E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Haemophilia B |
Hemofilia B |
|
E.1.1.1 | Medical condition in easily understood language |
Bleeding disorder, inherited deficiency in clotting factor IX |
Desorden de la sangre, deficiencia en factor de coagulación IX |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 14.1 |
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 | 100000004850 |
|
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 NNC-0156-0000-0009. |
?Evaluar la inmunogenicidad de NNC-0156-0000-0009. |
|
E.2.2 | Secondary objectives of the trial |
? To evaluate clinical efficacy of haemostasis (treatment of bleeding episodes) of NNC-0156-0000-0009 ? To evaluate clinical efficacy of NNC-0156-0000-0009 in long term bleeding prophylaxis (number of bleeding episodes during prophylaxis) ? To evaluate efficacy of NNC-0156-0000-0009 by the surrogate marker for efficacy, FIX activity ? To evaluate general safety of NNC-0156-0000-0009 |
?Evaluar la eficacia clínica de la hemostasia (tratamiento de los episodios hemorrágicos) con NNC-0156-0000-0009 ?Evaluar la eficacia clínica de NNC-0156-0000-0009 en la profilaxis de las hemorragias a largo plazo (número de episodios hemorrágicos durante la profilaxis) ?Evaluar la eficacia de NNC-0156-0000-0009 mediante el marcador indirecto de la eficacia, la actividad del FIX ?Evaluar la seguridad general de NNC-0156-0000-0009 |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Previous participation in NN7999-3747 and/or NN7999-3773 |
?Participación previa en NN7999-3747 o NN7999-3773 |
|
E.4 | Principal 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 ? 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) ? 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 |
?Antecedentes conocidos de inhibidores del FIX basándose en las historias clínicas existentes, las revisiones de los informes de laboratorio y las entrevistas con los pacientes y los RL ?Inhibidores del FIX actuales ? 0,6 UB ?Trastornos de la coagulación congénitos o adquiridos distintos de la hemofilia B. ?Acontecimientos trombóticos arteriales anteriores (p. ej., infarto de miocardio y trombosis intracraneal) o trombosis venosa profunda o embolia pulmonar (según las historias clínicas disponibles). ? Cualquier enfermedad o trastorno (incluidos los hepáticos, renales, inflamatorios y desórdenes mentales) que, según el criterio del investigador, pueda entrañar un peligro para el paciente o interferir en la participación en el ensayo o en su resultado. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Incidence of inhibitory antibodies against FIX defined as titre ?0.6 BU |
?Incidencia de anticuerpos inhibidores contra el FIX, definiendo la inhibición como un título ? 0,6 UB |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
The efficacy endpoints will be evaluated based on all available information until the EOT visit. The safety endpoints will be evaluated based on all available information until the last visit. |
Los criterios de valoración de la eficacia se evaluaran basándose en toda la información disponible hasta la visita de FDT. Los criterios de valoración de la seguridad se evaluaran basándose en toda la información disponible hasta la última visita. |
|
E.5.2 | Secondary end point(s) |
? Haemostatic effect of NNC-0156-0000-0009 when used for treatment of bleeding episodes, assessed on a four-point 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 during routine prophylaxis ? FIX trough levels ? Adverse Events (AEs) and Serious Adverse Events (SAEs) ? General safety endpoints including laboratory parameters, physical examination and vital signs |
?Efecto hemostático de NNC-0156-0000-0009 cuando se utiliza para el tratamiento de los episodios hemorrágicos, evaluado en una escala de cuatro puntos para la respuesta hemostática (excelente, buena, moderada o escasa) y contando como éxitos las respuestas excelentes y buenas y como fracasos las respuestas moderadas o escasas. ?Número de episodios hemorrágicos durante la profilaxis sistemática ?Concentraciones mínimas de FIX ?Acontecimientos adversos (AA) y acontecimientos adversos graves (AAG) ?Criterios de valoración generales de la seguridad, como los parámetros analíticos, la exploración física y las constantes vitales |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
The efficacy endpoints will be evaluated based on all available information until the EOT visit. The safety endpoints will be evaluated based on all available information until the last visit. |
Los criterios de valoración de la eficacia se evaluaran basándose en toda la información disponible hasta la visita de FDT. Los criterios de valoración de la seguridad se evaluaran basándose en toda la información disponible hasta la última visita. |
|
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 | 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 | Yes |
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) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 1 |
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 |
European Union |
Canada |
Japan |
Malaysia |
Russian Federation |
South Africa |
Taiwan |
Thailand |
United States |
|
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 | 3 |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 4 |
E.8.9.2 | In all countries concerned by the trial months | 7 |
E.8.9.2 | In all countries concerned by the trial days | 0 |