This is an experimental non-randomized clinical study aimed at expanding the indications for the use of biological drugs with the aim of using them for the pathogenetic therapy of children with congenital ichthyosis.
This is an experimental non-randomized clinical study aimed at expanding the indications for the use of biological drugs with the aim of using them for the pathogenetic therapy of children with congenital ichthyosis. The study will include 50 children aged 6 months to 18 years with a clinically and genetically confirmed diagnosis of congenital ichthyosis. Patients will be divided into 4 groups who will receive symptomatic therapy (using active external agents, emollients and / or systemic retinoids) or biologics targeting the cytokines IL-12 / IL-23, IL-4 / IL-13 and IL -17A. Immunophenotyping of all patients will be performed, the cytokine profile and spectrum of sensitization and the degree of NF-kB activation in lymphocytes will be determined. In experimental group №3, 10 patients with Netherton syndrome will receive dupilumab, in experimental group №2, 10 patients will receive ustekinumab, and in experimental group №1 10 patients will receive secukinumab. Efficiency will be assessed using the Ichthyosis Area Severity Index (IASI), determination of the level of TEWL, and the change in quality of life will also be assessed using the Children's Dermatological Life Quality Index (CDLQI) in comparison from baseline, than at 16 and 52 weeks. Throughout the study, the safety profile (registration of the development of infectious diseases) will be assessed.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
SINGLE
Enrollment
50
Pathogenetic therapy with biologic drugs
Pathogenetic therapy with biologic drugs
Pathogenetic therapy with biologic drugs
National Medical Research Center for Children's Health
Moscow, Russia
RECRUITINGChange in Ichthyosis Area Severity Index (IASI)
Clinical measures included the Ichthyosis Area Severity Index (IASI), which integrates erythema (IASI-E) and scaling (IASI-S)
Time frame: From Baseline up to 16 weeks
Change in Ichthyosis Area Severity Index (IASI)
Clinical measures included the Ichthyosis Area Severity Index (IASI), which integrates erythema (IASI-E) and scaling (IASI-S)
Time frame: 52 weeks
Transepidermal water loss (TEWL) level change
Change in transepidermal water loss (TEWL) while rebuilding the epidermal barrier of the skin (an indicator of the effectiveness of the therapy). Measured in g/hm2.
Time frame: From Baseline up to 16 weeks
Transepidermal water loss (TEWL) level change
Change in transepidermal water loss (TEWL) while rebuilding the epidermal barrier of the skin (an indicator of the effectiveness of the therapy). Measured in g/hm2.
Time frame: 52 weeks
Change in the Children's Dermatology Life Quality Index (CDLQI)
increasing The Children's Dermatology Life Quality Index, as an indicator of the effectiveness of therapy. The CDLQI is calculated by summing the score of each question resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired. The CDLQI can also be expressed as a percentage of the maximum possible score of 30.
Time frame: From Baseline up to 16 weeks
Change in the Children's Dermatology Life Quality Index (CDLQI)
Change in the Children's Dermatology Life Quality Index (CDLQI), as an indicator of the effectiveness of therapy. The CDLQI is calculated by summing the score of each question resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired. The CDLQI can also be expressed as a percentage of the maximum possible score of 30.
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Active external agents, Emollients, systemic retinoids if needed
Time frame: 52 weeks