Study of Recombinant Factor IX Fc Fusion Protein (rFIXFc) in Participants With Hemophilia B
NCT ID: NCT01027364
Last Updated: 2020-12-19
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
123 participants
INTERVENTIONAL
2009-12-31
2012-07-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Fixed Weekly Interval
50 IU/kg rFIXFc via intravenous (IV) injection once every 7 days initially, then at a dose indicated by the participant's baseline pharmacokinetic (PK) assessment that ensured a target trough of 1% to 3% above baseline or higher, as clinically indicated. Adjustments to the initial weekly dose of rFIXFc (50 IU/kg) were to be made based on baseline PK assessments, occurrence of spontaneous bleeding episodes, and the trough levels, which were to be monitored at Weeks 4, 16, 26, and 39.
Prior to the first dose of rFIXFc, participants in the Sequential PK subgroup were to receive a single dose of 50 IU/kg BeneFIX administered IV in the clinic, followed by PK sampling. A single dose of 50 IU/kg rFIXFc was administered following a 120-hour washout from BeneFIX, followed by PK sampling for a baseline PK profiling. At Week 26 (±1 week) subjects were to receive a single dose of 50 IU/kg rFIXFc for repeat PK profiling.
Factor IX (rFIXFc)
rFIX
Individualized Interval
100 IU/kg rFIXFc via IV injection once every 10 days initially, then at an interval derived from the baseline PK assessment that ensured a target trough of 1% to 3% above baseline or higher, as clinically indicated. Adjustments to the initial 10-day interval were to be made based on baseline PK assessments and trough levels, which were monitored at Weeks 4, 16, 26, and 39.
Factor IX (rFIXFc)
On Demand
20 to 100 IU/kg rFIXFc via IV injection, or the dose indicated by the participant's baseline PK to target a plasma level of 20% to 100%, as needed for the treatment of mild to severe bleeding episodes
Factor IX (rFIXFc)
Surgery
The surgical period and dosing are dependent on the type of surgery the participant undergoes. Participants who started the study in one of the other treatment arms prior to surgery will return to the original treatment arm. Participants who joined the study in the Surgery arm will be assigned to one of the other treatment arms following post-operative rehabilitation.
Factor IX (rFIXFc)
Interventions
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Factor IX (rFIXFc)
rFIX
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Diagnosed with hemophilia B (baseline Factor IX level less than or equal to 2%)
* History of at least 100 exposure days to any Factor IX product
* Platelet count ≥100,000 cells/μL
Exclusion Criteria
* Kidney or liver dysfunction
* Diagnosed with another coagulation defect other than hemophilia B
* Prior history of anaphylaxis associated with any Factor IX or intravenous (IV) immunoglobulin administration
12 Years
MALE
No
Sponsors
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Swedish Orphan Biovitrum
INDUSTRY
Bioverativ Therapeutics Inc.
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director
Role: STUDY_DIRECTOR
Bioverativ Therapeutics Inc.
Locations
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Research Site
Phoenix, Arizona, United States
Research Site
Sacramento, California, United States
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Aurora, Colorado, United States
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Chicago, Illinois, United States
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Indianapolis, Indiana, United States
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East Lansing, Michigan, United States
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Philadelphia, Pennsylvania, United States
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Pittsburgh, Pennsylvania, United States
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Seattle, Washington, United States
Research Site
Camperdown, New South Wales, Australia
Research Site
Adelaide, South Australia, Australia
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Perth, Western Australia, Australia
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Brussels, , Belgium
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Leuven, , Belgium
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Campinas, , Brazil
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Calgary, Alberta, Canada
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Toronto, Ontario, Canada
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Montreal, Quebec, Canada
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Vancouver, , Canada
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Beijing, , China
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Guangzhou, , China
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Shanghai, , China
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Tianjin, , China
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Lyon, , France
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Marseille, , France
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Berlin, , Germany
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Bonn, , Germany
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Pokfulam, , Hong Kong
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Shatin, , Hong Kong
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Ludhiana, Punjab, India
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Vellore, Tamil Nadu, India
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Bangalore, , India
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Pune, , India
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Florence, , Italy
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Milan, , Italy
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Kasihara-City, , Japan
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Kawasaki, , Japan
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Kitakyushu, , Japan
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Nagoya, , Japan
Ressearch Site
Suginami-ku, , Japan
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Tokyo, , Japan
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Lodz, , Poland
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Warsaw, , Poland
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Moscow, , Russia
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Saint Petersburg, , Russia
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Johannesburg, Gauteng, South Africa
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Cape Town, Western Cape, South Africa
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Malmo, , Sweden
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Stockholm, , Sweden
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Cambridge, , United Kingdom
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London, , United Kingdom
Countries
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References
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Powell JS, Pasi KJ, Ragni MV, Ozelo MC, Valentino LA, Mahlangu JN, Josephson NC, Perry D, Manco-Johnson MJ, Apte S, Baker RI, Chan GC, Novitzky N, Wong RS, Krassova S, Allen G, Jiang H, Innes A, Li S, Cristiano LM, Goyal J, Sommer JM, Dumont JA, Nugent K, Vigliani G, Brennan A, Luk A, Pierce GF; B-LONG Investigators. Phase 3 study of recombinant factor IX Fc fusion protein in hemophilia B. N Engl J Med. 2013 Dec 12;369(24):2313-23. doi: 10.1056/NEJMoa1305074. Epub 2013 Dec 4.
Shapiro AD, Kulkarni R, Ragni MV, Chambost H, Mahlangu J, Oldenburg J, Nolan B, Ozelo MC, Foster MC, Willemze A, Barnowski C, Jain N, Winding B, Dumont J, Lethagen S, Barnes C, Pasi KJ. Post hoc longitudinal assessment of the efficacy and safety of recombinant factor IX Fc fusion protein in hemophilia B. Blood Adv. 2023 Jul 11;7(13):3049-3057. doi: 10.1182/bloodadvances.2022009230.
Other Identifiers
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2009-014295-21
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
998HB102
Identifier Type: -
Identifier Source: org_study_id