Back to results

Safety, Pharmacokinetics and Efficacy of Dupilumab in Patients ≥6 Months to <6 Years With Moderate-to-Severe Atopic Dermatitis (Liberty AD PRESCHOOL) (Liberty AD)

Primary Purpose

Dermatitis, Atopic

Status

Completed

Phase

Phase 2

Locations

International

Study Type

Interventional

Intervention

Dupilumab

Matching placebo

About this trial

This is an interventional treatment trial for Dermatitis, Atopic focused on measuring Eczema

Eligibility Criteria

6 Months - 5 Years (Child)All SexesDoes not accept healthy volunteers

Key Inclusion Criteria

Diagnosis of atopic dermatitis (AD) according to the American Academy of Dermatology consensus criteria at the screening visit
Participants with documented recent history (within 6 months before the screening visit) of inadequate response to topical AD medication(s)
IGA score at screening and baseline visits
part A: IGA = 4
part B: IGA ≥3
EASI score at screening and baseline visits
part A: EASI ≥21
part B: EASI ≥16
Body Surface Area (BSA) involvement at screening and baseline visits
part A: ≥15%
part B: ≥10%
At least 11 (of a total of 14*) applications of a topical emollient (moisturizer) during the 7 consecutive days immediately before the baseline visit (not including the day of randomization) (for part B of the study only)
Baseline worst scratch/itch score weekly average score for maximum scratch/itch intensity ≥4 (for part B of the study only)
At least 11 (of a total of 14) daily applications of low potency TCS during the 2-week TCS standardization period (beginning on day -14) leading up to the baseline visit (for part B of the study only).

Key Exclusion Criteria

Prior treatment with dupilumab
History of important side effects of low potency topical corticosteroids (only applicable for part B of the study)
Having used immunosuppressive/immunomodulating drugs within 4 weeks before the baseline visit
Treatment with a live (attenuated) vaccine within 4 weeks before the baseline visit
Active chronic or acute infection requiring treatment with systemic antibiotics, antivirals, antiprotozoals, or antifungals within 2 weeks before the baseline visit.
Known or suspected immunodeficiency, known history of human immunodeficiency virus (HIV) infection or HIV seropositivity at the screening visit, established diagnosis of HBV infection or HBV seropositivity at screening, established diagnosis of HCV infection or HCV seropositivity at screening
History of malignancy at any time before the baseline visit
Diagnosed active endoparasitic infections or at high risk of these infections
Severe concomitant illness(es) that, in the investigator's judgment, would adversely affect the patient's participation in the study
Body weight <5 kg or ≥30 kg at baseline (only applicable part B of the study)

Note: Other protocol defined Inclusion/ Exclusion criteria apply

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Arm Label

Part A (Open label Dupilumab): Age cohorts 1 & 2

Part B (Double-blind): Dupilumab dose 1

Part B (Double-blind): Dupilumab dose 2

Part B (Double-Blind): Placebo

Arm Description

Age cohort 1: ≥2 years old to <6 years old Age cohort 2: ≥6 months to <2 years old

The results of part A will be used to guide the selection of dose levels and dosing frequency for part B.

Outcomes

Primary Outcome Measures

Part A: Maximum Observed Serum Concentration (Cmax) of Functional Dupilumab

Serum concentration of functional dupilumab was reported.

Part A: Dose Normalized Maximum Observed Serum Concentration (Cmax/Dose) of Dupilumab

Dose normalized was calculated as Cmax obtained directly from the concentration versus time curve divided by dose. Cmax/dose was measured in Milligrams per Liter/Milligrams per Kilogram ([mg/L]/[mg/kg]).

Part A: Time to Reach the Maximum Serum Concentration (Tmax) of Dupilumab

Tmax was obtained directly from the concentration versus time curve.

Part A: Last Quantifiable Serum Concentration (Clast) of Dupilumab

Clast is the last measurable serum concentration of dupilumab.

Part A: Time of the Last Quantifiable Serum Concentration (Tlast) of Dupilumab

Tlast was defined as the last time point with a measurable serum concentration of dupilumab.

Part A: Area Under the Serum Concentration-Time Curve From Time Zero to the Time of the Last Measurable Concentration (AUClast) of Dupilumab

AUClast was defined as area under the serum concentration time-curve from zero to the last measured concentration.

Part A: Dose Normalized Area Under the Serum Concentration-Time Curve From Time Zero to the Time of the Last Measurable Concentration (AUClast/Dose) of Dupilumab

Dose normalized AUClast was calculated by AUClast/dose.

Part A: Number of Participants With at Least One Treatment-Emergent Adverse Event (TEAE)

Adverse Event (AE) was defined as any untoward medical occurrence in a participant administered a study drug which may/may not have a causal relationship with study drug. Serious AE (SAE) was defined as any untoward medical occurrence that resulted in any of following outcomes: death, life-threatening, required initial/prolonged in-participant hospitalization, persistent/significant disability/incapacity, congenital anomaly/birth defect/considered as medically important event. TEAE was defined as AE starting/worsening after first intake of study drug. TEAEs included participants with both SAEs and non-SAEs. Number of participants with TEAEs is reported.

Part A: Number of Participants With TEAEs by Severity According to Qualitative Toxicity Scale

Severity of TEAEs were graded using Qualitative Toxicity Scale, as follows: Mild: Participant is aware of the event or symptom, but the event or symptom is easily tolerated; Moderate: Participant experiences sufficient discomfort to interfere with or reduce his or her usual level of activity; Severe: Significant impairment of functioning: the participant is unable to carry out his or her usual activities. Number of participants with TEAEs by severity were reported.

Part B: Percentage of Participants With Investigator's Global Assessment (IGA) Score 0 or 1 at Week 16

The IGA is an assessment scale used in clinical studies to rate the severity of AD globally, based on a 5-point scale ranging from 0 to 4 where 0 = clear; 1=almost clear; 2=mild; 3=moderate; 4=severe. A negative change from baseline indicated improvement. Percentage of participants with IGA score of '0' or '1' is reported.

Part B: Percentage of Participants With Eczema Area and Severity Index (EASI) -75 (EASI-75) (≥75% Improvement From Baseline) at Week 16

The EASI score is used to measure the severity and extent of AD and measured erythema, infiltration, excoriation and lichenification on 4 anatomic regions of the body: head, trunk, upper, and lower extremities. The total EASI score ranges from 0 (minimum) to 72 (maximum) points, with the higher scores indicating the worse severity of AD. EASI-75 responders were the participants who achieved ≥75% overall improvement in EASI score from baseline at Week 16.

Secondary Outcome Measures

Part A: Number of Participants With Serious TEAEs and Severe TEAEs

Adverse Event (AE) was defined as any untoward medical occurrence in a participant administered a study drug which may/may not have a causal relationship with study drug. A serious TEAE was an AE that resulted in any of the following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect. Severe TEAE: significant impairment of functioning the participant is unable to carry out his or her usual activities.

Part A: Percent Change From Baseline in EASI Score at Week 4

The EASI score is used to measure the severity and extent of AD and measured erythema, infiltration, excoriation, and lichenification on 4 anatomic regions of the body: head, trunk, upper, and lower extremities. The total EASI score ranges from 0 (minimum) to 72 (maximum) points, with the higher scores indicated the worse severity of AD. A negative change from baseline indicated improvement.

Part A: Percent Change From Baseline in SCORing Atopic Dermatitis (SCORAD) Score at Week 4

The SCORAD is used to assess the extent and severity of AD. Extent and severity of eczema as well as subjective symptoms (insomnia, etc) were assessed and scored. SCORAD total score ranges from 0 (absent disease) to 103 (severe disease). A negative change from baseline indicated improvement.

Part A: Percentage of Participants With IGA Score 0 or 1 at Week 4

The IGA is an assessment scale used in clinical studies to rate the severity of AD globally, based on a 5-point scale ranging from 0 to 4 where 0 = clear; 1 = almost clear; 2 = mild; 3 = moderate; 4 = severe. Percentage of participants with IGA score of '0' or '1' were reported.

Part A: Number of Participants With at Least One Positive Treatment-Emergent Anti-Drug Antibodies (ADA)

Treatment boosted (TB) Response: Any post-dose positive result at least 9-fold over the baseline level when baseline is positive; Treatment emergent (TE) Response: Post-dose positive result when baseline results were negative.

Part B: Number of Participants With at Least One Serious Adverse Event (SAE) Through Week 16

Part B: Number of Participants With at Least One Skin Infection Treatment Emergent Adverse Event (TEAE) (Excluding Herpetic Infection) Through Week 16

Part B: Number of Participants With at Least One Positive Treatment-Emergent ADA

Treatment emergent (TE): Post-dose positive result when baseline results were negative.

Part B: Percent Change From Baseline in EASI Score at Week 16

The EASI score is used to measure the severity and extent of AD and measures erythema, infiltration, excoriation, and lichenification on 4 anatomic regions of the body: head, trunk, upper, and lower extremities. The total EASI score ranges from 0 (minimum) to 72 (maximum) points, with the higher scores indicating the worse severity of AD. A negative change from baseline indicated improvement.

Part B: Percent Change From Baseline in Weekly Average of Daily Worst Scratch/Itch/Numerical Rating Scale (NRS) at Week 16

Pruritus NRS is an assessment tool used to report intensity of participant's pruritus (itch), both average & maximum intensity, during 24-hr recall period. Participants were asked two questions: 1) For average itch intensity: how would you rate your itch overall (on average) during the previous 24 hrs; 2) For maximum itch intensity: How would you rate your itch at the worst moment during the previous 24 hrs? Both questions were rated on a scale: 0-10 with 0=no itch & 10=worst itch imaginable. A negative change from baseline indicated improvement.

Part B: Percentage of Participants With Improvement (Reduction From Baseline) of Weekly Average of Daily Worst Scratch/Itch/NRS ≥4 Points at Week 16

Pruritus NRS is an assessment tool used to report intensity of subject's pruritus (itch), both average & maximum intensity, during 24-hr recall period. Subjects were asked two questions: 1) For average itch intensity: how would you rate your itch overall (on average) during the previous 24 hrs; & 2) For maximum itch intensity: How would you rate your itch at the worst moment during the previous 24 hrs? Both questions were rated on a scale: 0-10 with 0=no itch & 10=worst itch imaginable.

Part B: Percentage of Participants With Improvement (Reduction From Baseline) of Weekly Average of Daily Worst Scratch/Itch/NRS ≥3 Points at Week 16

Pruritus NRS is an assessment tool used to report intensity of participant's pruritus (itch), both average & maximum intensity, during 24-hr recall period. Participants were asked two questions: 1) For average itch intensity: how would you rate your itch overall (on average) during the previous 24 hrs; & 2) For maximum itch intensity: How would you rate your itch at the worst moment during the previous 24 hrs? Both questions were rated on a scale: 0-10 with 0=no itch & 10=worst itch imaginable.

Part B: Percentage of Participants Who Achieved EASI-50 (≥50% Improvement From Baseline) at Week 16

The EASI score is used to measure the severity and extent of AD and measured erythema, infiltration, excoriation, and lichenification on 4 anatomic regions of the body: head, trunk, upper, and lower extremities. The total EASI score ranges from 0 (minimum) to 72 (maximum) points, with the higher scores indicating the worse severity of AD. EASI-50 responders were the participants who achieved ≥50% overall improvement in EASI score from baseline at Week 16.

Part B: Percentage of Participants Who Achieved EASI-90 (≥90% Improvement From Baseline) at Week 16

The EASI score is used to measure the severity and extent of AD and measured erythema, infiltration, excoriation, and lichenification on 4 anatomic regions of the body: head, trunk, upper, and lower extremities. The total EASI score ranges from 0 (minimum) to 72 (maximum) points, with the higher scores indicating the worse severity of AD. EASI-90 responders were the participant who achieved ≥90% overall improvement in EASI score from baseline at Week 16.

Part B: Change From Baseline in Percent Body Surface Area (BSA) Affected by Atopic Dermatitis (AD) at Week 16

BSA affected by AD was assessed for each section of the body (the possible highest score for each region was: head and neck [9%], anterior trunk [18%], back [18%], upper limbs [18%], lower limbs [36%], and genitals [1%]). It was reported as a percentage of all major body sections combined. A negative change from baseline indicated improvement.

Part B: Change From Baseline in Patient Oriented Eczema Measure (POEM) at Week 16

The POEM is a 7-item questionnaire that assesses disease symptoms (dryness, itching, flaking, cracking, sleep loss, bleeding and weeping) with a scoring system of 0 (absent disease) to 28 (severe disease) (high score indicative of poor quality of life [QOL]). A negative change from baseline indicated improvement.

Part B: Percent Change From Baseline in SCORing Atopic Dermatitis (SCORAD) at Week 16

The SCORAD index is a clinical tool for assessing the severity of atopic dermatitis (AD). Extent and intensity of eczema as well as subjective signs (insomnia, etc.) are assessed and scored. Total score ranges from 0 (absent disease) to 103 (severe disease). A negative change from baseline indicated improvement.

Part B: Change From Baseline in Participant's Sleep Quality NRS at Week 16

A sleep diary is completed by the parent/caregiver, included 2 questions assessing the caregiver's sleep, and 6 questions assessing the child's sleep based on caregiver observation. Sleep diary items, either alone or in combination serve as subjective measures of sleep quality, difficulty falling asleep, nighttime awakenings, and sleep duration. Sleep quality is measured using an 11-point NRS (0 to 10) in which 0 indicates worst possible sleep while 10 indicates best possible sleep.

Part B: Change From Baseline in Participant's Skin Pain NRS at Week 16

Skin pain was assessed by the parent/caregiver and measured using a 11-point scale (0 to 10) in which 0 indicated no pain while 10 indicated worst pain possible. A negative change from baseline indicated improvement.

Part B: Change From Baseline in Dermatitis Family Index (DFI) at Week 16

DFI is a 10-item questionnaire with items inquiring about housework, food preparation, sleep, family leisure activity, shopping, expenditure, tiredness, emotional distress, relationships, and impact of helping with treatment on the primary caregiver's life. DFI questions were scored on a four-point Likert scale ranging from 0 to 3, so that the total DFI score ranges from 0 to 30. Timeframe of reference was the past week. A higher DFI score indicated greater impairment in family Quality of life (QOL) as affected by atopic dermatitis. A negative change from baseline indicated improvement.

Part B: Change From Baseline in Children's Dermatology Life Quality Index (CDLQI) at Week 16

CDLQI is a validated 10 question tool to measure impact of skin disease on QOL in children by assessing how much the skin problem has affected the subjects over past week. Nine questions were scored as follows: Very much = 3, Quite a lot = 2, Only a little = 1, Not at all or unanswered = 0. Question 7 has an added possible response, which was scored as 3. CDLQI equals the sum of the score of each question (max. = 30, min. = 0). Higher the score, the greater the impact on QOL. A negative change from baseline indicated improvement.

Part B: Change From Baseline in Infants' Dermatology Quality of Life Index (IDQOL) at Week 16

Infants' Dermatitis Quality of Life Index (IDQOL) is used to evaluate quality of life for subjects of age less than 4 years. IDQOL questionnaires were designed for infants (below the age of 4 years) with atopic dermatitis. The IDQOL was calculated by summing the score of each question resulting in a maximum of 30 and a minimum of 0. The higher the score in each questionnaire, the more quality of life is impaired. A negative change from baseline indicated improvement.

Part B: Percentage of Topical Corticosteroid (TCS) Medication-free Days From Baseline to Week 16

Percentage of TCS medication-free days was calculated as the number of days that a subject used neither TCS/TCI nor system rescue therapy divided by the study days.

Part B: Mean Weekly Dose of Low Potency TCS in Grams From Baseline to Week 16

Mean weekly dose of TCS in grams/week for low potency TCS from baseline to Week 16 is reported.

Part B: Mean Weekly Dose of TCS in Grams for Medium or High Potency TCS From Baseline to Week 16

Mean weekly dose of TCS in grams/week for medium or high potency TCS from baseline to Week 16 is reported.

Part B: Mean Number of Caregiver Missed Work Days Through Week 16

Mean of number of caregiver missed work days through Week 16 is reported.

Full Information

NCT ID

NCT03346434

First Posted

February 6, 2017

Last Updated

July 27, 2022

Sponsor

Regeneron Pharmaceuticals

Collaborators

Sanofi

1. Study Identification

Unique Protocol Identification Number

NCT03346434

Brief Title

Safety, Pharmacokinetics and Efficacy of Dupilumab in Patients ≥6 Months to <6 Years With Moderate-to-Severe Atopic Dermatitis (Liberty AD PRESCHOOL)

Acronym

Liberty AD

Official Title

A Phase 2/3 Study Investigating the Pharmacokinetics, Safety, and Efficacy of Dupilumab in Patients Aged ≥6 Months to <6 Years With Moderate-to-Severe Atopic Dermatitis

Study Type

Interventional

2. Study Status

Record Verification Date

July 2022

Overall Recruitment Status

Completed

Study Start Date

November 30, 2017 (Actual)

Primary Completion Date

July 8, 2021 (Actual)

Study Completion Date

July 8, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Regeneron Pharmaceuticals

Collaborators

Sanofi

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

This study is a 2-part (parts A and B) phase 2/3 study to evaluate the safety, pharmacokinetics (PK) and efficacy of dupilumab in participants 6 months to less than 6 years of age with moderate-to-severe atopic dermatitis (AD).

Detailed Description

Part A (open-label, single-ascending-dose, sequential cohort phase 2 study): Primary objective is to characterize the safety and PK of dupilumab administered as a single dose in pediatric participants, 6 months to less than 6 years of age, with severe AD. Secondary objective is to evaluate the efficacy and immunogenicity of a single dose of dupilumab in participants 6 months to less than 6 years of age with severe AD. Part B (randomized, double-blind, parallel-group, placebo-controlled phase 3 study): Primary objective is to demonstrate the efficacy of multiple doses of dupilumab over 16 weeks of treatment when administered concomitantly with topical corticosteroids (TCS) in pediatric participants, 6 months to less than 6 years of age, with moderate-to-severe AD. Secondary objective is to assess the safety and immunogenicity of multiple doses of dupilumab over 16 weeks of treatment when administered concomitantly with TCS in participants 6 months to less than 6 years of age with moderate-to-severe AD.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Dermatitis, Atopic

Keywords

Eczema

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2, Phase 3

Interventional Study Model

Single Group Assignment

Model Description

Part A: Single-ascending-dose cohorts staggered by age; Part B: Parallel Group

Masking

None (Open Label)

Masking Description

Part A: Open Label; Part B: Masked, Randomized

Allocation

Non-Randomized

Enrollment

202 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Part A (Open label Dupilumab): Age cohorts 1 & 2

Arm Type

Experimental

Arm Description

Age cohort 1: ≥2 years old to <6 years old Age cohort 2: ≥6 months to <2 years old

Arm Title

Part B (Double-blind): Dupilumab dose 1

Arm Type

Experimental

Arm Description

The results of part A will be used to guide the selection of dose levels and dosing frequency for part B.

Arm Title

Part B (Double-blind): Dupilumab dose 2

Arm Type

Experimental

Arm Description

The results of part A will be used to guide the selection of dose levels and dosing frequency for part B.

Arm Title

Part B (Double-Blind): Placebo

Arm Type

Experimental

Intervention Type

Drug

Intervention Name(s)

Dupilumab

Other Intervention Name(s)

DUPIXENT®, REGN668, SAR231893

Intervention Description

Solution for injection, subcutaneous (SC)

Intervention Type

Drug

Intervention Name(s)

Matching placebo

Intervention Description

Solution for injection, subcutaneous (SC)

Primary Outcome Measure Information:

Title

Part A: Maximum Observed Serum Concentration (Cmax) of Functional Dupilumab

Description

Serum concentration of functional dupilumab was reported.

Time Frame

Post-dose on Days 1, 3, 8, 18, and 29

Title

Part A: Dose Normalized Maximum Observed Serum Concentration (Cmax/Dose) of Dupilumab

Description

Time Frame

Post-dose on Days 1, 3, 8, 18, and 29

Title

Part A: Time to Reach the Maximum Serum Concentration (Tmax) of Dupilumab

Description

Tmax was obtained directly from the concentration versus time curve.

Time Frame

Post-dose on Days 1, 3, 8, 18, and 29

Title

Part A: Last Quantifiable Serum Concentration (Clast) of Dupilumab

Description

Clast is the last measurable serum concentration of dupilumab.

Time Frame

Post-dose on Days 1, 3, 8, 18, and 29

Title

Part A: Time of the Last Quantifiable Serum Concentration (Tlast) of Dupilumab

Description

Tlast was defined as the last time point with a measurable serum concentration of dupilumab.

Time Frame

Post-dose on Days 1, 3, 8, 18, and 29

Title

Part A: Area Under the Serum Concentration-Time Curve From Time Zero to the Time of the Last Measurable Concentration (AUClast) of Dupilumab

Description

AUClast was defined as area under the serum concentration time-curve from zero to the last measured concentration.

Time Frame

Post-dose on Days 1, 3, 8, 18, and 29

Title

Part A: Dose Normalized Area Under the Serum Concentration-Time Curve From Time Zero to the Time of the Last Measurable Concentration (AUClast/Dose) of Dupilumab

Description

Dose normalized AUClast was calculated by AUClast/dose.

Time Frame

Post-dose on Days 1, 3, 8, 18, and 29

Title

Part A: Number of Participants With at Least One Treatment-Emergent Adverse Event (TEAE)

Description

Time Frame

Baseline up to Week 4

Title

Part A: Number of Participants With TEAEs by Severity According to Qualitative Toxicity Scale

Description

Time Frame

Baseline up to Week 4

Title

Part B: Percentage of Participants With Investigator's Global Assessment (IGA) Score 0 or 1 at Week 16

Description

Time Frame

Week 16

Title

Part B: Percentage of Participants With Eczema Area and Severity Index (EASI) -75 (EASI-75) (≥75% Improvement From Baseline) at Week 16

Description

Time Frame

Week 16

Secondary Outcome Measure Information:

Title

Part A: Number of Participants With Serious TEAEs and Severe TEAEs

Description

Time Frame

Baseline up to Week 4

Title

Part A: Percent Change From Baseline in EASI Score at Week 4

Description

Time Frame

Week 4

Title

Part A: Percent Change From Baseline in SCORing Atopic Dermatitis (SCORAD) Score at Week 4

Description

Time Frame

Week 4

Title

Part A: Percentage of Participants With IGA Score 0 or 1 at Week 4

Description

The IGA is an assessment scale used in clinical studies to rate the severity of AD globally, based on a 5-point scale ranging from 0 to 4 where 0 = clear; 1 = almost clear; 2 = mild; 3 = moderate; 4 = severe. Percentage of participants with IGA score of '0' or '1' were reported.

Time Frame

Week 4

Title

Part A: Number of Participants With at Least One Positive Treatment-Emergent Anti-Drug Antibodies (ADA)

Description

Time Frame

Baseline up to Day 57

Title

Part B: Number of Participants With at Least One Serious Adverse Event (SAE) Through Week 16

Time Frame

Baseline through Week 16

Title

Part B: Number of Participants With at Least One Skin Infection Treatment Emergent Adverse Event (TEAE) (Excluding Herpetic Infection) Through Week 16

Time Frame

Baseline through Week 16

Title

Part B: Number of Participants With at Least One Positive Treatment-Emergent ADA

Description

Treatment emergent (TE): Post-dose positive result when baseline results were negative.

Time Frame

Baseline up to Day 197

Title

Part B: Percent Change From Baseline in EASI Score at Week 16

Description

Time Frame

Week 16

Title

Part B: Percent Change From Baseline in Weekly Average of Daily Worst Scratch/Itch/Numerical Rating Scale (NRS) at Week 16

Description

Time Frame

Week 16

Title

Part B: Percentage of Participants With Improvement (Reduction From Baseline) of Weekly Average of Daily Worst Scratch/Itch/NRS ≥4 Points at Week 16

Description

Time Frame

Week 16

Title

Part B: Percentage of Participants With Improvement (Reduction From Baseline) of Weekly Average of Daily Worst Scratch/Itch/NRS ≥3 Points at Week 16

Description

Pruritus NRS is an assessment tool used to report intensity of participant's pruritus (itch), both average & maximum intensity, during 24-hr recall period. Participants were asked two questions: 1) For average itch intensity: how would you rate your itch overall (on average) during the previous 24 hrs; & 2) For maximum itch intensity: How would you rate your itch at the worst moment during the previous 24 hrs? Both questions were rated on a scale: 0-10 with 0=no itch & 10=worst itch imaginable.

Time Frame

Week 16

Title

Part B: Percentage of Participants Who Achieved EASI-50 (≥50% Improvement From Baseline) at Week 16

Description

The EASI score is used to measure the severity and extent of AD and measured erythema, infiltration, excoriation, and lichenification on 4 anatomic regions of the body: head, trunk, upper, and lower extremities. The total EASI score ranges from 0 (minimum) to 72 (maximum) points, with the higher scores indicating the worse severity of AD. EASI-50 responders were the participants who achieved ≥50% overall improvement in EASI score from baseline at Week 16.

Time Frame

Week 16

Title

Part B: Percentage of Participants Who Achieved EASI-90 (≥90% Improvement From Baseline) at Week 16

Description

The EASI score is used to measure the severity and extent of AD and measured erythema, infiltration, excoriation, and lichenification on 4 anatomic regions of the body: head, trunk, upper, and lower extremities. The total EASI score ranges from 0 (minimum) to 72 (maximum) points, with the higher scores indicating the worse severity of AD. EASI-90 responders were the participant who achieved ≥90% overall improvement in EASI score from baseline at Week 16.

Time Frame

Week 16

Title

Part B: Change From Baseline in Percent Body Surface Area (BSA) Affected by Atopic Dermatitis (AD) at Week 16

Description

Time Frame

Week 16

Title

Part B: Change From Baseline in Patient Oriented Eczema Measure (POEM) at Week 16

Description

Time Frame

Week 16

Title

Part B: Percent Change From Baseline in SCORing Atopic Dermatitis (SCORAD) at Week 16

Description

Time Frame

Week 16

Title

Part B: Change From Baseline in Participant's Sleep Quality NRS at Week 16

Description

Time Frame

Week 16

Title

Part B: Change From Baseline in Participant's Skin Pain NRS at Week 16

Description

Time Frame

Week 16

Title

Part B: Change From Baseline in Dermatitis Family Index (DFI) at Week 16

Description

Time Frame

Week 16

Title

Part B: Change From Baseline in Children's Dermatology Life Quality Index (CDLQI) at Week 16

Description

Time Frame

Week 16

Title

Part B: Change From Baseline in Infants' Dermatology Quality of Life Index (IDQOL) at Week 16

Description

Time Frame

Week 16

Title

Part B: Percentage of Topical Corticosteroid (TCS) Medication-free Days From Baseline to Week 16

Description

Percentage of TCS medication-free days was calculated as the number of days that a subject used neither TCS/TCI nor system rescue therapy divided by the study days.

Time Frame

Baseline up to Week 16

Title

Part B: Mean Weekly Dose of Low Potency TCS in Grams From Baseline to Week 16

Description

Mean weekly dose of TCS in grams/week for low potency TCS from baseline to Week 16 is reported.

Time Frame

Baseline up to Week 16

Title

Part B: Mean Weekly Dose of TCS in Grams for Medium or High Potency TCS From Baseline to Week 16

Description

Mean weekly dose of TCS in grams/week for medium or high potency TCS from baseline to Week 16 is reported.

Time Frame

Baseline up to Week 16

Title

Part B: Mean Number of Caregiver Missed Work Days Through Week 16

Description

Mean of number of caregiver missed work days through Week 16 is reported.

Time Frame

Baseline through Week 16

10. Eligibility

Sex

All

Minimum Age & Unit of Time

6 Months

Maximum Age & Unit of Time

5 Years

Accepts Healthy Volunteers

Eligibility Criteria

Key Inclusion Criteria Diagnosis of atopic dermatitis (AD) according to the American Academy of Dermatology consensus criteria at the screening visit Participants with documented recent history (within 6 months before the screening visit) of inadequate response to topical AD medication(s) IGA score at screening and baseline visits part A: IGA = 4 part B: IGA ≥3 EASI score at screening and baseline visits part A: EASI ≥21 part B: EASI ≥16 Body Surface Area (BSA) involvement at screening and baseline visits part A: ≥15% part B: ≥10% At least 11 (of a total of 14*) applications of a topical emollient (moisturizer) during the 7 consecutive days immediately before the baseline visit (not including the day of randomization) (for part B of the study only) Baseline worst scratch/itch score weekly average score for maximum scratch/itch intensity ≥4 (for part B of the study only) At least 11 (of a total of 14) daily applications of low potency TCS during the 2-week TCS standardization period (beginning on day -14) leading up to the baseline visit (for part B of the study only). Key Exclusion Criteria Prior treatment with dupilumab History of important side effects of low potency topical corticosteroids (only applicable for part B of the study) Having used immunosuppressive/immunomodulating drugs within 4 weeks before the baseline visit Treatment with a live (attenuated) vaccine within 4 weeks before the baseline visit Active chronic or acute infection requiring treatment with systemic antibiotics, antivirals, antiprotozoals, or antifungals within 2 weeks before the baseline visit. Known or suspected immunodeficiency, known history of human immunodeficiency virus (HIV) infection or HIV seropositivity at the screening visit, established diagnosis of HBV infection or HBV seropositivity at screening, established diagnosis of HCV infection or HCV seropositivity at screening History of malignancy at any time before the baseline visit Diagnosed active endoparasitic infections or at high risk of these infections Severe concomitant illness(es) that, in the investigator's judgment, would adversely affect the patient's participation in the study Body weight <5 kg or ≥30 kg at baseline (only applicable part B of the study) Note: Other protocol defined Inclusion/ Exclusion criteria apply

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Clinical Trial Management

Organizational Affiliation

Regeneron Pharmaceuticals

Official's Role

Study Director

Facility Information:

Facility Name

Regeneron Investigational Site

City

Birmingham

State/Province

Alabama

ZIP/Postal Code

35209

Country

United States

Facility Name

Regeneron Investigational Site

City

Gilbert

State/Province

Arizona

ZIP/Postal Code

85234

Country

United States

Facility Name

Regeneron Investigational Site

City

Long Beach

State/Province

California

ZIP/Postal Code

90808

Country

United States

Facility Name

Regeneron Investigational site

City

Los Angeles

State/Province

California

ZIP/Postal Code

90027

Country

United States

Facility Name

Regeneron Investigational Site

City

Palo Alto

State/Province

California

ZIP/Postal Code

94304

Country

United States

Facility Name

Regeneron Investigational Site

City

Rolling Hills Estates

State/Province

California

ZIP/Postal Code

90274

Country

United States

Facility Name

Regeneron Investigational Site

City

San Diego

State/Province

California

ZIP/Postal Code

92123

Country

United States

Facility Name

Regeneron Investigational Site

City

Washington

State/Province

District of Columbia

ZIP/Postal Code

20010

Country

United States

Facility Name

Regeneron Investigational Site

City

Coral Gables

State/Province

Florida

ZIP/Postal Code

33146

Country

United States

Facility Name

Regeneron Investigational Site

City

Tampa

State/Province

Florida

ZIP/Postal Code

33612-3807

Country

United States

Facility Name

Regeneron Investigational Site

City

Columbus

State/Province

Georgia

ZIP/Postal Code

31904

Country

United States

Facility Name

Regeneron Investigational Site

City

Macon

State/Province

Georgia

ZIP/Postal Code

31217

Country

United States

Facility Name

Regeneron Investigational Site

City

Sandy Springs

State/Province

Georgia

ZIP/Postal Code

39328

Country

United States

Facility Name

Regeneron Investigational Site

City

Chicago

State/Province

Illinois

ZIP/Postal Code

60611

Country

United States

Facility Name

Regeneron Investigational Site

City

Rockville

State/Province

Maryland

ZIP/Postal Code

20850

Country

United States

Facility Name

Regeneron Investigational Site

City

Boston

State/Province

Massachusetts

ZIP/Postal Code

02115

Country

United States

Facility Name

Regeneron Investigational Site

City

Ann Arbor

State/Province

Michigan

ZIP/Postal Code

48109-5314

Country

United States

Facility Name

Regeneron Investigational Site

City

Ypsilanti

State/Province

Michigan

ZIP/Postal Code

48197

Country

United States

Facility Name

Regeneron Investigational Site

City

Saint Louis

State/Province

Missouri

ZIP/Postal Code

63104-1003

Country

United States

Facility Name

Regeneron Investigational Site

City

Lincoln

State/Province

Nebraska

ZIP/Postal Code

68505

Country

United States

Facility Name

Regeneron Investigational Site

City

Hackensack

State/Province

New Jersey

ZIP/Postal Code

07601

Country

United States

Facility Name

Regeneron Investigational Site

City

Forest Hills

State/Province

New York

ZIP/Postal Code

113756

Country

United States

Facility Name

Regeneron Investigational Site

City

New York

State/Province

New York

ZIP/Postal Code

10032

Country

United States

Facility Name

Regeneron Investigational Site

City

Rochester

State/Province

New York

ZIP/Postal Code

14642

Country

United States

Facility Name

Regeneron Investigational Site

City

Portland

State/Province

Oregon

ZIP/Postal Code

97239

Country

United States

Facility Name

Regeneron Investigational Site

City

Philadelphia

State/Province

Pennsylvania

ZIP/Postal Code

19104

Country

United States

Facility Name

Regeneron Investigational Site

City

Charleston

State/Province

South Carolina

ZIP/Postal Code

29425

Country

United States

Facility Name

Regeneron Investigational Site

City

North Charleston

State/Province

South Carolina

ZIP/Postal Code

29420

Country

United States

Facility Name

Regeneron Investigational Site

City

Austin

State/Province

Texas

ZIP/Postal Code

78723

Country

United States

Facility Name

Regeneron Investigational Site

City

San Antonio

State/Province

Texas

ZIP/Postal Code

78218

Country

United States

Facility Name

Regeneron Investigational Site

City

Norfolk

State/Province

Virginia

ZIP/Postal Code

23502

Country

United States

Facility Name

Regeneron Investigational Site

City

Seattle

State/Province

Washington

ZIP/Postal Code

98105

Country

United States

Facility Name

Regeneron Investigational Site

City

Milwaukee

State/Province

Wisconsin

ZIP/Postal Code

53226

Country

United States

Facility Name

Regeneron Investigational Site

City

Osnabrück

State/Province

Lower Saxony

ZIP/Postal Code

49074

Country

Germany

Facility Name

Regeneron Investigational site

City

Muenster

State/Province

North Rhine-Westphal

ZIP/Postal Code

48149

Country

Germany

Facility Name

Regeneron Investigational Site

City

Dresden

State/Province

Sachsen

ZIP/Postal Code

01307

Country

Germany

Facility Name

Regeneron Investigational site

City

Frankfurt/ Main

ZIP/Postal Code

60590

Country

Germany

Facility Name

Regeneron Investigational Site

City

Kiel

ZIP/Postal Code

24105

Country

Germany

Facility Name

Regeneron Investigational Site

City

Muenchen

ZIP/Postal Code

80337

Country

Germany

Facility Name

Regeneron Investigational site

City

Muenchen

ZIP/Postal Code

80802

Country

Germany

Facility Name

Regeneron Investigational Site

City

Bialystok

ZIP/Postal Code

15-453

Country

Poland

Facility Name

Regeneron Investigational Site

City

Katowice

ZIP/Postal Code

40-648

Country

Poland

Facility Name

Regeneron Investigational Site

City

Krakow

ZIP/Postal Code

31-011

Country

Poland

Facility Name

Regeneron Investigational Site

City

Ostrowiec Swietokrzyski

ZIP/Postal Code

27-400

Country

Poland

Facility Name

Regeneron Investigational Site

City

Warszawa

ZIP/Postal Code

01-142

Country

Poland

Facility Name

Regeneron Investigational Site

City

Warszawa

ZIP/Postal Code

01-817

Country

Poland

Facility Name

Regeneron Investigational Site

City

Manchester

State/Province

Lancashire

ZIP/Postal Code

M13 9WL

Country

United Kingdom

Facility Name

Regeneron Investigational Site

City

Sheffield

State/Province

South Yorkshire

ZIP/Postal Code

S10 2TH

Country

United Kingdom

12. IPD Sharing Statement

Citations:

PubMed Identifier

36116481

Citation

Paller AS, Simpson EL, Siegfried EC, Cork MJ, Wollenberg A, Arkwright PD, Soong W, Gonzalez ME, Schneider LC, Sidbury R, Lockshin B, Meltzer S, Wang Z, Mannent LP, Amin N, Sun Y, Laws E, Akinlade B, Dillon M, Kosloski MP, Kamal MA, Dubost-Brama A, Patel N, Weinreich DM, Yancopoulos GD, O'Malley JT, Bansal A; participating investigators. Dupilumab in children aged 6 months to younger than 6 years with uncontrolled atopic dermatitis: a randomised, double-blind, placebo-controlled, phase 3 trial. Lancet. 2022 Sep 17;400(10356):908-919. doi: 10.1016/S0140-6736(22)01539-2.

Results Reference

derived

Learn more about this trial

Safety, Pharmacokinetics and Efficacy of Dupilumab in Patients ≥6 Months to <6 Years With Moderate-to-Severe Atopic Dermatitis (Liberty AD PRESCHOOL)

We'll reach out to this number within 24 hrs

Safety, Pharmacokinetics and Efficacy of Dupilumab in Patients ≥6 Months to <6 Years With Moderate-to-Severe Atopic Dermatitis (Liberty AD PRESCHOOL) (Liberty AD)

Dermatitis, Atopic

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial