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Sponsors and Collaborators: |
Istituto Giannina Gaslini Pediatric Rheumatology International Trials Organisation (PRINTO) |
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Information provided by: | Istituto Giannina Gaslini |
ClinicalTrials.gov Identifier: | NCT00323960 |
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 dermatomyositis (JDM): 5-year phase III single-blind, RCT in children with newly diagnosed JDM: prednisone (PDN) versus PDN plus methotrexate (MTX) versus PDN plus Cyclosporine A. The trial is aimed to find out the treatment regimen associated with the lowest occurrence of flare and the lowest drug related toxicity
Condition | Intervention | Phase |
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Juvenile Dermatomyositis |
Drug: Prednisone Drug: Cyclosporine A Drug: Methotrexate |
Phase III |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Open Label, Active Control, Parallel Assignment, Safety/Efficacy Study |
Official Title: | Five-Year Single-Blind, Phase III Effectiveness Randomised Actively Controlled Clinical Trial in New Onset Juvenile Dermatomyositis: Prednisone Versus Prednisone Plus Cyclosporine a Versus Prednisone Plus Methotrexate |
Estimated Enrollment: | 120 |
Study Start Date: | May 2006 |
Estimated Study Completion Date: | May 2011 |
Primary Completion Date: | December 2006 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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2: Active Comparator |
Drug: Cyclosporine A
5 mg/Kg/day in 2 oral doses
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3: Active Comparator |
Drug: Methotrexate
15-20 mg/m2 once per week
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1: Active Comparator |
Drug: Prednisone
2 mg/Kg/day
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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 46 countries (110 in 21 EU States and 75 in 25 extra-EU States), with a randomised clinical trials (RCT) in juvenile dermatomyositis (JDM): 5-year phase III single-blind, RCT in children with newly diagnosed JDM: prednisone (PDN) versus PDN plus methotrexate (MTX) versus PDN plus Cyclosporine A. The trial 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 JDM 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 JDM.
The proposed trial in JDM (prednisone [PDN] versus PDN plus methotrexate [MTX] versus PDN plus cyclosporine [CsA]), 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.
Ages Eligible for Study: | 1 Year to 18 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Newly diagnosed and untreated children with probable or definite diagnosis of JDM according to the Bohan and Peter criteria. If a muscle biopsy will be performed (optional) it will be read by the pathologists of the participating centres (light and immunofluorescence). Slides of paraffin-embedded sections from all patients will be re-viewed by a blinded myopathologist at the PRINTO coordinating centre.
Duly executed, written, informed consent obtained from the parents/patient.
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Exclusion Criteria:
Live vaccines not allowed during the entire duration of the trial.
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Contact: Nicolino Ruperto, MD, MPH | 0039-010-382854 | nicolaruperto@ospedale-gaslini.ge.it |
Contact: Anna Tortorelli, B.A. Hons | 0039-010-393425 | annatortorelli@ospedale-gaslini.ge.it |
Italy | |
Istituto Giannina Gaslini | Recruiting |
Genoa, Italy, 16147 |
Principal Investigator: | Nicolino Ruperto, MD, MPH | Istituto Giannina Gaslini _ PRINTO |
Study Chair: | Alberto Martini, MD, Prof. | Istituto Giannina Gaslini_PRINTO |
Responsible Party: | Paediatric Rheumatology International Trials Organization (PRINTO) ( Dr Nicola Ruperto ) |
Study ID Numbers: | IGG-PRINTO-002, AIFA, Myositis Association |
Study First Received: | May 9, 2006 |
Last Updated: | May 12, 2008 |
ClinicalTrials.gov Identifier: | NCT00323960 |
Health Authority: | Italy: Ministry of Health |
Juvenile dermatomyositis randomised actively controlled clinical trial prednisone |
cyclosporine methotrexate effectiveness |
Prednisone Juvenile dermatomyositis Cyclosporine Skin Diseases Clotrimazole Miconazole Tioconazole Cyclosporins Folic Acid |
Myositis Dermatomyositis Muscular Diseases Musculoskeletal Diseases Neuromuscular Diseases Polymyositis Connective Tissue Diseases Idiopathic myopathy Methotrexate |
Antimetabolites Anti-Inflammatory Agents Anti-Infective Agents Antimetabolites, Antineoplastic Immunologic Factors Molecular Mechanisms of Pharmacological Action Antineoplastic Agents Physiological Effects of Drugs Hormones, Hormone Substitutes, and Hormone Antagonists Reproductive Control Agents Hormones Therapeutic Uses Antifungal Agents |
Abortifacient Agents Dermatologic Agents Nucleic Acid Synthesis Inhibitors Antineoplastic Agents, Hormonal Nervous System Diseases Enzyme Inhibitors Abortifacient Agents, Nonsteroidal Folic Acid Antagonists Immunosuppressive Agents Glucocorticoids Pharmacologic Actions Antirheumatic Agents |