Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
Antihemophilic Factor- Plasma/Albumin Free Method · 3 trials · 1 indication
Inhibitory antibodies to FVIII were measured using the Nijmegen modification of the Bethesda assay. Incidence of an FVIII inhibitory antibody was defined as an inhibitor level ≥0.6 Bethesda units \[BU\].
Comparisons between prophylactic and on-demand treatment were based on ABR estimates from a negative binomial regression model, taking into account the treatment regimen, target joints and age at screening, and duration of the observation period for efficacy.
| Arm | Type | Description |
|---|---|---|
| <6 years old | EXPERIMENTAL | - |
| ≥6 to <12 years | EXPERIMENTAL | - |
| Prophylaxis | EXPERIMENTAL | - |
| On-demand | EXPERIMENTAL | - |
| Cohort 1 | EXPERIMENTAL | Low dose of ADVATE followed by low dose of BAX 855 |
| Cohort 2 | EXPERIMENTAL | High dose of ADVATE followed by high dose of BAX 855 |
| Name | Type | Description |
|---|---|---|
| Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method | BIOLOGICAL | Pharmacokinetic (PK) analysis of ADVATE |
| PEGylated Recombinant Factor VIII | BIOLOGICAL | Pharmacokinetic (PK) analysis of BAX 855 |
Inclusion Criteria: * Severe hemophilia A (Factor VIII (FVIII) \<1%) determined by central laboratory. * \<12 years old at the time of screening. * Participants aged ≥6 to \<12 years of age have been previously treated with plasma-derived and/or recombinant Factor VIII (rFVIII) concentrate(s) for a...