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| ID | Type | Description | Link |
|---|---|---|---|
| 2016-000765-22 | EudraCT Number |
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The purpose of this post-approval study is to evaluate the safety and efficacy of Benefix in subjects with hemophilia B in usual care settings in China.
The purpose of this post-approval study is to provide supplementary information relating to the use of BeneFIX in Chinese subjects with hemophilia B, especially on the safety and efficacy in different populations of Chinese hemophilia B patients, in particular in pediatric patients <6 years of age, pediatric patients ≥6 to ≤12 years of age, Previously Untreated Patients (PUPs) , subjects receiving prophylaxis treatment after enrollment in the study, and severe patients (FIX activity <1%).
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Benefix | Experimental | This is a single arm study. Subjects will be treated with Benefix by the investigator according to usual care in China and in accord with the China BeneFIX Package Insert. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Benefix | Drug | Subjects will be treated by the investigator according to usual care in China and in accord with the China BeneFIX Package Insert. The treatment duration is approximately 6 months (±7 days) or approximately 50 Exposure Days (EDs) (±5 EDs) (see protocol definition on EDs) whichever occurs first. |
| Measure | Description | Time Frame |
|---|---|---|
| Percentage of Participants Who Developed Factor IX Inhibitor | Factor IX (FIX) inhibitor development was defined as any Bethesda inhibitor titer greater than the laboratory's normal range or Bethesda inhibitor titer >=0.6 Bethesda Unit (BU)/mL. | Up to 6 months |
| Number of Participants With Allergic Reactions | FIX product allergy was defined as a hypersensitivity reaction to a FIX product with symptoms such as hives, urticaria, tightness of chest, wheezing, hypotension, and anaphylaxis based on investigator's judgments. | Up to 6 months |
| Number of Participants With Thrombotic Events | Thrombotic event was defined as any event associated with formation of a blood clot including catheter-associated thrombi and thrombotic complications. | Up to 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a product or medical device, regardless of the causality with the treatment or usage. A serious adverse event (SAE) was any untoward occurrence at any dose that resulted in death; was life threatening (immediate risk of death); required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity; resulted in congenital anomaly/birth defect. AEs included both serious and non-serious AEs. Treatment-emergent AEs were those with initial onset or increasing in severity after the first dose of study drug. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Nanfang Hospital, Southern Medical University | Guangzhou | Guangdong | 510515 | China | ||
| The Affiliated Hospital of Guizhou Medical University |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 34032739 | Derived | Yang R, Wu R, Sun J, Sun F, Rupon J, Huard F, Korth-Bradley JM, Xu L, Luo B, Liu YC, Rendo P. First open-label, single-arm, prospective study of real-world use of FIX replacement therapy in a predominantly pediatric hemophilia B population in China. Medicine (Baltimore). 2021 May 28;100(21):e26077. doi: 10.1097/MD.0000000000026077. |
| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
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This study enrolled 70 participants.
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| ID | Title | Description |
|---|---|---|
| FG000 | BeneFIX | Participants received intravenous (IV) infusions of BeneFIX for on-demand or prophylaxis treatment at a dose and frequency prescribed by the participant's treating physician in accordance with the China BeneFIX Package Insert for 6 months (±7 days) or 50 Exposure Days (EDs) (±5 EDs), whichever occurred first. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
The baseline analysis population included all enrolled participants who received the study drug.
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| ID | Title | Description |
|---|---|---|
| BG000 | BeneFIX | Participants received intravenous (IV) infusions of BeneFIX for on-demand or prophylaxis treatment at a dose and frequency prescribed by the participant's treating physician in accordance with the China BeneFIX Package Insert for 6 months (±7 days) or 50 Exposure Days (EDs) (±5 EDs), whichever occurred first. |
| 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 | Percentage of Participants Who Developed Factor IX Inhibitor | Factor IX (FIX) inhibitor development was defined as any Bethesda inhibitor titer greater than the laboratory's normal range or Bethesda inhibitor titer >=0.6 Bethesda Unit (BU)/mL. | All participants who received at least 1 dose of BeneFIX during the study. Participants in each arm were not mutually exclusive. | Posted | Number | 95% Confidence Interval | percentage of participants | Up to 6 months |
|
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The same event may appear as both an AE and a SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another participant, or one participant may have experienced both a serious and non-serious event during the study.
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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 | BeneFIX | Participants received intravenous (IV) infusions of BeneFIX for on-demand or prophylaxis treatment at a dose and frequency prescribed by the participant's treating physician in accordance with the China BeneFIX Package Insert for 6 months (±7 days) or 50 Exposure Days (EDs) (±5 EDs), whichever occurred first. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Factor IX inhibition | Blood and lymphatic system disorders | MedDRA 19.0 | Non-systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA 19.0 | Non-systematic Assessment |
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer, Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
Not provided
| 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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| ID | Term |
|---|---|
| D005164 | Factor IX |
| ID | Term |
|---|---|
| D004792 | Enzyme Precursors |
| D045762 | Enzymes and Coenzymes |
| D001779 | Blood Coagulation Factors |
| D001798 | Blood Proteins |
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|
| Up to 7 months (28 calendar days after end of 6-month or 50-exposure day study treatment) |
| Annualized Bleeding Rates (ABRs) in Participants Receiving Prophylaxis Treatment With BeneFIX During Their Prophylaxis Period | For prophylaxis period, annualized bleeding rate (ABR) was derived for each participant by the following formula: ABR = number of bleeds in prophylaxis period / (number of days in prophylaxis period/365.25). A prophylaxis period was defined as time from first prophylaxis infusion through 6 calendar days after the day of last prophylaxis infusion, or the day of study conclusion visit, whichever was earlier. A break in the prophylaxis period was any period of 28 days or longer in which no prophylaxis infusions were given. For a prophylaxis regimen to have been qualified to have ABR calculated, the sum of its periods needed to be >=14 days. | Up to 6 months or 50 exposure days whichever occurred first |
| Number of Spontaneous/Non-traumatic Breakthrough Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX | The prophylaxis infusion time, bleed start time and bleed type (etiology) were used to determine the number of spontaneous, non-traumatic breakthrough bleeds that occurred <=48 hours after a prophylaxis infusion. If there was more than 1 bleed location (eg, ankle and joint) with identical bleed start date and time, it was treated as 1 bleed occurrence. | Up to 6 months or 50 exposure days whichever occurred first |
| Annualized Bleeding Rates (ABRs) in Participants Receiving On-demand Treatment With BeneFIX During Their On-demand Period | For on-demand period, the annualized bleeding rate (ABR) was derived for each participant by the following formula: ABR = number of bleeds in on-demand period / (number of days in on-demand period/365.25). The on-demand period was defined as the entire time of enrollment in the study (ie, from the date of the enrollment visit through the day before the Final/Early Termination visit) except time in any prophylaxis period. The breaks in the prophylaxis period were considered on-demand periods. For an on-demand regimen to have been qualified to have ABR calculated, the sum of its periods needed to be >= 14 days. | Up to 6 months or 50 exposure days whichever occurred first |
| Number of Infusions Resulted in the Following Response to On-demand Treatment of Bleeds: Excellent, Good, Moderate, no Response | Response was assessed using 4-point On-Demand Hemostasis Efficacy Rating Scale. Excellent means definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with no additional infusion administered; good means definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with at least one additional infusion administered for complete resolution of the bleeding episode, or definite pain relief and/or improvement in signs of bleeding starting after 8 hours following the infusion, with no additional infusion administered; moderate means probable or slight improvement starting after 8 hours following the infusion, with at least one additional infusion administered for complete resolution of the bleeding episode; no response means no improvement between infusions or during the 24 hour interval following an infusion, or condition worsens. Both first infusions and follow-up infusions were included. | Up to 6 months or 50 exposure days whichever occurred first |
| Number of BeneFIX Infusions to Treat Each New Bleed | The number of BeneFIX infusions administered to treat each new bleed was calculated by adding the on-demand initial treatment and any on-demand follow-up treatments for the same bleed. If there was more than one bleed location (eg, ankle and joint) with identical bleed start date and time, it was treated as one bleed occurrence. | Up to 6 months or 50 exposure days whichever occurred first |
| Average Infusion Dose and Total Factor IX Consumption in On-demand Setting | The total amount (international units [IU]) infused for each BeneFIX infusion was summed to calculate the total factor IX consumption for each participant. The average infusion dose for each participant was calculated as his total factor IX consumption (in IU) divided by the number of infusions administered. | Up to 6 months or 50 exposure days whichever occurred first |
| Average Infusion Dose and Total Factor IX Consumption in Prophylaxis Setting | The total amount (international units [IU]) infused for each BeneFIX infusion was summed to calculate the total factor IX consumption for each participant. The average infusion dose for each participant was calculated as his total factor IX consumption (in IU) divided by the number of infusions administered. | Up to 6 months or 50 exposure days whichever occurred first |
| Average Infusion Dose and Total Factor IX Consumption in Recovery Setting | The total amount (international units [IU]) infused for each BeneFIX infusion was summed to calculate the total factor IX consumption for each participant. The average infusion dose for each participant was calculated as his total factor IX consumption (in IU) divided by the number of infusions administered. | Up to 6 months or 50 exposure days whichever occurred first |
| Percentage of Infusions With Less Than Expected Therapeutic Effects (LETEs) in On-demand Setting | Less than expected therapeutic effect (LETE) in the on-demand setting was defined as 2 successive "no response" ratings recorded after 2 successive BeneFIX drug infusions, respectively. | Up to 6 months or 50 exposure days whichever occurred first |
| Percentage of Infusions With Less Than Expected Therapeutic Effects (LETEs) in Prophylaxis Setting | Less than expected therapeutic effect (LETE) in the prophylaxis setting was defined as occurrence of any spontaneous bleed within 48 hours after a regularly scheduled prophylactic dose of BeneFIX (which was not used to treat a bleed). | Up to 6 months or 50 exposure days whichever occurred first |
| Guiyang |
| Guizhou |
| 550004 |
| China |
| Henan Provincial People's Hospital | Zhengzhou | Henan | 450003 | China |
| Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology | Wuhan | Hubei | 430030 | China |
| Xiangya Hospital of Centre-South University | Changsha | Hunan | 410008 | China |
| Department of Hematology,The First Affiliated Hospital of Soochow University | Suzhou | Jiangsu | 215006 | China |
| Department of Hematology,The Affiliated Hospital of Xuzhou Medical University | Xuzhou | Jiangsu | 221006 | China |
| Department of Hematology,Jiangxi Provincial People's Hospital | Nanchang | Jiangxi | 330006 | China |
| Blood Center of Shandong Province | Jinan | Shandong | 250014 | China |
| Ruijin Hospital Affiliated to Shanghai JiaoTong University School of Medicine/Hematology Department | Shanghai | Shanghai Municipality | 200025 | China |
| Shanxi Medical University Second Hospital | Taiyuan | Shanxi | 030001 | China |
| Chengdu Women's and Children's Central Hospital | Chengdu | Sichuan | 610073 | China |
| Blood Diseases Hospital, Chinese Academy of Medical Science (Institute of Hematology) | Tianjin | Tianjin Municipality | 300020 | China |
| Department of Hematology,The First Affiliated Hospital of Kunming Medical University | Kunming | Yunnan | 650032 | China |
| Hematology Department,Beijing Children's Hospital, Capital Medical University | Beijing | 100045 | China |
| Children's Hospital of Chongqing Medical University | Chongqing | 400014 | China |
| Other |
|
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| OG001 |
| Pediatric Participants ≥6 to ≤12 Years of Age |
Participants received intravenous (IV) infusions of BeneFIX for on-demand or prophylaxis treatment at a dose and frequency prescribed by the participant's treating physician in accordance with the China BeneFIX Package Insert for 6 months (±7 days) or 50 Exposure Days (EDs) (±5 EDs), whichever occurred first. |
| OG002 | Previously Untreated Participants (PUPs) | Participants received intravenous (IV) infusions of BeneFIX for on-demand or prophylaxis treatment at a dose and frequency prescribed by the participant's treating physician in accordance with the China BeneFIX Package Insert for 6 months (±7 days) or 50 Exposure Days (EDs) (±5 EDs), whichever occurred first. |
| OG003 | Participants Receiving Prophylaxis Treatment | Participants received intravenous (IV) infusions of BeneFIX for on-demand or prophylaxis treatment at a dose and frequency prescribed by the participant's treating physician in accordance with the China BeneFIX Package Insert for 6 months (±7 days) or 50 Exposure Days (EDs) (±5 EDs), whichever occurred first. This reporting arm included all participants who received prophylaxis treatment after enrollment in the study. |
| OG004 | Severe Participants (Factor IX Activity <1%) | Participants received intravenous (IV) infusions of BeneFIX for on-demand or prophylaxis treatment at a dose and frequency prescribed by the participant's treating physician in accordance with the China BeneFIX Package Insert for 6 months (±7 days) or 50 Exposure Days (EDs) (±5 EDs), whichever occurred first. |
| OG005 | Overall Participants | Participants received intravenous (IV) infusions of BeneFIX for on-demand or prophylaxis treatment at a dose and frequency prescribed by the participant's treating physician in accordance with the China BeneFIX Package Insert for 6 months (±7 days) or 50 Exposure Days (EDs) (±5 EDs), whichever occurred first. This reporting arm included all participants who received at least 1 dose of BeneFIX during the study. |
|
|
| Primary | Number of Participants With Allergic Reactions | FIX product allergy was defined as a hypersensitivity reaction to a FIX product with symptoms such as hives, urticaria, tightness of chest, wheezing, hypotension, and anaphylaxis based on investigator's judgments. | All participants who received at least 1 dose of BeneFIX during the study. Participants in each arm were not mutually exclusive. | Posted | Number | participants | Up to 6 months |
|
|
|
| Primary | Number of Participants With Thrombotic Events | Thrombotic event was defined as any event associated with formation of a blood clot including catheter-associated thrombi and thrombotic complications. | All participants who received at least 1 dose of BeneFIX during the study. Participants in each arm were not mutually exclusive. | Posted | Number | participants | Up to 6 months |
|
|
|
| Secondary | Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious Adverse Events (SAEs) | An adverse event (AE) was any untoward medical occurrence in a clinical investigation participant administered a product or medical device, regardless of the causality with the treatment or usage. A serious adverse event (SAE) was any untoward occurrence at any dose that resulted in death; was life threatening (immediate risk of death); required inpatient hospitalization or prolongation of existing hospitalization; resulted in persistent or significant disability/incapacity; resulted in congenital anomaly/birth defect. AEs included both serious and non-serious AEs. Treatment-emergent AEs were those with initial onset or increasing in severity after the first dose of study drug. | All participants who received at least 1 dose of BeneFIX during the study. Participants in each arm were not mutually exclusive. | Posted | Number | participants | Up to 7 months (28 calendar days after end of 6-month or 50-exposure day study treatment) |
|
|
|
| Secondary | Annualized Bleeding Rates (ABRs) in Participants Receiving Prophylaxis Treatment With BeneFIX During Their Prophylaxis Period | For prophylaxis period, annualized bleeding rate (ABR) was derived for each participant by the following formula: ABR = number of bleeds in prophylaxis period / (number of days in prophylaxis period/365.25). A prophylaxis period was defined as time from first prophylaxis infusion through 6 calendar days after the day of last prophylaxis infusion, or the day of study conclusion visit, whichever was earlier. A break in the prophylaxis period was any period of 28 days or longer in which no prophylaxis infusions were given. For a prophylaxis regimen to have been qualified to have ABR calculated, the sum of its periods needed to be >=14 days. | All participants who participated in at least 1 day of a prophylaxis period (ie, had at least 1 prophylaxis dose). Participants in each arm were not mutually exclusive. | Posted | Mean | Standard Deviation | episodes/year | Up to 6 months or 50 exposure days whichever occurred first |
|
|
|
| Secondary | Number of Spontaneous/Non-traumatic Breakthrough Bleeds Within 48 Hours of a Prophylaxis Dose of BeneFIX | The prophylaxis infusion time, bleed start time and bleed type (etiology) were used to determine the number of spontaneous, non-traumatic breakthrough bleeds that occurred <=48 hours after a prophylaxis infusion. If there was more than 1 bleed location (eg, ankle and joint) with identical bleed start date and time, it was treated as 1 bleed occurrence. | All participants with any spontaneous/non-traumatic breakthrough bleeds within 48 hours of a prophylaxis dose. Participants in each arm were not mutually exclusive. | Posted | Mean | Standard Deviation | breakthrough bleeds | Up to 6 months or 50 exposure days whichever occurred first | Prophylaxis Infusions with Bleeds | Prophylaxis Infusions with Bleeds |
|
|
|
| Secondary | Annualized Bleeding Rates (ABRs) in Participants Receiving On-demand Treatment With BeneFIX During Their On-demand Period | For on-demand period, the annualized bleeding rate (ABR) was derived for each participant by the following formula: ABR = number of bleeds in on-demand period / (number of days in on-demand period/365.25). The on-demand period was defined as the entire time of enrollment in the study (ie, from the date of the enrollment visit through the day before the Final/Early Termination visit) except time in any prophylaxis period. The breaks in the prophylaxis period were considered on-demand periods. For an on-demand regimen to have been qualified to have ABR calculated, the sum of its periods needed to be >= 14 days. | All participants who received on-demand treatment during on-demand period. Participants in each arm were not mutually exclusive. | Posted | Mean | Standard Deviation | episodes/year | Up to 6 months or 50 exposure days whichever occurred first |
|
|
|
| Secondary | Number of Infusions Resulted in the Following Response to On-demand Treatment of Bleeds: Excellent, Good, Moderate, no Response | Response was assessed using 4-point On-Demand Hemostasis Efficacy Rating Scale. Excellent means definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with no additional infusion administered; good means definite pain relief and/or improvement in signs of bleeding starting within 8 hours after an infusion, with at least one additional infusion administered for complete resolution of the bleeding episode, or definite pain relief and/or improvement in signs of bleeding starting after 8 hours following the infusion, with no additional infusion administered; moderate means probable or slight improvement starting after 8 hours following the infusion, with at least one additional infusion administered for complete resolution of the bleeding episode; no response means no improvement between infusions or during the 24 hour interval following an infusion, or condition worsens. Both first infusions and follow-up infusions were included. | All participants who had a bleed during the study for which on-demand treatment with BeneFIX was administered. Participants in each arm were not mutually exclusive. | Posted | Number | infusions | Up to 6 months or 50 exposure days whichever occurred first | Infusions | Infusions |
|
|
|
| Secondary | Number of BeneFIX Infusions to Treat Each New Bleed | The number of BeneFIX infusions administered to treat each new bleed was calculated by adding the on-demand initial treatment and any on-demand follow-up treatments for the same bleed. If there was more than one bleed location (eg, ankle and joint) with identical bleed start date and time, it was treated as one bleed occurrence. | All participants who had a bleed during the study for which on-demand treatment with BeneFIX was administered. Participants in each arm were not mutually exclusive. | Posted | Mean | Standard Deviation | infusions | Up to 6 months or 50 exposure days whichever occurred first | Bleeds | Bleeds |
|
|
|
| Secondary | Average Infusion Dose and Total Factor IX Consumption in On-demand Setting | The total amount (international units [IU]) infused for each BeneFIX infusion was summed to calculate the total factor IX consumption for each participant. The average infusion dose for each participant was calculated as his total factor IX consumption (in IU) divided by the number of infusions administered. | All participants who had a bleed during the study for which on-demand treatment with BeneFIX was administered. Participants in each arm were not mutually exclusive. | Posted | Mean | Standard Deviation | IU | Up to 6 months or 50 exposure days whichever occurred first |
|
|
|
| Secondary | Average Infusion Dose and Total Factor IX Consumption in Prophylaxis Setting | The total amount (international units [IU]) infused for each BeneFIX infusion was summed to calculate the total factor IX consumption for each participant. The average infusion dose for each participant was calculated as his total factor IX consumption (in IU) divided by the number of infusions administered. | All participants who received at least 1 dose of prophylaxis BeneFIX treatment during the study. Participants in each arm were not mutually exclusive. | Posted | Mean | Standard Deviation | IU | Up to 6 months or 50 exposure days whichever occurred first |
|
|
|
| Secondary | Average Infusion Dose and Total Factor IX Consumption in Recovery Setting | The total amount (international units [IU]) infused for each BeneFIX infusion was summed to calculate the total factor IX consumption for each participant. The average infusion dose for each participant was calculated as his total factor IX consumption (in IU) divided by the number of infusions administered. | All participants who received at least 1 recovery infusion with BeneFIX during the study. Participants in each arm were not mutually exclusive. | Posted | Mean | Standard Deviation | IU | Up to 6 months or 50 exposure days whichever occurred first |
|
|
|
| Secondary | Percentage of Infusions With Less Than Expected Therapeutic Effects (LETEs) in On-demand Setting | Less than expected therapeutic effect (LETE) in the on-demand setting was defined as 2 successive "no response" ratings recorded after 2 successive BeneFIX drug infusions, respectively. | All participants who had a bleed during the study for which on-demand treatment with BeneFIX was administered. Participants in each arm were not mutually exclusive. | Posted | Number | 95% Confidence Interval | percentage of infusions | Up to 6 months or 50 exposure days whichever occurred first | Bleeding Episodes | Bleeding Episodes |
|
|
|
| Secondary | Percentage of Infusions With Less Than Expected Therapeutic Effects (LETEs) in Prophylaxis Setting | Less than expected therapeutic effect (LETE) in the prophylaxis setting was defined as occurrence of any spontaneous bleed within 48 hours after a regularly scheduled prophylactic dose of BeneFIX (which was not used to treat a bleed). | All participants who received at least 1 dose of prophylaxis BeneFIX treatment during the study. Participants in each arm were not mutually exclusive. | Posted | Number | 95% Confidence Interval | percentage of infusions | Up to 6 months or 50 exposure days whichever occurred first | Prophylaxis Infusions | Prophylaxis Infusions |
|
|
|
| 2 |
| 70 |
| 55 |
| 70 |
| Oral mucosa haematoma | Gastrointestinal disorders | MedDRA 19.0 | Non-systematic Assessment |
|
| Peripheral swelling | General disorders | MedDRA 19.0 | Non-systematic Assessment |
|
| Pyrexia | General disorders | MedDRA 19.0 | Non-systematic Assessment |
|
| Nasopharyngitis | Infections and infestations | MedDRA 19.0 | Non-systematic Assessment |
|
| Upper respiratory tract infection | Infections and infestations | MedDRA 19.0 | Non-systematic Assessment |
|
| Contusion | Injury, poisoning and procedural complications | MedDRA 19.0 | Non-systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | MedDRA 19.0 | Non-systematic Assessment |
|
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Non-systematic Assessment |
|
| Joint range of motion decreased | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Non-systematic Assessment |
|
| Joint swelling | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Non-systematic Assessment |
|
| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 19.0 | Non-systematic Assessment |
|
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 19.0 | Non-systematic Assessment |
|
Pfizer has the right to review disclosures, requesting a delay of less than 60 days. Investigator will postpone single center publications until after disclosure of pooled data (all sites), less than 12 months from study completion/termination at all participating sites. Investigator may not disclose previously undisclosed confidential information other than study results.
| D020147 | Coagulation Protein Disorders |
| D006474 | Hemorrhagic Disorders |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D040181 | Genetic Diseases, X-Linked |
| D011506 |
| Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D011498 | Protein Precursors |
| D001685 | Biological Factors |
| SAEs |
|
| Prophylaxis Infusions with Bleeds |
|
| Infusions |
|
| Good |
|
| Moderate |
|
| No response |
|
| Bleeds |
|
| Average infusion dose |
|
| Average infusion dose |
|
| Average infusion dose |
|
| Bleeding Episodes |
|
| Prophylaxis Infusions |
|