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

NCT03245840 | Terminated Phase 3 Results FDA Drug

• 1 other identifier: SHP621-303
• Study Type: interventional
• Target: 133
• Locations: 1 country
• Sites: 45

Brief Summary

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.

Conditions

Eosinophilic Esophagitis (EoE)

Outcome Measures

Primary Outcomes (14)

• 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)

Study Arms (1)

Budesonide Oral Suspension

EXPERIMENTAL

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

Interventions

Budesonide oral suspension DRUG

BOS 10 mL twice daily.

Budesonide Oral Suspension

Eligibility Criteria

• Age: 11 Years - 55 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• 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 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.

Sponsors & Collaborators

• Shire: lead
• Takeda Development Center Americas, Inc.: collaborator

Study Sites (45)

Children's Hospital

Birmingham, Alabama, 35233, United States

Location

Phoenix Childrens Hospital

Phoenix, Arizona, 85016, United States

Location

Del Sol Research Management

Tucson, Arizona, 85712, United States

Location

Arkansas Gastroenterology

North Little Rock, Arkansas, 72117, United States

Location

Rady Children's Hospital San Diego

San Diego, California, 92123, United States

Location

Colorado Children's Hospital

Aurora, Colorado, 80045, United States

Location

Asthma and Allergy Associates PC

Colorado Springs, Colorado, 80907, United States

Location

Rocky Mountain Pediatric Gastroenterology

Denver, Colorado, 80218, United States

Location

Connecticut Clinical Research Foundation

Bristol, Connecticut, 06010, United States

Location

Connecticut Children's Medical Center

Hartford, Connecticut, 06106, United States

Location

Connecticut GI

Hartford, Connecticut, 06106, United States

Location

Nature Coast Clinical Research LLC

Inverness, Florida, 34452, United States

Location

Arnold Palmer Hospital for Children

Orlando, Florida, 32806, United States

Location

Childrens Center For Digestive Healthcare

Atlanta, Georgia, 30342, United States

Location

Gastroenterology Associates of Central Georgia

Macon, Georgia, 31201, United States

Location

Grand Teton Research Group

Idaho Falls, Idaho, 83404, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

Gastroenterology of Southern Indiana

New Albany, Indiana, 47150, United States

Location

University of Iowa Hospitals and Clinics

Iowa City, Iowa, 52242, United States

Location

Cotton O'Neil Clinical Research Center

Topeka, Kansas, 66606, United States

Location

Indiana University

Louisville, Kentucky, 40202, United States

Location

Gastroenterology Associates, LLC

Baton Rouge, Louisiana, 70809, United States

Location

Clinical Trials Management LLC

Metairie, Louisiana, 70006, United States

Location

Tufts Medical Center

Boston, Massachusetts, 00211, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Brigham and Womens Hospital

Chestnut Hill, Massachusetts, 02467, United States

Location

Minnesota Gastroenterology PA

Plymouth, Minnesota, 55446, United States

Location

Mount Sinai Hospital, Icahn School of Medicine

Astoria, New York, 11102, United States

Location

Long Island Gastrointestinal Research Group LLP

Great Neck, New York, 11023, United States

Location

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27514, United States

Location

Clinical Research of Charlotte

Charlotte, North Carolina, 28277, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

University of Cincinnati

Cincinnati, Ohio, 45267, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

Gastrointestinal and Liver Diseases Consultants PC

Dayton, Ohio, 45440, United States

Location

Great Lakes Gastroenterology

Mentor, Ohio, 44060, United States

Location

Digestive Disease Specialists, Inc.

Oklahoma City, Oklahoma, 73112, United States

Location

Greenville Hospital

Greenville, South Carolina, 29615, United States

Location

Gastro One

Germantown, Tennessee, 38138, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37212, United States

Location

Houston Endoscopy and Research Center

Houston, Texas, 77024, United States

Location

Advanced Research Institute

Ogden, Utah, 84405, United States

Location

Primary Children's Hospital, University of Utah

Salt Lake City, Utah, 84132, United States

Location

Emeritas Research Group

Lansdowne Town Center, Virginia, 20176, United States

Location

Blue Ridge Medical Research

Lynchburg, Virginia, 24502, United States

Location

Related Publications (1)

• Dellon ES, Katzka DA, Mukkada VA, Collins MH, Falk GW, Richmond CA, Terreri B, Thakur M, Boules M, Goodwin B, Hirano I. Long-term Safety and Efficacy of Budesonide Oral Suspension for Eosinophilic Esophagitis: A 4-Year, Phase 3, Open-Label Study. Clin Gastroenterol Hepatol. 2025 Nov;23(12):2155-2166.e5. doi: 10.1016/j.cgh.2024.12.024. Epub 2025 Feb 13.

  PMID: 39954913 DERIVED

Related Links

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

MeSH Terms

Conditions

Eosinophilic Esophagitis

Condition Hierarchy (Ancestors)

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

Limitations and Caveats

The study was discontinued prematurely as per sponsor's decision.

Results Point of Contact

• Title: Study Director
• Organization: Shire

ClinicalTransparency@shire.com

+1 866 842 5335

Study Officials

• Study Director

  Shire

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 8, 2017
• First Posted: August 10, 2017
• Study Start: October 5, 2017
• Primary Completion: April 26, 2022
• Study Completion: April 26, 2022
• Last Updated: February 19, 2025
• Results First Posted: December 7, 2022

Record last verified: 2022-11

Data Sharing

• IPD Sharing: Will not share

Locations

US (45)