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

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

To assess efficacy and safety, including immunogenicity of BAX 855 administered as prophylaxis and as on-demand therapy in adult and adolescent (12-65 years) previously treated patients (PTPs) with severe hemophilia A To determine the pharmacokinetic (PK) parameters of BAX 855.

Study Overview

• Status: Completed
• Conditions: Hemophilia A
• Intervention / Treatment: Biological: PEGylated Recombinant Factor VIII; Biological: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method; Biological: PEGylated Recombinant Factor VIII; Biological: PEGylated Recombinant Factor VIII

• Study Type: Interventional
• Enrollment (Actual): 159
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• Australia
  • South Australia
    • Adelaide, South Australia, Australia, 5000
      • Royal Adelaide Hospital
  • Victoria
    • Clayton, Victoria, Australia, 3168
      • The Alfred Hospital
  • Western Australia
    • Fremantle, Western Australia, Australia, 6160
      • Fremantle Hospital
    • Nedlands, Western Australia, Australia, 6009
      • Hollywood Specialist Centre
• Austria
  • Linz, Austria, 4020
    • Landes-Frauen-und Kinderklinik Linz
  • Vienna, Austria, 1090
    • AKH - Medizinische Universität Wien
• Bulgaria
  • Sofia, Bulgaria, 1527
    • SHAT of Oncohaematology Diseases
• Czechia
  • Brno, Czechia, 61300
    • Fakultni nemocnice Brno
  • Olomouc, Czechia, 775 20
    • Fakultni Nemocnice Olomouc
  • Praha 5, Czechia, 150 06
    • Fakultni nemocnice v Motole
• Germany
  • Berlin, Germany, 10249
    • Vivantes Klinikum im Friedrichshain - Landsberger Allee
  • Hamburg, Germany, 20246
    • Universitaetsklinikum Hamburg-Eppendorf
  • Hannover, Germany, 30159
    • Werlhof-Institut MVZ
  • Nordrhein Westfalen
    • Duisburg, Nordrhein Westfalen, Germany, 47051
      • Gerinnungszentrum Rhein-Ruhr
• Israel
  • Haifa, Israel, 3109601
    • Rambam Health Care Campus
  • Tel Aviv, Israel, 64239
    • Chaim Sheba Medical Center
• Japan
  • Aichi-Ken
    • Nagoya-shi, Aichi-Ken, Japan, 466-8560
      • Nagoya University Hospital
  • Fukuoka-Ken
    • Kitakyushu-shi, Fukuoka-Ken, Japan, 807-8556
      • University Of Occupational And Environmental Health Hospital
  • Hiroshima-Ken
    • Hiroshima-shi, Hiroshima-Ken, Japan, 734-8551
      • Hiroshima University Hospital
  • Hyogo-Ken
    • Nishinomiya-shi, Hyogo-Ken, Japan, 663-8501
      • Hyogo College of Medicine Hospital
  • Kanagawa-Ken
    • Kawasaki-shi, Kanagawa-Ken, Japan, 216-8511
      • St. Marianna University School of Medicine Hospital
  • Nara-Ken
    • Kashihara-shi, Nara-Ken, Japan, 634-8522
      • Nara Medical University Hospital
  • Tokyo-To
    • Shinjuku-ku, Tokyo-To, Japan, 160-0023
      • Tokyo Medical University Hospital
    • Suginami-ku, Tokyo-To, Japan, 167-8515
      • Ogikubo Hospital
• Korea, Republic of
  • Busan, Korea, Republic of, 602-739
    • Pusan National University Hospital
  • Daejeon, Korea, Republic of, 302-120
    • Eulji University Hospital
  • Seoul, Korea, Republic of, 134-727
    • Kyung Hee University Hospital at Gangdong
  • Ulsan, Korea, Republic of, 682-714
    • Ulsan University Hospital
  • Jeollanam-do
    • Hwasun, Jeollanam-do, Korea, Republic of, 519-763
      • Chonnam National University Hwasun Hospital
• Lithuania
  • Vilnius, Lithuania, 08661
    • Vilnius University Hospital Santariskiu Clinics, Public Institution
  • Vilnius, Lithuania, LT-08406
    • Children's Hospital, Affiliate of Vilnius University Hospital Santariskiu Klinikos
• Malaysia
  • Kuala Lumpur, Malaysia, 50400
    • Pusat Darah Negara
  • Pulau Pinang
    • George Town, Pulau Pinang, Malaysia, 10990
      • Hospital Pulau Pinang
  • Selangor
    • Klang, Selangor, Malaysia, 41200
      • Hospital Tengku Ampuan Rahimah
• Netherlands
  • Amsterdam, Netherlands, 1105 AZ
    • Academisch Medisch Centrum
• Poland
  • Gdansk, Poland, 80-952
    • Uniwersyteckie Centrum Kliniczne
  • Lodz, Poland, 93-510
    • Wojewodzki Szpital Specjalistyczny im.M.Kopernika w Lodzi
• Romania
  • Bucuresti, Romania, 011026
    • Sanador SRL
• Spain
  • Valencia, Spain, 46026
    • Hospital Universitari i Politecnic La Fe
  • Baleares
    • Palma de Mallorca, Baleares, Spain, 07010
      • Hospital Universitari Son Espases
  • La Coruña
    • A Coruña, La Coruña, Spain, 15006
      • Complejo Hospitalario Universitario A Coruña
  • Málaga
    • Malaga, Málaga, Spain, 29010
      • Hospital Regional Universitario de Málaga
• Sweden
  • Malmö, Sweden, 20502
    • Skånes Universitetssjukhus, Malmö
  • Stockholm, Sweden, 17176
    • Karolinska Universitetssjukhuset, Solna
• Switzerland
  • Zurich, Switzerland, 8091
    • Universitätsspital Zürich
• Taiwan
  • Taipei, Taiwan, 11490
    • Tri-Service General Hospital
• Ukraine
  • Donetsk, Ukraine, 83045
    • SI V.K.Gusak Emergency and Reconstructive Surgery Institute of NAMSU Center of IT
  • Lviv, Ukraine, 79044
    • SI Institute of Blood Pathology and Transfusion Medicine of NAMSU
• United Kingdom
  • Avon
    • Bristol, Avon, United Kingdom, BS2 8BJ
      • Bristol Royal Hospital for Children
  • Greater London
    • London, Greater London, United Kingdom, NW3 2QG
      • Royal Free Hospital
    • London, Greater London, United Kingdom, SE1 7EH
      • St Thomas' Hospital
  • Greater Manchester
    • Manchester, Greater Manchester, United Kingdom, M13 9WL
      • Royal Manchester Children's Hospital
    • Manchester, Greater Manchester, United Kingdom, M13 9WL
      • Manchester Royal Infirmary
  • Leicestershire
    • Leicester, Leicestershire, United Kingdom, LE1 5WW
      • Leicester Royal Infirmary
  • Oxfordshire
    • Oxford, Oxfordshire, United Kingdom, OX3 7LJ
      • Churchill Hospital
• United States
  • Colorado
    • Aurora, Colorado, United States, 80045
      • University of Colorado
  • Florida
    • Gainesville, Florida, United States, 32610
      • University of Florida, College of Medicine
  • Georgia
    • Atlanta, Georgia, United States, 30322
      • Children's Healthcare of Atlanta
  • Illinois
    • Peoria, Illinois, United States, 61614
      • Bleeding and Clotting Disorders Institute
  • Kentucky
    • Lexington, Kentucky, United States, 40504
      • University of Kentucky Medical Center
    • Louisville, Kentucky, United States, 40202
      • University of Louisville Hospital
  • Louisiana
    • New Orleans, Louisiana, United States, 70124
      • Tulane University Medical School
  • Missouri
    • Kansas City, Missouri, United States, 66211
      • Children's Mercy Hospitals & Clinics
  • New York
    • New York, New York, United States, 10065
      • Weill Cornell Medical College-New York Presbyterian Hospital
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27599
      • University of North Carolina Chapel Hill
    • Durham, North Carolina, United States, 27710
      • Duke University Medical Center
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center
    • Cleveland, Ohio, United States, 44195
      • The Cleveland Clinic Foundation
  • Pennsylvania
    • Hershey, Pennsylvania, United States, 17033
      • Penn State Milton S. Hershey Medical Center
  • South Carolina
    • Columbia, South Carolina, United States, 29203
      • Palmetto Health
  • Utah
    • Salt Lake City, Utah, United States, 84132
      • University of Utah Health Sciences Center
  • Washington
    • Seattle, Washington, United States, 98104
      • Puget Sound Blood Group

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 12 years to 65 years (ADULT, OLDER_ADULT, CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: Male

Description

Main Inclusion Criteria:

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

Main Exclusion Criteria:

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: PREVENTION
• Allocation: NON_RANDOMIZED
• Interventional Model: PARALLEL
• Masking: NONE

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Prophylaxis	Biological: PEGylated Recombinant Factor VIII; Prophylaxis treatment; Other Names: BAX 855; Biological: Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method; Pharmacokinetic (PK) evaluation of ADVATE; Other Names: ADVATE; Biological: PEGylated Recombinant Factor VIII; Pharmacokinetic (PK) evaluation of BAX 855; Other Names: BAX 855; Biological: PEGylated Recombinant Factor VIII; On-demand treatment; Other Names: BAX 855
EXPERIMENTAL: On-demand	Biological: PEGylated Recombinant Factor VIII; Prophylaxis treatment; Other Names: BAX 855; Biological: PEGylated Recombinant Factor VIII; Pharmacokinetic (PK) evaluation of BAX 855; Other Names: BAX 855; Biological: PEGylated Recombinant Factor VIII; On-demand treatment; Other Names: BAX 855

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Annualized Bleeding Rate (ABR)	Comparisons between prophylactic and on-demand treatment were based on ABR estimates from a negative binomial regression model, taking into account the treatment regimen, target joints and age at screening, and duration of the observation period for efficacy.	9 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Rate of Success of BAX 855 for Treatment of Bleeding Episodes	Success in the control of bleeding was defined as a rating of excellent or good using the Efficacy Rating Scale for Treatment of Bleeding Episodes measured 24 hours after initiation of treatment for the bleeding episode. EXCELLENT: Full relief of pain and cessation of objective signs of bleeding (eg, swelling, tenderness, and decreased range of motion in the case of musculoskeletal hemorrhage) after a single infusion. No additional infusion is required for the control of bleeding. Administration of further infusions to maintain hemostasis would not affect this scoring. GOOD: Definite pain relief and/or improvement in signs of bleeding after a single infusion. Possibly requires more than 1 infusion for complete resolution. FAIR: Probable and/or slight relief of pain and slight improvement in signs of bleeding after a single infusion. Required more than 1 infusion for complete resolution. NONE: No improvement or condition worsens.	At least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm.
Average Number of BAX 855 Infusions Needed for the Treatment of Bleeding Episodes		From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].
Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes	Interval between Bleeds in months was calculated as: Observation period for efficacy (in days)/(number of bleeds)*(12/365.2425)	From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].
Weight-adjusted Consumption of BAX 855 - Per Prophylactic Infusion and Pharmacokinetic (PK) Infusion		Prophylactic Infusion: ≥50 exposure days or 6 months (±2 weeks), whichever occurs last. PK Infusion: PK #1 Pre-infusion within 30 minutes; Post-infusion 10 min, and 0.5, 1, 3, 6, 24, 32, 48, 56 hours (h). PK #2 also at Post-infusion 96h
Weight-adjusted Consumption of BAX 855 - Per Treatment of Bleeding Episode (BE) and Per BE for Maintenance of Hemostasis	Infusions per bleeding episode for maintenance of hemostasis only includes infusions following the resolution of a bleed to maintain hemostasis.	Treatment of Bleeding Episode (BE): Minor/Moderate BE every 12 to 24 hours until bleeding is resolved; Major BE every 8 to 12 hours until bleeding is resolved. Per BE for Maintenance of Hemostasis: within 48 hours after bleeding episode resolution.
Percentage of Participants With Adverse Events	Adverse Events (AEs) and Serious Adverse Events (SAEs)	From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].
Immunogenicity - Number of Participants With Positive Inhibitory Antibodies to FVIII, Binding Antibodies to FVIII, PEG-VIII, PEG and Anti-CHO Antibodies at Study Completion/Termination	Number of participants who received BAX855, with immunogenicity data from study completion/termination visit. FVIII = factor VIII; PEG-VIII = polyethylene glycol-factor VIII; Anti-CHO = Anti-Chinese hamster ovary	From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].
Patient Reported Outcomes: Haemo-SYM Questionnaire, Change in Score From Baseline to End of Study	The HAEMO-SYM has two subscales: pain and bleeds. HAEMO-SYM subscale scores are calculated by taking the mean of the items in each subscale and transforming them to a 0 (none or absent) to 100 (very severe) scale. Given that higher scores indicate more severe symptoms on the Haemo-SYM and that the change scores were calculated as the value at study completion minus the value at baseline, a negative change score indicates an improvement (reduction in symptoms). Conversely, a positive change score indicates worsening symptoms.	Baseline; and end of study visit [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm].
Patient Reported Outcomes - Short Form (SF)-36, Change From Baseline to End of Study	Change from Baseline to End of Study for SF-36 Questionnaire is provided. Scores for individual SF-36 categories range from 0 to 100 with higher scores representing better health. Given that higher scores indicate better health-related quality of life (HRQoL) and that the change scores were calculated as the value at study completion minus the value at baseline, a negative change score indicates a worsening of HRQoL.	Baseline; and end of study visit [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm]
Pharmacokinetics (Pk) - Plasma Half-life (One-stage Clotting Assay)	Terminal half-life calculated as log_e2/λz where λz is the terminal elimination rate constant. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).	Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).
Pharmacokinetics (Pk) - Mean Residence Time (One-stage Clotting Assay)	The mean residence time (MRT) w as calculated as total area under the moment curve divided by the total area under the curve starting from the begin of infusion (or the end of infusion if start time is not available). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).	Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).
Pharmacokinetics (Pk) - Total Body Clearance (One-stage Clotting Assay)	Clearance in dL/(kg.h) will be calculated as the dose in IU/kg divided by the total area under the curve starting from the begin of infusion (or the end of infusion if start time is not available). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).	Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).
Pharmacokinetics (Pk) - Incremental Recovery Over Time (One-stage Clotting Assay)	Incremental recovery (IR) in (IU/dL)/ (IU/kg) calculated as: IR = (Cmax- (C pre-infusion)) / (Dose/kg), where C =concentration. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).	Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).
Pharmacokinetics (Pk) - Area Under the Concentration Versus Time Curve From 0 to Infinity (AUC0-∞) (One-stage Clotting Assay)	Calculated by WinNonlin NCA (Model 201, calculation method: Linear Trapezoidal Linear/Log Interpolation). Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).	Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).
Pharmacokinetics (Pk) - Apparent Volume of Distribution at Steady State (Vss) (One-stage Clotting Assay)	The apparent volume of distribution at steady state (Vss) will be calculated as: Vss = Clearance * Mean Residence Time. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).	Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).
Pharmacokinetics (Pk) - Maximum Plasma Concentration (Cmax) (One-stage Clotting Assay)	Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).	Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).
Pharmacokinetics (Pk) -Time to Maximum Concentration in Plasma (Tmax) (One-stage Clotting Assay)	Tmax in hours will be defined as the time to reach Cmax. Participants in the pharmacokinetic full analysis set (PKFAS) analysis set received an initial infusion of ADVATE for pharmacokinetic analysis (PK-1) followed by a washout period and an infusion of BAX 855 for a second pharmacokinetic analysis (PK-2). After at least 50 EDs of BAX 855, participants in the PK subgroup received another infusion of BAX 855 for pharmacokinetic analysis (PK-3).	Within 30 minutes prior to start of infusion; and post-infusion at 10, 30 minutes, and 1, 3, 6, 9, 24, 32, 48, 56, 72 (PK2 and PK3 only), and 96 hours (PK2 and PK3 only).
Change in Vital Signs From Screening - Temperature		Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination
Change in Vital Signs From Screening - Pulse Rate		Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination
Change in Vital Signs From Screening - Respiratory Rate		Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination
Changes in Vital Signs From Screening - Blood Pressure	Systolic Blood Pressure (SBP) Diastolic Blood Pressure (DBP)	Screening, week 2, week 4, exposure day 10-15, month 3, study completion/termination
Changes in Clinical Chemistry Laboratory Assessments From Screening - Albumin and Protein		Screening, week 2, week 4, month 3, study completion/termination
Changes in Clinical Chemistry Laboratory Assessments From Screening - Alkaline Phosphatase, Alanine Aminotransferase, Aspartate Aminotransferase	Alkaline Phosphatase (Alk Phos); Alanine Aminotransferase (Ala Amino); Aspartate Aminotransferase (Asp Amino)	Screening, week 2, week 4, month 3, study completion/termination
Changes in Clinical Chemistry Laboratory Assessments From Screening - Bicarbonate, Chloride, Glucose, Potassium, Sodium, Blood Urea Nitrogen (BUN)		Screening, week 2, week 4, month 3, study completion/termination
Changes in Clinical Chemistry Laboratory Assessments From Screening - Creatinine, and Bilirubin		Screening, week 2, week 4, month 3, study completion/termination
Changes in Hematology Laboratory Assessments From Screening - Basophils, Eosinophils, Lymphocytes, Monocytes, Neutrophils, Platelets, and Leukocytes		Screening, week 2, week 4, month 3, study completion/termination
Changes in Hematology Laboratory Assessments From Screening - Hematocrit		Screening, week 2, week 4, month 3, study completion/termination
Changes in Hematology Laboratory Assessments From Screening - Hemoglobin		Screening, week 2, week 4, month 3, study completion/termination
Changes in Hematology Laboratory Assessments From Screening - Erythrocytes		Screening, week 2, week 4, month 3, study completion/termination
Changes in Lipid Panel Assessments From Screening - Cholesterol; High Density Lipoprotein (HDL); Low Density Lipoprotein (LDL); Triglycerides; and Very Low Density Lipoprotein (VLDL)		Screening, week 2, week 4, month 3, study completion/termination

Collaborators and Investigators

• Sponsor: Baxalta now part of Shire

Publications and helpful links

General Publications

• Konkle BA, Stasyshyn O, Chowdary P, Bevan DH, Mant T, Shima M, Engl W, Dyck-Jones J, Fuerlinger M, Patrone L, Ewenstein B, Abbuehl B. Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A. Blood. 2015 Aug 27;126(9):1078-85. doi: 10.1182/blood-2015-03-630897. Epub 2015 Jul 8.

Study record dates

Study Major Dates

• Study Start (ACTUAL): January 31, 2013
• Primary Completion (ACTUAL): July 17, 2014
• Study Completion (ACTUAL): July 17, 2014

Study Registration Dates

• First Submitted: November 21, 2012
• First Submitted That Met QC Criteria: November 26, 2012
• First Posted (ESTIMATE): November 29, 2012

Study Record Updates

• Last Update Posted (ACTUAL): May 20, 2021
• Last Update Submitted That Met QC Criteria: April 30, 2021
• Last Verified: April 1, 2021

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A; Coagulants; Factor VIII; BAX 855
• Other Study ID Numbers: 261201; 2012-003599-38 (EUDRACT_NUMBER)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD 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 Supporting Information Type: STUDY_PROTOCOL; SAP; ICF; CSR