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Safety, Efficacy and Pharmacokinetics of NNC-0156-0000-0009 in Previously Treated Children With Haemophilia B. (paradigm™5)

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ClinicalTrials.gov Identifier: NCT01467427
Recruitment Status : Active, not recruiting
First Posted : November 8, 2011
Results First Posted : November 17, 2017
Last Update Posted : August 2, 2018
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions: Congenital Bleeding Disorder
Haemophilia B
Intervention: Drug: nonacog beta pegol

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Of the 19 sites that screened subjects, 17 sites enrolled subjects. The trial was therefore conducted at 17 sites in 8 countries, as follows: Canada: 1 site; Germany: 1 site; Italy: 1 site; Japan: 3 sites; Malaysia: 1 site; Taiwan: 1 site; United Kingdom: 3 sites; United States: 6 sites

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
Younger Children (0-6 Years) Subjects (0-6 years) received nonacog beta pegol 40 U/kg intravenous (i.v. once weekly for 52 weeks. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg i.v. Severe bleeding episodes were treated immediately with 80 U/kg.
Older Children (7-12 Years) Subjects (7-12 years) received nonacog beta pegol 40 U/kg intravenous (i.v. once weekly for 52 weeks. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg i.v. Severe bleeding episodes were treated immediately with 80 U/kg.

Participant Flow:   Overall Study
    Younger Children (0-6 Years)   Older Children (7-12 Years)
STARTED   12   13 
Completed Main Phase   11   13 
COMPLETED   0   0 
NOT COMPLETED   12   13 
Withdrawal by Subject                1                0 
Entered extension phase                11                11 
Not continuing in extension phase                0                2 



  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
Younger Children (0-6 Years) Subjects (0-6 years) received nonacog beta pegol 40 U/kg intravenous (i.v. once weekly for 52 weeks. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg i.v. Severe bleeding episodes were treated immediately with 80 U/kg.
Older Children (7-12 Years) Subjects (7-12 years) received nonacog beta pegol 40 U/kg intravenous (i.v. once weekly for 52 weeks. Bleeding episodes were treated as soon as they were identified. The dose for treatment of an uncomplicated mild/moderate bleeding episode, e.g. a joint bleed, was a single dose of nonacog beta pegol 40 U/kg i.v. Severe bleeding episodes were treated immediately with 80 U/kg.
Total Total of all reporting groups

Baseline Measures
   Younger Children (0-6 Years)   Older Children (7-12 Years)   Total 
Overall Participants Analyzed 
[Units: Participants]
 12   13   25 
Age 
[Units: Years]
Mean (Standard Deviation)
 3.1  (1.7)   9.6  (1.6)   6.5  (3.7) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
     
Female      0   0.0%      0   0.0%      0   0.0% 
Male      12 100.0%      13 100.0%      25 100.0% 


  Outcome Measures

1.  Primary:   Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU)   [ Time Frame: From 0 to 52 weeks ]

2.  Secondary:   Number of Bleeding Episodes During Prophylaxis   [ Time Frame: From 0 to 52 weeks ]

3.  Secondary:   Haemostatic Effect of N9-GP in Treatment of Bleeding Episodes by 4-point Categorical Scale for Haemostatic Response (Excellent, Good, Moderate and Poor)   [ Time Frame: From 0 to 52 weeks ]

4.  Secondary:   Incremental Recovery at 30 Minutes (IR30min)   [ Time Frame: Week 0 (30 minutes after first exposure) ]

5.  Secondary:   Trough Level (Single-dose )   [ Time Frame: Week 0 (one week after first exposure) ]

6.  Secondary:   Trough Level (Steady State)   [ Time Frame: Week 4 to 52 weeks ]

7.  Secondary:   Terminal Half-life (t1/2)   [ Time Frame: Week 0 (30 minutes until one week after first exposure) ]

8.  Primary:   Incidence of Inhibitory Antibodies Against Coagulation Factor IX (FIX) Defined as Titre Above or Equal to 0.6 Bethesda Units (BU)   [ Time Frame: From week 52 until the last patient has completed the trial (no later than 31-Oct-2018) ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

9.  Secondary:   Number of Bleeding Episodes During Prophylaxis   [ Time Frame: From week 52 until the last patient has completed the trial (no later than 31-Oct-2018) ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

10.  Secondary:   Trough Level (Steady State)   [ Time Frame: From week 52 until the patient has completed the trial (no later than 31-Oct-2018) ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  

11.  Secondary:   Haemostatic Effect of N9-GP in Treatment of Bleeding Episodes by 4-point Categorical Scale for Haemostatic Response (Excellent, Good, Moderate and Poor)   [ Time Frame: From week 52 until the last patient has completed the trial (no later than 31-Oct-2018) ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  


  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: Global Clinical Registry (GCR, 1452)
Organization: Novo Nordisk A/S
e-mail: clinicaltrials@novonordisk.com


Publications of Results:
Other Publications:
Safety, efficacy and pharmacokinetics of nonacog beta pegol (N9-GP) in prophylaxis and treatment of bleeding episodes in previously treated pediatric hemophilia B patients. Carcao M, Zak M, Abdul Karim F, Hanabusa H, Kearney S, Lu M-Y, Persson P, Rangarajan S, Santagostino E. Presented 06-Dec-2014 at the American Society of Hematology - 56th Annual Meeting - held in San Francisco, CA, US (poster #1513)


Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT01467427     History of Changes
Other Study ID Numbers: NN7999-3774
2011-000826-31 ( EudraCT Number )
U1111-1119-5013 ( Other Identifier: WHO )
JapicCTI- 121877 ( Registry Identifier: JAPIC )
First Submitted: October 31, 2011
First Posted: November 8, 2011
Results First Submitted: June 21, 2017
Results First Posted: November 17, 2017
Last Update Posted: August 2, 2018