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

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.

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®.

Study Overview

• Status: Completed
• Conditions: Haemophilia B
• Intervention / Treatment: Drug: Refixia®

• Study Type: Observational
• Enrollment (Actual): 28

Contacts and Locations

Study Locations

• Denmark
  • Søborg, Denmark
    • Novo Nordisk Investigational Site
• Japan
  • Aichi, Japan, 466-8560
    • Nagoya University Hospital_Blood Transfusion
  • Aomori, Japan, 030-8553
    • Aomori Prefectural Central Hospital_Pediatrics
  • Chiba, Japan, 270-2296
    • Matsudo City General Hospital_Pediatrics
  • Chiba, Japan, 286-8523
    • Japan Red Cross Narita Hospital_Hematology and Oncology
  • Chiba-shi, Chiba, Japan, 260-8677
    • Chiba University Hospital_Diabetes, Metabolism and Endocrinology
  • Hyōgo, Japan, 654-0047
    • Hyogo prefectural kobe children's hospital Dept. of Haem and Onclogy
  • Ibaraki, Japan, 300-0395
    • Tokyo Medical University Ibaraki Medical Center_Ibaraki
  • Ishikawa, Japan, 920-8530
    • Ishikawa Prefectural Central Hospital_Haematology
  • Kanagawa, Japan, 216-8511
    • St. Marianna University School of Medicine Hospital_Pediatrics
  • Kanagawa, Japan, 252-0375
    • Kitasato University Hospital_Blood Transfusion
  • Kitakyusyu-shi, Fukuoka, Japan, 807 8555
    • Hospital of the University of Occupational And Environmental Health Japan_Pediatrics
  • Maebashi-shi, Gunma, Japan, 371-8511
    • Gunma University Hospital_Hematology
  • Naha-shi, Okinawa, Japan, 902-8511
    • Naha City Hospital_Cardiovascular Medicine
  • Nara, Japan, 634-8522
    • Nara Medical University Hospital_Pediatrics
  • Nishinomiya-shi, Hyogo, Japan, 663 8051
    • The Hospital of Hyogo College of Medicine_Haematology
  • Saitama, Japan, 330-8777
    • Saitama Children's Med Centre_Hematology-Oncology
  • Tokushima, Japan, 773-8502
    • Tokushima Red Cross Hospital_Pediatrics
  • Tokushima, Japan, 770-8503
    • Tokushima University Hospital_Pediatrics
  • Tokyo, Japan, 160-0023
    • Tokyo Medical Univ. Hospital_Laboratory Medicine
  • Tokyo, Japan, 167-0035
    • Ogikubo Hospital_Blood Coagulation
  • Yamagata, Japan, 992-0035
    • Ishiyama Clinic
  • Yokohama-shi, Kanagawa, Japan, 241-0811
    • St. Marianna Univ., Yokohama City Seibu HP, Pediatrics Dept,

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Patients with haemophilia B

Description

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.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Patients with haemophilia B; 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®; Patients will be treated with commercially available Refixia® according to routine clinical practice at the discretion of the treating physician

What is the study measuring?

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)

Collaborators and Investigators

• Sponsor: Novo Nordisk A/S

Study record dates

Study Major Dates

• Study Start (Actual): August 23, 2019
• Primary Completion (Actual): September 30, 2025
• Study Completion (Actual): September 30, 2025

Study Registration Dates

• First Submitted: March 13, 2019
• First Submitted That Met QC Criteria: March 13, 2019
• First Posted (Actual): March 14, 2019

Study Record Updates

• Last Update Posted (Actual): April 24, 2026
• Last Update Submitted That Met QC Criteria: April 23, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Genetic Diseases, X-Linked; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Hemophilia B
• Other Study ID Numbers: NN7999-4404; U1111-1198-6270 (Other Identifier: World Health Organization (WHO))

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com

Drug and device information, study documents

• Studies a U.S. FDA-regulated device product: No