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Summary

Eligibility
for males ages up to 17 years
Location
at San Francisco, California and other locations
Dates
study started
estimated completion:

Description

Summary

The primary objective of the study is to evaluate the safety of rFIXFc (recombinant coagulation factor IX Fc fusion protein, BIIB029) in previously untreated participants with severe hemophilia B. Secondary objectives are to evaluate the efficacy of rFIXFc in the prevention and treatment of bleeding episodes in previously untreated participants (PUPs), and to evaluate rFIXFc consumption for prevention and treatment of bleeding episodes in previously untreated participants.

Official Title

An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc; BIIB029) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia B

Keywords

Hemophilia B prophylaxis treatment episodic treatment

Eligibility

For males ages up to 17 years

Key Inclusion Criteria:

  • Weight>=3.5 kilogram at the time of informed consent
  • Severe hemophilia B defined as <=2 IU/dl (<=2%) endogenous factor IX documented in the medical record or as tested during the Screening Period.

Key Exclusion Criteria:

  • History of positive inhibitor testing. A prior history of inhibitors is defined based on a patient's historical positive inhibitor test using the local laboratory Bethesda value for a positive inhibitor test (that is equal to or above lower limit of detection)
  • History of hypersensitivity reactions associated with any Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc) administration
  • Exposure to blood components or injection with a coagulation factor IX (FIX)concentrate (including plasma derived) other than rFIXFc
  • Injection with commercially available rFIXFc more than 28 days prior to Screening
  • More than 3 injections of commercially available rFIXFc prior to confirmation of eligibility
  • Other coagulation disorders in addition to hemophilia B
  • Any concurrent clinically significant major disease that, in the opinion of the Investigator, would make the subject unsuitable for enrollment (example HIV infection with CD4 lymphocyte count less than (<)200 cells/microliter (mcL) or a viral load greater than (>)200 particles/mcL, or any other known congenital or acquired immunodeficiency)
  • Current systemic treatment with chemotherapy and/or other immunosuppressant drugs. Use of steroids for treatment of asthma or management of acute allergic episodes or otherwise life-threatening episodes is allowed. Treatment in these circumstances should not exceed a 14-day duration
  • Participation within the past 30 days in any other clinical study involving investigational treatment
  • Current enrollment in any other clinical study involving investigational treatment
  • Inability to comply with study requirements
  • Other unspecified reasons that, in the opinion of the Investigator or Bioverativ, make the subject unsuitable for enrollment

NOTE: Other protocol-defined inclusion/exclusion criteria may apply.

Locations

  • Research Site not yet accepting patients
    Sacramento, California, 95817, United States
  • Research Site accepting new patients
    Torrance, California, 90502, United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Bioverativ Therapeutics Inc.
ID
NCT02234310
Phase
Phase 3
Study Type
Interventional
Last Updated
February 1, 2017
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