Safety and Efficacy of NNC-0156-0000-0009 after Long-Term Exposure in Patients with Haemophilia B
- Conditions
- Haemophilia B is a recessive X-linked congenital bleeding disorder, caused by mutations in the coagulation factor nine (FIX) gene, located in the distal part on the long arm of the X-chromosome. Haemophilia B is caused by any of a variety of genetic anomalies distributed throughout the gene, with almost every family.MedDRA version: 14.1Level: LLTClassification code 10018939Term: Haemophilia B (Factor IX)System Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2010-023072-17-IT
- Lead Sponsor
- OVO NORDISK
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 75
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. Previous participation in NN7999-3747 and/or NN7999-3773 3. 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: 1
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 2
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1
1. Known or suspected hypersensitivity to the trial product or related products 2. Dosing of any investigational drug within the last 30 days prior to the present trial (excluding N9-GP) 3. Known history of FIX inhibitors based on existing medical records, laboratory report reviews and patient and LAR interviews 4. Current FIX inhibitors =0.6 BU (central laboratory) 5. Congenital or acquired coagulation disorders other than haemophilia B 6. 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) 7. 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
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 NNC-0156-0000-0009;Primary end point(s): Incidence of inhibitory antibodies against FIX defined as titre =0.6 BU;Timepoint(s) of evaluation of this end point: End of trial;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 N9-GP by the surrogate marker for efficacy, FIX activity • To evaluate general safety 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
- Secondary Outcome Measures
Name Time Method Secondary end point(s): Secondary Efficacy Endpoints • Haemostatic effect of N9-GP when used for treatment of bleeding episodes, assessed on a fourpoint scale for haemostatic response (excellent, good, moderate and poor) by counting excellent and good as success and moderate and poor as failure • Number of bleeding episodes per patient during routine prophylaxis • FIX trough levels • The number of injections of N9-GP required per bleeding episode • Amount of N9-GP required per bleeding episode (U/kg BW/bleeding episodes Secondary Safety Endpoints • Adverse Events (AEs) and Serious Adverse Events (SAEs) • Host Cell Proteins (HCP)-antibodies • General safety endpoints including laboratory parameters, physical examination and vital signs;Timepoint(s) of evaluation of this end point: End of trial