Study of Initial Steroid Treatment in Young Children With Nephrotic Syndrome

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

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.

Study Overview

• Status: Withdrawn
• Conditions: Steroid-Sensitive Nephrotic Syndrome
• Intervention / Treatment: Drug: Placebo; Drug: Prednisolone

• Study Type: Interventional
• Phase: Phase 3

Contacts and Locations

Study Locations

• China
  • Shanghai, China
    • Shanghai Children's Medical Center
  • Shanghai, China
    • Shanghai Children's Hospital
  • Shanghai, China
    • Xinhua Hospital, Shanghai Jiaotong University School of Medicine
  • Shanghai
    • Shanghai, Shanghai, China, 200000
      • Children's Hospital of Fudan University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 6 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Children's Hospital of Fudan University
• Investigators: Principal Investigator: Hong Xu, PhD.MD., Children's Hospital of Fudan University

Study record dates

Study Major Dates

• Study Start (Anticipated): January 1, 2021
• Primary Completion (Anticipated): September 1, 2022
• Study Completion (Anticipated): October 1, 2022

Study Registration Dates

• First Submitted: August 26, 2020
• First Submitted That Met QC Criteria: August 28, 2020
• First Posted (Actual): September 2, 2020

Study Record Updates

• Last Update Posted (Actual): December 17, 2020
• Last Update Submitted That Met QC Criteria: December 14, 2020
• Last Verified: December 1, 2020

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Kidney Diseases; Urologic Diseases; Disease; Hypersensitivity; Syndrome; Nephrotic Syndrome; Nephrosis; Physiological Effects of Drugs; Anti-Inflammatory Agents; Antineoplastic Agents; Glucocorticoids; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Antineoplastic Agents, Hormonal; Prednisolone; Prednisone
• Other Study ID Numbers: CSICS

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No