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Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase
This study has been completed.
Sponsor:
Protalix
Information provided by (Responsible Party):
Protalix
ClinicalTrials.gov Identifier:
NCT00712348
First received: July 7, 2008
Last updated: May 30, 2014
Last verified: May 2014
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Purpose
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.
| Condition | Intervention | Phase |
|---|---|---|
| Gaucher Disease | Drug: Taliglucerase alfa | Phase 3 |
| Study Type: | Interventional |
| Study Design: | 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 |
Resource links provided by NLM:
Genetics Home Reference related topics:
Gaucher disease
MedlinePlus related topics:
Gaucher Disease
U.S. FDA Resources
Further study details as provided by Protalix:
Primary Outcome Measures:
- Hemoglobin [ Time Frame: Every 3 months from Baseline to Month 9 ]
Other Outcome Measures:
- 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
| Enrollment: | 31 |
| Study Start Date: | December 2008 |
| Study Completion Date: | May 2013 |
| Primary Completion Date: | April 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: Taliglucerase alfa
Open label taliglucerase alfa treatment
|
Drug: Taliglucerase alfa
Intravenous infusion every 2 weeks
Other Name: Plant cell expressed recombinant glucocerebrosidase (prGCD)
|
Detailed Description:
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.
Eligibility| Ages Eligible for Study: | 2 Years and older (Child, Adult, Senior) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
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
Contacts and Locations
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To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below.
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Please refer to this study by its ClinicalTrials.gov identifier: NCT00712348
Please refer to this study by its ClinicalTrials.gov identifier: NCT00712348
Locations
| 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 | |
Sponsors and Collaborators
Protalix
Investigators
| Principal Investigator: | Ari Zimran, MD | Shaare Zedek Medical Center, Jerusalem |
More Information
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
| Responsible Party: | Protalix |
| ClinicalTrials.gov Identifier: | NCT00712348 History of Changes |
| Other Study ID Numbers: |
PB-06-002 |
| Study First Received: | July 7, 2008 |
| Results First Received: | April 30, 2014 |
| Last Updated: | May 30, 2014 |
Keywords provided by Protalix:
|
Gaucher disease imiglucerase glucocerebrosidase enzyme replacement therapy lysosomal storage disorder |
Additional relevant MeSH terms:
|
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 |
ClinicalTrials.gov processed this record on September 21, 2017