A Multi-centre, Double-blind, Placebo Controlled, Proof of Concept Study to Evaluate the Efficacy and Tolerability of BAF312 in Patients With Polymyositis and Dermatomyositis
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 18
- 试验地点
- 1
- 主要终点
- Number of Participants Who Responded to BAF312
研究概览
简要总结
This study determined the efficacy, safety, tolerability and the PK profile of BAF312, a novel immunomodulator, in polymyositis and dermatomyositis patients who were not responsive to traditional immunosuppressive and/or corticosteroid therapy. The study consisted of a 12 week, randomized, placebo controlled period, followed by another 12 weeks where all subjects received BAF312 treatment.
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
- Double (Participant, Investigator)
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Patients with disease at least 3 months before study
- •Muscle weakness
- •Received corticosteroids with or with out disease modifying antirheumatic drugs at least 3 months before study however not responding to this therapy
排除标准
- •Other idiopathic inflammatory myopathies
- •Myopathy other than polymyositis and dermatomyositis
- •Patients with late stages of disease
研究组 & 干预措施
Placebo
干预措施: Placebo (Drug)
BAF312
干预措施: BAF312 (Drug)
结局指标
主要结局
Number of Participants Who Responded to BAF312
时间窗: 12 weeks
Preliminary clinical efficacy of BAF312 in patients with Polymyositis and dermatomyositis (PM/DM) using the International Myositis Assessment and Clinical Studies Group (IMACS) core set measures (including manual muscle testing, Physician's Global Activity Assessment (on a horizontal 10 cm visual analogue scale), Patient Global Activity Assessment (on a horizontal 10 cm visual analogue scale), Physical Function (Health Assessment Questionnaire), Muscle-associated Enzymes (CK, LDH, AST, ALT, aldolase) and Extra-Muscular Activity Assessment (Extra-muscular portion of Myositis Disease Activity Assessment Tool).
次要结局
- Number of Participants With a Change in Steroids Use After BAF312 Administration -Period 2(12 weeks)
- Summary of CRP Levels(12 weeks)
- Myositis Disease (MD) Activity Scores(Week 12)
- Health Assessment Questionnaire(Baseline, Week 12)
- Serum Levels of Muscle Enzymes(Baseline, Week 12)
- Mean Plasma Concentrations of BAF312(baseline to end of trial (day 196))
- Physician Global Activity Assessment(Baseline, Week 12)
- Patient Global Activity Assessment(Baseline, Week 12)
- Efficacy in Modifying Health-related Quality of Life Measured by SF-36(12 weeks)
- Manual Muscle Testing (MMT) - 8 Score(Baseline, Week 12)