Biomarkers and Outcome Predictors of Pediatric Nephrotic Syndrome: A Genetic, Transcriptomic, and Secretome Multiomics Study

Summary

Idiopathic Nephrotic Syndrome is a rare disease of the kidneys, which typically affects children. For most affected children there is the need of a prolonged treatment with drugs reducing the activity of the immune system, also resulting in many side effects. Those patients, who do not respond to treatment, are at risk of kidney damage and of dialysis or kidney transplantation. It is currently impossible to predict the response to treatment, leading to unnecessary therapies with side effects as well as unclear prognosis in the affected children. The response of the idiopathic nephrotic syndrome to medications acting on the immune system explains its important role in the occurrence of the disease.

With this study we aim to obtain predictors of the response to treatment right at the beginning of the disease, to adapt the therapy avoiding needless side effects. This will be done evaluating the blood and urine of affected children using state of the art molecular characterisation. We will evaluate the genetic predisposition, the cell trait changes and the presence of molecules in blood and urine that may affect the interaction between the immune system and the kidneys. We expect that the findings will improve treatment of children with idiopathic nephrotic syndrome and reduce the number of children suffering from unnecessary drugs related side effects.

Conditions

• Nephrotic Syndrome Steroid-Dependent
• Nephrotic Syndrome Steroid-Resistant
• Nephrotic Syndrome in Children
• Glomerulonephritis
• Proteinuria
• Hypoalbuminemia
• Kidney Diseases
• Chronic Kidney Disease

Sponsors & Collaborators

• Fondazione La Nuova Speranza ONLUS

  collaborator UNKNOWN

• ASSOCIAZIONE SINDROME NEFROSICA ITALIA

  collaborator UNKNOWN

• Nephie e.V.

  collaborator UNKNOWN

• NephCEurope

  collaborator UNKNOWN

• European Joint Programme on Rare Diseases (EJP RD JTC 2023)

  collaborator UNKNOWN

• ERKNet

  collaborator UNKNOWN

• Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

  lead OTHER

Principal Investigators

• Giovanni Montini, Doctor of Medicine · Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Eligibility

Max Age

18 Years

Sex

ALL

Healthy Volunteers

Yes

Timeline & Regulatory

Start

2025-02-15

Primary Completion

2027-12-01

Completion

2028-06-01