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A study in adolescents and adults with eosinophilic esophagitis (EoE) to measure the histologic response and determine if any reduction in dysphagia is achieved.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Oral Budesonide Suspension (OBS) | Experimental | Participants will receive Oral Budesonide Suspension (OBS) 10 milliliter (ml) of 0.2 milligram per milliliter (mg/ml) twice daily up to 16 weeks. |
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| Placebo | Placebo Comparator | Participants will receive oral dose of 10 ml of placebo matched with the experimental drug twice daily up to 16 weeks. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Oral Budesonide Suspension (OBS) | Drug | Oral Budesonide Suspension (OBS) 10 milliliter (ml) of 0.2 milligram per milliliter (mg/ml) twice daily up to 16 weeks. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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]×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 |
| Measure | Description | Time Frame |
|---|---|---|
| Change From Baseline in Dysphagia Symptom Questionnaire (DSQ) Combined Score at 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 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. |
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Inclusion Criteria
Exclusion Criteria
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| Name | Affiliation | Role |
|---|---|---|
| Study Director | Takeda | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital | Birmingham | Alabama | 35233 | United States | ||
| Phoenix Childrens Hospital |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 41711386 | Derived | Dellon ES, Katzka DA, Mukkada VA, Falk GW, Tahir MJ, Gugiu PC, Blau J, Terreri B. Effect of Budesonide Oral Suspension on Time to First Dysphagia Symptom Response and Dysphagia Symptom Resolution Outcomes in Patients With Eosinophilic Esophagitis. J Gastroenterol Hepatol. 2026 Mar;41(3):927-936. doi: 10.1111/jgh.70205. Epub 2026 Feb 19. | |
| 39735351 |
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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.
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A total of 318 participants were randomized and received the treatment (placebo or oral Budesonide suspension [OBS]) in a double blind fashion following a placebo lead-in phase and 296 participants completed the study.
This study was conducted at 72 sites in North America from 07 December 2015 (first participant enrolled) to 15 February 2019 (last participant completed).
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo | Participants received oral dose of 10 milliliter (ml) placebo matched with the Oral Budesonide Suspension (OBS) twice daily up to 16 weeks. |
| FG001 | Oral Budesonide Suspension (OBS) |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jan 26, 2018 | Jan 21, 2020 |
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| Placebo | Drug | Oral dose of 10 ml of placebo matched with the experimental drug. |
|
| Baseline, Week 16 |
| Change From Baseline in Total Endoscopy Score at the Final Treatment Period Evaluation (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. | 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) | 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. | Week 16 |
| Change From Baseline in the Peak Eosinophil Count at the Final Treatment Period Evaluation (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. | Baseline, Week 16 |
| Change From Baseline in the Histopathologic Epithelial Features Combined Total Score Ratio (TSR) at the Final Treatment Period Evaluation (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. | Baseline, Week 16 |
| Number of Participants With Dysphagia Symptom Response (Binary Response) at the Final Treatment Period Evaluation (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. | Week 16 |
| Number of Participants With Overall Binary Response I at the Final Treatment Period Evaluation (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. | Week 16 |
| Number of Participants With Overall Binary Response II at the Final Treatment Period Evaluation (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. | Week 16 |
| Change From Baseline in the Dysphagia Symptom Questionnaire (DSQ) + Pain Score (Questions 2 +3+4) at 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 + 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. | Baseline, Week 16 |
| Change From Baseline in the Dysphagia Symptom Questionnaire (DSQ) Pain Score (Question 4) at the Final Treatment Period Evaluation (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. | Baseline, Week 16 |
| Number of Participants With Treatment-Emergent Adverse Events (AE) | 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. | From start of study drug administration up to follow-up (Week 20) |
| Area Under the Plasma Concentration-Time Curve (AUCtau) Between the Defined Interval of Budesonide Doses | 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. | Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 |
| Maximum Observed Concentration (Cmax) of Budesonide in Plasma | The Cmax of budesonide in plasma was reported. | Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 |
| Time to Maximum Observed Plasma Concentration (Tmax) of Budesonide in Plasma | Tmax of budesonide in plasma was reported. | Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 |
| Terminal Rate Constant (Lambda Z) of Budesonide in Plasma | Lambda Z of budesonide in plasma was reported. | Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 |
| Terminal Half-Life (t1/2) of Budesonide in Plasma | t1/2 of budesonide in plasma was reported | Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 |
| Apparent Oral Clearance (CL/F) of Budesonide in Plasma | CL/F of budesonide in plasma was reported. | Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 |
| Apparent Volume of Distribution (Vz/F) of Budesonide in Plasma | Vz/F of budesonide in plasma was reported. | Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 |
| 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 |
| 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. Ther Adv Gastroenterol. 2024 Dec 23;17:17562848241307602. doi: 10.1177/17562848241307602. eCollection 2024. |
| 39631042 | Derived | Hirano 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. |
| 37718471 | Derived | Mukkada 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. |
| 34182150 | Derived | 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. |
| 33887475 | Derived | 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. |
Post placebo lead in phase (4 weeks) participants received OBS 10 ml (2 mg) twice daily up to 12 weeks.
| COMPLETED |
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| NOT COMPLETED |
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Full analysis set (FAS) included all randomized participants who received at least one dose of a double-blind investigational product (IP).
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| ID | Title | Description |
|---|---|---|
| BG000 | Placebo | Participants received oral dose of 10 milliliter (ml) placebo matched with the Oral Budesonide Suspension (OBS) twice daily up to 16 weeks. |
| BG001 | Oral Budesonide Suspension (OBS) | Post placebo lead in phase (4 weeks) participants received OBS 10 ml (2 mg) twice daily up to 12 weeks. |
| BG002 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
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| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Age was calculated as the difference between date of birth and date of informed consent, truncated to years. | Mean | Standard Deviation | Years |
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| Sex: Female, Male | Count of Participants | Participants |
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| Ethnicity (NIH/OMB) | Count of Participants | Participants |
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| Race (NIH/OMB) | Count of Participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
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| Primary | 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. | FAS included all randomized participants who received at least one dose of a double-blind IP. | Posted | Count of Participants | Participants | Week 16 |
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| Primary | 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]×14)/ Number of diaries reported with non-missing data. Dysphagia symptom response after 12 weeks of double blind treatment at Week 16 was reported. | FAS included all randomized participants who received at least one dose of a double-blind IP. | Posted | Count of Participants | Participants | Week 16 |
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| Secondary | Change From Baseline in Dysphagia Symptom Questionnaire (DSQ) Combined Score at 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 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. | FAS included all randomized participants who received at least one dose of a double-blind IP. Here the number of participants analyzed signifies participants who were evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | Score on scale | Baseline, Week 16 |
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| Secondary | Change From Baseline in Total Endoscopy Score at the Final Treatment Period Evaluation (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. | FAS included all randomized participants who received at least one dose of a double-blind IP. Here the number of participants analyzed signifies participants who were evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | score on scale | Baseline, Week 16 |
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| Secondary | 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) | 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. | FAS included all randomized participants who received at least one dose of a double-blind IP. | Posted | Count of Participants | Participants | Week 16 |
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| Secondary | Change From Baseline in the Peak Eosinophil Count at the Final Treatment Period Evaluation (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. | FAS included all randomized participants who received at least one dose of a double-blind IP. Here the number of participants analyzed signifies participants who were evaluable for this outcome measure at each specific category. | Posted | Least Squares Mean | Standard Error | eosinophil count | Baseline, Week 16 |
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| Secondary | Change From Baseline in the Histopathologic Epithelial Features Combined Total Score Ratio (TSR) at the Final Treatment Period Evaluation (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. | FAS included all randomized participants who received at least one dose of a double-blind IP. Here the number of participants analyzed signifies participants who were evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | score on scale | Baseline, Week 16 |
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| Secondary | Number of Participants With Dysphagia Symptom Response (Binary Response) at the Final Treatment Period Evaluation (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. | FAS included all randomized participants who received at least one dose of a double-blind IP. | Posted | Count of Participants | Participants | Week 16 |
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| Secondary | Number of Participants With Overall Binary Response I at the Final Treatment Period Evaluation (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. | FAS included all randomized participants who received at least one dose of a double-blind IP. | Posted | Count of Participants | Participants | Week 16 |
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| Secondary | Number of Participants With Overall Binary Response II at the Final Treatment Period Evaluation (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. | FAS included all randomized participants who received at least one dose of a double-blind IP. | Posted | Count of Participants | Participants | Week 16 |
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| Secondary | Change From Baseline in the Dysphagia Symptom Questionnaire (DSQ) + Pain Score (Questions 2 +3+4) at 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 + 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. | FAS included all randomized participants who received at least one dose of a double-blind IP. Here the number of participants analyzed signifies participants who were evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | Score on scale | Baseline, Week 16 |
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| Secondary | Change From Baseline in the Dysphagia Symptom Questionnaire (DSQ) Pain Score (Question 4) at the Final Treatment Period Evaluation (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. | FAS included all randomized participants who received at least one dose of a double-blind IP. Here the number of participants analyzed signifies participants who were evaluable for this outcome measure. | Posted | Least Squares Mean | Standard Error | score on scale | Baseline, Week 16 |
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| Secondary | Number of Participants With Treatment-Emergent Adverse Events (AE) | 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. | Safety analysis set included all participants who received at least one dose of any double-blind IP. | Posted | Count of Participants | Participants | From start of study drug administration up to follow-up (Week 20) |
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| Secondary | Area Under the Plasma Concentration-Time Curve (AUCtau) Between the Defined Interval of Budesonide Doses | 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. | PK set included all participants in the safety set who received BOS treatment and provided at least one quantifiable plasma concentration of budesonide. Here the number of participants analyzed signifies participants who were evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | h.pg/mL | Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 |
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| Secondary | Maximum Observed Concentration (Cmax) of Budesonide in Plasma | The Cmax of budesonide in plasma was reported. | PK set included all participants in the safety set who received BOS treatment and provided at least one quantifiable plasma concentration of budesonide. Here the number of participants analyzed signifies participants who were evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Picograms per milliliter (pg/mL) | Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 |
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| Secondary | Time to Maximum Observed Plasma Concentration (Tmax) of Budesonide in Plasma | Tmax of budesonide in plasma was reported. | PK Set included all participants in the safety set who received BOS treatment and provided at least one quantifiable plasma concentration of budesonide. Here the number of participants analyzed signifies participants who were evaluable for this outcome measure. | Posted | Median | Full Range | hour | Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 |
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| Secondary | Terminal Rate Constant (Lambda Z) of Budesonide in Plasma | Lambda Z of budesonide in plasma was reported. | PK set included all participants in the safety set who received BOS treatment and provided at least one quantifiable plasma concentration of budesonide. Here the number of participants analyzed signifies participants who were evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | per hour | Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 |
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| Secondary | Terminal Half-Life (t1/2) of Budesonide in Plasma | t1/2 of budesonide in plasma was reported | PK set included all participants in the safety set who received BOS treatment and provided at least one quantifiable plasma concentration of budesonide. Here the number of participants analyzed signifies participants who were evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | hour | Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 |
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| Secondary | Apparent Oral Clearance (CL/F) of Budesonide in Plasma | CL/F of budesonide in plasma was reported. | PK set included all participants in the safety set who received BOS treatment and provided at least one quantifiable plasma concentration of budesonide. Here the number of participants analyzed signifies participants who were evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Liter per hour (L/h) | Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 |
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| Secondary | Apparent Volume of Distribution (Vz/F) of Budesonide in Plasma | Vz/F of budesonide in plasma was reported. | PK set included all participants in the safety set who received BOS treatment and provided at least one quantifiable plasma concentration of budesonide. Here the number of participants analyzed signifies participants who were evaluable for this outcome measure. | Posted | Geometric Mean | Geometric Coefficient of Variation | Liter (L) | Pre-dose, 0.5, 1, 2, 3, 4, 6, 8, and 12 hours post-dose on Week 8, 12 and 16 |
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From start of study drug administration up to follow-up (Week 20)
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
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| EG000 | Placebo | Participants received oral dose of 10 milliliter (ml) placebo matched with the Oral Budesonide Suspension (OBS) twice daily up to 16 weeks. | 0 | 105 | 1 | 105 | 28 | 105 |
| EG001 | Oral Budesonide Suspension (OBS) | Post placebo lead in phase (4 weeks) participants received OBS 10 ml (2 mg) twice daily up to 12 weeks. | 0 | 213 | 2 | 213 | 63 | 213 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Localised intraabdominal fluid collection | Gastrointestinal disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Gastroenteritis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Sepsis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Ovarian cyst | Reproductive system and breast disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Oesophagitis | Gastrointestinal disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Vomiting | Gastrointestinal disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Oesophageal candidiasis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Oral candidiasis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Sinusitis | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Upper respiratory tract infection | Infections and infestations | MedDRA 18.0 | Non-systematic Assessment |
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| Procedural pain | Injury, poisoning and procedural complications | MedDRA 18.0 | Non-systematic Assessment |
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| ACTH stimulation test abnormal | Investigations | MedDRA 18.0 | Non-systematic Assessment |
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| Protein urine present | Investigations | MedDRA 18.0 | Non-systematic Assessment |
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| Urine leukocyte esterase positive | Investigations | MedDRA 18.0 | Non-systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Insomnia | Psychiatric disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Mood swings | Psychiatric disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Acne | Skin and subcutaneous tissue disorders | MedDRA 18.0 | Non-systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 18.0 | Non-systematic Assessment |
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If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Shire | +1 866 842 5335 | ClinicalTransparency@shire.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Mar 11, 2019 | Jan 21, 2020 | SAP_001.pdf |
| ID | Term |
|---|---|
| D057765 | Eosinophilic Esophagitis |
| ID | Term |
|---|---|
| D004941 | Esophagitis |
| D004935 | Esophageal Diseases |
| D005767 | Gastrointestinal Diseases |
| D004066 | Digestive System Diseases |
| D005759 | Gastroenteritis |
| D004802 | Eosinophilia |
| D007960 | Leukocyte Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006969 | Hypersensitivity, Immediate |
| D006967 | Hypersensitivity |
| D007154 | Immune System Diseases |
Not provided
Not provided
| Male |
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| Not Hispanic or Latino |
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| Unknown or Not Reported |
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| Asian |
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| Native Hawaiian or Other Pacific Islander |
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| Black or African American |
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| White |
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| More than one race |
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| Unknown or Not Reported |
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Post placebo lead in phase (4 weeks) participants received OBS 10 ml (2 mg) twice daily up to 12 weeks. |
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Post placebo lead in phase (4 weeks) participants received OBS 10 ml (2 mg) twice daily up to 12 weeks. |
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