Analyzing gene expression in children with idiopathic nephrotic syndrome to predict steroid response
Transcriptome Analysis in Idiopathic Nephrotic Syndrome: an Insight Into the Mechanisms of Steroid Responsiveness
This study is testing if looking at gene activity in children with idiopathic nephrotic syndrome can help predict how well they will respond to steroid treatment.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 60 (estimated) |
| Ages | 1 Year to 12 Years |
| Sex | All |
| Sponsor | IRCCS Burlo Garofolo Academic / other |
| Locations | 1 site (Trieste) |
| Trial ID | NCT06325137 on ClinicalTrials.gov |
What this trial studies
This observational study focuses on children aged 1 to 12 years diagnosed with idiopathic nephrotic syndrome (INS) to analyze their whole transcriptome. By utilizing RNA sequencing technologies, the study aims to identify molecular biomarkers that can predict steroid responsiveness before glucocorticoid treatment begins. This approach seeks to improve INS classification and potentially reduce unnecessary glucocorticoid treatments for patients who are unlikely to respond. The study also includes age-matched controls with congenital anomalies of the kidney and urinary tract for comparative analysis.
Who should consider this trial
Good fit: Ideal candidates for this study are children aged 1 to 12 years with a clinical diagnosis of idiopathic nephrotic syndrome.
Not a fit: Patients younger than 1 year or older than 12 years, as well as those with secondary or genetic forms of nephrotic syndrome, may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could lead to more personalized treatment strategies for children with idiopathic nephrotic syndrome, minimizing exposure to ineffective glucocorticoids.
How similar studies have performed: While the approach of using transcriptome analysis for predicting treatment response is innovative, similar studies have shown promise in other conditions, indicating potential for success in this area.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * clinical diagnosis of INS * age 1 - 12 years * signed informed consent by parents or legal guardians * For controls: aged-matched individuals with Congenital Anomalies of the Kidney and Urinary Tract (CAKUT) Exclusion Criteria: * Patients aged \<1 year or \> 12 years * Patients diagnosed with a secondary or genetic form of NS * For controls: the presence of coexisting chronic kidney failure, autoimmune or autoinflammatory disease, or endocrinologic disorders.
Where this trial is running
Trieste
- Institute for Maternal and Child Health - IRCCS "Burlo Garofolo" — Trieste, Italy (Recruiting)
Study contacts
- Study coordinator: Marco Pennesi, MD
- Email: marco.pennesi@burlo.trieste.it
- Phone: +39. 040-3785263
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.