A Study Following Males With Haemophilia A on Prophylaxis With Esperoct®

• Conditions: Haemophilia A
• Interventions: Drug: Turoctocog alfa pegol (N8-GP)

• Registration Number: NCT04574076
• Lead Sponsor: Novo Nordisk A/S

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2020-09-21
• Study First Submitted QC: 2020-09-28
• Study First Posted: 2020-10-05
• Study Start: 2020-10-23
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-19
• Last Update Posted: 2025-05-20
• Primary Completion: 2027-06-03
• Study Completion: 2027-06-03

Recruitment & Eligibility

• Status: ENROLLING_BY_INVITATION
• Sex: Male
• Target Recruitment: 60

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
N8-GP	Turoctocog alfa pegol (N8-GP)	Patients with haemophilia A

Primary Outcome Measures

Name	Time	Method
Number of Adverse Events (AEs) reported during the study period	From inclusion of the patient (Visit 1) until the end of study (Visit 3), duration 5-7 years	Count of events

Secondary Outcome Measures

Name	Time	Method
Number of Serious Adverse Events (SAEs) reported during the study period	From inclusion of the patient (Visit 1) until the end of study (Visit 3), duration 5-7 years	Count of events

Trial Locations

Locations (24)

Hospital Regional Universitario de Málaga; 🇪🇸 Málaga, Spain

AKH - Klin. Abt. f. Haematologie u. Haemostaseologie; 🇦🇹 Wien, Austria

KBC Zagreb, Rebro, Hemofilija centar; 🇭🇷 Zagreb, Croatia

FN Brno odd. hematologie; 🇨🇿 Brno, Czechia

UMHAT "Tsaritsa Yoanna-ISUL"; 🇧🇬 Sofia, Bulgaria

FN HK - IV. Interni hematologicka klinika; 🇨🇿 Hradec Kralove, Czechia

North Estonia Medical Centre Foundation; 🇪🇪 Tallinn, Estonia

Tartu University Hospital Haematology Clinic; 🇪🇪 Tartu, Estonia

Vivantes Klinikum am Friedrichshain - Innere Medizin - Angiologie und Hämostaseologie; 🇩🇪 Berlin, Germany

Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie; 🇩🇪 Bonn, Germany

"Laiko" General Hospital of Athens; 🇬🇷 Athens, Greece

Aghia Sophia Childrens' Hospital; 🇬🇷 Athens, Greece

MH Eü. Központ -Orszagos Haemophilia Kozpont; 🇭🇺 Budapest, Hungary

Azienda Ospedaliera-Universitaria Parma; 🇮🇹 Parma, Italy

A.O.U Città Salute Scienza Torino; 🇮🇹 Torino, Italy

Ospedale San Bortolo; 🇮🇹 Vicenza, Italy

Hospital of LUHS "Kauno Klinikos"; 🇱🇹 Kaunas, Lithuania

Republican Panevezys Hospital, Public consultation clinic; 🇱🇹 Panevezys, Lithuania

Vilnius University hospital Santaros klinikos; 🇱🇹 Vilnius, Lithuania

Centro Hospitalar Lisboa Norte-HSM; 🇵🇹 Lisboa, Portugal

ULS São João, E.P.E.; 🇵🇹 Porto, Portugal

Unilabs Slovensko, s. r. o.; 🇸🇰 Kosice, Slovakia

PeK - University Children's Hospital, Department of haematology; 🇸🇮 Ljubljana, Slovenia

Kinderspital Hämatologie, Zürich; 🇨🇭 Zürich, Switzerland