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

NCT04334057 | Completed

• 2 other identifiers: NN7088-4484U1111-1216-4626
• Study Type: observational
• Target: 23
• Locations: 1 country
• Sites: 19

Brief 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.

Conditions

Haemophilia A

Outcome Measures

Primary Outcomes (1)

• Number of adverse reactions (ARs) reported during the observation period

  Count

  From baseline (week 0) to end of study (week 104)

Secondary Outcomes (6)

• Number of serious adverse events (SAEs) reported during the observation period

  From baseline (week 0) to end of study (week 104)

• Number of serious adverse reactions (SARs) reported during the observation period

  From baseline (week 0) to end of study (week 104)

• Number of patients who have confirmed inhibitory antibodies against FVIII during the observation period

  From baseline (week 0) to end of study (week 104)

• Number of bleeding episodes requiring treatment for patients using Esperoct® during the observation period assessed by annual bleeding rate (ABR)

  From baseline (week 0) to end of study (week 104)

• Evaluation of the haemostatic response of Esperoct® measured as number of successes for treatment requiring bleeds

  From baseline (week 0) to end of study (week 104)

Study Arms (1)

Patients with haemophilia A

New patients who have not previously been exposed to Esperoct® (Turoctocog alfa pegol or N8-GP in clinical trials) are eligible for this study.

Drug: Turoctocog alfa pegol

Interventions

Turoctocog alfa pegol DRUG

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.

Patients with haemophilia A

Eligibility Criteria

• Sex: all
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Haemophilia A patients in routine clinical practice in Japan

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.
• Diagnosis of haemophilia A in males or females, no age limitation.
• New patients who have not previously been exposed to Esperoct®.

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 study product or related products.
• Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.

Sponsors & Collaborators

• Novo Nordisk A/S: lead

Study Sites (19)

Kurume University Hospital, Pediatrics

Fukuoka, 830-0011, Japan

Location

Gifu University Hospital

Gifu, 501-1194, Japan

Location

Saitama Medical University Hospital, Pediatrics

Iruma-gun, Saitama, 350 0495, Japan

Location

University Hospital Kyoto Prefectual University of Medicine

Kamigyo-ku, Kyoto, 602-8566, Japan

Location

St. Marianna University School of Medicine Hospital_Pediatrics

Kanagawa, 216-8511, Japan

Location

St. Marianna University School of Medicine Hospital

Kanagawa, 216-8511, Japan

Location

Nara Medical University Hospital_Pediatrics

Nara, 634-8522, Japan

Location

Nanbu Medical Center & Children's Medical Center

Okinawa, 901-1193, Japan

Location

Shibuya Children's Clinic, Department of Pediatric

Saitama, 350-0225, Japan

Location

Lake Children Clinic

Shiga, 520-2145, Japan

Location

Shizuoka Children's Hospital, Hematology-Oncology

Shizuoka, 420-8660, Japan

Location

Shizuoka Children's Hospital

Shizuoka, 420-8660, Japan

Location

Nippon Medical School Hospital

Tokyo, 113-8603, Japan

Location

Tokyo Medical Univ. Hospital_Laboratory Medicine

Tokyo, 160-0023, Japan

Location

Tokyo Medical Univ. Hospital

Tokyo, 160-0023, Japan

Location

Ogikubo Hospital_Tokyo

Tokyo, 167-0035, Japan

Location

Nihonkai Sogo Hospital_Internal Medicine

Yamagata, 998-8501, Japan

Location

Nihonkai Sogo Hospital

Yamagata, 998-8501, Japan

Location

St. Marianna Univ., Yokohama City Seibu HP, Pediatrics Dept,

Yokohama-shi, Kanagawa, 241-0811, Japan

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 Reporting Anchor & Disclosure (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: April 2, 2020
• First Posted: April 3, 2020
• Study Start: March 31, 2021
• Primary Completion: November 30, 2023
• Study Completion: January 10, 2024
• Last Updated: August 29, 2025

Record last verified: 2025-08

Data Sharing

• IPD Sharing: Will share

Locations

JP (19)