Budesonide oral suspension vs. placebo in pediatric EoE
Research type
Research Study
Full title
Double-blind, randomised, 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
IRAS ID
260340
Contact name
Marcus Auth
Contact email
Sponsor organisation
Dr Falk Pharma GmbH
Eudract number
2017-003737-29
Duration of Study in the UK
2 years, 10 months, 0 days
Research summary
Currently there is no drug authorised for the treatment of eosinophilic oesophagitis (EoE) in children and adolescents. Other formulations of the study drug, Budesonide, have been proven to be safe and effective in adults. Budesonide works locally by reducing inflammation of the oesophagus whilst minimising side effects. The Budesonide oral suspension that is used in this study has been developed for children and adolescents.
Patients with EoE aged 2 to <18 years and their parents/guardians will be invited to participate. Those who agree and consent/assent and who meet eligibility criteria will be assigned to one of three treatment groups during a 12-week double-blind (DB) phase. Those aged 2-11 will get either 0.5 or 1mg Budesonide daily or placebo. Those aged 12-17 will receive either 1 or 2mg Budesonide or placebo. Patients who are not in pathological remission and clinical response/remission after at least 8 weeks of DB phase, are invited to continue in the study on a 12-week Open Label Induction (OLI) phase, during which he/she will receive either 1mg daily if aged 2-11 or 2mg daily if 12-17. If the patient is in remission after the double blind or OLI phase, he/she can continue in the study on a 24-week open label extension phase (OLE) and receive either 0.5 or 1mg Budesonide daily if aged 2-11, or 1 or 2mg Budesonide if aged 12-17. After last treatment phase, there is a 3-week tapering phase should the patient not continue in the study, followed by a 4-week safety follow up. Patients will be assessed regularly throughout, blood and urine samples will be collected for analysis, along with biopsies during endoscopies. A number of patient and parent questionnaires and diaries are required.
75 patients in Europe and Israel will be included. This study will be run at two UK hospitals.
REC name
North West - Haydock Research Ethics Committee
REC reference
19/NW/0426
Date of REC Opinion
1 Aug 2019
REC opinion
Further Information Favourable Opinion