Post-marketing Surveillance (Use Result Surveillance) With Refixia®

Study Overview
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Brief Summary
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The participants are invited to take part in this study because they have Haemophilia B. The purpose of this study is to assess the safety and effectiveness of Refixia® about long-term routine use in patients with Haemophilia B. The participants will get Refixia® as prescribed to them by their study doctor. The study will last up to Sep 2025 for the participant. The participants may be asked to fill in the quality of life questionnaires (if they are above age of 15). The blood samples taken from the participants as part of routine clinical practice will also be used to investigate the safety for the long-term use of Refixia®.

Official Title
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Post-marketing Surveillance (Use Result Surveillance) With Refixia®. A Multicentre, Non-interventional Post Marketing Surveillance of Safety and Effectiveness of Refixia® in Routine Clinical Care With Haemophilia B Patients in Japan.

Conditions
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Haemophilia B

Intervention / Treatment
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* Drug: Refixia®


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Signed consent obtained before any study-related activities (study-related activities are any procedure related to recording of data according to the protocol). The decision to initiate treatment with commercially available Refixia® has been made by the patient/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study. At each site of this study, all patients will be registered consecutively from the first patient after the launch of Refixia® (consecutively registered system). Diagnosis of haemophilia B in males or females, no age limitation. Patients younger than 12 years old will continue to be registered for 3 years of recruitment period regardless of the target number of patients. New patients who have not been previously exposed to Refixia®. Also patients previously exposed to Refixia® in NN7999-3639, -3747, -3774, -3775 or -3895 clinical trial can be enrolled in this study. The patients who have participated in NN7999 -3774 or -3895 clinical trial can be enrolled in this study as continuous cases until 30-September-2024 (one year before planned end of study date). Exclusion Criteria: Previous participation in this study. Participation is defined as having given informed consent in this study Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation Known or suspected hypersensitivity to study product or related products.
Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Patients with haemophilia B<br>Both patients who have not previously been exposed to Refixia® and patients previously exposed to Refixia® in one of the clinical trials can be included. | Drug: Refixia®<br>* Patients will be treated with commercially available Refixia® according to routine clinical practice at the discretion of the treating physician<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Adverse Reactions (ARs) | Count of events | From baseline (week 0) to end of study (up to 6 years and 10 months) | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of Serious Adverse Events (SAEs) | Count of events | From baseline (week 0) to end of study (up to 6 years and 10 months) | 
| Number of Serious Adverse Reactions (SARs) | Count of events | From baseline (week 0) to end of study (up to 6 years and 10 months) | 
| Number of bleeding episodes as assessed by annualised bleeding rate (ABR) | Count of bleeding episodes | From baseline (week 0) to end of study (up to 6 years and 10 months) | 
| Number of treatment requiring bleeding episodes as assessed by ABR | Count of bleeding episodes | From baseline (week 0) to end of study (up to 6 years and 10 months) | 
| Haemostatic response of Refixia® in treatment of bleeds | Haemostatic response is assessed as success/failure based on a four-point scale (excellent, good, moderate and poor) by counting excellent and good as success and moderate and poor as failure. | From baseline (week 0) to end of study (up to 6 years and 10 months) | 
| Haemostatic response of Refixia® in treatment of bleeds in perioperative management during surgical procedures | Haemostatic response is assessed as success/failure based on a four-point scale (excellent, good, moderate and poor) by counting excellent and good as success and moderate and poor as failure. | From baseline (week 0) to end of study (up to 6 years and 10 months) |