Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

Study Overview
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Brief Summary
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This is a multi-center, open-label, switchover trial to assess the safety of taliglucerase alfa in 30 patients with Gaucher disease who are currently being treated with imiglucerase (Cerezyme®) enzyme replacement therapy.

Detailed Description
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This is a multi-center, open-label, switchover trial to assess the safety of taliglucerase alfa in 30 patients with Gaucher disease who are currently being treated with imiglucerase (Cerezyme®) ERT. Eligible patients will enter a 12-week Stability Evaluation Period to establish the stability of their disease. Patients with stable disease will then be switched from their imiglucerase treatment to receive intravenous (IV) infusions of taliglucerase alfa every two weeks for a total of 20 IV infusions. The dose of taliglucerase alfa will be equal to each patient's previous imiglucerase dose. The infusions will be administered at the selected investigational site (clinic/hospital), infusion center, or at home. At the end of the 9-month treatment period (20 visits, 38 weeks) eligible patients will be offered enrollment in an open-label extension study.

Official Title
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A Phase 3 Multicenter, Open-label, Switchover Trial to Assess the Safety and Efficacy of Plant Cell Expressed Recombinant Human Glucocerebrosidase in Patients With Gaucher Disease Treated With Imiglucerase

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 years or older Confirmed diagnosis of Gaucher disease by the enzymatic activity assay Stable Gaucher disease Treatment with imiglucerase (Cerezyme®) for at least 2 years and on a stable maintenance regimen (dose and regimen unchanged, except for situation of drug shortage) for at least the last six months Able to provide written informed consent Exclusion Criteria: Currently taking another experimental drug for any condition History of allergy to carrots History of allergy to beta lactam antibiotics Previous infusion reaction suspected to be allergic in nature to Cerezyme® or Ceredase® or receiving premedication to prevent infusion reactions Presence of HIV and/or HBsAg and/or hepatitis C infection Presence of unresolved anemia due to iron, folic acid or vitamin B12 deficiency Presence of any significant comorbidity that could confound the interpretation of the clinical response to taliglucerase alfa 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: 2 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
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Taliglucerase alfa<br>Open label taliglucerase alfa treatment | Drug: Taliglucerase alfa<br>* Intravenous infusion every 2 weeks<br>* Other names: Plant cell expressed recombinant glucocerebrosidase (prGCD);|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Hemoglobin |  | Every 3 months from Baseline to Month 9 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Gaucher disease, imiglucerase, glucocerebrosidase, enzyme replacement therapy, lysosomal storage disorder