Development of antibody against FVIII (inhibitors) in Untreated Patients or Minimally Blood Component-Treated Patients when Exposed Factor concentrates which can be made from human blood (plasma-derived) or manufactured in laboratory using genetically engineered cells that carry a human factor gene (recombinant): A non-commercial, international study with lots of sites and it is known which of the two by random assigned drugs will be take
- Conditions
- Inhibitor Development in Previously Untreated Patients (PUPs) or Minimally Blood Component-Treated Patients (MBCTPs) when Exposed to plasma-derived von Willebrand Factor-Containing Factor VIII (VWF/FVIII) Concentrates and to Recombinant Factor VIII (rFVIII) Concentrates: An Independent, International, Multicentre, Prospective, Controlled, Randomised, Open Label, Clinical TrialMedDRA version: 16.1Level: LLTClassification code 10018937Term: Haemophilia ASystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2009-011186-88-SK
- Lead Sponsor
- FONDAZIONE 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
- 300
•Male subjects
•Any ethnicity
•Age <6 years
•Severe haemophilia A (FVIII:C <1%), as confirmed by the central laboratory
oPatients with FVIII levels >1% and < 2% will be separately recorded in the screening list
•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
oPatients not meeting these criteria will be separately recorded in the screening list
•Negative inhibitor measurement at both local and central laboratory at screening
•Ability to comply with study requirements
•Signed informed consent of legal tutors
oPatients who will not accept to enter into the study or to be randomized will be separately recorded
Are the trial subjects under 18? yes
Number of subjects for this age range: 300
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
•Plasma FVIII level =1%, as assayed at the central laboratory
oThose patients originally diagnosed locally as severe but subsequently found to have FVIII levels ranging from 1% to 2% on testing at the central laboratory will be separately recorded in the screening list.
•Previous history of FVIII inhibitor
•Other congenital or acquired bleeding defects
•Concomitant congenital or acquired immunodeficiency
•Concomitant treatment with systemic immunosuppressive drugs
•Concomitant treatment with any investigational drug
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method