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

Haemophilia B Clinical Trial

Official title:

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

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT03745924
• Other study ID #: NN7999-4031
• Secondary ID: U1111-1165-8657E
• Status: Enrolling by invitation
• Start date: April 1, 2019
• Est. completion date: December 15, 2027

Study information

• Verified date: January 2024
• Source: Novo Nordisk A/S
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 60
• Est. completion date: December 15, 2027
• Est. primary completion date: December 15, 2027
• Accepts healthy volunteers: No
• Gender: Male
• Age group: N/A and older

Eligibility

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.

Related Conditions & MeSH terms

• Haemophilia B
• Hemophilia A
• Hemophilia B

Intervention

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

Locations

Country	Name	City	State
Austria	AKH - Klin. Abt. f. Haematologie u. Haemostaseologie	Wien
Belgium	Cliniques universitaires Saint-Luc - Service Hématologie	Bruxelles
Canada	University of Calgary Cumming School of Medicine	Calgary	Alberta
Canada	Univ of Alberta Hospital Res	Edmonton	Alberta
Canada	Hamilton Health Sciences Corp, Ontario	Hamilton	Ontario
Canada	Hamltn Hth Sci/McMstr Child Hosp	Hamilton	Ontario
Canada	Health Science Centre	St. John's	Newfoundland and Labrador
Canada	The Hospital for Sick Children	Toronto	Ontario
Canada	CancerCare Manitoba	Winnipeg	Manitoba
Croatia	KBC Zagreb, Rebro, Hemofilija centar	Zagreb
Czechia	FN Brno odd. hematologie	Brno
Czechia	Fakultni nemocnice Plzen - Lochotin	Plzen
Denmark	Skejby Blodsygdomme, blødercentret	Aarhus N
Finland	Helsinki University Central Hospital/Coagulation Disorder Un	Helsinki
Germany	Vivantes Klinikum am Friedrichshain	Berlin
Germany	Rheinische Friedrich-Wilhelms-Universität Bonn	Bonn
Greece	"Laiko" General Hospital of Athens	Athens
Greece	Aghia Sophia Childrens' Hospital	Athens
Norway	Klinisk forskningspost	Oslo
Portugal	Centro Hospitalar Lisboa Central - Hospital Dona Estefânia	Lisboa
Portugal	Hospital São José	Lisboa
United Kingdom	Aberdeen Royal Infirmary - Haematology	Aberdeen
United Kingdom	Arthur Bloom Haemophilia Centre	Cardiff
United Kingdom	Royal Manchester Children's Hospital	Manchester
United Kingdom	Royal Hallamshire Hospital	Sheffield

Sponsors (1)

Lead Sponsor	Collaborator
Novo Nordisk A/S

Countries where clinical trial is conducted

Austria,  Belgium,  Canada,  Croatia,  Czechia,  Denmark,  Finland,  Germany,  Greece,  Norway,  Portugal,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Adverse Drug Reactions (ADRs) (FIX inhibitors, allergic reactions, and thromboembolic events)	Count of events	From start of study period (week 0) to end of study period (up to 9 years)
Secondary	Number of Serious Adverse Events (SAEs)	Count of events	From start of study period (week 0) to up to 9 years
Secondary	Number of bleeding episodes during long-term routine use of N9-GP (prophylaxis) as assessed by annualised bleeding rate (ABR)	Count of episodes	From start of study period (week 0) to up to 9 years
Secondary	Number of treatment requiring bleeding episodes during long-term routine use of N9-GP (prophylaxis) as assessed by annualised bleeding rate (ABR)	Number of episodes	From start of study period (week 0) to up to 9 years
Secondary	Haemostatic effect of N9-GP when used for treatment of bleeding episodes	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
Secondary	Haemostatic response of N9-GP when used in perioperative management	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)