Back to resultsStudy of Initial Steroid Treatment in Young Children With Nephrotic Syndrome
Primary Purpose
Steroid-Sensitive Nephrotic Syndrome
Status
Withdrawn
Phase
Phase 3
Locations
China
Study Type
Interventional
Intervention
Prednisolone
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Steroid-Sensitive Nephrotic Syndrome
Eligibility Criteria
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
Sites / Locations
- Children's hospital of Fudan university
- Shanghai Children's Hospital
- Shanghai Children's Medical Center
- Xinhua Hospital, Shanghai Jiaotong University School of Medicine
Arms of the Study
Arm 1
Arm 2
Arm Type
Placebo Comparator
Experimental
Arm Label
3 months group
6 months group
Arm Description
Subjects will receive 12-weeks of placebo following randomization
12 Weeks of Prednisolone Therapy Subjects will add an additional 12 weeks of Prednisolone to follow pre-randomization standard of care prednisolone.
Outcomes
Primary Outcome Measures
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.
Secondary Outcome Measures
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
Time to first relapse (days)
Number of days from randomization to occurrence of first relapse
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
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.
Adverse events during 12-month period after 3 month standard treatment
Number of adverse events experienced, related or unrelated to corticosteroid use
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.
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.
Full Information
NCT ID
NCT04536181
First Posted
August 26, 2020
Last Updated
December 14, 2020
Sponsor
Children's Hospital of Fudan University
1. Study Identification
Unique Protocol Identification Number
NCT04536181
Brief Title
Study of Initial Steroid Treatment in Young Children With Nephrotic Syndrome
Official Title
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
Study Type
Interventional
2. Study Status
Record Verification Date
December 2020
Overall Recruitment Status
Withdrawn
Why Stopped
lack of funding
Study Start Date
January 1, 2021 (Anticipated)
Primary Completion Date
September 1, 2022 (Anticipated)
Study Completion Date
October 1, 2022 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Children's Hospital of Fudan University
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief Summary
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Steroid-Sensitive Nephrotic Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
0 (Actual)
8. Arms, Groups, and Interventions
Arm Title
3 months group
Arm Type
Placebo Comparator
Arm Description
Subjects will receive 12-weeks of placebo following randomization
Arm Title
6 months group
Arm Type
Experimental
Arm Description
12 Weeks of Prednisolone Therapy Subjects will add an additional 12 weeks of Prednisolone to follow pre-randomization standard of care prednisolone.
Intervention Type
Drug
Intervention Name(s)
Prednisolone
Other Intervention Name(s)
prednisone
Intervention Description
12 weeks prednisolone treatment
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
12 weeks simulated tablets
Primary Outcome Measure Information:
Title
Occurrence of frequent relapses of nephrotic syndrome at 12th month after 3 month standard treatment
Description
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.
Time Frame
12 month period after 3 month standard treatment
Secondary Outcome Measure Information:
Title
Number of relapses during 12 months follow up after 3 month standard treatment
Description
Number of nephrotic syndrome relapses per patient year during the 12-month period after 3 month standard treatment
Time Frame
12 month period after 3 month standard treatment
Title
Time to first relapse (days)
Description
Number of days from randomization to occurrence of first relapse
Time Frame
12 month period after 3 month standard treatment
Title
Cumulative prednisolone [or corticosteroid equivalent] received during 12 month period after 3 month standard treatment
Description
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
Time Frame
12 month period after 3 month standard treatment
Title
The use of steroid-sparing medications
Description
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.
Time Frame
12 month period after 3 month standard treatment
Title
Adverse events during 12-month period after 3 month standard treatment
Description
Number of adverse events experienced, related or unrelated to corticosteroid use
Time Frame
12 month period after 3 month standard treatment
Title
Change in height during 12-month period after 3 month standard treatment
Description
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.
Time Frame
12 month period after 3 month standard treatment
Title
Occurrence of steroid-dependent nephrotic syndrome at 12th month after 3 month standard treatment
Description
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.
Time Frame
12 month period after 3 month standard treatment
10. Eligibility
Sex
All
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
6 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Hong Xu, PhD.MD.
Organizational Affiliation
Children's Hospital of Fudan University
Official's Role
Principal Investigator
Facility Information:
Facility Name
Children's hospital of Fudan university
City
Shanghai
State/Province
Shanghai
ZIP/Postal Code
200000
Country
China
Facility Name
Shanghai Children's Hospital
City
Shanghai
Country
China
Facility Name
Shanghai Children's Medical Center
City
Shanghai
Country
China
Facility Name
Xinhua Hospital, Shanghai Jiaotong University School of Medicine
City
Shanghai
Country
China
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
Study of Initial Steroid Treatment in Young Children With Nephrotic Syndrome
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