Table 6.
Changes from baseline to Week 53 in mean plasma Gb3
| Parameter | 0.2 mg/kg EOW mean values | 0.2 mg/kg weekly mean values | 0.4 mg/kg weekly mean values | 0.2 mg/kg weekly minus EOW
|
||
|---|---|---|---|---|---|---|
| LSM difference | CI | P-value | ||||
| Plasma Gb3, nmol/mL | ||||||
| BL (n) | 6.1 (20) | 5.7 (19) | 5.3 (5) | −1.02 | 95.005% CI: | Unadjusted |
| Wk 53 (n) | 4.8 (17)a | 3.7 (18)b | 3.2 (5) | −2.13, 0.09 | P=0.0715 | |
| Change | −1.0 | −2.1 | −2.1 | |||
| 95% CI | −2.21, 0.11 | −4.30, 0.04 | −3.63, −0.53 | |||
| Males | ||||||
| BL (n) | 7.2 (14) | 7.7 (10) | 6.8 (2) | N/A | N/A | N/A |
| Wk 53 (n) | 5.5 (11) | 4.1 (10) | 4.0 (2) | |||
| Change | −1.6 | −3.6 | −2.8 | |||
| 95% CI | −3.36, 0.13 | −7.53, 0.32 | −13.33, 7.77 | |||
| Females | ||||||
| BL (n) | 3.5 (6) | 3.3 (9) | 4.3 (3) | N/A | N/A | N/A |
| Wk 53 (n) | 3.5 (6) | 3.1 (8) | 2.7 (3) | |||
| Change | −0.01 | −0.29 | −1.6 | |||
| 95% CI | −0.95, 0.93 | −0.93, 0.34 | −4.75, 1.54 | |||
Notes: Change is calculated based on mean values; all CIs are calculated based on the mean (descriptive or least squares).
a
Three patients discontinued before week 53.
b
One patient discontinued before week 53.
Abbreviations: BL, baseline; CI, confidence interval; Gb3, globotriaosylceramide; EOW, every other week; LSM, least squares mean; Wk, week; N/A, not applicable.