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

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 Overview

• Status: Not yet recruiting
• Conditions: Gaucher Disease; Gaucher Disease Type 1; Gaucher Disease Type 3
• Intervention / Treatment: Drug: Velaglucerase beta

• Study Type: Interventional
• Enrollment (Estimated): 10
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Charlene Song; Phone Number: +86 18817946880; Email: xiaoling.song@canbridgepharma.com
• Study Contact Backup: Name: Amelia Zhou; Phone Number: +86 18016327369; Email: feng.zhou@canrbdigepharma.com

Study Locations

• China
  • Shanghai Municipality
    • Shanghai, Shanghai Municipality, China
      • Room 202, 2nd Floor, 780 Cailun Road, Pilot Free Trade Zone
      • Contact: Amelia Zhou; Phone Number: +86 18016327369; Email: feng.zhou@canrbdigepharma.com

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years to 12 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: velaglucerase beta 60 U/kg; This is a single-arm study. All subjects will receive velaglucerase beta 60 U/kg IV Q2W for 37 weeks.	Drug: Velaglucerase beta; 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).

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Primary Endpoints	Incidence and number of TEAE, characterized by type, severity, seriousness (SAE), and relatedness to velaglucerase	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	From Baseline to Week 39

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Mean change in hemoglobin concentration (g/dL)		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	From Baseline to Week 39
Mean percent change in platelet count ( 10^9/L)		From Baseline to Week 39
Mean change in height Z-score		From Baseline to Week 39
Mean change in weight Z-score		From Baseline to Week 39

Collaborators and Investigators

• Sponsor: CANbridge Life Sciences Ltd.

Study record dates

Study Major Dates

• Study Start (Estimated): September 16, 2026
• Primary Completion (Estimated): February 25, 2028
• Study Completion (Estimated): February 25, 2028

Study Registration Dates

• First Submitted: August 31, 2026
• First Submitted That Met QC Criteria: September 28, 2026
• First Posted (Actual): September 29, 2026

Study Record Updates

• Last Update Posted (Actual): September 29, 2026
• Last Update Submitted That Met QC Criteria: September 28, 2026
• Last Verified: September 1, 2026

More Information

Terms related to this study

• Keywords: Gaucher disease; ERT; CAN103; Velaglucerase beta; Gaurunning
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Lipid Metabolism Disorders; Lysosomal Storage Diseases; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases, Nervous System; Sphingolipidoses; Lipidoses; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Gaucher Disease
• Other Study ID Numbers: CAN103-GD-301

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES

IPD Plan Description

only IPD used in the results publication

IPD Sharing Time Frame

Beginning 3 months and ending 3 years after the publication of results

IPD Sharing Access Criteria

The criteria that must be met the types of analyses that qualify for data sharing.

A proposal that describes planned analyses must be submitted.

• IPD Sharing Supporting Information Type: STUDY_PROTOCOL; SAP

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No