Systemic Lupus Erythematosus of ChildhoodNCT06586710
Interferon Pathway Activation in Monogenic and Nonmonogenic Forms of Pediatric SLE
A study testing Assessment activation of interferon pathway in people with Systemic Lupus Erythematosus of Childhood, run by Meyer Children's Hospital IRCCS at 3 sites in Italy.
Trial summary
Pediatric SLE includes monogenic forms, some of which involve the interferon type I (IFN-I) pathway. The IFN-I pathway is renally active in adult SLE and correlates with the extent of renal damage. Read more about this study
- Who is it for?
- Ages 1 month to 17 years
- Conditions the study is for
- Systemic Lupus Erythematosus of Childhood
- What is being tested?
- Assessment activation of interferon pathway
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 60 participants
- Where
- 3 sitesItaly
- Study dates
- 7 Jun 2023to 31 Dec 2025
- Phase
- Interventional
- 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 1 month to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Diagnosis of SLE arising before the age of majority (until the age of 18 years) according to SLICC and/or EULAR criteria 2019;
- Clinical, laboratory and/or histologic evidence of renal involvement manifested before the age of 18 years;
- Signature of informed consent.
Who cannot join
- Onset of renal disease after the age of 18 years;
- SLE secondary to drugs or associated with other diseases such as systemic sclerosis, rheumatoid arthritis, Sjögren's syndrome, and other connectivities.
Trial locations and contacts
3 sites in 1 country.
Carmela Errichiello, MD contact
Italy3 sites, 3 recruiting
- Recruiting
Meyer Children's Hospital IRCCS
Florence
Carmela Errichiello, MD
- Recruiting
IRCCS Gianna Gaslini
Genova
Andrea Angeletti
- Recruiting
IRCCS Humanitas Research Hospital
Rozzano
Gabriella Moroni
About this study
Official title: Interferon Pathway Activation in Monogenic and Non-monogenic Forms of Pediatric SLE With Renal Involvement. Multicenter Observational Study of Biological Samples.
Pediatric SLE includes monogenic forms, some of which involve the interferon type I (IFN-I) pathway. The IFN-I pathway is renally active in adult SLE and correlates with the extent of renal damage. In pediatric SLE, and particularly in lupus nephritis, activation of the IFN-I pathway has never been studied, nor is it known whether monogenic forms underlie more pronounced interferon activation.
Read the detailed description
Pediatric systemic lupus erythematosus (SLE) (cSLE), compared with adult SLE, is characterized by a more severe phenotype, with more marked hematologic, neuropsychiatric, and renal changes. Lupus nephritis is a pivotal manifestation of pediatric SLE and an important prognostic factor. It is hypothesized that activation of the interferon pathway is more pronounced in monogenic forms, in which the response to IFN-I represents the primary alteration and likely the main pathogenic mechanism.
This finding may also be relevant in light of the availability of new drugs that selectively target the IFN-I pathway.
Demonstration of IFN-I pathway activation could be used as a diagnostic algorithm in aggressive pediatric forms resistant to immunosuppressive therapy and represent a therapeutic target.
- Being tested
- Assessment activation of interferon pathway
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Assessment activation of interferon pathwayOther
- Peripheral blood collection (as part of routine blood draws) on which interferon signature will be performed at the time of enrollment and in case of remission and/or any renal flare.
- Renal biopsies (routinely performed for diagnostic purposes and during clinical follow-up) on which Myxovirus resistance protein 1 (MXA) expression and histopathologic characterization will be assessed.
- Collection of clinical and laboratory data from routine visits performed at baseline and 3, 6, 12, and 24 months (or last available visit) after the renal biopsy was performed.
| Study group | Role | What they receive |
|---|---|---|
| Activation of interferon pathway | Other | Assessment activation of interferon pathway on biological samples (blood and kidney biopsy) in cSLE patients Receives: Other: Assessment activation of interferon pathway |
What is being measured
The primary outcome is the main question the study is designed to answer.
Difference between monogenic and non-monogenic forms of cSLE
Quantification of the IFN-I target genes distinguishing between monogenic and non-monogenic forms.
Measured: At the enrollment, in case of renal flare, in case of disease remission
Evaluation of expression of MXA protein in renal biopsy
Evaluation of expression of MXA protein in renal biopsy (by fluorescence microscopy), distinguishing between genetic and non-genetic forms
Measured: Biopsy available at enrollment
Evaluation of the proportions of the various WHO histological classes of renal biopsy
Evaluation of the proportions of the various WHO histological classes of renal biopsy in patients with monogenic and non-monogenic lupus nephritis.
Histological diagnosis at renal biopsy: WHO histological pattern, activity index, chronicity index, renal TMA
Measured: At the end of the study (24 months after enrollment)
Secondary outcomes (2)
Phenotype characterization of cSLE
Description of clinical parameters, as SLEDAI-2K, in patients with cSLE and renal involvement, distinguishing between monogenic and non-monogenic forms.
Description of laboratory parameters as renal function (eGFR CKiD 25) in patients with cSLE and renal involvement, distinguishing between monogenic and non-monogenic forms
Measured: At the onset of the disease, 3, 6, 12, 24 months from the kidney biopsy,
Correlation between the clinical phenotype, response to treatment and amplification of the interferon pathway
Correlation between the clinical phenotype (laboratory parameters and during renal flare), response to treatment and amplification of the interferon pathway, distinguishing between monogenic and non-monogenic forms.
Clinical, laboratory and histological data relating to any renal flare: number of flares, date of the flare, months from the first biopsy diagnosis of lupus nephritis, clinical manifestations, data from the biopsy performed during the flare, WHO histological pattern compared with the first biopsy, activity index, chronicity index, induction therapy, maintenance therapy
Measured: At the onset of the disease, 3, 6, 12, 24 months from the kidney biopsy,
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Sep 2024
- Study start7 Jun 2023
- Primary completion30 Jun 2025 (estimated)
- Study completion31 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Systemic Lupus Erythematosus of ChildhoodStudies of the same condition, open and under way.
- More trials run by Meyer Children's Hospital IRCCSThe sponsor's other registered studies.
- 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 N/A
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 60 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Meyer Children's Hospital IRCCS
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Carmela Errichiello, Meyer Children's Hospital IRCCS
- Investigators
- Carmela Errichiello, Principal investigator, Meyer Children's Hospital IRCCS, Florence, Italy
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06586710
- Sponsor's protocol ID
- NEFRO-LES
- Status last verified
- Sep 2024
- First submitted
- 20 Feb 2024
- Last updated
- 19 Sep 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Interferon Pathway Activation in Monogenic and Non-monogenic Forms of Pediatric SLE With Renal Involvement. Multicenter Observational Study of Biological Samples.
- ClinicalTrials.gov ID
- NCT06586710
- Acronym / sponsor ID
- NEFRO-LES
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 60 participants (estimated)
- Lead sponsor
- Meyer Children's Hospital IRCCSUniversity, hospital or other
- Study start
- 7 Jun 2023 (actual)
- Primary completion
- 30 Jun 2025 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 19 Sep 2024 (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.
Pediatric SLE includes monogenic forms, some of which involve the interferon type I (IFN-I) pathway. The IFN-I pathway is renally active in adult SLE and correlates with the extent of renal damage. In pediatric SLE, and particularly in lupus nephritis, activation of the IFN-I pathway has never been studied, nor is it known whether monogenic forms underlie more pronounced interferon activation.
Detailed description
Pediatric systemic lupus erythematosus (SLE) (cSLE), compared with adult SLE, is characterized by a more severe phenotype, with more marked hematologic, neuropsychiatric, and renal changes. Lupus nephritis is a pivotal manifestation of pediatric SLE and an important prognostic factor. It is hypothesized that activation of the interferon pathway is more pronounced in monogenic forms, in which the response to IFN-I represents the primary alteration and likely the main pathogenic mechanism.
This finding may also be relevant in light of the availability of new drugs that selectively target the IFN-I pathway.
Demonstration of IFN-I pathway activation could be used as a diagnostic algorithm in aggressive pediatric forms resistant to immunosuppressive therapy and represent a therapeutic target.
Study design
- Primary purpose
- Basic science
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Activation of interferon pathway | Other | Other: Assessment activation of interferon pathwayAssessment activation of interferon pathway on biological samples (blood and kidney biopsy) in cSLE patients |
Who may qualify
- Ages
- 1 month to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Diagnosis of SLE arising before the age of majority (until the age of 18 years) according to SLICC and/or EULAR criteria 2019;
- Clinical, laboratory and/or histologic evidence of renal involvement manifested before the age of 18 years;
- Signature of informed consent.
Key exclusion criteria (2 listed)
- Onset of renal disease after the age of 18 years;
- SLE secondary to drugs or associated with other diseases such as systemic sclerosis, rheumatoid arthritis, Sjögren's syndrome, and other connectivities.
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 |
|---|---|---|
| Assessment activation of interferon pathway* Peripheral blood collection (as part of routine blood draws) on which interferon signature will be performed at the time of enrollment and in case of remission and/or any renal flare. * Renal biopsies (routinely performed for diagnostic purposes and during clinical follow-up) on which Myxovirus resistance protein 1 (MXA) expression and histopathologic characterization will be assessed. * Collection of clinical and laboratory data from routine visits performed at baseline and 3, 6, 12, and 24 months (or last available visit) after the renal biopsy was performed. | Other | Activation of interferon pathway |
Primary outcome
Difference between monogenic and non-monogenic forms of cSLE
Time frame: At the enrollment, in case of renal flare, in case of disease remission
Quantification of the IFN-I target genes distinguishing between monogenic and non-monogenic forms.
Evaluation of expression of MXA protein in renal biopsy
Time frame: Biopsy available at enrollment
Evaluation of expression of MXA protein in renal biopsy (by fluorescence microscopy), distinguishing between genetic and non-genetic forms
Evaluation of the proportions of the various WHO histological classes of renal biopsy
Time frame: At the end of the study (24 months after enrollment)
Evaluation of the proportions of the various WHO histological classes of renal biopsy in patients with monogenic and non-monogenic lupus nephritis. Histological diagnosis at renal biopsy: WHO histological pattern, activity index, chronicity index, renal TMA
2 secondary outcomes
Phenotype characterization of cSLE
Time frame: At the onset of the disease, 3, 6, 12, 24 months from the kidney biopsy,
Correlation between the clinical phenotype, response to treatment and amplification of the interferon pathway
Time frame: At the onset of the disease, 3, 6, 12, 24 months from the kidney biopsy,
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Sep 2024.
- Individual participant dataSharing planned: No
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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.
Carmela Errichiello, MD contact
Carmela Errichiello principal investigator
Meyer Children's Hospital IRCCS, Florence, Italy
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Assessment activation of interferon pathway in people with Systemic Lupus Erythematosus of Childhood, run by Meyer Children's Hospital IRCCS at 3 sites in Italy.
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 3 inclusion and 2 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 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in Italy. 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.
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 Carmela Errichiello, MD (055/5662563, carmela.errichiello@meyer.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Difference between monogenic and non-monogenic forms of cSLE (time frame: At the enrollment, in case of renal flare, in case of disease remission). The study lists 3 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 19 Sep 2024.