E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Active eosinophilic esophagitis and maintenance of remission in eosinophilic esophagitis |
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E.1.1.1 | Medical condition in easily understood language |
Allergic inflammatory disease of the gullet in its active and in its chronic symptom-free phase |
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E.1.1.2 | Therapeutic area | Diseases [C] - Digestive System Diseases [C06] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10064220 |
E.1.2 | Term | Eosinophilic esophagitis |
E.1.2 | System Organ Class | 100000004856 |
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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 |
Double-blind phase:
To prove superior efficacy of budesonide oral suspension compared to placebo in children and adolescents with eosinophilic esophagitis (EoE)
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E.2.2 | Secondary objectives of the trial |
Double-blind phase:
To further assess efficacy of budesonide oral suspension in children and adolescents with eosinophilic esophagitis (EoE)
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Inclusion criteria for DB-treatment phase:
• Signed informed consent
• Male or female patients, ≥2 to <18 years of age
• Confirmed clinico-pathological diagnosis of EoE according to established diagnostic criteria
• Clinically and histologically active EoE
• Indication for treatment with a steroid
• Negative pregnancy test in female patients of childbearing potential
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E.4 | Principal exclusion criteria |
Exclusion criteria for DB treatment phase:
• Erosive gastroesophageal reflux disease (GERD)
• Achalasia, scleroderma esophagus, or systemic sclerosis
• Other clinically evident causes than EoE for esophageal eosinophilia
• Any concomitant esophageal disease and relevant gastro-intestinal disease (celiac disease, inflammatory bowel disease, symptomatic Helicobacter pylori associated gastritis or any planned Helicobacter pylori eradication, oropharyngeal or esophageal bacterial, viral, or untreated or inadequately treated fungal infection [candida esophagitis])
• Any known relevant infectious diseases (e.g., AIDS defining disease, active tuberculosis, hepatitis B, or hepatitis C)
• Diabetes mellitus
• If careful medical monitoring is not ensured: cardiovascular disease, hypertension, osteoporosis, active peptic ulcer disease, glaucoma, cataract, or infection
• History of cancer in the last five years
• History of esophageal surgery at any time or of esophageal dilation procedures within the last 12 weeks prior to screening endoscopy, presence of an acute obstruction and/or need for an immediate endoscopic intervention due to a stricture
• In case of treatment equivalence of either steroid, PPI or dietary treatment: Patient/ parents wish to install a new or changed dietary or PPI approach
• Patient with high disease burden at screening and/or any patient with need for immediate treatment (incl. due to severe dysphagia, dehydration, loss of weight),
• Upper gastrointestinal bleeding within 8 weeks prior to screening endoscopy
• Existing or intended pregnancy or breast-feeding
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E.5 End points |
E.5.1 | Primary end point(s) |
Double-blind phase:
Rate of patients with pathological remission and clinical response at DB week 12
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
Double-blind phase:
- Rate of patients with histological remission at DB week 12
- Rate of patients with clinical response at DB week 12
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | Yes |
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) | Yes |
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 | 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 | Yes |
E.8.1.7.1 | Other trial design description |
plus 2 optional open-label verum-phases: 12 wk for induction and 24 wk for maintenance of remission |
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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 | 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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 24 |
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 |
Israel |
Germany |
Greece |
Netherlands |
Portugal |
Spain |
United Kingdom |
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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 | 3 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 30 |
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 | 3 |
E.8.9.2 | In all countries concerned by the trial days | 0 |