rFVIIa Prophylaxis in Children With Hemophilia A and Inhibitors

An Investigator-initiated Study on rFVIIa Prophylaxis in Children With Hemophilia A and Inhibitors - European Initiative to Prevent Joint Damage in Hemophilia A Children With Inhibitors

NCT01105546•Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

View on ClinicalTrials.gov

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.

Detailed Description

This is a multicentre, randomised, controlled study designed to gain evidence of the advantage of the prophylactic, daily treatment with recombinant activated FVII as compared to the conventional on demand therapy in reducing the bleeding frequency and preserving the orthopaedic status in hemophilic children with inhibitors.

Eligibility

Age

0 - 8 years

Sex

MALE

Healthy Volunteers

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.

Locations (15)

Children's Hospital Los Angeles

Los Angeles, California, United States

Emory University

Atlanta, Georgia, United States

Children's Mercy Hospital

Kansas City, Missouri, United States

Haemophilia Comprehensive Care Centre, Edouard Herriot University Hospital

Lyon, France

Vivantes Klinikum im Friedrichshain Haemophilia Care Center, Medical Center

Berlin, Germany, Germany

Klinikum Bremen-Mitte, Prof.-Hess-Kinderklinik

Bremen, Germany

Klinikum der Johann Wolfgang Goethe-Universitat

Frankfurt/M, Germany

Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

Milan, Italy, Italy

Azienda Ospedaliero-Universitaria Careggi Agenzia per l'emofilia e Centro di riferimento regionale per i disordini congeniti del sanguinamento

Florence, Italy

Centro Emofilia e Trombosi Unità Operativa di Ematologia Ospedale San Giovanni Bosco

Naples, Italy

Key Dates

Start Date

December 1, 2010

Primary Completion Date

February 1, 2014

Completion Date

April 1, 2014

Last Updated

April 8, 2013

Enrollment

ESTIMATED participants

Conditions

Hemophilia A With Inhibitors

Interventions

recombinant activated factor VII

DRUG

recombinant activated factor VII

DRUG