Five-year Actively Controlled Clinical Trial in New Onset Juvenile Dermatomyositis
Condition: Juvenile Dermatomyositis
MeSH terms: Dermatomyositis
Study type: Interventional
Phase: Phase 3

Summary: 
      This is a 5-year project, involving 185 partners from 46 countries ((110 in 21 European Union
      (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
    

Detailed 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 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 (CsA). 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.
    

Intervention name(s): 3 MPDN pulse + PDN; 3 MPDN pulse + PDN + CSA; 3 MPDN pulse + PDN + MTX
Intervention type(s): Drug; Drug; Drug
Intervention description(s): 3 methylprednisolone pulses followed (30 mg/kg/pulse max 1 gram) followed by prednisone 2 mg/Kg/day to be tapered to 0.2 mg/kg/day in 6 months and then discontinued in 2 years.; 3 methylprednisolone pulses followed (30 mg/kg/pulse max 1 gram) followed by prednisone 2 mg/Kg/day to be tapered to 0.2 mg/kg/day in 6 months and then discontinued in 2 years; Cyclosporine 5 mg/Kg/day in 2 oral doses; 3 methylprednisolone pulses followed (30 mg/kg/pulse max 1 gram) followed by prednisone 2 mg/Kg/day to be tapered to 0.2 mg/kg/day in 6 months and then discontinued in 2 years; Methotrexate 15-20 mg/m2 once per week. Patients treated with MTX will receive concomitant folic or folinic acid according to the attending physician decision.
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Primary purpose: Treatment
Keywords: Juvenile dermatomyositis; randomised actively controlled clinical trial; prednisone; cyclosporine; methotrexate; effectiveness