E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Generalized Myasthenia Gravis |
miastenia grave generalizada (MGg) |
|
E.1.1.1 | Medical condition in easily understood language |
Myasthenia Gravis |
miastenia grave |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 16.1 |
E.1.2 | Level | HLT |
E.1.2 | Classification code | 10071942 |
E.1.2 | Term | Myasthenia gravis and related conditions |
E.1.2 | System Organ Class | 100000004859 |
|
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 |
Assess the efficacy of eculizumab as compared with placebo in the treatment of refractory gMG based on the improvement in the MG-specific Activities of Daily Living profile (MG-ADL). |
Evaluar la eficacia de eculizumab en comparación con un placebo para el tratamiento de la MGg refractaria a partir de la mejora del perfil de actividades de la vida diaria específicas para la MG (MG-ADL). |
|
E.2.2 | Secondary objectives of the trial |
- Safety and tolerability of eculizumab as compared with placebo in gMG subjects - Efficacy of eculizumab compared with placebo - Describe the pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab - Characterize the effect of eculizumab as compared with placebo on Quality of Life measures |
- seguridad y la tolerabilidad generales de eculizumab en comparación con un placebo en pacientes con MGg - eficacia de eculizumab en comparación con un placebo - describir la farmacocinética (FC) y la farmacodinámica (FD) de eculizumab en pacientes con MGg - caracterizar el efecto de eculizumab frente al placebo según las mediciones de la calidad de vida (QoL) |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Male or female subjects ?18 years old - Diagnosis of MG - MGFA Clinical Classification Class II to IV at screening - MG-ADL total score must be ?6 at screening |
- Edad de los pacientes ? 18 años, hombres o mujeres - Diagnóstico de MG - Clase II a IV de la clasificación clínica de la MGFA en la selección - La puntuación total de las MG-ADL deberá ser ? 6 en la selección |
|
E.4 | Principal exclusion criteria |
- History of thymoma or other neoplasms of thymus - History of thymectomy within 12 months prior to screening - Weakness only affecting ocular or peri-ocular muscles (MGFA Class I) - MG crisis at screening (MGFA Class V) - Pregnancy or lactation - Unresolved meningococcal infection |
- Antecedentes de timoma u otras neoplasias del timo. - Antecedentes de timectomía en el periodo de 12 meses antes de la selección - Debilidad que únicamente afecte a los músculos oculares o perioculares (clase I de la MGFA) - Crisis de MG en la selección (clase V de la MGFA) - Embarazo o lactancia - Infección meningocócica no resuelta |
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E.5 End points |
E.5.1 | Primary end point(s) |
- change from baseline in the MG-ADL total score for eculizumab as compared with placebo |
- variación de las MG-ADL en la semana 26 con respecto al inicio |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
- Change from baseline in QMG total score - Proportion of subjects with at least a 3-point reduction in the MG-ADL total score and with no rescue therapy - Proportion of subjects with at least a 5 point reduction in the QMG total score - Change from baseline in the Myasthenia Gravis Composite (MGC) scale total score - Change from baseline in MG-QOL-15 |
- variación de QMG con respecto al inicio - Proporción de pacientes que hayan experimentado, como mínimo, una reducción de 3 puntos en la puntuación total de la escala MG-ADL entre el inicio y la semana 26 y que no hayan necesitado tratamiento de rescate - Proporción de pacientes que hayan experimentado, como mínimo, una reducción de 5 puntos en la puntuación total de la escala QMG entre el inicio y la semana 26 y que no hayan necesitado tratamiento de rescate - Variación de la puntuación de la escala MGC registrada en la semana 26 con respecto a la inicial - Variación en la puntuación de la escala MG-QOL15 registrada en la semana 26 con respecto al inicio |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
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 | 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 | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
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 | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
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 | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 44 |
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 |
Belgium |
Canada |
Denmark |
France |
Greece |
Ireland |
Italy |
Japan |
Austria |
Netherlands |
Norway |
Sweden |
Argentina |
Australia |
Brazil |
Finland |
Germany |
Spain |
Switzerland |
Turkey |
United Kingdom |
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
|
Last visit of the last patient |
|
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 | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 3 |