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Enrolling by Invitation

NCT Number: NCT03745924

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

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.

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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

Male

Study type

Observational

Primary location

AKH - Klin. Abt. f. Haematologie u. Haemostaseologie, Vienna, Austria

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Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • 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

Exclusion criteria

  • 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.

Treatment and study plan

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

Primary outcomes

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

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

    Count of events

Secondary outcomes

  1. Number of Serious Adverse Events (SAEs)

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

    Count of events

  2. Number of bleeding episodes during long-term routine use of N9-GP (prophylaxis) as assessed by annualised bleeding rate (ABR)

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

    Count of episodes

  3. Number of treatment requiring bleeding episodes during long-term routine use of N9-GP (prophylaxis) as assessed by annualised bleeding rate (ABR)

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

    Number of episodes

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

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

    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

  5. Haemostatic response of N9-GP when used in perioperative management

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

    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

Sponsors and collaborators

Lead sponsor

Novo Nordisk A/S

Industry

Registry information

Official study title

A Non-Interventional Post-Authorisation Safety Study (PASS) in Male Haemophilia B Patients Receiving Nonacog Beta Pegol (N9-GP) Prophylaxis Treatment

Important dates

Study start
2019
Primary completion
2027
Study completion
2027
First posted
Nov 19, 2018
Registry last updated
Jun 12, 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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