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BAX 326 Surgery Study in Hemophilia B Patients

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Baxalta US Inc.
ClinicalTrials.gov Identifier:
NCT01507896
First received: January 9, 2012
Last updated: December 22, 2016
Last verified: October 2016
Results First Received: July 27, 2016  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Hemophilia B
Intervention: Biological: Recombinant factor IX

  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
Enrollment was conducted at 10 clinical sites in 8 countries (Bulgaria, Czech Republic, Poland, Romania, Russia, Ukraine, Chile, Colombia).

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
30 unique participants enrolled for 41 surgical procedures, of which 1 participant discontinued before treatment with BAX326 but re-enrolled later for another surgical procedure. Note: a unique participant can undergo more than one surgical procedure.

Reporting Groups
  Description
Treatment With BAX326 Recombinant Factor IX (FIX): Following a loading dose with BAX326, participants received BAX326 as a bolus infusion. The treatment regimen was determined by the intensity and duration of the hemostatic challenge and the institution's standard of care. The dose was tailored to raise FIX concentration to at least 80%-100% of normal for major surgeries and to at least 30%-60% of normal for minor surgeries. Note: Treatment with BAX326 refers to unique participants treated with BAX326 which is less than the number of participants treated with BAX326 as unique participants could undergo more than one surgical procedure in this study. 30 unique participants were treated with BAX326 for 40 planned surgical procedures; of these, 2 unique participants were treated with BAX326 but did not undergo 2 surgical procedures (1 surgery per unique participant), therefore 28 unique participants underwent 38 surgical procedures.

Participant Flow:   Overall Study
    Treatment With BAX326
STARTED   30 [1] 
COMPLETED   28 [2] 
NOT COMPLETED   2 
Withdrawal by Subject                1 
The surgery was denied by the sponsor.                1 
[1] 30 unique participants had 40 surgeries planned (participants could have multiple surgeries).
[2] 28 unique participants had 38 surgeries (participants could have multiple surgeries).



  Baseline Characteristics
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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.
Note there were 30 unique participants with a total of 40 planned surgeries, as participants may have > 1 surgery. Baseline data reflects number of planned surgeries.

Reporting Groups
  Description
Treatment With BAX326 Recombinant Factor IX (FIX): Following a loading dose with BAX326, participants received BAX326 as a bolus infusion. The treatment regimen was determined by the intensity and duration of the hemostatic challenge and the institution's standard of care. The dose was tailored to raise FIX concentration to at least 80%-100% of normal for major surgeries and to at least 30%-60% of normal for minor surgeries. Note: Treatment with BAX326 refers to unique participants treated with BAX326 which is less than the number of participants treated with BAX326 as unique participants could undergo more than one surgical procedure in this study. 30 unique participants were treated with BAX326 for 40 planned surgical procedures; of these, 2 unique participants were treated with BAX326 but did not undergo 2 surgical procedures (1 surgery per unique participant), therefore 28 unique participants underwent 38 surgical procedures.

Baseline Measures
   Treatment With BAX326 
Overall Participants Analyzed 
[Units: Participants]
 40 
Age 
[Units: Years]
Mean (Standard Deviation)
 39.7  (11.2) 
Gender, Customized 
[Units: Planned surgeries]
 
Female   0 
Male   40 


  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Intraoperative Hemostatic Efficacy   [ Time Frame: On day of surgery ]

2.  Primary:   Actual Intraoperative Blood Loss   [ Time Frame: On day of surgery ]

3.  Primary:   Actual Intraoperative Blood Loss Compared to Average and Maximum Blood Loss Predicted Preoperatively by the Operating Surgeon   [ Time Frame: On day of surgery ]

4.  Primary:   Postoperative Hemostatic Efficacy at Drain Removal   [ Time Frame: At drain removal (from 1-3 days postoperatively) ]

5.  Primary:   Postoperative Hemostatic Efficacy at Postoperative Day 3   [ Time Frame: At postoperative day 3 (approximately 72 hours postoperatively) ]

6.  Primary:   Postoperative Hemostatic Efficacy on Day of Discharge   [ Time Frame: At discharge from hospital (from 1-3 days postoperatively for minor surgery and approximately 2 weeks postoperatively for major surgery) ]

7.  Primary:   Actual Postoperative Blood Loss   [ Time Frame: At drain removal (from 1-3 days postoperatively) ]

8.  Primary:   Actual Postoperative Blood Loss Compared to Average and Maximum Blood Loss Predicated Preoperatively by the Operating Surgeon   [ Time Frame: At postoperative day 3 (approximately 72 hours postoperatively) ]

9.  Primary:   Daily Weight-Adjusted Dose of BAX326 Per Participant   [ Time Frame: From initiation of surgery until discharge from hospital (from 1-3 days postoperatively for minor surgery and approximately 2 weeks postoperatively for major surgery) ]

10.  Primary:   Total Weight-Adjusted Dose of BAX326 Per Participant   [ Time Frame: From initiation of surgery until discharge from hospital (from 1-3 days postoperatively for minor surgery and approximately 2 weeks postoperatively for major surgery) ]

11.  Primary:   Number of Units of Blood Product Transfused   [ Time Frame: From initiation of surgery until discharge from hospital (from 1-3 days postoperatively for minor surgery and approximately 2 weeks postoperatively for major surgery) ]

12.  Primary:   Volume of Blood Product Transfused   [ Time Frame: From initiation of surgery until discharge from hospital (from 1-3 days postoperatively for minor surgery and approximately 2 weeks postoperatively for major surgery) ]

13.  Primary:   Safety: Number of Participants Who Developed Inhibitory Antibodies to Factor IX (FIX)   [ Time Frame: Throughout the study period (approximately 2 years 5 months) ]

14.  Primary:   Safety: Number of Participants Who Developed Total Binding Antibodies to Factor IX (FIX)   [ Time Frame: Throughout the study period (approximately 2 years 5 months) ]

15.  Primary:   Safety: Number of Adverse Events Related to BAX326   [ Time Frame: Throughout the study period (approximately 2 years 5 months) ]

16.  Primary:   Safety: Occurence of a Thrombotic Event   [ Time Frame: Throughout the study period (approximately 2 years 5 months) ]

17.  Primary:   Pre-Surgical Pharmacokinetics (PK): Area Under the Plasma Concentration Versus Time Curve (AUC) From 0 to 72 Hours Post-infusion Per Dose   [ Time Frame: Within 30 mins pre-infusion and post-infusion timepoints of 30 minutes, 6 hr, 24 hr, 48 hr and 72 hr ]

18.  Primary:   Pre-Surgical Pharmacokinetics (PK): Total Area Under the Plasma Concentration Versus Time Curve Per Dose (Total AUC/Dose)   [ Time Frame: Within 30 mins pre-infusion and post-infusion timepoints of 30 minutes, 6 hr, 24 hr, 48 hr and 72 hr ]

19.  Primary:   Pre-Surgical Pharmacokinetics (PK): Mean Residence Time (MRT)   [ Time Frame: Within 30 mins pre-infusion and post-infusion timepoints of 30 minutes, 6 hr, 24 hr, 48 hr and 72 hr ]

20.  Primary:   Pre-Surgical Pharmacokinetics (PK): Factor IX (FIX) Clearance (CL)   [ Time Frame: Within 30 mins pre-infusion and post-infusion timepoints of 30 minutes, 6 hr, 24 hr, 48 hr and 72 hr ]

21.  Primary:   Pre-Surgical Pharmacokinetics (PK): Incremental Recovery (IR) at 30 Min   [ Time Frame: Within 30 mins pre-infusion and post-infusion at 30 minutes ]

22.  Primary:   Pre-Surgical Pharmacokinetics (PK): Elimination Phase Half-life (T 1/2)   [ Time Frame: Within 30 mins pre-infusion and post-infusion timepoints of 30 minutes, 6 hr, 24 hr, 48 hr and 72 hr ]

23.  Primary:   Pre-Surgical Pharmacokinetics (PK): Volume of Distribution at Steady State (Vss)   [ Time Frame: Within 30 mins pre-infusion and post-infusion timepoints of 30 minutes, 6 hr, 24 hr, 48 hr and 72 hr ]

24.  Primary:   Incremental Recovery (IR) at 15±5 Minutes Following Loading Dose Prior to Surgery   [ Time Frame: Within 60 minutes prior to surgery and 15 ± 5 minutes after loading dose/rebolus, if applicable. ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
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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
Data provided for surgical procedures, treatments with BAX326 etc. and for unique participants (one unique participant could undergo more than one surgical procedure).


  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.


Results Point of Contact:  
Name/Title: Clinical Trial Registries and Results Disclosure
Organization: Baxalta US Inc
e-mail: ClinicalTrialsDisclosure@baxalta.com


Publications of Results:

Responsible Party: Baxalta US Inc.
ClinicalTrials.gov Identifier: NCT01507896     History of Changes
Other Study ID Numbers: 251002
2011-000413-39 ( EudraCT Number )
Study First Received: January 9, 2012
Results First Received: July 27, 2016
Last Updated: December 22, 2016