Five-Year Actively Controlled Clinical Trial in New Onset Juvenile Systemic Lupus Erythematosus Nephritis

Last updated: March 19, 2018
Sponsor: Istituto Giannina Gaslini
Overall Status: Trial Not Available

Phase

3

Condition

Lupus

Lupus Nephritis

Cutaneous Lupus Erythematosus

Treatment

N/A

Clinical Study ID

NCT00336414
IGG-PRINTO-003
  • Ages 1-18
  • All Genders

Study Summary

This is a 5-year project, involving 185 partners from 46 countries (110 in 21 EU States and 75 in 25 extra-EU States), with a randomised clinical trials (RCT) in juvenile systemic lupus erythematosus (JSLE): 5-year phase III single-blind, RCT in children with newly diagnosed, WHO class III, IV JSLE proliferative nephritis: PDN and oral cyclophosphamide (CYC) versus high dose intravenous (iv) CYC versus intermediate dose iv CYC, followed by maintenance with azathioprine.The trial is aimed to find out the treatment regimen associated with the lowest occurrence of flare and the lowest drug related toxicity.

Eligibility Criteria

Inclusion

Inclusion Criteria: Newly diagnosed children with untreated and biopsy proven revised WHO Class III, IVproliferative lupus nephritis and 24 hour proteinuria ≥ 500 mg/day. The kidney biopsyspecimen will be read by the renal pathologists of the participating centres (light andimmunofluorescence) (54). Slides of paraffin-embedded sections from all patients will bere-viewed by a blinded a renal pathologist at the PRINTO coordinating centre. Diagnosis of JSLE according to the ACR revised classification criteria (57); Age atenrolment ≤ 18 years. Female of child-bearing potential must have a negative pregnancy testat the beginning of the trial, and then every 3 months. If sexually active, they must agreeto use adequate contraception, throughout study participation, and must have no intentionof conceiving during the course of the study. Post-pubertal males must have no plans tofather a child during the study and agree to use adequate birth control methods if sexuallyactive. Ability to comply with the entire study procedures, ability to communicate meaningfullywith the investigational staff, competence to give written informed consent; to be appliedto the parents and/or patients, as appropriate Duly executed, written, informed consentobtained from the parents or other legal representative and/or the patient according torequirement of the local ethics committee.

Exclusion

Exclusion Criteria: Treatment with the CYC, AZA or mycophenolate mofetil anytime before randomisation. Neutrophil count <1,500 cell/mm3 and/or platelet count <50,000/mm3. History of poorcompliance with previous treatment. Evidence of current use of alcohol or illicit drugsabuse. Live vaccines not allowed during the entire duration of the trial.

Study Design

Study Start date:
June 01, 2006
Estimated Completion Date:
June 30, 2007

Study Description

Scientific objectives: The proposed project is aimed to improve treatment approaches for rare, severe and disabling paediatric rheumatic diseases (PRD). This goal will be achieved by the Paediatric Rheumatology International Trials Organisation (PRINTO) an international network whose main function is to provide a scientific base for current PRD treatments for which no evidence based data exist in the literature, and for drugs for which there is no support from industries.

This is a 5-year project, involving 185 partners from 46 countries (110 in 21 EU States and 75 in 25 extra-EU States), with a randomised clinical trials (RCT) in juvenile systemic lupus erythematosus (JSLE): 5-year phase III single-blind, RCT in children with newly diagnosed, WHO class III, IV JSLE proliferative nephritis: PDN and oral cyclophosphamide (CYC) versus high dose intravenous (iv) CYC versus intermediate dose iv CYC, followed by maintenance with azathioprine. The JSLE RCT is aimed to find out the treatment regimen associated with the lowest occurrence of flare and the lowest drug related toxicity. The retention on treatment will be used as main measure of effectiveness.

Methodology: The present protocol is the natural follow up of previous work conducted by PRINTO. In particular the RCT foreseen in this protocol is modelled after the successful completion of an early phase trial with MTX in juvenile idiopathic arthritis, and will use validated JSLE outcome measures for the evaluation of response to therapy.

It is the basic premise of this protocol that, without i) the involvement of the international paediatric rheumatology community, ii) the innovative type of mechanism described herein, these studies would never be conducted.

Objectives. The goals of the current protocol is therefore the natural follow-up of the objectives achieved with the previous grants and, in particular, of projects designed to discern new models for the successful conduct of clinical trials in children with rare diseases, and to develop standardized and validated measures for the evaluation of response to therapy in JSLE.

The proposed trials in in JSLE (oral cyclophosphamide [CYC] versus intermediate dose intravenous [iv] CYC versus high dose iv CYC) followed by maintenance therapy with azathioprine [AZA]), should serve as a model for the successful running of early phase clinical trials for severe and disabling rare diseases of childhood. The ultimate aim of these trials is to provide evidence-based information about the clinical utility of drugs in the management of rare paediatric conditions.

Connect with a study center

  • Istituto Giannina Gaslini

    Genoa, 16148
    Italy

    Site Not Available

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