Double-blind, randomized, placebo-controlled, phase III trial on the efficacy and tolerability of a 6-week treatment with budesonide effervescent tablets vs. placebo for induction of clinico-pathological remission in adult patients with active eosinophilic esophagitis
- Conditions
- oesophageal inflammation10017966
- Registration Number
- NL-OMON42672
- Lead Sponsor
- Dr. Falk Pharma GmbH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 18
- Signed informed consent,
- Male or female patients, 18 to 75 years of age,
- Confirmed clinico-pathological diagnosis of EoE according to established diagnostic criteria ,
- Active symptomatic and histological 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
- Clinical signs (i.e., acid regurgitation and/or heart burn) and 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,
- 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.
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Rate of patients with clinico-pathological remission at week 6 (LOCF) defined<br /><br>as fulfilling both criteria:<br /><br>- Histological remission, i.e., peak of <16 eos/mm2 hpf at week 6 (LOCF), AND<br /><br>- Resolution of symptoms (i.e., no or only minimal problems) defined as a<br /><br>severity of *2 points on 0 to 10-point (0-10) NRS for dysphagia AND<br /><br>a severity of *2 points on 0-10 NRS for pain during swallowing on<br /><br>each day in the week prior to week 6 (LOCF).</p><br>
- Secondary Outcome Measures
Name Time Method <p>A priori ordered key secondary endpoints (DB-phase):<br /><br>1. Rate of patients with histological remission, defined as a peak of <16<br /><br>eos/mm2 hpf at week 6 (LOCF),<br /><br>2. Change in the peak eos/mm2 hpf from baseline to week 6 (LOCF),<br /><br>3. Rate of patients with resolution of symptoms (i.e., no or only minimal<br /><br>problems) defined as a severity of *2 points on 0-10 NRS for dysphagia AND a<br /><br>severity of *2 points on 0-10 NRS for pain during swallowing on each day in the<br /><br>week prior to week 6 (LOCF),<br /><br>4. Rate of patients with total weekly Eosinophilic Esophagitis Activity Index *<br /><br>Patient-Reported Outcome (EEsAI-PRO) score of *20 at week 6 (LOCF),<br /><br>5. Rate of patients with an improvement from baseline to week 6 (LOCF) in the<br /><br>weekly Visual Dysphagia Question (VDQ) score,<br /><br>6. Rate of patients with an improvement from baseline to week 6 (LOCF) in the<br /><br>weekly *Avoidance, Modification, and Slow-eating (AMS) score.</p><br>