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CompletedNCT02585960PROPELUpdated May 25, 2021Results posted

BAX 855 PK-guided Dosing

A Phase 3 interventional study of PEGylated Recombinant Factor VIII and PEGylated Recombinant Factor VIII in Hemophilia A, sponsored by Baxalta now part of Shire. Completed at 83 sites in 22 countries. Open to participants aged 12 Years to 65 Years. Per ClinicalTrials.gov, last updated 2021-05-25.

Sponsored by Baxalta now part of Shire · Phase 3, Interventional, and Prevention

Phase
Phase 3
Study type
Interventional
Enrollment
135
Allocation
Randomized
Ages
12 Years to 65 Years
Sex
All
01

Study summary

  1. To compare the efficacy and safety of pharmacokinetic (PK)-guided treatment with BAX 855 targeting FVIII trough levels of 1-3% and approximately 10% (8-12%)
  2. To further characterize pharmacokinetic (PK) and pharmacodynamic (PD) parameters of BAX 855
02

Conditions studied

  • Hemophilia A

Browse trials for

03

In context

Hemophilia A

866 studies on the registry are indexed under Hemophilia A; 137 are open to participants now.

This study's enrollment of 135 is above the median of 28 across 512 interventional studies indexed under Hemophilia A.

Browse Hemophilia A studies →

Lead sponsor

Baxalta now part of Shire is the lead sponsor of 110 studies on the registry; none are open to participants now.

Of its 18 completed or terminated interventional studies of FDA-regulated products, 16 (89%) have results posted.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
12 Years to 65 Years
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Participants transitioning from another BAX 855 study who meet ALL of the following criteria are eligible for this study:

    1. Participant has completed the end of study visit of a BAX 855 study or is transitioning from the ongoing Baxalta Continuation Study 261302.
    2. Participant is either receiving on-demand treatment or prophylactic treatment with BAX 855 and had an Annual Bleed Rate (ABR) of ≥ 2 documented and treated during the past 12 months.
    3. Participant is human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm\^3, as confirmed by central laboratory.
    4. Participant is willing and able to comply with the requirements of the protocol.

  • Newly recruited participants (ie not transitioning from another BAX 855 study) including BAX855 naïve participants who meet ALL of the following criteria are eligible for this study:

    1. Participant has severe hemophilia A (FVIII clotting activity \< 1%) as confirmed by central laboratory OR by historically documented FVIII clotting activity performed by a certified clinical laboratory, optionally supported by a FVIII gene mutation consistent with severe hemophilia A
    2. Participant has been previously treated with plasma-derived FVIII concentrates or recombinant FVIII for ≥ 150 documented exposure days (EDs)
    3. Participant is either receiving on-demand treatment or prophylactic treatment and had an annual bleeding rate of ≥ 2 documented and treated during the past 12 months.
    4. Participant has a Karnofsky performance score of ≥ 60 at screening
    5. Participant is HIV-; or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm\^3, as confirmed by central laboratory at screening
    6. Participant is hepatitis C virus negative (HCV-) by antibody (if positive, additional PCR testing will be performed), as confirmed by central laboratory at screening; or HCV+ with chronic stable hepatitis
    7. If female of childbearing potential, participant presents with a negative urine pregnancy test and agrees to employ adequate birth control measures for the duration of the study
    8. Participant is willing and able to comply with the requirements of the protocol.

Exclusion criteria

EXCLUSION CRITERIA:

  • Participants transitioning from another BAX 855 study who meet ANY of the following criteria are not eligible for this study:

    1. Participant has developed a confirmed inhibitory antibody to FVIII with a titer of ≥ 0.6 BU using the Nijmegen modification of the Bethesda assay as determined at the central laboratory during the course of the previous BAX 855 study.
    2. Participant has been diagnosed with an acquired hemostatic defect other than hemophilia A.
    3. The participant's weight is \< 35 kg or > 100 kg.
    4. Participant's platelet count is \< 100,000/mL.
    5. Participant has an abnormal renal function (serum creatinine > 1.5 times the upper limit of normal).
    6. Participant has active hepatic disease with alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) levels ≥ 5 times the upper limit of normal.
    7. Participant is scheduled to receive a systemic immunomodulating drug (e.g. corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day, or α-interferon) other than anti-retroviral chemotherapy during the study.
    8. Participant has a clinically significant medical, psychiatric, or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant's safety or compliance.
    9. Participant is planning to take part in any other clinical study during the course of the study.
    10. Participant is a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (ie, children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study.

Newly recruited participants (ie not transitioning from another BAX 855 study) who meet ANY of the following criteria are not eligible for this study:

  1. Participant has detectable FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening.
  2. Participant has a history of confirmed FVIII inhibitors with a titer ≥ 0.6 Bethesda Units (BU) (as determined by the Nijmegen modification of the Bethesda assay or the assay employed with the respective cut-off in the local laboratory) at any time prior to screening.
  3. Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease).
  4. The participant's weight is \< 35 kg or > 100 kg.
  5. Participant's platelet count is \< 100,000/mL.
  6. Participant has known hypersensitivity towards mouse or hamster proteins, PEG or Tween 80.
  7. Participant has severe chronic hepatic dysfunction [eg, ≥ 5 times upper limit of normal alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST), as confirmed by central laboratory at screening, or a documented INR > 1.5].
  8. Participant has severe renal impairment (serum creatinine > 1.5 times the upper limit of normal).
  9. Participant has current or recent (\< 30 days) use of other pegylated drugs prior to study participation or is scheduled to use such drugs during study participation.
  10. Participant is scheduled to receive during the course of the study, a systemic immunomodulating drug (e.g. corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day, or α-interferon) other than anti-retroviral chemotherapy.
  11. Participant has participated in another clinical study involving an IP or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
  12. Participant has a medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance.
  13. Participant is a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (ie, children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study.
05

Study design

Phase
Phase 3
Primary purpose
Prevention
Allocation
Randomized
Intervention model
Parallel assignment
Masking
None (open label)
Enrollment
135 participants (actual)

Study arms

  • Experimental
    Pharmacokinetic (PK) evaluation of BAX 855

    Participants will first undergo an initial pharmacokinetic (PK) assessment. Following the PK assessment participants will be randomized to one of 2 dosing regimens.

    Biological: PEGylated Recombinant Factor VIII

  • Experimental
    FVIII trough target 1-3%

    Standard treatment arm - PK-guided dosing schedule to achieve a Factor VIII (FVIII) trough of 1-3%

    Biological: PEGylated Recombinant Factor VIII

  • Experimental
    FVIII trough target 8-12%

    Intensified treatment arm - PK-guided dosing schedule to achieve a Factor VIII (FVIII) trough of 8-12%

    Biological: PEGylated Recombinant Factor VIII

Interventions

  • BiologicalPEGylated Recombinant Factor VIII

    Pharmacokinetic (PK) evaluation

    Also known as: BAX855, BAX 855

  • BiologicalPEGylated Recombinant Factor VIII

    Standard treatment

    Also known as: BAX 855, BAX855

  • BiologicalPEGylated Recombinant Factor VIII

    Intensified treatment

    Also known as: BAX855, BAX 855

06

What researchers measure

Primary outcomes

  1. Percentage of Participants With a Total Annualized Bleeding Rate (ABR) of Zero for Second Six Months

    Annualized bleeding rate was determined by dividing the number of bleeds by observation period in years.

    Time frame: Day 183 to Day 364 (6 months)

Secondary outcomes

  1. Total Annualized Bleeding Rate for Second Six Months

    Annualized bleeding rate was determined by dividing the number of bleeds by observation period in years.

    Time frame: Day 183 to Day 364 (6 months)

  2. Annualized Spontaneous Bleeding Rate for Second Six Months

    Annualized spontaneous bleeding rate was determined by dividing the number of spontaneous bleeds by observation period in years. A bleed was defined as spontaneous if it was not related to injury/trauma.

    Time frame: Day 183 to Day 364 (6 months)

  3. Annualized Traumatic Bleeding Rate for Second Six Months

    Annualized traumatic bleeding rate was determined by dividing the number of traumatic bleeds by observation period in years. A bleed was defined as traumatic if it was related to injury/trauma.

    Time frame: Day 183 to Day 364 (6 months)

  4. Annualized Joint Bleeding Rate (AJBR) for Second Six Months

    Annualized joint bleeding rate was determined by dividing the number of joint bleeds by observation period in years. An acute joint bleed include some or all of the following: 'aura', pain, swelling, warmth of the skin over the joint, decreased range of motion and difficulty in using the limb compared with baseline or loss of function.

    Time frame: Day 183 to Day 364 (6 months)

  5. Total Weight-adjusted Consumption of BAX 855

    Total weight-adjusted consumption of BAX 855 were reported.

    Time frame: From start of study treatment up to 12 months (completion or termination)

  6. Number of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Number of Infusions

    The participant or caregiver rated the overall treatment response using a 4-point efficacy rating scale as Excellent: Full relief of pain and cessation of objective signs of bleeding after a single infusion and no additional infusion is required for the control of bleeding; Good: Definite pain relief and/or improvement in signs of bleeding after a single infusion and 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 and required more than 1 infusion for complete resolution and None: No improvement or condition worsens.

    Time frame: 8 hours after study drug administration

  7. Number of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Bleed Resolution

    The participant or caregiver rated the overall treatment response using a 4-point efficacy rating scale as Excellent: Full relief of pain and cessation of objective signs of bleeding after a single infusion and no additional infusion is required for the control of bleeding; Good: Definite pain relief and/or improvement in signs of bleeding after a single infusion and 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 and required more than 1 infusion for complete resolution and None: No improvement or condition worsens.

    Time frame: From start of study treatment up to bleed resolution (up to 12 months)

  8. Treatment of Bleeding Episodes: Number of BAX 855 Infusions Per Bleeding Episode Required Until Bleed Resolution

    Infusions of BAX 855 that were required until bleed resolution were reported.

    Time frame: From start of study treatment up to 12 months (completion or termination)

  9. Change From Baseline in Hemophilia Joint Health Score (HJHS)- Total Score

    HJHS was assessed based on the following components of the elbow, knee, and ankle joints: swelling, duration of swelling, muscle atrophy, crepitus on motion, flexion loss, extension loss, joint pain, and strength, together with an assessment of the global gait. The HJHS is a validated 11-item scoring tool based on radiologic and clinical evaluation, sensitive to detect early signs and minor changes. HJHS ranges from 0 to 124. Higher values in the HJHS represent worse situation for the participant.

    Time frame: Baseline, Month 12

  10. Number of Participants With Hemostatic Efficacy Ratings for BAX 855 Treatment of Operative Bleeds

    The participant or caregiver rated the overall treatment response using a 4-point efficacy rating scale as Excellent: Full relief of pain and cessation of objective signs of bleeding after a single infusion and no additional infusion is required for the control of bleeding; Good: Definite pain relief and/or improvement in signs of bleeding after a single infusion and 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 and required more than 1 infusion for complete resolution and None: No improvement or condition worsens. Hemostatic efficacy was evaluated intra-operatively (from start to end of the procedure), post-operatively (from the end of procedure up to 24 h post procedure), and perioperatively (from the start of procedure to participant discharge from hospital or 14 days after completion of procedure; whichever was first).

    Time frame: Day 0 through discharge or 14 days post-surgery

  11. Blood Loss Per Participant in Case of Surgery

    The intraoperative blood loss was measured by determining the volume of blood and fluid removal through suction into the collection container (waste box and/or cell saver) and the estimated blood loss into swabs and towels during the procedure, per the anesthesiologist's record. Postoperatively, blood loss was determined by the drainage volume collected, which mainly consisted of drainage fluid via vacuum or gravity drain, as applicable. In cases where no drain was present, blood loss was determined by the surgeon's clinical judgment, as applicable or entered as "not available". Blood loss was evaluated intra-operatively (from start to end of the procedure), post-operatively (from the end of procedure up to 24 h post procedure), and perioperatively (from the start of procedure to participant discharge from hospital or 14 days after completion of procedure; whichever was first).

    Time frame: Day 0 through discharge or 14 days post-surgery

  12. Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

    An AE was any unfavorable and unintended sign (an abnormal laboratory finding), symptom (rash, pain, discomfort, fever, dizziness, etc.), disease (peritonitis, bacteremia, etc.), or outcome of death temporally associated with the use of an investigational product (IP), whether or not considered causally related to the IP. A SAE was defined as an untoward medical occurrence that at any dose met one or more of the following criteria: outcome was fatal/results in death, life-threatening, required in-patient hospitalization or resulted in prolongation of an existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event that was not immediately life-threatening or resulted in death or required hospitalization but jeopardize the participant or required medical or surgical intervention to prevent any of the above outcomes.

    Time frame: From start of study treatment up to 12 months (completion or termination)

  13. Number of Participants With Clinically Significant Changes in Vital Signs Reported as Treatment Related Adverse Events

    Vital signs included systolic and diastolic blood pressure, pulse rate, respiratory rate, body temperature.

    Time frame: From start of study treatment up to 12 months (completion or termination)

  14. Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters Reported as Treatment Related Adverse Events

    Clinical laboratory assessments included clinical chemistry, hematology, lipid panel, genetics, T-cell, B-cell and NK cell (TBNK) and viral serology.

    Time frame: From start of study treatment up to 12 months (completion or termination)

  15. Number of Participants With Positive Inhibitory Antibodies and Binding Antibodies to Factor VIII (FVIII), BAX 855, Polyethylene Glycol (PEG), and Chinese Hamster Ovary (CHO) Protein

    Positive Inhibitory Antibodies and Binding Antibodies to Factor VIII (FVIII), BAX 855, Polyethylene Glycol (PEG), and Chinese Hamster Ovary (CHO) Protein were reported here.

    Time frame: From start of study treatment up to 12 months (completion or termination)

  16. Change From Baseline in Physical Component Scores (PCS) of the Short Form-36 (SF-36) Health Survey

    Short Form (36) Health Survey (SF-36) is a 36-item validated, generic health related quality of life (HR QoL) instrument. PCS is a summary scale of the dimensions physical functioning, role physical, bodily pain, and general health. The component score is normalized to a standard population. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both sub-scores and summary scores.

    Time frame: Baseline, Month 12 (completion or termination)

  17. Area Under the Plasma Concentration of BAX 855 From Zero to Infinity (AUC0-inf)

    Area under the plasma concentration versus time curve from time 0 to infinity of BAX 855 were reported.

    Time frame: Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion

  18. Incremental Recovery (IR) at Maximum Plasma Concentration (Cmax) of BAX 855

    IR at Cmax of BAX 855 were reported.

    Time frame: Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion

  19. Plasma Half-life (T1/2) of BAX 855

    T1/2 of BAX 855 in plasma were reported.

    Time frame: Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion

  20. Mean Residence Time (MRT) of BAX 855

    MRT of BAX 855 were reported.

    Time frame: Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion

  21. Maximum Plasma Concentration (Cmax) of BAX 855

    Cmax of BAX 855 were reported.

    Time frame: Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion

  22. Time to Maximum Concentration of BAX 855 in Plasma (Tmax)

    Tmax of BAX 855 were reported.

    Time frame: Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion

  23. Total Body Clearance (CL) of BAX 855

    Total body clearance of BAX 855 from blood by the kidney were reported.

    Time frame: Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion

  24. Volume of Distribution at Steady State (Vss)

    Volume of distribution was defined as the theoretical volume in which the total amount of drug was uniformly distributed to produce the desired blood concentration of a drug. Vss is the apparent volume of distribution at steadystate.

    Time frame: Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion

  25. Incremental Recovery (IR) Over Time

    Incremental recovery was calculated by BAX 855 increment (IU/dL) / BAX 855 dose (IU/kg).

    Time frame: Baseline, Month 3, 6, 7.5, 9, 10.5, 12 (Completion or termination)

07

Results

Posted Aug 26, 2019

Participant flow

The study was conducted at 62 study centers in 19 countries between 23 November 2015 (first participant first visit) and 05 August 2018 (last participant last visit).

PK/Safety Assessment Period
Participant flow — PK/Safety Assessment Period
MilestoneBAX 855-Low LevelBAX 855-High LevelBAX 855-Non-randomized
Started57586
Completed 1st 6 month period57530
Completed 2nd 6 month period52480
Completed52480
Not completed5106
Withdrew: Physician decision010
Withdrew: Withdrawal by subject125
Withdrew: Non-compliance to study procedures020
Withdrew: Other440
Withdrew: Withdrawn by sponsor010
Withdrew: Screen failure001
Prophylactic Treatment Period
Participant flow — Prophylactic Treatment Period
MilestoneBAX 855-Low LevelBAX 855-High LevelBAX 855-Non-randomized
Started52480
Completed52430
Not completed050
Withdrew: Major protocol deviation040
Withdrew: Subject less than 75% exposed to bax 855010

Outcome measures

PrimaryPercentage of Participants With a Total Annualized Bleeding Rate (ABR) of Zero for Second Six Months

Annualized bleeding rate was determined by dividing the number of bleeds by observation period in years.

Time frame:
Day 183 to Day 364 (6 months)
Reported as:
Number · Percentage of participants
Percentage of Participants With a Total Annualized Bleeding Rate (ABR) of Zero for Second Six Months
Percentage of participantsBAX 855-Low LevelBAX 855-High Level
Percentage of Participants With a Total Annualized Bleeding Rate (ABR) of Zero for Second Six Months42.162.1
Statistical analysis
  • BAX 855-Low Level vs BAX 855-High Level · Chi-squared · p = 0.0545 · Gaussian statistic estimate: 1.960 · 95% CI -0.038 to 3.958Approximately Gaussian Statistic is obtained by taking the square root of the Chi-squared test with continuity adjustment.
SecondaryTotal Annualized Bleeding Rate for Second Six Months

Annualized bleeding rate was determined by dividing the number of bleeds by observation period in years.

Time frame:
Day 183 to Day 364 (6 months)
Reported as:
Mean · Bleeds per year
Total Annualized Bleeding Rate for Second Six Months
Bleeds per yearBAX 855-Low LevelBAX 855-High Level
Total Annualized Bleeding Rate for Second Six Months3.603 ± 7.5121.649 ± 3.433
SecondaryAnnualized Spontaneous Bleeding Rate for Second Six Months

Annualized spontaneous bleeding rate was determined by dividing the number of spontaneous bleeds by observation period in years. A bleed was defined as spontaneous if it was not related to injury/trauma.

Time frame:
Day 183 to Day 364 (6 months)
Reported as:
Mean · Bleeds per year
Annualized Spontaneous Bleeding Rate for Second Six Months
Bleeds per yearBAX 855 - Low LevelBAX 855 - High Level
Annualized Spontaneous Bleeding Rate for Second Six Months2.489 ± 6.5540.737 ± 1.738
SecondaryAnnualized Traumatic Bleeding Rate for Second Six Months

Annualized traumatic bleeding rate was determined by dividing the number of traumatic bleeds by observation period in years. A bleed was defined as traumatic if it was related to injury/trauma.

Time frame:
Day 183 to Day 364 (6 months)
Reported as:
Mean · Bleeds per year
Annualized Traumatic Bleeding Rate for Second Six Months
Bleeds per yearBAX 855-Low LevelBAX 855-High Level
Annualized Traumatic Bleeding Rate for Second Six Months1.114 ± 2.0370.912 ± 2.647
SecondaryAnnualized Joint Bleeding Rate (AJBR) for Second Six Months

Annualized joint bleeding rate was determined by dividing the number of joint bleeds by observation period in years. An acute joint bleed include some or all of the following: 'aura', pain, swelling, warmth of the skin over the joint, decreased range of motion and difficulty in using the limb compared with baseline or loss of function.

Time frame:
Day 183 to Day 364 (6 months)
Reported as:
Mean · Bleeds per year
Annualized Joint Bleeding Rate (AJBR) for Second Six Months
Bleeds per yearBAX 855-Low LevelBAX 855-High Level
Annualized Joint Bleeding Rate (AJBR) for Second Six Months2.617 ± 7.3611.079 ± 2.553
SecondaryTotal Weight-adjusted Consumption of BAX 855

Total weight-adjusted consumption of BAX 855 were reported.

Time frame:
From start of study treatment up to 12 months (completion or termination)
Reported as:
Mean · International units per kilogram (IU/kg)
Total Weight-adjusted Consumption of BAX 855
International units per kilogram (IU/kg)BAX 855-Low LevelBAX 855-High Level
Total Weight-adjusted Consumption of BAX 8553984.593 ± 1678.4617030.714 ± 3208.049
SecondaryNumber of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Number of Infusions

The participant or caregiver rated the overall treatment response using a 4-point efficacy rating scale as Excellent: Full relief of pain and cessation of objective signs of bleeding after a single infusion and no additional infusion is required for the control of bleeding; Good: Definite pain relief and/or improvement in signs of bleeding after a single infusion and 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 and required more than 1 infusion for complete resolution and None: No improvement or condition worsens.

Time frame:
8 hours after study drug administration
Reported as:
Number · Treated Bleeds
Number of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Number of Infusions
Treated BleedsBAX 855-Low LevelBAX 855-High Level
Excellent: Bleeds treated with 1 infusion1917
Excellent: Bleeds treated with 2 infusions31
Excellent: Bleeds treated with 3 infusions01
Excellent: Bleeds treated with >= 4 infusions00
Good: Bleeds treated with 1 infusion3616
Good: Bleeds treated with 2 infusions166
Good: Bleeds treated with 3 infusions51
Good: Bleeds treated with >= 4 infusion22
Fair: Bleeds treated with 1 infusion20
Fair: Bleeds treated with 2 infusions70
Fair: Bleeds treated with 3 infusions40
Fair: Bleeds treated with >= 4 infusions10
None: Bleeds treated with 1 infusion10
None: Bleeds treated with 2 infusions10
None: Bleeds treated with 3 infusions01
None: Bleeds treated with >= 4 infusions01
SecondaryNumber of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Bleed Resolution

The participant or caregiver rated the overall treatment response using a 4-point efficacy rating scale as Excellent: Full relief of pain and cessation of objective signs of bleeding after a single infusion and no additional infusion is required for the control of bleeding; Good: Definite pain relief and/or improvement in signs of bleeding after a single infusion and 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 and required more than 1 infusion for complete resolution and None: No improvement or condition worsens.

Time frame:
From start of study treatment up to bleed resolution (up to 12 months)
Reported as:
Number · Treated Bleeds
Number of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Bleed Resolution
Treated BleedsBAX 855-Low LevelBAX 855-High Level
Excellent: Bleeds treated with 1 infusion5034
Excellent: Bleeds treated with 2 infusions42
Good:Bleeds treated with 1 infusion4724
Good:Bleeds treated with 2 infusions197
Good:Bleeds treated with 3 infusions63
Good:Bleeds treated with >= 4 infusions36
Fair:Bleeds treated with 1 infusion30
Fair:Bleeds treated with 2 infusions51
Fair:Bleeds treated with 3 infusions70
Fair:Bleeds treated with >= 4 infusions30
None:Bleeds treated with 2 infusions10
None:Bleeds treated with 3 infusions01
SecondaryTreatment of Bleeding Episodes: Number of BAX 855 Infusions Per Bleeding Episode Required Until Bleed Resolution

Infusions of BAX 855 that were required until bleed resolution were reported.

Time frame:
From start of study treatment up to 12 months (completion or termination)
Reported as:
Mean · Infusions
Treatment of Bleeding Episodes: Number of BAX 855 Infusions Per Bleeding Episode Required Until Bleed Resolution
InfusionsBAX 855-Low LevelBAX 855-High Level
Treatment of Bleeding Episodes: Number of BAX 855 Infusions Per Bleeding Episode Required Until Bleed Resolution1.6 ± 1.191.6 ± 1.36
SecondaryChange From Baseline in Hemophilia Joint Health Score (HJHS)- Total Score

HJHS was assessed based on the following components of the elbow, knee, and ankle joints: swelling, duration of swelling, muscle atrophy, crepitus on motion, flexion loss, extension loss, joint pain, and strength, together with an assessment of the global gait. The HJHS is a validated 11-item scoring tool based on radiologic and clinical evaluation, sensitive to detect early signs and minor changes. HJHS ranges from 0 to 124. Higher values in the HJHS represent worse situation for the participant.

Time frame:
Baseline, Month 12
Reported as:
Mean · Score on a scale
Change From Baseline in Hemophilia Joint Health Score (HJHS)- Total Score
Score on a scaleBAX 855-Low LevelBAX 855-High Level
Change From Baseline in Hemophilia Joint Health Score (HJHS)- Total Score-1.9 ± 5.25-1.1 ± 7.77
SecondaryNumber of Participants With Hemostatic Efficacy Ratings for BAX 855 Treatment of Operative Bleeds

The participant or caregiver rated the overall treatment response using a 4-point efficacy rating scale as Excellent: Full relief of pain and cessation of objective signs of bleeding after a single infusion and no additional infusion is required for the control of bleeding; Good: Definite pain relief and/or improvement in signs of bleeding after a single infusion and 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 and required more than 1 infusion for complete resolution and None: No improvement or condition worsens. Hemostatic efficacy was evaluated intra-operatively (from start to end of the procedure), post-operatively (from the end of procedure up to 24 h post procedure), and perioperatively (from the start of procedure to participant discharge from hospital or 14 days after completion of procedure; whichever was first).

Time frame:
Day 0 through discharge or 14 days post-surgery
Reported as:
Count of participants · Participants
Number of Participants With Hemostatic Efficacy Ratings for BAX 855 Treatment of Operative Bleeds
ParticipantsBAX 855-Low LevelBAX 855-High Level
Excellent: Intra-operative13
Excellent: Post-operative34
Excellent: Peri-operative24
Good: Intra-operative00
Good: Post-operative00
Good: Peri-operative00
Fair: Intra-operative01
Fair: Post-operative00
Fair: Peri-operative00
None: Intra-operative00
None: Post-operative00
None: Peri-operative00
Unknown: Intra-operative20
Unknown: Post-operative00
Unknown: Peri-operative10
SecondaryBlood Loss Per Participant in Case of Surgery

The intraoperative blood loss was measured by determining the volume of blood and fluid removal through suction into the collection container (waste box and/or cell saver) and the estimated blood loss into swabs and towels during the procedure, per the anesthesiologist's record. Postoperatively, blood loss was determined by the drainage volume collected, which mainly consisted of drainage fluid via vacuum or gravity drain, as applicable. In cases where no drain was present, blood loss was determined by the surgeon's clinical judgment, as applicable or entered as "not available". Blood loss was evaluated intra-operatively (from start to end of the procedure), post-operatively (from the end of procedure up to 24 h post procedure), and perioperatively (from the start of procedure to participant discharge from hospital or 14 days after completion of procedure; whichever was first).

Time frame:
Day 0 through discharge or 14 days post-surgery
Reported as:
Mean · Milliliters (mL)
Blood Loss Per Participant in Case of Surgery
Milliliters (mL)BAX 855-Low LevelBAX 855-High Level
Intra-operative: Observed4.400 ± 4.01772.000 ± 160.741
Intra-operative: Predicted Average10.600 ± 9.22765.833 ± 139.290
Intra-operative: Predicted Maximum20.800 ± 18.089128.333 ± 231.790
Post-operative: Observed—820.000 ± NA
Post-operative: Predicted Average10.000 ± 9.129145.000 ± 320.967
Post-operative: Predicted Maximum20.200 ± 17.987230.000 ± 475.563
SecondaryNumber of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)

An AE was any unfavorable and unintended sign (an abnormal laboratory finding), symptom (rash, pain, discomfort, fever, dizziness, etc.), disease (peritonitis, bacteremia, etc.), or outcome of death temporally associated with the use of an investigational product (IP), whether or not considered causally related to the IP. A SAE was defined as an untoward medical occurrence that at any dose met one or more of the following criteria: outcome was fatal/results in death, life-threatening, required in-patient hospitalization or resulted in prolongation of an existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect, was a medically important event that was not immediately life-threatening or resulted in death or required hospitalization but jeopardize the participant or required medical or surgical intervention to prevent any of the above outcomes.

Time frame:
From start of study treatment up to 12 months (completion or termination)
Reported as:
Count of participants · Participants
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
ParticipantsBAX 855-Low LevelBAX 855-High LevelBAX 855-Non-randomized
Number of Participants with SAE550
Number of Participants with AE35380
SecondaryNumber of Participants With Clinically Significant Changes in Vital Signs Reported as Treatment Related Adverse Events

Vital signs included systolic and diastolic blood pressure, pulse rate, respiratory rate, body temperature.

Time frame:
From start of study treatment up to 12 months (completion or termination)
Reported as:
Count of participants · Participants
Number of Participants With Clinically Significant Changes in Vital Signs Reported as Treatment Related Adverse Events
ParticipantsBAX 855-Low LevelBAX 855-High LevelBAX 855-Non-randomized
Number of Participants With Clinically Significant Changes in Vital Signs Reported as Treatment Related Adverse Events000
SecondaryNumber of Participants With Clinically Significant Changes in Clinical Laboratory Parameters Reported as Treatment Related Adverse Events

Clinical laboratory assessments included clinical chemistry, hematology, lipid panel, genetics, T-cell, B-cell and NK cell (TBNK) and viral serology.

Time frame:
From start of study treatment up to 12 months (completion or termination)
Reported as:
Count of participants · Participants
Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters Reported as Treatment Related Adverse Events
ParticipantsBAX 855-Low LevelBAX 855-High LevelBAX 855-Non-randomized
Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters Reported as Treatment Related Adverse Events210
SecondaryNumber of Participants With Positive Inhibitory Antibodies and Binding Antibodies to Factor VIII (FVIII), BAX 855, Polyethylene Glycol (PEG), and Chinese Hamster Ovary (CHO) Protein

Positive Inhibitory Antibodies and Binding Antibodies to Factor VIII (FVIII), BAX 855, Polyethylene Glycol (PEG), and Chinese Hamster Ovary (CHO) Protein were reported here.

Time frame:
From start of study treatment up to 12 months (completion or termination)
Reported as:
Count of participants · Participants
Number of Participants With Positive Inhibitory Antibodies and Binding Antibodies to Factor VIII (FVIII), BAX 855, Polyethylene Glycol (PEG), and Chinese Hamster Ovary (CHO) Protein
ParticipantsBAX 855-Low LevelBAX 855-High Level
Binding IgG antibodies to FVIII03
Binding IgM antibodies to FVIII00
Binding IgG antibodies to PEG-FVIII27
Binding IgM antibodies to PEG-FVIII01
Binding IgG antibodies to PEG00
Binding IgM antibodies to PEG13
Binding Ig antibodies to CHO00
Inhibitory antibodies to FVIII01
SecondaryChange From Baseline in Physical Component Scores (PCS) of the Short Form-36 (SF-36) Health Survey

Short Form (36) Health Survey (SF-36) is a 36-item validated, generic health related quality of life (HR QoL) instrument. PCS is a summary scale of the dimensions physical functioning, role physical, bodily pain, and general health. The component score is normalized to a standard population. Scores range from 0 to 100 with higher scores representing better health. There is no total overall score; scoring is done for both sub-scores and summary scores.

Time frame:
Baseline, Month 12 (completion or termination)
Reported as:
Mean · Score on a scale
Change From Baseline in Physical Component Scores (PCS) of the Short Form-36 (SF-36) Health Survey
Score on a scaleBAX 855-Low LevelBAX 855-High Level
Change From Baseline in Physical Component Scores (PCS) of the Short Form-36 (SF-36) Health Survey3.551 ± 8.3512.846 ± 8.658
SecondaryArea Under the Plasma Concentration of BAX 855 From Zero to Infinity (AUC0-inf)

Area under the plasma concentration versus time curve from time 0 to infinity of BAX 855 were reported.

Time frame:
Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion
Reported as:
Mean · International units*hour per deciliter
Area Under the Plasma Concentration of BAX 855 From Zero to Infinity (AUC0-inf)
International units*hour per deciliterBAX 855-Low LevelBAX 855-High LevelBAX 855-Non-randomized
Area Under the Plasma Concentration of BAX 855 From Zero to Infinity (AUC0-inf)2673 ± 877.32659 ± 10412214 ± 355.8
SecondaryIncremental Recovery (IR) at Maximum Plasma Concentration (Cmax) of BAX 855

IR at Cmax of BAX 855 were reported.

Time frame:
Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion
Reported as:
Mean · (IU/dL) / (IU/kg)
Incremental Recovery (IR) at Maximum Plasma Concentration (Cmax) of BAX 855
(IU/dL) / (IU/kg)BAX 855-Low LevelBAX 855-High LevelBAX 855-Non-randomized
Incremental Recovery (IR) at Maximum Plasma Concentration (Cmax) of BAX 8552.227 ± 0.52012.231 ± 0.54512.478 ± 0.2016
SecondaryPlasma Half-life (T1/2) of BAX 855

T1/2 of BAX 855 in plasma were reported.

Time frame:
Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion
Reported as:
Median · hour (h)
Plasma Half-life (T1/2) of BAX 855
hour (h)BAX 855-Low LevelBAX 855-High LevelBAX 855-Non-randomized
Plasma Half-life (T1/2) of BAX 85515.28 (8.77 to 31.9)14.66 (6.78 to 35.8)10.97 (8.67 to 12.5)
SecondaryMean Residence Time (MRT) of BAX 855

MRT of BAX 855 were reported.

Time frame:
Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion
Reported as:
Median · hour (h)
Mean Residence Time (MRT) of BAX 855
hour (h)BAX 855-Low LevelBAX 855-High LevelBAX 855-Non-randomized
Mean Residence Time (MRT) of BAX 85522.77 (12.6 to 41.7)21.50 (10.1 to 52.5)16.18 (12.6 to 18.2)
SecondaryMaximum Plasma Concentration (Cmax) of BAX 855

Cmax of BAX 855 were reported.

Time frame:
Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion
Reported as:
Mean · International units per deciliter(IU/dL)
Maximum Plasma Concentration (Cmax) of BAX 855
International units per deciliter(IU/dL)BAX 855-Low LevelBAX 855-High LevelBAX 855-Non-randomized
Maximum Plasma Concentration (Cmax) of BAX 855132.54 ± 31.83135.65 ± 33.10149.18 ± 12.86
SecondaryTime to Maximum Concentration of BAX 855 in Plasma (Tmax)

Tmax of BAX 855 were reported.

Time frame:
Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion
Reported as:
Median · hour (h)
Time to Maximum Concentration of BAX 855 in Plasma (Tmax)
hour (h)BAX 855-Low LevelBAX 855-High LevelBAX 855-Non-randomized
Time to Maximum Concentration of BAX 855 in Plasma (Tmax)0.467 (0.30 to 3.08)0.475 (0.25 to 3.25)0.417 (0.38 to 0.53)
SecondaryTotal Body Clearance (CL) of BAX 855

Total body clearance of BAX 855 from blood by the kidney were reported.

Time frame:
Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion
Reported as:
Mean · Deciliters per kilogram * hour (dL/kg*h)
Total Body Clearance (CL) of BAX 855
Deciliters per kilogram * hour (dL/kg*h)BAX 855-Low LevelBAX 855-High LevelBAX 855-Non-randomized
Total Body Clearance (CL) of BAX 8550.02477 ± 0.0095800.02624 ± 0.0093330.02774 ± 0.004385
SecondaryVolume of Distribution at Steady State (Vss)

Volume of distribution was defined as the theoretical volume in which the total amount of drug was uniformly distributed to produce the desired blood concentration of a drug. Vss is the apparent volume of distribution at steadystate.

Time frame:
Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion
Reported as:
Mean · Deciliters per kilogram (dL/kg)
Volume of Distribution at Steady State (Vss)
Deciliters per kilogram (dL/kg)BAX 855-Low LevelBAX 855-High LevelBAX 855-Non-randomized
Volume of Distribution at Steady State (Vss)0.5147 ± 0.12090.5158 ± 0.1062149.18 ± 12.86
SecondaryIncremental Recovery (IR) Over Time

Incremental recovery was calculated by BAX 855 increment (IU/dL) / BAX 855 dose (IU/kg).

Time frame:
Baseline, Month 3, 6, 7.5, 9, 10.5, 12 (Completion or termination)
Reported as:
Mean · IU/dL per IU/kg
Incremental Recovery (IR) Over Time
IU/dL per IU/kgBAX 855-Low LevelBAX 855-High Level
Baseline2.68 ± 0.5132.70 ± 0.450
Month 32.68 ± 0.5152.66 ± 0.459
Month 62.62 ± 0.5852.76 ± 0.552
Month 7.52.53 ± 0.3602.71 ± 0.545
Month 92.65 ± 0.5112.68 ± 0.545
Month 10.52.61 ± 0.4672.58 ± 0.584
Completion/ Termination2.71 ± 0.5532.73 ± 0.689

Adverse events

Collected over From start of study treatment up to 12 months (completion or termination). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
BAX 855-Low Level0/57 (0%)5/57 (8.8%)25/57 (43.9%)
BAX 855-High Level0/58 (0%)5/58 (8.6%)25/58 (43.1%)
BAX 855-Non-randomized0/6 (0%)0/6 (0%)0/6 (0%)
Most frequent serious events
Showing 10 of 11
Most frequent serious events
EventBAX 855-Low LevelBAX 855-High LevelBAX 855-Non-randomized
SynovitisMusculoskeletal and connective tissue disorders1/570/580/6
Abscess limbInfections and infestations1/570/580/6
AppendicitisInfections and infestations1/570/580/6
CellulitisInfections and infestations1/570/580/6
Head injuryInjury, poisoning and procedural complications1/571/580/6
Radius fractureInjury, poisoning and procedural complications1/570/580/6
Factor viii inhibitionBlood and lymphatic system disorders0/571/580/6
Hand fractureInjury, poisoning and procedural complications0/571/580/6
LacerationInjury, poisoning and procedural complications0/571/580/6
Multiple injuriesInjury, poisoning and procedural complications0/571/580/6
Most frequent other events
Most frequent other events
EventBAX 855-Low LevelBAX 855-High LevelBAX 855-Non-randomized
Upper respiratory tract infectionInfections and infestations6/5712/580/6
ArthralgiaMusculoskeletal and connective tissue disorders6/575/580/6
NasopharyngitisInfections and infestations6/575/580/6
HeadacheNervous system disorders5/576/580/6
DiarrhoeaGastrointestinal disorders3/572/580/6
RhinitisInfections and infestations3/573/580/6
SinusitisInfections and infestations3/570/580/6
Back painMusculoskeletal and connective tissue disorders1/573/580/6
PyrexiaGeneral disorders2/573/580/6

Baseline characteristics

Safety Analysis Set (SAS) included all participants enrolled who had at least one BAX 855 infusion.

Age, Continuous
Age, Continuous(Years)BAX 855-Low LevelBAX 855-High LevelBAX 855-Non-randomizedTotal
Mean31.1 ± 13.7631.2 ± 12.2225.8 ± 10.0330.9 ± 12.84
Sex: Female, Male
Sex: Female, Male(Participants)BAX 855-Low LevelBAX 855-High LevelBAX 855-Non-randomizedTotal
Female0000
Male57586121
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)BAX 855-Low LevelBAX 855-High LevelBAX 855-Non-randomizedTotal
Hispanic or Latino2529
Not Hispanic or Latino53534110
Unknown or Not Reported2002
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)BAX 855-Low LevelBAX 855-High LevelBAX 855-Non-randomizedTotal
Asian1418234
White4036480
Native Latin American1102
Mestizo0101
Other2204
08

Study locations

83 sites
  • Phoenix Childrens Hospital
    Phoenix, Arizona 85016-7710, United States
  • Arizona Hemophilia & Thrombosis Center, located within The University of Arizona Cancer Center
    Tucson, Arizona 85724, United States
  • Children's Hospital Los Angeles
    Los Angeles, California 90027, United States
  • University of Colorado
    Aurora, Colorado 80045, United States
  • University of Florida College of Medicine
    Gainesville, Florida 32610, United States
  • Emory University-ECC
    Atlanta, Georgia 30322, United States
  • University of Kentucky Medical Center
    Lexington, Kentucky 40504, United States
  • University of Louisville KCPCRU
    Louisville, Kentucky 40202, United States
  • Tulane University
    New Orleans, Louisiana 70112, United States
  • Boston Children's Hospital
    Boston, Massachusetts 02115, United States
  • University of Nebraska Medical Center
    Omaha, Nebraska 68198-5456, United States
  • Gulf States Hemophilia Centre
    Houston, Texas 77030, United States
  • University of Utah
    Salt Lake City, Utah 84132, United States
  • University of Washington
    Seattle, Washington 98104, United States
  • Royal Brisbane Women's Hospital
    Herston, Queensland 4006, Australia
  • The Perth Blood Institute
    Nedlands, Western Australia 6009, Australia
  • AKH - Medizinische Universität Wien
    Vienna, 1090, Austria
  • UMHAT "Sv. Georgi", EAD
    Plovdiv, 4002, Bulgaria
  • UMHAT 'Tsaritsa Yoanna - ISUL', EAD
    Sofia, 1527, Bulgaria
  • MHAT 'Sv. Marina', EAD, Clinic of Clinical Hematology
    Varna, 9010, Bulgaria
  • CHU Nice- Service hematologie
    Nice, Alpes Maritimes 06200, France
  • Hôpital Morvan
    Brest Cedex, Finistere 29609, France
  • CHU Rennes - Hopital Pontchaillou
    Rennes cedex 09, Ille Et Vilaine 35033, France
  • CHU de Caen - Hôpital Côte de Nacre
    Caen, 14003, France
  • CHU Charles Nicolle
    Rouen, 76031, France
  • HZRM Hamophilie Zentrum Rhein Main GmbH
    Mörfelden-Walldorf, Hessen 65446, Germany
  • Inst. f. Experimentelle Hamatologie u. Transfusionsmedizin
    Bonn, Nordrhein Westfalen 53127, Germany
  • COAGULATION RESEARCH CENTRE GmbH
    Duisburg, Nordrhein Westfalen 47051, Germany
  • Hamophiliezentrum/Gerinnungssprechstunde
    Berlin, 10249, Germany
  • MVZ Labor Dr. Reising-Ackermann
    Leipzig, 04289, Germany
  • University of Hong Kong
    Hong Kong, Hong Kong
  • Prince of Wales Hospital
    Shatin, 00000, Hong Kong
  • Magyar Honvedseg EK
    Budapest, 1134, Hungary
  • DE OEC Belgyógyászati Int
    Debrecen, 4032, Hungary
  • PTE ÁOK
    Pecs, 7624, Hungary
  • Chaim Sheba Medical Center
    Tel-Hashomer, 5262000, Israel
  • UOC Ematologia, Azienda ULSS 8 Asolo, Regione Veneto
    Castelfranco Veneto, Treviso 31033, Italy
  • Presidio Osped. Ferrarotto
    Catania, 90124, Italy
  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
    Milano, 20122, Italy
  • Umberto I Pol. di Roma-Università di Roma La Sapienza
    Roma, 00144, Italy
  • Fondazione Policlinico Universitario A. Gemelli
    Roma, 00168, Italy
  • AOU Citta della Salute e della Scienza - Presidio Molinette
    Torino, 10126, Italy
  • ULSS n. 6 "Vicenza"
    Vicenza, 36100, Italy
  • Hospital Ampang
    Ampang, Kuala Lumpur 68000, Malaysia
  • Hospital Queen Elizabeth
    Kota Kinabalu, Sabah 88586, Malaysia
  • Hospital Kuala Lumpur
    Kuala Lumpur, 50586, Malaysia
  • Hospital Melaka
    Melaka, 75400, Malaysia
  • Hospital Pulau Pinang
    Pulau Pinang, 10450, Malaysia
  • Oslo Universitetssykehus HF
    Oslo, 0372, Norway
  • Wojewodzki Szpital Specjalistyczny im. Mikolaja Kopernika, Klinika Hematologii
    Lodz, 93-510, Poland
  • Alvamed
    Poznań, 61-828, Poland
  • Instytut Hematologii Ii Transfuzjologii
    Warszawa, 02-776, Poland
  • SP Szpital Kliniczny Nr 1 we Wroclawiu
    Wroclaw, 50-367, Poland
  • Spitalul Clinic Judetean de Urgenta Brasov
    Brasov, 500365, Romania
  • Institutul Oncologic ClNa.
    Cluj Napoca, 400124, Romania
  • National University Hospital
    Singapore, 119074, Singapore
  • Singapore General Hospital- Parent
    Singapore, 169608, Singapore
  • KK Women's And Children's Hospital
    Singapore, 229899, Singapore
  • Hospital Universitari Son Espases
    Palma de Mallorca, Baleares 07010, Spain
  • Complejo Hospitalario Universitario A Coruña
    A Coruña, La Coruña 15006, Spain
  • Hospital Regional Universitario de Malaga
    Malaga, Málaga 29010, Spain
  • Hospital Universitario La Paz
    Madrid, 28046, Spain
  • Sahlgrenska Universitetssjukhuset
    Gothenburg, S-41345, Sweden
  • Karolinska Universitetssjukhuset
    Stockholm, 17176, Sweden
  • Universitätsspital Zürich
    Zürich, 8091, Switzerland
  • China Medical University Hospital
    Taichung, 40447, Taiwan
  • Taichung Veterans General Hospital
    Taichung, 40705, Taiwan
  • Tri-Service General Hospital
    Taipei, 11490, Taiwan
  • Acibadem Hastanesi
    Adana, 01130, Turkey
  • Akdeniz University
    Antalya, 07070, Turkey
  • Istanbul University
    Istanbul, 34098, Turkey
  • Ege University
    Izmir, 35040, Turkey
  • NChSH Okhmatdyt of MoHU Center of Children Oncohematology and Bone Marrow Transplantation
    Kyiv, 1135, Ukraine
  • Kyiv CCH #9 Dept of Surgery City SPC of Diagnostics & Treatment of Patients with HP
    Kyiv, 4112, Ukraine
  • SI Institute of Blood Pathology and Transfusion Medicine of NAMSU
    Lviv, 79044, Ukraine
  • M.V. Sklifosovskyi Poltava RCH Dept of Gematology HSEIU Ukrainian Medical Stomatological Academy
    Poltava, 36011, Ukraine
  • Bristol Royal Hospital for Children
    Bristol, Avon BS2 8BJ, United Kingdom
  • Royal Free Hospital
    London, Greater London NW3 2QG, United Kingdom
  • Royal Manchester Children's Hospital
    Manchester, Greater Manchester M13 9WL, United Kingdom
  • Southampton General Hospital
    Southampton, Hampshire SO16 6YD, United Kingdom
  • Leicester Royal Infirmary
    Leicester, Leicestershire LE1 5WW, United Kingdom
  • Churchill Hospital
    Oxford, Oxfordshire OX3 7LJ, United Kingdom
  • University Hospital of Wales
    Cardiff, West Glamorgan CF14 4XN, United Kingdom
09

References and documents

Publications

  • Klamroth R, Windyga J, Radulescu V, Collins PW, Stasyshyn O, Ibrahim HM, Engl W, Tangada SD, Savage W, Ewenstein B. Rurioctocog alfa pegol PK-guided prophylaxis in hemophilia A: results from the phase 3 PROPEL study. Blood. 2021 Apr 1;137(13):1818-1827. doi: 10.1182/blood.2020005673. PubMed 33150384 ↗

Study documents

  • Study protocol · Jan 13, 2015
  • Study protocol · Mar 20, 2015
  • Study protocol · May 12, 2015
  • Study protocol · Sep 4, 2015
  • Study protocol · Oct 18, 2016
  • Statistical analysis plan · Sep 19, 2018

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: Yes — Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.

Supporting information: Study protocol, Sap, Icf, Csr

10

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on May 25, 2021, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT02585960
Lead sponsor
Baxalta now part of Shire
Collaborators
Baxalta Innovations GmbH, now part of Shire
Responsible party
Sponsor
First posted
Oct 26, 2015
Start date
Nov 23, 2015
Primary completion
Aug 5, 2018
Completion
Aug 5, 2018
Results posted
Aug 26, 2019
Last update
May 25, 2021

Study contacts

Study Director
study director · Takeda

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

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