rFVIIa Prophylaxis in Children With Hemophilia A and Inhibitors

NCT ID: NCT01105546

Last Updated: 2013-04-08

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2
• Total Enrollment: 50 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-12-31
• Study Completion Date: 2014-04-30

Brief 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.

Conditions

Hemophilia A With Inhibitors

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

prophylaxis

prophylaxis with recombinant activated FVII 90 µg/kg/day i.v.

Group Type: EXPERIMENTAL

recombinant activated factor VII

Intervention Type: DRUG

90 µg/kg/day i.v.

on demand treatment

treatment of bleeding episodes with 270 µg/kg (first/single dose) or 90 µg/kg i.v. every 2-3 hours until bleeding resolution

Group Type: ACTIVE_COMPARATOR

recombinant activated factor VII

Intervention Type: DRUG

treatment of bleeding episodes with 270 µg/kg (first/single dose) or 90 µg/kg i.v. every 2-3 hours until bleeding resolution

Interventions

recombinant activated factor VII

90 µg/kg/day i.v.

Intervention Type: DRUG

recombinant activated factor VII

treatment of bleeding episodes with 270 µg/kg (first/single dose) or 90 µg/kg i.v. every 2-3 hours until bleeding resolution

Intervention Type: DRUG

Other Intervention Names

NovoSeven; NovoSeven

Eligibility 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.

• Maximum Eligible Age: 8 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

OTHER

Sponsor Role: lead

Responsible Party

Elena Santagostino

MD

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Elena Santagostino, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Locations

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

Napoli, , 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

Countries

United States; France; Germany; Italy; Romania; Spain

Other Identifiers

IND 14503

Identifier Type: OTHER

Identifier Source: secondary_id

ENJOIH 01

Identifier Type: -

Identifier Source: org_study_id