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CompletedNCT02736409ORBIT2Updated Feb 19, 2025Results posted

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)

A Phase 3 interventional study of Oral Budesonide Suspension (OBS) and Placebo in Eosinophilic Esophagitis (EoE), sponsored by Shire. Completed at 62 sites in United States. Open to participants aged 11 Years to 55 Years. Per ClinicalTrials.gov, last updated 2025-02-19.

Sponsored by Shire (part of Takeda) · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
219
Allocation
Randomized
Ages
11 Years to 55 Years
Sex
All
01

Study 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.

02

Conditions studied

  • Eosinophilic Esophagitis (EoE)
03

In context

Eosinophilic Esophagitis

238 studies on the registry are indexed under Eosinophilic Esophagitis; 52 are open to participants now.

This study's enrollment of 219 is above the median of 41 across 136 interventional studies indexed under Eosinophilic Esophagitis.

Browse Eosinophilic Esophagitis studies →

Lead sponsor

Shire is the lead sponsor of 351 studies on the registry; 2 are open to participants now.

Of its 48 completed or terminated interventional studies of FDA-regulated products, 48 (100%) have results posted.

First submitted before Shire became part of Takeda.

Browse Shire studies →All Takeda studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
11 Years to 55 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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

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.
05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Double (Participant, Investigator)
Enrollment
219 participants (actual)

Study arms

  • Experimental
    Arms A OBS Completers/ Responders

    Arm A Oral Budesonide Suspension Completers/ Responders

    Drug: Oral Budesonide Suspension (OBS)

  • Placebo comparator
    Arm B OBS Completers/ Responders

    Arm B Oral Budesonide Suspension Completers/ Responders. 1:1 randomization for Arms A and B

    Drug: Placebo

  • Experimental
    Arm C OBS Completers/ Non-Responders

    Arm C Oral Budesonide Suspension Completers/ Non-Responders

    Drug: Oral Budesonide Suspension (OBS)

  • Experimental
    Arm D Placebo Completers

    Arm D Placebo Completers

    Drug: Oral Budesonide Suspension (OBS)

Interventions

06

What researchers measure

Primary outcomes

  1. Proportion of Participants Who Had Relapse During the Entire Week 36 Period

    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.

    Time frame: Week 36

Secondary outcomes

  1. Proportion of Participants With Long-term Treatment Response From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study

    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.

    Time frame: Week 36

  2. Proportion of Participants With Long-term Treatment Response From Current Study (SHP621-302 [NCT02736409]) Baseline at 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.

    Time frame: Week 36

  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

    Histology response was defined as a peak eosinophil count of \<or=6/HPF across all available levels at Week 12 and Week 36.

    Time frame: 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

    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.

    Time frame: Baseline of SHP621-301 (NCT02605837), Week 12 and Week 36

  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

    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.

    Time frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 12 and Week 36

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

    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.

    Time frame: Baseline of induction study (SHP621-301 [NCT02605837]), Week 12 and Week 36

  7. Change From Current Study (SHP621-302 [NCT02736409]) Baseline in Total Endoscopy Score at 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.

    Time frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 12 and Week 36

  8. Change From Induction Study (SHP621-301 [NCT02605837]) Baseline in Peak Eosinophil Count at Week 12 and Week 36 of Current Study

    Change from induction study (SHP621-301 \[NCT02605837\]) baseline in peak eosinophil count at Week 12 and Week 36 of current study was reported.

    Time frame: Baseline of induction study (SHP621-301 [NCT02605837]), Week 12 and Week 36

  9. Change From Current Study (SHP621-302 [NCT02736409]) Baseline in Peak Eosinophil Count at 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.

    Time frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 12 and Week 36

  10. Proportion of Participants Who Had Peak Eosinophil Count Less Than (<) 15/High-Powered Field (HPF) at 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.

    Time frame: Week 12 and Week 36

  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

    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.

    Time frame: Week 12 and Week 36

  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

    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.

    Time frame: Baseline of induction study (SHP621-301 [NCT02605837]), Week 12 and Week 36

  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

    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.

    Time frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 12 and Week 36

  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

    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.

    Time frame: Baseline of induction study (SHP621-301 [NCT02605837]), Week 12 and Week 36

  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

    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.

    Time frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 12 and Week 36

  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

    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.

    Time frame: Week 12 and Week 36

  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

    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.

    Time frame: Week 12 and Week 36

  18. Change in the DSQ Combined Score (Questions 2+3) From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study

    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.

    Time frame: Baseline of induction study (SHP621-301 [NCT02605837]), Week 36

  19. Change in the DSQ Combined Score (Questions 2+3) From Current Study (SHP621-302 [NCT02736409]) Baseline at 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.

    Time frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 36

  20. Percent Change in the DSQ Combined Score (Questions 2+3) From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study

    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.

    Time frame: Baseline of induction study (SHP621-301 [NCT02605837]), Week 36

  21. Percent Change in the DSQ Combined Score (Questions 2+3) From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36

    Percent change in DSQ combined score from baseline to final treatment period (Week 36) of current study was reported.

    Time frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 36

  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

    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.

    Time frame: Week 12 and Week 36

  23. Proportion of Participants Who Had Overall Binary Response I From Current Study (SHP621-302 [NCT02736409]) Baseline at 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.

    Time frame: Week 12 and Week 36

  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

    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.

    Time frame: Week 12 and Week 36

  25. Proportion of Participants Who Had Overall Binary Response II From Current Study (SHP621-302 [NCT02736409]) Baseline at 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.

    Time frame: Week 12 and Week 36

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

    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.

    Time frame: Baseline of induction study (SHP621-301 [NCT02605837]), Week 36

  27. Change in the DSQ+ Pain Score (Questions 2+3+4) From Current Study (SHP621-302 [NCT02736409]) Baseline at 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.

    Time frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 36

  28. Change in the DSQ Pain Score (Question 4) From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study

    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.

    Time frame: Baseline of induction study (SHP621-301 [NCT02605837]), Week 36

  29. Change in the DSQ Pain Score (Question 4) From Current Study (SHP621-302 [NCT02736409]) Baseline at 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.

    Time frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 36

  30. Number of Participants With Treatment Emergent Adverse Events (TEAE's)

    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.

    Time frame: From start of the study drug administration up to follow up (Week 40)

  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

    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.

    Time frame: Baseline of induction study (SHP621-301 [NCT02605837]), Week 36

  32. Change in DXA Imaging Results (Location: Lumbar Spine [L1-L4]) From Current Study (SHP621-302 [NCT02736409]) Baseline at 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.

    Time frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 36

  33. Change in DXA Imaging Results (Location: Whole Body) From Baseline of Induction Study (SHP621-301 [NCT02605837]) at Week 36 of Current Study

    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.

    Time frame: Baseline of induction study (SHP621-301 [NCT02605837]), Week 36

  34. Change in DXA Imaging Results (Location: Whole Body) From Current Study (SHP621-302 [NCT02736409]) Baseline at 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.

    Time frame: Baseline of Current Study (SHP621-302 [NCT02736409]), Week 36

Other outcomes

  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

    Eosinophil histology relapse was defined as an eosinophil count of \>or=15 eos/HPF from at least 2 of 3 levels of the esophagus.

    Time frame: Week 12 and Week 36

07

Results

Posted Jan 13, 2021

Participant flow

This study was conducted at 70 sites in United States from 01 April 2016 (first participant enrolled) to 12 November 2019 (last participant completed).

Participant flow — Overall Study
MilestoneFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Started232510665
Completed19228053
Not completed432612
Withdrew: Withdrawal by subject42173
Withdrew: Non-compliance with study drug0001
Withdrew: Adverse event0154
Withdrew: Other0011
Withdrew: Pregnancy0010
Withdrew: Lost to follow-up0021
Withdrew: Physician decision0002

Outcome measures

PrimaryProportion of Participants Who Had Relapse During the Entire Week 36 Period

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.

Time frame:
Week 36
Reported as:
Number · proportion of participants
Proportion of Participants Who Had Relapse During the Entire Week 36 Period
proportion of participantsFull Responder BOS-PBO GroupFull Responder BOS-BOS Group
Proportion of Participants Who Had Relapse During the Entire Week 36 Period0.430.24
Statistical analysis
  • Full Responder BOS-PBO Group vs Full Responder BOS-BOS Group · Fisher Exact · p = 0.131 · Difference in proportion: -0.19 · 95% CI -0.452 to 0.082
SecondaryProportion of Participants With Long-term Treatment Response From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study

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.

Time frame:
Week 36
Reported as:
Number · proportion of participants
Proportion of Participants With Long-term Treatment Response From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study
proportion of participantsNon-Responder BOS-BOS Group
Proportion of Participants With Long-term Treatment Response From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study0.13
SecondaryProportion of Participants With Long-term Treatment Response From Current Study (SHP621-302 [NCT02736409]) Baseline at 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.

Time frame:
Week 36
Reported as:
Number · proportion of participants
Proportion of Participants With Long-term Treatment Response From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36
proportion of participantsNon-Responder BOS-BOS Group
Proportion of Participants With Long-term Treatment Response From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 360.06
SecondaryProportion 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

Histology response was defined as a peak eosinophil count of \<or=6/HPF across all available levels at Week 12 and Week 36.

Time frame:
Week 12 and Week 36
Reported as:
Number · proportion of participants
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
proportion of participantsFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Week 120.040.760.270.57
Week 360.300.520.210.38
SecondaryProportion 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

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.

Time frame:
Baseline of SHP621-301 (NCT02605837), Week 12 and Week 36
Reported as:
Number · proportion of participants
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
proportion of participantsFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Week 120.740.840.470.57
Week 360.780.840.460.58
SecondaryProportion 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

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.

Time frame:
Baseline of Current Study (SHP621-302 [NCT02736409]), Week 12 and Week 36
Reported as:
Number · proportion of participants
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
proportion of participantsFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Week 120.130.440.240.43
Week 360.390.400.250.42
SecondaryChange From Induction Study (SHP621-301 [NCT02605837]) Baseline in Total Endoscopy Score at Week 12 and Week 36 of Current Study

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.

Time frame:
Baseline of induction study (SHP621-301 [NCT02605837]), Week 12 and Week 36
Reported as:
Mean · Score on scale
Change From Induction Study (SHP621-301 [NCT02605837]) Baseline in Total Endoscopy Score at Week 12 and Week 36 of Current Study
Score on scaleFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Week 12-1.9 ± 0.51-5.3 ± 0.69-3.8 ± 0.43-4.7 ± 0.46
Week 36-2.7 ± 0.60-4.2 ± 0.84-4.3 ± 0.47-4.4 ± 0.46
SecondaryChange From Current Study (SHP621-302 [NCT02736409]) Baseline in Total Endoscopy Score at 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.

Time frame:
Baseline of Current Study (SHP621-302 [NCT02736409]), Week 12 and Week 36
Reported as:
Mean · Score on scale
Change From Current Study (SHP621-302 [NCT02736409]) Baseline in Total Endoscopy Score at Week 12 and Week 36
Score on scaleFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Week 120.7 ± 0.67-1.0 ± 0.69-0.3 ± 0.28-2.6 ± 0.46
Week 360.2 ± 0.700.0 ± 0.70-0.6 ± 0.34-2.3 ± 0.57
SecondaryChange From Induction Study (SHP621-301 [NCT02605837]) Baseline in Peak Eosinophil Count at Week 12 and Week 36 of Current Study

Change from induction study (SHP621-301 \[NCT02605837\]) baseline in peak eosinophil count at Week 12 and Week 36 of current study was reported.

Time frame:
Baseline of induction study (SHP621-301 [NCT02605837]), Week 12 and Week 36
Reported as:
Mean · eosinophil count
Change From Induction Study (SHP621-301 [NCT02605837]) Baseline in Peak Eosinophil Count at Week 12 and Week 36 of Current Study
eosinophil countFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Week 12-20.8 ± 13.41-60.1 ± 9.26-33.2 ± 5.14-70.3 ± 7.74
Week 36-59.8 ± 13.24-40.3 ± 13.23-33.2 ± 5.50-57.2 ± 7.68
SecondaryChange From Current Study (SHP621-302 [NCT02736409]) Baseline in Peak Eosinophil Count at 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.

Time frame:
Baseline of Current Study (SHP621-302 [NCT02736409]), Week 12 and Week 36
Reported as:
Mean · eosinophil count
Change From Current Study (SHP621-302 [NCT02736409]) Baseline in Peak Eosinophil Count at Week 12 and Week 36
eosinophil countFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Week 1266.0 ± 11.527.5 ± 3.567.6 ± 3.95-54.4 ± 5.65
Week 3625.5 ± 7.8326.9 ± 11.186.8 ± 4.63-39.1 ± 6.94
SecondaryProportion of Participants Who Had Peak Eosinophil Count Less Than (<) 15/High-Powered Field (HPF) at 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.

Time frame:
Week 12 and Week 36
Reported as:
Number · proportion of participants
Proportion of Participants Who Had Peak Eosinophil Count Less Than (<) 15/High-Powered Field (HPF) at Week 12 and Week 36
proportion of participantsFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Week 120.130.760.370.63
Week 360.520.600.290.42
SecondaryProportion of Participants Who Had Peak Eosinophil Count Less Than or Equal to (< or =) 1/High-Powered Field (HPF) at 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.

Time frame:
Week 12 and Week 36
Reported as:
Number · proportion of participants
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
proportion of participantsFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Week 120.000.600.150.42
Week 360.130.440.140.26
SecondaryChange 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

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.

Time frame:
Baseline of induction study (SHP621-301 [NCT02605837]), Week 12 and Week 36
Reported as:
Mean · eosinophil count
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
eosinophil countFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Proximal: Week 12-9.2 ± 13.96-23.3 ± 4.94-24.0 ± 4.38-41.6 ± 6.65
Proximal: Week 36-21.0 ± 7.09-19.6 ± 6.43-24.2 ± 4.44-42.3 ± 7.03
Mid: Week 12-14.8 ± 10.80-53.4 ± 9.85-32.7 ± 4.80-53.8 ± 6.16
Mid: Week 36-36.8 ± 9.89-47.7 ± 9.21-29.1 ± 5.05-40.8 ± 7.01
Distal: Week 12-17.1 ± 12.11-44.0 ± 6.74-23.0 ± 5.17-53.6 ± 6.64
Distal: Week 36-51.2 ± 13.69-25.3 ± 12.45-26.4 ± 5.38-40.2 ± 7.71
SecondaryChange 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

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.

Time frame:
Baseline of Current Study (SHP621-302 [NCT02736409]), Week 12 and Week 36
Reported as:
Mean · eosinophil count
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
eosinophil countFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Proximal: Week 1226.5 ± 10.442.8 ± 2.146.5 ± 2.92-30.8 ± 4.73
Proximal: Week 3610.0 ± 3.956.9 ± 4.686.8 ± 3.25-23.1 ± 4.91
Mid: Week 1238.4 ± 10.505.4 ± 2.883.2 ± 4.20-41.4 ± 5.55
Mid: Week 3617.9 ± 7.7212.5 ± 6.817.3 ± 4.50-28.9 ± 7.00
Distal: Week 1255.8 ± 10.047.3 ± 3.517.8 ± 3.66-41.6 ± 5.09
Distal: Week 3621.3 ± 6.4724.2 ± 10.504.4 ± 4.03-29.5 ± 6.20
SecondaryChange 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

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.

Time frame:
Baseline of induction study (SHP621-301 [NCT02605837]), Week 12 and Week 36
Reported as:
Mean · score on scale
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
score on scaleFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Combined Grade TSR: Week 12-0.1 ± 0.04-0.2 ± 0.03-0.2 ± 0.02-0.3 ± 0.02
Combined Grade TSR: Week 36-0.2 ± 0.03-0.2 ± 0.04-0.1 ± 0.02-0.2 ± 0.03
Combined Stage TSR: Week 12-0.1 ± 0.04-0.2 ± 0.03-0.2 ± 0.02-0.2 ± 0.02
Combined Stage TSR: Week 36-0.2 ± 0.03-0.2 ± 0.03-0.1 ± 0.02-0.2 ± 0.02
SecondaryChange From Current Study (SHP621-302 [NCT02736409]) Baseline in the Histopathologic Epithelial Features Combined Total Score (Grade and Stage) at 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.

Time frame:
Baseline of Current Study (SHP621-302 [NCT02736409]), Week 12 and Week 36
Reported as:
Mean · score on scale
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
score on scaleFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Combined Grade TSR: Week 120.2 ± 0.050.0 ± 0.020.0 ± 0.01-0.2 ± 0.02
Combined Grade TSR: Week 360.1 ± 0.030.1 ± 0.030.0 ± 0.02-0.2 ± 0.03
Combined Stage TSR: Week 120.2 ± 0.050.0 ± 0.020.0 ± 0.01-0.2 ± 0.02
Combined Stage TSR: Week 360.1 ± 0.030.1 ± 0.030.0 ± 0.02-0.2 ± 0.03
SecondaryProportion 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

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.

Time frame:
Week 12 and Week 36
Reported as:
Number · proportion of participants
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
proportion of participantsFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Week 120.520.800.410.52
Week 360.740.760.410.57
SecondaryProportion 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

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.

Time frame:
Week 12 and Week 36
Reported as:
Number · proportion of participants
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
proportion of participantsFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Week 120.130.360.190.37
Week 360.300.360.190.38
SecondaryChange in the DSQ Combined Score (Questions 2+3) From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study

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.

Time frame:
Baseline of induction study (SHP621-301 [NCT02605837]), Week 36
Reported as:
Mean · score on scale
Change in the DSQ Combined Score (Questions 2+3) From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study
score on scaleFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Change in the DSQ Combined Score (Questions 2+3) From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study-19.40 ± 3.627-23.28 ± 2.477-15.35 ± 1.673-17.33 ± 2.269
SecondaryChange in the DSQ Combined Score (Questions 2+3) From Current Study (SHP621-302 [NCT02736409]) Baseline at 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.

Time frame:
Baseline of Current Study (SHP621-302 [NCT02736409]), Week 36
Reported as:
Mean · score on scale
Change in the DSQ Combined Score (Questions 2+3) From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36
score on scaleFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Change in the DSQ Combined Score (Questions 2+3) From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36-4.00 ± 1.928-3.84 ± 1.682-4.39 ± 1.356-10.24 ± 2.183
SecondaryPercent Change in the DSQ Combined Score (Questions 2+3) From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study

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.

Time frame:
Baseline of induction study (SHP621-301 [NCT02605837]), Week 36
Reported as:
Mean · percent change
Percent Change in the DSQ Combined Score (Questions 2+3) From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study
percent changeFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Percent Change in the DSQ Combined Score (Questions 2+3) From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study-79.56 ± 7.370-79.77 ± 6.195-55.82 ± 5.048-61.44 ± 8.653
SecondaryPercent Change in the DSQ Combined Score (Questions 2+3) From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36

Percent change in DSQ combined score from baseline to final treatment period (Week 36) of current study was reported.

Time frame:
Baseline of Current Study (SHP621-302 [NCT02736409]), Week 36
Reported as:
Mean · percent change
Percent Change in the DSQ Combined Score (Questions 2+3) From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36
percent changeFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Percent Change in the DSQ Combined Score (Questions 2+3) From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36-47.00 ± 15.371-40.78 ± 19.216-10.18 ± 16.050-39.04 ± 21.413
SecondaryProportion of Participants Who Had Overall Binary Response I From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 12 and Week 36 of Current Study

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.

Time frame:
Week 12 and Week 36
Reported as:
Number · proportion of participants
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
proportion of participantsFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Week 120.040.640.150.42
Week 360.260.480.130.28
SecondaryProportion of Participants Who Had Overall Binary Response I From Current Study (SHP621-302 [NCT02736409]) Baseline at 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.

Time frame:
Week 12 and Week 36
Reported as:
Number · proportion of participants
Proportion of Participants Who Had Overall Binary Response I From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 12 and Week 36
proportion of participantsFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Week 120.000.320.050.29
Week 360.090.200.060.17
SecondaryProportion of Participants Who Had Overall Binary Response II From Induction Study SHP621-301 (NCT02605837) Baseline at Week 12 and Week 36 of Current Study

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.

Time frame:
Week 12 and Week 36
Reported as:
Number · proportion of participants
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
proportion of participantsFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Week 120.040.640.120.38
Week 360.260.440.120.28
SecondaryProportion of Participants Who Had Overall Binary Response II From Current Study (SHP621-302 [NCT02736409]) Baseline at 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.

Time frame:
Week 12 and Week 36
Reported as:
Number · proportion of participants
Proportion of Participants Who Had Overall Binary Response II From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 12 and Week 36
proportion of participantsFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Week 120.000.280.040.26
Week 360.040.200.060.17
SecondaryChange in the DSQ + Pain Score (Questions 2+3+4) From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study

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.

Time frame:
Baseline of induction study (SHP621-301 [NCT02605837]), Week 36
Reported as:
Mean · score on scale
Change in the DSQ + Pain Score (Questions 2+3+4) From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study
score on scaleFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Change in the DSQ + Pain Score (Questions 2+3+4) From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study-25.86 ± 5.023-31.10 ± 4.015-21.25 ± 2.299-23.17 ± 3.221
SecondaryChange in the DSQ+ Pain Score (Questions 2+3+4) From Current Study (SHP621-302 [NCT02736409]) Baseline at 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.

Time frame:
Baseline of Current Study (SHP621-302 [NCT02736409]), Week 36
Reported as:
Mean · score on scale
Change in the DSQ+ Pain Score (Questions 2+3+4) From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36
score on scaleFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Change in the DSQ+ Pain Score (Questions 2+3+4) From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36-5.67 ± 2.704-4.23 ± 2.003-5.59 ± 1.825-14.12 ± 2.944
SecondaryChange in the DSQ Pain Score (Question 4) From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study

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.

Time frame:
Baseline of induction study (SHP621-301 [NCT02605837]), Week 36
Reported as:
Mean · score on scale
Change in the DSQ Pain Score (Question 4) From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study
score on scaleFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Change in the DSQ Pain Score (Question 4) From Induction Study (SHP621-301 [NCT02605837]) Baseline at Week 36 of Current Study-6.46 ± 1.667-7.92 ± 2.130-5.90 ± 0.907-5.83 ± 1.356
SecondaryChange in the DSQ Pain Score (Question 4) From Current Study (SHP621-302 [NCT02736409]) Baseline at 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.

Time frame:
Baseline of Current Study (SHP621-302 [NCT02736409]), Week 36
Reported as:
Mean · score on scale
Change in the DSQ Pain Score (Question 4) From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36
score on scaleFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Change in the DSQ Pain Score (Question 4) From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36-1.67 ± 1.039-0.39 ± 0.495-1.20 ± 0.634-3.88 ± 1.101
SecondaryNumber of Participants With Treatment Emergent Adverse Events (TEAE's)

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.

Time frame:
From start of the study drug administration up to follow up (Week 40)
Reported as:
Count of participants · Participants
Number of Participants With Treatment Emergent Adverse Events (TEAE's)
ParticipantsFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Number of Participants With Treatment Emergent Adverse Events (TEAE's)14217249
SecondaryChange 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

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.

Time frame:
Baseline of induction study (SHP621-301 [NCT02605837]), Week 36
Reported as:
Mean · Z score
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
Z scoreFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
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—-0.185 ± 0.24750.020 ± 0.36370.100 ± 0.4247
SecondaryChange in DXA Imaging Results (Location: Lumbar Spine [L1-L4]) From Current Study (SHP621-302 [NCT02736409]) Baseline at 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.

Time frame:
Baseline of Current Study (SHP621-302 [NCT02736409]), Week 36
Reported as:
Mean · Z score
Change in DXA Imaging Results (Location: Lumbar Spine [L1-L4]) From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36
Z scoreFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Change in DXA Imaging Results (Location: Lumbar Spine [L1-L4]) From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36—-0.240 ± 0.74950.062 ± 0.48390.071 ± 0.4783
SecondaryChange in DXA Imaging Results (Location: Whole Body) From Baseline of Induction Study (SHP621-301 [NCT02605837]) at Week 36 of Current Study

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.

Time frame:
Baseline of induction study (SHP621-301 [NCT02605837]), Week 36
Reported as:
Mean · Z score
Change in DXA Imaging Results (Location: Whole Body) From Baseline of Induction Study (SHP621-301 [NCT02605837]) at Week 36 of Current Study
Z scoreFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Change in DXA Imaging Results (Location: Whole Body) From Baseline of Induction Study (SHP621-301 [NCT02605837]) at Week 36 of Current Study—0.190 ± 0.0424-0.023 ± 0.45100.244 ± 0.5543
SecondaryChange in DXA Imaging Results (Location: Whole Body) From Current Study (SHP621-302 [NCT02736409]) Baseline at 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.

Time frame:
Baseline of Current Study (SHP621-302 [NCT02736409]), Week 36
Reported as:
Mean · Z score
Change in DXA Imaging Results (Location: Whole Body) From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36
Z scoreFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Change in DXA Imaging Results (Location: Whole Body) From Current Study (SHP621-302 [NCT02736409]) Baseline at Week 36—0.065 ± 0.0636-0.070 ± 0.40600.140 ± 0.4173
Other pre-specifiedProportion 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

Eosinophil histology relapse was defined as an eosinophil count of \>or=15 eos/HPF from at least 2 of 3 levels of the esophagus.

Time frame:
Week 12 and Week 36
Reported as:
Number · proportion of participants
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
proportion of participantsFull Responder BOS-PBO GroupFull Responder BOS-BOS Group
Histologic Relapse before or at Week 120.650.12
Histologic Relapse before or at Week 360.780.36

Adverse events

Collected over From start of the study drug administration up to follow up (Week 40). Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Full Responder BOS-PBO Group0/23 (0%)1/23 (4.3%)9/23 (39.1%)
Full Responder BOS-BOS Group0/25 (0%)0/25 (0%)10/25 (40%)
Non-Responder BOS-BOS Group0/106 (0%)6/106 (5.7%)26/106 (24.5%)
PBO-BOS Group0/65 (0%)5/65 (7.7%)18/65 (27.7%)
Most frequent serious events
Showing 10 of 11
Most frequent serious events
EventFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
Back painMusculoskeletal and connective tissue disorders1/230/250/1060/65
Anaphylactic reactionImmune system disorders0/230/251/1062/65
Anaphylactic shockImmune system disorders0/230/250/1061/65
Herpes simplexInfections and infestations0/230/250/1061/65
Head injuryInjury, poisoning and procedural complications0/230/250/1061/65
Neck injuryInjury, poisoning and procedural complications0/230/250/1061/65
Non-cardiac chest painGeneral disorders0/230/251/1060/65
AppendicitisInfections and infestations0/230/251/1060/65
Humerus fractureInjury, poisoning and procedural complications0/230/251/1060/65
Abortion spontaneousPregnancy, puerperium and perinatal conditions0/230/251/1060/65
Most frequent other events
Showing 10 of 15
Most frequent other events
EventFull Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS Group
GastritisGastrointestinal disorders0/234/252/1060/65
NasopharyngitisInfections and infestations3/231/256/1063/65
VomitingGastrointestinal disorders0/230/2513/1061/65
DiarrhoeaGastrointestinal disorders1/233/252/1064/65
Upper respiratory tract infectionInfections and infestations1/233/259/1062/65
DysphagiaGastrointestinal disorders2/231/252/1061/65
NauseaGastrointestinal disorders2/230/259/1064/65
FatigueGeneral disorders2/232/252/1061/65
Seasonal allergyImmune system disorders2/231/251/1060/65
SinusitisInfections and infestations2/231/254/1063/65

Baseline characteristics

Full Analysis Set (FAS) consisted of all randomized participants who received at least 1 dose of investigational product in the NCT02736409 (SHP621-302) study.

Age, Continuous
Age, Continuous(Years)Full Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS GroupTotal
Mean36.1 ± 11.6636.8 ± 14.0933.1 ± 12.0233.5 ± 12.6134.0 ± 12.40
Sex: Female, Male
Sex: Female, Male(Participants)Full Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS GroupTotal
Female711422686
Male16146439133
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Full Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS GroupTotal
Hispanic or Latino00415
Not Hispanic or Latino232510162211
Unknown or Not Reported00123
Race (NIH/OMB)
Race (NIH/OMB)(Participants)Full Responder BOS-PBO GroupFull Responder BOS-BOS GroupNon-Responder BOS-BOS GroupPBO-BOS GroupTotal
American Indian or Alaska Native00101
Asian00000
Native Hawaiian or Other Pacific Islander00011
Black or African American01405
White22249562203
More than one race00000
Unknown or Not Reported10629
08

Study locations

62 sites
  • Children's Hospital
    Birmingham, Alabama 35233, United States
  • Phoenix Childrens Hospital
    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
  • 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
  • Arnold Palmer Hospital For Children
    Orlando, Florida 32806, 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
  • Grand Teton Research Group, PLLC
    Idaho Falls, Idaho, United States
  • Northwestern University
    Chicago, Illinois 60611, United States
  • University of Illinois College of Medicine at Peoria Pediatric Subspecialty Clinic
    Peoria, Illinois 61603, United States
  • Indiana University
    Indianapolis, Indiana 46202, United States
  • Aquiant Research
    New Albany, Indiana 47150, 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 02111, 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
  • Minnesota Gastroenterology PA
    Plymouth, Minnesota 55446, United States
  • Mayo Clinic
    Rochester, Minnesota 55905, United States
  • Bozeman Health Deaconess Hospital
    Bozeman, Montana 59718, United States
  • Long Island Gastrointestinal Research Group LLP
    Great Neck, New York 11023, United States
  • Mount Sinai Medical Center
    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
  • Children's Hospital of Philadelphia
    Philadelphia, Pennsylvania 19104, United States
  • University of Pennsylvania
    Philadelphia, Pennsylvania 19104, 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
  • 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
  • Primary Children's Hospital
    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
09

References and documents

Publications

  • 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. PubMed 39735351 ↗
  • 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. PubMed 34182150 ↗

Study documents

  • Study protocol · Dec 19, 2016
  • Statistical analysis plan · Dec 4, 2019

Documents are hosted by the registry — open the source record to download them.

Individual participant data

Plan to share: No — 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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Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Feb 19, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT02736409
Lead sponsor
Shire
Responsible party
Sponsor
First posted
Apr 13, 2016
Start date
Apr 1, 2016
Primary completion
Nov 12, 2019
Completion
Nov 12, 2019
Results posted
Jan 13, 2021
Last update
Feb 19, 2025

Study contacts

Study Director
study director · Shire

Oversight

Data monitoring committee
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Dec 2020. You cannot join it, but the record below documents what was studied.

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Discussion

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Studies of the drug tested here: Budesonide

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