Evaluation of Viscous Budesonide Formulation Efficacy in Pediatric Eosinophilic Esophagitis: A Pilot Clinical Study

The primary objective of this clinical study is the **evaluation of the efficacy** of a new viscous budesonide suspension in children and adolescents diagnosed with **Eosinophilic Esophagitis** (EoE). This objective is clinically relevant as it aims to determine the therapeutic potential of this formulation in managing EoE, a chronic immune-mediated condition characterized by eosinophilic infiltration of the esophagus, which can lead to significant morbidity in pediatric populations.

Secondary objectives include:

• Evaluation of clinical response, which will provide insights into the symptomatic improvement in patients.
• Evaluation of endoscopic response, assessing the histological changes in the esophagus post-treatment.
• Safety evaluation of the new viscous budesonide suspension, ensuring the formulation's safety profile is acceptable for pediatric use.

The clinical trial focuses on evaluating the efficacy of a new viscous **budesonide** suspension in children and adolescents diagnosed with **Eosinophilic Esophagitis** (EoE). The study population includes both male and female participants aged 4 to 18 years. Participants are required to have a confirmed diagnosis of EoE, characterized by more than 15 eosinophils per high-power field in esophageal biopsies, and must have shown a lack of response to proton pump inhibitors or experienced a flare-up after discontinuation of these inhibitors. The trial includes individuals who have previously undergone topical steroid therapy without success or who experienced a flare-up post-therapy. The study population is considered vulnerable, and informed written consent from a parent or legal representative, along with verbal consent from the child, is mandatory. Female participants of childbearing age must undergo a urine pregnancy test and adhere to acceptable contraceptive methods during the study. The sponsor has not provided information regarding the total number of participants in the trial.

The clinical trial is designed to evaluate the efficacy of a new viscous **budesonide** suspension in children and adolescents diagnosed with **Eosinophilic Esophagitis** (EoE). This study is a phase IV, randomized, double-blind, controlled trial, with an estimated duration from October 2024 to April 2025. Participants will be randomly assigned to receive either the investigational product or a control, with neither the participants nor the investigators aware of the group assignments, ensuring the double-blind nature of the trial. The trial will involve multiple study visits, beginning with an inclusion (screening) visit to confirm eligibility based on specific criteria, including age between 4-18 years and a confirmed diagnosis of EoE with a peak intraepithelial eosinophil count of ≥15/HPF.

Following the screening, participants will undergo a series of follow-up visits at specified intervals to monitor the reduction or disappearance of eosinophil count in the esophageal mucosa, as well as to assess symptomatic and endoscopic scores. The primary endpoint is the reduction of eosinophil count to less than 15 EoE/HPF after 12 weeks of therapy. Secondary endpoints include the reduction of symptomatic and endoscopic scores and the monitoring of adverse events (AEs) and severe adverse events (SAEs) at weeks 6 and 12. The end-of-study visit will conclude the trial, where final assessments will be conducted to evaluate the overall efficacy and safety of the treatment.

Participant involvement is expected to last for a maximum of 12 weeks, corresponding to the treatment period. Conditions that may lead to early termination from the study include non-compliance with the study protocol, withdrawal of consent, or the occurrence of significant adverse events that compromise participant safety. The trial aims to provide valuable insights into the therapeutic potential of the new budesonide formulation for managing EoE in the pediatric population.

The clinical trial involves the use of **Budesonide**, an experimental medication formulated as a **powder for inhalation**. In this study, Budesonide is administered in a novel galenic formulation, where it is dissolved in Wagner Base Solution by the Hospital Pharmacy. The medication is intended for **oral use**. The maximum daily dose of Budesonide is 4 mg, with a total maximum dose of 336 mg over the course of the treatment. The treatment period is set for a maximum of 12 weeks. Budesonide is a chemical substance and is not classified as an orphan drug. The primary objective of the trial is to evaluate the efficacy of this new viscous Budesonide suspension in children and adolescents diagnosed with **Eosinophilic Esophagitis (EoE)**.

In addition to the experimental treatment, the study may involve the use of **cortisonic** as a non-experimental treatment. This is considered a standard-of-care therapy and is not the primary focus of the trial. The administration of cortisonic will follow standard medical guidelines and will be used to support the management of EoE symptoms as necessary. Participant compliance with the dosing schedule will be monitored throughout the trial to ensure adherence to the treatment protocol.

The efficacy of the new viscous budesonide suspension in children and adolescents with **Eosinophilic Esophagitis** (EoE) will be assessed through a series of primary and secondary endpoints. The primary endpoint is the reduction or disappearance of eosinophil count in the oesophageal mucosa, with a target of less than 15 eosinophils per high power field (HPF) after 12 weeks of therapy (T12). Secondary endpoints include the reduction of the symptomatic score and the endoscopic score, both calculated after 12 weeks of therapy (T12). Additionally, the monitoring and collection of adverse events (AEs) and severe adverse events (SAEs) will occur at 6 weeks (T6) and 12 weeks (T12) to evaluate the safety profile of the treatment. These efficacy parameters will be measured and collected using validated clinical and laboratory methods, ensuring a comprehensive assessment of the treatment's impact on the disease.