Title
Survey of Inhibitors in Plasma-Product Exposed Toddlers
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
Phase
Phase 4Lead Sponsor
Fondazione Angelo Bianchi BonomiStudy Type
InterventionalStatus
Completed Results PostedIndication/Condition
Hemophilia AIntervention/Treatment
antihemophilic factor viii human ...Study Participants
303The primary objective of the study is to assess the immunogenicity of VWF/FVIII and of rFVIII concentrates by determining the frequency of inhibitor development in previously untreated patients (PUPs) or minimally blood component-treated (MBCTPs) in the first 50 EDs or in the first 3 years from enrollment, whichever occurs first.
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Patients meeting the enrollment criteria will be consecutively enrolled at each participating centre, randomized to be treated exclusively with a single FVIII product either plasma-derived or recombinant, and followed up until inhibitor development or until 50 exposure days (EDs) or 3 years from enrolment have elapsed, whichever comes first. Study products, belonging to the class of rFVIII concentrates and to the class of plasma-derived VWF/FVIII concentrates, will be provided for free to the patients for all the duration of the study
Maximum dosage : 50IU per kilo. 2-3 times per week or on demand during acute episode of bleeding
Maximum dosage : 50IU per kilo. 2-3 times per week or on demand during acute episode of bleeding
Plasma-derived vWF/FVIII
Inclusion Criteria: Male subjects Any ethnicity Age <6 years Severe haemophilia A (FVIII:C <1%), as confirmed at enrolment by the central laboratory. o Those patients diagnosed locally as severe but subsequently found to have FVIII levels >= 1% on testing at the central laboratory will be separately recorded in the screening list. Previously untreated (0 EDs to any FVIII concentrates 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. Exclusion Criteria: Previous history of FVIII inhibitor Other congenital or acquired bleeding defects 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. Concomitant congenital or acquired immunodeficiency Concomitant treatment with systemic immunosuppressive drugs Concomitant treatment with any investigational drug
| Event Type | Organ System | Event Term | pd vWF/FVIII | rFVIII |
|---|
Expressed with the numebr of patients for each group who developed FVIII inhibitors. PUPs: Previously Untreated Patients MBCTPs: Minimally Blood Component-Treated Patients
Number of participants for each group who developed transient inhibitors (this means, those inhibitors which disappeared spontaneously within 6 months without immunotolerance treatment).
Number of EDs: Number of Exposure Days (EDs) after which the inhibitors develop
Inhibitor Titre at Onset
