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OBS in Adolescent and Adults With EOE: A Phase II, Randomized, Double-Blind, Placebo Controlled, Study With an Open Label Extension

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ClinicalTrials.gov Identifier: NCT01642212
Recruitment Status : Completed
First Posted : July 17, 2012
Results First Posted : December 7, 2015
Last Update Posted : December 7, 2015
Sponsor:
Information provided by (Responsible Party):
Shire

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition Eosinophilic Esophagitis
Interventions Drug: Oral Budesonide Suspension (MB-9)
Drug: Placebo
Enrollment 93
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Single-blind Placebo Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg Placebo to Open-label Oral Budesonide Suspension (OBS) 2 mg Oral Budesonide Suspension (OBS) 2 mg to Open-label OBS 2 mg
Hide Arm/Group Description Eligible participants entered a 4-week, single-blind, placebo baseline period. Participants ingested the first dose of placebo in the clinic in the presence of study center personnel, and the remaining doses were ingested at home. Participants took placebo twice daily (bid); once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid. Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid. Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid. During the first 12 weeks of open-label extension treatment, participants took 2 mg OBS (formulation MB-9) once daily (qd) at bedtime. The volume per dose was 10 mL of oral liquid (10 mL qd; 0.2 mg/mL). Thereafter, an optional dose increase to 1.5 mg twice daily (bid) (7.5 mL bid; 0.2 mg/mL) and then to 2 mg bid (10 mL bid; 0.2 mg/mL) was allowed for subjects whose response to the 2 mg once daily dose was inadequate, as determined by the Investigator; a dose decrease to 2 mg once daily was allowed at any time. During the first 12 weeks of open-label extension treatment, participants took 2 mg OBS (formulation MB-9) once daily (qd) at bedtime. The volume per dose was 10 mL of oral liquid (10 mL qd; 0.2 mg/mL). Thereafter, an optional dose increase to 1.5 mg twice daily (bid) (7.5 mL bid; 0.2 mg/mL) and then to 2 mg bid (10 mL bid; 0.2 mg/mL) was allowed for subjects whose response to the 2 mg once daily dose was inadequate, as determined by the Investigator; a dose decrease to 2 mg once daily was allowed at any time.
Period Title: Single-blind Baseline
Started 119 0 0 0 0
Completed 93 0 0 0 0
Not Completed 26 0 0 0 0
Reason Not Completed
Did Not Meet Eligibility Criteria             26             0             0             0             0
Period Title: Double-blind Treatment
Started 0 42 51 0 0
Completed 0 39 49 0 0
Not Completed 0 3 2 0 0
Reason Not Completed
Not Specified             0             2             1             0             0
Non-compliance             0             1             0             0             0
Adverse Event             0             0             1             0             0
Period Title: Open-label Extension
Started 0 0 0 37 [1] 45 [2]
Completed 0 0 0 27 37
Not Completed 0 0 0 10 8
Reason Not Completed
Adverse Event             0             0             0             3             0
Sponsor Decision             0             0             0             7             4
Consent Withdrawn             0             0             0             0             2
Lost to Follow-up             0             0             0             0             1
Not Specified             0             0             0             0             1
[1]
Two participants completed double-blind treatment but did not enter the open-label extension.
[2]
Four participants completed double-blind treatment but did not enter the open-label extension.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg Total
Hide Arm/Group Description Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid. Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid. Total of all reporting groups
Overall Number of Baseline Participants 42 51 93
Hide Baseline Analysis Population Description
The Safety Analysis Set, defined as all randomized participants who received at least 1 dose of double-blind study drug (placebo or OBS); subjects were analyzed based on the actual treatment received.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 42 participants 51 participants 93 participants
20.8  (7.50) 22.3  (7.92) 21.6  (7.73)
Age, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 42 participants 51 participants 93 participants
<18 Years Of Age 17 18 35
>/= 18 Years Of Age 25 33 58
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 42 participants 51 participants 93 participants
Female
13
  31.0%
16
  31.4%
29
  31.2%
Male
29
  69.0%
35
  68.6%
64
  68.8%
1.Primary Outcome
Title Percent of Participants Who Were Histologic Responders
Hide Description Histologic response was defined as a peak eosinophil count </= 6/high power field (light microscopy) (HPF) across all esophageal levels at the final treatment evaluation (Week 16). An independent, central pathologist determined the peak eosinophil count from the proximal, mid-, and distal levels and selected the maximum peak value. Histopathology data were collected in a blinded fashion.
Time Frame Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
The modified Intent-to-Treat (MITT) Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Measure Type: Number
Unit of Measure: percent of participants
2.6 38.8
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
2.Primary Outcome
Title Change From Baseline in The Dysphagia Symptom Questionnaire (DSQ) Score at The Final Treatment Period Evaluation
Hide Description Participants' dysphagia symptoms were evaluated using the 4-item DSQ. The questionnaire was developed by the Sponsor, as an ePRO measure, according to the principles of the Final Guidance for Industry for Patient Reported Outcome Measures (PRO Guidance December 2009). 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).The DSQ score was calculated based on responses to Questions 2 and 3 [14 x (sum of points from Questions 2 and 3 in the daily DSQ)/number of diaries with non-missing data]. Baseline was the DSQ score of the 14-day period before randomization. A negative change from baseline indicates that symptoms decreased.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
The MITT Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Least Squares Mean (Standard Error)
Unit of Measure: scores on a scale
-7.53  (1.910) -14.27  (1.682)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0096
Comments LS mean estimates were determined using an ANCOVA model including treatment group as a factor and the baseline score as a covariate.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS mean difference
Estimated Value -6.75
Confidence Interval (2-Sided) 95%
-11.808 to -1.687
Estimation Comments [Not Specified]
3.Secondary Outcome
Title Change From Baseline in The DSQ Score Over Time
Hide Description Participants' dysphagia symptoms were evaluated using the 4-item DSQ. The questionnaire was developed by the Sponsor, as an ePRO measure, according to the principles of the Final Guidance for Industry for Patient Reported Outcome Measures (PRO Guidance December 2009). 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).The DSQ score was calculated based on responses to Questions 2 and 3 [14 x (sum of points from Questions 2 and 3 in the daily DSQ)/number of diaries with non-missing data]. Baseline was the DSQ score of the 14-day period before randomization. A negative change from baseline indicates that symptoms decreased.
Time Frame Baseline, Weeks 8 and 12
Hide Outcome Measure Data
Hide Analysis Population Description
The MITT Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Least Squares Mean (Standard Error)
Unit of Measure: scores on a scale
Week 8, n = 37, 49 -4.92  (1.673) -7.41  (1.453)
Week 12, n = 36, 49 -7.42  (2.065) -10.33  (1.770)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of Week 8
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2649
Comments LS mean estimates were determined using the ANCOVA model including treatment group as a factor and baseline DSQ score as a covariate.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS mean difference
Estimated Value -2.49
Confidence Interval (2-Sided) 95%
-6.895 to 1.920
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of Week 12
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2878
Comments LS mean estimates were determined using the ANCOVA model including treatment group as a factor and baseline DSQ score as a covariate.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS mean difference
Estimated Value -2.91
Confidence Interval (2-Sided) 95%
-8.322 to 2.501
Estimation Comments [Not Specified]
4.Secondary Outcome
Title Change From Baseline in The DSQ Score For The 50th Percentile of Participants at The Final Treatment Period Evaluation
Hide Description A cumulative distribution function curve was constructed to illustrate the cumulative proportion of participants (x-axis) vs. the change in the DSQ score from baseline to the final treatment evaluation (y-axis). The 50th percentile is participants with a DSQ score that is in the middle of the distribution of all scores. A negative change from baseline indicates that symptoms decreased.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
The MITT Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Measure Type: Number
Unit of Measure: scores on a scale
-6.35 -11.20
5.Secondary Outcome
Title Percent of Participants With a Peak Eosinophil Count </= 15/High Power Field (Light Microscopy) (HPF) And </= 1/HPF at The Final Treatment Period Evaluation
Hide Description An independent, central pathologist determined the peak eosinophil count from the proximal, mid-, and distal levels and selected the maximum peak value across all available esophagus levels. Histopathology data were collected in a blinded fashion. The values reported are for participants with histologic response.
Time Frame Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
The MITT Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Measure Type: Number
Unit of Measure: percent of participants
</= 15 Eosinophils/HPF 7.9 46.9
</= 1 Eosinophils/HPF 0 30.6
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of </= 15 Eosinophils/HPF
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0001
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of </= 1 Eosinophils/HPF
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
6.Secondary Outcome
Title Percent of Participants With a >/= 30% And >/= 50% Reduction In The DSQ Score From Baseline to The Final Treatment Period Evaluation
Hide Description Participants' dysphagia symptoms were evaluated using the 4-item DSQ. The questionnaire was developed by the Sponsor, as an ePRO measure, according to the principles of the Final Guidance for Industry for Patient Reported Outcome Measures (PRO Guidance December 2009). 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).The DSQ score was calculated based on responses to Questions 2 and 3 [14 x (sum of points from Questions 2 and 3 in the daily DSQ)/number of diaries with non-missing data]. Baseline was the DSQ score of the 14-day period before randomization.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
The MITT Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Measure Type: Number
Unit of Measure: percent of participants
>/= 30% DSQ Score Reduction 44.7 69.4
>/= 50% DSQ Score Reduction 39.5 63.3
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of >/= 30% DSQ score reduction
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0206
Comments [Not Specified]
Method Pearson's chi-square
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of >/= 50% DSQ score reduction
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0275
Comments [Not Specified]
Method Pearson's chi-square
Comments [Not Specified]
7.Secondary Outcome
Title Percent of Participants Who Were Overall Responders at The Final Treatment Period Evaluation
Hide Description Overall response was defined as a reduction in the 2-week DSQ score of >/= 30% and >/= 50% from baseline to the final treatment period evaluation and a peak eosinophil count of </= 6/high power field (light microscopy) (HPF) across all available esophageal levels at the final treatment period evaluation. An independent, central pathologist determined the peak eosinophil count from the proximal, mid-, and distal levels and selected the maximum peak value. Histopathology data were collected in a blinded fashion.
Time Frame Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
The MITT Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Measure Type: Number
Unit of Measure: percent of participants
Responder and >/= 30% DSQ Score Reduction 2.6 26.5
Responder and >/= 50% DSQ Score Reduction 2.6 20.4
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of response and >/= 30% DSQ score reduction
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0026
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of response and >/= 50% DSQ score reduction
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0199
Comments [Not Specified]
Method Fisher Exact
Comments [Not Specified]
8.Secondary Outcome
Title Change From Baseline in The Histopathologic Epithelial Features Combined Total Score at The Final Treatment Period Evaluation
Hide Description Each esophageal biopsy specimen was evaluated microscopically by an independent, central pathologist for signs of epithelial inflammation and lamina propria fibrosis. Histopathologic epithelial features of each available esophageal level biopsy consisting of basal layer hyperplasia, eosinophil peak, dilated intercellular spaces, eosinophil microabcesses, surface layering, surface alteration, and apoptotic epithelial cells were scored and summed. Histopathology data were collected in a blinded fashion. Histopathology epithelial features were scored for both grade and stage. Each feature had a possible score of 0-3 for grade as well as stage. Thus each of the 3 levels had a possible score of 21, and a possible total grade or stage score of 63 for a maximum combined score of 126. The grade and stage score of the lamina propria was not included because the biopsy material was not available. A negative change from baseline indicates that epithelial inflammation decreased.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
The MITT Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Least Squares Mean (Standard Error)
Unit of Measure: scores on a scale
-1.41  (2.970) -23.75  (2.602)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments [Not Specified]
Method ANCOVA
Comments LS mean based on the ANCOVA model including treatment group as a factor and baseline as a covariate.
Method of Estimation Estimation Parameter LS mean difference
Estimated Value -22.34
Confidence Interval (2-Sided) 95%
-30.345 to -14.334
Estimation Comments [Not Specified]
9.Secondary Outcome
Title Change From Baseline in The Total Endoscopy Score at The Final Treatment Period Evaluation
Hide Description The gross endoscopic appearance of the esophageal surface was evaluated by a blinded study center physician. Endoscopic findings with separate evaluations of the proximal and distal esophagus were recorded with respect to 5 major categories, including exudates or plaques, fixed esophageal rings, edema, furrows, and strictures. The endoscopy score was the sum of the scores for the 5 major categories – grade 0-1 for strictures; grade 0-2 for exudates or plaques, edema, and furrows; and grade 0-3 for fixed esophageal rings for the proximal and distal locations. The maximum endoscopy score was 10 points 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). Baseline was defined as the endoscopy score at screening. A negative change from baseline indicates that appearance improved.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
The MITT Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Least Squares Mean (Standard Error)
Unit of Measure: scores on a scale
0.19  (0.530) -3.58  (0.466)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments LS mean estimates based on the ANCOVA model including treatment group as a factor and baseline value as a covariate.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS mean difference
Estimated Value -3.76
Confidence Interval (2-Sided) 95%
-5.172 to -2.358
Estimation Comments [Not Specified]
10.Secondary Outcome
Title Change From Baseline in The Peak Eosinophil Count at Each Available Esophageal Level at The Final Treatment Period Evaluation
Hide Description An independent, central pathologist determined the peak eosinophil count from the proximal, mid-, and distal levels and selected the maximum peak value across all available esophagus levels. Histopathology data were collected in a blinded fashion. Baseline was defined as the score at screening. A negative change from baseline indicates that eosinophil count decreased.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
The MITT Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Least Squares Mean (Standard Error)
Unit of Measure: eosinophils/HPF
Proximal, n = 32, 45 -30.06  (6.798) -65.56  (5.704)
Mid-, n = 36, 46 -17.34  (9.244) -76.90  (8.173)
Distal, n = 38, 49 -1.90  (9.852) -69.28  (8.667)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of proximal eosinophil count
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0002
Comments LS mean estimates based on the ANCOVA model including treatment group as a factor and baseline value as a covariate.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS mean difference
Estimated Value -35.50
Confidence Interval (2-Sided) 95%
-53.438 to -17.570
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of mid- eosinophil count
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments LS mean estimates based on the ANCOVA model including treatment group as a factor and baseline value as a covariate.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS mean difference
Estimated Value -59.56
Confidence Interval (2-Sided) 95%
-84.173 to -34.948
Estimation Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of distal eosinophil count
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments LS mean estimates based on the ANCOVA model including treatment group as a factor and baseline value as a covariate.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS mean difference
Estimated Value -67.38
Confidence Interval (2-Sided) 95%
-93.573 to -41.180
Estimation Comments [Not Specified]
11.Secondary Outcome
Title Change From Baseline in The Physician’s Global Assessment (PGA) of Disease Activity at The Final Treatment Period Evaluation
Hide Description The physician Investigator (or qualified physician’s assistant or nurse practitioner) completed the PGA to provide the global assessment of eosinophilic esophagitis (EoE) disease activity using a 0 to 100 mm visual analog scale (VAS) scale. The VAS is a 100 mm horizontal line on which the right extreme (100) is labeled “worst possible disease activity” and the left extreme (0) is labeled “no disease activity”. The PGA raters were instructed to consider the line for the VAS a continuum with their own medical opinion or judgment of extremes on either end and to draw a vertical line at a point that best approximates the participant's current level of EoE disease activity. A negative change from baseline indicates that disease activity decreased.
Time Frame Baseline, Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
The MITT Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Least Squares Mean (Standard Error)
Unit of Measure: scores on a scale
-10.16  (4.199) -28.61  (3.695)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0015
Comments LS mean estimates were based on the ANCOVA model including treatment group as a factor and baseline value as a covariate.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS mean difference
Estimated Value -18.44
Confidence Interval (2-Sided) 95%
-29.593 to -7.293
Estimation Comments [Not Specified]
12.Secondary Outcome
Title Distribution of Responses For The Patient Global Impression of Change (PGIC) Survey at The Final Treatment Evaluation
Hide Description Participants evaluated the change in their dysphasia (food passing slowly/difficulty swallowing) since the start of the study (screening) by choosing 1 of 7 responses on the PGIC survey: much worse (-3), worse (-2), a little worse (-1), no change (0), a little better (1), better (2), or much better (3). The values reported are the percent of participants who chose that response.
Time Frame Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
The MITT Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Measure Type: Number
Unit of Measure: percent of participants
Much Worse 0 0
Worse 2.6 2.1
A Little Worse 13.2 2.1
No Change 15.8 12.5
A Little Better 28.9 25.0
Better 28.9 25.0
Much Better 10.5 33.3
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of distribution of scores across all responses
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1170
Comments P-value for comparison of the treatment difference was determined by Cochran-Mantel-Haenszel row mean score test.
Method Cochran-Mantel-Haenszel
Comments [Not Specified]
13.Secondary Outcome
Title Percent of Participants With Improved Symptoms on The Eosinophilic Esophagitis (EoE) Symptom Survey at The Final Treatment Period Evaluation
Hide Description The EoE survey assessed the following symptoms: heartburn, chest pain, regurgitation, abdominal pain, nausea, and vomiting. Participants indicated, by checking a box, if they had a change in symptoms (excluding dysphasia or food impaction) within the past 2 weeks. Baseline was defined as the assessment before randomization. Responses were as follows regarding symptoms at the final treatment evaluation: Improved - participant reported a specific symptom at baseline, but changed to no specific symptom (‘Yes’ to ‘No’); No change - participant reported or did not report a specific symptom at both baseline and the final treatment evaluation; Worsened -participant did not report a specific symptom at baseline, but changed to report that specific symptom (‘No’ to ‘Yes’).
Time Frame Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
The MITT Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Measure Type: Number
Unit of Measure: percent of participants
Heartburn 10.5 18.4
Chest Pain 23.7 18.4
Regurgitation 15.8 20.4
Abdominal Pain 15.8 10.2
Nausea 10.5 16.3
Vomiting 5.3 4.1
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of heartburn
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1947
Comments P-value for comparison of the treatment difference at baseline vs. final treatment evaluation was determined by a Cochran-Mantel-Haenszel row mean score test. All responses (improved, no change, worsened) were analyzed collectively for each symptom.
Method Cochran-Mantel-Haenszel
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of chest pain
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.8029
Comments P-value for comparison of the treatment difference at baseline vs. final treatment evaluation was determined by a Cochran-Mantel-Haenszel row mean score test. All responses (improved, no change, worsened) were analyzed collectively for each symptom.
Method Cochran-Mantel-Haenszel
Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of regurgitation
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4963
Comments P-value for comparison of the treatment difference at baseline vs. final treatment evaluation was determined by a Cochran-Mantel-Haenszel row mean score test. All responses (improved, no change, worsened) were analyzed collectively for each symptom.
Method Cochran-Mantel-Haenszel
Comments [Not Specified]
Show Statistical Analysis 4 Hide Statistical Analysis 4
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of abdominal pain
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5257
Comments P-value for comparison of the treatment difference at baseline vs. final treatment evaluation was determined by a Cochran-Mantel-Haenszel row mean score test. All responses (improved, no change, worsened) were analyzed collectively for each symptom.
Method Cochran-Mantel-Haenszel
Comments [Not Specified]
Show Statistical Analysis 5 Hide Statistical Analysis 5
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of nausea
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5219
Comments P-value for comparison of the treatment difference at baseline vs. final treatment evaluation was determined by a Cochran-Mantel-Haenszel row mean score test. All responses (improved, no change, worsened) were analyzed collectively for each symptom.
Method Cochran-Mantel-Haenszel
Comments [Not Specified]
Show Statistical Analysis 6 Hide Statistical Analysis 6
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of vomiting
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2886
Comments P-value for comparison of the treatment difference at baseline vs. final treatment evaluation was determined by a Cochran-Mantel-Haenszel row mean score test. All responses (improved, no change, worsened) were analyzed collectively for each symptom.
Method Cochran-Mantel-Haenszel
Comments [Not Specified]
14.Secondary Outcome
Title Percent of Participants With No Change in Symptoms on The EoE Symptom Survey at The Final Treatment Period Evaluation
Hide Description The EoE survey assessed the following symptoms: heartburn, chest pain, regurgitation, abdominal pain, nausea, and vomiting. Participants indicated, by checking a box, if they had a change in symptoms (excluding dysphasia or food impaction) within the past 2 weeks. Baseline was defined as the assessment before randomization. Responses were as follows regarding symptoms at the final treatment evaluation: Improved - participant reported a specific symptom at baseline, but changed to no specific symptom (‘Yes’ to ‘No’); No change - participant reported or did not report a specific symptom at both baseline and the final treatment evaluation; Worsened -participant did not report a specific symptom at baseline, but changed to report that specific symptom (‘No’ to ‘Yes’).
Time Frame Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
The MITT Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Measure Type: Number
Unit of Measure: percent of participants
Heartburn 86.8 71.4
Chest Pain 68.4 71.4
Regurgitation 71.1 73.5
Abdominal Pain 76.3 85.7
Nausea 86.8 77.6
Vomiting 81.6 91.8
15.Secondary Outcome
Title Percent of Participants With Worsened Symptoms on The EoE Symptom Survey at The Final Treatment Period Evaluation
Hide Description The EoE survey assessed the following symptoms: heartburn, chest pain, regurgitation, abdominal pain, nausea, and vomiting. Participants indicated, by checking a box, if they had a change in symptoms (excluding dysphasia or food impaction) within the past 2 weeks. Baseline was defined as the assessment before randomization. Responses were as follows regarding symptoms at the final treatment evaluation: Improved - participant reported a specific symptom at baseline, but changed to no specific symptom (‘Yes’ to ‘No’); No change - participant reported or did not report a specific symptom at both baseline and the final treatment evaluation; Worsened -participant did not report a specific symptom at baseline, but changed to report that specific symptom (‘No’ to ‘Yes’).
Time Frame Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
The MITT Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Measure Type: Number
Unit of Measure: percent of participants
Heartburn 2.6 10.2
Chest Pain 7.9 10.2
Regurgitation 13.2 6.1
Abdominal Pain 7.9 4.1
Nausea 2.6 6.1
Vomiting 13.2 4.1
16.Secondary Outcome
Title Percent of Participants Without Symptoms on The EoE Symptom Survey at The Final Treatment Period Evaluation
Hide Description This outcome assessed the symptoms of participants who were symptom-free at baseline. The EoE survey assessed the following symptoms: heartburn, chest pain, regurgitation, abdominal pain, nausea, and vomiting. Participants indicated, by checking a box, if they had a change in symptoms (excluding dysphasia or food impaction) within the past 2 weeks. Baseline was defined as the assessment before randomization. Responses were as follows regarding symptoms at the final treatment evaluation: No change - participant did not report a specific symptom at both baseline and the final treatment evaluation; Worsened -participant did not report any symptom at baseline, but changed to report at least 1 symptom at the final treatment evaluation.
Time Frame Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
The MITT Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Measure Type: Number
Unit of Measure: percent of participants
71.1 83.7
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of symptoms of participants with no symptoms at baseline
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1600
Comments P-value for comparison of the treatment difference at baseline vs. final treatment evaluation was determined by a Cochran-Mantel-Haenszel row mean score test. Both responses (no change, worsened) were analyzed collectively.
Method Cochran-Mantel-Haenszel
Comments [Not Specified]
17.Secondary Outcome
Title Percent of Participants With New Symptoms on The EoE Symptom Survey at The Final Treatment Period Evaluation
Hide Description This outcome assessed the symptoms of participants who were symptom-free at baseline. The EoE survey assessed the following symptoms: heartburn, chest pain, regurgitation, abdominal pain, nausea, and vomiting. Participants indicated, by checking a box, if they had a change in symptoms (excluding dysphasia or food impaction) within the past 2 weeks. Baseline was defined as the assessment before randomization. Responses were as follows regarding symptoms at the final treatment evaluation: No change - participant reported or did not report a specific symptom at both baseline and the final treatment evaluation; Worsened -participant did not report any symptom at baseline, but changed to report at least 1 symptom at the final treatment evaluation.
Time Frame Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
The MITT Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Measure Type: Number
Unit of Measure: percent of participants
28.9 16.3
18.Secondary Outcome
Title Percent of Participants Who Were Symptom Responders on The DSQ+Pain Scale at The Final Treatment Period Evaluation
Hide Description Participants' dysphagia symptoms were evaluated using the 4-item DSQ. The questionnaire was developed by the Sponsor, as an ePRO measure, according to the principles of the Final Guidance for Industry for Patient Reported Outcome Measures (PRO Guidance December 2009). All participants used a diary, and responded to Question 1 (did you eat solid food) and Question 2 (did food pass slowly or get stuck). If the answer to Question 2 was 'No', the diary ended for that day. If a participant answered 'Yes', he/she advanced to Question 3 (did you have to do anything to make the food go down or get relief) and Question 4 (extent to which the participant experienced pain while swallowing).The DSQ+pain response was defined as a >/= 30% and >/= 50% reduction from baseline in the combined score from Questions 2, 3, and 4. The 2-week DSQ+pain score was calculated by adding points from Questions 2, 3, and 4 and then taking the average of the available scores over each 2-week interval.
Time Frame Week 16
Hide Outcome Measure Data
Hide Analysis Population Description
The MITT Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Measure Type: Number
Unit of Measure: percent of participants
>/= 30% DSQ+pain Score Reduction 47.4 67.3
>/= 50% DSQ+pain Score Reduction 39.5 65.3
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of >/= 30% DSQ+pain score reduction
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0606
Comments [Not Specified]
Method Pearson's chi-square
Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of >/= 50% DSQ+pain score reduction
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0165
Comments [Not Specified]
Method Pearson's chi-square
Comments [Not Specified]
19.Secondary Outcome
Title Percent of Days That Participants Reported That They Avoided Solid Food During The Baseline And Treatment Periods
Hide Description Participants' dysphagia symptoms were evaluated using the 4-item DSQ. The questionnaire was developed by the Sponsor, as an ePRO measure, according to the principles of the Final Guidance for Industry for Patient Reported Outcome Measures (PRO Guidance December 2009). 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). Values were calculated for all the days that Question 1 was answered from 14 days prior to baseline visit up to the final treatment period evaluation.
Time Frame From 14 days prior to the baseline visit to the final treatment period evaluation
Hide Outcome Measure Data
Hide Analysis Population Description
The MITT Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Mean (Inter-Quartile Range)
Unit of Measure: percentage of days
1.2
(0.0 to 1.1)
0.7
(0.0 to 1.2)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.7817
Comments [Not Specified]
Method Wilcoxon (Mann-Whitney)
Comments [Not Specified]
20.Secondary Outcome
Title Change From Baseline in The Scores of DSQ Question 1 During The Treatment Period
Hide Description Participants' dysphagia symptoms were evaluated using the 4-item DSQ. The questionnaire was developed by the Sponsor, as an ePRO measure, according to the principles of the Final Guidance for Industry for Patient Reported Outcome Measures (PRO Guidance December 2009). 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). Question 1 is rated as Yes (score=0) or No (score=1); higher values indicate a worse outcome. Baseline was the DSQ score of the 14-day period before randomization. A negative change from baseline indicates that symptoms decreased.
Time Frame Baseline, Weeks 8, 12, and 16
Hide Outcome Measure Data
Hide Analysis Population Description
The MITT Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Least Squares Mean (Standard Error)
Unit of Measure: scores on a scale
Week 8, n = 37, 49 0.02  (0.050) -0.08  (0.043)
Week 12, n = 36, 49 0.05  (0.091) 0.02  (0.078)
Week 16, n = 38, 49 0.05  (0.079) 0.06  (0.069)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of Week 8
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1686
Comments Estimates are determined using an ANCOVA model including treatment group as a factor and baseline DSQ score as a covariate.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS mean difference
Estimated Value -0.09
Confidence Interval (2-Sided) 95%
-0.222 to 0.040
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of Week 12
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.8046
Comments Estimates are determined using an ANCOVA model including treatment group as a factor and baseline DSQ score as a covariate.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS mean difference
Estimated Value -0.03
Confidence Interval (2-Sided) 95%
-0.269 to 0.210
Estimation Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of Week 16
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.9239
Comments Estimates are determined using an ANCOVA model including treatment group as a factor and baseline DSQ score as a covariate.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS mean difference
Estimated Value 0.01
Confidence Interval (2-Sided) 95%
-0.199 to 0.219
Estimation Comments [Not Specified]
21.Secondary Outcome
Title Change From Baseline in The Scores of DSQ Question 4 During The Treatment Period
Hide Description Participants' dysphagia symptoms were evaluated using the 4-item DSQ. The questionnaire was developed by the Sponsor, as an ePRO measure, according to the principles of the Final Guidance for Industry for Patient Reported Outcome Measures (PRO Guidance December 2009). 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). Question 4 is rated as None, I had no pain (score=0), mild pain (score=1), moderate pain (score=2), severe pain (score=3), or very severe pain (score=4); 4 is the worst pain. Baseline was the DSQ score of the 14-day period before randomization. A negative change from baseline indicates that symptoms decreased.
Time Frame Baseline, Weeks 8, 12, and 16
Hide Outcome Measure Data
Hide Analysis Population Description
The MITT Analysis Set: all randomized participants who received at least 1 dose of double-blind study drug and had both an evaluable post-baseline biopsy during the treatment period and a post-baseline DSQ score. Participants were analyzed based on the randomization schedule, regardless of the treatment actually received.
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg
Hide Arm/Group Description:
Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Participants took 2 mg OBS (formulation MB-9) twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid.
Overall Number of Participants Analyzed 38 49
Least Squares Mean (Standard Error)
Unit of Measure: scores on a scale
Week 8, n = 37, 47 -2.52  (0.778) -3.25  (0.690)
Week 12, n = 36, 47 -2.97  (0.775) -4.89  (0.678)
Week 16, n = 38, 47 -3.08  (0.824) -4.87  (0.741)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of Week 8
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4835
Comments Estimates are determined using an ANCOVA model including treatment group as a factor and baseline DSQ score as a covariate.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS mean difference
Estimated Value -0.73
Confidence Interval (2-Sided) 95%
-2.806 to 1.339
Estimation Comments [Not Specified]
Show Statistical Analysis 2 Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of Week 12
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0662
Comments Estimates are determined using an ANCOVA model including treatment group as a factor and baseline DSQ score as a covariate.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS mean difference
Estimated Value -1.92
Confidence Interval (2-Sided) 95%
-3.974 to 0.132
Estimation Comments [Not Specified]
Show Statistical Analysis 3 Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Double-blind Placebo, Double-blind Oral Budesonide Suspension (OBS) 2 mg
Comments Analysis of Week 16
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1091
Comments Estimates are determined using an ANCOVA model including treatment group as a factor and baseline DSQ score as a covariate.
Method ANCOVA
Comments [Not Specified]
Method of Estimation Estimation Parameter LS mean difference
Estimated Value -1.80
Confidence Interval (2-Sided) 95%
-4.006 to 0.410
Estimation Comments [Not Specified]
Time Frame [Not Specified]
Adverse Event Reporting Description This section reports treatment-emergent AEs (TEAEs) for the Safety Analysis Set, which was defined as all randomized participants who received at least 1 dose of double-blind study drug (placebo or OBS). Adverse events experienced during the placebo baseline period were defined as events that began before the first dose of double-blind study drug.
 
Arm/Group Title Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg Placebo to Open-label Oral Budesonide Suspension (OBS) 2 mg Oral Budesonide Suspension (OBS) 2 mg to Open-label OBS 2 mg
Hide Arm/Group Description Participants took placebo twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid. Participants took 2mg OBS (formulation MB-9) 2 mg twice daily (bid) at home for 12 weeks. Study drug was administered once in the morning after breakfast and once in the evening at bedtime; the volume per dose was 10 mL of oral liquid. During the first 12 weeks of open-label extension treatment, participants took 2 mg OBS (formulation MB-9) once daily (qd) at bedtime. The volume per dose was 10 mL of oral liquid (10 mL qd; 0.2 mg/mL). Thereafter, an optional dose increase to 1.5 mg twice daily (bid) (7.5 mL bid; 0.2 mg/mL) and then to 2 mg bid (10 mL bid; 0.2 mg/mL) was allowed for subjects whose response to the 2 mg once daily dose was inadequate, as determined by the Investigator; a dose decrease to 2 mg once daily was allowed at any time. During the first 12 weeks of open-label extension treatment, participants took 2 mg OBS (formulation MB-9) once daily (qd) at bedtime. The volume per dose was 10 mL of oral liquid (10 mL qd; 0.2 mg/mL). Thereafter, an optional dose increase to 1.5 mg twice daily (bid) (7.5 mL bid; 0.2 mg/mL) and then to 2 mg bid (10 mL bid; 0.2 mg/mL) was allowed for subjects whose response to the 2 mg once daily dose was inadequate, as determined by the Investigator; a dose decrease to 2 mg once daily was allowed at any time.
All-Cause Mortality
Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg Placebo to Open-label Oral Budesonide Suspension (OBS) 2 mg Oral Budesonide Suspension (OBS) 2 mg to Open-label OBS 2 mg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg Placebo to Open-label Oral Budesonide Suspension (OBS) 2 mg Oral Budesonide Suspension (OBS) 2 mg to Open-label OBS 2 mg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   0/42 (0.00%)   1/51 (1.96%)   1/37 (2.70%)   0/45 (0.00%) 
Gastrointestinal disorders         
Food poisoning  1  0/42 (0.00%)  1/51 (1.96%)  0/37 (0.00%)  0/45 (0.00%) 
Injury, poisoning and procedural complications         
Hand Fracture  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Road Traffic Accident  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (17.0)
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 2%
Double-blind Placebo Double-blind Oral Budesonide Suspension (OBS) 2 mg Placebo to Open-label Oral Budesonide Suspension (OBS) 2 mg Oral Budesonide Suspension (OBS) 2 mg to Open-label OBS 2 mg
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   21/42 (50.00%)   20/51 (39.22%)   29/37 (78.38%)   27/45 (60.00%) 
Blood and lymphatic system disorders         
Leukopenia  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Lymphadenopathy  1  0/42 (0.00%)  1/51 (1.96%)  0/37 (0.00%)  1/45 (2.22%) 
Cardiac disorders         
Arrhythmia  1  1/42 (2.38%)  0/51 (0.00%)  0/37 (0.00%)  0/45 (0.00%) 
Ear and labyrinth disorders         
Ear discomfort  1  0/42 (0.00%)  0/51 (0.00%)  0/37 (0.00%)  1/45 (2.22%) 
Vertigo  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Endocrine disorders         
Goitre  1  0/42 (0.00%)  1/51 (1.96%)  0/37 (0.00%)  1/45 (2.22%) 
Gastrointestinal disorders         
Food poisoning  1  0/42 (0.00%)  2/51 (3.92%)  0/37 (0.00%)  0/45 (0.00%) 
Abdominal discomfort  1  1/42 (2.38%)  0/51 (0.00%)  0/37 (0.00%)  1/45 (2.22%) 
Abdominal pain  1  1/42 (2.38%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Abdominal pain lower  1  1/42 (2.38%)  0/51 (0.00%)  0/37 (0.00%)  0/45 (0.00%) 
Breath odour  1  1/42 (2.38%)  0/51 (0.00%)  0/37 (0.00%)  0/45 (0.00%) 
Constipation  1  1/42 (2.38%)  0/51 (0.00%)  0/37 (0.00%)  0/45 (0.00%) 
Diarrhoea  1  1/42 (2.38%)  1/51 (1.96%)  0/37 (0.00%)  2/45 (4.44%) 
Diarrhoea haemorrhagic  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Dyspepsia  1  1/42 (2.38%)  0/51 (0.00%)  0/37 (0.00%)  0/45 (0.00%) 
Eosinophilic oesophagitis  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Erosive oesophagitis  1  0/42 (0.00%)  0/51 (0.00%)  0/37 (0.00%)  1/45 (2.22%) 
Gastrooesophageal reflux disease  1  0/42 (0.00%)  0/51 (0.00%)  0/37 (0.00%)  1/45 (2.22%) 
Haematochezia  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Nausea  1  0/42 (0.00%)  1/51 (1.96%)  1/37 (2.70%)  0/45 (0.00%) 
Oesophageal food impaction  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Oral pain  1  1/42 (2.38%)  0/51 (0.00%)  0/37 (0.00%)  0/45 (0.00%) 
Stomatitis  1  1/42 (2.38%)  0/51 (0.00%)  0/37 (0.00%)  0/45 (0.00%) 
Toothache  1  1/42 (2.38%)  0/51 (0.00%)  0/37 (0.00%)  0/45 (0.00%) 
Vomiting  1  1/42 (2.38%)  1/51 (1.96%)  0/37 (0.00%)  1/45 (2.22%) 
General disorders         
Chest pain  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Discomfort  1  0/42 (0.00%)  0/51 (0.00%)  0/37 (0.00%)  1/45 (2.22%) 
Fatigue  1  0/42 (0.00%)  1/51 (1.96%)  0/37 (0.00%)  1/45 (2.22%) 
Pyrexia  1  1/42 (2.38%)  1/51 (1.96%)  1/37 (2.70%)  2/45 (4.44%) 
Vessel puncture site haematoma  1  1/42 (2.38%)  0/51 (0.00%)  0/37 (0.00%)  0/45 (0.00%) 
Vessel puncture site swelling  1  1/42 (2.38%)  0/51 (0.00%)  0/37 (0.00%)  0/45 (0.00%) 
Immune system disorders         
Allergy to arthropod sting  1  0/42 (0.00%)  0/51 (0.00%)  0/37 (0.00%)  1/45 (2.22%) 
Food allergy  1  0/42 (0.00%)  1/51 (1.96%)  1/37 (2.70%)  1/45 (2.22%) 
Hypersensitivity  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  1/45 (2.22%) 
Seasonal allergy  1  1/42 (2.38%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Infections and infestations         
Bronchitis  1  0/42 (0.00%)  0/51 (0.00%)  0/37 (0.00%)  1/45 (2.22%) 
Campylobacter infection  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Ear infection  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Gastroenteritis  1  0/42 (0.00%)  0/51 (0.00%)  0/37 (0.00%)  1/45 (2.22%) 
Gastroenteritis viral  1  1/42 (2.38%)  1/51 (1.96%)  0/37 (0.00%)  2/45 (4.44%) 
Gastrointestinal viral infection  1  0/42 (0.00%)  0/51 (0.00%)  0/37 (0.00%)  1/45 (2.22%) 
Hordeolum  1  0/42 (0.00%)  0/51 (0.00%)  0/37 (0.00%)  1/45 (2.22%) 
Influenza  1  1/42 (2.38%)  1/51 (1.96%)  1/37 (2.70%)  2/45 (4.44%) 
Nasopharyngitis  1  4/42 (9.52%)  3/51 (5.88%)  4/37 (10.81%)  5/45 (11.11%) 
Oesophageal candidiasis  1  0/42 (0.00%)  1/51 (1.96%)  4/37 (10.81%)  0/45 (0.00%) 
Oral candidiasis  1  0/42 (0.00%)  1/51 (1.96%)  1/37 (2.70%)  1/45 (2.22%) 
Oropharyngeal candidiasis  1  0/42 (0.00%)  0/51 (0.00%)  0/37 (0.00%)  1/45 (2.22%) 
Otitis media  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  1/45 (2.22%) 
Pharyngitis streptococcal  1  0/42 (0.00%)  0/51 (0.00%)  0/37 (0.00%)  1/45 (2.22%) 
Pilonidal cyst  1  0/42 (0.00%)  0/51 (0.00%)  0/37 (0.00%)  1/45 (2.22%) 
Post procedural infection  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Rhinitis  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Sinusitis  1  1/42 (2.38%)  2/51 (3.92%)  1/37 (2.70%)  1/45 (2.22%) 
Upper respiratory tract infection  1  2/42 (4.76%)  3/51 (5.88%)  4/37 (10.81%)  3/45 (6.67%) 
Urinary tract infection  1  0/42 (0.00%)  1/51 (1.96%)  0/37 (0.00%)  1/45 (2.22%) 
Vaginitis bacterial  1  0/42 (0.00%)  0/51 (0.00%)  0/37 (0.00%)  1/45 (2.22%) 
Viral infection  1  0/42 (0.00%)  1/51 (1.96%)  0/37 (0.00%)  2/45 (4.44%) 
Viral upper respiratory tract infection  1  0/42 (0.00%)  0/51 (0.00%)  0/37 (0.00%)  1/45 (2.22%) 
Vulvovaginal mycotic infection  1  1/42 (2.38%)  0/51 (0.00%)  2/37 (5.41%)  0/45 (0.00%) 
Injury, poisoning and procedural complications         
Wrist fracture  1  0/42 (0.00%)  0/51 (0.00%)  3/37 (8.11%)  0/45 (0.00%) 
Arthropod bite  1  1/42 (2.38%)  0/51 (0.00%)  1/37 (2.70%)  1/45 (2.22%) 
Arthropod sting  1  0/42 (0.00%)  0/51 (0.00%)  0/37 (0.00%)  1/45 (2.22%) 
Clavicle fracture  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Concussion  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Contusion  1  1/42 (2.38%)  0/51 (0.00%)  0/37 (0.00%)  0/45 (0.00%) 
Excoriation  1  0/42 (0.00%)  1/51 (1.96%)  1/37 (2.70%)  0/45 (0.00%) 
Foreign body  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Laceration  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Ligament sprain  1  1/42 (2.38%)  1/51 (1.96%)  1/37 (2.70%)  1/45 (2.22%) 
Post-traumatic neck syndrome  1  1/42 (2.38%)  0/51 (0.00%)  0/37 (0.00%)  0/45 (0.00%) 
Tendon rupture  1  0/42 (0.00%)  0/51 (0.00%)  0/37 (0.00%)  2/45 (4.44%) 
Investigations         
Eosinophil count increased  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Weight decreased  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  1/45 (2.22%) 
Metabolism and nutrition disorders         
Decreased appetite  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Iron deficiency  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Vitamin D deficiency  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Musculoskeletal and connective tissue disorders         
Arthralgia  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Back pain  1  1/42 (2.38%)  0/51 (0.00%)  0/37 (0.00%)  0/45 (0.00%) 
Bursitis  1  0/42 (0.00%)  0/51 (0.00%)  0/37 (0.00%)  1/45 (2.22%) 
Musculoskeletal stiffness  1  0/42 (0.00%)  0/51 (0.00%)  0/37 (0.00%)  1/45 (2.22%) 
Pain in jaw  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Nervous system disorders         
Dizziness  1  1/42 (2.38%)  0/51 (0.00%)  1/37 (2.70%)  1/45 (2.22%) 
Headache  1  0/42 (0.00%)  1/51 (1.96%)  3/37 (8.11%)  1/45 (2.22%) 
Sinus headache  1  1/42 (2.38%)  0/51 (0.00%)  0/37 (0.00%)  0/45 (0.00%) 
Tension headache  1  0/42 (0.00%)  1/51 (1.96%)  0/37 (0.00%)  1/45 (2.22%) 
Psychiatric disorders         
Stress  1  1/42 (2.38%)  0/51 (0.00%)  0/37 (0.00%)  0/45 (0.00%) 
Respiratory, thoracic and mediastinal disorders         
Asthma  1  1/42 (2.38%)  0/51 (0.00%)  3/37 (8.11%)  0/45 (0.00%) 
Asthma exercise induced  1  0/42 (0.00%)  0/51 (0.00%)  0/37 (0.00%)  1/45 (2.22%) 
Dysphonia  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Dyspnoea  1  0/42 (0.00%)  1/51 (1.96%)  1/37 (2.70%)  0/45 (0.00%) 
Increased upper airway secretion  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Nasal congestion  1  0/42 (0.00%)  1/51 (1.96%)  0/37 (0.00%)  1/45 (2.22%) 
Oropharyngeal pain  1  2/42 (4.76%)  2/51 (3.92%)  0/37 (0.00%)  1/45 (2.22%) 
Rales  1  0/42 (0.00%)  0/51 (0.00%)  0/37 (0.00%)  1/45 (2.22%) 
Skin and subcutaneous tissue disorders         
Acne  1  0/42 (0.00%)  1/51 (1.96%)  2/37 (5.41%)  0/45 (0.00%) 
Alopecia  1  1/42 (2.38%)  0/51 (0.00%)  0/37 (0.00%)  0/45 (0.00%) 
Dermatitis contact  1  0/42 (0.00%)  1/51 (1.96%)  0/37 (0.00%)  2/45 (4.44%) 
Eczema  1  1/42 (2.38%)  0/51 (0.00%)  0/37 (0.00%)  0/45 (0.00%) 
Rash  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  1/45 (2.22%) 
Skin lesion  1  0/42 (0.00%)  0/51 (0.00%)  1/37 (2.70%)  0/45 (0.00%) 
Urticaria  1  1/42 (2.38%)  0/51 (0.00%)  0/37 (0.00%)  0/45 (0.00%) 
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (17.0)
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
Results Point of Contact
Name/Title: Study Physician
Organization: Shire Development LLC
Phone: +1 866 842 5335
Responsible Party: Shire
ClinicalTrials.gov Identifier: NCT01642212     History of Changes
Other Study ID Numbers: MPI-101-06
First Submitted: July 13, 2012
First Posted: July 17, 2012
Results First Submitted: September 30, 2015
Results First Posted: December 7, 2015
Last Update Posted: December 7, 2015