Epidemiology and Immunology of Hemophilia A Inhibitors

NCT ID: NCT00005518

Last Updated: 2016-05-13

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Study Classification

OBSERVATIONAL

Study Start Date

1998-06-30

Study Completion Date

2003-08-31

Brief Summary

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To determine the risk factors associated with inhibitor formation in hemophilia A and to study the mechanism of tolerance in the murine hemophilia A model.

Detailed Description

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BACKGROUND:

The major hypothesis of this study is that suppression of immunoregulatory cytokines will prevent factor VIII inhibitor formation in the hemophilia A murine model: if successful, a treatment protocol could be developed for individuals identified to be at highest risk for inhibitor formation. Through this approach to the epidemiology and immunology of inhibitor formation, the health care of individuals with hemophilia may be improved.

The study is in response to a Request for Applications released in 1998 on the Immunogenetics of Inhibitor Formation in Hemophilia. .

DESIGN NARRATIVE:

A case-control study is conducted comparing prevalent hemophilia patients with inhibitors identified in the HMS Study with three age-matched controls without inhibitors to determine clinical and laboratory characteristics associated with inhibitor development. A histocompatibility study is conducted identifying patterns of HLA Class II antigens by molecular typing in hemophilic subjects identified in HMS, with and without inhibitors, which may be linked to inhibitor formation. An animal tolerance study is conducted in the hemophilia A murine model, evaluating blockade of T cell costimulatory pathways by CTLA4-Ig and anti-CD40L mAb, to prevent inhibitor formation.

The study completion date listed in this record was obtained from the "End Date" entered in the Protocol Registration and Results System (PRS) record.

Conditions

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Blood Disease Hemophilia A

Eligibility Criteria

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Inclusion Criteria

No eligibility criteria
Maximum Eligible Age

100 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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National Heart, Lung, and Blood Institute (NHLBI)

NIH

Sponsor Role lead

Principal Investigators

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M.V. Ragni

Role:

University of Pittsburgh

Other Identifiers

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R01HL061937

Identifier Type: NIH

Identifier Source: secondary_id

View Link

5038

Identifier Type: -

Identifier Source: org_study_id

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