A Multi-centre, Single-blind Trial Evaluating Safety and Efficacy, including Pharmacokinetics, of NNC-0156-0000-0009 when used for Treatment and Prophylaxis of Bleeding Episodes in Patients with Haemophilia B

Phase 3

Completed

Conditions: blood clotting disorder
Haemophilia B
10064477

Registration Number: NL-OMON36266

Lead Sponsor: ovo Nordisk

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 2

Inclusion Criteria

* Male patients, aged 13-70 years, with moderately severe or severe congenital haemophilia B with a FIX activity *2% according to medical records
NB: In The Netherlands only patients aged 18-70 years will be included
* History of at least 150 exposure days to other FIX products
* Patients currently treated on-demand with at least 6 bleeding episodes during the last 12 months or at least 3 bleeding episodes during the last 6 months, or patients currently on prophylaxis

Exclusion Criteria

* Known history of FIX inhibitors based on existing medical records, laboratory report reviews and patient and LAR interviews
* Current FIX inhibitors *0.6 BU (central laboratory)
* HIV positive with a viral load *400,000 copies/mL and/or CD4+ lymphocyte count *200/*L
* Congenital or acquired coagulation disorders other than haemophilia B
* Previous arterial thrombotic events (e.g. myocardial infarction and intracranial thrombosis) or previous deep venous thrombosis or pulmonary embolism (as defined by available medical records)
* Platelet count <50.000 platelets/*l at screening (local laboratory)
* ALT >3 times the upper limit of normal reference ranges at screening (central laboratory)
* Creatinine level *1.5 times above upper normal limit at screening (central laboratory)
* Immune modulating or chemotherapeutic medication

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Primary Endpoint: Incidence of inhibitory antibodies against FIX defined as<br /><br>titre *0.6 BU.</p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Key Secondary Endpoints:<br /><br>* Haemostatic effect of N9-GP when used for treatment of bleeding episodes,<br /><br>assessed as success/failure based on a four-point scale for haemostatic<br /><br>response (excellent, good, moderate and poor) by counting excellent and good as<br /><br>success and moderate and poor as failure<br /><br>* Number of bleeding episodes per patient during routine prophylaxis<br /><br>* FIX trough levels<br /><br>* Adverse Events (AEs) and Serious Adverse Events (SAEs)<br /><br>* Host Cell Proteins (HCP)-antibodies<br /><br>* General safety endpoints including laboratory parameters, physical<br /><br>examination and vital signs</p><br>