rFVIIa Prophylaxis in Children With Hemophilia A and Inhibitors (ENJOIH)

April 5, 2013 updated by: Elena Santagostino, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

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

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.

Study Overview

Status

Unknown

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Anticipated)

50

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • France
      • Lyon, France, 69003
        • Haemophilia Comprehensive Care Centre, Edouard Herriot University Hospital
  • Germany
      • Berlin, Germany, 10249
        • Vivantes Klinikum im Friedrichshain Haemophilia Care Center, Medical Center
      • Bremen, Germany, 28205
        • Klinikum Bremen-Mitte, Prof.-Hess-Kinderklinik
      • Frankfurt/M, Germany, 60590
        • Klinikum der Johann Wolfgang Goethe-Universität
  • Italy
      • Florence, Italy, 50134
        • Azienda Ospedaliero-Universitaria Careggi Agenzia per l'emofilia e Centro di riferimento regionale per i disordini congeniti del sanguinamento
      • Milan, Italy, 20122
        • Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
      • Napoli, Italy, 80144
        • Centro Emofilia e Trombosi Unità Operativa di Ematologia Ospedale San Giovanni Bosco
  • Romania
      • Bucarest, Romania
        • National Institute for Transfusional Hematology
      • Timisoara, Romania, 300011
        • Spitaluc Clinic de Urgenta pentru Copii Louis Turcanu, University of Medicine and Pharmacy
  • Spain
      • Barcellona, Spain, 08035
        • Unitat Hemofilia, Hospital Vall d'Hebron
      • Madrid, Spain, 28046
        • Centro de Hemofilia, Hospital Universitario La Paz
      • Valencia, Spain, 46009
        • Unidad de Coagulopatias Congenitas, Hospital Universitario la Fe
  • United States
    • California
      • Los Angeles, California, United States, 90027
        • Children's Hospital Los Angeles
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory University
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Children's Mercy Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 8 years (Child)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: prophylaxis
prophylaxis with recombinant activated FVII 90 µg/kg/day i.v.
Drug: recombinant activated factor VII
90 µg/kg/day i.v.
Other Names:
  • NovoSeven
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
Other Names:
  • NovoSeven
Active Comparator: 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
Drug: recombinant activated factor VII
90 µg/kg/day i.v.
Other Names:
  • NovoSeven
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
Other Names:
  • NovoSeven

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Total number of joint bleeds.
Time Frame: 18 months
18 months

Secondary Outcome Measures

Outcome Measure
Time Frame
Joint status evaluated by the Hemophilia Joint Health Score
Time Frame: 18 months
18 months
Number of adverse events and serious adverse events.
Time Frame: 18 months
18 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Investigators

  • Principal Investigator: Elena Santagostino, MD, PhD, Fondazione IRCCS Cà Granda, Ospedale Maggiore Policlinico, Milano

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

December 1, 2010

Primary Completion (Anticipated)

February 1, 2014

Study Completion (Anticipated)

April 1, 2014

Study Registration Dates

First Submitted

April 15, 2010

First Submitted That Met QC Criteria

April 15, 2010

First Posted (Estimate)

April 16, 2010

Study Record Updates

Last Update Posted (Estimate)

April 8, 2013

Last Update Submitted That Met QC Criteria

April 5, 2013

Last Verified

April 1, 2013

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ENJOIH 01
  • IND 14503 (Other Identifier: FDA)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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