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Summary

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

Description

Summary

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

Keywords

Hemophilia A prophylaxis treatment Hemophilia episodic treatment Factor VIII Immunoglobulin Fc Fragments

Eligibility

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.

Locations

  • Research Site accepting new patients
    Sacramento, California, 95817, United States
  • Research Site accepting new patients
    Duarte, California, 91010, United States
  • Research Site accepting new patients
    Torrance, California, 90509, United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Bioverativ Therapeutics Inc.
ID
NCT02234323
Phase
Phase 3
Lead Scientist
Kristin Shimano
Study Type
Interventional
Last Updated
May 1, 2017
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