An Investigator-sponsored study on rFVIIa prophylaxis in children with haemophilia A and inhibitorsEstudio promovido por el investigador sobre profilaxis con rFVIIa en niños con hemofilia A e inhibidores - ENJOIH 01

• Conditions: Hemofilia A; MedDRA version: 9.1Level: LLTClassification code 10056492Term: Haemophilia A with anti factor VIII

• Registration Number: EUCTR2009-010172-21-ES
• Lead Sponsor: Elena Santagostino

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-06-25
• Last Update Posted: 26 August 2013

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Male
• Target Recruitment: 50

Inclusion Criteria

- Patients with haemophilia A who have been treated with factor VIII on-demand or on prophylaxis and who have developed inhibitors to factor VIII.
- Age < 8 years.
- = or < 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 ITI.
- 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 (patient or guardian) of reconstituting and injecting the study drug (as judged by the nvestigator).
- Informed consent by parents or guardians.
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

- ITI already started.
- Known or suspected allergy to the study drug or any of its components.
- Administration of any investigational product within 30 days prior to randomisation.
- Other coagulation disorders than congenital haemophilia 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/mcL at screening.
- Surgery within one month or planned major and/or orthopaedic surgery.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: The objective of this study is to evaluate the efficacy and safety of a prophylactic regimen with rFVIIa in reducing the frequency of joint bleeds and the development of signs of joint damage including synovitis, as measured by the Hemophilia Joint Health Score (HJHS), in children with haemophilia A who develop high-responding inhibitors.;Secondary Objective: not applicable;Primary end point(s): Clinical outcome will be evaluated at Month 18 (final visit), or earlier if the patient completes the study before, i.e., if factor VIII recovery is = or > 25%.<br><br>Primary efficacy endpoints:<br>- Total number of joint bleeds<br>- Number of re-bleeds in the same joint (re-bleed is defined as bleed occurring in the same joint within 72 hours of last treatment given).<br>- Time interval between haemorrhages in the same joint.