A Study Following Males With Haemophilia B on Prophylaxis With Refixia/REBINYN

NCT03745924 | Enrolling By Invitation

• 3 other identifiers: NN7999-4031U1111-1165-8657EUPAS26592
• Study Type: observational
• Target: 60
• Locations: 13 countries
• Sites: 29

Brief 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. 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

Outcome Measures

Primary Outcomes (1)

• Number of Adverse Drug Reactions (ADRs) (FIX inhibitors, allergic reactions, and thromboembolic events)

  Count of events

  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)

  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)

  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)

  From start of study period (week 0) to up to 9 years

• Haemostatic effect of N9-GP when used for treatment of bleeding episodes

  From start of study period (week 0) to up to 9 years

• Haemostatic response of N9-GP when used in perioperative management

  From start of study period (week 0) to end of study period (up to 9 years)

Study Arms (1)

Patients with haemophilia B

Patients with haemophilia B without current inhibitors

Drug: Nonacog beta pegol

Interventions

Nonacog beta pegol DRUG

Participants are treated with commercially available nonacog beta pegol (N9-GP) according to local clinical practice at the discretion of the treating physician

Patients with haemophilia B

Eligibility Criteria

• Sex: male
• 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 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (29)

AKH - Klin. Abt. f. Haematologie u. Haemostaseologie

Vienna, 1090, Austria

Location

Cliniques universitaires Saint-Luc - Service Hématologie

Brussels, 1200, Belgium

Location

UZ Antwerpen - UZA - Kinderhemato-Oncologie

Edegem, 2650, Belgium

Location

UZ Leuven - Hart en Vaatziekten

Leuven, 3000, Belgium

Location

University of Calgary Cumming School of Medicine

Calgary, Alberta, T2N 2T9, Canada

Location

Univ of Alberta Hospital Res

Edmonton, Alberta, T6G 2V2, Canada

Location

CancerCare Manitoba

Winnipeg, Manitoba, R3E 0V9, Canada

Location

Health Science Centre

St. John's, Newfoundland and Labrador, A1B 3V6, Canada

Location

Hamilton Health Sciences Corp, Ontario

Hamilton, Ontario, L8N 3Z5, Canada

Location

Hamltn Hth Sci/McMstr Child Hosp

Hamilton, Ontario, L8N 3Z5, Canada

Location

The Hospital for Sick Children

Toronto, Ontario, M5G 1X8, Canada

Location

KBC Zagreb, Rebro, Hemofilija centar

Zagreb, 10 000, Croatia

Location

FN Brno odd. hematologie

Brno, 625 00, Czechia

Location

Fakultni nemocnice Plzen - Lochotin

Pilsen, 304 60, Czechia

Location

Skejby Blodsygdomme, blødercentret

Aarhus N, 8200, Denmark

Location

Helsinki University Central Hospital/Coagulation Disorder Un

Helsinki, 00290, Finland

Location

Vivantes Netzwerk für Gesundheit GmbH - Vivantes Klinikum im Friedrichshain

Berlin, 10249, Germany

Location

Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie

Bonn, 53127, Germany

Location

"Laiko" General Hospital of Athens

Athens, 11527, Greece

Location

"Laiko" General Hospital of Athens

Athens, GR-11527, Greece

Location

Aghia Sophia Childrens' Hospital

Athens, GR-11527, Greece

Location

Klinisk forskningspost

Oslo, 0372, Norway

Location

Hospital São José

Lisbon, 1150-199, Portugal

Location

Unidade Local de Saúde São José EPE- Hospital D. Estefânia

Lisbon, 1169-045, Portugal

Location

Universitätsspital Zürich - Klinik für Medizinische Onkologie und Hämatologie

Zurich, 8091, Switzerland

Location

Aberdeen Royal Infirmary - Haematology

Aberdeen, AB25 2ZN, United Kingdom

Location

University Hospital of Wales - Haemophilia

Cardiff, CF14 4XW, United Kingdom

Location

Royal Manchester Children's Hospital

Manchester, M13 9WL, United Kingdom

Location

Royal Hallamshire Hospital

Sheffield, S10 2JF, United Kingdom

Location

MeSH Terms

Conditions

Hemophilia B

Interventions

nonacog beta pegol

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 Officials

• Clinical Reporting Anchor and Disclosure (1452)

  Novo Nordisk A/S

  STUDY DIRECTOR

Study Design

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

Study Record Dates

• First Submitted: November 15, 2018
• First Posted: November 19, 2018
• Study Start: April 1, 2019
• Primary Completion (Estimated): December 15, 2027
• Study Completion (Estimated): December 15, 2027
• Last Updated: December 22, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share

Locations

BE (3); CA (7); AU (1); FI (1); NO (1); CR (1); CZ (2); PT (2); GB (4); DE (2); GR (3); CH (1); DK (1)