Study of Velaglucerase Beta ERT in Children With Gaucher Disease

NCT07847411 | Not Yet Recruiting Phase 3

• 1 other identifier: CAN103-GD-301
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Gaucher Disease; Gaucher Disease Type 1; Gaucher Disease Type 3

Keywords

Gaucher disease; ERT; CAN103; Velaglucerase beta; Gaurunning

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (5)

• Mean change in hemoglobin concentration (g/dL)

  From baseline to Week 39

• Mean percent change in MN 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

Study Arms (1)

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.

Drug: Velaglucerase beta

Interventions

Velaglucerase beta DRUG

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).

velaglucerase beta 60 U/kg

Eligibility Criteria

• Age: 2 Years - 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• CANbridge Life Sciences Ltd.: lead

Study Sites (1)

Room 202, 2nd Floor, 780 Cailun Road, Pilot Free Trade Zone

Shanghai, Shanghai Municipality, China

Location

MeSH Terms

Conditions

Gaucher Disease

Condition Hierarchy (Ancestors)

Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Brain Diseases, Metabolic, Inborn; Brain Diseases, Metabolic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Lysosomal Storage Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Central Study Contacts

Charlene Song

CONTACT

+86 18817946880; xiaoling.song@canbridgepharma.com

Amelia Zhou

CONTACT

+86 18016327369; feng.zhou@canrbdigepharma.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 31, 2026
• First Posted: September 29, 2026
• Study Start: September 16, 2026
• Primary Completion (Estimated): February 25, 2028
• Study Completion (Estimated): February 25, 2028
• Last Updated: September 29, 2026

Record last verified: 2026-09

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP
• Time Frame: Beginning 3 months and ending 3 years after the publication of results
• 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.

Locations

CN (1)