*Treatment-experienced: patients previously treated with
imiglucerase for a minimum of 30 consecutive months prior to switching to VPRIV2
†Patients aged ≥4 years currently being treated on a stable dose of imiglucerase for type 1 Gaucher
disease may be switched to VPRIV at the last imiglucerase dose administered 2 weeks prior1
12-MONTH SWITCH: STUDY 034
STUDY 034 DESIGN (N=41)2
(no washout)
15–60 U/KG EOW (n=40)
PRIMARY OBJECTIVE
To evaluate the safety of every-other-week dosing of VPRIV in patients with type 1 Gaucher disease who were previously treated with imiglucerase.2
SECONDARY OBJECTIVES
Absolute change from baseline to 12 months in hemoglobin concentration; and percentage change from baseline to 12 months in platelet count, and spleen and liver volumes normalized by body weight.2
Over 12 months, stability vs. baseline was maintained in all four clinical parameters.2
HEMOGLOBIN CONCENTRATION
- Median hemoglobin concentration at baseline: 13.8 g/dL2
- Mean change at 12 months: −0.1 g/dL (within the pre-defined efficacy criterion of ±1 g/dL)2
PLATELET COUNT
- Median platelet count at baseline: 162 × 109/L2
- Mean change at 12 months: +7.0% (within the pre-defined efficacy criterion of ±20%)2
SPLEEN VOLUME
- Median spleen volume at baseline: 2.5 MN2
- Mean change at 12 months: −5.6% (within the pre-defined efficacy criterion of ±15%)2
LIVER VOLUME
- Median liver volume at baseline: 0.8 MN2
- Mean change at 12 months: 0.0% (within the pre-defined efficacy criterion of ±15%)2
MEAN CHANGE FROM BASELINE IN
HEMOGLOBIN CONCENTRATION2
12-month data – median baseline: 13.8 g/dL; mean change from baseline:  −0.1 g/dL (within the pre-defined efficacy criterion of ±1 g/dL)2
MEAN % CHANGE FROM BASELINE IN
PLATELET COUNT2
12-month data – median baseline: 162 × 109/L; mean change from baseline: +7.0% (within the pre-defined efficacy criterion of ±20%)2
MEAN % CHANGE FROM BASELINE IN
SPLEEN VOLUME2
12-month data – median baseline: 2.5 MN; mean change from baseline: –5.6% (within the pre-defined efficacy criterion of ±15%)2
MEAN % CHANGE FROM BASELINE IN
LIVER VOLUME2
12-month data – median baseline: 0.8 MN; mean change from baseline: 0.0% (within the pre-defined efficacy criterion of ±15%)2