Systemic Lupus Erythematosus Nephritis Clinical Trial
Official title:
Five-Year Single-Blind, Phase III Effectiveness Randomised Actively Controlled Clinical Trial in New Onset Juvenile Systemic Lupus Erythematosus Nephritis: Oral Cyclophosphamide Versus High Dose Intravenous Cyclophosphamide Versus Intermediate Dose Intravenous Cyclophosphamide
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.
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.
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| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT03842787 -
Anti-ficolin-3 Antibodies in Lupus Nephritis
|