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Eligibility
for males ages up to 5 years
Location
at San Francisco, California and other locations
Dates
study started
estimated completion:
Principal Investigator

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
    Indianapolis, Indiana, 46260, USA
  • Research Site not yet accepting patients
    Louisville, Kentucky, 40202, USA
  • Research Site not yet accepting patients
    Brewer, Maine, 04412, USA
  • Research Site accepting new patients
    St. Louis, Maryland, 63104, USA
  • Research Site accepting new patients
    East Lansing, Michigan, 48823, USA
  • Research Site accepting new patients
    Minneapolis, Minnesota, 55404, USA
  • Research Site not yet accepting patients
    Rochester, New York, 14621, USA
  • Research Site accepting new patients
    Columbus, Ohio, 43205, USA
  • Research Site accepting new patients
    Brisbane, 4029, Australia
  • Research Site accepting new patients
    Parkville, 3052, Australia
  • Research Site accepting new patients
    Perth, 6008, Australia
  • Research Site accepting new patients
    Westmead, 2145, Australia
  • Research Site accepting new patients
    Campinas, 13083-878, Brazil
  • Research Site not yet accepting patients
    Canoas, 92425-900, Brazil
  • Research Site not yet accepting patients
    Ribeirão Preto, 14048-900, Brazil
  • Research Site not yet accepting patients
    Rio de Janeiro, 20211-030, Brazil
  • Research Site not yet accepting patients
    São Paulo, 01227-200, Brazil
  • Research Site accepting new patients
    Hamilton, L8N 3Z5, Canada
  • Research Site accepting new patients
    London, N6A 4G5, Canada
  • Research Site accepting new patients
    Toronto, M5G 1X8, Canada
  • Research Site not yet accepting patients
    Aahus N, 8200, Denmark
  • Research Site accepting new patients
    Caen cedex 9, 14033, France
  • Research Site not yet accepting patients
    Le Kremlin Bicêtre cedex, 94275, France
  • Research Site accepting new patients
    Lille Cedex, 59037, France
  • Research Site accepting new patients
    Nantes Cedex 1, 44093, France
  • Research Site accepting new patients
    Strasbourg Cedex 2, 67200, France
  • Research Site not yet accepting patients
    Toulouse cedex, 31059, France
  • Research Site accepting new patients
    Tours cedex 9, 37044, France
  • Research Site accepting new patients
    Bonn, 53127, Germany
  • Research Site not yet accepting patients
    Duesseldorf, 40225, Germany
  • Research Site accepting new patients
    Frankfurt, 60590, Germany
  • Research Site accepting new patients
    Dublin, Ireland
  • Research Site accepting new patients
    Florence, 50139, Italy
  • Research Site accepting new patients
    Genova, 16148, Italy
  • Research Site accepting new patients
    Milan, 20122, Italy
  • Research Site accepting new patients
    Padova, 35128, Italy
  • Research Site accepting new patients
    Rome, 161, Italy
  • Research Site accepting new patients
    Rome, 165, Italy
  • Research Site accepting new patients
    Christchurch, New Zealand
  • Research Site accepting new patients
    Gdansk, 80-952, Poland
  • Research Site accepting new patients
    Warszawa, 02-091, Poland
  • Research Site accepting new patients
    Barcelona, Spain
  • Research Site accepting new patients
    Madrid, 28046, Spain
  • Research Site accepting new patients
    Stockholm, 171 76, Sweden
  • Research Site accepting new patients
    Cambridge, Cambridgeshire, CB2 2QQ, United Kingdom
  • Research Site accepting new patients
    Basingstoke, Hampshire, RG24 9NA, United Kingdom
  • Research Site accepting new patients
    Whitechapel, London, E1 188, United Kingdom
  • Research Site accepting new patients
    Cardiff, CF14 4XW, United Kingdom
  • Research Site accepting new patients
    London, WC1N3JH, United Kingdom

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
April 2017
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