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
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 139
- 试验地点
- 1
- 主要终点
- 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%.
研究概览
简要总结
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
详细描述
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 1 Year 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- 未提供
排除标准
- 未提供
研究组 & 干预措施
Methylprednisolone pulse (MPDN)+PDN+CSA
MPDN= methylprednisolone pulse PDN= prednisone or equivalent CSA= cyclosporine A
干预措施: 3 MPDN pulse + PDN + CSA (Drug)
MPDN+PDN+MTX
MPDN= methylprednisolone pulse PDN= prednisone or equivalent MTX= methotrexate
干预措施: 3 MPDN pulse + PDN + MTX (Drug)
MPDN+PDN
MPDN= methylprednisolone PDN= prednisone or equivalent
干预措施: 3 MPDN pulse + PDN (Drug)
结局指标
主要结局
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%.
时间窗: 6 months
The PRINTO Juvenile Dermatomyositis (JDM) core set variables are: 1. muscle strength by the mean of the Childhood Myositis Assessment Scale (CMAS); 2. physician's global assessment of disease activity on a 10 cm Visual Analogue Scale (VAS); 3. global disease activity assessment by the mean of the Disease Activity Index (DAS); 4. parent's/patient's global assessment of overall well-being on a 10 cm VAS; 5. functional ability assessment by the mean of the Childhood Health Assessment Questionnaire (CHAQ) 6. health-related quality of life assessment.
Time to Clinical Remission
时间窗: 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.
次要结局
- Time to Prednisone, or Equivalent, Discontinuation(60 months)
- Time to Major Therapeutic Changes(60 months)
研究者
Nicolino Ruperto, MD, MPH
Senior Scientist of PRINTO
Istituto Giannina Gaslini