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An International Randomised Controlled Trial Of Immune Tolerance Induction

7 Years
Not Enrolling
Hemophilia A With Inhibitors

Thank you

Trial Information

An International Randomised Controlled Trial Of Immune Tolerance Induction

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

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

- Age > 7 years at the start of ITI.

- Inability or unwillingness to comply with the protocol.

- Previous attempt at ITI.

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Success-rate and partial success-rate

Outcome Time Frame:

Up to 69 months

Safety Issue:


Principal Investigator

Donna M DiMichele, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Weill Cornell Medical College-NY Presybetrian Hospital


United States: Institutional Review Board

Study ID:




Start Date:

July 2002

Completion Date:

December 2012

Related Keywords:

  • Hemophilia A With Inhibitors
  • Hemophilia A



Children's Hospital of Philadelphia Philadelphia, Pennsylvania  19104
Newark Beth Israel Medical Center Newark, New Jersey  07112
Hackensack University Medical Center Hackensack, New Jersey  07601
Children's Hospital of Orange County Orange, California  92668
All Children's Hospital St. Petersburg, Florida  33701
Maine Children's Cancer Program Scarborough, Maine  04074-9308
St. Jude Children's Research Hospital Memphis, Tennessee  38105-2794
Children's Hospital Medical Center of Akron Akron, Ohio  44308
Children's Hospital of the King's Daughters Norfolk, Virginia  23507
Tufts - New England Medical Center Boston, Massachusetts  02111
Mount Sinai Medical Center New York, New York  10029
Cincinnati Children's Hospital Medical Center Cincinnati, Ohio  45229-3039
Columbus Children's Hospital Columbus, Ohio  43205-2696
City of Hope Medical Center Duarte, California  91010
Vanderbilt University Medical Center Nashville, Tennessee  37232-2516
Children's Hospital Boston Boston, Massachusetts  02115
Children's Memorial Hospital Chicago, Illinois  60614
Tulane University Hospital and Clinic New Orleans, Louisiana  70112
NY Presbyterian Hospital New York, New York  10021
Oregon Health and Science University Portland, Oregon  97201
Children's Healthcare of Atlanta Atlanta, Georgia  30342
The Hemophilia Center of Western Pennsylvania Pittsburgh, Pennsylvania  15213
University of North Carolina at Chapel Hill Chapel Hill, North Carolina  27599
University of Alabama Birmingham Medical Center Birmingham, Alabama  35233
Mountain States Regional Hemophilia and Thrombosis Center Aurora, Colorado  80045
Rush Presbyterian St. Lukes Chicago, Illinois  60612
Comprehensive Bleeding Disorders Center Peoria, Illinois  61614
Indiana Hemophilia & Thrombosis Center Indianapolis, Indiana  46034
University of Michigan Health Hospitals Ann Arbor, Michigan  48109
MSU Centers for Bleeding & Clotting Disorders East Lansing, Michigan  48824
Children's Hospital Minneapolis Minneapolis, Minnesota  55404
Mayo Comprehensive Hemophilia Center Rochester, Minnesota  55905
Kansas City Regional Hemophilia Center-The Children's Mercy Hospital Kansas City, Missouri  64108
Saint Michael's Medical Center Newark, New Jersey  07102
Ted R. Montoya Hemophilia Treatment Center Albuquerque, New Mexico  87131
St. Christopher's Hospital for Children, Section of Hem/Onc Philadelphia, Pennsylvania  19134
University of Texas Health Science Center-Gulf States Hemophilia & Thrombosis Center Houston, Texas  77030
Naval Medical Center Portsmouth, Virginia  23708
Comprehensive Center for Bleeding Disorders Milwaukee, Wisconsin  53201