Brief Title
Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B
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
Brief Summary
The primary objective of the study was to evaluate the safety of recombinant coagulation
factor IX Fc fusion protein (rFIXFc, BIIB029) in previously untreated patients (PUPs) with
severe hemophilia B. Secondary objectives were to evaluate the efficacy of rFIXFc in the
prevention and treatment of bleeding episodes in PUPs, and to evaluate rFIXFc consumption for
prevention and treatment of bleeding episodes in PUPs.
Study Phase
Phase 3
Study Type
Interventional
Primary Outcome
Percentage of Participants With Confirmed Inhibitor Development as Measured by the Nijmegen-Modified Bethesda Assay
Secondary Outcome
Annualized Number of Bleeding Episodes (Spontaneous and Traumatic) Per Participant (Annualized Bleeding Rate [ABR])
Condition
Hemophilia B
Intervention
rFIXFc
Study Arms / Comparison Groups
Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc)
Description: Participants received rFIXFc intravenous (IV) injection as follows: Prophylactic treatment regimen: started with rFIXFc 50 International Units per kilogram (IU/kg) weekly until a participant reached at least 50 exposure days (ED=24-hour period in which greater than or equal to (>=1) injection/dose of rFIXFc was given) to rFIXFc, withdrawal from study or end of study. Adjustments to dose and dosing interval was based on incremental recovery, subsequent Factor IX (FIX) levels, physical activity, bleeding pattern, in accordance with local standards of care for prophylactic regimen (PR). Treatment with episodic (on demand) regimen can be initiated before PR at investigators discretion. Episodic (On demand; optional): rFIXFc at individual doses based on participant's clinical condition, type and severity of bleeding event until PR.
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Biological
Estimated Enrollment
33
Start Date
November 13, 2014
Completion Date
August 20, 2019
Primary Completion Date
August 20, 2019
Eligibility Criteria
Key Inclusion Criteria:
- Weight >=3.5 kilogram at the time of informed consent.
- Severe hemophilia B was defined as less than or equal to (<=)2 International Units per
deciliter (IU/dL) (<=2 percent [%]) endogenous FIX 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 was defined based
on a participant'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 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 have made the participant unsuitable for enrollment (example HIV
infection with cluster of differentiation 4 (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 was allowed. Treatment in these circumstances
should not have exceeded 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,
would have made the participant unsuitable for enrollment.
NOTE: Other protocol-defined inclusion/exclusion criteria may apply.
Gender
Male
Ages
N/A - 17 Years
Accepts Healthy Volunteers
No
Contacts
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Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT02234310
Organization ID
998HB303
Secondary IDs
2013-003629-27
Responsible Party
Sponsor
Study Sponsor
Bioverativ, a Sanofi company
Collaborators
Swedish Orphan Biovitrum
Study Sponsor
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Verification Date
July 2020