bleeding disorder hemofilia
Conditions
Interventions
BAX 855 will be administered at the Baseline Visit and at all study visits
except for Visit 5 ± 1 EDs and Visit 15 ± 1 EDs at a fixed dose to determine IR
(seeSection 8.7.3.1). Subjects will receive
Sponsors
Baxalta Innovations GmbH
Eligibility
Age
2 Years to 11 Years
Inclusion criteria
Inclusion criteria: 1. Subject is 200 cells/mm3, as confirmed by central laboratory at screening 5. Parent or legally authorized representative is willing and able to comply with the requirements of the protocol
Exclusion criteria
Exclusion criteria: 1. Subject has detectable FVIII inhibitory antibodies (* 0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening 2. Subject has a history of FVIII inhibitory antibodies (* 0.6 BU using the Nijmegen modification of the Bethesda assay or the Bethesda assay) at any time prior to screening 3. Subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease) 4. Subject has been previously treated with any type of FVIII concentrate other than ADVATE or BAX 855, or was administered ADVATE BAX 855 or plasma transfusion for * 3 EDs at any time prior to screening 5. Subject receives > 2 EDs of ADVATE in total during the periods prior to enrollment and during the screening period, up until the baseline visit. 6. The subject's weight is anticipated to be 5 times upper limit of normal alanine aminotransferase (ALT), aspartate aminotransferase (AST), or a documented INR > 1.5] in his medical history or at the time of screening 10. Subject has severe renal impairment (serum creatinine > 1.5 times the upper limit of normal) 11. Subject has current or recent (
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of FVIII inhibitor development. | — |
Secondary
| Measure | Time frame |
|---|---|
| Safety: * Binding IgG and IgM antibodies to FVIII, PEG-FVIII and PEG * AEs and SAEs * Clinically significant changes in vital signs and clinical laboratory parameters (hematology and clinical chemistry) Efficacy: * Annualized bleeding rate (ABR) for prophylactic and on-demand treatment * Number of BAX 855 infusions per bleeding episode * Overall hemostatic efficacy rating at 24 h after initiation of treatment and at resolution of bleed * Weight-adjusted consumption of BAX 855 per month, per year and per event (prophylaxis, treatment of bleeding episode) and the number of infusions per month and per year Assessment of intra-, post- and perioperative hemostatic efficacy in case of surgery Intra- and postoperative blood loss in case of surgery Pharmacokinetics: * IR at baseline and over time * Half-life at baseline (optional) Additional Outcome Measures for ItI: Primary: the sucess rate of ITI therapy with BAX 855. Secondary: the rate of partial success and failure of ITI with BAX 855. ABR during ITI Weight-adjusted consumption of BAX 855 per month and per year for each ITI regimen employed. Catheter-related complicatons. Binding IgG and IgM antibodies to FVIII, PEG-FVIII, and PEG. | — |
Countries
Netherlands
Outcome results
None listed