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

NCT01092962 | Completed Phase 3 DMC

• 1 other identifier: P071221
• Study Type: interventional
• Target: 70
• Locations: 1 country
• Sites: 1

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.

Conditions

Idiopathic Nephrotic Syndrome

Keywords

Children; Mycophenolate mofetil; Cyclophosphamide; Idiopathic nephrotic steroid sensitive syndrome

Outcome Measures

Primary Outcomes (1)

• 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.

  Months 1, 3, 6, 9, 12, 15, 18, 21, 24

Secondary Outcomes (4)

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

  Months 1, 3, 6, 9, 12, 15, 18, 21, 24

• Cumulative steroid dose received during the years before and under treatment

  Months 1, 3, 6, 9, 12, 15, 18, 21, 24

• Comparison of growth data, during the year before and under treatment

  Months 1, 3, 6, 9, 12, 15, 18, 21, 24

• Pharmacokinetics measurement of MPA and relation with efficacy in case of treatment with MMF

  One month after beginning MMF

Study Arms (2)

Mycophenolate mofetil

EXPERIMENTAL

Mycophenolate mofetil

Drug: Mycophenolate mofetil

Cyclophosphamide

ACTIVE COMPARATOR

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

Drug: Cyclophosphamide

Interventions

Cyclophosphamide DRUG

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

Also known as: Endoxan (BAXTER)

Cyclophosphamide

Mycophenolate mofetil DRUG

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

Also known as: Cellcept (Roche)

Mycophenolate mofetil

Eligibility Criteria

• Age: 2 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Assistance Publique - Hôpitaux de Paris: lead

Study Sites (1)

Robert Debre Hospital, AP-HP

Paris, Paris, 75019, France

Location

Related Publications (1)

• 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.

  PMID: 39513526 DERIVED

MeSH Terms

Conditions

Nephrosis, Lipoid

Interventions

Cyclophosphamide; Mycophenolic Acid

Condition Hierarchy (Ancestors)

Nephrosis; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Caproates; Acids, Acyclic; Carboxylic Acids; Fatty Acids; Lipids

Study Officials

• Véronique BAUDOUIN, MD

  Assistance Publique - Hôpitaux de Paris

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 26, 2010
• First Posted: March 25, 2010
• Study Start: September 1, 2010
• Primary Completion: February 1, 2015
• Study Completion: February 1, 2015
• Last Updated: March 26, 2015

Record last verified: 2013-08

Locations

FR (1)