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

• Sponsor: Novo Nordisk A/S (Industry)
• Overall Status: Enrolling by invitation
• CT.gov ID: NCT04334057
• Collaborator: (none)
• Enrollment: 15
• Locations: 14
• Anticipated Duration (Months): 47
• Patients Per Site: 1.1
• Patients Per Site Per Month: 0

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Haemophilia A	Drug: Turoctocog alfa pegol

Study Design

• Study Type: Observational
• Anticipated Enrollment: 15 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• 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
• Actual Study Start Date: Mar 31, 2021
• Anticipated Primary Completion Date: Feb 28, 2025
• Anticipated Study Completion Date: Feb 28, 2025

Arms and Interventions

Arm

Intervention/Treatment

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; 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.

Outcome Measures

Primary Outcome Measures

1. Number of adverse reactions (ARs) reported during the observation period [From baseline (week 0) to end of study (week 104)]

   Count

Secondary Outcome Measures

1. Number of serious adverse events (SAEs) reported during the observation period [From baseline (week 0) to end of study (week 104)]

   Count

2. Number of serious adverse reactions (SARs) reported during the observation period [From baseline (week 0) to end of study (week 104)]

   Count

3. Number of patients who have confirmed inhibitory antibodies against FVIII during the observation period [From baseline (week 0) to end of study (week 104)]

   Count

4. 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)]

   Count

5. 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)]

   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

6. Evaluation of the haemostatic response of Esperoct® measured as number of successes in treatment of bleeds in perioperative management during surgical procedures [From baseline (week 0) to end of study (week 104)]

   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

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A and Older
• Sexes Eligible for Study: All

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Novo Nordisk Investigational Site	Fukuoka		Japan	830-0011
2	Novo Nordisk Investigational Site	Gifu		Japan	501-1194
3	Novo Nordisk Investigational Site	Iruma-gun, Saitama		Japan	350 0495
4	Novo Nordisk Investigational Site	Kanagawa		Japan	216-8511
5	Novo Nordisk Investigational Site	Nara		Japan	634-8522
6	Novo Nordisk Investigational Site	Okinawa		Japan	901-1193
7	Novo Nordisk Investigational Site	Saitama		Japan	350-0225
8	Novo Nordisk Investigational Site	Shiga		Japan	520-2145
9	Novo Nordisk Investigational Site	Shizuoka		Japan	420-8660
10	Novo Nordisk Investigational Site	Tokyo		Japan	113-8603
11	Novo Nordisk Investigational Site	Tokyo		Japan	160-0023
12	Novo Nordisk Investigational Site	Tokyo		Japan	167-0035
13	Novo Nordisk Investigational Site	Yamagata		Japan	998-8501
14	Novo Nordisk Investigational Site	Yokohama-shi, Kanagawa		Japan	241-0811

Sponsors and Collaborators

• Novo Nordisk A/S

Investigators

• Study Director: Clinical Reporting Anchor & Disclosure (1452), Novo Nordisk A/S

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: Novo Nordisk A/S
• ClinicalTrials.gov Identifier: NCT04334057
• Other Study ID Numbers: NN7088-4484; U1111-1216-4626
• First Posted: Apr 3, 2020
• Last Update Posted: Mar 2, 2022
• Last Verified: Feb 1, 2022

• Individual Participant Data (IPD) Sharing Statement: Yes
• Plan to Share IPD: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Additional relevant MeSH terms: Hemophilia A

Study Results

No Results Posted as of Mar 2, 2022