Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase
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ClinicalTrials.gov Identifier: NCT00712348 |
Recruitment Status :
Completed
First Posted : July 10, 2008
Results First Posted : June 5, 2014
Last Update Posted : October 4, 2018
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Condition or disease | Intervention/treatment | Phase |
---|---|---|
Gaucher Disease | Drug: Taliglucerase alfa | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 31 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | 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 |
Study Start Date : | December 2008 |
Actual Primary Completion Date : | April 2013 |
Actual Study Completion Date : | May 2013 |

Arm | Intervention/treatment |
---|---|
Experimental: Taliglucerase alfa
Open label taliglucerase alfa treatment
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Drug: Taliglucerase alfa
Intravenous infusion every 2 weeks
Other Name: Plant cell expressed recombinant glucocerebrosidase (prGCD) |
- Hemoglobin [ Time Frame: Every 3 months from Baseline to Month 9 ]
- Platelet Count [ Time Frame: Every 3 months from Baseline to Month 9 ]
- Spleen Volume [ Time Frame: Baseline and 9 Months ]Spleen volume measured by MRI in mL
- Liver Volume [ Time Frame: Baseline and 9 months ]Liver volume measured by MRI

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 2 Years and older (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00712348
United States, Florida | |
University Research Foundation for Lysosomal Storage Diseases, Inc. | |
Coral Springs, Florida, United States, 33065 | |
United States, Georgia | |
Department of Human Genetics, Emory University School of Medicine | |
Decatur, Georgia, United States, 30033 | |
United States, New York | |
Neurogenetics, NYU at Rivergate | |
New York, New York, United States, 10016 | |
Australia, Victoria | |
Bone Marrow Transplant Service, The Royal Melbourne Hospital | |
Parkville, Victoria, Australia | |
Canada, Ontario | |
Mount Sinai Hospital | |
Toronto, Ontario, Canada, M5G 1X5 | |
Israel | |
Rambam Medical Center | |
Haifa, Israel, 31096 | |
Shaare Zedek Medical Center | |
Jerusalem, Israel | |
Spain | |
Sala de Hematologia, Hospital Universitario Miguel Servet | |
Zaragoza, Spain, 50009 | |
United Kingdom | |
Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust | |
Cambridge, United Kingdom | |
Royal Free Hospital | |
London, United Kingdom, NW3 2QG |
Responsible Party: | Pfizer |
ClinicalTrials.gov Identifier: | NCT00712348 |
Other Study ID Numbers: |
PB-06-002 |
First Posted: | July 10, 2008 Key Record Dates |
Results First Posted: | June 5, 2014 |
Last Update Posted: | October 4, 2018 |
Last Verified: | September 2018 |
Gaucher disease imiglucerase glucocerebrosidase enzyme replacement therapy lysosomal storage disorder |
Gaucher Disease 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 Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders |