rFVIIa Prophylaxis in Children With Hemophilia A and Inhibitors

Status unconfirmed · Phase 2

Conditions studied: Hemophilia A With Inhibitors

In brief

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.

Key facts

Study ID

NCT01105546

Run by

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

People needed

50

Starts

2010-12-01

Expected to finish

2014-04-01

Last updated by the study team

2013-04-08

Who can join

Age: any, up to 8. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• 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.

You may not qualify if…

• 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.

Where it is running

• 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
• National Institute for Transfusional Hematology — Bucharest, Romania, Romania
• Spitaluc Clinic de Urgenta pentru Copii Louis Turcanu, University of Medicine and Pharmacy — Timișoara, Romania, Romania
• Unitat Hemofilia, Hospital Vall d'Hebron — Barcelona, Spain, Spain
• Centro de Hemofilia, Hospital Universitario La Paz — Madrid, Spain, Spain
• Unidad de Coagulopatias Congenitas, Hospital Universitario la Fe — Valencia, Spain, Spain

Full record on ClinicalTrials.gov

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