Participant baseline demographics and disease characteristics
| Characteristic . | Participant 1 . | Participant 2 . | Participant 3 . |
|---|---|---|---|
| Age at enrolment, y | 43 | 50 | 47 |
| Weight, kg | 89 | 81 | 82 |
| HIV status | Nonreactive | Reactive (controlled) | Reactive (controlled) |
| HBV status | Negative | Negative | Negative |
| HCV status | Resolved | Resolved | Resolved |
| Baseline FIX activity levels, IU/dL | 1 | <1 | <1 |
| Prescreening FIX treatment | Prophylaxis (EHL) | Prophylaxis (EHL) | Prophylaxis (EHL) |
| ABR during 1 year before screening∗ | 3 | 1 | 5† |
| AAV5 NAb status at screening (titer)‡ | Positive (48) | Positive (44) | Positive (25) |
| AAV5 NAb status at day of dosing (titer)‡ | Positive (22) | Positive (33) | Positive (20) |
| Characteristic . | Participant 1 . | Participant 2 . | Participant 3 . |
|---|---|---|---|
| Age at enrolment, y | 43 | 50 | 47 |
| Weight, kg | 89 | 81 | 82 |
| HIV status | Nonreactive | Reactive (controlled) | Reactive (controlled) |
| HBV status | Negative | Negative | Negative |
| HCV status | Resolved | Resolved | Resolved |
| Baseline FIX activity levels, IU/dL | 1 | <1 | <1 |
| Prescreening FIX treatment | Prophylaxis (EHL) | Prophylaxis (EHL) | Prophylaxis (EHL) |
| ABR during 1 year before screening∗ | 3 | 1 | 5† |
| AAV5 NAb status at screening (titer)‡ | Positive (48) | Positive (44) | Positive (25) |
| AAV5 NAb status at day of dosing (titer)‡ | Positive (22) | Positive (33) | Positive (20) |
Table reproduced from von Drygalski et al.17,19
EHL, extended half-life; HBV, hepatitis B virus; HCV, hepatitis C virus.
Total bleeds (treated + untreated).
This participant experienced a further spontaneous bleed in the left hip between screening and enrolment (bleed onset: study day −30) that is not represented in their ABR for the year before screening.
AAV5 NAb data considered positive if titer was ≥2. Luciferase cell-based assay.