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Double-blind (neither physician nor patient knows of the actual treatment which can be with or without active substance), randomized (patient will be allocated to a certain treatment group by chance), placebo-controlled (one of the treatment groups receives medication without active substance), phase II/III study on the efficacy and tolerability of oral budesonide suspension in comparison with placebo in children and adolescents with eosinophilic esophagitis

Phase 1
Conditions
Active eosinophilic esophagitis and maintenance of remission in eosinophilic esophagitis
MedDRA version: 20.1Level: LLTClassification code 10064220Term: Eosinophilic esophagitisSystem Organ Class: 100000004856
Therapeutic area: Diseases [C] - Digestive System Diseases [C06]
Registration Number
EUCTR2017-003737-29-GR
Lead Sponsor
Dr. Falk Pharma GmbH
Brief Summary

Not available

Detailed Description

Not available

Recruitment & Eligibility

Status
ot Recruiting
Sex
All
Target Recruitment
75
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

Are the trial subjects under 18? yes
Number of subjects for this age range: 75
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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

Study & Design

Study Type
Interventional clinical trial of medicinal product
Study Design
Not specified
Primary Outcome Measures
NameTimeMethod
Main Objective: Double-blind phase: <br>To prove superior efficacy of budesonide oral suspension compared to placebo in children and adolescents with eosinophilic esophagitis (EoE)<br>;Secondary Objective: Double-blind phase: <br>To further assess efficacy of budesonide oral suspension in children and adolescents with eosinophilic esophagitis (EoE)<br><br>;Primary end point(s): Double-blind phase: <br>Rate of patients with pathological remission and clinical response at DB week 12<br>;Timepoint(s) of evaluation of this end point: week 12
Secondary Outcome Measures
NameTimeMethod
Secondary end point(s): Double-blind phase: <br>- Rate of patients with histological remission at DB week 12<br>- Rate of patients with clinical response at DB week 12<br>;Timepoint(s) of evaluation of this end point: week 12
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