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| ID | Type | Description | Link |
|---|---|---|---|
| 2011-002437-19 | EudraCT Number |
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The purpose of this study is to assess BAX 326 pharmacokinetic parameters, to evaluate its hemostatic efficacy, safety, immunogenicity, and changes in health-related quality of life in pediatric patients.
The secondary outcome measure: Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours (h) Post-infusion analysis was not done due to the different time-points for the last PK blood sample, AUC0-72 h was redundant and only total AUC was included in the PK analysis.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| BAX326 < 6 years of age | Experimental |
| |
| BAX326 6 to <12 years of age | Experimental |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| BAX326 | Biological | All participants underwent a pharmacokinetic evaluation with BAX326 (recombinant Factor IX) followed by twice weekly prophylactic treatment for 6 months or for at least 50 exposure days, whichever occurred last. |
| Measure | Description | Time Frame |
|---|---|---|
| Adverse Events (AEs) Possibly or Probably Related to BAX326 | Throughout study period (approximately 17 months) |
| Measure | Description | Time Frame |
|---|---|---|
| Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours Post-infusion Per Dose (AUC 0-72h/Dose) | Within 30 mins pre-infusion and 4 post-infusion timepoints | |
| Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to Infinity Post-infusion Per Dose (Total AUC/Dose) |
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Main Inclusion Criteria:
Main Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| LNJP Maulana Azad Medical College & Associated Hospitals | New Delhi | 110002 | India | |||
| University Pediatric Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 25495591 | Result | Urasinski T, Stasyshyn O, Andreeva T, Rusen L, Perina FG, Oh MS, Chapman M, Pavlova BG, Valenta-Singer B, Abbuehl BE. Recombinant factor IX (BAX326) in previously treated paediatric patients with haemophilia B: a prospective clinical trial. Haemophilia. 2015 Mar;21(2):196-203. doi: 10.1111/hae.12548. Epub 2014 Dec 11. |
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Enrollment was conducted at 11 clinical sites in 6 countries (United Kingdom, Poland, Romania, Russian Federation, Ukraine, India). A total of 23 participants were enrolled in the study. Of these, 11 were < 6 years of age and 12 were 6 to <12 years of age.
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| ID | Title | Description |
|---|---|---|
| FG000 | Pediatric Participants < 6 Years of Age | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. |
| FG001 | Pediatric Participants 6 to <12 Years of Age | Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Pediatric Participants < 6 Years of Age | Pediatric participants < 6 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Adverse Events (AEs) Possibly or Probably Related to BAX326 | Full analysis set | Posted | Number | AEs considered related to BAX326 | Throughout study period (approximately 17 months) |
|
Throughout study period (approximately 17 months)
AEs were monitored from the screening visit until the study completion/termination visit.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Full Analysis Set | Comprised of all participants who received at least one infusion of study product |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Device related infection | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Abdominal pain | Gastrointestinal disorders | MedDRA (Unspecified) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Shire | +1 866 842 5335 | ClinicalTransparency@shire.com |
| ID | Term |
|---|---|
| D002836 | Hemophilia B |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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|
| Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details. |
| Pharmacokinetics (PK): Mean Residence Time (MRT) | Computed as total area under the first moment curve (total AUMC) divided by the total area under the concentration versus time curve (total AUC) | Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details. |
| Pharmacokinetics (PK): Factor IX (FIX) Clearance (CL) | Computed as the dose divided by total Area under the curve (AUC) | Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details. |
| Pharmacokinetics (PK): Incremental Recovery (IR) | The rise in FIX activity in IU/dL per unit dose administered in IU/kg. Calculated as follows: (FIX activity at post-infusion minus FIX activity at pre-infusion) divided by weight-adjusted dose | Within 30 mins pre-infusion and 30 mins post-infusion |
| Pharmacokinetics (PK): Elimination Phase Half-life (T 1/2) | Calculated as log_e2/λ, where λ is the regression slope in the terminal phase of the least absolute deviations regression model | Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details. |
| Pharmacokinetics (PK): Volume of Distribution at Steady State (Vss) | Computed as Clearance (CL) * Mean residence time (MRT) | Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details. |
| Pharmacokinetics (PK): Incremental Recovery (IR) Over Time | IR calculated as follows: (FIX activity at post-infusion minus FIX activity at pre-infusion) divided by weight-adjusted dose. IR is determined at baseline (PK analysis), Week 5, Week 13 and Week 26 timepoints. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants > 6 years of age; pediatric participants 6 to <12 years of age; pharmacokinetic Full Analysis Set (PKFAS). | Within 30 mins pre-infusion and 30 mins post-infusion at baseline, Week 5, Week 13 and Week 26. |
| Hemostatic Efficacy: Treatment of Bleeding Episodes: Number of Infusions Per Bleeding Episode | Throughout study period (approximately 17 months) |
| Hemostatic Efficacy: Treatment of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Resolution of Bleed | Rating Scale for Treatment of bleeding episodes (4-point ordinal scale): - 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 required for the control of bleeding. Administration of further infusions to maintain hemostasis did 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 single infusion. Required more than 1 infusion for complete resolution. - None: No improvement or condition worsens. | Throughout study period (approximately 17 months) |
| Hemostatic Efficacy: Prophylaxis: Annualized Bleeding Rate (ABR) | The annualized bleeding rate (ABR) during prophylaxis was calculated only for participants who had adequate treatment time for bleeding rate assessment (i.e., more than 3 months of prophylaxis treatment). The observation period for prophylaxis was to be the time between the first and the last prophylactic infusions. The treatment period for surgery was to be excluded from the bleed rate calculation. ABR calculated as (Number of bleeding episodes/observed treatment period in days) * 365.25. | Throughout study period (approximately 17 months) |
| Consumption of BAX326: Number of Infusions Per Month | Throughout study period (approximately 17 months) |
| Consumption of BAX326: Number of Infusions Per Year | Throughout study period (approximately 17 months) |
| Consumption of BAX326: Weight-adjusted Consumption Per Month | Throughout study period (approximately 17 months) |
| Consumption of BAX326: Weight-adjusted Consumption Per Year (Annualized) | Throughout study period (approximately 17 months) |
| Consumption of BAX326: Weight-adjusted Consumption Per Event | Event includes prophylactic infusions of study product and infusions of study product for treatment of bleeding episodes (BEs). | Throughout study period (approximately 17 months) |
| Safety and Immunogenicity: Number of Participants Who Developed Inhibitory Antibodies to Factor IX (FIX) | Throughout study period (approximately 17 months) |
| Safety and Immunogenicity: Number of Participants Who Developed Total Binding Antibodies to Factor IX (FIX) | If more than 2-dilution increase as compared to pre-study level at screening and titers verified for specificity in the confirmatory assay. AB=antibodies in category for outcome measure data. | Throughout study period (approximately 17 months) |
| Safety: Number of Participants With Severe Allergic Reactions, e.g. Anaphylaxis | Throughout study period (approximately 17 months) |
| Safety: Number of Participants With Thrombotic Events | Throughout study period (approximately 17 months) |
| Safety: Number of Participants With Clinically Significant Changes in Routine Laboratory Parameters (Haematology and Clinical Chemistry), and Vital Signs | Categories consist of Clinically Significant (CS) changes in haemaotology parameters, clinical chemistry parameters and vital signs. Abbreviations in categories; Clin=clinical; params=parameters | Throughout study period (approximately 17 months) |
| Safety: Number of Participants Who Developed Antibodies to Chinese Hamster Ovary (CHO) Proteins and Recombinant Furin (rFurin) | If more than 2-dilution increase as compared to pre-study level at screening and titers verified for specificity in the confirmatory assay. | Throughout study period (approximately 17 months) |
| Health-related Quality of Life (HRQoL): PedsQL™ Change From Baseline in Total Score | For this study, the PedsQL™ questionnaires for participants 2 to 7 years of age (parent-proxy versions for age groups 2-4 years and 5-7 years) and PedsQL™ Child version for participants 8 to 12 years of age were used. The Peds-QL is a generic Health-Related Quality of Life (HR QoL) instrument designed specifically for a pediatric population. It captures the following domains: general health/activities, feelings/emotional, social functioning, school functioning. A 5-point score is used for each domain: from 0 (never) to 4 (almost always). Items are reversed scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0 so that higher scores indicate better quality of life (QoL). The total score is the mean (average) of all scores from the 4 domains. The change from baseline in total score is reported- a positive score indicates a better QoL compared to baseline and a negative score indicates a poorer QoL compared to baseline. | Baseline and 6 months |
| Health-related Quality of Life (HRQoL): Haemo-QoL, Change From Baseline in Total Score | The Haemo-QoL is a quality of life (QoL) assessment instrument for children and adolescents with haemophilia. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. For the Haemo-QoL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100. | Baseline and 6 months |
| Health Resource Use: Number of Hospitalizations | The number of hospitalizations per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set. | Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26 |
| Health Resource Use: Length of Hospitalization | The length of hospitalization per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set. | Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26 |
| Health Resource Use: Unscheduled Doctor's Office Visits | The number of unscheduled doctor's Office visits per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set. | Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26 |
| Health Resource Use: Emergency Room Visits | The number of Emergency Room visits per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set. | Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26 |
| Health Resource Use: Days Lost From School | The number of days lost from school per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set. | Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26 |
| Krakow |
| 30-663 |
| Poland |
| Stanislaw Popowski Provincial Specialist Pediatric Hospital | Olsztyn | 10-561 | Poland |
| Professor Tadeusz Sokolowski Independent Public Teaching Hospital of the Pomeranian Medical University in Szczecin | Szczecin | 71-252 | Poland |
| S.C. Sanador SRL | Bucharest | 11156 | Romania |
| Louis Turcanu Emergency Children's Hospital | Timișoara | 300011 | Romania |
| Pediatric Regional Clinical Hospital, Hematology Department | Krasnodar | 350007 | Russia |
| Republican Center for Hemophilia Treatment | Saint Petersburg | 195213 | Russia |
| Regional Clinical Hospital | Yekaterinburg | 620149 | Russia |
| State Institution "Institute of Blood Pathology and Transfusion Medicine of the Academy of Medical Sciences of Ukraine" | Lviv | 79044 | Ukraine |
| Manchester Children´s Hospital | Manchester | M13 9WL | United Kingdom |
| BG001 | Pediatric Participants 6 to <12 Years of Age | Pediatric participants 6 to <12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| OG001 |
| Pediatric Participants 6 to < 12 Years of Age |
Pediatric participants 6 to < 12 years of age. All pediatric participants underwent pharmacokinetic (PK) assessment followed by prophylactic treatment with the study product. All participants received the same dosing schedule of study product during the study. After a washout period of 5-7 days, participants received an initial infusion of study product at a dose of 75±5 IU/kg for the PK assessment. For the prophylactic regimen, participants were treated with the recommended dose of 50 IU/kg of study product twice weekly ranging from 40-80 IU/kg for 26±1 weeks or for at least 50 EDs to study product, whichever occurred last. |
| OG002 | Full Analysis Set | Comprised of all participants who received at least one infusion of study product |
|
|
| Secondary | Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to 72 Hours Post-infusion Per Dose (AUC 0-72h/Dose) | On completion of the study, prospective changes to the planned statistical analysis were made not to analyze AUC 0-72h due to the different time points for the last PK blood sample. Only total AUC [i.e. AUC 0-infinity] was included in the PK analysis. | Posted | Within 30 mins pre-infusion and 4 post-infusion timepoints |
|
|
| Secondary | Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve From 0 to Infinity Post-infusion Per Dose (Total AUC/Dose) | All participants randomized to 2 groups: Group 1 - BAX326 infusion in the morning PK timepoints: Pre-infusion and post-infusion at 15-30 minutes, then 7, 28 and 52 hours Group 2 - BAX326 infusion in the afternoon PK timepoints: Pre-infusion and post-infusion of 15-30 minutes, then 4, 24 and 69 hours | Posted | Mean | Standard Deviation | IU*hour (hr)/dL | Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details. |
|
|
|
| Secondary | Pharmacokinetics (PK): Mean Residence Time (MRT) | Computed as total area under the first moment curve (total AUMC) divided by the total area under the concentration versus time curve (total AUC) | All participants randomized to 2 groups: Group 1 - BAX326 infusion in the morning PK timepoints: Pre-infusion and post-infusion at 15-30 minutes, then 7, 28 and 52 hours Group 2 - BAX326 infusion in the afternoon PK timepoints: Pre-infusion and post-infusion of 15-30 minutes, then 4, 24 and 69 hours | Posted | Mean | Standard Deviation | hours (hr) | Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details. |
|
|
|
| Secondary | Pharmacokinetics (PK): Factor IX (FIX) Clearance (CL) | Computed as the dose divided by total Area under the curve (AUC) | All participants randomized to 2 groups: Group 1 - BAX326 infusion in the morning PK timepoints: Pre-infusion and post-infusion at 15-30 minutes, then 7, 28 and 52 hours Group 2 - BAX326 infusion in the afternoon PK timepoints: Pre-infusion and post-infusion of 15-30 minutes, then 4, 24 and 69 hours | Posted | Mean | Standard Deviation | dL/(kg*hr) | Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details. |
|
|
|
| Secondary | Pharmacokinetics (PK): Incremental Recovery (IR) | The rise in FIX activity in IU/dL per unit dose administered in IU/kg. Calculated as follows: (FIX activity at post-infusion minus FIX activity at pre-infusion) divided by weight-adjusted dose | Posted | Mean | Standard Deviation | IU/dL : IU/kg | Within 30 mins pre-infusion and 30 mins post-infusion |
|
|
|
| Secondary | Pharmacokinetics (PK): Elimination Phase Half-life (T 1/2) | Calculated as log_e2/λ, where λ is the regression slope in the terminal phase of the least absolute deviations regression model | All participants randomized to 2 groups: Group 1 - BAX326 infusion in the morning PK timepoints: Pre-infusion and post-infusion at 15-30 minutes, then 7, 28 and 52 hours Group 2 - BAX326 infusion in the afternoon PK timepoints: Pre-infusion and post-infusion of 15-30 minutes, then 4, 24 and 69 hours | Posted | Mean | Standard Deviation | hours (hr) | Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details. |
|
|
|
| Secondary | Pharmacokinetics (PK): Volume of Distribution at Steady State (Vss) | Computed as Clearance (CL) * Mean residence time (MRT) | All participants randomized to 2 groups: Group 1 - BAX326 infusion in the morning PK timepoints: Pre-infusion and post-infusion at 15-30 minutes, then 7, 28 and 52 hours Group 2 - BAX326 infusion in the afternoon PK timepoints: Pre-infusion and post-infusion of 15-30 minutes, then 4, 24 and 69 hours | Posted | Mean | Standard Deviation | dL/kg | Within 30 mins pre-infusion and 4 post-infusion timepoints. Refer to Population Description below for more details. |
|
|
|
| Secondary | Pharmacokinetics (PK): Incremental Recovery (IR) Over Time | IR calculated as follows: (FIX activity at post-infusion minus FIX activity at pre-infusion) divided by weight-adjusted dose. IR is determined at baseline (PK analysis), Week 5, Week 13 and Week 26 timepoints. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants > 6 years of age; pediatric participants 6 to <12 years of age; pharmacokinetic Full Analysis Set (PKFAS). | Posted | Mean | Standard Deviation | IU/dL : IU/kg | Within 30 mins pre-infusion and 30 mins post-infusion at baseline, Week 5, Week 13 and Week 26. |
|
|
|
| Secondary | Hemostatic Efficacy: Treatment of Bleeding Episodes: Number of Infusions Per Bleeding Episode | Participants in the Full Analysis Set who had bleeding episodes | Posted | Number | Bleeding Episodes | Throughout study period (approximately 17 months) |
|
|
|
| Secondary | Hemostatic Efficacy: Treatment of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Resolution of Bleed | Rating Scale for Treatment of bleeding episodes (4-point ordinal scale): - 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 required for the control of bleeding. Administration of further infusions to maintain hemostasis did 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 single infusion. Required more than 1 infusion for complete resolution. - None: No improvement or condition worsens. | Participants in the Full Analysis Set who had bleeding episodes | Posted | Number | Bleeding Episodes | Throughout study period (approximately 17 months) | Bleeding Episodes | Bleeding Episodes |
|
|
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| Secondary | Hemostatic Efficacy: Prophylaxis: Annualized Bleeding Rate (ABR) | The annualized bleeding rate (ABR) during prophylaxis was calculated only for participants who had adequate treatment time for bleeding rate assessment (i.e., more than 3 months of prophylaxis treatment). The observation period for prophylaxis was to be the time between the first and the last prophylactic infusions. The treatment period for surgery was to be excluded from the bleed rate calculation. ABR calculated as (Number of bleeding episodes/observed treatment period in days) * 365.25. | Posted | Mean | Standard Deviation | Bleeding episodes per year | Throughout study period (approximately 17 months) |
|
|
|
| Secondary | Consumption of BAX326: Number of Infusions Per Month | Posted | Mean | Standard Deviation | Infusions per month | Throughout study period (approximately 17 months) |
|
|
|
| Secondary | Consumption of BAX326: Number of Infusions Per Year | Posted | Mean | Standard Deviation | Infusions per year | Throughout study period (approximately 17 months) |
|
|
|
| Secondary | Consumption of BAX326: Weight-adjusted Consumption Per Month | Posted | Mean | Standard Deviation | IU/kg per month | Throughout study period (approximately 17 months) |
|
|
|
| Secondary | Consumption of BAX326: Weight-adjusted Consumption Per Year (Annualized) | Posted | Mean | Standard Deviation | IU/kg per year | Throughout study period (approximately 17 months) |
|
|
|
| Secondary | Consumption of BAX326: Weight-adjusted Consumption Per Event | Event includes prophylactic infusions of study product and infusions of study product for treatment of bleeding episodes (BEs). | Posted | Mean | Standard Deviation | IU/kg | Throughout study period (approximately 17 months) |
|
|
|
| Secondary | Safety and Immunogenicity: Number of Participants Who Developed Inhibitory Antibodies to Factor IX (FIX) | Posted | Number | Participants | Throughout study period (approximately 17 months) |
|
|
|
| Secondary | Safety and Immunogenicity: Number of Participants Who Developed Total Binding Antibodies to Factor IX (FIX) | If more than 2-dilution increase as compared to pre-study level at screening and titers verified for specificity in the confirmatory assay. AB=antibodies in category for outcome measure data. | Posted | Number | Participants | Throughout study period (approximately 17 months) |
|
|
|
| Secondary | Safety: Number of Participants With Severe Allergic Reactions, e.g. Anaphylaxis | Posted | Number | Participants | Throughout study period (approximately 17 months) |
|
|
|
| Secondary | Safety: Number of Participants With Thrombotic Events | Posted | Number | Participants | Throughout study period (approximately 17 months) |
|
|
|
| Secondary | Safety: Number of Participants With Clinically Significant Changes in Routine Laboratory Parameters (Haematology and Clinical Chemistry), and Vital Signs | Categories consist of Clinically Significant (CS) changes in haemaotology parameters, clinical chemistry parameters and vital signs. Abbreviations in categories; Clin=clinical; params=parameters | Posted | Number | Participants | Throughout study period (approximately 17 months) |
|
|
|
| Secondary | Safety: Number of Participants Who Developed Antibodies to Chinese Hamster Ovary (CHO) Proteins and Recombinant Furin (rFurin) | If more than 2-dilution increase as compared to pre-study level at screening and titers verified for specificity in the confirmatory assay. | Posted | Number | Participants | Throughout study period (approximately 17 months) |
|
|
|
| Secondary | Health-related Quality of Life (HRQoL): PedsQL™ Change From Baseline in Total Score | For this study, the PedsQL™ questionnaires for participants 2 to 7 years of age (parent-proxy versions for age groups 2-4 years and 5-7 years) and PedsQL™ Child version for participants 8 to 12 years of age were used. The Peds-QL is a generic Health-Related Quality of Life (HR QoL) instrument designed specifically for a pediatric population. It captures the following domains: general health/activities, feelings/emotional, social functioning, school functioning. A 5-point score is used for each domain: from 0 (never) to 4 (almost always). Items are reversed scored and linearly transformed to a 0-100 scale as follows: 0=100, 1=75, 2=50, 3=25, 4=0 so that higher scores indicate better quality of life (QoL). The total score is the mean (average) of all scores from the 4 domains. The change from baseline in total score is reported- a positive score indicates a better QoL compared to baseline and a negative score indicates a poorer QoL compared to baseline. | Participants in the Full Analysis Set who had PedsQL™ data for baseline and 6 months | Posted | Mean | Standard Deviation | Scores on a scale | Baseline and 6 months |
|
|
|
| Secondary | Health-related Quality of Life (HRQoL): Haemo-QoL, Change From Baseline in Total Score | The Haemo-QoL is a quality of life (QoL) assessment instrument for children and adolescents with haemophilia. As a hemophilia-specific instrument, this measure assesses very specific aspects of dealing with hemophilia. For the Haemo-QoL, higher scores indicate a worse quality of life. Scores on a scale range between 0 and 100. | Participants in the Full Analysis Set who had Haemo-QoL data for baseline and 6 months | Posted | Mean | Standard Deviation | Scores on a scale | Baseline and 6 months |
|
|
|
| Secondary | Health Resource Use: Number of Hospitalizations | The number of hospitalizations per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set. | Posted | Median | Full Range | Hospitalizations | Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26 |
|
|
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| Secondary | Health Resource Use: Length of Hospitalization | The length of hospitalization per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set. | Posted | Median | Full Range | Days | Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26 |
|
|
|
| Secondary | Health Resource Use: Unscheduled Doctor's Office Visits | The number of unscheduled doctor's Office visits per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set. | Participants who had unscheduled visits to a doctor's office | Posted | Median | Full Range | Visits | Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26 |
|
|
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| Secondary | Health Resource Use: Emergency Room Visits | The number of Emergency Room visits per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set. | Posted | Median | Full Range | Visits | Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26 |
|
|
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| Secondary | Health Resource Use: Days Lost From School | The number of days lost from school per participant. Number of participants contributing data (N) for this outcome measure is included in the category title in the order: pediatric participants < 6 years of age; pediatric participants 6 to <12 years of age; Full Analysis Set. | Participants who missed days from school | Posted | Median | Full Range | Days | Baseline (Pharmacokinetic [PK] assessment), Week 5, Week 13 and Week 26 |
|
|
|
| 3 |
| 23 |
| 15 |
| 23 |
| Humerus fracture | Injury, poisoning and procedural complications | MedDRA (Unspecified) | Systematic Assessment |
|
| Hemarthrosis | Musculoskeletal and connective tissue disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Subcutaneous hemorrhage | Skin and subcutaneous tissue disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Toothache | Gastrointestinal disorders | MedDRA (Unspecified) | Systematic Assessment |
|
| Bronchitis | Infections and infestations | MedDRA (Unspecified) | Non-systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
|
| Respiratory tract infection viral | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
|
| Rhinitis | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA (Unspecified) | Systematic Assessment |
|
| Immunology test abnormal | Investigations | MedDRA (Unspecified) | Systematic Assessment | Total binding antibodies (ABs) to FIX developed in 4 subjects, AB to FIX and rFurin developed in 1 subject, and AB to rFurin developed twice in 1 subject. All ABs were of indeterminate specificity and too low to be verified in the confirmatory assay. |
|
| Headache | Nervous system disorders | MedDRA (Unspecified) | Systematic Assessment |
|
Baxalta's agreements with PIs may vary per individual PI, but contain common elements. For this study, PIs may be restricted from independently publishing results without prior approval.
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
|
| Week 13 (N=10, 11, 21) |
|
| Week 26 (N=10, 11, 21) |
|
|
| Bleeding episodes controlled with ≥ 3 infusions |
|
| Title | Measurements |
|---|---|
|
| Fair |
|
| None |
|
|
|
| Number with CS changes in vital signs |
|
|
| Title | Measurements |
|---|---|
|
|
| Week 26 (N=11, 11, 22) |
|
| Week 5 |
|
| Week 13 (N=1, 1, 2) |
|
| Week 26 (N=NA, 1, 1) |
|
|
| Week 13 (N=11, 11, 22) |
|
| Week 26 (N=11, 11, 22) |
|
|
| Week 13 (N=11, 11, 22) |
|
| Week 26 (N=11, 11, 22) |
|
|
| Week 13 (N=11, 11, 22) |
|
| Week 26 (N=11, 11, 22) |
|