Clinical trial NCT02610816: Eosinophilic Esophagitis Intervention Trial-1 Food vs. 4 Food Elimination Diet Followed by Swallowed Glucocorticoids
Official title: Eosinophilic Esophagitis (EoE) Intervention Trial-Randomized 1 Food Elimination vs. 4 Food Elimination Diet Followed by Swallowed Glucocorticoids
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Eosinophilic Esophagitis
Eosinophilic Gastrointestinal Disorders (EGIDs)
Lead sponsor: Children's Hospital Medical Center, Cincinnati
Sponsor class: OTHER
Collaborators: name: Patient-Centered Outcomes Research Institute
First posted: 2015-11-20
Enrollment: 67
Sex: ALL
Minimum age (years): 6
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief summary: The purpose of this interventional study is to test and compare the effectiveness of two elimination diets-the 1-food elimination diet (1FED, milk only) and the 4-food elimination diet (4FED, milk, egg, wheat, and soy) for eosinophilic esophagitis (EoE). The study will also test the effectiveness of swallowed glucocorticoid therapy in some of the study participants for whom diet therapy was not effective.

Detailed description: This study will consist of two phases, plus a screening period. During the screening period, subject eligibility for the study will be determined. During Phase 1, qualified participants will be randomly assigned to one of two elimination diet therapies--the 1FED or the 4FED. Participants will remain on the assigned dietary therapy for 12 weeks. At the end of 12 weeks of therapy, esophageal biopsies from participant's standard of care (i.e. normal, routine care) endoscopy will be evaluated to determine disease status. Participants whose EoE is in remission (i.e. \<15 eos/hpf) will be done with the study.

Participants whose EoE is still active (i.e. ≥15 eos/hpf) will continue into Phase 2 of the study. During Phase 2, participants who were on 1FED in Phase 1 will receive 4FED therapy for 12 weeks, and participants who were on 4FED during Phase 1 will receive swallowed glucocorticoid therapy for 12 weeks. At the end of 12 weeks of therapy, esophageal biopsies from participant's standard of care (i.e. normal, routine care) endoscopy will be evaluated to determine disease status.

Eligibility criteria: Inclusion Criteria:

* Have diagnosis of EoE (based on consensus criteria)
* Are aged 6 to 17 years
* Have histologically confirmed active disease \>15 eosinophils/hpf in either distal or proximal esophagus within 12 weeks of screening visit
* Proton Pump Inhibitor (PPI) confirmation
* Symptomatic (have experienced symptoms within the last month prior to enrollment)
* Has a negative urine pregnancy test at screening if of childbearing potential. Females of childbearing potential must have a negative urine pregnancy test (β-hCG) prior to enrollment into the study (i.e., at screening). Subsequently, these participants must agree to use adequate birth control measures (e.g., condom, oral/injectable/subcutaneous contraceptives, intrauterine device, or sexual abstinence) during the study and for at least one month after the last dose of study drug which will be documented in the source documents.

Exclusion Criteria:

* Have been treated with topical swallowed steroids within the last 2 months or systemic steroids within the past 3 months
* Have eosinophilia in segments of the GI tract other than the esophagus
* Have been diagnosed with a GI malabsorption disorder (i.e., Inflammatory bowel disease, Crohn's disease) or Celiac disease
* Are currently on dietary therapy avoiding milk
* Have concurrent H pylori gastritis or parasitic infection
* Are unable to obtain esophagogastroduodenoscopy with esophageal biopsies at Cincinnati Children's Hospital Medical Center (CCHMC) or other participating institution within 4 weeks of study completion
* Have previously failed (in a clinical trial setting) dietary therapy with one of these regimens or topical steroid treatment with fluticasone at a total dose of 1760 mcg per day.
* Have definitely responded (in a clinical trial setting) to either dietary therapy avoiding these antigens or to swallowed fluticasone at a total dose of 1760 mcg per day
* Are concurrently receiving any of the prohibited medications listed in Table 2
* On immunotherapy for pollen (if not on maintenance therapy) or immunoglobulin E (IgE)-mediated food allergy.

Primary outcomes:
measure: Change From Baseline in Pediatric EoE Symptom Score Version 2.0 (PEESS V2.0) at 12 Weeks
timeFrame: Baseline and 12 weeks
measure: Within-group Comparisons (Baseline v. Week 12) of PEESS V2.0 Scores
timeFrame: Baseline and 12 weeks
Secondary outcomes:
measure: Percent of Participants in Histologic Remission (<15 Eosinophils Per High Power Field) at 12 Weeks
timeFrame: 12 weeks
measure: Percent of Participants on Swallowed Glucocorticoids (SGC) in Histologic Remission (<15 Eos/Hpf) at 12 Weeks in Phase 2
timeFrame: 12 weeks
measure: Percent of 1FED Non-responders on 4FED in Histologic Remission (<15 Eos/Hpf) at 12 Weeks in Phase 2
timeFrame: 12 weeks
measure: Change From Baseline in Pediatric Quality of Life Inventory Version 3.0 EoE Module (PedsQL 3.0 EoE) at 12 Weeks
timeFrame: Baseline and 12 weeks
measure: Change From Baseline in Pediatric Quality of Life Inventory Version 4.0 (PedsQL 4.0) Generic Core Scales at 12 Weeks
timeFrame: Baseline and 12 weeks
measure: Change From Baseline in Endoscopic Reference Score at 12 Weeks
timeFrame: Baseline and 12 weeks
measure: Percent of Participants With Positive and Negative Milk Skin Prick Tests Responding to 1FED
timeFrame: Baseline and 12 weeks
Other outcomes:
Locations:
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital Medical Center
state: Ohio
Study officials:
affiliation: Children's Hospital Medical Center, Cincinnati
name: Marc E Rothenberg, MD, PhD
role: PRINCIPAL_INVESTIGATOR
References: