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
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