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Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

Primary Purpose

Gaucher Disease

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Taliglucerase alfa
Sponsored by
Pfizer
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Gaucher Disease focused on measuring Gaucher disease, imiglucerase, glucocerebrosidase, enzyme replacement therapy, lysosomal storage disorder

Eligibility Criteria

2 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • University Research Foundation for Lysosomal Storage Diseases, Inc.
  • Department of Human Genetics, Emory University School of Medicine
  • Neurogenetics, NYU at Rivergate
  • Bone Marrow Transplant Service, The Royal Melbourne Hospital
  • Mount Sinai Hospital
  • Rambam Medical Center
  • Shaare Zedek Medical Center
  • Sala de Hematologia, Hospital Universitario Miguel Servet
  • Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust
  • Royal Free Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Taliglucerase alfa

Arm Description

Open label taliglucerase alfa treatment

Outcomes

Primary Outcome Measures

Hemoglobin

Secondary Outcome Measures

Full Information

First Posted
July 7, 2008
Last Updated
September 5, 2018
Sponsor
Pfizer
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1. Study Identification

Unique Protocol Identification Number
NCT00712348
Brief Title
Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase
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
Study Type
Interventional

2. Study Status

Record Verification Date
September 2018
Overall Recruitment Status
Completed
Study Start Date
December 2008 (undefined)
Primary Completion Date
April 2013 (Actual)
Study Completion Date
May 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Pfizer

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Gaucher Disease
Keywords
Gaucher disease, imiglucerase, glucocerebrosidase, enzyme replacement therapy, lysosomal storage disorder

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
31 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Taliglucerase alfa
Arm Type
Experimental
Arm Description
Open label taliglucerase alfa treatment
Intervention Type
Drug
Intervention Name(s)
Taliglucerase alfa
Other Intervention Name(s)
Plant cell expressed recombinant glucocerebrosidase (prGCD)
Intervention Description
Intravenous infusion every 2 weeks
Primary Outcome Measure Information:
Title
Hemoglobin
Time Frame
Every 3 months from Baseline to Month 9
Other Pre-specified Outcome Measures:
Title
Platelet Count
Time Frame
Every 3 months from Baseline to Month 9
Title
Spleen Volume
Description
Spleen volume measured by MRI in mL
Time Frame
Baseline and 9 Months
Title
Liver Volume
Description
Liver volume measured by MRI
Time Frame
Baseline and 9 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Accepts Healthy Volunteers
No
Eligibility 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
Facility Information:
Facility Name
University Research Foundation for Lysosomal Storage Diseases, Inc.
City
Coral Springs
State/Province
Florida
ZIP/Postal Code
33065
Country
United States
Facility Name
Department of Human Genetics, Emory University School of Medicine
City
Decatur
State/Province
Georgia
ZIP/Postal Code
30033
Country
United States
Facility Name
Neurogenetics, NYU at Rivergate
City
New York
State/Province
New York
ZIP/Postal Code
10016
Country
United States
Facility Name
Bone Marrow Transplant Service, The Royal Melbourne Hospital
City
Parkville
State/Province
Victoria
Country
Australia
Facility Name
Mount Sinai Hospital
City
Toronto
State/Province
Ontario
ZIP/Postal Code
M5G 1X5
Country
Canada
Facility Name
Rambam Medical Center
City
Haifa
ZIP/Postal Code
31096
Country
Israel
Facility Name
Shaare Zedek Medical Center
City
Jerusalem
Country
Israel
Facility Name
Sala de Hematologia, Hospital Universitario Miguel Servet
City
Zaragoza
ZIP/Postal Code
50009
Country
Spain
Facility Name
Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust
City
Cambridge
Country
United Kingdom
Facility Name
Royal Free Hospital
City
London
ZIP/Postal Code
NW3 2QG
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
24950666
Citation
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.
Results Reference
derived

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Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

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