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Study Evaluating On-Demand Treatment Of Xyntha In Chinese Subjects

This study has been completed.
Sponsor:
Information provided by:
Wyeth is now a wholly owned subsidiary of Pfizer
ClinicalTrials.gov Identifier:
NCT00868530
First received: March 19, 2009
Last updated: April 1, 2011
Last verified: April 2011
History of Changes
Results First Received: December 3, 2010  
Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Hemophilia A
Intervention: Biological: Xyntha

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Participants were recruited in China from September 2008 to December 2009.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Xyntha Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator’s prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.

Participant Flow:   Overall Study
    Xyntha  
STARTED     53  
COMPLETED     49  
NOT COMPLETED     4  
Adverse Event                 3  
Lost to Follow-up                 1  



  Baseline Characteristics
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Reporting Groups
  Description
Xyntha Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator’s prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.

Baseline Measures
    Xyntha  
Number of Participants  
[units: participants]
 		  53  
Age  
[units: Years]
Mean ± Standard Deviation 		  23.2  ± 10.0  
Gender  
[units: Participants]
 		 
Female     0  
Male     53  



  Outcome Measures
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1.  Primary:   Investigator Hemostatic Efficacy Assessment 8 Hours Post Infusion   [ Time Frame: 8 hours post infusion ]

Measure Type Primary
Measure Title Investigator Hemostatic Efficacy Assessment 8 Hours Post Infusion
Measure Description The Investigator Hemostatic Efficacy Assessment was based on a 4-point rating scale (Excellent = 1: definite pain relief or improvement in signs of bleeding, with no additional infusion, Good = 2: definite pain relief or improvement in signs of bleeding, Moderate = 3: probable or slight improvement, No Response = 4: no improvement at all between infusions).
Time Frame 8 hours post infusion  
Safety Issue No  

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
The Full Analysis Set (FAS) consisted of all participants who were treated and had at least 1 evaluable efficacy assessment after treatment.

Reporting Groups
  Description
Xyntha Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator’s prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.

Measured Values
    Xyntha  
Number of Participants Analyzed  
[units: participants]
 		  51  
Investigator Hemostatic Efficacy Assessment 8 Hours Post Infusion  
[units: Units on a scale]
Mean ± Standard Deviation 		  1.86  ± 0.65  

No statistical analysis provided for Investigator Hemostatic Efficacy Assessment 8 Hours Post Infusion



2.  Primary:   Investigator Hemostatic Efficacy Assessment 24 Hours Post Infusion   [ Time Frame: 24 hours post infusion ]

Measure Type Primary
Measure Title Investigator Hemostatic Efficacy Assessment 24 Hours Post Infusion
Measure Description The Investigator Hemostatic Efficacy Assessment was based on a 4-point rating scale (Excellent = 1: definite pain relief or improvement in signs of bleeding, with no additional infusion, Good = 2: definite pain relief or improvement in signs of bleeding, Moderate = 3: probable or slight improvement, No Response = 4: no improvement at all between infusions).
Time Frame 24 hours post infusion  
Safety Issue No  

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
The FAS consisted of all participants who were treated and had at least 1 evaluable efficacy assessment after treatment.

Reporting Groups
  Description
Xyntha Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator’s prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.

Measured Values
    Xyntha  
Number of Participants Analyzed  
[units: participants]
 		  51  
Investigator Hemostatic Efficacy Assessment 24 Hours Post Infusion  
[units: Units on a scale]
Mean ± Standard Deviation 		  1.74  ± 0.61  

No statistical analysis provided for Investigator Hemostatic Efficacy Assessment 24 Hours Post Infusion



3.  Primary:   Number of Participants With Factor VIII (FVIII) Inhibitor Development   [ Time Frame: Day 1 and Month 6 or Early Termination Visit ]

Measure Type Primary
Measure Title Number of Participants With Factor VIII (FVIII) Inhibitor Development
Measure Description Incidence of FVIII inhibitor was defined as any result determined as positive at local laboratory, and confirmed at central laboratory. Incidence was stratified by participant exposure history: Minimally Treated Patients (MTPs): those who had received at least 1 prior FVIII infusion, and <= 100 documented Exposure Days (EDs), while Previously Treated Patients (PTPs): those who had received >100 documented prior EDs. When number of prior EDs for an individual was not known to be at least 100, participants were included in the MTP population.
Time Frame Day 1 and Month 6 or Early Termination Visit  
Safety Issue Yes  

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
The FAS consisted of all participants who were treated and had at least 1 evaluable efficacy assessment after treatment.

Reporting Groups
  Description
Xyntha Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator’s prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.

Measured Values
    Xyntha  
Number of Participants Analyzed  
[units: participants]
 		  51  
Number of Participants With Factor VIII (FVIII) Inhibitor Development  
[units: Participants]
 		 
MTP (n=34)     6  
PTP (n=17)     1  

No statistical analysis provided for Number of Participants With Factor VIII (FVIII) Inhibitor Development



4.  Secondary:   FVIII Recovery : Change From Baseline in FVIII Concentration   [ Time Frame: Day 1 and Month 6 or Early Termination Visit ]

Measure Type Secondary
Measure Title FVIII Recovery : Change From Baseline in FVIII Concentration
Measure Description FVIII recovery was assessed by evaluating the change in FVIII concentration at 6 months compared to baseline.
Time Frame Day 1 and Month 6 or Early Termination Visit  
Safety Issue No  

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
The FAS consisted of all participants who were treated and had at least 1 evaluable efficacy assessment after treatment. Participants with missing data were not included.

Reporting Groups
  Description
Xyntha Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator’s prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.

Measured Values
    Xyntha  
Number of Participants Analyzed  
[units: participants]
 		  42  
FVIII Recovery : Change From Baseline in FVIII Concentration  
[units: IU/dL per IU/kg]
Mean ± Standard Deviation 		  -0.11  ± 0.45  


Statistical Analysis 1 for FVIII Recovery : Change From Baseline in FVIII Concentration
Groups [1] Xyntha
Method [2] t-test, 2 sided
P Value [3] 0.120
[1] Additional details about the analysis, such as null hypothesis and power calculation:
  No text entered.
[2] Other relevant information, such as adjustments or degrees of freedom:
  No text entered.
[3] Additional information, such as whether or not the p-value is adjusted for multiple comparisons and the a priori threshold for statistical significance:
  No text entered.



5.  Secondary:   Number of Participants With Less Than Expected Therapeutic Effect (LETE)   [ Time Frame: 24 hours after each of 2 successive infusion, up to 6 months ]

Measure Type Secondary
Measure Title Number of Participants With Less Than Expected Therapeutic Effect (LETE)
Measure Description The incidence of LETE, defined for on-demand treatment as no response after each of 2 successive infusions within 24 hours for the same bleeding event in the absence of confounding factors.
Time Frame 24 hours after each of 2 successive infusion, up to 6 months  
Safety Issue No  

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
The FAS consisted of all participants who were treated and had at least 1 evaluable efficacy assessment after treatment.

Reporting Groups
  Description
Xyntha Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator’s prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.

Measured Values
    Xyntha  
Number of Participants Analyzed  
[units: participants]
 		  51  
Number of Participants With Less Than Expected Therapeutic Effect (LETE)  
[units: Participants]
 		  0  

No statistical analysis provided for Number of Participants With Less Than Expected Therapeutic Effect (LETE)



6.  Secondary:   Number of Participants With Thrombosis Allergic-Type Reactions   [ Time Frame: Baseline up to 6 months ]

Measure Type Secondary
Measure Title Number of Participants With Thrombosis Allergic-Type Reactions
Measure Description No text entered.
Time Frame Baseline up to 6 months  
Safety Issue Yes  

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
The Safety Set (SS) consisted of all participants who had taken at least 1 dose of investigational drug.

Reporting Groups
  Description
Xyntha Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator’s prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.

Measured Values
    Xyntha  
Number of Participants Analyzed  
[units: participants]
 		  53  
Number of Participants With Thrombosis Allergic-Type Reactions  
[units: Participants]
 		  1  

No statistical analysis provided for Number of Participants With Thrombosis Allergic-Type Reactions



7.  Secondary:   Number of Participants With Thrombosis   [ Time Frame: Baseline up to 6 months ]

Measure Type Secondary
Measure Title Number of Participants With Thrombosis
Measure Description No text entered.
Time Frame Baseline up to 6 months  
Safety Issue Yes  

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
The SS consisted of all participants who had taken at least 1 dose of investigational drug.

Reporting Groups
  Description
Xyntha Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator’s prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.

Measured Values
    Xyntha  
Number of Participants Analyzed  
[units: participants]
 		  53  
Number of Participants With Thrombosis  
[units: Participants]
 		  0  

No statistical analysis provided for Number of Participants With Thrombosis



8.  Other Pre-specified:   Frequency of Xyntha Infusions Required Per Hemorrhage   [ Time Frame: Day 1 to Month 6 or Early Termination Visit ]

Measure Type Other Pre-specified
Measure Title Frequency of Xyntha Infusions Required Per Hemorrhage
Measure Description The mean frequency of Xyntha infusions per hemorrhage was calculated as total number of injections throughout the study divided by total number of hemorrhagic events.
Time Frame Day 1 to Month 6 or Early Termination Visit  
Safety Issue No  

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
The FAS consisted of all participants who were treated and had at least 1 evaluable efficacy assessment after treatment.

Reporting Groups
  Description
Xyntha Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator’s prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.

Measured Values
    Xyntha  
Number of Participants Analyzed  
[units: participants]
 		  51  
Frequency of Xyntha Infusions Required Per Hemorrhage  
[units: Infusions]
Mean ± Standard Deviation 		  1.16  ± 0.72  

No statistical analysis provided for Frequency of Xyntha Infusions Required Per Hemorrhage



9.  Other Pre-specified:   Average Dose of Xyntha Infusions Required Per Hemorrhage   [ Time Frame: Day 1 to Month 6 or Early Termination Visit ]

Measure Type Other Pre-specified
Measure Title Average Dose of Xyntha Infusions Required Per Hemorrhage
Measure Description The average dose of Xyntha per hemorrhagic event was calculated as total dose of Xyntha throughout the study (in IU) divided by total number of hemorrhage incidence.
Time Frame Day 1 to Month 6 or Early Termination Visit  
Safety Issue No  

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
The FAS consisted of all participants who were treated and had at least 1 evaluable efficacy assessment after treatment.

Reporting Groups
  Description
Xyntha Participants received on-demand treatments with Xyntha (which occurred each time a participant experienced bleeding episode during the active phase of the study) according to investigator’s prescription over a 6-month (calendar day) period. A single 50 International Unit (IU)/kg (+/-5 IU/kg) intravenous (IV) bolus infusion of Xyntha was given for recovery assessments.

Measured Values
    Xyntha  
Number of Participants Analyzed  
[units: participants]
 		  51  
Average Dose of Xyntha Infusions Required Per Hemorrhage  
[units: Dose/Bleed (IU)]
Mean ± Standard Deviation 		  1226.28  ± 1208.49  

No statistical analysis provided for Average Dose of Xyntha Infusions Required Per Hemorrhage




  Serious Adverse Events
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  Other Adverse Events
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  More Information
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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.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Limitations and Caveats
Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.  


Results Point of Contact:  
Name/Title: Pfizer ClinicalTrials.gov Call Center
Organization: Pfizer, Inc.
phone: 1-800-718-1021
e-mail: ClinicalTrials.gov_Inquiries@pfizer.com


No publications provided


Responsible Party: Director, Clinical Trial Disclosure Group, Pfizer, Inc
ClinicalTrials.gov Identifier: NCT00868530     History of Changes
Other Study ID Numbers: 3082B2-3316, B1831015
Study First Received: March 19, 2009
Results First Received: December 3, 2010
Last Updated: April 1, 2011
Health Authority: China: Food and Drug Administration