Post-Marketing Surveillance (Use-results Surveillance) With Esperoct®

Haemophilia A Clinical Trial

Official title:

Post-Marketing Surveillance (Use-results Surveillance) With Esperoct®. A Multi-centre, Prospective, Observational, Non-interventional Post-marketing Study to Investigate the Long-term Safety and Effectiveness of Esperoct® in Haemophilia A Patients Under Routine Clinical Practice Conditions in Japan

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT04334057
• Other study ID #: NN7088-4484
• Secondary ID: U1111-1216-4626
• Status: Enrolling by invitation
• Start date: March 31, 2021
• Est. completion date: February 28, 2025

Study information

• Verified date: November 2023
• Source: Novo Nordisk A/S
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

The purpose of this study is to assess the safety and effectiveness of Esperoct® for long-term routine use in patients with Haemophilia A. Participants will get Esperoct® as prescribed by their doctor. The study will last for about 2 years for each participant.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 15
• Est. completion date: February 28, 2025
• Est. primary completion date: February 28, 2025
• Gender: All
• Age group: N/A and older

Eligibility

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.

• Diagnosis of haemophilia A in males or females, no age limitation.

• New patients who have not previously been exposed to Esperoct®.

Exclusion Criteria:

• Previous participation in this study. Participation is defined as having given informed consent in this study.

• Known or suspected hypersensitivity to study product or related products.

• Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.

Related Conditions & MeSH terms

• Haemophilia A
• Hemophilia A

Intervention

Drug:
• Turoctocog alfa pegol
Patients will be treated with commercially available Esperoct® according to routine clinical practice at the discretion of the treating physician. 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.

Locations

Country	Name	City	State
Japan	Novo Nordisk Investigational Site	Fukuoka
Japan	Novo Nordisk Investigational Site	Gifu
Japan	Novo Nordisk Investigational Site	Iruma-gun, Saitama
Japan	Novo Nordisk Investigational Site	Kamigyo-ku, Kyoto
Japan	Novo Nordisk Investigational Site	Kanagawa
Japan	Novo Nordisk Investigational Site	Nara
Japan	Novo Nordisk Investigational Site	Okinawa
Japan	Novo Nordisk Investigational Site	Saitama
Japan	Novo Nordisk Investigational Site	Shiga
Japan	Novo Nordisk Investigational Site	Shizuoka
Japan	Novo Nordisk Investigational Site	Tokyo
Japan	Novo Nordisk Investigational Site	Tokyo
Japan	Novo Nordisk Investigational Site	Tokyo
Japan	Novo Nordisk Investigational Site	Yamagata
Japan	Novo Nordisk Investigational Site	Yokohama-shi, Kanagawa

Sponsors (1)

Lead Sponsor	Collaborator
Novo Nordisk A/S

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of adverse reactions (ARs) reported during the observation period	Count	From baseline (week 0) to end of study (week 104)
Secondary	Number of serious adverse events (SAEs) reported during the observation period	Count	From baseline (week 0) to end of study (week 104)
Secondary	Number of serious adverse reactions (SARs) reported during the observation period	Count	From baseline (week 0) to end of study (week 104)
Secondary	Number of patients who have confirmed inhibitory antibodies against FVIII during the observation period	Count	From baseline (week 0) to end of study (week 104)
Secondary	Number of bleeding episodes requiring treatment for patients using Esperoct® during the observation period assessed by annual bleeding rate (ABR)	Count	From baseline (week 0) to end of study (week 104)
Secondary	Evaluation of the haemostatic response of Esperoct® measured as number of successes for treatment requiring bleeds	Count, assessed based on a four-point scale (excellent, good, moderate and poor) by counting excellent and good as success and moderate and poor as failure	From baseline (week 0) to end of study (week 104)
Secondary	Evaluation of the haemostatic response of Esperoct® measured as number of successes in treatment of bleeds in perioperative management during surgical procedures	Count, assessed as success/failure based on a four-point scale for haemostatic response (excellent, good, moderate and poor) by counting excellent and good as success and moderate and poor as failure	From baseline (week 0) to end of study (week 104)