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

Phase 3
Terminated
Conditions
Eosinophilic Esophagitis (EoE)
Interventions
Registration Number
NCT03245840
Lead Sponsor
Shire
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.

Detailed Description

Not available

Recruitment & Eligibility

Status
TERMINATED
Sex
All
Target Recruitment
133
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 & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Budesonide Oral SuspensionBudesonide oral suspensionParticipants 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.
Primary Outcome Measures
NameTimeMethod
Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Serious TEAEsFrom start of study drug administration up to End of study (EOS) (Up to Month 53)

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.

Change From Baseline in Bone Mineral Density (BMD) For Adolescents Assessed by Dual-Energy X-ray Absorptiometry (DXA) Scan at Month 12Baseline, 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.

Change From Baseline in BMD For Adolescents Assessed by DXA Scan at Month 24Baseline, 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.

Change From Baseline in Cortisol Level After Adrenocorticotropic Hormone (ACTH) Stimulation at Month 12Baseline, 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.

Change From Baseline in Cortisol Level After ACTH Stimulation at Month 36Baseline, 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.

Change From Baseline in Cortisol Level After ACTH Stimulation at Month 48Baseline, 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.

Change From Baseline in Cortisol Level After ACTH Stimulation at EOS (Up to Month 53)Baseline, 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.

Number of Participants With Clinically Significant Change From Baseline in Vital SignsFrom start of study drug administration up to EOS (Up to Month 53)

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.

Change From Baseline in BMD For Adolescents Assessed by DXA Scan at Month 36Baseline, 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.

Change From Baseline in BMD For Adolescents Assessed by DXA Scan at EOS (Up to Month 53)Baseline, 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.

Number of Participants With Clinically Significant Changes in Clinical Laboratory AssessmentsFrom start of study drug administration up to EOS (Up to Month 53)

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.

Change From Baseline in BMD For Adolescents Assessed by DXA Scan at Month 48Baseline, 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.

Change From Baseline in Cortisol Level After ACTH Stimulation at Month 24Baseline, 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.

Number of Participants With Clinically Significant Physical Examination FindingsFrom start of study drug administration up to EOS (Up to Month 53)

Number of participants with clinically significant physical examination findings were reported. Clinical significance was determined by investigator.

Secondary Outcome Measures
NameTimeMethod

Trial Locations

Locations (45)

Connecticut GI

🇺🇸

Hartford, Connecticut, United States

Emeritas Research Group

🇺🇸

Lansdowne Town Center, Virginia, United States

Blue Ridge Medical Research

🇺🇸

Lynchburg, Virginia, United States

Tufts Medical Center

🇺🇸

Boston, Massachusetts, United States

Del Sol Research Management

🇺🇸

Tucson, Arizona, United States

Arkansas Gastroenterology

🇺🇸

North Little Rock, Arkansas, United States

Asthma and Allergy Associates PC

🇺🇸

Colorado Springs, Colorado, United States

Connecticut Clinical Research Foundation

🇺🇸

Bristol, Connecticut, United States

Connecticut Children's Medical Center

🇺🇸

Hartford, Connecticut, United States

Nature Coast Clinical Research LLC

🇺🇸

Inverness, Florida, United States

Childrens Center For Digestive Healthcare

🇺🇸

Atlanta, Georgia, United States

Northwestern University

🇺🇸

Chicago, Illinois, United States

Gastroenterology Associates of Central Georgia

🇺🇸

Macon, Georgia, United States

Grand Teton Research Group

🇺🇸

Idaho Falls, Idaho, United States

Gastroenterology of Southern Indiana

🇺🇸

New Albany, Indiana, United States

University of Iowa Hospitals and Clinics

🇺🇸

Iowa City, Iowa, United States

Cotton O'Neil Clinical Research Center

🇺🇸

Topeka, Kansas, United States

Gastroenterology Associates, LLC

🇺🇸

Baton Rouge, Louisiana, United States

Clinical Trials Management LLC

🇺🇸

Metairie, Louisiana, United States

Boston Children's Hospital

🇺🇸

Boston, Massachusetts, United States

Brigham and Womens Hospital

🇺🇸

Chestnut Hill, Massachusetts, United States

Minnesota Gastroenterology PA

🇺🇸

Plymouth, Minnesota, United States

Mount Sinai Hospital, Icahn School of Medicine

🇺🇸

Astoria, New York, United States

Long Island Gastrointestinal Research Group LLP

🇺🇸

Great Neck, New York, United States

Clinical Research of Charlotte

🇺🇸

Charlotte, North Carolina, United States

Cleveland Clinic

🇺🇸

Cleveland, Ohio, United States

Gastrointestinal and Liver Diseases Consultants PC

🇺🇸

Dayton, Ohio, United States

Great Lakes Gastroenterology

🇺🇸

Mentor, Ohio, United States

Greenville Hospital

🇺🇸

Greenville, South Carolina, United States

Gastro One

🇺🇸

Germantown, Tennessee, United States

Houston Endoscopy and Research Center

🇺🇸

Houston, Texas, United States

Advanced Research Institute

🇺🇸

Ogden, Utah, United States

Phoenix Childrens Hospital

🇺🇸

Phoenix, Arizona, United States

Children's Hospital

🇺🇸

Birmingham, Alabama, United States

Rady Children's Hospital San Diego

🇺🇸

San Diego, California, United States

Colorado Children's Hospital

🇺🇸

Aurora, Colorado, United States

Rocky Mountain Pediatric Gastroenterology

🇺🇸

Denver, Colorado, United States

Arnold Palmer Hospital for Children

🇺🇸

Orlando, Florida, United States

Indiana University

🇺🇸

Louisville, Kentucky, United States

University of North Carolina at Chapel Hill

🇺🇸

Chapel Hill, North Carolina, United States

Cincinnati Children's Hospital Medical Center

🇺🇸

Cincinnati, Ohio, United States

University of Cincinnati

🇺🇸

Cincinnati, Ohio, United States

Digestive Disease Specialists, Inc.

🇺🇸

Oklahoma City, Oklahoma, United States

Vanderbilt University Medical Center

🇺🇸

Nashville, Tennessee, United States

Primary Children's Hospital, University of Utah

🇺🇸

Salt Lake City, Utah, United States

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