Phase 3
Completed N=139
Five-year Actively Controlled Clinical Trial in New Onset Juvenile Dermatomyositis
Source: ClinicalTrials.gov NCT00323960 ↗Enrolled (actual)
139
Serious AEs
5.8%
Results posted
Mar 2021
Primary outcomePrimary: Responder Status Defined as 20% Improvement in at Least 3 Core Set Variables With no More Than 1 of the Remaining Variables, (Muscle Strength Excluded), Worsened by > 30%. — 24; 32; 33 Participants — p=0.0228
◆ Published Evidence
Highly cited
232citations · ~23 / year
Prednisone versus prednisone plus ciclosporin versus prednisone plus methotrexate in new-onset juvenile dermatomyositis: a randomised trial.
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
Linked Publications (3)
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Prednisone versus prednisone plus ciclosporin versus prednisone plus methotrexate in new-onset juvenile dermatomyositis: a randomised trial.
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The PRINTO juvenile dermatomyositis trial.
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The PRINTO juvenile dermatomyositis trial - Authors' reply.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Responder Status Defined as 20% Improvement in at Least 3 Core Set Variables With no More Than 1 of the Remaining Variables, (Muscle Strength Excluded), Worsened by > 30%. |
24; 32; 33 | 0.0228 sig |
| PRIMARY Time to Clinical Remission |
5.99; 4.95; 13.39 | 0.012 sig |
| SECONDARY Time to Major Therapeutic Changes |
30.52; 17.52; 13.92 | 0.009 sig |
| SECONDARY Time to Prednisone, or Equivalent, Discontinuation |
15.91; 27.82; 24.42 | 0.002 sig |
Eligibility Criteria
Inclusion Criteria. Each patient must meet all the following criteria in order to participate in this trial:
- 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.
- Age at enrolment ≤ 18 years.
- 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.
- 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
- Duly executed, written, informed consent obtained from the parents/patient.
Exclusion Criteria. Any of the following will exclude a patient from this trial:
- Neutrophil count <1, 500/mm3 and/or platelet count <50,000/mm3
- Demonstration of cutaneous or gastrointestinal ulceration of JDM related pulmonary disease or cardiomyopathy at the time of diagnosis.
- History of poor compliance.
- Evidence of current use of alcohol or illicit drugs abuse.
- 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.
- Non compliance with study medication administration
- Enrolment in other therapeutic trials.
Data sourced from ClinicalTrials.gov (NCT00323960) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.