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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.
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.
Age
2 - No limit years
Sex
ALL
Healthy Volunteers
No
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, United States
Bone Marrow Transplant Service, The Royal Melbourne Hospital
Parkville, Victoria, Australia
Mount Sinai Hospital
Toronto, Ontario, Canada
Rambam Medical Center
Haifa, Israel
Shaare Zedek Medical Center
Jerusalem, Israel
Sala de Hematologia, Hospital Universitario Miguel Servet
Zaragoza, Spain
Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust
Cambridge, United Kingdom
Royal Free Hospital
London, United Kingdom
Start Date
December 1, 2008
Primary Completion Date
April 1, 2013
Completion Date
May 1, 2013
Last Updated
October 4, 2018
31
ACTUAL participants
Taliglucerase alfa
DRUG
Lead Sponsor
Pfizer
NCT05992532
NCT06573723
Data Source & Attribution
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