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 - Paradigm 2
- Conditions
- Haemophilia BMedDRA version: 13.1Level: PTClassification code 10016077Term: Factor IX deficiencySystem Organ Class: 10010331 - Congenital, familial and genetic disorders
- Registration Number
- EUCTR2010-023069-24-IT
- Lead Sponsor
- OVO NORDISK
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 72
1.Informed consent obtained before any trial-related activities. (Trial-related activities are any procedure that would not have been performed during normal management of the patient.) 2.Male patients with moderately severe or severe congenital haemophilia B with a FIX activity =2% according to medical records 3.History of at least 150 exposure days to other FIX products 4.Patients currently on prophylaxis or 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 5.Age 13-70 years (both inclusive) 6.Body Mass Index = 35 7.The patient and/or LAR is capable of assessing a bleeding episode, keeping a diary, capable of home treatment of bleeding episodes and otherwise capable of following the trial procedures
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
1.Known or suspected hypersensitivity to trial product or related products 2.Previous participation in this trial defined as withdrawal after administration of N9-GP 3.Receipt of any investigational drug 30 days prior to enrolment into the trial 4.Known history of FIX inhibitors based on existing medical records, laboratory report reviews and patient and LAR interviews 5.Current FIX inhibitors >0.6 BU (central laboratory) 6.HIV positive, defined by medical records with viral load >400,000 copies/mL and/or CD4+ lymphocyte count >200/?L. If the data is not available in medical records within last 6 months, CD4+ will be measured at the screening visit 7.Congenital or acquired coagulation disorders other than haemophilia B 8.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) 9.Platelet count <50.000 platelets/?l at screening (local laboratory) 10.ALT >3 times the upper limit of normal reference ranges at screening (central laboratory) 11.Creatinine level >1.5 times above upper normal limit at screening (central laboratory) 12.Immune modulating or chemotherapeutic medication 13.Any disease (liver, kidney, inflammatory and mental disorders included) or condition which, according to the Investigator’s judgement, could imply a potential hazard to the patient, interfere with trial participation, or interfere with trial outcome 0.6 BU (central laboratory)
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the immunogenicity of N9-GP;Secondary Objective: To evaluate clinical efficacy of haemostasis (treatment of bleeding episodes) of N9-GP To evaluate clinical efficacy of N9-GP in long term bleeding prophylaxis (number of bleeding episodes during prophylaxis) To evaluate the efficacy of NNC-0156-0000-0009 by the surrogate marker for efficacy, FIX activity To evaluate general safety of N9-GP To evaluate PK properties of N9-GP To evaluate Patient Reported Outcomes (PRO), including health-related and disease-specific quality of life and patient treatment satisfaction To evaluate the health economic impact of N9-GP treatment;Primary end point(s): Incidence of inhibitory antibodies against FIX defined as titre =0.6 B
- Secondary Outcome Measures
Name Time Method