Switchover Trial From Imiglucerase to Plant Cell Expressed Recombinant Human Glucocerebrosidase

Phase 3

Completed

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.

Recruitment & 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

Arm && Interventions

Group	Intervention	Description
Taliglucerase alfa	Taliglucerase alfa	Open label taliglucerase alfa treatment

Primary Outcome Measures

Name	Time	Method
Hemoglobin	Every 3 months from Baseline to Month 9

Secondary Outcome Measures

Name	Time	Method

Locations (10): Shaare Zedek Medical Center
🇮🇱
Jerusalem, Israel
Royal Free Hospital
🇬🇧
London, United Kingdom
Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust
🇬🇧
Cambridge, United Kingdom
Sala de Hematologia, Hospital Universitario Miguel Servet
🇪🇸
Zaragoza, Spain
Rambam Medical Center
🇮🇱
Haifa, Israel
University Research Foundation for Lysosomal Storage Diseases, Inc.
🇺🇸
Coral Springs, Florida, 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
Department of Human Genetics, Emory University School of Medicine
🇺🇸
Decatur, Georgia, United States