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BAX 326 (rFIX) Continuation Study

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ClinicalTrials.gov Identifier: NCT01286779
Recruitment Status : Completed
First Posted : January 31, 2011
Results First Posted : September 10, 2018
Last Update Posted : September 10, 2018
Sponsor:
Information provided by (Responsible Party):
Shire ( Baxalta now part of Shire )

Study Type: Interventional
Study Design: Allocation: Non-Randomized;   Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition: Hemophilia B
Intervention: Biological: BAX 326 (Recombinant factor IX)

  Participant Flow

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 40 clinical sites in 18 countries. A total of 117 participants were enrolled. Of these, 65 participants transitioned from BAX326 pivotal study, 20 participants transitioned from BAX326 pediatric study and 32 participants were newly recruited.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Of 117 enrolled participants, 115 received treatment with IP. All 85 participants who transitioned from the pivotal/pediatric studies continued to receive IP in this study. Of the 32 newly recruited participants, 30 received treatment with IP. 1 participant did not meet the entry criteria and 1 participant discontinued the study prior treatment.

Reporting Groups
  Description
BAX 326 Participants treated with BAX 326

Participant Flow:   Overall Study
    BAX 326
STARTED   115 
COMPLETED   96 
NOT COMPLETED   19 
Withdrawal by Subject                9 
Protocol Violation                5 
Physician Decision                2 
Participant had scheduled surgery                1 
Participant moved to another country                1 
Discontinued by sponsor                1 



  Baseline Characteristics

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.
No text entered.

Reporting Groups
  Description
BAX 326 Participants treated with BAX 326

Baseline Measures
   BAX 326 
Overall Participants Analyzed 
[Units: Participants]
 115 
Age 
[Units: Years]
Mean (Standard Deviation)
 29.6  (16.39) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
 
Female      0   0.0% 
Male      115 100.0% 
Race/Ethnicity, Customized 
[Units: Participants]
Count of Participants
 
Race   
White      99  86.1% 
Black or African American      1   0.9% 
Asian      10   8.7% 
Other      5   4.3% 


  Outcome Measures

1.  Primary:   Adverse Events Possibly or Probably Related to the Investigational Product   [ Time Frame: Assessed (based on patient diary) every 3 months until study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last). ]

2.  Secondary:   Treatment of Bleeding Episodes: Number of Infusions Per Bleeding Episode Required Until Bleed Resolution   [ Time Frame: Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last). ]

3.  Secondary:   Treatment of Bleeding Episodes: Overall Hemostatic Efficacy Rating at Resolution of Bleed   [ Time Frame: Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last). ]

4.  Secondary:   Annualized Bleed Rate During Prophylaxis Treatment   [ Time Frame: For prophylactic treatment the period from first to last prophylactic infusion is considered. ]

5.  Secondary:   Consumption of BAX 326: Number of Infusions Per Month and Per Year   [ Time Frame: Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last). ]

6.  Secondary:   Consumption of BAX 326: Weight Adjusted Consumption Per Month and Per Year   [ Time Frame: Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last). ]

7.  Secondary:   Consumption of BAX326: Weight Adjusted Consumption Per Bleeding Episode   [ Time Frame: Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last). ]

8.  Secondary:   Development of Inhibitory and Total Binding Antibodies to Factor IX   [ Time Frame: Laboratory assessment for immunology were done at screening, at exposure day 1, at week 4 (± 1 week), at month 3 (±1 week), thereafter, every 3 months (± 1 week) and at study completion/termination. ]

9.  Secondary:   Development of Antibodies to Chinese Hamster Ovary Proteins (CHO Proteins) and rFurin   [ Time Frame: Laboratory assessment for immunology were done at screening, at exposure day 1, at week 4 (± 1 week), at month 3 (±1 week), thereafter, every 3 months (± 1 week) and at study completion/termination. ]

10.  Secondary:   Occurrence of Severe Allergic Reactions and Thrombotic Events   [ Time Frame: Throughout the study from screening to study completion (when BAX326 is licensed in the respective country or the participant has accumulated approximately 100 exposure days to BAX 326, whichever is last). ]

11.  Secondary:   Clinical Significant Changes in Routine Laboratory Parameters and Vital Signs   [ Time Frame: Measurements at screening and at study completion/termination are included in the analysis. ]

12.  Secondary:   Pharmacokinetics: Incremental Recovery (IR) Over Time   [ Time Frame: IR over time was measured as Baseline and at Completion/Termination visit within 30 minutes pre-infusion and at 30 (± 5) minutes post-infusion. ]

13.  Secondary:   Pharmacokinetics: Area Under the Plasma Concentration Versus Time Curve From Time 0 to Infinity (AUC 0-∞)   [ Time Frame: PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours ]

14.  Secondary:   Pharmacokinetics: Elimination Phase Half-life (T1/2)   [ Time Frame: PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours ]

15.  Secondary:   Pharmacokinetics: Mean Residence Time (MRT)   [ Time Frame: PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours ]

16.  Secondary:   Pharmacokinetics: Systemic Clearance (CL)   [ Time Frame: PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours ]

17.  Secondary:   Pharmacokinetics: Volume of Distribution at Steady State (Vss)   [ Time Frame: PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours ]

18.  Secondary:   Pharmacokinetics: Incremental Recovery (IR)   [ Time Frame: PK assessments were done within 30 minutes pre-infusion and post-infusion at 30 (± 5) minutes, 9 hours (± 30 minutes), 24 (± 2) hours, 48 (± 2) hours and 72 (± 2) hours. ]

19.  Secondary:   Changes in Health Related Quality of Life (HR QoL) Based on Questionnaire SF-36   [ Time Frame: Baseline at exposure day 1 and at study completion/termination. ]

20.  Secondary:   Changes in Health Related Quality of Life Using the Peds QL   [ Time Frame: Baseline at exposure day 1 and at study completion/termination. ]

21.  Secondary:   Changes in Health Related Quality of Life (HR QoL) Based on Questionnaire Haemo-QoL and Haem-A-QoL   [ Time Frame: Baseline at exposure day 1 and at study completion/termination. ]

22.  Secondary:   Changes in Health Related Quality of Life (HR QoL) Based on Questionnaire EQ-5D and Pain Score.   [ Time Frame: Baseline at exposure day 1 and at study completion/termination. ]


  Serious Adverse Events


  Other Adverse Events


  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.


  More Information

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: Study Director
Organization: Baxalta, now part of Shire
phone: +1 866 842 5335
e-mail: ClinicalTransparency@shire.com



Responsible Party: Shire ( Baxalta now part of Shire )
ClinicalTrials.gov Identifier: NCT01286779     History of Changes
Other Study ID Numbers: 251001
2010-022726-33 ( EudraCT Number )
First Submitted: January 26, 2011
First Posted: January 31, 2011
Results First Submitted: June 21, 2018
Results First Posted: September 10, 2018
Last Update Posted: September 10, 2018