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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT01092962
First Posted: March 25, 2010
Last Update Posted: March 26, 2015
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris
  Purpose

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.


Condition Intervention Phase
Idiopathic Nephrotic Syndrome Drug: Cyclophosphamide Drug: Mycophenolate mofetil Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Cyclophosphamide Versus Mycophenolate Mofetil for Children With Steroid-dependent Idiopathic Nephrotic Syndrome : a Multicenter Randomized Controlled Trial

Resource links provided by NLM:


Further study details as provided by Assistance Publique - Hôpitaux de Paris:

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. [ Time Frame: Months 1, 3, 6, 9, 12, 15, 18, 21, 24 ]

Secondary Outcome Measures:
  • 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 ]
  • Cumulative steroid dose received during the years before and under treatment [ Time Frame: Months 1, 3, 6, 9, 12, 15, 18, 21, 24 ]
  • Comparison of growth data, during the year before and under treatment [ Time Frame: Months 1, 3, 6, 9, 12, 15, 18, 21, 24 ]
  • Pharmacokinetics measurement of MPA and relation with efficacy in case of treatment with MMF [ Time Frame: One month after beginning MMF ]

Enrollment: 70
Study Start Date: September 2010
Study Completion Date: February 2015
Primary Completion Date: February 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Mycophenolate mofetil
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)
Drug: Cyclophosphamide
2mg/kg/day during 12 weeks (cumulative dose 148mg/kg)
Other Name: Endoxan (BAXTER)

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

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Ages Eligible for Study:   2 Years to 16 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01092962


Locations
France
Robert Debre Hospital, AP-HP
Paris, France, 75019
Sponsors and Collaborators
Assistance Publique - Hôpitaux de Paris
Investigators
Principal Investigator: Véronique BAUDOUIN, MD Assistance Publique - Hôpitaux de Paris
  More Information

Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT01092962     History of Changes
Other Study ID Numbers: P071221
First Submitted: February 26, 2010
First Posted: March 25, 2010
Last Update Posted: March 26, 2015
Last Verified: August 2013

Keywords provided by Assistance Publique - Hôpitaux de Paris:
Children
Mycophenolate mofetil
Cyclophosphamide
Idiopathic nephrotic steroid sensitive syndrome

Additional relevant MeSH terms:
Syndrome
Nephrotic Syndrome
Nephrosis
Nephrosis, Lipoid
Disease
Pathologic Processes
Kidney Diseases
Urologic Diseases
Cyclophosphamide
Mycophenolic Acid
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Antibiotics, Antineoplastic
Antibiotics, Antitubercular
Antitubercular Agents
Anti-Bacterial Agents
Anti-Infective Agents
Enzyme Inhibitors