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Dose-Ranging Study of Oral Viscous Budesonide in Pediatrics With Eosinophilic Esophagitis

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ClinicalTrials.gov Identifier: NCT00762073
Recruitment Status : Completed
First Posted : September 30, 2008
Results First Posted : October 5, 2015
Last Update Posted : June 11, 2021
Sponsor:
Information provided by (Responsible Party):
Takeda ( 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 (EoE)
Interventions Drug: budesonide
Drug: placebo
Enrollment 82
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Placebo Low Dose Medium Dose High Dose
Hide Arm/Group Description Participants received placebo twice daily at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks with a 3 week taper period. Participants received oral budesonide suspension (OBS) 0.05 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 0.35 mg (2 to 9 years) or 0.50 mg (10 to 18 years), followed by a 3 week taper period. Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 1.4 mg (2 to 9 years) or 2.0 mg (10 to 18 years), followed by a 3 week taper period. Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 2.8 mg (2 to 9 years) or 4.0 mg (10 to 18 years), followed by a 3 week taper period.
Period Title: Overall Study
Started 21 21 20 20
Completed 17 17 18 [1] 17
Not Completed 4 4 2 3
Reason Not Completed
Lack of Efficacy             1             1             0             0
Adverse Event             1             0             1             1
Significant Subject Noncompliance             0             1             0             1
Withdrawal by Subject             1             2             1             1
Not on treatment for >/= 77 days             1             0             0             0
[1]
One participant was enrolled and randomized then withdrew consent and did not receive study drug.
Arm/Group Title Placebo Low Dose Medium Dose High Dose Total
Hide Arm/Group Description Participants received placebo twice daily at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks with a 3 week taper period. Participants received oral budesonide suspension (OBS) 0.05 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 0.35 mg (2 to 9 years) or 0.50 mg (10 to 18 years), followed by a 3 week taper period. Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 1.4 mg (2 to 9 years) or 2.0 mg (10 to 18 years), followed by a 3 week taper period. Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 2.8 mg (2 to 9 years) or 4.0 mg (10 to 18 years), followed by a 3 week taper period. Total of all reporting groups
Overall Number of Baseline Participants 21 21 19 20 81
Hide Baseline Analysis Population Description
The Safety Analysis Set, defined as all randomized participants who received at least one dose of double-blind study drug. Treatment group assignment was the treatment actually received. One participant in the medium dose group was enrolled and randomized then withdrew consent and did not receive study drug.
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 21 participants 21 participants 19 participants 20 participants 81 participants
9.2  (4.36) 9.0  (5.88) 10.2  (4.89) 8.1  (4.58) 9.1  (4.93)
Age, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants 21 participants 19 participants 20 participants 81 participants
2 to 9 years
12
  57.1%
12
  57.1%
10
  52.6%
12
  60.0%
46
  56.8%
10 to 18 years
9
  42.9%
9
  42.9%
9
  47.4%
8
  40.0%
35
  43.2%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 21 participants 21 participants 19 participants 20 participants 81 participants
Female
5
  23.8%
4
  19.0%
2
  10.5%
4
  20.0%
15
  18.5%
Male
16
  76.2%
17
  81.0%
17
  89.5%
16
  80.0%
66
  81.5%
1.Primary Outcome
Title Percent of Participants Who Responded to Therapy
Hide Description Response was defined as a ≥50% reduction from baseline in the eosinophilic esophagitis (EoE) clinical symptom score (CSS) and a reduction in peak eosinophil count to ≤6/high power field (light microscopy) from esophageal biopsies collected at the final evaluation. The EoE CSS, scored from 0 to 18 by a doctor, assessed 6 categories: 1) heartburn, 2) abdominal pain, 3) nocturnal awakening with symptoms, 4) nausea, regurgitation, or vomiting, 5) anorexia or early satiety, and 6) dysphagia, odynophagia, or food impaction (a severe symptom). Each domain was scored as follows, based on symptoms in the 2 weeks prior to the assessment: 0 = No symptoms and no coping behaviors required; 1 = Mild: Symptoms limited to 1-3 days or no symptoms because coping behaviors were required to avoid symptoms; 2 = Moderate: Symptoms on >3 days, with or without minor coping behaviors; 3 = Severe: Symptoms interfered with activities of daily living or symptoms persisted and required major coping behaviors.
Time Frame 12 weeks after the start of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
The Full Analysis Set (FAS), defined as participants who received at least one dose of study drug and had evaluable post-baseline esophageal biopsy and clinical symptom data.
Arm/Group Title Placebo Low Dose Medium Dose High Dose
Hide Arm/Group Description:
Participants received placebo twice daily at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.05 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 0.35 mg (2 to 9 years) or 0.50 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 1.4 mg (2 to 9 years) or 2.0 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 2.8 mg (2 to 9 years) or 4.0 mg (10 to 18 years), followed by a 3 week taper period.
Overall Number of Participants Analyzed 18 17 19 17
Measure Type: Number
Unit of Measure: percentage of participants
5.6 11.8 52.6 47.1
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Low Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5282
Comments p-values comparing each active treatment group to placebo were determined by a logistic regression analysis with treatment (all four groups) and age as main effects.
Method Regression, Logistic
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 2.239
Estimation Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Medium Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0092
Comments p-values comparing each active treatment group to placebo were determined by a logistic regression analysis with treatment (all four groups) and age as main effects.
Method Regression, Logistic
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 18.860
Estimation Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo, High Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0174
Comments p-values comparing each active treatment group to placebo were determined by a logistic regression analysis with treatment (all four groups) and age as main effects.
Method Regression, Logistic
Comments [Not Specified]
Method of Estimation Estimation Parameter Odds Ratio (OR)
Estimated Value 15.009
Estimation Comments [Not Specified]
2.Secondary Outcome
Title Percent of Participants With Histologic Response
Hide Description Histologic response was defined as a maximum peak eosinophil count at the final treatment evaluation of ≤6 eosinophils/high power field (light microscopy). The maximum peak was identified by examining the peak eosinophil counts obtained from the proximal, mid, and distal esophageal biopsies and selecting the maximum value.
Time Frame 12 weeks after the start of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS, defined as participants who received at least one dose of study drug and had evaluable post-baseline esophageal biopsy and clinical symptom data.
Arm/Group Title Placebo Low Dose Medium Dose High Dose
Hide Arm/Group Description:
Participants received placebo twice daily at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.05 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 0.35 mg (2 to 9 years) or 0.50 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 1.4 mg (2 to 9 years) or 2.0 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 2.8 mg (2 to 9 years) or 4.0 mg (10 to 18 years), followed by a 3 week taper period.
Overall Number of Participants Analyzed 18 17 19 17
Measure Type: Number
Unit of Measure: percentage of participants
5.6 23.5 52.6 94.1
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Low Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1786
Comments p-values were determined by a logistic regression analysis with treatment (all four groups) and age group as main effects.
Method Regression, Logistic
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Medium Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0090
Comments p-values were determined by a logistic regression analysis with treatment (all four groups) and age group as main effects.
Method Regression, Logistic
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo, High Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0001
Comments p-values were determined by a logistic regression analysis with treatment (all four groups) and age group as main effects.
Method Regression, Logistic
Comments [Not Specified]
3.Secondary Outcome
Title Percent of Participants With Histologic Remission
Hide Description Histologic remission was defined as a maximum peak eosinophil count at the final treatment evaluation of ≤1 eosinophils/high power field (light microscopy). The maximum peak was identified by examining the peak eosinophil counts obtained from the proximal, mid, and distal esophageal biopsies and selecting the maximum value.
Time Frame 12 weeks after the start of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS, defined as participants who received at least one dose of study drug and had evaluable post-baseline esophageal biopsy and clinical symptom data.
Arm/Group Title Placebo Low Dose Medium Dose High Dose
Hide Arm/Group Description:
Participants received placebo twice daily at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.05 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 0.35 mg (2 to 9 years) or 0.50 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 1.4 mg (2 to 9 years) or 2.0 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 2.8 mg (2 to 9 years) or 4.0 mg (10 to 18 years), followed by a 3 week taper period.
Overall Number of Participants Analyzed 18 17 19 17
Measure Type: Number
Unit of Measure: percentage of participants
0.0 11.8 42.1 76.5
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Low Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4959
Comments p-values were determined by a logistic regression analysis with treatment (all four groups) and age group as main effects.
Method Regression, Logistic
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Medium Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0040
Comments p-values were determined by a logistic regression analysis with treatment (all four groups) and age group as main effects.
Method Regression, Logistic
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo, High Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments p-values were determined by a logistic regression analysis with treatment (all four groups) and age group as main effects.
Method Regression, Logistic
Comments [Not Specified]
4.Secondary Outcome
Title Percent Change From Baseline in Peak Eosinophil Count
Hide Description The maximum peak number of eosinophils at baseline and at the final treatment evaluation was identified by examining the peak eosinophil counts obtained from the proximal, mid, and distal esophageal biopsies and selecting the maximum value. A negative change from baseline indicates that eosinophil count has decreased.
Time Frame Baseline, 12 weeks after the start of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS, defined as all participants who received at least one dose of study drug and had evaluable post-baseline esophageal biopsy and clinical symptom data.
Arm/Group Title Placebo Low Dose Medium Dose High Dose
Hide Arm/Group Description:
Participants received placebo twice daily at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.05 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 0.35 mg (2 to 9 years) or 0.50 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 1.4 mg (2 to 9 years) or 2.0 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 2.8 mg (2 to 9 years) or 4.0 mg (10 to 18 years), followed by a 3 week taper period.
Overall Number of Participants Analyzed 18 17 19 17
Mean (Standard Deviation)
Unit of Measure: percent change
7.93  (84.162) -52.62  (51.22) -44.02  (89.106) -94.75  (19.615)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Low Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0108
Comments p-values comparing percent change from baseline for each active treatment group to placebo were determined by an ANCOVA model with treatment and age strata as main effects and baseline as a covariate.
Method ANCOVA
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Medium Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0208
Comments p-values comparing percent change from baseline for each active treatment group to placebo were determined by an ANCOVA model with treatment and age strata as main effects and baseline as a covariate.
Method ANCOVA
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo, High Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.0001
Comments p-values comparing percent change from baseline for each active treatment group to placebo were determined by an ANCOVA model with treatment and age strata as main effects and baseline as a covariate.
Method ANCOVA
Comments [Not Specified]
5.Secondary Outcome
Title Change From Baseline in Endoscopy Score
Hide Description Esophageal endoscopy was used to assess the level of inflammation and eosinophilia. Four categories of endoscopic findings were evaluated and scored for this study: (1) pallor and diminished vascular markings; (2) furrowing with thickened mucosa; (3) presence of white mucosal plaques; and (4) concentric rings or strictures. For each category, 0 points were allocated if no esophageal sites were involved, 1 point if 1 or 2 esophageal sites were involved, and 2 points for pan-esophageal involvement (see Aceves et al., 2007). The maximum possible endoscopy score was 8 points. A negative change from baseline indicates that esophageal inflammation decreased.
Time Frame Baseline, 12 weeks after the start of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS, defined as all participants who received at least one dose of study drug and had evaluable post-baseline esophageal biopsy and clinical symptom data.
Arm/Group Title Placebo Low Dose Medium Dose High Dose
Hide Arm/Group Description:
Participants received placebo twice daily at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.05 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 0.35 mg (2 to 9 years) or 0.50 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 1.4 mg (2 to 9 years) or 2.0 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 2.8 mg (2 to 9 years) or 4.0 mg (10 to 18 years), followed by a 3 week taper period.
Overall Number of Participants Analyzed 18 17 19 17
Mean (Standard Deviation)
Unit of Measure: scores on a scale
-0.6  (2.28) -0.9  (1.90) -1.3  (2.23) -2.2  (1.81)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Low Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1095
Comments p-values comparing each active treatment group to placebo were determined by an ANCOVA model with treatment and age strata as main effects and baseline as a covariate
Method ANCOVA
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Medium Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0264
Comments p-values comparing each active treatment group to placebo were determined by an ANCOVA model with treatment and age strata as main effects and baseline as a covariate
Method ANCOVA
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo, High Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0010
Comments p-values comparing each active treatment group to placebo were determined by an ANCOVA model with treatment and age strata as main effects and baseline as a covariate
Method ANCOVA
Comments [Not Specified]
6.Secondary Outcome
Title Percent of Participants With Clinical Response
Hide Description Response was defined as a ≥50% reduction from baseline in the eosinophilic esophagitis (EoE) clinical symptom score (CSS). The EoE CSS, scored from 0 to 18 by a doctor, assessed 6 categories: 1) heartburn, 2) abdominal pain, 3) nocturnal awakening with symptoms, 4) nausea, regurgitation, or vomiting, 5) anorexia or early satiety, and 6) dysphagia, odynophagia, or food impaction (a severe symptom). Each domain was scored as follows, based on symptoms in the 2 weeks prior to the assessment: 0 = No symptoms and no coping behaviors required; 1 = Mild: Symptoms limited to 1-3 days or no symptoms because coping behaviors were required to avoid symptoms; 2 = Moderate: Symptoms on >3 days, with or without minor coping behaviors; 3 = Severe: Symptoms interfered with activities of daily living or symptoms persisted and required major coping behaviors.
Time Frame 12 weeks after the start of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS, defined as participants who received at least one dose of study drug and had evaluable post-baseline esophageal biopsy and clinical symptom data.
Arm/Group Title Placebo Low Dose Medium Dose High Dose
Hide Arm/Group Description:
Participants received placebo twice daily at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.05 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 0.35 mg (2 to 9 years) or 0.50 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 1.4 mg (2 to 9 years) or 2.0 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 2.8 mg (2 to 9 years) or 4.0 mg (10 to 18 years), followed by a 3 week taper period.
Overall Number of Participants Analyzed 18 17 19 17
Measure Type: Number
Unit of Measure: percentage of participants
77.8 64.7 78.9 52.9
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Low Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3769
Comments p-values comparing each active treatment group to placebo were determined by a logistic regression analysis with treatment (all four groups) and age as main effects.
Method Regression, Logistic
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Medium Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.9363
Comments p-values comparing each active treatment group to placebo were determined by a logistic regression analysis with treatment (all four groups) and age as main effects.
Method Regression, Logistic
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo, High Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1235
Comments p-values comparing each active treatment group to placebo were determined by a logistic regression analysis with treatment (all four groups) and age as main effects.
Method Regression, Logistic
Comments [Not Specified]
7.Secondary Outcome
Title Percent of Participants With Clinical Remission
Hide Description Clinical remission was defined as an eosinophilic esophagitis (EoE) clinical symptom score (CSS) of zero. EoE CSS, scored from 0 to 18 by a doctor, assessed 6 categories: 1) heartburn, 2) abdominal pain, 3) nocturnal awakening with symptoms, 4) nausea, regurgitation, or vomiting, 5) anorexia or early satiety, and 6) dysphagia, odynophagia, or food impaction (a severe symptom). Each domain was scored as follows, based on symptoms in the 2 weeks prior to the assessment: 0 = No symptoms and no coping behaviors required; 1 = Mild: Symptoms limited to 1-3 days or no symptoms because coping behaviors were required to avoid symptoms; 2 = Moderate: Symptoms on >3 days, with or without minor coping behaviors; 3 = Severe: Symptoms interfered with activities of daily living or symptoms persisted and required major coping behaviors.
Time Frame 12 weeks after the start of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS, defined as all participants who received at least one dose of study drug and had evaluable post-baseline esophageal biopsy and clinical symptom data.
Arm/Group Title Placebo Low Dose Medium Dose High Dose
Hide Arm/Group Description:
Participants received placebo twice daily at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.05 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 0.35 mg (2 to 9 years) or 0.50 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 1.4 mg (2 to 9 years) or 2.0 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 2.8 mg (2 to 9 years) or 4.0 mg (10 to 18 years), followed by a 3 week taper period.
Overall Number of Participants Analyzed 18 17 19 17
Measure Type: Number
Unit of Measure: percentage of participants
33.3 17.6 31.6 17.6
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Low Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3444
Comments p-values comparing each active treatment group to placebo were determined by a logistic regression analysis with treatment (all four groups) and age as main effects.
Method Regression, Logistic
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Medium Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.9258
Comments p-values comparing each active treatment group to placebo were determined by a logistic regression analysis with treatment (all four groups) and age as main effects.
Method Regression, Logistic
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo, High Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3215
Comments p-values comparing each active treatment group to placebo were determined by a logistic regression analysis with treatment (all four groups) and age as main effects.
Method Regression, Logistic
Comments [Not Specified]
8.Secondary Outcome
Title Percent Change From Baseline in Eosinophilic Esophagitis (EoE) Clinical Symptom Score (CSS)
Hide Description The EoE CSS, scored from 0 to 18 by a doctor, assessed 6 categories: 1) heartburn, 2) abdominal pain, 3) nocturnal awakening with symptoms, 4) nausea, regurgitation, or vomiting, 5) anorexia or early satiety, and 6) dysphagia, odynophagia, or food impaction (a severe symptom). Each domain was scored as follows, based on symptoms in the 2 weeks prior to the assessment: 0 = No symptoms and no coping behaviors required; 1= Mild: Symptoms limited to 1-3 days or no symptoms because coping behaviors were required to avoid symptoms; 2= Moderate: Symptoms on >3 days, with or without minor coping behaviors; 3= Severe: Symptoms interfered with activities of daily living or symptoms persisted and required major coping behaviors. A negative change from baseline indicates that symptoms decreased.
Time Frame Baseline, 12 weeks after the start of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS, defined as all participants who received at least one dose of study drug and had evaluable post-baseline esophageal biopsy and clinical symptom data.
Arm/Group Title Placebo Low Dose Medium Dose High Dose
Hide Arm/Group Description:
Participants received placebo twice daily at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.05 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 0.35 mg (2 to 9 years) or 0.50 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 1.4 mg (2 to 9 years) or 2.0 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 2.8 mg (2 to 9 years) or 4.0 mg (10 to 18 years), followed by a 3 week taper period.
Overall Number of Participants Analyzed 18 17 19 17
Mean (Standard Deviation)
Unit of Measure: percent change
-64.46  (45.759) -60.83  (30.347) -65.89  (32.382) -47.21  (40.790)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Low Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6729
Comments p-values comparing percent change from Baseline for each active treatment group to placebo were determined by an ANCOVA model with treatment and age strata as main effects and baseline as a covariate.
Method ANCOVA
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Medium Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.8894
Comments p-values comparing percent change from Baseline for each active treatment group to placebo were determined by an ANCOVA model with treatment and age strata as main effects and baseline as a covariate.
Method ANCOVA
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo, High Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1532
Comments p-values comparing percent change from Baseline for each active treatment group to placebo were determined by an ANCOVA model with treatment and age strata as main effects and baseline as a covariate.
Method ANCOVA
Comments [Not Specified]
9.Secondary Outcome
Title Change From Baseline in Physician's Global Assessment Score of Disease Severity
Hide Description Physician investigators were asked to complete a visual analog scale (VAS) to provide a global assessment of eosinophilic esophagitis (EoE) activity in each participant. The VAS was a 100-mm horizontal line on which the right extreme (100) was labeled "worst possible disease activity" and the left (0) was labeled "no disease activity." Investigators were instructed to consider the line for the VAS as a continuum with their own opinion of extremes on either end. Investigators drew a vertical line at a point that best approximated the participant's current level of EoE disease activity. The investigator was to take into consideration how esophageal disease was impacting the participant's daily activities. The following instruction was given to the investigators: "Using the visual analog scale below, please mark a vertical line on the scale to indicate your assessment of EoE activity in this participant at this time." A negative change from baseline indicates that symptoms decreased.
Time Frame Baseline, 12 weeks after the start of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
The FAS, defined as all participants who received at least one dose of study drug and had evaluable post-baseline esophageal biopsy and clinical symptom data.
Arm/Group Title Placebo Low Dose Medium Dose High Dose
Hide Arm/Group Description:
Participants received placebo twice daily at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.05 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 0.35 mg (2 to 9 years) or 0.50 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 1.4 mg (2 to 9 years) or 2.0 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 2.8 mg (2 to 9 years) or 4.0 mg (10 to 18 years), followed by a 3 week taper period.
Overall Number of Participants Analyzed 18 17 19 16
Mean (Standard Deviation)
Unit of Measure: scores on a scale
-38.9  (28.02) -30.2  (27.11) -39.3  (22.89) -35.7  (28.49)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Placebo, Low Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.4197
Comments p-values comparing each active treatment group to placebo were determined by an ANCOVA model with treatment and age strata as main effects and baseline as a covariate.
Method ANCOVA
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Placebo, Medium Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.9787
Comments p-values comparing each active treatment group to placebo were determined by an ANCOVA model with treatment and age strata as main effects and baseline as a covariate.
Method ANCOVA
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection Placebo, High Dose
Comments [Not Specified]
Type of Statistical Test Superiority or Other (legacy)
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.8987
Comments p-values comparing each active treatment group to placebo were determined by an ANCOVA model with treatment and age strata as main effects and baseline as a covariate.
Method ANCOVA
Comments [Not Specified]
10.Secondary Outcome
Title Maximum Plasma Concentration (Cmax) of Budesonide
Hide Description On the day that pharmacokinetic (PK) blood samples were obtained, each participant delayed the morning dose of study medication until instructed to dose in the clinic. The sampling timepoints included pre-dose (0), and 0.5, 1, 2, 3, 4, 6, and 8 hours post-dose. The lower limit of quantitation (LLOQ) for the analytical method was approximately 20 pg/mL in plasma using 0.2 mL of the sample. Because the PK analyses for the medium and high dose oral budesonide suspension (OBS) groups were based on plasma samples collected following administration of identical single doses of OBS, the data for the medium-dose group (OBS once-daily) and high-dose group (OBS twice-daily) were summarized together.
Time Frame Week 2, 4, or 8, or at the Final Treatment Evaluation
Hide Outcome Measure Data
Hide Analysis Population Description
The Pharmacokinetic (PK) Set, defined as all participants in the safety analysis set who received oral budesonide suspension (OBS) and had sufficient PK samples to calculate PK parameters.
Arm/Group Title 0.35 mg Dose 0.50 mg Dose 1.4 mg Dose 2.0 mg Dose
Hide Arm/Group Description:
Participants 2 to 9 years old received oral budesonide suspension (OBS) 0.05 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc), with a total daily dose of 0.35 mg.
Participants 10 to 18 years old received oral budesonide suspension (OBS) 0.05 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc), with a total daily dose of 0.50 mg.
Participants 2 to 9 years old received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and either placebo or OBS 0.2 mg/ml after breakfast (qAM, pc), with a total daily dose of 1.4 mg (medium dose group) or 2.8 mg (high dose group).
Participants 10 to 18 years old received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and either placebo or OBS 0.2 mg/ml after breakfast (qAM, pc), with a total daily dose of 2.0 mg (medium dose group) or 4.0 mg (high dose group).
Overall Number of Participants Analyzed 4 5 15 13
Mean (Standard Deviation)
Unit of Measure: pg/mL
492.0  (417.81) 195.0  (64.37) 1019.5  (670.18) 958.4  (527.64)
11.Secondary Outcome
Title Time to Maximum (Tmax) And Half Maximum (T1/2) Plasma Concentration of Budesonide
Hide Description On the day that pharmacokinetic (PK) blood samples were obtained, each participant delayed the morning dose of study medication until instructed to dose in the clinic. The sampling timepoints included pre-dose (0), and 0.5, 1, 2, 3, 4, 6, and 8 hours post-dose. The lower limit of quantitation (LLOQ) for the analytical method was approximately 20 pg/mL in plasma using 0.2 mL of the sample. Because the PK analyses for the medium and high dose oral budesonide suspension (OBS) groups were based on plasma samples collected following administration of identical single doses of OBS, the data for the medium-dose group (OBS once-daily) and high-dose group (OBS twice-daily) were summarized together. T1/2 is the time to terminal elimination half-life.
Time Frame Week 2, 4, or 8, or at the Final Treatment Evaluation
Hide Outcome Measure Data
Hide Analysis Population Description
The PK Set, defined as all participants in the safety analysis set who received oral budesonide suspension (OBS) and had sufficient PK samples to calculate PK parameters.
Arm/Group Title 0.35 mg Dose 0.50 mg Dose 1.4 mg Dose 2.0 mg Dose
Hide Arm/Group Description:
Participants 2 to 9 years old received oral budesonide suspension (OBS) 0.05 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc), with a total daily dose of 0.35 mg.
Participants 10 to 18 years old received oral budesonide suspension (OBS) 0.05 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc), with a total daily dose of 0.50 mg.
Participants 2 to 9 years old received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and either placebo or OBS 0.2 mg/ml after breakfast (qAM, pc), with a total daily dose of 1.4 mg (medium dose group) or 2.8 mg (high dose group).
Participants 10 to 18 years old received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and either placebo or OBS 0.2 mg/ml after breakfast (qAM, pc), with a total daily dose of 2.0 mg (medium dose group) or 4.0 mg (high dose group).
Overall Number of Participants Analyzed 4 5 15 13
Mean (Standard Deviation)
Unit of Measure: hours
T1/2 3.288  (0.8265) 3.398  (0.7841) 3.472  (2.6753) 3.528  (1.0223)
Tmax 0.68  (0.360) 1.20  (0.447) 0.93  (0.372) 1.12  (0.546)
12.Secondary Outcome
Title Area Under The Plasma Concentration-Time Curve (AUC) of Budesonide From Time Zero to Time of The Last Measurable Concentration (AUC0-last)
Hide Description On the day that pharmacokinetic (PK) blood samples were obtained, each participant delayed the morning dose of study medication until instructed to dose in the clinic. The sampling timepoints included pre-dose (0), and 0.5, 1, 2, 3, 4, 6, and 8 hours post-dose. The lower limit of quantitation (LLOQ) for the analytical method was approximately 20 pg/mL in plasma using 0.2 mL of the sample. Because the PK analyses for the medium and high dose oral budesonide suspension (OBS) groups were based on plasma samples collected following administration of identical single doses of OBS, the data for the medium-dose group (OBS once-daily) and high-dose group (OBS twice-daily) were summarized together.
Time Frame Week 2, 4, or 8, or at the Final Treatment Evaluation
Hide Outcome Measure Data
Hide Analysis Population Description
The PK Set, defined as all participants in the safety analysis set who received oral budesonide suspension (OBS) and had sufficient PK samples to calculate PK parameters.
Arm/Group Title 0.35 mg Dose 0.50 mg Dose 1.4 mg Dose 2.0 mg Dose
Hide Arm/Group Description:
Participants 2 to 9 years received oral budesonide suspension (OBS) 0.05 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc), with a total daily dose of 0.35 mg.
Participants 10 to 18 years received oral budesonide suspension (OBS) 0.05 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc), with a total daily dose of 0.50 mg.
Participants 2 to 9 years old received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and either placebo or OBS 0.2 mg/ml after breakfast (qAM, pc), with a total daily dose of 1.4 mg (medium dose group) or 2.8 mg (high dose group).
Participants 10 to 18 years received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and either placebo or OBS 0.2 mg/ml after breakfast (qAM, pc), with a total daily dose of 2.0 mg (medium dose group) or 4.0 mg (high dose group).
Overall Number of Participants Analyzed 4 5 15 13
Mean (Standard Deviation)
Unit of Measure: hr*pg/mL
1139.5  (800.84) 743.8  (425.26) 3259.3  (2109.37) 3636.9  (1769.88)
13.Secondary Outcome
Title Percent of Participants With Potential Corticosteroid-Related Treatment-Emergent Adverse Events (TEAEs)
Hide Description Corticosteroid-Related TEAEs included candidiasis, oesophageal candidiasis, crying, psychomotor hyperactivity, aggression, anger, anxiety, conduct disorder, emotional disorder, insomnia, or mood altered mood. Corticosteroid-Related TEAEs were assessed systematically during the treatment and taper periods.
Time Frame 15 weeks after the start of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Analysis Set, defined as all randomized participants who received at least one dose of double-blind study drug.
Arm/Group Title Placebo Low Dose Medium Dose High Dose
Hide Arm/Group Description:
Participants received placebo twice daily at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.05 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 0.35 mg (2 to 9 years) or 0.50 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 1.4 mg (2 to 9 years) or 2.0 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 2.8 mg (2 to 9 years) or 4.0 mg (10 to 18 years), followed by a 3 week taper period.
Overall Number of Participants Analyzed 21 21 19 20
Measure Type: Number
Unit of Measure: percentage of participants
9.5 9.5 21.0 15.0
14.Secondary Outcome
Title Mean Change in Blood Pressure (BP) at End of Treatment
Hide Description BP was assessed for each treatment group at baseline and at each post-baseline visit including the final treatment evaluation.
Time Frame Baseline, 12 weeks after the start of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
The Safety Analysis Set, defined as all randomized participants who received at least one dose of double-blind study drug.
Arm/Group Title Placebo Low Dose Medium Dose High Dose
Hide Arm/Group Description:
Participants received placebo twice daily at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.05 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 0.35 mg (2 to 9 years) or 0.50 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 1.4 mg (2 to 9 years) or 2.0 mg (10 to 18 years), followed by a 3 week taper period.
Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks, with a total daily dose of 2.8 mg (2 to 9 years) or 4.0 mg (10 to 18 years), followed by a 3 week taper period.
Overall Number of Participants Analyzed 21 21 19 20
Mean (Standard Deviation)
Unit of Measure: mmHg
Systolic BP -1.5  (14.74) 1.8  (11.13) 3.5  (8.20) 8.0  (13.58)
Diastolic BP -3.1  (10.31) 0.5  (8.89) 3.1  (9.18) 5.1  (8.83)
Time Frame [Not Specified]
Adverse Event Reporting Description Adverse events are presented for the Safety Analysis Set, defined as all randomized participants who received at least one dose of double-blind study drug. Treatment group assignment was the treatment actually received. One participant in the medium dose group was enrolled and randomized then withdrew consent and did not receive study drug.
 
Arm/Group Title Placebo Low Dose Medium Dose High Dose
Hide Arm/Group Description Participants received placebo twice daily at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks with a 3 week taper period. Participants received oral budesonide suspension (OBS) 0.05 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks with a 3 week taper period. Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and placebo after breakfast (qAM, pc) for 12 weeks with a 3 week taper period. Participants received oral budesonide suspension (OBS) 0.2 mg/mL at bedtime (hs) and after breakfast (qAM, pc) for 12 weeks with a 3 week taper period.
All-Cause Mortality
Placebo Low Dose Medium Dose High Dose
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--      --/--      --/--    
Hide Serious Adverse Events
Placebo Low Dose Medium Dose High Dose
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/21 (0.00%)      0/21 (0.00%)      0/19 (0.00%)      1/20 (5.00%)    
Metabolism and nutrition disorders         
Diet refusal  1  0/21 (0.00%)  0 0/21 (0.00%)  0 0/19 (0.00%)  0 1/20 (5.00%)  1
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (12.0)
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Placebo Low Dose Medium Dose High Dose
Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   10/21 (47.62%)      13/21 (61.90%)      16/19 (84.21%)      17/20 (85.00%)    
Eye disorders         
Conjunctivitis  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Gastrointestinal disorders         
Diarrhoea  1  0/21 (0.00%)  0 3/21 (14.29%)  5 1/19 (5.26%)  1 3/20 (15.00%)  4
Constipation  1  0/21 (0.00%)  0 1/21 (4.76%)  1 0/19 (0.00%)  0 3/20 (15.00%)  3
Vomiting  1  2/21 (9.52%)  2 0/21 (0.00%)  0 2/19 (10.53%)  2 2/20 (10.00%)  2
Abdominal pain  1  1/21 (4.76%)  1 1/21 (4.76%)  1 1/19 (5.26%)  1 1/20 (5.00%)  1
Nausea  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 1/20 (5.00%)  2
Abdominal pain upper  1  0/21 (0.00%)  0 0/21 (0.00%)  0 0/19 (0.00%)  0 2/20 (10.00%)  3
Aphthous stomatitis  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Flatulence  1  0/21 (0.00%)  0 0/21 (0.00%)  0 0/19 (0.00%)  0 1/20 (5.00%)  1
Oral pain  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Teething  1  0/21 (0.00%)  0 0/21 (0.00%)  0 0/19 (0.00%)  0 1/20 (5.00%)  1
General disorders         
Pyrexia  1  3/21 (14.29%)  3 3/21 (14.29%)  3 2/19 (10.53%)  2 4/20 (20.00%)  5
Early Satiety  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Feeling jittery  1  0/21 (0.00%)  0 0/21 (0.00%)  0 0/19 (0.00%)  0 1/20 (5.00%)  1
Pain  1  0/21 (0.00%)  0 1/21 (4.76%)  1 1/19 (5.26%)  1 0/20 (0.00%)  0
Thirst  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Immune system disorders         
Milk allergy  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Infections and infestations         
Sinusitis  1  1/21 (4.76%)  1 2/21 (9.52%)  3 2/19 (10.53%)  2 3/20 (15.00%)  4
Nasopharyngitis  1  1/21 (4.76%)  1 2/21 (9.52%)  2 3/19 (15.79%)  5 1/20 (5.00%)  1
Influenza  1  0/21 (0.00%)  0 1/21 (4.76%)  1 2/19 (10.53%)  3 1/20 (5.00%)  1
Ear infection  1  0/21 (0.00%)  0 1/21 (4.76%)  1 1/19 (5.26%)  1 0/20 (0.00%)  0
Upper respiratory tract infection  1  0/21 (0.00%)  0 1/21 (4.76%)  1 0/19 (0.00%)  0 2/20 (10.00%)  2
Candidiasis  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Oesophageal candidiasis  1  0/21 (0.00%)  0 0/21 (0.00%)  0 0/19 (0.00%)  0 1/20 (5.00%)  1
Pharyngitis  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Respiratory tract infection viral  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Viral infection  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Bronchitis  1  0/21 (0.00%)  0 0/21 (0.00%)  0 0/19 (0.00%)  0 1/20 (5.00%)  1
Injury, poisoning and procedural complications         
Animal bite  1  0/21 (0.00%)  0 0/21 (0.00%)  0 0/19 (0.00%)  0 1/20 (5.00%)  1
Arthropod sting  1  0/21 (0.00%)  0 0/21 (0.00%)  0 0/19 (0.00%)  0 1/20 (5.00%)  1
Meniscus lesion  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Upper limb fracture  1  1/21 (4.76%)  1 0/21 (0.00%)  0 0/19 (0.00%)  0 1/20 (5.00%)  1
Investigations         
Blood alkaline phosphatase increased  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Blood pressure increased  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Weight decreased  1  0/21 (0.00%)  0 1/21 (4.76%)  1 0/19 (0.00%)  0 1/20 (5.00%)  1
Metabolism and nutrition disorders         
Dehydration  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Failure to thrive  1  0/21 (0.00%)  0 0/21 (0.00%)  0 0/19 (0.00%)  0 1/20 (5.00%)  1
Musculoskeletal and connective tissue disorders         
Limb discomfort  1  0/21 (0.00%)  0 0/21 (0.00%)  0 0/19 (0.00%)  0 1/20 (5.00%)  1
Musculoskeletal stiffness  1  0/21 (0.00%)  0 0/21 (0.00%)  0 0/19 (0.00%)  0 1/20 (5.00%)  1
Muscle spasms  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Nervous system disorders         
Headache  1  1/21 (4.76%)  2 1/21 (4.76%)  1 3/19 (15.79%)  4 2/20 (10.00%)  2
Dizziness  1  1/21 (4.76%)  1 0/21 (0.00%)  0 2/19 (10.53%)  2 0/20 (0.00%)  0
Crying  1  0/21 (0.00%)  0 1/21 (4.76%)  1 0/19 (0.00%)  0 1/20 (5.00%)  1
Psychomotor hyperactivity  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 1/20 (5.00%)  1
Psychiatric disorders         
Insomnia  1  1/21 (4.76%)  1 1/21 (4.76%)  1 1/19 (5.26%)  1 0/20 (0.00%)  0
Anxiety  1  0/21 (0.00%)  0 2/21 (9.52%)  2 0/19 (0.00%)  0 0/20 (0.00%)  0
Emotional Disorder  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Renal and urinary disorders         
Pollakiuria  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Respiratory, thoracic and mediastinal disorders         
Cough  1  1/21 (4.76%)  1 4/21 (19.05%)  5 4/19 (21.05%)  4 0/20 (0.00%)  0
Oropharyngeal pain  1  1/21 (4.76%)  1 3/21 (14.29%)  3 2/19 (10.53%)  2 2/20 (10.00%)  3
Nasal congestion  1  1/21 (4.76%)  1 1/21 (4.76%)  1 0/19 (0.00%)  0 3/20 (15.00%)  3
Asthma  1  1/21 (4.76%)  2 0/21 (0.00%)  0 1/19 (5.26%)  3 0/20 (0.00%)  0
Bronchial hyperreactivity  1  0/21 (0.00%)  0 0/21 (0.00%)  0 0/19 (0.00%)  0 1/20 (5.00%)  1
Epistaxis  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Rhinorrhoea  1  1/21 (4.76%)  1 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Skin and subcutaneous tissue disorders         
Rash  1  0/21 (0.00%)  0 3/21 (14.29%)  5 3/19 (15.79%)  4 1/20 (5.00%)  1
Cellulitis  1  0/21 (0.00%)  0 0/21 (0.00%)  0 0/19 (0.00%)  0 1/20 (5.00%)  1
Eczema  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Ingrowing nail  1  0/21 (0.00%)  0 0/21 (0.00%)  0 0/19 (0.00%)  0 1/20 (5.00%)  1
Pruritus  1  0/21 (0.00%)  0 0/21 (0.00%)  0 0/19 (0.00%)  0 1/20 (5.00%)  1
Vascular disorders         
Hot flush  1  0/21 (0.00%)  0 0/21 (0.00%)  0 1/19 (5.26%)  1 0/20 (0.00%)  0
Indicates events were collected by systematic assessment
1
Term from vocabulary, MedDRA (12.0)
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
Layout table for Results Point of Contact information
Name/Title: Study Director
Organization: Shire
Phone: +1 866 842 5335
EMail: ClinicalTransparency@shire.com
Layout table for additonal information
Responsible Party: Takeda ( Shire )
ClinicalTrials.gov Identifier: NCT00762073    
Other Study ID Numbers: MPI-101-01
First Submitted: September 29, 2008
First Posted: September 30, 2008
Results First Submitted: August 3, 2015
Results First Posted: October 5, 2015
Last Update Posted: June 11, 2021