Safety and Efficacy of turoctocog alfa pegol (N8-GP) in Previously Untreated Patients with Haemophilia A

Safety and Efficacy of turoctocog alfa pegol (N8-GP) in Previously Untreated Patients with Haemophilia A An open-label single-arm multicentre non-controlled phase 3a trial investigating safety and efficacy of N8-GP in prophylaxis and treatment of bleeding episodes in previously untreated paediatric patients with severe haemophilia A - pathfinder™6

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2013-004025-88-DE

Enrollment

125

Registered

2014-02-18

Start date

2014-04-28

Completion date

Unknown

Last updated

2021-10-26

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Haemophilia A MedDRA version: 20.0 Level: LLT Classification code 10018938 Term: Haemophilia A (Factor VIII) System Organ Class: 100000004850

Interventions

Product Name: N8-GP rFVIII Pharmaceutical Form: Powder and solvent for solution for injection INN or Proposed INN: TUROCTOCOG ALFA PEGOL Current Sponsor code: NNC129-1003 Concentration unit: U unit(s)

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: - Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial - Male, age =65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: - Any history of FVIII inhibitor (defined by medical records) - Known or suspected hypersensitivity to trial product or related products - Previous participation in this trial. Participation is defined as administration of trial product - Receipt of any investigational medicinal product within 30 days before screening - Congenital or acquired coagulation disorder other than haemophilia A. Any chronic disorder or severe disease which, in the opinion of the Investigator, might jeopardise patient’s safety or compliance with the protocol. - Patient’s parent(s)/legally acceptable representative(s) mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate immunogenicity of N8-GP (turoctocog alfa pegol) in previously untreated patients (PUPs) with severe haemophilia A;Secondary Objective: - To evaluate safety other than immunogenicity of N8-GP (turoctocog alfa pegol) in PUPs with severe haemophilia A - To evaluate efficacy of N8-GP (turoctocog alfa pegol) in PUPs with severe haemophilia A o in long-term prophylaxis treatment (bleeding preventive effect) o in the treatment of bleeding episodes;Primary end point(s): Incidence of FVIII inhibitors;Timepoint(s) of evaluation of this end point: The primary endpoint will be evaluated when the first 50 PUPs have reached at least 50 exposure days and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days.	—

Measure

Time frame

Main Objective: To evaluate immunogenicity of N8-GP (turoctocog alfa pegol) in previously untreated patients (PUPs) with severe haemophilia A;Secondary Objective: - To evaluate safety other than immunogenicity of N8-GP (turoctocog alfa pegol) in PUPs with severe haemophilia A - To evaluate efficacy of N8-GP (turoctocog alfa pegol) in PUPs with severe haemophilia A o in long-term prophylaxis treatment (bleeding preventive effect) o in the treatment of bleeding episodes;Primary end point(s): Incidence of FVIII inhibitors;Timepoint(s) of evaluation of this end point: The primary endpoint will be evaluated when the first 50 PUPs have reached at least 50 exposure days and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days.

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Secondary

Measure	Time frame
Secondary end point(s): - Frequency of adverse events including serious adverse events and medical events of special interest. - Incidence of confirmed high titre inhibitors (defined as inhibitor titre > 5BU). - Number of breakthrough bleeding episodes during prophylaxis with N8-GP (annualised bleeding rate). - Haemostatic effect of N8-GP in treatment of bleeding episodes, assessed by a predefined 4-point haemostatic response scale “excellent”, “good”, “moderate” and “none”).;Timepoint(s) of evaluation of this end point: The secondary endpoints will be evaluated when the first 50 PUPs have reached at least 50 exposure days and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days.	—

Measure

Time frame

Secondary end point(s): - Frequency of adverse events including serious adverse events and medical events of special interest. - Incidence of confirmed high titre inhibitors (defined as inhibitor titre > 5BU). - Number of breakthrough bleeding episodes during prophylaxis with N8-GP (annualised bleeding rate). - Haemostatic effect of N8-GP in treatment of bleeding episodes, assessed by a predefined 4-point haemostatic response scale “excellent”, “good”, “moderate” and “none”).;Timepoint(s) of evaluation of this end point: The secondary endpoints will be evaluated when the first 50 PUPs have reached at least 50 exposure days and at end of trial. End of trial will be up to 4 years after the first patient has reached 100 exposure days.

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Countries

Algeria, Australia, Austria, Bulgaria, Canada, European Union, France, Germany, Greece, Israel, Italy, Japan, Malaysia, Portugal, Romania, Spain, Taiwan, Thailand, United States

Contacts

Public ContactGlobal Clinical Registry (GCR,1452)

Novo Nordisk A/S

clinicaltrials@novonordisk.com

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026