rFVIIa Prophylaxis in Children With Hemophilia A and Inhibitors

• Status: Unknown status
• Trial end date: 2014-04-01
• Target enrollment: 0
• Participant gender: Male

Summary

The study evaluates the efficacy and safety of a prophylactic treatment with recombinant activated FVII in reducing the frequency of joint bleeds and the development of joint damage in children with hemophilia A who develop high-titer inhibitors.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
• Treatments: Factor VIII

Criteria

Inclusion Criteria:

• Patients with hemophilia A who have been treated with factor VIII on demand or on prophylaxis and who have developed inhibitors to factor VIII

• ≤ 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.

• Candidates to start daily ITI with FVIII doses 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 (caregiver) of reconstituting and injecting the study drug

• Informed consent by parents or legal guardians.

Exclusion Criteria:

• ITI already started

• Known or suspected hypersensitivity to the active substance or to any of the excipients of the study drug

• Administration of any investigational product within 30 days prior to randomisation

• Other coagulation disorders than congenital hemophilia 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.