Plant Cell Expressed Recombinant Human Glucocerebrosidase Extension Trial

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Protalix
ClinicalTrials.gov Identifier:
NCT00705939
First received: June 25, 2008
Last updated: July 14, 2014
Last verified: July 2014
  Purpose

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: Taliglucerase alfa
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: Parallel 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: Spleen Volume at Baseline and Months 12, 24, and 36 ] [ Designated as safety issue: No ]
    Spleen volume measured by MRI


Secondary Outcome Measures:
  • Liver Volume [ Time Frame: Liver volume at Baseline and Months 12, 24 and 36 ] [ Designated as safety issue: No ]
    Liver volume measured by MRI

  • Hemoglobin [ Time Frame: Hemoglobin at Baseline and Months 12, 24 and 36 ] [ Designated as safety issue: No ]
  • Platelet Count [ Time Frame: Platelet count at Baseline and Months 12, 24 and 36 ] [ Designated as safety issue: No ]

Other Outcome Measures:
  • Spleen Volume Multiples of Normal (MN) [ Time Frame: Baseline and Months 12, 24, and 36 ] [ Designated as safety issue: No ]
    Spleen volume measured by MRI. Normal spleen volume is 2 mL/kg × body weight (kg)

  • Liver Volume Multiples of Normal (MN) [ Time Frame: Baseline and Months 12, 24 and 36 ] [ Designated as safety issue: No ]
    Liver volume measured by MRI. Normal liver volume is 25 mL/kg × body weight (kg).


Enrollment: 45
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: Naive 30 Units/kg
Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)
Drug: Taliglucerase alfa
Intravenous infusion every 2 weeks
Other Names:
  • Plant Cell Expressed Recombinant Human Glucocerebrosidase
  • prGCD
Experimental: Naive 60 Units/kg
Continue taliglucerase alfa treatment from PB-06-001 (NCT00376168)
Drug: Taliglucerase alfa
Intravenous infusion every 2 weeks
Other Names:
  • Plant Cell Expressed Recombinant Human Glucocerebrosidase
  • prGCD
Experimental: Switchover
Continue taliglucerase alfa treatment from PB-06-002 (NCT00712348)
Drug: Taliglucerase alfa
Intravenous infusion every 2 weeks
Other Names:
  • Plant Cell Expressed Recombinant Human Glucocerebrosidase
  • 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.

  Eligibility

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

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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00705939

Locations
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
Chile
Pontificia Universidad Catolica de Chile
Santiago, Chile
Israel
Rambam Medical Center
Haifa, Israel, 31096
Shaare Zedek Medical Center
Jerusalem, Israel
South Africa
Morningside Medi-Clinic
Morningside, South Africa, 2196
Spain
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, Israel
  More Information

No publications provided

Responsible Party: Protalix
ClinicalTrials.gov Identifier: NCT00705939     History of Changes
Other Study ID Numbers: PB-06-003
Study First Received: June 25, 2008
Results First Received: April 30, 2014
Last Updated: July 14, 2014
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
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 July 23, 2014