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 | LLT |
E.1.2 | Classification code | 10018939 |
E.1.2 | Term | Haemophilia B (Factor IX) |
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 haemostatic effect of N9-GP during surgical procedures in patients with haemophilia B |
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E.2.2 | Secondary objectives of the trial |
• To evaluate the general safety, including immunogenicity of N9-GP, when used for prevention and treatment of bleeding throughout the surgical period • To evaluate the haemostatic effect of N9-GP during the post-operative period |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1.Written informed consent from the patient or the child’s parent/legal guardian 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.Age 13-70 years (both inclusive) 5.Body Mass Index (BMI) ≤ 35 6.Scheduled major surgery (refer to section 5.2.1 for a definition) 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 products or related products 2.Receipt of any investigational drug except N9-GP, within the last 30 days prior to present trial 3.Known history of FIX inhibitors based on available medical record, laboratory report reviews and patient and patient’s LAR interviews 4.Current FIX inhibitors ≥0.6 BU (central laboratory) 5.HIV positive, defined by medical records with viral load ≥400,000 copies/mL and/or CD4+ lymphocyte count ≤200/μL. If the data is not available in medical records within last 6 months, CD4+ will be measured at the screening visit. 6.Congenital or acquired coagulation disorders other than haemophilia B 7.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) 8.Platelet count < 50,000 platelets/μL at screening 9.ALT > 3 times the upper limit of normal reference ranges at screening 10.Creatinine level ≥ 1.5 times above upper normal limit at screening 11.Immune modulating or chemotherapeutic medication. 12.Any disease or condition (liver, kidney and inflammatory disorders included) which, according to the Investigator’s judgement, could imply a potential hazard to the patient, interfere with the trial participation or trial outcome |
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E.5 End points |
E.5.1 | Primary end point(s) |
• Haemostatic effect during surgery evaluated by the four-point response scale, assessed by the Investigator/Surgeon at the day of surgery - Four-point response scale: Excellent, good, moderate, poor |
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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 | No |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
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 | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
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) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
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 | 7 |
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 | 0 |
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 | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |