Survey of Inhibitors in Plasma-Product Exposed Toddlers

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

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