E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Focal Onset Seizures (FOS) |
Crisis Epilépticas Focales (CEF) |
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E.1.1.1 | Medical condition in easily understood language |
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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 | LLT |
E.1.2 | Classification code | 10016843 |
E.1.2 | Term | Focal seizures |
E.1.2 | System Organ Class | 100000004852 |
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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 |
The primary objective for this study is evaluate the long-term safety and tolerability of NBI-921352 administered for up to 107 weeks in subjects with FOS |
El objetivo primario del estudio es evaluar la seguridad y la tolerabilidad a largo plazo de NBI-921352 administrado durante un máximo de 107 semanas en sujetos con CEF |
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E.2.2 | Secondary objectives of the trial |
The secondary objective for this study is to investigate the effect of NBI-921352 on long-term seizure control in subjects with FOS. |
El objetivo secundario del estudio es • Investigar el efecto de NBI-921352 en el control a largo plazo de las crisis epilépticas en sujetos con CEF. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Provided informed consent. • Completed 11 weeks of treatment in Study NBI-921352-FOS2021. • Anticipated use of stable treatment with at least 1 but not more than 4 ASMs during the Blinded Dose Conversion Period and for the first week of the Open-Label Treatment Period. • Female subjects of childbearing potential must agree to use contraception consistently from Day 1 until the final study visit or 30 days after the last dose of study treatment, whichever is longer. • Male subjects must agree to use effective barrier contraception consistently from Day 1 until 30 days after the last dose of study treatment. • Be able to keep accurate seizure diaries • Willing and able to comply with all study procedures and restrictions. |
• Otorgó el consentimiento informado. • Ha completado 11 semanas de tratamiento en el estudio NBI-921352-FOS2021. • Uso previsto de un tratamiento estable con al menos 1 pero no más de 4 anticonvulsivos durante el periodo de conversión de dosis ciega y durante la primera semana del periodo de tratamiento abierto. • Las mujeres en edad fértil deben aceptar usar un método anticonceptivo de forma sistemática desde el día 1 hasta la visita de fin del estudio o 30 días después de la última dosis del tratamiento del estudio, lo que sea más largo. • Los varones deben aceptar usar un método anticonceptivo de barrera eficaz de forma sistemática desde el día 1 hasta 30 días después de la última dosis del tratamiento del estudio. • Ser capaz de mantener diarios de convulsiones de forma precisa. • Estar dispuesto a cumplir todos los procedimientos y las restricciones del estudio. |
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E.4 | Principal exclusion criteria |
Subjects will be excluded from the study if they meet any of the following criteria: • Pregnant or breastfeeding or planning to become pregnant during the study. • Have developed any other disorder for which the treatment takes priority over the treatment of FOS or is likely to interfere with study treatment or impair treatment compliance. • Anticipated that the subject will require treatment with at least 1 of the disallowed concomitant medications (Section 7.1.1) during the study. • Plan to use any investigational drug (other than the study treatment) during the study. • Any medical condition or personal circumstance that in the opinion of the investigator exposes the subject to unacceptable risk by participating in the study or prevents adherence to the protocol. • In the investigator’s opinion, the subject is not capable of adhering to the protocol requirements, including the requirement to travel to the study sites for study visits, or is unsuitable for any reason. |
Se excluirá del estudio a los participantes que cumplan cualquiera de los siguientes criterios: • Que esté embarazada, en periodo de lactancia o planeando quedarse embarazada durante el estudio. • Que presente cualquier otro trastorno cuyo tratamiento tenga prioridad sobre el tratamiento de FOS o que pueda interferir con el tratamiento del estudio o perjudicar el cumplimiento del mismo. • Que necesite tratamiento con al menos 1 de los medicamentos concomitantes no permitidos (apartado 7.1.1) durante el estudio. • Que tenga planificado usar cualquier fármaco en investigación (distinto del tratamiento del estudio) durante el estudio. • Que sufra de cualquier afección médica o circunstancia personal que, en opinión del investigador, le exponga a un riesgo inaceptable al participar en el estudio o que impida el cumplimiento del protocolo. • Que, en opinión del investigador, no sea capaz de cumplir los requisitos del protocolo, incluido el requisito de traslado a los centros del estudio para las visitas del estudio, o que no sea un participante apto por cualquier motivo. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Subject incidence of serious treatment-emergent adverse events (TEAEs), TEAEs leading to study treatment discontinuation, and fatal TEAEs |
•Incidencia de acontecimientos adversos graves surgidos durante el tratamiento (AAST), AAST que provoquen la interrupción del tratamiento del estudio y AAST mortales |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Throughout the study |
Durante todo el estudio |
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E.5.2 | Secondary end point(s) |
• Percentage change from baseline in 28-day FOS frequency during the treatment period • Treatment response defined as ≥50% reduction in 28-day FOS frequency during the treatment period compared with baseline |
• El cambio porcentual con respecto al inicio en la frecuencia de CEF a los 28 días durante el periodo de tratamiento • Respuesta al tratamiento definida como una reducción ≥50 % en la frecuencia de CEF a los 28 días durante el periodo de tratamiento en comparación con los valores iniciales |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Baseline, Treatment Period |
Visita basal, Periodo de tratamiento |
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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 | 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) | 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 | 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 | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 26 |
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 |
Australia |
Belgium |
Czechia |
Hungary |
Italy |
Spain |
United Kingdom |
France |
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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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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 | 3 |
E.8.9.1 | In the Member State concerned months | 1 |
E.8.9.1 | In the Member State concerned days | 12 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
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 | 12 |