pathfinder9: A Study Following Males With Haemophilia A on Prophylaxis With Esperoct®

• Sponsor: Novo Nordisk A/S (Industry)
• Overall Status: Enrolling by invitation
• CT.gov ID: NCT04574076
• Collaborator: (none)
• Enrollment: 60
• Locations: 15
• Anticipated Duration (Months): 79.3
• Patients Per Site: 4
• Patients Per Site Per Month: 0.1

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Haemophilia A	Drug: Turoctocog alfa pegol (N8-GP)

Study Design

• Study Type: Observational
• Anticipated Enrollment: 60 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: A Multinational, Prospective, Open Labelled, Non-controlled, Non-interventional Post-authorisation Study of Turoctocog Alfa Pegol (N8-GP) During Long-term Routine Prophylaxis and Treatment of Bleeding Episodes in Patients With Haemophilia A
• Actual Study Start Date: Oct 23, 2020
• Anticipated Primary Completion Date: Jun 3, 2027
• Anticipated Study Completion Date: Jun 3, 2027

Arms and Interventions

Arm

Intervention/Treatment

N8-GP Patients with haemophilia A

Drug: Turoctocog alfa pegol (N8-GP); 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.

Outcome Measures

Primary Outcome Measures

1. Number of Adverse Events (AEs) reported during the study period [From inclusion of the patient (Visit 1) until the end of study (Visit 3), duration 5-7 years]

   Count of events

Secondary Outcome Measures

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]

   Count of events

Eligibility Criteria

Criteria

• Ages Eligible for Study: N/A and Older
• Sexes Eligible for Study: Male
• Accepts Healthy Volunteers: No

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.

• Male patients of all ages, according to local label, are allowed in this study

• Diagnosis of severe or moderate Haemophilia A

Exclusion Criteria:

• 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

• Clinical suspicion or presence of FVIII inhibitors at time of inclusion

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Novo Nordisk Investigational Site	Wien		Austria	1090
2	Novo Nordisk Investigational Site	Sofia		Bulgaria	1527
3	Novo Nordisk Investigational Site	Zagreb		Croatia	10 000
4	Novo Nordisk Investigational Site	Brno		Czechia	625 00
5	Novo Nordisk Investigational Site	Hradec Kralove		Czechia	500 05
6	Novo Nordisk Investigational Site	Berlin		Germany	10249
7	Novo Nordisk Investigational Site	Bonn		Germany	53127
8	Novo Nordisk Investigational Site	Athens		Greece	GR-11527
9	Novo Nordisk Investigational Site	Budapest		Hungary	H-1134
10	Novo Nordisk Investigational Site	Torino		Italy	10126
11	Novo Nordisk Investigational Site	Vicenza		Italy	36100
12	Novo Nordisk Investigational Site	Porto		Portugal	4200-319
13	Novo Nordisk Investigational Site	Ljubljana		Slovenia	1000
14	Novo Nordisk Investigational Site	Málaga		Spain	29009
15	Novo Nordisk Investigational Site	Zürich		Switzerland	8032

Sponsors and Collaborators

• Novo Nordisk A/S

Investigators

• Study Director: Clinical Transparency (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: NCT04574076
• Other Study ID Numbers: NN7088-4029; EUPAS36536; U1111-1235-6007
• First Posted: Oct 5, 2020
• Last Update Posted: Mar 22, 2022
• Last Verified: Mar 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 22, 2022