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
- Conditions
- Patients with severe Haemophilia A with a high responding inhibitor (more than 5 BU/ml) that have failed to respond to immune tolerance induction with VWF free FVIII concentrates (plasma derived or recombinant).
- Registration Number
- EUCTR2008-007019-33-GB
- Lead Sponsor
- niversity of Milan
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- Male
- Target Recruitment
- 50
1) Severe haemophilia A (FVIII less than 1% of normal)
2) Males of any age
3) High responders (peak titre more than 5 BU/ml)
4) Any inhibitor at time of enrolment
5) With ability and willingness to participate in the study
6) Previous ITI course of at least 9 months with a VWF-free FVIII concentrate at any dosage, such as recombinant FVIII and/or monoclonally purified FVIII
7) 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
1) concomitant systemic treatment with drugs with immunosuppressive side effects (eg corticosteriods, if used more than 5 days every 3 months and/or at a dose of more than 2 mg/kg or 60 mg/day), azathiorpine, cyclophosphamide, high dose immunoglobulin)
2) use of protein A column or plasmapheresis
3) treatment with interferons
4) concomitant experimental treatment
5) previous history of myocardial infarction and/or cerebral stroke
6) high risk of cardiovascular, cerebrovascular or other thromboembolic events as deemed by the treating clinician
7) patients with known hypersenitivity to any of the constituents of any of the FVIII products that can be used in the study
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: 1) Maintenance of immune tolerance which is defined as an absence of relapse, assayed up to 12 months after the achievement of tolerance. <br>2) Time to achieve success, either partial or complete (as defined). <br>3) Safety, the assessment and evaluation of any adverse events occurring during the treatment.<br>4) 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.<br>
- Secondary Outcome Measures
Name Time Method