Hemophilia Inhibitor Previously Untreated Patient Study



Status:Active, not recruiting
Conditions:Anemia, Hematology
Therapuetic Areas:Hematology
Healthy:No
Age Range:Any
Updated:11/3/2017
Start Date:July 2011
End Date:December 2020

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Study of Immunologic Determinants of Inhibitor Development in Previously Untreated Patients With Hemophilia

Hemophilia A is a congenital bleeding disorder caused by deficiency of factor VIII (FVIII)
and is treated by replacement therapy with FVIII concentrate. Approximately 30% of people
with severe hemophilia A develop neutralizing antibodies, called FVIII inhibitors, which
interfere with the function of FVIII concentrates. The reason that some, but not all, people
with severe hemophilia A develop inhibitors is incompletely understood. Understanding
individual and environmental risk factors is important to be able to prevent and possibly
treat inhibitors. This study will look at individual and treatment characteristics in babies
with severe hemophilia A who have not yet received treatment with FVIII (called Previously
Untreated Patients, or PUPS). Subjects in the study will be asked to provide diaries of
treatments, medications, and illnesses. Treatment will be directed by the subjects'
physician, but all subjects will receive Advate, a third-generation recombinant FVIII
product. Subjects will have blood drawn for laboratory tests, which include studies of the
immune system and genetic studies of the FVIII mutation, before and 7-9 days after the first
treatment with FVIII, and 5 days (+/-2 days) after the 5th, 10th, 20th, 30th, 40th, and 50th
days of treatment with FVIII (exposure days). The duration of the study will be first 50
treatments or 3 years, whichever comes first.


Inclusion Criteria:

- Severe hemophilia A with FVIII activity < 1% normal

- Weight > 3.5 kg at the time of baseline study evaluation

- Informed consent, approved by appropriate Institutional Review Board/Independent
Ethics Committee, has been administered, signed, and dated

Exclusion Criteria:

- Prior exposure to clotting factor concentrates or blood products

- Other chronic disease

- Currently participating in another investigational drug study.
We found this trial at
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Salt Lake City, Utah 84108
Principal Investigator: Hassan Yaish, MD
Phone: 801-662-4700
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201 Dowman Dr
Atlanta, Georgia 30303
(404) 727-6123
Principal Investigator: Shannon Meeks, M.D.
Phone: 404-727-1608
Emory University Emory University, recognized internationally for its outstanding liberal artscolleges, graduate and professional schools,...
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Chapel Hill, North Carolina 27599
(919) 962-2211
Principal Investigator: Paul Monahan, M.D.
Phone: 919-843-4984
University of North Carolina at Chapel Hill Carolina’s vibrant people and programs attest to the...
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1200 Moursund Street
Houston, Texas 77030
(713) 798-4951
Principal Investigator: Donald Yee, MD
Phone: 832-822-4861
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7000 Fannin St
Houston, Texas 77030
(713) 500-4472
Principal Investigator: Deborah L Brown, MD
Phone: 713-500-8360
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Cincinnati, Ohio 45229
Principal Investigator: Ralph Gruppo, M.D.
Phone: 513-636-8617
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Dallas, Texas 75235
Principal Investigator: Janna Journeycake, MD
Phone: 214-730-5437
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Indianapolis, Indiana 46260
Principal Investigator: Amy Shapiro, M.D.
Phone: 317-871-0000
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Lexington, Kentucky
859) 257-9000
Principal Investigator: Vlad Radulescu, MD
Phone: 859-257-6033
University of Kentucky The University of Kentucky is a public, land grant university dedicated to...
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New Orleans, Louisiana 70112
Principal Investigator: Rebecca Kruse-Jarres, M.D.
Phone: 504-988-3562
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New York, New York 10021
Principal Investigator: Catherine McGuinn, MD
Phone: 212-746-3400
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Pittsburgh, Pennsylvania 15213
Principal Investigator: Margaret Ragni, M.D.
Phone: 412-209-7280
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3181 Southwest Sam Jackson Park Road
Portland, Oregon 97239
Principal Investigator: Michael Recht, MD
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Vienna,
Principal Investigator: Christoph Male, MD
Phone: 43 1 40400 2110/3232
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