RecruitingNCT02207894

A Survey on the Success of Inhibitor Elimination Using Individualized Concentrate Selection and Controlled ITI

A Survey on the Success of Inhibitor Elimination Using Individualized Concentrate Selection and Controlled (High Dose) Immune Tolerance Induction (ITI)

Sponsor

Haemophilia Centre Rhine Main

Enrollment

300 participants

Start Date

Aug 1, 2006

Study Type

OBSERVATIONAL

Conditions

Haemophilia A

Summary

This research program is initiated to evaluate and document data on the success of ITI in 300 haemophilia A patients with newly developed or already existing FVIII-inhibitors (also patients who might potentially have failed in earlier ITIs), which will be treated with ITI - preferably high-dose based on individualized product selection, in order to improve management of this potentially devastating complication of haemophilia treatment. In order to investigate the role of in vitro tests on individual ITI success rate in patients undergoing ITI, the inhibitor plasma samples can be assayed against different FVIII concentrates using the following in vitro tests: Batch selection, Thrombin generation assay (TGA), Thrombin Generation Test (TGT) to monitor FVIII efficacy, Epitope mapping,IgG Subclasses specific for FVIII, Immunogenotyping.

Eligibility

Sex: MALE

Inclusion Criteria2

Based on the decision of the treating physicians in the participating centres, male patients at any age suffering from severe (FVIII activity \< 1%), moderate (FVIII activity \>1% - 5%), or mild (FVIII activity \> 5%) haemophilia A will be included into this post marketing observation if relevant inhibitor levels (\> 0.6 BU) have been detected, or - in case of an inhibitor level \<0.6 BU - with reduced recovery or half-life of FVIII.
The observation is also open for patients who failed an earlier ITI attempt.