A Safety, Efficacy and Pharmacokinetics Study of a Recombinant Fusion Protein Linking Coagulation Factor IX With Albumin (rIX-FP) in Children With Hemophilia B

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
CSL Behring
ClinicalTrials.gov Identifier:
NCT01662531
First received: August 7, 2012
Last updated: April 3, 2016
Last verified: April 2016
Results First Received: April 3, 2016  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Hemophilia B
Intervention: Biological: rIX-FP

  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
Of the 18 sites that were activated, subjects were enrolled from 17 sites in 10 countries.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Subjects <12 years of age with severe hemophilia B (FIX activity of ≤2%) were planned to be enrolled in the study, including 11 to 12 subjects in each age group (6 to <12 years and <6 years of age). Of 29 subjects screened, 27 subjects were enrolled and treated with rIX-FP.

Reporting Groups
  Description
rIX-FP

Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP) will be administered by IV infusion as routine weekly prophylaxis and episodic treatment for bleeding episodes.

rIX-FP: Recombinant Fusion Protein Linking Coagulation Factor IX with Albumin (rIX-FP)


Participant Flow:   Overall Study
    rIX-FP  
STARTED     27  
COMPLETED     27  
NOT COMPLETED     0  



  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.
All 27 subjects were included in the Safety, Pharmacokinetic and Efficacy populations.

Reporting Groups
  Description
Age < 6 Years

Subjects less than 6 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study.

Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.

Age 6 to <12 Years

Subjects between 6 and less than 12 years of age who received a single dose of 50 IU/kg rIX-FP on Day 1 during the pharmacokinetic phase of the study.

Subjects received weekly (7-day) routine prophylaxis treatment with an initial weekly dose of 35 to 50 IU/kg rIX-FP, which may have been adjusted based on protocol-specified criteria.

Total Total of all reporting groups

Baseline Measures
    Age < 6 Years     Age 6 to <12 Years     Total  
Number of Participants  
[units: participants]
  12     15     27  
Age  
[units: years]
Mean (Standard Deviation)
  3.2  (1.70)     8.1  (1.41)     5.9  (2.93)  
Gender  
[units: participants]
     
Female     0     0     0  
Male     12     15     27  



  Outcome Measures
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1.  Primary:   Incremental Recovery Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product   [ Time Frame: 30 minutes after infusion ]

2.  Primary:   Half-life (t1/2) Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product   [ Time Frame: Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose ]

3.  Primary:   Area Under the Concentration Versus Time Curve From Time Point Zero to the Last Sample With Quantifiable Drug Concentration (AUClast)   [ Time Frame: Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose ]

4.  Primary:   Clearance for FIX Activity Following a Single Intravenous Dose of 50 IU/kg rIX-FP or Previous FIX Product   [ Time Frame: Pre-dose, 30 minutes, 3, 24, 48, 72 120, 168, 240 and 336 hours post-dose ]

5.  Primary:   Number of Subjects Developing Inhibitors to Factor IX (FIX)   [ Time Frame: 12 months ]

6.  Secondary:   Number of Subjects With Treatment-related Adverse Events   [ Time Frame: 12 months ]

7.  Secondary:   Number of Subjects Developing Antibodies Against rIX-FP   [ Time Frame: 12 months ]

8.  Secondary:   Number of Bleeding Episodes Requiring One, Two or More Than Two Infusions of rIX-FP to Achieve Hemostasis   [ Time Frame: Approximately 12 months ]

9.  Secondary:   Consumption of rIX-FP During Routine Prophylaxis   [ Time Frame: 12 months ]


  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
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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.


Results Point of Contact:  
Name/Title: Clinical Trial Disclosure Manager
Organization: CSL Behring
phone: Use email contact
e-mail: clinicaltrials@cslbehring.com



Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT01662531     History of Changes
Other Study ID Numbers: CSL654_3002
2011-006032-23 ( EudraCT Number )
Study First Received: August 7, 2012
Results First Received: April 3, 2016
Last Updated: April 3, 2016
Health Authority: Australia: Department of Health and Ageing Therapeutic Goods Administration
Austria: Federal Office for Safety in Health Care
Canada: Health Canada
Czech Republic: State Institute for Drug Control
France: Agence Nationale de Sécurité du Médicament et des produits de santé
Germany: Paul-Ehrlich-Institut
Italy: The Italian Medicines Agency
Russia: Ministry of Health of the Russian Federation
Spain: Agencia Española de Medicamentos y Productos Sanitarios