Patient disposition and baseline demographics
| Patient disposition, n | |
| Enrolled and dosed | 41 |
| Withdrawals | 5 |
| Inconvenience (parent decision; visit 5) | 1 |
| Adverse events of worsening leukopenia and neutropenia (visit 6) | 1 |
| Pregnancy (visit 8 and 14)* | 2 |
| Personal reasons (visit 9) | 1 |
| Discontinuation of rFXIII treatment due to detection of non-neutralizing Abs | 3† |
| Completed trial | 33 |
| Baseline demographics, n | 41 |
| Age, y | |
| Mean (SD) | 26.4 (15.9) |
| Median | 23.0 |
| Minimum/maximum | 7.0/60.0 |
| Sex, n (%) | |
| Female | 18 (44) |
| Male | 23 (56) |
| Race, n (%) | |
| White | 28 (68) |
| Asian | 5 (12) |
| Black or African American | 2 (5) |
| Other | 5 (12) |
| Unknown‡ | 1 (2) |
| Patient disposition, n | |
| Enrolled and dosed | 41 |
| Withdrawals | 5 |
| Inconvenience (parent decision; visit 5) | 1 |
| Adverse events of worsening leukopenia and neutropenia (visit 6) | 1 |
| Pregnancy (visit 8 and 14)* | 2 |
| Personal reasons (visit 9) | 1 |
| Discontinuation of rFXIII treatment due to detection of non-neutralizing Abs | 3† |
| Completed trial | 33 |
| Baseline demographics, n | 41 |
| Age, y | |
| Mean (SD) | 26.4 (15.9) |
| Median | 23.0 |
| Minimum/maximum | 7.0/60.0 |
| Sex, n (%) | |
| Female | 18 (44) |
| Male | 23 (56) |
| Race, n (%) | |
| White | 28 (68) |
| Asian | 5 (12) |
| Black or African American | 2 (5) |
| Other | 5 (12) |
| Unknown‡ | 1 (2) |
One pregnancy was identified through pregnancy screening and the other was reported by the investigator.
Of the 3 patients who discontinued rFXIII treatment due to the detection of non-neutralizing Abs, the decision was made by the Novo Nordisk Safety Committee for 2 patients (following recommendations from contracted external experts who reviewed data from these patients) and by the patient's parents for 1 patient. All 3 patients remained in the trial for follow-up and completed all trial-related activity visits.
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