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

Novo Nordisk

Status: Invitation-only

Conditions: Haemophilia B

Treatments: Drug: Nonacog beta pegol

Study type: Observational

Funder types: Industry

Identifiers

NCT03745924

U1111-1165-8657 (Other Identifier)

NN7999-4031

EUPAS26592 (Registry Identifier)

Details and patient eligibility

About

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.

Enrollment: 60 estimated patients

Sex: Male

Volunteers: No Healthy Volunteers

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.

Trial design

60 participants in 1 patient group

Patients with haemophilia B

Description:

Patients with haemophilia B without current inhibitors

Treatment:

Drug: Nonacog beta pegol