A Phase 2/3 interventional study of Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method and PEGylated Recombinant Factor VIII in Hemophilia A, sponsored by Baxalta now part of Shire. Completed at 72 sites in 20 countries. Open to male participants aged 12 Years to 65 Years. Per ClinicalTrials.gov, last updated 2021-05-20.
Sponsored by Baxalta now part of Shire (part of Takeda) · Phase 2/3, Interventional, and Prevention
To assess efficacy and safety, including immunogenicity of BAX 855 administered as prophylaxis and as on-demand therapy in adult and adolescent (12-65 years) previously treated patients (PTPs) with severe hemophilia A To determine the pharmacokinetic (PK) parameters of BAX 855.
885 studies on the registry are indexed under Hemophilia A; 139 are open to participants now.
This study's enrollment of 159 is above the median of 27 across 523 interventional studies indexed under Hemophilia A.
Browse Hemophilia A studies →Baxalta now part of Shire is the lead sponsor of 111 studies on the registry; none are open to participants now.
Of its 18 completed or terminated interventional studies of FDA-regulated products, 16 (89%) have results posted.
First submitted before Baxalta became part of Takeda.
Counted across the registry records on this site, refreshed daily.
Main Inclusion Criteria:
Main Exclusion Criteria:
Biological: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method · Biological: PEGylated Recombinant Factor VIII
Biological: PEGylated Recombinant Factor VIII
Drug page: Antihemophilic factor, human recombinant
Pharmacokinetic (PK) evaluation of ADVATE
Also known as: ADVATE
Pharmacokinetic (PK) evaluation of BAX 855
Also known as: BAX 855
Prophylaxis treatment
Also known as: BAX 855
On-demand treatment
Also known as: BAX 855
Annualized Bleeding Rate (ABR)
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.
Time frame: 9 months
Rate of Success of BAX 855 for Treatment of Bleeding Episodes
Success in the control of bleeding was defined as a rating of excellent or good using the Efficacy Rating Scale for Treatment of Bleeding Episodes measured 24 hours after initiation of treatment for the bleeding episode. 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 is required for the control of bleeding. Administration of further infusions to maintain hemostasis would 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 a single infusion. Required more than 1 infusion for complete resolution. NONE: No improvement or condition worsens.
Time frame: At least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm.
Average Number of BAX 855 Infusions Needed for the Treatment of Bleeding Episodes
Time frame: From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].
Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes
Interval between Bleeds in months was calculated as: Observation period for efficacy (in days)/(number of bleeds)\*(12/365.2425)
Time frame: From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].
Weight-adjusted Consumption of BAX 855 - Per Prophylactic Infusion and Pharmacokinetic (PK) Infusion
Time frame: Prophylactic Infusion: ≥50 exposure days or 6 months (±2 weeks), whichever occurs last. PK Infusion: PK #1 Pre-infusion within 30 minutes; Post-infusion 10 min, and 0.5, 1, 3, 6, 24, 32, 48, 56 hours (h). PK #2 also at Post-infusion 96h
Weight-adjusted Consumption of BAX 855 - Per Treatment of Bleeding Episode (BE) and Per BE for Maintenance of Hemostasis
Infusions per bleeding episode for maintenance of hemostasis only includes infusions following the resolution of a bleed to maintain hemostasis.
Time frame: Treatment of Bleeding Episode (BE): Minor/Moderate BE every 12 to 24 hours until bleeding is resolved; Major BE every 8 to 12 hours until bleeding is resolved. Per BE for Maintenance of Hemostasis: within 48 hours after bleeding episode resolution.
Percentage of Participants With Adverse Events
Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time frame: From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].
Immunogenicity - Number of Participants With Positive Inhibitory Antibodies to FVIII, Binding Antibodies to FVIII, PEG-VIII, PEG and Anti-CHO Antibodies at Study Completion/Termination
Number of participants who received BAX855, with immunogenicity data from study completion/termination visit. FVIII = factor VIII; PEG-VIII = polyethylene glycol-factor VIII; Anti-CHO = Anti-Chinese hamster ovary
Time frame: From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].
Patient Reported Outcomes: Haemo-SYM Questionnaire, Change in Score From Baseline to End of Study
The HAEMO-SYM has two subscales: pain and bleeds. HAEMO-SYM subscale scores are calculated by taking the mean of the items in each subscale and transforming them to a 0 (none or absent) to 100 (very severe) scale. Given that higher scores indicate more severe symptoms on the Haemo-SYM and that the change scores were calculated as the value at study completion minus the value at baseline, a negative change score indicates an improvement (reduction in symptoms). Conversely, a positive change score indicates worsening symptoms.
Time frame: Baseline; and end of study visit [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm].
Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study
Change from Baseline to End of Study for SF-36 Questionnaire is provided. Scores for individual SF-36 categories range from 0 to 100 with higher scores representing better health. Given that higher scores indicate better health-related quality of life (HRQoL) and that the change scores were calculated as the value at study completion minus the value at baseline, a negative change score indicates a worsening of HRQoL.
Time frame: Baseline; and end of study visit [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm]
Pharmacokinetics (Pk) - Plasma Half-life (One-stage Clotting Assay)
Terminal half-life calculated as log\_e2/λz where λz is the terminal elimination rate constant. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
Time frame: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).
Pharmacokinetics (Pk) - Mean Residence Time (One-stage Clotting Assay)
The mean residence time (MRT) w as calculated as total area under the moment curve divided by the total area under the curve starting from the begin of infusion (or the end of infusion if start time is not available). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
Time frame: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).
Pharmacokinetics (Pk) - Total Body Clearance (One-stage Clotting Assay)
Clearance in dL/(kg.h) will be calculated as the dose in IU/kg divided by the total area under the curve starting from the begin of infusion (or the end of infusion if start time is not available). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
Time frame: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).
Pharmacokinetics (Pk) - Incremental Recovery Over Time (One-stage Clotting Assay)
Incremental recovery (IR) in (IU/dL)/ (IU/kg) calculated as: IR = (Cmax- (C pre-infusion)) / (Dose/kg), where C =concentration. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
Time frame: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).
Pharmacokinetics (Pk) - Area Under the Concentration Versus Time Curve From 0 to Infinity (AUC0-∞) (One-stage Clotting Assay)
Calculated by WinNonlin NCA (Model 201, calculation method: Linear Trapezoidal Linear/Log Interpolation). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
Time frame: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).
Pharmacokinetics (Pk) - Apparent Volume of Distribution at Steady State (Vss) (One-stage Clotting Assay)
The apparent volume of distribution at steady state (Vss) will be calculated as: Vss = Clearance \* Mean Residence Time. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
Time frame: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).
Pharmacokinetics (Pk) - Maximum Plasma Concentration (Cmax) (One-stage Clotting Assay)
Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
Time frame: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).
Pharmacokinetics (Pk) -Time to Maximum Concentration in Plasma (Tmax) (One-stage Clotting Assay)
Tmax in hours will be defined as the time to reach Cmax. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
Time frame: Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).
Change in Vital Signs From Screening - Temperature
Time frame: Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination
Change in Vital Signs From Screening - Pulse Rate
Time frame: Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination
Change in Vital Signs From Screening - Respiratory Rate
Time frame: Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination
Changes in Vital Signs From Screening - Blood Pressure
Systolic Blood Pressure (SBP) Diastolic Blood Pressure (DBP)
Time frame: Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination
Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein
Time frame: Screening, week 2, week 4, month 3, study completion/termination
Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase
Alkaline Phosphatase (Alk Phos); Alanine Aminotransferase (Ala Amino); Aspartate Aminotransferase (Asp Amino)
Time frame: Screening, week 2, week 4, month 3, study completion/termination
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)
Time frame: Screening, week 2, week 4, month 3, study completion/termination
Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin
Time frame: Screening, week 2, week 4, month 3, study completion/termination
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes
Time frame: Screening, week 2, week 4, month 3, study completion/termination
Changes in Hematology Laboratory Assessments From Screening - Hematocrit
Time frame: Screening, week 2, week 4, month 3, study completion/termination
Changes in Hematology Laboratory Assessments From Screening - Hemoglobin
Time frame: Screening, week 2, week 4, month 3, study completion/termination
Changes in Hematology Laboratory Assessments From Screening - Erythrocytes
Time frame: Screening, week 2, week 4, month 3, study completion/termination
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)
Time frame: Screening, week 2, week 4, month 3, study completion/termination
Participants were enrolled (signed informed consent) at 72 sites.
| Milestone | Prophylaxis | On-demand |
|---|---|---|
| Started | 121 | 17 |
| Completed | 109 | 17 |
| Not completed | 12 | 0 |
| Withdrew: Adverse event | 4 | 0 |
| Withdrew: Withdrawal by subject | 2 | 0 |
| Withdrew: Protocol violation | 4 | 0 |
| Withdrew: Other - screen failure | 1 | 0 |
| Withdrew: Other - surgical procedure | 1 | 0 |
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.
| Bleeds per year | Prophylaxis | On-demand |
|---|---|---|
| Annualized Bleeding Rate (ABR) | 4.3 (3.4 to 5.5) | 43.4 (25.2 to 74.8) |
Success in the control of bleeding was defined as a rating of excellent or good using the Efficacy Rating Scale for Treatment of Bleeding Episodes measured 24 hours after initiation of treatment for the bleeding episode. 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 is required for the control of bleeding. Administration of further infusions to maintain hemostasis would 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 a single infusion. Required more than 1 infusion for complete resolution. NONE: No improvement or condition worsens.
| Bleeding episodes | Participants With a Bleeding Episode |
|---|---|
| Rate of Success of BAX 855 for Treatment of Bleeding Episodes | 0.96 (0.91 to 0.98) |
| Infusions | Prophylaxis | On-demand |
|---|---|---|
| Average Number of BAX 855 Infusions Needed for the Treatment of Bleeding Episodes | 1.37 ± 0.80 | 1.21 ± 0.35 |
Interval between Bleeds in months was calculated as: Observation period for efficacy (in days)/(number of bleeds)\*(12/365.2425)
| Participants | Prophylaxis | On-demand |
|---|---|---|
| No bleed | 45 | 0 |
| >6 Months | 5 | 0 |
| 6 Months | 20 | 0 |
| 5 Months | 3 | 0 |
| 4 Months | 0 | 0 |
| 3 Months | 11 | 0 |
| 2 Months | 16 | 0 |
| ≤1 Month | 20 | 17 |
| IU/kg | All Study Participants |
|---|---|
| Per Prophylactic Infusion (N= 5941 Infusions) | 44.51 ± 4.556 |
| Per PK Infusion (N= 50 Infusions) | 45.48 ± 2.592 |
Infusions per bleeding episode for maintenance of hemostasis only includes infusions following the resolution of a bleed to maintain hemostasis.
| IU/kg | All Study Participants |
|---|---|
| Per Treatment of BE (N= 592 bleeds) | 37.44 ± 28.105 |
| Per BE for Maintenance of Hemostasis (N=34 bleeds) | 39.29 ± 34.206 |
Adverse Events (AEs) and Serious Adverse Events (SAEs)
| percent of participants | All Study Participants |
|---|---|
| SAE, Moderate, Unrelated | 0.7 |
| SAE, Severe, Unrelated | 2.9 |
| nSAE, Mild, Unrelated | 40.1 |
| nSAE, Mild, Related | 3.6 |
| nSAE, Moderate, Unrelated | 19.0 |
| nSAE, Moderate, Related | 1.5 |
| nSAE, Unknown Severity, Unrelated | 0.7 |
| nSAE, Severe, Unrelated | 1.5 |
Number of participants who received BAX855, with immunogenicity data from study completion/termination visit. FVIII = factor VIII; PEG-VIII = polyethylene glycol-factor VIII; Anti-CHO = Anti-Chinese hamster ovary
| Participants | Prophylaxis | On-demand |
|---|---|---|
| Inhibitory Antibodies to FVIII (N= 112, 14) | 0 | 0 |
| IgG: Binding Antibodies FVIII (N= 117, 15) | 0 | 0 |
| IgM: Binding Antibodies FVIII (N= 117, 15) | 0 | 0 |
| IgG: Binding Antibodies PEG (N= 117, 15) | 0 | 0 |
| IgM: Binding Antibodies PEG (N= 117, 15) | 0 | 0 |
| IgG: Binding Antibodies PEG-FVIII (N= 117, 15) | 0 | 1 |
| IgM: Binding Antibodies PEG-FVIII (N= 117, 15) | 0 | 0 |
| CHO-Protein Antibodies (N= 117, 15) | 0 | 0 |
The HAEMO-SYM has two subscales: pain and bleeds. HAEMO-SYM subscale scores are calculated by taking the mean of the items in each subscale and transforming them to a 0 (none or absent) to 100 (very severe) scale. Given that higher scores indicate more severe symptoms on the Haemo-SYM and that the change scores were calculated as the value at study completion minus the value at baseline, a negative change score indicates an improvement (reduction in symptoms). Conversely, a positive change score indicates worsening symptoms.
| Score on a scale | Prophylaxis | On-demand |
|---|---|---|
| Bleed Severity Total Score | -4.17 ± 17.05 | -4.24 ± 15.71 |
| Pain Severity Total Score | -1.22 ± 12.50 | -0.17 ± 11.88 |
Change from Baseline to End of Study for SF-36 Questionnaire is provided. Scores for individual SF-36 categories range from 0 to 100 with higher scores representing better health. Given that higher scores indicate better health-related quality of life (HRQoL) and that the change scores were calculated as the value at study completion minus the value at baseline, a negative change score indicates a worsening of HRQoL.
| Score on a scale | Prophylaxis | On-demand |
|---|---|---|
| Physical Functioning (N= 97, 12) | 0.49 ± 5.27 | -2.46 ± 4.29 |
| Role-physical (N= 97, 12) | 1.31 ± 7.36 | -3.67 ± 9.01 |
| Bodily Pain (N= 97, 12) | 2.08 ± 8.19 | 0.60 ± 4.44 |
| General Health (N= 96, 12) | 0.40 ± 6.43 | -0.28 ± 9.04 |
| Vitality (N= 96, 12) | -0.38 ± 7.43 | 0.26 ± 9.36 |
| Social Functioning (N= 97, 12) | 0.90 ± 7.54 | -3.18 ± 6.35 |
| Role Emotional (N= 97, 12) | -0.20 ± 8.46 | 0.65 ± 7.92 |
| Mental Health (N= 96, 12) | 0.09 ± 7.26 | -3.29 ± 7.95 |
| Physical Component Score (N= 96, 12) | 1.36 ± 5.76 | -1.58 ± 4.97 |
| Mental Component Score (N= 96, 12) | -0.37 ± 7.38 | -1.14 ± 5.03 |
Terminal half-life calculated as log\_e2/λz where λz is the terminal elimination rate constant. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
| hours | Pharmacokinetic Analysis Participants |
|---|---|
| PK-1: ADVATE | 10.40 ± 2.244 |
| PK-2: BAX 855 | 14.30 ± 3.838 |
| PK-3: BAX 855, After ≥50 Exposure Days (N=22) | 16.02 ± 4.922 |
The mean residence time (MRT) w as calculated as total area under the moment curve divided by the total area under the curve starting from the begin of infusion (or the end of infusion if start time is not available). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
| hours | Pharmacokinetic Analysis Participants |
|---|---|
| PK-1: ADVATE | 12.86 ± 3.044 |
| PK-2: BAX 855 | 19.56 ± 5.315 |
| PK-3: BAX 855, After ≥50 Exposure Days (N=22) | 20.65 ± 4.821 |
Clearance in dL/(kg.h) will be calculated as the dose in IU/kg divided by the total area under the curve starting from the begin of infusion (or the end of infusion if start time is not available). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
| dL/(kg*hours) | Pharmacokinetic Analysis Participants |
|---|---|
| PK-1: ADVATE | 0.04551 ± 0.021725 |
| PK-2: BAX 855 | 0.02760 ± 0.020288 |
| PK-3: BAX 855, After ≥50 Exposure Days (N=22) | 0.02474 ± 0.008225 |
Incremental recovery (IR) in (IU/dL)/ (IU/kg) calculated as: IR = (Cmax- (C pre-infusion)) / (Dose/kg), where C =concentration. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
| (IU/dL)/(IU/kg) | Pharmacokinetic Analysis Participants |
|---|---|
| PK-1: ADVATE | 2.372 ± 0.5357 |
| PK-2: BAX 855 | 2.493 ± 0.6944 |
| PK-3: BAX 855, After ≥50 Exposure Days (N=22) | 2.297 ± 0.6377 |
Calculated by WinNonlin NCA (Model 201, calculation method: Linear Trapezoidal Linear/Log Interpolation). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
| (IU*hours)/dL | Pharmacokinetic Analysis Participants |
|---|---|
| PK-1: ADVATE | 1168.0 ± 425.40 |
| PK-2: BAX 855 | 2073.3 ± 778.41 |
| PK-3: BAX 855, After ≥50 Exposure Days (N=22) | 2008.7 ± 631.53 |
The apparent volume of distribution at steady state (Vss) will be calculated as: Vss = Clearance \* Mean Residence Time. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
| dL/kg | Pharmacokinetic Analysis Participants |
|---|---|
| PK-1: ADVATE | 0.5487 ± 0.20213 |
| PK-2: BAX 855 | 0.4715 ± 0.14602 |
| PK-3: BAX 855, After ≥50 Exposure Days (N=22) | 0.4970 ± 0.15756 |
Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
| IU/dL | Pharmacokinetic Analysis Participants |
|---|---|
| PK-1: ADVATE | 108.45 ± 26.250 |
| PK-2: BAX 855 | 113.68 ± 30.259 |
| PK-3: BAX 855, After ≥50 Exposure Days (N=22) | 103.34 ± 29.311 |
Tmax in hours will be defined as the time to reach Cmax. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).
| hours | Pharmacokinetic Analysis Participants |
|---|---|
| PK-1: ADVATE | 0.296 ± 0.1662 |
| PK-2: BAX 855 | 0.397 ± 0.2632 |
| PK-3: BAX 855, After ≥50 Exposure Days (N=22) | 0.467 ± 0.6044 |
| Celsius | Prophylaxis | On-demand |
|---|---|---|
| Week 2 (N= 118, 17) | 0.00 (-0.20 to 0.20) | 0.00 (0.00 to 0.20) |
| Week 4 (N= 118, 15) | 0.00 (-0.30 to 0.20) | 0.00 (-0.40 to 0.10) |
| Exposure day 10-15 (N= 31, 10) | -0.10 (-0.30 to 0.10) | 0.00 (-0.30 to 0.30) |
| Month 3 (N= 112, 17) | 0.00 (-0.20 to 0.20) | 0.00 (-0.10 to 0.10) |
| Completion/Termination (N= 116, 15) | 0.00 (-0.20 to 0.20) | 0.00 (-0.10 to 0.30) |
| beats per minute | Prophylaxis | On-demand |
|---|---|---|
| Week 2 (N= 118, 17) | 3.0 (-3.0 to 9.0) | 2.0 (-3.0 to 5.0) |
| Week 4 (N= 117, 15) | 2.0 (-4.0 to 10.0) | 2.0 (-5.0 to 4.0) |
| Exposure day 10-15 (N= 31, 10) | 3.0 (-3.0 to 10.0) | 4.0 (0.0 to 5.0) |
| Month 3 (N= 112, 17) | 2.0 (-5.5 to 6.5) | 1.0 (-6.0 to 8.0) |
| Completion/Termination (N= 116, 15) | 2.0 (-6.0 to 8.5) | 3.0 (-5.0 to 10.0) |
| breaths per minute | Prophylaxis | On-deamand |
|---|---|---|
| Week 2 (N= 117, 17) | 0.0 (-1.0 to 1.0) | 0.0 (-1.0 to 2.0) |
| Week 4 (N= 118, 15) | 0.0 (-1.0 to 1.0) | 0.0 (-1.0 to 2.0) |
| Exposure day 10-15 (N= 31, 10) | 0.0 (-1.0 to 2.0) | 0.0 (-1.0 to 0.0) |
| Month 3 (N= 112, 16) | 0.0 (-2.0 to 2.0) | 0.0 (-2.0 to 1.0) |
| Completion/Termination (N= 115, 15) | 0.0 (-2.0 to 1.0) | -1.0 (-2.0 to 2.0) |
Systolic Blood Pressure (SBP) Diastolic Blood Pressure (DBP)
| mmHg | Prophylaxis | On-demand |
|---|---|---|
| SBP-Week 2 (N= 118, 17) | 0.0 (-7.0 to 6.0) | -2.0 (-9.0 to 7.0) |
| SBP-Week 4 (N= 118, 15) | -0.5 (-9.0 to 7.0) | -1.0 (-5.0 to 0.0) |
| SBP-Exposure day 10-15 (N= 31, 10) | 0.0 (-5.0 to 4.0) | 3.5 (0.0 to 6.0) |
| SBP-Month 3 (N= 112, 17) | 0.0 (-7.0 to 7.5) | -5.0 (-12.0 to 4.0) |
| SBP-Completion/Termination (N= 116, 15) | 0.0 (-10.0 to 7.5) | 0.0 (-8.0 to 4.0) |
| DBP-Week 2 (N= 118, 17) | 0.0 (-8.0 to 5.0) | -2.0 (-9.0 to 5.0) |
| DBP-Week 4 (N= 118, 15) | -0.5 (-6.0 to 5.0) | -6.0 (-12.0 to 7.0) |
| DBP-Exposure day 10-15 (N= 31, 10) | 0.0 (-9.0 to 3.0) | -2.5 (-6.0 to 5.0) |
| DBP-Month 3 (N= 112, 17) | 0.0 (-7.0 to 7.5) | -4.0 (-9.0 to 5.0) |
| DBP-Completion/Termination (N= 116, 15) | 0.0 (-6.0 to 5.0) | -2.0 (-10.0 to 5.0) |
| g/L | Prophylaxis | On-demand |
|---|---|---|
| Albumin: Week 2 (N= 112, 17) | -1.0 (-2.5 to 1.0) | -1.0 (-2.0 to 0.0) |
| Albumin: Week 4 (N= 112, 14) | -1.0 (-3.0 to 0.5) | -1.0 (-2.0 to 0.0) |
| Albumin: Month 3 (N= 106, 17) | -1.0 (-3.0 to 1.0) | -1.0 (-2.0 to 1.0) |
| Albumin: Completion/Termination (N= 112, 14) | 0.0 (-2.0 to 2.0) | -1.0 (-2.0 to -1.0) |
| Protein: Week 2 (N= 115, 17) | -1.0 (-4.0 to 1.0) | -2.0 (-4.0 to 0.0) |
| Protein: Week 4 (N= 114, 14) | -1.0 (-4.0 to 1.0) | -2.0 (-5.0 to -1.0) |
| Protein: Month 3 (N= 109, 17) | -1.0 (-3.0 to 2.0) | -1.0 (-3.0 to 2.0) |
| Protein: Completion/Termination (N= 114, 15) | -1.0 (-3.0 to 2.0) | -1.0 (-4.0 to 0.0) |
Alkaline Phosphatase (Alk Phos); Alanine Aminotransferase (Ala Amino); Aspartate Aminotransferase (Asp Amino)
| Units per Liter | Prophylaxis | On-demand |
|---|---|---|
| Alk Phos: Week 2 (N= 115, 17) | -1.0 (-7.0 to 3.0) | -5.0 (-7.0 to 2.0) |
| Alk Phos: Week 4 (N= 114, 14) | -4.0 (-9.0 to 2.0) | -4.5 (-10.0 to 1.0) |
| Alk Phos: Month 3 (N= 109, 17) | -2.0 (-9.0 to 4.0) | -5.0 (-11.0 to 4.0) |
| Alk Phos: Completion/Termination (N= 114, 15) | 0.0 (-9.0 to 6.0) | -5.0 (-9.0 to 1.0) |
| Ala Amino: Week 2 (N= 112, 17) | 1.0 (-4.0 to 4.5) | -2.0 (-6.0 to 5.0) |
| Ala Amino: Week 4 (N= 112, 14) | 0.0 (-5.0 to 4.0) | -2.5 (-7.0 to 1.0) |
| Ala Amino: Month 3 (N= 106, 17) | 1.0 (-6.0 to 5.0) | 1.0 (-4.0 to 12.0) |
| Ala Amino: Completion/Termination (N= 112, 14) | -1.0 (-7.0 to 4.0) | 2.5 (-3.0 to 5.0) |
| Asp Amino: Week 2 (N= 110, 17) | 0.5 (-2.0 to 5.0) | -1.0 (-6.0 to 2.0) |
| Asp Amino: Week 4 (N= 110, 14) | 0.0 (-4.0 to 3.0) | -3.5 (-7.0 to 0.0) |
| Asp Amino: Month 3 (N= 103, 17) | 1.0 (-3.0 to 5.0) | 1.0 (-3.0 to 2.0) |
| Asp Amino: Completion/Termination (N= 110, 14) | 1.0 (-5.0 to 3.0) | 3.5 (-3.0 to 6.0) |
| mmol/L | Prophylaxis | On-demand |
|---|---|---|
| Bicarbonate: Week 2 (N= 111, 16) | 0.10 (-1.60 to 1.40) | 1.10 (-1.50 to 2.60) |
| Bicarbonate: Week 4 (N= 110, 13) | 0.40 (-0.90 to 2.40) | 0.10 (-1.50 to 3.00) |
| Bicarbonate: Month 3 (N= 111, 16) | 0.60 (-1.30 to 1.90) | 0.60 (-1.30 to 1.75) |
| Bicarbonate: Completion/Termination (N= 117, 13) | 1.20 (-0.60 to 2.60) | 2.20 (1.00 to 3.90) |
| Chloride: Week 2 (N= 115, 17) | 0.0 (-1.0 to 2.0) | -2.0 (-3.0 to 1.0) |
| Chloride: Week 4 (N= 114, 14) | 1.0 (-1.0 to 2.0) | -1.0 (-2.0 to 0.0) |
| Chloride: Month 3 (N= 109, 17) | 0.0 (-1.0 to 2.0) | 1.0 (-1.0 to 2.0) |
| Chloride: Completion/Termination (N= 114, 15) | 0.0 (-1.0 to 2.0) | -1.0 (-2.0 to 1.0) |
| Glucose: Week 2 (N= 112, 17) | -0.10 (-0.55 to 0.55) | -0.30 (-0.80 to 0.20) |
| Glucose: Week 4 (N= 112, 14) | 0.10 (-0.40 to 0.70) | -0.05 (-0.50 to 0.50) |
| Glucose: Month 3 (N= 106, 17) | 0.00 (-0.50 to 0.50) | 0.10 (-0.50 to 0.30) |
| Glucose: Completion/Termination (N= 115, 14) | 0.00 (-0.50 to 0.60) | -0.10 (-0.50 to 0.60) |
| Potassium: Week 2 (N= 115, 17) | 0.00 (-0.20 to 0.30) | 0.10 (-0.20 to 0.10) |
| Potassium: Week 4 (N= 114, 14) | 0.05 (-0.20 to 0.30) | 0.10 (-0.10 to 0.20) |
| Potassium: Month 3 (N= 109, 17) | 0.00 (-0.30 to 0.30) | 0.10 (-0.10 to 0.40) |
| Potassium: Completion/Termination (N= 114, 15) | 0.00 (-0.20 to 0.20) | 0.00 (-0.10 to 0.40) |
| Sodium: Week 2 (N= 115, 17) | 0.0 (-1.0 to 1.0) | -1.0 (-2.0 to 1.0) |
| Sodium: Week 4 (N= 114, 14) | 0.0 (-1.0 to 1.0) | -1.5 (-3.0 to 0.0) |
| Sodium: Month 3 (N= 109, 17) | 0.0 (-2.0 to 1.0) | 0.0 (-2.0 to 2.0) |
| Sodium: Completion/Termination (N= 114, 15) | -1.0 (-2.0 to 1.0) | -1.0 (-3.0 to 0.0) |
| BUN: Week 2 (N= 115, 17) | 0.00 (-0.60 to 0.70) | -0.20 (-0.40 to 0.70) |
| BUN: Week 4 (N= 113, 14) | 0.00 (-0.70 to 0.70) | -0.10 (-0.30 to 0.50) |
| BUN: Month 3 (N= 109, 17) | 0.30 (-0.30 to 0.90) | -0.40 (-0.70 to 0.30) |
| BUN: Completion/Termination (N= 114, 15) | 0.30 (-0.70 to 1.10) | 0.00 (-1.30 to 1.00) |
| µmol/L | Prophylaxis | On-demand |
|---|---|---|
| Creatinine: Week 2 (N= 115, 17) | 0.0 (-7.0 to 4.0) | 1.0 (-3.0 to 4.0) |
| Creatinine: Week 4 (N= 114, 14) | 0.0 (-5.0 to 4.0) | 0.5 (-6.0 to 6.0) |
| Creatinine: Month 3 (N= 109, 17) | 0.0 (-6.0 to 2.0) | 3.0 (-3.0 to 6.0) |
| Creatinine: Completion/Termination (N= 114, 15) | 0.0 (-4.0 to 5.0) | 6.0 (-4.0 to 9.0) |
| Bilirubin: Week 2 (N= 109, 17) | 0.00 (-3.00 to 2.00) | -2.00 (-5.00 to 2.00) |
| Bilirubin: Week 4 (N= 109, 14) | 0.00 (-2.00 to 2.00) | -0.50 (-5.00 to 3.00) |
| Bilirubin: Month 3 (N= 103, 17) | 0.00 (-3.00 to 2.00) | 1.00 (-3.00 to 2.00) |
| Bilirubin: Completion/Termination (N= 109, 14) | 0.00 (-3.00 to 2.00) | -0.50 (-2.00 to 3.00) |
| giga (10^9) cells per liter (Gi/L) | Prophylaxis | On-demand |
|---|---|---|
| Basophils: Week 2 (N= 108, 17) | 0.000 (-0.015 to 0.010) | 0.000 (-0.010 to 0.020) |
| Basophils: Week 4 (N= 109, 14) | 0.000 (-0.010 to 0.010) | 0.005 (-0.020 to 0.020) |
| Basophils: Month 3 (N= 103, 16) | 0.000 (-0.020 to 0.020) | 0.000 (-0.010 to 0.010) |
| Basophils: Completion/Termination (N= 107, 14) | -0.010 (-0.020 to 0.010) | 0.000 (-0.020 to 0.020) |
| Eosinophils: Week 2 (N= 108, 17) | 0.010 (-0.020 to 0.050) | 0.030 (0.000 to 0.060) |
| Eosinophils: Week 4 (N= 109, 14) | 0.020 (-0.020 to 0.060) | 0.035 (-0.010 to 0.070) |
| Eosinophils: Month 3 (N= 103, 16) | 0.010 (-0.040 to 0.050) | 0.010 (-0.005 to 0.050) |
| Eosinophils: Completion/Termination (N= 107, 14) | 0.010 (-0.030 to 0.050) | 0.045 (0.010 to 0.080) |
| Lymphocytes: Week 2 (N= 108, 17) | -0.005 (-0.215 to 0.140) | 0.280 (-0.070 to 0.640) |
| Lymphocytes: Week 4 (N= 109, 14) | -0.060 (-0.240 to 0.210) | 0.175 (0.030 to 0.350) |
| Lymphocytes: Month 3 (N= 103, 16) | 0.030 (-0.200 to 0.260) | 0.130 (-0.120 to 0.500) |
| Lymphocytes: Completion/Termination (N= 107, 14) | 0.040 (-0.160 to 0.260) | 0.030 (-0.070 to 0.460) |
| Monocytes: Week 2 (N= 108, 17) | 0.005 (-0.050 to 0.070) | 0.060 (-0.050 to 0.130) |
| Monocytes: Week 4 (N= 109, 14) | 0.020 (-0.060 to 0.050) | -0.010 (-0.070 to 0.040) |
| Monocytes: Month 3 (N= 103, 16) | 0.000 (-0.060 to 0.050) | 0.020 (-0.015 to 0.085) |
| Monocytes: Completion/Termination (N= 107, 14) | 0.010 (-0.050 to 0.080) | 0.060 (-0.040 to 0.080) |
| Neutrophils: Week 2 (N= 108, 17) | -0.165 (-0.870 to 0.540) | 0.060 (-0.490 to 0.420) |
| Neutrophils: Week 4 (N= 109, 14) | -0.130 (-0.680 to 0.450) | -0.140 (-0.300 to 0.540) |
| Neutrophils: Month 3 (N= 103, 16) | -0.070 (-0.860 to 0.490) | -0.425 (-0.725 to 0.625) |
| Neutrophils: Completion/Termination (N= 107, 14) | 0.050 (-0.820 to 0.480) | 0.055 (-0.570 to 1.150) |
| Platelets: Week 2 (N= 107, 16) | -1.0 (-21.0 to 25.0) | 13.5 (-6.5 to 34.5) |
| Platelets: Week 4 (N= 108, 13) | 2.0 (-19.5 to 22.5) | -8.0 (-18.0 to 7.0) |
| Platelets: Month 3 (N= 102, 15) | 1.5 (-21.0 to 21.0) | -3.0 (-24.0 to 17.0) |
| Platelets: Completion/Termination (N= 105, 13) | 0.0 (-20.0 to 20.0) | -1.0 (-15.0 to 14.0) |
| Leukocytes: Week 2 (N= 108, 17) | -0.185 (-0.910 to 0.560) | 0.030 (-0.550 to 1.080) |
| Leukocytes: Week 4 (N= 109, 14) | -0.180 (-0.930 to 0.640) | -0.005 (-0.670 to 0.750) |
| Leukocytes: Month 3 (N= 103, 16) | -0.040 (-1.070 to 0.620) | 0.220 (-0.760 to 1.085) |
| Leukocytes: Completion/Termination (N= 107, 14) | -0.020 (-0.900 to 0.680) | 0.250 (-0.710 to 1.800) |
| Percentage red blood cells | Prophylaxis | On-demand |
|---|---|---|
| Week 2 (N= 107, 17) | -0.010 (-0.030 to 0.010) | 0.000 (-0.020 to 0.000) |
| Week 4 (N= 108, 13) | -0.010 (-0.020 to 0.005) | -0.010 (-0.020 to 0.000) |
| Month 3 (N= 102, 16) | 0.000 (-0.020 to 0.010) | 0.000 (-0.020 to 0.010) |
| Completion/Termination (N= 106, 14) | 0.000 (-0.010 to 0.020) | -0.005 (-0.030 to 0.030) |
| g/L | Prophylaxis | On-demand |
|---|---|---|
| Week 2 (N= 108, 17) | -1.0 (-5.0 to 4.0) | 3.0 (-1.0 to 6.0) |
| Week 4 (N= 109, 14) | -1.0 (-5.0 to 5.0) | 0.0 (-2.0 to 7.0) |
| Month 3 (N= 103, 16) | 1.0 (-3.0 to 8.0) | 3.5 (-1.5 to 8.5) |
| Completion/Termination (N= 107, 14) | 2.0 (-4.0 to 6.0) | 4.5 (-6.0 to 9.0) |
| TI/L | Prophylaxis | On-demand |
|---|---|---|
| Week 2 (N= 108, 17) | -0.10 (-0.20 to 0.10) | 0.10 (-0.20 to 0.10) |
| Week 4 (N= 109, 14) | 0.00 (-0.20 to 0.20) | 0.00 (-0.20 to 0.10) |
| Month 3 (N= 103, 16) | 0.00 (-0.10 to 0.20) | 0.00 (-0.10 to 0.20) |
| Completion/Termination (N= 107, 14) | 0.00 (-0.10 to 0.20) | 0.00 (-0.20 to 0.20) |
| mmol/L | Prophylaxis | On-demand |
|---|---|---|
| Cholesterol: Week 2 (N= 115, 17) | -0.130 (-0.390 to 0.200) | -0.100 (-0.770 to 0.260) |
| Cholesterol: Week 4 (N= 114, 14) | -0.050 (-0.470 to 0.240) | -0.190 (-0.780 to 0.120) |
| Cholesterol: Month 3 (N= 109, 17) | -0.070 (-0.330 to 0.360) | 0.040 (-0.220 to 0.400) |
| Cholesterol: Completion/Termination (N= 114, 15) | -0.015 (-0.340 to 0.320) | 0.080 (-0.400 to 0.480) |
| HDL: Week 2 (N= 115, 17) | 0.000 (-0.100 to 0.080) | -0.130 (-0.190 to -0.020) |
| HDL: Week 4 (N= 114, 14) | -0.015 (-0.120 to 0.100) | -0.095 (-0.130 to 0.030) |
| HDL: Month 3 (N= 109, 17) | 0.010 (-0.110 to 0.100) | -0.020 (-0.130 to 0.050) |
| HDL: Completion/Termination (N= 114, 15) | -0.020 (-0.150 to 0.070) | 0.030 (-0.120 to 0.110) |
| LDL: Week 2 (N= 114, 17) | -0.070 (-0.330 to 0.170) | -0.090 (-0.650 to 0.000) |
| LDL: Week 4 (N= 112, 13) | -0.080 (-0.350 to 0.245) | -0.320 (-0.470 to -0.210) |
| LDL: Month 3 (N= 108, 17) | -0.030 (-0.255 to 0.380) | 0.000 (-0.410 to 0.200) |
| LDL: Completion/Termination (N= 113, 15) | 0.050 (-0.310 to 0.310) | -0.050 (-0.450 to 0.380) |
| Triglycerides: Week 2 (N= 115, 17) | -0.0600 (-0.4200 to 0.2600) | 0.1700 (-0.0100 to 0.3500) |
| Triglycerides: Week 4 (N= 114, 14) | -0.0800 (-0.4000 to 0.2300) | 0.1500 (-0.2500 to 0.6200) |
| Triglycerides: Month 3 (N= 109, 17) | -0.1600 (-0.4700 to 0.2100) | 0.3600 (0.2000 to 0.5200) |
| Triglycerides: Completion/Termination (N= 114, 15) | 0.0000 (-0.3800 to 0.2300) | 0.1200 (-0.0400 to 0.2100) |
| VLDL: Week 2 (N= 114, 17) | -0.0150 (-0.1900 to 0.1200) | 0.0800 (0.0000 to 0.1600) |
| VLDL: Week 4 (N= 112, 13) | -0.0350 (-0.1800 to 0.1050) | 0.0400 (-0.1100 to 0.2400) |
| VLDL: Month 3 (N= 108, 17) | -0.0700 (-0.2250 to 0.0950) | 0.1600 (0.0900 to 0.2400) |
| VLDL: Completion/Termination (N= 113, 15) | 0.0000 (-0.1700 to 0.1000) | 0.0600 (-0.0200 to 0.1000) |
Collected over From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| All Study Participants | — | 5/137 (3.6%) | 26/137 (19%) |
| Event | All Study Participants |
|---|---|
| HERPES ZOSTER INFECTION NEUROLOGICALInfections and infestations | 1/137 |
| HUMERUS FRACTUREInjury, poisoning and procedural complications | 1/137 |
| MUSCLE HAEMORRHAGEMusculoskeletal and connective tissue disorders | 1/137 |
| OSTEOARTHRITISMusculoskeletal and connective tissue disorders | 1/137 |
| NEUROENDOCRINE CARCINOMANeoplasms benign, malignant and unspecified (incl cysts and polyps) | 1/137 |
| Event | All Study Participants |
|---|---|
| NasopharyngitisInfections and infestations | 13/137 |
| Upper respiratory tract infectionInfections and infestations | 9/137 |
| HeadacheNervous system disorders | 7/137 |
All Study Participants
| Age, Continuous(Years) | Prophylaxis | On-demand | Total |
|---|---|---|---|
| Mean | 29.8 ± 12.53 | 31.5 ± 11.05 | 30.0 ± 12.34 |
| Sex: Female, Male(Participants) | Prophylaxis | On-demand | Total |
|---|---|---|---|
| Female | 0 | 0 | 0 |
| Male | 121 | 17 | 138 |
Plan to share: Yes — Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Supporting information: Study protocol, Statistical analysis plan, Informed consent form, Clinical study report
Access criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
Data sharing details ↗ · vivli.org
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Studies of the drug tested here: Antihemophilic factor, human recombinant