Double-blind (neither physician nor patient knows of the actual treatment which can with or without active substance), randomized (patient will be allocated to a certain treatment group by chance), phase III study on the efficacy and tolerability of a 48-week treatment with two different doses of budesonide effervescent tablets in comparison with placebo for maintenance of clinico-pathological remission in adult patients with eosinophilic esophagitis

• Register: EUCTR
• Last refreshed on: 5 April 2021
• Main ID: EUCTR2014-001485-99-DE
• Date of registration: 29/06/2015
• Prospective Registration: Yes
• Primary sponsor: Dr. Falk Pharma GmbH
• Public title: Double-blind (neither physician nor patient knows of the actual treatment which can with or without active substance), randomized (patient will be allocated to a certain treatment group by chance), phase III study on the efficacy and tolerability of a 48-week treatment with two different doses of budesonide effervescent tablets in comparison with placebo for maintenance of clinico-pathological remission in adult patients with eosinophilic esophagitis
• Scientific title: Double-blind, randomized, placebo-controlled, phase III study on the efficacy and tolerability of a 48-week treatment with two different doses of budesonide effervescent tablets vs. placebo for maintenance of clinico-pathological remission in adult patients with eosinophilic esophagitis - Maintenance of remission with budesonide effervescent tabl. vs. placebo in eosinophilic esophagitis
• Date of first enrolment: 08/12/2015
• Target sample size: 204
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2014-001485-99
• 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: Open-label: possible 6-week phases for induction or re-induction of remission;48-96w extension phase; 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): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Belgium, Denmark, Germany, Netherlands, Spain, Switzerland, United Kingdom

Contacts

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

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

Key inclusion & exclusion criteria

Inclusion criteria:
- Signed informed consent,
- Male or female patients, 18 to 75 years of age,
- Confirmed clinico-pathological diagnosis of EoE according to established diagnostic criteria ,
- Clinico-pathological remission of EoE
- A documented trial with proton pump inhibitors (PPIs) in order to rule out PPI-responsive esophageal eosinophilia,
- Negative pregnancy test in females of childbearing potential at baseline visit

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 181
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 23

Exclusion criteria:
- Clinical signs (i.e., acid regurgitation and/or heart burn) and/or endoscopic signs (at least Los Angeles Classification of Esophagitis Grade A) of gastroesophageal reflux disease (GERD),
- History of abnormal results in case of an optionally performed pH monitoring of the distal esophagus,
- Patients with PPI-responsive esophageal eosinophilia
- 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, oropharyngeal or esophageal bacterial, viral, or fungal infection [candida esophagitis]),
- Any relevant systemic disease (e.g., AIDS, active tuberculosis),
- If careful medical monitoring is not ensured: cardiovascular disease, diabetes mellitus, osteoporosis, active peptic ulcer disease, glaucoma, cataract, or infection,
- Liver cirrhosis or portal hypertension,
- History of cancer in the last five years (except for non-metastatic cancers, e.g., basalioma),
- History of esophageal surgery at any time or of esophageal dilation procedures within the last 8 weeks prior to screening visit,
- Upper gastrointestinal bleeding within 8 weeks prior to screening visit,
- Existing or intended pregnancy or breast-feeding.



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

Health Condition(s) or Problem(s) studied

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

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

Intervention(s)

Product Name: 1 mg budesonide effervescent tablet for orodispersible use
Product Code: BUET 1mg
Pharmaceutical Form: Effervescent tablet
INN or Proposed INN: BUDESONIDE
CAS Number: 51333-22-3
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 1-
Pharmaceutical form of the placebo: Effervescent tablet
Route of administration of the placebo: Oral use

Product Name: 0.5 mg budesonide effervescent tablet for orodispersible use
Product Code: BUET 0.5 mg
Pharmaceutical Form: Effervescent tablet
INN or Proposed INN: BUDESONIDE
CAS Number: 51333-22-3
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 0.5-
Pharmaceutical form of the placebo: Effervescent tablet
Route of administration of the placebo: Oral use

Primary Outcome(s)

Secondary Objective: Double-blind phase:
- To study safety and tolerability in the form of adverse events and laboratory parameters
- To assess patients’ quality of life.

Open-label re-induction phase:
- To study re-induction of clinical response in patients with a clinical or histological relapse or having experienced a food impaction which needed endoscopic intervention,

Open-label extension phase:
- To study maintenance of clinical remission in patients who completed the double-blind phase without a clinical or histological relapse

Exploratory:
• To study biomarkers in EoE.

Timepoint(s) of evaluation of this end point: after 48 weeks of double-blind phase

Main Objective: Double-blind phase:
- To assess the efficacy of a 48-week treatment with 2 x 0.5 mg/d or 2 x 1 mg/d budesonide effervescent tablets vs. placebo for the maintenance of clinico-pathological remission in adult patients with eosinophilic esophagitis (EoE).

Primary end point(s): Rate of patients free of treatment failure after 48 weeks of treatment

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: after 48 weeks of double-blind phase

Secondary end point(s): Secondary efficacy endpoints:
- Rate of patients with histological relapse at week 48 of the double-blind phase
- Rate of patients with a clinical relapse, have experienced a food impaction which needed endoscopic intervention, or needed an endoscopic dilation during the DB treatment phase

Secondary ID(s)

Acronym

NCT02493335

BUL-2/EER

Source(s) of Monetary Support

Dr. Falk Pharma GmbH

Ethics review

Status: Approved
Approval date: 08/12/2015
Contact: