Post-marketing Surveillance (Use Result Surveillance) With Refixia®

Haemophilia B Clinical Trial

Official title:

Post-marketing Surveillance (Use Result Surveillance) With Refixia®. A Multicentre, Non-interventional Post Marketing Surveillance of Safety and Effectiveness of Refixia® in Routine Clinical Care With Haemophilia B Patients in Japan.

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT03875547
• Other study ID #: NN7999-4404
• Secondary ID: U1111-1198-6270
• Status: Enrolling by invitation
• Start date: August 23, 2019
• Est. completion date: September 30, 2025

Study information

• Verified date: July 2023
• Source: Novo Nordisk A/S
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The participants are invited to take part in this study because they have Haemophilia B. The purpose of this study is to assess the safety and effectiveness of Refixia® about long-term routine use in patients with Haemophilia B. The participants will get Refixia® as prescribed to them by their study doctor. The study will last up to Sep 2025 for the participant. The participants may be asked to fill in the quality of life questionnaires (if they are above age of 15). The blood samples taken from the participants as part of routine clinical practice will also be used to investigate the safety for the long-term use of Refixia®.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 20
• Est. completion date: September 30, 2025
• Est. primary completion date: September 30, 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

• Signed consent obtained before any study-related activities (study-related activities are any procedure related to recording of data according to the protocol).

• The decision to initiate treatment with commercially available Refixia® has been made by the patient/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study. At each site of this study, all patients will be registered consecutively from the first patient after the launch of Refixia® (consecutively registered system).

• Diagnosis of haemophilia B in males or females, no age limitation. Patients younger than 12 years old will continue to be registered for 3 years of recruitment period regardless of the target number of patients.

• New patients who have not been previously exposed to Refixia®. Also patients previously exposed to Refixia® in NN7999-3639, -3747, -3774, -3775 or -3895 clinical trial can be enrolled in this study. The patients who have participated in NN7999 -3774 or -3895 clinical trial can be enrolled in this study as continuous cases until 30-September-2024 (one year before planned end of study date).

Exclusion Criteria:

• Previous participation in this study. Participation is defined as having given informed consent in this study

• Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation

• Known or suspected hypersensitivity to study product or related products.

Related Conditions & MeSH terms

• Haemophilia B
• Hemophilia A
• Hemophilia B

Intervention

Drug:
• Refixia®
Patients will be treated with commercially available Refixia® according to routine clinical practice at the discretion of the treating physician

Locations

Country	Name	City	State
Denmark	Novo Nordisk Investigational Site	Søborg
Japan	Novo Nordisk Investigational Site	Aichi
Japan	Novo Nordisk Investigational Site	Aomori
Japan	Novo Nordisk Investigational Site	Chiba
Japan	Novo Nordisk Investigational Site	Chiba-shi, Chiba
Japan	Novo Nordisk Investigational Site	Hyogo
Japan	Novo Nordisk Investigational Site	Ibaraki
Japan	Novo Nordisk Investigational Site	Kanagawa
Japan	Novo Nordisk Investigational Site	Kanagawa
Japan	Novo Nordisk Investigational Site	Kitakyusyu-shi, Fukuoka
Japan	Novo Nordisk Investigational Site	Maebashi-shi, Gunma
Japan	Novo Nordisk Investigational Site	Naha-shi, Okinawa
Japan	Novo Nordisk Investigational Site	Nara
Japan	Novo Nordisk Investigational Site	Nishinomiya-shi, Hyogo
Japan	Novo Nordisk Investigational Site	Saitama
Japan	Novo Nordisk Investigational Site	Tokushima
Japan	Novo Nordisk Investigational Site	Tokushima
Japan	Novo Nordisk Investigational Site	Tokyo
Japan	Novo Nordisk Investigational Site	Tokyo
Japan	Novo Nordisk Investigational Site	Yamagata
Japan	Novo Nordisk Investigational Site	Yokohama-shi, Kanagawa

Sponsors (1)

Lead Sponsor	Collaborator
Novo Nordisk A/S

Countries where clinical trial is conducted

Denmark,  Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Adverse Reactions (ARs)	Count of events	From baseline (week 0) to end of study (up to 6 years and 10 months)
Secondary	Number of Serious Adverse Events (SAEs)	Count of events	From baseline (week 0) to end of study (up to 6 years and 10 months)
Secondary	Number of Serious Adverse Reactions (SARs)	Count of events	From baseline (week 0) to end of study (up to 6 years and 10 months)
Secondary	Number of bleeding episodes as assessed by annualised bleeding rate (ABR)	Count of bleeding episodes	From baseline (week 0) to end of study (up to 6 years and 10 months)
Secondary	Number of treatment requiring bleeding episodes as assessed by ABR	Count of bleeding episodes	From baseline (week 0) to end of study (up to 6 years and 10 months)
Secondary	Haemostatic response of Refixia® in treatment of bleeds	Haemostatic response is assessed as success/failure based on a four-point scale (excellent, good, moderate and poor) by counting excellent and good as success and moderate and poor as failure.	From baseline (week 0) to end of study (up to 6 years and 10 months)
Secondary	Haemostatic response of Refixia® in treatment of bleeds in perioperative management during surgical procedures	Haemostatic response is assessed as success/failure based on a four-point scale (excellent, good, moderate and poor) by counting excellent and good as success and moderate and poor as failure.	From baseline (week 0) to end of study (up to 6 years and 10 months)