rFVIIa Prophylaxis in Children With Hemophilia A and Inhibitors

Hemophilia A With Inhibitors Clinical Trial

— 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

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT01105546
• Other study ID #: ENJOIH 01
• Secondary ID: IND 14503
• Status: Active, not recruiting
• Phase: Phase 2
• First received: April 15, 2010
• Last updated: April 5, 2013
• Start date: December 2010
• Est. completion date: April 2014

Study information

• Verified date: April 2013
• Source: Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico
• Contact: n/a
• Is FDA regulated: No
• Health authority: Italy: Ethics CommitteeItaly: National Monitoring Centre for Clinical Trials - Ministry of HealthItaly: The Italian Medicines AgencyUnited States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 50
• Est. completion date: April 2014
• Est. primary completion date: February 2014
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A to 8 Years

Eligibility

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 Design

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

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia A With Inhibitors

Intervention

Drug:
• recombinant activated factor VII
90 µg/kg/day i.v.
• 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

Locations

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

Sponsors (1)

Lead Sponsor	Collaborator
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Countries where clinical trial is conducted

United States,  France,  Germany,  Italy,  Romania,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Total number of joint bleeds.		18 months	No
Secondary	Joint status evaluated by the Hemophilia Joint Health Score		18 months	No
Secondary	Number of adverse events and serious adverse events.		18 months	Yes