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

Official title

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

Overview

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.

Interventions

• 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

• 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)]

Secondary outcome measures (5)

• Number of Serious Adverse Events (SAEs) [Time frame: 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]
• 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]
• 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]
• 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)]

Eligibility 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.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Cohort

Study locations

Canada · 7 centers

• University of Calgary Cumming School of Medicine — Calgary
• Univ of Alberta Hospital Res — Edmonton
• CancerCare Manitoba — Winnipeg
• Health Science Centre — St. John's
• Hamilton Health Sciences Corp, Ontario — Hamilton
• Hamltn Hth Sci/McMstr Child Hosp — Hamilton
• The Hospital for Sick Children — Toronto

United Kingdom · 4 centers

• Aberdeen Royal Infirmary - Haematology — Aberdeen
• University Hospital of Wales - Haemophilia — Cardiff
• Royal Manchester Children's Hospital — Manchester
• Royal Hallamshire Hospital — Sheffield

Belgium · 3 centers

• Cliniques universitaires Saint-Luc - Service Hématologie — Brussels
• UZ Antwerpen - UZA - Kinderhemato-Oncologie — Edegem
• UZ Leuven - Hart en Vaatziekten — Leuven

Czechia · 3 centers

• Fakultní nemocnice Brno — Brno
• FN Brno odd. hematologie — Brno
• Fakultni nemocnice Plzen - Lochotin — Pilsen

Greece · 3 centers

• "Laiko" General Hospital of Athens — Athens
• "Laiko" General Hospital of Athens — Athens
• Aghia Sophia Childrens' Hospital — Athens

Germany · 2 centers

• Vivantes Netzwerk für Gesundheit GmbH - Vivantes Klinikum im Friedrichshain — Berlin
• Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie — Bonn

Portugal · 2 centers

• ULS De São José, E.P.E - Hospital São José_Serviço de Imunohemoterapia — Lisbon
• ULS De São José, E.P.E.- Hospital D. Estefânia — Lisbon

Austria · 1 center

• AKH - Klin. Abt. f. Haematologie u. Haemostaseologie — Vienna

Croatia · 1 center

• KBC Zagreb_Hematology — Zagreb

Denmark · 1 center

• Skejby Blodsygdomme, blødercentret — Aarhus N

Finland · 1 center

• Coagulation Disorder Unit — Helsinki

Norway · 1 center

• Klinisk forskningspost — Oslo

Switzerland · 1 center

• Universitätsspital Zürich - Klinik für Medizinische Onkologie und Hämatologie — Zurich

Identifiers

NCT: NCT03745924 · NN7999-4031 · U1111-1165-8657 · EUPAS26592

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗