Who may be eligible for the Study of Velaglucerase Beta ERT in Children With Gaucher Disease trial?

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.

ClinicalTrials.gov record NCT07847411

Study of Velaglucerase Beta ERT in Children With Gaucher Disease

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Oct 10, 2026

A Multi-center, Open-label Study to Evaluate the Safety and Efficacy of Velaglucerase Beta Enzyme Replacement Therapy in Children With Gaucher Disease

About this page. This page shows the study's official ClinicalTrials.gov record ↗, with added help to make it easier to understand. We explain medical terms, highlight key details in plain language, and suggest questions you may want to ask the study team.

The record is periodically copied from ClinicalTrials.gov and may lag behind changes. AI summaries are checked by software, not a clinician.Read moreShow less

The official record is written by the study sponsor and published on ClinicalTrials.gov. It may not always reflect the latest changes. Before making any decisions, please confirm important information with the study team and your own healthcare team.

Where you see "AI summary", AI was used to create a plain-language explanation and software checked it against the source. It has not been reviewed by a clinician, so it may contain mistakes or leave out important details.

StatusNot yet recruitingThe study has been registered but has not started enrolling. Sites and contacts may not be listed yet.

Study typeInterventionalA study in which researchers assign participants to one or more interventions, such as a drug, procedure, device, behavioural programme or other approach, to measure their effects.

PhasePhase 3A later-stage study, usually with more participants, designed to give stronger evidence about how well a treatment works and how safe it is. It often compares the treatment with standard care or a placebo, and results may support an application for approval.

Age2 years old and older

SexAll

LocationChina

AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This Phase 3, open-label, single-arm study gives velaglucerase beta to children with Gaucher disease (types 1 or 3). All participants receive 60 U/kg IV every two weeks for 37 weeks and are followed through Week 39. The study measures safety, organ volumes, blood counts, growth, pharmacokinetics/pharmacodynamics, and immune response to the drug.

What it is studying. The study tests the safety and treatment effects of velaglucerase beta given every two weeks in children with Gaucher disease. It also measures drug levels and activity (PK/PD), immune responses to the drug (immunogenicity), spleen and liver volumes, blood counts, and growth.

What the researchers hope to learn. Collect safety data (treatment-emergent adverse events); measure changes in spleen and liver volume, hemoglobin, platelet count, and growth; and characterize pharmacokinetics, pharmacodynamics, and immunogenicity of velaglucerase beta in children with Gaucher disease (types 1 and 3).

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

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.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug: Velaglucerase beta

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

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 criteria

Treatment with investigational drugs (including ambroxol) within 30 days or 5 half-lives, whichever is longer, prior to screening;
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;
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 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;
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).
Injection with a live vaccine within 30 days of the first study dose.

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants will receive velaglucerase beta by intravenous (IV) infusion at 60 U/kg every two weeks for 37 weeks, with two weeks of follow-up through Week 39. The study includes MRI scans to measure spleen and liver volume, periodic blood tests to measure hemoglobin and platelet counts and to assess drug levels and immune response (PK/PD and immunogenicity), and measurement of height and weight. You will be monitored for side effects and treatment-emergent adverse events from baseline to Week 39.

How the study is structured. This is a Phase 3 interventional study that is open-label and has a single treatment group. Everyone enrolled receives velaglucerase beta 60 U/kg by IV every two weeks for 37 weeks, with follow-up through Week 39. The study records treatment-emergent adverse events and measures MRI-based spleen and liver volumes, hemoglobin and platelet counts, height and weight, plus pharmacokinetic/pharmacodynamic and immunogenicity testing. There is no placebo or comparison group and the treatment is not masked.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

The registry says recruitment has not started. When is it expected to open, and can I register interest now?
The listed sites are in China. Is any of them reachable for me, and does the study help with travel?
Which of the eligibility criteria would need to be checked with tests before I could join?

More on Gaucher disease

Clinical trials64 trials listed for Gaucher diseaseEvery trial the registry lists for Gaucher disease, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Gaucher disease researchLatest monthly update: September 2026. Includes: Platelet release. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start: Sep 16, 2026 (estimated)
Primary completion: Feb 25, 2028 (estimated)
Study completion: Feb 25, 2028 (estimated)
Target enrollment: 10 participants (estimated)
Last updated on registry: Sep 29, 2026
Data verified: Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor: CANbridge Life Sciences Ltd.

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Charlene Song 📞 +86 18817946880 ✉ xiaoling.song@canbridgepharma.com
Amelia Zhou 📞 +86 18016327369 ✉ feng.zhou@canrbdigepharma.com

Participating sites (1)

Room 202, 2nd Floor, 780 Cailun Road, Pilot Free Trade Zone
Shanghai, Shanghai Municipality · China
- Amelia Zhou · +86 18016327369 · feng.zhou@canrbdigepharma.com

Terms used on this page · explanations by MedicalUpdates.ai

open-label: No masking is used, so the assigned intervention is not deliberately hidden from participants or study staff.
single-arm: All participants receive the same treatment or follow the same plan. There is no comparison group within the study.
PK: How the body absorbs, distributes, breaks down and removes a drug. It is studied by measuring drug levels or related substances at set times after a dose.
Written informed consent: The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
legally authorized representative: A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
prior to screening: The checks done before enrolment to see whether a person qualifies, such as tests, scans or a review of medical history.
investigational: A treatment that is being tested and has not been approved for this use. It may be approved for a different condition. Being studied does not mean it works or is available outside the study.
5 half-lives: The time it takes for the amount of a drug in the body to fall by half. 'Five half-lives' is often used as the point at which a previous drug has essentially left the body.
corticosteroids: Steroid medicines, such as prednisone, that reduce inflammation. Studies often require a stable dose at entry or measure whether the study treatment allows the dose to be reduced.
Inclusion Criteria: The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria: The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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