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

Trial statusNot yet recruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeUp to 18
SponsorFondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

About this trial

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.

Eligibility criteria

Qualifiers

Clinical diagnosis of idiopathic nephrotic syndrome (INS) with nephrotic range proteinuria (uPr/uCr ratio > 2 mg/mg).

Hypoalbuminemia with serum albumin < 3.0 g/dL.

Presence of edema.

No prior treatment for idiopathic nephrotic syndrome.

Disqualifiers

Diagnosis of congenital or infantile nephrotic syndrome (age < 1 year).

Diagnosis of secondary nephrotic syndrome.

Presence of glomerulonephritis, autoimmune diseases, or vasculitis.

Lack of signed informed consent by a parent or legal guardian.

Trial design

Treatments tested in this trial

  • Not listed

Trial groups

350 Participants
are grouped into 3 trial groups

Locations

This trial has no locations

Sponsors and collaborators

Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico

Lead sponsor

Fondazione La Nuova Speranza ONLUS

Collaborator

ASSOCIAZIONE SINDROME NEFROSICA ITALIA

Collaborator

Nephie e.V.

Collaborator

NephCEurope

Collaborator

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

Collaborator

ERKNet

Collaborator