A Study in Adolescents and Adults With Eosinophilic Esophagitis (EoE) Measuring Histologic Response and Determine if Reduction in Dysphagia is Achieved
Oral Budesonide Suspension (OBS) in Adolescent and Adult Subjects (11 to 55 Years of Age, Inclusive) With Eosinophilic Esophagitis: A Phase 3 Randomized, Double-blind, Placebo-controlled Study
1 other identifier
interventional
318
1 country
77
Brief Summary
A study in adolescents and adults with eosinophilic esophagitis (EoE) to measure the histologic response and determine if any reduction in dysphagia is achieved.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_3
Started Dec 2015
Typical duration for phase_3
77 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
November 4, 2015
CompletedFirst Posted
Study publicly available on registry
November 16, 2015
CompletedStudy Start
First participant enrolled
December 7, 2015
CompletedPrimary Completion
Last participant's last visit for primary outcome
January 24, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
February 15, 2019
CompletedResults Posted
Study results publicly available
March 16, 2020
CompletedFebruary 19, 2025
May 1, 2021
3.1 years
November 4, 2015
January 22, 2020
January 30, 2025
Conditions
Outcome Measures
Primary Outcomes (2)
Number of Participants With Histologic Response at the Final Treatment Period Evaluation (Week 16)
Histologic response was defined as a peak eosinophil count of less than or equal to (\<=) 6/ high-powered field (HPF) across all available esophageal levels at final treatment period evaluation (Week 16). Histologic response after 12 weeks of double blind treatment at Week 16 was reported.
Week 16
Number of Participants With Dysphagia Symptom Response at the Final Treatment Period Evaluation (Week 16)
Dysphagia symptom response was defined as greater than or equal to (\>=) 30 percent (%) reduction in the Dysphagia Symptom Questionnaire (DSQ) combined score (questions 2+3). DSQ contained 4 questions, all participants used a diary, and responded to Questions 1 (did you eat solid food) and 2 (did food pass slowly or get stuck). If the participant's answer to Question 2 was 'No', the diary ended for that day. If a participant answered 'Yes', he/she advanced to Questions 3 (did you have to do anything to make the food go down or get relief) and 4 (extent to which the participant experienced pain while swallowing). DSQ score= (\[sum of points from questions 2+3 in the daily DSQ\]Ă—14)/ Number of diaries reported with non-missing data. Dysphagia symptom response after 12 weeks of double blind treatment at Week 16 was reported.
Week 16
Secondary Outcomes (18)
Change From Baseline in Dysphagia Symptom Questionnaire (DSQ) Combined Score at the Final Treatment Period Evaluation (Week 16)
Baseline, Week 16
Change From Baseline in Total Endoscopy Score at the Final Treatment Period Evaluation (Week 16)
Baseline, Week 16
Number of Participants With Peak Eosinophil Count Less Than (<)15/High-Powered Field (HPF) or Less Than or Equal to (<=)1/High-Powered Field (HPF) at the Final Treatment Period Evaluation (Week 16)
Week 16
Change From Baseline in the Peak Eosinophil Count at the Final Treatment Period Evaluation (Week 16)
Baseline, Week 16
Change From Baseline in the Histopathologic Epithelial Features Combined Total Score Ratio (TSR) at the Final Treatment Period Evaluation (Week 16)
Baseline, Week 16
- +13 more secondary outcomes
Study Arms (2)
Oral Budesonide Suspension (OBS)
EXPERIMENTALParticipants will receive Oral Budesonide Suspension (OBS) 10 milliliter (ml) of 0.2 milligram per milliliter (mg/ml) twice daily up to 16 weeks.
Placebo
PLACEBO COMPARATORParticipants will receive oral dose of 10 ml of placebo matched with the experimental drug twice daily up to 16 weeks.
Interventions
Oral Budesonide Suspension (OBS) 10 milliliter (ml) of 0.2 milligram per milliliter (mg/ml) twice daily up to 16 weeks.
Eligibility Criteria
You may qualify if:
- Participants is able to provide written informed consent (participant, parent or legal guardian, and, as appropriate, participant assent) to participate in the study before completing any study-related procedures.
- Participant is male or female aged 11-55 years, inclusive, at time of consent.
- Participant has histologic evidence of eosinophilic esophagitis (EoE) with a peak eosinophil count of greater than or equal to (\>=) 15/ high-powered field (HPF), from 2 of 3 (proximal, mid-, and/or distal) levels of the esophagus at the screening endoscopy.
- Participant has a history of clinical symptoms of esophageal dysfunction (for example, eating problems, abdominal pain, heartburn, dysphagia, vomiting, food impaction, weight loss) intermittently or continuously at screening (Visit -1).
- Participants must have experienced dysphagia (response of "yes" to question 2 on Dysphagia Symptom Questionnaire \[DSQ\]) on a minimum of 4 days and completed the DSQ on \>= 70 percent (%) of days in any 2 consecutive weeks of the screening period and in the last 2 weeks prior to the baseline visit (Visit 1).
- Participant must not have PPI-responsive EoE based on esophageal biopsies performed after the patient has been on at least 8 weeks of high-dose PPI therapy (high-dose therapy refers to the total daily dose, which may have been administered as a once or twice daily dosing regimen). This may occur at the time of the qualifying esophagogastroduodenoscopy (EGD) (in which case the same proton pump inhibitor (PPI) regimen must be continued), or this may have been done previously (in which case PPI therapy may have been stopped if there was no response to therapy based on esophageal biopsy results). If PPI responsiveness was excluded by a previous EGD and biopsy, the historical EGD and biopsy must have been performed after the patient had been on a minimum of 6 weeks of high-dose PPI therapy.
- Participant will be on a stable (no changes) diet \>=3 months prior to the screening visit (Visit -1).
- Participant is willing and able to continue any dietary therapy, environmental therapy, and/or medical regimens (including gastric acid suppression) in effect at the screening visit (Visit -1). There should be no change to these regimens during study participation.
- All female participants must have a negative serum pregnancy test (beta-human chorionic gonadotropin \[β-hCG\]) prior to enrollment into the study. Females of childbearing potential must agree to continue acceptable birth control measures (for example, abstinence, stable oral contraceptives, or double-barrier methods) throughout study participation.
- Participant is willing and has an understanding and ability to fully comply with study procedures and restrictions defined in this protocol.
You may not qualify if:
- Participant has any condition or abnormality (including laboratory abnormalities), current or past, that, in the opinion of the principal investigator or medical monitor, would compromise the safety of the participant or interfere with or complicate the assessment of signs or symptoms of EoE. Such conditions may include psychiatric problems; neurologic deficits or disease; developmental delay; cardiovascular, metabolic, or pulmonary disease; or previous gastroesophageal surgery. These should be discussed with the medical monitor.
- Participant has used immunomodulatory therapy within 8 weeks prior to the qualifying EGD or between the qualifying EGD and baseline visit (Visit 1) or anticipates using immunomodulatory therapy during the treatment period (except for any ongoing regimen of allergy shots). Use of long-acting immunomodulatory therapy (for example, Rituxan) within 3 months of the qualifying EGD should be reviewed with the medical monitor.
- Participant has been using swallowed topical corticosteroid for EoE or systemic corticosteroid for any condition within the 4 weeks prior to the qualifying EGD, between the qualifying EGD and baseline visit (Visit 1), or anticipates use during the treatment period; any temporary use (less than or equal to \[\<=\]7 days) or initiation of new steroid treatment during the study should be documented and discussed with the medical monitor prospectively but cannot occur within 4 weeks of the final EGD.
- Participant has been on inhaled steroids and has not been on stable treatment for \>=3 months prior to screening visit (Visit -1). Participants on inhaled steroids need to stay on a stable treatment during study participation. Participant has been on intranasal steroids and has not been on stable treatment for a minimum of 4 weeks prior to the qualifying EGD. After the qualifying EGD, participants with seasonal allergic rhinitis may resume (or discontinue) intranasal corticosteroids based on the participant's usual treatment regimen for allergy season.
- Participant has initiated, discontinued, or changed dosage regimen of PPIs, H2 antagonists, antacids, or leukotriene inhibitors for any condition (such as gastroesophageal reflux disease, asthma or allergic rhinitis) within the 4 weeks prior to the qualifying EGD, between the qualifying EGD and baseline visit (Visit 1), or anticipates changes in the use of such medications during the treatment period.
- Participant has been using cytochrome P450 3A4 (CYP450 3A4) inhibitors (for example, ketoconazole, grapefruit juice) within the 2 weeks prior to the baseline visit (Visit 1) or within 5 half-lives (whichever is greater) or anticipates using such medications during the treatment period.
- Participant has an appearance on qualifying EGD of an esophageal stricture (high-grade), as defined by the presence of a lesion that does not allow passage of a diagnostic adult upper endoscope (for example, with an insertion tube diameter of greater than \[\>\]9 millimeter \[mm\]).
- Participant is on a pure liquid diet or the 6-food elimination diet.
- Participant has had an esophageal dilation within the 3 months prior to screening (Visit -1).
- Participant has presence of esophageal varices at the screening endoscopy.
- Participant has any current disease of the gastrointestinal tract, aside from EoE, including eosinophilic gastritis, enteritis, colitis, or proctitis; inflammatory bowel disease; or celiac disease.
- Participant has other diseases causing or associated with EoE, including hypereosinophilic syndrome, collagen vascular disease, vasculitis, achalasia, or parasitic infection.
- Participant has current evidence of oropharyngeal or esophageal candidiasis.
- Participant has a potentially serious acute or chronic viral infection or immunodeficiency condition, including tuberculosis, fungal, bacterial, viral/parasite infection, ocular herpes simplex, herpes esophagitis, or chicken pox/measles.
- Participant has upper gastrointestinal bleeding within 4 weeks prior to the screening visit (Visit - 1) or between the screening visit and baseline visit (Visit 1).
- +9 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Shirelead
Study Sites (77)
Children's Hospital
Birmingham, Alabama, 35233, United States
Phoenix Childrens Hospital
Phoenix, Arizona, 85016, United States
Del Sol Research Management
Tucson, Arizona, 85710, United States
Adobe Clinical Research LLC
Tucson, Arizona, 85712, United States
Arkansas Gastroenterology
North Little Rock, Arkansas, 72117, United States
GW Research, Inc.
Chula Vista, California, 91910, United States
Rady Children's Hospital San Diego
San Diego, California, 92123, United States
Colorado Children's Hospital
Aurora, Colorado, 80045, United States
Asthma and Allergy Associates PC
Colorado Springs, Colorado, 80907, United States
Rocky Mountain Pediatric Gastroenterology
Lone Tree, Colorado, 80124, United States
Rocky Mountain Clinical Research LLC
Wheat Ridge, Colorado, 80033, United States
Connecticut Clinical Research Foundation
Bristol, Connecticut, 06010, United States
Connecticut GI, PC - Research Division
Farmington, Connecticut, 06032, United States
Connecticut Children's Medical Center
Hartford, Connecticut, 06106, United States
Nature Coast Clinical Research LLC
Inverness, Florida, 34452, United States
Borland Groover Clinic
Jacksonville, Florida, 32256, United States
Arnold Palmer Hospital For Children
Orlando, Florida, 32806, United States
Accord Clinical Research LLC
Port Orange, Florida, 32129, United States
Children's Center for Digestive Health Care
Atlanta, Georgia, 30342, United States
Gastroenterology Associates of Central Georgia, LLC
Macon, Georgia, 31201, United States
Gastrointestinal Specialists of Georgia
Marietta, Georgia, 30060, United States
Grand Teton Research Group, PLLC
Idaho Falls, Idaho, 83404, United States
Ann and Robert H Lurie Childrens Hospital of Chicago
Chicago, Illinois, 60611, United States
Northwestern University
Chicago, Illinois, 60611, United States
Center for Children's Digestive Health
Park Ridge, Illinois, 60068, United States
University of Illinois College of Medicine at Peoria Pediatric Subspecialty Clinic
Peoria, Illinois, 61603, United States
OSF St Francis Medical Center
Peoria, Illinois, 61637, United States
Indiana University
Indianapolis, Indiana, 46202, United States
Gastroenterology of Southern Indiana
New Albany, Indiana, 47150, United States
University of Iowa Hospitals and Clinics
Iowa City, Iowa, 52242, United States
Cotton O'Neil Clinical Research Center
Topeka, Kansas, 66606, United States
Gastroenterology Associates LLC
Baton Rouge, Louisiana, 70809, United States
Clinical Trials Management LLC
Metairie, Louisiana, 70006, United States
Louisiana Research Center LLC
Shreveport, Louisiana, 71105, United States
Clinical Trials of America LA LLC - PPDS
West Monroe, Louisiana, 71291, United States
Tufts Medical Center
Boston, Massachusetts, 00211, United States
Boston Children's Hospital
Boston, Massachusetts, 02115, United States
Brigham and Womens Hospital
Boston, Massachusetts, 02115, United States
University of Michigan
Ann Arbor, Michigan, 48109, United States
Clinical Research Institute of Michigan
Chesterfield, Michigan, 48047, United States
West Michigan Clinical Research
Wyoming, Michigan, 49519, United States
University of Minnesota
Minneapolis, Minnesota, 55454, United States
Minnesota Gastroenterology PA
Plymouth, Minnesota, 55446, United States
Mayo Clinic
Rochester, Minnesota, 55905, United States
Bozeman Health Deaconess Hospital
Bozeman, Montana, 59718, United States
South Jersey Gastroenterology
Marlton, New Jersey, 08053, United States
Long Island Gastrointestinal Research Group LLP
Great Neck, New York, 11023, United States
Mount Sinai Hospital
New York, New York, 10029, United States
Asheville Gastroenterology Associates PA
Asheville, North Carolina, 28801, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27514, United States
Clinical Research of Charlotte
Charlotte, North Carolina, 28277, United States
Clinical Trials of America-NC, LLC - PPDS
Mount Airy, North Carolina, 27030, United States
Consultants For Clinical Research Inc
Cincinnati, Ohio, 45219, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
University of Cincinnati
Cincinnati, Ohio, 45267, United States
Cleveland Clinic
Cleveland, Ohio, 44195, United States
Gastrointestinal and Liver Diseases Consultants PC
Dayton, Ohio, 45415, United States
Great Lakes Gastroenterology
Mentor, Ohio, 44060, United States
Digestive Disease Specialists, Inc.
Oklahoma City, Oklahoma, 73112, United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104, United States
University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
Greenville Hospital System
Greenville, South Carolina, 29605, United States
Greenville Hospital System
Greenville, South Carolina, 29615, United States
Gastro One
Germantown, Tennessee, 38138, United States
Vanderbilt University Medical Center
Nashville, Tennessee, 37212, United States
San Antonio Military Medical Center
Fort Sam Houston, Texas, 78234, United States
Baylor College of Medicine
Houston, Texas, 77030, United States
Houston Endoscopy and Research Center
Houston, Texas, 77079, United States
Digestive Health Center
Pasadena, Texas, 77505, United States
Texas Digestive Disease Consultants
Southlake, Texas, 76092, United States
Advanced Research Institute
Ogden, Utah, 84092, United States
Primary Children's Hospital, University of Utah
Salt Lake City, Utah, 84113, United States
Advanced Research Institute
Sandy City, Utah, 84092, United States
Emeritas Research Group
Lansdowne Town Center, Virginia, 20176, United States
Blue Ridge Medical Research
Lynchburg, Virginia, 24502, United States
Carilion Clinic
Roanoke, Virginia, 24013, United States
University of Wisconsin
Madison, Wisconsin, 53792, United States
Related Publications (5)
Dellon ES, Collins MH, Katzka DA, Mukkada VA, Falk GW, Zhang W, Goodwin B, Terreri B, Boules M, Desai NK, Hirano I. Effect of randomized treatment withdrawal of budesonide oral suspension on clinically relevant efficacy outcomes in patients with eosinophilic esophagitis: a post hoc analysis. Therap Adv Gastroenterol. 2024 Dec 23;17:17562848241307602. doi: 10.1177/17562848241307602. eCollection 2024.
PMID: 39735351DERIVEDHirano I, Collins MH, Katzka DA, Mukkada VA, Falk GW, Terreri B, Boules M, Zhang W, Desai NK, Dellon ES. Effect of Esophageal Dilation History on Efficacy Outcomes in Patients With Eosinophilic Esophagitis Receiving Budesonide Oral Suspension. Am J Gastroenterol. 2024 Nov 12;120(7):1502-1510. doi: 10.14309/ajg.0000000000003197.
PMID: 39631042DERIVEDMukkada VA, Gupta SK, Gold BD, Dellon ES, Collins MH, Katzka DA, Falk GW, Williams J, Zhang W, Boules M, Hirano I, Desai NK. Pooled Phase 2 and 3 Efficacy and Safety Data on Budesonide Oral Suspension in Adolescents with Eosinophilic Esophagitis. J Pediatr Gastroenterol Nutr. 2023 Dec 1;77(6):760-768. doi: 10.1097/MPG.0000000000003948. Epub 2023 Sep 18.
PMID: 37718471DERIVEDDellon ES, Collins MH, Katzka DA, Mukkada VA, Falk GW, Morey R, Goodwin B, Eisner JD, Lan L, Desai NK, Williams J, Hirano I; ORBIT2/SHP621-302 Investigators. Long-Term Treatment of Eosinophilic Esophagitis With Budesonide Oral Suspension. Clin Gastroenterol Hepatol. 2022 Jul;20(7):1488-1498.e11. doi: 10.1016/j.cgh.2021.06.020. Epub 2021 Jun 26.
PMID: 34182150DERIVEDHirano I, Collins MH, Katzka DA, Mukkada VA, Falk GW, Morey R, Desai NK, Lan L, Williams J, Dellon ES; ORBIT1/SHP621-301 Investigators. Budesonide Oral Suspension Improves Outcomes in Patients With Eosinophilic Esophagitis: Results from a Phase 3 Trial. Clin Gastroenterol Hepatol. 2022 Mar;20(3):525-534.e10. doi: 10.1016/j.cgh.2021.04.022. Epub 2021 Apr 19.
PMID: 33887475DERIVED
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Results Point of Contact
- Title
- Study Director
- Organization
- Shire
Study Officials
- STUDY DIRECTOR
Study Director
Takeda
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 4, 2015
First Posted
November 16, 2015
Study Start
December 7, 2015
Primary Completion
January 24, 2019
Study Completion
February 15, 2019
Last Updated
February 19, 2025
Results First Posted
March 16, 2020
Record last verified: 2021-05
Data Sharing
- IPD Sharing
- Will not share
De-identified individual participant data from this particular study will not be shared as the data are subject to contractual (or consent) provisions that prohibit transfer to third parties.