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

NCT04996485 | Unknown Phase 4

• 1 other identifier: 12269723
• Study Type: interventional
• Target: 50
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Congenital Ichthyosis

Keywords

Congenital ichthyosis; Netherton syndrome; Autosomal recessive congenital ichthyosis; Biologic therapy

Outcome Measures

Primary Outcomes (2)

• 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

Secondary Outcomes (4)

• Transepidermal water loss (TEWL) level change

  From Baseline up to 16 weeks

• Transepidermal water loss (TEWL) level change

  52 weeks

• Change in the Children's Dermatology Life Quality Index (CDLQI)

  From Baseline up to 16 weeks

• Change in the Children's Dermatology Life Quality Index (CDLQI)

  52 weeks

Study Arms (4)

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.

Drug: Secukinumab Injection

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.

Drug: Ustekinumab Injection

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.

Drug: Dupilumab Injection

Control group (Symptomatic therapy)

ACTIVE COMPARATOR

symptomatic therapy with emollients + systemic retinoids

Other: Symptomatic therapy

Interventions

Secukinumab Injection DRUG

Pathogenetic therapy with biologic drugs

Experimental group №1 (Secukinumab )

Ustekinumab Injection DRUG

Pathogenetic therapy with biologic drugs

Experimental group №2 (Ustekinumab)

Dupilumab Injection DRUG

Pathogenetic therapy with biologic drugs

Experimental group №3 (Dupilumab)

Symptomatic therapy OTHER

Active external agents, Emollients, systemic retinoids if needed

Control group (Symptomatic therapy)

Eligibility Criteria

• Age: 6 Months - 18 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• National Medical Research Center for Children's Health, Russian Federation: lead

Study Sites (1)

National Medical Research Center for Children's Health

Moscow, 119296, Russia

RECRUITING

MeSH Terms

Conditions

Ichthyosis Vulgaris; Netherton Syndrome

Interventions

secukinumab; Ustekinumab; dupilumab

Condition Hierarchy (Ancestors)

Ichthyosis; Skin Abnormalities; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Skin Diseases, Genetic; Genetic Diseases, Inborn; Keratosis; Skin Diseases; Skin and Connective Tissue Diseases; Abnormalities, Multiple; Ichthyosiform Erythroderma, Congenital; Infant, Newborn, Diseases

Intervention Hierarchy (Ancestors)

Antibodies, Monoclonal, Humanized; Antibodies, Monoclonal; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Central Study Contacts

Karine O. Avetisyan, MD

CONTACT

+79260869259; avetisyan.karine@mail.ru

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NON RANDOMIZED
• Masking: SINGLE
• Who Masked: INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 21, 2021
• First Posted: August 9, 2021
• Study Start: March 1, 2021
• Primary Completion: March 1, 2023
• Study Completion: June 1, 2023
• Last Updated: August 9, 2021

Record last verified: 2021-07

Locations

RU (1)