Participant disposition, demographics, and baseline clinical characteristics
| Characteristic . | Cohort 1 (50 IU/kg efanesoctocog alfa) . | Cohort 2 (65 IU/kg efanesoctocog alfa) . |
|---|---|---|
| Enrolled, n | 10 | 14 |
| Included in safety analysis* | 10 (100) | 14 (100) |
| Included in pharmacokinetic analysis† | 9 (90) | 14 (100) |
| Completed study‡ | 10 (100) | 14 (100) |
| Age, mean (range), y | 35 (25-55) | 41 (24-58) |
| Weight, mean (range), kg | 79.3 (51.0-113.0) | 95.3 (53.0-130.9) |
| White race | 10 (100) | 14 (100) |
| Ongoing or resolved medical/surgical history§ | ||
| Hepatic | 7 (70) | 13 (92.9) |
| Musculoskeletal | 9 (90) | 11 (78.6) |
| Cardiovascular | 1 (10) | 8 (57.1) |
| Gastrointestinal | 2 (20) | 2 (14.3) |
| Other | 0 (0) | 2 (14.3) |
| Time since hemophilia diagnosis, mean (SD), y | 34.1 (9.9) | 38.8 (9.7) |
| No. of bleeds in past 12 mo, median (range) | 48 (1-65) | 48 (3-96) |
| Prestudy FVIII regimen¶ | ||
| On demand | 8 (80) | 13 (92.9) |
| Prophylaxis | 2 (20) | 1 (7.1) |
| Characteristic . | Cohort 1 (50 IU/kg efanesoctocog alfa) . | Cohort 2 (65 IU/kg efanesoctocog alfa) . |
|---|---|---|
| Enrolled, n | 10 | 14 |
| Included in safety analysis* | 10 (100) | 14 (100) |
| Included in pharmacokinetic analysis† | 9 (90) | 14 (100) |
| Completed study‡ | 10 (100) | 14 (100) |
| Age, mean (range), y | 35 (25-55) | 41 (24-58) |
| Weight, mean (range), kg | 79.3 (51.0-113.0) | 95.3 (53.0-130.9) |
| White race | 10 (100) | 14 (100) |
| Ongoing or resolved medical/surgical history§ | ||
| Hepatic | 7 (70) | 13 (92.9) |
| Musculoskeletal | 9 (90) | 11 (78.6) |
| Cardiovascular | 1 (10) | 8 (57.1) |
| Gastrointestinal | 2 (20) | 2 (14.3) |
| Other | 0 (0) | 2 (14.3) |
| Time since hemophilia diagnosis, mean (SD), y | 34.1 (9.9) | 38.8 (9.7) |
| No. of bleeds in past 12 mo, median (range) | 48 (1-65) | 48 (3-96) |
| Prestudy FVIII regimen¶ | ||
| On demand | 8 (80) | 13 (92.9) |
| Prophylaxis | 2 (20) | 1 (7.1) |
Unless otherwise noted, data are n (%).
SD, standard deviation.
The safety analysis included participants who received ≥1 dose of efanesoctocog.
The PK analysis included participants who had adequate blood samples for PK assessments, as determined by the sponsor.
Defined as participants who did not discontinue early.
Includes conditions present in >10% of participants in either group.
The most recent regimen that the participant was receiving prior to the study.