Scientific Substantiation and Assessment of the Effectiveness of Pathogenetic Methods of Therapy for Congenital Ichthyosis in Children

Congenital Ichthyosis Clinical Trial

Official title:

Scientific Substantiation and Assessment of the Effectiveness of Pathogenetic Methods of Therapy for Congenital Ichthyosis in Children

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04996485
• Other study ID #: 12269723
• Status: Recruiting
• Phase: Phase 4
• Start date: March 1, 2021
• Est. completion date: June 1, 2023

Study information

• Verified date: July 2021
• Source: National Medical Research Center for Children's Health, Russian Federation
• Contact: Karine O. Avetisyan, MD
• Phone: +79260869259
• Email: avetisyan.karine[@]mail.ru
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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.

Description:

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 50
• Est. completion date: June 1, 2023
• Est. primary completion date: March 1, 2023
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 6 Months to 18 Years

Eligibility

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.

• At the time of participation in the study, the age of the subject is not less than 6 months and not more than 18 years.

• At the time of participation in the study, a clinical diagnosis was established:

Congenital ichthyosis with various clinical forms, with the exception of vulgar and X - linked congenital ichthyosis (genetic research is not a prerequisite for participation in the study).

• 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:

• Subjects who have an allergic reaction to ustekinumab, secukinumab or other components of the drugs.

• Subjects who have bacterial and/or fungal diseases.

• Subjects who have problems in dynamic observation.

• Subjects who will have a worsening of clinical symptoms

Related Conditions & MeSH terms

• Congenital Ichthyosis
• Ichthyosis
• Ichthyosis, Lamellar

Intervention

Drug:
• Secukinumab Injection
Pathogenetic therapy with biologic drugs
• Ustekinumab Injection
Pathogenetic therapy with biologic drugs
• Dupilumab Injection
Pathogenetic therapy with biologic drugs

Other:
• Symptomatic therapy
Active external agents, Emollients, systemic retinoids if needed

Locations

Country	Name	City	State
Russian Federation	National Medical Research Center for Children's Health	Moscow

Sponsors (1)

Lead Sponsor	Collaborator
National Medical Research Center for Children's Health, Russian Federation

Country where clinical trial is conducted

Russian Federation, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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
Secondary	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
Secondary	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.	52 weeks
Secondary	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
Secondary	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