severe multi-relapsing or steroid-dependent INS.
Conditions
Interventions
Product Name: Autologous mesenchymal stromal cells (MSC)
Pharmaceutical Form: Solution for infusion
Product Name: Autologous mesenchymal stromal cells (MSC)
Pharmaceutical Form: Solution for infusion
Sponsors
BAMBINO GESU' CHILDREN'S HOSPITAL
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: • 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 (=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: • 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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the feasibility and safety of harvesting from their bone marrow, isolating and expanding ex vivo in an approved facility and then infusing autologous MSC in children and young adults with severe multi-relapsing or steroid-dependent INS.;Secondary Objective: 1. To evaluate whether the therapeutic use of autologous MSC is able to prevent disease relapse despite tapering of prednisone and/or other immunosuppressive treatments in children and young adults with severe multi-relapsing or steroid-dependent INS. 2. To assess whether the therapeutic use of autologous MSC may reduce the need for prednisone and/or other immunosuppressive agents to prevent and treat further disease relapses; 3. To evaluate whether tapering or withdrawal of immunosuppressant therapy is associated with regression of the related toxicities, such as growth retardation, hypertension, impaired glucose tolerance and obesity, infections, B cell suppression; 4. To evaluate the immunomodulatory effect of the MSC infusion in vivo in a clinical setting. ;Primary end point(s): Safety outcomes will include serious and non-serious adverse events, including acute reactions during MSC infusion, infectious episodes and malignancies.;Timepoint(s) of evaluation of this end point: at 12 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): - Recurrence of INS, defined as 3+ or more positive Albustix dipsticks for proteinuria on 3 consecutive days or by positive dipsticks (1+ to 3+) for 7 consecutive days, in the 12 months before and in the 12 months following MSC infusions. - The dose of immunosuppressive therapy required to prevent further INS relapses - Adverse effects of immunosuppressive therapy, such as arterial hypertension and need for antihypertensive therapy, obesity and impaired glucose tolerance, dyslipidemia, renal dysfunction, stunted statural growth, infections, immunocompetence; - Kidney function at baseline and at one year after MSC administration. ;Timepoint(s) of evaluation of this end point: at 12 months | — |
Countries
Italy
Contacts
Public ContactMarina Vivarelli
BAMBINO GESU' CHILDREN'S HOSPITAL
Outcome results
None listed