Back to resultsScientific Substantiation and Assessment of the Effectiveness of Pathogenetic Methods of Therapy for Congenital Ichthyosis in Children
Primary Purpose
Congenital Ichthyosis
Status
Unknown status
Phase
Phase 4
Locations
Russian Federation
Study Type
Interventional
Intervention
Secukinumab Injection
Ustekinumab Injection
Dupilumab Injection
Symptomatic therapy
Sponsored by
About this trial
This is an interventional treatment trial for Congenital Ichthyosis focused on measuring Congenital ichthyosis, Netherton syndrome, Autosomal recessive congenital ichthyosis, Biologic therapy
Eligibility Criteria
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
Sites / Locations
- National Medical Research Center for Children's HealthRecruiting
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm 4
Arm Type
Experimental
Experimental
Experimental
Active Comparator
Arm Label
Experimental group №1 (Secukinumab )
Experimental group №2 (Ustekinumab)
Experimental group №3 (Dupilumab)
Control group (Symptomatic therapy)
Arm Description
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.
Ustekinumab - subcutaneous injections in the shoulder on schedule 0; 1 month, then every 2 months up to 52 weeks of therapy.
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.
symptomatic therapy with emollients + systemic retinoids
Outcomes
Primary Outcome Measures
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)
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)
Secondary Outcome Measures
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.
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.
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.
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.
Full Information
NCT ID
NCT04996485
First Posted
July 21, 2021
Last Updated
July 30, 2021
Sponsor
National Medical Research Center for Children's Health, Russian Federation
1. Study Identification
Unique Protocol Identification Number
NCT04996485
Brief Title
Scientific Substantiation and Assessment of the Effectiveness of Pathogenetic Methods of Therapy for Congenital Ichthyosis in Children
Official Title
Scientific Substantiation and Assessment of the Effectiveness of Pathogenetic Methods of Therapy for Congenital Ichthyosis in Children
Study Type
Interventional
2. Study Status
Record Verification Date
July 2021
Overall Recruitment Status
Unknown status
Study Start Date
March 1, 2021 (Actual)
Primary Completion Date
March 1, 2023 (Anticipated)
Study Completion Date
June 1, 2023 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
National Medical Research Center for Children's Health, Russian Federation
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Product Manufactured in and Exported from the U.S.
No
Data Monitoring Committee
No
5. Study Description
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.
Detailed 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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Congenital Ichthyosis
Keywords
Congenital ichthyosis, Netherton syndrome, Autosomal recessive congenital ichthyosis, Biologic therapy
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 4
Interventional Study Model
Parallel Assignment
Masking
Investigator
Allocation
Non-Randomized
Enrollment
50 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Experimental group №1 (Secukinumab )
Arm Type
Experimental
Arm Description
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.
Arm Title
Experimental group №2 (Ustekinumab)
Arm Type
Experimental
Arm Description
Ustekinumab - subcutaneous injections in the shoulder on schedule 0; 1 month, then every 2 months up to 52 weeks of therapy.
Arm Title
Experimental group №3 (Dupilumab)
Arm Type
Experimental
Arm Description
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.
Arm Title
Control group (Symptomatic therapy)
Arm Type
Active Comparator
Arm Description
symptomatic therapy with emollients + systemic retinoids
Intervention Type
Drug
Intervention Name(s)
Secukinumab Injection
Intervention Description
Pathogenetic therapy with biologic drugs
Intervention Type
Drug
Intervention Name(s)
Ustekinumab Injection
Intervention Description
Pathogenetic therapy with biologic drugs
Intervention Type
Drug
Intervention Name(s)
Dupilumab Injection
Intervention Description
Pathogenetic therapy with biologic drugs
Intervention Type
Other
Intervention Name(s)
Symptomatic therapy
Intervention Description
Active external agents, Emollients, systemic retinoids if needed
Primary Outcome Measure Information:
Title
Change in Ichthyosis Area Severity Index (IASI)
Description
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
Title
Change in Ichthyosis Area Severity Index (IASI)
Description
Clinical measures included the Ichthyosis Area Severity Index (IASI), which integrates erythema (IASI-E) and scaling (IASI-S)
Time Frame
52 weeks
Secondary Outcome Measure Information:
Title
Transepidermal water loss (TEWL) level change
Description
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
Title
Transepidermal water loss (TEWL) level change
Description
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
Title
Change in the Children's Dermatology Life Quality Index (CDLQI)
Description
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
Title
Change in the Children's Dermatology Life Quality Index (CDLQI)
Description
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.
Time Frame
52 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
Accepts Healthy Volunteers
Eligibility Criteria
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
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Karine O. Avetisyan, MD
Phone
+79260869259
Email
avetisyan.karine@mail.ru
Facility Information:
Facility Name
National Medical Research Center for Children's Health
City
Moscow
ZIP/Postal Code
119296
Country
Russian Federation
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Karine Avetisyan, MD
Phone
+79260869259
Email
Avetisyan.karine@mail.ru
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Learn more about this trial
Scientific Substantiation and Assessment of the Effectiveness of Pathogenetic Methods of Therapy for Congenital Ichthyosis in Children
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