Table 1 Patient disposition in the taliglucerase alfa clinical studiesa
Patient population and study | Enrolled | Completed | Notes |
|---|---|---|---|
Adult treatment-naive patients | |||
PB-06-001 [15] | 33 | 29 | • 1 patient withdrew consent before first dose • 3 patients discontinued |
PB-06-003 [18] | 26 | 23 | • 3 patients not recruited to PB-06-003 • 3 patients discontinued |
PB-06-007 [21] | 19 | 17 | • 4 patients not recruited into PB-06-007 • 2 patients discontinued |
Adult treatment-switched patients | |||
PB-06-002 [16] | 26 | 25 | • 1 patient discontinued |
PB-06-003 [19] | 19 | 10 | • 6 patients from PB-06-002 not recruited into PB-06-003, 5 of whom continued with compassionate use program • 1 patient non-compliant • 4 patients discontinued • 4 patients treated for 30–33 total months but transitioned to commercial use before study completion |
Paediatric treatment-naïve patients | |||
PB-06-005 [17] | 11 | 11 | |
PB-06-006 [20] | 10 | 9 | • 1 patient from PB-06-005 not recruited into PB-06-006 and continued with compassionate use program • 1 patient dropped out during PB-06-006 |
Paediatric treatment-switched patients | |||
PB-06-002 [16] | 5 | 5 | |
PB-06-006 [20] | 5 | 4 | • 2 patients completed 27 months of treatment and continued with compassionate use program • 2 patients completed 33 months of treatment • 1 patient lost to follow-up |