Clinical validation of Factor VIII allo-antibody assays in patients with severe hemophilia A (PSTOL 15).
Recruiting
- Conditions
- severe haemophilia A patients, low titer assay, factor VIII antibodies, negative Bethesda assay, pharmacokinetic
- Registration Number
- NL-OMON23098
- Lead Sponsor
- Radboud University Nijmegen Medical Centre
- Brief Summary
/A
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Not specified
- Target Recruitment
- 150
Inclusion Criteria
Inclusion criteria for patients with severe haemophilia A without inhibitors are:
1. Less than 1% factor VIII activity (severe clinical phenotype of haemophilia A);
Exclusion Criteria
Exclusion criteria for patients with severe haemophilia A without inhibitors are:
1. Known allergy to plasma proteins;
Study & Design
- Study Type
- Observational non invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method 1. To study the correlation between the antibody levels measured with the different assays and the pharmaco kinetic parameters;<br /><br>2. To calculate the sensitivity, specificity, negative and positive predictive value of the different assays with respect to the pharmaco kinetics of factor VIII.
- Secondary Outcome Measures
Name Time Method 1. Correlation of FVIII pharmaco kinetic parameters and inhibitor levels with von Willebrand factor. The von Willebrand factor may influence pharmacokinetic parameters of factor VIII as von Willebrand Factor (including ABO serology) is the chaperone molecule of factor VIII (Ref 13,14). Therefore it may also affect antibody levels;<br /><br>2. Correlation of FVIII pharmaco kinetic parameters and inhibitor levels with total and transformed á2-macroglobulin. Factor VIII and á2-macroglobulin share one receptor for clearing the proteins from circulation, the lipoprotein receptor-related protein (LRP). Therefore, á2-macroglobulin levels may influence the pharmacokinetic parameters of factor VIII, factor VIII antibodies as well as the correlation between both in humans as it does affect factor VIII kinetics in mice (ref 15,16,17).