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

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:

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

MedlinePlus related topics: Gaucher's Disease

Drug Information available for: Taliglucerase alfa Imiglucerase

Genetic and Rare Diseases Information Center resources: Gaucher Disease Sphingolipidosis

U.S. FDA Resources

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 by Protalix

Additional publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Pastores GM, Petakov M, Giraldo P, Rosenbaum H, Szer J, Deegan PB, Amato DJ, Mengel E, Tan ES, Chertkoff R, Brill-Almon E, Zimran A. A Phase 3, multicenter, open-label, switchover trial to assess the safety and efficacy of taliglucerase alfa, a plant cell-expressed recombinant human glucocerebrosidase, in adult and pediatric patients with Gaucher disease previously treated with imiglucerase. Blood Cells Mol Dis. 2014 Dec;53(4):253-60. doi: 10.1016/j.bcmd.2014.05.004. Epub 2014 Jun 18.


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 Brain Diseases Brain Diseases, Metabolic Brain Diseases, Metabolic, Inborn Central Nervous System Diseases Genetic Diseases, Inborn Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors	Lipidoses Lysosomal Storage Diseases Lysosomal Storage Diseases, Nervous System Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Sphingolipidoses

ClinicalTrials.gov processed this record on February 27, 2015