A Multi-center Randomized Controlled Trial of Rituximab for Refractory Nephrotic Syndrome in Children
试验速览
- 阶段
- 2 期
- 发起方
- 入组人数
- 88
- 试验地点
- 1
- 主要终点
- The remission rate of steroid-resistant nephrotic syndrome patients after rituximab treatment
研究概览
简要总结
Anti-CD20 agent has been proposed as a rescue therapy for refractory nephrotic syndrome(NS) on the basis of favorable clinical observations. Yet the long-term effect on maintaining remission or the likelihood of becoming rituximab-dependent is unclear and the information on the safety profile of rituximab is limited. This trial was designed to investigate the safety and efficacy of Rituximab in children with refractory NS.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Single (Participant)
入排标准
- 年龄范围
- 1 Year 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •(A)steroid/calcineurin inhibitor resistant nephrotic syndrome
- •steroid resistance: no remission after 4 weeks of daily oral corticosteroid (60mg/m2/day)
- •calcineurin inhibitor resistance: no remission after 3 months of therapeutic dose administration of cyclosporine and/or tacrolimus
- •no remission defined by persistent proteinuria of nephrotic range for the last 3 months
- •post-transplant patients were included in the study
- •(B)steroid-dependent nephrotic syndrome
- •Steroid-dependent patients who had been on various known medications (such as corticosteroids, cyclophosphamide, chlorambucil, calcineurin inhibitors, levamisole ..) continuously for more than 2 years
- •definition of dependency: more than two consecutive relapse events in 2 weeks after discontinuation of steroid or calcineurin inhibitor
- •no improvement in relapsing frequency with calcineurin inhibitor use
- •unable to continue with calcineurin inhibitor due to side effects
- •unable to continue with calcineurin inhibitor due to prolonged use (over 2 years)
- •other conditions in which the clinician considers difficult to control disease with steroids or calcineurin inhibitors only.
排除标准
- •previous rituximab use
- •secondary nephrotic syndrome
- •estimated GFR <60mL/min/1.73m2 or under 50% of age-matched standard GFR
- •chronic or acute active infection (e.g. hepatitis B,C, herpes, varicella zoster)
- •prior live vaccine inoculation within 1 month (from the study enrollment)
- •cardiovascular diseases, pulmonary or pleural diseases
- •uncontrolled hypertension
- •leukocytopenia (absolute neutrophil count <1500/mm3) or thrombocytopenia (<75000/mm3)
- •pregnancy
研究组 & 干预措施
steroid-resistant
Steroid-resistant group: n=27 , enroll all for treatment
干预措施: Rituximab (Drug)
steroid-dependent-rituximab
steroid-responsive group: n=38
干预措施: Rituximab (Drug)
steroid-dependent-placebo
steroid-responsive group: n=23
干预措施: Placebo (Drug)
结局指标
主要结局
The remission rate of steroid-resistant nephrotic syndrome patients after rituximab treatment
时间窗: within 6 months from the initiation of treatment
The rate of maintaining remission in steroid-dependent nephrotic syndrome patients
时间窗: within 6 months from the initiation of treatment versus placebo control
次要结局
未报告次要终点
研究者
Hee Gyung Kang
Associate Professor
Seoul National University Childrens Hospital