NMF-CsA-Dupi Trial
2021年8月17日 更新者：Suzanne G.M.A. Pasmans、Erasmus Medical Center
Use of the NMF Biomarker as Predictive Diagnostic for Effective Use of Cyclosporine and Dupilumab in the Treatment of Atopic Dermatitis
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.
研究概览
研究类型
介入性
注册 (预期的)
318
阶段
- 不适用
联系人和位置
本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。
学习联系方式
- 姓名：Suzanne G.M.A. Pasmans, Prof
- 电话号码：+31 6 53524299
- 邮箱：s.pasmans@erasmusmc.nl
学习地点
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Zuid-Holland
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Rotterdam、Zuid-Holland、荷兰
- 招聘中
- Erasmus MC - Sophia Children's Hospital
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接触：
- prof. S.G.M.A. Pasmans, MD PhD
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参与标准
研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。
资格标准
适合学习的年龄
2年 至 18年 (孩子、成人)
接受健康志愿者
不
有资格学习的性别
全部
描述
Inclusion Criteria:
- Children and adolescents, aged 2-18 years, with moderate-to-severe atopic dermatitis (diagnosed according to the UK working party criteria)
- Patient and parents/guardians able to participate in the study and willing to give written informed consent
- EASI (Eczema Area Severity Index) ≥ 6 at screening and baseline (corresponding with moderate-to-severe disease)
- IGA (Investigator Global Assessment) ≥ 3 at screening and baseline (corresponding with moderate-to-severe disease)
Exclusion Criteria:
- Children under the age of 2 years and patients older than 18 years
- Contraindication for ciclosporin
- Contraindication for dupilumab
- Patient (or one of the parents/guardians) not willing to be randomized
- Children with a history of any known primary immunodeficiency disorder
- Children with a history of cancer
- EASI < 6 at screening or baseline
- IGA < 3 at screening or baseline
学习计划
本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。
研究是如何设计的？
设计细节
- 主要用途：治疗
- 分配：随机化
- 介入模型：并行分配
- 屏蔽：单身的
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
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有源比较器：Topical corticosteroids (control)
This group will receive topical corticosteroids.
|
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.
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有源比较器：Systemic cyclosporine
This group will receive topical corticosteroids and systemic cyclosporin.
|
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.
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.
其他名称：
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有源比较器：Systemic dupilumab
his group will receive topical corticosteroids and systemic dupilumab.
|
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.
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.
其他名称：
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研究衡量的是什么？
主要结果指标
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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EASI
大体时间：t = 0, 1 month, 2 months, 3 months and 6 months
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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
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次要结果测量
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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EASI75
大体时间：t = 1 month, 2 months, 3 months and 6 months
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Relative reduction of 75% from baseline EASI without the use of rescue medication
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t = 1 month, 2 months, 3 months and 6 months
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IGA 0 or IGA 1
大体时间：t = 0, 1 month, 2 months, 3 months and 6 months
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Proportion of patients that achieved IGA 0 or IGA 1 (Investigator's Global Assessment) without the use of rescue medication.
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t = 0, 1 month, 2 months, 3 months and 6 months
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NRS-11 reduction for itch ≥ 4 points
大体时间：t = 0, 1 month, 2 months, 3 months and 6 months
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Proportion of patients that achieved a reduction ≥4 points on the Numeric Rating Scale-11 (0-10) for itch intensity.
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t = 0, 1 month, 2 months, 3 months and 6 months
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POEM
大体时间：t = 0, 1 month, 2 months, 3 months and 6 months
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Change from baseline in Patient-Oriented Eczema Measure questionnaire (0-28) over the course of 6 months, with higher scores meaning worse outcomes.
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t = 0, 1 month, 2 months, 3 months and 6 months
|
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SCORAD
大体时间：t = 0, 1 month, 2 months, 3 months and 6 months
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Change from baseline in Scoring Atopic Dermatitis scale (0-103) over the course of 6 months, with higher scores meaning worse outcomes.
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t = 0, 1 month, 2 months, 3 months and 6 months
|
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RECAP
大体时间：t = 0, 1 month, 2 months, 3 months and 6 months
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Change from baseline in the Recap of Atopic Eczema questionnaire (0-28) over the course of 6 months, with higher scores meaning worse outcomes.
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t = 0, 1 month, 2 months, 3 months and 6 months
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其他结果措施
结果测量 |
措施说明 |
大体时间 |
|---|---|---|
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CDLQI ≥4 years
大体时间：t = 0, 3 months and 6 months
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Children's Dermatology Life Quality Index, in the context of a cost-effectiveness analysis
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t = 0, 3 months and 6 months
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IDQoL <4 years
大体时间：t = 0, 3 months and 6 months
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Infants' Dermatitis Quality of Life Index, in the context of a cost-effectiveness analysis
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t = 0, 3 months and 6 months
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Emollients and steroid use in frequency and tubes used
大体时间：t = 0, 1 month, 2 months, 3 months, 4 months, 5 months and 6 months
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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.
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t = 0, 1 month, 2 months, 3 months, 4 months, 5 months and 6 months
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Healthcare costs related to the treatment of AD
大体时间：Over the course of 6 months
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In context of a cost-effectiveness analysis: To assess medical specialist care, hospitalization, medication, and other costs directly associated with the treatment and recurrence.
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Over the course of 6 months
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Adverse events
大体时间：Over the course of 6 months
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Adverse events related to therapy as reported at any time during treatment by patient, custodian or investigator.
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Over the course of 6 months
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NMF measured via Raman spectroscopy
大体时间：t = - 2 weeks, 0, 3 months and 6 months
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Natural Moisturizing Factor, to acquire more knowledge about external and internal factors that influence the NMF biomarker
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t = - 2 weeks, 0, 3 months and 6 months
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Microbiome profile
大体时间：t = 0, 3 months and 6 months
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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.
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t = 0, 3 months and 6 months
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Humoral blood panel (systemic arms)
大体时间：t = 0, 1 month, 3 months and 6 months
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Changes in IgE during systemic treatment over the course of 6 months.
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t = 0, 1 month, 3 months and 6 months
|
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Humoral blood panel (topical arm)
大体时间：t = 0 and 6 months
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Changes in IgE during topical treatment over the course of 6 months.
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t = 0 and 6 months
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Cellular blood panel (systemic arm)
大体时间：t = 0, 1 month, 3 months and 6 months
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Changes in leucocyte differentiation during systemic treatment over the course of 6 months.
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t = 0, 1 month, 3 months and 6 months
|
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FLG null mutations
大体时间：t = 0
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Genotyping on skin barrier proteins, to acquire more knowledge about external and internal factors that influence atopic dermatitis and the NMF biomarker
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t = 0
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Activity of atopy
大体时间：t = 0, 3 months and 6 months
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The activity of rhinoconjunctivitis, asthma and food allergy examined by a pediatric allergist and pediatric pulmonologist.
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t = 0, 3 months and 6 months
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Psychosocial factors (CBCL)
大体时间：t = 0
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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.
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t = 0
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Psychosocial factors (OBVL)
大体时间：t = 0
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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
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t = 0
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合作者和调查者
在这里您可以找到参与这项研究的人员和组织。
研究记录日期
这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。
研究主要日期
学习开始 (实际的)
2021年8月16日
初级完成 (预期的)
2024年1月1日
研究完成 (预期的)
2025年1月1日
研究注册日期
首次提交
2021年4月29日
首先提交符合 QC 标准的
2021年5月6日
首次发布 (实际的)
2021年5月7日
研究记录更新
最后更新发布 (实际的)
2021年8月18日
上次提交的符合 QC 标准的更新
2021年8月17日
最后验证
2021年8月1日
更多信息
与本研究相关的术语
其他相关的 MeSH 术语
其他研究编号
- MEC-2019-0568
- 2019-003247-30 (EudraCT编号)
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