E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Amyotrophic Lateral Sclerosis (ALS) |
Sclerosi Laterale Amiotrofica (SLA) |
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E.1.1.1 | Medical condition in easily understood language |
A disease that affects nerve cells in the brain and the spinal cord. |
Una malattia che colpisce le cellule nervose del cervello e del midollo spinale |
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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 | 10002026 |
E.1.2 | Term | Amyotrophic lateral sclerosis |
E.1.2 | System Organ Class | 10029205 - Nervous system disorders |
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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 |
To evaluate the long-term safety of oral edaravone at a dose of 105 once daily for 10 days out of a 14-day period, followed by a 14-day drug-free period for 96 weeks of treatment or until the drug is commercially available in that country. |
Valutare la sicurezza a lungo termine di edaravone orale alla dose di 105 mg somministrata una volta al giorno per 10 giorni di un periodo di 14 giorni, seguito da un periodo di 14 giorni senza l’assunzione del farmaco, per 96 settimane di trattamento o finché il farmaco non sarà disponibile sul mercato del paese interessato |
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E.2.2 | Secondary objectives of the trial |
not applicable |
non applicabile |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Subjects must provide signed and dated informed consent form (ICF) to participate in the study. Subjects must be able (in the judgement of the Investigator) to understand the nature of the study and all risks involved with participation in the study. 2. Subjects must be willing to cooperate and comply with all protocol restrictions and requirements. 3. Subjects who successfully completed Study MT-1186-A01. |
1. I soggetti devono fornire il modulo di consenso informato (ICF) firmato e datato per partecipare allo studio. I soggetti devono essere in grado (secondo il parare dello Sperimentatore) di comprendere la natura dello studio e tutti i rischi che comporta la partecipazione allo stesso. 2. I soggetti devono essere disposti a collaborare e attenersi alle restrizioni e ai requisiti del protocollo. 3. Soggetti che hanno completato con esito positivo lo Studio MT-1186-A01. |
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E.4 | Principal exclusion criteria |
1. Subjects of childbearing potential unwilling to use a highly effective method of contraception from Visit 1 until 3 months after the last dose of study medication. Refer to Appendix 2 of the Protocol for additional contraceptive information. 2. Subjects who have a significant risk of suicide. Subjects with any suicidal behavior or suicidal ideation of type 4 (active suicidal ideation with some intent to act, without a pecific plan) or type 5 (active suicidal ideation with specific plan and intent) based on the Columbia-Suicide Severity Rating Scale (C-SSRS) at Visit 1. 3. Subjects who are not eligible to continue in the study, as judged by the Investigator. 4. Subjects who are unable to take their medications orally or through a PEG/RIG tube. |
1. Soggetti in età fertile non disposti a utilizzare un metodo contraccettivo altamente efficace a partire dalla Visita 1 e fino a 3 mesi dopo l’assunzione dell'ultima dose di farmaco in studio. Per ulteriori informazioni riguardo alla contraccezione, fare riferimento all’Appendice 2 del Protocollo. 2. Soggetti che presentano un rischio suicidario significativo. Soggetti che presentano qualsiasi comportamento suicidario o ideazione suicidaria di tipo 4 (ideazione suicidaria attiva con intenzione di agire, senza un piano specifico) o di tipo 5 (ideazione suicidaria attiva con un piano e un’intenzione specifici) in base alla scala di valutazione della gravità della suicidalità della Columbia University (C-SSRS) alla Visita 1. 3. Soggetti che, secondo il parere dello Sperimentatore, non sono idonei a proseguire lo studio. 4. Soggetti che non sono in grado di assumere il proprio farmaco per via orale o mediante sonda PEG/RIG. |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary safety endpoints are to evaluate the safety and tolerability of oral edaravone and include the following safety assessments: - Adverse events (AEs), adverse drug reactions (ADRs), and treatmentemergent adverse events ([TEAEs] eg, grade, incidence and severity); - Physical examination; - Body weight; - 12-lead electrocardiogram (ECG) parameters; - Vital signs (heart rate, systolic and diastolic blood pressure, and axillary, oral, or tympanic body temperature); - Laboratory safety assessments (eg, hematology, chemistry, and urinalysis); - Columbia–Suicide Severity Rating Scale (C-SSRS) |
Gli endpoint di sicurezza primari sono valutare la sicurezza e la tollerabilità di edaravone orale e includono le seguenti valutazioni di sicurezza: - Eventi avversi (AE), reazioni avverse al farmaco (ADR) ed eventi avversi emergenti dal trattamento ([TEAE] ad es. grado, incidenza e gravità); - Esame obiettivo; - Peso corporeo; - Parametri di elettrocardiogramma a 12 derivazioni (ECG); - Parametri vitali (frequenza cardiaca, pressione sanguigna sistolica e diastolica, temperatura ascellare, orale o timpanica); - Valutazioni di sicurezza di laboratorio (ad es. ematologia, chimica e analisi delle urine); - Scala di valutazione della gravità della suicidalità della Columbia University (C SSRS) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Visit 1-9 Visit 1, 2, 3, 5, 7, 9 Visit 1, 2, 3, 5, 7, 9 Visit 1, 5, 9 Visit 1, 2, 3, 5, 7, 9 Visit 1, 3, 5, 7, 9 Visit 1, 3, 5, 7, 9 |
Visita 1-9 Visita 1, 2, 3, 5, 7, 9 Visita 1, 2, 3, 5, 7, 9 Visita 1, 5, 9 Visita 1, 2, 3, 5, 7, 9 Visita 1, 3, 5, 7, 9 Visita 1, 3, 5, 7, 9 |
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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 | 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 | 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 | 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 | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 8 |
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 |
Canada |
Japan |
United States |
France |
Germany |
Italy |
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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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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 | 1 |
E.8.9.1 | In the Member State concerned days | 18 |
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 | 11 |
E.8.9.2 | In all countries concerned by the trial days | 0 |