Eosinophilic Esophagitis
Conditions
Keywords
Eosinophil, Reflux, Esophagus, Food allergy, Trouble Swallowing, Pediatrics
Brief summary
Eosinophilic Esophagitis (EoE) is a disorder in which a type of white blood cell (eosinophil) involved in allergic reactions, enter and cover the walls of the esophagus (food pipe). As a result, the esophagus becomes swollen and blocked making it difficult to swallow, and this leads to heartburn. In severe cases it can lead to food getting stuck in the esophagus and poor growth or weight loss in children The main purpose of this research study is to compare the effectiveness of two different types of treatment for EoE: 1. Oral viscous budesonide- which is a steroid medication that has been used to treat asthma. Recently, it has been used as a therapy to treat the eosinophilic inflammation in EoE, and 2. Specific food elimination. The study will also be looking to see if a blood test is useful in following the progression of EoE. Currently the only way to follow EoE is by repeating endoscopy, which is a more invasive test.
Detailed description
This study is a open label, randomized, prospective clinical trial comparing the efficacy of a topical steroid preparation to an antigen (food) elimination diet. A total of 40 participants will be recruited for this study that are positive for food allergens. Study participants will be randomly assigned to treatments as described below, and the duration of each treatment phase will be 16 weeks. The subjects will be randomized to group A and B. Subject and study team will NOT be blinded to the group assignments. Group A will receive oral viscous budesonide at a dose of 1 or 2 mg daily depending on the height. Group B will receive an elimination diet (elimination of the foods the subject is allergic to).
Interventions
DRUGOral Budesonide
This group will receive oral viscous budesonide at a dose of 1 or 2 mg depending on the height divided twice a day
OTHERElimination diet
This group will receive an elimination diet
Sponsors
Baylor College of Medicine
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
3 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
1. Signed written informed consent and assent if applicable prior to performing any study specific procedure. 2. Male or female subjects aged 3-17 years old. 3. Diagnosis of EoE within 2 months of enrollment (greater than or equal to 15 eosinophils per high powered field in both proximal or distal esophageal specimens). 4. Subjects who have failed at least a two month trial of proton pump inhibitor. 5. A female subject of childbearing potential who is or may become sexually active agrees to routinely use contraception from the time of signing informed consent and assent until 30 days from end of study. 6. Positive allergy testing on prick and/or patch testing.
Exclusion criteria
1. Subjects who are responsive to at least a two month trial of a proton pump inhibitor. 2. Diagnosis of Inflammatory Bowel Disease or static encephalopathy. 3. Prior abdominal surgery and other organ disorder not including atopic diseases. 4. Previous esophageal surgical procedure. 5. Previous esophageal congenital disorders such as tracheal esophageal fistula and esophageal atresia. 6. Positive for pregnancy. 7. Previous therapy within 6 weeks with oral or swallowed steroids or strict dietary elimination of major allergens 8. Presence of increased eosinophils in the stomach, small intestine, large intestine, and colon based on Debrosse et al.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Peak Number of Eosinophils/High Powered Field | 16 weeks | The primary outcome will be the peak eosinophil/high powered field on repeat biopsy after the treatment phase. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Quality of Life Survey Score | 16 weeks | PedsQL total survey score to assess overall quality of life before and after treatment |
| Symptom Score | 16 weeks | Assess the subject's symptoms using the validated tool termed Pediatric Eosinophilic Esophagitis (EoE) Symptom Severity Module by measuring % of normal bolus transit swallow and % of normal peristaltic esophageal body contractions. |
| Exploratory Studies | Conclusion of study | Proteomic studies search for certain proteins in the blood that may be linked to active EoE. Immune analysis will help identify better methods for diagnosis, treatment, and prevention of immune system changes leading to diseases. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Positive for Food Allergy: Group A Oral Budesonide
Oral Budesonide: This group will receive oral viscous budesonide at a dose of 1 or 2 mg depending on the height divided twice a day. | 2 |
| Positive for Food Allergy: Group B Elimination diet
Elimination diet: This group will receive an elimination diet | 1 |
| Total | 3 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Withdrawal by Subject | 2 | 1 |
Baseline characteristics
| Characteristic | Positive for Food Allergy: Group A | Positive for Food Allergy: Group B | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 2 Participants | 1 Participants | 3 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 0 Participants | 0 Participants | 0 Participants |
| EoE endoscopy score Patient 1 | 18 cells/high powered field | 0 cells/high powered field | 18 cells/high powered field |
| EoE endoscopy score Patient 2 | 0 cells/high powered field | 21 cells/high powered field | 21 cells/high powered field |
| EoE endoscopy score Patient 3 | 30 cells/high powered field | 0 cells/high powered field | 30 cells/high powered field |
| 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 | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 2 Participants | 1 Participants | 3 Participants |
| Race (NIH/OMB) White | 0 Participants | 0 Participants | 0 Participants |
| Region of Enrollment United States | 2 participants | 1 participants | 3 participants |
| Sex: Female, Male Female | 1 Participants | 0 Participants | 1 Participants |
| Sex: Female, Male Male | 1 Participants | 1 Participants | 2 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 0 | 0 / 0 |
| other Total, other adverse events | 0 / 0 | 0 / 0 |
| serious Total, serious adverse events | 0 / 0 | 0 / 0 |
Outcome results
Primary
Peak Number of Eosinophils/High Powered Field
The primary outcome will be the peak eosinophil/high powered field on repeat biopsy after the treatment phase.
Time frame: 16 weeks
Population: None of the enrolled participants had a repeat biopsy because they did not finish the trial due to undue stress and anxiety.
Secondary
Exploratory Studies
Proteomic studies search for certain proteins in the blood that may be linked to active EoE. Immune analysis will help identify better methods for diagnosis, treatment, and prevention of immune system changes leading to diseases.
Time frame: Conclusion of study
Population: Analysis was not performed.
Secondary
Quality of Life Survey Score
PedsQL total survey score to assess overall quality of life before and after treatment
Time frame: 16 weeks
Population: None of the enrolled participants had a PedQL because they did not finish the trial due to undue stress and anxiety.
Secondary
Symptom Score
Assess the subject's symptoms using the validated tool termed Pediatric Eosinophilic Esophagitis (EoE) Symptom Severity Module by measuring % of normal bolus transit swallow and % of normal peristaltic esophageal body contractions.
Time frame: 16 weeks
Population: Pediatric Eosinophilic Esophagitis (EoE) Symptom Severity Module was not completed because the subjects did not finish the trial due to undue stress and anxiety.