Study to Determine the Safety and Efficacy of rFIXFc in Previously Untreated Males With Severe Hemophilia B
NCT ID: NCT02234310
Last Updated: 2022-03-25
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
33 participants
INTERVENTIONAL
2014-11-13
2019-08-20
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Recombinant Coagulation Factor IX Fc Fusion Protein (rFIXFc)
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.
rFIXFc
Adjustments to the dose and interval of rFIXFc was made in this study based on investigator discretion using available pharmacokinetic (PK) data, subsequent FIX trough and peak levels, level of physical activity, and bleeding pattern, in accordance with local standards of care for a prophylactic regimen. There was an option to start study dosing as episodic treatment (on-demand).
Interventions
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rFIXFc
Adjustments to the dose and interval of rFIXFc was made in this study based on investigator discretion using available pharmacokinetic (PK) data, subsequent FIX trough and peak levels, level of physical activity, and bleeding pattern, in accordance with local standards of care for a prophylactic regimen. There was an option to start study dosing as episodic treatment (on-demand).
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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.
Exclusion Criteria
* 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.
17 Years
MALE
No
Sponsors
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Swedish Orphan Biovitrum
INDUSTRY
Bioverativ, a Sanofi company
INDUSTRY
Responsible Party
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Locations
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Research Site
Sacramento, California, United States
Research Site
Washington D.C., District of Columbia, United States
Research Site
Atlanta, Georgia, United States
Research Site
Indianapolis, Indiana, United States
Research Site
Louisville, Kentucky, United States
Research Site
New Orleans, Louisiana, United States
Research Site
East Lansing, Michigan, United States
Research Site
Traverse City, Michigan, United States
Research Site
Columbus, Ohio, United States
Research Site
Portland, Oregon, United States
Research Site
Pittsburgh, Pennsylvania, United States
Research Site
Westmead, New South Wales, Australia
Research Site
Aarhus, , Denmark
Hopital Cardiologique - CHU Lille
Lille, Nord, France
Research Site
Lyon, Rhone, France
Research Site
Dublin, , Ireland
Research Site
Milan, , Italy
Research Site
Napoli, , Italy
Research Site
Parma, , Italy
Research Site
Roma, , Italy
Research Site
Utrecht, , Netherlands
Research Site
Auckland, , New Zealand
Research Site
Warsaw, , Poland
Research Site
Malmo, , Sweden
Research Site
Cambridge, Cambridgeshire, United Kingdom
Research Site
Whitechapel, London, United Kingdom
Research Site
London, , United Kingdom
Countries
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References
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Nolan B, Recht M, Rendo P, Falk A, Foster M, Casiano S, Rauch A, Shapiro A. Prophylaxis with recombinant factor IX Fc fusion protein reduces the risk of bleeding and delays time to first spontaneous bleed event in previously untreated patients with haemophilia B: A post hoc analysis of the PUPs B-LONG study. Eur J Haematol. 2024 Oct;113(4):485-492. doi: 10.1111/ejh.14252. Epub 2024 Jun 25.
Nolan B, Klukowska A, Shapiro A, Rauch A, Recht M, Ragni M, Curtin J, Gunawardena S, Mukhopadhyay S, Jayawardene D, Winding B, Fischer K, Liesner R. Final results of the PUPs B-LONG study: evaluating safety and efficacy of rFIXFc in previously untreated patients with hemophilia B. Blood Adv. 2021 Jul 13;5(13):2732-2739. doi: 10.1182/bloodadvances.2020004085.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2013-003629-27
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
998HB303
Identifier Type: -
Identifier Source: org_study_id
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