HIPS: Hemophilia Inhibitor Previously Untreated Patient Study

Sponsor
The University of Texas Health Science Center, Houston (Other)
Overall Status
Completed
CT.gov ID
NCT01652027
Collaborator
Rho, Inc. (Industry), Baxter Healthcare Corporation (Industry)
25
17
104
1.5
0

Study Details

Study Description

Brief Summary

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 or Disease Intervention/Treatment Phase
  • Drug: FVIII concentrate

Study Design

Study Type:
Observational
Actual Enrollment :
25 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
Study of Immunologic Determinants of Inhibitor Development in Previously Untreated Patients With Hemophilia
Actual Study Start Date :
Jul 1, 2011
Actual Primary Completion Date :
Mar 1, 2020
Actual Study Completion Date :
Mar 1, 2020

Arms and Interventions

Arm Intervention/Treatment
Previously Untreated Patients with Hemophilia A

Drug: FVIII concentrate
usual treatment as directed by treating physician

Outcome Measures

Primary Outcome Measures

  1. Total number of FOXP3-positive T regulatory cells in the circulation [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

  1. FVIII-specific T-cells [50 exposure days to FVIII or 3 years, whichever comes first]

    FVIII-specific T-cells will be compared before and after exposure to FVIII

Eligibility Criteria

Criteria

Ages Eligible for Study:
N/A and Older
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Locations

Site City State Country Postal Code
1 Emory University Atlanta Georgia United States 30322
2 Indiana Hemophilia and Thrombosis Center Indianapolis Indiana United States 46260
3 University of Kentucky Lexington Kentucky United States 40536
4 Tulane University New Orleans Louisiana United States 70112
5 Cornell University New York New York United States 10065
6 University of North Carolina at Chapel Hill Chapel Hill North Carolina United States 27599
7 Cincinnati Children's Hospital Cincinnati Ohio United States 45229
8 University of Oregon Portland Oregon United States
9 Hemophilia Center of Western Pennsylvania Pittsburgh Pennsylvania United States 15213
10 North Texas Comprehensive Hemophilia Center Dallas Texas United States 75235
11 Baylor College of Medicine Houston Texas United States 77030
12 University of Texas Health Science Center-Houston Houston Texas United States 77030
13 Intermountain Hemophilia and Thrombosis Center Salt Lake City Utah United States 84108
14 Medical University of Vienna Vienna Austria A-1090
15 Angelo Bianchi Bonomi Hemophilia & Thrombosis Center Milan Italy 20122
16 Emma Children's Hospital AMC Amsterdam Netherlands 1105
17 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

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Deborah Brown, Associate Professor, The University of Texas Health Science Center, Houston
ClinicalTrials.gov Identifier:
NCT01652027
Other Study ID Numbers:
  • HSC-MS-11-0202
  • HSC-MS-11-0202
First Posted:
Jul 27, 2012
Last Update Posted:
Nov 10, 2020
Last Verified:
Nov 1, 2020
Keywords provided by Deborah Brown, Associate Professor, The University of Texas Health Science Center, Houston
Additional relevant MeSH terms:

Study Results

No Results Posted as of Nov 10, 2020