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

Shire

Status and phase: Completed; Phase 2

Conditions: Eosinophilic Esophagitis (EoE)

Treatments: Drug: placebo; Drug: budesonide

Study type: Interventional

Funder types: Industry

Identifiers

NCT00762073

MPI-101-01

Details and patient eligibility

About

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.

Enrollment: 82 patients

Sex: All

Ages: 2 to 18 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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82 participants in 4 patient groups, including a placebo group

1

Placebo Comparator group

Treatment:

Drug: placebo

2

Experimental group

Description:

Low Dose Group

Treatment:

Drug: budesonide

3

Experimental group

Description:

Medium Dose Group

Treatment:

Drug: budesonide

4

Experimental group

Description:

High Dose Group

Treatment:

Drug: budesonide