Survey of Inhibitors in Plasma-Product Exposed Toddlers

• Status: Completed
• Trial end date: 2015-05-01
• Target enrollment: 0
• Participant gender: Male

Summary

The 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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• Phase: Phase 4
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Fondazione Angelo Bianchi Bonomi
• Collaborator: Sintesi Research Srl
• Treatments: Factor VIII

Criteria

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