Principal Investigator: Hassan Yaish
Keywords: haemophilia A , prophylaxis , bleeding episodes Department: Pediatric Administration
IRB Number: 00102282
Specialty: Pediatrics, General
Sub Specialties:
Recruitment Status: Recruiting

Contact Information

Rebbecca Hanshew

Brief Summary

 This is a multi-national, non-randomised, open label phase 3 trial investigating the safety (including immunogenicity) and efficacy of N8-GP in prophylaxis and treatment of bleeding episodes in a paediatric population of PUPs (age below 6 years) with severe haemophilia A (factor VIII baseline level < 1%). There will only be one treatment arm and no comparator.

Primary Objective

  • To evaluate immunogenicity of N8-GP (turoctocog alfa pegol) in previously untreated patients (PUPs) with severe haemophilia A

Primary Endpoint

  • Incidence of FVIII inhibitors

Secondary Objectives

  • To evaluate safety other than immunogenicity of N8-GP (turoctocog alfa pegol) in PUPs with severe haemophilia A
  • To evaluate efficacy of N8-GP (turoctocog alfa pegol) in PUPs with severe haemophilia A
    • in long-term prophylaxis treatment (bleeding preventive effect)
    • in the treatment of bleeding episodes


Inclusion Criteria

  1. Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial
  2. Male, age < 6 years of age at the time of signing informed consent
  3. Diagnosis of severe haemophilia A (FVIII activity level < 1%) based on medical records or central laboratory results
  4. No prior use of purified factor VIII containing clotting products (5 previous exposure days to blood components are acceptable)

Exclusion Criteria

  1. Any history of FVIII inhibitor (defined by medical records)
  2. Known or suspected hypersensitivity to trial product or related products
  3. Previous participation in this trial. Participation is defined as first dose administered of trial product
  4. Receipt of any investigational medicinal product within 30 days before screening
  5. Congenital or acquired coagulation disorder other than haemophilia A
  6. Any chronic disorder or severe disease which, in the opinion of the Investigator, might jeopardise patient’s safety or compliance with the protocol
  7. Patient’s parent(s)/LAR(s) mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation