Study of Initial Steroid Treatment in Young Children With Nephrotic Syndrome

Study Overview
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Brief Summary
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This study is a multicentric, prospective, randomized, double blind, placebo controlled trial compared the efficacy of 6 months vs. 3 months of initial prednisolone therapy in decreasing the incidence of FRNS with steroid-sensitive nephrotic syndrome in children age 1 year up to 6 years. Three months tapering prednisolone or placebo are administrated follow by open-label 3 months standard prednisolone therapy.The cumulative incidence of FRNS and adverse events for one year were evaluated in the 3-month and 6-month groups.

Official Title
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Comparative Study of 3 or 6 Months Initial Steroid Treatment in Children Under 6 Years of Age With Steroid Sensitive Nephrotic Syndrome：a Randomized, Double-blind, Placebo-controlled Study

Conditions
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Steroid-Sensitive Nephrotic Syndrome

Intervention / Treatment
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* Drug: Prednisolone
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Idiopathic, steroid-sensitive, first episode of nephrotic syndrome Age 12 months up to 6 years Written informed consent Exclusion Criteria: Nephrotic syndrome known to be secondary to a systemic disorder, e.g., Immunoglobulin A (IgA) nephropathy, systemic lupus erythematosus, Henoch Schonlein purpura, vasculitis, , hepatitis B or Alport syndrome. Therapy with prednisolone for prior episodes of nephrotic syndrome Persistent estimated glomerular filtration rate (GFR) <75 ml/min/1.73 m2 Patients who show relapse during the first 3 months of pre-randomization corticosteroid therapy for nephrotic syndrome Patients with initial steroid resistance Patients who are allergic to glucocorticoids The compliance of patients or their guardians is poor

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 6 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: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: 3 months group<br>Subjects will receive 12-weeks of placebo following randomization | Drug: Placebo<br>* 12 weeks simulated tablets<br>|
| Experimental: 6 months group<br>12 Weeks of Prednisolone Therapy Subjects will add an additional 12 weeks of Prednisolone to follow pre-randomization standard of care prednisolone. | Drug: Prednisolone<br>* 12 weeks prednisolone treatment<br>* Other names: prednisone;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Occurrence of frequent relapses of nephrotic syndrome at 12th month after 3 month standard treatment | Proportion of patients with frequent relapses at 12th month after 3 month standard treatment.Frequent relapses nephrotic syndrome was defined as two or more relapses within 6 months after completing initial treatment, or four relapses within any period of 12 months, including relapses during initial treatment. | 12 month period after 3 month standard treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Number of relapses during 12 months follow up after 3 month standard treatment | Number of nephrotic syndrome relapses per patient year during the 12-month period after 3 month standard treatment | 12 month period after 3 month standard treatment | 
| Time to first relapse (days) | Number of days from randomization to occurrence of first relapse | 12 month period after 3 month standard treatment | 
| Cumulative prednisolone [or corticosteroid equivalent] received during 12 month period after 3 month standard treatment | Total amount of prednisolone [or corticosteroid equivalent] received, as mg/kg/day or mg/m2/day as intervention and for treatment of relapses, during 12 months follow up after 3 month standard treatment | 12 month period after 3 month standard treatment | 
| The use of steroid-sparing medications | The proportion of patients in each study arm treated with steroid-sparing strategies or medications.it is a binary varibale (1/0). The variable wolud be sette into 1, if the patients use the steroid-sparing medications such as cyclophosphamide, levamisole, mycophenolate mofetil，rituximab and so on. | 12 month period after 3 month standard treatment | 
| Adverse events during 12-month period after 3 month standard treatment | Number of adverse events experienced, related or unrelated to corticosteroid use | 12 month period after 3 month standard treatment | 
| Change in height during 12-month period after 3 month standard treatment | The standard deviation scores (SDS) for height will be measured at 12th month and randomization. Change in height is SDS at 12 month minus that of randomization. | 12 month period after 3 month standard treatment | 
| Occurrence of steroid-dependent nephrotic syndrome at 12th month after 3 month standard treatment | Proportion of patients with steroid-dependent nephrotic syndrome at 12th month after 3 month standard treatment.Steroid-dependent nephrotic syndrome was defined as steroid-sensitive nephrotic syndrome with 2 or more consecutive relapses during tapering or within 14 days of stopping steroids. | 12 month period after 3 month standard treatment |