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| Sponsor: | Protalix |
|---|---|
| Information provided by: | Protalix |
| ClinicalTrials.gov Identifier: | NCT00712348 |
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®) ERT.
| Condition | Intervention | Phase |
|---|---|---|
|
Gaucher Disease |
Drug: Taliglucerase alfa |
Phase III |
| Study Type: | Interventional |
| Study Design: | Control: Uncontrolled Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: 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 |
| Estimated Enrollment: | 15 |
| Study Start Date: | December 2008 |
| Estimated Study Completion Date: | December 2010 |
| Estimated Primary Completion Date: | December 2010 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Taliglucerase alfa: Experimental
Open label taliglucerase alfa treatment
|
Drug: Taliglucerase alfa
Intravenous infusion every 2 weeks
|
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 |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| Contact: Raul Chertkoff, MD | +972-4-9889488 | raul@protalix.com |
| United States, Florida | |
| University Research Foundation for Lysosomal Storage Diseases, Inc. | Recruiting |
| Coral Springs, Florida, United States, 33065 | |
| Contact: Neal J Weinreb, MD 954-755-1904 boneal@winning.com | |
| Principal Investigator: Neal J Weinreb, MD | |
| United States, Georgia | |
| Department of Human Genetics, Emory University School of Medicine | Recruiting |
| Decatur, Georgia, United States, 30033 | |
| Contact: Paul M Fernhoff, MD 404-778-8500 pfernhoff@genetics.emory.edu | |
| Principal Investigator: Paul M Fernhoff, MD | |
| United States, New York | |
| Neurogenetics, NYU at Rivergate | Recruiting |
| New York, New York, United States, 10016 | |
| Contact: Gregory M Pastores, MD 212-263-8344 gregory.pastores@med.nyu.edu | |
| Contact: Michele Ford (212) 263-6981 michele.ford@nyumc.org | |
| Principal Investigator: Gregory M Pastores, MD | |
| Australia, Victoria | |
| Bone Marrow Transplant Service, The Royal Melbourne Hospital | Recruiting |
| Parkville, Victoria, Australia | |
| Contact: Jeffrey Sizer, MD jeff.szer@mh.org.au | |
| Principal Investigator: Jeffrey Sizer, MD | |
| Canada, Ontario | |
| Mount Sinai Hospital | Recruiting |
| Toronto, Ontario, Canada, M5G 1X5 | |
| Contact: Dominick Amato, MD (416) 586-4800 Ext 5189 damato@mtsinai.on.ca | |
| Principal Investigator: Dominick Amato, MD | |
| Israel | |
| Rambam Medical Center | Recruiting |
| Haifa, Israel, 31096 | |
| Contact: Hanna Rosenbaum, MD +972-4-854-3651 h_rosenbaum@rambam.health.gov.il | |
| Principal Investigator: Hanna Rosenbaum, MD | |
| Shaare Zedek Medical Center | Recruiting |
| Jerusalem, Israel | |
| Contact: Ari Zimran, MD 972-2-655-5673 zimran@md.huji.ac.il | |
| Principal Investigator: Ari Zimran, MD | |
| Spain | |
| Sala de Hematologia, Hospital Universitario Miguel Servet | Recruiting |
| Zaragoza, Spain, 50009 | |
| Contact: Pilar Giraldo, MD +34-976765546 pgiraldo@salud.aragon.es | |
| Principal Investigator: Pilar Giraldo, MD | |
| United Kingdom | |
| Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust | Recruiting |
| Cambridge, United Kingdom | |
| Contact: Tim Cox, MD tmc12@medschl.cam.ac.uk | |
| Principal Investigator: Tim Cox, MD | |
| Royal Free Hospital | Recruiting |
| London, United Kingdom, NW3 2QG | |
| Contact: Atul Mehta, MD +44-2078302814 atul.mehta@royalfree.nhs.uk | |
| Principal Investigator: Atul Mehta, MD | |
| Principal Investigator: | Ari Zimran, MD | Shaare Zedek Medical Center, Jerusalem |
More Information
| Responsible Party: | Protalix Biotherapeutics ( Einat Almon ) |
| Study ID Numbers: | PB-06-002 |
| Study First Received: | July 7, 2008 |
| Last Updated: | March 3, 2010 |
| ClinicalTrials.gov Identifier: | NCT00712348 History of Changes |
| Health Authority: | United States: Food and Drug Administration; Israel: Ministry of Health |
|
Gaucher disease imiglucerase glucocerebrosidase enzyme replacement therapy lysosomal storage disorder |
|
Lipid Metabolism, Inborn Errors Sphingolipidoses Metabolic Diseases Reticuloendotheliosis Lysosomal Storage Diseases, Nervous System Lysosomal Storage Diseases Nervous System Diseases Central Nervous System Diseases Brain Diseases |
Lymphatic Diseases Metabolism, Inborn Errors Genetic Diseases, Inborn Brain Diseases, Metabolic, Inborn Lipidoses Gaucher Disease Lipid Metabolism Disorders Brain Diseases, Metabolic |
