An Investigator-initiated study on rFVIIa prophylaxis in children with haemophilia A and inhibitors - ENJOIH 01

• Conditions: Hemophilia A; MedDRA version: 9.1Level: LLTClassification code 10056492Term: Haemophilia A with anti factor VIII

• Registration Number: EUCTR2009-010172-21-DE
• Lead Sponsor: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-09-15
• Last Update Posted: 19 August 2013

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 50

Inclusion Criteria

•Male patients with haemophilia A who have been treated with FVIII on demand or on prophylaxis and who have developed inhibitors to FVIII.
•Age < 8 years.
•= 2 years from the time of first inhibitor detection.
•High-responding inhibitors (historical peak > 5 BU/mL) and known anamnestic response in case of negative inhibitor titre.
•Patient is candidate for daily ITI treatment with doses of FVIII ranging from 50 IU/kg/day to 200 IU/kg/day.
•Maximal two bleedings in the same joint within the last 6 months before entering the study or maximal six joint bleeds in the same joint within 2 years.
•Adequate venous access for daily infusion and capable (patient or caregiver) of reconstituting and injecting the study drug (as judged by the Investigator).
•Informed consent by parents or legal guardians.

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

•ITI already started.
•Known or suspected hypersensitivity to the active substance, or to any of the excipients of the study drug, or to mouse, hamster or bovine protein..
•Administration of any investigational product within 30 days prior to randomisation.
•Other coagulation disorders than congenital haemophilia A.
•Family history of thrombosis at an early age (< 40 years), known thrombophilia, any previous thrombosis including catheter-related deep vein thrombosis, previous neonatal thrombosis.
•Known pseudo tumours.
•Known severe liver disease.
•Platelet count < 50,000 platelets/µL at screening.
•Surgery within one month or planned major and/or orthopaedic surgery.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified