Brief Title
Survey of Inhibitors in Plasma-Product Exposed Toddlers
Official Title
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
Brief 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. .
Detailed Description
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
Study Phase
Phase 4
Study Type
Interventional
Primary Outcome
To Assess the Immunogenicity of Plasma Derived VWF/FVIII and rFVIII Concentrates by Determining the Frequency of Inhibitor Development in the First 50 EDs or in the First 3 Years From Enrolment, Whichever Comes First in PUPs and MBCTs
Secondary Outcome
To Evaluate the Anamnestic Response of Inhibitor Patients
Condition
Hemophilia A
Intervention
PLASMA DERIVED Factor VIII
Study Arms / Comparison Groups
PLASMA DERIVED Factor VIII
Description: Plasma-derived vWF/FVIII
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
303
Start Date
January 2010
Completion Date
May 2015
Primary Completion Date
May 2015
Eligibility 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
Gender
Male
Ages
1 Minute - 6 Years
Accepts Healthy Volunteers
No
Contacts
Pier M. Mannucci, Professor, ,
Location Countries
Argentina
Location Countries
Argentina
Administrative Informations
NCT ID
NCT01064284
Organization ID
ABB - 09 - 001
Secondary IDs
2009-011186-88
Responsible Party
Sponsor
Study Sponsor
Fondazione Angelo Bianchi Bonomi
Collaborators
Sintesi Research Srl
Study Sponsor
Pier M. Mannucci, Professor, Principal Investigator, Fondazione Ca' Granda Ospedale Maggiore Policlinico Milano
Verification Date
July 2017