Start All 26 a 1.834 0.409 1.845 Phase I * Aged 13 1.871 0.270 1.822 2-5 Aged 13 a 1.796 0.521 1.916 6-12 Start All 30 b 1.568 0.509 1.586 Phase II * Aged 14 b 1.446 0.457 1.563 2-5 Aged 16 1.676 0.542 1.602 6-12 3 All 55 d 1.698 0.402 1.682 Months Aged 28 1.549 0.330 1.604 2-5 Aged 27 c 1.852 0.418 1.805 6-12 6 All 53 e 1.765 0.464 1.760 months Aged 26 c 1.672 0.396 1.709 2-5 Aged 27 c 1.854 0.512 1.826 6-12 Note: a = data not available for 1 patient; b = data not available for 2 patients; c = data not available for 3 patients; d = data not available for 4 patients; e = data not available for 6 patients; data were not available for 2 patients (6–12 years) at 3 and 6 months due to premature withdrawal from the study; other missing IVR values were not evaluable. * = Phase I includes all patients who underwent PK analysis in Cycles 1 and 2; Phase II specifies recovery at start of open treatment phase and therefore includes only patients who did not undergo PK analysis. Comment: In patients aged 2-12 years (ITT population), based on the CHR assay mean IVR values over time were above 1.5% per IU/kg, and ranged from 1.568% to 1.834% per IU/kg. The mean IVR values over time (ITT population) ranged from 1.446% to
Start Phase I * All 26 a 1.834 0.409 1.845 Aged 2-5 13 1.871 0.270 1.822 Aged 6-12 13 a 1.796 0.521 1.916 Start Phase II * All 30 b 1.568 0.509 1.586 Aged 2-5 14 b 1.446 0.457 1.563 Aged 6-12 16 1.676 0.542 1.602 3 Months All 55 d 1.698 0.402 1.682 Aged 2-5 28 1.549 0.330 1.604 Aged 6-12 27 c 1.852 0.418 1.805 6 months All 53 e 1.765 0.464 1.760 Aged 2-5 26 c 1.672 0.396 1.709 Aged 6-12 27 c 1.854 0.512 1.826 Note: a = data not available for 1 patient; b = data not available for 2 patients; c = data not available for 3 patients; d = data not available for 4 patients; e = data not available for 6 patients; data were not available for 2 patients (6–12 years) at 3 and 6 months due to premature withdrawal from the study; other missing IVR values were not evaluable. * = Phase I includes all patients who underwent PK analysis in Cycles 1 and 2; Phase II specifies recovery at start of open treatment phase and therefore includes only patients who did not undergo PK analysis. Comment: In patients aged 2-12 years (ITT population), based on the CHR assay mean IVR values over time were above 1.5% per IU/kg, and ranged from 1.568% to 1.834% per IU/kg. The mean IVR values over time (ITT population) ranged from 1.446% to
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