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Cyclophosphamide Versus Mycophenolate Mofetil for the Treatment of Steroid-dependent Nephrotic Syndrome in Children (NEPHROMYCY)

Primary Purpose

Idiopathic Nephrotic Syndrome

Status
Completed
Phase
Phase 3
Locations
France
Study Type
Interventional
Intervention
Cyclophosphamide
Mycophenolate mofetil
Sponsored by
Assistance Publique - Hôpitaux de Paris
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Idiopathic Nephrotic Syndrome focused on measuring Children, Mycophenolate mofetil, Cyclophosphamide, Idiopathic nephrotic steroid sensitive syndrome

Eligibility Criteria

2 Years - 16 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Robert Debre Hospital, AP-HP

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

Mycophenolate mofetil

Cyclophosphamide

Arm Description

Mycophenolate mofetil

Cumulative dose of 148mg/kg of cyclophosphamide in 84 days (2mg/kg/day during 12 weeks)

Outcomes

Primary Outcome Measures

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 Measures

In case of relapse, steroid threshold dose to maintain a remission compare to those before inclusion in the study
Cumulative 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

Full Information

First Posted
February 26, 2010
Last Updated
March 25, 2015
Sponsor
Assistance Publique - Hôpitaux de Paris
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1. Study Identification

Unique Protocol Identification Number
NCT01092962
Brief Title
Cyclophosphamide Versus Mycophenolate Mofetil for the Treatment of Steroid-dependent Nephrotic Syndrome in Children
Acronym
NEPHROMYCY
Official Title
Cyclophosphamide Versus Mycophenolate Mofetil for Children With Steroid-dependent Idiopathic Nephrotic Syndrome : a Multicenter Randomized Controlled Trial
Study Type
Interventional

2. Study Status

Record Verification Date
August 2013
Overall Recruitment Status
Completed
Study Start Date
September 2010 (undefined)
Primary Completion Date
February 2015 (Actual)
Study Completion Date
February 2015 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Assistance Publique - Hôpitaux de Paris

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief 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.
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Idiopathic Nephrotic Syndrome
Keywords
Children, Mycophenolate mofetil, Cyclophosphamide, Idiopathic nephrotic steroid sensitive syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
70 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Mycophenolate mofetil
Arm Type
Experimental
Arm Description
Mycophenolate mofetil
Arm Title
Cyclophosphamide
Arm Type
Active Comparator
Arm Description
Cumulative dose of 148mg/kg of cyclophosphamide in 84 days (2mg/kg/day during 12 weeks)
Intervention Type
Drug
Intervention Name(s)
Cyclophosphamide
Other Intervention Name(s)
Endoxan (BAXTER)
Intervention Description
2mg/kg/day during 12 weeks (cumulative dose 148mg/kg)
Intervention Type
Drug
Intervention Name(s)
Mycophenolate mofetil
Other Intervention Name(s)
Cellcept (Roche)
Intervention Description
1200mg/m²/jour in two divided doses during 18 months
Primary Outcome Measure Information:
Title
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
Secondary Outcome Measure Information:
Title
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
Title
Cumulative steroid dose received during the years before and under treatment
Time Frame
Months 1, 3, 6, 9, 12, 15, 18, 21, 24
Title
Comparison of growth data, during the year before and under treatment
Time Frame
Months 1, 3, 6, 9, 12, 15, 18, 21, 24
Title
Pharmacokinetics measurement of MPA and relation with efficacy in case of treatment with MMF
Time Frame
One month after beginning MMF

10. Eligibility

Sex
All
Minimum Age & Unit of Time
2 Years
Maximum Age & Unit of Time
16 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Véronique BAUDOUIN, MD
Organizational Affiliation
Assistance Publique - Hôpitaux de Paris
Official's Role
Principal Investigator
Facility Information:
Facility Name
Robert Debre Hospital, AP-HP
City
Paris
ZIP/Postal Code
75019
Country
France

12. IPD Sharing Statement

Learn more about this trial

Cyclophosphamide Versus Mycophenolate Mofetil for the Treatment of Steroid-dependent Nephrotic Syndrome in Children

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