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

NCT03875547 | Completed

• 2 other identifiers: NN7999-4404U1111-1198-6270
• Study Type: observational
• Target: 28
• Locations: 2 countries
• Sites: 23

Brief Summary

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®.

Conditions

Haemophilia B

Outcome Measures

Primary Outcomes (1)

• Number of Adverse Reactions (ARs)

  Count of events

  From baseline (week 0) to end of study (up to 6 years and 10 months)

Secondary Outcomes (6)

• Number of Serious Adverse Events (SAEs)

  From baseline (week 0) to end of study (up to 6 years and 10 months)

• Number of Serious Adverse Reactions (SARs)

  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)

  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

  From baseline (week 0) to end of study (up to 6 years and 10 months)

• Haemostatic response of Refixia® in treatment of bleeds

  From baseline (week 0) to end of study (up to 6 years and 10 months)

Study Arms (1)

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®

Interventions

Refixia® DRUG

Patients will be treated with commercially available Refixia® according to routine clinical practice at the discretion of the treating physician

Patients with haemophilia B

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Patients with haemophilia B

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (23)

Novo Nordisk Investigational Site

Søborg, Denmark

Location

Nagoya University Hospital_Blood Transfusion

Aichi, 466-8560, Japan

Location

Aomori Prefectural Central Hospital_Pediatrics

Aomori, 030-8553, Japan

Location

Matsudo City General Hospital_Pediatrics

Chiba, 270-2296, Japan

Location

Japan Red Cross Narita Hospital_Hematology and Oncology

Chiba, 286-8523, Japan

Location

Chiba University Hospital_Diabetes, Metabolism and Endocrinology

Chiba-shi, Chiba, 260-8677, Japan

Location

Hyogo prefectural kobe children's hospital Dept. of Haem and Onclogy

Hyōgo, 654-0047, Japan

Location

Tokyo Medical University Ibaraki Medical Center_Ibaraki

Ibaraki, 300-0395, Japan

Location

Ishikawa Prefectural Central Hospital_Haematology

Ishikawa, 920-8530, Japan

Location

St. Marianna University School of Medicine Hospital_Pediatrics

Kanagawa, 216-8511, Japan

Location

Kitasato University Hospital_Blood Transfusion

Kanagawa, 252-0375, Japan

Location

Hospital of the University of Occupational And Environmental Health Japan_Pediatrics

Kitakyusyu-shi, Fukuoka, 807 8555, Japan

Location

Gunma University Hospital_Hematology

Maebashi-shi, Gunma, 371-8511, Japan

Location

Naha City Hospital_Cardiovascular Medicine

Naha-shi, Okinawa, 902-8511, Japan

Location

Nara Medical University Hospital_Pediatrics

Nara, 634-8522, Japan

Location

The Hospital of Hyogo College of Medicine_Haematology

Nishinomiya-shi, Hyogo, 663 8051, Japan

Location

Saitama Children's Med Centre_Hematology-Oncology

Saitama, 330-8777, Japan

Location

Tokushima University Hospital_Pediatrics

Tokushima, 770-8503, Japan

Location

Tokushima Red Cross Hospital_Pediatrics

Tokushima, 773-8502, Japan

Location

Tokyo Medical Univ. Hospital_Laboratory Medicine

Tokyo, 160-0023, Japan

Location

Ogikubo Hospital_Blood Coagulation

Tokyo, 167-0035, Japan

Location

Ishiyama Clinic

Yamagata, 992-0035, Japan

Location

St. Marianna Univ., Yokohama City Seibu HP, Pediatrics Dept,

Yokohama-shi, Kanagawa, 241-0811, Japan

Location

MeSH Terms

Conditions

Hemophilia B

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 13, 2019
• First Posted: March 14, 2019
• Study Start: August 23, 2019
• Primary Completion: September 30, 2025
• Study Completion: September 30, 2025
• Last Updated: April 24, 2026

Record last verified: 2026-04

Data Sharing

• IPD Sharing: Will share

Locations

JP (22); DK (1)