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An Extension Study to Evaluate Maintenance of Efficacy and Long-term Treatment Effect of Oral Budesonide Suspension (OBS) in Adults and Adolescents With Eosinophilic Esophagitis (EoE) (ORBIT2)

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ClinicalTrials.gov Identifier: NCT02736409
Recruitment Status : Completed
First Posted : April 13, 2016
Results First Posted : January 13, 2021
Last Update Posted : January 13, 2021
Sponsor:
Information provided by (Responsible Party):
Takeda ( Shire )

Brief Summary:
This is a multicenter, double- blind extension study of Oral Budesonide Suspension (OBS) in adults and adolescents (11 to 55 years of age, inclusive) with Eosinophilic Esophagitis (EoE) who have completed participation in the SHP621-301 induction study (NCT02605837). The primary objective is to evaluate the maintenance of efficacy of OBS over 36 weeks. Maintenance of efficacy will be measured by the peak eosinophilic count and Dysphagia Symptom Questionnaire (DSQ) score.

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

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 219 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 3, Multicenter, Double-blind Extension Study to Evaluate Maintenance of Efficacy of Oral Budesonide Suspension (OBS) and Long-term Treatment Effect of OBS in Adolescent and Adult Subjects (11 to 55 Years of Age, Inclusive) With Eosinophilic Esophagitis (EoE)
Actual Study Start Date : April 1, 2016
Actual Primary Completion Date : November 12, 2019
Actual Study Completion Date : November 12, 2019

Resource links provided by the National Library of Medicine

Drug Information available for: Budesonide

Arm Intervention/treatment
Experimental: Arms A OBS Completers/ Responders
Arm A Oral Budesonide Suspension Completers/ Responders
Drug: Oral Budesonide Suspension (OBS)
OBS 2mg twice daily

Placebo Comparator: Arm B OBS Completers/ Responders
Arm B Oral Budesonide Suspension Completers/ Responders. 1:1 randomization for Arms A and B
Drug: Placebo
Matching Placebo dose

Experimental: Arm C OBS Completers/ Non-Responders
Arm C Oral Budesonide Suspension Completers/ Non-Responders
Drug: Oral Budesonide Suspension (OBS)
OBS 2mg twice daily

Experimental: Arm D Placebo Completers
Arm D Placebo Completers
Drug: Oral Budesonide Suspension (OBS)
OBS 2mg twice daily




Primary Outcome Measures :
  1. Proportion of Participants Who Had Relapse During the Entire Week 36 Period [ Time Frame: Week 36 ]
    Relapse (Yes/No) was defined as meeting both the eosinophil histology relapse criterion and the dysphagia symptom relapse criterion. Eosinophil histology relapse was defined as an eosinophil count of greater than or equal to(> or =) 15 per high-power field (eos/hpf) from at least 2 of 3 levels of the esophagus. Dysphagia symptom relapse was defined as having at least 4 days of dysphagia (with answer 'Yes' for question 2 in DSQ [Dysphagia Symptom Questionnaire]) in the 2-week period prior to the scheduled visit, as determined by the DSQ.


Secondary Outcome Measures :
  1. Proportion of Participants With Long-term Treatment Response From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study [ Time Frame: Week 36 ]
    Long-term response from SHP621-301 baseline defined: histologic response,defined as peak eosinophil count of less than or equal to(< or =)6/HPF across all available esophageal levels at Week 36 and Dysphagia symptom response 1,defined as >or= 30 percent(%) reduction in DSQ combined score(Questions [Q] 2+3) from baseline of SHP621-301 to Week 36.DSQcontain 4 questions,all participants used diary,responded to Q1(didyoueatsolid food),Q2(did food pass slowly or get stuck). If participant's answer to Q2 was No,diary ended for day. If participant answered Yes,he/she advanced to Q3(didyouhavetodoanything to make food go downorget relief), Q4(extent to which participant experienced pain while swallowing).DSQ combined score= ([sum of points from Q2+3 in daily DSQ]×14)/Number of diaries reported with non-missing data.Scale range was0-2 forQ2 and 0-4 forQ 3.Scale range for DSQ combined score was 0-84.Highervaluesrepresentingworseoutcome.Negative change from baseline indicates symptoms decreased.

  2. Proportion of Participants With Long-term Treatment Response From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36 [ Time Frame: Week 36 ]
    Long-term response from baseline defined: histologic response, defined as peak eosinophil count of less than or equal to(< or =) 6/HPF across all available esophageal levels at Week 36 and Dysphagia symptom response 1,defined as >or= 30 percent(%) reduction in DSQ combined score (Questions [Q] 2+3) from baseline of SHP621-301 to Week 36.DSQcontain 4 questions,all participants used diary,responded to Q1(did you eat solid food),Q2(did food pass slowly or get stuck). If participant's answer to Q2 was No,diary ended for day. If participant answered Yes,he/she advanced to Q3(didyouhavetodoanything to make food go downorget relief), Q4(extent to which participant experienced pain while swallowing).DSQ combined score= ([sum of points from Q2+3 in daily DSQ]×14)/Number of diaries reported with non-missing data.Scale range was0-2 forQ2 and 0-4 forQ 3.Scale range for DSQ combined score was 0-84. Higher values representing worse outcome.Negative change from baseline indicates symptoms decreased.

  3. Proportion of Participants Who Had a Histologic Response (Eosinophil Count of Less Than or Equal to (<or=6)/High-Powered Field [HPF]) at Week 12 and Week 36 [ Time Frame: Week 12 and Week 36 ]
    Histology response was defined as a peak eosinophil count of <or=6/HPF across all available levels at Week 12 and Week 36.

  4. Proportion of Participants Who Had at Least a 30 Percent (%) Change in DSQ Combined Score From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 12 and Week 36 [ Time Frame: Baseline of SHP621-301 (NCT02605837), Week 12 and Week 36 ]
    Dysphagia symptom response with respect to the baseline of induction study (SHP621-301 [NCT02605837]) was defined as 30% reduction in DSQ combined score (questions 2+3) at Week 12 and Week 36 of current study from baseline of the induction study. DSQ contain 4 questions, all participants used a diary and responded to Q1 (did you eat solid food), Q2 (did food pass slowly or get stuck). If participant's answer to Q2 was No, then diary ended for that day. If participant answered Yes,he/she advanced to Q3 (did you have to do anything to make food go down or get relief) and Q4 (extent to which participant experienced pain while swallowing). DSQ combined score= ([sum of points from Q2+3 in daily DSQ] × 14)/Number of diaries reported with non-missing data. Scale range was 0-2 for Q2 and 0-4 for Q3. Scale range for DSQ combined score was 0-84. Higher values representing worse outcome. Negative change from baseline indicates symptoms decreased.

  5. Proportion of Participants Who Had at Least a 30 Percent (%) Change in DSQ Combined Score From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 12 and Week 36 [ Time Frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 12 and Week 36 ]
    Dysphagia symptom response with respect to the baseline of current study (SHP621-302 [NCT02736409]) was defined as 30% reduction in DSQ combined score (questions 2+3) at Week 12 and Week 36 from baseline of the current study. DSQ contain 4 questions, all participants used diary and responded to Q1 (did you eat solid food), Q2 (did food pass slowly or get stuck). If participant's answer to Q2 was "No", then diary ended for that day. If participant answered "Yes",he/she advanced to Q3 (did you have to do anything to make food go down or get relief) and Q4 (extent to which participant experienced pain while swallowing). DSQ combined score= ([sum of points from Q2+3 in daily DSQ] × 14)/Number of diaries reported with non-missing data. Scale range was 0-2 for Q2 and 0-4 for Q 3. Scale range for DSQ combined score was 0-84. Higher values representing a worse outcome. A Negative change from baseline indicates symptoms decreased.

  6. Change From Induction Study (SHP621-301 [NCT02605837]) Baseline in Total Endoscopy Score at Week 12 and Week 36 of Current Study [ Time Frame: Baseline of induction study (SHP621-301 [NCT02605837]), Week 12 and Week 36 ]
    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.

  7. Change From Current Study (SHP621-302 [NCT02736409]) Baseline in Total Endoscopy Score at Week 12 and Week 36 [ Time Frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 12 and Week 36 ]
    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.

  8. Change From Induction Study (SHP621-301 [NCT02605837]) Baseline in Peak Eosinophil Count at Week 12 and Week 36 of Current Study [ Time Frame: Baseline of induction study (SHP621-301 [NCT02605837]), Week 12 and Week 36 ]
    Change from induction study (SHP621-301 [NCT02605837]) baseline in peak eosinophil count at Week 12 and Week 36 of current study was reported.

  9. Change From Current Study (SHP621-302 [NCT02736409]) Baseline in Peak Eosinophil Count at Week 12 and Week 36 [ Time Frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 12 and Week 36 ]
    Change from current study (SHP621-302 [NCT02736409]) baseline in peak eosinophil count at Week 12 and Week 36 was reported.

  10. Proportion of Participants Who Had Peak Eosinophil Count Less Than (<) 15/High-Powered Field (HPF) at Week 12 and Week 36 [ Time Frame: Week 12 and Week 36 ]
    Proportion of participants who had Peak Eosinophil Count less than (<) 15/HPF at Week 12 and Week 36 were reported.

  11. Proportion of Participants Who Had Peak Eosinophil Count Less Than or Equal to (< or =) 1/High-Powered Field (HPF) at Week 12 and Week 36 [ Time Frame: Week 12 and Week 36 ]
    Proportion of participants who had Peak Eosinophil Count less than or equal to (< or =) 1/High-Powered Field (HPF) at Week 12 and Week 36 were reported.

  12. Change From Induction Study (SHP621-301 [NCT02605837]) Baseline in Peak Eosinophil Count for Each Available Esophageal Level (Proximal, Mid, and Distal) at Week 12 and Week 36 of Current Study [ Time Frame: Baseline of induction study (SHP621-301 [NCT02605837]), Week 12 and Week 36 ]
    Change from induction study (SHP621-301 [NCT02605837]) baseline in peak eosinophil count for each available esophageal level (proximal, mid, distal) at Week 12 and Week 36 of current study were reported.

  13. Change From Current Study (SHP621-302 [NCT02736409]) Baseline in Peak Eosinophil Count for Each Available Esophageal Level (Proximal, Mid, and Distal) at Week 12 and Week 36 [ Time Frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 12 and Week 36 ]
    Change from current study (SHP621-302 [NCT02736409]) baseline in peak eosinophil count for each available esophageal level (proximal, mid, distal) at Week 12 and Week 36 were reported.

  14. Change From Induction Study (SHP621-301 [NCT02605837]) Baseline in the Histopathologic Epithelial Features Combined Total Score (Grade and Stage) at Week 12 an Week 36 of Current Study [ Time Frame: Baseline of induction study (SHP621-301 [NCT02605837]), Week 12 and Week 36 ]
    Eight histopathologic epithelial features (basal layer hyperplasia, eosinophil peak, abscesses, surface layering, dilated intercellular spaces, surface alteration, dyskeratotic epithelial cells, lamina propria fibrosis) are scored on a 4-point scale (0=normal, 3=worst) for both the severity of the abnormality (i.e., grade) and the amount of tissue affected by the abnormality (i.e. 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.

  15. Change From Current Study (SHP621-302 [NCT02736409]) Baseline in the Histopathologic Epithelial Features Combined Total Score (Grade and Stage) at Week 12 and Week 36 [ Time Frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 12 and Week 36 ]
    Eight histopathologic epithelial features (basal layer hyperplasia, eosinophil peak, abscesses, surface layering, dilated intercellular spaces, surface alteration, dyskeratotic epithelial cells, lamina propria fibrosis) are scored on a 4-point scale (0=normal, 3=worst) for both the severity of the abnormality (i.e., grade) and the amount of tissue affected by the abnormality (i.e. 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.

  16. Proportion of Participants Who Had Greater Than or Equal to (>or=) 50 Percent (%) Reduction in DSQ Combined Score From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 12 and Week 36 [ Time Frame: Week 12 and Week 36 ]
    Dysphagia symptom response from the baseline of SHP621-301, was defined as >or=50% reduction in DSQ combined score (questions [Q] 2+3) from baseline of the induction study at Week 12 and 36 of current study. DSQ contained 4Q, all participants used diary, and responded to Q1(did you eat solid food) and Q2(didfood passslowly or get stuck). If participant's answer to Q2 was 'No', then diary ended for that day. If a participant answered 'Yes', he/she advanced to Q3(did you have to do anything to make food go down or get relief) and 4(extent to which participant experienced pain while swallowing). DSQ combined score= ([sum of points from Q2+3 in daily DSQ]×14)/ Number of diaries reported with non-missing data. Scale range was 0-2 for Q2 and 0-4 for Q3, with higher values representing a worse outcome. Scale range for DSQ combined score was 0-84, with higher values representing a worse outcome. Anegative change from baseline indicates that symptoms decreased.

  17. Proportion of Participants Who Had Greater Than or Equal to (>or=) 50 Percent (%) Reduction in DSQ Combined Score From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 12 and Week 36 [ Time Frame: Week 12 and Week 36 ]
    Dysphagia symptom response from the baseline of SHP621-302, was defined as >or=50% reduction in DSQ combined score (questions 2+3) from baseline of the induction study at Week 36 of current study. DSQ contained 4Q, all participants used diary, and responded to Q1 (did you eat solid food) and Q2 (did food pass slowly or get stuck). If participant's answer to Q2 was 'No', then diary ended for that day. If a participant answered 'Yes', he/she advanced to Q3 (did you have to do anything to make food go down or get relief) and 4(extent to which participant experienced pain while swallowing). DSQ combined score= ([sum of points from Q2+3 in daily DSQ]×14)/ Number of diaries reported with non-missing data. Scale range was 0-2 for Q2 and 0-4 for Q3, 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.

  18. Change in the DSQ Combined Score (Questions 2+3) From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study [ Time Frame: Baseline of induction study (SHP621-301 [NCT02605837]), Week 36 ]
    DSQ contain 4 questions (Q), all participants used a diary, responded to Q1 (did you eat solid food), Q2 (did food pass slowly or get stuck). If participant's answer to Q2 was 'No', then diary ended for that day. If participant answered 'Yes', he/she advanced to Q3 (did you have to do anything to make food go down or get relief), Q4 (extent to which participant experienced pain while swallowing). DSQ combined score= ([sum of points from Q2+3 in daily DSQ] × 14)/Number of diaries reported with non-missing data. Scale range was 0-2 for Q2 and 0-4 for Q3. Scale range for DSQ combined score was 0-84. Higher values representing a worse outcome. A Negative change from baseline indicates symptoms decreased.

  19. Change in the DSQ Combined Score (Questions 2+3) From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36 [ Time Frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 36 ]
    DSQ contain 4 questions, all participants used a diary, responded to Q1 (did you eat solid food), Q2 (did food pass slowly or get stuck). If participant's answer to Q2 was 'No', then diary ended for that day. If participant answered 'Yes', he/she advanced to Q3 (did you have to do anything to make food go down or get relief), Q4 (extent to which participant experienced pain while swallowing). DSQ combined score= ([sum of points from Q2+3 in daily DSQ] × 14)/Number of diaries reported with non-missing data. Scale range was 0-2 for Q2 and 0-4 for Q3. Scale range for DSQ combined score was 0-84. Higher values representing a worse outcome. A Negative change from baseline indicates symptoms decreased. Change in DSQ combined score (Questions 2+3) from baseline of current study at Week 36 was reported.

  20. Percent Change in the DSQ Combined Score (Questions 2+3) From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study [ Time Frame: Baseline of induction study (SHP621-301 [NCT02605837]), Week 36 ]
    Percent change in DSQ combined score from Induction Study (SHP621-301 [NCT02605837]) baseline to current study (SHP621-302 [NCT02736409]) final treatment period (Week 36) was reported.

  21. Percent Change in the DSQ Combined Score (Questions 2+3) From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36 [ Time Frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 36 ]
    Percent change in DSQ combined score from baseline to final treatment period (Week 36) of current study was reported.

  22. Proportion of Participants Who Had Overall Binary Response I From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 12 and Week 36 of Current Study [ Time Frame: Week 12 and Week 36 ]
    Overall binary response I was defined as if participant had peak eosinophil count of <=6/HPF across all esophagus levels and achieved a minimum of 30% reduction in DSQ combined score from baseline of the induction study (SHP621-301 [NCT02605837]) at Week 12 and Week 36. 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.

  23. Proportion of Participants Who Had Overall Binary Response I From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 12 and Week 36 [ Time Frame: Week 12 and Week 36 ]
    Overall binary response I was defined as if participant had peak eosinophil count of <=6/HPF across all esophagus levels and achieved a minimum of 30% reduction in DSQ combined score from baseline at Week 12 and Week 36. 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.

  24. Proportion of Participants Who Had Overall Binary Response II From Induction Study SHP621-301 (NCT02605837) Baseline at Week 12 and Week 36 of Current Study [ Time Frame: Week 12 and Week 36 ]
    Overall binary response II was defined as if participant had peak eosinophil count of <=6/HPF across all esophagus levels and achieved a minimum of 50% reduction in DSQ combined score from baseline of the SHP621-301 study at Week 12 and Week 36. 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.

  25. Proportion of Participants Who Had Overall Binary Response II From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 12 and Week 36 [ Time Frame: Week 12 and Week 36 ]
    Overall binary response II was defined as if participant had peak eosinophil count of <=6/HPF across all esophagus levels and achieved a minimum of 30% reduction in DSQ combined score from baseline at Week 12 and Week 36. 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.

  26. Change in the DSQ + Pain Score (Questions 2+3+4) From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study [ Time Frame: Baseline of induction study (SHP621-301 [NCT02605837]), Week 36 ]
    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.

  27. Change in the DSQ+ Pain Score (Questions 2+3+4) From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36 [ Time Frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 36 ]
    DSQ contained 4 questions, all participants used a diary, and responded to Questions (Q) 1 (did you eat solid food) and 2 (did food pass slowly or get stuck). If the participant's answer to Q2 was 'No', the diary ended for that day. If a participant answered 'Yes', he/she advanced to Q3 (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. Question 1 was excluded from the DSQ + pain score. DSQ + pain score=(Sum of points from questions 2+3+4 in the daily DSQ)*14 Days/Number of dairies reported with non-missing data. Scale range was 0 - 2 for Q2, 0 - 4 for Q3 and 0 - 4 for Q4, 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.

  28. Change in the DSQ Pain Score (Question 4) From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study [ Time Frame: Baseline of induction study (SHP621-301 [NCT02605837]), Week 36 ]
    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 Question 4 only. DSQ pain score=(sum of points from questions 4 in the daily DSQ)*14 days/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.

  29. Change in the DSQ Pain Score (Question 4) From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36 [ Time Frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 36 ]
    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 Question 4 only. DSQ pain score=(sum of points from questions 4 in the daily DSQ)*14 days/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.

  30. Number of Participants With Treatment Emergent Adverse Events (TEAE's) [ Time Frame: From start of the study drug administration up to follow up (Week 40) ]
    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 started or deteriorated on or after the date of the first dose of double-blind IP in SHP621-302 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.

  31. Change in DXA (Dual-Energy X-ray Absorptiometry) Imaging Results (Location: Lumbar Spine [L1-L4]) From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study [ Time Frame: Baseline of induction study (SHP621-301 [NCT02605837]), Week 36 ]
    The sites for DXA measurement were the lumber spine (L1-L4 preferred) and total body less head. DXA scans for bone mineral density (BMD) and body composition measurements were done only for adolescent participants aged 11-17 years based on the age group in the SHP621-301 study and were completed throughout the SHP621-302 study even if participants turned 18 years. Baseline of SHP621-301 is determined at Week 0 in the SHP621-301 study. Here in this outcome measure DXA measurement of L1-L4 were reported. Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. A Z-score of 0 is equal to the mean. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean.

  32. Change in DXA Imaging Results (Location: Lumbar Spine [L1-L4]) From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36 [ Time Frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 36 ]
    The sites for DXA measurement were the lumber spine (L1-L4 preferred) and total body less head. DXA scans for bone mineral density (BMD) and body composition measurements were done only for adolescent participants aged 11-17 years based on the age group in the SHP621-301 study and were completed throughout the SHP621-302 study even if participants turned 18 years. Here in this outcome measure DXA measurement of L1-L4 were reported. Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. A Z-score of 0 is equal to the mean. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean.

  33. Change in DXA Imaging Results (Location: Whole Body) From Baseline of Induction Study (SHP621-301 [NCT02605837]) at Week 36 of Current Study [ Time Frame: Baseline of induction study (SHP621-301 [NCT02605837]), Week 36 ]
    The sites for DXA measurement were the lumber spine (L1-L4 preferred) and total body less head. DXA scans for bone mineral density (BMD) and body composition measurements were done only for adolescent participants aged 11-17 years based on the age group in the SHP621-301 study and were completed throughout the SHP621-302 study even if participants turned 18 years. Baseline of SHP621-301 is determined at Week 0 in the SHP621-301 study. Here in this outcome measure DXA measurement of whole body (except head) were reported. Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. A Z-score of 0 is equal to the mean. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean.

  34. Change in DXA Imaging Results (Location: Whole Body) From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36 [ Time Frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 36 ]
    The sites for DXA measurement were the lumber spine (L1-L4 preferred) and total body less head. DXA scans for bone mineral density (BMD) and body composition measurements were done only for adolescent participants aged 11-17 years based on the age group in the SHP621-301 study and were completed throughout the SHP621-302 study even if participants turned 18 years. Here in this outcome measure DXA measurement of whole body (except head) were reported. Z-score indicates the number of standard deviations away from a reference population in the same age range and with the same sex. A Z-score of 0 is equal to the mean. Negative numbers indicate values lower than the mean and positive numbers indicate values higher than the mean.


Other Outcome Measures:
  1. Proportion of Participants Who Had a Histologic Response (Eosinophil Count of Greater Than or Equal to (>or=15)/High-Powered Field [HPF]) Before or at Week 12 and Before or at Week 36 [ Time Frame: Week 12 and Week 36 ]
    Eosinophil histology relapse was defined as an eosinophil count of >or=15 eos/HPF from at least 2 of 3 levels of the esophagus.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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

Inclusion Criteria:

  1. Subject completed SHP621-301 induction study.
  2. Subject is able to provide written informed consent (subject, parent or legal guardian and, as appropriate, subject assent) to participate in the study before completing any study-related procedures.
  3. Subject is male or female aged 11-55 years, inclusive, at time of consent for SHP621-301 study.
  4. Subject is willing and able to continue any dietary therapy, environmental therapy, and/or medical regimens (including gastric acid suppression; see exclusions below) in effect at the screening visit (Visit 0). There should be no changes to these regimens during study participation.
  5. All female subjects must have a negative serum pregnancy test (beta-human chorionic gonadotropin [β-hCG]) prior to enrollment into the study. Females of childbearing potential must agree to continue acceptable birth control measures (eg, abstinence, stable oral contraceptives, or double-barrier methods) throughout study participation and for 30 days following the last dose of investigational product.
  6. Subject is willing and has an understanding and ability to fully comply with study procedures including DSQ compliance (completed the DSQ on ≥70% of days in any 2 consecutive weeks of the screening period)and restrictions defined in this protocol

Exclusion Criteria:

  1. Subject has changes in medications that could affect the study or diet in the weeks since the final treatment evaluation visit (Visit 4) of the SHP621-301 study.
  2. Subject using immunomodulatory therapy since the final treatment evaluation visit (Visit 4) of the SHP621-301 study or anticipated use of immunomodulatory therapy during the treatment period (except for any ongoing regimen of allergy shots); any temporary use (≤7 days) or initiation of new steroid treatment during the study should be documented and discussed with the medical monitor prospectively but cannot occur within 4 weeks of scheduled EGDs.
  3. Subject using swallowed topical corticosteroid for EoE or systemic corticosteroid for any condition since the final treatment evaluation visit (Visit 4) of the SHP621-301 study or anticipated use during the treatment period; any temporary use (≤7 days) or initiation of new steroid treatment during the study should be documented and discussed with medical monitor prospectively but cannot occur within the 4 weeks of the scheduled EGDs.
  4. Subject on inhaled or intranasal steroids and not on a stable dose between the baseline visit (Visit 1) of the SHP621-301 study and the screening EGD of this study.
  5. Subject has initiated, discontinued, or changed dosage regimen of proton pump inhibitors (PPIs), H2 antagonists, antacids, antihistamines, or leukotriene inhibitors for any condition (such as gastroesophageal reflux disease, asthma or allergic rhinitis) since the final treatment evaluation visit (Visit 4) of the SHP621-301 study or anticipated changes in the use of such medications during the treatment period.
  6. Subject using Cytochrome P450 3A4 inhibitors (eg, ketoconazole, grapefruit juice) since the final treatment evaluation visit (Visit 4) of the SHP621-301 study or anticipated use of such medications during the treatment period.
  7. Subject has an appearance on screening EGD of an esophageal stricture (high grade), as defined by the presence of a lesion that does not allow passage of a diagnostic adult upper endoscope (eg, with an insertion tube diameter of >9mm).
  8. Subject is on a pure liquid diet or the six-food elimination diet.
  9. Subject has presence of esophageal varices at the EGD at the final treatment evaluation visit (Visit 4) of the SHP621-301 study.
  10. Subject has any current disease of the gastrointestinal tract, aside from EoE, including eosinophilic gastritis, enteritis, colitis, or proctitis, inflammatory bowel disease, or celiac disease.
  11. Subject has other diseases causing or associated with esophageal eosinophilia, including hypereosinophilic syndrome, collagen vascular disease, vasculitis, achalasia, or parasitic infection.
  12. Subject 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 4) of the SHP621-301 study is not an exclusion as long as the subject received treatment for candidiasis and is expected to respond to treatment.

  13. Subject has acute or chronic infection or immunodeficiency condition, including tuberculosis, fungal, bacterial, viral/parasite infection, ocular herpes simplex, or chicken pox/measles.
  14. Subject has upper gastrointestinal bleeding identified in the EGD at the final treatment evaluation visit (Visit 4) of the SHP621-301 study or since the final treatment evaluation visit (Visit 4) of the SHP621-301 study.
  15. Subject has evidence of active infection with Helicobacter pylori.
  16. Subject has evidence of unstable asthma since the final treatment evaluation visit (Visit 4) of the SHP621-301 study.
  17. Subject is female and pregnant or nursing.
  18. Subject has a history of intolerance, hypersensitivity, or idiosyncratic reaction to budesonide (or any other corticosteroids), or to any other ingredients of the study medication.
  19. Subject has a history or high risk of noncompliance with treatment or regular clinic visits.
  20. Subject is on sucralfate or anticipates using sucralfate during the treatment period.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02736409


Locations
Show Show 62 study locations
Sponsors and Collaborators
Shire
Investigators
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Study Director: Study Director Shire
  Study Documents (Full-Text)

Documents provided by Takeda ( Shire ):
Study Protocol  [PDF] December 19, 2016
Statistical Analysis Plan  [PDF] December 4, 2019

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT02736409    
Other Study ID Numbers: SHP621-302
First Posted: April 13, 2016    Key Record Dates
Results First Posted: January 13, 2021
Last Update Posted: January 13, 2021
Last Verified: December 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
URL: https://vivli.org/ourmember/takeda/
Additional relevant MeSH terms:
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Esophagitis
Eosinophilic Esophagitis
Esophageal Diseases
Gastrointestinal Diseases
Digestive System Diseases
Gastroenteritis
Eosinophilia
Leukocyte Disorders
Hematologic Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Budesonide
Anti-Inflammatory Agents
Bronchodilator Agents
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anti-Asthmatic Agents
Respiratory System Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists