The purpose of this study is to evaluate the long-term safety of every other week dosing of Gene-Activated® human glucocerebrosidase (GA-GCB, velaglucerase alfa) intravenously in patients with type 1 Gaucher disease.
Type 1 Gaucher disease, the most common form,accounts for more than 90% of all cases and does not involve the CNS. Typical manifestations of type 1 Gaucher disease include hepatomegaly, splenomegaly, thrombocytopenia, bleeding tendencies, anemia, hypermetabolism, skeletal pathology, growth retardation, pulmonary disease, and decreased quality of life. Gene-Activated® human glucocerebrosidase (GA-GCB,velaglucerase alfa) is produced in a continuous human cell line using proprietary gene-activation technology and has an identical amino acid sequence to the naturally occurring human enzyme. GA-GCB contains terminal mannose residues that target the enzyme to the macrophages-the primary target cells in Gaucher disease. This study was designed to determine the long-term safety of GA-GCB in men, women, and children with Type 1 Gaucher disease.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
95
Intravenous infusion, every other week (EOW)
Los Angeles Medical Center
Los Angeles, California, United States
Overall Summary of Treatment Emergent Adverse Events
Safety was evaluated by an analysis of adverse events (AEs), concomitant medication use, clinical laboratory tests, vital signs during the infusion of study drug, physical examination, and the development of anti-velaglucerase alfa. No formal comparisons or statistical tests were applied for the safety analyses, including for differences between the groups.
Time frame: Baseline to termination of study
Change From Baseline to 24 Months in Hemoglobin Concentration for Each Treatment Group
Time frame: Baseline to 24 months
Change From Baseline to 24 Months in Platelet Counts for Each Treatment Group
Time frame: Baseline to 24 months
Change From Baseline to 24 Months in Normalized Liver Volume for Each Treatment Group
Time frame: Baseline to 24 months
Percentage Change From Baseline to 24 Months in Normalized Spleen Volume for Each Treatment Group
Time frame: Baseline to 24 months
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