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

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

NL-OMON

Registry ID

NL-OMON43797

Enrollment

Registered

2011-11-14

Start date

2012-07-03

Completion date

Unknown

Last updated

2024-05-06

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

Conditions

blood clothing disorder Haemophillia A

Interventions

Injections with N8-GP every 4 days (prophylaxis) and/or injections with N8-GP at the first signs of a bleeding episode (on-demand). In the Extension Phase it is also possible to have injections with

Eligibility

Age

18 Years to 99 Years

Inclusion criteria

Inclusion criteria: - Male patients with severe congenital haemophilia A (FVIII activity

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
Co-Primary Endpoints * The Incidence rate of FVIII-inhibitors 0.6 BU Annualised bleeding rate for patients receiving prophylaxis treatment	—

Secondary

Measure	Time frame
Secondary Endpoints * 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 * Consumption of N8-GP (number of infusions and U/kg) per bleed * Consumption of N8-GP (number of infusions and U/kg per month and per year) during prophylaxis and on-demand treatment * Haemostatic effect as measured by recovery and trough levels Factor VIII (in all patients receiving prophylaxis treatment) * Patient Reported Outcomes and Health Economic outcomes * Adverse Events (AEs) and Serious Adverse Events (SAEs) reported during the trial * Changes in vital signs (BP, pulse, temperature, respiratory rate) * Pharmacokinetic Outcomes	—

Measure

Time frame

Secondary Endpoints * 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 * Consumption of N8-GP (number of infusions and U/kg) per bleed * Consumption of N8-GP (number of infusions and U/kg per month and per year) during prophylaxis and on-demand treatment * Haemostatic effect as measured by recovery and trough levels Factor VIII (in all patients receiving prophylaxis treatment) * Patient Reported Outcomes and Health Economic outcomes * Adverse Events (AEs) and Serious Adverse Events (SAEs) reported during the trial * Changes in vital signs (BP, pulse, temperature, respiratory rate) * Pharmacokinetic Outcomes

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Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)