Clinical trial NCT04574076: A Study Following Males With Haemophilia A on Prophylaxis With Esperoct®
Official title: A Multinational, Prospective, Open Labelled, Non-controlled, Non-interventional Post-authorisation Study of Turoctocog Alfa Pegol (N8-GP) During Long-term Routine Prophylaxis and Treatment of Bleeding Episodes in Patients With Haemophilia A
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Haemophilia A
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2020-10-05
Enrollment: 60
Sex: MALE
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: PROBABILITY_SAMPLE
Study population: Patients with haemophilia A

Brief summary: This study will collect information on side effects and how well Esperoct® (turoctocog alfa pegol (N8-GP)) works during long-term treatment (prophylaxis) in males with haemophilia A. Participants in this study will get the same treatment as they would normally get, if they were not participating in the study. All visits at the clinic are done in the same way as participants are used to, when visiting their doctor. During visits at the clinic participants might be asked for some relevant tests if considered useful by the study doctor. During the visits the study doctor might ask if participants had any side effects since the last study visit. Participants will be asked to note down in their own diary the number of bleeds and how these were treated, as well as their regular prophylaxis. Participation in the study will last for about 5-7 years, depending on when participants 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 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 Esperoct® 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.
* Male patients of all ages, according to local label, are allowed in this study
* Diagnosis of severe or moderate Haemophilia A

Exclusion Criteria:

* Previous participation in this study. Participation is defined as having given informed consent in this study
* Known or suspected hypersensitivity to N8-GP or related products
* Mental incapacity, unwillingness or language barriers precluding adequate understanding and cooperation
* Clinical suspicion or presence of FVIII inhibitors at time of inclusion

Primary outcomes:
measure: Number of Adverse Events (AEs) reported during the study period
timeFrame: From inclusion of the patient (Visit 1) until the end of study (Visit 3), duration 5-7 years
Secondary outcomes:
measure: Number of Serious Adverse Events (SAEs) reported during the study period
timeFrame: From inclusion of the patient (Visit 1) until the end of study (Visit 3), duration 5-7 years
Other outcomes:
Locations:
city: Wien
country: Austria
facility: AKH - Klin. Abt. f. Haematologie u. Haemostaseologie
city: Sofia
country: Bulgaria
facility: UMHAT "Tsaritsa Yoanna-ISUL"
city: Zagreb
country: Croatia
facility: KBC Zagreb, Rebro, Hemofilija centar
city: Brno
country: Czechia
facility: FN Brno odd. hematologie
city: Hradec Kralove
country: Czechia
facility: FN HK - IV. Interni hematologicka klinika
city: Tallinn
country: Estonia
facility: North Estonia Medical Centre Foundation
city: Tartu
country: Estonia
facility: Tartu University Hospital Haematology Clinic
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: Budapest
country: Hungary
facility: MH Eü. Központ -Orszagos Haemophilia Kozpont
city: Parma
country: Italy
facility: Azienda Ospedaliera-Universitaria Parma
city: Torino
country: Italy
facility: A.O.U Città Salute Scienza Torino
city: Vicenza
country: Italy
facility: Ospedale San Bortolo
city: Kaunas
country: Lithuania
facility: Hospital of LUHS "Kauno Klinikos"
city: Panevezys
country: Lithuania
facility: Republican Panevezys Hospital, Public consultation clinic
city: Vilnius
country: Lithuania
facility: Vilnius University hospital Santaros klinikos
city: Vilnius
country: Lithuania
facility: Vilnius University hospital Santaros klinikos
city: Lisboa
country: Portugal
facility: Centro Hospitalar Lisboa Norte-HSM
city: Porto
country: Portugal
facility: ULS São João, E.P.E.
city: Kosice
country: Slovakia
facility: Unilabs Slovensko, s. r. o.
city: Ljubljana
country: Slovenia
facility: PeK - University Children's Hospital, Department of haematology
city: Málaga
country: Spain
facility: Hospital Regional Universitario de Málaga
city: Zürich
country: Switzerland
facility: Kinderspital Hämatologie, Zürich
Study officials:
affiliation: Novo Nordisk A/S
name: Clinical Transparency (1452)
role: STUDY_DIRECTOR
References: