Cyclophosphamide Versus Mycophenolate Mofetil for the Treatment of Steroid-dependent Nephrotic Syndrome in Children (NEPHROMYCY)
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.
Idiopathic Nephrotic Syndrome
Drug: Mycophenolate mofetil
|Study Design:||Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
|Official Title:||Cyclophosphamide Versus Mycophenolate Mofetil for Children With Steroid-dependent Idiopathic Nephrotic Syndrome : a Multicenter Randomized Controlled Trial|
- 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. [ Time Frame: Months 1, 3, 6, 9, 12, 15, 18, 21, 24 ] [ Designated as safety issue: Yes ]
- In case of relapse, steroid threshold dose to maintain a remission compare to those before inclusion in the study [ Time Frame: Months 1, 3, 6, 9, 12, 15, 18, 21, 24 ] [ Designated as safety issue: Yes ]
- Cumulative steroid dose received during the years before and under treatment [ Time Frame: Months 1, 3, 6, 9, 12, 15, 18, 21, 24 ] [ Designated as safety issue: Yes ]
- Comparison of growth data, during the year before and under treatment [ Time Frame: Months 1, 3, 6, 9, 12, 15, 18, 21, 24 ] [ Designated as safety issue: Yes ]
- Pharmacokinetics measurement of MPA and relation with efficacy in case of treatment with MMF [ Time Frame: One month after beginning MMF ] [ Designated as safety issue: Yes ]
|Study Start Date:||September 2010|
|Estimated Study Completion Date:||September 2014|
|Estimated Primary Completion Date:||September 2014 (Final data collection date for primary outcome measure)|
Experimental: Mycophenolate mofetil
Drug: Mycophenolate mofetil
1200mg/m²/jour in two divided doses during 18 months
Other Name: Cellcept (Roche)
Active Comparator: Cyclophosphamide
Cumulative dose of 148mg/kg of cyclophosphamide in 84 days (2mg/kg/day during 12 weeks)
2mg/kg/day during 12 weeks (cumulative dose 148mg/kg)
Other Name: Endoxan (BAXTER)
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.
Please refer to this study by its ClinicalTrials.gov identifier: NCT01092962
|Robert Debre Hospital, AP-HP|
|Paris, France, 75019|
|Principal Investigator:||Véronique BAUDOUIN, MD||Assistance Publique - Hôpitaux de Paris|