A Multi-centre Double-blind, Placebo Controlled, Proof of Concept Study to Evaluate the Efficacy and Tolerability of BAF312 in Patients With Polymyositis
试验速览
- 阶段
- 2 期
- 状态
- 终止
- 入组人数
- 14
- 试验地点
- 1
- 主要终点
- Change From Baseline at Week 12 for BAF312 2 mg, 10 mg or Placebo (Once Daily) for Combined Efficacy Endpoint: Manual Muscle Testing in 24 Muscles (MMT24)
研究概览
简要总结
This study assessed the efficacy, safety and tolerability of BAF312 administered orally in patients with clinically active polymyositis and also in patients with polymyositis who had shown inadequate response to corticosteroids and or DMARDs (disease modifying antirheumatic drugs).
详细描述
This study was stopped prematurely due to overall slow recruitment and no evidence for efficacy in a parallel study in dermatomyositis with an assumed similar pathophysiology. With very small sample sizes per group the overall results for this study including primary and all other efficacy and PD data are inconclusive
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
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
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
- •"definite" or "probable" for polymyositis at least three months before Baseline
- •active disease as defined by elevated CK levels, or other enzymes, or MRI/biopsy if enzymes are normal, and persisting muscle weakness
- •stable dose of corticosteroid for at least 2 weeks prior to Baseline and should not have received a medium or high dose in the last 8 weeks prior to study entry.
- •patients treated with methotrexate must have been on a stable dose for at least 6 weeks prior to Baseline.
排除标准
- •Patients with overlap polymyositis, late-stage polymyositis, or other types of myositis.
- •Preexisting severe cardiac or pulmonary involvement, malignancy of any organ system or significant eye diseases.
- •Uncontrolled diabetes mellitus or diabetes complicated with organ involvement.
- •Pregnant or nursing (lactating) women
研究组 & 干预措施
Placebo
5 placebo tablets daily during non-titration phase
干预措施: Placebo (Drug)
BAF312 2mg
1 tablet of BAF312 2 mg + 4 tablets of Placebo daily during non-titration phase
干预措施: BAF312 (Drug)
BAF312 10 mg
5 tablets of BAF312 2 mg daily during non-titration phase
干预措施: BAF312 (Drug)
结局指标
主要结局
Change From Baseline at Week 12 for BAF312 2 mg, 10 mg or Placebo (Once Daily) for Combined Efficacy Endpoint: Manual Muscle Testing in 24 Muscles (MMT24)
时间窗: Baseline, at 12 weeks
Manual Muscle Testing Scoring Sheet: Neck flexors, neck extensors and other designated muscles bilaterally (Biceps brachii, Deltoid middle, Quadriceps, Gluteus maximus, Gluteus medius, Trapezius, Iliopsoas, Hamstrings, Wrist extensors, Wrist Flexors, Ankle plantar flexors and Ankle dorsiflexors) were tested on a 0-10 scale by the Investigator. Posterior credibility interval from Bayesian analysis displayed as confidence interval. The scores range was 0 to 260. Higher scores indicate better outcome.
Percent Change From Baseline at Week 12 for BAF312 2 mg, 10 mg or Placebo (Once Daily) Serum Creatine Kinase (CK) Levels
时间窗: Baseline, at 12 weeks
Serum creatine kinase (CK) were analyzed as part of the blood chemistry panel. Posterior credibility interval from Bayesian analysis displayed as confidence interval. The variable CK was log-transformed for statistical analysis and after estimation was converted to percent change from baseline divided by the mean baseline
次要结局
- Six-minute Walking Distance (6MWD) at Week 12(Baseline, 12 weeks)
- Six-minute Walking Distance (6MWD) at Week 24(Baseline, 24 weeks)
- BAF312 Trough Plasma Concentrations (PK Set)(-7 Baseline, day 28, 56, 84)