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.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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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

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.

Country

City, postcode or hospital

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

Other trials in France

Belgium1 site

Queen Fabiola Universitary Children's Hospital
Brussels, Brussels Capital, 1020

Other trials in Belgium

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).

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 groups in NCT01268033
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.

Dec 2010May 2014

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.
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)

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