A phase 2 study to evaluate the efficacy of allogeneic human cord blood-derived mesenchymal stromal cells in pediatric patients with steroid-dependent nephrotic syndrome in maintaining remission after immunosuppressive therapy withdrawal

A phase 2 open-label study to evaluate the efficacy of allogeneic human cord blood-derived mesenchymal stromal cells in maintaining remission after immunosuppressive therapy withdrawal in pediatric patients with steroid-dependent nephrotic syndrome - Reduce immunosuppression with Atmp in NS ChildrEn - RACE

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2018-001162-42-IT

Enrollment

Registered

2019-03-14

Start date

2018-08-21

Completion date

Unknown

Last updated

2024-10-21

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Steroid dependent nephrotic syndrome MedDRA version: 20.0 Level: SOC Classification code 10038359 Term: Renal and urinary disorders System Organ Class: 10038359 - Renal and urinary disorders

Interventions

Product Name: Cellule mesenchimali stromali di origine cordonale (CB-MSC) per uso allogenico) Product Code: CF-CB-MSC Pharmaceutical Form: Suspension for injection Current Sponsor code: CF-CB-MSC Conc

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. Age between 3 and 18 years; 2. Clinical diagnosis of SDNS; 3. Disease remission maintained by chronic therapy (at least 6 months) with either: - Use of a combination of 2 or more immunosuppressive drugs - use of 1 of the calcineurin inhibitors (Cyclosporin or Tacrolimus); 4. Absence of proteinuria (PrU/CrU =65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1. Age < 3 years or = 19 years; 2. Resistant/refractory NS; 3. Presence of genetic mutations associated with NS; 4. eGFR less than 70 ml/min/1.73m^2; 5. Thrombophilic condition; 6. Pregnancy or lactating; 7. Evidence of an uncooperative attitude; 8. Any evidence that the patient will be unable to complete the trial follow-up.

Design outcomes

Primary

Measure	Time frame
Main Objective: To evaluate whether CF-CB-MSC therapy is able to prevent NS recurrence for at least 6 months after complete withdrawal of immunosuppressive treatment in children with SDNS.;Secondary Objective: To assess: - whether CB-MSC therapy may reduce the need for steroids and other immunosuppressive agents to prevent and treat further disease relapses; - the time to recurrence of proteinuria after treatment; - the dosage and time schedule of CF-CB-MSC treatments needed to maintain remission of proteinuria after immunosuppressive treatment withdrawal; - the treatment safety profile; - the regression of related toxicities, such as hypertension, impaired glucose tolerance and dyslipidemia.;Primary end point(s): The percentage of patients without NS recurrence after complete withdrawal of immunosuppressive treatment for at least 6 months.;Timepoint(s) of evaluation of this end point: 6 months	—

Measure

Time frame

Main Objective: To evaluate whether CF-CB-MSC therapy is able to prevent NS recurrence for at least 6 months after complete withdrawal of immunosuppressive treatment in children with SDNS.;Secondary Objective: To assess: - whether CB-MSC therapy may reduce the need for steroids and other immunosuppressive agents to prevent and treat further disease relapses; - the time to recurrence of proteinuria after treatment; - the dosage and time schedule of CF-CB-MSC treatments needed to maintain remission of proteinuria after immunosuppressive treatment withdrawal; - the treatment safety profile; - the regression of related toxicities, such as hypertension, impaired glucose tolerance and dyslipidemia.;Primary end point(s): The percentage of patients without NS recurrence after complete withdrawal of immunosuppressive treatment for at least 6 months.;Timepoint(s) of evaluation of this end point: 6 months

—

Secondary

Measure	Time frame
Secondary end point(s): - The percentage of adverse events; - The time to recurrence of nephrotic syndrome; - The percentage of participants achieving a reduction in the immunosuppressive therapy; - The dose of immunosuppressive therapy to prevent further NS relapses; - Quality of life.;Timepoint(s) of evaluation of this end point: 6 months	—

Countries

Italy

Contacts

Public ContactU.O.C. Nefrologia, Dialisi e Trapia

FONDAZIONE IRCCS CA' GRANDA OSPEDALE MAGGIORE POLICLINICO

giovanni.montini@unimi.it0255032336

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026