Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

Study Overview
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Brief Summary
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Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer) in the cells of the monocyte-macrophage system. This is an extension trial to Study NCT00376168 and NCT00712348.

Detailed Description
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This will be a multi-center, double-blind, parallel group, extension trial to assess the safety and efficacy of prGCD in patients completing NCT00376168. Patients will receive IV infusion of prGCD every two weeks at the selected medical center. The duration of the extension study will be fifteen months. There will be two treatment groups: 30 units/kg every 2 weeks or 60 units/kg every 2 weeks.

Official Title
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A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients 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: Successful completion of Protocol PB-06-001 The patient signs informed consent Exclusion Criteria: Currently taking another experimental drug for any condition Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease Pregnant or nursing Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the patient's compliance with the requirements of the study

Ages Eligible for Study
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Minimum Age: 18 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: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Naive 30 Units/kg<br>Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168) | Drug: Taliglucerase alfa<br>* Intravenous infusion every 2 weeks<br>* Other names: prGCD;|
| Experimental: Naive 60 Units/kg<br>Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168) | Drug: Taliglucerase alfa<br>* Intravenous infusion every 2 weeks<br>* Other names: prGCD;|
| Experimental: Switchover<br>Continue taliglucerase alfa treatment from PB-06-002 (NCT00712348) | Drug: Taliglucerase alfa<br>* Intravenous infusion every 2 weeks<br>* Other names: prGCD;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Spleen Volume | Spleen volume measured by MRI | Spleen Volume at Baseline and Months 12, 24, and 36 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Liver Volume | Liver volume measured by MRI | Liver volume at Baseline and Months 12, 24 and 36 | 
| Hemoglobin |  | Hemoglobin at Baseline and Months 12, 24 and 36 | 
| Platelet Count |  | Platelet count at Baseline and Months 12, 24 and 36 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Gaucher Disease, Enzyme replacement therapy