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

Primary Purpose

Hemophilia A With Inhibitors

Status
Unknown status
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
recombinant activated factor VII
recombinant activated factor VII
Sponsored by
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Hemophilia A With Inhibitors focused on measuring hemophilia A, inhibitors, prophylaxis, immune tolerance induction

Eligibility Criteria

undefined - 8 Years (Child)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • Children's Hospital Los Angeles
  • Emory University
  • Children's Mercy Hospital
  • Haemophilia Comprehensive Care Centre, Edouard Herriot University Hospital
  • Vivantes Klinikum im Friedrichshain Haemophilia Care Center, Medical Center
  • Klinikum Bremen-Mitte, Prof.-Hess-Kinderklinik
  • Klinikum der Johann Wolfgang Goethe-Universitat
  • Azienda Ospedaliero-Universitaria Careggi Agenzia per l'emofilia e Centro di riferimento regionale per i disordini congeniti del sanguinamento
  • Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
  • Centro Emofilia e Trombosi Unità Operativa di Ematologia Ospedale San Giovanni Bosco
  • National Institute for Transfusional Hematology
  • Spitaluc Clinic de Urgenta pentru Copii Louis Turcanu, University of Medicine and Pharmacy
  • Unitat Hemofilia, Hospital Vall d'Hebron
  • Centro de Hemofilia, Hospital Universitario La Paz
  • Unidad de Coagulopatias Congenitas, Hospital Universitario la Fe

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

prophylaxis

on demand treatment

Arm Description

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

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

Outcomes

Primary Outcome Measures

Total number of joint bleeds.

Secondary Outcome Measures

Joint status evaluated by the Hemophilia Joint Health Score
Number of adverse events and serious adverse events.

Full Information

First Posted
April 15, 2010
Last Updated
April 5, 2013
Sponsor
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
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1. Study Identification

Unique Protocol Identification Number
NCT01105546
Brief Title
rFVIIa Prophylaxis in Children With Hemophilia A and Inhibitors
Acronym
ENJOIH
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
Study Type
Interventional

2. Study Status

Record Verification Date
April 2013
Overall Recruitment Status
Unknown status
Study Start Date
December 2010 (undefined)
Primary Completion Date
February 2014 (Anticipated)
Study Completion Date
April 2014 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A With Inhibitors
Keywords
hemophilia A, inhibitors, prophylaxis, immune tolerance induction

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
50 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
prophylaxis
Arm Type
Experimental
Arm Description
prophylaxis with recombinant activated FVII 90 µg/kg/day i.v.
Arm Title
on demand treatment
Arm Type
Active Comparator
Arm Description
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
Intervention Name(s)
recombinant activated factor VII
Other Intervention Name(s)
NovoSeven
Intervention Description
90 µg/kg/day i.v.
Intervention Type
Drug
Intervention Name(s)
recombinant activated factor VII
Other Intervention Name(s)
NovoSeven
Intervention Description
treatment of bleeding episodes with 270 µg/kg (first/single dose) or 90 µg/kg i.v. every 2-3 hours until bleeding resolution
Primary Outcome Measure Information:
Title
Total number of joint bleeds.
Time Frame
18 months
Secondary Outcome Measure Information:
Title
Joint status evaluated by the Hemophilia Joint Health Score
Time Frame
18 months
Title
Number of adverse events and serious adverse events.
Time Frame
18 months

10. Eligibility

Sex
Male
Maximum Age & Unit of Time
8 Years
Accepts Healthy Volunteers
No
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Elena Santagostino, MD, PhD
Organizational Affiliation
Fondazione IRCCS Cà Granda, Ospedale Maggiore Policlinico, Milano
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's Hospital Los Angeles
City
Los Angeles
State/Province
California
ZIP/Postal Code
90027
Country
United States
Facility Name
Emory University
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Children's Mercy Hospital
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Facility Name
Haemophilia Comprehensive Care Centre, Edouard Herriot University Hospital
City
Lyon
ZIP/Postal Code
69003
Country
France
Facility Name
Vivantes Klinikum im Friedrichshain Haemophilia Care Center, Medical Center
City
Berlin
ZIP/Postal Code
10249
Country
Germany
Facility Name
Klinikum Bremen-Mitte, Prof.-Hess-Kinderklinik
City
Bremen
ZIP/Postal Code
28205
Country
Germany
Facility Name
Klinikum der Johann Wolfgang Goethe-Universitat
City
Frankfurt/M
ZIP/Postal Code
60590
Country
Germany
Facility Name
Azienda Ospedaliero-Universitaria Careggi Agenzia per l'emofilia e Centro di riferimento regionale per i disordini congeniti del sanguinamento
City
Florence
ZIP/Postal Code
50134
Country
Italy
Facility Name
Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico
City
Milan
ZIP/Postal Code
20122
Country
Italy
Facility Name
Centro Emofilia e Trombosi Unità Operativa di Ematologia Ospedale San Giovanni Bosco
City
Napoli
ZIP/Postal Code
80144
Country
Italy
Facility Name
National Institute for Transfusional Hematology
City
Bucarest
Country
Romania
Facility Name
Spitaluc Clinic de Urgenta pentru Copii Louis Turcanu, University of Medicine and Pharmacy
City
Timisoara
ZIP/Postal Code
300011
Country
Romania
Facility Name
Unitat Hemofilia, Hospital Vall d'Hebron
City
Barcellona
ZIP/Postal Code
08035
Country
Spain
Facility Name
Centro de Hemofilia, Hospital Universitario La Paz
City
Madrid
ZIP/Postal Code
28046
Country
Spain
Facility Name
Unidad de Coagulopatias Congenitas, Hospital Universitario la Fe
City
Valencia
ZIP/Postal Code
46009
Country
Spain

12. IPD Sharing Statement

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rFVIIa Prophylaxis in Children With Hemophilia A and Inhibitors

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