A Multi-national Trial Evaluating Safety and Efficacy, including Pharmacokinetics, of NNC 0129-0000-1003 when Administered for Treatment and Prophylaxis of Bleeding in Patients with Haemophilia A

A Multi-national Trial Evaluating Safety and Efficacy, including Pharmacokinetics, of NNC 0129-0000-1003 (N8-GP) when Administered for Treatment and Prophylaxis of Bleeding in Patients with Haemophilia A - pathfinder™ 2

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2011-001142-15-DE

Enrollment

180

Registered

2011-12-21

Start date

2012-01-10

Completion date

Unknown

Last updated

2019-02-28

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 10018937 Term: Haemophilia A System Organ Class: 100000004850

Interventions

Product Name: N8-GP rFVIII Pharmaceutical Form: Powder and solvent for solution for injection INN or Proposed INN: N/A CAS Number: 1309086-46-1

Eligibility

Sex/Gender

Male

Inclusion criteria

Inclusion criteria: - Male patients with severe congenital haemophilia A (FVIII activity =65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: - Previous participation in this trial defined as withdrawal after administration N8-GP - Any history of FVIII inhibitors - FVIII inhibitors = 0.6 BU/mL at screening - HIV positive, defined by medical records with CD4+ count =200/µL or a viral load of >400000 copies/mL If the data is not available in medical records within last 6 months, CD4+ will be measured at the screening visit - Congenital or acquired coagulation disorders other than haemophilia A - Previous significant thromboembolic events (e.g. myocardial infarction, cerebrovascular disease or deep venous thrombosis) as defined by available medical records - Platelet count 3 times the upper limit of normal reference ranges at central laboratory - Creatinine level = 1.5 times above upper normal limit (according to central laboratory reference ranges) - Ongoing immune modulating or chemotherapeutic medication

Design outcomes

Primary

Measure	Time frame
Main Objective: - To evaluate the immunogenicity of NNC 0129-0000-1003 (hereafter referred to as N8-GP) in previously treated patients with Haemophilia A - To evaluate the clinical efficacy of N8-GP in bleeding prophylaxis (number of bleeds during prophylaxis) ; Secondary Objective: - To evaluate the clinical efficacy of N8-GP when treating bleeds in patients with haemophilia A - To evaluate the safety of N8-GP when used for prevention of bleeds and treatment of bleeds in patients with haemophilia A - To evaluate PK properties of N8-GP - To evaluate Patient Reported Outcomes - To evaluate the health economic impact of N8-GP treatment - Generation of a population based PK-model for N8-GP ; Primary end point(s): - The Incidence rate of FVIII-inhibitors =0.6 BU - Annualised bleeding rate in the prophylaxis arm ;Timepoint(s) of evaluation of this end point: The endpoints will be analysed based on all available information after approximately 24 and 36 months and until the end of trial (EOT) visit.	—

Measure

Time frame

Main Objective: - To evaluate the immunogenicity of NNC 0129-0000-1003 (hereafter referred to as N8-GP) in previously treated patients with Haemophilia A - To evaluate the clinical efficacy of N8-GP in bleeding prophylaxis (number of bleeds during prophylaxis) ; Secondary Objective: - To evaluate the clinical efficacy of N8-GP when treating bleeds in patients with haemophilia A - To evaluate the safety of N8-GP when used for prevention of bleeds and treatment of bleeds in patients with haemophilia A - To evaluate PK properties of N8-GP - To evaluate Patient Reported Outcomes - To evaluate the health economic impact of N8-GP treatment - Generation of a population based PK-model for N8-GP ; Primary end point(s): - The Incidence rate of FVIII-inhibitors =0.6 BU - Annualised bleeding rate in the prophylaxis arm ;Timepoint(s) of evaluation of this end point: The endpoints will be analysed based on all available information after approximately 24 and 36 months and until the end of trial (EOT) visit.

—

Secondary

Measure	Time frame
Secondary end point(s): - Haemostatic effect of N8-GP when used for treatment of bleeds, assessed on a four-point scale for haemostatic response (excellent, good, moderate and none) by counting excellent and good as success and moderate and none as failure.;Timepoint(s) of evaluation of this end point: The endpoints will be analysed based on all available information until the end of trial (EOT) visit and up to approximately 24 and 36 months.	—

Measure

Time frame

Secondary end point(s): - Haemostatic effect of N8-GP when used for treatment of bleeds, assessed on a four-point scale for haemostatic response (excellent, good, moderate and none) by counting excellent and good as success and moderate and none as failure.;Timepoint(s) of evaluation of this end point: The endpoints will be analysed based on all available information until the end of trial (EOT) visit and up to approximately 24 and 36 months.

—

Countries

Australia, Brazil, Bulgaria, Croatia, Denmark, France, Germany, Hungary, Italy, Japan, Korea, Republic of, Malaysia, Netherlands, Norway, Russian Federation, Spain, Sweden, Switzerland, Taiwan, Turkey, United Kingdom, 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