Trial Outcomes & Findings for Oral Cromolyn Sodium for the Treatment of Eosinophilic Esophagitis (NCT NCT02371941)
NCT ID: NCT02371941
Last Updated: 2023-10-26
Results Overview
Measure of the peak esophageal eosinophil count on esophageal biopsy specimens after 2 months of therapy
Recruitment status
COMPLETED
Study phase
PHASE4
Target enrollment
16 participants
Primary outcome timeframe
2 months
Results posted on
2023-10-26
Participant Flow
Participant milestones
| Measure |
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
|
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
|
|---|---|---|
|
Overall Study
STARTED
|
9
|
7
|
|
Overall Study
COMPLETED
|
9
|
6
|
|
Overall Study
NOT COMPLETED
|
0
|
1
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Oral Cromolyn Sodium for the Treatment of Eosinophilic Esophagitis
Baseline characteristics by cohort
| Measure |
Oral Cromolyn
n=9 Participants
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
|
Placebo
n=7 Participants
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
|
Total
n=16 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
9 Participants
n=99 Participants
|
7 Participants
n=107 Participants
|
16 Participants
n=206 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Continuous
|
10.4 years
STANDARD_DEVIATION 4.1 • n=99 Participants
|
12.8 years
STANDARD_DEVIATION 4 • n=107 Participants
|
11.5 years
STANDARD_DEVIATION 4 • n=206 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=99 Participants
|
3 Participants
n=107 Participants
|
8 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=99 Participants
|
4 Participants
n=107 Participants
|
8 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
8 Participants
n=99 Participants
|
7 Participants
n=107 Participants
|
15 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
1 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
9 participants
n=99 Participants
|
7 participants
n=107 Participants
|
16 participants
n=206 Participants
|
PRIMARY outcome
Timeframe: 2 monthsMeasure of the peak esophageal eosinophil count on esophageal biopsy specimens after 2 months of therapy
Outcome measures
| Measure |
Oral Cromolyn
n=9 Participants
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
|
Placebo
n=6 Participants
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
|
|---|---|---|
|
Peak Esophageal Eosinophil Count
|
57.3 Peak # of eosinophils counted per field
Standard Deviation 44.02
|
71.3 Peak # of eosinophils counted per field
Standard Deviation 52.7
|
SECONDARY outcome
Timeframe: Baseline as compared to 2 monthsSymptom 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.
Outcome measures
| Measure |
Oral Cromolyn
n=9 Participants
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
|
Placebo
n=6 Participants
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
|
|---|---|---|
|
Symptoms as Measured by Pediatric Eosinophilic Esophagitis Symptom Score
Baseline PEES score
|
37.89 units on a scale
Standard Deviation 12.2
|
30.3 units on a scale
Standard Deviation 13.7
|
|
Symptoms as Measured by Pediatric Eosinophilic Esophagitis Symptom Score
2 month PEES score
|
17.5 units on a scale
Standard Deviation 12.8
|
22.3 units on a scale
Standard Deviation 19.1
|
Adverse Events
Oral Cromolyn
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Oral Cromolyn
n=9 participants at risk
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
|
Placebo
n=7 participants at risk
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
|
|---|---|---|
|
Gastrointestinal disorders
nausea
|
55.6%
5/9 • Number of events 5 • 2 months
|
14.3%
1/7 • Number of events 1 • 2 months
|
|
Gastrointestinal disorders
abdominal pain
|
44.4%
4/9 • Number of events 4 • 2 months
|
28.6%
2/7 • Number of events 2 • 2 months
|
|
Nervous system disorders
headache
|
44.4%
4/9 • Number of events 4 • 2 months
|
14.3%
1/7 • Number of events 1 • 2 months
|
Additional Information
Dr. Jay Lieberman
The University of Tennessee Health Science Center
Phone: 901-287-7337
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place