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

Sponsor

Novo Nordisk A/S (Industry)

Overall Status

Enrolling by invitation

CT.gov ID

NCT03875547

Collaborator

(none)

Enrollment

Locations

73.3

Anticipated Duration (Months)

0.9

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

Brief 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®.

Condition or Disease	Intervention/Treatment	Phase
Haemophilia B	Drug: Refixia®

Study Design

Study Type:

Observational

Anticipated Enrollment :

20 participants

Observational Model:

Cohort

Time Perspective:

Prospective

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.

Actual Study Start Date :

Aug 23, 2019

Anticipated Primary Completion Date :

Sep 30, 2025

Anticipated Study Completion Date :

Sep 30, 2025

Arms and Interventions

Arm	Intervention/Treatment
Patients with haemophilia B Both patients who have not previously been exposed to Refixia® and patients previously exposed to Refixia® in one of the clinical trials can be included.	Drug: Refixia® Patients will be treated with commercially available Refixia® according to routine clinical practice at the discretion of the treating physician

Arm

Intervention/Treatment

Patients with haemophilia B

Both patients who have not previously been exposed to Refixia® and patients previously exposed to Refixia® in one of the clinical trials can be included.

Drug: Refixia®

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

Outcome Measures

Primary Outcome Measures

Number of Adverse Reactions (ARs) [From baseline (week 0) to end of study (up to 6 years and 10 months)]
Count of events

Secondary Outcome Measures

Number of Serious Adverse Events (SAEs) [From baseline (week 0) to end of study (up to 6 years and 10 months)]
Count of events
Number of Serious Adverse Reactions (SARs) [From baseline (week 0) to end of study (up to 6 years and 10 months)]
Count of events
Number of bleeding episodes as assessed by annualised bleeding rate (ABR) [From baseline (week 0) to end of study (up to 6 years and 10 months)]
Count of bleeding episodes
Number of treatment requiring bleeding episodes as assessed by ABR [From baseline (week 0) to end of study (up to 6 years and 10 months)]
Count of bleeding episodes
Haemostatic response of Refixia® in treatment of bleeds [From baseline (week 0) to end of study (up to 6 years and 10 months)]
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.
Haemostatic response of Refixia® in treatment of bleeds in perioperative management during surgical procedures [From baseline (week 0) to end of study (up to 6 years and 10 months)]
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.

Eligibility Criteria

Criteria

Ages Eligible for Study:

N/A and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	Country	Postal Code
1	Novo Nordisk Investigational Site	Søborg	Denmark
2	Novo Nordisk Investigational Site	Aichi	Japan	466-8560
3	Novo Nordisk Investigational Site	Aomori	Japan	030-8553
4	Novo Nordisk Investigational Site	Chiba-shi, Chiba	Japan	260-8677
5	Novo Nordisk Investigational Site	Chiba	Japan	270-2296
6	Novo Nordisk Investigational Site	Chiba	Japan	286-8523
7	Novo Nordisk Investigational Site	Hyogo	Japan	654-0047
8	Novo Nordisk Investigational Site	Ibaraki	Japan	300-0395
9	Novo Nordisk Investigational Site	Ishikawa	Japan	920-8530
10	Novo Nordisk Investigational Site	Kanagawa	Japan	252-0375
11	Novo Nordisk Investigational Site	Kitakyusyu-shi, Fukuoka	Japan	807 8555
12	Novo Nordisk Investigational Site	Maebashi-shi, Gunma	Japan	371-8511
13	Novo Nordisk Investigational Site	Naha-shi, Okinawa	Japan	902-8511
14	Novo Nordisk Investigational Site	Nara	Japan	634-8522
15	Novo Nordisk Investigational Site	Nishinomiya-shi, Hyogo	Japan	663 8051
16	Novo Nordisk Investigational Site	Saitama	Japan	330-8777
17	Novo Nordisk Investigational Site	Tokushima	Japan	770-8503
18	Novo Nordisk Investigational Site	Tokushima	Japan	773-8502
19	Novo Nordisk Investigational Site	Tokyo	Japan	160-0023
20	Novo Nordisk Investigational Site	Tokyo	Japan	167-0035
21	Novo Nordisk Investigational Site	Yamagata	Japan	992-0035
22	Novo Nordisk Investigational Site	Yokohama-shi, Kanagawa	Japan	241-0811

Sponsors and Collaborators

Novo Nordisk A/S

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Novo Nordisk A/S

ClinicalTrials.gov Identifier:

NCT03875547

Other Study ID Numbers:

NN7999-4404
U1111-1198-6270

First Posted:

Mar 14, 2019

Last Update Posted:

Mar 15, 2022

Last Verified:

Mar 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Yes

Plan to Share IPD:

Yes

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Hemophilia A

Hemophilia B

Study Results

No Results Posted as of Mar 15, 2022