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 del sangrado, deficiencia hereditaria en el factor IX de couagulación |
|
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 | 18.0 |
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 immunogenicity of N9-GP (nonacog beta pegol) |
Evaluar la inmunogeneicidad de N9-GP (nonacog beta pegol) |
|
E.2.2 | Secondary objectives of the trial |
- To evaluate safety of N9-GP (nonacog beta pegol) - To evaluate efficacy of N9-GP (nonacog beta pegol) o in long-term prophylaxis treatment o in the treatment of bleeding episodes o through the surrogate marker: FIX activity o through monitoring of number of doses and consumption of N9-GP |
?Evaluar la seguridad de N9-GP (nonacog beta pegol) ?Evaluar la eficacia de N9-GP (nonacog beta pegol) o en el tratamiento profiláctico a largo plazo o en el tratamiento de los episodios hemorrágicos o por medio del marcador indirecto: actividad del FIX o por medio del seguimiento del número de dosis y del consumo de N9-GP |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial - Male < 6 years of age at the time of signing informed consent - Patients with the diagnosis of haemophilia B (FIX activity level ? 2%) based on medical records or central laboratory results - Previously untreated or exposed to FIX containing products less than or equal to 3 exposure days (5 previous exposure days to blood components are acceptable) |
-Obtención del consentimiento informado antes de realizar cualquier actividad relacionada con el ensayo. Se consideran actividades relacionadas con el ensayo todos los procedimientos que se lleven a cabo como parte del ensayo, incluidas las actividades para determinar la idoneidad para el mismo. -Pacientes varones < 6 años de edad en el momento de la firma del consentimiento informado. -Pacientes con un diagnóstico de hemofilia B (nivel de actividad del FIX ? 2 %) basado en la historia clínica o los resultados del laboratorio central. -Pacientes no tratados previamente o con una exposición a productos con FIX inferior o igual a 3 días de exposición (se aceptan 5 días de exposición previas a hemoderivados). |
|
E.4 | Principal exclusion criteria |
- Any history of FIX inhibitors (defined by medical records) - Known or suspected hypersensitivity to trial product or related products - Previous participation in this trial. Participation is defined as first dose administered of trial product - Receipt of any investigational medicinal product within 30 days before screening - Congenital or acquired coagulation disorder other than haemophilia B - Any chronic disorder or severe disease which, in the opinion of the Investigator, might jeopardise patient?s safety or compliance with the protocol - Patient?s parent(s)/LAR(s) mental incapacity, unwillingness to cooperate, or a language barrier precluding adequate understanding and cooperation |
?Antecedentes de uso de inhibidores del FIX (según la historia clínica). ?Hipersensibilidad conocida o presunta al producto del ensayo o productos relacionados. ?Participación previa en este ensayo. La participación se define como la administración de la primera dosis del producto del ensayo. ?Recepción de cualquier producto en investigación en los 30 días previos a la selección ?Trastornos de la coagulación congénitos o adquiridos distintos de la hemofilia B. ?Cualquier trastorno crónico o enfermedad importante que, en opinión del investigador, pueda poner en peligro la seguridad del paciente o el cumplimiento del protocolo. ?Incapacidad mental, falta de cooperación o barrera idiomática que impida una correcta comprensión y colaboración de los padres o el tutor legal del paciente. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Incidence of inhibitory antibodies against FIX |
Incidencia de anticuerpos inhibidores contra el FIX |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
All objectives/endpoints will be evaluated when the first 20 PUPs have reached at least 50 EDs, when the first 40 PUPs have reached 100 EDs, and at end of trial. End of trial will be up to 4 years after the patient has reached 100 EDs. |
Todos los objetivos/criterios de valoración se evaluarán cuando los primeros 20 PNTP PSTP hayan alcanzado al menos 50 DE, cuando los primeros 40 PNTP PSTP hayan alcanzado 100 DE y al final del ensayo. El final del ensayo tendrá lugar 4 años como máximo después de que el paciente haya alcanzado 100 DE |
|
E.5.2 | Secondary end point(s) |
- Number and frequency of adverse events, serious adverse events, and Medical Events of Special interest - Number of breakthrough bleeding episodes during prophylaxis (annualised bleeding rate) -Haemostatic effect by 4-point haemostatic response scale ("excellent", "good", "moderate" and "poor") |
-Número y frecuencia de acontecimientos adversos, acontecimientos adversos graves y acontecimientos médicos graves de interés especial. -Número de episodios hemorrágicos intercurrentes durante la profilaxis (tasa anualizada de hemorragias). -Efecto hemostático mediante la escala de respuesta de 4 puntos ("excelente", "buena", "leve" o "pobre") |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
All objectives/endpoints will be evaluated when the first 20 PUPs have reached at least 50 EDs, when the first 40 PUPs have reached 100 EDs, and at end of trial. End of trial will be up to 4 years after the patient has reached 100 EDs. |
Todos los objetivos/criterios de valoración se evaluarán cuando los primeros 20 PNTP PSTP hayan alcanzado al menos 50 DE, cuando los primeros 40 PNTP PSTP hayan alcanzado 100 DE y al final del ensayo. El final del ensayo tendrá lugar 4 años como máximo después de que el paciente haya alcanzado 100 DE |
|
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 | Yes |
E.6.13.1 | Other scope of the trial description |
Immunogenicity |
Inmunogeneicidad |
|
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) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
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 | 10 |
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 | No |
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 |
Australia |
Canada |
European Union |
Israel |
Japan |
Malaysia |
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 | 6 |
E.8.9.1 | In the Member State concerned months | 3 |
E.8.9.1 | In the Member State concerned days | 28 |
E.8.9.2 | In all countries concerned by the trial years | 6 |
E.8.9.2 | In all countries concerned by the trial months | 3 |
E.8.9.2 | In all countries concerned by the trial days | 28 |