International Immune Tolerance Study

NCT ID: NCT00212472

Last Updated: 2009-12-07

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

TERMINATED

Clinical Phase

NA

Total Enrollment

134 participants

Study Classification

INTERVENTIONAL

Study Start Date

2002-07-31

Study Completion Date

2012-12-31

Brief Summary

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The purpose of this study is to see if a low-dose arm or a high dose-arm of immune tolerance is more effective in eliminating inhibitors in patients with hemophilia A.

Detailed Description

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Subjects will be randomized into a low-dose or high-dose immune tolerance regimen and this study will compare the success rates, the time to achieve tolerance,the complications and the cost of both regimens.It will also aim to identify predictors of successful immune tolerance.

Conditions

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Hemophilia A With Inhibitors

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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1

Low-dose treatment (50 FVIII u/kg three times a week).

Group Type ACTIVE_COMPARATOR

Factor VIII concentrates

Intervention Type DRUG

To be determined at the discretion of the investigator.

Low-dose treatment

Intervention Type OTHER

50 FVIII u/kg three times a week.

2

High-dose treatment (200 FVIII u/kg per day).

Group Type ACTIVE_COMPARATOR

Factor VIII concentrates

Intervention Type DRUG

To be determined at the discretion of the investigator.

High-dose treatment

Intervention Type OTHER

200 FVIII u/kg per day.

Interventions

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Factor VIII concentrates

To be determined at the discretion of the investigator.

Intervention Type DRUG

Low-dose treatment

50 FVIII u/kg three times a week.

Intervention Type OTHER

High-dose treatment

200 FVIII u/kg per day.

Intervention Type OTHER

Eligibility Criteria

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Inclusion Criteria

* Severe hemophilia A (FVIII level \<1%).
* A maximum historical inhibitor titer of between 5 BU and 200 BU that must be confirmed once prior to the beginning of ITI.
* The inhibitor titer should be \<10 BU at the start of ITI, confirmed once.
* The inhibitor must be present for \<24 months when ITI begins.
* Maximum age of 7 at the start of ITI.
* Willingness to comply with the protocol.

Exclusion Criteria

* Moderate or mild hemophilia A (FVIII level \>1%).
* Spontaneous disappearance of the inhibitor prior to ITI.
* Historical maximum inhibitor titer \<5 BU or \> 200 BU before starting ITI.
* Inhibitor titer \> 10 BU at the start of ITI.
* Inhibitor present for more than 24 months before starting ITI.
* Systemic immunomodulatory drug therapy during immune tolerance e.g. corticosteroids (\< 5 days every 2 months maximum dose 2 mg/kg or 60 mg/day), azathioprine, cyclophosphamide, high-dose immunoglobulin or the use of a protein A column or plasmapheresis.
* Age \> 7 years at the start of ITI.
* Inability or unwillingness to comply with the protocol.
* Previous attempt at ITI.
Maximum Eligible Age

7 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Manchester University NHS Foundation Trust

OTHER_GOV

Sponsor Role collaborator

New York Presbyterian Hospital

OTHER

Sponsor Role lead

Responsible Party

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New York Presbyterian Hospital

Principal Investigators

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Donna M DiMichele, MD

Role: PRINCIPAL_INVESTIGATOR

Weill Cornell Medical College-NY Presybetrian Hospital

Charles Hay, MD

Role: PRINCIPAL_INVESTIGATOR

Manchester Royal Infirmary

Locations

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University of Alabama Birmingham Medical Center

Birmingham, Alabama, United States

Site Status

City of Hope Medical Center

Duarte, California, United States

Site Status

Children's Hospital of Orange County

Orange, California, United States

Site Status

Mountain States Regional Hemophilia and Thrombosis Center

Aurora, Colorado, United States

Site Status

All Children's Hospital

St. Petersburg, Florida, United States

Site Status

Children's Healthcare of Atlanta

Atlanta, Georgia, United States

Site Status

Rush Presbyterian St. Lukes

Chicago, Illinois, United States

Site Status

Children's Memorial Hospital

Chicago, Illinois, United States

Site Status

Comprehensive Bleeding Disorders Center

Peoria, Illinois, United States

Site Status

Indiana Hemophilia & Thrombosis Center

Indianapolis, Indiana, United States

Site Status

Tulane University Hospital and Clinic

New Orleans, Louisiana, United States

Site Status

Maine Children's Cancer Program

Scarborough, Maine, United States

Site Status

Tufts - New England Medical Center

Boston, Massachusetts, United States

Site Status

Children's Hospital Boston

Boston, Massachusetts, United States

Site Status

University of Michigan Health Hospitals

Ann Arbor, Michigan, United States

Site Status

MSU Centers for Bleeding & Clotting Disorders

East Lansing, Michigan, United States

Site Status

Children's Hospital Minneapolis

Minneapolis, Minnesota, United States

Site Status

Mayo Comprehensive Hemophilia Center

Rochester, Minnesota, United States

Site Status

Kansas City Regional Hemophilia Center-The Children's Mercy Hospital

Kansas City, Missouri, United States

Site Status

Hackensack University Medical Center

Hackensack, New Jersey, United States

Site Status

Newark Beth Israel Medical Center

Newark, New Jersey, United States

Site Status

Saint Michael's Medical Center

Newark, New Jersey, United States

Site Status

Ted R. Montoya Hemophilia Treatment Center

Albuquerque, New Mexico, United States

Site Status

NY Presbyterian Hospital

New York, New York, United States

Site Status

Mount Sinai Medical Center

New York, New York, United States

Site Status

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States

Site Status

Children's Hospital Medical Center of Akron

Akron, Ohio, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Columbus Children's Hospital

Columbus, Ohio, United States

Site Status

Oregon Health and Science University

Portland, Oregon, United States

Site Status

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Site Status

St. Christopher's Hospital for Children, Section of Hem/Onc

Philadelphia, Pennsylvania, United States

Site Status

The Hemophilia Center of Western Pennsylvania

Pittsburgh, Pennsylvania, United States

Site Status

St. Jude Children's Research Hospital

Memphis, Tennessee, United States

Site Status

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status

University of Texas Health Science Center-Gulf States Hemophilia & Thrombosis Center

Houston, Texas, United States

Site Status

Children's Hospital of the King's Daughters

Norfolk, Virginia, United States

Site Status

Naval Medical Center

Portsmouth, Virginia, United States

Site Status

Comprehensive Center for Bleeding Disorders

Milwaukee, Wisconsin, United States

Site Status

Countries

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United States

Other Identifiers

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ITI

Identifier Type: -

Identifier Source: org_study_id