[{"data":1,"prerenderedAt":-1},["ShallowReactive",2],{"health-studies-list:{\"overallStatus\":[\"RECRUITING\",\"AVAILABLE\",\"NOT_YET_RECRUITING\"],\"leadSponsorName\":\"CANbridge Life Sciences Ltd.\",\"orderBy\":\"LastUpdateSubmitDate:desc\",\"size\":25,\"offset\":0}":3,"health-study-condition:":48},{"pageToken":4,"total":5,"offset":6,"count":5,"results":7},null,1,0,[8],{"id":9,"slug":10,"hasResults":11,"nctId":12,"briefTitle":13,"officialTitle":14,"acronym":4,"eligibilityCriteria":15,"healthyVolunteers":11,"sex":16,"minAge":17,"maxAge":18,"enrollmentInfo":19,"targetDuration":4,"studyType":22,"phases":23,"briefSummary":25,"conditions":26,"keywords":30,"overallStatus":36,"whyStopped":4,"lastUpdateSubmitDate":37,"lastUpdatePostDateStruct":38,"startDateStruct":41,"completionDateStruct":43,"leadSponsor":45,"locationsCount":5},"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":20,"type":21},10,"ESTIMATED","INTERVENTIONAL",[24],"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.",[27,28,29],"Gaucher Disease","Gaucher Disease Type 1","Gaucher Disease Type 3",[31,32,33,34,35],"Gaucher disease","ERT","CAN103","Velaglucerase beta","Gaurunning","NOT_YET_RECRUITING","2026-09-28",{"date":39,"type":40},"2026-09-29","ACTUAL",{"date":42,"type":21},"2026-09-16",{"date":44,"type":21},"2028-02-25",{"name":46,"class":47},"CANbridge Life Sciences Ltd.","INDUSTRY",""]