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

Institute of Hospitalization and Scientific Care (IRCCS)

Status and phase

Unknown

Phase 2

Conditions

Hemophilia A With Inhibitors

Treatments

Drug: recombinant activated factor VII

Study type

Interventional

Funder types

Other

Identifiers

NCT01105546

IND 14503 (Other Identifier)

ENJOIH 01

Details and patient eligibility

About

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.

Full 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.

Enrollment

50 estimated patients

Sex

Male

Ages

Under 8 years old

Volunteers

No 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.