for males ages up to 5 years (full criteria)
at San Francisco, California and other locations
study started
estimated completion
Tiffany Lin



The primary objective of the study is to evaluate the safety of rFVIIIFc (BIIB031) in previously untreated participants with severe hemophilia A. The secondary objectives are to evaluate the efficacy of rFVIIIFc in the prevention and treatment of bleeding episodes in previously untreated patients (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.

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


Hemophilia A prophylaxis treatment Hemophilia episodic treatment Factor VIII rFVIIIFc


For males ages up to 5 years

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 subject privacy regulations.
  • Weight >=3.5 kg at the time of screening.
  • Severe hemophilia A defined as <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.
  • 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.


  • Research Site
    San Francisco California 94143 United States
  • Research Site
    Sacramento California 95817 United States

Lead Scientist

  • Tiffany Lin
    As a faculty member at UCSF Pediatric Hematology/Oncology, I see patients with pediatric hematologic and oncologic disorders although the majority of my time is spent in hematologic diseases such as patients with bleeding disorders, thromboses, immune cytopenias or aplastic anemia, bone marrow failure syndromes, and more.


in progress, not accepting new patients
Start Date
Completion Date
Bioverativ Therapeutics Inc.
Phase 3
Study Type
Last Updated