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| Name | Class |
|---|---|
| DBV Technologies | INDUSTRY |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Viaskin Milk 500 mcg | Active Comparator | Viaskin patch containing milk protein. The patch is applied to the skin |
|
| Viaskin Placebo | Placebo Comparator | Viaksin patch without any milk protein. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Viaskin Milk 500 mcg | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Maximum Esophageal Eosinophil Count From Baseline to End of Double-blind Treatment. (Intent to Treat Population) | 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. | From baseline to month 11 (end of double blind phase) |
| Change in Eosinophils/High Power Field at End of Double-blind (DB) (Per Protocol Patients) | 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. | Month 11(end of double blind phase) |
| Measure | Description | Time Frame |
|---|---|---|
| Eosinophilic Esophagitis Symptom Score (Intent to Treat Population) | Symptoms of Eosinophilic Esophagitis range from abdominal pain, gastroesophageal reflux, vomiting, and difficult swallowing. Investigator assessment of the subject's symptoms was completed on a 4-point Likert scale for 3 separate symptoms (vomiting, abdominal pain and dysphagia). Investigator assessment of the subject's symptoms was completed on a 4-point Likert scale for 3 separate symptoms (vomiting, abdominal pain and dysphagia). (0-none, 1-mild, 2-moderate, 3-severe, ). Total score is reported with a range of 0 to 9. A lower score is better. |
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Inclusion Criteria:
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:
Subjects on β-blocking agents, angiotensin-converting enzyme inhibitors, angiotensin receptor blockers, calcium channel blockers or tricyclic antidepressant therapy.
Subjects undergoing any type of immunotherapy to any food within one year prior to Visit 1.
Subjects presently on aeroallergen immunotherapy and unwilling or unable to discontinue.
Subjects currently treated with anti-tumor necrosis factor drugs or anti-Immunoglobulin E (IgE) drugs or any biologic immunomodulatory therapy within one year prior to Visit 1.
Allergy or known hypersensitivity to placebo excipients either of the Viaskin® or Tegaderm®.
Subjects suffering from generalized dermatologic diseases with no intact skin zones to apply the Viaskin®, or urticarial and mast cell disorders such as chronic idiopathic urticaria.
Subjects (or parents of subjects) with obvious excessive anxiety and unlikely to cope with the conditions of an upper endoscopy and biopsy.
Past or current disease(s), which in the opinion the sponsor-investigator, may affect the subject's participation in this study including but not limited to active autoimmune disorders, immunodeficiency, malignancy, uncontrolled diseases (hypertension, psychiatric (especially anxiety), cardiac), or other disorders (e.g., liver, gastrointestinal, kidney, cardiovascular, pulmonary disease, or blood disorders).
Any history of drug or alcohol abuse in the past five years.
Subjects unable to follow the protocol and the protocol requirements.
Participation in another clinical intervention study in the three months prior to Study Visit 1.
Subjects on any experimental drugs or treatments.
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| Name | Affiliation | Role |
|---|---|---|
| Antonella Cianferoni, MD, PhD | Children's Hospital of Philadelphia | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| The Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19104 | United States |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 31100455 | Derived | Spergel JM, Elci OU, Muir AB, Liacouras CA, Wilkins BJ, Burke D, Lewis MO, Brown-Whitehorn T, Cianferoni A. Efficacy of Epicutaneous Immunotherapy in Children With Milk-Induced Eosinophilic Esophagitis. Clin Gastroenterol Hepatol. 2020 Feb;18(2):328-336.e7. doi: 10.1016/j.cgh.2019.05.014. Epub 2019 May 14. |
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| ID | Title | Description |
|---|---|---|
| FG000 | Viaskin Milk 500 mcg | Viaskin patch containing milk protein. The patch is applied to the skin 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. |
| FG001 | Viaskin Placebo | Viaksin patch without any milk protein. Viaskin Placebo: Biological: Viaskin Placebo Subjects epicutaneously administered daily (up to 24 hours application per day) with a patch containing a matching placebo formulation. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | Viaskin Milk 500 mcg | Viaskin patch containing milk protein. The patch is applied to the skin 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. |
| BG001 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change in Maximum Esophageal Eosinophil Count From Baseline to End of Double-blind Treatment. (Intent to Treat Population) | 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. | Posted | Mean | Standard Deviation | Eosinophils (Eos)/high power field (HPF) | From baseline to month 11 (end of double blind phase) |
|
The length of time is 1 year from the start of the study to end of double blind phase
All adverse events (AEs) were collected from the start of the study. . All Treatment emergent AEs are reported
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Viaskin Milk 500 mcg | Viaskin patch containing milk protein. The patch is applied to the skin 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. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Vocal Cord Dysfunction | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| General GI Symptoms | Gastrointestinal disorders | MedDRA (12.0) | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Jonathan Spergel | The Children's Hospital of Philadelphia | 215 590 1000 | spergel@email.chop.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| SAP | No | Yes | No | Statistical Analysis Plan | Jan 5, 2018 | Jan 5, 2019 | SAP_000.pdf |
| Prot | Yes | No | No | Study Protocol | Aug 21, 2017 | Jan 5, 2019 | Prot_001.pdf |
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| ID | Term |
|---|---|
| D057765 | Eosinophilic Esophagitis |
| D016269 | Milk Hypersensitivity |
| ID | Term |
|---|---|
| D004941 | Esophagitis |
| D004935 | Esophageal Diseases |
| D005767 | Gastrointestinal Diseases |
| D004066 | Digestive System Diseases |
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|
| Viaskin Placebo | Drug | Biological: Viaskin Placebo Subjects epicutaneously administered daily (up to 24 hours application per day) with a patch containing a matching placebo formulation. |
|
|
| Total Symptom Score at End of DB Phase, Month 11 |
| Esophageal Endoscopy Score (ITT) | 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. The scores are summed including both minor and major criteria. Total score is presented and lower score is better. The range is from 0-12 | At end of DB phase, at 11 months |
| Eosinophils Per HPF at End of Double Bind Protocol (Per Protocol) Patients | Maximum Eosinophils/HPF after milk reintroduction at the end of double bind phase | End of DB Phase, at 11 months |
| Pediatric Eosinophilic Esophagitis Symptom Score (ITT) | Measure of Pediatric Eosinophilic Esophagitis symptom Score (PEESS) at the end of DB phase for the Intent to Treat Population The PEESS® is a 20 question survey asking patient symptom intensity and frequency on a 5 point scale (0 to 4) for each question for the preceding month. Therefore, the total score can range from 0 to 80. The total score is reported and lower score is better | Month 11, end of Double Blind Phase |
| Endoscopy Score (Per Protocol Patients) | 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. The scores including both major and minor criteria are summed. Total score is presented and lower score is better. The range is from 0-12 | Month 11 (end of double blind phase) |
| Pediatric Eosinophil Esophagitis Symptom Score (PP Population) | Pediatric Eosinophilic Esophagitis Symptom Score at end of DB phase using the validated Pediatric Eosinophilic Esophagitis Symptom Score (PEESS).The PEESS® is a 20 question survey asking patient symptom intensity and frequency on a 5 point scale (0 to 4) for each question for the preceding month. Therefore, the total score can range from 0 to 80. The total score is reported and lower score is better. | Month 11, End of Double Blind Placebo Control |
| Viaskin Placebo |
Viaksin patch without any milk protein. Viaskin Placebo: Biological: Viaskin Placebo Subjects epicutaneously administered daily (up to 24 hours application per day) with a patch containing a matching placebo formulation. |
| BG002 | Total | Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Eosinophils (Eos) per high power field (HPF) on Milk | Mean | Standard Deviation | Eos/hpf |
|
| OG001 | Viaskin Placebo | Viaksin patch without any milk protein. Viaskin Placebo: Biological: Viaskin Placebo Subjects epicutaneously administered daily (up to 24 hours application per day) with a patch containing a matching placebo formulation. |
|
|
| Primary | Change in Eosinophils/High Power Field at End of Double-blind (DB) (Per Protocol Patients) | 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. | Posted | Mean | Standard Deviation | Eosinophils (Eos)/high power field | Month 11(end of double blind phase) |
|
|
|
| Secondary | Eosinophilic Esophagitis Symptom Score (Intent to Treat Population) | Symptoms of Eosinophilic Esophagitis range from abdominal pain, gastroesophageal reflux, vomiting, and difficult swallowing. Investigator assessment of the subject's symptoms was completed on a 4-point Likert scale for 3 separate symptoms (vomiting, abdominal pain and dysphagia). Investigator assessment of the subject's symptoms was completed on a 4-point Likert scale for 3 separate symptoms (vomiting, abdominal pain and dysphagia). (0-none, 1-mild, 2-moderate, 3-severe, ). Total score is reported with a range of 0 to 9. A lower score is better. | Posted | Mean | Standard Deviation | units on a scale | Total Symptom Score at End of DB Phase, Month 11 |
|
|
|
| Secondary | Esophageal Endoscopy Score (ITT) | 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. The scores are summed including both minor and major criteria. Total score is presented and lower score is better. The range is from 0-12 | Posted | Mean | Standard Deviation | score on a scale | At end of DB phase, at 11 months |
|
|
|
| Secondary | Eosinophils Per HPF at End of Double Bind Protocol (Per Protocol) Patients | Maximum Eosinophils/HPF after milk reintroduction at the end of double bind phase | Posted | Mean | Standard Deviation | Eos/hpf | End of DB Phase, at 11 months |
|
|
|
| Secondary | Pediatric Eosinophilic Esophagitis Symptom Score (ITT) | Measure of Pediatric Eosinophilic Esophagitis symptom Score (PEESS) at the end of DB phase for the Intent to Treat Population The PEESS® is a 20 question survey asking patient symptom intensity and frequency on a 5 point scale (0 to 4) for each question for the preceding month. Therefore, the total score can range from 0 to 80. The total score is reported and lower score is better | Posted | Mean | Standard Deviation | units on a scale | Month 11, end of Double Blind Phase |
|
|
|
| Secondary | Endoscopy Score (Per Protocol Patients) | 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. The scores including both major and minor criteria are summed. Total score is presented and lower score is better. The range is from 0-12 | Posted | Mean | Standard Deviation | score on a scale | Month 11 (end of double blind phase) |
|
|
|
| Secondary | Pediatric Eosinophil Esophagitis Symptom Score (PP Population) | Pediatric Eosinophilic Esophagitis Symptom Score at end of DB phase using the validated Pediatric Eosinophilic Esophagitis Symptom Score (PEESS).The PEESS® is a 20 question survey asking patient symptom intensity and frequency on a 5 point scale (0 to 4) for each question for the preceding month. Therefore, the total score can range from 0 to 80. The total score is reported and lower score is better. | Posted | Mean | Standard Deviation | score on a scale | Month 11, End of Double Blind Placebo Control |
|
|
|
| 0 |
| 15 |
| 0 |
| 15 |
| 15 |
| 15 |
| EG001 | Viaskin Placebo | Viaksin patch without any milk protein. Viaskin Placebo: Biological: Viaskin Placebo Subjects epicutaneously administered daily (up to 24 hours application per day) with a patch containing a matching placebo formulation. | 0 | 5 | 1 | 5 | 5 | 5 |
| Upper Respiratory Infections | Respiratory, thoracic and mediastinal disorders | MedDRA (12.0) | Non-systematic Assessment |
|
| Administration Site Conditions | General disorders | MedDRA (12.0) | Systematic Assessment |
|
| Ear infections | Ear and labyrinth disorders | MedDRA (12.0) | Systematic Assessment |
|
| Muscloskeletal | Musculoskeletal and connective tissue disorders | MedDRA (12.0) | Systematic Assessment |
|
| Skin | Skin and subcutaneous tissue disorders | MedDRA (12.0) | Systematic Assessment |
|
| Infections | Infections and infestations | MedDRA (12.0) | Systematic Assessment |
|
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| D005759 |
| Gastroenteritis |
| D004802 | Eosinophilia |
| D007960 | Leukocyte Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
| D006969 | Hypersensitivity, Immediate |
| D006967 | Hypersensitivity |
| D007154 | Immune System Diseases |
| D005512 | Food Hypersensitivity |