E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Multiple Sclerosis |
Esclerosis Múltiple |
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E.1.1.1 | Medical condition in easily understood language |
Multiple Sclerosis |
Esclerosis Múltiple |
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E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
To describe efficacy, tolerability and convenience of teriflunomide treatment through the evaluation of PROs |
Describir la eficacia, tolerabilidad y comodidad del tratamiento con teriflunomida mediante la evaluación de PROs |
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E.2.2 | Secondary objectives of the trial |
- To describe disease progression using PRO - To describe clinical outcomes (ie treated relapses) in teriflunomide treated patients - To describe the change in cognition in teriflunomide treated patients - To describe safety of teriflunomide in patients treated (based on adverse event reporting) - To describe adherence and persistence to teriflunomide treatment - To describe quality of life, activity and leisure over the period of teriflunomide treatment - To compare patient determined disease steps (PDDS) (1) and expanded disability status scale (EDSS) (2) in assessing MS disease progression |
? Describir la progresión de la enfermedad utilizando PROs ? Describir los resultados clínicos (es decir, recidivas tratadas) en los pacientes tratados con teriflunomida ? Describir el cambio en la cognición en los pacientes tratados con teriflunomida ? Describir la seguridad de teriflunomida en los pacientes tratados (basándose en la notificación de acontecimientos adversos) ? Describir la adherencia y persistencia al tratamiento con teriflunomida ? Describir la calidad de vida, actividad y ocio a lo largo del período del tratamiento con teriflunomida ? Comparar las etapas de la enfermedad determinadas por el paciente (patient determined disease steps [PDDS] y la escala ampliada del estado de discapacidad (expanded disability status scale [EDSS]) a la hora de evaluar la progresión de la enfermedad de EM |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Patients with a relapsing form of MS (RMS) - Having signed written informed consent |
? Pacientes con una forma recidivante de EM (EMR), de acuerdo con el etiquetado local ? Haber firmado el consentimiento informado por escrito |
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E.4 | Principal exclusion criteria |
- 18 years of age - Current or history of receiving teriflunomide - Previous treatment with leflunomide within 6 months prior to baseline - Patients with preexisting acute or chronic liver disease, or those with serum alanine aminotransferase (ALT) greater than 2 times the upper limit of normal (ULN) - Known history of active tuberculosis (TB) or latent TB infection, either diagnosed by standard medical practice and guidelines (including skin or blood test, as appropriate) or according to local labeling - Known history of severe immunodeficiency, AIDS, bone marrow disease, acute or severe active infections - Women who are pregnant or breast-feeding - Female patients with a positive pregnancy test at screening or women of child-bearing potential who do not agree to use reliable contraception throughout the course of the study - Male patients: unwilling to use reliable contraception during the course of the study or according to local labeling |
Inferior a 18 años Estar recibiendo actualmente o haber recibido teriflunomida Tratamiento previo con leflunomida en los 6 meses previos a la visita basal Pacientes con enfermedad hepática crónica o aguda preexistente, o aquellos con alanina aminotransferasa sérica (ALT) mayor de 2 veces el límite superior de la normalidad (LSN) Antecedentes conocidos de infección por tuberculosis (TB) activa o TB latente, diagnosticada de acuerdo con las guías y la práctica médica estándar (incluyendo pruebas cutáneas o de sangre, según corresponda) o de acuerdo con el etiquetado local Antecedentes conocidos de inmunodeficiencia severa, SIDA, enfermedad de médula ósea, infecciones activas severas o agudas Mujeres embarazadas o en período de lactancia Mujeres con una prueba de embarazo positiva en el momento de la selección o mujeres en edad fértil que no acepten utilizar un método anticonceptivo fiable durante el transcurso del estudio Pacientes varones que no estén dispuestos a utilizar un método anticonceptivo fiable durante el transcurso del estudio o de acuerdo con el etiquetado local |
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E.5 End points |
E.5.1 | Primary end point(s) |
Assessment at 48 weeks (W48/EOT) of global satisfaction with teriflunomide treatment, measured by the Treatment Satisfaction Questionnaire for Medicine (TSQM) version 1.4 score |
Evaluación a las 48 semanas (S48/FDT) de la satisfacción global con el tratamiento con teriflunomida, medida mediante la puntuación del Cuestionario de Satisfacción con el Tratamiento de Medicamentos (Treatment Satisfaction Questionnaire for Medicine [TSQM]) versión 1.4 |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
1 - Change in TSQM version 1.4 from baseline to W4 and to W48/EOT of teriflunomide treatment in patients switching from another DMT 2 - Change in TSQM version 1.4 from W4 to W48/EOT in naïve patients 3 - Disease progression: change from baseline to W48/EOT of teriflunomide treatment, measured by the Patient Determined Disease Steps (PDDS) scale 4 - Disease progression: change from baseline to W24 and to W48/EOT of teriflunomide treatment, measured by the Multiple Sclerosis Performance Scale (MSPS) score (4, 5) 5 - Clinical outcomes: treated relapse, time to first treated relapse 6 - Change in cognition: change from baseline to W48/EOT of teriflunomide treatment as measured by the Symbol Digit Modalities Test (SDMT) score 7 - Occurrence of adverse events (AEs) based on AE reporting at each visit 8 - Adherence (ie, compliance) and persistence (ie, duration of exposure) to teriflunomide treatment over 48 weeks 9 - Quality of life: change from baseline to W48/EOT of teriflunomide treatment, measured by the Multiple Sclerosis International Quality of Life (MusiQoL) score (6) and Stern leisure activity scale (7). 10 - EDSS score at baseline and at W48/EOT |
1. Cambio en el TSQM versión 1.4 desde la visita basal hasta la S4 y hasta la S48/FDT del tratamiento con teriflunomida en pacientes que cambian desde otra TME 2. Cambio en el TSQM versión 1.4 desde la S4 hasta la S48/FDT en pacientes sin tratamiento previo 3. Progresión de la enfermedad: cambio desde la visita basal hasta la S48/FDT del tratamiento con teriflunomida, medido mediante la escala de Etapas de la Enfermedad Determinadas por el Paciente (PDDS) 4. Progresión de la enfermedad: cambio desde la visita basal hasta la S24 y hasta la S48/FDT del tratamiento con teriflunomida, medido mediante la puntuación de la Escala de Rendimiento de la Esclerosis Múltiple (Multiple Sclerosis Performance Scale [MSPS]) 5. Resultados clínicos: recidiva tratada, tiempo hasta la primera recidiva tratada 6. Cambio en la cognición: cambio desde la visita basal hasta la S48/FDT del tratamiento con teriflunomida medido mediante la puntuación del Test de las Modalidades de Símbolos y Dígitos (Symbol Digit Modalities Test [SDMT]) 7. Aparición de acontecimientos adversos (AA) basada en la notificación de AA en cada visita 8. Adherencia (i.e., cumplimiento) y persistencia (i.e., duración de la exposición) al tratamiento con teriflunomida a lo largo de 48 semanas 9. Calidad de vida: cambio desde la visita basal hasta la S48/FDT del tratamiento con teriflunomida, medido mediante la puntuación de la Calidad de Vida Internacional en Esclerosis Múltiple (Multiple Sclerosis International Quality of Life [MusiQoL]) y la escala de actividades de ocio de Stern (7). 10. Puntuación de la Escala Ampliada del Estado de Discapacidad (Expanded Disability Status Scale [EDSS]) en la visita basal y en la S48/FDT |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1 - Baseline to W4 and W4 to W48 2 - W4 to W48 3 - Baseline to W48 4 - Baseline to W24 and W24 to W48 5 - Over 48 weeks 6 - Baseline to W48 7/8 - Over 48 weeks 9 - Baseline to W48 10 - At baseline and at W48 |
1. Desde la visita basal hasta la S4 y hasta la S48 2. Desde S4 y hasta la S48 3. Desde la visita basal hasta la S48 4. Desde la visita basal hasta la S24 y desde S24 hasta la S48 5. Durante las 48 semanas 6. Desde la visita basal hasta la S48 7/8 Durante las 48 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 | Yes |
E.6.13.1 | Other scope of the trial description |
convenience and adherence of teriflunomide treatment |
la comodidad y el cumplimiento del tratamiento teriflunomida |
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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) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
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.8.2.4 | 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 | 9 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 119 |
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 |
Canada |
Chile |
Switzerland |
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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Last Subject Last Visist (LSLV) |
Última visita del último paciente |
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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 | 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 |