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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Experimental group №1 (Secukinumab ) | Experimental | Secukinumab - subcutaneous injections into the shoulder according to the schedule of 0,1,2,3 weeks, then injections 1 time in 3 months up to 52 weeks of therapy. |
|
| Experimental group №2 (Ustekinumab) | Experimental | Ustekinumab - subcutaneous injections in the shoulder on schedule 0; 1 month, then every 2 months up to 52 weeks of therapy. |
|
| Experimental group №3 (Dupilumab) | Experimental | Dupilumab - subcutaneous injections in the shoulder: for patients weighing from 15 to <30 kg: initial dose - 600 mg (2 injections of 300 mg), then 300 mg every 4 weeks; for patients weighing from 30 to <60 kg: initial dose - 400 mg (2 injections of 200 mg), then 200 mg every 2 weeks; for patients weighing 60 kg or more: the initial dose is 600 mg (2 injections of 300 mg), then 300 mg every 2 weeks. |
|
| Control group (Symptomatic therapy) | Active Comparator | symptomatic therapy with emollients + systemic retinoids |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Secukinumab Injection | Drug | Pathogenetic therapy with biologic drugs |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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) | 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) | 52 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | From Baseline up to 16 weeks |
| Transepidermal water loss (TEWL) level change |
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Inclusion Criteria:
The subject has signed an informed consent; parental or legal representative consent for patients under 18 years of age, as well as additional consent for patients aged ≥ 15 and <18.
Subjects should have at least moderate IASI erythema associated with his / her ichthyosis, and a decrease in the quality of life according to CDLQI ≥ 10
Absence of signs of severe infectious diseases (pneumonia, tuberculosis, etc.)
No previous history of the use of the following genetically engineered biological drugs: ustekinumab, secukinumab, dupilumab
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Karine O. Avetisyan, MD | Contact | +79260869259 | avetisyan.karine@mail.ru |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| National Medical Research Center for Children's Health | Recruiting | Moscow | 119296 | Russia |
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| Ustekinumab Injection | Drug | Pathogenetic therapy with biologic drugs |
|
| Dupilumab Injection | Drug | Pathogenetic therapy with biologic drugs |
|
| Symptomatic therapy | Other | Active external agents, Emollients, systemic retinoids if needed |
|
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. |
| 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. | 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. | 52 weeks |
| ID | Term |
|---|---|
| D016112 | Ichthyosis Vulgaris |
| D056770 | Netherton Syndrome |
| ID | Term |
|---|---|
| D007057 | Ichthyosis |
| D012868 | Skin Abnormalities |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D012873 | Skin Diseases, Genetic |
| D030342 | Genetic Diseases, Inborn |
| D007642 | Keratosis |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D000015 | Abnormalities, Multiple |
| D016113 | Ichthyosiform Erythroderma, Congenital |
| D007232 | Infant, Newborn, Diseases |
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| ID | Term |
|---|---|
| C555450 | secukinumab |
| D000069549 | Ustekinumab |
| C582203 | dupilumab |
| ID | Term |
|---|---|
| D061067 | Antibodies, Monoclonal, Humanized |
| D000911 | Antibodies, Monoclonal |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
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