Efficacy and Safety of Rituximab Versus Mycophenolate Mofetil in Children With Steroid-dependent Nephrotic Syndrome

Phase 2RecruitingNCT05843968

Children's Hospital of Chongqing Medical University46 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Nephrotic Syndrome in Children

Interventions

RituximabDRUG

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.

Mycophenolate MofetilDRUG

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

Eligibility

Sex: ALLMin age: 3 YearsMax age: 16 Years

Medical Language ↔ Plain English

Inclusion Criteria: * Children with a definite diagnosis of SDNS are included in the study during relapse treatment. * Age 3-16 years. * Steroid dependent dose≤0.3mg/kg/day. * Cumulative steroid use for ≥6 months. * Ability to swallow tablet. * Guardians understand the characteristics and personal consequences of clinical trial. * Guardians willing to give informed written consent. Exclusion Criteria: * Diagnosis of secondary NS, such as secondary to lupus nephritis, hepatitis B-related nephritis, purpura nephritis, etc. * Anti-neutrophil cytoplasmic antibodies(ANCA) positive or complement C3 level decreased. * Diagnosis of hereditary nephrotic syndrome. * Full dose of prednisone (2mg/kg/day, maximum 60mg) are used for 14 days after relapse and urine protein don't turn negative. * Estimated glomerular filtration rate (eGFR) \<90mL/min per 1.73m\^2 at study entry. * Those who with a known allergy to Mycophenolate Mofetil and their excipients or to Rituximab and its excipients. * Those who refuse to participate in the trial. * Those who participate other clinical trials. * Those who with positive HBV serological markers (HBsAg or/and HBeAg or/and HBcAb), HCV positive patients or patients with abnormal liver function (ALT,AST,or bilirubin\>2 or more times the upper limit of the normal range and persistently elevated for 2 weeks). * Severe leukopenia (white blood cells\<3.0×10\^9), severe anemia (hemoglobin\<90g/l), and thrombocytopenia (platelets\<100×10\^9) at study entry. * History of pancreatitis or definite gastrointestinal ulcers and/or gastrointestinal bleeding within 6 months. * Those who with congenital or acquired immune deficiency, or with active tuberculosis, active CMV and other infections. * Those who with other serious physical or mental illnesses. * History of malignant tumor within 5 years. * Those who with congenital heart disease, arrhythmia, heart failure and other serious cardiovascular diseases. * Those who with serious infections requiring intravenous antibiotics.

Locations (1)

Children's Hospital of Chongqing Medical University

Chongqing, Chongqing Municipality, China

RECRUITING

Outcomes

Primary Outcomes

12-month relapse-free survival rate

The rate of no relapse within 12 months.

Time frame: 12 months

Secondary Outcomes

6-month relapse-free survival

Relapse-free survival within 6 months.

Time frame: 6 months

6-month relapse-free survival rate

The rate of no relapse within 6 months.

Time frame: 6 months

12-month relapse-free survival

Relapse-free survival within 12 months.

Time frame: 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.

Time frame: Months 6,12

Cumulative steroid dosage

The total dosage of steroid from the beginning to the end of the trial.

Time frame: 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.

Time frame: 12 months

Time of first relapse

The first time to relapse after patients taking part in this study.

Time frame: 12 months

Central Contacts

Yang Haiping, Doctor

CONTACT

8618983703661 oyhp@hospital.cqmu.edu.cn

Data from ClinicalTrials.gov

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