Gaucher DiseasePhase 3NCT07847411
Study of Velaglucerase Beta ERT in Children With Gaucher Disease
A Phase 3 study testing Velaglucerase beta in people with Gaucher Disease and Gaucher Disease Type 1, run by CANbridge Life Sciences Ltd. at 1 site in China.
Trial summary
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 more about this study
- Who is it for?
- Ages 2 to 12 years
- Conditions the study is for
- Gaucher Disease, Gaucher Disease Type 1, Gaucher Disease Type 3
- What is being tested?
- Velaglucerase beta
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 10 participants
- Where
- 1 siteChina
- Study dates
- 16 Sep 2026to 25 Feb 2028
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site
Room 202, 2nd Floor, 780 Cailun Road, Pilot Free Trade Zone
Shanghai, Shanghai Municipality
Amelia Zhou, +86 18016327369, feng.zhou@canrbdigepharma.com
About this study
Official title: 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.
- Being tested
- Velaglucerase beta
- Keywords
- Gaucher disease, ERT, CAN103, Velaglucerase beta, Gaurunning
- Medical subject headings
- Gaucher Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Velaglucerase betaDrug
All subjects will receive velaglucerase beta 60 U/kg IV Q2W for 37 weeks with two weeks of follow-up through Week 39 (9 months).
| Study group | Role | What they receive |
|---|---|---|
| velaglucerase beta 60 U/kg | Experimental | This is a single-arm study. All subjects will receive velaglucerase beta 60 U/kg IV Q2W for 37 weeks. Receives: Drug: Velaglucerase beta |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary Endpoints
Incidence and number of TEAE, characterized by type, severity, seriousness (SAE), and relatedness to velaglucerase
Measured: From Baseline to Week 39
Mean percent change in MN spleen volume
Normal spleen volume = 0.2% body weight. Multiples of normal (MN) spleen volume= actual MRI testing volume / normal spleen volume
Measured: From Baseline to Week 39
Secondary outcomes (5)
Mean change in hemoglobin concentration (g/dL)
Measured: From baseline to Week 39
Mean percent change in MN liver volume
Normal liver volume = 2.5% body weight. Multiples of normal (MN) liver volume= actual MRI testing volume / normal liver volume
Measured: From Baseline to Week 39
Mean percent change in platelet count ( 10^9/L)
Measured: From Baseline to Week 39
Mean change in height Z-score
Measured: From Baseline to Week 39
Mean change in weight Z-score
Measured: From Baseline to Week 39
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Sep 2026
- Study start16 Sep 2026 (estimated)
- Primary completion25 Feb 2028 (estimated)
- Study completion25 Feb 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Gaucher DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 10 participants (estimated)
Sponsor and investigators
- Lead sponsor
- CANbridge Life Sciences Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07847411
- Sponsor's protocol ID
- CAN103-GD-301
- Status last verified
- Sep 2026
- First submitted
- 31 Aug 2026
- Last updated
- 29 Sep 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: only IPD used in the results publication
Study at a glance
From the registry record, last updated by the sponsor on 29 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center, Open-label Study to Evaluate the Safety and Efficacy of Velaglucerase Beta Enzyme Replacement Therapy in Children With Gaucher Disease
- ClinicalTrials.gov ID
- NCT07847411
- Acronym / sponsor ID
- CAN103-GD-301
- Keywords
- Gaucher disease, ERT, CAN103, Velaglucerase beta, Gaurunning
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 10 participants (estimated)
- Lead sponsor
- CANbridge Life Sciences Ltd.Industry (pharma, biotech, devices)
- Study start
- 16 Sep 2026 (estimated)
- Primary completion
- 25 Feb 2028 (estimated)
- Study completion
- 25 Feb 2028 (estimated)
- First posted
- 29 Sep 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| velaglucerase beta 60 U/kg | Experimental | Drug: Velaglucerase betaThis is a single-arm study. All subjects will receive velaglucerase beta 60 U/kg IV Q2W for 37 weeks. |
Who may qualify
- Ages
- 2 to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (8 listed)
- 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:
- 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.
Key exclusion criteria (7 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Velaglucerase betaAll subjects will receive velaglucerase beta 60 U/kg IV Q2W for 37 weeks with two weeks of follow-up through Week 39 (9 months). | Drug | velaglucerase beta 60 U/kg |
Primary outcome
Primary Endpoints
Time frame: From Baseline to Week 39
Incidence and number of TEAE, characterized by type, severity, seriousness (SAE), and relatedness to velaglucerase
Mean percent change in MN spleen volume
Time frame: From Baseline to Week 39
Normal spleen volume = 0.2% body weight. Multiples of normal (MN) spleen volume= actual MRI testing volume / normal spleen volume
5 secondary outcomes
Mean change in hemoglobin concentration (g/dL)
Time frame: From baseline to Week 39
Mean percent change in MN liver volume
Time frame: From Baseline to Week 39
Mean percent change in platelet count ( 10^9/L)
Time frame: From Baseline to Week 39
Mean change in height Z-score
Time frame: From Baseline to Week 39
Mean change in weight Z-score
Time frame: From Baseline to Week 39
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Sep 2026.
- Individual participant dataSharing planned: Yes. only IPD used in the results publication
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Velaglucerase beta in people with Gaucher Disease and Gaucher Disease Type 1, run by CANbridge Life Sciences Ltd. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 29 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Charlene Song (+86 18817946880, xiaoling.song@canbridgepharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary Endpoints (time frame: From Baseline to Week 39). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Sep 2026.