Étude de l'Effet de la Rapamycine Sur la Force Musculaire et la réponse Immunitaire au Cours de la Myosite à Inclusions: étude RAPAMI"
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 44
- 试验地点
- 1
- 主要终点
- stabilization of quadiceps strength measured by myometry
研究概览
简要总结
Sporadic Inclusion Body Myositis (IBM) is the most frequent inflammatory myopathy in patients over 50. It is a slowly progressive, but today untreatable (notably by classical immunosuppressants) disease.
Rapamycin used in organ transplantation blocks the activity of T effector cells, preserves T regulatory cells and induces autophagy (protein degradation), all parameters impaired during IBM.
RAPAMI is a prospective, randomised, controlled, double blind, monocentric, phase IIb trial evaluating rapamycine against placebo.
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, Outcomes Assessor)
入排标准
- 年龄范围
- 45 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •IBM defined by the Benveniste & Hilton-Jones ( Neuromuscul Disord. 2010;20: 414-21) or Llyod criteria (Neurology 2014; 83: 426-433)
排除标准
- •Impossiblility to walk 10 meters
- •Hypersensitivity to rapamycin or one compound of the oral solution
- •Severe respiratory insufficiency (FVC < 50% and/or FEV1 < 50%)
- •Severe chronic kidney disease (Estimated Glomerular Filtration Rate < 15 ml/min and/or proteinuria > 0.3 g/24h)
- •Chronic liver disease (cirrhosis and/or ALT/AST > 2.5 normal values)
- •Cancer non in remission (necessitating specific treatment) during the past 12 months
- •Connective Tissue Disease non in remission (necessitating specific treatment) during the past 12 months
- •Pregnancy
- •Seropositivity for HIV, HCV or HBV
- •Total cholesterolemia > 8 mmol/l
- •Triglyceridemia > 5 mmol/l
- •Hemoglobinemia < 11 g/dL
- •Thrombopenia < 100 000/mm3
- •Neutropenia < 1500/ mm3
- •Lymphopenia < 1000/ mm3
研究组 & 干预措施
Rapamycin
rapamycin 1 mg/ml oral solution, 2 mg/day (2 ml/day), once a day, during one year
干预措施: Rapamycin (Drug)
Placebo
Placebo oral solution, 2 ml/day, once a day, during one year
干预措施: Placebo (Drug)
结局指标
主要结局
stabilization of quadiceps strength measured by myometry
时间窗: 52 weeks
次要结局
- measure of inflammation by MRI(52 weeks)
- Quality of life by different scales(52 weeks)
- Measure of the tolerance(52 weeks)
- comparison of 6 minutes walking test(52 weeks)
- stabilization of hand grip strength measured by myometry(52 weeks)
- composite measure of the handicap(52 weeks)
- measures of muscle fatty replacement by MRI(52 weeks)