Hemophilia Inhibitor Previously Untreated Patient Study (HIPS)

November 9, 2020 updated by: Deborah Brown, The University of Texas Health Science Center, Houston

Study of Immunologic Determinants of Inhibitor Development in Previously Untreated Patients With Hemophilia

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

25

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Austria
      • Vienna, Austria, A-1090
        • Medical University of Vienna
  • Italy
      • Milan, Italy, 20122
        • Angelo Bianchi Bonomi Hemophilia & Thrombosis Center
  • Netherlands
      • Amsterdam, Netherlands, 1105
        • Emma Children's Hospital AMC
  • Sweden
      • Malmo, Sweden, Se-205 02
        • Malmö Centre for Thrombosis and Haemostasis
  • United States
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory University
    • Indiana
      • Indianapolis, Indiana, United States, 46260
        • Indiana Hemophilia and Thrombosis Center
    • Kentucky
      • Lexington, Kentucky, United States, 40536
        • University of Kentucky
    • Louisiana
      • New Orleans, Louisiana, United States, 70112
        • Tulane University
    • New York
      • New York, New York, United States, 10065
        • Cornell University
    • North Carolina
      • Chapel Hill, North Carolina, United States, 27599
        • University of North Carolina at Chapel Hill
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital
    • Oregon
      • Portland, Oregon, United States
        • University of Oregon
    • Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • Hemophilia Center of Western Pennsylvania
    • Texas
      • Dallas, Texas, United States, 75235
        • North Texas Comprehensive Hemophilia Center
      • Houston, Texas, United States, 77030
        • Baylor College of Medicine
      • Houston, Texas, United States, 77030
        • University of Texas Health Science Center-Houston
    • Utah
      • Salt Lake City, Utah, United States, 84108
        • Intermountain Hemophilia and Thrombosis Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients with severe hemophilia A who have not previously been treated with Factor VIII concentrates.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Previously Untreated Patients with Hemophilia A
Drug: FVIII concentrate
usual treatment as directed by treating physician

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
50 exposure days to FVIII or 3 years, whichever comes first

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
50 exposure days to FVIII or 3 years, whichever comes first

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

The University of Texas Health Science Center, Houston

Collaborators

Baxter Healthcare Corporation
Rho, Inc.

Investigators

  • Principal Investigator: Elena Santagostino, M.D., Maggiore Hospital and University of Milan

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 1, 2011

Primary Completion (Actual)

March 1, 2020

Study Completion (Actual)

March 1, 2020

Study Registration Dates

First Submitted

June 3, 2011

First Submitted That Met QC Criteria

July 26, 2012

First Posted (Estimate)

July 27, 2012

Study Record Updates

Last Update Posted (Actual)

November 10, 2020

Last Update Submitted That Met QC Criteria

November 9, 2020

Last Verified

November 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • HSC-MS-11-0202 (Other Identifier: University of Texas HSC-Houston Committee for the Protection of Human Subjects)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Hemophilia A

Clinical Trials on FVIII concentrate

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Malawi

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Subscribe