RANDOMISED STUDY OF FIRST CHOICE IMMUNOTOLERANCE INDUCTION IN PATIENTS WITH SEVERE TYPE A HAEMOPHILIA WITH INHIBITOR AT HIGH RISK OF FAILURE: COMPARISON OF INDUCTION OF IMMUNE TOLERANCE WITH FVIII CONCENTRATES WITH OR WITHOUT VON WILLEBRAND FACTOR - RES.I.ST.NAÏVE
- Conditions
- SEVERE TYPE A HAEMOPHILIAMedDRA version: 9.1Level: LLTClassification code 10010432Term: Congenital deficiency of other clotting factors
- Registration Number
- EUCTR2008-007016-15-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
- 148
a. severe hemophilia A (FVIII<1%) b. male patients, any age; c. high responders (peak inhibitor levels > 5 BU); d. any inhibitor level at study enrolment; e. ability and willingness to participate in to the study; f. 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. no concomitant experimental treatment; h. at least one of the following risk factors for ITI failure: i. peak inhibitor titer > 200 BU ii. titer at ITI start > 10 BU iii. age > 7 years iv. time between inhibitor occurrence and ITI > 2 years i. no previous ITI attempt.
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 at evaluating whether FVIII/VWF concentrates can induce more frequently or more rapidly immune tolerance to FVIII in haemophilia A patients with high-responding inhibitors at high risk to fail in comparison 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 can induce more frequently or more rapidly immune tolerance to FVIII in haemophilia A patients with high-responding inhibitors at high risk to fail in comparison with VWF-free FVIII concentrates.
- Secondary Outcome Measures
Name Time Method