The PRO-FUTURE Project
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Identification of Diagnostic Biomarkers.
- Who it may be relevant to
- Registry conditions: Ureteropelvic Junction Obstruction, Chronic Kidney Diseases. Basic parameters: 1 Day — 5 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- Italy
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
PRecocious biOmarkers oF Unilateral ureTero-pelvic jUnction obstRuction in childrEn
Overview
The goal of this observational study is to learn about specific biomarkers of unilateral ureteropelvic junction obstruction (UPJO) in children undergoing surgical intervention for unilateral UPJO compared with controls. The main question it aims to answer are: * Are Urinary single-cell and extracellular vesicles (EVs) screening useful to stage the intrarenal injury and repair processes in UPJO babies? * Do babies with unilateral UPJO have a whole blood gene expression profiling (WBGEP) allowing an accurate unilateral UPJO diagnosis?
Detailed description
Congenital anomalies of the kidney and urinary tract (CAKUT) are the main cause of chronic kidney disease in children. UPJO is the most common CAKUT. It is defined as impeded urine outflow from the renal pelvis to the ureter, which may result in progressive kidney damage (KD). Renal imaging is the current diagnostic approach, even if substantial shortcomings are present to reliably determine a significant obstruction. Consequently, a follow up is often needed with the risk of progressive KD. The main hypothesis of this proposal is that biofluids (whole blood and urine) could allow a more precise diagnosis and risk stratification of unilateral UPJO compared with the available diagnostic techniques.
In this project the investigators define the UPJO as an hydronephrosis needing of surgical correction according to 2 of the following criteria: anterior-posterior diameter of the pelvis (APDP) ≥ 30 mm, split renal function (SRF) \< 40% at the Tc99mMag3 scintigraphy (Mag3S), SRF with delta \> 10% at follow-up Mag3S, delayed wash-out, progressive increase of the APDP at follow-up ultrasounds, and 4th degree hydronephrosis.
The investigators will extensively investigate for specific biomarkers of UPJO at different levels. The investigators will focus 1. on the urine by investigating for intrarenal remodeling processes and searching for kidney-specific biomarkers by urinary single-cell and EVs screening (aim 1). Moreover, based on the hitherto revealed capability of blood cells to perceive organ-specific illnesses, 2. the investigators will perform WBGEP of babies with unilateral UPJO to identify early biomarkers of disease progression (aim 2).
For the aims 1 and 2, the investigators will enroll 85 patients aged 0-5 years, 35 with unilateral UPJO and 50 controls without hydronephrosis needing surgical correction for hernia, hydrocele or phimosis.
This project could improve the management of patients with suspect of UPJO -starting from birth- by a better understanding of the pathophysiological mechanisms underlying KD and by the identification of early biomarkers of obstruction, reducing the costs and the complications related to either a tardive or not needed surgical correction and reducing the risk of KD development.
Interventions
- Diagnostic test Identification of Diagnostic Biomarkers
Urinary single-cell and EVs screening to stage the intrarenal injury and repair processes in UPJO babies and identification of the whole blood gene expression profiling of babies with unilateral UPJO. Data deriving from patients with unilateral UPJO will be compared with data deriving from controls.
Primary outcome measures
- Urine Biomarkers [Time frame: 48 months]
- Blood Biomarkers [Time frame: 48 months]
Eligibility criteria
Inclusion criteria
- unilateral UPJO
- 0-5years of age
Exclusion criteria
- bilateral UPJO
- previous surgical intervention on the urinary tract
- bladder or lower urinary tract symptoms or malformations
- nephro- or urolithiasis, vesicoureteral reflux
- urinary tract infections.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Case-control
Study locations
Italy · 5 centers
- Univeristà degli Studi della Campania Luigi Vanvitelli — Naples
- Department of Clinical Sciences and Community Health, University of Milano — Milan
- Fondazione IRCCS Ca' Granda-Ospedale Maggiore Policlinico — Milan
- Obstetrics and Gynecology Unit, University of Modena and Reggio Emilia — Modena
- AORN Santobono-Pausilipon — Naples
Identifiers
NCT: NCT06382233 · PRO-FUTURE project