Milk Patch for Eosinophilic Esophagitis (SMILEE)
|Eosinophilic Esophagitis Milk Allergy||Drug: Viaskin Milk 500 mcg Drug: Viaskin Placebo||Phase 2|
|Study Design:||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||A Double, Blind Placebo-controlled, Randomized Trial to Study Efficacy and Safety of the Viaskin Milk for Treating Milk Induced Eosinophilic Esophagitis in Children|
- Change in maximum esophageal eosinophil count from baseline to end of double-blind treatment. [ Time Frame: From baseline to month 11 ]Esophageal biopsy samples will be obtained prior to randomization and after completion of treatment. Intraepithelial eosinophils will be counted in all high powered fields (HPFs) using light microscopy. A HPF will be counted only if at least half of the field is occupied by tissue. The maximum eosinophil count per HPF will be reported for each esophageal biopsy site (at each of 2 levels).
- Change in Eosinophilic Esophagitis symptom score between baseline and end of each treatment period. [ Time Frame: From baseline to end of treatment at month 11 and month 22 ]Symptoms of Eosinophilic Esophagitis range from abdominal pain, gastroesophageal reflux, vomiting, and difficult swallowing. The symptoms will range from none to very severe as in Spergel54. The symptoms will be in 3 categories and individual symptoms and total symptoms scores will be collected on a range of 0-4 (0-none, 1-mild, 2-moderate, 3-severe and 4-very severe).
- Change in Esophageal Endoscopy Score between baseline and end of each treatment period. [ Time Frame: From baseline to end of treatment at month 11 and month 22 ]Upper endoscopies with biopsies (2 each of proximal and distal, plus any inflamed areas) will be completed before and after each treatment period. Each endoscopy will be scored using a validated standardized measure which examines 4 major esophageal features (rings, furrows, exudates and edema) and the presence of minor features of narrow caliber esophagus, feline esophagus, stricture and crepe paper esophagus. Each feature is graded: 0-none, 1 mild, 2-moderate, 3-severe.
- Change in the Eosinophilic Esophagitis Quality of Life (QOL) Score between baseline and end of each treatment period. [ Time Frame: From baseline to end of treatment at month 11 and month 22 ]Quality of life is a measurement of a subject's overall well-being. QOL will be measured by the validated age specific PedsQL Eosinophilic Esophagitis tool. The 33-item questionnaire includes for self-rating of symptoms (chest/throat/stomach pain and nausea/vomiting, trouble swallowing), Treatment, Worry (about treatment and disease), Communication with others, Food and Eating (allergies and limitations), Food Feelings (emotions associated with food allergies). The format is a Likert response scale, with higher scores indicating better HRQOL and lower EoE symptoms and problems.
- Time to development symptoms after milk reintroduction (month 9) until the end of each treatment period. [ Time Frame: At Month 9 and Month 20 ]Milk will be reintroduced to all subjects 9 months after the second endoscopy in a similar amount and duration as done during the screening period while the subject is on a proton pump inhibitor. A daily symptom diary will be completed by subjects to record dietary information, symptoms and medications or treatment taken for symptoms.
- Change in T cell count between baseline and end of each treatment period. [ Time Frame: From baseline to end of treatment at month 11 and month 22 ]
- Change in IgE levels specific to milk between baseline and end of each treatment period. [ Time Frame: From baseline to end of treatment at month 11 and month 22 ]
- Change in IgG4 levels specific to milk between baseline and end of each treatment period. [ Time Frame: From baseline to end of treatment at month 11 and month 22 ]
|Study Start Date:||October 2015|
|Estimated Study Completion Date:||April 2019|
|Estimated Primary Completion Date:||December 2018 (Final data collection date for primary outcome measure)|
Active Comparator: Viaskin Milk 500 mcg
Viaskin patch containing milk protein. The patch is applied to the skin
Drug: Viaskin Milk 500 mcg
Biological: Viaskin Milk 500 mcg Subjects epicutaneously administered daily (up to 24 hours application per day) with a patch containing 500 mcg cow's milk proteins.
Placebo Comparator: Viaskin Placebo
Viaksin patch without any milk protein.
Drug: Viaskin Placebo
Biological: Viaskin Placebo Subjects epicutaneously administered daily (up to 24 hours application per day) with a patch containing a matching placebo formulation.
This is a double-blind, placebo-controlled, randomized trial to study the efficacy and safety of Viaskin® Milk, an allergen extract of milk administered epicutaneously using the Viaskin® epicutaneous delivery system (EPIT) in subjects from 4 to 17 years old with a milk induced Eosinophilic Esophagitis. Subjects will be randomized in a 3:1 ratio into two different treatment groups, to receive EPIT with Viaskin® Milk (500 µg of milk proteins) or placebo.
Subjects who complete the double-blind treatment period (approximately 11 months), will automatically rollover into an open label treatment period (additional 11 months). All subjects will receive the 500 µg (micrograms) Viaskin Milk patch.
Please refer to this study by its ClinicalTrials.gov identifier: NCT02579876
|United States, Pennsylvania|
|The Children's Hospital of Philadelphia|
|Philadelphia, Pennsylvania, United States, 19104|
|Principal Investigator:||Antonella Cianferoni, MD, PhD||Children's Hospital of Philadelphia|