A prospective study to assess safety and efficacy of the use of bone-marrow derived MESenchymal stromal cells as immunomodulatory therapy for children and young adults with severe and difficult-to-treat frequentlyrelapsing or steroid-dependent idiopathic NEPHrotic syndrome: the MESNEPH study - MESNEPH STUDY

BAMBINO GESU' CHILDREN'S HOSPITA0 sites20 target enrollmentStarted: September 14, 2018Last updated: February 7, 2022

Overview

No summary available.

•Males and females aged 5 to 40 years.
•Steroid\-dependent or multirelapsing INS patients with 2 or more relapses in the previous year in spite of prednisone and/or one or more other immunosuppressive steroid\-sparing agent. Only patients reported to invariably relapse upon treatment tapering or withdrawal who are on stable (from at least 1 month) complete (\<0\.3 g/24h for adults or \<4 mg/h/m2 for children) or partial (\<3\.5 g/24h for adults or \<40 mg/h/m2 for children) remission of the INS will be included. Rituximab treatment can have been previously performed but reconstitution of B cells, defined as total CD19/CD20 lymphocyte count above 5% of total lymphocytes by cytofluorimetry, must have occurred and must be recorded.
•Histological diagnosis of MCD, FSGS, mesangial proliferative glomerulonephritis.
•Written informed consent (or consent from parents or tutors for underage patients, as appropriate).
•If applicable, female participants must have pregnancy test by beta\-HCG dosing and be negative.
•Patients of child\-bearing or child\-fathering potential must be willing to practice and must contact their physician to agree with him/her on the most appropriate approach for birth control from the time of enrollment in this study and for 3 months after receiving the latest MSC infusion.
•Are the trial subjects under 18? yes
•Number of subjects for this age range: 12
•F.1\.2 Adults (18\-64 years) yes
•F.1\.2\.1 Number of subjects for this age range 8

•Advanced renal failure (creatinine clearance less than 20 ml/min/1\.73m2\), calculated using the Schwartz formula or the Cockroft\-Gault formula, as appropriate;
•Refractory or persistent NS;
•Genetic mutations associated with intrinsic abnormalities of the glomerular barrier;
•Pregnancy or lactating;
•Women of childbearing potential without following a scientifically accepted form of contraception;
•Infectious pathogen testing positive for active infection;
•Legal incapacity;
•Evidence of an uncooperative attitude;
•Previous diagnosis of: intellectual disability/mental retardation, dementia, schizophrenia.
•Any evidence that patient will not be able to complete the trial follow\-up.

Sponsor

BAMBINO GESU' CHILDREN'S HOSPITA