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Hemophilia Inhibitor Previously Untreated Patient Study (HIPS)

This study is ongoing, but not recruiting participants.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01652027
First Posted: July 27, 2012
Last Update Posted: November 1, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Collaborators:
Rho, Inc.
Baxter Healthcare Corporation
Information provided by (Responsible Party):
Deborah Brown, The University of Texas Health Science Center, Houston
  Purpose
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 Deborah Brown, 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

Enrollment: 25
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

  Eligibility

Information from the National Library of Medicine

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, Learn About Clinical Studies.


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.
Criteria

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
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01652027


Locations
United States, Georgia
Emory University
Atlanta, Georgia, United States, 30322
United States, Indiana
Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, United States, 46260
United States, Kentucky
University of Kentucky
Lexington, Kentucky, United States, 40536
United States, Louisiana
Tulane University
New Orleans, Louisiana, United States, 70112
United States, New York
Cornell University
New York, New York, United States, 10065
United States, North Carolina
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, United States, 27599
United States, Ohio
Cincinnati Children's Hospital
Cincinnati, Ohio, United States, 45229
United States, Oregon
University of Oregon
Portland, Oregon, United States
United States, Pennsylvania
Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, United States, 15213
United States, Texas
North Texas Comprehensive Hemophilia Center
Dallas, Texas, United States, 75235
Baylor College of Medicine
Houston, Texas, United States, 77030
University of Texas Health Science Center-Houston
Houston, Texas, United States, 77030
United States, Utah
Intermountain Hemophilia and Thrombosis Center
Salt Lake City, Utah, United States, 84108
Austria
Medical University of Vienna
Vienna, Austria, A-1090
Italy
Angelo Bianchi Bonomi Hemophilia & Thrombosis Center
Milan, Italy, 20122
Netherlands
Emma Children's Hospital AMC
Amsterdam, Netherlands, 1105
Sweden
Malmo Centre for Thrombosis and Haemostasis
Malmo, Sweden, Se-205 02
Sponsors and Collaborators
The University of Texas Health Science Center, Houston
Rho, Inc.
Baxter Healthcare Corporation
Investigators
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
ClinicalTrials.gov 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 )
First Submitted: June 3, 2011
First Posted: July 27, 2012
Last Update Posted: November 1, 2017
Last Verified: October 2017

Keywords provided by Deborah Brown, 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