E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Moderate to severe myasthenia gravis |
Miastenia Gravis de grado moderado o severo |
|
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 autoimmune que causa debilidad en los músculos; esta causada por problemas de comunicación entre nervios y músculos. |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Immune System Diseases [C20] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 19.1 |
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 |
|
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 |
Evaluate the clinical efficacy of UCB7665 as an chronic-intermittent treatment in subjects with generalized myasthenia gravis (MG) who are classified as moderate to severe |
Evaluar la eficacia clínica del UCB7665 como tratamiento a largo plazo intermitente en sujetos con Miastenia Gravis (MG) generalizada de carácter moderado o severo |
|
E.2.2 | Secondary objectives of the trial |
- Gather data for future study planning, whether for chronic-intermittent treatment or a longer therapy option by evaluating the general concept that UCB7665 has a clinical effect in patients with generalized myasthenia gravis (MG) - Evaluate the safety and tolerability of UCB7665 administered by subcutaneous (sc) infusion in subjects with MG - Assess the effect of UCB7665 as measured by total immunoglobulin G (IgG) concentrations in serum |
- Recoger datos para un futuro plan de estudio, tanto para el tratamiento a largo plazo intermitente como para opciones de tratamiento más prolongadas, mediante la evaluando del concepto general de que el UCB7665 es clínicamente eficaz en pacientes con MG generalizada. - Evaluar la seguridad y la tolerabilidad del UCB7665 administrado en infusión subcutanea (sc) a sujetos con MG - Evaluar el efecto del UCB7665 según la medición de la concentración sérica de IgG total |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- Subject has a well-documented diagnosis of myasthenia gravis (MG) at Visit 1 (Screening), based on subject history and supported by previous evaluations - Subject would currently be considered for treatment with immunological therapy such as intravenous immunoglobulin/plasma exchange (IVIG/PLEX) by the investigator - Subject has documented evidence in his/her medical history or at Screening of detectable autoantibodies (anti-acetylcholine receptor+ve (Anti-AChR+ve) or anti-muscle specific kinase+ve (Anti- MuSK+ve) - Female subjects must either be: postmenopausal, permanently sterilized or if childbearing potential applicable will use a highly effective method of birth control - Male subjects must be willing to use a method of contraception |
- Sujeto con diagnóstico bien documentado de miastenia gravis (MG) en la Visita 1 (Selección), de acuerdo a sus antecedentes y con respaldo por evaluaciones previas - Sujeto que puede estar actualmente considerado por el investigador para posible tratamiento inmunológico, como inmunoglobulina intravenosa/plasmaféresis (IVIG/PLEX) - Sujeto con evidencia documentada en su historia médica o en la Selección de autoanticuerpos detectables [positividad de anti-receptor de acetilcolina (anti-AChR positivo) o positividad de anti-cinasa músculo-específica (anti-MuSK positivo)] - Si se trata de una mujer: en posmenopausia, sometida a esterilización permanente o, si es potencialmente fértil, que va a utilizar un método anticonceptivo altamente eficaz - Si se trata de un varón: conformidad en utilizar un método anticonceptivo |
|
E.4 | Principal exclusion criteria |
- Subject has previously received treatment in this study or subject has previously been exposed to UCB7665 - Subject has participated in another study of an investigational medicinal product (IMP; or a medical device) within the previous 30 days of Screening or is currently participating in another study of an investigational medicinal product (IMP; or a medical device) - Subject has a known hypersensitivity to any components of the IMP - Subject has a history of hyperprolinemia, since L-proline is a constituent of the UCB7665 IMP - Subjects with MG only affecting the ocular muscles - Subjects with severe weakness affecting oropharyngeal or respiratory muscles, or who have myasthenic crisis at Screening or impending crisis - Subject has quantitative myasthenia gravis (QMG) score of <11 at Baseline - Subject has a serum total immunoglobulin G (IgG) level <= 6g/L at Screening - Absolute neutrophil count <1500 cells/mm^3 - Subject has any medical condition (acute or chronic illness) or psychiatric condition that, in the opinion of the investigator, could jeopardize or would compromise the subject’s ability to participate in this study - Subject has any laboratory abnormality that, in the opinion of the investigator, is clinically significant, has not resolved at randomization, and could jeopardize or would compromise the subject’s ability to participate in this study - Subject has received a live vaccination within 8 weeks prior to the Baseline Visit; or intends to have a live vaccination during the course of the study or within 7 weeks following the final dose of IMP - Subject has received any experimental biological agent within or outside of a clinical study in the past 3 months or within 5 half-lives prior to Baseline (whichever is longer) |
- Sujeto que ha recibido tratamiento previamente en este estudio o que ha recibido previamente UCB7665 - Sujeto que ha participado en otro estudio con un producto en fase de investigación (medicamento o producto sanitario) en el plazo de los 30 días anteriores a la Selección o que está participando actualmente en otro estudio con un producto en fase de investigación (medicamento o producto sanitario) - Sujeto con hipersensibilidad conocida a cualquier componente del medicamento en investigación - Sujeto con historia de hiperprolinemia (ya que la L-prolina es un componente del medicamento en investigación UCB7665) - Sujeto con MG que sólo afecta a musculatura ocular - Sujeto con debilidad severa que afecta a músculos orofaríngeos o respiratorios, o con crisis miasténicas en la Selección o con amenaza de crisis - Sujeto con una puntuación de la escala cuantitativa de miastenia gravis (QMG) <11 en el Basal - Sujeto con una concentración sérica de inmunoglobulina G (IgG) total < =6 g/L en la Selección - Recuento absoluto de neutrófilos <1.500 /mm^3 - Sujeto con cualquier proceso médico (agudo o crónico) o psiquiátrico que, en opinión del investigador, podría poner en riesgo o comprometer su capacidad de participar en el estudio - Sujeto con una anomalía de laboratorio que, en opinión del investigador, es clínicamente importante, no se ha resuelto en la aleatorización o podría poner en riesgo o comprometer su capacidad de participar en el estudio - Sujeto que ha recibido una vacuna con gérmenes vivos en el plazo de las 8 semanas previas a la Visita Basal; o programado para recibir una vacuna con gérmenes vivos durante el curso del estudio o en el plazo de las 7 semanas siguientes a la última dosis del medicamento en investigación - Sujeto que ha recibido cualquier agente biológico experimental, dentro o fuera de un estudio clínico, en los 3 últimos meses o en el plazo de 5 semividas del agente antes del momento basal (eligiéndose el más prolongado de estos plazos) |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Change from Baseline in Quantitative Myasthenia Gravis (QMG) score to Visit 9 |
Variación respecto al momento basal de la puntuación de la escala cuantitativa de MG (QMG) en la visita 9 |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Baseline and Visit 9 (Day 29) |
Basal y Visita 9 (Día 29) |
|
E.5.2 | Secondary end point(s) |
Change from Baseline in Myasthenia Gravis-Composite score to Visit 9 Change from Baseline in Myasthenia Gravis-Activities of Daily Living (MGADL) score to Visit 9 |
Variación respecto al momento basal de la puntuación de la escala compuesta de MG (MG Composite) en la visita 9 Variación respecto al momento basal del cuestionario de actividades cotidianas con MG (MGADL) en la visita 9 |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Baseline and Visit 9 (Day 29) |
Basal y Visita 9 (Día 29) |
|
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 | Yes |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | Yes |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
Tolerability |
Tolerabilidad |
|
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) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 17 |
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 |
Czech Republic |
Denmark |
Germany |
Spain |
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 subject last visit (LSLV) |
Ultima Visita del Ultimo Sujeto (LSLV) |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 1 |
E.8.9.1 | In the Member State concerned months | 3 |
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 | 6 |
E.8.9.2 | In all countries concerned by the trial days | 15 |