Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Protalix
ClinicalTrials.gov Identifier:
NCT00712348
First received: July 7, 2008
Last updated: May 30, 2014
Last verified: May 2014
  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®) enzyme replacement therapy.


Condition Intervention Phase
Gaucher Disease
Drug: Taliglucerase alfa
Phase 3

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:


Further study details as provided by Protalix:

Primary Outcome Measures:
  • Hemoglobin [ Time Frame: Every 3 months from Baseline to Month 9 ] [ Designated as safety issue: No ]

Other Outcome Measures:
  • Platelet Count [ Time Frame: Every 3 months from Baseline to Month 9 ] [ Designated as safety issue: No ]
  • Spleen Volume [ Time Frame: Baseline and 9 Months ] [ Designated as safety issue: No ]
    Spleen volume measured by MRI in mL

  • Liver Volume [ Time Frame: Baseline and 9 months ] [ Designated as safety issue: No ]
    Liver volume measured by MRI


Enrollment: 31
Study Start Date: December 2008
Study Completion Date: May 2013
Primary Completion Date: April 2013 (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
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: 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
Results First Received: April 30, 2014
Last Updated: May 30, 2014
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 July 23, 2014