Cyclophosphamide Versus Mycophenolate Mofetil for the Treatment of Steroid-dependent Nephrotic Syndrome in Children
Information source: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov processed this data on August 23, 2015 Link to the current ClinicalTrials.gov record.
Condition(s) targeted: Idiopathic Nephrotic Syndrome
Intervention: Cyclophosphamide (Drug); Mycophenolate mofetil (Drug)
Phase: Phase 3
Status: Completed
Sponsored by: Assistance Publique - Hôpitaux de Paris Official(s) and/or principal investigator(s): Véronique BAUDOUIN, MD, Principal Investigator, Affiliation: Assistance Publique - Hôpitaux de Paris
Summary
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.
Clinical Details
Official title: Cyclophosphamide Versus Mycophenolate Mofetil for Children With Steroid-dependent Idiopathic Nephrotic Syndrome : a Multicenter Randomized Controlled Trial
Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment
Primary outcome: 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.
Secondary outcome: In case of relapse, steroid threshold dose to maintain a remission compare to those before inclusion in the studyCumulative steroid dose received during the years before and under treatment Comparison of growth data, during the year before and under treatment Pharmacokinetics measurement of MPA and relation with efficacy in case of treatment with MMF
Detailed description:
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.
Eligibility
Minimum age: 2 Years.
Maximum age: 16 Years.
Gender(s): Both.
Criteria:
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
Locations and Contacts
Robert Debre Hospital, AP-HP, Paris 75019, France
Additional Information
Starting date: September 2010
Last updated: March 25, 2015
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