E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 13.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10016077 |
E.1.2 | Term | Factor IX deficiency |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To evaluate the immunogenicity of N9-GP |
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E.2.2 | Secondary objectives of the trial |
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 |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
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 |
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E.4 | Principal exclusion criteria |
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) |
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E.5 End points |
E.5.1 | Primary end point(s) |
Incidence of inhibitory antibodies against FIX defined as titre ≥0.6 BU |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | Yes |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | Yes |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.3.1 | Comparator description |
- same IMP used at different dosage |
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E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 17 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | No |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 4 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 4 |
E.8.9.2 | In all countries concerned by the trial days | 0 |