Efficacy and Safety of NNC 0078-0000-0007 in Patients With Congenital Haemophilia and Inhibitors

Efficacy and Safety of NNC 0078-0000-0007 in Treatment of Acute Bleeding Episodes in Patients With Congenital Haemophilia and Inhibitors

This trial is conducted globally. The purpose of this trial is to confirm the efficacy and safety of NNC 0078-0000-0007 in patients with congenital haemophilia and inhibitors.

Scheduled dose visit in a non-bleeding state. Single dose of NNC 0078-0000-0007 (vatreptocog alfa (activated)) every 3 months.

Inclusion Criteria:

• Male patient with clinical diagnosis of congenital haemophilia A or B and inhibitors to coagulation factors VIII or IX
• Minimum of five bleeds requiring haemostatic drug treatment within the previous 12 months at trial entry

Exclusion Criteria:

• Previous participation in this trial defined as withdrawal after administration of trial product
• Patient has received an investigational medicinal product within 30 days prior to this trial
• Congenital or acquired coagulation disorders other than haemophilia A or B
• Any clinical signs or known history of arterial thrombotic events or of deep venous thrombosis or pulmonary embolism (as defined by available medical records)
• Platelet count of less than 50,000 platelets/mcL (at the screening visit)
• ALAT (alanine-transaminase) of more than 3 times the upper normal limit (according to laboratory reference ranges)
• Factor VIII/IX Immune Tolerance Induction regimen planned to occur during the trial
• Ongoing bleeding prophylaxis regimens or planned bleeding prophylaxis to occur during the trial
• HIV (Human Immunodeficiency Virus) positive with current CD4+ count of less than 200/mcL (defined by medical records)