Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease

• Mean Change From Baseline to Month 9 in Hemoglobin (Hgb) Concentration for Each Treatment Group. [ Time Frame: Baseline to Month 9 ] [ Designated as safety issue: No ]
  

• Change From Baseline to Month 9 in Platelet Counts for Each Treatment Group. [ Time Frame: Baseline to Month 9 ] [ Designated as safety issue: No ]
  Values shown are observed change from Baseline to Month 9.
  
  
• Change From Baseline to Month 9 in Normalized Liver Volume (Percent (%) Body Weight) for Each Treatment Group. [ Time Frame: Baseline to Month 9 ] [ Designated as safety issue: No ]
  Values shown are observed change from Baseline to Month 9. Measured by Magnetic resonance imaging (MRI). Liver volume has been normalized for percent (%) body weight for each treatment arm. Liver size relative to body weight = (Liver volume [cc]/Body weight [kg]*1000.
  
  
• Change From Baseline to Month 9 in Normalized Spleen Volume (Percent (%) Body Weight) for Each Treatment Group. [ Time Frame: Baseline to Month 9 ] [ Designated as safety issue: No ]
  Values shown are observed change from Baseline to month 9. Measured by Magnetic resonance imaging (MRI). Spleen volume was normalized for percent (%) of body weight for each treatment arm. Spleen size relative to body weight=(Spleen volume [cc]/Body weight [kg])*100. Ten patients in each treatment group were splenectomized and therefore excluded from the analysis.
  
  
• Change From Baseline to Month 9 in Plasma Chitotriosidase for Each Treatment Group. [ Time Frame: Baseline to Month 9. ] [ Designated as safety issue: No ]
  Values shown are observed change from Baseline to Month 9. Chitotriosidase levels were measured in 10 patients in the velaglucerase alfa group and 11 patients in the imiglucerase group. Units of measure is defined as nanomole per milliliter per hour.
  
  
• Change From Baseline to Month 9 in Plasma Chemokine (C-C Motif) Ligand 18 (CCL18) for Each Treatment Group. [ Time Frame: Baseline to Month 9 ] [ Designated as safety issue: No ]
  Values shown are observed change from Baseline to Month 9.
  
  
• Number of Patients Who Developed Antibody for Each Treatment Group. [ Time Frame: Baseline to Month 9 ] [ Designated as safety issue: Yes ]
  Measure type is actual number of patients who developed antibodies to treatment; GA-GCB or imiglucerase. Antibody detection was based upon serum samples collected at various time points throughout the study. Serum samples were screened using an enzyme-linked immunosorbent assay (ELISA) and positive antibody confirmation was determined using a radioimmunoprecipitation assay (RIP);positive samples were also tested for enzyme neutralizing activity. Patient samples were compared to internal assay controls (positive/negative), positive samples were determined based upon individual assay criteria.
  
  
• Time to Response- Comparison of GA-GCB and Imiglucerase on the Earliest Time to Respond as Assesed Via Hemoglobin Concentration [ Time Frame: Response rate at Month 9 compared to Baseline ] [ Designated as safety issue: No ]
  Time to response was defined as a ≥ 1 g/dL improvement in hemoglobin levels relative to Baseline. Units (%) correlates to the percentage of patients who had a change of ≥ 1 g/dL improvement in hemoglobin levels relative to Baseline during their participation in the study.
  
  

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 (velaglucerase alfa) 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 efficacy and safety of GA-GCB (velaglucerase alfa) in comparison to imiglucerase in men, women, and children with Type 1 Gaucher disease.