Idiopathic Nephrotic SyndromePhase 3NEPHROMYCYNCT01092962
Cyclophosphamide Versus Mycophenolate Mofetil for the Treatment of Steroid-dependent Nephrotic Syndrome in Children
A Phase 3 study testing Cyclophosphamide and Mycophenolate mofetil in people with Idiopathic Nephrotic Syndrome, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
Trial 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. Read more about this study
- Who is it for?
- Ages 2 to 16 years
- Conditions the study is for
- Idiopathic Nephrotic Syndrome
- What is being tested?
- Cyclophosphamide and Mycophenolate mofetil
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 1 siteFrance
- Study dates
- Sep 2010to Feb 2015
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Cyclophosphamide Versus Mycophenolate Mofetil for Children With Steroid-dependent Idiopathic Nephrotic Syndrome : a Multicenter Randomized Controlled Trial
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.
Read the 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
- Being tested
- Cyclophosphamide, Mycophenolate mofetil
- Keywords
- Children, Mycophenolate mofetil, Cyclophosphamide, Idiopathic nephrotic steroid sensitive syndrome
- Medical subject headings
- Nephrosis, Lipoid
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CyclophosphamideDrug
Also called Endoxan (BAXTER)
2mg/kg/day during 12 weeks (cumulative dose 148mg/kg)
Also called Cellcept (Roche)
1200mg/m²/jour in two divided doses during 18 months
| Study group | Role | What they receive |
|---|---|---|
| Mycophenolate mofetil | Experimental | Mycophenolate mofetil Receives: Drug: Mycophenolate mofetil |
| Cyclophosphamide | Active comparator | Cumulative dose of 148mg/kg of cyclophosphamide in 84 days (2mg/kg/day during 12 weeks) Receives: Drug: Cyclophosphamide |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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
Measured: Months 1, 3, 6, 9, 12, 15, 18, 21, 24
Cumulative steroid dose received during the years before and under treatment
Measured: Months 1, 3, 6, 9, 12, 15, 18, 21, 24
Comparison of growth data, during the year before and under treatment
Measured: Months 1, 3, 6, 9, 12, 15, 18, 21, 24
Pharmacokinetics measurement of MPA and relation with efficacy in case of treatment with MMF
Measured: One month after beginning MMF
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Mar 2010 (estimated)
- Study startSep 2010
- Primary completionFeb 2015
- Study completionFeb 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Idiopathic Nephrotic SyndromeStudies of the same condition, open and under way.
- More trials run by Assistance Publique - Hôpitaux de ParisThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 70 participants (actual)
Sponsor and investigators
- Lead sponsor
- Assistance Publique - Hôpitaux de Paris
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Véronique BAUDOUIN, MD, Principal investigator, Assistance Publique - Hôpitaux de Paris
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01092962
- Sponsor's protocol ID
- P071221
- Acronym
- NEPHROMYCY
- Status last verified
- Aug 2013
- First submitted
- 26 Feb 2010
- Last updated
- 26 Mar 2015
- Results posted
- No
- Data monitoring committee
- Yes
Publications
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.
PubMed 39513526
Study at a glance
From the registry record, last updated by the sponsor on 26 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Cyclophosphamide Versus Mycophenolate Mofetil for Children With Steroid-dependent Idiopathic Nephrotic Syndrome : a Multicenter Randomized Controlled Trial
- ClinicalTrials.gov ID
- NCT01092962
- Acronym / sponsor ID
- NEPHROMYCY / P071221
- Conditions
- Idiopathic Nephrotic Syndrome
- Keywords
- Children, Mycophenolate mofetil, Cyclophosphamide, Idiopathic nephrotic steroid sensitive syndrome
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 70 participants (actual)
- Lead sponsor
- Assistance Publique - Hôpitaux de ParisUniversity, hospital or other
- Study start
- Sep 2010
- Primary completion
- Feb 2015 (actual)
- Study completion
- Feb 2015 (actual)
- First posted
- 25 Mar 2010 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Mycophenolate mofetil | Experimental | Drug: Mycophenolate mofetilMycophenolate mofetil |
| Cyclophosphamide | Active comparator | Drug: CyclophosphamideCumulative dose of 148mg/kg of cyclophosphamide in 84 days (2mg/kg/day during 12 weeks) |
Who may qualify
- Ages
- 2 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CyclophosphamideAlso: Endoxan (BAXTER)2mg/kg/day during 12 weeks (cumulative dose 148mg/kg) | Drug | Cyclophosphamide |
| Mycophenolate mofetilAlso: Cellcept (Roche)1200mg/m²/jour in two divided doses during 18 months | Drug | Mycophenolate mofetil |
Primary outcome
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
4 secondary outcomes
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Mar 2015.
- Derived from this study: PubMed 39513526Larkins 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.
Related trials for Idiopathic Nephrotic Syndrome
RecruitingPhase 2
Rituximab Plus Cyclosporine in Idiopathic Membranous Nephropathy
Nephrotic Syndrome, Proteinuria
Active, not recruitingPhase 3
Clinical Study of Rituximab for the Treatment for Idiopathic Membranous Nephropathy With Nephrotic Syndrome
Glomerulonephritis, Membranous, Nephrotic Syndrome，Idiopathic
Not yet recruiting
Comparison of Efficacy and Safety of Albumin Versus Fresh Frozen Plasma in Managing Diuretic Resistant Edema in Children With Idiopathic Nephrotic Syndrome.
Idiopathic Nephrotic Syndrome (INS)
Not yet recruitingEarly Phase 1
RD06-05 Universal CD19/BCMA CAR-T for Refractory Pediatric Autoimmune Diseases
Autoimmune Diseases, SLE - Systemic Lupus Erythematosus
RecruitingPhase 2
Atacicept in Multiple Glomerular Diseases
pMN, IgAN
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Véronique BAUDOUIN, MD principal investigator
Assistance Publique - Hôpitaux de Paris
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Cyclophosphamide and Mycophenolate mofetil in people with Idiopathic Nephrotic Syndrome, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 Mar 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01092962, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Mar 2015.