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Study of Velaglucerase Beta ERT in Children With Gaucher Disease

ConditionsGaucher DiseaseGaucher Disease Type 1Gaucher Disease Type 3
Trial statusNot yet recruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age2-12
SponsorCANbridge Life Sciences Ltd.

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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;

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

Disqualifiers

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;

Trial design

Design model

Single group

Treatments tested in this trial

  • 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).

Treatment groups

10 Participants
are divided into 1 treatment group
Group A: velaglucerase beta 60 U/kgExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Primary Endpoints

Incidence and number of TEAE, characterized by type, severity, seriousness (SAE), and relatedness to velaglucerase

Time frame
From Baseline to Week 39
2

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

Time frame
From Baseline to Week 39

Secondary outcomes

1

Mean change in hemoglobin concentration (g/dL)

Time frame
From baseline to Week 39
2

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

Time frame
From Baseline to Week 39
3

Mean percent change in platelet count ( 10^9/L)

Time frame
From Baseline to Week 39
4

Mean change in height Z-score

Time frame
From Baseline to Week 39

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

CANbridge Life Sciences Ltd.

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: