Study comparing the ability of two medications, namely oral tablets of mycophenolate mofetil and cyclophosphamide, in keeping disease well controlled in patients with frequently relapsing or steroid dependent nephrotic syndrome

Efficacy and Safety of Mycophenolate Mofetil versus Oral Cyclophosphamide in Children with Frequently Relapsing or Steroid Dependent Nephrotic Syndrome: An Open Label Randomized Controlled Trial

Status

Recruiting

Phases

Phase 4

Study type

Interventional

Source

CTRI

Registry ID

CTRI/2022/10/046891

Enrollment

130

Registered

2022-10-28

Start date

Unknown

Completion date

Unknown

Last updated

2026-01-12

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

Conditions

Health Condition 1: N040- Nephrotic syndrome with minor glomerular abnormality

Interventions

Intervention1: Mycophenolate mofetil (MMF): This shall be administered at a dose of 900-1200 mg/square meter body surface area every day in two divided doses for 12 months Control Intervention1: Oral

Eligibility

Inclusion criteria

Inclusion criteria: Patients with idiopathic steroid sensitive nephrotic syndrome with frequent relapses or steroid dependence in the preceding 12-months and with parents willing to give informed written consent and child giving informed written assent

Exclusion criteria

Exclusion criteria: Known secondary etiology (e.g., lupus erythematosus, IgA nephropathy, amyloidosis) Initial or late steroid resistance, currently or historically Therapy with oral or intravenous cyclophosphamide or MMF ever in the past Therapy with cyclosporine, tacrolimus, azathioprine or rituximab in the past 6 months Inability to swallow tablets or capsules of MMF Impaired renal function, as indicated by eGFR Leukopenia, neutropenia, thrombocytopenia, elevated transaminases Known chronic infection (tuberculosis, HIV, hepatitis B or C) or malignancy Residing more than 100 km away or unwilling to come for regular follow-up

Design outcomes

Primary

Measure	Time frame
The proportion of patients in sustained remissionTimepoint: 12-months	—

Secondary

Measure	Time frame
The frequency of relapsesTimepoint: 12 months;The proportion of patients with frequent relapsesTimepoint: 12 months;The proportion of patients with treatment failure (frequent steroid sensitive relapses, late steroid resistance, or serious adverse event due to the intervention)Timepoint: 12 months;The time to first relapse, frequent relapses, and treatment failureTimepoint: 12 months;The cumulative prednisolone requirement (mg/kg/day)Timepoint: 12 months;Types and rates of adverse effects, including leukopenia, neutropenia, thrombocytopenia, anemia; abdominal pain, diarrhea, vomiting; seizures; alopecia and elevated hepatic transaminases; infectionsTimepoint: 12 months;Changes in anthropometric and blood pressure standard deviation scoresTimepoint: 12 months;In a subset of 20 patients administered MMF, to perform therapeutic drug monitoring for mycophenolic acid (area under the curve, trough level) at 2-weeks, 6-months and at relapseTimepoint: 6 months;In a subset of 20 patients adminstered either agent, compare the proportions of immune cell subsets in peripheral blood of patientsTimepoint: 12 months	—

Measure

Time frame

The frequency of relapsesTimepoint: 12 months;The proportion of patients with frequent relapsesTimepoint: 12 months;The proportion of patients with treatment failure (frequent steroid sensitive relapses, late steroid resistance, or serious adverse event due to the intervention)Timepoint: 12 months;The time to first relapse, frequent relapses, and treatment failureTimepoint: 12 months;The cumulative prednisolone requirement (mg/kg/day)Timepoint: 12 months;Types and rates of adverse effects, including leukopenia, neutropenia, thrombocytopenia, anemia; abdominal pain, diarrhea, vomiting; seizures; alopecia and elevated hepatic transaminases; infectionsTimepoint: 12 months;Changes in anthropometric and blood pressure standard deviation scoresTimepoint: 12 months;In a subset of 20 patients administered MMF, to perform therapeutic drug monitoring for mycophenolic acid (area under the curve, trough level) at 2-weeks, 6-months and at relapseTimepoint: 6 months;In a subset of 20 patients adminstered either agent, compare the proportions of immune cell subsets in peripheral blood of patientsTimepoint: 12 months

—

Countries

India

Contacts

Public ContactAditi Sinha

All India Institute of Medical Sciences

aditisinhaaiims@gmail.com9899145489

Outcome results

None listed

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