Phase 2
N=82
Dose-Ranging Study of Oral Viscous Budesonide in Pediatrics With Eosinophilic Esophagitis
Eosinophilic Esophagitis (EoE)
Bottom Line
View on ClinicalTrials.gov: NCT00762073 ↗Enrolled (actual)
82
Serious AEs
1.2%
Results posted
Oct 2015
Primary outcome: Primary: Percent of Participants Who Responded to Therapy — 5.6; 11.8; 52.6; 47.1 percentage of participants — p=0.5282
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- budesonide (Drug); placebo (Drug)
- Age
- Pediatric, Adult · 2+ yrs
- Sex
- All
- Sponsor
- Shire
- Primary completion
- Apr 2010
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent of Participants Who Responded to Therapy |
5.6; 11.8; 52.6; 47.1 | 0.5282 |
| SECONDARY Percent of Participants With Histologic Response |
5.6; 23.5; 52.6; 94.1 | 0.1786 |
| SECONDARY Percent of Participants With Histologic Remission |
0.0; 11.8; 42.1; 76.5 | 0.4959 |
| SECONDARY Percent Change From Baseline in Peak Eosinophil Count |
7.93; -52.62; -44.02; -94.75 | 0.0108 sig |
| SECONDARY Change From Baseline in Endoscopy Score |
-0.6; -0.9; -1.3; -2.2 | 0.1095 |
| SECONDARY Percent of Participants With Clinical Response |
77.8; 64.7; 78.9; 52.9 | 0.3769 |
| SECONDARY Percent of Participants With Clinical Remission |
33.3; 17.6; 31.6; 17.6 | 0.3444 |
| SECONDARY Percent Change From Baseline in Eosinophilic Esophagitis (EoE) Clinical Symptom Score (CSS) |
-64.46; -60.83; -65.89; -47.21 | 0.6729 |
| SECONDARY Change From Baseline in Physician's Global Assessment Score of Disease Severity |
-38.9; -30.2; -39.3; -35.7 | 0.4197 |
| SECONDARY Maximum Plasma Concentration (Cmax) of Budesonide |
492.0; 195.0; 1019.5; 958.4 | — |
| SECONDARY Time to Maximum (Tmax) And Half Maximum (T1/2) Plasma Concentration of Budesonide |
3.288; 3.398; 3.472; 3.528; 0.68; 1.20 | — |
| SECONDARY Area Under The Plasma Concentration-Time Curve (AUC) of Budesonide From Time Zero to Time of The Last Measurable Concentration (AUC0-last) |
1139.5; 743.8; 3259.3; 3636.9 | — |
| SECONDARY Percent of Participants With Potential Corticosteroid-Related Treatment-Emergent Adverse Events (TEAEs) |
9.5; 9.5; 21.0; 15.0 | — |
| SECONDARY Mean Change in Blood Pressure (BP) at End of Treatment |
-1.5; 1.8; 3.5; 8.0; -3.1; 0.5 | — |
Summary
This is a randomized, placebo-controlled, parallel-arm, dose-ranging study in subjects with eosinophilic esophagitis, 2-18 years of age. Eligible subjects will be randomized into one of four treatment groups. The Treatment Period will be 12 weeks during which subjects will visit the clinic at study weeks 0 (Baseline Visit), 2, 4, 8 and 12 (Final Treatment Evaluation) for clinical symptom assessment and safety evaluation (including adverse events and vital signs). All study treatments (active drug and placebo) will be administered orally twice daily during the Treatment Period, once in the morning after breakfast and once in the evening at bedtime.
Eligibility Criteria
Inclusion Criteria
- Male and female subjects between the ages of 2-18 years, inclusive
- History of clinical symptoms of esophageal dysfunction intermittently or continuously
- Histologic evidence of EoE with a peak eosinophil count of greater than or equal to 20 eosinophils per HPF, from two or more levels of the esophagus, within six weeks prior to the Baseline Visit
- At the Baseline Visit, subjects must have symptoms with a total EoE Clinical Symptom Score of greater than or equal to 3
- Willingness and ability to continue the dietary therapy, environmental therapy, and/or medical regimens (including gastric acid suppression, if any) in effect at the Screening Visit
- Females of childbearing potential must have a negative serum pregnancy test (beta human chorionic gonadotropin) prior to randomization into the study and sexually active subjects must agree to continue acceptable birth control measures throughout the duration of the study
- Written informed consent (parent or legal guardian) and, as appropriate, subject assent
Exclusion Criteria
- Current use of immunomodulatory therapy (or anticipated use within 12 weeks following the Baseline Visit)
- Diagnosis of inflammatory bowel disease
- Chronic viral infection or immunodeficiency condition (current)
- Use of swallowed topical corticosteroids for EoE in the 1 month prior to the biopsy required for entrance to this study or at any time between the biopsy and the Baseline Visit
- Use of systemic (oral or parenteral) corticosteroid within 1 month prior to the biopsy required for entrance to this study or at any time between the biopsy and the Baseline Visit
- Morning plasma cortisol level below the lower limit of normal (per Central Laboratory reference range) at the Screening Visit
- Upper gastrointestinal bleeding within 1 month prior to the Screening Visit or between the Screening Visit and Baseline Visit
- Current use of anticoagulants
- Current disease of the gastrointestinal tract aside from the current EoE diagnosis
- Evidence of concurrent eosinophilic gastritis, enteritis, colitis, or proctitis
- Evidence of active infection with Helicobacter pylori
- Evidence of unstable asthma or changes in asthma or allergic rhinitis therapy within 1 month prior to the biopsy required for entrance to this study
- Any female who is pregnant, who is planning to become pregnant, or who is breast-feeding
- Current evidence or history of hypersensitivity or idiosyncratic reaction to budesonide or any other ingredients of the study medication
- Current evidence of oropharyngeal or esophageal candidiasis
- Receipt of an investigational drug within 30 days prior to the biopsy required for entrance to this study
- Any condition or abnormality that, in the opinion of the Principal Investigator, would compromise the safety of the subject or successful conduct of the study
Data sourced from ClinicalTrials.gov (NCT00762073). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.