Velaglucerase beta enzyme replacement for children with Gaucher disease
Tests velaglucerase beta in children with Gaucher disease not yet treated.
Plain-language title generated from the registry record
Registry title: Study of Velaglucerase Beta ERT in Children With Gaucher Disease
Key facts
- Where
- Shanghai, ChinaSee the site
- Phase
- Phase 3
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 2 and 12 years old?
Source: NCT07847411, who can take part
Where in China
- Room 202, 2nd Floor, 780 Cailun Road, Pilot Free Trade ZoneShanghai, ChinaNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- CANbridge Life Sciences Ltd.
- Trial code
- NCT07847411
- Record updated
- 29 September 2026
- Original description
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.Read full original descriptionShow less
A Multi-center, Open-label Study to Evaluate the Safety and Efficacy of Velaglucerase Beta Enzyme Replacement Therapy 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: 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; Written informed consent provided by the subject's parent/legally authorized representative; Subject is less than 12 years of age; 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:; 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; GD-related hepatomegaly by palpation; or; GD-related thrombocytopenia, with a platelet count < 100 × 10\^9/L; 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: Treatment with investigational drugs (including ambroxol) within 30 days or 5 half-lives, whichever is longer, prior to screening; 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; Subject has received organ transplantation, including hematopoietic stem cell transplantation; Injection with a live vaccine within 30 days of the first study dose; Subject has non-Gaucher disease-related anemia (such as due to iron, folic acid, and/or vitamin B12 deficiency or infection/immune-mediated reasons); 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); 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.