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NMF-CsA-Dupi Trial

17 août 2021 mis à jour par: 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.

Aperçu de l'étude

Statut

Recrutement

Les conditions

Intervention / Traitement

Type d'étude

Interventionnel

Inscription (Anticipé)

318

Phase

  • N'est pas applicable

Contacts et emplacements

Cette section fournit les coordonnées de ceux qui mènent l'étude et des informations sur le lieu où cette étude est menée.

Coordonnées de l'étude

Lieux d'étude

  • Pays-Bas
    • Zuid-Holland
      • Rotterdam, Zuid-Holland, Pays-Bas
        • Recrutement
        • Erasmus MC - Sophia Children's Hospital
        • Contact:
          • prof. S.G.M.A. Pasmans, MD PhD

Critères de participation

Les chercheurs recherchent des personnes qui correspondent à une certaine description, appelée critères d'éligibilité. Certains exemples de ces critères sont l'état de santé général d'une personne ou des traitements antérieurs.

Critère d'éligibilité

Âges éligibles pour étudier

2 ans à 18 ans (Enfant, Adulte)

Accepte les volontaires sains

Non

Sexes éligibles pour l'étude

Tout

La description

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

Plan d'étude

Cette section fournit des détails sur le plan d'étude, y compris la façon dont l'étude est conçue et ce que l'étude mesure.

Comment l'étude est-elle conçue ?

Détails de conception

  • Objectif principal: Traitement
  • Répartition: Randomisé
  • Modèle interventionnel: Affectation parallèle
  • Masquage: Seul

Armes et Interventions

Groupe de participants / Bras
Intervention / Traitement
Comparateur actif: Topical corticosteroids (control)
This group will receive topical corticosteroids.
Médicament: 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.
Comparateur actif: Systemic cyclosporine
This group will receive topical corticosteroids and systemic cyclosporin.
Médicament: 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.
Médicament: Systemic cyclosporine

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.

Autres noms:
  • Néoral
Comparateur actif: Systemic dupilumab
his group will receive topical corticosteroids and systemic dupilumab.
Médicament: 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.
Médicament: Systemic dupilumab
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.
Autres noms:
  • DUPIXENT

Que mesure l'étude ?

Principaux critères de jugement

Mesure des résultats
Description de la mesure
Délai
EASI
Délai: t = 0, 1 month, 2 months, 3 months and 6 months
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

Mesures de résultats secondaires

Mesure des résultats
Description de la mesure
Délai
EASI75
Délai: t = 1 month, 2 months, 3 months and 6 months
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
Délai: t = 0, 1 month, 2 months, 3 months and 6 months
Proportion of patients that achieved IGA 0 or IGA 1 (Investigator's Global Assessment) without the use of rescue medication.
t = 0, 1 month, 2 months, 3 months and 6 months
NRS-11 reduction for itch ≥ 4 points
Délai: t = 0, 1 month, 2 months, 3 months and 6 months
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
Délai: t = 0, 1 month, 2 months, 3 months and 6 months
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
Délai: t = 0, 1 month, 2 months, 3 months and 6 months
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
Délai: t = 0, 1 month, 2 months, 3 months and 6 months
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

Autres mesures de résultats

Mesure des résultats
Description de la mesure
Délai
CDLQI ≥4 years
Délai: t = 0, 3 months and 6 months
Children's Dermatology Life Quality Index, in the context of a cost-effectiveness analysis
t = 0, 3 months and 6 months
IDQoL <4 years
Délai: t = 0, 3 months and 6 months
Infants' Dermatitis Quality of Life Index, in the context of a cost-effectiveness analysis
t = 0, 3 months and 6 months
Emollients and steroid use in frequency and tubes used
Délai: t = 0, 1 month, 2 months, 3 months, 4 months, 5 months and 6 months
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
Délai: Over the course of 6 months
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
Délai: Over the course of 6 months
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
Délai: t = - 2 weeks, 0, 3 months and 6 months
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
Délai: t = 0, 3 months and 6 months
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)
Délai: t = 0, 1 month, 3 months and 6 months
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)
Délai: t = 0 and 6 months
Changes in IgE during topical treatment over the course of 6 months.
t = 0 and 6 months
Cellular blood panel (systemic arm)
Délai: t = 0, 1 month, 3 months and 6 months
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
Délai: t = 0
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
Délai: t = 0, 3 months and 6 months
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)
Délai: t = 0
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)
Délai: t = 0
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

Collaborateurs et enquêteurs

C'est ici que vous trouverez les personnes et les organisations impliquées dans cette étude.

Parrainer

Erasmus Medical Center

Collaborateurs

ZonMw: The Netherlands Organisation for Health Research and Development

Dates d'enregistrement des études

Ces dates suivent la progression des dossiers d'étude et des soumissions de résultats sommaires à ClinicalTrials.gov. Les dossiers d'étude et les résultats rapportés sont examinés par la Bibliothèque nationale de médecine (NLM) pour s'assurer qu'ils répondent à des normes de contrôle de qualité spécifiques avant d'être publiés sur le site Web public.

Dates principales de l'étude

Début de l'étude (Réel)

16 août 2021

Achèvement primaire (Anticipé)

1 janvier 2024

Achèvement de l'étude (Anticipé)

1 janvier 2025

Dates d'inscription aux études

Première soumission

29 avril 2021

Première soumission répondant aux critères de contrôle qualité

6 mai 2021

Première publication (Réel)

7 mai 2021

Mises à jour des dossiers d'étude

Dernière mise à jour publiée (Réel)

18 août 2021

Dernière mise à jour soumise répondant aux critères de contrôle qualité

17 août 2021

Dernière vérification

1 août 2021

Plus d'information

Termes liés à cette étude

Mots clés

Termes MeSH pertinents supplémentaires

Autres numéros d'identification d'étude

  • MEC-2019-0568
  • 2019-003247-30 (Numéro EudraCT)

Plan pour les données individuelles des participants (IPD)

Prévoyez-vous de partager les données individuelles des participants (DPI) ?

NON

Informations sur les médicaments et les dispositifs, documents d'étude

Étudie un produit pharmaceutique réglementé par la FDA américaine

Non

Étudie un produit d'appareil réglementé par la FDA américaine

Non

produit fabriqué et exporté des États-Unis.

Non

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