Haemophilia BNCT03875547
Post-marketing Surveillance (Use Result Surveillance) With Refixia®
An observational study of Haemophilia B, run by Novo Nordisk A/S at 23 sites in 2 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Haemophilia B
- What is being tested?
- Refixia®
- How many people
- 28 participants
- Where
- 23 sitesin 2 countries
- Study dates
- 23 Aug 2019to 30 Sep 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with haemophilia B
Who may be able to join
- 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).
Who cannot join
- 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.
Trial locations and contacts
23 sites in 2 countries.
23 sites in 2 countries
Japan22 sites
Nagoya University Hospital_Blood Transfusion
Aichi, 466-8560
Aomori Prefectural Central Hospital_Pediatrics
Aomori, 030-8553
Matsudo City General Hospital_Pediatrics
Chiba, 270-2296
Japan Red Cross Narita Hospital_Hematology and Oncology
Chiba, 286-8523
Chiba University Hospital_Diabetes, Metabolism and Endocrinology
Chiba-shi, Chiba, 260-8677
Hyogo prefectural kobe children's hospital Dept. of Haem and Onclogy
Hyōgo, 654-0047
Tokyo Medical University Ibaraki Medical Center_Ibaraki
Ibaraki, 300-0395
Ishikawa Prefectural Central Hospital_Haematology
Ishikawa, 920-8530
St. Marianna University School of Medicine Hospital_Pediatrics
Kanagawa, 216-8511
Kitasato University Hospital_Blood Transfusion
Kanagawa, 252-0375
Hospital of the University of Occupational And Environmental Health Japan_Pediatrics
Kitakyusyu-shi, Fukuoka, 807 8555
Gunma University Hospital_Hematology
Maebashi-shi, Gunma, 371-8511
Naha City Hospital_Cardiovascular Medicine
Naha-shi, Okinawa, 902-8511
Nara Medical University Hospital_Pediatrics
Nara, 634-8522
The Hospital of Hyogo College of Medicine_Haematology
Nishinomiya-shi, Hyogo, 663 8051
Saitama Children's Med Centre_Hematology-Oncology
Saitama, 330-8777
Tokushima University Hospital_Pediatrics
Tokushima, 770-8503
Tokushima Red Cross Hospital_Pediatrics
Tokushima, 773-8502
Tokyo Medical Univ. Hospital_Laboratory Medicine
Tokyo, 160-0023
Ogikubo Hospital_Blood Coagulation
Tokyo, 167-0035
Ishiyama Clinic
Yamagata, 992-0035
St. Marianna Univ., Yokohama City Seibu HP, Pediatrics Dept,
Yokohama-shi, Kanagawa, 241-0811
About this study
Official 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.
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
- Being tested
- Refixia®
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Refixia®Drug
Patients will be treated with commercially available Refixia® according to routine clinical practice at the discretion of the treating physician
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Refixia® |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Adverse Reactions (ARs)
Count of events
Measured: From baseline (week 0) to end of study (up to 6 years and 10 months)
Secondary outcomes (6)
Number of Serious Adverse Events (SAEs)
Count of events
Measured: From baseline (week 0) to end of study (up to 6 years and 10 months)
Number of Serious Adverse Reactions (SARs)
Count of events
Measured: 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
Measured: 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
Measured: 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.
Measured: 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.
Measured: From baseline (week 0) to end of study (up to 6 years and 10 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Mar 2019
- Study start23 Aug 2019
- Primary completion30 Sep 2025
- Study completion30 Sep 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Haemophilia BStudies of the same condition, open and under way.
- More trials run by Novo Nordisk A/SThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 28 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03875547
- Sponsor's protocol ID
- NN7999-4404
- Other IDs
- U1111-1198-6270
- Status last verified
- Apr 2026
- First submitted
- 13 Mar 2019
- Last updated
- 24 Apr 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
Study at a glance
From the registry record, last updated by the sponsor on 24 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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.
- ClinicalTrials.gov ID
- NCT03875547
- Acronym / sponsor ID
- NN7999-4404
- Conditions
- Haemophilia B
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 28 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 23 Aug 2019 (actual)
- Primary completion
- 30 Sep 2025 (actual)
- Study completion
- 30 Sep 2025 (actual)
- First posted
- 14 Mar 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with haemophilia B | – | Drug: Refixia®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. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with haemophilia B
Key inclusion criteria (4 listed)
- 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).
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Refixia®Patients will be treated with commercially available Refixia® according to routine clinical practice at the discretion of the treating physician | Drug | Patients with haemophilia B |
Primary outcome
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
6 secondary outcomes
Number of Serious Adverse Events (SAEs)
Time frame: From baseline (week 0) to end of study (up to 6 years and 10 months)
Number of Serious Adverse Reactions (SARs)
Time frame: 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)
Time frame: 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
Time frame: From baseline (week 0) to end of study (up to 6 years and 10 months)
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 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)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Apr 2026.
- Individual participant dataSharing planned: Yes. According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
23 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Haemophilia B, run by Novo Nordisk A/S at 23 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in Japan, Denmark. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Adverse Reactions (ARs) (time frame: From baseline (week 0) to end of study (up to 6 years and 10 months)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Apr 2026; AzTrials last refreshed its copy on 6 Nov 2026.