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

Phase 3

Completed

Conditions: blood clothing disorder
Haemophillia A
10064477

Registration Number: NL-OMON43797

Lead Sponsor: ovo Nordisk

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 7

Inclusion Criteria

- Male patients with severe congenital haemophilia A (FVIII activity <1%, according to medical records)
- Documented history of at least 150 EDs to other FVIII products
- Age * 12 years and body weight * 35 kg (except for Croatia, The Netherlands, France, Russia and Israel where the lower age limit will be 18 years)

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 < 50,000 platelets/µL (laboratory value at the screening visit)
- ALAT > 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

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Co-Primary Endpoints<br /><br>* The Incidence rate of FVIII-inhibitors 0.6 BU<br /><br> Annualised bleeding rate for patients receiving prophylaxis treatment</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Secondary Endpoints<br /><br>* Haemostatic effect of N8-GP when used for treatment of bleeds, assessed on a<br /><br>four-point scale for haemostatic response (excellent, good, moderate and none)<br /><br>by counting excellent and good as success and moderate and none as failure<br /><br>* Consumption of N8-GP (number of infusions and U/kg) per bleed<br /><br>* Consumption of N8-GP (number of infusions and U/kg per month and per year)<br /><br>during prophylaxis and on-demand treatment<br /><br>* Haemostatic effect as measured by recovery and trough levels Factor VIII (in<br /><br>all patients receiving prophylaxis treatment)<br /><br>* Patient Reported Outcomes and Health Economic outcomes<br /><br>* Adverse Events (AEs) and Serious Adverse Events (SAEs) reported during the<br /><br>trial<br /><br>* Changes in vital signs (BP, pulse, temperature, respiratory rate)<br /><br>* Pharmacokinetic Outcomes</p><br>