E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Treatment of celiac disease |
Trattamento della celiachia |
|
E.1.1.1 | Medical condition in easily understood language |
Inflammation in the small intestine |
Infiammazione nell'intestino tenue |
|
E.1.1.2 | Therapeutic area | Diseases [C] - Digestive System Diseases [C06] |
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 assess 3 different doses and 2 different dosing schedules of ZED1227 capsules for efficacy in improvement of the duodenal mucosal morphology and improvement of celiac disease symptoms assessed by Celiac Disease Symptom Diary (CDSD) in celiac disease subjects experiencing symptoms and having mucosal damage despite gluten-free diet. |
Valutare 3 diverse dosi e 2 diversi schemi di dosaggio di ZED1227 capsule per l'efficacia nel miglioramento della morfologia della mucosa duodenale e nel miglioramento dei sintomi della celiachia valutati in base al Diario dei sintomi della malattia celiaca (CDSD) in soggetti celiaci che manifestano sintomi e hanno danni alla mucosa nonostante una dieta senza glutine. |
|
E.2.2 | Secondary objectives of the trial |
To assess efficacy of ZED1227 capsules for
• Improvement and changes of duodenal mucosal morphology as measured by morphometry (VH:CrD), • Improvement and changes of celiac disease symptoms assessed by CDSD, • Changes of inflammatory cell subsets in duodenal biopsies, • Changes in serum markers of celiac inflammation, • Safety and tolerability in terms of adverse events and laboratory parameters, • Subjects’ quality of life. |
Valutare l'efficacia delle capsule ZED1227 nel:
• Miglioramento e modificazioni della morfologia della mucosa duodenale misurata mediante morfometria (VH:CrD), • Miglioramento e modifica dei sintomi della celiachia valutati mediante CDSD, • Cambiamenti di sottoinsiemi di cellule infiammatorie nelle biopsie duodenali, • Cambiamenti nei marcatori sierici dell'infiammazione celiaca, • Sicurezza e tollerabilità in termini di eventi avversi e parametri di laboratorio, • Qualità della vita dei soggetti. |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
•Documented initial biopsy proven diagnosis of celiac disease at least 12 months prior to V0 •Adherence to a gluten-free diet (GFD) for at least 12 months prior to V0 •Human leukocyte antigen DQ (HLA-DQ) typing compatible with celiac disease •At least one moderate or severe gastrointestinal symptom (i.e., diarrhoea, abdominal pain, bloating or nausea) during the last 4 weeks prior to Baseline Visit •Negative diagnosis of Helicobacter pylori infection
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•Diagnosi comprovata di celiachia documentata da biopsia iniziale almeno 12 mesi prima di V0 •Adesione a una dieta priva di glutine (GFD) per almeno 12 mesi prima della V0 •Tipizzazione dell'antigene leucocitario umano DQ (HLA-DQ) compatibile con la celiachia •Almeno un sintomo gastrointestinale moderato o grave (ad es. diarrea, dolore addominale, gonfiore o nausea) durante le ultime 4 settimane prima della visita di riferimento •Diagnosi negativa di infezione da Helicobacter pylori |
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E.4 | Principal exclusion criteria |
•Selective immunoglobulin A deficiency •Subjects diagnosed to have confirmed refractory celiac disease type I or II •Severe complications of celiac disease •Any concomitant diseases of the intestinal tract in addition to celiac disease •Evidence of relevant systemic disease •Suspicion of acute infection with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2) or confirmed diagnosis of acute coronavirus disease 2019 (COVID-19)
|
•Deficit selettivo di immunoglobuline A •Soggetti con diagnosi di celiachia refrattaria confermata di tipo I o II •Gravi complicazioni della celiachia •Eventuali malattie concomitanti del tratto intestinale oltre alla celiachia •Evidenza di malattia sistemica rilevante •Sospetto di infezione acuta con sindrome respiratoria acuta grave coronavirus 2 (SARS-CoV-2) o diagnosi confermata di malattia acuta da coronavirus 2019 (COVID-19) |
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E.5 End points |
E.5.1 | Primary end point(s) |
Multi-component endpoint: -improvement of intestinal mucosal morphology -improvement in Non-Stool GI Specific Symptom Score OR as an improvement in Diarrhoea Severity Score
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Endpoint multicomponente: -miglioramento della morfologia della mucosa intestinale -miglioramento del punteggio relativo ai sintomi specifici del tratto gastrointestinale non fecale OPPURE miglioramento del punteggio di gravità della diarrea |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
12 weeks, from Baseline Visit B1 (week 5) to Visit 7 (week 17) |
12 settimane, dalla visita di riferimento B1 (settimana 5) alla visita 7 (settimana 17) |
|
E.5.2 | Secondary end point(s) |
• Improvement of intestinal mucosal morphology • Changes in intestinal mucosal morphology • Changes in Patient Reported Outcomes (PRO) • Change in health-related quality of life (EQ-5D-5L) • Change in serological markers |
• Miglioramento della morfologia della mucosa intestinale • Cambiamenti nella morfologia della mucosa intestinale • Modifiche dei risultati riportati dai pazienti (PRO) • Cambiamento nella qualità della vita correlata alla salute (EQ-5D-5L) • Modifica dei marcatori sierologici |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
12 weeks, from Baseline Visit B1 (week 5) to Visit 7 (week 17) |
12 settimane, dalla visita di riferimento B1 (settimana 5) alla visita 7 (settimana 17) |
|
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 | Yes |
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 |
Patient-reported outcome (=PRO) |
Esito riportato dal paziente |
|
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 | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | Yes |
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 | 9 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 40 |
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 |
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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 | 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 |