PK parameters following the first dose and following 3- to 6-months of treatment (repeat PK in Part 3)
| . | Mean (CV %) (N = 64) . | ||
|---|---|---|---|
| Parameter (unit) . | Initial PK total (N = 64) . | Repeat PK (N = 30) . | |
| Initial . | Repeat . | ||
| IR,*† (IU/dL)/(IU/kg) | 1.85 (21.8) | 1.90 (21.0) | 1.99 (17.7) |
| Cmax,*† IU/dL | 99.9 (19.9) | 103 (19.3) | 108 (17.2) |
| AUCt, IU·h/dL | 1780 (34.5) | 1783 (33.3) | 1850 (33.0) |
| AUCinf, IU·h/dL | 1830 (34.9) | 1840 (33.9) | 1880 (34.5) |
| CL, mL/kg, h | 3.15 (38.2) | 3.13 (32.6) | 3.05 (36.0) |
| Vss, mL/kg | 59.5 (23.9) | 60.3 (22.2) | 53.1 (16.4) |
| t1/2, h | 14.1 (27.1) | 14.2 (29.0) | 12.9 (29.4) |
| MRT, h | 20.3 (26.4) | 20.2 (27.8) | 18.9 (28.5) |
| . | Mean (CV %) (N = 64) . | ||
|---|---|---|---|
| Parameter (unit) . | Initial PK total (N = 64) . | Repeat PK (N = 30) . | |
| Initial . | Repeat . | ||
| IR,*† (IU/dL)/(IU/kg) | 1.85 (21.8) | 1.90 (21.0) | 1.99 (17.7) |
| Cmax,*† IU/dL | 99.9 (19.9) | 103 (19.3) | 108 (17.2) |
| AUCt, IU·h/dL | 1780 (34.5) | 1783 (33.3) | 1850 (33.0) |
| AUCinf, IU·h/dL | 1830 (34.9) | 1840 (33.9) | 1880 (34.5) |
| CL, mL/kg, h | 3.15 (38.2) | 3.13 (32.6) | 3.05 (36.0) |
| Vss, mL/kg | 59.5 (23.9) | 60.3 (22.2) | 53.1 (16.4) |
| t1/2, h | 14.1 (27.1) | 14.2 (29.0) | 12.9 (29.4) |
| MRT, h | 20.3 (26.4) | 20.2 (27.8) | 18.9 (28.5) |
Median (range) age of the 64 subjects included in the PK analysis is 26 (12-58) years. The CV represents the percentage of variability in each parameter and is calculated as the SD divided by the mean.
AUCinf, area under the curve extrapolated to infinity; AUCt, area under the curve to the last sample with quantifiable drug concentration; CL, clearance; CV, coefficient of variation; MRT, mean residence time; t1/2, terminal half-life; Vss, volume of distribution at steady state.
For IR and Cmax, the total number of participants with available pre-dose–corrected measurements was N = 63 at the initial PK assessment, and N = 29 at the repeat PK assessment.
For IR and Cmax, pre-dose correction was performed by subtracting each participant’s FVIII activity level before dosing from the activity level obtained at each time point after dosing. All other parameters are pre-dose uncorrected.