A Study in Adolescents and Adults With Eosinophilic Esophagitis (EoE) Measuring Histologic Response and Determine if Reduction in Dysphagia is Achieved

• ClinicalTrials.gov Identifier: NCT02605837
• Recruitment Status: Completed
• First Posted: November 16, 2015
• Results First Posted: March 16, 2020
• Last Update Posted: June 8, 2021
• Sponsor: Shire
• Information provided by (Responsible Party): Takeda ( Shire )

Study Description

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.

Condition or disease	Intervention/treatment	Phase
Eosinophilic Esophagitis (EoE)	Drug: Oral Budesonide Suspension (OBS); Drug: Placebo	Phase 3

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 318 participants
• Allocation: Randomized
• Intervention Model: Parallel Assignment
• Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
• Primary Purpose: Treatment
• Official Title: 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
• Actual Study Start Date: December 7, 2015
• Actual Primary Completion Date: January 24, 2019
• Actual Study Completion Date: February 15, 2019

Arms and Interventions

Arm	Intervention/treatment
Experimental: Oral Budesonide Suspension (OBS); Participants will receive Oral Budesonide Suspension (OBS) 10 milliliter (ml) of 0.2 milligram per milliliter (mg/ml) twice daily up to 16 weeks.	Drug: Oral Budesonide Suspension (OBS); Oral Budesonide Suspension (OBS) 10 milliliter (ml) of 0.2 milligram per milliliter (mg/ml) twice daily up to 16 weeks.
Placebo Comparator: Placebo; Participants will receive oral dose of 10 ml of placebo matched with the experimental drug twice daily up to 16 weeks.	Drug: Placebo; Oral dose of 10 ml of placebo matched with the experimental drug.

Outcome Measures

Primary Outcome Measures :

1. Number of Participants With Histologic Response at the Final Treatment Period Evaluation (Week 16) [ Time Frame: 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.
2. Number of Participants With Dysphagia Symptom Response at the Final Treatment Period Evaluation (Week 16) [ Time Frame: 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]×14)/ Number of diaries reported with non-missing data. Dysphagia symptom response after 12 weeks of double blind treatment at Week 16 was reported.

Secondary Outcome Measures :

1. Change From Baseline in Dysphagia Symptom Questionnaire (DSQ) Combined Score at the Final Treatment Period Evaluation (Week 16) [ Time Frame: Baseline, Week 16 ]
   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 combined score= ([sum of points from questions 2+3 in the daily DSQ]×14)/ Number of diaries reported with non-missing data. Scale range was 0 - 2 for question 2 and 0 - 4 for question 3, with higher values representing a worse outcome. Scale range for DSQ combined score was 0 - 84, with higher values representing a worse outcome. A negative change from baseline indicates that symptoms decreased. Change from baseline in DSQ after 12 weeks of double blind treatment at Week 16 was reported.
2. Change From Baseline in Total Endoscopy Score at the Final Treatment Period Evaluation (Week 16) [ Time Frame: Baseline, Week 16 ]
   Endoscopic findings with separate evaluations of the proximal and distal esophagus were recorded with respect to 5 categories: 1) exudates or plaques (grade 0-2); 2) fixed esophageal rings (grade 0-3); 3) edema (grade 0-2); 4) furrows (grade 0-2); and 5) strictures (grade 0-1). An endoscopy score for each category was calculated and summed for each anatomic location (proximal and distal). The minimum and maximum endoscopy score was 0 and 10 points respectively for each location (proximal and distal) and the total endoscopy score was the sum of the scores for the proximal and distal locations (maximum total score of 20 points respectively). The higher score indicated worse appearance. A negative change from baseline indicates that appearance improved. Endoscopic findings after 12 weeks of double blind treatment at Week 16 were reported.
3. 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) [ Time Frame: Week 16 ]
   Participant was considered as responder at Week 16 if he/she had peak eosinophil count of <15/HPF or <=1/HPF across all esophagus levels. Number of participants with peak eosinophil count < 15/HPF or <=1/HPF after 12 weeks of double blind treatment at Week 16 were reported.
4. Change From Baseline in the Peak Eosinophil Count at the Final Treatment Period Evaluation (Week 16) [ Time Frame: Baseline, Week 16 ]
   Change from baseline in the peak eosinophil count after 12 weeks of double blind treatment at week 16 for each available esophageal level (proximal, mid, distal, maximum) were reported.
5. Change From Baseline in the Histopathologic Epithelial Features Combined Total Score Ratio (TSR) at the Final Treatment Period Evaluation (Week 16) [ Time Frame: Baseline, Week 16 ]
   Change from baseline in histopathologic epithelial features combined total score of grade and stage ratio after 12 weeks of double blind treatment at Week 16 were reported by measuring eight histopathologic epithelial features: basal layer hyperplasia, eosinophil density, eosinophil micro-abscesses, eosinophil surface layering, dilated intercellular spaces, surface epithelial alteration, dyskeratotic epithelial cells, lamina propria fibrosis were scored on a 4-point scale (0=normal, 3=worst) for both the severity of the abnormality (grade) and the amount of tissue affected by the abnormality (stage). Thus each of the 3 levels had a minimum score of 0 and maximum possible score of 24, and a possible total grade or stage score of 72 for a maximum combined score of 144. Combined total score ratio (TSR) =(proximal TSR + mid TSR + distal TSR)/N, where N is the number of non missing sections for TSR. A negative change from baseline indicates that epithelial inflammation decreased.
6. Number of Participants With Dysphagia Symptom Response (Binary Response) at the Final Treatment Period Evaluation (Week 16) [ Time Frame: Week 16 ]
   Dysphagia symptom response (binary response [i.e, responders versus. non-responders]) was defined as a >=50% reduction in the DSQ combined score (questions 2+3), from baseline to the final treatment period evaluation (Week 16). 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]×14)/ Number of diaries reported with non-missing data. Number of participants with binary response after 12 weeks of double blind treatment at Week 16 were reported.
7. Number of Participants With Overall Binary Response I at the Final Treatment Period Evaluation (Week 16) [ Time Frame: Week 16 ]
   Overall binary response I was defined as a reduction in the DSQ score of >=30% from baseline to the final treatment period (week 16) evaluation and a peak eosinophil count of <=6/HPF across all esophageal levels at the final treatment period evaluation. Participant was considered as responder at Week 16 if he/she achieved a minimum of 30% reduction in DSQ combined score between baseline and Week 16 and has peak eosinophil count of <=6/HPF across all esophagus levels. Number of participants with overall binary response I after 12 weeks of double blind treatment at Week 16 were reported.
8. Number of Participants With Overall Binary Response II at the Final Treatment Period Evaluation (Week 16) [ Time Frame: Week 16 ]
   Overall binary response II was defined as a reduction in the DSQ score of >=50% from baseline to the final treatment period evaluation and a peak eosinophil count of <=6/HPF across all esophageal levels at the final treatment period evaluation. Participant was considered as responder at Week 16 if he/she achieved a minimum of 50% reduction in DSQ combined score between baseline and Week 16 and had peak eosinophil count of <=6/HPF across all esophagus levels. Number of participants with overall binary response II after 12 weeks of double blind treatment at Week 16 were reported.
9. Change From Baseline in the Dysphagia Symptom Questionnaire (DSQ) + Pain Score (Questions 2 +3+4) at the Final Treatment Period Evaluation (Week 16) [ Time Frame: Baseline, Week 16 ]
   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 + pain score was calculated by summing the scores of responses to questions 2, 3, and 4 by using following formula: DSQ + pain score= ([sum of points from questions 2+3+4 in the daily DSQ] ×14)/ Number of diaries reported with non-missing data. Scale range was 0 - 2 for question 2, 0 - 4 for question 3 and 0 - 4 for question 4, with higher values representing a worse outcome. Scale range for DSQ + pain score was 0 - 140, with higher values representing a worse outcome. A negative change from baseline indicates that symptoms decreased.
10. Change From Baseline in the Dysphagia Symptom Questionnaire (DSQ) Pain Score (Question 4) at the Final Treatment Period Evaluation (Week 16) [ Time Frame: Baseline, Week 16 ]
    DSQ pain score was calculated by summing the scores of responses to Question 4 (extent to which the participant experienced pain while swallowing) only, by using the following formula: DSQ pain score= [(sum of points from question 4 in the daily DSQ)×14]/ Number of diaries reported with non-missing data. Scale range was 0 - 4 for question 4, with higher values representing a worse outcome. Scale range for DSQ pain score was 0 - 56, with higher values representing a worse outcome. A negative change from baseline indicates that symptoms decreased. Change from baseline in DSQ pain score (question 4) after 12 weeks of double blind treatment at Week 16 were reported.
11. Number of Participants With Treatment-Emergent Adverse Events (AE) [ Time Frame: From start of study drug administration up to follow-up (Week 20) ]
    An AE was any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. TEAEs were defined as AEs that start or deteriorate on or after the first dose of double-blind IP (Week 44) and through the safety follow-up contact, or 31 days after the last dose of IP for participants who did not have a safety follow-up contact. Number of participants with TEAE's were reported.
12. Area Under the Plasma Concentration-Time Curve (AUCtau) Between the Defined Interval of Budesonide Doses [ Time Frame: Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 ]
    Area under the curve for the defined interval between doses (12 hours), calculated using the linear-up/log-down trapezoidal rule.The AUCtau of plasma budesonide was reported. Hours times pico grams per milliliter was abbreviated as h.pg/mL.
13. Maximum Observed Concentration (Cmax) of Budesonide in Plasma [ Time Frame: Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 ]
    The Cmax of budesonide in plasma was reported.
14. Time to Maximum Observed Plasma Concentration (Tmax) of Budesonide in Plasma [ Time Frame: Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 ]
    Tmax of budesonide in plasma was reported.
15. Terminal Rate Constant (Lambda Z) of Budesonide in Plasma [ Time Frame: Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 ]
    Lambda Z of budesonide in plasma was reported.
16. Terminal Half-Life (t1/2) of Budesonide in Plasma [ Time Frame: Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 ]
    t1/2 of budesonide in plasma was reported
17. Apparent Oral Clearance (CL/F) of Budesonide in Plasma [ Time Frame: Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 ]
    CL/F of budesonide in plasma was reported.
18. Apparent Volume of Distribution (Vz/F) of Budesonide in Plasma [ Time Frame: Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 ]
    Vz/F of budesonide in plasma was reported.

Eligibility Criteria

• Ages Eligible for Study: 11 Years to 55 Years (Child, Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria

• 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.

Exclusion Criteria

• 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).
• Participant has evidence of active infection with Helicobacter pylori.
• Participant has evidence of unstable asthma within 4 weeks prior to the screening visit (Visit -1) and between the screening visit and baseline visit (Visit 1).
• Participant is female and pregnant or nursing.
• Participant has a history of intolerance, hypersensitivity, or idiosyncratic reaction to budesonide (or any other corticosteroids) or to any other ingredients of the investigational product.
• Participant has taken part and received intervention in an interventional study related to EoE (except for an interventional study for a topical swallowed steroid) within 6 months prior to the screening visit (Visit -1), or any investigational study within 30 days prior to the screening visit (Visit -1). An investigational topical swallowed steroid must have been discontinued at least 30 days prior to the screening visit (Visit -1).
• Participant has a history or high risk of noncompliance with treatment or regular clinic visits.
• Participant has previously completed, discontinued, or withdrawn from this study.
• Participant has participated in a previous clinical study involving oral budesonide suspension (OBS) (SHP621).
• Participant anticipates using sucralfate during the study.

Contacts and Locations

Locations

United States, Alabama

Children's Hospital

Birmingham, Alabama, United States, 35233

United States, Arizona

Phoenix Childrens Hospital

Phoenix, Arizona, United States, 85016

Del Sol Research Management

Tucson, Arizona, United States, 85710

Adobe Clinical Research LLC

Tucson, Arizona, United States, 85712

United States, Arkansas

Arkansas Gastroenterology

North Little Rock, Arkansas, United States, 72117

United States, California

GW Research, Inc.

Chula Vista, California, United States, 91910

Rady Children's Hospital San Diego

San Diego, California, United States, 92123

United States, Colorado

Colorado Children's Hospital

Aurora, Colorado, United States, 80045

Asthma and Allergy Associates PC

Colorado Springs, Colorado, United States, 80907

Rocky Mountain Pediatric Gastroenterology

Lone Tree, Colorado, United States, 80124

Rocky Mountain Clinical Research LLC

Wheat Ridge, Colorado, United States, 80033

United States, Connecticut

Connecticut Clinical Research Foundation

Bristol, Connecticut, United States, 06010

Connecticut GI, PC - Research Division

Farmington, Connecticut, United States, 06032

Connecticut Children's Medical Center

Hartford, Connecticut, United States, 06106

United States, Florida

Nature Coast Clinical Research LLC

Inverness, Florida, United States, 34452

Borland Groover Clinic

Jacksonville, Florida, United States, 32256

Arnold Palmer Hospital For Children

Orlando, Florida, United States, 32806

Accord Clinical Research LLC

Port Orange, Florida, United States, 32129

United States, Georgia

Children's Center for Digestive Health Care

Atlanta, Georgia, United States, 30342

Gastroenterology Associates of Central Georgia, LLC

Macon, Georgia, United States, 31201

Gastrointestinal Specialists of Georgia

Marietta, Georgia, United States, 30060

United States, Idaho

Grand Teton Research Group, PLLC

Idaho Falls, Idaho, United States, 83404

United States, Illinois

Ann and Robert H Lurie Childrens Hospital of Chicago

Chicago, Illinois, United States, 60611

Northwestern University

Chicago, Illinois, United States, 60611

Center for Children's Digestive Health

Park Ridge, Illinois, United States, 60068

University of Illinois College of Medicine at Peoria Pediatric Subspecialty Clinic

Peoria, Illinois, United States, 61603

OSF St Francis Medical Center

Peoria, Illinois, United States, 61637

United States, Indiana

Indiana University

Indianapolis, Indiana, United States, 46202

Gastroenterology of Southern Indiana

New Albany, Indiana, United States, 47150

United States, Iowa

University of Iowa Hospitals and Clinics

Iowa City, Iowa, United States, 52242

United States, Kansas

Cotton O'Neil Clinical Research Center

Topeka, Kansas, United States, 66606

United States, Louisiana

Gastroenterology Associates LLC

Baton Rouge, Louisiana, United States, 70809

Clinical Trials Management LLC

Metairie, Louisiana, United States, 70006

Louisiana Research Center LLC

Shreveport, Louisiana, United States, 71105

Clinical Trials of America LA LLC - PPDS

West Monroe, Louisiana, United States, 71291

United States, Massachusetts

Tufts Medical Center

Boston, Massachusetts, United States, 00211

Boston Children's Hospital

Boston, Massachusetts, United States, 02115

Brigham and Womens Hospital

Boston, Massachusetts, United States, 02115

United States, Michigan

University of Michigan

Ann Arbor, Michigan, United States, 48109

Clinical Research Institute of Michigan

Chesterfield, Michigan, United States, 48047

West Michigan Clinical Research

Wyoming, Michigan, United States, 49519

United States, Minnesota

University of Minnesota

Minneapolis, Minnesota, United States, 55454

Minnesota Gastroenterology PA

Plymouth, Minnesota, United States, 55446

Mayo Clinic

Rochester, Minnesota, United States, 55905

United States, Montana

Bozeman Health Deaconess Hospital

Bozeman, Montana, United States, 59718

United States, New Jersey

South Jersey Gastroenterology

Marlton, New Jersey, United States, 08053

United States, New York

Long Island Gastrointestinal Research Group LLP

Great Neck, New York, United States, 11023

Mount Sinai Hospital

New York, New York, United States, 10029

United States, North Carolina

Asheville Gastroenterology Associates PA

Asheville, North Carolina, United States, 28801

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, United States, 27514

Clinical Research of Charlotte

Charlotte, North Carolina, United States, 28277

Clinical Trials of America-NC, LLC - PPDS

Mount Airy, North Carolina, United States, 27030

United States, Ohio

Consultants For Clinical Research Inc

Cincinnati, Ohio, United States, 45219

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States, 45229

University of Cincinnati

Cincinnati, Ohio, United States, 45267

Cleveland Clinic

Cleveland, Ohio, United States, 44195

Gastrointestinal and Liver Diseases Consultants PC

Dayton, Ohio, United States, 45415

Great Lakes Gastroenterology

Mentor, Ohio, United States, 44060

United States, Oklahoma

Digestive Disease Specialists, Inc.

Oklahoma City, Oklahoma, United States, 73112

United States, Pennsylvania

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States, 19104

University of Pennsylvania

Philadelphia, Pennsylvania, United States, 19104

United States, South Carolina

Greenville Hospital System

Greenville, South Carolina, United States, 29605

Greenville Hospital System

Greenville, South Carolina, United States, 29615

United States, Tennessee

Gastro One

Germantown, Tennessee, United States, 38138

Vanderbilt University Medical Center

Nashville, Tennessee, United States, 37212

United States, Texas

San Antonio Military Medical Center

Fort Sam Houston, Texas, United States, 78234

Baylor College of Medicine

Houston, Texas, United States, 77030

Houston Endoscopy and Research Center

Houston, Texas, United States, 77079

Digestive Health Center

Pasadena, Texas, United States, 77505

Texas Digestive Disease Consultants

Southlake, Texas, United States, 76092

United States, Utah

Advanced Research Institute

Ogden, Utah, United States, 84092

Primary Children's Hospital, University of Utah

Salt Lake City, Utah, United States, 84113

Advanced Research Institute

Sandy, Utah, United States, 84092

United States, Virginia

Emeritas Research Group

Lansdowne Town Center, Virginia, United States, 20176

Blue Ridge Medical Research

Lynchburg, Virginia, United States, 24502

Carilion Clinic

Roanoke, Virginia, United States, 24013

United States, Wisconsin

University of Wisconsin

Madison, Wisconsin, United States, 53792

Sponsors and Collaborators

Shire

Investigators

• Study Director: Study Director; Takeda

  Study Documents (Full-Text)

Documents provided by Takeda ( Shire ):

Study Protocol  [PDF] January 26, 2018

Statistical Analysis Plan  [PDF] March 11, 2019

More Information

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Dellon 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.

Hirano 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. Erratum In: Clin Gastroenterol Hepatol. 2022 Oct;20(10):2418.

• Responsible Party: Shire
• ClinicalTrials.gov Identifier: NCT02605837
• Other Study ID Numbers: SHP621-301
• First Posted: November 16, 2015
• Results First Posted: March 16, 2020
• Last Update Posted: June 8, 2021
• Last Verified: May 2021

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: Yes
• Plan Description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
• Supporting Materials: Study Protocol; Statistical Analysis Plan (SAP); Informed Consent Form (ICF); Clinical Study Report (CSR)
• Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
• URL: https://vivli.org/ourmember/takeda/

Additional relevant MeSH terms:

Esophagitis; Eosinophilic Esophagitis; Esophageal Diseases; Gastrointestinal Diseases; Digestive System Diseases; Gastroenteritis; Eosinophilia; Leukocyte Disorders; Hematologic Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Budesonide; Anti-Inflammatory Agents; Bronchodilator Agents; Autonomic Agents; Peripheral Nervous System Agents; Physiological Effects of Drugs; Anti-Asthmatic Agents; Respiratory System Agents; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists