Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase - Full Text View

Sponsor:	Protalix
Information provided by (Responsible Party):	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:	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

Resource links provided by NLM:

Genetics Home Reference related topics: Chanarin-Dorfman syndrome cholesteryl ester storage disease Farber lipogranulomatosis Gaucher disease Schindler disease succinic semialdehyde dehydrogenase deficiency

MedlinePlus related topics: Gaucher's Disease

Drug Information available for: Alglucerase Imiglucerase

U.S. FDA Resources

Further study details as provided by Protalix:

Primary Outcome Measures:

Adverse events [ Time Frame: Continuous ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:

Platelet count [ Time Frame: Monthly ] [ Designated as safety issue: Yes ]

Estimated Enrollment:	30
Study Start Date:	December 2008
Estimated Study Completion Date:	October 2011
Primary Completion Date:	July 2011 (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
Genders Eligible for Study:	Both
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

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

No publications provided

Responsible Party:	Protalix
ClinicalTrials.gov Identifier:	NCT00712348 History of Changes
Other Study ID Numbers:	PB-06-002
Study First Received:	July 7, 2008
Last Updated:	August 22, 2011
Health Authority:	United States: Food and Drug Administration; Israel: Ministry of Health

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 February 13, 2012