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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier:
NCT01092962
First received: February 26, 2010
Last updated: September 2, 2013
Last verified: August 2013
  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
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

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 ] [ Designated as safety issue: Yes ]

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 ] [ 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 ]

Enrollment: 70
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)
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

Ages Eligible for Study:   2 Years to 16 Years
Genders Eligible for Study:   Both
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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: 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

No publications provided

Responsible Party: Assistance Publique - Hôpitaux de Paris
ClinicalTrials.gov Identifier: NCT01092962     History of Changes
Other Study ID Numbers: P071221
Study First Received: February 26, 2010
Last Updated: September 2, 2013
Health Authority: France: Ministry of Health

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

Additional relevant MeSH terms:
Nephrotic Syndrome
Nephrosis, Lipoid
Nephrosis
Kidney Diseases
Urologic Diseases
Cyclophosphamide
Mycophenolate mofetil
Mycophenolic Acid
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions
Antirheumatic Agents
Therapeutic Uses
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists
Antibiotics, Antineoplastic
Enzyme Inhibitors

ClinicalTrials.gov processed this record on July 10, 2014