Trial Outcomes & Findings for Effect of Dupilumab (Anti-IL4Rα) on the Host-Microbe Interface in Atopic Dermatitis (NCT NCT03389893)

NCT ID: NCT03389893

Last Updated: 2021-10-04

Results Overview

Staphylococcus aureus (S. aureus) abundance was measured by microbial DNA (femA qPCR) on lesional skin at Day 28, measurement is expressed in relative Colony Forming Units (rCFU)/cm\^2). The abundance of S. aureus is summarized as the geometric mean ratio (and corresponding 95% confidence interval) between the dupilumab and placebo arms. The geometric mean ratio is reported from an ANCOVA model with fixed effects for clinical site, disease severity at Day 0 (as measured by EASI \>21.1 \[severe\] or ≤21.1 \[non-severe\]) and S. aureus abundance at Day 0.

Recruitment status

TERMINATED

Study phase

PHASE4

Target enrollment

72 participants

Primary outcome timeframe

Day 28 (Post treatment initiation)

Results posted on

2021-10-04

Participant Flow

72 participants were randomized from July 2018 to March 2020 from 9 clinical sites.First participant enrollment occurred on July 25, 2018.

Informed consent was obtained from potentially eligible individuals who underwent a screening visit to determine eligibility.

Participant milestones

Participant milestones
Measure
Dupilumab
Participants received a loading dose of two 300 mg subcutaneous injections on Day 0 followed by 300 mg subcutaneous injections every two weeks (Days 14 and 28).
Placebo
Placebo contained the identical formulation as the dupilumab formulation without the active monoclonal antibody and was given by exactly the same route and schedule through Day 28.
Dupilumab to Open-Label Extension Dupilumab
Open Label Extension (OLE): Participants started a 10 week OLE on Day 42, beginning with a loading dose of two subcutaneously administered injections (one 300 mg dose of dupilumab and one dose of placebo, in order to protect prior masking/blind). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Placebo to Open-Label Extension Dupilumab
Open Label Extension (OLE): Participants started a 10 week OLE on Day 42, beginning with a loading dose of dupilumab (two 300 mg subcutaneous injections \[total of 600 mg\]-protection of prior masking/blind maintained). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Randomized Double Blind Period
STARTED
46
26
0
0
Randomized Double Blind Period
COMPLETED
45
26
0
0
Randomized Double Blind Period
NOT COMPLETED
1
0
0
0
Open Label Extension and Follow-Up
STARTED
0
0
45
26
Open Label Extension and Follow-Up
COMPLETED
0
0
41
25
Open Label Extension and Follow-Up
NOT COMPLETED
0
0
4
1

Reasons for withdrawal

Reasons for withdrawal
Measure
Dupilumab
Participants received a loading dose of two 300 mg subcutaneous injections on Day 0 followed by 300 mg subcutaneous injections every two weeks (Days 14 and 28).
Placebo
Placebo contained the identical formulation as the dupilumab formulation without the active monoclonal antibody and was given by exactly the same route and schedule through Day 28.
Dupilumab to Open-Label Extension Dupilumab
Open Label Extension (OLE): Participants started a 10 week OLE on Day 42, beginning with a loading dose of two subcutaneously administered injections (one 300 mg dose of dupilumab and one dose of placebo, in order to protect prior masking/blind). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Placebo to Open-Label Extension Dupilumab
Open Label Extension (OLE): Participants started a 10 week OLE on Day 42, beginning with a loading dose of dupilumab (two 300 mg subcutaneous injections \[total of 600 mg\]-protection of prior masking/blind maintained). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Randomized Double Blind Period
Withdrawal by Subject
1
0
0
0
Open Label Extension and Follow-Up
Lost to Follow-up
0
0
2
1
Open Label Extension and Follow-Up
Physician Decision
0
0
1
0
Open Label Extension and Follow-Up
Participant Moved
0
0
1
0

Baseline Characteristics

Effect of Dupilumab (Anti-IL4Rα) on the Host-Microbe Interface in Atopic Dermatitis

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
Dupilumab+Open Label Extension and Follow-Up
n=45 Participants
Participants received a loading dose of two 300 mg subcutaneous injections on Day 0 followed by 300 mg subcutaneous injections every two weeks (Days 14 and 28). Participants started a 10 week OLE on Day 42, beginning with a loading dose of two subcutaneously administered injections (one 300 mg dose of dupilumab and one dose of placebo, in order to protect prior masking/blind). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Placebo+Open Label Extension and Follow-Up
n=26 Participants
Placebo contained the identical formulation as the dupilumab formulation without the active monoclonal antibody and was given by exactly the same route and schedule through Day 28. Participants started a 10 week OLE on Day 42, beginning with a loading dose of dupilumab (two 300 mg subcutaneous injections \[total of 600 mg\]-protection of prior masking/blind maintained). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Total
n=71 Participants
Total of all reporting groups
Age, Continuous
36.6 years
STANDARD_DEVIATION 15.56 • n=5 Participants
37.3 years
STANDARD_DEVIATION 15.61 • n=7 Participants
36.8 years
STANDARD_DEVIATION 15.47 • n=5 Participants
Sex: Female, Male
Female
17 Participants
n=5 Participants
7 Participants
n=7 Participants
24 Participants
n=5 Participants
Sex: Female, Male
Male
28 Participants
n=5 Participants
19 Participants
n=7 Participants
47 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Hispanic or Latino
4 Participants
n=5 Participants
4 Participants
n=7 Participants
8 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Not Hispanic or Latino
39 Participants
n=5 Participants
22 Participants
n=7 Participants
61 Participants
n=5 Participants
Ethnicity (NIH/OMB)
Unknown or Not Reported
2 Participants
n=5 Participants
0 Participants
n=7 Participants
2 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 Participants
n=5 Participants
Race (NIH/OMB)
Asian
7 Participants
n=5 Participants
7 Participants
n=7 Participants
14 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
8 Participants
n=5 Participants
3 Participants
n=7 Participants
11 Participants
n=5 Participants
Race (NIH/OMB)
White
21 Participants
n=5 Participants
13 Participants
n=7 Participants
34 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
5 Participants
n=5 Participants
1 Participants
n=7 Participants
6 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
3 Participants
n=5 Participants
2 Participants
n=7 Participants
5 Participants
n=5 Participants
Region of Enrollment
United States
45 Participants
n=5 Participants
26 Participants
n=7 Participants
71 Participants
n=5 Participants
Body Weight
80.7 kilograms (Kg)
STANDARD_DEVIATION 19.45 • n=5 Participants
79.4 kilograms (Kg)
STANDARD_DEVIATION 18.58 • n=7 Participants
80.2 kilograms (Kg)
STANDARD_DEVIATION 19.01 • n=5 Participants
Height
170.9 centimeters (Cm)
STANDARD_DEVIATION 9.27 • n=5 Participants
175.3 centimeters (Cm)
STANDARD_DEVIATION 11.74 • n=7 Participants
172.5 centimeters (Cm)
STANDARD_DEVIATION 10.38 • n=5 Participants
Body Mass Index (BMI)
27.5 Kg/m^2
STANDARD_DEVIATION 5.69 • n=5 Participants
25.7 Kg/m^2
STANDARD_DEVIATION 4.90 • n=7 Participants
26.9 Kg/m^2
STANDARD_DEVIATION 5.45 • n=5 Participants
SCORing Atopic Dermatitis (SCORAD)
62.1 units on a scale
STANDARD_DEVIATION 11.7 • n=5 Participants
59.9 units on a scale
STANDARD_DEVIATION 10.86 • n=7 Participants
61.3 units on a scale
STANDARD_DEVIATION 11.38 • n=5 Participants
Eczema Area and Severity Index (EASI) Score
28.4 units on a scale
STANDARD_DEVIATION 12.16 • n=5 Participants
28.6 units on a scale
STANDARD_DEVIATION 10.89 • n=7 Participants
28.5 units on a scale
STANDARD_DEVIATION 11.63 • n=5 Participants
Eczema Area and Severity Index (EASI) Score < 21.1
16 Participants
n=5 Participants
10 Participants
n=7 Participants
26 Participants
n=5 Participants
Nottingham Eczema Severity Score
13.5 units on a scale
STANDARD_DEVIATION 1.49 • n=5 Participants
13.6 units on a scale
STANDARD_DEVIATION 1.21 • n=7 Participants
13.5 units on a scale
STANDARD_DEVIATION 1.38 • n=5 Participants
Investigator Global Assessment (IGA)
Moderate
16 Participants
n=5 Participants
11 Participants
n=7 Participants
27 Participants
n=5 Participants
Investigator Global Assessment (IGA)
Severe
29 Participants
n=5 Participants
15 Participants
n=7 Participants
44 Participants
n=5 Participants
Average itch in the past 24 hours as assessed by Pruritus Numerical Rating Scale (NRS)
6.7 units on a scale
STANDARD_DEVIATION 2.17 • n=5 Participants
7.0 units on a scale
STANDARD_DEVIATION 1.48 • n=7 Participants
6.8 units on a scale
STANDARD_DEVIATION 1.94 • n=5 Participants
Self-reported history of Eczema Herpeticum
1 Participants
n=5 Participants
1 Participants
n=7 Participants
2 Participants
n=5 Participants
Self-reported history of Staph infection
15 Participants
n=5 Participants
9 Participants
n=7 Participants
24 Participants
n=5 Participants
Self-reported history of food allergy
15 Participants
n=5 Participants
14 Participants
n=7 Participants
29 Participants
n=5 Participants
Self-reported history of animal allergies
30 Participants
n=5 Participants
19 Participants
n=7 Participants
49 Participants
n=5 Participants
Self-reported pets living in the participant's home
26 Participants
n=5 Participants
10 Participants
n=7 Participants
36 Participants
n=5 Participants
Self-reported smoking in the participant's home
11 Participants
n=5 Participants
3 Participants
n=7 Participants
14 Participants
n=5 Participants

PRIMARY outcome

Timeframe: Day 28 (Post treatment initiation)

Population: Modified intent-to-treat: All participants who were randomized and have Staphylococcus aureus abundance measured at Day 0 and Day 28.

Staphylococcus aureus (S. aureus) abundance was measured by microbial DNA (femA qPCR) on lesional skin at Day 28, measurement is expressed in relative Colony Forming Units (rCFU)/cm\^2). The abundance of S. aureus is summarized as the geometric mean ratio (and corresponding 95% confidence interval) between the dupilumab and placebo arms. The geometric mean ratio is reported from an ANCOVA model with fixed effects for clinical site, disease severity at Day 0 (as measured by EASI \>21.1 \[severe\] or ≤21.1 \[non-severe\]) and S. aureus abundance at Day 0.

Outcome measures

Outcome measures
Measure
Dupilumab+Open Label Extension and Follow-Up
n=45 Participants
Participants received a loading dose of two 300 mg subcutaneous injections on Day 0 followed by 300 mg subcutaneous injections every two weeks (Days 14 and 28). Participants started a 10 week OLE on Day 42, beginning with a loading dose of two subcutaneously administered injections (one 300 mg dose of dupilumab and one dose of placebo, in order to protect prior masking/blind). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Placebo+Open Label Extension and Follow-Up
n=26 Participants
Placebo contained the identical formulation as the dupilumab formulation without the active monoclonal antibody and was given by exactly the same route and schedule through Day 28. Participants started a 10 week OLE on Day 42, beginning with a loading dose of dupilumab (two 300 mg subcutaneous injections \[total of 600 mg\]-protection of prior masking/blind maintained). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Staphylococcus Aureus Abundance on Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
86.01 Relative Colony Forming Unit (rCFU)/cm^2
Interval 34.07 to 217.11
2608.4 Relative Colony Forming Unit (rCFU)/cm^2
Interval 835.02 to 8147.99

SECONDARY outcome

Timeframe: Day 0 (Prior to treatment), 3, 7, 14, 21, 42, 77 and 112

Population: Modified intent-to-treat: All participants who were randomized and have Staphylococcus aureus abundance measured at Day 0 and Day 28.

Staphylococcus aureus (S. aureus) abundance was measured by microbial DNA (femA qPCR) on lesional skin at Days 0, 3, 7, 14, 21, 42, 77 and 112. The abundance of S. aureus is summarized as the geometric mean ratio (GMR) and corresponding 95% confidence interval between the dupilumab and placebo arms. The GMR is reported from a linear mixed model for repeated measures with fixed effects for S. aureus abundance on lesional skin at Day 0, treatment arm, clinical site, disease severity at Day 0 as measured by EASI ≥ 21.1 or \< 21.1, time point of measurement (as a categorical variable), and an interaction term between treatment arm and pre-specified time point(s). A linear mixed model similar to the model specified above was fit for every time point measured. The model was used to estimate the GMR between treatment arms at Days 77 and 112 and the ratio within treatment arm between Day 77 and Day 42 and between Day 112 and Day 42.

Outcome measures

Outcome measures
Measure
Dupilumab+Open Label Extension and Follow-Up
n=45 Participants
Participants received a loading dose of two 300 mg subcutaneous injections on Day 0 followed by 300 mg subcutaneous injections every two weeks (Days 14 and 28). Participants started a 10 week OLE on Day 42, beginning with a loading dose of two subcutaneously administered injections (one 300 mg dose of dupilumab and one dose of placebo, in order to protect prior masking/blind). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Placebo+Open Label Extension and Follow-Up
n=26 Participants
Placebo contained the identical formulation as the dupilumab formulation without the active monoclonal antibody and was given by exactly the same route and schedule through Day 28. Participants started a 10 week OLE on Day 42, beginning with a loading dose of dupilumab (two 300 mg subcutaneous injections \[total of 600 mg\]-protection of prior masking/blind maintained). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Staphylococcus Aureus Abundance on Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 3
833.82 Relative Colony Forming Unit (rCFU)/cm^2
Interval 411.45 to 1689.74
3028.88 Relative Colony Forming Unit (rCFU)/cm^2
Interval 1256.63 to 7300.56
Staphylococcus Aureus Abundance on Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 7
337.48 Relative Colony Forming Unit (rCFU)/cm^2
Interval 138.69 to 821.16
900.59 Relative Colony Forming Unit (rCFU)/cm^2
Interval 290.95 to 2787.59
Staphylococcus Aureus Abundance on Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 14
205.06 Relative Colony Forming Unit (rCFU)/cm^2
Interval 91.16 to 461.27
2690.79 Relative Colony Forming Unit (rCFU)/cm^2
Interval 964.61 to 7505.96
Staphylococcus Aureus Abundance on Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 21
179.91 Relative Colony Forming Unit (rCFU)/cm^2
Interval 80.71 to 401.02
2468.64 Relative Colony Forming Unit (rCFU)/cm^2
Interval 916.89 to 6646.62
Staphylococcus Aureus Abundance on Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 42
177.79 Relative Colony Forming Unit (rCFU)/cm^2
Interval 79.02 to 400.05
1112.55 Relative Colony Forming Unit (rCFU)/cm^2
Interval 409.02 to 3026.14
Staphylococcus Aureus Abundance on Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 77
56.85 Relative Colony Forming Unit (rCFU)/cm^2
Interval 20.44 to 158.08
150.81 Relative Colony Forming Unit (rCFU)/cm^2
Interval 43.93 to 517.81
Staphylococcus Aureus Abundance on Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 112
42.35 Relative Colony Forming Unit (rCFU)/cm^2
Interval 14.91 to 120.28
174.19 Relative Colony Forming Unit (rCFU)/cm^2
Interval 53.18 to 570.53

SECONDARY outcome

Timeframe: Day 0 (Prior to treatment), 3, 7, 14, 21, 28, 42, 77 and 112

Population: Modified intent-to-treat: All participants who were randomized and have Staphylococcus aureus abundance measured at Day 0 and Day 28.

Staphylococcus aureus (S. aureus) abundance was measured by microbial DNA (femA qPCR) on non-lesional skin at Days 0, 3, 7, 14, 21, 28, 42, 77 and 112. The abundance of S. aureus is summarized as the geometric mean ratio (GMR) and corresponding 95% confidence interval between the dupilumab and placebo arms. The GMR is reported from a linear mixed model for repeated measures with fixed effects for S. aureus abundance on non-lesional skin at Day 0, treatment arm, clinical site, disease severity at Day 0 as measured by EASI ≥ 21.1 or \< 21.1, time point of measurement (as a categorical variable), and an interaction term between treatment arm and pre-specified time point(s). A linear mixed model similar to the model specified above was fit for every time point measured. The model estimated the GMR between treatment arms at Days 77 and 112 and the ratio within treatment arm between Day 77 and Day 42 and between Day 112 and Day 42.

Outcome measures

Outcome measures
Measure
Dupilumab+Open Label Extension and Follow-Up
n=45 Participants
Participants received a loading dose of two 300 mg subcutaneous injections on Day 0 followed by 300 mg subcutaneous injections every two weeks (Days 14 and 28). Participants started a 10 week OLE on Day 42, beginning with a loading dose of two subcutaneously administered injections (one 300 mg dose of dupilumab and one dose of placebo, in order to protect prior masking/blind). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Placebo+Open Label Extension and Follow-Up
n=26 Participants
Placebo contained the identical formulation as the dupilumab formulation without the active monoclonal antibody and was given by exactly the same route and schedule through Day 28. Participants started a 10 week OLE on Day 42, beginning with a loading dose of dupilumab (two 300 mg subcutaneous injections \[total of 600 mg\]-protection of prior masking/blind maintained). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Staphylococcus Aureus Abundance on Non-lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 3
221.75 Relative Colony Forming Unit (rCFU)/cm^2
Interval 99.74 to 493.01
277.21 Relative Colony Forming Unit (rCFU)/cm^2
Interval 100.25 to 766.52
Staphylococcus Aureus Abundance on Non-lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 7
143.19 Relative Colony Forming Unit (rCFU)/cm^2
Interval 66.56 to 308.04
539.97 Relative Colony Forming Unit (rCFU)/cm^2
Interval 201.61 to 1446.22
Staphylococcus Aureus Abundance on Non-lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 14
118.15 Relative Colony Forming Unit (rCFU)/cm^2
Interval 57.86 to 241.27
326.55 Relative Colony Forming Unit (rCFU)/cm^2
Interval 132.38 to 805.48
Staphylococcus Aureus Abundance on Non-lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 42
98.97 Relative Colony Forming Unit (rCFU)/cm^2
Interval 44.01 to 222.55
191.30 Relative Colony Forming Unit (rCFU)/cm^2
Interval 70.36 to 520.1
Staphylococcus Aureus Abundance on Non-lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 112
76.71 Relative Colony Forming Unit (rCFU)/cm^2
Interval 27.37 to 214.99
146.35 Relative Colony Forming Unit (rCFU)/cm^2
Interval 45.23 to 473.54
Staphylococcus Aureus Abundance on Non-lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 21
93.32 Relative Colony Forming Unit (rCFU)/cm^2
Interval 44.33 to 196.46
128.56 Relative Colony Forming Unit (rCFU)/cm^2
Interval 51.17 to 323.01
Staphylococcus Aureus Abundance on Non-lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 28
61.67 Relative Colony Forming Unit (rCFU)/cm^2
Interval 29.55 to 128.72
369.74 Relative Colony Forming Unit (rCFU)/cm^2
Interval 145.96 to 936.6
Staphylococcus Aureus Abundance on Non-lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 77
86.91 Relative Colony Forming Unit (rCFU)/cm^2
Interval 32.27 to 234.09
182.19 Relative Colony Forming Unit (rCFU)/cm^2
Interval 57.37 to 578.6

SECONDARY outcome

Timeframe: Day 0 (Prior to treatment), 3, 7, 14, 21, 28, 42, 77 and 112

Population: Modified intent-to-treat (all participants who were randomized and have Staphylococcus aureus abundance measured at Day 0 and Day 28) with evaluable basal TEWL at any post-baseline time point.

TEWL assessment is a noninvasive in vivo measurement of water loss across the stratum corneum that is used to characterize skin barrier function. Basal TEWL =baseline measure (prior to tape stripping). An increase in TEWL values shows damage to the skin barrier function. Basal TEWL was measured on non-lesional and lesional skin. Basal TEWL is summarized as the mean difference between the dupilumab and placebo arms. The mean difference is reported from two linear mixed models for repeated measures (lesional and non-lesional) with fixed effects for treatment arm, basal TEWL at Day 0, clinical site, and disease severity at Day 0 as measured by EASI ≥ 21.1 or \< 21.1 at Days 3, 7, 14, 21, 28, and 42. Two linear mixed models similar to the models specified above were fit for every time point measured. The model was used to estimate the mean difference between treatment arms at Days 77 and 112 and the ratio within treatment arm between Day 77 and Day 42 and between Day 112 and Day 42.

Outcome measures

Outcome measures
Measure
Dupilumab+Open Label Extension and Follow-Up
n=44 Participants
Participants received a loading dose of two 300 mg subcutaneous injections on Day 0 followed by 300 mg subcutaneous injections every two weeks (Days 14 and 28). Participants started a 10 week OLE on Day 42, beginning with a loading dose of two subcutaneously administered injections (one 300 mg dose of dupilumab and one dose of placebo, in order to protect prior masking/blind). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Placebo+Open Label Extension and Follow-Up
n=26 Participants
Placebo contained the identical formulation as the dupilumab formulation without the active monoclonal antibody and was given by exactly the same route and schedule through Day 28. Participants started a 10 week OLE on Day 42, beginning with a loading dose of dupilumab (two 300 mg subcutaneous injections \[total of 600 mg\]-protection of prior masking/blind maintained). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Transepidermal Water Loss (TEWL) of Non-lesional and Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 7 - Lesional
40.08 g/m^2/hour
Interval 33.79 to 46.37
41.15 g/m^2/hour
Interval 32.7 to 49.61
Transepidermal Water Loss (TEWL) of Non-lesional and Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 28 - Lesional
24.93 g/m^2/hour
Interval 19.97 to 29.89
32.03 g/m^2/hour
Interval 25.93 to 38.13
Transepidermal Water Loss (TEWL) of Non-lesional and Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 42 - Lesional
25.34 g/m^2/hour
Interval 20.58 to 30.1
33.33 g/m^2/hour
Interval 27.46 to 39.19
Transepidermal Water Loss (TEWL) of Non-lesional and Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 3 - Non-lesional
24.24 g/m^2/hour
Interval 20.87 to 27.6
23.55 g/m^2/hour
Interval 19.05 to 28.05
Transepidermal Water Loss (TEWL) of Non-lesional and Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 7 - Non-lesional
22.58 g/m^2/hour
Interval 19.6 to 25.57
20.23 g/m^2/hour
Interval 16.28 to 24.18
Transepidermal Water Loss (TEWL) of Non-lesional and Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 14 - Non-lesional
23.30 g/m^2/hour
Interval 19.9 to 26.71
23.20 g/m^2/hour
Interval 18.77 to 27.63
Transepidermal Water Loss (TEWL) of Non-lesional and Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 21 - Non-lesional
19.55 g/m^2/hour
Interval 16.54 to 22.57
22.86 g/m^2/hour
Interval 18.78 to 26.95
Transepidermal Water Loss (TEWL) of Non-lesional and Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 28 - Non-lesional
17.55 g/m^2/hour
Interval 15.08 to 20.03
19.31 g/m^2/hour
Interval 16.11 to 22.51
Transepidermal Water Loss (TEWL) of Non-lesional and Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 42 - Non-lesional
16.95 g/m^2/hour
Interval 14.5 to 19.4
19.12 g/m^2/hour
Interval 16.17 to 22.07
Transepidermal Water Loss (TEWL) of Non-lesional and Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 77 - Non-lesional
15.45 g/m^2/hour
Interval 12.6 to 18.29
17.55 g/m^2/hour
Interval 14.27 to 20.82
Transepidermal Water Loss (TEWL) of Non-lesional and Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 112 - Non-lesional
13.97 g/m^2/hour
Interval 11.55 to 16.39
15.86 g/m^2/hour
Interval 13.05 to 18.68
Transepidermal Water Loss (TEWL) of Non-lesional and Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 3 - Lesional
33.46 g/m^2/hour
Interval 28.52 to 38.39
42.99 g/m^2/hour
Interval 36.57 to 49.41
Transepidermal Water Loss (TEWL) of Non-lesional and Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 14 - Lesional
31.96 g/m^2/hour
Interval 27.19 to 36.74
31.49 g/m^2/hour
Interval 25.47 to 37.51
Transepidermal Water Loss (TEWL) of Non-lesional and Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 21 - Lesional
28.14 g/m^2/hour
Interval 23.25 to 33.02
34.11 g/m^2/hour
Interval 27.74 to 40.48
Transepidermal Water Loss (TEWL) of Non-lesional and Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 77 - Lesional
24.54 g/m^2/hour
Interval 18.94 to 30.13
25.58 g/m^2/hour
Interval 19.14 to 32.02
Transepidermal Water Loss (TEWL) of Non-lesional and Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 112 - Lesional
22.99 g/m^2/hour
Interval 16.79 to 29.18
22.82 g/m^2/hour
Interval 15.87 to 29.78

SECONDARY outcome

Timeframe: Day 0 (Prior to treatment), 7, 14, 21, 28, 42, 77 and 112

Population: Modified intent-to-treat (all participants who were randomized and have Staphylococcus aureus abundance measured at Day 0 and Day 28) with evaluable TEWL AUC at any post-baseline time point.

TEWL skin barrier assessment was assessed prior to tape stripping and repeated after 5, 10, and 15 tape strips. TEWL AUC was calculated using the trapezoidal rule and represents skin barrier integrity. An increase in TEWL values shows damage to the skin barrier function. TEWL AUC was measured on non-lesional skin at Days 0, 7, 14, 21, 28, 42, 77 and 112. The mean difference (between Groups) is reported from a linear mixed model for repeated measures with a random effect for participant and fixed effects for treatment arm, TEWL AUC at Day 0, clinical site, and disease severity at Day 0 as measured by EASI ≥ 21.1 or \< 21.1 at Days 7, 14, 21, 28, and 42. A linear mixed model similar to the model specified above was fit for every time point measured. The model was used to estimate the mean difference between treatment arms at Days 77 and 112 and the mean difference within treatment arm between Day 77 and Day 42 and between Day 112 and Day 42.

Outcome measures

Outcome measures
Measure
Dupilumab+Open Label Extension and Follow-Up
n=43 Participants
Participants received a loading dose of two 300 mg subcutaneous injections on Day 0 followed by 300 mg subcutaneous injections every two weeks (Days 14 and 28). Participants started a 10 week OLE on Day 42, beginning with a loading dose of two subcutaneously administered injections (one 300 mg dose of dupilumab and one dose of placebo, in order to protect prior masking/blind). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Placebo+Open Label Extension and Follow-Up
n=26 Participants
Placebo contained the identical formulation as the dupilumab formulation without the active monoclonal antibody and was given by exactly the same route and schedule through Day 28. Participants started a 10 week OLE on Day 42, beginning with a loading dose of dupilumab (two 300 mg subcutaneous injections \[total of 600 mg\]-protection of prior masking/blind maintained). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Transepidermal Water Loss (TEWL) Area Under the Curve (AUC) on Non-Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 7
527.79 g/m^2/hour
Interval 466.25 to 589.33
442.74 g/m^2/hour
Interval 363.86 to 521.62
Transepidermal Water Loss (TEWL) Area Under the Curve (AUC) on Non-Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 14
504.80 g/m^2/hour
Interval 426.5 to 583.09
526.70 g/m^2/hour
Interval 424.66 to 628.74
Transepidermal Water Loss (TEWL) Area Under the Curve (AUC) on Non-Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 28
390.92 g/m^2/hour
Interval 328.07 to 453.76
483.02 g/m^2/hour
Interval 405.23 to 560.81
Transepidermal Water Loss (TEWL) Area Under the Curve (AUC) on Non-Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 21
451.40 g/m^2/hour
Interval 385.72 to 517.07
533.89 g/m^2/hour
Interval 444.83 to 622.96
Transepidermal Water Loss (TEWL) Area Under the Curve (AUC) on Non-Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 42
397.22 g/m^2/hour
Interval 341.24 to 453.2
445.98 g/m^2/hour
Interval 378.96 to 513.01
Transepidermal Water Loss (TEWL) Area Under the Curve (AUC) on Non-Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 77
363.15 g/m^2/hour
Interval 298.76 to 427.53
417.52 g/m^2/hour
Interval 344.23 to 490.81
Transepidermal Water Loss (TEWL) Area Under the Curve (AUC) on Non-Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 112
320.95 g/m^2/hour
Interval 266.16 to 375.74
343.92 g/m^2/hour
Interval 284.63 to 403.2

SECONDARY outcome

Timeframe: Day 0 (Prior to treatment), 7, 14, 21, 28, 42, 77 and 112

Population: Modified intent-to-treat (all participants who were randomized and have Staphylococcus aureus abundance measured at Day 0 and Day 28) with evaluable TEWL at any post-baseline time point.

The skin tape strip collection was comprised of 1 set of 15 strips from non-lesional skin and was collected as part of the TEWL skin barrier assessment. The TEWL values measured at every 5 tape strips were used to model the TEWL slope. TEWL slope assesses skin barrier integrity. An increase in TEWL values shows damage to the skin barrier function. TEWL slope is summarized as the mean difference (and corresponding 95% confidence interval) between the dupilumab and placebo arms. The mean difference in slope for each Day is reported from a linear mixed model for repeated measures with a fixed effects for basal TEWL prior to tape stripping, treatment arm, clinical site, disease severity at Day 0 as measured by EASI ≥ 21.1 or \< 21.1, tape strip number, and an interaction term between treatment arm and tape strip number.

Outcome measures

Outcome measures
Measure
Dupilumab+Open Label Extension and Follow-Up
n=43 Participants
Participants received a loading dose of two 300 mg subcutaneous injections on Day 0 followed by 300 mg subcutaneous injections every two weeks (Days 14 and 28). Participants started a 10 week OLE on Day 42, beginning with a loading dose of two subcutaneously administered injections (one 300 mg dose of dupilumab and one dose of placebo, in order to protect prior masking/blind). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Placebo+Open Label Extension and Follow-Up
n=26 Participants
Placebo contained the identical formulation as the dupilumab formulation without the active monoclonal antibody and was given by exactly the same route and schedule through Day 28. Participants started a 10 week OLE on Day 42, beginning with a loading dose of dupilumab (two 300 mg subcutaneous injections \[total of 600 mg\]-protection of prior masking/blind maintained). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Transepidermal Water Loss (TEWL) Slope on Non-Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 21
1.43 g/m^2/hour
Interval 0.95 to 1.91
1.98 g/m^2/hour
Interval 1.37 to 2.6
Transepidermal Water Loss (TEWL) Slope on Non-Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 7
1.77 g/m^2/hour
Interval 1.25 to 2.29
1.84 g/m^2/hour
Interval 1.18 to 2.51
Transepidermal Water Loss (TEWL) Slope on Non-Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 14
1.56 g/m^2/hour
Interval 1.17 to 1.96
1.78 g/m^2/hour
Interval 1.28 to 2.28
Transepidermal Water Loss (TEWL) Slope on Non-Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 28
1.38 g/m^2/hour
Interval 0.97 to 1.79
1.73 g/m^2/hour
Interval 1.23 to 2.23
Transepidermal Water Loss (TEWL) Slope on Non-Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 42
1.47 g/m^2/hour
Interval 0.96 to 1.97
1.76 g/m^2/hour
Interval 1.15 to 2.37
Transepidermal Water Loss (TEWL) Slope on Non-Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 77
1.15 g/m^2/hour
Interval 0.79 to 1.52
1.42 g/m^2/hour
Interval 0.99 to 1.85
Transepidermal Water Loss (TEWL) Slope on Non-Lesional Skin, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 112
1.03 g/m^2/hour
Interval 0.7 to 1.36
1.30 g/m^2/hour
Interval 0.94 to 1.67

SECONDARY outcome

Timeframe: Day 0 (Prior to treatment), 3, 7, 14, 21, 28, 42, 77 and 112

Population: Modified intent-to-treat: All participants who were randomized and have Staphylococcus aureus abundance measured at Day 0 and Day 28.

EASI is a composite score (range: 0-72) measuring physical signs of atopic dermatitis, including area of involvement and severity. Severity components include: erythema, papulation, excoriation and lichenification \[0=absent, 1=mild, 2=moderate, 3=severe\] for each body region (head/neck, trunk, arms, legs). Area of involvement (%) is assessed for each body region. Area and severity of each body region is weighted based on size of region, and region scores are added for the total score. Scores ≤7 are considered mild, \>7 and ≤21 are considered moderate, and \>21 are considered severe. The mean EASI score in each treatment arm is reported from a linear mixed model for repeated measures with fixed effects for EASI at Day 0, treatment arm, clinical site, time point of measurement, and an interaction term between treatment arm and pre-specified time point(s), Additionally, a similar model was fit using all time points.

Outcome measures

Outcome measures
Measure
Dupilumab+Open Label Extension and Follow-Up
n=45 Participants
Participants received a loading dose of two 300 mg subcutaneous injections on Day 0 followed by 300 mg subcutaneous injections every two weeks (Days 14 and 28). Participants started a 10 week OLE on Day 42, beginning with a loading dose of two subcutaneously administered injections (one 300 mg dose of dupilumab and one dose of placebo, in order to protect prior masking/blind). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Placebo+Open Label Extension and Follow-Up
n=26 Participants
Placebo contained the identical formulation as the dupilumab formulation without the active monoclonal antibody and was given by exactly the same route and schedule through Day 28. Participants started a 10 week OLE on Day 42, beginning with a loading dose of dupilumab (two 300 mg subcutaneous injections \[total of 600 mg\]-protection of prior masking/blind maintained). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Eczema Area and Severity Index (EASI) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 7
25.96 units on a scale
Interval 23.56 to 28.36
24.92 units on a scale
Interval 21.92 to 27.91
Eczema Area and Severity Index (EASI) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 3
26.52 units on a scale
Interval 24.21 to 28.83
24.60 units on a scale
Interval 21.73 to 27.46
Eczema Area and Severity Index (EASI) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 14
20.36 units on a scale
Interval 17.92 to 22.81
23.13 units on a scale
Interval 20.08 to 26.19
Eczema Area and Severity Index (EASI) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 21
16.57 units on a scale
Interval 13.98 to 19.16
22.75 units on a scale
Interval 19.51 to 25.98
Eczema Area and Severity Index (EASI) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 28
15.09 units on a scale
Interval 12.47 to 17.72
21.67 units on a scale
Interval 18.36 to 24.99
Eczema Area and Severity Index (EASI) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 42
11.90 units on a scale
Interval 9.1 to 14.7
20.40 units on a scale
Interval 16.83 to 23.96
Eczema Area and Severity Index (EASI) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 77
8.84 units on a scale
Interval 6.67 to 11.0
7.54 units on a scale
Interval 4.89 to 10.19
Eczema Area and Severity Index (EASI) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 112
6.97 units on a scale
Interval 5.19 to 8.76
5.53 units on a scale
Interval 3.45 to 7.61

SECONDARY outcome

Timeframe: Day 0 (Prior to treatment), 3, 7, 14, 21, 28, 42, 77 and 112

Population: Modified intent-to-treat: All participants who were randomized and have Staphylococcus aureus abundance measured at Day 0 and Day 28.

Investigator Global Assessment (IGA) score is a subjective scale measuring disease severity. Based on a 5-point scale from 0 (completely clear) to 4 (severe). Defined score of 0 = clear, 1 = almost clear, 2 = mild, 3 = moderate, 4 = severe. The mean IGA score in each treatment arm is reported from a linear mixed model for repeated measures with fixed effects for IGA at Day 0, treatment arm, clinical site, disease severity at Day 0 as measured by EASI ≥ 21.1 or \< 21.1, time point of measurement, and an interaction term between treatment arm and pre-specified time point(s). Additionally, a similar model was fit using all time points.

Outcome measures

Outcome measures
Measure
Dupilumab+Open Label Extension and Follow-Up
n=45 Participants
Participants received a loading dose of two 300 mg subcutaneous injections on Day 0 followed by 300 mg subcutaneous injections every two weeks (Days 14 and 28). Participants started a 10 week OLE on Day 42, beginning with a loading dose of two subcutaneously administered injections (one 300 mg dose of dupilumab and one dose of placebo, in order to protect prior masking/blind). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Placebo+Open Label Extension and Follow-Up
n=26 Participants
Placebo contained the identical formulation as the dupilumab formulation without the active monoclonal antibody and was given by exactly the same route and schedule through Day 28. Participants started a 10 week OLE on Day 42, beginning with a loading dose of dupilumab (two 300 mg subcutaneous injections \[total of 600 mg\]-protection of prior masking/blind maintained). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Investigator Global Assessment (IGA) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 7
3.32 units on a scale
Interval 3.18 to 3.45
3.28 units on a scale
Interval 3.11 to 3.45
Investigator Global Assessment (IGA) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 28
2.72 units on a scale
Interval 2.52 to 2.92
3.28 units on a scale
Interval 3.02 to 3.54
Investigator Global Assessment (IGA) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 3
3.49 units on a scale
Interval 3.34 to 3.64
3.28 units on a scale
Interval 3.09 to 3.47
Investigator Global Assessment (IGA) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 14
3.12 units on a scale
Interval 2.96 to 3.28
3.16 units on a scale
Interval 2.96 to 3.37
Investigator Global Assessment (IGA) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 21
2.86 units on a scale
Interval 2.67 to 3.05
3.20 units on a scale
Interval 2.96 to 3.44
Investigator Global Assessment (IGA) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 42
2.58 units on a scale
Interval 2.36 to 2.81
3.24 units on a scale
Interval 2.95 to 3.53
Investigator Global Assessment (IGA) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 77
2.20 units on a scale
Interval 1.92 to 2.48
2.20 units on a scale
Interval 1.84 to 2.55
Investigator Global Assessment (IGA) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 112
1.94 units on a scale
Interval 1.63 to 2.24
1.89 units on a scale
Interval 1.51 to 2.27

SECONDARY outcome

Timeframe: Day 0 (Prior to treatment), 3, 7, 14, 21, 28, 42, 77 and 112

Population: Modified intent-to-treat: All participants who were randomized and have Staphylococcus aureus abundance measured at Day 0 and Day 28.

SCORAD is a composite index comprising a) the amount/extent of body surface area affected, b) subjective symptom visual analog assessments of itch and sleep loss \[itch: 0 (no itch) to 10 (worst itch imaginable) / sleep loss: 0 (no sleep loss) to 10 (worst imaginable sleep loss)\], and c) 6 disease intensity assessments \[dryness, erythema, edema/papulation, excoriation, lichenification and oozing/crusting, each graded from 0-3: 0 (none), 1 (mild), 2 (moderate) and 3 (severe). The score ranges from 0 (no AD present) to 103 (severe). The mean SCORAD score in each treatment arm is reported from a linear mixed model for repeated measures with fixed effects for SCORAD at Day 0, treatment arm, clinical site, disease severity at Day 0 as measured by EASI ≥ 21.1 or \< 21.1, time point of measurement, and an interaction term between treatment arm and pre-specified time point(s). Additionally, a similar model was fit using all time points.

Outcome measures

Outcome measures
Measure
Dupilumab+Open Label Extension and Follow-Up
n=45 Participants
Participants received a loading dose of two 300 mg subcutaneous injections on Day 0 followed by 300 mg subcutaneous injections every two weeks (Days 14 and 28). Participants started a 10 week OLE on Day 42, beginning with a loading dose of two subcutaneously administered injections (one 300 mg dose of dupilumab and one dose of placebo, in order to protect prior masking/blind). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Placebo+Open Label Extension and Follow-Up
n=26 Participants
Placebo contained the identical formulation as the dupilumab formulation without the active monoclonal antibody and was given by exactly the same route and schedule through Day 28. Participants started a 10 week OLE on Day 42, beginning with a loading dose of dupilumab (two 300 mg subcutaneous injections \[total of 600 mg\]-protection of prior masking/blind maintained). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
SCORing Atopic Dermatitis (SCORAD) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 7
54.78 units on a scale
Interval 51.7 to 57.86
54.96 units on a scale
Interval 51.14 to 58.78
SCORing Atopic Dermatitis (SCORAD) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 21
39.40 units on a scale
Interval 35.53 to 43.28
51.87 units on a scale
Interval 47.01 to 56.73
SCORing Atopic Dermatitis (SCORAD) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 3
56.91 units on a scale
Interval 53.46 to 60.35
53.12 units on a scale
Interval 48.78 to 57.46
SCORing Atopic Dermatitis (SCORAD) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 14
46.55 units on a scale
Interval 43.09 to 50.0
52.01 units on a scale
Interval 47.66 to 56.73
SCORing Atopic Dermatitis (SCORAD) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 28
36.34 units on a scale
Interval 32.64 to 40.04
50.99 units on a scale
Interval 46.33 to 55.66
SCORing Atopic Dermatitis (SCORAD) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 42
32.22 units on a scale
Interval 28.38 to 36.06
51.02 units on a scale
Interval 46.15 to 55.9
SCORing Atopic Dermatitis (SCORAD) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 77
27.63 units on a scale
Interval 23.87 to 31.38
28.18 units on a scale
Interval 23.53 to 32.83
SCORing Atopic Dermatitis (SCORAD) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 112
23.92 units on a scale
Interval 20.25 to 27.59
25.01 units on a scale
Interval 20.57 to 29.46

SECONDARY outcome

Timeframe: Day 0 (Prior to treatment), 3, 7, 14, 21, 28, 42, 77 and 112

Population: Modified intent-to-treat: All participants who were randomized and have Staphylococcus aureus abundance measured at Day 0 and Day 28.

Pruritus NRS scale is an assessment tool that is used to report the average intensity of a participant's pruritus (itch) during a 24-hour recall period. Participants were asked to report the average itch experienced during the past 24 hours on a scale of 0 - 10 \[0= no itch; 10= worst imaginable itch\]). The mean Pruritus NRS score in each treatment arm is reported from a linear mixed model for repeated measures with fixed effects for Pruritus NRS at Day 0, treatment arm, clinical site, disease severity at Day 0 as measured by EASI ≥ 21.1 or \< 21.1, time point of measurement, and an interaction term between treatment arm and pre-specified time point(s). Additionally, a similar model was fit using all time points.

Outcome measures

Outcome measures
Measure
Dupilumab+Open Label Extension and Follow-Up
n=45 Participants
Participants received a loading dose of two 300 mg subcutaneous injections on Day 0 followed by 300 mg subcutaneous injections every two weeks (Days 14 and 28). Participants started a 10 week OLE on Day 42, beginning with a loading dose of two subcutaneously administered injections (one 300 mg dose of dupilumab and one dose of placebo, in order to protect prior masking/blind). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Placebo+Open Label Extension and Follow-Up
n=26 Participants
Placebo contained the identical formulation as the dupilumab formulation without the active monoclonal antibody and was given by exactly the same route and schedule through Day 28. Participants started a 10 week OLE on Day 42, beginning with a loading dose of dupilumab (two 300 mg subcutaneous injections \[total of 600 mg\]-protection of prior masking/blind maintained). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Pruritus Numerical Rating Scale (Pruritus NRS) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 3
5.37 units on a scale
Interval 4.73 to 6.01
5.64 units on a scale
Interval 4.83 to 6.45
Pruritus Numerical Rating Scale (Pruritus NRS) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 7
5.46 units on a scale
Interval 4.83 to 6.09
5.48 units on a scale
Interval 4.69 to 6.28
Pruritus Numerical Rating Scale (Pruritus NRS) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 14
4.48 units on a scale
Interval 3.91 to 5.05
5.29 units on a scale
Interval 4.58 to 6.0
Pruritus Numerical Rating Scale (Pruritus NRS) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 21
4.20 units on a scale
Interval 3.62 to 4.79
5.22 units on a scale
Interval 4.5 to 5.93
Pruritus Numerical Rating Scale (Pruritus NRS) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 28
3.67 units on a scale
Interval 3.07 to 4.28
5.14 units on a scale
Interval 4.37 to 5.9
Pruritus Numerical Rating Scale (Pruritus NRS) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 42
3.40 units on a scale
Interval 2.79 to 4.01
5.18 units on a scale
Interval 4.41 to 5.94
Pruritus Numerical Rating Scale (Pruritus NRS) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 77
2.55 units on a scale
Interval 2.05 to 3.05
1.88 units on a scale
Interval 1.28 to 2.48
Pruritus Numerical Rating Scale (Pruritus NRS) Score Within Each Treatment Group, Dupilumab+Open Label Extension and Follow-Up Versus Placebo+Open Label Extension and Follow-Up
Day 112
2.38 units on a scale
Interval 1.83 to 2.94
1.95 units on a scale
Interval 1.27 to 2.64

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 0 (Prior to treatment), 16 weeks

16S rRNA microbiome data (e.g., bacterial sequence reads) will be employed to identify changes in community composition and diversity at lesional and non-lesional skin sites prior to and throughout dupilumab or placebo treatment.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 0 (Prior to treatment), 16 weeks

The aim is to assess the effect of dupilumab on the skin transcriptome in lesional and non-lesional skin. Inclusion in this exploratory aim is restricted to non-University of Rochester Medical Center study participants only.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 0 (Prior to treatment) and 7

The aim is to assess the effect of dupilumab on gene expression in the skin transcriptome of non-lesional skin.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 0 (Prior to treatment), 7, and 21

The aim is to assess the effect of dupilumab on the gene expression in the skin transcriptome of lesional skin.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 0 (Prior to treatment), 16 weeks

The aim is to assess the effect of dupilumab on lipids, which play a role in the skin barrier, will be extracted from the skin tape strips and measured using mass spectrometry methodology. Skin tape strip method allows characterization of components of the epidermis, dermis, and immune cells present in the skin.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 0 (Prior to treatment), 16 weeks

The aim is to assess the effect of dupilumab on the expression of the bacterium Staphylococcus aureus (S. aureus) superantigens and toxins on lesional and non-lesional skin.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Days 0 (Prior to treatment), 7 and 21

The aim is to assess the effect of dupilumab on non-lesional skin barrier structure and Langerhans cells (LC) by confocal imaging. Inclusion in this exploratory aim is limited to University of Rochester Medical Center study participants.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 0 (Prior to treatment), 16 weeks

The aim is to assess the effect of dupilumab on the function of the skin microbiome (e.g., the ability of Coagulase-negative staphylococci isolates \[CoNS\] to kill S. aureus) in lesional and non-lesional skin.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Days 0 (Prior to treatment), 14 and 28

The aim is to assess the effect of dupilumab on PBMC immunoprofiles.Flow cytometry analysis will be performed on PBMCs, using phenotyping panels to identify resting leukocyte populations, as well as T cell responses to antigens and myeloid responses to Toll-like receptor (TLR) ligands.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 0 (Prior to treatment), 16 weeks

The aim is to assess the effect of dupilumab on serum biomarkers (e.g. T helper type 2 \[Th2\] biomarkers).

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 0 (Prior to treatment), 16 weeks

The presence of anti-drug antibodies will be assessed and compared between intervention groups.

Outcome measures

Outcome data not reported

OTHER_PRE_SPECIFIED outcome

Timeframe: Day 0 (Prior to treatment)

Towards discovery and replication of susceptibility loci in atopic dermatitis pathogenesis.

Outcome measures

Outcome data not reported

Adverse Events

Dupilumab+Open Label Extension and Follow-Up

Serious events: 1 serious events
Other events: 27 other events
Deaths: 0 deaths

Placebo+Open Label Extension and Follow-Up

Serious events: 0 serious events
Other events: 17 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
Dupilumab+Open Label Extension and Follow-Up
n=46 participants at risk
Participants received a loading dose of two 300 mg subcutaneous injections on Day 0 followed by 300 mg subcutaneous injections every two weeks (Days 14 and 28). Participants started a 10 week OLE on Day 42, beginning with a loading dose of two subcutaneously administered injections (one 300 mg dose of dupilumab and one dose of placebo, in order to protect prior masking/blind). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Placebo+Open Label Extension and Follow-Up
n=26 participants at risk
Placebo contained the identical formulation as the dupilumab formulation without the active monoclonal antibody and was given by exactly the same route and schedule through Day 28. Participants started a 10 week OLE on Day 42, beginning with a loading dose of dupilumab (two 300 mg subcutaneous injections \[total of 600 mg\]-protection of prior masking/blind maintained). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Injury, poisoning and procedural complications
Intentional overdose
2.2%
1/46 • Number of events 1 • Up to 182 days
0.00%
0/26 • Up to 182 days
Psychiatric disorders
Schizoaffective disorder bipolar type
2.2%
1/46 • Number of events 1 • Up to 182 days
0.00%
0/26 • Up to 182 days

Other adverse events

Other adverse events
Measure
Dupilumab+Open Label Extension and Follow-Up
n=46 participants at risk
Participants received a loading dose of two 300 mg subcutaneous injections on Day 0 followed by 300 mg subcutaneous injections every two weeks (Days 14 and 28). Participants started a 10 week OLE on Day 42, beginning with a loading dose of two subcutaneously administered injections (one 300 mg dose of dupilumab and one dose of placebo, in order to protect prior masking/blind). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Placebo+Open Label Extension and Follow-Up
n=26 participants at risk
Placebo contained the identical formulation as the dupilumab formulation without the active monoclonal antibody and was given by exactly the same route and schedule through Day 28. Participants started a 10 week OLE on Day 42, beginning with a loading dose of dupilumab (two 300 mg subcutaneous injections \[total of 600 mg\]-protection of prior masking/blind maintained). Participants then maintained a regimen of 300 mg of dupilumab by subcutaneous injection every two weeks through Day 98. The participants then entered a follow-up period until Day 182.
Eye disorders
Conjunctivitis allergic
2.2%
1/46 • Number of events 1 • Up to 182 days
7.7%
2/26 • Number of events 2 • Up to 182 days
Eye disorders
Dry eye
8.7%
4/46 • Number of events 6 • Up to 182 days
3.8%
1/26 • Number of events 1 • Up to 182 days
Gastrointestinal disorders
Nausea
6.5%
3/46 • Number of events 3 • Up to 182 days
0.00%
0/26 • Up to 182 days
Infections and infestations
Conjunctivitis
8.7%
4/46 • Number of events 4 • Up to 182 days
11.5%
3/26 • Number of events 3 • Up to 182 days
Infections and infestations
Furuncle
0.00%
0/46 • Up to 182 days
7.7%
2/26 • Number of events 2 • Up to 182 days
Infections and infestations
Nasopharyngitis
13.0%
6/46 • Number of events 7 • Up to 182 days
7.7%
2/26 • Number of events 3 • Up to 182 days
Infections and infestations
Upper respiratory tract infection
15.2%
7/46 • Number of events 10 • Up to 182 days
11.5%
3/26 • Number of events 3 • Up to 182 days
Injury, poisoning and procedural complications
Fall
2.2%
1/46 • Number of events 1 • Up to 182 days
11.5%
3/26 • Number of events 3 • Up to 182 days
Injury, poisoning and procedural complications
Skin abrasion
2.2%
1/46 • Number of events 1 • Up to 182 days
11.5%
3/26 • Number of events 3 • Up to 182 days
Nervous system disorders
Headache
2.2%
1/46 • Number of events 1 • Up to 182 days
15.4%
4/26 • Number of events 6 • Up to 182 days
Skin and subcutaneous tissue disorders
Dermatitis atopic
15.2%
7/46 • Number of events 16 • Up to 182 days
26.9%
7/26 • Number of events 11 • Up to 182 days
Skin and subcutaneous tissue disorders
Eczema
26.1%
12/46 • Number of events 12 • Up to 182 days
19.2%
5/26 • Number of events 7 • Up to 182 days
Skin and subcutaneous tissue disorders
Pruritus
8.7%
4/46 • Number of events 4 • Up to 182 days
11.5%
3/26 • Number of events 4 • Up to 182 days

Additional Information

Director, Clinical Research Operations Program

DAIT/NIAID

Phone: 240-669-5064

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place