Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

Gaucher Disease Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00712348
• Other study ID #: PB-06-002
• Status: Completed
• Phase: Phase 3
• Start date: December 2008
• Est. completion date: May 2013

Study information

• Verified date: September 2018
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 31
• Est. completion date: May 2013
• Est. primary completion date: April 2013
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Gaucher Disease

Intervention

Drug:
• Taliglucerase alfa
Intravenous infusion every 2 weeks

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Countries where clinical trial is conducted

United States,  Australia,  Canada,  Israel,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	Platelet Count		Every 3 months from Baseline to Month 9
Other	Spleen Volume	Spleen volume measured by MRI in mL	Baseline and 9 Months
Other	Liver Volume	Liver volume measured by MRI	Baseline and 9 months
Primary	Hemoglobin		Every 3 months from Baseline to Month 9