Baseline demographics and patient characteristics of the safety analysis set (n = 103)
| Median (range) age, mo* | 7.0 (0.2-48.0) |
| Age categories, y, n (%) | |
| <0.5 | 42 (40.8) |
| ≥0.5 to <1 | 38 (36.9) |
| 1 | 14 (13.6) |
| 2 | 6 (5.8) |
| 3 | 2 (1.9) |
| 4 | 1 (1.0) |
| Race, n (%) | |
| White | 79 (76.7) |
| Black or African American | 2 (1.9) |
| Asian | 5 (4.9) |
| Native Hawaiian or other Pacific Islander | 2 (1.9) |
| Not reported owing to confidentiality regulations | 4 (3.9) |
| Other | 11 (10.7) |
| Ethnicity, n (%) | |
| Hispanic or Latino | 17 (16.5) |
| Not Hispanic or Latino | 76 (73.8) |
| Not reported owing to confidentiality regulations | 10 (9.7) |
| Geographic location, n (%)† | |
| Europe | 50 (48.5) |
| North America | 31 (30.1) |
| Other | 22 (21.4) |
| Family history of inhibitors, n (%) | |
| Yes | 20 (19.4) |
| No | 71 (68.9) |
| Unknown | 12 (11.7) |
| F8 genotype, n (%) | |
| Exon 7 deletion (no frameshift) | 1 (1.0) |
| Intron 22 inversion | 47 (45.6) |
| Nonsense | 10 (9.7) |
| Frameshift | 16 (15.5) |
| Large structural change (>50 bp) | 5 (4.9) |
| Splice site change | 3 (2.9) |
| Missense | 4 (3.9) |
| Unknown | 17 (16.5) |
| Median (range) age, mo* | 7.0 (0.2-48.0) |
| Age categories, y, n (%) | |
| <0.5 | 42 (40.8) |
| ≥0.5 to <1 | 38 (36.9) |
| 1 | 14 (13.6) |
| 2 | 6 (5.8) |
| 3 | 2 (1.9) |
| 4 | 1 (1.0) |
| Race, n (%) | |
| White | 79 (76.7) |
| Black or African American | 2 (1.9) |
| Asian | 5 (4.9) |
| Native Hawaiian or other Pacific Islander | 2 (1.9) |
| Not reported owing to confidentiality regulations | 4 (3.9) |
| Other | 11 (10.7) |
| Ethnicity, n (%) | |
| Hispanic or Latino | 17 (16.5) |
| Not Hispanic or Latino | 76 (73.8) |
| Not reported owing to confidentiality regulations | 10 (9.7) |
| Geographic location, n (%)† | |
| Europe | 50 (48.5) |
| North America | 31 (30.1) |
| Other | 22 (21.4) |
| Family history of inhibitors, n (%) | |
| Yes | 20 (19.4) |
| No | 71 (68.9) |
| Unknown | 12 (11.7) |
| F8 genotype, n (%) | |
| Exon 7 deletion (no frameshift) | 1 (1.0) |
| Intron 22 inversion | 47 (45.6) |
| Nonsense | 10 (9.7) |
| Frameshift | 16 (15.5) |
| Large structural change (>50 bp) | 5 (4.9) |
| Splice site change | 3 (2.9) |
| Missense | 4 (3.9) |
| Unknown | 17 (16.5) |
Percentages are based on the number of subjects with available data in the safety analysis set.
bp, base pair.
Age at the time of informed consent.
Europe includes France (4 subjects), Germany (5 subjects), Ireland (5 subjects), Italy (11 subjects), Poland (8 subjects), Spain (1 subject), Sweden (2 subjects), and the United Kingdom (14 subjects). North America includes Canada (11 subjects) and the United States (20 subjects). Other countries include Australia (7 subjects), Brazil (14 subjects), and New Zealand (1 subject).