Survey of Inhibitors in Plasma-Product Exposed Toddlers
- Conditions
- Hemophilia A.Hereditary factor VIII deficiency
- Registration Number
- IRCT201104106161N1
- Lead Sponsor
- Fondazione Angelo Bianchi Bonomi
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Complete
- Sex
- Male
- Target Recruitment
- 300
1-Male subjects; 2-Any ethnicity; 3-Age <6 years; 4-Severe haemophilia A (FVIII:C <1%), as confirmed by the central laboratory; 5-Previously untreated (0 EDs to any FVIII concentrate or blood products) or minimally treated (<5 EDs) with blood components, namely whole blood, fresh frozen plasma, packed red blood cells, platelets or cryoprecipitate; 6-Negative inhibitor measurement at both local and central laboratory at screening; 7-Ability to comply with study requirements; 8-Signed informed consent of legal tutors. Exclusion criteria:
1-Plasma FVIII level =1%, as assayed at the central laboratory; 2-Previous history of FVIII inhibitor; 3-Other congenital or acquired bleeding defects; 4-Concomitant congenital or acquired immunodeficiency; 5-Concomitant treatment with systemic immunosuppressive drugs; 6-Concomitant treatment with any investigational drug.
Not provided
Study & Design
- Study Type
- interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method To assess the immunogenicity of plasma derived VWF/FVIII and rFVIII concentrates. Timepoint: in the first 50 EDs or in the first 3 years from enrolment. Method of measurement: by determining the frequency of inhibitor development.
- Secondary Outcome Measures
Name Time Method To evaluate modality of occurrence of inhibitors (number of EDs, titre at onset, etc). Timepoint: in the first 50 EDs or in the first 3 years from enrolment, whichever comes first. Method of measurement: record and analysis of characteristics during the treatment course.