NCT07847411Not yet recruitingPhase 3
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
Source: ClinicalTrials.gov · Last updated 29 Sept 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Not yet recruiting
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 10 (estimated)
- First submitted
- 31 Aug 2026
- Start date
- 16 Sept 2026
- Primary completion date
- 25 Feb 2028
- Lead sponsor
- CANbridge Life Sciences Ltd.
- Diseases
- Gaucher disease
- NCT number
- NCT07847411
What you commit to
- Study type: Interventional · Phase 3
- Planned participants: 10
- From 16 Sept 2026 to the primary completion date, 25 Feb 2028
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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.
Other illness
- 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).
Lab values
- 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;
Who the study is looking for
- 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.
Age in the registry: 2 years to 12 years · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Room 202, 2nd Floor, 780 Cailun Road, Pilot Free Trade Zone Coordinator of this site? Confirm its status | Shanghai | China | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.