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

Study Overview
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Brief Summary
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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.

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

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

Conditions
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Congenital Bleeding Disorder, Haemophilia A With Inhibitors, Haemophilia B With Inhibitors

Intervention / Treatment
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* Drug: vatreptacog alfa (activated)
* Drug: eptacog alfa (activated)


Participation Criteria
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Eligibility Criteria
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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)

Ages Eligible for Study
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Minimum Age: 12 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: rFVIIa<br> | Drug: eptacog alfa (activated)<br>* 1-3 doses per bleeding episode<br>|
| Experimental: vatreptocog alfa<br> | Drug: vatreptacog alfa (activated)<br>* 1-3 doses per bleeding episode<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Effective Bleeding Control Defined as no Additional Haemostatic Medication (Other Than Trial Product) Given |  | Within 12 hours of first trial product administration | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Effective and Sustained Bleeding Control |  | Up to 48 hours after first trial product administration | 
| Number of Doses of Trial Product Given for Each Acute Bleed |  | Up to 6 hours after first trial product administration | 
| Number of Adverse Events | Any untoward medical occurrence in a patient or clinical investigation patient administered a pharmaceutical product and which does not necessarily have to have a causal relationship with this treatment. | Adverse events were captured from the time of consent to 1 month (+14 days) after last administration of trial product. | 
| Immunogenicity (Inhibitor Development) | Immunogenicity was tested by formation of neutralising antibodies towards vatreptacog alfa and/or FVII. Radioimmunoassay using [125I]-labelled vatreptacog alfa or rFVIIa was used to screen plasma samples for development of anti-drug antibodies | Adverse events were captured from the time of consent to the end of trial visit 1 month (+14 days) after last administration of trial product. |