Clinical trial NCT00712348: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Gaucher Disease
Keywords: Gaucher disease
imiglucerase
glucocerebrosidase
enzyme replacement therapy
lysosomal storage disorder
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2008-07-10
Enrollment: 31
Sex: ALL
Minimum age (years): 2
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Hemoglobin
timeFrame: Every 3 months from Baseline to Month 9
Secondary outcomes:
Other outcomes:
measure: Platelet Count
timeFrame: Every 3 months from Baseline to Month 9
measure: Spleen Volume
timeFrame: Baseline and 9 Months
measure: Liver Volume
timeFrame: Baseline and 9 months
Locations:
city: Coral Springs
country: United States
facility: University Research Foundation for Lysosomal Storage Diseases, Inc.
state: Florida
city: Decatur
country: United States
facility: Department of Human Genetics, Emory University School of Medicine
state: Georgia
city: New York
country: United States
facility: Neurogenetics, NYU at Rivergate
state: New York
city: Parkville
country: Australia
facility: Bone Marrow Transplant Service, The Royal Melbourne Hospital
state: Victoria
city: Toronto
country: Canada
facility: Mount Sinai Hospital
state: Ontario
city: Haifa
country: Israel
facility: Rambam Medical Center
city: Jerusalem
country: Israel
facility: Shaare Zedek Medical Center
city: Zaragoza
country: Spain
facility: Sala de Hematologia, Hospital Universitario Miguel Servet
city: Cambridge
country: United Kingdom
facility: Lysosomal Disorders Service, Addenbrookes Hospital NHS Trust
city: London
country: United Kingdom
facility: Royal Free Hospital
Study officials:
References:
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.