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 Trial. - SIPPET
- Conditions
- PEDIATRIC MALE PATIENTS BETWEEN 0 AND 6 YEARS WITH SEVERE HAEMOPHILIA AMedDRA version: 9.1Level: LLTClassification code 10018937
- Registration Number
- EUCTR2009-011186-88-IT
- Lead Sponsor
- FONDAZIONE CENTRO EMOFILIA E TROMBOSI ANGELO BIANCHI BONOMI
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 300
Male subjects Any ethnicity Age <6 years Severe haemophilia A (FVIII:C <1%), as confirmed by the central laboratory o Patients 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 o Patients 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 o Patients 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:
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 o Those 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