E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Haemophilia A |
Hemofilia A |
|
E.1.1.1 | Medical condition in easily understood language |
Bleeding disorder, inherited deficiency in clotting factor VIII |
Trastorno hemorrágico, deficiencia hereditaria del factor VIII de coagulación |
|
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 | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10018938 |
E.1.2 | Term | Haemophilia A (Factor VIII) |
E.1.2 | System Organ Class | 100000011915 |
|
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 assess the efficacy of concizumab administered s.c. once daily in preventing bleeding episodes in patients with severe haemophilia A without inhibitors |
Evaluar la eficacia de concizumab administrado s.c. una vez al día para prevenir los episodios hemorrágicos en pacientes con hemofilia A grave sin inhibidores |
|
E.2.2 | Secondary objectives of the trial |
- To assess the longer-term efficacy of concizumab in patients with severe haemophilia A without inhibitors - To assess the safety of concizumab in patients with severe haemophilia A without inhibitors - To assess the immunogenicity of concizumab in patients with severe haemophilia A without inhibitors |
-Evaluar la eficacia a más largo plazo de concizumab en pacientes con hemofilia A grave sin inhibidores. -Evaluar la seguridad de concizumab en pacientes con hemofilia A grave sin inhibidores. -Evaluar la inmunogenicidad de concizumab en pacientes con hemofilia A grave sin inhibidores. |
|
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 the suitability for the trial - Male patients aged 18 years or older at the time of signing informed consent, diagnosed with severe haemophilia A (FVIII activity below 1%), based on medical records or results at screening |
-Obtención del consentimiento informado antes de realizar ninguna de las actividades relacionadas con el ensayo. Se consideran actividades relacionadas con el ensayo todos los procedimientos realizados como parte del mismo, incluidas las actividades para determinar la idoneidad para participar en el ensayo. -Pacientes varones, de 18 años o más de edad en el momento de firmar el consentimiento informado, con diagnóstico de hemofilia A grave (actividad del FVIII menor del 1%), basándose en la historia clínica o los resultados obtenidos en el período de selección. |
|
E.4 | Principal exclusion criteria |
- Known or suspected hypersensitivity to trial product(s) or related products - Known inherited or acquired bleeding disorder other than haemophilia A - Presence of inhibitors (neutralising antibodies) to Factor VIII (equal to or above 0.6 Bethesda Units) at screening measured by the Nijmegen method |
-Hipersensibilidad conocida o sospechada a los productos del ensayo o a otros productos relacionados. -Trastorno hemorrágico hereditario o adquirido conocido distinto de la hemofilia A. -Presencia de inhibidores (anticuerpos neutralizantes) del factor VIII (igual o mayor de 0,6 unidades Bethesda) en la selección, medidos con el método de Nijmegen. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
The number of bleeding episodes |
El número de episodios hemorrágicos |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
During at least 24 weeks from treatment onset |
Durante 24 semanas, como mínimo, desde el comienzo del tratamiento. |
|
E.5.2 | Secondary end point(s) |
1. The number of spontaneous bleeding episodes 2. The number of treatment emergent adverse events (TEAEs) |
-Número de episodios hemorrágicos espontáneos -Número de acontecimientos adversos de aparición durante el tratamiento (TEAE) |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
1.+ 2. During at least 24 weeks from treatment onset
All endpoints referring to the time frame of at least 24 weeks will be evaluated in the main part of the trial, defined to end when the last patient has completed a minimum of 24 weeks of treatment with trial product (or have withdrawn). In addition, number of bleeding episodes during 76 weeks of treatment with prophylactic concizumab will be analysed. |
1.+ 2. Durante 24 semanas, como mínimo, desde el comienzo del tratamiento
Todos los criterios de valoración que hacen referencia al plazo de 24 semanas, como mínimo, se evaluarán en la parte principal del ensayo, cuyo final se ha definido cuando el último paciente haya completado un mínimo de 24 semanas de tratamiento con el producto del ensayo (o se haya retirado). Además, se analizará el número de episodios hemorrágicos durante 76 semanas de tratamiento en profilaxis con concizumab. |
|
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 | Yes |
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 |
Inmunogenicidad |
|
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
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 | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 13 |
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 |
European Union |
Japan |
Thailand |
Turkey |
Ukraine |
United States |
|
E.8.7 | Trial has a data monitoring committee | Yes |
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 | 5 |
E.8.9.1 | In the Member State concerned days | 15 |
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 | 5 |
E.8.9.2 | In all countries concerned by the trial days | 15 |