| Primary | Study Part 1- Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours Per Dose | Computed using the linear trapezoidal method. The concentration at 72 hours was interpolated from the two nearest sampling time points or extrapolated using the last quantifiable concentration and the terminal rate constant λz. λz was estimated from the slope of natural log-linear fitting to latter quantifiable concentrations, with largest adjusted R^2. | Pharmacokinetic Per Protocol Analysis Set (PKPPAS) -Participants who participated in Study Parts 1-3 and completed Study Part 1 without any major protocol deviations | Posted | | Median | Inter-Quartile Range | (IU·hr/dL) / (IU/kg) | | 72 hours | | | | ID | Title | Description |
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| OG000 | BAX326 | | | OG001 | BeneFIX | |
| | | Title | Denominators | Categories |
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| | | Title | Measurements |
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| - OG00014.30(11.20 to 15.95)
- OG00113.42(11.08 to 15.10)
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| | Group IDs | Group Description | Statistical Method | Statistical Comment | P-Value | P-Value Comment | Parameter Type | Parameter Value | Dispersion Type | Dispersion Value | Confidence Interval Sides | Confidence Interval % | CI Lower Limit | CI Upper Limit | CI Lower Limit Comment | CI Upper Limit Comment | Estimate Comment | Tested Non-Inferiority | Non-Inferiority Type | Non-Inferiority Comment | Other Analysis Description |
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| | | | | | Ratio of Geometric Means | 1.063 | | | 2-Sided | 90 | 1.03 | 1.09 | | | | | Non-Inferiority or Equivalence (legacy) | Bioequivalence will be established if 90% C.I. of ratio is contained completely in the margins of equivalence of 0.8 to 1.25. | |
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| Secondary | Study Parts 1 and 3: Area Under the Plasma Concentration/Time Curve From Time 0 to Infinity Per Dose (AUC0-∞/ Dose) | Defined as (AUC0-t + Ct)/ λz/ dose, where t is the time of last quantifiable concentration, Ct is the last quantifiable concentration. λz will be estimated from the slope of natural log-linear fitting to latter quantifiable concentrations, with largest adjusted R^2. The objective of Study Part 3 was to re-evaluate the Pharmacokinetic (PK) parameters for BAX 326 after a period of 6 months of treatment, in participants who accumulated at least 30 EDs to BAX 326, and to compare them with those determined in the same participants participating in Study Part 1. | Pharmacokinetic Per Protocol Analysis Set (PKPPAS) -Participants who participated in Study Parts 1-3 and completed Study Part 1 without any major protocol deviations | Posted | | Median | Inter-Quartile Range | (IU·hr)/ dL/ (IU/kg) | | 0-30 minutes before infusion up to 72 hours post-infusion | | | | ID | Title | Description |
|---|
| OG000 | Study Part 1: BAX326 | PK infusion with BAX326 at 75 ± 5 IU/kg | | OG001 | Study Part 1: BeneFIX | PK infusion with BeneFIX at 75 ± 5 IU/kg | | OG002 | Study Part 3: BAX326 | PK infusion with BAX326 at 75 ± 5 IU/kg Study Part 3 only evaluated BAX326 (ie BeneFIX was not evaluated in Study Part 3) |
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| Secondary | Study Parts 1 and 3: Mean Residence Time (MRT) | Computed as Area under the moment curve 0-∞ (AUMC0-∞) / AUC0-∞- TI/2, where AUMC0-∞ will be determined in a similar manner as AUC0-∞ and TI represents infusion duration [hr] The objective of Study Part 3 was to re-evaluate the Pharmacokinetic (PK) parameters for BAX 326 after a period of 6 months of treatment, in participants who accumulated at least 30 EDs to BAX 326, and to compare them with those determined in the same participants participating in Study Part 1. | Pharmacokinetic Per Protocol Analysis Set (PKPPAS) -Participants who participated in Study Parts 1-3 and completed Study Part 1 without any major protocol deviations | Posted | | Median | Inter-Quartile Range | Hour | | 0-30 minutes before infusion up to 72 hours post-infusion | | | | ID | Title | Description |
|---|
| OG000 | Study Part 1: BAX326 | PK infusion with BAX326 at 75 ± 5 IU/kg | | OG001 | Study Part 1: BeneFIX | PK infusion with BeneFIX at 75 ± 5 IU/kg | | OG002 | Study Part 3: BAX326 | PK infusion with BAX326 at 75 ± 5 IU/kg Study Part 3 only evaluated BAX326 (ie BeneFIX was not evaluated in Study Part 3) |
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| Secondary | Study Parts 1 and 3: Clearance (CL) | Computed as Dose/ AUC0-∞. The objective of Study Part 3 was to re-evaluate the Pharmacokinetic (PK) parameters for BAX 326 after a period of 6 months of treatment, in participants who accumulated at least 30 EDs to BAX 326, and to compare them with those determined in the same participants participating in Study Part 1. | Pharmacokinetic Per Protocol Analysis Set (PKPPAS) -Participants who participated in Study Parts 1-3 and completed Study Part 1 without any major protocol deviations | Posted | | Median | Inter-Quartile Range | dL/(kg·hr) | | 0-30 minutes before infusion up to 72 hours post-infusion | | | | ID | Title | Description |
|---|
| OG000 | Study Part 1: BAX326 | PK infusion with BAX326 at 75 ± 5 IU/kg | | OG001 | Study Part 1: BeneFIX | PK infusion with BeneFIX at 75 ± 5 IU/kg | | OG002 | Study Part 3: BAX326 | PK infusion with BAX326 at 75 ± 5 IU/kg Study Part 3 only evaluated BAX326 (ie BeneFIX was not evaluated in Study Part 3) |
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| Secondary | Study Parts 1 and 3: Incremental Recovery at Cmax (IR at Cmax) | Defined as (Cmax - Cpre-infusion)/Dose, where maximum concentration (Cmax) will be determined as the highest concentration achieved within one hour after infusion. The objective of Study Part 3 was to re-evaluate the Pharmacokinetic (PK) parameters for BAX 326 after a period of 6 months of treatment, in participants who accumulated at least 30 EDs to BAX 326, and to compare them with those determined in the same participants participating in Study Part 1. | Pharmacokinetic Per Protocol Analysis Set (PKPPAS) -Participants who participated in Study Parts 1-3 and completed Study Part 1 without any major protocol deviations | Posted | | Median | Inter-Quartile Range | (IU/dL) / (IU/kg) | | 0-30 minutes before infusion up to 1 hour post-infusion | | | | ID | Title | Description |
|---|
| OG000 | Study Part 1: BAX326 | PK infusion with BAX326 at 75 ± 5 IU/kg | | OG001 | Study Part 1: BeneFIX | PK infusion with BeneFIX at 75 ± 5 IU/kg | | OG002 | Study Part 3: BAX326 | PK infusion with BAX326 at 75 ± 5 IU/kg Study Part 3 only evaluated BAX326 (ie BeneFIX was not evaluated in Study Part 3) |
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| Secondary | Incremental Recovery (IR) at 30 Minutes Over Time | IR at 30 Minutes was measured at the following time points during the study: - Part 1 or Part 2, Exposure Day (ED) 1. (If participant was present for Study Part 1, then ED 1 from Part 1 was used. If Participant entered study in Study Part 2, then ED 1 from Part 2 was used.) - Part 2: Week 5 - Part 2: Week 13 - Part 2 or Part 3: Week 26 (Week 26 of study participation) - Study Completion or Termination Visit | Pharmacokinetic Per Protocol Analysis Set (PKPPAS) -Participants who participated in Study Parts 1-3 and completed Study Part 1 without any major protocol deviations | Posted | | Median | Inter-Quartile Range | (IU/dL) / (IU/kg) | | 0-30 minutes before infusion and 30 minutes post-infusion | | | | ID | Title | Description |
|---|
| OG000 | Part 1 or Part 2, ED 1 | PK infusion with BAX326 at 75 ± 5 IU/kg | | OG001 | Part 2: Week 5 | PK infusion with BeneFIX at 75 ± 5 IU/kg | | OG002 | Part 2: Week 13 | PK infusion with BAX326 at 75 ± 5 IU/kg Study Part 3 only evaluated BAX326 (ie BeneFIX was not evaluated in Study Part 3) | | OG003 |
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| Secondary | Change in Incremental Recovery (IR) at 30 Minutes Over Time | The median changes in IR at 30 Minutes, calculated as the change in IR value from exposure day 1 (ED1). | Pharmacokinetic Per Protocol Analysis Set (PKPPAS) -Participants who participated in Study Parts 1-3 and completed Study Part 1 without any major protocol deviations | Posted | | Median | Inter-Quartile Range | (IU/dL) / (IU/kg) | | 0-30 minutes before infusion and 30 minutes post-infusion | | | | ID | Title | Description |
|---|
| OG000 | Part 2: Week 5 | PK infusion with BAX326 at 75 ± 5 IU/kg | | OG001 | Part 2: Week 13 | | | OG002 | Part 2 or Part 3: Week 26 | | | OG003 | Study Completion or Termination Visit | |
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| Secondary | Study Parts 1 and 3: Half Life (T 1/2) | Elimination phase half-life will be determined as ln2/ λz. The objective of Study Part 3 was to re-evaluate the Pharmacokinetic (PK) parameters for BAX 326 after a period of 6 months of treatment, in participants who accumulated at least 30 EDs to BAX 326, and to compare them with those determined in the same participants participating in Study Part 1. | Pharmacokinetic Per Protocol Analysis Set (PKPPAS) -Participants who participated in Study Parts 1-3 and completed Study Part 1 without any major protocol deviations | Posted | | Median | Inter-Quartile Range | Hour | | 0-30 minutes before infusion up to 72 hours post-infusion | | | | ID | Title | Description |
|---|
| OG000 | Study Part 1: BAX326 | PK infusion with BAX326 at 75 ± 5 IU/kg | | OG001 | Study Part 1: BeneFIX | PK infusion with BeneFIX at 75 ± 5 IU/kg | | OG002 | Study Part 3: BAX326 | PK infusion with BAX326 at 75 ± 5 IU/kg Study Part 3 only evaluated BAX326 (ie BeneFIX was not evaluated in Study Part 3) |
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| Secondary | Study Parts 1 and 3: Volume of Distribution at Steady State (Vss) | Vss computed as CL·MRT. The objective of Study Part 3 was to re-evaluate the Pharmacokinetic (PK) parameters for BAX 326 after a period of 6 months of treatment, in participants who accumulated at least 30 EDs to BAX 326, and to compare them with those determined in the same participants participating in Study Part 1. | Pharmacokinetic Per Protocol Analysis Set (PKPPAS) -Participants who participated in Study Parts 1-3 and completed Study Part 1 without any major protocol deviations | Posted | | Median | Inter-Quartile Range | dL/kg | | 0-30 minutes before infusion up to 72 hours post-infusion | | | | ID | Title | Description |
|---|
| OG000 | Study Part 1: BAX326 | PK infusion with BAX326 at 75 ± 5 IU/kg | | OG001 | Study Part 1: BeneFIX | PK infusion with BeneFIX at 75 ± 5 IU/kg | | OG002 | Study Part 3: BAX326 | PK infusion with BAX326 at 75 ± 5 IU/kg Study Part 3 only evaluated BAX326 (ie BeneFIX was not evaluated in Study Part 3) |
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| Secondary | Study Part 2: Annualized Bleed Rate (ABR) During Treatment With BAX326 | ABR during prophylaxis (twice-weekly) in Part 2 was calculated as (Number of bleeding episodes/observed treatment period in days) * 365.25. The treatment period on prophylaxis was defined as time between the first and the last prophylactic infusions and ABR on prophylaxis was calculated for participants who received a minimum of 3 months of prophylactic treatment with BAX326. | Full Analysis Set - Prophylactic cohort | Posted | | Median | Inter-Quartile Range | Bleeds per year | | Study Part 2 = 26 weeks ± 1 week (Note: Study Part 1 = 2-4 weeks) | | | | ID | Title | Description |
|---|
| OG000 | BAX326 Prophylaxis | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. Participants received a minimum of three months prophylactic treatment |
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| Secondary | Bleeding Episodes Treated With 1, 2 or ≥3 Infusions of BAX326 by Bleeding Site and Cause | The number of bleeding episodes treated with 1, 2, or ≥3 infusions of BAX326 to achieve adequate hemostasis. Only infusions required until resolution of bleed were considered. | | Posted | | Number | | Bleeding episodes | | Study Part 2 = 26 weeks ± 1 week (Study Part 2 began at week 3-5) | bleeding episodes | bleeding episodes | | ID | Title | Description |
|---|
| OG000 | Bleeding Site: Target Joint | | | OG001 | Bleeding Site: Non-Target Joint | | | OG002 | Bleeding Site: All Joint | | | OG003 | Bleeding Site: Non-Joint | | | OG004 | Bleeding Cause: Spontaneous | | | OG005 | Bleeding Cause: Injury | | | OG006 |
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| Secondary | Hemostatic Efficacy at Resolution of All Bleeding Episodes (BEs) Treated With BAX326 by Bleeding Site and Cause | Rating Scale for Treatment of BEs (4-point ordinal scale): -Excellent: Full relief of pain and cessation of objective signs of bleeding (eg, swelling, tenderness, and decreased range of motion in the case of musculoskeletal hemorrhage) after a single infusion. No additional infusion required for the control of bleeding. Administration of further infusions to maintain hemostasis did not affect this scoring. -Good: Definite pain relief and/or improvement in signs of bleeding after a single infusion. Possibly requires more than 1 infusion for complete resolution. -Fair: Probable and/or slight relief of pain and slight improvement in signs of bleeding after single infusion. Required more than 1 infusion for complete resolution. -None: No improvement or condition worsens. | | Posted | | Number | | Bleeding episodes | | At bleed resolution throughout the study period of 22 months (Study Parts 1, 2, and 3) | Bleeding episodes | Bleeding episodes | | ID | Title | Description |
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| OG000 | Bleeding Site: Target Joint | | | OG001 | Bleeding Site: Non-Target Joint | | | OG002 | Bleeding Site: All Joint | | | OG003 | Bleeding Site: Non-Joint |
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| Secondary | Total Weight-adjusted Dose Per Bleeding Episode (BEs) of All BEs Treated With BAX326 by Bleeding Site and Cause | | | Posted | | Median | Inter-Quartile Range | IU/kg | | Study Part 2 = 26 weeks ± 1 week (Note: Study Part 1 = 2-4 weeks) | Bleeding Episodes | Bleeding Episodes | | ID | Title | Description |
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| OG000 | Bleeding Site: Target Joint | | | OG001 | Bleeding Site: Non-Target Joint | | | OG002 | Bleeding Site: All Joint | | | OG003 | Bleeding Site: Non-Joint | | | OG004 | Bleeding Cause: Spontaneous | | | OG005 | Bleeding Cause: Injury | | | OG006 | Bleeding Cause: Unknown | |
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| Secondary | Consumption of BAX326 Per Event Per Participant | Weight-adjusted consumption of BAX326 by event per participant, i.e., for prophylactic treatment and for treatment of bleeds until resolution of bleed. | | Posted | | Median | Inter-Quartile Range | IU/kg | | Study Part 2 = 26 weeks ± 1 week (Note: Study Part 1 = 2-4 weeks) | | | | ID | Title | Description |
|---|
| OG000 | Prophylactic Treatment | | | OG001 | Bleeding Treatment | Includes all participants who received any infusions for bleeding treatment in the Full Analysis Set. (ie includes all On-demand arm (N=14) and 33 participants from the prophylaxis arm who experienced a bleeding episode) |
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| Secondary | Consumption of BAX326 Per Participant: Median Number of Infusions Per Month | | | Posted | | Median | Inter-Quartile Range | Infusions | | Study Part 1 = 2-4 weeks, Study Part 2 = 26 weeks ± 1 week (Prophylaxis and On-Demand period), Study Part 3 = 1 week (Total = 29-31 weeks) | | | | ID | Title | Description |
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| OG000 | Prophylaxis | | | OG001 | On-Demand | |
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| Secondary | Consumption of BAX326 Per Participant: Median Weight-adjusted Consumption Per Month | | | Posted | | Median | Inter-Quartile Range | IU/kg | | Study Part 1 = 2-4 weeks, Study Part 2 = 26 weeks ± 1 week (Prophylaxis and On-Demand period), Study Part 3 = 1 week (Total = 29-31 weeks) | | | | ID | Title | Description |
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| OG000 | Prophylaxis | | | OG001 | On-Demand | |
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| Secondary | Number of Participants Who Developed Inhibitory Antibodies to Factor IX (FIX) | | | Posted | | Number | | participants | | Study Part 1 = 2-4 weeks, Study Part 2 = 26 weeks ± 1 week, Study Part 3 = 1 week (Total = 29-31 weeks) | | | | ID | Title | Description |
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| OG000 | All Study Participants | |
| | | Title | Denominators | Categories |
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| Secondary | Occurrence of Total Binding Antibodies of Indeterminate Specificity (Within Assay Variability) | Occurrence of total binding antibodies of indeterminate specificity (within assay variability) to FIX, antibodies to CHO proteins and rFurin is defined by a dilution of 2 or less increase as compared to levels at screening visit (e.g. negative to 1:20 or 1:40). | | Posted | | Number | | participants | | Study Part 1 = 2-4 weeks, Study Part 2 = 26 weeks ± 1 week, Study Part 3 = 1 week (Total = 29-31 weeks) | | | | ID | Title | Description |
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| OG000 | All Study Participants | |
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| Secondary | Occurrence of Treatment Related Total Binding Antibodies | Occurrence of treatment related total binding antibodies to Factor IX (FIX), antibodies to Chinese hamster ovary (CHO) proteins, and recombinant furin (rFurin) is defined by more than 2-dilution increase as compared to levels at screening visit and confirmed specificity (e.g. negative to 1:80) | | Posted | | Number | | participants | | Study Part 1 = 2-4 weeks, Study Part 2 = 26 weeks ± 1 week, Study Part 3 = 1 week (Total = 29-31 weeks) | | | | ID | Title | Description |
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| OG000 | All Study Participants | |
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| Secondary | Number of Participants Who Experienced Severe Allergic Reactions (e.g. Anaphylaxis) | | | Posted | | Number | | participants | | Study Part 1 = 2-4 weeks, Study Part 2 = 26 weeks ± 1 week, Study Part 3 = 1 week (Total = 29-31 weeks) | | | | ID | Title | Description |
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| OG000 | All Study Participants | |
| | | Title | Denominators | Categories |
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| Secondary | Number of Participants Who Experienced Thrombotic Events | | | Posted | | Number | | participants | | Study Part 1 = 2-4 weeks, Study Part 2 = 26 weeks ± 1 week, Study Part 3 = 1 week (Total = 29-31 weeks) | | | | ID | Title | Description |
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| OG000 | All Study Participants | |
| | | Title | Denominators | Categories |
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| Secondary | Number of Participants With Clinically Significant Changes in Laboratory Parameters: Clinical Chemistry | Clinically significant changes in chemistry assessments for Alanine Aminotransferase, Albumin, Alkaline Phosphatase, Aspartate Aminotransferase, Bicarbonate, Bilirubin, Blood Urea Nitrogen, Chloride, Glucose, Potassium, Protein (Serum), Sodium. Clinically Significant (CS) defined as: -1. The abnormal value constitutes an adverse event (AE) and, -2. The abnormal value is a symptom of or related to a disease that is already recorded as an AE in Case Report Form (CRF). | | Posted | | Number | | participants | | Study Part 1 = 2-4 weeks, Study Part 2 = 26 weeks ± 1 week, Study Part 3 = 1 week (Total = 29-31 weeks) | | | | ID | Title | Description |
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| OG000 | All Study Participants | |
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| Secondary | Number of Participants With Clinically Significant Changes in Laboratory Parameters: Hematology | Clinically significant changes in hematology assessments for Basophils, Basophils/Leukocytes, Eosinophils, Eosinophils/Leukocytes, Erythrocyte Mean Corpuscular Hemoglobin Concentration, Erythrocyte Mean Corpuscular Volume, Erythrocytes, Hematocrit, Hemoglobin, Leukocytes, Lymphocytes, Lymphocytes/Leukocytes, Monocytes, Monocytes/Leukocytes, Neutrophils, Neutrophils/Leukocytes, Platelets, | | Posted | | Number | | participants | | Study Part 1 = 2-4 weeks, Study Part 2 = 26 weeks ± 1 week, Study Part 3 = 1 week (Total = 29-31 weeks) | | | | ID | Title | Description |
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| OG000 | All Study Participants | |
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| Secondary | Number of Participants With Clinically Significant Changes in Laboratory Parameters: Vital Signs | Clinically significant changes in vital signs assessments for pulse rate, systolic/diastolic blood pressure, respiratory rate, body temperature | | Posted | | Number | | participants | | Study Part 1 = 2-4 weeks, Study Part 2 = 26 weeks ± 1 week, Study Part 3 = 1 week (Total = 29-31 weeks) | | | | ID | Title | Description |
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| OG000 | All Study Participants | |
| | |
| Secondary | Number of Participants With Clinically Significant Changes in Laboratory Parameters: Thrombogenic Markers | Clinically significant changes in thrombogenic markers assessments for thrombin-antithrombin (TAT), prothrombin fragment 1.2, and D-dimer as evaluated by an independent Data Monitoring Committee (DMC) | | Posted | | Number | | participants | | Study Part 1 = 2-4 weeks, Study Part 2 = 26 weeks ± 1 week, Study Part 3 = 1 week (Total = 29-31 weeks) | | | | ID | Title | Description |
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| OG000 | All Study Participants | |
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| Secondary | Number of Adverse Events (AEs) After BAX326 Treatment | | | Posted | | Number | | adverse events | | Study Part 1 = 2-4 weeks, Study Part 2 = 26 weeks ± 1 week, and Study Part 3 = 1 week (Total = 29-31 weeks) | | | | ID | Title | Description |
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| OG000 | AEs - Not Related | | | OG001 | AEs - Related | Probable, possible, or unknown causality assessment of an AE will be counted as "related". |
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| Secondary | Number of Participants With Adverse Events (AEs) After BAX326 Treatment | | | Posted | | Number | | participants | | Study Part 1 = 2-4 weeks, Study Part 2 = 26 weeks ± 1 week, and Study Part 3 = 1 week (Total = 29-31 weeks) | | | | ID | Title | Description |
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| OG000 | Participants With AEs - Not Related | | | OG001 | Participants With AEs - Related | Probable, possible, or unknown causality assessment of an AE will be counted as "related". |
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| Secondary | EuroQoL (Quality of Life)-5 Dimensions (EQ-5D) Total Index Scores | EQ-5D is a participant answered questionnaire scoring 5 dimensions - mobility, self-care, usual activities, pain/discomfort and anxiety/depression. The EQ-5D total score ranges from 0 (worst health state) to 1 (perfect health state) and 1 reflects the best outcome. | | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline at either Study Part 1, or Study Part 2, and End of Study (study weeks 29-31) | | | | ID | Title | Description |
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| OG000 | Prophylaxis: Part 1 or Part 2, Exposure Day 1 (Baseline) | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | Prophylaxis: End of Study | | | OG002 | Prophylaxis: Change From Baseline | | | OG003 | On-Demand: Part 1 or Part 2, Exposure Day 1 (Baseline) | On-Demand Dosing and Frequency (Guidance): • Early hemarthrosis, muscle bleed, oral bleed= FIX level required(%) 20-40 IU/dL. Repeat every 24 hours. Duration: at least 1 day, until bleeding episode as indicated by pain is resolved or healing is achieved • More extensive hemarthrosis, muscle bleed, hematoma= FIX level required(%) 30-60 IU/dL. Repeat infusion every 24 hours for 3-4 days or more until pain and acute disability are resolved • Life threatening hemorrhages= FIX level required(%) 60-100 IU/dL. Repeat infusion every 8-24 hours until threat resolves Required dose calculated using IR determined of first 16 participants who completed Part 1 according to: Body weight (kg) x desired FIX rise (%) (IU/dL) x {reciprocal of observed recovery} Given an anticipated recovery of 0.8 [IU/dl]/[IU/kg], required units calculated by: Body weight (kg) x desired FIX rise (%or (IU/dL) x 1.3 IU/kg Amount administered & frequency should be oriented to individual clinical effectiveness |
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| Secondary | EuroQoL (Quality of Life)-5 Dimensions Visual Analogue Scale (EQ-5D VAS) Scores | Participant rated questionnaire to assess health-related quality of life in terms of a single index value. The VAS component rates current health state on a scale from 0 (worst imaginable health state) to 100 (best imaginable health state); higher scores indicate a better quality of life. | | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline at either Study Part 1, or Study Part 2, and End of Study (study weeks 29-31) | | | | ID | Title | Description |
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| OG000 | Prophylaxis: Part 1 or Part 2, Exposure Day 1 (Baseline) | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | Prophylaxis: End of Study | | | OG002 | Prophylaxis: Change From Baseline | | | OG003 | On-Demand: Part 1 or Part 2, Exposure Day 1 (Baseline) | On-Demand Dosing and Frequency (Guidance): • Early hemarthrosis, muscle bleed, oral bleed= FIX level required(%) 20-40 IU/dL. Repeat every 24 hours. Duration: at least 1 day, until bleeding episode as indicated by pain is resolved or healing is achieved • More extensive hemarthrosis, muscle bleed, hematoma= FIX level required(%) 30-60 IU/dL. Repeat infusion every 24 hours for 3-4 days or more until pain and acute disability are resolved • Life threatening hemorrhages= FIX level required(%) 60-100 IU/dL. Repeat infusion every 8-24 hours until threat resolves Required dose calculated using IR determined of first 16 participants who completed Part 1 according to: Body weight (kg) x desired FIX rise (%) (IU/dL) x {reciprocal of observed recovery} Given an anticipated recovery of 0.8 [IU/dl]/[IU/kg], required units calculated by: Body weight (kg) x desired FIX rise (%or (IU/dL) x 1.3 IU/kg Amount administered & frequency should be oriented to individual clinical effectiveness |
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| Secondary | General Pain Assessment Through a Visual Analog Scale (VAS) | Participant rated assessment of health-related quality of life. The VAS Pain Scale rates current health state on a scale from 0 (no pain) to 100 (worst imaginable pain). For the pain scale, a higher score indicates worse pain. | | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline at either Study Part 1, or Study Part 2, and End of Study (study weeks 29-31) | | | | ID | Title | Description |
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| OG000 | Prophylaxis: Part 1 or Part 2, Exposure Day 1 (Baseline) | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | Prophylaxis: End of Study | | | OG002 | Prophylaxis: Change From Baseline | | | OG003 | On-Demand: Part 1 or Part 2, Exposure Day 1 (Baseline) | On-Demand Dosing and Frequency (Guidance): • Early hemarthrosis, muscle bleed, oral bleed= FIX level required(%) 20-40 IU/dL. Repeat every 24 hours. Duration: at least 1 day, until bleeding episode as indicated by pain is resolved or healing is achieved • More extensive hemarthrosis, muscle bleed, hematoma= FIX level required(%) 30-60 IU/dL. Repeat infusion every 24 hours for 3-4 days or more until pain and acute disability are resolved • Life threatening hemorrhages= FIX level required(%) 60-100 IU/dL. Repeat infusion every 8-24 hours until threat resolves Required dose calculated using IR determined of first 16 participants who completed Part 1 according to: Body weight (kg) x desired FIX rise (%) (IU/dL) x {reciprocal of observed recovery} Given an anticipated recovery of 0.8 [IU/dl]/[IU/kg], required units calculated by: Body weight (kg) x desired FIX rise (%or (IU/dL) x 1.3 IU/kg Amount administered & frequency should be oriented to individual clinical effectiveness |
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| Secondary | Short Form (36) Health Survey (SF-36): HRQoL 'Physical Component Score' (PCS) | The PCS is a summary scale of the dimensions physical functioning, role physical, bodily pain, and general health. The component score is normalized to a standard population. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. | | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline at either Study Part 1, or Study Part 2, and End of Study (study weeks 29-31) | | | | ID | Title | Description |
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| OG000 | Prophylaxis: Part 1 or Part 2, Exposure Day 1 (Baseline) | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | Prophylaxis: End of Study | | | OG002 | Prophylaxis: Change From Baseline | | | OG003 | On-Demand: Part 1 or Part 2, Exposure Day 1 (Baseline) | On-Demand Dosing and Frequency (Guidance): • Early hemarthrosis, muscle bleed, oral bleed= FIX level required(%) 20-40 IU/dL. Repeat every 24 hours. Duration: at least 1 day, until bleeding episode as indicated by pain is resolved or healing is achieved • More extensive hemarthrosis, muscle bleed, hematoma= FIX level required(%) 30-60 IU/dL. Repeat infusion every 24 hours for 3-4 days or more until pain and acute disability are resolved • Life threatening hemorrhages= FIX level required(%) 60-100 IU/dL. Repeat infusion every 8-24 hours until threat resolves Required dose calculated using IR determined of first 16 participants who completed Part 1 according to: Body weight (kg) x desired FIX rise (%) (IU/dL) x {reciprocal of observed recovery} Given an anticipated recovery of 0.8 [IU/dl]/[IU/kg], required units calculated by: Body weight (kg) x desired FIX rise (%or (IU/dL) x 1.3 IU/kg Amount administered & frequency should be oriented to individual clinical effectiveness |
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| Secondary | SF-36: HRQoL 'Mental Health' (MH) | Quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. | | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline at either Study Part 1, or Study Part 2, and End of Study (study weeks 29-31) | | | | ID | Title | Description |
|---|
| OG000 | Prophylaxis: Part 1 or Part 2, Exposure Day 1 (Baseline) | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | Prophylaxis: End of Study | | | OG002 | Prophylaxis: Change From Baseline | | | OG003 | On-Demand: Part 1 or Part 2, Exposure Day 1 (Baseline) | On-Demand Dosing and Frequency (Guidance): • Early hemarthrosis, muscle bleed, oral bleed= FIX level required(%) 20-40 IU/dL. Repeat every 24 hours. Duration: at least 1 day, until bleeding episode as indicated by pain is resolved or healing is achieved • More extensive hemarthrosis, muscle bleed, hematoma= FIX level required(%) 30-60 IU/dL. Repeat infusion every 24 hours for 3-4 days or more until pain and acute disability are resolved • Life threatening hemorrhages= FIX level required(%) 60-100 IU/dL. Repeat infusion every 8-24 hours until threat resolves Required dose calculated using IR determined of first 16 participants who completed Part 1 according to: Body weight (kg) x desired FIX rise (%) (IU/dL) x {reciprocal of observed recovery} Given an anticipated recovery of 0.8 [IU/dl]/[IU/kg], required units calculated by: Body weight (kg) x desired FIX rise (%or (IU/dL) x 1.3 IU/kg Amount administered & frequency should be oriented to individual clinical effectiveness |
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| Secondary | SF-36: HRQoL Physical Functioning' (PF) | Quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. | | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline at either Study Part 1, or Study Part 2, and End of Study (study weeks 29-31) | | | | ID | Title | Description |
|---|
| OG000 | Prophylaxis: Part 1 or Part 2, Exposure Day 1 (Baseline) | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | Prophylaxis: End of Study | | | OG002 | Prophylaxis: Change From Baseline | | | OG003 | On-Demand: Part 1 or Part 2, Exposure Day 1 (Baseline) | On-Demand Dosing and Frequency (Guidance): • Early hemarthrosis, muscle bleed, oral bleed= FIX level required(%) 20-40 IU/dL. Repeat every 24 hours. Duration: at least 1 day, until bleeding episode as indicated by pain is resolved or healing is achieved • More extensive hemarthrosis, muscle bleed, hematoma= FIX level required(%) 30-60 IU/dL. Repeat infusion every 24 hours for 3-4 days or more until pain and acute disability are resolved • Life threatening hemorrhages= FIX level required(%) 60-100 IU/dL. Repeat infusion every 8-24 hours until threat resolves Required dose calculated using IR determined of first 16 participants who completed Part 1 according to: Body weight (kg) x desired FIX rise (%) (IU/dL) x {reciprocal of observed recovery} Given an anticipated recovery of 0.8 [IU/dl]/[IU/kg], required units calculated by: Body weight (kg) x desired FIX rise (%or (IU/dL) x 1.3 IU/kg Amount administered & frequency should be oriented to individual clinical effectiveness |
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| Secondary | SF-36: HRQoL Role-Physical (RP) | Quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. | | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline at either Study Part 1, or Study Part 2, and End of Study (study weeks 29-31) | | | | ID | Title | Description |
|---|
| OG000 | Prophylaxis: Part 1 or Part 2, Exposure Day 1 (Baseline) | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | Prophylaxis: End of Study | | | OG002 | Prophylaxis: Change From Baseline | | | OG003 | On-Demand: Part 1 or Part 2, Exposure Day 1 (Baseline) | On-Demand Dosing and Frequency (Guidance): • Early hemarthrosis, muscle bleed, oral bleed= FIX level required(%) 20-40 IU/dL. Repeat every 24 hours. Duration: at least 1 day, until bleeding episode as indicated by pain is resolved or healing is achieved • More extensive hemarthrosis, muscle bleed, hematoma= FIX level required(%) 30-60 IU/dL. Repeat infusion every 24 hours for 3-4 days or more until pain and acute disability are resolved • Life threatening hemorrhages= FIX level required(%) 60-100 IU/dL. Repeat infusion every 8-24 hours until threat resolves Required dose calculated using IR determined of first 16 participants who completed Part 1 according to: Body weight (kg) x desired FIX rise (%) (IU/dL) x {reciprocal of observed recovery} Given an anticipated recovery of 0.8 [IU/dl]/[IU/kg], required units calculated by: Body weight (kg) x desired FIX rise (%or (IU/dL) x 1.3 IU/kg Amount administered & frequency should be oriented to individual clinical effectiveness |
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| Secondary | SF-36: HRQoL Role-Emotional | Quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. | | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline at either Study Part 1, or Study Part 2, and End of Study (study weeks 29-31) | | | | ID | Title | Description |
|---|
| OG000 | Prophylaxis: Part 1 or Part 2, Exposure Day 1 (Baseline) | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | Prophylaxis: End of Study | | | OG002 | Prophylaxis: Change From Baseline | | | OG003 | On-Demand: Part 1 or Part 2, Exposure Day 1 (Baseline) | On-Demand Dosing and Frequency (Guidance): • Early hemarthrosis, muscle bleed, oral bleed= FIX level required(%) 20-40 IU/dL. Repeat every 24 hours. Duration: at least 1 day, until bleeding episode as indicated by pain is resolved or healing is achieved • More extensive hemarthrosis, muscle bleed, hematoma= FIX level required(%) 30-60 IU/dL. Repeat infusion every 24 hours for 3-4 days or more until pain and acute disability are resolved • Life threatening hemorrhages= FIX level required(%) 60-100 IU/dL. Repeat infusion every 8-24 hours until threat resolves Required dose calculated using IR determined of first 16 participants who completed Part 1 according to: Body weight (kg) x desired FIX rise (%) (IU/dL) x {reciprocal of observed recovery} Given an anticipated recovery of 0.8 [IU/dl]/[IU/kg], required units calculated by: Body weight (kg) x desired FIX rise (%or (IU/dL) x 1.3 IU/kg Amount administered & frequency should be oriented to individual clinical effectiveness |
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| Secondary | SF-36: HRQoL Bodily Pain | Quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. | | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline at either Study Part 1, or Study Part 2, and End of Study (study weeks 29-31) | | | | ID | Title | Description |
|---|
| OG000 | Prophylaxis: Part 1 or Part 2, Exposure Day 1 (Baseline) | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | Prophylaxis: End of Study | | | OG002 | Prophylaxis: Change From Baseline | | | OG003 | On-Demand: Part 1 or Part 2, Exposure Day 1 (Baseline) | On-Demand Dosing and Frequency (Guidance): • Early hemarthrosis, muscle bleed, oral bleed= FIX level required(%) 20-40 IU/dL. Repeat every 24 hours. Duration: at least 1 day, until bleeding episode as indicated by pain is resolved or healing is achieved • More extensive hemarthrosis, muscle bleed, hematoma= FIX level required(%) 30-60 IU/dL. Repeat infusion every 24 hours for 3-4 days or more until pain and acute disability are resolved • Life threatening hemorrhages= FIX level required(%) 60-100 IU/dL. Repeat infusion every 8-24 hours until threat resolves Required dose calculated using IR determined of first 16 participants who completed Part 1 according to: Body weight (kg) x desired FIX rise (%) (IU/dL) x {reciprocal of observed recovery} Given an anticipated recovery of 0.8 [IU/dl]/[IU/kg], required units calculated by: Body weight (kg) x desired FIX rise (%or (IU/dL) x 1.3 IU/kg Amount administered & frequency should be oriented to individual clinical effectiveness |
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| Secondary | SF-36: HRQoL Mental Health | Quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. | | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline at either Study Part 1, or Study Part 2, and End of Study (study weeks 29-31) | | | | ID | Title | Description |
|---|
| OG000 | Prophylaxis: Part 1 or Part 2, Exposure Day 1 (Baseline) | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | Prophylaxis: End of Study | | | OG002 | Prophylaxis: Change From Baseline | | | OG003 | On-Demand: Part 1 or Part 2, Exposure Day 1 (Baseline) | On-Demand Dosing and Frequency (Guidance): • Early hemarthrosis, muscle bleed, oral bleed= FIX level required(%) 20-40 IU/dL. Repeat every 24 hours. Duration: at least 1 day, until bleeding episode as indicated by pain is resolved or healing is achieved • More extensive hemarthrosis, muscle bleed, hematoma= FIX level required(%) 30-60 IU/dL. Repeat infusion every 24 hours for 3-4 days or more until pain and acute disability are resolved • Life threatening hemorrhages= FIX level required(%) 60-100 IU/dL. Repeat infusion every 8-24 hours until threat resolves Required dose calculated using IR determined of first 16 participants who completed Part 1 according to: Body weight (kg) x desired FIX rise (%) (IU/dL) x {reciprocal of observed recovery} Given an anticipated recovery of 0.8 [IU/dl]/[IU/kg], required units calculated by: Body weight (kg) x desired FIX rise (%or (IU/dL) x 1.3 IU/kg Amount administered & frequency should be oriented to individual clinical effectiveness |
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| Secondary | SF-36: HRQoL Vitality | Quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. | | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline at either Study Part 1, or Study Part 2, and End of Study (study weeks 29-31) | | | | ID | Title | Description |
|---|
| OG000 | Prophylaxis: Part 1 or Part 2, Exposure Day 1 (Baseline) | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | Prophylaxis: End of Study | | | OG002 | Prophylaxis: Change From Baseline | | | OG003 | On-Demand: Part 1 or Part 2, Exposure Day 1 (Baseline) | On-Demand Dosing and Frequency (Guidance): • Early hemarthrosis, muscle bleed, oral bleed= FIX level required(%) 20-40 IU/dL. Repeat every 24 hours. Duration: at least 1 day, until bleeding episode as indicated by pain is resolved or healing is achieved • More extensive hemarthrosis, muscle bleed, hematoma= FIX level required(%) 30-60 IU/dL. Repeat infusion every 24 hours for 3-4 days or more until pain and acute disability are resolved • Life threatening hemorrhages= FIX level required(%) 60-100 IU/dL. Repeat infusion every 8-24 hours until threat resolves Required dose calculated using IR determined of first 16 participants who completed Part 1 according to: Body weight (kg) x desired FIX rise (%) (IU/dL) x {reciprocal of observed recovery} Given an anticipated recovery of 0.8 [IU/dl]/[IU/kg], required units calculated by: Body weight (kg) x desired FIX rise (%or (IU/dL) x 1.3 IU/kg Amount administered & frequency should be oriented to individual clinical effectiveness |
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| Secondary | SF-36: HRQoL Social Functioning | Quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. | | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline at either Study Part 1, or Study Part 2, and End of Study (study weeks 29-31) | | | | ID | Title | Description |
|---|
| OG000 | Prophylaxis: Part 1 or Part 2, Exposure Day (ED) 1 (Baseline) | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | Prophylaxis: End of Study | | | OG002 | Prophylaxis: Change From Baseline | | | OG003 | On-Demand: Part 1 or Part 2, Exposure Day (ED) 1 (Baseline) | On-Demand Dosing and Frequency (Guidance): • Early hemarthrosis, muscle bleed, oral bleed= FIX level required(%) 20-40 IU/dL. Repeat every 24 hours. Duration: at least 1 day, until bleeding episode as indicated by pain is resolved or healing is achieved • More extensive hemarthrosis, muscle bleed, hematoma= FIX level required(%) 30-60 IU/dL. Repeat infusion every 24 hours for 3-4 days or more until pain and acute disability are resolved • Life threatening hemorrhages= FIX level required(%) 60-100 IU/dL. Repeat infusion every 8-24 hours until threat resolves Required dose calculated using IR determined of first 16 participants who completed Part 1 according to: Body weight (kg) x desired FIX rise (%) (IU/dL) x {reciprocal of observed recovery} Given an anticipated recovery of 0.8 [IU/dl]/[IU/kg], required units calculated by: Body weight (kg) x desired FIX rise (%or (IU/dL) x 1.3 IU/kg Amount administered & frequency should be oriented to individual clinical effectiveness |
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| Secondary | SF-36: HRQoL General Health | Quality of life survey response as measured using the SF-36 questionnaire. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both subscores and summary scores. The raw data from the SF-36 items were transformed to norm based scores for each of the 8 HRQoL/SF-36 health domain scores. | | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline at either Study Part 1, or Study Part 2, and End of Study (study weeks 29-31) | | | | ID | Title | Description |
|---|
| OG000 | Prophylaxis: Part 1 or Part 2, Exposure Day 1 (Baseline) | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | Prophylaxis: End of Study | | | OG002 | Prophylaxis: Change From Baseline | | | OG003 | On-Demand: Part 1 or Part 2, Exposure Day 1 (Baseline) | On-Demand Dosing and Frequency (Guidance): • Early hemarthrosis, muscle bleed, oral bleed= FIX level required(%) 20-40 IU/dL. Repeat every 24 hours. Duration: at least 1 day, until bleeding episode as indicated by pain is resolved or healing is achieved • More extensive hemarthrosis, muscle bleed, hematoma= FIX level required(%) 30-60 IU/dL. Repeat infusion every 24 hours for 3-4 days or more until pain and acute disability are resolved • Life threatening hemorrhages= FIX level required(%) 60-100 IU/dL. Repeat infusion every 8-24 hours until threat resolves Required dose calculated using IR determined of first 16 participants who completed Part 1 according to: Body weight (kg) x desired FIX rise (%) (IU/dL) x {reciprocal of observed recovery} Given an anticipated recovery of 0.8 [IU/dl]/[IU/kg], required units calculated by: Body weight (kg) x desired FIX rise (%or (IU/dL) x 1.3 IU/kg Amount administered & frequency should be oriented to individual clinical effectiveness |
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| Secondary | Pediatric Quality of Life Questionnaire (PedsQL) Physical Health Summary Score (Ages 12-16) | The Peds-QL is a generic Health-Related Quality of Life (HR QoL) instrument designed specifically for a pediatric population. It captures the following domains: general health/activities, feelings/emotional, social functioning, school functioning. For this study, the Peds-QL for 12 to 16-year-old subjects was used. Higher scores indicate better quality of life (QOL) for all domains of the Peds-QL. This modular instrument uses a 5-point scale: from 0 (never) to 4 (almost always). Items are reversed scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0. 4 dimensions (physical, emotional, social, & school functioning) are scored. | | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline at either Study Part 1, or Study Part 2, and End of Study (study weeks 29-31) | | | | ID | Title | Description |
|---|
| OG000 | Prophylaxis: Part 1 or Part 2, Exposure Day 1 (Baseline) | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | Prophylaxis: End of Study | | | OG002 | Prophylaxis: Change From Baseline | | |
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| Secondary | Pediatric Quality of Life Questionnaire (PedsQL) Psychosocial Health Summary Score (Ages 12-16) | The Peds-QL is a generic Health-Related Quality of Life (HR QoL) instrument designed specifically for a pediatric population. It captures the following domains: general health/activities, feelings/emotional, social functioning, school functioning. For this study, the Peds-QL for 12 to 16-year-old subjects was used. Higher scores indicate better quality of life (QOL) for all domains of the Peds-QL. This modular instrument uses a 5-point scale: from 0 (never) to 4 (almost always). Items are reversed scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0. 4 dimensions (physical, emotional, social, & school functioning) are scored. | | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline at either Study Part 1, or Study Part 2, and End of Study (study weeks 29-31) | | | | ID | Title | Description |
|---|
| OG000 | Prophylaxis: Part 1 or Part 2, Exposure Day 1 (Baseline) | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | Prophylaxis: End of Study | | | OG002 | Prophylaxis: Change From Baseline | | |
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| Secondary | Pediatric Quality of Life Questionnaire (PedsQL) Total Score (Ages 12-16) | The Peds-QL is a generic Health-Related Quality of Life (HR QoL) instrument designed specifically for a pediatric population. It captures the following domains: general health/activities, feelings/emotional, social functioning, school functioning. For this study, the Peds-QL for 12 to 16-year-old subjects was used. Higher scores indicate better quality of life (QOL) for all domains of the Peds-QL. This modular instrument uses a 5-point scale: from 0 (never) to 4 (almost always). Items are reversed scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0. 4 dimensions (physical, emotional, social, & school functioning) are scored. | | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline at either Study Part 1, or Study Part 2, and End of Study (study weeks 29-31) | | | | ID | Title | Description |
|---|
| OG000 | Prophylaxis: Part 1 or Part 2, Exposure Day 1 (Baseline) | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | Prophylaxis: End of Study | | | OG002 | Prophylaxis: Change From Baseline | | | OG003 |
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| Secondary | Health-Related Quality of Life (HRQoL) Disease-specific: Haem-A-QoL | The Haem-A-QOL instrument has been developed and used in hemophilia A patients. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. The areas covered by this instrument are: physical health, sports/leisure, school/work, dealing with hemophilia, and outlook for the future. For the Haem-A-QOL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100. | | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline at either Study Part 1, or Study Part 2, and End of Study (study weeks 29-31) | | | | ID | Title | Description |
|---|
| OG000 | Prophylaxis: Part 1 or Part 2, Exposure Day 1 (Baseline) | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | Prophylaxis: End of Study | | | OG002 | Prophylaxis: Change From Baseline | | | OG003 | On-Demand: Part 1 or Part 2, Exposure Day 1 (Baseline) | On-Demand Dosing and Frequency (Guidance): • Early hemarthrosis, muscle bleed, oral bleed= FIX level required(%) 20-40 IU/dL. Repeat every 24 hours. Duration: at least 1 day, until bleeding episode as indicated by pain is resolved or healing is achieved • More extensive hemarthrosis, muscle bleed, hematoma= FIX level required(%) 30-60 IU/dL. Repeat infusion every 24 hours for 3-4 days or more until pain and acute disability are resolved • Life threatening hemorrhages= FIX level required(%) 60-100 IU/dL. Repeat infusion every 8-24 hours until threat resolves Required dose calculated using IR determined of first 16 participants who completed Part 1 according to: Body weight (kg) x desired FIX rise (%) (IU/dL) x {reciprocal of observed recovery} Given an anticipated recovery of 0.8 [IU/dl]/[IU/kg], required units calculated by: Body weight (kg) x desired FIX rise (%or (IU/dL) x 1.3 IU/kg Amount administered & frequency should be oriented to individual clinical effectiveness |
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| Secondary | Health-Related Quality of Life (HRQoL) Disease-specific: Haemo-QoL - Participants On-Demand (Ages 12-16) | The Haemo-QoL is a quality of life (QoL) assessment instrument for children and adolescents with haemophilia. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. For the Haemo-QoL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100. | | Posted | | Mean | Standard Deviation | Score on a scale | | Baseline at either Study Part 1, or Study Part 2, and End of Study (study weeks 29-31) | | | | ID | Title | Description |
|---|
| OG000 | Part 1 or Part 2, Exposure Day 1 (Baseline) | | | OG001 | End of Study | | | OG002 | Change From Baseline | |
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| Secondary | Health Resource Use - Number of Hospitalizations | | | Posted | | Median | Full Range | Hospitalizations | | Study Part 1 = 2-4 weeks, Study Part 2 = 26 weeks ± 1 week, Study Part 3 = 1 week (Total = 29-31 weeks) | | | | ID | Title | Description |
|---|
| OG000 | Prophylaxis | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | On-Demand | On-Demand Dosing and Frequency (Guidance): • Early hemarthrosis, muscle bleed, oral bleed= FIX level required(%) 20-40 IU/dL. Repeat every 24 hours. Duration: at least 1 day, until bleeding episode as indicated by pain is resolved or healing is achieved • More extensive hemarthrosis, muscle bleed, hematoma= FIX level required(%) 30-60 IU/dL. Repeat infusion every 24 hours for 3-4 days or more until pain and acute disability are resolved • Life threatening hemorrhages= FIX level required(%) 60-100 IU/dL. Repeat infusion every 8-24 hours until threat resolves Required dose calculated using IR determined of first 16 participants who completed Part 1 according to: Body weight (kg) x desired FIX rise (%) (IU/dL) x {reciprocal of observed recovery} Given an anticipated recovery of 0.8 [IU/dl]/[IU/kg], required units calculated by: Body weight (kg) x desired FIX rise (%or (IU/dL) x 1.3 IU/kg Amount administered & frequency should be oriented to individual clinical effectiveness |
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| Secondary | Health Resource Use - Total Days of Hospital Stay | | | Posted | | Median | Full Range | Days | | Study Part 1 = 2-4 weeks, Study Part 2 = 26 weeks ± 1 week, Study Part 3 = 1 week (Total = 29-31 weeks) | | | | ID | Title | Description |
|---|
| OG000 | Prophylaxis | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. |
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| Secondary | Health Resource Use - Emergency Room Visits | | | Posted | | Median | Full Range | Visits | | Study Part 1 = 2-4 weeks, Study Part 2 = 26 weeks ± 1 week, Study Part 3 = 1 week (Total = 29-31 weeks) | | | | ID | Title | Description |
|---|
| OG000 | Prophylaxis | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | On-Demand | On-Demand Dosing and Frequency (Guidance): • Early hemarthrosis, muscle bleed, oral bleed= FIX level required(%) 20-40 IU/dL. Repeat every 24 hours. Duration: at least 1 day, until bleeding episode as indicated by pain is resolved or healing is achieved • More extensive hemarthrosis, muscle bleed, hematoma= FIX level required(%) 30-60 IU/dL. Repeat infusion every 24 hours for 3-4 days or more until pain and acute disability are resolved • Life threatening hemorrhages= FIX level required(%) 60-100 IU/dL. Repeat infusion every 8-24 hours until threat resolves Required dose calculated using IR determined of first 16 participants who completed Part 1 according to: Body weight (kg) x desired FIX rise (%) (IU/dL) x {reciprocal of observed recovery} Given an anticipated recovery of 0.8 [IU/dl]/[IU/kg], required units calculated by: Body weight (kg) x desired FIX rise (%or (IU/dL) x 1.3 IU/kg Amount administered & frequency should be oriented to individual clinical effectiveness |
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| Secondary | Health Resource Use - Unscheduled Doctor's Office Visits | | | Posted | | Median | Full Range | Visits | | Study Part 1 = 2-4 weeks, Study Part 2 = 26 weeks ± 1 week, Study Part 3 = 1 week (Total = 29-31 weeks) | | | | ID | Title | Description |
|---|
| OG000 | Prophylaxis | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | On-Demand | On-Demand Dosing and Frequency (Guidance): • Early hemarthrosis, muscle bleed, oral bleed= FIX level required(%) 20-40 IU/dL. Repeat every 24 hours. Duration: at least 1 day, until bleeding episode as indicated by pain is resolved or healing is achieved • More extensive hemarthrosis, muscle bleed, hematoma= FIX level required(%) 30-60 IU/dL. Repeat infusion every 24 hours for 3-4 days or more until pain and acute disability are resolved • Life threatening hemorrhages= FIX level required(%) 60-100 IU/dL. Repeat infusion every 8-24 hours until threat resolves Required dose calculated using IR determined of first 16 participants who completed Part 1 according to: Body weight (kg) x desired FIX rise (%) (IU/dL) x {reciprocal of observed recovery} Given an anticipated recovery of 0.8 [IU/dl]/[IU/kg], required units calculated by: Body weight (kg) x desired FIX rise (%or (IU/dL) x 1.3 IU/kg Amount administered & frequency should be oriented to individual clinical effectiveness |
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| Secondary | Health Resource Use - Days Lost From Work or School | | | Posted | | Median | Full Range | Days | | Study Part 1 = 2-4 weeks, Study Part 2 = 26 weeks ± 1 week, Study Part 3 = 1 week (Total = 29-31 weeks) | | | | ID | Title | Description |
|---|
| OG000 | Prophylaxis | Prophylactic treatment of 50 IU/kg BAX326 twice weekly. The dose range is 40-60 IU/kg and may be increased up to 75 IU/kg, if required. | | OG001 | On-Demand | On-Demand Dosing and Frequency (Guidance): • Early hemarthrosis, muscle bleed, oral bleed= FIX level required(%) 20-40 IU/dL. Repeat every 24 hours. Duration: at least 1 day, until bleeding episode as indicated by pain is resolved or healing is achieved • More extensive hemarthrosis, muscle bleed, hematoma= FIX level required(%) 30-60 IU/dL. Repeat infusion every 24 hours for 3-4 days or more until pain and acute disability are resolved • Life threatening hemorrhages= FIX level required(%) 60-100 IU/dL. Repeat infusion every 8-24 hours until threat resolves Required dose calculated using IR determined of first 16 participants who completed Part 1 according to: Body weight (kg) x desired FIX rise (%) (IU/dL) x {reciprocal of observed recovery} Given an anticipated recovery of 0.8 [IU/dl]/[IU/kg], required units calculated by: Body weight (kg) x desired FIX rise (%or (IU/dL) x 1.3 IU/kg Amount administered & frequency should be oriented to individual clinical effectiveness |
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