An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Previously Untreated Males With Severe Hemophilia A
NCT ID: NCT02234323
Last Updated: 2022-04-11
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
108 participants
INTERVENTIONAL
2015-01-12
2019-09-23
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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rFVIIIFc
Participants were to receive rFVIIIFc as follows- Prophylaxis regimen (PR): rFVIIIFc 25-80 international units per kilogram (IU/kg), at 3- to 5-day intervals until participant reached greater than or equal to (\>=) 50 exposure days (ED: 24-hour period in which \>=1 injection/dose of rFVIIIFc was given), or study withdrawal/end of study. Adjustments to dose/dosing interval was done as needed by investigator; Treatment with an optional ER (Episodic regimen) can be initiated before PR at investigators discretion; ITI: rFVIIIFc 200 IU/kg, daily for participants who, after exposure to rFVIIIFc, had positive high titer inhibitor (\>=5.00 Bethesda Units per milliliter \[BU/mL\]) or positive low titer inhibitor (\>=0.60 and \<5.00 BU/mL) and had poorly controlled bleeding despite increased rFVIIIFc doses, or required bypassing agent to treat bleeding.
rFVIIIFc
Administered as specified in arm description
Interventions
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rFVIIIFc
Administered as specified in arm description
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Weight \>=3.5 kg at the time of screening.
* Severe hemophilia A defined as less than (\<) 1 IU/dL (\<1%) endogenous FVIII documented in the medical record or as tested during the Screening Period.
Exclusion Criteria
* History of positive inhibitor testing. A prior history of inhibitors was defined based on a patient's historical positive inhibitor test using the local laboratory Bethesda value for a positive inhibitor test (ie, equal to or above lower level of detection).
* History of hypersensitivity reactions associated with any rFVIIIFc administration.
* Other coagulation disorder(s) in addition to hemophilia A.
* Any concurrent clinically significant major disease that, in the opinion of the Investigator, would make the participant unsuitable for enrollment.
* Current systemic treatment with chemotherapy and/or other immunosuppressant drugs.
5 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
Little Rock, Arkansas, United States
Research Site
Duarte, California, United States
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Sacramento, California, United States
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San Francisco, California, United States
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Torrance, California, United States
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Indianapolis, Indiana, United States
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Louisville, Kentucky, United States
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Brewer, Maine, United States
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East Lansing, Michigan, United States
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Minneapolis, Minnesota, United States
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St Louis, Missouri, United States
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Buffalo, New York, United States
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New York, New York, United States
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Rochester, New York, United States
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Columbus, Ohio, United States
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Portland, Oregon, United States
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Philadelphia, Pennsylvania, United States
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Dallas, Texas, United States
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Dallas, Texas, United States
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Milwaukee, Wisconsin, United States
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Brisbane, , Australia
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Parkville, , Australia
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Perth, , Australia
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Westmead, , Australia
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Campinas, , Brazil
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Canoas, , Brazil
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Ribeirão Preto, , Brazil
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Rio de Janeiro, , Brazil
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São Paulo, , Brazil
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Hamilton, , Canada
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London, , Canada
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Toronto, , Canada
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Caen, , France
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Le Kremlin-Bicêtre, , France
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Lille, , France
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Nantes, , France
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Strasbourg, , France
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Toulouse, , France
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Tours, , France
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Berlin, , Germany
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Bonn, , Germany
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Düsseldorf, , Germany
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Frankfurt, , Germany
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Hannover, , Germany
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Dublin, , Ireland
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Vicenza, VI, Italy
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Bari, , Italy
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Florence, , Italy
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Genova, , Italy
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Milan, , Italy
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Napoli, , Italy
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Padua, , Italy
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Rome, , Italy
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Rome, , Italy
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Leiden, , Netherlands
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Utrecht, , Netherlands
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Auckland, , New Zealand
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Christchurch, , New Zealand
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Gdansk, , Poland
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Krakow, , Poland
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Lublin, , Poland
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Warsaw, , Poland
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Barcelona, , Spain
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Madrid, , Spain
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Stockholm, , Sweden
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Cambridge, Cambridgeshire, United Kingdom
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Basingstoke, Hampshire, United Kingdom
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Whitechapel, London, United Kingdom
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Cardiff, , United Kingdom
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London, , United Kingdom
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Sheffield, , United Kingdom
Countries
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References
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Carcao M, Schiavulli M, Kulkarni R, Rendo P, Foster M, Santagostino E, Casiano S, Konigs C. A post hoc analysis of previously untreated patients with severe hemophilia A who developed inhibitors in the PUPs A-LONG trial. Blood Adv. 2024 Mar 26;8(6):1494-1503. doi: 10.1182/bloodadvances.2023011475.
Konigs C, Ozelo MC, Dunn A, Kulkarni R, Nolan B, Brown SA, Schiavulli M, Gunawardena S, Mukhopadhyay S, Jayawardene D, Winding B, Carcao M. First study of extended half-life rFVIIIFc in previously untreated patients with hemophilia A: PUPs A-LONG final results. Blood. 2022 Jun 30;139(26):3699-3707. doi: 10.1182/blood.2021013563.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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2013-005512-10
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
997HA306
Identifier Type: -
Identifier Source: org_study_id
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