Five-year Actively Controlled Clinical Trial in New Onset Juvenile Dermatomyositis

• Status: Completed
• Trial end date: 2015-11-29
• Target enrollment: 0
• Participant gender: All

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

• Phase: Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Istituto Giannina Gaslini
• Collaborator: Pediatric Rheumatology International Trials Organization
• Treatments: Cyclosporine; Cyclosporins; Methotrexate; Methylprednisolone; Methylprednisolone acetate; Methylprednisolone Hemisuccinate; Prednisolone; Prednisolone acetate; Prednisolone hemisuccinate; Prednisolone phosphate; Prednisone

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.