Clinical trial NCT01642212: OBS in Adolescent and Adults With EOE: A Phase II, Randomized, Double-Blind, Placebo Controlled, Study With an Open Label Extension
Official title: Oral Budesonide Suspension (OBS) in Adolescent and Adult Subjects (11-40 Years of Age)With Eosinophilic Esophagitis: A Phase II, Randomized, Double-Blind, Placebo-Controlled Study With an Open Label Extension
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Eosinophilic Esophagitis (EoE)
Keywords: EoE
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2012-07-17
Enrollment: 93
Sex: ALL
Minimum age (years): 11
Maximum age (years): 40
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a clinical trial to test an experimental drug for the treatment of Eosinophilic Esophagitis (EoE)

Detailed description: Eosinophilic Esophagitis (EoE) is an inflammatory disorder of the esophagus and is a recognized clinical entity. Symptoms include feeding problems, heartburn, regurgitation, vomiting, abdominal pain and food impaction. The symptoms of EoE may be similar to gastroesophageal reflux disease (GERD) but do not resolve with gastric acid suppression. EoE is defined histologically as the presence of \> 15 intraepithelial eosinophils per high power fields on one or more esophageal biopsy specimens.

This Phase II study is comparing oral budesonide (OBS) to placebo to demonstrate that OBS induces a histologic response and a symptom response using a Dysphagia Symptom Questionnaire over a 16 week course of therapy.

Eligibility criteria: Inclusion Criteria:

* Males and Females, age 11-40
* Histologic evidence of EoE
* History of clinical symptoms of EoE including dysphagia
* Willing to continue with dietary, environmental or medical therapy
* Ability to read and understand english
* Written Consent

Exclusion Criteria:

* Any Medical condition that may compromise the safety of the subjects or interfere with the signs and symptoms of EoE
* Use of immunomodulatory therapy
* Current use of swallowed corticosteroids
* Esophageal strictures,varices or upper GI bleed
* Other current diseases of the GI tract
* Current viral infection or immunodeficiency condition
* Pregnancy
* Hypersensitivity to budesonide
* History of non compliance

Primary outcomes:
measure: Percent of Participants Who Were Histologic Responders
timeFrame: Week 16
measure: Change From Baseline in The Dysphagia Symptom Questionnaire (DSQ) Score at The Final Treatment Period Evaluation
timeFrame: Baseline, Week 16
Secondary outcomes:
measure: Change From Baseline in The DSQ Score Over Time
timeFrame: Baseline, Weeks 8 and 12
measure: Change From Baseline in The DSQ Score For The 50th Percentile of Participants at The Final Treatment Period Evaluation
timeFrame: Baseline, Week 16
measure: Percent of Participants With a Peak Eosinophil Count </= 15/High Power Field (Light Microscopy) (HPF) And </= 1/HPF at The Final Treatment Period Evaluation
timeFrame: Week 16
measure: Percent of Participants With a >/= 30% And >/= 50% Reduction In The DSQ Score From Baseline to The Final Treatment Period Evaluation
timeFrame: Baseline, Week 16
measure: Percent of Participants Who Were Overall Responders at The Final Treatment Period Evaluation
timeFrame: Week 16
measure: Change From Baseline in The Histopathologic Epithelial Features Combined Total Score at The Final Treatment Period Evaluation
timeFrame: Baseline, Week 16
measure: Change From Baseline in The Total Endoscopy Score at The Final Treatment Period Evaluation
timeFrame: Baseline, Week 16
measure: Change From Baseline in The Peak Eosinophil Count at Each Available Esophageal Level at The Final Treatment Period Evaluation
timeFrame: Baseline, Week 16
measure: Change From Baseline in The Physician's Global Assessment (PGA) of Disease Activity at The Final Treatment Period Evaluation
timeFrame: Baseline, Week 16
measure: Distribution of Responses For The Patient Global Impression of Change (PGIC) Survey at The Final Treatment Evaluation
timeFrame: Week 16
measure: Percent of Participants With Improved Symptoms on The Eosinophilic Esophagitis (EoE) Symptom Survey at The Final Treatment Period Evaluation
timeFrame: Week 16
measure: Percent of Participants With No Change in Symptoms on The EoE Symptom Survey at The Final Treatment Period Evaluation
timeFrame: Week 16
measure: Percent of Participants With Worsened Symptoms on The EoE Symptom Survey at The Final Treatment Period Evaluation
timeFrame: Week 16
measure: Percent of Participants Without Symptoms on The EoE Symptom Survey at The Final Treatment Period Evaluation
timeFrame: Week 16
measure: Percent of Participants With New Symptoms on The EoE Symptom Survey at The Final Treatment Period Evaluation
timeFrame: Week 16
measure: Percent of Participants Who Were Symptom Responders on The DSQ+Pain Scale at The Final Treatment Period Evaluation
timeFrame: Week 16
measure: Percent of Days That Participants Reported That They Avoided Solid Food During The Baseline And Treatment Periods
timeFrame: From 14 days prior to the baseline visit to the final treatment period evaluation
measure: Change From Baseline in The Scores of DSQ Question 1 During The Treatment Period
timeFrame: Baseline, Weeks 8, 12, and 16
measure: Change From Baseline in The Scores of DSQ Question 4 During The Treatment Period
timeFrame: Baseline, Weeks 8, 12, and 16
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Phoenix Children's Hospital
state: Arizona
city: Orange
country: United States
facility: Children's Hospital of Orange County
state: California
city: San Diego
country: United States
facility: UCSD Rady Children's Hospital
state: California
city: Aurora
country: United States
facility: The Children's Hospital
state: Colorado
city: Atlanta
country: United States
facility: Children's Center for Digestive Healthcare
state: Georgia
city: Chicago
country: United States
facility: Northwestern Scool of Medicine
state: Illinois
city: Chicago
country: United States
facility: Children's Memorial Hospital
state: Illinois
city: Park Ridge
country: United States
facility: Center for Children's Digestive Health
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University Health University Hospital
state: Indiana
city: Indianapolis
country: United States
facility: Riley Hospital for Children
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa Hospitals and Clinics
state: Iowa
city: Boston
country: United States
facility: Tufts Medical Center
state: Massachusetts
city: Boston
country: United States
facility: Children's Hospital Boston
state: Massachusetts
city: Rochester
country: United States
facility: The Mayo Clinic
state: Minnesota
city: Chapel Hill
country: United States
facility: University of North Carolina at Chapel Hill
state: North Carolina
city: Cincinnati
country: United States
facility: The Cincinnati Center for Eosinophilic Disorders
state: Ohio
city: Mentor
country: United States
facility: Great Lakes Gastroenterology
state: Ohio
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Providence
country: United States
facility: Rhode Island Hospital
state: Rhode Island
city: Greenville
country: United States
facility: Children's Center for Digestive Health
state: South Carolina
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Salt Lake City
country: United States
facility: University of Utah Healthcare
state: Utah
city: Roanoke
country: United States
facility: Carilion Pediatric Gastroenterology
state: Virginia
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References:
citation: Dellon ES, Collins MH, Katzka DA, Hudgens S, Lan L, Williams J, Vera-Llonch M, Hirano I. Improvements in Dysphagia and Pain With Swallowing in Patients With Eosinophilic Esophagitis Receiving Budesonide Oral Suspension. Clin Gastroenterol Hepatol. 2021 Apr;19(4):699-706.e4. doi: 10.1016/j.cgh.2020.03.060. Epub 2020 Apr 6.
citation: Hirano I, Dellon ES, Collins MH, Williams J, Lan L, Katzka DA. Clinical Features at Baseline Cannot Predict Symptom Response to Placebo in Patients With Eosinophilic Esophagitis. Clin Gastroenterol Hepatol. 2019 Sep;17(10):2126-2128.e1. doi: 10.1016/j.cgh.2018.11.045. Epub 2018 Nov 28.
citation: Dellon ES, Katzka DA, Collins MH, Gupta SK, Lan L, Williams J, Hirano I. Safety and Efficacy of Budesonide Oral Suspension Maintenance Therapy in Patients With Eosinophilic Esophagitis. Clin Gastroenterol Hepatol. 2019 Mar;17(4):666-673.e8. doi: 10.1016/j.cgh.2018.05.051. Epub 2018 Jun 12.
citation: Dellon ES, Katzka DA, Collins MH, Hamdani M, Gupta SK, Hirano I; MP-101-06 Investigators. Budesonide Oral Suspension Improves Symptomatic, Endoscopic, and Histologic Parameters Compared With Placebo in Patients With Eosinophilic Esophagitis. Gastroenterology. 2017 Mar;152(4):776-786.e5. doi: 10.1053/j.gastro.2016.11.021. Epub 2016 Nov 23.