Oral Cromolyn Sodium for the Treatment of Eosinophilic Esophagitis

A Randomized, Double-blind, Placebo-controlled Study of the Use of Oral Cromolyn Sodium for the Treatment of Eosinophilic Esophagitis

This is a randomized, double-blind, placebo-controlled study of oral cromolyn sodium when made into a viscous preparation for the treatment of eosinophilic esophagitis.

Study Overview

• Status: Completed
• Conditions: Eosinophilic Esophagitis
• Intervention / Treatment: Drug: Placebo; Drug: oral cromolyn sodium

Detailed Description

Eosinophilic esophagitis is an antigen-mediated allergic disease of the esophagus characterized by symptoms of gastrointestinal complaints and eosinophilic inflammation limited to the esophagus. Currently, first line therapeutic recommendations include swallowed, topical steroids or dietary therapy. While both work for the majority of patients, they both have limitations.

The investigators are examining the use of oral cromolyn sodium as a treatment for this condition. This medication is a non-steroid that is already approved for other conditions. When taken orally, it is essentially not absorbed systemically, so side effects are minimal. There is only 1 brief, retrospective report of its use in this condition suggesting it does not work. However, from studying swallowed, topical steroids, it may require formulating the medication into a viscous preparation for it to work.

• Study Type: Interventional
• Enrollment (Actual): 16
• Phase: Phase 4

Contacts and Locations

Study Locations

• United States
  • Tennessee
    • Memphis, Tennessee, United States, 38105
      • LeBonheur Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 7 months to 16 years (Child, Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Diagnosis of eosinophilic esophagitis

Exclusion Criteria:

• Concomitant treatment with swallowed corticosteroids. Any prior use of swallowed corticosteroids will require a 4 week washout period.
• Pregnancy (all females of child bearing potential will have urine pregnancy test done at baseline).
• Evidence of pathologic eosinophilia in other locations in the GI tract.
• Participation in another research protocol
• Reduced Renal or Hepatic Function (all subjects will have a baseline creatinine and liver function panel drawn)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Oral cromolyn; Subjects randomized to the experimental arm will receive oral cromolyn sodium. The dose will be per current package insert for oral cromolyn: Subjects 2-12 years of age - 100 mg (1 ampule) 4 times daily Subjects 13-18 years of age - 200 mg (2 ampules) 4 times daily	Drug: oral cromolyn sodium; Oral cromolyn sodium; Other Names: Gastrocrom
Placebo Comparator: Placebo; Subjects randomized to placebo will receive normal saline ampules Subjects 2-12 years of age - 1 ampule 4 times daily Subjects 13-18 years of age - 2 ampules 4 times daily	Drug: Placebo; Saline

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Peak Esophageal Eosinophil Count	Measure of the peak esophageal eosinophil count on esophageal biopsy specimens after 2 months of therapy	2 months

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Symptoms as Measured by Pediatric Eosinophilic Esophagitis Symptom Score	Symptom scores will be measures at baseline, 1 month, and 2 months using a symptom scoring assessment tool known as the Pediatric Eosinophilic Esophagitis Symptom Score (PEESS). The PEESS is a validated symptom score that is completed by the parent of the patient. It encompasses frequency and severity of EoE related symptoms over the past month. The minimum score = 0. The max score = 98 The higher the score/value, the more severe and/or frequent the symptoms and therefore a worse outcome.	Baseline as compared to 2 months

Collaborators and Investigators

• Sponsor: University of Tennessee
• Investigators: Principal Investigator: Jay A Lieberman, MD, The University of Tennessee Health Science Center

Publications and helpful links

General Publications

• Lieberman JA, Zhang J, Whitworth J, Cavender C. A randomized, double-blinded, placebo-controlled study of the use of viscous oral cromolyn sodium for the treatment of eosinophilic esophagitis. Ann Allergy Asthma Immunol. 2018 May;120(5):527-531. doi: 10.1016/j.anai.2018.03.006. Epub 2018 Mar 12.

Study record dates

Study Major Dates

• Study Start: December 1, 2014
• Primary Completion (Actual): July 1, 2017
• Study Completion (Actual): December 1, 2017

Study Registration Dates

• First Submitted: February 11, 2015
• First Submitted That Met QC Criteria: February 25, 2015
• First Posted (Estimated): February 26, 2015

Study Record Updates

• Last Update Posted (Actual): October 26, 2023
• Last Update Submitted That Met QC Criteria: October 24, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Digestive System Diseases; Immune System Diseases; Hypersensitivity, Immediate; Hematologic Diseases; Gastrointestinal Diseases; Gastroenteritis; Hypersensitivity; Esophageal Diseases; Leukocyte Disorders; Eosinophilia; Eosinophilic Esophagitis; Esophagitis; Physiological Effects of Drugs; Anti-Inflammatory Agents; Immunologic Factors; Anti-Asthmatic Agents; Respiratory System Agents; Anti-Allergic Agents; Mast Cell Stabilizers; Cromolyn Sodium
• Other Study ID Numbers: 13-02521-FB

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO