Randomised Study of First Time 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
- Conditions
- Patients with severe Haemophilia A with a high responding inhibitor (more than 5 BU/ml)
- Registration Number
- EUCTR2008-007016-15-GB
- Lead Sponsor
- niversity of Milan
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- A
- Sex
- Male
- Target Recruitment
- 148
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) At least one of the following risk factors for ITI failure
- peak inhibitor titre more than 200 BU/ml
- inhibitor titre at start of treatment more than 10 BU/ml
- more than 7 years of age
- more than 2 years between inhibitor occurrence and ITI
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 corticosteroids, if used more 5 days every 3 months and/or at a dose of more than 2mg/kg or 60mg/day), azathioprine, cyclophosphamide, high dose immunoglobulin)
2) use of a protein A column or plasmapheresis
3) treatment with interferons
4) concomitant experimental treatment
5) previous ITI attempt
6) previous history of myocardial infarction and/or cerebral stroke
7) high risk of cardiovascular, cerebrovascular or other thrombembolic events as deemed by the treating physician
8) patients with known hypersensitivity to anu of the 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 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 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 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 concentrates
- Secondary Outcome Measures
Name Time Method