Hemophilia Inhibitor Previously Untreated Patient Study (HIPS)

This study is currently recruiting participants.
Verified February 2013 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: February 6, 2013
Last verified: February 2013
  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-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
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, Texas
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   
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: February 6, 2013
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 April 22, 2014