Clinical Research Directory

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

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.

ARREST-NEPHROSIS - Austrian Resistant Nephrotic Syndrome Treatment Response Registry and Biobank

Nephrotic syndrome is the clinical phenotype of a heterogeneous group of glomerular diseases that may present with varying degrees of urinary protein loss (proteinuria), dysproteinemia in the blood, fluid retention and impaired renal function. The AustRian RESistanT NEPHROtic Syndrome Treatment Response RegIStry and Biobank (ARREST-NEPHROSIS) sets out to achieve the following goals, as typical categories of rare disease registries 1. Obtaining real world data on practice patterns and outcomes 2. Networking between affected patients, families, and clinicians. 3. Establish a patient base for facilitated recruitment in studies of drugs, medical devices, and products 4. Development of a Biobank to enable research of potential biomarkers and therapy or disease courses