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| ID | Type | Description | Link |
|---|---|---|---|
| 2019-003247-30 | EudraCT Number |
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| Name | Class |
|---|---|
| ZonMw: The Netherlands Organisation for Health Research and Development | OTHER |
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The goal of this study is to investigate whether stratification of children with atopic dermatitis on the NMF biomarkers results in an improvement of effectiveness and efficiency in the use of systemic treatment (ciclosporin and dupilumab) in moderate-to-severe atopic dermatitis.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Topical corticosteroids (control) | Active Comparator | This group will receive topical corticosteroids. |
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| Systemic cyclosporine | Active Comparator | This group will receive topical corticosteroids and systemic cyclosporin. |
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| Systemic dupilumab | Active Comparator | his group will receive topical corticosteroids and systemic dupilumab. |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Topical corticosteroids | Drug | Topical corticosteroids (TCS) are registered for patients of all ages, and are together with emollients, the pillars in the basic treatment of atopic dermatitis. In this study, patients in both the intervention groups and control group are treated with daily emollients and a TCS of moderate to high potency if needed. Rescue medication with TCS of higher potency may be prescribed if basic therapy is inadequate in controlling AD symptoms. |
| Measure | Description | Time Frame |
|---|---|---|
| EASI | Change from baseline Eczema Area and Severity Index (0-72) over the course of 6 months, with higher scores meaning worse outcomes. | t = 0, 1 month, 2 months, 3 months and 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| EASI75 | Relative reduction of 75% from baseline EASI without the use of rescue medication | t = 1 month, 2 months, 3 months and 6 months |
| IGA 0 or IGA 1 | Proportion of patients that achieved IGA 0 or IGA 1 (Investigator's Global Assessment) without the use of rescue medication. |
| Measure | Description | Time Frame |
|---|---|---|
| CDLQI ≥4 years | Children's Dermatology Life Quality Index, in the context of a cost-effectiveness analysis | t = 0, 3 months and 6 months |
| IDQoL <4 years | Infants' Dermatitis Quality of Life Index, in the context of a cost-effectiveness analysis |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Suzanne G.M.A. Pasmans, Prof | Contact | +31 6 53524299 | s.pasmans@erasmusmc.nl |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Erasmus MC - Sophia Children's Hospital | Recruiting | Rotterdam | South Holland | Netherlands |
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| ID | Term |
|---|---|
| D003876 | Dermatitis, Atopic |
| ID | Term |
|---|---|
| D012873 | Skin Diseases, Genetic |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D003872 | Dermatitis |
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| ID | Term |
|---|---|
| D000305 | Adrenal Cortex Hormones |
| D016572 | Cyclosporine |
| C582203 | dupilumab |
| ID | Term |
|---|---|
| D006728 | Hormones |
| D006730 | Hormones, Hormone Substitutes, and Hormone Antagonists |
| D003524 | Cyclosporins |
| D010456 | Peptides, Cyclic |
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This is a partially-blinded randomized controlled trial that investigates if NMF-biomarker status has an effect on the effectiveness of systemic treatment with CsA in children with moderate-to-severe AD. It also investigates whether stratification on the NMF biomarker results in an improvement of efficiency in the use of systemic treatment with CsA in the treatment of moderate-to-severe AD.
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Personnel involved assessing disease severity are blinded for the NMF biomarker result.
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| Systemic cyclosporine | Drug | Systemic cyclosporine A (CsA) is an immunosuppressive therapy and is a registered treatment for AD in adults. According to national guidelines, CsA is the first choice for systemic treatment in children with moderate-to-severe AD. For CsA a starting dose of 4-5mg/kg/day is administered orally and then tapered down to 2-3mg/kg/day depending on clinical effect. Two doses will be taken at two fixed times per day. Treatment with systemic CsA will be continued for a total of 6 months. |
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| Systemic dupilumab | Drug | Dupilumab (DUPIXENT) is indicated for the treatment of children of 6 years and older with moderate-to-sever atopic dermatitis whos disease is not adequately controlled with topical prescription therapies or when those therapies are not advisable. DUPIXENT can be used with or without topical corticosteroids. rm: Active Comparator: Systemic dupilumab Dupilumab (DUPIXENT) is administered as a solution by subdermal injection according to national guidelines, based on age and body weight. |
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| t = 0, 1 month, 2 months, 3 months and 6 months |
| NRS-11 reduction for itch ≥ 4 points | Proportion of patients that achieved a reduction ≥4 points on the Numeric Rating Scale-11 (0-10) for itch intensity. | t = 0, 1 month, 2 months, 3 months and 6 months |
| POEM | Change from baseline in Patient-Oriented Eczema Measure questionnaire (0-28) over the course of 6 months, with higher scores meaning worse outcomes. | t = 0, 1 month, 2 months, 3 months and 6 months |
| SCORAD | Change from baseline in Scoring Atopic Dermatitis scale (0-103) over the course of 6 months, with higher scores meaning worse outcomes. | t = 0, 1 month, 2 months, 3 months and 6 months |
| RECAP | Change from baseline in the Recap of Atopic Eczema questionnaire (0-28) over the course of 6 months, with higher scores meaning worse outcomes. | t = 0, 1 month, 2 months, 3 months and 6 months |
| t = 0, 3 months and 6 months |
| Emollients and steroid use in frequency and tubes used | In context of a cost-effectiveness analysis: To assess the use of topical medication, including emollients, expressed in number of grams and/or used tubes, and changes therein during systemic treatment. | t = 0, 1 month, 2 months, 3 months, 4 months, 5 months and 6 months |
| Healthcare costs related to the treatment of AD | In context of a cost-effectiveness analysis: To assess medical specialist care, hospitalization, medication, and other costs directly associated with the treatment and recurrence. | Over the course of 6 months |
| Adverse events | Adverse events related to therapy as reported at any time during treatment by patient, custodian or investigator. | Over the course of 6 months |
| NMF measured via Raman spectroscopy | Natural Moisturizing Factor, to acquire more knowledge about external and internal factors that influence the NMF biomarker | t = - 2 weeks, 0, 3 months and 6 months |
| Microbiome profile | To investigate differences in microbiome profiles between patients with normal vs low NMF, and to investigate changes from baseline in microbiome profile during treatment, periodic swabs of nose, lesional skin, non-lesional skin and faeces will be obtained from patients. | t = 0, 3 months and 6 months |
| Humoral blood panel (systemic arms) | Changes in IgE during systemic treatment over the course of 6 months. | t = 0, 1 month, 3 months and 6 months |
| Humoral blood panel (topical arm) | Changes in IgE during topical treatment over the course of 6 months. | t = 0 and 6 months |
| Cellular blood panel (systemic arm) | Changes in leucocyte differentiation during systemic treatment over the course of 6 months. | t = 0, 1 month, 3 months and 6 months |
| FLG null mutations | Genotyping on skin barrier proteins, to acquire more knowledge about external and internal factors that influence atopic dermatitis and the NMF biomarker | t = 0 |
| Activity of atopy | The activity of rhinoconjunctivitis, asthma and food allergy examined by a pediatric allergist and pediatric pulmonologist. | t = 0, 3 months and 6 months |
| Psychosocial factors (CBCL) | To investigate the influence of psychosocial factors in the patient on pediatric atopic dermatitis as assessed by the CBCL (Child Behaviour Checklist). Patients are assessed by questions grouped in empirically based syndrome scales: Aggressive Behavior, Anxious/Depressed, Attention Problems, Rule-Breaking Behavior, Somatic Complaints, Social Problems, Thought Problems, Withdrawn/Depressed. Higher percentile scores per scale indicate worse outcomes. | t = 0 |
| Psychosocial factors (OBVL) | To investigate the influence of psychosocial factors in the family on pediatric atopic dermatitis as assessed by the OBVL (OpvoedingsBelastingVragenLijst / Parenting Stress Questionnaire), with higher percentile scores indicating worse outcomes | t = 0 |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D017443 | Skin Diseases, Eczematous |
| D006969 | Hypersensitivity, Immediate |
| D006967 | Hypersensitivity |
| D007154 | Immune System Diseases |
| D047028 | Macrocyclic Compounds |
| D011083 | Polycyclic Compounds |
| D010455 | Peptides |
| D000602 | Amino Acids, Peptides, and Proteins |