rFVIIa Prophylaxis in Children With Hemophilia A and Inhibitors

NCT01105546 | Unknown Phase 2 DMC

• 2 other identifiers: ENJOIH 01IND 14503
• Study Type: interventional
• Target: 50
• Locations: 6 countries
• Sites: 15

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.

Conditions

Hemophilia A With Inhibitors

Keywords

hemophilia A; inhibitors; prophylaxis; immune tolerance induction

Outcome Measures

Primary Outcomes (1)

• Total number of joint bleeds.

  18 months

Secondary Outcomes (2)

• Joint status evaluated by the Hemophilia Joint Health Score

  18 months

• Number of adverse events and serious adverse events.

  18 months

Study Arms (2)

prophylaxis

EXPERIMENTAL

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

Drug: recombinant activated factor VII

on demand treatment

ACTIVE COMPARATOR

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

Interventions

recombinant activated factor VII DRUG

90 µg/kg/day i.v.

Also known as: NovoSeven

prophylaxis

Eligibility Criteria

• Age: Up to 8 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico: lead

Study Sites (15)

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

Location

Emory University

Atlanta, Georgia, 30322, United States

Location

Children's Mercy Hospital

Kansas City, Missouri, 64108, United States

Location

Haemophilia Comprehensive Care Centre, Edouard Herriot University Hospital

Lyon, 69003, France

Location

Vivantes Klinikum im Friedrichshain Haemophilia Care Center, Medical Center

Berlin, Germany, 10249, Germany

Location

Klinikum Bremen-Mitte, Prof.-Hess-Kinderklinik

Bremen, 28205, Germany

Location

Klinikum der Johann Wolfgang Goethe-Universitat

Frankfurt/M, 60590, Germany

Location

Angelo Bianchi Bonomi Hemophilia and Thrombosis Center, Fondazione IRCCS Cà Granda Ospedale Maggiore Policlinico

Milan, Italy, 20122, Italy

Location

Azienda Ospedaliero-Universitaria Careggi Agenzia per l'emofilia e Centro di riferimento regionale per i disordini congeniti del sanguinamento

Florence, 50134, Italy

Location

Centro Emofilia e Trombosi Unità Operativa di Ematologia Ospedale San Giovanni Bosco

Napoli, 80144, Italy

Location

National Institute for Transfusional Hematology

Bucharest, Romania, Romania

Location

Spitaluc Clinic de Urgenta pentru Copii Louis Turcanu, University of Medicine and Pharmacy

Timișoara, Romania, 300011, Romania

Location

Unitat Hemofilia, Hospital Vall d'Hebron

Barcelona, Spain, 08035, Spain

Location

Centro de Hemofilia, Hospital Universitario La Paz

Madrid, Spain, 28046, Spain

Location

Unidad de Coagulopatias Congenitas, Hospital Universitario la Fe

Valencia, Spain, 46009, Spain

Location

MeSH Terms

Conditions

Hemophilia A

Interventions

recombinant FVIIa

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Elena Santagostino, MD, PhD

  Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD

Study Record Dates

• First Submitted: April 15, 2010
• First Posted: April 16, 2010
• Study Start: December 1, 2010
• Primary Completion: February 1, 2014
• Study Completion: April 1, 2014
• Last Updated: April 8, 2013

Record last verified: 2013-04

Locations

IT (3); DE (3); US (3); ES (3); FR (1); RO (2)