E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
Atopic dermatitis / Eczema |
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E.1.1.2 | Therapeutic area | Diseases [C] - Skin and Connective Tissue Diseases [C17] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10012438 |
E.1.2 | Term | Dermatitis atopic |
E.1.2 | System Organ Class | 10040785 - Skin and subcutaneous tissue disorders |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The primary objective of the study is to demonstrate the efficacy of dupilumab as a monotherapy in patients ≥12 years to <18 years of age with moderate-to-severe AD. |
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E.2.2 | Secondary objectives of the trial |
The secondary objective of the study is to assess the safety of dupilumab as a monotherapy in patients ≥12 years to <18 years of age with moderate-to-severe AD. |
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E.2.3 | Trial contains a sub-study | Yes |
E.2.3.1 | Full title, date and version of each sub-study and their related objectives |
The purpose of the genomic analyses is to identify genomic associations with clinical or biomarker response, other clinical outcome measures and possible AEs. |
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E.3 | Principal inclusion criteria |
• Male or female ≥12 to <18 years of age at time of screening visit • Diagnosis of AD according to the American Academy of Dermatology consensus criteria at screening visit • IGA ≥3 at screening and baseline visit • EASI ≥16 at the screening and baseline visit • Baseline Pruritus NRS average score for maximum itch intensity ≥4 • ≥10% BSA of AD involvement at the screening and baseline visits • With documented recent history (within 6 months before the screening visit) of inadequate response to topical AD medication(s) or for whom topical treatments is medically inadvisable |
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E.4 | Principal exclusion criteria |
• Participation in a prior dupilumab clinical study • Treatment with topical corticosteroids (TCS) or topical calcineurin inhibitors (TCI) within 2 weeks before the baseline visit • Having used immunosuppressive/immunomodulating drugs within 4 weeks before the baseline visit • Body weight <30 kg at baseline • 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 before the baseline visit • Diagnosed active endoparasitic infections or at high risk of these infections • Patient is female who is pregnant, breastfeeding, or planning to become pregnant or breastfeed during the study • Patient is female of childbearing potential and sexually active, who is unwilling to use adequate methods of contraception throughout the duration of the study and for 120 days after the last dose of study drug |
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E.5 End points |
E.5.1 | Primary end point(s) |
Proportion of participants with Investigator Global Assessment (IGA) 0 to 1 (on a 5-point scale) |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
- Proportion of participants with Eczema Area and Severity Index (EASI)-75 (≥75% improvement from baseline) - Percent change in EASI score - Percent change in weekly average of daily peak Pruritus Numerical Rating Scale (NRS) - Proportion of participants with improvement (reduction) of weekly average of daily peak Pruritus NRS ≥3 - Proportion of participants with improvement (reduction) of weekly average of daily peak Pruritus NRS ≥4 - Proportion of patients with EASI-50 (≥50% improvement from baseline) - Proportion of patients with EASI-90 (≥90% improvement from baseline) - Time to onset of effect on pruritus during the 16-week treatment period (≥3 point reduction of weekly average of peak Pruritus NRS from baseline - Time to onset of effect on pruritus during the 16-week treatment period (≥4 point reduction of weekly average of peak Pruritus NRS from baseline - Change in percent body surface area (BSA) affected by AD - Percent change in SCORing Atopic Dermatitis (SCORAD) - Change in Children's Dermatology Life Quality Index (CDLQI) - Change in Patient Oriented Eczema Measure (POEM) - Change in weekly average of daily peak Pruritus NRS - Percent change in weekly average of daily peak Pruritus NRS - Change in Hospital Anxiety and Depression Scale (HADS) - Proportion of participants with improvement (reduction) of weekly average of daily peak Pruritus NRS ≥4 - Incidence of skin-infection treatment-emergent adverse events (TEAEs) (excluding herpetic infections) - Incidence of serious TEAEs [Time Frame: Baseline to week 16]
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Yes |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | Yes |
E.6.13.1 | Other scope of the trial description |
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E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | Yes |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | Yes |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 3 |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | Yes |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 1 |
E.8.9.2 | In all countries concerned by the trial months | 2 |