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Completed

NCT Number: NCT03875547

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

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®.

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Key information

Conditions

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

Sex eligibility

All sexes

Study type

Observational

Primary location

Novo Nordisk Investigational Site, Søborg, Denmark

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

Refixia®

Drug

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

Primary outcomes

  1. Number of Adverse Reactions (ARs)

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

    Count of events

Secondary outcomes

  1. Number of Serious Adverse Events (SAEs)

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

    Count of events

  2. Number of Serious Adverse Reactions (SARs)

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

    Count of events

  3. Number of bleeding episodes as assessed by annualised bleeding rate (ABR)

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

    Count of bleeding episodes

  4. Number of treatment requiring bleeding episodes as assessed by ABR

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

    Count of bleeding episodes

  5. Haemostatic response of Refixia® in treatment of bleeds

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

    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.

  6. Haemostatic response of Refixia® in treatment of bleeds in perioperative management during surgical procedures

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

    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.

Sponsors and collaborators

Lead sponsor

Novo Nordisk A/S

Industry

Registry information

Official study title

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.

Important dates

Study start
2019
Primary completion
2025
Study completion
2025
First posted
Mar 14, 2019
Registry last updated
Apr 24, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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