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

• Register: EUCTR
• Last refreshed on: 18 September 2023
• Main ID: EUCTR2017-003737-29-NL
• Date of registration: 09/04/2019
• Prospective Registration: Yes
• Primary sponsor: Dr. Falk Pharma GmbH
• Public title: 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
• Scientific title: Double-blind, randomized, placebo-controlled, Phase II/III trial on the efficacy and tolerability of treatment with budesonide oral suspension vs. placebo in children and adolescents with eosinophilic esophagitis - Budesonide oral suspension vs. placebo in pediatric eosinophilic esophagitis
• Date of first enrolment: 01/04/2020
• Target sample size: 75
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2017-003737-29
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: yes Other trial design description: plus 2 optional open-label verum-phases: 12 wk for induction and 24 wk for maintenance of remission If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 3
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): yes Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Germany, Greece, Israel, Netherlands, Portugal, Spain, United Kingdom

Contacts

• Name: Dept. of Clin. Res. & Development
• Affiliation: Dr. Falk Pharma GmbH

• Name: Dept. of Clin. Res. & Development
• Affiliation: Dr. Falk Pharma GmbH

Key inclusion & exclusion criteria

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 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 ecquivalence 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 treatemtn (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



Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Digestive System Diseases [C06]

Active eosinophilic esophagitis and maintenance of remission in eosinophilic esophagitis
MedDRA version: 20.1 Level: LLT Classification code 10064220 Term: Eosinophilic esophagitis System Organ Class: 100000004856

Intervention(s)

Product Name: Budesonide oral suspension [0.2 mg/ml]
Pharmaceutical Form: Oral suspension
INN or Proposed INN: BUDESONIDE
CAS Number: 51333-22-3
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 0.2-
Pharmaceutical form of the placebo: Oral suspension
Route of administration of the placebo: Oral use

Primary Outcome(s)

Main Objective: Double-blind phase:
To prove superior efficacy of budesonide oral suspension compared to placebo in children and adolescents with eosinophilic esophagitis (EoE)

Primary end point(s): Double-blind phase:
Rate of patients with pathological remission and clinical response at DB week 12

Timepoint(s) of evaluation of this end point: week 12

Secondary Objective: Double-blind phase:
To further assess efficacy of budesonide oral suspension in children and adolescents with eosinophilic esophagitis (EoE)

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: week 12

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

Secondary ID(s)

2017-003737-29-DE

BUU-5/EEA

Acronym

Source(s) of Monetary Support

Dr. Falk Pharma GmbH

Ethics review

Status: Approved
Approval date: 01/04/2020
Contact: