Eosinophilic Esophagitis
Conditions
Brief summary
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.
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.
Interventions
DRUGoral cromolyn sodium
Oral cromolyn sodium
DRUGPlacebo
Saline
Sponsors
University of Tennessee
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
2 Years to 18 Years
Healthy volunteers
No
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)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Peak Esophageal Eosinophil Count | 2 months | Measure of the peak esophageal eosinophil count on esophageal biopsy specimens after 2 months of therapy |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Symptoms as Measured by Pediatric Eosinophilic Esophagitis Symptom Score | Baseline as compared to 2 months | 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. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| 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
oral cromolyn sodium: Oral cromolyn sodium | 9 |
| 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
Placebo: Saline | 7 |
| Total | 16 |
Baseline characteristics
| Characteristic | Placebo | Total | Oral Cromolyn |
|---|---|---|---|
| Age, Categorical <=18 years | 7 Participants | 16 Participants | 9 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Continuous | 12.8 years STANDARD_DEVIATION 4 | 11.5 years STANDARD_DEVIATION 4 | 10.4 years STANDARD_DEVIATION 4.1 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 1 Participants | 1 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 7 Participants | 15 Participants | 8 Participants |
| Region of Enrollment United States | 7 participants | 16 participants | 9 participants |
| Sex: Female, Male Female | 3 Participants | 8 Participants | 5 Participants |
| Sex: Female, Male Male | 4 Participants | 8 Participants | 4 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 9 | 0 / 7 |
| other Total, other adverse events | 8 / 9 | 4 / 7 |
| serious Total, serious adverse events | 0 / 9 | 0 / 7 |
Outcome results
Primary
Peak Esophageal Eosinophil Count
Measure of the peak esophageal eosinophil count on esophageal biopsy specimens after 2 months of therapy
Time frame: 2 months
| Arm | Measure | Value (MEDIAN) | Dispersion |
|---|---|---|---|
| Oral Cromolyn | Peak Esophageal Eosinophil Count | 57.3 Peak # of eosinophils counted per field | Standard Deviation 44.02 |
| Placebo | Peak Esophageal Eosinophil Count | 71.3 Peak # of eosinophils counted per field | Standard Deviation 52.7 |
Secondary
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.
Time frame: Baseline as compared to 2 months
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Oral Cromolyn | Symptoms as Measured by Pediatric Eosinophilic Esophagitis Symptom Score | 2 month PEES score | 17.5 units on a scale | Standard Deviation 12.8 |
| Oral Cromolyn | Symptoms as Measured by Pediatric Eosinophilic Esophagitis Symptom Score | Baseline PEES score | 37.89 units on a scale | Standard Deviation 12.2 |
| Placebo | Symptoms as Measured by Pediatric Eosinophilic Esophagitis Symptom Score | Baseline PEES score | 30.3 units on a scale | Standard Deviation 13.7 |
| Placebo | Symptoms as Measured by Pediatric Eosinophilic Esophagitis Symptom Score | 2 month PEES score | 22.3 units on a scale | Standard Deviation 19.1 |