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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Elena Santagostino, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
ClinicalTrials.gov Identifier:
NCT01105546
First received: April 15, 2010
Last updated: April 5, 2013
Last verified: April 2013
  Purpose

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.


Condition Intervention Phase
Hemophilia A With Inhibitors
Drug: recombinant activated factor VII
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico:

Primary Outcome Measures:
  • Total number of joint bleeds. [ Time Frame: 18 months ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Joint status evaluated by the Hemophilia Joint Health Score [ Time Frame: 18 months ] [ Designated as safety issue: No ]
  • Number of adverse events and serious adverse events. [ Time Frame: 18 months ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 50
Study Start Date: December 2010
Estimated Study Completion Date: April 2014
Estimated Primary Completion Date: February 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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 Name: 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
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 Name: NovoSeven

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.

  Eligibility

Ages Eligible for Study:   up to 8 Years
Genders Eligible for Study:   Male
Accepts Healthy Volunteers:   No
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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01105546

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

No publications provided

Responsible Party: Elena Santagostino, MD, Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
ClinicalTrials.gov Identifier: NCT01105546     History of Changes
Other Study ID Numbers: ENJOIH 01, IND 14503
Study First Received: April 15, 2010
Last Updated: April 5, 2013
Health Authority: Italy: Ethics Committee
Italy: National Monitoring Centre for Clinical Trials - Ministry of Health
Italy: The Italian Medicines Agency
United States: Food and Drug Administration

Keywords provided by Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico:
hemophilia A
inhibitors
prophylaxis
immune tolerance induction

Additional relevant MeSH terms:
Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants
Hematologic Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on September 14, 2014