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The goal of this clinical trial is to evaluate the efficacy and safety of rituximab(RTX) and mycophenolate mofetile(MMF) in the treatment of children with low-dose steroid-dependent nephrotic syndrome(SDNS).
Idiopathic nephrotic syndrome(INS) is the most common glomerular disease in childhood. Currently, steroids are the primary treatment, but there are significant steroid-related toxicity, such as growth disorders, behavior changes, obesity, Cushing's syndrome, eye disease, osteoporosis, etc.
Both MMF and RTX have been shown to be effective in the treatment of SDNS, and there is a lack of prospective controlled studies to explore the optimal treatment regimen for low-dose SDNS. Therefore, the investigators will conduct a single-center, randomized controlled trial to evaluate the efficacy and safety of twice-daily rituximab(RTX) versus mycophenolate mofetil(MMF) in the treatment of children with low-dose steroid-dependent nephrotic syndrome(SDNS).
After the start of the study, all participants will be screened consecutively and eligible participants will be included in the study. Bias of potential influencing factors will be addressed by inclusion as covariates in the statistical analysis. Independent clinical site monitoring to ensure the safety and integrity of clinical data while patients adhere to the study protocol will focus on source data documentation, strict adherence to data correctness and study procedures, such as randomization and treatment.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Rituximab | Experimental | 2 doses of rituximab 375 mg/m^2 (Maximum 500mg/day)at 6 months intervals |
|
| Mycophenolate Mofetil | Active Comparator | MMF 20~30mg/kg/day,BID |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Rituximab | Drug | 2 doses of rituximab 375 mg/m^2(maximum 500mg/day) at 6 months intervals. Half an hour before rituximab infusion: oral acetaminophen 15mg/kg, oral or intramuscular antihistamine, methylprednisolone 2mg/kg IV. Trimethoprim-sulfamethoxazole should be administered orally from the initiation of rituximab therapy until peripheral-blood B cell recovery to prevent pneumocystis infection. |
| Measure | Description | Time Frame |
|---|---|---|
| 12-month relapse-free survival rate | The rate of no relapse within 12 months. | 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| 6-month relapse-free survival | Relapse-free survival within 6 months. | 6 months |
| 6-month relapse-free survival rate | The rate of no relapse within 6 months. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Yang Haiping, Doctor | Contact | 8618983703661 | oyhp@hospital.cqmu.edu.cn |
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital of Chongqing Medical University | Recruiting | Chongqing | Chongqing Municipality | 400014 | China |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 39513526 | Derived | Larkins NG, Hahn D, Liu ID, Willis NS, Craig JC, Hodson EM. Non-corticosteroid immunosuppressive medications for steroid-sensitive nephrotic syndrome in children. Cochrane Database Syst Rev. 2024 Nov 8;11(11):CD002290. doi: 10.1002/14651858.CD002290.pub6. |
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| ID | Term |
|---|---|
| D009404 | Nephrotic Syndrome |
| ID | Term |
|---|---|
| D009401 | Nephrosis |
| D007674 | Kidney Diseases |
| D014570 | Urologic Diseases |
| D052776 | Female Urogenital Diseases |
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| ID | Term |
|---|---|
| D000069283 | Rituximab |
| D009173 | Mycophenolic Acid |
| ID | Term |
|---|---|
| D058846 | Antibodies, Monoclonal, Murine-Derived |
| D000911 | Antibodies, Monoclonal |
| D000906 | Antibodies |
| D007136 | Immunoglobulins |
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|
|
| Mycophenolate Mofetil | Drug | Mycophenolate Mofetil 20-30 mg/kg/day BID,then adjust the dosage of drugs(maximum 2g/day) to maintian the concentration for MPA-AUC is 30-50μg.h/ml. Total duration:1year. Steroids dose:1.5mg/kg(maximum 40mg) qod for 2 weeks and gradually taper by 0.3 mg/kg every 2 weeks |
|
|
| 6 months |
| 12-month relapse-free survival | Relapse-free survival within 12 months. | 12 months |
| Proportion of frequent relapses | The proportion of frequent relapses.Frequent relapsing NS:≥2 relapses per 6 months within 6 months of disease onset or ≥4 relapses per 12 months in any subsequent 12-month period. | Months 6,12 |
| Cumulative steroid dosage | The total dosage of steroid from the beginning to the end of the trial. | 12 months |
| Number of relapses within 0-12,0-6, and 7-12 months | Number of relapses within 0-6 months,7-12 months, and total within 0-12 months. | 12 months |
| Time of first relapse | The first time to relapse after patients taking part in this study. | 12 months |
| Adverse event | It is a binary variable(1/0).The varibale would be setted as "1" if any adverse events occours including obesity, hypertrichosis, psychological disorders,glaucoma,neutropenia,hypogammaglobulinemia, rituximab-related lung injury, fatal hepatitis reactivation, multifocal leukoencephalopathy,severe diarrhea, severe infection. Adverse events graded according to Common Terminology Criteria For Adverse Events (CTCAE v4.0) | 12 months |
| Proportion of participants who discontinued steroids | Proportion of participants who discontinued steroids at 12 months | 12 months |
| Height standard deviation score(SDS) | SDS of height at 6 and 12 months, absolute and relative changes in SDS from baseline to 12 months. | months 0,6,12 |
| Body mass index(BMI) | Weight and height will be combined to report BMI in kg/m^2. BMI at 6 and 12 months, absolute and relative changes in BMI from baseline to 12 months. | Months 0,6,12 |
| Quality of life score | The researchers assess the quality of life of the participants useing Pediatric Quality of Life Inventory (PedsQL). | Baseline to 12 months |
| Cost of treatment | The researchers calculate their cost during the study. | 12 months |
| D005261 |
| Female Urogenital Diseases and Pregnancy Complications |
| D000091642 | Urogenital Diseases |
| D052801 | Male Urogenital Diseases |
| D007162 | Immunoproteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D000602 | Amino Acids, Peptides, and Proteins |
| D012712 | Serum Globulins |
| D005916 | Globulins |
| D002208 | Caproates |
| D000144 | Acids, Acyclic |
| D002264 | Carboxylic Acids |
| D009930 | Organic Chemicals |
| D005227 | Fatty Acids |
| D008055 | Lipids |