Haemophilia BNCT03745924
A Study Following Males With Haemophilia B on Prophylaxis With Refixia/REBINYN
An observational study of Haemophilia B, run by Novo Nordisk A/S at 30 sites in 13 countries.
Trial summary
This study will collect information on side effects and how well Refixia/REBINYN works during long-term treatment (prophylaxis) in males with haemophilia B. While taking part in this study, participants will receive the same treatment as given to them by their study doctor. Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Haemophilia B
- What is being tested?
- Nonacog beta pegol
- How many people
- 60 participants
- Where
- 30 sitesin 13 countries
- Study dates
- 1 Apr 2019to 15 Dec 2027
- Phase
- Observational
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
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with haemophilia B
Who may be able to join
- Signed informed consent obtained before any study-related activities (study-related activities are any procedure related to recording of data according to the protocol).
- Male patients at any age with haemophilia B assigned to N9-GP prophylaxis treatment
- Decision to initiate treatment with commercially available N9-GP has been made by the patient(s)/Legally Authorised Representative(s) (LAR(s)) and the treating physician before and independently from the decision to include the patient in this study
Who cannot join
- Previous participation in this study. Participation is defined as signed informed consent
- Known or suspected hypersensitivity to N9-GP or related products
- Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation
- Clinical suspicion or presence of FIX inhibitor at time of inclusion.
Trial locations and contacts
30 sites in 13 countries. Contact the study team or a site near you to ask about taking part.
30 sites in 13 countries
Canada7 sites
University of Calgary Cumming School of Medicine
Calgary, Alberta, T2N 2T9
Univ of Alberta Hospital Res
Edmonton, Alberta, T6G 2V2
Hamilton Health Sciences Corp, Ontario
Hamilton, Ontario, L8N 3Z5
Hamltn Hth Sci/McMstr Child Hosp
Hamilton, Ontario, L8N 3Z5
Health Science Centre
St. John's, Newfoundland and Labrador, A1B 3V6
The Hospital for Sick Children
Toronto, Ontario, M5G 1X8
CancerCare Manitoba
Winnipeg, Manitoba, R3E 0V9
United Kingdom4 sites
Aberdeen Royal Infirmary - Haematology
Aberdeen, AB25 2ZN
University Hospital of Wales - Haemophilia
Cardiff, CF14 4XW
Royal Manchester Children's Hospital
Manchester, M13 9WL
Royal Hallamshire Hospital
Sheffield, S10 2JF
Belgium3 sites
Cliniques universitaires Saint-Luc - Service Hématologie
Brussels, 1200
UZ Antwerpen - UZA - Kinderhemato-Oncologie
Edegem, 2650
UZ Leuven - Hart en Vaatziekten
Leuven, 3000
Czechia3 sites
Fakultní nemocnice Brno
Brno, 625 00
FN Brno odd. hematologie
Brno, 625 00
Fakultni nemocnice Plzen - Lochotin
Pilsen, 304 60
Greece3 sites
"Laiko" General Hospital of Athens
Athens, 11527
"Laiko" General Hospital of Athens
Athens, GR-11527
Aghia Sophia Childrens' Hospital
Athens, GR-11527
Germany2 sites
Vivantes Netzwerk für Gesundheit GmbH - Vivantes Klinikum im Friedrichshain
Berlin, 10249
Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie
Bonn, 53127
Portugal2 sites
ULS De São José, E.P.E - Hospital São José_Serviço de Imunohemoterapia
Lisbon, 1150-199
ULS De São José, E.P.E.- Hospital D. Estefânia
Lisbon, 1169-045
Austria1 site
AKH - Klin. Abt. f. Haematologie u. Haemostaseologie
Vienna, 1090
Switzerland1 site
Universitätsspital Zürich - Klinik für Medizinische Onkologie und Hämatologie
Zurich, 8091
About this study
Official title: A Non-Interventional Post-Authorisation Safety Study (PASS) in Male Haemophilia B Patients Receiving Nonacog Beta Pegol (N9-GP) Prophylaxis Treatment
This study will collect information on side effects and how well Refixia/REBINYN works during long-term treatment (prophylaxis) in males with haemophilia B. While taking part in this study, participants will receive the same treatment as given to them by their study doctor. All visits at the clinic are done in the same way as the participants are used to. During visits at the clinic, participants might be asked for some relevant tests if considered useful by their study doctor. During the visits, the participants study doctor might ask if the participants had any side effects since their last study visit. The participants will be asked to note down the number of bleeds and the treatment of their bleeds as well as their regular prophylaxis. During the visits to the clinic, the participants will be asked to answer some questionnaires about their quality of life and their ability to be physically active. The participant's participation in the study will last for 4-9 years, depending on when they join the study. Participants are free to leave the study at any time and for any reason. This will not affect their current and future medical care.
- Conditions
- Haemophilia B
- Being tested
- Nonacog beta pegol
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Nonacog beta pegolDrug
Participants are treated with commercially available nonacog beta pegol (N9-GP) according to local clinical practice at the discretion of the treating physician
| Study group | Role | What they receive |
|---|---|---|
| Patients with haemophilia B | Patients with haemophilia B without current inhibitors Receives: Drug: Nonacog beta pegol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Adverse Drug Reactions (ADRs) (FIX inhibitors, allergic reactions, and thromboembolic events)
Count of events
Measured: From start of study period (week 0) to end of study period (up to 9 years)
Secondary outcomes (5)
Number of Serious Adverse Events (SAEs)
Count of events
Measured: From start of study period (week 0) to up to 9 years
Number of bleeding episodes during long-term routine use of N9-GP (prophylaxis) as assessed by annualised bleeding rate (ABR)
Count of episodes
Measured: From start of study period (week 0) to up to 9 years
Number of treatment requiring bleeding episodes during long-term routine use of N9-GP (prophylaxis) as assessed by annualised bleeding rate (ABR)
Number of episodes
Measured: From start of study period (week 0) to up to 9 years
Haemostatic effect of N9-GP when used for treatment of bleeding episodes
Count of bleeding episodes. Haemostatic effect is assessed as success/failure based on a four-point scale for haemostatic response (excellent, good, moderate and poor) by counting excellent and good as success and moderate and poor as failure
Measured: From start of study period (week 0) to up to 9 years
Haemostatic response of N9-GP when used in perioperative management
Count of bleeding episodes. Haemostatic response is assessed as success/failure based on a four-point scale for haemostatic response (excellent, good, moderate and poor) by counting excellent and good as success and moderate and poor as failure
Measured: From start of study period (week 0) to end of study period (up to 9 years)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Nov 2018
- Study start1 Apr 2019
- Primary completion15 Dec 2027 (estimated)
- Study completion15 Dec 2027 (estimated)
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
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Reporting Anchor and Disclosure (1452), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03745924
- Sponsor's protocol ID
- NN7999-4031
- Other IDs
- U1111-1165-8657, EUPAS26592
- Status last verified
- Jun 2026
- First submitted
- 15 Nov 2018
- Last updated
- 12 Jun 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