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Trial record 1 of 1 for:    refixia | oslo, Norway
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A Study Following Males With Haemophilia B on Prophylaxis With Refixia/REBINYN

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03745924
Recruitment Status : Enrolling by invitation
First Posted : November 19, 2018
Last Update Posted : March 15, 2022
Sponsor:
Information provided by (Responsible Party):
Novo Nordisk A/S

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.

Condition or disease Intervention/treatment
Haemophilia B Drug: Nonacog beta pegol

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Study Type : Observational
Estimated Enrollment : 60 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Non-Interventional Post-Authorisation Safety Study (PASS) in Male Haemophilia B Patients Receiving Nonacog Beta Pegol (N9-GP) Prophylaxis Treatment
Actual Study Start Date : April 1, 2019
Estimated Primary Completion Date : December 15, 2027
Estimated Study Completion Date : December 15, 2027

Resource links provided by the National Library of Medicine


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




Primary Outcome Measures :
  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 Outcome Measures :
  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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with haemophilia B
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03745924


Locations
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Austria
Novo Nordisk Investigational Site
Wien, Austria, 1090
Belgium
Novo Nordisk Investigational Site
Bruxelles, Belgium, 1200
Novo Nordisk Investigational Site
Edegem, Belgium, 2650
Novo Nordisk Investigational Site
Leuven, Belgium, 3000
Canada, Alberta
Novo Nordisk Investigational Site
Calgary, Alberta, Canada, T2N 2T9
Novo Nordisk Investigational Site
Edmonton, Alberta, Canada, T6G 2V2
Canada, Manitoba
Novo Nordisk Investigational Site
Winnipeg, Manitoba, Canada, R3E 0V9
Canada, Newfoundland and Labrador
Novo Nordisk Investigational Site
St. John's, Newfoundland and Labrador, Canada, A1B 3V6
Canada, Ontario
Novo Nordisk Investigational Site
Hamilton, Ontario, Canada, L8N 3Z5
Novo Nordisk Investigational Site
Toronto, Ontario, Canada, M5G 1X8
Croatia
Novo Nordisk Investigational Site
Zagreb, Croatia, 10 000
Finland
Novo Nordisk Investigational Site
Helsinki, Finland, 00290
Germany
Novo Nordisk Investigational Site
Berlin, Germany, 10249
Novo Nordisk Investigational Site
Bonn, Germany, 53127
Norway
Novo Nordisk Investigational Site
Oslo, Norway, 0372
Portugal
Novo Nordisk Investigational Site
Lisboa, Portugal, 1150-199
Novo Nordisk Investigational Site
Lisboa, Portugal, 1169-045
Switzerland
Novo Nordisk Investigational Site
Zürich, Switzerland, 8091
United Kingdom
Novo Nordisk Investigational Site
Aberdeen, United Kingdom, AB25 2ZN
Novo Nordisk Investigational Site
Cardiff, United Kingdom, CF14 4XW
Sponsors and Collaborators
Novo Nordisk A/S
Investigators
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Study Director: Clinical Reporting Anchor and Disclosure (1452) Novo Nordisk A/S
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Responsible Party: Novo Nordisk A/S
ClinicalTrials.gov Identifier: NCT03745924    
Other Study ID Numbers: NN7999-4031
U1111-1165-8657 ( Other Identifier: World Health Organization (WHO) )
EUPAS26592 ( Registry Identifier: EU PAS Register )
First Posted: November 19, 2018    Key Record Dates
Last Update Posted: March 15, 2022
Last Verified: March 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
URL: http://novonordisk-trials.com

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Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked