Study to Determine the Safety and Efficacy of rFIXFc in Untreated Males With Severe Hemophilia B



Status:Active, not recruiting
Conditions:Hematology
Therapuetic Areas:Hematology
Healthy:No
Age Range:Any - 17
Updated:4/5/2019
Start Date:November 2014
End Date:August 2019

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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

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.


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.
We found this trial at
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