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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.
Sponsor:
Information provided by (Responsible Party):
Bioverativ Therapeutics Inc.
ClinicalTrials.gov Identifier:
NCT01425723
First received: August 19, 2011
Last updated: May 5, 2017
Last verified: May 2017
  Purpose

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
Intervention Model: Parallel Assignment
Masking: No masking
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 Bioverativ Therapeutics Inc.:

Primary Outcome Measures:
  • Occurrence of inhibitor development [ Time Frame: Up to 4 years ]

Secondary Outcome Measures:
  • The annualized number of bleeding episodes (spontaneous and traumatic) per participant [ Time Frame: Up to 4 years ]
  • The annualized number of spontaneous joint bleeding episodes per participant [ Time Frame: Up to 4 years ]
  • The total number of days of exposure per participant per year [ Time Frame: Up to 4 years ]
    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 ]
  • Physicians global assessment of participant's response to his treatment regimen using a 4-point scale [ Time Frame: Up to 4 years ]
    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 ]
    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: June 2017
Estimated Primary Completion Date: June 2017 (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)
  Eligibility

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

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 ClinicalTrials.gov identifier: NCT01425723

  Show 50 Study Locations
Sponsors and Collaborators
Bioverativ Therapeutics Inc.
Investigators
Study Director: Medical Director Bioverativ Therapeutics Inc.
  More Information

Responsible Party: Bioverativ Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT01425723     History of Changes
Other Study ID Numbers: 9HB01EXT
2011-003075-11
Study First Received: August 19, 2011
Last Updated: May 5, 2017

Keywords provided by Bioverativ Therapeutics Inc.:
B-LONG
B-YOND
B-LONG Extension
rFIXFc
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

ClinicalTrials.gov processed this record on July 21, 2017