A Study Following Males With Haemophilia B on Prophylaxis With Refixia/REBINYN
- Conditions
- Haemophilia B
- Interventions
- Registration Number
- NCT03745924
- Lead Sponsor
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ENROLLING_BY_INVITATION
- Sex
- Male
- Target Recruitment
- 60
- 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
- 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 & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Patients with haemophilia B Nonacog beta pegol Patients with haemophilia B without current inhibitors
- Primary Outcome Measures
Name Time Method Number of Adverse Drug Reactions (ADRs) (FIX inhibitors, allergic reactions, and thromboembolic events) From start of study period (week 0) to end of study period (up to 9 years) Count of events
- Secondary Outcome Measures
Name Time Method 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 Count of episodes
Haemostatic effect of N9-GP when used for treatment of bleeding episodes 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
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 Number of episodes
Number of Serious Adverse Events (SAEs) From start of study period (week 0) to up to 9 years Count of events
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) 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
Trial Locations
- Locations (22)
Univ of Alberta Hospital Res
🇨🇦Edmonton, Alberta, Canada
FN Brno odd. hematologie
🇨🇿Brno, Czechia
AKH - Klin. Abt. f. Haematologie u. Haemostaseologie
🇦🇹Wien, Austria
Hamilton Health Sciences Corp, Ontario
🇨🇦Hamilton, Ontario, Canada
Cliniques universitaires Saint-Luc - Service Hématologie
🇧🇪Bruxelles, Belgium
CancerCare Manitoba
🇨🇦Winnipeg, Manitoba, Canada
Health Science Centre
🇨🇦St. John's, Newfoundland and Labrador, Canada
Hamltn Hth Sci/McMstr Child Hosp
🇨🇦Hamilton, Ontario, Canada
The Hospital for Sick Children
🇨🇦Toronto, Ontario, Canada
KBC Zagreb, Rebro, Hemofilija centar
🇭🇷Zagreb, Croatia
Fakultni nemocnice Plzen - Lochotin
🇨🇿Plzen, Czechia
Skejby Blodsygdomme, blødercentret
🇩🇰Aarhus N, Denmark
Helsinki University Central Hospital/Coagulation Disorder Un
🇫🇮Helsinki, Finland
Vivantes Klinikum am Friedrichshain - Innere Medizin - Angiologie und Hämostaseologie
🇩🇪Berlin, Germany
Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie
🇩🇪Bonn, Germany
"Laiko" General Hospital of Athens
🇬🇷Athens, Greece
Aghia Sophia Childrens' Hospital
🇬🇷Athens, Greece
Klinisk forskningspost
🇳🇴Oslo, Norway
Hospital São José
🇵🇹Lisboa, Portugal
Centro Hospitalar Lisboa Central - Hospital Dona Estefânia
🇵🇹Lisboa, Portugal
Arthur Bloom Haemophilia Centre
🇬🇧Cardiff, United Kingdom
Royal Hallamshire Hospital
🇬🇧Sheffield, United Kingdom