Five-year Actively Controlled Clinical Trial in New Onset Juvenile Dermatomyositis
Condition: Juvenile Dermatomyositis
MeSH terms: Dermatomyositis
Intervention: 3 MPDN pulse + PDN; 3 MPDN pulse + PDN + CSA; 3 MPDN pulse + PDN + MTX
Intervention description: 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.
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 139.0
Sex: All
Minimum age: 1 Year
Maximum age: 18 Years
Healthy volunteers: No
Keywords: Juvenile dermatomyositis; randomised actively controlled clinical trial; prednisone; cyclosporine; methotrexate; effectiveness

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
    

Eligibility criteria:

        Inclusion Criteria. Each patient must meet all the following criteria in order to
        participate in this trial:

          1. Newly diagnosed and untreated children (only treatment with 1 NSAID is allowed and/or
             prednisone >1 mg/kg/day for no more than 1 month from diagnosis) with probable or
             definite diagnosis of JDM according to published (12;13). 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 PRINTO.

          2. Age at enrolment ≤ 18 years.

          3. Female of child-bearing potential must have a negative pregnancy test at the beginning
             of the trial, and then every 3 months. If sexually active, they must agree to use
             adequate contraception, throughout study participation, and must have no intention of
             conceiving during the course of the study. Post-pubertal males must have no plans to
             father a child during the study and agree to use adequate birth control methods if
             sexually active.

          4. Ability to comply with the entire study procedures, ability to communicate
             meaningfully with the investigational staff, competence to give written informed
             consent; to be applied to the parents and/or patients, as appropriate

          5. Duly executed, written, informed consent obtained from the parents/patient.

        Exclusion Criteria. Any of the following will exclude a patient from this trial:

          1. Neutrophil count <1,500/mm3 and/or platelet count <50,000/mm3

          2. Demonstration of cutaneous or gastrointestinal ulceration of JDM related pulmonary
             disease or cardiomyopathy at the time of diagnosis.

          3. History of poor compliance.

          4. Evidence of current use of alcohol or illicit drugs abuse.

          5. Live vaccines not allowed during the entire duration of the trial.

        Dropout Criteria. Patients will be considered "treatment failures", and dropped from the
        trial but included in efficacy analysis, if any of the following will occur during the
        active period of the trial.

          1. Non compliance with study medication administration

          2. Enrolment in other therapeutic trials.