Phase 3
Completed N=202
Safety, Pharmacokinetics and Efficacy of Dupilumab in Patients ≥6 Months to <6 Years With Moderate-to-Severe Atopic Dermatitis (Liberty AD PRESCHOOL)
Dermatitis, Atopic
Source: ClinicalTrials.gov NCT03346434 ↗
Enrolled (actual)
202
Serious AEs
3.0%
Results posted
Jul 2022
Primary outcomePrimary: Part A: Maximum Observed Serum Concentration (Cmax) of Functional Dupilumab — 25.2; 49.8; 20.1; 46.1 Milligrams per Liter (mg/L)
◆ Published Evidence
Emerging
10citations · ~5 / year
Dupilumab is Efficacious in Young Children with Atopic Dermatitis Regardless of Type 2 Comorbidities.
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).
Linked Publications (5)
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Dupilumab is Efficacious in Young Children with Atopic Dermatitis Regardless of Type 2 Comorbidities.
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Immunogenicity of dupilumab in adult and pediatric patients with atopic dermatitis.
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The Safety Data of Dupilumab for the Treatment of Moderate‑to‑Severe Atopic Dermatitis in Infants, Children, Adolescents, and Adults.
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Dupilumab Efficacy in Children with Atopic Dermatitis with Different Phenotypes and Endotypes: A Case Series.
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Long-Term Safety and Efficacy of Dupilumab Treatment in Children Aged 6 Months to 5 Years with Severe Atopic Dermatitis.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Part A: Maximum Observed Serum Concentration (Cmax) of Functional Dupilumab |
25.2; 49.8; 20.1; 46.1 | — |
| PRIMARY Part A: Dose Normalized Maximum Observed Serum Concentration (Cmax/Dose) of Dupilumab |
8.39; 8.30; 6.70; 7.68 | — |
| PRIMARY Part A: Time to Reach the Maximum Serum Concentration (Tmax) of Dupilumab |
1.97; 1.95; 2.10; 1.92 | — |
| PRIMARY Part A: Last Quantifiable Serum Concentration (Clast) of Dupilumab |
6.64; 6.14; 5.64; 15.1 | — |
| PRIMARY Part A: Time of the Last Quantifiable Serum Concentration (Tlast) of Dupilumab |
14.8; 26.5; 8.56; 16.0 | — |
| PRIMARY Part A: Area Under the Serum Concentration-Time Curve From Time Zero to the Time of the Last Measurable Concentration (AUClast) of Dupilumab |
198; 622; 123; 493 | — |
| PRIMARY 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 |
66.0; 104; 41.0; 82.1 | — |
| PRIMARY Part A: Number of Participants With at Least One Treatment-Emergent Adverse Event (TEAE) |
3; 2; 7; 7 | — |
| PRIMARY Part A: Number of Participants With TEAEs by Severity According to Qualitative Toxicity Scale |
1; 2; 4; 5; 2; 0 | — |
| PRIMARY Part B: Percentage of Participants With Investigator's Global Assessment (IGA) Score 0 or 1 at Week 16 |
3.9; 27.7 | < 0.0001 sig |
| PRIMARY Part B: Percentage of Participants With Eczema Area and Severity Index (EASI) -75 (EASI-75) (≥75% Improvement From Baseline) at Week 16 |
10.7; 53.0 | < 0.0001 sig |
| SECONDARY Part A: Number of Participants With Serious TEAEs and Severe TEAEs |
1; 0; 1; 0; 0; 0 | — |
| SECONDARY Part A: Percent Change From Baseline in EASI Score at Week 4 |
-26.6; -48.7; -22.4; -43.2 | — |
| SECONDARY Part A: Percent Change From Baseline in SCORing Atopic Dermatitis (SCORAD) Score at Week 4 |
-18.6; -31.9; -22.4; -28.1 | — |
| SECONDARY Part A: Percentage of Participants With IGA Score 0 or 1 at Week 4 |
10.0; 0.0; 10.0; 10.0 | — |
| SECONDARY Part A: Number of Participants With at Least One Positive Treatment-Emergent Anti-Drug Antibodies (ADA) |
0; 0; 1; 0; 5; 4 | — |
| SECONDARY Part B: Number of Participants With at Least One Serious Adverse Event (SAE) Through Week 16 |
4; 0 | — |
| SECONDARY Part B: Number of Participants With at Least One Skin Infection Treatment Emergent Adverse Event (TEAE) (Excluding Herpetic Infection) Through Week 16 |
19; 10 | — |
| SECONDARY Part B: Number of Participants With at Least One Positive Treatment-Emergent ADA |
0; 1 | — |
| SECONDARY Part B: Percent Change From Baseline in EASI Score at Week 16 |
-19.6; -70.0 | < 0.0001 sig |
| SECONDARY Part B: Percent Change From Baseline in Weekly Average of Daily Worst Scratch/Itch/Numerical Rating Scale (NRS) at Week 16 |
-2.2; -49.4 | < 0.0001 sig |
| SECONDARY Part B: Percentage of Participants With Improvement (Reduction From Baseline) of Weekly Average of Daily Worst Scratch/Itch/NRS ≥4 Points at Week 16 |
8.9; 48.1 | < 0.0001 sig |
| SECONDARY Part B: Percentage of Participants With Improvement (Reduction From Baseline) of Weekly Average of Daily Worst Scratch/Itch/NRS ≥3 Points at Week 16 |
9.9; 53.3 | < 0.0001 sig |
| SECONDARY Part B: Percentage of Participants Who Achieved EASI-50 (≥50% Improvement From Baseline) at Week 16 |
20.2; 68.7 | < 0.0001 sig |
| SECONDARY Part B: Percentage of Participants Who Achieved EASI-90 (≥90% Improvement From Baseline) at Week 16 |
2.8; 25.3 | = 0.0001 sig |
| SECONDARY Part B: Change From Baseline in Percent Body Surface Area (BSA) Affected by Atopic Dermatitis (AD) at Week 16 |
-10.74; -35.00 | < 0.0001 sig |
| SECONDARY Part B: Change From Baseline in Patient Oriented Eczema Measure (POEM) at Week 16 |
-3.8; -12.9 | < 0.0001 sig |
| SECONDARY Part B: Percent Change From Baseline in SCORing Atopic Dermatitis (SCORAD) at Week 16 |
-16.2; -54.7 | < 0.0001 sig |
| SECONDARY Part B: Change From Baseline in Participant's Sleep Quality NRS at Week 16 |
0.34; 2.04 | < 0.0001 sig |
| SECONDARY Part B: Change From Baseline in Participant's Skin Pain NRS at Week 16 |
-0.62; -3.93 | < 0.0001 sig |
| SECONDARY Part B: Change From Baseline in Dermatitis Family Index (DFI) at Week 16 |
-2.68; -10.48 | < 0.0001 sig |
| SECONDARY Part B: Change From Baseline in Children's Dermatology Life Quality Index (CDLQI) at Week 16 |
-2.5; -10.0 | < 0.0001 sig |
| SECONDARY Part B: Change From Baseline in Infants' Dermatology Quality of Life Index (IDQOL) at Week 16 |
-1.95; -10.91 | < 0.0001 sig |
| SECONDARY Part B: Percentage of Topical Corticosteroid (TCS) Medication-free Days From Baseline to Week 16 |
0.04; 0.21 | = 0.0015 sig |
| SECONDARY Part B: Mean Weekly Dose of Low Potency TCS in Grams From Baseline to Week 16 |
13.4; 10.5 | = 0.0997 |
| SECONDARY Part B: Mean Weekly Dose of TCS in Grams for Medium or High Potency TCS From Baseline to Week 16 |
6.1; 3.0 | — |
| SECONDARY Part B: Mean Number of Caregiver Missed Work Days Through Week 16 |
5.05; 2.49 | — |
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
Data sourced from ClinicalTrials.gov (NCT03346434) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.