Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

This study has been completed.
Information provided by (Responsible Party):
Protalix Identifier:
First received: June 25, 2008
Last updated: August 13, 2013
Last verified: August 2013

Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the accumulation of substrate glucocerebroside (GlcCer) in the cells of the monocyte-macrophage system.

This is an extension trial to Study NCT00376168 and NCT00712348.

Condition Intervention Phase
Gaucher Disease
Drug: Plant Cell Expressed Recombinant Human Glucocerebrosidase
Phase 3

Access to an investigational treatment associated with this study is no longer available outside the clinical trial.   More info ...

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Multicenter, Double-Blind, Extension Trial of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patients With Gaucher Disease

Resource links provided by NLM:

Further study details as provided by Protalix:

Primary Outcome Measures:
  • Spleen Volume [ Time Frame: 3, 9, and 15 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Liver Volume [ Time Frame: 3, 9, and 15 months ] [ Designated as safety issue: No ]

Enrollment: 44
Study Start Date: June 2008
Study Completion Date: August 2013
Primary Completion Date: May 2012 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1
Continue treatment from previous study
Drug: Plant Cell Expressed Recombinant Human Glucocerebrosidase
Intravenous infusion every 2 weeks
Other Name: prGCD

Detailed Description:

This will be a multi-center, double-blind, parallel group, extension trial to assess the safety and efficacy of prGCD in patients completing NCT00376168. Patients will receive IV infusion of prGCD every two weeks at the selected medical center. The duration of the extension study will be fifteen months. There will be two treatment groups: 30 units/kg every 2 weeks or 60 units/kg every 2 weeks.


Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Successful completion of Protocol PB-06-001
  • The patient signs informed consent

Exclusion Criteria:

  • Currently taking another experimental drug for any condition
  • Presence of severe neurological signs and symptoms, defined as complete ocular paralysis, overt myoclonus or history of seizures, characteristic of neuronopathic Gaucher disease
  • Pregnant or nursing
  • 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 identifier: NCT00705939

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
Pontificia Universidad Catolica de Chile
Santiago, Chile
Rambam Medical Center
Haifa, Israel, 31096
Shaare Zedek Medical Center
Jerusalem, Israel
South Africa
Morningside Medi-Clinic
Morningside, South Africa, 2196
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
Principal Investigator: Ari Zimran, MD Shaare Zedek Medical Center, Jerusalem, Israel
  More Information

No publications provided

Responsible Party: Protalix Identifier: NCT00705939     History of Changes
Other Study ID Numbers: PB-06-003
Study First Received: June 25, 2008
Last Updated: August 13, 2013
Health Authority: United States: Food and Drug Administration
Israel: Ministry of Health

Keywords provided by Protalix:
Gaucher Disease
Enzyme replacement therapy

Additional relevant MeSH terms:
Gaucher Disease
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
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders processed this record on April 22, 2014