search
Back to results

BAX 855 PK-guided Dosing (PROPEL)

Primary Purpose

Hemophilia A

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
PEGylated Recombinant Factor VIII
PEGylated Recombinant Factor VIII
PEGylated Recombinant Factor VIII
Sponsored by
Baxalta now part of Shire
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional prevention trial for Hemophilia A

Eligibility Criteria

12 Years - 65 Years (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

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:

  • 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.

Sites / Locations

  • Phoenix Childrens Hospital
  • Arizona Hemophilia & Thrombosis Center, located within The University of Arizona Cancer Center
  • Children's Hospital Los Angeles
  • University of Colorado
  • University of Florida College of Medicine
  • Emory University-ECC
  • University of Kentucky Medical Center
  • University of Louisville KCPCRU
  • Tulane University
  • Boston Children's Hospital
  • University of Nebraska Medical Center
  • Gulf States Hemophilia Centre
  • University of Utah
  • University of Washington
  • Royal Brisbane Women's Hospital
  • The Perth Blood Institute
  • AKH - Medizinische Universität Wien
  • UMHAT "Sv. Georgi", EAD
  • UMHAT 'Tsaritsa Yoanna - ISUL', EAD
  • MHAT 'Sv. Marina', EAD, Clinic of Clinical Hematology
  • CHU Nice- Service hematologie
  • Hôpital Morvan
  • CHU Rennes - Hopital Pontchaillou
  • CHU de Caen - Hôpital Côte de Nacre
  • CHU Charles Nicolle
  • HZRM Hamophilie Zentrum Rhein Main GmbH
  • Inst. f. Experimentelle Hamatologie u. Transfusionsmedizin
  • COAGULATION RESEARCH CENTRE GmbH
  • Hamophiliezentrum/Gerinnungssprechstunde
  • MVZ Labor Dr. Reising-Ackermann
  • University of Hong Kong
  • Prince of Wales Hospital
  • Magyar Honvedseg EK
  • DE OEC Belgyógyászati Int
  • PTE ÁOK
  • Chaim Sheba Medical Center
  • UOC Ematologia, Azienda ULSS 8 Asolo, Regione Veneto
  • Presidio Osped. Ferrarotto
  • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
  • Umberto I Pol. di Roma-Università di Roma La Sapienza
  • Fondazione Policlinico Universitario A. Gemelli
  • AOU Citta della Salute e della Scienza - Presidio Molinette
  • ULSS n. 6 "Vicenza"
  • Hospital Ampang
  • Hospital Queen Elizabeth
  • Hospital Kuala Lumpur
  • Hospital Melaka
  • Hospital Pulau Pinang
  • Oslo Universitetssykehus HF
  • Wojewodzki Szpital Specjalistyczny im. Mikolaja Kopernika, Klinika Hematologii
  • Alvamed
  • Instytut Hematologii Ii Transfuzjologii
  • SP Szpital Kliniczny Nr 1 we Wroclawiu
  • Spitalul Clinic Judetean de Urgenta Brasov
  • Institutul Oncologic ClNa.
  • National University Hospital
  • Singapore General Hospital- Parent
  • KK Women's And Children's Hospital
  • Hospital Universitari Son Espases
  • Complejo Hospitalario Universitario A Coruña
  • Hospital Regional Universitario de Malaga
  • Hospital Universitario La Paz
  • Sahlgrenska Universitetssjukhuset
  • Karolinska Universitetssjukhuset
  • Universitätsspital Zürich
  • China Medical University Hospital
  • Taichung Veterans General Hospital
  • Tri-Service General Hospital
  • Acibadem Hastanesi
  • Akdeniz University
  • Istanbul University
  • Ege University
  • NChSH Okhmatdyt of MoHU Center of Children Oncohematology and Bone Marrow Transplantation
  • Kyiv CCH #9 Dept of Surgery City SPC of Diagnostics & Treatment of Patients with HP
  • SI Institute of Blood Pathology and Transfusion Medicine of NAMSU
  • M.V. Sklifosovskyi Poltava RCH Dept of Gematology HSEIU Ukrainian Medical Stomatological Academy
  • Bristol Royal Hospital for Children
  • Royal Free Hospital
  • Royal Manchester Children's Hospital
  • Southampton General Hospital
  • Leicester Royal Infirmary
  • Churchill Hospital
  • University Hospital of Wales

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

Experimental

Arm Label

Pharmacokinetic (PK) evaluation of BAX 855

FVIII trough target 1-3%

FVIII trough target 8-12%

Arm Description

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

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

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

Outcomes

Primary Outcome Measures

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.

Secondary Outcome Measures

Total Annualized Bleeding Rate for Second Six Months
Annualized bleeding rate was determined by dividing the number of bleeds by observation period in years.
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.
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.
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.
Total Weight-adjusted Consumption of BAX 855
Total weight-adjusted consumption of BAX 855 were reported.
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.
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.
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.
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.
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).
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).
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.
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.
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.
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.
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.
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.
Incremental Recovery (IR) at Maximum Plasma Concentration (Cmax) of BAX 855
IR at Cmax of BAX 855 were reported.
Plasma Half-life (T1/2) of BAX 855
T1/2 of BAX 855 in plasma were reported.
Mean Residence Time (MRT) of BAX 855
MRT of BAX 855 were reported.
Maximum Plasma Concentration (Cmax) of BAX 855
Cmax of BAX 855 were reported.
Time to Maximum Concentration of BAX 855 in Plasma (Tmax)
Tmax of BAX 855 were reported.
Total Body Clearance (CL) of BAX 855
Total body clearance of BAX 855 from blood by the kidney were reported.
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.
Incremental Recovery (IR) Over Time
Incremental recovery was calculated by BAX 855 increment (IU/dL) / BAX 855 dose (IU/kg).

Full Information

First Posted
October 21, 2015
Last Updated
May 3, 2021
Sponsor
Baxalta now part of Shire
Collaborators
Baxalta Innovations GmbH, now part of Shire
search

1. Study Identification

Unique Protocol Identification Number
NCT02585960
Brief Title
BAX 855 PK-guided Dosing
Acronym
PROPEL
Official Title
Phase 3, Prospective, Randomized, Multi-center Clinical Study Comparing the Safety and Efficacy of BAX 855 Following PK-guided Prophylaxis Targeting Two Different FVIII Trough Levels in Subjects With Severe Hemophilia A
Study Type
Interventional

2. Study Status

Record Verification Date
May 2021
Overall Recruitment Status
Completed
Study Start Date
November 23, 2015 (Actual)
Primary Completion Date
August 5, 2018 (Actual)
Study Completion Date
August 5, 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Baxalta now part of Shire
Collaborators
Baxalta Innovations GmbH, now part of Shire

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
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%) To further characterize pharmacokinetic (PK) and pharmacodynamic (PD) parameters of BAX 855

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A

7. Study Design

Primary Purpose
Prevention
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
135 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Pharmacokinetic (PK) evaluation of BAX 855
Arm Type
Experimental
Arm Description
Participants will first undergo an initial pharmacokinetic (PK) assessment. Following the PK assessment participants will be randomized to one of 2 dosing regimens.
Arm Title
FVIII trough target 1-3%
Arm Type
Experimental
Arm Description
Standard treatment arm - PK-guided dosing schedule to achieve a Factor VIII (FVIII) trough of 1-3%
Arm Title
FVIII trough target 8-12%
Arm Type
Experimental
Arm Description
Intensified treatment arm - PK-guided dosing schedule to achieve a Factor VIII (FVIII) trough of 8-12%
Intervention Type
Biological
Intervention Name(s)
PEGylated Recombinant Factor VIII
Other Intervention Name(s)
BAX855, BAX 855
Intervention Description
Pharmacokinetic (PK) evaluation
Intervention Type
Biological
Intervention Name(s)
PEGylated Recombinant Factor VIII
Other Intervention Name(s)
BAX 855, BAX855
Intervention Description
Standard treatment
Intervention Type
Biological
Intervention Name(s)
PEGylated Recombinant Factor VIII
Other Intervention Name(s)
BAX855, BAX 855
Intervention Description
Intensified treatment
Primary Outcome Measure Information:
Title
Percentage of Participants With a Total Annualized Bleeding Rate (ABR) of Zero for Second Six Months
Description
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 Outcome Measure Information:
Title
Total Annualized Bleeding Rate for Second Six Months
Description
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)
Title
Annualized Spontaneous Bleeding Rate for Second Six Months
Description
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)
Title
Annualized Traumatic Bleeding Rate for Second Six Months
Description
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)
Title
Annualized Joint Bleeding Rate (AJBR) for Second Six Months
Description
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)
Title
Total Weight-adjusted Consumption of BAX 855
Description
Total weight-adjusted consumption of BAX 855 were reported.
Time Frame
From start of study treatment up to 12 months (completion or termination)
Title
Number of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Number of Infusions
Description
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
Title
Number of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Bleed Resolution
Description
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)
Title
Treatment of Bleeding Episodes: Number of BAX 855 Infusions Per Bleeding Episode Required Until Bleed Resolution
Description
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)
Title
Change From Baseline in Hemophilia Joint Health Score (HJHS)- Total Score
Description
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
Title
Number of Participants With Hemostatic Efficacy Ratings for BAX 855 Treatment of Operative Bleeds
Description
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
Title
Blood Loss Per Participant in Case of Surgery
Description
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
Title
Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs)
Description
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)
Title
Number of Participants With Clinically Significant Changes in Vital Signs Reported as Treatment Related Adverse Events
Description
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)
Title
Number of Participants With Clinically Significant Changes in Clinical Laboratory Parameters Reported as Treatment Related Adverse Events
Description
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)
Title
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
Description
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)
Title
Change From Baseline in Physical Component Scores (PCS) of the Short Form-36 (SF-36) Health Survey
Description
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)
Title
Area Under the Plasma Concentration of BAX 855 From Zero to Infinity (AUC0-inf)
Description
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
Title
Incremental Recovery (IR) at Maximum Plasma Concentration (Cmax) of BAX 855
Description
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
Title
Plasma Half-life (T1/2) of BAX 855
Description
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
Title
Mean Residence Time (MRT) of BAX 855
Description
MRT of BAX 855 were reported.
Time Frame
Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion
Title
Maximum Plasma Concentration (Cmax) of BAX 855
Description
Cmax of BAX 855 were reported.
Time Frame
Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion
Title
Time to Maximum Concentration of BAX 855 in Plasma (Tmax)
Description
Tmax of BAX 855 were reported.
Time Frame
Pre-infusion, 15 - 30 minutes, 3, 8, 24, 48, 72 and 96 hours post-infusion
Title
Total Body Clearance (CL) of BAX 855
Description
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
Title
Volume of Distribution at Steady State (Vss)
Description
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
Title
Incremental Recovery (IR) Over Time
Description
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)

10. Eligibility

Sex
All
Minimum Age & Unit of Time
12 Years
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
INCLUSION CRITERIA: Participants transitioning from another BAX 855 study who meet ALL of the following criteria are eligible for this study: Participant has completed the end of study visit of a BAX 855 study or is transitioning from the ongoing Baxalta Continuation Study 261302. 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. Participant is human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm^3, as confirmed by central laboratory. 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: 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 Participant has been previously treated with plasma-derived FVIII concentrates or recombinant FVIII for ≥ 150 documented exposure days (EDs) 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. Participant has a Karnofsky performance score of ≥ 60 at screening Participant is HIV-; or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm^3, as confirmed by central laboratory at screening 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 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 Participant is willing and able to comply with the requirements of the protocol. EXCLUSION CRITERIA: Participants transitioning from another BAX 855 study who meet ANY of the following criteria are not eligible for this study: 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. Participant has been diagnosed with an acquired hemostatic defect other than hemophilia A. The participant's weight is < 35 kg or > 100 kg. Participant's platelet count is < 100,000/mL. Participant has an abnormal renal function (serum creatinine > 1.5 times the upper limit of normal). Participant has active hepatic disease with alanine aminotransferase (ALT) and/or aspartate aminotransferase (AST) levels ≥ 5 times the upper limit of normal. 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. 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. Participant is planning to take part in any other clinical study during the course of the study. 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: Participant has detectable FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening. 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. Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease). The participant's weight is < 35 kg or > 100 kg. Participant's platelet count is < 100,000/mL. Participant has known hypersensitivity towards mouse or hamster proteins, PEG or Tween 80. 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]. Participant has severe renal impairment (serum creatinine > 1.5 times the upper limit of normal). 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. 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. 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. 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. 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Study Director
Organizational Affiliation
Takeda
Official's Role
Study Director
Facility Information:
Facility Name
Phoenix Childrens Hospital
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85016-7710
Country
United States
Facility Name
Arizona Hemophilia & Thrombosis Center, located within The University of Arizona Cancer Center
City
Tucson
State/Province
Arizona
ZIP/Postal Code
85724
Country
United States
Facility Name
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
University of Colorado
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
University of Florida College of Medicine
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32610
Country
United States
Facility Name
Emory University-ECC
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
University of Kentucky Medical Center
City
Lexington
State/Province
Kentucky
ZIP/Postal Code
40504
Country
United States
Facility Name
University of Louisville KCPCRU
City
Louisville
State/Province
Kentucky
ZIP/Postal Code
40202
Country
United States
Facility Name
Tulane University
City
New Orleans
State/Province
Louisiana
ZIP/Postal Code
70112
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
University of Nebraska Medical Center
City
Omaha
State/Province
Nebraska
ZIP/Postal Code
68198-5456
Country
United States
Facility Name
Gulf States Hemophilia Centre
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
University of Utah
City
Salt Lake City
State/Province
Utah
ZIP/Postal Code
84132
Country
United States
Facility Name
University of Washington
City
Seattle
State/Province
Washington
ZIP/Postal Code
98104
Country
United States
Facility Name
Royal Brisbane Women's Hospital
City
Herston
State/Province
Queensland
ZIP/Postal Code
4006
Country
Australia
Facility Name
The Perth Blood Institute
City
Nedlands
State/Province
Western Australia
ZIP/Postal Code
6009
Country
Australia
Facility Name
AKH - Medizinische Universität Wien
City
Vienna
ZIP/Postal Code
1090
Country
Austria
Facility Name
UMHAT "Sv. Georgi", EAD
City
Plovdiv
ZIP/Postal Code
4002
Country
Bulgaria
Facility Name
UMHAT 'Tsaritsa Yoanna - ISUL', EAD
City
Sofia
ZIP/Postal Code
1527
Country
Bulgaria
Facility Name
MHAT 'Sv. Marina', EAD, Clinic of Clinical Hematology
City
Varna
ZIP/Postal Code
9010
Country
Bulgaria
Facility Name
CHU Nice- Service hematologie
City
Nice
State/Province
Alpes Maritimes
ZIP/Postal Code
06200
Country
France
Facility Name
Hôpital Morvan
City
Brest Cedex
State/Province
Finistere
ZIP/Postal Code
29609
Country
France
Facility Name
CHU Rennes - Hopital Pontchaillou
City
Rennes cedex 09
State/Province
Ille Et Vilaine
ZIP/Postal Code
35033
Country
France
Facility Name
CHU de Caen - Hôpital Côte de Nacre
City
Caen
ZIP/Postal Code
14003
Country
France
Facility Name
CHU Charles Nicolle
City
Rouen
ZIP/Postal Code
76031
Country
France
Facility Name
HZRM Hamophilie Zentrum Rhein Main GmbH
City
Mörfelden-Walldorf
State/Province
Hessen
ZIP/Postal Code
65446
Country
Germany
Facility Name
Inst. f. Experimentelle Hamatologie u. Transfusionsmedizin
City
Bonn
State/Province
Nordrhein Westfalen
ZIP/Postal Code
53127
Country
Germany
Facility Name
COAGULATION RESEARCH CENTRE GmbH
City
Duisburg
State/Province
Nordrhein Westfalen
ZIP/Postal Code
47051
Country
Germany
Facility Name
Hamophiliezentrum/Gerinnungssprechstunde
City
Berlin
ZIP/Postal Code
10249
Country
Germany
Facility Name
MVZ Labor Dr. Reising-Ackermann
City
Leipzig
ZIP/Postal Code
04289
Country
Germany
Facility Name
University of Hong Kong
City
Hong Kong
Country
Hong Kong
Facility Name
Prince of Wales Hospital
City
Shatin
ZIP/Postal Code
00000
Country
Hong Kong
Facility Name
Magyar Honvedseg EK
City
Budapest
ZIP/Postal Code
1134
Country
Hungary
Facility Name
DE OEC Belgyógyászati Int
City
Debrecen
ZIP/Postal Code
4032
Country
Hungary
Facility Name
PTE ÁOK
City
Pecs
ZIP/Postal Code
7624
Country
Hungary
Facility Name
Chaim Sheba Medical Center
City
Tel-Hashomer
ZIP/Postal Code
5262000
Country
Israel
Facility Name
UOC Ematologia, Azienda ULSS 8 Asolo, Regione Veneto
City
Castelfranco Veneto
State/Province
Treviso
ZIP/Postal Code
31033
Country
Italy
Facility Name
Presidio Osped. Ferrarotto
City
Catania
ZIP/Postal Code
90124
Country
Italy
Facility Name
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
City
Milano
ZIP/Postal Code
20122
Country
Italy
Facility Name
Umberto I Pol. di Roma-Università di Roma La Sapienza
City
Roma
ZIP/Postal Code
00144
Country
Italy
Facility Name
Fondazione Policlinico Universitario A. Gemelli
City
Roma
ZIP/Postal Code
00168
Country
Italy
Facility Name
AOU Citta della Salute e della Scienza - Presidio Molinette
City
Torino
ZIP/Postal Code
10126
Country
Italy
Facility Name
ULSS n. 6 "Vicenza"
City
Vicenza
ZIP/Postal Code
36100
Country
Italy
Facility Name
Hospital Ampang
City
Ampang
State/Province
Kuala Lumpur
ZIP/Postal Code
68000
Country
Malaysia
Facility Name
Hospital Queen Elizabeth
City
Kota Kinabalu
State/Province
Sabah
ZIP/Postal Code
88586
Country
Malaysia
Facility Name
Hospital Kuala Lumpur
City
Kuala Lumpur
ZIP/Postal Code
50586
Country
Malaysia
Facility Name
Hospital Melaka
City
Melaka
ZIP/Postal Code
75400
Country
Malaysia
Facility Name
Hospital Pulau Pinang
City
Pulau Pinang
ZIP/Postal Code
10450
Country
Malaysia
Facility Name
Oslo Universitetssykehus HF
City
Oslo
ZIP/Postal Code
0372
Country
Norway
Facility Name
Wojewodzki Szpital Specjalistyczny im. Mikolaja Kopernika, Klinika Hematologii
City
Lodz
ZIP/Postal Code
93-510
Country
Poland
Facility Name
Alvamed
City
Poznań
ZIP/Postal Code
61-828
Country
Poland
Facility Name
Instytut Hematologii Ii Transfuzjologii
City
Warszawa
ZIP/Postal Code
02-776
Country
Poland
Facility Name
SP Szpital Kliniczny Nr 1 we Wroclawiu
City
Wroclaw
ZIP/Postal Code
50-367
Country
Poland
Facility Name
Spitalul Clinic Judetean de Urgenta Brasov
City
Brasov
ZIP/Postal Code
500365
Country
Romania
Facility Name
Institutul Oncologic ClNa.
City
Cluj Napoca
ZIP/Postal Code
400124
Country
Romania
Facility Name
National University Hospital
City
Singapore
ZIP/Postal Code
119074
Country
Singapore
Facility Name
Singapore General Hospital- Parent
City
Singapore
ZIP/Postal Code
169608
Country
Singapore
Facility Name
KK Women's And Children's Hospital
City
Singapore
ZIP/Postal Code
229899
Country
Singapore
Facility Name
Hospital Universitari Son Espases
City
Palma de Mallorca
State/Province
Baleares
ZIP/Postal Code
07010
Country
Spain
Facility Name
Complejo Hospitalario Universitario A Coruña
City
A Coruña
State/Province
La Coruña
ZIP/Postal Code
15006
Country
Spain
Facility Name
Hospital Regional Universitario de Malaga
City
Malaga
State/Province
Málaga
ZIP/Postal Code
29010
Country
Spain
Facility Name
Hospital Universitario La Paz
City
Madrid
ZIP/Postal Code
28046
Country
Spain
Facility Name
Sahlgrenska Universitetssjukhuset
City
Gothenburg
ZIP/Postal Code
S-41345
Country
Sweden
Facility Name
Karolinska Universitetssjukhuset
City
Stockholm
ZIP/Postal Code
17176
Country
Sweden
Facility Name
Universitätsspital Zürich
City
Zürich
ZIP/Postal Code
8091
Country
Switzerland
Facility Name
China Medical University Hospital
City
Taichung
ZIP/Postal Code
40447
Country
Taiwan
Facility Name
Taichung Veterans General Hospital
City
Taichung
ZIP/Postal Code
40705
Country
Taiwan
Facility Name
Tri-Service General Hospital
City
Taipei
ZIP/Postal Code
11490
Country
Taiwan
Facility Name
Acibadem Hastanesi
City
Adana
ZIP/Postal Code
01130
Country
Turkey
Facility Name
Akdeniz University
City
Antalya
ZIP/Postal Code
07070
Country
Turkey
Facility Name
Istanbul University
City
Istanbul
ZIP/Postal Code
34098
Country
Turkey
Facility Name
Ege University
City
Izmir
ZIP/Postal Code
35040
Country
Turkey
Facility Name
NChSH Okhmatdyt of MoHU Center of Children Oncohematology and Bone Marrow Transplantation
City
Kyiv
ZIP/Postal Code
1135
Country
Ukraine
Facility Name
Kyiv CCH #9 Dept of Surgery City SPC of Diagnostics & Treatment of Patients with HP
City
Kyiv
ZIP/Postal Code
4112
Country
Ukraine
Facility Name
SI Institute of Blood Pathology and Transfusion Medicine of NAMSU
City
Lviv
ZIP/Postal Code
79044
Country
Ukraine
Facility Name
M.V. Sklifosovskyi Poltava RCH Dept of Gematology HSEIU Ukrainian Medical Stomatological Academy
City
Poltava
ZIP/Postal Code
36011
Country
Ukraine
Facility Name
Bristol Royal Hospital for Children
City
Bristol
State/Province
Avon
ZIP/Postal Code
BS2 8BJ
Country
United Kingdom
Facility Name
Royal Free Hospital
City
London
State/Province
Greater London
ZIP/Postal Code
NW3 2QG
Country
United Kingdom
Facility Name
Royal Manchester Children's Hospital
City
Manchester
State/Province
Greater Manchester
ZIP/Postal Code
M13 9WL
Country
United Kingdom
Facility Name
Southampton General Hospital
City
Southampton
State/Province
Hampshire
ZIP/Postal Code
SO16 6YD
Country
United Kingdom
Facility Name
Leicester Royal Infirmary
City
Leicester
State/Province
Leicestershire
ZIP/Postal Code
LE1 5WW
Country
United Kingdom
Facility Name
Churchill Hospital
City
Oxford
State/Province
Oxfordshire
ZIP/Postal Code
OX3 7LJ
Country
United Kingdom
Facility Name
University Hospital of Wales
City
Cardiff
State/Province
West Glamorgan
ZIP/Postal Code
CF14 4XN
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
Yes
IPD Sharing Plan Description
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.
IPD Sharing Access Criteria
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
IPD Sharing URL
https://vivli.org/ourmember/takeda/
Citations:
PubMed Identifier
33150384
Citation
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.
Results Reference
derived

Learn more about this trial

BAX 855 PK-guided Dosing

We'll reach out to this number within 24 hrs