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

February 1, 2024 updated by: Novo Nordisk A/S

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.

Study Overview

Status

Enrolling by invitation

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Estimated)

60

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Austria
      • Wien, Austria, 1090
        • AKH - Klin. Abt. f. Haematologie u. Haemostaseologie
  • Belgium
      • Bruxelles, Belgium, 1200
        • Cliniques universitaires Saint-Luc - Service Hématologie
  • Canada
    • Alberta
      • Calgary, Alberta, Canada, T2N 2T9
        • University of Calgary Cumming School of Medicine
      • Edmonton, Alberta, Canada, T6G 2V2
        • Univ of Alberta Hospital Res
    • Manitoba
      • Winnipeg, Manitoba, Canada, R3E 0V9
        • CancerCare Manitoba
    • Newfoundland and Labrador
      • St. John's, Newfoundland and Labrador, Canada, A1B 3V6
        • Health Science Centre
    • Ontario
      • Hamilton, Ontario, Canada, L8N 3Z5
        • Hamilton Health Sciences Corp, Ontario
      • Hamilton, Ontario, Canada, L8N 3Z5
        • Hamltn Hth Sci/McMstr Child Hosp
      • Toronto, Ontario, Canada, M5G 1X8
        • The Hospital for Sick Children
  • Croatia
      • Zagreb, Croatia, 10 000
        • KBC Zagreb, Rebro, Hemofilija centar
  • Czechia
      • Brno, Czechia, 625 00
        • FN Brno odd. hematologie
      • Plzen, Czechia, 304 60
        • Fakultni nemocnice Plzen - Lochotin
  • Denmark
      • Aarhus N, Denmark, 8200
        • Skejby Blodsygdomme, blødercentret
  • Finland
      • Helsinki, Finland, 00290
        • Helsinki University Central Hospital/Coagulation Disorder Un
  • Germany
      • Berlin, Germany, 10249
        • Vivantes Klinikum am Friedrichshain
      • Bonn, Germany, 53127
        • Rheinische Friedrich-Wilhelms-Universität Bonn
  • Greece
      • Athens, Greece, GR-11527
        • "Laiko" General Hospital of Athens
      • Athens, Greece, GR-11527
        • Aghia Sophia Childrens' Hospital
  • Norway
      • Oslo, Norway, 0372
        • Klinisk forskningspost
  • Portugal
      • Lisboa, Portugal, 1150-199
        • Hospital Sao Jose
      • Lisboa, Portugal, 1169-045
        • Centro Hospitalar Lisboa Central - Hospital Dona Estefânia
  • United Kingdom
      • Aberdeen, United Kingdom, AB25 2ZN
        • Aberdeen Royal Infirmary - Haematology
      • Cardiff, United Kingdom, CF14 4XW
        • Arthur Bloom Haemophilia Centre
      • Manchester, United Kingdom, M13 9WL
        • Royal Manchester Children's Hospital
      • Sheffield, United Kingdom, S10 2JF
        • Royal Hallamshire Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Patients with haemophilia B

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Patients with haemophilia B
Patients with haemophilia B without current inhibitors
Drug: Nonacog beta pegol
Participants are treated with commercially available nonacog beta pegol (N9-GP) according to local clinical practice at the discretion of the treating physician

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
From start of study period (week 0) to end of study period (up to 9 years)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of Serious Adverse Events (SAEs)
Time Frame: From start of study period (week 0) to up to 9 years
Count of events
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)
Time Frame: From start of study period (week 0) to up to 9 years
Count of episodes
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)
Time Frame: From start of study period (week 0) to up to 9 years
Number of episodes
From start of study period (week 0) to up to 9 years
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
From start of study period (week 0) to up to 9 years
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
From start of study period (week 0) to end of study period (up to 9 years)

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novo Nordisk A/S

Investigators

  • Study Director: Clinical Reporting Anchor and Disclosure (1452), Novo Nordisk A/S

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

April 1, 2019

Primary Completion (Estimated)

December 15, 2027

Study Completion (Estimated)

December 15, 2027

Study Registration Dates

First Submitted

November 15, 2018

First Submitted That Met QC Criteria

November 15, 2018

First Posted (Actual)

November 19, 2018

Study Record Updates

Last Update Posted (Actual)

February 2, 2024

Last Update Submitted That Met QC Criteria

February 1, 2024

Last Verified

January 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • NN7999-4031
  • U1111-1165-8657 (Other Identifier: World Health Organization (WHO))
  • EUPAS26592 (Registry Identifier: EU PAS Register)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

According to the Novo Nordisk disclosure commitment on novonordisk-trials.com

Drug and device information, study documents

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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