Childhood Idiopathic Nephrotic SyndromePhase 2/3NEPHRUTIXNCT01268033
Efficacy of Rituximab For the Treatment of Calcineurin Inhibitors Dependent Nephrotic Syndrome During Childhood
A Phase 2/3 study testing Rituximab in people with Childhood Idiopathic Nephrotic Syndrome, run by University Hospital, Limoges at 24 sites in 2 countries.
Trial summary
Background Idiopathic nephrotic syndrome is a rare disease beginning during childhood and treated with immunosuppressants (i.e. steroids, mycophenolate mofetil, cyclophosphamide, cyclosporine). Read more about this study
- Who is it for?
- Ages 2 to 18 years
- Conditions the study is for
- Childhood Idiopathic Nephrotic Syndrome
- What is being tested?
- Rituximab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 26 participants
- Where
- 24 sitesin 2 countries
- Study dates
- Dec 2010to May 2014
- Phase
- Phase 2/3
- 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 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Male or Female patients over 2 and under 18 years, with an idiopathic nephrotic syndrome (NS)
- Steroid Sensitive Nephrotic Syndrome (according to the French pediatric protocol).
- NEPHRUTIX
- Calcineurin inhibitor Dependent NS or NS for which anticalcineurin treatment has not been effective. Others immunosuppressive treatments (MMF) must have failed to control the disease activity.
- Effective contraception for girls of childbearing age.
- The patient is able to understand and has signed a written informed consent OR the parent or legal guardian is able to understand and has signed a written informed consent, which must be obtained prior to the initiation of any study procedure
Who cannot join
- Terminal renal failure requiring dialysis/transplantation
- Transcutaneous oxygen stauration < 97%
- Clinical or Radiological brochopulmonar or pleural abnormality
- Asymptomatic carrier of Hepatitis B virus our history of Hepatitis B
- Contraindication to Rituximab (RTX)
- Parents/patient refusing to participate in the study
Trial locations and contacts
24 sites in 2 countries.
24 sites in 2 countries
France23 sites
Chu Amiens
Amiens, Amiens, 80054
Chu Besancon
Besançon, Besancon, 25030
Chu Bordeaux
Bordeaux, Bordeaux, 33076
Chu Brest
Brest, Brest, 29609
CHU CAEN
Caen, Caen, 14033
Chu Clermont Ferrand
Clermont-Ferrand, Clermont Ferrand, 63058
Chu Grenoble
Grenoble, Grenoble, 38043
Chu Lille
Lille, Lille, 59800
Chu Limoges
Limoges, Limoges, 87042
AP-HM - Hôpital La Timone
Marseille, Marseille, 13385
Chu Montpellier
Montpellier, Montpellier, 34295
Chu Nantes
Nantes, Nantes, 44033
CHU NICE
Nice, Nice, 06202
AP-HP - Hôpital Necker
Paris, Paris, 75015
AP-HP - Hôpital Trousseau
Paris, Paris, 75571
CHU REIMS - American Memorial Hospital
Reims, Reims, 51092
Chu Rennes
Rennes, Rennes, 35000
Chu Rouen
Rouen, Rouen, 76031
Chu Saint Etienne
Saint-Etienne, Saint Etienne, 42055
Chu Strasbourg
Strasbourg, Strasbourg, 67098
Chu Toulouse
Toulouse, Toulouse, 31059
Chu Tours
Tours, Tours, 37044
Chu Nancy
Vandœuvre-lès-Nancy, Vandoeuvre Les Nancy, 54511
Belgium1 site
Queen Fabiola Universitary Children's Hospital
Brussels, Brussels Capital, 1020
About this study
Official title: A Prospective, Randomized, Double Blind, Placebo-controlled Phase II/III Study Evaluating the Efficacy of Rituximab in the Prevention of Relapse of Calcineurin Inhibitors Dependent Idiopathic Nephrotic Syndrome of Childhood
Background
Idiopathic nephrotic syndrome is a rare disease beginning during childhood and treated with immunosuppressants (i.e. steroids, mycophenolate mofetil, cyclophosphamide, cyclosporine).
Read more
Renal function of patients suffering from severe, steroid-dependent nephrotic syndrome with failure or toxic side effects of other immunosuppressant treatments is a major matter of concern.
Cyclosporine endangers renal parenchyma (fibrosis) in these patients who must take this treatment for years. At the same time, low doses of cyclosporine allow proteinuria to reappear, which provokes degradation of renal function by focal segmental glomerulosclerosis. Some recent data lead to the conclusion that Rituximab may be effective in such a disease, with a cyclosporin sparing effect.
Purpose
The aim of the study is to evaluate the efficacy of Rituximab versus placebo in the treatment of pediatric patients suffering from severe cyclosporine-dependent nephrotic syndrome.
Abstract Patients will be included in the study in a period of remission of proteinuria. Two infusions of Rituximab - at the dose of 375 mg/m²- or placebo will be administered at one week of interval. Other immunosuppressant treatments will be gradually tapered off with the same tapering pattern in both groups. In case of relapse of nephrotic syndrome, the blinding code will be broken. Rituximab will then be infused to patients having received placebo.
Read the detailed description
After infusions of Rituximab or placebo, patients will be examined by their nephrologist on a monthly basis during five months. Follow up will be focused on proteinuria, albuminemia, lymphocyte phenotyping and Rituximab pharmacokinetics
- Conditions
- Childhood Idiopathic Nephrotic Syndrome
- Being tested
- Rituximab, Placebo
- Keywords
- rituximab, idiopathic nephrotic syndrome, minimal change disease, focal and segmental glomerulosclerosis
- Medical subject headings
- Nephrotic Syndrome, Nephrosis, Lipoid, Glomerulosclerosis, Focal Segmental
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
RituximabDrug
two infusions - at the dose of 375 mg/m²- will be administered at one week of interval
PlaceboDrug
two infusions - at the dose of 375 mg/m² - will be administrered at one week of interval
| Study group | Role | What they receive |
|---|---|---|
| Rituximab | Experimental | two infusions of Rituximab - at the dose of 375 mg/m² Receives: Drug: Rituximab |
| placebo | Placebo comparator | two infusions of placebo Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proteinuria with relapse of nephrotic syndrome (Serum albumin < 30 g/L) within 5 months
Proteinuria with relapse of nephrotic syndrome (Serum albumin < 30 g/L) within 5 months
Measured: 5 months
Secondary outcomes (4)
- dosing of rituximab for toxicity during and/or after infusion
- toxicity during and/or after infusion
Measured: 5 months
- dosing of rituximab for pharmacokinetics
- dosing of rituximab for pharmacokinetics
Measured: 5 months
- dosing of lymphocyte
- lymphocyte phenotyping
Measured: 5 months
Pediatric Quality of life inventory
Pediatric Quality of life inventory
Measured: 5 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Dec 2010 (estimated)
- Study startDec 2010
- Primary completionMay 2014
- Study completionMay 2014
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 Childhood Idiopathic Nephrotic SyndromeStudies of the same condition, open and under way.
- More trials run by University Hospital, LimogesThe 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Hospital, Limoges
- Sponsor type
- University, hospital or other
- Collaborators
- Hoffmann-La Roche
- Responsible party
- Sponsor
- Investigators
- Vincent GUIGONIS, MD, Principal investigator, CHU Limoges
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01268033
- Sponsor's protocol ID
- I08013
- Acronym
- NEPHRUTIX
- Status last verified
- Mar 2015
- First submitted
- 15 Dec 2010
- Last updated
- 23 Mar 2015
- Results posted
- No
- Data monitoring committee
- No
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 23 Mar 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Randomized, Double Blind, Placebo-controlled Phase II/III Study Evaluating the Efficacy of Rituximab in the Prevention of Relapse of Calcineurin Inhibitors Dependent Idiopathic Nephrotic Syndrome of Childhood
- ClinicalTrials.gov ID
- NCT01268033
- Acronym / sponsor ID
- NEPHRUTIX / I08013
- Conditions
- Childhood Idiopathic Nephrotic Syndrome
- Keywords
- rituximab, idiopathic nephrotic syndrome, minimal change disease, focal and segmental glomerulosclerosis
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- University Hospital, LimogesUniversity, hospital or other
- Collaborators
- Hoffmann-La Roche
- Study start
- Dec 2010
- Primary completion
- May 2014 (actual)
- Study completion
- May 2014 (actual)
- First posted
- 29 Dec 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.
Background
Idiopathic nephrotic syndrome is a rare disease beginning during childhood and treated with immunosuppressants (i.e. steroids, mycophenolate mofetil, cyclophosphamide, cyclosporine).
Renal function of patients suffering from severe, steroid-dependent nephrotic syndrome with failure or toxic side effects of other immunosuppressant treatments is a major matter of concern.
Cyclosporine endangers renal parenchyma (fibrosis) in these patients who must take this treatment for years. At the same time, low doses of cyclosporine allow proteinuria to reappear, which provokes degradation of renal function by focal segmental glomerulosclerosis. Some recent data lead to the conclusion that Rituximab may be effective in such a disease, with a cyclosporin sparing effect.
Purpose
The aim of the study is to evaluate the efficacy of Rituximab versus placebo in the treatment of pediatric patients suffering from severe cyclosporine-dependent nephrotic syndrome.
Abstract Patients will be included in the study in a period of remission of proteinuria. Two infusions of Rituximab - at the dose of 375 mg/m²- or placebo will be administered at one week of interval. Other immunosuppressant treatments will be gradually tapered off with the same tapering pattern in both groups. In case of relapse of nephrotic syndrome, the blinding code will be broken. Rituximab will then be infused to patients having received placebo.
Detailed description
After infusions of Rituximab or placebo, patients will be examined by their nephrologist on a monthly basis during five months. Follow up will be focused on proteinuria, albuminemia, lymphocyte phenotyping and Rituximab pharmacokinetics
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Rituximab | Experimental | Drug: Rituximabtwo infusions of Rituximab - at the dose of 375 mg/m² |
| placebo | Placebo comparator | Drug: Placebotwo infusions of placebo |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Male or Female patients over 2 and under 18 years, with an idiopathic nephrotic syndrome (NS)
- Steroid Sensitive Nephrotic Syndrome (according to the French pediatric protocol).
- NEPHRUTIX
- Calcineurin inhibitor Dependent NS or NS for which anticalcineurin treatment has not been effective. Others immunosuppressive treatments (MMF) must have failed to control the disease activity.
- Effective contraception for girls of childbearing age.
Key exclusion criteria (6 listed)
- Terminal renal failure requiring dialysis/transplantation
- Transcutaneous oxygen stauration < 97%
- Clinical or Radiological brochopulmonar or pleural abnormality
- Asymptomatic carrier of Hepatitis B virus our history of Hepatitis B
- Contraindication to Rituximab (RTX)
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 |
|---|---|---|
| Rituximabtwo infusions - at the dose of 375 mg/m²- will be administered at one week of interval | Drug | Rituximab |
| Placebotwo infusions - at the dose of 375 mg/m² - will be administrered at one week of interval | Drug | placebo |
Primary outcome
Proteinuria with relapse of nephrotic syndrome (Serum albumin < 30 g/L) within 5 months
Time frame: 5 months
Proteinuria with relapse of nephrotic syndrome (Serum albumin < 30 g/L) within 5 months
4 secondary outcomes
- dosing of rituximab for toxicity during and/or after infusion
Time frame: 5 months
- dosing of rituximab for pharmacokinetics
Time frame: 5 months
- dosing of lymphocyte
Time frame: 5 months
Pediatric Quality of life inventory
Time frame: 5 months
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 23 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.
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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.
Vincent GUIGONIS, MD principal investigator
CHU Limoges
24 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Rituximab in people with Childhood Idiopathic Nephrotic Syndrome, run by University Hospital, Limoges at 24 sites in 2 countries.
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 6 inclusion and 6 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 23 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 24 sites in France, Belgium. 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. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Proteinuria with relapse of nephrotic syndrome (Serum albumin < 30 g/L) within 5 months (time frame: 5 months). 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 NCT01268033, 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 23 Mar 2015.