E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Focal Onset Seizures (FOS) |
Crisi convulsive a esordio focale (FOS) |
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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 |
L’obiettivo primario di questo studio è valutare la sicurezza e la tollerabilità a lungo termine di NBI-921352 somministrato per un massimo di 107 settimane in soggetti con FOS. |
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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. |
L’obiettivo secondario di questo studio è studiare l’effetto di NBI921352 sul controllo delle crisi convulsive a lungo termine nei soggetti con FOS. |
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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. |
• Fornitura del consenso informato. • Completamento di 11 settimane di trattamento nello Studio NBI-921352-FOS2021. • Uso previsto di un trattamento stabile con almeno 1, ma non più di 4, farmaci anticonvulsivanti (ASM) durante il periodo di conversione della dose in cieco e per la prima settimana del periodo di trattamento in aperto. • Per i soggetti di sesso femminile in età fertile: accettare di utilizzare un metodo contraccettivo in maniera costante dal Giorno 1 fino alla visita finale dello studio o 30 giorni dopo l’ultima dose del trattamento dello studio, a seconda di quale periodo sia più lungo. • Per i soggetti di sesso maschile: accettare di utilizzare un metodo contraccettivo di barriera efficace in maniera costante a partire dal Giorno 1 fino a 30 giorni dopo l’ultima dose del trattamento dello studio. • Capacità di tenere diari accurati delle crisi convulsive. • Soggetti disposti a osservare tutte le procedure e le restrizioni dello studio e in grado di farlo. |
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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 XML File Identifier: VA7v3zp9wIoEv5V2VRvImtFBvzU= Page 10/25 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. |
I soggetti saranno esclusi dallo studio se soddisfano uno qualsiasi dei seguenti criteri: • Soggetti femminili in gravidanza o allattamento o che stanno pianificando una gravidanza durante lo studio. • Sviluppo di qualsiasi altro disturbo il cui trattamento abbia priorità rispetto al trattamento delle FOS oppure possa interferire con il trattamento dello studio o compromettere l’aderenza al trattamento. • Previsione che il soggetto richieda un trattamento con almeno 1 dei farmaci concomitanti non consentiti (Sezione 7.1.1) durante lo studio. • Utilizzo pianificato di qualsiasi farmaco sperimentale (diverso dal trattamento dello studio) durante lo studio. • Qualsiasi condizione medica o circostanza personale che, a giudizio dello sperimentatore, esponga il soggetto a un rischio inaccettabile durante la partecipazione allo studio o impedisca la sua aderenza al protocollo. • Giudizio dello sperimentatore secondo cui il soggetto non è in grado di aderire ai requisiti del protocollo, incluso il requisito di recarsi presso i centri dello studio per le visite dello studio, o non è idoneo per qualsiasi 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 |
Incidenza nei soggetti di eventi avversi seri emergenti dal trattamento (TEAE), TEAE che portano all’interruzione del trattamento dello studio e TEAE fatali |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Throughout the study |
Per l’intera durata dello studio |
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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 |
• Variazione percentuale dal basale nella frequenza di FOS a 28 giorni durante il periodo di trattamento • Risposta al trattamento definita come una riduzione =50% nella frequenza di FOS a 28 giorni durante il periodo di trattamento rispetto al basale |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Baseline, Treatment Period |
Basale, periodo di trattamento |
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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 | No |
E.6.6 | Pharmacokinetic | Yes |
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) | 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 | 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 | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 4 |
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 | Information not present in EudraCT |
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 |
France |
Italy |
Spain |
United Kingdom |
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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 | 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 | 4 |
E.8.9.2 | In all countries concerned by the trial months | 2 |
E.8.9.2 | In all countries concerned by the trial days | 20 |