[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100651289":3},{"organization":4,"armGroups":7,"interventions":10,"overallOfficials":18,"centralContacts":22,"locations":32,"responsibleParty":47,"collaborators":49,"id":53,"slug":54,"hasResults":55,"nctId":56,"briefTitle":57,"officialTitle":58,"acronym":10,"eligibilityCriteria":59,"healthyVolunteers":55,"sex":60,"minAge":61,"maxAge":62,"enrollmentInfo":63,"targetDuration":10,"studyType":66,"phases":10,"briefSummary":67,"conditions":68,"keywords":10,"overallStatus":35,"whyStopped":10,"lastUpdateSubmitDate":70,"lastUpdatePostDateStruct":71,"startDateStruct":74,"completionDateStruct":76,"leadSponsor":78,"locationsCount":79},{"fullName":5,"class":6},"Yale University","OTHER",[8,12,15],{"label":9,"type":10,"description":11,"interventionNames":10},"Neuronopathic Gaucher Disease",null,"Participants with GD2 or GD3 and documented pulmonary disease and\u002For massive lymphadenopathy.",{"label":13,"type":10,"description":14,"interventionNames":10},"Refractory Skeletal Disease","Participants with GD1 or GD3, ages 10-75, with persistent skeletal disease despite long-term therapy.",{"label":16,"type":10,"description":17,"interventionNames":10},"Gaucher-Parkinson Overlap","Adult participants only with Gaucher disease and clinical features of Parkinson disease.",[19],{"name":20,"affiliation":5,"role":21},"Pramod K Mistry, MD, PhD","PRINCIPAL_INVESTIGATOR",[23,28],{"name":24,"role":25,"phone":26,"phoneExt":10,"email":27},"Pramod Yang, BS, CCRG","CONTACT","203-785-3412","Ruhua.Yang@yale.edu",{"name":29,"role":25,"phone":30,"phoneExt":10,"email":31},"Pramod K. Mistry, MD, PhD","2036057878","pramod.mistry@yale.edu",[33],{"facility":34,"status":35,"city":36,"state":37,"zip":38,"country":39,"countryCode":40,"cosmosGeoPoint":41,"geoPoint":46,"contacts":10},"Yale New Haven Health System","RECRUITING","New Haven","Connecticut","06520","United States","US",{"type":42,"coordinates":43},"Point",[44,45],-72.92816,41.30815,{"lat":45,"lon":44},{"type":48,"investigatorFullName":10,"investigatorTitle":10,"investigatorAffiliation":10,"oldNameTitle":10,"oldOrganization":10},"SPONSOR",[50],{"name":51,"class":52},"Sanofi-Yale External Research Collaboration","UNKNOWN","100651289","advancing-integrated-therapies-for-gaucher-disease-100651289",false,"NCT07758816","Advancing Integrated Therapies for Gaucher Disease","Advancing Integrated Therapies for Gaucher Disease: Neuronopathic Innovation and Combination Strategies for Refractory Skeletal Disease","Inclusion Criteria:\n\nAim 1\n\n* Confirmed diagnosis of GD2 or GD3 based on genotype and phenotype\n* Evidence of pulmonary infiltrative disease and\u002For mediastinal or mesenteric lymphadenopathy\n* Receiving or eligible for standard-of-care therapy\n* Age ≥ 3 months\n* Ability to provide informed consent (or parental consent with assent as appropriate)\n\nAim2\n\n* ages 10-75\n* persistent skeletal disease despite long-term therapy\n\nAim3\n\n* Gaucher disease and clinical features of Parkinson disease\n* Ability to provide informed consent\n\nExclusion Criteria:\n\nAim 1\n\n* Inability to comply with observational follow-up\n* Any condition that, in the investigator's judgment, precludes safe participation","ALL","3 Months","75 Years",{"count":64,"type":65},30,"ESTIMATED","OBSERVATIONAL","The purpose of this study is to better understand the natural history, clinical outcomes, and biological features of Gaucher disease in patients receiving standard medical care",[69],"Gaucher Disease","2026-08-05",{"date":72,"type":73},"2026-08-11","ACTUAL",{"date":75,"type":73},"2026-05-06",{"date":77,"type":65},"2030-09",{"name":5,"class":6},1]