Pilot Study of Natalizumab in the Treatment of Patients With Inclusion Body Myositis
试验速览
- 阶段
- 1 期
- 入组人数
- 6
- 试验地点
- 1
- 主要终点
- Determine if natalizumab is effective and safe in the treatment of patients with IBM (muscle biopsies)
研究概览
简要总结
Muscle biopsies of patients with Inclusion Body Myositis (IBM) have demonstrated a T-cell predominant inflammatory infiltrate, therefore, new agents targeting T -cell mediated cell death may be a novel treatment for IBM. Such an agent capable of preventing T-cell movement out of the vasculature, such as natalizumab, may be beneficial in IBM patients. Six patients will be recruited to participate in this phase I trial.
详细描述
This is a phase I open label, non-placebo controlled trial evaluating the safety and efficacy of natalizumab in patients with IBM. Pre and post treatment muscle biopsies including measurements of the degree of inflammation as well as the types of inflammatory cells will be measured. The investigators will also assess quality of life, the inclusion body myositis functional rating score (IBM-FRS), and patient and physician global impression of change. Manual muscle testing and quantitative dynamometry will also be evaluated to see if patient's strength improves.
Patients who are eligible to participate and have signed a consent form will have a muscle biopsy performed at baseline and at the end of the study. Physical and neurological exams, as well as IBM-FRS, safety labs will be conducted and collected at monthly visits. Patients will start natalizumab therapy at 300mg intravenously every 4 weeks for 24 weeks. All study related procedures will be conducted at Phoenix Neurological Associates, as well as all infusions and muscle biopsies
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 21 Years 至 85 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Definite diagnosis of sporadic IBM through previous muscle biopsies
- •Muscle function adequate for quantitative muscle testing
- •JC virus negative at screening
排除标准
- •Previous therapy with natalizumab.
- •Treatment with other immunosuppressive agents within the last 12 months
- •Quadriceps strength less than or equal to 2/5 at baseline
- •Known malignancy
- •Pregnancy or breastfeeding
- •History of abnormal laboratory results indicative of any significant medical disease that would preclude the use of natalizumab
- •Any clinically significant infectious illness in the 30 days before enrollment
研究组 & 干预措施
Natalizumab
Natalizumab therapy will be given at 300mg intravenously every 4 weeks for 24 weeks
干预措施: Natalizumab (Drug)
结局指标
主要结局
Determine if natalizumab is effective and safe in the treatment of patients with IBM (muscle biopsies)
时间窗: 12 months
muscle biopsies to determine inflammation
Determine if natalizumab is effective and safe in the treatment of patients with IBM (manual muscle testing)
时间窗: 12 months
MMT - manual muscle testing to determine strength
次要结局
- Secondary outcome measures will involve an assessment of pre and post treatment muscle biopsies including measurements of the degree of inflammation as well as the types of inflammatory cells. (blood levels)(12 months)
- Secondary outcome measures will involve an assessment of pre and post treatment muscle biopsies including measurements of the degree of inflammation as well as the types of inflammatory cells. (pain scores)(12 months)
- Secondary outcome measures will involve an assessment of pre and post treatment muscle biopsies including measurements of the degree of inflammation as well as the types of inflammatory cells. (functional rating score scale)(12 months)