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This study aims to observe the safety and efficacy of the Xyntha Solofuse prefilled syringe in the setting of routine practice. The primary objective is to detect medically significant events (factor VIII inhibitor). The secondary objective is to observe the overall efficacy and safety of the Xyntha Solofuse prefilled syringe including serious adverse events. In this open-label, non-comparative, observational, non-interventional, retrospective and multi-center study, post-marketing surveillance data will be collected retrospectively for up to 6 months from the initial administration day of the Xyntha Solofuse prefilled syringe injected into patients who have been administered the Xyntha Solofuse prefilled syringe.
As specified in the product approval issued by the Ministry of Food and Drug Safety, the study will be conducted for 4 years from the approval date. At least 600 study subjects will be enrolled in this study to meet the MFDS requirements. Although 600 is the assigned number of study subjects, the number of cases will be adjusted considering the actual number of enrolled subjects after the study start day.
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| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | An AE was any untoward medical occurrence in participants who received Xyntha without regard to possibility of causal relationship. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment Emergent Adverse Event (TEAE) was adverse event that started or worsened in severity after first administration of Xyntha Solofuse up to 6 months. AEs included both serious and non-serious adverse event. | From the first administration of Xyntha up to 6 months |
| Number of Participants With Adverse Events (AEs) by Severity | An AE was any untoward medical occurrence in participants who received Xyntha without regard to possibility of causal relationship. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs were classified according to the severity in 3 categories a) mild - AEs does not interfere with participant's usual function b) moderate - AEs interferes to some extent with participant's usual function c) severe - AEs interferes significantly with participant's usual function, and was determined based on investigator's discretion. | From the first administration of Xyntha up to 6 months |
| Number of Participants Who Discontinued Due to Adverse Events | An AE was any untoward medical occurrence in participants who received study drug without regard to possibility of causal relationship. | From the first administration of Xyntha up to 6 months |
| Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events | Treatment-related AE was any untoward medical occurrence attributed to Xyntha in a participant who received study drug. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Relatedness to treatment was assessed by investigator. AEs included both serious and non-serious AEs. |
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-. Inclusion criteria
To be eligible to enroll in this study, the study subjects will have to meet all the following inclusion criteria:
Patients who satisfy the following criteria are not included in the study according to the local labeling:
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The study population is Hemophilia A (congenital factor VIII deficiency) patients who have been administered the Xyntha Solofuse prefilled syringe (as part of routine treatment at the Korean health care canter which has certified investigators).
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| Name | Affiliation | Role |
|---|---|---|
| Pfizer CT.gov Call Center | Pfizer | Study Director |
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| Label | URL |
|---|---|
| To obtain contact information for a study center near you, click here. | View source |
| To obtain contact information for a study center near you, click here. | View source |
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Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical\_trials/trial\_data\_and\_results/data\_requests.
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| ID | Title | Description |
|---|---|---|
| FG000 | Xyntha | Participants who were prescribed with Xyntha Solofuse prefilled syringe, as part of routine treatment, were observed in this study for up to 6 months from the initial administration of Xyntha Solofuse. |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
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Analysis was performed on all participants who received at least 1 dose of Xyntha solofuse prefilled syringe.
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| ID | Title | Description |
|---|---|---|
| BG000 | Xyntha | Participants who were prescribed with Xyntha Solofuse prefilled syringe, as part of routine treatment, were observed in this study for up to 6 months from the initial administration of Xyntha Solofuse. |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Customized | Count of Participants |
| 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 | Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) | An AE was any untoward medical occurrence in participants who received Xyntha without regard to possibility of causal relationship. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Treatment Emergent Adverse Event (TEAE) was adverse event that started or worsened in severity after first administration of Xyntha Solofuse up to 6 months. AEs included both serious and non-serious adverse event. | The safety population included all participants who received at least one dose of Xyntha Solofuse prefilled syringe. | Posted | Count of Participants | Participants | From the first administration of Xyntha up to 6 months |
|
From the first administration of Xyntha Solofuse prefilled syringe up to 6 months
Same event may appear as both an AE and SAE. However, what is presented are distinct events. An event may be categorized as serious in one participant and as non-serious in another, or a participant may have experienced both a serious and non-serious event. Analysis performed on safety analysis set.
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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 | Xyntha | Participants who were prescribed with Xyntha Solofuse prefilled syringe, as part of routine treatment, were observed in this study for up to 6 months from the initial administration of Xyntha Solofuse. |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Fever | General disorders | WHO-ART 092 | Non-systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Pfizer ClinicalTrials.gov Call Center | Pfizer Inc. | 1-800-718-1021 | ClinicalTrials.gov_Inquiries@pfizer.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | May 17, 2018 | Jan 7, 2019 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 20, 2018 | Jan 7, 2019 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D006467 | Hemophilia A |
| ID | Term |
|---|---|
| D025861 | Blood Coagulation Disorders, Inherited |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| From the first administration of Xyntha up to 6 months |
| Number of Participants Who Died Due to Adverse Events | An AE was any untoward medical occurrence in participants who received Xyntha without regard to possibility of causal relationship. | From the first administration of Xyntha up to 6 months |
| Number of Participants With Overall Responses on a 4-Point Scale to the Injections Used to Treat Bleeding: On-Demand Treatment According to Surgery | On-demand treatment according to surgery: treatment to increase factor in preparation for surgery. Overall responses to all injection of Xyntha Solofuse prefilled syringe used to treat bleeding in on-demand treatment (administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) according to surgery was assessed on 4 point scale of 1=excellent, 2=good, 3=moderate and 4=no response, higher score = better response. Excellent= Definite pain relief and/or improvement in signs of bleeding within 8 hours after an infusion, with no additional infusion, good= Definite pain relief and/or improvement in signs of bleeding within 8 hours after an infusion, with 1 additional infusion, moderate= Probable/slight improvement starting after 8 hours following infusion, with >=1 additional infusion administered for complete resolution of bleeding episode and no response= No improvement at all between infusions/during 24 hour interval following an infusion/condition worsens | From the first administration of Xyntha up to 6 months |
| Number of Participants With Overall Responses on a 4-Point Scale to the Injections Used to Treat Bleeding: On-Demand Treatment According to Bleeding | On-demand treatment according to bleeding: treatment administered for spontaneous bleeding/abrasion; not requiring surgery. Overall responses to all injection of Xyntha used to treat bleeding in on-demand treatment (administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) according to bleeding was assessed on 4 point scale of 1=excellent, 2=good, 3=moderate,4=no response, higher score=better response. Excellent= Definite pain relief and/or improvement in signs of bleeding within 8 hours after an infusion, with no additional infusion, good= Definite pain relief and/or improvement in signs of bleeding within 8 hours after an infusion, with 1 additional infusion, moderate= Probable/slight improvement after 8 hours following infusion, with >=1 additional infusion administered for complete resolution of bleeding episode and no response= No improvement at all between infusions/during 24 hour interval following an infusion, or condition worsens. | From the first administration of Xyntha up to 6 months |
| Number of Participants With Less Than Expected Therapeutic Effect (LETE): On-Demand Treatment According to Surgery | LETE for on-demand treatment (administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) was defined as "no response" rated after each infusion of 2 consecutive infusions within 24 hours after on-demand treatment. | Within 24 hours of on-demand treatment (anytime within the observation period of 6 months) |
| Number of Participants With Less Than Expected Therapeutic Effect (LETE): On-Demand Treatment According to Bleeding | LETE for on-demand treatment ((administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) was defined as "no response" rated after each infusion of 2 consecutive infusions within 24 hours after on-demand treatment) was defined as "no response" rated after each infusion of 2 consecutive infusions within 24 hours after on-demand treatment. | Within 24 hours of on-demand treatment (anytime within the observation period of up to 6 months) |
| Number of Infusions Required to Treat Each New Bleeding Episode | It was calculated as total number of injections given throughout the study divided by total number of bleeding events. | From the first administration of Xyntha up to 6 months |
| Average Dose of Infusions Per Bleeding Event: On-Demand Treatment According to Surgery | The average dose of Xyntha per bleeding event according to surgery in on-demand treatment was calculated as total dose of Xyntha (in IU) throughout the study divided by total number of bleeding event. On-demand treatment (administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) according to surgery means treatment to increase factor in preparation for surgery. | From the first administration of Xyntha up to 6 months |
| Average Dose of Infusions Per Bleeding Event: On-Demand Treatment According to Bleeding | The average dose of Xyntha per bleeding event was calculated as total dose of Xyntha throughout the study (in International Units [IU]) divided by total number of bleeding incidence. On-demand treatment (administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) according to bleeding means treatment administered due to spontaneous bleeding or abrasion. | From the first administration of Xyntha up to 6 months |
| Percentage of Participants With Bleeding Event | From the first administration of Xyntha up to 6 months |
| Annualized Bleeding Rates (ABRs) | Annualized bleeding rate defined as number of bleeds under prophylactic setting (defined as bleeding occurred after 48 hours of prophylactic therapy [administration of Xyntha not for the treatment of a bleed but for the prevention of bleeding]) divided by (/) [(number of prophylactic therapy participants)*0.5)] | Within 48 hours after the prophylactic administration of Xyntha (within the duration of 6 months) |
| Number of Participants With Less Than Expected Therapeutic Effect (LETE): Prophylactic Therapy | Less than expected therapeutic effect for prophylaxis therapy defined as breakthrough (spontaneous/non-traumatic) bleeding occurred within 48 hours of prophylaxis infusion (defined as: administration of Xyntha not for the treatment of a bleed but for the prevention of bleeding). | From the first administration of Xyntha up to 6 months |
| Average Dose of Infusions Per Bleeding Event: Prophylactic Therapy | The average dose of Xyntha per bleeding event according to prophylaxis therapy treatment was calculated as total dose of Xyntha (in IU) throughout the study divided by total number of bleeding event. Prophylaxis therapy defined as breakthrough (spontaneous/non-traumatic) bleeding occurred within 48 hours of prophylaxis infusion (defined as administration of Xyntha not for the treatment of a bleed but for the prevention of bleeding). | From the first administration of Xyntha up to 6 months |
| Total Factor VIII Consumption | Total factor VIII consumption for each participant was calculated by sum of the total amount of Xyntha Solofuse (in IU) infused for each Xyntha Solofuse infusion. | 6 months |
| Participants |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race and Ethnicity Not Collected | Race and Ethnicity were not collected from any participant. | Race and Ethnicity were not collected from any participant. | Count of Participants | Participants |
|
| Duration of the disease, Continuous | Duration of Hemophilia A in the study participants is provided in this baseline measure. | Mean | Standard Deviation | Years |
|
| Genetic mutation of factor VIII | Presence of Genetic mutation of factor VIII was analyzed and provided in the categories of Yes (Present), No (not present) and Unknown. | Count of Participants | Participants |
|
| Previous exposure to plasma-derived factor VIII | Previous exposure of participants (before enrollment into the study) to plasma-derived factor VIII was determined and provided by using the categories as Yes (had exposure), No (did not have exposure) and Unknown. | Count of Participants | Participants |
|
| Previous used factor VIII therapy | Previous (before enrollment into the study) use of factor VIII therapy by participants was determined and provided by using the categories as Yes (had exposure) and No (did not have exposure). | Count of Participants | Participants |
|
| Personal history of allergic reactions to factor VIII products | Personal history of allergic reactions to factor VIII products was analyzed and provided as in the categories of 'Yes' (had allergy) and 'No (did not have allergy)'. | Count of Participants | Participants |
|
| Family history of hemophilia A | History of hemophilia A in the family of participants was determined and provided as in the categories of 'Yes' (had history of hemophilia disease) and 'No' (did not have history of hemophilia disease). | Count of Participants | Participants |
|
| Family history of factor VIII inhibitors | History of factor VIII inhibitors in the family of participants was determined and provided as in the categories of 'Yes' (had history of factor VIII inhibitors) and 'No' (did not have history of factor VIII inhibitors). | Count of Participants | Participants |
|
| Family history of allergic reactions to factor VIII products | History of allergic reactions to factor VIII products in the family of participants was determined and provided as in the categories of 'Yes' (had history of allergy) and 'No' (did not have allergy). | Count of Participants | Participants |
|
| Disease severity | Disease Severity was determined based on activity of coagulation factor and was categorized into different level of categories which included as Mild (5 percent [%] to 40%), Moderate (1 to 5%) and Severe (less than [<] 1%). | Count of Participants | Participants |
|
| Medical history | Medical history of hemophilia A in the family of participants was determined and provided as in the categories of 'Yes' (had history of hemophilia A) and 'No' (did not have history of hemophilia A). | Count of Participants | Participants |
|
| Renal Disorder | Participants with renal disorder was determined and provided as in the categories of 'Yes' (had renal disorder besides Haemophilia A) and 'No'(did not have renal disorder). | Count of Participants | Participants |
|
| Hepatic Disorder | Participants with hepatic disorder was determined and provided as in the categories of 'Yes' (had hepatic disorder besides Haemophilia A) and 'No' (did not have hepatic disorder). | Count of Participants | Participants |
|
| Personal History of Allergic Reactions to Factor VIII Products (within 12 months) | Personal history of allergic reactions of participants to factor VIII products was analyzed and provided as in the categories of 'Yes' (had allergy) and 'No (did not had allergy) | Count of Participants | Participants |
|
| Concomitant treatments | Concomitant treatments usage was determined in participants and provided here in the categories of 'Yes' (participants who had concomitant medication) and 'No (participants who did not have concomitant medication)'. Concomitant treatment included any other medicine/drug, which participants received other than Xyntha. | Count of Participants | Participants |
|
| Concomitant therapy | Concomitant therapy was determined in participants and provided here in the categories of 'Yes' (participants who had receive concomitant therapy) and 'No (participants who did not have received concomitant therapy)'. Concomitant therapy included any other therapy which participants were received other than study therapy. | Count of Participants | Participants |
|
Participants who were prescribed with Xyntha Solofuse prefilled syringe, as part of routine treatment, were observed in this study for up to 6 months from the initial administration of Xyntha Solofuse. |
|
|
| Primary | Number of Participants With Adverse Events (AEs) by Severity | An AE was any untoward medical occurrence in participants who received Xyntha without regard to possibility of causal relationship. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. AEs were classified according to the severity in 3 categories a) mild - AEs does not interfere with participant's usual function b) moderate - AEs interferes to some extent with participant's usual function c) severe - AEs interferes significantly with participant's usual function, and was determined based on investigator's discretion. | The safety population included all participants who received at least one dose of Xyntha Solofuse prefilled syringe. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Count of Participants | Participants | From the first administration of Xyntha up to 6 months |
|
|
|
| Primary | Number of Participants Who Discontinued Due to Adverse Events | An AE was any untoward medical occurrence in participants who received study drug without regard to possibility of causal relationship. | The safety population included all participants who received at least one dose of Xyntha Solofuse prefilled syringe. | Posted | Count of Participants | Participants | From the first administration of Xyntha up to 6 months |
|
|
|
| Primary | Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events | Treatment-related AE was any untoward medical occurrence attributed to Xyntha in a participant who received study drug. SAE was an AE resulting in any of the following outcomes or deemed significant for any other reason: death; initial or prolonged inpatient hospitalization; life-threatening experience (immediate risk of dying); persistent or significant disability/incapacity; congenital anomaly. Relatedness to treatment was assessed by investigator. AEs included both serious and non-serious AEs. | The safety population included all participants who received at least one dose of Xyntha Solofuse prefilled syringe. | Posted | Count of Participants | Participants | From the first administration of Xyntha up to 6 months |
|
|
|
| Primary | Number of Participants Who Died Due to Adverse Events | An AE was any untoward medical occurrence in participants who received Xyntha without regard to possibility of causal relationship. | The safety population included all participants who received at least one dose of Xyntha Solofuse prefilled syringe. | Posted | Count of Participants | Participants | From the first administration of Xyntha up to 6 months |
|
|
|
| Primary | Number of Participants With Overall Responses on a 4-Point Scale to the Injections Used to Treat Bleeding: On-Demand Treatment According to Surgery | On-demand treatment according to surgery: treatment to increase factor in preparation for surgery. Overall responses to all injection of Xyntha Solofuse prefilled syringe used to treat bleeding in on-demand treatment (administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) according to surgery was assessed on 4 point scale of 1=excellent, 2=good, 3=moderate and 4=no response, higher score = better response. Excellent= Definite pain relief and/or improvement in signs of bleeding within 8 hours after an infusion, with no additional infusion, good= Definite pain relief and/or improvement in signs of bleeding within 8 hours after an infusion, with 1 additional infusion, moderate= Probable/slight improvement starting after 8 hours following infusion, with >=1 additional infusion administered for complete resolution of bleeding episode and no response= No improvement at all between infusions/during 24 hour interval following an infusion/condition worsens | The efficacy analysis set included all participants who received at least one dose of Xyntha Solofuse prefilled syringe. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Count of Participants | Participants | From the first administration of Xyntha up to 6 months |
|
|
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| Primary | Number of Participants With Overall Responses on a 4-Point Scale to the Injections Used to Treat Bleeding: On-Demand Treatment According to Bleeding | On-demand treatment according to bleeding: treatment administered for spontaneous bleeding/abrasion; not requiring surgery. Overall responses to all injection of Xyntha used to treat bleeding in on-demand treatment (administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) according to bleeding was assessed on 4 point scale of 1=excellent, 2=good, 3=moderate,4=no response, higher score=better response. Excellent= Definite pain relief and/or improvement in signs of bleeding within 8 hours after an infusion, with no additional infusion, good= Definite pain relief and/or improvement in signs of bleeding within 8 hours after an infusion, with 1 additional infusion, moderate= Probable/slight improvement after 8 hours following infusion, with >=1 additional infusion administered for complete resolution of bleeding episode and no response= No improvement at all between infusions/during 24 hour interval following an infusion, or condition worsens. | The efficacy analysis set included all participants who received at least one dose of Xyntha Solofuse prefilled syringe. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Count of Participants | Participants | From the first administration of Xyntha up to 6 months |
|
|
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| Primary | Number of Participants With Less Than Expected Therapeutic Effect (LETE): On-Demand Treatment According to Surgery | LETE for on-demand treatment (administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) was defined as "no response" rated after each infusion of 2 consecutive infusions within 24 hours after on-demand treatment. | The efficacy analysis set included all participants who received at least one dose of Xyntha Solofuse prefilled syringe. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Count of Participants | Participants | Within 24 hours of on-demand treatment (anytime within the observation period of 6 months) |
|
|
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| Primary | Number of Participants With Less Than Expected Therapeutic Effect (LETE): On-Demand Treatment According to Bleeding | LETE for on-demand treatment ((administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) was defined as "no response" rated after each infusion of 2 consecutive infusions within 24 hours after on-demand treatment) was defined as "no response" rated after each infusion of 2 consecutive infusions within 24 hours after on-demand treatment. | The efficacy analysis set included all participants who received at least one dose of Xyntha Solofuse prefilled syringe. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Count of Participants | Participants | Within 24 hours of on-demand treatment (anytime within the observation period of up to 6 months) |
|
|
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| Primary | Number of Infusions Required to Treat Each New Bleeding Episode | It was calculated as total number of injections given throughout the study divided by total number of bleeding events. | The efficacy analysis set included all participants who received at least one dose of Xyntha Solofuse prefilled syringe. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | Infusions | From the first administration of Xyntha up to 6 months |
|
|
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| Primary | Average Dose of Infusions Per Bleeding Event: On-Demand Treatment According to Surgery | The average dose of Xyntha per bleeding event according to surgery in on-demand treatment was calculated as total dose of Xyntha (in IU) throughout the study divided by total number of bleeding event. On-demand treatment (administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) according to surgery means treatment to increase factor in preparation for surgery. | The efficacy analysis set included all participants who received at least one dose of Xyntha Solofuse prefilled syringe. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | IU/event | From the first administration of Xyntha up to 6 months |
|
|
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| Primary | Average Dose of Infusions Per Bleeding Event: On-Demand Treatment According to Bleeding | The average dose of Xyntha per bleeding event was calculated as total dose of Xyntha throughout the study (in International Units [IU]) divided by total number of bleeding incidence. On-demand treatment (administration of an unscheduled dose of Xyntha Solofuse prefilled syringe to stop bleeding) according to bleeding means treatment administered due to spontaneous bleeding or abrasion. | The efficacy analysis set included all participants who received at least one dose of Xyntha Solofuse prefilled syringe. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | IU/event | From the first administration of Xyntha up to 6 months |
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| Primary | Percentage of Participants With Bleeding Event | The efficacy analysis set included all participants who received at least one dose of Xyntha Solofuse prefilled syringe. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Number | percentage of participants | From the first administration of Xyntha up to 6 months |
|
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| Primary | Annualized Bleeding Rates (ABRs) | Annualized bleeding rate defined as number of bleeds under prophylactic setting (defined as bleeding occurred after 48 hours of prophylactic therapy [administration of Xyntha not for the treatment of a bleed but for the prevention of bleeding]) divided by (/) [(number of prophylactic therapy participants)*0.5)] | The efficacy analysis set included all participants who received at least one dose of Xyntha Solofuse prefilled syringe. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Number | bleeds per participant | Within 48 hours after the prophylactic administration of Xyntha (within the duration of 6 months) |
|
|
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| Primary | Number of Participants With Less Than Expected Therapeutic Effect (LETE): Prophylactic Therapy | Less than expected therapeutic effect for prophylaxis therapy defined as breakthrough (spontaneous/non-traumatic) bleeding occurred within 48 hours of prophylaxis infusion (defined as: administration of Xyntha not for the treatment of a bleed but for the prevention of bleeding). | The efficacy analysis set included all participants who received at least one dose of Xyntha Solofuse prefilled syringe. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Count of Participants | Participants | From the first administration of Xyntha up to 6 months |
|
|
|
| Primary | Average Dose of Infusions Per Bleeding Event: Prophylactic Therapy | The average dose of Xyntha per bleeding event according to prophylaxis therapy treatment was calculated as total dose of Xyntha (in IU) throughout the study divided by total number of bleeding event. Prophylaxis therapy defined as breakthrough (spontaneous/non-traumatic) bleeding occurred within 48 hours of prophylaxis infusion (defined as administration of Xyntha not for the treatment of a bleed but for the prevention of bleeding). | The efficacy analysis set included all participants who received at least one dose of Xyntha Solofuse prefilled syringe. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | IU/event | From the first administration of Xyntha up to 6 months |
|
|
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| Primary | Total Factor VIII Consumption | Total factor VIII consumption for each participant was calculated by sum of the total amount of Xyntha Solofuse (in IU) infused for each Xyntha Solofuse infusion. | The efficacy analysis set included all participants who received at least one dose of Xyntha Solofuse prefilled syringe. Here, "Overall Number of Participants Analyzed" signifies those participants who were evaluable for this outcome measure. | Posted | Mean | Standard Deviation | IU | 6 months |
|
|
|
| 22 |
| 105 |
| 0 |
| 105 |
| 15 |
| 105 |
| Hypertension | Cardiac disorders | WHO-ART 092 | Non-systematic Assessment |
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| Dizziness | Nervous system disorders | WHO-ART 092 | Non-systematic Assessment |
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| Dyspepsia | Gastrointestinal disorders | WHO-ART 092 | Non-systematic Assessment |
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| Factor VIII antibody positive | Vascular disorders | WHO-ART 092 | Non-systematic Assessment |
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| Haemorrhage NOS | Vascular disorders | WHO-ART 092 | Non-systematic Assessment |
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| Periodontal destruction | Gastrointestinal disorders | WHO-ART v 92 | Non-systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | WHO-ART v 92 | Non-systematic Assessment |
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| Myalgia | Musculoskeletal and connective tissue disorders | WHO-ART v 92 | Non-systematic Assessment |
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| Skeletal Pain | Musculoskeletal and connective tissue disorders | WHO-ART v 92 | Non-systematic Assessment |
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| Sepsis | Infections and infestations | WHO-ART v 92 | Non-systematic Assessment |
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| Pharyngitis | Respiratory, thoracic and mediastinal disorders | WHO-ART v 92 | Non-systematic Assessment |
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| Haematuria | Renal and urinary disorders | WHO-ART v 92 | Non-systematic Assessment |
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| Renal calculus | Renal and urinary disorders | WHO-ART v 92 | Non-systematic Assessment |
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| Dermatitis fungal | Skin and subcutaneous tissue disorders | WHO-ART v 92 | Non-systematic Assessment |
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| Laceration | Skin and subcutaneous tissue disorders | WHO-ART v 92 | Non-systematic Assessment |
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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 |
|
| Severe |
|
|
| Title | Measurements |
|---|
|
| No response |
|
| Title | Measurements |
|---|
|
| No response |
|