An Open Label Study to Determine the Safety and Efficacy of Replacement Factor VIII Protein (Known as rFVIIIFc) in Untreated Males With Severe Hemophilia A

Study ID
STU 072015-042

Cancer Related
No

Healthy Volunteers
Yes

Study Sites

  • Children’s Medical Center (Dallas, Plano, Southlake)

Contact
Anna Winborn
2144568185
anna.winborn@childrens.com

Principal Investigator
Janna Journeycake, M.D.

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 Overview

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.

Eligibility

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.