or 5 half-lives (whichever is longer) before signing ICF\n* Current participation in a clinical trial or past participation (within 30 days of enrolment into this study) in a study involving invasive procedures\n* Past use of Genistein or Kineret (anakinra) within 4 months before signing ICF\n* Serious drug allergy or hypersensitivity\n* Contraindication for lumbar puncture or MRI\n* History of bleeding disorder or current use of medications that, in the opinion of the investigator, place them at risk of bleeding following lumbar puncture\n\nThe above information is not intended to contain all considerations relevant to a patient's potential participation in this clinical trial.","17 Years",{"count":83,"type":19},10,[60,61],"A Phase I\u002F II, open-label study, designed to evaluate the safety and explore efficacy of the study drug in development for the treatment of MPS IIIB patients.",[87],"Mucopolysaccharidosis III-B","2025-08-25",{"date":90,"type":39},"2025-09-02",{"date":92,"type":39},"2024-11-05",{"date":94,"type":19},"2030-04-30",{"name":45,"class":46},""]