Clinical trial NCT03875547: Post-marketing Surveillance (Use Result Surveillance) With Refixia®
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.
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Haemophilia B
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2019-03-14
Enrollment: 20
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with haemophilia B

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®.


Eligibility criteria: 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.

Primary outcomes:
measure: Number of Adverse Reactions (ARs)
timeFrame: From baseline (week 0) to end of study (up to 6 years and 10 months)
Secondary outcomes:
measure: Number of Serious Adverse Events (SAEs)
timeFrame: From baseline (week 0) to end of study (up to 6 years and 10 months)
measure: Number of Serious Adverse Reactions (SARs)
timeFrame: From baseline (week 0) to end of study (up to 6 years and 10 months)
measure: Number of bleeding episodes as assessed by annualised bleeding rate (ABR)
timeFrame: From baseline (week 0) to end of study (up to 6 years and 10 months)
measure: Number of treatment requiring bleeding episodes as assessed by ABR
timeFrame: From baseline (week 0) to end of study (up to 6 years and 10 months)
measure: Haemostatic response of Refixia® in treatment of bleeds
timeFrame: From baseline (week 0) to end of study (up to 6 years and 10 months)
measure: Haemostatic response of Refixia® in treatment of bleeds in perioperative management during surgical procedures
timeFrame: From baseline (week 0) to end of study (up to 6 years and 10 months)
Other outcomes:
Locations:
city: Søborg
country: Denmark
facility: Novo Nordisk Investigational Site
city: Aichi
country: Japan
facility: Nagoya University Hospital_Blood Transfusion
city: Chiba-shi, Chiba
country: Japan
facility: Chiba University Hospital_Diabetes, Metabolism and Endocrinology
city: Hyogo
country: Japan
facility: Hyogo prefectural kobe children's hospital
city: Ibaraki
country: Japan
facility: Tokyo Medical University Ibaraki Medical Center_Ibaraki
city: Kanagawa
country: Japan
facility: St. Marianna University School of Medicine Hospital_Pediatrics
city: Kanagawa
country: Japan
facility: Kitasato University Hospital
city: Kitakyusyu-shi, Fukuoka
country: Japan
facility: Hospital of the University of Occupational And Environmental Health Japan, Pediatrics
city: Maebashi-shi, Gunma
country: Japan
facility: Gunma University Hospital, Dept. of Hematology
city: Naha-shi, Okinawa
country: Japan
facility: Naha City Hospital
city: Nara
country: Japan
facility: Nara Medical University Hospital_Pediatrics
city: Nishinomiya-shi, Hyogo
country: Japan
facility: The Hospital of Hyogo College of Medicine
city: Saitama
country: Japan
facility: Saitama Children's Med Centre_Hematology-Oncology
city: Tokushima
country: Japan
facility: Tokushima Red Cross Hospital
city: Tokyo
country: Japan
facility: Tokyo Medical Univ. Hospital_Laboratory Medicine
city: Tokyo
country: Japan
facility: Ogikubo Hospital_Tokyo
city: Yamagata
country: Japan
facility: Ishiyama Clinic
Study officials:
References: