A Double, Blind Placebo-controlled, Randomized Trial to Study Efficacy and Safety of the Viaskin Milk for Treating Milk Induced Eosinophilic Esophagitis in Children
Overview
- Phase
- Phase 2
- Intervention
- Viaskin Milk 500 mcg
- Conditions
- Eosinophilic Esophagitis
- Sponsor
- Children's Hospital of Philadelphia
- Enrollment
- 20
- Locations
- 1
- Primary Endpoint
- Change in Maximum Esophageal Eosinophil Count From Baseline to End of Double-blind Treatment. (Intent to Treat Population)
- Status
- Completed
- Last Updated
- 6 years ago
Overview
Brief Summary
This is a single-site, double-blind, placebo-controlled, randomized trial to study efficacy and safety of the Viaskin® Milk Patch for children with milk induced Eosinophilic Esophagitis (EoE). 20 subjects will be randomized 3:1 to Viaskin® Milk or placebo patch.
Detailed Description
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.
Investigators
Antonella Cianferoni
Assistant Professor
Children's Hospital of Philadelphia
Eligibility Criteria
Inclusion Criteria
- •Well-documented medical history of Eosinophilic Esophagitis after ingestion of milk and currently following a strict milk-free diet.
- •Upper endoscopy and biopsy at initial clinical evaluation during Visit 2 showing greater than 15 eosinophils/high power field (HPF) isolated to the esophagus meeting the consensus diagnosis of Eosinophilic Esophagitis, after milk was re-introduced into the subject's diet (30 ml/day for 1 week to 2 months), while the subject was on proton pump inhibitor.
- •Upper endoscopy and biopsy at second clinical evaluation during Visit 3 showing less than 5 eosinophils per HPF isolated to the esophagus after a minimum of 6 weeks under milk-free diet, and while the subject is on proton pump inhibitor.
- •Negative pregnancy test for female subjects of childbearing potential. Females of childbearing potential must use effective method of contraception to prevent pregnancy and agree to continue to practice an acceptable method of contraception for the duration of participation in the study. Sexual abstinence will be accepted as an effective method of contraception for girls below 18 years of age.
- •Subjects and/or parents/guardians willing to comply with all study requirements during their participation in the study.
- •Signed informed consent from parent(s)/guardian(s) of children \< 18 years + children's assent.
- •Subjects agree to maintain a constant diet during the trial, with the exception of milk.
- •Subjects will maintain constant medications for asthma and allergic rhinitis during the trial.
Exclusion Criteria
- •Subjects with a history of severe anaphylaxis to milk with the following symptoms: hypotension, hypoxia, neurological compromise (collapse, loss of consciousness or incontinence), Quincke Edema or requiring intubation.
- •Active IgE- mediated milk allergy.
- •Pregnancy or lactation.
- •Subjects with other eosinophilic gastrointestinal disorders.
- •Subjects on swallowed corticosteroids or anti-leukotrienes for Eosinophilic Esophagitis.
- •Subjects with symptomatic allergy to pollens whose symptoms during the corresponding pollen season might interfere with the recording of symptoms during the upper endoscopy/biopsy, if the upper endoscopy/biopsy is conducted during the pollen season. The Investigator will have to ensure that the period for conducting the upper endoscopy for such a subject will be outside of the pollen season.
- •Subjects treated with systemic long-acting corticosteroids (depot corticosteroids) within 12 weeks prior to Visit 1 and/or systemic short-acting corticosteroid within 4 weeks prior to Visit 1 or any systemic corticosteroid at screening.
- •Subjects with asthma conditions defined as follows:
- •Uncontrolled persistent asthma by National Asthma Education and Prevention Program Asthma guidelines (2007).
- •at least two systemic corticosteroid courses for asthma in the past year or one oral corticosteroid course for asthma in the past three months;
Arms & Interventions
Viaskin Milk 500 mcg
Viaskin patch containing milk protein. The patch is applied to the skin
Intervention: Viaskin Milk 500 mcg
Viaskin Placebo
Viaksin patch without any milk protein.
Intervention: Viaskin Placebo
Outcomes
Primary Outcomes
Change in Maximum Esophageal Eosinophil Count From Baseline to End of Double-blind Treatment. (Intent to Treat Population)
Time Frame: From baseline to month 11 (end of double blind phase)
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). The maximum eosinophil count for each patient will be calculated from either level. For the final outcome, mean from each individual patients maximum eosinophil count/hpf along with standard deviation will be calculated.
Change in Eosinophils/High Power Field at End of Double-blind (DB) (Per Protocol Patients)
Time Frame: Month 11(end of double blind phase)
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). The maximum eosinophil count for each patient will be calculated from either level. For the final outcome, mean from each individual patients maximum eosinophil count/hpf along with standard deviation will be calculated.
Secondary Outcomes
- Esophageal Endoscopy Score (ITT)(At end of DB phase, at 11 months)
- Eosinophils Per HPF at End of Double Bind Protocol (Per Protocol) Patients(End of DB Phase, at 11 months)
- Pediatric Eosinophil Esophagitis Symptom Score (PP Population)(Month 11, End of Double Blind Placebo Control)
- Eosinophilic Esophagitis Symptom Score (Intent to Treat Population)(Total Symptom Score at End of DB Phase, Month 11)
- Pediatric Eosinophilic Esophagitis Symptom Score (ITT)(Month 11, end of Double Blind Phase)
- Endoscopy Score (Per Protocol Patients)(Month 11 (end of double blind phase))