Study of Velaglucerase Beta ERT in Children With Gaucher Disease

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.

Inclusion Criteria: 1. 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; 2. Written informed consent provided by the subject's parent/legally authorized representative; 3. Subject is less than 12 years of age; 4. 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: 1. GD-related anemia, with a decrease of ≥1 g/dL in hemoglobin concentration below the lower limit of normal for age of the central laboratory; or 2. GD-related hepatomegaly by palpation; or 3. GD-related thrombocytopenia, with a platelet count \< 100 × 10\^9/L; 5. 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. Exclusion Criteria: 1. Treatment with investigational drugs (including ambroxol) within 30 days or 5 half-lives, whichever is longer, prior to screening; 2. 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; 3. Subject has non-Gaucher disease-related anemia (such as due to iron, folic acid, and/or vitamin B12 deficiency or infection/immune-mediated reasons); 4. Subject has had a prior hepatectomy and/or splenectomy (including partial liver and/or splenectomy) or plans to have a hepatectomy and/or splenectomy (including partial liver and/or splenectomy) during the study; 5. Subject has received organ transplantation, including hematopoietic stem cell transplantation; 6. 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). 7. Injection with a live vaccine within 30 days of the first study dose.