Trial Outcomes & Findings for Milk Patch for Eosinophilic Esophagitis (NCT NCT02579876)
NCT ID: NCT02579876
Last Updated: 2019-07-10
Results Overview
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.
COMPLETED
PHASE2
20 participants
From baseline to month 11 (end of double blind phase)
2019-07-10
Participant Flow
Participant milestones
| Measure |
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.
|
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.
|
|---|---|---|
|
Overall Study
STARTED
|
15
|
5
|
|
Overall Study
COMPLETED
|
14
|
5
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Milk Patch for Eosinophilic Esophagitis
Baseline characteristics by cohort
| Measure |
Viaskin Milk 500 mcg
n=15 Participants
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.
|
Viaskin Placebo
n=5 Participants
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.
|
Total
n=20 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
15 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
20 Participants
n=206 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Age, Continuous
|
10.46 years
STANDARD_DEVIATION 2.99 • n=99 Participants
|
12.71 years
STANDARD_DEVIATION 2.03 • n=107 Participants
|
10.69 years
STANDARD_DEVIATION 3.64 • n=206 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
15 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
15 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
20 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
15 participants
n=99 Participants
|
5 participants
n=107 Participants
|
20 participants
n=206 Participants
|
|
Eosinophils (Eos) per high power field (HPF) on Milk
|
72.4 Eos/hpf
STANDARD_DEVIATION 29.52 • n=99 Participants
|
60.93 Eos/hpf
STANDARD_DEVIATION 49.39 • n=107 Participants
|
64 Eos/hpf
STANDARD_DEVIATION 44.5 • n=206 Participants
|
PRIMARY outcome
Timeframe: 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.
Outcome measures
| Measure |
Viaskin Milk 500 mcg
n=14 Participants
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.
|
Viaskin Placebo
n=5 Participants
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.
|
|---|---|---|
|
Change in Maximum Esophageal Eosinophil Count From Baseline to End of Double-blind Treatment. (Intent to Treat Population)
|
-10.8 Eosinophils (Eos)/high power field (HPF)
Standard Deviation 37.73
|
-24.2 Eosinophils (Eos)/high power field (HPF)
Standard Deviation 63
|
PRIMARY outcome
Timeframe: 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.
Outcome measures
| Measure |
Viaskin Milk 500 mcg
n=7 Participants
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.
|
Viaskin Placebo
n=2 Participants
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.
|
|---|---|---|
|
Change in Eosinophils/High Power Field at End of Double-blind (DB) (Per Protocol Patients)
|
-26.86 Eosinophils (Eos)/high power field
Standard Deviation 22.53
|
42.5 Eosinophils (Eos)/high power field
Standard Deviation 31.82
|
SECONDARY outcome
Timeframe: Total Symptom Score at End of DB Phase, Month 11Symptoms 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.
Outcome measures
| Measure |
Viaskin Milk 500 mcg
n=14 Participants
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.
|
Viaskin Placebo
n=5 Participants
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.
|
|---|---|---|
|
Eosinophilic Esophagitis Symptom Score (Intent to Treat Population)
|
1.07 units on a scale
Standard Deviation 1.49
|
1.40 units on a scale
Standard Deviation 1.14
|
SECONDARY outcome
Timeframe: At end of DB phase, at 11 monthsUpper 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
Outcome measures
| Measure |
Viaskin Milk 500 mcg
n=14 Participants
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.
|
Viaskin Placebo
n=5 Participants
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.
|
|---|---|---|
|
Esophageal Endoscopy Score (ITT)
|
1.93 score on a scale
Standard Deviation 1.58
|
1.60 score on a scale
Standard Deviation 1.67
|
SECONDARY outcome
Timeframe: End of DB Phase, at 11 monthsMaximum Eosinophils/HPF after milk reintroduction at the end of double bind phase
Outcome measures
| Measure |
Viaskin Milk 500 mcg
n=7 Participants
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.
|
Viaskin Placebo
n=2 Participants
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.
|
|---|---|---|
|
Eosinophils Per HPF at End of Double Bind Protocol (Per Protocol) Patients
|
25.57 Eos/hpf
Standard Deviation 31.19
|
95 Eos/hpf
Standard Deviation 63.64
|
SECONDARY outcome
Timeframe: Month 11, end of Double Blind PhaseMeasure 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
Outcome measures
| Measure |
Viaskin Milk 500 mcg
n=14 Participants
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.
|
Viaskin Placebo
n=5 Participants
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.
|
|---|---|---|
|
Pediatric Eosinophilic Esophagitis Symptom Score (ITT)
|
11.15 units on a scale
Standard Deviation 11.07
|
13.2 units on a scale
Standard Deviation 7.33
|
SECONDARY outcome
Timeframe: Month 11 (end of double blind phase)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
Outcome measures
| Measure |
Viaskin Milk 500 mcg
n=7 Participants
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.
|
Viaskin Placebo
n=2 Participants
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.
|
|---|---|---|
|
Endoscopy Score (Per Protocol Patients)
|
1.43 score on a scale
Standard Deviation 1.51
|
3 score on a scale
Standard Deviation 1.41
|
SECONDARY outcome
Timeframe: Month 11, End of Double Blind Placebo ControlPediatric 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.
Outcome measures
| Measure |
Viaskin Milk 500 mcg
n=7 Participants
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.
|
Viaskin Placebo
n=2 Participants
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.
|
|---|---|---|
|
Pediatric Eosinophil Esophagitis Symptom Score (PP Population)
|
12.2 score on a scale
Standard Deviation 8.57
|
17 score on a scale
Standard Deviation 1.41
|
Adverse Events
Viaskin Milk 500 mcg
Viaskin Placebo
Serious adverse events
| Measure |
Viaskin Milk 500 mcg
n=15 participants at risk
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.
|
Viaskin Placebo
n=5 participants at risk
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.
|
|---|---|---|
|
Respiratory, thoracic and mediastinal disorders
Vocal Cord Dysfunction
|
0.00%
0/15 • 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
|
20.0%
1/5 • Number of events 1 • 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
|
Other adverse events
| Measure |
Viaskin Milk 500 mcg
n=15 participants at risk
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.
|
Viaskin Placebo
n=5 participants at risk
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.
|
|---|---|---|
|
Gastrointestinal disorders
General GI Symptoms
|
66.7%
10/15 • Number of events 10 • 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
|
40.0%
2/5 • Number of events 2 • 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
|
|
Respiratory, thoracic and mediastinal disorders
Upper Respiratory Infections
|
46.7%
7/15 • Number of events 7 • 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
|
100.0%
5/5 • Number of events 5 • 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
|
|
General disorders
Administration Site Conditions
|
80.0%
12/15 • Number of events 12 • 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
|
100.0%
5/5 • Number of events 5 • 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
|
|
Ear and labyrinth disorders
Ear infections
|
6.7%
1/15 • Number of events 1 • 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
|
0.00%
0/5 • 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
|
|
Musculoskeletal and connective tissue disorders
Muscloskeletal
|
13.3%
2/15 • Number of events 2 • 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
|
20.0%
1/5 • Number of events 1 • 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
|
|
Skin and subcutaneous tissue disorders
Skin
|
6.7%
1/15 • Number of events 1 • 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
|
0.00%
0/5 • 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
|
|
Infections and infestations
Infections
|
53.3%
8/15 • Number of events 8 • 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
|
60.0%
3/5 • Number of events 3 • 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
|
Additional Information
Jonathan Spergel
The Children's Hospital of Philadelphia
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place