Congenital IchthyosisPhase 4NCT04996485
Scientific Substantiation and Assessment of the Effectiveness of Pathogenetic Methods of Therapy for Congenital Ichthyosis in Children
A Phase 4 study testing Secukinumab Injection, Ustekinumab Injection and Dupilumab Injection in people with Congenital Ichthyosis, run by National Medical Research Center for Children's Health, Russian Federation at 1 site in Russia.
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. Read more about this study
- Who is it for?
- Ages 6 months to 18 yearsHealthy volunteers can take part
- Conditions the study is for
- Congenital Ichthyosis
- What is being tested?
- Secukinumab Injection, Ustekinumab Injection and Dupilumab Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 1 siteRussia
- Study dates
- 1 Mar 2021to 1 Jun 2023
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 18 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
Russia1 site, 1 recruiting
- Recruiting
National Medical Research Center for Children's Health
Moscow, 119296
Karine Avetisyan, MD, +79260869259, Avetisyan.karine@mail.ru
About this study
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.
Read the 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.
- Conditions
- Congenital Ichthyosis
- Being tested
- Secukinumab Injection, Ustekinumab Injection, Dupilumab Injection, Symptomatic therapy
- Keywords
- Congenital ichthyosis, Netherton syndrome, Autosomal recessive congenital ichthyosis, Biologic therapy
- Medical subject headings
- Ichthyosis Vulgaris, Netherton Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Secukinumab InjectionDrug
Pathogenetic therapy with biologic drugs
Ustekinumab InjectionDrug
Pathogenetic therapy with biologic drugs
Dupilumab InjectionDrug
Pathogenetic therapy with biologic drugs
Symptomatic therapyOther
Active external agents, Emollients, systemic retinoids if needed
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: 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. Receives: Drug: Dupilumab Injection |
| Control group (Symptomatic therapy) | Active comparator | symptomatic therapy with emollients + systemic retinoids Receives: Other: Symptomatic therapy |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 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)
Measured: 52 weeks
Secondary outcomes (4)
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.
Measured: 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.
Measured: 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.
Measured: 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.
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Aug 2021
- Study start1 Mar 2021
- Primary completion1 Mar 2023 (estimated)
- Study completion1 Jun 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Congenital IchthyosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Investigator
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Medical Research Center for Children's Health, Russian Federation
- Sponsor type
- Other government
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04996485
- Sponsor's protocol ID
- 12269723
- Status last verified
- Jul 2021
- First submitted
- 21 Jul 2021
- Last updated
- 9 Aug 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 9 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Scientific Substantiation and Assessment of the Effectiveness of Pathogenetic Methods of Therapy for Congenital Ichthyosis in Children
- ClinicalTrials.gov ID
- NCT04996485
- Acronym / sponsor ID
- 12269723
- Conditions
- Congenital Ichthyosis
- Keywords
- Congenital ichthyosis, Netherton syndrome, Autosomal recessive congenital ichthyosis, Biologic therapy
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- National Medical Research Center for Children's Health, Russian FederationOther government
- Study start
- 1 Mar 2021 (actual)
- Primary completion
- 1 Mar 2023 (estimated)
- Study completion
- 1 Jun 2023 (estimated)
- First posted
- 9 Aug 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (investigator)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| Experimental group №1 (Secukinumab ) | Experimental | Drug: Secukinumab InjectionSecukinumab - 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 | Drug: Ustekinumab InjectionUstekinumab - 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 | Drug: Dupilumab InjectionDupilumab - 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 | Other: Symptomatic therapysymptomatic therapy with emollients + systemic retinoids |
Who may qualify
- Ages
- 6 months to 18 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Secukinumab InjectionPathogenetic therapy with biologic drugs | Drug | Experimental group №1 (Secukinumab ) |
| Ustekinumab InjectionPathogenetic therapy with biologic drugs | Drug | Experimental group №2 (Ustekinumab) |
| Dupilumab InjectionPathogenetic therapy with biologic drugs | Drug | Experimental group №3 (Dupilumab) |
| Symptomatic therapyActive external agents, Emollients, systemic retinoids if needed | Other | Control group (Symptomatic therapy) |
Primary outcome
Change in Ichthyosis Area Severity Index (IASI)
Time frame: From Baseline up to 16 weeks
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)
Time frame: 52 weeks
Clinical measures included the Ichthyosis Area Severity Index (IASI), which integrates erythema (IASI-E) and scaling (IASI-S)
4 secondary outcomes
Transepidermal water loss (TEWL) level change
Time frame: From Baseline up to 16 weeks
Transepidermal water loss (TEWL) level change
Time frame: 52 weeks
Change in the Children's Dermatology Life Quality Index (CDLQI)
Time frame: From Baseline up to 16 weeks
Change in the Children's Dermatology Life Quality Index (CDLQI)
Time frame: 52 weeks
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Aug 2021.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Secukinumab Injection, Ustekinumab Injection and Dupilumab Injection in people with Congenital Ichthyosis, run by National Medical Research Center for Children's Health, Russian Federation at 1 site in Russia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 9 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Russia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Karine O. Avetisyan, MD (+79260869259, avetisyan.karine@mail.ru), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Ichthyosis Area Severity Index (IASI) (time frame: From Baseline up to 16 weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Aug 2021.