Risk Factors and Biomarkers for Diagnosis and Treatment of EoE

• ClinicalTrials.gov Identifier: NCT01988285
• Recruitment Status: Enrolling by invitation
• First Posted: November 20, 2013
• Last Update Posted: March 22, 2023
• Sponsor: University of North Carolina, Chapel Hill
• Collaborator: National Institutes of Health (NIH)
• Information provided by (Responsible Party): University of North Carolina, Chapel Hill

Study Description

Brief Summary:

Purpose: To validate our previously developed model of risk factors for diagnosis of eosinophilic esophagitis (EoE), and to assess the utility of serum levels of biomarkers in diagnosis and monitoring of treatment of EoE. Participants: Up to a total of 800 participants (approximately 400 with EoE and 400 without EoE) who are referred for upper endoscopy. Procedures: This is a prospective cohort study. Patients will be compared with newly diagnosed EoE to non-EoE controls, obtain baseline measures, reassess EoE patients after treatment, and also reassess untreated controls. Data collection will include questionnaire results, blood collection, and tissue collection (esophageal biopsies performed during upper endoscopy per pre-specified protocol). Blood will be analyzed for the presence of the candidate biomarkers. Patients will be compared with newly diagnosed EoE to non-EoE controls, obtain baseline measures, reassess EoE patients after treatment, and also reassess untreated controls. Data collection will include questionnaire results, blood collection, possible saliva collection, possible urine collection, and tissue collection (esophageal biopsies performed during upper endoscopy per pre-specified protocol). Blood will be analyzed for the presence of the candidate biomarkers, including eotaxin-3 and IL-13.

Condition or disease	Intervention/treatment
Eosinophilic Esophagitis	Other: Specimen Collection; Other: Questionnaires

Study Design

• Study Type: Observational
• Estimated Enrollment: 800 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Risk Factors and Biomarkers for Diagnosis and Treatment of EoE
• Study Start Date: July 2011
• Estimated Primary Completion Date: August 2025
• Estimated Study Completion Date: August 2025

Groups and Cohorts

Group/Cohort	Intervention/treatment
Dysphagia and GERD controls; The cross-sectional arm will consist of patients who are having a clinically indicated endoscopy for reflux and/or dysphagia. Cross-sectional participants will have specimens collected and complete a questionnaire.	Other: Specimen Collection; Blood collection, possible saliva collection, possible urine collection, and biopsy collection; Other: Questionnaires; Subjects will complete a questionnaire utilizing validated symptom-specific instruments (Mayo Dysphagia Scale; GERD-HRQL Scale). The questionnaire will also record patient demographics, medical history, and current and past medication use
Prospective Longitudinal EoE Cases; The prospective longitudinal group will be subjects who have a positive EoE diagnosis during initial endoscopy. This prospective group will be followed up at their clinically indicated endoscopy after their standard of care clinical therapy of swallowed steroids. Prospective longitudinal participants will have specimens collected and complete questionnaires s prior to their standard of care clinical therapy of swallowed steroids and at their clinically indicated follow up upper endoscopy.	Other: Specimen Collection; Blood collection, possible saliva collection, possible urine collection, and biopsy collection; Other: Questionnaires; Subjects will complete a questionnaire utilizing validated symptom-specific instruments (Mayo Dysphagia Scale; GERD-HRQL Scale). The questionnaire will also record patient demographics, medical history, and current and past medication use

Outcome Measures

Primary Outcome Measures :

1. Risk factors in EoE diagnosis [ Time Frame: Enrollment (day 1) ]
   To validate our model of clinical, endoscopic, and histopathologic risk factors for diagnosis of EoE by calculating the EoE risk score which includes subject's age, allergic conditions, endoscopic findings, and eosinophil count. Subject's EoE risk scores will be compared between EoE patients and two non-EoE control groups: patients with dysphagia and patients with gastroesophageal reflux disease (GERD) who are not responsive to proton pump inhibitor (PPI) therapy.

Secondary Outcome Measures :

1. Inflammatory serum biomarker measures [ Time Frame: Enrollment (day 1) ]
   To assess operating characteristics of serum biomarkers eotaxin-3 and IL-13 for diagnosis of incident cases of EoE as compared with two non-EoE control groups: patients with dysphagia and patients with GERD who are not responsive to PPI therapy.

Other Outcome Measures:

1. Serum biomarker change following treatment in EoE cases [ Time Frame: Enrollment (Day 1) and approximately 8 weeks after initial EGD ]
   To determine the effect of clinically indicated topical steroid therapy on non-invasive serum biomarkers eotaxin-3 and IL-13 for monitoring treatment response in incident EoE cases.

Biospecimen Retention:   Samples With DNA

Esophageal, gastric, and duodenal biopsies, blood samples, saliva, urine.

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No
• Sampling Method: Non-Probability Sample

Study Population

The source of the study population will be patients aged 18 or older presenting at the gastrointestinal (Gl) Endoscopy Clinic at UNC-Chapel Hill for elective upper endoscopy with dysphagia and/or reflux symptoms.

Criteria

Inclusion Criteria:

1. Referral for upper endoscopy (esophagogastroduodenoscopy; EGD) to evaluate specific symptoms (dysphagia, heartburn, reflux, chest pain, regurgitation, abdominal pain, nausea, or vomiting).
2. Age 18 or older

Exclusion Criteria:

1. Acute GI bleeding.
2. Known cancer of the esophagus, stomach, or small bowel.
3. Prior esophageal resection.
4. Esophageal varices.
5. Active anticoagulation or bleeding diathesis.
6. Medical instability (determined by the performing endoscopist) to precludes performing EGD.
7. Pregnancy
8. Inability to read or understand the consent form and questionnaire.

Contacts and Locations

Locations

United States, North Carolina

University of North Carolina, Chapel Hill

Chapel Hill, North Carolina, United States, 27599

Sponsors and Collaborators

University of North Carolina, Chapel Hill

National Institutes of Health (NIH)

Investigators

• Principal Investigator: Evan S Dellon, MD, MPH; University of North Carolina, Chapel Hill

More Information

• Responsible Party: University of North Carolina, Chapel Hill
• ClinicalTrials.gov Identifier: NCT01988285
• Other Study ID Numbers: 11-1122
• First Posted: November 20, 2013
• Last Update Posted: March 22, 2023
• Last Verified: March 2023

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

Keywords provided by University of North Carolina, Chapel Hill:

Esophagitis; Eosinophilic Esophagitis; Esophageal Diseases; Gastrointestinal Diseases; Digestive System Diseases

Additional relevant MeSH terms:

Eosinophilic Esophagitis; Esophagitis; Esophageal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Gastroenteritis; Eosinophilia; Leukocyte Disorders; Hematologic Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases