Cyclophosphamide Versus Mycophenolate Mofetil for Children With Steroid-dependent Idiopathic Nephrotic Syndrome : a Multicenter Randomized Controlled Trial
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 70
- 试验地点
- 1
- 主要终点
- Relapse of nephrotic syndrome (defined by occurrence of proteinuria ≥ 0,25 g/mmol of CREATININURIA (or ≥ 2g/g) with hypoalbuminemia ≤ 30g/L AND/OR dipsticks >2+ during 3 days and proteinuria/CREATININURIA ratio ≥ 0,25 g/mmol) during 2 years.
研究概览
简要总结
Idiopathic nephrotic syndrome is steroid-sensitive in more than 90% of cases in children. However 60% of cases are steroid dependent and required treatment with immunosuppressive agent. Cyclophosphamide and ciclosporin are used for long time to reduce steroid dependency, but duration of these treatments should be restricted because of gonadotoxicity for cyclophosphamide and nephrotoxicity for ciclosporin.
Mycophenolate mofetil appears as an alternative treatment without gonadotoxicity and nephrotoxicity. However, contrary to cyclophosphamide, mycophenolate mofetil does not seem to have a residual action so that treatment must be maintained during months or years.
The aim of the study is to compare efficacy of cyclophosphamide and mycophenolate mofetil in steroid dependent nephrotic syndrome in children.
详细描述
Aim of the study: Comparison of efficacy of cyclophosphamide 148mg/kg in 12 weeks and mycophenolate mofetil 1200mg/m² during 18 months, in children with steroid dependent nephrotic syndrome.
The 70 patients will be recruited in the 26 centres of paediatric nephrology in France, included and randomized at the time of a relapse. They will receive the same steroid treatment in the 2 arms.
The primary point will be occurrence of a relapse during the 24 months of follow-up. Detection of relapse will be done by using dipsticks and confirm by biological dosages (albuminemia and proteinuria/CREATININURIA ratio). Clinical and biological check up will be done every 3 months during all the study.
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
- None
入排标准
- 年龄范围
- 2 Years 至 16 Years（Child）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •children 2 to 16 years old
- •steroid dependency ≥30mg/m² eod
- •or steroid dependency ≥15mg/m² eod and occurrence of : at least 2 relapses in 1 year, adverse event of steroid therapy (height rate ≤-1SD, obesity, other complication) or severe complication of nephrotic syndrome (thrombosis, collapse, severe infection,...)
- •inform consent
排除标准
- •steroid resistant nephrotic syndrome
- •prior treatment with cyclophosphamide, mycophenolate mofetil or cyclosporine
- •absence of contraception in pubescent girls
- •allergy to cyclophosphamide or mycophenolate mofetil
- •malignant disease
- •treatment with other immunosuppressant treatment or with non-steroid anti-inflammatory or anti proteinuric medication (enzyme converse antagonist and angiotensin II receptor antagonist)
- •absence of inform consent
- •participation to other therapeutic trial
研究组 & 干预措施
Mycophenolate mofetil
Mycophenolate mofetil
干预措施: Mycophenolate mofetil (Drug)
Cyclophosphamide
Cumulative dose of 148mg/kg of cyclophosphamide in 84 days (2mg/kg/day during 12 weeks)
干预措施: Cyclophosphamide (Drug)
结局指标
主要结局
Relapse of nephrotic syndrome (defined by occurrence of proteinuria ≥ 0,25 g/mmol of CREATININURIA (or ≥ 2g/g) with hypoalbuminemia ≤ 30g/L AND/OR dipsticks >2+ during 3 days and proteinuria/CREATININURIA ratio ≥ 0,25 g/mmol) during 2 years.
时间窗: Months 1, 3, 6, 9, 12, 15, 18, 21, 24
次要结局
- In case of relapse, steroid threshold dose to maintain a remission compare to those before inclusion in the study(Months 1, 3, 6, 9, 12, 15, 18, 21, 24)
- Cumulative steroid dose received during the years before and under treatment(Months 1, 3, 6, 9, 12, 15, 18, 21, 24)
- Comparison of growth data, during the year before and under treatment(Months 1, 3, 6, 9, 12, 15, 18, 21, 24)
- Pharmacokinetics measurement of MPA and relation with efficacy in case of treatment with MMF(One month after beginning MMF)