Dermatitis, AtopicPhase 2/3Liberty ADNCT03346434
Safety, Pharmacokinetics and Efficacy of Dupilumab in Patients ≥6 Months to <6 Years With Moderate-to-Severe Atopic Dermatitis (Liberty AD PRESCHOOL)
A Phase 2/3 study testing Dupilumab in people with Dermatitis, Atopic, run by Regeneron Pharmaceuticals at 48 sites in 4 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 6 months to 5 years
- Conditions the study is for
- Dermatitis, Atopic
- What is being tested?
- Dupilumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 202 participants
- Where
- 48 sitesin 4 countries
- Study dates
- 30 Nov 2017to 8 Jul 2021
- Phase
- Phase 2/3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 5 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Show all 14Show fewer
- 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).
Who cannot join
- 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
Show all 11Show fewer
- 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
Trial locations and contacts
48 sites in 4 countries.
48 sites in 4 countries
United States33 sites
Regeneron Investigational Site
Ann Arbor, Michigan, 48109-5314
Regeneron Investigational Site
Austin, Texas, 78723
Regeneron Investigational Site
Birmingham, Alabama, 35209
Regeneron Investigational Site
Boston, Massachusetts, 02115
Regeneron Investigational Site
Charleston, South Carolina, 29425
Regeneron Investigational Site
Chicago, Illinois, 60611
Regeneron Investigational Site
Columbus, Georgia, 31904
Regeneron Investigational Site
Coral Gables, Florida, 33146
Regeneron Investigational Site
Forest Hills, New York, 113756
Regeneron Investigational Site
Gilbert, Arizona, 85234
Regeneron Investigational Site
Hackensack, New Jersey, 07601
Regeneron Investigational Site
Lincoln, Nebraska, 68505
Regeneron Investigational Site
Long Beach, California, 90808
Regeneron Investigational site
Los Angeles, California, 90027
Regeneron Investigational Site
Macon, Georgia, 31217
Regeneron Investigational Site
Milwaukee, Wisconsin, 53226
Regeneron Investigational Site
New York, New York, 10032
Regeneron Investigational Site
Norfolk, Virginia, 23502
Regeneron Investigational Site
North Charleston, South Carolina, 29420
Regeneron Investigational Site
Palo Alto, California, 94304
Regeneron Investigational Site
Philadelphia, Pennsylvania, 19104
Regeneron Investigational Site
Portland, Oregon, 97239
Regeneron Investigational Site
Rochester, New York, 14642
Regeneron Investigational Site
Rockville, Maryland, 20850
Regeneron Investigational Site
Rolling Hills Estates, California, 90274
Regeneron Investigational Site
San Antonio, Texas, 78218
Regeneron Investigational Site
San Diego, California, 92123
Regeneron Investigational Site
Sandy Springs, Georgia, 39328
Regeneron Investigational Site
Seattle, Washington, 98105
Regeneron Investigational Site
St Louis, Missouri, 63104-1003
Regeneron Investigational Site
Tampa, Florida, 33612-3807
Regeneron Investigational Site
Washington D.C., District of Columbia, 20010
Regeneron Investigational Site
Ypsilanti, Michigan, 48197
Germany7 sites
Regeneron Investigational Site
Dresden, Saxony, 01307
Regeneron Investigational site
Frankfurt am Main, 60590
Regeneron Investigational Site
Kiel, 24105
Regeneron Investigational Site
München, 80337
Regeneron Investigational site
München, 80802
Regeneron Investigational site
Münster, North Rhine-Westphal, 48149
Regeneron Investigational Site
Osnabrück, Lower Saxony, 49074
Poland6 sites
Regeneron Investigational Site
Bialystok, 15-453
Regeneron Investigational Site
Katowice, 40-648
Regeneron Investigational Site
Krakow, 31-011
Regeneron Investigational Site
Ostrowiec Świętokrzyski, 27-400
Regeneron Investigational Site
Warsaw, 01-142
Regeneron Investigational Site
Warsaw, 01-817
United Kingdom2 sites
Regeneron Investigational Site
Manchester, Lancashire, M13 9WL
Regeneron Investigational Site
Sheffield, South Yorkshire, S10 2TH
About this study
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
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).
Read the 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.
- Conditions
- Dermatitis, Atopic
- Being tested
- Dupilumab, Matching placebo
- Keywords
- Eczema
- Medical subject headings
- Dermatitis, Atopic, Eczema
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DupilumabDrug
Also called DUPIXENT®, REGN668, SAR231893
Solution for injection, subcutaneous (SC)
Matching placeboDrug
Solution for injection, subcutaneous (SC)
| Study group | Role | What they receive |
|---|---|---|
| Part A (Open label Dupilumab): Age cohorts 1 & 2 | Experimental | Age cohort 1: ≥2 years old to <6 years old
Age cohort 2: ≥6 months to <2 years old Receives: Drug: Dupilumab |
| Part B (Double-blind): Dupilumab dose 1 | Experimental | The results of part A will be used to guide the selection of dose levels and dosing frequency for part B. Receives: Drug: Dupilumab |
| Part B (Double-blind): Dupilumab dose 2 | Experimental | The results of part A will be used to guide the selection of dose levels and dosing frequency for part B. Receives: Drug: Dupilumab |
| Part B (Double-Blind): Placebo | Experimental | Not described Receives: Drug: Matching placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part A: Maximum Observed Serum Concentration (Cmax) of Functional Dupilumab
Serum concentration of functional dupilumab was reported.
Measured: Post-dose on Days 1, 3, 8, 18, and 29
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]).
Measured: Post-dose on Days 1, 3, 8, 18, and 29
Part A: Time to Reach the Maximum Serum Concentration (Tmax) of Dupilumab
Tmax was obtained directly from the concentration versus time curve.
Measured: Post-dose on Days 1, 3, 8, 18, and 29
Part A: Last Quantifiable Serum Concentration (Clast) of Dupilumab
Clast is the last measurable serum concentration of dupilumab.
Measured: Post-dose on Days 1, 3, 8, 18, and 29
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.
Measured: Post-dose on Days 1, 3, 8, 18, and 29
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.
Measured: Post-dose on Days 1, 3, 8, 18, and 29
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.
Measured: Post-dose on Days 1, 3, 8, 18, and 29
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.
Measured: Baseline up to Week 4
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.
Measured: Baseline up to Week 4
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.
Measured: Week 16
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.
Measured: Week 16
Secondary outcomes (26)
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.
Measured: Baseline up to Week 4
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.
Measured: Week 4
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.
Measured: Week 4
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.
Measured: Week 4
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.
Measured: Baseline up to Day 57
Part B: Number of Participants With at Least One Serious Adverse Event (SAE) Through Week 16
Measured: Baseline 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
Measured: Baseline 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.
Measured: Baseline up to Day 197
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.
Measured: Week 16
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.
Measured: Week 16
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.
Measured: Week 16
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.
Measured: Week 16
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.
Measured: 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.
Measured: 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.
Measured: Week 16
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.
Measured: Week 16
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.
Measured: Week 16
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.
Measured: Week 16
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.
Measured: Week 16
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.
Measured: Week 16
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.
Measured: Week 16
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.
Measured: Week 16
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.
Measured: Baseline up to Week 16
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.
Measured: Baseline up to Week 16
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.
Measured: Baseline up to Week 16
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.
Measured: Baseline through Week 16
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Nov 2017
- Study start30 Nov 2017
- Primary completion8 Jul 2021
- Study completion8 Jul 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (13)Papers the sponsor has linked to this study.
- Other trials for Dermatitis, AtopicStudies of the same condition, open and under way.
- More trials run by Regeneron PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Model description
- Part A: Single-ascending-dose cohorts staggered by age; Part B: Parallel Group
- Masking
- None (open label)
- Enrolment
- 202 participants (actual)
Sponsor and investigators
- Lead sponsor
- Regeneron Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Sanofi
- Responsible party
- Sponsor
- Investigators
- Clinical Trial Management, Study director, Regeneron Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03346434
- Sponsor's protocol ID
- R668-AD-1539
- Other IDs
- 2016-000955-28, 2016
- Acronym
- Liberty AD
- Status last verified
- Jul 2022
- First submitted
- 6 Feb 2017
- Last updated
- 28 Jul 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Boguniewicz M, Sher LD, Paller AS, Arkwright PD, Yoshihara S, Smith Begolka W, Chen Z, Shah P, Rodriguez Marco A. Dupilumab is Effective in Young Children with Atopic Dermatitis Regardless of Type 2 Comorbidities. J Asthma Allergy. 2026 May 19;19:590176. doi: 10.2147/JAA.S590176. eCollection 2026.
PubMed 42266404Paller AS, Pinter A, Wine Lee L, Aschoff R, Zdybski J, Schnopp C, Khokhar FA, Praestgaard AH, Bansal A, Shumel B, Bastian M. Long-Term Safety and Efficacy of Dupilumab Treatment in Children Aged 6 Months to 5 Years with Severe Atopic Dermatitis. Paediatr Drugs. 2026 May;28(3):307-320. doi: 10.1007/s40272-026-00747-4. Epub 2026 Apr 2.
PubMed 41926052Langley RG, Gherardi G, Coleman A, Ardeleanu M, Rodriguez-Marco A, Levy S, Bansal A, Chen Z, Rossi AB, Shumel B, Khokhar FA. The Safety Data of Dupilumab for the Treatment of Moderate-to-Severe Atopic Dermatitis in Infants, Children, Adolescents, and Adults. Am J Clin Dermatol. 2025 Nov;26(6):981-1002. doi: 10.1007/s40257-025-00952-w. Epub 2025 Sep 24.
PubMed 40993471Rossi AB, Mello AM, Zahn J. Dupilumab Efficacy in Children with Atopic Dermatitis with Different Phenotypes and Endotypes: A Case Series. Adv Ther. 2025 Jul;42(7):3186-3206. doi: 10.1007/s12325-025-03150-6. Epub 2025 May 8.
PubMed 40338484Kamal MA, Kosloski MP, Lai CH, Partridge MA, Rajadhyaksha M, Kanamaluru V, Bansal A, Shabbir A, Shumel B, Ardeleanu M, Richards SM, Yan H, Xu CR, Rodriguez-Marco A, Xiao J, Khokhar FA, Gherardi G, Babilonia E, Maloney J, Mortensen E, Akinlade B, Braunstein N, Stahl N, Torri A, Davis JD, DiCioccio AT. Immunogenicity of dupilumab in adult and pediatric patients with atopic dermatitis. Front Immunol. 2024 Nov 11;15:1466372. doi: 10.3389/fimmu.2024.1466372. eCollection 2024.
PubMed 39588375Boguniewicz M, Sher LD, Paller AS, Arkwright PD, Yoshihara S, Chen Z, Shah P, Marco AR. Dupilumab is Efficacious in Young Children with Atopic Dermatitis Regardless of Type 2 Comorbidities. Adv Ther. 2024 Dec;41(12):4601-4616. doi: 10.1007/s12325-024-02998-4. Epub 2024 Oct 29.
PubMed 39470878Paller AS, Siegfried EC, Simpson EL, Cork MJ, Sidbury R, Chen IH, Khokhar FA, Xiao J, Dubost-Brama A, Bansal A. Dupilumab Safety and Efficacy up to 1 Year in Children Aged 6 Months to 5 Years with Atopic Dermatitis: Results from a Phase 3 Open-Label Extension Study. Am J Clin Dermatol. 2024 Jul;25(4):655-668. doi: 10.1007/s40257-024-00859-y. Epub 2024 May 14.
PubMed 38743155Paller AS, Siegfried EC, Cork MJ, Arkwright PD, Eichenfield LF, Ramien M, Khokhar FA, Chen Z, Zhang A, Cyr SL. Infections in Children Aged 6 Months to 5 Years Treated with Dupilumab in a Placebo-Controlled Clinical Trial of Moderate-to-Severe Atopic Dermatitis. Paediatr Drugs. 2024 Mar;26(2):163-173. doi: 10.1007/s40272-023-00611-9. Epub 2024 Jan 24.
PubMed 38267692Paller AS, Pinter A, Wine Lee L, Aschoff R, Zdybski J, Schnopp C, Praestgaard A, Bansal A, Shumel B, Prescilla R, Bastian M. Efficacy and Safety of Dupilumab Treatment with Concomitant Topical Corticosteroids in Children Aged 6 Months to 5 Years with Severe Atopic Dermatitis. Adv Ther. 2024 Mar;41(3):1046-1061. doi: 10.1007/s12325-023-02753-1. Epub 2024 Jan 9.
PubMed 38194047Siegfried EC, Simpson EL, Cork MJ, Arkwright PD, Wine Lee L, Chen Z, Prescilla R, Bansal A, Levit NA, Rodriguez Marco A. Dupilumab Treatment Leads to Rapid and Consistent Improvement of Atopic Dermatitis in All Anatomical Regions in Patients Aged 6 Months to 5 Years. Dermatol Ther (Heidelb). 2023 Sep;13(9):1987-2000. doi: 10.1007/s13555-023-00960-w. Epub 2023 Jul 22.
PubMed 37480432Yang N, Ye Y, Shao J, Wu H, Xu Q, Zhu J, Liu J, Li Z. Efficacy of Dupilumab in Children 6 Months to 11 Years Old With Atopic Dermatitis: A Retrospective Real-World Study in China. Dermatitis. 2024 Jan-Feb;35(S1):S39-S46. doi: 10.1089/derm.2022.0069. Epub 2023 Feb 17.
PubMed 36800177Paller AS, Siegfried EC, Cork MJ, Wollenberg A, Arkwright PD, Gonzalez ME, Lockshin B, Chen Z, Bansal A, Levit NA, Prescilla R. Laboratory Safety from a Randomized 16-Week Phase III Study of Dupilumab in Children Aged 6 Months to 5 Years with Moderate-to-Severe Atopic Dermatitis. Paediatr Drugs. 2023 Jan;25(1):67-77. doi: 10.1007/s40272-022-00553-8. Epub 2022 Dec 19.
PubMed 36529811Paller 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.
PubMed 36116481
Study at a glance
From the registry record, last updated by the sponsor on 28 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03346434
- Acronym / sponsor ID
- Liberty AD / R668-AD-1539
- Conditions
- Dermatitis, Atopic
- Keywords
- Eczema
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 202 participants (actual)
- Lead sponsor
- Regeneron PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- Sanofi
- Study start
- 30 Nov 2017 (actual)
- Primary completion
- 8 Jul 2021 (actual)
- Study completion
- 8 Jul 2021 (actual)
- First posted
- 17 Nov 2017 (actual)
- Results
- Posted 28 Jul 2022
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 4
- Model description
- Part A: Single-ascending-dose cohorts staggered by age; Part B: Parallel Group
| Arm | Type | Receives |
|---|---|---|
| Part A (Open label Dupilumab): Age cohorts 1 & 2 | Experimental | Drug: DupilumabAge cohort 1: ≥2 years old to <6 years old Age cohort 2: ≥6 months to <2 years old |
| Part B (Double-blind): Dupilumab dose 1 | Experimental | Drug: DupilumabThe results of part A will be used to guide the selection of dose levels and dosing frequency for part B. |
| Part B (Double-blind): Dupilumab dose 2 | Experimental | Drug: DupilumabThe results of part A will be used to guide the selection of dose levels and dosing frequency for part B. |
| Part B (Double-Blind): Placebo | Experimental | Drug: Matching placebo |
Who may qualify
- Ages
- 6 months to 5 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (14 listed)
- 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
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DupilumabAlso: DUPIXENT®, REGN668, SAR231893Solution for injection, subcutaneous (SC) | Drug | Part A (Open label Dupilumab): Age cohorts 1 & 2, Part B (Double-blind): Dupilumab dose 1, Part B (Double-blind): Dupilumab dose 2 |
| Matching placeboSolution for injection, subcutaneous (SC) | Drug | Part B (Double-Blind): Placebo |
Primary outcome
Part A: Maximum Observed Serum Concentration (Cmax) of Functional Dupilumab
Time frame: Post-dose on Days 1, 3, 8, 18, and 29
Serum concentration of functional dupilumab was reported.
Part A: Dose Normalized Maximum Observed Serum Concentration (Cmax/Dose) of Dupilumab
Time frame: Post-dose on Days 1, 3, 8, 18, and 29
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
Time frame: Post-dose on Days 1, 3, 8, 18, and 29
Tmax was obtained directly from the concentration versus time curve.
Part A: Last Quantifiable Serum Concentration (Clast) of Dupilumab
Time frame: Post-dose on Days 1, 3, 8, 18, and 29
Clast is the last measurable serum concentration of dupilumab.
Part A: Time of the Last Quantifiable Serum Concentration (Tlast) of Dupilumab
Time frame: Post-dose on Days 1, 3, 8, 18, and 29
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
Time frame: Post-dose on Days 1, 3, 8, 18, and 29
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
Time frame: Post-dose on Days 1, 3, 8, 18, and 29
Dose normalized AUClast was calculated by AUClast/dose.
Part A: Number of Participants With at Least One Treatment-Emergent Adverse Event (TEAE)
Time frame: Baseline up to Week 4
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
Time frame: Baseline up to Week 4
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
Time frame: 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
Time frame: 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.
26 secondary outcomes
Part A: Number of Participants With Serious TEAEs and Severe TEAEs
Time frame: Baseline up to Week 4
Part A: Percent Change From Baseline in EASI Score at Week 4
Time frame: Week 4
Part A: Percent Change From Baseline in SCORing Atopic Dermatitis (SCORAD) Score at Week 4
Time frame: Week 4
Part A: Percentage of Participants With IGA Score 0 or 1 at Week 4
Time frame: Week 4
Part A: Number of Participants With at Least One Positive Treatment-Emergent Anti-Drug Antibodies (ADA)
Time frame: Baseline up to Day 57
Part B: Number of Participants With at Least One Serious Adverse Event (SAE) Through Week 16
Time frame: Baseline 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
Time frame: Baseline through Week 16
Part B: Number of Participants With at Least One Positive Treatment-Emergent ADA
Time frame: Baseline up to Day 197
Part B: Percent Change From Baseline in EASI Score at Week 16
Time frame: Week 16
Part B: Percent Change From Baseline in Weekly Average of Daily Worst Scratch/Itch/Numerical Rating Scale (NRS) at Week 16
Time frame: Week 16
Part B: Percentage of Participants With Improvement (Reduction From Baseline) of Weekly Average of Daily Worst Scratch/Itch/NRS ≥4 Points at Week 16
Time frame: Week 16
Part B: Percentage of Participants With Improvement (Reduction From Baseline) of Weekly Average of Daily Worst Scratch/Itch/NRS ≥3 Points at Week 16
Time frame: Week 16
Part B: Percentage of Participants Who Achieved EASI-50 (≥50% Improvement From Baseline) at Week 16
Time frame: Week 16
Part B: Percentage of Participants Who Achieved EASI-90 (≥90% Improvement From Baseline) at Week 16
Time frame: Week 16
Part B: Change From Baseline in Percent Body Surface Area (BSA) Affected by Atopic Dermatitis (AD) at Week 16
Time frame: Week 16
Part B: Change From Baseline in Patient Oriented Eczema Measure (POEM) at Week 16
Time frame: Week 16
Part B: Percent Change From Baseline in SCORing Atopic Dermatitis (SCORAD) at Week 16
Time frame: Week 16
Part B: Change From Baseline in Participant's Sleep Quality NRS at Week 16
Time frame: Week 16
Part B: Change From Baseline in Participant's Skin Pain NRS at Week 16
Time frame: Week 16
Part B: Change From Baseline in Dermatitis Family Index (DFI) at Week 16
Time frame: Week 16
Part B: Change From Baseline in Children's Dermatology Life Quality Index (CDLQI) at Week 16
Time frame: Week 16
Part B: Change From Baseline in Infants' Dermatology Quality of Life Index (IDQOL) at Week 16
Time frame: Week 16
Part B: Percentage of Topical Corticosteroid (TCS) Medication-free Days From Baseline to Week 16
Time frame: Baseline up to Week 16
Part B: Mean Weekly Dose of Low Potency TCS in Grams From Baseline to Week 16
Time frame: Baseline up to Week 16
Part B: Mean Weekly Dose of TCS in Grams for Medium or High Potency TCS From Baseline to Week 16
Time frame: Baseline up to Week 16
Part B: Mean Number of Caregiver Missed Work Days Through Week 16
Time frame: Baseline through Week 16
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Jul 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 42266404Boguniewicz M, Sher LD, Paller AS, Arkwright PD, Yoshihara S, Smith Begolka W, Chen Z, Shah P, Rodriguez Marco A. Dupilumab is Effective in Young Children with Atopic Dermatitis Regardless of Type 2 Comorbidities. J Asthma Allergy. 2026 May 19;19:590176. doi: 10.2147/JAA.S590176. eCollection 2026.
- Derived from this study: PubMed 41926052Paller AS, Pinter A, Wine Lee L, Aschoff R, Zdybski J, Schnopp C, Khokhar FA, Praestgaard AH, Bansal A, Shumel B, Bastian M. Long-Term Safety and Efficacy of Dupilumab Treatment in Children Aged 6 Months to 5 Years with Severe Atopic Dermatitis. Paediatr Drugs. 2026 May;28(3):307-320. doi: 10.1007/s40272-026-00747-4. Epub 2026 Apr 2.
- Derived from this study: PubMed 40993471Langley RG, Gherardi G, Coleman A, Ardeleanu M, Rodriguez-Marco A, Levy S, Bansal A, Chen Z, Rossi AB, Shumel B, Khokhar FA. The Safety Data of Dupilumab for the Treatment of Moderate-to-Severe Atopic Dermatitis in Infants, Children, Adolescents, and Adults. Am J Clin Dermatol. 2025 Nov;26(6):981-1002. doi: 10.1007/s40257-025-00952-w. Epub 2025 Sep 24.
- Derived from this study: PubMed 40338484Rossi AB, Mello AM, Zahn J. Dupilumab Efficacy in Children with Atopic Dermatitis with Different Phenotypes and Endotypes: A Case Series. Adv Ther. 2025 Jul;42(7):3186-3206. doi: 10.1007/s12325-025-03150-6. Epub 2025 May 8.
- Derived from this study: PubMed 39588375Kamal MA, Kosloski MP, Lai CH, Partridge MA, Rajadhyaksha M, Kanamaluru V, Bansal A, Shabbir A, Shumel B, Ardeleanu M, Richards SM, Yan H, Xu CR, Rodriguez-Marco A, Xiao J, Khokhar FA, Gherardi G, Babilonia E, Maloney J, Mortensen E, Akinlade B, Braunstein N, Stahl N, Torri A, Davis JD, DiCioccio AT. Immunogenicity of dupilumab in adult and pediatric patients with atopic dermatitis. Front Immunol. 2024 Nov 11;15:1466372. doi: 10.3389/fimmu.2024.1466372. eCollection 2024.
- Derived from this study: PubMed 39470878Boguniewicz M, Sher LD, Paller AS, Arkwright PD, Yoshihara S, Chen Z, Shah P, Marco AR. Dupilumab is Efficacious in Young Children with Atopic Dermatitis Regardless of Type 2 Comorbidities. Adv Ther. 2024 Dec;41(12):4601-4616. doi: 10.1007/s12325-024-02998-4. Epub 2024 Oct 29.
- Derived from this study: PubMed 38743155Paller AS, Siegfried EC, Simpson EL, Cork MJ, Sidbury R, Chen IH, Khokhar FA, Xiao J, Dubost-Brama A, Bansal A. Dupilumab Safety and Efficacy up to 1 Year in Children Aged 6 Months to 5 Years with Atopic Dermatitis: Results from a Phase 3 Open-Label Extension Study. Am J Clin Dermatol. 2024 Jul;25(4):655-668. doi: 10.1007/s40257-024-00859-y. Epub 2024 May 14.
- Derived from this study: PubMed 38267692Paller AS, Siegfried EC, Cork MJ, Arkwright PD, Eichenfield LF, Ramien M, Khokhar FA, Chen Z, Zhang A, Cyr SL. Infections in Children Aged 6 Months to 5 Years Treated with Dupilumab in a Placebo-Controlled Clinical Trial of Moderate-to-Severe Atopic Dermatitis. Paediatr Drugs. 2024 Mar;26(2):163-173. doi: 10.1007/s40272-023-00611-9. Epub 2024 Jan 24.
- Derived from this study: PubMed 38194047Paller AS, Pinter A, Wine Lee L, Aschoff R, Zdybski J, Schnopp C, Praestgaard A, Bansal A, Shumel B, Prescilla R, Bastian M. Efficacy and Safety of Dupilumab Treatment with Concomitant Topical Corticosteroids in Children Aged 6 Months to 5 Years with Severe Atopic Dermatitis. Adv Ther. 2024 Mar;41(3):1046-1061. doi: 10.1007/s12325-023-02753-1. Epub 2024 Jan 9.
- Derived from this study: PubMed 37480432Siegfried EC, Simpson EL, Cork MJ, Arkwright PD, Wine Lee L, Chen Z, Prescilla R, Bansal A, Levit NA, Rodriguez Marco A. Dupilumab Treatment Leads to Rapid and Consistent Improvement of Atopic Dermatitis in All Anatomical Regions in Patients Aged 6 Months to 5 Years. Dermatol Ther (Heidelb). 2023 Sep;13(9):1987-2000. doi: 10.1007/s13555-023-00960-w. Epub 2023 Jul 22.
- Derived from this study: PubMed 36800177Yang N, Ye Y, Shao J, Wu H, Xu Q, Zhu J, Liu J, Li Z. Efficacy of Dupilumab in Children 6 Months to 11 Years Old With Atopic Dermatitis: A Retrospective Real-World Study in China. Dermatitis. 2024 Jan-Feb;35(S1):S39-S46. doi: 10.1089/derm.2022.0069. Epub 2023 Feb 17.
- Derived from this study: PubMed 36529811Paller AS, Siegfried EC, Cork MJ, Wollenberg A, Arkwright PD, Gonzalez ME, Lockshin B, Chen Z, Bansal A, Levit NA, Prescilla R. Laboratory Safety from a Randomized 16-Week Phase III Study of Dupilumab in Children Aged 6 Months to 5 Years with Moderate-to-Severe Atopic Dermatitis. Paediatr Drugs. 2023 Jan;25(1):67-77. doi: 10.1007/s40272-022-00553-8. Epub 2022 Dec 19.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trial Management study director
Regeneron Pharmaceuticals
48 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Dupilumab in people with Dermatitis, Atopic, run by Regeneron Pharmaceuticals at 48 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 48 sites in United States, Germany, Poland, United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Jul 2022). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Part A: Maximum Observed Serum Concentration (Cmax) of Functional Dupilumab (time frame: Post-dose on Days 1, 3, 8, 18, and 29). The study lists 11 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03346434, which also lists 13 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Jul 2022.