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Long-Term Safety and Efficacy of rFIXFc in the Prevention and Treatment of Bleeding Episodes in Previously Treated Participants With Hemophilia B (B-YOND)

This study is ongoing, but not recruiting participants.
Information provided by (Responsible Party):
Biogen Identifier:
First received: August 19, 2011
Last updated: September 29, 2016
Last verified: September 2016

The primary objective of the study is to evaluate the long-term safety of rFIXFc in participants with hemophillia B.

The secondary objective of this study is to evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes.

Condition Intervention Phase
Severe Hemophilia B
Biological: rFIXFc
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: An Open-Label, Multicenter, Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor IX Fusion Protein (rFIXFc) in the Prevention and Treatment of Bleeding Episodes in Previously Treated Subjects With Hemophilia B

Resource links provided by NLM:

Further study details as provided by Biogen:

Primary Outcome Measures:
  • Occurrence of inhibitor development [ Time Frame: Up to 4 years ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • The annualized number of bleeding episodes (spontaneous and traumatic) per participant [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]
  • The annualized number of spontaneous joint bleeding episodes per participant [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]
  • The total number of days of exposure per participant per year [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]
    One exposure day (ED) is equivalent to a 24 hour period in which rFIXFc is dosed.

  • The consumption of rFIXFc as total dose per kg per participant per year per treatment regimen [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]
  • Physicians global assessment of participant's response to his treatment regimen using a 4-point scale [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]
    Investigators will record assessments of each participant's response to his rFIXIFc regimen grading on the following 4-point scale: excellent, effective, partially effective, ineffective.

  • Participant's/ caregiver's assessment of response to treatment of bleeding episodes using a 4-point scale [ Time Frame: Up to 4 years ] [ Designated as safety issue: No ]
    Using the eDiary, each participant or the participant's caregiver will rate the treatment response to any bleeding episode using the following 4-point scale (excellent, good, moderate, or none).

Enrollment: 120
Study Start Date: December 2011
Estimated Study Completion Date: December 2018
Estimated Primary Completion Date: December 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: On-Demand
The individual dose of rFIXFc to treat bleeding episodes will be based on participant's clinical condition, type and severity of the bleeding event, and if indicated, Factor IX peak (recovery) levels.
Biological: rFIXFc
Administered as specified in the treatment arm.
Other Names:
  • coagulation factor IX (recombinant) Fc fusion protein
  • Alprolix
  • BIIB029
Experimental: Prophylaxis
Weekly prophylaxis, individualized prophylaxis or personalized prophylaxis available.
Biological: rFIXFc
Administered as specified in the treatment arm.
Other Names:
  • coagulation factor IX (recombinant) Fc fusion protein
  • Alprolix
  • BIIB029

Detailed Description:
Participants will follow either a prophylaxis or on-demand regimen. The starting dose in this study will be determined by the clinical profile of the patient in the preceding studies, B-LONG 998HB102 (NCT01027364) and Kids B-LONG study 9HB02PED (NCT01440946)

Ages Eligible for Study:   Child, Adult, Senior
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No

Key Inclusion Criteria:

  • Subjects who have completed studies 998HB102 (NCT01027364) or 9HB02PED (NCT01440946) or other studies with rFIXFc
  • Ability to understand the purposes & risks of the study and provide signed and dated informed consent.

Key Exclusion Criteria:

  • High-titer inhibitor (>/=5.00 BU/mL)

NOTE: Other protocol defined Inclusion/ Exclusion criteria may apply.

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01425723

  Show 46 Study Locations
Sponsors and Collaborators
Study Director: Medical Director Biogen
  More Information

Responsible Party: Biogen Identifier: NCT01425723     History of Changes
Other Study ID Numbers: 9HB01EXT  2011-003075-11 
Study First Received: August 19, 2011
Last Updated: September 29, 2016
Health Authority: Belgium: Federal Agency for Medicinal Products and Health Products
France: Afssaps - Agence française de sécurité sanitaire des produits de santé (Saint-Denis)
Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)
Ireland: Irish Medicines Board
China: Food and Drug Administration
Australia: Department of Health and Ageing Therapeutic Goods Administration
India: Ministry of Health
Hong Kong: Department of Health
Italy: Ministry of Health
South Africa: Department of Health
Japan: Pharmaceuticals and Medical Devices Agency (PMDA)
Brazil: Ministry of Health
Sweden: Medical Products Agency
Germany: Federal Institute for Drugs and Medical Devices
Canada: Health Canada
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Poland: Office for Registration of Medicinal Products, Medical Devices and Biocidal Products
United States: Food and Drug Administration

Keywords provided by Biogen:
B-LONG Extension
Severe Hemophilia B

Additional relevant MeSH terms:
Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked processed this record on December 02, 2016