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A European Network for randomised actively controlled clinical trials in pediatric rheumatic diseases : Parenteral methotrexate in low versus higher doses in juvenile chronic arthritis

Objective

Objectives: $LThe goal of this project is to provide a mechanism whereby new and promising approaches to the management of PRD, including rare conditions and complications, can be tested in a systematic and scientific manner. This goal will be the first effort of a European research network composed by 11 countries member of the European Economic Community. The main function of this network is to facilitate and coordinate the development, conduct, analysis, and reporting of Randomised Actively Controlled Clinical Trials (RACCT), in which all arms of the study receive active treatment with approved drugs.

Brief description:
The pediatric rheumatic diseases (PRD) are rare conditions, associated with substantial morbidity, and monetary costs. Drugs for the treatment of PRD are now being used in new dosages, new routes of administration, new combinations and for disease complications. There is little incentive for pharmaceutical companies to fund large-scale trials of these new approaches. Consequently, data regarding the safety and efficacy of these treatment regimens tends to be from anedoctal, small, uncontrolled, non-randomised case series. Moreover, the ethics and effectiveness of conducting any placebo-controlled trial, even in adults has recently come under intense debate.
This network will undertake only those studies which are not fundable through pharmaceutical industry or other sources. Each trial will be designed to build on the current "standard of care" in such a way that the cost of the medications, clinic visits, and laboratory monitoring, will be paid by the usual method for clinical care in each participating European State. The amount of data to be collected for these trials, in addition to that obtained for routine follow-up, will be minimal, and all participants will volunteer the effort. Importantly, studies of rare conditions or complications of disease in which patient recruitment may have to be extended over several years, would become possible. Work content:
a) A RACCT in children with juvenile chronic arthritis: parenteral methotrexate in low versus higher doses.$Lb) Cross-cultural adaptation, and validation, in up to 11 European countries of two health related quality of life instruments: the Childhood Health Assessment Questionnaire and the Childhood Health Questionnaire.$Lc) Development of a centralized computerized databank designed to be used as an international resource for the pediatric rheumatology community.$LMethodology: The approach taken in this project is fashioned after that of the Pediatric Oncology Group. The proposed trial, and nearly all future studies, would be considered appropriate for consideration by the network only if: 1) the new therapeutic approach is deemed to have legitimate promise of being effective and safe; 2) the trial will not be fundable by conventional means (i.e. pharmaceutical industry); and 3) the usual method of cost reimbursement for clinical care will bear the patient-related expenses. It is anticipated that most trials will be RACCT studies. Personnel at the coordinating centre, will assist principal investigators in protocol development, and provide the expertise and equipment to initiate the trial recruit subjects, gather and analyze data and report results.$L$LKeywords:$LJuvenile Chronic Arthritis, Methotrexate, Quality of Life, Randomised Controlled Clinical Trial.

Funding Scheme

CON - Coordination of research actions

Coordinator

IRCCS POLICLINICO SAN MATTEO - ISTITUTO DI RICOVERO E CURA A CARATTERE SCIENTIFICO
Address
P. Le Golgi 2
27100 Pavia
Italy