Efficacy and Safety of NNC-0156-0000-0009 During Surgical Procedures in Subjects With Haemophilia B

Study Overview
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Brief Summary
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This trial is conducted in Africa, Asia, Europe and the United States of America (USA). The aim of the trial is to evaluate the safety and efficacy of NNC-0156-0000-0009 (nonacog beta pegol) during surgical procedures in patients with haemophilia B.

Official Title
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An Open-label, Multi-centre, Un-controlled Trial to Assess Efficacy and Safety of NNC-0156-0000-0009 During Surgical Procedures in Patients With Haemophilia B

Conditions
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Congenital Bleeding Disorder, Haemophilia B

Intervention / Treatment
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* Drug: nonacog beta pegol


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patients with haemophilia B with a FIX activity below or equal to 2% Male patients with moderately severe or severe congenital haemophilia B with a FIX activity below or equal to 2% according to medical records History of at least 150 exposure days to other FIX products Scheduled major surgery Exclusion Criteria: Known history of FIX (coagulation factor nine) inhibitors based on existing medical records, laboratory report reviews and patient and LAR (legal acceptable representative) interviews Current FIX (coagulation factor nine) inhibitors above or equal to 0.6 Bethesda Units (central laboratory) Previous arterial thrombotic events (e.g. myocardial infarction and intracranial thrombosis) or previous deep venous thrombosis or pulmonary embolism (as defined by available medical records) ALT (alanine aminotransferase) 3 times the upper limit of normal reference ranges at screening (central laboratory) Immune modulating or chemotherapeutic medication

Ages Eligible for Study
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Minimum Age: 13 Years
Maximum Age: 70 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: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Patients enrolled in trial<br>New patients will be included as well as transferred patients from Paradigm™ 2 or Paradigm™ 4 trial | Drug: nonacog beta pegol<br>* The patients will receive nonacog beta pegol at screening just prior to and during surgical intervention, administered intravenously (into the vein).<br>* Other names: NNC-0156-0000-0009;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Haemostatic Effect During Surgery Evaluated by the Four-point Response Scale (Excellent, Good, Moderate, Poor) | Haemostatic effect during surgery was evaluated immediately after surgery (last stitch) using a fourpoint response scale: - Four-point response scale: Excellent, good, moderate, poor. The evaluation was done by the surgeon, anaesthesiologist and/or investigator based on experience as follows: Excellent: Better than expected/predicted in this type of procedure. Good: As expected in this type of procedure. Moderate: Less than optimal for the type of procedure but haemostatic response maintained without change of treatment regimen. Poor: Bleeding due to inadequate therapeutic response with adequate dosing, change of regimen required. | At the day of surgery | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Consumption of NNC-0156-0000-0009 (U/kg Body Weight) | Mean consumption of nonacog beta pegol (U/kg) used for treatment per patient before surgery, during surgery (the time from knife to skin until last stitch) and post-operative period. | During surgery (the time from knife to skin until last stitch) and post-operative period (day 1 to day 13) | 
| Transfusion Requirements (Fulfilling Transfusion Criteria) | Mean quantity of transfusion during surgery (the time from knife to skin until last stitch) and the postoperative period (Day 1-13).None of the patients required transfusions beyond Day 6 hence no values presented for days 7-13. | during surgery (the time from knife to skin until last stitch) and post-operative period (day 1 to day 13) | 
| Haemoglobin Pre- and Post-surgery Start | The mean pre-surgery and post surgery haemoglobin level. | 0, 1 hour, 24 hours. | 
| Incidence of Adverse Events (AEs) | The number of adverse events per patient years of exposure, reported during the trial period. Number is the only available option here, the data presented are rate of AEs. | during the trial period (2-8 weeks prior to day of surgery (transferred subjects) or 2-4 weeks prior to day of surgery (new subjects) until 4 weeks after post-operative period (day 1 to day 13) | 
| Incidence of Serious Adverse Events (SAE) | The number of serious adverse events per patient years of exposure, reported during the trial period. Number is the only available option here, the data presented are rate of AEs. | During the trial period (2-8 weeks prior to day of surgery (transferred subjects) or 2-4 weeks prior to day of surgery (new subjects) until 4 weeks after post-operative period (day 1 to day 13) | 
| Incidence of Inhibitors Against FIX (Coagulation Factor Nine) | Number of patients with inhibitory antibodies | During the trial period (2-8 weeks prior to day of surgery (transferred subjects) or 2-4 weeks prior to day of surgery (new subjects) and every 4 weeks after post-operative period (day 1 to day 13) |