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An International Randomised Controlled Trial Of Immune Tolerance Induction


N/A
N/A
7 Years
Not Enrolling
Both
Hemophilia A With Inhibitors

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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
tolerance.


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.

Type of Study:

Interventional

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:

No

Principal Investigator

Donna M DiMichele, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Weill Cornell Medical College-NY Presybetrian Hospital

Authority:

United States: Institutional Review Board

Study ID:

ITI

NCT ID:

NCT00212472

Start Date:

July 2002

Completion Date:

December 2012

Related Keywords:

  • Hemophilia A With Inhibitors
  • Hemophilia A

Name

Location

Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania  19104
Newark Beth Israel Medical CenterNewark, New Jersey  07112
Hackensack University Medical CenterHackensack, New Jersey  07601
Children's Hospital of Orange CountyOrange, California  92668
All Children's HospitalSt. Petersburg, Florida  33701
Maine Children's Cancer ProgramScarborough, Maine  04074-9308
St. Jude Children's Research HospitalMemphis, Tennessee  38105-2794
Children's Hospital Medical Center of AkronAkron, Ohio  44308
Children's Hospital of the King's DaughtersNorfolk, Virginia  23507
Tufts - New England Medical CenterBoston, Massachusetts  02111
Mount Sinai Medical CenterNew York, New York  10029
Cincinnati Children's Hospital Medical CenterCincinnati, Ohio  45229-3039
Columbus Children's HospitalColumbus, Ohio  43205-2696
City of Hope Medical CenterDuarte, California  91010
Vanderbilt University Medical CenterNashville, Tennessee  37232-2516
Children's Hospital BostonBoston, Massachusetts  02115
Children's Memorial HospitalChicago, Illinois  60614
Tulane University Hospital and ClinicNew Orleans, Louisiana  70112
NY Presbyterian HospitalNew York, New York  10021
Oregon Health and Science UniversityPortland, Oregon  97201
Children's Healthcare of AtlantaAtlanta, Georgia  30342
The Hemophilia Center of Western PennsylvaniaPittsburgh, Pennsylvania  15213
University of North Carolina at Chapel HillChapel Hill, North Carolina  27599
University of Alabama Birmingham Medical CenterBirmingham, Alabama  35233
Mountain States Regional Hemophilia and Thrombosis CenterAurora, Colorado  80045
Rush Presbyterian St. LukesChicago, Illinois  60612
Comprehensive Bleeding Disorders CenterPeoria, Illinois  61614
Indiana Hemophilia & Thrombosis CenterIndianapolis, Indiana  46034
University of Michigan Health HospitalsAnn Arbor, Michigan  48109
MSU Centers for Bleeding & Clotting DisordersEast Lansing, Michigan  48824
Children's Hospital MinneapolisMinneapolis, Minnesota  55404
Mayo Comprehensive Hemophilia CenterRochester, Minnesota  55905
Kansas City Regional Hemophilia Center-The Children's Mercy HospitalKansas City, Missouri  64108
Saint Michael's Medical CenterNewark, New Jersey  07102
Ted R. Montoya Hemophilia Treatment CenterAlbuquerque, New Mexico  87131
St. Christopher's Hospital for Children, Section of Hem/OncPhiladelphia, Pennsylvania  19134
University of Texas Health Science Center-Gulf States Hemophilia & Thrombosis CenterHouston, Texas  77030
Naval Medical CenterPortsmouth, Virginia  23708
Comprehensive Center for Bleeding DisordersMilwaukee, Wisconsin  53201