Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

NCT00712348 | Completed Phase 3 Results DMC

• 1 other identifier: PB-06-002
• Study Type: interventional
• Target: 31
• Locations: 6 countries
• Sites: 10

Brief Summary

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.

Conditions

Gaucher Disease

Keywords

Gaucher disease; imiglucerase; glucocerebrosidase; enzyme replacement therapy; lysosomal storage disorder

Outcome Measures

Primary Outcomes (1)

• Hemoglobin

  Every 3 months from Baseline to Month 9

Other Outcomes (3)

• Platelet Count

  Every 3 months from Baseline to Month 9

• Spleen Volume

  Baseline and 9 Months

• Liver Volume

  Baseline and 9 months

Study Arms (1)

Taliglucerase alfa

EXPERIMENTAL

Open label taliglucerase alfa treatment

Drug: Taliglucerase alfa

Interventions

Taliglucerase alfa DRUG

Intravenous infusion every 2 weeks

Also known as: Plant cell expressed recombinant glucocerebrosidase (prGCD)

Taliglucerase alfa

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Pfizer: lead

Study Sites (10)

University Research Foundation for Lysosomal Storage Diseases, Inc.

Coral Springs, Florida, 33065, United States

Location

Department of Human Genetics, Emory University School of Medicine

Decatur, Georgia, 30033, United States

Location

Neurogenetics, NYU at Rivergate

New York, New York, 10016, United States

Location

Bone Marrow Transplant Service, The Royal Melbourne Hospital

Parkville, Victoria, Australia

Location

Mount Sinai Hospital

Toronto, Ontario, M5G 1X5, Canada

Location

Rambam Medical Center

Haifa, 31096, Israel

Location

Shaare Zedek Medical Center

Jerusalem, Israel

Location

Sala de Hematologia, Hospital Universitario Miguel Servet

Zaragoza, 50009, Spain

Location

Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust

Cambridge, United Kingdom

Location

Royal Free Hospital

London, NW3 2QG, United Kingdom

Location

Related Publications (1)

• 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.

  PMID: 24950666 DERIVED

MeSH Terms

Conditions

Gaucher Disease; Lysosomal Storage Diseases

Interventions

taliglucerase alfa

Condition Hierarchy (Ancestors)

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; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Lipidoses; Lipid Metabolism, Inborn Errors; Metabolic Diseases; Nutritional and Metabolic Diseases; Lipid Metabolism Disorders

Results Point of Contact

• Title: VP Medical Affairs
• Organization: Protalix Ltd.

raul@protalix.com

+972-4-9028100

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 7, 2008
• First Posted: July 10, 2008
• Study Start: December 1, 2008
• Primary Completion: April 1, 2013
• Study Completion: May 1, 2013
• Last Updated: October 4, 2018
• Results First Posted: June 5, 2014

Record last verified: 2018-09

Locations

US (3); AU (1); GB (2); ES (1); IL (2); CA (1)