Guardian 4

Principal Investigator: Hassan Yaish
Keywords: Turoctocog Alfa , Hemophilia , Pediatric Department: Pediatric Administration
IRB Number: 00065271 Co Investigator: Richard Lemons
Specialty: Pediatric Hematology and Oncology
Sub Specialties: Hemophilia
Recruitment Status: Not yet recruiting

Contact Information

Tiffanie Hales

Brief Summary

Primary Objective

-To evaluate safety of turoctocog alfa in paediatric previously untreated patients (PUP) with
haemophilia A.
Secondary Objectives
-To evaluate efficacy and safety of turoctocog alfa in treatment of bleeds in paediatric PUP with
haemophilia A
-To evaluate preventive effect of turoctocog alfa on bleeds in paediatric PUP with haemophilia A
-To evaluate patient reported outcomes (PRO) impact of turoctocog alfa treatment
-To evaluate the health economic (HE) impact of turoctocog alfa treatment.

Inclusion Criteria

1. Informed consent obtained before any trial-related activities (trial-related activities are any procedure that would not have been performed during normal management of the patient)

2. Male patients diagnosed with congenital severe haemophilia A (FVIII </=1%)

3. Age < 6 years
4. No prior use of purified clotting FVIII products (previous exposure, equal to or less than 5 ED
to blood components, e.g. cryoprecipitate, fresh frozen plasma is accepted) including commercially available NovoEight/Novoeight.

Exclusion Criteria

1. Known or suspected allergy to hamster protein or intolerance to trial product(s) or related products

2. Previous participation in this trial defined as withdrawal after administration of trial product
3. Congenital or acquired coagulation disorders other than haemophilia A
4. Any history of FVIII inhibitor
5. On-going or planned treatment during the trial with immunomodulatory agents (e.g. intravenous
immunoglobulin (IVIG)), routine systemic corticosteroids)
6. Any disease or condition which, judged by the Investigator, could imply a potential hazard to
the patient, interfere with the trial participation or trial outcome including renal and/or liver failure
7. Unwillingness, language or other barriers precluding adequate understanding and/or cooperation
from parents and/or legal representatives and child
8. The receipt of any investigational product within 30 days prior to inclusion in this trial