Study 032: 12-Month Parallel-Dose Study (n=25)2
Study 032: 12-Month Parallel-Dose StudY Objectives2
R
(n=25)
EOW (n=13)
EOW (n=12)
-
Primary: Change in hemoglobin concentration from baseline to 12 months in the 60 U/kg treatment group.2
-
Secondary: Included changes from baseline to 12 months in hemoglobin concentration in the 45 U/kg treatment group; changes from baseline to 12 months in platelet count, and spleen and liver volumes for both treatment groups.2
At 12 months, clinically relevant differences from baseline were observed for the primary endpoint. Differences from baseline were observed for secondary endpoints.*2
MEAN CHANGE (%) FROM BASELINE2
12 months VPRIV 60 U/kg EOW
Increasing platelet and
hemoglobin count
Decreasing spleen and
liver volume
23%
n=12
66%
n=12
-50%
n=12
-17%
n=12
*This decrease was not
statistically significant
after adjusting for
multiple tests
*This decrease was not statistically significant after adjusting for multiple tests
100
50
0
-50
-100
Primary endpoint
Hemoglobin concentration
-
Mean hemoglobin concentration at baseline: 10.6 g/dL1
-
Mean change at 12 months:
2.4 g/dL ± 0.3 (SE)1,2
Primary endpoint
Secondary endpoints
Platelet count
-
Mean platelet count at baseline: 97 × 109/L1
-
Mean change at 12 months:
51 × 109/L ± 12 (SE)1,2
Spleen volume
-
Mean spleen volume at baseline: 2.9% of body weight1
-
Mean change at 12 months:
−1.9% ± 0.5 of body weight1,2
Liver volume
-
Mean liver volume at baseline: 3.6% of body weight1
-
Mean change at 12 months: −0.84% ± 0.33 of body weight1,2
Secondary endpoints
EOW, every other week; R, randomized; SE, standard error.
Study 039: 9-Month Head-to-Head
(Non-inferiority) Study (n=34)2
Study 039: 9-MONTH HEAD-TO-HEAD (NON-INFERIORITY) STUDY OBJECTIVES2
R
(n=34)
EOW (n=17)
60 U/kg EOW
(n=17)
-
Primary: Difference in the mean change from baseline to month 9 in hemoglobin concentration between the two groups.2
-
Secondary: Included differences in changes from baseline between treatment groups in mean platelet count, and spleen and liver volumes.2
VPRIV demonstrated non-inferiority to imiglucerase.2
MEAN CHANGE (%) FROM BASELINE3*
9 months imiglucerase 60 U/kg EOW
9 months VPRIV 60 U/kg EOW
Increasing platelet and
hemoglobin count
Decreasing spleen and
liver volume
14%
n=17
77%
n=17
-50%
n=7
-25%
n=17
100
50
0
-50
-100
Primary endpoint
Hemoglobin concentration
-
Mean hemoglobin concentration at baseline:* VPRIV, 11.0 g/dL; imiglucerase, 10.6 g/dL1,2
-
Mean treatment difference (VPRIV to imiglucerase) at 9 months: 0.1 g/dL ± 0.4 (SE)1,2
Primary endpoint
Secondary endpoints
Platelet count
-
Mean platelet count at baseline:* VPRIV, 171 × 109/L; imiglucerase, 188.0 × 109/L1,2
-
Mean treatment difference (VPRIV to imiglucerase) at 9 months: −39 × 109/L2
Spleen volume
-
Mean spleen volume at baseline:* VPRIV, 3.4% of body weight; imiglucerase, 1.4% of body weight1,2
-
Mean treatment difference (VPRIV to imiglucerase) at 9 months: 0.1% of body weight2
Liver volume
-
Mean liver volume at baseline:* VPRIV, 4.3% of body weight; imiglucerase, 4.0% of body weight1,2
-
Mean treatment difference (VPRIV to imiglucerase) at 9 months: −0.1% of body weight2
Secondary endpoints
*Baseline values are for both treatment groups
EOW, every other week; R, randomized; SE, standard error.
Study 044: 5-Year Long-Term Extension Study (n=57)2
Study 044: 5-Year Long-Term Extension Study OBJECTIVES2
Overall VPRIV
(n=41)
Imiglucerase to
VPRIV (n=16)
60 U/kg EOW (n=57)*
-
Primary: To evaluate the long-term safety of VPRIV treatment.2
-
Secondary: To evaluate the effects of treatment on hemoglobin concentration, platelet count, and liver and spleen volumes.2
*One patient from each group in Study 039 did not participate in Study 044
At 24 months of VPRIV treatment, patients demonstrated improvements from baseline in clinical parameters. Improvements were maintained for up to 5 years.1,2
MEAN INCREASE IN HEMOGLOBIN CONCENTRATION2
12-month data – baseline:† 10.6 g/dL; mean change from baseline: 2.4 g/dL ± 0.3 (SE)1,3
24-month data – baseline:† 11.0 g/dL; mean change from baseline: 2.8 g/dL2,4
MEAN INCREASE IN PLATELET COUNT2
12-month data – baseline:† 97 × 109/L; mean change from baseline: 51 × 109/L ± 12 (SE) 1,3
24-month data – baseline:† 108.6 × 109/L; mean change from baseline: 87.9 × 109/L 2,4
MEAN DECREASE IN SPLEEN VOLUME2
12-month data – baseline:† 2.9% of body weight; mean change from baseline: −1.9% ± 0.5 of body weight (SE) 1,3
24-month data – baseline:† 3.8% of body weight; mean change from baseline: −2.7% of body weight2,4
MEAN DECREASE IN LIVER VOLUME2
12-month data – baseline:† 3.6% of body weight; mean change from baseline: −0.84% ± 0.33 of body weight (SE)1,3‡
‡The decrease was not statistically significant after adjusting for multiple tests
24-month data – baseline:† 4.0% of body weight; mean change from baseline −1.2% of body weight2,4
†Baseline is defined as before the first dose of VPRIV in Studies 032 and 039
EOW, every other week; SE, standard error.
