E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Subjects with moderate to severe crohn's disease |
Soggetti con malattia di Chron attiva da moderata a grave |
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E.1.1.1 | Medical condition in easily understood language |
Subjects with moderate to severe crohn's disease |
Soggetti con malattia di Chron attiva da moderata a grave |
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E.1.1.2 | Therapeutic area | Body processes [G] - Immune system processes [G12] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 17.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10013099 |
E.1.2 | Term | Disease Crohns |
E.1.2 | System Organ Class | 100000004856 |
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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 |
To evaluate the long-term safety and tolerability of MT-1303 in subjects with moderate to severe active CD |
Valutare la sicurezza e la tollerabilità a lungo termine di MT-1303 in soggetti con MC attiva da moderata a grave |
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E.2.2 | Secondary objectives of the trial |
• To evaluate the long-term effects of MT-1303 on clinical outcomes in subjects with moderate to severe active CD • To explore the pharmacodynamic (PD) effects of MT-1303 in subjects with moderate to severe active CD.
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• Valutare gli effetti a lungo termine di MT-1303 sugli esiti clinici in soggetti con MC attiva da moderata a grave • Esplorare gli effetti farmacodinamici (pharmacodynamic, PD) di MT-1303 nei soggetti con MC attiva da moderata a grave
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. The subject showed a decrease in CDAI of at least 70 points from the baseline CDAI score (i.e., Visit 2) at Week 12 in the double-blind MT-1303-E13 study. 2. The subject completed the 14 week Treatment Period in the double-blind MT-1303-E13 study as per Protocol. 3. The subject is able to provide written informed consent and to comply with the requirements of the MT-1303-E14 Protocol during the study. 4. For subjects of reproductive potential, two methods of contraception must be used throughout the study and for 12 weeks after cessation of study medication. At least one of the methods of contraception must be a barrier method.
For detailed information, please refer to the Protocol. |
1. Il soggetto presentava un CDAI ridotto di almeno 70 punti rispetto al punteggio del CDAI al basale (ossia alla Visita 2) alla Settimana 12 nello studio MT-1303-E13 in doppio cieco; 2. Il soggetto ha completato il periodo di trattamento di 14 settimane nello studio MT-1303-E13 in doppio cieco secondo le indicazioni riportate nel protocollo; 3. Il soggetto è in grado di fornire un consenso informato scritto e di soddisfare i requisiti del protocollo MT-1303-E14; 4. I soggetti in età fertile dovranno adottare due metodi contraccettivi per l’intera durata dello studio e per le 12 settimane successive all’interruzione del farmaco dello studio. Almeno uno dei metodi contraccettivi deve essere un metodo barriera.
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E.4 | Principal exclusion criteria |
1. Permanent discontinuation of study medication prior to the EOT Visit in MT-1303-E13 2. Newly diagnosed diabetes mellitus during the double blind MT-1303-E13 study.
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1. Interruzione permanente del farmaco dello studio prima della Visita EOT dello studio MT-1303-E13 2. Diabete mellito di nuova diagnosi durante lo studio MT-1303-E13 in doppio cieco
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E.5 End points |
E.5.1 | Primary end point(s) |
Safety, assessed by: - Adverse events (AEs) - Vital signs (pulse rate, blood pressure [BP] and body temperature) - 12-lead ECG - Holter ECG monitoring - Routine safety laboratory assessments - Physical examination (including skin assessment) - OCT
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Sicurezza, valutata mediante: • EA • Segni vitali • ECG a 12 derivazioni • Monitoraggio ECG con Holter • Esami di laboratorio di routine sulla sicurezza • Esame obiettivo (compresa valutazione cutanea) • Tomografia ottica computerizzata (Optical coherence tomography, OCT)
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Various time points throughout the study - please refer to the detailed ‘Time and Events Schedule’ table in the protocol for full details. |
Diversi punti temporali durante lo studio. Per maggiori informazioni consultare il calendario del tempo e degli eventi nel protocollo. |
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E.5.2 | Secondary end point(s) |
Efficacy • Proportion of subjects who achieve a 70 point decrease from MT-1303-E13 baseline in Crohn’s disease activity index (CDAI) score (i.e., CDAI 70) at Protocol-scheduled visits • Proportion of subjects who achieve a 100-point decrease from MT-1303-E13 baseline in CDAI score (i.e., CDAI 100) at Protocol-scheduled visits • Proportion of subjects who achieve clinical remission (i.e., CDAI score of <150) at Protocol-scheduled visits • CDAI score and change from MT-1303-E13 baseline in CDAI score at Protocol scheduled visits • Proportion of subjects in corticosteroid-free remission at end of treatment (EOT)
Pharmacodynamics • Lymphocyte counts and lymphocyte subsets, their change from MT-1303-E13 baseline and percentage of MT-1303-E13 baseline at Protocol-scheduled visits • C-reactive protein (CRP) and faecal calprotectin value and their change from MT-1303-E13 baseline at Protocol-scheduled visits.
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Efficacia • Percentuale di soggetti che, alle visite programmate del protocollo, raggiungono una riduzione di 70 punti nel punteggio del CDAI (ossia CDAI 70) rispetto al basale di MT-1303-E13; • Percentuale di soggetti che, alle visite programmate del protocollo, raggiungono una riduzione di 100 punti nel punteggio del CDAI (ossia CDAI 100) rispetto al basale di MT-1303-E13; • Percentuale di soggetti che, alle visite programmate del protocollo, raggiungono una remissione clinica (ossia punteggio del CDAI <150); • Punteggio del CDAI e variazione del punteggio del CDAI rispetto al basale di MT-1303-E13 alle visite programmate del protocollo; • Percentuale di soggetti in remissione senza corticosteroidi alla EOT; Farmacodinamica • Conta dei linfociti e dei sottogruppi di linfociti, loro variazione rispetto al basale di MT-1303-E13 e percentuale del basale di MT-1303-E13 alle visite programmate del protocollo • Valori della proteina C reattiva (PCR) e della calprotectina nelle feci e loro variazione rispetto al basale di MT-1303-E13 alle visite programmate del protocollo.
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Various time points throughout the study - please refer to the detailed ‘Time and Events Schedule’ table in the protocol for full details. |
Diversi punti temporali durante lo studio. Per maggiori informazioni consultare il calendario del tempo e degli eventi nel protocollo. |
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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 | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
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 | 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.8.2.4 | 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 | 9 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 57 |
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 |
Czech Republic |
France |
Germany |
Hungary |
Israel |
Italy |
Japan |
Netherlands |
Poland |
Slovakia |
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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 |
Ultima visita dell'ultimo paziente |
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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 | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
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 | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |