NCT01064284

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

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 303 (actual)
• Sponsor: Fondazione Angelo Bianchi Bonomi · Academic / Other
• Sex: Male
• Age: 1 Minute – 6 Years
• Healthy volunteers: Not accepted

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

Conditions

• Hemophilia A

Interventions

Type	Name	Description
DRUG	PLASMA DERIVED Factor VIII	Maximum dosage : 50IU per kilo. 2-3 times per week or on demand during acute episode of bleeding
DRUG	Recombinant FVIII	Maximum dosage : 50IU per kilo. 2-3 times per week or on demand during acute episode of bleeding

Timeline

• Start date: 2010-01-01
• Primary completion: 2015-05-01
• Completion: 2015-05-01
• First posted: 2010-02-08
• Last updated: 2017-08-25
• Results posted: 2017-04-07

Locations

48 sites across 14 countries: United States, Argentina, Austria, Brazil, Chile, Egypt, India, Iran, Italy, Mexico, Saudi Arabia, South Africa, Spain, Turkey (Türkiye)