[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-study-detail:100658007":3},{"organization":4,"outcomesModule":7,"designInfo":30,"detailedDescription":19,"studyPopulation":19,"armGroups":36,"interventions":43,"overallOfficials":19,"centralContacts":49,"locations":59,"responsibleParty":74,"collaborators":19,"id":76,"slug":77,"hasResults":78,"nctId":79,"briefTitle":80,"officialTitle":81,"acronym":19,"eligibilityCriteria":82,"healthyVolunteers":78,"sex":83,"minAge":84,"maxAge":85,"enrollmentInfo":86,"targetDuration":19,"studyType":89,"phases":90,"briefSummary":92,"conditions":93,"keywords":97,"overallStatus":102,"whyStopped":19,"lastUpdateSubmitDate":103,"lastUpdatePostDateStruct":104,"startDateStruct":107,"completionDateStruct":109,"leadSponsor":111,"locationsCount":112},{"fullName":5,"class":6},"CANbridge Life Sciences Ltd.","INDUSTRY",{"primaryOutcomes":8,"secondaryOutcomes":16,"otherOutcomes":19},[9,13],{"measure":10,"description":11,"timeFrame":12},"Primary Endpoints","Incidence and number of TEAE, characterized by type, severity, seriousness (SAE), and relatedness to velaglucerase","From Baseline to Week 39",{"measure":14,"description":15,"timeFrame":12},"Mean percent change in MN spleen volume","Normal spleen volume = 0.2% body weight. Multiples of normal (MN) spleen volume= actual MRI testing volume \u002F normal spleen volume",[17,21,24,26,28],{"measure":18,"description":19,"timeFrame":20},"Mean change in hemoglobin concentration (g\u002FdL)",null,"From baseline to Week 39",{"measure":22,"description":23,"timeFrame":12},"Mean percent change in MN liver volume","Normal liver volume = 2.5% body weight. Multiples of normal (MN) liver volume= actual MRI testing volume \u002F normal liver volume",{"measure":25,"description":19,"timeFrame":12},"Mean percent change in platelet count ( 10^9\u002FL)",{"measure":27,"description":19,"timeFrame":12},"Mean change in height Z-score",{"measure":29,"description":19,"timeFrame":12},"Mean change in weight Z-score",{"allocation":31,"interventionModel":32,"interventionModelDescription":19,"primaryPurpose":33,"observationalModel":19,"timePerspective":19,"maskingInfo":34},"NA","SINGLE_GROUP","TREATMENT",{"masking":35,"maskingDescription":19,"whoMasked":19},"NONE",[37],{"label":38,"type":39,"description":40,"interventionNames":41},"velaglucerase beta 60 U\u002Fkg","EXPERIMENTAL","This is a single-arm study. All subjects will receive velaglucerase beta 60 U\u002Fkg IV Q2W for 37 weeks.",[42],"Drug: Velaglucerase beta",[44],{"type":45,"name":46,"description":47,"armGroupLabels":48,"otherNames":19},"DRUG","Velaglucerase beta","All subjects will receive velaglucerase beta 60 U\u002Fkg IV Q2W for 37 weeks with two weeks of follow-up through Week 39 (9 months).",[38],[50,55],{"name":51,"role":52,"phone":53,"phoneExt":19,"email":54},"Charlene Song","CONTACT","+86 18817946880","xiaoling.song@canbridgepharma.com",{"name":56,"role":52,"phone":57,"phoneExt":19,"email":58},"Amelia Zhou","+86 18016327369","feng.zhou@canrbdigepharma.com",[60],{"facility":61,"status":19,"city":62,"state":63,"zip":19,"country":64,"countryCode":65,"cosmosGeoPoint":66,"geoPoint":71,"contacts":72},"Room 202, 2nd Floor, 780 Cailun Road, Pilot Free Trade Zone","Shanghai","Shanghai Municipality","China","CN",{"type":67,"coordinates":68},"Point",[69,70],121.45806,31.22222,{"lat":70,"lon":69},[73],{"name":56,"role":52,"phone":57,"phoneExt":19,"email":58},{"type":75,"investigatorFullName":19,"investigatorTitle":19,"investigatorAffiliation":19,"oldNameTitle":19,"oldOrganization":19},"SPONSOR","100658007","phase-3-study-of-velaglucerase-beta-ert-in-children-with-gaucher-disease-100658007",false,"NCT07847411","Study of Velaglucerase Beta ERT in Children With Gaucher Disease","A Multi-center, Open-label Study to Evaluate the Safety and Efficacy of Velaglucerase Beta Enzyme Replacement Therapy in Children With Gaucher Disease","Inclusion Criteria:\n\n1. Subject has a documented diagnosis of GD1 or GD3 according to the diagnostic criteria of the Expert Consensus on the Diagnosis and Treatment of Gaucher Disease in Chinese Children 2021;\n2. Written informed consent provided by the subject's parent\u002Flegally authorized representative;\n3. Subject is less than 12 years of age;\n4. Subject has GD-related splenomegaly, defined as at least 2 to 3 cm below the left costal margin by palpation, and one or more of the following:\n\n   1. GD-related anemia, with a decrease of ≥1 g\u002FdL in hemoglobin concentration below the lower limit of normal for age of the central laboratory; or\n   2. GD-related hepatomegaly by palpation; or\n   3. GD-related thrombocytopenia, with a platelet count \\\u003C 100 × 10\\^9\u002FL;\n5. Subject has not received ERT or SRT for at least 3 months prior to screening; Female subjects of childbearing age have a negative serum pregnancy test during screening.\n\nExclusion Criteria:\n\n1. Treatment with investigational drugs (including ambroxol) within 30 days or 5 half-lives, whichever is longer, prior to screening;\n2. Subjects have received erythropoietin, whole blood transfusion or transfusion of red blood cells, or long-term (continuous treatment for more than 3 months) systemic corticosteroids 3 months prior to screening; or received a platelet transfusion within 1 month prior to screening;\n3. Subject has non-Gaucher disease-related anemia (such as due to iron, folic acid, and\u002For vitamin B12 deficiency or infection\u002Fimmune-mediated reasons);\n4. Subject has had a prior hepatectomy and\u002For splenectomy (including partial liver and\u002For splenectomy) or plans to have a hepatectomy and\u002For splenectomy (including partial liver and\u002For splenectomy) during the study;\n5. Subject has received organ transplantation, including hematopoietic stem cell transplantation;\n6. Subject has had a history of CTCAE Grade 3 or above infusion-related reaction or hypersensitivity reaction to imiglucerase or other ERTs (approved or experimental).\n7. Injection with a live vaccine within 30 days of the first study dose.","ALL","2 Years","12 Years",{"count":87,"type":88},10,"ESTIMATED","INTERVENTIONAL",[91],"PHASE3","This is a Phase 3, multicenter, open-label, single-arm study to evaluate the safety, efficacy, PK, PD, and immunogenicity of velaglucerase beta in untreated children with GD1 or GD3 for 39 weeks.",[94,95,96],"Gaucher Disease","Gaucher Disease Type 1","Gaucher Disease Type 3",[98,99,100,46,101],"Gaucher disease","ERT","CAN103","Gaurunning","NOT_YET_RECRUITING","2026-09-28",{"date":105,"type":106},"2026-09-29","ACTUAL",{"date":108,"type":88},"2026-09-16",{"date":110,"type":88},"2028-02-25",{"name":5,"class":6},1]