Clinical trial NCT03745924: A Study Following Males With Haemophilia B on Prophylaxis With Refixia/REBINYN
Official title: A Non-Interventional Post-Authorisation Safety Study (PASS) in Male Haemophilia B Patients Receiving Nonacog Beta Pegol (N9-GP) Prophylaxis Treatment
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Haemophilia B
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2018-11-19
Enrollment: 60
Sex: MALE
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: 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Number of Adverse Drug Reactions (ADRs) (FIX inhibitors, allergic reactions, and thromboembolic events)
timeFrame: From start of study period (week 0) to end of study period (up to 9 years)
Secondary outcomes:
measure: Number of Serious Adverse Events (SAEs)
timeFrame: From start of study period (week 0) to up to 9 years
measure: Number of bleeding episodes during long-term routine use of N9-GP (prophylaxis) as assessed by annualised bleeding rate (ABR)
timeFrame: From start of study period (week 0) to up to 9 years
measure: Number of treatment requiring bleeding episodes during long-term routine use of N9-GP (prophylaxis) as assessed by annualised bleeding rate (ABR)
timeFrame: From start of study period (week 0) to up to 9 years
measure: Haemostatic effect of N9-GP when used for treatment of bleeding episodes
timeFrame: From start of study period (week 0) to up to 9 years
measure: Haemostatic response of N9-GP when used in perioperative management
timeFrame: From start of study period (week 0) to end of study period (up to 9 years)
Other outcomes:
Locations:
city: Wien
country: Austria
facility: AKH - Klin. Abt. f. Haematologie u. Haemostaseologie
city: Bruxelles
country: Belgium
facility: Cliniques universitaires Saint-Luc - Service Hématologie
city: Edmonton
country: Canada
facility: Univ of Alberta Hospital Res
state: Alberta
city: Winnipeg
country: Canada
facility: CancerCare Manitoba
state: Manitoba
city: St. John's
country: Canada
facility: Health Science Centre
state: Newfoundland and Labrador
city: Hamilton
country: Canada
facility: Hamilton Health Sciences Corp, Ontario
state: Ontario
city: Hamilton
country: Canada
facility: Hamltn Hth Sci/McMstr Child Hosp
state: Ontario
city: Toronto
country: Canada
facility: The Hospital for Sick Children
state: Ontario
city: Zagreb
country: Croatia
facility: KBC Zagreb, Rebro, Hemofilija centar
city: Brno
country: Czechia
facility: FN Brno odd. hematologie
city: Plzen
country: Czechia
facility: Fakultni nemocnice Plzen - Lochotin
city: Aarhus N
country: Denmark
facility: Skejby Blodsygdomme, blødercentret
city: Helsinki
country: Finland
facility: Helsinki University Central Hospital/Coagulation Disorder Un
city: Berlin
country: Germany
facility: Vivantes Klinikum am Friedrichshain - Innere Medizin - Angiologie und Hämostaseologie
city: Bonn
country: Germany
facility: Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie
city: Athens
country: Greece
facility: "Laiko" General Hospital of Athens
city: Athens
country: Greece
facility: "Laiko" General Hospital of Athens
city: Athens
country: Greece
facility: Aghia Sophia Childrens' Hospital
city: Oslo
country: Norway
facility: Klinisk forskningspost
city: Lisboa
country: Portugal
facility: Hospital São José
city: Lisboa
country: Portugal
facility: Centro Hospitalar Lisboa Central - Hospital Dona Estefânia
city: Cardiff
country: United Kingdom
facility: Arthur Bloom Haemophilia Centre
city: Sheffield
country: United Kingdom
facility: Royal Hallamshire Hospital
Study officials:
affiliation: Novo Nordisk A/S
name: Clinical Reporting Anchor and Disclosure (1452)
role: STUDY_DIRECTOR
References: