Corticosteroid Effect on Achalasia Variant EGJOO

NCT06588348 | Active Not Recruiting Early Phase 1 FDA Drug

• 1 other identifier: 022-001 Steroid Study
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

EGJOO is a disorder in which the muscles of the esophagus (swallowing tube) do not function in a coordinated fashion so that swallowed material does not pass easily into the stomach. EGJOO often causes symptoms of swallowing difficulties and chest pain. The cause of EGJOO and its optimal treatment are not clear. The investigators research team suspects that EGJOO might be caused by an allergy that involves the esophagus, and that treatment with medications called corticosteroids might improve function of the esophageal muscles. The purpose of this study is to learn how corticosteroid therapy affects the muscles of the esophagus in patients suffering with EGJOO.

Conditions

Esophagogastric Junction Outflow Obstruction

Keywords

EGJOO; Corticosteroid

Outcome Measures

Primary Outcomes (1)

• Response in esophageal motility to study intervention

  Median integrated relaxation pressure (IRP) of the lower esophageal sphincter (LES) obtained through esophageal manometry procedure

  Assessed upon being on study intervention medication for 14 days

Study Arms (1)

Steroid Treatment

EXPERIMENTAL

All participants are in the treatment arm. Patients with Esophagogastric Junction Outflow Obstruction (EGJOO) will undergo steroid treatment for 14 days. On the last day of the 14 day treatment, patient will undergo esophageal manometry testing to assess for treatment response. They will also be asked to complete follow-up Eckardt and BEDQ surveys.

Drug: Steroid treatment; Procedure: Esophageal Manometry; Other: Survey

Interventions

Steroid treatment DRUG

All enrolled subjects will be prescribed a 14-day course of corticosteroid therapy in the form of Prednisone 20 mg to be taken once daily by mouth.

Also known as: Prednisone

Steroid Treatment

Esophageal Manometry PROCEDURE

On the final day of Prednisone use, all subjects will undergo esophageal manometry testing to assess for treatment response.

Steroid Treatment

Survey OTHER

All enrolled subjects will be asked to complete follow-up Eckardt and BEDQ surveys.

Also known as: Eckardt and Brief Esophageal Dysphagia Questionnaire (BEDQ)

Steroid Treatment

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• EGJOO manometric diagnosis based on CC v4.0 criteria
• Primary symptom of dysphagia and/or non-cardiac chest pain
• Additional objective evidence of obstruction on Timed Barium Esophagram and/or EndoFLIP
• The treating physician has determined that invasive therapy (botulinum toxin injection, pneumatic dilation, POEM, or Heller myotomy) is indicated.

You may not qualify if:

• History of prior foregut surgery
• History of esophageal botulinum toxin injection within 6 months of study enrollment
• Presence of hiatal hernia \> 2 centimeters
• Presence of esophageal mass
• Obstructing esophageal stricture or ring on endoscopy
• Reflux esophagitis (LA Grades B-D)
• Subjects on current immunosuppression or immune modulating therapy
• Chronic opioid use
• Previously diagnosed extrinsic compression of the gastroesophageal junction
• Concomitant Eosinophilic Esophagitis with uncontrolled mucosal disease who have not tried at least one standard therapy
• Contraindication to the use of oral corticosteroids
• History and/or current diagnosis of Diabetes

Sponsors & Collaborators

• Baylor Research Institute: lead

Study Sites (1)

Baylor University Medical Center

Dallas, Texas, 75246, United States

Location

Related Publications (10)

• Yadlapati R, Kahrilas PJ, Fox MR, Bredenoord AJ, Prakash Gyawali C, Roman S, Babaei A, Mittal RK, Rommel N, Savarino E, Sifrim D, Smout A, Vaezi MF, Zerbib F, Akiyama J, Bhatia S, Bor S, Carlson DA, Chen JW, Cisternas D, Cock C, Coss-Adame E, de Bortoli N, Defilippi C, Fass R, Ghoshal UC, Gonlachanvit S, Hani A, Hebbard GS, Wook Jung K, Katz P, Katzka DA, Khan A, Kohn GP, Lazarescu A, Lengliner J, Mittal SK, Omari T, Park MI, Penagini R, Pohl D, Richter JE, Serra J, Sweis R, Tack J, Tatum RP, Tutuian R, Vela MF, Wong RK, Wu JC, Xiao Y, Pandolfino JE. Esophageal motility disorders on high-resolution manometry: Chicago classification version 4.0(c). Neurogastroenterol Motil. 2021 Jan;33(1):e14058. doi: 10.1111/nmo.14058.

  PMID: 33373111 BACKGROUND

• Richter JE, Clayton SB. Diagnosis and Management of Esophagogastric Junction Outflow Obstruction. Am J Gastroenterol. 2019 Apr;114(4):544-547. doi: 10.14309/ajg.0000000000000100. No abstract available.

  PMID: 30848733 BACKGROUND

• Sadowski DC, Ackah F, Jiang B, Svenson LW. Achalasia: incidence, prevalence and survival. A population-based study. Neurogastroenterol Motil. 2010 Sep;22(9):e256-61. doi: 10.1111/j.1365-2982.2010.01511.x. Epub 2010 May 11.

  PMID: 20465592 BACKGROUND

• Enestvedt BK, Williams JL, Sonnenberg A. Epidemiology and practice patterns of achalasia in a large multi-centre database. Aliment Pharmacol Ther. 2011 Jun;33(11):1209-14. doi: 10.1111/j.1365-2036.2011.04655.x. Epub 2011 Apr 11.

  PMID: 21480936 BACKGROUND

• Okeke FC, Raja S, Lynch KL, Dhalla S, Nandwani M, Stein EM, Chander Roland B, Khashab MA, Saxena P, Kumbhari V, Ahuja NK, Clarke JO. What is the clinical significance of esophagogastric junction outflow obstruction? evaluation of 60 patients at a tertiary referral center. Neurogastroenterol Motil. 2017 Jun;29(6). doi: 10.1111/nmo.13061. Epub 2017 Apr 9.

  PMID: 28393437 BACKGROUND

• Schupack D, Katzka DA, Geno DM, Ravi K. The clinical significance of esophagogastric junction outflow obstruction and hypercontractile esophagus in high resolution esophageal manometry. Neurogastroenterol Motil. 2017 Oct;29(10):1-9. doi: 10.1111/nmo.13105. Epub 2017 May 23.

  PMID: 28544670 BACKGROUND

• Clayton SB, Patel R, Richter JE. Functional and Anatomic Esophagogastic Junction Outflow Obstruction: Manometry, Timed Barium Esophagram Findings, and Treatment Outcomes. Clin Gastroenterol Hepatol. 2016 Jun;14(6):907-911. doi: 10.1016/j.cgh.2015.12.041. Epub 2016 Jan 12.

  PMID: 26792374 BACKGROUND

• Samo S, Qayed E. Esophagogastric junction outflow obstruction: Where are we now in diagnosis and management? World J Gastroenterol. 2019 Jan 28;25(4):411-417. doi: 10.3748/wjg.v25.i4.411.

  PMID: 30700938 BACKGROUND

• Vaezi MF, Pandolfino JE, Yadlapati RH, Greer KB, Kavitt RT. ACG Clinical Guidelines: Diagnosis and Management of Achalasia. Am J Gastroenterol. 2020 Sep;115(9):1393-1411. doi: 10.14309/ajg.0000000000000731.

  PMID: 32773454 BACKGROUND

• Nelson M, Zhang X, Genta RM, Turner K, Podgaetz E, Paris S, Cardenas J, Gu J, Leeds S, Ward M, Nguyen A, Konda V, Furuta GT, Pan Z, Souza RF, Spechler SJ. Lower esophageal sphincter muscle of patients with achalasia exhibits profound mast cell degranulation. Neurogastroenterol Motil. 2021 May;33(5):e14055. doi: 10.1111/nmo.14055. Epub 2020 Dec 6.

  PMID: 33280206 BACKGROUND

MeSH Terms

Interventions

Prednisone; Surveys and Questionnaires

Intervention Hierarchy (Ancestors)

Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Data Collection; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Officials

• Chanakyaram Reddy, MD

  Baylor Health Care System

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NA
• Masking: NONE
• Purpose: BASIC SCIENCE
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principle Investigator

Study Record Dates

• First Submitted: August 23, 2024
• First Posted: September 19, 2024
• Study Start: July 26, 2022
• Primary Completion (Estimated): November 1, 2026
• Study Completion (Estimated): November 1, 2026
• Last Updated: January 28, 2026

Record last verified: 2026-01

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)