BAX 326 (Recombinant Factor IX): Evaluation of Safety, Immunogenicity, and Hemostatic Efficacy in Previously Treated Patients With Severe (FIX Level < 1%) or Moderately Severe (FIX Level <= 2%) Hemophilia B - A Continuation Study

简要总结

主要结局

次要结局

• Annualized Bleed Rate During Prophylaxis Treatment(For prophylactic treatment the period from first to last prophylactic infusion is considered.)
• Development of Antibodies to Chinese Hamster Ovary Proteins (CHO Proteins) and rFurin(Laboratory assessment for immunology were done at screening, at exposure day 1, at week 4 (± 1 week), at month 3 (±1 week), thereafter, every 3 months (± 1 week) and at study completion/termination.)
• Consumption of BAX 326: Weight Adjusted Consumption Per Month and Per Year(Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).)
• Treatment of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Resolution of Bleed(Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).)
• Consumption of BAX 326: Number of Infusions Per Month and Per Year(Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).)
• Treatment of Bleeding Episodes: Number of Infusions Per Bleeding Episode Required Until Bleed Resolution(Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).)
• Occurrence of Severe Allergic Reactions and Thrombotic Events(Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).)
• Pharmacokinetics: Area Under the Plasma Concentration Versus Time Curve From Time 0 to Infinity (AUC 0-∞)(PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours)
• Pharmacokinetics: Volume of Distribution at Steady State (Vss)(PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours)
• Pharmacokinetics: Elimination Phase Half-life (T1/2)(PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours)
• Consumption of BAX326: Weight Adjusted Consumption Per Bleeding Episode(Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).)
• Development of Inhibitory and Total Binding Antibodies to Factor IX(Laboratory assessment for immunology were done at screening, at exposure day 1, at week 4 (± 1 week), at month 3 (±1 week), thereafter, every 3 months (± 1 week) and at study completion/termination.)
• Clinical Significant Changes in Routine Laboratory Parameters and Vital Signs(Measurements at screening and at study completion/termination are included in the analysis.)
• Pharmacokinetics: Incremental Recovery (IR) Over Time(IR over time was measured as Baseline and at Completion/Termination visit within 30 minutes pre-infusion and at 30 (± 5) minutes post-infusion.)
• Changes in Health Related Quality of Life (HR QoL) Based on Questionnaire SF-36(Baseline at exposure day 1 and at study completion/termination.)
• Pharmacokinetics: Mean Residence Time (MRT)(PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours)
• Pharmacokinetics: Systemic Clearance (CL)(PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours)
• Pharmacokinetics: Incremental Recovery (IR)(PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours.)
• Changes in Health Related Quality of Life (HR QoL) Based on Questionnaire Haemo-QoL and Haem-A-QoL(Baseline at exposure day 1 and at study completion/termination.)
• Changes in Health Related Quality of Life Using the Peds QL(Baseline at exposure day 1 and at study completion/termination.)
• Changes in Health Related Quality of Life (HR QoL) Based on Questionnaire EQ-5D and Pain Score.(Baseline at exposure day 1 and at study completion/termination.)