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

Status

Indicates the current recruitment status or the expanded access status.
Unknown

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Anticipated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
50

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Total number of joint bleeds.
Time Frame: 18 months
18 months

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 1, 2010

Primary Completion (Anticipated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
February 1, 2014

Study Completion (Anticipated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
April 1, 2014

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 15, 2010

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 15, 2010

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
April 16, 2010

Study Record Updates

Last Update Posted (Estimate)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
April 8, 2013

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
April 5, 2013

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
April 1, 2013

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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