E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Generalized Myasthenia Gravis |
Miastenia gravis generalizada |
|
E.1.1.1 | Medical condition in easily understood language |
Myasthenia gravis is an autoimmune disease that causes weakness in your muscles; it is caused by a communication problem between nerves and muscles. |
La miastenia gravis es una enfermedad autoinmunitaria que causa debilidad en los músculos; Es causada por un problema de comunicación entre los nervios y los músculos. |
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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 | 21.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10028417 |
E.1.2 | Term | Myasthenia gravis |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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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 evaluate the ability of study participants with generalized Myasthenia Gravis (gMG) to successfully self-administer rozanolixizumab after training in the self-administration technique using the syringe driver and manual push methods |
Evaluar la capacidad de los participantes en el estudio con gMG para autoadministrarse con éxito el rozanolixizumab, tras su formación en la técnica de autoadministración, utilizando los métodos de syringe driver y manual push |
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E.2.2 | Secondary objectives of the trial |
To evaluate the safety of subcutaneous (SC) self-administration |
Evaluar la seguridad de la autoadministración SC del rozanolixizumab |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Study participant must have a documented diagnosis of generalized Myasthenia Gravis (gMG) - Study participant is willing to perform and capable of performing home self-administration - Study participant is considered for additional rozanolixizumab treatment with the posology proposed in this study - Body weight >=35 kg - Study participants may be male or female |
- Los participantes en el estudio deben tener un diagnóstico documentado de Miastenia gravis (gMG) - El participante del estudio está dispuesto a realizar y es capaz de realizar la autoadministración domiciliaria - Se considera que el participante del estudio recibe rozanolixizumab adicional Tratamiento con la posología propuesta en este estudio - Peso corporal >=35 kg - Los participantes del estudio pueden ser hombres o mujeres |
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E.4 | Principal exclusion criteria |
- Study participant has a known hypersensitivity to any components of the study medication or other anti-neonatal Fc receptor (FcRn) medications - Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current or history of nontuberculous mycobacterial infection (NTMBI) - Study participant has a clinically relevant active infection or a history of serious infection (resulting in hospitalization or requiring IV antibiotic treatment) within 6 weeks before the Baseline Visit - The study participant previously participated in any rozanolixizumab MG study and met any mandatory withdrawal or mandatory study drug discontinuation criteria - Study participant has received a live vaccination within 4 weeks before starting treatment, or a Bacillus Calmette Guérin (BCG) vaccine within 1 year before starting treatment; or intends to have a live vaccination during the course of the study or within 8 weeks following the last dose of rozanolixizumab - Study participant with severe (defined as Grade 3 on the Myasthenia Gravis Activities of Daily Living (MG-ADL) scale) weakness affecting oropharyngeal or respiratory muscles, or who has myasthenic crisis or impending crisis |
- El participante del estudio tiene una hipersensibilidad conocida a cualquiera de los componentes del medicamento del estudio u otros medicamentos anti-receptor Fc neonatal (FcRn). - Participante del estudio con una infección de tuberculosis (TB) conocida, con alto riesgo de contraer una infección de TB o una infección de tuberculosis latente (LTBI), o una infección por micobacterias no tuberculosas actual o anterior (NTMBI) - El participante del estudio tiene una infección activa clínicamente relevante o antecedentes de infección grave (que resultó en hospitalización o requirió tratamiento con antibióticos por vía intravenosa) dentro de las 6 semanas anteriores a la visita inicial - El participante del estudio participó previamente en cualquier estudio de rozanolixizumab MG y cumplió con los criterios obligatorios de retiro o interrupción obligatoria del fármaco del estudio. - El participante del estudio ha recibido una vacuna viva dentro de las 4 semanas antes de comenzar el tratamiento, o una vacuna de Bacillus Calmette Guérin (BCG) dentro de 1 año antes de comenzar el tratamiento; o tiene la intención de recibir una vacuna viva durante el curso del estudio o dentro de las 8 semanas posteriores a la última dosis de rozanolixizumab - Participante del estudio con debilidad grave (definida como grado 3 en la escala de actividades de la vida diaria (MG-ADL) de miastenia gravis) que afecta los músculos orofaríngeos o respiratorios, o que tiene una crisis miasténica o una crisis inminente |
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E.5 End points |
E.5.1 | Primary end point(s) |
1. Successful self-administration of rozanolixizumab (with correct use of syringe driver and manual push, respectively) during the Self-administration Period at Visit 13 2. Successful self-administration of rozanolixizumab (with correct use of syringe driver and manual push, respecively) during the Self-administration Period at Visit 19 |
1.Autoadministración con éxito de rozanolixizumab (con uso correcto del syringe driver y del manual push, respectivamente) durante los Períodos de Autoadministración, en la Visita 13. 2. Autoadministración con éxito de rozanolixizumab (con uso correcto del syringe driver y del manual push, respectivamente) durante los Períodos de Autoadministración, en la Visita 19. |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
1. Visit 13 (Week 12; last dose of Self-administration Period 1) 2. Visit 19 (Week 18; last dose of Self-administration Period 2) |
1. Visita 13 (Semana 12; última dosis Período de Autoadministración 1) 2. la Visita 19 (Semana 18; última dosis del Período de Autoadministración 2) |
|
E.5.2 | Secondary end point(s) |
1. Occurrence of Treatment-Emergent Adverse Events (TEAEs) after syringe driver or manual push self-administration from Visit 2 up to the End of Study Visit 2. Occurrence of local site reactions up to 24 hours after each administration during the Training Period and Self-administration Periods 3. Occurrence of medication errors associated with adverse reactions during the 2 Self-administration Periods of the study |
1. Presentación de acontecimientos adversos surgidos durante el tratamiento (treatment-emergent adverse events [TEAEs]) tras la autoadministración mediante syringe driver o manual push desde la Visita 2 hasta la Visita de Fin del Estudio 2. Presentación de reacciones locales en la zona de administración en las 24 horas siguientes a cada administración durante el Periodo de Formación y los Periodos de Autoadministración.
3. Producción de errores de medicación que cursen con reacciones adversas durante los 2 Periodos de Autoadministración del estudio. |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. From Visit 2 (Week 1) up to the End of Study Visit (Visit 21 (Week 26)) 2. Up to 24 hours after each administration during the Training Period (Baseline to Visit 7 (Week 6) and Self-administration Periods (Visit 8 (Week 7) to Visit 19 (Week 18)) 3. During the Self-administration Periods (Visit 8 (Week 7) to Visit 19 (Week 18)) |
1. Desde la Visita 2 (Semana 1) hasta la Visita de Fin de Estudio (Visita 21 (Semana 26)) 2. Hasta 24 horas después de cada administración durante el Período de Formación (Línea base hasta la Visita 7 (Semana 6) y Períodos de Autoadministración (Visita 8 (Semana 7) hasta la Visita 19 (Semana 18)) 3. Durante los Períodos de Autoadministración (Visita 8 (Semana 7) a Visita 19 (Semana 18)) |
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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 | 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 | Yes |
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 | Information not present in EudraCT |
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 | 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 | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 12 |
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 |
Japan |
United States |
Poland |
Spain |
Germany |
Italy |
Georgia |
United Kingdom |
Serbia |
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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
|
LVLS |
LVLS/ 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 | |
E.8.9.1 | In the Member State concerned months | 7 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 1 |
E.8.9.2 | In all countries concerned by the trial days | 20 |