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Post-Marketing Surveillance To Observe Safety And Efficacy Of Xyntha Solofuse Prefilled Syringe

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03034044
Recruitment Status : Completed
First Posted : January 27, 2017
Results First Posted : April 5, 2019
Last Update Posted : April 5, 2019
Sponsor:
Information provided by (Responsible Party):
Pfizer

Study Type Observational
Study Design Observational Model: Case-Only;   Time Perspective: Retrospective
Conditions Factor VIII Deficiency, Congenital
Factor 8 Deficiency, Congenital
Autosomal Hemophilia A
Classic Hemophilia
Hemophilia A, Congenital
Enrollment 106
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Xyntha
Hide Arm/Group Description 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.
Period Title: Overall Study
Started 106
Received Xyntha 105
Completed 105
Not Completed 1
Reason Not Completed
Enrolled, but not received Xyntha             1
Arm/Group Title Xyntha
Hide Arm/Group Description 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.
Overall Number of Baseline Participants 105
Hide Baseline Analysis Population Description
Analysis was performed on all participants who received at least 1 dose of Xyntha solofuse prefilled syringe.
Age, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
< 30 years Number Analyzed 105 participants
19
  18.1%
30- 39 years Number Analyzed 105 participants
29
  27.6%
40- 49 years Number Analyzed 105 participants
39
  37.1%
50- 59 years Number Analyzed 105 participants
11
  10.5%
>= 60 years Number Analyzed 105 participants
7
   6.7%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 105 participants
Female
0
   0.0%
Male
105
 100.0%
Race and Ethnicity Not Collected   [1] [2] 
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 0 participants
[1]
Measure Description: Race and Ethnicity were not collected from any participant.
[2]
Measure Analysis Population Description: Race and Ethnicity were not collected from any participant.
Duration of the disease, Continuous   [1] 
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 105 participants
32.4  (16.9)
[1]
Measure Description: Duration of Hemophilia A in the study participants is provided in this baseline measure.
Genetic mutation of factor VIII   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Yes Number Analyzed 105 participants
0
   0.0%
No Number Analyzed 105 participants
36
  34.3%
Unknown Number Analyzed 105 participants
69
  65.7%
[1]
Measure Description: Presence of Genetic mutation of factor VIII was analyzed and provided in the categories of Yes (Present), No (not present) and Unknown.
Previous exposure to plasma-derived factor VIII   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Yes Number Analyzed 105 participants
51
  48.6%
No Number Analyzed 105 participants
34
  32.4%
Unknown Number Analyzed 105 participants
20
  19.0%
[1]
Measure Description: 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.
Previous used factor VIII therapy   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Yes Number Analyzed 105 participants
93
  88.6%
No Number Analyzed 105 participants
12
  11.4%
[1]
Measure Description: 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).
Personal history of allergic reactions to factor VIII products   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Yes Number Analyzed 105 participants
0
   0.0%
No Number Analyzed 105 participants
105
 100.0%
[1]
Measure Description: 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)'.
Family history of hemophilia A   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Yes Number Analyzed 105 participants
23
  21.9%
No Number Analyzed 105 participants
82
  78.1%
[1]
Measure Description: 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).
Family history of factor VIII inhibitors   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Yes Number Analyzed 105 participants
0
   0.0%
No Number Analyzed 105 participants
105
 100.0%
[1]
Measure Description: 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).
Family history of allergic reactions to factor VIII products   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Yes Number Analyzed 105 participants
0
   0.0%
No Number Analyzed 105 participants
105
 100.0%
[1]
Measure Description: 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).
Disease severity   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Mild Number Analyzed 105 participants
13
  12.4%
Moderate Number Analyzed 105 participants
8
   7.6%
Severe Number Analyzed 105 participants
80
  76.2%
Unknown Number Analyzed 105 participants
4
   3.8%
[1]
Measure Description: 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%).
Medical history   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Yes Number Analyzed 105 participants
45
  42.9%
No Number Analyzed 105 participants
60
  57.1%
[1]
Measure Description: 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).
Renal Disorder   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Yes Number Analyzed 105 participants
2
   1.9%
No Number Analyzed 105 participants
103
  98.1%
[1]
Measure Description: 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).
Hepatic Disorder   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Yes Number Analyzed 105 participants
17
  16.2%
No Number Analyzed 105 participants
88
  83.8%
[1]
Measure Description: 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).
Personal History of Allergic Reactions to Factor VIII Products (within 12 months)   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Yes Number Analyzed 105 participants
2
   1.9%
No Number Analyzed 105 participants
103
  98.1%
[1]
Measure Description: 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)
Concomitant treatments   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Yes Number Analyzed 105 participants
36
  34.3%
No Number Analyzed 105 participants
69
  65.7%
[1]
Measure Description: 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.
Concomitant therapy   [1] 
Measure Type: Count of Participants
Unit of measure:  Participants
Yes Number Analyzed 105 participants
2
   1.9%
No Number Analyzed 105 participants
103
  98.1%
[1]
Measure Description: 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.
1.Primary Outcome
Title Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Hide Description 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.
Time Frame From the first administration of Xyntha up to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population included all participants who received at least one dose of Xyntha Solofuse prefilled syringe.
Arm/Group Title Xyntha
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 105
Measure Type: Count of Participants
Unit of Measure: Participants
AEs
15
  14.3%
SAEs
0
   0.0%
2.Primary Outcome
Title Number of Participants With Adverse Events (AEs) by Severity
Hide Description 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.
Time Frame From the first administration of Xyntha up to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Xyntha
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 105
Measure Type: Count of Participants
Unit of Measure: Participants
Mild Number Analyzed 15 participants
10
  66.7%
Moderate Number Analyzed 15 participants
5
  33.3%
Severe Number Analyzed 15 participants
0
   0.0%
3.Primary Outcome
Title Number of Participants Who Discontinued Due to Adverse Events
Hide Description An AE was any untoward medical occurrence in participants who received study drug without regard to possibility of causal relationship.
Time Frame From the first administration of Xyntha up to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population included all participants who received at least one dose of Xyntha Solofuse prefilled syringe.
Arm/Group Title Xyntha
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 105
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
4.Primary Outcome
Title Number of Participants With Treatment-Related Adverse Events (AEs) and Serious Adverse Events
Hide Description 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.
Time Frame From the first administration of Xyntha up to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population included all participants who received at least one dose of Xyntha Solofuse prefilled syringe.
Arm/Group Title Xyntha
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 105
Measure Type: Count of Participants
Unit of Measure: Participants
AEs
2
   1.9%
SAEs
0
   0.0%
5.Primary Outcome
Title Number of Participants Who Died Due to Adverse Events
Hide Description An AE was any untoward medical occurrence in participants who received Xyntha without regard to possibility of causal relationship.
Time Frame From the first administration of Xyntha up to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
The safety population included all participants who received at least one dose of Xyntha Solofuse prefilled syringe.
Arm/Group Title Xyntha
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 105
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
6.Primary Outcome
Title Number of Participants With Overall Responses on a 4-Point Scale to the Injections Used to Treat Bleeding: On-Demand Treatment According to Surgery
Hide Description 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
Time Frame From the first administration of Xyntha up to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Xyntha
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Measure Type: Count of Participants
Unit of Measure: Participants
Excellent
6
 100.0%
Good
0
   0.0%
Moderate
0
   0.0%
No response
0
   0.0%
7.Primary Outcome
Title Number of Participants With Overall Responses on a 4-Point Scale to the Injections Used to Treat Bleeding: On-Demand Treatment According to Bleeding
Hide Description 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.
Time Frame From the first administration of Xyntha up to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Xyntha
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 31
Measure Type: Count of Participants
Unit of Measure: Participants
Excellent
28
  90.3%
Good
3
   9.7%
Moderate
0
   0.0%
No response
0
   0.0%
8.Primary Outcome
Title Number of Participants With Less Than Expected Therapeutic Effect (LETE): On-Demand Treatment According to Surgery
Hide Description 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.
Time Frame Within 24 hours of on-demand treatment (anytime within the observation period of 6 months)
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Xyntha
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
9.Primary Outcome
Title Number of Participants With Less Than Expected Therapeutic Effect (LETE): On-Demand Treatment According to Bleeding
Hide Description 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.
Time Frame Within 24 hours of on-demand treatment (anytime within the observation period of up to 6 months)
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Xyntha
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 31
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
10.Primary Outcome
Title Number of Infusions Required to Treat Each New Bleeding Episode
Hide Description It was calculated as total number of injections given throughout the study divided by total number of bleeding events.
Time Frame From the first administration of Xyntha up to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Xyntha
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 31
Mean (Standard Deviation)
Unit of Measure: Infusions
1.22  (0.57)
11.Primary Outcome
Title Average Dose of Infusions Per Bleeding Event: On-Demand Treatment According to Surgery
Hide Description 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.
Time Frame From the first administration of Xyntha up to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Xyntha
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 6
Mean (Standard Deviation)
Unit of Measure: IU/event
2619.29  (1089.07)
12.Primary Outcome
Title Average Dose of Infusions Per Bleeding Event: On-Demand Treatment According to Bleeding
Hide Description 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.
Time Frame From the first administration of Xyntha up to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Xyntha
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 31
Mean (Standard Deviation)
Unit of Measure: IU/event
2140.08  (640.32)
13.Primary Outcome
Title Percentage of Participants With Bleeding Event
Hide Description [Not Specified]
Time Frame From the first administration of Xyntha up to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Xyntha
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 90
Measure Type: Number
Unit of Measure: percentage of participants
6.67
14.Primary Outcome
Title Annualized Bleeding Rates (ABRs)
Hide Description 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)]
Time Frame Within 48 hours after the prophylactic administration of Xyntha (within the duration of 6 months)
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Xyntha
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 90
Measure Type: Number
Unit of Measure: bleeds per participant
13.33
15.Primary Outcome
Title Number of Participants With Less Than Expected Therapeutic Effect (LETE): Prophylactic Therapy
Hide Description 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).
Time Frame From the first administration of Xyntha up to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Xyntha
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 90
Measure Type: Count of Participants
Unit of Measure: Participants
6
   6.7%
16.Primary Outcome
Title Average Dose of Infusions Per Bleeding Event: Prophylactic Therapy
Hide Description 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).
Time Frame From the first administration of Xyntha up to 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Xyntha
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 90
Mean (Standard Deviation)
Unit of Measure: IU/event
1881.33  (531.88)
17.Primary Outcome
Title Total Factor VIII Consumption
Hide Description 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.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
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.
Arm/Group Title Xyntha
Hide Arm/Group Description:
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.
Overall Number of Participants Analyzed 90
Mean (Standard Deviation)
Unit of Measure: IU
106116.16  (55747.74)
Time Frame From the first administration of Xyntha Solofuse prefilled syringe up to 6 months
Adverse Event Reporting Description 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.
 
Arm/Group Title Xyntha
Hide Arm/Group Description 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.
All-Cause Mortality
Xyntha
Affected / at Risk (%)
Total   22/105 (20.95%) 
Hide Serious Adverse Events
Xyntha
Affected / at Risk (%)
Total   0/105 (0.00%) 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Xyntha
Affected / at Risk (%)
Total   15/105 (14.29%) 
Cardiac disorders   
Hypertension * 1  2/105 (1.90%) 
Gastrointestinal disorders   
Dyspepsia * 1  3/105 (2.86%) 
Periodontal destruction * 2  1/105 (0.95%) 
General disorders   
Fever * 1  1/105 (0.95%) 
Infections and infestations   
Sepsis * 2  1/105 (0.95%) 
Musculoskeletal and connective tissue disorders   
Arthralgia * 2  1/105 (0.95%) 
Myalgia * 2  1/105 (0.95%) 
Skeletal Pain * 2  1/105 (0.95%) 
Nervous system disorders   
Dizziness * 1  1/105 (0.95%) 
Renal and urinary disorders   
Haematuria * 2  1/105 (0.95%) 
Renal calculus * 2  1/105 (0.95%) 
Respiratory, thoracic and mediastinal disorders   
Pharyngitis * 2  4/105 (3.81%) 
Skin and subcutaneous tissue disorders   
Dermatitis fungal * 2  1/105 (0.95%) 
Laceration * 2  1/105 (0.95%) 
Vascular disorders   
Factor VIII antibody positive * 1  1/105 (0.95%) 
Haemorrhage NOS * 1  1/105 (0.95%) 
1
Term from vocabulary, WHO-ART 092
2
Term from vocabulary, WHO-ART v 92
*
Indicates events were collected by non-systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Pfizer ClinicalTrials.gov Call Center
Organization: Pfizer Inc.
Phone: 1-800-718-1021
EMail: ClinicalTrials.gov_Inquiries@pfizer.com
Layout table for additonal information
Responsible Party: Pfizer
ClinicalTrials.gov Identifier: NCT03034044    
Other Study ID Numbers: B1831086
First Submitted: November 30, 2016
First Posted: January 27, 2017
Results First Submitted: January 7, 2019
Results First Posted: April 5, 2019
Last Update Posted: April 5, 2019