E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Generalized Myasthenia Gravis |
MIASTENIA GRAVIS GENERALIZADA |
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E.1.1.1 | Medical condition in easily understood language |
Myasthenia Gravis |
Miastenia Gravis |
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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 | 18.0 |
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 |
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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 long-term safety of eculizumab in subjects with refractory gMG. |
Evaluar la seguridad a largo plazo de eculizumab en sujetos con MGg resistente al tratamiento. |
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E.2.2 | Secondary objectives of the trial |
- To evaluate the long-term efficacy of eculizumab in subjects with refractory gMG as measured by the improvement or maintenance of the MG-specific Activities of Daily Living profile (MG-ADL). - To evaluate the long-term efficacy of eculizumab by additional efficacy measures including: Quantitative Myasthenia Gravis (QMG) score, Myasthenia Gravis Composite (MGC) score, and Improvement or maintenance in primary symptoms that are most clinically meaningful to the subjects. - To characterize the effect of eculizumab on quality of life measures. - To describe the pharmacokinetics (PK) and pharmacodynamics (PD) of eculizumab in subjects with gMG. |
?Evaluar la eficacia a largo plazo de eculizumab en sujetos con MGg resistente al tratamiento medida por la mejoría o el mantenimiento del perfil de actividades de la vida diaria específico para la MG (MG-specific Activities of Daily Living profile, MG-ADL). ?Evaluar la eficacia a largo plazo de eculizumab a través de otras mediciones de la eficacia, por ejemplo: puntuación de la escala cuantitativa de la miastenia gravis (Quantitative Myasthenia Gravis, QMG); puntuación compuesta de la miastenia gravis (Myasthenia Gravis Composite, MGC) y mejoría o mantenimiento de los principales síntomas que, desde el punto de vista clínico, resultan más significativos para los sujetos. ?Caracterizar el efecto de eculizumab en las mediciones de la calidad de vida. ?Describir la farmacocinética (FC) y la farmacodinámica (FD) de eculizumab en sujetos con MGg. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Subject has completed the ECU-MG-301 trial. 2. Subject has given written informed consent. 3. Subject is willing and able to comply with the protocol requirements for the duration of the trial. 4. Female subjects of child-bearing potential must have a negative pregnancy test (serum human chorionic gonadotropin [HCG]). All subjects must practice an effective, reliable and medically approved contraceptive regimen during the trial and for up to 5 months following discontinuation of treatment. |
1.El sujeto ha completado el ensayo ECU-MG-301. 2.El sujeto ha otorgado su consentimiento informado por escrito. 3.El sujeto está dispuesto y es capaz de cumplir con los requisitos del protocolo mientras dure el ensayo. 4.Las participantes en edad fértil deberán obtener resultados negativos en la prueba de embarazo (gonadotropina coriónica humana [GCH] en suero). Todos los sujetos deben utilizar un tratamiento anticonceptivo eficaz, fiable y médicamente aprobado durante el ensayo y hasta 5 meses después de interrumpir el tratamiento. |
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E.4 | Principal exclusion criteria |
1. Subjects who withdrew from the ECU-MG-301 trial as a result of an AE related to trial drug. 2. Female subjects who are pregnant, breastfeeding or intend to conceive during the course of the trial. 3. Any medical condition or circumstances that, in the opinion of the Investigator, might interfere with the subject?s participation in the trial, pose any added risk for the subject, or confound the assessment of the subjects. |
1.Los sujetos que se retiren del ensayo ECU-MG-301 como consecuencia de un AA relacionado con el fármaco del ensayo. 2.Participantes que estén embarazadas, en periodo de lactancia o que tengan intención de quedarse embarazadas durante el transcurso del ensayo. 3.Cualquier patología o circunstancia que, a juicio del investigador, pudiera interferir en la participación del sujeto en el ensayo, suponer algún riesgo adicional para el sujeto o generar confusión en la evaluación de los sujetos. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Change from baseline in the MG-ADL total score. |
Variación de la puntuación total en la escala MG-ADL con respecto al inicio |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
From week 1 to 182 |
De la semana 1 a la 182 |
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E.5.2 | Secondary end point(s) |
1. Change from baseline in QMG total score. 2. Proportion of subjects with at least a 3-point reduction in the MG-ADL total score from baseline and with no rescue therapy. 3. Proportion of subjects with at least a 5-point reduction in the QMG total score from baseline and with no rescue therapy. 4. Change from baseline in the MGC scale total score. 5. Change from baseline in MG-QOL-15. |
1.Variación de la puntuación total de QMG con respecto al inicio. 2.Proporción de sujetos que hayan experimentado, como mínimo, una reducción de 3 puntos en la puntuación total de la escala MG-ADL con respecto al inicio y que no hayan necesitado tratamiento de rescate. 3.Proporción de sujetos que hayan experimentado, como mínimo, una reducción de 5 puntos en la puntuación total de la escala QMG con respecto al inicio y que no hayan necesitado tratamiento de rescate. 4.Variación en la puntuación total de la escala MGC con respecto al inicio. 5.Variación en la puntuación del cuestionario MG-QOL-15 con respecto al inicio. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
From week 1 to 182 |
De la semana 1 a la 182 |
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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 | 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 | Yes |
E.8.1.7.1 | Other trial design description |
inducción con enmascaramiento seguida de la fase abierta de mantenimiento |
Induction blinded phase followed by an open label maintenance phase |
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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 | 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 |
Argentina |
Australia |
Austria |
Belgium |
Brazil |
Canada |
Denmark |
Finland |
France |
Germany |
Greece |
Ireland |
Italy |
Japan |
Netherlands |
Norway |
Spain |
Sweden |
Switzerland |
Turkey |
United Kingdom |
United States |
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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
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 4 |
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 | 4 |
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 |