Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

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

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.

Study Overview

• Status: Completed
• Conditions: Gaucher Disease
• Intervention / Treatment: Drug: Taliglucerase alfa

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.

• Study Type: Interventional
• Enrollment (Actual): 31
• Phase: Phase 3

Contacts and Locations

Study Locations

• Australia
  • Victoria
    • Parkville, Victoria, Australia
      • Bone Marrow Transplant Service, The Royal Melbourne Hospital
• Canada
  • Ontario
    • Toronto, Ontario, Canada, M5G 1X5
      • Mount Sinai Hospital
• Israel
  • Haifa, Israel, 31096
    • Rambam Medical Center
  • Jerusalem, Israel
    • Shaare Zedek Medical Center
• Spain
  • Zaragoza, Spain, 50009
    • Sala de Hematologia, Hospital Universitario Miguel Servet
• United Kingdom
  • Cambridge, United Kingdom
    • Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust
  • London, United Kingdom, Nw3 2QG
    • Royal Free Hospital
• United States
  • Florida
    • Coral Springs, Florida, United States, 33065
      • University Research Foundation for Lysosomal Storage Diseases, Inc.
  • Georgia
    • Decatur, Georgia, United States, 30033
      • Department of Human Genetics, Emory University School of Medicine
  • New York
    • New York, New York, United States, 10016
      • Neurogenetics, NYU at Rivergate

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 2 years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Taliglucerase alfa; Open label taliglucerase alfa treatment	Drug: Taliglucerase alfa; Intravenous infusion every 2 weeks; Other Names: Plant cell expressed recombinant glucocerebrosidase (prGCD)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Hemoglobin	Every 3 months from Baseline to Month 9

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Platelet Count		Every 3 months from Baseline to Month 9
Spleen Volume	Spleen volume measured by MRI in mL	Baseline and 9 Months
Liver Volume	Liver volume measured by MRI	Baseline and 9 months

Collaborators and Investigators

• Sponsor: Pfizer

Publications and helpful links

General Publications

• 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.

Study record dates

Study Major Dates

• Study Start: December 1, 2008
• Primary Completion (Actual): April 1, 2013
• Study Completion (Actual): May 1, 2013

Study Registration Dates

• First Submitted: July 7, 2008
• First Submitted That Met QC Criteria: July 7, 2008
• First Posted (Estimate): July 10, 2008

Study Record Updates

• Last Update Posted (Actual): October 4, 2018
• Last Update Submitted That Met QC Criteria: September 5, 2018
• Last Verified: September 1, 2018

More Information

Terms related to this study

• Keywords: enzyme replacement therapy; glucocerebrosidase; Gaucher disease; lysosomal storage disorder; imiglucerase
• Additional Relevant MeSH Terms: Metabolic Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Metabolism, Inborn Errors; Lysosomal Storage Diseases; Lipid Metabolism Disorders; Brain Diseases, Metabolic; Brain Diseases, Metabolic, Inborn; Sphingolipidoses; Lysosomal Storage Diseases, Nervous System; Lipidoses; Lipid Metabolism, Inborn Errors; Gaucher Disease
• Other Study ID Numbers: PB-06-002