Nephrotic SyndromeNCT06325098
Implementation of a Diagnostic Workflow for Personalized Diagnosis of Nephrotic Syndrome
A study testing Anti-nephrin antibodies and u-RPC cultures in people with Nephrotic Syndrome, run by Meyer Children's Hospital IRCCS at 1 site in Italy.
Trial summary
Nephrotic syndrome (NS) is a clinical picture common to several diseases resulting from damage to podocytes and glomerular filtration barrier. Currently, there is limited consensus regarding the diagnostic pathway and management of the specific etiology. Read more about this study
- Who is it for?
- Ages 1 month to 40 years
- Conditions the study is for
- Nephrotic Syndrome
- What is being tested?
- Anti-nephrin antibodies and u-RPC cultures
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 150 participants
- Where
- 1 siteItaly
- Study dates
- 26 Jan 2022to 30 Jun 2026
- 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 40 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Clinical diagnosis of NS (SSNS, SRNS, or NS relapsed after transplantation regardless of initial response to steroid therapy)
- Age below 40 years at disease onset
- Availability of clinical information
- Signed informed consent form
Who cannot join
- Age at onset above 40 years
- Kidney biopsy proving lesions other than FSGS and MCD
Trial locations and contacts
1 site in 1 country.
Italy1 site, 1 recruiting
- Recruiting
Meyer Children's Hospital IRCCS
Florence
Paola Romagnani
About this study
Nephrotic syndrome (NS) is a clinical picture common to several diseases resulting from damage to podocytes and glomerular filtration barrier. Currently, there is limited consensus regarding the diagnostic pathway and management of the specific etiology. Some patients show complete response to first-line steroid therapy (steroid-sensitive nephrotic syndrome, SSNS), especially in children and young adults. The prognosis of this group is generally favorable. In contrast, patients unresponsive to steroids (steroid-resistant NS, SRNS) frequently undergo immunosuppressive therapies, which are burdened with numerous side effects. Resistance to treatment is associated with a high likelihood of progression to chronic renal disease (CKD) and kidney failure (ESKD). Recent evidence suggests that immunological mechanisms (including permeabilizing factors) are involved in the pathogenesis of post-transplant NS recurrence and SSNS.
Providing patients with NS with a correct diagnosis is the cornerstone of personalized medicine, reducing morbidity and side effects of therapies, ensuring their appropriate prescription, and slowing or preventing progression to ESKD.
- Conditions
- Nephrotic Syndrome
- Being tested
- Anti-nephrin antibodies, u-RPC cultures
- Medical subject headings
- Nephrotic Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Anti-nephrin antibodiesDiagnostic test
Assessment of anti-nephrin antibodies on serum samples and in vitro study of permeabilizing effect on 3D organ-on-a-chip models and STED microscopy on healthy human kidneys.
u-RPC culturesDiagnostic test
Urine sample collection for u-RPC cultures
| Study group | Role | What they receive |
|---|---|---|
| Patients with SSNS | Experimental | Patients with SSNS and patients undergoing renal transplantation for NS with post-transplant disease recurrence will be enrolled for serum sampling for the study of immunological and/or permeabilizing factors (including anti-nephrin antibodies). Receives: Diagnostic Test: Anti-nephrin antibodies |
| Patients with SRNS undergoing genetic testing by WES | Experimental | Patients with SRNS undergoing genetic testing by WES and variant prioritization will undergo serum sampling for the study of immunologic and/or permeabilizing factors (including the anti-nephrin antibodies), and urine sampling for u-RPC cultures. Receives: Diagnostic Test: Anti-nephrin antibodies; Diagnostic Test: u-RPC cultures |
What is being measured
The primary outcome is the main question the study is designed to answer.
Role of anti-nephrin antibodies in the pathogenesis of NS
The presence of circulating permeabilizing factors, including anti-nephrin antibodies, will be evaluated in serum samples of patients with NS and/or kidney transplant recipients with NS recurrence. The results will be correlated with the clinical history of disease in order to understand the disease pathomechanisms and the risk of recurrence.
Measured: From enrollment until the last patient visit (up to 12 months from enrollment)
Secondary outcomes (3)
Functional role of VUS in the pathogenesis of SRNS
The functional role of VUS identified by WES will be assessed by in vitro studies on u-RPCs and 3D organ-on-a-chip models.
The results will be expressed as diagnostic rate, i.e., number of conclusive diagnosis allowed by functional studies of VUS/number of patients enrolled in the study.
Measured: From enrollment until the last patient last visit (up to 12 months from enrollment)
Identification of potential predictive biomarkers of renal outcome.
The identification of potential biomarkers of disease progression and kidney failure will be performed by gene expression by scRNA-seq of u-RPCs. The results will be correlated to clinical data.
Measured: From enrollment until the last patient visit (up to 12 months from enrollment)
Cost-effectiveness analysis
A modeled cost-effectiveness analysis (including direct and indirect medical costs) will be performed to compare the proposed diagnostic algorithm with the current standard-of-care.
Measured: From enrollment of the last patient until the last visit (up to 12 months from enrollment)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Mar 2024
- Study start26 Jan 2022
- Primary completion31 Jan 2026 (estimated)
- Study completion30 Jun 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Nephrotic SyndromeStudies 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
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Model description
- Patients with nephrotic syndrome (SSNS, SRNS)
- Masking
- None (open label)
- Enrolment
- 150 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Meyer Children's Hospital IRCCS
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Paola Romagnani, Meyer Children's Hospital IRCCS
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06325098
- Sponsor's protocol ID
- PER-NEPH
- Status last verified
- Mar 2024
- First submitted
- 1 Mar 2024
- Last updated
- 22 Mar 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 22 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Implementation of a Diagnostic Workflow for Personalized Diagnosis of Nephrotic Syndrome
- ClinicalTrials.gov ID
- NCT06325098
- Acronym / sponsor ID
- PER-NEPH
- Conditions
- Nephrotic Syndrome
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 150 participants (estimated)
- Lead sponsor
- Meyer Children's Hospital IRCCSUniversity, hospital or other
- Study start
- 26 Jan 2022 (actual)
- Primary completion
- 31 Jan 2026 (estimated)
- Study completion
- 30 Jun 2026 (estimated)
- First posted
- 22 Mar 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.
Nephrotic syndrome (NS) is a clinical picture common to several diseases resulting from damage to podocytes and glomerular filtration barrier. Currently, there is limited consensus regarding the diagnostic pathway and management of the specific etiology. Some patients show complete response to first-line steroid therapy (steroid-sensitive nephrotic syndrome, SSNS), especially in children and young adults. The prognosis of this group is generally favorable. In contrast, patients unresponsive to steroids (steroid-resistant NS, SRNS) frequently undergo immunosuppressive therapies, which are burdened with numerous side effects. Resistance to treatment is associated with a high likelihood of progression to chronic renal disease (CKD) and kidney failure (ESKD). Recent evidence suggests that immunological mechanisms (including permeabilizing factors) are involved in the pathogenesis of post-transplant NS recurrence and SSNS.
Providing patients with NS with a correct diagnosis is the cornerstone of personalized medicine, reducing morbidity and side effects of therapies, ensuring their appropriate prescription, and slowing or preventing progression to ESKD.
Study design
- Primary purpose
- Diagnosis
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Patients with nephrotic syndrome (SSNS, SRNS)
| Arm | Type | Receives |
|---|---|---|
| Patients with SSNS | Experimental | Diagnostic Test: Anti-nephrin antibodiesPatients with SSNS and patients undergoing renal transplantation for NS with post-transplant disease recurrence will be enrolled for serum sampling for the study of immunological and/or permeabilizing factors (including anti-nephrin antibodies). |
| Patients with SRNS undergoing genetic testing by WES | Experimental | Diagnostic Test: Anti-nephrin antibodies, Diagnostic Test: u-RPC culturesPatients with SRNS undergoing genetic testing by WES and variant prioritization will undergo serum sampling for the study of immunologic and/or permeabilizing factors (including the anti-nephrin antibodies), and urine sampling for u-RPC cultures. |
Who may qualify
- Ages
- 1 month to 40 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Clinical diagnosis of NS (SSNS, SRNS, or NS relapsed after transplantation regardless of initial response to steroid therapy)
- Age below 40 years at disease onset
- Availability of clinical information
- Signed informed consent form
Key exclusion criteria (2 listed)
- Age at onset above 40 years
- Kidney biopsy proving lesions other than FSGS and MCD
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 |
|---|---|---|
| Anti-nephrin antibodiesAssessment of anti-nephrin antibodies on serum samples and in vitro study of permeabilizing effect on 3D organ-on-a-chip models and STED microscopy on healthy human kidneys. | Diagnostic test | Patients with SRNS undergoing genetic testing by WES, Patients with SSNS |
| u-RPC culturesUrine sample collection for u-RPC cultures | Diagnostic test | Patients with SRNS undergoing genetic testing by WES |
Primary outcome
Role of anti-nephrin antibodies in the pathogenesis of NS
Time frame: From enrollment until the last patient visit (up to 12 months from enrollment)
The presence of circulating permeabilizing factors, including anti-nephrin antibodies, will be evaluated in serum samples of patients with NS and/or kidney transplant recipients with NS recurrence. The results will be correlated with the clinical history of disease in order to understand the disease pathomechanisms and the risk of recurrence.
3 secondary outcomes
Functional role of VUS in the pathogenesis of SRNS
Time frame: From enrollment until the last patient last visit (up to 12 months from enrollment)
Identification of potential predictive biomarkers of renal outcome.
Time frame: From enrollment until the last patient visit (up to 12 months from enrollment)
Cost-effectiveness analysis
Time frame: From enrollment of the last patient until the last visit (up to 12 months from enrollment)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Mar 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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Anti-nephrin antibodies and u-RPC cultures in people with Nephrotic Syndrome, run by Meyer Children's Hospital IRCCS at 1 site 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 4 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 22 Mar 2024. 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 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 Paola Romagnani, Prof, MD, PhD (055 5662562, paola.romagnani@meyer.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Role of anti-nephrin antibodies in the pathogenesis of NS (time frame: From enrollment until the last patient visit (up to 12 months from enrollment)). 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 22 Mar 2024.