A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

Study Overview
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Brief Summary
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This is a multi-center, double-blind trial to assess the safety and efficacy of taliglucerase alfa in untreated subjects (2 to <18 years old) with Gaucher disease randomly assigned to treatment with one of two doses, 30 or 60 units/kg. Subjects will receive an intravenous (IV) infusion of taliglucerase alfa every two weeks. The total duration of treatment will be 12 months. At the end of the 12-month treatment period eligible subjects will be offered enrollment in an open-label extension study if taliglucerase alfa is not commercially available.

Official Title
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A Multicenter, Double-blind, Randomized Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

Conditions
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Gaucher Disease

Intervention / Treatment
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* Drug: Taliglucerase alfa


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Males and females 2 to <18 years old. Diagnosis of Gaucher disease with leukocyte acid β-glucosidase activity ≤30% of the mean of the reference range for healthy subjects. Subjects who have not received enzyme replacement therapy (ERT) in the past or who have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody assay. Subjects who have not received substrate reduction therapy (SRT) in the past 12 months. Subjects whose clinical condition, in the opinion of the investigator, requires treatment with enzyme replacement therapy (ERT). Exclusion Criteria: Currently taking another investigational drug for any condition. Presence of neurological signs and symptoms characteristic of Gaucher disease with complex neuronopathic features other than longstanding oculomotor gaze palsy. Presence of unresolved anemia due to iron, folic acid, or vitamin B12 deficiency Previous hypersensitivity reaction to Cerezyme® (imiglucerase) or Ceredase® (alglucerase). History of allergy to carrots. Presence of HIV, HBsAg or hepatitis C infections. Subject's parent(s) or legal guardian(s) are unable to understand the nature, scope and possible consequences of the study. Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the subject's compliance with the requirements of the study.

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 17 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: 30 units/kg<br> | Drug: Taliglucerase alfa<br>* Taliglucerase alfa for infusion every two weeks for 12 months<br>* Other names: prGCD, plant cell expressed glucocerebrosidase;|
| Experimental: 60 units/kg<br> | Drug: Taliglucerase alfa<br>* Taliglucerase alfa for infusion every two weeks for 12 months<br>* Other names: prGCD, plant cell expressed glucocerebrosidase;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hemoglobin | median and interquartile range for change from baseline in haemoglobin | Every 3 months for 12 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Chitotriosidase | Percent change from baseline in chitotriosidase | Every 3 months for 12 months | 
| Spleen Volume | Spleen volume measured by MRI | Baseline and Month 12 | 
| Platelet Count | Mean and standard deviation of platelet count per cubic mm | Baseline and 12 months | 
| Chemokine (C-C Motif) Ligand 18 (CCL18) | Percent change from baseline in CCL18 | Every 3 months for 12 months | 
| Liver Volume | Liver volume measured by MRI | Baseline and Month 12 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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gaucher disease, pediatric