Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study)

A Phase 2/3, Multi-Center, Open Label Study of Efficacy, Safety, and Pharmacokinetics of PEGylated Recombinant Factor VIII (BAX 855) Administered for Prophylaxis and Treatment of Bleeding in Previously Treated Patients With Severe Hemophilia A

Status

Completed

Phases

Phase 2Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT01736475

Enrollment

159

Registered

2012-11-29

Start date

2013-01-31

Completion date

2014-07-17

Last updated

2021-05-20

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hemophilia A

Brief summary

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.

Interventions

BIOLOGICALAntihemophilic Factor (Recombinant) - Plasma/Albumin Free Method

Pharmacokinetic (PK) evaluation of ADVATE

BIOLOGICALPEGylated Recombinant Factor VIII

Pharmacokinetic (PK) evaluation of BAX 855

Study design

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

PREVENTION

Masking

NONE

Eligibility

Sex/Gender

MALE

Age

12 Years to 65 Years

Healthy volunteers

Inclusion criteria

Main Inclusion Criteria: * Participant and/or legal representative has/have voluntarily provided signed informed consent * Participant is 12 to 65 years old at the time of screening * Participant is male with severe hemophilia A (Factor VIII (FVIII) clotting activity \< 1%) as confirmed by central laboratory at screening after the appropriate washout period or a documented FVIII clotting activity \<1% * Participant has been previously treated with plasma-derived FVIII concentrates or recombinant FVIII for ≥150 documented exposure days (EDs) * Participant is currently receiving prophylaxis or on-demand therapy with FVIII * Participant is willing and able to comply with the requirements of the protocol Main

Exclusion criteria

* Participant has detectable FVIII inhibitory antibodies (≥ 0.6 Bethesda Units (BU) using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening * Participant has history of FVIII inhibitory antibodies (≥ 0.4 BU using the Nijmegen modification of the Bethesda assay or ≥ 0.6 BU using the Bethesda assay) at any time prior to screening * Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease).

Design outcomes

Primary

Measure	Time frame	Description
Annualized Bleeding Rate (ABR)	9 months	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.

Secondary

Measure	Time frame	Description
Average Number of BAX 855 Infusions Needed for the Treatment of Bleeding Episodes	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	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].	Interval between Bleeds in months was calculated as: Observation period for efficacy (in days)/(number of bleeds)\*(12/365.2425)
Weight-adjusted Consumption of BAX 855 - Per Prophylactic Infusion and Pharmacokinetic (PK) Infusion	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	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.	Infusions per bleeding episode for maintenance of hemostasis only includes infusions following the resolution of a bleed to maintain hemostasis.
Percentage of Participants With Adverse Events	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].	Adverse Events (AEs) and Serious Adverse Events (SAEs)
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	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 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
Patient Reported Outcomes: Haemo-SYM Questionnaire, Change in Score From Baseline to End of Study	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].	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.
Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	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]	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.
Pharmacokinetics (Pk) - Plasma Half-life (One-stage Clotting Assay)	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).	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).
Pharmacokinetics (Pk) - Mean Residence Time (One-stage Clotting Assay)	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).	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).
Pharmacokinetics (Pk) - Total Body Clearance (One-stage Clotting Assay)	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).	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).
Pharmacokinetics (Pk) - Incremental Recovery Over Time (One-stage Clotting Assay)	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).	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).
Pharmacokinetics (Pk) - Area Under the Concentration Versus Time Curve From 0 to Infinity (AUC0-∞) (One-stage Clotting Assay)	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).	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).
Pharmacokinetics (Pk) - Apparent Volume of Distribution at Steady State (Vss) (One-stage Clotting Assay)	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).	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).
Rate of Success of BAX 855 for Treatment of Bleeding Episodes	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.	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.
Pharmacokinetics (Pk) -Time to Maximum Concentration in Plasma (Tmax) (One-stage Clotting Assay)	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).	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).
Change in Vital Signs From Screening - Temperature	Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination	—
Change in Vital Signs From Screening - Pulse Rate	Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination	—
Change in Vital Signs From Screening - Respiratory Rate	Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination	—
Changes in Vital Signs From Screening - Blood Pressure	Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination	Systolic Blood Pressure (SBP) Diastolic Blood Pressure (DBP)
Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein	Screening, week 2, week 4, month 3, study completion/termination	—
Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Screening, week 2, week 4, month 3, study completion/termination	Alkaline Phosphatase (Alk Phos); Alanine Aminotransferase (Ala Amino); Aspartate Aminotransferase (Asp Amino)
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Screening, week 2, week 4, month 3, study completion/termination	—
Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin	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	Screening, week 2, week 4, month 3, study completion/termination	—
Changes in Hematology Laboratory Assessments From Screening - Hematocrit	Screening, week 2, week 4, month 3, study completion/termination	—
Changes in Hematology Laboratory Assessments From Screening - Hemoglobin	Screening, week 2, week 4, month 3, study completion/termination	—
Changes in Hematology Laboratory Assessments From Screening - Erythrocytes	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)	Screening, week 2, week 4, month 3, study completion/termination	—
Pharmacokinetics (Pk) - Maximum Plasma Concentration (Cmax) (One-stage Clotting Assay)	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).	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).

Countries

Australia, Austria, Bulgaria, Czechia, Germany, Israel, Japan, Lithuania, Malaysia, Netherlands, Poland, Romania, South Korea, Spain, Sweden, Switzerland, Taiwan, Ukraine, United Kingdom, United States

Participant flow

Recruitment details

Participants were enrolled (signed informed consent) at 72 sites.

Pre-assignment details

A total of 159 participants provided informed consent and were screened for study participation, of which there were 21 screen failures. 138 participants were assigned to the prophylactic arm or the on-demand treatment regimen.

Participants by arm

Arm	Count
Prophylaxis	121
On-demand	17
Total	138

Withdrawals & dropouts

Period	Reason	FG000
Overall Study	Adverse Event	4
Overall Study	Other - Screen failure	1
Overall Study	Other - Surgical procedure	1
Overall Study	Protocol Violation	4
Overall Study	Withdrawal by Subject	2

Baseline characteristics

Characteristic	Prophylaxis	On-demand	Total
Age, Continuous	29.8 Years STANDARD_DEVIATION 12.53	31.5 Years STANDARD_DEVIATION 11.05	30.0 Years STANDARD_DEVIATION 12.34
Sex: Female, Male Female	0 Participants	0 Participants	0 Participants
Sex: Female, Male Male	121 Participants	17 Participants	138 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	— / —
other Total, other adverse events	26 / 137
serious Total, serious adverse events	5 / 137

Outcome results

Primary

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

Population: Full Analysis Set

Arm	Measure	Value (LEAST_SQUARES_MEAN)
Prophylaxis	Annualized Bleeding Rate (ABR)	4.3 Bleeds per year
On-demand	Annualized Bleeding Rate (ABR)	43.4 Bleeds per year

Comparison: Ratio Annualized Bleeding Rate (ABR) Prophylaxis/On-demand: Prophylaxis treatment w ill be considered to be successful if the upper limit of the 95% CI for the ratio between treatment regimen does not exceed 0.5 (corresponding to a 50% reduction of the mean ABR compared to the on-demand treatment). H01: μ1 ≥0.5\*μ2 Ha1: μ1\<0.5\*μ2 w here μ1 and μ2 are the mean ABRs in on prophylaxis and on-demand, respectivelyp-value: <0.000195% CI: [0.06, 0.19]Regression, Negative binomial

Secondary

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].

Population: Participants from the Full Analysis Set who experienced at least one bleeding episode.

Arm	Measure	Value (MEAN)	Dispersion
Prophylaxis	Average Number of BAX 855 Infusions Needed for the Treatment of Bleeding Episodes	1.37 Infusions	Standard Deviation 0.8
On-demand	Average Number of BAX 855 Infusions Needed for the Treatment of Bleeding Episodes	1.21 Infusions	Standard Deviation 0.35

Secondary

Change in Vital Signs From Screening - Pulse Rate

Time frame: Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination

Population: Safety Analysis Set

Arm	Measure	Group	Value (MEDIAN)
Prophylaxis	Change in Vital Signs From Screening - Pulse Rate	Week 4 (N= 117, 15)	2.0 beats per minute
Prophylaxis	Change in Vital Signs From Screening - Pulse Rate	Month 3 (N= 112, 17)	2.0 beats per minute
Prophylaxis	Change in Vital Signs From Screening - Pulse Rate	Exposure day 10-15 (N= 31, 10)	3.0 beats per minute
Prophylaxis	Change in Vital Signs From Screening - Pulse Rate	Completion/Termination (N= 116, 15)	2.0 beats per minute
Prophylaxis	Change in Vital Signs From Screening - Pulse Rate	Week 2 (N= 118, 17)	3.0 beats per minute
On-demand	Change in Vital Signs From Screening - Pulse Rate	Completion/Termination (N= 116, 15)	3.0 beats per minute
On-demand	Change in Vital Signs From Screening - Pulse Rate	Week 2 (N= 118, 17)	2.0 beats per minute
On-demand	Change in Vital Signs From Screening - Pulse Rate	Week 4 (N= 117, 15)	2.0 beats per minute
On-demand	Change in Vital Signs From Screening - Pulse Rate	Exposure day 10-15 (N= 31, 10)	4.0 beats per minute
On-demand	Change in Vital Signs From Screening - Pulse Rate	Month 3 (N= 112, 17)	1.0 beats per minute

Secondary

Change in Vital Signs From Screening - Respiratory Rate

Time frame: Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination

Population: Safety Analysis Set

Arm	Measure	Group	Value (MEDIAN)
Prophylaxis	Change in Vital Signs From Screening - Respiratory Rate	Week 4 (N= 118, 15)	0.0 breaths per minute
Prophylaxis	Change in Vital Signs From Screening - Respiratory Rate	Month 3 (N= 112, 16)	0.0 breaths per minute
Prophylaxis	Change in Vital Signs From Screening - Respiratory Rate	Exposure day 10-15 (N= 31, 10)	0.0 breaths per minute
Prophylaxis	Change in Vital Signs From Screening - Respiratory Rate	Completion/Termination (N= 115, 15)	0.0 breaths per minute
Prophylaxis	Change in Vital Signs From Screening - Respiratory Rate	Week 2 (N= 117, 17)	0.0 breaths per minute
On-demand	Change in Vital Signs From Screening - Respiratory Rate	Completion/Termination (N= 115, 15)	-1.0 breaths per minute
On-demand	Change in Vital Signs From Screening - Respiratory Rate	Week 2 (N= 117, 17)	0.0 breaths per minute
On-demand	Change in Vital Signs From Screening - Respiratory Rate	Week 4 (N= 118, 15)	0.0 breaths per minute
On-demand	Change in Vital Signs From Screening - Respiratory Rate	Exposure day 10-15 (N= 31, 10)	0.0 breaths per minute
On-demand	Change in Vital Signs From Screening - Respiratory Rate	Month 3 (N= 112, 16)	0.0 breaths per minute

Secondary

Change in Vital Signs From Screening - Temperature

Time frame: Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination

Population: Safety Analysis Set

Arm	Measure	Group	Value (MEDIAN)
Prophylaxis	Change in Vital Signs From Screening - Temperature	Week 4 (N= 118, 15)	0.00 Celsius
Prophylaxis	Change in Vital Signs From Screening - Temperature	Month 3 (N= 112, 17)	0.00 Celsius
Prophylaxis	Change in Vital Signs From Screening - Temperature	Exposure day 10-15 (N= 31, 10)	-0.10 Celsius
Prophylaxis	Change in Vital Signs From Screening - Temperature	Completion/Termination (N= 116, 15)	0.00 Celsius
Prophylaxis	Change in Vital Signs From Screening - Temperature	Week 2 (N= 118, 17)	0.00 Celsius
On-demand	Change in Vital Signs From Screening - Temperature	Completion/Termination (N= 116, 15)	0.00 Celsius
On-demand	Change in Vital Signs From Screening - Temperature	Week 2 (N= 118, 17)	0.00 Celsius
On-demand	Change in Vital Signs From Screening - Temperature	Week 4 (N= 118, 15)	0.00 Celsius
On-demand	Change in Vital Signs From Screening - Temperature	Exposure day 10-15 (N= 31, 10)	0.00 Celsius
On-demand	Change in Vital Signs From Screening - Temperature	Month 3 (N= 112, 17)	0.00 Celsius

Secondary

Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein

Time frame: Screening, week 2, week 4, month 3, study completion/termination

Population: Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.

Arm	Measure	Group	Value (MEDIAN)
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein	Albumin: Completion/Termination (N= 112, 14)	0.0 g/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein	Albumin: Week 2 (N= 112, 17)	-1.0 g/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein	Albumin: Week 4 (N= 112, 14)	-1.0 g/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein	Albumin: Month 3 (N= 106, 17)	-1.0 g/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein	Protein: Week 2 (N= 115, 17)	-1.0 g/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein	Protein: Week 4 (N= 114, 14)	-1.0 g/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein	Protein: Month 3 (N= 109, 17)	-1.0 g/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein	Protein: Completion/Termination (N= 114, 15)	-1.0 g/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein	Protein: Completion/Termination (N= 114, 15)	-1.0 g/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein	Protein: Week 2 (N= 115, 17)	-2.0 g/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein	Albumin: Week 2 (N= 112, 17)	-1.0 g/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein	Protein: Month 3 (N= 109, 17)	-1.0 g/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein	Albumin: Week 4 (N= 112, 14)	-1.0 g/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein	Protein: Week 4 (N= 114, 14)	-2.0 g/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein	Albumin: Month 3 (N= 106, 17)	-1.0 g/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein	Albumin: Completion/Termination (N= 112, 14)	-1.0 g/L

Secondary

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

Population: Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.

Arm	Measure	Group	Value (MEDIAN)
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Ala Amino: Week 2 (N= 112, 17)	1.0 Units per Liter
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Ala Amino: Week 4 (N= 112, 14)	0.0 Units per Liter
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Ala Amino: Month 3 (N= 106, 17)	1.0 Units per Liter
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Ala Amino: Completion/Termination (N= 112, 14)	-1.0 Units per Liter
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Asp Amino: Week 2 (N= 110, 17)	0.5 Units per Liter
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Asp Amino: Week 4 (N= 110, 14)	0.0 Units per Liter
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Asp Amino: Month 3 (N= 103, 17)	1.0 Units per Liter
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Asp Amino: Completion/Termination (N= 110, 14)	1.0 Units per Liter
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Alk Phos: Week 2 (N= 115, 17)	-1.0 Units per Liter
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Alk Phos: Week 4 (N= 114, 14)	-4.0 Units per Liter
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Alk Phos: Month 3 (N= 109, 17)	-2.0 Units per Liter
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Alk Phos: Completion/Termination (N= 114, 15)	0.0 Units per Liter
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Alk Phos: Month 3 (N= 109, 17)	-5.0 Units per Liter
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Ala Amino: Week 2 (N= 112, 17)	-2.0 Units per Liter
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Asp Amino: Month 3 (N= 103, 17)	1.0 Units per Liter
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Ala Amino: Week 4 (N= 112, 14)	-2.5 Units per Liter
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Alk Phos: Week 4 (N= 114, 14)	-4.5 Units per Liter
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Ala Amino: Month 3 (N= 106, 17)	1.0 Units per Liter
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Asp Amino: Completion/Termination (N= 110, 14)	3.5 Units per Liter
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Ala Amino: Completion/Termination (N= 112, 14)	2.5 Units per Liter
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Alk Phos: Completion/Termination (N= 114, 15)	-5.0 Units per Liter
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Asp Amino: Week 2 (N= 110, 17)	-1.0 Units per Liter
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Alk Phos: Week 2 (N= 115, 17)	-5.0 Units per Liter
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Asp Amino: Week 4 (N= 110, 14)	-3.5 Units per Liter

Secondary

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

Population: Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.

Arm	Measure	Group	Value (MEDIAN)
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Potassium: Completion/Termination (N= 114, 15)	0.00 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Sodium: Week 2 (N= 115, 17)	0.0 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	BUN: Week 4 (N= 113, 14)	0.00 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	BUN: Month 3 (N= 109, 17)	0.30 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Bicarbonate: Week 2 (N= 111, 16)	0.10 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Bicarbonate: Week 4 (N= 110, 13)	0.40 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Bicarbonate: Month 3 (N= 111, 16)	0.60 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Bicarbonate: Completion/Termination (N= 117, 13)	1.20 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Chloride: Week 2 (N= 115, 17)	0.0 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Chloride: Week 4 (N= 114, 14)	1.0 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Chloride: Month 3 (N= 109, 17)	0.0 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Chloride: Completion/Termination (N= 114, 15)	0.0 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Glucose: Week 2 (N= 112, 17)	-0.10 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Glucose: Week 4 (N= 112, 14)	0.10 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Glucose: Month 3 (N= 106, 17)	0.00 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Glucose: Completion/Termination (N= 115, 14)	0.00 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Potassium: Week 2 (N= 115, 17)	0.00 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Potassium: Week 4 (N= 114, 14)	0.05 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Potassium: Month 3 (N= 109, 17)	0.00 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Sodium: Week 4 (N= 114, 14)	0.0 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Sodium: Month 3 (N= 109, 17)	0.0 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Sodium: Completion/Termination (N= 114, 15)	-1.0 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	BUN: Week 2 (N= 115, 17)	0.00 mmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	BUN: Completion/Termination (N= 114, 15)	0.30 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Glucose: Week 2 (N= 112, 17)	-0.30 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Potassium: Completion/Termination (N= 114, 15)	0.00 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	BUN: Completion/Termination (N= 114, 15)	0.00 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Sodium: Week 2 (N= 115, 17)	-1.0 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Glucose: Week 4 (N= 112, 14)	-0.05 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	BUN: Week 4 (N= 113, 14)	-0.10 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Sodium: Week 4 (N= 114, 14)	-1.5 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Glucose: Month 3 (N= 106, 17)	0.10 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Bicarbonate: Week 2 (N= 111, 16)	1.10 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	BUN: Week 2 (N= 115, 17)	-0.20 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Bicarbonate: Week 4 (N= 110, 13)	0.10 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Glucose: Completion/Termination (N= 115, 14)	-0.10 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Bicarbonate: Month 3 (N= 111, 16)	0.60 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Sodium: Month 3 (N= 109, 17)	0.0 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Bicarbonate: Completion/Termination (N= 117, 13)	2.20 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Potassium: Week 2 (N= 115, 17)	0.10 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Chloride: Week 2 (N= 115, 17)	-2.0 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	BUN: Month 3 (N= 109, 17)	-0.40 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Chloride: Week 4 (N= 114, 14)	-1.0 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Potassium: Week 4 (N= 114, 14)	0.10 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Chloride: Month 3 (N= 109, 17)	1.0 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Sodium: Completion/Termination (N= 114, 15)	-1.0 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Chloride: Completion/Termination (N= 114, 15)	-1.0 mmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)	Potassium: Month 3 (N= 109, 17)	0.10 mmol/L

Secondary

Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin

Time frame: Screening, week 2, week 4, month 3, study completion/termination

Population: Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.

Arm	Measure	Group	Value (MEDIAN)
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin	Creatinine: Week 2 (N= 115, 17)	0.0 µmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin	Creatinine: Week 4 (N= 114, 14)	0.0 µmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin	Creatinine: Month 3 (N= 109, 17)	0.0 µmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin	Creatinine: Completion/Termination (N= 114, 15)	0.0 µmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin	Bilirubin: Week 2 (N= 109, 17)	0.00 µmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin	Bilirubin: Week 4 (N= 109, 14)	0.00 µmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin	Bilirubin: Month 3 (N= 103, 17)	0.00 µmol/L
Prophylaxis	Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin	Bilirubin: Completion/Termination (N= 109, 14)	0.00 µmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin	Bilirubin: Completion/Termination (N= 109, 14)	-0.50 µmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin	Creatinine: Week 2 (N= 115, 17)	1.0 µmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin	Bilirubin: Week 2 (N= 109, 17)	-2.00 µmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin	Creatinine: Week 4 (N= 114, 14)	0.5 µmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin	Bilirubin: Month 3 (N= 103, 17)	1.00 µmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin	Creatinine: Month 3 (N= 109, 17)	3.0 µmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin	Bilirubin: Week 4 (N= 109, 14)	-0.50 µmol/L
On-demand	Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin	Creatinine: Completion/Termination (N= 114, 15)	6.0 µmol/L

Secondary

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

Population: Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.

Arm	Measure	Group	Value (MEDIAN)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Monocytes: Month 3 (N= 103, 16)	0.000 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Eosinophils: Week 2 (N= 108, 17)	0.010 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Monocytes: Completion/Termination (N= 107, 14)	0.010 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Lymphocytes: Week 2 (N= 108, 17)	-0.005 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Neutrophils: Week 2 (N= 108, 17)	-0.165 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Basophils: Week 4 (N= 109, 14)	0.000 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Neutrophils: Week 4 (N= 109, 14)	-0.130 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Lymphocytes: Week 4 (N= 109, 14)	-0.060 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Neutrophils: Month 3 (N= 103, 16)	-0.070 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Eosinophils: Week 4 (N= 109, 14)	0.020 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Neutrophils: Completion/Termination (N= 107, 14)	0.050 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Lymphocytes: Month 3 (N= 103, 16)	0.030 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Platelets: Week 2 (N= 107, 16)	-1.0 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Basophils: Completion/Termination (N= 107, 14)	-0.010 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Platelets: Week 4 (N= 108, 13)	2.0 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Lymphocytes: Completion/Termination (N= 107, 14)	0.040 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Platelets: Month 3 (N= 102, 15)	1.5 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Eosinophils: Month 3 (N= 103, 16)	0.010 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Platelets: Completion/Termination (N= 105, 13)	0.0 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Monocytes: Week 2 (N= 108, 17)	0.005 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Leukocytes: Week 2 (N= 108, 17)	-0.185 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Basophils: Month 3 (N= 103, 16)	0.000 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Leukocytes: Week 4 (N= 109, 14)	-0.180 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Monocytes: Week 4 (N= 109, 14)	0.020 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Leukocytes: Month 3 (N= 103, 16)	-0.040 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Eosinophils: Completion/Termination (N= 107, 14)	0.010 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Leukocytes: Completion/Termination (N= 107, 14)	-0.020 giga (10^9) cells per liter (Gi/L)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Basophils: Week 2 (N= 108, 17)	0.000 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Leukocytes: Completion/Termination (N= 107, 14)	0.250 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Basophils: Week 2 (N= 108, 17)	0.000 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Basophils: Week 4 (N= 109, 14)	0.005 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Basophils: Month 3 (N= 103, 16)	0.000 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Basophils: Completion/Termination (N= 107, 14)	0.000 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Eosinophils: Week 2 (N= 108, 17)	0.030 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Eosinophils: Week 4 (N= 109, 14)	0.035 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Eosinophils: Month 3 (N= 103, 16)	0.010 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Eosinophils: Completion/Termination (N= 107, 14)	0.045 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Lymphocytes: Week 2 (N= 108, 17)	0.280 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Lymphocytes: Week 4 (N= 109, 14)	0.175 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Lymphocytes: Month 3 (N= 103, 16)	0.130 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Lymphocytes: Completion/Termination (N= 107, 14)	0.030 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Monocytes: Week 2 (N= 108, 17)	0.060 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Monocytes: Week 4 (N= 109, 14)	-0.010 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Monocytes: Month 3 (N= 103, 16)	0.020 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Monocytes: Completion/Termination (N= 107, 14)	0.060 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Neutrophils: Week 2 (N= 108, 17)	0.060 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Neutrophils: Week 4 (N= 109, 14)	-0.140 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Neutrophils: Month 3 (N= 103, 16)	-0.425 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Neutrophils: Completion/Termination (N= 107, 14)	0.055 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Platelets: Week 2 (N= 107, 16)	13.5 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Platelets: Week 4 (N= 108, 13)	-8.0 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Platelets: Month 3 (N= 102, 15)	-3.0 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Platelets: Completion/Termination (N= 105, 13)	-1.0 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Leukocytes: Week 2 (N= 108, 17)	0.030 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Leukocytes: Week 4 (N= 109, 14)	-0.005 giga (10^9) cells per liter (Gi/L)
On-demand	Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes	Leukocytes: Month 3 (N= 103, 16)	0.220 giga (10^9) cells per liter (Gi/L)

Secondary

Changes in Hematology Laboratory Assessments From Screening - Erythrocytes

Time frame: Screening, week 2, week 4, month 3, study completion/termination

Population: Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.

Arm	Measure	Group	Value (MEDIAN)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Erythrocytes	Week 2 (N= 108, 17)	-0.10 TI/L
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Erythrocytes	Week 4 (N= 109, 14)	0.00 TI/L
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Erythrocytes	Month 3 (N= 103, 16)	0.00 TI/L
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Erythrocytes	Completion/Termination (N= 107, 14)	0.00 TI/L
On-demand	Changes in Hematology Laboratory Assessments From Screening - Erythrocytes	Completion/Termination (N= 107, 14)	0.00 TI/L
On-demand	Changes in Hematology Laboratory Assessments From Screening - Erythrocytes	Week 2 (N= 108, 17)	0.10 TI/L
On-demand	Changes in Hematology Laboratory Assessments From Screening - Erythrocytes	Month 3 (N= 103, 16)	0.00 TI/L
On-demand	Changes in Hematology Laboratory Assessments From Screening - Erythrocytes	Week 4 (N= 109, 14)	0.00 TI/L

Secondary

Changes in Hematology Laboratory Assessments From Screening - Hematocrit

Time frame: Screening, week 2, week 4, month 3, study completion/termination

Population: Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.

Arm	Measure	Group	Value (MEDIAN)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Hematocrit	Week 2 (N= 107, 17)	-0.010 Percentage red blood cells
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Hematocrit	Week 4 (N= 108, 13)	-0.010 Percentage red blood cells
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Hematocrit	Month 3 (N= 102, 16)	0.000 Percentage red blood cells
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Hematocrit	Completion/Termination (N= 106, 14)	0.000 Percentage red blood cells
On-demand	Changes in Hematology Laboratory Assessments From Screening - Hematocrit	Completion/Termination (N= 106, 14)	-0.005 Percentage red blood cells
On-demand	Changes in Hematology Laboratory Assessments From Screening - Hematocrit	Week 2 (N= 107, 17)	0.000 Percentage red blood cells
On-demand	Changes in Hematology Laboratory Assessments From Screening - Hematocrit	Month 3 (N= 102, 16)	0.000 Percentage red blood cells
On-demand	Changes in Hematology Laboratory Assessments From Screening - Hematocrit	Week 4 (N= 108, 13)	-0.010 Percentage red blood cells

Secondary

Changes in Hematology Laboratory Assessments From Screening - Hemoglobin

Time frame: Screening, week 2, week 4, month 3, study completion/termination

Population: Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.

Arm	Measure	Group	Value (MEDIAN)
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Hemoglobin	Week 2 (N= 108, 17)	-1.0 g/L
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Hemoglobin	Week 4 (N= 109, 14)	-1.0 g/L
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Hemoglobin	Month 3 (N= 103, 16)	1.0 g/L
Prophylaxis	Changes in Hematology Laboratory Assessments From Screening - Hemoglobin	Completion/Termination (N= 107, 14)	2.0 g/L
On-demand	Changes in Hematology Laboratory Assessments From Screening - Hemoglobin	Completion/Termination (N= 107, 14)	4.5 g/L
On-demand	Changes in Hematology Laboratory Assessments From Screening - Hemoglobin	Week 2 (N= 108, 17)	3.0 g/L
On-demand	Changes in Hematology Laboratory Assessments From Screening - Hemoglobin	Month 3 (N= 103, 16)	3.5 g/L
On-demand	Changes in Hematology Laboratory Assessments From Screening - Hemoglobin	Week 4 (N= 109, 14)	0.0 g/L

Secondary

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

Population: Safety Analysis Set - Subset of participants with both screening visit data and follow on time point data.

Arm	Measure	Group	Value (MEDIAN)
Prophylaxis	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	Cholesterol: Week 2 (N= 115, 17)	-0.130 mmol/L
Prophylaxis	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	Cholesterol: Week 4 (N= 114, 14)	-0.050 mmol/L
Prophylaxis	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	Cholesterol: Month 3 (N= 109, 17)	-0.070 mmol/L
Prophylaxis	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	Cholesterol: Completion/Termination (N= 114, 15)	-0.015 mmol/L
Prophylaxis	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	HDL: Week 2 (N= 115, 17)	0.000 mmol/L
Prophylaxis	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	HDL: Week 4 (N= 114, 14)	-0.015 mmol/L
Prophylaxis	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	HDL: Month 3 (N= 109, 17)	0.010 mmol/L
Prophylaxis	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	HDL: Completion/Termination (N= 114, 15)	-0.020 mmol/L
Prophylaxis	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	LDL: Week 2 (N= 114, 17)	-0.070 mmol/L
Prophylaxis	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	LDL: Week 4 (N= 112, 13)	-0.080 mmol/L
Prophylaxis	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	LDL: Month 3 (N= 108, 17)	-0.030 mmol/L
Prophylaxis	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	LDL: Completion/Termination (N= 113, 15)	0.050 mmol/L
Prophylaxis	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	Triglycerides: Week 2 (N= 115, 17)	-0.0600 mmol/L
Prophylaxis	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	Triglycerides: Week 4 (N= 114, 14)	-0.0800 mmol/L
Prophylaxis	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	Triglycerides: Month 3 (N= 109, 17)	-0.1600 mmol/L
Prophylaxis	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	Triglycerides: Completion/Termination (N= 114, 15)	0.0000 mmol/L
Prophylaxis	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	VLDL: Week 2 (N= 114, 17)	-0.0150 mmol/L
Prophylaxis	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	VLDL: Week 4 (N= 112, 13)	-0.0350 mmol/L
Prophylaxis	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	VLDL: Month 3 (N= 108, 17)	-0.0700 mmol/L
Prophylaxis	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	VLDL: Completion/Termination (N= 113, 15)	0.0000 mmol/L
On-demand	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	VLDL: Week 4 (N= 112, 13)	0.0400 mmol/L
On-demand	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	Cholesterol: Week 2 (N= 115, 17)	-0.100 mmol/L
On-demand	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	LDL: Month 3 (N= 108, 17)	0.000 mmol/L
On-demand	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	Cholesterol: Week 4 (N= 114, 14)	-0.190 mmol/L
On-demand	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	Triglycerides: Completion/Termination (N= 114, 15)	0.1200 mmol/L
On-demand	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	Cholesterol: Month 3 (N= 109, 17)	0.040 mmol/L
On-demand	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	LDL: Completion/Termination (N= 113, 15)	-0.050 mmol/L
On-demand	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	Cholesterol: Completion/Termination (N= 114, 15)	0.080 mmol/L
On-demand	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	VLDL: Completion/Termination (N= 113, 15)	0.0600 mmol/L
On-demand	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	HDL: Week 2 (N= 115, 17)	-0.130 mmol/L
On-demand	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	Triglycerides: Week 2 (N= 115, 17)	0.1700 mmol/L
On-demand	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	HDL: Week 4 (N= 114, 14)	-0.095 mmol/L
On-demand	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	VLDL: Week 2 (N= 114, 17)	0.0800 mmol/L
On-demand	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	HDL: Month 3 (N= 109, 17)	-0.020 mmol/L
On-demand	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	Triglycerides: Week 4 (N= 114, 14)	0.1500 mmol/L
On-demand	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	HDL: Completion/Termination (N= 114, 15)	0.030 mmol/L
On-demand	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	VLDL: Month 3 (N= 108, 17)	0.1600 mmol/L
On-demand	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	LDL: Week 2 (N= 114, 17)	-0.090 mmol/L
On-demand	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	Triglycerides: Month 3 (N= 109, 17)	0.3600 mmol/L
On-demand	Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)	LDL: Week 4 (N= 112, 13)	-0.320 mmol/L

Secondary

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

Population: Safety Analysis Set

Arm	Measure	Group	Value (MEDIAN)
Prophylaxis	Changes in Vital Signs From Screening - Blood Pressure	SBP-Week 2 (N= 118, 17)	0.0 mmHg
Prophylaxis	Changes in Vital Signs From Screening - Blood Pressure	SBP-Week 4 (N= 118, 15)	-0.5 mmHg
Prophylaxis	Changes in Vital Signs From Screening - Blood Pressure	SBP-Exposure day 10-15 (N= 31, 10)	0.0 mmHg
Prophylaxis	Changes in Vital Signs From Screening - Blood Pressure	SBP-Month 3 (N= 112, 17)	0.0 mmHg
Prophylaxis	Changes in Vital Signs From Screening - Blood Pressure	SBP-Completion/Termination (N= 116, 15)	0.0 mmHg
Prophylaxis	Changes in Vital Signs From Screening - Blood Pressure	DBP-Week 2 (N= 118, 17)	0.0 mmHg
Prophylaxis	Changes in Vital Signs From Screening - Blood Pressure	DBP-Week 4 (N= 118, 15)	-0.5 mmHg
Prophylaxis	Changes in Vital Signs From Screening - Blood Pressure	DBP-Exposure day 10-15 (N= 31, 10)	0.0 mmHg
Prophylaxis	Changes in Vital Signs From Screening - Blood Pressure	DBP-Month 3 (N= 112, 17)	0.0 mmHg
Prophylaxis	Changes in Vital Signs From Screening - Blood Pressure	DBP-Completion/Termination (N= 116, 15)	0.0 mmHg
On-demand	Changes in Vital Signs From Screening - Blood Pressure	DBP-Exposure day 10-15 (N= 31, 10)	-2.5 mmHg
On-demand	Changes in Vital Signs From Screening - Blood Pressure	SBP-Week 2 (N= 118, 17)	-2.0 mmHg
On-demand	Changes in Vital Signs From Screening - Blood Pressure	DBP-Week 2 (N= 118, 17)	-2.0 mmHg
On-demand	Changes in Vital Signs From Screening - Blood Pressure	SBP-Week 4 (N= 118, 15)	-1.0 mmHg
On-demand	Changes in Vital Signs From Screening - Blood Pressure	DBP-Completion/Termination (N= 116, 15)	-2.0 mmHg
On-demand	Changes in Vital Signs From Screening - Blood Pressure	SBP-Exposure day 10-15 (N= 31, 10)	3.5 mmHg
On-demand	Changes in Vital Signs From Screening - Blood Pressure	DBP-Week 4 (N= 118, 15)	-6.0 mmHg
On-demand	Changes in Vital Signs From Screening - Blood Pressure	SBP-Month 3 (N= 112, 17)	-5.0 mmHg
On-demand	Changes in Vital Signs From Screening - Blood Pressure	DBP-Month 3 (N= 112, 17)	-4.0 mmHg
On-demand	Changes in Vital Signs From Screening - Blood Pressure	SBP-Completion/Termination (N= 116, 15)	0.0 mmHg

Secondary

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

Population: Safety Analysis Set (SAS) - who received BAX855 during the study period. Note: one participant was assigned to the prophylactic arm but did not receive BAX855 (only received ADVATE, during the screening period).

Arm	Measure	Group	Value (NUMBER)
Prophylaxis	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	Inhibitory Antibodies to FVIII (N= 112, 14)	0 Participants
Prophylaxis	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	IgG: Binding Antibodies FVIII (N= 117, 15)	0 Participants
Prophylaxis	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	IgM: Binding Antibodies FVIII (N= 117, 15)	0 Participants
Prophylaxis	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	IgG: Binding Antibodies PEG (N= 117, 15)	0 Participants
Prophylaxis	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	IgM: Binding Antibodies PEG (N= 117, 15)	0 Participants
Prophylaxis	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	IgG: Binding Antibodies PEG-FVIII (N= 117, 15)	0 Participants
Prophylaxis	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	IgM: Binding Antibodies PEG-FVIII (N= 117, 15)	0 Participants
Prophylaxis	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	CHO-Protein Antibodies (N= 117, 15)	0 Participants
On-demand	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	CHO-Protein Antibodies (N= 117, 15)	0 Participants
On-demand	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	Inhibitory Antibodies to FVIII (N= 112, 14)	0 Participants
On-demand	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	IgM: Binding Antibodies PEG (N= 117, 15)	0 Participants
On-demand	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	IgG: Binding Antibodies FVIII (N= 117, 15)	0 Participants
On-demand	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	IgM: Binding Antibodies PEG-FVIII (N= 117, 15)	0 Participants
On-demand	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	IgM: Binding Antibodies FVIII (N= 117, 15)	0 Participants
On-demand	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	IgG: Binding Antibodies PEG-FVIII (N= 117, 15)	1 Participants
On-demand	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	IgG: Binding Antibodies PEG (N= 117, 15)	0 Participants

Secondary

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)

Population: Study participants from the Full Analysis Set (FAS) who received BAX855 during the study period. Note: one participant was assigned to the prophylactic arm (thus was included in the FAS) and received only ADVATE during the screening period.

Arm	Measure	Group	Value (NUMBER)
Prophylaxis	Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes	No bleed	45 Participants
Prophylaxis	Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes	>6 Months	5 Participants
Prophylaxis	Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes	6 Months	20 Participants
Prophylaxis	Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes	5 Months	3 Participants
Prophylaxis	Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes	4 Months	0 Participants
Prophylaxis	Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes	3 Months	11 Participants
Prophylaxis	Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes	2 Months	16 Participants
Prophylaxis	Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes	≤1 Month	20 Participants
On-demand	Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes	≤1 Month	17 Participants
On-demand	Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes	No bleed	0 Participants
On-demand	Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes	4 Months	0 Participants
On-demand	Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes	>6 Months	0 Participants
On-demand	Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes	2 Months	0 Participants
On-demand	Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes	6 Months	0 Participants
On-demand	Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes	3 Months	0 Participants
On-demand	Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes	5 Months	0 Participants

Secondary

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].

Population: Full Analysis Set - Subset of participants with both baseline and study completion HAEMO-SYM scores

Arm	Measure	Group	Value (MEAN)	Dispersion
Prophylaxis	Patient Reported Outcomes: Haemo-SYM Questionnaire, Change in Score From Baseline to End of Study	Bleed Severity Total Score	-4.17 Score on a scale	Standard Deviation 17.05
Prophylaxis	Patient Reported Outcomes: Haemo-SYM Questionnaire, Change in Score From Baseline to End of Study	Pain Severity Total Score	-1.22 Score on a scale	Standard Deviation 12.5
On-demand	Patient Reported Outcomes: Haemo-SYM Questionnaire, Change in Score From Baseline to End of Study	Bleed Severity Total Score	-4.24 Score on a scale	Standard Deviation 15.71
On-demand	Patient Reported Outcomes: Haemo-SYM Questionnaire, Change in Score From Baseline to End of Study	Pain Severity Total Score	-0.17 Score on a scale	Standard Deviation 11.88

Secondary

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]

Population: Full Analysis Set - Subset of participants with both baseline and study completion SF-36 scores

Arm	Measure	Group	Value (MEAN)	Dispersion
Prophylaxis	Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	Physical Functioning (N= 97, 12)	0.49 Score on a scale	Standard Deviation 5.27
Prophylaxis	Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	Role-physical (N= 97, 12)	1.31 Score on a scale	Standard Deviation 7.36
Prophylaxis	Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	Bodily Pain (N= 97, 12)	2.08 Score on a scale	Standard Deviation 8.19
Prophylaxis	Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	General Health (N= 96, 12)	0.40 Score on a scale	Standard Deviation 6.43
Prophylaxis	Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	Vitality (N= 96, 12)	-0.38 Score on a scale	Standard Deviation 7.43
Prophylaxis	Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	Social Functioning (N= 97, 12)	0.90 Score on a scale	Standard Deviation 7.54
Prophylaxis	Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	Role Emotional (N= 97, 12)	-0.20 Score on a scale	Standard Deviation 8.46
Prophylaxis	Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	Mental Health (N= 96, 12)	0.09 Score on a scale	Standard Deviation 7.26
Prophylaxis	Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	Physical Component Score (N= 96, 12)	1.36 Score on a scale	Standard Deviation 5.76
Prophylaxis	Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	Mental Component Score (N= 96, 12)	-0.37 Score on a scale	Standard Deviation 7.38
On-demand	Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	Mental Health (N= 96, 12)	-3.29 Score on a scale	Standard Deviation 7.95
On-demand	Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	Physical Functioning (N= 97, 12)	-2.46 Score on a scale	Standard Deviation 4.29
On-demand	Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	Social Functioning (N= 97, 12)	-3.18 Score on a scale	Standard Deviation 6.35
On-demand	Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	Role-physical (N= 97, 12)	-3.67 Score on a scale	Standard Deviation 9.01
On-demand	Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	Mental Component Score (N= 96, 12)	-1.14 Score on a scale	Standard Deviation 5.03
On-demand	Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	Bodily Pain (N= 97, 12)	0.60 Score on a scale	Standard Deviation 4.44
On-demand	Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	Role Emotional (N= 97, 12)	0.65 Score on a scale	Standard Deviation 7.92
On-demand	Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	General Health (N= 96, 12)	-0.28 Score on a scale	Standard Deviation 9.04
On-demand	Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	Physical Component Score (N= 96, 12)	-1.58 Score on a scale	Standard Deviation 4.97
On-demand	Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	Vitality (N= 96, 12)	0.26 Score on a scale	Standard Deviation 9.36

Secondary

Percentage of Participants With Adverse Events

Adverse Events (AEs) and Serious Adverse Events (SAEs)

Population: Safety Analysis Set (SAS) - All participants treated with BAX 855 were analyzed as a single group (ie on-demand and prophylaxis treatment regimens were analyzed as a single group).

Arm	Measure	Group	Value (NUMBER)
Prophylaxis	Percentage of Participants With Adverse Events	SAE, Moderate, Unrelated	0.7 percent of participants
Prophylaxis	Percentage of Participants With Adverse Events	SAE, Severe, Unrelated	2.9 percent of participants
Prophylaxis	Percentage of Participants With Adverse Events	nSAE, Mild, Unrelated	40.1 percent of participants
Prophylaxis	Percentage of Participants With Adverse Events	nSAE, Mild, Related	3.6 percent of participants
Prophylaxis	Percentage of Participants With Adverse Events	nSAE, Moderate, Unrelated	19.0 percent of participants
Prophylaxis	Percentage of Participants With Adverse Events	nSAE, Moderate, Related	1.5 percent of participants
Prophylaxis	Percentage of Participants With Adverse Events	nSAE, Unknown Severity, Unrelated	0.7 percent of participants
Prophylaxis	Percentage of Participants With Adverse Events	nSAE, Severe, Unrelated	1.5 percent of participants

Secondary

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).

Population: Pharmacokinetic full analysis set (PKFAS)

Arm	Measure	Group	Value (MEAN)	Dispersion
Prophylaxis	Pharmacokinetics (Pk) - Apparent Volume of Distribution at Steady State (Vss) (One-stage Clotting Assay)	PK-1: ADVATE	0.5487 dL/kg	Standard Deviation 0.20213
Prophylaxis	Pharmacokinetics (Pk) - Apparent Volume of Distribution at Steady State (Vss) (One-stage Clotting Assay)	PK-2: BAX 855	0.4715 dL/kg	Standard Deviation 0.14602
Prophylaxis	Pharmacokinetics (Pk) - Apparent Volume of Distribution at Steady State (Vss) (One-stage Clotting Assay)	PK-3: BAX 855, After ≥50 Exposure Days (N=22)	0.4970 dL/kg	Standard Deviation 0.15756

Secondary

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).

Population: Pharmacokinetic full analysis set (PKFAS)

Arm	Measure	Group	Value (MEAN)	Dispersion
Prophylaxis	Pharmacokinetics (Pk) - Area Under the Concentration Versus Time Curve From 0 to Infinity (AUC0-∞) (One-stage Clotting Assay)	PK-1: ADVATE	1168.0 (IU*hours)/dL	Standard Deviation 425.4
Prophylaxis	Pharmacokinetics (Pk) - Area Under the Concentration Versus Time Curve From 0 to Infinity (AUC0-∞) (One-stage Clotting Assay)	PK-2: BAX 855	2073.3 (IU*hours)/dL	Standard Deviation 778.41
Prophylaxis	Pharmacokinetics (Pk) - Area Under the Concentration Versus Time Curve From 0 to Infinity (AUC0-∞) (One-stage Clotting Assay)	PK-3: BAX 855, After ≥50 Exposure Days (N=22)	2008.7 (IU*hours)/dL	Standard Deviation 631.53

Secondary

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).

Population: Pharmacokinetic full analysis set (PKFAS)

Arm	Measure	Group	Value (MEAN)	Dispersion
Prophylaxis	Pharmacokinetics (Pk) - Incremental Recovery Over Time (One-stage Clotting Assay)	PK-1: ADVATE	2.372 (IU/dL)/(IU/kg)	Standard Deviation 0.5357
Prophylaxis	Pharmacokinetics (Pk) - Incremental Recovery Over Time (One-stage Clotting Assay)	PK-2: BAX 855	2.493 (IU/dL)/(IU/kg)	Standard Deviation 0.6944
Prophylaxis	Pharmacokinetics (Pk) - Incremental Recovery Over Time (One-stage Clotting Assay)	PK-3: BAX 855, After ≥50 Exposure Days (N=22)	2.297 (IU/dL)/(IU/kg)	Standard Deviation 0.6377

Secondary

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).

Population: Pharmacokinetic full analysis set (PKFAS)

Arm	Measure	Group	Value (MEAN)	Dispersion
Prophylaxis	Pharmacokinetics (Pk) - Maximum Plasma Concentration (Cmax) (One-stage Clotting Assay)	PK-1: ADVATE	108.45 IU/dL	Standard Deviation 26.25
Prophylaxis	Pharmacokinetics (Pk) - Maximum Plasma Concentration (Cmax) (One-stage Clotting Assay)	PK-2: BAX 855	113.68 IU/dL	Standard Deviation 30.259
Prophylaxis	Pharmacokinetics (Pk) - Maximum Plasma Concentration (Cmax) (One-stage Clotting Assay)	PK-3: BAX 855, After ≥50 Exposure Days (N=22)	103.34 IU/dL	Standard Deviation 29.311

Secondary

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).

Population: Pharmacokinetic full analysis set (PKFAS)

Arm	Measure	Group	Value (MEAN)	Dispersion
Prophylaxis	Pharmacokinetics (Pk) - Mean Residence Time (One-stage Clotting Assay)	PK-1: ADVATE	12.86 hours	Standard Deviation 3.044
Prophylaxis	Pharmacokinetics (Pk) - Mean Residence Time (One-stage Clotting Assay)	PK-2: BAX 855	19.56 hours	Standard Deviation 5.315
Prophylaxis	Pharmacokinetics (Pk) - Mean Residence Time (One-stage Clotting Assay)	PK-3: BAX 855, After ≥50 Exposure Days (N=22)	20.65 hours	Standard Deviation 4.821

Secondary

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).

Population: Pharmacokinetic full analysis set (PKFAS)

Arm	Measure	Group	Value (MEAN)	Dispersion
Prophylaxis	Pharmacokinetics (Pk) - Plasma Half-life (One-stage Clotting Assay)	PK-1: ADVATE	10.40 hours	Standard Deviation 2.244
Prophylaxis	Pharmacokinetics (Pk) - Plasma Half-life (One-stage Clotting Assay)	PK-2: BAX 855	14.30 hours	Standard Deviation 3.838
Prophylaxis	Pharmacokinetics (Pk) - Plasma Half-life (One-stage Clotting Assay)	PK-3: BAX 855, After ≥50 Exposure Days (N=22)	16.02 hours	Standard Deviation 4.922

Secondary

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).

Population: Pharmacokinetic full analysis set (PKFAS)

Arm	Measure	Group	Value (MEAN)	Dispersion
Prophylaxis	Pharmacokinetics (Pk) -Time to Maximum Concentration in Plasma (Tmax) (One-stage Clotting Assay)	PK-1: ADVATE	0.296 hours	Standard Deviation 0.1662
Prophylaxis	Pharmacokinetics (Pk) -Time to Maximum Concentration in Plasma (Tmax) (One-stage Clotting Assay)	PK-2: BAX 855	0.397 hours	Standard Deviation 0.2632
Prophylaxis	Pharmacokinetics (Pk) -Time to Maximum Concentration in Plasma (Tmax) (One-stage Clotting Assay)	PK-3: BAX 855, After ≥50 Exposure Days (N=22)	0.467 hours	Standard Deviation 0.6044

Secondary

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).

Population: Pharmacokinetic full analysis set (PKFAS)

Arm	Measure	Group	Value (MEAN)	Dispersion
Prophylaxis	Pharmacokinetics (Pk) - Total Body Clearance (One-stage Clotting Assay)	PK-1: ADVATE	0.04551 dL/(kg*hours)	Standard Deviation 0.021725
Prophylaxis	Pharmacokinetics (Pk) - Total Body Clearance (One-stage Clotting Assay)	PK-2: BAX 855	0.02760 dL/(kg*hours)	Standard Deviation 0.020288
Prophylaxis	Pharmacokinetics (Pk) - Total Body Clearance (One-stage Clotting Assay)	PK-3: BAX 855, After ≥50 Exposure Days (N=22)	0.02474 dL/(kg*hours)	Standard Deviation 0.008225

Secondary

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.

Population: Full Analysis Set - All bleeding episodes treated with BAX 855 in participants on on-demand and prophylaxis treatment regimens were analyzed as a single group.

Arm	Measure	Value (NUMBER)
Prophylaxis	Rate of Success of BAX 855 for Treatment of Bleeding Episodes	0.96 Bleeding episodes

Secondary

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

Population: Full Analysis Set (FAS) - Note: data analyzed by subsets of FAS (1) participants who received BAX855 prophylactic infusion or (2) BAX855 pharmacokinetic (PK) participants. - Subset of participants who received BAX855 prophylactic infusion: N= 120 - Subset of BAX855 pharmacokinetic (PK) participants: N=26

Arm	Measure	Group	Value (MEAN)	Dispersion
Prophylaxis	Weight-adjusted Consumption of BAX 855 - Per Prophylactic Infusion and Pharmacokinetic (PK) Infusion	Per Prophylactic Infusion (N= 5941 Infusions)	44.51 IU/kg	Standard Deviation 4.556
Prophylaxis	Weight-adjusted Consumption of BAX 855 - Per Prophylactic Infusion and Pharmacokinetic (PK) Infusion	Per PK Infusion (N= 50 Infusions)	45.48 IU/kg	Standard Deviation 2.592

Secondary

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.

Population: Full Analysis Set (FAS) - Note: data analyzed by subsets of FAS (1) participants who received BAX855 for treatment of BEs (2) BAX855 for Maintenance of Hemostasis. - Subset of participants who received BAX855 for treatment of BEs: N= 92 - Subset BAX855 for Maintenance of Hemostasis participants: N=16

Arm	Measure	Group	Value (MEAN)	Dispersion
Prophylaxis	Weight-adjusted Consumption of BAX 855 - Per Treatment of Bleeding Episode (BE) and Per BE for Maintenance of Hemostasis	Per Treatment of BE (N= 592 bleeds)	37.44 IU/kg	Standard Deviation 28.105
Prophylaxis	Weight-adjusted Consumption of BAX 855 - Per Treatment of Bleeding Episode (BE) and Per BE for Maintenance of Hemostasis	Per BE for Maintenance of Hemostasis (N=34 bleeds)	39.29 IU/kg	Standard Deviation 34.206

Source: ClinicalTrials.gov · Data processed: Mar 4, 2026

Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study)

Conditions

Brief summary

Related papers

Interventions

Sponsors

Study design

Eligibility

Inclusion criteria

Exclusion criteria

Design outcomes

Primary

Secondary

Countries

Participant flow

Recruitment details

Pre-assignment details

Participants by arm

Withdrawals & dropouts

Baseline characteristics

Adverse events

Outcome results

Annualized Bleeding Rate (ABR)

Average Number of BAX 855 Infusions Needed for the Treatment of Bleeding Episodes

Change in Vital Signs From Screening - Pulse Rate

Change in Vital Signs From Screening - Respiratory Rate

Change in Vital Signs From Screening - Temperature

Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein

Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase

Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)

Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin

Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes

Changes in Hematology Laboratory Assessments From Screening - Erythrocytes

Changes in Hematology Laboratory Assessments From Screening - Hematocrit

Changes in Hematology Laboratory Assessments From Screening - Hemoglobin

Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)

Changes in Vital Signs From Screening - Blood Pressure

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 With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes

Patient Reported Outcomes: Haemo-SYM Questionnaire, Change in Score From Baseline to End of Study

Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study

Percentage of Participants With Adverse Events

Pharmacokinetics (Pk) - Apparent Volume of Distribution at Steady State (Vss) (One-stage Clotting Assay)

Pharmacokinetics (Pk) - Area Under the Concentration Versus Time Curve From 0 to Infinity (AUC0-∞) (One-stage Clotting Assay)

Pharmacokinetics (Pk) - Incremental Recovery Over Time (One-stage Clotting Assay)

Pharmacokinetics (Pk) - Maximum Plasma Concentration (Cmax) (One-stage Clotting Assay)

Pharmacokinetics (Pk) - Mean Residence Time (One-stage Clotting Assay)

Pharmacokinetics (Pk) - Plasma Half-life (One-stage Clotting Assay)

Pharmacokinetics (Pk) -Time to Maximum Concentration in Plasma (Tmax) (One-stage Clotting Assay)

Pharmacokinetics (Pk) - Total Body Clearance (One-stage Clotting Assay)

Rate of Success of BAX 855 for Treatment of Bleeding Episodes

Weight-adjusted Consumption of BAX 855 - Per Prophylactic Infusion and Pharmacokinetic (PK) Infusion

Weight-adjusted Consumption of BAX 855 - Per Treatment of Bleeding Episode (BE) and Per BE for Maintenance of Hemostasis