SIPPET STUDY (Survey of Inhibitors in Plasma-Product Exposed Toddlers)
Research type
Research Study
Full 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
IRAS ID
51291
Contact name
Jayanthi Alamelu
Sponsor organisation
FONDAZIONE CENTRO EMOFILIA E TROMBOSI ANGELO BIANCHI BONOMI
Eudract number
2009-011186-88
Clinicaltrials.gov Identifier
Research summary
The study is an interventional, non-profit, Investigator-initiated, pilot, open label, parallel group, randomised, comparator, multicentre and international, Phase IV clinical trial on inhibitor frequency in patientspreviously untreated (PUPs) or minimally blood component-treated (MBCTPs) when exposed to plasma-derived VWF/FVIII concentrates or to recombinant(r) FVIII concentrates.Haemophilia A is a rare congenital bleeding disorder characterised by a deficient coagulation protein, named factor VIII, and affecting 1 out of 10,000 people. One of the major complications of the treatment of Haemophilia A is the development of antibodies against factor VIII (called inhibitors) that inhibit factor VIII activity (FVIII:C)leading to a poor control of bleeding.The risk of inhibitor production is higher in patients with severe Haemophilia A than in patients with mild or moderate disease. Inhibitor development is the most challenging complication of haemophilia treatment. It is important to know whether or not plasma-derived and recombinant products are associated with a different risk of inhibitor development in previously untreated patients(PUPs). Unfortunately, no randomized clinical trials are available to date to provide the evidence needed.This study aims to assess the immunogenicity of plasma-derived VWF/FVIII and that of rFVIII concentrates by determining the frequency of inhibitor development in PUPs and MBCTPs in the first 50 exposure days (EDs) or in the first 3 years from enrolment, whichever comes first.This study aims to test the hypothesis that plasma-derived VWF/FVIII products are less immunogenic than rFVIII products. For this reasons two classes of products, and not two specific products belonging to these two classes, will be compared. patients will be randomised to receive one product from the two classes of products that will be evaluated. The brand name belonging to the assigned class will be selected depending on the products marketed and available by class in each participating country.
REC name
London - Riverside Research Ethics Committee
REC reference
10/H0706/39
Date of REC Opinion
17 Sep 2010
REC opinion
Further Information Favourable Opinion