A Safety, Tolerability, and Pharmacokinetics Study of a Single Intravenous Injection of Recombinant Coagulation Factor VIII Fc - Von Willebrand Factor - XTEN Fusion Protein (rFVIIIFc-VWF-XTEN) (BIVV001) in Previously Treated Adults With Severe Hemophilia A (EXTEN-A)

Completed · Phase 1/Phase 2

Conditions studied: Hemophilia A

In brief

The primary purpose was to assess the safety and tolerability of a single intravenous (IV) administration of BIVV001 in adult previously treated patients (PTPs) with severe hemophilia A.

Key facts

Study ID: NCT03205163
Run by: Bioverativ, a Sanofi company
People needed: 16
Starts: 2017-08-28
Expected to finish: 2018-11-12
Last updated by the study team: 2022-04-19

Who can join

Age: 18 and older, up to 65. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

Ability of the participant, or his legally authorized representative (e.g., parent or legal guardian) if applicable in accordance with local regulations, to understand the purpose and risks of the study and provide signed and dated informed consent/assent and authorization to use confidential health information in accordance with national and local participant privacy regulations.
Severe hemophilia A, defined as less than (<) 1 international units per deciliter (IU/dL) (<1 percent [%]) endogenous FVIII at screening as determined by the one-stage clotting assay from the central laboratory. If the initial screening result was greater than or equal to (>=) 1%, then a repeat endogenous FVIII activity level was performed using the one stage clotting assay from the central laboratory. If the repeated result was < 1 IU/dL (<1%), then the participant met this inclusion requirement.
Previous treatment for hemophilia A, defined as at least 150 documented prior exposure days to any recombinant and/or plasma-derived FVIII and/or cryoprecipitate products at Day 1. Fresh frozen plasma treatment must not be considered in the count for documented exposure days.
Platelet count >=100,000 cells/ microliter (mcL) at screening (test performed by the central laboratory and reviewed prior to the Day 1 Advate dose).
A participant known to be human immunodeficiency virus (HIV) antibody positive, either previously documented or identified from screening assessments, must have the following results prior to Day 1 Advate dose: cluster of differentiation 4 (CD4) lymphocyte count greater than (>) 200 cells/millimeter (mm)\^3; viral load of <400 copies/mL.

You may not qualify if…

Medical History:
Any concurrent clinically significant major disease that, in the opinion of the Investigator, made the participant unsuitable for enrollment.
Serious active bacterial or viral infection (other than chronic hepatitis or HIV) present within 30 days of screening.
Other known coagulation disorder(s) in addition to hemophilia A.
History of hypersensitivity or anaphylaxis associated with any FVIII product.
Known or suspected allergy to mice, hamsters, or any ingredient in Advate.
History of a positive inhibitor test or clinical signs of decreased response to FVIII administrations. Family history of inhibitors not excluded the participant.
Medications and Procedures:
Current enrollment or participation within 30 days prior to screening in any other investigational study.
Other:
Inability to comply with study requirements as assessed by the Investigator.
Other unspecified reasons that, in the opinion of the Investigator or Sponsor, made the participant unsuitable for enrollment.

Where it is running

University of California Los Angeles Medical Center — Los Angeles, California, United States
Indiana Hemophilia and Thrombosis Center — Indianapolis, Indiana, United States
University of Iowa Hospitals & Clinics — Iowa City, Iowa, United States
Michigan State University — East Lansing, Michigan, United States
Mississippi Center for Advanced Medicine — Madison, Mississippi, United States
Hemophilia Center of Western PA — Pittsburgh, Pennsylvania, United States
University of Washington — Seattle, Washington, United States
Nara Medical University Hospital — Kashihara-shi, Nara, Japan
Tokyo Medical University Hospital — Shinjuku-Ku, Tokyo-To, Japan
Ogikubo Hospital — Tokyo, Tokyo-To, Japan

Full record on ClinicalTrials.gov

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