Hemophilia Inhibitor Previously Untreated Patient Study (HIPS)

This study is currently recruiting participants. (see Contacts and Locations)
Verified June 2014 by The University of Texas Health Science Center, Houston
Sponsor:
Collaborators:
Rho
Baxter Healthcare Corporation
Information provided by (Responsible Party):
Deborah Brown, The University of Texas Health Science Center, Houston
ClinicalTrials.gov Identifier:
NCT01652027
First received: June 3, 2011
Last updated: June 17, 2014
Last verified: June 2014
  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 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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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: January 2017
Estimated Primary Completion Date: January 2017 (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

Genders Eligible for Study:   Both
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
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 ClinicalTrials.gov identifier: NCT01652027

Contacts
Contact: Deborah L. Brown, M.D. 713-500-8360 deborah.brown@uth.tmc.edu
Contact: Sharyne Donfield, PhD 919-595-6225 sharyne_donfield@rhoworld.com

Locations
United States, Georgia
Emory University Recruiting
Atlanta, Georgia, United States, 30322
Contact: Shannon Meeks, M.D.    404-727-1608    shannon.meeks@choa.org   
Contact: David Hellwedge    404-785-4677    david.hellwege@emoryhealthcare.org   
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    ashapiro@ihtc.org   
Contact: Erin Wiles    317-871-0011 ext 269    ewiles@ihtc.org   
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    vcradu2@email.uky.edu   
Contact: Jennifer Samuelson    859-257-1000    jennifer.samuelson@uky.edu   
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    rkruseja@tulane.edu   
Contact: Carol DeKernion    504-988-3566    cdekern@tulane.edu   
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    cam9061@med.cornell.edu   
Contact: Fraustina Hsu    212-746-4126    frh2005@med.cornell.edu   
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    paul_monahan@med.unc.edu   
Contact: Robin Kellerman    919-0843-3942    roberta_kellerman@med.unc.edu   
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    ralph.gruppo@cchmc.org   
Contact: Justin Pennington    513-803-4617    justin.pennington@cchmc.org   
Principal Investigator: Ralph Gruppo, M.D.         
United States, Oregon
University of Oregon Recruiting
Portland, Oregon, United States
Contact: Michael Recht, MD       rechtm@ohsu.edu   
Contact: susan Lattimore    503 418 4495    lattimor@ohsu.edu   
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    ragni@dom.pitt.edu   
Contact: Judith Kadosh    412-209-7263    JKadosh@itxm.org   
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    janna.journeycake@utsouthwestern.edu   
Contact: Anna Winborn    214-456-8185    anna.winborn@childrens.com   
Principal Investigator: Janna Journeycake, MD         
University of Texas Health Science Center-Houston Recruiting
Houston, Texas, United States, 77030
Contact: Deborah L. Brown, M.D.    713-500-8360    deborah.brown@uth.tmc.edu   
Contact: Madeline Cantini, BSN    713-500-8377    madeline.cantini@uth.tmc.edu   
Principal Investigator: Deborah L Brown, MD         
Baylor College of Medicine Recruiting
Houston, Texas, United States, 77030
Contact: Donald Yee, MD    832-822-4861    DLYEE@txch.org   
Contact: Margaret Nagel    832-824-1538    menagel@txch.org   
Principal Investigator: Donald Yee, MD         
United States, Utah
Intermountain Hemophilia and Thrombosis Center Recruiting
Salt Lake City, Utah, United States, 84108
Contact: Hassan Yaish, MD    801-662-4700    Hassan.Yaish@imail.org   
Contact: Tiffanie Hales    801-213-3367    Tiffanie.Hales@hsc.utah.edu   
Principal Investigator: Hassan Yaish, MD         
Austria
Medical University of Vienna Not yet recruiting
Vienna, Austria, A-1090
Contact: Christoph Male, MD    43 1 40400 2110/3232    christoph.male@meduniwien.ac.at   
Contact: Eva Wissmann    43 1 40400 3488    eva.wissmann@meduniwien.ac.at   
Principal Investigator: Christoph Male, MD         
Italy
Angelo Bianchi Bonomi Hemophilia & Thrombosis Center Not yet recruiting
Milan, Italy, 20122
Contact: Elena Santagostino, MD    39 02 5503 5308    Elena.Santagostino@policlinico.mi.it   
Contact: Vega Rusconi    39 02 5503 3623    vega.rusconi@gmail.com   
Principal Investigator: Elena Santagostino, MD         
Netherlands
Emma Children's Hospital AMC Recruiting
Amsterdam, Netherlands, 1105
Contact: Karin Fijnvandraaat, M.D.    31 20 566 2727    c.j.fijnvandraat@amc.uva.nl   
Contact: Samanth Gouw, M.D.    31 20 5662727    s.c.gouw@amc.uva.nl   
Principal Investigator: Karin Fijnvandraat, MD         
Sweden
Malmo Centre for Thrombosis and Haemostasis Recruiting
Malmo, Sweden, Se-205 02
Contact: Jenny Klintman, M.D.    46 40 331952    jenny.klintman@med.lu.se   
Contact: Marcus Ljungksvist    46 046 222 0000    marcus.ljungkvist@med.lu.se   
Principal Investigator: Jenny Klintman, M.D.         
Sponsors and Collaborators
The University of Texas Health Science Center, Houston
Rho
Baxter Healthcare Corporation
Investigators
Principal Investigator: Elena Santagostino, M.D. Maggiore Hospital and University of Milan
  More Information

No publications provided

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
Study First Received: June 3, 2011
Last Updated: June 17, 2014
Health Authority: United States: Institutional Review Board

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

ClinicalTrials.gov processed this record on September 30, 2014