International Immune Tolerance Study

• Status: Terminated
• Trial end date: 2012-12-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: N/A
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: New York Presbyterian Hospital
• Collaborators: Central Manchester University Hospitals NHS Foundation Trust; Manchester University NHS Foundation Trust
• Treatments: Factor VIII

Criteria

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.