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Hemophilia Inhibitor Previously Untreated Patient Study (HIPS)

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2016 by The University of Texas Health Science Center, Houston
Rho, Inc.
Baxter Healthcare Corporation
Information provided by (Responsible Party):
Deborah Brown, The University of Texas Health Science Center, Houston Identifier:
First received: June 3, 2011
Last updated: May 27, 2016
Last verified: May 2016
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.

Condition Intervention
Hemophilia A
Drug: FVIII concentrate

Study Type: Observational
Study Design: Observational Model: Cohort
Time Perspective: Prospective
Official Title: Study of Immunologic Determinants of Inhibitor Development in Previously Untreated Patients With Hemophilia

Resource links provided by NLM:

Further study details as provided by The University of Texas Health Science Center, Houston:

Primary Outcome Measures:
  • Total number of FOXP3-positive T regulatory cells in the circulation [ Time Frame: 50 exposure days to FVIII or 3 years, whichever comes first ]
    FoxP3(a protein involved in immune system responses)-positive T regulatory cells in the circulation will be compared before and after exposure to FVIII.

Secondary Outcome Measures:
  • FVIII-specific T-cells [ Time Frame: 50 exposure days to FVIII or 3 years, whichever comes first ]
    FVIII-specific T-cells will be compared before and after exposure to FVIII

Biospecimen Retention:   Samples With DNA
Plasma samples, lymphocyte cell lines, Genomic DNA, RNA

Estimated Enrollment: 50
Study Start Date: July 2011
Estimated Study Completion Date: December 2020
Estimated Primary Completion Date: December 2019 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
Previously Untreated Patients with Hemophilia A Drug: FVIII concentrate
usual treatment as directed by treating physician


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with severe hemophilia A who have not previously been treated with Factor VIII concentrates.

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT01652027

Contact: Deborah L. Brown, M.D. 713-500-8360
Contact: Sharyne Donfield, PhD 919-595-6225

United States, Georgia
Emory University Recruiting
Atlanta, Georgia, United States, 30322
Contact: Shannon Meeks, M.D.    404-727-1608   
Contact: David Hellwedge    404-785-4677   
Principal Investigator: Shannon Meeks, M.D.         
United States, Indiana
Indiana Hemophilia and Thrombosis Center Recruiting
Indianapolis, Indiana, United States, 46260
Contact: Amy Shapiro, M.D.    317-871-0000   
Contact: Erin Wiles    317-871-0011 ext 269   
Principal Investigator: Amy Shapiro, M.D.         
United States, Kentucky
University of Kentucky Not yet recruiting
Lexington, Kentucky, United States, 40536
Contact: Vlad Radulescu, MD    859-257-6033   
Contact: Jennifer Samuelson    859-257-1000   
Principal Investigator: Vlad Radulescu, MD         
United States, Louisiana
Tulane University Recruiting
New Orleans, Louisiana, United States, 70112
Contact: Rebecca Kruse-Jarres, M.D.    504-988-3562   
Contact: Carol DeKernion    504-988-3566   
Principal Investigator: Rebecca Kruse-Jarres, M.D.         
United States, New York
Cornell University Not yet recruiting
New York, New York, United States, 10065
Contact: Catherine McGuinn, MD    212-746-3400   
Contact: Fraustina Hsu    212-746-4126   
Principal Investigator: Catherine McGuinn, MD         
United States, North Carolina
University of North Carolina at Chapel Hill Recruiting
Chapel Hill, North Carolina, United States, 27599
Contact: Paul Monahan, M.D.    919-843-4984   
Contact: Robin Kellerman    919-0843-3942   
Principal Investigator: Paul Monahan, M.D.         
United States, Ohio
Cincinnati Children's Hospital Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Ralph Gruppo, M.D.    513-636-8617   
Contact: Justin Pennington    513-803-4617   
Principal Investigator: Ralph Gruppo, M.D.         
United States, Oregon
University of Oregon Recruiting
Portland, Oregon, United States
Contact: Michael Recht, MD   
Contact: susan Lattimore    503 418 4495   
Principal Investigator: Michael Recht, MD         
United States, Pennsylvania
Hemophilia Center of Western Pennsylvania Recruiting
Pittsburgh, Pennsylvania, United States, 15213
Contact: Margaret Ragni, M.D.    412-209-7280   
Contact: Judith Kadosh    412-209-7263   
Principal Investigator: Margaret Ragni, M.D.         
United States, Texas
North Texas Comprehensive Hemophilia Center Recruiting
Dallas, Texas, United States, 75235
Contact: Janna Journeycake, MD    214-730-5437   
Contact: Anna Winborn    214-456-8185   
Principal Investigator: Janna Journeycake, MD         
Baylor College of Medicine Recruiting
Houston, Texas, United States, 77030
Contact: Donald Yee, MD    832-822-4861   
Contact: Margaret Nagel    832-824-1538   
Principal Investigator: Donald Yee, MD         
University of Texas Health Science Center-Houston Recruiting
Houston, Texas, United States, 77030
Contact: Deborah L. Brown, M.D.    713-500-8360   
Contact: Madeline Cantini, BSN    713-500-8377   
Principal Investigator: Deborah L Brown, MD         
United States, Utah
Intermountain Hemophilia and Thrombosis Center Recruiting
Salt Lake City, Utah, United States, 84108
Contact: Hassan Yaish, MD    801-662-4700   
Contact: Tiffanie Hales    801-213-3367   
Principal Investigator: Hassan Yaish, MD         
Medical University of Vienna Not yet recruiting
Vienna, Austria, A-1090
Contact: Christoph Male, MD    43 1 40400 2110/3232   
Contact: Eva Wissmann    43 1 40400 3488   
Principal Investigator: Christoph Male, MD         
Angelo Bianchi Bonomi Hemophilia & Thrombosis Center Not yet recruiting
Milan, Italy, 20122
Contact: Elena Santagostino, MD    39 02 5503 5308   
Contact: Vega Rusconi    39 02 5503 3623   
Principal Investigator: Elena Santagostino, MD         
Emma Children's Hospital AMC Recruiting
Amsterdam, Netherlands, 1105
Contact: Karin Fijnvandraaat, M.D.    31 20 566 2727   
Contact: Samanth Gouw, M.D.    31 20 5662727   
Principal Investigator: Karin Fijnvandraat, MD         
Malmo Centre for Thrombosis and Haemostasis Recruiting
Malmo, Sweden, Se-205 02
Contact: Jenny Klintman, M.D.    46 40 331952   
Contact: Marcus Ljungksvist    46 046 222 0000   
Principal Investigator: Jenny Klintman, M.D.         
Sponsors and Collaborators
The University of Texas Health Science Center, Houston
Rho, Inc.
Baxter Healthcare Corporation
Principal Investigator: Elena Santagostino, M.D. Maggiore Hospital and University of Milan
  More Information

Responsible Party: Deborah Brown, Associate Professor, The University of Texas Health Science Center, Houston Identifier: NCT01652027     History of Changes
Other Study ID Numbers: HSC-MS-11-0202
HSC-MS-11-0202 ( Other Identifier: University of Texas HSC-Houston Committee for the Protection of Human Subjects )
Study First Received: June 3, 2011
Last Updated: May 27, 2016

Keywords provided by The University of Texas Health Science Center, Houston:
FVIII inhibitors

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn processed this record on May 23, 2017