Five-year Actively Controlled Clinical Trial in New Onset Juvenile Dermatomyositis (PRINTOJDMTR)
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|ClinicalTrials.gov Identifier: NCT00323960|
Recruitment Status : Completed
First Posted : May 10, 2006
Results First Posted : March 16, 2021
Last Update Posted : March 16, 2021
|Condition or disease||Intervention/treatment||Phase|
|Juvenile Dermatomyositis||Drug: 3 MPDN pulse + PDN Drug: 3 MPDN pulse + PDN + CSA Drug: 3 MPDN pulse + PDN + MTX||Phase 3|
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.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||139 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|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|
|Actual Study Start Date :||May 31, 2006|
|Actual Primary Completion Date :||May 12, 2011|
|Actual Study Completion Date :||November 29, 2015|
Active Comparator: Methylprednisolone pulse (MPDN)+PDN+CSA
MPDN= methylprednisolone pulse PDN= prednisone or equivalent CSA= cyclosporine A
Drug: 3 MPDN pulse + PDN + CSA
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
Active Comparator: MPDN+PDN+MTX
MPDN= methylprednisolone pulse PDN= prednisone or equivalent MTX= methotrexate
Drug: 3 MPDN pulse + PDN + MTX
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.
Active Comparator: MPDN+PDN
MPDN= methylprednisolone PDN= prednisone or equivalent
Drug: 3 MPDN pulse + PDN
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.
- 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%. [ Time Frame: 6 months ]
The PRINTO Juvenile Dermatomyositis (JDM) core set variables are:
- muscle strength by the mean of the Childhood Myositis Assessment Scale (CMAS);
- physician's global assessment of disease activity on a 10 cm Visual Analogue Scale (VAS);
- global disease activity assessment by the mean of the Disease Activity Index (DAS);
- parent's/patient's global assessment of overall well-being on a 10 cm VAS;
- functional ability assessment by the mean of the Childhood Health Assessment Questionnaire (CHAQ)
- health-related quality of life assessment.
- Time to Clinical Remission [ Time Frame: 60 months ]Clinical remission is defined as the status of inactive disease for at least 6 continuous months defined as normal muscle strength (CMAS equal to 52) and physician global assessment of disease activity equal to 0.
- Time to Major Therapeutic Changes [ Time Frame: 60 months ]Time to major therapeutic changes is defined as the addition of CSA or MTX or any other disease-modifying antirheumatic drug (DMARS) in any of the 3 groups or discontinuation of assigned therapy for any reason including adverse events. Retention on treatment was used as main measure of effectiveness.
- Time to Prednisone, or Equivalent, Discontinuation [ Time Frame: 60 months ]Prednisone or equivalent glucocorticoid discontinuation is defined as the complete discontinuation of glucocorticoids
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00323960
|Istituto Giannina Gaslini|
|Genoa, Italy, 16147|
|Principal Investigator:||Nicolino Ruperto, MD, MPH||Istituto Giannina Gaslini _ PRINTO Senior Scientist|
|Study Chair:||Alberto Martini, MD, Prof.||Istituto Giannina Gaslini_PRINTO Chairman|