Study to Evaluate the Safety, Pharmacokinetics and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Subjects With Severe Hemophilia A
NCT ID: NCT01181128
Last Updated: 2021-01-08
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
165 participants
INTERVENTIONAL
2010-11-30
2012-08-31
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
PARALLEL
TREATMENT
NONE
Study Groups
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Individualized (Tailored) Prophylaxis
On rFVIIIFc Day 0, all participants underwent pharmacokinetic (PK) analysis with 50 IU/kg rFVIIIFc to estimate their PK parameters and guide the appropriate dose or interval of dosing. A subset of participants (Sequential PK subgroup) also had PK analyses performed with a single dose of 50 IU/kg Advate (Advate Day 0) within 8 weeks prior to rFVIIIFc Day 0. A \>= 96 hour washout was performed before the PK dose of Advate or rFVIIIFc was administered. Repeat PK profiling with a single dose of 50 IU/kg rFVIIIFc was conducted at Week 14 or after 12 to 24 weeks of prophylaxis with rFVIIIFc.
After PK assessments, all participants started twice weekly treatment with 25 IU/kg of rFVIIIFc via intravenous (IV) injection on Day 1 and 50 IU/kg on Day 4, followed by individualized dose and interval modification within the range of 25 to 65 IU/kg every 3 to 5 days, as determined by rFVIIIFc PK analysis, to maintain a trough level of 1% to 3% (or higher, as clinically indicated) FVIII activity.
Factor VIII (rFVIIIFc)
Advate®
Weekly Prophylaxis
65 IU/kg of rFVIIIFc via IV injection every 7 days
Factor VIII (rFVIIIFc)
Episodic (On-Demand) Dosing
10 to 50 IU/kg rFVIIIFc via IV injection, as required to treat a bleeding episode
Factor VIII (rFVIIIFc)
Interventions
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Factor VIII (rFVIIIFc)
Advate®
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Diagnosed with severe hemophilia A, defined as \<1 IU/dL (\<1%) endogenous Factor VIII)
* History of at least 150 documented prior exposure days to any Factor VIII product
* Platelet count ≥100,000 cells/μL
Exclusion Criteria
* Kidney and liver dysfunction
* Diagnosed with other coagulation disorder(s) in addition to hemophilia A
* Prior history of hypersensitivity or anaphylaxis associated with any FVIII or 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
Little Rock, Arkansas, United States
Research Site
Los Angeles, California, United States
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Orange, California, United States
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Sacramento, California, United States
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San Diego, California, United States
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Washington D.C., District of Columbia, United States
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Indianapolis, Indiana, United States
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Iowa City, Iowa, United States
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Louisville, Kentucky, United States
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New Orleans, Louisiana, United States
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Boston, Massachusetts, United States
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East Lansing, Michigan, United States
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Las Vegas, Nevada, United States
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New York, New York, United States
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Chapel Hill, North Carolina, United States
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Philadelphia, Pennsylvania, United States
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Philadelphia, Pennsylvania, United States
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Pittsburgh, Pennsylvania, United States
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Houston, Texas, United States
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Salt Lake City, Utah, United States
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Seattle, Washington, United States
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Camperdown, New South Wales, Australia
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Adelaide, South Australia, Australia
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Perth, Western Australia, Australia
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Vienna, , Austria
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Brussels, , Belgium
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Campinas, , Brazil
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Calgary, , Canada
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Toronto, , Canada
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Vancouver, , Canada
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Lyon, , France
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Bonn, Northwest, Germany
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Berlin, State of Berlin, Germany
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Hong Kong, , Hong Kong
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Bangalore, Karna, India
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Pune, Mahara, India
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New Delhi, National Capital Territory of Delhi, India
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Ludhiana, Punjab, India
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Vellore, Tamil Nadu, India
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Ramat Gan, , Israel
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Florence, FI, Italy
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Milan, MI, Italy
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Vicenza, VI, Italy
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Kashihara-shi, , Japan
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Kawasaki-shi, , Japan
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Kitakyushu-shi, , Japan
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Nagoya, , Japan
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Shinjuku-ku, , Japan
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Suginami-ku, , Japan
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Auckland, , New Zealand
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Christchurch, , New Zealand
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Palmerston North, , New Zealand
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Johannesburg Parktown, Gauteng, South Africa
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Cape Town, W Cape, South Africa
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Barcelona, , Spain
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Madrid, , Spain
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Gothenburg, , Sweden
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Zurich, , Switzerland
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Basingstoke, , United Kingdom
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Cambridge, , United Kingdom
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Glasgow, , United Kingdom
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London, , United Kingdom
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London, , United Kingdom
Research Site
London, , United Kingdom
Countries
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References
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Mahlangu J, Powell JS, Ragni MV, Chowdary P, Josephson NC, Pabinger I, Hanabusa H, Gupta N, Kulkarni R, Fogarty P, Perry D, Shapiro A, Pasi KJ, Apte S, Nestorov I, Jiang H, Li S, Neelakantan S, Cristiano LM, Goyal J, Sommer JM, Dumont JA, Dodd N, Nugent K, Vigliani G, Luk A, Brennan A, Pierce GF; A-LONG Investigators. Phase 3 study of recombinant factor VIII Fc fusion protein in severe hemophilia A. Blood. 2014 Jan 16;123(3):317-25. doi: 10.1182/blood-2013-10-529974. Epub 2013 Nov 13.
Shapiro AD, Ragni MV, Kulkarni R, Oldenberg J, Srivastava A, Quon DV, Pasi KJ, Hanabusa H, Pabinger I, Mahlangu J, Fogarty P, Lillicrap D, Kulke S, Potts J, Neelakantan S, Nestorov I, Li S, Dumont JA, Jiang H, Brennan A, Pierce GF. Recombinant factor VIII Fc fusion protein: extended-interval dosing maintains low bleeding rates and correlates with von Willebrand factor levels. J Thromb Haemost. 2014 Nov;12(11):1788-800. doi: 10.1111/jth.12723. Epub 2014 Oct 10.
Raheja P, Kragh N, Bystricka L, Eriksson D, Aroui K, Mezghani M, Barbier S, Linari S. Long-term efmoroctocog alfa prophylaxis improves perceived pain, mental, and physical health in patients with hemophilia A: post hoc analysis of phase III trials using patient-reported outcomes. Ther Adv Hematol. 2024 Jul 30;15:20406207241257917. doi: 10.1177/20406207241257917. eCollection 2024.
Katragadda S, Neelakantan S, Diao L, Wong N. Population Pharmacokinetic Analysis of Recombinant Factor VIII Fc Fusion Protein in Subjects With Severe Hemophilia A: Expanded to Include Pediatric Subjects. J Clin Pharmacol. 2021 Jul;61(7):889-900. doi: 10.1002/jcph.1854. Epub 2021 Apr 14.
Other Identifiers
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997HA301
Identifier Type: -
Identifier Source: org_study_id
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