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

NCT04574076 | Enrolling By Invitation

• 3 other identifiers: NN7088-4029EUPAS36536U1111-1235-6007
• Study Type: observational
• Target: 60
• Locations: 13 countries
• Sites: 24

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.

Conditions

Haemophilia A

Outcome Measures

Primary Outcomes (1)

• Number of Adverse Events (AEs) reported during the study period

  Count of events

  From inclusion of the patient (Visit 1) until the end of study (Visit 3), duration 5-7 years

Secondary Outcomes (1)

• 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

Study Arms (1)

N8-GP

Patients with haemophilia A

Drug: Turoctocog alfa pegol (N8-GP)

Interventions

Turoctocog alfa pegol (N8-GP) DRUG

Patients will be treated with commercially available N8-GP for prophylaxis and treatment of bleeding episodes according to routine clinical practice at the discretion of the treating physician. The decision to initiate treatment with commercially available N8-GP 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.

N8-GP

Eligibility Criteria

• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Probability Sample

Study Population

Patients with haemophilia A

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (27)

AKH - Klin. Abt. f. Haematologie u. Haemostaseologie

Vienna, 1090, Austria

Location

UMHAT "Tsaritsa Yoanna-ISUL"

Sofia, 1527, Bulgaria

Location

KBC Zagreb, Rebro, Hemofilija centar

Zagreb, 10 000, Croatia

Location

FN Brno odd. hematologie

Brno, 625 00, Czechia

Location

FN HK - IV. Interni hematologicka klinika

Hradec Králové, 500 05, Czechia

Location

North Estonia Medical Centre Foundation

Tallinn, 13419, Estonia

Location

Tartu University Hospital Haematology Clinic

Tartu, 50406, Estonia

Location

Vivantes Netzwerk für Gesundheit GmbH - Vivantes Klinikum im Friedrichshain

Berlin, 10249, Germany

Location

Universitätsklinikum Bonn - Institut für Experimentelle Hämatologie

Bonn, 53127, Germany

Location

"Laiko" General Hospital of Athens

Athens, 11527, Greece

Location

"Laiko" General Hospital of Athens

Athens, GR-11527, Greece

Location

Aghia Sophia Childrens' Hospital

Athens, GR-11527, Greece

Location

MH Eü. Központ -Orszagos Haemophilia Kozpont

Budapest, H-1134, Hungary

Location

Azienda Ospedaliera-Universitaria Parma

Parma, 43126, Italy

Location

Policlinico Umberto I Sezione Ematologia

Roma, 00161, Italy

Location

A.O.U Città Salute Scienza Torino

Torino, 10126, Italy

Location

Ospedale San Bortolo

Vicenza, 36100, Italy

Location

Hospital of LUHS "Kauno Klinikos"

Kaunas, LT-50161, Lithuania

Location

Republican Panevezys Hospital, Public consultation clinic

Panevezys, LT-35144, Lithuania

Location

Vilnius University hospital Santaros klinikos

Vilnius, 08661, Lithuania

Location

Vilnius University hospital Santaros klinikos

Vilnius, LT-08661, Lithuania

Location

Centro Hospitalar Lisboa Norte-HSM

Lisbon, 1649-035, Portugal

Location

ULS São João, E.P.E.

Porto, 4200-319, Portugal

Location

Unilabs Slovensko, s. r. o.

Košice, 04011, Slovakia

Location

PeK - University Children's Hospital, Department of haematology

Ljubljana, 1000, Slovenia

Location

Hospital Regional Universitario de Málaga

Málaga, 29009, Spain

Location

Kinderspital Hämatologie, Zürich

Zurich, 8032, Switzerland

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Clinical Transparency (1452)

  Novo Nordisk A/S

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 21, 2020
• First Posted: October 5, 2020
• Study Start: October 23, 2020
• Primary Completion (Estimated): June 3, 2027
• Study Completion (Estimated): June 3, 2027
• Last Updated: December 22, 2025

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will share

Locations

CH (1); LI (4); HU (1); ES (1); AU (1); PT (2); GR (3); DE (2); SL (1); BU (1); CR (1); IT (4); CZ (2)