rFVIIa Prophylaxis in Children With Hemophilia A and Inhibitors
This trial is active, not recruiting.
|Condition||hemophilia a with inhibitors|
|Treatments||recombinant activated factor vii|
|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|
|Los Angeles, CA||Children's Hospital Los Angeles||no longer recruiting|
|Atlanta, GA||Emory University||no longer recruiting|
|Kansas City, MO||Children's Mercy Hospital||no longer recruiting|
|Lyon, France||Haemophilia Comprehensive Care Centre, Edouard Herriot University Hospital||no longer recruiting|
|Berlin, Germany||Vivantes Klinikum im Friedrichshain Haemophilia Care Center, Medical Center||no longer recruiting|
|Bremen, Germany||Klinikum Bremen-Mitte, Prof.-Hess-Kinderklinik||no longer recruiting|
|Frankfurt/M, Germany||Klinikum der Johann Wolfgang Goethe-Universitat||no longer recruiting|
|Florence, Italy||Azienda Ospedaliero-Universitaria Careggi Agenzia per l'emofilia e Centro di riferimento regionale per i disordini congeniti del sanguinamento||no longer recruiting|
|Milan, Italy||Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico||no longer recruiting|
|Napoli, Italy||Centro Emofilia e Trombosi Unità Operativa di Ematologia Ospedale San Giovanni Bosco||no longer recruiting|
|Bucarest, Romania||National Institute for Transfusional Hematology||no longer recruiting|
|Timisoara, Romania||Spitaluc Clinic de Urgenta pentru Copii Louis Turcanu, University of Medicine and Pharmacy||no longer recruiting|
|Barcellona, Spain||Unitat Hemofilia, Hospital Vall d'Hebron||no longer recruiting|
|Madrid, Spain||Centro de Hemofilia, Hospital Universitario La Paz||no longer recruiting|
|Valencia, Spain||Unidad de Coagulopatias Congenitas, Hospital Universitario la Fe||no longer recruiting|
|Endpoint classification||efficacy study|
|Intervention model||parallel assignment|
Total number of joint bleeds.
time frame: 18 months
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
Male participants up to 8 years old.
- 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.
- 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.
|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.|
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