E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Duchenne Muscular Dystrophy (DMD) |
Distrofia muscular de Duchenne |
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E.1.1.1 | Medical condition in easily understood language |
DMD is a genetic disease characterised by rapidly progressive muscle weakness and wasting which leads to severe disability. |
La DMD es una enfermedad genética caracterizada por debilidad muscular y pérdida de peso rápidamente progresiva que conduce a una discapacidad severa |
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E.1.1.2 | Therapeutic area | Diseases [C] - Musculoskeletal Diseases [C05] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10013801 |
E.1.2 | Term | Duchenne muscular dystrophy |
E.1.2 | System Organ Class | 10010331 - Congenital, familial and genetic disorders |
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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 assess the long-term safety of idebenone in DMD patients who completed the SIDEROS study. |
Evaluar la seguridad a largo plazo de idebenona en pacientes con DMD que hayan completado el estudio SIDEROS. |
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E.2.2 | Secondary objectives of the trial |
To describe the long-term evolution of respiratory function in idebenone-treated DMD patients who completed the SIDEROS study, classified by background factors including, but not limited to age, DMD history (e.g. time of loss of ambulation, mutation type), type of steroid regimen and study treatment assignment in the SIDEROS study. |
Describir la evolución a largo plazo de la función respiratoria de los pacientes con DMD tratados con idebenona que hayan completado el estudio SIDEROS, clasificada por factores de referencia incluidos, entre otros, la edad, los antecedentes de DMD (p. ej., tiempo transcurrido hasta la pérdida de ambulación o tipo de mutación), el tipo de tratamiento con esteroides y el tratamiento asignado en el estudio SIDEROS. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Completion of the SIDEROS study at Visit 8/ Week 78. Signed and dated Informed Consent Form. |
Criterios de inclusión 1.Haber completado el estudio SIDEROS en la visita 8/semana 78 2.Formulario de consentimiento informado firmado y fechado para SIDEROS-E |
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E.4 | Principal exclusion criteria |
1. Patients who discontinued SIDEROS study prematurely (i.e. did not attend all visits from V1 to V8). 2. Safety, tolerability or other issues arising during the course of the SIDEROS study which in the opinion of the Investigator may put the patient at significant risk or may interfere significantly with the patient’s participation in the SIDEROS-E study. 3.Use of any investigational drug other than the study medication.
Enrolment in SIDEROS-E of siblings of randomized SIDEROS patients is allowed if they meet all the inclusion and none of the exclusion criteria above. |
1.Los pacientes que hayan interrumpido prematuramente el estudio SIDEROS (es decir, no han asistido a todas las visitas desde la V1 a la V8) 2.Problemas relacionados con la seguridad, la tolerabilidad u otros motivos que se presenten durante el transcurso del estudio SIDEROS que, en la opinión del investigador, puedan poner en grave riesgo al paciente en el estudio SIDEROS-E o interferir de modo significativo en la participación del paciente en el estudio SIDEROS-E 3.3.Uso de algún fármaco en fase de investigación que no sea la medicación del estudio |
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary: Standard safety assessments, including number of premature discontinuations of study treatment due to adverse events, incidence and severity of adverse events, actual values and changes from baseline in safety laboratory parameters, vital signs and electrocardiogram (ECG). |
•Evaluaciones de seguridad estándar, entre las que se incluyen número de interrupciones prematuras del tratamiento del estudio debido a acontecimientos adversos, incidencia y gravedad de los acontecimientos adversos, valores y cambios reales respecto a la visita inicial en los parámetros de seguridad de laboratorio, constante vitales y electrocardiograma (ECG). |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Electrocardiogram: baseline Adverse events, safety laboratory parameters, vital signs: 26 weeks , 52 weeks, 78 weeks, 82 weeks |
Electrocardiograma: basal Eventos adversos, parámetros de laboratorio de seguridad, signos vitales: 26 semanas, 52 semanas, 78 semanas, 82 semanas |
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E.5.2 | Secondary end point(s) |
Secondary: Change from Baseline in Forced Vital Capacity (FVC) as percent of predicted (FVC%p), Peak Expiratory Flow (PEF) as percent of predicted (PEF%p) and Forced Expiratory Volume in 1 second (FEV1) as percent of predicted (FEV1%p). |
•Cambio desde la visita inicial en la capacidad vital forzada (FVC) en el porcentaje previsto (FVC%p), en el flujo espiratorio máximo (PEF) en el porcentaje previsto (PEF%p) y en el volumen espiratorio forzado en un segundo (FEV1) en el porcentaje previsto (FEV1%p). |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Forced Vital Capacity (FVC), Peak Expiratory Flow (PEF)Forced Expiratory Volume in 1 second (FEV1) : baseline, 26 weeks , 52 weeks, 78 weeks |
Capacidad vital forzada (FVC), flujo espiratorio máximo (PEF) Volumen espiratorio forzado en 1 segundo (FEV1): valor inicial, 26 semanas, 52 semanas, 78 semanas |
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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 | 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.8.3 |
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 | 36 |
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 |
Austria |
Belgium |
France |
Germany |
Israel |
Italy |
Netherlands |
Spain |
Sweden |
Switzerland |
United Kingdom |
United States |
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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
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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 | |
E.8.9.1 | In the Member State concerned months | 19 |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial months | 19 |