Trial Outcomes & Findings for The Role Of Gastroesophageal Reflux Disease (GERD) in Eosinophilic Esophagitis (NCT NCT00728481)
NCT ID: NCT00728481
Last Updated: 2016-01-14
Results Overview
Subjects with Esophageal eosinophilia experiencing a histological response to treatment. Subjects were considered to have histological response to treatment if both sets of biopsies (from the distal and mid-esophagus) had, on average, less than 5 eosinophils per high power field (eos/hpf) at the 6-month biopsies.
COMPLETED
PHASE2/PHASE3
51 participants
Baseline, 6 months
2016-01-14
Participant Flow
Subjects with a clinical diagnosis of Eosinophilic Esophagitis (EE) were recruited by mail using a Mayo Clinic proprietary database and also by direct contact in the Mayo Clinic Esophageal Diseases speciality clinic in Rochester, MN. Subjects were enrolled from 3/2009 to 8/2010.
Patients with esophageal eosinophilic infiltration of greater than or equal to 15 eos/hpf as determined by 4 biopsies of the esophagus, then underwent a 24-hour transnasal catheter-based ambulatory pH study to determine amount of distal esophageal acid exposure, and were placed in one of 2 treatment arms based on positive or negative pH results.
Participant milestones
| Measure |
Esomeprazole
Proton pump inhibitor; Nexium 40mg capsule taken twice daily by mouth for 6 weeks for subjects with positive 24 hour pH study
|
Budesonide
Corticosteroid therapy; oral viscous Pulmicort Respules 1 gram taken by mouth orally twice daily (mixed with 1 gram packet of Sucralose \[Splenda-registered trademark\]) for 6 weeks in subjects with negative 24 hour pH studies (without GERD)
|
|---|---|---|
|
Overall Study
STARTED
|
19
|
32
|
|
Overall Study
COMPLETED
|
18
|
28
|
|
Overall Study
NOT COMPLETED
|
1
|
4
|
Reasons for withdrawal
| Measure |
Esomeprazole
Proton pump inhibitor; Nexium 40mg capsule taken twice daily by mouth for 6 weeks for subjects with positive 24 hour pH study
|
Budesonide
Corticosteroid therapy; oral viscous Pulmicort Respules 1 gram taken by mouth orally twice daily (mixed with 1 gram packet of Sucralose \[Splenda-registered trademark\]) for 6 weeks in subjects with negative 24 hour pH studies (without GERD)
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
|
Overall Study
Adverse Event
|
0
|
1
|
|
Overall Study
Study medication non-compliance
|
0
|
2
|
|
Overall Study
Incorrectly assigned to opposite arm
|
1
|
0
|
Baseline Characteristics
The Role Of Gastroesophageal Reflux Disease (GERD) in Eosinophilic Esophagitis
Baseline characteristics by cohort
| Measure |
Esomeprazole
n=19 Participants
Proton pump inhibitor; Nexium 40mg capsule taken twice daily by mouth for 6 weeks for subjects with positive 24 hour pH study
|
Budesonide
n=32 Participants
Corticosteroid therapy; oral viscous Pulmicort Respules 1 gram taken by mouth orally twice daily (mixed with 1 gram packet of Sucralose \[Splenda-registered trademark\]) for 6 weeks in subjects with negative 24 hour pH studies (without GERD)
|
Total
n=51 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
47.5 years
STANDARD_DEVIATION 14.9 • n=99 Participants
|
33.7 years
STANDARD_DEVIATION 10.4 • n=107 Participants
|
39 years
STANDARD_DEVIATION 13.9 • n=206 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=99 Participants
|
13 Participants
n=107 Participants
|
20 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=99 Participants
|
19 Participants
n=107 Participants
|
31 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
19 participants
n=99 Participants
|
32 participants
n=107 Participants
|
51 participants
n=206 Participants
|
|
Mean number of eosinophils per high powered field in esophagus
|
35.6 eos/hpf
STANDARD_DEVIATION 24.7 • n=99 Participants
|
46.7 eos/hpf
STANDARD_DEVIATION 26.5 • n=107 Participants
|
41.2 eos/hpf
STANDARD_DEVIATION 27.7 • n=206 Participants
|
|
Endoscopic evidence of GERD
Multiple rings (felinization or corrugations)
|
14 Participants
n=99 Participants
|
18 Participants
n=107 Participants
|
32 Participants
n=206 Participants
|
|
Endoscopic evidence of GERD
Hiatal hernia
|
15 Participants
n=99 Participants
|
10 Participants
n=107 Participants
|
25 Participants
n=206 Participants
|
|
Endoscopic evidence of GERD
Erosive esophagitis
|
7 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
9 Participants
n=206 Participants
|
|
Number of participants with frequent GERD/dysphagia symptoms
Frequent symptoms
|
15 participants
n=99 Participants
|
0 participants
n=107 Participants
|
15 participants
n=206 Participants
|
|
Number of participants with frequent GERD/dysphagia symptoms
No or infrequent symptoms
|
3 participants
n=99 Participants
|
17 participants
n=107 Participants
|
20 participants
n=206 Participants
|
|
Number of participants with frequent GERD/dysphagia symptoms
No symptoms
|
0 participants
n=99 Participants
|
15 participants
n=107 Participants
|
15 participants
n=206 Participants
|
PRIMARY outcome
Timeframe: Baseline, 6 monthsSubjects with Esophageal eosinophilia experiencing a histological response to treatment. Subjects were considered to have histological response to treatment if both sets of biopsies (from the distal and mid-esophagus) had, on average, less than 5 eosinophils per high power field (eos/hpf) at the 6-month biopsies.
Outcome measures
| Measure |
Esomeprazole
n=18 Participants
Proton pump inhibitor; Nexium 40mg capsule taken twice daily by mouth for 6 weeks for subjects with positive 24 hour pH study
|
Budesonide
n=28 Participants
Corticosteroid therapy; oral viscous Pulmicort Respules 1 gram taken by mouth orally twice daily (mixed with 1 gram packet of Sucralose \[Splenda-registered trademark\]) for 6 weeks in subjects with negative 24 hour pH studies (without GERD)
|
|---|---|---|
|
Histological Response to Treatment
Subjects with histological response
|
11 Participants
|
16 Participants
|
|
Histological Response to Treatment
Subjects with nonsignificant histological response
|
7 Participants
|
12 Participants
|
PRIMARY outcome
Timeframe: Baseline, 6 monthsSubjects with Esophageal eosinophilia experiencing a response in their dysphagia symptoms to treatment. Symptomatic improvement in symptoms was defined as a score of at least two levels lower than the baseline dysphagia symptom question on the Mayo Dysphagia Questionnaire-30 days (MDQ-30). Dysphagia symptoms were determined based on the MDQ-30 question: 'How would you rate the severity of your trouble swallowing in the past 30 days' with a 5 point scale ranging from 'does not bother me at all' to 'very severe, markedly affects my lifestyle'. Patients must have marked a score of 3 or higher corresponding to 'Moderate, cannot be ignored, but does not affect my lifestyle' to be included in the study.
Outcome measures
| Measure |
Esomeprazole
n=18 Participants
Proton pump inhibitor; Nexium 40mg capsule taken twice daily by mouth for 6 weeks for subjects with positive 24 hour pH study
|
Budesonide
n=28 Participants
Corticosteroid therapy; oral viscous Pulmicort Respules 1 gram taken by mouth orally twice daily (mixed with 1 gram packet of Sucralose \[Splenda-registered trademark\]) for 6 weeks in subjects with negative 24 hour pH studies (without GERD)
|
|---|---|---|
|
Symptomatic Response to Treatment
Subjects with response in dysphagia symptoms
|
11 Participants
|
15 Participants
|
|
Symptomatic Response to Treatment
Subjects with no response in dysphagia symptoms
|
7 Participants
|
13 Participants
|
SECONDARY outcome
Timeframe: Baseline, 6 monthsPopulation: The analysis population includes only subjects with a histologic response. Subjects were considered to have a histological response to treatment if both sets of biopsies (from distal \& mid-esophagus) had, on average, less than 5 eos/hpf.
Dysphagia symptoms were determined based on the Mayo Dysphagia Questionnaire-30 days (MDQ-30), using the question: 'How would you rate the severity of your trouble swallowing in the past 30 days' with a 5 point scale ranging from 'does not bother me at all' to 'very severe, markedly affects my lifestyle'. Symptomatic improvement was defined as only an improvement of 2 levels on this question.
Outcome measures
| Measure |
Esomeprazole
n=11 Participants
Proton pump inhibitor; Nexium 40mg capsule taken twice daily by mouth for 6 weeks for subjects with positive 24 hour pH study
|
Budesonide
n=16 Participants
Corticosteroid therapy; oral viscous Pulmicort Respules 1 gram taken by mouth orally twice daily (mixed with 1 gram packet of Sucralose \[Splenda-registered trademark\]) for 6 weeks in subjects with negative 24 hour pH studies (without GERD)
|
|---|---|---|
|
Change in Dysphagia Symptoms in Subjects With Histological Response to Treatment
Dysphagia symptoms improved
|
5 Participants
|
11 Participants
|
|
Change in Dysphagia Symptoms in Subjects With Histological Response to Treatment
Dysphagia symptoms did not improve
|
6 Participants
|
5 Participants
|
SECONDARY outcome
Timeframe: Baseline, 6 monthsPopulation: The analysis population for this item only included subjects with a non-significant histologic response. Subjects were considered to have a histological response to treatment if both sets of 6-month biopsies (from distal \& mid-esophagus) had, on average, less than 5 eos/hpf.
Dysphagia symptoms were determined based on the Mayo Dysphagia Questionnaire-30 days (MDQ-30), using the question: 'How would you rate the severity of your trouble swallowing in the past 30 days' with a 5 point scale ranging from 'does not bother me at all' to 'very severe, markedly affects my lifestyle'. Patients must have marked a score of 3 or higher corresponding to 'Moderate, cannot be ignored, but does not affect my lifestyle' to be included in the study.
Outcome measures
| Measure |
Esomeprazole
n=7 Participants
Proton pump inhibitor; Nexium 40mg capsule taken twice daily by mouth for 6 weeks for subjects with positive 24 hour pH study
|
Budesonide
n=12 Participants
Corticosteroid therapy; oral viscous Pulmicort Respules 1 gram taken by mouth orally twice daily (mixed with 1 gram packet of Sucralose \[Splenda-registered trademark\]) for 6 weeks in subjects with negative 24 hour pH studies (without GERD)
|
|---|---|---|
|
Change in Dysphagia Symptoms in Subjects With Non-significant Histological Response to Treatment
Dysphagia symptoms responded to treatment
|
6 Participants
|
4 Participants
|
|
Change in Dysphagia Symptoms in Subjects With Non-significant Histological Response to Treatment
Dysphagia symptoms did not respond
|
1 Participants
|
8 Participants
|
SECONDARY outcome
Timeframe: Baseline, 6 monthsMultiple concentric rings or furrows of the esophagus is an endoscopic finding traditionally ascribed to eosinophilic esophagitis.
Outcome measures
| Measure |
Esomeprazole
n=18 Participants
Proton pump inhibitor; Nexium 40mg capsule taken twice daily by mouth for 6 weeks for subjects with positive 24 hour pH study
|
Budesonide
n=28 Participants
Corticosteroid therapy; oral viscous Pulmicort Respules 1 gram taken by mouth orally twice daily (mixed with 1 gram packet of Sucralose \[Splenda-registered trademark\]) for 6 weeks in subjects with negative 24 hour pH studies (without GERD)
|
|---|---|---|
|
Participants With Presence of Esophageal Rings/Furrows at Six Month Endoscopy
|
11 participants
|
17 participants
|
SECONDARY outcome
Timeframe: Baseline, 6 monthsOutcome measures
| Measure |
Esomeprazole
n=18 Participants
Proton pump inhibitor; Nexium 40mg capsule taken twice daily by mouth for 6 weeks for subjects with positive 24 hour pH study
|
Budesonide
n=28 Participants
Corticosteroid therapy; oral viscous Pulmicort Respules 1 gram taken by mouth orally twice daily (mixed with 1 gram packet of Sucralose \[Splenda-registered trademark\]) for 6 weeks in subjects with negative 24 hour pH studies (without GERD)
|
|---|---|---|
|
Participants With Presence of Erosive Esophagitis at Six Month Endoscopy
|
1 participants
|
8 participants
|
Adverse Events
Esomeprazole
Budesonide
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Esomeprazole
n=18 participants at risk
Proton pump inhibitor; Nexium 40mg capsule taken twice daily by mouth for 6 weeks for subjects with positive 24 hour pH study
|
Budesonide
n=28 participants at risk
Corticosteroid therapy; oral viscous Pulmicort Respules 1 gram taken by mouth orally twice daily (mixed with 1 gram packet of Sucralose \[Splenda-registered trademark\]) for 6 weeks in subjects with negative 24 hour pH studies (without GERD)
|
|---|---|---|
|
Immune system disorders
Hives
|
5.6%
1/18 • Number of events 1 • Baseline to 6 months
|
3.6%
1/28 • Number of events 1 • Baseline to 6 months
|
Additional Information
Dr. Amy Foxx-Orenstein, D.O., Associate Prof. of Medicine, College of Medicine
Mayo Clinic
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place