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BAX 326 (rFIX) Continuation Study

Primary Purpose

Hemophilia B

Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
BAX 326 (Recombinant factor IX)
Sponsored by
Baxalta now part of Shire
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia B

Eligibility Criteria

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

Main Inclusion Criteria:

  • Subject and/or legal representative has/have voluntarily provided signed informed consent
  • Subject has completed Baxter clinical study 250901 (pivotal study) or Baxter clinical study 251101 (pediatric study)
  • Subject was 12 to 65 years old at the time of screening for Study 250901 or < 12 years old at the time of screening for Study 251101
  • Subject has severe (FIX level < 1%) or moderately severe (FIX level 1-2%) hemophilia B (based on the one stage activated partial thromboplastin time (aPTT) assay), as tested at screening at the central laboratory
  • Subject has not developed an inhibitory FIX antibody during Baxter Pivotal Study 250901 or Pediatric Study 251101

Main Exclusion Criteria:

  • Subject received factor IX product(s) other than BAX 326 upon completion of Baxter Pivotal Study 250901 or Pediatric Study 251101
  • Subject has been diagnosed with an acquired hemostatic defect other than hemophilia B
  • For subjects transferring from Pivotal Study 250901: Subject's weight is < 35 kg or > 120 kg
  • Subject is planned to take part in any other clinical study, with the exception of BAX 326 Surgery study as described in this protocol, during the course of the Continuation Study

Sites / Locations

  • Instituto de Hematología y Medicina Clíncia Rubén Dávoli
  • UNIFESP - Universidade Estadual de Sao Paulo
  • Specialized Haematological Hospital "Joan Pavel"
  • Hospital Dr. Sotero del Rio
  • Hospital de San Jose
  • Centro Medico Imbanaco
  • Hospital Pablo Tobon Uribe
  • Klinika detské hematologie a onkologie
  • Maulana Azad Medical College and Associated Hospital
  • St. James's Hospital, National Center for Hereditary Coagulation Disorders
  • University Hospital Policlinico Vittorio Emanuele, Hospital Ferrarotto Alessi
  • University Hospital Careggi, Agency of Hemophilia - Regional Reference Center for Inherited Bleeding
  • University of Foggia Riuniti Hospital, Department of Clinical and Experimental Medicine
  • Hospital San Giovanni Bosco, Center for Hemophilia and Thrombosis, Department of Hematology
  • Padova University Hospital, Medical Clinic II, Center for Hemophilia
  • Nara Medical University, Department of Pediatrics
  • Tokyo Medical University
  • Ogikubo Hospital
  • Hematology and Transplantology Clinic, University Clinic Centre - Medical University Hospital
  • University Pediatric Hospital in Cracow
  • Medical College of the Jagiellonian University, Department of Hematology
  • Copernicus Hospital, Medical University in Lodz, Department of Hematology
  • Professor Tadeusz Sokolowski Independent Public Teaching Hospital No. 1 of the Pomeranian Medical University in Szczecin
  • Klinika Hematologii
  • Institute of Haematology and Transfusion Medicine
  • Prof. Dr. C.T. Nicolau National Institute for Transfusional Hematology
  • Louis Turcanu Emergency Clinical Children´s Hospital
  • Regional clinical hospital
  • Federal State Institution Kirov Hematology and Blood Transfusion Research Institute under the Federal Agency for High-Tech Medical Care
  • Pediatric Regional Clinical Hospital, Hematology Department
  • Hematology Research Center RAMS, Department of Reconstructive Orthopedics for Haemophilia Patients
  • Republican Center for Hemophilia Treatment Outpatient Clinic No. 37
  • Malmö University Hospital, Department of Coagulation Disorders
  • Taipei Medical University Hospital
  • Chung-Ho Memorial Hospital
  • China Medical University Hospital
  • Taichung Veterans General Hospital
  • National Taiwan University Hospital
  • Tri-Service General Hospital
  • State Institution "Institute of Blood Pathology and Transfusion Medicine of the Academy of Medical Sciences of Ukraine"
  • Katharine Dormandy Haemophilia Centre and Haemostasis Unit, Royal Free Hospital
  • Manchester Children's Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

BAX 326

Arm Description

Outcomes

Primary Outcome Measures

Adverse Events Possibly or Probably Related to the Investigational Product
Possibly or probably related adverse events that occurred during or after first BAX326 infusion.

Secondary Outcome Measures

Treatment of Bleeding Episodes: Number of Infusions Per Bleeding Episode Required Until Bleed Resolution
Number of Infusions of BAX326 that were required until bleed resolution.
Treatment of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Resolution of Bleed
Overall clinical efficacy rating of bleeding episodes was done at resolution of bleed according these rating scale: Excellent=Full relief of pain and cessation of objective signs of bleeding after a single infusion. No additional infusion is required for the control of bleeding. Administration of further infusion would not affect the 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.
Annualized Bleed Rate During Prophylaxis Treatment
Annualized bleed rate (ABR) was calculated as (number of bleeding episodes/observed treatment period in days)*365.25
Consumption of BAX 326: Number of Infusions Per Month and Per Year
The number of infusions consumed per month and per year for the prophylactic and on-demand treatment regimens.
Consumption of BAX 326: Weight Adjusted Consumption Per Month and Per Year
The weight adjusted consumption of BAX 326 per month and per year for the prophylactic and on-demand treatment regimens.
Consumption of BAX326: Weight Adjusted Consumption Per Bleeding Episode
The weight adjusted consumption of BAX 326 per bleeding episode for the prophylactic and on-demand treatment regimens. Only infusions required until the resolution of bleed are considered.
Development of Inhibitory and Total Binding Antibodies to Factor IX
Testing for inhibitory and total binding antibodies to Factor IX (FIX). Development during study means negative at screening and positive at any subsequent visit. Treatment emergent means more than 2-dilution increase as compared to the pre-study titer at screening.
Development of Antibodies to Chinese Hamster Ovary Proteins (CHO Proteins) and rFurin
Testing for antibodies to CHO proteins and rFurin. Development during study means negative at screening and positive at any subsequent visit. Treatment emergent means more than 2-dilution increase as compared to the pre-study titer at screening.
Occurrence of Severe Allergic Reactions and Thrombotic Events
The occurrence of severe allergic reactions and thrombotic events was assessed.
Clinical Significant Changes in Routine Laboratory Parameters and Vital Signs
Hematology panel consists of complete blood count (hemoglobin, hematocrit, erythrocytes, leukocytes) with differential (ie, basophils, eosinophils, lymphocytes, monocytes, neutrophils), mean corpuscular volume, mean corpuscular hemoglobin concentration and platelet count. Clinical chemistry panel consists of sodium, potassium, chloride, bicarbonate, total protein, albumin, alanine aminotransferase (ALT), aspartate aminotransferase (AST), total bilirubin, alkaline phosphatase, blood urea nitrogen, creatinine and glucose. Vital signs include body temperature, respiratory rate, pulse rate, supine systolic and diastolic blood pressure. CS=clinically significant, NCS=not clinically significant. Change from Screening to End of Study is reported.
Pharmacokinetics: Incremental Recovery (IR) Over Time
PK infusion with investigational product was administered after a wash out period of at least 5 days. Incremental recovery is calculated as IR30min = (C30min [IU/dL] - Cpre-infusion [IU/dL]) / dose per kg body weight [IU/kg] where C30min and Cpre-infusion relate to the unadjusted concentration values.
Pharmacokinetics: Area Under the Plasma Concentration Versus Time Curve From Time 0 to Infinity (AUC 0-∞)
After a wash out period of at least 5 days PK infusion with investigational product was administered. AUC 0-∞ is defined as AUC 0-t + Ct/lambda z, where t is the time of last quantifiable concentration, Ct is the last quantifiable concentration.
Pharmacokinetics: Elimination Phase Half-life (T1/2)
PK infusion with investigational product was administered after a wash out period of at least 5 days. Elimination phase half-life is calculated as T1/2=log e (2) / lambda z where the elimination rate constant (lambda z) will be obtained by log e - linear fitting using least squares deviation to at least the last 3 quantifiable concentrations above pre-infusion level.
Pharmacokinetics: Mean Residence Time (MRT)
PK infusion with investigational product was administered after a wash out period of at least 5 days. Mean residence time is calculated as total area under the moment curve divided by the total area under the curve. MRT=(AUMC0-∞[h2*IU/dL])/(AUC0-∞[h*IU/dL]) - TI/2 where AUMC0-∞ is determined in a similar manner as AUC0-∞ and TI represents infusion duration in hours.
Pharmacokinetics: Systemic Clearance (CL)
PK infusion with investigational product was administered after a wash out period of at least 5 days. Systemic clearance is balculated as the dose in IU/kg divided by the total AUC. CL= Dose[IU/kg] / AUC0-∞[h*IU/dL]
Pharmacokinetics: Volume of Distribution at Steady State (Vss)
PK infusion with investigational product was administered after a wash out period of at least 5 days. Apparent steady state volume of distribution is calculated as Vss = CL * MRT CL=Systemic Clearance and MRT=Mean residence time
Pharmacokinetics: Incremental Recovery (IR)
PK infusion with investigational product was administered after a wash out period of at least 5 days. Incremental recovery is calculated as IR30min = (C30min [IU/dL] - Cpre-infusion [IU/dL]) / dose per kg body weight [IU/kg] where C30min and Cpre-infusion relate to the unadjusted concentration values.
Changes in Health Related Quality of Life (HR QoL) Based on Questionnaire SF-36
The Short Form (36) Health Survey (SF-36) is a 36-item validated, generic HR QoL instrument suitable for participants of 17 years of age or older. The SF-36 consists of eight scaled scores (vitality, physical functioning, bodily pain, general health, mental health, physical role functioning, emotional role functioning, social role functioning) which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. The lower the score the more disability. The higher the score the less disability. The mental health component summary score ranged from 19.5 to 64.2 with higher scores indicating less disability. The physical health component summary scores ranged from 18.6 to 59.6 with higher scores indicating less disability.
Changes in Health Related Quality of Life Using the Peds QL
The Pediatric Quality of Life Inventory (Peds QL) is a generic health related quality of life instrument designed specifically for a pediatric population and captures following domains: physical functioning, emotional functioning, social functioning and school functioning. The Peds-QL total score consist of all 23 items of all domains. Score range from 0-100 and higher scores indicate better quality of life (collected scores ranged from 44.6 to 98.9). The Peds-QL Physical Health Summary score consists of 8 items from the physical functioning domain. Score range from 0-100 and higher scores indicate better quality of life (collected scores ranged from 40.6 to 100.0) The Psychosocial Health Summary score consists of 15 items from the emotional, social and school functioning domains. Score range from 0 to 100 and higher scores indicate better quality of life (collected scores ranged from 46.7 to 100.0).
Changes in Health Related Quality of Life (HR QoL) Based on Questionnaire Haemo-QoL and Haem-A-QoL
The Hemophilia Quality of Life Questionnaire (Haemo-QoL) and the Hemophilia Quality of Life Questionnaire for Adults (Haem-A-Qol) instruments have been developed and used in hemophilia A patients. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. The areas covered by this instrument are: physical health, sports/leisure, school/work, dealing with hemophilia, and outlook for the future. Haemo-QoL is used for participants aged 8 to 16 years and total scores range from 0 to 100 with higher scores indicating low quality of life (collected scores ranged from 0.0 to 44.3) Haem-A-QoL is used for participants aged 17 years and older and total scores range from 0 to 100 with higher scores indicating low quality of life (collected scores ranged from 4.9 to 76.8).
Changes in Health Related Quality of Life (HR QoL) Based on Questionnaire EQ-5D and Pain Score.
The EQ-5D captures overall HR QoL (phyiscal, mental and social functioning). A health utility score can be calculated from this measure, adult and proxy versions available. EQ-5D Visual Analog Scale (EQ-5D VAS):Respondents specify their level of agreement to a statement by indicating a position along a continuous line between two endpoints (scale range from 0 to 100). Score 0 corresponds to the worst health you can imagine and score 100 corresponds to the best health you can imagine (collected scores ranged from 10-100). EQ-5D Total Index is based on general population valuation surveys. Responses to 5 questions are converted to an Index value and score range from 0 to 1, with higher scores indicating better quality of life. Total Index was derived on US population (collected scores ranged from 0.4-1). General pain assessment (Pain score) is done through a visual analog scale (VAS), scores ranging from 0 to 100 with higher scores indicating more pain (collected scores ranged 0-87).

Full Information

First Posted
January 26, 2011
Last Updated
April 30, 2021
Sponsor
Baxalta now part of Shire
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1. Study Identification

Unique Protocol Identification Number
NCT01286779
Brief Title
BAX 326 (rFIX) Continuation Study
Official Title
BAX 326 (Recombinant Factor IX): Evaluation of Safety, Immunogenicity, and Hemostatic Efficacy in Previously Treated Patients With Severe (FIX Level < 1%) or Moderately Severe (FIX Level <= 2%) Hemophilia B - A Continuation Study
Study Type
Interventional

2. Study Status

Record Verification Date
April 2021
Overall Recruitment Status
Completed
Study Start Date
April 12, 2011 (Actual)
Primary Completion Date
June 29, 2017 (Actual)
Study Completion Date
June 29, 2017 (Actual)

3. Sponsor/Collaborators

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

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
The purpose of this BAX 326 Continuation Study is to further investigate incremental recovery over time, the hemostatic efficacy, the safety, immunogenicity, and health-related quality of life (HR QoL) of BAX 326 in previously treated patients (PTPs) with severe and moderately severe hemophilia B who participated in BAX 326 pivotal study 250901 or BAX 326 pediatric study 251101.

6. Conditions and Keywords

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

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
117 (Actual)

8. Arms, Groups, and Interventions

Arm Title
BAX 326
Arm Type
Experimental
Intervention Type
Biological
Intervention Name(s)
BAX 326 (Recombinant factor IX)
Intervention Description
The treatment with BAX 326 will be at the discretion of the investigator and will consist of either twice weekly prophylactic treatment with 50 IU/kg, modified prophylaxis, or on-demand treatment.
Primary Outcome Measure Information:
Title
Adverse Events Possibly or Probably Related to the Investigational Product
Description
Possibly or probably related adverse events that occurred during or after first BAX326 infusion.
Time Frame
Assessed (based on patient diary) every 3 months until study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).
Secondary Outcome Measure Information:
Title
Treatment of Bleeding Episodes: Number of Infusions Per Bleeding Episode Required Until Bleed Resolution
Description
Number of Infusions of BAX326 that were required until bleed resolution.
Time Frame
Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).
Title
Treatment of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Resolution of Bleed
Description
Overall clinical efficacy rating of bleeding episodes was done at resolution of bleed according these rating scale: Excellent=Full relief of pain and cessation of objective signs of bleeding after a single infusion. No additional infusion is required for the control of bleeding. Administration of further infusion would not affect the scoring. Good=Definite pain relief and/or improvement in signs of bleeding after a single infusion. Possibly requires more than 1 infusion for complete resolution. Fair=Probable and/or slight relief of pain and slight improvement in signs of bleeding after a single infusion. Required more than 1 infusion for complete resolution. None=No improvement or condition worsens.
Time Frame
Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).
Title
Annualized Bleed Rate During Prophylaxis Treatment
Description
Annualized bleed rate (ABR) was calculated as (number of bleeding episodes/observed treatment period in days)*365.25
Time Frame
For prophylactic treatment the period from first to last prophylactic infusion is considered.
Title
Consumption of BAX 326: Number of Infusions Per Month and Per Year
Description
The number of infusions consumed per month and per year for the prophylactic and on-demand treatment regimens.
Time Frame
Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).
Title
Consumption of BAX 326: Weight Adjusted Consumption Per Month and Per Year
Description
The weight adjusted consumption of BAX 326 per month and per year for the prophylactic and on-demand treatment regimens.
Time Frame
Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).
Title
Consumption of BAX326: Weight Adjusted Consumption Per Bleeding Episode
Description
The weight adjusted consumption of BAX 326 per bleeding episode for the prophylactic and on-demand treatment regimens. Only infusions required until the resolution of bleed are considered.
Time Frame
Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).
Title
Development of Inhibitory and Total Binding Antibodies to Factor IX
Description
Testing for inhibitory and total binding antibodies to Factor IX (FIX). Development during study means negative at screening and positive at any subsequent visit. Treatment emergent means more than 2-dilution increase as compared to the pre-study titer at screening.
Time Frame
Laboratory assessment for immunology were done at screening, at exposure day 1, at week 4 (± 1 week), at month 3 (±1 week), thereafter, every 3 months (± 1 week) and at study completion/termination.
Title
Development of Antibodies to Chinese Hamster Ovary Proteins (CHO Proteins) and rFurin
Description
Testing for antibodies to CHO proteins and rFurin. Development during study means negative at screening and positive at any subsequent visit. Treatment emergent means more than 2-dilution increase as compared to the pre-study titer at screening.
Time Frame
Laboratory assessment for immunology were done at screening, at exposure day 1, at week 4 (± 1 week), at month 3 (±1 week), thereafter, every 3 months (± 1 week) and at study completion/termination.
Title
Occurrence of Severe Allergic Reactions and Thrombotic Events
Description
The occurrence of severe allergic reactions and thrombotic events was assessed.
Time Frame
Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last).
Title
Clinical Significant Changes in Routine Laboratory Parameters and Vital Signs
Description
Hematology panel consists of complete blood count (hemoglobin, hematocrit, erythrocytes, leukocytes) with differential (ie, basophils, eosinophils, lymphocytes, monocytes, neutrophils), mean corpuscular volume, mean corpuscular hemoglobin concentration and platelet count. Clinical chemistry panel consists of sodium, potassium, chloride, bicarbonate, total protein, albumin, alanine aminotransferase (ALT), aspartate aminotransferase (AST), total bilirubin, alkaline phosphatase, blood urea nitrogen, creatinine and glucose. Vital signs include body temperature, respiratory rate, pulse rate, supine systolic and diastolic blood pressure. CS=clinically significant, NCS=not clinically significant. Change from Screening to End of Study is reported.
Time Frame
Measurements at screening and at study completion/termination are included in the analysis.
Title
Pharmacokinetics: Incremental Recovery (IR) Over Time
Description
PK infusion with investigational product was administered after a wash out period of at least 5 days. Incremental recovery is calculated as IR30min = (C30min [IU/dL] - Cpre-infusion [IU/dL]) / dose per kg body weight [IU/kg] where C30min and Cpre-infusion relate to the unadjusted concentration values.
Time Frame
IR over time was measured as Baseline and at Completion/Termination visit within 30 minutes pre-infusion and at 30 (± 5) minutes post-infusion.
Title
Pharmacokinetics: Area Under the Plasma Concentration Versus Time Curve From Time 0 to Infinity (AUC 0-∞)
Description
After a wash out period of at least 5 days PK infusion with investigational product was administered. AUC 0-∞ is defined as AUC 0-t + Ct/lambda z, where t is the time of last quantifiable concentration, Ct is the last quantifiable concentration.
Time Frame
PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours
Title
Pharmacokinetics: Elimination Phase Half-life (T1/2)
Description
PK infusion with investigational product was administered after a wash out period of at least 5 days. Elimination phase half-life is calculated as T1/2=log e (2) / lambda z where the elimination rate constant (lambda z) will be obtained by log e - linear fitting using least squares deviation to at least the last 3 quantifiable concentrations above pre-infusion level.
Time Frame
PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours
Title
Pharmacokinetics: Mean Residence Time (MRT)
Description
PK infusion with investigational product was administered after a wash out period of at least 5 days. Mean residence time is calculated as total area under the moment curve divided by the total area under the curve. MRT=(AUMC0-∞[h2*IU/dL])/(AUC0-∞[h*IU/dL]) - TI/2 where AUMC0-∞ is determined in a similar manner as AUC0-∞ and TI represents infusion duration in hours.
Time Frame
PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours
Title
Pharmacokinetics: Systemic Clearance (CL)
Description
PK infusion with investigational product was administered after a wash out period of at least 5 days. Systemic clearance is balculated as the dose in IU/kg divided by the total AUC. CL= Dose[IU/kg] / AUC0-∞[h*IU/dL]
Time Frame
PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours
Title
Pharmacokinetics: Volume of Distribution at Steady State (Vss)
Description
PK infusion with investigational product was administered after a wash out period of at least 5 days. Apparent steady state volume of distribution is calculated as Vss = CL * MRT CL=Systemic Clearance and MRT=Mean residence time
Time Frame
PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours
Title
Pharmacokinetics: Incremental Recovery (IR)
Description
PK infusion with investigational product was administered after a wash out period of at least 5 days. Incremental recovery is calculated as IR30min = (C30min [IU/dL] - Cpre-infusion [IU/dL]) / dose per kg body weight [IU/kg] where C30min and Cpre-infusion relate to the unadjusted concentration values.
Time Frame
PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours.
Title
Changes in Health Related Quality of Life (HR QoL) Based on Questionnaire SF-36
Description
The Short Form (36) Health Survey (SF-36) is a 36-item validated, generic HR QoL instrument suitable for participants of 17 years of age or older. The SF-36 consists of eight scaled scores (vitality, physical functioning, bodily pain, general health, mental health, physical role functioning, emotional role functioning, social role functioning) which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. The lower the score the more disability. The higher the score the less disability. The mental health component summary score ranged from 19.5 to 64.2 with higher scores indicating less disability. The physical health component summary scores ranged from 18.6 to 59.6 with higher scores indicating less disability.
Time Frame
Baseline at exposure day 1 and at study completion/termination.
Title
Changes in Health Related Quality of Life Using the Peds QL
Description
The Pediatric Quality of Life Inventory (Peds QL) is a generic health related quality of life instrument designed specifically for a pediatric population and captures following domains: physical functioning, emotional functioning, social functioning and school functioning. The Peds-QL total score consist of all 23 items of all domains. Score range from 0-100 and higher scores indicate better quality of life (collected scores ranged from 44.6 to 98.9). The Peds-QL Physical Health Summary score consists of 8 items from the physical functioning domain. Score range from 0-100 and higher scores indicate better quality of life (collected scores ranged from 40.6 to 100.0) The Psychosocial Health Summary score consists of 15 items from the emotional, social and school functioning domains. Score range from 0 to 100 and higher scores indicate better quality of life (collected scores ranged from 46.7 to 100.0).
Time Frame
Baseline at exposure day 1 and at study completion/termination.
Title
Changes in Health Related Quality of Life (HR QoL) Based on Questionnaire Haemo-QoL and Haem-A-QoL
Description
The Hemophilia Quality of Life Questionnaire (Haemo-QoL) and the Hemophilia Quality of Life Questionnaire for Adults (Haem-A-Qol) instruments have been developed and used in hemophilia A patients. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. The areas covered by this instrument are: physical health, sports/leisure, school/work, dealing with hemophilia, and outlook for the future. Haemo-QoL is used for participants aged 8 to 16 years and total scores range from 0 to 100 with higher scores indicating low quality of life (collected scores ranged from 0.0 to 44.3) Haem-A-QoL is used for participants aged 17 years and older and total scores range from 0 to 100 with higher scores indicating low quality of life (collected scores ranged from 4.9 to 76.8).
Time Frame
Baseline at exposure day 1 and at study completion/termination.
Title
Changes in Health Related Quality of Life (HR QoL) Based on Questionnaire EQ-5D and Pain Score.
Description
The EQ-5D captures overall HR QoL (phyiscal, mental and social functioning). A health utility score can be calculated from this measure, adult and proxy versions available. EQ-5D Visual Analog Scale (EQ-5D VAS):Respondents specify their level of agreement to a statement by indicating a position along a continuous line between two endpoints (scale range from 0 to 100). Score 0 corresponds to the worst health you can imagine and score 100 corresponds to the best health you can imagine (collected scores ranged from 10-100). EQ-5D Total Index is based on general population valuation surveys. Responses to 5 questions are converted to an Index value and score range from 0 to 1, with higher scores indicating better quality of life. Total Index was derived on US population (collected scores ranged from 0.4-1). General pain assessment (Pain score) is done through a visual analog scale (VAS), scores ranging from 0 to 100 with higher scores indicating more pain (collected scores ranged 0-87).
Time Frame
Baseline at exposure day 1 and at study completion/termination.

10. Eligibility

Sex
All
Maximum Age & Unit of Time
65 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Main Inclusion Criteria: Subject and/or legal representative has/have voluntarily provided signed informed consent Subject has completed Baxter clinical study 250901 (pivotal study) or Baxter clinical study 251101 (pediatric study) Subject was 12 to 65 years old at the time of screening for Study 250901 or < 12 years old at the time of screening for Study 251101 Subject has severe (FIX level < 1%) or moderately severe (FIX level 1-2%) hemophilia B (based on the one stage activated partial thromboplastin time (aPTT) assay), as tested at screening at the central laboratory Subject has not developed an inhibitory FIX antibody during Baxter Pivotal Study 250901 or Pediatric Study 251101 Main Exclusion Criteria: Subject received factor IX product(s) other than BAX 326 upon completion of Baxter Pivotal Study 250901 or Pediatric Study 251101 Subject has been diagnosed with an acquired hemostatic defect other than hemophilia B For subjects transferring from Pivotal Study 250901: Subject's weight is < 35 kg or > 120 kg Subject is planned to take part in any other clinical study, with the exception of BAX 326 Surgery study as described in this protocol, during the course of the Continuation 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
Instituto de Hematología y Medicina Clíncia Rubén Dávoli
City
Rosario
ZIP/Postal Code
2000
Country
Argentina
Facility Name
UNIFESP - Universidade Estadual de Sao Paulo
City
Sao Paulo
ZIP/Postal Code
040024-002
Country
Brazil
Facility Name
Specialized Haematological Hospital "Joan Pavel"
City
Sofia
ZIP/Postal Code
1233
Country
Bulgaria
Facility Name
Hospital Dr. Sotero del Rio
City
Santiago
Country
Chile
Facility Name
Hospital de San Jose
City
Bogotá
Country
Colombia
Facility Name
Centro Medico Imbanaco
City
Cali
Country
Colombia
Facility Name
Hospital Pablo Tobon Uribe
City
Medellin
ZIP/Postal Code
005543
Country
Colombia
Facility Name
Klinika detské hematologie a onkologie
City
Prague
ZIP/Postal Code
150 06
Country
Czechia
Facility Name
Maulana Azad Medical College and Associated Hospital
City
New Delhi
ZIP/Postal Code
110002
Country
India
Facility Name
St. James's Hospital, National Center for Hereditary Coagulation Disorders
City
Dublin
ZIP/Postal Code
8
Country
Ireland
Facility Name
University Hospital Policlinico Vittorio Emanuele, Hospital Ferrarotto Alessi
City
Catania
ZIP/Postal Code
95124
Country
Italy
Facility Name
University Hospital Careggi, Agency of Hemophilia - Regional Reference Center for Inherited Bleeding
City
Florence
ZIP/Postal Code
50134
Country
Italy
Facility Name
University of Foggia Riuniti Hospital, Department of Clinical and Experimental Medicine
City
Foggia
ZIP/Postal Code
71100
Country
Italy
Facility Name
Hospital San Giovanni Bosco, Center for Hemophilia and Thrombosis, Department of Hematology
City
Naples
ZIP/Postal Code
80144
Country
Italy
Facility Name
Padova University Hospital, Medical Clinic II, Center for Hemophilia
City
Padova
ZIP/Postal Code
35128
Country
Italy
Facility Name
Nara Medical University, Department of Pediatrics
City
Nara
ZIP/Postal Code
634-8251
Country
Japan
Facility Name
Tokyo Medical University
City
Tokyo
ZIP/Postal Code
160-0023
Country
Japan
Facility Name
Ogikubo Hospital
City
Tokyo
ZIP/Postal Code
167-0035
Country
Japan
Facility Name
Hematology and Transplantology Clinic, University Clinic Centre - Medical University Hospital
City
Gdansk
ZIP/Postal Code
80-952
Country
Poland
Facility Name
University Pediatric Hospital in Cracow
City
Krakow
ZIP/Postal Code
30-663
Country
Poland
Facility Name
Medical College of the Jagiellonian University, Department of Hematology
City
Krakow
ZIP/Postal Code
31-501
Country
Poland
Facility Name
Copernicus Hospital, Medical University in Lodz, Department of Hematology
City
Lodz
ZIP/Postal Code
93-510
Country
Poland
Facility Name
Professor Tadeusz Sokolowski Independent Public Teaching Hospital No. 1 of the Pomeranian Medical University in Szczecin
City
Szczecin
ZIP/Postal Code
71-252
Country
Poland
Facility Name
Klinika Hematologii
City
Warsaw
ZIP/Postal Code
00-579
Country
Poland
Facility Name
Institute of Haematology and Transfusion Medicine
City
Warsaw
ZIP/Postal Code
02-776
Country
Poland
Facility Name
Prof. Dr. C.T. Nicolau National Institute for Transfusional Hematology
City
Bucharest
ZIP/Postal Code
11156
Country
Romania
Facility Name
Louis Turcanu Emergency Clinical Children´s Hospital
City
Timisoara
Country
Romania
Facility Name
Regional clinical hospital
City
Ekaterinburg
ZIP/Postal Code
620149
Country
Russian Federation
Facility Name
Federal State Institution Kirov Hematology and Blood Transfusion Research Institute under the Federal Agency for High-Tech Medical Care
City
Kirov
ZIP/Postal Code
610027
Country
Russian Federation
Facility Name
Pediatric Regional Clinical Hospital, Hematology Department
City
Krasnodar
ZIP/Postal Code
350007
Country
Russian Federation
Facility Name
Hematology Research Center RAMS, Department of Reconstructive Orthopedics for Haemophilia Patients
City
Moscow
ZIP/Postal Code
125167
Country
Russian Federation
Facility Name
Republican Center for Hemophilia Treatment Outpatient Clinic No. 37
City
St. Petersburg
ZIP/Postal Code
195213
Country
Russian Federation
Facility Name
Malmö University Hospital, Department of Coagulation Disorders
City
Malmö
ZIP/Postal Code
205 02
Country
Sweden
Facility Name
Taipei Medical University Hospital
City
Taipei City
State/Province
Taipei
ZIP/Postal Code
110
Country
Taiwan
Facility Name
Chung-Ho Memorial Hospital
City
Kaohsiung
ZIP/Postal Code
807
Country
Taiwan
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
National Taiwan University Hospital
City
Taipei
ZIP/Postal Code
10002
Country
Taiwan
Facility Name
Tri-Service General Hospital
City
Taipei
ZIP/Postal Code
114
Country
Taiwan
Facility Name
State Institution "Institute of Blood Pathology and Transfusion Medicine of the Academy of Medical Sciences of Ukraine"
City
Lviv
ZIP/Postal Code
79044
Country
Ukraine
Facility Name
Katharine Dormandy Haemophilia Centre and Haemostasis Unit, Royal Free Hospital
City
London
ZIP/Postal Code
NW3 2QG
Country
United Kingdom
Facility Name
Manchester Children's Hospital
City
Manchester
ZIP/Postal Code
M13 9WL
Country
United Kingdom

12. IPD Sharing Statement

Citations:
PubMed Identifier
32866032
Citation
Windyga J, Stasyshyn O, Lissitchkov T, Mamonov V, Serban M, Rusen L, Ploder B, Tangada S. Safety, Immunogenicity, and Hemostatic Efficacy of Nonacog Gamma in Patients With Severe or Moderately Severe Hemophilia B: A Continuation Study. Clin Appl Thromb Hemost. 2020 Jan-Dec;26:1076029620950836. doi: 10.1177/1076029620950836.
Results Reference
derived

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BAX 326 (rFIX) Continuation Study

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