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

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 prGCD in 15 patients with Gaucher disease who are currently being treated with imiglucerase (Cerezyme®) ERT.

Condition	Intervention	Phase
Gaucher Disease	Drug: plant expressed recombinant human glucocerebrosidase (prGCD)	Phase III

Study Type:	Interventional
Study Design:	Treatment, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
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 primary carnitine deficiency 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:	15
Study Start Date:	December 2008
Estimated Study Completion Date:	February 2010
Estimated Primary Completion Date:	February 2010 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
prGCD: Experimental Open label prGCD treatment	Drug: plant expressed recombinant human glucocerebrosidase (prGCD) Intravenous infusion every 2 weeks

Detailed Description:

This is a multi-center, open-label, switchover trial to assess the safety of prGCD in 15 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 prGCD every two weeks for a total of 20 IV infusions. The dose of prGCD will be equal to each patient's previous imiglucerase dose. The infusions will be administered at the selected investigational site (clinic/hospital) 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:	18 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Males and females, 18 years or older
Female patients of child-bearing potential and male patients with female partners of child-bearing potential must agree to use a medically acceptable method of contraception, not including the rhythm method
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 unchanged) for at least the last six months
Able to provide written informed consent

Exclusion Criteria:

Currently taking another experimental drug for any condition
Pregnant or nursing or planning to become pregnant
History of allergy to carrots
Presence of anti-glucocerebrosidase (GCD) antibodies
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 prGCD
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

Contacts

Contact: Raul Chertkoff, MD

+972-4-9889488

raul@protalix.com

Locations

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
Israel
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

Sponsors and Collaborators

Protalix

Investigators

Principal Investigator:

Ari Zimran, MD

Shaare Zedek Medical Center, Jerusalem

More Information

No publications provided

Responsible Party:	Protalix Biotherapeutics ( Einat Almon )
Study ID Numbers:	PB-06-002
Study First Received:	July 7, 2008
Last Updated:	August 19, 2009
ClinicalTrials.gov Identifier:	NCT00712348 History of Changes
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:

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

ClinicalTrials.gov processed this record on November 11, 2009