Mycophenolate Mofetil in Pediatric Steroid Dependent Nephrotic Syndrome

Study Overview
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Brief Summary
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Idiopathic nephrotic syndrome is generally responsive to steroid therapy, but some patients need other immunosuppressants to reduce steroid dependency. The long-term use should be restricted due to adverse effects of cyclosporine, such as hypertension and nephrotoxicity. Mycophenolate mofetil for steroid-dependent nephrotic syndrome has been reported to have similar efficacy and fewer undesirable effects to other drugs in mainly observational studies. To determine the efficacy of mycophenolate mofetil in the management of steroid-dependent nephrotic syndrome, the investigators designed this prospective randomized controlled study.

Official Title
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A Prospective, Randomized, Open-label Study Evaluating the Efficacy of Mycophenolate Mofetil in the Prevention of Relapse of Steroid Dependent Nephrotic Syndrome in Children

Conditions
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Nephrotic Syndrome

Intervention / Treatment
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* Drug: Mycophenolate mofetil


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Steroid-dependent patients who had been on calcineurin inhibitors continuously for more than 1 year in complete remission Exclusion Criteria: Hereditary or secondary nephrotic syndrome Estimated glomerular filtration rate (GFR) < 60 mg/min/1.73 m^2 Body weight <16 kg leukocytopenia (absolute neutrophil count < 2000/mm^3) or anemia(Hct < 25%) Severe gastrointestinal disease chronic or acute active infection (e.g. hepatitis B,C, herpes, varicella zoster) prior live vaccine inoculation within 6 weeks (from the study enrollment) GOT/GPT elevation or hyperbilirubinemia malignant disease Pregnant or Breast feeding Previous history of mycophenolate mofetil use Participation to other therapeutic trial within recent 3 months Deficiency of the enzyme hypoxanthine-guanine phosphoribosyltransferase

Ages Eligible for Study
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Minimum Age: 2 Years
Maximum Age: 24 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Mycophenolate mofetil<br> | Drug: Mycophenolate mofetil<br>* Mycophenolate mofetil 20 30mg/kg/day PO until nephrotic syndrome relapse (max. 1 year)<br>* Other names: Myrept capsule/tablet;|
| No Intervention: Control<br> | |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| relapse free time |  | 1 year | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| adverse effect |  | 1 year | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Mycophenolate mofetil, Immunosuppressive Agents, Nephrosis