Haemophilia ANCT04334057
Post-Marketing Surveillance (Use-results Surveillance) With Esperoct®
An observational study of Haemophilia A, run by Novo Nordisk A/S at 20 sites in Japan.
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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Haemophilia A
- What is being tested?
- Turoctocog alfa pegol
- How many people
- 23 participants
- Where
- 20 sitesJapan
- Study dates
- 31 Mar 2021to 10 Jan 2024
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- Child, Adult, Older adult
Study population: Haemophilia A patients in routine clinical practice in Japan
Who may be able to join
- 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®.
Who cannot join
- 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.
Trial locations and contacts
20 sites in 1 country.
20 sites in 1 country
Japan20 sites
Kurume University Hospital, Pediatrics
Fukuoka, 830-0011
Gifu University Hospital_The Third Dept. of Internal Medicine
Gifu, 501-1194
Gifu University Hospital
Gifu, 501-1194
Saitama Medical University Hospital, Pediatrics
Iruma-gun, Saitama, 350 0495
University Hospital Kyoto Prefectual University of Medicine
Kamigyo-ku, Kyoto, 602-8566
St. Marianna University School of Medicine Hospital_Pediatrics
Kanagawa, 216-8511
St. Marianna University School of Medicine Hospital
Kanagawa, 216-8511
Nara Medical University Hospital_Pediatrics
Nara, 634-8522
Nanbu Medical Center & Children's Medical Center_Pediatric Hematology
Okinawa, 901-1193
Shibuya Children's Clinic, Department of Pediatric
Saitama, 350-0225
Lake Children Clinic
Shiga, 520-2145
Shizuoka Children's Hospital, Hematology-Oncology
Shizuoka, 420-8660
Shizuoka Children's Hospital
Shizuoka, 420-8660
Nippon Medical School Hospital_Haematology
Tokyo, 113-8603
Tokyo Medical Univ. Hospital_Laboratory Medicine
Tokyo, 160-0023
Tokyo Medical Univ. Hospital
Tokyo, 160-0023
Ogikubo Hospital_Blood Coagulation
Tokyo, 167-0035
Nihonkai Sogo Hospital_Internal Medicine
Yamagata, 998-8501
Nihonkai Sogo Hospital
Yamagata, 998-8501
St. Marianna Univ., Yokohama City Seibu HP, Pediatrics Dept,
Yokohama-shi, Kanagawa, 241-0811
About this study
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
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
- Being tested
- Turoctocog alfa pegol
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Turoctocog alfa pegolDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Drug: Turoctocog alfa pegol |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of adverse reactions (ARs) reported during the observation period
Count
Measured: From baseline (week 0) to end of study (week 104)
Secondary outcomes (6)
Number of serious adverse events (SAEs) reported during the observation period
Count
Measured: From baseline (week 0) to end of study (week 104)
Number of serious adverse reactions (SARs) reported during the observation period
Count
Measured: From baseline (week 0) to end of study (week 104)
Number of patients who have confirmed inhibitory antibodies against FVIII during the observation period
Count
Measured: 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)
Count
Measured: 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
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
Measured: From baseline (week 0) to end of study (week 104)
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
Measured: From baseline (week 0) to end of study (week 104)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2020
- Study start31 Mar 2021
- Primary completion30 Nov 2023
- Study completion10 Jan 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Haemophilia AStudies of the same condition, open and under way.
- More trials run by Novo Nordisk A/SThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novo Nordisk A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Reporting Anchor & Disclosure (1452), Study director, Novo Nordisk A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04334057
- Sponsor's protocol ID
- NN7088-4484
- Other IDs
- U1111-1216-4626
- Status last verified
- Jun 2026
- First submitted
- 2 Apr 2020
- Last updated
- 15 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
Study at a glance
From the registry record, last updated by the sponsor on 15 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04334057
- Acronym / sponsor ID
- NN7088-4484
- Conditions
- Haemophilia A
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Novo Nordisk A/SIndustry (pharma, biotech, devices)
- Study start
- 31 Mar 2021 (actual)
- Primary completion
- 30 Nov 2023 (actual)
- Study completion
- 10 Jan 2024 (actual)
- First posted
- 3 Apr 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with haemophilia A | – | Drug: Turoctocog alfa pegolNew patients who have not previously been exposed to Esperoct® (Turoctocog alfa pegol or N8-GP in clinical trials) are eligible for this study. |
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- Haemophilia A patients in routine clinical practice in Japan
Key inclusion criteria (4 listed)
- 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®.
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Turoctocog alfa pegolPatients 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. | Drug | Patients with haemophilia A |
Primary outcome
Number of adverse reactions (ARs) reported during the observation period
Time frame: From baseline (week 0) to end of study (week 104)
Count
6 secondary outcomes
Number of serious adverse events (SAEs) reported during the observation period
Time frame: From baseline (week 0) to end of study (week 104)
Number of serious adverse reactions (SARs) reported during the observation period
Time frame: From baseline (week 0) to end of study (week 104)
Number of patients who have confirmed inhibitory antibodies against FVIII during the observation period
Time frame: 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)
Time frame: 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
Time frame: From baseline (week 0) to end of study (week 104)
Evaluation of the haemostatic response of Esperoct® measured as number of successes in treatment of bleeds in perioperative management during surgical procedures
Time frame: From baseline (week 0) to end of study (week 104)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jun 2026.
- Individual participant dataSharing planned: Yes. According to the Novo Nordisk disclosure commitment on novonordisk-trials.com
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Reporting Anchor & Disclosure (1452) study director
Novo Nordisk A/S
20 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Haemophilia A, run by Novo Nordisk A/S at 20 sites in Japan.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 20 sites in Japan. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of adverse reactions (ARs) reported during the observation period (time frame: From baseline (week 0) to end of study (week 104)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Jun 2026.