This trial is active, not recruiting.

Condition hemophilia a with inhibitors
Treatments recombinant activated factor vii
Phase phase 2
Sponsor Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
Start date December 2010
End date February 2014
Trial size 50 participants
Trial identifier NCT01105546, ENJOIH 01, IND 14503


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.

United States No locations recruiting
Other countries No locations recruiting

Study Design

Allocation randomized
Endpoint classification efficacy study
Intervention model parallel assignment
Masking open label
Primary purpose treatment
prophylaxis with recombinant activated FVII 90 µg/kg/day i.v.
recombinant activated factor vii NovoSeven
90 µg/kg/day i.v.
(Active Comparator)
treatment of bleeding episodes with 270 µg/kg (first/single dose) or 90 µg/kg i.v. every 2-3 hours until bleeding resolution
recombinant activated factor vii NovoSeven
treatment of bleeding episodes with 270 µg/kg (first/single dose) or 90 µg/kg i.v. every 2-3 hours until bleeding resolution

Primary Outcomes

Total number of joint bleeds.
time frame: 18 months

Secondary Outcomes

Joint status evaluated by the Hemophilia Joint Health Score
time frame: 18 months
Number of adverse events and serious adverse events.
time frame: 18 months

Eligibility Criteria

Male participants up to 8 years old.

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.

Additional Information

Official title 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
Principal investigator Elena Santagostino, MD, PhD
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.
Trial information was received from ClinicalTrials.gov and was last updated in April 2013.
Information provided to ClinicalTrials.gov by Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico.