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

NCT ID: NCT01092962

Last Updated: 2015-03-26

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 70 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2010-09-30
• Study Completion Date: 2015-02-28

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.

Conditions

Idiopathic Nephrotic Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Mycophenolate mofetil

Mycophenolate mofetil

Group Type: EXPERIMENTAL

Mycophenolate mofetil

Intervention Type: DRUG

1200mg/m²/jour in two divided doses during 18 months

Cyclophosphamide

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

Group Type: ACTIVE_COMPARATOR

Cyclophosphamide

Intervention Type: DRUG

2mg/kg/day during 12 weeks (cumulative dose 148mg/kg)

Interventions

Cyclophosphamide

2mg/kg/day during 12 weeks (cumulative dose 148mg/kg)

Intervention Type: DRUG

Mycophenolate mofetil

1200mg/m²/jour in two divided doses during 18 months

Intervention Type: DRUG

Other Intervention Names

Endoxan (BAXTER); Cellcept (Roche)

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

• Minimum Eligible Age: 2 Years
• Maximum Eligible Age: 16 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Assistance Publique - Hôpitaux de Paris

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Véronique BAUDOUIN, MD

Role: PRINCIPAL_INVESTIGATOR

Assistance Publique - Hôpitaux de Paris

Locations

Robert Debre Hospital, AP-HP

Paris, Paris, France

Countries

France

References

Larkins NG, Hahn D, Liu ID, Willis NS, Craig JC, Hodson EM. Non-corticosteroid immunosuppressive medications for steroid-sensitive nephrotic syndrome in children. Cochrane Database Syst Rev. 2024 Nov 8;11(11):CD002290. doi: 10.1002/14651858.CD002290.pub6.

Reference Type: DERIVED

PMID: 39513526 (View on PubMed)

Other Identifiers

P071221

Identifier Type: -

Identifier Source: org_study_id