Brief Title
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
Official Title
An Open-Label, Multicenter Evaluation of the Safety and Efficacy of Recombinant Coagulation Factor VIII Fc Fusion Protein (rFVIIIFc; BIIB031) in the Prevention and Treatment of Bleeding in Previously Untreated Patients With Severe Hemophilia A
Brief Summary
The primary objective of the study was to evaluate the safety of rFVIIIFc (BIIB031) in
previously untreated participants (PUPs) with severe hemophilia A. The secondary objectives
were to evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding
episodes in PUPs, to evaluate rFVIIIFc consumption for the prevention and treatment of
bleeding episodes in PUPs, and to describe experience with the use of rFVIIIFc for immune
tolerance induction (ITI) in participants with inhibitors.
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 A
Intervention
rFVIIIFc
Study Arms / Comparison Groups
rFVIIIFc
Description: 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.
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
108
Start Date
January 12, 2015
Completion Date
September 23, 2019
Primary Completion Date
September 23, 2019
Eligibility Criteria
Key Inclusion Criteria:
- Ability of the participant's legally authorized representative (e.g. their parent or
legal guardian) to understand the purpose and risks of the study and provide signed
and dated informed consent and authorization to use confidential health information in
accordance with national and local participant privacy regulations.
- 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.
Key Exclusion Criteria:
- Any exposure to blood components, factor VIII replacement products, including
commercially available rFVIIIFc at any time prior to or during screening.
- 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.
Note: Other protocol defined Inclusion/Exclusion criteria may apply.
Gender
Male
Ages
N/A - 5 Years
Accepts Healthy Volunteers
No
Contacts
, ,
Location Countries
Australia
Location Countries
Australia
Administrative Informations
NCT ID
NCT02234323
Organization ID
997HA306
Secondary IDs
2013-005512-10
Responsible Party
Sponsor
Study Sponsor
Bioverativ, a Sanofi company
Collaborators
Swedish Orphan Biovitrum
Study Sponsor
, ,
Verification Date
July 2020