Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

Condition(s) targeted: Gaucher Disease

Intervention: Taliglucerase alfa (Drug)

Phase: Phase 3

Status: Recruiting

Sponsored by: Protalix

Official(s) and/or principal investigator(s):
Ari Zimran, MD, Principal Investigator, Affiliation: Shaare Zedek Medical Center, Jerusalem

Overall contact:
Raul Chertkoff, MD, Phone: +972-4-9889488, Email: raul@protalix.com

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.

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 design: Control: Uncontrolled, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Primary outcome: Adverse events

Secondary outcome: Platelet count

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.

Minimum age: 2 Years. Maximum age: N/A. Gender(s): Both.

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

Raul Chertkoff, MD, Phone: +972-4-9889488, Email: raul@protalix.com

Rambam Medical Center, Haifa 31096, Israel; Recruiting
Hanna Rosenbaum, MD, Phone: +972-4-854-3651, Email: h_rosenbaum@rambam.health.gov.il
Hanna Rosenbaum, MD, Principal Investigator

Shaare Zedek Medical Center, Jerusalem, Israel; Recruiting
Ari Zimran, MD, Phone: 972-2-655-5673, Email: zimran@md.huji.ac.il
Ari Zimran, MD, Principal Investigator

Sala de Hematologia, Hospital Universitario Miguel Servet, Zaragoza 50009, Spain; Recruiting
Pilar Giraldo, MD, Phone: +34-976765546, Email: pgiraldo@salud.aragon.es
Pilar Giraldo, MD, Principal Investigator

Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust, Cambridge, United Kingdom; Recruiting
Tim Cox, MD, Email: tmc12@medschl.cam.ac.uk
Tim Cox, MD, Principal Investigator

Royal Free Hospital, London NW3 2QG, United Kingdom; Recruiting
Atul Mehta, MD, Phone: +44-2078302814, Email: atul.mehta@royalfree.nhs.uk
Atul Mehta, MD, Principal Investigator

University Research Foundation for Lysosomal Storage Diseases, Inc., Coral Springs, Florida 33065, United States; Recruiting
Neal J Weinreb, MD, Phone: 954-755-1904, Email: boneal@winning.com
Neal J Weinreb, MD, Principal Investigator

Department of Human Genetics, Emory University School of Medicine, Decatur, Georgia 30033, United States; Recruiting
Paul M Fernhoff, MD, Phone: 404-778-8500, Email: pfernhoff@genetics.emory.edu
Paul M Fernhoff, MD, Principal Investigator

Neurogenetics, NYU at Rivergate, New York, New York 10016, United States; Recruiting
Gregory M Pastores, MD, Phone: 212-263-8344, Email: gregory.pastores@med.nyu.edu
Michele Ford, Phone: (212) 263-6981, Email: michele.ford@nyumc.org
Gregory M Pastores, MD, Principal Investigator

Mount Sinai Hospital, Toronto, Ontario M5G 1X5, Canada; Recruiting
Dominick Amato, MD, Phone: (416) 586-4800 Ext 5189, Email: damato@mtsinai.on.ca
Dominick Amato, MD, Principal Investigator

Bone Marrow Transplant Service, The Royal Melbourne Hospital, Parkville, Victoria, Australia; Recruiting
Jeffrey Szer, MD, Email: jeff.szer@mh.org.au
Jeffrey Szer, MD, Principal Investigator

Starting date: December 2008
Last updated: July 15, 2010