Continuation Study With Budesonide Oral Suspension (BOS) for Adolescent and Adult Participants With Eosinophilic Esophagitis (EoE)

A Phase 3, Multicenter, Open-label Continuation Study With Budesonide Oral Suspension (BOS) for Adolescent and Adult Subjects With Eosinophilic Esophagitis (EoE)

This is a continuation study of Budesonide Oral Suspension (BOS) in adults and adolescents with Eosinophilic Esophagitis (EoE) who have completed participation in the SHP621-302 extension study. The purpose of this study is to see if BOS is safe and well tolerated over the long-term in adolescents and adults with EoE.

Study Overview

• Status: Terminated
• Conditions: Eosinophilic Esophagitis (EoE)
• Intervention / Treatment: Drug: Budesonide oral suspension

• Study Type: Interventional
• Enrollment (Actual): 133
• Phase: Phase 3

Contacts and Locations

Study Locations

• United States
  • Alabama
    • Birmingham, Alabama, United States, 35233
      • Children's Hospital
  • Arizona
    • Phoenix, Arizona, United States, 85016
      • Phoenix Childrens Hospital
    • Tucson, Arizona, United States, 85712
      • Del Sol Research Management
  • Arkansas
    • North Little Rock, Arkansas, United States, 72117
      • Arkansas Gastroenterology
  • California
    • San Diego, California, United States, 92123
      • Rady Children's Hospital San Diego
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Colorado Children's Hospital
    • Colorado Springs, Colorado, United States, 80907
      • Asthma and Allergy Associates PC
    • Denver, Colorado, United States, 80218
      • Rocky Mountain Pediatric Gastroenterology
  • Connecticut
    • Bristol, Connecticut, United States, 06010
      • Connecticut Clinical Research Foundation
    • Hartford, Connecticut, United States, 06106
      • Connecticut Children's Medical Center
    • Hartford, Connecticut, United States, 06106
      • Connecticut GI
  • Florida
    • Inverness, Florida, United States, 34452
      • Nature Coast Clinical Research LLC
    • Orlando, Florida, United States, 32806
      • Arnold Palmer Hospital for Children
  • Georgia
    • Atlanta, Georgia, United States, 30342
      • Childrens Center For Digestive Healthcare
    • Macon, Georgia, United States, 31201
      • Gastroenterology Associates of Central Georgia
  • Idaho
    • Idaho Falls, Idaho, United States, 83404
      • Grand Teton Research Group
  • Illinois
    • Chicago, Illinois, United States, 60611
      • Northwestern University
  • Indiana
    • New Albany, Indiana, United States, 47150
      • Gastroenterology of Southern Indiana
  • Iowa
    • Iowa City, Iowa, United States, 52242
      • University of Iowa Hospitals and Clinics
  • Kansas
    • Topeka, Kansas, United States, 66606
      • Cotton O'Neil Clinical Research Center
  • Kentucky
    • Louisville, Kentucky, United States, 40202
      • Indiana University
  • Louisiana
    • Baton Rouge, Louisiana, United States, 70809
      • Gastroenterology Associates, LLC
    • Metairie, Louisiana, United States, 70006
      • Clinical Trials Management LLC
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital
    • Boston, Massachusetts, United States, 00211
      • Tufts Medical Center
    • Chestnut Hill, Massachusetts, United States, 02467
      • Brigham and Womens Hospital
  • Minnesota
    • Plymouth, Minnesota, United States, 55446
      • Minnesota Gastroenterology PA
  • New York
    • Astoria, New York, United States, 11102
      • Mount Sinai Hospital, Icahn School of Medicine
    • Great Neck, New York, United States, 11023
      • Long Island Gastrointestinal Research Group LLP
  • North Carolina
    • Chapel Hill, North Carolina, United States, 27514
      • University of North Carolina at Chapel Hill
    • Charlotte, North Carolina, United States, 28277
      • Clinical Research of Charlotte
  • Ohio
    • Cincinnati, Ohio, United States, 45229
      • Cincinnati Children's Hospital Medical Center
    • Cincinnati, Ohio, United States, 45267
      • University of Cincinnati
    • Cleveland, Ohio, United States, 44195
      • Cleveland Clinic
    • Dayton, Ohio, United States, 45440
      • Gastrointestinal and Liver Diseases Consultants PC
    • Mentor, Ohio, United States, 44060
      • Great Lakes Gastroenterology
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73112
      • Digestive Disease Specialists, Inc.
  • South Carolina
    • Greenville, South Carolina, United States, 29615
      • Greenville Hospital
  • Tennessee
    • Germantown, Tennessee, United States, 38138
      • Gastro One
    • Nashville, Tennessee, United States, 37212
      • Vanderbilt University Medical Center
  • Texas
    • Houston, Texas, United States, 77024
      • Houston Endoscopy and Research Center
  • Utah
    • Ogden, Utah, United States, 84405
      • Advanced Research Institute
    • Salt Lake City, Utah, United States, 84132
      • Primary Children's Hospital, University of Utah
  • Virginia
    • Lansdowne Town Center, Virginia, United States, 20176
      • Emeritas Research Group
    • Lynchburg, Virginia, United States, 24502
      • Blue Ridge Medical Research

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

• Participant completed the SHP621-302 (NCT02736409) extension study and is considered by the investigator to potentially benefit from continued BOS investigational treatment.
• Participant 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.
• Females of childbearing potential must agree to continue acceptable birth control measures (example (e.g.): abstinence, surgically sterile male partner, 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 as defined in protocol.

Exclusion Criteria:

• Participant has changes in medications or diet during the SHP621-302 (NCT02736409) study that could affect participation in this continuation study.
• Participant anticipates using swallowed topical corticosteroid for EoE or systemic corticosteroid for any condition 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 should be avoided within 4 weeks of the scheduled esophagogastroduodenoscopy (EGDs).
• Participant anticipates use of Cytochrome P450 3A4 inhibitors (e.g., ketoconazole, grapefruit juice) during the continuation study.
• Participant has an appearance at the EGD at the final treatment evaluation visit of SHP621-302 (NCT02736409) (Visit 8) 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 (e.g., with an insertion tube diameter of greater than (>) 9 millimeter [mm]).
• Participant has presence of esophageal varices at the EGD at the final treatment evaluation visit (Visit 8) of the SHP621-302 (NCT02736409) study.
• 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 esophageal eosinophilia, including hypereosinophilic syndrome, collagen vascular disease, vasculitis, achalasia, or parasitic infection.
• Participant has oropharyngeal or esophageal candidiasis that failed to respond to previous treatment. Diagnosis with oropharyngeal or esophageal candidiasis at or since the final treatment evaluation visit (Visit 8) of the SHP621-302 (NCT02736409) study is not an exclusion as long as the participant is expected to respond to treatment.
• Participant has a potentially serious acute or chronic infection or immunodeficiency condition, including tuberculosis, fungal, bacterial, viral/parasite infection, ocular herpes simplex, or chicken pox/measles.
• Participant has upper gastrointestinal bleeding identified at the EGD at the final treatment evaluation visit (Visit 8) of the SHP621-302 (NCT02736409) study.
• Participant has evidence of active infection with Helicobacter pylori.
• Participant has evidence of unstable asthma.
• 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 study medication.
• Participant has a history or high risk of noncompliance with treatment or regular clinic visits.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Budesonide Oral Suspension; Participants received 10 milliliters (mL) of budesonide oral suspension at a concentration of 0.2 milligram per milliliter (mg/mL), twice daily, for up to 4 years 5 months.	Drug: Budesonide oral suspension; BOS 10 mL twice daily.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEs	An adverse event (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. A Serious Adverse Event (SAE) was any untoward medical occurrence (whether considered to be related to investigational product or not) that at any dose: results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant disability/incapacity, is a congenital abnormality/birth defect, and is an important medical event. Both serious and Non-serious TEAEs were reported in this outcome measure. TEAEs were defined as AEs with a start date on or after the first dose of investigational product or a start date before the date of the first dose of investigational product that increased in severity or after the date of the first dose.	From start of study drug administration up to End of study (EOS) (Up to Month 53)
Number of Participants With Clinically Significant Physical Examination Findings	Number of participants with clinically significant physical examination findings were reported. Clinical significance was determined by investigator.	From start of study drug administration up to EOS (Up to Month 53)
Number of Participants With Clinically Significant Change From Baseline in Vital Signs	Participants were assessed by investigator for any clinically significant changes in vital parameters like temperature, systolic and diastolic blood pressure, pulse, respiratory rate, BMI, and weight. Vital signs were assessed after the participant had been in a supine position for at least 5 minutes immediately prior to the assessment. The criteria for clinically significant change was as per the investigators discretion.	From start of study drug administration up to EOS (Up to Month 53)
Change From Baseline in Bone Mineral Density (BMD) For Adolescents Assessed by Dual-Energy X-ray Absorptiometry (DXA) Scan at Month 12	The sites for DXA measurement were the lumbar spine at lumbar vertebrae 1 to 4 (L1-L4) and whole body. DXA scans for determination of BMD and body composition was performed in participants aged 11-17 years. Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. In this study, the BMD Z-score is considered abnormal when the z-score is less than (<) -2, suggesting a worse outcome (i.e., osteoporosis). Change from baseline in BMD for adolescents assessed by DXA Scan at Month 12 was reported.	Baseline, Month 12
Change From Baseline in BMD For Adolescents Assessed by DXA Scan at Month 24	The sites for DXA measurement were the lumbar spine at lumbar vertebrae 1 to 4 (L1-L4) and whole body. DXA scans for determination of BMD and body composition was performed in participants aged 11-17 years. Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. In this study, the BMD Z-score is considered abnormal when the z-score is <-2, suggesting a worse outcome (i.e., osteoporosis) and vice versa. Change from baseline in BMD for adolescents assessed by DXA Scan at Month 24 was reported.	Baseline, Month 24
Change From Baseline in BMD For Adolescents Assessed by DXA Scan at Month 36	The sites for DXA measurement were the lumbar spine at lumbar vertebrae 1 to 4 (L1-L4) and whole body. DXA scans for determination of BMD and body composition was performed in participants aged 11-17 years. Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. In this study, the BMD Z-score is considered abnormal when the z-score is < -2, suggesting a worse outcome (i.e., osteoporosis) and vice versa. Change from baseline in BMD for adolescents assessed by DXA Scan at Month 36 was reported.	Baseline, Month 36
Change From Baseline in BMD For Adolescents Assessed by DXA Scan at Month 48	The sites for DXA measurement were the lumbar spine at lumbar vertebrae 1 to 4 (L1-L4) and whole body. DXA scans for determination of BMD and body composition was performed in participants aged 11-17 years. Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. In this study, the BMD Z-score is considered abnormal when the z-score is < -2, suggesting a worse outcome (i.e., osteoporosis) and vice versa. Change from baseline in BMD for adolescents assessed by DXA Scan at Month 48 was reported.	Baseline, Month 48
Change From Baseline in BMD For Adolescents Assessed by DXA Scan at EOS (Up to Month 53)	The sites for DXA measurement were the lumbar spine at lumbar vertebrae 1 to 4 (L1-L4) and whole body. DXA scans for determination of BMD and body composition was performed in participants aged 11-17 years. Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. In this study, the BMD Z-score is considered abnormal when the z-score is < -2, suggesting a worse outcome (i.e., osteoporosis) and vice versa. Change from baseline in BMD for adolescents assessed by DXA Scan at EOS (up to Month 53) was reported.	Baseline, EOS (Up to Month 53)
Change From Baseline in Cortisol Level After Adrenocorticotropic Hormone (ACTH) Stimulation at Month 12	ACTH testing was a standard procedure to measure the levels of cortisol in the blood following the injection of a synthetic form of ACTH (250 microgram [mcg]). The type of synthetic and route of administration was per investigator discretion. The change from baseline in cortisol levels was calculated at Month 12 and reported in this outcome measure.	Baseline, Month 12
Change From Baseline in Cortisol Level After ACTH Stimulation at Month 24	ACTH testing was a standard procedure to measure the levels of cortisol in the blood following the injection of a synthetic form of ACTH (250 microgram [mcg]). The type of synthetic and route of administration was per investigator discretion. The change from baseline in cortisol levels was calculated at Month 24 and reported in this outcome measure.	Baseline, Month 24
Change From Baseline in Cortisol Level After ACTH Stimulation at Month 36	ACTH testing was a standard procedure to measure the levels of cortisol in the blood following the injection of a synthetic form of ACTH (250 microgram [mcg]). The type of synthetic and route of administration was per investigator discretion. The change from baseline in cortisol levels was calculated at Month 36 and reported in this outcome measure.	Baseline, Month 36
Change From Baseline in Cortisol Level After ACTH Stimulation at Month 48	ACTH testing was a standard procedure to measure the levels of cortisol in the blood following the injection of a synthetic form of ACTH (250 microgram [mcg]). The type of synthetic and route of administration was per investigator discretion. The change from baseline in cortisol levels was calculated at Month 48 and reported in this outcome measure.	Baseline, Month 48
Change From Baseline in Cortisol Level After ACTH Stimulation at EOS (Up to Month 53)	ACTH testing was a standard procedure to measure the levels of cortisol in the blood following the injection of a synthetic form of ACTH (250 microgram [mcg]). The type of synthetic and route of administration was per investigator discretion. The change from baseline in cortisol levels was calculated at EOS (up to Month 53) and reported in this outcome measure.	Baseline, EOS (Up to Month 53)
Number of Participants With Clinically Significant Changes in Clinical Laboratory Assessments	Clinical laboratory parameters included hematology, chemistry, urinalysis; urine pregnancy test. Number of participants with potential clinically significant changes in laboratory parameters which were deemed clinically meaningful by the investigator were reported.	From start of study drug administration up to EOS (Up to Month 53)

Collaborators and Investigators

• Sponsor: Shire
• Collaborators: Takeda Development Center Americas, Inc.
• Investigators: Study Director: Study Director, Shire

Publications and helpful links

Helpful Links

• To obtain more information on the study, click here/on this link

Study record dates

Study Major Dates

• Study Start (Actual): October 5, 2017
• Primary Completion (Actual): April 26, 2022
• Study Completion (Actual): April 26, 2022

Study Registration Dates

• First Submitted: August 8, 2017
• First Submitted That Met QC Criteria: August 8, 2017
• First Posted (Actual): August 10, 2017

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: January 30, 2025
• Last Verified: November 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Immune System Diseases; Digestive System Diseases; Gastrointestinal Diseases; Hypersensitivity, Immediate; Hypersensitivity; Eosinophilia; Leukocyte Disorders; Hematologic Diseases; Esophageal Diseases; Gastroenteritis; Esophagitis; Eosinophilic Esophagitis; Physiological Effects of Drugs; Anti-Inflammatory Agents; Autonomic Agents; Peripheral Nervous System Agents; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Respiratory System Agents; Anti-Asthmatic Agents; Bronchodilator Agents; Budesonide
• Other Study ID Numbers: SHP621-303

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO
• IPD Plan Description: 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.
• IPD Sharing 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.
• IPD Sharing Supporting Information Type: Study Protocol; Statistical Analysis Plan (SAP); Informed Consent Form (ICF); Clinical Study Report (CSR)

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No