# Distensibility and Remodeling as markers of Therapeutic Response in Eosinophilic Esophagitis

> **NIH NIH R21** · CHILDREN'S HOSP OF PHILADELPHIA · 2020 · $178,750

## Abstract

Project Summary
 Eosinophilic esophagitis (EoE) is a rare allergic disease characterized by esophageal infiltration of
eosinophils. This inflammatory infiltrate leads to dysphagia, impaction of food, and esophageal stricture. There
is no FDA approved medication for EoE and the only available therapy to halt disease progression is utilizing
restrictive diets or off label steroid preparations which are fraught with compliance issues and inconsistent dosing
respectively. Thus, there is a critical need for FDA approved therapies in order to improve outcomes.
 Clinical trials to date have used a diverse number of clinical scoring symptoms, histologic evaluations
and physician assessments. Due in part to the heterogeneity in clinical symptoms of affected children and adults,
demonstrating improved symptom outcomes has been a major obstacle in early EoE trials. Having an objective
measure of esophageal function in this patient population would improve clinical trial outcomes. Endoluminal
Functional Lumen Imagaing Probe or EndoFLIP is a new technique that uses high-resolution impedance
palimetry to determine the distensibility of hollow gastrointestinal organs including the esophagus. Our
preliminary work has shown that in patients with active EoE, there is a significantly less distensible esophagus.
We therefore seek to use EndoFLIP to provide critical data to inform the design of future interventional trials.
We hypothesize the EndoFLIP will serve as a practical drug development tool to provide an objective outcome
measures of therapeutic response in EoE clinical trials. We will first validate use of the EndoFLIP in its ability to
capture treatment response in a well-defined pediatric EoE population and determine its relationship to other
currently available outcome measures (Aim 1). We will then define an approach to pediatric EoE clinical trials
using EndoFLIP measurements as an outcome measure by establishing an expected effect size for stratified
age groups and treatment duration (Aim 2). This project will provide necessary and sufficient data for designing
trials addressing key questions in the treatment of EoE in children. Incorporation of a novel esophageal clinical
outcome assessment will fill a critical gap in our current tools available for drug development for this rare disease.
 The two primary investigators and study sites, both with expertise in EoE, are uniquely suited to
accomplish these aims and have successfully partnered to enroll subjects in clinical studies using EndoFLIP and
publish original and novel data on the use of EndoFLIP in this patient population.

## Key facts

- **NIH application ID:** 9996820
- **Project number:** 5R21TR003039-02
- **Recipient organization:** CHILDREN'S HOSP OF PHILADELPHIA
- **Principal Investigator:** CALIES D Menard-Katcher
- **Activity code:** R21 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 2020
- **Award amount:** $178,750
- **Award type:** 5
- **Project period:** 2019-08-15 → 2022-06-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/9996820

## Citation

> US National Institutes of Health, RePORTER application 9996820, Distensibility and Remodeling as markers of Therapeutic Response in Eosinophilic Esophagitis (5R21TR003039-02). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/9996820. Licensed CC0.

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