IMMUNE TOLERANCE INDUCTION STUDY IN PATIENTS WITH SEVERE TYPE A HAEMOPHILIA WITH INHIBITOR AFTER FAILURE OF A PREVIOUS INDUCTION OF IMMUNE TOLERANCE WITH FVIII CONCENTRATES WITHOUT VON WILLEBRAND FACTOR - RES.I.ST.EXPERIENCED
- Conditions
- SEVERE TYPE A HAEMOPHILIAMedDRA version: 9.1Level: LLTClassification code 10010432Term: Congenital deficiency of other clotting factors
- Registration Number
- EUCTR2008-007019-33-IT
- Lead Sponsor
- FONDAZIONE CENTRO EMOFILIA E TROMBOSI ANGELO BIANCHI BONOMI
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- Male
- Target Recruitment
- 50
a. severe hemophilia A (FVIII<1%) b. male patients, any age; c. high responders (peak inhibitor levels > 5 BU); d. with any inhibitor level at study enrolment; e. with ability and willingness to participate to the study; f. with no concomitant systemic treatment with drugs with immunosuppressive side effects (eg. Corticosteroids, if used more than 5 days every iii months and/or at a dose of > 2mg/kg or 60 mg/day, azathioprine, cyclophosphamide, high?dose immunoglobulin as well as the use of a protein A column or plasmapheresis, interferons);g. with no concomitant experimental treatment. h. previous ITI course of at least 9 monthswith a VWF-free FVIII concentrate at any dosage, such as recombinant FVIII and/or monoclonally purified FVIII. i. patients who initially succeeded to clear the inhibitor and then relapsed can be included
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
see inclusion criteria
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: The study is aimed to evaluate whether FVIII/vWF concentrates successfully induce immune tolerance in patients who have already experienced and failed immune tolerance induction with VWF-free FVIII concentrates.;Secondary Objective: I.Maintenance of immune tolerance II.Time to achieve success III.Safety - compliance to treatment IV.Cost of care;Primary end point(s): The study is aimed to evaluate whether FVIII/vWF concentrates successfully induce immune tolerance in patients who have already experienced and failed immune tolerance induction with VWF-free FVIII concentrates.
- Secondary Outcome Measures
Name Time Method