E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Relapsing-remitting multiple sclerosis (RRMS) |
Sclerosi Multipla recidivante Remittente |
|
E.1.1.1 | Medical condition in easily understood language |
Multiple Sclerosis |
Sclerosi multipla |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Nervous System Diseases [C10] |
MedDRA Classification |
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 long-term safety and tolerability of MT-1303 in subjects
with RRMS |
Valutare la sicurezza e la tollerabilità a lungo termine di MT- 1303 nei soggetti con SMRR. |
|
E.2.2 | Secondary objectives of the trial |
• To evaluate the long-term effects of MT-1303 on magnetic resonance
imaging (MRI) parameters, clinical outcomes and health-related quality
of life in subjects with RRMS
• To evaluate the pharmacodynamics of MT-1303 in subjects with RRMS |
• Valutare gli effetti a lungo termine di MT-1303 sui parametri della risonanza magnetica per immagini (RMI), sugli esiti clinici e sulla qualità della vita correlata allo stato di salute nei soggetti con SMRR.
• Valutare la farmacodinamica di MT-1303 nei soggetti con SMRR
|
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Completion of the 24-week Treatment Period in MT-1303-E04 as per
protocol |
Completamento del periodo di trattamento della durata di 24 settimane nello studio MT- 1303-E04 come da protocollo |
|
E.4 | Principal exclusion criteria |
1. Permanent discontinuation of study medication prior to the End of
Treatment (EOT) Visit in MT-1303-E04
2. Newly diagnosed diabetes mellitus during MT-1303-E04 |
1. Sospensione permanente del farmaco dello studio prima della visita di fine trattamento (End of Treatment, EOT) nello studio MT-1303-E04.
2. Diagnosi recente di diabete mellito effettuata durante lo studio MT-1303-E04. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Safety Assessments
• Adverse events (AEs)
• Vital signs
• 12-lead electrocardiogram (ECG)
• 3-lead Holter ECG monitoring
• Routine safety laboratory assessments
• Physical examination
• Optical coherence tomography (OCT) |
Valutazioni di sicurezza
1. Eventi avversi (EA)
2. Segni vitali
3. Elettrocardiogramma (ECG) a 12 derivazioni
4. Monitoraggio ECG a 3 derivazioni secondo Holter
5. Valutazioni di laboratorio standard sulla sicurezza
6. Esame obiettivo
7. Tomografia a coerenza ottica (Optical Coherence Tomography, OCT) |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
Various timepoints throughout the study - please refer to the detailed
Time and Events Schedule in the protocol for full details |
Vari momenti durante lo studio. Si prega di consultare la scheda Ore ed Eventi per maggiori dettagli. |
|
E.5.2 | Secondary end point(s) |
Clinical Efficacy Endpoints:
• Annualised Relapse Rate (ARR)
• Time to first confirmed relapse
• Proportion of subjects who remain relapse-free at End of Treatment
(EOT)
• Change from baseline in total Expanded Disability Status Scale (EDSS)
score at EOT
• Change from baseline in total Multiple Sclerosis Functional Composite
(MSFC) score at EOT
MRI Endpoints:
• Number of MRI Gd-enhanced T1-weighted lesions
• Number and volume of new or enlarged T2-weighted lesions
• Change and percent change in brain volume at EOT
• Magnetisation Transfer Ratio (MTR) related endpoints will be explored.
Details to be specified in the Statistical Analysis Plan (SAP) (selected
centres only)
Pharmacodynamic Endpoints:
• Lymphocyte counts
• Lymphocyte subsets (selected centres only)
Subject-reported Endpoints:
• Change from baseline in MSQOL-54 at EOT. |
Endpoint clinici di efficacia
1. Tasso di recidive annualizzato (Annualised Relapse Rate, ARR)
2. Tempo alla prima recidiva confermata
3. Percentuale di soggetti che non manifestano recidive
4. Variazione rispetto al basale del punteggio EDSS (Expanded Disability Status Scale) totale e del punteggio totale sulla scala Multiple Sclerosis Functional Composite (MSFC) alla fine del trattamento.
Endpoint valutati con la RMI
1. Numero di lesioni T1-pesate evidenziate con gadolinio
2. Numero e volume di lesioni T2-pesate nuove o ingranditesi
3. Variazione e percentuale di variazione del volume cerebrale alla fine del trattamento.
Endpoint del rapporto del trasferimento di magnetizzazione
(Magnetisation Transfer Ratio, MTR) indicati in dettaglio nel Piano di analisi statistico (Statistical Analysis Plan, SAP) (solo presso i centri selezionati) Endpoint farmacodinamici
1. Conte linfocitarie
2. Sottogruppi linfocitari (solo presso i centri selezionati)
Endpoint riportati dal soggetto:
1. Variazione rispetto al basale nel questionario sulla qualità della vita correlata alla sclerosi multipla (MSQOL-54) alla fine del trattamento. |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
Various timepoints throughout the study - please refer to the detailed
Time and Events Schedule in the protocol for full details |
Vari momenti durante lo studio. Si prega di consultare la scheda Ore ed Eventi per maggiori dettagli |
|
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 | 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 | Yes |
E.6.13.1 | Other scope of the trial description |
Tolerability |
Tollerabilità |
|
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 | Yes |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
Questo è uno studio a 2 gruppi paralelli.La prima parte è in doppio cieco; la seconda parte è in ape |
This study is a parallel group 2 part study. Part 1 is double-blind; Part 2 is open label |
|
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 | 3 |
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 | 5 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 63 |
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 |
Bosnia and Herzegovina |
Bulgaria |
Canada |
Croatia |
Czech Republic |
Finland |
Germany |
Hungary |
Italy |
Latvia |
Lithuania |
Poland |
Russian Federation |
Serbia |
Spain |
Sweden |
Switzerland |
Ukraine |
United Kingdom |
|
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
|
|
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 | 6 |
E.8.9.1 | In the Member State concerned days | |
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 | 2 |