Severe hemophilia A (FVIII<1%) MedDRA version: 17.1 Level: LLT Classification code 10060612 Term: Hemophilia A System Organ Class: 100000004850
Conditions
Interventions
Product Name: PEGylated rFVIII
Product Code: BAX855 250IU/vial
Pharmaceutical Form: Lyophilisate and solvent for solution for injection
INN or Proposed INN: Not applicable
Current Sponsor code: BAX-85
Sponsors
Baxter Innovations GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. The subject has severe hemophilia A (FVIII =65 years) no F.1.3.1 Number of subjects for this age range
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 confirmed history of FVIII inhibitory antibodies (=0.6 BU using the Nijmegen modification of the Bethesda assay or =0.6 BU using the Bethesda assay) at any time prior to screening. 3. Subject has known hypersensitivity towards mouse or hamster proteins, PEG, or Tween 80. 4. Subject has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand’s disease). 5. Subject’s platelet count is 1.5). 7. Subject has severe renal impairment (serum creatinine >1.5 times ULN). 8. Subject is scheduled to receive during the course of the study, an immunomodulating drug (eg, corticosteroid agents at a dose equivalent to hydrocortisone >10 mg/day, or a-interferon) other than anti-retroviral chemotherapy. 9. Subject has current or recent (<30 days) use of other PEGylated drugs prior to study participation or is scheduled to use such drugs during study participation. 10. Subject has participated in another clinical study involving an IP or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study. 11. Subject has a medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the Investigator, would affect subject safety or compliance. 12. Subject’s legal representative is a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (ie, children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate FVIII inhibitory antibodies (=0.6 BU using the Nijmegen modification of the Bethesda assay);Secondary Objective: 1. To evaluate the PK parameters of BAX 855 in pediatric PTPs <12 years of age. 2. To monitor incremental recovery (IR) of BAX 855 over time. 3. To evaluate hemostatic efficacy of BAX 855 in the management of acute bleeding episodes and for prophylaxis over a period of 6 months. 4. To assess all adverse events (AEs) possibly or probably related to BAX 855. 5. To evaluate immunogenicity (binding antibodies to FVIII, BAX 855, PEG, and Chinese hamster ovary [CHO]) and clinically significant changes in routine laboratory parameters (hematology, clinical chemistry, and lipids) and vital signs. Exploratory Objective(s): 1. To evaluate changes in HRQoL and health resource use.;Primary end point(s): The primary outcome measure is the incidence of FVIII inhibitory antibodies ;Timepoint(s) of evaluation of this end point: 6 months (±2 weeks) or at least 50 EDs | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Pharmacokinetics 1. Area under the plasma concentration versus time curve from 0 to 8 hours post infusion (AUC0-8), AUC0-8/dose, MRT, clearance (CL), IR, T1/2, volume of distribution at steady state (Vss) following an initial dose of ADVATE followed by BAX 855. 2. IR over time. Hemostatic Efficacy 1. Annualized bleeding rate (ABR). 2. Consumption of BAX 855: number of infusions and weight-adjusted consumption per month and per year. 3. Number of infusions per bleeding episode, overall hemostatic efficacy rating at resolution of bleed. 4. Weight-adjusted consumption per bleeding episode. Safety 1. All AEs and SAEs possibly or probably related to BAX 855. 2. Clinical significant changes in vital signs (pulse, respiration, supine blood pressure, and temperature) and clinical laboratory parameters (hematology, clinical chemistry, and lipids). 3. Assessment of binding antibodies to FVIII, BAX 855, PEG, and CHO.;Timepoint(s) of evaluation of this end point: 6 months (±2 weeks) or at least 50 EDs | — |
Countries
Australia, Austria, Belgium, Bulgaria, Czech Republic, Germany, Hong Kong, Ireland, Japan, Korea, Republic of, Lithuania, Malaysia, Netherlands, Poland, Romania, Spain, Taiwan, Thailand, Turkey, Ukraine, United Kingdom, United States
Contacts
Public ContactEli Taube
Baxter Innovations GmbH
Outcome results
None listed